Bersama Kita Sehat - : Biotechnology https://edusehat.com/en/rss/category/Biotechnology-136 Bersama Kita Sehat - : Biotechnology en 2025-2055 PS Global Media - Hak Cipta Astrocytes Preserve Memory Persistence Through Ankyrin-2 Protein in Mice https://edusehat.com/en/astrocytes-preserve-memory-persistence-through-ankyrin-2-protein-in-mice https://edusehat.com/en/astrocytes-preserve-memory-persistence-through-ankyrin-2-protein-in-mice Wed, 08 Jul 2026 22:10:09 +0700 Edusehat Astrocytes, Preserve, Memory, Persistence, Through, Ankyrin-2, Protein, Mice Although scientists have long studied how memories are formed in the brain, how certain memories persist over time for learning and cognitive function remains unclear. 

A new study published in Nature Communications titled, “Astrocytic ankyrin-2 enables memory persistence in the mouse hippocampus,” suggests that astrocytes play a critical role in long-term memory through the regulatory protein ankyrin-2 (Ank2). 

Removing Ank2 function led to significantly impaired memory in mice after after two weeks. Under normal conditions, these mice showed standard locomotion, sociability, and recent memory immediately after learning.  

Astrocytes lacking Ank2 formed significantly less physical contacts with nearby engram neurons, the specialized neurons for memory storage. Additionally, the maintenance of long-term potentiation (LTP) was impaired while normal synaptic transmission remained intact. The findings suggest that astrocytes stabilize the neural circuits required for preserving memories long after they are formed. 

On the molecular level, researchers found that Ank2 is required for brain-derived neurotrophic factor (BDNF) signaling through the astrocytic TrkB.T1 receptor and IP3R2-mediated calcium signaling. In the absence of Ank2, calcium signaling weakened, astrocytes failed to undergo normal structural remodeling, and showed reduced ability to maintain contacts with memory-encoding neurons. 

The researchers further demonstrated that hippocampal BDNF infusion normally strengthens long-term memory persistence, but this effect disappeared when astrocytic Ank2 was deleted, showing that Ank2 is essential for BDNF-dependent memory stabilization. 

To determine whether astrocytic BDNF signaling alone is sufficient to enhance memory, the team developed an optogenetic tool called Opto-T1. Activation of this pathway promoted astrocyte remodeling, maintained long-term potentiation, and significantly enhanced remote memory without affecting recent memory.  

“Our findings show that astrocytes are not passive support cells, but active regulators that determine how long memories last,” said Wuhyun Koh, PhD, senior research fellow at Institute for Basic Science (IBS) and corresponding author of the study. “By identifying Ank2 as a key regulator of astrocyte remodeling and BDNF signaling, we have uncovered a new mechanism that helps stabilize long-term memories and opens new avenues for understanding and potentially treating memory disorders.” 

The researchers indicate the study provides a new framework for understanding how astrocytes contribute to neurological diseases. 

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]]> Germline‑Targeting HIV Vaccine Generates Broadly Neutralizing Antibodies in Primates https://edusehat.com/en/germlinetargeting-hiv-vaccine-generates-broadly-neutralizing-antibodies-in-primates https://edusehat.com/en/germlinetargeting-hiv-vaccine-generates-broadly-neutralizing-antibodies-in-primates Wed, 08 Jul 2026 11:25:10 +0700 Edusehat Germline‑Targeting, HIV, Vaccine, Generates, Broadly, Neutralizing, Antibodies, Primates For years, HIV has resisted traditional vaccine strategies. The virus’s staggering antigenic diversity, rapid mutation rate, and glycan‑shielded envelope have made it extraordinarily difficult for the immune system to generate antibodies capable of recognizing HIV’s vulnerable sites. Yet a small number of people living with HIV do develop broadly neutralizing antibodies (bnAbs)—rare antibodies that can target conserved regions of the virus despite its shape‑shifting defenses. These bnAbs have long been viewed as templates for next‑generation vaccine design, but reliably eliciting them through vaccination has remained out of reach.

A team led by scientists at La Jolla Institute for Immunology (LJI) and Scripps Research now reports a potential breakthrough. In a study published in Nature, the researchers demonstrated that a germline‑targeting HIV vaccine can elicit bnAbs in outbred nonhuman primates. The study is titled, “Vaccination elicits HIV broadly neutralizing antibodies in primates.”

Germline targeting represents a fundamentally different vaccine design philosophy. As the authors wrote, it is “a conceptually radical vaccine design approach to elicit bnAbs, aiming to prime rare bnAb‑precursor B cells possessing pre‑determined human genetic and structural features shared with template bnAbs, and then guide B cell affinity maturation to potent bnAb evolution with heterologous boosters.”

To test this strategy, the team engineered protein immunogens that mimic key HIV envelope structures known to initiate bnAb development. Rhesus macaques received a priming immunogen designed to activate naive B cells, followed by a sequence of booster shots that guided those cells through the necessary maturation steps. “This series of vaccinations will guide, or ‘walk,’ a B cell from its naive state to its broadly neutralizing state,” explained co-first author and LJI instructor Patrick Madden, PhD.

The researchers reported that bnAb‑class memory B cells emerged in at least half of the animals, and “serum bnAb activity developed in 44% of animals.” In the strongest responder, bnAb titers reached titers “expected to confer protection against diverse HIV isolates,” according to the authors.

Human translation is already underway. The priming immunogen used in this study has been evaluated in the HVTN 144 trial and is currently being tested in the Phase I IAVI G004 trial. Shane Crotty, PhD, LJI professor and CSO, noted that the approach may perform even better in humans due to immunogenetic factors.

The next challenge is optimization—refining booster sequences, improving response rates, and ultimately demonstrating protection. But this study provides long‑sought proof of principle, according to the authors: “Germline-targeting vaccines can reproducibly elicit prespecified classes of bnAbs to prespecified epitopes under endogenous conditions, supporting further optimization of this approach for HIV vaccine development.”

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]]> Novartis to Acquire Myricx Bio for Up to $1.5B, Adding Cancer-Fighting ADC Payload Platform https://edusehat.com/en/novartis-to-acquire-myricx-bio-for-up-to-15b-adding-cancer-fighting-adc-payload-platform https://edusehat.com/en/novartis-to-acquire-myricx-bio-for-up-to-15b-adding-cancer-fighting-adc-payload-platform Wed, 08 Jul 2026 11:25:09 +0700 Edusehat Novartis, Acquire, Myricx, Bio, for, 1.5B, Adding, Cancer-Fighting, ADC, Payload, Platform Novartis has agreed to acquire Myricx Bio, a London-based developer of next-generation antibody-drug conjugates (ADCs), for up to $1.5 billion in a deal designed to bolster the buyer’s oncology pipeline with a next-generation ADC payload platform designed to fight cancer.

Privately-held Myricx specializes in developing ADCs that use N-myristoyltransferase inhibitor (NMTi) payloads, an approach designed to deliver a differentiated cancer-killing payload directly to tumor cells. Myricx says its ADC approach holds the potential to address limitations of TOPO-1 inhibitors, tubulin inhibitors, and other commonly used ADC payload classes—ranging from toxicity to healthy cells, to tumor resistance, to dose-limiting adverse events.

The acquisition deal is designed to combine Myricx’s two lead ADC assets and next-generation first-in-class NMTi payload platform with Novartis’ expertise in developing cancer therapies.

According to Myricx Bio, preclinical data suggests that its NMTi payload may have broad activity across multiple solid tumors, including TOPO-1-resistant models, and may enable more effective use of ADCs in settings where existing payload classes have limitations. NMT is an enzyme responsible for the addition of myristic acid, a 14-carbon fatty acid, to the N-terminus of multiple proteins that are crucial for cancer cell survival.

“ADCs have become an important part of cancer treatment, but there remains a clear need for new payload mechanisms to overcome resistance and expand their impact for patients,” Fiona Marshall, PhD, Novartis’ president of biomedical research, said in a statement. “Myricx Bio has developed a promising NMTi payload platform with a differentiated mechanism that could broaden the use of ADCs across multiple tumor settings.”

Marshall added that the Myricx Bio acquisition “reflects our strategy to scale innovative platforms, as we have with radioligand therapies, to deliver more durable, transformative treatments for patients.” In February, Novartis announced plans to build a 46,000-square-foot radioligand therapy (RLT) manufacturing site in the Dallas-Fort Worth suburb of Denton, TX.

Novartis investors reacted by sending its shares traded on the SIX Swiss exchange down 2% Monday, from CHF 127.92 ($157.69) to CHF 125.10 ($154.22). Novartis’ American Depositary Shares (ADSs) traded on the New York Stock Exchange dipped 3%, from $159.90 to $155.08 as of 10:13 a.m. ET

Mohit Rawat, Myricx Bio’s CEO

Until now, Myricx has said little about its two lead NMTi-ADC candidates, except to disclose on its website that it is prioritizing one that targets B7-H3 and the other, HER2, “based on compelling preclinical efficacy and safety data across multiple solid tumor-associated antigens and cancer cell types.”

Novartis agreed to shell out $1.1 billion cash upfront plus up to $400 million tied to achieving milestones. The transaction is expected to close in the second half of this year, subject to satisfaction or waiver of customary closing conditions, including regulatory approvals.

“Transformative promise”

“We are delighted that Novartis recognizes the transformative promise of our NMTi-ADC platform to deliver this next-generation of potential first-in-class, highly differentiated ADC therapeutics,” stated Mohit Rawat, Myricx Bio’s CEO. “Together with Novartis, we look forward to building upon our work to transform the landscape of cancer treatment.”

Rawat joined Myricx last year with the goal of steering the company through preclinical development and its next stage of growth.

Founded in 2019, Myricx Bio was spun out from Imperial College London and the Francis Crick Institute by Ed Tate, PhD; Roberto Solari, PhD; and Andrew Bell, PhD, with support from Cancer Research UK, as well as seed investment from Brandon Capital and Sofinnova Partners.

Myricx’s co-founders and their collaborative teams discovered that NMT played a vital role in maintaining multiple critical, diverse cellular processes in cancer cells, including vesicle trafficking, growth factor signaling, cancer cell survival, mitochondrial biogenesis, and cancer cell metabolism.

Under CTO Robin Carr, PhD, Myricx Bio raised £90 million ($114 million) in a Series A financing in mid-2024 led by Novo Holdings and Abingworth, joined by British Business Bank, Cancer Research Horizons, Eli Lilly, and existing investors. This enabled the company to scale its operations and expand the team to rapidly advance its pipeline.

The planned acquisition of Myrocx Bio is Novartis’ third major deal this year focused on boosting its cancer pipeline.

On June 24, Antares Therapeutics announced it would receive $105 million upfront from Novartis through a strategic collaboration to discover, develop, and commercialize small molecule therapies against promising but historically undruggable oncology targets. Novartis also committed to paying Antares up to $1.8 billion tied to achieving additional option exercise, development, regulatory, and commercial milestones, as well as tiered royalties on global net sales.

And in March, Novartis committed up to $2 billion upfront toward acquiring Pikavation Therapeutics, a subsidiary of Synnovation Therapeutics that specializes in developing PI3Kα inhibitor programs designed to treat forms of cancer. Novartis also agreed to pay up to $1 billion in payments tied to achieving development, regulatory, and commercial milestones.

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]]> Vertex Eyes Expansion Beyond Cystic Fibrosis with Planned $10B Crinetics Buyout https://edusehat.com/en/vertex-eyes-expansion-beyond-cystic-fibrosis-with-planned-10b-crinetics-buyout https://edusehat.com/en/vertex-eyes-expansion-beyond-cystic-fibrosis-with-planned-10b-crinetics-buyout Wed, 08 Jul 2026 07:50:07 +0700 Edusehat Vertex, Eyes, Expansion, Beyond, Cystic, Fibrosis, with, Planned, 10B, Crinetics, Buyout Vertex Pharmaceuticals has agreed to acquire Crinetics Pharmaceuticals for $10 billion cash, the companies said, in a deal that would expand the buyer’s rare disease portfolio beyond its anchor indication of cystic fibrosis (CF), by adding an approved treatment and a pipeline anchored by two Phase III candidates, all predicted to generate more than $5 billion in annual revenue.

Based in San Diego, Crinetics focuses on discovering, developing, and commercializing therapeutics for endocrine diseases. The company’s first marketed drug Palsonify® (paltusotine), an oral SST2 agonist, was approved by the FDA in September as the first and to date only once-daily oral therapy for adults with acromegaly, a debilitating condition which affects an estimated 20,000 Americans. Palsonify won European Commission approval in April and is under review by regulators elsewhere in the world.

Palsonify has enjoyed rapid uptake among acromegaly patients, with Crinetics reporting the drug generated net product revenue of $10.3 million during the first quarter, with 232 patients enrolling for treatment. Approximately 70% of patients treated with Palsonify at the end of Q1 were on reimbursed therapy—reflecting payers increasingly agreeing to cover the treatment, according to the company.

Within the first two quarters of its U.S. launch, Palsonify was prescribed by 263 unique healthcare providers.

Under its generic name paltusotine, the drug is in Phase III study for a second indication of carcinoid syndrome, a rare condition resulting from neuroendocrine tumors.

‘Excellent strategic fit’

Reshma Kewalramani, MD, Vertex Pharmaceuticals CEO and President

“Crinetics is an excellent strategic fit for Vertex, with its focus on serious diseases in specialty markets with significant unmet need, well-understood causal human biology, and potentially best-in-class medicines that could deliver transformative benefit to patients,” Reshma Kewalramani, MD, Vertex’s CEO and president, said in a statement. “We believe Vertex can build on the strong momentum of the Palsonify launch by applying our experience in commercializing medicines for rare genetic diseases.”

Crinetics investors agreed, roaring their approval of the pending acquisition as the company’s shares all but doubled in early trading Tuesday, zooming 99% to $83.54 as of 10:28 am ET from yesterday’s closing price of $42.03. Vertex shares dipped 2% to $516.48 from $529.59 at Monday’s closing bell.

Also in late-stage development is Crinetics’ lead pipeline candidate atumelnant, an oral adrenocorticotropic hormone (ACTH) antagonist now under development for congenital adrenal hyperplasia (CAH) and ACTH-dependent Cushing’s syndrome.

In classic CAH, a rare chronic genetic disease with 17,000 addressable patients in the U.S., atumelnant is in a pair of clinical trials. One is a Phase III study in adults with the most common cause of the disease, 21-hydroxylase deficiency (21-OHD). The study’s estimated primary completion date is May 2027 (NCT07144163). The other trial is a Phase II/III study in children ages one to <18, which has an estimated primary completion date of March 2030 (NCT07159841).

Earlier Phase II studies of atumelnant showed that patients treated with the therapy achieved near normalization of excess androgen levels on physiologic replacement doses of glucocorticoids—a therapeutic profile that Crinetics  has said positions atumelnant to become the leading treatment for people with CAH.

Atumelnant (formerly CRN04894) is also being developed for ACTH-dependent Cushing’s syndrome, and is under study in a Phase Ib/IIa open-label, multiple-ascending dose exploratory study (NCT05804669) designed to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamic biomarker responses associated with the treatment.

‘Significant potential’

“We are also excited by the significant potential of atumelnant to transform the treatment landscape for CAH, setting a new standard of care where patients do not have to choose between managing their excess adrenal androgens and enduring the side effects of high-dose steroids,” Kewalramani said.

One analyst said a Vertex buyout would be good news for Crinetics.

“This is a solid outcome for CRNX, given stock pressure from the near-term Palsonify launch (generally slow and steady launch but (+) [positive] progress by CRNX so far) and the fact that key Phase III catalyst for CAH isn’t until late 2027/28,” Jefferies equity analyst Dennis Ding wrote today in a research note.

In a regulatory filing yesterday, Crinetix shared an email it sent to employees, stating: “We have always been confident in the ability of Crinetics to achieve our plan and were not actively looking to sell the company when Vertex approached us. However, after careful consideration, our board unanimously determined that the transaction is in the best interests of our shareholders.”

Crinetics’ pipeline of more than 10 disclosed candidates includes:

  • CRN09682, a Phase I nonpeptide drug conjugate candidate being developed to treat somatostatin receptor 2 (SST2) expressing neuroendocrine tumors and other SST2 expressing solid tumors.
  • Discovery-phase preclinical programs focused on endocrine targets that include thyroid stimulating hormone (TSH), parathyroid hormone (PTH), somatostatin receptor 3 (SST3), growth hormone (GH), glucagon-like peptide 1 (GLP-1), and glucose-dependent insulinotropic polypeptide (GIP), as well as GPCR-targeted oncology indications.

Vertex said the deal was expected to contribute immediately to revenue growth via the ongoing launch of Palsonify, which the company says has blockbuster (greater than $1 billion in annual sales) potential in acromegaly. Longer term, Vertex says, atumelnant could also generate multiple billions of dollars in CAH, with additional revenue potential in Cushing’s syndrome.

$5B revenue forecast

R. Scott Struthers, PhD, Crinetics’ Co-founder and CEO

At peak year, Palsonify and atumelnant could deliver more than $5 billion in combined annual revenue, Vertex said, and thus contribute toward its goal of delivering sustained double-digit revenue growth, plus industry leading operating margins. The transaction is expected to add to non-GAAP operating income as of 2029.

Jefferies analyst Ding commented that atumelnant in CAH is expected to generate the largest share of the projected $5 billion, as in $2 billion to $3 billion, plus another $1 billion to $2 billion for Cushing’s syndrome–with the remaining $1 billion to be generated by Palsonify in acromegaly.

Scotiabank analyst Louise Chen told Reuters: “The deal ​adds a fifth vertical, endocrinology, which helps diversify VRTX’s concentration in CF.”

That concentration has proven lucrative for Vertex: During Q1, CF treatments generated $2.915 billion in total revenues, 98% of the company’s total revenue of $2.987 billion.

Vertex has agreed to acquire all outstanding shares of Crinetics common stock for $85 per share cash, in a deal valued at $8.8 billion net of estimated cash acquired. Vertex said it expects to finance the acquisition using a combination of cash on hand and debt, supported by $4.5 billion of fully committed bridge financing from Bank of America and Morgan Stanley Senior Funding.

Vertex finished the first quarter with cash, cash equivalents, and total marketable securities of $13 billion, up from $12.3 billion as of December 31, 2025. The company attributed the increase primarily due to cash flows from operating activities, partially offset by repurchases of Vertex’s common stock.

The transaction is expected to close in the third quarter subject to customary closing conditions, including receipt of regulatory approvals and approval by Crinetics shareholders.

“Nearly 18 years ago, we founded Crinetics with a clear goal of transforming the lives of patients living with endocrine-related diseases. Today marks a historic milestone as we embark on this next chapter with Vertex,” stated R. Scott Struthers, PhD, Crinetics’ co-founder and CEO. “Vertex’s global infrastructure and commercial footprint will serve to amplify the reach of our science and allow us to maximize the impact of Palsonify, atumelnant and our pipeline.”

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]]> A New Approach to Supporting Quality of Biologics https://edusehat.com/en/a-new-approach-to-supporting-quality-of-biologics https://edusehat.com/en/a-new-approach-to-supporting-quality-of-biologics Wed, 08 Jul 2026 04:10:12 +0700 Edusehat New, Approach, Supporting, Quality, Biologics
Diane McCarthy
Diane McCarthy, PhD
Vice President,
Global Biologics, USP

The United States Pharmacopeia (USP) is proposing a new approach for its work to support the quality and availability of selected biologic medicines. Building on decades of experience in setting public standards for protein therapeutics, from insulins to growth hormones, USP is now advancing an evolved approach tailored to the complexity of biologics.

At the center of this evolution is the launch of emerging standards for biologics on USP’s Emerging Standards Platform. This platform enables an agile, iterative approach to shaping a potential standard and has grown in scope from focusing on analytical methods for small-molecule medicines to proposals for digital standards and now includes product-specific methods of analysis for biologics. This platform allows concepts in early development to be shared with the community prior to any compendial consideration in order to solicit feedback and stimulate a dialogue that contributes to its evolution.

The initial release includes methods for the analysis of Bevacizumab, Epoetin, Interferon beta 1-a, and Rituximab, spanning both glycosylated proteins and monoclonal antibodies. Together, these are intended to engage stakeholders and facilitate collaboration.

GEN sat down with Diane McCarthy, PhD, USP vice president of biologics, to discuss why USP published these tools, how industry can use these emerging standards, and what lies ahead.

 

GEN:  Last month, USP published “Emerging Standards for Bevacizumab, Epoetin,Interferon beta 1-a, and Rituximab.” What are these emerging standards?

Diane McCarthy: Emerging standards are concepts that USP shares with the scientific community to bring awareness and stimulate early discussion around a specific molecule, method, or attribute. Scientific experts may test or apply these emerging standards, submit comments, or propose additional test methods to be added. Emerging standards are developed outside of the normal compendial process to enable early stakeholder feedback. If an emerging standard evolves to the point of entering the official USP Documentary Standards process, it would go through USP’s formal notice and comment procedures through publication in the Pharmacopeial Forum.

The format of these emerging standards is different from current compendial standards and is intended to accommodate the natural heterogeneity of biologics that arise due to production in living cells and different manufacturing processes. The emerging standard focuses on quality attributes—such as biological activity, glycosylation, charge variants, and size variants—and establishing suitable test methods while providing greater flexibility in terms of analytical methods and acceptance criteria.

These emerging standards are also paired with reference standards that are intended to support system suitability testing and assay performance monitoring. These materials can help manufacturers ensure consistency in their analytical methods and generate reliable, consistent data over time.

Finally, it’s important to emphasize that emerging standards continue USP’s tradition of welcoming constructive feedback to advance the development of standards and tools that support the quality of medicines.

 

GEN: Why is USP publishing these emerging standards for biologics now?

McCarthy: USP’s release comes at a defining moment. Policymakers are seeking ways to broaden patient access to quality, affordable biological medicines, including biosimilars. The urgency of this work is underscored by what has been described as the “biosimilars void.”1 Over the next decade, approximately 118 biologic drugs are expected to lose patent protection, yet only 12 currently have biosimilars in development. This gap represents a potential $230+ billion market opportunity for biosimilar competition in the U.S. alone and highlights a significant missed opportunity to reduce costs and expand patient access to more affordable biologic therapies.

Adding biologics to the emerging standard platform also advances a USP Convention resolution—adopted by 450+ organizations across 50+ countries—to expand availability of and access to quality-assured biologics products.

By taking this step to publish emerging standards, USP is responding to stakeholder requests and joining pharmacopeias around the world as they expand their portfolios of standards to support biologics. The publication of these emerging standards for comment reflects USP’s commitment to advancing public health worldwide through science-based, publicly accessible quality tools developed in a transparent and collaborative manner.

 

GEN:  How does USP envision the industry and regulatory authorities using an Emerging Standard?

McCarthy: These emerging standards are aligned with the evolving paradigm for biosimilar development, where analytical characterization plays a central role. Increasingly, regulators are recognizing that comparative analytical assessments, supported by pharmacokinetic and immunogenicity data, may be sufficient to demonstrate biosimilarity, reducing reliance on large, costly clinical studies.

We see several ways industry can leverage these emerging standards.

First, they provide a strong starting point for method development. Sponsors can use these publicly available analytical approaches in combination with reference products and in-house standards to assess product quality attributes and design robust analytical strategies early in development.

Second, the accompanying reference standards support system suitability testing and assay control. This is particularly important for helping ensure method performance, enabling data trending, and reducing variability across laboratories and over time.

Third, these emerging standards promote greater alignment between manufacturers and regulators. One of the challenges we’ve heard consistently from global regulators is the wide variability in analytical approaches submitted in biologics applications. By providing common tools and methods, we can help streamline review processes and improve regulatory predictability.

 

GEN: How are emerging standards different from official USP standards?

McCarthy: There are several important distinctions.

First, in contrast to current official USP standards, emerging standards are potential standards in their initial development phase and may never become official.

Second, emerging standards focus on quality attributes rather than prescribing market specifications. Instead of embedding fixed product specifications as in traditional monographs, they emphasize the critical attributes that define product quality, recognizing that specifications may be process-dependent. Manufacturers are expected to work with relevant regulatory authorities to define product-specific specifications.

Third, they are designed with flexibility in mind. Emerging standards provide multiple analytical methods to assess a given quality attribute and allow for the use of scientifically justified alternatives, including, where appropriate, compendial methods from other pharmacopeias. This approach supports innovation, keeps pace with evolving technologies, and can help improve the efficiency of development and manufacturing.

However, the emerging standards also leverage existing USP standards that help establish quality across an entire product class or multiple types of products. For example, emerging standards may cite cross-cutting standards such as USP General Chapter <129> Analytical Procedures for Recombinant Therapeutic Monoclonal Antibodies or <509> Residual DNA Testing, as well as chapters related to assessment of microbial contamination and other broadly applicable compendial tests.

 

GEN:  You’ve highlighted that these are open for comment. What type of feedback are you seeking?

McCarthy: At a high level, we want feedback on the utility of the overall approach. At a technical level, we are interested in feedback on the methods themselves. Are they appropriate, sufficiently detailed, and representative of current best practices? Are there alternative methods that should be included? One of the key features of the emerging standards platform is the ability for stakeholders to submit their own methods for consideration.

We are also looking for feedback on gaps, whether additional quality attributes or orthogonal techniques should be incorporated, and on usability, including how easy the emerging standards are to interpret and apply.

 

GEN:  As USP looks forward, what will be the next steps for these emerging standards, as well as other potential biologics standards?

McCarthy: Now that we have published the first four emerging standards for comment, our immediate focus is on engagement. We will collect more stakeholder feedback during the comment period and use that to refine and potentially republish updated versions of these emerging standards.

In parallel, we will continue expanding the portfolio of emerging standards to additional biologics—particularly high-impact therapeutic proteins and monoclonal antibodies, while incorporating lessons learned from this initial release.

We also continue to build out our reference standards pipeline, ensuring a reliable supply and robust characterization to support these methods. Regulators have made it clear that product-specific reference standards are critical for establishing system suitability and controlling assay performance over time, and that remains a key focus.

Longer term, if we have sufficient stakeholder and regulatory support, we may consider evolving those emerging standards that show broad utility into official USP documentary standards, including monographs.

Ultimately, this initiative is about enabling global alignment on biologics quality, supporting efficient development, manufacturing, and regulatory review to increase the availability of biologic and biosimilar therapies for patients around the world.

 

Reference

  1. IQVIA “Assessing the Biosimilar Void in the U.S.” Institute Report. Feb 3, 2025

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]]> Biotech and Biomed Engineering Grad and Certificate Programs to Debut at Auburn https://edusehat.com/en/biotech-and-biomed-engineering-grad-and-certificate-programs-to-debut-at-auburn https://edusehat.com/en/biotech-and-biomed-engineering-grad-and-certificate-programs-to-debut-at-auburn Wed, 08 Jul 2026 04:10:11 +0700 Edusehat Biotech, and, Biomed, Engineering, Grad, and, Certificate, Programs, Debut, Auburn Biomedical engineering graduate and certificate programs are coming to Auburn University. Led by the department of chemical engineering and the Biomedical Engineering Advisory Committee, the state‑approved programs will serve the entire college of engineering and the broader Auburn University community, according to university officials.

These offerings include a doctoral degree, thesis and non‑thesis master’s degrees, and two graduate certificates, all drawing on the university’s expertise in advanced biomedical technologies and biotechnology.

“The biotechnology and biomanufacturing sectors within the state of Alabama are growing quickly, and they need engineers who are prepared to contribute on day one,” said Mario Eden, PhD, dean of engineering. “These offerings position Auburn as a conduit for that workforce, producing graduates with the technical depth and hands‑on experience industry partners are asking for and enhancing the college’s capacity to support the state’s growing innovation economy.”

Biomedical engineering blends principles of engineering, biology, physics, and medicine to advance human health. It focuses on developing technologies and systems that improve how diseases and injuries are understood, diagnosed, monitored, and treated. This interdisciplinary field includes:

  • Biomanufacturing, tissue engineering, and regenerative medicine
  • Drug delivery and pharmaceutical engineering
  • Computational modeling, data science, and artificial intelligence
  • Biomechanics, biomaterials, and rehabilitation engineering
  • Medical imaging, medical devices, wearable technologies, biosensors, and diagnostics

Auburn’s biomedical engineering lineup will involve more than 20 faculty members across the college.

“This effort gives us a clear framework for graduate study in a field where our faculty have already built real momentum,” said Selen Cremaschi, PhD, chair of the department of chemical engineering. “It brings that activity into a coordinated structure that supports rigorous graduate experiences and reflects our identity as a research driven college.

The biomedical engineering certificate program is expected to launch in Fall 2026 with a full program launch scheduled for Fall 2027.

 

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]]> The Quest for Large-Scale DNA https://edusehat.com/en/the-quest-for-large-scale-dna https://edusehat.com/en/the-quest-for-large-scale-dna Wed, 08 Jul 2026 04:10:10 +0700 Edusehat The, Quest, for, Large-Scale, DNA Although genome editing was not a new concept, as zinc finger and TALEN platforms were already in use, the discovery of CRISPR-Cas9 shifted genome-editing research and clinical translation into high gear. But just like other platforms, this new kid on the block was not applicable to every editing situation for every genetic disease.

A longstanding desire in the field is a one-and-done, mutation-agnostic cure for genetic diseases that result from numerous mutations in a gene or from large-scale chromosomal structural variations, including deletions, duplications, inversions, and translocations. Gene therapies for these genetic indications require large-scale DNA manipulation, presenting different technical and regulatory challenges than correcting single-nucleotide point mutations.

Scientists donned their Indiana Jones hats to search for this holy grail of genome editing. Promising approaches under exploration included bridge recombinases, large serine recombinases, and CRISPR-associated transposases (CASTs), as well as immune-evasive DNA cargoes like circular single-stranded DNA (cssDNA), which may address the innate toxicity of double-stranded DNA (dsDNA) payloads.

Still, delivery can remain a conundrum for large payloads. For the most part, current delivery mechanisms are size-limited in terms of payloads, as are the workarounds using mRNA formats and leveraging reverse transcriptase.

The thirst is there, and the quest will continue. New genome-editing tools applicable to large DNA cargoes and delivery mechanisms will be refined, putting potential cures in sight for some deplorable diseases.

Bridge recombinases

A new class of programmable genome-editing tools, bridge recombinases are the first RNA-guided DNA recombinases providing a distinct mechanism for manipulating DNA.1,2

The system has two key components: the recombinase enzyme, which catalyzes the DNA rearrangement, and a bridge RNA guide with two independently programmable loops. The target-binding loop controls genomic locus targeting, and the donor-binding loop specifies the donor payload.

Reprogramming the bridge RNA to change the configuration and orientation of the target and donor sites allows the system to be redirected to perform excision of a desired sequence from the genome or inversion of a DNA segment in place. The modularity means a single two-component system can perform all three fundamental DNA rearrangements—insertion, excision, and inversion—through a single unified mechanism.

“The most immediate advantage is the scale of DNA that bridge recombinases can manipulate,” said Patrick Hsu, PhD, co-founder and core investigator of the Arc Institute and assistant professor of pathology at Stanford University. “A technology that can operate at the scale of whole-gene replacement or correct structural variants opens up a new class of genetic interventions.” Multi-kilobase insertions, inversions up to 0.93 Mb, and excisions up to 0.13 Mb have all been demonstrated in human cells.3

Bridge recombination also does not rely on dsDNA breaks. The recombinase catalyzes strand exchange directly through a covalent intermediate, making the outcome deterministic in a way that nuclease-dependent approaches are not. This indicates that the system may have advantages in post-mitotic cells for therapeutic applications. About a quarter of the size of Cas9, the system can be encoded in delivery vectors with limited capacity.

“The technology is still in development. While our current efficiency and specificity numbers (20% insertion efficiency with 82% on-target) represent a meaningful proof-of-concept in human cells, improving both metrics will be necessary for safe and effective therapeutic applications,” said Hsu. The diversity of bridge-recombinase systems found in nature continues to be explored.

Large serine recombinases

“For simpler cases where we want to insert a DNA payload into a fixed safe harbor site, we are working on large serine recombinases (LSRs),” said Hsu. While these enzymes lack the RNA programmability of bridge recombinases, they offer very high efficiency and specificity of insertion and are effectively unidirectional, leading to very stable insertions of large DNA cargoes into the human genome.

Bridge recombinases diagram
Bridge recombinases have a dual targeting capability that enables these systems to insert new genetic material, delete unwanted regions, or flip existing DNA segments, all in a single, programmable step. [Chiara Ricci-Tam, Arc Institute]
A 2025 Nature Biotechnology paper described an LSR enzyme engineered to enable site-specific insertions of multi-kilobase DNA payloads with 53% efficiency and 97% genome-wide specificity. Importantly, it was demonstrated that LSRs work well in non-dividing cells, including primary human T cells.4

Stylus Medicine, a company Hsu co-founded, intends to advance LSRs for in vivo genetic therapies. “I am excited to see the new therapies that will emerge from combining recombinase technology with machine learning-assisted protein engineering and advances in DNA and effector delivery for challenging disease contexts,” said Hsu.

CRISPR-associated transposases

CASTs are naturally occurring bacterial systems that utilize nuclease-deficient CRISPR machinery to integrate DNA at genomic locations specified by guide RNAs (gRNA). “While CRISPR is often used to cut DNA, CASTs instead use CRISPR systems to guide site-specific DNA transposition,” said Isaac Witte, PhD, department of chemistry and chemical biology at Harvard University.

In 2019, two research groups—one at Columbia University led by Sam Sternberg, PhD, and the other at the Broad Institute of MIT and Harvard, headed by Feng Zhang, PhD—found that CASTs use CRISPR systems to target DNA transposition by a transposase complex. Further work demonstrated that CASTs were very efficient bacterial genome editors.

CASTs can mobilize multi-kilobase-scale DNA cargoes, and their naturally evolved transposition mechanism avoids forming dsDNA breaks in the genome. The problem was that the wild-type systems exhibited extremely low (often ≤0.1% of treated cells) or undetected integration activity in human cells.

Collaborating with the Sternberg lab, the lab of David Liu, PhD, from the Broad Institute of MIT and Harvard, used PACE (phage-assisted continuous evolution), a directed evolution platform developed by the Liu lab, to enhance the efficiency of CAST transposition.

In PACE, bacteriophages, which infect host bacteria, encode evolving genes in place of an essential gene for phage replication. This essential gene is instead encoded by host bacteria. “In PACE, you link the desired activity of the evolving biomolecule to the expression of this essential gene. In this case, we linked targeted DNA integration to the replication of phages encoding evolving CAST protein components,” said Witte.

A series of modifications ensured efficient enhancement of activity, resulting in the generation of an evolved variant of the CAST transposase protein TnsB that mediated over 200-fold improved integration activity in human cells. The TnsB protein contained ten individual mutations scattered throughout the predicted structure, which contributed to improved activity.

The evolved TnsB was combined with other PACE-evolved and rationally engineered CAST components to yield evoCAST, a system optimized for human-cell integration activity, published in Science.5

The evoCAST DNA integration does not require formation of dsDNA breaks in the genome, resulting in undetected levels of insertion and deletion mutations (indels) commonly found in traditional methods of gene insertion like nuclease-stimulated, homology-directed repair (HDR). In addition, evoCAST can be easily reprogrammed to genomic sites of interest by changing the gRNA sequence, and it supports a variety of DNA payload sizes, ranging from less than 1 kb to at least 15 kb.

A potential limitation, however, is that evoCAST is molecularly complex, containing seven distinct protein subunits, making the total coding size (~8.5kb) relatively large compared to around 5 kb for Cas9.

Big-picture limitations center on delivery, according to Witte, such as mitigating the cytotoxicity of foreign dsDNA in most therapeutically relevant cell types.  Additionally, reducing the size and the number of distinct components required for integration activity may facilitate evoCAST applications in vivo. Next steps include harnessing the naturally existing diversity of CAST systems to develop a more diverse repertoire of CASTs for genome editing in human cells.

Circular single stranded DNA

Full Circle Therapeutics’ genome writing technology centers on an immune evasive DNA modality, a mini-cssDNA, called C4DNA—circular, clean, concealed, and customizable up to 20 kb.

use of cssDNA as a novel, immune-evasive large DNA modality
The image illustrates the use of cssDNA as a novel, immune-evasive large DNA modality for immune cell engineering via DNA writing, for potential treatment of cancer, autoimmunity, and other diseases. [Caitlin Rausch for Full Circle Therapeutics]
“The holy grail of gene editing is kilobase DNA integration. While most studies focus on new editing enzyme discovery, we address the challenge from the donor side. To integrate gene-size DNA in a specific locus, the choices of donor cargo templates are ds, ss, circular, or linear DNA. Workarounds using RNA formats and leveraging reverse transcriptase are still size-limited,” said Howard Wu, PhD, co-founder and CSO at Full Circles Therapeutics.

The company has commercialized over 350 research-grade cssDNA for primary sequences and is developing processes for GMP-grade products for clinical applications. According to Wu, initially, the company’s founder, Richard Shan, intended to supply linear cssDNA as a DNA commodity for researchers in the gene-integration field. The starting material was cssDNA that was cleaved into linear strands. A serendipitous benchmark experiment using cssDNA as a control demonstrated surprisingly better integration performance than its linear counterpart.6 The unexpected results led to a foundational patent describing the use of cssDNA for targeted genomic integration.

After benchmarking the different DNA formats, cssDNA appeared superior and compatible with various CRISPR-Cas systems, along with other meganuclease editing systems such as TALEN. Next, they evaluated the hypothesis that immunogenicity due to dsDNA could be eliminated if mobile genetic elements like transposase systems and LSRs could use cssDNA.

Collaborating with a team at Harvard Medical School led by Benjamin Kleinstiver, PhD, they demonstrated that naked unmodified cssDNA, combined with piggyback transposases or LSRs, enables kilobase writing, albeit inefficiently. One way to improve integration efficiency was to design an oligo that could fuse to the cssDNA with hydrogen bonding to form a partial duplex. A 30- to 60-mer partial duplex showed good integration efficiency when compared to dsDNA, while remaining immune silent.

In another approach, the team modified the nuclear editor and installed a peptide sequence identified from a bacterial genome with a strong binding affinity with cssDNA. In this case, the modified Cas9 became an engineered molecular chaperone to recruit the DNA molecule and form a complex, effectively loading and delivering the genome engineering complex into the nucleus and direct to the targeted genome.7

Continued collaboration with Kleinstiver’s lab aimed to improve integration efficiency. The approach, in this case, used a partial duplex cssDNA that reconstituted a recombinase recognition sequence. The scientists termed this integration through nucleus-synthesized template addition of large lengths (INSTALL). INSTALL is compatible with diverse genome engineering nucleases and RNA-guided recombinases for high-fidelity kilobase-scale human genome writing.8

“We welcome partners,” said Wu. “It is prime time to talk about DNA medicines.”

 

 

References

  1. Hiraizumi, M, Perry NT, Durrant, MG, et al. Structural mechanism of bridge RNA-guided recombination. Nature2024; 630:994-1002. doi:10.1038/s41586-024-07570-2
  2. Durrant, MG, Perry NT, Pai JJ, et al. Bridge RNAs direct programmable recombination of target and donor DNA. Nature2025:630:984-993. doi:10.1038/s41586-024-07552-4
  3. Perry NT, Bartie LJ, Katrekar D, et al. Megabase-scale human genome rearrangement with programmable bridge recombinases. Science. 2026 Mar 12;391(6790):eadz0276. doi:10.1126/science.adz0276
  4. Fanton, A, Bartie, LJ, Martins JQ, et al. Site-specific DNA insertion into the human genome with engineered recombinases. Nat Biotechnol. 2025 Nov 6. doi:10.1038/s41587-025-02895-3
  5. Witte IP, Lampe GR, Eitzinger S, et al. Programmable gene insertion in human cells with a laboratory-evolved CRISPR-assoc iated transposase. Science. 2025 May 15;388(6748). doi:1126/science.adt5199
  6. Xie K, Starzyk J, Majumdar I, et al. Efficient non-viral immune cell engineering using circular single-stranded DNA-mediated genomic integration. Nat Biotechnol. 2025 Nov;43(11):1821-1832. doi:10.1038/s41587-024-02504-9
  7. Nam H, Xie K, Majumdar I, et al. Engineering tripartite gene editing machinery for highly efficient non-viral targeted genome integration. Nat Commun. 2025; 16:4569. doi:1038/s41467-025-59790-3
  8. Tou CJ, Xie K, Ferreira da Silva J, et al. Immune evasive DNA donors and recombinases license kilobase-scale writing. Nature. 2026 Mar 11. doi:10.1038/s41586-026-10241-z

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]]> AI Reveals Hidden Brain Lesions in Multiple Sclerosis MRI https://edusehat.com/en/ai-reveals-hidden-brain-lesions-in-multiple-sclerosis-mri https://edusehat.com/en/ai-reveals-hidden-brain-lesions-in-multiple-sclerosis-mri Wed, 08 Jul 2026 04:10:09 +0700 Edusehat Reveals, Hidden, Brain, Lesions, Multiple, Sclerosis, MRI It has long been known that brain gray matter plays a key role in multiple sclerosis (MS) disease progression and cognitive impairment, but because magnetic resonance imaging (MRI) has only been able to detect lesions in white matter, neither clinicians nor researchers have had a way to detect or monitor gray matter (cortical) lesions. And while many new drugs developed in the past decade can slow disease progression significantly, they primarily work on reducing white matter lesions.

A University at Buffalo (UB)-led team now reports that it has found a way to use artificial intelligence to reveal these otherwise invisible cortical lesions by reviewing existing MRI scans. The researchers say the significance of finally being able to see what has been known as one of the most important indicators in MS disease progression cannot be overstated.

“Detecting previously invisible cortical lesions on conventional legacy MRI scans has major implications for MS research and clinical care,” commented Robert Zivadinov, MD, PhD, SUNY distinguished professor in the Department of Neurology and director of the Buffalo Neuroimaging Analysis Center (BNAC) in the Jacobs School of Medicine and Biomedical Sciences at UB. “The ability to see for the first time these previously hidden indicators of MS disease progression, including cognitive impairment and disability, is an important advance.”

Added Michael G. Dwyer, PhD, associate professor of neurology and biomedical informatics in the Jacobs School and a researcher with BNAC, “What this collaboration has been able to accomplish is a real success story for applying AI in the medical arena. We now have access to these incredibly useful data on MRI scans that were there but you couldn’t see them without using AI to pull them out. The computational methods are finally at the point where we can do this.”

Zivadinov is senior author, Dwyer first and corresponding author of the team’s published paper in Communications Medicine, titled “Quantifying cortical lesions in multiple sclerosis MRI datasets using multi-contrast post- processing and deep learning.”

“Multiple sclerosis (MS) affects both the inner, connectivity-oriented portions of the brain (white matter) and the outer layer of the brain (the cortex),” the authors explained. While the involvement of cortical lesions in MS has been known almost since the identification of MS in the late 19th century, they weren’t included on diagnostic criteria until the 21st century. And even when they were included, it was noted that their use would be greatly limited due to the current capabilities of clinical MRI.

“Historically, research and clinical care in MS have focused on white matter, where focal demyelinating lesions are a hallmark of the disease,” they continued. And although there are now many therapies that can almost completely halt the incidence of new white-matter lesions in individuals with MS, they haven’t had the same impact on clinical progression, the team continued.

Over more recent decades it’s been found that gray matter is affected from the earliest MS disease stages, and it’s become evident that gray matter pathology is more than secondary to white matter damage. “From a clinical perspective, cortical lesions are strongly associated with clinical disability and cognitive impairment,” the authors stated. “They may also have more prognostic value than white matter lesions for disability and disease course.”

There’s an urgent need for in vivo imaging methods that can show gray matter lesions, they stressed. Dwyer added, “We have all been very frustrated, knowing that these cortical lesions were there but not being able to see them. There’s a lot of ongoing damage that continues to happen in MS that you won’t see with conventional MRI, but that histopathologists have been clearly demonstrating for decades on postmortem tissue.”

For their newly reported study the team applied advanced image processing techniques, including artificial intelligence, to standard MRI scans from a large MS clinical trial. “Recently, several post-processing methods, including synthetic contrasts and artificial intelligence (AI)-based approaches, have shown potential for enhancing cortical lesion detection on conventional MRI data,” they noted. “These methods have the potential to reanalyze existing clinical-trial data to answer key mechanistic questions about both MS development and about treatment effects.”

The AI approaches the researchers used, building on work from co-authors from the Netherlands, were designed to extrapolate vital information from the relationships between multiple images that can’t be seen on a single image.

The researchers combined multiple image-processing techniques, including a new one they developed called MMCLE, or multimodal cortical lesion enhancement. They then applied these techniques to MRI scans from the large, phase III FDA regulatory ORATORIO clinical trial, a study of the MS drug Ocrelizumab that included more than 700 participants.

They found that while individual images of a patient’s brain revealed mostly white matter lesions, once they applied the AI-based image processing methods to multiple different contrast images, they were able to see anywhere from 15 to 20 cortical lesions for each patient, more than 11,000 for the whole dataset. “We confirmed that cortical lesions can be clearly visualized and quantified with these methods,” they stated. “Using deep learning, we also confirmed that the simultaneous use of multiple contrasts improves quantification.”

Dwyer explained further, “If you look on the original scans, you generally can’t see the cortical lesions, but generative AI is very powerful because it can look between the scans and detect tiny differences between them. Because it sees those minor discrepancies, AI can reveal that there’s something going wrong there, that the tissue is not behaving like healthy tissue. The trained models can view multiple MRI images together and synthesize them and synthesize what had been missing.”

Zivadinov added “This work, which has revealed that there is so much invisible pathology in the brain, will have tremendous impact for reviewing data from past clinical trials and also for those going forward,” he says.

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]]> A New Development Playbook for PROTACs https://edusehat.com/en/a-new-development-playbook-for-protacs https://edusehat.com/en/a-new-development-playbook-for-protacs Wed, 08 Jul 2026 00:35:16 +0700 Edusehat New, Development, Playbook, for, PROTACs
Shanghao Li
Shanghao Li, PhD
International Marketing Associate Director, La, boratory Testing Division, WuXi AppTec

As proteolysis-targeting chimeras (PROTACs) mature from scientific breakthrough to clinical modality, a candidate’s degradation potency is no longer enough to justify its advancement. A strong degrader is not necessarily a strong drug candidate if liabilities in exposure, safety, selectivity, manufacturability, or dosing strategy emerge later. As the field advances, success will depend on whether sponsors can integrate chemistry, pharmacology, safety, manufacturability, and clinical strategy early enough to translate promising degraders into viable medicines.

PROTACs have helped redefine what may be possible in drug discovery. By harnessing the ubiquitin-proteasome system to eliminate disease-relevant proteins, rather than simply inhibiting their activity, they have expanded the scope of targets that may be therapeutically addressable. For the past several years, much of the excitement around PROTACs has centered on this breakthrough mechanism. The field has been driven by the promise of degrading previously “undruggable” proteins, improving selectivity, and potentially overcoming resistance mechanisms that limit traditional inhibitors. But as clinical programs progress and the modality moves closer to late-stage regulatory milestones, the central question is changing.

The issue is no longer whether targeted protein degradation works. It is whether promising degraders can be translated into clinically and commercially viable therapies.

That transition marks an important phase of maturity for PROTAC development. The next wave of progress will depend on translational discipline and the ability to balance potency with developability, pharmacology, safety, manufacturability, and clinical feasibility from the earliest stages of development.

Entering a new phase of maturity

Early PROTAC innovation was rightly focused on validating the modality itself. Demonstrating that a heterobifunctional molecule could recruit an E3 ligase, drive ubiquitination of a target protein, and induce selective degradation was a foundational scientific achievement. That early work established targeted protein degradation as part of a broader wave of transformational therapeutic modalities, alongside approaches such as RNA interference therapeutics and antibody–drug conjugates, that have expanded what drug developers can target and how they think about translation.

Today, however, the field is operating under a different set of expectations. As more candidates advance through clinical development, sponsors must show not just that a PROTAC can degrade a target, but that it can do so with an exposure profile, safety margin, formulation strategy, and manufacturing pathway appropriate for clinical applications. A potent degrader in vitro may still fail to become a viable development candidate if it cannot achieve sufficient intracellular exposure, is metabolically unstable, if its degradation profile extends beyond the intended target set, or if its chemistry introduces manufacturing and formulation complications that slow advancement.

In other words, the scientific novelty of a modality can carry a program only so far before technical feasibility must be addressed for a candidate to advance. For PROTACs, that moment has arrived.

Potency alone is an incomplete metric

Degradation potency remains important. Maximum degradation, degradation half-life, and related pharmacodynamic measures are essential for understanding whether a molecule is engaging its biology as intended. But potency on its own can be misleading, particularly when it becomes the dominant criterion for candidate selection.

PROTACs are not conventional inhibitors. Their event-driven, catalytic mechanism introduces complexities that make exposure-response relationships less intuitive than those seen with traditional small molecules. Biological effects may persist after plasma concentrations decline, while higher concentrations do not necessarily lead to greater activity. In some cases, excessive exposure may even reduce degradation efficiency because of saturation effects that limit productive ternary complex formation.

This means the “best degrader” in a screening cascade is not always the best drug candidate. A molecule may demonstrate impressive degradation in a cellular assay while carrying liabilities that emerge only later, such as poor permeability, limited oral bioavailability, rapid linker metabolism, high nonspecific binding, unstable analytical performance, or off-target degradation driven by ligase biology or ternary complex behavior. If those issues are not considered early, potency can create a false sense of confidence in a degrader’s potential for clinical use.

Development workflows fall short

One reason translational issues emerge so frequently in PROTAC programs is that many development workflows still reflect assumptions built around traditional small molecules. In those models, discovery, DMPK, bioanalysis, toxicology, and chemistry, manufacturing, and controls (CMC) often proceed in a staged or partially sequential manner, with each function evaluating modality-relevant properties within its own domain before handing it forward.

With targeted protein degraders, however, early chemistry decisions can directly influence permeability, intracellular exposure, metabolic clearance, assay reliability, biodistribution, and manufacturability. Linker design, ligand selection, and overall polarity are not simply medicinal chemistry concerns; they shape how the molecule behaves across the entire development continuum. Likewise, a bioanalytical challenge may obscure the interpretation of PK/PD relationships, complicate dose optimization, or delay confidence in candidate selection.

The same is true for safety. Because PROTACs eliminate proteins rather than transiently inhibiting them, the consequences of target engagement can differ meaningfully from those associated with conventional inhibitors. On-target toxicity may emerge when complete or prolonged degradation is not tolerated, even if partial functional inhibition is acceptable. Off-target effects may arise not only from target promiscuity, but also from E3 ligase recruitment and unintended ternary complex formation. These risks cannot be addressed effectively if safety is considered only after potency and exposure have been optimized.

Traditional workflows can also underweight manufacturability and CMC considerations. PROTACs are generally handled as small molecules, but their structural complexity can create multi-step synthesis challenges, impurity-control difficulties, and formulation constraints much earlier than teams may expect. When these issues are discovered late, promising programs can lose momentum for reasons that have little to do with biology.

The core issue is not organizational design alone. It is that PROTACs expose the limits of linear decision-making. They require earlier integration because the liabilities that determine success are tightly interconnected.

PROTAC-specific development

If PROTACs require a different development model, what would it look like?

First, a successful PROTAC development plan should begin with balanced optimization across parameters rather than sequential, single-parameter optimization. Candidate selection should account not only for degradation potency, but also for permeability, solubility, metabolic stability, intracellular exposure, selectivity, formulation feasibility, and synthetic tractability. Programs that rank candidates holistically are better positioned to recognize which molecules are genuinely translatable.

Second, the PK/PD strategy should be built around the biology of degradation. Because systemic exposure does not fully explain pharmacological effect, teams increasingly need direct measures of target degradation and recovery kinetics, not just plasma concentration data. Mechanistic PK/PD models can help connect degradation durability, protein resynthesis, and dosing schedule in a way that better reflects how PROTACs work in vivo.

Third, bioanalysis should be treated as a strategic enabler rather than a downstream technical function. PROTACs can introduce assay complications, including nonspecific binding, chromatographic artifacts, and instability across matrices. Robust analytical methods are essential not only for quantitation but for making reliable decisions about exposure, disposition, and translation across study systems.

Fourth, safety assessment must expand beyond conventional assumptions. Early proteomic profiling, tissue distribution analysis, and evaluation of degradation selectivity can help identify liabilities before they become entrenched in a program. For PROTACs, understanding where degradation occurs, how long it persists, and what unintended proteins may be affected is central to designing an acceptable therapeutic window.

Finally, CMC and manufacturability should be considered earlier than many teams may be accustomed to. A molecule with compelling pharmacology but limited synthetic scalability, poor solid-state properties, or unstable formulation behavior may not be a strong development candidate. Integrating these realities earlier supports smarter program prioritization and reduces late-stage surprises.

Taken together, these elements define a development playbook centered on translation. They signal a maturation of the field, in which the emphasis shifts from demonstrating biological power to establishing overall developability, recognizing that promising degraders must ultimately succeed as integrated therapeutic candidates, not just mechanistic innovations.

Sponsors can improve the odds

For sponsors advancing PROTAC programs, translation should be a design principle from the beginning. That starts with cross-functional alignment early in discovery. Chemistry, DMPK, bioanalysis, safety, and CMC teams should work together to shape candidate criteria, so that trade-offs are recognized early, and optimization reflects the realities of development rather than the priorities of any one function.

It also means adopting more realistic success metrics. Degradation data should remain central, but it should be interpreted alongside developability, not in isolation from it. Sponsors may also benefit from building translational assays and biomarkers earlier. The ability to directly measure target degradation and connect it to pharmacodynamic effect can strengthen decision-making throughout preclinical and clinical development. In a modality where traditional exposure markers may be incomplete, translational pharmacology can provide strategic direction.

Most importantly, teams should resist the temptation to force PROTACs into a conventional small-molecule framework. These candidates may be classified as small molecules for many regulatory purposes, but functionally, they behave as a distinct modality. Treating them as such allows the development strategy to evolve in step with biology.

The next phase of PROTAC success

PROTACs have already shifted the pharmacological landscape around drugability. Their next contribution may be just as important by forcing the industry to rethink what a good development strategy looks like for complex, mechanism-driven therapeutics. As the field matures, successful drug sponsors will be those who can translate degradation into a developable, manufacturable, safe, and clinically meaningful therapy. That requires a different playbook built on integration, balanced optimization, and translational discipline. For PROTACs, that is no longer a future concern. It is the central challenge of the present.

 

Shanghao Li, PhD, currently serves as international marketing associate director in the Laboratory Testing Division at WuXi AppTec.

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]]> KLK1 Expands Possibilities to Restore Vascular Health https://edusehat.com/en/klk1-expands-possibilities-to-restore-vascular-health https://edusehat.com/en/klk1-expands-possibilities-to-restore-vascular-health Wed, 08 Jul 2026 00:35:15 +0700 Edusehat KLK1, Expands, Possibilities, Restore, Vascular, Health

Knowing that the protein tissue kallikrein-1 (KLK1) is effective in treating ischemic diseases is one thing. Manufacturing it as a recombinant protein has been quite another. So, when DiaMedica Therapeutics cracked the manufacturing aspect, it was well on its way toward commercializing KLK1 therapeutics.

The manufacturing breakthrough came when researchers realized that protein activity (which is essential for therapeutic benefit) was linked to certain glycosylation patterns. DiaMedica engineered the molecule to reflect those glycosylations and also made two changes to the amino acid sequence to improve manufacturability. “Then we partnered with Catalent,” Rick Pauls, president and CEO, says. “We are using its GPEx® technology with CHO cells,” which produces more cells within the same timeframe and thus lowers manufacturing costs.

Tenacity in action

This happened neither easily nor quickly. To understand the measure of this achievement, we need to look at DiaMedica’s history.

KLK1 came to DiaMedica’s attention because of liver research. “A liver physiologist cut the vagus nerve [which regulates liver metabolism] and discovered that the rats, effectively, became diabetic,” Pauls recounts. “We hypothesized that when a healthy person consumed a meal, the liver releases something that acts as an insulin sensitizer. We did some basic work and identified KLK1 as that insulin sensitizer.”

The company was founded in 2004 to develop a KLK1 therapeutic for complications related to Type II diabetes. Those trials failed. “It’s a long story,” says Pauls, that left the company “pretty close to bankrupt.”

DiaMedica, though, was tenacious. “We knew there was a human urine form of this protein that had been used for a few decades in Asia to treat acute ischemic stroke, and a porcine form treating hypertension for decades as well,” Pauls recalls. DiaMedica had the protein and the manufacturing know-how to produce active, recombinant proteins, and—with KLK1 levels low in stroke patients—a reason to pivot.

Ischemic stroke and preeclampsia

Its lead compound, DM199 (rinvecalinase alfa), is enrolling patients in Phase II/III trials for acute ischemic stroke. Called the ReMEDy2 trial, the company anticipates an interim readout near year’s end. Additionally, Phase I and II studies for preeclampsia and Phase II studies for fetal growth restriction are underway.

“This is protein restoration,” Pauls says. It targets ischemic stroke patients who have missed the three-to-four-hour post-stroke treatment window for tissue plasminogen activator (tPA) therapeutics or mechanical thrombectomy. Those patients constitute approximately 80% of acute ischemic strokes today, so “there is a huge unmet medical need,” Pauls says.

DM199 works by restoring normal levels of the KLK1 protein. KLK1, in turn, is thought to enhance the production of nitric oxide, prostacyclin, and endothelium-derived hyperpolarizing factor. Pouring through their own preclinical and clinical results, the DiaMedica team noticed that DM199 consistently enhanced blood circulation and lowered blood pressure.

That realization drove the team to also target preeclampsia, a hypertensive disease of pregnancy that Pauls says may be the company’s most exciting application for investors.

Unlike approved blood pressure therapeutics, DM199 does not cross the placental barrier, a critical safety feature that protects the fetus. After examining early clinical data, DiaMedica scientists also realized that increasing blood flow to the placenta could target the root cause of the disease and perhaps gain another few weeks of crucial time in utero for the fetus.

“Today, there are no approved treatments. Mothers are given labetalol and nifedipine to control blood pressure and to extend the baby’s time in utero for only a few days.” Results are less than ideal, and the consequences can be severe.

Pauls says, “Some 40% of babies born before 28 weeks could have long-term disabilities, and 10 to 15% will have problems with eyesight for life. There’s been a real lack of drugs in development because developers are worried about harming the baby.”

An investigator-led Phase II clinical trial is enrolling. Later this year, the company plans to initiate its own Phase II study focused on early-onset preeclampsia after recently receiving regulatory clearance to start the study in Canada.

If the molecule eventually is approved for preeclampsia, DM199 seems poised to become, perhaps, the first approved treatment that offers the potential to extend gestational days and possibly address a root cause of preeclampsia.

Leveraging the pivot

Unlike many biopharmaceutical companies, DiaMedica has been able to bypass some of the usual first steps by leveraging existing studies on KLK1, as well as existing clinical data for stroke and preeclampsia.

That allows researchers to focus on humans without the translational issues inherent in animal studies. It also helps the company identify the human subgroups most likely to benefit from these treatments and the most appropriate dosing regimen early. “Having that clinical data helps de-risk our program and gives a better possibility of success,” Pauls says, because, as he points out, “Animals are not the same as people.”

Once the company pivoted to its current indications six or seven years ago, the challenge shifted from getting and manufacturing the active form of the protein to selecting the best indications and assembling the right team members.

“In the early days, maybe we didn’t have the right level of experience with limited capital,” he admits. Today, “we’ve been able to bring people on board who have brought drugs to market.”

Readouts due in 2027

Currently, the company is focused tightly on its clinical trials. The next step for DiaMedica is to get readouts from many of those, with five readouts on various aspects of the programs expected between now and the end of 2027. Each of those readouts will report on about 30 patients and will be factors in the design of a subsequent pivotal trial.

Additionally, an interim analysis of the first 200 patients in its acute ischemic stroke trial is expected by the end of the year, Pauls says. “If we see a drug effect that’s comparable to our Phase II trial or the data with the urine form (of KLK1) from China—which treats close to a million patients per year—we’ll be looking at completing enrollment the following quarter for stroke and then for preeclampsia. DiaMedica is dedicated to offering second chances to acute ischemic stroke patients and others who haven’t had them before, all while pivoting to new opportunities itself. Now, as trials advance, Pauls says, “I think this should be a straightforward path.”

DiaMedica Therapeutics

Location: 301 Carlson Parkway, Suite 210, Minneapolis, MN 55305

Phone: (763) 496-5192

Principal: Rick Pauls, president and CEO

Number of Employees: 35

Focus: DiaMedica is a clinical-stage company developing recombinant KLK1 protein restoration therapeutics for acute ischemic stroke, preeclampsia, fetal growth restriction, and other conditions with large unmet needs.

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]]> AI Tackles Tuberculosis, Identifies Drugs that Penetrate Bacteria Membrane https://edusehat.com/en/ai-tackles-tuberculosis-identifies-drugs-that-penetrate-bacteria-membrane https://edusehat.com/en/ai-tackles-tuberculosis-identifies-drugs-that-penetrate-bacteria-membrane Wed, 08 Jul 2026 00:35:12 +0700 Edusehat Tackles, Tuberculosis, Identifies, Drugs, that, Penetrate, Bacteria, Membrane According to the World Health Organization (WHO), tuberculosis, caused by the bacterium Mycobacterium tuberculosis (Mtb), is the world’s deadliest single-agent caused infection, responsible for 1.23 million deaths in 2024. The bacterium’s outer cell membrane is difficult hard to penetrate, making few drugs effective in treating the disease.   

In a new study published in Nature Microbiology titled, “Identification of chemical features for improved outer membrane permeation in mycobacteria using machine learning,” researchers from University of Massachusetts (UMass) Amherst have developed new methods to measure which chemical compounds can cross the outer bacterial membrane. 

“Mtb is unique,” said Sloan Siegrist, PhD, associate professor of microbiology at UMass Amherst. “Not only does it have two membranes that protect the cell from antimicrobial chemical compounds that we might use to kill it, its outer membrane is unlike any other biological barrier out there.” 

Siegrist’s lab specializes in finding vulnerabilities in the mycomembrane to develop drugs that can effectively treat tuberculosis. However, traditional drug discovery has relied on low throughput experimental screens. In 2023, Siegrist, in collaboration with Marcos Pires, PhD, professor of chemistry at the University of Virginia, published Peptidoglycan Accessibility Click-Mediated AssessmeNt (PAC-MAN), a method that can test many compounds in parallel. 

“Marcos and I wanted to harness measurements of known chemicals to predict compound uptake for unknown chemicals, so we brought in computational biologists and chemists, including my colleague Anna Green, PhD, from UMass Amherst’s Manning College of Information and Computer Sciences,” said Siegrist. 

Green uses computation to understand patterns in biological compounds. “Small molecules can be particularly difficult to analyze computationally,” she says. “Because they come in all different sizes with a wide range of molecular connections, you can’t describe them with a single measurement, by weight, say, or size.” 

Green and colleagues designed a machine learning model, the Mycobacterial Permeability neural Network (MycoPermeNet), trained on the PAC-MAN screening data. The model can predict how readily a compound permeates the mycomembrane from its chemical structure alone and points to the physical properties that help a compound penetrate Mtb’s defenses. 

“The mycomembrane lets some molecules through and keeps others out,” says Green. “There must be something about this membrane, and about the chemistry of each molecule, that decides which ones get in—and our combined tools help us figure out which ones can get through, and why.” 

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]]> Bioprocessing at Full Throttle https://edusehat.com/en/bioprocessing-at-full-throttle https://edusehat.com/en/bioprocessing-at-full-throttle Wed, 08 Jul 2026 00:35:11 +0700 Edusehat Bioprocessing, Full, Throttle In biomanufacturing, scale has long been synonymous with success. Bigger bioreactors, larger facilities, and expanded footprints traditionally defined the path to higher output. But that paradigm is shifting with intensified bioprocessing. Today, the industry is embracing a more nuanced, efficient approach—one that prioritizes productivity over size, agility over rigidity, and integration over segmentation. Intensified bioprocessing is not just an incremental improvement; it is a fundamental rethinking of how biologics are made.

“Intensified bioprocessing aims to improve the productivity and efficiency of biomanufacturing,” explains Julie Kozaili, PhD, principal scientist at Asahi Kasei Bioprocess. “This is often achieved by designing new processes or modifying existing ones to increase output per unit time or equipment volume.”

That deceptively simple definition captures a sweeping transformation. Instead of relying on traditional batch processes, intensification often involves running at higher cell densities, integrating multiple process steps, and transitioning toward continuous or semi-continuous operations.

The implications are significant. Intensified processes can reduce facility size, minimize resource consumption, and shorten development timelines—all while maintaining or even improving product quality. For an industry under constant pressure to deliver therapies faster, these advantages are hard to ignore.

The urgency behind intensification is driven by both scientific and economic realities. Many modern therapeutics—particularly viral vectors and gene therapies—face inherent production challenges. Low yields, complex manufacturing requirements, and stringent quality standards make scaling difficult and expensive.

In viral-vector development, one of the central bottlenecks is simply producing enough material. Clinical applications often require a minimum effective dose volume, yet production systems struggle to generate sufficient yield, forcing manufacturers to concentrate limited output into small delivery formats. Legacy adherent cell culture technologies compound the problem by relying on scale-out strategies—adding more units rather than increasing efficiency—making cost reductions difficult as production expands.

Intensified bioprocessing offers a different path. It “is important because it allows manufacturers to increase capacity without new facilities, reduce equipment footprint, reduce media, buffer, and utility usage per gram of product, and shorten scale-up, tech transfer, and time-to-clinic timelines,” Kozaili says.

For companies working with unstable or complex molecules, speed can be just as important as scale. Faster processing reduces the risk of degradation and accelerates the path from development to commercialization.

Beyond cost: speed and flexibility

Although cost savings are often cited as a benefit of intensification, industry leaders emphasize that its true value lies beyond the cost of goods. “Intensified bioprocessing is less about driving down cost and more about enabling speed, flexibility, and fit,” says Mark Schofield, PhD, director of science at Cytiva. “For monoclonal antibodies in particular, the industry’s priorities are getting to launch faster, making better use of existing facilities, and being able to respond to uncertain or fluctuating demand.”

This shift in perspective reflects broader changes in the biopharmaceutical landscape. Pipelines are increasingly diverse, with smaller patient populations and more specialized therapies. Manufacturing systems must be adaptable, capable of switching between products or scaling production up and down as needed. “Intensification helps companies do all three by rethinking how processes are designed and scaled,” Schofield adds.

Companies such as Repligen are advancing upstream intensification through perfusion-based systems designed to sustain high cell densities and continuous productivity. Perfusion cell culture, a cornerstone of many intensified strategies, continuously feeds fresh media while removing waste and product, allowing cells to remain in an optimal growth state over extended periods. This approach not only improves yield but also creates a more stable and controlled production environment compared to traditional fed-batch methods. Repligen’s filtration and analytical technologies further support this shift by enabling continuous clarification and real-time monitoring, helping bridge the gap between process development and scalable manufacturing.

Beyond large platform providers, a growing number of specialized innovators are helping push intensified bioprocessing forward, particularly in high-demand areas like viral-vector manufacturing and upstream control.

Meanwhile, Batavia Biosciences is tackling one of the most persistent challenges in gene therapy: low viral-vector yields. Traditional adherent cell culture systems often require scaling out—adding more equipment rather than increasing efficiency—which drives up costs without significantly improving productivity. Batavia’s intensified approach centers on integrated solutions that combine optimized cell lines, streamlined purification processes, and novel bioreactor designs to dramatically increase output. By enabling higher yields within a smaller footprint, these strategies effectively miniaturize manufacturing, making it possible to produce clinical and commercial quantities without the need for large-scale facilities.

Together, these efforts underscore a key theme in intensified bioprocessing: innovation is not confined to a single step or technology. Instead, it is emerging across the entire workflow, from upstream cell culture to downstream purification and process analytics.

Intensified bioprocessing
On the left, rigid legacy thinking and siloed bioprocessing models dominate a complex industrial environment. On the right, intensified bioprocessing enables agile, integrated systems where collaborative teams continuously test, optimize, and scale innovative manufacturing solutions. [Image generated with Google Gemini]

Real-world applications

The promise of intensified bioprocessing is being realized through a growing ecosystem of technologies. Asahi Kasei Bioprocess, for example, has developed solutions that support intensification at multiple stages. “We support intensified bioprocessing across upstream and downstream operations,” Kozaili explains, pointing to innovations such as hollow-fiber microfilters for high-intensity cell culture clarification and advanced virus filtration systems designed for continuous processing.

These technologies are engineered to handle the increased throughput associated with intensified upstream processes. High-density cultures generate larger volumes of product, which must be efficiently clarified, purified, and stabilized without compromising quality.

CRB Horizons: Life Sciences Report chart
Continuous implementation is a key element of intensified bioprocessing, and a company’s size impacts its key challenges. [CRB Horizons: Life Sciences Report]
Downstream, continuous virus filtration systems can operate at low flux over extended periods while maintaining robust viral clearance. Inline buffer formulation systems further streamline workflows by eliminating the need for large storage tanks and ensuring consistent buffer quality in real time.

Automation and integration are also key components. New ultrafiltration and diafiltration systems are being designed for flexibility, allowing them to be deployed upstream or downstream and enabling seamless process integration.

Designing for intensification

Though technology is a crucial enabler, successful intensification requires more than just new equipment. It demands a holistic approach to process and facility design. “At CRB, our role is to help clients translate emerging process concepts into facilities that are safe, operable, and scalable,” says John Rubero, senior fellow in purification bioprocessing.

One of the defining characteristics of today’s intensification efforts is that they are often partial or hybrid implementations. Fully continuous, end-to-end processes remain relatively rare. Instead, manufacturers are adopting elements of intensification—such as integrating continuous perfusion with multi-column capture chromatography—within otherwise traditional workflows. This incremental approach allows companies to realize benefits without fully overhauling their operations. It also provides a pathway for future evolution as technologies mature.

Despite its advantages, intensified bioprocessing is not without challenges. One of the most significant is bridging the gap between process development and commercial-scale implementation. “While the practice of linking unit operations together is largely accepted, real-time control of an end-to-end continuous process remains challenging,” Rubero explains.

In traditional batch processes, control strategies are relatively straightforward because lot traceability is easy to maintain. But intensified systems—especially continuous ones—require real-time monitoring and advanced control strategies to ensure process stability and product quality.

“It is not realistic or necessary to find and assign a sensor to monitor each critical process parameter or critical quality attribute,” Rubero says. “Instead, a combination of direct measurements, soft sensors, multivariate models, and process understanding is required for effective process control.”

So, the industry is moving toward integrated approaches that combine process analytical technology (PAT) with mechanistic and data-driven models. These systems enable more sophisticated monitoring and control but are still evolving in terms of reliability and adoption.

Operational barriers

Technical challenges are only part of the equation. Intensification also requires a shift in mindset—one that can be difficult for organizations accustomed to established manufacturing paradigms. “In many cases, the technologies are either new or have novel applications, creating a learning curve,” Kozaili acknowledges.

Training gaps, operational changes, and resistance to new approaches can slow adoption. Teams must adjust not only their processes but also their thinking, moving away from long-standing practices toward more dynamic, integrated systems.

As Schofield notes, “adopting new approaches inevitably comes with skepticism.” Externally, there can be hesitation to move away from established technologies. Internally, organizations might question how intensified solutions might impact existing product lines. Those discussions, however, are part of the transition.

Despite these challenges, momentum is building. As intensified technologies demonstrate their value in real-world applications, resistance is gradually diminishing. “Over time, evidence and adoption speak for themselves,” Schofield says.

Kozaili emphasizes the importance of organizational alignment. “We had to change the company’s established mindset by securing support to develop these technologies and clearly show the value of these approaches,” she explains.

Collaboration also plays a key role. For technology providers, working closely with customers to test and refine solutions helps build confidence and accelerate adoption. “For our customers, it’s about finding the right partners to test the technologies, while providing appropriate feedback for improvement,” Kozaili adds.

Looking ahead, the trajectory of intensified bioprocessing is clear. Purpose-built facilities designed specifically for intensified operations will become more common, replacing retrofitted batch plants that struggle to accommodate new workflows, because intensified bioprocessing is no longer a niche concept reserved for early adopters. It is rapidly becoming a central pillar of modern biomanufacturing strategy.

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]]> Biomanufacturing in Space to Be Key Topic at ISSCR 2026 https://edusehat.com/en/biomanufacturing-in-space-to-be-key-topic-at-isscr-2026 https://edusehat.com/en/biomanufacturing-in-space-to-be-key-topic-at-isscr-2026 Tue, 07 Jul 2026 21:00:10 +0700 Edusehat Biomanufacturing, Space, Key, Topic, ISSCR, 2026 Scientists from the Cedars-Sinai Board of Governors Regenerative Medicine Institute, including investigators from the Cedars-Sinai Biomanufacturing Center, say they will share groundbreaking discoveries and discuss new frontiers in research at ISSCR 2026. The annual meeting of the International Society for Stem Cell Research will take in Montreal from July 8–11.

The Cedars-Sinai Center for Space Medicine Research has taken a special interest in biomanufacturing in space. It studies how microgravity aboard the International Space Station and other space platforms can be used to manufacture higher-quality biomedical products.

Researchers investigate the production of stem cells, organoids, engineered tissues, exosomes, and biopharmaceuticals, taking advantage of the reduced effects of gravity on cell growth and three-dimensional tissue formation.

The center collaborates with NASA, commercial space companies, and biotechnology partners to determine whether space-based manufacturing can yield therapies with improved quality, consistency, and function. Its long-term goal is to translate discoveries made in space into scalable manufacturing methods that advance regenerative medicine, drug development, and personalized healthcare on Earth.

At the upcoming ISSCR 2026 meeting, Arun Sharma, PhD, director of the Center for Space Medicine Research, will participate in a session co-sponsored by Cedars-Sinai on regenerative medicine in low Earth orbit. The focus of Sharma’s talk is accelerating development of organoid-based disease modeling and stem cell therapies due to increased access to microgravity, as well as in-space biomanufacturing.

Avinash Srivastava, PhD, a biomedical scientist in the Cedars-Sinai Biomanufacturing Center, is presenting information on the center’s proprietary integrated induced pluripotent stem cell biomanufacturing platform. The platform integrates standardized manufacturing with advanced bioprocessing to facilitate the scalable production of high-quality engineered cell therapies.

Dhruv Sareen, PhD, associate professor of Biomedical Sciences and founding director of the Cedars-Sinai Biomanufacturing Center, is presenting research on the integration of an in situ seed plating system into the center’s manufacturing workflow to streamline production of complex induced pluripotent stem cell lines for clinical-grade and research use.

 

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]]> Unlocking Microbiome Function with Anaerobic Workflows and Metabolic Phenotyping https://edusehat.com/en/unlocking-microbiome-function-with-anaerobic-workflows-and-metabolic-phenotyping https://edusehat.com/en/unlocking-microbiome-function-with-anaerobic-workflows-and-metabolic-phenotyping Tue, 07 Jul 2026 10:15:09 +0700 Edusehat Unlocking, Microbiome, Function, with, Anaerobic, Workflows, and, Metabolic, Phenotyping

Over the last decade, the microbiome has shifted from a scientific curiosity to one of the most promising frontiers in biology and medicine. Whether it’s gut microbes that influence the host’s metabolism, an oral microbe that prevents cavities, or a microbial consortium that improves immunotherapy response in cancer—each discovery brings growing excitement.

Unlocking Microbiome Function with Anaerobic Workflows and Metabolic Phenotyping

As the excitement has grown, so too has the realization that identifying microbes is only part of the story. Preserving microbial viability and physiological relevance—particularly with regard to anaerobes and microaerophiles—throughout collection, transport, and cultivation is a critical prerequisite for accurately measuring microbial behavior.

The field is also shifting from descriptive microbiome research towards mechanistic understanding—using metabolic phenotyping, which directly measures microbial activity including nutrient and substrate utilization, cell growth, stress response, and more. Because only when we truly understand how microbes behave can we begin to shape them into powerful tools for healing.

Next generation sequencing (NGS) has transformed our ability to identify which species are present in a microbial community (“who’s there”) and what they may potentially be capable of. However, gene presence does not guarantee gene expression or necessarily reflect real-world activity (“what the microbes are doing”). Two strains may carry similar metabolic genes yet behave very differently under gutrelevant conditions.

These are some of the important functional questions sequencing alone can’t answer:

  • What metabolic pathways are actually active?

  • How do the microbes adapt to various environments and interact?

  • What is the optimal environment to produce critical metabolites?

As the field pushes toward developing live biotherapeutic products, evidence-backed probiotics, and next-gen biomarkers, understanding these functional traits is essential.

This eBook explores how researchers can move beyond correlation and towards mechanism in microbiome research—from preserving physiologically relevant microbial communities to directly measuring microbial function through phenotyping.

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]]> New sa‑mRNA and LNP Platform to Support Korea’s Hantavirus Vaccine Initiative https://edusehat.com/en/new-samrna-and-lnp-platform-to-support-koreas-hantavirus-vaccine-initiative https://edusehat.com/en/new-samrna-and-lnp-platform-to-support-koreas-hantavirus-vaccine-initiative Tue, 07 Jul 2026 10:15:08 +0700 Edusehat New, sa‑mRNA, and, LNP, Platform, Support, Korea’s, Hantavirus, Vaccine, Initiative With hantavirus thrust into the public spotlight in recent months, a new effort in South Korea aims to advance vaccine development against the rodent-borne pathogen using mRNA technologies.

Korea University College of Medicine has been selected to lead a government-supported initiative focused on developing next-generation hantavirus vaccines. The program will be conducted through the institution’s Vaccine Innovation Center.

Hantaviruses are carried primarily by rodents and can infect humans through exposure to contaminated urine, droppings, or saliva. Depending on the viral strain, infection can cause hantavirus pulmonary syndrome (HPS), a severe respiratory illness, or hemorrhagic fever with renal syndrome (HFRS), a disease characterized by kidney dysfunction and bleeding complications. Although relatively rare, hantavirus infections can carry high mortality rates and remain a public health concern in parts of Asia, Europe, and the Americas.

The renewed focus on vaccine development comes amid growing interest in preparedness for emerging and re-emerging infectious diseases. While mRNA technology gained worldwide recognition during the COVID-19 pandemic, researchers have increasingly explored its application against a broader range of viral threats.

According to Korea University, the newly funded program will leverage the rapid development potential of mRNA platforms to generate vaccine candidates targeting hantavirus infection.

“The Vaccine Innovation Center is the only private-sector vaccine research and development institute in Korea established to carry forward the scientific legacy of Dr. Ho-Wang Lee, who first discovered the hantavirus,” said Hee-Jin Cheong, MD, PhD, director of the Vaccine Innovation Center. “Beginning with hantavirus vaccine development, we aim to lead infectious disease research in Korea and contribute to improving public health.”

The initiative will draw on two domestically developed technologies: self-amplifying mRNA (sa-mRNA) and a next-generation lipid nanoparticle (LNP) delivery platform. Unlike conventional mRNA vaccines, sa-mRNA contains genetic instructions that enable replication of the RNA within cells, potentially generating stronger immune responses while requiring lower doses. The platform is intended to support rapid vaccine development and manufacturing while reducing dependence on overseas intellectual property.

The project builds on research conducted over the past two years at the Vaccine Innovation Center in collaboration with Moderna. The new program will seek to translate preclinical study findings into a next-generation vaccine candidate developed in partnership with biotechnology companies.

Under the two-year project timeline, researchers will spend the first year optimizing vaccine candidates and evaluating their efficacy. The second year will focus on Good Manufacturing Practice (GMP)-compliant production and safety testing.

Researchers have increasingly viewed mRNA platforms as particularly attractive because they can be rapidly redesigned when emerging threats arise. Lessons learned from COVID-19 vaccine development have helped establish manufacturing, regulatory, and clinical frameworks that may accelerate future vaccine programs.

As concern over emerging infectious diseases continues to shape global health priorities, programs such as this one may help expand the range of vaccine technologies available to combat pathogens that have historically received limited research attention.

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]]> Pharma Races to Scale AI as Billions Flow into Drug Discovery https://edusehat.com/en/pharma-races-to-scale-ai-as-billions-flow-into-drug-discovery https://edusehat.com/en/pharma-races-to-scale-ai-as-billions-flow-into-drug-discovery Tue, 07 Jul 2026 03:05:09 +0700 Edusehat Pharma, Races, Scale, Billions, Flow, into, Drug, Discovery “I’ve always believed the No.1 application of AI should be to improve human health.” 

–Demis Hassabis, PhD, CEO of Google DeepMind and Isomorphic Labs, 2024 Nobel Laureate in Chemistry 

The infrastructure moment for AI-driven drug discovery continues to accelerate, with billion-dollar investments flowing into end-to-end platforms driven by models and compute, rather than single drug assets.

Underpinning this trend is the proliferation of AI reasoning workflows that accelerate biomedical research and large integrated datasets spanning genomics, transcriptomics, proteomics, metabolomics, and more. Together, these capabilities are enabling more powerful models of biological complexity for a new era of programmable therapeutics guided by prediction and rational design.

“This isn’t about developing therapeutics for a particular indication or target,” explained Max Jaderberg, PhD, president of Isomorphic Labs, on the Training Data podcast. Instead, the Google DeepMind spinout is building a general design engine applicable to any disease area.

Investors and pharma giants have rallied behind that vision. In May, Isomorphic announced a whopping $2.1 billion raise led by Thrive Capital. The AI drug developer has also secured major partnerships with Novartis, Eli Lilly, and Johnson & Johnson to embed AI-driven discovery workflows into pharma’s R&D pipeline.

While traditional drug discovery programs can be limited to known binding pockets revealed by structural biology, Isomorphic’s platform, known as IsoDD (Isomorphic Labs Drug Design Engine), expands the druggable landscape by probing previously inaccessible biology.

The platform’s capabilities include predicting induced-fit interactions, in which proteins change shape upon ligand binding, and identifying cryptic binding pockets that remain hidden in the absence of a ligand. IsoDD is also versatile across multiple drug modalities, including de novo antibodies and other large biologics.

The Isomorphic Labs Drug Design Engine is able to predict the location of cryptic pockets at protein interfaces. A cryptic pocket is a ‘hidden’ binding site on a protein that is invisible under normal conditions but opens up when a specific molecule interacts with it. [Isomorphic Labs]
Isomorphic is only one vignette of DeepMind’s growing influence in life sciences. The AlphaFold developer is now building the AI scientist to accelerate the scientific method. In May, the team published a Nature study describing Co-Scientist, a multi-agent system built with Google’s Gemini that demonstrated an array of therapeutic applications, including drug repurposing, novel target discovery, and explaining mechanisms of anti-microbial resistance.

Decoupled from clinical proof

The industry’s investment in AI extends well beyond Isomorphic Labs. In recent months, a wave of major partnerships has emerged to train biological foundation models with proprietary datasets from leading pharma companies.

In May, Genesis Molecular AI and Incyte announced an expanded collaboration with a potential payoff that exceeds $1 billion. The partnership will apply the GEMS (Genesis Exploration of Molecular Space) platform for protein-ligand structure and property prediction across a wider set of difficult targets in Incyte’s pipeline, while incorporating Incyte’s proprietary data to improve GEMS’s performance.

Just two weeks later, AI biologics company Chai Discovery unveiled a licensing agreement with Pfizer that provides the pharmaceutical giant with early access to Chai-3, the company’s AI model for de novo antibody design, as well as a custom model trained on Pfizer’s proprietary data.

Meanwhile, Lilly has emerged as one of the industry’s most aggressive adopters of AI. In addition to securing its own AI-focused partnership with Chai in January, Lilly recently selected Tamarind Bio to host the inference infrastructure for TuneLab 2.0, a federated AI/ML drug discovery platform that gives biotech partners access to models trained on Lilly’s proprietary data.

Observing this massive investment into AI-native biotechs, commentators on social media were quick to note that few AI-designed drugs have reached the clinic.

In Isomorphic’s case, biotech and AI analyst Andrii Buvailo, PhD, posits that Thrive and Google’s parent company, Alphabet, have deep conviction in the company’s platform, AlphaFold lineage, and pharma partnerships, and are locking in ownership before clinical data resets the company’s valuation.

The alternative scenario, writes Buvailo on LinkedIn, is that the AI drug discovery valuation cycle has fully decoupled from clinical proof, and “we are watching capital chase computational promise on its own terms.”

Previously unsolvable

As the AI biology ecosystem grows increasingly crowded, some investors are explaining how they make their bets.

For Rohan Ganesh, a partner at Obvious Ventures, differentiation comes from pursuing problems that others are unable to tackle. He points to Obvious portfolio company, Inceptive, which is developing foundation models for sequence-based medicines that generalize across programs, including RNA interference (RNAi) therapies that silence disease-causing genes.

Benedetta Bernasconi, part of Inceptive Operations, observes automated RNA synthesis at the Inceptive wet lab in Palo Alto. [Inceptive]
Inceptive is led by CEO Jakob Uszkoreit, co-author of the seminal paper, “Attention Is All You Need,” which introduced the transformer architecture underpinning today’s large language models. Recently, the company announced a collaboration with Alnylam Pharmaceuticals to advance small interfering (si)RNA design by modeling target mRNAs while jointly exploring novel chemical modifications to enhance potency and efficacy. That partnership is worth up to $2 billion with upfront consideration of $30 million.

Ganesh also argues that owning business outcomes may be the most important aspect of differentiation. As an example, another Obvious-backed company, Inductive Bio, builds virtual labs that combine AI chemistry assistants, predictive ADMET (absorption, distribution, metabolism, excretion, and toxicity) and PK (pharmacokinetics) models, and human-relevant digital organ technologies to surface key risks earlier and accelerate candidate nomination timelines by months.

The platform gained external validation in February, when Inductive placed first in the OpenADMET-ExpansionRx blind challenge, a benchmarking competition in which participants predict properties of previously unseen compounds from real-world drug programs.

“A model that’s accurate but doesn’t change the pace or probability of success in the clinic is meaningless,” Ganesh told GEN.

An abstract visualization of RNA and cellular biology, representing how generative AI and biological foundation models can design and optimize RNA sequences, enabling the development of therapeutics with specific functional properties. [Inceptive]
When Jim Tananbaum, MD, founded Foresite Capital in 2011, he believed that data, science, and machine learning were going to dominate the conversation for the foreseeable decades. Foresite was among the early investors in data generation for causal analysis and went on to back some of the leading players in the genomics space, including 10x Genomics and Element Biosciences.

A key metric of AI’s success, according to Tananbaum, is whether the technology can unlock previously intractable problems, such as neurological disease. In this vein, Foresite-backed Insitro, founded by CEO Daphne Koller, PhD, announced an expanded collaboration with Bristol Myers Squibb to advance a broadened portfolio of therapeutic programs for amyotrophic lateral sclerosis (ALS) in March.

Foresite is also among the investors of closely watched AI unicorn, Xaira Therapeutics, which launched in 2024 with more than $1 billion in funding. Xaira has spent its initial years building virtual cell models trained on scalable single-cell perturbation datasets to advance target and mechanism-of-action discovery, patient stratification, and toxicity prediction.

“Genetic, biochemical, and multiomic data go hand-in-hand in untangling the biological relationships that will be fundamental for automating discovery,” Tananbaum told GEN.

Window for innovation

Jory Bell, general partner at Playground Global, concurs that “the special sauce” is in the data, not the model. He cites portfolio company Manifold Bio, which is building an AI-driven platform that scales in vivo measurements for biologics, such as PK and biodistribution, valuable for addressing challenges in tissue-specific delivery.

“Any biotech startup these days will be using AI as a core part of workflow, so the critical question is how you actually apply the AI,” Bell told GEN.

Simon Barnett, partner at Dimension, describes an investment thesis where small, focused groups effectively using machine learning will be wildly successful, regardless of whether they pursue therapeutic assets.

Notably, Dimension led Tamarind’s $13.6 million Series A in February, betting that as biology foundation models mature, the industry will move from piecemeal adoption to large-scale deployment of integrated model ecosystems.

“Platform companies need strong, informed views on whether frontier AI labs may eventually subsume their technology,” says Barnett. “Everyone needs something uniquely valuable that confers a durable advantage, whether it’s their team, cycle time, data assets, structural positioning, or something else.”

Dimension’s early bets paid off earlier this year, when portfolio company Coefficient Bio, a roughly 10-person AI drug discovery start-up founded by former Genentech scientists, was acquired by Anthropic for $400 million.

At SynBioBeta’s annual conference in May, Eric Kauderer-Abrams, PhD, head of biology and life sciences at Anthropic, said the team has focused primarily on the technical core, training AI assistant, Claude, in scientific fundamentals spanning chemistry, structural biology, and bioinformatics.

“Our thinking with the [Coefficient] acquisition was to accelerate the other side for biotech operators,” said Kauderer-Abrams. “How do we actually plan out and manage a biotech program from start to finish and make choices along the way?”

Taken together, Dov Gertz, PhD, co-founder and CEO of Converge Bio, reiterates that modern AI, particularly deep neural networks and their derivatives, has powered a dramatic transition from predictive modeling to generative design. However, the shift is still early, having only taken hold in the past decade. “Don’t expect a generatively designed molecule to reach patients for another seven years,” he tempered on LinkedIn.

Nevertheless, now is the time to invest.

“If you wait for that first FDA approval before engaging with the technology, you’ve likely already missed the most valuable window for innovation,” wrote Gertz. “Drug discovery rewards those who can see where the field is heading, not just where it is today.”

While time will tell how these bets translate in the clinic, one belief is deepening across the industry: that AI’s most important application is to improve human health.

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]]> Chimeric Allergen Receptor Treg Cells Suppress Allergic Asthma in Mice https://edusehat.com/en/chimeric-allergen-receptor-treg-cells-suppress-allergic-asthma-in-mice https://edusehat.com/en/chimeric-allergen-receptor-treg-cells-suppress-allergic-asthma-in-mice Tue, 07 Jul 2026 03:05:08 +0700 Edusehat Chimeric, Allergen, Receptor, Treg, Cells, Suppress, Allergic, Asthma, Mice Genetically engineered CAR T cells expressing artificial receptor proteins are increasingly used in the clinic to boost the immune system’s response against leukemias and other cancers. Researchers at Lausanne University Hospital and University of Lausanne, and at Center for Human Immunology Lausanne, have now adapted this approach to suppress the immune system’s response to a common birch pollen allergen. The investigators developed regulatory T cells (Tregs) armed with chimeric allergen receptors (CAlleR Tregs), which in tests reduced or preventing asthma symptoms in mice sensitized to the allergen. The team suggests that their technique could eventually be used to treat a wide variety of allergies in humans.

“Our study provides proof-of-concept and preclinical evidence that CAlleR Tregs redirected against a birch pollen allergen can downmodulate birch pollen–induced allergic asthma,” said study lead Yannick D. Muller, MD, PhD, an associate professor at Lausanne University Hospital and the University of Lausanne.

Muller and colleagues reported on their study in Journal of Experimental Medicine (JEM) in a paper titled “Chimeric allergen receptor regulatory T cells suppress birch pollen allergic airway inflammation,” concluding “These findings unveil a novel mechanism for targeting soluble antigens and highlight the potential of CAlleR Tregs to prevent and treat severe allergies.”

Asthma affects over 300 million people worldwide, around 60% of whom suffer from allergic asthma. Allergens trigger an immune response in a patient’s airways, causing inflammation, excessive mucus production, and difficulty in breathing. “Allergic asthma is driven by an exacerbated type 2 immune response, characterized by the overproduction of IL-4, IL-5, and IL-13 by Th2 cells,” the authors explained. These cytokines trigger events that ultimately result in airway hyperresponsiveness, and airway mucus plugging, which is the primary cause of death in asthma.

Allergen immunotherapy (AIT), which involves the administration of gradually increasing doses of allergen, is the only treatment that addresses the underlying cause of asthma. Yet, it is not recommended for patients with severe asthma, representing the most vulnerable population at greatest risk of asthma-related morbidities and mortality. “This highlights the need for new, safe, and durable treatments for restoring allergen tolerance in severe allergic asthma,” Muller commented.

Regulatory T cells (Tregs) are immune cells that can dampen the body’s immune responses and prevent excessive inflammation. Tregs are being investigated as potential therapies for a variety of inflammatory and autoimmune disorders. “Importantly, Tregs can be expanded ex vivo and reinfused with multiple clinical trials evaluating their potential in autoimmune and inflammatory disorders,” the team continued. “However, Treg therapy has shown only limited efficacy, which has been mostly attributed to the lack of antigen specificity.”

Muller and colleagues wondered whether they could boost the therapeutic potential of Tregs by genetically engineering them to express receptor proteins that recognize specific allergens. This approach is analogous to the CAR T cell method that is now commonly used to treat cancers: cytotoxic T cells are engineered to express chimeric antigen receptors that specifically recognize proteins on the surface of cancer cells, directing the immune system to attack and kill the tumor cells.

A leading cause of allergic asthma is birch tree pollen, to which 8–16% of the European population are sensitive. The birch allergen Bet v1 is the most abundant allergenic protein, the authors commented. And while AIT for birch pollen–associated rhinitis and asthma has been shown to be effective, it is contraindicated for patients with severe and uncontrolled asthma. “This highlights the unmet need for new, safe, and durable treatments for restoring allergen tolerance in severe allergic asthma.”

Muller’s team constructed chimeric allergen receptors (CAlleRs) that specifically recognize the Bev v1 component of birch tree pollen. These CAlleRs were based on antibodies isolated from a birch-allergic patient, linked to protein signaling domains that can activate Treg cells. “We identified and characterized four novel anti–birch-specific antibodies and generated single-chain variable fragments (scFvs) fused to a CD28-ζ signaling domain,” the investigators noted.

Exposure to the birch pollen allergen, when stabilized by noncompetitive antibodies, boosted the suppressive activity of Tregs expressing these CAlleRs. The researchers found that simultaneous binding by a CAlleR and a non-competing antibody promotes receptor–allergen cross-linking underlying a novel mechanism to induce T cell activation by soluble antigens. “This mechanism opens new avenues not only for rewiring synthetic receptors against any soluble antigens including autoantigens for therapeutic intervention but also for delineating a more global pathway for antigen cross-presentation in allergies.”

Muller and colleagues injected these CAlleR-expressing Tregs into mice that were already allergic to birch pollen. When these treated animals were re-exposed to birch pollen, they showed decreased signs of allergic inflammation, reduced mucus production, and increased lung function. Next, the researchers injected CAlleR-expressing Tregs into mice that had never been exposed to birch pollen. When these animals were subsequently exposed to pollen, they failed to develop any asthma symptoms.

The team concluded, “These findings unveil a novel mechanism for targeting soluble antigens and highlight the potential of CAlleR Tregs to prevent and treat severe allergies.”  Muller added, “Future studies should evaluate the persistence and stability of CAlleR Tregs over time and define the optimal modalities for implementing such a therapeutic approach.” CAlleRs could also be developed that specifically suppress the immune response to other common allergens, including house dust mites or certain food allergens.” Future work should evaluate whether such approach could also be suitable to restore tolerance against food allergies,” the investigators said.

The post Chimeric Allergen Receptor Treg Cells Suppress Allergic Asthma in Mice appeared first on GEN - Genetic Engineering and Biotechnology News.

]]> ALS Drug Extends Survival in Mice, Targets TDP-43 Low-Complexity Domain https://edusehat.com/en/als-drug-extends-survival-in-mice-targets-tdp-43-low-complexity-domain https://edusehat.com/en/als-drug-extends-survival-in-mice-targets-tdp-43-low-complexity-domain Mon, 06 Jul 2026 23:30:11 +0700 Edusehat ALS, Drug, Extends, Survival, Mice, Targets, TDP-43, Low-Complexity, Domain In a new study published in Nature Aging titled, “Therapeutic targeting of the conserved region within the low-complexity domain of TDP-43 is neuroprotective and extends survival in amyotrophic lateral sclerosis mice,” researchers from University of Arizona present a new therapeutic target to shield nerve cells from the damage of ALS.  

“Current FDA-approved treatments for ALS provide only modest benefits. There is an urgent need for a real breakthrough,” said Xinglong Wang, PhD, corresponding author of the study a professor at the R. Ken Coit College of Pharmacy.  

ALS is difficult to treat because diagnosis occurs after substantial nerve cell damage. Causes of ALS are unclear. Fewer than one in 10 cases are inherited through a known genetic mutation. More than 90% of cases arise sporadically with no family history or clear genetic cause. However, nearly all cases demonstrate abnormal TDP-43 aggregation inside nerve cells, which often informs post-mortem diagnosis. 

“We asked a simple question that had never been tested: is there one specific part of TDP-43 that’s causing the harm, something a drug could switch off without disturbing the rest?” Wang said.  

The team found a region of TDP-43 were disease-causing mutations clustered. When this region was deleted in mice, the nerve cell death caused by TDP-43 dropped sharply while normal protein function remained intact. The researchers identified experimental drug, XL20, which could latch onto the target region in the TDP-43 protein. Notably, the drug could cross the blood-brain barrier. 

In mice, the XL20 extended median survival by approximately a week, protected nerve cells and reduced muscle weakness. When XL20 was tested on human motor neurons, the specialized nerve cells in the brain and spinal cord, the experimental drug reversed damage.

Wang says XL20 represents a promising candidate for future clinical development. As ALS typically develops over months to years after symptoms first appear, earlier treatment could provide greater opportunity to slow disease progression.  

Additionally, the study’s findings may have applications for other neurological diseases. The same TDP-43 pathology is central to limbic-predominant age-related TDP-43 encephalopathy (LATE), a common dementia which affects roughly one in three people over 80. TDP-43 pathology is also found in more than half of Alzheimer’s patients and is associated with faster cognitive decline. 

“The same TDP-43 pathology is implicated in several other neurodegenerative diseases,” Wang said. “If future studies show this approach works in those diseases as well, it could eventually benefit a much larger patient population.” 

The post ALS Drug Extends Survival in Mice, Targets TDP-43 Low-Complexity Domain appeared first on GEN - Genetic Engineering and Biotechnology News.

]]> StockWatch: New UC Data Sparks Smoother Sailing for Abivax https://edusehat.com/en/stockwatch-new-uc-data-sparks-smoother-sailing-for-abivax https://edusehat.com/en/stockwatch-new-uc-data-sparks-smoother-sailing-for-abivax Mon, 06 Jul 2026 05:35:08 +0700 Edusehat StockWatch:, New, Data, Sparks, Smoother, Sailing, for, Abivax Less than a month after its stock roller-coastered on safety signals associated with its late-stage ulcerative colitis (UC) drug candidate obefazimod, shares of Abivax (Euronext Paris and Nasdaq: ABVX) enjoyed smoother sailing this past week—namely a 63% surge in Europe and a 50% leap in the United States over four trading days, following more positive data that appeared to reassure investors.

Abivax declared that obefazimod “delivered meaningful clinical benefit” to adults with moderately to severely active UC in the ABTECT Maintenance Part 2 supplemental portion of its Phase III UC maintenance program, with 37.2% of induction nonresponders achieving clinical remission and 34.5% achieving endoscopic remission at Week 44 following continued 50 mg treatment. Of those patients, 61.5% also showed clinical response, 48.0% endoscopic improvement, and 44.6% Histologic-Endoscopic Mucosal Improvement (HEMI).

In patients whose doses were escalated to 50 mg, clinical remission was recaptured in 45.5% of patients who relapsed during ABTECT Maintenance Part 1—a result Abivax said supported a practical dose-escalation strategy for regaining and sustaining disease control over time.

Of special interest to investors, no new safety signals were seen since earlier this month, when Abivax disclosed various malignancies in nine patients among the 580 enrolled in the study. The earlier disclosure triggered price plunges of 44% for both Abivax’s ordinary shares traded on Euronext Paris and the company’s American depositary shares (ADSs) traded on the Nasdaq Global Market.

Established risk factors

The latest data from ABTECT Maintenance Part 2 showed four total cases of non-melanoma skin cancer (NMSC)—two in the study’s 25 mg arm, two in the 50 mg arm: “All occurred in patients with established NMSC risk factors including advanced age, thiopurine use, prior skin cancer history, and failure of multiple prior advanced therapies,” Abivax stated.

Significantly, incidence rates of malignancies (including NMSCs) when adjusted for patient-year exposure were well within the pre-defined background reference ranges based on previous UC studies.

Exposure-adjusted incidence rates (EAIRs) for malignancies excluding NMSC were 0.48 and 0.69 events per 100 person-years (PYs) in the all-active combined (50 mg + 25 mg) and 50 mg cohorts, respectively, and for NMSC were 0.95 and 0.69 events per 100 PYs, in the all-active combined (50 mg + 25 mg) and 50 mg cohorts respectively, all consistent with expected UC background rates

EAIRs for malignancies excluding non-melanoma skin cancer (NMSC) were 0.48 per 100 PYs in the all-active combined (50 mg + 25 mg) cohort, and 0.69 events per 100 PYs in the 50 mg cohort. For NMSC, EAIRs were 0.95 in the all-active combined cohort and 0.69 in the 50 mg cohort. All those results were consistent, Abivax said, with expected UC background rates ranging from 0.30–0.70 for malignancies excluding NMSC, and 0.70–1.40 for NMSC.

“Paradigm-defining treatment”

“The expanded cumulative safety data further strengthens our confidence in the long-term safety profile of obefazimod and reinforces the favorable benefit-risk profile for our program as we prepare for our planned NDA [New Drug Application] submission later this year,” Abivax CEO Marc de Garidel stated. “We believe this growing body of evidence positions obefazimod, if approved, to become a paradigm-defining treatment option for patients living with ulcerative colitis.”

Investors appeared to share de Garidel’s optimism. The data sparked a buying surge among investors, who sent Abivax shares traded on Euronext Paris soaring 39% the day after the announcement, from €83.30 ($94.71) to €115.50 ($131.31) on Tuesday. The shares rose another 1.7% Wednesday, closing at €117.50 ($133.59), then climbed another 9% Thursday to €127.80 ($145.28) before finishing the week with a 6% increase, to €135.80 ($154.38) and a 63% one-week gain.

On Nasdaq, Abivax ADSs surged 50% for the week, consisting of a roughly 39% leap Tuesday from $96.15 to $133.26. From there, shares dipped 0.5% the following day to $132.56, before rebounding 9% Thursday, finishing the Independence Day holiday-shortened week at $144.65.

Wednesday was a shorter trading day than usual since the company requested a temporary, single-day halt. Abivax requested the halt to price an upsized offering of its U.S. American depositary shares (ADSs), which increased from the originally announced $600 million to $800 million—6.4 million ADSs at $125 per ADS, which the company expected would extend its cash runway into the second quarter of 2029.

The offering closed Thursday at $920 million, of which approximately $874.1 million consisted of net proceeds, after underwriters exercised in full their option to purchase 960,000 additional ADSs representing 15% of the total number initially sold in the offering.

The size of the offering appeared, based on investor chatter cited by Stocktwits, an effort to dampen speculation about Abivax being a prime candidate for a buyout; the company appears in GEN’s most recent A-List of Top 10 Takeover Targets of 2026. But the upsizing of the offering rekindled buyout speculation by individual or “retail” investors, the same outlet reported Thursday.

Abivax said it intends to use the net proceeds toward expenses relating to potential commercialization of obefazimod in the United States; clinical R&D expenses, primarily related to UC and Crohn’s disease; and the remainder, if any, for general corporate purposes.

Leerink Partners, Morgan Stanley, Piper Sandler, and Guggenheim Securities were joint bookrunning managers for the offering, while LifeSci Capital acted as a passive bookrunning manager and Van Lanschot Kempen as the lead manager.

“Response rates (clinical & endoscopic remission) in this portion also appear compelling, especially given the refractory nature of patients in this subset, reaffirming obe’s best-in-disease efficacy,” Thomas J. Smith, senior managing director, immunology and metabolism, and a senior research analyst with Leerink Partners, commented in a research note.

“Should allay investor concerns”

“We believe this update further de-risks obe’s profile in UC and Crohn’s and should allay investor concerns following Part 1 maintenance data released earlier this month,” Smith added.

Smith raised his firm’s 12-month price target on Abivax shares 6%, from $140 to $148.

Two other firms also raised their price targets on Abivax stock:

  • BTIG (Julian Harrison)—Up 17%, from $150 to $175, maintaining “Buy” rating.
  • Wedbush Securities (David Nierengarten)—Up 22% from $90 to $110, maintaining “Neutral” rating.

Even more positive feedback on the latest data came from Faisal Khurshid, a managing director and equity research analyst with Jefferies. Khurshid upgraded his firm’s rating on Abivax’s stock from “Hold” to “Buy,” and boosted Jefferies’ price target 46%, from $108 to $158.

On June 1, Khurshid downgraded Jefferies’ rating on Abivax from “Buy” to “Hold,” citing the safety concerns he said have since been addressed.

“Mgmt. did a nice job addressing investor concerns w/ how they presented the safety data [June 29] vs. the Part 1 update. On top of that, the efficacy profile strengthens w/ each add’l piece of data,” Khurshid observed.

He also cautioned: “There is still risk on cash runway, catalyst path, and commercial needs for a pot’l standalone launch. But ultimately, good data should generate value.”

The biggest outstanding risk for Abivax, Khurshid wrote, is the need for significant resources associated with a commercial launch for an indication in inflammatory bowel disease (IBD): “We still think pot’l of asset better realized w/ a strategic partner.”

Obefazimod is a small molecule upregulator of miR-124, an anti-inflammatory microRNA. It enhances the selective splicing of a single long noncoding RNA to generate miR-124, which downregulates cytokines and chemokines shown to promote inflammation, including tumor necrosis factor (TNF) alpha, IL-6, monocyte chemoattractant protein-1 (MCP-1), and IL-17, as well as Th17+ cells.

Under its former name ABX464, obefazimod was initially developed against HIV but was repurposed to fight inflammatory conditions based on its anti-inflammatory effect.

Leaders and laggards

  • Elicio Therapeutics (Nasdaq: ELTX) shares tumbled 37% from $5.14 to $3.22 Thursday after the developer of immunotherapies for high-prevalence cancers said it entered into a definitive securities purchase agreement led by two new “fundamental institutional investors” with participation from a large existing shareholder—all undisclosed—to purchase 4,380,313 shares of Elicio common stock through a registered direct offering. The offering is expected to result in gross proceeds of approximately $15 million before deducting placement agents’ fees and other expenses, Elicio said. Titan Partners, a division of American Capital Partners, is acting as lead placement agent while B. Riley Securities is acting as co-placement agent.
  • Takeda Pharmaceutical (Tokyo Stock Exchange: 4502) shares increased 2.4% from ¥5,150 ($31.91) to ¥5,274 ($32.68) Thursday and rose another 1.6% to ¥5,359 ($33.20) after the pharma announced an up to $600 million artificial intelligence (AI)-based drug discovery collaboration with Insilico Medicine (Hong Kong Exchange: 3696.HK). Insilico agreed to use its end-to-end platform in leading AI-driven discovery to identify molecules meeting predefined scientific and early development criteria, while Takeda agreed to apply its global development capabilities to advance selected candidates through clinical validation across its therapeutic areas. Takeda gained exclusive worldwide rights to develop, manufacture, and commercialize novel therapeutics selected through the collaboration. Takeda’s American depositary shares (NYSE: TAK) rose 5% from $15.95 to $16.77 Thursday (U.S. markets were closed Friday for the Independence Day holiday). Insilico shares fell 4.1% from HKD 39.62 ($4.97) to an even HKD 38 ($4.77) Thursday and slid 1.6% to HKD 37.38 ($4.66) Friday.

The post StockWatch: New UC Data Sparks Smoother Sailing for Abivax appeared first on GEN - Genetic Engineering and Biotechnology News.

]]>  A device that revives eyeballs from dead donors could make eye transplants possible https://edusehat.com/en/a-device-that-revives-eyeballs-from-dead-donors-could-make-eye-transplants-possible https://edusehat.com/en/a-device-that-revives-eyeballs-from-dead-donors-could-make-eye-transplants-possible Sat, 04 Jul 2026 07:05:02 +0700 Edusehat  A, device, that, revives, eyeballs, from, dead, donors, could, make, eye, transplants, possible
  • A device that keeps eyes alive: Researchers have built a device that pumps oxygen-rich fluid through a removed eyeball's artery, slowing the rapid degeneration that makes whole-eye transplants so difficult. Pig eyes kept inside it stayed viable for up to 10 hours.

  • Dead eyes that can still "see": Untreated pig eyes lost the ability to respond to light the moment they were removed — but after just 15 minutes of perfusion inside the device, that ability came back.

  • Human eyes next: The team tested the device on eyes from six deceased donors, and found the perfused eyes preserved their retinas significantly better than untreated ones — a promising early sign for eventual transplantation.

  • A long road ahead: Whether eyes treated this way could actually restore sight won't be known until one is successfully transplanted — something that has never yet worked in a human patient.

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    It’s not easy to transplant a whole human eye. The surgery is difficult. And the eyes themselves start to degenerate as soon as they’ve left the body. When surgeons attempted it a few years ago, the newly-transplanted eye wasn’t able to see.

    But researchers believe they might have a solution: a device that maintains and revives freshly removed eyeballs using a technique called perfusion. Perfusion works by providing surgically-removed organs with some of the oxygen and nutrients they typically get when they’re inside a body. Treated eyes don’t degrade as quickly, and appear to retain the ability to transmit electrical signals, and potentially see. The device could one day make eye transplantations a viable possibility.

    “It’s really cool,” says Shannon Tessier at Massachusetts General Hospital, who was not involved in the research but studies perfusion of other organs. “It could be a new frontier for retina preservation.”

    Pia Cosma at the Centre for Genomic Regulation at the Barcelona Institute of Science and Technology in Spain and her colleagues have spent years developing their device. The Eye-in-a-Care-Box (ECaBox), as they call it, delivers an oxygen-rich supply of fluid through the artery that normally supplies the eye with blood.

    The eye itself sits on a “bed,” and excess fluids are drained away. And while the device itself is sealed to maintain a specific temperature and pressure, a clear window on its side allows researchers to study and image the eye while it’s inside.

    Cosma and her colleagues started experimenting with pig eyes, which are anatomically similar to human eyes but easier to get hold of (the team got theirs from a local slaughterhouse).

    Pig eyes that are kept at room temperature outside of the device start to degenerate pretty quickly. The team found that cells in the eye shrank, and the eyes started to lose their structure. Cooling the organs didn’t help preserve them, either—the eyes degenerated within 24 hours even when they were kept at 4°C (39°F).

    But eyes kept in the EcABox fared much better. 24 hours later, tests suggested the prefused eyes were “significantly more viable” than eyes that hadn’t been maintained in the device.

    The perfused eyes also seemed to be able to respond to light, suggesting they might technically be able to see if they were transplanted. Untreated pig eyes lost this ability as soon as they were removed from the animal. But it came back after about 15 minutes of perfusion, according to the scientists behind the work. A few of the treated eyes kept going for 10 hours or more.

    Cosma and her colleagues described the work in a preprint article that has not yet been peer reviewed, and did not want to comment on the work.

    After success with the pig eyes, the team members then tested their device on human eyes. They first collected 12 eyes from six people who had died. In each case, one of each pair of eyes was put in the device, while the other was not. Again, the perfused eyes did better—and their retinas were preserved.

    Cosma and her colleagues hope that their device could offer scientists a new way to study eye treatments—one that doesn’t involve experimenting on living animals. They also hope that, with some improvements, the ECaBox might provide a way to maintain and revive donated human eyes for whole-eye transplantation.

    Whole-eye transplants have been attempted in the past, mostly in research animals, with limited success. In May 2023, a team at NYU Langone transplanted an eye along with part of a face to a man who had survived a high-voltage electrical accident that resulted in the loss of much of the left side of his face, including his left eye, two years earlier. Although the man recovered well, he wasn’t able to see out of the transplanted eye.

    We won’t know whether eyes treated in the ECaBox could do any better until they have been transplanted, says Tessier. 

    In the meantime, Cosma and her colleagues plan to use a newer version of their device to collect more human eyes for research. “We are planning to develop a portable, surgery-room ECaBox to minimize [degradation] in heart-beating donor eyes, when they become available,” they write.

    ]]>     The UK’s generational tobacco ban might not work. I’m supporting it anyway. https://edusehat.com/en/the-uks-generational-tobacco-ban-might-not-work-im-supporting-it-anyway https://edusehat.com/en/the-uks-generational-tobacco-ban-might-not-work-im-supporting-it-anyway Fri, 03 Jul 2026 20:20:02 +0700 Edusehat The, UK’s, generational, tobacco, ban, might, not, work., I’m, supporting, anyway.
  • A permanent, generational ban: The UK's new Tobacco and Vapes Act permanently bans tobacco sales to anyone born after January 1, 2009—forever, no matter their age. It's an unprecedented "endgame" approach designed to eliminate smoking entirely, not just reduce it.
  • No guarantee it will work: New Zealand passed a similar law in 2022, only to see it repealed two years later. The Maldives is the only country that has actually implemented one, and it's too soon to know if it's working.
  • A once-radical idea going mainstream: Eleven years ago, advocates pushing generational bans were told they were crazy. Today, 23 Massachusetts towns have enacted similar policies, and the UK law has health agencies worldwide asking: can we do this here?
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    As the parent of two little girls, I often think about how their childhood is different from mine. The seven-year-old is learning about AI at school. The five-year-old is given internet-based homework every week. And they are both absolutely repulsed by the idea of smoking.

    That was not the prevailing sentiment when I was young. My parents smoked. The customers at our family’s restaurant smoked. Cartoon characters smoked. My friends and I would buy little cigarette-box-shaped packets of sugary white sticks and pretend to smoke in the playground. Smoking was a central part of our culture.

    Which is why the UK’s recent passing of a generational sales ban on tobacco products feels like such a big deal. As part of the Tobacco and Vapes Act 2026, retailers are prohibited from selling tobacco products to anyone born after January 1, 2009, in perpetuity. It doesn’t matter when those people turn 18—or 38 or 68, for that matter. It will always be illegal to sell to anyone born after that date.

    This is what’s described as an “endgame” approach. While many tobacco control strategies—such as taxation or gory imagery—aim to reduce consumption, policies like the UK’s are designed to eliminate it entirely. It’s a new approach, and no one knows whether it will work.

    The Maldives was the first country to implement a generational smoking ban, in November last year. It’s too soon to say how that has panned out.

    Nor do we know if these laws will even last. In 2022, New Zealand passed a similar generational sales ban as part of a broader anti-smoking law. But it was never enacted—the law was repealed by a new government in February 2024.

    In the UK, both major parties support the ban. But Nigel Farage, whose right-wing party has seen a recent surge in support, has promised that “the generational smoking ban will not last long if Reform gets the chance to start rebuilding our mismanaged country.”

    Chris Bostic, an attorney and former policy director for the advocacy group Action on Smoking and Health, says he and his colleagues began promoting the idea of a generational ban in the United States 11 years ago. Back then, they struggled to win support, even from major health charities. “People said we were crazy … [and] that this was impossible,” he says. Opponents argued that bans would infringe on personal freedoms.

    “The public health argument is: Well, what about freedom from addiction?” says Britta Matthes, a tobacco control researcher at the University of Bath in the UK. Most people who smoke began when they were teenagers, want to quit, and wish they’d never started. Tobacco is arguably the most harmful consumer product of all time. It will kill half its users who don’t quit, according to the World Health Organization.

    It also kills people who don’t smoke. Of the 7 million who die from tobacco every year, 1.6 million are nonsmokers who were exposed to secondhand smoke, according to the WHO.

    Generational sales bans are a long-term strategy that will only protect future smokers. Most experts agree that people who already smoke should be a main consideration for any policy, and that a multipronged approach is probably the best way to go. Janet Hoek at the University of Otago, who has explored tobacco control policies in New Zealand, believes that enforcing very low limits on nicotine levels and banning filters—an environmental scourge that does not make smoking safer, as many people believe—might be a “powerful combination,” for example.

    But preventing teenagers from starting to smoke in the first place is an enticing prospect, even among the majority of people who smoke. And it’s starting to look a lot less radical.

    The US has quietly been making progress on a smaller scale. Since 2021, Brookline, a town in the Boston area, has banned the sale of tobacco products to anyone born after January 1, 2000. The idea has spread. Today there are 23 towns in Massachusetts with similar bans, says Bostic. Nine towns across Minnesota, New York, and California have implemented other endgame policies.

    The UK law has normalized the idea more than ever, he adds. His colleagues are already fielding calls from health agencies around the world. “People [are] saying, Wow I can’t believe the UK just did this—can we do this here?” he says.

    Norms change. Like many other millennials, I vividly remember my first night out after a ban on indoor smoking took effect. My clothes didn’t stink! My hair still felt clean! And my throat wasn’t scratchy the next morning! Now that’s just normal. I hope a tobacco-free world can be the new normal for my kids.

    ]]>     Synthetic Organizers Aid Creation of Reproducible Kidney Organoids from Stem Cells https://edusehat.com/en/synthetic-organizers-aid-creation-of-reproducible-kidney-organoids-from-stem-cells https://edusehat.com/en/synthetic-organizers-aid-creation-of-reproducible-kidney-organoids-from-stem-cells Fri, 03 Jul 2026 06:00:03 +0700 Edusehat Synthetic, Organizers, Aid, Creation, Reproducible, Kidney, Organoids, from, Stem, Cells University of Southern California (USC) researchers have paired a biological discovery with an engineering feat to create more faithful, reproducible kidney organoid structures, grown from human pluripotent stem cells (hPSCs). By mapping the developing human kidney, the scientists identified a previously unrecognized developmental axis that helps organize the kidney’s nephrons, which are their filtering units. The team then engineered Wnt-secreting “synthetic organizer” cells to recreate aspects of this developmental environment in organoids.

    Their advance makes the organoids more reliable models for studying disease and evaluating potential therapies, while supporting long-term efforts to generate transplantable kidney tissue. “It is important that we’re starting to get good reproducibility from organoid models that can lead to robust preclinical models of cell function and disease to benefit patients,” said Nils Lindström, PhD, assistant professor of stem cell biology and regenerative medicine at the Keck School of Medicine of USC. Lindström is co-corresponding author of the team’s published report in Science, titled “Patterning human kidney organoids with synthetic Wnt-secreting organizers.” In their paper, the researchers reported, “Our findings link a spatial organizing geometry in the developing human kidney to controllable engineering in vitro.”

    “Stem cell–derived organoids have emerged as systems for modeling organ development and generating complex tissue structures in vitro,” the authors wrote. Over the past decade, organoid work has relied on cells’ ability to self-organize into tissue-like structures, often in response to adding chemicals and proteins that act broadly in the whole organoid. “Although this capacity enables organoids to recapitulate many developmental programs, it limits experimental control over tissue architecture, often producing structures that vary between cultures and are difficult to engineer reproducibly,” the team continued. “Understanding how to impose spatial patterning in organoid systems is therefore an important challenge.”

    In embryos, spatial patterning is often organized by localized signalling centers, known as developmental organizers. But how organizing geometry is controlled in the developing kidney, and whether it can be recreated in vitro, hasn’t been known.

    For their reported study, the team combined spatial transcriptomics of the developing human kidney with synthetic engineering. “We mapped this organizing geometry in developing human kidneys and tested whether a minimal cue of localized WNT signaling could restore spatial control of nephron patterning in organoids,” they explained.

    The project began by making tools to copy developmental signals. Postdoctoral researcher Fokion Glykofrydis, PhD, in the Morsut lab, engineered a “synthetic organizer” cell that secreted a Wnt protein that their spatial transcriptomics and other analyses indicated was involved in spatial patterning during kidney development. Graduate student Connor Fausto from the Lindström lab proposed an experiment to test how this Wnt-secreting cell would affect organoid nephrons.

    The experiments revealed that the synthetic organizer enabled two key processes essential for building organs: controlling the identity of cells and influencing the shape of developing structures. The synthetic organizer serves as a localized and targeted source that secretes controllable amounts of specific Wnt proteins within the organoid itself. These are key signals that help shape the developing kidney. This creates a signaling environment much more similar to a naturally developing kidney and gives researchers a way to control where and how kidney structures form.

    Co-corresponding author Leonardo Morsut, PhD, associate professor of stem cell biology and regenerative medicine, and biomedical engineering at the Keck School of Medicine and USC Viterbi School of Engineering, said, “With our approach, we are trying to control self-organization, and work with it as opposed to try to completely override it.”

    Lindström expected Wnt to trigger nephrons to change their identity into cells capable of forming connections with the urine drainage system. What surprised him was that the nephrons also changed shape and elongated toward the source of the Wnt signal, which doesn’t happen when signals are delivered uniformly to the whole organoid. Compared with the developmental process seen in traditional kidney organoids, this elongation toward the Wnt source is more similar to what happens in a naturally developing kidney.

    “A single, localized signal did two things at once. It changed what the cells became and physically pulled the tubules toward the source,” Lindström said. “You would not see that with a uniform chemical bath of signals.” Engineered WNT-secreting cellular organizers introduced into kidney organoids restored organizing geometry, the authors noted, “… biasing distal nephron differentiation and orienting nephron morphogenesis toward the signal source, which demonstrates that developmental signaling geometry can be reconstructed synthetically to control tissue patterning.”

    The team identified a previously unrecognized axis, a direction along which the developing kidney organizes itself. Developmental biologists have long known about the nephron’s classic “proximal-distal (PD) axis,” which runs from its blood-filtering end to its urine-drainage end. The new axis is defined instead by how close each part of the nephron sits to the collecting duct, the tube system that drains urine and releases Wnt signals during development. Those signals tell the nephron what shape to take and which way to point.

    “The study shows that there’s an undiscovered axis that sets up how a nephron looks and forms,” said Lindström. “It’s not every day that you find something new in human development at that level.”

    Most kidney organoids contain only nephrons and lack the collecting duct that supplies this local Wnt signal, so they have no such axis and organize in a radially symmetrical pattern. By mapping how kidney cells respond to Wnt at specific locations in the developing kidney, the team recreated that environment in organoids with the synthetic organizer, producing structures that are both more developmentally faithful and more reproducible.

    “Introducing tunable WNT-secreting synthetic organizers (SOs) in organoids restored canonical WNT responses, biased distal nephron differentiation, and oriented nephron morphogenesis toward the WNT source,” the investigators stated in summary. The combined results, they suggested, “… demonstrate that the spatial geometry observed in vivo can be reconstructed synthetically to control early nephron patterning and morphogenesis … Synthetic organizers provide a modular way to restore missing spatial interactions without reconstructing the entire collecting duct lineage, complementing approaches that rebuild collecting duct–to–nephron cellular interactions.”

    For Morsut, the synthetic organizer is one of several tools his lab is building to control how tissues form, and the one he is most excited about, because it steers development in a way that is powerful but not intrusive. “The synthetic organizer is just a little cluster of cells that don’t build anything themselves,” said Morsut. “But they produce a powerful field that aligns the stem cells and gives them a direction.”

    Synthetic organizers offer a modular strategy to reintroduce spatial signaling interactions that are often absent in conventional organoid cultures, the team suggested. “This approach should be broadly applicable to other organoid systems in which spatial signaling environments play instructive roles during development, providing a framework for linking developmental biology with the rational engineering of tissue architecture.

    Aligning cells is something embryos do repeatedly as they build themselves, Morsut noted, and the study shows it can now be put to work in an engineering setting, steering the process toward a desired outcome. “At the beginning of my talks, I always show a video of embryonic development,” said Morsut. “You start from a single cell, and you get to a complete organism, and that’s as close to magic as it gets. Now, we open a possibility of controlling this magic technology for building organs. This study shows that we can do that, and I’m excited to see what others will do in other contexts.”

    The post Synthetic Organizers Aid Creation of Reproducible Kidney Organoids from Stem Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ASMS 2026: Solving Proteomics’ Next Bottleneck https://edusehat.com/en/asms-2026-solving-proteomics-next-bottleneck https://edusehat.com/en/asms-2026-solving-proteomics-next-bottleneck Fri, 03 Jul 2026 02:20:11 +0700 Edusehat ASMS, 2026:, Solving, Proteomics’, Next, Bottleneck At the 74th American Society for Mass Spectrometry (ASMS) Conference in San Diego, the obvious story was hardware. Vendors showcased faster acquisition, higher sensitivity, alternative fragmentation, spatial workflows, and software ecosystems. New or highlighted platforms and workflows came from Waters, Thermo Fisher Scientific, Sciex, Bruker, Biognosys, and Evosep.

    But after several days of talks, posters, hallway conversations, and interviews with senior figures in mass spectrometry (MS)-based proteomics, the deeper story was not simply that instruments are getting better. The field is beginning to look past the instrument. The mass spectrometer is still central, but the question is shifting: what has to happen around it for proteomics to become clinically useful, scalable, trusted, and routine?

    Beyond the instrument

    Jennifer Van Eyk, PhD, professor of cardiology, biomedical sciences, pathology, and laboratory medicine, and director of the Advanced Clinical Biosystems Research Institute at Cedars-Sinai Health Science University, put it most directly: “I think mass spec is no longer the limitation. We have the sensitivity, the throughput, and the accuracy at discovery and targeted levels.”

    Jennifer Van Eyk, PhD [Gustav Ceder]
    That is a remarkable statement in a field long defined by instrument performance. Van Eyk was not saying that MS innovation is finished. She pointed to continuing gains in quantitation, protein structure, conformational analysis, post-translational modifications (PTMs), top-down proteomics, and protein dynamics. But for clinical impact, she argued, the next bottlenecks are increasingly sample preparation, data analysis, standardization, harmonization, and quality control.

    Joshua Coon, PhD, professor of biomolecular chemistry at the University of Wisconsin-Madison and the Pyle Chair at the Morgridge Institute for Research, saw instrument speed as the force opening new applications. Faster scanning mass analyzers are allowing deeper proteome coverage, more post-translational modification (PTM) measurements, and shorter runs. Ryan Kelly, PhD, professor of chemistry and biochemistry at Brigham Young University, framed the same shift as a throughput problem. “Now the mass spec is so fast that we need to figure out how to feed it faster,” he said. In plasma proteomics, Coon said, faster instruments, nanoparticle-based enrichment, and improved chromatography are moving the field from hundreds

    Joshua Coon, PhD [Gustav Ceder]
    toward thousands of detectable proteins in blood.

    John R. Yates III, PhD, the John Lytton Young Endowed Chair in the department of integrative structural and computational biology at Scripps Research, highlighted electron activation dissociation methods and the possibility that high-throughput workflows could push MS deeper into plasma and population-level studies. He described targeted affinity platforms as powerful for “known knowns” because they measure targets defined in advance. “But with mass spectrometry,” he added, “you can look for unknown unknowns, which is where the gold lies.”

    John R. Yates III, PhD [Gustav Ceder]
    The point cuts to the heart of where the field now stands, and a recurring ASMS tension. The future of proteomics is not a choice between platforms. It is a division of labor. Targeted affinity technologies have become central to large-scale plasma proteomics and population studies. MS remains uniquely powerful for unbiased discovery, tissue proteomics, complex sample matrices, protein modifications, structural diversity, and biology that is not yet named.

    From depth to trust

    If the first era of modern proteomics was about seeing more, the next may be about measuring better. Devin Schweppe, PhD, assistant professor in the Department of Genome Sciences at the University of Washington, described the current moment as a “duality.” Instruments can now deliver deep coverage, and computational tools are making interpretation faster. Together, he said, they are creating “a comfort level with trusting the data.”

    Devin Schweppe, PhD [Gustav Ceder]
    Trust came up repeatedly. For discovery biology, a strong signal can be enough to generate a hypothesis. For clinical practice, it is not. Van Eyk said clinical-grade assays are “way harder than people think they are.” A research study can iterate. A clinical assay has to deliver the same measurement today, in five weeks, in six months, and years later. Once a test is locked, “you can’t go, ‘Oh no, we should have had this extra protein in there,’” she said. “It’s done.”

    This distinction matters across assay types. Targeted MS methods such as multiple reaction monitoring (MRM) and parallel reaction monitoring (PRM) can provide absolute quantification, but only for preselected proteins. Data-independent acquisition (DIA), meanwhile, has moved discovery proteomics closer to translation by improving reproducibility and scalability. DIA is still often used for relative quantification, but its ability to capture patterns across tens or hundreds of proteins may become important as clinical decision-making moves beyond single biomarkers and reference intervals.

    The field is responding to these demands. David Kotol, PhD, R&D manager at ProteomEdge, discussed an independently validated nine-protein plasma panel designed to improve emergency department triage and imaging decisions for patients with suspected venous thromboembolism, compared with D-dimer alone.

    David Kotol, PhD [Gustac Ceder]
    Kotol described a shift “from relative protein measurements toward robust, multiplexed absolute quantification.” He emphasized stable isotope-labeled protein standards added early in sample preparation to monitor digestion efficiency, downstream analytical variation, and multi-peptide quantification. These standards cannot remove variation introduced during sample collection, handling, or storage. But they can make the analytical workflow more transparent and transferable.

    The clinical gap

    Mathieu Lavallée-Adam, PhD, associate professor in the department of biochemistry, microbiology and immunology and director of the specialization in bioinformatics at the University of Ottawa, gave the least glamorous answer to what still blocks clinical translation. “My answer is going to be boring,” he said. “It’s going to be education.”

    Mathieu Lavallée-Adam, PhD [Gustav Ceder]
    Lavallée-Adam argued that many clinicians and biomedical researchers still do not fully understand what modern MS can do. Too often, the outside view is still: give me a list of differentially expressed proteins. But MS-based proteomics has moved beyond lists, into proteoforms, structural information, PTMs, protein dynamics, and flexible acquisition. “We’re past that now,” he said. “The main barrier is our inability to communicate the possibilities that we offer.”

    Sasha Singh, PhD, assistant professor of medicine at Harvard Medical School, associate scientist at Brigham and Women’s Hospital, and director of proteomics research at the Center for Interdisciplinary Cardiovascular Sciences (CICS), described this translation role from inside a hospital environment. “That’s actually my role at the hospital,” Singh said. “I am a liaison between the technology and the application scientist.”

    Sasha Singh, PhD [Gustav Ceder]
    The translation is becoming harder because proteomics is diversifying. End users often need to distinguish among discovery MS, which can provide broad relative quantification; targeted MS, which can provide absolute concentrations for selected proteins; and targeted affinity proteomics, which can scale well for plasma cohorts but is limited by predefined assays and available binding reagents. Singh added that different technologies may produce profiles that do not fully overlap. Rather than treating that as a failure, she suggested it reveals something real: the circulation contains many subproteomes, and different technologies enrich different views.

    AI with guardrails

    No 2026 conference escapes artificial intelligence (AI), and ASMS was no exception. But the mood among the researchers was cautious rather than breathless.

    Lavallée-Adam said agent-based AI was dominating conversations in his part of the field. The dream is seductive: put a sample on an instrument, ask an AI agent to maximize protein identifications or optimize a method, and let it select the best protocol. But he drew a clear line between potential and reality. “Are such agents really driving change? It’s unclear at this point,” he said. “I think it’s unproven.”

    Still, AI-assisted acquisition strategies are entering workflows. Lavallée-Adam’s group works on real-time MS data acquisition, where software analyzes data as it is acquired and adapts the run to the biological question. Instead of measuring the same abundant proteins repeatedly, the system can decide it has seen enough and move on to new targets. In that sense, AI becomes less a magical oracle than an instrument assistant.

    Faster instruments are generating more data, and faster analysis is needed to keep up. Schweppe also argued that open-source tools remain essential because they let laboratories build on one another’s work rather than rebuild it.

    More than abundance

    Much of the clinical proteomics effort is focused on plasma because it is minimally invasive and suitable for screening, longitudinal sampling, and routine monitoring. But even in blood, researchers are learning that plasma is only part of the story.

    Roman Fischer, PhD, associate professor and head of the Discovery Proteomics Facility at the Target Discovery Institute, University of Oxford, pushed the conversation back toward biology. Plasma alone does not capture the full circulating system, he noted. Peripheral blood mononuclear cells, extracellular vesicles, microvesicles, and other compartments may contain disease-relevant information that conventional workflows miss. “We have to be more sophisticated in addressing the compartments of the blood,” Fischer said.

    Roman Fischer, PhD [Gustav Ceder]
    He also pointed to the proteoform problem. A single gene can give rise to many transcripts, isoforms, modified proteins, and glycosylated forms. These differences may affect activity, localization, disease pathways, and therapy response. Capturing that diversity is not possible with targeted affinity assays alone. It requires deeper characterization of the proteome, not only quantification.

    Yates offered a clinical example. His group has been developing protein-footprinting approaches that can detect conformational changes in proteins in blood. In one transthyretin amyloid cardiomyopathy project, he said, abundance alone was not the answer. The important signal was how the protein folded or misfolded. That kind of assay moves proteomics beyond proteins going up or down, into structural disease biology.

    Van Eyk’s work on remote sampling devices pointed to another future: patient-collected blood samples that make longitudinal cardiovascular studies easier, more inclusive, and better matched to real clinical questions.

    In the background was a broader translational arc: discovery, verification, clinical validation, health economics, and access. Plasma proteomics highlights included Lekha Sleno, PhD, professor at Université du Québec à Montréal, who is combining nanoparticle enrichment with isotope-enabled targeted proteomics, and a CinderBio breakfast seminar featuring Fredrik Edfors, PhD, assistant professor at KTH Royal Institute of Technology and SciLifeLab, and Simion Kreimer, PhD, senior research project advisor in the Proteomics and Metabolomics Core at Cedars-Sinai Health Science University.

    The seminar focused on accelerated plasma proteomics, rapid digestion workflows, stable isotope standards, Human Protein Atlas resources, and faster enzyme workflows that can reduce lead times. The common message was that sample preparation, quantification, and validation may become as decisive as instrument resolution.

    The next bottleneck

    ASMS 2026 was not short on technical spectacle. High-resolution instruments, electron-based fragmentation, narrow-window DIA, rapid acquisition, MS imaging, top-down workflows, and AI-enabled software all had their moment. But the most interesting conversations were less about spectacle than maturity.

    Proteomics is no longer trying only to prove that it can see more. It is trying to prove that it can measure consistently, explain biology more deeply, support drug development, fit into clinical laboratories, and eventually improve patient decisions.

    That means the next bottleneck is distributed across the ecosystem: sample preparation, standards, software, education, reimbursement, clinical menus, regulatory validation, open tools, and the ability to translate technical power into something a clinician can use.

    Longer term, integrated proteomics, other omics, imaging, clinical data, and AI may support not only single biomarkers, but interpretable molecular patterns, longitudinal trajectories, and digital-twin-like models of patient biology.

    The field spent decades making proteins visible. The next challenge is making proteomic measurements dependable enough to act on.

    The post ASMS 2026: Solving Proteomics’ Next Bottleneck appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Merck and Insilico Make Deals, Claude Science’s Debut, Vaccines for Neglected Diseases https://edusehat.com/en/merck-and-insilico-make-deals-claude-sciences-debut-vaccines-for-neglected-diseases https://edusehat.com/en/merck-and-insilico-make-deals-claude-sciences-debut-vaccines-for-neglected-diseases Fri, 03 Jul 2026 02:20:10 +0700 Edusehat Merck, and, Insilico, Make, Deals, Claude, Science’s, Debut, Vaccines, for, Neglected, Diseases More big biotech deals on the docket this week. First, Merck KGaA is buying Bio-Techne for $11.3 billion to expand its presence in high-growth life science markets. We dive into the details of this deal and then turn our attention to a $2.5 million collaboration to use artificial intelligence to find drug candidates for neuroimmune disorders. That deal involves Insilico Medicine and SK Biopharmaceuticals. Still on the theme of AI, we discuss Anthropic’s Claude Science, the latest entrant to the growing ecosystem of tech platforms specialized for biology, and a set of models for antibiotic design and vaccine target prediction. Lastly, we dig into two recent publications that discuss vaccines for Nipah virus and one of its relatives, and for treating schistosomiasis.

    Listed below are links to the GEN stories referenced in this episode of Touching Base:

    Merck KGaA to Acquire Bio-Techne for $11.3B, Expanding Life Science Tools Presence

    By Alex Philippidis, GEN Edge, June 25, 2026

    Insilico, SK Launch Up-to-$2.5B Neuroimmune AI Drug Collaboration

    By Alex Philippidis, GEN Edge, June 28, 2026

    Claude Science Is Here, Antibiotics Designed by Text Prompt Among Applications

    By Fay Lin, PhD, GEN Edge, June 30, 2026

    Schistosomiasis Vaccine Shows Strong Immune Memory in Early Clinical Trials

    GEN, June 29, 2026

    Nipah and Hendra Viruses: Antibody Cocktail Provides Complete Protection in Hamster Model

    GEN, June 26, 2026

    Touching Base Podcast

    Hosted by Corinna Singleman, PhD

    Behind the Breakthroughs

    Hosted by Jonathan D. Grinstein, PhD

    The post Merck and Insilico Make Deals, Claude Science’s Debut, Vaccines for Neglected Diseases appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Elixirgen Builds Rare Disease Pipeline Around Telomere Biology Disorders and DMD https://edusehat.com/en/elixirgen-builds-rare-disease-pipeline-around-telomere-biology-disorders-and-dmd https://edusehat.com/en/elixirgen-builds-rare-disease-pipeline-around-telomere-biology-disorders-and-dmd Fri, 03 Jul 2026 02:20:09 +0700 Edusehat Elixirgen, Builds, Rare, Disease, Pipeline, Around, Telomere, Biology, Disorders, and, DMD During the 2026 BIO International convention in San Diego, GEN sat down with Aki Ko, CEO of Elixirgen Therapeutics, to discuss the company’s multi-platform technology development efforts. The company, which was founded in 2017, is developing what it believes are breakthrough technologies that target telomere biology disorders (TBD) and aging as well as mRNA-based therapies.

    Its therapy for addressing telomere biology disorders, based on its proprietary ZSCAN4 approach, is the furthest to the clinic. The biotech company will also target other aging-related diseases with the technology. Meanwhile, its efforts in the mRNA space are currently focused on Duchenne Muscular Dystrophy (DMD), although there are plans to pursue other targets there as well. 

    Ko founded the company with CSO Minoru Ko, MD, PhD, in 2017. Elixirgen’s 15 employees are based in Baltimore in its office space in the Johns Hopkins Medical campus, although there is no affiliation with the university. This location offers some advantages to the company, according to Ko. Specifically, “we have a wet lab and an animal lab” that has “helped us go from in vivo to in vitro very quickly to test concepts or optimize formulations and things like that.” 

    Recently, Elixirgen announced an option agreement with Japan’s Nippon Shinyaku focused on DMD. Under the terms of the agreement, Elixirgen will be responsible for the development of an asset dubbed EXG-7001, a locally administered, full-length dystrophin mRNA therapeutic that is currently in preclinical development for the treatment of DMD.

    As part of the deal, Nippon Shinyaku will provide funding for the developmental costs of the therapy. Meanwhile, Elixirgen will receive an upfront payment and is eligible to receive additional development and sales-based milestone payments if the option were to be exercised. Also, Nippon Shinyaku may obtain exclusive worldwide rights to commercialize EXG-7001.

    “Current approaches for treating DMD focus on delivering or restoring an incomplete dystrophin protein, and there still remains a significant unmet need for a therapy that can successfully deliver a full-length dystrophin protein,” Ko said in comments about the announcement. “By design, EXG-7001 has the potential to deliver the full-length, complete dystrophin protein that is missing in DMD patients, regardless of their genetic mutation.”

    EXG-7001 leverages one of Elixirgen’s core technologies. The company has developed a platform for delivering mRNA-based therapies that it claims addresses the major delivery limitations of current methods. “The key features are that it is a lipid nanoparticle-free, localized mRNA therapeutics platform,” Ko explained to GEN. With this approach, “we’re avoiding some of the complications of gene therapies and delivering genes systemically by going local” and avoiding liver accumulation, which remains “a big issue” for mRNA therapeutics. 

    The system has two components. The first component, called RNA tether, is designed to ensure that the RNA stays in the tissue that is injected without migrating to the liver. The second component is the mRNA cargo itself, which the company calls Bobcat® mRNA. Though the lead indication for this technology is DMD, there are other diseases involving large genes that the company could target. 

    “We’re able to express the full length protein as mRNA as a single strand” and “it stays where you administer it, which is kind of unusual,” Ko said. Combining RNA tether and Bobcat makes it possible to express large genes and localize them to target tissues even without accumulation in the liver. Preclinical data has demonstrated its effectiveness in mice with no safety concerns associated with administration or treatment. “A full length dystrophin being given to kind of key muscles could potentially change quality of life,” particularly for the non-ambulatory population, Ko said. 

    Beyond EXG-7001, Elixigen has other candidates in its pipeline that are much closer to the clinic. Its lead candidate is currently in Phase I/II testing at Cincinnati Children’s Hospital Medical Center. This is an ex vivo cell therapy based on the company’s ZSCAN4 technology, which is designed to extend the telomeres of stem cells in “a controlled way” using a telomerase-independent mechanism. EXG-34217 is comprised of autologous CD34+ hematopoietic stem cells that have been treated ex vivo with EXG-001, a non-integrating, non-transmissible, temperature-sensitive Sendai virus vector encoding human ZSCAN4.

    The features of that technology were identified by the company’s CSO and his team while he worked at the National Institutes of Health’s National Institute on Aging. In 2024, the U.S. Food and Drug Administration granted Rare Pediatric Disease Designation to the treatment, dubbed EXG-34217, for the treatment of patients with dyskeratosis congenita and related telomere biology disorders. 

    “Telomeres obviously have a relationship with aging, and there are in fact genetic diseases associated with short telomeres and telomerase mutations,” CEO Ko told GEN at BIO. People with TBDs are “born with shorter telomeres typically, but also have a mutation in their telomerase so they are not necessarily maintaining them either.” The result is a type of premature aging, so conditions like bone marrow failure and cytopenia happen earlier in the life of the patient. In fact, “bone marrow failure is one of the largest issues” affecting both adults and young children, CEO Ko said. 

    One treatment option in these cases is allogeneic hematopoietic stem cell transplantation (HSCT), he continued. However, people with short telomeres have more fragile genomes that are less resistant to chemotherapy and radiotherapy and are at greater risk of cancer even after HSCT treatment. In an ideal scenario, it would be possible to postpone or avoid HSCT for these patients, and the company’s ZSCAN4-based therapy could make it possible to do that. 

    The treatment is currently being tested in adult and pediatric patients in Cincinnati. “We started in adults because this is first-in-human,” but the disease is also very severe in children, Ko said. “Our target ultimately is to make sure as many people with TBDs can get this if they need it.” Early clinical results published in 2025 in a paper in NEJM Evidence show durable telomere extension overall with no treatment-related safety concerns observed over a 24-month and 5-month period after infusion. The trial has been going on for some time, and “we have a lot of longer-term data now” and are “looking toward potential accelerated approval.”

    But targeting TBDs is just one indication. “Short telomeres manifest in many different ways,” Ko said. Other potential targets for the company’s technology are aging-related diseases, including things like idiopathic pulmonary fibrosis. 

    To date, Elixirgen has raised roughly $34 million from existing investors.

    The post Elixirgen Builds Rare Disease Pipeline Around Telomere Biology Disorders and DMD appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Peptistar Began Operation of the Asahi Kasei FO-MD System at Manufacturing Scale https://edusehat.com/en/peptistar-began-operation-of-the-asahi-kasei-fo-md-system-at-manufacturing-scale https://edusehat.com/en/peptistar-began-operation-of-the-asahi-kasei-fo-md-system-at-manufacturing-scale Thu, 02 Jul 2026 22:45:10 +0700 Edusehat Peptistar, Began, Operation, the, Asahi, Kasei, FO-MD, System, Manufacturing, Scale Japanese CDMO Peptistar reports that it has integrated Asahi Kasei’s forward osmosis–membrane distillation (FO–MD) system into its facility for trial production of active pharmaceutical ingredients (APIs).

    Asahi Kasei announced in 2018 the development of a system that dehydrates and concentrates liquids without the application of heat or pressure. This reduces the number of freeze-drying batches and the amount of time required for freeze-drying, thereby shortening API manufacturing time. Peptistar has begun operation of the system at manufacturing scale as part of its evaluation toward GMP production.

    FOMD-system-for-concentration-without-heating-or-pressurization-installed-at-Peptistars-peptide-and-oligonucleotide-API-manufacturing-facility [Asahi Kasei]
    FOMD system for concentration without heating or pressurization installed at Peptistar’s peptide and oligonucleotide API manufacturing facility. [Asahi Kasei]
    Recently, demand for APIs has shifted from traditional, high-volume small molecules to a broader need across biologics, peptides, oligonucleotides, viral vectors, and more, according to officials at both companies. API needs are becoming increasingly complex due to their high specificity and growing role in next-generation therapeutics.

    Some of the next-generation APIs such as peptides and oligonucleotides are heat sensitive. Their manufacturing processes have thus relied on the costly, time-consuming, and energy-intensive freeze-drying method, which can remove solvents without heating, to obtain APIs with high quality explains an Asahi spokesperson.

    Although the freeze-drying process can be shortened by concentrating the raw material solution to reduce the volume of liquid feed prior to the freeze-drying step, conventional concentration technologies such as vacuum distillation carry the risk of quality degradation due to heating, and the formation of precipitates caused by changes in solvent composition during the concentration step, adds the spokesperson.

    Overview-of-the-FOMD-system-for-concentration-without-heating-or-pressurization. [Asahi Kasei]
    Overview of the FOMD system for concentration without heating or pressurization. [Asahi Kasei]
    Asahi Kasei’s system for forward osmosis (FO) and membrane distillation (MD) addresses such manufacturing challenges by concentrating the raw material solution for pharmaceutical applications without applying heat or pressure, notes another Asahi official, explaining that FO utilizes an osmotic pressure difference across a membrane to remove water from liquids, achieving highly concentrated API solutions under mild conditions. MD leverages a vapor pressure difference across a membrane to remove volatile components such as acetonitrile, alcohol, or ammonia, at or below room temperature.

    Asahi Kasei says it looks forward to studying the prospects for future commercialization of the FO–MD system.

     

    The post Peptistar Began Operation of the Asahi Kasei FO-MD System at Manufacturing Scale appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     First 3D Structure of Malaria’s “Moving Junction” Solves Infection Mystery https://edusehat.com/en/first-3d-structure-of-malarias-moving-junction-solves-infection-mystery https://edusehat.com/en/first-3d-structure-of-malarias-moving-junction-solves-infection-mystery Thu, 02 Jul 2026 08:25:08 +0700 Edusehat First, Structure, Malaria’s, “Moving, Junction”, Solves, Infection, Mystery For nearly half a century, scientists have known that malaria parasites force their way into human red blood cells (RBCs) through a ring-shaped structure called the moving junction (MJ). What no one could work out was what it actually does. The structure assembles, does its job, and dissipates in the space of 60 seconds—gone before anyone can get a close look.

    A team at Columbia University has now finally caught the moving junction in the act. By freezing parasites at the onset of invasion and lifting the intact complex straight out of the cell, the researchers obtained the first high-resolution view of its three-dimensional structure. What they saw overturned a decades-old assumption about how the parasite gets in. Rather than a passive doorway, the moving junction turns out to be a molecular machine that actively remodels the host cell’s membrane to help the parasite force its way inside.

    The findings detail how the team obtained the structure and then used it as a blueprint to design a mini-protein, from scratch, that blocks invasion—a proof of concept for a new kind of antimalarial drug.

    “We’ve known for decades that this structure is essential for the parasite to get into a cell, but not how it actually works,” said Chi-Min Ho, PhD, an assistant professor in the Department of Microbiology and Immunology at Columbia University Vagelos College of Physicians and Surgeons and the study’s senior author. “Pulling it directly out of the parasite intact let us finally ask that question directly.”

    Ho is senior author of the team’s published paper in Cell, titled “Structural basis for host membrane binding and remodeling by invading malaria parasites.” In their paper, the team stated in summary, “This work represents a major step toward resolving the decades-long mystery surrounding the structure and function of the malarial MJ, underscoring the power of pursuing native structures and laying the foundation for structure-guided design of next-generation antimalarials.”

    Malaria still kills roughly 600,000 people a year, the overwhelming majority of them young children in sub-Saharan Africa, and the parasite is steadily becoming resistant to frontline drugs. “Malaria morbidity and mortality are directly linked to the invasion and replication of the malaria parasite Plasmodium falciparum in human red blood cells (RBCs),” the authors wrote. The malaria parasite life cycle involves two hosts, humans and Anopheles mosquitoes, and infecting human RBCs and hepatocytes, as well as mosquito salivary glands.

    The disease starts with a single event: a parasite breaking into a red blood cell. “Parasites establish infection by invading host cells in a rapid and precisely choreographed process …” the team continued. In an infected person, trillions of parasites are released and invade every 48 hours in synchronized waves. This rhythmic cycle of rupture and reinvasion drives the periodic fevers malaria is known for. “After gliding, reorientation, and initial attachment, parasite internalization is initiated by the formation of a ring-shaped ultrastructure called the moving junction (MJ), which anchors the parasite to the host cell,” the researchers explained.

    The same moving junction machinery is used across every species and every stage of the parasite’s life cycle, which has made it one of the most sought-after targets in malaria research. For antimalarial drug and vaccine development, block it, and you stop infection at its source.

    The moving junction has been a puzzle since 1978, when scientists first observed in electron microscopy images a mysterious thickening of the membrane where parasite meets cell. Researchers eventually identified the four parasite proteins—AMA1, RON2, RON4, and RON5—that assemble into the junction’s basic building block, and confirmed that all were essential for invasion. But what the structure actually did remained unknown, because it survives for a minute or so and refuses to reassemble in a test tube. “Efforts to address this critical gap in understanding have been thwarted by the short-lived (60–90s) nature of the complex, as well as by the difficulty of recapitulating it in heterologous systems for detailed biochemical and structural study,” the researchers stated.

    The Columbia team got around this by stopping invasion mid-stride. Using a compound that halts the parasite’s internal motor without preventing the junction from forming, they stalled parasites partway into red blood cells, then extracted the fully assembled AMA1-RON complex—the building block from which the whole junction is constructed—and imaged it with cryo-electron microscopy (cryo-EM), a technique where molecules are flash-frozen and imaged with an electron beam at extremely high magnifications to reveal their shape in atomic detail. The result was a sharp, three-dimensional view of that building block. The researchers noted that it was quite strikingly shaped like a sailboat, with the AMA1 protein forming a “sail” above the cell surface and the three RON proteins forming a broad “hull” pressed against the membrane below.

    The biggest surprise was in the hull, where the team found clues that finally hinted at the moving junction’s role in invasion. The face of the structure pressed against the host membrane is blanketed with positively charged anchors, and the surface is studded with short helices that drive deep into the membrane like wedges. “These short helices insert asymmetrically into one leaflet of the membrane, displacing lipid headgroups and applying lateral pressure to generate local membrane deformations.”

    Both features are widely recognized hallmarks of a well-known family of cellular machines that bend and reshape membranes. Their structural findings, they noted in their report, reveal “a highly unusual molecular staple that exhibits the hallmarks of a powerful membrane-remodelling machine.”

    To test whether the structure could indeed deform a membrane, the researchers synthesized the parasite’s wedge-like helices and added them to artificial membrane bubbles. The membranes thinned and punctured. Meanwhile, weakened versions of the helices left the bubbles intact. The team concluded that the moving junction appears to pull the host membrane into shape, likely working in concert with the parasite’s motor to lever the parasite inside.

    “It had been pictured as a kind of series of staples or spot-welds, making up a passive ring the parasite hauls itself through,” said Meseret Haile, the study’s first author and a PhD candidate in Ho’s lab. “What we see instead is a machine built to reshape the host cell’s own membrane. That changes how we think about the whole event.” In their paper, the team added, “Our work reveals that, although visually suggestive of canonical tight junctions, the MJ differs fundamentally in function, serving as a dynamic portal that orchestrates parasite internalization, rather than a static adhesion molecule.”

    Beyond finally revealing how the moving junction allows the parasite to invade, the structure also gave the team a precise map of where and how AMA1 grips its partner protein, the contact that holds the entire junction together. Using a machine learning-powered protein-design tool together with their structural information, the researchers designed a mini-protein to break that grip. Their best candidate blocked parasites from invading red blood cells in a dose-dependent way and left already-infected cells unaffected, confirming that it works specifically by stopping entry rather than through general toxicity.

    The designed mini-protein is a first proof of concept, not a drug, and will need considerable refinement before it could be tested in people. But it demonstrates an exciting new strategy: using near-native structures to design invasion-blocking mini-proteins against a target that has long frustrated conventional approaches. The same structure also clarifies how several leading anti-malaria antibodies work, information that could feed back into vaccine design. “Our successful proof of principle demonstrates the potential power of context-driven binder design for challenging systems, offering a previously unexplored avenue for therapeutic intervention,” they wrote. “In addition to their therapeutic potential, these binders may also serve as powerful tools for probing the functional relevance of specific protein interactions.”

    Daphne Kaxiras, an MD-PhD student in Ho’s lab who led the inhibitor design, said, “Once we could see the target in its real setting, designing something to block it became a tractable problem. That’s the part we’re most eager to build on.”

    The team’s approach, imaging fragile complexes captured directly from the organism and using them to guide design, may apply to many other parasites and pathogens that are notoriously difficult to study.

    The post First 3D Structure of Malaria’s “Moving Junction” Solves Infection Mystery appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Biological Order Emerges from Tissue Boundaries, Drives Embryo Development https://edusehat.com/en/biological-order-emerges-from-tissue-boundaries-drives-embryo-development https://edusehat.com/en/biological-order-emerges-from-tissue-boundaries-drives-embryo-development Thu, 02 Jul 2026 04:50:09 +0700 Edusehat Biological, Order, Emerges, from, Tissue, Boundaries, Drives, Embryo, Development In a new study in Nature Materials titled, “Boundary geometry controls a topological defect transition that determines lumen nucleation in embryonic development,” researchers from European Molecular Biology Laboratory (EMBL) describe how interactions between tissue geometry impact development. 

    In an early-stage mouse embryo, cells of the epiblast are polarized and give rise to all major tissues. The team investigated the fundamental principles governing the behavior of polarized cells that are present in bulk and the impact of physical constraints at tissue borders. By focusing on how cellular orientations influence each other and their environment, the researchers built a minimal model that predicts how organization changes when interactions are altered.

    “For me, as a physicist, I may know why something works, but it’s still kind of magic to see that it’s all true in messy biological systems,” said Pamela Guruciaga, PhD, postdoctoral researcher at EMBL and co-first author of the study. “It was also super interesting coming from a pure physics perspective to come up with a common language to work with biologists.” 

    In the cup-shaped epiblast, results showed different boundaries led to varying orientations for epiblast cells. When the boundary was lined with the extracellular matrix, the cells oriented perpendicularly. In contrast, when the epiblast was in direct contact with a neighboring tissue without a matrix, the cells aligned parallel to the boundary. The researchers found that the combination of these two orientations result in the appearance of structures, known as “topological defects.” 

    “These are points in space where it is undefined in which direction an object should point,” explained Guruciaga. “For example, if a set of arrows is arranged in a starburst pattern, the center is a point where all directions are equivalent. These points are super relevant because they are very robust; you cannot easily destroy them.” 

    To directly test whether the boundary shape controls the number of defects, the authors  altered the geometry of the epiblast. Perturbing embryo shape induced the formation of additional lumina at the predicted positions.  

    “What I find most exciting is that these results identify a very general physical principle,” said Anna Erzberger, PhD, group leader at EMBL and co-corresponding author of the study. “We show that geometry alone can determine orientation patterns in three dimensions, independent of the microscopic details of the system. That means shape itself can act as a robust control parameter—not just in embryos, but across a wide range of biological and physical systems.” 

    The post Biological Order Emerges from Tissue Boundaries, Drives Embryo Development appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     In Huntington’s Mouse, Optogenetic Activation of VIP Neurons Restores Brain Function https://edusehat.com/en/in-huntingtons-mouse-optogenetic-activation-of-vip-neurons-restores-brain-function https://edusehat.com/en/in-huntingtons-mouse-optogenetic-activation-of-vip-neurons-restores-brain-function Thu, 02 Jul 2026 04:50:08 +0700 Edusehat Huntington’s, Mouse, Optogenetic, Activation, VIP, Neurons, Restores, Brain, Function Huntington’s disease is a devastating brain disorder in which damage to nerve cells leads to progressively worsening cognitive and movement abilities. While the genetic mutation responsible for the condition is well known, the details of how the disease disrupts brain circuits have not been clearly understood. Now, researchers have identified and tracked neurons involved in Huntington’s disease progression and used optogenetics to selectively activate these neurons and improve the debilitating deficits of the condition.

    The study is published in Nature in the paper, “Restoring cortical disinhibition improves Huntington’s disease phenotypes.”

    “This work shows that correcting specific imbalances in brain circuits can restore function, even in a complex neurodegenerative condition, and highlights the potential of targeting defined cell types to promote recovery,” said Takaki Komiyama, PhD, professor in the UC San Diego Departments of Neurobiology (School of Biological Sciences) and Neurosciences (School of Medicine).

    Huntington’s disease is caused by a trinucleotide repeat mutation in the Huntingtin (HTT) gene. While the mutation is well known, the neural networks connected with the disease progression have been more elusive.

    This work aimed to map the neural circuits that expose the networks involved at the onset and spread of the disease’s debilitating symptoms. In transgenic mice carrying the same mutation as human patients, the researchers evaluated how different types of brain cells in the motor cortex are affected in Huntington’s disease. Advanced imaging techniques allowed the researchers to track the activity of these cortical neurons as the disorder progressed.

    The researchers found that the disease disrupts the balance of activity across different cell types, including cortical inhibitory neurons.

    “Cortical inhibitory cells have received little attention in Huntington’s disease, as for a long time they were considered to be spared from neurodegeneration,” said Irina Dudanova, PhD, previously based at the Max Planck Institute for Biological Intelligence, now at the University of Würzburg in Germany. “Surprisingly, we detected profound changes in their activity, with some cell types being overactive and some nearly silent.”

    Huntington's
    The activity of neuron types in the brain is imbalanced in mice with Huntington’s disease. The image depicts an example field-of-view from inhibitory (left) VIP (vasoactive intestinal peptide) neurons and excitatory (right) neurons recorded during behavior. Activity traces from a selected neuron for each type are shown above the images. [Sonja Blumenstock, Komiyama Lab, UC San Diego]

    In particular, a class of inhibitory neurons known as vasoactive intestinal peptide (VIP) neurons, exhibited significantly reduced activity. VIP neuron activity is essential for normal learning, as these cells enable the brain to adapt and refine brain circuits during learning.

    Reduced VIP neuron activity, the researchers reasoned, could be impairing the brain’s ability to function and learn properly. They sought to activate these cells to re-engage brain states that support learning. They tested this idea using optogenetics to stimulate VIP neurons.

    “By activating the VIP inhibitory cell type, we gradually restored more normal activity patterns, and, very importantly, we also saw an improvement in the ability of the mouse to learn a motor task,” said Sonja Blumenstock, PhD, assistant project scientist at UC San Diego.

    The results confirm VIP neurons as a key point of vulnerability in Huntington’s disease as well as a promising target for therapy. As to how this process works, the results suggest that modulating VIP neurons opens a “gate” that enables learning-related brain plasticity.

    “This intervention restored more normal patterns of activity in the brain and improved movement in affected mice,” said Komiyama. “Importantly, the improvements persisted for days after stimulation ended, suggesting that the treatment triggered lasting beneficial changes in brain circuits rather than only temporary effects.”

    The study provides important indications of where research could focus to normalize human brain function and facilitate brain recovery. Komiyama envisions a future scenario in which scientists could non-invasively activate the brain from outside the skull using novel approaches.

    “Our study shows that despite the genetic defect, a precise intervention into the brain circuitry can lead to significant improvements in motor symptoms,” said Dudanova. “If we know which cells to target, we can retune the brain’s abnormal activity patterns. This gives hope for future therapies.”

    The research also shows that corrections to specific brain circuit imbalances can restore function in a highly complex neurodegenerative condition, with similar potential in other disorders.

    “We have come up with a way to allow the diseased brain to learn better,” said Komiyama. “The approach can improve behavior in diseased mice, and our hope is that a related approach will help people with impairment in their learning abilities.”

    The post In Huntington’s Mouse, Optogenetic Activation of VIP Neurons Restores Brain Function appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Trait Combining Key to More Effective Vector Production Hosts https://edusehat.com/en/trait-combining-key-to-more-effective-vector-production-hosts https://edusehat.com/en/trait-combining-key-to-more-effective-vector-production-hosts Thu, 02 Jul 2026 01:15:12 +0700 Edusehat Trait, Combining, Key, More, Effective, Vector, Production, Hosts HEK293 cells may be the most common host used in viral vector production, but they are far from ideal, says the author of a new study, who argues that gene therapy firms will need more effective alternatives to support commercial growth.

    The study, by a team at University College Dublin and services firm APC, examined the manufacturing systems used to make the recombinant adeno-associated viruses (rAAVs) on which many gene therapies rely.

    And the key finding is that not one of the eight commercial cell lines used to date—including the most widely-used line, HEK293—is ideal.

    Lead author, James Conheady, from APC, tells GEN, “Current rAAV production methods using existing cell lines struggle to meet clinical demands, contributing to the expensive price-tag associated with rAAV-based gene therapies.

    “Novel cell lines may be able to produce rAAVs at higher yields and/or with improved quality, which ultimately could help make these therapies more accessible to the people who need them.”

    Shortcomings

    To date, eight different host cell systems have been used to produce rAAVs, with each having strengths and weaknesses.

    For example, some cell lines generate rAAV capsids that do not contain the desired genetic material. These empty vectors are a problem because they generate an immune response without providing a therapeutic effect.

    Other cell lines struggle to make enough capsids. For example, the recommended dose for systemically delivered gene therapies is upwards of 1 × 1014 vg/kg of a patient’s bodyweight. The yield per production run for HEK293 cells is only around 1010.

    Cost is another issue.

    According to Conheady and co-authors, the GMP-grade plasmids and transfection reagents used to modify cell lines such that the vectors they produce contain the genes of interest account for a significant proportion of the price of the resulting therapies.

    Alternative systems

    Given these shortcomings, it is no surprise that the search for more effective alternative hosts is already underway.

    Conheady says, “At the end of the day, rAAV manufacturers are all looking for the same things from their upstream process—high titers, improved full/empty ratios, and transduction rates.”

    Current cell line development efforts are focused on combining desirable traits, Conheady adds, with characteristics such as resistance to apoptosis, diminished antiviral immune response, and secretion profiles being among the most sought after.

    “Many of the traits identified in this review are aligned with modifications that have been shown to be beneficial in the context of rAAV production in HEK293 cells. For example, secretion of vector particles from the cell into the production medium can greatly simplify downstream operations and can be influenced by knocking out genes involved in endosomal trafficking.

    “The ideal cell line should also be resistant to transfection and virus-induced apoptosis, to produce significant vector quantities. Knockout of the pro-apoptotic BAX and BAK1 genes has been shown to improve vector yields,” he says.

    Whether industry will ever see these efforts pay off and agree on the “ideal” cell line remains to be seen, according to Conheady.

    “Manufacturers will require significant grounds to agree on a standardized approach, a novel cell line may need to vastly outperform all others in relation to yield and quality characteristics—as the saying goes, ‘you stick with what you have until you have better’.”

    The post Trait Combining Key to More Effective Vector Production Hosts appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Blueprint to Fill the Manufacturing Talent Gap https://edusehat.com/en/blueprint-to-fill-the-manufacturing-talent-gap https://edusehat.com/en/blueprint-to-fill-the-manufacturing-talent-gap Thu, 02 Jul 2026 01:15:11 +0700 Edusehat Blueprint, Fill, the, Manufacturing, Talent, Gap As biopharmaceutical companies expand facilities and reshore some manufacturing operations, the industry faces a shortfall of trained workers for its manufacturing facilities. While the Bureau of Labor Statistics recently predicted some 19,000 jobs would be created, PhRMA last year predicted the creation of 100,000 new jobs. Both predictions leave a gap between those jobs and the trained workforce.

    To close the gap, Manus, a next-gen industrial biotechnology company, and BioMADE have developed an apprenticeship program that can become a blueprint for other companies to develop their own training. “The program can be scaled so development for other [companies] can be faster, down the road,” says Maren Wehrs, PhD, program manager at BioMADE.

    “We are trying to build a fairly comprehensive training program that spans fermentation operations as well as downstream purification,” Christine Santos, PhD, CTO, Manus, tells GEN.

    Focus: Hands-on learning

    “It’s focused on hands-on experiential learning,” Santos continues, “with an extensive curriculum that will include deep dives on the practical aspects of running the equipment, such as so sterilization, safety, contamination control, process monitoring, and analytics. It will also delve into some of the technical aspects, like scale-up principles, as well as decision-making, problem-solving, teamwork, and communications.”

    The work occurs at a Manus pilot facility in Augusta, GA. The first cohort starts in July and completes at year’s end, with another cohort beginning in January. After 18 months, “We hope to have a blueprint for an apprenticeship program that could be deployed at any other facility,” Santos says, including new BioMADE pilot facilities or those of other companies.

    “We would offer access to the curriculum and the blueprint for [others] to deploy. We’ve spent the past few months formalizing the curriculum,” Santos says. It was developed with input from the University of Georgia, but apprentices needn’t be enrolled in a university program to participate.

    Manus’ interest in apprenticeships stems from its 2018 acquisition of a decommissioned NutraSweet manufacturing facility in Augusta for its cell factories and bioprocesses.

    “We had the task of recommissioning the facility and rebuilding the workforce to operate it,” Santos recounts. “We were able to rehire some of the NutraSweet employees [and regain their institutional knowledge], but to build out further, we had a huge challenge finding workers who were trained for biomanufacturing operations. We had to invest in a lot of hands-on training.”

    This program is one of a few offered directly by a biomanufacturer. More commonly, companies participate in workforce training consortia to develop potential manufacturing workers.

    The post Blueprint to Fill the Manufacturing Talent Gap appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Standardizing Personalized CRISPR Gene-Editing Therapies https://edusehat.com/en/standardizing-personalized-crispr-gene-editing-therapies https://edusehat.com/en/standardizing-personalized-crispr-gene-editing-therapies Thu, 02 Jul 2026 01:15:09 +0700 Edusehat Standardizing, Personalized, CRISPR, Gene-Editing, Therapies The revolutionary success of Baby KJ, the first patient to be treated with a personalized CRISPR gene-editing therapy, is spurring the industry to develop platforms for standardizing the manufacturing of future individualized therapies.

    That’s the topic of a talk by Kok-Seong Lim, PhD, a pharmaceutical leader in CMC development, at the Bioprocessing Summit in Boston.

    “Baby KJ was the first proof that individualized gene editing therapy was doable, and, at the same time, in the background, there are manufacturing platforms now being set up that maybe we’re not hearing so much about in the media,” he says.

    According to Lim, manufacturers seeking to develop standardized platforms for personalized CRISPR gene-editing therapies using liquid nanoparticles (LNP), the same technology used for Baby KJ, will need to “lock in” their lipid formulation they’re going to use for future manufacturing, which may vary depending on the target organ and therapeutic indication.

    After selecting their raw materials, they will also need to lock in their manufacturing process parameters, such as the microfluidic mixing conditions and lipid compositions. Likewise, he says, although the target gene may need to be customized for different patients, certain core components, such as the mRNA encoding the CRISPR-Cas enzyme, could remain unchanged across multiple patients.

    This type of standardization may help establish a more scalable and reproducible manufacturing platform for personalized gene-editing therapies, he believes.

    Going forward, Lim says, eventually companies may need to look at standardizing their regulatory CMC data package for regulatory filing, such as determining the appropriate extent of their impurity profiling and the overall scope of stability studies.

    “Impurity profiling may not need to be as extensive for individualized and personalized treatments because they’re manufactured for a single patient only and the stability requirements may only need to support the timeframe needed for the patient’s treatment,” he says.

    Lim adds that the Innovative Genomics Institute (IGI), Penn Medicine, and their collaborators, who treated Baby KJ, are currently working toward clinical trials to treat the next group of patients, but details of the specific LNP configurations for each future patient have not been disclosed.

    As well as talking about LNPs, Lim will also discuss AAV technology for personalized CRISPR gene-editing therapies. The technology, he explains, is less popular within the industry than LNPs, due to concerns about potential toxicity, side effects, and manufacturing complexity, but it still merits consideration as a platform technology when it delivers patient benefits.

    The post Standardizing Personalized CRISPR Gene-Editing Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Digitize or Fall Behind https://edusehat.com/en/digitize-or-fall-behind https://edusehat.com/en/digitize-or-fall-behind Thu, 02 Jul 2026 01:15:08 +0700 Edusehat Digitize, Fall, Behind Bioprocessing companies risk slowing scientific progress unless they embrace digital-data capture and greater collaboration, according to Alexander Seyf, CEO of Autolomous, a company developing digital manufacturing solutions for cell and gene therapies.

    Speaking about the industry’s biggest challenges, Seyf describes poor data management as the “elephant in the room,” arguing that too much crucial information remains trapped in paper records, spreadsheets, and isolated systems.

    “Everybody wants to have AI,” Seyf says. “But where do you have your data? If it’s in binders, there’s not much you can do.”

    According to Seyf, the path toward more efficient manufacturing, stronger clinical outcomes, and meaningful AI applications begins with digitizing information from the earliest stages of research. He believes many organizations make the mistake of waiting until their science is mature before investing in digital infrastructure. “The sooner you start, the better it is,” he says. “Pen and paper do not prevail, and pen and paper do not transfer.”

    Seyf argues that the consequences extend far beyond operational inefficiencies. When data remain inaccessible or fragmented, researchers lose opportunities to learn from past experiments, identify patterns, and accelerate scientific discovery. He stresses that the industry must become more willing to share non-commercially sensitive knowledge, particularly in areas such as rare diseases and advanced therapies, where patient populations are limited. “We are all here to serve patients,” he says. “Protect your intellectual property, but also share the learnings.”

    One of his strongest criticisms is directed at the scientific community’s tendency to focus almost exclusively on successful outcomes. Seyf believes failed studies and unsuccessful trials often contain lessons that could prevent others from repeating the same mistakes. “A lot of publications want to publicize only the good news,” he says. “That’s fundamentally wrong. We need to learn from failures.”

    To illustrate his point, Seyf compares the biotechnology sector with the aviation industry. Modern airlines routinely share information about incidents and technical problems to prevent future accidents, creating a culture of collective learning and safety. “If something goes wrong, everybody in the world knows about it and knows how it was managed,” he says. “We are also dealing with people’s lives. The only way for us to improve is to share.”

    Seyf also highlights the growing role of AI in healthcare. Although consumer AI systems have benefited from vast amounts of publicly available information, healthcare still operates with a relatively small pool of accessible data, he says. Expanding that foundation, he argues, could unlock major advances in diagnosis, drug development, and personalized medicine. “Imagine what we could do,” he says. “The progression of science is unlimited.”

    For commercial bioprocessors, his recommendation is straightforward: digitize from day one. Capturing research, development, manufacturing, and clinical data in digital formats not only improves collaboration but also preserves institutional knowledge when employees move on. “Every time a scientist leaves, the knowledge goes with them,” Seyf says. “But when it is digital, the knowledge stays with the company.”

    As cell and gene therapies continue to evolve, Seyf believes the industry faces a choice. It can continue operating in silos, or it can embrace transparency, digitalization, and collaboration to speed innovation and deliver better outcomes for patients. “The reason humanity has progressed,” he says, “is because we shared.”

    The post Digitize or Fall Behind appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Roundtables: Longevity’s Next Frontier: “Reprogramming” Your Body https://edusehat.com/en/roundtables-longevitys-next-frontier-reprogramming-your-body https://edusehat.com/en/roundtables-longevitys-next-frontier-reprogramming-your-body Wed, 01 Jul 2026 07:25:02 +0700 Edusehat Roundtables:, Longevity’s, Next, Frontier:, “Reprogramming”, Your, Body Listen to the session or watch below

    Billions of dollars are flooding into efforts to reverse aging as scientists explore ways to return cells to a younger state. But how far off are these experimental treatments? Will they really work? Watch a conversation exploring longevity’s new focus.

    Speakers: Mary Beth Griggs, science editor and Jessica Hamzelou, senior biotechnology reporter

    Recorded on June 30, 2026

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    ]]>     Cytiva Completes Doubling of Utah Site’s Liquid Media Production Capacity https://edusehat.com/en/cytiva-completes-doubling-of-utah-sites-liquid-media-production-capacity https://edusehat.com/en/cytiva-completes-doubling-of-utah-sites-liquid-media-production-capacity Wed, 01 Jul 2026 07:20:08 +0700 Edusehat Cytiva, Completes, Doubling, Utah, Site’s, Liquid, Media, Production, Capacity Cytiva has completed an expansion of its Logan, UT, facility that effectively doubles its liquid media production capacity, a project designed to support supply chain continuity for customers relying on the company for their cell culture needs.

    The company has completed its animal-derived component-free (ADCF) liquid media expansion facility (A1X), Pierre-Alain Ruffieux, Cytiva group executive, bioprocess, told GEN in an interview conducted from the company’s booth during the Biotechnology Innovation Organization (BIO) International Convention recently held in San Diego. He said the completion was celebrated with a ceremony on the site.

    Cytiva detailed the expansion project in a May 12 post on its website: The ADCF liquid media expansion facility (A1X) has larger mixing tanks than the existing facility, supporting batch sizes from 700 L up to 13,000 L—compared with batch sizes of 100 L to 10,000 L supported by Cytiva’s existing facility.

    Also, the A1X facility uses mixing tanks and liquid media transfer lines comprised of AL6XN and 316 L stainless steel. This differs from the existing facility equipment, which is comprised solely of 316 L stainless steel. AL6XN is a low-carbon, high-purity stainless-steel alloy that is more resistant to wear and corrosion than 316 L, representing an upgrade to the product contact layer versus the existing facility equipment.

    The expanded site’s added liquid capacity comes from the addition of three manifold fill lines, three filling manifolds, six mixing tanks, six formulation booths, and a utility building to support large volume liquid media production. Housed in the utility building are a 45,000 L tank and process water system, a 55,000 L tank and water for injection system, a clean steam generator, and additional supporting utilities.

    “In addition to the added capacity, Cytiva has updated several aspects of the manufacturing floor layout and equipment, improvements designed to shorten production cycle time, improve safety, and minimize product risk,” the company explained. “The updates also establish closed systems for cleaning and a controlled environment for the transport and handling of raw materials and finished goods.”

    Previously, Cytiva completed expanding its dry powder and liquid media manufacturing capacity for large-volume customers and added high-speed bottle filling for smaller-volume users. The company also opened an expanded staging area for finished goods, as well as a new centralized 10,000-square-foot quality control lab to support increased manufacturing.

    AI’s “two major impacts”

    Pierre-Alain Ruffieux, Cytiva group executive, bioprocess

    During a wide-ranging interview, Ruffieux discussed Cytiva’s approach to AI and several recent Cytiva announcements.

    “We see two major impacts from AI on what we are doing,” Ruffieux explained. “The first one, and I always like to start with the customers because it’s really our focus: We see our customers accelerating and increasing the number of targets they are doing. AI is helping them to have more targets and in a faster time,” Ruffieux said. “It’s putting pressure on the CMC folks, and I think it’s where we play: They ask us to provide innovative solutions to go faster.”

    Cytiva’s focus on AI is two-fold, he continued.

    “One, we are developing intelligent equipment which is using AI to be easier for customers to use and which are more functional; that is one aspect. It’s also delivering more experience in a shorter time frame,” Ruffieux said. “It’s a kind of next level of DoE [design of experiments], but it’s also delivering a productivity aspect because the goal is to have equipment which requires either fewer people or fewer people with less specific knowledge of the equipment.”

    Like a growing number of companies in and outside biopharma, Ruffieux said, Cytiva has fully embraced AI “to make our product better, to make the customer experience better, but also to improve our internal processes.”

    “Faster and better”

    “We see AI helping us to develop software, writing new software to go faster and better. AI is very powerful for reviewing documents and doing things,” he explained. “It’s amazing what we can do both in writing code, but also perhaps as importantly, as we validate the code and we test everything, the use of AI is allowing our people to work in a much more comprehensive way, in a much faster way.”

    AI also adds a layer, he said, to the continuous improvement ethos that Cytiva and other Danaher-owned companies practice through the Danaher Business System (DBS). Since the mid-1980s, Danaher has carried out an ongoing company-wide Kaizen or continuous improvement effort based on lean manufacturing and anchored on DBS, a common culture and operating system focused on people, plans, processes, and performance.

    “AI is an additional pillar to this system, really helping the company to be more efficient and to drive business,” Ruffieux said.

    Cytiva’s customers, he continued, have not specifically asked about AI. So what are customers telling the company that they want?

    “What customers want is Cytiva delivering solutions which help them to innovate, produce drugs, and accelerate these processes. And AI is one of the attributes, but they don’t have a specific task on AI,” Ruffieux replied. “In discussing with senior customers, people are interested in the outcome, not in the product itself. So it’s not AI for AI, it’s AI for a business outcome. And in life science, the business outcome is quality. It’s reliability. It’s speed. It’s customers asking, can we help them to be better?”

    AMT designation

    Last month, Cytiva hailed the FDA’s granting its Advanced Manufacturing Technology (AMT) designation to the company for its Elevecta™ transient cell line for adeno-associated virus (AAV) manufacturing, one of the first gene therapy manufacturing technologies to receive the designation. Customers using the Elevecta transient cell line will benefit, according to Cytiva, from a clear, predictable regulatory and quality framework for gene therapy development.

    Through its AMT designation, the FDA recognizes drug manufacturing technologies that it deems to have elevated the reliability, quality, and robustness of advanced therapeutics manufacturing. By enabling a streamlined Chemistry, Manufacturing, and Controls (CMC) review and frequent communication with the FDA, designees count on the AMT designation to help accelerate their manufacturing-related development timelines and create a meaningful advantage through faster time to market.

    “This recognition by the FDA is giving confidence and trust for our customers: If they use this cell line to produce AAV, they know that the agency has seen the technical advantage and it’s confidence on the regulatory pathway,” Ruffieux said. “This recognition by that regulatory body is giving trust to the work of the company in helping customers develop drugs, which is really where we position ourselves as true partners.”

    Elevecta is designed to significantly reduce the formation and encapsidation of host cell DNA (hcDNA).

    “What is beautiful with that is, we get a reduction of 99% of the host cell DNA. You don’t have to worry any more about the host cell DNA which is coming with your product. Again, that is a huge advantage for the customer using that,” Ruffieux said. “This is the kind of innovation we are really proud to bring to our customers.”

    Operating from hubs in Marlborough, MA, Amersham, U.K., Uppsala, Sweden, and Shanghai, Cytiva is a unit of Danaher that was re-launched in 2020 after Danaher spent $21.4 billion for the former biopharma business of GE Healthcare Life Sciences. Danaher oversees a global family of more than 20 operating companies focused on biotech and life sciences, as well as diagnostics, water quality, and product identification.

    Bringing “the entire workflow”

    Earlier this month, the company said that eight of its 2,000 L single-use Xcellerex bioreactors were among equipment contained in the new GMP-2 manufacturing facility inaugurated in Wuhan, China, by Chime Biologics, a decade-long customer that has used equipment made by Cytiva and its predecessor company.

    “I want to put that in a larger context: At Cytiva, we really bring to the customers the entire workflow, which is really exciting for small to mid-sized customers. Coming to us, they really get a full facility that is working, really, from A−Z,” Ruffieux said. “It’s starting from an expansion of the cell line, to freezing the drug substance. It’s about a fully integrated solution that helps the customer to have that. And we have multiple facilities like that, that we are building every year for customers across the world.”

    “We make significant investments to be able to supply our customers with what they need into different regions, in-region-for-region,” Ruffieux said.

    In-region-for-region refers to Cytiva’s ongoing effort to satisfy customer demand for manufacturing tools and services usable within their regions of the world.

    “This is really helping us and the customer to secure supply independent of any disruption,” he added. “Since COVID-19, we have seen multiple disruptions worldwide. And really, our original presence is giving confidence to customers that they will get what they need, independent of whatever crisis is happening across the world.”

    Worldwide, the United States and European Union have championed “reshoring” efforts by drug developers and tools/technology providers across biopharma to manufacture more of their products within their regions rather than in China or elsewhere in Asia.

    “When there is investment, it’s definitely always a tailwind,” Ruffieux said. “We welcome investment, and we are happy to support all customers to put up new facilities, and for the opportunity these facilities offer to position our equipment.”

    The post Cytiva Completes Doubling of Utah Site’s Liquid Media Production Capacity appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     New Agentic Capabilities for Tasks Across the Complete Research Workflow https://edusehat.com/en/new-agentic-capabilities-for-tasks-across-the-complete-research-workflow https://edusehat.com/en/new-agentic-capabilities-for-tasks-across-the-complete-research-workflow Wed, 01 Jul 2026 03:45:09 +0700 Edusehat New, Agentic, Capabilities, for, Tasks, Across, the, Complete, Research, Workflow Officials at Elsevier say the company is expanding LeapSpace, a research-grade AI workspace, with new agentic capabilities that help “researchers carry out an even greater range of tasks within their complex workflow to drive better outcomes with confidence.”

    Designed specifically for the end-to-end research workflow, LeapSpace was created to accelerate discovery, help researchers calibrate the strength of the evidence, and support critical thinking. LeapSpace draws on 20+ million full-text peer-reviewed articles and books from Elsevier and over 1,000 new content licensing partners, including Sage Publishing, Emerald Publishing, IOP Publishing, and NEJM Group. as well as 100+ million scientific records from 7,000+ publishers on Scopus.

    Results are grounded in peer-reviewed literature, citations are traceable to sources, Trust Cards help researchers calibrate the strength of evidence, and the researcher remains in control, with every recommended change requiring approval, notes an Elsevier spokesperson.

    General-purpose AI tools can generate text, summarize articles and automate some tasks. But researchers require something more demanding: the latest trusted peer-reviewed content, verifiable citations, transparent reasoning, research integrity safeguards, and enterprise-grade security and privacy, according to Stuart Whayman, president, corporate markets, Elsevier, adding that this is what LeapSpace is built for.

    Built with research-grade AI, LeapSpace is already delivering results for thousands of researchers around the world: 97% report time savings, with more than half saving over 50% of their research time, points out Whayman, LeapSpace is now extending support to writing—the task researchers most want AI to help with: more than half find writing clearly and concisely to convey complex ideas a challenge, rising to 60% among students and early-career researchers.

     

    The post New Agentic Capabilities for Tasks Across the Complete Research Workflow appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     iPSC-Derived Retinal Endothelial Cells Offer Platform for Studying Diseases https://edusehat.com/en/ipsc-derived-retinal-endothelial-cells-offer-platform-for-studying-diseases https://edusehat.com/en/ipsc-derived-retinal-endothelial-cells-offer-platform-for-studying-diseases Wed, 01 Jul 2026 03:45:08 +0700 Edusehat iPSC-Derived, Retinal, Endothelial, Cells, Offer, Platform, for, Studying, Diseases Biomedical engineers at Duke University have for the first time used induced pluripotent stem cells (iPSCs) to grow specialized blood vessel cells critical to retinal health.

    When injected into mouse models of retinal disease, these “retinal endothelial cells” (iRECs) integrated into the damaged tissue to regenerate blood vessels and restore retinal function. The team also demonstrated these cells’ ability to form functional retinal vascular tissue in a lab-grown environment, providing a pathway to model and research various eye diseases.

    The results point toward the potential of using these retinal cells and models to develop new methods of impactful vision loss treatments and eye disorder research. “Retinal vascular diseases affect millions of people in the U.S., but our understanding remains limited, hindering our ability to discover and develop new therapeutics,” said Sharon Gerecht, PhD, the Paul M. Gross Distinguished professor and chair of Biomedical Engineering at Duke. “Using human stem cells, we generated the cells found in retinal blood vessels, paving the way for new therapeutic approaches.”

    Gerecht is senior and corresponding author of the researchers’ published paper in Nature Biomedical Engineering, titled “Derivation of functional retinal endothelial cells from human pluripotent stem cells for therapeutics and modeling.” In their report the authors suggested that their iREC differentiation strategy will “… advance cell therapy and disease modeling, accelerating the discovery of treatments for retinal microvascular diseases.”

    The old saying that the eyes are windows into the soul is more accurate than one might think. Neurons from the retina—the back part of the eye that detects light—extend directly to the brain, technically making the eyes part of the central nervous system.

    Also like the brain, the retina has a blood barrier that strictly controls what gets in and out including oxygen, nutrients, water and pharmaceuticals. While this barrier keeps the retina healthy and relatively protected from disease-causing agents, it also makes treating the retina difficult. “Retinal tissue has the highest energy and oxygen usage in the body due to the retina’s intense and continuous neuronal activity,” the authors further explained. “This demand leads to a crucial reliance on the inner blood–retina barrier (iBRB) to maintain ocular homeostasis.”

    The barrier is formed by blood vessel tissue comprising a tight network of retinal endothelial cells, which form the inner layer of blood vessels, in concert with other specialized cells called pericytes and astrocytes. “Retinal endothelial cells (RECs) in the iBRB are continuous endothelial cells (ECs) that form tight junctions to regulate the diffusion of small molecules, such as ions and water, across their cell–cell interface,” the investigators continued. The specificity of these cells and the fact that they do not form in other areas of the body make the complex tissue difficult to heal or to grow from scratch.

    This image depicts both healthy (right) and deteriorated (left) human retinal endothelial cells, which are essential for maintaining eye sight. The deterioration is caused by low oxygen and high glucose levels, mimicking conditions found in diabetic retinopathy, the leading cause of vision loss in working-age people in the United States. [Duke University]
    This image depicts both healthy (right) and deteriorated (left) human retinal endothelial cells, which are essential for maintaining eye sight. The deterioration is caused by low oxygen and high glucose levels, mimicking conditions found in diabetic retinopathy, the leading cause of vision loss in working-age people in the United States. [Duke University]
    “When this specialized blood vessel tissue begins to break down, it can cause a lot of different diseases that lead to vision loss,” said Parker Esswein, a PhD student working in the Gerecht laboratory and co-first author of the paper. “While there are sources of retinal endothelial cells, being able to grow a continuous supply from scratch could offer many advantages for those working in the field.”

    These retinal endothelial cells are currently collected and grown from real patients, making them relatively expensive with a limited supply. “A renewable source of human iBRB endothelium is thus vital for advancing eye research and treatment development,” the team noted in their paper.

    To expand access, reduce cost and control variability, the Gerecht lab wanted to see if they could grow them from iPSCs. These are essentially mature adult cells reprogrammed to become primal versions of themselves that can then grow into a wide variety of other cell types.

    To do this, Esswein and Ying-Yu Lin, PhD, a former PhD student in Gerecht’s lab, took commercial iPSCs and used a well-established procedure to get them to grow into common endothelial cells that form the inner layer of most of the body’s blood vessels. The researchers then used a specialized cocktail of growth factors to coax the cells into becoming the specific type of endothelial cells found in the retina. “… we differentiated human induced pluripotent stem cells into retinal endothelial cells (iRECs) via the Wnt–β-catenin pathway, namely Norrin–Frizzled4 signaling,” they explained.

    Once successful, the researchers put their development to the test. In benchtop experiments, the team was able to get the iRECs to form the same networks and structures that they do within the body. The team then subjected these lab-grown tissues to low oxygen and high glucose levels, which are detrimental conditions often seen within real people. These conditions are fundamental causes of diabetic retinopathy (DR), the leading cause of vision loss in working-age people in the United States, and caused the tissue barrier to break down just like it does in patients. They wrote in summary, “Overall, we were able to robustly recapitulate the DR phenotype in 2D and 3D with our iRECs, exemplifying their ability to be utilized for in vitro disease modeling and to elucidate aberrant pathways and therapeutic targets.”

    The researchers then tried their lab-grown cells as a therapy for mouse models with weak, unstructured retinal blood vessels. When injected into the mice before any actual vision loss occurred, these cells successfully integrated into the existing tissue and helped develop strong blood vessels with strong barriers. “When injected into oxygen-induced retinopathy mice, iRECs integrated into the host vascular network and revascularized the ischemic eye, rescuing the tissue,” they stated.

    “The tests showed that these lab-grown cells have promise for preventative treatments, especially since they should be easier and cheaper to obtain using our technique,” Esswein said. “And while our benchtop experiments did not attempt to model a wide variety of specific eye diseases in these studies, we’re confident we can create excellent human tissue models in the lab to help better understand these diseases and uncover therapies.”

    Moving forward, the researchers are planning to explore these potential uses for their retinal endothelial cells both in their laboratory and through emerging industry partnerships. The group also has a patent pending that covers both the stem cell-based therapeutics and in vitro modeling for drug discovery and testing. In their paper they concluded “Our study establishes functional human iRECs and microphysiological iBRB models that facilitate mechanistic studies aimed at identifying therapeutic targets and promoting the revascularization of injured retinas, thereby supporting treatment advancement.”

    The post iPSC-Derived Retinal Endothelial Cells Offer Platform for Studying Diseases appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Claude Science is Here, Antibiotics Designed by Text Prompt Among Applications https://edusehat.com/en/claude-science-is-here-antibiotics-designed-by-text-prompt-among-applications https://edusehat.com/en/claude-science-is-here-antibiotics-designed-by-text-prompt-among-applications Wed, 01 Jul 2026 03:45:07 +0700 Edusehat Claude, Science, Here, Antibiotics, Designed, Text, Prompt, Among, Applications Anthropic has released Claude Science, an AI workbench for scientists that consolidates fragmented research tools, including over 60 scientific databases and connectors pre-configured for genomics, proteomics, structural biology, and more, into a single reasoning layer. The platform joins an increasingly crowded ecosystem of tech platforms specialized for biology and aims to accelerate scientific discovery by making domain expertise more accessible.

    Anthropic’s life science partners are delivering applications. Basecamp Research is targeting global public health, where drug-resistant infections play a role in nearly five million deaths per year. The London-based team has announced that its antibiotic design and vaccine target prediction EDEN models will now be available through Claude Science.

    A metagenomic foundation model, EDEN demonstrated a 97% success rate when designing functional peptides with high potency against World Health Organization (WHO) critical-priority and multidrug-resistant pathogens. The work was done in collaboration with César de la Fuente, PhD, presidential associate professor at the University of Pennsylvania.

    In a Claude Science demo, Oliver Vince, PhD, co-founder at Basecamp, uploaded a sample patient microbiology report. When given a simple natural language prompt, the platform designed peptides, predicted their efficacy, and provided a shortlist of candidates most likely to succeed in experiments in minutes.

    While generating human-ready antibiotics at the click of a button is still a step away, Vince said democratizing these tools is a powerful first step, particularly for researchers in regions where accelerated computing infrastructure is not readily accessible.

    “Most models require you to be a computational scientist,” Vince told GEN Edge. “Now, potentially any clinician in the world can chat with Claude and design an antibiotic that may work.”

    “From a strategic perspective, you want the people with the most agency to solve the problem,” added Phil Lorenz, PhD, CTO at Basecamp. “Not the model builders who are two or three steps removed.”

    Full stack

    Founded in 2019, Basecamp has spent its initial years building a full computational stack spanning data, models, and therapeutic assets.

    In addition to antibiotics and vaccines, the company’s U.S. office, based in Cambridge and led by Jonathan Finn, PhD, Basecamp CSO and former CSO of Tome Biosciences, has fine-tuned EDEN for programmable gene insertion. The approach places large therapeutic DNA sequences at precise locations in the human genome, expanding upon CRISPR-based approaches that use small edits to address a limited number of indications.

    EDEN’s generalizability is enabled by training on BaseData, the company’s proprietary dataset composed of 9.8 billion protein sequences collected over 200 diverse and extreme locations, including thermal springs, polar ice, and high-altitude plateaus, across more than 30 countries. The database provides a 10-fold expansion of known protein diversity when compared to all public databases combined.

    In March, the team published the compounding advantages of BaseData on model performance in a technical report on scaling laws for metagenomics. Basecamp is steadily pushing forward that data diversity through the Trillion Gene Atlas, a partnership with Anthropic, NVIDIA, PacBio, and Ultima Genomics that aims to scale BaseData 100-fold over the next two years.

    Vince emphasizes that model deployment and integration into real-world workflows will be critical for these models to reach their full potential. Basecamp anticipates releasing more applications over the next year.

    “I think it will surprise people what these models can do,” he said.

    The post Claude Science is Here, Antibiotics Designed by Text Prompt Among Applications appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Two Proteins with Opposing Functions Found to Support Healthy Skin Maintenance https://edusehat.com/en/two-proteins-with-opposing-functions-found-to-support-healthy-skin-maintenance https://edusehat.com/en/two-proteins-with-opposing-functions-found-to-support-healthy-skin-maintenance Tue, 30 Jun 2026 09:50:09 +0700 Edusehat Two, Proteins, with, Opposing, Functions, Found, Support, Healthy, Skin, Maintenance Research headed by a team at Stanford Medicine has identified two proteins with opposing functions that are involved in orchestrating the development and maintenance of healthy skin.

    The proteins, NEDD8 and SUMO2, are part of a family called ubiquitin-like proteins (UBLs), and the researchers believe that modulating their activity with topical drugs could reduce inflammation, aid wound healing, and slow or halt the growth of skin cancer.

    “These two ubiquitin-like protein systems are remarkably dedicated and opposite in their functions,” said Paul Khavari, MD, PhD, chair of dermatology at the Stanford School of Medicine and senior author of the study. “One promotes the stem-cell state while the other drives differentiation. It’s like having two opposing forces that determine a cell’s fate.”

    Added clinical instructor of dermatology Mårten Winge, MD, PhD, “What’s really exciting is how specific these effects are. When we manipulate one system or the other, we see very clear and opposite outcomes. This specificity is unusual for ubiquitin-like pathways and makes these systems particularly attractive for therapeutic targeting.”

    Khavari, who is the Carl J. Herzog Professor in Dermatology in the School of Medicine, chief of dermatology at Veterans Affairs Palo Alto, and a member of the Stanford Cancer Institute, is senior author, and Winge is co-lead author of the researchers’ published paper in Science, titled “Ubiquitin-like proteins NEDD8 and SUMO2 control epithelial homeostasis, regeneration, and inflammation.” The work was carried out in collaboration with researchers at Icahn School of Medicine at Mount Sinai.

    Stratified epithelial tissues, such as the skin’s epidermis, differentiate to form protective barriers against environmental attacks, the authors wrote. “This process involves coordinated modulation of thousands of genes and is disrupted in many inflammatory or neoplastic diseases.

    Ubiquitination controls the targeted destruction and disposal of unneeded proteins in a cell. “Ubiquitin and related ubiquitin-like proteins (UBLs) comprise a major layer of protein regulation,” the team continued. The study by Khavari and colleagues has now found that in the skin, certain ubiquitin-like proteins switch on or off wide swaths of genes involved in cellular growth and development. In particular, they trigger progenitor, or stem, cells in the lower layer of the skin to either mature and migrate to the skin surface or to self-renew.

    The outer layer of your skin can be considered as two distinct compartments. On the lower level, progenitor cells or skin-specific stem cells wait to transform into keratinocytes, a more specialized cell type forming the critical skin barrier that keeps moisture in (and out),  excludes infection-causing pathogens, repels DNA-damaging ultraviolet rays, and harbors the nerve endings that allow us to sense our surroundings.

    These progenitor cells divide just enough to keep their numbers robust. But when needed—after injury or infection or when skin cells naturally slough off—a subset of progenitor cells differentiate and migrate to the skin’s surface. Disruptions in this delicate balance between stem cell maintenance and their maturation into adult keratinocytes can lead to psoriasis, poor wound healing, and skin cancer.

    The researchers were interested in understanding how the differentiation switch is flipped. “We hypothesized that differentiation-dependent proteomic remodeling diverges from RNA-level effects due to posttranslational protein modifications,” they noted. They used a wide swath of experimental approaches to assess dynamic changes in the expression of thousands of genes and proteins at various stages of keratinocyte differentiation. The results found that the maturing cells expressed increasing levels of genes and proteins involved in skin formation and decreasing levels of others associated with stem cell maintenance. Many of the proteins that decreased during differentiation bore small molecular tags that identify locations recognized by other proteins in the ubiquitin pathways—giving a hint that ubiquitination may be involved in the differentiation switch the researchers were seeking.

    Disrupting the expression of more than 200 genes in the ubiquitin pathway during keratinocyte maturation highlighted two subpathways essential for proper differentiation: NEDDylation and SUMOylation. Hobbling the NEDDylation pathway supercharged differentiation, while blocking SUMOylation prevented differentiation. Similar results were obtained when the pathways were blocked pharmacologically with existing drugs in both human keratinocytes grown in the laboratory and in human skin organoids—three-dimensional sheets of tissue about the size of a quarter that mimic the multicellular structure of human skin.

    Next, the researchers genetically engineered laboratory mice such that the expression of either Nedd8 or Sumo2—two key proteins in the NEDDylation and SUMOylation pathways—could be blocked when a triggering molecule is applied to the animals’ skin. They found that the skin of the mice developed abnormally when either Nedd8 or Sumo2 expression was halted, showing that both proteins are necessary for proper skin development.

    “Generation of conditional knockout mice established essential roles for NEDD8 in progenitor maintenance, skin regeneration, and inflammation, whereas SUMO2 was required for differentiation,” they commented. Mice unable to make Nedd8 had an overgrowth of keratinocytes on their skin’s surface (similar to psoriasis), and animals lacking Sumo2 showed impaired differentiation and a loss of the distinct layers that make up healthy skin.

    In addition to changes in the skin cells, the loss of Nedd8 and Sumo2 led to striking changes in the amounts and kinds of immune cells populating the skin. Nedd8 loss resulted in an increase in the numbers of immune cells called neutrophils in the skin and caused inflammation, while Sumo2 loss caused an increase in the numbers of another immune cell called a T cell. “In skin, NEDD8 maintained the undifferentiated epidermal state, enabled wound healing, and restrained neutrophilic inflammation,” they said. “SUMO2 promoted proper epidermal differentiation and suppressed T lymphocyte infiltration.”

    Khavari commented: “We’re not just changing individual cells—we’re changing the whole tissue microenvironment. Manipulating these pathways could have therapeutic applications for wounds, inflammation, skin aging, and even cancer.”

    Further experiments showed that the effect of Nedd8 on cell differentiation is due to its association with an RNA-binding protein called HNRNPU. “NEDD8 loss modulated the RNA binding and stabilizing functions of HNRNPU,” the team explained. In the absence of Nedd8, HNRNPU latches onto and stabilizes sets of RNA messages encoding genes for proteins essential for the differentiation of progenitor cells into keratinocytes, but when Nedd8 attaches to HNRNPU, the protein instead binds to and stabilizes RNA messages encoding proteins necessary for progenitor cell maintenance.

    “Thus, NEDD8 and SUMO2 play opposite roles in epithelial homeostasis, regeneration, and inflammation, demonstrating multiple ways ubiquitin-like networks govern tissue homeostasis,” the team reported in their paper. “The researchers are now exploring whether topical drug treatments targeting the NEDDylation or SUMOylation pathways could tilt the balance of keratinocyte differentiation to progenitor cell maintenance and to treat a variety of skin diseases and disorders.

    “The beauty of understanding these fundamental switches is that we can apply them to multiple disease states,” said co-lead author Leandra Jackrazi, an MD/PhD student. “Whether it’s promoting wound healing, reducing inflammation, or controlling cancer growth, having the ability to toggle between stemlike and differentiated states opens many doors.”

    The post Two Proteins with Opposing Functions Found to Support Healthy Skin Maintenance appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO 2026: Industry PDUFA negotiators describe their agreement with FDA https://edusehat.com/en/bio-2026-industry-pdufa-negotiators-describe-their-agreement-with-fda https://edusehat.com/en/bio-2026-industry-pdufa-negotiators-describe-their-agreement-with-fda Tue, 30 Jun 2026 02:45:03 +0700 Edusehat BIO, 2026:, Industry, PDUFA, negotiators, describe, their, agreement, with, FDA Industry leaders at BIO 2026 discussed the proposed PDUFA VIII next steps.

    After a year of discussion with the industry and 127 meetings with federal officials over six months, negotiators reached agreement on a  Prescription Drug User Fee Act (PDUFA) Commitment Letter that one described as “back to basics.”

    The resulting plan for PDUFA, which sets commitments for FDA’s operations, was an important accomplishment, according to the negotiator, Steve Berman, VP for Science and Regulatory Affairs Strategy at the Biotechnology Innovation Organization (BIO).

    “This return to basics shouldn’t be misunderstood as an unambitious PDUFA,” said Berman, who joined a June 24 BIO International Convention panel to help explain the agreement. “What we were able to achieve through these negotiations was rather remarkable, and we really think it’s going to help make the entire drug development ecosystem more efficient and effective.”

    As the main biopharma industry association, BIO was among the leaders in negotiating the PDUFA VIII proposal. BIO’s representatives in the negotiations with the FDA included Berman and Annetta Beauregard, BIO SVP of Science & Regulatory Affairs. Representatives from BIO’s membership were also in the PDUFA negotiations, including Robert J. Berlin, Head of Regulatory Policy at Vertex Pharmaceuticals.

    Berman, Beauregard, and Berlin joined other experts on the panel in San Diego to help members of the biotech industry understand the contents of the PDUFA VIII commitment and its significance for innovation and patients.

    First enacted in 1992, the PDUFA is an agreement that allows the U.S. Food and Drug Administration (FDA) to charge drug makers fees for new drug applications and for drugs that are already approved. Thanks to these user fees, the FDA has been able to become more efficient in regulating prescription drugs. The PDUFA agreement is updated and reauthorized by Congress every five years, and the eighth version of the agreement is due for reauthorization next year.

    To achieve the current draft commitment letter, BIO assessed industry needs through extensive consultation with its members, then joined meetings with the FDA and a few other industry members. The draft they agreed upon is set to be shared this summer and put through public review this fall, before being finalized and sent to Congress.

    Improvements in the PDUFA VIII proposal

    Improvements planned for PDUFA VIII would include enhanced communications during the application process, so drug makers are better able to understand and comply with FDA requirements, Berman said. There are also plans for efficiencies to make drug review faster.

    “For example, if you have a single product designed to target multiple indications reviewed by multiple review divisions throughout the FDA, it’s now going to be possible to meet with all of those divisions at once, to increase efficiency for both FDA and the biopharmaceutical industry,” he explained.

    There are also plans to improve the part of the drug approval process that deals with Chemistry, Manufacturing, and Controls (CMC) data, an issue that causes complications with about 50% of FDA applications, panelists said. PDUFA VIII includes more opportunities for interaction to solve those problems, they said.

    “It was a very collaborative negotiation with FDA and industry because none of us want to see 50% complete responses for manufacturing, and we looked for opportunities to have that dialogue earlier and often,” said Beauregard.

    Berman agreed, saying that both sides sought to resolve this issue and other issues with a focus on patient needs.

    “There’s no reason for patients to have delays in access to safe and effective medicines from preventable issues that could be discussed. And we’re really excited about these new mechanisms to have those discussions,” he said.

    What’s next for PDUFA and public involvement

    While the full draft agreement is only shared with the public this fall, it is possible to learn more about it by accessing the minutes of the meeting, said panel moderator Alexis Miller, U.S. Lead, Global Regulatory Policy at Merck.

    Panelist Barrett Tenbarge, Partner at Faegre Drinker, explained the next steps in the process. The current commitment letter is being reviewed by the Office of Management and Budget (OMB). Next, there will be a process of public review, probably sometime this fall, followed by submission of the document to Congress, where action is anticipated in January.

    The panelists said they were optimistic about achieving a PDUFA proposal that will assist in the important process of drug development and commercialization.

    “What drug developers really need in order to be successful is they need predictability and process, and PDUFA provides that so they can bring innovative therapies to patients and do it in a system that they understand the rules of and can rely on,” Berman said.

    The post BIO 2026: Industry PDUFA negotiators describe their agreement with FDA appeared first on Bio.News.

    ]]>     CCRM Ireland Would Be Established to Hasten Translation of Advanced Therapies Into Patient Treatments https://edusehat.com/en/ccrm-ireland-would-be-established-to-hasten-translation-of-advanced-therapies-into-patient-treatments https://edusehat.com/en/ccrm-ireland-would-be-established-to-hasten-translation-of-advanced-therapies-into-patient-treatments Tue, 30 Jun 2026 02:40:12 +0700 Edusehat CCRM, Ireland, Would, Established, Hasten, Translation, Advanced, Therapies, Into, Patient, Treatments Rinn Advanced Therapies, Ireland’s national research center for personalized immune cell therapies, signed a Memorandum of Understanding (MOU) with CCRM, which focuses on cell and gene therapy development and commercialization. The agreement outlines a strategic collaboration to explore establishing a CCRM-affiliated advanced therapies hub in Ireland.

    The proposed initiative, referred to as CCRM Ireland, is designed to position Ireland as a key node within CCRM’s global network of advanced therapies hubs and further strengthen Ireland’s expertise in next-generation biomedicine. CCRM’s global network comprises CCRM in Canada, CCRM Australia, and CCRM Nordic in Sweden.

    “The idea of collaborating with CCRM to establish CCRM Ireland is very attractive because of our shared commitment to improving patient outcomes,” said Sakis Mantalaris, PhD, director of Rinn Advanced Therapies. “By combining Rinn Advanced Therapies’ focus on novel personalized immune cell therapeutics with CCRM’s global platform, CCRM Ireland can accelerate the translation of cutting-edge science into accessible, high-quality treatments.”

    Through this collaboration, Rinn Advanced Therapies will lead the evaluation of how Ireland’s integrated ecosystem—spanning academia, health care, biomanufacturing and research—can be aligned with CCRM’s model for accelerating the development of advanced therapies. The partnership will explore how to advance the design and clinical translation and delivery of personalized immune cell therapies, while also leveraging Ireland’s biopharmaceutical manufacturing skills.

    CCRM Ireland would potentially support investment, venture creation and commercialization pathways, following CCRM Canada’s proven model.

    “As cell and gene therapies move from scientific promise to clinical reality, no single organization, region or country can build this industry alone,” says Michael May, president and CEO, CCRM. “CCRM’s global hubs are designed to connect world-class research, manufacturing expertise, capital and talent into a coordinated network that accelerates the development and commercialization of advanced therapies.

    By creating hubs around the world, and in the spirit of the Prime Minister of Canada’s call for middle-power countries to work together, with CCRM Ireland, we can help innovators overcome barriers to scale, strengthen local ecosystems and, most importantly, bring life-changing treatments to patients faster.”

    Rinn Advanced Therapies brings together a network that includes universities, hospitals, and national organizations with a shared mission to develop and deliver personalized immune cell therapies that are more effective, accessible and affordable for patients.

    CCRM will contribute its expertise in establishing and operating advanced therapies hubs, drawing on its experience in Canada and its growing international network. This includes proven frameworks in governance, GMP manufacturing, quality systems and commercialization.

     

    The post CCRM Ireland Would Be Established to Hasten Translation of Advanced Therapies Into Patient Treatments appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Shilpa Commissions Integrated ADC Drug Substance GMP Manufacturing Facility https://edusehat.com/en/shilpa-commissions-integrated-adc-drug-substance-gmp-manufacturing-facility https://edusehat.com/en/shilpa-commissions-integrated-adc-drug-substance-gmp-manufacturing-facility Tue, 30 Jun 2026 02:40:11 +0700 Edusehat Shilpa, Commissions, Integrated, ADC, Drug, Substance, GMP, Manufacturing, Facility India-based Shilpa Biologicals commissioned an antibody–drug conjugate (ADC) GMP manufacturing facility, purpose-built and designed to meet global regulatory approval standards including U.S. FDA, EMA, and other major health authority requirements. The facility is fully operational, with GMP qualification protocols now underway.

    According to Sridevi Khambhampaty, CEO, Shilpa Biologicals, “The manufacturing of highly potent compounds has been a core pillar of Shilpa’s identity, and this ADC drug substance facility adds a new sophisticated dimension to the capabilities of the Shilpa group. We now offer global biotech and pharma partners a uniquely integrated ADC facility built with the knowledge of our existing high potency manufacturing excellence.”

    “India has the scientific talent and now, with this facility, the infrastructure to be a serious and trusted partner in global ADC drug substance manufacturing,” said Vishnukant Bhutada, managing director, Shilpa Medicare. “We are ready to partner with the world’s leading oncology innovators.”

    The post Shilpa Commissions Integrated ADC Drug Substance GMP Manufacturing Facility appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Iron Accumulation Drives Neurodegeneration via Chronic Stress Pathway https://edusehat.com/en/iron-accumulation-drives-neurodegeneration-via-chronic-stress-pathway https://edusehat.com/en/iron-accumulation-drives-neurodegeneration-via-chronic-stress-pathway Tue, 30 Jun 2026 02:40:10 +0700 Edusehat Iron, Accumulation, Drives, Neurodegeneration, via, Chronic, Stress, Pathway Neurodegenerative diseases affect tens of millions of people worldwide. A new study published in Cell Death Discovery titled, “Sustained dysregulation of iron and glutathione homeostasis induces chronoferroptosis, a persistent ferroptotic adaptation in neuronal cells,” points to iron accumulation as a key target in the effort to predict, prevent, and treat neurodegenerative diseases. 

    “Resilience has become a huge topic of discussion when it comes to Alzheimer’s disease and other neurodegenerative disorders, trying to make the brain more resilient in the face of stressors that contribute to neurodegeneration,” said Pam Maher, PhD, co-corresponding author and a research professor at the Salk Institute. “Our study reveals that cells lose resilience when iron hits a certain level, making neurons more susceptible to stressors that damage or even kill them.” 

    Found in dark leafy greens, starchy cereals, lean meats, seafood, and other common foods, iron helps red blood cells develop, carries oxygen, makes hormones, and engages in key functions across the immune system and energy production. 

    “It’s one of the most important minerals in the body,” says co-corresponding author Nawab John Dar, PhD, a postdoctoral researcher in Maher’s lab. “So, it isn’t the iron itself that is a problem with age. It is this accumulation of iron over time that is the problem.” 

    The authors suggest iron buildup is caused by a failure in iron export machinery. Using a human-derived nerve cell line, the study generated a progressive model of iron accumulation in neuronal cells. They compared the effects of both acute (between six and eight hours) and chronic (nine days) exposure to iron and found the chronoferroptosis pathway. 

    Traditionally, ferroptosis was considered an iron-dependent cell death pathway related to lipid peroxidation. “It is like the cellular equivalent of when a cooking oil or nut goes bad. The fats in that oil or nut have undergone peroxidation,” explains Maher. 

    Chronoferroptosis adds the dimension of time to ferroptosis. The pathway does not necessarily end in cell death, but rather, ferroptosis can act as a cellular stress pathway. 

    “We think these coordinated alterations in iron-handling and antioxidant defense proteins make chronically exposed neurons vulnerable to neurodegenerative pathology,” said Dar. “Entering this state of chronoferroptosis may set neurons up for age-related failure.” 

    “It’s not the amount of iron that seals the fate of these cells,” Dar continued. “It’s the amount of time they spend under stress.” 

    Researchers aspire to detect when iron accumulation starts stressing neurons to develop new interventions for addressing iron imbalances to keep neurons resilient. 

    “It’s not something we worked on in this paper, but our lab has developed several compounds to inhibit this pathway,” says Maher. “This could really be a promising therapeutic route for boosting neuron resilience and staving off neurodegeneration as we grow older.” 

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    ]]>     Schistosomiasis Vaccine Shows Strong Immune Memory in Early Clinical Trials https://edusehat.com/en/schistosomiasis-vaccine-shows-strong-immune-memory-in-early-clinical-trials https://edusehat.com/en/schistosomiasis-vaccine-shows-strong-immune-memory-in-early-clinical-trials Tue, 30 Jun 2026 02:40:09 +0700 Edusehat Schistosomiasis, Vaccine, Shows, Strong, Immune, Memory, Early, Clinical, Trials Helminth parasites of the Schistosoma genus cause roughly 290,000 deaths annually, primarily in tropical and subtropical regions. In addition, an estimated 250 million people are currently chronically infected with Schistosoma parasites—with an additional 800 million people at risk of getting the infection—making schistosomiasis second only to malaria among the world’s deadliest tropical parasitic diseases.

    The larvae, which live in fresh water, penetrate the skin and develop into adults. Schistosomiasis can be found in nearly 80 countries and is common in sub-Saharan Africa.

    Now, new research shows promise for a vaccine being tested to prevent and treat schistosomiasis. SchistoShield® (Sm-p80 + GLA-SE) is a leading vaccine candidate for schistosomiasis that has successfully completed Phase I (USA) and Phase Ib (Africa) safety and immunogenicity clinical trials. Findings in a new report suggest that the vaccine triggered an adaptive immune effector and memory responses.

    This work is published in npj Vaccines in the paper, “Schistosomiasis vaccine SchistoShield® induces functional immune memory responses in U.S. and African populations.”

    Afzal Siddiqui, PhD, director of the Center for Tropical Medicine and Infectious Diseases and chair of the Department of Immunology and Molecular Microbiology at the TTUHSC School of Medicine has devoted decades to creating SchistoShield.

    In this study, samples taken from people who’ve received trial doses of the vaccine in both the United States and Africa now demonstrate the vaccine’s effectiveness. Using Peripheral Blood Mononuclear Cells (PBMCs) obtained from intercontinental Phase I and Phase Ib trial participants, the team analyzed adaptive immune effector and memory responses to SchistoShield.

    “The SchistoShield vaccine,” Siddiqui notes, “induced robust cell-mediated effector and memory responses, hallmarks of a potentially efficacious vaccine against schistosome/helminth parasites.”

    More specifically, the paper reports results demonstrating that “the vaccine induced pronounced effector and memory T-cell responses. Upon recall with Sm-p80 antigen, cytokines including IFN-γ, TNF-α, IL-17A, IL-9, and granzyme B were produced, indicating the generation of functionally heterogeneous CD4 T-helper and cytotoxic lymphocyte responses. Consistent with T-helper responses that promote humoral immunity, Sm-p80 antigen-specific antibody-secreting plasmablasts were detected in vaccinated volunteers who were tracked longitudinally.”

    “The people we have vaccinated, in both the U.S. and in Africa, have the memory response, both B-cell and T-cell-based,” Siddiqui said. “The vaccine is doing what it is supposed to. But always remember that these trials are very small 50 to 100 people. Now it has to go to thousands of people. So that’s where we are moving into.”

    Schistosomiasis is considered a “neglected disease” because it predominantly affects impoverished communities in tropical and subtropical regions. There’s only one drug available to treat people, but it does not prevent re-infection. Through his efforts, and the support of TTUHSC, federal grants and national and international charitable and non-profit groups, Siddiqui has been able to develop SchistoShield as a humanitarian effort, rather than making it for profit.

    “Our purpose from the beginning has been to expand access to care,” Lori Rice-Spearman, PhD, president of TTUHSC said. “Dr. Siddiqui’s work reflects that commitment through research that could help address a disease affecting millions of people around the world.”

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    ]]>     Tecan Integrates Agentic AI Into Its Introspect Lab Analytics Platform https://edusehat.com/en/tecan-integrates-agentic-ai-into-its-introspect-lab-analytics-platform https://edusehat.com/en/tecan-integrates-agentic-ai-into-its-introspect-lab-analytics-platform Mon, 29 Jun 2026 23:05:10 +0700 Edusehat Tecan, Integrates, Agentic, Into, Its, Introspect, Lab, Analytics, Platform Tecan reported the integration of agentic AI capabilities into its lab analytics platform Introspect, leveraging the NVIDIA BioNeMo Agent Toolkit, which enable AI agents to access scientific AI capabilities within the Introspect platform. The goal is to help laboratories to optimize operations.

    According to Tecan, agentic AI will allow laboratories to move beyond traditional monitoring and reactive troubleshooting toward proactive actions that help prevent issues before they impact performance, quality, or scientific outcomes. Early access to the enhanced Introspect platform is available, with applications focused on pharmaceutical, biotechnology, and clinical laboratory environments.

    A milestone in the collaboration announced in 2026, this agentic AI development demonstrates advancement of Tecan and Nvidia’s shared vision of enabling data-driven laboratories with AI-powered platforms designed to accelerate scientific discovery and improve laboratory productivity, notes a company spokesperson, who adds that agentic AI introduces a new paradigm for laboratory operations.

    Rather than identifying problems after they occur, intelligent agents can continuously analyze laboratory data, workflows, and system performance to uncover hidden patterns that limit throughput, constrain scalability, or reduce operational efficiency, explains Mukta Acharya, executive vice president and head of the life sciences business division at Tecan. By transforming data into recommended actions, laboratories can accelerate decision-making, optimize resource utilization, and proactively improve overall productivity, she continues.

    “Agentic AI has the potential to reshape how laboratories operate. By combining Tecan’s laboratory expertise with NVIDIA’s BioNeMo Agent Toolkit, we are enabling a new generation of intelligent laboratory solutions that can proactively support scientists, improve productivity, and help accelerate scientific outcomes,” says Acharya.

    The work with Nvidia reportedly also focuses on the agentic guardrails required for the responsible and reliable deployment of AI in laboratory environments. These safeguards are designed to support transparency, reliability, and controlled automation, helping in the establishment of agentic AI as a technology to support key research and operational workflows.

    The post Tecan Integrates Agentic AI Into Its Introspect Lab Analytics Platform appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Insilico, SK Launch Up-to-$2.5B Neuroimmune AI Drug Collaboration https://edusehat.com/en/insilico-sk-launch-up-to-25b-neuroimmune-ai-drug-collaboration https://edusehat.com/en/insilico-sk-launch-up-to-25b-neuroimmune-ai-drug-collaboration Mon, 29 Jun 2026 05:10:07 +0700 Edusehat Insilico, Launch, Up-to-2.5B, Neuroimmune, Drug, Collaboration Insilico Medicine will partner with SK Biopharmaceuticals to discover new artificial intelligence (AI)-based drug candidates for disorders affecting the neuroimmune area of the central nervous system (CNS), through a collaboration that could generate up to $2.5 billion for the AI-based drug developer.

    Insilico agreed to apply its Pharma.AI platform, which addresses target validation, generative chemistry, and molecule optimization, along with its preclinical drug discovery expertise, to discover, design, and optimize candidates for neuroimmune indications against targets that will originate with SK.

    SK will contribute its development and clinical capabilities in neuroimmune disorders, steering the late-stage development and commercialization of all resulting programs.

    “Some of the collaborations we do are very focused on target discovery, but here it’s more focused on the delivery of the real drug,” Alex Zhavoronkov, PhD, Insilico’s founder, co-CEO, and chief business officer, told GEN in an interview at his company’s exhibition-hall booth during the Biotechnology Innovation Organization (BIO) International Convention, held recently in San Diego.

    “Basically, we are being brought in to develop a drug, to discover and take it to a certain point, after which the partner takes it over. And they usually have a lot of choices to do it with other partners,” Zhavoronkov explained. “But they trust AI. They like AI. They like the way we design drugs and like our speed and efficiency.”

    Headquartered in Seongnam, South Korea, SK Biopharmaceuticals is a global biotech focused on the research, development, and commercialization of new therapies for CNS disorders and beyond, including radiopharmaceutical and targeted protein degradation therapies. In 2020, SK became the first Korean pharma to independently develop and commercialize a novel drug in the United States, the epilepsy treatment Xcopri® (cenobamate), after it won FDA approval a year earlier.

    Beyond epilepsy

    “This collaboration represents an important milestone in expanding our growth beyond epilepsy into new CNS therapeutic areas, building on the deep CNS expertise we have established through the successful development and commercialization of cenobamate,” Donghoon Lee, SK Biopharmaceuticals’ president and CEO, said in a statement. “By combining Insilico’s AI-powered drug discovery platform with SK Biopharmaceuticals’ clinical development and U.S. commercialization capabilities, we believe we can accelerate the discovery of innovative CNS therapies for patients.”

    “Beyond a single program, we see this collaboration as a scalable and repeatable growth platform that can be leveraged for future target discovery and development opportunities,” Lee added.

    SK Biopharmaceuticals is part of the SK Group, South Korea’s second-largest family-owned chaebol or conglomerate, after Samsung Group, and a chaebol whose holdings include the vaccine developer SK Bioscience, the contract development and manufacturing organization (CDMO) SK Pharmteco, and SK Hynix, a supplier of high bandwidth memory (HBM) chips that power the AI processors of Nvidia and AMD. SK Hynix and a sister chaebol company, SK Telecom, are investors in Rebellions, a Korean dedicated fabless design company specializing in manufacturing AI neural processing units optimized for data centers and large language models.

    Insilico’s AI-based drug development background complemented SK’s focus on leveraging AI and digital technologies across drug discovery, development, and treatment, SK Biopharmaceuticals concluded.

    “Very difficult space”

    “After you have done this,” Zhavoronkov said, pointing to a graphic showing Insilico’s AI-based pipeline, “people know that we can do this. The question is, can we do it in neuroimmunology? That is a very difficult space, one of the most difficult disease areas to tackle, given the need to develop molecules with properties that include high levels of safety and brain penetration.”

    Insilico’s pipeline includes one candidate designed to treat CNS disorders—ISM8969, a Phase I oral brain penetrant NLRP3 inhibitor, which the company is co-developing with Hygtia Therapeutics under an exclusive global license and co-development collaboration. Both companies hold 50% worldwide rights to ISM8969, with Insilico eligible to receive up to $66 million in upfront and milestone payments from Hygtia, an incubatee of Shenzhen Pengfu Fund of Fosun Health Capital and Fosun Pharma.

    Insilico is leading initial clinical development of ISM8969, from IND submission through execution of the Phase I trial (NCT07581431) for the drug’s initial indication of Parkinson’s disease. Hygtia will lead subsequent global clinical studies, regulatory submissions, and commercialization activities.

    Discovered using the company’s generative AI platform Chemistry42, ISM8969 has shown strong efficacy, favorable safety, and robust blood-brain barrier (BBB) penetration, leading to marked anti-inflammatory activity in preclinical studies, according to Insilico.

    Unlike other drug developers that concentrate on a few therapeutic areas, Insilico maintains a pipeline of 40+ programs across a wide variety of indications, including idiopathic pulmonary fibrosis (IPF), cancer, obesity and metabolic diseases, pain, and inflammatory diseases, including inflammatory bowel disease.

    Longevity focus

    “We focus on aging. That’s what we care about,” Zhavoronkov declared. “Most of the programs that we like to work on are focused on longevity.”

    Furthest along in clinical studies is rentosertib (formerly called ISM001-055), a small molecule designed to treat idiopathic pulmonary fibrosis (IPF) by targeting Traf2- and NCK-interacting kinase (TNIK), a serine/threonine kinase whose activation plays a crucial role in cellular processes that include signal transduction pathways essential for fibrosis development.

    Rentosertib has completed a 12-week Phase IIa trial (NCT05938920) conducted across 22 sites in China, with results published in Nature Medicine, and is in a separate Phase II trial in the United States. In the Chinese trial, rentosertib met its primary endpoint of safety and tolerability across all dose levels, and showed positive results for the secondary efficacy endpoint, wherein a dose-dependent forced vital capacity (FVC) improvement was seen.

    “We’re preparing for the next step. When that gets announced, it’s going to be a big deal. Hopefully sooner than later. Like, much sooner than much later,” Zhavoronkov said.

    As in later this year?

    “It’s in the second half, but maybe closer to the earlier second half,” he replied.

    The U.S. trial has not progressed as quickly as the Chinese trial. “We have not seen a trial slower than that in our history. Enrollment is just extremely slow because our criteria for enrollment are very high. Also, there are not that many [IPF] patients compared with China, where it was just much faster,” Zhavoronkov said.

    Given the slow speed of the U.S. trial, he said, it would be more worthwhile to just start a Phase IIb or Phase III following more data from China. “It’s a game of chess, so to speak. You need to time it [an additional trial], and you need to properly adjust to the realities of enrollment.”

    In April, Insilico received investigational new drug (IND) clearance from China’s Center for Drug Evaluation (CDE) to begin a Phase I study of inhalable rentosertib in IPF—the company’s 13th pipeline program to receive IND clearance. The study will evaluate the safety, tolerability, and pharmacokinetic (PK) profiles of rentosertib inhalation solution—first through a randomized, double-blind, placebo-controlled trial in healthy participants involving single and multiple ascending dose cohorts; then through a non-randomized, open-label evaluation in IPF patients who will receive multiple doses. Approximately 80 people are expected to be enrolled.

    “Most promising”

    “IPF is the most promising disease for longevity therapeutic testing because the patients are old. And even normal people up to 65, they start losing force valve capacity quite a bit, like the amount of air you can breathe out of your lungs. And it’s like 30, 40 milliliters a year. IPF patients can lose up to 400 milliliters,” Zhavoronkov said. “That’s the critical measure of lung function, and that’s what we measure in the study.”

    Insilico researchers chronicled the drug’s discovery and early development in Nature Biotechnology in March 2024, detailing a novel target discovered by Insilico’s target identification engine, PandaOmics, and a novel molecular structure designed by its generative chemistry engine, Chemistry42. Both are specific-function platforms within the company’s AI platform, Pharma.AI.

    “We are making massive progress on the AI side,” Zhavornkov said.

    Massive enough that users should expect to see either tweaks in the platform or new platforms? “100%, you’re going to see a complete rewall,” he replied, as in a secure, self-contained AI environment or “walled garden” pursued by AI developers during commercial inflection points.

    “We have so many new next-generation tools right now that it’s actually very difficult to productize them. Because at the lab level and at the platform level, we see superintelligence already. I’m talking about, we can probably go from prompt to drug in some areas: You basically prompt it, and you could make it and potentially take it,” Zhavoronkov explained. “I think we’re there. It’s just, fortunately, you have to do all the nitty-gritty testing and then clinical studies.”

    Insilico’s other Phase II program is ISM5411, a gut-restricted molecule designed to treat inflammatory bowel disease (IBD) by taking aim at another anti-aging target, PHD 1/2. Unlike with rentosertib, clinical studies for the PHD1/2 inhibitor have found it easier to recruit patients in the United States than in China, where fewer patients are diagnosed with the disease.

    The program, formerly called ISM012-042, was shown in preclinical studies to restore intestinal barrier function and alleviate gut inflammation in multiple experimental colitis models, while exhibiting favorable safety and pharmacokinetic profiles, according to a 2024 study published in Nature Biotechnology. The program is one of two that target PHD 1/2; the other is a small molecule designed to treat anemia of chronic kidney disease, for which Greater China rights have been outlicensed to TaiGen.

    Longevity-linked targets

    PHD 1/2, TNIK, and NLRP3 are three of numerous longevity-linked targets for the drug candidates within Insilico’s growing pipeline. Among the others that are targets of candidates in the clinic or IND-cleared:

    • ENPP1 (ectonucleotide phosphodiesterase 1), a target of a program designed to treat anti-PD-1/-L1 resistant cancers, and has won IND clearance.
    • KAT6 (lysine acetyltransferase 6 ) and KIF18A (kinesin family member 18A), targets of MEN2312 and MEN2501, respectively, are both Phase I cancer-fighting candidates outlicensed to Menarini Group through collaborations launched in 2024 and 2025.
    • MAT2A (methionine adenosyltransferase 2α), a target of a Phase I small molecule candidate designed to treat MTAP -/- (methylthioadenosine phosphorylase deficient) cancer.
    • QPCTL (glutaminyl-peptide cyclotransferase-like protein), a target of a first-in-class Phase I oral small molecule cancer immunotherapy for cold tumors being co-developed in partnership with Fosun.
    • TEAD (transcriptional enhanced associate domain), a target of ISM6631, a Phase I “pan-TEAD” (TEAD 1/2/3/4) inhibitor designed to treat mesothelioma and solid tumors that include epithelioid hemangioendothelioma (EHE), meningioma, glioblastoma, liposarcoma, and pancreatic cancers.
    • USP1 (ubiquitin-specific protease 1), a target of a Phase I BRCA-mutated cancer drug outlicensed to Exelixis under a 2023 collaboration.

    “Our differentiation from everybody else is novelty—novelty of the target,” Zhavoronkov said. “Nobody I know in our industry has such a large number of absolutely novel targets that have never been in the clinic before or that are novel for indication. But with novelty comes a great risk. And pharma doesn’t want to take that risk up until a certain point.”

    “Very often, you need to spend a long time in the process of discovery and then development in order to license a drug,” he added. “Once you license a drug, usually in Insilico’s case, some of the pharma companies actually like to get some access to AI technologies, and then it would be structured as a licensing class collaboration.”

    Second multi-billion-dollar collaboration

    SK Biopharmaceuticals is the second multi-billion-dollar collaboration announced by Insilico this year. The first was an up-to-$2.75 billion discovery and development partnership with Eli Lilly, to which Insilico granted an exclusive global license to develop, manufacture, and commercialize what the companies described in an announcement only as “potentially best-in-class, novel oral therapeutics in preclinical development for certain indications,” without detailing the therapeutic areas where the companies plan to partner.

    “Those are early preclinical drugs that have incredible properties. I like to use the term maximally multi-parameter optimized molecule or MMOMs,” Zhavoronkov said.

    Lilly agreed to pay Insilico $115 million upfront, as well as development, regulatory, and commercial milestones plus tiered royalties on future sales. The deal continued and expanded a relationship that began late in 2023, when Lilly inked a licensing agreement allowing it to access Insilico’s Pharma.AI software suite.

    The Lilly collaboration will allow Insilico and Zhavoronkov to work with Jiye Shi, PhD, the pharma giant’s senior vice president of discovery technology & platforms and early molecule discovery, who has specialized in research on integrating machine learning and AI into the pharmaceutical pipeline. Previously at UCB, he led a computational biology team that used machine learning and computational design to create bimekizumab, a humanized interleukin-17A and F antagonist hailed as one of the first, if not the first, AI-based dual-targeting monoclonal antibodies to reach the market, where it is sold as Bimzelx® (bimekizumab-bkzx).

    In February, Shi and Zhavoronkov co-authored a paper outlining a vision for a “prompt-to-drug” pipeline, where AI not only generates novel hypotheses and designs optimized drug candidates but also orchestrates synthesis, validation, and clinical planning in a closed-loop system.

    “The realization of a true ‘prompt-to-drug’ pipeline, in which a natural language request initiates a fully autonomous drug development program, is no longer a distant aspiration. With the development of modular AI platforms, humanoid-in-the-loop robotics, and multi-agent systems, the foundational components for this vision are already operational,” wrote Shi, Zhavoronkov, and co-author David Gennert, PhD, a medical writer who at the time was Insilico’s senior scientific writer and editor.

    Insilico’s collaboration with SK, Zhavoronkov said, reflects how AI “has transformed from being a fairy tale or a promise, to being a real tool that is used routinely to discover and develop drugs.”

    “This is basically production level,” he added. “We’re not trying to do a pilot here.”

    The post Insilico, SK Launch Up-to-$2.5B Neuroimmune AI Drug Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Backed by $165M, Bionyra Pharma Launches to Advance Inflammatory Disease Biologics https://edusehat.com/en/backed-by-165m-bionyra-pharma-launches-to-advance-inflammatory-disease-biologics https://edusehat.com/en/backed-by-165m-bionyra-pharma-launches-to-advance-inflammatory-disease-biologics Sat, 27 Jun 2026 06:35:07 +0700 Edusehat Backed, 165M, Bionyra, Pharma, Launches, Advance, Inflammatory, Disease, Biologics Though he is trained as a gastroenterologist and scientist, Frédéric Marrache, MD, PhD, has always had something of an entrepreneurial itch. Following his post-doctoral program and a stint in management consulting, he made his way to Sanofi where he would work on early- to mid-stage drug development programs focused on immune-mediated diseases.

    “This was right around the time when Sanofi, together with Regeneron, was finalizing the development of Dupixent,” a prescription biologic injection used to treat multiple inflammatory conditions, he told GEN. Those experiences gave him “meaningful insights” into patient care as well as about “how to develop therapies in this space.”

    One of those insights was the scale of the unmet medical need in the immune-driven inflammatory disease space. Though some large pharma companies have developed products for the space already, “I had a few insights about what could be differentiated,” he said. That led him to engage with a team at Sofinnova Partners in early 2025. “I came in with my insights about patient needs, immunology, and target selection, and [my] view on right and wrong assets,” he said. “They came with experience in building companies” and “we mapped out the entire asset space specifically on the target and pathway of interest.” 

    Those discussions led to the launch of Bionyra Pharma, a clinical-stage biopharmaceutical company that is developing next-generation biologics for severe immunological and inflammatory diseases. The company emerged from stealth this week after raising $165 million in an oversubscribed Series A. The round was co-led by Jeito Capital and Sofinnova Partners with participation from Arkin Bio, Sanofi Ventures, Sixty Degree Capital, Vives Partners and Apollo Health Ventures. 

    Marrache serves as the co-founder and CEO of the company. In addition to the financing, Mehdi Ainouche, partner at Jeito Capital; Anta Gkelou, partner at Sofinnova Partners; Avital Adler, principal at Arkin Bio; and Laia Crespo, partner at Sanofi Ventures, will join Bionyra’s board of directors.

    “When we co-founded Bionyra with Frédéric, our conviction in both the company and his leadership was grounded in his deep expertise in immune and inflammatory diseases,” said Sofinnova’s Gkelou. “Looking ahead, we are focused on advancing these programs with the aim of bringing meaningful new treatment options to patients.” 

    Specifically, the funds will support Bionyra’s efforts to advance mono and multispecific antibodies for various inflammatory conditions including atopic dermatitis and inflammatory bowel disease (IBD). 

    Right out of the gate, Bionyra is launching a pipeline of three clinical and near-clinical anti-inflammatory therapies, some of which are already in clinical trials. The company’s first asset, BYN-002 is a TL1A monoclonal antibody with the potential to treat IBD and other TL1A-relevant indications. This therapy is currently in a fully-enrolled Phase I study in healthy people. Its next candidate, BYN-003, is a TL1A*IL-23p19 bispecific antibody that is also in Phase I testing. Both assets have been improved with half-life extension (HLE) engineering to maximize efficacy and patient benefit.

    Generally speaking, “TL1A is a game changer target right now in [immunology and inflammation] with great results in inflammatory disease,” he said. However, it is likely that this target will be relevant across multiple indications. To that end, Bionyra is keeping its options open in terms of what it will target with its TL1A assets. “Whether it’s going to be in the inflammatory bowel disease space, whether we go for another indication space or whether we decide to develop it in combination in any of these indications, that’s an option,” he said. 

    For now, the focus is on validating the safety and efficacy of both therapies in healthy volunteers. “That’s especially a question around the bispecific antibody” because there will likely be questions around the immunogenicity, he noted. “Our advantage here is that our bispecific is built on the backbone of our monospecific, so at least we have some level of early validation here, and we hope to present some results soon.”  

    A third candidate, BYN-001, is an IL-25 monoclonal antibody that has also benefited from HLE technology. It is currently in the IND-stage for atopic dermatitis and type 2 inflammation. While there are several assets in development that aim to target type 2 inflammation, once all of the me-too drugs are excluded, the field becomes narrower, Marrache said while explaining the rationale for choosing this particular drug candidate for Bionyra’s portfolio. “IL-25 has been known to be a strong driver of type 2 inflammation for some time,” he said.

    Furthermore, some recently published early clinical data from a competitor, who are developing their own asset for IL-25, “clearly validated the pathway and suggested potential for differentiation.” At the time, Bionyra was already exploring the same target space so “we were able to move very quickly” and find what, Marrache believes, is the “most potent IL-25 antibody out there” with the “longest half life.” 

    Two of the assets BYN-002 and BYN-003 were licensed from TrueLab Biopharmaceutical. Under the terms of the agreement Bionyra was granted exclusive worldwide rights, excluding Greater China, to research, develop, manufacture and commercialize both therapies. TrueLab is eligible to receive up to $985 million in total consideration related to both assets, including the upfront payment as well as development, regulatory, and commercial milestone payments. The agreement also includes tiered royalties on future net sales. In addition, TrueLab has a single-digit equity stake in Bionyra Pharma following completion of its Series A financing. 

    For its part, BYN-001 was licensed from NovaRock Biotherapeutics. Bionyra is also progressing additional preclinical assets including some from TrueLab. It will support these efforts with some of the funds from the Series A.

    The post Backed by $165M, Bionyra Pharma Launches to Advance Inflammatory Disease Biologics appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Intravesical CAR T-Cell Therapy Reduces Bladder Cancer Growth in Preclinical Model https://edusehat.com/en/intravesical-car-t-cell-therapy-reduces-bladder-cancer-growth-in-preclinical-model https://edusehat.com/en/intravesical-car-t-cell-therapy-reduces-bladder-cancer-growth-in-preclinical-model Sat, 27 Jun 2026 03:00:10 +0700 Edusehat Intravesical, CAR, T-Cell, Therapy, Reduces, Bladder, Cancer, Growth, Preclinical, Model Researchers at Weill Cornell Medicine and Roswell Park Comprehensive Cancer Center have genetically engineered CAR T cells that specifically target and kill bladder cancer (BCa) cells. Through their preclinical study the team, co-led by Taha Merghoub, PhD, a professor at Weill Cornell Medicine, identified the protein MUC16 as a clinically relevant target for bladder cancer, and demonstrated that direct delivery of MUC16-targeting CAR T cells into the bladder via a catheter can control bladder tumors in mice. The investigators say their study raises hopes that a similar approach may be effective in humans.

    The team reported on their results in Journal of Experimental Medicine, in a paper titled “Intravesical mesothelin-based CAR T cells targeting MUC16 effectively control bladder cancer in preclinical models,” concluding that their findings “… not only establish MUC16 as a clinically relevant target for anti-BCa CAR T-cell therapy, but also suggest that intravesical delivery, a commonly used administration route in urological practice, represents a viable, easy-to-implement, and more effective strategy of antitumoral adoptive CAR T-cell transfer.”

    Approximately 600,000 new cases of bladder cancer are diagnosed worldwide each year, causing nearly 200,000 deaths, the authors wrote. Treatment generally involves surgical removal of the tumor followed by chemotherapy or immunotherapy. But these approaches are associated with high recurrence and progression rates, often necessitating complete removal of the bladder, a life-altering procedure that can lead to significant complications. “Intravesical therapies are the mainstay of bladder cancer (BCa) management, but their efficacy is limited by toxicities and recurrences,” they continued. “Given these challenges there is a significant unmet clinical need, driving renewed interest in bladder-sparing therapies for patients with high-risk bladder cancer who are unfit or unwilling to have their bladder removed,” Merghoub said.

    CAR T cells are immune cells genetically engineered to express an artificial receptor protein capable of specifically targeting cancer cells. This type of immunotherapy has been successfully used to treat many different types of blood cancer. But success against solid tumors has so far been limited due to challenges that include poor tumor infiltration and off-target toxicity. Merghoub and colleagues attempted to overcome these issues by creating CAR T cells with high specificity for bladder cancer cells and then delivering them directly to the bladder via a catheter, known as intravesical delivery.

    The team developed an antigen discovery pipeline, through which they identified MUC16 as a promising BCa target. “In this study, we leveraged a computational antigen-identification pipeline, which prioritized high tumor specificity and minimal pan-tissue expression to rationally identify MUC16 as a potential target for BCa-directed CAR T-cell therapy,” they stated. The researchers also noted that MUC16 and its soluble form, CA-125, have previously been identified as prognostic biomarkers for BCa, and MUC16 has been investigated as a CAR T-cell therapy target in other malignancies, and particularly ovarian cancer.

    Through their newly reported study the investigators found that MUC16 is highly expressed on the surface of many bladder cancer cells, including types that are resistant to existing therapies, but is largely absent from normal bladder cells and other healthy tissues. “Given its favorable expression profile, absence in normal bladder, and high expression across a broad spectrum of bladder tumors analyzed collectively spanning a total of 1,292 patients, including those recalcitrant to existing therapies, MUC16 was selected as the lead candidate for BCa-specific CAR T-cell therapy development,” they wrote.

    The researchers then generated CAR T cells that target MUC16. In initial tests these CAR T cells were able to kill MUC16-positive tumors grown in the lab from patient-derived bladder cancer cells. Merghoub and colleagues then tested the ability of the MUC16-targeting CAR T cells to control the growth of human bladder cancer cells implanted in the bladders of mice. The team found that the CAR T cells were ineffective when administered intravenously, but when delivered intravesically, they reduced tumor growth and extended survival. When administered directly into the bladder, the CAR T cells were unable to spread into the rest of the body, minimizing the risk of any side effects in other tissues. “Intravesical delivery of these CAR T cells reduced the growth of BCa xenografts and prolonged survival in xenograft-bearing mice, showing superior efficacy compared with typical systemic CAR T-cell administration,” the investigators noted.

    “Development of engineered T cells for solid tumors has been challenging, in part due to normal tissue expression of potential target antigens,” Wolchok says. “Using a compartmentalized delivery system allows us to overcome this hurdle and hopefully come one step closer to broader use of CAR and transgenic T cells for common solid tumors, like bladder cancer.”

    “Our findings establish MUC16 as a clinically relevant target for CAR T-cell therapy in bladder cancer, and highlight that intravesical delivery, a commonly used administration route in urological practice, represents a feasible, effective, and readily easy-to-implement strategy for adoptive CAR T-cell transfer,” Merghoub said. “This approach could be useful for both initial treatment of bladder cancer as well as treatment refractory subsets of tumors, offering an attractive therapeutic option for patients who may have limited therapeutic alternatives besides bladder removal.”

    In their paper the team also suggest that their findings “… lay the groundwork for refining CAR T-cell therapies targeting other antigens for BCa.”

    The post Intravesical CAR T-Cell Therapy Reduces Bladder Cancer Growth in Preclinical Model appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Genome Editing at the Turning Point—Bringing CRISPR to Clinical Reality https://edusehat.com/en/genome-editing-at-the-turning-pointbringing-crispr-to-clinical-reality https://edusehat.com/en/genome-editing-at-the-turning-pointbringing-crispr-to-clinical-reality Sat, 27 Jun 2026 03:00:09 +0700 Edusehat Genome, Editing, the, Turning, Point—Bringing, CRISPR, Clinical, Reality

    Image of Laura Sepp-Lorenzino, PhD

    Laura Sepp-Lorenzino, PhD

    Former CSO
    Intellia Therapeutics

    Panelist

    Image of Laura Sepp-Lorenzino, PhD

    Laura Sepp-Lorenzino, PhD

    Laura Sepp-Lorenzino, PhD, is scientific advisor and former chief scientific officer at Intellia Therapeutics, a clinical-stage genome editing company developing potential curative CRISPR-based medicines. Previously, she was vice president and head of Nucleic Acid Therapies at Vertex Pharmaceuticals and part of External Innovation. She also held roles at Alnylam Pharmaceuticals and Merck & Co. In addition, she currently serves as the director of the American Society of Gene and Cell Therapy (ASGCT).
    Image of Shengdar Tsai, PhD

    Shengdar Tsai, PhD

    Associate Member,
    Department of Hematology
    St. Jude Children’s Research Hospital

    Panelist

    Image of Shengdar Tsai, PhD

    Shengdar Tsai, PhD

    Shengdar Tsai, PhD, is an associate member in the Department of Hematology at St. Jude Children’s Research Hospital. His lab’s research focuses on developing genome editing technologies for therapeutics, with a special interest in editing human HSCs for treatment of hemoglobinopathies such as sickle cell disease and T cells for cancer immunotherapy. In 2020, he was chosen as one of the American Society for Gene and Cell Therapy (ASGCT) Outstanding New Investigators.

    Broadcast Date: 
    • Time: 

    Complex biologics such as bifunctional antibodies are opening new therapeutic possibilities in oncology, but these molecules present CRISPR is now a household term. Stories of genome editing therapies like Casgevy and Baby KJ have been headlines in our inboxes for years. Despite those cases, and the optimism they bring, genome editing has had significant challenges moving into the clinic.

    This GEN Live show will bring together a panel of leading experts to break down the latest advances, innovations, and challenges shaping genome editing. The discussion will cover a lot of bases: CRISPR breakthroughs, emerging gene editing platforms, clinical trial milestones, regulatory shifts, access, off-target effects and safety considerations, and the growing role of large-scale population genomics in guiding precision therapies. Please join us to learn more and—because we plan to take questions from the audience—please bring your questions for our panelists, too!

    Produced with support from:

    seqWell logo

    The post Genome Editing at the Turning Point—Bringing CRISPR to Clinical Reality appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Circio’s circVec and Tcelltech’s nanoSMAR Technologies Combined to Generate Nextgen In vivo CAR-T and TCR-T Cells https://edusehat.com/en/circios-circvec-and-tcelltechs-nanosmar-technologies-combined-to-generate-nextgen-in-vivo-car-t-and-tcr-t-cells https://edusehat.com/en/circios-circvec-and-tcelltechs-nanosmar-technologies-combined-to-generate-nextgen-in-vivo-car-t-and-tcr-t-cells Fri, 26 Jun 2026 23:25:10 +0700 Edusehat Circio’s, circVec, and, Tcelltech’s, nanoSMAR, Technologies, Combined, Generate, Nextgen, vivo, CAR-T, and, TCR-T, Cells Norway-based Circio and Tcelltech, based in Germany, will collaborate using the double-stranded, non-integrating nanoSMAR vector platform for the development of next generation engineered T-cell therapies.

    Engineered T-cell therapies such as CAR-T have transformed the treatment of certain cancers. However, ex vivo manufacturing remains complex, and the shift towards in vivo approaches currently relies on viral vectors that have significant safety concerns, according to Richard Harbottle, PhD, head of vector technology and manufacturing at Tcelltech. By integrating the technologies developed by Circio and Tcelltech, the parties aim to engineer T-cells with enhanced and sustained CAR/TCR expression, without the need for viral vectors, he adds.

    “The combination of Tcelltech’s non-viral, episomal nanoSMAR DNA vector platform with Circio’s circVec expression technology holds great promise for the development of in vivo gene delivery systems that are non-disruptive to target cells, maintain high expression levels, and enable straightforward, cost-effective manufacturing,” says Harbottle. “Furthermore, the exceptionally large cargo capacity of nanoSMAR vectors—beyond what is achievable with viral approaches—enables the design of complex, and sophisticated constructs incorporating multiple payload genes and regulatory elements.”

    Circio and Tcelltech will combine Circio’s circVec circular RNA expression technology with Tcelltech’s non-viral, high-cargo capacity nanoSMAR vector platform and evaluate the combination in engineered T cells through a staged research program. An initial proof-of-concept phase will compare how strongly and how durably the different technology combinations drive gene expression in primary human T cells, followed by a functional phase in which CD19-directed CAR T cells are generated and tested for their ability to kill tumor cells.

    “In vivo T-cell therapy is one of the most exciting frontiers for our circVec technology and is a rapidly advancing approach that could make these therapies more scalable and accessible,” adds Victor Levitsky, PhD, CSO of Circio. “Tcelltech´s universal nanoSMAR platform is a promising and differentiated delivery technology for T-cells, which we expect will act synergistically with circVec-enhanced payload expression.

    “This collaboration fits into Circio’s broad business development strategy of testing circVec across multiple modalities and delivery systems to identify the optimal technology combination and identify the most promising therapeutic avenues.”

     

     

     

    The post Circio’s circVec and Tcelltech’s nanoSMAR Technologies Combined to Generate Nextgen In vivo CAR-T and TCR-T Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Nipah and Hendra Viruses: Antibody Cocktail Provides Complete Protection in Hamster Model https://edusehat.com/en/nipah-and-hendra-viruses-antibody-cocktail-provides-complete-protection-in-hamster-model https://edusehat.com/en/nipah-and-hendra-viruses-antibody-cocktail-provides-complete-protection-in-hamster-model Fri, 26 Jun 2026 23:25:09 +0700 Edusehat Nipah, and, Hendra, Viruses:, Antibody, Cocktail, Provides, Complete, Protection, Hamster, Model Nipah virus—and the closely related Hendra virus—are zoonotic pathogens causing severe respiratory and neurological disease with high mortality rates. Outbreaks are rare but often devastating, with mortality rates ranging from 40 to 75 percent. There are no approved human vaccines or therapeutics for people infected with these viruses.

    Now, an international research team led by investigators at the Icahn School of Medicine at Mount Sinai has developed the first fully human monoclonal antibody cocktail shown to provide complete protection against Nipah and Hendra virus infection—even when treatment was given after infection had begun. The findings represent an important step toward developing the first antibody-based therapy for Nipah virus and establish a promising strategy for combating emerging infectious diseases.

    This work is published in Science Translational Medicine, in the paper, “A cocktail of human mAbs targeting the henipavirus fusion and receptor binding proteins provides cross-species neutralization.”

    “One of the biggest challenges in developing treatments for henipaviruses is that human survivor samples are extremely rare,” said Axel Guzman-Solis, a graduate student in the Department of Microbiology at the Icahn School of Medicine. “We wanted to determine whether we could create fully human antibodies that target the virus in multiple ways at once, making it much more difficult for the virus to evolve resistance.”

    The researchers used vaccinated humanized mice with the fusion protein (F) and receptor binding protein (RBP) of Nipah virus with the goal of isolating monoclonal antibodies. The investigators discovered two antibodies, 8G3 and 2A1, which targeted the RBP and F proteins, respectively, and together, could neutralize the virus and limit the potential for immune escape. Because the antibodies work through independent mechanisms, they create multiple barriers to infection and make it more difficult for the virus to develop resistance.

    Using cryo-EM, the researchers discovered that the 2A1 antibody neutralizes the virus by stabilizing a sugar-containing structure on the viral fusion protein rather than displacing it, as scientists had anticipated. This previously unrecognized strategy may help explain the antibody’s potency and resilience against viral escape.

    “We were surprised to find that the antibody essentially embraces a structure on the virus that many antibodies try to move out of the way,” said Benhur Lee, MD, chair in microbiology at the Icahn School of Medicine. “The finding suggests that stabilizing a viral protein can sometimes be just as effective—or even more effective—than disrupting it.”

    When administered together, the antibody cocktail completely protected hamsters from lethal Nipah virus infection. The treatment remained effective even after infection was established, an encouraging result for a disease that progresses rapidly and carries a high fatality rate.

    The findings may have broader implications for pandemic preparedness. Because many viruses rely on multiple proteins to infect cells, the researchers believe this dual-targeting strategy could be adapted for other high-priority pathogens.

    “This work provides a blueprint for developing antibody therapies that are more resistant to viral evolution,” said Lee. “Rather than relying on a single target, we can attack a virus at multiple vulnerable points simultaneously.”

    Next steps include studies in nonhuman primates, evaluation of long-term safety, and efforts to optimize the antibodies for clinical use. The team is also exploring next-generation antibody formats, including single molecules capable of targeting multiple viral proteins simultaneously, as well as approaches that could broaden protection against additional members of the henipavirus family.

    “As zoonotic outbreaks continue to emerge around the world, there is an urgent need for therapies that can be deployed quickly against high-consequence pathogens,” said Lee. “Our long-term goal is to translate these discoveries into practical tools that help protect people during future outbreaks.”

    The post Nipah and Hendra Viruses: Antibody Cocktail Provides Complete Protection in Hamster Model appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Heat waves mess with your brain. Scientists are trying to figure out why. https://edusehat.com/en/heat-waves-mess-with-your-brain-scientists-are-trying-to-figure-out-why https://edusehat.com/en/heat-waves-mess-with-your-brain-scientists-are-trying-to-figure-out-why Fri, 26 Jun 2026 19:40:03 +0700 Edusehat Heat, waves, mess, with, your, brain., Scientists, are, trying, figure, out, why.
  • Heat scrambles our thinking: Research on firefighters shows that even 15 minutes of intense heat exposure makes it harder to focus and control attention. Scientists still don't know what days-long heat waves do to our minds—or how long the effects last.

  • Mental illness makes heat deadlier: Hospital admissions for people with mental-health conditions rise nearly 10% during heat waves. People with schizophrenia were three times more likely to die during Canada's record-breaking 2021 heat wave.

  • Young people are especially at risk: Suicide rates among 15-to-24-year-olds in the US rise nearly 3% for every 1°C increase in monthly temperature—more than double the rate seen in older adults. Heat may also permanently alter brain development in young children.

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    It’s been hot in London this week. Really hot. A dangerous heat wave has hit Western Europe. Yesterday, the UK recorded its highest ever June temperature at 36.1 °C (about 97 °F). But as the weather app on my phone confirmed, it felt like 39 °C.

    It’s frightening that we are seeing such temperatures in the UK in June. According to the Met Office, the country’s national weather and climate service, June temperatures peaked at an average 19 °C (66 °F) in England between 1991 and 2020. Across Europe, the heat wave is likely to cause thousands of deaths. There will be other awful consequences for agriculture, infrastructure, and the health system.

    But this week I want to look at what the heat does to our minds and brains. Personally, I’ve found it almost impossible to think straight. The heat is distracting and my mind is foggy. I dread to think about the conditions of people who work outdoors, in even hotter regions.

    It’s not just exhaustion and confusion. The effects of heat on the brain can be deadly. And researchers are still trying to figure out why.

    Studies have confirmed that as temperatures rise, people seem to get more irritable and more violent. Most of these studies are based on associations, though. It’s difficult to directly study how a heat wave might affect our thinking, says Catherine Thompson, a cognitive psychologist at Liverpool Hope University. 

    She has been studying the effects of extreme heat on firefighters instead. It’s easier to measure people’s cognitive skills before and after they undergo scheduled training that involves entering a burning building.  

    It’s early days, but the team found that firefighters found it harder to focus and control their attention immediately after heat exposure—something people in heat waves can empathize with, I’m sure. 

    The firefighters’ skills returned to normal after 20 minutes or so of cooling down. But they’d experienced just 15 minutes of intense heat exposure. Thompson doesn’t know what the effects of living through a days-long heat wave might be—or how long they’ll last. Figuring that out might involve shipping cognitive test kits to thousands of people during the few days’ notice of an impending heat wave. “My guess [is] that no one’s done it because it’s just so difficult to do,” says Thompson. 

    Still, researchers can learn about some of the impacts of heat waves through studies after the fact. And those studies suggest that the heat seems to have more disastrous outcomes for people with mental-health disorders. 

    Those outcomes become apparent when temperatures rise above what is considered typical for a given region. “There seems to be a correlation where the hotter it gets, especially during the hottest times of the year, the worse the mental-health outcomes,” says Joshua Wortzel, who directs the Heat-Mind Lab at Hartford HealthCare in Connecticut.

    In a study published in 2023, Emma Lawrence at the University of Oxford, who studies the effect of climate change on mental health, and her colleagues reviewed the evidence linking mental-health outcomes to ambient outdoor temperatures. They found that during heat waves, there was a 9.7% increase in the rate of hospital admissions for people with such conditions. 

    “People who live with mental-health conditions are among the most susceptible to the physical impacts of heat,” says Lawrence. People with schizophrenia were found to have been three times more likely to die during the record-breaking heat wave that affected Canada in 2021, for example.

    In order to protect people, we need a better understanding of the mechanisms underlying these effects. After all, a lot of things change when it’s very, very hot. Some people may end up stuck indoors, avoiding outdoor play and exercise, and it can be difficult to get a good night of sleep, for example. Sleep, socializing, and exercise are all really important for our mental health. 

    But whether unusual heat does something specific to our brains is, as Wortzel puts it, “the million-dollar question.”

    Research in lab animals suggests that excessive heat can alter the way chemical signals work in our brain. The levels of neurotransmitters like serotonin, for example, seem to increase when rats and mice are exposed to high temperatures, according to multiple studies. The heat may also interfere with the way networks in our brains communicate with each other. It might affect the way oxygen reaches our brain cells.

    “There are so many biological reasons why brains may be negatively affected by heat,” says Wortzel.

    Emerging research suggests that for whatever reason, children and young people are among the most vulnerable. In research published earlier this week, Wortzel and his colleagues saw a 2.97% increase in the suicide rate among people in the US aged 15 to 24 for every 1 °C increase in average monthly temperature. That’s more than double the increase seen in people over the age of 24 (which is concerning in its own right).

    Other work hints that heat exposure might have long-term consequences for children’s brain development. Babies who were exposed to either extreme heat or cold appeared to have altered white matter by the time they were nine to 12 years old—although it’s not clear how these impacts might affect an individual child.

    “It seems that extreme temperature exposure for very young children may affect their brain development,” says Lawrence, who spoke to me from Oxford. She was meant to be in London for Climate Action Week, but her event, which focused on extreme heat, ended up being canceled … owing to the extreme heat.

    We are living through the effects of climate change. And that brings a new urgency to the question of how heat affects our brains. Children born in 2020 are predicted to experience around seven times the number of heat waves their grandparents did, says Lawrance. “[We] need to be serious about adapting to a warming world.”

    This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.

    ]]>     BIO 2026: ‘I am alive today because of biotech’ https://edusehat.com/en/bio-2026-i-am-alive-today-because-of-biotech https://edusehat.com/en/bio-2026-i-am-alive-today-because-of-biotech Fri, 26 Jun 2026 08:55:05 +0700 Edusehat BIO, 2026:, ‘I, alive, today, because, biotech’ From sickle cell disease to multiple sclerosis and childhood leukemia, patients and advocates reflected on becoming pioneers in therapies that are changing medicine.

    For most of her life, Jennelle Stephenson was the girl who was sick all the time. Born with sickle cell disease, she grew up with a set of rules: don’t get too cold, don’t get too hot, drink a gallon of water a day, always know where the nearest hospital is. Pain was simply the weather of her life.

    “I didn’t know any different,” she said. “That was my normal.”

    So when, after 26 years, she found a clinical trial for a gene therapy no one with her condition had ever received, the decision was clear.

    “It wasn’t necessarily me choosing between safety and risk,” Stephenson told the audience at a panel at the 2026 BIO International Convention featuring patients who had each been the first, or among the first, to undergo a transformative cell or gene therapy. “It came down to me choosing between two uncertainties. What was certain was that I was living 26 years in pain, and that had to change.”

    Eight and a half years later, she has not had a single sickle cell crisis – not one.

    The strangest part, she said, was the absence of the identity that had come with it.  “Sickness had been a place to hide,” she explained. “When that was taken away, I was left with myself, and I didn’t know who I was.” She has spent the years since figuring it out, with three Italian greyhounds, two cats, and the Tennessee mountains where she now hikes.

    “I am alive today because of biotech,” she said.

    ‘You do the extreme things, because that’s the path of hope’

    Marci McCue shares her experience being the first patient to get CAR-T cell therapy for MS at the BIO 2026 International Convention in San Diego.
    Marci McCue shares her experience being the first patient to get CAR-T cell therapy for MS at the 2026 Biotechnology Innovation Organization (BIO) International Convention in San Diego.

    Beside Stephenson sat Marci McCue, the first patient to receive CAR-T therapy in a clinical trial for multiple sclerosis (MS). When her MS began moving fast, McCue refused to wait, proactively reaching out to neurologists requesting clinical trials.

    The way she explains the decision to go first has the logic of someone who had already done the math. “If somebody told you you were going to die in a car accident, you would probably take extreme measures to just not get in a car,” she said. “So you do the extreme things, because that’s the path of hope.”

    She remembers the moment her diagnosis stopped being a secret. Her teenage daughter was studying gene and cell therapy in a high school biology class, in a coffee shop, just as McCue was going to be the first patient in the clinical trial. “I said, that’s exactly what I’m doing,” she recalled. “And she just starts crying, and then I’m crying. But it’s okay, because I have a solution now. We have an answer.”

    What changed for her, she said, is the kind of thing science struggles to measure: “Now that worry is gone, and I can give my children a path forward.”

    The world’s first pediatric CAR-T patient for leukemia

    The third seat belonged to Tom Whitehead, whose daughter Emily became the world’s first pediatric patient to receive CAR-T cell therapy for leukemia. Emily was five, and her family faced a choice between taking her home for hospice and trying a therapy no child had ever received.

    “It didn’t take us very long to sign the consent,” Whitehead said.

    Just 23 days after her infusion, Emily was cancer-free.

    She is considered cured today, in a case that helped spark a cancer immunotherapy revolution that has since benefited more than 50,000 patients worldwide. The Whiteheads built a foundation around a single phrase, “hope over hospice,” and in the past year alone, they have helped 40 families find an advanced-therapy trial instead of giving up.

    Whitehead still works full-time as a power lineman. “I didn’t realize the impact it would have on so many others,” he said. “But I’m very glad that it did.”

    Why early gene and cell therapy patients keep advocating

    INDY NXT driver James Roe speaks at the Storytelling Stage at the 2026 Biotechnology Innovation Organization (BIO) International Convention in San Diego.
    INDY NXT driver James Roe speaks at the Storytelling Stage at the 2026 Biotechnology Innovation Organization (BIO) International Convention in San Diego.

    None of them treated being first as the end of the story. “I’m patient one. That means there’s going to be more, and that there needs to be more,” McCue said. “If we don’t keep pushing, if we don’t keep building on these experiments, we won’t ever get to that big breakthrough where a cure is possible, where it’s affordable, where it’s accessible.”

    That conviction – that going first is about the people who come next – ran through the convention beyond the panel. On the Storytelling Stage, INDY NXT driver James Roe, who has raced at the top of motorsport while managing type 2 asthma since childhood, drew the same line between his sport and the science around him.

    “In racing, we’re constantly refining our engineering, analyzing performance data, and chasing our approach to gain every possible advantage on track,” he said. “Managing my asthma diagnosis has also come in stages over my career, and with the help of fitness and medication, I’ve adapted to high-intensity environments where my heart rate can often average more than 130 to 150 beats per minute.”

    These are the stories BIO’s Fight of Our Lives campaign was created to share: the human face of an industry marking 50 years of breakthroughs, with McCue among its featured patients.

    For the patients who went first, the breakthrough has already arrived. What they ask of the industry is that the next ones not have to wait as long.

    Roe put that charge to the room directly: “The work happening in biotech today isn’t just about what’s next in science; it’s about what’s next for people, it’s about opening doors, expanding limits, and giving people a chance to chase goals they often hadn’t thought possible,” he said. “So, I challenge you to continue to innovate your purpose. Think about the person on the other side of every breakthrough, every trial, and every decision. Someone’s life can be completely transformed by your work.”

    The post BIO 2026: ‘I am alive today because of biotech’ appeared first on Bio.News.

    ]]>     BIO 2026: Biotech threatened by pressures on patent protection https://edusehat.com/en/bio-2026-biotech-threatened-by-pressures-on-patent-protection https://edusehat.com/en/bio-2026-biotech-threatened-by-pressures-on-patent-protection Fri, 26 Jun 2026 08:55:05 +0700 Edusehat BIO, 2026:, Biotech, threatened, pressures, patent, protection At a time when U.S. biotech leadership is being challenged, the patent system that enables life-saving innovations is beset by a toxic stew of challenges.

    There are concerted campaigns by commercial interests pushing myths about patents, pressures to change the patent system due to the quickly evolving nature of digital technology, and a political will on both sides of the aisle to chip away at IP protections in a misguided attempt to impact drug prices.

    “It is an extremely concerning moment for IP, in particular for the biopharma industry, but in general for innovators in the United States,” according to Andrei Iancu, a former director of the U.S. Patent and Trademark Office (USPTO) and partner at Sullivan & Cromwell LLP. Iancu was a participant in a June 25 panel entitled “Patents in the Crosshairs: Confronting a Coordinated Assault on Intellectual Property,” one of several panels about IP at the 2026 BIO International Convention.

    This situation represents a real threat to biotech, said Joe Franklin, Chief Legal and Policy Officer for the Biotechnology Innovation Organization (BIO) and the moderator of the panel.

    “Patent law is very complicated, yet fundamental to the biotech industry,” Franklin said. “And it’s very difficult to work through some of these questions from a policy perspective because of that complexity. But it’s essential.”

    Tom Stoll, Senior Director of Federal Government Affairs at Genentech, listed various threats to IP coming from regulators, legislators, and the courts. Proposed legislation that would offer harmful solutions to problems that are not really problems include four different types of laws addressing multiple patents, or “patent thickets”; legislation targeting “pay for delay” and “product hopping”; and legislation regarding “skinny labels” that seek to empower generics development by weakening IP protection.

    Reasons for pressures on IP

    As several panelists noted, some of the most vocal advocacy for weakening patents comes from Silicon Valley. Digital innovations become obsolete quickly, and those seeking to build new innovations based on existing programs do not want to wait 20 years for a patent to expire.

    Another pressure, coming from both sides of the aisle, is the popular idea that reducing patent protections would be a way to make the drug industry more competitive and drive prices down, but this is a fallacy, panelists said.

    “There’s a lot of people who believe that patents stifle competition. I would actually argue the opposite, for life sciences in particular, especially biotech, where the timelines to get to market and the cost to develop a product are extremely high,” said panelist Dede Willis, CEO of Orbit Geonomics. “The patents are actually increasing competition because they’re enabling new technologies to be funded and commercialized.”

    Another fallacy behind weakening patents to reduce prices is the idea that drug makers set prices by themselves, said panelist Ipsita Smolinski, Founder & Managing Director of Capitol Street. She noted the impact that pharmacy benefit managers (PBMs), the 340B program, and other factors have on prices.

    Potential solutions for patent reform

    One approach to reducing pressure for patent reform from digital tech developers might be to create one system of shorter patents for software and another system providing the traditional protection prescription drugs need, according to Iancu.

    “It would provide new and useful protections for those new technologies, but also leave the system alone – remove the pressure – for the technologies that need protection,” he explained.

    It’s not an easy fix, according to Iancu. Achieving this kind of wide-ranging reform would be difficult and pose a lot of challenges, including for biotech innovations that involve digital innovation, he explained.

    All the attacks on the patent system are happening at a time when many pro-patent lawmakers are retiring from Congress, according to Smolinski. She said it is important to educate the new lawmakers about the importance of patents. Also, drug companies themselves should add to the advocacy efforts being undertaken by BIO and other organizations.

    “If we’re not engaging, we’re not doing our job,” she said.

    The post BIO 2026: Biotech threatened by pressures on patent protection appeared first on Bio.News.

    ]]>     AI Framework Surfaces New CAR T Target with Multi‑Cancer Potential https://edusehat.com/en/ai-framework-surfaces-new-car-t-target-with-multicancer-potential https://edusehat.com/en/ai-framework-surfaces-new-car-t-target-with-multicancer-potential Fri, 26 Jun 2026 08:55:03 +0700 Edusehat Framework, Surfaces, New, CAR, Target, with, Multi‑Cancer, Potential A new study in Cell describes an AI‑enabled strategy that could accelerate the search for next‑generation CAR T cell targets—an enduring bottleneck in expanding the therapy beyond blood cancers. The work, titled “AI‑driven discovery of GPNMB CAR T cells as a multi‑cancer therapy,” was led by researchers at the Perelman School of Medicine at the University of Pennsylvania and Penn’s Abramson Cancer Center, with collaborators at the Icahn School of Medicine at Mount Sinai and RWTH Aachen University.

    The Penn team developed a human‑in‑the‑loop AI framework designed to systematically nominate antigens suitable for CAR T cell therapy. Rather than replacing expert judgment, the system integrates large language models (LLMs) with single-cell RNA sequencing datasets from human skin cancer and healthy tissue to generate and refine target lists that scientists then evaluate experimentally.

    The challenge is well known: while CAR T therapies have transformed treatment for several hematologic malignancies, identifying safe, selective targets in solid tumors remains slow and labor‑intensive. “Discovering a good CAR target is like trying to find a needle in a haystack, except the haystack keeps growing as more sequencing data becomes available,” said lead author Daniel Baker, PhD, who completed the work under the mentorship of Carl June, MD, and Zoltan Arany, MD, PhD. LLMs, Baker added, excel at scanning broad datasets, while human experts “go deep”—a complementary pairing the team sought to formalize.

    To test the framework, the researchers focused on skin cancer, integrating four publicly available single‑cell RNA‑seq datasets with additional public resources. More than 10,000 potential antigens were filtered using criteria relevant to CAR T design, including tumor composition, tissue specificity, and clinical feasibility. Multiple LLMs then repeatedly simulated target nomination—1,000 independent runs—to reduce noise and mitigate hallucinations. The resulting consensus list was reviewed by the team, who selected Glycoprotein non-metastatic melanoma protein B (GPNMB) as the top candidate.

    The researchers then engineered a GPNMB‑directed CAR T cell and validated its activity across several preclinical models. In mouse studies, the CAR T cells eliminated tumors not only in melanoma—the original focus of the dataset—but also in monoblastic leukemia and colorectal adenocarcinoma, suggesting broader therapeutic potential. These findings align with the paper’s highlight that GPNMB is expressed across a wide range of tumor types.

    The full framework is included in the methods section to enable adoption by other groups. The Penn team plans to apply the approach to additional cancer types and continue advancing the GPNMB CAR T candidate toward potential clinical translation.

    According to June, “this study represents one of the first uses of large language models in the field of cell and gene therapy, including CAR T cell therapy.” The framework is intentionally modular and disease‑agnostic, designed to accommodate new datasets and future LLMs as they evolve. Arany emphasized the broader implications: “This is only the tip of the iceberg, as agentic AI is on the rise.”

    The post AI Framework Surfaces New CAR T Target with Multi‑Cancer Potential appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO 2026: 340B faces growing calls for transparency, patient-focused reform https://edusehat.com/en/bio-2026-340b-faces-growing-calls-for-transparency-patient-focused-reform https://edusehat.com/en/bio-2026-340b-faces-growing-calls-for-transparency-patient-focused-reform Fri, 26 Jun 2026 05:15:02 +0700 Edusehat BIO, 2026:, 340B, faces, growing, calls, for, transparency, patient-focused, reform “The 340B drug pricing program was created to help safety net providers serve vulnerable patients,” said Ashley John, Director of Issue Advocacy at Novartis. “But we really have seen tremendous growth over the past few years, especially after the [Affordable Care Act], and as transparency slowly increases, it is very clear that hospitals need better criteria for which patients they are serving.”

    Her comments came on Day 3 of the 2026 BIO International Convention and reflect an undeniable reality: 340B is at a crossroads.

    340B is complex, lacks transparency

    “When I think of 340B being at a crossroads, I think that this program actually epitomizes the tension that we have in health policy, between access and affordability,” said Darshana Patel, Ph.D., Member of the California State Assembly for District 76.

    Her words were supported by research presented by Rory Martin, Ph.D., Senior Principal at Market Access Center of Excellence for IQVIA, and Patrick Wildman, Senior Vice President of Advocacy & Government Relations at the Lupus Foundation of America.

    “When the program started 34 years ago, there were two nonprofit hospitals participating in the program,” said Martin. “Today, there are 2,000, and the sheer size and scope of the program is distorting the entire U.S. healthcare system.”

    Additionally, Martin points out, the original intent of the program was to help vulnerable populations gain access to drugs as treatments that they couldn’t otherwise.

    “There have now been multiple studies,” Martin continued, “that call this into question. It doesn’t seem to be happening. Even worse than that, it’s actually costing patients more. It’s increasing their costs.”

    Based on research by Martin and his colleagues, it would seem that for-profit hospital systems are, in fact, being subsidized by the program to the tune of $16-$24 billion.

    This is an astounding number in and of itself, but it is even more surprising when you consider that these hospitals claim the program does not cost patients or taxpayers any money. But Martin and his colleagues found that the program’s costs were more in the area of $180 to $200 billion as of last year.

    “I think it would be wonderful if you could have a $200 billion free lunch, but I just don’t think that’s very realistic,” he said.

    The impact of ‘duplicate discounts’

    One of the reasons these costs are increasing, IQVIA found, is that 340B entities are, in essence, double-dipping through duplicate discounts.

    “Drug makers provide Medicaid discounts to participating hospitals for qualifying drugs,” Martin explained. “They also provide discounts in the form of negotiated rebates. These are given to [pharmacy benefit managers] and to payers, to employers, to help lower net cost of drugs, and you can get this particular situation, which is called a duplicate discount, where you have both a 340B discount and a negotiated rebate on the same drugs for the same patient.”

    This can not only drive up costs but also mean savings are not passed on to patients.

    On 340B, patients deserve transparency – and a voice

    “Many patients don’t know what 340B is,” explained Wildman. And what they don’t know can hurt them.

    “It’s a decision of what do we do?” Wildman said. “Nothing, and let the program continue to grow exponentially and be driven by revenues, with patients being an afterthought? Or do we take a closer look at the burden, see how it can benefit patients, how it’s not benefiting patients, and make sure that the patient’s voice and concerns and needs and access to care are front and center in debate and reform discussions.”

    And like IQVIA, the Lupus Foundation of America, along with the Arthritis Foundation, found that 340B costs were not just high, they were staggering.

    “Besides the lack of transparency in the program, which was a challenge, one of the first things that really popped out to us was the revenue in just our two disease areas [lupus and arthritis] is over $2.2 billion in revenue in the 340B program,” said Wildman. “And that really surprised us.”

    Wildman and his team were only looking at Medicare Part B and Part D in three states: California, Oklahoma, and Alabama, and for a restricted patient population.

    But these numbers do need to be exposed, and to do that, more and more 340B transparency bills are coming before state and federal legislatures.

    “As a scientist back at Genentech, bringing a single drug to market required so much testing, rigorous data, peer review, and evidence,” recalled Assemblywoman Patel. “I would love to see that kind of rigor applied to policymaking.”

    The post BIO 2026: 340B faces growing calls for transparency, patient-focused reform appeared first on Bio.News.

    ]]>     BIO 2026: FDA Leadership Confront Workforce Losses, China Competition in Drug Development https://edusehat.com/en/bio-2026-fda-leadership-confront-workforce-losses-china-competition-in-drug-development https://edusehat.com/en/bio-2026-fda-leadership-confront-workforce-losses-china-competition-in-drug-development Fri, 26 Jun 2026 05:10:10 +0700 Edusehat BIO, 2026:, FDA, Leadership, Confront, Workforce, Losses, China, Competition, Drug, Development SAN DIEGO — The U.S. Food and Drug Administration (FDA) is in the middle of a cultural and operational shift that goes beyond leadership changes. U.S.–China biotechnology competition is driving discussions around regulatory reform in the U.S. where traditional paradigms are being reviewed and reconsidered, particularly for rare diseases. And patient perspectives need to be a more integral part of the drug development continuum.  

    Those were some of the major themes that emerged from a town hall that took place at this year’s Biotechnology Innovation Organization (BIO) meeting in San Diego, which featured members of the current FDA leadership team. 

    John Crowley, BIO president and CEO, moderated the discussion with the acting directors of Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) and the acting chief of staff at the FDA. During the hour-long conversation in a room packed to the hilt with BIO attendees, they spoke about the agency’s current priorities and its plans to increase its headcount, among other initiatives. 

    Much of the discussion centered on ongoing plans to stabilize the agency’s workforce following the massive reduction in staffing implemented by the Department of Government Efficiency (DOGE) as well as departures of several leaders in rapid succession. The panelists acknowledged the disruptions to operations, the loss of institutional knowledge, and the past unpredictability at the agency, but did not dwell on it.  

    The consensus seems to be that stabilizing the agency’s workforce is an important prerequisite for successfully launching several planned initiatives. In fact, Michael Davis, MD, PhD, acting director of CDER, noted that this has been one of his top priorities. His initial efforts were aimed at “fortifying the center and specifically the workforce” as well as finding ways to retain staff retention and boost recruitment.  

    The conversation covered plans to improve overall morale, boost staff numbers, and to refocus on executing the agency’s mission. That includes implementing “some initiatives that were announced” or “have been in discussion for some time” and thinking through what is needed to support those programs, said Lowell Zeta, JD, acting chief of staff at the FDA.  

    Karim Mikhail, CBER acting director, stated that in addition to working through existing submissions, his team is also planning for future challenges and ways to address them quickly to avoid backlogs.  

    In terms of recruitment, the agency is looking to fill more than 2,200 authorized positions across the agency, Zeta said. About 600 people are currently being onboarded as part of the hiring push “so we feel like we’re making good progress.” CDER’s Davis said he is open to “bringing back good people” who would be interested in returning, as well as recruiting new candidates interested in public health who have the requisite skills.  

    The agency is also intentional about its efforts to minimize attrition, including offering opportunities for staff to meet with leadership to discuss challenges and support needs. And those efforts may be working. In CDER, for example, staff attrition has slowed to its historical rate. 

    Modernizing clinical development 

     Earlier this week, the FDA announced a slate of early actions aimed at “modernizing” and expediting early and late-stage clinical development. These were unveiled as part of Operation TrailBlazer, a U.S. Department of Health and Human Services initiative. The proposed changes are aimed at streamlining Phase I submission requirements so that drug developers have more clarity about what is necessary at this stage and what can be deferred. The agency is seeking public comments from the scientific community on some of these proposed actions.  

     The panelists positioned the proposed actions as a fundamental shift from the traditional comprehensive review approach to drug development towards a more adaptive design process. “Everybody understands the challenge we have,” Mikhail said. “We have incredible rigor” but “we need to make sure that we’re also as fast as we are rigorous.”   

    Importantly, the agency is also seeking to make patient perspectives more central to the drug development process. Asked by Crowley how this will work, Davis shared an anecdote about taking part in a listening session coordinated by the FDA for parents of patients with the rare disorder, Smith-Magenis syndrome. Asking questions like “What is it like to have children with this condition? What effect does that have on the children? What effect does that have on the family dynamic?” makes it “more real when connecting the data to what families and patients are experiencing.”  

    China crisis  

    Another major theme here and indeed throughout the conference was maintaining U.S. competitiveness and leadership in biotech. The panelists acknowledged China’s current competitive advantage in terms of the development of its biotech infrastructure and the reality of clinical trials moving overseas due to increasing costs and the regulatory burden in the U.S.  

    As Crowley put it, “China frankly is eating our lunch” and “we’re forcing so many of our innovators and companies to go to China” for early-stage clinical trials. In this climate, he noted that the FDA has a crucial role to play.  

    The FDA has traditionally been viewed as the “guardian of public health, which is an important, primary role,” Crowley said, but “this notion of being a beacon of innovation and U.S. competitiveness tied to our national security is a new and important role.” The panelists also highlighted the growing use of artificial intelligence tools, digital health technologies, and wearable sensors as an important source of innovation within the agency

    The FDA has recently signaled a willingness to revisit decisions it made over the past several months if those companies whose applications were rejected choose to resubmit them. “I want to make sure that we’re getting the decisions right in a way they have the confidence of the American public,” Davis said. “I think the public really trusts the FDA to make the right decisions” and “doing this closely with the multidisciplinary expert staff that we have.” 

    To be clear, the agency is not going to approve everything, Mikhail said. But it will make sure that patient safety is prioritized, and that a multidisciplinary group of scientific experts at the FDA provide critical input.  

    “I think everybody wants what is best for the patients,” he said. So “making sure that safety is paramount” and that “everybody is on the same page with regards to that second chance.”  

    The post BIO 2026: FDA Leadership Confront Workforce Losses, China Competition in Drug Development appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Merck KGaA to Acquire Bio-Techne for $11.3B, Expanding Life Science Tools Presence https://edusehat.com/en/merck-kgaa-to-acquire-bio-techne-for-113b-expanding-life-science-tools-presence https://edusehat.com/en/merck-kgaa-to-acquire-bio-techne-for-113b-expanding-life-science-tools-presence Fri, 26 Jun 2026 05:10:09 +0700 Edusehat Merck, KGaA, Acquire, Bio-Techne, for, 11.3B, Expanding, Life, Science, Tools, Presence Merck KGaA, Darmstadt, Germany, has agreed to acquire Bio-Techne for approximately $11.3 billion, the companies said today, in a deal designed to position the buyer as more of a leader across the life science value chain by expanding its presence in high-growth, next-generation life-sci markets with Bio-Techne’s tools, analytical technologies, and consumables.

    The deal would add Bio-Techne’s multiomics offerings, analytical technologies, and integrated workflow solutions to German Merck’s platforms and services in research, bioprocessing and advanced therapeutics, with the aim of creating a combined company capable of helping customers from discovery and translational research through development, testing and commercial manufacturing.

    Merck KGaA added that acquiring Bio-Techne would directly deliver on its mid- to long-term strategic agenda, which focuses on adding to its high-growth value drivers, integrated workflows, platformed capabilities—as well as scaling and sourcing innovation through merger-and-acquisition (M&A) deals like the Bio-Techne transaction.

    That transaction is the latest in a series of acquisitions for Merck KGaA totaling more than $35 billion, including in the U.S. with acquisitions such as Millipore (for about $7 billion in 2010), as well as Sigma-Aldrich (for $17 billion in a deal announced in 2014 and completed the following year), Versum Materials (for €5.8 billion [about $6.6 billion] in 2019), and last year, SpringWorks Therapeutics (for $3.95 billion).

    Merck KGaA said it would also benefit from Bio-Techne’s position as a leading provider of materials, analytics, and process technologies to cell therapy developers. Bio-Techne expects to acquire the ownership in Wilson Wolf it does not own immediately following the end of calendar year 2027 under the terms of a two-part forward contract between the company and Wilson Wolf, a manufacturer of cell culture devices, including the G-Rex product line. Bio-Techne holds 19.9% of Wilson Wolf that it acquired in the fiscal year that ended June 30, 2023.

    Merck KGaA employs more than 14,000 people in the U.S. across over 70 company and customer sites.

    The $11.3 billion Bio-Techne acquisition is the new third largest biopharma merger-and-acquisition (M&A) deal announced so far this year, behind the €10.7 billion ($12.268 billion) cash buyout offer for Italian-based Recordati being pursued by CVC Capital Partners and Groupe Bruxelles Lambert, which aim to take the company private; and Sun Pharmaceutical Industries’ planned $11.75 billion purchase of Organon, the women’s health drug developer spun out of Merck & Co., in a deal expected to close in early 2027.

    The previous third-largest M&A deal this year, now fourth-largest, is the $10.9 billion AbbVie purchase of Apogee Therapeutics, announced on Monday. The fifth largest deal is GlaxoSmithKline (GSK)’s planned $10.6 billion buyout of Nuvalent,  announced June 9 and expected to close in the third quarter.

    “Outstanding fit”

    “Bio-Techne is an outstanding fit that directly supports our strategic direction focused on delivering cutting-edge products and solutions across the entire industry value chain—from lab customers to those manufacturing in the biotech and pharmaceutical industries,” Kai Beckmann, chairman of the executive board and group CEO of Merck KGaA, Darmstadt, Germany, said in a statement.

    “By combining Bio-Techne’s scientific depth, innovation engine and differentiated portfolio with the global scale, manufacturing excellence and customer reach of Merck KGaA, Darmstadt, Germany, we are in a strong position to address some of the most important opportunities in life sciences and support our customers in accelerating the next generation of scientific discovery and therapeutic innovation. This positions us to deliver compelling strategic and financial benefits for shareholders, customers and employees,” Beckmann added.

    Those benefits, according to German Merck, include immediate accretion to the company’s earnings before interest, taxes, depreciation, and amortization (EBITDA) pre margin for both the Group as a while and its Life Science business segment upon closing of the acquisition deal.

    The Life Sciences segment finished last year with €8.98 billion ($10.36 billion) in revenue.  Merck KGaA does not break down its businesses further than its three segments, which also include healthcare (drug development, focused on oncology, neurology and immunology, and “global health” treatments such as for malaria) and electronics (high-tech materials).

    The deal is expected to close by late 2026 or early 2027, subject to satisfying customary closing conditions that include obtaining regulatory approvals and approval by Bio-Techne shareholders.

    Bio-Techne’s board of directors and the corporate bodies overseeing Merck KGaA, Darmstadt, Germany, have already approved the transaction, which will also add to earnings per share (EPS) by year three after closing, German Merck said.

    €140M in “synergies”

    Merck KGaA said it will carry out cost-cutting “synergies” of approximately €140 million (about $159.3 million) that are expected to be fully realized by the third year after closing.

    The planned acquisition will be funded through a combination of existing cash on hand and proceeds from new debt, Merck KGaA said, adding that it will preserve its “strong” investment-grade credit rating.

    For Minneapolis-based Bio-Techne, the acquisition is expected to increase its geographic and omnichannel access for its customers through integration of its offerings with those of Merck KGaA through a synergistic platform.

    Bio-Techne has more than 3,000 employees, with approximately 2,300 employees based in the U.S. The company operates 34 global locations and 15 manufacturing facilities across the U.S., Canada, the U.K., Switzerland and China, and generated net sales of more than $1.2 billion in the fiscal year that ended June 30, 2025.

    A leader in recombinant proteins with a half-century of heritage in next-generation R&D and new modalities, Bio-Techne said it would bring to German Merck a globally recognized portfolio of cytokines, growth factors, antibodies, and immunoassay kits. Bio-Techne is expected to strengthen the analytical and bioprocess solutions of Merck KGaA by adding to its offerings ProteinSimple, a leader in automated protein detection and analysis instruments. Bio-Techne added that its RNAscope and related in situ hybridization technologies would strengthen the capabilities of Merck KGaA, in spatial biology and diagnostics.

    “For 50 years, Bio-Techne has enabled scientific breakthroughs across proteomics, spatial biology, and novel therapeutics,” stated Kim Kelderman, president and CEO of Bio-Techne. “This transaction is a testament to the remarkable company our team has built and to the enduring value we create for our customers and stakeholders.”

    Muted enthusiasm

    Bio-Techne investors appeared to share only muted enthusiasm for the deal, as the company’s shares traded on Nasdaq rose just 19.8% to $70.53 as of 12:48 pm ET, from Wednesday’s close of $58.88 per share. Merck KGaA shares traded on XETRA rose 4.93% to €147.00 ($167.25).

    Puneet Souda, senior managing director, life science tools and diagnostics, and a senior research analyst with Leerink Partners, offered a possible explanation in a research note today: “The acquisition appears to be only a 24% premium to yesterday’s close and 26x the Street’s forecast for FY27 [enterprise value]/EBITDA compared to 16x for its LST [life science technologies] peer group.”

    “We see the acquisition multiple undervaluing what is a highly accretive asset in our view,” Souda wrote. “Historically, TECH [Bio-Techne’s stock ticker] traded at much higher multiples given their highly accretive consumables profile (80%+ consumables) of consistent 70%+ gross margins and operating margin potential.”

    One rival company in particular may benefit from the deal, Souda said: “The announcement is likely to be viewed positive for peer LST companies today, especially RVTY [Revvity] in our view.”

    At $73 per share cash, the deal price represents a 36% premium to Bio-Techne’s one-month volume weighted average trading price.

    “As part of Merck KGaA, Darmstadt, Germany, we will have greater scale and expanded capabilities to accelerate innovation and deepen our impact. Together, we will empower our customers to tackle the most important challenges in science and healthcare, helping to improve outcomes worldwide,” Kelderman added.

    The post Merck KGaA to Acquire Bio-Techne for $11.3B, Expanding Life Science Tools Presence appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Antibiotics Trigger Protein Sharing Among Bacteria, Aiding Persister Cells https://edusehat.com/en/antibiotics-trigger-protein-sharing-among-bacteria-aiding-persister-cells https://edusehat.com/en/antibiotics-trigger-protein-sharing-among-bacteria-aiding-persister-cells Fri, 26 Jun 2026 05:10:08 +0700 Edusehat Antibiotics, Trigger, Protein, Sharing, Among, Bacteria, Aiding, Persister, Cells New research headed by scientists at Baylor College of Medicine suggests that when bacteria are under antibiotic attack, it is not “every man for himself.” The team developed a genetic system in Escherichia coli to track how the cells transferred proteins between them. The results indicated that bacterial populations work as a team to survive antibiotics, pooling their resources and helping quiescent or dormant cells survive. Using different techniques, including high-resolution imaging, the team found that antibiotic treatment induced the transfer of proteins between different E. coli strains, and between E. coli and other species of bacteria.

    They discovered that antibiotics stimulate bacteria to differentiate into groups of what they describe as vesicle-producing, and protein-receiving cells, and that antibiotic “persisters” with reduced protein synthesis acquire proteins released by their neighbors. The discoveries may help to explain why some bacteria are hard to eliminate, and also point to potential future approaches to improve antibiotic effectiveness.

    “Antibiotics are designed to kill bacteria or stop them from growing,” said Christophe Herman, PhD, professor of molecular and human genetics and of molecular virology and microbiology at Baylor. “Yet many times, antibiotics leave behind a small group of survivors. These survivors are not genetically resistant; instead, they temporarily shut down certain parts of their metabolism, entering a dormant-like state that allows them to endure treatment and later regrow. Understanding how survivors form and remain is a major challenge in fighting persistent infections.”

    Herman is senior and co-corresponding author of the team’s published paper in Science,” (“Antibiotics stimulate protein transfer to persister cells,”) in which the team further explained, “Protein uptake enhanced the antibiotic persistence of recipient cells, revealing that vesicle exchange promotes bacterial survival during antibiotic treatment.”

    Scientists have long known that bacteria can help each other resist antibiotics by sharing genes that provide antibiotic resistance. But as the authors pointed out, “Whereas horizontal gene transfer is known to spread antibiotic resistance genes, far less is understood about the mechanisms and effects of horizontal protein transfer.”

    Antibiotic treatment stimulates vesicle production, so for their current study, Herman and colleagues investigated whether bacteria could also directly share proteins. Previous studies had indicated that bacteria can share proteins, but the experimental evidence was not clear. “To directly measure horizontal transfer, we constructed a genetic system in Escherichia coli consisting of a donor and a recipient strain.”

    First author Alice X. Wen, a Baylor McNair Scholar in the Medical Scientist Training Program (MD/PhD), working in the Herman lab, further explained, “To detect protein transfer, we designed a sensitive system using the bacterium Escherichia coli. We engineered one group of bacteria (donors) to make a special enzyme called Cre, and another group of the same bacteria (recipients) to contain a genetic ‘switch’ that could only flip if Cre protein entered the recipient.”

    Using this system, investigators discovered that when donor and recipient bacteria were grown together, protein transfer occurred but was rare under normal conditions. In contrast, when the bacteria were exposed to low, non-lethal levels of antibiotics, protein transfer increased by thousands of times. “We then investigated how proteins were moving from one cell to another,” Wen said. “We found that the transfer still occurred when donor cells were removed, leaving behind only the liquid in which they had grown. This ruled out direct cell-to-cell contact and pointed to something released into the environment.”

    By combining biochemical techniques and advanced microscopy, the team discovered that the proteins were transported by tiny membrane vesicles. These structures, which look like tiny bubbles, are made of bacterial membrane that pinch off from cells and float freely. “Bacterial membrane vesicles, which contain proteins, have been proposed as mediators of horizontal protein transfer,” they pointed out. “Additionally, antibiotic treatment stimulates vesicle production.”

    Looking closer at their experimental system, the team found that the recipient cells showed strong signs of dormancy—these cells slowed down protein production, reduced their metabolism, and activated genes associated with persistence, such as HipA. “Recipient cells with high HipA activity were more likely to take up protein-carrying vesicles and survive antibiotic treatment,” Wen said. “When HipA was removed, both protein uptake and survival dropped.”

    Protein transfer also helped dormant bacteria survive exposure to lethal antibiotic doses after vesicle transfer; that is, exposing cells to an increased concentration of vesicles before antibiotic treatment led to increased survival. “Protein uptake enhanced the antibiotic persistence of recipient cells, revealing that vesicle exchange promotes bacterial survival during antibiotic treatment,” the authors stated. The results suggested that transferred proteins helped dormant cells endure stress while their own protein production was shut down. “Uptake of key proteins, such as ribosomal components, metabolic enzymes, or DNA repair factors, from active neighbors may help persisters endure proteome-damaging stress despite reduced protein synthesis.”

    Herman said, “Our study shows that antibiotics cause a genetically identical group of bacteria to differentiate into two distinct groups: donor cells that respond by releasing protein-filled vesicles, and recipient cells that become dormant but capable of taking up proteins from incoming vesicles, which helps them survive,” Herman said. “This teamwork allows vulnerable members of a bacterial population to persist in the face of a potentially deadly antibiotic attack.”

    The researchers are interested in identifying the proteins in vesicles that contribute to recipient persistence. Understanding donor-recipient interactions among bacteria opens new doors in the fight against chronic and persistent infections. In conclusion, the authors stated that their work “… reveals that antibiotics stimulate the differentiation of bacteria into distinct groups of vesicle-producing and protein-receiving cells, which allows antibiotic persisters with decreased protein synthesis to acquire proteins secreted from active neighbors. New strategies to eliminate persisters could be developed by inhibiting or hijacking horizontal protein transfer.”

    The post Antibiotics Trigger Protein Sharing Among Bacteria, Aiding Persister Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     InduPro Licenses Lonza’s Linker Payload Technologies and Bioconjugation Platforms https://edusehat.com/en/indupro-licenses-lonzas-linker-payload-technologies-and-bioconjugation-platforms https://edusehat.com/en/indupro-licenses-lonzas-linker-payload-technologies-and-bioconjugation-platforms Thu, 25 Jun 2026 22:00:11 +0700 Edusehat InduPro, Licenses, Lonza’s, Linker, Payload, Technologies, and, Bioconjugation, Platforms Lonza and InduPro signed a licensing agreement to support the advancement of innovative antibody–drug conjugate (ADC) therapies. According to Lonza officials, the company, through one of its affiliated companies, will grant InduPro a non-exclusive, worldwide license to its proprietary GlycoConnect®, HydraSpace® and linker-payload technologies. The technologies, which will be applied to the development of ADCs targeting up to two oncology antigens, are intended to support the advancement of highly targeted cancer therapies.

    InduPro will combine its proprietary bispecific antibody capabilities with Lonza’s ADC platform. By leveraging these complementary technologies, the companies aim to develop differentiated therapeutic approaches designed to address complex diseases such as cancer, where precision targeting and efficacy remain critically important, notes Jan Vertommen, vice president of commercial development, advanced synthesis, Lonza.

    “By combining our expertise in bioconjugation technologies and manufacturing with InduPro’s innovative proximity guided antibody platform, we reinforce our commitment to enabling our licensing partners and supporting the advancement of next-generation ADC programs,” says Vertommen.

    “This agreement represents an important step in advancing our pipeline of proximity-driven bispecific ADCs,” adds Prakash Raman, CEO, InduPro. “By combining InduPro’s ability to identify novel, disease-specific co-target pairs with Lonza’s industry-leading ADC technologies, we aim to develop differentiated, first-in-class therapeutics that improve selectivity, expand therapeutic windows, and ultimately deliver better outcomes for patients with hard-to-treat tumors.”

    The post InduPro Licenses Lonza’s Linker Payload Technologies and Bioconjugation Platforms appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO 2026: American biotech leadership protects patients and national security https://edusehat.com/en/bio-2026-american-biotech-leadership-protects-patients-and-national-security https://edusehat.com/en/bio-2026-american-biotech-leadership-protects-patients-and-national-security Thu, 25 Jun 2026 11:20:03 +0700 Edusehat BIO, 2026:, American, biotech, leadership, protects, patients, and, national, security U.S. biotech leadership is essential to ensure patients have access to the best medicines and America can maintain its national security. But that leadership is not guaranteed.

    “We need to close the gaps in biotech policy to make sure we are maintaining American leadership and enabling our companies in partnership with other stakeholders, such as government, to be able to meet urgent patient needs,” according to Kelly Seagraves, VP of National Security & International Affairs at the Biotechnology Innovation Organization (BIO).

    At the 2026 BIO International Convention, Seagraves led representatives from government and industry on a panel exploring challenges to the predominance of American biotech. Their conversation echoed themes addressed the day before in a fireside chat focused on funding medical countermeasures needed to address biological threats.

    Both discussions underscored the need for public-private cooperation to ensure that the biotech industry can help maintain American security.

    As Seagraves explained in an interview ahead of BIO 2026, when America leads in biotech innovation and drug discovery, patients in the U.S. will be the first to access new treatments. From a national security standpoint, biotech superiority means America can rapidly respond to a public health crisis or a bioterrorism threat and provide its military with the best medical support, Seagraves added.

    The need for a strong American biotech industry was the subject of a report commissioned by Congress and produced by the National Security Commission on Emerging Biotechnology (NSCEB).

    Government action needed

    Caitlin Frazer, Executive Director of the NSCEB, urged government action to ensure the recommendations in the NSCEB report are implemented.

    “As unsexy as bureaucracy sounds, it is really about getting the United States government into gear, making sure that somebody is looking after whether the regulatory framework can catch the innovations coming out of ARPA-H, making sure that there’s somebody for the biotech industry to talk to about the capabilities that they’re developing in a national interest,” said Frazer, who also urged industry action. “There are ample opportunities to be a part of the advocacy effort to Congress.”

    The Advanced Research Project Agency for Health (ARPA-H) is a federal government agency that advances the development of high-impact solutions to challenging health problems. Rafid Fadul, ARPA-H’s Chief Medical Officer, represented the agency on the panel.

    Some of the NSCEB report’s 49 recommendations have been implemented, but legislative and executive actions are still needed, Frazer said. One key step would be establishing “a national biotechnology coordinator at the White House, to create and implement an interagency strategy,” Frazer said.

    “Probably the most important thing that we propose empowering this person to do is to streamline biotechnology product regulation.”

    The value of a coordinator was underscored by Megan Frisk, Chief Strategy Officer of Alloy Therapeutics’ Vigilance Division, which is focused on readiness solutions for the company. Frisk previously worked for the National Security Council at the White House as a director for biotechnology risk.

    “You have all these different departments and agencies. Some live on the ‘promote’ side, some live on the ‘protect’ side,” she said. “They have different budgets. They have different authorities. They have different missions. And frankly, they don’t always need to work with each other, so you have to create this environment.”

    What the biotech industry can do

    Mike Gaffney, CEO of Cellphire Therapeutics, Inc., and a member of BIO’s Board, spoke about the challenges of securing the support needed to bring a product to market – particularly around clinical trials. Cellphire makes solutions to treat hemorrhaging, offering an alternative to Liquid Stored Platelets (LSP), which have a short shelf life. Cellphire offers a transformational innovation for victims in mass casualty events or military personnel wounded on the battlefield.

    “Local trials take too long, they cost too much, and patients aren’t getting the benefit of what the industry can provide,” he said. According to Gaffney, biotech is a “core customer” of the U.S. Food and Drug Administration (FDA), which regulates clinical trials, so the industry has a powerful voice to demand change.

    “We’re the ones employing people. If we’re not engaged in that conversation, then we’re not doing our jobs right,” he explained.

    Public-private partnership

    Emily Wheeler with Mark O'Neill of ASPR at BIO 2026

    The challenges around developing biotech solutions for emergency preparedness was covered in the fireside chat between Emily Wheeler, BIO’s VP of Infectious Disease Policy, and Mark O’Neill, Chief of Staff for Administration for Strategic Preparedness and Response (ASPR).

    O’Neill said the government wants to fund biotechs developing medical countermeasures to address biosecurity risks. These companies should engage with the Biopharmaceutical Manufacturing Preparedness (BioMaP) Consortium and the Rapid Response Partnership Vehicle (RRPV), he said.

    “Many of the medical countermeasures needed to protect the nation against CBRN (Chemical, Biological, Radiological, and Nuclear) threats and emerging infectious diseases do not have a sustainable commercial market,” said Wheeler. “Public-private partnerships are therefore essential to ensure these products can be developed, manufactured, and maintained as part of our national preparedness and biodefense infrastructure.”

    The post BIO 2026: American biotech leadership protects patients and national security appeared first on Bio.News.

    ]]>     After 50 years of biotech, can AI help answer the next impossible question? https://edusehat.com/en/after-50-years-of-biotech-can-ai-help-answer-the-next-impossible-question https://edusehat.com/en/after-50-years-of-biotech-can-ai-help-answer-the-next-impossible-question Thu, 25 Jun 2026 07:45:03 +0700 Edusehat After, years, biotech, can, help, answer, the, next, impossible, question What started as a conversation, in a bar, written on a napkin in 1976, between biochemist Dr. Herbert W. Boyer and investor Robert A. Swanson, turned into the creation and commercialization of recombinant DNA (rDNA) technology to engineer bacteria to produce vital human proteins.

    “Standing here today, I can’t help but reflect on the historic firsts that brought us to this moment,” said Fritz Bittenbender, Board Chair at the Biotechnology Innovation Organization (BIO), and Senior Vice President of Public Affairs and Access at Genentech.

    “We watched the recombinant DNA yield human insulin,” he continued. “We saw multiple antibodies pioneer targeted therapeutics, and most recently, we witnessed the incredible leap of mRNA vaccine technology, providing the breakthrough that we can now use our old cells to train our immune system on how to fight and heal from within.”

    But biotech’s 50-year anniversary is not just about looking back; it is also about looking forward and asking, What is the next impossible question?

    To begin to answer that, award-winning journalist Katie Couric sat down with Ashley Magargee, CEO of Genentech, and Kimberly Powell, Vice President of Healthcare at NVIDIA, on the Wednesday morning mainstage at the 2026 BIO International Convention.

    The trio discussed the growing partnership between biotech and AI to accelerate innovation, enable scientists to work at a higher level, and make healthcare more personalized – giving us more years with our loved ones.

    Can AI make drug discovery cheaper and faster?

    Biotech has yielded some amazing breakthroughs – but it’s not easy.

    “We still have 90% of all molecules that enter clinical studies fail, and that’s an incredible failure rate,” said Magargee. “And it’s because drug discovery is very, very difficult – every aspect of it is very difficult.”

    Biotech development is like running a series of marathons, she explained, from figuring out which disease a company wants to treat, to building a molecule, to designing clinical studies, to recruiting patients for clinical studies. And with each step comes the risk of failure.

    “It takes 10 to 15 years to get medicine to patients at a cost of $2 billion on average,” she noted.

    That’s why many biotech leaders are looking to AI not only to shorten timelines and reduce costs in drug discovery, but also to expand its potential by training AI agents on datasets that can then analyze ever more data that humans simply cannot process individually.

    “It’s not about effort. It’s not even about expertise. It’s about predicting better the capacity of biology. Can we predict better what’s not going to work and what will work, so that we can then put our focus and emphasis there,” said Magargee.

    Magargee and Powell believe the math supports this.

    “The potential therapies in the world are essentially infinite,” said Powell. “The number of chemicals out there that could be a therapeutic is 10^60 – that’s 60 zeros. The number of proteins that could be a potential therapy is 10^180. It makes you think of the metaphor of the needle in the haystack. Part of solving that challenge is creating conditions where we could potentially model biology in a computer in a way that is far beyond human capabilities.”

    And there have been early successes.

    “We’ve been able to design a molecule recently that usually would have taken us years to do, and we did it in a matter of months,” said Magargee.

    AI is the tool, not the scientist

    Genentech, NVIDIA, and Katie Couric at the 2026 BIO International Convention - discussing AI and biotech's 50th anniversary.

    There has been some anxiety around the AI revolution, not only because it is a brave new world of technological progress, but also because its effects on the job market are already evident.

    But Magargee and Powell are more interested in using AI as a tool, not a replacement for human ingenuity.

    “I think the first concern you always hear about is, Oh, this is going to take away people’s jobs. And the way we view it is, actually, we need more scientists than we ever have before, because when they’re equipped and empowered with these tools, they’re asking bigger and more important questions than have ever been able to ask before,” said Magargee.

    And, in particular, Magargee and Powell discussed how AI could be used effectively in the regulatory space to accelerate the review and approval process.

    “The Food and Drug Administration’s systems are based on a very different era,” said Magargee, “when there was much more linear drug discovery. That drug development era, and those systems have to modernize.”

    The modernization of both innovation and regulation is important because one cannot be constrained by the other. Patients do not have time.

    “It’s going to be not just faster computers or better algorithms or more interesting science,” concluded Magargee to applause, “it’s going to be so many more birthdays for people, it’s going to be so many more memories, and so much more time with your loved ones, and that’s going to be the impact that we’re going to have together.”

    “Science is fundamentally changing,” added Powell, “and I believe that we will create the conditions where medicines will be created not just for populations at large, but for a person of one. It will be specialized just for you.”

    The post After 50 years of biotech, can AI help answer the next impossible question? appeared first on Bio.News.

    ]]>     Drug Targets LDL Receptor Pathway to Control Cholesterol https://edusehat.com/en/drug-targets-ldl-receptor-pathway-to-control-cholesterol https://edusehat.com/en/drug-targets-ldl-receptor-pathway-to-control-cholesterol Thu, 25 Jun 2026 07:40:08 +0700 Edusehat Drug, Targets, LDL, Receptor, Pathway, Control, Cholesterol Cholesterol-related heart disease remains the leading cause of death worldwide, and while doctors have more tools than ever to treat it, many patients still can’t achieve safe cholesterol levels or can’t tolerate the side effects of available medications. Researchers at the University of California (UC), San Diego, School of Medicine have now uncovered a hidden biological pathway, dependent on a protein known as Ral, which explains why high-cholesterol diets steadily chip away at our body’s ability to clear harmful low-density lipoprotein (LDL) cholesterol from the blood. The team‘s preclinical study, including tests in mice, also identified a drug candidate already proven safe in humans that could potentially target the pathway.

    “We’ve known for a long time that a high-cholesterol diet reduces the liver’s ability to clear cholesterol from the blood, but we didn’t fully understand why,” said Alan Saltiel, PhD, professor of medicine at UC San Diego School of Medicine and director of the UC San Diego/UCLA Diabetes Research Center. “This new discovery explains a critical piece of that puzzle.” Saltiel is senior author of the researchers’ published paper in Nature, titled “Dietary cholesterol activates a Ral-dependent pathway driving LDLR turnover,” in which they concluded, “Together, our findings reveal a Ral-dependent signalling pathway as a key regulator of LDLR turnover and cholesterol homeostasis.”

    Disruptions in cholesterol homeostasis are closely linked to an increased risk of atherosclerosis and cardiovascular disease (CVD), the authors wrote. “Elevated low-density lipoprotein cholesterol (LDL-C) significantly contributes to CVD by promoting the formation of atherosclerotic plaques in arteries.”

    The liver is the main organ involved in removing cholesterol from the blood so it can be broken down and used elsewhere. This is done through LDL receptors (LDLRs), which sit on the surface of liver cells and act like docking stations, grabbing LDL cholesterol from the bloodstream and pulling it inside the cell for processing. “LDLRs have a crucial role in the uptake of LDL-C from the circulation by hepatocytes,” the investigators continued. The more LDL receptors on liver cells, the more cholesterol gets cleared from the blood, which is why most cholesterol-lowering drugs, such as statins or PCSK9 inhibitors, work by preserving or increasing the number of these receptors. However, the team noted, such treatments have their limitations. “The molecular switches that coordinate LDLR trafficking and turnover in response to nutritional cues, including high dietary cholesterol, remain poorly defined.”

    The new research, carried out in mice and in human cells, reveals a previously unknown mechanism that quietly works against the cholesterol removal process, slowly reducing the number of LDL receptors and contributing to high blood cholesterol. The team found that this process begins when a protein called Ral—which Saltiel has previously studied in fat cells—is activated by high dietary cholesterol. “We describe here a previously unrecognized role for Ral signaling in orchestrating LDLR cellular trafficking and lysosomal routing in hepatocytes under chronic cholesterol stress,” the team stated.

    Their studies showed that the more Ral is activated, the fewer LDL receptors remain available to clear cholesterol from the blood. This depletion process ultimately relies on a lysosomal protease enzyme called cathepsin A (CTSA). They further explained, “Ral engages the endocytic RalBP1–REPS1 complex to promote LDLR internalization and lysosomal routing, where LDLR is degraded by the lysosomal protease cathepsin A (CTSA).”

    The researchers also found that blocking CTSA with a selective small molecule inhibitor (SAR164653) was enough to stabilize LDL receptors and dramatically lower circulating LDL cholesterol in mice. “Pharmacological inhibition of CTSA activity increases hepatic LDLR function and improves cholesterol clearance, offering a potential new therapeutic strategy for hypercholesterolaemia and cardiovascular disease,” they stated.

    “There’s still a real need for new cholesterol-lowering options, since some people can’t get to safe levels even with the drugs we have now,” said Saltiel. “This new pathway we discovered is completely separate from anything that existing drugs target, so it gives us a new opportunity to fill that gap.”

    After a fundamental biological breakthrough, it typically takes significant additional research to find drugs that target it. However, in this case, a CTSA inhibitor has already been through the early stages of drug development, with the initial goal of treating heart failure. While it was eventually shelved for strategic reasons, the drug had previously advanced to a Phase I clinical trial, where it was successfully tested for safety.

    This discovery suggests that the investigational drug is already ready for testing in a Phase II trial for high cholesterol. “Luckily, there’s an experimental drug sitting on the shelf that’s already been shown to be safe in humans,” said Saltiel. “We hope to test whether this might be effective by conducting a clinical trial, which could potentially bring a new treatment option to patients much sooner than would have been expected.”

    The post Drug Targets LDL Receptor Pathway to Control Cholesterol appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO 2026: Gov. Tina Kotek on Oregon’s thriving bioscience sector https://edusehat.com/en/bio-2026-gov-tina-kotek-on-oregons-thriving-bioscience-sector https://edusehat.com/en/bio-2026-gov-tina-kotek-on-oregons-thriving-bioscience-sector Thu, 25 Jun 2026 04:10:03 +0700 Edusehat BIO, 2026:, Gov., Tina, Kotek, Oregon’s, thriving, bioscience, sector What will it take to build a stronger, more resilient U.S. life sciences industry? In a June 23 fireside chat at the 2026 BIO International Convention, Oregon Gov. Tina Kotek shared her perspective on her state’s thriving biosciences sector and advancing bold strategies to grow innovation, strengthen supply chains, and compete for the future.

    The following is an edited transcript of her remarks and interview with Bio.News.

    Q: What sets Oregon apart in creating or attracting companies, developing supporting services, and drawing investment?

    A: I would start off with talent. I think the DNA in Oregon is about innovation, creativity. I think we pride ourselves on being changemakers, and I think that lends itself to research and development. We like to make things in Oregon; we’ve made a lot of things over the years, and we have a very strong ecosystem in biosciences, making things people need. It’s also a great place to recruit to. People say, “I want to live in Oregon because it’s beautiful.”

    Oregon already has a very attractive life science and bioscience ecosystem: 75,000 jobs in our state in that sector, 2500 businesses that are connected to it.

    Under my leadership, we have established fast-track permitting for large-scale investments, so if you are an existing company and want to grow or would like to come to Oregon, we are open to making that easier for you.

    We are looking at expanding our research and development tax credit to include our life sciences partners, because we know that for those start-ups, the ability to get into the pipeline with new innovations is really critical.

    We have so many opportunities for people across our state, and all I would say is, if you’re looking for something, we probably have it, and it’s a great place to grow. And, really importantly, it’s a great place to live. And so bringing talent to Oregon is kind of easy, and we are there to make it easy for you.

    Q: Academic institutions, including academic research communities in Oregon, have been major stakeholders in the formative stages of growing our industry. In what ways have you supported this innovation ecosystem?

    A: Our private sector partners are really important to us: Genentech, Thermo Fisher, Lonza, other companies. And I think where we have been very successful in working on some of our apprenticeship programs and some of the very specific biotech sector-type things that we can connect with our higher education community colleges, our universities. That, I think, has been very helpful for us to take it to the next level.

    Last December, I announced a Prosperity Roadmap to focus on things like workforce development. It is about a tax structure that promotes innovation; I think next year we need to adjust our R&D credit. It’s about making sure that our economic development tools and apparatuses actually work for companies, so you’ve got to ask questions of people, you have to say is that really working for you and listen to the answers.

    Q: Oregon has seen efforts over the past few years to expand the state’s R&D Tax Credit, which is currently limited to semiconductor R&D activities, to include life science R&D. We would love to see this pass! What do you see as the most significant barriers to this?

    A: As I said, I want to see expansion of the R&D tax credit. I’ve said to the Prosperity Council – which is part of my roadmap for being more competitive – that we all have to be careful about the way we collect taxes. We need to agree on all the details so they are paying for things that matter. I want to be able to say if we make this change or that change, that we are going to see particular types of investment that will create good-paying jobs, provide the affordable living-wage jobs that people need to live in our state.

    Q: In your state, Nike co-founder Phil Knight and his wife, Penny, pledged an unprecedented $2 billion to the Knight Cancer Institute at Oregon Health Sciences University. This has provided incredible momentum for comprehensive cancer care, and it has also been a catalyst for startups in the region. Can you describe what this has meant for the innovation ecosystem in Portland?

    A: That gift from the Knights is transformational – the $2 billion gift for the Knight Cancer Institute with Oregon Health and Science Institute in Portland, which is on top of the $1 billion investment nearly a decade ago for cancer research. Now it’s about cancer treatment, so there’ll be a direct connection between the R&D that’s happening at OHSU, which is becoming an international destination for cancer treatment. What’s great about that is the intersection between going from research to actual treatment in one setting.

    I think it’s important to support startups. I think of the risks that people take when they launch startups, and it’s really been great to meet some of these folks, because they just have so much passion. They’re saying, “I want to try this thing, and if I can get a little bit of help, I’m going to the next level.”

    Q: Can you talk about your coalition to cooperate with other Western Governors?

    A: I did vaccine work when I was a kid advocate, so I’m very aware of CDC recommendations and things like that. I know it made sense years ago to write into our law that we’re going to follow CDC vaccine guidelines. And then, of course, the world changed, and so the Governors of California, Washington, and Hawaii also joined us. We said we need to make sure that people get their vaccines.

    It came together really quickly, actually, to have a West Coast health alliance that said we’re going to have our own scientifically-based standards on vaccine approval and that our insurance companies are going to pay for them.

    Q: You have been an advocate for biotechnology in your state and beyond. BIO is a nonpartisan organization, and we work with the Democratic Governors Association, among other groups, and you have been an advocate for biotech there. This could be politically risky as a Democrat because many voters just think “big pharma.” Why is it so important for you to advocate for biotech in places like DGA?

    A: My message to the industry is that the way to combat the lack of trust is to just be open and say, “What’s the goal? What are we all trying to do together?”

    Let’s focus again on the goal: to make things that help people. What’s been great about the companies who are in Oregon is that they do understand that they’re dependent on the workforce there and the community and making sure people have what they need. When we’re all working together, we can move mountains.

    The post BIO 2026: Gov. Tina Kotek on Oregon’s thriving bioscience sector appeared first on Bio.News.

    ]]>     Stripe, Anthropic, and OpenAI are backing an effort to stop respiratory infections https://edusehat.com/en/stripe-anthropic-and-openai-are-backing-an-effort-to-stop-respiratory-infections-11729 https://edusehat.com/en/stripe-anthropic-and-openai-are-backing-an-effort-to-stop-respiratory-infections-11729 Thu, 25 Jun 2026 04:10:03 +0700 Edusehat Stripe, Anthropic, and, OpenAI, are, backing, effort, stop, respiratory, infections
  • A $500 million bet against respiratory infections: Stripe, Anthropic, OpenAI, and other donors are backing a new nonprofit called Intercept, aimed at eliminating respiratory viruses entirely—starting with the cold and flu.

  • The economics of the sniffles are worse than you think: The average person spends a decent part of their lifetime sick with colds—yet drug companies have little financial incentive to fix this.

  • Modern science may finally make it possible: A University of Washington vaccine designer convinced Stripe's leadership that new tools—RNA drugs, engineered proteins, even nasal sprays that trap viruses—could work against many viruses at once.

  • Clean air like clean water: Intercept also plans to fund studies of large-scale air-purification systems for schools and offices, possibly using ultraviolet light to neutralize airborne viruses the way municipalities filter drinking water.

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    The common cold comes for us all—often more than once a year. And there is no way to prevent it. The best you can do is take vitamin C and stay away from people with the sniffles.

    Now the payment company Stripe, founded by brothers Patrick and John Collison, says it will fund a new $500 million nonprofit whose goal is preventing both the common cold and the flu. Its eventual aim is to get rid of respiratory viruses altogether.

    The new organization, called Intercept, will use grants and investments to back prevention approaches, including vaccines, as well as large-scale air-cleaning systems for schools, offices, and other public spaces.

    In addition to Stripe, other funders include Anthropic, Flu Lab, and the OpenAI Foundation, as well as Bill Gates and several traders at the quantitative investing fund Jane Street Capital, according to an Intercept spokesperson.

    “I think we treat respiratory infections as a minor nuisance, but have really underweighted the burden that they impose on society,” says Nan Ransohoff, the Stripe executive leading the initiative along with Charlie Petty, a venture capitalist who joined Stripe this year. On average, people spend 5% of their lifetime fighting a cold or the flu, according to Ransohoff.

    Despite that, drug companies put relatively little effort into preventing colds. Part of the problem is that the sniffles are caused by more than 200 different viruses, according to the American Lung Association, with rhinoviruses being the most common culprits. There are so many that it typically doesn’t pay to try to stop any one of them with a vaccine. “When pharma companies look at it, it’s not as attractive as other things they could work on,” says Ransohoff. “So it hasn’t attracted the resources.”

    Stripe previously organized a $1.8 billion program called Frontier to encourage the development of carbon removal technology, as a way of countering climate change. Ransohoff says removing carbon from the atmosphere and getting rid of respiratory viruses are similar in that each is “technically possible” but they “lack commercial incentives.”

    The concept for Intercept took shape after Ransohoff started talking to David Veesler, a structural biologist and vaccine designer at the University of Washington, who argued that it’s possible to come up with broad countermeasures that work against many viruses at once. 

    “He effectively sort of nerd-sniped me,” Ransohoff says of Veesler. “He convinced me that this is technically possible. He also helped me understand that some of the reasons that this hasn’t been done before was sort of an incentive problem.”

    Veesler says the growing tool kit available to scientists includes RNA drugs, antibodies, and computational protein design. For instance, one idea is to engineer virus-grabbing proteins that people could spray in their nasal passages, to catch viruses before they cause infection.

     “Most people just accept these viruses as a fact of life, and that got us thinking: Do we have to accept it?” says Veesler. “The more we thought about it, the more we realized that many of these problems have not been worked on with modern technologies.”

    The project takes inspiration from efforts to fight the covid-19 virus, where Veesler’s group was among those involved in the speedy development of vaccines, antiviral drugs, and antibodies. 

    According to Ransohoff, Intercept’s advisors will include Peter Marks, a former top FDA official, as well as Moncef Slaoui, the pharmaceutical executive who led the US coronavirus vaccine effort, Operation Warp Speed.

    A key challenge for Intercept will be coming up with ways to counter many viruses at one time. That accounts for the interest in air-cleaning technology, such as using strong ultraviolet light to inactivate viruses. The idea, the group says, is to remove them from the air in the same way municipalities remove impurities from the water supply before it’s piped to people’s homes.

    The US funds about $6.5 billion a year in virus research through the National Institute of Allergy and Infectious Disease, or NIAID. But that agency’s budget hasn’t grown in recent years, leaving more room for private philanthropy.

    And Stripe’s Collison brothers have become some of the most reliable philanthropists in viral research. After giving away “fast grants” to help labs during the covid-19 pandemic, they later joined other donors who committed $650 million to establish the Arc Institute in Palo Alto, California, which has developed AI models for biological research.

    “The diversity of viruses is just too large and seems daunting, so people don’t even try,” says Veesler. “I’m happy that someone is ready to help scientists, not accepting the status quo, and doing something different.”

    ]]>     BIO 2026: CEO Calls for U.S. Biotech Urgency and International Competitiveness https://edusehat.com/en/bio-2026-ceo-calls-for-us-biotech-urgency-and-international-competitiveness https://edusehat.com/en/bio-2026-ceo-calls-for-us-biotech-urgency-and-international-competitiveness Thu, 25 Jun 2026 04:05:08 +0700 Edusehat BIO, 2026:, CEO, Calls, for, U.S., Biotech, Urgency, and, International, Competitiveness SAN DIEGO — Biotechnology is entering one of the most transformative periods in its history. But, according to Biotechnology Innovation Organization (BIO) CEO John Crowley, outdated regulations, rising development costs, and global competition threaten to slow progress unless policymakers act.

    At the 2026 BIO International Convention in San Diego this week—which drew “roughly 20,000 attendees,” according to the organizers—Crowley outlined a vision for the future of biotechnology centered on accelerating clinical research, embracing artificial intelligence, and maintaining U.S. leadership in a rapidly evolving global bioeconomy.

    The grassroots gauntlet

    Crowley’s personal journey as a father shaped his path into biotechnology. In the late 1990s, two of his children were diagnosed with a rare form of muscular dystrophy. He left Bristol-Myers Squibb’s marketing department to co-found a biotechnology company with an Oklahoma academic researcher over scientific progress.

    The struggle to get funding was immense. Crowley reflected on his first BIO convention in 2000 amidst the excitement of the Human Genome Project: “I came and there were tens of thousands of people partnering as there is today, still a quarter of a century later. Being the 31-year-old CEO of a small startup in Oklahoma City with no money, literally nobody signed up to meet with me and nobody accepted my meeting request.”

    Crowley recalled going to the main stage, where a gentleman, rendered quadriplegic through a horse accident, came out on the stage and said, “Biotechnology—it’s a great big word that just means hope. It’s my hope that someday I can hold my wife’s hand on the beach or throw a ball to my kids.”

    Crowley, empty-handed, returned to Oklahoma City and was able to scrounge up the funds for his startup, Novazyme Pharmaceuticals, which was ultimately funded by home equity loans and credit card advances to develop rare disease treatments. Just one year later, Novazyme was acquired by Genzyme Corporation for $225 million.

    The experience engrained in Crowley two main concepts: first, developing therapeutics doesn’t always start in big pharma but, rather, often has grassroots origins; second, and relatedly, it’s an almost impossible battle for anyone outside of big pharma to fight.

    “That’s the way so much of our science happens,” Crowley said. “It comes out of great universities, and it’s a scientist and entrepreneur—and increasingly, families, patients, and patient advocates—leading the way and going through the whole journey, running that gauntlet of making medicines.”

    Modernizing clinical trials and accessible AI

    To achieve the vision of maximizing the development and reach of biotechnology, Crowley identified a handful of problems, including the need to change the current system of clinical trials. Crowley praised the FDA’s new “Project Trailblazer” initiative to modernize experimental therapy human testing. He argued that clinical trials have become excessively burdensome and costly, limiting innovation and delaying patient access to new treatments.

    Over the past year, Crowley and BIO have worked with regulators and industry stakeholders to identify development bottlenecks. “The FDA needs to continue to be the gold standard of the world,” he said, while emphasizing that modernization is necessary to make the agency a stronger “beacon of innovation.” BIO has proposed several reforms, including measures designed to streamline trial approvals and improve the efficiency of regulatory review.

    Describing recent discussions among BIO’s board of directors, which includes executives from both major pharmaceutical companies and small biotechnology startups, Crowley said there were two major strategic topics that emerged that dominated the conversation: China and AI.

    For AI, the question wasn’t about whether it could revolutionize biotechnology; rather, it had to do with making AI capabilities accessible to companies of all sizes. Crowley noted a major disparity. “Our biggest companies have the resources and the focus to think about AI. They’ve got hundreds or more people focused on this. Our small companies don’t have those resources,” he said.

    Crowly continued, “It’s also a challenge because in our industry we would work on such long timelines, and it’s hard for an entrepreneur and biotech of a small or a mid-sized company who’s invested years to get to…starting Phase III, and all of a sudden you’ve got this massive disruptive technology. That’s exactly what AI is going to be.”

    The solution, according to Crowley, is for BIO to be at the forefront to enable the rapid implementation of AI into drug development paradigms, clinical trials, and the regulatory review process.

    Challenging China

    Crowley’s most stressed point was that the United States must remain competitive against growing international rivals, particularly China. “Drug development has just gotten too costly and burdensome, and it takes too much time,” said Crowley. In this [global] bioeconomy where we need to compete and outcompete countries like China, these are reforms that are needed.”

    He characterized biotechnology as a matter of national security and argued that the United States should treat the industry as a strategic asset. While supporting bipartisan efforts in Washington to strengthen domestic biotechnology capabilities, he cautioned against policies that could create unintended consequences or limit access to potentially life-saving technologies.

    “The world is a better, safer, healthier, and more prosperous place when the United States and its allies continue to lead in biotechnology,” Crowley said.

    China has identified biotechnology as a strategic priority through multiple national development plans and has invested heavily in scientific infrastructure, manufacturing capacity, and research capabilities. Crowley argued that the most effective response is not isolation but improving the competitiveness of the U.S. innovation ecosystem.

    Crowley repeatedly returned to what he described as “man-made problems” holding the industry back. While scientific challenges will always exist, Crowley said barriers such as complex regulations, insufficient research funding, delays in patient access, and rising out-of-pocket healthcare costs are obstacles that policymakers can address. “We can’t come to this convention and cure every cancer,” he said. “But if we get together with policymakers and lawmakers, we can pretty quickly solve a lot of these man-made problems if we have the will.”

    50 years down, 50 years ahead

    As biotechnology celebrates more than 50 years of innovation, Crowley argued that the industry’s future will depend not only on scientific breakthroughs but also on its ability to modernize the systems that govern how those breakthroughs reach patients.

    “I hope you see, when you’re here at this convention, that it captures that entrepreneurial spirit,” said Crowley. “It has to be grounded in great science and research, and it’s an exciting time to be in biotech, not just reflecting about all our successes and our many failures and challenges along the way in 50 years and looking out in the months, years, and next 50 years about what biotechnology can do to extend and enhance life and to alleviate an enormous amount of human suffering.”

    With advances in gene editing, genomic medicine, artificial intelligence, and cell therapies accelerating simultaneously, Crowley believes the next era of biotechnology could surpass anything seen before—provided the industry can remove the barriers standing in its way.

    The post BIO 2026: CEO Calls for U.S. Biotech Urgency and International Competitiveness appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     First-in-Human Stem Cell Therapy Trial for Huntington’s Disease Begins at UCI Health https://edusehat.com/en/first-in-human-stem-cell-therapy-trial-for-huntingtons-disease-begins-at-uci-health https://edusehat.com/en/first-in-human-stem-cell-therapy-trial-for-huntingtons-disease-begins-at-uci-health Thu, 25 Jun 2026 04:05:07 +0700 Edusehat First-in-Human, Stem, Cell, Therapy, Trial, for, Huntington’s, Disease, Begins, UCI, Health The world’s first in-human embryonic stem cell-derived clinical trial for Huntington’s disease has launched at UCI Health, the clinical arm of the University of California, Irvine. The Phase Ib/IIa trial will evaluate the safety of hNSC-01 neural stem cells derived from embryonic stem cells delivered to the brain by a specialized neurological mapping and targeting stereotactic system.

    Huntington’s disease is a fatal, progressive genetic disorder that gradually destroys brain cells. It usually begins between the ages of 35 and 50 with symptoms that include involuntary movements, difficulty thinking and planning daily tasks, and mood changes such as depression. If successful, this therapy could prolong independent living and significantly reduce long-term care costs.

    “This clinical trial highlights the important role that an interdisciplinary academic and clinical team together with the HD families, plays in advancing medicine,” said Leslie M. Thompson, PhD, professor of psychiatry and human behavior at UC Irvine. “We are grateful to our patients and their incredible families for their bravery to provide hope for others with very few options.”

    The first patient received the intervention at UCI Health Irvine (home to Orange County’s first adult bone marrow/stem cell transplant and cellular therapy program) in May. A second patient is scheduled to receive the intervention in July.

    “The first patient intervention went very well. To date, they haven’t reported any serious adverse events,” said Ravi Rajmohan, MD, UCI Health neurologist. “This trial may help us move one step closer to a future with available treatments that could potentially slow the progression of Huntington’s disease.”

    The therapy, hNSC-01, uses pluripotent neural stem cells derived from embryonic stem cells, which were manufactured through the UC Davis GMP facility. In animal studies, the cells have been shown to protect existing brain cells, replace lost cells, rebuild impaired brain circuits, release helpful proteins, such as brain-derived neurotrophic factor (BDNF), and reduce harmful protein accumulations that damage brain cells. The stem cells were also shown to be safe over long periods in mice.

    The clinical trial will enroll 21 people ages 18 to 65 with early-stage Huntington’s disease. Twelve participants will be enrolled into a Phase Ib dose-escalation group and nine in a Phase IIa expansion group.

    The stem cells are implanted during a roughly six-hour surgical procedure done under general anesthesia. While lying face down in an MRI scanner, the patient receives stem cells implanted directly into the striatum deep in the brain, using a purchased proprietary therapy-enabling platform for navigation and surgical delivery. Damage to the striatum, which is responsible for motor control, decision-making, motivation and more, causes Huntington’s disease symptoms. Subjects will be closely monitored for safety as well as preliminary signs of potential benefit.

    The clinical trial is made possible by a $12 million grant from the California Institute of Regenerative Medicine (CIRM), and the trial is coordinated through the UC Irvine Alpha Clinic.

    The post First-in-Human Stem Cell Therapy Trial for Huntington’s Disease Begins at UCI Health appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Stripe, Anthropic and OpenAI are backing an effort to stop respiratory infections https://edusehat.com/en/stripe-anthropic-and-openai-are-backing-an-effort-to-stop-respiratory-infections https://edusehat.com/en/stripe-anthropic-and-openai-are-backing-an-effort-to-stop-respiratory-infections Thu, 25 Jun 2026 00:35:03 +0700 Edusehat Stripe, Anthropic, and, OpenAI, are, backing, effort, stop, respiratory, infections
  • A $500 million bet against respiratory infections: Stripe, Anthropic, OpenAI, and other donors are backing a new nonprofit called Intercept, aimed towards eliminating respiratory viruses entirely—starting with the cold and flu.

  • The economics of the sniffles are worse than you think: The average person spends a decent part of their lifetime sick with colds—yet drug companies have little financial incentive to fix it.

  • Modern science may finally make it possible: A University of Washington vaccine designer convinced Stripe's leadership that new tools—RNA drugs, engineered proteins, even nasal sprays that trap viruses—could work against many viruses at once.

  • Clean air like clean water: Intercept also plans to fund studies of large-scale air-purification systems for schools and offices, possibly using ultraviolet light to neutralize airborne viruses the way municipalities filter drinking water.

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    The common cold comes for us all—often more than once a year. And there is no way to prevent it. The best you can do is take vitamin C and stay away from people with the sniffles.

    Now, the payment company Stripe, founded by brothers Patrick and John Collison, says it will fund a new $500-million nonprofit whose goal is preventing both the common cold and the flu. Its eventual aim is to get rid of respiratory viruses altogether.

    The new organization, called Intercept, will use grants and investments to back prevention approaches, including vaccines, as well as large-scale air-cleaning systems for schools, offices, and other public spaces.

    In addition to Stripe, other funders include Anthropic, Flu Lab, the OpenAI Foundation, as well as Bill Gates and several traders at the quantitative investing fund Jane Street Capital, according to an Intercept spokesperson.

    “I think we treat respiratory infections as a minor nuisance, but have really underweighted the burden that they impose on society,” says Nan Ransohoff, the Stripe executive leading the initiative along with Charlie Petty, a venture capitalist who joined Stripe this year. The average person will spend 5% of their lifetime fighting a cold or the flu, according to Ransohoff.

    Despite that, drug companies put relatively little effort into preventing colds. Part of the problem is that the sniffles are caused by more than 200 different viruses, according to the American Lung Association, with rhinoviruses being the most common culprits. There are so many that it typically doesn’t pay to try to stop any one of them with a vaccine. “When pharma companies look at it, it’s not as attractive as other things they could work on,” says Ransohoff. “So it hasn’t attracted the resources.”

    Stripe previously organized a $1.8 billion program called Frontier to encourage the development of carbon removal technology, as a way of countering climate change. Ransohoff says removing carbon from the atmosphere and getting rid of respiratory viruses are similar in that each is “technically possible” but they “lack commercial incentives.”

    The concept for Intercept took shape after Ransohoff started talking to David Veesler, a structural biologist and vaccine designer at the University of Washington, who argued that it’s possible to come up with broad countermeasures that work against many viruses at once. 

    “He effectively sort of nerd-sniped me,” Ransohoff says of Vessler. “He convinced me that this is technically possible. He also helped me understand that some of the reasons that this hasn’t been done before was sort of an incentive problem.”

    Veesler says the growing toolkit available to scientists includes RNA drugs, antibodies, and computational protein design. For instance, one idea is to engineer virus-grabbing proteins that people could spray in their nasal passages, to catch viruses before they can infect people.

     “Most people just accept these viruses as a fact of life, and that got us thinking: do we have to accept it?” says Veesler. “The more we thought about it, the more we realized that many of these problems have not been worked on with modern technologies.”

    The project takes inspiration from efforts to fight the covid-19 virus, where Veesler’s group was among those involved in the speedy development of vaccines, anti-viral drugs, and antibodies. 

    According to Ransohoff, Intercept’s advisers will include Peter Marks, a former top FDA official, as well as Moncef Slaoui, the pharmaceutical executive who led the US coronavirus vaccine effort, Operation Warp Speed.

    A key challenge for Intercept will be coming up with ways to counter many—even all—viruses at one time. That accounts for the group’s interest in air-cleaning technology, such as using strong ultraviolet light to inactivate viruses. The idea, the group says, is to remove viruses from the air in the same way municipalities remove impurities from the water supply before it’s piped to people’s homes.

    The US funds about $6.5 billion a year in virus research through the National Institute of Allergy and Infectious Disease, or NIAID. But that agency’s budget hasn’t grown in recent years, leaving more room for private philanthropy.

    And Stripe’s Collison brothers have become some of the most reliable philanthropists in viral research. After giving away “fast grants” to help labs during the covid-19 pandemic, they later joined other donors who committed $650 million to establish the Arc Institute, in Palo Alto, which has developed AI models for biological research.

    “The diversity of viruses is just too large and seems daunting, so people don’t even try,” says Veesler. “I’m happy that someone is ready to help scientists, not accepting the status quo, and doing something different.”

    ]]>     Spotlight on RNA Therapeutics https://edusehat.com/en/spotlight-on-rna-therapeutics https://edusehat.com/en/spotlight-on-rna-therapeutics Thu, 25 Jun 2026 00:30:45 +0700 Edusehat Spotlight, RNA, Therapeutics

    REGISTER NOW

    Image of Drew Weissman, MD, PhD

    Drew Weissman, MD, PhD

    Professor in Vaccine Research
    Penn Medicine

    Panelist

    Image of Drew Weissman, MD, PhD

    Drew Weissman, MD, PhD

    Drew Weissman, MD, PhD, is a world-renowned physician and Roberts Family Professor in Vaccine Research at Penn Medicine. He is best known for his contributions to RNA biology and the development of COVID-19 RNA vaccines. Weissman and Katalin Karikó, PhD, were jointly awarded the 2023 Nobel Prize in Medicine for their discoveries that enabled the modified mRNA technology used in Pfizer-BioNTech and Moderna’s vaccines to prevent COVID-19. More than 15 years ago, Weissman and Karikó found a way to modify mRNA and developed a delivery technique to package the mRNA in lipid nanoparticles. The COVID-19 RNA vaccine received FDA approval in August 2021.

    Weissman is one of the academic leaders of the NSF AIRFoundry, an effort to leverage AI to improve, accelerate, and scale the design, manufacture, and delivery of RNA, which officially opened in April 2026. Weissman’s lab is currently working on a pan-coronavirus vaccine, a universal flu vaccine, and a vaccine to prevent herpes. They are working with Penn colleagues to develop cancer therapeutics with mRNA technology. And they are developing a SARS-CoV-2 mRNA vaccine with Chulalongkorn University in Thailand to help residents of Thailand and other Asian countries access lifesaving vaccines.

    Before joining Penn in 1997, Weissman was a fellow at the National Institutes of Health studying HIV in the lab of Anthony Fauci, MD. Weissman received his bachelor’s degree and master’s degree from Brandeis University. He earned his MD and PhD from Boston University and completed his residency at Beth Israel Hospital.
    Image of Zachary Ives, PhD

    Zachary Ives, PhD

    Professor of Computer and Information Science
    University of Pennsylvania

    Panelist

    Image of Zachary Ives, PhD

    Zachary Ives, PhD

    Zachary Ives, PhD, is the department chair and Adani President’s Distinguished Professor of Computer and Information Science at the University of Pennsylvania. Zack’s research interests include data integration and sharing, data provenance and trustworthiness, and machine learning systems. He is a recipient of the National Science Foundation (NSF) CAREER award, and an alumnus of the DARPA Computer Science Study Panel and Information Science and Technology advisory panel. He has also been awarded the Christian R. and Mary F. Lindback Foundation Award for Distinguished Teaching and an IEEE Technical Committee on Data Engineering Education Award, and he is a fellow of the ACM.

     

    Zack is one of the academic leaders of the U.S. NSF Artificial Intelligence-driven RNA BioFoundry (NSF AIRFoundry), an $18-million effort to leverage AI to improve, accelerate, and scale the design, manufacture, and delivery of RNA. The center officially opened in April 2026.

    Zack studied computer science at Sonoma State University and holds a PhD in computer science from the University of Washington. He joined the faculty of Penn in 2003. He is a co-author of the textbook Principles of Data Integration. He has been an associate editor for the Proceedings of the VLDB Endowment and The VLDB Journal.
    Image of Silvi Rouskin, PhD

    Silvi Rouskin, PhD

    Asst. Professor of Microbiologyy
    Harvard Medical School

    Panelist

    Image of Silvi Rouskin, PhD

    Silvi Rouskin, PhD

    Born in Bulgaria, Silvi Rouskin, PhD, is an assistant professor of microbiology at Harvard Medical School. She is the winner of the 2021 Vilcek Prize for Creative Promise in Biomedical Science. Following a six-year spell at the Whitehead Institute, where she was the Andria and Paul Heafy Whitehead Fellow, Silvi joined the faculty of Harvard Medical School in 2021.

    Silvi’s Harvard lab studies alternative RNA structures and the myriad roles they have in both viral and human biology. In particular, the lab studies how RNA folding informs alternative splicing and how misfolding can lead to disease. The lab developed DMS-MaPseq (dimethyl sulfate mutational profiling with sequencing) and DREEM (Detection-of-RNA-folding-Ensembles-using-Expectation-Maximization) algorithm to distinguish multiple RNA conformations formed by the same underlying sequence in vivo at single nucleotide resolution.

    Silvi immigrated to the United States as a teenager to pursue a career in science. She holds a degree in physics from Florida Institute of Technology and a PhD in biochemistry and molecular biology from the University of California, San Francisco. Her interest in RNA began while working as a staff research associate in the lab of Joseph DeRisi, PhD, at UCSF, where she began developing techniques for the detection of viruses associated with human disease.

    Broadcast Date: 
    • Time: 

    In anticipation of RNA Day (on August 1), GEN invites you to join our exciting Spotlight virtual event on RNA Therapeutics on Wednesday, July 29.

    We are living in a “post-genomic” world where RNA is no longer just a messenger but a programmable drug and molecular therapeutic. From the global impact of mRNA vaccines to advances in RNA editing and the potential of circular RNA, the field of RNA therapeutics is truly taking off. RNA is rapidly becoming a universal software for precision medicine.

    Over 2.5 hours, this GENSpotlight on RNA Therapeutics brings you three interlinked sessions that feature outstanding researchers exploring various aspects of RNA biology and therapeutics, including:

    • A keynote panel including two founding members of the AIRFoundry (Artificial Intelligence-driven RNA BioFoundry) at the University of Pennsylvania—Zachary Ives, PhD, and Nobel laureate Drew Weissman, MD, PhD

    • A talk from Silvi Rouskin, PhD, a leading microbiologist at Harvard Medical School, presenting new research on alternative RNA structures and their relevance in health and disease

    • Presentations from our two sponsors, 4basebio and Aldevron

    • Registration to our Spotlight on RNA Therapeutics is entirely free. We look forward to celebrating RNA Day with you (a few days early).

    Produced with support from:

    4basebio logo

    Aldevron Logo

    The post Spotlight on RNA Therapeutics appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Medra Launches Reasoning Layer for Drug Discovery Robotics https://edusehat.com/en/medra-launches-reasoning-layer-for-drug-discovery-robotics https://edusehat.com/en/medra-launches-reasoning-layer-for-drug-discovery-robotics Thu, 25 Jun 2026 00:30:34 +0700 Edusehat Medra, Launches, Reasoning, Layer, for, Drug, Discovery, Robotics As AI infrastructure for drug discovery continues to proliferate with reasoning workflows capable of generating hypotheses, candidate molecules, and experimental plans, Medra CEO Michelle Lee, PhD, argues that physical AI is the solution to addressing the next bottleneck: experimental validation at scale. 

    “Building foundation models in biology that can predict and cure disease will take thousands of years of data generation,” Lee explained in an interview with GEN Edge. “The more I looked at the field, the more I realized that this data problem is actually a robotics problem.”   

    In a new collaboration with the Defense Advanced Research Projects Agency (DARPA), Medra has launched AI Experimentalist, the scientific reasoning layer of its robotics platform. The system translates high-level research goals expressed in natural language into executable workflows that span the entire experimental cycle, from literature review, wet-lab execution, data analysis, and protocol refinement. 

    In a blog post, Medra presents an example where scientists prompt to “build an Epidermal Growth Factor Receptor (EGFR) blocking antibody assay cascade.” AI Experimentalist can propose small optimizations in execution, including testing linear DNA templates in parallel, optimizing expression conditions, and feeding results immediately into the next run, for compounding time savings from days to hours. 

    Partners can access AI Experimentalist through physical AI labs deployed on site at customer facilities or operated remotely through Medra’s flagship science laboratory, Medra Lab 001 (ML001), which unveiled in April and touts running experiments 24/7. Medra describes the 38,000 square foot facility as the largest autonomous lab in the United States. 

    Artisanal nature 

    In contrast to industrial automation, which has been powerful for repeatable tasks, such as combinatorial chemistry and screening, physical AI equips the same hardware with sensors to enable intelligent decision-making. 

    While many robotics players in biology are focused on the manufacturing step, Medra has the ambitious goal of accelerating end-to-end drug discovery campaigns. 

    “The artisanal nature of science is actually what makes certain experiments work and others fail,” said Lee. She noted that seemingly subtle variables, such as the angle of a pipette or the precise timing of mixing reagents, can have an outsized impact on experimental outcomes.  

    Medra is currently working with partners across academia, biopharma, and government to run and develop assays across a wide array of applications, including antibody discovery, protein engineering, gene editing, and cell biology. 

    Looking ahead, Lee says the bottleneck is not robotic capability, but integration and deployment. AI Experimentalist addresses this challenge through a multi-agent architecture and model-agnostic harness that allows Medra to incorporate new biological AI models and scientific agents. Among them are NVIDIA Nemotron models for protocol editing and optimization and the newly launched NVIDIA BioNeMo Agent Toolkit. 

    “The flexibility of physical AI will be incredibly key in making scientific discovery truly autonomous,” asserts Lee. 

    The post Medra Launches Reasoning Layer for Drug Discovery Robotics appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     WHO Selects NIBRT as Training Hub to Help LMICs Build Biopharma Capacity https://edusehat.com/en/who-selects-nibrt-as-training-hub-to-help-lmics-build-biopharma-capacity https://edusehat.com/en/who-selects-nibrt-as-training-hub-to-help-lmics-build-biopharma-capacity Thu, 25 Jun 2026 00:30:32 +0700 Edusehat WHO, Selects, NIBRT, Training, Hub, Help, LMICs, Build, Biopharma, Capacity Ireland’s National Institute for Bioprocessing Research and Training (NIBRT) will help biopharma engineers hone their automation and AI skills as part of a new World Health Organization (WHO) network.

    The WHO named the University College Dublin-based organization as its newest training center, explaining it will provide engineers with context-specific skills courses aligned with “regional priorities, regulatory environments.”

    NIBRT spokesman Killian O’Driscoll tells GEN, “Following a competitive application process, NIBRT has now been designated as the WHO Training Center for the European Region. NIBRT will work with partners and stakeholders to identify the skills gaps within the region and provide the appropriate training solutions, which will involve a blend of online, classroom, and practical training on biopharma manufacturing.”

    Engineers who take part will be taught how to use advanced bioprocessing technologies in a variety of manufacturing settings, according to O’Driscoll, who says the plan is to use the organization’s syllabus as a foundation.

    “Training will cover all aspects of biopharma manufacturing based on NIBRT’s award-winning curriculum, including drug substance, drug product, QC, engineering, digitalization, etc. Automation, digitalization, AI, and related areas are a core component of the NIBRT curriculum and will form part of the training solutions,” he adds.

    LMIC capacity

    The WHO established the Biomanufacturing Workforce Training Initiative in 2023 to address critical skills gaps across the biomanufacturing value chain and enable countries to translate technological advances into sustainable local production.

    NIBRT is now one of seven institutions selected. The rest of the network consists of the Institut Pasteur de Dakar in Senegal, the Council for Scientific and Industrial Research in South Africa, the Oswaldo Cruz Foundation in Brazil, the Translational Health Science and Technology Institute in India, Egypt’s Center for Continuing Professional Development, and Peking University in China.

    The initiative directly supports World Health Assembly resolution WHA74.6, which called on member states to strengthen local production of medicines and other health technologies to prepare for emergencies.

    This will be a focus of NIBRT’s training activities, according to O’Driscoll.

    “One of the key actions the WHO identified following the COVID-19 pandemic was to increase biopharma manufacturing capabilities within lower-middle-income countries (LMICs). The WHO’s Biomanufacturing Workforce Training Initiative addresses critical skills gaps in the biomanufacturing value chain to support sustainable local production of vaccines and biotherapeutics in LMICs,” he says.

    In a press statement, director-general, Tedros Adhanom Ghebreyesus, PhD, said, “We have designated regional training centers in each of WHO’s six regions to build the skilled workforce needed to sustain local production of vaccines and biologics. They will operate as part of a coordinated global network, delivering context-specific training aligned with regional priorities, regulatory environments, and languages.”

    The post WHO Selects NIBRT as Training Hub to Help LMICs Build Biopharma Capacity appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Scaling Stem-Cell Manufacturing for Therapies https://edusehat.com/en/scaling-stem-cell-manufacturing-for-therapies https://edusehat.com/en/scaling-stem-cell-manufacturing-for-therapies Thu, 25 Jun 2026 00:30:31 +0700 Edusehat Scaling, Stem-Cell, Manufacturing, for, Therapies Human pluripotent stem cells (hPSCs) have long been viewed as one of regenerative medicine’s most promising raw materials. Now, as more than 100 clinical trials evaluate hPSC-derived therapies for diseases ranging from Parkinson’s disease to heart failure and type 1 diabetes, attention is turning toward a crucial challenge: how to manufacture these cells reliably and economically at industrial scale.

    According to Kevin Cyrys and Robert Zweigerdt, PhD, both of Hannover Medical School in Germany, the field has entered a new phase. Rather than simply demonstrating that stem cells can be grown in bioreactors, researchers are increasingly focused on creating robust production platforms that can deliver consistent quality across facilities and patient populations.

    “Human pluripotent stem cells can serve as an unlimited, renewable ‘raw material’ for essentially any therapeutic cell product,” the authors wrote, highlighting the technology’s potential to overcome limitations associated with donor-derived tissues and organs.

    The manufacturing challenge is substantial. While some therapies, such as treatments for age-related macular degeneration, require only tens of thousands of cells per dose, others may demand billions of cells for a single patient treatment. Conventional laboratory-scale methods are unlikely to meet such requirements efficiently.

    To address this gap, developers are increasingly adopting three-dimensional suspension cultures in bioreactors. Compared with traditional two-dimensional cell culture systems, bioreactors provide tighter control over temperature, oxygen levels, pH, and carbon dioxide while supporting automated, closed-system manufacturing compatible with good manufacturing practice (GMP) standards.

    The field has already demonstrated notable progress across multiple therapeutic areas. Researchers have developed scalable processes for producing cardiomyocytes, pancreatic islet cells, hepatocyte-like cells, neural tissues, and immune effectors derived from hPSCs. Some cardiac manufacturing platforms have reported production of billions of cardiomyocytes in liter-scale bioreactors, while immune-cell manufacturing programs have successfully expanded induced pluripotent stem cell-derived natural killer cells in 1–10 L systems while maintaining product quality.

    Yet scaling production involves more than increasing cell yields. “Industrial-scale success depends on more than headline totals,” Cyrys and Zweigerdt note, citing the importance of volumetric productivity, production time, reproducibility, and integration of expansion, differentiation, and downstream processing into a coherent GMP-ready workflow.

    Looking ahead, Cyrys and Zweigerdt argue that the next generation of stem-cell manufacturing will be defined by data-driven process control. They predict that AI-enabled systems will help move the industry from retrospective quality analysis toward real-time decision support, ultimately improving comparability between batches and strengthening product definitions across manufacturing networks.

    Despite ongoing challenges involving cost, quality control, and regulatory compliance, the authors conclude that stem-cell bioprocessing has already crossed an important threshold. Scalable culture systems are no longer the primary obstacle. Instead, the focus has shifted toward engineering reliable industrial processes capable of transforming complex stem-cell biology into reproducible therapeutic products.

    The post Scaling Stem-Cell Manufacturing for Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Recoded E. coli Promises More Scalable Weight Loss Drug Production https://edusehat.com/en/recoded-e-coli-promises-more-scalable-weight-loss-drug-production https://edusehat.com/en/recoded-e-coli-promises-more-scalable-weight-loss-drug-production Thu, 25 Jun 2026 00:30:20 +0700 Edusehat Recoded, coli, Promises, More, Scalable, Weight, Loss, Drug, Production The manufacturing of weight loss drugs at large scale could get cheaper and more sustainable thanks to an engineered strain of Escherichia coli (E. coli) bacteria.

    The fully recoded E. coli, designed to use only 61 codons to synthesize proteins, is now being rolled out as a new method for manufacturing peptides with non-natural chemistries.

    That’s according to Constructive Bio, the company that recoded the E.coli and now hopes this synthetic strain will transform the production of some high-volume hard-to-manufacture protein/peptide therapeutics.

    “Our key message is that we’re able to produce long peptides containing non-canonical amino acids to deliver therapeutic proteins at scale by biomanufacturing,” explains Rob Salmon, PhD, head of bioprocess at Constructive Bio.

    “And our key differentiator is there’s currently a market in, for example, weight loss drugs.”

    According to Salmon, glucagon-like peptide-1 (GLP-1) agonists for weight loss are currently produced using chemical synthesis approaches such as solid phase peptide synthesis, which is hard to scale and generates high volumes of toxic waste.

    By contrast, the synthetic E. coli strain can potentially produce these peptides using fermentation via standardized industrial processes, he says.

    “We want to fit into standardized industrial unit operations and, through that, scale to thousands of liters of product that we can sell to the market,” he explains.

    The strain was developed as part of research into reducing the number of codons needed to synthesize proteins in an organism from 64 to 61, allowing slots for three new non-canonical amino acids, according to the company.

    A schematic demonstrating how non-canonical amino acids are incorporated into a protein or peptide chain using the ribosome in Constructive Bio’s Syn61 strain of E. coli. [Constructive Bio]
    Constructive Bio was founded in 2022 to take the strain forward into industrial applications, including optimizing for applications such as antibody fragments or the long peptides used for GLP-1 agonist therapies.

    Since then, the optimized strain has been taken through some industrial fermentations and demonstrated promising titers, he explains, adding that he will present results at the upcoming Bioprocessing Summit in Boston.

    “We’re challenging some of the assumptions from chemists that biology can’t be used to do this,” he says.

    The post Recoded E. coli Promises More Scalable Weight Loss Drug Production appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Novel Feeder Cell Line Dramatically Expands NK Cell Production https://edusehat.com/en/novel-feeder-cell-line-dramatically-expands-nk-cell-production https://edusehat.com/en/novel-feeder-cell-line-dramatically-expands-nk-cell-production Thu, 25 Jun 2026 00:30:09 +0700 Edusehat Novel, Feeder, Cell, Line, Dramatically, Expands, Cell, Production Allogeneic natural killer (NK) cells appear promising as an adoptive cell therapy (ACT) that targets cancer. They’re limited, however, by production methods that can’t readily produce these cells in therapeutically relevant quantities.

    Researchers led by Sang-Ki Kim, DVM, PhD, professor, Kongju National University in Korea, and CSO at Vaxcell Bio, along with Seung-Hwan Lee, PhD, professor, University of Ottawa, appear to have solved that bottleneck with an engineered version of the feeder cell line known as ARH-77, a B-lymphoblast cell line that stimulates NK cells. Even in its unmodified form, ARH-77 cells expanded NK cells extracted from peripheral blood samples 681-fold after 28 days. In contrast, K562, the cell line typically used, enabled 155-fold expansion during that time.

    That expansion pales in comparison to that of the engineered cell line. The now-modified ARH-77 cells, modified to express four specific stimulatory ligands, expanded NK cells by 101,241-fold in 28 days. Making the same modifications to the K562 cells, however, improved production only 4.4-fold. In each of the cell lines, purity and cytotoxicity were considered equivalent.

    Kim, Lee, and colleagues chose the ligands B7-H6, CD137L, IL-15, and IL-15Rα to provide multi-axis stimulation to enhance NK cell activation and proliferation as well as to enhance persistence. For example, B7-H6 stimulates production and exhibits early cytotoxic benefits, but those benefits dissipated by week four. CD137L appears to compensate for that attenuation, the scientists report. Notably, the feeder performance was consistent across donors.

    While these ligands were more effective than other ligands the team considered, they stress that more work is needed to “formally establish the added value of each ligand.” They also want to evaluate the engineered ARH-77 in terms of in vivo persistence and anti-tumor activity against additional models. Large-scale manufacturing constraints also should be considered in future studies.

    Because feeder cell performance is considered stable across the donor population, Kim and Lee suggest their engineered ARH-77 cell line may be a reliable option for NK cell expansion as therapeutic production scales up. As the scientists note, “These findings establish ARH-77 as a promising alternative feeder cell platform that could enhance the scalability, consistency, and potency of allogeneic NK cell manufacturing for clinical adoptive immunotherapy.”

     

    The post Novel Feeder Cell Line Dramatically Expands NK Cell Production appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Biotech at 50: Can the innovation ecosystem deliver the next generation of breakthroughs? https://edusehat.com/en/biotech-at-50-can-the-innovation-ecosystem-deliver-the-next-generation-of-breakthroughs https://edusehat.com/en/biotech-at-50-can-the-innovation-ecosystem-deliver-the-next-generation-of-breakthroughs Wed, 24 Jun 2026 21:00:04 +0700 Edusehat Biotech, 50:, Can, the, innovation, ecosystem, deliver, the, next, generation, breakthroughs Biotechnology’s first 50 years were built on a uniquely American formula: world-class universities, federal research funding, venture capital investment, and partnerships between academia and industry.

    The question facing industry leaders at the 2026 BIO International Convention is whether that formula can continue to deliver breakthroughs for the next 50 years.

    That was the focus of Tuesday’s super session, The Innovation Mandate: Strengthening the Biopharma Ecosystem for the Next Generation, sponsored by Genentech and featuring leaders from government, industry, and academia.

    The biotechnology industry traces its origins to such a partnership. In 1976, venture capitalist Robert Swanson and University of California, San Francisco scientist Herbert Boyer joined forces to found Genentech, considered the world’s first biotechnology company.

    “That’s what makes our system in America so special,” said Fritz Bittenbender, Senior Vice President of Public Affairs and Access at Genentech.

    But speakers warned that the ecosystem responsible for decades of scientific progress faces growing challenges, from uncertainty surrounding federal research funding to increasing global competition for talent, capital, and innovation.

    “When we see budget cuts happening at the NIH, that actually has a horribly negative effect on our country’s ability to be able to innovate in the future,” because it leads to an exodus of talent, research, and science, explained Bittenbender

    “Capital is fungible,” said Andrew Lam, PharmD, Managing Director and Head of Biotech Private Equity at the Ally Bridge Group. “It will seek out the best innovation around the world.”

    “We have to have science continue to dictate where the funding should go,” Bittenbender added.

    How do we maintain American competitiveness in a global ecosystem?

    “The world has changed dramatically,” said Lam. “In fact, the velocity of innovation is only as solid as the protection mechanisms and investment we put into it, and because of that, we need to think more globally. How do we continue to maintain our lead in terms of our very vibrant ecosystem within biopharma?”

    China’s rise in the biotech industry is something to watch – and perhaps learn from.

    “China is spending a trillion dollars right now on research,” said Bittenbender. “They have a five-year plan to dominate the bioscience industry, and it’s a very comprehensive plan. What we are missing from a policy perspective in the United States is a holistic, comprehensive plan.”

    To maintain America’s competitive advantage, we need robust institutions, and the federal government must support them.

    “I think there’s a misperception that if we cut back on NIH funding, other types of private investment dollars are going to step in and fill that void, and that’s simply not true,” explained Erin Trish, Ph.D., Co-Director at the USC Schaeffer Center for Health Policy & Economics. “What the research shows is that public and private funding are complements, not substitutes.”

    Throughout the discussion, speakers returned to a common theme: scientific breakthroughs do not happen in isolation. They emerge from an ecosystem that connects researchers, universities, investors, companies, regulators, and policymakers. The challenge now is ensuring that the ecosystem remains strong enough to support the next generation of discoveries.

    “What are the next 50 years going to be?” Bittenbender asked. “What are the new treatments? What are the new modalities? And how can government be thinking about that?”

    The post Biotech at 50: Can the innovation ecosystem deliver the next generation of breakthroughs? appeared first on Bio.News.

    ]]>     California Still Golden Despite Job Losses: Industry Group https://edusehat.com/en/california-still-golden-despite-job-losses-industry-group https://edusehat.com/en/california-still-golden-despite-job-losses-industry-group Wed, 24 Jun 2026 20:55:08 +0700 Edusehat California, Still, Golden, Despite, Job, Losses:, Industry, Group SAN DIEGO—California’s three life sciences clusters all lost jobs last year, yet the industry remains a major engine of innovation and economic growth, according to a report released by the state’s largest life sciences organization to coincide with the Biotechnology Industry Organization (BIO) International Convention being held here.

    BIOCOM California quantified the economic impact of the Golden State’s life sciences industry as generating $394 billion in economic output in 2025—a figure that goes beyond the direct impact of the 406,505 people employed by life sciences employers across the state. The impact figure includes indirect impact (activity generated through suppliers, vendors, and subcontractors supporting the industry) and induced impact (the household spending generated by workers employed in both life-sci organizations and supporting industries).

    When indirect and induced impact are accounted for, the life sciences sustain 1,079,365 jobs statewide, the report stated.

    However, all three of the state’s top-tier life-sci clusters—the San Francisco Bay Area, San Diego, and the Los Angeles/Orange County region—saw decreases in employment within the industry last year, according to the report.

    San Francisco ranks second in the latest edition of GEN’s nationally-quoted A-List of Top 10 U.S. Biopharma Clusters, unchanged from a year ago, while San Diego slid one position to sixth, and LA/Orange County slipped one notch to eighth.

    “Continued biotech winter”

    “California, like the other states in the country, are still showing the effects of the pandemic and the recovery from that, because it was such a large run up of investment and hiring and building of new space, followed by a pretty significant drop off in 2022, 23,” Tim Scott BIOCOM California’s president and CEO, explained in an interview with GEN conducted at the organization’s booth within the convention’s exhibition floor.

    “And then we have the continued biotech winter that’s been caused mostly through the instability at the federal level in terms of policy,” Scott added.

    He cited NIH funding cuts, the delay in re-authorizing the Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) seed funding programs, tariffs, and the “most favorite nation” drug pricing framework championed by the Trump administration as a vehicle for lowering drug prices: “All of these things have led the industry and the investors in the industry to pause.”

    Most of the life-sci job decline was concentrated in the Bay Area and San Diego regions, which together accounted for 88% of job losses.

    The San Francisco Bay Area saw its life-sci workforce slide 2.7% from 2024 to 137,779 jobs last year, driven mainly by decreasing employment in scientific/research tools, biotechnology, and biopharmaceuticals. The San Diego region finished 2025 with 61,866 jobs, a 2.55% decline from the previous year, due primarily to the loss of jobs in the R&D in physical, engineering, and life sciences and electromedical and electrotherapeutic apparatus manufacturing sectors.

    Greater Los Angeles, which BIOCOM California defines as Los Angeles, San Bernardino, and Ventura counties, saw its employment base shrink 0.5% year-over-year, to 143,153 last year, with the largest employment decrease coming in drug wholesaler positions. Orange County’s life-sci workforce also dipped by 0.5%, sliding to 57,213 jobs, driven by cuts in scientific/research tools and medical devices and equipment employment—though Orange County also saw increases in biotechnology and research and testing jobs.

    “Significant driver”

    “In spite of the slight decrease in growth this year, we’re still at about $400 billion in economic output for California in the life sciences. That’s the second largest industry in California,” Scott said. “It’s still a significant driver of economic activity and of innovation.”

    Another driver of innovation, NIH funding, stayed flat last year compared to 2024 at $5.23 billion for all of California. But the number of NIH awards statewide fell 8.5% from 9,384 in 2024 to 8,587 in 2025.

    Life science manufacturing jobs fell by 2.1% last year to 143,572 jobs, though they still accounted for more than one-third (35.3%) of all of the industry jobs in the state. Across 31 life science industry sub-sectors, 23 recorded job losses, with the largest declines in medical laboratories and R&D within the physical, engineering, and life sciences job category.

    But the state’s life-sci manufacturing segment is eventually expected to grow as drug developers either strive to meet growing demand, reshore their production in the United States to avoid tariffs, or both. Gilead Sciences began construction in September 2025 of a new 180,000 square-foot development and manufacturing facility, part of a companywide $32 billion U.S. investment strategy. Two months later, Novartis opened a 10,000-square-foot radioligand therapy (RLT) manufacturing facility for cancer treatments in Carlsbad, CA, the pharma giant’s third U.S.-based RLT site.

    While biopharmas and contract manufacturers have announced hundreds of billions of dollars in new projects, projects announced for California remain mostly under construction, so hiring levels have not yet risen to account for the new manufacturing activity, Scott said.

    Potential challenges loom

    Two more potential challenges loom for California life science companies—one from Washington, the other from Sacramento.

    Scott said BIOCOM California is paying attention to federal efforts aimed at further scrutinizing activity between U.S. and Chinese biopharmas.

    Earlier this month, Reps. John Moolenaar (R-MI), chairman of the Select Committee on China, and Congresswoman Debbie Dingell (D-MI), introduced the Biotech Investment National Security Act (BINSA). BINSA would amend the Comprehensive Outbound Investment National Security (COINS) Act, enacted last year, by adding pharmaceutical and biological product development to the list of sectors subject to screening of investments by the U.S. government.

    The measure would subject U.S. pharmaceutical licensing deals, joint ventures, and equity investments with Chinese covered foreign persons to U.S. Treasury Department review, as well as explicitly cover licensing deals involving technology and intellectual property. BINSA also requires the Secretary of War (formerly Defense) to assess within 60 days whether U.S. capital investment in Chinese biotechnology negatively affects national security and military readiness.

    “We’re trying to find the balance between protecting American interests with regard to intellectual property and also competing with China. And we’re balancing that with cooperating with China,” Scott said. “You can imagine a politician in Washington, D.C., wants to really protect our interests. A biotech entrepreneur in California wants to go anywhere in the world to find resources to be able to move their drug toward the clinic.”

    In Sacramento, Gov. Gavin Newsom, who leaves office at year’s end when his second term expires, has proposed permanently limiting the amount of business tax credits that a corporation can claim each year. Starting in 2027, corporate taxpayers would be allowed to claim a maximum of either $5 million or 50% of their pre-credit tax liability, whichever is greater. The limit would not affect taxpayers with less than $5 million in credits.

    According to California’s Legislative Analyst’s Office (LAO), recent tax collection data shows that fewer than 100 corporate taxpayers in California would be affected. LAO has estimated that the proposal would raise $850 million in 2026–27, since the cap would only apply to part of the fiscal year, and $1.7 billion to $1.8 billion annually between 2027–28 and 2029–30.

    However, the R&D credit likely accounts for most of the proposal’s fiscal effect, according to the LAO, since the R&D credit accounts for the overwhelming majority of business credit usage and carry-forward balances. And the roughly 100 affected businesses include many of the largest biopharma giants, Scott said.

    “That is a really big tool for engaging pharma and encouraging investment in California. Without the R&D tax credit, companies are less likely to want to invest in California,” Scott asserted. “The R&D tax credit has had a direct effect on driving the growth of the biotech industry in California.”

    The post California Still Golden Despite Job Losses: Industry Group appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Gene Editing Pioneer Sangamo Files for Chapter 11 Bankruptcy; Agrees to Sell Assets https://edusehat.com/en/gene-editing-pioneer-sangamo-files-for-chapter-11-bankruptcy-agrees-to-sell-assets https://edusehat.com/en/gene-editing-pioneer-sangamo-files-for-chapter-11-bankruptcy-agrees-to-sell-assets Wed, 24 Jun 2026 13:45:08 +0700 Edusehat Gene, Editing, Pioneer, Sangamo, Files, for, Chapter, Bankruptcy, Agrees, Sell, Assets A journey that has lasted more than 30 years for Sangamo Therapeutics, a pioneering gene editing biotech company in the Bay Area, has reached an unwanted milestone as the company filed for Chapter 11 bankruptcy protection.

    Concurrent with its starting voluntary Chapter 11 proceedings in the U.S. Bankruptcy Court for the District of Delaware, Sangamo simultaneously entered into two separate asset sale agreements: Eli Lilly has agreed to acquire Sangamo’s capsid delivery platform, zinc finger nuclease (ZFN) platform, modular integrase (MINT) platform, and prion disease program, ST-506. Astellas Pharma has agreed to take over Sangamo’s Fabry disease program, isaralgagene civaparvovec (ST-920). 

    To clinch the deals, Lilly and Astellas have agreed to be “stalking horse” bidders when Sangamo’s assets are sold in a future bankruptcy court auction. The stalking horse bids do not include the clinical-stage ST-503 program to treat chronic neuropathic pain, the giroctocogene fitelparvovec program to treat hemophilia A, and Sangamo’s cell therapy and regulatory T cell (Treg) assets. Sangamo said these are expected to remain available to interested bidders at the auction.

    “We believe this process provides a clear framework to pursue value-maximizing transactions,” said Sandy Macrae, Sangamo’s CEO. “Our priority is to execute a disciplined and efficient sale process while supporting all of our stakeholders. We are also pleased to have signed agreements with two large pharmaceutical companies to serve as stalking horse bidders in the process, underscoring the strategic interest in our assets.”

    Ed Lanphier, founder, Sangamo Therapeutics

    Founded by Ed Lanphier in 1995, Sangamo became an early developer of zinc-finger nucleases (ZFNs), one of the first established gene editing platforms. In 2005, Sangamo scientists led by Fyodor Urnov, PhD, Phil Gregory, PhD, and Mike Holmes, PhD, demonstrated the use of ZFNs to engineer a base substitution in human DNA. The term “genome editing” was born around that report. Sangamo’s technology became the first gene editing platform to enter the clinic, initially for patients with human immunodeficiency virus (HIV), followed by a series of rare genetic diseases. 

    More recently, the biotech branded itself as a “genomic medicine” company. In 2023, Sangamo trumpeted promising clinical data from its first-in-human Phase I/II STAAR trial (NCT04046224) in Fabry disease. All 25 patients dosed in the STAAR study have continued to show sustained, elevated α-Gal A levels, up to three years for the longest-treated patient. However, later that year, Sangamo deferred additional spending on planning a future Phase III program for ST-920, absent a collaboration partner or additional external funding.  

    Sangamo made the move as part of a restructuring that included a similar deferral of spending on chimeric antigen receptor-modified regulatory T-cell (CAR-Treg) therapies, the elimination of 40% of its U.S. workforce, and the narrowing of its pipeline. Sangamo said it was refocusing its spending on developing epigenetic regulation therapies treating neurological diseases, as well as novel adeno-associated virus (AAV) capsid delivery technologies. 

    In 2024, Sangamo shares surged 69% after it reached alignment with the FDA on a regulatory pathway to Accelerated Approval for ST-920 in advance of submitting a biologics license application (pre-BLA). However, Dennis Ding, an equity analyst with Jefferies, argued that the news posed little threat to the developer of the sole marketed drug for the rare disorder, Galafold® (migalastat), marketed by Amicus Therapeutics.  

    Last month, Sangamo said it remained in the process of completing a rolling BLA submission to the FDA for Accelerated Approval of ST-920 based on the mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study. Two-year eGFR data may serve as confirmatory evidence for traditional approval, Sangamo said the FDA affirmed. 

    Sangamo was also advancing the Chemistry, Manufacturing and Controls (CMC) module, ahead of completion of the rolling BLA submission for ST-920, which the company said it expected this summer (subject to the ability to secure adequate additional funding), while it was continuing to commercialize the Fabry gene therapy. 

    In reporting first-quarter results, Sangamo said that it had submitted preclinical and clinical modules for review, while also submitting its antibody assay companion diagnostic, designed to screen patients for eligibility with ST-920, to the FDA’s Center for Devices and Radiological Health (CDRH).  

    Sangamo reported a $31-million net loss on revenue that plunged 78% year over year to $1.4 million from $6.4 million. Sangamo said $5 million of that decrease reflected Pfizer’s termination early last year of its collaboration with Sangamo to develop a hemophilia A gene therapy, giroctocogene fitelparvovec.  

    The termination occurred six months after Sangamo and Pfizer partnered to report positive Phase III data for giroctocogene fitelparvovec. The gene therapy met its primary endpoint in the Phase III AFFINE trial (NCT04370054) compared with Factor VIII (FVIII) replacement. 

    Sliding doors 

    Speaking several years ago with The CRISPR Journal, a peer-reviewed journal and sister publication of GEN, Sangamo founder Edward Lanphier reflected on the company’s bright beginnings. In 1994–95, he recalled, he became aware of research being done by Jeremy Berg, PhD, and Srinivasan Chandrasegaran, PhD, on engineering zinc finger proteins (ZFPs).  

    A watt-hour meter, an electric usage measuring device designed and patented by the great-grandfather of Sangamo Therapeutics founder Ed Lanphier

    “While it was certainly unclear what making novel DNA-binding proteins might do, novel DNA sequences represented the other half of this equation—an agnostic vector plus a platform for developing novel transgenes. I became quite interested in that, and thus in starting Sangamo,” Lanphier remembered.

    After founding Sangamo in 1995, Lanphier joined the company full-time two years later. Sangamo’s name was derived from some fascinating family history—Lanphier’s great-grandfather, a Yale-educated electrical engineer, founded a company in Sangamon County, IL, during the 1890s. 

    He designed and patented “the watt hour meter—the thing that sits on the side of buildings and goes around and around recording electricity,” Lanphier recalled. The Sangamo Electric Company manufactured various electronic components before being sold in the 1970s to Schlumberger.

    Lanphier remembered “this incredibly cool logo from Sangamo Electric. I asked my dad, ‘‘What do you think?’’ He said, ‘‘That would be great!’’ And so, I started Sangamo Biosciences.” 

    While ZFNs showed immense promise as a commercial gene editing platform, they were difficult and expensive to manufacture. The dramatic arrival of CRISPR in 2012–13 quickly pushed ZFNs onto the fringes of the clinical gene editing space. 

    “When the [gene editing] movie is written, I know it is going to focus exclusively on the Broad and Berkeley and Charpentier and their work. But it is completely unfair—not to me but to Fyodor and Ed [Rebar] and Philip and Mike Holmes and Jeff Miller and the dozens of people who did create this field.” 

    Lanphier was asked why Sangamo never joined the CRISPR revolution a decade ago. “My perspective was always that [CRISPR] is bacterial—it is nonspecific, it is immunogenic. It’s a great research tool. It’s going to give a lot of visibility to genome editing. When people actually want to use it therapeutically, that’s when they will end up talking to us.”  

    Alas for Sangamo, that eventuality did not materialize.

    The post Gene Editing Pioneer Sangamo Files for Chapter 11 Bankruptcy; Agrees to Sell Assets appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO 2026: As capital returns, focus on quality over quantity https://edusehat.com/en/bio-2026-as-capital-returns-focus-on-quality-over-quantity https://edusehat.com/en/bio-2026-as-capital-returns-focus-on-quality-over-quantity Wed, 24 Jun 2026 10:15:04 +0700 Edusehat BIO, 2026:, capital, returns, focus, quality, over, quantity “Last year was a very business-as-usual year,” said Daniel Chancellor, VP of Thought Leadership at Norstella, “and has been followed by a year that was totally anything but.”

    His comments came during the presentation of a new report, The State of Emerging Biotech: Investment, Deals, and Pipelines Report, produced by the Biotechnology Innovation Organization (BIO) and Norstella and released at the 2026 BIO International Convention on June 23.

    Despite the changes, the industry has produced 58 novel approved therapeutics, tracking with the long-term average of 60 per year.

    While last year’s report found the biotech industry acting bearishly during the post-2021 slump, it is no longer in recovery mode. Investors are concentrating resources on later-stage, clinically validated assets over preclinical drugs. Capital has returned, dealmaking is strong, and pipelines are maturing. With more R&D funding directed toward fewer drugs, the industry is prioritizing efficiency above all else.

    How efficiency is reshaping the biopharma pipeline

    That trend is reshaping the entire industry – in the U.S. and worldwide.

    “The pipeline contracted last year, and it wasn’t just a small contraction,” said Chancellor. “It was a meaningful 4 percent decline in the number of drugs that the industry as a whole is developing. This includes U.S. companies, Chinese companies, European countries, everywhere globally, and also preclinical stage assets.”

    The global biopharma R&D pipeline hit 22,940 for new drugs under development in 2026. This number reflects a 3.9% decline in the pipeline – the first time in 15 years – despite a 45% year-over-year increase in R&D funding.

    But this isn’t necessarily bad news.

    Biopharma companies are playing a short game to play a long game, shifting support to drugs beyond the preclinical phase. Preclinical programs fell 14%, while Phase II and Phase III programs grew roughly 9%.

    Accordingly, biopharma companies killed off more new drugs than ever before, introducing killer tests earlier in the pipeline to weed out drugs not showing promise.

    They are also spending more time on each drug. The median timeline from Phase I testing to FDA approval is now 9.3 years, which has increased by one month over the past five years.

    In return, they are being rewarded with higher success rates in late-stage trials among their more developed drugs, which are receiving more funding.

    “More of the later-stage companies are very successful in progressing, because there’s been a lot of focus – from both large pharma and from the investors – on later-stage products to refill the pipelines of the larger companies,” said Chad Wessel, Senior Director, Emerging Companies Special Initiatives at BIO.

    While early-stage companies are not necessarily out of the game, they have to be smart.

    “Early-stage companies need to be nimble and flexible,” he said.

    Drug programs maintain established trends

    Despite shifting priorities in the pipeline, trends in the types of drugs prioritized are relatively stable. Oncology drugs, for example, now account for 47% of drugs in development, a 3% increase from 2024.

    For comparison, neurological drugs occupy a remote second place at 10% of drugs in development.

    A substantial minority of drugs produced for oncology and neurology address rare diseases. Since 70% of drugs for both disease categories are produced by emerging biotechs, this may reflect these smaller companies attempting to find a niche within the industry to differentiate themselves from their many competitors.

    VC is driving emerging biotechs

    Emerging biotechs remain the industry’s innovation engine. New companies account for 41 of 58 FDA novel drug approvals in 2025, roughly 71% of all FDA approvals. They’re now developing 72% of the U.S. clinical pipeline, up from 55% a year ago.

    While venture capital sees this trend, they are favoring those with demonstrated potential.

    In 2025, venture capital investment reached its highest point since the COVID pandemic at $23 billion, a 23% increase over the previous year. During that same period, clinical-stage funding increased by 55%, while preclinical funding declined by 17%.

    Notably, from a 2023 lull of 42 rounds, U.S. $100M+ financings have climbed two years running – to 57 in 2024 and 65 in 2025, a 55% recovery. A record 22 Series A mega-rounds in 2025 show the rebound is led at the earliest stage, as reported by BIO.

    The number of VC deals with companies in clinical-stage rounds jumped 37%, the second year in a row this figure exceeded deals with companies in pre-clinical rounds.

    What does this mean? Capital is available, but investors increasingly want human clinical data before writing large checks.

    The impact of layoffs

    The emphasis on efficiency also extends to workforce practices. Layoff announcements in the first three quarters of 2025 were among the highest in the post-pandemic era at 65, 61, and 60, respectively, before falling sharply to 37 in Q4. Employees at small-to-mid companies were equally likely to be laid off as their counterparts in large companies.

    But conversations at the 2026 BIO Convention have proven hopeful, as many early-stage companies are starting to ramp up hiring in an eager venture market.

    “There is a lot of traction in the landscape for small emerging companies,” concluded Wessel. “So long as they have good science and they’re able to progress.”

    The post BIO 2026: As capital returns, focus on quality over quantity appeared first on Bio.News.

    ]]>     BIO 2026: Gary Sinise, Marcus Freeman on leadership, service, and resilience https://edusehat.com/en/bio-2026-gary-sinise-marcus-freeman-on-leadership-service-and-resilience https://edusehat.com/en/bio-2026-gary-sinise-marcus-freeman-on-leadership-service-and-resilience Wed, 24 Jun 2026 10:15:03 +0700 Edusehat BIO, 2026:, Gary, Sinise, Marcus, Freeman, leadership, service, and, resilience Leadership is often defined by how people respond to adversity.

    That theme ran through the 2026 Biotechnology Innovation Organization (BIO) International Convention main stage on Tuesday, where actor and philanthropist Gary Sinise, Notre Dame head football coach Marcus Freeman, and BIO President and CEO John F. Crowley shared stories of setbacks, sacrifice, and resilience.

    Though they come from different backgrounds and life experiences, each arrived at a similar conclusion: progress often comes from confronting challenges head-on.

    Crowley opened the morning by reflecting on the founding of Genentech in 1976 and the work that remains for patients still waiting for new treatments.

    “Before the biotechnology revolution, triple-negative breast cancer had a five-year survival rate of essentially zero. It was an unmitigated death sentence,” he said. “Today, its five-year survival rate is nearly 80%.” And similar statistics can be given for a host of other diseases, from melanoma and myeloma to HIV/AIDS and sickle cell disease.

    “And soon it will be time, and each of us in this room at this convention, after a few days, to go back to our labs and offices and to try to do it even better and faster, because time itself is so very precious.”

    ‘The greatest lessons come from failure’

    Notre Dame head football coach Marcus Freeman at the 2026 BIO International Convention
    Notre Dame head football coach Marcus Freeman at the 2026 BIO International Convention

    That sense of urgency framed the conversation with Freeman, who looked back on some of the most difficult moments of his tenure leading the Notre Dame football program.

    After taking over as head coach in 2021, Freeman endured several painful early losses that prompted him to reexamine his leadership.

    “I started to question myself.”

    Yet Freeman said those experiences ultimately shaped him more than any victory.

    “The greatest lessons come from failure, because those are inside lessons.”

    He learned that leaders are allowed to have doubts, but they cannot lead with them. While he could sit in his office questioning what had gone wrong, he said the team needed a coach who could walk into the room and say, “Here’s what we got to do. Here’s what we got to fix.”

    And, Freeman said, success is ultimately measured not by wins and losses, but by the impact leaders have on others.

    “Success is about who you become on the inside as you continue to rise on this journey of life,” he said, “and if on the inside you truly care about helping others and lifting as you climb, man, you’re going to impact a lot more people than if you just worry about how high you climbed by yourself.”

    Meeting the challenge of a life-changing diagnosis

    The focus on service and purpose continued during Crowley’s conversation with Sinise, whose decades-long support for veterans eventually led to the creation of the Gary Sinise Foundation.

    Following the September 11 attacks and the wars in Afghanistan and Iraq, Sinise became deeply involved in supporting service members and military families, “trying to help them raise awareness, raise more funding to provide more services.”

    The same commitment to perseverance would later be tested much closer to home.

    In 2018, Sinise’s son Mac was diagnosed with chordoma, a rare cancer that affects roughly 300 Americans each year. After months of unexplained pain—the family thought it was from a bike accident—doctors finally discovered the source of the problem.

    “The spine surgeon put him in the CT scanner and saw an orange-sized tumor on his sacrum. And that changed—that changed everything.”

    Over the next several years, Mac endured surgeries, chemotherapy, radiation treatments, and increasing physical limitations over the next five and a half years.

    “He was a tremendous musician and composer,” Sinise said. “But he wasn’t thinking about music for the longest time because of the cancer fight.”

    At the beginning of 2023, after years of treatments and hospital visits, Mac told his father he had been thinking about a piece of music he had started in college but never finished.

    “That was just music to my ears. I thought this was fantastic,” Sinise recalled.

    Mac partnered with several musicians, including members of Sinise’s own band, to complete the composition, “Arctic Circles.” The project reignited his creative ambitions and eventually grew into a full album, Resurrection and Revival, which he completed in late 2023 just weeks before his death.

    Afterward, Sinise discovered additional unfinished music on his son’s computer and worked to bring it to life, resulting in two additional albums built from Mac’s unreleased compositions.

    The experience inspired Sinise’s upcoming memoir, Graceful Warrior: A True Story of a Son, a Father, and a Family Who Carried Each Other Through, which is scheduled for release on Nov. 10.

    “Like so many, you know, we all go through terrible things. It’s just human nature. And what happens in life. But I think we all learn things from each other when we watch how somebody can meet challenges and adapt and then overcome.”

    For an industry built on tackling some of the world’s most difficult scientific and medical challenges, it was a message that resonated throughout BIO 2026.

    The post BIO 2026: Gary Sinise, Marcus Freeman on leadership, service, and resilience appeared first on Bio.News.

    ]]>     BIO 2026: Biotech dealmakers navigate uncertainty, pursue opportunities https://edusehat.com/en/bio-2026-biotech-dealmakers-navigate-uncertainty-pursue-opportunities https://edusehat.com/en/bio-2026-biotech-dealmakers-navigate-uncertainty-pursue-opportunities Wed, 24 Jun 2026 03:05:03 +0700 Edusehat BIO, 2026:, Biotech, dealmakers, navigate, uncertainty, pursue, opportunities From billion-dollar acquisitions to early-stage collaborations, biotech dealmaking is showing renewed momentum despite an increasingly complex policy and regulatory environment.

    On Day 1 of the 2026 Biotechnology Innovation Organization (BIO) International Convention in San Diego, two sessions examined both sides of the equation: how companies are navigating uncertainty in today’s market, and how smart business development can create opportunity even in challenging conditions.

    That focus on biotech dealmaking is one reason thousands of executives, investors, and business development leaders have gathered in San Diego this week. While policy uncertainty dominated many conversations, speakers repeatedly emphasized that partnerships, licensing agreements, acquisitions, and strategic collaborations remain the engine that moves innovation from the lab to patients.

    And despite uncertainty, deal activity remains robust.

    Anamaria Sudarov, Ph.D., Managing Director at Wells Fargo, pointed to a broad range of transactions taking shape across the industry.

    “What that represents is what we have seen, really frankly – starting in Q4 2025 and in the first two Qs of 2026 – an incredibly broad spectrum of the type of transactions and deals getting done,” she said.

    Those transactions range from targeted discovery collaborations to multi-billion-dollar acquisitions. However, the most active area remains bolt-on transactions in the $1–5 billion range, where acquirers are seeking deals rooted in specific assets, particularly those with mid-stage clinical data, noted Sudarov.

    The challenge is that biotech dealmaking today must account for risks that are difficult to quantify.

    Asked how geopolitical and policy uncertainty is being incorporated into transactions, Chad Diehl, J.D., Legal Team Lead, Licensing & Acquisitions and Alliance Management at Astellas Pharma, said companies are wrestling with variables that are impossible to model with certainty. Buyers still want to do deals and have tools to bridge valuation gaps, but both sides need to recognize that significant uncertainty could materially affect the economics of a transaction.

    “Because it’s an unknown, it’s not flowing through to the financial valuation that’s on paper,” said Diehl. “What the market is today, it wasn’t 10 years ago. And it’s not what it’s going to be in five years.”

    How biotech companies can seize dealmaking opportunities

    But uncertainty has not eliminated opportunity.

    Speaking to Bio.News after her session, Casarine Chong, J.D., MBA, General Counsel, R&D, Business Development and Strategy at CSL, emphasized the importance of adaptability.

    “For biotech firms with global aspirations, it’s important to stay nimble and agile. Whether it is dealing with new strategies in accelerating development globally, adapting to regulatory requirements, or addressing the ever-evolving geopolitical nature of our environment, they need to stay adaptable and flexible, positioning themselves to be the partner of choice and establishing their own global footprint to become a commercial-stage company.”

    In a separate session focused on business development, executives shared lessons from deals that helped transform their organizations.

    Joseph Lasaga, EVP and Chief Business Officer at Rigel Pharmaceuticals, argued that speed can be a competitive advantage.

    “Be in the front of the line at all times,” he said.

    Austin Hackett, VP of Business Development at Innoviva, encouraged companies to look in “under-prioritized areas,” especially if you’re “the only one there.”

    Jesse Shefferman, co-founder, director, and CEO of Protara Therapeutics, emphasized the importance of credibility and authenticity during negotiations.

    “One of the traps you can fall into is posturing that you’re bigger or better or more well-funded than you actually are,” Shefferman said. “I think showing up authentically human in this field that’s governed by dollars and cents goes a long way.”

    The post BIO 2026: Biotech dealmakers navigate uncertainty, pursue opportunities appeared first on Bio.News.

    ]]>     Nextgen Platform Combines VectorBuilder and Maxcyte Technologies to Boost Clinical-Grade Cell Engineering https://edusehat.com/en/nextgen-platform-combines-vectorbuilder-and-maxcyte-technologies-to-boost-clinical-grade-cell-engineering https://edusehat.com/en/nextgen-platform-combines-vectorbuilder-and-maxcyte-technologies-to-boost-clinical-grade-cell-engineering Wed, 24 Jun 2026 03:00:11 +0700 Edusehat Nextgen, Platform, Combines, VectorBuilder, and, Maxcyte, Technologies, Boost, Clinical-Grade, Cell, Engineering VectorBuilder and MaxCyte formed a strategic partnership focused on co-developing a new gene delivery solution using VectorBuilder’s MiniVec™ plasmid system and MaxCyte’s clinical electroporation platform for ex vivo cell engineering.

    Officials at both companies say that ex vivo cell therapies such as CAR-T, CAR-NK, and iPSC-based treatments have gained significant traction, but critical challenges in safety and manufacturability continue to limit their broader application. They explain that existing gene delivery methods present significant trade-offs: traditional electroporation of conventional DNA or RNA often results in poor target cell viability or limited therapeutic durability, while lentiviral vectors, though widely used, carry high production costs and potential safety risks such as malignancy arising from vector integration into the host genome.

    What is collectively needed, both companies maintain, are more efficient, safer, and scalable approaches to cell engineering.

    The partnership between VectorBuilder and MaxCyte has been designed to address these challenges by developing the next-generation electroporation-based ex vivo gene delivery solution through the integration of VectorBuilder’s MiniVec backbone with MaxCyte’s Flow Electroporation® technology.

    MiniVec is described as a miniaturized plasmid backbone that eliminates the need for antibiotic- or additive-based selection during fermentation to simplify translation to GMP-grade production. Its reduced prokaryotic sequences have been shown to improve yield and performance across a broad range of applications, according to VectorBuilder.

    Flow Electroporation relies on a continuous-flow process that reduces cellular stress, preserving cell viability and functionality while enabling scalable, highly efficient gene delivery. This two-pronged approach is designed to deliver significantly improved cell viability and higher transfection efficiency compared to conventional models, pointed out Maher Masoud, president and CEO of MaxCyte.

    “Cell therapy development requires delivering therapies that are manufacturable, scalable, and commercially viable,” he said. “We believe we can enable a new standard for nonviral gene delivery—one that enhances cell quality, improves manufacturing efficiency, and provides developers with a more streamlined path from research through commercialization.”

    “This partnership combines our complementary strengths to establish a next-generation platform for efficient, safe, and scalable electroporation-based cell engineering for therapies such as CAR-T.

    “As both companies have extensive expertise in GMP-compliant clinical development solutions, the combined platform is well aligned to enable a seamless development pipeline from clinical trials through to commercialization,” noted Bruce Lahn, PhD, founder and chief scientist of VectorBuilder. “Our aim is to provide a platform delivering high-end performance with an optimal cost-of-goods and price-per-dose model to ease scale-up, commercial strategy, and fundraising efforts for drug developers.”

    The post Nextgen Platform Combines VectorBuilder and Maxcyte Technologies to Boost Clinical-Grade Cell Engineering appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Nvidia Unveils Science Reasoning AI Suite with BioNeMo Agent Toolkit https://edusehat.com/en/nvidia-unveils-science-reasoning-ai-suite-with-bionemo-agent-toolkit https://edusehat.com/en/nvidia-unveils-science-reasoning-ai-suite-with-bionemo-agent-toolkit Wed, 24 Jun 2026 03:00:10 +0700 Edusehat Nvidia, Unveils, Science, Reasoning, Suite, with, BioNeMo, Agent, Toolkit Nvidia has announced the NVIDIA BioNeMo Agent Toolkit, which turns complex scientific workflows into agent-executable tasks, including model selection, input preparation, workflow execution, output inspection, and results explanation.

    The toolkit includes NVIDIA BioNeMo and is powered by NVIDIA NIM microservices, NVIDIA Parabricks, NVIDIA NeMo, and NVIDIA Nemotron and has applications across protein structure prediction, molecular docking, generative chemistry, genomic analysis, protein design, and biomarker discovery.  

    “For the first time, researchers can build AI agents that understand scientific knowledge, use scientific tools, and execute scientific workflows,” said Jensen Huang, founder and CEO of Nvidia, in a press release. “This is a new way to do science—one that can dramatically accelerate discovery across biology, chemistry, genomics, and medicine.” 

    Nvidia has entered collaborations with research organizations, including the Arc Institute, Open Molecular Software Foundation, and the University of Washington’s Institute for Protein Design (IPD). The partnership with IPD has accelerated runtimes for the biomolecular complex prediction tool, RosettaFold3, resulting in two times faster performance than the prior generation model.  

    “Every tool we’ve built for protein design is only as powerful as the scientists who can efficiently access it,” said David Baker, PhD, professor of biochemistry at the University of Washington and director of the Institute for Protein Design, in a public release. “The next leap in science won’t come from a single discovery; it will come from the speed of iterative designs and agents that can repeatedly reason through the complexity of biology at a speed humans never could.” 

    The toolkit’s applications include virtual screening, where agents identify promising small-molecule drug candidates by generating compound designs, docking them to a target, predicting binding strength, and filtering for developability properties. The agent can then output which candidates should be prioritized to compress timelines. 

    In genomic analysis and target discovery, agents can identify genetic insights and biological targets from raw sequencing data. Agents can also connect real-world data to reasoning models for biomedical research, improving the efficiency and accuracy of clinical development processes, including literature review, protocol generation, clinical trial screening, and pharmacovigilance. In medical imaging analysis, agents can process, segment, synthesize, and reason over medical imaging data to support biomarker discovery. 

    AI-native biology companies, including Boltz, Basecamp Research, Chai Discovery, PerturbAI, Dyno, and Proxima, have collaborated with NVIDIA to develop tools to accelerate therapeutic design workflows. Diagnostics and pharmaceutical companies, including Lilly and Natera, are using BioNeMo Agent Toolkit to scale agentic workflows across discovery, translational research, and clinical insight. 

    The post Nvidia Unveils Science Reasoning AI Suite with BioNeMo Agent Toolkit appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Three Subtypes of Severe Pneumonia Might Inform Personalized Therapies https://edusehat.com/en/three-subtypes-of-severe-pneumonia-might-inform-personalized-therapies https://edusehat.com/en/three-subtypes-of-severe-pneumonia-might-inform-personalized-therapies Wed, 24 Jun 2026 03:00:09 +0700 Edusehat Three, Subtypes, Severe, Pneumonia, Might, Inform, Personalized, Therapies The results of a study headed by researchers at the University of Cambridge suggest that severe pneumonia has three different subtypes, a discovery that could help to explain why some patients in intensive care units (ICUs) recover from their illness faster than others, and for some patients, the disease can be life-threatening. Rather than assessing patients’ symptoms, the Cambridge team analyzed fluid taken from the lungs of patients admitted to the hospital with suspected pneumonia. Their results indicated that although each of the three different “pneumotypes” of severe pneumonia was associated with how the patients recovered, none could be reliably identified using standard blood tests.

    The researchers suggest that their findings could in the future help inform personalized therapeutic strategies, allowing individual patients to receive the most appropriate treatment. Andrew Conway Morris, PhD, at the Department of Medicine at the University of Cambridge and an ICU consultant at Addenbrooke’s Hospital, Cambridge, is senior author of the team’s published paper in Nature Communications, titled “Pulmonary inflammation in severe pneumonia is characterised by compartmentalised and mechanistically distinct sub-phenotypes.”

    Pneumonia is the commonest infectious cause of death worldwide, responsible for an estimated 2.5 million deaths per year, the researchers noted. In severe cases, patients may need to be admitted to an ICU and given mechanical ventilation. Severe pneumonia accounts for six in 10 infections managed in intensive care, and spread of the infection within ICUs is a significant concern.

    Doctors have long struggled to understand why patients whose condition looks similar clinically can have very different recoveries. Some respond quickly to treatment, while others remain critically ill for weeks or even die. “Despite the considerable burden of pneumonia, the syndrome is incompletely understood, and diagnosis is difficult,” the team explained.

    Conway Morris said, “Even though we’re able to treat the initial infection, many patients with severe pneumonia still struggle to come off the ventilator and can develop lung failure. Therapies to tackle inflammation in the lungs have had mixed results in clinical trials—some suggest they are beneficial, others that they’re harmful.”

    Severe pneumonia is usually diagnosed through a combination of symptoms, imaging, and blood tests. Symptoms typically include fever or hypothermia, low oxygen levels, breathing difficulties, and confusion. “The current approach of classifying patients by their clinical syndromes—sepsis, acute respiratory distress syndrome, and so on—without looking at the underlying biology risks missing what’s key,” Conway Morris noted. “Instead of asking ‘Does this patient have pneumonia?’, we should be asking ‘What’s the inflammatory pattern in this patient’s lungs?’”

    For their newly reported study, Conway Morris and team recruited 80 patients admitted with suspected severe pneumonia to the ICU at Addenbrooke’s Hospital. Instead of relying only on blood tests or scans, however, the Cambridge team analyzed the patient’s immune cells, inflammatory signals, and gene activity in bronchoalveolar lavage samples. “Here, we perform multifaceted assessments of bronchoalveolar transcriptome, cytokines, microbiology, and clinical features to biologically characterise a cohort of patients with suspected severe pneumonia,” they reported in their paper. The researchers discovered three distinct biological types—or pneumotypes (Pn)—of severe pneumonia, none of which could be reliably detected using standard blood tests, even though they were strongly linked to how patients recovered.

    “Using bulk RNA sequencing of bronchoalveolar fluid, we have identified three phenotypes in the lungs of patients with lung injury and suspected pneumonia,” they stated. “These phenotypes were reflected in the differential immune cell populations and inflammatory proteins.”

    The most common pneumotype—accounting for almost half (49%) of cases—was characterized by immune suppression, significant damage to the lining of the lungs, and bleeding in the alveoli (tiny air sacs within the lungs). There were fewer signs of inflammation, which may explain why treatments targeting inflammation can fail or even harm some patients. “Pn1, the most common, is characterized by low alveolar cytokines, expanded tolerogenic macrophages, and epithelial damage,” the investigators reported.

    The second pneumotype—accounting for just under a quarter (23%) of cases—was characterized by a balanced immune response and active repair of damage to the lungs. Patients were most likely to recover faster from this pneumotype and require the shortest time on the ventilator, even though they initially looked just as ill as the others. “Pn2 displays the fastest resolution, exhibiting a balanced immune response and epithelial-endothelial repair signatures,” they continued.

    Patients with the most dangerous pneumotype—the one that most resembles “classic” pneumonia—spent the longest on mechanical ventilation and had prolonged critical illness. They had severe and persistent inflammation, with a flood of immature immune cells in the lung. This group may be most likely to respond to anti-inflammatory therapies, the team said. “Pn3 is characterized by immature neutrophil infiltration, IL-6-STAT3 activation, and longer duration of mechanical ventilation,” the scientists stated.

    First author Dr. Mark Jeffrey, at the Department of Medicine at the University of Cambridge, added, “Even though on the surface, all of the patients seemed to have similar types of pneumonia, with comparable illness severity, oxygen levels, and clinical diagnoses, their outcomes were very different. It was only when we drilled down and looked at patterns of inflammation that the differences became apparent. Severe pneumonia is not a single disease, but several biologically distinct conditions that happen to look alike. This helps explain why ‘one-size-fits-all’ treatments—including some immune-modulating drugs—have often failed in clinical trials.”

    Interestingly, the authors added in their report, “Each of the Pneumotypes contained both patients with and without confirmed pneumonia, implying common mechanisms underpinning lung injury arising from different mechanisms.”

    The tests used to determine the pneumotypes are too complex to enable rapid classification, but the researchers hope to develop a simplified tool that could help them stratify the patients and ultimately offer tailored treatments.

    Co-author Vilas Navapurkar, MBChB, from the John Farman Intensive Care Unit at Addenbrooke’s Hospital, said, “If we know which subtype of pneumonia an individual has, we can potentially tailor their treatment more precisely, boosting the immune response in some, while calming harmful inflammation in others. This has the potential to help critically ill patients, reduce deaths from pneumonia, shorten ICU stays, and cut unnecessary antibiotic use.”

    The team also noted that while their study identified three Pneumotypes, it’s likely that others may exist, which might be identified in larger studies. In conclusion, they wrote, “… we have identified and validated three pulmonary confined endotypes in patients with severe pneumonia and lung injury. These phenotypes are underpinned by distinct mechanisms and have differential outcomes. The mechanisms point to different therapeutic options, as well as extending our understanding of the biology of lung inflammation in the context of severe pneumonia.”

    The post Three Subtypes of Severe Pneumonia Might Inform Personalized Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cytomos’ AuraCyt Digital Predictive Cell Analytics Platform Showcased at BIO Conference https://edusehat.com/en/cytomos-auracyt-digital-predictive-cell-analytics-platform-showcased-at-bio-conference https://edusehat.com/en/cytomos-auracyt-digital-predictive-cell-analytics-platform-showcased-at-bio-conference Tue, 23 Jun 2026 23:25:09 +0700 Edusehat Cytomos’, AuraCyt, Digital, Predictive, Cell, Analytics, Platform, Showcased, BIO, Conference Cytomos reports that it is showcasing its flagship cell analytic intelligence platform AuraCyt®, together with its novel Celledonia® benchtop technology, which is designed to boost predictive cell analytics. Current challenges in the bioprocessing space include slow, label-dependent analytical methods that are inefficient to scale-up and provide limited predictive insight, according to Cytomos.

    The AuraCyt platform measures intrinsic cell physics to generate AI-ready digital fingerprints that reveal the multi-dimensional state and behavior of the cell. The benchtop cell analytics intelligence system combines the compact Celledonia analyzer with the sensors of the AuraCyt modules to transform complex cellular data into actionable insights, notes a Cytomos spokesperson, who points out that this label-free, single-cell analysis system predicts future productivity, stability, and manufacturability.

    This enables earlier insights and real-time decision-making, lowering risk, providing scalable impact, optimizing process and improving product consistency, says the spokesperson. The operation reportedly provides demonstrable value across various applications, such as reducing CLD timelines by up to 40%, saving up to 65% of resources in lentivirus batch production, and reducing CAR T process time by up to 30%, comments Cytomos’ executive chair, Alan Raymond.

    The company is showcasing this technology at its pod in BIO Business Forum Zone D (Exhibit Hall 2221 UK Pavillion), from 22-25th of June, 2026.

     

    The post Cytomos’ AuraCyt Digital Predictive Cell Analytics Platform Showcased at BIO Conference appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO 2026: Where AI is delivering – and where biotech must go next https://edusehat.com/en/bio-2026-where-ai-is-delivering-and-where-biotech-must-go-next https://edusehat.com/en/bio-2026-where-ai-is-delivering-and-where-biotech-must-go-next Tue, 23 Jun 2026 09:10:03 +0700 Edusehat BIO, 2026:, Where, delivering, –, and, where, biotech, must, next Artificial Intelligence is on the lips of everyone at the 2026 BIO International Convention.

    And as the second-annual AI Summit began on June 22, everyone was talking about one headline in particular: the $2.5 billion deal between Insilico Medicine and SK Biopharmaceuticals. The deal leverages Insilico’s Pharma.AI platform with SK Biopharmaceuticals’ expertise.

    With this ramp-up in technology and dealmaking, the AI Summit explored several key questions. Where is AI delivering? What does effective adoption look like? How do we ensure we have high-quality datasets to train effective scientific agents?

    “We have to think about how we’re going to get smarter about the medicines that we’re inventing for patients, and how we can bring massive amounts of data that we have now to the research forefront,” said Fritz Bittenbender, Senior Vice President of Public Affairs and Access at Genentech and BIO Board Chair. “How can we use AI to actually sift through that data and find targets faster? How can we use AI to help us develop molecules that would take scientists years and years to develop what we can do in months or weeks now?”

    Where is AI delivering today?

    Many of the conversations focused on where AI is most effectively used today.

    As Benchling found in their 2026 AI Report, the highest rate of effective usage has been in “knowledge extraction (76% adopting), protein structure and property models (71%), scientific reporting (66%), and target identification (58%).”

    The turnaround time when using these tools is notable: “50% of biotechs report faster time-to-target today and 56% expect cost reductions within two years as automation and agentic workflows scale.”

    In some cases, lab experiments are going from months to days, while agents are training on available data to perform even faster down the line.

    “It’s not perfect yet,” warned Joshua Meier, Co-Founder of Chai Discovery. “But we’re in a very different regime now than we were even a year ago. I think that’s one of the reasons why we’re just starting to see even more progress, because when you can use the models to then go and generate more data, then you’ve got this really exciting flywheel.”

    Good data is key to good AI agents – and there is plenty of it out there. Yet access remains difficult for many reasons.

    “It’s difficult sometimes in a larger, sort of more conventional R&D organization to just generate a lot of data just because,” noted Mary Rozenman, Ph.D., CBO/CFO at Insitro.

    Rozenman recalls the early days of AI, when Insitro’s founder, Daphne Koller, literally wrote the book on probabilistic graphical models. “One of the early concepts was that there is a tremendous amount of information content in data that captures human health and disease,” explained Rozenman. If researchers went in objectively, without a pre-existing hypothesis and with the right computational tools, they would likely uncover important insights.

    “It still feels hard for people to trust-fall into their data,” observed Rosenman.

    ‘Most organizations are adopting AI incorrectly’

    As many experts pointed out throughout the day, adoption overall is broad rather than deep.

    “Senior executives have to lead from the front, and they have to not just be cheerleaders, but also be users of the technology,” said Sajith Wickramasekara, CEO and co-founder of Benchling.

    “My core contention is that most organizations are adopting AI incorrectly,” admitted Wickramasekara. “They are sprinkling a little bit of it on their jobs as it is, and it requires more of a fundamental rethink.”

    Wickramasekara pointed to Cutko knives and the door-to-door salespeople of yesterday. Say it’s 2003, and Jack and Jill are selling knives just as the internet age is taking off. Jack spends every day going door-to-door and only experiments with digital advertising in the evening and on the weekends. He gets one or two more leads, and he feels good. Jill, on the other hand, stops going door-to-door completely. Her sales dry up, but after a year, she is able to get back up to flat sales.

    “But if you ask yourself, who’s better off a year later, two years later, the answer is, I think, pretty obvious – nobody sells knives door to door anymore,” Wickramasekara pointed out. “Every pre-AI company of any scale is kind of being Jack right now, and that they need to be Jill.”

    How biotechs can incorporate AI

    As the panelists discussed, AI can quickly solve time and cost problems in R&D, even if the initial price tag is steep.

    “What’s wild about that is that not only are we sitting faced with these once-in-a-lifetime – or many lifetimes – technologies, not only are we sitting with this vast unmet patient need, but people are doing the same thing over and over again,” said Rozenman.

    “It totally makes sense why people don’t want to embrace clinical risk upfront,” she continued. “We’re all taught to reduce the risk, but at this moment with this incredible set of technologies and the pace of innovation, the models are just getting better and better and better. We should be challenging ourselves as an industry to do better and identify a way to get therapeutic programs not only first in class, but also with a higher probability of success.”

    “At this moment, we all owe ourselves and each other and patients that kind of bold perspective.”

    Her words were met with spontaneous applause.

    The post BIO 2026: Where AI is delivering – and where biotech must go next appeared first on Bio.News.

    ]]>     BIO 2026: The business of biotech runs on partnerships https://edusehat.com/en/bio-2026-the-business-of-biotech-runs-on-partnerships https://edusehat.com/en/bio-2026-the-business-of-biotech-runs-on-partnerships Tue, 23 Jun 2026 09:10:03 +0700 Edusehat BIO, 2026:, The, business, biotech, runs, partnerships A $2.5 billion biotech AI deal announced at the beginning of the 2026 BIO International Convention grabbed headlines, but it’s only one of many partnership discussions that will begin, move forward, or culminate in a deal this week.

    Partnering is a major reason the Biotechnology Innovation Organization (BIO) hosts the BIO International Convention, the world’s largest gathering of the biotech industry, taking place in San Diego from June 22-25.

    It was therefore fitting that Insilico Medicine and SK Biopharmaceuticals announced their new agreement at BIO 2026. The deal will see Insilico leverage its Pharma.AI platform and SK Biopharmaceuticals contribute its medical expertise “to discover AI-enabled innovative drug candidates in the neuroimmune area of the central nervous system.” Insilico could earn a potential total of $2.5 billion.

    With close to 70,000 partnering meetings scheduled, the BIO International Convention is sure to drive more exciting deals.

    “BIO is a great place for partnering. I met with a viral vector manufacturing partner for the first time at BIO that ended up in a multi-million-dollar contract,” said Audrey Greenberg of the Mayo Clinic’s Mayo Venture Partner ahead of the convention.

    Convention-goers awaiting partnership discussions in one of the convention’s meeting spaces spoke about why they come to the event.

    “Most of our advertising, if you will, is done in person, or word of mouth, and we take these meetings very seriously,” said Richard Brown, managing partner at the global business development firm Plexus Ventures. “Every year, I come to BIO. I’ve been here since I was at Lilly 24 years ago.”

    “Why BIO? Because you need a fairly integrated view of the partnering space, of the multi-stakeholder takes, views, priorities,” said Lennart Spindler an investor with the Dementia Discovery Fund, SV Health Investors, a leader in venture funds seeking therapeutics for neurodegenerative disorders.

    BIO 2026 is a hub for partnering

    As development challenges increase, the unique opportunities offered by the BIO International Convention become all the more important, according to Mackensie Vernetti, SVP of Partnering at the Biotechnology Organization (BIO).

    “While factors in the market may change, we see the same levels of success throughout our events because partnering is essential to the industry,” she said. “People get quite creative with the kind of deals and meetings they make.”

    The partnering at the BIO International Convention is powered by the BIO Partnering(TM) system, BIO’s bespoke platform allows participants to enter what they’re looking for and find other partners whose interests match theirs. The platform then schedules a time for the meeting and sets a location from among the 2,000 meeting rooms and booths available throughout the vast convention center.

    According to Vernetti, BIO 2026 has seen a record level of partnering activity with more than 65,000 meetings arranged through the system by opening day, and new meetings are being scheduled.

    The benefit of the BIO International Convention is that it draws the entire biotech ecosystem, bringing every type of potential collaborator to San Diego.

    “This is the opportunity for a company to meet a greater variety of partners than they could meet in any other one place,” said Bernard Fallon, BIO VP of Industry Programs. “And investors can get a very efficient view of the state of innovation.”

    One example of innovation on display is the BIO International Convention’s Start-up Stadium, where 50 small biotechs from around the world will pitch their innovations on stage, he noted.

    Other business development assistance from BIO

    Beyond the BIO International Convention, BIO enables partnering at the annual BIO Investment and Growth Summit, held in Miami this year. “This is a more compact, curated event for the investor community and the early-stage venture-ready companies to interact,” according to Fallon.

    Another initiative is BIO’s webinar series, “Raising Capital”, which has featured ARPA-H offering advice on obtaining federal seed funding, and BIO Chief Legal Officer Joe Franklin joining lawyers from Covington to discuss global out-licensing considerations for manufacturers.

    BIO also runs Investor Connect Plus in cooperation with LaunchBio. BIO member companies can apply to meet one-on-one with institutional and strategic investors who are actively deploying capital.

    “This partnership will help match the right innovators with the right investors to deliver more groundbreaking treatments to patients,” according to Brad Zakes, BIO SVP of Emerging Companies and Economic Development.

    Encouraging innovative new treatments is the ultimate goal of all BIO business development efforts.

    The post BIO 2026: The business of biotech runs on partnerships appeared first on Bio.News.

    ]]>     Long‑Range Gene Networks Uncover 641 New Schizophrenia‑Associated Genes https://edusehat.com/en/longrange-gene-networks-uncover-641-new-schizophreniaassociated-genes https://edusehat.com/en/longrange-gene-networks-uncover-641-new-schizophreniaassociated-genes Tue, 23 Jun 2026 09:05:08 +0700 Edusehat Long‑Range, Gene, Networks, Uncover, 641, New, Schizophrenia‑Associated, Genes Schizophrenia’s genetic landscape just expanded dramatically. A new study in Nature Genetics identifies 641 previously unrecognized genes associated with schizophrenia, thanks to a modeling framework that captures how distant genetic variants regulate gene expression through co‑expression networks. The work reframes schizophrenia not as a collection of isolated genetic hits, but as a disorder shaped by long‑range regulatory relationships across the brain. The study is titled, “Co‑expression‑based models improve eQTL predictions for transcriptome‑wide association studies and highlight new schizophrenia‑associated genes.”

    The research team, led by Giulio Pergola, PhD, at the Lieber Institute for Brain Development (LIBD), developed two trans‑aware predictive models—INGENE and MODULE—that quantify how variants far from a gene influence its expression through co‑regulated partners. Traditional transcriptome‑wide association studies (TWAS) focus almost exclusively on cis‑expression quantitative trait loci (cis–eQTLs), variants within ±1 Mb of a gene. But as the paper noted, “Most transcriptome‑wide association approaches primarily model local (cis) genetic effects, leaving much of gene regulation unexplained.” By contrast, the new models incorporate distal (trans) regulatory effects, capturing regulatory relationships that behave more like social networks than neighborhood blocks.

    Using RNA‑seq data from six human post‑mortem brain regions and genetic data from more than 102,000 individuals, the team integrated cis‑based predictors (CIS, EpiXcan) with their new trans‑based frameworks. The combined approach improved gene‑expression prediction for 18,744 genes, and when applied to Psychiatric Genomics Consortium (PGC3) datasets, it identified 766 schizophrenia‑associated genes, including 641 not previously detected by TWAS.

    Pergola said the field has been “looking for the light under the lamppost, focusing only on genes close to disease‑associated DNA variants.” By illuminating long‑range interactions, he explained, “we’ve essentially turned on lights across the entire neighborhood, revealing how distant genetic variants coordinate to build the genetic basis of schizophrenia.”

    The findings converge on pathways involved in glutamate signaling, neuronal communication, immune processes, and neurodevelopment—biological systems repeatedly implicated in psychiatric risk. MODULE‑derived trans‑single nucleotide polymorphisms (SNPs) showed particularly strong enrichment for schizophrenia‑associated variants, and many overlapped with cis‑eQTLs for transcription factors such as GATAD2A, RERE, IRF3, and SP4, all previously prioritized in schizophrenia GWAS.

    Daniel Weinberger, MD, CEO and director of LIBD, emphasized the shift in perspective: “Schizophrenia risk isn’t just about individual genes acting one after another—it’s about how networks of genes work together. Understanding these coordinated genetic programs brings us closer to precision psychiatry.”

    By demonstrating that trans‑regulatory architecture is both detectable and biologically meaningful, the study provides a roadmap for expanding TWAS beyond local effects. It also underscores the importance of integrating multi‑region brain transcriptomics with large‑scale genetic cohorts to reveal disease‑relevant regulatory relationships.

    The post Long‑Range Gene Networks Uncover 641 New Schizophrenia‑Associated Genes appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AI Discovers Potential Antimicrobial “Prionin” Peptides https://edusehat.com/en/ai-discovers-potential-antimicrobial-prionin-peptides https://edusehat.com/en/ai-discovers-potential-antimicrobial-prionin-peptides Tue, 23 Jun 2026 05:30:10 +0700 Edusehat Discovers, Potential, Antimicrobial, “Prionin”, Peptides Prions are best known for their role in rare, fatal neurodegenerative diseases. But a new study by researchers at the University of Pennsylvania suggests that proteins in this family may also conceal molecular fragments that can kill bacteria, including drug-resistant strains.

    The Penn scientists used a deep learning platform called APEX 1.1 to scan millions of short protein fragments derived from nearly 3,000 prion and prion-like proteins. The search identified more than a thousand candidate antimicrobial peptides, which they called “prionins.” In tests 59 synthesized prionins inhibited bacterial pathogens, and two reduced Acinetobacter baumannii burden in mice.

    The discovery is unexpected because prions are usually discussed in the context of misfolding, aggregation, and brain disease—not immunity or antibiotic discovery. The new findings suggest that useful biological activities may be hidden inside proteins whose known roles have little to do with infection, and that artificial intelligence can help reveal them.

    “Prions have long been seen almost entirely through the lens of disease,” said César de la Fuente, PhD, associate professor and director of the Machine Biology Group at the University of Pennsylvania. “Our work shows that when AI looks across biology at scale, even proteins with a dark reputation can contain useful molecular instructions. In this case, those instructions point to possible new antibiotics.”

    De la Fuente is senior and corresponding author of the researchers’ published paper in Nature Microbiology, titled “Deep learning reveals antimicrobial peptides within prions.”

    Antibiotic resistance is among the most urgent challenges in medicine, and many existing antibiotics were discovered by searching traditional natural sources. The new study takes a different route: instead of asking where antibiotics usually come from, it asks whether biology has hidden antimicrobial molecules in places scientists would not normally look.

    Certain amyloid-associated protein sequences may participate in host defense, the authors wrote. “Several amyloid-associated proteins, including amyloid-β and the cellular prion protein, have been reported to display antimicrobial or host-protective activities, raising the possibility that aggregation-prone proteins may encode cryptic antimicrobial fragments within their primary sequence.” But until now, scientists had not systematically searched prion and prion-like proteins at scale to ask whether they broadly encode hidden antimicrobial peptides.

    “Whether such encrypted peptides are broadly embedded across prion and prion-like proteins has not been systematically examined,” the researchers continued. The Penn team took on that task, using AI to move from scattered observations to a global search across millions of possible protein fragments. They mined prion-related proteins with APEX1.1, a deep learning platform for antimicrobial peptide (AMP) discovery. “… using deep learning, we screened 19.3 million fragments from 2,897 curated prion-related proteins and identified 1,179 candidate antimicrobial peptides, which we term prionins,” they stated.

    To test the predictions, the researchers synthesized 75 prionins and evaluated them against a panel of clinically relevant bacterial pathogens, including multidrug-resistant strains. Fifty-nine inhibited at least one pathogen, and 42 showed potent activity at concentrations of 16 micromolar or lower against at least one pathogen.

    The team then examined how the molecules worked. Many active prionins damaged bacterial membranes, a common mechanism used by antimicrobial peptides. Importantly, several candidates also showed early signs of selectivity: hemolysis was rare, and 16 active peptides showed neither measurable hemolysis nor cytotoxicity at the highest concentrations tested.

    Two of the strongest candidates were tested in a mouse skin-infection model caused by Acinetobacter baumannii, a difficult-to-treat pathogen. A single topical dose of each peptide significantly lowered the bacterial burden, with effects comparable to the antibiotic polymyxin B in the model tested. The researchers observed no treatment-associated weight loss. In summary, they wrote, “What makes this exciting is that the predictions held up experimentally,” said Marcelo D T Torres, PhD, co-first author of the study. “We went from millions of hidden protein fragments to synthesized molecules that killed bacteria in the lab, and then to candidates that worked in an animal infection model. That is the difference between an AI screen and a true discovery platform.”

    The findings build on the de la Fuente lab’s broader effort to mine the biological world for “encrypted peptides”—short, hidden sequences within larger proteins that can have biological functions when isolated. Previous work from the group has searched human proteins, extinct organisms, archaea, microbiomes, and venoms. The prion study expands that concept into one of biology’s most unexpected protein classes.

    The study also raises a provocative possibility at the intersection of neurodegeneration and innate immunity. It does not establish that these peptides are naturally released during infection or function physiologically in host defense, they stated. But it suggests that prion and prion-like proteins may contain cryptic antimicrobial sequences, opening a new way to think about prion biology and its possible links to immunity. “… it establishes prion-related proteins as a productive source space for antibiotic discovery and provides a framework for testing whether cryptic peptides contribute to defense in specific biological contexts.”

    The researchers emphasize that this is an early discovery, not a new treatment ready for patients. The study does not change the established role of misfolded prions in devastating neurodegenerative disease. Instead, it suggests prion and prion-like proteins as a rich and previously overlooked source space for antibiotic discovery. “Our findings identify prion and prion-like proteins as an unexpectedly rich reservoir of encrypted AMPs,” the authors concluded. “This expands a growing view that antimicrobial activity can be hidden within proteins not canonically annotated as immune effectors and extends that concept to prion biology … These results connect prion-related sequence space to antimicrobial function and highlight unconventional protein classes as sources of antibiotic leads.”

    “For a long time, drug discovery has been limited not only by what we can test, but by where we choose to look,” de la Fuente said. “AI is changing that. It gives us a way to search the hidden layers of biology and ask whether molecules associated with one story—in this case, disease—may also carry another story with therapeutic potential.”

    The post AI Discovers Potential Antimicrobial “Prionin” Peptides appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AbbVie to Acquire Apogee Therapeutics for $10.9B https://edusehat.com/en/abbvie-to-acquire-apogee-therapeutics-for-109b https://edusehat.com/en/abbvie-to-acquire-apogee-therapeutics-for-109b Tue, 23 Jun 2026 01:55:08 +0700 Edusehat AbbVie, Acquire, Apogee, Therapeutics, for, 10.9B SAN DIEGO — AbbVie has agreed to acquire Apogee Therapeutics for $10.9 billion, the companies said today, in a deal designed to bolster the buyer’s pipeline with an atopic dermatitis (AD) candidate set to advance to Phase III trials during the second half of this year, and being positioned as a potential challenger to a top-selling drug.

    Apogee’s lead candidate zumilokibart, an IL-13 inhibitor also called APG777, is a long-acting treatment that according to the company holds “pipeline-in-a-product potential” because of the opportunity it has for treating a variety of immunology and inflammation (I&I) diseases for which the drug is under study.

    “We continue to believe that Apogee’s zumilokibart is one of the more attractive assets in the I&I space, and its current valuation proves this out,” Edward Nash, a managing director and senior biotechnology analyst with Canaccord Genuity, wrote this morning in a research note. “The company, since its 2022 inception, has continued to deliver strong clinical results for zumilokibart in atopic dermatitis. The BIG [emphasis in original] differentiator for the drug is its potential to be dosed once every three or six months, which was just demonstrated in recently announced updates from the Phase II trial.”

    Last month, Apogee announced positive 16-week data from Part B of its Phase II APEX trial (NCT06395948) assessing zumilokibart in moderate-to-severe AD. The trial met its primary and secondary endpoints with high statistical significance, as 65.9% of patients treated with mid-dose zumilokibart achieved EASI-75 (41.9% placebo adjusted).

    Based on these results and subject to positive regulatory feedback, Apogee said it planned to move forward in its Phase III trials with the mid-dose, which achieved the best clinical activity of the three doses tested and was well-tolerated.

    To support those Phase III trials and continued late phase development and potential commercialization of zumilokibart, Apogee last month entered into a strategic financing for up to $1.3 billion in flexible, non-dilutive total capital.

    The capital includes up to $800 million of synthetic royalty funding and access of up to $500 million in senior corporate debt available by mutual consent of Blackstone and Apogee. Blackstone agreed to provide the synthetic royalty funding in exchange for low-to-mid single digit tiered royalties for 15 years on worldwide annual sales of zumilokibart. The royalties decrease with increasing sales, with zero royalties paid out on global annual sales exceeding $8 billion.

    Third-largest deal, so far

    The $10.9 billion Apogee acquisition is the new third largest biopharma merger-and-acquisition (M&A) deal announced so far this year, behind the €10.7 billion ($12.268 billion) cash buyout offer for Italian-based Recordati being pursued by CVC Capital Partners and Groupe Bruxelles Lambert, which aim to take the company private; and Sun Pharmaceutical Industries’ planned $11.75 billion purchase of Organon, the women’s health drug developer spun out of Merck & Co., in a deal expected to close in early 2027.

    The previous third-largest M&A deal this year, now fourth-largest, is GlaxoSmithKline (GSK)’s planned $10.6 billion buyout of Nuvalent,  announced June 9 and expected to close in the third quarter.

    For AbbVie, the deal for Apogee adds to its pipeline in I&I, a category the biopharma giant dominated when its multi-indication blockbuster Humira® (adalimumab) was the world’s best-selling drug, before it lost patent exclusivity in the European Union in 2018 and the U.S. in 2023—after which it slipped from the top of GEN’s annual A-Lists of Top 10 Best-Selling Drugs.

    However, AbbVie has developed two successful I&I drugs in recent years, Skyrizi® (risankizumab)  and Rinvoq® (upadacitinib)—with Skyrizi ranking No. 6 on GEN’s latest best-selling drugs A-List, generating $17.562 billion in sales last year (up 49.9% from 2024) and $4.483 billion in Q1 2026, up 30.9% from Q1 2025.

    “For more than two decades, AbbVie has led and shaped the field of immunology bringing the science, scale and expertise needed to address some of the most complex diseases,” Robert A. Michael, AbbVie’s chairman and CEO, said in a statement. “The acquisition of Apogee further builds on our existing leadership, strengthening our ability to deliver innovative medicines to patients who need better options while also creating significant long-term value for shareholders.”

    Apogee investors signaled support for the buyout with a surge of stock buying that sent the company’s shares soaring 47% in early day trading from $90.38 to $132.65 as of 10:21 am ET. AbbVie shares rose 4.5% from $216.49 to $226.24.

    Potential Dupixent® challenger

    Apogee is positioning zumilokibart as a potential challenger to Dupixent® (dupilumab), the blockbuster drug for AD and other indications that is co-marketed by Sanofi, which records global net sales, and Regeneron Pharmaceuticals.

    Dupixent ranked No. 5 among “Top 10 Best-Selling Drugs” as ranked by GEN in a recent A-List, with $18.124 billion (€15.714 billion) in 2025 sales, up 20.2% from the $15.077 billion (€13.072 billion) that the drug racked up in 2024. Dupixent carried that momentum into the first quarter of this year, garnering $4.9 billion (€4.2 billion) in sales as recorded by Sanofi, up 33% from a year earlier. 

    However, Dupixent is set to lose key U.S. patent exclusivity in 2031, giving Apogee and other AD drug developers time, they hope, to bring new treatments to market that can successfully compete when Dupixent loses its IP protection.

    In addition to AD, zumilokibart is also being developed to treat asthma and eosinophilic esophagitis (EoE). The EoE program is set to advance into mid-stage clinical study as Apogee plans to launch the Phase IIb ELEVATE trial in the second half of this year.

    Apogee has generated positive Phase Ib data for zumilokibart in asthma, and is on course to advance that program into the Phase IIb ASPIRE trial, set to launch in the first half of 2027.

    Two other programs, both of them combination therapies that include zumilokibart, round out Apogee’s pipeline. APG279, a combination of zumilokibart and APG990, an OX40L inhibitor, is an AD candidate now in a Phase I trial (NCT07027527) comparing the the safety, tolerability, and pharmacokinetic (PK) parameters of the combination vs. Dupixent in adults with moderate-to-severe atopic dermatitis (AD).

    Apogee cites preclinical studies showing that APG279 has driven closer to JAK-like inhibition of Type 1, 2, and 3 signaling compared to approved or in-development biologics, with the potential for best-in-class dosing and better tolerability in AD and a variety of other I&I diseases.

    One-two punch

    Apogee reasons that its chances of treating AD are enhanced by a proverbial one-two punch combining deep and sustained inhibition of Type 2 inflammation through zumilokibart’s inhibition of IL-13 with broader inhibition of Type 1-3 inflammation through APG990’s inhibition of OX40L.

    The other combination program, APG273, is a preclinical combination of zumilokibart with APG333, a TSLP (thymic stromal lymphopoietin) that is being developed to treat asthma and COPD. Apogee has said it plans to announce additional plans for clinical studies later this year.

    “Apogee’s pipeline adds highly differentiated clinical-stage assets, further expanding our robust immunology portfolio in areas of significant patient need, including atopic dermatitis and asthma,” Michael added. With our deep scientific expertise and proven capabilities, we are uniquely positioned to rapidly advance these programs and continue to transform the standard of care in inflammatory diseases.”

    AbbVie has agreed to acquire all outstanding shares of Apogee for $135.11 per share cash, a 49.5% premium from the stock’s closing price on Friday.

    The boards of AbbVie and Apogee have unanimously approved the transaction, which is expected to close in the third quarter subject to customary closing conditions, including Apogee shareholder approval and receipt of regulatory approvals.

    “This transaction reflects the strength of Apogee’s vision, our team’s dedication and the significant progress we’ve made advancing zumilokibart and our differentiated pipeline,” stated Apogee CEO Michael Henderson, MD. “Since our founding, we’ve focused on developing transformative therapies for patients with inflammatory diseases while creating value for shareholders. This transaction delivers substantial shareholder value and positions our programs to reach their full potential.”

    “We believe AbbVie can advance zumilokibart and our portfolio while expanding their impact for patients worldwide,”  Henderson added.

    The post AbbVie to Acquire Apogee Therapeutics for $10.9B appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO 2026 begins in San Diego, marking 50 years of biotech innovation https://edusehat.com/en/bio-2026-begins-in-san-diego-marking-50-years-of-biotech-innovation https://edusehat.com/en/bio-2026-begins-in-san-diego-marking-50-years-of-biotech-innovation Mon, 22 Jun 2026 22:25:04 +0700 Edusehat BIO, 2026, begins, San, Diego, marking, years, biotech, innovation The Biotechnology Innovation Organization (BIO) International Convention kicks off in San Diego – and 2026 is a special year.

    “This is a special year for BIO, and to be back here at Convention in San Diego and in California, the state where biotech started 50 years ago,” said BIO President & CEO John F. Crowley. “For half a century, biotechnology has improved the lives of men and women around the world, providing hope to so many.”

    The BIO International Convention is the world’s largest gathering of the biotech industry. Taking place from June 22-25, 20,000 attendees are expected this year, and 70,000 meetings scheduled through the BIO Partnering™ platform are expected to take place onsite.

    The theme of this year’s convention is “Driven by Purpose.” 

    “It’s an exciting time and an inflection point. In many ways, our greatest challenges are man-made,” said Crowley. “As we look at all the great needs still out there, never before have we had so many technologies that offer hope to so many.” 

    BIO 2026 features AI Summit, special mainstage guests

    The 2026 convention will put a special focus on how AI can make innovation faster.

    BIO 2026 begins on Monday, June 22, with the second-annual AI Summit, featuring seven marquee panels. Throughout the week, a total of 20 sessions will look at various AI use cases.

    “AI systems will likely have a central role in over the next several years in almost every component of the biotech ecosystem,” Joe Franklin, BIO Chief Legal and Policy Officer, told Bio.News.

    The birth of the biotech industry, with the founding of Genentech 50 years ago, will be marked during multiple sessions, including a super session on June 23 on strengthening the biopharma ecosystem for the next generation. On June 24, award-winning journalist Katie Couric will moderate a conversation entitled “Driven by Innovation” with Ashley Magargee, CEO of Genentech, and Kimberly Powell, VP of Healthcare at NVIDIA.

    Other highlights include actor Gary Sinise, whose foundation supports veterans, first responders, and their families, and who produced an album with his son to raise awareness of chordoma, a rare cancer. INDY NXT racer James Roe will discuss his asthma journey and advocacy, and Notre Dame Football Coach Marcus Freeman will discuss what it means to be “Driven by Purpose,” drawing on lessons in leadership, teamwork, and resilience.

    Networking and cutting-edge innovation

    Networking and partnering will be an essential part of the BIO International Convention, facilitated by the BIO Partnering™ system. Whether they’re seeking investment, licensing deals, manufacturers, or development partners, participants can register their interests in advance in the BIO Partnering™ system. 

    The BIO Partnering™ system will schedule meetings and reserve a meeting location from among the 2,000 meeting rooms and booths set up at the San Diego convention site.

    Potential partners seeking new innovations can find a host of examples at the Start-up Stadium, where representatives of 50 exciting emerging biotechs from around the world will pitch their companies on stage.

    How to follow our coverage

    Bio.News will cover discussions and interview attendees throughout the BIO International Convention to make sure you don’t miss anything important. Themes we’ll be following include how the biotech ecosystem delivers outcomes, pressures on biotech, and the impact of policy.

    Click here to subscribe and receive the Bio.News Newsletter in your inbox every day this week.

    The post BIO 2026 begins in San Diego, marking 50 years of biotech innovation appeared first on Bio.News.

    ]]>     Breast Milk Fatty Acid Shapes Immune Development in Mice https://edusehat.com/en/breast-milk-fatty-acid-shapes-immune-development-in-mice https://edusehat.com/en/breast-milk-fatty-acid-shapes-immune-development-in-mice Mon, 22 Jun 2026 22:20:10 +0700 Edusehat Breast, Milk, Fatty, Acid, Shapes, Immune, Development, Mice In a new study published in Science titled, “Maternal trans-vaccenic acid shapes neonatal T cell development and early-life immune imprinting,” researchers from the University of Chicago have found that trans-vaccenic acid (TVA), the most abundant trans fatty acid in human breast milk, helps boost immune system development in mice. 

    Nursing female mice that were fed a diet enriched with TVA passed the nutrient to their pups, leading to increased production of immune cells during early development. Genetic analyses showed that TVA exposure during breastfeeding reprogrammed immune cells to improve responses to pathogens. Mice that were nursed on TVA-enriched milk responded faster to infections with viruses or common bacteria, even into adulthood. 

    “It’s common knowledge that breastfeeding is important for neonatal immune development and overall health, but breast milk is so complex that it seems almost impossible that one single molecule would be sufficient to change a baby’s immune development,” said Jing Chen, PhD, professor of medicine at UChicago and co-corresponding author on the study. “So, it was very surprising to see that during this crucial stage of development, one nutrient derived from the mother’s diet and delivered through breastfeeding has such a tremendous effect.” 

    TVA is a long-chain fatty acid found in meat and dairy products from grazing animals such as cows and sheep. The human and mouse body must obtain TVA through diet.  

    Pups who were nursed by mothers with a diet enriched with TVA demonstrated a broader and more effective immune cell population, particularly CD4+ T cells that are important for adaptive immunity. Mice raised on TVA-enriched breast milk responded more quickly and had higher survival rates when exposed to the flu virus or Salmonella. 

    “We saw that only postnatal exposure to TVA through breastfeeding is important to train the neonatal T cells, and this can have long-lasting imprinting effects,” Chen said. “Even in adulthood, when we challenged the mice with influenza, the ones that were exposed to higher TVA levels during breastfeeding responded better when battling the infection.” 

    The team also analyzed TVA levels in breast milk and blood samples from human nursing mothers and infants. They found that higher TVA levels in breast milk were closely linked to higher TVA levels in infants’ blood. In preterm infants, levels of circulating TVA correlated with similar shifts in immune responses seen in mice.

    Higher TVA levels in human breast milk were also associated with reduced risk of bronchopulmonary dysplasia, a chronic inflammatory lung disease that affects premature infants with underdeveloped lungs and increased susceptibility to respiratory infection. 

    Chen hopes for more research on the possibilities for supplementing diets with TVA during pregnancy and breastfeeding, or infant formula. The team will also investigate additional fatty acids and nutrients found in breast milk to understand their benefits. 

    “There are close to 40 fatty acids in total in breast milk, along with hundreds of other components,” Chen said. “So, I think it’s safe for us to say that we believe there could be additional fatty acids and nutrients that can do something similar.” 

    The post Breast Milk Fatty Acid Shapes Immune Development in Mice appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Brain-Infiltrating T Cells Linked to Social Deficits in Autism Mouse Model https://edusehat.com/en/brain-infiltrating-t-cells-linked-to-social-deficits-in-autism-mouse-model https://edusehat.com/en/brain-infiltrating-t-cells-linked-to-social-deficits-in-autism-mouse-model Mon, 22 Jun 2026 22:20:09 +0700 Edusehat Brain-Infiltrating, Cells, Linked, Social, Deficits, Autism, Mouse, Model The prevalence of autism spectrum disorder (ASD) is roughly one in 36 people, with a male-to-female ratio of 4:1. The disorder is known to be influenced by multiple factors, both genetic (gene mutations and copy number variations) and environmental, such as infections during pregnancy. However, the role of immunity in genetic ASD remains unclear.

    One area of interest lies in lymphocytes—cells that are known to shape neurodevelopment and behavior. But their roles in neurodevelopmental disorders are not well defined.

    Now, new research shows that a subset of T cells—γδ T cells—can infiltrate the brain and contribute to changes in social behavior in a genetic mouse model that mimics behavioral features of ASD. Depleting these cells from the brain increased sociability, suggesting that targeting abnormal immune function during neurodevelopment may offer interventions for ASD.

    This work is published in Science Immunology in the paper, “CXCL16-mediated recruitment of γδ T cells to the brain reduces sociability in mice.”

    Infections during pregnancy can induce the release of interleukin-17A (IL-17A) from T helper 17 cells and γδ T cells. Prior research has linked this type of maternal immune activation to neurodevelopmental disorders, but there is a lack of evidence connecting IL-17A and social behaviors in genetic mouse models.

    To investigate this further, a team of researchers from the Division of Allergy and Immunology in the Medical Institute of Bioregulation at Kyushu University, in Fukuoka, Japan, studied 15q11-13 duplication (15q dup) mice—a mouse model that mimics a chromosome duplication found in some humans with ASD. These mice also demonstrate reduced social interactions, behavioral inflexibility, and increased anxiety-like behaviors.

    The team analyzed immune cell populations in the brains of the 15q dup mice. Their findings suggest an increase in γδ T cells in the developing brains when compared with wild-type mice.

    Using single-cell RNA sequencing (scRNA-seq), the team uncovered that this was most likely due to microglia in the brain expressing the chemokine CXCL16, which promotes immune cell migration. CXCL16 was highly expressed in the brains of 15q dup mice and contributed to increased infiltration of γδ T cells.

    In addition, experiments revealed that deleting IL-17A–producing γδ T cells or blocking them with antibodies after birth increased sociability and reduced anxiety-like behaviors in the 15q dup mice.

    Taken together, the authors note that these findings suggest that “immune dysregulation contributes to social behavior deficits in 15q dup mice, consistent with observations in maternal immune activation models, and may represent a potential target for interventions for ASD-associated differences in social behavior.”

    The post Brain-Infiltrating T Cells Linked to Social Deficits in Autism Mouse Model appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     StockWatch: Positive Phase III Data Sells Investors on Intellia https://edusehat.com/en/stockwatch-positive-phase-iii-data-sells-investors-on-intellia https://edusehat.com/en/stockwatch-positive-phase-iii-data-sells-investors-on-intellia Mon, 22 Jun 2026 11:35:08 +0700 Edusehat StockWatch:, Positive, Phase, III, Data, Sells, Investors, Intellia A second positive Phase III data readout in two months is a major reason why Intellia Therapeutics (Nasdaq: NTLA) shares have soared more than 76% over the past six months—including a 29% surge this past week that followed the CRISPR gene editing therapy developer’s lead pipeline candidate lonvoguran ziclumeran (lonvo-z) meeting three key secondary endpoints in a pivotal study in patients with hereditary angioedema (HAE).

    Lonvo-z met the Phase III HAELO trial’s (NCT06634420) primary endpoint, Intellia announced back in April, by showing an 87% reduction (p<0.0001) in mean monthly attacks in the lonvo-z arm vs. the placebo arm during the efficacy evaluation period (weeks 5-28). Lonvo-z also aced the trial’s key secondary endpoint by showing that 62% of the 52 patients in the lonvo-z arm were entirely attack-free and therapy-free for the six-month efficacy evaluation period, vs. just 11% of the 28 patients in the placebo arm (p<0.0001).

    On June 13, Intellia presented and published additional data showing lonvo-z to have achieved positive results on three other key secondary endpoints:

    • Monthly rate of attacks requiring on-demand treatment, Weeks 5–28, mean (0.19 vs. 1.79, 95% CI).
    • Monthly rate of moderate/severe attacks, Weeks 5–28, mean (0.11 vs. 1.23, 95% CI).
    • Change from baseline to Week 28 in AE-QoL total score, mean ( — 23.51 vs. — 6.47, 95% CI).

    Intellia presented the data at the European Academy of Allergy & Clinical Immunology (EAACI) Annual Congress 2026 in Istanbul, Türkiye, and published the results in The New England Journal of Medicine.

    The data was strong enough for Intellia to continue the rolling Biologics License Application (BLA) submission that it began in April. The company expects to complete the BLA filing by year’s end and hopes to gain FDA approval and launch lonvo-z in the first half of 2027.

    “Super pleased”

    “We were super pleased with the results that we saw,” John Leonard, MD, Intellia’s president and CEO, told GEN. “We’re essentially replicating what we’ve seen throughout the program, where most of the patients reached a status of no attacks, no therapies over the course of this extended observation period. For those patients who didn’t, they appeared to be on their way to reaching that kind of a state. And critically important was that every single patient who got the drug was off long-term prophylaxis.”

    “Across the board, and across all subgroups, the drug performed extremely well. So, we think it speaks to physicians, it’s going to speak to payers in terms of how they think about the drug and its ultimate, excellent utility,” Leonard said.

    Investors and analysts appeared to share that enthusiasm this past week, as Intellia’s stock price rose over three of the four trading sessions following the release of data on the secondary endpoints. Intellia shares jumped 23% on June 15, the first trading day since the news, rising from $12.11 to $14.92. After a day of profit-taking that saw shares dip 2.5%, to $14.55, Intellia’s stock resumed its upward trajectory, rising nearly 4.5% to $15.20 on Wednesday, then another 3% Thursday, closing the week at $15.67. Markets were closed on Friday for the Juneteenth holiday.

    Since December 18, 2025, when Intellia shares closed at $8.88, the stock has soared nearly 76.5%, accounting for most of its one-year gain of 62%. Lonvo-z accounted for three of Intellia’s four stock price peaks in 2026: The dosing of the first patient in HAELO, announced January 22, led the stock to climb 13%, from $14.03 to $15.90.

    Shares surged 12% March 2, from $13.78 to $15.44, when the FDA lifted a clinical hold on the company’s Phase III MAGNITUDE trial (NCT06128629) assessing nexiguran ziclumeran (nex-z) in transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The FDA imposed the hold after an elderly patient died during a study of Nex-z, an in vivo CRISPR-based therapy developed in partnership with Regeneron Pharmaceuticals (Nasdaq: REGN) to treat ATTR-CM by inactivating the TTR gene.

    The third peak, an 8% gain from $15.31 to $16.57 on April 22, followed Intellia reporting positive data for lonvo-z, showing that it met HAELO’s primary endpoint, while the fourth peak followed the secondary endpoint announcement.

    Misperceived market

    “Hereditary angioedema has, in the last 10 to 15 years, had a variety of therapies arrive that are better than the ones that were there 20 years ago. Twenty years ago, circumstances were pretty grim for patients with HAE,” Leonard recalled. “I think some investors have looked at this incorrectly as a satisfied market, only because there are other therapies available.”

    Among those therapies are three that won FDA approval last year. Last August, the agency approved Dawnzera® (donidalorsen), a prekallikrein-directed antisense oligonucleotide designed to prevent HAE attacks in patients ages 12+, marketed by Ionis Pharmaceuticals (Nasdaq: IONS). A month earlier, the FDA authorized Ekterly® (sebetralstat), a plasma kallikrein inhibitor indicated for the treatment of acute attacks of HAE in patients ages 12+, marketed in the United States by KalVista Pharmaceuticals (Nasdaq: KALV).

    And in June 2025, the FDA approved Andembry® (garadacimab-gxii), an activated Factor XII (FXIIa) inhibitor (monoclonal antibody) and the first long-term prophylactic HAE treatment designed to target Factor XIIa, administered as a once-monthly subcutaneous injection for patients ages 12+, marketed by CSL Behring, the largest business unit of Australian-owned CSL (ASX: CSL).

    “What we’re showing is that a lot of efficacy and a lot of utility has been left on the table, and that it’s possible for patients to get pretty close to something resembling a normal person who does not have HAE and all of the things, benefits that come with that,” Leonard said. “As people have looked at the data more completely, I think they’re seeing more and more that that’s the case, and maybe some of those original premises that they had are not quite correct.”

    In research notes, three analysts said Intellia’s latest data strengthened the company’s future case to regulators for pursuing approval of lonvo-z as a one-time HAE treatment.

    “We view these data as furthering Intellia’s case for regulatory approval following its expected completion of a rolling BLA,” Myles R. Minter, PhD, a partner and biotechnology analyst with William Blair, wrote June 15.

    A day earlier, Jefferies equity analyst Maury Raycroft, PhD, commented that Intellia’s latest data will help lonvo-z gain more than a foothold in the HAE market.

    “Positive implications”

    “Big picture, we believe total HAE data have positive implications for commercial positioning” of lonvo-z, Raycroft wrote. “Editing is expected to be durable (we have seen longer term ph.I/II data out to 3-yrs); therefore, NTLA’s approach could eliminate need for lifelong chronic tx [therapy], justifying the value proposition of a 1X tx, despite competition in a crowded HAE space.”

    Raycroft cited market research from Intellia showing that 64% of surveyed patients on LTPs [long-term prophylaxis drugs] are extremely likely to transition to a one-time therapy, while 54% surveyed docs expressed intent to prescribe such a treatment.

    Mani Foroohar, MD, senior managing director, genetic medicines, and a senior research analyst with Leerink Partners, said Intellia’s latest results “again demonstrate lonvo-z’s clean safety and best-in-class efficacy and convenience.”

    Writing in NEJM, the team of HAELO investigators reported no serious adverse events in patients treated with lonvo-z: “The most common adverse reactions were infusion-related reactions, which were generally transient and resolved without intervention. Elevated levels of serum aspartate aminotransferase and alanine aminotransferase, which occurred in approximately 10 to 15% of patients treated with lonvo-z, were transient, asymptomatic, and resolved without intervention.”

    Foroohar sided with optimistic investors over their pessimistic counterparts in arguing that patients will warm up to a one-time treatment, though it will likely be costlier than current therapies.

    Bears and bulls

    “Bears argue limited patient demand to move up the innovation curve in a market with several approved treatments. We take the other side of this and see onetime therapy (vs lifetime chronic dosing) and patient desire to be attack-free as potent tailwinds to adoption,” Foroohar wrote. “Longer follow-up and crossover data (caveat – small n [number of patients studied]) provide an early glimpse at the improving profile of lonvo-z over time. We look to more data ahead of 1H27 launch to further educate physicians/patients.

    “Subgroup analyses demonstrate clear benefit across all patient populations (prior LTP use, historical attack severity/frequency, etc.), supporting broad uptake as SoC [standard of care] across HAE—recognizing this will take time to play out as physicians gain comfort with this (likely) first approved in vivo gene editing therapy,” Foroohar added.

    Intellia has not set a price for lonvo-z.

    “We have said publicly we’re not going to set any new records beyond prices that have been precedented,” Leonard said.

    HAE patients, he continued, “are some of the most costly patients that payers have. They’re small in number, but high in cost, with the therapies they take and their healthcare resource utilization exceeding $1 million a year.

    “When you consider that these are patients that are oftentimes treated, or first diagnosed in adolescence or young adulthood, the lifetime costs are frighteningly high,” Leonard explained. “We are confident that, and we have this as an intended outcome, that we will save lifetime health, resources in very, very substantial terms, in a way that payers see and can recognize. We want to make it easy for patients to get onto the therapy, and we want to make it very competitive, cost-competitive for physicians taking care of them.”

    Leaders and laggards

    • Elicio Therapeutics (Nasdaq: ELTX) shares plunged 72.5% from $14.85 to $4.08 on June 15 after the developer of immunotherapies to treat high-prevalence cancers said it was evaluating multiple strategic financing and partnership opportunities to advance its planned Phase III adjuvant pancreatic cancer immunotherapies program and broader AMP platform. The action came after ELI-002 7P, a 7-peptide formulation of its lead candidate ELI-002, failed the Phase II AMPLIFY-7P trial (NCT05726864) in patients with mKRAS-driven pancreatic ductal adenocarcinoma (PDAC). ELI-002 7P missed the pre-specified primary endpoint of disease-free survival (DFS) in the intent-to-treat population. Elicio said the ELI-002 7P arm had a higher proportion of R1 resected (higher residual disease) patients vs. the observation arm (19% vs. 10%), and that post-hoc analyses showed significant DFS improvement (R0: HR 0.65, p=0.048) in the 121 lower residual disease (R0 completely resected) patients, a subgroup representing approximately 84% of enrolled patients. Elicio said the trial results will shape a Phase III strategy focused on a defined R0 resected population and additional ELI-002 7P dosing.
    • Neumora Therapeutics (Nasdaq: NMRA) shares plummeted 49% from $1.78 to 91 cents on June 15 after the brain disease drug developer said it was chopping its workforce by approximately 35% or about 34 jobs, ending development of its major depressive disorder (MDD) candidate navacaprant, and refocusing on advancing the rest of its pipeline. The actions came after navacaprant missed statistical significance on the primary and key secondary endpoints of the Phase III KOASTAL-2 trial (NCT06058013) and KOASTAL-3 trial (NCT06058039) in MDD. The primary endpoint was the change from baseline to week 6 on the Montgomery-Åsberg Depression Rating Scale (MADRS).  Neumora projected the job cuts would save it approximately $10 million annually, to be partially offset this year by approximately $2 million in one-time restructuring costs. Neumora said current cash and cash equivalents are expected to provide runway into Q3 2027, including multiple expected key clinical milestones. Neumora’s pipeline includes NMRA-511 in Alzheimer’s disease agitation, NMRA-898 in schizophrenia, and NMRA-215 in cardiometabolic disease.
    • uniQure (Nasdaq: QURE) shares zoomed 78% from $26.99 to $48.16 Wednesday after the gene therapy developer announced the FDA’s revised position that a three-year analysis from its two-trial, Phase I/II study (United States, NCT04120493, and Europe (NCT05243017) of AMT-130 in Huntington’s disease was now acceptable as the primary basis of a Biologics License Application (BLA) for accelerated approval of the gene therapy. Researchers hailed “game-changing” data last year showing significant slowing of Huntington’s disease (HD) progression, but the FDA disagreed while its Center for Biologics Evaluation and Research (CBER) was headed by Vinayak (Vinay) Prasad, MD, who resigned in April. uniQure said the FDA seeks to align on the confirmatory study design prior to the BLA submission, including considering allowing concurrent control on standard-of-care therapy instead of a sham procedure. “FDA communicated that they would work as expeditiously as possible with uniQure on this effort. The company is committed to conducting the confirmatory study without delay and expects to further align with the FDA on the details of such a study prior to BLA submission,” uniQure stated.

    The post StockWatch: Positive Phase III Data Sells Investors on Intellia appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Brain-computer interface trials are taking off https://edusehat.com/en/brain-computer-interface-trials-are-taking-off https://edusehat.com/en/brain-computer-interface-trials-are-taking-off Fri, 19 Jun 2026 22:35:03 +0700 Edusehat Brain-computer, interface, trials, are, taking, off
  • A "first power user" of brain implants: Casey Harrell, paralyzed and unable to speak coherently due to ALS, has spent nearly three years using a brain-computer interface to work, reconnect with loved ones, and read to his daughter — calling it "nothing short of revolutionary."

  • The field is rapidly expanding: Since 2024, the number of people implanted with brain electrodes has more than doubled to an estimated 150, with companies like Neuralink, Synchron, and China's Neuracle all running active trials.

  • The technology keeps getting better — but questions remain: BCIs have evolved from basic cursor control to full speech decoding with voice cloning, yet researchers still don't fully understand why devices eventually stop working in some patients
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    This week, I covered the story of Casey Harrell—a man with ALS who is “the first power user” of a brain implant, according to the researchers who worked with him. Harrell is paralyzed and unable to speak coherently without the device. He has now spent almost three years using a brain-computer interface (BCI) that enables him to “speak,” surf the web, and perform his job as a climate activist, largely independently.

    Since Harrell was implanted with the device, in July 2023, a team at the University of California, Davis, has worked with him to adjust and improve its offerings. They’ve refined its accuracy, for example. And they’ve introduced settings including a privacy mode and a “profanity filter” that lets Harrell talk to his daughter without risking accidental swearing.

    Harrell told me that, for him, the device is “nothing short of revolutionary!” It has enabled him to maintain an income, reconnect with friends and family, and read to his daughter. 

    The team that developed his BCI is one of several working on ways to use technology to allow people with paralysis to communicate, engage with the online world, and regain some independence. And Harrell is one of a growing number of people volunteering their brains to, as he puts it, “pay it forward and do the scientific research … [and] get some personal benefit.”

    Over the past couple of years, the number of BCI trial volunteers has soared. This year, China became the first country to approve a BCI for medical use. Advances in technology are allowing engineers to provide more features than ever. BCI research is properly taking off.

    I should first point out that BCIs come in different forms. Harrell’s device includes a set of electrodes embedded in his brain that pick up the electrical activity associated with speech. Those electrodes are connected to two docking ports on top of his head that can be plugged into a computer.

    That computer is loaded with software trained to decode his brain signals into phonemes (units of sound in speech) and predict what Harrell wants to say. He can then use an eye gaze tracker to make any corrections before the speech is played out loud.

    But some BCIs don’t need to be “plugged in”—they’re fully implanted and wireless. Others are less invasive; they might involve placing wired electrodes on the surface of the brain or simply wearing a cap of electrodes, for example. There are trade-offs—the closer you get to the neurons you want to record from, the better your signal will be. But generally speaking, the more invasive the surgery, the higher the risk of complications.

    BCIs can also have different functions. Harrell has ALS, but most BCIs in use today are sitting in the brains of people with spinal cord injuries. Typically, these individuals have some degree of paralysis; for example, they may be unable to move their arms and legs, but their face and ability to speak are unaffected. In those cases, BCIs can be used to control other kinds of devices that might help with mobility.

    In 2024, Michelle Patrick-Krueger, then at the University of Houston, and her colleagues published a roundup of all trials of BCIs conducted between 1998, which is when they believe the first device was implanted, and the end of 2023. They identified 21 research groups that, among them, had trialed BCIs in a total of 67 volunteers.

    “Since then, that number has increased a lot,” says Mariska Vansteensel, a BCI researcher at University Medical Center Utrecht. In January, Neuralink (the BCI company founded by trillionaire Elon Musk) announced that it has implanted 21 people with its device in the past two years.

    Synchron, another BCI company, is currently testing its devices in trials in North America and Australia. Shanghai-based Neuracle has been trialing a BCI since November 2024, and it recently obtained approval for the device to be used outside of clinical trials. Precision Neuroscience, cofounded by a former co-creator of rival Neuralink, is also trialing its BCI, which sits on the surface of the brain.

    At the same time, academic research has continued. The UC Davis team that worked with Harrell is part of BrainGate—a BCI research effort that has been running for the past two decades. Other academic teams are exploring a variety of devices, from the fully implanted to the minimally invasive.

    Since 2024, when Patrick-Krueger’s paper was published, the number of people who have been implanted with a brain electrode has more than doubled, according to Vansteensel. “My current estimation would be around 150 people,” she says.

    The technology is improving too. Take the BrainGate trial, for example. The first 17 years of that trial focused on the use of what researchers call “point-and-click” communication—allowing users to control a cursor and “click” with their brain activity. But in recent years the team has pivoted toward decoding speech, says David Brandman, the lead investigator on the team (and the person who implanted Harrell’s electrodes). Today, Harrell’s device uses a voice clone—the speech it produces is based on previous recordings of Harrell’s voice.

    But BCIs are still experimental. And plenty of questions remain about who might benefit from them—and how long the devices will last. So far, most BCIs have been implanted in people with spinal cord injuries. We know even less about how they might benefit other people who have ALS, for example. In some cases where the devices initially helped people with ALS—even someone who was completely locked in—the BCIs eventually stopped working. And scientists don’t really know why.

    The only way they’ll find out is through more research—and the participation of volunteers like Harrell. So it’s exciting to see trials truly take off. And I promise I’ll update you on where they stand two years from now.

    This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.

    ]]>     New report details how U.S. can maintain biotech competitiveness https://edusehat.com/en/new-report-details-how-us-can-maintain-biotech-competitiveness https://edusehat.com/en/new-report-details-how-us-can-maintain-biotech-competitiveness Fri, 19 Jun 2026 04:35:03 +0700 Edusehat New, report, details, how, U.S., can, maintain, biotech, competitiveness The Reagan-Udall Foundation for the FDA just released a new report on strengthening early-stage drug development in the United States. The report reflects insights shared by academic medical centers, biopharmaceutical companies, clinical research organizations (CROs), patient organizations, and regulatory agencies in a March 2026 roundtable. Biotechnology Innovation Organization (BIO) provided funding for the project.

    The report comes as China accelerates its drug development capabilities, challenging the United States’ historical biotech leadership. To maintain global competitiveness—and continue developing innovative medicines for American patients—the United States must strengthen its support for research and development to enable a more efficient pathway for innovative treatments to reach patients.

    The report details key challenges to early-stage U.S. drug development and lays out a number of core recommendations to address them.

    Current challenges

    The United States is on the verge of losing its historic lead in drug development. Our share of the global pharmaceutical pipeline has decreased from 47% in 2013 to 36% in 2025, barely ahead of China.

    The U.S. also has recorded an outright decline in early-stage “Phase 1” clinical trials. The report observes that clinical development has become more difficult and expensive, and that existing regulatory frameworks are slow to adapt to these new challenges. That, in turn, could lead to a reduction in new therapies—and a dependence on China and others for key treatments. The United States must strengthen support and incentives for early-stage drug development.

    Key recommendations from the report

    • Modernize Investigational New Drug Requirements and Processes: Updating submission requirements, improving review processes, and clarifying data requirements for Investigational New Drugs would allow companies to begin human testing for promising therapies more quickly while maintaining high safety standards.
    • Modernize Phase 1 Clinical Trials and Tools: More innovative clinical trial designs that include patient perspectives earlier and allow AI-enabled tools can improve Phase 1 Trials.
    • Optimize FDA–Sponsor Early-Stage Engagements and Processes: Increasing communication and clarifying FDA review requirements would reduce uncertainty for clinical trial sponsors as they pursue early-stage drug development.
    • Build a Streamlined and Dedicated U.S. Phase 1 Infrastructure: Building and funding a network of Phase 1 trial sites could streamline the clinical trial process, allowing greater early-stage development.
    • Mitigate Litigation Risk for Phase 1 Clinical Trial Sites: Exploring reforms that lower unnecessary risks for trial sponsors could reduce barriers to pursuing clinical trials while maintaining rigorous standards for patient safety.

    To maintain global competitiveness and preserve patient access to promising new medicines, the United States must strengthen support for cutting-edge R&D. That starts with removing barriers and actively incentivizing the early-stage trials that lead to breakthrough therapies.

    Read the report.

    The post New report details how U.S. can maintain biotech competitiveness appeared first on Bio.News.

    ]]>     Historic Biotech IPO, Merck, Protillion’s AI Deal, Testing a Lassa–Rabies Vaccine https://edusehat.com/en/historic-biotech-ipo-merck-protillions-ai-deal-testing-a-lassarabies-vaccine https://edusehat.com/en/historic-biotech-ipo-merck-protillions-ai-deal-testing-a-lassarabies-vaccine Fri, 19 Jun 2026 04:30:10 +0700 Edusehat Historic, Biotech, IPO, Merck, Protillion’s, Deal, Testing, Lassa–Rabies, Vaccine We are still talking about big pharma deals and biotech fundraising in this episode. The big news this week was Parabilis Medicines’s history-making IPO. We dive into the drug developer’s plans for the eye-popping $770.5 million that it raised. Next, we discuss the details of a collaboration between Merck and Protillion Biosciences to use artificial intelligence to discover multiple therapeutic candidates. Turning to some newly published research, we discuss the early results of a first-in-human clinical trial that is testing a dual vaccine against Lassa fever and rabies, a CRISPR system engineered to selectively trigger cancer cell death by chromatin shredding, and a novel mRNA delivery platform for delivering gene therapies starting with Duchenne muscular dystrophy.

     

     

    Listed below are links to the GEN stories referenced in this episode of Touching Base:

    StockWatch: Parabilis Medicines Makes Wall Street History with $770.5M IPO
    By Alex Philippidis, GEN Edge, June 14, 2026

    Merck, Protillion Launch AI Drug Discovery Collaboration with Up-to-$510M in Milestone Payments
    By Alex Philippidis, GEN Edge, June 16, 2026

    First-in-Human Trial Reports Promising Dual Lassa–Rabies Vaccine Data
    GEN, June 9, 2026

    CRISPR Shreds Undruggable Cancer Cells with Precision
    By Fay Lin, PhD, GEN Edge, June 8, 2026

    New mRNA Delivery Platform Restores Muscle Function in DMD Models
    GEN, June 11, 2026

    Touching Base Podcast
    Hosted by Corinna Singleman, PhD

    Behind the Breakthroughs
    Hosted by Jonathan D. Grinstein, PhD

    The post Historic Biotech IPO, Merck, Protillion’s AI Deal, Testing a Lassa–Rabies Vaccine appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Impact of Early Life Adversity on Epigenome at Molecular Level Mapped in Macaques https://edusehat.com/en/impact-of-early-life-adversity-on-epigenome-at-molecular-level-mapped-in-macaques https://edusehat.com/en/impact-of-early-life-adversity-on-epigenome-at-molecular-level-mapped-in-macaques Fri, 19 Jun 2026 04:30:09 +0700 Edusehat Impact, Early, Life, Adversity, Epigenome, Molecular, Level, Mapped, Macaques Research headed by scientists at Arizona State University and Vanderbilt University suggest that experiences we face early in life may leave their marks on our health in ways that echo across decades, and even across the entire body.

    The team’s study involves a unique group of free-living rhesus macaques who have been followed their entire lives to document their experiences. Pairing the animals’ histories with genomic data from 12 tissues collected in adulthood, the study has generated some of the clearest molecular evidence yet that early life adversity (ELA) leaves a lasting, system-wide impression at the epigenome, the biological layer on top of the human genome that regulates gene activity.

    The team examined DNA methylation (DNAm) patterns, which can represent telltale aging hallmarks of the epigenome. DNA methylation is one of the most well-studied markers of aging and can be used to build “epigenetic clocks” that estimate both an organism’s chronological age—how long it has been alive—and biological age, which is how old it appears physiologically. Through their newly reported study, the researchers developed highly precise tissue-specific clocks, capable of predicting age within about one year of an individual’s chronological age.

    Their findings challenge a common assumption that early adversity uniformly accelerates biological aging. Instead, the results suggest a more nuanced model, in which early experiences alter the trajectory of molecular aging, amplifying the effects of aging in some tissues, such as the pituitary, but not others. These findings further suggest that the well-documented effects of early adversity on health operate, at least in part, through mechanisms that are not directly linked to aging.

    “Our goal was to understand how aging unfolds across the body, and how early experiences might influence that process,” said Noah Snyder-Mackler, PhD, a professor in Arizona State University’s School of Life Sciences. “What we found is that early life adversity leaves a coordinated epigenetic signature that spans multiple tissues—but it doesn’t simply accelerate aging in a uniform way.”

    Snyder-Mackler is co-senior author of the team’s published paper in Science, titled “Age and early life adversity shape heterogeneity of the epigenome across tissues in macaques.” In their research article summary the team concluded “By generating multi-tissue DNAm data across the life course in animals with known social histories, we reveal a fundamental contrast in epigenomic remodeling: Age-associated epigenetic variations are highly tissue dependent, whereas the molecular effect of ELA represents a more coordinated, organism-wide response.”

    Aging is universal, the authors wrote, but the pace of decline varies significantly both between and within individuals. “Identifying early patterns and biomarkers of aging—before overt pathophysiology—could enable earlier clinical intervention, and refining how patterns are linked between organs may clarify which aging hallmarks are shared across tissues versus which must be assayed in specific organs to predict disease.”

    ELA has been linked to age-related diseases and reduced lifespan in both humans and other social animals. However, the team noted, “… we still know little about how early life exposures shape the biological mechanisms of aging across tissues, especially at the molecular level.” This is difficult to study in humans, because detailed life course information in combination with multi-tissue molecular data is very rare.

    For their reported research the team studied 237 macaques, who live in semi-natural conditions on Cayo Santiago (colloquially referred to as “Monkey Island”), a 38-acre island off Puerto Rico’s east coast. The island is inhabited by over 1,500 free-ranging rhesus macaques and managed by the University of Puerto Rico and Caribbean Primate Research Center. By integrating multi-tissue DNA methylation collected in adulthood with detailed records of early life experiences, the team uncovered how adversity and aging interacted to shape biology at the molecular level.

    “We focused on DNAm, an epigenetic modification that is one of the most studied hallmarks of aging,” the authors wrote. “… DNAm can be used for estimating ‘biological’ age using ‘epigenetic clocks,’ a measure linked to disease and mortality.” The team generated a dataset integrating multi-tissue DNAm profiles with extensive data on social and environmental conditions during the animals’ development, creating the opportunity to examine how age and early life adversity affect DNAm across the body.

    What they found was that despite the epigenetic precision, aging did not occur uniformly across the body. Instead, the researchers found that age-related changes in DNA methylation were highly tissue dependent. Yet even amid this diversity, individuals showed a degree of internal consistency. Animals that appeared “biologically older” in one tissue tended to appear older in other tissues as well, suggesting that aging operates as a partially coordinated process across the body.

    “Age-associated DNAm was predominantly tissue dependent, yet tissue-specific epigenetic clocks showed that epigenetic aging was relatively consistent within individuals,” the investigators stated. Co-senior author Amanda Lea, PhD, assistant professor of biological sciences at Vanderbilt University, added, “At a molecular level, aging looks very different depending on which tissue you examine,” said. “Blood, which is most commonly measured in human studies, only captures part of the picture.” Some tissues, like the thymus and pituitary gland, showed particularly strong and distinct age-related patterns, while others exhibited more subtle changes.

    The study’s most novel insights came from examining early life adversity—defined through naturally occurring conditions such as maternal loss, low maternal social status, or growing up in a crowded social group. These experiences were not only associated with changes in DNA methylation, but in a strikingly coordinated way across tissues. “We found that each type of adversity tends to affect specific regions of the genome,” said Lea. “But once it targets those regions, the effects are often shared across multiple tissues.”

    In total, the team identified thousands of genomic regions where DNA methylation was associated with early life adversity. These regions frequently overlapped with those affected by aging—but importantly, the direction of the effects was not consistent. “In some cases, adversity-related changes looked like accelerated aging. In others, they went in the opposite direction,” explained co-lead author Rachel Petersen, PhD, a Vanderbilt postdoctoral researcher. “This tells us that early adversity doesn’t simply ‘speed up’ aging.

    “Instead, it reshapes the epigenome in more complex ways.” In their paper the researchers note, “Although ELA targeted many of the same loci as age, the directions of effects differed, which indicates that ELA does not uniformly increase epigenetic age.” Instead, they continued, “… ELA leaves a coordinated, cross-tissue epigenetic signature that is distinct from—yet intertwined with—age-related differences, which advances our understanding of how early environments sculpt the molecular foundations of aging and disease.”

    The study also highlights the importance of studying multiple tissues. Many previous studies have relied on blood samples, which are relatively easy to collect. However, the new findings show that this approach may miss critical aspects of how aging and environmental exposures affect the body. “Different tissues have their own epigenetic landscapes and respond differently to both age and adversity,” said co-lead author Baptiste Sadoughi, DVM, an ASU postdoctoral researcher. “To fully understand health and disease, we need to take a whole-body perspective.”

    The use of rhesus macaques, which share many biological and social similarities with humans, adds to the study’s relevance. Unlike laboratory animals, these macaques live in complex social environments, allowing researchers to capture naturally occurring variation in life experiences. “This kind of dataset is incredibly rare,” said Lea. “It allows us to connect detailed life histories with molecular changes across the body in a way that simply isn’t possible in most human studies.” In their research article summary the team noted that their collective findings “…  advance our understanding of how early environments sculpt the molecular foundations of aging and establish this comprehensive tissue atlas as a valuable resource for the scientific community.”

    Beyond its scientific contributions, the research has important implications for understanding the developmental origins of health and disease. By showing how early experiences shape the epigenome across tissues, it provides a potential mechanism linking childhood conditions to later-life outcomes. “Early life is a critical window for biological development,” said Snyder-Mackler. “Our findings suggest that experiences during this period can leave lasting marks on the genome that influence health trajectories over the lifespan.”

    At the same time, the complexity of the results offers a note of caution. Because all types of adversity do not have uniform effects, predicting long-term consequences will require a more detailed understanding of context, timing, and individual variation. “This is not a simple story,” Lea said. “But that’s what makes it exciting. We’re beginning to see how life experiences are written into our biology—and why those signatures might vary within and between individuals.” As researchers continue to explore the interplay between environment, epigenetics and aging, studies like this one are helping to redefine what it means to grow older—not just as a function of time, but as a dynamic process shaped by the unique experiences that can truly define our lives.

    The post Impact of Early Life Adversity on Epigenome at Molecular Level Mapped in Macaques appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     China Sets Framework for Advanced Therapeutic Development https://edusehat.com/en/china-sets-framework-for-advanced-therapeutic-development https://edusehat.com/en/china-sets-framework-for-advanced-therapeutic-development Fri, 19 Jun 2026 00:55:11 +0700 Edusehat China, Sets, Framework, for, Advanced, Therapeutic, Development China’s Order 818—Regulations on the Administration of Clinical Research and Clinical Translation and Application of Biomedical New Technologies—establishes a new commercialization pathway for cell and gene therapeutics, gene editing, and other advanced therapeutics. More importantly, it signals China’s intent to accelerate local innovation and standardize the drug development environment, thereby becoming a more important player in advanced therapy development and manufacturing.

    Order 818 allows therapeutics whose mechanisms of action are at the cellular or molecular level to be clinically translated at 3A hospitals (tertiary care hospitals, of which there are approximately 1,700) without requiring National Medical Products Administration (NMPA) drug registration. Once translational approval is granted, hospitals may begin charging patients for these treatments.

    A good move

    While the move appears to be viewed positively by the biopharma industry, some details are still being ironed out. For example, boundary delineation guidelines still need to be issued to specify which technologies fall under Order 818 and which remain under the NMPA’s purview. Interpretations of the order generally suggest that therapies intended for mass manufacturing and wide distribution will be governed by the NMPA, while personalized therapeutics may use the Order 818 pathway.

    “We definitely see this as a positive signal,” Boyang Wang, founder of Singapore-based global longevity fund Immortal Dragons, tells GEN. Because advanced therapeutics are intricate and often personalized, it’s quite difficult for them to use the standard NMPA pathway. Before Order 818 was enacted on May 1, “biomedical companies in China would initiate investigator-initiated trials and partner with any training medical institution of their choice.”

    Order 818 provides a standardized structure that ensures only hospitals capable of advanced therapy development are involved. It also provides a one-to-five-year risk observation window before the technology can shift from the tech track to a drug track, Wang says. That effectively provides a level of Chinese exclusivity before the therapeutic developer can file for approval with regulators outside China.

    Ramifications

    For international firms partnering with Chinese companies to develop advanced therapeutics, the new regulatory pathway mainly triggers a review of existing contracts and milestone clauses.

    The greatest disruption may be for Chinese companies that are in the midst of investigator-initiated trials for advanced therapies at smaller institutions. This affects contract research and manufacturing organizations, too, who are producing materials, such as stem cells, for investigator-initiated trials.

    lab researchers
    Boundary guidelines are pending, but Order 818 is expected to cover personalized therapies, while mass-market products remain under NMPA oversight. [STAP/Getty Images]
    Speaking to GEN, Todd Liao, partner at Morgan Lewis & Bockius’s Singapore office, emphasizes the need for foreign companies invested in China to review their contracts.

    “Legacy agreements were written for a single-pathway world that no longer fully describes the Chinese landscape. If milestones are defined solely by NMPA events, a licensee commercializing [a therapeutic] through hospitals may never trigger them.” The solution, he says, “is to redefine milestones around clinical and commercial outcomes [such as] ‘first fee-paying patient’ rather than ‘first NMPA-approved commercial sale.’”

    Liao says he expects “a wave of proactive contract amendments. It becomes contentious only if ignored.” Any disputes, he adds, will most likely be tried internationally rather than in Chinese courts.

    To succeed under this dual-pathway system, Liao advises multinational corporations to define success by what happens in the clinic and the market, not the regulatory pathway. Therefore, he says:

    • Use pathway-agnostic milestone language
    • Require the Chinese partner to notify you before selecting the translation pathway so you can assess the global intellectual property (IP) and data implications
    • Address human genetic resources’ joint IP requirements upfront to maintain global commercial control

    “The overall regulatory direction is actually loosening, not tightening,” Liao says, citing new, “centralized oversight for clinical research and clinical transformation applications of biomedical new technologies.”

    As he elaborates, “Notably, on May 8, 2026, the National Health Commission released a consultation draft that proposes to exclude pure clinical data, imaging data, and protein data from human genetic resources restrictions entirely, limiting the scope to nucleic acid sequence data only. It also introduces same-day filing confirmations for international clinical trials.”

    Unless and until that proposal is enacted, export controls for Chinese human genetic resources remain strict, which may affect data-sharing arrangements for non-Chinese companies seeking to license or acquire Chinese cell and gene therapies. Basically, under current laws, Wang says, human genetic resource data “can never leave Chinese soil.” Analyses based on that data may be less restricted, and the differences can be subtle. Understandably, the close collaborations with 3A hospitals required under Order 181 could complicate data export decisions.

    pharma vials
    Advanced therapies such as CAR T, cell therapy, and gene therapy require highly coordinated systems—regulatory clarity, manufacturing standards, hospital infrastructure, quality oversight, and predictable development pathways. [Zorazhuang/Getty Images]
    Additionally, the applicability of Order 818 to free trade zones has not yet been specified. Currently, the 2024 Foreign Investment Negative List prohibits multinational companies from investing in China’s cell and gene therapy development at a national level. Such investment is allowed, however, within the free trade zones of Beijing, Shanghai, Guangdong, and Hainan, but only under the NMPA product registration pathway. The so-called Negative List has not yet been updated to reflect the Order 818 pathway.

    “Advanced therapies such as CAR T, cell therapy, and gene therapy require highly coordinated systems—regulatory clarity, manufacturing standards, hospital infrastructure, quality oversight, and predictable development pathways,” Jeremy Levin, PhD, chairman of Ovid Therapeutics and Opthera, chairman Emeritus of BIO, and author of the recently released Biotech in the Balance: Saving a Strategic Industry in an Age of Distrust, points out. “China is clearly trying to standardize and industrialize that environment.”

    “If implemented consistently, that could make China a more attractive environment for advanced therapeutics development, and potentially accelerate local innovation, manufacturing, and partnerships,” Levin says. “China intends to compete at the highest levels of biotechnology over the long term.”

    While some of the points of Order 818 are being refined in terms of their interrelationships with other trade regulations, “the clear signal is that biotech, healthcare, and biomedical technologies are at the top of senior government officials’ priority list,” Wang says. “They want to develop this sector. They want investment, and they want to leverage international capital and expertise. If any of the terms of this [order] will stifle innovation development, they will likely make modifications.”

     

    The post China Sets Framework for Advanced Therapeutic Development appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     IDBS and Alchemi Agree to Accelerate AI-Driven Biopharma Regulatory Filings https://edusehat.com/en/idbs-and-alchemi-agree-to-accelerate-ai-driven-biopharma-regulatory-filings https://edusehat.com/en/idbs-and-alchemi-agree-to-accelerate-ai-driven-biopharma-regulatory-filings Fri, 19 Jun 2026 00:55:09 +0700 Edusehat IDBS, and, Alchemi, Agree, Accelerate, AI-Driven, Biopharma, Regulatory, Filings IDBS, which provides cloud software for biopharma, and Alchemi, an applied AI company, partnered to connect AI agents to data captured and contextualized in the IDBS Polar platform across the drug development lifecycle. By capturing such data at the point of creation within a single governed backbone, Polar delivers the data foundation that makes agentic AI viable in regulated environments, explains IDBS in a statement.

    Preparing regulatory filings remains one of biopharma’s most persistent bottlenecks. CMC teams spend months assembling data, drafting reports, and reconstructing process histories, while current AI tools often break the compliance chain by pulling data out of validated systems, according to an IDBS spokesperson, adding that connecting Alchemi’s purpose-built agents to Polar’s AI-ready data foundation keeps validated data traceable and auditable.

    CMC technical reports, clinical study reports, and submission dossiers can be drafted faster and routed through human-in-the-loop workflows for review and sign-off, maintaining compliance, points out the IDBS official. According to Alchemi, in customer deployments across biopharma, teams have produced documents of this type up to 70% faster using Alchemi’s agents.

    “A regulatory filing that takes a team weeks, our agents draft in minutes, at submission-ready quality, with the compliance trail intact, because they work straight from governed data in Polar,” reports Anuj Chadha, co-founder, Alchemi.

    “Our mission is to accelerate life-changing therapies to patients, and as part of Danaher, that means turning ideas into impact with speed and certainty,” says Pietro Forgione, general manager, IDBS. “[Our company] delivers a governed data backbone that makes AI-ready data available across the biopharma lifecycle. By partnering with Alchemi, we can accelerate critical regulatory milestones using high-quality, compliant AI agents,” said Pietro Forgione, general manager, IDBS.

    The post IDBS and Alchemi Agree to Accelerate AI-Driven Biopharma Regulatory Filings appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Nirrin Technologies and S.T. Japan Partner on Next-Gen Protein Quantitation Tech https://edusehat.com/en/nirrin-technologies-and-st-japan-partner-on-next-gen-protein-quantitation-tech https://edusehat.com/en/nirrin-technologies-and-st-japan-partner-on-next-gen-protein-quantitation-tech Thu, 18 Jun 2026 21:20:10 +0700 Edusehat Nirrin, Technologies, and, S.T., Japan, Partner, Next-Gen, Protein, Quantitation, Tech Nirrin Technologies appointed S.T. Japan as its exclusive distribution partner for the TALOSTM protein quantitation system in Japan. The Japanese company will purchase a TALOS demonstration system, establishing local evaluation capabilities that will allow prospective customers throughout Japan to assess the technology using their own samples and workflows.

    “Japan is one of the world’s leading centers for biopharmaceutical innovation and manufacturing,” according to Bryan Hassell, PhD, CEO of Nirrin Technologies. “S.T. Japan has built a strong reputation for helping customers adopt advanced analytical technologies through deep technical expertise and exceptional support.”

    TALOS is designed to replace traditional variable pathlength UV workflows with a simpler fixed-path near-infrared measurement approach, noted Hassell. By directly measuring absorption from the peptide backbone, TALOS enables rapid protein quantitation across a wide concentration range without dilution, protein-specific calibration models, extinction coefficient determination, or manual pathlength adjustment, he explained.

    “TALOS represents a unique advancement in protein quantitation, enabling rapid, consistent measurements with operational simplicity. These capabilities make it particularly well-suited for modern bioprocessing workflows, from early process development through GMP manufacturing,” said Takao Nakagawa, president, S. T. Japan. “As Japan’s biopharmaceutical industry continues to expand and evolve, TALOS has the potential to make a significant impact, driving efficiencies and reducing complexity across the drug development and manufacturing continuum.”

    The post Nirrin Technologies and S.T. Japan Partner on Next-Gen Protein Quantitation Tech appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Perseverance, Persistence Key to CAR T Success, Say Ross Prize Winners Carl June and Michel Sadelain https://edusehat.com/en/perseverance-persistence-key-to-car-t-success-say-ross-prize-winners-carl-june-and-michel-sadelain https://edusehat.com/en/perseverance-persistence-key-to-car-t-success-say-ross-prize-winners-carl-june-and-michel-sadelain Thu, 18 Jun 2026 21:20:09 +0700 Edusehat Perseverance, Persistence, Key, CAR, Success, Say, Ross, Prize, Winners, Carl, June, and, Michel, Sadelain NEW YORK CITY — Patients, colleagues and peers gathered in midtown Manhattan last week to celebrate Carl June, MD, and Michel Sadelain, MD, PhD, who shared the 13th annual Ross Prize in Molecular Medicine.

    The prize, which is made possible by the generosity of Feinstein Institutes board vice chairman Jack Ross and his wife, Robin, assistant vice president of principal gifts at the Northwell Foundation, recognizes biomedical scientists whose discoveries have transformed how medicine is practiced. Established in 2013, it is awarded annually through the Feinstein Institutes’ peer-reviewed, open-access journal Molecular Medicine.

    June and Sadelain are both well-known in immunotherapy circles but could not disguise their delight at being recognized for their pioneering work in developing CAR T-cell therapy for cancer treatment.

    June is an immunologist and cancer researcher at the University of Pennsylvania’s Perelman School of Medicine. He serves as the director of both the Center for Cellular Immunotherapies and the Parker Institute for Cancer Immunotherapy at Penn. Sadelain, who holds dual French and Canadian citizenship, is a professor of medicine at Columbia University’s Vagelos College of Physicians and Surgeons, where he directs the Columbia Initiative in Cell Engineering and Therapy.

    With so many people wanting a chance to congratulate and interact with June and Sadelain—some even requesting autographs—it was hard to get more than a few minutes of their time to chat. June told me the award was a “huge honor” personally as well as a great way to recognize the efforts of those who have worked with him over the past three decades. “It’s a great way for the public to learn more about the value of what was initially basic research and how it can actually affect lives,” June said.

    Sadelain expressed similar sentiments. ”I’m so fortunate that somebody looked at our work and thought that it warranted such recognition,” he told me. Additionally, the presence of both patients and students “who are curious and have a sense that there is something that that would like to get involved with” at the award ceremony reinforced both the human impact and educational value of his work.

    That kind of curiosity coupled with persistence certainly served Sadelain and June well in their careers. An important theme that both honorees acknowledged is that scientific breakthroughs often require decades of dedicated work despite repeated setbacks.

    As June noted, prior to 2011, when the first checkpoint inhibitor therapy was approved, and then in 2017, when the first CAR T-cell therapy was approved “there were many decades when cancer immunotherapy was tried and failed.” Multiple disappointing results can make science review boards nervous and cause federal funding sources to dry up. For the CAR T field, things changed once regulators and scientists realized that immunotherapies could work but “that initial paradigm shift” was needed for acceptance, June said.

    Sadelain concurred. Scientists entering the field today are far less likely to face the same challenges that he and June and their teams had to contend with. “When we opened the first clinical trials, we couldn’t find patients,” he told me. “Today, there are waiting lists.”

    A brief history of CAR T

    The first CAR T trial was not in cancer but in HIV. In the 1990s, June’s lab explored the possibility of using T cells to treat the disease which at the time lacked treatments. “My first clinical protocol began when I was in the Navy in Bethesda and it was called RV 100,” he said in his award lecture. The work was done as part of a joint navy/army effort and marked “the first time we gave T cells to patients.”

    Carl June, MD, director of the Center for Cellular Immunotherapies and the Parker Institute for Cancer Immunotherapy at University of Pennsylvania, giving his lecture at the 13th annual Ross Prize Awards [Uduak Thomas]
    Carl June, MD, director of the Center for Cellular Immunotherapies and the Parker Institute for Cancer Immunotherapy at University of Pennsylvania, giving his lecture at the 13th annual Ross Prize Awards [Uduak Thomas]

    For that first protocol, June’s team took patients with late-stage AIDS and expanded their T cells in the lab, without making any genetic modifications. They then infused those cells back into the patients to determine whether they could restore their immune systems.

    “We thought it would be pouring fuel on fire” in the sense that “adding T cells back to the patients would cause more HIV replication.” But that did not happen because the scientists expanded the cells in a way that made them resist reinfection by downregulating the HIV co-receptor. The first clinical trial, published in 2002, showed a dose-dependent increase in T-cell counts following the infusions without a corresponding increase in viral load. June and his colleagues conducted three additional trials, which showed that the infused cells survived on average more than 10 years in patients.

    Following those trials, June and his team engineered T cells with a receptor composed of the CD4 molecule fused to the CD3 zeta signaling chain of the T-cell receptor. Over the next five years, June’s group ran trials using these first-generation CAR T cells, demonstrating that they were safe and persisted in the body. Those findings provided important safety data as CAR T technology began moving towards oncology applications.

    There were of course disappointments along the way. Several studies using the first-generation CARs did not demonstrate clinical benefit in cancer patients (in contrast to HIV patients). Enthusiasm for the technology waned and skepticism about its potential grew. Still June and his collaborators persisted. The second-generation CAR design incorporated a co-stimulatory domain rather than relying solely on CD3 zeta signaling. Specifically, they used 4-1BB in combination with CD3 zeta to target CD19-positive leukemia cells.

    And that’s when the tide turned. The first patient treated with this new generation of cells, a 67-year-old man with end-stage leukemia, achieved a complete response. In total, three of the first patients that were treated responded. “We didn’t know if we were really lucky at that time or not, but it was a striking result,” June said.

    Today, there are seven FDA-approved CAR T-cell therapies, and more than 60,000 patients have been treated. Several additional products are in development with many different designs being tested in thousands of labs. June closed by presenting some new, unpublished studies including a program in glioblastoma. It’s clear that June’s work is far from done.

    Sadelain’s milestones

    Sadelain said there were four major milestones that marked efforts to bring CAR T cells to the clinic. First was the development of methods for introducing genes into primary T cells. Before these techniques were available, scientists largely studied genes in leukemia cell lines as a surrogate for normal T cells. As such, critical aspects of T-cell biology remained poorly understood, including how they respond to antigen, when they proliferate, and when they undergo cell death.

    Michel Sadelain, MD, PhD. professor of medicine at Columbia University’s Vagelos College of Physicians and Surgeons and director of the Columbia Initiative in Cell Engineering and Therapy, giving his lecture at the 13th Ross Prize Awards [Uduak Thomas].
    Michel Sadelain, MD, PhD. professor of medicine at Columbia University’s Vagelos College of Physicians and Surgeons and director of the Columbia Initiative in Cell Engineering and Therapy, giving his lecture at the 13th Ross Prize Awards [Uduak Thomas].

    Sadelain’s second milestone was the identification of the gene encoding the CD3 zeta chain, which sparked efforts to engineer fusion receptors that enabled T cells to recognize and kill target cells in a sustained way. Third was identifying a target that could be studied in the lab. Sadelain’s lab settled on CD19, which was known to be expressed in lymphomas and leukemias.

    The fourth milestone was less a scientific discovery and more of a realization. If scientists wanted to bring T cells into clinical use, “you had to do it yourself,” Sadelain said. “There was no industry interested in developing or manufacturing cells as medicines.”

    While CAR T-cell therapies today are spreading beyond cancer to other disease areas, there are still challenges to solve in oncology. CAR T cells do not yet work well in solid tumors, Sadelain said. Despite some promising clinical results, “it’s clear that what had been designed for these blood cancers cannot be applied exactly as is to solid tumors,” Sadelain said. “They can be applied exactly as is to autoimmunity perhaps but not solid tumors.”

    Sadelain said the first challenge is the T cell itself. Once the engineered cells are released into the patient’s bloodstream, they have to reach the tumor and penetrate its defenses, which is not easy. Even if they are able to penetrate the tumor, they may not work because tumors have evolved mechanisms to shut off the immune response in order to survive. “The good news is that many of these mechanisms are understood today,” so the next step is to figure out how to engineer T cells that can overcome these mechanisms.

    Other challenges include identifying suitable targets and developing ways to support the CAR T cells to ensure they persist. Lastly, scientists need a way to manufacture these cells at sufficient scale to make treatments more affordable and accessible. It may be possible to lower the cost to patients through better reimbursement or policy changes “but some of that can be improved through biology.”

    Sadelain went on to describe three CAR designs that go beyond the foundation models and could be the treatments of the future. These designs aim to improve on some previous shortcomings, including a longer lifespan and requiring orders of magnitude lower doses than their predecessors. Sadelain closed by noting that it took nearly four decades for the field to get from “the very first ideas to where we are today,” but “it’s not static by any means.”

    “These molecules keep getting better and better, and that’s why we are optimistic,” he said. Moving forward, “we need persistence combined with potency. We need greater sensitivity. I think many of these beautiful receptors are on the way to deliver these results.”

    From discovery to deployment

    Following the awards, I spoke with Kevin Tracey, MD, president and CEO of the Feinstein Institutes. He told me that the Ross Prize celebrates the complete scientific journey from discovery to deployment. “We live in a time where we benefit from all the work that came before us by brilliant people who used science and medicine and technology to eradicate diseases that some people have never even heard of and will never see,” he said. “But somehow, some of that of the importance and the optimism of that gets lost in the modern era we’re living in.”

    Established in 2013, Tracey said the Ross Prize is unique because it celebrates that “rare individual who sets out to solve a problem, to cure a disease” and “stays with the entire process from discovery to development to deployment.” Sadelain and June “have lived that for decades,” Tracey said. Tens of thousands of people “are alive because of what these two men did and all of their colleagues.”

    But the Ross Prize is also important at a time of rising anti-science sentiments, amplified by some news media and social media platforms. “What we’re losing is the tradition of storytelling and the creation of stories and themes that bind us all together for a common good. Stories have to be told or they are lost,” he said. The Ross Prize is “an opportunity to tell those stories and to celebrate that excellence.”

    While the prize has always focused on rewarding excellence in science that forms the basis of new therapies, Tracey told me that over the past five years or so, the focus has expanded from basic research to include research that has made it into clinical use. There are many worthy science prizes that recognize “very elegant science,” Tracey said. But much of the downstream utility of that early science is “maybe decades in the making, and we decided to focus on the small number of times it actually does go the whole way. Those people deserve a prize too!”

    Decisions about the awardees each year are made by committee, with representatives from multiple institutions. It has always been a tough decision selecting a few winners from the hundreds of nominations, Tracey acknowledged, “but we always come to a consensus.”

    The next transformative therapy

    The transformation from risky experimental therapy to standard of care for some cancers demonstrates how scientific consensus can completely reverse. These days, the future is certainly bright for immunotherapies far beyond its original oncology focus.

    Both honorees expressed excitement about the possibilities while maintaining an awareness of practical limitations and reiterating the need for continued research. “I think basically all blood cancers will be treated with some kind of cell therapy,” June predicted. “That’s more of an engineering problem now.” Where advances are still needed, he said, is in similar therapies for solid cancers, something that both he and Sadelain are working on with their respective teams.

    Asked whether in vivo CAR T-cell therapy could be the next transformative therapy, June said: “It’s very early. Just two months ago, the first in vivo CAR T cells were reported, and they had a mixture of toxicity and efficacy in myeloma, so that’s great.” But “it’s too early now to know how long [they] will last and how safe” they will prove. He also noted the cost savings that in vivo CAR T therapies could offer, not just for cancer. “There are 10 times more people that have autoimmune disease than cancer. If we have a way to make it cheaper and more readily available, that’s what we really need.”

    Looking ahead, Sadelain said “there are many new potential directions that are really tantalizing.” There are, of course, many more cancers that need effective treatments, but researchers are starting to look to other areas as well including organ transplantation, neurodegenerative diseases, and infectious diseases.

    Among the important questions left to answer is how to produce these cells? “If this starts working for some more common diseases… we’re going to hit a bottleneck,” Sadelain noted. Could allogeneic cells from healthy volunteers be adapted to work for some recipients? Or could we use T-cells made from pluripotent stem cells? “That’s a very interesting direction.” Another “exciting new frontier” is emerging from in vivo studies, although there is still much to learn about their efficacy and toxicity, especially in cases where multiple doses are required.

    The Ross Prize committee is already thinking about next year’s honorees. In a few weeks, Tracey said a new batch of emails will be sent to solicit nominations for next year’s awards. The awardees for the 14th Ross Prize will be selected in January 2027.

    The post Perseverance, Persistence Key to CAR T Success, Say Ross Prize Winners Carl June and Michel Sadelain appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Personalized Cartilage Graft Developed for Life-Threatening Infant Airway Narrowing https://edusehat.com/en/personalized-cartilage-graft-developed-for-life-threatening-infant-airway-narrowing https://edusehat.com/en/personalized-cartilage-graft-developed-for-life-threatening-infant-airway-narrowing Thu, 18 Jun 2026 03:25:10 +0700 Edusehat Personalized, Cartilage, Graft, Developed, for, Life-Threatening, Infant, Airway, Narrowing A study led by researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a new method of using decellularized cartilage with patient-specific cells to help enlarge the pediatric airways narrowed as a result of severe subglottic stenosis (SGS). The condition is a narrowing of the airway below the vocal cords and above the trachea, and affects an estimated 20,000 infants per year.

    Researchers demonstrated in a preclinical model that this new method of airway reconstruction was faster, more effective, and able to overcome issues, such as donor site morbidity, insufficient tissue volume and delayed timeline, associated with the current standard grafts used for laryngotracheal reconstruction (LTR).

    Riccardo Gottardi, PhD, assistant professor with the Perelman School of Medicine at the University of Pennsylvania and leader of the Bioengineering and Biomaterials (Bio2) lab, and Ian Jacobs, MD, medical director of the Center for Pediatric Airway Disorders in the Division of Otolaryngology (ENT) at CHOP, co-led the research, which is reported in Nature Communications, in a paper titled “A translational approach to airway reconstruction leveraging decellularized meniscus and cartilage progenitor cells.” In their paper the team said, “This technology has the potential to revolutionize the field of pediatric LTR.”

    Severe subglottic stenosis (SGS) develops in children almost exclusively as a response to intubation, and affects nearly 1.5% of the over 200,000 infants in intensive care units each year in the United States, the authors explained. The most severe cases require laryngotracheal reconstruction (LTR), an open airway surgery that is used to enlarge the airway by implanting cartilage taken from a rib cage. While LTR is used to successfully treat thousands of children with subglottic stenosis, in many cases, young children often lack enough costal cartilage—the cartilage connecting our ribs to the sternum—for these grafts.

    As a result, operations often need to be delayed, leaving the child attached to a tracheostomy tube until they are older and grown enough to supply sufficiently sized cartilage for an effective LTR, and there is a higher risk of needing follow-up surgery because the airway is at risk of narrowing again. “In adults, LTR has a 90% success rate with low rates of revision,” the team stated. “However, in children, success rates significantly drop, and the incidence of restenosis requiring revision surgery increases to over 24%.”

    To improve this process and reduce the risk of these potential complications, Gottardi and Jacobs and colleagues have been looking at tissue engineering a laryngotracheal graft. Tissue engineering, the team wrote, could provide “an ideal alternative to autologous cartilage grafts to alleviate unnecessary comorbidities as well as reduce surgical time.” However, the complexity of the trachea prevents the use of conventional cartilage engineering techniques for this procedure.

    “We needed something that could be equivalent to a piece of cartilage, integrate well with the surrounding tissue, be well tolerated by the patient, behave like native tissues and regrow and be part of the airway,” Gottardi said. “This required quite a bit of creative thinking because of the additional challenges in children who are so small and still growing.”

    To overcome the limitations of existing methods, the researchers, led by former Gottardi lab member Paul Gehret, PhD, created a first-of-its-kind scaffold based on porcine meniscal cartilage decellularization (MEND – MENiscus Decellularization). They realized that if the cells, elastin, and blood vessels present in the meniscus are “digested” away, the meniscal cartilage becomes amenable to recellularization and integration while being less likely to provoke an immune response. In their paper the researchers explained, “Building on the strengths of previous decellularized therapies, we established an innovative approach that leverages the selective enzymatic removal of the elastin fibers and blood vessels uniquely present in the fibro-elastic cartilage of the meniscus to create microchannels, which support cellular invasion while substantially preserving native structure.”

    Using ear-derived cartilage progenitor cells (eCPCs), which can mature into cartilage-producing chondrocytes, the researchers demonstrated that MEND can be recellularized after the removal of elastin and blood vessels and suitable for implantation in less than a month. “Notably, porcine menisci, such as those used in this study, are a highly abundant cartilage source, being easily available as a waste product of the food industry, which can ensure no shortage of material for surgeons to shape into an ideal implant,” the team noted.

    Importantly, the new method needed to work in a clinically relevant timeframe. In a real-world scenario, clinicians may only have one or two months to be able to perform the procedure when it can still benefit the patient. Harvesting seed cells within days and creating a scaffold within three to four weeks is significantly less time than the six months that was typically needed for engineered cartilage. “Here we demonstrate that MEND can be fully recellularized in three days with ear-derived cartilage progenitor cells and reaches structural and functional maturation suitable for implant within three weeks of chondrogenic differentiation, a time frame compatible with clinical translation,” the authors stated.

    They validated their technology in a preclinical rabbit in vivo model, and demonstrated better performance than costal cartilage, the standard of care, with no instances of adverse events reported. “Our results demonstrate airway expansion, graft reepithelialization, neocartilage formation, and integration with adjacent native laryngotracheal cartilage at three months,” the team stated. “Notably, MEND implants perform better in all outcomes than autologous costal cartilage, the standard of care.”

    These findings will be further validated prior to proposing the procedure for patients suffering from severe subglottic stenosis. “These results demonstrate the feasibility of our translational tissue engineering approach to laryngotracheal reconstruction and could overcome the autograft-associated limitations in pediatric patients, decreasing the need for invasive revision surgery,” the investigators concluded.

    “This research shows really promising data that suggests this novel approach could overcome the autograft-associated limitations we sometimes encounter when attempting laryngotracheal reconstruction in infants,” Jacobs said. “With more research, we expect this could decrease the need for invasive surgery, and we may be able to apply the technology to other conditions that require a cartilage graft.”

    The post Personalized Cartilage Graft Developed for Life-Threatening Infant Airway Narrowing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO 2026: What to know about the AI Summit https://edusehat.com/en/bio-2026-what-to-know-about-the-ai-summit https://edusehat.com/en/bio-2026-what-to-know-about-the-ai-summit Wed, 17 Jun 2026 23:55:03 +0700 Edusehat BIO, 2026:, What, know, about, the, Summit “AI systems will likely have a central role over the next several years in almost every component of the biotech ecosystem,” says Joe Franklin, Chief Legal and Policy Officer at the Biotechnology Innovation Organization (BIO), “including what we consider the life cycle of biotech products—from early stage development to designing the product molecule, all the way through to R&D, clinical trials, non-clinical studies, and even regulatory process.”

    This is why BIO is kicking off the 2026 International Convention in San Diego with the second annual AI Summit, convening the industry’s leading minds to discuss opportunities and challenges around AI in the biotech ecosystem.

    The BIO International Convention is featuring 20 AI sessions covering a variety of use cases. The AI Summit itself will be held on Monday, June 22, with seven marquee sessions throughout the afternoon. 

    BIO 2026 convenes AI experts

    “We need to realize that with all of these AI tools, the underlying AI technologies are actually very different from one use case to the next, both when it comes to implementation considerations or policy considerations,” says Franklin. “There’s so much strategic development to make sure that we’re focusing our firepower on the right AI issues to advance the ecosystem.”

    In accordance with its mission, BIO is focused on understanding how it can create value for members across these areas while also advancing innovation and access for patients.

    “One of the important roles that BIO can play in AI issues is convening,” Franklin goes on to explain. “Given the speed of AI technology development, adoption is uneven. Uneven adoption means that there’s a really great opportunity for experts in the field, companies, technology developers, researchers, to learn from each other, and BIO can be the entity that brings together all of those voices in one place.”

    And indeed, the 2026 Convention’s AI Summit is bringing together some of the industry’s AI vanguard, including representatives from NVIDIA, Eli Lilly, the Mayo Clinic, Ginkgo Bioworks, Bristol Myers Squibb, and many more. 

    “The next wave of AI in life sciences isn’t a single model—it’s networks of specialized agents collaborating to solve the hardest problems in biology,” says Stacie Calad-Thomson, PhD, Business Development Lead of Pharma Labs and Manufacturing at NVIDIA. “From generative molecular design to lab automation execution and clinical trial optimization, agentic AI will fundamentally change the speed and economics of drug discovery. The companies that harness this now will compress what used to take years into months, and NVIDIA and its partners are working together to make that future real for patients.”

    Robust AI policy for a strong future

    BIO is also developing ongoing AI policy priorities to make sure that companies, lawmakers, and regulators can keep pace with this rapidly advancing technology. 

    “We’re looking to identify where all of the proposed or potential AI policies have implications for our members,” continues Franklin. “Where can the policy environment go to enable appropriate adoption of AI in biopharma? Where do policymakers need more education, including about the latest and greatest use cases of AI in biopharma? And where are there risks to AI adoption that we can be uniquely ready to help policymakers work through?”

    And when it comes to the creation of the policy priorities and education, the AI Summit is a major voice in informing their development. 

    How to participate

    If you are interested in getting more informed on AI’s role in the biotech industry, connecting with AI life science leaders, or joining the conversation around AI’s adoption along the biotech and life sciences ecosystem, you can attend any of BIO’s upcoming AI sessions.

    AI Summit panels on June 22 include:

    Additional AI sessions include:

    The post BIO 2026: What to know about the AI Summit appeared first on Bio.News.

    ]]>     Age-Related Inflammation Linked to R-Loop Nucleic Acids, Opens Therapies https://edusehat.com/en/age-related-inflammation-linked-to-r-loop-nucleic-acids-opens-therapies https://edusehat.com/en/age-related-inflammation-linked-to-r-loop-nucleic-acids-opens-therapies Wed, 17 Jun 2026 23:50:14 +0700 Edusehat Age-Related, Inflammation, Linked, R-Loop, Nucleic, Acids, Opens, Therapies In a new study published in Nature Aging titled, “Nuclear export of R-loop by the DDX1 and XPO1 complex promotes senescence-associated secretory phenotype and inflammaging,” researchers from the University of Texas (UT) MD Anderson Cancer Center have uncovered a previously unknown connection between R-loop nucleic acid structures and age-related inflammation or inflammaging. The results support new intervention options for chronic inflammation and subsequent health conditions.  

    In preclinical models, the administration of KPT-330 (selinexor) prevented export of R-loops and led to significant improvement in inflammation, liver damage, fat gain, muscle loss and overall lifespan.   

    “Chronic, widespread inflammation is a driving factor in many age-related diseases, including cancer, and our research has discovered one reason why this happens,” said Rugang Zhang, PhD, professor and chair of Experimental Therapeutics at UT MD Anderson and corresponding author on the study. “Understanding the cause is the first step toward developing treatments. We saw encouraging results using a drug that has already been tested in humans, paving the way for potential clinical use to alleviate age-related conditions.”

    Cells begin releasing signals that contribute to chronic inflammation once they enter senescence and stop dividing. Researchers have now pinpointed R-loops as a key component to modulating these inflammatory signals.

    An R-loop is a temporary cellular structure created during transcription, when a double strand of RNA and DNA becomes tangled with a third displaced single strand of DNA. While R-loops are traditionally confined to the cell nucleus, the study found that cells in senescence increasingly export R-loops into the cytoplasm. These R-loops attach to fragments of DNA debris to trigger chronic inflammation.

    This study identified the two proteins involved in exporting R-loops, DDX1 and XPO1. DDX1 attaches to the R-loop inside the nucleus to facilitate export. XPO1 allows the R-loops to be transported into the cytoplasm by forming a complex with DDX1.

    Researchers administered KPT-330, a FDA-approved drug for treating multiple myeloma that blocks nuclear export. The R-loops remain trapped inside the nucleus and could not trigger an inflammatory response.

    The study showed that shutting down nuclear export by blocking XPO1 in preclinical mouse models suppressed inflammaging, reduced liver fibrosis, lowered systemic inflammatory markers, and reversed age-related body composition changes.

    In a separate experiment, the same inflammatory alarm enabled the immune system to find and eliminate precancerous cells. The authors state that future studies could explore blocking DDX1 specifically, instead of shutting down all nuclear export, to mitigate side effects.

    The post Age-Related Inflammation Linked to R-Loop Nucleic Acids, Opens Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Solvent Recovery Gains Ground in Bioprocessing https://edusehat.com/en/solvent-recovery-gains-ground-in-bioprocessing https://edusehat.com/en/solvent-recovery-gains-ground-in-bioprocessing Wed, 17 Jun 2026 23:50:12 +0700 Edusehat Solvent, Recovery, Gains, Ground, Bioprocessing In addition to making products, a bioprocessor should be thinking more about solvents. “Solvent recovery is becoming a critical part of operational resilience and fiscal strategy for bioprocessing companies,” says Rudy Morin, engineering and modular solutions manager at Koch Modular. “It provides three primary benefits: operating expense reductions, supply-chain independence, and corporate sustainability.”

    The financial case is especially compelling for bioprocessors that rely on high-purity HPLC- or USP-grade solvents, which are costly to procure and expensive to dispose of properly. By recovering and reusing solvents from waste streams, companies can meaningfully cut virgin-solvent purchases while reducing hazardous waste disposal costs. Morin notes that the economics tend to work in a bioprocessor’s favor. “The payback periods are often attractive given quantities and solvent costs, and the recovery system quickly becomes a predictable long-term asset,” he says.

    Supply-chain reliability has become an equally pressing concern. Geopolitical tensions, severe weather, and transportation disruptions have laid bare the vulnerabilities in chemical sourcing. Solvent recovery addresses this by creating a closed-loop supply within the facility itself. “A solvent-recovery system gives bioprocessing facilities total control over their solvent supply and business continuity,” Morin says.

    Implementation, however, is rarely straightforward. Bioprocessing waste streams typically contain multiple solvents, water, and solids. Such mixtures often demand sophisticated separation technologies, such as pressure-swing distillation, extractive distillation, vacuum distillation, or liquid-liquid extraction. “Each additional unit operation adds intricate controls and operational variables, making the initial design phase absolutely critical,” Morin says.

    Capital costs present another barrier. Automated systems generally start in the low seven-figure range, with complexity driving costs higher. To manage risk, Morin recommends combining pilot testing, process simulation, and modular construction. Pilot testing clarifies feed characteristics and yields critical process data; simulation software optimizes designs before a dollar is spent on construction; and modular fabrication reduces project risk by moving work off-site, compressing schedules, and tightening quality control.

    Looking ahead, Morin sees meaningful opportunities in advanced extraction technologies and modular deployment. Liquid-liquid extraction, for instance, can recover products directly from fermentation broths while enabling downstream solvent recycling. For facilities considering the leap, his advice is practical: start with a clear design basis, conduct a rigorous economic analysis, and engage experienced process-engineering partners early. “Any solvent-recovery stream is worth a conversation with an experienced process engineering company, given the multiple potential benefits,” he says.

    As cost pressures and sustainability commitments converge, solvent recovery is increasingly positioned not as a compliance measure but as a strategic investment—one capable of delivering measurable value across operations, supply chains, and environmental performance.

    The post Solvent Recovery Gains Ground in Bioprocessing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Bacterial Expression Tech Prompts NorthX and enGenes Collaboration https://edusehat.com/en/bacterial-expression-tech-prompts-northx-and-engenes-collaboration https://edusehat.com/en/bacterial-expression-tech-prompts-northx-and-engenes-collaboration Wed, 17 Jun 2026 23:50:11 +0700 Edusehat Bacterial, Expression, Tech, Prompts, NorthX, and, enGenes, Collaboration Bacterial expression systems are becoming more versatile, say Swedish contractor NorthX Biologics and Austrian technology firm enGenes Biotech, who have teamed up to create an integrated, E.coli-based protein production platform.

    E.coli-based protein expression systems are not a new idea. They have been around since the 1970s and used to make everything from recombinant human insulin to treatments for growth hormone deficiency.

    They are generally cheaper than expression systems that use mammalian cells, primarily due to lower reagent costs. They also tend to be faster to set up, easier to scale, and have a reduced risk of viral contamination.

    Typically, E.coli-based systems are selected for the manufacture of simple proteins that do not require human-like post-translational modifications such as glycosylation. However, recent advances in strain engineering are starting to expand their scope.

    As a result, more biopharmaceutical firms are considering them for commercial-scale protein production, says Ola Tuvesson, CTO at NorthX, who cites this demand as a driver for the enGenes partnership.

    “The biggest growth vectors are biosimilar manufacturing, peptide therapeutics, and antibody fragments, including ADC components. Advances in glycosylation engineering are also expanding what the platform can address at the higher end of biologics complexity.

    “Several trends are reinforcing this direction: sustained pricing pressure pushing biopharma away from higher-cost production systems for this segment of the pipeline, growing investment in CRISPR-based strain engineering that extends microbial platforms into more complex program types, and a manufacturing infrastructure that needs to keep pace with industry demand,” Tuvesson tells GEN.

    Integrated pathway

    NorthX Biologics and enGenes’ strategy is to offer an integrated service that covers everything from strain design through manufacture.

    Tuvesson says, “The E. coli development to GMP manufacturing pathway typically covers expression system development, process development, scale-up, and transfer into GMP production. While the technical steps are well established, the challenge in many programs is that these stages are often handled by different providers, leading to delays, rework, and increased scale-up risk.

    “The pathway we are establishing addresses this fragmentation by integrating expression development and GMP manufacturing into a single, aligned workflow. This reduces handovers, improves data continuity, and helps ensure that the expression system is optimized from the start for manufacturing at scale,” he adds.

    The new pathway will combine high-throughput screening platforms, multi‑fermenter systems, and multivariate experimental design with advanced analytical support and scale-up engineering.

    According to NorthX Biologics and enGenes Biotech, the idea is to enable rapid iteration and the generation of decision-grade data early in development, supporting more robust and scalable processes.

    There is an option to extend the partnership, according to Tuvesson, who says, “The collaboration may also open up opportunities to implement more continuous manufacturing approaches over time.”

    The firms plan to monetize the platform by providing it to biopharma customers as a manufacturing service, rather than out-licensing, as Tuvesson explains.

    “Expression systems and processes developed for a specific protein can be transferred under standard commercial terms. However, the pathway itself is not a standalone licensable product.

    “The focus is instead on integrating expression development and manufacturing into a single workflow, reducing handovers and enabling faster development timelines,” he says.

    The post Bacterial Expression Tech Prompts NorthX and enGenes Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Continuous Production Platform Offers New Gene Therapy Options https://edusehat.com/en/continuous-production-platform-offers-new-gene-therapy-options https://edusehat.com/en/continuous-production-platform-offers-new-gene-therapy-options Wed, 17 Jun 2026 23:50:10 +0700 Edusehat Continuous, Production, Platform, Offers, New, Gene, Therapy, Options A new continuous bioprocessing platform for advanced therapy medicinal products (ATMPs) could help manufacturers increase yields and improve productivity, allowing them to treat larger groups of patients.

    The platform, developed by an independent U.K. innovation center, the Cell and Gene Therapy (CGT) Catapult, aims to provide an additional option for manufacturing gene therapies.

    “We’re not going to get rid of batch processing, but it is another tool for people to use when developing and manufacturing gene therapies and other ATMPs that could tackle some [existing] bottlenecks,” explains Bilal Ozdoganoglu, an associate senior scientist at the CGT Catapult.

    Ozdoganoglu spoke at the Bioprocessing Summit Europe in March about the downstream capture and polishing step of the continuous bioprocessing platform earlier this year.

    According to Ozdoganoglu, the platform aims to use continuous bioprocessing to overcome the problems of low yields and productivity, allowing ATMPs to cater to larger groups of patients while taking advantage of economies of scale.

    The platform uses perfusion technology in the upstream, followed by, in the downstream, a clarification step and multi-column chromatography.

    “Multi-column chromatography is almost bread and butter of more traditional biologics, but it’s relatively new in the gene therapy space,” he says.

    “As such, we had quite a few challenges to overcome to allow a system originally built for mAbs to cater for gene therapies.”

    For the polishing step, Ozdoganoglu explains that they heavily relied on digital modeling to generate parameters they could take into the laboratory.

    According to Ozdoganoglu, the recovery rates and overall performance of the continuous system were slightly better than, or comparable to, traditional batch processing.

    The next step, he explains, is to develop the platform further so it becomes an alternative to batch processing.

    As such, he says, the CGT Catapult is looking for collaborators, including therapy developers as well as vendors, automation suppliers, and contract development and manufacturing organizations (CDMOs).

    The post Continuous Production Platform Offers New Gene Therapy Options appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Analytics Map Purification Optimization Tradeoffs https://edusehat.com/en/analytics-map-purification-optimization-tradeoffs https://edusehat.com/en/analytics-map-purification-optimization-tradeoffs Wed, 17 Jun 2026 23:50:09 +0700 Edusehat Analytics, Map, Purification, Optimization, Tradeoffs Speed or quality? When it comes to two-step chromatography purification, biopharmaceutical manufacturers want both, despite knowing, realistically, that each choice involves tradeoffs.

    With purity, stability, toxicity, processing times, and costs hanging in the balance, the key optimization questions, therefore, are which purification efforts deliver the greatest return and how they can be combined to achieve the ultimate, optimal balance.

    A small multinational team of researchers is among the first to address that question with an analytical model “to jointly manage speed-quality tradeoffs and stage-specific lead-time constraints in purification operations,” Yasemin Limon, PhD, assistant professor, Bilkent University, tells GEN. This method guides optimization decisions, helping biomanufacturers decide how aggressively to intervene at each purification step of a serial, two-step chromatographic purification process based upon the costs of the intervention and the time constraints of the purification steps.

    The model, based on queueing network theory, captures what the authors call “practically relevant” tradeoffs, correlating intervention efforts, their effects on stability timeframes, and the probability of quality enhancement. It was developed by Limon and colleagues, Tugce Martagan, PhD, associate professor, Northeastern University, and Ananth Krishnamurthy, PhD, professor, Indian Institute of Management Bangalore.

    “Understanding how much and at which stations interventions should be applied allows biomanufacturers to optimize system performance without compromising on manufacturing lead times,” the team reports. Thus, the risk of long wait times between steps that may cause product deterioration is reduced.

    They divided purification optimization steps into two categories: Type I—those that improve batch quality without increasing purification processing time (such as selecting better resins or reagents)—and Type II—those that increase both batch purity and purification processing times (such as reducing flow rates).

    For each category, they evaluated how each optimization affected stage-specific lead-time constraints and how those constraints varied between the two categories of interventions.

    Choices are interrelated

    “Optimal intervention efforts change with costs,” they acknowledge. Here are the key takeaways:

    • Under-investing in upstream purification pushes purification downstream, where increasing the polishing time may risk product stability
    • For Type I interventions, put maximum effort into the least expensive options until product stability becomes a constraint
    • For Type II interventions, each decision affects both quality and processing times. Characterize process times at each chromatography step and document stability-based time windows to create a reference chart that can be used repeatedly
    • Shortening the stability window for step two necessitates more aggressive purification at step one. Fresh time constraints—related to new molecular stability data, for example—should not be evaluated in isolation
    • Create a reference map for the range of operating conditions typically encountered in your facilities, along with possible interventions, their costs, and stability-based time effects. Use this as a real-time reference on the manufacturing floor

    “The optimal policy depends on costs, processing times, and lead-time constraints,” Limon says. “Decisions at the first and second chromatography steps are interdependent.” Map those effects early to guide decisions in real time.

    She recommends turning the model into a decision map. “A manufacturer can estimate its own process parameters (batch arrival rates, processing times at each purification step, stability-based time limits, intervention costs, and the effect of each intervention on quality and processing time) and use the model to identify which intervention policy is optimal under those conditions.

    “Distinguish carefully between interventions that improve quality without increasing processing time and interventions that improve quality but slow the process,” Limon continues. “The first type affects lead time mainly through congestion at the downstream step, while the second type directly affects processing time and can make stage-specific lead-time constraints restrictive. Therefore, firms should quantify how interventions change processing time, congestion, and feasibility with respect to stability-based time windows.”

    The post Analytics Map Purification Optimization Tradeoffs appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Pancreatic Cancer Cell Death Triggered by Caspase‑8 Blockade in Preclinical Models https://edusehat.com/en/pancreatic-cancer-cell-death-triggered-by-caspase8-blockade-in-preclinical-models https://edusehat.com/en/pancreatic-cancer-cell-death-triggered-by-caspase8-blockade-in-preclinical-models Wed, 17 Jun 2026 09:30:10 +0700 Edusehat Pancreatic, Cancer, Cell, Death, Triggered, Caspase‑8, Blockade, Preclinical, Models Pancreatic cancer remains one of the most lethal malignancies, notorious for its late detection, rapid progression, and stubborn resistance to many therapeutic strategies clinicians have tried. Despite decades of effort, standard treatments have delivered only incremental gains, and the disease is projected to become the second leading cause of cancer‑related death within this decade. Now, researchers at the University of Cologne’s Center for Molecular Medicine Cologne (CMMC) have uncovered a surprising vulnerability in KRAS‑mutant pancreatic tumors—one that primes them for a potent form of programmed cell death.

    In a study published in Nature Communications titled “Oncogenic KRAS-driven type I interferon signaling primes pancreatic cancer for necroptosis,” the team reported that oncogenic KRAS, the defining driver mutation in roughly 90% of pancreatic ductal adenocarcinomas (PDAC), activates a type I interferon signaling program that inadvertently primes tumor cells to necroptosis, an inflammatory form of regulated cell death. However, “KRAS‑mutated tumors have a previously unknown Achilles heel,” said senior author Silvia von Karstedt, PhD. “By switching off the tumor cells’ defense mechanisms, we can significantly kill these tumors.”

    The defense mechanism in question is caspase‑8, a protein long known for its role in apoptosis but increasingly recognized as a gatekeeper that prevents necroptosis. The Cologne team found that KRAS‑driven interferon signaling induces high expression of necroptosis‑related interferon‑stimulated genes—including MLKL—creating a state in which tumor cells become heavily dependent on caspase‑8 for survival.

    Using genetically engineered mouse models, the researchers showed that deleting caspase‑8 specifically in KRAS‑driven pancreatic lesions triggered widespread necroptotic cell death and eliminated most precursor lesions. “Cancer cell-specific deletion of caspase‑8 is sufficient to trigger necroptotic cell death, eliminating most pancreatic precursor lesions,” the authors reported in their paper.

    Furthermore, in aggressive PDAC mouse models and human patient‑derived tumor organoids, pharmacologic caspase inhibition significantly reduced tumor burden.

    First author Sofya Tishina, PhD, emphasizes the translational potential: “The findings provide strong evidence that certain forms of pancreatic cancer could be specifically targeted for treatment based on their dependence on caspase‑8. In the long term, this could help develop new therapies for patients who currently have very limited treatment options.”

    Beyond pancreatic cancer, the study’s pan‑cancer transcriptomic analysis revealed that tumors with high Ras pathway activity and strong interferon signatures also exhibit elevated necroptosis gene expression, hinting at broader applicability. As the authors concluded in their paper, their work “reveals a KRAS-induced IFN program that sensitizes tumor cells to necroptosis, highlighting a therapeutic vulnerability in PDAC with broader relevance across IFN-activated cancers.”

    The post Pancreatic Cancer Cell Death Triggered by Caspase‑8 Blockade in Preclinical Models appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Merck, Protillion Launch AI Drug Discovery Collaboration with Up-to-$510M in Milestone Payments https://edusehat.com/en/merck-protillion-launch-ai-drug-discovery-collaboration-with-up-to-510m-in-milestone-payments https://edusehat.com/en/merck-protillion-launch-ai-drug-discovery-collaboration-with-up-to-510m-in-milestone-payments Wed, 17 Jun 2026 09:30:09 +0700 Edusehat Merck, Protillion, Launch, Drug, Discovery, Collaboration, with, Up-to-510M, Milestone, Payments Merck & Co. will partner with Protillion Biosciences to discover multiple new therapy candidates through a collaboration that could generate up to $510 million in milestone payments for the artificial intelligence-based drug design company whose “lab-in-the-loop” approach combines AI with a continuous feedback loop of experimental wet-lab data.

    The collaboration, launched through a multi-target discovery collaboration and license agreement, is designed to combine Merck’s global expertise in discovering novel therapeutics with Protillion’s Prot-MaP™ on-chip antibody discovery platform.

    Prot-MaP, short for Protein Display on a Massively Parallel Array, is designed to facilitate AI-based optimization of therapeutic antibodies through the quantitative analysis of protein libraries with unprecedented speed, scale, and precision, characterizing millions of variants per run and avoiding the common pitfalls of model overfitting.

    The result, according to Protillion, is the identification of optimized biologics with sophisticated therapeutic profiles such as pH-dependent sweeping and multi-target specificity—profiles that are difficult to achieve with traditional methods.

    Protillion says Prot-MaP is intended to enable the engineering of novel biologics by generating megascale, just-in-time quantitative antibody binding datasets for protein design AI. The platform enables the generation of tens of millions of clusters of immobilized proteins directly on an Illumina DNA sequencing flow cell through efficient tethered in situ transcription and translation.

    Prot-MaP was invented by the company’s CEO and co-founder, Curtis Layton, and co-founder Will Greenleaf, PhD, a professor of genetics at Stanford University School of Medicine and a member of Protillion’s Scientific Advisory Board. After receiving his PhD in computational biology from Duke University, Layton studied with Greenleaf as a postdoctoral fellow in the genetics department at Stanford Medicine.

    Curtis Layton, PhD, CEO and co-founder of Protillion Biosciences

    Layton developed Prot-MaP while working in Greenleaf’s lab, then organized Protillion in 2019 to commercialize the technology. Layton’s work pioneered a new approach to high-throughput interrogation of biochemical systems, tackling ultra-high-impact technology approaches for drug discovery by uniting fields that included protein engineering, next-generation sequencing technology, molecular biology, in vitro transcription and translation, computational biology, software development, and various engineering disciplines.

    Days rather than months

    “Prot-MaP is a technology platform that allows us to test millions of protein interactions simultaneously, generating an unprecedented amount of data in a matter of days rather than months. For example, we can rapidly evaluate large libraries of therapeutic protein candidates to see how they bind to different targets and how they behave under different biological conditions,” Robert Hollingsworth, PhD, Protillion’s CSO, told GEN.

    “We then combine that data with proprietary AI and machine learning tools to understand what drives the best-performing proteins and quickly design improved candidates. This gives us the ability to engineer antibodies with highly specific characteristics, such as stronger and more precise target binding, the ability to engage multiple targets, or the ability to activate only under certain physiological conditions,” Hollingsworth explained. “In practical terms, Prot-MaP helps us discover and optimize better drug candidates faster, with a level of insight and precision that has not previously been possible at this scale.”

    Prot-MaP allows Protillion to test up to one million protein variants simultaneously in a single experiment and generate results in as little as 48 hours.

    “Because we operate multiple proprietary platforms in parallel, we can rapidly scale that capability and generate enormous amounts of experimental data on demand,” Hollingsworth explained.

    What makes Prot-MaP unique, he continued, is not just its scale, but its combination of scale, speed, and AI.

    Robert Hollingsworth, PhD, Protillion Biosciences’ CSO

    “The platform is designed to seamlessly connect high-throughput protein testing with proprietary machine learning models, allowing us to quickly identify promising drug candidates, understand what makes them work, and design improved versions,” Hollingsworth said. “This enables us to tackle everything from discovering entirely new therapeutic molecules to optimizing existing candidates for multiple desired characteristics. In practical terms, Prot-MaP helps us find and develop better biologic medicines faster and more efficiently than traditional approaches.

    Opposite approach

    How does Prot-MaP overcome the complexity of protein molecules, which has long been a hurdle in protein and biologics design?

    “Many companies start with AI and then look for data. We took the opposite approach,” Hollingsworth said.

    Rather than relying primarily on computer predictions of protein structure, he elaborated, Protillion can apply Prot-MaP to directly generate large-scale functional data and identify the best therapeutic candidates based on real-world experimental results.

    “What makes this especially powerful is the sheer scale of the data we can generate. While much of the industry has focused on applying AI to relatively limited biological datasets, we believe that the biggest challenge in drug discovery is obtaining enough high-quality data to truly understand the complexity of protein function. Prot-MaP was built to solve that problem,” Hollingsworth said.

    By generating millions of protein measurements in parallel, Protillion says, it can create the kind of rich, large-scale datasets needed to train more powerful and predictive machine learning models. Those models, in turn, help design and optimize better therapeutic candidates faster and with greater precision.

    “Prot-MaP combines high-throughput experimentation with AI. The platform allows us to rapidly generate the data, and the AI helps us learn from it—creating a cycle that accelerates the discovery of next-generation biologic medicines,” Hollingsworth said.

    Speaking with GEN, Layton said Protillion and Merck have charted a course for the start of their drug discovery collaboration.

    “Our first two programs focus on inflammatory diseases, where we see significant unmet medical need and strong opportunities for differentiation,” he said.

    Immune-mediated inflammatory disorders are Merck’s specialty within its therapeutic area of focus in immunology. Merck focuses on several other therapeutic areas, which include oncology, vaccines, infectious diseases, cardiometabolic and respiratory diseases, neuroscience, and ophthalmology.

    “However, the Prot-MaP platform’s capabilities extend far beyond inflammation, enabling the discovery and development of novel biologics across a broad range of therapeutic areas,” Layton added. “As we continue to advance the platform, we expect to expand into additional disease areas where its unique capabilities can have the greatest impact.”

    Tech-focused pipeline collaborations

    Merck has launched several tech-focused pipeline collaborations in recent months aimed at replenishing its cancer and immunology pipelines, with the goal of developing new therapies that can recoup the billions of dollars in sales the pharma giant will lose as patent exclusivity expires in the United States and elsewhere for its aging blockbusters, including cancer immunotherapy Keytruda® (pembrolizumab) and Gardasil® 9 (Human Papillomavirus 9-valent Vaccine, Recombinant).

    In March, Merck inked an up-to-$2.2 billion collaboration with Quotient Therapeutics to apply Quotient’s somatic genomics platform to discover novel drug targets in inflammatory bowel disease (IBD). Also that month, Merck launched a partnership with Infinimmune to apply its Anthrobody® discovery platform and GLIMPSE™ antibody language model to identify and develop antibody candidates against multiple undisclosed Merck-designated targets. Merck agreed to pay Infinimmune an undisclosed upfront payment and up to $838 million in milestone payments.

    Merck has also launched several tech-focused partnerships, with partners that include:

    • Google Cloud—An up-to-$1 billion collaboration announced in April to deploy an agentic platform across Merck’s R&D, manufacturing, commercial, and corporate functions. Google Cloud engineers are working alongside Merck teams to deploy Google Cloud’s most sophisticated AI, including Gemini Enterprise.
    • Tempus AI—An expanded, multi-year collaboration of undisclosed value announced in March, aimed at accelerating discovery and development of precision medicine biomarkers, and supporting Merck’s oncology and potentially broader therapeutic portfolios.
    • Mayo Clinic—An R&D agreement of undisclosed value to apply AI, advanced analytics, and multimodal clinical data to support drug discovery and development. The agreement integrates Mayo Clinic’s Platform architecture, as well as clinical and genomic datasets, with Merck’s ambition of harnessing AI-enabled virtual cell technologies to enhance disease understanding, improve target identification, and drive early development decisions.

    In its latest collaboration, Merck has agreed to pay Protillion an undisclosed upfront payment, plus up to $510 million in payments tied to achieving research, development, and commercial milestones toward the successful development of an unspecified number of therapies.

    “Compelling opportunity”

    “Powerful emerging technologies offer the potential to transform the speed and precision with which we characterize protein landscapes and identify novel therapeutic candidates,” Juan Alvarez, PhD, vice president, discovery biologics at Merck Research Laboratories, said in a statement. “Protillion’s platform offers a compelling opportunity, and we look forward to working with the team to advance these programs.”

    Illumina’s venture capital arm, Illumina Ventures, is among investors in Protillion, having joined ARCH Venture Partners in 2022 to co-lead an $18 million financing in 2022.

    Based in Carlsbad, CA, Protillion has grown rapidly to a workforce of 30 people. In March, Protillion hired Robert Hollingsworth, PhD, a drug development executive with more than 30 years’ experience in biopharma, as CSO through a placement by executive search firm CollectiveMinds. Before joining Protillion, Hollingsworth was CSO at Shoreline Therapeutics, and earlier held positions in companies that included Pfizer (as vp and CSO of cancer vaccines and immunotherapeutics), Pharmacia & Upjohn (since absorbed into Pfizer), GlaxoSmithKline (GSK), and MedImmune (acquired by AstraZeneca).

    Protillion says it is continuing to expand its team and facilities, with the aim of supporting its internal pipeline and high-value strategic partnerships.

    “We plan to hire six more FTEs [full-time equivalents] by the end of the year,” Layton said.

    The post Merck, Protillion Launch AI Drug Discovery Collaboration with Up-to-$510M in Milestone Payments appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Single-Cell RNA Sequencing Reveals Gene Activity Changes in Crohn’s Disease https://edusehat.com/en/single-cell-rna-sequencing-reveals-gene-activity-changes-in-crohns-disease https://edusehat.com/en/single-cell-rna-sequencing-reveals-gene-activity-changes-in-crohns-disease Wed, 17 Jun 2026 05:55:11 +0700 Edusehat Single-Cell, RNA, Sequencing, Reveals, Gene, Activity, Changes, Crohn’s, Disease Researchers from the Wellcome Sanger Institute, Cambridge University Hospitals NHS Foundation Trust (CUH), and Open Targets have created a detailed cellular study of Crohn’s disease (CD), mapping how gene activity changes across more than 50 cell types in the gut. (Founded in 2014, Open Targets is a pre-competitive, public-private partnership that uses human genetics and genomics data to systematically identify and prioritize drug targets.)

    Co-led by Tim Raine, MD, PhD, consultant gastroenterologist at Cambridge University Hospitals NHS Foundation Trust, the team analyzed over a million gut cells from people with Crohn’s and from healthy controls, comparing changes in the gut lining and identifying immune cells that drive inflammation. The resulting single cell RNA-sequencing (sc-RNA-seq) resource, IBDverse, characterizes each cell type and those whose activity shifts in disease, uncovering new molecular and cellular signatures of immune activity in the gut lining.

    Co-first author Monika Krzak, PhD, formerly at the Wellcome Sanger Institute and now based at the Institute of Metabolic Science, University of Cambridge, said, “Crohn’s disease is complex, variable and deeply personal to every individual living with it, which is why understanding it at the level of single cells is so important. By creating this unprecedented map of more than one million gut cells, we are giving researchers around the world a powerful new tool to uncover how inflammation begins, persists and may one day be stopped. This is the kind of open science that can accelerate discoveries and bring us closer to better treatments for patients.”

    Raine added, “There is an urgent need for increased understanding of the biology of Crohn’s disease if we are to develop more effective and safe medications for people living with this condition. The patients who contributed to this research have helped us build insight into the different ways that gut function and immune function are disrupted in the disease, and with the insight comes immediate new avenues for drug development and targeted therapies.”

    Reported in Nature Genetics (“Single-cell RNA sequencing of terminal ileal biopsies identifies signatures of Crohn’s disease pathogenesis,”) the research revealing the cell types and molecular changes involved in Crohn’s inflammation is one of two complementary studies—the other a paper recently published in Nature—built on IBDverse to investigate different aspects of the disease.

    In their newly published report the team concluded, “These findings establish a comprehensive cellular and molecular framework for CD, offering insights into disease mechanisms and therapeutic opportunities.”

    Inflammatory bowel disease (IBD) is an umbrella term used to describe disorders that cause chronic inflammation of the gastrointestinal tract. Over half a million people in the U.K. are estimated to be living with IBD, which includes Crohn’s disease and ulcerative colitis.

    Crohn’s is a chronic condition that causes inflammation and ulcers in the digestive tract, from the mouth to the anus, often affecting the small intestine and colon. However, the authors noted, “Although inflammation is most commonly observed in the terminal ileum, CD exhibits substantial heterogeneity in disease location, severity and behavior, both between patients and within patients, over time.”

    Although inflammation is most commonly observed in the terminal ileum—the final section of the small intestine—Crohn’s is found in many locations of the body and with variation in severity both between patients and within patients over time.

    While therapies targeting immune cells have improved clinical outcomes for some patients, non-response to treatment remains high, with 15% of Crohn’s patients requiring surgery within five years of diagnosis. Consequently, there is an urgent need to better understand the etiology of CD in order to broaden therapeutic opportunities,” the investigators stated.

    For their newly reported study the researchers took and analyzed biopsies from 111 patients with Crohn’s and a history of current or previous terminal ileitis—inflammation of the ileum—and 232 healthy volunteers. The team performed single-cell RNA sequencing to measure gene expression in individual cells. “Single-cell RNA sequencing (scRNA-seq) technologies provide a high-throughput means to dissect complex tissues at the resolution of single cells and cell types,” they noted.

    By creating a comprehensive map of cellular and molecular differences in Crohn’s compared to healthy controls, the researchers established IBDverse as a result—an online data resource of over 1,185,000 cells isolated from small intestine samples. The large IBDverse dataset will serve as an open resource for future research.

    Using the data, the scientists identified genes that are abnormally expressed in Crohn’s and those where expression is specific to given cell types and cellular processes.

    One of the study’s most striking findings was a ‘molecular scar’ in the gut lining. Even after visible inflammation had healed, genes that help send messages to the immune system stayed switched on in the gut’s stem cells—the cells that constantly renew the lining. This suggests that an episode of inflammation leaves a lasting mark on these cells, which may shape how the gut responds to inflammation in future. In their paper the authors noted, “We uncovered epithelial changes marked by interferon-driven upregulation of major histocompatibility complex class I molecules that persisted in progenitor cells after macroscopic inflammation resolution.”

    The researchers also identified a population of macrophages—immune cells that engulf and digest cellular debris—with high expression of the gene ITGA4. These cells were key drivers of inflammation through the JAK/STAT pathway, which carries signals from the cell surface to the nucleus to switch genes on and off. “ITGA4+ macrophages were identified as key inflammatory drivers, showing enriched JAK–STAT signaling and cytokine expression (interleukin-6 (IL-6), IL-12 and IL-23),” the investigators stated. Drugs that block this pathway, known as JAK inhibitors, are already used to treat IBD, which points to these macrophages as a likely target of therapies.

    Co-first author Tobi Alegbe, PhD, at the Wellcome Sanger Institute and Open Targets, said “For inflammatory bowel diseases like Crohn’s and ulcerative colitis, it’s still unclear what is going wrong in the gut cells to cause inflammation. We have been able to compare gut cells of hundreds of people with and without IBD. This has given us new insight into the genes and cell types that are involved during active disease, and lays the groundwork for similar approaches to understand diseases of other major organs like eczema and asthma.”

    Co-senior author Carl Anderson, PhD, at the Wellcome Sanger Institute, said “What makes this study different is that we designed replication in from the start and found that even with hundreds of patients and standardized protocols, fewer than half of the gene expression changes we detected in one cohort replicated in the other. That’s a sobering finding for the field. The biology that did replicate consistently points to the gut lining itself as a key player in Crohn’s with a molecular signature in epithelial cells that persists even after inflammation has resolved. We don’t yet know what that persistence means, but it likely influences how the gut responds to future inflammatory insults.”

    The post Single-Cell RNA Sequencing Reveals Gene Activity Changes in Crohn’s Disease appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Six Takeaways from the Danaher Bioprocessing Summit https://edusehat.com/en/six-takeaways-from-the-danaher-bioprocessing-summit https://edusehat.com/en/six-takeaways-from-the-danaher-bioprocessing-summit Wed, 17 Jun 2026 05:55:10 +0700 Edusehat Six, Takeaways, from, the, Danaher, Bioprocessing, Summit The Danaher Bioprocessing Summit, “The Next Era of Bioprocessing: From Promise to Patient Impact,” took place in London earlier this month. The event brought together officials from Danaher companies (Cytiva, Pall, Beckman Coulter Life Sciences, IDBS, and Leica Microsystems), along with biopharma manufacturers and researchers to discuss how the industry can accelerate the transition from scientific breakthroughs to commercial therapies.

    Key themes included:

    • AI-driven bioprocess development and manufacturing
    • Intensified and continuous bioprocessing
    • Digitalization and connected data ecosystems
    • Improving productivity and reducing cost of goods
    • Cell and gene therapy manufacturing challenges
    • Scaling production of high-demand biologics, including GLP-1 therapies
    • Advanced analytics and process control
    • Sustainability in biomanufacturing

    The conference emphasized that future competitive advantage in biomanufacturing will come less from building additional capacity and more from increasing productivity, speed, and process intelligence across the development-to-manufacturing workflow.

    Based on the formal presentations, roundtable discussion groups, and conversations among speakers, panelists, and attendees, six key takeaway ideas emerged.

    The old manufacturing playbook no longer fits

    Biomanufacturing was built for large batches of standardized therapies. The next generation of medicines—cell and gene therapies, targeted and complex biologics, N-of-1 treatments—doesn’t fit that mold. As molecular diversity increases, the field is shifting toward smaller, parallel and distributed systems that can flex to meet the complexity of individualized medicine. This means faster decision-making, new investment models, and process designs that are data-driven and purpose-built from the start rather than adapted from previous playbooks.

    Manufacturing can no longer be treated as a downstream problem. It has to be part of the scientific conversation from day one.

    Automation and AI are making personalized scale possible

    For years, the promise of personalized medicine ran into a hard wall: you can’t manufacture one patient’s therapy the same way you manufacture a million doses of a traditional drug. Integrating automation, AI and high-throughput experimentation is changing that equation.

    These tools are enabling a shift from large-batch production to small-batch and even patient-specific manufacturing while improving efficiency, regulatory consistency, and access to advanced therapies. AI and digital tools are also compressing process development timelines, making it possible to design more tailored, adaptive manufacturing approaches without sacrificing rigor.

    Prediction is becoming a competitive advantage

    The organizations gaining ground are not merely reacting to problems faster but actually anticipating them. Digital twins powered by integrated data are helping teams model outcomes before committing resources, accelerating timelines and reducing risk. And real-time, molecular and submolecular-level data—shared across interoperable systems—are enabling more precise, proactive decision-making at every stage of development and manufacturing. Investing in this area with critical infrastructure is essential.

    Breakthroughs require collaboration across the whole ecosystem

    It turned out that the most consistent theme across both days of the Summit was this: no single organization can accomplish what’s needed alone. Partnerships across academia, industry, and regulators are accelerating how all kinds of therapies, especially gene therapies, move from discovery to approved treatment. Earlier alignment between developers, manufacturers, and regulators is reducing friction and compressing timelines.

    A central theme surfaced throughout the conference: the science is not the problem. The bioindustry already possesses powerful technologies and therapeutic capabilities. Future success depends on rapidly implementing them through manufacturing, regulatory alignment, supplier collaboration, and partnerships. Organizations that execute most effectively will shape the next decade of medicine. [Danaher]
    A central theme surfaced throughout the conference: the science is not the problem. The bioindustry already possesses powerful technologies and therapeutic capabilities. Future success depends on rapidly implementing them through manufacturing, regulatory alignment, supplier collaboration, and partnerships. Organizations that execute most effectively will shape the next decade of medicine. [Danaher]
    What makes these partnerships work is not goodwill alone but transparency, shared incentives, and data-driven collaboration that keeps everyone oriented around the same outcomes. The organizations making the most progress are those treating collaboration as a core capability.

     

    Regulatory models are evolving with the science, and sustainability is now a procurement requirement, not a values statement 

     

    Most of the frameworks that currently govern drug development were built for an earlier era of medicine. As therapies grow more complex and more personalized, those frameworks must change. Early engagement and risk-based approaches are helping bring complex therapies to patients faster without compromising scientific rigor. For rare diseases, which collectively affect an estimated 300 million people worldwide, tailored regulatory pathways are becoming essential.

    Summit speakers agreed that regulators are not the obstacle they’re sometimes assumed to be. They want to move faster too, and building the right collaborative infrastructure makes that possible.

    Environmental and social criteria are moving from corporate commitments into day-to-day supplier decisions. Organizations are integrating sustainability standards into procurement frameworks, requiring verified supplier commitments and shared performance targets as part of doing business.

    Far from being separate from operational strategy, this is part of building supply chains resilient enough to support long-term innovation at scale. Progress toward net-zero goals, Summit participants agreed, accelerates when sustainability is wired into commercial relationships rather than managed alongside them.

    Across two days and numerous conversations, one key point kept surfacing: the science is not the problem. The biopharma industry has the tools, the knowledge, and the therapeutic advances to transform how medicine is made and delivered. What it lacks is the collective will to operationalize them at speed. The next phase of progress will be won in manufacturing facilities, regulatory negotiations, supplier contracts, and partnerships that connect all of them.

    The final conclusion: organizations that close the gap first will define what the next decade of medicine looks like.

    To watch recorded keynote presentations, panel discussions, and fireside chats from the Danaher Summit on Bioprocessing, click here.

    The post Six Takeaways from the Danaher Bioprocessing Summit appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     As policy ecosystem threatens biotech, new treatments could drop by over 50% in 20 years https://edusehat.com/en/as-policy-ecosystem-threatens-biotech-new-treatments-could-drop-by-over-50-in-20-years https://edusehat.com/en/as-policy-ecosystem-threatens-biotech-new-treatments-could-drop-by-over-50-in-20-years Tue, 16 Jun 2026 22:50:03 +0700 Edusehat policy, ecosystem, threatens, biotech, new, treatments, could, drop, over, 50, years For decades, the United States has led the world in developing new medicines, turning scientific breakthroughs into treatments that save and improve lives. But a new analysis suggests that leadership is at risk.

    A new report from Magnolia Market Access finds that a combination of policy changes—a lack of research funding, regulatory staffing uncertainty, drug pricing proposals, and others—could significantly slow the pace of innovation, ultimately reducing the number of new treatments coming to market by up to 55% over the next two decades.

    What’s changing?

    The U.S. biopharmaceutical ecosystem depends on a delicate balance: early-stage research funding, a predictable regulatory process, and incentives that support high-risk investment. The report finds that all three parts of that system are facing enormous pressure.

    Research funding is becoming less stable

    Federal support, particularly through the National Institutes of Health (NIH), plays a critical role in early discovery. But recent funding disruptions and declines in grant opportunities are already slowing the flow of new ideas and scientific breakthroughs.

    Regulatory capacity is strained

    The U.S. Food and Drug Administration (FDA) is facing staffing losses and reduced institutional expertise, which will make the drug and treatment approval process less predictable and increase risk for developers.

    Pricing policies are reshaping investment decisions

    Changes introduced by the Inflation Reduction Act (IRA), along with international reference pricing proposals such as Most Favored Nation (MFN), are shortening the window for companies to recoup research investments, potentially discouraging work on complex or high-risk therapies.

    Why does this matter for innovation?

    Individually, these changes each have a significant impact. But their combined effect could be far greater than the sum of their parts.

    When research funding is uncertain, early discoveries that fuel new breakthroughs decline. When the regulatory process is less predictable, fewer therapies advance. When financial incentives weaken, investment shifts away from drug development. Together, these dynamics reinforce one another, creating a cycle that slows innovation throughout the entire pipeline.

    Magnolia’s modeling suggests that these pressures could lead to a significant reduction in new treatments over the next 20 years, potentially up to 55% fewer medicines than expected under current trends.

    What’s at stake for patients?

    At its core, this is not just a policy or economic issue; it is a patient issue.

    Fewer new medicines mean fewer treatment options for people facing serious or life-threatening conditions. The impact could be especially acute for patients with rare diseases, cancer, and other conditions where scientific progress is still emerging and options are limited.

    The effects may also take years to fully appear. Drug development often spans a decade or more, meaning today’s policy decisions will shape the treatments available to patients for a generation.

    At the same time, there are broader implications. The United States has long been a global leader in biomedical innovation, attracting talent, investment, and research. But as uncertainty grows, companies may shift research and development to other countries like China, potentially weakening the U.S.’s position as a biotech leader and slowing access to new therapies domestically.

    A call for a more coordinated approach

    The report emphasizes that no single policy is driving these risks. Rather, it is the interaction of multiple changes across the ecosystem.

    That means solutions will also need to be coordinated, ensuring stable research funding, predictable and efficient regulatory processes, and a policy environment that continues to incentivize innovation, particularly in areas of high unmet need.

    The stakes are high: a future in which the U.S leads the world in biotech innovation and brings new treatments to patients will depend on the choices we make today.

    The post As policy ecosystem threatens biotech, new treatments could drop by over 50% in 20 years appeared first on Bio.News.

    ]]>     BioTrinity 2026 Showcases Delivery Technologies and Promising Therapeutic Candidates https://edusehat.com/en/biotrinity-2026-showcases-delivery-technologies-and-promising-therapeutic-candidates https://edusehat.com/en/biotrinity-2026-showcases-delivery-technologies-and-promising-therapeutic-candidates Tue, 16 Jun 2026 22:45:09 +0700 Edusehat BioTrinity, 2026, Showcases, Delivery, Technologies, and, Promising, Therapeutic, Candidates European biotechs presented a range of interesting technologies and novel therapeutic biotechs at the recent BioTrinity conference, organized by BioUK, formerly OBN. The meeting highlighted a spectrum of cell and protein-based therapies, as well as novel delivery methods, with eye-catching investments and partnering opportunities.

    One of the stand-out technologies discussed by U.K.-based University of Southampton spin-out Renovos Biologics was the use of synthetic nanoclays. However, these are not being used for their traditional application of controlling drug release, but rather, for medical delivery. According to Agnieszka Janeczek, PhD, “We are developing an injectable, biodegradable nanoclay for use with bone morphogenetic protein 2 (BMP-2), a crucial growth factor in bone regeneration. We intend to use this on patients who have had spinal fusions after trauma or sports injuries.”

    Current treatments to encourage bone growth after spinal fusion, such as BMP-2 in its current formulations, are poorly retained around the spine, causing inflammation or, worse, bone growth outside the spine. This often leads to patients needing revision surgery at a cost of up to $50,000. “BMP-2 was used around fusions in the cervical spine, but the growth of bone and inflammation caused some patients to develop breathing difficulties and has resulted in a black box warning against using BMP-2 for this application,” noted Janeczek.

    To overcome these issues, the company has developed a synthetic nanoclay that provides a localized environment conducive to cell infiltration. The nanoclay retains the BMP-2 bioactive molecule until newly regenerated tissue gradually replaces it.

    “We have used RENOVITE® with BMP-2 so that this protein is injectable with a 23-gauge needle to deliver BMP-2 in a gel to allow precise templating of new bone formation, and therefore safer and more efficient bone fusion,” she continued. “Using our nanoclay, we see better quality bone growth as the bone grows evenly around and through the fusion, unlike BMP-2 on its own, which can sometimes initiate bone growth around the fusion to create an eggshell effect with bone only on the outside.”

    spinal surgery
    Renovos Biologics, which is developing an injectable, biodegradable nanoclay for use with bone morphogenetic protein 2, intends to use this crucial growth factor in patients who have had spinal fusions after trauma or sports injuries. [Vadym Terelyuk/Getty Images]
    The Renovos nanoclay retains the BMP-2 protein more readily around the fusion. As a result, it is possible to administer BMP-2 in a lower dosage, giving it the potential benefit of use in lower-cost markets, said Janeczek, and concluded that “Twenty-one percent of lumbar fusions are in patients that are younger than 45 years old, and the market for bone fusion could be worth $24.5 billion by 2035. Our lead asset RENOVITE BMP-2 will enter first-in-human trials by 2027, and we welcome investment to help assess this game-changing product in the clinic.”

     

    Patching up wounds

    Thomas Hafner, CEO of Onya Therapeutics, based in Ebbw Vale, U.K., stated that “For 25 years, wound care has been an innovation desert because therapies are difficult to scale and are not easy to adapt. This is not a glamorous area, but it is a massive opportunity.”

    To address this issue, several companies are developing devices, artificial skin, or anti-microbials. Others are developing sponges for managing exuding wounds, making them thicker for better absorption. As Hafner pointed out, they are doing this without asking, “Is this the right approach? “Wound care is a massive problem. For example, 30% of patients with diabetic foot ulcers will lose a limb within five years. Globally, there are triple the number of patients with chronic wounds as there are with cancer—wound care is a $400 billion crisis in plain sight.”

    Currently, with wound care, 90% of the costs are for the labor to manage the wound and only 6% for the products themselves. To address this, Onya says it is developing a treatment that could collapse the 90% costs of wound management by accelerating healing. The company calls it active exudate therapy.

    “In chronic wounds, the amount of exudate increases, and so instead of mopping up the exudate with dressings, we have found a way of turning off the tap. By doing this, we encourage healing and reduce amputations, potentially saving billions of dollars,” Hafner noted.

    Onya, which closed a £2.6 million seed financing round in 2025, is utilizing a compound known as OTX-PP01, based on potassium permanganate, a molecule with long-established clinical safety and broad antimicrobial and astringent action. This compound is delivered via a patch. “Unlike other wound management treatments, we don’t absorb the fluid; instead, our OTX-PP01 patch supports reduction of excessive exudate by addressing chronic inflammation, and creating the conditions for healing to restart,” said Hafner.

    foot wound
    Onya Therapeutics’ OTX-PP01 patch supports reduction of excessive exudate by addressing chronic inflammation, and creating the conditions for healing to restart. [Victor Golmer/Getty Images]
    The compound does this in two ways: first, it targets exudate with an astringent action that can reduce excessive wound fluid at the source, targeting the inflammatory cycle that can stall healing. Second, it addresses inflammation using a broad-spectrum antimicrobial action targeting pathogens through oxidation.

    “OTX-PP01 is not a new molecule. It has over 150 years of safe clinical use, but the delivery method is new,” explained Hafner. “The patch is easy to fit, and a nurse can put it on the wound and leave it on for 15 minutes for the compound to absorb and do its job of healing. After removing the patch, a nurse can then redress the wound.”

    Onya has designed its OTX-PP01 patch product to treat diabetic foot ulcers, venous leg ulcers, and pressure ulcers. It does not have to undertake Phase I studies as these were waived based on the established safety profile of the active compound. “We have a Phase II/III adaptive trial design, which we hope to complete in the next four to five years. Our aim with OTX-PP01 is to transform wound management to wound healing,” said Hafner.

    Eyes on the prize

    Therapies for treating ophthalmic diseases were also noteworthy at BioTrinity, with StemSight and Link Biologics presenting data on their promising clinical candidates. StemSight, based in Tampere, Finland, is a biotech company spun out from Tampere University. The firm is developing off-the-shelf induced pluripotent stem cell (iPSC) therapies to cure corneal blindness.

    According to Laura Koivusalo, PhD, CEO and founder of StemSight, the market value of regenerative medicine products across a variety of indications was $63 billion in 2026. However, the bottleneck for making these therapies widely available is manufacturing and scaleup. Koivusalo said that “Recently in Japan, the first iPSC treatments have been approved for use, and the advantage of using iPSCs combined with biomaterials is that they provide efficient and durable therapies.”

    Her company is developing a therapy to treat limbal stem cell deficiency (LSCD), a rare disease that causes blurry vision due to corneal epithelium loss and eventually blindness. There are over 240,000 sufferers worldwide, and there are currently no treatments for this condition available for most patients.

    “The only treatment option for LSCD is available only to patients with one healthy eye,” Koivusalo explained. “Scientists can harvest limbal stem cells from the healthy eye and transplant them into the diseased one to restore the damaged surface cells. The therapy proves that the cell transplantation approach works, but this is a personalized autologous treatment. Additionally, if both eyes have LSCD, and the patient is truly blind, this treatment is not possible as there are no healthy stem cells available to harvest. That’s where the high value of cure is really measured.”

    StemSight is developing an allogenic limbal stem cell therapy, which uses gene-edited iPSCs to reduce immunogenicity. Currently, the firm can produce one hundred patient doses per batch using a GMP-compliant process. It then freezes these cells until their delivery on a biomaterial carrier for patients.

    eye checkup
    StemSight and Link Biologics presented data in their talks on their promising clinical candidates for ophthalmic diseases. [Shironosov/Getty Images]
    “Our process produces high-quality stem cells for a low cost of goods,” continued Koivusalo. “We are currently in late preclinical development with our lead product, STE-101, and have shown that we can regenerate corneal epithelium in rodent models using imaging and histology. These are objective measures of efficacy, as we cannot ask rodents if they can read a chart.”

    StemSight is aiming to treat ten patients in a Phase I/II study starting early 2028.

    Unlike StemSight, Link Biologics, a U.K.-based spin-out from the University of Manchester, is developing first-in-class biologics to treat Dry Eye Disease (DED) and Wet Age-related Macular Degeneration (AMD). The firm is developing therapies based on TSG-6 (Tumor Necrosis Factor-α-Stimulated Gene/Protein-6), a secreted glycoprotein that has an endogenous role to protect tissues from inflammatory damage and promote repair. Link’s TSG-6-based therapies have enhanced activities compared to the native protein and thus have a unique combination of anti-inflammatory, tissue-protective, and tissue-reparative properties.

    Reuben Dawkins, CEO and co-founder of Link Biologics, stated that “We are using these protein biologics to treat DED and wet AMD because these are billion-dollar markets where there are limited treatment options available. Current standard of care for DED, for example, involves cyclosporine, which has a 15% response rate, or Xiidra, where 50% of pivotal trials did not show improvement in key efficacy measures vs no treatment. Overall, nine in ten DED patients stop using their initial medication within one year.”

    The company’s lead candidate, LB001, is in preclinical development for the treatment of DED. Dawkins presented data to show that in mouse models, twice daily administration of LB001 for seven days reduced corneal epithelial damage and suppressed inflammatory markers compared to a branded cyclosporine (Restasis).

    “Unlike other treatments, LB001 has a dual tissue-repair and anti-inflammatory action which may make this molecule more effective than current therapies,” noted Dawkins. “We have completed CMC so are able to manufacture LB001 to GMP standards, and it is stable as an eye drop formulation. We will be taking LB001 into a 180-patient Phase I/II clinical trial, which we aim to commence in 2027.”

    Sue Pearson, PhD, is a freelance writer based in the U.K.

     

     

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    ]]>     Scramblase Dynamics Uncovered by Single-Vesicle Fluorescence Microscopy https://edusehat.com/en/scramblase-dynamics-uncovered-by-single-vesicle-fluorescence-microscopy https://edusehat.com/en/scramblase-dynamics-uncovered-by-single-vesicle-fluorescence-microscopy Tue, 16 Jun 2026 22:45:08 +0700 Edusehat Scramblase, Dynamics, Uncovered, Single-Vesicle, Fluorescence, Microscopy A new single-protein analysis technique gives researchers newfound ability to study scramblases, physiologically important proteins that translocate phospholipids bidirectionally across cell membranes.  

    In the study published in Nature Structural & Molecular Biology titled, “A single-vesicle fluorescence microscopy platform to quantify phospholipid scrambling,” researchers from Weill Cornell Medicine and Ruhr University Bochum have developed a fluorescence imaging-based technique to measure the activity rates of individual scramblase proteins. 

    “I’m excited about this new platform as it is versatile and provides unprecedented information on exactly how fast a single scramblase works,” said Anant Menon, PhD, professor of biochemistry and biophysics at Weill Cornell Medicine and co-corresponding author of the study.

    Scramblases are key drug targets with roles in the assembly of cell membranes, modification of proteins with sugars, cell survival, muscle development and molecular trafficking. Yet, strategies for understanding scramblase dynamics have been limited.

    Traditionally, researchers purify scramblase proteins for further study using vesicles to record average scramblase activity. However, this bulk approach is unable to measure the transport rate of individual scramblases and capture how scramblase variability impact biological processes.

    The authors used fluorescently-tagged scramblases to achieve high resolution and evaluated a scramblase protein, known as VDAC1, best known as a membrane channel protein within mitochondria. Two copies of VDAC must align to provide a pathway for lipid movement. These dimers have a wide range of scrambling rates, from fewer than 100 to more than 1,000 lipids per second.

    “These findings indicate that only certain dimer conformations are capable of rapid scrambling, directly validating predictions from computer simulations,” Menon said.

    The team demonstrated the versatility of their approach by applying the platform to measure lipid-scrambling by opsin, a cell-membrane receptor and scramblase that is involved in light-detection in the eye. Results showed that individual opsin proteins scramble lipids faster than VDAC dimers, achieving rates in excess of 10,000 lipids per second.

    The new platform can study how drug molecules impact scramblase function. Additionally, the authors aim to combine their functional studies of scramblases with high-resolution imaging to understand how scramblase shape relates to activity rates. The team also plans to use the technique to study other lipid-moving proteins called flippases and floppases.

    The post Scramblase Dynamics Uncovered by Single-Vesicle Fluorescence Microscopy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Therapy-Resistant Residual Cancer Cell Dependencies Mapped https://edusehat.com/en/therapy-resistant-residual-cancer-cell-dependencies-mapped https://edusehat.com/en/therapy-resistant-residual-cancer-cell-dependencies-mapped Tue, 16 Jun 2026 12:00:10 +0700 Edusehat Therapy-Resistant, Residual, Cancer, Cell, Dependencies, Mapped Cancer drugs can shrink fast-growing tumors. But sometimes a few tumor cells survive. These “persister” cells seed new tumors, forcing cancer patients into arduous cycles of testing and treatment. The problem is that persister cells are rare—as few as one in a thousand tumor cells—and they’re genetically identical to the tumor, which makes them hard to find. Plus, their tenacity can be temporary, and by the time a scientist can get them in a petri dish, the qualities that helped them survive may have faded.

    To figure out how to beat them, researchers at the University of California, San Francisco (UCSF), built a robotic system that treats thousands of mini tumors at once in the laboratory. Their resulting ResMap platform lets scientists systematically identify, track, and treat surviving cells. The platform revealed shared features among persister cells that could help explain why cancer comes back—features that could be exploited by future drug therapies to beat them. “A few years ago, people were still asking whether persister cells were real,” said Xiaoxiao “Vany” Sun, PhD, an assistant researcher in the UCSF Department of Pharmaceutical Chemistry. “Now we can find them and test ideas for how to eliminate them.”

    Sun is first author of the team’s published paper in Science Advances, titled “ResMap: A community resource for systematic mapping of therapy-persistent residual cancer cell dependencies across contexts,” stating, “ResMap establishes a foundation for coordinated community efforts to accelerate rational persister-directed combination strategies toward the clinic.”

    Residual disease following targeted therapy remains a key challenge to achieving lasting responses in oncogene-driven cancers, the authors stated. Drug-tolerant persister cells, which the team describes as “subpopulations that survive initial therapy without stable genetic resistance,” can contribute to residual disease and seed tumor relapse. “Targeting drug-tolerant persister cells has emerged as an essential complement to oncogene-directed therapy, yet the field has lacked a unified framework to evaluate and prioritize candidate targets,” they wrote. “Understanding and targeting these cells have emerged as a promising strategy for achieving lasting therapeutic outcomes.”

    Cancer cell persistence was first described in 2010, the authors explained, and studies have linked persister survival to different biological processes and resulted in “an expanding list” of candidate therapeutic targets. However, they noted, “… despite over a decade of research, no persister-directed therapy has reached clinical approval.”

    For their reported study, the team gathered 94 drug candidates that other laboratories had flagged as potential persister therapies. They wanted to test each drug at different doses, on persisters from two types of lung cancer that had been treated with standard therapies. “As a testbed, we selected four lung cancer models: two with EGFR inhibitor osimertinib (EGFRi)–treated EGFRmut cell lines (PC9 and MGH134) and two with KRAS inhibitor sotorasib (KRASi)–treated KRASG12C cell lines (LU65 and MGH1138-1),” they wrote in summary. Each model was screened under normal oxygen and hypoxic conditions.

    It would require 10,000 painstaking, week-long experiments—so they built a robotic platform to eliminate the labor and inconsistency of doing it by hand.

    Thousands of miniature tumors sat in stacks of 384-well plates inside controlled incubators. A robotic arm, like those used in pharmaceutical drug screening, moved the plates between experimental stations. One station used sound waves to deposit tiny, precise doses of drug onto each tumor (first, a lung cancer therapy; then, an experimental persister therapy). Other stations stained the tumors with antibodies and took microscopic images of each tumor or group of persisters.

    The overall ResMap platform incorporated multiple components, the team explained. “… we developed the ResMap platform incorporating four integrated components: an automated high-throughput workflow, machine learning-based normalization, a persistence-specific metric, and a validated framework.”

    Their results showed that of the tested drugs, nine consistently weakened persister cells. The findings suggest that persister cells may share common vulnerabilities, even if they had emerged under different treatment conditions. “Initial screening identified 12 targets with conserved anti-persister activity across genotypes and oxygen environments; follow-up validation reproduced nine of these targets and revealed variable degrees of persister specificity relative to general cytotoxicity.” The investigators suggested that, “Collectively, these findings suggest that although persister biology involves multiple adaptive programs, targeting individual, well-chosen survival pathways may be sufficient to meaningfully reduce residual disease burden.”

    Steve Altschuler, PhD, professor of pharmaceutical chemistry at UCSF and co-senior author of the paper, said, “We expected each tumor to behave as its own special case. Instead, we found patterns that held up across many different samples, suggesting there may be underlying rules that can help predict which therapies are most likely to work.”

    The team plans to expand the platform to include more tumor types and treatment conditions. They hope the resulting dataset will be a resource to help researchers eliminate persister cells before they can give rise to drug-resistant disease. “ResMap provides a community resource for coordinated validation efforts and rational combination design aimed at minimizing residual disease following anticancer therapy,” they stated.

    The post Therapy-Resistant Residual Cancer Cell Dependencies Mapped appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Silica Nanoparticles Induce Ferroptosis, Reprogram Immunity in Prostate Cancer Models https://edusehat.com/en/silica-nanoparticles-induce-ferroptosis-reprogram-immunity-in-prostate-cancer-models https://edusehat.com/en/silica-nanoparticles-induce-ferroptosis-reprogram-immunity-in-prostate-cancer-models Tue, 16 Jun 2026 08:25:10 +0700 Edusehat Silica, Nanoparticles, Induce, Ferroptosis, Reprogram, Immunity, Prostate, Cancer, Models Ultrasmall fluorescent core‑shell silica nanoparticles—best known for their roles in medical imaging applications—are now showing surprising therapeutic muscle. Originally engineered as inert carriers for imaging agents, these particles, called Cornell Prime dots (C’ dots), have steadily expanded their résumé. In a new preclinical study, researchers at Weill Cornell Medicine report that these engineered silica nanoparticles can directly kill prostate tumor cells while reawakening antitumor immunity, offering a potential new edge in a disease where immunotherapy has historically struggled.

    Prostate cancer remains one of the most immunologically “cold” solid tumors, with myeloid‑driven immune suppression, metabolic bottlenecks, and stromal remodeling that blunt the effects of checkpoint blockade. The new work suggests that C’ dots—when targeted to prostate‑specific membrane antigen (PSMA)—can break through these layers of resistance by triggering ferroptosis, remodeling the tumor microenvironment, and priming tumors for combination immunotherapy.

    “We’re very encouraged by these results; a treatment that directly induces tumor‑cell death while transforming the immune microenvironment, as this does, would represent a new clinical paradigm,” said senior author Michelle Bradbury, MD, PhD, the endowed professor of imaging research in radiology and director of the Molecular Imaging Innovations Institute at Weill Cornell Medicine and a neuroradiologist at NewYork-Presbyterian/Weill Cornell Medical Center.

    The study, published in Cancer Research and titled “Reprogramming of TLR–Ferroptosis Signaling and Immunometabolic Pathways Overcomes Myeloid Suppression to Improve Checkpoint Blockade in Prostate Cancer,” shows that the silica particles accumulate in prostate tumors and push cancer cells toward ferroptosis, a form of iron‑dependent cell death driven by runaway lipid peroxidation. Although the particles were originally designed for imaging, the team found that they often pick up positively charged iron ions in the bloodstream and shuttle them into tumor cells—effectively turning the particles into catalytic seeds for oxidative collapse.

    At the same time, the nanoparticles reshape the immune landscape. T cells, macrophages, and other immune populations shift from inert or suppressive states into robust antitumor activity, converting cold tumors into hot ones. “One of the most intriguing aspects of this work is the convergence of direct tumor cell killing with broad immune remodeling,” said co‑author Jedd Wolchok, MD, PhD, the Meyer director of the Sandra and Edward Meyer Cancer Center, professor of medicine at Weill Cornell Medicine, director of the Parker Institute for Cancer Immunotherapy at Weill Cornell Medicine Meyer Cancer Center, and an oncologist at NewYork-Presbyterian/Weill Cornell Medical Center.

    prostate cancer
    Multiplex immunofluorescence image of a prostate tumor 10 days after treatment with prostate-targeted C’ dots and immunotherapy, showing extensive infiltration of immune cells throughout the tumor. Different colors represent distinct immune cell populations, including anti-tumor T cells, helper T cells, regulatory T cells, and macrophages. The image illustrates the coordinated immune response triggered within the tumor following treatment. [Bradbury Lab]
    The therapeutic impact was most striking in survival experiments. C’ dots alone modestly extended survival in aggressive mouse models, as did checkpoint blockade alone. But the combination produced complete or near‑complete remissions in 40% of mice. Adding CSF‑1R blockade increased complete remissions to 50%.

    The researchers’ next steps include continuing to explore these ultrasmall core-shell silica particles, setting the stage for the platform’s translational potential.

    “By creating conditions that support a more effective antitumor immune response, these particles may help unlock the full potential of immunotherapy in prostate cancer, where durable responses have historically been difficult to achieve,” added Wolchok.

    The post Silica Nanoparticles Induce Ferroptosis, Reprogram Immunity in Prostate Cancer Models appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     This man with ALS is “the first power user” of a brain implant that lets him speak https://edusehat.com/en/this-man-with-als-is-the-first-power-user-of-a-brain-implant-that-lets-him-speak https://edusehat.com/en/this-man-with-als-is-the-first-power-user-of-a-brain-implant-that-lets-him-speak Tue, 16 Jun 2026 04:55:02 +0700 Edusehat This, man, with, ALS, “the, first, power, user”, brain, implant, that, lets, him, speak
  • First long-term "power user" of a speech implant: Casey Harrell, paralyzed by ALS, has logged over 3,800 hours using a brain-computer interface at home — far beyond what any previous user has achieved — communicating with 99% accuracy across a 125,000-word vocabulary.
  • Growing independence changes everything: Early on, researchers had to physically connect Harrell to the device themselves. Now his care partner handles it, meaning he wakes up, gets plugged in, and simply gets on with his day.
  • More than communication: Harrell uses the implant to surf the web, send emails, and continue his career as an environmental activist — and a profanity filter lets him read bedtime stories to his seven-year-old daughter.
  • The holy grail, with caveats: Experts call long-term, independent BCI use a landmark achievement, but warn results may vary — brain degeneration, scar tissue, and many patients' reluctance to undergo invasive surgery remain real obstacles to wider adoption.
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    Casey Harrell has had a set of electrodes embedded in his brain for almost three years. Harrell, who has amyotrophic lateral sclerosis (ALS) and is paralyzed, first used his brain-computer interface (BCI) to “speak” sentences with the help of a research team in 2023.

    Since then, Harrell has clocked thousands of hours of use. He can use the device largely independently, once he’s been “plugged in” with the help of a carer. His team has added new features to it, and Harrell also uses it to surf the web and perform his job.

    “Living with a disease like ALS, you are supposed to have diminished dreams. I do not,” Harrell tells MIT Technology Review. “Any one of these things would be an absolute godsend of improvement. To have all of them, and many, many more, is truly revolutionary.” 

    Within the first 22.6 months after the device was implanted, Harrell had used it for more than 3,800 hours at home without any researchers present, the team reported today in the journal Nature Medicine. “He’s the first power user of a speech BCI,” says team member Sergey Stavisky, a neuroengineer at the University of California, Davis.

    Decoding speech

    Three years ago, Harrell entrusted David Brandman, an associate professor of neurological surgery at the University of California, Davis, and his colleagues with his brain. Harrell, who was 45 at the time, had already been diagnosed with ALS, a degenerative disease that robs people of the use of their muscles.

    Harrell was dependent on others to control his wheelchair and to dress and feed him. He had difficulty speaking; people struggled to understand what he was saying. Then Brandman and his colleagues asked if he’d like to trial a brain implant that might help him communicate. “The industry was [on the] cusp of a transformation, and I wanted to be part of it,” says Harrell. He signed up.

    In July 2023, during a five-hour operation, doctors implanted four arrays of 64 electrodes each into his brain. Each pair of arrays was wired to a “pedestal” connection point—creating two docking locations on the exterior of his skull to connect the electrodes to a computer.

    The team had long been working on developing algorithms to decode brain activity into speech. Their system works by recording activity from the speech motor cortex—a region of the brain responsible for the movements that allow us to speak.

    “There are 39 phonemes that make up all the sounds in the [American] English language,” says Nicholas Card, a neuroengineer at UC Davis and member of the team. Mapping neural activity related to producing each of those phonemes can allow the team to create a personalized speech decoder and software that can “speak” those words. “We first go from brain data to phonemes, and then from phonemes to words,” he says.

    They started using the device around a month after the surgery. The team got Harrell’s speech decoder working on the first day, says Card. On that day in August, Harrell used the device to speak with a 50-word vocabulary, and 99.6% of the words were as he’d intended. That vocabulary was later expanded to 125,000 words with 97.5% accuracy.

    At the time, it was unclear how long the device might last. Brain-computer interfaces are still new—not many people have had them implanted for long periods of time. Scar tissue can form around electrodes in a person’s brain, interfering with their ability to pick up neural activity, for example. But that doesn’t seem to be the case for Harrell.

    Power user

    In another advance, Harrell is now able to use the device more independently. In 2023, members of the research team would have to visit Harrell at his home and physically connect and disconnect him from the device on the days he wanted to use it. Not anymore. The team has since automated more of the system—today, Harrell’s care partner can don and doff it for him. “He’ll wake up, get plugged in, and just get going,” says Stavisky.

    This is important, says Mariska Vansteesel, a BCI researcher at Utrecht Medical Center who was not involved in the trial. “For these technologies to be relevant for patients, we really need to test them in settings in which they will eventually be used … to demonstrate that it has value, that it’s usable, and that it functions well without the constant involvement of a research team,” she says.

    Casey Harrell uses his BCI to speak in “private mode.”

    The team has also worked to improve the system itself. It is now 99% accurate, says Stavisky. Harrell can also control a cursor—a game changer that enables him to use his personal computer to send text messages and emails, surf the web, and keep up with his job as an environmental activist.

    Over the years, the team has updated the system to accommodate specific requests from Harrell. He is now able to switch on a “privacy mode”—when active, any decoded text will be automatically deleted. He can also opt to use a “profanity filter” while he’s talking to his young daughter.

    “We have been able to add on to the software side of the device … improving the accuracy and adding more bells and whistles to enable me to be more independent when using the device,” says Harrell. “We are making the road as we walk it, or roll it, so to speak.”

    Nothing short of revolutionary

    Vansteesel cautions that while the device is working well for Harrell, there’s no guarantee it will work as well, or as long, for other people with ALS. Over the last decade, she has worked with a woman with ALS who used a fully implanted device to communicate using “brain clicks”—cursor clicks made using brain activity. The woman used her BCI for seven years, but it stopped working toward the end of that period, apparently due to brain degeneration.

    At any rate, not everyone with ALS will be willing to undergo invasive brain surgery, says Jane Huggins, who is developing noninvasive BCIs at the University of Michigan and was not involved in the trial. “Long-term, independent use with efficient and accurate communication is kind of the holy grail of BCI,” she says. “But we have been finding a consistent aversion to hospital stays among people with progressive conditions like ALS.”

    Harrell, however, calls the device “nothing short of revolutionary.” “This has allowed me to keep working and earn money and insurance for my family. This is reconnecting me with friends and family who are too shy or too afraid to come over and not be able to understand me,” Harrell says. “With my seven-year-old daughter, I am able to create a bond that I wasn’t before able to forge. Now I can read to them and help them sharpen their own reading skills. By doing so, I am able to share the responsibility of parenting with my wife, who does so much caregiving for me and also our daughter.”

    Stavisky and his colleagues hope to improve the device further still. “We’re never satisfied,” he says. One aim is to eventually restore Harrell’s “full voice.” They are working on a “brain-to-voice” system that could directly decode brain activity to a speaking voice, complete with natural-sounding cadence, inflection and intonation—a voice that could sound happy, angry, or sarcastic, for example.

    “I was quietly confident that I could get some personal benefit from the system,” says Harrell. “Never in a million years would I think that I would achieve this much.” 

    ]]>     ZoBio Introduces DNA-Encoded Library Service for Exploratory Drug Discovery Programs https://edusehat.com/en/zobio-introduces-dna-encoded-library-service-for-exploratory-drug-discovery-programs https://edusehat.com/en/zobio-introduces-dna-encoded-library-service-for-exploratory-drug-discovery-programs Tue, 16 Jun 2026 04:50:12 +0700 Edusehat ZoBio, Introduces, DNA-Encoded, Library, Service, for, Exploratory, Drug, Discovery, Programs Officials at Leiden, Netherlands-based CRO ZoBio say the company has launched a DNA-Encoded Library (DEL) discovery service, which is designed to help biotech and pharmaceutical companies generate validated, progressible hits against novel and challenging drug targets.

    The new offering combines structure-grade protein production, quantitative biophysics, DEL screening, off-DNA hit validation and X-ray crystallography into a single workflow, according to a company spokesperson, who explains that it enables clients to move beyond hit identification toward high-confidence starting points for drug discovery programs.

    ZoBio maintains that unlike transactional DEL screening approaches that focus solely on hit generation, its platform is designed to deliver biologically relevant, structurally characterized hit matter with clear potential for progression. The service is particularly suited to exploratory and difficult-to-drug targets, including protein–protein interactions (PPIs) and targets with poorly defined binding pockets, where conventional screening approaches often fail, notes Gregg Siegal, CEO of ZoBio.

    “Drug discovery teams today are increasingly focused on highly validated but technically challenging targets, where traditional screening approaches can struggle to deliver meaningful starting points,” he continues. “Our approach combines DEL technology with the structural biology, biophysics, and assay expertise needed to generate hits that clients can confidently progress.”

    Siegal also points out that the DEL service is library-agnostic, enabling clients to access commercially available DEL collections or apply ZoBio’s workflow to proprietary client-owned libraries. The integrated platform reportedly includes:

    • Structure-grade protein production and characterization
    • Quantitative biophysical assay development using techniques such as SPR
    • Biophysically informed DEL selection design
    • Interactive DEL data analysis and hit prioritization
    • Off-DNA hit resynthesis and orthogonal validation
    • Structural characterization through X-ray crystallography
    • Mechanistic insight to support downstream optimization

    The workflow is designed to support collaborative decision-making throughout the discovery process, helping clients rapidly establish whether difficult or exploratory targets are viable for further development, according to Siegal, who says that members of the ZoBio team will be available for meetings during the BIO International Convention in San Diego to discuss the new service.

     

     

     

     

    The post ZoBio Introduces DNA-Encoded Library Service for Exploratory Drug Discovery Programs appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     mRNA Flu Vaccine Shows Stronger, Longer-Lasting Immune Response https://edusehat.com/en/mrna-flu-vaccine-shows-stronger-longer-lasting-immune-response https://edusehat.com/en/mrna-flu-vaccine-shows-stronger-longer-lasting-immune-response Tue, 16 Jun 2026 04:50:10 +0700 Edusehat mRNA, Flu, Vaccine, Shows, Stronger, Longer-Lasting, Immune, Response Flu shots reduce hospitalizations and deaths for the roughly one billion people worldwide that get the flu each year. But they are less effective when the vaccine strains don’t closely match the viruses circulating in the community. Today’s vaccines are made months in advance of the flu season due to a long manufacturing process. When projections are off, strain mismatch can reduce the efficacy of the flu vaccines from about 60% (in a good year) down to 19%. A broader immune response could translate to a more effective vaccine even when the virus is changing faster than vaccine makers can update their shots.

    Now, an investigational mRNA influenza vaccine, developed by Moderna, helps the immune system recognize a wider range of influenza viruses than today’s standard flu shot, offering stronger and potentially longer-lasting protection. The vaccine is currently under review by the U.S. Food and Drug Administration and, if approved, would be the first mRNA vaccine against influenza.

    The findings are published in Nature Immunology in the paper, “mRNA-based influenza vaccine expands the breadth of the B cell response in humans.”

    “We are seeing that the mRNA flu vaccine doesn’t just boost the immune system’s response to what it has already seen, it can help expand and diversify the antibody response, covering a broader range of flu strains,” said Ali Ellebedy, PhD, professor in the department of pathology and immunology at WashU Medicine. “If we can make flu immunity broader and more durable, that could mean fewer hospitalizations and deaths, which translates into a major impact on public health.”

    In a separate Phase III clinical trial, Moderna found that its mRNA-based flu vaccine reduced the risk of illness by 26.6% more than the standard flu vaccine in older adults. Seeking to understand possible causes of this improved protection, the new study examined how immune responses to the mRNA-based flu vaccine differ from those of the standard vaccine.

    The researchers followed 75 adults ages 20 to 50 over either the 2022-2023 flu season or the 2023-2024 flu season. About half received the investigational mRNA vaccine (mRNA-1010). The other half got Fluarix, an approved flu shot containing inactivated influenza viruses. Both vaccine platforms targeted the same strains recommended by the World Health Organization for the two flu seasons.

    Analyzing blood samples, the researchers found a stronger immune response in participants who received the mRNA vaccine compared with participants who received the standard flu shot. Specifically, those given the mRNA vaccine produced more flu-specific antibodies and more flu-specific memory B cells.

    “Influenza is constantly evolving to evade our immune system,” said Hanover Matz, PhD, a postdoctoral research associate working in Ellebedy’s laboratory. “But if we can develop vaccines that activate diverse B cells that target a broad portfolio of flu viruses, we have a better chance of avoiding strain mismatches and potentially even reducing the frequency with which the vaccine is needed.”

    To investigate the vaccine’s ability to diversify B cells, the researchers studied germinal centers—where B cells improve their ability to recognize the virus and generate slightly different versions of themselves—in a subset of participants. It had not been previously understood if mRNA-based influenza virus vaccines can induce a superior germinal center (GC) response.

    Among 13 people receiving the mRNA flu vaccine, five developed flu-specific germinal center responses in the lymph nodes that persisted for the 26 weeks of the study. In contrast, persistent immune responses were not seen in the 15 participants who received the traditional flu shot.

    In addition, from four weeks after vaccination until the six-month mark, antibodies from mRNA vaccine recipients recognized and bound to many diverse flu strains across many decades of viral evolution, especially those known to cause the most widespread illness. Antibodies from standard vaccine recipients bound to fewer divergent virus strains.

    These findings, the authors note, reveal a key role for persistent GC responses in broadening the repertoire of vaccine-induced antibodies. “We are seeing that the mRNA flu vaccine is driving strong, persistent germinal center responses,” said Ellebedy. “This can broaden the antibody response and better arm the immune system against an ever-changing virus.”

    The post mRNA Flu Vaccine Shows Stronger, Longer-Lasting Immune Response appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     When Process Design Fails: 5 Common Planning Gaps That Create Downstream Purification Bottlenecks https://edusehat.com/en/when-process-design-fails-5-common-planning-gaps-that-create-downstream-purification-bottlenecks https://edusehat.com/en/when-process-design-fails-5-common-planning-gaps-that-create-downstream-purification-bottlenecks Tue, 16 Jun 2026 04:50:09 +0700 Edusehat When, Process, Design, Fails:, Common, Planning, Gaps, That, Create, Downstream, Purification, Bottlenecks

    Panelists

    Image of Victoria Hepworth

    Victoria Hepworth

    Product Manager
    Greenfield Global

    Panelist

    Image of Victoria Hepworth

    Victoria Hepworth

    With over a decade of hands-on experience in the biopharmaceutical industry, Victoria specializes in the design and implementation of ready-to-use solutions and single-use consumables supporting downstream purification processes. She brings a strategic, highly collaborative approach to problem-solving, partnering closely with cross-functional teams to deliver scalable improvements that enhance operational efficiency and drive successful results.

    Victoria is passionate about building strong team environments, supporting diverse learning styles, and applying data-driven decision-making to optimize performance across the process lifecycle.
    Image of Cole Cordes

    Cole Cordes

    Head of Manufacturing,
    Supply Chain and MSAT
    Bionova Scientific

    Panelist

    Image of Cole Cordes

    Cole Cordes

    With more than 30 years of leadership experience in the biopharmaceutical and life sciences industries, Cole specializes in manufacturing operations, supply chain strategy, and MSAT across the product lifecycle. He brings a strategic, results-driven approach to operational excellence, partnering with cross-functional teams to optimize manufacturing performance, strengthen supply reliability, and support successful commercialization.

    Cole is passionate about building high-performing organizations, developing talent, and leveraging data-driven decision-making to drive continuous improvement, scalability, and business growth.

    Broadcast Date: 
    • Time: 

    Downstream bottlenecks often stem from early process design decisions that fail to fully account for scale, variability, and the manufacturing realities of therapeutic modalities such as monoclonal antibodies.

    As upstream titers rise and novel modalities introduce added complexity, these early oversights can force reactive workarounds that impact throughput, cost, and product quality. By taking a more deliberate and forward-looking approach, teams can reduce downstream risk and build processes that are better equipped for manufacturing scale.

    In this GEN webinar, our speakers will examine five common planning gaps that can contribute to bottlenecks, including single-source material dependency, raw material pack size selection, sensitive buffer designs, and single-use systems designed without realistic failure modes. Using real-world MSAT and tech transfer examples, they will illustrate how, when overlooked, these drivers can lead to deviations, safety risks, and longer cycle times—and how to proactively address them. The webinar explores practical strategies that can help evaluate materials, buffer systems, and consumables through a scale‑ready lens—helping teams build more robust purification processes and avoid these common bottlenecks.

    A live Q&A session will follow the presentation offering you a chance to pose questions to our expert panelists.

    Produced with support from:

    Greenfield Global logo

    The post When Process Design Fails: 5 Common Planning Gaps That Create Downstream Purification Bottlenecks appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AI Predicts Gene Regulation for Drug Discovery Using Condensate Morphology https://edusehat.com/en/ai-predicts-gene-regulation-for-drug-discovery-using-condensate-morphology https://edusehat.com/en/ai-predicts-gene-regulation-for-drug-discovery-using-condensate-morphology Tue, 16 Jun 2026 01:15:08 +0700 Edusehat Predicts, Gene, Regulation, for, Drug, Discovery, Using, Condensate, Morphology In a study published in Cell titled, “Deep learning of functional perturbations from condensate morphology,” researchers at Princeton University have applied AI to understand how drugs affect the dynamics of key structures within the cell. The work introduces a tool that can map morphology to functional outcomes and shed light on markers of health. 

    The authors examined the changes in shape of biomolecular condensates, tiny droplets in cells that drive transcription and other gene regulation processes linked to disease, including Alzheimer’s, ALS and cancer. The findings support a robust system for monitoring and evaluating cellular responses to drugs at a single-cell level. 

    “The central problem in biology is how do you get emergent structure from individual molecular interactions,” said Cliff Brangwynne, PhD, professor of chemical and biological engineering at Princeton and corresponding author of the study. “The key innovation here was to develop a way to learn from the images and classify the patterns that are emergent.” 

    The team used an advanced microscope to image nucleolar morphology changes in hundreds of human cells under a range of drug-controlled conditions. Machine learning tools sorted the images into four basic categories based on the shape of the nucleolus, uncovering “cap” and “necklace” shapes linked to cellular stress responses.

    The authors ran a panel of drugs to examine the effect on nucleolar formation and measured changes in the condensate’s development. Varying concentrations caused different degrees of change in both caps and necklaces.  

    Two known anti-cancer drugs caused caps, while a third drug, called topotecan, triggered a new nucleolus morphology that the researchers labeled “flower.” While topotecan inhibits TOP1, an key enzyme during DNA replication, loss of TOP1 induced the flower shape and uncovered the enzyme’s role in maintaining nucleolar organization by regulating RNA processing. 

    “No one’s seen this flower morphology before,” said Brangwynne. “The network flagged it as not fitting neatly into the other three categories.” 

    The team also tested their neural network on other condensates related to RNA processes, observing similar dose-and-response results for drugs specific to nuclear speckles, a hub for messenger RNA activity, and condensates from respiratory syncytial virus. 

    This finding underscores the value of analyzing morphological changes. “You could be missing other important features,” said Anita Donlic, PhD, postdoctoral researcher and first author of the study. “Things that could tell you there’s new biology.” 

    The post AI Predicts Gene Regulation for Drug Discovery Using Condensate Morphology appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Patient groups praise Virginia PDAB veto https://edusehat.com/en/patient-groups-praise-virginia-pdab-veto https://edusehat.com/en/patient-groups-praise-virginia-pdab-veto Mon, 15 Jun 2026 21:40:03 +0700 Edusehat Patient, groups, praise, Virginia, PDAB, veto Patient advocates are praising Virginia Gov. Abigail Spanberger’s May 19 veto of Prescription Drug Affordability Board (PDAB) legislation (HB 483 and SB 271), arguing that similar efforts in other states have failed to lower costs for patients while creating uncertainty around access to medicines.

    Among those welcoming the decision were Tigerlily Foundation, a patient advocacy organization working to educate, advocate for, empower, and support young women, before, during, and after cancer.

    “We’re deeply grateful to Gov. Spanberger for putting patients first and protecting access,” said Maimah Karmo, Founder and CEO of the Tigerlily Foundation and 20-year breast cancer survivor. Our community has been through enough. We cannot afford to experiment with policies that risk taking away the very medicines we depend on to stay alive.”

    The Biotechnology Innovation Organization (BIO) also applauded Gov. Spanberger for her decision.

    “BIO applauds Gov. Spanberger’s decision to veto SB 271 and HB 483—legislation that would have established a Prescription Drug Affordability Board (PDAB) and imposed arbitrary price controls in Virginia. The Governor’s action recognized an important point: even well-intentioned healthcare policies must be carefully designed to avoid unintended consequences for patients, providers, employers, and Virginia’s innovation economy,” said Patrick J. Plues, Senior Vice President of State Government Affairs & Affiliate Relations at BIO.

    Three strikes, you’re out: Virginia should stop trying to push PDABs

    This is the third time that the Virginia Legislature has attempted to push through PDAB legislation in the last five years.

    “They previously passed legislation twice,” explained Brian Warren, Vice President of State Government Affairs at BIO. “However, it was vetoed by former Gov. Youngkin. The authors of the bill tried again with a new governor, though they ultimately were unsuccessful in convincing her of the approach in the bill.”

    The repeated attempts to push through this legislation, as advocates have expressed, is not only frustrating, but a notable waste of time.

    “A lot of people talk about this being an experimental policy, but at this point, we’ve seen it fail in other states and patients have paid the price,” said Karmo. “We do not want it here. It doesn’t help patients. And frankly, we are tired of watching legislators recycle policies that hurt us instead of listening to the people actually living with cancer every day. We could be using our time to develop legislation and policies that actually work and are informed by patients, not just politics.”

    Warren has worked closely with patient advocacy organizations in states that have implemented PDABs—like Colorado and Oregon—and have seen how problematic they can be.

    “I know that for patients who are paying attention, it’s been devastatingly stressful for them and their families,” said Warren. “I’ve been on the phone with rare disease patients in Colorado, in particular, who are panicking and asking, Do I have to move out of the state because I can’t get access to my medication if this were to be enacted?”

    The problem with PDABs, as well as similar drug price control policies such as Most Favored Nation (MFN) and the Inflation Reduction Act’s (IRA) “price negotiations” is that they do not address the issue of patient out-of-pocket cost and fail to take into account problematic middlemen, such as pharmacy benefit managers (PBMs) or 340B entities.

    “A lot of what has been going on legislatively feels like a bait and switch with the UPLs to MFN, and neither are going to save patients a single dollar,” said Karmo. “Our patients and community are especially worried about drug switching, because a lot of them are already having to do that with the cuts to Medicaid and other things going on right now. Their out-of-pocket costs have risen astronomically. We are living this. We are watching people have to choose between rent and their prescriptions. Making them also then potentially have to choose between switching to a drug that might not work for them when they’re already stable on a drug is not about affordability. That is failure.”

    These policies also threaten patient access to medicines, because prescription drugs may be harder to source.

    As Bio.News reported in 2024, ”The sale of drugs is not limited to in-state purchases. Many are working with out-of-state companies and organizations in a vast and complex medicine distribution network. The assumption that providers within a state only buy drugs in-state is a fundamentally incorrect assumption inherent in the PDAB system.”

    It has been these persistent problems with PDABs that have proven them to be harmful in states they have been implemented in, with one state, New Hampshire, actually repealing its board.

    “We are reaching a tipping point where a lot of the states that are considering PDABs are looking at other states that have tried to implement them and seeing that the process is inherently complex and unlikely to provide savings to patients where they need it most,” noted Warren. “Rather, they have spent a lot of money and time over the past several years on a program that is inherently unsuccessful while they could have done something more productive.”

    Focus on policy that works

    The other issue with how the legislation moved this year was that its review and consideration was notably less rigorous than in years past.

    “Perhaps it was because they thought they had already passed the legislation twice, so it felt like old news, but during the committee processes, it really just kind of sailed through without a lot of consideration,” explained Warren.

    In fact, it was not until Gov. Spanberger started voicing concerns with aspects of law, even adding amendments and notes, that there was any evidence that ins and outs of the legislation were really being investigated.

    “We were glad to see that the Governor voice concerns and add amendments, but it was disappointing that even within that process, the patient voice wasn’t being incorporated,” said Karmo. “We are the ones who wake up every morning and take these medications. We are the ones who skip doses because we can’t afford them. We should be listened to. Ultimately, we’re all grateful for the veto, but we still want to be and deserve to be a part of the process moving forward.”

    Gov. Spanberger noted in her veto that PBMs play a significant role in this in terms of what patients pay out of pocket, and only addressing the list price of a drug is not going to bring down the cost of medicines for patients.

    The governor’s insight is a good start, and advocates hope that it will lay the groundwork for better and more engaged policy development.

    “We’re glad that Gov. Spanberger is identifying PBM reform as an important policy,” said Karmo. “But let’s be clear: we already know these boards don’t work. We’ve seen them fail. We’ve watched patients panic. We have the evidence. So let’s stop rehashing the same unsuccessful thing over and over again and start doing something that actually helps patients.”

    “We need to take a step back and take a more holistic view of the healthcare system, because no one party is responsible for the high cost,” concluded Warren. “We need to listen to patients more than anybody. We are grateful this governor is taking a leading role in that.”

    The post Patient groups praise Virginia PDAB veto appeared first on Bio.News.

    ]]>     StockWatch: Parabilis Medicines Makes Wall Street History with $770.5M IPO https://edusehat.com/en/stockwatch-parabilis-medicines-makes-wall-street-history-with-7705m-ipo https://edusehat.com/en/stockwatch-parabilis-medicines-makes-wall-street-history-with-7705m-ipo Mon, 15 Jun 2026 07:05:09 +0700 Edusehat StockWatch:, Parabilis, Medicines, Makes, Wall, Street, History, with, 770.5M, IPO In what’s shaping up to be a historic year for biotech initial public offerings, Parabilis Medicines (Nasdaq: PBLS) made its mark on Wall Street this past week by pricing the largest-ever IPO by a drug developer. This upsized offering raised an eye-popping $770.5 million in gross proceeds.

    No sooner did Parabilis begin trading public shares on Wednesday, a day after offering 33.5 million shares of its common stock at $20 per share, than its stock price leaped 58% from the IPO price, closing its first full trading day at $31.60. Those shares slid 4% on profit taking Thursday, closing at $30.31, then fell another 10% Friday to finish the week at $27.26.

    A day earlier, Parabilis announced the closing of its IPO, including a full exercise by underwriters of their 30-day option to buy an additional 5.025 million shares at the IPO price, less underwriting discounts and commissions. That added another $100.5 million to the initial $670 million in gross proceeds, which translates to another $93.5 million in net proceeds. When added to the $618.2 million net that Parabilis garnered from the original IPO, the company’s haul from the offering rises to a no-less-eye-popping $711.7 million, according to Parabilis’ IPO final prospectus.

    Parabilis priced its IPO above its initial price range of $17–19 a share. The company initially planned to offer 25 million shares, then raised that offer to 33.3 million before adding 200,000 more shares to finalize its offering.

    Parabilis’ IPO surpasses the $625 million IPO carried out in April by Kailera Therapeutics (Nasdaq: KLRA), which topped the previous record-high among U.S. biotechs, the $604 million offering of Moderna (Nasdaq: MRNA) in December 2018, two years before the messenger RNA (mRNA) vaccine developer won FDA emergency authorization for its COVID-19 vaccine.

    Based in Cambridge, MA, Parabilis is a developer of drugs and antibody-drug conjugates (ADCs) targeting historically undruggable protein targets and based on stabilized helical peptides or Helicons™.  Parabilis says it has generated proprietary datasets, comprising millions of data points for hundreds of thousands of Helicons across dozens of drug-like properties, following a decade of Helicon drug discovery.

    “Unlike Kailera, which formed in 2024 and quickly stockpiled private capital before going public, Parabilis took a longer and far less linear road,” commented Ben Zercher, senior biotech & pharma analyst with PitchBook.

    Parabilis was founded in 2015 as FogPharma to commercialize technology developed in and in-licensed from the lab of Harvard University researcher and serial entrepreneur Gregory Verdine, PhD, who served as the company’s co-founder and CEO from 2015–2023.

    Six venture rounds

    While the company closed six venture rounds, Zercher noted, its valuation had fluctuated in the face of the post-pandemic period that saw private biotechs struggle, as well as leadership turnover and the rebrand through which FogPharma became Parabilis in 2024.

    The rebranded company built momentum last year, according to Zercher, on the strength of its lead candidate zolucatetide (formerly FOG-001), a stabilized peptide built using the company’s Helicon platform. Zolucatetide is the first and only direct inhibitor of the elusive β-catenin:TCF interaction, according to the company.

    Parabilis stated in its final prospectus that it plans to spend approximately $150 million in IPO proceeds toward continuing ongoing clinical development of zolucatetide in desmoid tumors, including continuation of dose expansion and the launch of a Phase III registrational trial to topline data.

    Approximately $120 million is set to be spent on continuing the ongoing clinical development of zolucatetide across several additional indications, including dose escalation and expansion in familial adenomatous polyposis (FAP); hepatocellular carcinoma, the most common type of primary liver cancer; and other rare tumors, with the aim of collecting data to support a registrational trial.

    Parabilis plans to use the largest share of its IPO proceeds, approximately $190 million, toward advancing its pipeline of additional programs—including its ETS-related gene (ERG) protein degrader, an allosteric androgen receptor in its active state (ARON), and beta-catenin degraders—to Phase I clinical data.

    “Our current pipeline is focused on various cancers and tumor types; however, we believe Helicons could also have broad applicability against targets in many diseases with substantial unmet need outside oncology, and we plan to evaluate other therapeutic areas in the future,” Parabilis stated in the final prospectus.

    The remainder of the proceeds would be used, Parabilis said, toward continued evolution of the Helicon platform for discovering and developing drug candidates, as well as toward general corporate purposes that include additional development efforts, working capital, and operating expenses.

    Zolucatetide received the FDA’s Fast Track designation last year, followed in March by the agency’s Orphan Drug designation. In January, Parabilis closed on a $305.2 million Series F crossover financing round by selling 49,518,175 shares at $6.1644 per share to various investors, garnering $304.5 million in net proceeds. Parabilis finished the first quarter with $329.039 million in cash and cash equivalents as of March 31.

    Fifteenth biopharma IPO so far in 2026

    Parabilis is the 15th and latest biotech or pharmaceutical company to carry out an IPO this year, raising a combined $12.11 billion in proceeds, according to PitchBook data. Biotech and pharma accounted for 15 IPOs in all of 2025, raising a combined $10.49 billion—an improvement in dollars over 2024’s $8.83 billion, which was raised in 33 IPOs.

    Seven of this year’s IPO companies have seen their shares rise since their initial offerings, led by the 466% share price increase of Veradermics (NYSE: MANE), a developer of treatments for dermatology and aesthetic conditions that closed Friday at $96.24 a share.

    “With the biotech window reopened, the volume of IPOs reflects a backlog of quality companies that kept building through the biotech funding downturn rather than a wave of hype,” Zercher added. “Where the pandemic-era class sold preclinical optionality, Parabilis and the 2026 cohort are being priced on de-risked clinical programs with clear regulatory paths.”

    In addition to its IPO, Parabilis said, it has also closed on selling 4,166,666 shares at $18 per share—90% of the IPO price per share—through a concurrent private placement to Regeneron Pharmaceuticals that has raised approximately $75 million in proceeds.

    Parabilis’ initial IPO filing, dated May 19, came just a day after Parabilis inked an up-to-$2.3 billion-plus strategic research collaboration with Regeneron to discover and develop an initial five candidates encompassing “antibody-Helicon conjugates,” a new form of ADCs aimed at challenging and historically undruggable targets by combining the cell permeability of small molecules with the binding capabilities of larger biologics, in order to reach targets long considered undruggable.

    Financial runway into H2 2029

    Regeneron agreed to pay Parabilis $50 million upfront toward launching the collaboration. That upfront payment, plus proceeds from the IPO and the company’s existing cash and cash equivalents, “will be sufficient to fund our operations into the second half of 2029,” Parabilis stated in its IPO final prospectus.

    According to that final prospectus, Parabilis ended the first quarter with a $45.316 million net loss, up 18% from its $38.326 million net loss of Q1 2025, as well as a net loss of $145.889 million for last year, up nearly 24% from its $117.914 million net loss for 2024. The company has no reported revenue.

    Parabilis’ accumulated deficit rose 8% during Q1, to $586.82 million from $541.504 million at the end of 2025.

    To fund its operations, Parabilis reported, it has raised a total of $876.8 million as of March 31. That total consisted of $811.8 million from sales of its convertible preferred stock, $15 million in borrowings under a term loan, and a $50 million Simple Agreement for Future Equity (SAFE).

    Leerink Partners, BofA Securities, Evercore ISI, and Guggenheim Securities are acting as active book-running managers for Parabilis’ IPO, while LifeSci Capital is acting as a passive book-running manager.

    Leaders and laggards

    • Propanc Biopharma (Nasdaq: PPCB) shares soared 80% from $1.35 to $2.43 Thursday after the Australian developer of therapies for pancreatic, ovarian, and colorectal cancers said it approved a share repurchase program authorizing the company to repurchase up to $5 million of its common stock. “The management team believes we are entering a transformative stage for the company,” CEO James Nathanielsz stated, citing recent progress by Propanc’s lead asset PRP, a first-in-class therapy designed to treat and prevent metastatic cancer from solid tumors, toward entering the clinic with a pivotal Phase Ib, first-in-human study in 30–40 advanced cancer patients. He also cited the company’s efforts to publish key scientific data, file patentable discoveries, and form partnerships with contract research organizations (CROs), contract development and manufacturing organizations (CDMOs), and suppliers: “The foundation is clearly there, and as a result, we believe we are undervalued significantly.”
    • Tango Therapeutics (Nasdaq: TNGX) shares rocketed 53% from $20.22 to $30.93 June 8 after the developer of precision oncology treatments based on synthetic lethality announced positive initial data from its ongoing Phase I/II trial (NCT06922591) assessing its next-generation, MTA-cooperative PRMT5 inhibitor candidate vopimetostat (TNG456) in combination with Revolution Medicines’ (Nasdaq: RVMD) RAS(ON) inhibitors daraxonrasib (RMC-6236) and zoldonrasib (RMC-9805) in patients with MTAP-deleted and RAS-mutant metastatic pancreatic ductal adenocarcinoma (PDAC). Tango reported that 92% of patients with PDAC in the trial’s vopimetostat plus daraxonrasib arm achieved an objective response, while patients with second and third line PDAC treated with the combination showed a six-month progression-free survival (PFS) rate of 90% (median PFS not yet reached), suggesting durability of clinical benefit. Tango said it plans to finalize the design of a Phase III randomized-controlled trial of the combination approach in front-line pancreatic cancer and disclose vopimetostat lung cancer monotherapy data in the second half of 2026.

    The post StockWatch: Parabilis Medicines Makes Wall Street History with $770.5M IPO appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Collagen Resides Inside Cells in Liquid Condensate-Like Form https://edusehat.com/en/collagen-resides-inside-cells-in-liquid-condensate-like-form https://edusehat.com/en/collagen-resides-inside-cells-in-liquid-condensate-like-form Sun, 14 Jun 2026 23:55:06 +0700 Edusehat Collagen, Resides, Inside, Cells, Liquid, Condensate-Like, Form A study by scientists at the Centre for Genomic Regulation (CRG) in Barcelona has found that collagen, the protein that builds skin, bones, tendons, and organs, exists inside cells as a liquid-like droplet rather than as the long, rigid rod-like structure we might find in textbooks.

    The team used techniques including high-resolution live-cell imaging to generate what they say is the first direct observation of how the most abundant protein in the human body, which accounts for around a third of total protein mass, exists naturally inside living cells.

    Collagen is built inside a cellular compartment called the endoplasmic reticulum (ER). The study specifically looked at a precursor form inside cells called procollagen 1 (PC1), which matures into type 1 collagen. Type 1 collagen is the most common type of collagen, consisting of around 90% of the body’s total collagen.

    “Inside a cell, collagens are not rigid molecules as one had assumed,” said ICREA research professor Vivek Malhotra, PhD, senior author of the study at the CRG. They are, in fact, very pliable, taking a liquid condensate form much like oil in a drop of water.”

    The liquid-like state may serve a protective function. Collagen’s job, once outside the cell, is to assemble into the rigid fibers that hold tissues together. The same process inside the cell would be catastrophic. “This is another way by which cells ensure that collagens probably never become fibrous inside the cell,” said Malhotra. “Because if it were to become fibrous, it would kill the cell.”

    The new findings have implications for how the body exports its primary structural building block from production sites inside cells. The researchers suggest cells avoid using conventional receptors or vesicles, which is the route established by work carried out in the 1980s and 1990s and recognized with a Nobel prize in 2013.

    Instead, they propose a “liquid extrusion” hypothesis, whereby collagen moves from its site of synthesis to the next compartment of the secretory pathway through capillary action. The new theory has important implications for wound healing, fibrosis, and cancer.

    Malhotra and colleagues describe their study and results in a paper in the Journal of Cell Biology titled “Procollagen 1 assembles into phase-separated condensates in the endoplasmic reticulum.”

    “Procollagen I (PC1) is assembled into a trimer within the lumen of the endoplasmic reticulum (ER),” the authors explained. Under a microscope, purified collagen looks like long, rigid rods of up to 400 nm in length, and this conformation is presumed to represent their assembled state in vivo, the team continued. “However, there is currently no direct experimental evidence demonstrating that PC1 adopts or is maintained in such a rigid, extended conformation within the ER lumen in vivo,” they wrote. Also, the vesicles that transport proteins out from their site of synthesis to the cell’s exterior are only 60 to 90 nanometers in diameter.

    Since collagen’s structure was first described more than half a century ago, the field of cell biology has asked how such large molecules can be transported out of cells. The canonical picture of the protein describes collagen only after it has left cells and assembled into the fibers that hold tissues together. The newly reported findings suggest that inside the cell, collagen is not yet assembled into that rod structure.

    Using high-resolution live-cell imaging of human hepatic stellate cells—the liver cells that produce collagen and drive scarring in liver fibrosis—the team showed that collagen inside the cell gathers into small droplets that merge, split, and exchange material with their surroundings. These are all signatures of a condensate, compartments of proteins that become so concentrated they disassociate from their surroundings, like droplets of oil in water.

    Most of cell biology has focused on condensates in the nucleus and on stress granules in the cytosol, said first author Soumya Bhattacharyya, PhD, a postdoctoral researcher in Malhotra’s lab. “We’re just beginning to understand condensates inside the endoplasmic reticulum.”

    The findings emerged from microscopy images taken by Bhattacharyya in May 2024. Bhattacharyya was using the liver cell system as a tool to study what happens when collagen production is increased in fibrotic cells. “I had no idea what it would lead to. But when we took the samples, what struck me were these bright spherical structures you can’t miss,” recalled Bhattacharyya.

    The initial reaction in the laboratory to a finding that challenged cell biology dogma was sceptical. “I thought it must be an artefact,” said Malhotra. In the months that followed, the team had to settle whether the protein clumping they observed inside the endoplasmic reticulum was junk. Cells have an elaborate system for detecting badly folded proteins and either refolding them or marking them for destruction, centered on a chaperone called BiP.

    If the collagen droplets were heaps of misfolded protein, the researchers would detect high levels of BiP. The droplets contained, instead, a mixture of helper proteins, including chaperones that specifically recognize properly folded collagen.

    Human liver cells showing collagen droplets inside the cell (green clusters), held in place by TANGO1 (magenta), with extracellular collagen fibres visible as the surrounding network. Cell nuclei are stained blue. [Soumya Bhattacharyya / Centre for Genomic Regulation]
    Human liver cells showing collagen droplets inside the cell (green clusters), held in place by TANGO1 (magenta), with extracellular collagen fibers visible as the surrounding network. Cell nuclei are stained blue. [Soumya Bhattacharyya/Centre for Genomic Regulation]
    The study also clarifies the function of TANGO1, a protein discovered by the Malhotra lab roughly two decades ago and known to be required for collagen export. When the researchers depleted TANGO1, the collagen droplets still formed but were no longer positioned at the ER exit sites (ERES) where cargo leaves the compartment. Collagen secretion dropped accordingly. “PC1 condensates were still formed after TANGO1 knockdown, indicating that TANGO1 is not required for condensate formation per se,” the investigators stated. “However, TANGO1 depletion caused a marked reduction in the association of PC1 condensates with ERES …”

    The discovery suggests TANGO1 acts as a mooring point that holds the droplet at the export site rather than as a conventional cargo receptor. The authors propose that collagen then leaves the cell by a physical process called wetting, in which the liquid droplet attaches to and flows through the exit site.

    Malhotra offers two possible physical mechanisms for this transfer. “Imagine you have a rubber ball with a nozzle, filled with liquid. You squeeze it, you force the liquid to come out of this little orifice. Is that the mechanism? Or is the liquid rising by capillary forces, just like nutrients flow up against gravity in plants by capillary action?”

    The proposed liquid extrusion mechanism remains a model, but the next experiments to obtain direct visualization of the export mechanism are already underway. The team also plans to develop a mouse model, in collaboration with external partners, to confirm the findings in living tissue. If the model is confirmed, the work has implications for several pathological conditions in which excess collagen secretion plays a central role, including liver, lung, and skin fibrosis, as well as for targeting the dense matrix that tumors use to shield themselves from chemotherapy and the immune system.

    “One of the major problems in cancer is that the cells secrete so many collagens and other proteins out into the extracellular matrix that they hide in a shell made of these components and become chemo- and immuno-refractory, meaning they are not seen by the chemical therapeutics or by the immune system,” Malhotra said. “People are trying to find ways to break this tissue cement, and our study could help inform those strategies.”

    The proposed collagen secretion model suggests that either degrading TANGO1 to prevent cargo from being captured at the exit site or dissolving the condensate itself to prevent the cargo from being properly organized in the first place could be new strategies worth exploring.

    The post Collagen Resides Inside Cells in Liquid Condensate-Like Form appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines https://edusehat.com/en/sonothera-raises-125m-to-develop-ultrasound-mediated-genetic-medicines https://edusehat.com/en/sonothera-raises-125m-to-develop-ultrasound-mediated-genetic-medicines Sat, 13 Jun 2026 04:45:09 +0700 Edusehat SonoThera, Raises, 125M, Develop, Ultrasound-Mediated, Genetic, Medicines Biotechnology company SonoThera has raised $125 million in an oversubscribed Series B financing round. The financing was led by Vida Ventures, with participation from ARK Invest, CureDuchenne Ventures, Leaps by Bayer, Otsuka Pharmaceutical, SymBiosis, UCB Ventures SA, Vivo Capital, and existing investors ARCH Venture Partners, Alexandria Venture Investments, Duquesne Family Office, Illumina Ventures, Johnson & Johnson Innovation – JJDC, Medical Excellence Capital, RA Capital, and Vertex Ventures HC.

    SonoThera will use the funds to advance its lead programs in Duchenne muscular dystrophy (DMD) and autosomal dominant polycystic kidney disease (ADPKD) in the clinic. The funds will also support efforts to expand its pipeline of targeted redosable genetic medicines across multiple organ systems and scale its proprietary platform technologies for safe, targeted therapy delivery.

    The company’s platform combines a proprietary ultrasound-mediated delivery technology dubbed RIPPLE™, with a payload engineering platform dubbed PORE™. The platforms are designed to support the development of DNA and RNA therapeutics, gene editing, and gene silencing approaches. SonoThera is using its tech to develop genetic medicines that it claims will address key limitations of conventional gene therapies including delivery challenges, payload size constraints, immune responses, safety events, and difficulties with redosing. 

    As Kenneth Greenberd, PhD, SonoThera’s co-founder and CEO, stated “we founded SonoThera to take a fundamentally different approach, with a platform designed to broaden the therapeutic possibilities of the field. We believe our technology has the potential to expand the range of diseases addressable by genetic medicines while enabling more precise, durable, safer, and repeatable therapies for patients.”

    SonoThera has already demonstrated the targeted delivery and expression capabilities of its platform across multiple tissues, including skeletal muscle, heart, liver, kidney, adipose, and brain. It has also shown that it can deliver large payloads such as full-length dystrophin for DMD and RNA-based payloads for gene silencing applications in preclinical studies. 

    The company expects to initiate its first clinical trial in DMD in 2027.

    Commenting on the financing, Rajul Jain, MD, managing director at Vida Ventures, said “we believe SonoThera, with its RIPPLE delivery and PORE payload engineering technologies, has the potential to unlock opportunities in diseases with significant unmet need that have been previously inaccessible to other genetic medicine approaches.” 

    In connection with the financing, Jain and Rakhshita Dhar, MS, vice president & head of Healthcare Venture Investments at Leaps by Bayer, have joined SonoThera’s Board of Directors.

    The post SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Hantavirus One-Shot mRNA Vaccine Fully Protects in Syrian Hamster Model https://edusehat.com/en/hantavirus-one-shot-mrna-vaccine-fully-protects-in-syrian-hamster-model https://edusehat.com/en/hantavirus-one-shot-mrna-vaccine-fully-protects-in-syrian-hamster-model Sat, 13 Jun 2026 04:45:07 +0700 Edusehat Hantavirus, One-Shot, mRNA, Vaccine, Fully, Protects, Syrian, Hamster, Model Last month, the Andes virus outbreak on a Dutch cruise ship departing from Argentina brought a transmission context for hantavirus, that was previously unprecedented, to the forefront. The Andes virus is the only member of the hantavirus family that is capable of efficient person-to-person spread through close contact with respiratory secretions. Other hantaviruses are typically spread through contact with infected rodents, making the Andes virus a much more significant public health threat.

    While at sea, the outbreak spread among passengers and crew, infecting 13 people and killing three. The cruise passengers have since returned to their home countries, 23 in total. Because a person can carry the virus for weeks before showing any symptoms, health agencies are facing a complex challenge of identifying everyone who was exposed. There are currently no vaccines or preventive treatments approved for the virus; this travel-related outbreak brought the need for vaccine development to the forefront.

    Researchers at The University of Texas Medical Branch (UTMB) had previously developed and tested two mRNA vaccines against intramuscular Andes virus challenge in golden Syrian hamsters (“1-methylpseudouridine-modified or non-modified mRNA modalities encoding the envelope glycoproteins, Gn and Gc, in a single open reading frame.”)

    When tested in the Syrian hamster model, both mRNA vaccines were efficacious in hamsters using a two-dose regimen. Recognizing that a fast-moving international outbreak doesn’t allow time for patients to wait weeks between shots, the team retested the vaccines to determine whether a single dose would be effective.

    Now, a new report shares the finding that the vaccine provided full protection against the Andes hantavirus after a single dose.

    This work is published in The Lancet in the paper, “Single-dose mRNA vaccines against Andes hantavirus.”

    Alexander Bukreyev, PhD, head of the Laboratory of Viral Pathogenesis and Vaccine Development at UTMB, said that the group is working to fast-track these single-dose vaccines into human clinical trials.

    The results exceeded expectations. When testing the vaccines in an animal model that mimics human disease, the scientists found that a single shot provided 100% protection against a lethal dose of the virus. Even when the researchers significantly lowered the dosage to a fraction of the original amount, the results remained definitive.

    “Every vaccinated animal remained completely healthy and showed no symptoms or weight loss,” said Michelle Meyer, PhD, senior scientist in the Bukreyev Laboratory. “When we looked at the tissues from the vaccinated animals a month after infection, the virus was entirely gone. The vaccines triggered a powerful immune response, creating protective antibodies in as little as 14 days.”

    Because the Andes virus can take a relatively long time to make a human severely ill, these fast-acting vaccines could serve a dual purpose, possibly functioning as an emergency tool for people who have already been exposed.

    “If given quickly to high-risk contacts during an outbreak, such as the Andes virus situation on the cruise ship, the vaccines could theoretically jump-start their immune systems fast enough to intercept the virus—stopping it from replicating and preventing them from getting sick or spreading it further,” Bukreyev said.

    The post Hantavirus One-Shot mRNA Vaccine Fully Protects in Syrian Hamster Model appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Why “reprogramming” is the buzziest approach to reversing aging right now https://edusehat.com/en/why-reprogramming-is-the-buzziest-approach-to-reversing-aging-right-now https://edusehat.com/en/why-reprogramming-is-the-buzziest-approach-to-reversing-aging-right-now Fri, 12 Jun 2026 21:40:03 +0700 Edusehat Why, “reprogramming”, the, buzziest, approach, reversing, aging, right, now
  • Reprogramming is the new frontier in anti-aging research: Scientists are exploring ways to return cells to a younger state, building on a Nobel Prize–winning discovery that certain genetic factors can transform adult cells into stem cells capable of becoming virtually any cell type.
  • Big money is flooding in: Billions of dollars from billionaires like Yuri Milner and Sam Altman are backing companies like Altos Labs and Retro Biosciences, signaling serious investor confidence in reprogramming's potential to extend healthy human lifespans.
  • Past anti-aging trends have stumbled: Earlier excitement around telomere lengthening and "zombie cell" removal faded after disappointing human trials—a cautionary reminder that promising mouse studies don't always translate, and reprogramming faces the same unproven leap.
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    Earlier this week, Life Biosciences, a biotech company focused on reversing age-related diseases, announced that it had dosed its first volunteer. A person with glaucoma has had an experimental treatment injected straight into their eyeball.

    The idea is to try to treat the disease—which can cause vision loss—by regenerating healthy nerves in the eye. But David Sinclair, the chairman and cofounder of the company behind the trial, hopes to go further. If the treatment can reverse glaucoma, perhaps similar treatments can reverse other diseases of aging. Maybe, just maybe, they can reverse aging altogether.

    The approach is designed to work by “reprogramming” cells to a younger state. It’s one of many strategies being explored by biotech companies looking to slow and reverse the process of aging. But of all of them, it seems to be the one that is truly taking off.

    Aging is complicated. As we get older, we experience so many changes across pretty much all our biological systems. Scientists have tried to categorize these effects. In 2013, one team published a seminal paper describing nine “hallmarks of aging.” That list features many of the processes scientists have attempted to target. But some of those targets have fallen in and out of fashion over the years.

    Take telomere attrition, for example. Telomeres are DNA sequences at the ends of our chromosomes, often likened to the plastic caps that stop the ends of our shoelaces from fraying. When cells divide, telomeres shorten until, eventually, the DNA is vulnerable to damage.

    When I started reporting on aging, telomere shortening was all the rage. Shrinking telomeres had been linked to age-related diseases of the heart and brain. Shortened telomeres were considered a sign of premature aging. In 2017 Liz Parrish, CEO of the biotech company BioViva, injected herself with an experimental gene therapy that she hoped might lengthen her telomeres.

    Then it suddenly seemed to go out of style. Research continued, but all the excitement within the aging and longevity community seemed to move on to another hallmark. (Parrish also continued with self-experimentation; she calls herself “the most genetically modified person on Earth.”)

    That hallmark was cellular senescence. This happens when cells stop dividing but don’t die, instead entering a “zombie” state in which they churn out chemicals that can cause harmful inflammation.

    Senescent cells gradually accumulate in pretty much every organ studied, where they are thought to contribute to age-related damage. Why not just periodically clear them out? When a team of scientists took that approach in mice in 2011, they found they could delay the onset of age-related conditions like cataracts and hunchback. The treated mice even looked younger.

    But when scientists at Unity Biotechnology trialed a similar approach in people with osteoarthritis and an age-related eye condition in the late 2010s and early 2020s, the results were disappointing. The company laid off every employee in May last year and has since shuttered entirely.

    Again, that doesn’t mean senolytic drugs that target “zombie cells” won’t work. But it feels as if many in the field have moved on. These days, the buzz is all about ✨reprogramming✨.

    The idea here is to essentially return cells to a young state. It’s based on the Nobel Prize–winning discovery that four genetic factors can turn an adult cell into a stem cell, which can be encouraged to develop into pretty much any other cell type.

    Some promising studies in mice suggest that this approach might help wind back the clock. It seems to improve tissue healing, restore vision, and even improve learning and memory.

    Running parallel to all this research are repeated injections of hundreds of millions of dollars in funding. In 2021, my colleague Antonio Regalado reported on the founding of the biotech company Altos Labs to pursue reprogramming for rejuvenation.

    Altos was funded by the billionaire Yuri Milner—reportedly along with Jeff Bezos, among others—to the tune of $3 billion, a previously unheard-of figure for a biotech startup. Other well-funded companies have since sprung up in this space.

    There’s Retro Biosciences, for instance, which is pursuing reprogramming (among other approaches) in an effort to add 10 years of healthy life to human lifespans. Retro’s launch was supported by $180 million from OpenAI’s Sam Altman. Last month, the company announced a valuation of $1.8 billion.

    NewLimit, another billionaire-backed biotech exploring reprogramming, says it has promising results from research in mice. It plans to trial a drug designed to rejuvenate the liver in people next year. Last week, the company announced it had raised $435 million toward reaching that goal, among others.

    Life Biosciences, which was founded by the Harvard biologist David Sinclair, most recently secured $80 million to support its research. The eye trial is now officially underway, but Sinclair also has plans for whole-body rejuvenation. Earlier this week, he told my colleague Antonio that he plans to test a “highly, highly confidential” oral reprogramming drug as part of a $101 million competition organized by the XPrize Foundation. 

    Reprogramming has certainly caught the attention of scientists, biotech companies, and investors. Studies in mice are hugely promising. Human trials are launching. And research in the field has billions of dollars’ worth of support.A lot of people in the field are really excited about reprogramming. But it comes with risks. And we still don’t know if it will work. The question now is: Do we finally have a rejuvenation drug within reach? And if not, what will the next research trend look like?

    This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.

    ]]>     Inside Interoception: The hidden sense of how you feel inside https://edusehat.com/en/inside-interoception-the-hidden-sense-of-how-you-feel-inside https://edusehat.com/en/inside-interoception-the-hidden-sense-of-how-you-feel-inside Fri, 12 Jun 2026 21:40:02 +0700 Edusehat Inside, Interoception:, The, hidden, sense, how, you, feel, inside MIT Technology Review Explains: Let our writers untangle the complex, messy world of science and technology to help you understand what’s coming next. You can read more from the series here.

    Your brain lives in the dark space of your skull. Yet it knows when the wind lifts the hairs on your skin, when your heart is racing, when your gut tightens with fear.

    It’s also, right now, predicting what you’ll read next as your eyes move across this page. It’s picking up signals that help it make sense of what’s happening around you and prepare you to act if you need to stay safe. You aren’t usually aware that your brain is doing all that.

    Our senses take in information at a staggering rate—roughly 11 million bits flood in every second from our skin, eyes, ears, and more. That’s nearly three paperback novels’ worth of data every second. Only a sliver reaches our conscious awareness.  Researchers estimate that our conscious minds can process roughly 10 to 60 bits of information per second, about the rate at which you’re reading this sentence. That’s a ratio of about one conscious bit to hundreds of thousands of unconscious bits.

    And that’s a mercy. As Moriah Thomason, a neuroscientist at NYU Langone, says, “Thank goodness we’re built like this. There’s a layer of what we have access to in conscious awareness. And then we have a right-under-the-surface amount. There is only a certain amount we are meant to ‘hold in mind’ in order to function successfully.” 

    What you are aware of: Your stomach growling when you’re hungry. Your palms sweating before you speak in public. The breath you just took, if you pay attention to it. Even your heartbeat, which some people can sense from the inside without feeling their pulse in their wrist.

    Scientists have a word for how we sense ourselves from the inside: interoception. 

    The term was coined in 1906 by the British neurophysiologist Charles Sherrington. For most of the 20th century it remained largely confined to textbooks. Today, thanks to a 2021 Nobel Prize and new tools that can map the interoceptive system across the body, the study of this facility is suddenly quite hot. As researchers decode how signals move between body and brain, a clearer picture is starting to take shape—with implications for how we understand and treat conditions from obesity to chronic pain to anxiety.

    The field began to take off in the 1990s. In 1994, the neurologist Antonio Damasio published a book with a pointed title: Descartes’ Error. He challenged the historical separation of thinking and feeling, arguing that our ability to choose and act is driven by feelings, and those feelings in turn are shaped by the body’s signals, such as your gut clenching or your skin going clammy. When we lose that connection between feeling and thinking, as one of Damasio’s patients did after surgery to treat a brain tumor, we may still be able to reason with perfect logic about the pros and cons of traveling on a Tuesday or a Wednesday. But without the emotional signals that help us predict what a choice will feel like, our reason spins and circles, and we cannot decide.

    A contemporary of Damasio’s, the neuroscientist Bud Craig, spent his career asking one question: How do you feel? He charted how the brain builds an inner map of the body and updates it in real time every moment you are alive.

    Think of the captain’s bridge on the USS Enterprise, where a live map displays the status of the ship’s critical systems: oxygen levels, energy availability, hull integrity, shield strength. Another set of indicators senses things outside the ship: asteroid belts, enemy ships, radiation, life signs, and spatial anomalies not yet understood.

    Your brain, only about the size of your two fists pressed together, creates a map like this for your entire body, along with a map of the outside world, from data streaming in through your five senses. Together, they feed into your brain’s working model of you in the world, now and across time—where you are, who you are, your expectations for what’s about to happen (based on everything you know), and what all that means for you.

    When someone asks “How are you doing?” we consult our maps and report back on our status. We might say we’re happy, depleted, anxious, or energetic. These feelings are always a braid of emotional and physical sensations. They’re what your interoceptive navigational system serves up to your awareness when you sense yourself from the inside.

    As we grow up, we learn to interpret what these sensations mean—interpretations that, in turn, can alter our physiology, emotions, and behavior. Research by the psychologist Alia Crum shows that people who embrace a “stress is enhancing” mindset produce more growth hormones than people who have a “stress is debilitating” mindset. They also experience more positive emotions and greater cognitive flexibility.

    Language also matters. We learn words for the textures of our feelings—words that then shape how we feel and act. People low in emotional “granularity”—as the psychologist Marc Brackett calls the ability to distinguish between closely related feelings—react more impulsively under stress and are less able to find meaning in difficult experiences. But mindsets and emotional intelligence are malleable. We can learn that “anxious” is different from “terrified,” and we can even reframe how we interpret our body’s sensations. Instead of thinking of the butterflies in our bellies as annoying, we can welcome them as our body’s way of preparing us for a peak performance.

    Scientists have long understood that the interoceptive information informing these lived experiences travels via two major systems: nerves and humors (blood and lymph). Now they’re actively studying a third system—the “interstitium,” a network of fluid-filled spaces woven throughout the body’s connective fascia that may also play a role in communication.

    But until recently, scientific understanding of this interoceptive system looked like a high-level schematic that left out vital details—how information travels from the outside environment in, how it moves from your body to your brain, and how it is integrated and interpreted within your brain. Researchers are now racing to explore what the neuroscientist Catherine Tallon-Baudry calls this “new continent of awareness.”

    The wandering highway

    One of the most active areas of research centers on the vagus nerve, the main component of the parasympathetic nervous system and an information highway carrying news from your organs up to your brain and back down to your body. The vagus has become a celebrity nerve, ubiquitous in wellness podcasts and trauma therapy. “Tone your vagus nerve.” “Activate your parasympathetic system.” The language suggests a single thing you can target, like a muscle. The reality, as Steve Liberles at Harvard Medical School is discovering, is far more interesting.

    Liberles has spent most of his career mapping what he calls “the great wide unknown” of one of our largest and longest nerves. He speaks the way he works—methodically, without overselling. But the questions driving him are huge. How do we sense our body’s inner state? What information flows through which channels? And how does the brain decide what to do with it?

    “When I’m nervous giving a talk in front of a thousand people,” he says, “my heart might race. I might get butterflies in my stomach. I might get goosebumps on my skin.” We all know what he’s talking about.

    “It’s bizarre,” he muses. “Your brain has to send a signal to the gut, and then the gut back to the brain, to tell you you’re nervous?” He pauses. “This just shows there is this intimate connectivity between the brain and the body that’s real.”

    The vagus is often called the calming nerve, because it controls “rest and digest” functions that quiet our body after the sympathetic nervous system revs us up with “fight or flight” impulses to handle danger or stress. 

    But it is also doing something else: It’s listening to us inside. Anatomists have known for over a century that roughly 80% of its fibers carry information upward, from body to brain. Think of it as a two-lane highway with far more traffic headed north. What scientists are just beginning to understand in detail is what those signals are saying. 

    Liberles is decoding the vagus with molecular precision and finding that its messaging system is unexpectedly diverse. So far, his research has uncovered dozens of types of vagus nerve cells, each wired to a specific organ. Team Red relays information about the heart; Team Blue, the gut.

    Within those teams, each courier has a unique job that’s different from those all its teammates perform. Liberles found 10 types in the lungs alone. Until then, only one lung reflex had ever been identified, in 1868. One nerve courier carries information about breathing rate; another the stretch of your lungs; yet another information about airway threats, like food going down the wrong pipe.

    “It’s super exciting to think about what each of these neurons is doing,” he told me in a conversation last fall, a flash of intensity breaking through the calm. “Where does it go in the body? What is it sensing? What is it controlling?”

    The doors of the cell

    Liberles is mapping the vagus information highways. But highways need on-ramps for signals to enter. For years, one of neurobiology’s biggest mysteries was the molecular on-ramp for our sense of touch.

    Somewhere, something in our bodies was converting physical force into an electrical signal that the nervous system could understand. But no one knew how. 

    Solving that mystery required a scientist willing to trust a hunch when the data couldn’t show the way. 

    Ardem Patapoutian grew up in Lebanon and fled the country’s civil war at 18, landing in Los Angeles, where he delivered pizzas and wrote horoscopes for a local newspaper before falling in love with science at UCLA.

    In the 1990s, as a postdoc at the University of California, San Francisco, he became fascinated with our sense of touch—the last of the five major senses not yet understood at the molecular level. The lung stretch signal that Liberles’s vagus neurons carry to the brain? No one had ever figured out how that signal began.

    “How do you feel the embrace of a loved one? How do your fingers distinguish one texture of hair from another?” Patapoutian invites us to wonder in his 2021 Nobel Prize lecture. The problem: Most cellular communication works through chemistry. But mechanical force offers no molecule to bind. How does the body translate physical pressure into the electrochemical language that neurons speak?

    Scientists knew that the answer had to be an ion channel—a protein gate embedded in cell membranes that opens to let electrically charged particles into the cell. But tracking down the one responsible for touch turned out to be absurdly difficult. Ion channels are a hundred thousandth the size of a cell, invisible to ordinary microscopes. Worse, they don’t resemble each other. You can’t recognize one by its shape or its sequence of amino acids. Even with one right in front of you, nothing would tell you it was there.

    At Scripps, where he works now, Patapoutian decided to try an unusual approach. He’d try to find cells that showed sensitivity to touch and destroy their internal genetic blueprint one gene at a time—hunting for the move that would make the cell go numb. It was tedious, expensive, and possibly a dead end. “A lot of people made fun of us,” he says.

    Two years in, Patapoutian’s collaborator Bertrand Coste had burned through half his postdoctoral appointment with no results. Patapoutian said: Another 30 genes, and then we decide whether to continue.

    What kept them going, Patapoutian told me, was informed intuition. “As you gain more experience, you have this sense of what’s going to work, what’s not going to work. Sometimes the data cannot answer the question of when to stop or when to continue. There has to be another process. If you start trusting it, it gives you an avenue to continue.”

    Coste knocked out candidate gene 72. Flatline. The cell had gone numb.

    They’d found it—the mechanism behind something you feel every day.

    They named the protein they identified PIEZO, from the Greek piezi, meaning pressure. There are two variations, PIEZO1 and PIEZO2, each responsible for sensing different kinds of pressure in the body. They’re elegant in their design—over 2,500 amino acids folded into a three-bladed propeller-shaped gate embedded in cell membranes. When pressure stretches the membrane, the gate opens and electrically charged ions flood through, translating physical pressure into an electrical signal that the brain can understand—all within milliseconds.

    Patapoutian calls scientific discovery a dream that survives reality. He won the Nobel Prize in medicine in 2021 for his discovery of PIEZO, sharing the award with David Julius of UCSF for his work on how cells sense temperature. Now researchers are finding PIEZO proteins everywhere—skin, organs, blood vessels, and even red blood cells, where they help the cells squeeze through narrow capillaries. They’re how your brain knows where your hand is in space without looking at it, a sense called proprioception. They’re in plants too, enabling roots to sense pressure as they push down into the earth.

    PIEZO was just the beginning. With a $14.5 million grant from the US National Institutes of Health, Patapoutian and his collaborators are now mapping the body’s entire interoceptive system—as many internal senses as he can find, he says.8

    Patapoutian has translated his discovery into a unique form of public outreach. At scientific conferences, he sometimes rolls up his sleeve mid-lecture to reveal half his arm covered in ink—a gigantic PIEZO protein in exquisite anatomical detail, its blades spreading across his biceps. Then he flexes. The tattoo flexes with him, the structure bending exactly as the real protein does when pressure opens the gate.

    “At a pub or a party,” he explains, smiling, “how else would I demonstrate this beautiful structure?”

    Orchestrating the field

    Steve Liberles is mapping a major interoception highway. Ardem Patapoutian discovered the gates of touch. Meanwhile, Wen Chen at the National Institutes of Health is pulling the field together, putting neuroscientists, immunologists, physiologists, and clinicians into the same room. The demand, she says, has been enormous.

    She tested her pitch at a dinner party with NIH colleagues a few years ago. You’re hungry right now—that’s interoception. You’re thirsty—that’s interoception. Heads nodded as she pointed around the table.

    “We can’t have just the brain or just the body,” she told me. “We need to look at the whole person.”

    In 2018 she organized a symposium on interoception where Liberles was one of the invitees, along with researchers and practitioners of meditation and yoga. “It was not their thing,” she says, laughing as she recalls how uncomfortable some of the researchers looked. But the practitioners were excited to finally meet scientists who were studying the inner mechanisms of what they did.

    That was followed by a series of NIH workshops on interoception that spanned topics from basic science to clinical practice. Patapoutian was the keynote speaker for the first one. 

    The NIH began funding scientists to chart the neural circuits of interoception and bringing them together to talk about their findings. Partway through one of these meetings, the equipment failed for an hour. More than 1,000 people stayed online, waiting for it to come back.

    “We were shocked at the turnout,” she says. “There was much bigger interest than we could have imagined.”

    Chen is now building infrastructure to match the demand: a formal community, funding mechanisms, a venue where cardiologists and neuroscientists and clinicians can all find each other. And she’s redefining the field as she goes; interoception is not a one-way signal from body to brain but a continuous two-way communication system, each direction shaping the other in real time.10   

    Liberles’s nervousness on stage is that two-way loop in action. Signals from his racing heart and belly butterflies travel up to the brain, which weaves them into an interpretation: This is anxiety, and this is what to do to handle it. His actions produce fresh signals that the brain reads in light of its ongoing predictions about what will happen next. In the body-brain communication loop, each player constantly updates the other.

    I asked Wen what her work on interoception might mean for another inner sense: intuition. “People talk about ‘gut feelings,’” I said. “How does that relate to interoception?”

     “Intuition might be the bridge where interoception moves from unconscious processing to conscious awareness,” she answered. “If that’s true, then intuition is not magic. It’s physiology.”

    But it depends on how we read the signals. Intuition is like pain. It tells you something, but it’s not always clear what. “Perhaps we can treat intuition as a source of data,” she says. “Meaningful, but probably not complete.”

    “Maybe we can be grounded in both—in feeling and fact.”

    Which raises a more personal question: What do you do with the signals your body is sending?

    One avenue for exploration is therapeutic intervention—both pharmacological and neural stimulation. Vagal nerve stimulation has treated epilepsy and depression for four decades, but as Liberles puts it, it’s like pressing all the keys on the piano to hit one note. Weight-loss drugs like Ozempic act in part through vagal pathways but can cause nausea as a side effect, because the targeting isn’t precise enough. Map the body’s circuits with enough accuracy and you might hit the note you actually want.

    Another area of active research is psychological and behavioral—teaching people how to detect and even shape interoceptive signals. Low interoceptive awareness is linked to mental-health disorders and stress-related physical conditions.11 But like emotional intelligence, it’s not fixed. Researchers are finding that people can boost their body awareness by, for example, learning to detect their heartbeats from the inside—now a common measure of interoceptive awareness.12 Other interventions focus on body-based therapies and conscious activation of the parasympathetic “rest and digest” system to improve emotional and physical well-being. The placebo effect is another example of the mind acting on the body through expectation alone.

    The signals we once dismissed as vague feelings—when your gut tightens before you know why, when your body says yes or no before your mind catches up—those are real. How we interpret them and whether we act on them is another frontier.

    It’s clear that gut feelings play a role in scientific research, especially when the path forward looks foggy. Patapoutian’s informed intuition kept him and his colleagues going long enough to find PIEZO, a reminder that major discoveries often start with a hunch that is later tested against evidence. Chen puts it well: Maybe we can be grounded in both feeling and fact.

    Katherine W. Isaacs is a writer and senior lecturer at the MIT Sloan School of Management. Her teaching and research focus on the intersection of psychology, technology, and innovation. Originally trained as a biologist and later as a social psychologist, she is currently working on a book called Gut Feel, about intuition, interoception, and embodied decision-making.

    ]]>     Laser‑Driven Phase Contrast Enhances Cryo‑EM Resolution of Small Proteins https://edusehat.com/en/laserdriven-phase-contrast-enhances-cryoem-resolution-of-small-proteins https://edusehat.com/en/laserdriven-phase-contrast-enhances-cryoem-resolution-of-small-proteins Fri, 12 Jun 2026 10:45:11 +0700 Edusehat Laser‑Driven, Phase, Contrast, Enhances, Cryo‑EM, Resolution, Small, Proteins You know when you are at the eye doctor getting an updated prescription, and suddenly the world snaps into sharper focus? Physicists at the University of California (UC), Berkeley, have now done something similar for electron microscopy. By introducing phase contrast into a cryo‑electron microscope, they have delivered dramatically sharper images of some of biology’s smallest and most elusive proteins.

    The advance comes from a new laser phase plate (LPP), described in the paper “Laser phase plate improves structure determination of small proteins by cryo‑EM,” which was published recently in Science. Led by physicist Holger Mueller, PhD, of UC Berkeley and Lawrence Berkeley National Laboratory, the team demonstrated that a laser‑driven phase plate can overcome one of cryo‑EM’s most persistent limitations: poor contrast for small proteins.

    Cryo-EM images of two proteins, apoferritin and hemoglobin, taken without and with a laser phase plate. The images are analyzed in a computer to produce detailed 3D structures of the proteins. [Holger Müller, Jessie Zhang/UC Berkeley]
    Cryo‑EM has transformed structural biology over the past decade, earning a Nobel Prize in 2017 for enabling high‑resolution structures without crystallization. But despite its impact, the technique still struggles with proteins below ~70 kilodaltons—a size range that includes about 90% of the human proteome. “Because of signal-to-noise limitations, the majority of human and animal proteins are too small to be analyzed by these methods [cryo-EM and cryoelectron tomography]. The increase in signal-to-noise ratio provided by this laser phase plate is expected to overcome these important limitations.”

    The new LPP begins to address that problem. The LPP uses an intense, continuous‑wave laser to shift the phase of the electron beam itself. This produces true phase contrast without dimming or destabilizing the beam. Mueller described the laser focus as “75 kilowatts focused to a few microns… That’s more powerful than what you use for welding. It has more power than a military laser. It builds up the brightest continuous laser focus ever.”

    Installed in a custom Thermo Fisher Titan Krios, the LPP immediately improved the clarity and resolvability of small proteins, including hemoglobin, which sits at the lower limit of what today’s cryo‑EM instruments can handle. As the authors wrote in the abstract: “Here, we show that the laser phase plate (LPP)… enhances the resolution in single-particle reconstruction of small proteins by improving specimen-motion correction, recovery of information from the early frames, as well as particle visualization, 3D classification, and alignment.”

    phase plate cover Cryo-EM
    A laser (purple) is powerfully amplified by highly polished mirrors and focused on the electron beam (blue) to shift its phase and increase the cryo-EM microscope’s contrast, allowing biologists to image smaller proteins and the crowded structures inside cells. [Sayo Studio]
    These improvements were achieved using standard defocus ranges and reconstruction workflows. “For the most challenging cases—small particles, bad specimens—the laser produces a very considerable advantage,” Mueller said.

     

    The impact extends beyond single‑particle analysis. Cryo‑electron tomography (cryo‑ET), which assembles multiple angular views of a molecule or protein into a three-dimensional image, stands to benefit even more. “With cryo-ET, we’re looking at small, very complicated cellular material that’s incredibly crowded inside the cell,” said Bridget Carragher, PhD, founding technical director of imaging at Biohub. “It’s like a forest of trees, and you’re trying to find one leaf on one tree in there. Cryo-ET needs a dramatic step forward in contrast, so we can start to see what’s going on inside the cell. That’s what the laser phase plate promises to give us.”

    Biohub is developing a dual‑laser version of the system, designed to reduce component wear and minimize aberrations. Meanwhile, Mueller’s team is pushing toward imaging proteins as small as 17 kilodaltons, a threshold that would open access to vast regions of the human proteome previously invisible to cryo‑EM.

    “This technology is a step function change for biology,” said Stephani Otte, PhD, Biohub’s vice president of imaging science. “What was once invisible will become visible—and that changes everything about how we understand disease.”

    “The bottom line is, if you have a large protein and a really good sample—a fresh one or one frozen without bubbles, for example—you may not need the phase plate to get a single, high-quality image. But for a small protein and a bad sample, laser-on is best,” Mueller said. “This could fill an enormous gap in our knowledge of protein structures that can’t be crystallized or are too small for today’s cryo-EM. And it will be revolutionary for cryo-ET.”

    The post Laser‑Driven Phase Contrast Enhances Cryo‑EM Resolution of Small Proteins appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     New mRNA Delivery Platform Restores Muscle Function in DMD Models https://edusehat.com/en/new-mrna-delivery-platform-restores-muscle-function-in-dmd-models https://edusehat.com/en/new-mrna-delivery-platform-restores-muscle-function-in-dmd-models Fri, 12 Jun 2026 03:35:11 +0700 Edusehat New, mRNA, Delivery, Platform, Restores, Muscle, Function, DMD, Models Although gene therapy has shown promise for the treatment of Duchenne muscular dystrophy (DMD), the limitations of viral vectors have proven challenging to clinical advancement. Now, a new treatment platform delivered skeletal-muscle-targeted full-length DMD mRNA systemically in a murine model of DMD, successfully restoring the production of dystrophin, and dramatically improve muscle strength, endurance, and function in vivo.

    The approach uses allogenically engineered targeting extracellular vesicles (DMD t-EVs)— which offer distinct benefits over current viral-based gene therapies, including reduced side effects and the ability to transfer the entire DMD gene. The researchers engineered the EVs with special tags that directly target skeletal muscles after being injected into the bloodstream. The work also demonstrated the safety and biocompatibility of DMD t-EVs in non-human primates, supporting their translational potential.

    “Our new platform overcomes the limitations of current viral-based gene therapies, allowing for the delivery of full-length mRNA, restoring wild-type translation of dystrophin and significantly improving muscle function,” said Betty Kim, MD, PhD, in the department of neurosurgery at UT MD Anderson. “We are highly encouraged by these results, which provide a blueprint for mRNA-loaded EVs as a next-generation therapeutic strategy.”

    The study, published today in Nature Biomedical Engineering, is entitled, “Skeletal-muscle-targeted non-viral delivery of full-length DMD mRNA for Duchenne muscular dystrophy.”

    DMD is a severe genetic disorder caused by mutations in the DMD gene that prevent dystrophin production, which helps stabilize and protect muscle cells during contractions in healthy individuals. Without dystrophin, the muscles become easily damaged, leading to eventual inflammation and cell death. DMD primarily affects males, with symptoms such as delayed walking and waddling usually appearing in early childhood. As the disease progresses, it leads to loss of walking ability, scoliosis, heart problems and eventual respiratory failure.

    Because DMD is the longest known gene in the human genome, current viral-based gene therapies are unable to carry the full length. These limitations result in the loss of the gene’s full function and prevent challenges like dose-limiting toxicities, immune reactions, and other adverse reactions including death.

    These side effects have resulted in the removal of at least one Food and Drug Administration-approved gene therapy from the market and are why researchers have been trying to develop alternative ways of safely delivering the full-length DMD gene.

    In this study, the researchers loaded the full-length DMD mRNA into EVs that were engineered to specifically target and bind to skeletal muscles. Injection of these mRNA-loaded EVs led to an increase in dystrophin protein expression as well as improved muscle strength and function in preclinical models, with no serious side effects.

    Importantly, the treatment stayed on target inside of skeletal muscles and did not trigger any immune responses or toxicities commonly seen with viral-based treatments, even after repeated dosage.

    Future studies are needed to determine the full safety of EV-mediated mRNA platforms for clinical trials, including whether they can be delivered to cardiac muscles, as heart conditions are commonly seen in advanced disease. However, based on these results, the authors point out this could be a promising method beyond treating Duchenne muscular dystrophy, also potentially serving as a broader “protein restoration” or cellular reprogramming platform.

    “Given that we are now able to replace very large proteins, this platform- and disease-agnostic approach could potentially open doors far beyond rare genetic disorders and traditional gene therapy applications,” Kim said. “It’s possible this could ultimately enable restoration of proteins lost not only through inherited diseases but also from acquired or degenerative processes, including cancer, autoimmune disorders, neurodegeneration, fibrosis and other chronic diseases.”

    The post New mRNA Delivery Platform Restores Muscle Function in DMD Models appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Potential Cocaine Addiction Targets Identified Through Genetic Mapping in Rats https://edusehat.com/en/potential-cocaine-addiction-targets-identified-through-genetic-mapping-in-rats https://edusehat.com/en/potential-cocaine-addiction-targets-identified-through-genetic-mapping-in-rats Fri, 12 Jun 2026 03:35:10 +0700 Edusehat Potential, Cocaine, Addiction, Targets, Identified, Through, Genetic, Mapping, Rats Scientists at the University of California San Diego have reported the results of a genome-wide association study in rats that identified key biological drivers of cocaine addiction. Using a genetically diverse group of nearly 900 rats to map genetic markers associated with compulsive drug use, the researchers uncovered a potential new therapeutic target that resides in the liver rather than in the brain.

    Current research in this field often focuses on the brain, but the UC San Diego team’s findings suggest that how the body metabolizes cocaine may be just as critical in determining whether somebody develops an addiction.

    “Finding a liver-based enzyme that shapes cocaine-taking behavior was a real ‘aha’ moment for us,” said Olivier George, PhD, a professor of psychiatry at UC San Diego School of Medicine. The George lab led the addiction behavioral studies that provided the foundation for the research. “It reminds us that addiction isn’t only in the brain. It’s a complex puzzle involving how the entire body processes the drug.”

    George is co-corresponding author of the team’s published paper in Nature Communications, titled “Genome-wide association study of cocaine self-administration behavior in Heterogeneous Stock rats.”

    Cocaine use disorder (CUD) has a strong genetic component, the authors noted. “Twin studies estimate the heritability of cocaine dependence to be as high as 70%, a finding supported by recent comprehensive reviews,” they wrote.  GWAS have also uncovered a significant heritable component, the team continued, with single nucleotide polymorphism (SN)-based heritability estimated at 27-30%. However, scientists have struggled to pinpoint the specific genes that make certain individuals more vulnerable to addiction.

    “The paucity of significant and replicated associations for CUD limits our understanding of this disorder, hampering our ability to identify novel pharmacological targets,” the investigators added. Co-corresponding author Abraham A. Palmer, PhD, professor of psychiatry at UC San Diego School of Medicine, who led the project’s intensive genetic modeling and analysis, further commented, “Identifying those genes in an important goal, because drugs could then be developed to target those genes, shifting genetically susceptible people to become more like genetically resistant people.”

    To investigate further, the team carried out a GWAS in nearly 900 outbred Heterogeneous Stock (HS) rats—a model system capable of mimicking the vast genetic diversity found in human populations. By using HS rats the team was able to capture the critical differences between individuals who are genetically susceptible to addiction and those who are naturally more resistant. “Prior work has established the phenotypic diversity of HS rats across a broad range of addiction-relevant behaviors, including cocaine self-administration,” the researchers commented.

    “The extended access model allowed us to characterize escalating intake, increased motivation to take the drug, and compulsive-like behavior despite negative consequences.” In addition to the GWAS results the researchers carried out a range of secondary analysis strategies to uncover what they describe as novel genetic drivers of cocaine self-administration behaviors.

    Analyzing millions of genetic markers in each animal, the team discovered six major genetic regions linked to addiction-like behaviors, such as the escalation of drug intake and the time elapsed between doses. The researchers identified in the rats a specific group of carboxylesterase genes that are orthologous to the human CES1 gene, which are responsible for creating the enzyme that metabolizes cocaine. The study found that variations in these genes are closely linked to how frequently and compulsively rats self-administered the drug.

    The findings also replicated a known genetic link found in humans (Trak2), providing a vital translational bridge between animal research and human medicine. This replication strengthens the argument that the biological pathways identified in the lab could eventually lead to real-world therapies. “Genes associated with CUD in humans remain limited, however our GWAS identified one gene (Trak2) that has also been identified by human GWAS of CUD, and the novel identification of Ces1 offers a fresh avenue for future studies,” they stated.

    The collective findings suggest that by targeting the enzymes that metabolize cocaine with medicines, scientists might be able to alter how the drug affects the body, potentially reducing its addictive impact. In their paper they concluded “Our results replicate previous loci associated with CUD in humans and provide several novel biological insights including the potential of pharmacological strategies targeting carboxylesterases.”

    Palmer said, “This work showcases the power of long-term, team-science collaboration that pairs experts in rodent behavior with quantitative geneticists. A decade of coordinated effort across multiple cohorts and federal partners made possible a discovery that no single lab could achieve alone.”

    First author Montana Kay Lara, PhD, a postdoctoral researcher at UC San Diego School of Medicine, who helped bridge the gap between the study’s behavioral and genetic components, said, “Seeing the Ces1 signal validate a hypothesis that has been circulating for decades is incredibly exciting. It gives us a concrete target to test whether changing how cocaine is metabolized can blunt the drive toward compulsive use.”

    The research team is now moving into the next phase of the project, which involves investigating exactly how these genetic mutations change function of the enzyme. They also hope to use the study’s extensive Preclinical Addiction Biobanks—collections of blood, urine, brain and other tissue samples—to identify biological markers that could one day help predict an individual’s risk of developing a substance use disorder.

    The researchers hope that by leveraging this resource, they and other scientists working in this space will be able to translate genetic discoveries into diagnostic tools and new treatments that can help stabilize individuals struggling with addiction.

    The post Potential Cocaine Addiction Targets Identified Through Genetic Mapping in Rats appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Fighting Antimicrobial Resistance with Biomaterials and Phages https://edusehat.com/en/fighting-antimicrobial-resistance-with-biomaterials-and-phages https://edusehat.com/en/fighting-antimicrobial-resistance-with-biomaterials-and-phages Fri, 12 Jun 2026 00:00:10 +0700 Edusehat Fighting, Antimicrobial, Resistance, with, Biomaterials, and, Phages Antimicrobial resistance (AMR) is a significant global health threat, with its impact felt across all regions of the world.1 According to the World Health Organization (WHO), AMR is responsible for an estimated 700,000 deaths annually worldwide, and this figure is projected to rise to 10 million deaths per year by 2050 if current trends persist.

    Notably, the number of deaths attributable to AMR in many countries surpasses those caused by diabetes, kidney diseases, digestive disorders, and other non-communicable diseases. AMR has profound implications for clinical practice, affecting the management of infections across various healthcare settings. Here, we will discuss recent advances using novel biomaterials and phage for combating AMR.

    Wounds, in particular, are susceptible to colonization by AMR bacteria, complicating wound healing and increasing the risk of serious complications such as sepsis and amputation, which in turn exacerbates the chronic wound burden. Chronic wounds impact the healthcare system because of their increasing prevalence and cost. The rapid growth of AMR further limits the effectiveness of standard antibiotic therapies, necessitating the use of more potent and costly antimicrobial agents, which may have adverse effects and contribute to further resistance development.

    The most common bacteria isolated from chronic wounds include species of Staphylococcus (47–55%), primarily S. aureus and S. epidermidis, P. aeruginosa (25–33.6%), Acinetobacter spp., Enterococcus faecalis, and Enterobacteriaceae such as Escherichia coli, Klebsiella pneumoniae, and Enterobacter spp.2 Many of these bacteria have developed persistent AMR, such as methicillin-resistant S. aureus (MRSA). They are highly resistant to commonly used antibiotics and, therefore, limit treatment options for wound infection. To combat the growing threat of infected wounds with AMR bacteria, Han and colleagues devised a creative approach to directly degrade proteins responsible for bacterial growth.3

    UDP-N-acetylmuramoyl-L-alanine-D-glutamate ligase (MurD) is a prime target for combating antibiotic resistance in bacteria as it catalyzes the synthesis of peptidoglycan, the predominant structural component in bacterial cell walls. Han et al. developed a bacterial nanoinducer (bacNID) designed to specifically degrade MurD, effectively inhibiting the growth of both Gram-positive and Gram-negative bacteria.

    Two critical, interconnected challenges

    “Our paper addresses two critical, interconnected challenges in global public health and antibacterial therapy: 1) The crisis of antibiotic resistance. Bacteria rapidly evolve resistance to conventional antibiotics through mechanisms such as membrane permeability changes, target mutations, enzymatic inactivation, and efflux pumps; and 2)  The failure of the traditional drug development model. The pharmaceutical industry faces a >95% failure rate in developing new antibiotics. Even when new drugs are found, bacteria often develop resistance quickly, and many candidates suffer from poor pharmacokinetics, systemic toxicity, and an inability to selectively target bacteria over healthy host cells,” says Guangjun Nie, PhD, senior author of the paper and professor at the National Center for Nanoscience and Technology, Beijing, China.

    Nie adds that by moving away from the “one-target-one-drug” inhibition model, their study solves the problem of how to kill bacteria without giving them a chance to evolve resistance. “It achieves this by hijacking the bacteria’s own protein degradation machinery to destroy essential proteins such as MurD that are necessary for cell wall synthesis.”

    The team first conjugated MurD-targeting peptides (pMurD) on gold nanoparticles, alongside the addition of a rapidly degradable SsrA peptide tag. The role of the SsrA tag is to bind MurD, thereby “tricking” the bacterial ClpXP protease into degrading MurD. Gold nanoparticles function as a peptide delivery vehicle that is taken up directly by the bacteria to circumvent the potential membrane permeability barrier. In this way, bacNID can destroy MurD, which is needed to synthesize the cell wall, leading to bacterial death.

    bacterial research
    The rapid growth of AMR limits the effectiveness of standard antibiotic therapies, necessitating the use of more potent and costly antimicrobial agents, which may have adverse effects and contribute to further resistance development. [10174593_258/Getty Images]
    The team showed that bacNID was able to specifically inhibit model Gram-positive and Gram-negative bacteria with a dose-dependent degradation profile while exhibiting low cytotoxicity towards nontargeted mammalian cells. BacNID also specifically targeted MurD while sparing other Mur ligases. This approach can also be used with other nanoparticle vehicles, such as platinum, making it a versatile method for universal inhibition of diverse AMR bacteria.

    To improve mechanistic understanding, the team utilized a variety of techniques and discovered that bacNID-treated bacteria suffered from cell wall damage, leading to leakage of a significant amount of DNA and ATP. Interestingly, compared with conventional antibiotics, treatment with bacNID did not lead to the formation of resistance after sustained treatment.

    Using an in vivo infected skin wound model, the authors showed that bacNID treatment not only reduced infection burden but also promoted better wound healing outcomes, including greater skin cell proliferation, neo-angiogenesis, and lower inflammation. To expand the applicability of their method, bacNID was also tested in a S. aureus-infected nonhealing keratitis model and S. typhimurium-induced colitis model, showing great efficacy in both diseases.

    “While our current study focuses on MurD, a major future step is to apply the bacNID platform to degrade other essential bacterial proteins. Readers can expect the team to develop bacNIDs against different targets in various pathogenic bacteria (e.g., targeting virulence factors or other metabolic enzymes). Future iterations of bacNIDs may incorporate stimuli-responsive nanotechnology (e.g., pH or enzyme-sensitive linkages) to ensure that the degradation-inducing activity is activated only within the specific microenvironment of the infection site, further minimizing off-target effects,” says Nie.

    “We will also conduct more in-depth mechanistic studies to definitively elucidate why targeted protein degradation fails to induce the antibiotic resistance observed with conventional therapies.”

    Guangjun Nie, PhD, professor at the National Center for Nanoscience and Technology, Beijing, China, with his team of scientists in the lab. [Guangjun Nie]
    Guangjun Nie, PhD, professor at the National Center for Nanoscience and Technology, Beijing, China, with his team of scientists in the lab. [Guangjun Nie]

    Trick bacteria with bacteria

    Owing to an aging population, there is a rise in the use of implants, but these implants are prone to the formation of bacterial biofilm. The extracellular polymeric material in biofilm is known to reduce antibiotic penetration while creating an immunosuppressive environment, leading to impaired antimicrobial responses. In particular, orthopedic implants provide a conducive habitat for hematogenous bacteria for growth and formation of biofilm. Yang and colleagues hypothesized that bacteria that cause implant infection can be repackaged as drug carriers to penetrate biofilm for intra-film drug delivery.4

    “Genetically modified bacteria have emerged as a promising delivery platform for diverse biomedical applications, ranging from cancer immunotherapy to infectious disease treatment. However, the clinical translation of current live bacterial biotherapeutics remains hindered by two major bottlenecks: unresolved in vivo safety concerns and the requirement for sophisticated species-specific genetic engineering. By exploiting the inherent life cycle of biofilms, our chemically primed bacterial triggers enable localized drug release deep inside biofilm structures, achieving effective biofilm eradication across genetically distinct bacterial and fungal infection models,” says Wei Tao, PhD, senior author and professor at Harvard Medical School.

    The team first prepared bacteria by subjecting them to calcium chloride to increase membrane porosity and enhance their ability to uptake exogenous drugs like antibiotics. Ultraviolet radiation was then used to deactivate the bacterial membrane repair mechanism, creating irreversible membrane pores. They found that the modified bacteria, i.e., tricker, was able to migrate and thrive in biofilm and eventually, release exogenous drugs that are otherwise, challenging for delivery.

    As a biofilm matures, surrounding bacteria are known to be attracted to and integrated into it. The team first labeled their tricker bacteria with a fluorescent dye and found that the bacteria were integrated throughout the biofilm with 80% coverage. However, a caveat is that the integration is most effective if the tricker and biofilm bacterial species are the same. The team discovered that while modified S. aureus can penetrate the core of S. aureus biofilm in 60 minutes, modified E. coli barely penetrates S. aureus biofilm. Likewise, modified E. coli can penetrate the core of E. coli biofilm, while modified S. aureus can penetrate E. coli biofilm with a much lower efficiency. Once in the biofilm, the chemically modified and inactivated bacteria were found to lyse, especially at hypoxic and acidic conditions.

    Besides preventing antibiotic penetration, biofilm can also release bacterial-derived materials that suppress the immune system, particularly macrophages. For instance, it is well-characterized that S. aureus biofilms can bias macrophages towards an anti-inflammatory M2 phenotype, characterized by impaired antimicrobial peptide production, elevated arginase-1 (Arg-1), and attenuated inducible nitric oxide synthase (iNOS) expression. Interestingly, Yang and colleagues found that tricker bacteria were able to modify the metabolic states of the biofilm, resulting in enhanced production of I-arginine via iNOS to generate nitric oxide to improve bacterial clearance capacity.

    Using an in vivo model of subcutaneous implant infection, the team found that there was an observable increase in mature dendritic cell and M1-like macrophage activation in the lymph nodes. The amount of memory B cells and antibodies with antimicrobial immune memory functions was also increased. After primary bacterial inoculation and intervention, the team reintroduced MRSA and found that 86% of treated mice rejected MRSA while all mice in the control group succumbed to the infection. This finding suggested that treatment with tricker bacteria was able to evoke innate and adaptive immune system endogenously for better control of AMR, with potential for bacterial-specific systemic memory to prevent relapse.

    Finally, the strategy was tested in a murine bone infection model. By tracking cytokine levels and tissue histology, the team showed that their strategy was biologically safe. An MRSA rechallenge to the contralateral knee also led to a significant drop in biofilm burden in treated mice, providing convincing evidence of immune memory.

    “To advance its clinical translation, the antibacterial efficacy of this approach will be further validated in large animal models, including rabbits, pigs, and dogs. This strategy exhibits enormous potential for future clinical translation of personalized antibacterial therapeutics, which enables highly efficient and precise treatment by profiling patient-derived pathogens and designing tailored “tricker” bacteria. Moreover, the current approach is adaptable to polymicrobial infections. Future work will also explore the feasibility of combining modified bacteria with other antibacterial agents or functional materials to optimize therapeutic performance,” adds Tao.

    Wei Tao, PhD, professor at the Harvard Medical School (far right, first row) and his research team. [Wei Tao]
    Wei Tao, PhD, professor at the Harvard Medical School (far right, first row) and his research team. [Wei Tao]

    Using phage cocktail in clinical trials

    Biofilm-related vascular graft infections (VGIs) are a major therapeutic challenge attributing to persistent, antibiotic-resistant bacteria residing in retained grafts. Graft explant is not always possible due to patient factors and surgical technical challenges. To effectively preserve the graft, treatment of VGI is typically a prolonged course of parenteral antibiotics followed by long-term suppressive antimicrobial therapy. Yet, graft survival rate is low, and recurrent infection is common.

    Phages are viruses that specifically infect bacterial cells and can cause bacterial lysis. They have been shown to be active against both biofilm-forming bacteria and can even enhance antibiotic activity by eliciting phage-antibiotic synergies to combat AMR. Chung and colleagues made use of a phage cocktail to treat a 36-year-old female patient with refractory P. aeruginosa mediastinitis and vascular graft infection.5

    “Our paper addresses key translational barriers to effective treatment of VGI caused by multidrug-resistant, biofilm-forming pathogens. Firstly, antibiotic failure in biofilm-associated infections as VGI pathogens embedded within biofilms exhibit marked tolerance to antibiotics, leading to persistent infection and relapse despite prolonged therapy. Secondly, escalating AMR as resistant subpopulations emerge under antibiotic pressure, further limiting treatment options in already complex infections. Thirdly, the lack of timely, personalized therapy as conventional phage therapy workflows are slow, making timely intervention difficult in acute or deteriorating cases.

    Next, unpredictable phage–antibiotic interactions, such as phage-antibiotic synergy, are not reliably identified or optimised in routine clinical workflows. Finally, fragmented clinical-laboratory integration, as there is limited integration between real-time microbiology, pharmacology, and clinical decision-making to enable adaptive therapy,” says Andrea Kwa, PhD, senior author and associate professor at the SingHealth-Duke-National University of Singapore Medical School.

    The team set up a multidimensional evaluation workflow to identify the most suitable therapeutic phages from the Singapore Phage Repository. Screening began with phage susceptibility testing of the four P. aeruginosa clinical isolates using spot and plaque assays, before other assays to identify the most potent cocktail. As phages are highly immunogenic when administered intravenously, the team also performed systemic inflammation monitoring and found that the patient tolerated the phages well.

    The team found that their phage cocktail was able to restore antibiotic susceptibility by altering the efflux capacity of the bacteria. This positively impacted the antibiotic options for the patient. For instance, fluoroquinolone susceptibility was restored, resuscitating its use as an oral suppressive antibiotic for the long-term management of VGI.

    Kwa adds that building on this proof-of-concept, her team’s next phase focuses on scaling, standardization, and integration of timely bespoke phage–antibiotic therapy into routine clinical practice. “Our key future directions include scaling up of rapid-response phage platforms, such as expansion of phage libraries/repositories with well-characterized, clinically ready phages with faster turnaround for matching and deployment, overcoming current procurement delays. We will also develop standardized precision workflows for phage susceptibility testing and phage–antibiotic synergy testing.  Our team will also enhance regulatory and translational readiness of our technology for GMP-compatible production pipelines to enable scalable clinical deployment.”

    AMR is a serious healthcare issue affecting the world. With the rising use of antibiotics in farms and clinical settings, this problem needs to be taken seriously. Unfortunately, the development of antibiotics is slow and mostly unsuccessful, thus requiring a new approach for society to effectively treat AMR. Biomaterials offer a new avenue to deliver tricker bacteria into biofilm to improve intra-film drug delivery and to activate the suppressed immune system, while also inhibiting intra-bacterial growth mechanisms. Phage is also becoming a popular option, especially for personalized medicine, and this therapy may see even greater efficacy when combined with biomaterials such as hydrogel to improve its delivery and reduce systemic immunogenicity.

    References

    1. Bertagnolio S, Dobreva Z, Centner CM, et al. WHO global research priorities for antimicrobial resistance in human health. Lancet Microbe. Elsevier Ltd. 2024;5(11). doi:10.1016/S2666-5247(24)00134-4
    2. Uberoi A, McCready-Vangi A, Grice EA. The wound microbiota: microbial mechanisms of impaired wound healing and infection. Nat Rev Microbiol. Nature Research. 2024;22(8):507-521. doi:10.1038/s41579-024-01035-z
    3. Han L, Huang W, Pan X, et al. Utilizing nanoinducers for precision degradation of bacterial protein to mitigate antibiotic resistance. Nature Communications . 2025;16(1). doi:10.1038/s41467-025-66221-w
    4. Yang C, Saiding Q, Chen W, et al. Chemically modified and inactivated bacteria enable intra-biofilm drug delivery and long-term immunity against implant infections. Nat Biomed Eng. Published online January 16, 2026. doi:10.1038/s41551-025-01600-8
    5. Chung SJ, Liu Y, Thong S, et al. Timely bespoke phage-antibiotic combination to treat refractory Pseudomonas aeruginosa mediastinitis and vascular graft infection. Nat Commun. Published online January 9, 2026. doi:10.1038/s41467-025-68136-y

     

     

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    ]]>     Brain Aneurysm Study Identifies Structural, Immune Markers of Rupture Risk https://edusehat.com/en/brain-aneurysm-study-identifies-structural-immune-markers-of-rupture-risk https://edusehat.com/en/brain-aneurysm-study-identifies-structural-immune-markers-of-rupture-risk Thu, 11 Jun 2026 05:35:10 +0700 Edusehat Brain, Aneurysm, Study, Identifies, Structural, Immune, Markers, Rupture, Risk According to some estimates, stroke is the second leading cause of death globally. One of the causes of a severe type of stroke are brain aneurysms. Now data from a new study suggests that certain cells in the brain may cause aneurysms to weaken and rupture. And it helps explain why some aneurysms burst while others do not. It also opens a door to new ways of potentially predicting and preventing strokes. All of the findings are covered in a new Nature Neuroscience paper titled “Cerebrovascular vulnerability and fibrosis in human brain aneurysms.”

    Brain aneurysms, which are bulges in blood vessels in the brain, can go unnoticed for years before rupturing causing a severe, often deadly type of stroke. About one in 50 people in the U.S. has a brain aneurysm but predicting which ones are most dangerous remains challenging. Aneurysms can be repaired surgically or using other minimally invasive procedures but those decisions depend on the size and location of the aneurysm as well as patient specific risk factors. With the current study, “we’ve made major steps toward solving the mystery of how aneurysms form,” said Ethan Winkler, MD, PhD, assistant professor of neurological surgeon and senior author of the Nature Neuroscience study. “We’ve identified the cast of characters involved and seen which ones are implicated at different phases of disease.”

    To get to those answers, Winkler and his team analyzed more than 100,000 individual cells from human aneurysms and healthy brain arteries. From these data, they identified 19 transcriptionally distinct cell types and determined which genes were active in each. They also mapped how the cells were organized within the blood vessel wall.

    “Our atlas of human brain aneurysms, as well as cell-resolution spatial transcriptomics, revealed that pathological cerebrovascular remodeling occurs with the loss of structurally supportive smooth muscle cells and the emergence of activated perivascular fibroblasts, which re-populate the vascular wall and express multiple genes linked to aneurysm risk,” the scientists wrote. 

    Specifically, they found that vessels in aneurysm tissue had disorganized layers, and that many of the smooth muscle cells that allows the vessel walls to expand and contract had disappeared. In their place were scar-forming fibroblasts, which the team dubbed “activated fibroblasts.” These stiffened the arterial wall, making it less able to flex as blood flowed through. These cells also expressed genes that are linked to an inherited risk of aneurysm. The scientists also identified a type of macrophage that accumulated inside the arterial wall near the fibroblasts. The data showed that these specialized macrophages express a gene that is typically associated with bone tissue. 

    Further testing revealed the presence of a feedback look between the two cell types. Specifically, the activated fibroblasts release a signal that triggers the macrophages to produce enzymes that degrade the blood vessel’s structural support. The scientists confirmed that this was the case by blocking the signals sent to the macrophages. They observed that the macrophages were less likely to produce the destructive enzymes when the signal was blocked. 

    This process where vessel walls lose muscle cells followed by the buildup of scar tissue and immune cell activation helps explain why smaller aneurysms, which are often considered low risk, can still rupture. It jibes with Winkler’s own clinical experiences. He noted that more than half of the ruptures that he treated early in his career occurred in aneurysms below the typical surgical threshold of seven millimeters.  

    This study brings scientists and clinicians one step closer to understanding how aneurysms form and perhaps being able to intervene earlier to prevent them. As the scientists note in the paper, “the molecular blueprint provided by this study substantially extends our mechanistic understanding of brain aneurysms and nominates new cells and pathways with translational promise for the development of therapeutic options.” This could involve blocking the signals that fibroblasts send or by inhibiting the immune response to those signals.               

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    ]]>     Origins of First Eukaryotes Linked to Contributions from Multiple Bacteria and Giant Viruses https://edusehat.com/en/origins-of-first-eukaryotes-linked-to-contributions-from-multiple-bacteria-and-giant-viruses https://edusehat.com/en/origins-of-first-eukaryotes-linked-to-contributions-from-multiple-bacteria-and-giant-viruses Thu, 11 Jun 2026 05:35:09 +0700 Edusehat Origins, First, Eukaryotes, Linked, Contributions, from, Multiple, Bacteria, and, Giant, Viruses All cells in animals, plants, fungi, and protists share a fundamental characteristic, in that they are eukaryotic cells. These are essentially complex cells with specialized internal compartments. The cells that make up our bodies are no exception.

    How this type of cell emerged is one of the great questions in biology. For decades, the dominant explanation has placed acquisition of the mitochondrion as the ultimate turning point. It’s thought that an archaeon established a symbiotic relationship with a bacterium, which eventually became the mitochondrion, and this alliance opened the door to cellular complexity.

    A study led by Toni Gabaldón, PhD, an ICREA researcher at IRB Barcelona and the Barcelona Supercomputing Center-Centro Nacional de Supercomputación (BSC-CNS) now rethinks this view. While the research does not deny the central role of the mitochondrion, it suggests that the origin of complex cells was a longer, more gradual and more collaborative process than had previously been thought. Challenging the idea that cellular complexity emerged from a single evolutionary encounter, the study results point instead to a gradual process of interactions among different microorganisms that lasted for millions of years. The findings identify contributions from several bacteria, in addition to the one that gave rise to the mitochondria, and suggest that giant viruses may have acted as vehicles for genetic transfer.

    “For a long time, we have explained the origin of complex cells as a story with two main protagonists: an archaeon and the bacterium that gave rise to the mitochondrion,” said Gabaldón. “Our study suggests that this narrative is incomplete and that there were more actors on stage, including other bacterial groups and giant viruses that may have facilitated gene exchange.” The team published their findings in Nature, in a paper titled “Gene ancestries reveal diverse microbial associations during eukaryogenesis.”

    “The origin of eukaryotes remains a central enigma in biology,” the authors wrote. Unlike studies with dinosaurs, the origin of eukaryotes cannot be reconstructed from visible bones or fossils. It likely occurred about two billion years ago in microscopic organisms, of which barely any direct traces remain. “The current consensus on eukaryogenesis revolves around scenarios that always involve an endosymbiotic relationship with extensive gene transfer between an alphaproteobacterial endosymbiont and a host with an Asgard archaeal ancestry,” the team noted. However, the footprints of this evolution are still present in today’s genomes.

    To trace them, the team approached the problem as a form of computational molecular archaeology, using the computing power of the MareNostrum series of supercomputers to analyze public genomic data spanning biodiversity as a whole.

    The researchers first reconstructed the repertoire of gene and protein families of the last common ancestor of all eukaryotes, known as LECA (last eukaryotic common ancestor). “Our analysis provided a revised reconstruction of the last eukaryotic common ancestor (LECA) proteome, in which we traced the phylogenetic origin of each protein family,” they wrote. The investigators then analyzed its evolutionary origin by comparing these families against databases containing tens of thousands of bacterial, archaeal, and viral genomes.

    “We are trying to reconstruct a story that took place billions of years ago and for which we have no direct fossils. That is why we have been very conservative: we only kept the most robust evolutionary signals—those with a strength comparable to the signals already accepted for the ancestral archaeon and for the bacterium that gave rise to the mitochondrion,” explain study co-authors Moisès Bernabeu, PhD, Saioa Manzano-Morales, PhD, and Marina Marcet-Houben, PhD, who are researchers in the Comparative Genomics group led by Gabaldón at IRB Barcelona and the BSC.

    After more than five years of work using complex mathematical models and processing large volumes of genomic sequences, the team was able to detect signals that would otherwise have remained invisible.

    Beyond the mitochondrion, the study identifies two particularly relevant bacterial signals: Myxococcota and Planctomycetota. The former are related to metabolic functions, including processes linked to lipids and membranes. The latter are bacteria known for their structural complexity, featuring internal compartments that are unusual for bacterial organisms. “Transfers from these donors have been identified in earlier studies, including small-scale detailed ones such as the acquisition of some steroid biosynthesis enzymes from Myxococcota,” the team stated.

    Their analyses indicate that these contributions did not happen all at once. Planctomycetota appear as an older signal, whereas Myxococcota and the bacterium that gave rise to the mitochondrion show signals that are closer in time. “We found compelling evidence for multiple waves of horizontal gene transfer from diverse bacterial donors, with some likely to have preceded mitochondrial endosymbiosis,” the scientists suggested.

    One of the most unexpected findings of the study is that some genes integrated during the early evolution of eukaryotes appear to come from giant viruses, specifically Nucleocytoviricota. These viruses have genomes that are much larger than those of most known viruses, and they infect single-celled eukaryotic organisms.

    The authors propose that these viruses could have acted as vehicles for genetic transfer between microorganisms coexisting in the same ecosystem, facilitating exchanges that helped shape the ancestral genome of eukaryotic cells. “Our results confirm and expand earlier results supporting sizeable gene flow from diverse prokaryotic ancestors preceding the LECA4, and uncover a role for viruses as potential mediators of such transfers,” the scientists stated.

    This vision fits with the idea that the ancestors of eukaryotic cells lived in environments rich in microbial communities, such as microbial mats, where different microorganisms coexist in layers under varying chemical conditions. In this context, genetic exchanges would have allowed them to acquire new biological capabilities over time. “Microorganisms are known to form complex communities such as microbial mats or complex biofilms, of which viruses also form active part, and it is reasonable to consider that the ancestors of the LECA lived in such complex environments,” they stated.

    The study addresses one of the major questions in biology: how the complexity of the cells that form our bodies came to be. By reconstructing the genetic traces of that process, the work provides a new perspective on a key episode in the history of life: the origin of the cellular lineage to which animals, plants, fungi, and protists belong. “Taken together, our results suggest that ancient eukaryotes may have originated within complex microbial ecosystems through a succession of diverse associations that left a footprint of horizontally transferred genes.”

    The paper expands on a line of research initiated by Gabaldón in 2016, when he published a study in Nature that already suggested the mitochondrion might have been acquired relatively late in the process of eukaryotic origins. Now, with much more genomic data available and more powerful computational tools, the team has been able to analyze in greater detail which other organisms left their mark on that common ancestor.

    “All genomes preserve traces of their history. In the case of eukaryotes, those traces tell us of ancient alliances between microorganisms. Understanding them helps us answer a very profound question: what we are and where we come from,” commented Gabaldón.

    The post Origins of First Eukaryotes Linked to Contributions from Multiple Bacteria and Giant Viruses appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     GSK to Acquire Nuvalent for $10.6B, Boosting Cancer Pipeline with Precision NSCLC Treatments https://edusehat.com/en/gsk-to-acquire-nuvalent-for-106b-boosting-cancer-pipeline-with-precision-nsclc-treatments https://edusehat.com/en/gsk-to-acquire-nuvalent-for-106b-boosting-cancer-pipeline-with-precision-nsclc-treatments Thu, 11 Jun 2026 05:35:08 +0700 Edusehat GSK, Acquire, Nuvalent, for, 10.6B, Boosting, Cancer, Pipeline, with, Precision, NSCLC, Treatments GlaxoSmithKline (GSK) has agreed to acquire Nuvalent for $10.6 billion, the companies said, in a deal designed to strengthen the buyer’s cancer pipeline with Nuvalent’s precision oncology treatments—including three non-small cell lung cancer (NSCLC) therapies, of which two are under FDA review with decisions expected later this year.

    Boston-based Nuvalent’s pipeline is headed by the ROS1 inhibitor zidesamtinib (NVL-520) and the ALK inhibitor eladalkib (NVL-655), which according to the company represent potential best-in-class, next-generation, highly selective treatments for NSCLC. Both are brain penetrant. The FDA has set target decision dates of September 18 for zidesamtinib and November 27 for neladalkib, both of which have been granted the agency’s Breakthrough Therapy and Orphan Drug designations.

    Zidesamtinib is designed to treat NSCLC tumors driven by ROS1 that have developed resistance to currently available ROS1 inhibitors, including tumors with the prevalent G2032R “solvent front” resistance mutation. Zidesamtinib is selective in order to minimize CNS adverse events related to off-target inhibition of the tropomyosin receptor kinase (TRK) family, and potentially drive durable responses for patients with ROS1-mutant variants, Nuvalent says.

    Eladalkib was created to address treating tumors driven by ALK that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with both single or compound treatment-emergent ALK mutations such as those involving the G1202R “solvent front” mutation. Eladalkib is also designed to avoiding TRK family inhibition and to treat brain metastases.

    The third NSCLC asset of Nuvalent, NVL-330, is a HER2 inhibitor now under study in Phase I trials for HER2-altered NSCLC. In addition, Nuvalent’s pipeline includes an unspecified number of preclinical programs focused on “addressing the limitations of existing therapies for clinically proven kinase targets in oncology,” the company states on its website.

    “Today’s acquisition is a multi-product deal, consistent with our approach to acquire assets that have clinically proven targets and meaningfully address an efficacy and/or tolerability gap,” GSK CEO Luke Miels said in a statement. “The two lead products are potential best-in-class assets that could launch this year if approved by the FDA and offer significant new treatment options to patients with two forms of non-small cell lung cancer.”

    GSK investors were less enthusiastic as its shares on the London Stock Exchange on Monday dipped 0.5% to 1,903.50 pence. However, Nuvalent shares jumped 39% on Nasdaq to $123.25.

    The $10.6 billion Nuvalent acquisition is the third largest merger-and-acquisition (M&A) deal announced this year, behind the €10.7 billion ($12.355 billion) cash buyout offer for Italian-based Recordati being pursued by CVC Capital Partners and Groupe Bruxelles Lambert, which aim to take the company private; and Sun Pharmaceutical Industries’ planned $11.75 billion purchase of Organon, the women’s health drug developer spun out of Merck & Co., in a deal expected to close in early 2027.

    Immediate sales opportunities

    The Nuvalent candidates, GSK added, present immediate new sales growth opportunities, improving profit contributions from 2027, and a platform in lung cancer for rapid expansion with GSK’s Ris-Rez, a B7-H3 targeted antibody-drug conjugate (ADC) now in Phase III clinical development.

    In a presentation to investors after announcing a series of business updates on May 27, Nuvalent projected an ROS1+ NSCLC treatment could generate ~$1.4 billion to $2.1 billion in peak year sales, with about 40% of those sales (from ~$570 million to $855M million) expected to come from the U.S.—multiples above the ~$150 million in peak year sales attained in 2019 by Xalkori® (crizotinib), marketed by Pfizer and Merck KGaA.

    An ALK+ NSCLC treatment would potentially be even more lucrative, Nuvalent said last month, with projected worldwide peak year sales ranging from ~$3.4 billion to $5 billion, of which the U.S. would account for 40% of sales, or between ~$1.35 billion and $2 billion—well above the $519 million in peak sales attained in 2023 by Alecensa® (alectinib), marketed by Genentech, a member of the Roche Group and created by Roche-owned Chugai Pharmaceutical.

    “Since our founding, we have leveraged our deep expertise in chemistry and structure-based drug design to develop a portfolio of novel, potentially best-in-class kinase inhibitors. Our close collaboration with leading physician-scientists and patient advocates has driven remarkable enrolment, accelerating development and building confidence in the clinical profile of these drugs,” Nuvalent CEO James Porter, PhD, stated. “We’re excited that GSK has recognized the significant value these programs can offer patients and shares our vision for practice-changing innovation.”

    Positive pivotal data

    In announcing the acquisition, GSK cited positive pivotal data Nuvalent presented at the IASLC 2025 World Conference on Lung Cancer and the 2026 ASCO Annual Meeting. Data at both conferences showed potential best-in-class profiles for zidesamtinib and neladalkib, with both treatments designed to deliver longer effective treatment with better quality of life than current therapies, through high target-selectivity, durable treatment response, improved tolerability, enhanced blood-brain barrier penetration for tumor spread, and broader coverage of ALK and ROS1 mutations.

    ROS1- and ALK-altered NSCLC primarily affect non-smoking adults aged 40-50, GSK and Nuvalent said—a patient population the companies described as uniquely defined and engaged.

    GSK said it will commence a tender offer to acquire all of Nuvalent’s outstanding shares of Class A and Class B common stock at a purchase price of $124 per share in cash within 10 business days. The expected purchase price represents a 40% premium to the last closing price and a 26% premium to the 30 calendar day volume-weighted average price.

    Net of cash acquired, GSK estimated its aggregate investment in Nuvalent to be $9.4 billion.

    GSK said the acquisition will not change its 2026 full-year guidance range of 7-9% core operating profit and core EPS growth. The acquisition is expected to contribute to revenue growth from 2027, be incremental to GSK’s existing ambition for sales of >£40 billion (>$53.56 billion) by 2031, and strengthen the company’s core operating profit through the two-year period of loss of exclusivity for its aging blockbuster dolutegravir (2028-2030).

    Dolutegravir is an HIV-1 integrase strand transfer inhibitor (INSTI) marketed as the monotherapy Tivicay® by Viiv Healthcare, in which GSK holds a 78.3% majority stake (and Shionogi, the remaining 21.7% after Pfizer cashed out its 11.7% stake, receiving $1.88 billion). Dolutegravir is also included in Viiv’s fixed-dose HIV combination therapies Dovato (dolutegravir and lamivudine) and Juluca (dolutegravir and rilpivirine).

    Adding to core profit, EPS

    GSK said it expected to add to its core operating profit in 2027 and core earnings per share (EPS) in 2029 by acquiring Nuvalent, even after accounting for cost-cutting synergies and “reprioritization,” which it defines as the shifting of personnel, capital, and other resources away from lower-yield, early-stage research or legacy programs toward higher-value clinical assets and corporate activities. Nuvalent reported 228 full-time employees, of which 144 are engaged in R&D, in its Form 10-K annual report for 2025, filed February 26.

    Should the transaction close in Q3 2026 as expected, GSK said it expects low single-digit percentage dilution to core EPS this year through 2028.

    The company said it will fund the Nuvalent acquisition primarily from new and existing debt facilities plus cash, with no impact expected to its credit rating. GSK ended Q1 with £3.442 billion ($4.608 billion) in cash and cash equivalents, up 1.3% from £3.397 billion ($4.548 billion) at the end of 2025.

    The transaction is subject to customary closing conditions, including the tender of a majority of Nuvalent’s outstanding shares of Class A common stock in the tender offer and the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Act in the U.S. Soon after the closing of the tender offer, GSK expects to acquire any remaining shares of Nuvalent through a second-step merger under Delaware law at the same price per share.

    GSK said it will account for the transaction as a business combination and assume Nuvalent’s existing revenue-sharing arrangements of low-single-digit royalties payable to Royalty Pharma and Deerfield. Royalty Pharma in December acquired for up to $315 million a pre-existing royalty interest in zidesamtinib and neladalkib from an undisclosed third party. Deerfield is Nuvalent’s largest shareholder.

    “GSK’s proven track record, infrastructure, and expertise will support the successful commercialization of zidesamtinib and neladalkib, as well as accelerate advancement of our broader discovery pipeline,” Porter added.

    The post GSK to Acquire Nuvalent for $10.6B, Boosting Cancer Pipeline with Precision NSCLC Treatments appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO 2026: Partnering meetings fill up for industry’s biggest gathering https://edusehat.com/en/bio-2026-partnering-meetings-fill-up-for-industrys-biggest-gathering https://edusehat.com/en/bio-2026-partnering-meetings-fill-up-for-industrys-biggest-gathering Thu, 11 Jun 2026 02:00:04 +0700 Edusehat BIO, 2026:, Partnering, meetings, fill, for, industry’s, biggest, gathering The last time the BIO International Convention was in San Diego, in 2024, Ram May-Ron of the non-dilutive funding specialists FreeMind Group began discussing a strategic partnership with a large pharma company. Discussion continued at BIO 2025 in Boston, where they signed a pre-agreement term sheet.

    “BIO is just an amazing opportunity to meet so many investors, partners, clients,” May-Ron said. “Everyone in the same building; you get to be very, very efficient.”

    This year, the BIO International Convention brings the world’s largest gathering of the biotechnology industry back to San Diego during June 22-25, for expert panels, company presentations—and a lot of fruitful networking.

    “We have 35,000 scheduled meetings as of June 8. We’re expecting to end with a record of 70,000 or above,” said Mackensie Vernetti, SVP of Partnering at the Biotechnology Innovation Organization (BIO).

    Whether they’re looking for investment, licensing deals, manufacturers, or partners for development, participants in the BIO International Convention can register their interests ahead of time in the BIO Partnering™ system.

    By leveraging the system, partners with matching needs can find one another and set up meetings before arriving. The BIO Partnering™ system will arrange a time that works for both partners and reserve a meeting place from among the 2,000 meeting rooms and booths set up at the San Diego convention site.

    With meetings filling up at a rapid clip, Vernetti recommended BIO 2026 participants start engaging with the BIO Partnering™ platform right away.

    “A lot of people want to have all of their meetings confirmed by the time they arrive,” she explained. “Of course we continue to schedule meetings up through the event so we can accommodate any last-minute requests.”

    Finding start-ups and investors

    One reason participants might want to keep a few meeting slots open is the Start-up Stadium, where representatives of exciting new small firms from around the world pitch their companies on stage for the benefit of potential partners.

    “This year we have our most international group ever, with a delegation from the European Investment Council as well as companies presenting from Taiwan for a total of 50 presentations,” said Bernard Fallon, BIO VP of Industry Programs. “All of these companies are in our partnering system, and our attendees have an opportunity to meet these new start-ups early.”

    In an investment market that can be challenging, the BIO International Convention provides an important opportunity, Fallon said.

    “The BIO International Convention brings together the largest biotechnology community under one roof,” he explained. “This is the opportunity for a company to meet a greater variety of partners than they could meet in any other one place. And investors can get a very efficient view of the state of innovation in any given therapeutic area.”

    The BIO International Convention also provides a constant for the industry, according to Vernetti.

    “The market and investment climate go up and down, but year over year, we continue to have consistent partnering meeting activity—breaking or getting close to records in attendance and partnering,” she said. “We bring the whole biotech ecosystem together, so whether it’s straightforward investment or licensing deals, or other collaborations for research and co-development to advance your program, people are finding that at the BIO International Convention.”

    Even if participants can’t make meetings for their specific partnering goals, Vernetti recommended they take advantage of the contacts they can make through the platform.

    “Even if they’re not able to meet on site, a lot of the value is in the database and being able to reach out to those people. It’s still worthwhile to send meeting requests to at least exchange contact information to meet later on.”

    As May-Ron of FreeMind Group can attest, a connection made at one BIO International Convention can pay off later—perhaps even at the next year’s convention.

    The post BIO 2026: Partnering meetings fill up for industry’s biggest gathering appeared first on Bio.News.

    ]]>     Innovative Tech Testing in Response to GMP Revisions https://edusehat.com/en/innovative-tech-testing-in-response-to-gmp-revisions https://edusehat.com/en/innovative-tech-testing-in-response-to-gmp-revisions Thu, 11 Jun 2026 01:55:20 +0700 Edusehat Innovative, Tech, Testing, Response, GMP, Revisions Recent revisions to EU manufacturing guidelines are changing how drug makers test processing technologies, according to the author of a new study, who cites growing use of an approach known as PUPSIT as an example.

    PUPSIT, or pre-use post-sterilization integrity testing, is used to verify the integrity of sterilizing-grade filters after they have been sterilized, but before they have been used in a biopharmaceutical manufacturing process.

    The idea is to make sure the filter has not been compromised during handling or sterilization and is still capable of retaining microorganisms, according to lead author Martin Glanz, Dr. rer. nat., senior principal scientist at Cytiva.

    “Operationally, this typically involves wetting the membrane, venting the system, and then carrying out an integrity test such as a bubble point or forward-flow test. These methods essentially measure gas flow through the wetted membrane and confirm whether the filter meets its defined specifications,” he tells GEN.

    Conventional verification processes focus on testing filters after they have been used, which, Glanz says, means that faults can be missed.

    “The main benefit of PUPSIT compared to older approaches, which often relied heavily on post-use testing, is that you detect any potential defects before product exposure.

    “That’s quite important, because defects can sometimes be masked during filtration— for example, due to fouling or plugging—and might not show up afterward. PUPSIT helps close that gap and strengthens overall sterility assurance,” he says.

    The biopharmaceutical industry’s use of PUPSIT has increased since 2022, when EU GMP Annex 1, which covers the manufacture of sterile drugs, was revised to include stricter environmental monitoring and quality control requirements.

    Glanz adds, “Even though PUPSIT isn’t always an absolute requirement, it is generally expected unless there is a well-justified, risk-based rationale not to perform it. Through PIC/S, this expectation is spreading beyond Europe as well.”

    Adoption challenges

    Switching from post-use verification strategies has significant potential benefits. However, implementing the approach can be challenging because, compared with traditional approaches, PUPSIT requires some additional steps, Glanz says.

    “Companies often run into issues such as increased process complexity: you’re adding steps like wetting, venting, and testing that need to be controlled carefully.

    “Additional connections can also introduce contamination risks, especially downstream of the sterilizing filter. In more manual setups, operator dependency becomes a real factor, and achieving reliable wetting can be trickier than it sounds.

    “On top of that,” he continues, “more complex assemblies come with typical engineering challenges: dead legs, hold-up volumes, or simply designs that are harder to keep compliant. So, while the regulatory acceptance is clearly there, many organizations are still refining how to implement PUPSIT in a robust and efficient way.”

    And technology—specifically single-use systems—is key to this refining process.

    “There’s a clear trend toward single-use, preassembled flow paths, which help reduce handling and variability. At the same time, integrity testing technologies are evolving, both in terms of sensitivity and integration.

    “Solutions that can assess not just the filter, but the system as a whole, are becoming increasingly relevant. Ultimately, it comes down to combining good engineering with reliable, well-validated procedures,” Glanz says.

    Future

    The emergence of automated testing systems is also likely to increase biopharma industry use of PUPSIT, according to Glanz.

    “The benefits are quite tangible: automated wetting, venting, and testing steps; tighter control over process parameters; fewer manual interventions, particularly on the sterile side; and improved repeatability.

    “Automation also enables better documentation, with electronic records and audit trails integrated directly into the system. In a way, this shifts the focus from operator execution to system design and validation, which aligns well with current regulatory thinking,” he says.

    The post Innovative Tech Testing in Response to GMP Revisions appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Updated Amplification Tool Rapidly Detects Mycoplasma https://edusehat.com/en/updated-amplification-tool-rapidly-detects-mycoplasma https://edusehat.com/en/updated-amplification-tool-rapidly-detects-mycoplasma Thu, 11 Jun 2026 01:55:19 +0700 Edusehat Updated, Amplification, Tool, Rapidly, Detects, Mycoplasma Nucleic acid amplification techniques (NAAT) are recommended by pharmacopoeias in the United States, the EU, and Japan as an alternative to traditional culture and indicator cell methods used to detect Mycoplasma in biological products. Used for in-process controls and release testing, NAAT is an improvement over traditional methods that take up to 28 days, but attempts to balance broad species coverage and sensitivity favor one over the other.

    Recently, Chinese researchers developed a NAAT that overcomes those challenges, according to a recent paper by scientists at the National Institute for Food and Drug Control, Yeasen Biotechnology, and Xi’an Jiaotong-Liverpool University.

    Rather than surpassing existing technologies, “It optimizes the core pain points of mainstream multiplex NAATs,” encompassing their advantages and forming others, senior author Xiaoliang Sun, PhD, scientist, genomics division, Yeasen Biotechnology, tells GEN.

    For example, for cell therapy products, Sun says this NAAT technique, “cuts Mycoplasma tests from a 28-day culture to several hours, enabling same-day batch release and avoiding cell product expiry scrap.” It also “accelerates finished-product release for short shelf-life recombinant proteins and monoclonal antibodies.”

    The three pairs of primer-probe sets in this assay cover 183 Mollicutes species by targeting Mycoplasma-specific conserved regions. “This fills the detection gap [experienced by] some rare strains…and makes it suitable for trace contamination screening in biopharmaceutical production,” Sun points out.

    To objectively assess this method, they compared it to traditional 165 rRNA degenerate PCR, strain-specific NAATs, and mainstream multiplex NAATs cited in the literature.

    The analysis shows the new assay offers “single-copy detection sensitivity (validated by 10 pharmacopoeia standard strains), no cross-reactivity (validated with 14 non-Mycoplasma genera and six engineered cell lines), short amplicons of 100 to 200 bp, amplification efficiency of 95–105%, and excellent repeatability,” Sun says. This ensures specificity and improves detection consistency. Consequently, the method “meets the strain detection requirement of Chinese and European pharmacopoeia for full-process regulatory scenarios.

    “The detection process is compatible with existing qPCR platforms without requiring special equipment and can be directly applied to full-process scenarios, such as raw material screening, cell bank verification, and finished product release,” Sun continues, “achieving more comprehensive compliance.”

    There are limitations, though. “Detection performance for unrecorded Mycoplasma strains from extreme environments or highly variable subspecies has not been verified… leaving a potential detection gap,” he cautions. Additionally, “The use of three pairs of primer-probe sets increases the reagent cost per sample.” Therefore, scientists may best use this method for initial screening, followed by digital PCR confirmation.

    For biopharmaceutical manufacturers, Sun says this assay’s main advantages are “comprehensiveness, stability, and practicability.” Future work is envisioned to expand the assay’s applicability parameters and robustness.

    The post Updated Amplification Tool Rapidly Detects Mycoplasma appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Gorilla Adenovirus Brings Natural Edge to Cancer Therapy https://edusehat.com/en/gorilla-adenovirus-brings-natural-edge-to-cancer-therapy https://edusehat.com/en/gorilla-adenovirus-brings-natural-edge-to-cancer-therapy Thu, 11 Jun 2026 01:55:18 +0700 Edusehat Gorilla, Adenovirus, Brings, Natural, Edge, Cancer, Therapy When ReiThera’s scientists first turned to a gorilla-derived adenovirus, they were thinking about vaccines. What they found instead might reshape how the field thinks about virus-based therapy for cancer.

    Angelo Raggioli, PhD, head of technology development at ReiThera, describes a platform built on a gorilla adenovirus that was discovered, not designed. He adds: “Several therapeutically relevant properties appear to be embedded in the native biology of the vector itself.” For example, the group C gorilla adenovirus showed low seroprevalence in humans, reduced liver sequestration after systemic delivery in mouse models, a natural tendency to go to the lungs, and intrinsic replication selectivity in human cancer cells, sparing non-cancerous cells.

    That combination addresses some of the most persistent obstacles in virus-based therapy. Pre-existing immunity against common vectors can blunt therapeutic efficacy before treatment begins, while off-target organ uptake—particularly hepatic sequestration of adenoviral vectors—remains a fundamental challenge for intravenous administration. A gorilla-derived isolate sidesteps both problems: it is naturally distant from human adenoviruses (avoiding the impact of pre-existing immunity shaped by prior exposure), and serendipitously avoids liver sequestration.

    The biodistribution profile is equally important. Reduced liver targeting after systemic delivery, plus an attraction to the lungs, positions the platform not only for oncology applications but potentially for pulmonary gene therapy.

    Raggioli adds that adenoviral vectors offer cargo capacities of about 36 kilobases, which is a significant advantage over the roughly 4.5-kilobase ceiling of adeno-associated viruses. For diseases requiring delivery of large transgenes, that capacity difference could be clinically decisive.

    In oncology settings, the vector has demonstrated selective replication in tumor cells while sparing normal tissue, which was a property the ReiThera team observed rather than engineered. “While much of the field is actively engineering vectors to retarget specific tissues, in this case, we started from the natural tropism of the virus and began exploring how to leverage those native biological properties therapeutically,” Raggioli explains.

    The platform has also been armed with therapeutic payloads. As a proof of concept, the team encoded a single-chain anti-HER3 antibody directly into the viral genome, achieving selective expression in replication-permissive tumor cells. This positions the platform within a broader trend in oncolytic virology: viruses are increasingly expected to serve not merely as cytolytic agents but as localized delivery systems for antibodies, immune modulators, and other complex biologics.

    That evolution reflects a shifting understanding of how oncolytic viruses actually work. Tumor cell lysis alone is no longer considered sufficient; the immunological consequences of that lysis—whether it triggers a productive antitumor immune response—are central to therapeutic activity. Engineering for that immunogenic conversion while preserving replication potency and tumor specificity represents one of the field’s most demanding design challenges.

    “An additional strength of the platform is that, for vaccine and oncolytic applications, we can manage the entire process internally, from genome engineering to clinical-grade manufacturing,” Raggioli says.

    What began as a search for a better vaccine backbone has yielded something potentially more versatile: a vector whose biology may be doing therapeutic work that other platforms have to build in from scratch.

    The post Gorilla Adenovirus Brings Natural Edge to Cancer Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     In Silico Devices May Improve Drug Manufacturability https://edusehat.com/en/in-silico-devices-may-improve-drug-manufacturability https://edusehat.com/en/in-silico-devices-may-improve-drug-manufacturability Thu, 11 Jun 2026 01:55:17 +0700 Edusehat Silico, Devices, May, Improve, Drug, Manufacturability Using in silico tools to augment physical experiments can help identify manufacturability issues early in development. That’s according to an AI technology company that spoke on a recent panel.

    BigHat Biosciences, which was among the companies presenting at PEGS Boston, explained that developing in silico models of antibody yields using a cell-free expression system allows the exploration of a wider range of mutations.

    And this, in turn, lets companies better optimize their product for manufacturability.

    “There’s only so many experiments you can do by putting an antibody into CHO [Chinese Hamster Ovary] cells,” explains Hunter Elliott, PhD, vice president of machine learning at BigHat Biosciences.

    “With in silico tools augmenting that exploration side, we can build models that make predictions for improved sequences, screening many more antibodies in silico than we need to send to the lab.”

    Elliott’s talk came alongside a panelist who argued it was important for preclinical researchers to communicate with the manufacturing departments of their companies to make a success of the latest generation of harder-to-manufacture drugs.

    Speaking about his own products, Elliott argues that using in silico tools to explore a wider range of possible antibody mutations means it’s possible to select the handful with the highest possible yields as well as other improved biophysical properties.

    “You’re derisking your processes because you’re combining your experiments with the in silico tools you’re using,” he says.

    Talking about the panel, Elliott adds that discussion around the limitations of models included the lack of publicly available data on manufacturability and developability, especially for potential products that have failed to make it to the clinic.

    Some have expressed concern that using in silico tools might also accidentally screen out the best-performing antibodies. Although, he says, “my personal opinion is that the ability to predict properties of sequences without sending them into the lab makes it easier for us to optimize from a suboptimal starting sequence.”

    “With these models, we can keep this imperfect antibody in the loop and take it forward through several rounds of optimization, and then, instead of a candidate molecule being killed early on, it might be engineered into manufacturability.”

    The post In Silico Devices May Improve Drug Manufacturability appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Symeres Expands Spray Drying Capabilities at Its U.S. New Jersey Site https://edusehat.com/en/symeres-expands-spray-drying-capabilities-at-its-us-new-jersey-site https://edusehat.com/en/symeres-expands-spray-drying-capabilities-at-its-us-new-jersey-site Thu, 11 Jun 2026 01:55:15 +0700 Edusehat Symeres, Expands, Spray, Drying, Capabilities, Its, U.S., New, Jersey, Site Netherlands CRDMO Symeres expanded its spray drying capabilities at its Cranbury, NJ CMC development site to support formulation development for poorly soluble and development-challenged small-molecule drug candidates.

    The expanded capability is designed to support bioavailability enhancement strategies, including amorphous solid dispersions (ASDs), particle engineering and solubility optimization for compounds progressing from preclinical development through Phase II clinical activities.

    The investment strengthens the company’s integrated CMC offering by combining spray drying, formulation sciences, analytical characterization, solid-state sciences, and process development within a single development environment, according to Henning Steinhagen, CEO of Symeres. This enables sponsors to progress from early formulation screening through to clinical-ready material within one coordinated scientific framework, reducing tech-transfer risk, accelerating decision-making, and improving development continuity, he adds.

    By expanding spray drying capabilities within its integrated Cranbury CMC site, Symeres says it can help clients address developability challenges earlier, reduce operational complexity, and support faster progression into clinical development. [Symeres]
    By expanding spray drying capabilities within its integrated Cranbury CMC site, Symeres says it can help clients address developability challenges earlier, reduce operational complexity, and support faster progression into clinical development. [Symeres]
    “Our investment in spray drying reflects our commitment to supporting customers across the entire drug development journey,” continues Steinhagen. “While Symeres is widely recognized for its discovery expertise, this expanded capability further strengthens our ability to support complex molecules through development and into the clinic.”

    “An increasing proportion of modern small molecule drug candidates require advanced formulation approaches to achieve acceptable bioavailability and clinical performance,” explains Paul O’Shea, managing director at Exemplify BioPharma, a Symeres company. “By expanding our spray drying capabilities within our integrated Cranbury CMC site, we can help clients address developability challenges earlier, reduce operational complexity, and support faster progression into clinical development.”

    A Symeres spokesperson, who says the Cranbury site now supports laboratory-scale and pilot-scale spray drying workflows for a range of formulation development activities, including rapid material screening, process optimization and scalable process development, notes that the platform is particularly suited to Biopharmaceutical Classification System (BCS) Class II and IV compounds, highly lipophilic molecules and targeted therapies requiring enhanced oral exposure.

    The post Symeres Expands Spray Drying Capabilities at Its U.S. New Jersey Site appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     The “steroid olympics” were a circus—and a window into our culture https://edusehat.com/en/the-steroid-olympics-were-a-circusand-a-window-into-our-culture https://edusehat.com/en/the-steroid-olympics-were-a-circusand-a-window-into-our-culture Wed, 10 Jun 2026 22:15:03 +0700 Edusehat The, “steroid, olympics”, were, circus—and, window, into, our, culture Testosterone. Methenolone. Nandrolone. Human growth hormone and EPO. Meldonium, modafinil, and mixed amphetamine salts. Clomiphene, anastrozole, levothyroxine, and liothyronine. Patches and capsules, creams and pills. A whole galaxy of steroids, metabolic modulators, and synthetic hormones coursing through the blood of a few dozen swimmers, sprinters, and weightlifters. And millions of dollars up for grabs for athletes who could break world records and usher in the age of superhumanity.

    On Sunday, May 24, at a $50 million arena built in a casino parking lot in Las Vegas, I witnessed a libertarian thought experiment come to life. The inaugural Enhanced Games were the first sporting competition where participants were encouraged to take performance-enhancing drugs. The founders say they’re challenging dated sporting norms and helping to build a world where we can all live better, longer lives. Critics say the event is an embarrassment, that it glamorizes the use of dangerous substances and puts lives at risk. 

    The open-air venue was compact and decked out in bright blue, with a six-lane, 100-meter track down one side, a four-lane Olympic-length swimming pool down the other, and a weightlifting platform and stage at the front. You could see the golden façade of the Trump Hotel looming in the background. The scene had all the trappings of an NFL game, with the too-loud music and crowd work on the big screen—a “flex cam”  gave the well-muscled an excuse to unveil their biceps. Between events, adverts flashed up for the line of performance products sold by Enhanced, the company behind the event: injectable peptides that supposedly support cellular energy and skin elasticity, daily supplement powders with names like “Stronger” and “Longer.”

    James Magnussen, wearing his race goggles and cap, stretches his arms above him
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    the Enhanced Games stadium from an elevated viewing angle
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    Australian swimmer James Magnussen was the first athlete to sign up with Enhanced but hasn’t broken any world records. He finished last in his two events in Las Vegas.

    The day started with the weightlifters, under the blazing sun. But by 4 p.m., only one of them had even attempted a world-record lift. Two had pulled out injured. Some athletes were competing without taking drugs because of the money on offer, and as the competition went on, they had the better of their enhanced peers: Hunter Amstrong, a 25-year-old American swimmer and triple Olympic medalist, won the backstroke by more than a second. In the men’s 100-meter sprint, the non-enhanced US athlete Fred Kerley romped to an easy victory. “Man, they gotta do better than that,” he said of his doped opponents in his post-race interview. “They need to train a little harder, get on that shit a little bit more.”

    At the bar, bodybuilders swapped before-and-after pictures and talked about their stacks, and VCs and finance bros traded LinkedIn details. Lukas Lakutsin, a 6-foot-10, 354-pound Russian bodybuilder who was milling around the entrance to the VIP suites, initially told me he didn’t use any performance-enhancing drugs. Except testosterone replacement therapy, of course. But he didn’t think that really counted. “I’m almost 34 years old,” he said. “I need to do this to stay strong.”

    close up shot of a man's muscled chest
    The “protocol” for Enhanced athletes only includes FDA-approved drugs. While Enhanced’s team might make recommendations, individuals have the final say on what they want to take, if anything.
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    Jeremy Sigal, an influencer and author, wore a USA tank top that showed off hugely muscled arms adorned with prison tattoos. He told me he was proudly natural, in both his health and his personal life. “I’ve got an exceptional credit score,” he said. He has written 12 books on marketing and leadership. Later, I looked up his most recent book online. It’s called Simp to Pimp: 10 Steps to Fix Why She’s Not Banging You and lists AI as a coauthor.

    What I saw in Las Vegas probably wasn’t the future of sport. But it was a perfect encapsulation of our present moment, as Silicon Valley biohackers, alt-right looksmaxxers, Make America Healthy Again boosters, and longevity-obsessed scientists all vie to remake reality in their own image. For them, the Enhanced Games offered a glimpse of a future where medical advances push the human race to new heights, and where they never have to get old. 

    I’ve tracked Enhanced’s journey from a crazy idea scribbled on a napkin to a public company valued at $1.2 billion. Behind the scenes, there have been power struggles, life-changing victories, and moments of total farce. As I recently, finally, watched the games unfold, two questions bounced around my head: Were they right? And what does that mean for the rest of us?

    In December 2022, the Australian entrepreneur Aron D’Souza flew to Miami to spend New Year’s Eve with his friend and mentor Peter Thiel. A decade earlier, D’Souza had helped Thiel orchestrate the lawsuit that bankrupted Gawker—a stunning revenge against the gossipy New York media blog that had outed him as gay. Now he was armed with a disruptive idea that he thought Thiel, the billionaire cofounder of PayPal and Palantir, would love. It was inspired by the buff bodies he’d been seeing at the gym, highlighting a disconnect between a workout culture where the use of steroids was an open secret and a sporting establishment where it was, at least on paper, an inviolable taboo.

    His initial pitch was provocative and confrontational: a grand sporting event to rival the Olympic Games, where competitors could take any substance they wanted—their body, their choice. The first time I met D’Souza, in the spring of 2024, he had founded the company and attracted some initial investment but seemed obsessed with taking on the fat cats at the International Olympic Committee and reinventing sports (even though he didn’t seem to be a huge sports fan himself). On Enhanced’s Discord server, I found a folder full of memes with names like IOC Clowns.jpg. The whole thing felt very unserious.

    That would change. 

    D’Souza told me that Thiel had previously introduced him to Christian Angermayer, a German biotech billionaire, who would come onboard at Enhanced. He’s funded clinical trials of psychedelics through his company Atai Life Sciences and is helping bring them into the medical mainstream as a treatment for depression and anxiety. Angermayer says he spotted an opportunity to do the same thing for steroids. What he really wants is to redefine medicine, he told me. Its focus has already changed from treating disease to trying to prevent it; actively enhancing people’s health, he says, is just the next logical step.

    By early 2024, Angermayer had brought his own people into key roles. The team included Michael Sagner, an anti-aging expert and private doctor who works with many of Hollywood’s leading men, and Max Martin, who has the jawline and cheekbones of an Instagram looksmaxxing influencer and the boundless enthusiasm of a puppy. (He started his own enhancement program a few years ago, when he was just 27.) Sagner would head up Enhanced’s medical commission, making sure the games were safe for the athletes. It was Martin’s job to make sure they actually happened. 

    a group of men in business suits posing at the desk of the NYSE
    In early May, Enhanced began trading on the New York Stock Exchange with an initial value of $1.2 billion. Christian Angermayer stands far right with Max Martin to his left (front row), and Aron D’Souza next to him.
    LEV RADIN/ZUMA PRESS WIRE VIA ALAMY

    Tensions sparked as D’Souza’s freewheeling style clashed with the more sensible image that Sagner and others were now keen to present. “It was not just his personality and his abrasive way of talking,” Sagner told me recently. “Even when he was briefed on a scientific fact, he would just completely ignore it and say something outrageous.”

    But the more outrageous D’Souza got, the more attention his idea received. In February 2024, James Magnussen, a retired Australian swimmer, became the organization’s first official athlete, and Enhanced promised to pay a million dollars to him, or anyone else, who could break the world record in the 50-meter freestyle.

    The notion of a “steroid olympics,” as many have dubbed the Enhanced Games, had been kicking around for decades—for instance, in a Wired article from the early 2000s and an SNL sketch from the 1980s. Two things helped finally make the Enhanced Games a reality. First, in November 2024, Donald Trump was again elected president of the United States. The Biden administration had been actively hostile to the games, but the founders saw a more receptive political environment in Trump world. Not long after the election, Enhanced announced a new tranche of funding led by 1789 Capital, a venture capital firm whose partners include Donald Trump Jr.

    And second, in February 2025, an enhanced swimmer finished the 50-meter freestyle faster than anyone in human history. It wasn’t Magnussen, though. He had been injecting himself with testosterone to grow muscle, plus a cocktail of peptides that aimed to speed up recovery—but his journey hadn’t quite worked the way he’d planned.

    A combination of reputational issues (no pools wanted to host his training) and physical complications (the regimen did help him get stronger, but he packed on so much muscle that it slowed him down in the water) meant he watched from the sidelines as the Bulgarian-Greek swimmer Kristian Gkolomeev—who had finished fifth at the Paris Olympics in 2024—came in two-hundredths of a second under the record and won a million-dollar payout from Enhanced. The idea has always been that breaking records would effectively prove the legitimacy of this enhancement project: Look what we can do now. 

    Over the shoulders of Shane Ryan (left) and James Magnussen (right) as they sit and talk poolside
    Enhanced swimmers like Magnussen (right) wore supersuits to compete, though they’ve been banned by World Aquatics since 2010.
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    Gkolomeev, though, had a different motivation for participating: “One successful year in the Enhanced Games and I could make as much as I would in almost 10 careers,” he told me not long after setting the new record (notably, wearing a kind of “supersuit” that’s been banned by World Aquatics since 2010). Enhanced was paying its athletes a regular salary, on top of any potential bonus. And he had a young family to support and feared that the four-year stretch to the next Olympics would be long and precarious. 

    In May 2025, with a world record in the bag and a friendly administration in the White House, Enhanced was ready to announce its first games: They’d take place in May 2026 at Resorts World in Las Vegas. 

    At the same time, D’Souza made another big reveal: Enhanced Performance Products, a line of supplements available for a monthly subscription. The Enhanced Games now seemed less like a sporting event and more like a loss leader for selling testosterone injections, GLP-1s, or a range of peptides that are claimed, with little scientific evidence, to improve sleep or skin elasticity. Perhaps it was all a brilliantly executed marketing stunt.

    “The games themselves now seem almost secondary to what appears to be an online marketplace for hormones, peptides, and other performance-enhancing compounds,” says Astrid Kristine Bjørnebekk, a steroids expert at Oslo University Hospital. “From my perspective, this significantly changes the nature of the project. It is one thing to organize a closed sporting event built around controversial principles, but openly marketing and commercializing substances such as testosterone, hGH, GLP-1 drugs, peptides, and other pharmacological compounds is something else entirely.”

    As the games approached, more athletes joined. Some were genuinely elite. The US sprinter Kerley—who is serving a two-year ban for missing three drug tests—had won silver in the 100 meters in the Tokyo Olympics and a bronze in Paris. Ben Proud, a British swimmer, had won silver at the Paris Olympics and dozens of medals at world and European championships and the Commonwealth Games. He had been mulling over joining the Enhanced Games ever since the idea first emerged, but the tipping point seemed to come when Gkolomeev’s record was announced. 

    Some participants, like Magnussen and another swimmer, Megan Romano, had been tempted out of retirement. Romano hadn’t swum competitively for almost a decade. Others were at the start of their careers but ready to cash in their chips and bid goodbye to Olympic dreams for a potential six-figure payday. The $1 million payouts were reserved for records in the two flagship events—the 50-meter freestyle and the 100-meter sprint—but winning any other event would mean a prize of $250,000, with an additional $250,000 bonus for setting a world record.

    Athletes would get paid even if they just showed up and finished last—as much as $50,000. This is all on top of the salaries that stretched into six figures in some cases, making the payout from the games more than many athletes make in a year.

    Sport’s governing bodies reacted to each new athlete announcement with fury. World Aquatics threatened to ban for life any athlete who participated in the games, even if they didn’t take any drugs. Enhanced responded with an $800 million antitrust lawsuit against the global swimming organization, the World Anti-Doping Agency, and USA Swimming, alleging misuse of monopoly power.

    Emmanuel Matadi (left) and Fred Kerley (right) running on the track
    American Fred Kerley (right) won the 100-meter sprint without performance enhancing drugs.
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    In November 2025, a court in New York dismissed the case. Three days later, D’Souza, the mind behind the entire project, was out. A notice on Enhanced’s website said he had “transitioned out of the company’s day-to-day operations.” Martin would take over as CEO. “The investors basically said we need someone a bit more serious,” Sagner told me. In conversations, execs at Enhanced played down any suggestion of a feud—D’Souza was simply the ideas man, with little interest in the day-to-day dreariness of actually running a company. (Enhanced spokesperson Chris Jones wrote in a statement that “there is no tension between Aron and Enhanced that I’m aware of.” D’Souza did not respond to a request for comment.)

    I got the sense that Enhanced, in its new iteration as a pharmaceutical subscription company, was almost embarrassed by the games. When I visited enhanced.com a couple of months before the event, they had been relegated to a sub-heading on the home page. D’Souza’s showmanship had helped get attention for what was becoming a run-of-the-mill telehealth business like Hims & Hers—albeit one well timed to take advantage of a shifting regulatory landscape around peptides, which Robert F. Kennedy Jr., the US secretary of health and human services, has been pushing the FDA to approve despite a lack of evidence that they’re actually effective. 

    Sagner is still loosely involved with Enhanced, but he says the medical commission was not consulted before it launched its line of performance products. (Jones did not respond to a question regarding this claim.) Sagner is scathing about what he sees as the “hype” around peptides. “I can tell you already, peptides do nothing,” he says—with the exception of human growth hormone and GLP-1. “The peptides that people use, black-market peptides that they buy online—they do nothing. We have tested them; 80% of them contain nothing. It’s saline solution, salt water, and some of them are contaminated.”

    At the end of January 2026, a group of around 40 swimmers, weightlifters, and sprinters arrived in Abu Dhabi to start their individualized enhancement “protocol,” as Enhanced calls it. Officially, they would be taking part in a clinical trial, pending approval by the Abu Dhabi government and overseen by Guido Pieles, a Qatar-based cardiologist who has taken over the reins of Enhanced’s medical commission from Sagner.

    Canadian weightlifter Boady Santavy strains to lift a barbell which is currently level with his hips
    The day started with the weightlifters, but by late afternoon, only one of them had even attempted a world-record lift.
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    They would be allowed to choose only from a menu  of specific FDA-approved drugs. Pieles broke them down into five categories: testosterone variants and growth hormones, which can both boost muscle mass; metabolic modulators that can tweak how the body burns fat; stimulants like Adderall to improve focus; and EPO, which can increase the amount of oxygen the blood is able to carry. While Enhanced’s team might recommend particular things, the athletes would have the final say on what they wanted to take, if anything. (As Oslo University’s Bjørnebekk points out, FDA approval “does not mean the substances are inherently safe, particularly not when used for enhancement purposes.”) 

    There would be regular blood tests, heart scans, and brain scans and access to the best training facilities money could buy. Pieles and others say the clinical trial will help inform the line of supplements Enhanced is offering consumers, but there’s actually very little overlap between the drugs the athletes were taking and the substances the company is currently selling.  

    Not long after they arrived in the Middle East, the athletes were awakened by the sound of explosions at a military base near their hotel. The US and Israel had struck Iran, and the Iranian regime was responding by peppering the region with missiles. “It wasn’t a pleasant situation,” says Andrii Govorov, the world record holder in the 50-meter butterfly, who a year earlier had become one of the first swimmers to join Enhanced. Govorov had some experience in these matters—back in Ukraine, he’d had a business selling cars that helped fund his swimming career, but he’d lost it after the Russian invasion.

    Cody Miller standing by the pool in profile
    SAEED RAHBARAN
    attendees sitting in the bleachers
    SAEED RAHBARAN
    close-up of the calloused and powdered hands of a weightlifter with remnants and marks left by the tape.
    SAEED RAHBARAN

    Swimming, sprinting, and weightlifting were the focus of the first Enhanced Games but in many ways the sports were the sideshow.

    The conflict exacerbated delays in getting approval for the clinical trial and sourcing the drugs, and as a result, what was supposed to be a 12-week enhancement protocol got cut down to eight weeks. The athletes didn’t actually start taking the drugs until toward the end of March. For those who had always been clean, that represented the irreversible crossing of a line. “The first injection was very emotional, very tricky to navigate,” says Proud. “For me, that was the day I went from the Ben Proud that I always knew to a new person.”

    Proud was joined in the enhancement program by his girlfriend, Emily Barclay, who had swum at college level without ever appearing at a major international event; she was working as a swimming teacher at a school in England. After that first injection, they left Abu Dhabi and spent a few days in Dubai as they reckoned with what they had done. “I just couldn’t be around the team,” Proud says. “I wanted to be by myself and feel those feelings, because it is a big deal to make that step, and I felt it.”

    Those feelings were soon forgotten, though, as the drugs kicked in. Proud says he had incredible energy, and a drive to train that he hadn’t experienced before. Shania Collins, an American sprinter, says she had “increased strength, increased recovery, and increased mental clarity at practice.” Sagner and several athletes admitted there were some side effects: acne and some swelling around the joints; unwanted hair growth for the women, unwanted hair loss for the men.

    close-up of runner Tristan Evelyn in profile
    Like Kerley, sprinter Tristan Evelyn from Barbados competed without taking any drugs. She too won big in Vegas, besting her Enhanced peers in two events.
    SAEED RAHBARAN

    One thing the athletes wouldn’t talk about, though, is what drugs they were actually taking. They all had the same reason: not wanting to encourage copycats who might take enhancements without a doctor on hand to tailor programs to their needs. 

    The one exception was Thor Björnsson (testosterone, deca-durabolin, anastrozole, halotestin), a hulking Icelandic deadlifter and former World’s Strongest Man who played The Mountain on Game of Thrones. Björnsson first heard about the games on Joe Rogan’s podcast and was immediately interested. The rules for strongman competitions are somewhat less stringent than those for Olympic sports, though, and he actually had to reduce the number of substances he was taking to meet Enhanced’s FDA requirements.

    Hafþór Júlíus Björnsson holding a barbell at mid thigh
    Icelandic strongman Thor Björnsson actually had to reduce the number of substances he was taking to meet Enhanced’s FDA requirements.
    SAEED RAHBARAN

    There is some debate over how much doping some of the athletes were actually doing. In a conversation last year, Gkolomeev told me he’d only really been “microdosing,” and he confirmed that his 2026 enhancement program was largely the same. Sagner says the doses the athletes were taking were a fraction of the amounts some Olympic athletes had been caught using in the past. I heard that a few athletes had decided not to take steroids or growth hormones and were only using modafinil, a narcolepsy medication that’s thought to improve focus.

    The day before the games, I asked Angermayer what it would mean if clean athletes like Kerley and Armstrong won their events—what impact it would have on Enhanced’s business model of using sports as a showcase for its line of performance products if the people using those products didn’t actually win anything. “I know what you mean, but mostly our business model is headlines to drive attention,” he said. “Any debate is good for us.” 

    In early May, Enhanced began trading on the New York Stock Exchange with an initial value of $1.2 billion.

    That same week, it was finally go time. The athletes and coaches left Abu Dhabi and flew to Las Vegas, where they were put up in five-star luxury at the Conrad hotel inside Resorts World while they made their final preparations. 

    When I got there a few weeks later, toward the end of May, I found it jarring to see these hulking presences walking around the casino in their Enhanced sportswear, weaving their way through packs of half-drunk tourists, with slot machines flashing in the background and cigarette smoke hanging in the air. I had expected the games to be a bigger deal within the city itself, but they were just one of a thousand things happening in Vegas that weekend—drowned out by a series of BTS shows at the football stadium, by the Golden Knights in the NHL playoffs, by No Doubt’s residency at the Sphere.

    If this was a sporting earthquake, it was one whose tremors were mainly being felt online, where bodybuilding influencers livestreamed to their followers on Kick and Twitch, and where thousands watched on YouTube and Rumble. (D’Souza once told me he’d had “every major sports broadcaster” vying for the rights; in the end, Enhanced struck an exclusive streaming deal with Roku in the US.) 

    A group of well-heeled guests in the VIP area face left to pose for a photographer
    No tickets were sold, so the crowd was a mix of invited guests, investors, and influencers, some of whom had reportedly been flown in on a chartered jet.
    SAEED RAHBARAN

    On the morning of the games, Enhanced held a medical symposium that was supposed to provide a taste of the company’s long-term objectives. The first speaker was Bryan Johnson, the longevity-obsessed entrepreneur famous for plowing his personal fortune into wild attempts to reverse his aging: receiving transfusions of his teenage son’s plasma, measuring his nighttime erections, taking more than 100 supplement pills a day. He spends $2 million per year on all this, but he looked pale and vampiric as he delivered the slightly off-brand message that, really, the most important thing was getting a good night’s sleep: “You don’t need to chase IV infusions; you don’t need to chase crystals. You don’t really need to do much of anything.”

    At 2 p.m., I took two escalators from the conference room down to the arena, where spectators were filtering in. Though it had cost $50 million, it had been constructed in just three and a half weeks, and it showed; on the media tour the previous day, there were still loose screws on the floor of the bleachers. 

    There were a few thousand seats in an open grandstand down one side, and two rows of VIP suites on the other. No tickets were sold, so it was a strange mix of invited guests, investors, and influencers, some of whom had reportedly been flown in from Los Angeles on a chartered jet. The rapper Tyga was the biggest name to grace the “blue carpet,” although I did also spot Fabio James, a Michael Jackson look-alike who has had surgery to make the resemblance even stronger. Rumors swirled that Peter Thiel might show up; they proved unfounded.

    looking up at a balcony of excited attendees including a person at center who is dressed to resemble Michael Jackson.
    In attendance was Fabio James, a Michael Jackson look-alike who has had surgery to make the resemblance even stronger.
    SAEED RAHBARAN

    A few hours before the doors opened, journalists got a stern message from the organizers trying to bar us from interviewing guests. Still, I talked to a Cambridge professor who wanted to use Enhanced as a case study in innovation for his MBA students, a retired Brazilian swimmer with the Olympic rings tattooed on his forearm, and a biotech investor wearing an Enron hat. Proud’s family and friends were sheltered from the blazing sun in the shadow of the big screen. 

    D’Souza was nowhere to be seen. Nor was he really mentioned at all—not during the introductory press conference, where Martin was introduced as the “founder of the Enhanced Games,” nor during the event itself, where the athletes showered praise on Angermayer and Martin. But the tens of millions D’Souza had banked from the stock listing likely softened any blow. Plus, he’s already moved on to his next provocative venture: an AI-powered arbitration platform designed to scrutinize the work of journalists on behalf of the rich and powerful.

    As the sun set behind the hills, casting the arena in soft gold light, there were still no world records. That and the wins for clean athletes seemed to put the whole Enhanced project in jeopardy—the knives were already being sharpened online. I asked the organizers whether this threatened the legitimacy of the project. 

    A wet Marius Kusch lays on the ground grimacing as though his eyes are stinging
    German swimmer Marius Kusch was among the dozen or so athletes who hit personal bests in Vegas.
    SAEED RAHBARAN

    “Our response is that enhancements help athletes improve and, in some cases, break records. And yes, some non-enhanced athletes also won—because talent and ability also matter,” Enhanced’s Jones emailed last week. “Breaking world records is incredibly hard as the margin is infinitesimal, as we witnessed. Ignoring that 13 athletes some of whom 10 years later broke personal bests is disingenuous and selective reporting.” 

    Megan Romano was one of them, swimming faster in the 50-meter freestyle at 35 than she had at 22. And Emily Barclay knocked two seconds off her fastest time in the 100-meter freestyle, coming in second in that event and winning the 50-meter freestyle; she went home with a check for $375,000. “No one’s ever heard of this girl,” said Enhanced swim coach Brett Hawke afterwards. “She’s retired; she’s a nobody. She comes out tonight and swims a time that would have got a bronze medal in Paris.” For all the talk of “superhumanity” and pushing the boundaries of performance, making a 35-year-old feel 22 again is probably the perfect marketing message for the products Enhanced wants to sell. 

    Megan Romano stands on the winners area, holding aloft her trophy while Christian Angermeyer and other Enhanced Game participants clap for her.
    Angermayer cheers on swimmer Megan Romano, who swam faster in the 50-meter freestyle at 35 than she did at 22.
    SAEED RAHBARAN

    Enhanced’s executives say people should take enhancements only with medical supervision, but price could be a barrier to heeding that advice. The battery of health tests the company was giving its athletes in the run-up to the games cost $25,000 per athlete per month. The drugs themselves start at $75 a month and go up toward $200. While Jones says the products “are in line with industry price points,” there were almost certainly people watching who saw the drug-altered physiques of athletes like Gkolomeev or Magnussen and decided to find cheaper, less safe alternatives on unlicensed websites.

    “Many of these substances require medical supervision and prescriptions, and several are associated with potentially serious long-term health consequences,” says Bjørnebekk. “Presenting them in this lifestyle-oriented and commercial format risks normalizing use while downplaying the medical risks and uncertainties.”

    Kristian Gkolomeev with his arm raised
    Although his world record-breaking time won’t stand as the official record, swimmer Kristian Gkolomeev will walk away from the Enhanced Games with a million dollar prize in the 50-meter freestyle.
    SAEED RAHBARAN

    Before the night was over,  Gkolomeev again had the chance to right the Enhanced ship. The final event of the night was the men’s 50-meter freestyle swim. His 2025 time had been surpassed by the Australian swimmer Cam McEvoy (without a supersuit) at the China Swimming Open a couple of months before, so he needed to lose another two-hundredths of a second to beat the new record of 20.88 seconds. 

    Gkolomeev was wearing the same supersuit he’d used the previous year, and he’d shaved off his mustache for a little extra streamlining. But he messed up his start—doing four kicks instead of five—and was trailing Proud at the halfway mark. His long arms levered him forward, though, and he reached the wall in 20.81. The spectators were on their feet as “WORLD RECORD” flashed red on the big screen. Martin vaulted over the glass partition from the VIP suites, beaming, to embrace Gkolomeev. They had their record.

    Or did they? Online, people shared screenshots from the video feed, purporting to show that the clock had stopped before Gkolomeev’s hand touched the pressure sensor at the end of the pool. An Enhanced spokesperson gave a statement to the Guardian dismissing this as “completely unfounded internet drivel.” But hey—live by the sword, die by the sword. It’s quite possible Gkolomeev didn’t care. He had another million in the bank. 

    It remains to be seen if it’ll work out so well for the other athletes. Enhanced organizers recently announced a prize of $10 million for anyone who can break Usain Bolt’s 100-meter world record in 2027. They are adamant that the games will happen again next year. If they don’t, dozens of sporting careers will be over, and the athletes will join the long list of victims of VC-backed disruption.

    My personal prediction is that Enhanced will pivot away from the risk and uncertainty of a flagship event—the company’s valuation plunged by almost $800 million when markets opened after what was perceived as an underwhelming set of results in Vegas. I expect you’ll see individual stunts and challenges, tightly controlled and filmed for virality and probably featuring your favorite YouTubers—think Björnsson bench-pressing Jake Paul.

    D’Souza’s initial idea has served its purpose by capturing the world’s attention. But that won’t necessarily translate into success either. Though the company has had plenty of hype over the last 12 months, SEC filings published as part of its stock exchange listing reveal that it generated only $2,755 in revenue from its enhancements business in the first three months of 2026. Would what happened in Vegas be enough to juice sales?

    Max Martin with his mouth open wide to cheer from the VIP stands
    Martin, Enhanced’s CEO, cheers on athletes from the stands. Company leadership insists the competition will take place again next year.
    SAEED RAHBARAN

    As the athletes gathered on the stage to receive their prizes, Martin took the microphone and addressed the crowd. “Enhanced is culture,” he said. “We are at the pulse of where the world is going.” On this, at least, he’s probably right. Testosterone replacement therapy is rapidly moving into the mainstream, and while the science may still not be there on peptides, they have certainly exploded in popularity in the two years since Enhanced launched. And there are undoubtedly more substances yet to be discovered that will promise to improve people’s lives, or at least hold their appearance in stasis. The enhanced age is upon us, whether we want it or not. 

    As the fireworks went off and the Killers closed out the event with “When You Were Young” (“Congratulations to … whoever deserves it,” said frontman Brandon Flowers), I wondered what that might mean for us mere mortals. Invoking Hunter S. Thompson’s Fear and Loathing in Las Vegas in a story about drugs and Las Vegas may be a cliché, but it struck me that fear played a big part in all of this. Fear of missing out. Fear of getting old. Fear of never making a dime on your life’s pursuit. Fear of waking up one morning and seeing your flabby, sunken face in the mirror while everyone around you shines and grins and thrives with white-toothed, alien smiles.

    Megan Romano in cap and goggles with her dry robe stands backlit by pink event lighting and stage fog
    Before joining Enhanced, Romano had not swum competitively in almost a decade.
    SAEED RAHBARAN

    But the big problem with Enhanced’s vision of superhumanity is the question of who gets to join in. “People will be able to enhance themselves if they have enough money,” Sagner had told me the night before the games. The rest of us, I fear, will just have to function as normal human beings.

    Amit Katwala is a journalist and author covering science, culture, and where they collide. His latest book is Tremors in the Blood: Murder, Obsession and the Birth of the Lie Detector. He is based in London.

    ]]>     The State of Biologics Testing Report 2026 https://edusehat.com/en/the-state-of-biologics-testing-report-2026 https://edusehat.com/en/the-state-of-biologics-testing-report-2026 Wed, 10 Jun 2026 22:10:12 +0700 Edusehat The, State, Biologics, Testing, Report, 2026

    The State of Biologics Testing 2026

    The success of modern biologics depends not only on groundbreaking science but on the robustness, speed, and credibility of the testing programs that support them. Across the industry, long established testing paradigms are being re-examined as new modalities emerge, regulatory agencies promote innovation and animal reduction, and development timelines continue to compress.

    Charles River publishes The State of Biologics Testing 2026 to help document and interpret this moment of change. Based on Charles River’s deep involvement in biologics testing worldwide, and informed by interviews with leaders across biopharma, quality, and regulatory functions, the report captures how organizations are navigating the transition—from traditional compendial methods to advanced technologies, digital tools, and risk-based approaches.

    This report is intended to inform discussion and decision making, not to prescribe solutions. Produced in collaboration with GEN, it reflects the collective voice of an industry adapting to new scientific and regulatory realities.

    The post The State of Biologics Testing Report 2026 appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     10x Genomics Acquires Proteintech Genomics, Expanding Proteomics Capablities https://edusehat.com/en/10x-genomics-acquires-proteintech-genomics-expanding-proteomics-capablities https://edusehat.com/en/10x-genomics-acquires-proteintech-genomics-expanding-proteomics-capablities Wed, 10 Jun 2026 22:10:11 +0700 Edusehat 10x, Genomics, Acquires, Proteintech, Genomics, Expanding, Proteomics, Capablities 10x Genomics has acquired Proteintech Genomics for an undisclosed price, the companies said, in a deal driven by the buyer’s commitment to expanding its multiomics presence—in this case, by strategically expanding its proteomics capabilities.

    The acquisition is intended to bring together 10x’s expertise in scalable single-cell and spatial biology platforms with Proteintech Genomics’ capabilities in protein detection. Founded in 2022, Proteintech Genomics specializes in life science technologies enabling researchers to apply single-cell and spatial multiomics tools toward discovery. The company develops fully optimized and highly multiplexed proteomic assays designed for ready integration into various single-cell and spatial analysis application workflows.

    By helping researchers harmonize RNA and protein analysis, Proteintech Genomics has positioned itself as an alternative to the multiple vendors and their workflows traditionally needed to accomplish both analyses, as researchers increasingly combine transcriptomic- and proteomics-based data to gain complementary insights into cellular identity, state, and function.

    “Proteintech Genomics strengthens our capabilities in one of the most exciting and rapidly evolving areas of biology: proteomics,” Michael Schnall-Levin, 10x’s CTO, chief strategy officer, and founding scientist, told GEN. “While transcriptomic technologies have advanced tremendously over the last decade, we believe there is still significant opportunity to push protein-based analysis much further, particularly in combination with RNA and other analytes.”

    “We believe the future of biological analysis will increasingly integrate single-cell, proteomic, and spatial information,” Schnall-Levin added. “This acquisition reflects our conviction that proteomics will be an important part of that future and expands our ability to support richer multiomic workflows across our portfolio.”

    Among Proteintech Genomics’ technologies is its Human Discovery Panel, which, according to the company, is the largest antibody-based single-cell protein panel. The panel allows researchers to simultaneously profile 347 DNA-barcoded antibodies covering 325 distinct protein targets, alongside transcriptomic measurements.

    “Significant opportunities”

    “We see significant opportunities to continue expanding both plex and content over time. One of the exciting aspects of bringing Proteintech Genomics into 10x is the ability to invest more deeply in future proteomic innovation,” Schnall-Levin explained. “While we aren’t announcing specific products today, directionally we’re interested in enabling higher-plex measurements, broader biological content, and more accessible workflows. Those are all areas where we see substantial opportunity going forward.”

    The Human Discovery Panel is also designed to support integrated analysis of intracellular proteins, cell surface proteins, and transcriptomic profiles within sequencing-compatible workflows. The panel is compatible with 10x’s Flex chemistry, including the Flex Apex assay, the company’s fastest-growing single-cell assay.

    “Proteintech Genomics brings innovative protein detection technologies, including the Human Discovery Panel, as well as deep expertise in protein biology, antibody panel development, and assay design. Together, we believe we can move faster, pursue more ambitious product development efforts, and make high-quality multiomic workflows more accessible and scalable for researchers,” Schnall-Levin said.

    He said one attraction to 10x of combining with Proteintech Genomics was the combination it offered to 10x of technical performance and ecosystem fit: “Proteintech Genomics has been a longtime 10x partner, and its products were purpose-built to work with 10x workflows. Our customers are already using these technologies together today, which gives us a strong foundation to innovate more quickly and bring even more powerful multiomic capabilities to the field.”

    Through its acquisition of Proteintech Genomics, 10x aims to achieve its vision of enabling multimodal biological analysis across its single-cell and spatial platforms—including protein capabilities planned for the Atera spatial platform, unveiled during the American Association for Cancer Research (AACR) conference.

    Atera can run up to 800 1 cm2 whole transcriptome samples (FFPE and fresh frozen) per year, with flexible run configurations, and a greater than 5 cm² imageable area per slide (for greater than 2,000 mm² total tissue per run when using all four slides). The Atera WTA (Whole Transcriptome Analysis) panel targets more than 18,000 genes, with stackable customization of 1,000-gene Atera Select panels available now—and optional stacking of up to three 1,000-gene panels coming in the future.

    “We’ve already spoken publicly about future protein capabilities planned for Atera, and this acquisition strengthens our ability to deliver on that vision while continuing to advance integrated approaches across both our single-cell and spatial platforms, Schnall-Levin said.

    Higher-plex proteomics challenge

    10x’s interest in higher-plex proteomics makes sense since it is the fastest-growing submarket in the $11 billion core proteomic space, set to grow by double digits, according to Leerink Partners. But a Leerink analyst cautioned that the company will find it challenging to grow in the space as it absorbs costs associated with Atera and Flex Apex.

    “TXG leveraging itself to higher-plex proteomics is helpful,” Puneet Souda, senior managing director, life science tools and diagnostics, and a senior research analyst with Leerink, wrote in a research note. “We believe the deal accelerates higher-plex proteomics content for Atera, which adds to its long-term value. TXG can also bundle single-cell proteomic kits, which is a small but growing area of single-cell research.”

    “Nonetheless, we still expect the digestion of Flex Apex and Atera-driven freezing/impact to weigh on near-term growth, particularly in a challenged academic funding backdrop,” Souda added.

    Investors did not appear to share that concern, as 10x shares dipped 0.6% as of 12:21 p.m. ET, to an even $29.00 from $29.18 at Monday’s close. The share fluctuated between $28.93 and $30.87 during Tuesday morning trading.

    Ci Chu, senior vice president, AI-enabled discovery for Xaira Therapeutics, said in a statement included within 10x’s announcement that the acquisition reflected the importance of integrating protein biology with single-cell and spatial technologies.

    “Biology is bigger than transcriptomics alone,” Chu stated. “Bringing scalable protein measurements into single cell and spatial biology is an important step toward richer, more predictive views of cellular state—and ultimately, virtual cell models that better reflect the complexity of living systems.”

    Potential clinical applications

    In addition to facilitating expansion in multiomics, the acquisition of Proteintech Genomics could help 10x achieve another longer-term goal, namely, looking beyond its traditional focus on research tools for academic, government, and industry customers, by strengthening its clinical diagnostic offerings.

    10x expanded into clinical diagnostics during the J.P. Morgan 44th Annual Healthcare Conference, when the company announced three partnerships with top-tier institutions: Brigham and Women’s Hospital and Dana-Farber Cancer Institute, both in Boston, as well as the New York-based Cancer Research Institute. 10x also committed to building its own CLIA-certified laboratory within about a year, with the goal of enabling clinical deployment of diagnostics that will come up with such collaborations.

    Integrating Proteintech Genomics tools into clinical settings will be a longer-term priority since the company’s offerings are for research use only and not intended for diagnostics procedures.

    “Protein measurements are highly relevant to many translational research applications, particularly in areas such as immunology, oncology, and neurology. Researchers in academia and biopharma increasingly want to combine protein and RNA measurements to better understand cellular function, therapeutic response, and disease biology,” Schnall-Levin said. “Longer term, we believe single-cell and spatial proteomics are likely to play a meaningful role in diagnostics.”

    10x and Proteintech Genomics did not disclose the price or other financial terms of the acquisition, though 10x said it believes the transaction “will not meaningfully impact its near-term financial outlook.”

    10x finished the first quarter with a net loss of $13.47 million, less than half its $34.358 million net loss of Q1 2025, on revenue that shrunk 3% year-over-year, to $150.843 million from $154.883 million in the first three months of last year. The company reported $490.285 million in cash and cash equivalents as of March 31, up 3% from $473.966 million as of December 31, 2025.

    Proteintech Genomics is a subsidiary of Proteintech Group, a developer of high-plex proteomic solutions for single-cell and spatial applications; both companies are privately held.

    San Diego-based Proteintech Genomics’ workforce consists of what Schnall-Levin described as “a small team of eight people,” including CEO Kristopher Nazor, PhD.

    “We’re going to keep that team,” Schnall-Levin declared.

    Added Nazor: “From day one, Proteintech Genomics was built around the belief that RNA and protein measurements are most powerful when used together.”

    “Because our technologies were designed to integrate with 10x workflows, joining the 10x team feels like a natural next chapter,” Nazor continued. “We are excited about the opportunity to accelerate innovation together and expand access to integrated multiomic approaches for researchers around the world.”

    The post 10x Genomics Acquires Proteintech Genomics, Expanding Proteomics Capablities appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     mRNA Tails Play Key Role in Folding Regulatory Proteins https://edusehat.com/en/mrna-tails-play-key-role-in-folding-regulatory-proteins https://edusehat.com/en/mrna-tails-play-key-role-in-folding-regulatory-proteins Wed, 10 Jun 2026 22:10:10 +0700 Edusehat mRNA, Tails, Play, Key, Role, Folding, Regulatory, Proteins mRNA 3′ UTRs have hundreds of highly conserved nucleotides, but their biological roles are unclear. In a new study published in Cell titled, “mRNA 3′ UTRs chaperone intrinsically disordered regions to control protein activity,” researchers from Memorial Sloan Kettering (MSK) Cancer Center now demonstrates that mRNA 3′ UTRs play a key role assisting the folding of regulatory proteins. 

    “The traditional view is that only specialized proteins act as ’chaperones’ to help other proteins fold correctly,” said Christine Mayr, MD, PhD, a member of the Sloan Kettering Institute and corresponding author on the paper. “Our research shows that RNA can do this, too—and that mRNAs act as their own chaperones for a group of important, hard-to-fold proteins.” 

    While 3′ UTRs have traditionally been dismissed as key regulators, Mayr emphasizes that thousands of human 3′ UTRs have highly conserved sequences across vertebrates, offering a clue of their function. “Biology doesn’t usually preserve things that aren’t needed,” she says. 

    Many larger, complex regulatory proteins, such as the transcription factors MYC, UTX, and JMJD3, possess long, flexible regions, named intrinsically disordered regions (IDRs), that do not fold into stable structures on their own.

    The study showed that cells solve this folding problem using specialized compartments, known as mesh-like condensates. The 3′ UTR promotes IDR–IDR interactions and suppresses folding between domains. Results suggest that this chaperone activity prevents interference between hydrophobic clusters in the IDR with folding of the structured domain. 

    The team identified more than 2,700 genes with highly conserved 3′ UTRs, or about one in every eight protein-coding genes in the human genome. The proteins expressed by these genes contain intrinsically disordered regions that require RNA chaperones to facilitate folding. 

    “What we show is that for thousands of regulatory proteins in human cells, the genetic code alone isn’t enough to make a functional protein—you need the RNA chaperone too,” said Mayr. 

    The study has practical implications for laboratory research. For thousands of regulatory proteins, removing the 3′ UTR allows researchers to study the misfolded, and less active version of the protein. 

    The post mRNA Tails Play Key Role in Folding Regulatory Proteins appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     T-Cell Synapse Formation Is Restrained by PTPN22–PSTPIP1 Signaling https://edusehat.com/en/t-cell-synapse-formation-is-restrained-by-ptpn22pstpip1-signaling https://edusehat.com/en/t-cell-synapse-formation-is-restrained-by-ptpn22pstpip1-signaling Wed, 10 Jun 2026 11:15:09 +0700 Edusehat T-Cell, Synapse, Formation, Restrained, PTPN22–PSTPIP1, Signaling T cells don’t simply switch on—they reshape themselves. When these immune sentinels recognize a target, they rapidly reorganize their internal scaffolding to build an immunological synapse, a nanoscale interface that determines how strongly they respond. But that architectural overhaul needs brakes. Without them, T cells risk becoming hypersensitive, reacting to weak cues, and drifting toward autoimmunity. Now, new work reveals that one of those brakes—PTPN22 (proline-serine-threonine phosphatase–interacting protein 1)—acts not only on signaling molecules but also on the cytoskeletal machinery that sculpts the synapse itself.

    In a study published in Science Signaling, lead author Megan Joseph, PhD, of University College London and colleagues uncover how PTPN22 interacts with the cytoskeletal adaptor protein PSTPIP1 to restrain actin remodeling at the T‑cell synapse. Their paper, “PTPN22 regulates T-cell synapse formation through PSTPIP1-dependent actin remodeling,” shows that this phosphatase plays a previously unappreciated role at the plasma membrane, shaping how T cells respond to antigens of varying affinity. As the authors wrote, “These findings uncover a PTPN22–PSTPIP1 signaling axis that is critical for regulating cytoskeletal remodeling and receptor organization, providing insights into T-cell hyperactivation that may be relevant to autoimmune disease.”

    PTPN22 is already well known as a negative regulator of early T‑cell activation. Variants in the gene, including the autoimmune‑associated R620W allele, have been linked to diseases ranging from lupus to rheumatoid arthritis. Using super‑resolution DNA‑PAINT imaging, Joseph et al. visualized how T cells reorganize their actin networks as they engage activating ligands. In wild‑type Jurkat cells, PTPN22 helped maintain orderly actin dynamics. In its absence, however, PSTPIP1 accumulated at T cell receptors (TCRs), disrupting Arp2/3‑dependent actin polymerization and generating dense central F‑actin foci, as well as enhanced Ca2+ signaling, especially under low-affinity stimulation of the TCR, according to the paper.

    This hyper‑remodeling had functional consequences. PTPN22‑deficient cells became unusually sensitive to low‑affinity antigens, responding more vigorously than their wild‑type counterparts. “Autoimmunity is inherently linked to immune tolerance mechanisms normally associated with low-affinity TCR responses to self, which, when breeched lead to inappropriate immune reactions. To better understand how PTPN22 contributes to these processes, we used WT and PTPN22 KO TCR−/− Jurkat cells engineered to express a transgenic TCR with high affinity for the pTax peptide and low affinity for the pHuD peptide,” the authors wrote.

    Joseph and colleagues suggest that understanding this axis could inform both autoimmune research and efforts to modulate T‑cell activation in cancer immunotherapy. By mapping how PTPN22 and PSTPIP1 coordinate actin remodeling, the study provides a mechanistic foothold for exploring how synapse architecture shapes immune outcomes.

    The post T-Cell Synapse Formation Is Restrained by PTPN22–PSTPIP1 Signaling appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Fifteenth In Vivo Lentiviral Vector-Based Therapeutic Technology Added to VIVEbiotech’s CGT Platform https://edusehat.com/en/fifteenth-in-vivo-lentiviral-vector-based-therapeutic-technology-added-to-vivebiotechs-cgt-platform https://edusehat.com/en/fifteenth-in-vivo-lentiviral-vector-based-therapeutic-technology-added-to-vivebiotechs-cgt-platform Wed, 10 Jun 2026 04:05:13 +0700 Edusehat Fifteenth, Vivo, Lentiviral, Vector-Based, Therapeutic, Technology, Added, VIVEbiotech’s, CGT, Platform Spain-based CDMO VIVEbiotech added its 15th in vivo lentiviral vector-based therapeutic program using its platform. These programs, several of which have already received regulatory clearance for clinical trials, including from the FDA, span a range of applications such as in vivo CAR T, rare diseases, gene editing, and vaccines, according to the company.

    Growing interest in in vivo cell and gene therapies is driving significant investment, given their potential to address some of the manufacturing and commercialization challenges associated with current ex vivo approaches. However, the direct administration of lentiviral vectors imposes significantly more stringent requirements on the quality attributes of the final product, notes Jon Alberdi, CEO of VIVEbiotech. Accordingly, process control must be optimized to improve both yield and purity. As manufacturing becomes increasingly complex, the scope of analytical characterization is also expanding.

    “In vivo lentiviral vectors have the potential to transform treatment paradigms through faster administration and direct therapeutic delivery,” says Alberdi. “However, these advantages come with more stringent manufacturing requirements—from achieving the required purity profile to ensuring consistent performance at scale.”

    “As interest in in vivo delivery continues to grow, we are witnessing a fundamental shift in how gene therapies are developed and brought to patients,” adds Marie Fertin, chief custom solution and process development officer at VIVEbiotech. “Our continued investment in capabilities reflects both our confidence in this field and our commitment to enabling our partners.”

    The company’s platform has been specifically designed to preserve lentiviral vector integrity throughout the manufacturing process by minimizing shear stress and maintaining cell health, thereby ensuring high vector functionality, continues Fertin. By integrating process intensification strategies with optimized transfection conditions, reduced reagent usage, and improved productivity, the upstream setup contributes to enhanced yields and a significant reduction in cost of goods, she maintains.

    Beyond manufacturing, VIVEbiotech reports that it has developed a fully customized analytical framework tailored to in vivo lentiviral vectors, specializing in advanced vector characterization, including potency assay development. A full testing panel is proposed following regulatory feedback received for direct in vivo administration.

    A company spokesperson points out that VIVEbiotech also supports large-scale manufacturing of in vivo programs. With more than 3,000 sqm of GMP facilities and seven cleanrooms, VIVEbiotech says it works to ensure timely delivery across development stages. An ongoing expansion plan will further increase manufacturing capacity by 2028, supporting the growing demand for in vivo therapies.

     

    The post Fifteenth In Vivo Lentiviral Vector-Based Therapeutic Technology Added to VIVEbiotech’s CGT Platform appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Incyte to Acquire Vega Therapeutics for Up-to-$2B, Growing Hematology Pipeline with Phase III VWD Candidate https://edusehat.com/en/incyte-to-acquire-vega-therapeutics-for-up-to-2b-growing-hematology-pipeline-with-phase-iii-vwd-candidate https://edusehat.com/en/incyte-to-acquire-vega-therapeutics-for-up-to-2b-growing-hematology-pipeline-with-phase-iii-vwd-candidate Wed, 10 Jun 2026 04:05:12 +0700 Edusehat Incyte, Acquire, Vega, Therapeutics, for, Up-to-2B, Growing, Hematology, Pipeline, with, Phase, III, VWD, Candidate Incyte has agreed to acquire Vega Therapeutics for up to $2 billion, the companies said, in a deal designed to bolster the buyer’s hematology pipeline with antibody assets led by VGA039, a Phase III candidate for von Willebrand disease (VWD).

    Vega, a wholly owned subsidiary of privately held Star Therapeutics, focuses on developing treatments for bleeding disorders. Vega’s lead candidate VGA039 could, if approved, be the first subcutaneous prophylactic therapy with a more convenient once-monthly, self-administered dosing regimen for patients with VWD, compared with current therapies requiring more frequent (2-3x/week) intravenous infusions.

    VGA039 is a monoclonal antibody designed to modulate Protein S with the aim of improving hemostasis, potentially improving the body’s ability to control bleeding in numerous bleeding disorders. VGA039 is under study in the Phase III VIVID-6 trial (NCT07115004), a global single arm crossover study designed to investigate the safety and efficacy of subcutaneous administration of VGA039 as prophylaxis for bleeding in patients with every type of VWD, including those with a high disease burden.

    VIVID-6’s estimated completion date is October 2028, with data expected to be read out in early 2029.

    “VGA039 fits directly into our strategy of building a top-tier growth company for the future,” Incyte CEO Bill Meury said in a statement. “It is a first-in-class, Phase III asset with compelling early data, a manageable development path and the potential to become an important new growth driver in one of our core therapeutic areas, hematology. The transaction has all of the attributes we look for in business development opportunities.”

    $1B+ opportunity

    In a presentation to analysts Monday morning, Incyte quantified that potential market opportunity as “$1B+ global net sales opportunity.”—an estimate with which three analysts concur:

    “VGA039 has the potential to address a clear unmet need for a practical, targeted therapy for von Willebrand disease, and even with conservative assumptions around pricing and market penetration, VGA039 has a clear path to a more than $1 billion market opportunity,” Matt Phipps, PhD, partner and group head of biotechnology equity research with William Blair, wrote Monday in a research note.

    “Overall, we believe the deal for VGA039 fits well into Incyte’s current hematology franchise and capabilities and offers a relatively de-risked Phase III asset with blockbuster commercial potential in the 2030s,” Phipps added.

    Jessica Fye, a managing director and senior equity research analyst with J.P. Morgan, was also bullish on VGA039’s commercial potential: “We think mgmt [management] framing VGA039 as a potential $1bn+ global sales opportunity is credible and think it should be able to leverage some of INCY’s existing presence with hematology centers.”

    Faisal Khurshid, equity analyst with Jefferies, agreed that VGA039 “could have blockbuster potential” assuming it is priced at about $500,000/year compared with the $0.5 to $1 million range of current prophylactic therapies, and assuming ~2,000 patients at hemophilia treatment centers receive frequent IV prophylaxis out of 7,000-10,000 patients who have severe or recurrent bleeds.

    “We feel that VGA039 largely fits INCY’s strategic goals and is well-positioned to succeed in Ph[ase III],” Khurshid wrote in a research note.

    Despite the positive comments from analysts, Incyte shares dipped 1.7% Monday, from $102.38 to $100.64, though the stock rebounded Tuesday in early trading, rising nearly 3% to $103.31 as of 10:25 a.m. ET.

    Eligible for voucher

    VGA039 has received the FDA’s Fast Track, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease (RPD) designations. The RPD designation made Star Therapeutics eligible to receive a Rare Pediatric Disease Priority Review Voucher (PRV) upon approval of a Biologics License Application for VGA039—eligibility that would transfer to Incyte if its acquisition of Vega occurs as planned. The voucher may be redeemed to obtain priority review for a subsequent marketing application or transferred or sold to another sponsor.

    The Breakthrough Therapy designation was supported by interim data from the Phase I/II multidose study (NCT05776069) of VGA039 in adult and adolescent patients with VWD, showing substantial bleed reductions across all types of VWD and all types of bleeds. The data was presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2025.

    Vega’s pipeline includes two other programs, both preclinical—a complement therapy program, and an undisclosed program.

    Looking beyond Jakafi®

    Acquiring Vega and its pipeline is among moves Incyte has undertaken in recent months under Meury to recoup the billions of dollars in sales that it stands to lose once its aging blockbuster Jakafi® (ruxolitinib) loses patent exclusivity in 2028—one of the Top 20 Drugs Heading for the Patent Cliff through 2029, according to a recent GEN A-List.

    Jakafi, marketed outside the U.S, as Jakavi®, generated $3.093 billion in net product revenues last year, up 11% from $2.792 billion in 2024. Jakafi finished the first quarter with $757.755 million in net product revenues, up 7% from $709,412 in Q1 2025.

    Incyte has agreed to pay Star $1.25 billion upfront for Vega, plus up to $750 million in payments tied to achieving sales milestones.

    The boards of Incyte and Star have approved the acquisition deal, through which Incyte will acquire all of Vega’s outstanding shares through a stock purchase agreement. The deal is subject to expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary closing conditions.

    Incyte expects to incur an R&D charge of approximately $1.25 billion, to be included in third quarter and full year 2026 GAAP and non-GAAP results, as a result of the acquisition.

    However, the benefits of an acquisition by Incyte outweigh its costs, Vega and parent Star Therapeutics reason.

    “This milestone reflects our team’s deep commitment to innovation and underscores our strategy to develop first-in-class and best-in-class therapies for serious conditions with high unmet need,” stated Adam Rosenthal, PhD, Star’s founder and CEO.

    The post Incyte to Acquire Vega Therapeutics for Up-to-$2B, Growing Hematology Pipeline with Phase III VWD Candidate appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     First-in-Human Trial Reports Promising Dual Lassa–Rabies Vaccine Data https://edusehat.com/en/first-in-human-trial-reports-promising-dual-lassarabies-vaccine-data https://edusehat.com/en/first-in-human-trial-reports-promising-dual-lassarabies-vaccine-data Wed, 10 Jun 2026 04:05:11 +0700 Edusehat First-in-Human, Trial, Reports, Promising, Dual, Lassa–Rabies, Vaccine, Data Researchers at the University of Maryland School of Medicine’s Center for Vaccine Development and Global Health (CVD) reported encouraging interim results from an early clinical trial that tested a new dual vaccine against Lassa fever and rabies. The study found the vaccine to be safe and induced immune responses against both the Lassa fever and rabies viruses (RB). There are currently no vaccines against Lassa fever on the market.

    “This vaccine is designed to protect against two viruses of global health importance,” said study principal investigator Justin Ortiz, MD, MS, professor of medicine at UMSOM and vaccine researcher at CVD. “By combining targets into a single product, it could reduce the need for separate vaccination efforts and streamline delivery in settings where access is limited.”

    Ortiz is first and corresponding author of the researchers’ published paper in Nature Medicine, titled “Adjuvanted inactivated rabies virus-vectored Lassa virus vaccine in healthy adults: a phase 1 trial,” in which they said, “If efficacy is confirmed, this combination vaccine could help protect populations from two priority pathogens and have a meaningful public health impact in regions where both diseases remain major threats.”

    The World Health Organization has identified Lassa virus (LASV) as a public health threat in western Africa and made Lassa fever a priority disease for research. “Transmission occurs primarily through contact with food or household items contaminated by urine or feces from Mastomys rodents, although person-to-person spread can occur via exposure to bodily fluids or contaminated surfaces,” the investigators noted in their paper. Like Ebola, Lassa virus can trigger severe illness and periodic outbreaks in African nations.

    Lassa virus infections occur in 300,000 people every year resulting in 5,000 deaths, according to figures cited by the authors, but these numbers are likely an underestimate due to limited surveillance. The disease is particularly dangerous in pregnancy with over 80% of late-term infections resulting in deaths to the mother or fetus. Additionally, regions where Lassa fever is common, such parts of Western and Sub-Saharan Africa, also have a high burden of rabies, with thousands of deaths annually, a disease that is almost always fatal once symptoms develop. The newly reported first-in-human trial was designed to evaluate the safety and immunogenicity of an adjuvanted inactivated rabies virus expressing the Lassa virus glycoprotein complex (GPC) on the surface of the virus. “Scientists at Thomas Jefferson University developed an inactivated rabies-vectored combination vaccine derived from an attenuated rabies strain, LASSARAB, expressing both the rabies glycoprotein and the LASV (Josiah strain) GPC,” the authors explained. “The RABV platform provides a well-established foundation for a dual-target vaccine.”

    For the randomized, controlled trial 54 healthy adult volunteers from the Baltimore area were randomly assigned to receive different doses of LASSARAB, with an adjuvant or a licensed rabies vaccine control. Participants received two vaccine doses 28 days apart. Immune responses were studied through 61 days post-vaccination for an interim analysis. The results indicate that LASSARAB was safe with no serious adverse events (AEs) reported after vaccination. Additionally, the candidate vaccine induced rapid and robust antibody responses against both Lassa and rabies viruses when compared with the control, which only induced an immune response against rabies virus.

    The study is ongoing, and vaccine safety and immune responses will be further studied through 394 days post-vaccination. “The final study report will be prepared after study completion and will include serious AEs and AEs of special interest through day 394, protocol-defined exploratory LASV and RABV antibody responses assessed at days 121 and 394, and any additional post hoc analyses, as applicable,” the team stated.

    If the results indicate continued elevated immune responses from vaccination, researchers will proceed with more advanced clinical trials. Importantly, this investigational vaccine can be freeze-dried for storage, enabling distribution to areas of the world where it may be difficult to maintain cold chains. Importantly, this investigational vaccine can be freeze-dried for storage, enabling distribution to areas of the world where it may be difficult to maintain cold chains.

    “These data support the feasibility of a bivalent rabies-vectored vaccine integrated into routine immunization for regions where cold-chain capacity is limited,” the team added.

    “This study highlights CVD’s commitment to tackling diseases of global significance,” commented Stefan Kappe, PhD, director of the Center for Vaccine Development and Global Health and the Myron M. Levine Endowed Professor of Pediatrics. “LASSARAB not only targets diseases of concern but utilizes a platform that could make distribution attainable in the areas of the world that are most affected by these diseases.”

    Added UMSOM dean Mark T. Gladwin, MD, “Climate change is causing Lassa fever to extend its reach far beyond its Nigerian and West African origins, putting an estimated 700 million people at risk worldwide. By 2070, the number of countries across Africa that will develop ecological conditions suitable for Lassa virus spread could drastically increase, so a vaccine to prevent this deadly infection is desperately needed.”

    Last year, before results were available, the trial was highlighted by Nature Medicine in its 2025 feature, “Eleven clinical trials that will shape medicine in 2026,” which identified studies to watch based on their potential to address major unmet health needs.

    The post First-in-Human Trial Reports Promising Dual Lassa–Rabies Vaccine Data appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Complete Connectome of Fruit Fly Central Nervous System Now Open-Source https://edusehat.com/en/complete-connectome-of-fruit-fly-central-nervous-system-now-open-source https://edusehat.com/en/complete-connectome-of-fruit-fly-central-nervous-system-now-open-source Wed, 10 Jun 2026 00:25:09 +0700 Edusehat Complete, Connectome, Fruit, Fly, Central, Nervous, System, Now, Open-Source A new study published in Nature titled, “Distributed control circuits across a brain-and-cord connectome”, describes a complete wiring diagram of all the connections between neurons in the central nervous system of an adult fruit fly for translational applications.

    The work was completed by an international team led by multiple labs at Harvard Medical School (HMS) and Princeton University. The team has made the entire connectome accessible online to propel research into complex behaviors and other fundamentals of the nervous system. 

    The fruit fly, Drosophila melanogaster, offers an effective model as they are easy to breed and maintain in the lab. Despite having a relatively simple nervous system made up of around 160,000 neurons, they exhibit complex behaviors such as navigation, social interaction, learning, and responding to sensory cues. 

    To build the connectome, the team used electron microscopy to produce millions of images of neurons and neural connections. AI tools aligned the images into a cohesive 3D map. 

    “It is really important to have a central nervous system connectome that is as complete as possible so we can link up the brain and body and start thinking about behavior holistically,” said Wei-Chung Allen Lee, PhD, associate professor of neurobiology at HMS and co-corresponding author on the study. 

    The connectome shows how each neuron connects in the brain and nerve cord at the synapse level. While the map doesn’t span the fly’s entire body, the team used identifiable neurons and literature review to connect the central nervous system to neurons in appendages and sensory organs. 

    The authors have already used the connectome to explore motor control. While a longstanding idea in neuroscience is for a centralized controller in the brain to make decisions about actions, the authors discovered that motor control in the fruit fly mostly occurs at a local level. For example, movement of a fly’s leg is primarily controlled by the neural circuits for that leg. The local circuits for one leg then communicates with other appendages to carry out complex coordinated movements, such as walking. 

    “The brain and nerve cord connectomes are each useful on their own, but until you can bridge the two, it’s hard to understand how information moves between the brain and the body,” said co-first author Helen Yang, PhD, a research fellow in neurobiology at HMS. 

    Looking ahead, the researchers plan to add more information to the connectome, including data describing neuropeptides, molecules that support neuron communication. Insights from the connectome may reveal fundamental principles about how nervous systems operate across species, including in humans. 

    The post Complete Connectome of Fruit Fly Central Nervous System Now Open-Source appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     David Sinclair plans to test whole-body rejuvenation drugs in the XPrize competition https://edusehat.com/en/david-sinclair-plans-to-test-whole-body-rejuvenation-drugs-in-the-xprize-competition https://edusehat.com/en/david-sinclair-plans-to-test-whole-body-rejuvenation-drugs-in-the-xprize-competition Tue, 09 Jun 2026 20:55:02 +0700 Edusehat David, Sinclair, plans, test, whole-body, rejuvenation, drugs, the, XPrize, competition
  • A bold new bet on whole-body rejuvenation: Harvard biologist David Sinclair plans to test an oral "reprogramming" drug on human volunteers as part of a $101 million XPrize competition.

  • Chemicals instead of gene therapy: Sinclair's new drug candidate — code-named SL-100 — uses drugs to mimic the effects of embryonic genes, and will attempt to reset aging across the body.

  • Experts urge caution: Other scientists warn that similar chemical reprogramming efforts have so far proven either ineffective at low doses or outright toxic at high ones — and Sinclair's unpublished animal data has yet to face outside scrutiny.

  • The field's bigger problem: Scientists still can't agree on how to reliably measure aging or age reversal, making the XPrize competition as much about establishing scientific standards as it is about crowning a winner.
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    The outspoken longevity scientist David Sinclair has been predicting that one day, you’ll go to the doctor and get a prescription that will make you 10 years younger.

    Now MIT Technology Review has learned that he has plans to launch human tests of an oral “reprogramming” drug as part of a $101 million competition organized by the XPrize Foundation. 

    The foundation is offering cash awards to teams able to “restore” a person to an earlier apparent age, as measured by improvements in immune, cognitive, and muscle function. 

    The grand prize goes to any team able to show a 10-year (or greater) relative improvement after one year of treatment. 

    Reached by phone, Sinclair, a biologist at Harvard Medical School, confirmed that he plans to give an oral drug mixture to volunteers in a bid to seek “evidence for age restoration in humans.”

    The trial, if it goes forward, will be a significant new development in the race to harness so-called epigenetic reprogramming. That technology is based on the discovery, 20 years ago, of powerful genes able to turn an adult cell into a stem cell similar to those found in embryos.

    The age-reversal effect is believed to occur via a resetting of molecular controls on DNA known as epigenetic marks, which help determine a cell’s overall metabolism and identity.

    Companies are now racing to use that phenomenon for a new form of rejuvenation medicine. Only this January, one of Sinclair’s companies, Life Biosciences, made news by winning approval to launch an initial human trial using a set of powerful reprogramming genes. The company announced today it had treated its first patient. 

    But that test involves a complex gene therapy and is limited to patients’ eyes, where it could treat conditions like glaucoma. 

    Sinclair’s new plan is bolder: a reprogramming drug you’d swallow in order to promote such effects across the body. 

    “What we’re aiming to do is to epigenetically restore the animal and eventually the person,” he says. “It is true that we’ve been doing extensive animal studies with the oral agent and are looking to compete in the XPrize.”

    This alternative method, chemical reprogramming, uses drugs to mimic the effects of the embryonic genes. That is significant because drug compounds can travel through the bloodstream, reaching most or all cells in a person’s body. 

    Some experts expressed caution, saying the chemical process, at least as used in labs, is extremely harsh and not even particularly effective. “Who doesn’t dream of whole-body rejuvenation? I think it’s a great goal,” says Sergiy Velychko, founder of Soxogen, a stealth reprogramming company in Boston. “But these chemicals are used in very, very high concentrations for cell reprogramming.”

    Sinclair declined to describe the exact makeup of the drug candidate, code-named SL-100, calling its contents “highly, highly confidential.”

    However, he has previously published lab studies of what he called “epigenetic age-reversal cocktails,” which mixed powerful chemicals with known supplements and commercially available medicines. 

    It’s those latter components that would be easiest to test on people, since doctors are free to prescribe them, even for unusual objectives like age reversal. James Clement, head of Betterhumans, an organization that specializes in life-extension studies using existing drugs, said in a message that he is “running clinical trials” of an oral reprogramming cocktail for Sinclair’s XPrize team.

    Sinclair’s team is competing in the XPrize Healthspan Competition, launched in 2023. It follows several previous competitions that focused on commercial spaceflight, lunar landings, and other goals. The XPrize Foundation is led by executive chairman Peter Diamandis, also an active promoter of longevity research.

    “If two teams are equivalent, they would split the award,” says Jamie Justice, a doctor and executive director for the contest, which was bankrolled by Saudi Arabia’s Hevolution Foundation, “But it will be incredibly hard to even get to one winner.”

    Justice says a judging panel is now in the process of picking 10 finalists from 65 teams that have been exploring health foods, lifestyle interventions, digital trackers, and drug compounds. 

    Sinclair’s team, Justice says, was a late entrant to the contest, but like all teams, it would be required to move into wider human tests starting this year. “You have to be ready and in trials,” she says.

    The race to harness the reprogramming phenomenon and apply it to living people is heating up, even outside the XPrize competition. On June 2, a startup called NewLimit, founded by the crypto billionaire Brian Armstrong, said it had raised a further $435 million, from investors including Peter Thiel’s Founders Fund, to support what it calls “age reprogramming.” 

    The company says it is working toward delivering genetic reprogramming instructions to the liver, to treat diseases of that organ.

    But Sinclair has been saying that whole-body rejuvenation is a possibility too. And for that, chemicals, rather than gene therapy, could be the most practical strategy. 

    Sinclair says his lab has been searching for such compounds and is starting to use AI “to improve the oral agents that we’re testing.”

    Chemical reprogramming cocktails, as used in labs, typically involve a mix of vitamins, approved drugs, and experimental molecules. For instance, one recipe Sinclair filed a patent on includes the supplement forskolin,  the antidepressant tranylcypromine, and an experimental chemical, laduviglusib, which has been tested against Alzheimer’s, among other ingredients.

    “In those days it was a six-factor cocktail,” Sinclair says of his earlier research. “But we’ve come a long way. I can’t disclose what’s in it, but it’s an improvement and an advance on that, and we’ve done a number of animal studies. They are not published, but we’ve been doing them for a long time, and we want to make sure that we’ve done a full investigation of safety and efficacy before we release any of the data.”

    While Sinclair’s results aren’t published, other teams say attempts to reverse the age of entire animals using chemical drugs haven’t worked yet. Last year, the lab of Vadim Gladyshev, another Harvard biologist and a member of a different XPrize team, reported on its attempt to rejuvenate mice by installing pumps in their bodies that released controlled doses of seven compounds.

    Gladyshev says the procedure proved to be toxic. “The idea was to see if we could rejuvenate whole animals. Unfortunately, we have not found [the right] conditions,” he says. “At low concentrations there was no effect, and high concentrations were toxic.”

    Gladyshev says he doesn’t know what is in Sinclair’s cocktail, but says that “trying to improve the combinations makes sense.”

    Sinclair, who is the author of several books on aging and has a large social media following, has frequently been criticized by other scientists for making unproven rejuvenation claims. 

    In 2024, he resigned as president of the Academy for Health and Lifespan Research after claiming that a supplement developed by a company his brother runs had “reversed” the age of dogs, a claim for which there was so little evidence that one scientist called it a “lie.”

    Part of the problem is that scientists still disagree on how to measure aging. And they don’t have a reliable way to measure age reversal, either, should it ever be achieved.

    Justice, the XPRIZE director, says a primary purpose of the competition is to solve that problem by encouraging the development of standardized measures of aging. That is so that anti-aging drugs can be assessed reliably, and, one-day, approved by regulators if they work.

     “We as a scientific field have been forced to ask, ‘If a medicine improves how we age, how would we know?” Justice said during a public meeting with FDA officials in May. “If something worked, what would convince us as scientists, what’s meaningful to the general public?”

    Finalists in the Healthspan competition will be announced in August.

    ]]>     Section 232 tariff uncertainty remains high as onshoring plan deadline looms https://edusehat.com/en/section-232-tariff-uncertainty-remains-high-as-onshoring-plan-deadline-looms https://edusehat.com/en/section-232-tariff-uncertainty-remains-high-as-onshoring-plan-deadline-looms Tue, 09 Jun 2026 17:20:04 +0700 Edusehat Section, 232, tariff, uncertainty, remains, high, onshoring, plan, deadline, looms Strengthening America’s biopharma manufacturing base and biotechnology leadership are goals both BIO and the Trump administration share. But as companies work to advance those aims and navigate the Section 232 pharmaceutical tariff framework, the Department of Commerce has issued no additional guidance on tariff implementation or exempted products, leaving biotechnology companies increasingly uncertain about the impact of Section 232 tariffs as the deadline for implementation moves closer.

    On May 13, Commerce released a Federal Register Notice (FRN) outlining how manufacturers can apply for company‑specific “onshoring agreements” that can reduce tariff burdens under the recent Presidential Proclamation, “Adjusting Imports of Pharmaceuticals and Pharmaceutical Ingredients Into the United States.” These agreements will determine whether a company faces tariff rates as high as 100% on certain imported pharmaceutical products or qualifies for reduced rates of 20%—or 0% for companies that also have a Most Favored Nation (MFN) pricing deal.

    Commerce is requiring companies to submit onshoring plan applications by June 12. With that deadline fast approaching, companies have a narrow window to prepare applications. Yet key questions regarding eligibility, scope, and implementation of these agreements remain unresolved. Below are several areas where greater clarity is needed.

    Which products will be exempt?

    Perhaps the most pressing question biotech companies need answered: Which products will be subject to tariffs, and which will qualify for exemptions?

    The FRN doesn’t provide any additional guidance on exemptions under the recent Presidential Proclamation. As a result, companies are being asked to submit detailed information about their products, sourcing, and supply chains without certainty about which products are even in scope. For many firms, this uncertainty could compound the burden of compiling documentation to submit to Commerce.

    That would be a significant operational challenge under any timeline. But with applications due this week, it could be especially difficult. Companies are also considering concerns about the transmission and handling of potentially large volumes of business-sensitive information.

    How will tariffs apply to complex supply chains?

    The FRN implies that input materials for patented pharmaceuticals will be subject to tariffs—regardless of whether those inputs are themselves patented. But it offers little clarity on how tariffs will apply when patented and non-patented products share the same manufacturing inputs, or when imported ingredients flow across multiple products with different regulatory or tariff classifications.

    Most biotech companies don’t manufacture a single product in a single facility using a single set of inputs. They operate across interconnected global supply chains involving multiple suppliers, manufacturing partners, and production stages spanning different countries.

    Commerce’s current framework leaves these companies with no clear roadmap for how to account for that complexity.

    How are pre‑commercial biotech products impacted?

    The FRN doesn’t clarify whether pre‑commercial products—or the ingredients used to develop them—will be subject to tariffs. Finished pre-commercial drug products may be exempt under the Harmonized Tariff Schedule of the United States. But the input materials purchased commercially to develop and manufacture those products in the United States may still be subject to tariffs. This potential exposure disproportionately impacts U.S.-based innovators, with particularly serious implications for emerging biotech companies.

    Smaller biotech firms often spend years conducting research, running clinical trials, and navigating the FDA approval process before earning revenue. They rely heavily on outside investment and typically operate with limited cash reserves—about 35% of biotech firms currently have less than a year’s worth of cash on hand. Without clarity on tariff exposure, these companies cannot reliably plan manufacturing strategies, attract investors, or assess the viability of developing new therapies.

    What counts as an onshoring commitment?

    Commerce has yet to clearly define what constitutes a “successful onshoring commitment.” Furthermore, the administration hasn’t clarified the inclusion of questions regarding MFN pricing deals in applications for onshoring agreements.

    Without more explicit guidance, companies are forced to guess what constitutes a sufficient investment. Smaller innovators with extremely limited capital are unsure whether purchasing new equipment for a contract manufacturing partner qualifies. Meanwhile, established companies with significant existing domestic footprints are left wondering what additional benchmarks are required to secure the same tariff relief offered to those building their first domestic facilities.

    Can companies realistically meet the timeline?

    Building new or modernizing existing biopharma manufacturing facilities is a complex, lengthy process. BIO members consistently report that shifting contract manufacturing facilities—which are relied upon by nearly 90% of biotechnology companies—can take up to eight years for approved medicines. Constructing new facilities can cost up to $2 billion and require 5-10 years to complete. Even routine manufacturing transitions require extensive regulatory review.

    Yet companies are now being asked to submit detailed onshoring plans within 30 days—while major implementation questions remain unanswered.

    The Bottom Line

    U.S. biotechnology companies are facing major business, investment, and supply chain decisions as the June 12 deadline rapidly approaches—and ongoing uncertainty isn’t conducive to innovation.

    BIO will continue engaging with the administration and Congress to develop policies that strengthen the domestic biopharma manufacturing base in a strategic, sustainable way and support the innovators working to bring new therapies to patients.

    The post Section 232 tariff uncertainty remains high as onshoring plan deadline looms appeared first on Bio.News.

    ]]>     AI Reimagines Caffeine as a Molecular Off‑Switch for Engineered Cells https://edusehat.com/en/ai-reimagines-caffeine-as-a-molecular-offswitch-for-engineered-cells https://edusehat.com/en/ai-reimagines-caffeine-as-a-molecular-offswitch-for-engineered-cells Tue, 09 Jun 2026 10:05:10 +0700 Edusehat Reimagines, Caffeine, Molecular, Off‑Switch, for, Engineered, Cells A cup of coffee can mean many things: a daily part of our routine, a moment of calm, a midday boost. But zoom in past the steam, past the roasted aromatics, down to the caffeine molecule itself, and a different story emerges. At the Texas A&M Health Institute of Biosciences and Technology, researchers have turned this everyday stimulant into something far more unexpected: a molecular “pause button” for engineered cells.

    In a study published in the Journal of the American Chemical Society (JACS), the team unveiled CODS, a caffeine‑operated dissociation system built using AI‑guided de novo protein design. The paper, “AI‑Guided De Novo Design of a Caffeine‑Induced Protein Dissociation System,” describes how the group reprogrammed “an existing caffeine-responsive chemically induced proximity (CIP) module into a ligand-dependent dissociation system.”

    “AI is changing how we design biology,” said senior author Yubin Zhou, MD, PhD. “Instead of relying only on protein parts that already exist in nature, we can now design new mini proteins with specific behaviors. Here, we used AI to help turn caffeine into a precise trigger for controlling engineered cells.”

    caffeine molecular switch
    A team of Texas A&M Health researchers led by Yubin Zhou, MD, PhD, is using caffeine to precisely control engineered cells, a step toward safer and more responsive therapies. [Texas A&M University]
    The CODS system pairs a caffeine‑binding protein with a synthetic mini‑binder designed using the BindCraft platform. In the absence of caffeine, the two components stay locked together. Add caffeine, and the complex snaps apart, releasing the binder and shutting down the attached cellular function. As Tianlu Wang, PhD, a postdoctoral fellow in the Zhou lab, put it, “Many genetically-encoded molecular tools act like accelerators. CODS gives us something closer to a brake or pause button.”

    The team demonstrated CODS across several biological contexts. In engineered gene circuits, caffeine addition sharply reduced transcriptional activity. In a rewired pyroptosis pathway, caffeine triggered inflammatory cell death by freeing the active domain of gasdermin D. And in perhaps the most translational example, CODS served as a conditional deactivator for CAR T cells, temporarily dampening their activity without destroying the therapeutic cells.

    “Powerful therapies need powerful control,” Zhou said. “By combining AI‑designed proteins, high‑performance computing, and familiar small molecules, we are building a new language for communicating with engineered cells.”

    The design process itself leaned heavily on computation. Graduate student Brendan McKee led the AI‑guided binder design and molecular modeling, while Tatsuki Nonomura spearheaded the molecular engineering and live‑cell validation. The Texas A&M High Performance Research Computing service provided the infrastructure needed to run large‑scale simulations. “High‑performance computing was essential for this project,” Zhou noted. “It helped us move from a conceptual idea to a functional molecular switch much faster.”

    Although caffeine is not a therapeutic molecule, its safety and familiarity make it an appealing control signal. As Zhou emphasized, “Coffee will not replace medicine. But caffeine can help us imagine medicines that are more controllable, more responsive, and safer for patients.” The researchers’ next steps include further testing in therapeutic cells, animal models, and disease-relevant settings before moving toward clinical use.

    CODS now joins a growing toolkit of AI‑designed molecular switches, offering a blueprint for future systems responsive to other safe, accessible molecules. As programmable cell therapies advance, the ability to modulate them with something as simple as caffeine may prove unexpectedly powerful.

    The authors report that a patent application covering the CODS platform has been filed by Texas A&M University, with Y.Z., T.N., B.M., and T.W. listed as inventors (U.S. Provisional Patent Application No. 64/022,078).

    The post AI Reimagines Caffeine as a Molecular Off‑Switch for Engineered Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Defective HIV RNA Linked to Persistent Viremia During Long-Term ART https://edusehat.com/en/defective-hiv-rna-linked-to-persistent-viremia-during-long-term-art https://edusehat.com/en/defective-hiv-rna-linked-to-persistent-viremia-during-long-term-art Tue, 09 Jun 2026 10:05:09 +0700 Edusehat Defective, HIV, RNA, Linked, Persistent, Viremia, During, Long-Term, ART Antiretroviral therapy (ART) has enabled most people living with HIV to live long and healthy lives. However, a small portion of people experience detectable traces of the virus, known as nonsuppressible viremia (NSV), despite strict adherence to long-term treatment regimens and the absence of symptoms. The results of a study headed by researchers at Johns Hopkins University School of Medicine now suggest that most cases of NSV are explained by defective and noninfectious copies of the virus.

    The study, which involved more than 50 people, found that while traces of HIV-1 RNA can persist in blood after optimal therapy, cases of non-suppressible viremia are driven by HIV-1 RNA with defects in a piece of the RNA known as 5’-leader. The team developed a digital PCR (dPCR) assay, CLAWS (Capturing 5′ Leader Anomalies Without Sequencing), that distinguishes intact from defective 5′L RNA.

    “From a clinical perspective, this is important because people with HIV are taught that the absolute goal of their medication is to achieve undetectable viral load, and they worry,” said Francesco R. Simonetti, MBChBD, PhD, an assistant professor of medicine in the Division of Infectious Diseases at Johns Hopkins University School of Medicine. The new findings, said Simonetti and his team, should provide relief to many people living with HIV who fear a viral rebound or who are concerned about transmitting the virus to partners despite taking effective treatment.

    Simonetti is senior and corresponding author of the team’s report in Nature Communications (“5′ leader defects drive persistent HIV-1 viremia on long-term ART”), in which they stated, “These findings identify 5′L-defective genomes as the predominant driver of NSV and establish CLAWS as a practical tool for monitoring viremia in clinical and cure-related settings.”Modern antiretroviral therapies, which date back to 1996, prevent HIV from infecting new populations of immune system cells, but aren’t able to retroactively prevent previously infected cells from releasing HIV viral particles. Since those cells usually represent a small portion of infected cells after a person is on stable therapy, most people with HIV (PWH) who take antiretroviral therapies are able to bring their viral loads to clinically undetectable levels in their blood.

    However, in some cases, which are estimated to occur less than 1% of the time, people may experience clinically detectable levels after taking long-term antiretroviral drug therapy. “Traces of HIV-1 RNA can persist in plasma despite long-term suppressive antiretroviral therapy (ART), the authors stated. This could happen years later, or, in less frequent cases, they may have never achieved undetectable levels. “The sources of NSV remain poorly defined, in part due to limited tools to characterize plasma HIV-1 RNA,” the team continued. “NSV raises concerns for virologic failure, transmission, and immune activation, complicates ART management, causing anxiety and ultimately affecting the quality of life of PWH.”

    For the newly reported study, the investigators examined blood samples from 52 people living with HIV who had detectable loads of the virus despite taking long-term antiretroviral drug therapy. These samples, which were assessed from 32 people and compared to an additional 20 samples, were collected between 2021 and 2025. The majority of participants were white men, between ages 58 and 68, and received care in the United States, Canada, and Denmark. The researchers found that most detectable forms of the virus, around 95%, were due to defective copies, and most defects were due to mutations or deletions in the 5’-leader region of HIV-1 RNA. This region is known to orchestrate the production of copies of the virus, but in this case, the defects prevented the generation of infectious virus.

    “In 31 participants from the original NSV cohort and an additional 20 participants from the validation cohort, RNA transcribed from defective proviruses accounted for a median of 95% of plasma HIV-1 RNA, firmly establishing their central role in persistent viremia,” the investigators wrote in summary.

    The study offers evidence that clinicians can now study the virus in blood plasma and confirm if clinically detectable levels are due to defective copies released from one or a few T-cell clones, said Simonetti. If so, he added, this could eliminate the need for extra medications and could prevent related complications. It could also help people living with HIV have access to surgeries or other procedures, such as hip or knee replacements or organ transplants, and participate in clinical studies if they know they have HIV under control.

    “We know that these defective proviruses cannot infect new cells, but they are still clinically relevant,” said Simonetti. “Think of how many extra visits, extra drugs, extra costs, and tests they’ve been causing. It’s also clear from the new study that, over time on treatment, intact proviruses that make virus are pruned away, while defective ones escape the immune system,” he said. “Now we want to understand these differences in immune recognition to uncover HIV’s vulnerabilities.”

    Similar to using a liquid biopsy to detect cancer mutations in DNA, the CLAWS assay developed by the researchers uses advanced technology to identify detectable viral loads that are due to defective copies. The method is cost-effective and can be broadly used in HIV clinics and research settings.

    In their paper, the authors wrote in conclusion, “In summary, our results establish 5’L-defective proviruses as the major source of NSV and introduce CLAWS as a practical tool for dissecting persistent viremia. Beyond clarifying mechanisms of HIV-1 persistence, CLAWS provides immediate translational utility for clinical monitoring and HIV-1 cure research.”

    The post Defective HIV RNA Linked to Persistent Viremia During Long-Term ART appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Chikungunya Vaccine Development in Africa Accelerated by ACT-CHIK https://edusehat.com/en/chikungunya-vaccine-development-in-africa-accelerated-by-act-chik https://edusehat.com/en/chikungunya-vaccine-development-in-africa-accelerated-by-act-chik Tue, 09 Jun 2026 06:30:09 +0700 Edusehat Chikungunya, Vaccine, Development, Africa, Accelerated, ACT-CHIK Institut Pasteur is launching ACT-CHIK (Accelerating Clinical Trials for CHIKungunya Vaccine in Africa), a four-year research project funded by the Global Health EDCTP3 Joint Undertaking under the European Union’s Horizon Europe program that aims to advance clinical trials and prepare for the manufacturing of a chikungunya vaccine in Africa.

    With €15.3 million in EU funding, ACT-CHIK will advance the development of MV-CHIK—a measles-virus-based chikungunya vaccine originally developed at Institut Pasteur—through a large-scale Phase Ib/III clinical trial in four African countries, while preparing for technology transfer to an African vaccine manufacturer.

    “Chikungunya remains a neglected disease in Africa despite its growing burden. ACT-CHIK represents a unique opportunity to generate critical clinical data in the populations that need this vaccine most, while simultaneously building the foundation for regional vaccine manufacturing on the continent,” notes Sotiris Missailidis, DPhil, ACT-CHIK project coordinator at Institut Pasteur.

    Chikungunya is a mosquito-borne viral disease transmitted by Aedes aegypti and Aedes albopictus mosquitoes. It causes debilitating symptoms, including high fever, severe joint pain that can persist for months or even years, headache, rash, and fatigue. Over the past two decades, the number of chikungunya cases reported across Africa has risen sharply. Yet, the disease remains largely underdiagnosed and under-reported, particularly in regions where multiple arboviruses and malaria co-circulate. Climate change is further expanding the range of mosquito vectors, increasing the risk of outbreaks across the globe, and most notably in Africa.

    Although chikungunya vaccines have recently become available, their use remains limited largely among travelers, with cost and access constraints hindering their deployment in endemic regions. The MV-CHIK candidate is designed to be accessible to populations in endemic areas and aims to support local production. This positioning will address a major gap in equitable access to vaccination and to strengthen outbreak preparedness in regions where the need is greatest.

    The MV-CHIK vaccine is a live-attenuated, recombinant vaccine using the well-established measles virus Schwarz vaccine strain as a vector—a platform technology originally developed at the Institut Pasteur in Paris. Six Phase I and II clinical trials conducted in Europe, the United States, and Puerto Rico, including approximately 600 adult participants in total, have demonstrated satisfactory safety, tolerability, and immunogenicity profiles.

    Building on these results, ACT-CHIK will conduct a Phase Ib/III multicenter, international clinical trial to evaluate the safety and immunogenicity of MV-CHIK in adults, adolescents, and children living in Rwanda, Kenya, Nigeria, and Senegal. By enrolling 940 participants across both endemic and non-endemic areas, the trial will generate essential data to advance the clinical development plan for African populations, including younger age groups.

    Beyond clinical evaluation, the project has a strategic manufacturing dimension. ACT-CHIK will conduct comprehensive due diligence, gap analysis, and prepare for the technology transfer of the MV-CHIK vaccine manufacturing process to the Institut Pasteur de Dakar (IPD), Africa’s only WHO-prequalified vaccine manufacturer. Fundação Oswaldo Cruz (Fiocruz) in Brazil, a fellow member of the Pasteur Network, will prepare the clinical trial materials and contribute its extensive vaccine manufacturing expertise to the technology transfer process.

    The project will also develop a regulatory pathway for the licensure of the MV-CHIK vaccine in Africa through engagement with national regulatory authorities and the World Health Organization prequalification teams, to obtain prequalification.

    ACT-CHIK directly supports Africa’s ambition—as set by the African Union—to produce 60% of the continent’s vaccine needs locally by 2040, and is aligned with the European Union’s Team Europe Initiative on Manufacturing and Access to Vaccines (TEI MAV+).

    “ACT-CHIK will mobilize the full breadth of expertise at Institut Pasteur de Dakar: from clinical trials to cutting-edge virology and immunology laboratories, from vaccine research to manufacturing expertise. This project embodies our vision: an Africa that develops, evaluates, and produces its own vaccines—for the populations that need them most,” notes Ibrahima Socé Fall, PhD, CEO of Institut Pasteur de Dakar.

    The ACT-CHIK consortium brings together seven partner institutions with complementary expertise:

    • Institut Pasteur (Paris, France) — Project coordinator; developer of the MV-CHIK vaccine platform
    • University of Rwanda (Kigali, Rwanda) — Scientific project leadership; clinical trial site
    • Institut Pasteur de Dakar (Dakar, Senegal) — Vaccine technology transfer recipient; clinical laboratory assays; clinical trial site
    • Fundação Oswaldo Cruz – Fiocruz (Rio de Janeiro, Brazil) — Clinical trial material manufacturing (fill & finish); technology transfer support
    • Irrua Specialist Teaching Hospital (Irrua, Nigeria) — Clinical trial site; Coordinating Principal Investigator
    • Kenya Medical Research Institute – KEMRI (Nairobi, Kenya) — Clinical trial site; Lead of data dissemination and communication
    • International Vaccine Institute – IVI (Seoul, South Korea) — Clinical trial sponsor, regulatory strategy, and capacity building

    The post Chikungunya Vaccine Development in Africa Accelerated by ACT-CHIK appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     CiteSentinel Launched to Detect and Prevent AI Hallucinations in Legal Citations https://edusehat.com/en/citesentinel-launched-to-detect-and-prevent-ai-hallucinations-in-legal-citations https://edusehat.com/en/citesentinel-launched-to-detect-and-prevent-ai-hallucinations-in-legal-citations Tue, 09 Jun 2026 02:55:12 +0700 Edusehat CiteSentinel, Launched, Detect, and, Prevent, Hallucinations, Legal, Citations Legal tech startup BrentWorks reports that it has launched CiteSentinel, a dedicated platform built specifically to detect and prevent AI hallucinations in legal citations, including those related to biotechnology. The tool scans legal documents and flags case law, statutes, and legal authorities that may be fabricated, misstated, or otherwise erroneous, before they reach a judge, according to the company.

    Courts across the country are increasingly sanctioning attorneys who submit briefs containing invented case citations, a well-documented byproduct of generative AI drafting tools that produce authoritative-sounding, but entirely fictional, legal authority, says BrentWorks co-founder Brent Britton, a technology attorney and MIT-trained engineer. CiteSentinel was designed to close that verification gap, giving attorneys a fast and easy way to confirm that every citation in a filing corresponds to a real case, a real statute, and a real legal authority, he adds.

    “The legal profession is learning, in very public ways, that AI doesn’t just make mistakes, it confidently lies to your face,” continues Britton. “CiteSentinel is about restoring trust. It lets lawyers move fast with the irresistible efficiencies of generative AI while still filing documents reciting authorities they can stand behind. It also enables them to scan opposing counsel’s documents, giving them a competitive edge in the courtroom.”

    AI hallucinations
    When a brief containing fabricated citations reaches the court, the question of who drafted it quickly becomes secondary to the question of whose name is on it. [BestForBest/Getty Images]
    Many attorneys who do not personally use AI to draft documents are discovering they have a problem anyway, Britton points out. Opposing counsel may have used AI. Co-counsel may have. Contract attorneys and paralegals almost certainly have access to it and may be using it without disclosing that fact. When a brief containing fabricated citations reaches the court, the question of who drafted it quickly becomes secondary to the question of whose name is on it, he explains.

    CiteSentinel lets attorneys scan any document, their own, a colleague’s, or an adversary’s, for citation errors before those errors become their problem, notes Britton. Attorneys who review opposing counsel’s filings with CiteSentinel gain an additional advantage: the ability to identify and challenge citations to authorities that simply do not exist, he says.

    Unlike traditional research platforms that focus on finding more information, states Britton, CiteSentinel was created to confirm that the law cited in a document is real. Attorneys can scan:

    • Their own AI-assisted drafts, before filing
    • Submissions from co-counsel, contract attorneys, and support staff
    • Opposing counsel’s filings, for strategic advantage
    • Any document where citation accuracy carries professional or ethical weight

    BrentWorks’ other co-founder is Brent Hunter, a technologist who applied neural networks to finance in 1993. He cites CiteSentinel as the first in a series of products the company will be releasing for the practice of law in the age of AI.

    Both BrentWorks’ co-founders agree that AI hallucinations pose particular risks in biotechnology-related legal matters because cases often depend on highly technical evidence, including patent claims, prior art, clinical trial data, FDA regulatory history, scientific publications, expert witness testimony, freedom-to-operate analyses, and licensing agreements. In this context, an AI system could invent scientific references that do not exist, mischaracterize FDA guidance documents, fabricate patent precedents, incorrectly summarize clinical trial results, or generate inaccurate prior-art searches. Such errors can undermine legal arguments, regulatory submissions, and intellectual property strategies.

    “Biotech litigation is where AI hallucinations turn genuinely dangerous. You have a system trained to sound authoritative now injecting phantom patent precedents and counterfeit clinical data into documents that determine whether a drug reaches patients or a patent survives challenge,” explains Brent Britton. “In this domain, where the technical record is everything, a ghost FDA guidance document or a fabricated prior art reference can unravel an entire legal strategy and years of work along with it. The law has always been a high-stakes information game, and right now the machines are playing it with synthetic cards.”

    As CiteSentinel expands beyond just case citation verification, “it will be the truth layer that keeps all players honest,” he predicts.

     

    The post CiteSentinel Launched to Detect and Prevent AI Hallucinations in Legal Citations appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     CRISPR Shreds Undruggable Cancer Cells with Precision https://edusehat.com/en/crispr-shreds-undruggable-cancer-cells-with-precision https://edusehat.com/en/crispr-shreds-undruggable-cancer-cells-with-precision Mon, 08 Jun 2026 23:20:08 +0700 Edusehat CRISPR, Shreds, Undruggable, Cancer, Cells, with, Precision When Jingkun Zeng, PhD, joined the lab of Nobel laureate, Jennifer Doudna, PhD, as a postdoctoral researcher in 2024, he was not interested in applying CRISPR for gene editing.

    The molecular scissors had demonstrated extraordinary clinical promise in correcting single-point mutations, most strikingly in Baby KJ’s case, where a rare metabolic disorder once presented a 50% mortality rate in infancy.  

    Yet, Zeng had his ambitious sights on stopping cancer progression, where the biology “became messy.” Cancer can be driven by hundreds of thousands of mutations, making it nearly impossible to correct each mutation one-by-one to restore healthy function. 

    Zeng, who completed his PhD training in cancer evolution at The Francis Crick Institute, aimed to develop new CRISPR-based technology that could therapeutically access the undruggable tumor suppressor protein, p53. Mutations in this “guardian of the genome” are found in nearly half of all cancers, and up to 70–90% of cases of the most deadly tumors, including ovarian, pancreatic, and non-small cell lung cancer. 

    In a new study published in Nature titled, “Targeting Cancer-Specific Mutations with RNA-Triggered Chromatin Shredding,” Zeng and colleagues from Innovative Genomics Institute (IGI), University of California (UC) Berkeley, UC San Francisco (UCSF), and Gladstone Institutes, have now engineered a CRISPR system to selectively trigger cancer cell death by chromatin shredding. 

    The approach recognizes cancer cells using the RNA-guided nuclease, CRISPR-Cas12a2, to recognize mutant p53 mRNA transcripts. Therapeutic effectiveness was demonstrated in mouse models of lung and liver tumors. 

    Bacterial roots 

    Mutations in p53 are early drivers in the cancer-causing cascade, making the tumor suppressor one of the most sought-after targets in cancer therapy. Yet despite decades of effort, no approved p53 drugs exist on the market. 

    Unlike many druggable proteins, p53 lacks a well-defined binding pocket traditionally required by established modalities, such as small molecules or antibodies. Additionally, most cancer therapeutics are designed to inhibit disease-driving proteins, whereas restoring p53 function demands precise, controlled activation of a tumor suppressor. 

    “It’s the first time we managed to target p53 with such precision,” Zeng told GEN, emphasizing that CRISPR-Cas12a2 can distinguish healthy and disease cells that differed by just one nucleotide.

    The novel drug modality takes advantage of CRISPR’s bacterial roots as a defense system that protects against infection by cutting the genetic material of invading viruses, preventing replication and spread.

    Zeng also emphasizes that the guide RNA is easily programmable for additional therapeutic areas, such as destroying viral infected cells or abnormal cells due to aging. The technology can also be multiplexed to recognize multiple cancer mutations simultaneously.

    The work joins a growing industry effort to develop scalable and generalizable genetic medicines. 

    Looking ahead, the authors aim to improve the delivery efficiency to cancer cells, a longstanding challenge across CRISPR therapies. The team is also undergoing collaborations to apply the technology across diverse cancer types, including brain, prostate, and ovarian cancer. 

    The post CRISPR Shreds Undruggable Cancer Cells with Precision appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Lawmakers warn against weakening IP for medicines in House hearing https://edusehat.com/en/lawmakers-warn-against-weakening-ip-for-medicines-in-house-hearing https://edusehat.com/en/lawmakers-warn-against-weakening-ip-for-medicines-in-house-hearing Mon, 08 Jun 2026 19:45:03 +0700 Edusehat Lawmakers, warn, against, weakening, for, medicines, House, hearing Lawmakers defended strong IP protections as essential to biotech innovation that saves lives in a June 4 Congressional hearing that also considered the role of generics and biosimilars.

    The same day that the Supreme Court ruled that a generic drug maker was not infringing patent rights in the Hikma case, witnesses and lawmakers argued that weakening patents only harms innovation without impacting the cost of prescription drugs in a hearing before the House Judiciary Subcommittee on Courts, Intellectual Property, Artificial Intelligence, and the Internet.

    “Strong patent protections are why the United States has been a global leader in the discovery and development of lifesaving medications, with more than one-half of all new drugs invented in the United States,” said Subcommittee Ranking Member Hank Johnson (D-GA) in his prepared opening statement.

    Other lawmakers in the hearing spoke of the importance of the innovation that is enabled by IP.

    “We’re actually living at a time that’s incredibly exciting for drug discovery with timelines for discovery and testing rapidly accelerating,” said Rep. Kevin Kevin Kiley (R-CA) “I think there’s a lot to be optimistic about.”

    Rep. Deborah Ross (D-NC) reminded the hearing that she represents North Carolina’s Research Triangle, a hub for biomedical innovation.

    “We have small scrappy startups putting it all on the line for the potential to find transformative cures, as well as large companies that leverage their resources to invest in the next blockbuster medicine,” Ross said. “Underlying all of this innovation and the hundreds of billions of dollars in economic activity it generates is our nation’s robust patent system. Strong intellectual property protections are the foundation that that success is built on.”

    Regulation of generics and IP

    The hearing included discussion of generic drugs and proposed changes to IP legislation that would weaken patent protections to favor generic drugs. Rep. Ross said the current system of laws regulating IP on prescription drugs is a thoughtful, bipartisan creation that encourages innovation while enabling generic drugs and does not need to be changed.

    Witness Jamie Simpson, Chief Policy Officer and Counsel of the Council for Innovation Promotion, agreed that the system does much to enable generics.

    “The Hatch-Waxman Act, for example, took generics from roughly 13% of the market in 1983 to 90% today—it has been remarkably successful in bringing lower-cost alternatives to patients, but it strikes a thoughtful balance in doing so, and Congress should be careful not to upset that balance,” according to Simpson’s written testimony.

    As witness Krita Carver noted, the 90% market share for generics makes the U.S. an outlier, with the average generic market share in Organization for Economic Co-operation and Development (OECD) countries being 41%.

    Rep. Kiley argued that innovation precedes imitation. “If there was no new innovator who created the underlying drug, what would be the opportunity for a generic to come along?” he asked.

    Threats to IP protections

    The hearing addressed the idea of multiple patents on drugs. Critics have called multiple patents “patent thickets,” arguing that they are used to prevent generic competition. A proposal mentioned during the hearing, the ETHIC Act, would single out legally valid drug patents and render them unenforceable, while allowing similar patents on other technologies to remain enforecable. It would make it harder for innovators to protect their IP in court.

    Subcommittee Chair Darrell Issa (R-CA) and Justice Committee Ranking Member Jamie Raskin (D-MD) expressed concerns about “patent thickets.” Witnesses Michael Carrier of Rutgers Law School and Rachel Goode of Fresenius Kabi promoted the ETHIC Act as a solution, but other lawmakers and witnesses explained the legitimate need for multiple patents.

    Ranking Member Johnson said multiple patents are used when researchers develop improvements to drugs after those drugs receive Food and Drug Administration (FDA) approval.

    “Reliable protections also encourage companies to continue to invest in improving existing treatments, because medical invention should not end when a drug reaches the market,” he said.

    Another proposal, The Skinny Labels, Big Savings Act, which would weaken existing legal protections against induced infringement, was promoted by its sponsors, Rep. Ben Cline (R-VA) and Rep. Zoe Lofgren (D-CA). Simpson explained how the proposed changes would undermine legitimate legal protections for innovators.

    Simpson noted that the Congressional Budget Office (CBO) released a 2024 study into several proposals to accelerate generic and biosimilar market entry. “It concluded that each would reduce average drug prices only marginally—between 0.1% and 1.0%, or in some cases less than 0.1%, in 2031,” said her written statement.

    Simpson warned about the impact of weaker IP protections. She said measures reducing IP protections create opportunities for other countries, notably China, to take away U.S. biotech leadership.

    A written statement submitted for the hearing by the Biotechnology Innovation Organization (BIO) offered a similar point of view.

    “Our patent system serves the important function of fostering innovation and U.S. global leadership in the life sciences. This continued leadership is being challenged by our foreign competitors, especially the People’s Republic of China. Further weakening patent protections would aid our foreign competitors at the expense of this strategically important sector,” said BIO’s statement.

    The post Lawmakers warn against weakening IP for medicines in House hearing appeared first on Bio.News.

    ]]>     StockWatch: Abivax Survives a Roller Coaster Week https://edusehat.com/en/stockwatch-abivax-survives-a-roller-coaster-week https://edusehat.com/en/stockwatch-abivax-survives-a-roller-coaster-week Mon, 08 Jun 2026 08:55:09 +0700 Edusehat StockWatch:, Abivax, Survives, Roller, Coaster, Week More acutely than most companies, Abivax (Euronext Paris and Nasdaq: ABVX) felt firsthand the ups and downs of the proverbial stock market roller coaster this past week as investor fears over safety signals associated with the French biotech’s late-stage ulcerative colitis (UC) drug candidate briefly overshadowed its positive Phase III clinical results.

    Shares of Abivax skidded on June 2, the first trading day after the company announced positive topline results from the Phase III ABTECT maintenance trial (NCT05535946), which is assessing obefazimod in adults with moderately to severely active UC.

    Abivax’s stock plummeted after analysts from Jefferies, Morgan Stanley, Truist Securities, and Wedbush Securities raised as a safety concern a portion of data showing malignancies in nine patients among the 580 enrolled in the study. Jefferies reacted the strongest among the firms, downgrading Abivax’s stock rating from “Buy” to “Hold” and lowering its 12-month price target on the company’s shares 44%, from $160 to $90.

    “Cancer signal complicates matters,” Faisal Khurshid, a managing director and equity research analyst with Jefferies, wrote in a research note. “Promise of the drug is to deliver strong efficacy w/ oral convenience and w/out JAK safety baggage. However, cancer cases seen in maintenance break our thesis. Even if proven to be not drug-related or v[ery] low incidence, we expect an overhang to investor interest, strategic optionality, and commercial uptake.”

    Wedbush analyst David Nierengarten upgraded Abivax on the overall ABTECT data, from “Underperform” to “Neutral,” though he lowered the firm’s target price 18%, from $110 to $90. But he also cautioned, according to published reports, that the data increase the risk of obefazimod being approved with a black box warning on its label.

    The other two firms lowered their price targets as well:

    • Morgan Stanley (Judah Frommer)—Down 9% from $145 to $132, maintaining its “Overweight” rating.
    • Truist (Greg Renza)—Down 4% from $140 to $135, maintaining its “Buy” rating.

    Of the nine patients diagnosed with malignancies, seven received the higher 50 mg dosage of obefazimod, one the lower 25 mg dose, and one placebo. Squamous cell carcinoma cases were seen in three of the patients—one dosed at 25 mg, the others 50 mg. Basal cell carcinoma cases emerged in two 50 mg patients and one placebo patient, while cases of prostate cancer, breast cancer, and colonic dysplasia were seen in one patient each, all dosed at 50 mg.

    In its announcement of the ABTECT data, Abivax stated that the prostate, breast, and colon cancer cases “were considered unrelated to treatment by investigators.” Among the squamous cell and basal cell cases, two of the four 50 mg patients were deemed “not/unlikely related to drug,” while of the remaining two cases, one had a medical history of skin cancer, the company added.

    Swinging negative, then positive

    Yet the cancer and pre-cancerous cell cases nevertheless led investors to sell off their Abivax shares on June 2. Abivax’s ordinary shares traded on Euronext Paris plummeted 44% from €111.80 ($128.81) to €63.10 ($72.70), while the company’s American depositary shares (ADSs) traded on the Nasdaq Global Market also plunged 44% from $129.69 to $72.50.

    No sooner did Abivax shares start to free-fall, however, than a more positive narrative emerged among another set of analysts. They cited the ABTECT maintenance study’s overall positive findings, which showed that both doses of the first-in-class miR-124 enhancer met the study’s primary endpoint by showing positive absolute clinical remission rates of 50.8% for the 25 mg dose and 51.3% for the 50 mg dose. Both results blew away the 10.4% absolute clinical remission rate of placebo.

    When adjusted for placebo, obefazimod showed placebo-adjusted clinical remission rates of ∆39.3% for the 25 mg dose and ∆40.3% for the 50 mg (both p<0.0001).

    Obefazimod also met all of ABTECT’s key secondary endpoints, including endoscopic improvement, endoscopic remission, histologic-endoscopic mucosal improvement  (HEMI), corticosteroid free clinical remission, and sustained clinical remission.

    In endoscopic remission, Abivax showed in an investor presentation, obefazimod showed placebo-adjusted endoscopic remission rates of 38% (50 mg) and 31% (25 mg), outperforming nine marketed drugs that carry indications in UC—which ranged from 8% by both Omvoh® (mirikizumab-mrkz) marketed by Eli Lilly (NYSE: LLY) and  Skyrizi® (risankizumab-rzaa) marketed by AbbVie (NYSE: ABBV), to 20% shown by AbbVie’s Rinvoq® (upadacitinib), Tremfya® (guselkumab) marketed by Johnson & Johnson (NYSE: JNJ), and Velsipity® (etrasimod) marketed by Pfizer (NYSE: PFE).

    “While most therapies cluster in the 8% to 20% range, obefazimod stands alone at 31% and 38%, demonstrating a level of efficacy that is well beyond the range achieved by other treatment classes. This finding is particularly important because achieving endoscopic remission has been consistently associated with lower relapse rates and better long-term patient outcomes,” Abivax CEO Marc de Garidel said June 1, addressing analysts during a conference call held to discuss the ABTECT study results.

    He also cited Abivax’s Phase IIa/IIb open-label extension study (NCT05177835), designed to assess the long-term safety and the efficacy profile of 25 mg obefazimod given once daily in subjects previously enrolled in two earlier Phase II trials, ABX464-102 (NCT03368118) or ABX464-104 (NCT04023396). Data from the extension study announced in May showed that patients receiving 50 mg of obefazimod for two to four years and then transitioned to 25 mg for up to an additional three years maintained durable clinical remission and a favorable safety profile for up to seven years of treatment exposure.

    Abivax said it plans to submit a New Drug Application (NDA) submission to the FDA for obefazimod late in the fourth quarter.

    “Transformational potential”

    “Taken together, we believe these results validate the transformational potential of obefazimod and position us well as we advance toward our planned NDA submission in late 2026,” de Garidel said, declaring: “Today’s results firmly establish obefazimod as a potential new standard of care for the treatment of ulcerative colitis.”

    Thomas J. Smith, senior managing director, immunology and metabolism, and a senior research analyst with Leerink Partners, appeared to agree with de Garidel in a research note.

    “We believe obe[fazimod]’s results suggest a best-in-disease maintenance profile, especially among oral therapeutics in UC,” Smith wrote in his initial research note on Abivax’s ABTECT data. “We continue to believe that obe features the most compelling late-stage clinical profile for a novel oral agent in IBD [inflammatory bowel disease] amid heightened M&A/BD [business development] activity that underscores large pharma’s interest in the space.”

    Several big-money merger-and-acquisition (M&A) deals in recent years have centered around pharma giants buying developers of drugs for UC, Crohn’s disease, and other forms of IBD. The biggest of these was Merck & Co. (NYSE: MRK) acquiring Prometheus Biosciences for $10.8 billion in 2023, while Lilly bought out Morphic Therapeutic for about $3.2 billion in 2024; Roche Holding (SIX Swiss Exchange: ROP and RO; OTCQX: RHHBY) snapped up Telavant Holdings for up-to-$7.25 billion in 2023; and Pfizer acquired Arena Pharmaceuticals for $6.7 billion in a deal completed in 2022.

    Abivax itself has found itself rumored as a potential candidate for being acquired—it made GEN’s A-List of Top 10 Takeover Targets of 2026—since last summer, when the company reported dazzling data from two Phase III trials, ABTECT-1 (ABX464-105; NCT05507203) and ABTECT-2 (ABX464-106; NCT05507216).

    As for the reported malignancies, Smith commented in a follow-up research note: “We do not view the reported malignancies as an outsized or approvability-limiting safety risk, particularly given the absolute low event counts, investigator assessment that the non-NMSC malignancies were unrelated to treatment, IDMC [independent data monitoring commission] adjudication supporting the safety profile, lack of organ-specific clustering, and mitigating circumstances noted across each case.”

    Obefazimod is a small molecule upregulator of miR-124, an anti-inflammatory microRNA. It enhances the selective splicing of a single long noncoding RNA to generate miR-124, which downregulates cytokines and chemokines shown to promote inflammation, including tumor necrosis factor (TNF) alpha, IL-6, monocyte chemoattractant protein-1 (MCP-1), and IL-17, as well as Th17+ cells.

    Under its former name ABX464, obefazimod was initially developed against HIV but was repurposed to fight inflammatory conditions based on its anti-inflammatory effect.

    “Best in indication”

    Also bullish on Abivax is Citizens JMP Securities, where Jason Butler, managing director, biotechnology equity research, raised his firm’s price target 43%, from $131 to $187, based on ABTECT’s positive data exceeding expectations. Butler maintained Citizens JMP’s “Market Outperform” rating on Abivax shares.

    “We view efficacy (~40% placebo-adjusted clinical remission rate) as best in indication, easily surpassing drugs across all approved classes of UC therapies,” Butler wrote. “We believe the Phase [III] efficacy and safety results, together with its oral once-daily profile, support that obefazimod can be transformational to the UC treatment landscape, benefiting both earlier-stage and more severe/refractory patients.”

    “Importantly, we also view the safety profile as compelling and are comfortable that the drug is not associated with a clear malignancy risk (which is the primary debate driving stock volatility post-market),” Butler added.

    The more positive feedback from analysts apparently swayed investors, as Abivax’s stock price rebounded roughly 40% on both exchanges since the initial nosedive. The Nasdaq shares vaulted 24% to $90.15 on Wednesday, then rose another 16% to $104.93 Thursday before dipping 3% Friday, finishing the week at $101.53 and with a 23.5% five-day decline.

    On Euronext Paris, Abivax shares bounced back 13% to €71.25 ($82.09) on Wednesday, jumped another 18% Thursday to €83.95 ($96.72), then finished Friday up 5% to €87.85 ($101.21) and a 22.5% five-day decline.

    Butler said the maintenance study’s data suggested that Abivax could potentially generate more positive clinical results for obefazimod in its ongoing Phase IIb ENHANCE-CD induction trial in Crohn’s disease (NCT06456593), which is expected to read out in mid-2027. He added that ABTECT’s results were strong enough to support approval of both the 25 mg and 50 mg doses: “While we anticipate malignancies to be included in the label, we continue to believe in the differentiated profile of obefazimod.”

    Speaking with analysts, de Garidel agreed with pursuing approvals for obefazimod at both 25 mg and 50 mg: “Our thinking is that both doses will be very helpful for patients, and we plan to file at year-end with those two doses for maintenance.”

    Leaders and laggards

    • Absci (Nasdaq: ABSI) shares climbed 24% from $5.94 to $7.34 Thursday after Leerink Partners initiated coverage of the generative AI-based drug developer with an “Outperform” rating and a 12-month price target of $12. Mani Foroohar, MD, senior managing director, genetic medicines, and a senior research analyst with Leerink, lauded the company’s drug mechanism of blocking prolactin (PRL)–prolactin receptor (PRLR) signaling as seen in its lead pipeline candidate ABS-201, an anti-prolactin receptor antibody in Phase I/II studies for androgenetic alopecia that advanced into the clinic last year, with a Phase II study in endometriosis expected to start in the fourth quarter: “We see a totality of evidence supporting PRLR blockade in these indications, and see even partial/early clinical validation opening a clear path to valuation >double the current market cap (~$1.1B fully diluted),” Foroohar wrote in a research note.
    • Fulcrum Therapeutics (Nasdaq: FULC) shares jumped 9.5% from $3.37 to $3.69 Friday after the developer of small molecule drugs for rare blood disorders disclosed in a regulatory filing that it will slash its workforce approximately 85%—from 57 to nine full-time employees—in a cost-cutting move expected to be substantially completed during the second quarter. Fulcrum said it expects to incur approximately $4.2 million in charges related to the layoffs, consisting primarily of employee severance, employee benefits, and related costs. Fulcrum vowed to “significantly” reduce its operating expenses and launched a strategic review after scrapping its lead pipeline program to develop pociredir as a treatment for sickle cell disease (SCD). The development followed the FDA expressing heightened concerns about pociredir’s risks and benefits in fighting SCD due to an unexpectedly high rate of secondary blood cancers seen with another PRC2 inhibitor, Tazverik®(tazemetostat), marketed by Ipsen (Euronext Paris: IPN).
    • Oculis Holding (Nasdaq: OCS) shares tumbled 36% from $22.70 to $14.51 June 1, after the Swiss neuroophthalmology and ophthalmology drug developer said it did not plan to pursue an FDA filing seeking approval for its OCS-01 eye drops in diabetic macular edema (DME). Oculis acknowledged that OCS-01 failed two Phase III trials, DIAMOND-1 (NCT05066997) and DIAMOND-2 (NCT06172257), by missing their primary endpoint, mean change in best corrected visual acuity early treatment diabetic retinopathy study (BCVA ETDRS) letter score at Week 52. The key secondary endpoint of the proportion of patients with ≥15-letter gain in BCVA was not met in both trials, though another secondary endpoint, retinal thickness as measured by OCT, showed a “substantial and persistent” reduction with OCS-01 vs vehicle at all visits in DIAMOND-2, and at all visits except Week 52 in DIAMOND-1.

    The post StockWatch: Abivax Survives a Roller Coaster Week appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Bio-Techne, Refeyn Partner on Workflow for Bispecific Antibody, Biosimilar Characterization https://edusehat.com/en/bio-techne-refeyn-partner-on-workflow-for-bispecific-antibody-biosimilar-characterization https://edusehat.com/en/bio-techne-refeyn-partner-on-workflow-for-bispecific-antibody-biosimilar-characterization Sat, 06 Jun 2026 05:40:13 +0700 Edusehat Bio-Techne, Refeyn, Partner, Workflow, for, Bispecific, Antibody, Biosimilar, Characterization This week, Bio-Techne, a provider of life science tools, reagents, and diagnostic product, and Refeyn, a pioneer in mass photometry technology, announced the launch of an integrated workflow for characterizing charge and size variants in bispecific antibodies and biosimilars. 

    The workflow combines Bio-Techne’s R&D Systems MauriceFlex™ imaged capillary isoelectric focusing (icIEF) fractionation system with Refeyn’s TwoMP mass photometry platform. The combined solution makes it possible for researchers to directly correlate charge heterogeneity with molecular weight and aggregation at single‑molecule resolution in four hours.

    “Bispecifics are the fastest growing segment within next‑generation antibodies, but they are very difficult to characterize,” said Gerry Mackay, CEO of Refeyn. That difficulty is due to the structural complexity of these molecules. Incomplete characterization can delay development, increase manufacturing risks, and lead to costly late-stage failures. “This approach directly addresses one of the biggest challenges by combining icIEF fractionation with mass photometry. Researchers can now interrogate charge and size variants together in a single workflow,” he said. 

    Within the workflow, charge variants are first separated using the MauriceFlex system. They are then analyzed on Refeyn’s TwoMP platform. The system requires a nanogram‑level sample and reveals size distribution and aggregation at single‑molecule resolution. 

    Together, the technologies enable direct characterization of aggregation and size within icIEF-resolved charge variants, something which is not accessible with standalone methods. It reduces reliance on multiple tests and supports faster, more efficient process development. 

    These capabilities are a boon for customers, according to Will Geist, president of Bio‑Techne’s Protein Sciences Segment. “Enabling deeper characterization with less sample helps them reduce risk, control costs, and make better decisions earlier in development.” 

    The post Bio-Techne, Refeyn Partner on Workflow for Bispecific Antibody, Biosimilar Characterization appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Twin Prime Editing Enables Rapid Trait Stacking in Crops https://edusehat.com/en/twin-prime-editing-enables-rapid-trait-stacking-in-crops https://edusehat.com/en/twin-prime-editing-enables-rapid-trait-stacking-in-crops Sat, 06 Jun 2026 05:40:12 +0700 Edusehat Twin, Prime, Editing, Enables, Rapid, Trait, Stacking, Crops Researchers working to advance genome engineering in crops face many challenges, including simultaneously introducing diverse genome edits. Although a major goal of modern crop breeding is to efficiently combine multiple desirable traits by “stacking” the favorable alleles that contribute to those traits in a single crop variety, current strategies are time-consuming and inefficient.

    Now, a team led by Caixia Gao, PhD, professor at the Institute of Genetics and Developmental Biology of the Chinese Academy of Sciences, has developed a genome engineering platform that allows multiple trait stacking in crops by combining gene knockout, precise sequence editing, and chromosome engineering within a single framework. The advance is “a twin prime editing-based knockout (TKO) system that installs stop codon clusters (SCCs) for precise translational termination with minimal in-frame mutations.” TKO achieved knockout efficiencies of up to 70.5%, 58.6% and 75.1% in rice, maize, and wheat protoplasts, respectively.

    This work was published in Nature Biotechnology in the article, “Multiplexed, precise genome engineering in monocots with twin prime editing systems.”

    The researchers first developed a precise and efficient gene knockout tool called twin prime editing (twinPE)-mediated gene knockout (TKO), which precisely inserts a small fragment containing a stop codon cluster at the target site. TKO achieves predictable gene disruption through precise installation of stop codons, avoiding in-frame indels caused by insertions or deletions in multiples of three nucleotides, which are often seen in Cas9 systems.

    In protoplasts, TKO demonstrated efficient knockout capabilities in monocot crops such as rice, wheat, and maize. In regenerated T0 rice plants, the average efficiency for single gene knockout reached 96.8%.

    To eliminate cross-editing between different loci and to achieve precise, safe multiplex gene knockout, the researchers developed 10 orthogonal TKO systems, enabling efficient simultaneous knockout of up to 10 genes. Unlike Cas9-mediated multiplex editing, which can lose effectiveness because in-frame mutations accumulate across multiple targets, the orthogonal TKO systems maintain high knockout efficiency even when multiple genes or homologous gene copies are edited simultaneously.

    Building on TKO, the researchers then developed two integrated genome engineering platforms, TRIM1 and TRIM2—forming a unified platform known as TRIM.

    TRIM1 combines TKO with prime editing-based sequence modification, enabling simultaneous gene knockout, base substitution, insertion, deletion, duplication, and inversion within a single editing framework. In regenerated T0 rice plants, TRIM1 achieved simultaneous knockout of one gene together with homozygous precise editing of three additional targets with an efficiency of 22.8%.

    TRIM2 incorporates a prime editor–Cre recombinase fusion protein and enables kilobase-scale DNA insertion, replacement, deletion, inversion, and chromosomal translocation through recombinase-assisted genome engineering.

    Unlike existing genome editing tools that typically perform only a limited number of sequence modifications, TRIM integrates gene knockout, small-scale precise sequence editing, and large-scale chromosome engineering into a single platform. This “all-in-one” platform provides a powerful way to rapidly stack multiple favorable alleles, thus enhancing precision breeding of complex traits in monocot crops.

    The post Twin Prime Editing Enables Rapid Trait Stacking in Crops appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     How Germinal Centers Generate Antibodies Through Noisy Rounds of Mutation and Selection https://edusehat.com/en/how-germinal-centers-generate-antibodies-through-noisy-rounds-of-mutation-and-selection https://edusehat.com/en/how-germinal-centers-generate-antibodies-through-noisy-rounds-of-mutation-and-selection Sat, 06 Jun 2026 05:40:11 +0700 Edusehat How, Germinal, Centers, Generate, Antibodies, Through, Noisy, Rounds, Mutation, and, Selection A study tracking thousands of B cells across more than 100 germinal centers (GCs) in mice has revealed how the system consistently produces highly effective antibodies. The findings overturn longstanding ideas about how germinal centers function, revealing that they are far more selective than once thought, and challenge the idea that antibody improvement is driven mainly by rare growth “bursts” among the most successful B cells. The discovery could have implications for immune cell evolution, and ultimately guide the design of vaccines against rapidly mutating pathogens like influenza. It could also lead to new ways of studying evolution itself.

    “The traditional, mechanistic view of germinal centers is to think of them as selection machines sorting out the best antibodies,” said research lead Gabriel D. Victora, PhD, head of the Laboratory of Lymphocyte Dynamics at The Rockefeller University. “But when you look very, very closely, you see a process that’s almost essentially random—a little bit better than a coin toss—which repeats many times until the immune system arrives at the right answer consistently. That’s much more akin to how evolution operates than the way a machine does.”

    Victora and colleagues reported on their findings in Cell, in a paper titled “Replaying germinal center evolution on a quantified affinity landscape.”

    Inside germinal centers, B cells rapidly mutate and compete to produce antibodies that bind successively better to pathogens. “Darwinian evolution of immunoglobulin genes within germinal centers (GCs) underlies the progressive increase in antibody affinity following antigen exposure,” the authors wrote. That puts B cells under intense pressure to optimize a single trait: binding affinity, or how well an antibody recognizes its target.

    But how they accomplish that feat has very much remained an open question, the team noted. “Whereas the cellular mechanics of how competition between B cells increases affinity are well established, the evolutionary dynamics of this process are less clear.” Because weak and strong B cells often coexist side by side in the germinal center, scientists have long wondered whether the immune system temporarily preserves weaker cells in case they later acquire useful mutations. The phenomenon of clonal bursts, in which the descendants of a single B cell rapidly take over an entire germinal center, are also poorly understood.

    The authors explained that GC B cells evolve by rapidly mutating only two Ig genes, which are the heavy chain (Igh) and light chain (either Igk or Igl). Victora’s team engineered mice in which all competing B cells began with the same antibody sequences, allowing them to replay a single evolutionary process across more than 100 germinal centers at once. “… we established a system in which GCs are composed entirely of B cells carrying the same pre-rearranged Igh and Igk genes, ensuring identical starting specificity and affinity,” they explained. Victora added, “We simplified it to the bare bones, and asked how repeatable is the exact sequence of mutations that leads to stronger antibodies.”

    Once each of the B cells was primed with the exact same unmutated antibody sequence, the team triggered germinal center formation through immunization. They then tracked the resulting sprint toward immune efficiency with multiphoton microscopy and laser-based photoactivation, and sequenced thousands of individual B cells across 119 germinal centers.

    With this data, the team managed to construct a detailed family tree that mapped how different lineages of B cells had developed. They also built a mutational dictionary, using deep mutational scanning (DMS), a technique that links almost every possible amino-acid change to antibody performance. This advance allowed the team to determine how mutations affected binding strength and structural stability simply by reading a cell’s DNA sequence.

    “DMS was the big technical advance here,” says first author Ashni Vora, PhD, a graduate fellow in the lab. “With it we could determine the affinities of thousands of cells just by looking at their sequence, without having to produce an antibody.”

    The researchers compare the resulting picture to a casino game. Watching a single B cell evolve inside a germinal center looked almost random, with some cells rapidly expanding, others disappearing, and even promising mutations failing as if random chance ruled the day. Some germinal centers were overtaken by clonal bursts while others contained many competing lineages with no clear winner. The differences had little to do with affinity or merit. “We find that, even in this simplified setting, GC selection yields widely divergent tree topologies, ranging from clonal-burst-type structures to multi-pronged GCs where multiple line ages evolve in parallel,” they noted.

    But the team discovered that the germinal center game is rigged. In a casino, the house always wins not because of the odds on any individual game, but because a slight statistical bias is built into the system and repeated thousands of times. Germinal centers appear to operate similarly. Each round of cellular competition is only slightly biased toward cells carrying beneficial mutations, and random chance means that there is often little correlation between affinity and success. But by repeating that same noisy, almost random process over and over across many germinal centers, the immune system ultimately produces stronger antibodies.

    “If you see someone get a jackpot, you might wonder how the casino makes money,” Victora says. “The answer is that the casino puts in a little bit of bias, so that you win and you lose, but on average, you lose more than you win. If there are just one or two people playing, the casino might lose money due to random chance. But if there are a thousand people playing, it’s going to average out and the house wins. That’s essentially how germinal centers work.”

    The researchers also found that the immune system favors mutations that are easiest for its cellular machinery to generate, rather than the mutations that would produce the strongest antibodies. And by tracking B cell lineages over time, they also showed that germinal centers are far more selective than previously thought, rapidly eliminating inferior B cells. “By combining phylogenetic reconstructions with a fitness landscape inferred from populations sampled over time, we show that both the apparent permissiveness of GCs to low-affinity lineages and the apparent early plateau in affinity maturation are best explained by survivorship biases that distort the histories of lineages present at sampling,” the investigators wrote in summary.

    Taken together, the findings overturn several longstanding ideas about how germinal centers function and may provide new tools for vaccine developers hoping to steer antibody evolution against influenza and HIV. “What was once theoretical speculation about what must happen in the germinal center, we are now showing in action—the real thing,” Victora says.

    At the same time, this work also illustrates how germinal centers could become a powerful model for studying evolution more broadly. Scientists have long relied on bacteria grown in the lab over many generations to plumb the depths of evolutionary biology and determine how much of evolution is driven by random chance. In clarifying the rules governing germinal centers, the researchers revealed why the immune system could offer a potentially more tractable experimental avenue: Unlike bacterial evolution, which centers around adapting to many possible survival strategies, B cells are all aiming for the same target. “I see this as an opening salvo in a longer effort to understand evolution by using the immune system as a model,” Victora added.

    The post How Germinal Centers Generate Antibodies Through Noisy Rounds of Mutation and Selection appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     A Billion-Dollar Deal, Trial Trouble, Biohub Updates, and Vaccine Research News https://edusehat.com/en/a-billion-dollar-deal-trial-trouble-biohub-updates-and-vaccine-research-news https://edusehat.com/en/a-billion-dollar-deal-trial-trouble-biohub-updates-and-vaccine-research-news Sat, 06 Jun 2026 01:55:09 +0700 Edusehat Billion-Dollar, Deal, Trial, Trouble, Biohub, Updates, and, Vaccine, Research, News In this week’s episode, we start with news that Fulcrum Therapeutics is scrapping its lead pipeline program for sickle cell disease following concerns from the FDA about the drug’s risks and benefits. Also on the docket, news of Eli Lilly’s latest spending. The pharma giant has added its genetic medicines pipeline and capabilities by signing a $1.9 billion agreement with Ascidian Therapeutics to develop RNA exon editors for treating inherited kidney diseases. Next up, updates to an open-source model for binder design and protein function mapping. Finally, on the peer-review front, we dive into some of the latest vaccine research including insights into the mechanisms that cross-reactive T cells use to target multiple viral species in a single family and an improvement to standard polio vaccines.

     

     

    Listed below are links to the GEN stories referenced in this episode of Touching Base:

    Fulcrum Halts Development of SCD Candidate Pociredir, Sets Strategic Review
    By Alex Philippidis and Kevin Davies, PhD, GEN Edge, June 2, 2026

    Lilly, Ascidian Launch Up-to-$1.9B RNA Exon Editor Collaboration Targeting Inherited Kidney Diseases
    By Alex Philippidis, GEN Edge, June 3, 2026

    Biohub Releases Protein Biology World Model to Address Disease
    By Fay Lin, PhD, GEN Edge, May 27, 2026

    Cross-Reactive T Cells Could Point to Broad Vaccines or Treatments for Measles, Nipah Virus
    GEN, June 2, 2026

    Experimental Adjuvant Could Strengthen Mucosal Immunity with Injectable Polio Vaccines 
    GEN, June 4, 2026

    Touching Base Podcast
    Hosted by Corinna Singleman, PhD

    Behind the Breakthroughs
    Hosted by Jonathan D. Grinstein, PhD


    Produced with support from:

    skpharmteco logo

    The post A Billion-Dollar Deal, Trial Trouble, Biohub Updates, and Vaccine Research News appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Are AI chatbots making us lose control of our brains? https://edusehat.com/en/are-ai-chatbots-making-us-lose-control-of-our-brains https://edusehat.com/en/are-ai-chatbots-making-us-lose-control-of-our-brains Fri, 05 Jun 2026 22:15:03 +0700 Edusehat Are, chatbots, making, lose, control, our, brains
  • Attention spans are in freefall. Psychologist Gloria Mark found that average attention spans dropped from two and a half minutes in 2003 to just 47 seconds by 2020—and the constant switching is directly linked to rising stress levels.
  • AI may be making our brains lazy. When we outsource writing, summarizing, and evaluating to tools like ChatGPT, we skip the "depth of processing" that helps us actually learn and think critically—and those cognitive muscles can atrophy from disuse.
  • Even our emotional intelligence is at risk. AI companions require none of the effort that real relationships demand, and Mark warns that if current trends continue, loneliness, purposelessness, and emotional decline will only deepen.
  • The fix is effort, not abstinence. Mark isn't calling for a tech ban—she's calling for intentionality: read the book, skip the GPS, meet friends in person. The harder the task, she says, the greater the reward.
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    This week I’ve been at SXSW London. There’s been music, film, and a lot—and I mean a lot—of talk about AI. I also had the opportunity to sit down with Gloria Mark, a psychologist at the University of California, Irvine, who has spent the last 30 years studying how people interact with digital technologies.

    Early in her career, the biggest concerns were the potential impacts of internet and email use on our brains. We may laugh those concerns off today, but it’s true that as the technologies became more ubiquitous and ingrained in our daily lives, our attention spans began to shrink.

    Mark is worried that things are only getting worse. The title of our session was “Have we lost control of our brains?” Unfortunately, Mark told me, the answer is yes.

    Around two decades ago, Mark started wondering about how our use of devices might affect our attention spans. She set up what she calls “living laboratories,” using sensors and trackers to monitor adult volunteers’ attention, mood, and behavior when they were using devices.

    In 2003, she found that the average user had an attention span of around two and a half minutes. That’s how long people could spend focused on one thing before moving on to something else. “That surprised me at the time,” she told me during our session on Wednesday. “I thought: Wow, this is really short.”

    But when she repeated the experiment in 2012, she found that attention spans had shrunk—all the way down to around 75 seconds on average, she said. In research she conducted between 2014 and 2020, attention spans shrank further still—to a mere 47 seconds, on average. Yikes.

    And it’s not good for us. Mark told me that she’s found switching our attention so frequently is stressful. “We would have people wear heart rate monitors, and … we would see direct correlation between switching attention fast and stress going up,” she told me.

    All this distraction makes it harder for us to get stuff done, too. “It just takes longer to do any single task if you’re switching your attention,” she told me. “It’s not great for performance. It’s not great for our emotional well-being.”

    And that’s for adults. What about the effects of digital technologies on children? A few months ago, Meta (which owns Facebook and Instagram) and Google’s YouTube were ordered to pay millions of dollars in damages to a 20-year-old woman who had accused the companies of creating products that led her to develop a childhood addiction.

    Just a couple of weeks ago, Meta settled another lawsuit, this one brought by a rural school district in Kentucky. The district had also accused the company of designing addictive products that were harmful to students and had sought more than $60 million to cover the costs of their mental-health needs. Around 1,200 other school districts are taking similar legal action against social media companies.

    But social media isn’t all bad, all the time. It can provide opportunities for some people, including those from marginalized groups, to form connections that might otherwise be difficult. A 2024 survey of LGBTQ+ teenagers found that while some described social media as a place of rejection and fear, others described it as a place where they felt a sense of belonging, where they could develop friendships and cultivate their identity.

    In truth, we can’t definitively say what effects using social media is having on children across the board, says Mark. “There have been lots and lots of studies, and the evidence is to date inconclusive,” she told me. (Despite what you might read in best-selling books on the subject.)

    Mark is hopeful that large, long-term studies might finally start shedding a bit more light on this question. An effort of this nature is underway in Australia, which enacted a social media ban for under-16s at the end of last year.

    Given this uncertainty over a 20-year-old technology, I wondered if Mark had any thoughts on the potential impacts of AI—an obviously much newer offering that within the space of a couple of years appears to have become deeply integrated into our digital lives.

    She told me she’s worried.

    When we put in effort to do something—such as evaluating or summarizing content—we’re doing what’s known as “depth of processing,” she told me. “When you’re actively engaged with information, you’re processing it on a very deep level,” she said. “Then you’re more likely to learn it, to understand it, [and] to retain it.”

    That’s not happening when most people use AI bots like ChatGPT, Claude, and Gemini. When we ask these tools to write, summarize, or evaluate for us, we’re no longer doing that depth of processing. “You’re deferring your cognitive work to AI,” she said. “And it’s not good for us.”

    The risk is that our cognitive abilities will weaken over time. “If you’re not constantly exercising your muscles, they can atrophy,” Mark said. “And that’s exactly what can happen with our minds.” People with weaker critical thinking skills are more likely to fall prey to misinformation, she added.

    Interactions with AI-powered “synthetic companions” can be just as harmful. Relationships between human beings take work—time, effort, and understanding. None of that is needed if you’re forming a relationship with a sycophantic bot. The “muscle” we risk atrophying here is emotional intelligence, which surveys suggest is already on the decline, said Mark.

    She’s not painting a particularly rosy picture.

    “If we continue on this trajectory, attention spans are diminished, loneliness is rising, boredom is rising, emotional intelligence decreasing, and actually our sense of purpose, according to studies, is also decreasing,” she said.

    Luckily, she thinks we can course-correct by changing our relationship with these technologies. The key factor is effort.

    The more effort we put into something, the deeper the satisfaction we stand to gain, Mark told me. That means making an effort to read a book rather than skimming its summary, and to meet with friends in person when you can. Try not to use GPS in places where you can probably manage without it.

    “I love technology; we can’t give it up,” she told me. “[But] we have to learn how to create new life routines.”

    This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.

    ]]>     Brain-Targeted Drug Discovery Barriers Drive Deep Science Ventures and Medicines Discovery Catapult Deal https://edusehat.com/en/brain-targeted-drug-discovery-barriers-drive-deep-science-ventures-and-medicines-discovery-catapult-deal https://edusehat.com/en/brain-targeted-drug-discovery-barriers-drive-deep-science-ventures-and-medicines-discovery-catapult-deal Fri, 05 Jun 2026 22:10:09 +0700 Edusehat Brain-Targeted, Drug, Discovery, Barriers, Drive, Deep, Science, Ventures, and, Medicines, Discovery, Catapult, Deal Deep Science Ventures (DSV) and Medicine Discovery Catapult (MDC) agreed to collaborate to address challenges in delivering medicines into the brain.

    One of medicine’s greatest challenges is ensuring that treatments reach the precise area of the body where they are needed. While recent scientific breakthroughs have identified numerous targets for neurological conditions, the difficulty of effectively transporting these treatments across the blood-brain barrier and into the central nervous system (CNS) remains a primary challenge for global health.

    According to the World Health Organization’s Global Status Report on Neurology, over 40% of the global population is living with CNS diseases, making them a leading global cause of ill health and disability.

    Directly addressing critical gaps in healthcare means these innovations have the potential to improve patient outcomes while creating clinical and commercial opportunities for biotech and pharma companies. Developing new solutions could unlock access for rare neurological disorders and expand treatment to large or underserved patient populations, including those with diseases such as Alzheimer’s, Parkinson’s, and various brain cancers.

    The first phase of the partnership will see an in-depth review of the current medicines landscape conducted to identify opportunities for innovation. This information will then be used to identify systemic gaps in brain-entry technologies. The long-term ambition is for novel approaches that meet the investment criteria of the partners to be spun out into new ventures focused on high-impact solutions and to provide them with pre-seed funding.

    Alzheimers research
    Credit: Minerva Studio/Getty Images

    A core part of DSV’s approach involves building future founding teams to form new companies that will address challenges across multiple sectors. Future founders will work on opportunities that have been pre-scoped by DSV, de-risking the standard founder proposition.

    By combining DSV’s venture-building model with MDC’s drug discovery expertise and infrastructure, the partnership will aim to develop new approaches to ensure life-changing medicines reach the brain, according to Adam Tomassi-Russell, senior director, DSV.

    “The blood-brain barrier remains one of the most complex issues in modern medicine and with over 40% of the world’s population facing neurological conditions, it’s imperative that we find an optimal solution to this problem,” said Tomassi-Russell. “By pooling our venture-creation expertise with MDC’s discovery capabilities, we can offer the right founders a frictionless environment in which to tackle the CNS delivery gap. If we can solve the ‘how’ of brain entry more effectively, we can unlock a new frontier of CNS therapeutics and address the huge unmet need in these diseases.”

    “At MDC, we are committed to transforming bold ideas into better treatments,” added Nicola Heron, chief strategy officer, MDC. “This collaboration presents an opportunity to discover new technologies that could have a significant impact on patients and society. Through this partnership, we will strengthen the ecosystem for CNS innovation in the U.K. and beyond, enabling more medicines to reach patients faster.”

     

    The post Brain-Targeted Drug Discovery Barriers Drive Deep Science Ventures and Medicines Discovery Catapult Deal appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Novel Intracellular Pathway Identified That Protects Against Viral and Bacterial Infection https://edusehat.com/en/novel-intracellular-pathway-identified-that-protects-against-viral-and-bacterial-infection https://edusehat.com/en/novel-intracellular-pathway-identified-that-protects-against-viral-and-bacterial-infection Fri, 05 Jun 2026 11:25:09 +0700 Edusehat Novel, Intracellular, Pathway, Identified, That, Protects, Against, Viral, and, Bacterial, Infection A common concept of the immune system is that of white blood cells putting up a fight against invading pathogens in the bloodstream. Researchers have now detailed a separate but equally important route by which our bodies fight infection—directly inside already infected cells. The team, co-led by Leo James, PhD, and Tyler Rhinesmith, PhD, at MRC Laboratory of Molecular Biology, defined a previously undescribed method of fighting pathogen invaders—and which they called  “antibody-directed xenophagy” (ADX)—where cells can digest bacteria and viruses, including Salmonella and adenoviruses, that cross the cell membrane. The scientists found that regulation of ADX is dependent on the intracellular protein, TRIM21, which James’s lab had previously shown protects from viral infection by binding to antibody-coated viruses in the cell cytosol, triggering virus degradation.

    “People have talked about viral xenophagy before as a sort of concept, but if you look in literature, there aren’t any good examples where people have shown this operating to potently block infection,” said James. “In our single study, we’ve gone from the discovery of something completely unknown [ADX], all the way through molecular mechanism, its function in cells into animals, and demonstrated physiological importance.”

    The discovery of the ADX pathway may have potential future medical implications. While far more study is needed, the research points to the feasibility that antibody or small molecule therapeutics could be used to treat infections by marking pathogens in the blood so TRIM21 can recognize and jumpstart ADX once they enter cells.

    James, Rhinesmith, and colleagues reported on their findings in Molecular Cell, in a paper titled “TRIM21 induces selective autophagy of viruses and bacteria,” stating, “We propose that TRIM21 evolved through competition with pathogens to induce autophagy of diverse and complex substrates, potentially explaining its versatility for targeted protein degradation.”

    Typically, the body will respond to an infection by creating antibodies that latch onto the invaders in the blood to alert immune cells, such as white blood cells, to destroy them. Sometimes, those antibody-bound pathogens evade the immune cells and infect healthy cells. This is where antibody-directed xenophagy becomes involved.

    Using CRISPR-Cas9 and quantitative imaging, the team determined that once an antibody-labeled pathogen enters a cell, ADX begins with the specialized protein TRIM21, which flags the pathogen with a ubiquitin marker that signals to the cell that it has been invaded.

    TRIM21 is an intracellular E3 ubiquitin ligase protein that binds to antibodies and catalyzes ubiquitination. Prior work by James’s group had found that TRIM protects against viral infection by binding to antibody-coated viruses in the cell, triggering ubiquitination and viral degradation.

    “Recently, we and others have shown that the degradative adaptability of TRIM21 extends to a wide range of additional substrates beyond viral capsid proteins,” the team further pointed out. “TRIM21 is an exceptionally versatile ubiquitin ligase that can be directed by antibodies to target oligomeric protein scaffolds, viral capsids, and proteopathic aggregates for intracellular degradation.”

    However, the mechanism used by cells to degrade the tagged viruses wasn’t known. “… how such a large and complex substrate is quickly and efficiently degraded remains unclear.”

    Rhinesmith, a post-doc in James’s group, conducted a genome-wide CRISPR-Cas9 knockout screen, individually removing every gene across the human genome and testing how its deletion impacted TRIM21-triggered degradation of viruses. The results were striking, revealing a previously undescribed process by which TRIM21 is able to trigger autophagy of cell-invading viruses.

    Autophagy is a conserved cellular process through which damaged or toxic cellular components are delivered to specialist acidic organelles to be degraded and recycled. While this process plays a key role in maintaining cellular health, its ability to protect against invading viral pathogens hasn’t been well studied.

    Staff scientist Anna Albecka developed a high-fidelity confocal microscopy platform that allowed the team to visualize previously unidentified events in the TRIM21 restriction mechanism. The team observed binding of TRIM21 to antibody-coated viruses inside cells, in real time. The microscopy results showed that after TRIM21 ubiquitinates the invading virus complex, ubiquitin stimulates the assembly of autophagy components around viruses, including LC3, a marker for membranous compartments called autophagosomes.

    Working with Claudia Puri and David C. Rubinsztein at the U.K. Dementia Research Institute, Cambridge, the team used super-resolution microscopy to visualize the assembly of these autophagosome membranes around individual viral particles coated in antibodies and TRIM21. Together, these observations revealed the stepwise process by which incoming virions are incarcerated inside sealed, LC3-positive autophagosomes.

    Albecka was further able to show that these virus-containing autophagosomes are ultimately delivered to acidic lysosomes, resulting in the degradation of each virus into harmless peptides and nucleotides. Significantly, the study suggests that antiviral autophagy is a highly effective strategy deployed by cells to protect themselves from infection, and provides new tools for investigating this process.

    Inspired by the ability of TRIM21 to activate by clustering around clients of very different architectures, the team next sought to understand whether it could also intercept a completely different type of pathogen: bacteria. The team used antibodies and a novel live cell microscopy method to track bacterial growth inside mouse cells. They observed the same ADX pathway that intercepts viral infection also potently restricts the growth of intracellular Salmonella. This discovery is significant because it explains how TRIM21 is able to intercept and trigger the degradation of invading pathogens of many complex structures and diverse lineages. “Importantly, our data explain how TRIM21 can degrade large and highly complex substrates,” the authors stated. “The need to intercept and destroy phylogenetically and structurally diverse pathogens may have driven the evolution of TRIM21’s very broad substrate versatility.”

    By leveraging the intrinsic flexibility of the autophagy pathway, ADX can adapt to and degrade a variety of large and difficult targets. The findings indicate that the cell does not require a bespoke defense strategy for every individual pathogen. Instead, it employs a universal strategy, reliant on TRIM21, to redirect the cell’s existing autophagy machinery to any harmful material tagged with antibodies. This adaptability makes ADX clinically important for human immunity and, excitingly, a potential target for therapeutic enhancement.

    “TRIM21 is unique because it uses the antibodies attached to the invading virus or bacteria to alert the cell,” said James. Rhinesmith added, “We show in the paper that on top of non-enveloped viruses, it’s also able to target bacteria along the same pathway. It seems that you trigger ubiquitination of whatever pathogen has antibodies around it through TRIM21, and this is the key step that leads to autophagy of the bacteria or the virus.”

    This ability for cells to fight back from the inside doesn’t appear limited to specific cells within our body. The research team tested for the presence and action of TRIM21 against adenovirus in a range of human cell lines, as well as living mouse models in the case of Salmonella. These experiments indicated that ADX-mediated immunity is likely ubiquitous throughout the human body. “TRIM21 is expressed from what we call an ‘interferon-stimulated gene,’ which means that it is upregulated during infection, so your body makes it all the time, everywhere,” said James. “And the reason why you make it everywhere is so that you can potentially protect any cell or tissue.”

    Though ADX may sound like a backup for our immune system for when pathogens evade our first lines of defense, the authors noted that this could be an equally important primary mode of protective immunity. “Our data shows that without TRIM21, a significant component of protective immunity in vivo against viruses is lost. In practice, immunity works because we’ve got different mechanisms operating together,” James said.

    TRIM21 is the first intracellular protein discovered to stimulate ADX immunity, but there may be others that have equally broad or specific pathogen targets. Part of the research team’s next steps is determining the existence of other ADX-stimulating proteins and what limitations there may be to TRIM21’s function.

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    ]]>     D&amp;D‑seq Uses Base Editing to Map DNA–Protein Interactions in Single Cells https://edusehat.com/en/ddseq-uses-base-editing-to-map-dnaprotein-interactions-in-single-cells https://edusehat.com/en/ddseq-uses-base-editing-to-map-dnaprotein-interactions-in-single-cells Fri, 05 Jun 2026 11:25:08 +0700 Edusehat D&D‑seq, Uses, Base, Editing, Map, DNA–Protein, Interactions, Single, Cells A new molecular recording strategy is giving researchers a way to capture DNA–protein interactions in single cells, including the weak and transient contacts that shape gene regulation but often slip past existing assays. The method, called D&D‑seq (docking and deamination followed by sequencing), layers a base‑editing enzyme onto an antibody‑binding nanobody, turning fleeting interactions into durable sequence marks.

    The paper is titled “Single-cell mapping of regulatory DNA-protein interactions,” and was published recently in Cell.

    “D&D-seq couples an antibody-binding nanobody to a cytosine base editor, a combination that enables detection of weak or transient factor binding through targeted cytosine-to-uracil [C→U] editing at protein-bound genomic sites,” the authors wrote. Those edits become a molecular breadcrumb trail, revealing where regulatory proteins have interacted with the genome.

    This approach directly addresses a long‑standing gap in the field. Traditional methods for mapping transcription factor binding, such as ChIP‑seq or CUT&RUN, “cannot be easily incorporated into high-throughput single-cell workflows, limiting applications to bulk analysis or to single-cell profiling of only the strongest interacting chromatin factors. Single-cell profiling of TF binding in primary samples has been mainly restricted to inferential approaches based on expression levels of downstream TF target genes or through motif analysis of assay for transposase-accessible chromatin using sequencing (ATAC-seq) peaks, but identification of specific TF-binding sites requires more direct methods,” according to the authors.

    The team demonstrated that D&D‑seq can map binding sites for transcription factors and other regulatory proteins, like chromatin remodeling proteins, across multiple cell types and conditions. One application involved profiling CTCF binding in primary T cells carrying an IDH2 mutation commonly found in leukemia. Because D&D‑seq operates at single‑cell resolution, it exposes heterogeneity in regulatory wiring that is often masked in population‑level assays.

    Crucially, the method is platform‑agnostic. The authors showed that D&D‑seq can be integrated into standard single‑cell multiomics workflows, including ATAC‑seq, scATAC‑seq, and whole‑genome sequencing. That compatibility allows researchers to pair DNA–protein interaction maps with chromatin accessibility, gene expression, and genomic variation—all within the same cell.

    As transcription factors and other regulatory proteins increasingly emerge as therapeutic targets, tools that reveal how these factors behave in patient‑derived cells will be essential. D&D‑seq offers a way to monitor how mutations, drugs, or engineered perturbations reshape regulatory landscapes at single‑cell resolution.

    “We’re entering an era of medicine in which transcription factors and other gene-activity regulators will increasingly be therapeutic targets,” said Dan Landau, MD, PhD, the Bibliowicz Family professor of medicine and a member of the Sandra and Edward Meyer Cancer Center and the Englander Institute for Precision Medicine at Weill Cornell, who is also an oncologist at NewYork-Presbyterian/Weill Cornell Medical Center. “This kind of technology should have an important role in developing and evaluating such therapies.”

    Although the method is still evolving, its conceptual elegance and technical flexibility have already sparked broad interest. By turning DNA into a recording surface for protein activity, D&D‑seq opens a new window into the “regulome”—one that captures the subtle, transient interactions that drive cellular identity and disease.

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    ]]>     Stipple Bio and Lonza Agree to Focus on Advancing Oncology ADC Therapies https://edusehat.com/en/stipple-bio-and-lonza-agree-to-focus-on-advancing-oncology-adc-therapies https://edusehat.com/en/stipple-bio-and-lonza-agree-to-focus-on-advancing-oncology-adc-therapies Fri, 05 Jun 2026 07:50:11 +0700 Edusehat Stipple, Bio, and, Lonza, Agree, Focus, Advancing, Oncology, ADC, Therapies Lonza and Stipple Bio signed a multi-target licensing agreement to support the development of next-generation precision oncology ADC therapies.

    Officials at Stipple Bio say the company is leveraging its Pointillist Platform to identify tumor-specific cell surface epitopes, which can enable the development of high therapeutic index medicines designed to avoid on-target/off-tumor toxicity. Under the agreement, Stipple Bio will gain target-specific access to Lonza’s ADC technology platform to design potential first-in-class and best-in-class ADC products, including STP-100.

    This collaboration combines Stipple Bio’s epitope discovery capabilities with Lonza’s GlycoConnect antibody conjugation technology, HydraSpace® polar spacer technology, and a toxSYN® linker payload. In addition, Lonza is eligible to receive upfront, clinical, regulatory and commercial milestone payments, plus royalties on net sales of resulting products. Lonza is responsible for manufacturing components that are related to its proprietary technologies, and Stipple Bio is responsible for the R&D, manufacturing, and commercialization of the ADCs.

    “We value the opportunity to work with Stipple Bio to support their innovative epitope discovery approach with our advanced ADC technologies,” said Jan Vertommen, head of commercial development, advanced synthesis, Lonza. “By combining their science with Lonza’s established bioconjugation platforms and efficient, scalable manufacturing capabilities, we aim to help Stipple Bio progress more precise and effective ADC programs with confidence and speed.”

    “ADCs have become a core pillar of cancer treatment, and as the field advances, increasingly sophisticated design is translating into stronger efficacy and reduced off-target effects,” added Jeff Landau, CEO, Stipple Bio. “We are pleased to be partnering with Lonza and believe that their clinically validated platform will be instrumental in enabling us to translate that design sophistication into effective and better tolerated therapies.”

    The post Stipple Bio and Lonza Agree to Focus on Advancing Oncology ADC Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Experimental Adjuvant Could Strengthen Mucosal Immunity with Injectable Polio Vaccines https://edusehat.com/en/experimental-adjuvant-could-strengthen-mucosal-immunity-with-injectable-polio-vaccines https://edusehat.com/en/experimental-adjuvant-could-strengthen-mucosal-immunity-with-injectable-polio-vaccines Fri, 05 Jun 2026 07:50:10 +0700 Edusehat Experimental, Adjuvant, Could, Strengthen, Mucosal, Immunity, with, Injectable, Polio, Vaccines The injectable form of the polio vaccine has proven effective at preventing illness but it does not block the transmission of the virus as well as the oral version of the vaccine. That is because the virus is usually transmitted through contaminated food or water and is first exposed to the GI tract, where the oral vaccine induces a mucosal immune response. To date, several countries no longer use the oral vaccine because there is a small risk of infection. It is also possible for people who receive the injected polio vaccine to spread the virus even though they are asymptomatic. 

    Now according to data from an Massachusetts Institute of Technology-led study, it may be possible to modify the injectable vaccine so that it can also promote a mucosal immune response. This way, the vaccine could support polio eradication efforts without the risks of the oral polio vaccine. Details are published in a new Science Advances paper titled “Am80-Lipid nanoparticles serve as an enteric mucosal adjuvant 3 following parenteral immunization with inactivated polio vaccine.”

    In comments that shed some light on the thinking behind the work, Ana Jaklenec, PhD, a principal investigator in MIT’s Koch Institute for Integrative Cancer Research, stated that while “people who are vaccinated with the injectable vaccine are not getting sick” they may be helping spread the highly contagious virus. “Mucosal immunity could help lower that shedding and ideally eliminate it,” she said. 

    Her team’s version of the vaccine comprises an injectable, inactivated polio vaccine delivered with a nanoparticle-based adjuvant that helps steer immune cells to the mucosal lining of the intestine. Digging into the details, Jaklenec and her team worked with a group at Harvard Medical School who have shown previously that using a derivative of vitamin A as a vaccine adjuvant can help stimulate immune cells to go into the GI tract. 

    Though the adjuvant, known as Am80, generates a strong response, one challenge is that it needs to be injected for several days in a row, which is not feasible for most vaccine campaigns. To eliminate the need for repeated vaccinations, the scientists used a lipid nanoparticle (LNP) as a delivery vehicle that releases the adjuvant slowly over several days.

    Armed with the updated vaccine, the scientists moved on to testing it in rats. For their tests, the scientists injected the standard inactivated polio vaccine along with a separate injection of Am80 encapsulated in LNPs. They also delivered boosters to the rats at four and eight weeks. 

    Following injection, LNPs accumulate in the lymph nodes where they interact with B and T cells that are also exposed to the polio vaccine. The interaction stimulates the cells to produce two surface proteins that direct them to the GI tract. Additionally, the B cells produce IgA antibodies, which protect body surfaces from infection by coating the mucosal membranes. Lastly the rats produce IgG antibodies in the bloodstream, which are similar to the antibodies produced in response to the standard injected polio vaccine. 

    Overall, in the rats, they found that administering the vaccine and adjuvant produced a two-fold increase in the type of antibodies needed for mucosal immunity compared to the inactivated vaccine alone. Essentially, “by adding Am80 to lipid nanoparticle as an adjuvant, we are combining the safety of IPV with an adjuvant that can produce the mucosal immunity that normally you can only get with OPV,” said Behnaz Eshaghi, PhD, a postdoctoral student at MIT and lead author of the paper. 

    For their next steps, the scientists plan to test the improved vaccine in other large animal models where they will inject the vaccine and adjuvant mixed together. More broadly, Am80 and similar adjuvants could help scientists design improved vaccines for other pathogens that infect the GI tract or for diseases that infect the lungs or reproductive tract. 

    The post Experimental Adjuvant Could Strengthen Mucosal Immunity with Injectable Polio Vaccines appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Open Letter: In Support of Mandatory Nucleic Acid Synthesis Screening and Recordkeeping https://edusehat.com/en/open-letter-in-support-of-mandatory-nucleic-acid-synthesis-screening-and-recordkeeping https://edusehat.com/en/open-letter-in-support-of-mandatory-nucleic-acid-synthesis-screening-and-recordkeeping Fri, 05 Jun 2026 07:50:09 +0700 Edusehat Open, Letter:, Support, Mandatory, Nucleic, Acid, Synthesis, Screening, and, Recordkeeping

    An open letter, In Support of Mandatory Nucleic Acid Synthesis Screening and Recordkeeping, published late on the evening of June 3, 2026, and signed by life sciences researchers, technologists, national security experts and former White House officials, is calling for mandatory screening of synthetic nucleic acids. This effort is significant because it highlights that screening is a rare point of consensus for a wide coalition of science and technology experts and is widely seen as both pro-AI and pro-safety.

    From the CEOs of the major labs to AI skeptics and safety organizations to luminaries in the life sciences, public health, and national security, there is wide agreement that we need stronger screening guardrails. The letter calls on US lawmakers to codify mandatory nucleic acid synthesis screening, including recordkeeping, in order to combat the development of biological weapons at the scale of AI. The open letter reads as follows:

    As life sciences researchers, builders of AI and biotechnology, and experts with a wide range of views on how to approach AI policy, we call on legislators to make screening of orders for synthetic nucleic acids—and the equipment needed to make them—mandatory. 
    The ability to order synthetic DNA online has accelerated vaccine development, powered basic research, and made it possible for small teams to access capabilities that used to be confined to major institutions. Since the publication of protocols to reconstruct viruses from strands of DNA more than two decades ago, it has also been recognized as a point in the biotechnology supply chain where a bad actor could cause outsized harm. Recognizing the vulnerability, synthesis companies formed the International Gene Synthesis Consortium in 2009 to develop and implement voluntary safeguards against misuse.

    While the issue is not new, the pace of progress in artificial intelligence is. AI systems now outperform PhD-level virologists on questions about highly technical laboratory procedures in their own domains of expertise. The evidence about what this means for present-day biosecurity threats is genuinely mixed, but the trend is hard to dispute. AI systems are improving rapidly, and alongside incredible benefits to science and medicine, there is a real possibility that the knowledge barriers which have historically prevented bad actors from obtaining biological weapons will meaningfully erode.

    Support for screening does not depend on any particular view of AI; the biosecurity case has been recognized by scientists and governments for decades. Screening is also one of the best understood and least disruptive biosecurity measures available. It asks providers of synthesized DNA and manufacturers of synthesis machines to check synthesis requests for sequences of concern and to verify customer legitimacy before shipping orders. Providers should also record synthesis orders and sequence data to support legitimate biosecurity investigations, so that any threat that might evade initial screening can be traced back to its source — including when individual sequences would not raise concern in isolation. Awareness of traceability itself deters misuse.

    Many of the largest and most responsible providers in the industry already screen and record orders voluntarily because it is well understood that they have an important role to play in maintaining public trust in and mitigating potential misuse of this important technology.

    For these reasons, the undersigned support mandatory nucleic acid synthesis screening, including recordkeeping, in the United States.

    Given the pace at which the underlying technology is changing, we believe the need is urgent. Congress should act this session, and we applaud the legislative efforts currently underway. To ensure a consistent national standard rather than a patchwork of conflicting laws, states should also consider implementing requirements based on existing federal and industry guidelines.

    This is a rare moment of agreement across stakeholders that are often at odds. We hope policymakers will meet it with decisive action.

    Sincerely,

    You can find the full list of signatories and the letter here. I am a media consultant working with the two organizations that are the primary organizers of the letter: the Institute for Progress (IFP) and the Foundation for American Innovation (FAI). The best email contact regarding the open letter is letter@screendna.org.

    Carrie Hutcheson is senior director of the Glen Echo Group in Washington, DC.  

     

    The post Open Letter: In Support of Mandatory Nucleic Acid Synthesis Screening and Recordkeeping appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Immune Response Activated by RNA Splicing Opens Targeted Therapies https://edusehat.com/en/immune-response-activated-by-rna-splicing-opens-targeted-therapies https://edusehat.com/en/immune-response-activated-by-rna-splicing-opens-targeted-therapies Thu, 04 Jun 2026 05:55:16 +0700 Edusehat Immune, Response, Activated, RNA, Splicing, Opens, Targeted, Therapies In a new study published in Nature Communications titled, “Native long-read RNA sequencing of human monocytes reveals activation-induced alternative splicing toward functional isoforms,” researchers at University Medical Center (UMC) Utrecht have uncovered a previously underappreciated mechanism that helps immune cells respond rapidly to infections. The team showed that alternative RNA splicing plays a central role in shaping immune responses. The results provide new insights into immune-mediated diseases, such as infections, rheumatoid arthritis and lupus, and open the door to more targeted therapies. 

    The study focused on monocytes, a type of innate immune cell that acts as a first responder to pathogens. Using long-read RNA sequencing, the authors generated a comprehensive map of full-length RNA transcripts in human monocytes before and after activation. They identified more than 24,000 isoforms, the majority of which have never been described, revealing a previously hidden layer of molecular complexity. 

    Notably, immune activation triggers widespread ‘isoform switching.’ Rather than simply turning genes on or off, monocytes shift toward producing longer, fully functional RNA variants that are more likely to be translated into proteins. These isoforms contain complete coding sequences, fewer non-coding interruptions, and greater structural complexity, all features associated with more effective protein production. 

    “In our study we also confirmed that these RNA changes have real functional consequences,” said Jorg van Loosdregt, PhD, associate professor at UMC Utrecht and corresponding author of the study. “By integrating data on protein synthesis and ribosome activity, we demonstrated that the observed isoform shifts are linked to increased production of immune effector proteins. This shows that alternative splicing directly enhances the cell’s ability to respond to infection or inflammation.” 

    While previous studies have linked conditions, such as rheumatoid arthritis and lupus, to genetic variation affecting RNA splicing, the study demonstrates that disease mechanisms may also depend on which isoforms are produced and how efficiently they are translated into proteins. 

    “Our study underscores the importance of studying gene regulation at the isoform level. Traditional methods may overlook critical changes that only become visible with full-length RNA analysis,” said van Loosdregt. “The adoption of long-read sequencing technologies could therefore transform research into immune function and disease mechanisms.” 

    Emerging approaches, such as antisense oligonucleotides or drugs that influence splicing factors, may enable more precise modulation of the immune system and the development of targeted treatments for immune-mediated diseases. 

    The post Immune Response Activated by RNA Splicing Opens Targeted Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Immune Cell Phenotyping: Cell Surface Architecture Informs Disease Biology https://edusehat.com/en/immune-cell-phenotyping-cell-surface-architecture-informs-disease-biology https://edusehat.com/en/immune-cell-phenotyping-cell-surface-architecture-informs-disease-biology Thu, 04 Jun 2026 05:55:15 +0700 Edusehat Immune, Cell, Phenotyping:, Cell, Surface, Architecture, Informs, Disease, Biology

    Image of Erdinc Sezgin, PhD

    Erdinc Sezgin, PhD

    Senior Lecturer
    Karolinska Institutet

    Panelist

    Image of Erdinc Sezgin, PhD

    Erdinc Sezgin, PhD

    Erdinc Sezgin, PhD, leads the Cell Signalling, Immunity and Nanoimaging (CSI:Nano) Lab at Karolinska Institutet and SciLifeLab in Stockholm, Sweden. His lab works on biophysical principles underlying cellular processes in health and disease, developing advanced imaging, chemical biology, and synthetic biology tools to reveal the molecular mechanisms governing cellular physiology and disease processes.
    Image of Hanna van Ooijen, PhD

    Hanna van Ooijen, PhD

    Scientific Affairs Manager
    Pixelgen Technologies

    Panelist

    Image of Hanna van Ooijen, PhD

    Hanna van Ooijen, PhD

    Hanna van Ooijen, PhD, serves as the scientific affairs manager at Pixelgen Technologies, a Stockholm-based biotechnology company advancing spatial proteomics and single-cell protein interactomics. In her role, she works at the intersection of immunology, translational research, and emerging spatial biology technologies, helping researchers apply advanced tools to better understand immune cell behavior in areas such as oncology, cell therapy, and autoimmune disease research. Hanna is particularly interested in how nanoscale organization and protein interactions shape immune cell activity, and she has contributed to scientific outreach and presentations on next-generation approaches for profiling immune cells at single-cell resolution.  She earned her PhD from KTH Royal Institute of Technology, where her research focused on understanding the factors that regulate cytotoxic immune cell function, with a particular emphasis on cellular heterogeneity and immune cell dynamics.

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    • Time: 

    The biophysical properties of the plasma membrane actively shape immune cell function, providing key insights into chronic disease and immune dysfunction. Measuring membrane order across immune cell populations can reveal functionally distinct cell states invisible to canonical surface markers and open new avenues for therapeutics.

    In this GEN webinar, Erdinc Sezgin, PhD, Karolinska Institutet, will present how his lab profiled plasma membrane order across 12 immune cell subtypes simultaneously in healthy donors and patients with long COVID and chronic lymphocytic leukemia. He will also share how sorting NK cells by membrane order, combined with transcriptomics and the Proximity Network Assay (PNA) from Pixelgen Technologies, uncovered distinct subsets differing in cytotoxic potential, migratory capacity, and surface protein organization for biomedical applications.

    Key takeaways include:

    • How plasma membrane order varies across immune cell types in chronic disease

    • Using biophysical membrane order to identify NK cell subsets that cannot be distinguished by surface markers alone

    • How spatial surface proteomics via PNA separates functionally distinct NK cell populations

    • How membrane order profiling can complement standard immunophenotyping workflows

    A live Q&A session will follow the presentation offering you a chance to pose questions to our expert panelists.

    Produced with support from:

    Pixelgen logo

    The post Immune Cell Phenotyping: Cell Surface Architecture Informs Disease Biology appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     NIIMBL to Support Vector Production and AI-Ready Training Projects https://edusehat.com/en/niimbl-to-support-vector-production-and-ai-ready-training-projects https://edusehat.com/en/niimbl-to-support-vector-production-and-ai-ready-training-projects Thu, 04 Jun 2026 05:55:14 +0700 Edusehat NIIMBL, Support, Vector, Production, and, AI-Ready, Training, Projects Viral vector production and training schemes designed to foster development of an AI-ready workforce dominate the list of projects selected for support by the U.S. National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL).

    The institute, a public-private partnership focused on advancing manufacturing and solving industry challenges, announced its latest funding awards, explaining that the 39 recipients would support U.S. production and talent development.

    Sandeep Kedia, NIIMBL senior technology fellow and project call program lead, says the projects “represent the kind of innovation needed to strengthen the nation’s biopharmaceutical manufacturing capabilities.

    “By bringing together advanced process analytical technologies, AI-driven optimization, and next-generation production platforms, our members are helping accelerate the adoption of transformative technologies across the industry,” he adds.

    Several of the selected projects focus on the production of adeno-associated viral (AAV) vectors—hollow viruses used to deliver genetic information—which play a crucial role in cell and gene therapy manufacturing.

    For example, researchers at Michigan Technological University will work with industry partners on an aqueous two-phase continuous vector purification system. The aim is to boost yield while reducing cost, labor, and analytical complexity.

    Similarly, a team at North Carolina State aims to develop “improved purification materials that can better capture full AAVs, along with machine-learning software that identifies optimal process conditions.”

    The third vector-focused project will see an MIT group work with EMD Millipore, Landmark Bio, and Virica Biotech to try to reduce the number of empty viral capsids inadvertently made during vector production.

    The researchers will combine an approach called decoupled replication-initiated vector encapsulation, or DRIVE, with various process control strategies to create a platform that makes high-titer, high-quality rAAVs.

    According to the MIT team, “By reducing [the proportion of] empty capsids, the approach can streamline downstream purification, reduce time and cost, and improve the overall quality of gene therapy products.”

    AI-ready workforce

    In addition to the technology projects, NIIMBL will support several training programs with an emphasis on ensuring the next generation of biopharmaceutical engineers are AI-ready, according to workforce director John Balchunas.

    “Our workforce initiatives are designed to meet talent needs head‑on by creating more innovative pathways into biomanufacturing careers,” he says, adding, “These new projects will strengthen partnerships and ensure that learners can gain the skills needed to thrive in a rapidly evolving biopharma industry.”

    One such project will see a team at Texas A&M University’s National Center for Therapeutics Manufacturing expand an existing effort called NeuroPipes, which seeks to interest neurodiverse people in careers in biopharma. The aim is to provide technical skills training that prepares neurodivergent adults for careers in drug manufacturing.

    Another project will see Wistar Institute researchers set up BioPATH, a national consortium focused on advancing workforce training in biomanufacturing, AI, and automation.

    The idea, according to the Wistar team and collaborators at the International Academy of Automation Engineering, is to “bridge the gap between foundational bioprocess and GMP knowledge and the emerging needs of automation, data-driven manufacturing, and digitally enabled quality systems.”

    The post NIIMBL to Support Vector Production and AI-Ready Training Projects appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Web App Helps Flag Antibodies Where Manufacturability Might Be an Issue https://edusehat.com/en/web-app-helps-flag-antibodies-where-manufacturability-might-be-an-issue https://edusehat.com/en/web-app-helps-flag-antibodies-where-manufacturability-might-be-an-issue Thu, 04 Jun 2026 05:55:13 +0700 Edusehat Web, App, Helps, Flag, Antibodies, Where, Manufacturability, Might, Issue Researchers have developed an open-source web app to help drug manufacturers and developers identify unstable antibodies prone to aggregation. The team from Oxford University says the Therapeutic Antibody Profiler 2 (TAP2) can compare the fragment variable component of a proposed antibody to successful clinical-stage antibodies.

    According to Clare Gillis, a researcher in bioinformatics and computational biology, the app has the potential to help companies begin process development. “It can help them if they already know their antibody binds as they want, but they need to know if it will pass through the whole developability and manufacturability pipeline,” she says.

    TAP2 uses five easily calculable physiochemical metrics based on surface residues of the antibody, Gillis says. These are more likely to affect manufacturability.

    The web app metrics are selected to model aspects of antibody behavior, such as hydrophobicity, she adds. If there are big patches of hydrophobic residues on the outside of the antibody, then it’s more likely to be reactive and, thus, less likely to remain stable as a formulated drug product.

    Likewise, Gillis explains, if the surface of the antibody features large patches of positive or negative charge, it is likely to have nonspecific reactions that will cause destabilization and aggregation.

    With the TAP2 app, companies can flag early amber or red warnings for antibodies where manufacturability might be an issue. In addition, the group also offers a web app profiler for therapeutic nanobodies, TNP, as well as Humatch, an app that can help tweak antibodies to be more ”human-like” and less likely to cause immune reactions in patients, she says.

    About Humatch, Gillis says, “you can add a best single point mutation and then iterate over and over until the model believes the antibody is fully humanized.” The app works for any antibody with paired heavy and light chain variable domains (VH and VL), she says, and can potentially help manufacturers of harder-to-produce products that don’t exist in nature.

    The post Web App Helps Flag Antibodies Where Manufacturability Might Be an Issue appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Bioproduction Pivots from Centralized to Regional Support https://edusehat.com/en/bioproduction-pivots-from-centralized-to-regional-support https://edusehat.com/en/bioproduction-pivots-from-centralized-to-regional-support Thu, 04 Jun 2026 05:55:12 +0700 Edusehat Bioproduction, Pivots, from, Centralized, Regional, Support The global biopharma industry is placing increasing importance on regional support rather than only centralized expertise to help complex programs advance. A key benefit is access to local expertise in or near their time zone.

    The localization movement “is part of a global shift [in which] companies are assessing how they balance cost, quality, and risks across regions rather than relying on any single market,” Jessay Devassy, PhD, global R&D director, Ecolab Life Sciences, tells GEN.

    Ecolab opened a bioprocessing applications lab in Korea this spring. “Being in Korea allows the exchange of ideas in an iterative fashion…so knowledge moves seamlessly between regions. That’s much easier if you’re in their proximity,” Devassy points out.

    This is the company’s first bioprocessing lab in Asia. Situated in Dongtan, Korea, it supports process development studies from early- to commercial-scale, focusing on biologics’ downstream purification.

    Korea was a logical choice. “Korea is highly advanced in manufacturing,” Devassy continues. Now it’s evolving from a manufacturing hub to a comprehensive biopharma ecosystem, with active contributions from R&D all the way through clinical development, with home-grown and multinational companies alike.

    With its biologics manufacturing history, “I think Korea has become one of the most trusted locations globally,” he says. “Its quality standards are well-aligned with North American and European standards.” Consequently, global clients are assured that the same approaches and standards are applied to development as in the United States or Europe.

    Korea’s aspirations

    Government support is part of that. The Korean government designated biopharma as a strategic industry after COVID-19 and reiterated that goal in 2023’s Third Five-year Comprehensive Plan for Development and Support for the Bio-Pharmaceutical Industry. Key points include developing two blockbuster drugs by 2027, doubling pharmaceutical exports to $16 billion, and positioning Korea among the top six nations for pharmaceutical development.

    At the end of 2025:

    • New venture capital investments in biotech and medical companies reached $830 million, up approximately 11% from the prior year.
    • Total venture investments in the biotech and medical sector rose more than 29% from 2024, more than for any other industry.
    • Continuous bioprocessing is expected to experience a compound annual growth rate of nearly 20% between 2025 and 2030, reaching revenues exceeding $21 million.

    Challenges

    The competition to attract biopharma companies is robust. India is the fastest-growing Asia-Pacific market, but, Devassy says, “China has a cost advantage…[in] manufacturing and development.” It’s also the largest biopharma market in the Asia-Pacific region.

    “Japan has more established domestic systems for biomanufacturing,” Devassy continues. According to Grand View Horizon, Japan leads the pack for projected revenue from continuous bioprocessing to 2030.

    Devassy positions Korea “somewhere in between” China and Japan. “It’s strong technically, but is still navigating regulatory complexity and global competition.” Currently, it generates 2.2% of the world’s continuous bioprocessing revenues.

    “Biopharma exports from Korea have seen strong growth recently…and Ecolab is playing a strong part in supporting the manufacturers behind that growth,” Devassy says. “This is our first step toward making our global expertise accessible to growing markets in Asia.”

    The post Bioproduction Pivots from Centralized to Regional Support appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Gentler Cell Separation Methods Gain Momentum https://edusehat.com/en/gentler-cell-separation-methods-gain-momentum https://edusehat.com/en/gentler-cell-separation-methods-gain-momentum Thu, 04 Jun 2026 05:55:12 +0700 Edusehat Gentler, Cell, Separation, Methods, Gain, Momentum The race to commercialize cell therapies is forcing bioprocessing innovators to confront one of the field’s most persistent manufacturing bottlenecks: isolating fragile hematopoietic stem cells (HSCs) without compromising their therapeutic potential. “HSCs are extremely rare and extremely delicate,” says Sophie He, PhD, vice president of cell therapy and head of mergers and acquisitions at Bracco. “Trying to isolate HSCs while preserving their therapeutic function is extremely difficult.”

    The challenge begins with biology itself. CD34+ hematopoietic stem and progenitor cells typically account for just one to three percent of mobilized apheresis collections and one to four percent of bone marrow populations, while the most primitive long-term HSCs can represent less than one-tenth of a percent of total marrow cells. That rarity means every processing step matters.

    For manufacturers scaling autologous and allogeneic therapies, the result is a difficult balancing act between purity and yield. Conventional enrichment workflows often sacrifice one to achieve the other. “To get higher purity, traditionally one gets lower yield,” He explains. “Every wash or transfer step in the isolation process results in cell loss.” The problem is magnified by the fact that HSCs rely on preserving self-renewal, multipotency, and engraftment capability—functions that can easily be disrupted during processing, ultimately reducing clinical effectiveness.

    As developers move toward commercial-scale manufacturing, traditional magnetic separation systems are facing growing scrutiny. According to He, magnetic columns can expose HSCs to damaging shear forces, compression, and membrane stress because of their fragile membranes and cytoskeletons. Processing times can also become a major operational burden. “Magnetic columns can require more than 10 hours to completely process larger mobilized apheresis starting material,” she says. “That could lead to apoptosis and metabolic stress.” The lengthy workflows create additional challenges for scalability and reproducibility across manufacturing sites, particularly as companies transition from small clinical batches to commercial production runs.

    Newer approaches are gaining attention for their ability to handle cells more gently while supporting larger-scale workflows. Among them, microbubble-based separation uses buoyancy rather than magnetic force to isolate HSCs. He says the technology reduces mechanical stress on cells while also minimizing concerns about residual materials left behind during processing. The broader industry goal, however, extends beyond replacing one technology with another. Developers are searching for a platform simultaneously capable of delivering high purity, high yield, preserved cell functionality, and proven scalability.

    He describes the search for an ideal HSC isolation platform as “the holy grail” for cell-therapy bioprocessing at a commercial scale. In addition to biological performance, future systems must reduce operator dependency, integrate efficiently into manufacturing workflows, and support reproducibility across donors, sites, and operators. Regulatory clarity will also be essential before any technology can achieve widespread adoption. As regenerative medicine advances toward broader commercialization, the ability to isolate healthy stem cells consistently and at scale might determine which therapies successfully transition from experimental promise to industrial reality.

    The post Gentler Cell Separation Methods Gain Momentum appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     New Antibiotic, Manikomycin, Acts on Novel Ribosomal Target https://edusehat.com/en/new-antibiotic-manikomycin-acts-on-novel-ribosomal-target https://edusehat.com/en/new-antibiotic-manikomycin-acts-on-novel-ribosomal-target Thu, 04 Jun 2026 05:55:10 +0700 Edusehat New, Antibiotic, Manikomycin, Acts, Novel, Ribosomal, Target Traditionally, antibiotic discovery has involved the isolation of natural products from fungi and bacteria—largely actinomycetes. However, the perception that antibiotic-producing actinomycetes have yielded all they can, with little left to give, has motivated a recent shift toward novel antibiotic discovery processes.

    However, a new study from researchers at McMaster University presents the isolation of a novel antibiotic from Streptomyces rimosus that showed efficacy against multiple bacteria, including multidrug-resistant Enterobacteriaceae. In addition, the compound—known as manikomycin—is the first antibacterial agent known to target the E-site in the large ribosomal subunit, opening the door to an entirely new class of treatments.

    “Not a single antibiotic prescribed in clinics today does what manikomycin does,” says Gerry Wright, PhD, professor in the department of biochemistry and biomedical sciences at McMaster University in Ontario, Canada. “Not azithromycin, not tetracycline—none of them. So, we’ve not only found a brand-new drug candidate, but we’ve also established a brand-new target in bacteria that could potentially be exploited with other new drugs.”

    This work, published in Nature in the paper, “A natural depsipeptide antibiotic binds the E-site of the bacterial ribosome.”

    This discovery marks the fourth new antibiotic candidate from the Wright lab in just over a year, underscoring a promising new approach to drug discovery at a time when antibiotic resistance is a growing global threat.

    Given that many antibiotics used today target the ribosome, bacteria have evolved broad defense strategies against them. However, a drug targeting a different part of the ribosome will not face the same resistance mechanisms.

    Manikomycin binds in the E-site of the large subunit of the bacterial ribosome, the authors write, “preventing entry of the 3′ end of the tRNA into the E-site and effectively hindering the translocation step of protein synthesis in a sequence-context-specific manner.”

    “Even newly discovered drugs that attack those same old targets may quickly face resistance,” says Wright. “But, over the history of medicine, we’ve put absolutely no selective pressure on this particular target, so bacteria have no existing resistance mechanisms for manikomycin.”

    The discovery of manikomycin builds on work that began more than 75 years ago, when scientists first discovered that the soil bacterium Streptomyces rimosus produced oxytetracycline, a powerful new drug that would help usher medicine into the antibiotic age.

    While the breakthrough was one of several like discoveries made in the mid-1900s, S. rimosus and related bacteria have long since been abandoned as a potential source of new antibiotics.

    “There is an overwhelming perception in science that these bacteria have been mined completely dry—that we’ve found all there is to find,” Wright says. “Our lab has found that this is not at all the case.”

    Wright’s group, working with collaborators at the University of Illinois Chicago and the University of Hamburg in Germany, used an advanced fractionation method to uncover the new antibiotic. By filtering out oxytetracycline and other abundant compounds from the chemical mixtures produced by S. rimosus, the researchers were able to isolate scarcer molecules that had gone unnoticed over the years.

    “There is likely so much still to be discovered through fractionation,” says Manpreet Kaur, PhD, a postdoctoral fellow in Wright’s lab. “Revisiting the extracts of even-well studied bacteria like Streptomyces may lead to similar discoveries in the future.”

    Wright’s team is now advancing manikomycin toward clinical development. They have already shown that the new antibiotic is not toxic to human cells, and that it works well in a lab-controlled model of infection—key milestones on the early development pathway.

    The team is now working on optimizing the drug’s “residency time”—or how long it stays active in the body—and have produced 60 different derivatives with plans to push the best one forward.

    “We’re excited about this molecule’s potential,” Wright says. “There’s a clear path forward, and we may even be able to expand its spectrum so that it eventually affects even more bacteria, too.”

    The post New Antibiotic, Manikomycin, Acts on Novel Ribosomal Target appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cellares and TScan Agree to Evaluate Automated Manufacturing TSC-101 for Patients with Hematologic Malignancies https://edusehat.com/en/cellares-and-tscan-agree-to-evaluate-automated-manufacturing-tsc-101-for-patients-with-hematologic-malignancies https://edusehat.com/en/cellares-and-tscan-agree-to-evaluate-automated-manufacturing-tsc-101-for-patients-with-hematologic-malignancies Thu, 04 Jun 2026 05:55:09 +0700 Edusehat Cellares, and, TScan, Agree, Evaluate, Automated, Manufacturing, TSC-101, for, Patients, with, Hematologic, Malignancies IDMO Cellares and TScan Therapeutics will work together to evaluate automated clinical manufacturing of TSC-101, which is TScan’s lead TCR-T therapy candidate for patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

    TSC-101 is designed to treat residual disease and prevent relapse in patients with AML and MDS undergoing allogeneic hematopoietic cell transplantation (allo-HCT). The therapy candidate uses a gene modification approach to engineer T cells from a healthy donor into a patient-specific cell therapy product. As TScan advances TSC-101 toward a pivotal trial, which is expected to begin in the second quarter of 2026, the company is evaluating Cellares’ automated manufacturing platform as a scalable and economical path to future commercial demand.

    Under the agreement, Cellares will automate the TSC-101 manufacturing and testing processes on the Cell Shuttle, its end-to-end manufacturing platform, and the Cell Q, its automated quality control and release testing system. These closed-system, fully automated workflows are designed to reduce process variability, minimize labor intensity, and enable consistent execution across runs and geographies, according to a Cellares official.

    “As we prepare for the initiation of our pivotal study of TSC-101 this quarter, we are increasing our efforts for commercial readiness. Establishing a scalable and cost-efficient manufacturing strategy is a critical component. Cellares’ fully automated Cell Shuttle platform represents a promising approach to automating and scaling cell therapy production, with the potential to reduce manual processes and eliminate capacity constraints,” said Ray Lockard, chief manufacturing and quality officer of TScan Therapeutics.

    “Through this evaluation, we aim to determine how this technology could strengthen our long-term manufacturing network and support broader patient access, supporting our goal of delivering transformative therapies to patients as efficiently and reliably as possible.”

    “Patients with AML or MDS who remain at risk of relapse following transplant represent exactly the kind of underserved population that automated manufacturing was designed to reach,” added Fabian Gerlinghaus, co-founder and CEO of Cellares. “Bringing automation to a late-stage program like TSC-101, with its healthy donor-derived but patient-specific manufacturing model, is the kind of challenge the Cell Shuttle and Cell Q were built for, and we believe it represents the manufacturing economics any developer will need to reach a population of this scale.”

    The agreement adds TCR-engineered T cell therapies to Cellares’ portfolio of automated cell therapy modalities, which includes CAR T cell therapies, hematopoietic stem cell programs, and autologous progenitor T cell therapies.

     

    The post Cellares and TScan Agree to Evaluate Automated Manufacturing TSC-101 for Patients with Hematologic Malignancies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Small Molecules to Big Partnership: Incyte, Genesis Expand AI Collaboration to $1B+ https://edusehat.com/en/small-molecules-to-big-partnership-incyte-genesis-expand-ai-collaboration-to-1b https://edusehat.com/en/small-molecules-to-big-partnership-incyte-genesis-expand-ai-collaboration-to-1b Thu, 04 Jun 2026 05:55:08 +0700 Edusehat Small, Molecules, Big, Partnership:, Incyte, Genesis, Expand, Collaboration, 1B Drug collaborations don’t always work out as planned. Sometimes they work out better.

    When Incyte agreed last year to partner with artificial intelligence (AI) platform developer Genesis Molecular AI to research, discover, and develop at least two small molecule treatments, they designed a collaboration that would generate at least up to $620 million for Genesis, whose foundation models for molecular AI are designed to power agentic drug design and development.

    The companies now say they made enough progress over the past 15 months to expand their AI-based drug collaboration to encompass at least five targets—with a potential payoff for Genesis that has ballooned to over $1 billion.

    Behind that expansion, Incyte and Genesis say, is the promise shown so far by the two initial targets, both selected by Incyte as called for in the initial strategic collaboration. One is a “very hard-to-drug, novel target” for which the companies worked to create novel, first-in-class chemical matter, while the other is a target that other companies have sought to make druggable without success, Pablo J. Cagnoni, MD, Incyte’s president and global head of R&D, told GEN.

    “Novel targets create problems for obvious reasons. You don’t have any chemical matter that you know to start with. The collaboration with Genesis has jump-started that program significantly,” Cagnoni said of the first target. “You need a crystal structure, you need to know which particular site in the target you need to bind, and then you need to start making chemical substance against it.”

    “It’s easy to make chemical matter, it’s really hard to make medicines—so that was the optimization step that Genesis really helped us do,” Cagnoni added.

    The second target, he explained, required not only high potency and very high selectivity, but unique pharmaceutical and pharmacokinetic properties. The companies were able to incorporate those and other properties for the target with help from Genesis’s generative and predictive AI platform, Genesis Exploration of Molecular Space (GEMS).

    GEMS integrates AI and physics into models designed to generate and optimize drug molecules. GEMS’ generative diffusion model for structure prediction, Pearl—short for “Placing Every Atom in the Right Location”—was unveiled in an October 26 preprint showing it to have surpassed AlphaFold 3 and other open source baseline models on the public protein-ligand co-folding benchmark Runs N’ Poses (14.5% improvement) and the docking and molecular generation benchmark PoseBusters (14.2% improvement).

    ‘Substantial progress’

    “By being able to optimize multiple parameters at the same time with the help of the GEMS platform and our colleagues at Genesis, we were able to really make substantial progress that was eluding us with other technology,” Cagnoni said. “The collaboration with Genesis has allowed us to make significant progress on the path to an IND. We’re not quite there, but we’re getting pretty close to that.”

    The two targets, he said, represented opposite ends of the drug discovery spectrum: “For one, we had something that started to look like a drug but wasn’t good enough. For the other one, we had a great target and no drugs. So, taking a view of those two ends of the spectrum, convinced me that we had to expand this, make it as broad as possible, and that’s why we put in place a new collaboration.”

    As with their initial collaboration, the companies aren’t yet revealing the targets or therapeutic areas in which they are working, though Cagnoni said they fall within one of Incyte’s three current therapeutic areas of interest: hematology, oncology, and inflammation and autoimmunity, a narrower niche within the traditional I&I (inflammation and immunology) focus area.

    Through the expanded collaboration, Incyte will use its proprietary experimental data to train Genesis’ GEMS platform, with the aim of accelerating drug development across multiple programs.

    Options beyond five targets

    Incyte will select at least five new targets to develop with Genesis, with options to nominate additional collaboration targets over time. Incyte will have exclusive rights to develop and commercialize treatments developed through the collaboration.

    “We know what properties a priori we need to optimize for, always with some caveats,” Feinberg said. “We almost always know that we need to achieve potency, selectivity, a wide variety of ADME [absorption, distribution, metabolism, and excretion] properties. Usually, in a given program, something like 30 or so different ADME assays are routinely run to some degree of frequency. This can often feel like playing whack-a-mole, instead of the serious engineering task of multi-parameter optimization.”

    “Our aim,” he added, “is to render the drug discovery process as much like the latter and as little like the former.”

    Incyte has agreed to pay Genesis $120 million upfront—to consist of $80 million cash and a $40 million purchase of Genesis’ equity—and unspecified recurring research funding to support AI model training and inference computing. Incyte has also agreed to pay genesis up to $232 million in payments per target, tied to achieving preclinical and clinical development, regulatory, and sales milestones.

    The collaboration is the second AI-focused partnership announced by Incyte in late May. A day before the Genesis expansion announcement, Incyte said it had launched a separate strategic collaboration with Edison Scientific to employ its Kosmos AI platform for discovery and development work—namely enabling continuous learning from translational and clinical data, real-time synthesis of evidence and predictive models of therapeutic performance.

    Incyte and Edison disclosed the focus of their initial project: “high-impact” use cases in target discovery and validation and translational biology, where Edison’s AI capabilities will be embedded within Incyte’s research workflows. The companies said they aim to support more efficient exploration of experimental, clinical, and biomarker data with the potential to expand across Incyte’s broader R&D organization.

    As for Incyte’s collaboration with Genesis, if Genesis achieves all milestones across the five initial targets of the expanded partnership, including multiple indications and major territories, Incyte will pay the company more than $1 billion—as long as the aggregate peak annual net sales of the five products exceed specified milestones. Payments could grow to “several” billion dollars depending on how many additional collaboration targets are nominated, and how many milestones are achieved.

    Genesis is also eligible to receive royalties on sales of any approved collaboration products.

    Stanford spinout

    Genesis spun out in 2019 from the Stanford University lab of Vijay Pande, PhD, co-founder and managing partner of the venture capital firm VZVC and a former general partner at Andreessen Horowitz (a16z) and founding general partner of its bio funds. Feinberg was a graduate student in Pande’s lab who co-invented and co-authored key peer-reviewed papers detailing deep learning technologies.

    In 2020, Genesis won a $52 million Series A financing. The company has grown since then to raise $340 million, most of that consisting of $200 million Series B financing completed three years later, plus the $40 million strategic investment Incyte made in Genesis equity as part of the companies’ expanded partnership.

    In addition to a16z, Genesis’ investors have included NVentures, the venture capital arm of AI chip giant Nvidia, which has expanded in recent years into biopharma among other industries.

    Incyte is the fourth and latest biopharma giant to partner with Genesis on an AI-focused drug discovery and development collaboration applying GEMS. Genesis garnered $35 million upfront in launching its partnership with Gilead Sciences in 2024, and earlier announced past collaborations with Eli Lilly and Genentech, a Member of the Roche Group.

    “Our mission at Genesis is to create AI technologies that enable creating drugs that otherwise would not be possible,” Evan Feinberg, PhD, Genesis’ founder and CEO, told GEN. “And thanks to working with really, really elite drug discovery teams, like what Incyte has, we’re able to work on a wide spectrum of very important problems in drug discovery.”

    That work, he asserted, requires discerning the uniqueness of each potential target.

    “Every target is really its own special snowflake in some way. Every drug target really entails its own challenges, oftentimes requires its own special approach,” Feinberg said. “Over the past year, we were able to work on two very different programs, that each have their own challenges, and thereby enable us to adapt and deploy our GEMS AI platform in these very different settings, bringing one of those two targets much closer to IND, and for the other target finding the first-in-class chemical matter, which was a very exciting year of work.

    “Now we’re excited to address the challenges ahead with this, expanded partnership together,” Feniberg added.

    The post Small Molecules to Big Partnership: Incyte, Genesis Expand AI Collaboration to $1B+ appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Human Hookworm Engineered to Produce, Secrete Anti-Tetrodotoxin Antibody Into Preclinical Host Bloodstream https://edusehat.com/en/human-hookworm-engineered-to-produce-secrete-anti-tetrodotoxin-antibody-into-preclinical-host-bloodstream https://edusehat.com/en/human-hookworm-engineered-to-produce-secrete-anti-tetrodotoxin-antibody-into-preclinical-host-bloodstream Thu, 04 Jun 2026 05:55:07 +0700 Edusehat Human, Hookworm, Engineered, Produce, Secrete, Anti-Tetrodotoxin, Antibody, Into, Preclinical, Host, Bloodstream Hookworms, intestinal parasites that infect hundreds of millions of people in under-resourced tropical regions around the globe, have evolved to survive inside the human gut for years, secreting molecules that enable co-existence with their hosts. Now, researchers at Washington University School of Medicine in St. Louis have harnessed that biological mechanism for potential human benefit, engineering a human hookworm parasite, Ancylostoma ceylanicum, to produce and deliver a drug within a living host.

    Headed by Makedonka Mitreva, PhD, the Gordon R. Miller Professor in the John T. Milliken Department of Medicine’s Division of Infectious Diseases at WashU Medicine, the investigators report what they say is the first successful genetic modification of the human hookworm, which they engineered to produce an antibody that neutralizes tetrodotoxin (TTX), a deadly neurotoxin produced by pufferfish and other marine animals. The team’s preclinical study demonstrated that the modified hookworms colonized an animal host, and secreted the antitoxin into the host bloodstream, partially inactivating the toxin. They say the findings demonstrate that this drug production and delivery approach could potentially offer a long-term solution for multiple indications, including continuous treatment for chronic conditions, or for exposure to toxins in remote settings.

    “The hookworm has spent millions of years perfecting how to assure long-term survival inside a human host and how to get molecules out of its body and into ours,” said Mitreva. “We asked: What if we could add one more molecule to the roughly 1,000 things the worm already secretes, something therapeutically useful to people? This study shows that’s not just a concept. It works.”

    Mitreva and colleagues reported on their study in Nature Communications, in a paper titled “Transgenic hookworm secretes anti-tetrodotoxin human single chain antibody.” In their paper the team concluded that their achievement, “… represents a critical step towards the development of a transgenic human hookworm pharmaceutical biofactory platform with the potential to continuously, safely, and effectively deliver biologics in situ within patients.”

    “Hookworms have evolved to survive for years within the human host while minimally disrupting host homeostasis, and controlled human infections with hookworms are safe and well-tolerated in clinical settings, bolstering their potential for utility as pharmaceutical biofactories,” the authors wrote.

    Hookworms have already been studied as treatments for inflammatory bowel diseases such as ulcerative colitis, based on evidence that the anti-inflammatory molecules the worms secrete can dampen the immune responses that drive those conditions. Mitreva’s team set out to build on that foundation by engineering the worm to secrete a therapeutic of the researchers’ choosing, rather than relying solely on what the parasite produces naturally.

    The appeal of hookworms as a long-term drug production and delivery platform stems from a quirk of their biology. When a person is infected with a controlled number of hookworm larvae, which can be administered orally as a pill or through the skin like a lotion, the worms migrate to the small intestine and take up residence, often for years. Because they cannot multiply inside the host, the number of worms stays fixed, and the infection remains controlled. If the infection ever needs to be cleared, a single dose of an oral anti-parasitic drug eliminates the hookworms within 24 hours.

    To adapt hookworms for therapeutic use, Mitreva and her team drew on more than two decades of hookworm genomics research conducted at WashU Medicine. This depth of data helped them understand the organism’s biology from the cellular to the genetic level, allowing them to locate a viable site in the genome to insert the new gene carrying instructions for making the new antitoxin. The antibody selected for the team’s reported proof-of-concept study neutralizes tetrodotoxin, a paralyzing and potentially lethal toxin with no antidote.

    The project presented significant technical hurdles: gene-editing tools that work in other organisms had not been adapted for hookworms, and no one had previously achieved stable genetic modification in the species. Critically, they had to ensure the insertion wouldn’t disrupt surrounding gene activity and would prompt the worm to secrete the antitoxin out into the host.

    The team reported that blood collected from hamsters infected with the genetically modified hookworms partially neutralized tetrodotoxin, whereas blood from animals infected with unmodified worms had no neutralizing capability. Mitreva noted that the level of neutralization achieved in this initial study, while significant, likely represents only a fraction of what the platform can ultimately deliver. They wrote in summary “Here, we report on methodological, technical, and conceptual advances, demonstrating successful bioengineering of a human hookworm, Ancylostoma ceylanicum, to produce and secrete a human single-chain antibody, s16-HuScFv, that neutralizes tetrodotoxin (TTX).”

    Several components of what she calls a “configurable chassis” are still being optimized to increase the amount of therapeutic protein produced and secreted. Because the worm resides in the gut and a substantial portion of what it secretes remains there, rather than entering the bloodstream, the researchers expect that concentrations of therapeutic molecules in the intestine may be substantially higher than what was detected in circulation in this study, making the platform suitable for gut-directed therapies.

    In their paper the team wrote, “Building on the foundation that experimental human hookworm infection has been shown to be safe and well tolerated, here we present technological, methodological, and conceptual advances that have enabled the establishment of a genetically modified and tractable model system that can produce and deliver biologics … Taken together, this transgenic human hookworm platform highlights a promising approach in biotechnology that has the potential to significantly advance how we conceptualize disease treatment and prevention. Technologically, it also constitutes a notable advance in functional genomics for hookworms and helminths more broadly.”

    Mitreva added, “What we demonstrated here is that the concept works end to end—you can insert a gene, the worm produces the protein, the protein gets out of the worm, and it is functionally active in the host. From that starting point, we can optimize the platform and think carefully about which diseases stand to benefit most from a delivery system that is continuous, targeted and long-lasting. That’s a fundamentally different kind of pharmaceutical biofactory platform, and we think it opens possibilities that are very hard to achieve with any other platform.”

    Gut inflammatory diseases, including Crohn’s disease and ulcerative colitis, and food allergies are among the conditions Mitreva sees as strong candidates for future development. Diseases requiring small but sustained therapeutic concentrations, where compliance with repeated injections or infusions is a barrier, may also be well-suited to the platform. “Given the availability of controlled human infections, our disease-agnostic bioengineered hookworm platform offers a next-generation approach to address a suite of chronic human diseases, and with a single-dose administration, could potentially produce and deliver biologic medicines within the human host for years,” the authors wrote.

    Although natural hookworm infection may cause only mild digestive symptoms in healthy adults, chronic infection with large numbers of hookworms can be dangerous for children, pregnant people and malnourished or otherwise vulnerable individuals. Infection can lead to anemia, poor growth and development, pregnancy complications and, in extreme untreated cases, heart problems or death.

    This underscores the importance of keeping the infection strictly controlled for therapeutic use, Mitreva noted, which is possible because of the worms’ inability to reproduce without spending part of their life cycle in soil. “… as research progresses, it will be essential to ensure that these transgenic organisms do not have unintended ecological or human health impacts, maintaining a balance between innovation and safety,” the authors stated.

    Mitreva noted that biocontainment strategies, such as engineering the worms to be unable to produce eggs, are under consideration to protect hosts and their environments as the platform advances. “Future studies can also address biocontainment of the genetically modified organism (GMO) by engineering suicide genes and/or inducible promoters into the transgene,” the team suggested.

    The post Human Hookworm Engineered to Produce, Secrete Anti-Tetrodotoxin Antibody Into Preclinical Host Bloodstream appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Spatial Single‑Cell Platform Reveals Barriers to Antibody Delivery in Solid Tumors https://edusehat.com/en/spatial-singlecell-platform-reveals-barriers-to-antibody-delivery-in-solid-tumors https://edusehat.com/en/spatial-singlecell-platform-reveals-barriers-to-antibody-delivery-in-solid-tumors Wed, 03 Jun 2026 19:10:10 +0700 Edusehat Spatial, Single‑Cell, Platform, Reveals, Barriers, Antibody, Delivery, Solid, Tumors Targeting solid tumors remains one of oncology’s most persistent challenges. Even when a therapeutic antibody is well‑designed, and its molecular target is clear, the drug often struggles to reach its destination inside the dense, heterogeneous architecture of human tumors. Understanding why these agents fail in patients has been a longstanding blind spot in cancer pharmacology.

    A new study from Vanderbilt University Medical Center and Stanford University begins to close that gap. In work published in Nature Biotechnology, researchers developed a single-cell spatial pharmacology (SSP) platform, an experimental and analytical system that visualizes drug–tumor interactions directly in human solid tumors. The approach provides a high‑resolution view of drug delivery, target engagement, and the physical barriers that shape therapeutic response.

    Eben Rosenthal, MD, the Barry and Amy Baker professor and chair of otolaryngology–head and neck surgery at Vanderbilt Health, is senior author of the paper, titled “Single‑cell spatial pharmacobiology identifies conserved stromal barriers to therapeutic antibody delivery in human solid tumors.” Rosenthal and co‑author Guolan Lu, PhD, of Stanford University School of Medicine, developed SSP to quantify how antibody‑based therapies behave once they enter the tumor microenvironment.

    “Identifying the reason drugs fail in so many cancer patients is a high priority, and SSP can help,” Rosenthal said. “Current pharmacology tools and imaging methodologies do not provide the answers we need to understand which drugs fail due to poor delivery and which ones fail due to insufficient activity upon entering the tumor.”

    Using SSP, the team found pronounced spatial heterogeneity in both drug delivery and target engagement across head and neck, pancreatic, and other solid tumor types. The data point to a consistent culprit: the stromal architecture, known as the dense, noncancerous tissue surrounding tumors, which acts as a physical barrier that limits antibody penetration.

    “This approach allows us to examine how the drug distributes within the tumor, the cell types with which it interacts, how strongly it engages its molecular target, and how the architecture of the tumor microenvironment shapes its delivery and activity,” Rosenthal said.

    The study included analysis of panitumumab‑IRDye800CW, an antibody used in Phase I trials and which is under investigation for fluorescence‑guided surgery. Rosenthal’s group has long been at the forefront of integrating fluorescence imaging into cancer research and surgical oncology.

    “By directly measuring drug delivery at the site of targeted antibody therapy, SSP can distinguish tumor regions that are biologically unresponsive from those that are simply underexposed to the agent. We hope additional study in larger sample sizes of patients can help further validate the application of SSP to identify barriers to drug efficacy,” Rosenthal added.

    By exposing the physical and biological barriers that shape drug performance in human tissue, the platform offers a path toward designing tools that account for the true complexity of the tumor microenvironment.

    The post Spatial Single‑Cell Platform Reveals Barriers to Antibody Delivery in Solid Tumors appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Lilly, Ascidian Launch Up-to-$1.9B RNA Exon Editor Collaboration Targeting Inherited Kidney Diseases https://edusehat.com/en/lilly-ascidian-launch-up-to-19b-rna-exon-editor-collaboration-targeting-inherited-kidney-diseases https://edusehat.com/en/lilly-ascidian-launch-up-to-19b-rna-exon-editor-collaboration-targeting-inherited-kidney-diseases Wed, 03 Jun 2026 19:10:09 +0700 Edusehat Lilly, Ascidian, Launch, Up-to-1.9B, RNA, Exon, Editor, Collaboration, Targeting, Inherited, Kidney, Diseases Eli Lilly has expanded its genetic medicines pipeline and capabilities, agreeing to partner with Ascidian Therapeutics to develop RNA exon editors intended to treat inherited kidney diseases, through a collaboration that could generate more than $1.9 billion for the Boston biotech.

    The companies have agreed to launch a global research collaboration focused on discovering and developing treatments for undisclosed monogenic kidney diseases, with the option to expand into additional targets.

    At the heart of the collaboration are Ascidian’s RNA exon editors, which are designed to repair genetic instructions causing disease. A single RNA exon editor can address multiple mutations spanning multiple exons, resulting in the editing of multiple disease-causing exons simultaneously.

    “What that allows us to do is to replace many exons at once, thousands of bases at a time, and use endogenous cellular machinery,” Michael Ehlers, MD, PhD, Ascidian’s president and CEO, told GEN. “We’re not editing letters in the genetic code. We’re rewriting whole chapters at the kilobase scale.

    “It’s a very versatile technology to address a fairly wide-ranging set of genetic diseases, and potentially beyond as well,” he added.

    Since only exons need to be replaced within the diseased protein, the exon editing payload is small enough to fit in an adeno-associated virus (AAV) or in other viral or nonviral delivery vehicles, including lipid nanoparticles.

    Michael Ehlers, MD, PhD, president and CEO of Ascidian Therapeutics [Credit: Kevin Trimmer, courtesy Ascidian Therapeutics]
    “The therapeutic is an AAV that expresses the designed RNA exon editor. The AAV infects the target cells. The episome of the AAV dwells in the nucleus and expresses this engineered RNA molecule, our exon editor, that then conducts the trans-splicing,” Ehlers explained. “We very intentionally, across programs and things, really emphasized being a differentiated cargo company in designing these RNAs, and tried where we can to use precedented clinical delivery.”

    During the transcription of DNA into RNA, noncoding introns are usually removed, while exons that remain are spliced together to form messenger RNA (mRNA) that can be translated into protein. Mutations result in malformed proteins that cause disease.

    Ascidian’s RNA exon editors are designed to bind to target pre-mRNA through what the company calls a highly specific binding domain. The editor molecules are delivered as a DNA construct and transcribed into mutation-free, exon-only RNA, designed with a highly specific binding domain.

    Trans-splicing process

    Through a binding process called pre-mRNA trans-splicing, exon editors replace disease-causing exons, leading to what Ascidian said is expression of wild-type mRNA and protein at proper levels, in the right cells at the right time.

    The exon editors are designed to address large genes or genes with high mutational variance.

    “There are other excellent targets which are dominant genes, where maybe you’re uncertain whether the disease-causing mutations are dominant because they’ve got a dominant toxic gain-of-function phenotype, or they’re dominant because it’s a haploinsufficiency. The nice thing about RNA exon editing is that it doesn’t really matter to us. We simply replace the sequence with wild type, so we don’t have to worry about whether you need allele-specific knockdown in those cases, or whether you are at risk of having a haploinsufficiency phenotype,” Ehlers explained.

    The exon editing approach, Ascidian said, represents a sea change for RNA therapy, which has seen treatments incorporating antisense oligonucleotides, adenosine deaminase acting on RNA (ADAR)-mediated editing, and mRNA as applied in vaccines such as those for COVID-19, and in in vivo chimeric antigen receptor T-cell (CAR-T) therapies.

    “RNA exon editing is really quite different, because you can target and dodge RNAs, and instead of editing one base at a time, you’re changing big swaths of sequence at a time, and unlike a lot of other forms of editing, whether DNA or RNA—not all, but for many—there’s no need to introduce exogenous enzymes or exogenous proteins to be able to conduct the editing,” Ehlers said. “That allows Ascidian to use RNA exon editing to go after a different set of diseases, a different set of disease genes, and potentially a larger patient population.”

    In kidney disease, for example, more than 60 genetic diseases are known or suspected to affect the kidneys, with over 3.5 million Americans living with severe inherited kidney disease.

    “I would not say all of them are treatable, but a reasonable portion of them are, and we’re prioritizing some that are clearly important, ones where RNA exon editing is particularly uniquely suited, we believe, to addressing the underlying genetic cause,” Ehlers said.

    Undisclosed targets

    Ascidian has granted Lilly exclusive, target-specific rights to Ascidian’s RNA exon editing technology for undisclosed kidney disease targets. The number of targets was also not disclosed. Ascidian has agreed to lead discovery and specified preclinical activities, with Lilly agreeing to oversee additional preclinical work, clinical development, manufacturing, and commercialization. Ascidian said it retains rights to pursue other kidney-focused targets, independently or with additional partners.

    Ehlers said the Lilly collaboration resulted from conversations with the pharma giant held over several years that started with Ascidian discussing its research, and over time became more focused on the companies developing the research plan and programs that they agreed to work on.

    “About a year and a half ago, I would say, we kind of got really quite a bit more specific about, well, what would be some interesting areas that might fit with Lilly’s strategic interests and capabilities, where Ascidian’s technology and know-how in RNA biology and splicing biology and using RNA exon editing could apply,” Ehlers recalled.

    Genetic medicine expansion

    Ascidian’s RNA research and technologies fit with Lilly’s commitment in recent years to expand in genetic medicines. Lilly launched the $700 million Institute for Genetic Medicine in 2021 in Boston’s Fort Point section as part of a strategy of advancing gene and gene editing therapies, and RNA- and other nucleic acid-based therapeutics. The Institute operates in Boston and New York City, where Lilly-owned neuroscience gene therapy developer Prevail Therapeutics is based. Prevail was acquired in 2021 for up to $1.04 billion.

    A year later, Lilly expanded an RNA-focused collaboration with ProQR into a potential nearly $4 billion partnership to target disorders of the liver and nervous system by applying ProQR’s RNA-editing Axoimer™ platform. Lilly also snapped up another gene therapy developer, buying hearing loss-focused Akouos for up to $610 million, while last year Lilly purchased gene editing therapy developer Verve Therapeutics for up to $1.3 billion

    This year, among its numerous acquisition and collaboration deals, Lilly announced plans in February to buy out circular RNA cell therapy developer Orna Therapeutics for up to $2.4 billion, targeting advancements in cell therapy, and last month acquired nonviral DNA delivery-focused drug developer Engage Biologics for up to $202 million cash.

    In its latest collaboration with Ascidian, Lilly agreed to pay the biotech up to $1.9 billion, to consist of an undisclosed upfront payment and payments tied to achieving development and commercial milestones, as well as tiered royalties on global commercial sales.

    Targeting Stargardt

    Ascidian’s website discloses eight pipeline candidates, the most advanced of which is its sole clinical-phase candidate ACDN-01, a first-in-class RNA exon editing therapy designed to halt the progression of Stargardt disease or other ABCA4 retinopathies by targeting their genetic cause.

    ACDN-01 contains a healthy copy of exons of ABCA4 RNA, designed to replace the sections of the ABCA4 RNA that contain mutations, thus creating healthy ABCA4 RNA in the retina. According to Ascidian, this is intended to produce normal ABCA4 protein that can then help clear the eye of toxic waste products. The FDA has granted ACDN-01 its Fast Track and Rare Pediatric Disease Designations.

    “I anticipate this will be, by and large, the way that we will deliver exon editors. Although, in principle, it can be agnostic to the delivery method, it’s just whatever it takes to get that RNA exon editor into the cell, into the nucleus, effectively trans-splicing at the target.”

    ACDN-01 is under study in the Phase I/II STELLAR trial (NCT06467344), an open-label, single ascending dose clinical study assessing the safety, tolerability, and preliminary efficacy of the treatment candidate when delivered subretinally in participants with ABCA4-related retinopathies. Last month, Ascidian said it had completed the adult dose escalation portion of the STELLAR trial and expanded the study to subjects over 12 years of age.

    “We’re working as effectively as we can to get that up and going, so I’d say in the coming months,” Ehlers said when asked about the timing of the expansion.

    Ascidian is also conducting an observational prescreening study called STARPATH (NCT06445322), which is designed to identify children ages 5+ and adults with Stargardt who may be eligible for future clinical trials evaluating ACDN-01.

    ACDN-01 is among numerous candidates in clinical development to treat Stargardt. These candidates include genetic therapies such as:

    • Ocugen’s OCU410ST (AAV5-hRORA), a modifier gene therapy that uses an adeno-associated virus serotype 5 (AAV5) delivery platform to deliver the RORA (RAR-Related Orphan Receptor A) gene to the retina.
    • SpliceBio’s SB-007, a dual AAV gene therapy designed to restore expression of a functional, full-length ABCA4 protein in the retina through a protein splicing intein platform using two AAV serotype 8 (AAV8) vectors to overcome the size limitations of conventional AAVs, reconstituting biologically active ABCA4 through protein trans-splicing in target photoreceptor cells.
    • VeonGen Therapeutics’ VG801, a dual AAV gene therapy enabled by the company’s vgAAV capsid and vgRNA REVeRT large-gene delivery platform, which delivers the full-length functional ABCA4 gene.

    Single vector approach

    Ehlers said ACDN-01 would stand out from the other genetic therapies because of its single vector approach to delivery: “I’d say the others, because the nature of the ABCA4 gene, it’s too large just for gene replacement in a single vector. All the others have to use dual vector technologies to try to address that.”

    “You can imagine a single vector approach being potentially simpler and more technically feasible,” he added. “But of course, what matters is clinical data, and all these programs will play out in the clinic.”

    Also in Ascidian’s pipeline: A second retinal program in lead identification phase; four neuro and neuromuscular programs in phase from lead identification to lead optimization; and two lead identification-phase programs in undisclosed “other areas.” Ascidian is also partnering with Roche to discover and develop RNA exon editing therapeutics against undisclosed neurological targets, through an up-to-$1.842 billion collaboration ($42 million paid initially) launched in 2024.

    Following two years in stealth mode, Ascidian was formally launched in 2022 by venture capital firm Apple Tree Partners (ATP), which incubated the company and funded it with $50 million in Series A financing. Ehlers led that incubation as the company’s founding CEO, then chaired the company’s board while Romesh Subramanian, PhD, served as Ascidian’s president and CEO. After Subramanian left the company, Ehlers returned to Ascidian’s helm in 2023, when it closed on $40 million in Series A extension funding from ATP, with the goal of financing the development of ACDN-01 and other pipeline programs.

    Since then, Ascidian has grown its workforce to about 40 people, Ehlers said.

    “For a clinical stage editing company, to be in the clinic with $90 million in equity financing and have 40 people plus or minus is no small feat, and I think it has been accomplished by having the focus that we’ve had, and having just one of the best scientific teams that I’ve ever had the fortune of working with,” Ehlers said.

    “We might have little bits of growth here and there to be able to support this [Lilly] collaboration, to be able to expand the technology, but I’m not anticipating substantial growth,” he added. “We’ve built the company to be efficient and focused, and we’re going to maintain that going forward.”

    Lilly’s hot streak

    The Ascidian collaboration continues a hot streak of collaboration and acquisition deals for the pharma giant, which is flush with cash from sales of its blockbuster glucagon-like peptide 1 (GLP-1) receptor agonist drug tirzepatide, marketed for type 2 diabetes as Mounjaro® and for obesity as Zepbound®.

    So far this year, Lilly has either acquired or is acquiring 10 biotechs, most recently three privately-held developers of vaccines for infectious diseases purchased for a combined up to $3.83 billion cash—Vaccine Company for up to $1.55 billion, Curevo for up to $1.5 billion, and LimmaTech Biologics for up to $780 million.

    The Ascidian collaboration is Lilly’s third partnership with a biotech, announced just this week. The other two, totaling a combined amount of up to $4.304 billion-plus, were announced with Asian partners.

    Seoul-based Hanmi Pharmaceutical said Lilly had agreed to license from it the rights to develop, manufacture, and commercialize sonefpeglutide (LAPSGLP-2 analog), a Phase II glucagon-like peptide 2 (GLP-2) receptor agonist, worldwide excluding South Korea. Hanmi is now studying sonefpeglutide in a global Phase II trial (NCT04775706) in short bowel syndrome (SBS).

    Lilly agreed to pay Hanmi $75 million upfront, and up to an additional $1.185 billion in payments tied to achieving clinical development, regulatory approval, and commercialization milestones, plus royalties on sales following product launch.

    Beijing-based Haisco Pharmaceutical Group announced a licensing and research collaboration with Lilly to develop treatments across multiple undisclosed therapeutic areas. Haisco agreed to oversee discovery and identification of up to five “innovative target” programs, while Lilly agreed to lead IND-enabling studies, clinical development, and commercialization. Lilly will obtain exclusive global rights for some programs, as well as exclusive rights worldwide, excluding China, Hong Kong, Macau, and Taiwan, for the other programs.

    Lilly agreed to pay Haisco up to $87 million in upfront and near-term payments, up to $2.967 billion in unspecified milestone payments, and single-digit tiered royalties on future product sales.

    The post Lilly, Ascidian Launch Up-to-$1.9B RNA Exon Editor Collaboration Targeting Inherited Kidney Diseases appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Disease Detection Gets Boost from Keck’s New Brain Reference Map https://edusehat.com/en/disease-detection-gets-boost-from-kecks-new-brain-reference-map https://edusehat.com/en/disease-detection-gets-boost-from-kecks-new-brain-reference-map Wed, 03 Jun 2026 04:40:49 +0700 Edusehat Disease, Detection, Gets, Boost, from, Keck’s, New, Brain, Reference, Map Investigators at the USC Mark and Mary Stevens Neuroimaging and Informatics Institute (Stevens INI) at the Keck School of Medicine say they have created one of the largest reference models ever developed for the human brain, using diffusion MRI scans from more than 54,000 people to chart how the brain’s communication pathways develop, mature, and decline across the lifespan.

    Published in Nature Communications, the study “Lifespan normative modeling of brain microstructure” provides the equivalent of growth charts for the brain’s white matter, the vast network of neural wiring that allows brain regions to communicate, according to the Keck team, which adds that the novel tool offers researchers a new way to detect subtle patterns linked to aging, Alzheimer’s disease, schizophrenia risk, and other neurological and psychiatric conditions.

    “Just as pediatric growth charts help clinicians determine whether a child’s height or weight is developing as expected, these brain charts provide a reference for how the brain’s neural pathways typically change over the lifespan,” said Julio E. Villalón-Reina, MD, PhD, a postdoctoral researcher at the Stevens INI and the study’s first author. “That gives us a powerful new way to identify when an individual’s brain wiring falls outside the expected range.”

    To study white matter, the team used diffusion MRI, an imaging method that tracks how water moves through brain tissue. Because water movement is shaped by microscopic features such as nerve fibers and myelin, diffusion MRI can reveal subtle changes in tissue organization not visible on standard brain scans.

    After compiling diffusion MRI data from 54,583 individuals across 19 international datasets, the researchers built statistical growth and decline charts for the brain’s neural pathways.

    The researchers focused on four widely used measures of white matter microstructure across 21 major brain regions. By modeling how these measures vary by age and sex, they generated lifespan curves and percentile ranges that show what is typical at different stages of life.

    Statistical charts compiled from a large population allow brain abnormalities to be detected in new individuals. [Stevens INI]
    Statistical charts compiled from a large population allow brain abnormalities to be detected in new individuals. [Stevens INI]
    The results revealed that white matter follows distinct developmental and aging trajectories, with some measures reaching peak maturity in early adulthood and others later in midlife.

    “Brain development and brain aging are not uniform processes,” continued Villalón-Reina. “The brain’s neural pathways mature on distinct timelines, and some are more vulnerable to decline than others. Our model reveals this structure by merging data on a truly global scale.”

    The scientists also discovered evidence for a longstanding theory of brain aging, sometimes described as last in, first out. According to this theory, brain pathways that develop last in childhood and adolescence tend to be more susceptible to decline in older age. The researchers observed that white matter regions that mature later did indeed decline faster in old age, offering new insight linking brain development and aging.

    To demonstrate the model’s practical value, the researchers applied it to clinical datasets from people with mild cognitive impairment, dementia, and 22q11.2 deletion syndrome, a genetic condition that increases risk of schizophrenia.

    In each case, the model identified alterations in the brain’s circuitry that deviated from age-expected norms. Importantly, these deviations were not identical across individuals with the same diagnosis, highlighting the value of a person-specific approach.

    Paul M. Thompson, PhD, associate director of the Stevens INI and senior author of the study
    Paul M. Thompson, PhD, associate director of the Stevens INI and senior author of the study

    “This monumental study took seven years to complete,” explained Paul M. Thompson, PhD, associate director of the Stevens INI and senior author of the study. “The vast scale of the data and the fine scale of the brain features assessed means we can now evaluate your neural pathways relative to other people of the same age, sex, and demographics. We can see how your brain differs from what we would expect for a person of your age and sex, giving us a tool to use in clinical trials of treatments for dozens of brain diseases.”

    When applied to people with dementia and mild cognitive impairment, the model detected atypical white matter patterns in brain regions involved in memory and interregional communication. In people with 22q11.2 deletion syndrome, it identified deviations in multiple key neural pathways, helping researchers discover which brain systems develop differently.

    The reference charts may also help researchers evaluate treatments by tracking whether a person’s white matter measures move closer to the expected range, or whether a treatment slows the shift away from healthy patterns over time. The charts will now be used to compare more than 30 brain diseases and conditions, offering a common framework for studying how different disorders emerge, progress, and respond to intervention.

    The models are also a publicly available resource that can be extended as additional brain imaging data become available. The methods are now being used to study neurological, psychiatric, and neurodevelopmental disorders by providing a common reference standard for white matter microstructure across the lifespan.

    “This study demonstrates the power of large-scale, international data sharing to create tools the entire research community can use,” pointed out Arthur W. Toga, PhD, director of the Stevens INI and provost professor at USC. “By establishing a lifespan framework for the brain’s communication pathways, this work opens new opportunities to detect subtle disease-related changes, compare conditions more rigorously, and move toward a more individualized understanding of brain health.”

     

     

     

     

    The post Disease Detection Gets Boost from Keck’s New Brain Reference Map appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Fulcrum Halts Development of SCD Candidate Pociredir, Sets Strategic Review https://edusehat.com/en/fulcrum-halts-development-of-scd-candidate-pociredir-sets-strategic-review https://edusehat.com/en/fulcrum-halts-development-of-scd-candidate-pociredir-sets-strategic-review Wed, 03 Jun 2026 04:40:48 +0700 Edusehat Fulcrum, Halts, Development, SCD, Candidate, Pociredir, Sets, Strategic, Review Fulcrum Therapeutics said today it is scrapping its lead pipeline program to develop pociredir as a treatment for sickle cell disease (SCD) and launching a “comprehensive” review of strategic alternatives, after the FDA told the company it had heightened concerns about the drug’s risks and benefits in fighting the disease.

    Those concerns, which the agency raised with Fulcrum executives at a recent end-of-phase meeting, stemmed from an unexpectedly high rate of secondary blood cancers seen with another PRC2 inhibitor—Ipsen’s Tazverik® (tazemetostat), indicated to treat follicular lymphoma and epithelioid sarcoma, the company disclosed, based on meeting minutes received May 28.

    Ipsen voluntarily withdrew Tazverik from the market in March following adverse events of secondary blood cancers emerging from the ongoing Phase Ib/III SYMPHONY-1 trial (NCT04224493), which evaluated the drug in combination with lenalidomide plus rituximab (R2) vs R2 in follicular lymphoma. Ipsen inherited the drug when it acquired its developer Epizyme in 2022 for $247 million. Tazverik last year generated €40.6 million ($47.2 million) for Ipsen and another $2.5 million for Hutchmed, which marketed the drug in China. The withdrawal also affected Eisai, since it marketed Tazverik in Japan and manufactured the drug there.

    Pociredir is an oral small molecule polycomb repressive complex 2 (PRC2) inhibitor targeting embryonic ectoderm development (EED), and was discovered using Fulcrum’s discovery technology. Fulcrum has reasoned that inhibiting EED leads to potent downregulation of key fetal globin repressors including BCL11A, thereby causing an increase in fetal hemoglobin (HbF).

    “Fulcrum submitted information to FDA supporting the position that mechanistic differences between EED (pociredir’s target) and EZH2 (tazemetostat’s target), which perform different biological roles, were relevant to the benefit-risk assessment,” Fulcrum said in a statement. “While no new safety signals have been observed to date with pociredir, the FDA raised concerns regarding the potential malignancy risk associated with pociredir’s inhibition of the PRC2 complex.

    The agency considered Fulcrum’s position before rebuffing the company, concluding that any drug intervention targeting the PRC2 complex carries equivalent malignancy risk “regardless of the specific subunit engaged,” Fulcrum continued, based on pociredir’s previously disclosed preclinical malignancy observations.

    That left no viable regulatory path forward for further clinical development of pociredir, Fulcrum concluded.

    No path forward

    “Following a thorough review of regulatory feedback, the totality of available data, and the implications for a viable regulatory path, we have made the very difficult decision to discontinue development of pociredir,” stated Alex C. Sapir, Fulcrum’s president and CEO. “We arrived at this decision after discussion with the FDA, and despite robust elevations in fetal hemoglobin seen with pociredir and the potential for clinical benefit, we do not see a path forward with pociredir.”

    “We know the SCD community has faced many disappointments and setbacks related to innovation for this devastating disease,” Sapir added. “We are not only humbled but forever grateful to the SCD warriors, investigators, and broader SCD community who have worked tirelessly alongside Fulcrum to evaluate new treatment options for this devastating disease.”

    Pociredir is not the first SCD therapy to be scrapped. In 2024, Pfizer withdrew Oxbryta® (voxelotor) from the market, citing deaths and vaso-occlusive crises occurring in patients given Oxbryta in clinical studies. Hours before Pfizer’s announcement, the European Medicines Agency (EMA) disclosed findings from two Phase III trials of Oxbryta in which a total of 18 deaths occurred—all but two of them reported in patients who were dosed with the drug.

    Pfizer inherited Oxbryta when it acquired the drug’s original developer, Global Blood Therapeutics (GBT), for $5.4 billion, a deal completed in 2022.

    Vivien Sheehan, MD, PhD, director of Translational Sickle Cell Disease Research at Emory University School of Medicine, told GEN Fulcrum’s data was “reasonable, although not game changing.”

    “I don’t take it [Fulcrum’s halt to pociredir development] as an abandonment of SCD,” said Sheehan, who is also a member of the Discovery and Developmental Therapeutics Research Program at Winship Cancer Institute of Emory University. “There may not have been a path forward, and more efficacious drugs in the pipeline may have also influenced the decision.”

    She expressed greater enthusiasm for a potential SCD treatment being developed by Bristol Myers Squibb (BMS)—BMS-986470, an oral HbF-activating cereblon (CRBN) E3 ligase modulator (CELMoD™) agent designed as a potential first-in-class degrader of both transcription factors zinc finger and BTB domain containing 7A (ZBTB7A) and widely interspaced zinc finger (WIZ).

    BMS-986470 is now under study in an ongoing Phase I/II trial (NCT06481306) designed to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with SCD. The study’s estimated primary completion date is January 6, 2027, according to ClinicalTrials.gov.

    A 2024 preclinical study by a team of BMS researchers showed BMS-986470 to have generated “robust γ -globin induction activity leading to HbF levels predicted to significantly ameliorate SCD pathology.”

    $1.5B peak sales forecast

    In ending development of pociredir, Fulcrum has scrapped a drug that stood to generate as much as $1.5 billion in projected peak sales by 2038, according to BofA Securities, which predicted a 2029 commercial launch for the drug.

    Investors responded to Fulcrum’s halt to development of pociredir with a stock selloff that sent shares of Fulcrum on the Nasdaq Global Market plummeting 51% Tuesday, to $3.13 from yesterday’s closing price of $6.42.

    As a result of scraping development of pociredir, Fulcrum said, it will explore potential strategic alternatives, “including, but not limited to, a merger, acquisition, business combination, or other strategic transactions involving the company or its assets.”

    Fulcrum said it has also begun efforts to “significantly” reduce its operating expenses and preserve capital. Fulcrum finished the first quarter with $333.3 million in cash, cash equivalents, and marketable securities.

    “We believe that our existing cash, cash equivalents, and marketable securities as of March 31, 2026 will enable us to fund our operating expenses and capital expenditure requirements into 2029,” Fulcrum stated in its Form 10-Q regulatory filing for the first quarter.

    “With a strong balance sheet extending our cash runway into 2029, we are well positioned to advance pociredir through the next phase of clinical development,” Fulcrum stated in its April 27 press release.

    The company has not set a timeline for completing its strategic review, adding that it does not intend to provide further updates “unless and until the board of directors has approved a course of action, the review process is concluded, or other disclosure is otherwise determined to be appropriate.”

    Positive interim data

    As late as April 27, when it held its quarterly earnings call with analysts to discuss first quarter results, Fulcrum had conveyed optimism about pociredir’s clinical prospects.

    During the call, Sapir cited Fulcrum’s announcement of positive interim data from its Phase Ib PIONEER trial (NCT05169580) in February, which showed:

    • A mean absolute HbF increase of 12.2% at 12 weeks of treatment with pociredir, rising from a baseline of 7.1% to 19.3%—what the company said represented “a rapid, robust, and clinically relevant response,” with progression toward pan-cellular HbF induction as F-cells increased from 31% to 63%.
    • Absolute HbF levels ≥20% in 7 of 12 patients (58%), with all patients achieving at least a 6.5% absolute increase in HbF.
    • Improvements in markers of hemolysis, improved erythropoiesis, and a >1 g/dL increase in total hemoglobin.
    • Zero vaso-occlusive crises (VOCs) during the treatment period reported in seven of 12 patients (58%).

    “Importantly, pociredir has continued to be generally well tolerated with no treatment-related serious adverse events reported to date,” Sapir told analysts. “Taken together, these data reinforce our conviction in pociredir’s potential to address the underlying biology of sickle cell disease—and support our belief that pociredir has the potential to represent a differentiated, once-daily oral treatment option for patients.”

    The post Fulcrum Halts Development of SCD Candidate Pociredir, Sets Strategic Review appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cross-Reactive T Cells Could Point to Broad Vaccines or Treatments for Measles, Nipah Virus https://edusehat.com/en/cross-reactive-t-cells-could-point-to-broad-vaccines-or-treatments-for-measles-nipah-virus https://edusehat.com/en/cross-reactive-t-cells-could-point-to-broad-vaccines-or-treatments-for-measles-nipah-virus Wed, 03 Jun 2026 04:40:46 +0700 Edusehat Cross-Reactive, Cells, Could, Point, Broad, Vaccines, Treatments, for, Measles, Nipah, Virus T cells are some of the immune system’s most important fighters. They can stop tumor growth and fight off severe infections. Scientists at La Jolla Institute for Immunology (LJI) have now reported a study indicating how T cells target paramyxoviruses, a viral family that includes measles virus and Nipah virus.

    Paramyxoviruses are pathogens of pandemic concern. Measles virus is highly infectious, and Nipah virus has a high mortality rate. The new study shows how we might harness T cells to save lives. Headed by Alessandro Sette, PhD, the team systematically mapped human CD4+ T cell epitopes across Nipah and measles viruses, and analyzed T cells from donors who had previously received the MMR vaccine that protects against measles, and another paramyxovirus, mumps (as well as rubella), and who had not been exposed to Nipah virus. Their experiments showed that the two paramyxoviruses had conserved T cell epitopes (CTERs) in common, and that cross-reactive T cells can recognize multiple paramyxovirus species at once.

    Instead of vaccinating against one virus at a time, the researchers found that activating these cross-reactive T cells may protect against the wider paramyxovirus family. This broad protection is essential when you don’t know which virus will strike next.

    The discovery may guide the development of new vaccines and therapies that stop measles, Nipah, and other paramyxovirus infections before they turn deadly. “No one knows which particular viral species or strain of a virus might be responsible for an outbreak, as we’ve seen in the recent cases of Andes hantavirus,” Sette said.

    Sette and colleagues reported on their findings in Cell Reports Medicine, in a paper titled “Comprehensive mapping of human CD4+ T cell epitopes for Nipah and measles as prototype Paramyxoviruses,” concluding, “Collectively, these findings support the concept that CTER-based immunogen design can both broaden protective coverage and strategically harness existing population immunity while complementing neutralizing antibody-based vaccine approaches.”

    LJI Professor Alessandro Sette, Dr.Biol.Sci. [La Jolla Institute for Immunology]
    LJI Professor Alessandro Sette, PhD [La Jolla Institute for Immunology]
    The Paramyxoviridae family, which includes measles and Nipah viruses, represents “… a plethora of viruses that impact global human health,” the authors wrote. “Understanding adaptive immune responses to these viruses is critical for characterizing host-pathogen interactions and evaluating vaccine performance.”

    A part of the adaptive immune system T cells learn to target a specific threat. A T cell might respond to influenza virus infection but not malaria parasite infection. To do this T cells recognize specific epitopes on the pathogen. In general, T cell epitopes on one pathogen look very different from T cell epitopes on another pathogen.

    LJI Research Assistant Professor Alba Grifoni, Ph.D. [La Jolla Institute for Immunology]
    LJI Research Assistant Professor Alba Grifoni, PhD [La Jolla Institute for Immunology]
    But viruses may retain some “conserved” features that remain unchanged within viral families. LJI scientists have shown that some T cells can cross-react to different viruses, as long as the viruses share similar epitopes. In a series of landmark studies during the COVID-19 pandemic, Sette, LJI research assistant professor Alba Grifoni, PhD, LJI assistant professor Daniela Weiskopf, PhD, and professor and chief scientific officer Shane Crotty, PhD, showed that cross-reactive T cells can recognize the family resemblance between different coronaviruses. A person who had previously contracted a common cold coronavirus may already have T cells primed to recognize SARS-CoV-2, the coronavirus that causes COVID-19.

    More recently, Sette and Grifoni demonstrated that cross-reactive T cells may offer broad protection against the deadly Lassa virus and the wider viral family of arenaviruses. Their findings suggested that future vaccines and therapies could activate these cross-reactive T cells to protect against many dangerous viruses at once. Each study makes it clear that cross-reactive T cells are key to stopping emerging viruses.

    Measles is a threat worldwide, and while an effective vaccine is available, the authors cited figures indicating that there were over 10 million estimated infections worldwide in 2023. “Measles remains one of the main causes of morbidity and mortality in children, due to secondary infections from measles-induced immune suppression,” they noted. People in Southeast Asia also have to keep watch for a related paramyxovirus threat, Nipah virus, which is spread by bats. Cases are rare but can be deadly. “Nipah virus is another Paramyxovirus of concern due to high mortality rates, often mediated by fatal encephalitis,” the investigators wrote. Nipah virus has a fatality rate of 40-75%, which is much higher than measles. “Outbreaks are becoming more and more frequent, especially in the Malaysian region,” said Grifoni.

    The new LJI study suggests cross-reactive T cells may be just the weapons we need to combat the dangerous paramyxovirus family. The scientists worked with LJI’s John and Susan Major Center for Clinical Investigation to collect and analyze T cells from the blood of 31 study participants. These study participants had received their MMR vaccines, which protect against severe infection from the measles and mumps viruses (both are paramyxoviruses) and the rubella virus. As a result, the blood samples contained T cells that were ready to fight measles infection.

    LJI postdoctoral fellow Alison Tarke, PhD, and LJI senior staff scientist Ricardo Da Silva Antunes, PhD, led experiments to map the T cell epitopes on measles virus. These findings were important on their own. “Even though measles has been studied for quite some time, and there is a vaccine for measles, there was not a lot known about the specific T-cell response elicited by the measles vaccine,” Sette commented.

    Tarke and the LJI team then tested how these same T cells reacted to Nipah virus. From blood tests, the scientists knew that the study participants had never been infected with Nipah virus, yet they found that measles-fighting T cells could cross-react and also recognize Nipah virus. The two paramyxoviruses had conserved T cell epitope regions (CTERs) in common. “Focusing immune responses on these conserved regions could have a broad, protective capacity for the whole viral family,” says Sette. The authors added,

    The new study is the first to map T cell epitopes on Nipah virus. The researchers were able to zero in on a specific epitope shared between measles and Nipah viruses: a region of the viral fusion or “F” protein. A large number of cross-reactive T cells targeted this relatively small, conserved viral structure. “It appears that if someone is vaccinated against measles, their T cells will have some degree of cross-reactivity to Nipah,” said Sette. “That raises the possibility that during a Nipah outbreak, one could perhaps vaccinate people with a measles vaccine, and this cross-reactivity could potentially offer some benefit.”

    The authors further noted, “In light of these findings, current Nipah vaccine candidates, many of which focus primarily on whole-protein antigens selected to maximize neutralizing antibody responses, particularly F and G glycoproteins, could potentially be optimized by incorporating conserved T cell epitope regions.” Added Grifoni, “Activating T cells can be your first line of defense when you don’t know what’s going to be thrown at you.”

    In their paper the team concluded “With specific regard to vaccine strategies targeting Nipah or other paramyxoviruses, one anticipated outcome of focusing on CTERs, particularly those shared with measles and mumps viruses, is the potential to boost preexisting cross-reactive memory T cell responses in populations where MMR vaccination is widespread.”

    The post Cross-Reactive T Cells Could Point to Broad Vaccines or Treatments for Measles, Nipah Virus appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Kinase Droplets Activate Growth Signals, Path for Cancer Therapy https://edusehat.com/en/kinase-droplets-activate-growth-signals-path-for-cancer-therapy https://edusehat.com/en/kinase-droplets-activate-growth-signals-path-for-cancer-therapy Wed, 03 Jun 2026 00:50:09 +0700 Edusehat Kinase, Droplets, Activate, Growth, Signals, Path, for, Cancer, Therapy A new study published in Cell Reports titled, “Kinase condensates enrich ATP and trigger autophosphorylation,” suggests that cellular phase separation, a mechanism that organizes biomolecules into dense, liquid-like condensates, may play a previously underappreciated role in regulating kinase activity. The findings suggest that aberrant condensate formation could contribute to oncogenic signaling while also offering new opportunities for drug targeting. 

    “Many biological molecules have this propensity to spontaneously separate,” said Lindsay Case, PhD, assistant professor of biology at Massachusetts Institute of Technology (MIT) and corresponding author of the study. “We were really interested in asking, if we have these kinases forming droplets, what is the consequence of that in the context of signaling?” 

    Phase separation occurs when proteins condense into highly concentrated liquid-like droplets within cells, analogous to oil droplets separating from vinegar. Although biomolecular condensates have emerged as important organizers of cellular processes, their impact on kinase signaling has remained incompletely understood. 

    The researchers examined three kinases: focal adhesion kinase (FAK), Mst2, and Abl. Across all three systems, condensate formation increased kinase activity by concentrating enzymes and substrates, thereby promoting phosphorylation reactions. 

    For FAK, the team found that elevated protein levels were sufficient to drive droplet formation and activate downstream growth signaling. The findings raise the possibility that FAK overexpression in tumors could promote constitutive signaling through condensate formation, potentially contributing to cancer progression and metastasis. 

    “It was surprising that just by condensing this protein into a droplet, you can actually turn on a signaling pathway that should be turned off,” said Case. “If FAK concentration is too high, you’re always getting these droplets and you’re always signaling, regardless of what the receptors that are supposed to be controlling this are doing.” 

    Mst2 and Abl also phase separated at high concentrations, which led to increased activity. For Mst2, phase separation is a strategy that healthy cells use to control the Hippo signaling pathway, which promotes cell growth and survival. Phase separation can also lead both enzymes to phosphorylate additional targets, and activate different signaling pathways. 

    “It’s not just that you’re getting faster phosphorylation, but in those cases, the patterns of what is actually getting phosphorylated were very different inside of the droplet compared to what might be happening in a non-droplet context,” Case says. “The kinase is able to phosphorylate amino acid residues beyond the set of canonical sites that have been described before.” 

    Mechanistically, the team found that kinase condensates selectively concentrate ATP, the phosphate donor required for kinase activity. Positively charged regions within kinases appear to recruit negatively charged ATP molecules to support phosphorylation. 

    Using machine-learning analysis, the investigators predicted that approximately 45% of the roughly 500 human kinases possess the molecular features needed to form similar condensates. The findings suggest that phase separation may represent a widespread regulatory mechanism that could influence both normal cellular signaling and disease-associated kinase activity. 

    In future work, Case hopes to explore designing drugs that could mimic ATP’s ability to be attracted into droplets within a cell, which could reduce side effects.

    The post Kinase Droplets Activate Growth Signals, Path for Cancer Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Bio-IT World Keynote Highlights Collaborative Intelligence in AI-Driven Drug Discovery https://edusehat.com/en/bio-it-world-keynote-highlights-collaborative-intelligence-in-ai-driven-drug-discovery https://edusehat.com/en/bio-it-world-keynote-highlights-collaborative-intelligence-in-ai-driven-drug-discovery Tue, 02 Jun 2026 21:10:12 +0700 Edusehat Bio-IT, World, Keynote, Highlights, Collaborative, Intelligence, AI-Driven, Drug, Discovery BOSTON—A critical part of the conversation around the use of artificial intelligence (AI) in drug discovery focuses on the development of the foundation models that underpin AI-based applications and workflows. There are also discussions about federated learning and how it provides a secure path to accessing critical training data for AI models. These two themes underpinned the keynote panel that kicked off the second day of Bio-IT World Conference 2026, which took place last month in Boston. 

    Through presentations and a group discussion, the six-person panel painted a picture of the different types of foundation models and federated learning approaches, as well as ways to optimize AI’s performance for specific projects. Importantly, they discussed the AI Structural Biology (AISB) initiative, which provides a platform for pooling proprietary protein-ligand structure data to train OpenFold3, an AI model designed to precisely predict molecular interactions. In fact, several members of the panel were either directly or indirectly involved in the OpenFold consortium.   

    Woody Sherman, PhD Founder and Chief Innovation Officer, PsiThera [Uduak Thomas]
    Woody Sherman, PhD, founder and chief innovation officer, PsiThera [Uduak Thomas]

    That group included Woody Sherman, PhD, founder and chief innovation officer at PsiThera, who serves as chair of the OpenFold executive committee. Sherman reiterated the benefits of open-source platforms and how they are making inroads into the drug discovery space.   

    “We’re going to need these open platforms that we can all build on,” he said. “We can’t all be building our own foundation models from scratch. It just doesn’t make sense as an ecosystem. It is important to have these open platforms so that we can interact precompetitively, build the best foundation models, and then “we can get into federated learning.”    

    From AlphaFold to OpenFold 

    The non-profit OpenFold Consortium consists of scientists from over 40 technology companies, startups, pharma companies, and academic institutions. It builds on a lot of the progress made in the 2010s in terms of predicting protein structures from sequences, “a foundational problem in biochemistry.” That progress was quantified at least in part by efforts like the Critical Assessment of Structure Prediction (CAS) competitions, said Mohammed AlQuraishi, PhD, an assistant professor of systems biology at Columbia University, during his presentation.  

    The image shows Mohammed AlQuraishi, PhD Assistant Professor, Systems Biology Columbia University, one of the keynote speakers at the recent Bio-IT World Conference
    Mohammed AlQuraishi, PhD,
    assistant professor, systems biology,
    Columbia University [Uduak Thomas]

    AlphaFold and later iterations of the platform “compressed decades of progress in about four years,” he said. Besides reliably predicting protein structures, AlphaFold provided “calibrated predictions” that gave biologists a sense of the accuracy of its predictions. But there were limitations.   

    “It did not really have an understanding of anything other than just protein structure,” meaning it missed things like ions that were also part of the structure, he said. It also struggled to handle things like protein complexes, ligands, and cofactors. Another challenge was that although the computational models could predict targets that were closer to the training dataset used, their ability to make viable predictions dropped the further away the targets were from the training dataset. Additionally, “these models have limited ability to capture conformational changes,” AlQuraishi noted. “This becomes a major bottleneck in being able to reliably model allosteric modulators or cryptic pockets or similar types of systems.” Besides the technical limitations, there were also licensing limitations to consider.  

    AlQuraishi positioned OpenFold as an open source, high-performance, and reproducible alternative to AlphaFold that serves as a common platform for innovation for the community. “Partly it’s a code base, essentially a set of tools that allow [scientists] to build these types of models and extend them and apply them,” AlQuraishi explained. “It’s also an academic-industry consortium that provides a steerable mechanism for industry to support science that is open source and that’s broadly useful, but it’s also in tune with the needs of industry.”

    Federated learning and foundation models 

    The next set of presentations made the argument for using federated learning to leverage proprietary biopharma datasets to train AI models. The presentation from Jonathan Gilbert, PhD, senior director, ecosystem growth and contributor partnerships at Eli Lilly, offered an example of how the pharma company has used federated learning to improve model predictions in different contexts.  

    “It’s not surprising that companies are very sensitive to the proprietary data that they’ve spent incredible investments generating,” he said. With federated learning, models are trained in the environment where the data is housed, making it possible to “improve model performance while maintaining the privacy of the individual training sets.”   

    Eli Lilly launched the TuneLab platform in 2025, through which it provides access to its own AI and machine learning models to biotech companies at no cost—although those that choose to use the models are expected to contribute datasets to help improve them. “These are the same models that we use every day,” he said. “These models have been trained on decades of internal data sets. That’s maybe over a billion dollars in data that have been brought into models by Lilly.” 

    Jonathan Gilbert, PhD Senior Director, Ecosystem Growth and Contributor Partnerships, Eli Lilly and Company. [Uduak Thomas]
    Jonathan Gilbert, PhD, senior director, Ecosystem Growth and Contributor Partnerships, Eli Lilly and Company. [Uduak Thomas]

    Gilbert noted that since its launch, the appetite for TuneLab has been quite strong. At the time of the presentation, there were more than 75 partners in TuneLab, and it was being used in dozens of countries across three continents. Furthermore, during the meeting, Eli Lilly and Collaborative Drug Discovery (CDD), a provider of data management solutions for pharma and biotech, announced an agreement to integrate TuneLab into both the core and AI modules within the CDD Vault platform.  

    For now, TuneLab is focused on models for small molecules and antibody development, but there are plans to release additional models in the near future. Lilly is also working on additional partnerships similar to the one with Collaborative Drug Discovery. “This is an active work in progress and [we are] thinking [about] how we can scale this,” Gilbert said. And how can “[we] build a community to improve those models such that we can create medicines faster for more people.” 

    The presentation from José-Tomás Prieto, PhD, director of AI programs at Apheris, built on Gilbert’s presentation but focused on the complexities of implementing industrial federated learning setups. The key takeaway from his talk was that successfully implementing federated learning at an industrial scale is not a plug-and-play capability but rather a process that requires engineering rigor, data preparation without centralization, and enterprise-level deployment strategies. His company, Apheris, has experience with this process as they provide solutions that power federated networks for drug discovery. 

    José-Tomás Prieto, PhD Director of AI Programs Apheris. [Uduak Thomas]
    José-Tomás Prieto, PhD, director of AI programs, Apheris [Uduak Thomas]

    One of the networks that they support is the AI Structural Biology Network, a collaboration that brings together several of the top 20 biopharma companies. Its intent is to allow AI models designed to predict the 3D structure of molecule complexes to be trained on proprietary protein structure data. The common denominator for these and other networks that Apheris supports is that the models are trained on proprietary data in a secure way, so the data never leaves the environments of any of the nodes in the network.  

    “It’s obvious that there’s a lot of public data, but the public data skews toward well-characterized targets,” Prieto said. “The industry data complements that view, with more diverse data and sometimes higher quality data. And if there is something to learn about the AI world today is that you cannot necessarily model your way out of a data problem, and you can’t buy this data either.” Federated learning provides a solution to that problem. “It’s quite remarkable that a couple of years ago … it was mostly IT people making the decision of whether to use federated learning products,” he noted. “Today, we have business leaders trying to get ahold of this technology and leverage the power.” 

    Prieto also discussed some important considerations for building federated networks. To provide a sense of the complexity involved, “each one of these companies have their own network constraints, their own firewall rules, their own compute window that they have to negotiate with the cloud providers to make sure that the compute comes online at the right time.”  

    It is also important to consider “that data preparation without centralization is a new paradigm,” he continued. “Each company has [their] own ways of organizing the data or harmonizing your data,” as well as their own standards, but at the same time you have to have comparable training setups so that the foundation models can actually learn from this.” Furthermore, “your federated learning partner has to be able to work with your processes, has to be able to understand how to streamline the reviews, the security [and] the privacy requirements” among other things before projects can move forward. 

    A key point that both Prieto and Arman Zaribafiyan, PhD, head of strategic alliances, AI simulation at SandboxAQ, emphasized was that while federated models provide broad generalizations, fine-tuning them on specific, project-level data is crucial for translating model performance into practical impact for drug programs.  

    SandboxAQ has worked with the OpenFold consortium on its models and co-folding models, among other projects. “We are really living in exciting times when it comes to ML-accelerated drug discovery,” Zaribafiyan said. “There’s really an explosion of new models we see every day. And what we see at Sandbox with our partners in large pharma and biotech companies is that it’s getting a little bit overwhelming and harder to put these models into good use.” Furthermore, “a lot of these models are amazing in achieving great results on benchmarks, and it’s fascinating for publishing papers, but they fail to generalize to real drug discovery use cases.”

    Arman Zaribafiyan, PhD, Head of Strategic Alliances, AI Simulation SandboxAQ. [Uduak Thomas]
    Arman Zaribafiyan, PhD, head of strategic alliances, AI simulation, SandboxAQ [Uduak Thomas]

    Commenting on some of the lessons SandboxAQ has learned through its partnerships, Zaribafiyan noted that “fine-tuning could help a lot to bridge this gap.” SandboxAQ and others have published data showing that “even a small fine-tuning effort can dramatically change the predictive accuracy of these models.” Over the next few years, he believes these are going to become the norm: “We’re going to see more and more of these federated platforms we use for both pooling data but also for fine-tuning these models on project-specific data.”   

    Zaribafiyan closed his presentation with an announcement of a new platform from SandboxAq that connects quantitative models for drug discovery to large language models, allowing scientists to launch and run simulations and workflows using plain English, much like prompts written for ChatGPT. “No code required,” he said. 

    Foundational models at work in crop science and drug development 

    Christina Taylor, PhD, senior science fellow and computational molecular design lead at Bayer, focused on how her company has leveraged foundational models and AI to drive decisions in crop science and pharma.

    Christina Taylor, PhD Senior Science Fellow and Computational Molecular Design Lead Bayer. [Uduak Thomas]
    Christina Taylor, PhD, senior science fellow and computational molecular design lead, Bayer [Uduak Thomas]

    “I think that this community-driven software has really allowed faster innovation in the field overall,” and “sharing some of these foundational architectures allows everyone to be able to drive biomolecular AI work,” and “ has driven some of the very quick advancements we’ve seen in the field over the past few years.” Community projects like this also save time and are more sustainable since “everybody doesn’t need to be training their own foundational models.”   

    To date, these models have helped Taylor and her team better solve crystal structures. “One of the big problems with solving crystal structures is actually determining the phase, and by doing protein, we’re able to actually solve these structures faster and more efficiently,” she said. “Another thing is taking these foundational models and fine-tuning them … we’re using that quite regularly to improve our development of biomolecular pharmaceuticals as well as some of our crop science traits.” Other applications that Taylor and her team have used the models for include studying protein-protein interaction as well as for modeling enzyme catalysis.

    The post Bio-IT World Keynote Highlights Collaborative Intelligence in AI-Driven Drug Discovery appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Circio and GenAssist Collaborate on Gene Therapy for Muscle Disease and In Vivo Cell Therapy https://edusehat.com/en/circio-and-genassist-collaborate-on-gene-therapy-for-muscle-disease-and-in-vivo-cell-therapy https://edusehat.com/en/circio-and-genassist-collaborate-on-gene-therapy-for-muscle-disease-and-in-vivo-cell-therapy Tue, 02 Jun 2026 21:10:11 +0700 Edusehat Circio, and, GenAssist, Collaborate, Gene, Therapy, for, Muscle, Disease, and, Vivo, Cell, Therapy Oslo, Norway-based Circio and Suzhou, China-based GenAssist entered into a research collaboration to develop circVec-enhanced AAV vectors specifically engineered for in vivo cell therapy and targeted, low dose systemic gene therapy.

    Genetic muscle disease is an area of major unmet medical need, where current gene therapy’s high dosing requirements are associated with severe toxicity. By integrating Circio’s and GenAssist’s complementary technologies, the parties aim to develop joint next generation of AAV gene therapy candidates, according to a Circio spokesperson. The focus is on addressing genetic muscle conditions where high and broad muscle-specific expression is required at substantially lower therapeutic AAV doses than can be achieved by conventional AAV gene therapy.

    “Our second-generation AAV platform establishes a new benchmark for safety, utilizing highly tissue-specific, de-targeted capsids to dramatically lower systemic dosing while eliminating off-target toxicity,” said Chunyan He, PhD, CEO of GenAssist. “Through our collaboration with Circio, we integrate their unique circular RNA technology. This partnership directly addresses the core demands of next-generation genetic medicine, overcoming the traditional dose-expression trade-off to deliver safer and more effective therapies.”

    In addition, Circio and GenAssist will explore the potential of generating joint in vivo CAR T candidates for oncology and autoimmune applications. The collaboration will involve production of novel AAVs combining GenAssist´s T-cell targeting with the circVec expression cassette from Circio. The combined AAVs will subsequently be tested in vitro and in vivo, and if successful, candidates for further development will be nominated for preclinical development.

    “The targeted AAVs developed by GenAssist have the ability to specifically and efficiently transduce muscle or T-cells upon systemic delivery with near-complete liver de-targeting,” added Thomas Hansen, PhD, CTO of Circio. “The partnership between Circio and GenAssist will aim to evaluate whether the enhanced circVec expression acts synergistically with these targeted capsids and promoters.

    “This fits perfectly into Circio’s strategy of testing circVec in multiple tissues using different AAV variants, both internally and externally. This will allow us to identify new therapeutic avenues where circVec delivers a benefit, and forge partnerships potentially enabling multiple future development opportunities. China is a particularly interesting geography, with cutting edge science and accelerated pathways to establish early clinical data.”

    The post Circio and GenAssist Collaborate on Gene Therapy for Muscle Disease and In Vivo Cell Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Low-Cost, Portable Biotech Tools Improve Access to Bioresearch and Diagnostics https://edusehat.com/en/low-cost-portable-biotech-tools-improve-access-to-bioresearch-and-diagnostics https://edusehat.com/en/low-cost-portable-biotech-tools-improve-access-to-bioresearch-and-diagnostics Tue, 02 Jun 2026 21:10:10 +0700 Edusehat Low-Cost, Portable, Biotech, Tools, Improve, Access, Bioresearch, and, Diagnostics A global research team headed by scientists at University of Toronto’s Leslie Dan Faculty of Pharmacy has demonstrated the effectiveness of a suite of low-cost, portable biotechnology tools that are designed to improve access to laboratory research and diagnostics in resource-limited settings.

    The newly reported study highlights how decentralized biomanufacturing tools and freeze-dried reagents can help researchers produce high-value biological materials locally—reducing reliance on fragile international supply chains and expanding access to life sciences innovation globally.

    “For labs in low- and middle-income countries [LMICs], access to high-quality supplies and equipment is a chronic problem,” says research lead Keith Pardee, PhD, associate professor at the Leslie Dan Faculty of Pharmacy Pardee. “Shipping can take a long time, it’s expensive, and products often require a cold chain to retain their effectiveness. This research is in response to those challenges to develop tools that are more accessible for labs in lower-resource settings and improve research equity.”

    Pardee, alongside collaborators including Camila González, PhD, at the Universidad de los Andes, Bogotá, Fernán Federici, PhD, at Millennium Institute for Integrative Biology (iBio), Santiago, and Lindomar Pena, PhD, at Aggeu Magalhães Institute (IAM), Oswaldo Cruz Foundation (Fiocruz), Recife, reported on the study in Science Advances. In their paper, “International multisite implementation of distributed cell-free protein biomanufacturing to advance health and research equity,” the authors concluded, “This study lays the foundation for fundamental shifts in biotechnology manufacturing practices in LMICs and developing nations, moving from reliance on centralized and outsourced production facilities to adopting decentralized, local production platforms.”

    “Emerging biotechnologies hold transformative potential to strengthen economic and health security, while benefiting the planet,” the authors wrote. However, they pointed out, “Access to advanced tools, such as molecular diagnostics, life-saving treatments, and biomanufacturing infrastructure, remains largely concentrated in wealthier regions, thereby restricting access to transformative solutions for communities that need them most.”

    A key factor is the reliance on centralized bioproduction systems, “… which require sophisticated, capital-intensive infrastructure and cold supply chains that are often unavailable in resource-limited settings.” Access to healthcare is similarly affected by the availability of biomanufacturing capacity and biologistics, which can slow delivery of diagnostics, delay disease control programs, and limit the ability to carry out life sciences research.

    For their newly reported work the team focused on synthetic biology and cell-free systems—technologies that isolate and freeze-dry the molecular machinery needed to produce proteins commonly used in life sciences research. Because the reagents are freeze-dried, they can be shipped and stored without refrigeration, then reactivated simply by adding water. “One promising avenue to improving access is cell-free protein synthesis (CFPS), which offers the potential to empower communities through affordable, low-burden, on-site production of critical bioreagents, diagnostic tools, and therapeutic agents,” the researchers stated.

    They paired these systems with low-cost, adaptable hardware, including a 3D-printed hand-powered centrifuge developed by postdoctoral fellow Mohammad Simchi, PhD, at the Leslie Dan Faculty of Pharmacy. Together, the technologies enabled teams to produce a range of research proteins and diagnostic tools in diverse settings, from conventional laboratories to remote field locations.

    “With efficient, low-cost systems in place, rapid on-site production of high-value bioproducts for research, including growth factors, vaccines, and diagnostic enzymes, became achievable within a single day and at a fraction of the typical cost,” they commented.

    Using the platform, researchers successfully produced growth factors used in life sciences research and therapeutics, as well as a SARS-CoV-2 vaccine candidate tested in mice and diagnostic tools targeting several clinically relevant pathogens. Using molecular, cell-based, animal model, and clinical sample testing, the bioproducts were validated through proof-of-concept studies and multisite clinical trials. “Direct comparisons with high-cost commercial reagents, the current gold standards, demonstrated similar performance, efficiency, precision, and reproducibility,” the investigators further noted.

    First author Severino Jefferson Ribeiro da Silva, PhD, a postdoctoral fellow in Pardee’s lab, said, “Our work shows that it is possible to produce high-value bioreagents on site, essentially anywhere. Through this work, we demonstrated our tools across diverse international settings while maintaining performance comparable to commercial products.”

    The authors say that, to their knowledge, the study is the first to translate cell-free biomanufacturing from laboratory to real-world use, across multiple geographic settings, including those that historically have had limited access to the bioeconomy. “By prioritizing accessibility, affordability, and reproducibility, we show that cell-free biomanufacturing is a transformative tool for expanding global research capacity and, ultimately, health equity and participation in the bioeconomy.”

    A key component of the project involved testing the systems in a variety of environments across Canada and internationally. Da Silva travelled to the Algonquin Highlands to evaluate diagnostic tools for tick-borne pathogens and tuberculosis, while graduate student Quinn Matthews travelled to the Yukon where he produced and purified proteins using the portable system on a mountain outside Whitehorse.

    Collaborators in Chile, Brazil, Colombia, and India also tested the systems, helping ensure the technologies addressed the practical realities faced by researchers in different regions. The project involved extensive international collaboration, including regular meetings, student exchanges and knowledge sharing among participating teams.

    Da Silva says the research team experienced first-hand many of the logistical challenges their collaborators routinely face, including lengthy customs delays and damaged shipments containing critical reagents.

    “Those experiences highlighted how dependent many researchers and labs still are on fragile international supply chains. If a shipment is delayed, an entire project can stop,” says da Silva. “This work makes it possible to reduce that dependency by enabling local production of key proteins directly at the point of need.”

    The researchers say the long-term goal is to help research labs in remote and underserved regions gain access to high-quality diagnostics, research reagents and biomanufacturing capabilities produced closer to home, strengthening resilience against future supply chain disruptions while empowering their research capacity and address local healthcare needs. “With their low cost and operational simplicity, we see these platforms and similar disruptive technologies … as part of a new generation of tools that will help shape a future in which bioreagents, advanced diagnostics, and life-saving therapeutics are accessible to all.”

    Da Silva added, “This work is really about access and scientific empowerment. Many labs worldwide have the expertise and ideas to conduct life sciences and applied science research, but they face major challenges accessing key bioreagents and essential materials. Decentralized biomanufacturing could help reduce those barriers and make research and diagnostics more accessible globally.”

    The post Low-Cost, Portable Biotech Tools Improve Access to Bioresearch and Diagnostics appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Attacking Gout: Crystalys Sees Room for Its Dotinurad and Other Allopurinol Alternatives https://edusehat.com/en/attacking-gout-crystalys-sees-room-for-its-dotinurad-and-other-allopurinol-alternatives https://edusehat.com/en/attacking-gout-crystalys-sees-room-for-its-dotinurad-and-other-allopurinol-alternatives Tue, 02 Jun 2026 06:40:11 +0700 Edusehat Attacking, Gout:, Crystalys, Sees, Room, for, Its, Dotinurad, and, Other, Allopurinol, Alternatives Once labeled the “disease of kings” because of its association with consuming rich foods and alcohol, gout has emerged as a royal pain to a growing number of people. A 2024 study showed the prevalence of the most common form of inflammatory arthritis jumping 22.5% between 1990 and 2020, to 55.8 million people worldwide, with 95.8 million projected by 2050. An aging population and rising rates of metabolic conditions like obesity, hypertension, and chronic kidney disease have fueled gout’s growing prevalence.

    Yet recent gout-related approvals have been limited to supplemental applications and additional indications for existing treatments. In 2022, the FDA approved an expanded label for Krystexxa® (pegloticase) by authorizing the chronic, treatment-refractory gout drug to be combined with methotrexate, a combo shown to be more effective against the disease. Last year the FDA approved Glopbera®, a new liquid formulation of colchicine indicated for prevention of gout flares in adults. The liquid formulation allows doctors to adjust dosages more easily for patients with kidney or liver impairment.

    Among companies developing new gout treatments is Crystalys Therapeutics, which has dosed the first patient with its once daily oral, URAT1 inhibitor dotinurad in its Phase II AMETHYST trial (NCT07535034). The study is designed to assess dotinurad’s effectiveness in patients with gout who are intolerant or have a contraindication to xanthine oxidase inhibitors (XOIs) or have failed prior uricase treatment.

    AMETHYST is expected to enroll about 90 patients, with an estimated primary completion date of July 2027. The trial’s primary endpoint will be the percentage of patients with a serum uric acid (sUA) level of <6.0 mg/dL at Week 24 following dosing.

    ‘Important milestone’

    “Dosing the first patient in our Phase II AMETHYST study marks an important milestone for Crystalys, and for those living with gout who have limited treatment options,” said James M. Mackay, PhD, Crystalys’ president and CEO.

    That population, he said, represents about 10% of gout patients or ~1.5 million Americans who cannot tolerate the current standard of care, xanthine oxidase inhibitors (XOIs).  The most commonly prescribed drug for gout is the XOI allopurinol, a first-line treatment marketed in the U.S. as Zyloprim® by Casper Pharma and sold outside the U.S. as Zyloric® by Aspen Pharmacare, but also available as a generic drug.

    “We think that our target population for dotinurad to is about 500,000 to 600,000 patients in the U.S.,” Mackay estimated, adding the estimate was for the broader Phase III population, not the AMETHYST Phase II population. “It’s those patients who failed allopurinol and were referred to a rheumatologist. The rheumatologist has tried to up-titrate allopurinol but has still not been successful in getting the disease under control. The patient’s still experiencing gout flares, still has tophi and potentially joint damage. That’s our target patient population.

    In that class of patients, Crystalys envisions dotinurad succeeding in second-line treatment by outperforming allopurinol in their Phase III clinical trials.

    “Our goal is to go beyond serum uric acid lowering and actually show that our drug can actually impact the clinical manifestations of the disease, which allopurinol really doesn’t do significantly. And as a result of that, we wanted to position it as a second-line treatment,” Mackay said. “As time goes on and rheumatologists become comfortable with it, and PCPs [primary care physicians] who are referring their gout patients to rheumatologists will get comfortable with it and we may start to see some usage in the PCP market, but I imagine that payers are going to want patients to have failed on allopurinol before they’re prepared to pay for a new drug. This is why we are positioning it as a second line treatment in patients who have failed on standard of care.”

    Phase III triumph

    First-patient dosing in AMETHYST comes five days after a Crystalys rival, Swedish Orphan Biovitrum (Sobi), announced positive Phase III data for pozdeutinurad, a treatment for progressive gout which like dotinurad is a next-generation, once-daily oral URAT1 inhibitor.

    Sobi said May 21 that pozdeutinurad aced its pivotal 811-patient, placebo-controlled Phase III REDUCE-2 trial (NCT06439602) as both doses of the drug met the study’s primary efficacy endpoint, defined as the proportion of patients achieving an sUA level <6 mg/dL at month 6. The 75 mg high dose of pozdeutinurad led to 69.2% of patients achieving sUA level <6 mg/dL at month 6 and the 50 mg low dose, 56.6%, compared with 8.1% for placebo (p<0.0001).

    Sobi said it will report further detailed results at an upcoming scientific conference during Q4.

    “We are very encouraged by these results and their implications for patients whose gout remains inadequately controlled,” Lydia Abad-Franch, MD, Sobi’s head of R&D and medical affairs and chief medical officer, said in a statement. “These findings, including sustained urate lowering and a favorable efficacy and tolerability profile, support the potential of pozdeutinurad to address a significant unmet need and provide a strong foundation for regulatory submissions.”

    REDUCE-2 is one of two fully recruited 12-month, 800+-patient randomized, placebo-controlled Phase III trials in which Sobi is studying pozdeutinurad. The other is REDUCE-1 (NCT06846515), which is expected to read out data in the second half of this year.

    Sobi took over development of pozdeutinurad when it acquired the drug’s original developer, San Diego-based Arthrosi Therapeutics for up to $1.5 billion in a deal completed in February. Sobi agreed to pay $950 million in upfront cash plus up to $550 million cash in payments tied to achieving clinical, regulatory, and sales milestones under the companies’ acquisition deal, designed to strengthen the buyer’s gout drug franchise since pozdeutinurad is designed for patients whose treatment with first-line therapies proved unsuccessful.

    “Sobi’s acquisition, we actually view that as very positive: Good for gout patients. Good that pharmas are showing an interest in this space,” Mackay said.

    Sobi envisions pozdeutinurad as one of two gout drugs it aims to bring to market. The other is Nanoecapsulated Sirolimus plus Pegadricase (NASP, formerly SEL-212), a combination of the PEGylated recombinant uricase enzyme pegadricase and ImmTOR, a tolerogenic nanoparticle encapsulating the immunosuppressant sirolimus, being developed to treat uncontrolled gout. The FDA is evaluating Sobi’s biologics license application (BLA) for NASP, for which the agency has set a June 27 target action date under the Prescription Drug User Fee Act (PDUFA).

    Business case

    Mackay said Crystalys’ business case when it acquired dotinurad assumed that pozdeutinurad would be on the market with a similar profile: “We took a pretty conservative set of assumptions. Our market research that we did gave us a 65% market share versus 35% market share for the competitor [pozdeutinurad], and that’s with the profiles being the same.”

    “We actually believe we’re going to have a better profile for the molecule, and there are a lot of patients out there,” Mackay added. “It’s a big, big market, so there’s no doubt that there’s room for more than one player here.”

    How does dotinurad’s profile stand out compared with pozdeutinurad’s?

    “They’re both URAT1 inhibitors, but pozdeutinurad is not quite as potent as dotinurad, so we end up using lower dose levels than they do. They were using 50 and 75 mg in their Phase III trials. We’re using 2 and 4 (mg),” Mackay said.

    Mackay also cited dotinurad’s ability to target the URAT1 transporter without impacting the other transporters involved in regulating blood uric acid levels, the organic anion transporters OAT1 and OAT3, and ABCG2 (ATP-Binding Cassette Subfamily G Member 2): “We believe that that’s partially why we don’t have a renal tox liability, because it means that there’s more control over the excretion of the uric acid.”

    “This is a very, very big second-line space here. There are many, many patients who are uncontrolled, and so, we made the assumption that pozdeutinurad would be on the market alongside dotinurad.”

    To date, Sobi has the advantage of positive Phase III data showing reduced serum uric acid, with expectations for more positive data this year: “Later we will see the tophi reduction, the tophi resolution and the flare reduction. We’re expecting very strong data,” Lydia Abad-Franch, MD, MBA , Sobi’s head of R&D and chief medical officer, told analysts April 28 on the company’s Q1 earnings call.

    At its Capital Markets Day on February 18, Sobi told analysts that upon approval, followed by a launch scheduled for 2028, pozdeutinurad is expected to generate blockbuster-level peak sales of SEK 10 billion ($1.075 billion) from a progressive gout patient population it has pegged at more than 200,000 patients in the U.S. alone. “Pozdeutinurad represents the primary economic opportunity for Sobi in our gout franchise,” Guido Oelkers, Sobi’s president and CEO, said at the event.

    Long-term sales generator

    In announcing Sobi’s acquisition of Arthrosi in December, Oelkers said the company sees pozdeutinurad as a long-term sales generator: “The product has the potential to materially accelerate our growth until the mid-2030s, and beyond.”

    AMETHYST is among randomized, double-blind, multicenter trials Crystalys is conducting in the U.S. and European Union (E.U.) for dotinurad. Two of the trials are in Phase III, both aiming to evaluate dotinurad’s efficacy in lowering sUA at week 24:

    • RUBY (NCT07089875), a U.S. and E.U. study evaluating the safety and efficacy of dotinurad compared with a physician-determined stable dose of allopurinol in approximately 500 patients with hyperuricemia associated with gout. Study participants will be given dotinurad orally once daily for up to 64 weeks.
    • TOPAZ (NCT07089888), a U.S. study assessing the safety and efficacy of dotinurad compared to allopurinol in approximately 250 patients with tophaceous gout. Participants are being given dotinurad orally once daily for up to 76 weeks.

    Crystalys acquired dotinurad in 2024 by purchasing from Urica Therapeutics its license covering development and commercialization rights in the U.S. as well as Europe, the Middle East, and North Africa, from the drug’s discoverer, Japanese pharma Fuji Yakuhin. In return, Crystalys gave Urica—a subsidiary of Fortress Biotech—an equity stake in Crystalys and a 3% royalty on future net sales of dotinurad.

    In Asia, Fuji Yakuhin has licensed to Eisai rights to dotinurad, which is approved as a treatment for gout and hyperuricemia in China, Japan, Thailand, and the Philippines.

    25+ novel gout drugs

    As of September, more than 20 companies had developed over 25 novel drugs across various clinical stages for indications related to gout, according to DelveInsight. Among later phase drugs in clinical phases with gout indications:

    • Dapansutrile (OLT1177®)—Olatec Therapeutics’ oral NLRP3 inhibitor is under study in the Phase II/III PODAGRA II trial (NCT04971499) in roughly 300 patients with an acute gout flare. The study’s estimated completion date is December 31. In March, Olatec began studying dapansutrile in the Phase II DAPA-PD trial, a 12-month study of the drug as a treatment for Parkinson’s disease.
    • Epaminurad—JW Pharmaceutical said April 27 that it finished dosing the final patient in a Phase III trial (NCT05815901) designed to compare the safety and efficacy of the selective URAT1 inhibitor to febuxostat, which in the U.S. is a generic drug once marketed by Takeda Pharmaceutical as Uloric®.
    • Lingdolinurad (ABP-671)—Atom Therapeutics’ lead candidate, also a selective URAT1 inhibitor, is in Phase IIb/III trials worldwide, including the U.S., for indications that include chronic gout and hyperuricemia, and refractory and/or tophaceous gout. Last October at the American College of Rheumatology’s ACR Convergence 2025, Atom presented positive Phase IIa data for lingdolinurad and Phase I data for a separate gout flares candidate, ABP-745.

     XRx-026—XORTX Therapeutics’ Phase III gout candidate uses a proprietary formulation of oxypurinol, the active subunit of allopurinol, called XORLO™. XRx-026 is being developed for patients with allopurinol intolerant gout.

    Mackay, a veteran pharmaceutical executive, was a 30-year AstraZeneca executive who held several VP-level clinical and product positions with the pharma giant, where he led teams that advanced six drugs through development and commercialization across a range of therapy areas. He later oversaw development of AstraZeneca’s gout franchise as president and COO and then CEO of Ardea Biosciences, which remained an independent business unit following its acquisition in 2012 by AstraZeneca.

    In 2018, Mackay founded Aristea Therapeutics, an immunology focused company developing treatments for rare inflammatory disorders. As Aristea’s CEO, he led the company’s raising of $138 million between 2018 and 2023.

    That year, Mackay said, Aristea discovered an unexpected liver toxicity issue during Phase II trials of its lead drug RIST4721, which it licensed from AstraZeneca. RIST4721 was an antagonist of the CXCR2 protein that was being studied as a treatment for the inflammatory disorder palmoplantar pustulosis. Aristea’s board considered strategic alternatives before opting to end the RIST4721 development program—”in order to protect patient safety,” the company stated at the time—and dissolve Aristea.

    ‘Want to work with you’

    “Once the dust had settled a little bit, my main investor in Aristea Therapeutics came back to me and said, look, we want to work with you and the team again,” Mackay recalled.

    The investor was Novo Holdings, the asset manager of the foundation that controls Novo Nordisk.

    “They said, ‘We’re really interested in the gout space and would like to invest there,’” Mackay recalled. “Are you prepared to work with us and see if we can find an asset that, is worthy of developing and worthy of investment?”

    Mackay agreed.

    “We did a landscape search of all the molecules under development, and we identified dotinurad as the molecule that we felt had best-in-class safety and efficacy, and we decided to form Crystalys Therapeutics with Catalys Pacific and Novo Ventures as the company to, basically, develop dotinurad,” Mackay added.

    Novo Holdings and Catalys Pacific joined SR-One in launching Crystalys last September with a $205 million Series A that also saw participation from an investor syndicate that included Perceptive Xontogeny Venture Funds, Lightstone Ventures, AN Venture Partners, funds managed by abrdn Inc., KB Investments, Pontifax, Longwood Fund, Alexandria Venture Investments, Wedbush Healthcare Partners, and Prebys Ventures Fund.

    The financing extended Crystalys’ financial runway into end 2027—long enough, the company says, to allow it to carry out both the RUBY and TOPAZ trials: “We secured all the money that we need in order to deliver those programs,” Mackay said.

    Based in San Diego, Crystalys has a workforce of 14 staffers: “I expect we’ll probably double the size, so around 25 people by the time we get into the end of 2026.”

    Workforce growth will primarily take place in Crystalys’ R&D operations since the company’s focus will continue to be on its clinical trials—not only AMETHYST but the Phase III RUBY and TOPAZ studies as well.

    “I think it’ll be into 2027 before we start to build that commercial infrastructure,” Mackay added.

    The post Attacking Gout: Crystalys Sees Room for Its Dotinurad and Other Allopurinol Alternatives appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Organ Chips Move Towards Mainstream Drug Development, with Hurdles Ahead https://edusehat.com/en/organ-chips-move-towards-mainstream-drug-development-with-hurdles-ahead https://edusehat.com/en/organ-chips-move-towards-mainstream-drug-development-with-hurdles-ahead Tue, 02 Jun 2026 03:05:24 +0700 Edusehat Organ, Chips, Move, Towards, Mainstream, Drug, Development, with, Hurdles, Ahead In April 2025, the U.S. Food and Drug Administration (FDA) released a strategic roadmap to make animal testing the exception for preclinical safety and toxicity studies within the next three to five years. Central to that vision is the adoption of validated new approach methodologies (NAMs), including organ-on-chip systems. The National Institutes of Health reinforced that shift the same month by requiring that all new notices of funding involving animal models incorporate human-focused approaches such as organ chips and other NAMs. Similar changes are emerging globally. In November 2025, the U.K. government published its roadmap to largely phase out animal testing in research while accelerating the development and validation of alternative methods.

    For organ-on-chip developers, growing interest from federal agencies is a welcome trend. They are currently generating the data necessary to show that their technologies can work in stringent regulatory environments. However, there are still outstanding questions around validation standards, regulatory expectations, and how NAM data will be evaluated in submissions. At the same time, adoption remains slow, with drug developers continuing to rely largely on established animal models, which command billions in investment compared to the much smaller organ-chip sector.

    Still, it is clear that momentum is building behind NAMs. And in response, organ-chip developers are stepping up to ensure that their platforms can produce results when the time comes.

    From space flight to lab scale-up

    When the Artemis II astronauts launched their historic 10-day journey around the Moon in April 2026, they carried some unusual cargo: organ chips containing cells from their bone marrow. The chips are part of the AVATAR (A Virtual Astronaut Tissue Analog Response) investigation, which is using organ-on-chip devices to study the effects of deep-space radiation and microgravity on human health.

    Emulate's Organ Chip
    Emulate’s organ chips played a pivotal role in the recent Artemis II lunar mission. The so-called AVATAR experiment could change how space agencies study the effects of radiation and microgravity impact human health. [Emulate

    Before the trip, cells from the astronauts were harvested to create two sets of bone marrow chips: one set traveled beside the crew aboard their spacecraft, while another remained on Earth. The idea was to compare both sets of chips when the astronauts returned to Earth. More broadly, the AVATAR project also aims to provide proof-of-concept for including human organ chips in future missions.

    In 2025, Emulate announced that its organ-chip technology was selected to accompany the astronauts on their lunar fly-by. It is an exciting project for Emulate, which commercializes human organ-chip technology developed at the Wyss Institute for Biologically Inspired Engineering at Harvard University. But it is only one of several activities that the company has been involved in the recent past. The company’s liver organ chips were one of the first to be accepted for the FDA’s Innovative Science and Technology Approaches for New Drugs (ISTAND) program, which supports tools that fall outside the scope of existing qualification programs but may still be useful for drug development.

    Lorna Ewart
    Lorna Ewart, PhD
    Chief Scientific Officer
    Emulate

    In a conversation with GEN, Lorna Ewart, PhD, Emulate’s chief scientific officer, described 2025 as a pivotal year both externally—with announcements from multiple federal agencies promising increased support for organ chips—and internally, with the launch of Emulate’s new instrument, AVA, in June 2025 to address what Ewart describes as “key operational challenges” with the company’s first-generation platform. AVA has a higher throughput than its predecessor, enabling microfluidic workflows across 96 parallel organ chips or “emulations” in a single run. The company claims that it is the first organ-on-chip workstation to combine high-throughput microfluidic tissue culture with automated imaging in a self-contained environment.

    Interest in the instrument to date has come primarily from large pharmaceutical companies and mid-sized biotech firms, who need to run large numbers of chips in parallel. But, Ewart says, there is also strong interest from academic institutions and government agencies. Some of that interest is driven by AVA’s much smaller footprint. Compared to Emulate’s first-generation system, AVA is a compact benchtop system that does not require multiple incubators. The company has also reduced the size of each emulation, or chip equivalent, by about 50%, meaning that the new platform requires fewer cells and uses less media, helping to keep experimental costs down. “Academics are actually quite excited about getting their hands on it and looking at it as a core lab instrument where multiple labs will be able to use it.”

    AVA also addresses concerns about reproducibility, a consistent source of worry for drug developers, and one that Emulate has made a priority. The company has shared data showing that its liver-chip biology is reproducible both internally and externally in laboratories using AVA. The company has also taken steps to minimize technical variability within experiments as well as bias when running AVA at scale. “We need to make sure that the first chip array looks the same as chip array eight,” Ewart says. “If it doesn’t, there’s variability across those different [chip arrays] that will impact the way that a user can design, what we would refer to as a fully burdened experiment.”

    More complex, automated models

    When it first launched, U.K.-based organ-on-chip company CN Bio started with a liver-on-a-chip platform, but has since expanded to include various organ models, including intestine, lung, and kidney. The company’s commercial platform is built on technology developed in the laboratory of Linda Griffith, PhD, at the Massachusetts Institute of Technology.

    Tomasz Kostrzewski
    Tomasz Kostrzewski, PhD
    Chief Scientific Officer
    CN Bio

    Currently, CN Bio has applications in multiple arenas, including safety, toxicology, and disease modeling. “For example, in the toxicology space, we have a very well-known and well-utilized model of drug-induced liver injury,” Tomasz Kostrzewski, PhD, the company’s CSO, tells GEN. That model is being utilized by several global clinical research organizations to offer assays as a service. The company also has a multi-organ system that links its intestine and liver chip models, which can be used to predict the oral bioavailability of drugs, and a range of disease models for metabolic liver disease, chronic obstructive pulmonary disease, and more.

    Perhaps one of the biggest challenges, from Kostrzewski’s perspective, is the misconception among some stakeholders that organ chips can fully replace animal models today. That is not a position that the organ-chip community has advocated for, he says. The focus should be on “using these tools to answer the right question and [in] the right context of use at the right time alongside all those other approaches that are out there.”

    Development plans in the near future involve making incremental improvements that refine CN Bio’s platform over time. “One key area that we’re working on is immunology and adding in more complex immune cultures into our chips,” Kostrzewski says. Recently, “we presented some of the first data [incorporating] peripheral immune cells in our liver model and looking at the toxicity of monoclonal antibodies.” Some customers are building “neuronal blood brain barrier models on our platform” with an eye towards “understanding how drugs can penetrate across that barrier.” In parallel, the company is expanding into new organ systems, including kidney models, via partnerships.

    The company is also turning to automation to help customers scale their work. CN Bio’s open design integrates well with standard robotic systems, making it well-suited for high-throughput workflows, Kostrzewski says. Customers could run more chips in parallel as part of larger screening studies with more consistency and less human intervention. There is also the potential to incorporate sensing capabilities, much like those used in biomanufacturing, to monitor system performance in real time and generate functional readouts.

    In addition, the company is working to demonstrate to drug developers that organ chips can generate valuable translational data that predicts clinical outcomes. That certainly has been true for CN Bio as “we have a number of molecules that we have helped take to the clinic” that have been proven successful, says Kostrzewski. And there are customers using its organ chips “to make no-go decisions” regarding potential drug programs. “That’s the ultimate proof that these technologies do what they say,” he says.

    CN Bio’s PhysioMimix
    CN Bio’s PhysioMimix supports studies of metabolic liver disease, chronic obstructive pulmonary disease, and drug delivery in the brain. There are also efforts to develop additional organ systems using the technology. [CN Bio]

    Digital twin and multi-organ models

    Hesperos’ co-founders, James Hickman, PhD, and Michel Shuler, PhD, have been involved in the organ-chip space since its early conception. In fact, the technology that underpins the company’s services emerged from work that both scientists were doing independently in their laboratories. Today, the company provides drug development services using its Human-on-a-Chip® single- and multi-organ systems in areas such as neurodegenerative disease.

    In April, the company published a study in Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association focused on familial Alzheimer’s disease (fAD). Specifically, scientists at Hesperos and the University of Central Florida (UCF) used a neuromuscular junction (NMJ) multi-organ chip to show that fAD-associated mutations caused specific impairments in NMJ functions that occurred independently of brain pathology. Building on that work, Hesperos scientists and their collaborators are trying to understand what therapeutics could potentially be useful for both the peripheral and central nervous systems, as well as which would need to be specific for each.

    Last year, the company also demonstrated what they claim is the first true digital twin capability using an organ-on-chip platform. That capability is described in an Advanced Science paper where the scientists explain how a multi-organ system comprising human liver, spleen, endothelial tissues, and blood was used to replicate the full lifecycle of Plasmodium falciparum, the parasite responsible for malaria. They plan to publish additional studies on their work on digital twins. Additionally, like Emulate, Hesperos is also participating in the FDA’s ISTAND program.

    James Hickman
    James Hickman, PhD
    Co-founder
    Hesperos

    In a conversation with GEN, Hickman described the broader adoption of organ-on-chip technology as a mixed bag, with some people being more open to the technology and others showing more resistance. He noted that many in the community are still accustomed to using animal models, which may make them more reticent to change, but also acknowledged that animal testing is a multi-billion-dollar business. “There are a lot of people with a vested interest in keeping animal experimentation going,” he says. That means that although people may be interested in alternatives like organs-on-chips, from a practical perspective, it may be difficult for them to disengage from their reliance on animal models.

    He also pointed to the FDA’s evolving guidance on alternative technologies—and the lack of clarity—as one of the biggest hurdles. “People are still trying to get their hands around the FDA announcements on moving away from animal models,” and trying to understand what the agency wants to see, Hickman explained. “We have a pretty good idea of what that [might be needed and] we work with a couple of people [to] generate data along those lines,” he says. “The biggest thing is to start getting [clearer guidance] in terms of what they will accept in lieu of safety data.” There are also questions around whether good laboratory practice (GLP) requirements for these new approach methodologies need to mirror those for animal studies, given the differences between the systems. “Doing GLP is really expensive,” Hickman said, and requiring the same standards could effectively put many companies out of the running to conduct safety studies because they can’t afford it.

    Equally important is addressing the limited investment in organ chip and other alternative technologies. Hickman estimates that commercial NAM entities collectively generate hundreds of millions in revenue, compared to tens of billions secured by large animal CROs. Although federal agencies have committed to supporting NAMs, providing millions in funding, greater investment is needed for these alternative technologies to come into their own. Hickman added, “It’s a matter of trying to increase that capacity to really start showing that it’s a force in the industry versus a shiny new toy that people haven’t quite figured out what to do with.”

    The post Organ Chips Move Towards Mainstream Drug Development, with Hurdles Ahead appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Targeted Protein Degradation Broadens Its Scope https://edusehat.com/en/targeted-protein-degradation-broadens-its-scope https://edusehat.com/en/targeted-protein-degradation-broadens-its-scope Tue, 02 Jun 2026 03:05:22 +0700 Edusehat Targeted, Protein, Degradation, Broadens, Its, Scope Like any complex system, the cell depends on a tightly regulated quality control network to maintain order and prevent the accumulation of harmful proteins. This network governs protein homeostasis, including the synthesis, folding, trafficking, and ultimately the clearance of proteins. When these processes fail, aberrant or misfolded proteins can accumulate and drive disease.

    Targeted protein degradation (TPD) therapeutics seek to harness this intrinsic quality control machinery to selectively eliminate disease-causing proteins. Central to this approach is the principle of induced proximity, in which a designed molecule brings a target protein into close contact with a cellular effector, triggering its removal through endogenous degradation pathways.

    Two major systems underpin these processes. The ubiquitin-proteasome system governs the degradation of intracellular, soluble proteins, where targets are tagged with ubiquitin by a cascade of enzymes, including E3 ubiquitin ligases, and directed to the proteasome for destruction. In parallel, lysosome-mediated pathways handle larger, membrane-bound, extracellular, or aggregated proteins by routing them through endocytic or autophagic mechanisms for degradation.

    Building on these natural systems, a growing toolkit of TPD modalities has emerged. For example, proteolysis-targeting chimeras (PROTACs) exploit the ubiquitin-proteasome system, while newer approaches such as lysosome-targeting chimeras, including sortilin-based lysosome targeting chimeras (SORTACs), extend degradation to extracellular and membrane-associated proteins. Molecular glues, by contrast, stabilize interactions between E3 ligases and target proteins without requiring a bifunctional design, further expanding the scope of induced proximity strategies. Additional degrader technologies are being developed.

    Although first described more than 25 years ago, TPD is now entering a phase of rapid maturation and increasing therapeutic relevance. By operating through catalytic, event-driven mechanisms rather than traditional occupancy-based inhibition, these approaches offer the potential to address previously “undruggable” targets, overcome resistance mechanisms, and deliver more durable clinical responses. At the same time, key challenges remain, including expanding access to extracellular targets, improving target validation strategies, and navigating an increasingly complex and data-rich development landscape.

    Tackling the extracellular frontier

    Early TPD efforts have primarily targeted cytosolic proteins, leaving extracellular and membrane-bound targets (estimated to comprise about 40% of the human proteome) largely unaddressed.

    “Many key drivers of disease, including inflammatory cytokines, protein aggregates, and secreted factors, remain inaccessible to conventional PROTAC-based approaches,” says Simon Glerup, PhD, co-founder and CSO, Draupnir Bio, a spinout from Aarhus University (Denmark).

    Lab team photo
    Simon Glerup, PhD, co-founder and CSO, Draupnir Bio and Lab: Lab photo from left: Jonas Lende, Casper Larsen, Simon Glerup, Marianne Kristensen, Camilla Gustafsen, Amanda Simonsen, Line Slemming.

    The company is addressing this gap by utilizing its proprietary SORTAC platform, a modular, small-molecule technology designed to degrade extracellular proteins by harnessing the natural lysosomal clearance pathway. Glerup notes that “these targets are central to diseases such as neurodegeneration and inflammation, yet remain difficult to drug with existing modalities.”

    SORTACs are bifunctional small molecules composed of a sortilin-binding module linked to a target-binding ligand, enabling formation of a ternary complex between an extracellular disease protein and the lysosomal receptor sortilin, which drives internalization and degradation in lysosomes. Glerup elaborates, “Unlike antibody-based or intracellular TPD approaches, SORTACs combine the advantages of small molecules (such as potential oral delivery and tissue penetration) with catalytic, event-driven pharmacology. The platform has demonstrated hallmark TPD properties, including ternary complex formation and catalytic turnover, with in vitro and in vivo degradation of therapeutically relevant targets.”

    Glerup emphasizes that SORTACs enable degradation of both soluble and membrane-associated proteins and leverage receptor recycling to drive sustained target clearance.

    The company has launched a multi-partner Danish initiative, DESYNA (Degradation of Extracellular α-SYNuclein Aggregates) in collaboration with Aarhus University, focusing on Parkinson’s disease. Accumulation of α-synuclein aggregates is a key driver of disease, and the approach aims to selectively degrade these pathogenic species and halt their progression.

    Glerup believes extracellular TPD represents the next major wave of innovation in the field. “By extending TPD beyond the cell’s interior, the cytosol, SORTAC has the potential to unlock a large and previously inaccessible target space. With growing validation and collaborative efforts such as DESYNA, there is strong reason for optimism that this approach can deliver transformative therapies for diseases that currently lack effective treatment options.”

    Enabling TPD workflows

    Advancing TPD depends on a coordinated ecosystem of tools that support target validation, mechanistic interrogation, and translational predictions. Within this context, attention is increasingly focused on the central challenge of translating mechanistic promise into consistent patient benefits.

    Hannah Maple
    Hannah Maple, PhD
    Senior Director
    Bio-Techne

    “I think we are on the brink of seeing TPD and induced proximity truly usher in a new era in drug discovery as we await the first clinical approval of a PROTAC degrader,” says Hannah Maple, PhD, senior director at Bio-Techne®. At the same time, she notes that “one of the challenges with this as a new drug modality is to gain a deeper understanding of where the maximum patient benefit lies from a target and indication perspective.”

    That uncertainty places renewed emphasis on target validation strategies. Maple elaborates, “Driving efficacy versus standard of care in a predictable way remains a challenge, despite in many cases strong mechanistic rationale for degradation versus inhibition of a particular target. For this reason, I would keep target validation high on the list of key challenges for the field as it relates to driving clinical impact and patient benefit with this technology.”

    To support this critical transition, Maple says Bio-Techne has established long-standing collaborations with leading research groups to co-develop new technologies and support training of the next generation of TPD scientists. The company has also built an integrated portfolio of tools spanning biological reagents, chemical probes, and assay platforms with TPD-focused capabilities across its R&D Systems™ portfolio brand, including the Tocris™ small-molecule products.

    Maple provides an example. “Some of the most useful categories of tools for target exploration and validation in the context of TPD are the R&D Systems’ Tocris Tag Degradation Platforms and self-labeling protein tag platforms.” These approaches involve fusing a small protein tag to the protein of interest and pairing it with a complementary small-molecule ligand that binds the tag. The tag ligand is typically bifunctional and can be developed to recruit an E3 ligase to the protein-of-interest, eliciting degradation in a controllable, tunable manner.

    Within this ecosystem, protein-level tools support target interrogation and validation. Maple highlights self-labeling tag systems as particularly valuable. “Through our R&D Systems brand, we have built a leading portfolio of these technologies, and very recently launched BromoCatch™, a next-generation self-labeling tag platform that was co-developed with [the lab of Alessio Ciulli, PhD] at the Centre for Targeted Protein Degradation, University of Dundee. BromoCatch represents a powerful, modular platform that uses a low molecular weight protein tag. The benefit of this approach is to minimally perturb the native localization or function of the protein being tagged, versus prior larger tags that could cause undesired functional effects.”

    BromoCatch illustration
    BromoCatch™ is a small, rationally designed self-labeling tag platform for targeted protein analysis, manipulation, and degradation.
    [Bio-Techne]
    Complementing these approaches, the R&D Systems portfolio provides targeted degradation reagents such as dTAG-13, a heterobifunctional degrader used in tag-based systems to selectively eliminate engineered proteins of interest, offering a chemical alternative to genetic knockdown approaches.

    Maple reports that another impactful technology of Bio-Techne’s R&D Systems portfolio is their Simple WesternTM automated western blot instruments. She explains, “TPD heavily relies on western blotting, but scaling screening campaigns using this as a primary assay is a huge time and resource drain, with variable data quality and poor reproducibility. Simple Western technology allows researchers to get reliable, reproducible and quantitative degradation data on a fully automated instrument.”

    Enhancing pipeline intelligence

    Flavio Lima Bianchi
    Flavio Lima Bianchi
    Lead Research Analyst
    Beacon by Hanson Wade

    Keeping pace with the fast-moving TPD landscape can be daunting. “Part of the problem is that reliable data is hard to come by, particularly in regards to the advancements coming out of China, with developers still relying on their own, in-house methods to generate viable, orally bioavailable lead candidates at the cost of significant time and investment,” observes Flavio Lima Bianchi, lead research analyst at Beacon by Hanson Wade.

    As evidence of this challenge, Bianchi notes that despite PROTACs comprising roughly a third of the overall TPD landscape, “to date less than five percent of PROTACs have managed to progress into the clinic and only a select few drugs have reached late-state, pivotal studies.”

    The company is addressing these limitations in several ways. “We aggregate all available TPD data and render it into an easily searchable and digestible format. Too often is information siloed within organizations and, perhaps more importantly, failed degraders are rarely published or are quietly swept under the rug.”

    He continues, “Beacon leverages a mixture of publicly available and proprietary data obtained directly from developers to track every single TPD program globally and to lift the lid on both the successes and the failures, enabling developers to make better, more informed decisions.”

    While investigators relying on in-house methods may spend significant time searching available information, Bianchi emphasizes that their platform extends well beyond data access. “Beacon TPD is a subscription-based intelligence platform, providing users the ability to search comprehensive, curated preclinical, clinical, and commercial data across the induced proximity landscape. Aside from this primary search and retrieve function, Beacon’s additional functionalities include analyst reports, conference summaries, weekly newsletters and alerts, all designed to keep users abreast of the latest development within their field of interest.”

    Broadening TPD horizons

    Bio-Techne’s Maple envisions TPD expanding well beyond its original scope. “I think about TPD as one portion of a broader induced proximity revolution. The basic principles and technological breakthroughs that have driven TPD can be applied to targeted protein localization, stabilization, modulation, etc. This opens new optionality from a therapeutic standpoint and is also opening entire new fields of basic research enabled by these new principles and chemical tools.”

    The post Targeted Protein Degradation Broadens Its Scope appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Pharma’s Trial Problem: Outdated Systems, Broken Data, and the Coming AI Reset https://edusehat.com/en/pharmas-trial-problem-outdated-systems-broken-data-and-the-coming-ai-reset https://edusehat.com/en/pharmas-trial-problem-outdated-systems-broken-data-and-the-coming-ai-reset Tue, 02 Jun 2026 03:05:20 +0700 Edusehat Pharma’s, Trial, Problem:, Outdated, Systems, Broken, Data, and, the, Coming, Reset
    Erik Terjesen
    Erik Terjesen
    Managing Director
    Silicon Foundry, a Kearney Company

    Clinical development has become the most resource-intensive stage of drug innovation. Across the industry, clinical trials consume 60–70% of total R&D spending, a proportion that continues to rise as trials grow more complex, more data-heavy, and more operationally demanding. The irony is that while science has advanced dramatically, the underlying model for running trials still reflects assumptions from a pre-digital era. The result is an ecosystem in which timelines stretch, costs multiply, and meaningful efficiency gains remain elusive.

    AI has reached a level of maturity capable of reshaping this landscape, but its potential remains constrained by a fundamental issue the industry has been slow to confront. The data used to power these systems was never designed with AI in mind. In fact, the true crisis in clinical development today is structural and deeply rooted in how trial data is organized, contextualized, and interpreted.

    Why trial models are failing

    Clinical trials were built for physical sites, paper workflows, and slow-moving systems. Modern trials look nothing like that. They are distributed, data-heavy, biomarker-driven, and increasingly adaptive, yet they still run on infrastructure designed for a simpler era.

    For years, clinical operations have been organized around sites and checklists rather than continuous insight. Data moves in bursts, workflows remain fragmented, and systems rarely talk to one another. Precision medicine expanded what trials could ask of data, but the way trials actually operate has barely evolved.

    The problem isn’t only speed or scale. It’s also the quiet erosion of efficiency in places trial plans rarely account for. Across the industry, leaders describe a growing layer of “invisible waste”: repeated handoffs, duplicative manual work, incompatible data structures, and everyday operational friction that steadily stretches timelines and drives up costs, even though it seldom appears in formal project plans.

    AI changes the equation, but only if trial data can support it.

    Why AI stumbles in pharma

    There is no shortage of AI talent, tools, or ambition in the life sciences sector. What is scarce is data that AI can meaningfully learn from. Most early AI-for-clinical-trials initiatives failed not because the models were immature, but because the data they were fed was not curated with clinical intent.

    Two challenges define this crisis:

    1. General-purpose models cannot interpret clinical nuance.

    Models trained on large public corpora can identify patterns, but they lack clinical judgment. If the data is unstructured, inconsistently labeled, or lacks contextual metadata, the model will draw the wrong conclusions with absolute confidence. The well-known “ruler problem”—in which an AI system learned to detect malignant skin lesions based on the presence of a ruler beside the lesion—illustrates how easily models latch onto irrelevant signals.

    2. Pharma’s internal data is both rich and unusable.

    Organizations hold decades of trial data, but these assets are rarely AI-ready. Different study teams, CROs, and geographies used different standards. Biomarker and imaging data are often stored in systems that cannot communicate with EDC or safety platforms. And clinical notes, PDFs, and unstructured documents require interpretation that models cannot perform without curated training sets.

    AI amplifies the quality of the data it is given. If the input is clinically inconsistent, overgeneralized, or disconnected from the trial context, the outputs will be clinically meaningless.

    Recognizing this, many pharmas are now investing heavily in curated internal datasets, governance frameworks, and senior AI leadership, often in the form of newly created chief AI officer roles. These leaders are tasked with not just deploying tools, but rebuilding the data infrastructure from which future AI insights will emerge.

    The new AI toolkit for clinical trials

    Once the data foundation is strong, AI becomes a force multiplier across the entire trial lifecycle. Several categories show particularly high near-term impact potential.

    Clinical-grade language models: Purpose-built models that ingest curated internal datasets can help draft protocols, refine eligibility criteria, flag operational risks, and interpret historical trial performance. Unlike general-purpose systems, these models are tuned to reason the way experienced clinical scientists do.

    Multimodal AI for patient stratification and endpoint optimization: Integrating imaging, labs, digital biomarkers, and historical trial outcomes enables more precise cohort selection and improves the likelihood of detecting true therapeutic effect. These tools help convert today’s complex data streams into actionable insights.

    Synthetic and hybrid control arms: While still emerging, these approaches reduce dependence on large traditional control cohorts by incorporating real-world evidence and model-generated comparators when appropriate. The result is faster recruitment and more efficient statistical design.

    AI agents for operations: Operational agents can triage site queries, assist with eligibility adjudication, coordinate scheduling, and draft routine documentation. They are particularly helpful in reducing the administrative burden that slows trial execution.

    The most underestimated category, and the one with the most long-term potential, is clinical-driven AI, where the model is trained to interpret clinical data the way a researcher with a PhD or a clinician would. This approach addresses the core issue of context, which is essential for decision-making in regulated environments.

    From site-centric to data-centric trials

    Trials are gradually evolving away from rigid site-based infrastructure and toward data-centric execution. AI accelerates this shift by enabling continuous monitoring, adaptive decision-making, and greater representation across diverse populations. The next phase of this transition requires progress in several areas:

    • Reliable digital biomarkers collected via wearables and sensors that feed directly into the trial data ecosystem.
    • Real-world evidence integration that allows trial designs to incorporate external data while maintaining regulatory rigor.
    • Improved cohort diversity, supported by AI-driven recruitment models that identify and engage underrepresented populations.
    • Always-on trial oversight, where adaptive protocols adjust based on real-time data rather than periodic interim reviews.

    As these elements mature, trials will resemble dynamic learning systems rather than static sequences of predefined events.

    Pharma cannot do this alone

    The clinical-trial innovation ecosystem is now incredibly fragmented. A myriad of startups, many founded within the last five years, are attempting to solve different slices of the trial process. Some focus on recruitment; others on protocol simulation, operational automation, predictive enrollment, or digital biomarker analysis.

    This fragmentation creates noise but also opportunity. The organizations that succeed will be those that adopt a hybrid strategy, in which internal data expertise is paired with carefully selected external partners. Evaluating early-stage companies requires disciplined technical assessment and an understanding of which partners can meet enterprise requirements in a regulated environment.

    Pharma organizations also face a structural talent challenge. The best AI engineers often gravitate toward startups rather than large enterprises. This dynamic reinforces the need for partnership models that combine internal governance with external innovation rather than relying exclusively on one or the other.

    What AI can (and cannot) fix

    While AI can dramatically shorten timelines and improve decision-making, it is not a cure-all. It will not rescue a flawed trial design, replace human oversight, or eliminate the need for regulatory rigor. What it can do is accelerate the work around those elements, optimizing how protocols are developed, how patients are selected, how data is interpreted, and how milestones are achieved. The organizations that reap the greatest benefit will be those with disciplined data stewardship and a willingness to rethink long-held operational assumptions.

     

    Erik Terjesen is the managing director at Silicon Foundry, a Kearney Company

    The post Pharma’s Trial Problem: Outdated Systems, Broken Data, and the Coming AI Reset appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Top 10 U.S. Biopharma Clusters 2026 https://edusehat.com/en/top-10-us-biopharma-clusters-2026 https://edusehat.com/en/top-10-us-biopharma-clusters-2026 Tue, 02 Jun 2026 03:05:18 +0700 Edusehat Top, U.S., Biopharma, Clusters, 2026 Some of the forces that shape biopharma cluster development are constants year after year, such as the emergence of startups from university and research institute labs to develop new treatments, thanks to ideas backed by the brains of researchers and executives, and the bucks of serial entrepreneurs and other investors.

    But in recent years, several additional unique circumstances have come to reshape how much and especially where biopharmas choose to grow, Matthew Gardner, CBRE Americas Life Sciences Leader, shared with GEN recently.

    One is increased acquisition of lab and manufacturing properties by “mid-cap” biopharmas ranging between $2 billion and $10 billion in market capitalization (share price times the number of outstanding shares), as they seek to better control their supply chains by maintaining their own infrastructure in evolving from research- to commercialization-focused drug developers.

    “They might have been more likely to lease in a different circumstance. They’ve definitely caught an opportunity to jump in and take ownership. That has been an ongoing trend, and that has been true coast-to-coast in most of the major centers,” Gardner said.

    Among investor-owners, Gardner said, another transition has begun from pure-play biopharma real estate landlords to investors with broader portfolios encompassing healthcare—a reflection of how the two fields are increasingly converging. During December 2025 and January 2026, for example, the public real estate investment trust (REIT) Healthpeak shelled out $600 million to close on the acquisition of a 1.4-million square foot, 29-acre campus on Gateway Boulevard in South San Francisco, CA, from the nation’s largest biopharma REIT, Alexandria Real Estate Equities and BXP (formerly Boston Properties).

    Those and other investors aim to cash in on the improving climate for biopharmas seeking to raise capital, from a recovering venture capital market to increased merger-and-acquisition (M&A) activity, and, in recent weeks, a revived market for initial public offerings (IPO).

    Another key factor in recent cluster-building cited by Gardner is the “reshoring” of manufacturing in the U.S. by global biopharma giants, whether to satisfy growing demand for treatments—especially obesity drugs—or avoid tariffs, or both. While many of those new facilities are in manufacturing-heavy clusters like North Carolina and Greater Philadelphia, others have spread into Maryland and Virginia (the BioHealth Capital Region), and several new biomanufacturing sites have been built or are under construction in emerging clusters outside the Top 10—a trend GEN plans to explore in the coming weeks.

    Speaking of top 10 clusters, GEN presents its latest edition of its nationally- and regionally-cited annual A-List of its top 10 U.S. biopharma cluster rankings, designed to show which regions are most competitive in attracting life sciences leaders, companies, and institutions. Over more than a decade, GEN has based its rankings on five criteria:

    • Patents: Figures from the Patent Public Search database of the U.S. Patent and Trademark Office, showing the number of patent families containing the word “biotechnology” and towns and cities within a given region or state.
    • NIH funding: Figures for NIH funding were taken from the publicly available NIH Research Portfolio Online Reporting Tools (RePORT) database for the current federal fiscal year through May 4, plus all of fiscal year 2025 (October 1, 2024, through September 30, 2025).
    • Venture capital funding: Figures for all of 2025 and the first quarter of 2026 as compiled by regional life sciences groups and PitchBook, which joins with the National Venture Capital Association to publish the quarterly Venture Monitor reports.
    • Laboratory space: The total-size-of-market figure, in millions of square feet, as furnished by regional life sciences groups. In regions that did not compile such information, the figure cited is the highest by any of several commercial real estate companies, including CBRE Group, Colliers, Cushman & Wakefield, JLL, and Newmark.
    • Number of jobs: The preferred sources for job figures were regional life sciences groups. Alternative sources included commercial real estate firms.

    Top 10 U.S. Biopharma Clusters 2026

    1. Boston/Cambridge, MA

    Genentech in CT
    Genentech has agreed to more than triple its space, growing from 30,000 to 100,000 square feet, within 1 Milestone Street at the Harvard University-owned, Tishman Speyer-developed Enterprise Research Campus in Boston’s Allston section [Breakthrough Properties, Studio Gang & Henning Larsen]

    Years of growing into the nation’s top biopharma cluster have taken a toll on Boston and adjacent Cambridge, MA: The Wall Street Journal in December highlighted the inability of Boston-area PhDs to find work, while the region faces a glut of life sciences space as biopharmas and real estate developers scale back earlier plans—a 32.7% availability rate according to CBRE, up 70 basis points from Q1 2025. Takeda Pharmaceutical in March eliminated 247 jobs in Massachusetts, where the company has facilities in Lexington, MA, and Cambridge, part of a $1.3 billion restructuring that cut 634 jobs nationwide. Replimune in April chopped 223 jobs at its Woburn, MA, HQ, and Framingham, MA, manufacturing site after the FDA rejected its BLA seeking approval for RP1 [plus Bristol Myers Squibb’s Opdivo® (nivolumab)] for advanced melanoma. In February, Takeda placed 449,140 square feet within three Cambridge buildings on the sublease market, a week after Alexandria Real Estate Equities scrapped plans to convert 401 Park Drive in Boston’s Fenway section into lab space, with CEO and chief investment officer Peter M. Moglia saying the real estate investment trust was pivoting to meet growing demand for office space.

    Among the region’s growing life-science companies: Genentech agreed to more than triple its space, growing from 30,000 to 100,000 square feet within One Milestone Street at the Harvard University-owned, Tishman Speyer-developed Enterprise Research Campus in Boston’s Allston section. Hemab Therapeutics (based in Cambridge and Copenhagen) and Seaport Therapeutics (Boston) both priced IPOs on April 30, raising $301.5 million and $254.88 million, respectively—a day after Avalyn Pharma (Boston) garnered $300 million in its IPO. In March, Terrestrial Bio became the first life-science tenant at Allston Labworks (250 Western Avenue) by leasing 42,000 square feet at the mixed-use building within Boston’s Allston neighborhood, while AI Proteins in January inked a 40,000-square-foot lease at 660 Commonwealth Avenue, within Related Beal’s One Kenmore Square in Boston. Regional companies finding buyers in April include Boston-based Kelonia Therapeutics and Cambridge-based Ajax Therapeutics, both to be acquired by Eli Lilly (for up to $7 billion and up to $2.3 billion, respectively) and Framingham-based KalVista Pharmaceuticals, to be acquired by Italy’s Chiesi Group for about $1.9 billion.

    Boston/Cambridge enjoys the nation’s largest portfolio of lab space (63.2 million square feet according to industry group MassBio), but was bested by the San Francisco Bay Area in NIH funding (7,037 awards totaling $4.339 billion) following a year of government funding cuts. The region also placed second in VC ($6.85 billion in 2025, says MassBio; $1.59 billion in Q1 2026, according to PitchBook data cited by MassBio), but landed third in patents (29,621 families) and just fifth in jobs (117,108, according to MassBio).

     

    2. San Francisco Bay Area

    David A. Ricks and Jensen Huang shake hands
    Eli Lilly Chair and CEO David A. Ricks and Nvidia Founder, president, and CEO Jensen Huang announce the companies’ five-year, $1 billion partnership to create a “Co-Innovation AI Lab” designed to address key challenges in AI drug discovery, announced on January 12 during the J.P. Morgan 44th Annual Healthcare Conference in San Francisco. The lab will be located within the Bay Area. [Nvidia]

    Santa Clara, CA-based Nvidia and Eli Lilly electrified the annual J.P. Morgan Healthcare Conference, held in downtown San Francisco each January, by announcing a five-year, $1-billion collaboration to create a “Co-Innovation AI Lab” in the region to address key challenges in artificial intelligence (AI) drug discovery, powered by a supercomputer that went live in February. That welcome news aside, more than one-third of the region’s life-science space is available for lease (33.7% as of Q1, according to CBRE). And more space has entered the market: Pfizer confirmed plans in April to shut down its 164,000-square-foot research facility at 181 Oyster Point Blvd. in South San Francisco, CA, shifting employees to remote jobs. Cushman & Wakefield is marketing the space for sublease. Also, on the market in “South City” is a 21,552-square-foot lab building and surrounding 3.65 acres previously occupied by the U.S. Department of Agriculture, which is selling the building for just under $48 million. In May, Foster City, CA-based Gilead Sciences disclosed plans to lay off 108 employees based in Redwood City, CA, (and 84 in Rockville, MD) following its $7.8-billion acquisition of Arcellx.

    Not all the recent news is bad: Gladstone Institutes plans early next year to open approximately 20 new labs employing about 300 scientists within the 105,000 square feet it agreed to lease in March at 1450 Owens Street, within Alexandria Real Estate Equities’ Alexandria Center® for Science and Technology–Mission Bay Megacampus. Natera inked a 62,969-square-foot lease at Brittan West in San Carlos, CA, in February. And last fall, Elon Musk’s Neuralink leased the entire approximately 144,000-square-foot 499 Forbes Boulevard in South San Francisco. On the financing side, SF-based Breakout Ventures in March closed its $114-million Fund III, which aims to invest in founder-led companies applying AI in biopharma, while Palo Alto, CA-based Surf Bio, whose lead investor for its only institutional round was Breakout, was acquired by San Diego-based Halozyme Therapeutics for up to $400 million, in a deal announced in January.

    San Francisco and its suburbs topped Boston/Cambridge in VC ($7.8 billion in 2025, $1.5 billion in Q1 2026, both according to PitchBook). The Bay Area is second in three criteria: patents (35,166 families), lab space (54.3 million square feet according to Colliers), and jobs (150,491 according to BIOCOM California, but “more than 147,000” according to CBRE, both from last year). In NIH funding, the region is fourth (5,180 awards totaling $3.13 billion).

    3. BioHealth Capital Region (Maryland, Virginia, and Washington, D.C.)

    AstraZeneca new manufacturing facility in Rivanna Futures, near Charlottesville
    AstraZeneca has expanded the scope of its new manufacturing facility in Rivanna Futures, near Charlottesville, VA, into a $4.5 billion project designed to support manufacturing for weight management, metabolic, and cancer technologies, including antibody-drug conjugates. The project is expected to create 600 permanent jobs. [AstraZeneca]

    The BHCR takes in Virginia and Maryland, both of which benefited over the past year from the domestic “reshoring” of biomanufacturing by pharma giants. AstraZeneca in November announced $2 billion in plans for Maryland that include a major expansion of its biologics manufacturing facility in Frederick, MD, and a new clinical manufacturing facility in Gaithersburg, MD. A month earlier, AstraZeneca expanded the scope of its new manufacturing facility in Rivanna Futures, near Charlottesville, VA, into a $4.5-billion project designed to support manufacturing for weight management, metabolic, and cancer technologies, including antibody-drug conjugates. The project is expected to create 600 permanent jobs. Also last fall, Merck & Co. broke ground on a $3 billion, 400,000-square-foot Center of Excellence for Pharmaceutical Manufacturing at its longstanding site in Elkton, VA, while Eli Lilly announced plans for a $5-billion manufacturing facility just west of Richmond, VA, in Goochland County that will be the company’s first-ever dedicated, fully integrated active pharmaceutical ingredient (API) and drug product facility for its bioconjugate platform and monoclonal antibody portfolio. However, a longtime strength of the region—the headquarters presence of the NIH and FDA—is now among its most serious challenges as government funding cuts chopped the workforces of both agencies last year by 3,500 and 1,200 jobs, respectively, though the FDA in recent months has worked to hire 1,000+ new staffers to fill reviewer, inspector, and investigator roles. And in May, Gilead Sciences disclosed plans to lay off 84 employees in Rockville, MD (and 108 in Redwood City, CA) following its $7.8-billion acquisition of Arcellx.

    The BioHealth Capital Region fulfills its top-three cluster ambitions by continuing to lead the nation in patents (80,808 families) while placing third in NIH funding (4,665 awards totaling $3.474 billion) and lab space (37.208 million square feet according to JLL data cited by BHCR, including 9.2 million square feet of NIH labs in Bethesda, MD). The region is fourth in jobs (135,298, according to JLL and state data cited by BHCR), but seventh in venture capital ($1.117 billion in 2025, zero in Q1 2026, according to BHCR data).

     

    4. New York/New Jersey

    rendering of HELIX downtown campus
    In New Jersey, New Brunswick’s Planning Board in February approved the $468 million H-3, the third phase of the HELIX downtown campus, a 40-story 554,000 square foot tower, for which the city council approved a 30-year PILOT agreement that will generate $1.8 million a year in annual payments in lieu of taxes [DEVCO New Brunswick Development Corp.]

    The Big Apple will soon see a big biotech campus emerge, the $1.6 billion, 2-million-plus-square-foot Science Park and Research Campus (SPARC) Kips Bay, projected to create more than 15,000 jobs by combining life-science space with academic and public health facilities. Exterior demolition is scheduled for the third quarter, followed by construction next year. However, Johnson & Johnson has shifted operations of its JLABS@NYC incubator to site owner New York Genome Center, part of a corporate cutback of its incubator network. The 17-member Emerging Technology Advisory Board appointed by New York Gov. Kathy Hochul (D), who is seeking re-election this year, proposed numerous efforts in December to expand life sciences activity statewide, including a $65-million “Excellence” fund and a $40-million pre-commercialization fund. At deadline, the fate of those efforts was unknown despite a tentative agreement on May 7 of a $268-billion state budget.

    In New Jersey, New Brunswick’s Planning Board in February approved the $468-million H-3, the third phase of the HELIX downtown campus, a 40-story, 554,000-square-foot tower, for which the city council approved a 30-year PILOT agreement that will generate $1.8 million a year in annual payments in lieu of taxes. In suburban Westchester County, Regeneron Pharmaceuticals is completing a $1.8-billion HQ expansion in Tarrytown but has scuttled earlier plans to expand across the Hudson River into the Rockland County village of Suffern, where the company spent $39 million to buy an old Avon Cosmetics warehouse for conversion into an infectious disease lab and a cold storage facility. In February, Regeneron hired JLL to market the site for sublease.

    New York and its northern New Jersey suburbs lead the nation in NIH funding (7,033 awards totaling $4.396 billion) and are third in jobs (147,900, according to Cushman & Wakefield). From there, the region falls to the middle of the pack, placing fifth in VC ($1 billion in 2025 and about $400 million in Q1 2026, both according to PitchBook), and sixth in both lab space (25.5 million square feet, according to Colliers) and patents (12,523 families).

     

    5. Greater Philadelphia

    Eli Lilly Pennsylvania rendering
    Eli Lilly made history in January by announcing Pennsylvania’s largest-ever biotech project, a $3.5 billion biomanufacturing site planned for Upper Macungie Township, an hour’s drive northwest of Philadelphia. Lilly plans to base 850 jobs at the plant, which will produce retatrutide and other weight loss drugs when it becomes operational in 2031. Lilly also has plans for Philadelphia, namely a 44,000-square-foot Lilly Gateway Labs innovation hub in Center City West at 2300 Market set to open later this year. [Eli Lilly]

    Eli Lilly made history in January by announcing Pennsylvania’s largest-ever biotech project, a $3.5-billion biomanufacturing site planned for Upper Macungie Township, an hour’s drive northwest of Philadelphia. Lilly plans to base 850 jobs at the plant, which will produce retatrutide and other weight loss drugs when it becomes operational in 2031. Lilly also has plans for the City of Brotherly Love, namely a 44,000-square-foot Lilly Gateway Labs innovation hub in Center City West at 2300 Market set to open later this year. And, in Philadelphia’s Old City, Thermo Fisher Scientific last November opened its East Coast Advanced Therapies Collaboration Center (ATxCC) within the BioLabs for Advanced Therapeutics incubator.

    Thermo Fisher Scientific executives celebrated the opening of the East Coast Advanced Therapies Collaboration Center (ATxCC) in Philadelphia’s Old City
    Thermo Fisher Scientific executives last November celebrated the opening of the biotech tools giant’s East Coast Advanced Therapies Collaboration Center (ATxCC) in Philadelphia’s Old City, within the BioLabs for Advanced Therapeutics incubator. [Thermo Fisher Scientific]

    The region’s rich biotech history includes the first gene therapy Luxturna® marketed by Roche-owned Spark Therapeutics—which is completing its $575 million Gene Therapy Innovation Center in University City despite laying off more than half of its Philly staff last year. In March, TerraPower Isotopes announced plans for a $450-million radioisotope manufacturing facility designed to produce actinium-225 for cancer treatments. The project will employ 225, receive $10 million in state grants, and rise within The Bellwether District, the 1,300-acre former Philadelphia Energy Solutions refinery site. Greater Philadelphia has long benefited from innovations from its institutions, two of which won more than $100 million in NIH funding during the 2025 federal fiscal year, the Perelman School of Medicine at the University of Pennsylvania to Children’s Hospital of Philadelphia (CHOP)—which last year treated KJ Muldoon (“Baby KJ”), the world’s first patient to receive a personalized CRISPR gene-editing therapy (for CPS1 deficiency). The region’s needs for more C-suite talent and venture capital remain persistent challenges to cluster growth, stakeholders told The Philadelphia Inquirer in December, though Audrey Greenberg, chair of corporate development and “Mayo Venture Partner” at Mayo Clinic and founder of AG Capital Advisors, told the Inquirer: “I’m going to be starting my companies all here in Philadelphia, because that’s where I am.”

    Greater Philadelphia improved the most this year, climbing two positions in this year’s A-List after remaining fifth in patents (17,090 families) and rising to fifth in lab space (25.9 million square feet, according to Colliers’ data cited by Pennsylvania’s Department of Economic Development or DECD) and NIH funding (3,201 awards totaling $1.94 billion). The region jumped four spots to fifth in VC ($1.31 billion in 2025, $616 million in Q1 2026, says Colliers), but dipped to seventh in jobs (88,000, also according to DECD), including nearly 10,000 with cell and gene therapy expertise.

     

    6. San Diego

    Novartis' global Biomedical Research center in San Diego
    Novartis broke ground in February on a $1.1 billion, 466,000-square-foot global Biomedical Research center in San Diego, expected to house 1,000 employees when operational in 2029, three months after opening a radioligand therapy manufacturing facility for cancer treatments in Carlsbad, CA. [Novartis]

    The Biotechnology Innovation Organization (BIO) expects to draw 20,000 to its BIO International Convention when it returns this month to the San Diego Convention Center. The region remains a vibrant life-sciences cluster: Novartis broke ground in February on a $1.1-billion, 466,000-square-foot global Biomedical Research center in San Diego, expected to house 1,000 employees when operational in 2029, three months after opening a radioligand therapy manufacturing facility for cancer treatments in Carlsbad, CA. Eli Lilly in March completed its $1.2-billion acquisition of home-grown Ventyx Biosciences—months after the pharma opened a Lilly Gateway Labs innovation hub with Alexandria Real Estate Equities in Torrey Pines. The J. Craig Venter Institute—whose founder died April 29 at age 79—plans this summer to move its West Coast headquarters from the University of California San Diego campus in La Jolla to the downtown Research and Development District (RaDD), a $1.6-billion, 1.7-million-square-foot campus on the city’s Pacific coastline completed last year by San Diego-based developer IQHQ—which is fighting an investor’s fraud allegations related to a $50-million investment in 2020. Home-grown F5 Therapeutics (up to 10 employees) folded in March, while two other San Diego biotechs laid off employees this year: Gossamer Bio (65 employees, nearly half its workforce, as of May 15, following a Phase III trial failure) and BioAlta (70% of its staff, which was 41 as of December 31, 2025). In February, San Diego drug developer Iambic Therapeutics inked an up-to-$1.7-billion collaboration with Takeda Pharmaceutical, which will use Iambic’s AI technologies and wet lab capabilities to design and develop small molecule drugs. And global contract development and manufacturing organization (CDMO) Bora Biologics, in January, opened a $30-million expanded manufacturing facility with two to four 2,000-liter bioreactors, corresponding seed trains, and advanced downstream processing equipment.

    “America’s Finest City” and vicinity stayed third in VC ($1.9 billion in 2025, says PitchBook, $743 million in Q1 2026 according to a GEN spot-check of recent deals) and fourth in patents (18,314 families) but dipped to fifth in lab space (28.685 million square feet, according to CBRE). While the San Diego region last year rose to ninth in NIH funding (2,001 awards totaling $1.357 billion), it slid to ninth in jobs (71,448, according to year-old BIOCOM California data).

     

    7. North Carolina

    Roche’s Genentech subsidiary East Coast manufacturing facility in Holly Springs, NC
    Roche’s Genentech subsidiary in January expanded to $2 billion its planned investment in its first East Coast manufacturing facility in Holly Springs, NC, which broke ground last year and is set to support 500+ manufacturing jobs when operational by 2029. [Genentech]

    Always strong on drug manufacturing, North Carolina is among the biggest beneficiaries of biopharma’s reshoring push. In April, AbbVie announced a $1.4-billion, 185-acre drug production facility in Durham County near Research Triangle Park (RTP), expected to employ 734. Roche’s Genentech subsidiary in January expanded to $2 billion its planned investment in its first East Coast manufacturing facility in Holly Springs, NC, which broke ground last year and is set to support 500+ manufacturing jobs when operational by 2029. And in November 2025, Novartis said it will expand Tar Heel State operations into a flagship manufacturing hub by adding capabilities for sterile filling of biologics into syringes and vials at its current Durham site, constructing two new Durham facilities for manufacturing biologics and sterile packaging, and building a new Morrisville, NC, site to produce solid dosage tablets and capsules, including packaging. Morrisville is where Novartis also plans to build a 56,200-square-foot facility focused on API manufacturing for solid dosage tablets, capsules, and RNA therapeutics, a project announced April 30. Manufacturing sites account for most of the combined $24.5 billion in new or expanded facilities with a potential 15,000+ new jobs that life sciences companies have announced statewide since 2021, according to the state-funded North Carolina Biotechnology Center. As for startups, Raleigh-based Slate Medicines launched in February with $130 million in Series A financing to fund development of therapies led by its migraine candidate, the anti-PACAP monoclonal antibody SLTE-1009 licensed from Zhongshan, China-based DartsBio Pharmaceuticals, and set to start Phase I trials in mid-2026.

    The Tar Heel State climbed to fourth in VC ($1.6 billion in 2025, $276.8 million in Q1 2026, both according to the state-funded North Carolina Biotechnology Center). But North Carolina showed consistency on the other criteria, ranking seventh in NIH funding (2,248 awards totaling $1.589 billion) and lab space (18.6 million square feet, according to JLL), and eighth in jobs (76,000, says the Center) and patents (5,992 families).

     

    8. Los Angeles / Orange County, CA

    Amgen, Thousand Oaks, CA
    Amgen executives mark the groundbreaking for the biotech giant’s $600 million center for science and innovation being built within its Thousand Oaks, CA, headquarters campus, set to integrate Research & Development and Process Development teams to smoothen the transition from drug discovery to commercial manufacturing. [Amgen]

    The region’s biopharma anchor Amgen broke ground last fall on a $600-million center for science and innovation being built within its Thousand Oaks, CA, headquarters campus, set to integrate research & development and process development teams to smooth the transition from drug discovery to commercial manufacturing. “With the first shovel in the ground, we’re reaffirming something essential: We discover here, we manufacture here, we deliver for patients from Thousand Oaks to all around the world,” Amgen chairman and CEO Robert A. Bradway said. Regional industry group BioscienceLA CEO Stephanie Hsieh recently acknowledged the region’s fragmentation as a challenge—from 88 cities in LA County alone, to the numerous county, city, and private agencies focused on growing the bioindustry— while citing strengths such as corporate anchors Amgen, Takeda Pharmaceutical, and Gilead Sciences-owned Kite Pharma, plus institutions like USC, UCLA, Cedars-Sinai, and City of Hope.

    California signaled interest in growing the region’s biopharma industry last August when the state-funded California Jobs First Regional Investment Initiative awarded $23.92 million to a coalition led by Los Angeles County’s Department of Economic Opportunity (DEO) toward four programs intended to create 10,000 jobs by 2030. Most of the money ($19 million) was approved for a DEO revolving loan fund to support startups, especially those looking to graduate from the Larta Institute’s commercialization and capital access accelerator into lab space within Los Angeles County. Larta was awarded $3.3 million to expand its Heal.LA Bioscience & Healthcare Accelerator and assist small startups via its Larta Impact Fund, a revolving loan fund.

    Los Angeles/ Orange County would still lead the nation in jobs, based on a year-old BIOCOM California tally of 155,571, which also includes San Bernardino and Ventura counties; figures run as low as 116,000, compiled last year for the four counties plus Riverside and Santa Barbara counties (regional industry group SoCalBio). The region finished seventh in patents (7,211 families), eighth in lab space (11.7 million square feet, according to JLL), and 10th in both NIH funding (1,911 awards totaling $1.243 billion) and VC ($500 million in 2025, zero in Q1 2026, according to PitchBook).

     

    9. Chicagoland

    AbbVie, North Chicago
    AbbVie plans to build two new active pharmaceutical ingredient (API) manufacturing facilities totaling $380 million at its campus in North Chicago, IL, where the biopharma giant is headquartered. [AbbVie]

    At least one developer has pivoted to a large non-biotech tenant to help fill a Chicago campus once envisioned as a life-sciences mecca: Trammell Crow in March inked a $100-million, 169,860-square-foot lease with candy/chocolate giant Mars to base 600 jobs at 400 North Aberdeen Street within the Fulton Market campus. Other biotech spaces are in the works: In North Chicago, Rosalind Franklin University of Medicine and Science plans to nearly double the size of its Helix 51 biomedical incubator to just under 13,000 square feet by adding 6,000 square feet of new lab and office space, citing growing demand from early-stage biotechs. The expansion is expected to create space for up to 10 additional companies. Also in North Chicago, home-grown AbbVie announced plans to build two new API manufacturing facilities totaling $380 million at its campus in the Chicago suburb. The facilities—designed to support production of next-generation neuroscience and obesity treatments—are set to be fully operational in 2029. However, AbbVie opted to build its planned $1.4-billion biomanufacturing campus not in North Chicago but 821 miles southeast in Durham, NC. Across Illinois, biotech stakeholders have applauded Gov. J.B. Pritzker (D) for proposing to sweeten the state’s Research & Development Tax Credit program by allowing companies to transfer their credits for cash. “This is a transformative step for our startup and growth-stage ecosystem,” stated John Conrad, president and CEO of the Illinois Biotechnology Innovation Organization (iBIO). Pritzker is seeking a third term in November vs. Darren Bailey (R).

    The Windy City and vicinity rank sixth in both NIH funding (2,658 awards totaling $1.607 billion) and jobs (94,000, according to statewide industry group Illinois Biotechnology Innovation Organization or iBIO). The region places ninth in patents (5,569 families) and VC ($917.677 million in 2025, says iBIO, zero in Q1 2026,

     

    10. Seattle

    AGC Biologics, Element Research Center facility in Bothell, WA
    AGC Biologics, a global CDMO, expanded its regional research footprint last fall by signing a 37,575-square-foot lease at Element Research Center in Bothell, WA. [AGC Biologics]

    Seattle and the Greater Puget Sound’s strong base of academic and other nonprofit research institutions helped the region achieve consecutive years of Nobel laureates: Mary E. Brunkow, PhD, of the Institute for Systems Biology in Seattle co-won the 2025 prize in Physiology or Medicine a year after David Baker, PhD, director of the Institute for Protein Design at University of Washington (UW), co-won the 2024 prize in Chemistry. A UW spinout, Seattle-based 3D tissue model developer Curi Bio, closed in December on a $10-million Series B financing led by South Korean contract research organization DreamCIS. In April, Achieve Life Sciences (based in Seattle and Vancouver, BC) announced an up-to-$354 million private placement whose purposes include funding a Phase III trial and future commercialization of e-cigarette cessation candidate cytisinicline, while Athira Pharma landed up to $236 million in conjunction with acquiring exclusive rights from Sermonix Pharmaceuticals to the Phase III metastatic breast cancer candidate lasofoxifene. AGC Biologics, a global CDMO, expanded its regional research footprint last fall by signing a 37,575-square-foot lease at Element Research Center in Bothell, WA. However, Astellas Pharma told Washington state officials in April it will shutter the Seattle site of its Universal Cells subsidiary by 2028, with 50 employees to be impacted via layoffs or transfers to South San Francisco, CA, or Westborough, MA.

    Seattle and its suburbs placed highest at eighth in both NIH funding (eighth with 1,892 awards totaling $1.572 billion) and VC ($1.06 billion in 2025, zero in Q1 2026, according to industry group Life Science Washington). The region was ninth in lab space (11.46 million square feet, according to regional real estate firm Flinn Ferguson Cresa) and 10th in both jobs (48,765 according to Life Science Washington) and patents (5,416 families).

    The post Top 10 U.S. Biopharma Clusters 2026 appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Turning the Patent Cliff into a Bioplant Opportunity https://edusehat.com/en/turning-the-patent-cliff-into-a-bioplant-opportunity https://edusehat.com/en/turning-the-patent-cliff-into-a-bioplant-opportunity Tue, 02 Jun 2026 03:05:17 +0700 Edusehat Turning, the, Patent, Cliff, into, Bioplant, Opportunity

    The 2030 patent cliff may either decimate revenue streams or provide an opportunity for innovation that can transform the biopharmaceutical industry. As it stands today, some 200 biopharmaceuticals are scheduled to go off patent during the next four years, representing approximately $300 billion in revenue.

    That revenue hit can be softened if biopharma manufacturers replace traditional mammalian expression systems with a Lemna plant-based system. Susan Stipa, CEO and co-founder of Phylloceuticals, tells GEN the Lemna platform her team has developed can reduce operational costs by nearly 80%–90% per gram in the upstream part of the process and one-third the cost overall. That’s because Lemna-based production lacks the 12-month lag and need for sterile growth media associated with mammalian cell lines and has less need for viral deactivation.

    Demonstrating those points, Phylloceuticals’ Lemna-based approach produced microgram quantities of the PD-1 inhibitor pembrolizumab in only 16 weeks. Batch harvesting garnered “yields of approximately 0.6 grams purified mAb per kilogram of fresh weight,” Stipa says.

    This isn’t how production has been traditionally handled, she says. So, “most companies are making defensive plays—such as mergers and acquisitions, reformulations, and reducing headcounts. But…what if the patent cliff could be an opportunity?”

    The duckweed advantage

    In optimal conditions Lemna, a genus of small free-floating aquatic flowering plants also known as duckweed, can double within 36 hours. In the wild, five to seven days is normal. “It’s one of the most prolific plants in the world,” Stipa points out. That rapid doubling time creates a huge speed advantage for line development and scale-up. “Line development speed for Lemna is four to six months versus 18+ months for Chinese hamster ovaries (CHO) cells,” Stipa says, “primarily due to Lemna’s genetic stability and clonal growth.” It boasts inexpensive, animal serum-free growth medium, no adventitious viruses, a negative carbon footprint, and uses about 10% of the water used by CHO cell systems to produce mAbs. And, she adds, “There is near-zero impact from unforeseen environmental deviations, like power outages.”

    Susan Stipa
    Susan Stipa
    CEO, Co-Founder

    Importantly, “As a multicellular eukaryote, it possesses the advanced chaperones and complex post-translational modification machinery—specifically sophisticated N-glycosylation—required to correctly fold and stabilize large, bioactive human molecules. Our ability with duckweed to control sugars and, in particular, obtain human (or human-like) sugar profiles is what sets us apart.”

    Those features make Lemna an attractive alternative to the more expensive CHO and other mammalian cell lines. CHO cells require a very complex system, and “thousands of CHO cells must be screened to find the cell that produces the right protein and remains genetically stable. There can be genetic drift, but with Lemna, there is none,” Stipa points out. Mammalian cells are sensitive to environmental fluctuations and require skilled technicians to manage them.

    “Almost anything you can make in mammalian cells, you can make in duckweed, just a little bit better. And, yes, we do a bit better with folding,” she says.

    Yeast such as Pichia pastoris or Saccharomyces cerevisiae is another option, but Stipa points out, “Yeast is a story of quantity versus quality. It can produce a lot very quickly, and it does simple proteins very well, but when the protein size and complexity increase, productivity drops.”

    Building where there’s a need

    That said, Phylloceuticalshas a potentially broad client base that includes individual investigators needing microgram quantities up to contract development organizations, biosimilar manufacturers, and innovators. The company is still young, though. “We need to prove the platform is what the industry wants it to be,” Stipa says.

    Stipa developed a comprehensive view of the industry as a young cancer patient and through a career as a chemical process engineer who built biopharma facilities globally, and as a life sciences marketer exposed to many companies.

    Her time in marketing, in fact, led to the formation of Phylloceuticals. “I had developed brand strategy for so many start-ups only to see the scientist-founders lose the room pretty quickly,” Stipa says. “In today’s media-saturated world, innovative science also needs advocates able to tell incredibly compelling stories, and to tell them so they stick.”

    In 2024, Stipa and her co-founders, Lynn Dickey, PhD, now chief technology and science officer, and Bill Brydges, one of the original leaders of bioengineering firm Foster-Wheeler Biokinetics, incorporated Phylloceuticals.

    The company became operational in January 2025, opening its pilot facility in Rapid City, South Dakota. “Our location choice perfectly mirrors the bio-agility of our platform,” Stipa says. “Traditional mammalian cell [production facilities] are often tied to very specific legacy pharma hubs. The idea of Phylloceuticals is that we can be up and running anywhere the need is, and in underserved regions. Rapid City is at the core of one of the largest rural healthcare areas [in the U.S.].”

    That the facility was operational in only 12 weeks helped Phylloceuticals transition from friends and family financing to angel investment. Stipa says she expects to close the company’s first funding round soon, “to be followed immediately by a Series A round.”

    Initial focus: biosimilars

    The company’s focus on biosimilars is directly related to the patent cliff and the industry’s widely discussed onshoring. The COVID pandemic highlighted a flaw in the global supply chain that left nations dependent upon others for critical pharmaceutical ingredients. Plugging that gap with the Biosecure Act (signed into law December 2025) and Federal Acquisition Regulations that ban commerce with companies of concern, Stipa says, makes Phylloceuticals an attractive choice for low-cost, onshore, mAb production. “Beyond biosimilars, we are also very active in animal health biologics and ADC/RTL support,” she adds.

    Regulators—notably the FDA—are familiar with Lemna because of its commercial-scale use for food, and for pharmaceutical products that have been through Phase II, including β-interferon, although it hasn’t been used commercially for pharmaceutical products. Commercial scale has been on Stipa’s mind since the beginning. “From the very early months, we had a team beginning to think about what scale-up would look like. Even when we didn’t have the funds, we had advisors working on the scaleup question,” Stipa says. Currently, Phylloceuticals can make microgram-to-gram quantities. Its next phase is to make gram-to-kilogram quantities.

    Challenges

    “I think pharma rarely fails because of the science,” Stipa says. She says the company is still improving extraction from the apoplast (the network of cell walls and intercellular spaces that help transport water and nutrients) and scaling to commercial quantities.

    Instead, the big challenge for Phylloceuticals is simply innovating in an industry that has a legacy, multi-billion-dollar investment in stainless steel infrastructure. “Change is hard,” she acknowledges. But change is also inevitable, and the biopharmaceutical industry is hardly the first to face entrenched legacy equipment and processes.

    As an example, she cites Kodak, which invented the first digital camera in 1975 but didn’t commercialize it. Aside from its initial technical immaturity, digital photography “would challenge the paradigm of film and chemicals Kodak sold,” Stipa points out. Yet, today, more than 90% of all photos are digital, and film photography is a relatively small niche. Clearly, she says, “It is possible to shift a legacy mindset.

    “Our challenge is to find forward-thinking leaders who believe the same way [we do],” Stipa continues. The first two customers have signed on—one engaged in preclinical studies around joint disease, and one focused on animal health—which suggests such leaders are there and are open to new ways of doing things.

    “The industry is at a crossroads,” Stipa says. “I see this as an opportunity to help our partners transition from ‘how we’ve always done it’ to a model of bio-agility.”

    Phylloceuticals

    Location: 800 N. King Street, Suite 304, Wilmington, DE 19801

    Phone: (484) 883-8808

    Website: phylloceuticals.com

    Principal: Susan Stipa, CEO and co-founder

    Number of Employees: 8

    Focus: Phylloceuticals has developed a plant-based expression system using Lemna (duckweed) as the bioreactor that has produced a mAb in 16 weeks, start to finish.  As a multicell eukaryote, Lemna has the cellular machinery necessary to correctly fold and stabilize large, bioactive human molecules.

    The post Turning the Patent Cliff into a Bioplant Opportunity appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Neuropixels Opto Integrates Electrophysiology and Optogenetics to Probe Neuronal Function https://edusehat.com/en/neuropixels-opto-integrates-electrophysiology-and-optogenetics-to-probe-neuronal-function https://edusehat.com/en/neuropixels-opto-integrates-electrophysiology-and-optogenetics-to-probe-neuronal-function Tue, 02 Jun 2026 03:05:16 +0700 Edusehat Neuropixels, Opto, Integrates, Electrophysiology, and, Optogenetics, Probe, Neuronal, Function High-resolution extracellular electrophysiology is typically used to record from neurons in order to understand brain function. Combining electrophysiology with optogenetics allows researchers to test the causal role of specific neurons by activating or inactivating those populations while recording the effects of neural activity.

    Now, a new technology, co-developed by UCL scientists, simultaneously records and manipulates neuronal activity deep within the brain. The device, known as Neuropixels Opto and researched in mice, integrates electrophysiology and optogenetics in a single probe, enabling unprecedented insight into how individual neurons in the brain function and interact. By packing around 1,000 closely spaced recording sites onto an ultra-thin probe, it is possible to capture high-resolution signals from individual brain cells while monitoring large neural networks at the same time. The device could transform our understanding of neural circuits and neurological conditions, such as Alzheimer’s disease and schizophrenia.

    “This makes it possible, for the first time, to directly test how specific neurons influence the activity of surrounding circuits—revealing causal relationships between neuronal activity and brain function,” notes Matteo Carandini, PhD, a professor at the UCL Institute of Ophthalmology. “The ability to both record and control neuronal activity in the same experiment represents a significant advance for neuroscience.”

    This work is published in Nature Methods in the paper, “Neuropixels Opto: combining high-resolution electrophysiology and optogenetics.” The device, which packs 960 electrical recording sites and two sets of 14 light emitters onto a 70-μm-wide, 1-cm-long shank, allows spatially addressable optogenetic stimulation with blue and red light. The device allows researchers to monitor the electrical activity of hundreds of neurons while also selectively activating or silencing specific cells using light.

    “The brain processes information through complex patterns of electrical activity, with billions of neurons communicating via rapid electrical signals,” explains Carandini. “Understanding how these signals give rise to behavior, thought and disease requires tools that can both observe and influence neuronal activity.”

    “Until now, scientists have typically relied on separate approaches: electrophysiological probes to record neural activity, and optogenetics to control it,” Carandini adds. “Combining the two has proved challenging, particularly in deeper brain regions, where delivering light without disrupting sensitive recordings is technically difficult. Neuropixels Opto overcomes these limitations by integrating both capabilities into a single device, enabling simultaneous measurement and manipulation of neural circuits.”

    Karolina Socha, PhD, research fellow at UCL Institute of Ophthalmology, has used the probes to investigate the function of the cerebral cortex. “We were surprised to discover that the activity of neurons in the cortex can be remarkably localized. Up to now, we thought that neurons are so interconnected that there would be no way to activate some of them without activating many others,” she said. “The new Neuropixels Opto probes revealed that these neurons can operate not only in concert but also rather independently.”

    The technology may also have important implications for understanding neurological and psychiatric conditions. Many disorders, including schizophrenia, Alzheimer’s Disease and Parkinson’s Disease, are associated with disruptions in how neurons communicate. By providing a clearer picture of how neural circuits function in both healthy and diseased states, Neuropixels Opto could support the development of more targeted treatments.

    The post Neuropixels Opto Integrates Electrophysiology and Optogenetics to Probe Neuronal Function appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     China has approved the world’s first invasive brain-computer chip—here’s what’s next https://edusehat.com/en/china-has-approved-the-worlds-first-invasive-brain-computer-chipheres-whats-next https://edusehat.com/en/china-has-approved-the-worlds-first-invasive-brain-computer-chipheres-whats-next Mon, 01 Jun 2026 23:35:02 +0700 Edusehat China, has, approved, the, world’s, first, invasive, brain-computer, chip—here’s, what’s, next
  • The world's first approved invasive BCI: A coin-size device called NEO, developed by Shanghai startup Neuracle Technology, beat Neuralink and others to become the first invasive BCI approved for use beyond clinical trials, now available to paralysis patients in China.

  • China is betting big on brain tech: Beijing has fast-tracked NEO into its national health insurance system and named its brain-computer interface industry as one of six sectors critical to China's future. It signals an acceleration that experts say has no comparable national-level ambition anywhere else in the world.

  • This isn't a race—it's two different games: While the US chases breakthroughs, China is focused on scale and accessibility. Also, despite geopolitical tensions, US-China collaboration in neurotechnology quietly continues, with American firm Axoft already running trials in Shanghai.
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    One day last October, sitting in the courtyard of his house in China’s Henan province, Dong Hui decided to see if he could hold a pen to write. 

    Dong, 39, had sustained spinal cord injuries in a car accident six years earlier that left him paralyzed from the neck down. Slowly but determinedly, he wrote his name, “Thank you,” and then the date. This was the result of an 11-month-long rehabilitation enabled by an implant in his brain. Before that process, Dong could move his arms slightly but wasn’t able to use his fingers.

    “I couldn’t believe I was able to write again. I was so excited I even missed a stroke in my name,” he told MIT Technology Review on a video call. 

    In November 2024, Dong became one of the first people in China to be given an invasive brain-computer interface (BCI) through brain surgery. He had signed up for a clinical trial with the device’s developer one month after seeing on TV how a BCI had apparently enabled another paralyzed Chinese man to hold his granddaughter. 

    This March, the implant Dong uses became the first invasive BCI product in the world to be approved for use beyond clinical trials. It’s now available to some patients with paralysis in their limbs due to spinal cord injuries. We spoke to a range of experts to understand why the device was able to reach this global milestone, what makes this moment so significant, and what to expect next. 

    A world first

    Dong’s brain implant is a coin-size device called NEO. It was developed by Neuracle Technology, a Shanghai-based startup, together with researchers at Tsinghua University in Beijing. 

    During a procedure that took just over an hour and a half, the device’s sensors, which collect Dong’s brain signals, were placed on his dura mater, the tough outer layer of tissue that covers and protects the brain. The signals are transmitted to a computer by an implant placed on Dong’s skull. The computer then translates the signals into commands for a soft robotic glove Dong wears during the 2.5-hour training sessions he completes each day to help him learn to grab. 

    Dong started his rehabilitation around a week after surgery. “On the ninth day of my training, my right hand successfully grabbed a ball without the glove,” he says. “That was a miraculous moment.” 

    Now he continues with his training at home. He wants to be able to control his hands better in order to put on clothes, eat, and do other daily tasks without troubling his aging parents. 

    A growing number of people with traumatic injuries in China are now poised to tread a similar path thanks to NEO’s recent approval. According to China’s National Medical Products Administration, the bureau responsible for drug supervision, the product is suitable for patients between 18 and 60 who have paralysis in all limbs due to spinal cord injuries but still have some residual function in their arms. 

    NEO beat several other BCIs to approval, including one from Neuralink, a California-based company founded by Elon Musk. Since October 2023, Neuracle has conducted 36 clinical trials using NEO, including the one on Dong. Thirty-two of them took place in the space of a few months in 2025, with the details about one of the four first in-person trials published in a preprint paper last July. Neuracle did not reply to a request for comment from MIT Technology Review.

    One reason for NEO’s fast approval could be that it has a “relatively less invasive” design than counterparts such as Neuralink’s N1 brain chip, says Avinash Singh, a BCI researcher at the University of Technology Sydney. NEO’s eight sensors sit on top of the brain’s protective membrane while Neuralink’s N1 chip directly penetrates the cortex, the outermost layer of the brain itself. Neuracle’s device faces fewer regulatory constraints because it presents a lower risk of hemorrhage, glial scarring, and long-term signal degradation, Singh says.

    China’s strong support for its BCI industry also means that NEO was put on an expedited regulatory pathway; in comparison, the approval process of the US Food and Drug Administration can take several years, Singh adds.

    A big boost for BCIs

    NEO’s approval is hugely important for the global BCI industry, says Wang Shouyan, a neuroscientist at Fudan University in Shanghai who was not involved in research or trialing for NEO. Even though research and development on BCIs has taken place for several decades, most of it happened in the lab. The news means that BCIs are now ready for large-scale manufacturing and clinical use in China, Wang says. 

    For Dong, however, it means something much more personal. “Now, it will be able to help not only me, but also thousands and thousands of other patients suffering from spinal cord injuries in China who are tortured by despair each day,” he says of NEO. “It will bring them hope and change their lives.” 

    Days after NEO was approved, China started incorporating it into the country’s health insurance system by assigning it a unique code. This is one of the first steps toward a future where eligible Chinese patients pay a certain percentage of the BCI’s price if they need it during their treatment.

    The growth of China’s BCI industry is expected to accelerate thanks to the government’s policy support and financial backing. The country’s latest five-year plan, published on the same day Neuracle received its approval, lists BCI as one of six key industries important to China’s future tech competitiveness, alongside quantum technology, humanoid robots, and others. Several Chinese startups, including NeuroXess and StairMed, have already worked in the field for many years. 

    “China’s decision to double down on becoming a global leader in the field owes in part to what these companies have already accomplished,” says Meicen Sun, an information scientist at the University of Illinois Urbana-Champaign who studies information and technology policy. 

    But, Sun says, the biggest advantage China may have is that Chinese people, particularly patients like Dong, tend to welcome this technology and are genuinely enthusiastic about it. In comparison, in the US and Western Europe, testing technologies on human bodies elicits an “ick factor,” triggering concerns and even resistance, she says.

    Cooperation in a cold climate 

    NEO has become the world’s first invasive BCI to go commercial, but scientists interviewed by MIT Technology Review caution against comparing Chinese and US efforts through the lens of a race. 

    A race implies an endpoint, but it is hard to say where that is for the development of BCIs, says Nick Ramsey, a neuroscientist at Radboud University Nijmegen in the Netherlands. Also, the US and China have fundamentally different visions, Sun says. The US is primarily concerned with being the first to do something and achieving state-of-the-art performance, while winning to China means capturing more consumers and using technology to deliver solutions on a societal scale. 

    “Being exceptional and being accessible are two diametrically opposed definitions of winning,” Sun says. 

    In fact, neurotechnology has emerged as a rare tech sector where US-China collaboration is still happening despite geopolitical tensions. The US company Axoft,  based in Cambridge, Massachusetts, says it has teamed up with a Chinese company and a hospital in Shanghai to test its BCI on four patients in China and has plans to expand its trials in the country. 

    Looking forward, China’s BCI industry is expected to speed up its growth over the next five years thanks to strong government support. “There is no comparable national-level ambition or coordinated map elsewhere in the world at the moment,” says Singh.

    More BCIs are also in the pipeline for domestic approval in the country, including Beinao-1, developed by the Chinese Institute for Brain Research in Beijing and its affiliated startup, NeuCyber NeuroTech. The device, which sits on the dura mater, is designed to help those who have movement and speech difficulties due to spinal cord injuries or amyotrophic lateral sclerosis. These candidates could get the green light as early as 2028, Singh says. 

    ]]>     From Discovery to Development in Emerging Modalities https://edusehat.com/en/from-discovery-to-development-in-emerging-modalities https://edusehat.com/en/from-discovery-to-development-in-emerging-modalities Mon, 01 Jun 2026 23:30:20 +0700 Edusehat From, Discovery, Development, Emerging, Modalities Sponsored content brought to you by

    ProBio logo

    The world of biologics is moving far beyond traditional monoclonal antibodies, and companies across the biopharmaceutical landscape are racing to keep pace. From multispecific antibodies to antibody-drug conjugates (ADCs) and antibody–oligonucleotide conjugates, emerging modalities are reshaping how researchers think about therapeutic development. ProBio is at the center of that evolution.

    As innovation accelerates, so do the challenges. New therapeutic formats demand not only scientific creativity, but also highly adaptable development strategies that can move quickly from concept to clinic. For ProBio, that means building flexible platforms capable of supporting the entire journey—from discovery to IND-enabling studies and CMC development.

    “The emerging modalities in antibodies are being used across multiple therapeutic areas,” says Jingyuan Zhang, PhD, content marketing specialist at ProBio. “So, we are looking at this entire field and the challenges that people are likely to face.”

    That broad perspective is becoming increasingly important as drug developers face growing pressure to optimize candidates earlier in the pipeline. According to Zhang, success with these next-generation therapies often depends on decisions made long before clinical development begins. “You need to consider early-stage design as much as possible,” she adds.

    That philosophy—front-loading strategy to reduce downstream risk—is a major theme in ProBio’s work. Whether developing ADCs with complex linker chemistry or designing multispecific antibodies that require careful balancing of efficacy and safety, the company emphasizes early-stage planning as a critical differentiator.

    This focus was also reflected in ProBio’s recent presentation at the American Association for Cancer Research (AACR) Annual Meeting, where the company highlighted its integrated approach to supporting emerging modalities. The presentation underscored how early molecular design, manufacturability considerations, and translational planning can dramatically improve timelines and outcomes for developers working in oncology and beyond.

    AACR served as a fitting stage for that message. As one of the leading global forums for cancer research, the conference showcased the growing industry interest in novel antibody formats and precision-targeted therapies. For ProBio, it was an opportunity to demonstrate how service providers must evolve alongside the science itself.

    “The science has moved on so much that it’s enabled companies to move to a modality-first approach,” says Tracy Humphries, head of U.S. & E.U. regional marketing at ProBio. “Twenty years ago, it was all about just antibodies. They delivered major successes. Then we saw a fundamental rise in next-generation technologies that have opened the door for companies to look at how they can address unmet clinical needs in ways that will be more efficient or more effective than what they’re using currently.”

    That shift creates enormous opportunity, but it also requires service providers to move faster than ever before. Emerging modalities are not static categories; they are rapidly advancing fields where platform capabilities must be built almost in real time. As Zhang says, “When a new modality emerges, we need to rapidly establish capabilities spanning discovery through IND-enabling studies and CMC development so that companies can achieve proof of concept as quickly as possible.”

    Rather than acting as a traditional contract development partner, ProBio sees itself as a strategic collaborator. “We aim to work with customers regardless of how their needs evolve, positioning ourselves as a collaborative partner,” Zhang explains. “This approach helps accelerate proof-of-concept generation.”

    As the therapeutic frontier continues to expand, the companies best positioned for success will be those that can bridge innovation with execution. For ProBio, that means staying ahead of scientific trends while helping partners design smarter, faster, and with the future in mind. In the era of emerging modalities, antibodies may still be the foundation—but they are no longer the full story.

    The post From Discovery to Development in Emerging Modalities appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Multiomics Mass Spec Workflows in Drug Discovery https://edusehat.com/en/multiomics-mass-spec-workflows-in-drug-discovery https://edusehat.com/en/multiomics-mass-spec-workflows-in-drug-discovery Mon, 01 Jun 2026 23:30:19 +0700 Edusehat Multiomics, Mass, Spec, Workflows, Drug, Discovery Advances in end-to-end multiomics platforms and the underlying scientific knowledge now enable faster and more precise biomarker discovery, mechanistic insight generation, and therapeutic design—core drivers of modern drug discovery programs. Within this integrated ecosystem, mass spectrometry-based metabolomics serves as a central analytical modality, offering the ability to quantify large numbers of metabolites from a single sample with high sensitivity and rapid turnaround.

    Metabolomics supports biochemical pathway-level interpretation, where a primary biomarker can be contextualized alongside upstream and downstream metabolites to inform target identification, pathway modulation, and pharmacodynamic response assessment. Rather than focusing solely on the discovery of novel metabolites, emerging approaches emphasize the identification of characteristic metabolic signatures that differentiate disease states, therapeutic responses, or mechanistic subtypes.

    Realizing this potential requires the development and deployment of AI enabled data analysis workflows that can reduce interpretation time, expand the breadth of detectable targets, and uncover complex patterns of metabolite perturbation. These capabilities ultimately enhance the precision and effectiveness of targeted therapeutic development.

     

    Taraka Donti, PhD, is director of lab services at Revvity Omics.

    The post Multiomics Mass Spec Workflows in Drug Discovery appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Macrocyclic Peptide Drugs Unlocked, Membrane Permeability Screened at Scale https://edusehat.com/en/macrocyclic-peptide-drugs-unlocked-membrane-permeability-screened-at-scale https://edusehat.com/en/macrocyclic-peptide-drugs-unlocked-membrane-permeability-screened-at-scale Mon, 01 Jun 2026 23:30:18 +0700 Edusehat Macrocyclic, Peptide, Drugs, Unlocked, Membrane, Permeability, Screened, Scale Macrocyclic peptides are a promising drug modality that combine the oral convenience of small molecules with the high specificity of large biologics. Yet, they struggle with cell membrane permeability, limiting their ability to target disease interactions within cells.  

    In a new study published in Nature Chemical Biology titled, “Generation of membrane-permeable cyclic peptides inhibiting protein–protein interaction”, researchers from École Polytechnique Fédérale de Lausanne (EPFL) have developed a new method to generate and screen large libraries of synthetic cyclic peptides to identify compounds that can enter cells for therapeutic effect. 

    “We focused on small, less than 1000-Dalton, non-polar cyclic peptides that can enter cells by rapidly crossing the hydrophobic inner region of cell membranes,” says Christian Heinis, PhD, associate professor at EPFL. “The challenge was then to develop cyclic peptides with suitable shapes so that they can bind to targets of interest.” 

    The authors focused on protein interactions linked to inflammation, oxidative stress, and neurodegeneration, and cancer. The study synthesized and screened a library of 15,360 fully random cyclic peptides, all designed to be small, compact, and relatively nonpolar to support membrane permeability. The screen identified several compounds capable of disrupting the disease-associated Keap1–Nrf2 interaction. 

    The team optimized a cyclic peptide candidate, termed peptide 30, which combined strong target binding with membrane permeability. Peptide 30 inhibited the Keap1–Nrf2 interaction inside living cells in a dose-dependent assay. Compared with the natural Nrf2 sequence, peptide 30 had no electrical charge, fewer hydrogen bond donors, and lower polar surface area to support membrane permeability.  

    The study demonstrated that membrane-permeable cyclic peptides can be developed without starting from known ligands, natural products, or binding motifs, broadening access to intracellular targets previously considered difficult to drug. 

    “Our lab is now further advancing the technology to synthesize and screen even larger libraries of small, membrane-permeable cyclic peptides,” says Heinis. “And we are applying the technology to some of the most challenging protein–protein interaction targets, including big cancer targets like KRAS, b-catenin and c-Myc.” 

    Heinis’s group has patented the method and founded the spin-off company Orbis Medicines, which recently raised more than €90 million in Series A funding to further develop and apply the technology for drug discovery. 

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    ]]>     Mass Spectrometry’s Discovery Revolution https://edusehat.com/en/mass-spectrometrys-discovery-revolution https://edusehat.com/en/mass-spectrometrys-discovery-revolution Mon, 01 Jun 2026 23:30:15 +0700 Edusehat Mass, Spectrometry’s, Discovery, Revolution Mass spectrometry (MS) has quietly undergone one of the most consequential evolutions in modern drug discovery. Once viewed primarily as a confirmatory analytical tool, it is now reshaping how researchers identify, validate, and optimize therapeutic candidates. Across chemoproteomics, metabolomics, immunopeptidomics, and beyond, MS is increasingly positioned not at the end of the pipeline—but at its beginning, where the most crucial decisions are made.

    “Mass spectrometry is no longer just a downstream analytical checkpoint,” says Aaron Robitaille, PhD, the senior director of product & vertical marketing of mass spectrometry at Thermo Fisher Scientific. “It is increasingly serving as a discovery engine.”

    This shift reflects a broader transformation across the pharmaceutical industry: from hypothesis-driven experimentation toward data-rich, systems-level interrogation of biology.

    Seeing biology more clearly

    Drug discovery has always struggled with a fundamental problem: Biology is complex, noisy, and often opaque. Many of the molecules that determine therapeutic success are low in abundance, transient, or entirely unknown. MS addresses this challenge by enabling researchers to observe biological systems with unprecedented depth and specificity.

    According to Robitaille, MS now supports nearly every stage of early discovery—from target identification and engagement to pharmacokinetics and mechanism-of-action studies. One of its most transformative applications is chemoproteomics, where researchers can directly measure drug-protein interactions within living cells. This enables scientists to evaluate not just whether a compound binds its intended target, but also whether it interacts with unintended ones.

    Crucially, MS is moving upstream in the discovery pipeline. “What makes that important is not merely breadth. It is timing,” Robitaille notes. By enabling high-throughput screening with detailed molecular readouts, MS helps eliminate poor candidates earlier—saving time, cost, and effort.

    Technological advances are driving this shift. Historically, researchers faced trade-offs between speed and sensitivity, or between targeted and untargeted analyses. Newer platforms are collapsing these compromises. Hybrid acquisition methods, for example, allow targeted and untargeted data to be collected simultaneously in a single experiment, enabling both hypothesis testing and discovery.

    The Thermo Scientific Orbitrap Astral Zoom MS exemplifies this convergence. Built around parallelized acquisition and enhanced ion handling, the system delivers high throughput, deep proteome coverage, and precise quantitation—all in one platform. Its ability to process hundreds of samples per day while quantifying thousands of proteins illustrates how MS is becoming both scalable and decision-ready.

    Interrogating biology at scale

    For Mike Knierman, biopharma workflow manager at Agilent, the expanding role of MS reflects the growing complexity of new therapies. “Drug discovery today spans multiple therapeutic modalities, including small molecules, monoclonal antibodies, oligonucleotides, and cell-based therapies,” he explains. MS provides a unifying analytical backbone across this diversity.

    One of the most significant recent developments, Knierman emphasizes, is MS’s ability to interrogate biology at scale. Techniques such as proteomics, metabolomics (See Sidebar), and lipidomics allow researchers to observe how candidate drugs perturb entire cellular systems, rather than isolated targets. This systems-level insight is essential for understanding the mechanism of action and identifying off-target effects early in development.

    Emerging measurements—such as protein turnover—are also enabling new therapeutic strategies. These include targeted protein degradation approaches, which require a detailed understanding of dynamic protein lifecycles rather than static abundance.

    Agilent’s Revident LC/Q-TOF platform reflects this trend toward intelligent, high-resolution analysis. Designed for accurate-mass performance with built-in diagnostics, the system incorporates features that automate quality control and maintain data consistency. Its ultra-fast detector supports a wide dynamic range without sacrificing resolution, enabling confident identification and quantitation in complex biological samples.

    Equally important are workflow innovations. The platform’s Intelligent Reflex capabilities automate routine checks—such as calibration verification and carryover detection—reducing manual intervention and ensuring consistent performance. In drug discovery environments where throughput and reproducibility are crucial, these features help maintain data integrity while accelerating timelines.

    Ultimately, Knierman highlights MS as a driver of “biology-driven discovery,” where decisions are guided by comprehensive molecular data rather than limited readouts.

    A shift in discovery models

    Todd Stawicki, senior global market development manager for pharma, SCIEX, places MS within a broader transformation of drug discovery itself. The industry is moving away from traditional in vivo models toward more complex in vitro systems—such as organoids and tissue-based assays—in an effort to reduce impacts to laboratory animals and rising global regulatory efforts.

    This shift dramatically increases the number and complexity of experimental endpoints. “Many or most of these endpoints are best served by mass spectrometry,” Stawicki notes. As a result, MS is becoming indispensable for analyzing the rich datasets generated by these models.

    MS is also deeply embedded throughout the discovery lifecycle. In the early stages, it supports proteomics and complements genomic studies. It plays a central role in hit identification and lead optimization, and remains crucial in ADME (absorption, distribution, metabolism, and excretion) and DMPK (drug metabolism and pharmacokinetics) studies.

    Analysis of a system suitability test and rat plasma matrix
    Analysis of a system suitability test (SST, top) and rat plasma matrix (bottom) injections on the SCIEX 7500+ system for three drug compounds shows coefficients of variation (%CV) of three to five percent across more than 10,000 injections of rat plasma. [SCIEX]

    Technological innovation continues to expand MS’s capabilities. Acoustic ejection-based MS, for example, enables rapid, label-free screening, while advanced systems—like the SCIEX 7500+ system—address one of the field’s most persistent challenges: balancing sensitivity with dynamic range.

    As new drug modalities become more potent and targeted, they often exist at extremely low concentrations in complex biological matrices. This creates a dual requirement for high sensitivity and a broad quantitation range. The SCIEX 7500+ system meets this need, enabling accurate measurement across diverse tissues and concentration levels.

    Robustness is another key consideration. SCIEX Mass Guard technology, for instance, enhances system uptime, ensuring that high-throughput workflows can run reliably over extended periods. In an environment where delays can be costly, this operational stability is as important as analytical performance.

    Balancing throughput and insight

    Shimadzu’s perspective underscores the importance of versatility in modern MS workflows. “Mass spectrometry has become one of the most versatile analytical tools in drug discovery,” says Lihini Mendis, PhD, LCMS product specialist at Shimadzu Scientific Instruments, noting that it now supports everything from early screening to preclinical development.

    Triple-quadrupole MS systems
    Triple-quadrupole MS systems can be used in drug discovery for bioanalysis and studies of drug metabolism and pharmacokinetics. [Shimadzu Scientific Instruments]

    A major recent trend is the push toward higher throughput without compromising data quality. Rapid LC-MS methods and triple quadrupole systems are increasingly used to process large sample volumes efficiently, particularly in quantitative workflows such as bioanalysis and DMPK studies.

    At the same time, qualitative MS capabilities are expanding. High-resolution instruments, combined with advanced fragmentation techniques, allow researchers to gain deeper structural insights into complex molecules such as lipids and metabolites. This dual capability—quantitative precision and qualitative depth—enables scientists to answer both “how much” and “what exactly” within the same experiment, Mendis explains.

    Shimadzu’s portfolio reflects this balance. Single-quadrupole systems provide accessible, high-throughput screening, while triple-quadrupole platforms emphasize stability and reproducibility for quantitative analysis. High-resolution instruments extend capabilities into accurate-mass analysis and structural elucidation, all while maintaining user-friendly operation.

    The overarching goal is not complexity for its own sake, but meaningful data that supports confident decision-making. By focusing on workflow efficiency and reliability, Shimadzu aims to streamline the path from data acquisition to actionable insight.

    A proteoform-centric vision

    While incremental improvements in speed and sensitivity have driven much of MS innovation, Bruker’s recently introduced timsOmni system points toward a more fundamental shift: a move toward protein-centric analysis at the level of intact proteoforms—structurally distinct variants of proteins that arise from genetic mutations, alternative splicing, or post-translational modifications.

    The platform introduces a multimodal trapping approach that enables precise control over ion reactions, supporting a wide range of fragmentation techniques. This flexibility allows researchers to tailor experiments to extract detailed structural information from complex biomolecules.

    Rather than focusing solely on peptides or simplified representations of proteins, the system emphasizes intact protein analysis. This is particularly important for identifying proteoforms. These variants often play critical roles in disease but are difficult to detect using conventional approaches.

    The timsOmni platform enables detailed mapping of such variations, including modifications, such as acetylation and glycosylation, that influence protein function and cellular signaling. By combining high sensitivity with advanced fragmentation methods, it allows researchers to generate comprehensive sequence information and localize modifications with precision.

    Importantly, this capability extends beyond discovery into biopharma development and quality control. The ability to characterize therapeutic antibodies and other biologics at the proteoform level has significant implications for both efficacy and safety.

    Supporting software further enhances this capability by translating complex spectral data into actionable insights. Advanced algorithms enable de novo sequencing, charge state assignment, and modification identification, making it easier for researchers to navigate the complexity of proteoform analysis.

    Accelerating insights

    As therapeutic modalities become more complex, the need for faster, more precise characterization tools has never been greater. David Curtin, vice president and general manager, biologics business, Waters Analytical Sciences, Waters Corporation, highlights how emerging platforms are enabling researchers to generate deeper insights earlier in the development cycle—when those insights can have the greatest impact.

    As one example, Curtin describes the Xevo CDMS platform as a breakthrough in capability and accessibility. As the first dedicated benchtop charge-detection mass spectrometry system, it enables measurement across a wide spectrum of mega-mass biomolecules. Crucially, it supports “characterization in process development when decisions matter most,” Curtin says, allowing teams to act on high-quality data in real time.

    Speed is one of its most transformative advantages. “Xevo CDMS delivers accurate analysis in less than 10 minutes,” Curtin explains. This represents a dramatic improvement over traditional workflows that could take hours, days, or even weeks when outsourced. The result is a shift to same-day decision-making, fundamentally changing how process development is executed and optimized.

    Efficiency is another key differentiator. Curtin notes that “the system requires up to 100 times less sample than current methods,” addressing a long-standing limitation in biopharma research. With reduced sample demands, scientists can run more experiments per batch, leading to “lower cost, higher yields, fewer impurities, and faster time to market,” he says.

    Beyond operational improvements, the platform unlocks new scientific possibilities. Curtin emphasizes that it delivers direct mass and charge measurements for individual 100-kilodalton to 150-megadalton molecules, including complex structures such as glycosylated proteins, viral vectors like AAV, and lipid nanoparticles. In many of these cases, “CDMS isn’t just a better option; it’s the only option,” Curtin says.

    Ultimately, Curtin underscores the broader impact: researchers are now generating “fast, accurate orthogonal data” that validates existing approaches while opening entirely new lines of inquiry. Scientists, he says, are “asking and answering questions they couldn’t tackle before”—a powerful indicator of how this technology is advancing the development of therapies for diseases including cancer, heart disease, and Alzheimer’s.

    From data to decisions

    Across all these perspectives, a common theme emerges: MS is no longer defined by its ability to generate data, but by its ability to inform decisions. This clarity is transforming drug discovery. By revealing off-target effects, validating mechanisms of action, and identifying biomarkers at early stages, MS helps reduce uncertainty and improve success rates. It allows researchers to prioritize the most promising candidates and eliminate those unlikely to succeed.

    As Robitaille puts it, the ultimate value of modern MS lies in “the ability to see meaningful biology early enough to act on it.” In an industry where time, cost, and complexity are ever-increasing, that capability might prove to be one of the most important advances of all.

    The post Mass Spectrometry’s Discovery Revolution appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Novel Therapeutic Modalities Target the Undruggable https://edusehat.com/en/novel-therapeutic-modalities-target-the-undruggable https://edusehat.com/en/novel-therapeutic-modalities-target-the-undruggable Mon, 01 Jun 2026 23:30:14 +0700 Edusehat Novel, Therapeutic, Modalities, Target, the, Undruggable From small molecules and protein therapeutics to gene therapies, biotech industry players have placed their bets on a wide range of modalities that push the limits of what was once considered “druggable.”

    AI biologics company, Absci, focuses on rational antibody design to bypass labor-intensive experimental screens. The ability to computationally design antibodies from scratch, or de novo, without reference to a known binder, could transform an antibody drug market projected to reach $445 billion within the next five years.

    Unveiled in January, the company’s latest protein design model, Origin-1, generated developability-optimized antibodies that achieved nanomolar binding affinity and functional inhibition of IL36RA, a therapeutic target for squamous cell carcinomas. By simulating the delivery of pro-inflammatory cytokine, IL-36, the AI-designed drug candidate boosts intratumor immune response for cancer control.

    Origin-1 generates de novo antibodies for “zero-prior” epitopes, or target sites that lack structural data from known protein-protein complexes. Sean McClain, CEO of Absci, emphasizes the approach as a “more expansive” version of de novo design that requires only a monomeric structure as input to generate viable candidates.

    Nathaniel Bennett, PhD, co-founder at Xaira Therapeutics, highlights that Absci’s atomic-level experimental validation contributes to the field’s understanding of how AI will play a major role in therapeutic development, particularly for expanding the range of tractable drug targets.

    “This is a solid piece of work that shows how AI-driven antibody design continues to mature,” says Bennett, “particularly in settings with limited prior structural information.”

    Janani Iyer, PhD, head of AI/ML product at Absci, emphasizes that the targets that most often strike interest from pharma partners are typically less studied and lack epitope structure in the public domain. “We’re focused on building an AI platform technology that unlocks really unmet needs,” she said.

    Permanently bound

    While highly precise therapeutics, biologics, such as antibodies, are typically constrained to intravenous delivery. A growing number of biotech companies are expanding the capabilities of small molecules, which offer the advantage of convenient oral administration.

    Unveiled from stealth last October, Expedition Medicines leverages generative AI to design small-molecule drugs that target shallow pockets using covalent chemistry. The Flagship Pioneering spinout targets a range of traditionally undruggable sensors, regulators, and transcription factors, where disease is driven by interactions across protein surfaces. These small molecules remain inert inside the body until activated by the appropriate protein catalyst.

    “Small molecules have historically been more challenging for generative AI, but I think we are at an inflection point, with the right chemistry insights, data, algorithms, and compute finally coming together,” said Molly Gibson, PhD, CEO of Expedition.

    small-molecule
    Expedition Medicines leverages generative AI to design small-molecule drugs that hit shallow pockets using covalent chemistry. The approach targets a wealth of traditionally undruggable sensors, regulators, and transcription factors, where interactions across surfaces drive disease.
    [Expedition Medicines]

    She notes that Expedition’s technology contrasts with many of today’s molecular design efforts, which use 3D atomic positions to model reversible interactions in deep pockets.

    The company’s tech stack trains AI models on high-throughput mass spectrometry data that measures the potency of each small molecule against 20,000 sites in the proteome. These fit-for-purpose datasets are advantageous over DNA-encoded libraries (DELs), which are burdened by substantial noise that can limit predictive power.

    Expedition is focusing on demonstrating clinical proof points. In a partnership with Pfizer, the startup is identifying target molecules correlated with prostate cancer disease progression and treatment resistance. As a long-term goal, the team plans to expand the proteomics platform to additional modalities, such as proximity events that drive protein degradation or stability.

    Biologic in a pill

    AI drug developer, 1910 Genetics, has recently tackled macrocyclic peptides, a class that aims to combine the oral convenience of small molecules with the high specificity of biologics. Historically, these compounds have struggled to balance cell-membrane permeability with key therapeutic properties such as potency and solubility.

    To address this gap, 1910’s AI model, PEGASUS, is trained on a multi-modal dataset that generates billions of cyclic peptides separated by permeability-related characteristics and solvent-dependent computational simulations. PEGASUS was able to demonstrate the first cyclic peptides with more than two polar or ionizable fragments to achieve in vitro cell-membrane permeability.

    Jen Asher, PhD, founder and CEO of 1910, describes the model as a “versatile tool” that accelerates the design-make-test cycle by triaging compounds for synthesis, supporting lead optimization, and designing new starting peptides with desired properties.

    With a company name that references the year that the first patient was diagnosed with sickle cell disease in the United States, the first condition for which the field identified a molecular basis, 1910 is committed to multi-modality drug discovery. The company’s platform also houses CANDID-CNS, an AI model that predicts small molecule blood-brain barrier (BBB) penetration within Beyond-Rule-of-5 (bRo5) chemical space to advance therapies for neurological disease.

    With only about two percent of small-molecule drugs able to cross the BBB, accurate penetration prediction can identify promising candidates that are more likely to succeed in the clinic. The model achieved an 87% success rate for predicting bRo5 small molecule brain penetration and distribution, outperforming a 56% success rate for the industry standard, Pfizer’s CNS Multiparameter Optimization (CNS-MPO) score.

    Encrypted message

    Jacob Becraft, PhD, CEO at Strand Therapeutics, is placing his bet on programmable mRNA therapeutics for cancers and autoimmune diseases. Strand is among a vibrant genetic medicine ecosystem, where engineered vehicles, such as adeno-associated vectors (AAVs) and lipid nanoparticles (LNPs), deliver therapeutic genetic material into patient cells to produce therapeutic proteins. These medicines must achieve therapeutic potency in the right tissues while avoiding off-target effects. Yet, targeted delivery beyond the liver remains a challenge.

    STX-005 illustration
    STX-005 extends the same programmable mRNA platform behind STX-001 to in vivo CAR T therapy, using circular RNA and targeted systemic delivery to generate CAR T cells directly inside the body. The approach is designed to produce long-term, cell-specific expression without the ex vivo manufacturing required by conventional CAR T. The program extends the company’s work in targeted, safe, and effective systemic delivery and has potential applications to autoimmune diseases and blood cancers. [Strand Therapeutics]

    Strand’s technology addresses this gap by enabling selective mRNA expression within cancer cells while sparing healthy tissue. This approach allows mRNA to be delivered broadly while targeting expression to the intended tumor cells.

    “It’s like an encrypted message. It doesn’t matter who picks up my message because they can’t read it,” Becraft said. “If the protein doesn’t get created, then it’s not off-target.” The tech stack challenges the “old school mentality” that mRNA biodistribution is the key metric that defines off-target effects.

    Strand’s technology leverages a machine learning–driven approach that applies molecular sensors to detect microRNA expression signatures distinguishing tumor cells from healthy cell types. As an example, liver-specific microRNAs bind to target sites in the 3¢ UTR of the delivered mRNA to suppress its expression in healthy hepatocytes and prevent off-target effects.

    Last May, Strand announced the Phase I dose-escalation trial for STX001, a programmable, self-replicating mRNA therapy designed to treat advanced solid tumors by producing IL-12 directly in the tumor microenvironment. Notably, STX001 demonstrated an abscopal response, in which localized treatment of a single tumor led to a systemic immune response that reduced distant tumor sites. The company looks to advance the candidate to Phase II trials.

    As the therapeutic toolbox continues to expand, the field is working to close the “undruggable” gap.

    The post Novel Therapeutic Modalities Target the Undruggable appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     The Confidence Gap: Why Drug Discovery’s Data Explosion Hasn’t Solved Its Billion-Dollar Decision Problem https://edusehat.com/en/the-confidence-gap-why-drug-discoverys-data-explosion-hasnt-solved-its-billion-dollar-decision-problem https://edusehat.com/en/the-confidence-gap-why-drug-discoverys-data-explosion-hasnt-solved-its-billion-dollar-decision-problem Mon, 01 Jun 2026 23:30:13 +0700 Edusehat The, Confidence, Gap:, Why, Drug, Discovery’s, Data, Explosion, Hasn’t, Solved, Its, Billion-Dollar, Decision, Problem Laurence Arnold
    Laurence Arnold, PhD
    Head of R&D
    Pelago Bioscience

    We’ve never had more data in drug discovery. Yet despite this explosion in capability, our industry’s most fundamental challenge remains stubbornly intact: making confident early decisions about which drug programs deserve billion-dollar investments, and which should be shelved.

    It costs two to three billion dollars to bring a drug to market, with a 90% failure rate, often higher. These numbers mask something more troubling. We’re not just failing because biology is hard; we’re failing because the mountains of data we’re generating aren’t giving us what we actually need at decision points that matter.

    In my view, we don’t have a data volume problem—we have a data relevance problem.

    Biological activity is not relevance

    Traditional drug discovery relies on a “dissect and build” approach: isolate one variable, measure it in a controlled environment, then extrapolate. It’s disciplined. It’s reproducible. And it has delivered important medicines.

    But the persistently high failure rate in drug development tells us we’re reaching the limits of this approach. In reality, biology operates through cascading networks, feedback loops, and context-dependent equilibria. These are dynamic biological systems where cause and effect rarely follow straight lines.

    We’ve successfully drugged only about 650 of 20,000 potentially druggable proteins. Not because scientists lack talent, but because for most targets, we don’t have robust ways to measure what matters—the initiating molecular event in a biologically relevant context.

    We’re good at measuring activity. What we struggle with is measuring relevance.

    An assay telling you a compound binds to your target protein is useful, but does it bind in living cells? In the disease context that matters? With the pharmacokinetics to reach patients? A compound brilliant in a purified enzyme assay might never reach its target in cells, or it might hit off-targets producing effects through entirely different mechanisms.

    The result? Ever-expanding data sets that still don’t answer the critical question in modern drug discovery: Are we making the right decision?

    The cost of borrowed confidence

    There’s a human dimension here that rarely makes it into industry discussions. Despite what is often repeated in drug discovery circles, scientists in R&D are rewarded for being right, not for being bold.

    Most scientists think in terms of “future hindsight”: will we look back and realize we missed something obvious? The responsibility isn’t to push programs forward at all costs. It’s to execute each step well, knowing that most will fail. Success stories often appear bold in retrospect. In practice, they are usually built on careful, incremental decisions that gradually improve the odds.

    So, teams do their jobs with discipline and rigor. They hit milestones, generate data, and advance programs. Everyone knows 90% of projects will fail, but this one has shown activity in the assay, has a plausible mechanism, and has momentum. The data might not be perfect, but it’s good enough to keep going.

    Until it isn’t. And the failure comes late, after years of effort and hundreds of millions spent.

    Of course, failure is how science advances. But many of these failures were avoidable earlier. Hard-working teams just didn’t have data that would let them make the call with confidence when it mattered most, before massive resources were committed.

    What decision-ready evidence looks like

    The best experiment isn’t always the one that moves your program forward—it’s the one that tells you when to stop.

    Think of it as taking a stepladder to look over a thick hedge rather than hacking through it with an axe. You might not learn everything about what’s inside it, but you’ll know much faster whether there’s anything worth pursuing on the other side.

    The pharmaceutical industry has been built on a model of going through the hedge, but the resource cost and timelines are increasingly untenable. So, what would an alternative, evidence-driven discovery model look like?

    Evidence-driven discovery requires a hierarchy of questions. Before optimizing potency or selectivity, can you prove that engaging this target in this context produces therapeutically relevant effects? Not in an abstract system, but in actual disease biology.

    This is about front-loading proof of concept before investing in optimization. Measure the initiating molecular interaction early, free from tags or unnatural expression control, in cells and tissues that approximate disease.

    It also requires new frameworks for proof of target engagement. We’re seeing this with technologies that measure binding in native cellular contexts, patient-derived models, and translational designs that test hypotheses much earlier in preclinical development. The goal isn’t replacing traditional assays, but knowing which programs deserve that investment.

    Ultimately, the win comes from making the right decision at each stage, even when that decision is to stop.

    The path forward

    Successful programs will establish coherent lines of evidence from initial target engagement through preclinical models to human proof of concept—and they will do it fast enough to fail early when evidence doesn’t align.

    This means rigorously testing hypotheses in the real biological context of disease before perfecting molecules or committing billions of dollars.

    Some will argue this is unrealistic—that you need optimized compounds, and that shortcuts lead to false negatives killing promising programs. These concerns aren’t wrong; they’re just insufficient when the old model demonstrably isn’t working.

    The real question is whether the risk of earlier translational testing exceeds spending nine years and a billion dollars on a target that was never going to work.

    Making the call with confidence

    Here’s what I tell my team: Your job isn’t to get a drug to the clinic. Your job is to do each step exceptionally well, building evidence you can defend. Because if we’re systematic about gathering the right evidence early, and if we’re honest about what the data is—and isn’t—telling us, the statistics start working in our favor.

    The industry is moving toward evidence-first approaches—technologies validating targets in relevant contexts, translational frameworks testing hypotheses earlier, and computational tools trained on quality data.

    But all this data is just noise until it answers the question keeping many of us up at night: Can I make this call with confidence, or am I crossing my fingers and hoping?

    We won’t solve the 90% failure rate entirely. Biology is too complex. But we can close the confidence gap by using the right data, at the right time, to answer the key question: Should we keep going?

    And sometimes—often, even—the most valuable answer will be no.

     

    Laurence Arnold, PhD, is the Head of R&D at Pelago Bioscience.

    The post The Confidence Gap: Why Drug Discovery’s Data Explosion Hasn’t Solved Its Billion-Dollar Decision Problem appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Next Generation Biopharma Innovation https://edusehat.com/en/next-generation-biopharma-innovation https://edusehat.com/en/next-generation-biopharma-innovation Mon, 01 Jun 2026 23:30:12 +0700 Edusehat Next, Generation, Biopharma, Innovation Researchers are digging deeper into biology’s complexity. In preclinical research, the traditional in vivo models are simply not enough to fuel the engine with the relevant translational data needed to progress successfully to the clinic.

    As research needs evolve in immunology and immune-oncology—as focus on neuroscience increases and metabolic drugs such as GLP-1-based therapeutics become more prevalent—in vivo model suppliers are being requested to up the game on new platforms. In response, these suppliers are expanding their humanization platforms while developing advanced models that can be used to study complex and overlapping disease biology.

    Regulatory factors also affect this market. The continued focus on the reduction of the use of animals by U.S. and European regulatory authorities has further opened the door to new approach methodologies (NAMs). NAMs are not new. Organ-on-chip or microphysiological systems, organoids, and iPSCs have been available for years. Finally, these systems are entering the limelight. Although the NAM market still requires more standardization across platforms, these systems are starting to impact preclinical research.

    Building translational engines

    The Jackson Laboratory (JAX) recently launched its latest humanized model, the NSG®-SGM3-IL15-MHC I/II DKO (S15-DKO). The S15-DKO represents their latest advancement in generating PBMC-humanized mice, supporting broad engraftment of immune cell subtypes such as CD4+ and CD8+ T cells, CD33+ myeloid cells, and CD16+/CD56+ natural killer (NK) cells. The knockout of the murine MHC Class I/II receptors delays the onset of Graft vs. Host Disease (GvHD).

    Profile of the NSG-SGM3-IL15-DKO
    S15-DKO model is JAX’s latest advancement in generating PBMC-humanized mice, supporting broad engraftment of immune cell subtypes. The knockout of the murine MHC Class I/II receptors delays the onset of Graft vs. Host Disease (GvHD). [The Jackson Laboratory]

    The model also supports the engraftment of rare immune cell subsets, including gd T cells and CD19+/CD38+ B cells that retain the memory state of the donor PBMCs.

    Another advanced model for CD34+ hematopoietic stem cell (HSC) humanization, the NSG-FLT3-IL15 mouse generates a cellular-diverse human immune system encompassing myeloid cells, mature NK cells, functional dendritic cells, and T cells.

    Both models are available in naïve strains, or off-the-shelf pre-characterized PBMC- and HSC-engraftment, along with full preclinical services tailored to immuno-oncology and autoimmune drug discovery.

    “With the FDA’s renewed focus on reducing reliance on non-human primates in biologic development, demand for validated, translational preclinical models has never been higher,” said Luke Dimasi, senior director, JAX.

    The genetically humanized FcRn platform and the newly expanded Tg32 hALB mouse address this need. Lacking murine Fcgrt and albumin while expressing their human counterparts, the Tg32 hALB is the first model for studying the pharmacokinetics and pharmacodynamics of human albumin therapeutics, as well as human IgG and Fc-domain-based biologics. Preclinical mAb testing services are available.

    “Our offering extends beyond the vivarium,” Dimasi emphasized. JAX’s iPSC repository continues to grow with engineered lines carrying disease-relevant mutations linked to Alzheimer’s, Parkinson’s, ALS, and frontotemporal dementia. In 2025, JAX added HALO-tagged and TET-inducible lines to the collection. The acquisition and integration of the New York Stem Cell Foundation (NYSCF) brings complementary patient-derived iPSCs to the portfolio.

    “As the field moves towards new approach methodologies (NAMs), we are evolving alongside it,” Dimasi pointed out. “Our in vivo mouse capabilities give us decades of deeply validated biological context. We are now layering human iPSCs and AI-computational phenotyping on top of that foundation to build a convergent translational engine that no single approach could deliver alone.”

    Developing relevant models

    According to Jason Rashkow, PhD, product manager for research models, Charles River Laboratories, the company’s comprehensive collection of spontaneously developing rat models spans metabolic disease, diabetes, hypertension, and heart failure, providing strong translational relevance across cardiometabolic indications.

    Custom diet preconditioning services allow researchers to tailor disease progression to specific study objectives through strategic model selection and diet design. Standardized preconditioning offerings are planned. “This approach will accelerate study initiation, giving researchers faster access to these metabolic disease models,” said Rashkow.

    The increasing prevalence of GLP-1-based therapeutics and next-generation incretin and poly-agonist therapies expanding into cardiometabolic indications such as heart failure with preserved ejection fraction (HFpEF) is accelerating demand for advanced disease models. The combination of established disease models, standardized preconditioning approaches, and custom solutions reflects the complexity of modern metabolic drug development.

    In addition, optimization of the generation of CD34+ HSC-humanized mice continues. These models, developed on the severely immunodeficient NCG strain, support research in immuno-oncology, autoimmune disease, vaccine research, and related fields.

    As immuno-oncology research needs shift, so does the need for models that enable the study of NK cell-based therapies, tumor microenvironment reprogramming, and cancer vaccines. “Although variant NCG models expressing human cytokines or HLA transgenes begin to meet these needs, transgenes can influence humanization requirements,” Rashkow noted.

    To counteract this, the company expanded access to a peripheral blood mononuclear cell (PBMC) engrafted NCG variant strain carrying a double knockout for murine MHC class I and class II, which significantly delays the onset of GvHD, allowing for longer-term studies in the context of mature T cells.

    To better support researchers studying HLA-A2-restricted immune responses in vivo, humanization optimization of a NCG variant expressing human HLA-A*02:01 was completed. Further development of the humanization protocols for other variant strains will support next-generation immunotherapy discovery and translational research.

    Lastly, the expanded aged C57BL/6 mouse offerings support researchers investigating age-related disease. As a licensed distributor of JAX® Mice to researchers in Europe and Asia, Charles River Europe can now provide aged C57BL/6J mice up to 90 weeks of age. In North America, Charles River offers aged C57BL/6N mice up to 77+ weeks of age.

    Improving translational fidelity

    “Improved translational fidelity, increased demand for study-ready systems that better align with clinical endpoints, and the need to model complex and overlapping disease biology are driving model development,” related Michael Seiler, PhD, vice president of portfolio management, Taconic Biosciences.

    Complex modalities such as checkpoint inhibitors and engineered cell therapies require more complete immune system function and deeper phenotyping. Expansion of the FcResolv® NOG portfolio and huSelect™ services reduces murine immune interference and donor variability. Advanced flow cytometry panels support deeper, standardized

    immune profiling.

    Animals Alternatives
    With the goal of improving translation relevance, Taconic develops in vivo models that reflect complex and overlapping disease biology in immunology, immuno-oncology, neurobiology, and cardiometabolic indications. [Taconic Biosciences]

    Planned launches include platforms and models designed to support immuno-oncology, biologics, engineered cell therapies, infectious disease, and autoimmune research, with a focus on more complete and functional human immune system biology. Gene and protein analysis services are available.

    In neuroscience, the shift is toward better alignment with clinical disease biology, particularly in Alzheimer’s disease and neuroinflammation, along with increased focus on blood-brain barrier (BBB) biology and CNS delivery. Parkinson’s disease model offerings include aSyn KI/KO, PINK1 KO, and LRRK2 KO rat models.

    Future models include BBB-focused platforms such as TFRC and CD98, ARTE10 crosses with BBB models, and neuroimmunology-focused NOG variants, including IL-34 and TREM2-related models.

    The rapid growth of obesity therapeutics, including GLP-1 and next-generation incretin approaches, is accelerating demand for more predictive metabolic and liver models in cardiometabolic disease. A range of models are aimed at obesity, MASH, cardiovascular disease, and DMPK applications.

    Taconic is expanding its capabilities in transgene characterization, CRISPR off-target analysis, and tiered Custom Model Generation Solutions. The acquisition of TransCure bioServices significantly bolsters support of integrated in vivo study services, particularly in humanized immune system and immuno-oncology research. “We now offer a more seamless, end-to-end solution from model selection through study execution and data generation,” said Seiler.

    “We continue to evolve toward integrated solutions rather than standalone models. This includes expanded CMS and CMGS capabilities, humanization-as-a-service, deeper phenotyping and multiomic analysis, and partner-enabled data generation,” Seiler added.

    Importantly, the move toward integrating in vivo models with complementary technologies such as organoids, iPSCs, and AI-enabled analysis will influence how models are developed and deployed within research workflows.

    Standardizing NAMs

    The field is clearly shifting toward ready-to-use biology, producing a strong demand for standardized NAM platforms and services that deliver consistent, high-quality results. To facilitate scientists, MIMETAS continues to develop robust OrganoReady® models and advanced services, including immune-competent and vascularized systems across multiple organs.

    “Last year, we strengthened our fee-for-service capabilities and advanced several models to deliver high-quality biology in a consistent, scalable way,” said Paul Vulto, PhD, co-CEO and co-founder, MIMETAS. “We made strong progress in our kidney tubuloid research program, CAR T-related applications, and a BBB model under unidirectional flow.”

    The novel human distal nephron-on-chip model in the OrganoPlate® replicates physiologic sodium and water transport using primary human kidney cells. This three-dimensional microfluidic platform, as detailed in Kidney360, serves as a high-throughput tool for functional drug screening and investigating distal nephron physiology and disease.1

    A polarized kidney tubuloid in an OrganoPlate chip
    A polarized kidney tubuloid in an OrganoPlate chip showcases apical and basolateral access. Immunofluorescence 3D reconstruction demonstrates tubule polarization and barrier formation: blue, DNA; red, acetylated tubulin; and green, Na /KATPase. [MIMETAS]

    In addition, a three-dimensional BBB microvasculature model developed on the OrganoPlate Graft 48 UniFlow was evaluated in a recent Fluids Barriers CNS publication. Tri-cultures of endothelial cells, pericytes, and astrocytes were used to demonstrate that this pump-free, unidirectional perfused, three-dimensional BBB model outperformed simpler systems on vascular architecture and barrier function. Its high-throughput nature renders the model suitable for studies of BBB function in health, disease, and therapeutic development.2

    This year, the company’s UniFlow technology will be offered for in-lab use, enabling customers to create a stable, perfusable vascularized bed for endothelial tissues. New OrganoServices for gastrointestinal toxicity (GI tox) and drug-induced vascular injury (DIVI), alongside a multi-donor expansion of the OrganoReady Colon Organoid product, are also planned.

    A major trend in NAMs is the increased need for standardization and regulatory alignment across the field. With initiatives like IAMPS (Industry Alliance for MicroPhysiological Systems), of which MIMETAS is a founding member, industry innovators will work together to advance regulatory acceptance.

    The space is evolving quickly, but Vulto emphasized that their focus remains unchanged: building robust human models that help researchers make better decisions.

    Improving organoid access

    “Organoids are part of a broader innovation focus to help researchers work with more predictive models, more advanced tools, and more connected workflows across the path from discovery to development,” commented Heather Hargett, PhD, head of cell biology reagents franchise at MilliporeSigma, the U.S. and Canada Life Science business of Merck KGaA, Darmstadt, Germany.

    The regulatory landscape is becoming increasingly favorable to NAMs. In March 2026, the FDA issued a draft guidance to establish clear validation principles for NAMs, including organoids and in silico (or AI) models, when submitted in support of drug applications.

    Phasing out animal use for research and regulatory purposes is also supported by the European Commission’s Roadmap Towards Phasing Out Animal Testing for Chemical Safety Assessments.

    Patient-derived organoids
    Patient-derived organoids (PDOs) retain individual genetic and phenotypic characteristics, enabling drug response testing across diverse patient backgrounds and disease subtypes. The image shows immunocytochemical (ICC) characterization of human colon PDOs that are positive for the colon-specific marker CA II (green), nuclei (blue) and actin (red). [MilliporeSigma, the U.S. and Canada Life Science business of Merck KGaA, Darmstadt, Germany

    HUB’s advanced organoid capabilities are now being combined with the company’s cell culture expertise, manufacturing scale, global commercial reach, and broad life science portfolio to make organoids a more practical and scalable tool in drug discovery and translational research.

    Key priorities include expanding the validated organoid biobank across additional therapeutic areas, tissues, disease states, and patient backgrounds. “Last October, we announced a strategic partnership with Promega Corporation,” said Hargett. “By combining our organoid expertise with Promega’s advanced reporter technology, we aim to enable high-throughput screening that helps researchers identify safer and more effective drug candidates.”

    The case of petosemtamab, developed by Merus, is a notable example of the real-world impact of organoid technology. Petosemtamab’s efficacy was tested using HUB organoids. The EGFR x LGR5 bispecific antibody has received FDA Breakthrough Therapy Designation for use in combination with pembrolizumab for first-line treatment of PD-L1-positive recurrent/metastatic head and neck squamous cell carcinoma (HNSCC). A global Phase III trial is ongoing. Recently, Genmab acquired Merus for approximately $8 billion USD.

    Adopting organoid technology is a capital efficiency strategy, according to Hargett. Patient-derived organoids retain individual genetic and phenotypic characteristics, enabling drug response testing across diverse patient backgrounds and disease subtypes. Organoids support a “fail fast” approach by identifying non-viable candidates earlier, reducing costly late-stage clinical trial failures, and allowing companies to redirect resources toward the most promising programs.

     

    References

    1. Bernardi MDL, Dilmen E, Kurek D et al. A Novel Human Distal Tubuloid-on-a-Chip Model for Investigating Sodium and Water Transport Mechanisms. Kidney360. 2025 Nov 1;6(11):1981-1993. doi: 10.34067/KID.0000000992.
    2. Admiraal J, Emeh PO, Bokkers M et al. Building the blood-brain barrier: a scalable self-assembling 3D model of the brain microvasculature under unidirectional flow. Fluids Barriers CNS. 2026 Jan 23;23(1):29. doi: 10.1186/s12987-026-00765-x.

    The post Next Generation Biopharma Innovation appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     State of the Diagnostic Industry: Recombinants on the Rise https://edusehat.com/en/state-of-the-diagnostic-industry-recombinants-on-the-rise https://edusehat.com/en/state-of-the-diagnostic-industry-recombinants-on-the-rise Mon, 01 Jun 2026 23:30:11 +0700 Edusehat State, the, Diagnostic, Industry:, Recombinants, the, Rise

    Discovery of a fragile foundation

    Four years ago in GEN, Scripps Laboratories predicted that the clinical diagnostic industry was on the verge of a recombinant protein revolution. At the time, in vitro diagnostic (IVD) assay developers were opposed to using recombinant proteins as replacements for proteins derived from human or animal tissues, glands, organs, and fluids, so-called “native” proteins. The pushback was vigorous, even palpable.

    David A. George
    David A. George
    Director, Product Research
    Scripps Laboratories

    Today, the transition to recombinants is underway, as they are being approved and adopted in IVD assays around the globe. I witnessed firsthand the shortage of native starting materials and helped drive this shift by developing recombinants suitable for the IVD industry. Recombinants are now the most responsible option in many diagnostic areas for laboratories that care about long‑term risk management, supply chain resilience, sustainable sourcing, and price stability.

    The IVD industry relied far too long on a surprisingly fragile supply network. Many of the proteins used in diagnostic assays are purified from starting materials obtained from human donors, or from abattoirs in the case of animal-sourced materials. For decades, this system appeared satisfactory: native materials were available, performance was good, and IVD assays were being produced to meet global demand. The system appeared sustainable, and there was no visible reason to change; that is, until there was.

    Native sourcing becomes unsustainable

    The erosion of the native starting material supply chain was not a single, isolated event. It occurred over many years, even decades. Today, native raw materials for critical proteins in several diagnostic areas are unavailable in the quantities needed to support the growing IVD industry.

    Going back 10 to 15 years, human hearts and livers were becoming increasingly expensive and difficult to obtain. In addition, the quality of the donor organs made available to material manufacturing companies was deteriorating severely. Many organs were either resected or visibly diseased. The poor-quality hearts yielded less and less of the cardiac biomarkers creatine kinase MB (CK-MB), troponin I (TnI), and troponin T (TnT). Similarly, yields of the iron-storage protein ferritin from human livers decreased precipitously.

    Pituitary glands have a similar story of declining availability and spiking costs. Pituitaries are the source of follicle-stimulating hormone (FSH), luteinizing hormone (LH), prolactin (PRL), and thyroid-stimulating hormone (TSH). These hormones are essential to testing in reproductive medicine (FSH, LH, PRL) and thyroid disease (TSH). The pituitary gland is small, the size of a pea, and each human has only one. Several thousand glands are needed, from several thousand donors, for a single pituitary gland-extraction batch. Given the growing size of the reproductive and thyroid testing markets, such large-scale consumption of this limited resource was not sustainable.

    Animal-derived proteins are not immune to such supply chain disruptions. Changes in how porcine stomachs are processed at abattoirs around the world significantly reduced the intrinsic factor content available for purification. Porcine intrinsic factor has a high affinity for vitamin B12 and has been used for decades in metabolic diagnostics as the binding reagent in B12 assays. With the new stomach excision process resulting in lower yields, producing native intrinsic factor has become more challenging and expensive.

    One telling indicator that some areas of the native protein model are under strain is the behavior of IVD assay manufacturers themselves. Many long-standing hormone customers have implemented a “last time buy” strategy, purchasing native hormones in quantities to last three to five years. This tactic may bridge a short-term gap, but it signals a deeper, industry-wide revelation: continuing to build assay portfolios on such vulnerable raw materials is not aligned with long-term risk management.

    From skepticism to necessity

    When companies began presenting recombinant alternatives to the IVD industry, the reception was cool. Many companies would not entertain a discussion about recombinants, let alone consider evaluating them. The conventional wisdom was that native proteins were inherently superior in immunoassays, particularly for structurally complex proteins, like the 24-subunit ferritin molecule, or for glycosylated, two-subunit proteins, like FSH, LH, and TSH. To be fair, the recombinants available 10 or 20 years ago were not produced with the IVD industry in mind and did not perform up to industry standards.

    In only a few years, the IVD industry’s attitude toward recombinants has shifted dramatically. A willingness to evaluate them as replacements for native proteins has spread across the globe. The same diagnostic laboratories that refused to have a conversation about recombinants four years ago are now proactively soliciting their suppliers for recombinant alternatives to native proteins. Many global IVD leaders have implemented a mandate to switch to recombinant proteins wherever a native protein may be considered at risk of a raw material shortage. Furthermore, when a new assay is being developed, a “recombinant-first” approach is now the norm.

    I have also witnessed a cultural element to the shift. Some of the larger IVD companies have said that their scientific staff was reluctant to switch away from native proteins, but that the transition to recombinants is happening, regardless. This, too, demonstrates a broader understanding in the industry of the fallibility of the old native model.

    Recombinants taking over

    The most swift and dramatic transition to recombinant hormones is occurring in the fields of reproductive biology (FSH, LH, PRL) and thyroid disease (TSH). Historically, recombinant forms of these hormones performed poorly, so the resistance to evaluating recombinants was strong. As the supply of pituitary glands contracted, however, assay manufacturers were forced to confront the vulnerability of their supply chain. Fortunately, having inside knowledge of the pituitary supply constraints, our laboratory set out early to develop recombinant forms of these hormones. By the time the supply crisis hit, we were prepared with a full line of IVD-assay-tested recombinant hormones.

    Structure of recombinant bovine chymosin
    Credit: vdvornyk/ iStock / Getty Images Plus

    The response in the industry has been decisive and far-reaching. Most customers for native hormones have now tested, approved, and switched to recombinant versions. This change did not occur because native hormones suddenly became unusable, but because their supply became incompatible with the magnitude, reliability, and planning requirements of the industry. By contrast, recombinant hormones can be produced at scale in controlled systems with consistent quality and predictable availability.

    Cardiovascular diagnostics are following a similar path. Recombinant TnI, TnT, CK-MB, and myoglobin are being adopted quickly as replacements for the native forms derived from human hearts. The supply of suitable organs cannot keep pace with industry demand, as cardiovascular disease is on the rise globally and the growth of point-of-care testing continues. Recombinant cardiac markers offer a solution to organ supply shortages, meeting the industry’s high demand for these proteins, while maintaining the performance characteristics IVD laboratories expect.

    In anemia and metabolic diagnostics, the switch has not been immediate, but it is underway. Recombinant apoferritin (ferritin without iron) and recombinant human intrinsic factor are available to replace the native proteins, and they are being evaluated and approved. The global supply of native ferritin and intrinsic factor is diminishing, but the situation is not as dire as with heart- and pituitary-derived proteins. Thus, the transition is progressing, but is not as far along.

    Keys to producing recombinants

    To justify switching to a recombinant protein, the recombinant must perform comparably to the native protein it is intended to replace. Early recombinants did not perform well, resulting in the skepticism seen initially. In antibody-based assays, even subtle structural differences can translate into poor recognition, reduced sensitivity, or altered calibrator performance. Overcoming these issues requires more than simply expressing a protein in a convenient host; it requires a project development and testing strategy tailored to the nuances of IVD assay development.

    At Scripps, our intention was to devise and implement a strategy that would produce recombinants suitable for the IVD industry. The process involves appropriate gene, expression vector, and host cell line selection; tagless protein expression; early and extensive testing in antibody-based systems, including clinical analyzers; and a willingness to revisit any or all of these elements if the desired recombinant is not produced.

    This development strategy addresses the concern about recombinant protein performance in the IVD industry. When a recombinant biomarker performs well and can be supplied consistently, without relying on the unstable supply framework of donor materials, the recombinant becomes not just an acceptable option, but the preferred one.

    Looking ahead

    The IVD industry is at an inflection point, bending toward global acceptance of recombinant biomarkers. The constraints on native tissue supply and quality will not ease; in fact, they will likely intensify. Simultaneously, industry expectations surrounding ethical sourcing, supply chain stability, risk mitigation, and long-term cost control will become more stringent. Given this environment, continued reliance on donor materials is difficult to justify and is perhaps foolish.

    Recombinant proteins offer a way forward that unites consistent assay performance with sound business judgement. Disconnected from unreliable tissue supply networks, recombinants support sustainable and ethical sourcing practices, providing IVD assay manufacturers with a stable foundation for planning and growth. The experience of recent years—in reproductive biology, cardiology, and thyroid disease in particular—has shown that when recombinants are developed with clinical assay performance in mind, they can match or even exceed the standards set by native proteins.

    I have seen the industry’s view of recombinants evolve from skepticism to necessity. Focusing on tagless expression and rigorous early testing, recombinants can be produced not as lesser-quality replacements, but as robust solutions. As assay developers and IVD executives look ahead to the next decade of innovation, recombinants are no longer a speculative option. They are the most responsible path toward assuring the continued availability of the tests that patients and clinicians rely on daily.

     

    David A. George is director of product research at Scripps Laboratories.

    The post State of the Diagnostic Industry: Recombinants on the Rise appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     House hearing to examine bill balancing data privacy and R&amp;D needs https://edusehat.com/en/house-hearing-to-examine-bill-balancing-data-privacy-and-rd-needs https://edusehat.com/en/house-hearing-to-examine-bill-balancing-data-privacy-and-rd-needs Mon, 01 Jun 2026 20:00:03 +0700 Edusehat House, hearing, examine, bill, balancing, data, privacy, and, R&D, needs Researchers utilize health data to inform medical breakthroughs, but patients need to know their personal information stays confidential.

    The current patchwork of state laws regulating health data privacy does not provide American patients with consistent protection and creates a challenging environment for biotech innovators. Federal legislation to create a single regulatory framework that protects consumer privacy while enabling research will be examined in a June 3 Congressional hearing. The effort is supported by the Biotechnology Innovation Organization (BIO).

    “Navigating upwards of 20 distinct state data privacy laws creates immense operational challenges for biopharma companies, distracting critical resources away from clinical innovation and patient care,” according to Patrick Plues, BIO Senior Vice President, State Government Affairs. “Because data-driven research and digital health solutions do not stop at state lines, a single, comprehensive federal consumer data privacy standard is essential to provide uniform protection for patients while giving the industry the regulatory certainty needed to develop the next generation of life-saving therapies.”

    The House Energy & Commerce (E&C) Subcommittee on Commerce, Manufacturing & Trade hearing will examine the SECURE Data Act, a law providing clarity on handling all kinds of personal and financial data. It would allow consumers to obtain and delete their data held by a controller and to opt out of processing of their data. It would also permit appropriate use of clinical data that is driving medical innovation and scientific breakthroughs.

    “Unlocking the power of health care data to fuel innovation in medical research is at the heart of today’s health care revolution, where medicine is increasingly a collaboration between data science and clinical science realms,” according to written comments BIO submitted to the E&C Committee last year.

    Federal versus state protections for health data

    States have been seeking to protect personal data in general, and health data specifically, with their own regulations since the passage of the California Consumer Protection Act (CCPA) in 2018, followed later by Virginia’s 2023 VA Consumer Data Act. Washington State’s health-specific “My Health My Data Act” of 2024 has inspired other states to develop similar legislation.

    These policies may protect privacy, but they also create confusion for anyone handling data from more than one state.

    To ensure consistent health data protections, the SECURE Data Act would take advantage of existing federal privacy protections, including the Health Insurance Portability and Accountability Act (HIPAA) Privacy Rule, which sets a national standard for protection of health data.

    “Since its inception in 1996, experience has shown that HIPAA Covered Entities and Business Associates subject to the Privacy Rule have responsibly protected patient data,” per BIO’s comments to the E&C Committee. “HIPAA recognizes the careful balance between protecting patient privacy and facilitating biomedical research.”

    Other federal rules protecting health data include Food and Drug Administration (FDA) regulations for patients involved in clinical trials. These protections have successfully governed the rules for processing clinical data and allow for innovation in biotechnology for decades.

    Relying on proven federal regulation when applicable makes for more effective policy, according to Aiken Hackett, BIO’s Executive Vice President, Federal Government Relations.

    “The SECURE Data Act recognizes there are areas of health policy that are already doing the right things,” Hackett says. “It allows for clearer regulation that provides consistent patient protection while empowering biotech innovation and investment.”

    Read more about the June 3 hearing here. Watch it live or see a recording here.

    The post House hearing to examine bill balancing data privacy and R&D needs appeared first on Bio.News.

    ]]>     Measuring Direct, Bystander, and Off-Target ADC Killing with the HiBiT TCK Platform https://edusehat.com/en/measuring-direct-bystander-and-off-target-adc-killing-with-the-hibit-tck-platform https://edusehat.com/en/measuring-direct-bystander-and-off-target-adc-killing-with-the-hibit-tck-platform Mon, 01 Jun 2026 19:55:22 +0700 Edusehat Measuring, Direct, Bystander, and, Off-Target, ADC, Killing, with, the, HiBiT, TCK, Platform Sponsored content brought to you by

    promega logo

    The efficacy of biologic-based immunotherapies relies on their ability to induce apoptosis and cell death through the recruitment and activation of immune effector cells. Biologic-

    based immunotherapies come in a variety of formats, such as antibody drug conjugates (ADCs), chimeric antigen receptor (CAR) T cell therapy, and monoclonal antibodies (mAbs), to name a few. For payload-linked formats like ADCs, one challenge is verifying delivery of the cytotoxic payloads to only targeted cells in a heterogeneous tumor environment.

    To help address this challenge, Promega’s HiBiT Target Cell Killing (TCK) platform is a streamlined, bioluminescent cell-based system that measures cytotoxicity with specificity, simplicity, and sensitivity during therapeutic development. The platform supports all major cell-killing paradigms, including CAR-T cell-mediated killing, antibody-dependent cellular cytotoxicity (ADCC), T cell-dependent cellular cytotoxicity (TDCC), antibody-dependent cellular phagocytosis (ADCP), and ADC bystander killing.

    How HiBiT TCK works

    Target cells are engineered to express a HiBiT fusion protein that remains intracellular until cell death. Upon membrane disruption, HiBiT is released into the medium, where it binds cell-impermeable LgBiT to form functional NanoBiT® luciferase. The luminescent signal is proportional to target cell death alone, with no contribution from effector cells, making the platform ideal for co-culture experiments. The workflow requires no washing, loading, or staining steps and produces robust signal-to-noise with as few as 2,000 target cells per well.

    A growing library of thaw-and-use, functionally tested cell lines addresses blood cancer targets, including B cell lymphoma and leukemia, myeloid leukemia, and multiple myeloma (Raji, Ramos, H929), as well as solid tumor targets for ovarian carcinoma, breast adenocarcinoma, and lung carcinoma (SKOV3, SK-BR-3, OVCAR3). HiBiT-containing target cell lines can also be custom tailored.

    ADCs deliver cytotoxic payloads to antigen-expressing tumor cells, but tumors are antigenically heterogeneous. When an ADC’s payload is released inside a targeted cell, it can diffuse and kill neighboring antigen-negative cells. This bystander killing can extend therapeutic coverage across a mixed tumor, or it can damage healthy adjacent tissue. The outcome depends largely on linker-payload chemistry: Cell-permeable, cleavable payloads produce high bystander activity, while cell-impermeable payloads do not. Characterizing this activity during development is essential but difficult with conventional cytotoxicity assays, which cannot attribute cell death to specific populations in a co-culture.

    Principle of the HiBiT TCK Bioassay
    Principle of the HiBiT TCK Bioassay. Cytotoxic mAbs and/or effector cells are incubated with target cells expressing a HiBiT fusion protein. Upon killing of the target cell, the HiBiT fusion protein is released and binds extracellular LgBiT to create a functional NanoBiT® Luciferase enzyme. Luminescence is measured using a luciferase substrate and the GloMax® Discover System.

    ADC bystander testing

    The Bystander Killing Assay uses a three-cell-line design: wild-type target cells (HiBiT) to measure direct ADC cytotoxicity, wild-type target cells (“Dark”, no HiBiT) to serve as the antigen-expressing bystander driver, and antigen-KO cells (HiBiT) to specifically measure bystander killing. In the assay, ADC binds the “Dark” antigen-positive cell and the payload is internalized. If the released payload diffuses and kills neighboring antigen-KO HiBiT cells, the resulting luminescence is specific to bystander killing. Because HiBiT remains intracellular until membrane disruption, only dead target cells contribute signal. Effector or bystander driver cells do not.

    Proof of concept

    To demonstrate proof of concept, the HiBiT TCK bioassay was used on SKOV3 cells with two approved HER2-targeting ADCs, disitamab vedotin with a cleavable MMAE payload and ado-trastuzumab emtansine with a non-cleavable DM1 payload. The assay confirmed positive bystander killing with disitamab vedotin. Conversely, Kadcyla demonstrated a negative bystander killing effect. CellTiter-Glo™, which reports total well viability without distinguishing cell populations, was unable to make this distinction, demonstrating the power of the HiBiT TCK Bioassay. The same principle was validated with loncastuximab tesirine on Raji/CD19-KO co-cultures.

    The examples validate that the streamlined HiBiT TCK platform measures cytotoxicity with specificity, simplicity, and sensitivity. Since the resulting luminescent signal is specific to the engineered target cell, the bioassay is well-suited for mixed co-culture experiments during development efforts. Beyond bystander applications, the HiBiT TCK platform supports ADCC with PBMC effectors, TDCC with CD8+ T cells, and CAR-T killing assays across a 4–72 hour time course.

     

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    Learn more www.promega.com.

    The post Measuring Direct, Bystander, and Off-Target ADC Killing with the HiBiT TCK Platform appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ProPure™ Endotoxin-Free Proteins for Reliable Cancer Research https://edusehat.com/en/propure-endotoxin-free-proteins-for-reliable-cancer-research https://edusehat.com/en/propure-endotoxin-free-proteins-for-reliable-cancer-research Mon, 01 Jun 2026 19:55:21 +0700 Edusehat ProPure™, Endotoxin-Free, Proteins, for, Reliable, Cancer, Research Sponsored content brought to you by

    Sino Biological Logo

    In cancer research and therapy development, even trace levels of endotoxins (LPS) in recombinant proteins can severely distort results. In discovery and preclinical studies, endotoxins are silent disruptors of animal immunization, sensitive biological assays, and toxicity assessments, compromising results and safety evaluations. Endotoxin-free recombinant proteins are therefore essential for generating reliable research data and successful development of next-generation cancer therapeutics.

    Invisible interference in cancer therapy and vaccine development

    Endotoxin contamination can severely compromise antibody generation in animal models. Even small amounts of endotoxins can alter the host’s immune response, reducing antibody specificity, consistency, and overall quality. Endotoxin-contaminated recombinant proteins can subtly—but significantly—alter cellular behavior through immunostimulatory and cytotoxic effects. Endotoxin-induced systemic inflammation in animals can further disrupt experiments, potentially leading to study suspension or even termination.

    In cell-based studies, endotoxin contamination can be a hidden disruptor. Immune cells such as dendritic cells, macrophages, monocytes, and T cells can respond strongly even to trace amounts of endotoxins, leading to cytokine release, altered proliferation, or unexpected activation. These effects can easily produce misleading or non-reproducible results.

    The demand for endotoxin-free reagents is even more critical in the development of cancer vaccines. Since these therapies rely on precise modulation of the immune system, endotoxin contamination can trigger unintended immune activation, masking the true efficacy of the vaccine candidate and introducing safety risks. Using endotoxin-free proteins is therefore vital to accurately evaluate immunogenicity and support safe clinical translation.

    Sino Biological’s ProPure™ solution to minimize endotoxin risk

    While pharmacopeial guidelines such as USP <85> provide general limits for endotoxin, cutting-edge immunology and translational oncology studies often require far stricter control. Sino Biological’s ProPure endotoxin-free recombinant proteins are designed to eliminate this variable at the source, supporting reliable results from early discovery through IND-enabling studies. Produced at the state-of-the-art Center for Bioprocessing (C4B) in Houston, Texas, ProPure reagents are rigorously controlled to achieve levels as low as 0.05 EU/mg, with select products reaching an exceptional 0.01 EU/mg—over ten times lower than typical industry standards.

    By incorporating endotoxin-free proteins, researchers in cancer therapy and vaccine development can confidently achieve consistent and accurate results in critical applications, including:

    • Animal immunization for antibody generation—ensuring high-quality antibodies and predictable host immune responses.
    • Preclinical toxicology and pharmacokinetics (PK)—minimizing confounding immune activation in animal models.
    • In vitro cell proliferation and differentiation assays—reducing false positives caused by endotoxin-sensitive cells such as dendritic cells, macrophages, and T cells.
    • Precise detection and quantification of cytokines—supporting reliable immunological readouts and biomarker analyses.

    How ProPure achieves ultra-low endotoxin levels

    ProPure quality is not achieved by end-stage cleanup alone. C4B employs an integrated Prevention–Isolation–Detection strategy across the entire production lifecycle, ensuring that ProPure proteins arrive ready for use in the most demanding oncology and immunology applications.

    • Prevention at the source: Endotoxin-free plasmids and buffers, low endotoxin-binding plastics, and stringent clean-in-place (CIP) procedures minimize endotoxin introduction from cloning through purification.
    • Environmental isolation: The facility follows an E. coli-free principle, eliminating a major source of endotoxin introduction in recombinant protein production.
    • Dual detection: Each batch is tested using Limulus Amebocyte Lysate (LAL) and/or recombinant Factor C (rFC) assays for sensitive, redundant detection, and fully traceable batch data.
    ProPure illustration
    ProPure triple-control strategy for ultra-low endotoxin.

    With advanced technologies and rigorous quality control, Sino Biological delivers endotoxin-free proteins that meet the needs of highly sensitive research and translational applications. ProPure proteins help researchers reduce variability, improve reproducibility, and accelerate the development of next-generation cancer therapies.

     

     

    Learn more about ProPure™ endotoxin-free proteins at sinobiological.com/category/endotoxin-free-proteins.

    The post ProPure™ Endotoxin-Free Proteins for Reliable Cancer Research appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Spatial Atlasing: Why Sensitivity Is the Real Frontier https://edusehat.com/en/spatial-atlasing-why-sensitivity-is-the-real-frontier https://edusehat.com/en/spatial-atlasing-why-sensitivity-is-the-real-frontier Mon, 01 Jun 2026 19:55:20 +0700 Edusehat Spatial, Atlasing:, Why, Sensitivity, the, Real, Frontier Sponsored content brought to you by

    vizgen logo

    Cell atlasing efforts rest on a deceptively simple premise: To understand a tissue, you must find every cell in it, including the rare populations and transitional states whose biology is often the most clinically meaningful.

    This is where atlasing gets hard. Throughput is no longer the bottleneck. Sensitivity is. A platform that captures only a fraction of transcripts per cell fails to detect lower-abundance populations that define an atlas’s resolution and utility.

    A liver atlas that rewrites human zonation

    A recent Nature study by Yakubovsky and colleagues at the Weizmann Institute illustrates what sensitivity makes possible. They built a spatial atlas of the healthy human liver from live donors, avoiding the transcriptomic distortions of deceased or adjacent-normal tissue, and used the MERSCOPE® Platform with a 500-gene panel to validate cellular zonation at single-molecule resolution.

    What they found reshapes a long-standing model of liver biology. Hepatocyte functions long thought to be periportal in mammals, key urea cycle enzymes (OTC, NAGS, ASL), the gluconeogenic gene PCK2, and the master transcription factor HNF4A, are pericentrally zonated in humans. Kupffer cell localization is also inverted relative to mouse: in humans, these macrophages are enriched in the pericentral zone. None of this would have surfaced without high-sensitivity spatial transcriptomics.

    “MERSCOPE allowed us to validate zonation programs at single-molecule resolution. That sensitivity was essential to a reference atlas we could trust,” said Shalev Itzkovitz, PhD, an assistant professor at Weizmann Institute of Science and lead author on the Nature paper.

    MERFISH 2.0™:  built for the cells that might be missed

    MERFISH 2.0 offers improvements to per-cell transcript capture and signal-to-noise that expand the dynamic range over which low-abundance transcripts and rare cell types become reliably detected. Early disease states, transitional progenitors, sparse immune subsets, and niche stromal cells move firmly into the resolved fraction of the atlas.

    “When we set the design goals for MERFISH 2.0, the question we kept coming back to was: what are users still missing? Throughput wasn’t the answer, sensitivity was. Lowly expressed genes, and rare cells are where the most important biology often lives, and MERFISH 2.0 makes sure that rare events stop being the ones that get away,” said Jiang He, PhD, Co-founder and VP of Reagents, Vizgen.

    Why MERSCOPE Ultra™ Platform is the spatial atlas platform

    Besides sensitivity, atlases also require tissue areas large enough to capture biological context and analytical flexibility to interpret what is found. Four capabilities of the MERSCOPE Ultra Platform combine to produce atlas-grade data:

    Three cm² imaging area. Larger sections, multi-region samples, and cohort-scale studies without registration artifacts or sampling bias.

    MERFISH 2.0 sensitivity.  MERFISH 2.0 ensures rare populations and rare transcripts are accurately resolved.

    Tissue clearing. Many informative atlasing tissues, liver, brain, dense tumor samples, are optically challenging. Clearing reduces autofluorescence and scattering, preserving single-molecule signal across the full section thickness.

    Customizable segmentation and analysis. MERSCOPE’s pipeline lets researchers tune cell boundary detection and adapt clustering to the biology.

    Illustration of A Spatial Atlas of the Healthy Human Liver from Live Donors

    Getting to MERFISH 2.0 quickly

    MERSCOPE Pre-designed Panels with Add-on capabilities give researchers a direct path: Existing instruments remain compatible, and labs can apply the enhanced chemistry to projects already in progress. More than 20 validated Pre-designed Panels span human biology, oncology, and dedicated mouse studies.

    The atlasing moment

    The Human Cell Atlas and disease-focused atlasing efforts are moving from pilot to production scale, and the atlases built now will be cited and expanded on for years. The question is whether a platform finds the cells that matter most: the ones that change everything when you finally see them.

    “Cell atlases need more than cell-type identity. Spatial technologies like the MERSCOPE Platform are how we add location and function to that picture, and that context is what makes an atlas useful for understanding tissue biology, not just cataloguing it,” said Liat Alyagor, PhD, head of immunohistochemistry, Weizmann Institute of Science.

    That is the standard MERSCOPE Ultra was built to meet.

     

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    Learn more vizgen.com.

    The post Spatial Atlasing: Why Sensitivity Is the Real Frontier appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Soon…the First Organ-on-a-Chip Qualified Drug Development Tool https://edusehat.com/en/soonthe-first-organ-on-a-chip-qualified-drug-development-tool https://edusehat.com/en/soonthe-first-organ-on-a-chip-qualified-drug-development-tool Mon, 01 Jun 2026 19:55:19 +0700 Edusehat Soon…the, First, Organ-on-a-Chip, Qualified, Drug, Development, Tool Sponsored content brought to you by

    emulate bio logo

    Historical data indicate that animal models are not ideal for the determination of the efficacy and safety of human therapeutics. Ninety percent of drugs that pass animal studies do not receive regulatory approval. Improving predictive accuracy in preclinical tests is paramount, thus the movement toward more human-relevant models.

    The goal to reduce the use of animals in preclinical testing changes testing paradigms. In April 2025, the U.S. FDA’s Roadmap to Reducing Animal Testing in Preclinical Safety Studies outlined a strategic, stepwise approach to replace animal testing with scientifically validated new approach methodologies (NAMs), such as organ-on-a-chip systems, computational modeling, and advanced in vitro assays. FDA Modernization Acts 2.0 and 3.0 facilitated this activity by empowering the agency to accept NAMs in lieu of animal studies.

    Meanwhile, legislation from the EU, Directive 2010/63/EU, requires marketing authorization holders to integrate the 3Rs (Reduction, Refinement, and Replacement) and welfare standards for the treatment of animals in all aspects of the development, manufacture, and testing of medicines. In addition, last year, the U.K. delivered an expedited phase-out plan for animal use.

    But it all began in 2020 with the launch of the FDA Innovative Science and Technology Approaches for New Drugs (ISTAND) pilot program to provide a pathway to qualify novel drug development tools (DDTs) that did not fit within the agency’s existing qualification programs. Qualified DDTs are defined as having a proven, specific use and can be incorporated in  any drug development program for a particular context of use.

    The pilot has advanced to a permanent DDT qualification program. To date, ISTAND has accepted eight submissions–two tools that assess preclinical safety without using animals, two methods involving tissues, and one statistical approach.

    The rigorous ISTAND process

    In a 2022 Communications Medicine study to test drug-induced liver injury (DILI), 870 human Emulate Liver-Chips created with cells from three different human donors were challenged with 27 different drugs. The human Liver-Chip predicted human DILI with 87% sensitivity and 100% specificity, ~7 to 8 times more accurate than the comparable animal models.1 These results prompted Emulate to submit a Letter of Intent (LOI) to ISTAND in 2024.

    ISTAND accepted Emulate’s LOI for the first organ-on-a-chip DDT to predict DILI. The human Liver-Chip S1 was proposed to assess the risk of small molecule candidate drugs inducing DILI in adults to create human-relevant data for candidate drug IND submission.

    The LOI acceptance was the entry point in a three-step rigorous qualification process. ISTAND required Emulate to qualify the in vivo-like physiological functionality of the Liver-Chip S1, and quantify its ability to predict DILI risk through changes in tissue morphology as well as alterations in albumin and alanine transaminase (ALT) protein concentrations when the chips were challenged with toxic drugs administered across eight concentrations.

    Now, the Emulate Liver-Chip S1 is in the final stages of qualification. Two independent commercial users need to successfully produce similar results. Pending successful completion, the Liver-Chip will be the first FDA-approved DDT to assess the potential of a small-molecule candidate drug to cause DILI when a prior structurally similar small-molecule has shown DILI in the clinic.

    High-throughput capabilities

    Moving toward reduction and, in some cases, replacement of animal models demands both biological fidelity and throughput. For model development and target validation, the Zoë-CM2® Culture Module automates the precise condition needed to culture up to 12 chips.

    For high-throughput options, the AVA™ Emulation System is a self-contained Organ-on-a-Chip workstation that fuses high-throughput microfluidic tissue culture, full environmental control, and real-time imaging into a single, compact benchtop unit. The Chip-Array™

    consumable integrates 12 independent Organ-Chips into an SBS format for 96-well streamlined workflows with multichannel pipettes and automated liquid handlers.

     

    Reference

    1. Ewart, L., Apostolou, A., Briggs, S.A. et al. Performance assessment and economic analysis of a human Liver-Chip for predictive toxicology. Commun Med 2, 154 (2022). doi.org/10.1038/s43856-022-00209-1.

     

    GEN June 2026 Emulate QR Code

     

    Click Here to learn more about Emulate’s product portfolio emulatebio.com/resources/emulate-product-brochure.

     

    The post Soon…the First Organ-on-a-Chip Qualified Drug Development Tool appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Evaluating CNS Anti-inflammatory Therapies with Human Brain Organoids https://edusehat.com/en/evaluating-cns-anti-inflammatory-therapies-with-human-brain-organoids https://edusehat.com/en/evaluating-cns-anti-inflammatory-therapies-with-human-brain-organoids Mon, 01 Jun 2026 19:55:18 +0700 Edusehat Evaluating, CNS, Anti-inflammatory, Therapies, with, Human, Brain, Organoids Sponsored content brought to you by

    28bio logo

    Inflammatory pathways involving microglia, astrocytes, and cytokine signaling are widely implicated in disorders including Alzheimer’s disease, Parkinson’s disease, ALS, multiple sclerosis, and traumatic brain injury. Yet despite significant investment in anti-inflammatory therapies, clinical success has remained limited.

    A primary reason is that conventional preclinical models do not fully capture the complexity of human neuroimmune biology. Many therapies show encouraging results in animal studies but fail to reproduce those effects in human clinical trials.

    These limitations have become increasingly problematic as evidence linking neuroinflammation to disease progression continues to grow. Genome-wide association studies have identified immune-related genes associated with Alzheimer’s disease risk, while imaging and postmortem analyses have demonstrated close relationships between inflammatory activation, synaptic loss, and cognitive decline. Drug developers are therefore pursuing therapies directed at neuroimmune biology using models that lack functional human neuroimmune architecture.

    CNS-3D Inflammatory Organoids, recently introduced by 28bio, is an assay-ready immunocompetent 3D brain organoid model incorporating neurons, astrocytes, and microglia to evaluate efficacy of anti-inflammatory drugs by quantifying their ability to reduce inflammatory injury, preserve tissue health, and restore neuronal network activity.

    The inclusion of microglia is particularly important because it enables researchers to study inflammatory signaling within a more physiologically relevant cellular environment. Rather than measuring isolated cytokine responses in monoculture, researchers can examine how inflammatory activation propagates across interconnected neural and glial populations and how those changes affect tissue integrity and network behavior.

    Data presented recently at the Microphysiological Systems World Summit demonstrated distinct cellular responses following exposure to inflammatory stimuli (Fig. 1) including lipopolysaccharide (LPS) and TNF-α. According to the findings, LPS exposure generated a predominantly microglial inflammatory response, while TNF-α produced stronger astrocytic activation patterns. Cytokine profiling also demonstrated measurable increases in inflammatory mediators following stimulation.

    Differential microglial and astrocytic responses to inflammatory insults in CNS-3D Inflammatory Organoids.
    Figure 1. Differential microglial and astrocytic responses to inflammatory insults in CNS-3D Inflammatory Organoids. CNS-3D Inflammatory Organoids were treated with vehicle, LPS, or TNF-α and assessed by immunofluorescence staining for Iba1-positive microglia and GFAP-positive astrocytes. LPS induced a pronounced microglial response, whereas TNF-α preferentially increased astrocytic activation, highlighting stimulus-specific inflammatory phenotypes within the 3D CNS organoid model.

    These findings are highly relevant for therapeutic development because neuroinflammation is not a single biological process. Disease states may involve different combinations of microglial activation, astrocytic dysfunction, oxidative stress, and neuronal injury. Models capable of distinguishing between these responses provide a more predictive framework for evaluating therapeutic candidates.

    CNS-3D Inflammatory Organoids also support integration of functional calcium imaging with cytokine analysis, immunostaining, cytotoxicity assays, and molecular profiling. This approach addresses another persistent challenge in CNS drug development: many inflammatory assays quantify molecular markers without determining whether those changes correspond to preservation or disruption of neuronal function.

    As neurodegenerative drug discovery continues to confront translational issues, interest is growing in models capable of reproducing human-specific cellular interactions and functional neuroimmune responses. Human brain organoid models help bridge the gap between preclinical findings and clinical outcomes by providing a more physiologically relevant framework for evaluating anti-inflammatory therapeutics in the CNS.

     

    June 2026 28bio QR Code

     

    To learn more about CNS-3D Inflammatory Organoids, please visit 28bio.com.

     

    The post Evaluating CNS Anti-inflammatory Therapies with Human Brain Organoids appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Corning Advances the Organoid Revolution https://edusehat.com/en/corning-advances-the-organoid-revolution https://edusehat.com/en/corning-advances-the-organoid-revolution Mon, 01 Jun 2026 19:55:17 +0700 Edusehat Corning, Advances, the, Organoid, Revolution Sponsored content brought to you by

    Corning logo

    The rapid rise of new approach methodologies (NAMs) is reshaping drug development, and organoids are emerging as one of the field’s most promising technologies. With the FDA Modernization Act 2.0 removing the long-standing requirement for animal testing in many drug-development pathways, researchers and industry leaders are increasingly looking toward human-relevant systems that better predict clinical outcomes. Against this backdrop, Corning Life Sciences is positioning itself as a key enabler of the organoid revolution by helping scientists overcome persistent barriers related to complexity, reproducibility, and throughput.

    “Corning is helping to overcome challenges to adopting NAMs such as organoid models by providing specialized consumables and reagents that are essential to generating more in vivo-like models,” said Hilary Sherman, senior applications scientist at Corning Life Sciences. Sherman pointed to products including “Corning Matrigel Matrix, Transwell Permeable supports, and a wide variety of specialized plasticware for spheroid and organoid culture” as foundational technologies supporting the transition toward more predictive biological systems.

    The push toward NAMs adoption gained further momentum this year when the FDA released draft guidance on alternatives to animal testing in drug development. The agency emphasized that NAMs—including organoids, spheroids, organ-on-chip platforms, and computational models—can improve predictivity while reducing reliance on animal studies.

    Those priorities align closely with challenges the organoid field has wrestled with for years. During the GEN virtual event Spotlight on Organoids, Hans Clevers, MD, PhD, an organoid pioneer and distinguished professor at the Hubrecht Institute, stressed that standardization remains one of the field’s biggest hurdles.

    “We don’t even have a good definition of what an organoid is,” Clevers said during the GEN virtual event. “When is an organoid an organoid?” He added that “nothing is standardized and nothing is automated,” underscoring the need for scalable workflows that can transition organoid science from exploratory academic research into robust industrial platforms.

    Clevers nevertheless remains optimistic about the technology’s transformative potential. “The most important part is we can now grow structures that really represent a small part of the human body,” he said. “Animals are complete organisms, but they’re not humans.” According to Clevers, many diseases—particularly chronic human diseases—are poorly modeled in animals, limiting translational success in drug development.

    Corning sees education and workflow optimization as crucial to solving those problems. “Corning feels very strongly about supporting our customers by providing resources to educate scientists on how to create more in vivo-like models that are reproducible,” Sherman explained. “We do this through publishing novel applications, protocols, and webinars.”

    The company is also helping researchers streamline increasingly sophisticated organoid workflows. “We have several protocols and optimization guides that educate customers on how to culture organoids to ensure they are set up for success,” Sherman noted. “Additionally, we have many application notes demonstrating different ways of automating organoid assays to give researchers a starting point for their own work.”

    Automation and scalability are becoming especially important as organoids move deeper into pharmaceutical pipelines. At the GEN virtual event, Maya Gosztyla, PhD, co-founder and CSO of BrainStorm Therapeutics, described how her company’s platform as “a very high throughput and very scalable and reproducible version of brain organoids.”

    Her company is studying CDKL5 deficiency disorder, a rare genetic epilepsy. “The whole reason that we’re doing this work in brain organoids is that the mouse models of CDKL5 don’t recapitulate the symptoms of the disease,” Gosztyla explained.

    She believes regulatory changes are accelerating industry confidence in organoid-based drug discovery. “These regulatory shifts have basically allowed drug-discovery companies to show efficacy using an alternative like a brain-organoid model,” Gosztyla said, adding that such systems are “a lot more translational compared to something like a mouse.”

    For Corning, helping researchers achieve that translational promise means supporting every stage of organoid adoption—from foundational reagents to reproducible protocols and scalable automation strategies. As NAMs continue gaining regulatory and commercial traction, the ability to standardize organoid workflows might ultimately determine how quickly these human-centric systems become mainstream tools in drug discovery and development.

     

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    Learn more www.corning.com.

    The post Corning Advances the Organoid Revolution appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Illuminating the Drug Development Path with Cell-Based Reporter Assays https://edusehat.com/en/illuminating-the-drug-development-path-with-cell-based-reporter-assays https://edusehat.com/en/illuminating-the-drug-development-path-with-cell-based-reporter-assays Mon, 01 Jun 2026 19:55:16 +0700 Edusehat Illuminating, the, Drug, Development, Path, with, Cell-Based, Reporter, Assays Sponsored content brought to you by

    BPS Bioscience logo

    Selecting and advancing drug candidates through discovery and development is a long, resource-intensive process. Demonstrating efficacy, mechanism of action (MOA), and product quality requires robust functional data.

    In early discovery, researchers use high-throughput screening (HTS) to identify active compounds in a biologically relevant context. During lead characterization and validation, these assays generate reproducible, quantitative data to confirm activity and support candidate selection. In later stages, cell based assays are commonly used as potency assays to ensure reliability, consistency, and lot-to-lot comparability of biologics, supporting regulatory compliance.

    BPS Bioscience maintains upstream licensing agreements for its cell lines, enabling clients to operate within established regulatory frameworks. This approach mitigates downstream risks associated with third-party restrictions and supports a smoother transition from research to clinical and commercial use.

    Scientific rationale for using cell-based assays in biologics development

    Unlike biochemical assays, cell-based assays capture key parameters such as membrane permeability, receptor engagement, and downstream signaling in intact cells, providing a more accurate representation of biological activity. Genetically engineered cell lines include overexpression and knockout models used to validate therapeutic targets and assess compound activity. Inducible reporter assays are particularly valuable for studying signaling pathways. Luciferase reporters, placed under the control of pathway-specific response elements, enable sensitive, quantitative, and reproducible measurement of pathway activation.

    Reporter systems are broadly applicable across diverse cell types and signaling pathways, supporting HTS as well as more complex applications such as research in metabolism/obesity and immunotherapy, chimeric antigen receptor and T-cell receptor functional evaluation, antibody-dependent cellular cytotoxicity assays, and other co-culture models. Many biologics, including cytokine-targeting antibodies, peptides, and mimetics, are defined by their effects on specific signaling pathways. For example, GLP-1 receptor agonists activate cAMP-dependent signaling cascades, while anti-TL1A antibodies inhibit TL1A-mediated immune signaling. Accurately measuring these pathway-specific responses is essential for candidate selection and mechanistic validation.

    Activation of receptor signaling upon ligand binding triggers luciferase expression. The potency of a candidate drug can be assessed by simply measuring luciferase activity. [This illustration was created using BioRender.com]

    Applications

    Reporter cell lines enable a wide range of applications:

    1. Discovery and screening

      • Identify agonists or antagonists of specific signaling pathways

      • Screen compound libraries for selective modulators

    2. Mechanistic studies

      • Characterize MOA

      • Analyze pathway function and regulation

    3. Functional assays

      • Perform co-culture cytotoxicity assays to evaluate immune effector function

      • Support immunotherapy development and cell-based therapeutic evaluation

    BPS Bioscience reporter cell portfolio

    BPS Bioscience offers a comprehensive portfolio of pathway-specific reporter cell lines designed to support biologics development across multiple therapeutic areas. Reporter cell lines include IL-2, IL-6, and IL-15-responsive reporter cells, GLP-1-responsive models for metabolic research, and TL1A-responsive Jurkat cells.

    Luciferase-based reporter systems provide rapid, sensitive, and quantitative detection of cellular responses, enabling efficient compound screening, pathway analysis, and target validation. Supporting reagents, including optimized culture media and the One-Step™ Luciferase Assay System, further streamline experimental workflows and improve reproducibility.

    Advantages of luciferase reporter cell systems

    • Quantitative readouts enable precise measurement of pathway activity
    • High sensitivity allows detection of subtle biological effects
    • Low background and high signal-to-noise ratio ensure robust data
    • Compatibility with high-throughput formats supports large-scale screening

    Advantages of BPS Bioscience reporter cell lines

    • Optimized protocols and media simplify assay implementation
    • Human cell backgrounds improve physiological relevance (with select alternative models available)
    • Cost-effective workflows with minimal reagent requirements
    • Extensive validation, with data often benchmarked against clinically relevant compounds
    • Clonal cell lines ensure consistency and reduce variability over time

    Together, cell based reporter assays and their supporting tools enable efficient, pathway

    relevant evaluation of biologics from discovery through late stage development.

     

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    Learn more bpsbioscience.com.

     

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    ]]>     One Antibody, Fewer Scientific Surprises https://edusehat.com/en/one-antibody-fewer-scientific-surprises https://edusehat.com/en/one-antibody-fewer-scientific-surprises Mon, 01 Jun 2026 19:55:15 +0700 Edusehat One, Antibody, Fewer, Scientific, Surprises Sponsored content brought to you by

    bio x cell logo

    In biomedical research, promising programs rarely collapse for lack of scientific ambition. More often, they collapse under the weight of inconsistency. One assay produces compelling results, the next model delivers confusion, and suddenly, researchers are left wondering whether the biology changed or whether the tools did.

    That uncertainty sits at the heart of translational continuity, a concept gaining increased attention as drug-discovery pipelines become more complex and expensive. According to Cody Spencer, PhD, Director of Scientific Affairs at Bio X Cell, maintaining continuity across experimental systems is less about rigidly replicating conditions and more about reducing unnecessary variability.

    “I define translational continuity as the ability to study the same underlying biology as you move from early discovery into more complex preclinical models without introducing unnecessary variability,” Spencer explains.

    In practice, translational continuity means researchers can move from in vitro assays to organoids to in vivo mouse models while remaining confident that their findings reflect real biological phenomena, not artifacts created by inconsistent reagents or shifting methodologies. That distinction matters more than many researchers realize.

    The greatest threat to continuity, Spencer argues, is often surprisingly mundane: switching antibodies or suppliers midway through a research program. Even antibodies marketed against the same target protein can behave differently depending on clone selection, sequence, production methods, formulation, or purification standards. When an antibody’s functional profile—whether blocking, agonistic, or depleting—is well characterized, researchers can select tools aligned with their experimental goals from the start, reducing the need to switch reagents mid-program. “When you switch suppliers, you’re often introducing a new variable without fully realizing it,” Spencer says.

    Those differences might seem subtle initially, but they can snowball dramatically in translational studies. Inconsistent potency, altered dose responses, or unintended immune engagement can suddenly emerge even when earlier experiments appeared rock solid. Researchers then face a dangerous interpretive trap: Are they observing a genuine biological effect or merely the consequences of a reagent change? “That’s where you start to see promising early data that doesn’t hold up in more complex models,” Spencer notes.

    The consequences extend beyond scientific frustration. Failed translation burns time, funding, and institutional confidence. Entire programs can stall while teams attempt to reconcile conflicting datasets generated by technically different reagents presenting as equivalent tools. For companies operating in high-stakes therapeutic areas like immuno-oncology, autoimmune disease, and inflammatory disorders, that level of ambiguity can become extraordinarily expensive.

    The formulation of antibodies also plays a surprisingly large role in reproducibility, particularly in vivo. Preservatives, endotoxin contamination, and formulation inconsistencies can introduce unintended biological effects that distort experimental outcomes. “For in vivo studies, antibodies need to have ultra-low endotoxin levels and be free of preservatives to avoid introducing unintended biological effects,” Spencer explains.

    This emphasis on reproducibility has reinforced the case for recombinant antibodies, which are derived from defined sequences rather than traditional hybridoma methods. Recombinant production offers stronger lot-to-lot consistency and allows researchers to better control host species, isotype selection, and Fc functionality.

    That predictability becomes even more critical as antibody engineering grows more sophisticated. Bispecific antibodies, for example, can engage two targets simultaneously, enabling researchers to model increasingly complex biological interactions. But those advanced formats also amplify the risks associated with inconsistency. “Small changes can significantly impact activity,” Spencer warns.

    Ultimately, translational continuity is about preserving confidence. In an era where reproducibility concerns continue to challenge biomedical science, researchers are increasingly recognizing that experimental reliability depends not only on biological insight but also on the consistency of the tools used to generate it. “When translational continuity is strong, the data become much easier to interpret,” Spencer says. “If the biology is real, it should carry across systems.”

     

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    Learn more bioxcell.com.

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    ]]>     StockWatch: Agilent Shares Jump on Better than Expected Quarterly Results https://edusehat.com/en/stockwatch-agilent-shares-jump-on-better-than-expected-quarterly-results https://edusehat.com/en/stockwatch-agilent-shares-jump-on-better-than-expected-quarterly-results Mon, 01 Jun 2026 09:00:10 +0700 Edusehat StockWatch:, Agilent, Shares, Jump, Better, than, Expected, Quarterly, Results A strong quarterly earnings report that beat analyst expectations, growth that extended into core pharma and biotech tools, plus improved investor guidance on revenue, operating margin, and earnings per share (EPS) were enough to send shares of Agilent Technologies (NYSE: A) jumping to their best one-day increase in nearly 24 years this past week.

    Agilent shares surged 17% from $115.79 to $135.42 Thursday, the first trading day after the tools giant announced better-than-expected results for the second quarter of its 2026 fiscal year ending April 30. Agilent operates on a fiscal year that ends October 31. That was the best one-day performance since November 19, 2002, when the stock ballooned 16% to $134.50.

    Shares inched up another 0.12% Friday, closing the week at $135.54—giving Agilent an 18% gain for the week and seven straight positive trading days.

    Agilent finished Q2 of FY’26 with net income of $339 million or $1.20 a share, up 58% from $215 million or 75 cents a share a year earlier, on revenue that rose 10% to $1.835 billion from $1.668 billion in the year-ago quarter, the company announced after the close of trading on Wednesday.

    Agilent’s “core” growth—which excludes the impact of currency and acquisitions and divestitures within the past 12 months—was 6.3%, exceeding the 4.8% growth forecasted by a consensus of Wall Street analysts cited by the company, as well as its earlier investor guidance of growth ranging from 4% to 5.5%. The consensus also predicted Q2 revenue of $1.79 billion, according to FactSet data cited by Barron’s.

    Agilent moved quickly to raise the low end of its FY 2026 revenue guidance, from $7.3 billion to $7.39 billion, while trimming the upper end to $7.49 billion from $7.5 billion. The latest guidance remains above the initial FY 2026 forecast of between $7.3 billion and $7.4 billion.

    Agilent also raised its guidance on operating margin from 75 to 85 basis points, and on FY 2026 non-GAAP EPS, which is now projected to range from an even $6 to $6.10, an increase of eight cents at the midpoint from the previous range of $5.90 to $6.04, which was raised in February from the initial range of $5.86 to $6. The analyst consensus predicts a lower EPS of $5.97.

    “Strong follow-up quarter”

    “All in, F2Q was a strong follow-up quarter to the weather-related headwinds seen in F1Q and, looking ahead, we continue to see Agilent as a clean tools story to own with upside from reshoring, CDMO [contract development and manufacturing organization] capacity ramping and AI [artificial intelligence] investments in pharma over the long-term,” Casey Woodring, vice president, equity research with J.P. Morgan, and three colleagues wrote in a research note.

    In a separate report also issued by J.P. Morgan, Woodring and colleagues concluded that AI-driven shortening of timelines for target identification and hypothesis generation “could increase subsequent wet lab validation volume, benefiting high-throughput instruments and multi-omics tool providers, while potentially pressuring lower-throughput/animal-model-adjacent tools.”

    Agilent is one of seven biopharma tools developers likely to benefit as a result, the J.P. Morgan analysts wrote. The firm retains an “Overweight” rating and $180 price target on Agilent.

    Agilent president and CEO Padraig McDonnell told analysts on the company’s May 27 earnings call that pharma customers are leaning into AI to accelerate drug development and reduce the odds of costly late-stage failures.

    “There is a growing need for large-scale multi-modal datasets to train AI models, which will require significant investments in the wet lab,” McDonnell said. “By moving the needle on drug development ROI, AI holds the promise of putting our largest customer constituency on a better footing. A higher number of approvals coming through the drug pipeline should be a strong tailwind for us, given our leading position in downstream manufacturing QA/QC workflows.

    “Attractive setup”

    Puneet Souda, senior managing director, life science tools and diagnostics and a senior research analyst with Leerink Partners, added that given Agilent’s guidance mostly rose by the amount results beat consensus forecasts, “we continue to see an attractive setup for A [Agilent]” and likes the company’s position across multiple end-markets—citing chemicals and applied markets (C&AM), QA/QC instrumentation, and biopharma.

    Souda maintained Leerink’s “Outperform” rating on Agilent shares and raised the firm’s 12-month price target by 3%, to $170 from $165, based on the improved guidance.

    Michael Ryskin, research analyst with BofA Securities, showed even more enthusiasm about Agilent’s prospects, upgrading his firm’s rating on the company’s shares from “Neutral” to “Buy”—but trimming by 3% its price target from $150 to $145, a multiple reflecting a lower estimate of 18 times Agilent’s FY 2027 earnings before interest, taxes, depreciation, and amortization (EBITDA), from 19 times EBITDA.

    “Key growth drivers such as LC and GC replacement cycles continue to deliver (and remain in early innings), execution is solid across the board, and there were few new negative surprises to derail the momentum (despite widespread fears of chemicals slowdown),” Ryskin wrote. “Combined with a very reasonable valuation, and a myriad of issues elsewhere in tools, we see Agilent as an increasingly attractive asset.”

    So too did analysts from four other investment firms, which responded to Agilent’s results by raising their price targets on Agilent stock:

    • TD Cowen (Dan Brennan)—Up 5% from $147 to $155, maintaining “Buy” rating.
    • Barclays (Luke Sergott)—Up 3.6% from $140 to $145, maintaining “Overweight” rating.
    • RBC Capital (Dan Leonard)—Up 1.3%, from $153 to $155, maintaining “Outperform” rating.
    • Baird (Catherine Ramsey Schulte)—Up 1.3% from $156 to $158, maintaining “Outperform” rating. Schulte raised the target price 0.6% from $155 just on Tuesday.

    But Brandon Couillard, managing director, Life Science Tools & Diagnostics and an equity research analyst at Wells Fargo, lowered his firm’s price target on Agilent’s shares 3%, to $160 from $165, reasoning that the lower price would yield a more realistic 18.71% difference between the stock’s current price and what the firm predicts it is worth. Couillard maintained Wells Fargo’s “Overweight.”

    Instruments grew by high single digits during Agilent’s fiscal second quarter, driven by continued positive low double-digit revenue increases in liquid chromatography (LC), LC/mass spectrometry (MS), and gas chromatography (GC) instrument sales.

    “Best market share data I’ve seen”

    “I think we’ll continue to see strong momentum on the LC-MS and GC,” McDonnell told analysts. “We expect the LC replacement cycle to be a 200–300 bps [basis points, or 2–3%] tailwind to the LC growth. We’ve seen that normal trajectory of the replacement cycle, but continued momentum and funnels look really strong. I will say it was actually the best market share data I’ve seen.”

    “Not only are we replacing, but we’re also taking share in competitive accounts, which again, continues the momentum,” he added.

    McDonnell said the instrument sales surge was driven by three compelling reasons: Past underinvestment in replacing aging tools by biopharma customers; favorable capital expenditure or “CapEx” conditions in the United States and Europe, “and, of course, customer-focused innovations.”

    “We expect that to continue, and we’re seeing it across all markets,” McDonnell added.

    During the second fiscal quarter, Agilent said, its core pharma and biotech business rose 6%. Tycho Peterson, equity analyst with Jefferies, noted that Agilent’s biopharma results marked its fifth straight quarter of mid-single-digit growth, led by factors that, according to the company, include:

    • Replacement by customers of Agilent LC tools, which sent revenue in that segment up by low double digits
    • A third straight quarter of growth from biotech customers
    • A 20% jump year-to-date in tool purchases driven by the development of glucagon-like peptide 1 (GLP-1) receptor agonist drugs for obesity and diabetes indications, though down from the 50% leap seen in Agilent’s first fiscal quarter of this year
    • Low single-digit growth among small molecule drug developers

    “Large cap” biotechs with a market capitalization (share price times the number of outstanding shares) of $10 billion or more accounted for a low double-digit increase in sales that outpaced sales from small- and mid-cap biotech—though positive demand signals are emerging, tied to funding. And while China sales fell by high single digits overall, Agilent generated revenue that grew in the high teens year-over-year from biotech companies there, Peterson reported.

    Consumables “momentum”

    Biopharma activity is recorded within Agilent’s Life Sciences and Diagnostics Group (LDG) segment, which year-over-year rose 12% (9% core) to $732 million during the quarter. Among the company’s two other reporting segments, the Applied Markets Group (AMG) saw growth of 14% (11% core) to $344 million, while the Agilent CrossLab Group (ACG) grew 6% (2% core) to $759 million.

    LDG includes LC-MS instrument platforms, cell and biomolecular analysis, specialized CDMO services, pathology, companion diagnostics, and genomics. AMG includes GC-MS, spectroscopy, and vacuum technology platforms. ACG supports customers in all end markets through services, software and informatics, automation, and consumables.

    In consumables, Agilent said sales of its Altura Ultra Inert HPLC [high-performance LC] columns grew more than 50% quarter-over-quarter, reaching 75% of the top 20 biopharma accounts.

    “This rapid adoption reinforces the strength of the innovation engine and the unified commercial organization. We will continue to build on that strong initial momentum with additional waves of column launches,” McDonnell vowed.

    Another expected source of biopharma growth in the coming months, Peterson of Jefferies observed, is orders for new tools from drug and tools developers reshoring their operations in the United States. Re-shoring orders are expected by fiscal year-end 2026, with revenue contributions beginning in the 2027 fiscal year that begins on November 1 of this year.

    Biopharma is part of Agilent’s Life Sciences and Diagnostics Markets segment, which consists of seven areas of activity: Providing active pharmaceutical ingredients for oligo-based therapeutics, as well as solutions that include reagents, instruments, software, and consumables, which enable customers in the clinical and life sciences research areas to interrogate samples at the cellular and molecular level.

    “You look at the long-term drivers in pharma, you see redistribution of supply chains, expansion of biologics, and, of course, you see many other factors really helping. What I would say is that we’re very much downstream in QA/QC,” McDonnell said during the earnings call. “We’re in development as well. We’re right in that sweet spot for reshoring, replacement cycle, and any capacity or supply chain resilience around, feel really good about that.”

    Leaders and laggards

    • Q32 Bio (NASDAQ: QTTB) shares zoomed 81% from $7.09 to $12.85 Wednesday after the developer of therapies for alopecia areata and other autoimmune and inflammatory diseases entered into an approximately $55 million private placement of common stock and pre-funded warrants with “new and existing institutional and accredited investors.” Q32 agreed to issue and sell 6.725 million shares of its stock at $8 per share, plus pre-funded warrants to buy 150,000 shares at $7.9999. Q32 finished the first quarter with $50.8 million in cash and cash equivalents—enough to fund operations into the first half of 2028 when combined with guaranteed near-term milestone payments from selling Phase II complement inhibitor ADX-097 to Akebia Therapeutics, plus proceeds from an at-the-market stock offering received after Q1. BVF Partners led the financing with participation from RA Capital Management, OrbiMed, and Atlas Venture. Morgan Stanley acted as lead placement agent, and Oppenheimer & Co. acted as a placement agent.
    • Replimune Group (NASDAQ: REPL) shares rocketed 86% from $4.68 to $8.69 Friday after the developer of oncolytic immunotherapies agreed with the FDA on a path toward resubmission and reconsideration of the Biologics License Application (BLA) for its lead product candidate RP1 (vusolimogene oderparepvec) in combination with nivolumab to treat advanced melanoma. Repligen said it agreed to resubmit its BLA for RP1 “in the coming days,” while the FDA agreed to treat the BLA resubmission as an urgent matter and prioritize its review “in recognition of the significant unmet need for patients in the advanced melanoma community.” In April, the FDA stunned Repligen by rejecting its BLA for a second time, issuing a complete response letter (CRL) contending that data were not sufficient to allow for RP1 approval—an assertion Replimune vehemently rejects. The BLA is supported by data from the Phase II IGNYTE trial (NCT03767348)—a 34% response rate with a median duration of 24.8 months and a favorable safety profile.

    The post StockWatch: Agilent Shares Jump on Better than Expected Quarterly Results appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Sea Cucumber Tissues Demonstrate Natural Immortality in Seawater https://edusehat.com/en/sea-cucumber-tissues-demonstrate-natural-immortality-in-seawater https://edusehat.com/en/sea-cucumber-tissues-demonstrate-natural-immortality-in-seawater Mon, 01 Jun 2026 01:45:18 +0700 Edusehat Sea, Cucumber, Tissues, Demonstrate, Natural, Immortality, Seawater From the revived corpse of Frankenstein’s monster to the disembodied hand, “Thing,” in the Addams Family, reanimated tissue is one of the most enduring images in science fiction. The discovery of a sea floor-dwelling sea cucumber that scientists are calling a “real-life zombie” suggests that there may be some basis for that image in nature.

    Scientists headed by a team at Memorial University of Newfoundland showed the continued viability of amputated tissue from the sea cucumber Psolus fabricii for more than three years in natural seawater. It’s the first known report of the long-term survival—and continued growth—of discarded tissue outside of a highly controlled, sterilized environment.

    The discovery that these living P. fabricii explants (LiPfe) can survive for years in natural seawater without any supplementation challenges assumptions of what’s possible for tissue immortality and could have implications in areas including regenerative biology and tissue engineering. The findings could also lead to the development of experimental models for biological research that are more widely accessible, without the ethical and logistical challenges associated with many existing cell lines.

    “We haven’t grown a new, complete sea cucumber yet, but we are seeing pretty stunning growth and diversification of cells literally years after this tissue was removed,” said research lead Rachel Sipler, PhD, a Bigelow Laboratory for Ocean Sciences senior research scientist. “It’s like a lizard that loses its tail. We know some lizards can grow new tails; we’re talking about whether the tail can grow a new lizard.”

    Reporting on their findings in Science Advances (“Natural tissue immortality: Indefinite survival of sea cucumber explants,”) Sipler and colleagues stated, “Our findings challenge conventional perceptions of tissue immortality and present a new class of experimental model, free from ethical concerns, with substantial implications for regenerative biology, biomedical research, and tissue engineering.”

    Over the last 200 years, scientists have tried to achieve cellular and tissular survival outside living hosts, “… but efforts have been met with limited success due to the highly degradable nature of tissue itself,” the authors wrote. Since the mid-20th century, scientists have made significant breakthroughs with immortal cell lines, such as HeLa cells, that can be grown in a lab and proliferate indefinitely for long-term research. In earlier studies, tissue cultures have only been maintained under axenic conditions that are tightly controlled, rigorously maintained, and lack any bacteria or other organisms. Even then, they have not demonstrated signs of actual healing and growth, nor retained the ability to move independently. “While immortal cell lines demonstrate indefinite proliferation in vitro, they lack structural integrity and complex tissue interactions,” the team continued. “Achieving this with complex, structured tissue represents the next step.”

    Many echinoderms, including sea cucumbers, are known to display impressive regeneration capacity and negligible cell aging. “In the ongoing effort to understand tissue culture, regeneration, and immortality, researchers have naturally been drawn to echinoderms, a phylum with genetic and evolutionary links to vertebrates and examples of both extreme regenerative capacity and negligible cellular senescence,” the investigators noted. Lost tissue, though, was always assumed to eventually decay or die.

    Yet, in what Sipler calls a product of “keen observation,” the researchers noticed that some discarded tissue from a tube foot of a sea cucumber hadn’t decayed after a number of weeks. In fact, it seemed to be growing. The researchers then ran a number of experiments in flowing seawater with tissue removed from the feet, main body, and tentacles of three individuals of P. fabricii, a cold-water species of sea cucumber.

    They found evidence of diversifying cells, immune activity, and tissue reorganization in the explanted tissue. “In experimental trials, these explants, termed LiPfe (living immortal P. fabricii explants), displayed immune activity, cell cycling, tissue reorganization, and absorption of dissolved amino acids, underscoring their active living state,” they noted. And in the absence of a mouth, the cells appeared to be getting nutrients by absorbing amino acids dissolved in the seawater.

    Even after three years, when the researchers stopped the experiments in order to publish, the tissue was still active. This ability to survive in a complex, stressful environment, Sipler said, makes this cell line unique compared to other tissue cultures. “Compared to other cells or tissues grown under laboratory setups that required strict parameters, including axenic conditions, LiPfe required nothing apart from natural running seawater,” they wrote. “Comparative experiments conducted on explanted tissues from related species demonstrated no equivalent tissue survival, highlighting the unique properties of P. fabricii, which do not have parallels in the current literature.”

    “Natural seawater is just about the most microbially diverse, least clean approach we could take experimentally,” Sipler added. “Yet, that rich environment full of bacteria and all this organic matter was actually feeding them and allowing this tissue to heal and grow.”

    The implications for biomedical sciences and engineering, the authors said, are profound, with potential applications in everything from tissue regrowth to anti-microbial healing. In their paper, the authors stated, “The discovery of LiPfe challenges the boundary between organismal life and cellular autonomy, compelling a redefinition of what it means for tissue to be alive.”

    The discovery opens up new opportunities for biological research and education more broadly. The tissue they’ve preserved not only shows an unprecedented ability to maintain its structural integrity and complexity in culture. It can also be grown more easily in the lab and, as an invertebrate, isn’t subject to as many research restrictions, making it useful in contexts where there are legal obstacles or limited biosafety infrastructure for using human-based or other vertebrate cell lines.

    As an oceanographer, Sipler noted that the exciting discovery drives home the incredible untapped potential of ocean life. “The best advances in science are made when you find a natural analog for what you’re studying,” she said. “Here is this species that has this groundbreaking ability, and we had no idea. It’s a reminder of how much is yet to be discovered in the marine environment, and how important it is to protect these resources that may hold really valuable knowledge for us.”

    The post Sea Cucumber Tissues Demonstrate Natural Immortality in Seawater appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Targeting Metabolic Mechanism Restores Chemotherapy Sensitivity in Ovarian Cancer https://edusehat.com/en/targeting-metabolic-mechanism-restores-chemotherapy-sensitivity-in-ovarian-cancer https://edusehat.com/en/targeting-metabolic-mechanism-restores-chemotherapy-sensitivity-in-ovarian-cancer Mon, 01 Jun 2026 01:45:17 +0700 Edusehat Targeting, Metabolic, Mechanism, Restores, Chemotherapy, Sensitivity, Ovarian, Cancer Although many cancers can be successfully treated using platinum-based chemotherapies, which work by damaging DNA, a subset avoid cell death by repairing their own DNA. Ovarian cancers are an example. Patients whose tumors are DNA repair proficient historically face poor prognosis and their tumors commonly recur within months. 

    Now data from a new study done in cells and mice points to a potential metabolic target that could prevent tumor cells from repairing their own DNA, thus overcoming their resistance. The work was done by scientists from The Wistar Institute, Temple University, and their collaborators elsewhere. Details are published in a new Nature paper titled “αKG-mediated carnitine synthesis drives DNA repair via histone acetylation.” In it, they describe a metabolic process that is altered in cancer cells that makes them resistant to DNA-damaging agents. They have also identified a drug that can inhibit the pathway that may offer a strategy for overcoming chemotherapy resistance. 

    Specifically, the study centers on alpha-ketoglutarate (αKG), a metabolite which accumulates in DNA repair proficient ovarian tumors. First, the scientists confirmed αKG’s role in helping ovarian cancer cells repair DNA and survive chemotherapy treatment. They did this by using a CRISPR-based approach to systematically search for the enzyme that enables αKG to repair DNA. 

    Previous studies on αKG focused on its role in demethylation of proteins and other molecules. Though the scientific literature pointed towards demethylases as the key enzyme, the scientists focused onTMLHE, an enzyme that initiates the synthesis of carnitine, a molecule often associated with energy metabolism. “Finding TMLHE was the moment I thought, ‘Okay, this is going to be something bigger than what we expected,’” said Katherine Aird, PhD, professor and co-leader of the molecular and cellular oncogenesis program at The Wistar Institute and senior author of the study.

    The data indicated that elevated αKG activates TMLHE, which drives carnitine production. Carnitine then carries acetyl groups out of the mitochondria and into the nucleus where they are deposited onto histones. This loosens the DNA-histone complex which allows the cells repair machinery to access and fix DNA damage. 

    Next the team showed that when TMLHE or carnitine synthesis is blocked, histone acetylation does not occur which prevents the DNA repair machinery from doing its work. In these cases, the cells become significantly more sensitive to DNA-damaging chemotherapies. “The connection between αKG and methylation is well established—that’s what everyone studies,” said Nathaniel Snyder, PhD, associate professor in the Aging + Cardiovascular Discovery Center at Temple University School of Medicine. “What we found is that αKG is also controlling acetylation through a completely separate route, and that route turns out to be essential for DNA repair. That’s a new piece of biology that nobody had described before.”

    As part of the study, the scientists tested the effects of mildronate, a carnitine synthesis inhibitor, and cisplatin, a platinum-based DNA-damaging chemotherapy drug. They found that the combination of these treatments reduced the tumor burden in mouse models of ovarian cancer, while neither drug alone produced a significant effect. Additionally, patients with high TMLHE expression in tumor tissue had significantly worse progression-free survival post chemotherapy, and higher serum acetylcarnitine levels at diagnosis correlated with faster disease progression. 

    That latter finding suggests that it may one day be possible to use a routine blood test for circulating acetylcarnitine to identify patients that are most likely to resist standard platinum-based cancer treatments, and to benefit from a combination therapy. 

    The post Targeting Metabolic Mechanism Restores Chemotherapy Sensitivity in Ovarian Cancer appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     PRINCE: A Small-Molecule Switch for Safer Gene Editing https://edusehat.com/en/prince-a-small-molecule-switch-for-safer-gene-editing https://edusehat.com/en/prince-a-small-molecule-switch-for-safer-gene-editing Mon, 01 Jun 2026 01:45:17 +0700 Edusehat PRINCE:, Small-Molecule, Switch, for, Safer, Gene, Editing Although gene editing has enormous clinical promise, it still faces many obstacles that must be overcome before broad translation. For example, the genome editing field is continuously working to increase the safety of the technique. One way to do that is to control the duration of gene editing activity. However, achieving precise temporal control over these platforms is challenging.

    Scientists currently lack tools that can precisely control the duration of gene editing therapies, to halt their effects after a few months or years. Researchers have developed several potential solutions, including degradable protein- or RNA-based systems, but existing approaches face limitations such as only being applicable in the liver.

    Now, a new gene editing system, named PRINCE, with inducible nuclease proteins and guide RNAs, enables researchers to control the duration and specificity of gene therapies precisely with small molecules—potentially addressing a longstanding safety concern in the field.

    This work is published in Science Translational Medicine in the paper, “Coordinated regulation using small-molecule drugs enables controlled therapeutic genome editing and enhanced genomic precision in situ.“

    In the PRINCE CRISPR-Cas gene editing system, expression of the nuclease and the guide RNA is separately inducible by two approved small molecule drugs, enabling both temporal control and reduced off-target editing.

    The platform was stable for as long as two years after genomic integration in cultured human cells. In addition, a smaller system, called Little Prince, showed promising signs of efficacy in humanized mouse models of elevated cholesterol levels and age-related macular degeneration. Little Prince uses compact nucleases that can be delivered in a single adeno-associated viral vector (AAV) for delivery.

    In two mouse models, Little Prince reduced excessive cholesterol levels in mice with genetic hypercholesterolemia and showed signs of reducing lesion size in rodents with laser-induced choroidal neovascularization—a model of age-related macular degeneration.

    More specifically, Little Prince “ameliorated pathological phenotypes of hypercholesterolemia (average reductions of 45% and 47% in serum total cholesterol and low-density lipoprotein cholesterol, respectively) and neovascular age-related macular degeneration, with significantly reduced lesion size and leakage (P < 0.0001).”

    The system produced fewer off-target edits and lower off-target editing frequencies than constitutive nuclease expression, highlighting the utility of precise temporal control.

    “PRINCE and Little Prince also provide […] capabilities that might also be useful for research objectives that include lineage tracing and conditional genetic engineering work,” the authors noted. These results, they asserted, position PRINCE and Little Prince as controlled genome editing platforms with potential for in vivo, particularly in situ, therapeutic applications.

    The post PRINCE: A Small-Molecule Switch for Safer Gene Editing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Target for Aggressive Prostate Cancer Prevention Identified in Mice https://edusehat.com/en/target-for-aggressive-prostate-cancer-prevention-identified-in-mice https://edusehat.com/en/target-for-aggressive-prostate-cancer-prevention-identified-in-mice Mon, 01 Jun 2026 01:45:16 +0700 Edusehat Target, for, Aggressive, Prostate, Cancer, Prevention, Identified, Mice Researchers at Columbia University Irving Medical Center have identified a gene that drives the development of neuroendocrine prostate cancer (NEPC), an aggressive form of the disease. Their study showed that genetic or pharmacological inhibition of Sirtuin 1 prevents the growth of NEPC tumors in mice, laying the groundwork for future clinical studies aimed at developing new treatments for NEPC in humans.

    Research lead Cory Abate-Shen, PhD, a professor at Columbia University Vagelos College of Physicians and Surgeons, is co-senior author of the researchers’ published paper in Journal of Experimental Medicine, titled “A forward genetic screen identifies Sirtuin 1 as a driver of neuroendocrine prostate cancer,” in which the team noted, “We demonstrate that expression of Sirt1 promotes NEPC while its silencing or pharmacological inhibition suppresses the NEPC phenotype.”

    Prostate cancer is the most common cancer in men, and one in every six men will be affected by prostate cancer in their lifetime, the authors explained. Prostate cancer treatments have been focused on approaches to dampening androgen receptor (AR) signalling, and for men with recurrent or advanced prostate cancer the current standard of care is androgen deprivation therapy (ADT). However, it is well documented that ADT will eventually fail, leading to tumor recurrence and development of the ADT-insensitive aggressive prostate cancer variant, NEPC. “… while ADT initially leads to tumor regression, eventually tumors recur as castration-resistant prostate cancer (CRPC), so called because of its continued reliance on AR even in the absence of androgens,” the team continued.

    Positively stained NEPC markers (top) are lost when Sirt1 is silenced (bottom). [© 2026 Nunes de Almeida et al. Originally published in <em>Journal of Experimental Medicine</em>. https://doi.org/10.1084/jem.20241484]
    Positively stained NEPC markers (top) are lost when Sirt1 is silenced (bottom). [© 2026 Nunes de Almeida et al. Originally published in Journal of Experimental Medicine.]
    The process through which ADT-responsive tumors transition towards NEPC tumors—a phenomenon known as lineage plasticity—remains unknown. “Elucidating the mechanisms governing this process may improve treatment by overcoming plasticity-associated drug resistance,” Abate-Shen added.

    For their newly reported study the research team performed a Sleeping Beauty (SB) forwards genetic mutagenesis screen in mice looking for mutations that recurred across multiple independent prostate cancer tumors. They identified 75 candidate NEPC-promoting genes, the most promising of which was Sirtuin 1 (Sirt1). Sirt1 encodes an enzyme with a broad range of functions, including control of gene expression and metabolism.

    The group first looked to a human prostate cancer cell line to characterize the role of Sirt1. In these cells, the induction of NEPC produced an increase in the expression of genes predicted to be activated by SIRT1 and a corresponding decrease in those predicted to be downregulated by this protein. Confirming these results, the group found that activation of Sirt1 in cells with low SIRT1 expression levels led to a robust increase in key NEPC markers.

    Recapitulating their cell line data, the team found that silencing of Sirt1 profoundly reduced tumor growth in mice with NEPC, indicating that Sirt1 is indeed a promising target for NEPC treatment. They also treated the tumors with the FDA-approved SIRT1-inhibitor, Selisistat, which was originally developed for treatment of Huntington’s disease. Encouragingly, the researchers saw that Selisistat administration significantly reversed the NEPC phenotype. “Functional studies in human prostate cancer cell models and mouse organoid models using gain- and loss-of- function approaches in vitro and in vivo, as well as pharmacological inhibition, demonstrated that one of the top-ranked candidates, Sirt1, promotes NEPC,” they wrote in summary.

    Tumors surgically removed from Sirt1-silenced mice (bottom) are significantly smaller than tumors removed from mice with wild-type Sirt1 expression. [© 2026 Nunes de Almeida et al. Originally published in <em>Journal of Experimental Medicine</em>. https://doi.org/10.1084/jem.20241484]
    Tumors surgically removed from Sirt1-silenced mice (bottom) are significantly smaller than tumors removed from mice with wild-type Sirt1 expression. [© 2026 Nunes de Almeida et al. Originally published in Journal of Experimental Medicine.]
    “Our findings demonstrate that SIRT1 plays a pivotal role in promoting NEPC, revealing a context-dependent function that extends beyond general tumor growth to the regulation of lineage plasticity and neuroendocrine differentiation,” says Abate-Shen, adding that “this highlights SIRT1 as an attractive and clinically actionable target for lethal prostate cancer that warrants further investigation in future clinical studies.”

    In their paper the team concluded, “Overall, our study establishes a generalizable computational and experimental framework that integrates SB mutagenesis with phenotypic, genomic, and transcriptomic analyses to identify novel cancer drivers … Importantly, our data suggest that targeting SIRT1 may suppress or reverse progression toward NEPC.”

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    ]]>     Bonito Biosciences and DaltonTx Collaborate on Precision Delivery for Oligotherapeutics https://edusehat.com/en/bonito-biosciences-and-daltontx-collaborate-on-precision-delivery-for-oligotherapeutics https://edusehat.com/en/bonito-biosciences-and-daltontx-collaborate-on-precision-delivery-for-oligotherapeutics Mon, 01 Jun 2026 01:45:15 +0700 Edusehat Bonito, Biosciences, and, DaltonTx, Collaborate, Precision, Delivery, for, Oligotherapeutics Bonito Biosciences and DaltonTx agreed to collaborate to help advance next-generation precision delivery systems for Bonito’s oligotherapeutic medicines. The collaboration will initially focus on delivery to the central nervous system, combining Bonito’s functional delivery data engine and ligand discovery platform with DaltonTx’s AI systems which are designed to iteratively model, predict, and optimize precision delivery biology.

    Together, the companies will work to identify, optimize, and advance delivery ligands for conjugated oligotherapeutic payloads, including novel bispecific approaches for targeted CNS delivery, according to officials at both firms.

    Through its functional selection platform, Bonito screens hundreds of billions of encoded ligands against complex cellular delivery phenotypes, explained a Bonito spokesperson, adding that these datasets–physically generated and directly tested on cell systems of interest– capture high-dimensional information related to receptor engagement and cellular delivery.

    DaltonTx will apply its AI capabilities to model these data and to work on accelerating the design and optimization of next-generation delivery ligands through structural prediction, affinity mapping, and developability analysis. Together, the companies aim to establish an iterative learning loop for the rapid discovery of precision delivery systems for conjugated oligotherapeutics, noted Richard Wagner, CEO of Bonito.

    “This collaboration brings together two highly complementary capabilities: Bonito’s ability to generate uniquely rich functional delivery data at massive scale and DaltonTx’s deep expertise in adaptive AI systems for molecular design and optimization,” he continued. “We believe precision delivery is fundamentally a data and prediction problem. By combining closed-loop functional screening with AI-driven design, we aim to accelerate the discovery of delivery systems capable of reaching tissues and cell types that have historically remained inaccessible to oligotherapeutics.”

    “AI systems are only as powerful as the quality and dimensionality of the underlying biological data,” pointed out Garry Pairaudeau, CEO and co-founder of DaltonTx. “Bonito has built a uniquely differentiated platform for generating functional delivery datasets at extraordinary scale. Combining these data with DaltonTx’s agentic AI platform, which integrates AI, human expertise, and experimental data into one continuous learning engine, has the potential to significantly accelerate the discovery of next-generation delivery systems for conjugated oligotherapeutics.”

     

     

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    ]]>     Neuronal Protein Tracing Reveals How the Brain Routes Its Waste https://edusehat.com/en/neuronal-protein-tracing-reveals-how-the-brain-routes-its-waste https://edusehat.com/en/neuronal-protein-tracing-reveals-how-the-brain-routes-its-waste Mon, 01 Jun 2026 01:45:14 +0700 Edusehat Neuronal, Protein, Tracing, Reveals, How, the, Brain, Routes, Its, Waste The brain is one of the busiest organs in the body, constantly processing and reshaping itself. That activity produces an equally constant stream of molecular byproducts—proteins that need to be moved out before they accumulate. When those clearance routes slow or break down, waste lingers, and the consequences can be profound. In Alzheimer’s disease, for example, toxic proteins build up in vulnerable regions. Yet despite decades of research, scientists have lacked a clear view of how waste normally leaves the brain.

    A new study from the Gladstone Institutes offers the clearest picture yet of how the brain normally takes out its trash—and what happens when those routes fail. Published in Cell as “Physiological brain clearance architecture revealed by neuronal protein tracing,” the work introduces a method that traces waste proteins from the moment they are produced inside neurons to the moment they leave the brain.

    For decades, researchers have relied on injecting tracers into the cerebrospinal fluid (CSF) to visualize drainage. But this approach, while illuminating, shows all possible routes, instead of pinpointing the most-used exit. “These injected tracers disturb the very system we’re attempting to measure,” said lead author Andrew Yang, PhD, a Gladstone investigator. “We wanted to find a better way.”

    Yang’s team engineered neurons in mice to produce a fluorescent protein, ZsGreen, that could be followed as it exited the brain through its natural routes. This allowed the researchers to track waste as it moved into the dura, skull, nasal cavity, and lymph nodes—regions populated by specialized immune cells that interact with brain‑derived proteins.

    The resulting map diverged sharply from the field’s long‑held assumptions. Traditional CSF tracers had pointed to the cervical lymph nodes as a major drainage site. But the new method revealed that very little neuronal waste actually reaches those nodes. “We were surprised to find that very little ZsGreen drained to the cervical lymph nodes,” Yang said. “Instead, waste drained through the dura, skull, and nasal cavity. Our findings underscore why tracking waste proteins themselves, rather than movement of the cerebrospinal fluid, provides a more accurate understanding of waste clearance dynamics.”

    The team also uncovered a striking organizational principle: where a protein is made determines where it drains. Proteins produced in upper forebrain regions exited through upper routes, while those from deeper structures, such as the striatum, used lower pathways. The researchers call this the brain’s “nearest‑exit” model. “It’s like each brain region has a biological ZIP code system to ensure waste will be sent to the correct drainage site,” said Nalini Rao, PhD, a postdoctoral fellow. She noted that in aging or disease, these ZIP codes may become scrambled, potentially explaining why certain regions are more vulnerable to disorders like Alzheimer’s.

    Disease models reinforced the system’s fragility. In mice with acute inflammation, ZsGreen leaked directly into the bloodstream, bypassing normal routes. In an Alzheimer’s model, waste became trapped inside the brain, unable to drain effectively. “Understanding how diseases disrupt brain clearance could help us design therapeutics to target the brain border compartments and enhance waste removal,” Rao said.

    With their new tracing method, Yang’s group plans to probe how clearance changes across aging, sleep, and disease—and whether brain tumors exploit these pathways to evade immune detection. The architecture of brain waste disposal, once opaque, is now open for exploration.

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    ]]>     Illumina Announces MRD Kit Ahead of ASCO Meeting https://edusehat.com/en/illumina-announces-mrd-kit-ahead-of-asco-meeting https://edusehat.com/en/illumina-announces-mrd-kit-ahead-of-asco-meeting Mon, 01 Jun 2026 01:45:13 +0700 Edusehat Illumina, Announces, MRD, Kit, Ahead, ASCO, Meeting Ahead of the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, kicking off this weekend, Illumina has announced a new molecular residual disease (MRD) product. The distributed kit enables solid tumor MRD and blood cancer genomic profiling and, the company says, will enable more labs to adopt MRD detection for clinical research.

    It is the first in a new portfolio of WGS oncology research offerings, with additional solutions in development leveraging the latest advancements of the NovaSeq X. Illumina’s MRD research solution is available today for early access to select partners and will launch for global customers next year.

    “In precision healthcare, early and accurate detection of molecular residual disease is critical to monitoring patients during and after cancer treatment,” said Todd Christian, senior vice president of Services, Arrays, and Genomic Access at Illumina. “Illumina’s MRD solution for clinical research leverages the advanced sensitivity of whole-genome sequencing, coupled with unparalleled analysis, to enable our customers to more easily deliver the most precise information to advance MRD research. We aim to make WGS in oncology more accessible and scalable to support the integration of precision solutions into the standard of care.”

    The MRD solution supports fingerprinting through solid tumor samples, and MRD detection using blood samples, all compatible on NovaSeq Systems. The end-to-end research workflow can be completed in as fast as five days and is optimized for analytical sensitivity as low as 10 ppm, particularly important for early-stage and low-shedding tumors, including breast, ovarian, and renal.

    Illumina’s DRAGEN MRD analysis connects each fingerprint to serial circulating tumor DNA (ctDNA). The new MRD solution has been optimized across thousands of samples to develop and demonstrate a ctDNA detection algorithm with 99.5% analytical specificity to distinguish true tumor signals from background noise.

    Mayo Clinic evaluated the solution on a small sample cohort and found high concordance among previously characterized paired samples. The results were also highly correlated with clinical and imaging results over time. The team is planning to expand the cohort for additional research with Mayo Clinic and other academic partners.

    “We are looking forward to participating in early access and evidence generation for a tumor-informed, non-bespoke whole-genome sequencing approach to MRD,” said Gang Zheng, MD, PhD and professor of Laboratory Medicine and Pathology at Mayo Clinic. “We have seen early pilot results across several solid tumor clinical samples that demonstrated the potential utility of highly sensitive solid tumor MRD detection, and we continue to pilot technologies that help us efficiently progress in our ability to analyze and translate complex genomic arrays.”

    Built on recently announced NovaSeq X advancements, including 35B output and Q70 quality scores, a complementary research workflow that will deliver ultra-sensitive MRD detection in the single-digit ppm range leveraging duplex reads is currently in development.

    Illumina’s new oncology portfolio builds upon the integrated ecosystem of workflows, data and community across genomic, multiomic, and clinical research applications—anchored on the NovaSeq X.

    Illumina and Bristol Myers Squibb will jointly present a poster at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting on Sunday, May 31, from 9:00 a.m. to 12:00 p.m. (abstract ID 8591, poster board #381, Lung Cancer: Non–Small Cell Metastatic track).

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    ]]>     Antibiotic Design Strategy Overcomes Efflux-Mediated Resistance in Preclinical Study https://edusehat.com/en/antibiotic-design-strategy-overcomes-efflux-mediated-resistance-in-preclinical-study https://edusehat.com/en/antibiotic-design-strategy-overcomes-efflux-mediated-resistance-in-preclinical-study Mon, 01 Jun 2026 01:45:11 +0700 Edusehat Antibiotic, Design, Strategy, Overcomes, Efflux-Mediated, Resistance, Preclinical, Study Researchers headed by a team at King’s College London have developed a new way of designing antibiotics that could support the discovery of new treatments for drug-resistant infections.

    Designed to overcome one of the ways bacteria escape antibiotic treatment, the Efflux Resistance Breaker (ERB) approach allows researcher to chemically redesign antibiotics so that they are less easily removed from the cells by bacterial efflux pumps. The technology could also help revive antibiotics that have lost effectiveness due to the evolution of efflux-mediated resistance.

    Study lead Professor Khondaker Miraz Rahman, PhD, a professor of medicinal chemistry at King’s College London, said: “Antimicrobial resistance is rising, but the number of truly new antibiotics in development remains worryingly low. Our work shows that we can redesign antibiotics so they stay inside bacterial cells at higher concentrations and overcome resistance mechanisms that would normally make them ineffective. This approach could help us design better new antibiotics, but it could also help revive existing antibiotic classes that bacteria have learned to defeat.”

    Rahman is senior author of the team’s published paper in Journal of Medicinal Chemistry, titled “Designing Antibiotics with Inherent Resistance to Efflux as a Strategy to Revive Discovery against Multidrug-Resistant Pathogens.”

    Worldwide increase in antimicrobial resistance (AMR) is threatening new developments in antibiotics, the authors noted. “The development and approval of new antibiotics are currently being outpaced by the emergence of resistance to existing drugs, a trend that must be reversed to ensure the long-term effectiveness of antibiotics.”

    Many bacteria use molecular pumps, known as efflux pumps, to push antibiotics out of the cell before the drugs can reach levels high enough to kill them. This reduces the amount of antibiotic inside the bacteria and allows resistant infections to survive. Previous strategies have tried to combine antibiotics with separate efflux pump inhibitors (EPIs), the team continued. “Efflux pump inhibitors (EPIs) have been pursued as adjunct therapies to safeguard approved antibiotics prone to efflux-based resistance.” However, no EPI has yet been approved. “As well as a lack of mechanistic insight and biochemical information regarding efflux pumps, we opine that this failure is rooted in a fundamental flaw in the EPI-antibiotic combination approach: that the antibiotics remain unmodified substrates and can be effluxed by different pumps despite the presence of EPIs,” the authors noted.

    The study by Rahman and colleagues has now shown that antibiotics can be chemically redesigned so they are less easily removed by these pumps. Their approach builds resistance-breaking properties directly into the antibiotic molecule, meaning that the antibiotic is designed to protect itself from being pumped out, allowing it to remain inside the bacterial cell at higher concentrations, and so restoring its ability to kill bacteria even when resistance mechanisms are present.

    Importantly, the work shows that the ERB approach could support a new way of developing antibiotics by building resistance-breaking properties directly into their design. In their reported study the team developed ERB-modified fluoroquinolones and demonstrated their effectiveness against multiple bacterial pathogens, and in mouse infection models. The study provides an important proof of concept for antibiotic discovery, showing that maintaining high intracellular antibiotic concentration can help overcome resistance, including in bacteria that already show reduced susceptibility to existing antibiotics. “This study demonstrates that ERB modification enhances intracellular accumulation, reduces efflux susceptibility, and preserves antibacterial potency, as supported by complementary mechanistic, biochemical, and in vivo evidence,” the scientists concluded.

    Added J. Mark Sutton, PhD, at the UK Health Security Agency, a key collaborator on this project, “Efflux pumps are a major cause of antibiotic resistance because they reduce the concentration of drug inside the bacterial cell. This study shows that rational chemical design can be used to overcome that problem. By building efflux resistance directly into the antibiotic, we may be able to restore activity against bacteria that are no longer controlled by current drugs.”

    The researchers believe the ERB platform could be used as a general strategy to design antibiotics with built-in resilience to efflux-mediated resistance. Their team describes the ERB technology as a framework for developing next-generation antibiotics and for revitalizing existing drugs. “Beyond revitalizing existing drugs, ERB technology provides a general framework for designing next-generation antibiotics with built-in resilience to efflux-mediated resistance at the earliest stages of discovery,” they stated.

    The team says it will work towards commercializing the ERB technology and advancing antibiotics developed using this strategy towards clinical development, with the aim of translating this discovery into new treatment options for drug-resistant infections.

    The post Antibiotic Design Strategy Overcomes Efflux-Mediated Resistance in Preclinical Study appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AI-Powered Pan-Cancer Map Reveals Tertiary Lymphoid Structures https://edusehat.com/en/ai-powered-pan-cancer-map-reveals-tertiary-lymphoid-structures https://edusehat.com/en/ai-powered-pan-cancer-map-reveals-tertiary-lymphoid-structures Mon, 01 Jun 2026 01:45:10 +0700 Edusehat AI-Powered, Pan-Cancer, Map, Reveals, Tertiary, Lymphoid, Structures Researchers at The University of Texas MD Anderson Cancer Center have developed a spatial atlas of specialized immune structures known as tertiary lymphoid structures (TLSs), across multiple cancer types, revealing how key features vary across tumor types and influence patient outcomes. Led by Linghua Wang, MD, PhD, professor of genomic medicine, executive director and head of the Center for Cellular Language Intelligence, associate member of the James P. Allison Institute™, and focus area co-lead with the Institute for Data Science in Oncology at UT MD Anderson, the team developed scalable artificial intelligence (AI) frameworks to detect, profile and classify TLSs from spatial omics data and routine pathology slides.

    Tumors can contain TLSs with very different levels of organization, cellular composition and spatial relationships within tumor cells and the researchers’ newly reported study showed that these differences carry important biological and clinical information. The team suggests that their first-of-its-kind atlas indicates that TLS maturation state, spatial location, and composition within tumors may provide clinically meaningful information about cancer prognosis and treatment response. They also created a composite scoring system to more effectively stratify patients by prognosis and treatment response across different cancer types and treatment contexts.

    “Prior to this study, most of the focus on TLSs as biomarkers was simply on whether or not they were present and, in some cases, whether they were mature,” Wang said. “Here, we show that we can go much deeper. TLSs in tumor tissues are much more complex than that. Their maturation state, spatial location and composition within tumors can tell us critical information about the tumor immune microenvironment, treatment response and clinical outcomes.”

    Wang is senior author of the team’s published paper in Science, Titled “Pan-cancer spatial atlas of tertiary lymphoid structures.” In their paper the team concluded, “Together, this work provides a comprehensive landscape of TLS heterogeneity across cancers and establishes spatially defined TLS features and artificial intelligence (AI)–driven TLS classification as scalable tools for precision immuno-oncology.”

    The immune system’s response to a tumor is a highly coordinated effort taking place within the tumor microenvironment (TME), the authors explained. In some tumors, immune cells come together to form organized structures called tertiary lymphoid structures, or TLSs. These structures operate as local immune “hubs,” bringing together B cells, T cells, antigen-presenting cells and other supporting cells that help coordinate antitumor immune responses. “TLSs frequently develop within the tumor microenvironment (TME) and have been observed across a broad range of human solid tumors, where they contribute to lymphocyte activation, B cell immunity, and regulation of antitumor immune responses,” they noted.

    Previous studies have shown that TLSs—particularly those that are more mature—are often associated with better patient outcomes and improved responses to immunotherapy across a variety of cancer types. “The presence of TLSs has been linked to favorable responses to immune checkpoint blockade (ICB) and prolonged survival across multiple cancer types, fueling interest in TLSs as predictive biomarkers, prognostic indicators, and potential therapeutic targets. However, the presence of TLSs alone does not tell the whole story,” the scientists noted. “While it is well acknowledged that TLSs are important in cancer, our understanding of their cellular and molecular heterogeneity has remained limited, especially in their natural spatial context across large cohorts of human tumor samples.”

    “Although TLS presence has been associated with enhanced immune activity and improved outcomes in several settings, their maturation states, spatial locations relative to tumors, and context-dependent associations have not been systematically characterized at a pan-cancer scale, limiting a unified view of TLS biology and clinical utility,” they stated.

    For their reported study the team developed scalable computational frameworks to precisely detect, comprehensively profile and classify TLSs from spatial omics data. Leveraging this framework, the team built a pan-cancer spatial atlas of TLSs across 340 samples from 12 cancer types. This atlas allowed them to examine the TLS landscape in tumor tissues, to define how TLSs vary in key features, and identify transcriptional programs associated with TLS maturation. “By integrating transcriptomic, spatial, histopathological, and clinical data, we systematically characterized TLS abundance, spatial distribution, size, maturation states, and transcriptomic programs in 340 ST samples across 12 cancer types and examined their interactions with tumor cells and the surrounding TME,” they wrote in summary.

    The study found that TLSs vary substantially across tissues. As TLSs mature, they become more organized and undergo coordinated changes in immune, stromal, and vascular components. Further, their proximity to tumor cells is associated with spatial gradients of tumor signaling. These findings suggest that TLS maturation and spatial context are linked to distinct tumor signaling environments and may reflect important features of the tumor immune microenvironment.

    To make these insights more scalable, the team developed an AI framework to rapidly identify and classify TLSs from hematoxylin and eosin (H&E) whole-slide images (WSIs), pathology images that are routinely used in daily clinical care. Training this AI model makes the process of analyzing TLSs more easily translatable to the clinic, while also making the process significantly faster and more scalable. The AI framework enabled the researchers to go one step further, evaluating 25,088 TLSs from more than 3,000 whole-slide images across 10 independent cohorts and developing a TLS “composition score” for a given patient’s tumor. “By developing a scalable AI-enabled framework to detect and classify TLSs directly from routine H&E WSIs, we have extended TLS analysis from limited spatial datasets to thousands of tumors,” the team noted.

    This composition score captures not only the number of TLSs, but also their maturation states within a tumor. This method significantly outperformed conventional TLS measures in stratifying patients by prognosis and treatment response, suggesting that a more detailed view of TLS biology, accounting for maturation state, may provide more clinically meaningful information than TLS presence alone. “… we developed a data-driven, unsupervised TLS-based patient stratification framework that outperformed existing approaches in prognostic evaluation,” they commented.

    The TLS composite scoring approach must be validated in prospective clinical trials. If successful, the framework could support broader integration of TLS profiling into routine pathology workflows, since it uses routine pathology images. “Together, this work establishes generalizable and clinically scalable frameworks for TLS profiling and highlights TLS state composition as a key dimension of tumor immune organization with translational relevance. It also provides a foundation for prospective evaluation of TLS-informed biomarkers in clinical settings,” they stated.

    The findings raise important biological and therapeutic questions, the researchers suggest. One important observation from the study is that many TLSs in tumor tissues remain immature, and some are located away from tumor regions rather than within or adjacent to tumor cells. This suggests that future studies should investigate how to promote TLSs toward more mature and functional states, and how to enhance their spatial interaction with tumor cells and the broader tumor microenvironment.

    These efforts may help identify therapeutic strategies to promote effective TLS formation and maturation and enhance TLS-associated anti-tumor immune responses. In their paper the team concluded, “Prospective studies should test whether TLS composition improves risk and response modelling beyond established clinicopathologic and molecular predictors, and whether TLS-informed stratification can guide clinical trial design or therapeutic modulation strategies.”

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    ]]>     Pfizer, Innovent Ink Up-to-$10.5B+ Cancer Treatment Collaboration https://edusehat.com/en/pfizer-innovent-ink-up-to-105b-cancer-treatment-collaboration https://edusehat.com/en/pfizer-innovent-ink-up-to-105b-cancer-treatment-collaboration Mon, 01 Jun 2026 01:45:09 +0700 Edusehat Pfizer, Innovent, Ink, Up-to-10.5B, Cancer, Treatment, Collaboration Pfizer and Innovent Biologics will partner to research and develop 12 early-stage and de novo antibodies and antibody-drug conjugates (ADCs) designed to treat various cancers, the companies said today, through a collaboration that could generate up to $10.5 billion for the Chinese biotech.

    The companies said they have signed a strategic global licensing and collaboration agreement that includes licensing, co-development, and co-commercialization deals for both the ADCs, which would be created with payloads that differentiate them from other conjugates, as well as multi-specific antibodies, to be developed with unique designs and differentiated immune-engaging features.

    Pfizer and Innovent plan to work across a portfolio of 12 programs—eight early-stage programs originating with Innovent, and four Pfizer discovery programs. The companies said they will co-develop and share costs for selected programs as they advance them through clinical development.

    The collaboration is intended to marry Pfizer’s global scientific, clinical development, regulatory, and commercial scale capabilities with Innovent’s scientific discovery and clinical capabilities in oncology.

    “By combining Innovent’s discovery and early clinical development with Pfizer’s global research and development and commercialization capabilities, we have an opportunity not only to strengthen our pipeline, but to accelerate the delivery of breakthroughs that can redefine standards of care and make a meaningful difference in patients’ lives,” Jeff Legos, Pfizer’s chief oncology officer, said in a statement.

    Racing the ‘patent cliff’

    Like other biopharma giants, Pfizer is racing the proverbial “patent cliff” by building a pipeline of new treatments capable of recouping the billions that it stands to lose in coming years as its aging blockbuster drugs lose patent exclusivity in the U.S. and other key markets.

    GEN’s A-List of Top 20 Drugs Heading for the Patent Cliff through 2029 includes two Pfizer treatments. One is Prevnar 13/Prevenar 13 (pneumococcal 13-valent conjugate vaccine [diphtheria CRM197 Protein]), which lost U.S. exclusivity starting March 31; and the prostate cancer drug Xtandi® (enzalutamide), co-marketed with Astellas Pharma and set to lose U.S. exclusivity in 2027.

    Pfizer generated $6.494 billion last year, up 1% from 2024, and another $1.69 billion in the first quarter, up 2% from a year ago, in revenues from its Prevnar family, which includes Prevnar 20/Prevenar 20 (Pneumococcal 20-valent Conjugate Vaccine), as well as the Prevnar 13/Prevenar 13 vaccines.

    Xtandi racked up $2.194 billion in 2025, up 8% from a year earlier, and $444 million in Q1, down 3% from the year-ago quarter, in revenues in the U.S. and more than 90 other countries, including the EU and Japan—primarily reflecting alliance revenues and royalty revenues.

    Oncology focus

    Oncology is among therapeutic areas where Pfizer has sought to bolster its pipeline and portfolio of marketed drugs in recent years. The pharma giant acquired ADC-focused drug developer Seagen for $43 billion in 2023, a deal that cleared expected antitrust hurdles and doubled Pfizer’s oncology pipeline to some 60 programs spanning multiple modalities, including ADCs, small molecules, bispecifics and other immunotherapies.

    Last year, Pfizer launched an up-to-$1.5 billion global ex-China licensing agreement with another Chinese biotech, 3SBio. In that deal, Pfizer agreed to pay $1.25 billion upfront, make a $100 million equity investment in 3SBio equity, and pay up to $150 million in return for an option for exclusive development and commercialization rights in China to SSGJ-707, a bispecific antibody targeting PD-1 and VEGF.

    Outside of oncology, Pfizer acquired obesity drug developer Metsera for up-to-$10 billion last October following a bidding war with Novo Nordisk that ended with some help from Washington.

    And in February, Pfizer inked a commercialization deal of undisclosed value with Chinese drug developer Hangzhou Sciwind Biosciences giving Pfizer exclusive commercialization rights in China to the obesity therapy ecnoglutide, an new‑generation cAMP‑biased glucagon-like peptide 1 (GLP‑1) receptor agonist delivered via injection.

    While Innovent has successfully developed an obesity treatment, mazdutide, the company specializes in cancer drug development, building a combined portfolio and development pipeline of 37 programs—23 of them in oncology including the PD-1 inhibitor Tyvyt® (sintilimab). The fully human IgG4 monoclonal antibody was first approved in China in 2018 and is now indicated to treat three forms of non-small cell lung cancer, classic Hodgkin’s lymphoma, and forms of endometrial, gastric/esophageal, kidney, and liver cancers.

    ‘Greater speed and impact’

    “By leveraging both companies’ complementary resources, we can develop our early-stage oncology pipeline with greater speed and impact to help bring innovative therapies to patients more efficiently worldwide,” stated Hui Zhou, MD, PhD, Innovent’s chief R&D officer for its oncology pipeline. “Furthermore, co-developing and co-commercializing key projects in the U.S. and Europe expands Innovent’s global reach.”

    Innovent plans to carry out development of the Pfizer-partnered programs through Phase I, applying its discovery engine and robust early clinical capabilities, after which Pfizer will oversee future global development. Pfizer will receive an exclusive global license for four programs, and assume their global development costs, as well as an exclusive license outside Greater China for four other programs, having agreed to shoulder an unspecified majority of the development costs.

    Pfizer and Innovent also agreed to co-develop four programs globally, sharing their development costs. The companies plan to co-commercialize in the U.S., the European Union (E.U.), and the United Kingdom (U.K.), agreeing in return to share their profits—while Innovent will retain Greater China rights to these programs.

    Pfizer has agreed to pay Innovent $650 million upfront and up to $9.85 billion in payments tied to achieving development, regulatory, and commercial milestones. Pfizer also agreed to pay Innovent up to double-digit royalties on sales of each licensed product if approved, with the companies agreeing to share their profits in the U.S., the EU, and the U.K.

    The collaboration transaction is expected to close in the third quarter, subject to regulatory approvals.

    In October, Innovent launched an up to $11.4 billion collaboration with Takeda Pharmaceutical aimed at speeding up the development of Innovent’s next-generation immuno-oncology therapies as well as ADCs.

    The post Pfizer, Innovent Ink Up-to-$10.5B+ Cancer Treatment Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     The deadly Ebola outbreak is proving difficult to control https://edusehat.com/en/the-deadly-ebola-outbreak-is-proving-difficult-to-control https://edusehat.com/en/the-deadly-ebola-outbreak-is-proving-difficult-to-control Fri, 29 May 2026 23:20:03 +0700 Edusehat The, deadly, Ebola, outbreak, proving, difficult, control
  • No vaccine, no treatment: Unlike recent Ebola outbreaks, this one is caused by the Bundibugyo virus, for which no approved vaccine exists. Clinical trials for new ones are still months away.

  • Violence is making containment nearly impossible: Armed attacks have burned down two treatment centers and driven 18 infected patients back into the community. Conflict, damaged roads, and food insecurity have left health workers struggling to isolate cases or trace contacts.

  • US funding cuts have left the region exposed: Years of underinvestment, compounded by steep reductions in US global health funding under the Trump administration, have stripped away the surveillance systems and protective equipment needed to respond quickly
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    The alert was raised on May 5. Four health-care workers in the Ituri Province of the Democratic Republic of the Congo had died from an unknown illness within four days.

    Rapid response teams were sent to investigate, and tests at a research center in Kinshasa revealed the culprit: the Bundibugyo virus, one of the viruses that cause Ebola. Suspected cases of the disease have snowballed in the last few weeks. By May 24, the WHO had estimated that 223 people had died from the disease. There were over 900 suspected cases. Today’s figures are likely to be higher.

    A couple of weeks ago, I covered the hantavirus outbreak aboard a cruise ship. Three people sadly died, but the outbreak itself was kept under control. There have been no further deaths, and passengers have been safely repatriated. The picture for Ebola is far bleaker. And there are several reasons why.

    The most obvious is the disease itself. Ebola is a severe disease with an average 50% fatality rate. Previous outbreaks have resulted in thousands of deaths. (Hantavirus also has a high fatality rate, but it doesn’t usually spread as easily between humans.) 

    Between 2014 and 2016, an Ebola outbreak in West Africa caused more than 11,000 deaths. A more recent outbreak, which took place between 2018 and 2020, caused 2,299 deaths before being brought under control with a vaccination campaign.

    But those outbreaks were caused by the Zaire virus, which has a different genetic sequence. There is no vaccine for the Bundibugyo virus. We don’t know if the two vaccines approved for Zaire might also work for Bundibugyo. There’s a concern they might even make things worse by interfering with a person’s immune response to the virus.  

    Scientists are working on potential Bundibugyo vaccines. But the most advanced efforts are still months away from clinical trials. There are no specific antiviral treatments for the virus, either.

    So to control the outbreak, health-care workers are trying to stop the spread of the disease. Ebolaviruses can be transmitted to humans by animals including fruit bats, chimpanzees, and gorillas. They can then spread between people via contact with bodily fluids such as blood or vomit.

    That’s why the virus is often spread among family members, to health-care workers, and during some burial services. The WHO advises isolating people who have the virus in treatment centers. It also recommends safe burial measures that limit physical contact with the deceased, for example. Communities need to be informed about the virus and how it spreads, and health professionals should be on hand to diagnose cases and track them.

    That’s all easier said than done in an era of misinformation. Some members of the community even doubt whether the disease is real. There have been three attacks on health-care facilities in the region in recent weeks.

    Last week, two treatment centers were burned down. The first incident occurred after relatives of a deceased man were prohibited from retrieving his (infectious) body. As a result of the second incident, 18 suspected cases reentered the community.

    A couple of days later, a group of men unleashed gunfire at Mongbwalu General Hospital, which was also treating people with Ebola. They were demanding the bodies of their deceased relatives.

    There are more causes for concern when it comes to the spread of the virus. The Ebola outbreak is thought to have originated in Mongbwalu, a high-traffic mining hub. People who caught the virus in Mongbwalu are thought to have sought care in neighboring districts. And the wider province borders both South Sudan and Uganda. So far, Uganda has reported seven confirmed cases and one death. South Sudan’s health ministry has said it will strengthen surveillance, but no cases have been reported in the country so far. 

    Violence in the region is making it much harder to contain the spread of the virus, too. Conflict involving multiple armed groups, including deadly attacks on civilians, has hampered humanitarian and health-care efforts. Poor infrastructure and damaged roads make matters even worse. Food insecurity is ravaging the region as well—this year, nearly 10 million people in the region face acute hunger.

    Together, these factors are making it “nearly impossible” to isolate people with Ebola and trace others who have been in contact with them, WHO director general Tedros Adhanom Ghebreyesus said in a statement earlier this week.

    The dismantling of US aid programs hasn’t helped either. US government funding for international health projects has steeply declined since the start of President Donald Trump’s second term. These cuts have harmed disease surveillance systems, according to the International Rescue Committee, a humanitarian nonprofit.

    “Funding cuts have left the region dangerously exposed,” Heather Reoch Kerr, the organization’s country director for the Democratic Republic of the Congo, said in a statement. “Years of underinvestment and recent funding cuts have left many health facilities without adequate protective equipment, surveillance capacity, or frontline support needed to respond quickly and safely.”

    The US has mobilized emergency funding for the outbreak, and a spokesperson for the State Department has argued that none of the administration’s actions have hampered the Ebola response. But health experts counter that the damage has already been done.

    On May 17, the WHO declared the Ebola outbreak a public health emergency of international concern. In a statement on Wednesday, Tedros described the situation as “a catastrophic collision of disease and conflict with the Ebola outbreak in Ituri province outpacing the response.”In an online appeal to residents on Wednesday, ahead of an in-person visit, Tedros pleaded for a ceasefire and commended the spirit of community members. He also acknowledged the steep challenges they face. “You are already carrying so much: malaria, hunger, insecurity, and the daily struggle to keep your families safe,” he wrote in French. “And now Ebola. It’s not fair, and I won’t pretend otherwise.”

    This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.

    ]]>     Cystic Fibrosis Awareness Month: A patient advocate’s story https://edusehat.com/en/cystic-fibrosis-awareness-month-a-patient-advocates-story https://edusehat.com/en/cystic-fibrosis-awareness-month-a-patient-advocates-story Thu, 28 May 2026 00:20:04 +0700 Edusehat Cystic, Fibrosis, Awareness, Month:, patient, advocate’s, story Laura Bonnell always knew she wanted to be a news reporter.

    And throughout her entire career she was always on the hunt for answers. Little did she know, her investigative skills would have another use—as she navigated life as a mother to two daughters with cystic fibrosis (CF).

    May is both Cystic Fibrosis Awareness Month and Women’s Health Month. In observance of both campaigns, Bio.News sat down with Laura Bonnell, mother, patient advocate, and founder of the Bonnell Foundation: Living with Cystic Fibrosis, to discuss her experiences as a caregiver and advocate, and what more still needs to be done.

    Something is not right: Diagnosing CF

    “When Molly was born,” Bonnell recalled, “her pediatrician did not diagnose her. It was me saying, Something’s not right.”

    Initially, Bonnell’s pediatrician told Laura to go back to work, that she was an over-concerned full-time mom, but Bonnell couldn’t ignore the signs. Salty skin, greasy poop, distended stomach, failure to thrive, sinus issues—these were not the normal signs of a healthy baby.

    CF is a genetic disease that causes chronic and fatal lung infections, and interferes with digestion and every organ. The current life expectancy is about 56 years old. That’s a huge increase since Laura’s daughters were born in 1994 and 1997. Their life expectancy was 19 years old, which they have outlived. CF impacts about 40,000 people in the U.S. but that number is most likely low as many patients, in particular people of color, are underdiagnosed.

    Bonnell insisted Molly get tested. When the results came back positive, Bonnell was not surprised.

    Bonnell’s second daughter Emily was also born two and a half years later with CF. There is a one in four chance (or 25 percent) with each pregnancy that your child will have CF, if you’re a carrier. In 1989 scientists discovered the gene that causes CF through the Genome Project.

    When the Bonnell girls were diagnosed the sweat chloride test was the standard, and it still is today. The test measures the amount of salt produced on the skin during the test. If a patient tests 60 mmol/L or higher, then they have CF. Early diagnosis is important because it directly correlates to life expectancy, the sooner CF patients get on the proper medications, usually, the healthier they will be.

    “My husband Joe and I diagnosed Molly within three months of being born, but for some people it could be years,” Bonnell said. “The delay in diagnosis usually impacts the patients’ health and life expectancy negatively because medications don’t begin early enough.”

    Motherhood and CF advocacy

    The first pilot newborn screening programs for CF began in the early 1980s, and the screening process was dramatically improved in the 1990s by the inclusion of genetic tests that reduced false-positive results. The Centers for Disease Control and Prevention recommended universal screening for CF in 2004, but adoption still took a number of years.

    “I started advocating that CF be on the newborn screening panel almost immediately because University of Michigan pediatric pulmonologist Dr. Samya Nasr reached out to me,” Bonnell said. “In 2007, CF was finally added to newborn screening in my home state of Michigan.”

    Even though CF was included on the newborn screening panel in Michigan, Bonnell recognized the persistent challenges across the country.

    “There are more than 2,000 CF variants,” Bonnell explained. “If you’re testing for the more common variants, it tends to be white people. People of color generally have rare variants that often aren’t tested for in newborn screening which is part of the reason for late diagnosis. Michigan does a great job compared to other states—the fact that there is no consistency state by state, makes me crazy; your access to testing depends where you’re born. Last I checked, Mississippi tested for four different variants out of some 2,000, and Michigan tested for 66.”

    Ensuring newborns were screened for CF was just the start for Bonnell.

    Experiencing the challenges of navigating insurance claims, new treatment options, clinical trials, everything firsthand, soon led Bonnell to create the Bonnell Foundation. CF advocacy grew beyond a mother advocating for her daughters; it became a family affair—and a life mission. And yet again, Bonnell’s journalism background was a potent tool in her advocacy toolbelt.

    This came into sharp focus when Bonnell took her grown daughter Emily to Michigan’s Medicaid board as they decided whether or not to cover a new CF treatment.

    “It wasn’t a drug Emily was ever going to be on; it didn’t work for her CF variant, but we went to the Medicaid review board anyway so she could see how the system worked,” Bonnell recalled. “The room was packed. Doctors making the decision about whether or not to pay for the drug were not even facing the audience. They sat with their backs to the room at a U-shaped table. There was not one doctor on the board with any CF knowledge. I had hoped my daughter would witness the beauty of public advocacy and a Board that listened, but we were all disappointed.”

    Instead, Bonnell recalls Emily crying silently next to her as they listened to the board talk about CF patients as if they were little more than statistics.

    “We heard the board saying, Well, if they don’t take this medication, can we take it away from them?” Bonnell recalled. “It was the sickest, saddest thing I’ve witnessed. You’re talking about people’s lives and life-changing medication.”

    At the time of the hearing, Bonnell was working as a news reporter at WWJ Newsradio in Detroit. She asked her news director if he thought it was a legitimate story. When he said yes, Bonnell went back as a news reporter and asked the Board representative why they would not pay for this CF medication.

    “I truly believe the only reason the Board agreed to pay for the medication was because of all the media attention the hearing received. They felt pressure to pass it.”

    Who cares for the caregiver?

    Despite Bonnell’s tenacity, the strain of motherhood and caregiving for two children with a chronic disease had taken its toll. And when it comes to the impact on caregivers for CF patients, Bonnell’s experience was unique to the space.

    “When Molly and Emily were diagnosed, we were told they must be on a high fat diet because people with CF only digest about half the protein and fat they eat,” Bonnell explained. “We were told to throw butter in everything. And Joe and I were eating the same food. I gained a lot of weight over the years, partly because of the high fat foods, and partly because food was my drug to deal with every emotion.”

    It wasn’t until both Molly and Emily had grown that Bonnell started prioritizing herself.

    Spurred on by the onset of COVID, Bonnell began her journey to good health. “It seemed that overweight people were dying from COVID, and that scared me enough to start my weight loss.” Bonnell said.

    After three years of work, Bonnell lost more than 100 pounds and could recognize herself again.

    Bonnell says she wishes she would have asked for help, or got into mental health therapy sooner, and recommends this to parents with newly diagnosed kids.

    “Surround yourself with friends you can talk to and other CF parents who can relate,” she said.

    This is especially important given the fact that many caregivers are doing a full-time, highly specialized job for free—and then going on to work another, money-making job on top of that. So when it comes to CF caregiving, advocacy, and anything in between, Bonnell reminds that the old adage It takes a village, is important to remember.

    When asked what she wanted those outside of the CF community to know, Bonnell responded, “I think it is just to remember to have some basic human compassion. You never know what someone is going through at the grocery store. They could have the weight of the world on their shoulders.”

    Luckily, the Bonnell Foundation also facilitates a number of ways to get involved, from advocacy to grantmaking to education and beyond. If you are interested in learning more, visit: https://thebonnellfoundation.org/

    The post Cystic Fibrosis Awareness Month: A patient advocate’s story appeared first on Bio.News.

    ]]>     Human Gut Organoids with Functional Nerves Developed that Can Be Mass Produced https://edusehat.com/en/human-gut-organoids-with-functional-nerves-developed-that-can-be-mass-produced https://edusehat.com/en/human-gut-organoids-with-functional-nerves-developed-that-can-be-mass-produced Sat, 23 May 2026 05:05:11 +0700 Edusehat Human, Gut, Organoids, with, Functional, Nerves, Developed, that, Can, Mass, Produced Researchers at Cincinnati Children’s Hospital Medical Center and Nantes Université in France have designed 3D-printed scaffolding trays that will reportedly allow scientists to produce larger versions of functional human gut organoids twice as fast as previous methods—and these organoids grow their own nerve cells.

    This improved technology could help accelerate production of human mini-organ tissues that are large enough to be useful in patching damage or restoring diminished functions of a person’s small intestine, stomach, or colon. Such tissues also would be valuable for future disease studies and to more accurately evaluate organ damage risks linked to oral medications, according to the investigators.

    Details of the study “Large-scale and innervated functional human gut tissues for transplantation via transient spheroid confinement” appear in Nature Biomedical Engineering.

    Using a confined culture system (CCS), the team grew small intestine, colon, and stomach organoids from tiny spherical forms into centimeter-scale tubular forms nearly 10 times larger than previous methods. Also, unlike methods that require a complex effort to introduce nerve cells, these organoids develop a nervous system on their own.

    “By reaching transplantation maturity twice as fast and developing their own functional nerves, these organoids demonstrate how engineering principles can drive biological innovation,” said staff investigator Holly Poling, PhD. “Our confined culture system is more than a production method; it’s a scalable, flexible platform for building complex human tissues.”

    New production system prompts faster growth 

    Experts at Cincinnati Children’s Center for Stem Cell & Organoid Medicine  (CuSTOM) have been making miniature versions of digestive system organs for more than 15 years, working on improving the sophistication of the lab-grown tissues. More recently, the team has been developing methods to make enough customized tissue to transplant into patients to help patch organ damage or restore diminished specialized functions.

    The new technique uses 3D printing technology to make tray-like scaffolding molds from surgical resin, then filling the molds with degassed polydimethylsiloxane—a flexible rubber-like type of silicone.

    The new trays contain grooves designed to confine a collection of sphere-shaped organoids into a row, which encourages the spheroids to fuse together and mature. The fusions occur within a special mix of nutrients and other ingredients that support initial growth from induced pluripotent stem cells (iPSCs) into more complex organoids.

    By day six, the discrete spheroids develop into unified constructs along the grooves of the trays. These are moved into another hydrogel medium for continued growth for another eight days.

    By day 14, the organoid constructs have produced all the cell types and structures that previously required 28 days to achieve. These tissues are then transplanted into rodents that are genetically modified to minimize rejection risk.

    All of the transplanted tissues engrafted in rodents, the co-authors state. After growing in the rodents, the team produced as much as eight cm of functioning small intestine tissue, compared to approximately one cm of tissue using previous protocols. Not only were the structures much larger than previous methods, but now their neuromuscular function was also similar to native human tissue, representing a major advance.

    “We are now able not only to generate complex gastrointestinal organoids at scale, but also to guide their differentiation into functional tissues with integrated enteric neuronal networks,” noted senior author Maxime Mahe, PhD. “By leveraging a defined growth environment, the intrinsic self-organization capacity of the cells drives the formation of tissue structures that closely resemble the human gastrointestinal tract.”

    Researcher looking into microscope
    Researchers have been developing methods to make enough customized tissue to transplant into patients to help patch organ damage or restore diminished specialized functions. [Frazao Studio Latino/Getty Images]
    Jim Wells, PhD, a study co-author and chief scientific director at CuSTOM says the new technology overcomes key barriers to scale and function in organoid research and biomanufacturing.

    “This platform’s simplicity, reproducibility, and versatility make it accessible for widespread adoption,” said Wells. “In addition, the emergence of a self-organized nervous system within these organoids is particularly important for further studies of neurodevelopmental disorders.”

    Another step closer to human clinical trials

    Michael Helmrath, MD, a surgeon-scientist at Cincinnati Children’s who co-directs CuSTOM, has been working for more than a decade to develop intestine organoids sophisticated enough for transplantation in human patients.

    In 2017, Helmrath and colleagues demonstrated how to combine neural crest cells with intestinal tissue cells in a layered process to make the first human organoids with nerve function. His team also showed how intestine organoids could be grown larger by implanting them in a mouse to provide a blood supply. Ever since, intestine organoids have been getting more sophisticated, including versions with immune cells in addition to the specialized organ cells and nerves.

    Now the new process—involving rats instead of mice—produces larger amounts of tissue.

    “It is still not possible to grow complete, full-sized human organs in some sort of tank, but research like this has produced significant amounts of tissue that can be matched directly to individual patients,” explains Helmrath. “We believe such tissues, once transplanted, would further grow and multiply as part of the patient’s own organ to restore functions.”

    More research and development is needed before “CCS organoids” will be ready for human clinical trials, according to Helmrath. But if successes continue, organoid medicine may allow more infants and children with dysfunctional organs to be treated without ever needing a full organ transplant.

    The post Human Gut Organoids with Functional Nerves Developed that Can Be Mass Produced appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Skape Bio Unlocks Generalizable GPCR Drugs Using AI Protein Design https://edusehat.com/en/skape-bio-unlocks-generalizable-gpcr-drugs-using-ai-protein-design https://edusehat.com/en/skape-bio-unlocks-generalizable-gpcr-drugs-using-ai-protein-design Sat, 23 May 2026 05:05:09 +0700 Edusehat Skape, Bio, Unlocks, Generalizable, GPCR, Drugs, Using, Protein, Design The year was 2022. Chris Norn, PhD, was wrapping up his time as a postdoctoral researcher at the Institute for Protein Design (IPD) at University of Washington (UW). AlphaFold was taking the field by storm, while a new generation of deep learning tools was rapidly advancing de novo, or from-scratch, protein design with unprecedented success rates validated at atomic resolution. Within just a few years, these AI breakthroughs, widespread applications across pharmaceuticals, nanomaterials, biosensors, and more, would help earn Norn’s mentor, David Baker, PhD, the Nobel Prize in Chemistry.  

    “There’s so much dark space in biology. The precision of protein design was becoming incredible.” said Norn in an interview GEN. “Designing function from scratch is going to be incredibly impactful for treating diseases.” 

    Norn’s research investigated the subtle structural differences that caused G-protein-coupled receptors (GPCRs) to change conformation from a healthy state to disease driver. These integral membrane proteins are the largest protein family encoded by the human genome and represent approximately one-third of drug targets, across cancer, metabolic disease, and neurological disorders. Yet, they are traditionally difficult to hit because their accessible regions barely protrude from the cell membrane. 

    Today, Norn is co-founder and CEO of Skape Bio, a Copenhagen-based AI protein design company building a generalizable platform to target underexplored GPCRs and treat diseases once deemed undruggable. The team has published a new study in Nature demonstrating the design of functional miniproteins that target 11 GPCRs across a diversity of receptor families implicated in itch and pain, cancer, metabolic disorders, and migraine, with examples that penetrate deeply into hard-to-reach GPCR pockets. Notably, agonists were validated against three targets. 

    In a key example, the study designed a chemokine receptor antagonist that mobilizes hematopoietic stem and progenitor cells in a mouse model at a level comparable to a clinically used drug, with fewer side effects.  

    At the core of Skape Bio’s technology stack is a proprietary high-throughput platform that screens GPCRs directly within their native membrane environment, enabling accurate measurement of how conformational changes influence cell signaling and function. The approach represents a significant advance over traditional screening methods, which remove GPCRs from their membrane-embedded context and can fail to capture native structural dynamics. Over 100,000 miniprotein designs can be screened per target on a single-platform campaign. 

    Edin Muratspahić, PhD, postdoctoral research scholar at UW and co-corresponding author of the Nature study, highlights that the rise of de novo models, such as Baker lab’s RFdiffusion, has fueled the growing momentum for protein-based GPCR drugs. Compared to small molecules, protein therapeutics offer high selectivity, protease stability, and extended half-life. Notably, the small size of miniproteins allows better tissue penetration compared to antibodies. 

    “Many GPCRs remain underexplored because we didn’t have the tools to look at their pharmacology,” Muratspahić told GEN. “We’re excited to illuminate new biology beneficial to developing better and safer protein-based therapeutics.” 

    The post Skape Bio Unlocks Generalizable GPCR Drugs Using AI Protein Design appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Enzymes Involved in Cholesterol Transport May Point to New Cancer Therapies https://edusehat.com/en/enzymes-involved-in-cholesterol-transport-may-point-to-new-cancer-therapies https://edusehat.com/en/enzymes-involved-in-cholesterol-transport-may-point-to-new-cancer-therapies Sat, 23 May 2026 05:05:08 +0700 Edusehat Enzymes, Involved, Cholesterol, Transport, May, Point, New, Cancer, Therapies Some types of cancer have a relentless appetite for the metabolite cholesterol, using as much as they can access to accelerate their growth beyond the capabilities of normal cells. Research by scientists at Sanford Burnham Prebys Medical Discovery Institute and collaborators at the University of Illinois Chicago have now unveiled a potential method for turning the table on these tumors by subverting their cholesterol cravings.

    The researchers’ studies, in mice and in human cancer cells, revealed new insights into enzymes known as phosphatidylinositol 5-phosphate 4-kinases (PI5P4Ks) that help move cholesterol around cells. The researchers showed that without the help of these enzymes, a cholesterol traffic jam occurs, blocking the cancer cell’s ability to fuel tumor growth.

    Headed by Brooke Emerling, PhD, the director of and associate professor in the Cancer Metabolism and Microenvironment Program at the Sanford Burnham Prebys NCI-Designated Cancer Center, the team reported on its findings in Science Advances, in a paper titled “Noncanonical PI(4,5)P2 coordinates lysosome positioning through cholesterol trafficking.”

    The TP53 gene is mutated in roughly half of all cancers. Emerling and first author Ryan Loughran, PhD, a postdoctoral associate in the Emerling lab, focus on difficult-to-treat forms of breast cancer, where TP53 mutations are found in more than 84% of triple-negative breast cancers and three of every four HER2-amplified breast cancers.

    Cancer cells with a mutation in the tumor-suppressing TP53 gene are known to produce extra cholesterol. This may make them more vulnerable to starvation if scientists can put a stop to the steady supply of the lipid. “We need more ways to treat cancers with this common mutation,” said Emerling. “One of our main goals with this work was to find new treatment possibilities for the large subset of breast cancers harboring TP53 mutations,” said Loughran. “We recognized a real opportunity in targeting the enzymes that control cholesterol transport, especially since cancer cells depend on this process far more than normal cells do.”

    To better understand how to turn these cancers’ cholesterol consumption into a weakness, the research team turned to a family of cell membrane lipids known as phosphoinositides and the kinase enzymes that regulate them. The investigators had shown that a branch of the lipid enzyme family known as PI5P4Ks were required for the growth of cancers with TP53 mutations in mice, and they suspected that this tumor prevention was due to the enzymes’ role relocating cholesterol in the cell. “Our group has shown that suppression of the most catalytically active PI5P4K isoforms (α and β) in TP53-deficient cancer cells inhibits proliferation, and the deletion of these enzymes in Trp53-knockout mice confers protection from tumorigenesis,” the investigators wrote.

    “Normally, when mice lose TP53 as the guardian of their genomes, they are fated to die from cancer in four-to-eight months,” said Emerling. “When you delete these kinases, the animals are 100% protected and never develop a tumor—and cholesterol turned out to be one of the missing pieces in this puzzle.”

    The scientists conducted experiments in mouse and human cancer cells showing that PI5P4Ks influenced the movement and behavior of organelles that carry cholesterol around our cells. In cancer cells with TP53 mutations and PI5P4Ks, cholesterol-laden lysosomes were found near the exterior cell membrane. Without PI5P4Ks, lysosomes remained in the interior of the cells, near the nucleus.

    Location is critical for lysosomes transporting cholesterol. While positioned near the edge of the cell, lysosomes and their cargo are in proximity with many receptor proteins, enzymes and signaling molecules that exist around the cell membrane. This includes mechanistic target of rapamycin complex 1 (mTORC1), an enzyme that governs cell growth and runs amok in cancer. “When lysosome positioning is biased towards the cell nucleus, mTORC1 activation is suppressed,” said Loughran. “This connects directly to our previous work, where we found that the loss of these kinases triggers starvation-like states in cancer cells. “When PI5P4Ks are absent, the link between lysosomal cholesterol and mTORC1 is compromised, a bit like two ships passing in the night.”

    The change in lysosome position towards the cell’s interior that occurs without PI5P4Ks reduced interaction with mTORC1 and prevented it from sending signals associated with tumor growth. “The mTOR activation pathway is really what drives tumorigenesis, and so mTOR is an important target for cancer drug development,” said Emerling. “If we can target mTOR activity in aggressive cancers by blocking the sensing of cholesterol, that would be a promising treatment strategy.”

    In their report the authors noted in summary, “The dependence of p53-deficient tumor cells on PI5P4Ks has been previously attributed to their roles as critical modulators of cellular stress responses, including protection from oxidative stress, maintenance of mitochondrial health, and regulation of autophagy. We now identify a previously undescribed role for PI5P4Ks in maintaining lysosomal cholesterol homeostasis and mTORC1 signaling.”

    Previous research has looked at the use of statins as cancer drugs due to their ubiquity and safety as treatments for patients with high cholesterol. While more research is needed, studies so far suggest that tumors eventually acquire resistance to statins. “While cholesterol synthesis inhibitors such as statins have shown initial success, their efficacy is often compromised by the development of acquire resistance,” the team noted in the paper. “Consequently, strategies are being explored to disrupt cholesterol homeostasis more comprehensively by inhibiting its synthesis and intracellular transport.”

    Loughran added, “It is important for us to find other ways to more comprehensively cut cancer cells off from cholesterol to impede their growth.” Emerling further stated, “We’ll continue to explore blocking PI5P4Ks as a more targeted approach tailored to how tumors operate.”

    The post Enzymes Involved in Cholesterol Transport May Point to New Cancer Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Parabilis Files for IPO, a Day After Signing Up-to-$2.3B Regeneron Collaboration https://edusehat.com/en/parabilis-files-for-ipo-a-day-after-signing-up-to-23b-regeneron-collaboration https://edusehat.com/en/parabilis-files-for-ipo-a-day-after-signing-up-to-23b-regeneron-collaboration Sat, 23 May 2026 05:05:07 +0700 Edusehat Parabilis, Files, for, IPO, Day, After, Signing, Up-to-2.3B, Regeneron, Collaboration Parabilis Medicines, the developer of drugs and antibody-drug conjugates targeting historically undruggable protein targets and based on stabilized helical peptides or Helicons™, has filed for an initial public offering (IPO), joining a growing parade of companies seeking to raise capital by tapping into the improving market for first time biotech stocks.

    The company’s IPO filing came a day after Parabilis inked an up-to-$2.3 billion-plus strategic research collaboration with Regeneron Pharmaceuticals to discover and develop an initial five candidates encompassing “antibody-Helicon conjugates,” a new form of antibody-drug conjugates aimed at challenging and historically undruggable targets.

    Regeneron has agreed to purchase approximately $75 million of Parabilis common stock in a concurrent private placement, at 90% of the IPO price per share.

    It’s too soon to know how much money Parabilis plans to raise through the IPO. The company’s Form S-1 registration statement, filed Tuesday with the U.S. Securities and Exchange Commission (SEC), includes a placeholder “$100 million” figure that will inevitably be revised, and doesn’t say how many shares will be sold. Parabilis has applied to list its shares on The Nasdaq Global Market under the ticker symbol “PBLS.”

    It’s also too early to know how much of the proceeds will go toward the four priorities it highlighted in its registration statement. Two of the four priorities relate to Parabilis’ lead Helicon peptide candidate zolucatetide (formerly FOG-001), a first and only direct inhibitor of the elusive β-catenin:TCF interaction, according to the company. Parabilis stated that it plans to continue ongoing clinical development of zolucatetide in desmoid tumors, including continuation of dose expansion and the launch of a Phase III registrational trial to topline data.

    Parabilis also plans to continue ongoing clinical development of zolucatetide across several additional indications, including dose escalation and expansion in familial adenomatous polyposis (FAP); hepatocellular carcinoma, the most common type of primary liver cancer; and other rare tumors, with the aim of collecting data to support a registrational trial.

    ‘Expansive opportunity’

    “We believe zolucatetide provides clinical validation of our first-in-industry Helicon approach and represents an expansive opportunity for medical and commercial impact,” Parabilis stated.

    In addition, Parabilis plans to use IPO proceeds toward advancing its pipeline of additional programs—including its ERG protein degrader, an allosteric androgen receptor in its active state (ARON), and beta-catenin degraders—to Phase I clinical data; toward continued evolution of the Helicon platform for discovering and developing drug candidates; as well as toward general corporate purposes that include additional development efforts, working capital, and operating expenses.

    According to Parabilis, zolucatetide has been evaluated in more than 150 patients to date and has generated positive clinical data in solid tumors characterized by alterations in the Wnt/beta-catenin pathway. In the drug’s lead indication of desmoid tumors, researchers have seen tumor reductions in 100% of patients with a 74% objective response rate (ORR) in patients who have had at least two post-baseline scans.

    In March, Parabilis presented preliminary clinical data at the 11th Biennial Meeting of the International Society for Gastrointestinal Hereditary Tumors (InSiGHT) showing significant improvement in duodenal polyposis at 60 weeks in a patient with familial adenomatous polyposis (FAP) treated with zolucatetide in the company’s ongoing Phase I/II trial (NCT05919264).

    The patient showed a 52.2% reduction in desmoid tumor diameter, as well as “substantial” reductions in polyp number and size compared with a pre-treatment evaluation nearly two years prior, consistent with downstaging from Spigelman stage II to stage I.

    Parabilis says its Helicon discovery platform allows it to precisely tune potency, selectivity, and pharmacologic properties by integrating ligands and additional functionalities at multiple positions. The platform integrates artificial intelligence (AI)- and physics-based computational modeling with high-throughput peptide synthesis and experimental screening.

    “While our initial programs are focused on disrupting protein-protein interactions and inducing targeted protein degradation, we believe our platform can incorporate other advances in small molecule drug design and extend them to targets that are likely to remain out of reach for other modalities,” Parabilis stated.

    Parabilis was founded in 2015 as FogPharma to commercialize technology developed in and inlicensed from the lab of Harvard University researcher and serial entrepreneur Gregory Verdine, PhD. The company, which rebranded itself into Parabilis in 2024, says it has generated proprietary datasets, comprising millions of data points for hundreds of thousands of Helicons across dozens of drug-like properties, following a decade of Helicon drug discovery.

    ‘Continuous learning loop’

    “These data power a continuous learning loop that refines our models from target selection through lead optimization, enhancing our speed, precision, and ability to generate high quality molecules against difficult targets,” Parabilis explained. “As a result, our platform produces unique complex synthetic molecules at scale and a compounding advantage that we believe is difficult to replicate.”

    If it carries out the planned IPO, Parabilis would be the 12th biotech to go public this year. The 11 IPO companies to date have raised a combined $3.491 billion, compared to the $1.556 billion raised by 11 companies this time last year. Six of the 11 companies have seen their shares rise since their initial offerings, led by the 520% share price increase of Veradermics, a developer of treatments for dermatology and aesthetic conditions that closed Wednesday at $105.32 a share.

    This year’s largest IPO—and the largest of any biotech—was the $625 million offering of Kailera Therapeutics, a developer of therapies for obesity and weight management based on glucagon-like peptide receptor 1 (GLP-1) agonists, alone or in combination with glucose-dependent insulinotropic polypeptide (GIP) receptor agonists.

    Kailera last month completed what grew into a $718.75 million IPO last month that generated an estimated $662.1 million in net proceeds through the sale of 44,921,875 shares of common stock—including the exercise in full by underwriters of their option to purchase 5,859,375 additional shares—at the IPO price of $16 per share.

    Should Parabilis’ planned IPO raise the placeholder $100 million amount, it would increase by 30% the $329.039 million in cash and cash equivalents that the company reported as of March 31.

    Parabilis disclosed in its IPO filing that it ended the first quarter with a $45.316 million net loss, up 18% from its $38.342 million net loss of Q1 2025—as well as a net loss of $145.9 million for last year, up nearly 24% from its $117.9 million net loss for 2024. The company has no reported revenue.

    Parabilis’ accumulated deficit rose 8% during Q1, to $586.82 million from $541.504 million at the end of 2025.

    To fund its operations, Parabilis reported, it has raised a total $876.8 million as of March 31. That total consisted of $811.8 million from sales of its convertible preferred stock, $15 million in borrowings under a term loan and a $50 million Simple Agreement for Future Equity (SAFE).

    The IPO comes just four months after Parabilis completed its last financing, an oversubscribed $305 million Series F round completed in January and co-led by investment firms RA Capital Management, Fidelity Management & Research Co., and Janus Henderson Investors.

    Five investment firms were listed in the Form S-1 as underwriters for the IPO: Leerink Partners, BofA Securities, Evercore ISI, Guggenheim Securities, and LifeSci Capital.

    The post Parabilis Files for IPO, a Day After Signing Up-to-$2.3B Regeneron Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AI Scientists, Stock Tumble, Patent Lawsuit, and New Partnerships https://edusehat.com/en/ai-scientists-stock-tumble-patent-lawsuit-and-new-partnerships https://edusehat.com/en/ai-scientists-stock-tumble-patent-lawsuit-and-new-partnerships Sat, 23 May 2026 01:30:09 +0700 Edusehat Scientists, Stock, Tumble, Patent, Lawsuit, and, New, Partnerships Agentic AI is growing in its applications. Google DeepMind and Edison are leveraging the growing capabilities by developing AI Scientists. These platforms are poised to streamline the scientific process, aiding human scientists with a variety of tasks. Meanwhile, despite positive data in its Phase III DMD therapy trial, Regenxbio’s stock fell for a variety of reasons. 10X Genomics and Harvard University are suing Element Biosciences over patents for a multiomics platform. Finally, Bristol Meyers Squibb is partnering with Hengrui Pharma to develop 13 early-stage programs with the potential to grow their investment to a predicted $15 billion in sales.

    Listed below are links to the GEN stories referenced in this episode of Touching Base:

    The State of Precision Medicine Summit
    Join us June 3, 2026

    Google DeepMind and Edison Are Building the AI Scientist
    By Fay Lin, PhD, GEN Edge, May 19, 2026

    StockWatch: Regenxbio Tumbles Despite Positive Pivotal Data for DMD Gene Therapy Candidate
    By Alex Philippidis, GEN Edge, May 17, 2026

    10x Genomics, Harvard Target Element’s Multiomics Platform in Patent Lawsuit
    By Alex Philippidis, GEN Edge, May 12, 2026

    BMS, Hengrui Pharma Partner on 13 Programs in Up-to-$15.2B Collaboration
    By Alex Philippidis, GEN Edge, May 13, 2026

    Touching Base Podcast
    Hosted by Corinna Singleman, PhD

    Behind the Breakthroughs
    Hosted by Jonathan D. Grinstein, PhD

    The post AI Scientists, Stock Tumble, Patent Lawsuit, and New Partnerships appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     The Enhanced Games fit right in with the rest of 2026’s longevity vibes https://edusehat.com/en/the-enhanced-games-fit-right-in-with-the-rest-of-2026s-longevity-vibes https://edusehat.com/en/the-enhanced-games-fit-right-in-with-the-rest-of-2026s-longevity-vibes Fri, 22 May 2026 18:25:02 +0700 Edusehat The, Enhanced, Games, fit, right, with, the, rest, 2026’s, longevity, vibes
  • Drugs are the point: The inaugural Enhanced Games, held in Las Vegas this Sunday, openly encourages its 42 athletes to use performance-enhancing drugs — provided they're FDA-approved and medically supervised — with $1 million on offer for world records broken.

  • FDA-approved doesn't mean risk-free: Anabolic steroids, growth hormones, and other permitted substances carry serious health risks, including liver tumors, diabetes, and vision problems.

  • It fits the moment perfectly: From peptide clinics to optimized embryos, the Enhanced Games reflect a broader cultural obsession with pushing past human limits — one where just being human isn't enough anymore
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    This Sunday, a group of 42 athletes will gather in Las Vegas to compete in a somewhat unusual sporting competition. Participants in the inaugural Enhanced Games are being encouraged to take performance-enhancing drugs. The goal is to “push the boundaries of human performance.”

    The games’ organizers have said that competitors will only be taking substances that have been approved by the US Food and Drug Administration, and that they are all being medically monitored and supervised. But they have also said they expect to see world records broken—and are offering substantial prizes to athletes who succeed in doing so.

    As you might expect, the event is generating a mix of curiosity, excitement, and condemnation from various quarters. To me, it feels like very much a reflection of where we are today—an era of peptide-crazed looksmaxxing in which consumers are being encouraged to get thinner than ever, optimize for longevity, and have their “best baby.” It’s 2026, and if you’re not enhancing, what are you even doing?

    So, these games. They’ll feature competitions in four categories: swimming, track and field, weightlifting, and strongman (which also involves lifting weights). Many of the competitors already hold national and world records, and some are Olympic medalists. They’ve been paid a salary and will compete for prizes from a $25 million pot. The money has been a major draw for at least some of the athletes.

    Another draw is the opportunity to openly experiment with drugs that might boost their performance. In the world of elite sport, every microsecond and every millimeter counts. Athletes—most of whom arguably have genetics on their side already—follow meticulous diet, training, and recovery protocols and wear specially designed gear that allows them to reach for those performance bests.

    But within most sporting communities, there are limits. The World Anti-Doping Agency—an international outfit that fights the use of drugs in sports—maintains a lengthy list of “non-approved substances” that are banned in international sporting events. It features many anabolic steroids (which can build muscle), hormones (such as those that stimulate testosterone production or increase the ability of blood to carry oxygen), growth factors (which can stimulate muscle growth and repair, among other things), and more.

    Some of these substances have been FDA approved to treat health disorders. And that means they can be used by participants in the Enhanced Games, according to the organization’s rules.

    I’ll briefly point out the obvious here—just because a drug has been approved by the FDA doesn’t mean it’s totally safe for everyone and anyone. The risks associated with use of anabolic steroids, for example, include high blood pressure, acne, depression, and liver tumors. Growth hormone use can cause weak muscles, affect vision, and even lead to diabetes.

    “Technological doping,” or using improved equipment to gain advantage, has also been supported by the games’ organizers. Last year, participating swimmer Kristian Gkolomeev was reported to have broken a record in a 50-meter freestyle time trial while wearing a polyurethane “super” swimsuit. Such suits have been banned for use in the Olympics since a slew of record-breaking performances in 2008 and 2009. Back then, the swimming governing body ruled that they gave athletes an unfair advantage. But hey, this is the Enhanced Games, where the word “unfair” seems to have a completely different meaning.

    Can we expect more records to be broken on Sunday? Maybe. In addition to prize money for winning an event, any athlete who manages to beat a record stands to win up to $1 million, the sum also awarded to Gkolomeev last year following his time trial. But those performances won’t be recognized by official sporting bodies.

    Plenty of concerns have been raised about these games. Some argue that they are unsafe and promote risky drug use. Others see them as a “clown show,” and a slap in the face to “clean” athletes who train hard without the use of prohibited drugs. World Athletics president Sebastian Coe has said that anyone who takes part is “moronic,” and World Aquatics, which oversees international competitions in water sports, has banned Enhanced Games participants from its events and activities.

    But. The games—and the participating athletes—will still get a huge amount of attention. As a result, so will performance-enhancing drugs. Enhanced, the company behind the games, also runs an online store. There, you can buy a $52 T-shirt emblazoned with the message “I am Enhanced.”

    There is also a range of prescription drugs on offer, including peptides “to support recovery, vitality, and longevity.” One of these is a growth hormone that the FDA approved in 1997 for the treatment of children with “growth failure.” The compounded version offered on the Enhanced website, which is not FDA approved, is marketed for longevity, supporting deep sleep and “overall wellness and vitality.” (“Marketed” is the key word here. The drug has, again, not been approved for that purpose.)

    It all fits very well with the zeitgeist. Sure, we don’t yet have any drugs that are designed to extend human lifespan. But the search for anti-aging drugs is getting more attention—and funding—than ever. People, particularly women, are seemingly not allowed to visibly age anymore—we have filters and facelifts for that now. The idea that “death is wrong” is gaining acceptance.

    And self-experimentation is rife. “Biohacking” was shortlisted for Collins Dictionary’s Word of the Year in 2025. Peptides are everywhere, despite all the unknowns surrounding their safety and effectiveness. So are longevity clinics, despite the fact that most are selling unproven treatments. US states like Montana are making it easier for people to get hold of unapproved “therapies.”

    Companies are even offering would-be parents the option to choose the potential future children expected to live longest. Yep—you can supposedly optimize your embryos now, too.

    In this climate, the Enhanced Games don’t feel so radical. They feel entirely fitting for our era of questionable optimization despite the risks —an era when, apparently, being human is no longer enough.

    ]]>     AI Designs Miniprotein Switches for GPCR Targeting https://edusehat.com/en/ai-designs-miniprotein-switches-for-gpcr-targeting https://edusehat.com/en/ai-designs-miniprotein-switches-for-gpcr-targeting Fri, 22 May 2026 11:10:10 +0700 Edusehat Designs, Miniprotein, Switches, for, GPCR, Targeting Many scientists first encountered G protein–coupled receptors (GPCRs) as a looping sketch across the cell membrane in an early biology textbook. That simple diagram belied the complexity of a receptor family now known to govern vision, smell, hormone sensing, and the actions of countless medicines. Yet despite their centrality, designing molecules that can precisely switch GPCRs on or off has remained one of drug discovery’s most persistent challenges.

    A new study led by the UW Medicine Institute for Protein Design and Skape Bio demonstrates that AI‑driven de novo protein design can finally meet that challenge. The work, published recently in Nature, shows that computationally designed miniproteins—compact proteins under 100 amino acids—can be engineered to either activate or block GPCRs with high affinity, potency, and selectivity. The paper is titled “De novo design of miniproteins targeting G protein-coupled receptors.”

    The research team developed a suite of design strategies to create miniproteins capable of slipping into the deep, flexible pockets that govern GPCR signaling. These pockets shift shape depending on whether the receptor is active or inactive, making them difficult to target with conventional biologics. By designing molecules that recognize specific receptor states, the team generated agonists for receptors involved in itch and pain, and antagonists for receptors implicated in cancer, metabolic disease such as diabetes and obesity, and migraine.

    GPCR microprotein
    A tiny protein (pink) designed on a computer fits into a deep pocket (inset) of a cell surface receptor called a GPCR (blue), allowing scientists to switch cell signaling on or off. [Edin Muratspahić/UW Medicine Institute for Protein Design]
    “Protein design takes our understanding of how proteins fold and reverses it—asking if we can envision, with the aid of AI computing, a new protein that sticks to a target in a purpose-built way,” said senior author David Baker, PhD, director of the Institute for Protein Design, professor of biochemistry at the University of Washington School of Medicine, and a Howard Hughes Medical Institute Investigator. “This paper showcases how we can do this repeatedly for different GPCRs in ways that capitalize on their dynamic motion to either activate or inactivate them.”

    Cryo‑EM structures of five designed miniproteins closely matched their computational models, underscoring the accuracy of the design pipeline. In one mouse study, a designed chemokine‑receptor antagonist mobilized hematopoietic stem and progenitor cells at levels comparable to a clinically used drug—but with fewer side effects, according to the authors.

    For first author Edin Muratspahić, PhD, the moment of validation came when the designed molecules did more than bind. “Seeing computationally designed miniproteins not only bind but actually control GPCR signaling in living cells was a defining moment for me,” he said.

    A second major advance reported in the study is a high‑throughput “receptor diversion” screening system that evaluates tens of thousands of designed proteins directly in living human cells. Traditional GPCR screens often require purifying or stabilizing receptors—steps that can distort their natural signaling behavior. By keeping receptors in their native membrane environment, the new system accelerates discovery while preserving biological relevance.

    According to corresponding author Christoffer Norn, PhD, co‑founder of Skape Bio, the study lays out a roadmap for all‑computational design of GPCR ligands.

    The methods described in the paper are already being adapted at Skape Bio to explore GPCR targets involved in metabolic, inflammatory, and neurologic pathways—areas where conventional discovery efforts have often struggled.

    The post AI Designs Miniprotein Switches for GPCR Targeting appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Wacker Expands Service Offerings with Launch of Contract Research for Nucleic Acid-Based Therapies https://edusehat.com/en/wacker-expands-service-offerings-with-launch-of-contract-research-for-nucleic-acid-based-therapies https://edusehat.com/en/wacker-expands-service-offerings-with-launch-of-contract-research-for-nucleic-acid-based-therapies Fri, 22 May 2026 04:00:12 +0700 Edusehat Wacker, Expands, Service, Offerings, with, Launch, Contract, Research, for, Nucleic, Acid-Based, Therapies Wacker reports that it is launching contract research services (CRS) at its biotech center in Munich for R&D-grade pDNA, RNA, and LNPs for preclinical studies. The new offering complements services of subsidiary CDMO Wacker Biotech, which has sites in Germany, the Netherlands, and the U.S.

    In addition to producing pDNA, RNA, and LNP formulations, Wacker’s CRS team says it offers construct design services, including plasmid and RNA construct design as well as RNA engineering and optimization via partners, e.g., UTR, poly(A) and cap optimization. Company scientists also conduct lipid library screening and lipid nanoparticle formulation and provide functional assays and analytical services.

    By integrating early-stage R&D support with a globally interconnected GMP manufacturing network, CRS helps customers streamline development and reduce supply-chain fragmentation, maintains a Wacker spokesperson. The approach enables an accelerated path from design to delivery in the field of advanced therapies, while lowering risk and cost through resourcing in early phases and a scalable transfer to Wacker Biotech for clinical material, continued the company official.

    “Every RNA or LNP project is unique. Our goal is to provide flexible, customizable services that adapt to our clients’ specific needs in a rapidly evolving landscape,” explained Christian Dubiella, the CRS global program manager. “Too often, innovative, potentially life-saving therapeutic concepts die on the vine due to the high cost of developing even small amounts of drug substance for R&D studies. Our CRS enable us to serve highly specialized customers, especially small startups.”

    One of CRS’ first customers is SRTD Biotech, an emerging biotech in Germany, which is starting small scale on novel therapeutic approaches.

    “Our platform technology based on seRNAs (selectively expressed RNAs) can easily be adapted to numerous therapeutic areas by utilizing the transcriptome for selective cell targeting and fusogenic LNPs for organ-specific targeting,” said Bernd Hoffmann, CEO/CSO and cofounder of SRTD. “Wacker is an ideal partner on the road to realizing our vision of delivering seRNAs to patients to improve their lives.”

     

    The post Wacker Expands Service Offerings with Launch of Contract Research for Nucleic Acid-Based Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cytokine‑Armored CAR T Cells Overcome Antigen Heterogeneity in Glioma Model https://edusehat.com/en/cytokinearmored-car-t-cells-overcome-antigen-heterogeneity-in-glioma-model https://edusehat.com/en/cytokinearmored-car-t-cells-overcome-antigen-heterogeneity-in-glioma-model Fri, 22 May 2026 04:00:11 +0700 Edusehat Cytokine‑Armored, CAR, Cells, Overcome, Antigen, Heterogeneity, Glioma, Model Scientists at the UCLA Health Jonsson Comprehensive Cancer Center have developed a cytokine-armored CAR T-cell therapy that helps the immune system better attack aggressive brain tumors in mice. Their study showed that the treatment also reduced dangerous side effects that have long limited immune-based treatments for glioblastoma, which is one of the deadliest and most treatment-resistant brain cancers.

    The therapy works by reprogramming CAR T cells to release immune-stimulating proteins, IL-12 and DR-18, which activate the body’s own immune system, strengthening the overall anticancer response. This treatment approach improved tumor control in mouse models, including those carrying cancers made up of mixed cell populations that often escape treatment. Researchers also found that pairing the treatment with a second CAR T strategy targeting VEGF helped reduce side effects while preserving strong anti-tumor activity.

    The findings point to a potential new strategy for treating recurrent high-grade gliomas and other solid tumors that historically have been difficult to target with CAR T-cell therapy. Research lead Yvonne Chen, PhD, co-director of the Tumor Immunology and Immunotherapy Program at the UCLA Health Jonsson Comprehensive Cancer Center, is senior author of the study, which is published in Cancer Research, in a paper titled “Armored Chimeric Antigen Receptor-T Cell Therapy Targets Antigen-Heterogeneous Glioma.”

    Glioblastoma remains extremely difficult to treat because tumors suppress immune responses, contain diverse cancer cells, and create abnormal blood vessels that limit the effectiveness of immunotherapy. “Two features of glioblastoma pose formidable barriers to effective immunotherapy: tumor-antigen heterogeneity and a highly immunosuppressive tumor microenvironment (TME),” the team wrote. While CAR T-cell therapy has transformed treatment for certain blood cancers, success in solid tumors has been limited.

    “Early data from clinical evaluation of chimeric-antigen receptor (CAR) T-cell therapies for glioblastoma show a strong safety profile and promising signs of response, but durable efficacy remains elusive,” they continued. Chen added, “A key challenge in treating brain tumors, particularly glioblastoma, is that the tumor cells are often antigen heterogeneous, meaning they do not all express the same proteins that can be recognized by a given targeted therapy.” The researchers further stated, “The glioblastoma TME is characterized by a variety of dysfunctional tumor-associated cell types that support tumor growth and metastasis.” The most abundant of these are tumor-associated macrophages (TAMs), which can directly suppress immune-cell function and promote tumorigenesis.

    “We hypothesized that effective immunotherapy against brain tumors would have to engage naturally occurring immune cells, which can recognize a wide variety of target antigens, in the fight against cancer,” Chen noted.

    Because brain tumors are considered immunologically cold, meaning they do not naturally trigger a strong immune response, the researchers designed “armored CAR T cells” to activate immunity against the tumor. These CAR T cells were built to recognize a tumor antigen called IL-13Rα2, a protein commonly found on glioblastoma cells, while also secreting immune-stimulating proteins that recruit and activate the body’s immune cells.

    The team then tested multiple combinations of these “armor” molecules in immunocompetent mouse models of glioblastoma, using head-to-head comparisons to evaluate how each design affected tumor growth and immune activity. The CAR T cells were studied in several orthotopic glioma models, including tumors engineered to vary in antigen expression to better reflect the heterogeneity seen in human disease. After testing multiple combinations, researchers identified one especially potent pairing: IL-12 and decoy-resistant IL-18 (DR-18). “Through head-to-head in vivo comparisons of potentially synergistic armor combinations, we demonstrated that T cells expressing a CAR plus IL-12 and the decoy-resistant form of IL-18 (CAR-12.DR18 T cells) show strong efficacy against antigen-heterogeneous glioma in immunocompetent mice,” the investigators reported.

    The team showed that the therapy demonstrated the ability to eliminate tumors containing cancer cells that lacked the target recognized by the CAR T cells, a major hurdle in glioblastoma treatment because tumors can evolve and escape single-target therapies. “IL-12 and DR-18 work synergistically to activate the immune system, resulting in a dramatic influx of immune cells into the tumor-bearing brain,” stated Chen, who is also a professor of microbiology, immunology, and molecular genetics at UCLA and a member of the UCLA Broad Stem Cell Research Center. “The diverse immune-cell population recruited into the brain contributes to attacking the tumor, including ones that cannot be directly recognized by the CAR T cells themselves.”

    Because IL-12 can trigger dangerous inflammation, the researchers also explored ways to reduce side effects while maintaining anti-tumor activity. They found that adding a second engineered CAR T approach targeting VEGF—a protein that drives abnormal blood vessel growth and contributes to swelling in glioblastoma—helped reduce treatment-related toxicity while maintaining strong tumor control in mice. “Robust anti-tumor efficacy with effective toxicity mitigation was achieved via combined administration of CAR-12.DR18 T cells with CAR T cells that secrete an anti-vascular endothelial growth factor (VEGF-A) single-chain variable fragment. This combination therapy presents a clinically applicable strategy to overcome key barriers to effective treatment of glioblastoma,” the authors stated.

    “When developing novel therapies, we always have to balance considerations for safety and efficacy,” Chen said. “Potent cytokines such as IL-12 and DR-18 have toxicity potential, which is why we performed in-depth studies to understand the nature and severity of the toxicity and devised ways to counteract safety concerns while maintaining anti-tumor activity.”

    The findings point to a potential new strategy for treating recurrent high-grade gliomas. The researchers are now completing the necessary preclinical studies and raising funds to launch a Phase I clinical trial in patients with the disease.

    “We are very encouraged by the ability of our cytokine-armored CAR T cells to kill not only tumor cells that express IL-13Rα2, but also tumor cells that are not directly recognizable to the CAR T cells,” Chen said. “We are excited to have developed a clinical protocol that would allow us to bring this therapy to the clinic while also providing a detailed toxicity management plan to ensure patient safety.”

    The post Cytokine‑Armored CAR T Cells Overcome Antigen Heterogeneity in Glioma Model appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AI Model Offers Map of How Genes Work Together in Different Cellular Contexts https://edusehat.com/en/ai-model-offers-map-of-how-genes-work-together-in-different-cellular-contexts https://edusehat.com/en/ai-model-offers-map-of-how-genes-work-together-in-different-cellular-contexts Fri, 22 May 2026 04:00:11 +0700 Edusehat Model, Offers, Map, How, Genes, Work, Together, Different, Cellular, Contexts Scientists at the Icahn School of Medicine at Mount Sinai have created a new artificial intelligence (AI) model that helps reveal how genes function together inside human cells, offering a powerful new way to understand biology and disease. Their study, headed by Avi Ma’ayan, PhD, professor of pharmacological sciences and director of the Mount Sinai Center for Bioinformatics at the Icahn School of Medicine at Mount Sinai, introduces a gene set foundation model (GSFM) designed to learn patterns in how genes are grouped and function across thousands of biological contexts.

    The work draws inspiration from advances in large language models (LLMs) such as ChatGPT, which learn how words gain meaning depending on their context. In a similar way, a GSFM learns how genes behave differently depending on their cellular “context.”

    The model provides a new way to understand the structural and functional organization of genes and their products inside human cells. This improved understanding could eventually support the development of better diagnostics, biomarkers, and therapies. By mapping how genes relate to one another across many biological situations, the GSFM creates a reference framework that can help scientists interpret complex multiomics datasets more effectively, say the investigators. “The organization of genes within cells remains one of the major unsolved questions in biology,” Ma’ayan noted. “The GSFM helps address this by learning from millions of gene groupings derived from published research and gene expression datasets.”

    Ma’ayan is senior corresponding author of the team’s published paper in Patterns, titled “GSFM: A gene set foundation model pre-trained on a massive collection of diverse gene sets.”

    In their paper the authors explained, “Genes are a bit like words, and gene sets are a bit like sentences, because words are reused in different contexts to express unique meanings, and cells reuse genes to carry out different biological functions.”

    “Genes rarely act alone,” Ma’ayan further noted. “Instead, they participate in multiple biological processes, forming different molecular groupings depending on where and when they are active in the cell. A single gene can play different roles in different settings, much like a word can have different meanings in different sentences. Just as modern language models learn the meaning of words from context, we asked whether AI could learn the ‘meaning’ of genes in the same way. Our GSFM was designed to do exactly that.”

    To build the model, the researchers compiled millions of gene sets from published scientific studies and gene expression datasets. In total, the system learned from hundreds of thousands of independent research efforts.

    The AI model was trained in a way similar to solving a puzzle: it was given part of a gene set and asked to predict the missing pieces. Over time, it learned underlying patterns that describe how genes are grouped and interact.

    The AI model was then benchmarked against other approaches and demonstrated strong performance, including the ability to identify gene-gene and gene-function relationships before they were confirmed experimentally. To evaluate this, the model was trained using gene sets from publications up to a defined cutoff date, and then tested on whether it could predict discoveries reported in studies published after that cutoff date.

    “Unlike previous biological AI models that primarily rely on gene expression data, our GSFM is uniquely trained on gene sets, a different and largely underused type of biological information,” Ma’ayan stated. “This approach allows the model to integrate diverse data from many diseases, experimental methods, and research conditions, creating a unified representation of gene relationships across biology.”

    The team’s studies showed that the new model can help identify the function of poorly understood genes without immediate laboratory experiments, highlight genes involved in disease processes, and suggest potential new drug targets and biomarkers. The model offers a reusable knowledge system for many types of biomedical research data analysis tasks—for example, improved gene set enrichment analysis. In essence, the researchers suggested, GSFM offers a new “map” of how genes work together in different contexts. “Unlike prior methods that are mainly based on similarity of all genes to annotated genes, GSFM’s architecture can capture the more complex non-linear and multi-modal relationships between genes and the gene modules these genes constitute,” the investigators wrote. “GSFM’s ability to predict genes held out from known gene sets can be useful for many applications in computational systems biology.”

    GSFMs could enhance existing bioinformatics tools and improve the interpretation of data collected with omics technologies. One immediate application is in gene set enrichment analysis, a widely used method in molecular biology research. By improving how scientists interpret gene groupings, the model may help uncover new biological insights from both existing and future datasets.

    “Like the way LLMs predict the next word in a sentence, GSFM guesses the next missing gene when presented with a gene set,” the scientists stated. “With this power, GSFM can be used to reliably assign the most likely functions to understudied genes, and make gene set enrichment analysis more precise, ranking the most relevant enriched terms when presented with any query gene set.”

    The research team plans to expand the system by combining GSFM with other AI foundation models. One goal is to integrate it with language-based models to generate natural-language explanations of gene functions. Another future direction is combining GSFM with drug-focused AI models, with the long-term aim of predicting how drugs interact with cells and supporting the design of new therapeutics.

    “In summary, GSFM’s ability to distil knowledge from large amounts of unlabeled gene sets automatically, and to do so successfully across multiple sources of knowledge, can be translated into many ‘‘low-hanging fruit’’ hypotheses that could be tested in wet lab experiments to rapidly advance knowledge in biomedical research,” the investigators concluded.

    The gene pages and the GSFM model are accessible at https://gsfm.maayanlab.cloud and https://github.com/MaayanLab/gsfm.

    The post AI Model Offers Map of How Genes Work Together in Different Cellular Contexts appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Bio-IT World Celebrates 25 Years with Opening Plenary on Rare Disease Challenges and Opportunities https://edusehat.com/en/bio-it-world-celebrates-25-years-with-opening-plenary-on-rare-disease-challenges-and-opportunities https://edusehat.com/en/bio-it-world-celebrates-25-years-with-opening-plenary-on-rare-disease-challenges-and-opportunities Fri, 22 May 2026 04:00:09 +0700 Edusehat Bio-IT, World, Celebrates, Years, with, Opening, Plenary, Rare, Disease, Challenges, and, Opportunities BOSTON — Thomas Bartlett’s life changed in 2019 when he was diagnosed with late-onset myasthenia gravis (MG). The 15-year veteran of the Bio-IT World Conference and Expo took to the stage on Tuesday as one of seven speakers in the opening plenary session of this year’s conference, which focused on various aspects of rare disease research and treatment.   

    The session offered a poignant, but often uplifting, launch to the annual conference, which celebrated 25 years from its inception in 2002, when the event was produced by the IDG World Expo Group.   

    Speaking with Susan Ward, PhD, founder and executive director of the Collaborative Trajectory Analysis Project (cTAP), Bartlett described an active life and fulfilling work prior to his diagnosis, and some of the debilitating physical and emotional impact of his disease. “I have to plan everything. If I’m going to go out, I plan ahead of time where I’m going to go [and] the amount of time,” he said. “I have to plan recovery.” Bartlett’s MG has prevented him from working a full-time job, as he would need a full day of rest just to recover from each day. “The math doesn’t work.” 

    Bartlett is now an ambassador for MG Uniter Myasthenia Gravis, an online platform designed to support some 70,000 patients living with the disease in the United States alone. Though there are some treatments that alleviate disease symptoms, there currently is no cure. Bartlett’s disease was diagnosed early thanks to a quick-thinking primary care provider. A recurring theme in the session was the stark reality that many in the rare disease community wait many years for a diagnosis.   

    Of the estimated 7–8,000 rare genetic diseases, many not well understood. About one in 10 people in the U.S. “either has or will have a rare disease at some point,” Ward noted. In a conference of about 2,700 attendees, “there are going to be about 270 people on average who might have a rare disease. So the magnitude of the problem is huge, even though the numbers of people are quite small.”  

    Given the computational nature of the Bio-IT conference, Bartlett and Ward soon turned to data and the challenges with collecting and aggregating information from rare disease populations. Ward noted that centers of excellence in rare disease may have several patients but “no one center that has enough data for anybody to really learn much.” Aggregating data from multiple centers and across geographies is one possibility but due to the differences that exist between centers across states and countries, “you need a really rich and deep ontology” as well as “context for what those data mean,” she said.   

    And that’s not the only challenge. In many cases, rare diseases can present and progress differently in patients with the same condition. “Imagine you’re trying to design a clinical trial. You’ve got patients who are fluctuating [while] you’re really looking for patients who are slowly declining” and “patients who have intermittent remissions,” Ward noted. With that mix, “you’re going to have a very noisy trial.”   

    There are also other data sources that could provide value. Bartlett noted that wearable devices such as the Apple watch capture useful health-related data, but as it is not clinical data, physicians cannot use it. As someone with decades of tech experience—including a stint at Apple—Bartlett asked: “How do we change that?” How do we prove the patient’s experience with the data that we can collect, and work with companies and legislation” to “include real world data and evidence and compare that … with the clinical data and get a much broader picture.”   

    Bartlett’s closing comments focused on hope for people living with rare diseases. And with good reason given recent successes in development of gene therapies and other therapeutics. As a patient, “you need to have something that you can look forward to today,” he said. For now, MG is incurable but “we will find ways to get to that end game and ultimately have a cure or at least a high level of quality of life.” 

    Shortening the rare disease diagnostic journey 

    Sebastien Lefebvre, head of technology, data and AI at Aurelis Insights, has spent 10 years in the rare disease space in different capacities including developing platforms for digital decision support or “data-driven and AI-assisted support decisions.” Speaking with William Van Etten, PhD, co-founder, CEO and principal scientist, StarfleetBio, Lefebvre described Rare Answers, a clinical decision support platform for rare disease diagnostics that he worked on while at Alexion Pharmaceuticals. The platform was designed in collaboration with two children’s hospitals as well as a number of technology and data science companies. 

    An image showing Sebastien Lefebvre, head of technology, data and AI, Aurelis Insights (right) and William Van Etten, PhD, co-founder, CEO & Principal Scientist, StarfleetBio (left) in conversation at Bio-IT World 2026. [Uduak Thomas]
    Sebastien Lefebvre, head of technology, data and AI, Aurelis Insights (right) and William Van Etten, PhD, co-founder, CEO & Principal Scientist, StarfleetBio (left). [Uduak Thomas]
    He also described a second project in 2022 with the Rare-X program that analyzed data from public databases of rare and inherited diseases, drugs, and genes. Lefebvre and his colleagues hoped to produce an accurate assessment of the total number of rare diseases. Following extensive data cleaning and normalization—now made much simpler with advances in AI and machine learning—they arrived at a figure closer to 12,000. Of that number, between 80–87 percent have a genetic basis and about 80 percent had at least three associated phenotypic descriptors. That kind of information provides a viable starting point for applying AI-assisted data-driven diagnostic approaches. This is important because, as Bartlett noted, many patients with rare diseases wait years for a diagnosis. “It all starts with a diagnosis,” Lefebvre said. “[If] you don’t known what you’ve got, how can you [treat it].” 

    Van Etten is focused on making individual genomes truly private. The emergence of commercial personal genomics companies created a data privacy problem. Customers pay for their genomes to be sequenced by a company that holds the data, reads it, and sends periodic reports. He has developed an app called DNAVault that lets people host their genomes on their smartphones, putting data control back into their hands.  

    Van Etten’s new company, StarfleetBio, is partnering with his former consulting firm, BioTeam, and the Hubbard Center for Genomic Studies at the University of New Hampshire, to provide sequencing services. 

    “It used to be that we needed to centralize all the human genome data because you need a lot of compute to perform the analysis, but it’s really not required anymore,” he said. “We decided to decentralize it, where your genome is on your phone, you can generate your own reports, and nobody has access to it but you.”  

    Each encrypted genome is only accessible with a key unique to the individual’s phone. This way, only they individual can download their data and read it. Some audience members clearly approved of Van Etten’s app, with shouts of “Bravo!” from the back of the hall. (The app was later named one of three “Best of Show” winners at this year’s meeting.)  

    Among the features in the app is a fun kinship feature, which lets two people determine if they are related by placing their phones in close proximity as if sharing a Wi-Fi password. Another feature dubbed “origins” lets people track their ancestry over thousands of years via their Y-chromosome or mitochondrial DNA. Van Etten was particularly moved by the kind of insights this feature revealed about human relationships. “We found that all humans are far more closely related than we thought,” he said. “We all really [came from] the same 5,000–10,000 people from 50,000–70,000 years ago.”   

    Another app feature screens for the 81 ACMG medically actionable genes to provide health reports, while a final feature lets people ask questions about their genome and get answers much the same way one might enter a question into Google or ChatGPT.  

    Tying this to rare diseases, Van Etten is working on ways for app users to opt into participating in relevant research studies and clinical trials. The idea is that users could “toggle a switch” that would let alert the relevant researchers and then answer questions to help gauge eligibility. Importantly, this would all be done without people having to share their primary information.   

    Learning from rare diseases to treat common conditions 

    Another plenary conversation took place between Morgan Cheatham, MD, partner, head of healthcare & life sciences, Breyer Capital, and Catherine Brownstein, PhD, manager of the Molecular Genomics Core Facility at Boston Children’s Hospital and scientific director of the Manton Center for Orphan Disease Research Gene Discovery Core.   

    Morgan Cheatham, MD, Partner, Head of Healthcare & Life Sciences, Breyer Capital (left), and Catherine Brownstein, PhD, Manager of the Molecular Genomics Core Facility at Boston Children's Hospital and Scientific Director of the Manton Center for Orphan Disease Research Gene Discovery Core (right). [Uduak Thomas]
    Morgan Cheatham, MD, Partner, Head of Healthcare & Life Sciences, Breyer Capital (left), and Catherine Brownstein, PhD, Manager of the Molecular Genomics Core Facility at Boston Children’s Hospital and Scientific Director of the Manton Center for Orphan Disease Research Gene Discovery Core (right). [Uduak Thomas]
    Brownstein and Cheatham use OpenAI’s generative AI to help diagnose patients who in some cases had been waiting decades for answers. Importantly, “this was a zero-shot model,” Cheatham noted. “We didn’t do any specialized training of GPT-3, we just deployed the existing models and we’re able to return answers to families who have been waiting for sometimes over a decade.” 

     He also acknowledged the contributions of people living with rare diseases to many major drug modalities including CAR Ts and RNA medicines. “Many of those modalities were actually validated” with “the help of rare patients who were willing to participate in trials that allowed us to show the efficacy, the safety, and the durability of these modalities.”  

    According to Brownstein, a deeper understanding of rare conditions often has implications for more common conditions. “As someone who spent six years studying hypophosphatemic rickets …  it’s these extreme cases, these rare presentations of disorders where you don’t know the underlying etiology [that] inform the common diseases,” she said. Understand the biology behind hypophosphatemic rickets “has implications for bone density and osteoporosis that affects a ton of us in this room.”  

    Many opportunities were highlighted where some form of AI is already being used or could be applied. One company that Cheatham mentioned is applying AI to colonoscopies to characterize inflammation levels in the bowel in a standardized way with an eye towards connecting patients with ulcerative colitis and Crohn’s disease to relevant clinical trials. There are also opportunities in cardiology, neurology, pathology and more.    

    Giving more patients the right to try 

    In the closing conversation, Van Etten spoke with Dylan Livingston, founder and president of The Alliance for Longevity Initiatives (A4LI). Livingston is at the forefront of efforts aimed at implementing policies in different states that allow patients with rare diseases to try treatments that may benefit before they have been approved.

    The story of how Livingston, still in his 20s, got involved in healthcare policy is interesting. As a college senior during the Covid-19 lockdowns, “I started thinking about COVID as it relates to age [and] why … [I] would be pretty much completely unaffected by COVID and why my grandfather at 92 would most likely die,” he recalled. “It all comes back to aging, your immune response to these diseases and your immune response to chronic diseases overall.” That got him interested in the field of aging and longevity more broadly. 

    Dylan Livingston, founder and president of The Alliance for Longevity Initiatives (A4LI) (left) and William Van Etten, PhD (right) in conversation at Bio-IT World 2026 [Uduak Thomas]
    Dylan Livingston, founder and president of The Alliance for Longevity Initiatives (A4LI) (left) and William Van Etten, PhD (right) in conversation at Bio-IT World 2026 [Uduak Thomas]
    Livingston and his group have worked to pass laws in the state of Montana that extend eligibility under the Right to Try Act, a piece of federal legislation that lets people with terminal illnesses try therapeutics that may help them which are not yet fully approved. The issue with the Right To Try Act as it stands is that “the definition of who is eligible is very narrow” and restricted to people with months left to live “which made no sense to me” Livingston said. From his perspective, people just diagnosed with conditions like Alzheimer’s or Parkinson’s should also have the chance to access treatments which could potentially help them earlier in their journeys as those who are further along in their journeys.  

    Expanding the Right to Try provides a possible pathway to those treatments without requiring approval from the U.S. Food and Drug Administration, which may be years away.  

    Livingston and his team have been successful in expanding the law in Montana to cover people with age-related ailments as well as people with rare diseases, people recently diagnosed with terminal diseases, and people with disease that will eventually become terminal. Now he and his team are working on getting similar changes in place in New Hampshire. There are safeguards in place: the proposed treatment has to be prescribed by two physicians, pass through IRB review, and the therapy must have passed a Phase I testing. “What we’re trying to do is create a system that is safe enough to prevent as many tragedies as possible while also opening up access to as many people as possible,” Livingston said.    

    As an example of the benefit of changing the law, Livingston shared a story of a father whose son had died from a rare mitochondrial disease. The father has since had the genomes of his two other children sequenced, only to discover that they carry the same mitochondrial mutation. In this scenario, Montana’s model would allow the father in this instance to bypass the strict requirements of a drug trial and access treatments that could potentially help his children. 

    “Maybe it’s not as great in terms of a data collection standpoint for companies, but what we’re offering here [are] options for people that don’t have any other options.”   

    *Bio-IT World Conference & Expo, Boston; May 19-21, 2026. 

    The post Bio-IT World Celebrates 25 Years with Opening Plenary on Rare Disease Challenges and Opportunities appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Biomanufacturing Could Reshape Organ Transplantation https://edusehat.com/en/biomanufacturing-could-reshape-organ-transplantation https://edusehat.com/en/biomanufacturing-could-reshape-organ-transplantation Thu, 21 May 2026 02:40:15 +0700 Edusehat Biomanufacturing, Could, Reshape, Organ, Transplantation A persistent global shortfall has long defined the organ transplantation landscape, but a new generation of biofabrication and biomanufacturing technologies is positioning the field for a shift from scarcity to scale. As Boyang Wang, founder and CEO of Immortal Dragons, puts it bluntly, “There is a structural shortage,” even as transplant numbers reach record highs.

    In 2024, approximately 174,000 solid-organ transplants were performed worldwide, yet nearly 668,000 patients remained on waitlists, Wang says. The mismatch is stark—and deadly. “We have a therapy that works, but the input—organs—is fundamentally scarce and cannot be scaled,” Wang says, underscoring the central limitation of modern transplantation systems.

    This imbalance is driving a paradigm shift toward what Wang describes as a “replacement strategy” for medicine. Rather than attempting to repair every failing biological pathway, the idea is to replace entire organs. “The human body has hundreds of ways to fail, but only one way to work correctly,” he explains. “Trying to patch every individual failure mode is inherently inefficient.”

    At the center of this shift lies bioprocessing. The challenge is no longer just proving that engineered tissues can work, but manufacturing them reproducibly at scale. Early progress is evident in simpler tissues. Bioengineered vascular grafts, for example, have already demonstrated that “engineered tissues can be manufactured, regulated, and used in real patients,” marking an inflection point for the field, Wang notes.

    Scaling up to full organs, however, remains a formidable engineering problem. “The main blockers are vascularization and hierarchy,” Wang says, referring to the difficulty of building thick tissues with complex, multi-scale blood vessel networks. Without this architecture, engineered organs cannot sustain long-term function in vivo.

    Reproducibility presents another major hurdle. Moving from bespoke, lab-built constructs to standardized, GMP-grade products requires precise control over every step of the manufacturing process. “We need reproducible, GMP-grade biofabrication processes that can deliver organs as ‘products,’ not artisanal one-offs,” Wang emphasizes.

    Parallel advances in xenotransplantation are helping to expand supply in the near term. Gene-edited pig organs have shown increasing promise, with recent cases demonstrating months of sustained function in human recipients, Wang points out. These efforts, combined with advances in immunomodulation, could extend organ lifespans and broaden clinical applicability.

    Still, biology remains a constraint. “Even when you get an organ, it’s not a generic spare part,” Wang notes, pointing to immune rejection, compatibility challenges, and the burden of lifelong immunosuppression. These factors limit both access and long-term outcomes.

    Logistics also impose hard limits. Traditional donor organs degrade quickly, creating tight time windows for transplantation. “Organs can only stay viable for a very short cold ischemia window,” Wang says, underscoring how geography and coordination directly impact patient survival.

    Despite these challenges, momentum is building. “We’re past the sci-fi stage and into early clinical reality,” Wang observes, pointing to both engineered tissues and xenotransplants entering human trials.

    The road ahead will require advances not only in science but also in infrastructure. A future of scalable organ replacement will demand new regulatory pathways, reimbursement models, and healthcare delivery systems.

    If successful, biomanufacturing could fundamentally reshape transplantation—transforming it from a donor-limited procedure into a scalable, industrialized therapy. Wang envisions a world where life-saving organs are not found, but made.

    The post Biomanufacturing Could Reshape Organ Transplantation appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Mixed-Reality Fermentation Simulator Preps Workforce https://edusehat.com/en/mixed-reality-fermentation-simulator-preps-workforce https://edusehat.com/en/mixed-reality-fermentation-simulator-preps-workforce Thu, 21 May 2026 02:40:14 +0700 Edusehat Mixed-Reality, Fermentation, Simulator, Preps, Workforce Hands-on biomanufacturing training is expensive, regardless of whether that training occurs in manufacturing facilities where training may take production units offline, or in community colleges and universities where the availability of equipment and consumables may limit training time.

    A mixed-reality fermentation training platform dubbed BioSuite Virtual solves much of that challenge.

    Developed by Prism Immersive with funding from BioMADE and expertise from an industry consortium, BioSuite Virtual immerses learners in a world in which they interact with a virtual bioreactor in their physical space. Conversely, the perhaps more familiar augmented reality lets learners interact with physical objects with virtual overlays.

    BioSuite Virtual consists of more than 40 different modules across 12 chapters, starting with a short introduction to the biomanufacturing space, followed by content-specific modules.

    “It’s end-to-end fermentation training,” Jared DeCoste, PhD, CEO and co-founder of Prism Immersive, tells GEN. “Learners gain vital skills along the way as they assemble a bioreactor, sterilize it, inoculate it, add the media, set the controls, and perform a run. They monitor the run by taking samples and observing the fermentation conditions, making necessary adjustments throughout.” As a learner, “you can do things multiple times if you need to. You can start and stop. You can go at your own pace, all the way through the run.”

    Shaped by fermentation SMEs

    Training is based on best practices from industry subject matter experts—especially Amyris, which shared its processing best practices and expertise with Prism early on—and partners at Bioscience Core Skills Institute (BCSI), Northeastern University, and Harford Community College who shaped and piloted the software. Prism made these connections with the support of BioMADE’s member network.

    This lets all users learn from what Dan Beaupré, COO and co-founder of Prism, calls “the best of the best” in precision fermentation. “BioSuite Virtual is informed by dozens of subject matter experts [from industry],” he stresses.

    “BioSuite Virtual isn’t meant to completely supplant in-person training,” Beaupré adds. “It’s a precursor, where users can obtain literacy and develop operational familiarity with precision fermentation workflows before they touch real equipment.” Because they have this foundation, trainers can then focus on teaching more complex processes and scenarios.

    Prism’s first clients, community colleges, began using the virtual training tool this spring, and “about a dozen others” from Massachusetts to Hawaii are licensing it for use in the next academic year. DeCoste reports interest from contract development and manufacturing organizations and biopharma companies, too. “One of the great things about software is that you can modify it for the exact procedures utilized within [a specific] environment.”

    Going forward, Prism Immersive plans to create new modules in such areas as biosafety cabinet operations and aseptic training, “because that’s what industry is calling for,” Beaupré says.

    “All sorts of things are possible in XR [mixed, augmented, or virtual reality], as long as they’re well-designed,” Beaupré emphasizes. “Everything we do is intentional,” and knowledge checks are built in to reinforce and validate learning. After successfully completing the course, learners have the option to be credentialed through BCSI.

    The post Mixed-Reality Fermentation Simulator Preps Workforce appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Standardizing Cell Therapy Production with Technology Facelift https://edusehat.com/en/standardizing-cell-therapy-production-with-technology-facelift https://edusehat.com/en/standardizing-cell-therapy-production-with-technology-facelift Thu, 21 May 2026 02:40:13 +0700 Edusehat Standardizing, Cell, Therapy, Production, with, Technology, Facelift From a manufacturing standpoint, cell therapies are a disparate group of products, each requiring different starting materials and unique production processes. And, for an industry looking to standardize, this diversity is proving to be a challenge.

    So says Marta Costa, PhD, a principal scientist at Portugal-based R&D non-profit, IBET, and co-author of a new study looking at efforts to make cell therapy production more time and cost efficient.

    “Manufacturing cell therapies depends heavily on the cell type, therapeutic modality, and the clinical application, which makes production quite diverse. Different cell types have distinct requirements for cell sourcing, expansion, genetic engineering, and downstream processing,” she tells GEN.

    Costa cites the differences between patient-specific autologous therapies—where cells are collected, modified, and reinfused—and allogeneic therapies—which are made from donor-derived or stem cell banks for multiple patients.

    “Autologous manufacturing is individualized and tends to be decentralized, while allogeneic manufacturing explores scaled bioprocesses to produce larger cell batches in often centralized operations. Besides, even within the same therapeutic class, manufacturing can vary according to disease indication, donor material, genetic engineering strategy, and quality requirements,” she says.

    Technology

    Despite these challenges, industry’s ever-present desire for efficiency means standardization efforts continue. The current focus is on using closed, automated, and modular platforms to create reproducible workflows, Costa adds.

    “Key enabling technologies of next-generation cell therapies will likely explore automated and closed platforms to reduce the risk of variability introduced by manual operations, reduce labor intensity, and, overall, improve consistency in operations that range from the initial cell isolation and selection of starting material up to fill-and-finish.

    “In addition, tools like bioreactors, particularly when combined with process analytical technologies, provide tighter control over culture conditions and offer the opportunity to not only monitor but also adjust operations to ensure final cell quality,” she says.

    Digital standardization

    Digital technologies, such as electronic batch records, are also changing production, according to Costa, who says, “These strategies contribute not only to improve efficiency but also to enhance reproducibility, decrease COGs, and ensure compliance.”

    In the future, artificial intelligence will also have a role to play, Costa says, as cell therapy firms will use the technology to make production more reproducible and data-driven.

    “Although AI is unlikely to eliminate biological variability, its value probably lies in increasing process understanding and control. Examples of strategies already in place exploring AI are in predictive process control to optimize conditions before failures occur and in cell quality prediction, reducing reliance on end-point testing,” she said.

    AI could also help manufacturers determine which quality attributes have the biggest impact on therapeutic efficacy, according to Costa.

    “Identification of critical quality attributes is also another capability where AI could play a significant role, helping manufacturers understand which variables most strongly affect therapeutic performance.

    “And, of course, automation is already viewed as a practical pathway toward standardization because it reduces operator-to-operator variation, contamination risk, and batch failures,” she says.

    The post Standardizing Cell Therapy Production with Technology Facelift appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Mutating Antibodies for Easier Drug-Conjugate Manufacturing https://edusehat.com/en/mutating-antibodies-for-easier-drug-conjugate-manufacturing https://edusehat.com/en/mutating-antibodies-for-easier-drug-conjugate-manufacturing Thu, 21 May 2026 02:40:13 +0700 Edusehat Mutating, Antibodies, for, Easier, Drug-Conjugate, Manufacturing Scientists in the United States have developed a general-purpose antibody that they hope will help revolutionize antibody-drug conjugate (ADC) manufacturing. The team, from Johns Hopkins University, says they mutated the fragment crystallizable (FC) region, the part of an antibody that modulates immune response. The aim was to create new sites to attach molecules, including nanoparticle drugs or fluorescent markers for quality assurance.

    According to Jamie Spangler, PhD, associate professor of biomedical engineering and chemical & biomolecular engineering, the new antibodies could—in the future—lead to more effective and easier-to-manufacture drug conjugates.

    “The chemistry of antibody drug conjugates is so heterogeneous. It can be hard to characterize the drug-to-antibody ratio and to [do things like] maintain consistency in formulations.”

    To get around this problem, Spangler’s team installed six mutations on the FC region of an antibody that can act as attachment sites for a variety of molecules. The team was able to attach a dye to quantify how many sites were available and discovered the best productivity was found when using up to four sites.

    They emphasize that the sites can be used for many purposes.

    “You can attach whatever you want,” Spangler explains. “You could use [them] to make an antibody-dye conjugate or even a drug conjugate.”

    According to Spangler, the team has already shown that the mutations can be used to conjugate with nanoparticles. “We encapsulate the protein we want to deliver within the nanoparticle, and then we coat the surface with an antibody. The nanoparticle we’re carrying, in this case, contains some GFP [green fluorescent protein], which is a fluorescent readout, but we can attach that to an antibody.”

    After the antibody binds to a cell expressing the target, it’s internalized, and the nanoparticle can release its cargo, she explains. This system can be used for any number of purposes.

    “The sky’s the limit for how people want to use this in their own research and their own work,” she says. “It’s a fully tuneable and generalisable system, and we’d encourage people to think broadly and creatively about the different attachments they can use.”

    The post Mutating Antibodies for Easier Drug-Conjugate Manufacturing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     HELIX AI Model Accurately Predicts RNA Splicing, Unlocks Precision Medicine https://edusehat.com/en/helix-ai-model-accurately-predicts-rna-splicing-unlocks-precision-medicine https://edusehat.com/en/helix-ai-model-accurately-predicts-rna-splicing-unlocks-precision-medicine Thu, 21 May 2026 02:40:12 +0700 Edusehat HELIX, Model, Accurately, Predicts, RNA, Splicing, Unlocks, Precision, Medicine RNA splicing, in which different coding RNA, or exons, are joined together after noncoding regions, or introns, are removed, allows for a large array of RNA transcript isoforms with distinct sequences, and functions in tissue- and cell-type-specific patterns. Conversely, transcript isoform alterations can sensitively reflect dynamic changes in cellular states. Aberrant splicing is closely associated with major diseases, such as cancer.  

    In a new study published in Nature Computational Science titled, “HELIX: a scalable model for predicting context-dependent regulation of RNA splicing and isoform usage,” researchers from the Chinese Academy of Sciences have developed an AI-driven framework that enables highly accurate prediction of RNA splicing and isoform usage by integrating genomic sequence features with tissue-specific RNA binding protein (RBP) expression profiles. The work offers valuable insights for splicing regulatory patterns, pathogenic variant interpretation, and precision medicine research. 

    Isoform usage is jointly regulated by multiple layers of control, including regulatory elements, such as splicing enhancers and silencers on exons and introns, and tissue microenvironments. Scientists have been challenged to accurately characterize and predict RNA splicing and isoform usage across tissues, cell types, and disease states. 

    The study’s AI framework, Hierarchical Explainable LSTM for Isoform eXpression (HELIX), overcomes the limitations of conventional approaches via a two-layer deep-learning architecture. 

    First, the framework integrates DNA sequence information with the expression profiles of 1,499 RBPs. Long short-term memory (LSTM) networks are then employed to effectively capture the complex dependencies and competitive relationships among multiple splice sites. 

    This design enables precise, reliable prediction of RNA splicing and transcript isoform usage. The model was trained and optimized on large-scale short- and long-read RNA-seq datasets covering 30 distinct human tissues, allowing accurate quantification of complex transcript structures and isoform usage. Results show that HELIX substantially outperforms existing mainstream methods in both splicing strength prediction and overall isoform usage prediction. 

    In disease-related studies, HELIX deciphered aberrant RNA splicing and transcript isoform alterations. Notably, the researchers identified widespread splicing dysregulation and abnormal isoform usage in tumor cells using large colorectal cancer cohorts. 

    The results reveal strong correlations among such alterations and genomic mutations, RBP dysregulation, and patient clinical profiles. Results support that splicing abnormalities can serve as key molecular signatures for tumor progression and guiding patient stratification. 

    The team also developed scHELIX, a single-cell RNA sequencing extension of HELIX. scHELIX supports high-resolution profiling of transcript isoform usage across different cell types and tumor subpopulations, which offer a refined view of intratumoral heterogeneity. 

    The findings reveal distinct RNA splicing and isoform usage patterns among tumor subclones, providing new clues for tumor evolution research and potential therapeutic target discovery. 

    The post HELIX AI Model Accurately Predicts RNA Splicing, Unlocks Precision Medicine appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Collaborative Drug Discovery Inks Deal with Eli Lilly to Accelerate Biotech Innovation https://edusehat.com/en/collaborative-drug-discovery-inks-deal-with-eli-lilly-to-accelerate-biotech-innovation https://edusehat.com/en/collaborative-drug-discovery-inks-deal-with-eli-lilly-to-accelerate-biotech-innovation Thu, 21 May 2026 02:40:10 +0700 Edusehat Collaborative, Drug, Discovery, Inks, Deal, with, Eli, Lilly, Accelerate, Biotech, Innovation Lilly created Lilly TuneLab to accelerate biotech innovation by enabling participating companies to access models trained on Lilly’s proprietary research data. Through this agreement, biotech companies that use CDD Vault will be able to utilize select Lilly predictive models within their natural scientific workflows, according to Barry A. Bunin, Collaborative Drug Discovery (CDD) president and CEO.

    “By integrating TuneLab directly into CDD Vault, we are advancing CDD’s core vision to enable collaboration across drug discovery teams and organizations. We believe that solving the most complex challenges in drug discovery will depend on innovative collaboration models that provide broad access to research data and empower chemists and biologists to make informed, data-driven decisions,” said Bunin. “TuneLab’s ADMET models will fit in our secure CDD Vault software environment in natural workflows for experimental and computational scientists and with our growing CDD Vault ecosystem of biopharmaceutical companies.”

    This agreement paves the way for the planned integration of Lilly TuneLab in both the core and AI modules within CDD Vault for biotech companies that opt into the program. A company spokesperson explained that the agreement builds on CDD’s founding vision from 2004 to demonstrate the economics of efficiency of web-based collaboration.

    “TuneLab’s models are synergistic with our innovations such as Zero Click Models, Generative Bioisosteres, as well as Ultrafast Deep Learning similarity to SureChEMBL for novelty and Enamine libraries for convenient SAR-by-catalog,”  noted CDD research informatics senior scientist Peter Gedeck, PhD.

    The post Collaborative Drug Discovery Inks Deal with Eli Lilly to Accelerate Biotech Innovation appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Smile Please: Wisconsin Welcomes FUJIFILM Cellular Dynamics New Headquarters https://edusehat.com/en/smile-please-wisconsin-welcomes-fujifilm-cellular-dynamics-new-headquarters https://edusehat.com/en/smile-please-wisconsin-welcomes-fujifilm-cellular-dynamics-new-headquarters Thu, 21 May 2026 02:40:09 +0700 Edusehat Smile, Please:, Wisconsin, Welcomes, FUJIFILM, Cellular, Dynamics, New, Headquarters In January 2012, Kodak, a name synonymous with analog photography, filed for Chapter 11 bankruptcy protection, an event caused in part by the company’s inability to anticipate and adapt to the digital revolution.

    A similar fate might well have befallen its Japanese competitor, Fujifilm. But last year, the Japanese giant recorded record sales and record profits, priding itself on its ability to innovate and evolve.

    Part of that evolution and commitment to growth was on full display this week in Madison, Wisconsin, as Fujifilm executives and state officials officially opened the new facility of FUJIFILM Cellular Dynamics, Inc. (FCDI), which houses nearly 200 employees with a view to developing tomorrow’s life-saving therapies.

    “The collaboration between Wisconsin and Japan has been fantastic,” said Tomoyuki (Tom) Hasegawa, CEO of FCDI for the past four years. “We are making a global impact together.”

    The work here is “incredible,” Hasegawa continued. “All our employees are excited!”

    Among the dignitaries speaking prior to the official ribbon-cutting ceremony were Wisconsin Governor Tony Evers, a 74-year-old Democrat. “Cleaning out a drawer the other day,” Evers joked, “I found a box of Fujifilm. I’m not sure what to do with it!”

    Having toured Fujifilm’s Japan headquarters in 2019, Evers called the opening “a full-circle moment” and “a true Wisconsin success story.” The state’s expertise in stem cell biology began more than 20 years ago with the pioneering research of James Thompson, PhD, who co-founded Cellular Dynamics in 2004. The company was acquired by Fujifilm in 2015.

    The new facility will quadruple FCDI’s cell therapy research and manufacturing, which Evers said, “may hold the key to Parkinson’s disease, Alzheimer’s, cancers, autoimmune and liver disease and other serious health conditions.”

    Also speaking was Toshihisa “Toshi” Iida, a 30-year Fujifilm veteran who currently serves as corporate vice president and general manager of Fujifilm’s Life Sciences Strategy Headquarters and Bio CDMO Division.

    Fujifilm was founded in 1934, Iida said, with a continuous push to combine technology and innovation. “Our journey has not always been easy,” he said. In 2000, the company’s core film business accounted for 70 percent of the firm’s profits. The business did not just decline; “It was a collapse,” he said.

    “We overcame this challenge. I am a living witness to this transformation story.”

    While Fujifilm’s business has evolved and diversified, the company’s purpose pays tribute to its photographic roots: “Giving our world more smiles.” The company’s interest in human health dates back to its early years. Just two years after its founding, Fujifilm produced the first X-ray film. “Our technology must serve people and improve lives,” Iida said.

    Over the past 15 years, Fujifilm has invested some $10 billion in health and biologics. The investment in Madison is just a part of that bigger program. “This site is not just a building, but a platform to support innovation,” Iida said. “Japan is the birthplace of iPS cells. This has the potential to change medicine.”

    Field of dreams

    Hasegawa has been with Fujifilm for his entire professional career of 28 years, joining the company fresh from completing a law degree at the University of Tokyo. The one thing he knew was that he did not want to pursue a law career.

    It was the late 1990s and the dawn of the digital camera revolution. “The company’s transformation story sounded very interesting to me,” Hasegawa told me in an interview in the exhibit hall at the American Society of Gene and Cell Therapy in Boston. “Especially right after Windows 95 came out, those were very exciting days expanding the digital capability of the company globally.”

    Tom Hasegawa Fuji Film booth
    FUJIFILM Cellular Dynamics CEO Tom Hasegawa at his company’s booth at the American Society of Gene and Cell Therapy 2026 in Boston. [K. Davies]
    Before long, the company set its sights on the U.S. market, and Hasegawa moved to New York to lead that effort, before returning to Tokyo in 2011 and assuming direction of the global marketing team. Three years later, he moved into business development and was put in charge of corporate planning and cultural affairs.

    Hasegawa recalls reading about the induced pluripotent stem cells (iPSCs) by accident. In 2012, the Japanese scientist Shinya Yamanaka, PhD, famously won the Nobel Prize in Physiology or Medicine for the identification of factors crucial in the reprogramming of mature cells into pluripotent stem cells.

    Following the acquisition of Cellular Dynamics, Fujifilm “asked me to be in charge” of this fledgling program in regenerative medicine, Hasegawa said. “This is a very well-known story in Japan because [Yamanaka] won the Nobel Prize and it is a very interesting technique. He’s a superstar! That’s why Fujifilm believes that the cell and gene therapy [CGT] field could be our next field of dreams.”

    Thompson built a foundation in iPS cells in Cellular Dynamics and “ introduced lots of new [cell types] derived from iPS cells. Cellular Dynamics became a global leader for iPS-related products,” Hasegawa said. “Fujifilm’s concept was very interesting—we wanted to explore the synergy of imaging, analysis, and digital technologies to support the pharma industry. That’s what happened in 2015.”

    Hasegawa says he has “a big dream” for the company’s growth. “One way is to deliver our iPS cell products, which include cardiomyocyte cells and neuron cells. We have 40 types of cells for toxicity and efficacy testing for pharma companies to do their R&D. We are a leading supplier of these iPS cells, which have high quality and also the same donor cell for multiple neurons to enable isogenic research.”

    At one time, Hasegawa concedes, “we wanted to be a king of pharma, but we switched our strategy.” The company invested heavily in its services to support drug discovery through commercialization, including service lines across contract development and manufacturing (CDMO) and small-molecule drug development. In 2017, Fujifilm acquired reagent company Wako Pure Chemical, which has an HQ in Japan and a subsidiary in Richmond, VA.

    Another strand to Fujifilm’s bow is as a provider of materials including reagent kits. “As you remember [in cinema], film is not necessarily the hero—cameras and cameramen are the heroes,” Hasegawa said. “We support [R&D], so that kind of mindset is very beneficial.”

    Another thriving business is cell therapy utilizing iPSC technology. Hasegawa points to another subsidiary that provides cell therapy CDMO services in California. “We have an end-to-end service for cell therapy,” he said. In the cell therapy space, he is not looking for additional acquisitions, “because we shifted our strategy.” But for the supporting industry, “if there are good opportunities, we can of course have a discussion about it.”

    Fujifilm says its $200-million investment in the new FCDI facility will “help secure America’s supply chain for biotech and regenerative medicine.” The new facility will feature state-of-the-art resources, including a Center of Excellence for genome editing and laboratories for cell culture manufacturing and process development. The space will quadruple capacity for R&D and manufacturing and enhance the company’s capabilities in drug discovery support and process development.

    The company believes the investment will help Fujifilm keep pace with the rapidly expanding cell therapy market—and put a smile on the faces of patients and company executives alike.

    The post Smile Please: Wisconsin Welcomes FUJIFILM Cellular Dynamics New Headquarters appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Regeneron, Parabilis Ink Up-to-$2.3B Antibody-Peptide Conjugate Collaboration https://edusehat.com/en/regeneron-parabilis-ink-up-to-23b-antibody-peptide-conjugate-collaboration https://edusehat.com/en/regeneron-parabilis-ink-up-to-23b-antibody-peptide-conjugate-collaboration Wed, 20 May 2026 12:15:10 +0700 Edusehat Regeneron, Parabilis, Ink, Up-to-2.3B, Antibody-Peptide, Conjugate, Collaboration Regeneron Pharmaceuticals will partner with Parabilis Medicines to discover and develop an initial five candidates encompassing a new form of antibody-drug conjugates aimed at challenging and historically undruggable targets, through a strategic research collaboration that could generate up to $2.3 billion-plus for the Cambridge, MA, biotech.

    Regeneron will marry its antibody capabilities with Parabilis’ stabilized helical peptide or Helicon™ platform, to develop both Antibody-Helicon™ Conjugates (AHCs) as well as stand-alone therapies based on Helicons—stabilized, cell-penetrant alpha-helical peptides designed to engage intracellular protein targets, including flat surfaces that are not well suited to traditional small molecule binding.

    While ADCs traditionally use antibodies to selectively deliver drug payloads into target cells to induce their death from within, the AHCs envisioned by Regeneron and Parabilis would combine antibody-targeted cell access with Helicon payloads designed to selectively modulate specific intracellular proteins, including some long-undruggable proteins.

    “In addition to the potential of Helicons to address previously undruggable targets, the collaboration’s intent to couple Helicons to our VelocImmune® derived-antibodies so as to precisely deliver them to cells of interest represents an exciting new approach with the potential to create an entirely new therapeutic class that can span multiple therapeutic areas,” George D. Yancopoulos, MD, PhD, Regeneron’s board co-chair, president, and CSO, said in a statement.

    Regeneron has agreed to pay Parabilis $125 million, consisting of a $450 million upfront payment and commitment to invest $75 million in Parabilis’ next equity financing, subject to specified conditions. Regeneron also agreed to pay Parabilis payments tied to achieving development, regulatory, and commercial milestones, as well as tiered royalties up to the low double-digits on future net sales of any approved medicines resulting from the collaboration.

    Five initial targets

    With five initial targets, the collaboration agreement could generate up to approximately $2.2 billion in total milestone payments to Parabilis.

    Under the terms of the agreement, additional targets may be pursued upon additional option payments from Regeneron.

    Regeneron shares fell nearly 10% Monday, to $629.68, from Friday’s close of $698.25, and plateaued on Tuesday, inching up 0.1% to $630.30. The Monday drop reflected not the Parabilis deal but a clinical setback: Regeneron on Friday evening acknowledged the failure of a Phase III trial (NCT05352672) assessing two dose levels of the lymphocyte-activation gene-3 (LAG-3) inhibitor fianlimab in combination with a PD-1 inhibitor, Regeneron’s marketed drug Libtayo® (cemiplimab), as a first-line treatment for patients with previously-untreated, unresectable locally advanced or metastatic melanoma.

    Fianlimab plus cemiplimab failed the trial by not reaching statistical significance for the primary endpoint of improvement in progression-free survival (PFS) compared to monotherapy with another PD-1 inhibitor, Merck & Co.’s Keytruda® (pembrolizumab), the multi-indication cancer immunotherapy, Regeneron said, in an announcement released more than four hours after the close of financial markets.

    Parabilis, a privately held company which rebranded from FogPharma in 2024, rang in 2026 by announcing the closing of a $305 million Series F financing on January 8, with proceeds intended to support continued clinical development of its lead helicon peptide candidate zolucatetide (formerly FOG-001)—a first and only direct inhibitor of the elusive β-catenin:TCF interaction, according to the company—including progression toward a registrational trial in desmoid tumors and continued evaluation across genetically simple and more complex tumor types.

    Positive preliminary data

    In March, Parabilis presented preliminary clinical data at the 11th Biennial Meeting of the International Society for Gastrointestinal Hereditary Tumors (InSiGHT) showing significant improvement in duodenal polyposis at 60 weeks in a patient with familial adenomatous polyposis (FAP) treated with zolucatetide in the company’s ongoing Phase I/II trial (NCT05919264).

    The patient showed a 52.2% reduction in desmoid tumor diameter, as well as “substantial” reductions in polyp number and size compared with a pre-treatment evaluation nearly two years prior, consistent with downstaging from Spigelman stage II to stage I.

    The financing, Parabilis added, will also support advancement of its targeted discovery pipeline, including its prostate cancer franchise, and additional efforts to leverage the company’s Helicon platform to unlock long-undruggable disease targets.

    In addition to zolucatetide, Parabilis’ pipeline includes:

    • Two prostate cancer-fighting discovery phase programs, an ERG degrader Helicon program, and an androgen receptor degrader
    • A beta-catenin degrader Helicon program targeting mutations in the Wnt/β-catenin pathway, linked to 80–90% of cases of colorectal cancer, that is also in discovery phase
    • A Helicon-enabled alpha radioligand therapies (HEARTs) program against multiple cancer targets, a program partnered with ARTBIO, in hit identification phase.

    “Through our own pipeline, we have demonstrated the potential of Helicon peptides to directly inhibit or degrade several disease-driving proteins in oncology that have long been considered out of reach,” stated Mathai Mammen, MD, PhD, Parabilis’ chairman, CEO, and president. “We are thrilled to enter into a collaboration with Regeneron that builds on this foundation, combining the intracellular access and binding capabilities of our Helicons against challenging targets with antibodies from Regeneron.”

    The post Regeneron, Parabilis Ink Up-to-$2.3B Antibody-Peptide Conjugate Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Oncogenic Signaling Shaped by a Golgi Trafficking Protein Pair https://edusehat.com/en/oncogenic-signaling-shaped-by-a-golgi-trafficking-protein-pair https://edusehat.com/en/oncogenic-signaling-shaped-by-a-golgi-trafficking-protein-pair Wed, 20 May 2026 08:40:09 +0700 Edusehat Oncogenic, Signaling, Shaped, Golgi, Trafficking, Protein, Pair A new study in Science Signaling identifies a previously overlooked control point in receptor tyrosine kinase (RTK) signaling, one that operates not at the plasma membrane, but at the Golgi. The research, published as “Oncogenic receptor tyrosine kinase signaling is driven by the Golgi protein GOLPH3 and its interaction with MYO18A,” reveals that the Golgi‑localized proteins GOLPH3 and MYO18A act together to route RTKs to the cell surface, thereby setting the strength of growth‑factor signaling across multiple pathways.

    The work was led by Kyle Starost and colleagues at Case Western Reserve University School of Medicine and the University of California, San Diego. Their findings help explain why GOLPH3 is frequently amplified in human cancers and why its overexpression correlates with poor prognosis across tumor types.

    RTKs such as EGFR, insulin receptor, and PDGFR are central drivers of proliferation and survival in many cancers. Although RTK inhibitors are widely used clinically, resistance often emerges, underscoring the need for alternative strategies that modulate signaling upstream of the receptor. The new study identifies one such upstream node: the delivery of RTKs from the Golgi to the plasma membrane.

    Using an unbiased signaling analysis, the team found that siRNA knockdown of GOLPH3 or MYO18A impaired phosphorylation of EGFR at Tyr1068 and Tyr1086, as well as downstream AKT and ERK signaling. These defects persisted even when PI3K/AKT/mTOR signaling was pharmacologically blocked, demonstrating that GOLPH3 acts directly at the receptor level rather than through mTOR modulation.

    To pinpoint the mechanism, the researchers turned to trafficking assays. Imaging of endogenous EGFR showed that loss of GOLPH3 or MYO18A caused the receptor to accumulate in intracellular puncta rather than at the plasma membrane. A quantitative PDGFR‑GFP surface‑delivery assay confirmed that both proteins are required for Golgi‑to‑surface transport. Treatment with brefeldin A or golgicide A, which disrupt Golgi structure, produced similar reductions in surface receptor levels, reinforcing the conclusion that the GOLPH3–MYO18A complex is essential for RTK delivery.

    Overexpression experiments completed the mechanistic picture. Increasing GOLPH3 or MYO18A levels enhanced EGF‑stimulated phosphorylation of EGFR and AKT, while a GOLPH3 mutant unable to bind PI4P failed to do so. These results position the GOLPH3–MYO18A complex as a central determinant of RTK availability at the cell surface.

    The authors wrote, “The GOLPH3-MYO18A complex at the Golgi apparatus was required and rate-limiting for RTK signaling across the cell types and receptors assessed.” The findings suggest that targeting Golgi‑based trafficking machinery could offer a new therapeutic angle for tumors that rely on hyperactive RTK signaling or have developed resistance to RTK inhibitors.

    The post Oncogenic Signaling Shaped by a Golgi Trafficking Protein Pair appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Kraig Biocraft Labs Creates Immortalized Silk Gland Cell Line https://edusehat.com/en/kraig-biocraft-labs-creates-immortalized-silk-gland-cell-line https://edusehat.com/en/kraig-biocraft-labs-creates-immortalized-silk-gland-cell-line Wed, 20 May 2026 05:05:09 +0700 Edusehat Kraig, Biocraft, Labs, Creates, Immortalized, Silk, Gland, Cell, Line Kraig Biocraft Laboratories reports that company scientists created an immortalized silk gland cell line which Kraig officials say could form the foundation for a next-generation biotech platform with potential applications in biopharmaceutical manufacturing, therapeutic peptides, biologically active proteins, and advanced biomaterials.

    This development significantly expands the potential commercial reach of the company’s core technologies beyond recombinant spider silk fibers and textiles, according to Kim Thompson, founder and CEO.

    “This scientific achievement opens the potential for entirely new markets,” notes Thompson. “While our research team is expanding our portfolio and creating exciting new opportunities, management remains focused on the ongoing expansion of recombinant spider silk production and commercialization.”

    The company’s research team successfully isolated and established immortalized silk gland cells that demonstrate strong proliferative capacity, stable serial passaging, and robust long-term viability in vitro, he adds. Early testing has shown exceptionally strong recombinant protein expression and production capabilities, positioning the platform as a promising candidate for scalable industrial bioprocessing and recombinant protein manufacturing, continues Thompson.

    “The potential applications for this technology are extraordinarily broad,” maintains Xiaoli Zhang, PhD, Kraig Labs’ CSO. “We believe these immortalized silk gland cells could become the basis for a highly versatile biotechnology platform capable of supporting future work in therapeutics, vaccines, recombinant proteins, and next-generation biomaterials.”

    The immortalized cell line has also reportedly demonstrated adaptability toward suspension culture systems, which are critical for large-scale industrial manufacturing and modern bioprocessing. This capability could allow the platform to integrate with conventional bioprocessing infrastructure and support more efficient, scalable, and cost-effective production systems, points out Zhang.

     

    The post Kraig Biocraft Labs Creates Immortalized Silk Gland Cell Line appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     How Gut Bacteria Apply Reversible Epigenetic “Bet-Hedging” Strategy to Adapt to Change https://edusehat.com/en/how-gut-bacteria-apply-reversible-epigenetic-bet-hedging-strategy-to-adapt-to-change https://edusehat.com/en/how-gut-bacteria-apply-reversible-epigenetic-bet-hedging-strategy-to-adapt-to-change Wed, 20 May 2026 05:05:08 +0700 Edusehat How, Gut, Bacteria, Apply, Reversible, Epigenetic, “Bet-Hedging”, Strategy, Adapt, Change Researchers headed by a team at Icahn School of Medicine at Mount Sinai have discovered that many gut bacteria use a flexible survival strategy—known as epigenetic “bet-hedging”—to withstand disruptions such as antibiotics and diet changes.

    Studying infant and gut microbiomes, the investigators showed that microbes can switch between functional states, rather than relying solely on genetic mutations, to try to survive shifting conditions. While bet-hedging has been observed in disease-causing bacteria, this is the first study to show that it is widespread among the beneficial microbes that make up the healthy human gut.

    The findings shed light on a previously hidden layer of microbiome biology and may help explain why probiotics and fecal microbiota transplantation (FMT) produce inconsistent benefits across individuals.

    Gang Fang, PhD, professor of genetics and genomic sciences and director of the Center for Genomic AI and Microbiome Medicine at the Icahn School of Medicine at Mount Sinai, is senior and corresponding author of the team’s published paper in Cell Host & Microbe, titled “Epigenetic phase variation in the gut microbiome enhances bacterial adaptation.”

    The human gut microbiome is constantly being disturbed—by medications, illness, and shifts in diet. Yet it often rebounds, the investigators noted. “In response to these alterations, the gut microbiome shows a remarkable adaptive capacity,” they wrote. “Characterizing this adaptive capacity is crucial for understanding the dynamic relationship between the gut microbiome and host physiology, especially in the context of human health and disease.”

    Until now, scientists largely attributed this resilience to genetic mutations that accumulate over time.  But, as the authors continued, “Another mechanism of bacterial adaptation involves DNA methylation, which can regulate gene expression, enhance clonal heterogeneity, and mediate epigenetic phase variation (ePV, intra-strain epigenetic variation that leads to phenotypic differences … ePVs have been characterized in human pathogens, but their roles in commensals remain unclear.”

    Fang continued, “Our study shows that there is another mechanism at work. Even within a single group of genetically identical bacteria, a small subset of cells exists in a different epigenetic state—where chemical tags on the DNA change how genes are turned on or off without altering the genetic code itself. That means some cells are essentially preprogrammed to respond differently to stress, giving the population a built-in survival advantage when conditions suddenly change.”

    So when a stressor such as an antibiotic is introduced, this small subgroup can quickly become dominant because it is already primed to survive. When conditions change again, the population can shift back. This reversible strategy, known as “bet-hedging,” allows microbial communities to adapt rapidly to uncertainty.

    To carry out their work, the researchers combined advanced DNA sequencing, large-scale data analysis, and laboratory experiments. They used long-read sequencing technology to analyze stool samples from infants before and after antibiotic treatment, as well as from FMT donor-recipient pairs. This approach allowed them to detect both genetic structure and epigenetic modifications simultaneously.

    The scientists then analyzed more than 2,300 microbiome samples from previously published studies to determine how common this phenomenon is across individuals and bacterial species. To understand the mechanism in detail, the team isolated a beneficial gut bacterium, Akkermansia muciniphila, and tracked how its epigenetic states shifted in response to different antibiotics—identifying a specific gene involved in the process.

    “Focusing on an Akkermansia muciniphila isolate, we find a specific ePV regulating mucC, a gene of unknown function but whose heterologous expression enhances bacterial tolerance to antibiotics via a bet-hedging strategy,” they stated. “Our results indicate that in the human gut, ePVs may help bacterial populations regain heterogeneity after bottlenecks encountered during colonization of a new host or severe perturbations due to antibiotic exposures.”

    “Our work is the first to systematically demonstrate epigenetic bet-hedging across the human gut microbiome,” Fang noted. “It also identifies a specific gene that controls this switch in a beneficial bacterium and shows that the process is reversible—shifting in different directions depending on the type of antibiotic exposure. We were struck by how quickly small subpopulations could take over. In some cases, bacteria representing less than one percent of a population became dominant under changing conditions.”

    The research team also found significant diversity within what had been considered a single bacterial strain. Even closely related cells could behave differently, with distinct gene activity and stress responses—highlighting how much remains to be understood about the microbiome at a deeper level. The findings help explain why the microbiome is resilient yet difficult to predict, and why microbiome-based treatments can produce variable results.

    “At the same time, our study does not suggest that people should avoid antibiotics when they are medically necessary, nor does it recommend for or against any specific probiotic. Our research is aimed at understanding fundamental biology, not changing current medical care,” added Fang.

    “Compared with genetic phase variation, ePV offers several advantages in enhancing clonal heterogeneity,” the team noted. “The reversibility of ePV, without altering DNA sequence or incurring mutation costs, serves as an additional way for individual bacterial strains to adapt to diverse stresses … Our results indicate that in the human gut, ePVs may help bacterial populations regain heterogeneity after bottlenecks encountered during colonization of a new host or severe perturbations  due to antibiotic exposures.”

    The discoveries have several important implications for human health. In the field of probiotics, it may be that bacteria in a probiotic capsule are not in the same functional state as those that successfully establish themselves in the gut—potentially explaining inconsistent results. “Ultimately, our goal is to design probiotics that are better equipped to establish themselves in the gut and to develop therapies that support beneficial microbes while limiting harmful ones,” Fang said.

    For FMT-based treatments, differences in these epigenetic states between donors and recipients may influence how well microbiota transplants work. And when considering antibiotic recovery, some gut bacteria may survive antibiotic treatment not because they are genetically resistant, but because a subset of cells is already in a protective epigenetic state that allows rapid rebound after treatment ends.

    The research team plans to study larger groups of patients over time, particularly during and after antibiotic treatment and FMT. They also aim to explore whether similar mechanisms exist in other gut bacteria and to investigate how these epigenetic switches might be harnessed. In the longer term, understanding and potentially controlling these reversible switches could lead to more effective microbiome-based therapies, the investigators suggest.

    “These ePV-driven regulatory mechanisms open new opportunities for targeted epigenetic interventions to improve the desired functions of beneficial bacteria,” the scientists stated. “For example, by manipulating ePV, we may strategically boost the resilience and functional capabilities of beneficial bacteria, which might improve the success rates of probiotic engraftment and the efficacy of treatments for microbiota-associated conditions.”

    The post How Gut Bacteria Apply Reversible Epigenetic “Bet-Hedging” Strategy to Adapt to Change appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Colossal Biosciences is growing chickens in a 3D-printed artificial eggshell https://edusehat.com/en/colossal-biosciences-is-growing-chickens-in-a-3d-printed-artificial-eggshell https://edusehat.com/en/colossal-biosciences-is-growing-chickens-in-a-3d-printed-artificial-eggshell Wed, 20 May 2026 01:35:02 +0700 Edusehat Colossal, Biosciences, growing, chickens, 3D-printed, artificial, eggshell
  • Artificial eggshell, not artificial egg: Colossal Biosciences has grown baby chicks inside 3D-printed plastic containers coated with a silicone-based membrane that mimics an eggshell's oxygen exchange — a meaningful step, but scientists say the company is overselling it.

  • The moa is one target: Colossal's goal is resurrecting the giant moa, a 12-foot flightless bird hunted to extinction — which would require genetically rewriting thousands of DNA letters and scaling up the artificial eggs to the size of a salad spinner.

  • Scientists are skeptical: Researchers have been growing birds in artificial containers since 1998 and say Colossal's claims of a first-ever breakthrough are overblown — a familiar pattern for a company that last year also faced widespread rejection of its "dire wolf" resurrection claim.

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    The baby chicks were shifting and starting to pip—or trying to hatch. But not from an egg. 

    Instead, these chickens were growing inside transparent 3D-printed plastic cups at the Dallas headquarters of Colossal Biosciences.

    The biotech company today claimed it has developed a “fully artificial egg” as part of its effort to resurrect extinct avian species, including birds like the dodo and the giant moa.

    But “artificial eggshell” would probably be a better description for the invention. It’s an oval-shaped printed lattice, coated inside with a special silicone-based membrane that lets in oxygen, just as a real eggshell does. 

    To generate birds, Colossal took recently laid chicken eggs and carefully poured their contents into the artificial shells, where they continued growing. A window on top lets researchers peek inside.  

    “To see them all moving around in their artificial eggs was absolutely mind blowing,” says Andrew Pask, the company’s chief biology officer. “You really feel you can grow life outside of the womb.”

    Colossal was founded in 2021 with plans to use gene editing and reproductive technology to restore extinct species, including the woolly mammoth. It’s since raised more than $800 million toward what it now terms the “scalable and controllable” creation of animals.

    According to Pask, the egg technology could help conserve at-risk bird species. It could also play a role in a project to re-create the extinct giant moa, a flightless 12-foot-tall bird that once lived in New Zealand and laid four-liter eggs, larger than those of any living bird.

    But Colossal may be able build one that’s big enough. The company provided a photograph of a prototype 3D-printed egg so large that staff have started to call it the “salad spinner.”

    The moa went extinct after canoes carrying the ancestors of the Maori arrived on New Zealand’s South Island about 750 years ago. Archeological sites showcase the birds’ bones alongside stone cutting tools—clear evidence that they were hunted.

    To be clear—Colossal isn’t close to re-creating the moa. Before that could happen, scientists would need to study DNA data from old moa bones and insert thousands of genetic changes into the genome of an existing bird, something that’s still technically difficult to do—with or without an artificial egg.

    artificial womb for chicken embryos
    COLOSSAL BIOSCIENCES

    Some scientists also think Colossal is taking too much credit for its artificial eggshell, which it announced in a thundering YouTube video intoning that the company has solved the “impossible question of which came first, the chicken or the egg.”

    The video is pure Hollywood—it’s meant to be funny and exciting. But Colossal has a habit of antagonizing scientists by making false and exaggerated claims. Last year, for instance, the company said it had re-created the extinct dire wolf—a claim widely rejected by experts. 

    This time, Colossal’s fluffed-up assertion of having created the “first-ever shell-less incubation system” is what’s raising hackles among the small flock of scientists who’ve been working on the technology for years. 

    “Clearly an overstatement,” says Katsuya Obara, at the University of Tsukuba in Japan, who in 2024 hatched chickens from beneath transparent plastic film. “The technology here is essentially a modification of existing methods.”

    In fact, Obara notes, growing birds in artificial containers goes all the way back to 1998, when another Japanese group managed to do it with quail.

    What may be an advance by Colossal is the special membrane, which lets the embryo access more oxygen. Previous systems required scientists to supplement the gas—something that may not have been good for the chicks, as often some of them would fail to hatch. 

    The work on the artificial eggshell was carried out in Dallas by Colossal’s exogenous development team, or Exo Dev. That group is also trying to develop artificial wombs for mammals, starting with marsupials.

    “We’re looking at every single facet of what’s happening during a mammalian pregnancy to unpack exactly how we then go about recapitulating that,” says Pask.

    For that team, an artificial eggshell is a relatively quick and easy technical win. That’s because chickens are already an example of ex utero development. After an egg is laid, a small embryo sitting on top of the yolk starts growing, drawing nutrients from the yolk, the white, and even the shell., which provides calcium. (Colossal says it has to add ground-up calcium to the artificial eggs.)

    looking down into the artificial egg shell to see a developing chick embryo and its vascular structure
    COLOSSAL BIOSCIENCES

    In order to create a moa, Colossal will have to genetically alter another type of bird, changing potentially thousands of DNA letters. But so far, chickens are the only bird species that can be genetically engineered. And that’s via a tricky process of editing stem cells that produce egg and sperm. Scientists have to add or delete DNA letters from these cells and then inject them back into an egg. The resulting bird will carry the genetic change in its gonads—and then be able to pass it on. 

    Pask says Colossal’s idea is that it could modify avian stem cells enough toproduce moa-like sperm or eggs. But then you might have the odd situation of a chicken laying an egg with a moa embryo inside it. “You would have chickens making moa egg and moa sperm. But it’s still a chicken egg,” he says.

    Helen Sang, a professor emeritus at the Roslin Institute in the United Kingdom, says she’s not sure a moa embryo could survive on the yolk of a chicken egg, given evolutionary differences. “There are significant challenges to overcome to grow an embryo of a different species in artificial eggs,” says Sang.

    Just one of those is the huge size discrepancy. The amount of yolk in a chicken egg would hardly be enough to support the much larger moa chick. Yet Pask says that is exactly where the artificial egg will come in handy.

    He says it may be possible to use a fine needle to slowly “put 50 yolks together to make that yolk mass much larger.”

    “The chicken egg isn’t going to be big enough to support the growth of the moa through to term, to when it would normally hatch, but that’s when you could then take that egg, put it into the artificial egg environment, and then scale it up in size,” he says.

    So far, Pask says, the artificial egg is working well for chickens—almost too well. “We hatched 26 chickens and then [our CEO] asked us to put the brakes on. We have too many chickens running around.”

    ]]>     Google DeepMind and Edison Are Building the AI Scientist https://edusehat.com/en/google-deepmind-and-edison-are-building-the-ai-scientist https://edusehat.com/en/google-deepmind-and-edison-are-building-the-ai-scientist Wed, 20 May 2026 01:30:09 +0700 Edusehat Google, DeepMind, and, Edison, Are, Building, the, Scientist Google DeepMind and Edison Scientific are on an ambitious mission to build the AI scientist. These platforms propose to automate the scientific method using reasoning systems that connect hypothesis generation, experimental design, and data interpretation in one platform. In drug discovery, where traditional development timelines can stretch beyond a decade, such systems promise to dramatically accelerate the pace of biomedical research.

    The AlphaFold developer and the nonprofit home organization behind Edison, FutureHouse, originally introduced their respective systems, Co-Scientist and Robin, as bioRxiv preprints in early 2025. Those studies have now been published in Nature, marking another step toward a growing ecosystem of specialized AI agents for life science research. 

    Led by Demis Hassabis, PhD, CEO, and 2024 Nobel laureate in Chemistry, DeepMind is no stranger to expanding biomedicine. The team published a January Nature paper describing AlphaGenome, a unifying DNA sequence model for regulatory variant-effect prediction to support understanding of genome function and disease biology. 

    Additionally, DeepMind drug discovery spinout, Isomorphic Labs, recently made waves after securing a whopping $2.1 billion Series B led by Thrive Capital, signaling the industry’s growing investment in AI-driven therapeutics. 

    “I’ve always believed the No.1 application of AI should be to improve human health,” wrote Hassabis on LinkedIn when announcing Isomorphic’s blockbuster raise. 

    DeepMind’s newly published AI assistant, Co-Scientist, is a general-purpose multi-agent system built with Google’s Gemini and driven by natural language prompts. The platform demonstrated initial validation across three biomedical applications: drug repurposing for acute myeloid leukemia, novel target discovery for liver fibrosis, and explaining mechanisms of anti-microbial resistance. 

    Co-Scientist’s design scales test-time compute to iteratively reason, evolve, and improve the output as it gathers more knowledge. Researchers can also actively steer the system by refining generated ideas or providing feedback through the natural language chat.

    Vivek Natarajan, research scientist at DeepMind, emphasizes that time is a valuable commodity when tackling disease. Co-Scientist aims to support humans scientists in reaching answers to their problems much faster than before, from “months and years to minutes and hours.”

    “To realize this vision, we need to build in reliability, trustworthiness and ensure a collaborative human-AI interaction paradigm. We have done a lot of research on these aspects and we are continuing to improve,” Natarajan told GEN Edge.

    Closing the loop 

    Edison is the commercial spinout of FutureHouse, an AI scientist non-profit backed by former Google CEO Eric Schmidt and co-founded by Sam Rodriques, PhD, former group leader at The Francis Crick Institute and Edison’s CEO. The team’s newly published platform, Robin, leverages both OpenAI o4-mini and Anthropic Claude 3.7 to aid biological discovery.  

    In research tasks, Robin proposed repurposing Ripasudil, an existing drug for treatment of glaucoma, to address dry age-related macular degeneration (dAMD) via a novel mechanism that enhanced retinal pigment epithelial cell phagocytosis. The platform also suggested a circadian clock modulator, KL001, as an unexpected treatment for dAMD, illustrating the ability to make new connections not found in existing literature. Both insights were experimentally validated in patient-derived retinal pigment epithelium (RPE) cells. 

    Since Robin’s May 2025 preprint release, Edison unveiled an updated AI scientist, Kosmos, last November. Kosmos can reason over 175 million full-text papers, clinical trials and patents, and operate interactively as a colleague that can sends updates mid-run. The system is reported to perform hundreds of research tasks in parallel to compress months of work into a single day.  

    Today, Edison announced a collaboration with Incyte to employ Kosmos across the global pharma’s discovery and development pipeline. The partnership will focus on enabling continuous learning from translational and clinical data, real-time synthesis of evidence, and predictive models of therapeutic performance.

    Michaela Hinks, founding member of technical staff at Edison, says the main bottlenecks for AI scientist adoption are trust, validation, and the gap in end-to-end solutions.  

    “Most AI tools accelerate the cheaper and easier upstream work, but not the expensive and regulated downstream stages of scientific research,” Hinks told GEN Edge. 

    She also highlights Robin as the first demonstration of an agentic AI scientist generating a hypothesis that is tested and validated in patient-derived cells, not an immortalized cell line, supporting clinically actionable insights for patients in need. 

    Whether AI scientists will truly revolutionize discovery remains to be seen, but researchers are already beginning the experiment.  

    The post Google DeepMind and Edison Are Building the AI Scientist appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Colossal Biosciences is growing chickens in a 3D-printed container https://edusehat.com/en/colossal-biosciences-is-growing-chickens-in-a-3d-printed-container https://edusehat.com/en/colossal-biosciences-is-growing-chickens-in-a-3d-printed-container Tue, 19 May 2026 22:00:03 +0700 Edusehat Colossal, Biosciences, growing, chickens, 3D-printed, container
  • Artificial eggshell, not artificial egg: Colossal Biosciences has grown baby chicks inside 3D-printed plastic containers coated with a silicone-based membrane that mimics an eggshell's oxygen exchange — a meaningful step, but scientists say the company is overselling it.

  • The moa is one target: Colossal's goal is resurrecting the giant moa, a 12-foot flightless bird hunted to extinction — which would require genetically rewriting thousands of DNA letters and scaling up the artificial eggs to the size of a salad spinner.

  • Scientists are skeptical: Researchers have been growing birds in artificial containers since 1998 and say Colossal's claims of a first-ever breakthrough are overblown — a familiar pattern for a company that last year also faced widespread rejection of its "dire wolf" resurrection claim.

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    The baby chicks were shifting and starting to pip—or trying to hatch. But not from an egg. 

    Instead, these chickens were growing inside transparent 3D-printed plastic cups at the Dallas headquarters of Colossal Biosciences.

    The biotech company today claimed it has developed a “fully artificial egg” as part of its effort to resurrect extinct avian species, including birds like the dodo and the giant moa.

    But “artificial eggshell” would probably be a better description for the invention. It’s an oval-shaped printed lattice, coated inside with a special silicone-based membrane that lets in oxygen, just as a real eggshell does. 

    To generate birds, Colossal took recently laid chicken eggs and carefully poured their contents into the artificial shells, where they continued growing. A window on top lets researchers peek inside.  

    “To see them all moving around in their artificial eggs was absolutely mind blowing,” says Andrew Pask, the company’s chief biology officer. “You really feel you can grow life outside of the womb.”

    Colossal was founded in 2021 with plans to use gene editing and reproductive technology to restore extinct species, including the woolly mammoth. It’s since raised more than $800 million toward what it now terms the “scalable and controllable” creation of animals.

    According to Pask, the egg technology could help conserve at-risk bird species. It could also play a role in a project to re-create the extinct giant moa, a flightless 12-foot-tall bird that once lived in New Zealand and laid four-liter eggs, larger than those of any living bird.

    But Colossal may be able build one that’s big enough. The company provided a photograph of a prototype 3D-printed egg so large that staff have started to call it the “salad spinner.”

    The moa went extinct after canoes carrying the ancestors of the Maori arrived on New Zealand’s South Island about 750 years ago. Archeological sites showcase the birds’ bones alongside stone cutting tools—clear evidence that they were hunted.

    To be clear—Colossal isn’t close to re-creating the moa. Before that could happen, scientists would need to study DNA data from old moa bones and insert thousands of genetic changes into the genome of an existing bird, something that’s still technically difficult to do—with or without an artificial egg.

    artificial womb for chicken embryos
    COLOSSAL BIOSCIENCES

    Some scientists also think Colossal is taking too much credit for its artificial eggshell, which it announced in a thundering YouTube video intoning that the company has solved the “impossible question of which came first, the chicken or the egg.”

    The video is pure Hollywood—it’s meant to be funny and exciting. But Colossal has a habit of antagonizing scientists by making false and exaggerated claims. Last year, for instance, the company said it had re-created the extinct dire wolf—a claim widely rejected by experts. 

    This time, Colossal’s fluffed-up assertion of having created the “first-ever shell-less incubation system” is what’s raising hackles among the small flock of scientists who’ve been working on the technology for years. 

    “Clearly an overstatement,” says Katsuya Obara, at the University of Tsukuba in Japan, who in 2024 hatched chickens from beneath transparent plastic film. “The technology here is essentially a modification of existing methods.”

    In fact, Obara notes, growing birds in artificial containers goes all the way back to 1998, when another Japanese group managed to do it with quail.

    What may be an advance by Colossal is the special membrane, which lets the embryo access more oxygen. Previous systems required scientists to supplement the gas—something that may not have been good for the chicks, as often some of them would fail to hatch. 

    The work on the artificial eggshell was carried out in Dallas by Colossal’s exogenous development team, or Exo Dev. That group is also trying to develop artificial wombs for mammals, starting with marsupials.

    “We’re looking at every single facet of what’s happening during a mammalian pregnancy to unpack exactly how we then go about recapitulating that,” says Pask.

    For that team, an artificial eggshell is a relatively quick and easy technical win. That’s because chickens are already an example of ex utero development. After an egg is laid, a small embryo sitting on top of the yolk starts growing, drawing nutrients from the yolk, the white, and even the shell., which provides calcium. (Colossal says it has to add ground-up calcium to the artificial eggs.)

    looking down into the artificial egg shell to see a developing chick embryo and its vascular structure
    COLOSSAL BIOSCIENCES

    In order to create a moa, Colossal will have to genetically alter another type of bird, changing potentially thousands of DNA letters. But so far, chickens are the only bird species that can be genetically engineered. And that’s via a tricky process of editing stem cells that produce egg and sperm. Scientists have to add or delete DNA letters from these cells and then inject them back into an egg. The resulting bird will carry the genetic change in its gonads—and then be able to pass it on. 

    Pask says Colossal’s idea is that it could modify avian stem cells enough toproduce moa-like sperm or eggs. But then you might have the odd situation of a chicken laying an egg with a moa embryo inside it. “You would have chickens making moa egg and moa sperm. But it’s still a chicken egg,” he says.

    Helen Sang, a professor emeritus at the Roslin Institute in the United Kingdom, says she’s not sure a moa embryo could survive on the yolk of a chicken egg, given evolutionary differences. “There are significant challenges to overcome to grow an embryo of a different species in artificial eggs,” says Sang.

    Just one of those is the huge size discrepancy. The amount of yolk in a chicken egg would hardly be enough to support the much larger moa chick. Yet Pask says that is exactly where the artificial egg will come in handy.

    He says it may be possible to use a fine needle to slowly “put 50 yolks together to make that yolk mass much larger.”

    “The chicken egg isn’t going to be big enough to support the growth of the moa through to term, to when it would normally hatch, but that’s when you could then take that egg, put it into the artificial egg environment, and then scale it up in size,” he says.

    So far, Pask says, the artificial egg is working well for chickens—almost too well. “We hatched 26 chickens and then [our CEO] asked us to put the brakes on. We have too many chickens running around.”

    ]]>     Codon Optimization Isn’t Equal: Benchmarking Gene Design for Antibody Expression https://edusehat.com/en/codon-optimization-isnt-equal-benchmarking-gene-design-for-antibody-expression https://edusehat.com/en/codon-optimization-isnt-equal-benchmarking-gene-design-for-antibody-expression Tue, 19 May 2026 11:15:04 +0700 Edusehat Codon, Optimization, Isn’t, Equal:, Benchmarking, Gene, Design, for, Antibody, Expression

    Image of Justin Byers

    Justin Byers

    Founder and CEO
    Axio BioPharma

    Panelist

    Image of Justin Byers

    Justin Byers

    Justin Byers is the founder and CEO of Axio BioPharma. He holds a BS in biochemistry and molecular biology from Illinois State University and has held leadership roles at Illumina, Danaher, and Fujifilm. Throughout his career, Byers has led commercial, operational, and cross-functional initiatives supporting biologics programs from early development through manufacturing. He has worked closely with scientific teams to scale workflows, improve process rigor, and align technical execution with strategic objectives. At Axio, Byers oversees corporate strategy, partnerships, and scientific direction. His focus is positioning the company at the intersection of structured data and biologics workflow execution. Axio is accelerating biologics development through mAb production services for R&D while partnering with innovators and CDMOs to ensure the data required for rigorous decision making and a digitally enabled future is generated, structured, and accessible.
    Image of Daniel Lin-Arlow, PhD

    Daniel Lin-Arlow, PhD

    Chief Scientific Officer and Co-founder
    Ansa Biotechnologies

    Panelist

    Image of Daniel Lin-Arlow, PhD

    Daniel Lin-Arlow, PhD

    Daniel Lin-Arlow, PhD, is a scientist-entrepreneur with deep expertise in synthetic biology and biophysics. Motivated by firsthand challenges in obtaining DNA constructs for metabolic engineering in graduate school, he is deeply committed to providing scientists with the DNA constructs they need for their research. As Ansa’s founding CEO, Lin-Arlow grew the company from two employees in 2018 to more than 70 by 2024, raising over $130 million in venture capital and grant funding to support technology development and commercialization. He transitioned to the role of chief scientific officer in 2024, where he leads the development of new applications of the company’s technologies. Lin-Arlow received his PhD from the University of California, Berkeley for his work in Jay Keasling’s lab for developing the DNA synthesis technology commercialized by Ansa. Prior to graduate school, he was a scientific associate at D.E. Shaw Research where he studied the biophysical properties of G protein-coupled receptors, including how drugs bind and modulate their activity. Dan began his scientific career at MIT, where he earned dual SB degrees in math with computer science and biology, and developed computation tools for the analysis of regulation of gene expression at the Broad Institute of MIT and Harvard. Lin-Arlow is a co-inventor of nine patent families and has co-authored scientific publications in Nature, Science, Cell, PNAS, and Nature Biotechnology.

    Broadcast Date: 
    • Time: 

    Antibody expression titers are key drivers of screening efficiency in discovery, developability, manufacturing economics, and development timelines. Although it is possible to address poor antibody expression by increasing overall batch size and optimizing downstream processes, the root cause often lies in the underlying DNA sequences. Controlled benchmarking studies are helpful for systematically evaluating DNA construct design decisions that impact titers.

    In this GEN webinar, Justin Byers and Daniel Lin-Arlow, PhD, examine how enzymatic DNA synthesis and DNA construct design mitigate antibody expression challenges.

    Byers will walk through a controlled benchmarking study of codon-optimization approaches, including details of the study design and how structured, gene-to-protein workflows can help identify optimal constructs before they become downstream problems. He will show that under matched CHO and HEK293 conditions, antibody constructs codon-optimized with an AI codon language model had consistently higher transient expression titers than other approaches. The AI codon-optimized sequences contained “complex” features such as repeats and GC skew that challenge traditional gene synthesis processes but were readily manufactured by Ansa’s DNA synthesis platform. These results suggest that complex sequence features can be important for optimal gene expression, which makes the ability to manufacture them as relevant as the codon strategy.

    Lin-Arlow will present Ansa’s enzymatic DNA synthesis technology and the benefits to clients working on antibody production, cell and gene therapies, and other synthetic biology applications. Key takeaways include:

    • An AI-powered codon optimization strategy that measurably improves transient antibody expression yield

    • Why controlled side-by-side benchmarking under standardized conditions is the only reliable way to objectively evaluate DNA construct design choices

    • How integrating rigorous sequence evaluation upstream compresses timelines and reduces the risks of expression failures late in development

    • How Ansa’s fully enzymatic DNA synthesis addresses complex sequences, including: High or low GC content, secondary structures, inverted terminal repeats (ITRs), and homopolymers

    • The Ansa On-Time Guarantee—DNA orders shipped on time, or the complete order is free

    A live Q&A session will follow the presentation offering you a chance to pose questions to our expert panelists.

    Produced with support from:

    ANSA Biotechnology logo

    The post Codon Optimization Isn’t Equal: Benchmarking Gene Design for Antibody Expression appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Drug Target for Fragile X Syndrome Identified Through Preclinical Study https://edusehat.com/en/drug-target-for-fragile-x-syndrome-identified-through-preclinical-study https://edusehat.com/en/drug-target-for-fragile-x-syndrome-identified-through-preclinical-study Tue, 19 May 2026 07:35:11 +0700 Edusehat Drug, Target, for, Fragile, Syndrome, Identified, Through, Preclinical, Study UCLA Health researchers have identified a potential drug target for treating fragile X syndrome (FXS), the most common genetic cause of intellectual disability and autism that affects roughly one in 2,000 boys.

    Fragile X syndrome is caused by a mutation in a single gene, FMR1, that results in the loss of a protein critical for normal brain development and function. Headed by Carlos Portera-Cailliau, MD, PhD, professor of neurology at UCLA and member of the UCLA Brain Research Institute, the researchers, the team’s work in genetically engineered mice lacking the Fmr1 gene identified the synaptic protein EPAC2 as a potential therapeutic target for fragile X syndrome. Their study showed that blocking EPAC2 in the fragile X mouse model restored abnormal patterns of brain activity and improved several FXS-associated behavioral symptoms.

    Pertera-Cailliau is senior and corresponding author of the researchers published paper in Neuron, titled “Translatome profiling reveals opposing alterations in inhibitory and excitatory neurons of fragile X mice and identifies EPAC2 as a therapeutic target.”

    Fragile X syndrome is a prototypical neurodevelopmental disorder (NDD) characterized by intellectual disability, social anxiety, atypical sensory processing characterized heightened sensitivity to sensory input such as sound and touch, and a higher risk of seizures. Many also meet the criteria for an autism spectrum disorder diagnosis. “Symptoms of fragile X syndrome (FXS), the leading monogenic cause of intellectual disability and autism, are thought to arise from an excitation/inhibition (E/I) imbalance,” the authors stated.

    FXS is caused by mutations in the FMR1 gene, resulting in near complete loss of the fragile X messenger ribonucleoprotein (FMRP), an RNA-binding protein in neurons that plays different roles in cell compartments including the nucleus, axons and dendrites, including regulating mRNA translation at synapses, they explained. As it is caused by a change in a single gene, fragile X syndrome has long been considered a promising candidate for targeted therapies yet clinical trials to date have not produced an effective treatment. “Since the discovery of the genetic basis of FXS in 1991, several clinical trials have been undertaken—without success—and no specific treatments for FXS are currently available,” the investigators continued. “Thus, there is an urgent need to rethink therapeutic strategies for FXS.”

    For their newly reported study the researchers used genetically engineered knockout (KO) mice that lack Fmr1 to simulate fragile X syndrome. Using genetic sequencing, they found that levels of the gene EPAC2 were increased in the brain of fragile X mice. This was of potential interest as a target for therapy because the gene’s protein, EPAC2, is localized to synapses and is known to be important for learning and memory.

    The researchers then demonstrated that blocking EPAC2 in the fragile X mouse model, either genetically, or using an EPAC2 inhibitor compound, restored cortical circuit function and improved multiple behavioral symptoms associated with fragile X syndrome, including heightened sensitivity to touch, difficulties with social interaction and their susceptibility for seizures. “Perhaps the most exciting result is that treatment with an EPAC2 antagonist can rescue several behavioral phenotypes in Fmr1 KO mice,” the authors stated.

    “EPAC2 emerged as an attractive target because it was consistently altered across multiple types of brain cells in our analysis,” said the study’s first author Anand Suresh, PhD, a post-doctoral fellow in the laboratory of Portera-Cailliau. “When we blocked it, either genetically or with a drug compound, we saw meaningful improvements in both brain circuit function and behavior.”

    EPAC2 is expressed almost exclusively in the brain, which means drugs targeting it are less likely to cause unwanted effects elsewhere in the body. Suresh said this is an important consideration as researchers continue preclinical studies. “This bodes well for future preclinical trials and safety studies in humans, as compounds that target EPAC2 should not have off-target effects,” the authors stated in their report.

    For their study the UCLA investigators used an RNA sequencing technique to examine gene activity separately in two major classes of brain cells: those that excite and those that inhibit neural activity. Fragile X syndrome is thought to arise from an imbalance between these two systems. The analysis revealed striking differences in how the genetic mutation underlying Fragile X syndrome affects each cell type but also identified a small set of genes, including the one that encodes EPAC2, that were dysregulated in both.

    The researchers also found that EPAC2 levels appear to rise gradually as the brain matures, suggesting it may be a particularly relevant target for older children and adults with Fragile X syndrome, rather than only in early development. They concluded, “Our results should encourage the development of novel EPAC2 inhibitors for the treatment of FXS. More generally, our study exemplifies how transcriptomic approaches in animal models of neuropsychiatric conditions can be used to prioritize potential novel therapeutic targets.”

    The post Drug Target for Fragile X Syndrome Identified Through Preclinical Study appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Organ-on-Chip Method Designed to Zero In on Connection Between Diabetes and Dementia https://edusehat.com/en/organ-on-chip-method-designed-to-zero-in-on-connection-between-diabetes-and-dementia https://edusehat.com/en/organ-on-chip-method-designed-to-zero-in-on-connection-between-diabetes-and-dementia Tue, 19 May 2026 07:35:11 +0700 Edusehat Organ-on-Chip, Method, Designed, Zero, Connection, Between, Diabetes, and, Dementia A University of Bath-led research effort received £500,000 to develop an organ-on-chip device that replicates connections between the brain, gut, and pancreas. The GlucoBrain project is designed to allow researchers to track how signals move between the organs and uncover why diabetes may lead to changes in memory and cognition.

    Collaborators include investigators from the University of Oxford and Johns Hopkins. Their findings could pave the way for new treatments to improve the lives of millions of people affected by diabetes, dementia, or both, notes the team.

    Diabetes and Alzheimer’s disease are two of the world’s most pressing health problems, especially in aging societies. While diabetes is widely known to affect the heart, kidneys, and eyes, growing evidence suggests it is also linked with problems in memory, learning, and brain function. However, the biological mechanisms behind this link remain poorly understood.

    “Our gut, pancreas, and brain are constantly communicating via a network of signals, helping us regulate hunger and blood sugar,”  says Despina Moschou, PhD, project lead. “But we still don’t fully understand how these signals interact at a cellular level and why glucose levels are linked to cognitive decline. “By creating a connected system on a chip, we can study in real time how signals travel between organs, how diabetes may impair brain function, and how new drugs could help.”

    Most current knowledge on the link between diabetes and dementia comes from animal studies, simple cell cultures, and patient studies. While these are useful, they don’t fully and accurately capture all the complex interactions between our organs, hormones, and cells, points out Moschou.

    Organ-on-chip technology uses living human cells in miniature devices that mimic how organs work in the body. Unlike cell cultures grown in a petri dish, these devices allow cells to grow in three dimensions, receive a controlled supply of nutrients and interact more naturally. Researchers will also be able to isolate these individual organs and cell types to understand exactly how they communicate at a molecular level.

    The three-year project starts in October, bringing together engineers, clinicians, biologists and computer scientists to model the complex disease interactions. The team will first develop individual chip models for the gut, pancreas, and brain, before connecting them into a multi-organ system. They will gradually increase complexity and measure how each organ responds to glucose, hormones and different drug treatments.

    Researchers from the University of Oxford will provide core clinical expertise in diabetes and metabolic disease, ensuring models are physiologically accurate. The team from Johns Hopkins University brings specialist expertise in Alzheimer’s disease and brain organoids.

    GlucoBrain is a pilot project established to help researchers understand exactly how diseases like diabetes and dementia work at a deeper, biological level. This early-stage research will build the foundations for even more advanced and realistic models, bringing together more organs and cell types, explain team members. By harnessing the power of artificial intelligence, the devices have the potential to reveal new insights into how diseases emerge and develop.

    “Not only would these devices give us an unprecedented way to study diseases, but they could help speed up drug discovery and testing, reducing reliance on animal models and making results more relevant to humans,” continues Moschou. “In the long term, they could pave the way for personalized medicine, using a patient’s own cells to identify the most effective treatment.”

    The project is funded by the Engineering and Physical Sciences Research Council (EPSRC) Health Technologies Connectivity Awards.

     

    The post Organ-on-Chip Method Designed to Zero In on Connection Between Diabetes and Dementia appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Multiple Sclerosis Myelin Loss Revealed by Transcriptomic Analysis in Mice https://edusehat.com/en/multiple-sclerosis-myelin-loss-revealed-by-transcriptomic-analysis-in-mice https://edusehat.com/en/multiple-sclerosis-myelin-loss-revealed-by-transcriptomic-analysis-in-mice Tue, 19 May 2026 07:35:10 +0700 Edusehat Multiple, Sclerosis, Myelin, Loss, Revealed, Transcriptomic, Analysis, Mice More than one million people across the United States live with multiple sclerosis (MS), a disease that affects the brain, optic nerves, and spine. MS is characterized by overwhelming fatigue, muscle spasms, and vision problems, which can flare up and then subside over days, months, or even years. Studying the underlying damage to the nervous system is key to identifying new treatment paradigms for MS. 

    A new study published in Nature Communications titled, “A comparative transcriptomic analysis of mouse demyelination models and multiple sclerosis lesions,” compares two prevailing models, cuprizone (CPZ) and lysophosphatidylcholine (LPC), for the study of myelin loss and regeneration in an MS mouse model. 

    Katrina Adams, PhD, Gallagher Assistant Professor at University of Notre Dame, studies the role of the loss and regeneration of myelin on MS progression. As a fatty substance protects nerve cells, myelin envelopes the axons of the brain as they route the electrical signals that carry information throughout the nervous system. The damage and swelling that follow myelin loss in MS form distinct “lesions,” which vary in size, number and location in the nervous system. 

    “Our analysis of these two models of myelin loss and regeneration provides a road map based on robust scientific evidence that we hope will advance the study of MS and related diseases,” said Adams. 

    While both CPZ and LPC models degrade myelin, the timeline and localization of myelin loss varies. CPZ causes widespread loss of myelin over several weeks while LPC induces a lesion within days. This new research, which was funded by the National Multiple Sclerosis Society, points to specific scenarios in which one model is better suited, depending on which aspect of MS is under investigation. 

    “If you’re studying the myelin-producing cells and what’s happening to them in MS—are they stressed, dying or trying to repair?—CPZ is better, since the loss of myelin is more gradual,” Adams said. “For studying the immune cells that respond to the myelin loss, LPC may be better, since the immune response is more aggressive than in CPZ.” 

    The team also analyzed the resulting lesions from each preclinical model alongside data obtained from human MS tissue samples. Genetic maps of each type of tissue using single-cell RNA sequencing were constructed to examine the genetic changes that occurred in response to demyelination. 

    “By matching each model to features seen in diseased tissue from real patients, we can be sure that we’re targeting things that are actually causing disease in human patients,” Adams said. “There are so many potential paths to follow, so we want to make sure that the path chosen has direct relevance to MS patients.” 

    In addition to phenotypic differences, the genetic changes in diseased cells vary between the two models, an area of future exploration for the Adams research group. 

    Since MS flare-ups are primarily triggered by the immune system’s reaction to lesions, current clinical treatments focus on quelling this autoimmune response. The regeneration of lost myelin within MS lesions remains a promising yet unrealized drug target. 

    “The strategic use of these two preclinical models is essential for translating insights into therapies that might restore lost myelin,” Adams said. “We need to better understand the very process of demyelination in order to treat one of the root causes of this debilitating disorder.” 

    The post Multiple Sclerosis Myelin Loss Revealed by Transcriptomic Analysis in Mice appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO supports and seeks refinements to FDA’s Plausible Mechanism Framework https://edusehat.com/en/bio-supports-and-seeks-refinements-to-fdas-plausible-mechanism-framework https://edusehat.com/en/bio-supports-and-seeks-refinements-to-fdas-plausible-mechanism-framework Mon, 18 May 2026 20:55:04 +0700 Edusehat BIO, supports, and, seeks, refinements, FDA’s, Plausible, Mechanism, Framework Advances in individualized therapies targeting specific genetic conditions offer the promise of new treatments for rare diseases, but with so few patients, it can be extremely difficult to generate nonclinical data and near impossible to test these treatments in a standard clinical trial.

    The Food and Drug Administration (FDA) has proposed guidance for using a “Plausible Mechanism Framework” as an alternative means for proving safety and efficacy of individualized and disease-targeted therapies when a large randomized controlled trial is not feasible. The Biotechnology Innovation Organization (BIO) praises FDA’s proposed guidance in written comments, while also urging improvements to make it more practical and effective.

    “BIO and its members support the Agency’s intent and view this framework as an important progression in regulatory science,” says E’Lissa Flores, BIO Director, Science & Regulatory Affairs, who wrote BIO’s April 27 comment letter to the FDA. “We also believe several areas would benefit from additional clarity and operational detail to support consistent and predictable implementation.”

    BIO’s recommendations for the Plausible Mechanism Framework proposal include:

    • Broadening the scope to other cases: “We encourage the FDA to clarify that the framework’s applicability extends beyond individualized products and is principle‑based and modality‑agnostic, making it suitable for other therapeutic modalities and disease contexts with serious life-threatening conditions,” when appropriate, according to BIO’s comment letter.
    • Providing definitions and references to existing FDA guidances: “Several key guidance concepts outlined in the framework would benefit from clearer definition or reference to existing FDA guidance,” BIO’s comments say.
    • Expand flexibilities for Chemistry, Manufacturing, and Controls (CMC) expectations: “BIO members recommend that the draft guidance address feasible CMC expectations with respect to the individualized nature of these therapies and expand flexibilities when appropriate,” the comments say, “especially as current requirements for early CMC maturity are not yet as feasible for individualized therapies.”
    • Using prior knowledge and leveraging Platform Technology: “The guidance would be strengthened by additional inclusion of recommendations for platform technologies and the systematic use of prior knowledge across the product lifecycle,” the comments explain.
    • Fit-for-purpose post-approval safety and long-term follow-up for small population products: “We kindly advocate that the post-marketing safety monitoring and long-term follow-up expectations within the guidance reflect the realities of small patient populations and individualized products, particularly for n-of-1 therapies,” BIO’s comments say. “Traditional pharmacovigilance and confirmatory study paradigms may not be feasible in these settings.”
    • Clarify framework application and provide operational “how-to” implementation direction, including with examples: “We recommend the Agency clarify how the Plausible Mechanism Framework interfaces with existing programs such as Accelerated Approval, Breakthrough, Fast-Track, RMAT, the Platform Technology Designation Program, and targeted therapy guidances to ensure coherent and predictable regulatory pathways,” BIO comments.

    The comments from BIO envision further refinement of the guidance and anticipate further involvement in the process, concluding: “BIO and its members appreciate the FDA’s leadership and look forward to continued engagement as the guidance is finalized.”

    The post BIO supports and seeks refinements to FDA’s Plausible Mechanism Framework appeared first on Bio.News.

    ]]>     StockWatch: Regenxbio Tumbles Despite Positive Pivotal Data for DMD Gene Therapy Candidate https://edusehat.com/en/stockwatch-regenxbio-tumbles-despite-positive-pivotal-data-for-dmd-gene-therapy-candidate https://edusehat.com/en/stockwatch-regenxbio-tumbles-despite-positive-pivotal-data-for-dmd-gene-therapy-candidate Mon, 18 May 2026 10:05:08 +0700 Edusehat StockWatch:, Regenxbio, Tumbles, Despite, Positive, Pivotal, Data, for, DMD, Gene, Therapy, Candidate Regenxbio (NASDAQ: RGNX) shares nosedived 43% over two days late last week, reaching 52-week lows on consecutive days, despite generating positive pivotal Phase III data for its Duchenne muscular dystrophy (DMD) gene therapy candidate RGX-202.

    While the data was encouraging enough to enable discussion of Regenxbio bringing a second DMD gene therapy to the market, investors and analysts concluded it was not encouraging enough to pose a competitive threat to the developer of the first marketed DMD gene therapy, Sarepta Therapeutics (NASDAQ: SRPT), or to Solid Biosciences (NASDAQ), whose DMD gene therapy candidate SGT-003 is in Phase III as well as Phase I/II trials.

    Even worse, investors were jolted by Regenxbio’s disclosure that the FDA had recommended the company conduct a randomized controlled trial (RCT) to assess RGX-202 in DMD during talks with agency officials. Regenxbio sought to reassure investors in its first-quarter earnings press release by noting past FDA guidance that externally controlled trials “may be adequate for demonstrating substantial evidence of effectiveness, especially when the treatment effect is sufficiently large enough to overcome limitations of externally controlled trials.”

    Regenxbio plans to discuss its data with FDA officials at a future meeting. The agency has offered to review the RGX-202 data and alternative proposals, according to the company.

    “RGX-202 pivotal data point to potential entry of second DMD gene theory, but a possibility of RCT requirement makes market entry timing unclear,” Kostas Biliouris, PhD, a managing director on the biotechnology research team of Oppenheimer & Co., wrote in a research note.

    If the FDA does not insist on an RCT, RGX-202 could gain accelerated approval in 2027, Biliouris noted. Otherwise, the gene therapy is looking at not reaching the market for at least three additional years.

    “Completing an RCT study as a precursor to filing or a precursor to approval means that it’s very unlikely that any new gene therapy would be approved until 2030. And I think that scenario is really untenable for the [DMD] community,” Simpson said. “It’s the opposite of regulatory flexibility.”

    These regulatory and competitive concerns sent investors scrambling to sell Regenxbio shares late last week. The shares tumbled 38% from $10.04 to $6.24 Thursday, then slid another 8% Friday, sinking to $5.72 at the closing bell.

    Positive microdystrophin expression

    Regenxbio’s stock woes came despite the company announcing positive results from its pivotal Phase III portion of the Phase I/II/III AFFINITY DUCHENNE® trial (NCT05693142) of RGX-202. The company said the trial met its primary endpoint as 93% of participants (28 of 30) reached at least 10% microdystrophin expression at Week 12. A 31st participant refused a muscle biopsy and, as a result, did not have a Week 12 biopsy available for evaluation.

    Microdystrophin expression averaged 71.1% across all participants, and 41.6% in older boys, aged >8 years, with 80% of participants achieving >40% microdystrophin expression, Regenxbio said.

    “High unmet need remains for Duchenne patients as current options face limitations related to efficacy, safety, and access. The untreated Duchenne population continues to grow in the United States and globally. Physicians and patients need new next-generation options,” Curran M. Simpson, president and CEO, told analysts on the company’s first quarter earnings call.

    Regenxbio acknowledged two reports of treatment-related serious adverse events (~6.5% of treated patients): An 8-year-old patient developed subacute myocarditis, while a 10-year-old patient showed a case of asymptomatic liver injury.

    “Both were easily managed and resolved within weeks without sequelae,” Simpson told analysts.

    Biliouris acknowledged RGX-202’s positive microdystrophin but said it will not likely have a material impact on Sarepta and its marketed DMD gene therapy Elevidys® (delandistrogene moxeparvovec-rokl).

    Limited likelihood

    “RGX-202’s functional benefit remains unclear without RCT data, limiting the likelihood of AA [accelerated approval] given an already fully approved DMD gene therapy,” Biliouris said.

    He added that RGX-202’s safety profile could deteriorate once the gene therapy reaches the market and is being administered to patients, as happened with Elevidys after some 800 had been treated with the therapy, prompting Sarepta to halt shipments of Elevidys for non-ambulatory patients and pause a Phase III trial.

    The halt—plus a label update limiting Elevidys use to ambulatory patients—explains why the gene therapy’s net product revenue plunged 73% year-over-year in Q1, to $102 million from $375 million. Elevidys generated $898.7 million in 2025 revenue—it ranks second on GEN’s just published A-List of Top 10 Best-Selling Gene Therapies—which was 9.5% above 2024’s $820.8 million.

    The Q1 sales decline has sent Sarepta’s stock into decline: From $23.06 on May 6, before releasing Q1 results after that day’s closing bell, Sarepta shares have slumped 22.5%, to $17.88 on Friday.

    Elevidys sparked a showdown with the FDA last summer when the agency briefly demanded Sarepta also pause Elevidys shipments to ambulant patients following the second patient death tied to Elevidys, then reversed itself after, according to news reports, pleas to Congress, the FDA, and President Donald Trump by conservative leaders and DMD patient advocates—who launched a Change.org petition that garnered 1,900 signatures.

    Competitive advantage

    Despite the slumping sales and resulting stock decline, Biliouris noted that Sarepta and Elevidys have a significant competitive advantage over challengers: A 3-1/2 year first to market advantage, with statistically significant functional benefits reported from randomized trials, as well as what the analyst called “compelling” three-year positive topline follow-up data from ambulatory DMD patients in the 52-patient active arm in Part 1 of Sarepta’s EMBARK trial (Study SRP-9001-301, NCT05096221).

    That data showed significant improvements in North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and 10-meter walk/run (10MWR).

    “Even if RGNX secures AA, we expect minimal impact given the large DMD market size (can accommodate multiple companies) and potential Elevidys monopoly in the non-ambulatory market,” projected for 2027 and later, Biliouris wrote.

    According to Sarepta, Duchenne affects approximately 1 in 3,500 to 5,000 males born worldwide—some 300,000 people worldwide, according to research and patient care group Cure Duchenne. In the United States, about 15,000 young men and a few young women live with DMD, according to Parent Project Muscular Dystrophy estimates. A 2019 study found that most people with DMD become non-ambulatory around ages 10–12 and need assisted ventilation at around 20 years of age.

    Andrew Tsai, equity analyst with Jefferies, said Sarepta’s three-year data, including muscle MRI data, has only begun to be promoted by the company this year. Since it can take six months to go from “start form” initiating the treatment process to infusion with Elevidys, Tsai reasoned, “we expect momentum to rebuild progressively/steadily in Q3/Q4, restoring confidence in the ambulatory DMD oppty.”

    Some ~80% of ambulatory DMD patients remain untreated, Tsai noted, while Sarepta told investors in its Q1 earnings presentation that more than 1,300 patients have been treated with Elevidys in commercial settings or clinical trials as of May 5.

    “While Elevidys’ safety perception has changed, we think marketing efforts on muscle MRI data, long-term three-year EMBARK data, and no deaths in ambulatory DMD could entice patients/caregivers and physicians to adopt Elevidys more, widening the moat,” Tsai wrote.

    Maury Raycroft, PhD, a colleague of Tsai and equity analyst with Jefferies, wrote that Regenxbio’s data “reinforces microdystrophin as a surrogate, which is constructive for SLDB [Solid Biosciences].”

    Playing to strengths

    However, Raycroft added that Regenxbio’s safety events (notwithstanding immunosuppression) and limited regulatory clarity absent a pivotal RCT “play into SLDB’s strengths,” such as its use of a steroid-only prophylactic immunomodulation regimen (no safety issues to date) and its ongoing Phase III IMPACT DUCHENNE trial (NCT07160634), which is an RCT, thus a derisking factor from a regulatory standpoint.

    “We believe RGNX is relying on and will require reg[ulatory] flexibility, which incorporates add’l risk and limitations, especially w/ FDA leadership in flux,” Raycroft wrote. “We caught up w/ SLDB, who also pointed out that RGNX could run into challenges to run an RCT given their immunosuppressive regimen.”

    That regimen consisted of sirolimus, eculizumab, and steroids that included prednisone, researchers from Regenxbio and clinical partners reported in a poster presented at the International Congress of the World Muscle Society, held October 7–11, 2025, in Vienna.

    On May 7, Solid announced it had dosed the first patient in the IMPACT DUCHENNE trial in Australia, at the Children’s Hospital at Westmead. The multi-country, placebo-controlled, randomized, double-blind trial has a pre-specified primary endpoint of change from baseline at 18 months in time to rise from supine (TTR) velocity, based on a Type C meeting with the FDA.

    “With the initiation of a randomized, placebo-controlled clinical trial, we are reinforcing our conviction in SGT-003 and our long-standing commitment to generating well-controlled, high-quality data,” Gabriel Brooks, MD, Solid’s chief medical officer, said in a statement.

    Solid shares dipped 2% on news of the dosing, from $7.20 to $7.07. Since then, the shares have yo-oed, climbing 9% to $7.72 on May 12 but sliding 10% since then, to $6.92 on Friday.

    In its Phase I/II INSPIRE DUCHENNE study (NCT06138639), SGT-003 has also been administered to 46 patients, with approximately 30 participants dosed as of year-end 2025, Solid said.

    “Families living with Duchenne continue to face difficult treatment decisions in a setting of significant unmet medical need,” Brooks added. “Solid remains focused on helping inform the Duchenne community of potential additional treatment options through the responsible and rigorous clinical evaluation of SGT-003.”

    uniQure, Replimune gain as Makary exits FDA

    Two gene therapy developers saw their stocks enjoy significant gains after Martin A. Makary, MD, resigned as FDA commissioner.

    Makary’s resignation on May 12 capped nearly a week of speculation that he was about to exit the agency after a turbulent 13-month tenure. That tenure was marked in part by the elimination of 3,500 FDA positions as part of the Elon Musk-led Department of Government Efficiency (DOGE)-directed federal job cuts—as well as more frequent rejections of biologics license applications (BLAs) for new therapies, particularly gene therapies in rare disease indications.

    Those rejections were carried out by the agency’s Center for Biologics Evaluation and Research (CBER) during the two tenures of Vinayak (Vinay) Prasad, MD, as Center director. Prasad resigned the first time in August 2025 after less than three months at the CBER helm, after he led the FDA’s confrontation with Sarepta over patient deaths tied to Elevidys (see Regenxbio item, above). The second resignation was announced in March and took effect on April 30, after he led the FDA’s hardline stance and public criticism against uniQure (NASDAQ: QURE)’s Huntington’s disease (HD) gene therapy candidate AMT-130.

    While uniQure stock roller-coastered after Prasad’s second resignation, the stock jumped 21% in the four trading days between May 8, when an unnamed-source report about Makary being fired first surfaced in The Wall Street Journal, and May 13, the day after he resigned. uniQure rose 14.5% from $24.15 to $27.66 the day of the WSJ report, plateaued on May 10, dipping two cents to $27.64, then resumed their climb, rising 5% to $29.10 the following day before inching up another 0.2% to $29.17 on Wednesday.

    An even bigger winner among stocks, however, was Replimune Group (NASDAQ: REPL). The developer of oncolytic immunotherapies saw its shares rocket 59% after news surfaced of Makary exiting the FDA.

    Replimune has found itself in the FDA’s crosshairs over its biologics license application (BLA) for its lead product candidate RP1 (vusolimogene oderparepvec) in combination with nivolumab to treat advanced melanoma, instead issuing two complete response letters (CRLs)—one in April 2025, the other last month.

    On April 10, the FDA rejected Replimune’s BLA for a second time, issuing a complete response letter (CRL) contending that the data set upon which the agency’s breakthrough therapy designation was awarded was not sufficient to allow for RP1 approval—an assertion Replimune vehemently rejects.

    Replimune responded to the second BLA by criticizing the FDA for an inconsistent review process, saying the agency contradicted earlier guidance to the company and assessed the resubmitted BLA through a different review team that replaced the team that previously interacted with the company.

    Replimune also defended the combination therapy’s data in the Phase II IGNYTE trial (NCT03767348)—a 34% response rate with a median duration of 24.8 months and a favorable safety profile, the basis of the combo’s breakthrough therapy designation.

    Following the first news report of a Makary firing in the works, Replimune shares jumped 22% from $3.34 to $4.07. After slipping 8% to $3.74 the following trading day (May 11), Replimune rose 9% to $4.09 the following day after Makary resigned—then vaulted 30% to $5.30 on Wednesday.

    “Broadly, we see multiple options for experienced leaders who could help stabilize the Agency following the many leadership transitions, and believe the tendency toward the administration’s “Right to Try” could draw a next leader who is more permissive on drug approvals near-term positive on the space,” Brian Abrahams, MD, head of global healthcare research with RBC Capital Markets, wrote in a research note.

    Abrahams put forward six possible permanent successors to Makary:

    • Kyle Diamantas, current interim FDA commissioner; previously FDA deputy commissioner for human foods and senior counselor to Health and Human Services Secretary Robert F. Kennedy Jr.
    • Stephen Hahn, MD, CEO of Nucleus RadioPharma and a former FDA commissioner in President Donald Trump’s first administration (December 2019–January 2021).
    • Brett Giroir, MD, CEO of Altesa Biosciences; previously assistant secretary for health in Trump’s first term and an acting FDA commissioner (2019).
    • Sara Brenner, MD, HHS senior counselor for public health as of April 16; previously FDA principal deputy commissioner and acting FDA commissioner (January–April 2025).
    • Houman Hemmati, MD, PhD, a board-certified ophthalmologist and co-founder of Optigo Biotherapeutics, who is under consideration for CBER director.
    • Richard Pazdur, MD, a 26-year FDA veteran who retired in December 2025 after serving three weeks as CDER director (November–December 2025); previously founding director, FDA Oncology Center of Excellence (2017–2025).

    “If Makary’s ouster indeed stemmed from political disagreements (vapes, abortion), the next Commissioner could harbor more ideological views—which could compromise perceived Agency credibility—and just by virtue of having another change, this would likely exacerbate the mixed messages companies have been receiving around FDA’s bar for their drugs, one of the key regulatory challenges the sector has faced,” Abrahams cautioned.

    Leaders and laggards

    • Innate Pharma (Euronext Paris: IPH) shares jumped 35% from €1.23 ($1.42) to €1.66 ($1.92) on Wednesday after the Marseille, France-based developer of cancer drugs based on innate immunity and antibody engineering reported first-quarter results that beat analyst expectations. Innate finished Q1 with earnings per share of -0.1522, vs. the consensus forecast of -0.1616, on revenue of €2.6 million ($3.022 million) that was more than double (117% above) the €1.2 million ($1.395 million) reported in Q1 2025, thanks to partial or entire recognition of the proceeds received under collaboration agreements with AstraZeneca (NYSE, London Stock Exchange, and NASDAQ Stockholm: AZN) and Sanofi (Euronext Paris: SAN). Innate’s American depositary shares (ADSs) (NASDAQ: IPHA) rocketed 64% from $1.32 to $2.17 Wednesday.
    • Reviva Pharmaceuticals Holdings (NASDAQ: RVPH) shares plummeted 56% from 80 cents to 35 cents on Wednesday after the central nervous system (CNS), inflammatory, and cardiometabolic disease drug developer disclosed in a regulatory filing that the Nasdaq Hearings Panel had delisted the company’s stock, suspending it from trading on the exchange as of Thursday. Reviva said its shares will instead begin trading that day on the OTCQB Venture Market under its existing symbol. The Panel told Reviva that it failed to comply with Nasdaq’s minimum bid price of $1 per share required for continued listing on the Nasdaq Capital Market. Reviva disclosed the delisting the same day it reported first quarter results: The company narrowed its quarterly net loss year-over-year, finished Q1 with a net loss of approximately $3.2 million ($0.46 per share) vs. approximately $6.4 million ($2.61 per share) in the year-ago quarter.

    The post StockWatch: Regenxbio Tumbles Despite Positive Pivotal Data for DMD Gene Therapy Candidate appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ASGCT Q1 Landscape Report Paints Positive Picture for Gene and RNA Therapy https://edusehat.com/en/asgct-q1-landscape-report-paints-positive-picture-for-gene-and-rna-therapy https://edusehat.com/en/asgct-q1-landscape-report-paints-positive-picture-for-gene-and-rna-therapy Sat, 16 May 2026 04:05:10 +0700 Edusehat ASGCT, Landscape, Report, Paints, Positive, Picture, for, Gene, and, RNA, Therapy BOSTON – The CEO of the American Society for Gene and Cell Therapy (ASGCT), David Barrett, JD, presented highlights from the Society’s latest Landscape Report on Cell, Gene and RNA Therapy for the first quarter (Q1) of 2026.

    The ASGCT report is developed in conjunction with Citeline, a subsidiary of Norstella (a pharmaceutical intelligence provider covering drug development from preclinical to commercialization).

    Barrett said there are currently 42 gene therapies approved worldwide, along with 38 RNA therapies and 76 (non-genetically modified) cell therapies, which are steadily growing the field. Two cell therapies were approved in Japan in Q1.

    There was a small increase in deal-making, and a significant 30% increase in startup funding compared to the same period in 2025. “I think that signals and underscores a rebounding sector,” said Barrett.

    Of the eight gene therapies approved over the past 12 months, half were in the United States, with three more in China. “The regulatory pace is starting to pick up, another strong indicator for the future of our field,” Barrett said. It is a similar picture in RNA therapies. “We see a steady uptick over the course of the last year,” he added.

    Zooming out, Barrett estimated that there are more than 4,200 therapies currently in development, from preclinical through pre-registration. The vast majority of those (more than 4,130) are gene and genetically modified cell therapies, including about 1,300 RNA therapies.

    In the field of gene-modified cell therapies, CAR T continues to lead the pipeline for ex vivo gene therapies, with natural killer (NK) and T-cell receptors gaining traction. Not surprisingly, genetically modified cell therapy overwhelmingly targets cancers, but Barrett noted growth in the percentage of these therapies targeting immunological diseases, including lupus, multiple sclerosis, and HIV.

    Pipeline growth

    Barrett also noted growth and “a promising future” in the clinical trials pipeline. There are currently 350 Phase I, 319 Phase II, and 41 Phase III trials in gene therapy (up from 35 a year ago). “Hopefully, we will see a number of completed trials and FDA decisions in the near term,” said Barrett. A growing proportion of gene therapy trials (exceeding 60 percent) is for non-oncology indications.

    In the RNA space, “RNAi therapies are jumping,” said Barrett. The same cannot be said, however, for mRNA. “Unsurprisingly, mRNA therapies continue to slide quarter over quarter,” a symptom of “shaken confidence” in that space, he continued. RNA therapies are targeting primarily non-oncology indications, especially in rare diseases.

    Upcoming catalysts

    On the business front, Barrett noted there has been “a nice uptick” in Q1 in start-up funding compared to the same quarter last year, which he deemed “a really promising indication.” The number of start-ups historically has tended to hover between 5-20. For Q1, that number was 26.

    The Q1 report tracks various business catalysts anticipated through the end of 2027, including increased interest and uptake in expedited review designations—fast track, RMAT, orphan drug breakthroughs and other accelerated approval pathways.

    “FDA is getting a lot done… and hopefully we’ll see the same moving forward,” Barrett said.

     

    The full Landscape Report is available online from the ASGCT website.

    The post ASGCT Q1 Landscape Report Paints Positive Picture for Gene and RNA Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Multiomic ALS Study Links Peripheral Immune Infiltration to CNS Inflammation https://edusehat.com/en/multiomic-als-study-links-peripheral-immune-infiltration-to-cns-inflammation https://edusehat.com/en/multiomic-als-study-links-peripheral-immune-infiltration-to-cns-inflammation Sat, 16 May 2026 04:05:09 +0700 Edusehat Multiomic, ALS, Study, Links, Peripheral, Immune, Infiltration, CNS, Inflammation A new study from scientists at Northwestern University Feinberg School of Medicine sheds light on how amyotrophic lateral sclerosis (ALS) unfolds in the body. Specifically, they found that the disease proceeds through a “domino-like” sequence of events that begins with an early breakdown inside motor neurons that is followed by a damaging inflammatory response. Insights from this study could help explain why the disease worsens over time, why some patients progress faster than others, and how future treatments could be more personalized. Details of the work are available in a new Nature Neuroscience paper titled “Integrated single-cell and spatial transcriptomic profiling in ALS uncovers peripheral-to-central immune infiltration and reprogramming.”

    On average, patients with ALS live three years after symptoms begin, although some can survive closer to 10 years. Exactly what drives these differences in survival is unclear. “This study reveals that ALS is not a single event but a domino-like cascade that begins inside motor neurons with TDP-43 pathology and is then amplified by a damaging immune response in the bloodstream and spinal cord,” said David Gate, PhD, director of the Abrams Research Center on Neurogenomics at Feinberg and co-corresponding author on the study. 

    Specifically, the study found that immune cells converge at sites of motor neuron loss and TDP-43 pathology with distinct inflammatory patterns depending on the type of ALS and how quickly the disease progresses. As Evangelos Kiskinis, PhD, an associate professor of neurology and neuroscience at Feinberg and a co-corresponding author on the study, explained it, “the intensity of spinal cord inflammation” determines “how fast the disease progresses and how long they survive.” 

    To gain these insights, the scientists analyzed blood and spinal cord samples from living and deceased patients with both genetic and non-genetic forms of ALS, as well as controls. As part of the study, they used single-cell RNA sequencing technology to analyze blood from 40 living ALS patients and used spatial transcriptomics to analyze spinal cord tissue from 18 deceased participants. They also compared patients with non-genetic ALS to those with the genetic form of the disease to assess how immune activity differs across ALS types and disease stages. Lastly, they examined RNA from postmortem samples of 237 ALS patients to better understand the inflammatory responses within the central nervous system. 

    Using these methods, “we found the immune cells we detected in the blood of people living with ALS were inflamed, and we found the genes that mediate their inflammatory response in the spinal cord at the site of motor neurons,” Gate said. “These inflamed immune cells were associated with ALS pathology, giving some credence to our theory that the immune system is detrimental. It’s responding to pathology, and it’s causing the disease to be worse.”

    Additionally, patients whose disease advanced quickly had more activity in certain immune genes, while those with the genetic form of the disease had a different set of altered immune genes. In the spinal cord, these activated immune cells gathered directly at the locations of motor neuron loss and near the toxic protein buildups associated with ALS. “We saw that people with worse clinical ALS had more expression of complement genes, which are proteins that become activated as the body’s first-line immune defense against a pathogen or damage to the body,” Gate said.

    Now that they have identified a direct link between the immune system and ALS, Gate and his lab plan to study samples from a wider pool of patients. “Our next step is to map exactly how this immune reaction spreads throughout the entire motor circuit: from the brain, down through the spinal cord and out to the muscles,” he said. “By profiling the motor circuit in depth, we’ll get a much clearer picture of where and when inflammation drives faster progression.” 

    Meanwhile, Kiskinis and his team will test for a causal relationship between TDP-43 dysfunction and inflammation. “We’re trying to really define what is the mechanism that links TDP-43 dysfunction in nerve cells with inflammatory reactions,” he said.

    The post Multiomic ALS Study Links Peripheral Immune Infiltration to CNS Inflammation appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     RAGE Implicated in Worsening Breast Cancer Mortality with Age https://edusehat.com/en/rage-implicated-in-worsening-breast-cancer-mortality-with-age https://edusehat.com/en/rage-implicated-in-worsening-breast-cancer-mortality-with-age Sat, 16 May 2026 04:05:08 +0700 Edusehat RAGE, Implicated, Worsening, Breast, Cancer, Mortality, with, Age Researchers at Georgetown’s Lombardi Comprehensive Cancer Center have identified a mechanism that may help to explain why older people experience worse outcomes from breast cancer. The study in different mouse breast cancer models and in human breast cancers implicates RAGE (receptor for advanced glycation end-products), a cell surface receptor that amplifies inflammatory signaling, and which also becomes increasingly active with metastatic progression. The study findings in addition suggested that inhibiting RAGE may offer a well-tolerated adjunctive breast cancer therapy in older patients.

    “Our study addresses a major gap by showing that aging dramatically increases breast cancer metastasis and that this effect depends on RAGE, a receptor on the surface of cells that fuels inflammation,” said Barry Hudson, PhD, associate professor of oncology at Georgetown Lombardi. “Most laboratory studies rely on young mice, which has limited our understanding of how aging itself alters the host environment, including immune function and chronic inflammatory states that, in turn, influence cancer behavior.” Hudson is corresponding author of the researchers’ Communications Biology published paper titled “Aging promotes a RAGE-dependent increase in breast cancer metastasis.” In their paper the authors concluded that their findings “… identify RAGE as a mechanistic link between aging and metastasis and a potential therapeutic target in older patients.” They say the findings will also be featured in the Nature portfolio special collection, Cancer and Aging.

    Age is the primary risk factor for the development of adult cancers, including breast cancer, with almost half of new breast cancer diagnoses and more than half of breast cancer-specific deaths occurring in women aged 65 years and older, the authors wrote. And while advances in screening and therapy have improved survival, older women continue to have higher breast cancer-specific mortality. “Despite accumulating evidence that metastasis in murine breast cancer models increases with advancing host age, the mechanisms underlying this have not been elucidated, highlighting the need for further mechanistic studies,” the team continued.

    And while breast cancer is more prevalent in older women, most cancer research in mouse models has used young, 2–3-month-old adult mice, which are about equivalent in age to 15–20-year-old humans. Timing and chance presented Hudson and colleagues with opportunities to carry out their newly reported study. During COVID, when there was reduced laboratory activity, some of the research team’s mouse colonies aged longer than originally planned. This created a rare opening to study cancer in these older animals—normally a difficult and expensive endeavor—giving the scientists the ability to directly compare how tumors behave in younger versus older mice.

    RAGE is a proinflammatory molecule that is being considered as a therapeutic target in multiple aging-related diseases, including various cancers, cardiovascular and neurodegenerative diseases.

    Using three different mouse models of triple-negative breast cancer (TNBC), the researchers discovered that aged mice developed substantially more lung metastases than younger mice, despite similar primary tumor growth. The team then showed that genetic deletion of RAGE in mice almost completely eliminated this age-related surge in metastasis.

    Through their studies, the team demonstrated that aging increased levels of inflammatory molecules that activate RAGE. These included the proteins S100 and HMGB1, found in both primary tumors and at metastatic sites. These changes made it easier for cancer cells to invade and spread. “These findings show that aging doesn’t just increase cancer risk—it actively changes the body in ways that help tumors spread,” said Hudson. “RAGE appears to be a key mediator of these harmful age-related pathways.” In their paper the authors stated that their data “… suggest that aging promotes multiple prometastatic processes within the tumor and its microenvironment, and that RAGE is required for the induction of these inflammatory and tumor-promoting pathways in aged hosts.“

    The team also analyzed breast cancer data from more than 1,000 patients and found that higher expression of AGER (the gene encoding RAGE) and related inflammatory gene signatures were associated with worse outcomes in patients, supporting the clinical relevance of their findings. They noted, “… in human breast cancers, high AGER expression, as well as enrichment for mouse tumor-derived aging- and RAGE-associated gene signatures, predicted poorer outcomes, particularly in older women …Together, these data indicate that in older individuals with breast cancer, intratumor RAGE overexpression amplifies aging-associated transcriptional programs, linking age-dependent inflammation to promote metastatic progression.”

    RAGE is already being explored as a therapeutic target in several age-related diseases, highlighting its potential relevance in cancer. In prior work, the researchers had shown that the RAGE inhibitor TTP488 (azeliragon) can suppress breast cancer metastasis in preclinical models. In the current study, they also tested the drug in the lab and found that TTP488 was able to reduce tumor cell invasiveness that was induced by blood sera from aged mice.” Pharmacologic inhibition of RAGE by TTP488 (PF-04494700 or azeliragon) suppressed migration and invasion towards aged serum, further supporting the requirement of RAGE signaling for age-dependent metastasis,” the team noted.

    A clinical study is underway at Lombardi evaluating TTP488 in breast cancer patients receiving chemotherapy, with a focus on safety and cognitive outcome. The drug has demonstrated a favorable safety profile in people, making it an optimal choice for further study. “TTP488 has demonstrated an excellent safety profile in Phase I/II clinical studies in older adults with Alzheimer’s disease, supporting its potential for repurposing,” the authors wrote. “Therapeutic RAGE inhibition may provide a well-tolerated means to counteract inflammaging and improve cancer outcomes in the elderly, who often face limited treatment options due to toxicity,” the investigators wrote.

    “This study highlights the importance of the host environment in cancer,” Hudson added. “While cancer is often viewed as driven primarily by mutations intrinsic to tumor cells, systemic factors such as aging and inflammation play a critical role in shaping how cancers behave,” said Hudson. “Most deaths due to cancer occur because tumors spread to other organs, so understanding these influences may help identify new strategies to limit metastasis.”

    The post RAGE Implicated in Worsening Breast Cancer Mortality with Age appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Exaggerated MFN cost-saving estimates imperil U.S. innovation and investment https://edusehat.com/en/exaggerated-mfn-cost-saving-estimates-imperil-us-innovation-and-investment https://edusehat.com/en/exaggerated-mfn-cost-saving-estimates-imperil-us-innovation-and-investment Sat, 16 May 2026 00:35:04 +0700 Edusehat Exaggerated, MFN, cost-saving, estimates, imperil, U.S., innovation, and, investment The Administration’s Council of Economic Advisors (CEA) has released an analysis of savings expected from its “Most Favored Nation” deals with pharmaceutical manufacturers, but the methodology and assumptions raise serious questions about the findings. 

    The report looks at the 10-year impact of its MFN policies, ignoring that the current MFN deals are only three-year agreements.

    Notably, the assessment bakes in two contradictory ideas. It projects an environment where prices for new medicines in the United States drop by 30% within a 10-year period while also asserting that innovation will be unaffected.

    But such a dramatic reduction in prices would lead, inevitably, to less money available for research and development, and the report offered no details on how that deficit might be filled.

    This has prompted widespread skepticism about the findings:

    • “There’s just no way to verify it by the information that’s public so far,” Washington University law professor Rachel Sachs told STAT News.

    There are other concerns with the data, too. Projected savings in Medicaid are likely exaggerated because the report didn’t assess actual Medicaid discounts but rather used a crude underestimate of existing price concessions. And the report frequently compared TrumpRx prices to list prices, rather than the actual prices that patients might pay through other cash-pay channels.

    BIO’s View: The report claims “a net positive effect for incentives to innovate,” but a closer look at the numbers that the authors used—and the assumptions underlying those numbers—suggests something closer to wishful thinking. Other countries massively undervalue innovation, which is why European companies had 44% of the world’s clinical trials in 2009 and only 21% by 2024.

    Importing those prices will not make medicines more affordable for Americans; it will, however, undermine a sector that has delivered breakthroughs to patients while fueling economic growth in research and manufacturing from coast to coast. It’s time to simplify the system — with smarter solutions that ensure medicines are accessible and more affordable for American patients.

    The post Exaggerated MFN cost-saving estimates imperil U.S. innovation and investment appeared first on Bio.News.

    ]]>     The world is on track to miss its health targets https://edusehat.com/en/the-world-is-on-track-to-miss-its-health-targets https://edusehat.com/en/the-world-is-on-track-to-miss-its-health-targets Fri, 15 May 2026 21:00:06 +0700 Edusehat The, world, track, miss, its, health, targets Every year the World Health Organization publishes a global health statistics report. It features the numbers behind world health trends and, importantly, assesses whether we’re on track to reach ambitious goals set in 2015. It’s a bit like a health grade.

    The 2026 report was published on Wednesday. And the results aren’t looking brilliant. While we are seeing some improvements, they are uneven, and they’re far too slow.

    The targets themselves are part of the United Nations’ Sustainable Development Goals, a sprawling and ambitious plan focused on improving life around the world. The 17 goals were set to tackle poverty and climate change and to boost education, gender equality, health, and well-being, among many other quality of life issues. Those targets were meant to be met by 2030.

    Perhaps they were a little too ambitious. Here are the numbers and statistics that stood out to me on this year’s world health report card.

    1.3 million new cases of HIV in 2024

    Before the SDGs, there were the Millennium Development Goals. One MDG target was to halt and reverse the spread of HIV—and that target was exceeded by 2015. Back then, we were considered on track to “end the AIDS epidemic by 2030.”

    How depressing, then, to see that in 2024 there were an estimated 1.3 million new cases of HIV. That’s 40% lower than the figure from 2010. But it’s still 1.3 million additional people with HIV. The SDG target is to reduce HIV incidence by 90% by 2030—we’re not likely to meet it.

    10.7 million new cases of TB

    The picture is even bleaker for tuberculosis, which ranks 10th on the WHO’s list of top global causes of death. The goal was to reduce cases by 80% between 2015 and 2030. So far, cases have only fallen by a measly 12%. And when you break the change down by region, the Americas saw an increase of 13%

    An 8.5% rise in malaria cases

    And then there’s malaria, the mosquito-borne disease with a 7% fatality rate. The European region has been free of malaria since 2015, but the disease is a significant concern in many countries in the Global South, particularly in Africa. The goal was to lower rates by 90% between 2015 and 2030. In 2024, there were an estimated 282 million cases of malaria globally—representing an 8.5% increase in incidence rates.

    Antimalarial drug resistance is a major challenge here—forms of the malaria virus that are resistant to drugs have been confirmed or suspected in eight countries in Africa, according to a separate WHO report. Mosquitoes that are resistant to commonly used insecticides are present in nine African countries. And climate change, which can alter mosquito habitats, may be making things worse.

    42.8 million children are wasting

    We’re not meeting child health targets, either. Take malnutrition, for example. As of 2024, the global prevalence of wasting in children was 6.6%—that’s a staggering 42.8 million children who are literally wasting away because of a lack of adequate food. On the other end of the spectrum, 5.5% of children are now considered overweight. Both figures were meant to be below 5% by 2030, which now seems unlikely.

    Vaccination rates are dropping in the Americas

    Progress in improving childhood vaccination coverage has stalled. Globally, an estimated 76% of children are getting their second dose of a measles vaccine—a figure far below the the approximately 95% needed to prevent outbreaks. The Americas currently has lower rates of vaccine coverage for three of the four “core” vaccines than it did in 2015.

    This is partly due to a lack of investment, says Goodarz Danaei, an epidemiologist at the Harvard T.H. Chan School of Public Health. “But now we have a misinformation campaign going around vaccines that makes it worse,” he adds.

    The covid-19 pandemic didn’t exactly help, either. The impact on health services led to millions of children missing out on routine vaccinations.

    22.1 million pandemic-related deaths

    And of course the pandemic affected progress toward health goals in more direct ways: 7 million people died of covid-19. The WHO report estimates that, for each of these, there were an additional two “excess” deaths related to the pandemic, due to disruptions in health care, for example. That puts the total figure at 22.1 million pandemic-related deaths.

    A woman dies every two minutes from “maternal causes”

    Maternal mortality rates fell by about 40% between 2020 and 2023. But today’s rate equates to 712 maternal deaths every single day. That’s one every two minutes. The WHO report notes that we’d have to reduce the mortality rate by almost 15% per year in order to meet the 2030 target. This seems incredibly unlikely, particularly given the recent decimation of US funding for global aid programs, which is expected to result in thousands of additional maternal deaths.

    Progress has also slowed in reducing the risk of death from noninfectious diseases like cancer, diabetes and cardiovascular disease. “Overall, neither the world nor any WHO region is currently on track to meet the 2030 SDG target,” the report states.

    2.1 billion people struggle to afford health care

    Despite plans to make health care more affordable, a significant chunk of the population is being pushed into poverty by health-care costs. In 2022, 2.1 billion people faced financial hardship due to health spending—and 1.6 billion of them were living in or had been pushed into poverty.

    Across the board, there have been some important improvements in global health. But the achievements have not gone far enough. “The good news is that there is progress,” says Danaei. “But as always, the glass is half empty.”

    This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.

    ]]>     DNA‑Guided CRISPR Suggests a New Direction for RNA Editing https://edusehat.com/en/dnaguided-crispr-suggests-a-new-direction-for-rna-editing https://edusehat.com/en/dnaguided-crispr-suggests-a-new-direction-for-rna-editing Fri, 15 May 2026 20:55:16 +0700 Edusehat DNA‑Guided, CRISPR, Suggests, New, Direction, for, RNA, Editing CRISPR’s rise from obscure bacterial defense system to molecular scalpel has always hinged on one small component: the guide RNA. For years, that guide RNA—meticulously designed, modified, and optimized in countless labs—has been treated as an immutable feature of the system. CRISPR cuts where the RNA tells it to cut. That’s the central dogma of the system.

    But a new approach suggests the system is more flexible than anyone expected. The study, published in Nature Biotechnology, is titled “DNA-guided CRISPR–Cas12 for cellular RNA targeting.”

    Researchers at the University of Florida (UF) have developed the first CRISPR system that uses DNA, rather than RNA, to direct Cas enzymes to RNA targets. The platform, called ΨDNA, reprograms Cas12 nucleases to recognize and act on RNA using a DNA-based guide scaffold. The result is a fundamentally different way of controlling RNA inside cells—one “that extends Cas12 systems beyond genome editing and diagnostics to enable precise, programmable control of cellular transcriptomes and their epitranscriptomic marks,” according to the authors.

    The concept is rooted in a simple biological distinction. DNA stores the cell’s long-term instructions, but RNA carries the working copies. “Those RNA copies are like Xerox copies of the original manual, and sometimes those copies have errors,” said Piyush Jain, PhD, associate professor of chemical engineering at UF and lead author of the study. Errors in those working copies can drive disease, and targeting RNA offers a way to intervene without altering the underlying genome. But RNA‑guided CRISPR systems, such as Cas13, can suffer from instability and off‑target effects. “Existing RNA-targeting CRISPR systems rely on RNA guides to find their targets,” Jain said. “While effective, they can sometimes affect unintended molecules… They can also be costly and less stable.”

    ΨDNA takes a different approach. The team engineered a DNA guide that mimics the crRNA scaffold in reverse orientation, enabling AsCas12a and Cas12i1 to bind RNA and trigger strong single‑stranded DNA trans‑cleavage. As the abstract describes, “ΨDNA… enables RNA targeting by Cas12 nucleases… including 100% accurate hepatitis C virus RNA detection in clinical samples.” In human cell lines, ΨDNA achieved 70–95% knockdown of endogenous RNA transcripts, driven by mechanisms such as ribosome stalling and RNase H1 recruitment.

    Jain sees the work as a conceptual shift for CRISPR. “The most meaningful advance is that we show CRISPR‑Cas12 can be reprogrammed to target RNA using a DNA guide rather than an RNA guide,” he told GEN. “That is a real conceptual shift for the field.” Until now, RNA targeting has been dominated by RNA‑guided systems. ΨDNA demonstrates that Cas12 enzymes—traditionally DNA editors—can be redirected toward RNA “while preserving strong specificity and enabling multiple functions, including RNA detection for developing diagnostics, intracellular knockdown, multiplex targeting, dual DNA and RNA targeting, and effector fusion strategies for RNA modification and potential therapeutic strategies.”

    The discovery emerged from a structural puzzle. Simply swapping RNA bases for DNA bases does not work; Cas12 enzymes are thought to be tightly dependent on RNA scaffolds. “Several groups have tried to achieve DNA-guided CRISPR/Cas, but simply converting RNA bases to DNA bases doesn’t work,” Jain said. The breakthrough came from engineering a 3′ DNA handle that recreated the crRNA scaffold. Mutational screening revealed that a stem‑loop architecture was essential for activity, and recent cryo‑EM structures—solved in collaboration with David Taylor’s group at UT Austin—showed that AsCas12a has more structural flexibility than expected, allowing it to accommodate a DNA guide bound to an RNA target.

    What surprised the team most was how robust the system proved to be. “It was especially exciting to see that this was not just an in vitro curiosity,” Jain said. ΨDNA worked in clinical RNA detection, achieving 100% accuracy on hepatitis C virus samples, and functioned inside cells with lower off‑target effects than Cas13d.

    The platform’s modularity may be its most powerful feature. ΨDNA can be fused to RNase H1 for targeted RNA degradation or to METTL3 for epitranscriptomic editing. And because crRNA and ΨDNA can be codelivered, a single Cas12a enzyme can operate in two modes at once. “A single Cas12a effector can simultaneously edit DNA and regulate RNA,” Jain said. “This work starts to blur that boundary.”

    Looking ahead, the team is expanding both the mechanistic and translational sides of the platform. They are refining guide design rules, dissecting how ΨDNA‑guided Cas12 triggers knockdown, and exploring applications in diagnostics, multiplex RNA regulation, and ex vivo therapeutic settings. One emerging direction involves using the technology to repair donor organs before transplantation.

    More broadly, DNA guides offer practical advantages. They are easier to synthesize, more stable, and potentially more scalable than RNA guides. That combination could make ΨDNA a versatile platform for basic research, diagnostics, and future therapeutic engineering.

    After decades of CRISPR research built around RNA‑guided systems, ΨDNA introduces a new way to direct one of biology’s most powerful tools. As Jain put it, “At its core, this is about giving us better control—not just rewriting the instruction manual but also precisely managing how those instructions are used.”

    The post DNA‑Guided CRISPR Suggests a New Direction for RNA Editing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ASGCT 2026: Rare Instance of AAV Integration into Human Genome Linked to Brain Tumor https://edusehat.com/en/asgct-2026-rare-instance-of-aav-integration-into-human-genome-linked-to-brain-tumor https://edusehat.com/en/asgct-2026-rare-instance-of-aav-integration-into-human-genome-linked-to-brain-tumor Fri, 15 May 2026 20:55:14 +0700 Edusehat ASGCT, 2026:, Rare, Instance, AAV, Integration, into, Human, Genome, Linked, Brain, Tumor Rebecca Ahrens-Niklas
    Rebecca Ahrens-Niklas, MD, PhD

    BOSTON — A team at Children’s Hospital of Philadelphia (CHOP) led by Rebecca Ahrens-Niklas, MD, PhD, and Lindsey George, MD, has described a case of a brain tumor linked to a rare integration of adeno-associated virus (AAV).

    George presented the work at the American Society of Gene and Cell Therapy (ASGCT) conference in a plenary talk selected as the “presidential abstract” by ASGCT president, Terry Flotte, MD. The study, “Neuroepithelial tumor with AAV integration after intracisternal magna vector delivery,” was published in the New England Journal of Medicine.

    Lindsey George
    Lindsey George, MD

    Over the past 25 years, some 6,000 patients have been treated with some form of AAV gene therapy. In all that time, George said, there have been no established long-term safety concerns, although genome integration events have been reported in mouse and dog studies. But the case documented by George and colleagues at CHOP suggests that the gene therapy field may need to pay more attention to this potential occurrence.

     

    The story began with a 5-year-old boy with an inherited lysosomal disorder, severe MPS1 deficiency (Hurler subtype). The patient received enzyme replacement therapy at six weeks of age, followed by a cord blood stem cell transplant at age four months.

    Investigators chose to perform gene therapy when the patient was 13 months old to deliver the iduronidase (IDUA) gene. The vector chosen was an AAV9 serotype, using a cytomegalovirus enhancer and a chicken beta-actin promoter driving the gene. The virus was administered into the boy’s cisterna magna in the base of the skull.

    When the boy was five years old, a routine neurological scan revealed a large intraventricular mass that had not been observed two years earlier. Analysis of the tumor revealed it was a PLAG1-driven neuroepithelial tumor—indeed, PLAG1 expression was almost 300 times higher than in other central nervous system tumors studied at CHOP. (PLAG1 is usually only expressed during embryogenesis.)

    Surgery to remove the tumor was successful. Eight months after surgery, there are no signs of tumor growth. The boy is also showing advanced neurocognitive function.

     

    Tumor typing

    George described RNA sequencing of the tumor, which revealed the fusion of a fragment of the AAV9 vector cassette to exon 5 of the PLAG1 gene on chromosome 8. The resulting transcript is predicted to encode a PLAG1 derivative containing five zinc-finger DNA-binding domains and a C-terminal transcriptional activation domain, which was previously reported to function as a transcriptional activator.

    Adeno-associated virus
    Credit: Dr_Microbe / iStock / Getty Images Plus

    Curiously, the chimeric junction also included a segment of human chromosome 10, which George suspects originated during the vector manufacturing process. The integration event was present in about 40% of the total reads, suggesting integration into one of the two PLAG1 alleles.

    George concluded her talk by noting that while the clinical outcome in this patient is so far encouraging, this is evidence that AAV integration can be associated with oncogenesis. The study underscores the need to monitor the most heavily transduced tissues after AAV gene therapy.

    While the gene therapy community should be cautious in extrapolating this single case report across all AAV gene therapy programs, George said the study supports the use of the lowest feasible vector dose as well as tissue-specific promoters.

    A) Timeline of the patient’s medical history; B) Diagram of AAV gene therapy cassette. [The New England Journal of Medicine ©2026]
    George noted that detection of the integrated AAV vector DNA was challenging, in part because of rearrangements of vector DNA. The use of several complementary techniques—long-read DNA sequencing, targeted PCR amplification, and RNA sequencing—was required to confirm the integration.

    George and coworkers closed their paper, noting that, “Our findings support the hypothesis that rare AAV integration can contribute to human oncogenesis, which emphasizes the need to optimize gene delivery methods and monitor transduced tissues after treatment.”

    The post ASGCT 2026: Rare Instance of AAV Integration into Human Genome Linked to Brain Tumor appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ASGCT 2026: AI-Optimized Cas12l Gene Editor Offers Compact Cas9 Alternative https://edusehat.com/en/asgct-2026-ai-optimized-cas12l-gene-editor-offers-compact-cas9-alternative https://edusehat.com/en/asgct-2026-ai-optimized-cas12l-gene-editor-offers-compact-cas9-alternative Fri, 15 May 2026 20:55:12 +0700 Edusehat ASGCT, 2026:, AI-Optimized, Cas12l, Gene, Editor, Offers, Compact, Cas9, Alternative BOSTON — In a potentially significant advance for the genome editing field, researchers from the biotechnology company Caszyme and the Vilnius University Institute of Biotechnology in Lithuania have developed a potent and compact variant of Cas12l nuclease. Giedrius Gasiūnas, PhD, Caszyme co-founder and CEO, presented highlights of the research at ASGCT.

    The work represents “a great example of the potential of continued mining for novel Cas effectors within the bacterial metagenomic diversity dark matter,” said Rodolphe Barrangou, PhD, Editor in Chief of The CRISPR Journal, which will shortly be publishing a paper on the Lithuanian team’s results.

    “We need more diverse effectors to address the technical shortcomings of the CRISPR toolbox,” Barrangou continued. “This study is a great illustration of the potential of mining bacterial diversity.”

    Giedrius Gasiūnas, PhD, Caszyme CEO, presents Cas12l at ASGCT 2026.

    The Lithuanian team, including veteran gene editor Virginijus Siksnys, PhD—winner of the 2018 Kavli Prize with Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD, for CRISPR gene editing—used a hybrid approach to optimize Cas12l. By combining cryo-electron microscopy (cryo-EM) structure-guided design with artificial intelligence (AI) protein language models, the team was able to engineer a variant (Asp2Cas12l M82) that overcomes the known efficiency limitations of the Cas12l family.

    Although Cas9 has widespread utility, including clinical applications, researchers have long considered its relatively large size and requirement for G-rich protospacer adjacent motifs (PAMs) problematic. The Cas12l family, discovered in the Armatimonadota bacterial phylum, offers a more compact size (867 amino acids) and recognition of a C-rich PAM site.

    But wild-type Cas12l enzymes exhibit lower editing efficiencies and higher target-to-target variation compared to Cas9. According to Gasiūnas, the new M82 variant is “reliable, precise and adaptable,” and shows promise for a wide range of therapeutic applications.

    “Through our continued work exploring novel Cas systems, Caszyme is focused on advancing technologies that move beyond promise into practical use.”

    Path to potency

    The engineering of the M82 variant proceeded in two steps. First, the Caszyme researchers solved the 3D structure of Asp2Cas12l complexed with an sgRNA and DNA to high resolution (2.51 Å). This revealed a unique “bracelet” architecture whereby the nuclease encircles the DNA target via interlocking helical bundles and a proline-rich string.

    Next, the team introduced arginine substitutions at dozens of positions in the molecule to enhance electrostatic attraction to the negatively charged DNA backbone. This work included the production of an M67 variant, which provided a 7-fold improvement in indel editing over the wild-type nuclease.

    To engineer further refinements, the Caszyme group turned to AI, specifically the ESM-2 protein large language model. This model predicted evolutionary hotspots considered likely to preserve or enhance function. Integrating these AI-derived substitutions—Q572R in the bridge helix and F607S in the RuvC domain—resulted in the final M82 Cas12l variant, illustrating the value of AI-supported engineering rather than deploying protein-directed evolution.

    Rivaling Cas9

    Gasiūnas presented data showing that M82 possesses good activity across recalcitrant gene targets, reducing the target-to-target variation that plagues many novel nucleases. In head-to-head comparisons in HEK293T cells, M82 demonstrated an average indel editing rate of 67.4%, nearly identical to that of Cas9 at overlapping target sites. This potency was consistently maintained across several delivery formats, including plasmid DNA, mRNA, and ribonucleoprotein complexes.

    The Caszyme group also showed excellent M82 efficiency in homology-directed repair (HDR). In experiments targeting the AAVS1 locus, M82 facilitated a site-specific gene insertion frequency of 39%, outperforming Cas9 in the same context. Using single-stranded donor templates, HDR rates reached as high as 56%. Gasiūnas suggested that the staggered cut produced by Cas12l may inherently steer DNA repair toward precise correction rather than stochastic indels. With regard to safety, Caszyme found that M82 Cas12l maintained a high degree of on-target precision. Secondary editing signals were largely detected at or near the lower limits of assay sensitivity, suggesting a low risk of off-target cleavage.

    The compact size of the M82 variant makes it an attractive candidate for adeno-associated virus-mediated delivery, which has strict limits on cargo size. “It is no secret that the CRISPR space has faced challenges and concerns in recent years,” Gasiūnas said. “However, we are confident in M82’s ability to create headroom for scientists to stand up and innovate within.”

    Crowded field

    Cas12l is not the only compact Cas nuclease gaining attention, of course. In a talk preceding Gasiūnas’ presentation, Zhaoshi Wu, PhD, co-founder and chief technology officer of Shanghai-based Castalysis Bioscience, presented an update on Cas12n, details of which were first published in Molecular Cell in 2023. The nuclease was touted as being the first independent CRISPR-Cas complete gene family uncovered by Chinese scientists within China’s territory.

    Touted as a next-gen ultra-compact gene editing system, Cas12n (branded as alphaCas) consists of just 450 amino acids, and possesses structural similarity to TnpB. Cryo-EM structural analysis led the Chinese investigators to optimize the molecule for non-viral in vivo delivery. Preclinical experiments showed robust genome editing in a mouse model by targeting PCSK9 using lipid nanoparticle delivery, resulting in sharp drop in serum LDL levels.

    Wu said his company is on target to begin its first clinical before the end of 2026. But he faced an uncomfortable moment during audience questions. Fyodor Urnov, PhD, challenged Wu’s claim that an inherent advantage of Cas12n was its safety profile compared to Cas9. Urnov pointed out that Intellia Therapeutics has two ongoing Phase III in vivo trials using CRISPR-Cas9 that show no immunogenicity concerns using LNP delivery.

    Urnov later congratulated Wu on the rest of the company’s data and wished them success.

    The post ASGCT 2026: AI-Optimized Cas12l Gene Editor Offers Compact Cas9 Alternative appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ASGCT 2026: Beverly Davidson Offers Vehicle and Route for Huntington’s Disease Gene Therapy https://edusehat.com/en/asgct-2026-beverly-davidson-offers-vehicle-and-route-for-huntingtons-disease-gene-therapy https://edusehat.com/en/asgct-2026-beverly-davidson-offers-vehicle-and-route-for-huntingtons-disease-gene-therapy Fri, 15 May 2026 20:55:11 +0700 Edusehat ASGCT, 2026:, Beverly, Davidson, Offers, Vehicle, and, Route, for, Huntington’s, Disease, Gene, Therapy BOSTON – Geneticist Beverly Davidson, PhD, received the 2026 Outstanding Achievement Award from the American Society of Gene and Cell Therapy (ASGCT). Davidson is currently the chief scientific strategy officer at the Children’s Hospital of Philadelphia (CHOP) and a former president of ASGCT.

    Some of the research Davidson presented was conducted at a new biotech company she co-founded called Latus Bio, which earlier this month announced it had raised $97 million in a Series A round. The company develops novel AAVs to specifically target central nervous system (CNS) disorders, with a lead program in Huntington’s disease (HD).

    After thanking her mentors—Bill Kelly, MD, Michael Welsh, MD, and Kathy High, MD—Davidson turned her attention to presenting new advances in engineered gene therapies. Throughout her career, she has focused on improving adeno-associated viruses (AAVs) for CNS gene therapies, with a particular emphasis now on HD. Key elements include selecting the right cargo and developing the appropriate delivery vehicle. Her goal is to scale lab research in neurons, mouse models, and non-human primates (NHPs) to treat patients, including adults with HD.

    Major hurdles to tackling genetic diseases of the brain include scalability and a lack of potency, Davidson said. The search for alternative AAV serotypes to AAV2 that could target neuronal cells began back in 2000. IV administration does not provide sufficient targeting to the brain. Even AAVs that have been engineered to enter the brain from the blood have high peripheral exposure and a high cost of goods per patient, which significantly lowers scalability and impact. (In one study, liver biodistribution of AAV was many orders of magnitude higher than in the CNS.)

    Davidson focused on HD, the late-onset, dominantly inherited genetic disease. The identification of the gene harboring the HD mutation in the early 1990s by a consortium of researchers was one of the biggest success stories in human genetics. Even more remarkable was the underlying disease mechanism—the expansion in exon 1 of the gene of a triplet repeat sequence (CAG) producing an abnormally long string of glutamine residues in the huntingtin protein.

    The right target

    One of the major challenges in devising a gene therapy for HD is ensuring that the therapeutic reaches the right network—the deep brain and cortical areas. Therapies have to reach the right circuit, and the right cells in those circuits, Davidson said. Over the years, her group has tailored AAVs for delivery to the brain, inserting peptides into exposed loops of the virion to allow for targeting and unbiased diversity for blood-to-brain delivery. Nowadays, she said, machine learning approaches can be applied for further capsid improvements.

    Davidson’s CHOP lab developed a method for screening AAVs with enhanced potency for CNS therapies. After generating huge libraries containing tens of millions of novel capsids, the group performed serial enrichments to identify the most attractive capsids. After screening pools of injected capsids into two species of monkeys, a winning capsid emerged: AAV-DB-3.

    Davidson’s group infused AAV-DB-3 into NHPs, looking for targeting to the putamen (base of the forebrain) and caudate regions. Those results were published in Nature Communications in 2025.  “AAV-DB-3 really stood out for its ability to transduce deep layer cortical neurons that are important” in HD, Davidson said. Moreover, the results were achieved with relatively low doses and only required a single infusion per hemisphere, outperforming the widely used AAV5.

    Somatic instability

    With a promising delivery vehicle identified, Davidson next addressed the therapeutic strategy, which takes aim at the somatic expansion of the CAG repeat. This codon grows longer over time in certain cells in the brain, sometimes expanding to hundreds of repeats.

    MSH3 is a DNA repair protein that is required for CAG repeat expansions, as seen in mouse models of HD and other triplet repeat disorders, including myotonic dystrophy. Research led by Paul Ranum, PhD, who is a co-founder of Latus Bio, posted in a preprint on bioRxiv earlier this year, modeled the impact of lowering levels of MSH3 on somatic instability.

    Ranum and colleagues used an artificial microRNA showed to lower MSH3 levels in NHPs by 48-94 percent. Computational modeling suggests that this would reduce somatic instability and delay onset of HD symptoms by many years. Early studies using a well-known HD mouse model, the Q111 mouse, to assess biodistribution, quantify knockdowns, and assess the impact on somatic CAG repeat expansion. AAV-DB-3 expression is highest in the striatum and cortex at 16 weeks, dropping MSH3 levels by 50%.

    Davidson closed by emphasizing the need to ensure scalability for treatment beyond ultra-rare disorders. Latus hopes to file an Investigational New Drug application for its HD therapy, LTS-201, in the second half of 2026. At least two other biotech companies are also targeting MSH3 by other means.

     

     

    The post ASGCT 2026: Beverly Davidson Offers Vehicle and Route for Huntington’s Disease Gene Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Bayh-Dole Coalition report highlights eight American Innovators https://edusehat.com/en/bayh-dole-coalition-report-highlights-eight-american-innovators https://edusehat.com/en/bayh-dole-coalition-report-highlights-eight-american-innovators Fri, 15 May 2026 13:45:04 +0700 Edusehat Bayh-Dole, Coalition, report, highlights, eight, American, Innovators Recent medical breakthroughs are enabling better diagnostics for prostate cancer and Alzheimer’s and improving real-time imaging of tumors during surgery.

    The researchers behind these breakthroughs are among eight innovators highlighted by the Bayh-Dole Coalition in its fourth annual Faces of American Innovation report. Along with celebrating the innovators, the report calls attention to America’s system of intellectual property protections that enables inventors to commercialize their ideas.

    One essential piece of legislation is the 1980 Bayh-Dole Act, which says inventions created using research that received federal funds can be patented for private commercialization. Allowing private ownership of this IP enables the investment that brings new ideas to market, so people can benefit from these innovations.

    “Since its enactment, the impact of the Bayh-Dole system has been nothing short of extraordinary: $1 trillion contributed to the U.S. GDP, 6.5 million jobs supported, and more than 19,000 startups launched,” says the introduction of the Faces of American Innovation report. “More than four decades later, the system continues to deliver returns—and the stories in this report represent just a small fraction.”

    The Bayh-Dole Coalition, whose members include the Biotechnology Innovation Organization (BIO), was created to celebrate and protect the Bayh-Dole Act and promote policy that encourages American innovation.

    BIO’s membership in the coalition makes sense because biotech is one of the most research-heavy industries. Investors need to ensure the IP produced by biotech research is protected, so they can recoup their investments by commercializing innovations.

    BIO’s work toward protecting IP includes convening experts in its IP Counsels Committee, as well as more recent initiatives. BIO’s IP Task Force assembles experts from BIO staff and member companies who collaborate on IP strategy and specific policy activities. BIO’s Board has created the Economic Growth, Innovation, and Intellectual Property Committee, which sets BIO’s IP strategy and oversees the IP-related work performed by the IP Task Force and elsewhere at BIO.

    “We are working on Capitol Hill, in the courts, and in international fora to advocate and educate stakeholders about how IP supports a strong biotech ecosystem—and how best to protect it,” said Joe Franklin, BIO’s Chief Legal and Policy Officer.

    Innovators honored by the Bayh-Dole Coalition

    The Bayh-Dole Coalition will honor the innovators profiled in the report with the American Innovator Award at a June 3-4 event in Washington, DC. Rep. Deborah Ross (D-NC) is among the distinguished speakers scheduled for the event. This year’s Bayh-Dole awardees include:

    • Dr. Colleen Scott, chemist and associate professor at Mississippi State University, who discovered a new class of shortwave infrared (SWIR) imaging dyes with the potential to improve precise vizualization of tumors during cancer surgery.
    • Dr. Randall Bateman and Dr. David Holtzman, neurologists and professors at Washington University in St. Louis School of Medicine, who pioneered PrecivityAD® and PrecivityAD2™, the first blood-based diagnostic tests for Alzheimer’s disease, providing earlier, more efficient, and more accessible diagnosis.
    • Dr. Robert Dannals, professor of radiology at Johns Hopkins University School of Medicine, and Dr. Martin Pomper, professor and chair of radiology at the University of Texas Southwestern Medical Center, who developed Pylarify®, a Food and Drug Administration (FDA)-approved imaging agent for prostate cancer that improved detection.
    • Dr. Eric Fossum, vice provost of the Office of Entrepreneurship and Technology Transfer and senior professor at Dartmouth College, and Dr. Sabrina Kemeny, co-founder of tech startup TAP Systems, Inc., coinventors of “camera-on-a-chip” technology used in modern digital imaging and video across smartphones, medical devices, space exploration, and other application.
    • Dr. Carmel Majidi, engineering professor at Carnegie Mellon University, who created thermally conductive rubber called “Thubber,” a multifunctional soft, elastic, and conductive material that is transforming thermal management in robotics, electronics, and manufacturing systems.

    The post Bayh-Dole Coalition report highlights eight American Innovators appeared first on Bio.News.

    ]]>     Parkinson’s disease in women: Research gaps, treatment challenges, and new hope through GEM-PD https://edusehat.com/en/parkinsons-disease-in-women-research-gaps-treatment-challenges-and-new-hope-through-gem-pd https://edusehat.com/en/parkinsons-disease-in-women-research-gaps-treatment-challenges-and-new-hope-through-gem-pd Fri, 15 May 2026 13:45:03 +0700 Edusehat Parkinson’s, disease, women:, Research, gaps, treatment, challenges, and, new, hope, through, GEM-PD May is Women’s Health Month and in observation, Bio.News sat down with Ragasudha Botta, MBBS, PhD, MMSc, Senior Scientific Director, Critical Path for Parkinson’s (CPP) and Critical Path Institute (C-Path) to discuss how women’s lived experiences with Parkinson’s disease have too often been overlooked in clinical research and treatment development—and have even resulted in later diagnosis. From biological differences and disparities in care access to the promise of precision medicine initiatives like GEM-PD, we explore how researchers and advocates are working to ensure women with Parkinson’s are not just data points, but are active voices when it comes to drug development and patient care.

    What is Parkinson’s disease, and what does the current treatment landscape look like?

    Parkinson’s disease (PD) is a progressive neurodegenerative disorder that affects movement, but it also involves many non-motor symptoms that can shape a person’s daily life. Biologically, PD is characterized by loss of dopaminergic neurons and accumulation of misfolded alpha-synuclein pathology. Clinically, diagnosis still relies largely on motor features, particularly bradykinesia together with rigidity and/or resting tremor.

    The current treatment landscape has improved considerably, but it remains largely focused on symptom control rather than stopping the disease itself. Levodopa remains the most effective treatment for bradykinesia and other core motor symptoms, and dopaminergic therapies remain central to PD treatment. Other commonly used symptomatic treatments can improve symptoms and quality of life, but long-term treatment is often complicated by wearing-off, dyskinesias, or other adverse effects depending on the medication used. PD research is moving toward therapies that target disease biology, including alpha-synuclein aggregation, mitochondrial dysfunction, LRRK2 and GBA1-related pathways, gene therapy, and cell replacement strategies. 

    Despite this progress, no currently available treatment has been shown to definitively halt PD progression or prevent neurodegeneration. So, the current landscape is both encouraging and incomplete. We have increasingly effective ways to manage symptoms, but the field still urgently needs therapies that change the long-term course of the disease.

    How do biological differences and other gender-related factors significantly shape the lived experience of women with Parkinson’s disease?

    Sex and gender influence how women experience PD, but these differences are often overlooked. From a biological standpoint, women may have a different PD profile than men. Epidemiologically, men are diagnosed more often, but this does not mean Parkinson’s is less important in women. Women may face delays in diagnosis, reduced access to movement disorder specialists, and less informal caregiving support. In women, symptoms may present differently, with a greater burden of non-motor symptoms.

    Women may also respond differently to dopaminergic therapies. Some studies suggest women may experience more levodopa-induced dyskinesia, while access to device-aided therapies such as deep brain stimulation may be lower for women. This means that the same disease can translate into a different therapeutic journey for women, with different side effects, and care needs. 

    Biological sex differences strongly influence the lived experience of women with Parkinson’s. Many women are also caregivers themselves to spouses, children, grandchildren, or aging parents and their own symptoms may be minimized or accommodated quietly until the disease has already affected independence, employment, relationships, and emotional well-being.

    The key point is that women with Parkinson’s are not simply a “smaller group with PD.” Their biology, symptoms, treatment responses, social roles, caregiving realities, and access to care intersect. If research and clinical care do not capture these differences, we risk designing trials, endpoints, digital measures, and treatment pathways that do not fully reflect women’s real lives.

    So, improving outcomes for women with Parkinson’s requires a sex- and gender-informed approach like enrolling enough women in studies, analyzing outcomes by sex, capturing hormonal and reproductive history where relevant, prioritizing patient-reported outcomes, and listening carefully to women’s voices. Precision medicine in PD will remain incomplete unless it includes the lived experience of women as central evidence, not as an afterthought

    As a young investigator and clinician researcher, what attracted you to join C-Path and contribute as Senior Scientific Director of the CPP global consortium?

    What attracted me to C-Path and the CPP global consortium was the opportunity to connect my clinical background, research training, and personal motivation with work that can translate into accelerating PD drug development. My journey into medicine and neurology is deeply personal. I lost my mother to meningitis when I was in middle school, after symptoms such as neck stiffness, headache, and vomiting were initially dismissed as vitamin deficiency. That experience shaped my decision to become a doctor and drew me toward neurology.

    Over time, I developed an interest in neurodegenerative disorders. As a clinician, I often heard PD patients ask, “How long do I need to take these medicines?” and “Is there a cure?” Those questions stayed with me and pushed me toward Parkinson’s research, clinical trials, and drug development, ultimately leading me to pursue a Master of Medical Sciences at Harvard Medical School focused on clinical trials and drug development.

    During my PhD, my clinical research work at Centre for Brain Research, and later my work at Massachusetts General Hospital, I came to appreciate the power of high-quality data. My own research work benefited from CPP’s Integrated Parkinson’s database, including work now published in npj PD. That experience made CPP’s mission very real to me, as better data can help us understand which patients are at risk, design better trials, and move closer to therapies that are targeted to the right patients. This work also introduced me to Dr. Stephenson, Vice President at CPP, who has since been an important mentor and source of motivation in my journey.

    I joined C-Path and CPP because I wanted to contribute to that translational bridge, turning rigorous science, patient-level data, and global collaboration into evidence that can help answer the questions patients are still waiting on, i.e. “Can we do better, can we move faster, will there ever be a cure and can we bring real hope to people living with PD?”

    What is C-Path doing to accelerate drug development for Parkinson’s disease, and why was the GEM-PD initiative launched? What is GEM-PD?

    C-Path is helping accelerate Parkinson’s drug development by creating a neutral, precompetitive space where industry, academia, patient organizations, data scientists, and regulators can work together on the shared barriers that slow clinical trials. Through the CPP consortium, C-Path focuses on building drug development tools that can make Parkinson’s trials more efficient and informative. That includes integrated patient-level databases, disease progression models, clinical trial simulation tools, biomarkers, digital health technologies, and more meaningful clinical outcome measures. The goal is to help drug developers design better trials, identify the right patients, choose better endpoints, and generate evidence that is useful for regulatory decision-making.

    A major part of this work is the CPP Integrated Parkinson’s Database, which brings together anonymized patient-level data from observational studies and clinical trials. This includes data from 27 studies, more than 15,000 participants. This kind of data infrastructure is important because Parkinson’s is heterogeneous, and no single study can answer all the questions needed to improve trial design.

    The regulatory milestones of CPP include FDA and EMA support letters for dopamine transporter imaging as an enrichment biomarker, and an FDA Letter of Support related to α-synuclein seed amplification assay as an enrichment biomarker for trials in synuclein-related disorders. C-Path is not only generating science but is helping translate science into tools that regulators and drug developers can use.

    GEM-PD stands for Global Evidence in Medicine for Parkinson’s Disease. It aims to accelerate more personalized treatments by using diverse data, artificial intelligence, and digital health technologies, with the goal of improving detection, disease management, and therapies for women affected by Parkinson’s. GEM-PD launched in March 2025 and connects it with the broader momentum around sex-informed clinical evaluation and sex- and gender-informed Parkinson’s care. 

    GEM-PD is not simply a “women’s Parkinson’s project.” It is a precision medicine initiative. It asks: what are we missing when women are not fully represented in the data, in trial design, in symptom measurement, and in regulatory conversations? By answering those questions, GEM-PD can help ensure that future Parkinson’s therapies are developed for the real diversity of people living with the disease, not just for an average patient who may not represent everyone.

    Why is giving women with Parkinson’s disease representation and a voice so crucial?

    Giving women with Parkinson’s disease representation and a voice is crucial because many of their most burdensome symptoms are still not routinely brought into the clinical conversation. Many non-motor symptoms remain under-discussed in routine care. Because some of these symptoms feel private or embarrassing, patients may not raise them unless the clinician opens the door. So, the symptom remains invisible; not because it is unimportant, but because no one asked. This is a global issue. Women’s experiences of PD may vary across countries, cultures, and healthcare systems, in relation to access to care, stigma, and willingness to discuss symptoms. Because our database also includes patients outside the U.S., GEM-PD can help ensure that we are not defining women’s needs from a single healthcare context but are listening to a broader and more diverse global PD community.

    This is why “voice” matters as much as representation. Representation ensures that women are present in the data. Voice ensures that their actual experiences shape the questions we ask. It reminds us that Parkinson’s care should not depend only on what clinicians observe, but also on what patients may be quietly living with.

    Ultimately, giving women with Parkinson’s a voice is crucial because it changes what becomes visible. And once these experiences become visible, they can be measured, treated, and prioritized. Without listening to women directly, we risk defining Parkinson’s disease too narrowly and missing the symptoms that most affect their daily lives.

    What is the future for patients with Parkinson’s disease, and what are your hopes specifically for women affected by the disease?

    Our hope at C-Path, through GEM-PD, is to help advance a future in which women with Parkinson’s Disease are more equally represented in clinical trials, and where the scope of drug development includes new therapies that focus on women’s needs. Our hope is that the future of Parkinson’s disease will move toward care that is not only more biologically precise, but also more attentive to the realities patients live with every day. With FDA’s guidance increasingly emphasizing the importance of sex differences, this is not just a dream, it is becoming a necessary direction for more inclusive Parkinson’s research, care, and drug development.

    Recent reviews continue to emphasize that women may have distinct clinical profiles, including greater vulnerability to disabling motor complications and non-motor fluctuations, while also facing disparities in care. But the future should not stop at identifying biological differences. We also need to change what we routinely capture in clinical care and research. For women, our hope is that these experiences become part of the standard Parkinson’s conversation. When these symptoms and experiences are recorded consistently, they become usable data. That data can improve clinical care, refine patient-reported outcomes, guide trial design, and help develop therapies that address what patients need, not only what is easiest to measure.

    So, our hope is that women with Parkinson’s will no longer have to fit into a model of care built around an average patient who may not represent them. Instead, their biology, symptoms, priorities, and lived experience should help shape the next generation of precision medicine in PD.

    The post Parkinson’s disease in women: Research gaps, treatment challenges, and new hope through GEM-PD appeared first on Bio.News.

    ]]>     Implantable Living Materials Contain Infection-Sensing Bacteria That Release Therapeutics https://edusehat.com/en/implantable-living-materials-contain-infection-sensing-bacteria-that-release-therapeutics https://edusehat.com/en/implantable-living-materials-contain-infection-sensing-bacteria-that-release-therapeutics Fri, 15 May 2026 06:15:08 +0700 Edusehat Implantable, Living, Materials, Contain, Infection-Sensing, Bacteria, That, Release, Therapeutics Overcoming a major hurdle in the use of microbes as medicine, researchers at Harvard’s Wyss Institute and John A. Paulson School of Engineering and Applied Sciences (SEAS) have developed an “implantable living materials” (ILMs) platform comprising encapsulated infection-sensing bacteria that can release therapeutic molecules on demand but are kept physically separated from the surrounding tissue.

    Wyss Founding Core Faculty member David Mooney, PhD, and colleagues encapsulated a genetically engineered, therapeutic strain of E. coli bacteria within a biomaterial made from a hydrogel that was specifically designed to regulate bacterial growth and resist mechanical stresses, such as those present at physically active sites in the body, demonstrating that the bacteria could be confined for over six months.

    To evaluate the material’s clinical potential, the researchers transformed the ILM into an active therapeutic system by engineering the bacteria to detect chemical signals from Pseudomonas aeruginosa, a common cause of implant-related infections. In response to the pathogen, the engineered bacteria autonomously self-destructed to release an antibacterial protein that killed the P. aeruginosa. In a mouse model of joint infection, the system successfully reduced bacterial burden, demonstrating the potential of durable, programmable ILM-based therapeutics for long-term disease treatment. The researchers suggest that their development represents a shift from passive drug depots to autonomous, responsive—and living—therapeutic systems.

    “With this new strategy combining both an engineered material with designed mechanical features and genetically engineered microbes that produce therapeutic payloads on demand, we provide a generalizable framework for deploying future microbial medicines,” said Mooney. “The precision, safety, and therapeutic durability afforded by this ILM strategy could be a potential solution for treating a wider range of diseases and infections, enabling therapeutic efficacies that might surpass those of other drug delivery strategies.”

    Mooney, the Robert P. Pinkas Professor of Bioengineering at SEAS, is co-senior and corresponding author of the team’s published paper in Science, titled “Implantable living materials autonomously deliver therapeutics using contained engineered bacteria,” in which the authors concluded that their collective results “… establish ILMs as a foundation for deploying microbial medicines in vivo as autonomous therapeutic depots across diverse disease settings.”

    Patient recovery from many debilitating conditions and diseases could be sped up significantly and be more effective if drugs and therapeutic molecules were delivered right to where they are needed in the body, over the entire regenerative process, and in doses finely tuned to therapeutic needs. An intriguing way to achieve this is the use of implantable, synthetically engineered, living cells that can sense injury or disease-associated conditions in their environment and flexibly respond by producing the right amount of a therapeutic molecule.

    “Synthetically engineered cells are emerging as living therapeutic modalities, capable of sensing physiological conditions and producing bioactive payloads in vivo,” the authors wrote. Unlike conventional drugs, these “living therapeutics” can sustain themselves in vivo and survive in many biological environments, including tumors, inflamed tissues, infected tissues, and even within human cells.

    Bacteria are particularly attractive because they can be genetically programmed to release therapeutic molecules in response to specific biological signals. Bacteria can thrive in harsh physiological environments within the body, such as within infected or inflamed tissues, tissues undergoing mechanical movements, and tumors.

    Some such microbial therapies have even advanced into clinical trials to treat certain cancers, metabolic disorders, and the progression of kidney stones. However, thus far, such trials have failed, and microbes are feared to also pose significant safety risks because they cannot be contained at specific sites in the body. “… controlling microbial off-­target effects remains a key safety consideration because dissemination and associated toxicity have been reported across multiple clinical contexts,” the authors continued.

    Previous implantable biomaterial systems, such as hydrogels and capsule-like enclosures, have shown some success in confining microbes, but only for short periods—typically no more than two weeks. “Implantable hydrogels offer a physical strategy to confine therapeutic cells at target sites,” the investigators commented. “Such living materials hold promise as localized drug depots with the capacity to dynamically respond to diseased environments … In this work, we present an implantable material that encapsulates and confines bacteria, wherein synthetically engineered microbes produce therapeutic payloads from within.”

    First author Tesuhiro Harimoto, PhD, who spearheaded the project as a postdoctoral fellow in Mooney’s group, explained further, “In the beginning, we asked the seemingly simple question, what if we could design a material that safely encapsulates drug-delivering bacteria inside and allows therapeutic drugs to pass through to where they are needed.” Although scientists have extensively studied how physical parameters of synthetic materials change with tweaks made to their composition and chemical connections, “this was a big ask since the encapsulating material had to reconcile two often contradictory features: it needed to be sufficiently ‘stiff’ so that bacteria pushing against it from the inside can’t break it apart, and sufficiently ‘tough’ to provide a enclosure that protects against external physical stresses in mechanically active tissues.”

    Graphical abstract: "Implantable living materials autonomously deliver therapeutics using contained engineered bacteria" [Tetsuhiro Harimoto]
    Graphical abstract: Implantable living materials autonomously deliver therapeutics using contained engineered bacteria. [Tetsuhiro Harimoto]
    An expanding bacterial colony can exert pressures that are multiple orders of magnitude higher than those produced by mammalian cells. Also, the type of stresses produced by the body’s various mechanical forces, such as, for example, generated by tension in muscles or compression on joints, can fatigue a material over time and disrupt it from the outside. However, introducing too much stiffness can often make a material too brittle, which means that cracks can quickly propagate through it; and a high toughness, which, in principle, allows a material to resist fracturing, often makes it soft. “We hypothesized that fulfilling two key criteria for a material enables robust and durable containment of therapeutic bacteria: (i) resistance to the internal forces generated by proliferating bacteria and (ii) mechanical toughness sufficient to withstand deformation from surrounding tissues,” the team wrote.

    To realize ILMs, the team started with polyvinyl alcohol (PVA), which is already used clinically, and processed it to form nanoscale interactive crystalline domains.  The resulting scaffolds are simultaneously highly stiff and tough. “Finding out how to fabricate optimal hydrogels from PVA that are crosslinked through dense crystalline domains, and how to do this in a way that keeps the enclosed bacteria alive and active, was a big part of our study,” said Harimoto. The researchers included the bacteria in their fabrication process within tiny droplets of gelatin that protected them against desiccation and selective chemical manipulations.

    This strategy allowed them to fabricate an ideally stiff and tough material scaffold around the bacteria, using a combination of tolerable freeze-thaw cycles, salt conditions, and chemical treatment times. Late in the process, via a slight shift in temperature, the gelatin microgel could be dissolved to create internal voids for the bacteria to thrive in. Due to the tiny pore sizes within the PVA material, the bacteria remain constrained while the soluble molecules they produce can travel to other sites in the body.

    The resulting ILM safely contained the bacteria over extended time intervals of up to six months and was resistant to repeated mechanical stresses. “We developed a hydrogel scaffold with dual mechanical features: high stiffness to regulate bacterial proliferation and high toughness to resist material fracture under physiological stress,” the investigators stated. “This design achieved complete bacterial containment for six months and withstood multiple forms of mechanical loading that otherwise caused catastrophic material failure.”

    To provide proof-of-concept for ILMs, the team focused on the infection of implanted periprosthetic devices designed to treat fractures or bone loss around existing artificial joint replacements by pathogenic P. aeruginosa strains. Many treatments with periprosthetic devices fail due to infection, which goes along with inflammation and the spread of antibiotic resistance. “We evaluated the use of ILMs for periprosthetic joint infection in vivo,” they wrote. This model was designed to capture early postimplantation infection during which most infections arise in clinical settings.”

    To effectively treat this and other types of infection, the therapy-delivering bacteria within the ILM needed to be genetically engineered to function as a drug depot with autonomous “sense-and-respond” capabilities. To achieve this, the team installed a synthetic gene circuit in the E. coli strain that enabled the bacteria to sense a small diffusible metabolite produced by P. aeruginosa, known as N-acyl homoserine lactone (AHL), and, in response, activate a self-destruction gene to trigger cell lysis. The self-destruction process, triggered in a fraction of ILM bacteria, resulted in release from the ILM of a synthetic P. aeruginosa-killing protein called chimeric pyocin (ChPy) that the bacteria produce continuously. ChPy is toxic to P. aeruginosa, erasing the pathogen in the local ILM environment.

    “When we tethered a therapeutic ILM to a stainless steel periprosthetic device that was infected with a pathogenic P. aeruginosa strain isolated from a patient’s wound and implanted next to the femur bone of mice, it significantly reduced the pathogen burden while safely containing its engineered bacteria over a three-day treatment course,” said Harimoto. “In contrast, in mice that we treated with a non-therapeutic control ILM that did not produce ChPy, the numbers of P. aeruginosa bacteria continued to rise over the same time interval. This demonstrated the ability of therapeutic ILMs to autonomously sense and treat periprosthetic infection in vivo.”

    The researchers think that specifically engineered ILMs as a novel class of therapeutics with excellent safety features and locally targeted drug release capabilities have broad potential, ranging from tissue regeneration to immune modulation in a variety of disease settings. A patent application describing the use of ILMs for drug delivery has been filed.

    In their paper, the authors wrote in summary, “ILMs are distinct from other therapeutic modalities, such as drug-loaded depots and vaccines. By directly sensing pathogen-­derived signals and locally releasing antimicrobial payloads, ILMs enable rapid, antigen-independent intervention at the implant site. This localized, autonomous mode of action is well-suited for periprosthetic joint infection, where early intervention is critical.” Their collective results, the team suggests, “…establish ILMs as a foundation for deploying microbial medicines in vivo as autonomous therapeutic depots across diverse disease settings.”

    In a related perspective, Kaige Chen, PhD, and Quanyin Hu, PhD, at the School of Pharmacy, University of Wisconsin–Madison, acknowledge that further work will be needed to determine whether contained living therapeutics can function in vivo over long periods. Nevertheless, they said, “The study of Harimoto et al. addresses a central obstacle to deploying living therapeutics—keeping bacteria physically separated from the surrounding tissue. Chen and Hu further note that the in vivo findings in the artificial joint mouse model  “… could advance living therapeutics from short-lived proof-of-concept systems to durable, programmable medicines.”

    The post Implantable Living Materials Contain Infection-Sensing Bacteria That Release Therapeutics appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Molecular Anchors Help Tumor Therapies Stay Longer on Cancer Cells https://edusehat.com/en/molecular-anchors-help-tumor-therapies-stay-longer-on-cancer-cells https://edusehat.com/en/molecular-anchors-help-tumor-therapies-stay-longer-on-cancer-cells Fri, 15 May 2026 02:40:08 +0700 Edusehat Molecular, Anchors, Help, Tumor, Therapies, Stay, Longer, Cancer, Cells For cancer therapies to work, they need to stay in proximity to the target diseased tissues for long enough. To help with that challenge, a group of scientists, led by a team at University of California, San Francisco (UCSF), have developed a drug carrier that physically anchors itself to cancer cell membrane, which helps to improve drug retention and effectiveness. Full details are published in a new ACS Central Science paper titled “A Prodrug Strategy to Conditionally Trap Therapeutic Payloads for Improved Tumor Retention.”

    “Retaining drugs within tumors is an often-overlooked dimension of drug development that nevertheless greatly impacts the therapeutic window and outcomes,” said Michael Evans, PhD, a professor in the department of radiology and biomedical imaging at UCSF and a corresponding author on the study. In fact, approaches that deliver cancer therapeutics to tumors but lack dedicated mechanisms to ensure tumor retention often lose efficacy within a few days of drug administration. 

    Previously, Evans and others designed drug delivery systems called restricted interaction peptides or RIPs that can deliver diverse therapeutic cargos including cytotoxins and radioisotopes. They work by changing shape when processed by disease-associated enzymes. These allow them to embed in cell membranes, tethering their drug payloads in place, promoting cellular uptake and improving effectiveness. Building on that work, the scientists engineered RIPs to interact with fibroblast activation protein, a serine protease that is prevalent in solid tumors and fibrosis. 

    Imaging studies of cancer cell cultures showed that a fluorescently tagged RIP was rapidly taken up by the cells. Then when the scientists attached an anticancer drug, monomethyl auristatin E or MMAE, to the RIP, they found that the drug-peptide combination was as effective in killing cancer cells as the drug alone. Furthermore, when the drug-peptide combination was injected into mice with human cancers, it selectively targeted tumor tissue and was more effective at shrinking tumors than the unmodified drug with fewer side effects. The scientists observed similar results when they attached RIPs to radioactive copper isotopes which are commonly used in nuclear imaging and radiotherapy. 

    The scientists expect to initiate Phase I clinical imaging studies of the RIP-radioactive copper isotope pairing in human cancer patients later in 2026 in collaboration with a company that is developing RIPs into therapeutics. 

     

    The post Molecular Anchors Help Tumor Therapies Stay Longer on Cancer Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Visualizing Receptor Transport Within Neurons via Transcytosis https://edusehat.com/en/visualizing-receptor-transport-within-neurons-via-transcytosis https://edusehat.com/en/visualizing-receptor-transport-within-neurons-via-transcytosis Thu, 14 May 2026 08:45:09 +0700 Edusehat Visualizing, Receptor, Transport, Within, Neurons, via, Transcytosis As spindly, elongated cells, neurons must be able to transport proteins and receptors between distant sites in their cell bodies and axons to function properly. A new imaging study by researchers at Johns Hopkins University has now visualized the ebb and flow of the nerve growth factor receptor TrkA within neurons, via an unusual process known as transcytosis. Their study also explains how this phenomenon supports neuronal function and connectivity in mice.

    Senior and corresponding author Rejji Kuruvilla, PhD, at Johns Hopkins University Department of Biology, and colleagues reported on their findings in Science Signaling, in a paper titled “Transcytosis-mediated anterograde transport of the receptor TrkA mediates the formation of presynaptic sites in sympathetic neurons.” In their paper, the authors concluded, “These findings provide mechanistic insight into an atypical mode of receptor trafficking and demonstrate its physiological relevance in sympathetic neuron connectivity in mice … Our study suggests that transcytosis might be a more general mechanism than now appreciated for the targeted transport of trophic and guidance receptors, adhesion and synaptic proteins, as well as ion channels.”

    The axons of neurons are extremely long compared to their main cell bodies, with axon terminals sometimes residing a long distance from the cell nucleus. “Axon terminals can be meters away from cell bodies where many axonal membrane proteins with critical functions in regulating axon guidance and growth, neuronal survival, presynaptic organization, and synaptic transmission are made,” the authors wrote.

    Neurons need to be able to transport these proteins efficiently across these relatively vast distances. They do this by either directly sending the protein through a secretory pathway or via an indirect mechanism called transcytosis. The latter occurs when the central cell body takes in newly synthesized proteins or surface receptors, after which they move to axons through the cell cytoplasm. “Transcytosis is an atypical endocytosis-based mechanism, where newly synthesized proteins are first inserted on cell body surfaces, internalized, and anterogradely transported to axons,” the team continued.

    Transcytosis is still relatively obscure and enigmatic compared with the direct secretion method, and questions remain about how exactly it sustains the function and connectivity of neurons. “In contrast to the considerable progress made in understanding the direct secretory pathway, there is limited knowledge about transcytosis, specifically the underlying transport kinetics and organelles involved, whether it occurs in vivo, and its contributions to neuronal connectivity and function,” the investigators noted.

    Seeking answers, first author Kuruvilla, together with first author Guillermo Moya-Alvarado, PhD, and colleagues, used live cell imaging and electron microscopy to peer at the movement of receptors across compartments within mouse neurons.

    They visualized the trafficking dynamics and transcytosis of a receptor named TrkA. “The family of tropomyosin-related kinase (Trk) receptors provides a prominent example of membrane proteins that undergo long-distance axonal trafficking to control neuronal survival, axon growth, and synaptic transmission,” the scientists explained.

    Through their study, the authors noted various shifts in speed and direction as vesicles carried TrkA from the soma to axons. Using labeled TrkA proteins, the scientists also confirmed that transcytosis occurred within nerve terminals of living mice. “Live imaging and electron microscopy of compartmentalized cultures revealed that soma surface–derived TrkA proteins underwent dynamic transport within axons, with changes in speed, direction, and the vesicular organelles that carried them as they moved from proximal to distal axon compartments,” they stated. “In mice, soma surface–labeled TrkA proteins were observed in sympathetic nerve terminals, demonstrating that transcytosis occurs in vivo.”

    Assessing TrkA receptors transcytosis from cell bodies to nerve terminals in vivo. Superior cervical ganglion (SCG) in Ntrk1Flag mice, at postnatal day 2 to day 3 were injected in one of each paired ganglia per animal with the contralateral ganglion and target tissues (noninjected side) serving as internal controls to assess any systemic leakage of injected label. Representative image of the injected side. Flag (green) and sympathetic neurons (Tuj1, red) immunofluorescence in the superior cervical ganglia. DAPI is shown in blue. Scale bars, 50 μm. [All images and movies were generated by Guillermo Moya Alvarado]
    Assessing TrkA receptors transcytosis from cell bodies to nerve terminals in vivo. Superior cervical ganglion (SCG) in Ntrk1Flag mice, at postnatal day 2 to day 3, were injected in one of each paired ganglia per animal with the contralateral ganglion and target tissues (noninjected side) serving as internal controls to assess any systemic leakage of injected label. Representative image of the injected side. Flag (green) and sympathetic neurons (Tuj1, red) immunofluorescence in the superior cervical ganglia. DAPI is shown in blue. Scale bars, 50 μm. [All images and movies were generated by Guillermo Moya Alvarado]
    They also found that disrupting its transcytosis by introducing a point mutation into TrkA reduced the number and size of presynaptic sites and decreased synaptic transmission in culture and in rodents in vivo, confirming the importance of the process for neuronal physiology.  “These findings provide mechanistic insight into an atypical mode of receptor trafficking and demonstrate its physiological relevance in sympathetic neuron connectivity in mice,” the team concluded “Uncovering mechanisms of axon delivery has implications that extend beyond the healthy nervous system to understanding cell biological pathways that contribute to nerve repair after injury or neurodegeneration, because the correct complement of membrane proteins must be accurately targeted to regenerating axons to ensure functional recovery.”

    The post Visualizing Receptor Transport Within Neurons via Transcytosis appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Lupus Awareness Month meets Women’s Health Month: Advancing care, research, and advocacy https://edusehat.com/en/lupus-awareness-month-meets-womens-health-month-advancing-care-research-and-advocacy https://edusehat.com/en/lupus-awareness-month-meets-womens-health-month-advancing-care-research-and-advocacy Thu, 14 May 2026 05:15:04 +0700 Edusehat Lupus, Awareness, Month, meets, Women’s, Health, Month:, Advancing, care, research, and, advocacy May is both Lupus Awareness Month and Women’s Health Month. And the timing is apropos as lupus affects female patients at a far higher rate than it affects men, though researchers are still not sure why. 

    In observation, Bio.News sat down with the Lupus Foundation of America (LFA), which has led the fight to improve the lives of people affected by lupus through groundbreaking research, patient advocacy, education, and support for nearly 50 years. We discuss some of the persistent challenges facing lupus patients, the latest advances in research and treatment, and why greater awareness remains critical to improving outcomes for the estimated 1.5 million Americans living with lupus.

    1. What is the Lupus Foundation of America, and why was it founded?

    The Lupus Foundation of America (LFA) was founded in 1977 when leaders from more than 20 independent local and statewide lupus organizations recognized the need to raise awareness of lupus and place the disease on the nation’s healthcare agenda. For nearly five decades, LFA has grown into the only national organization dedicated exclusively to solving the complex challenges of lupus. 

    LFA’s mission is to improve the quality of life for all people affected by lupus through research, education, support, and advocacy. The organization is focused on reducing the time to diagnosis, expanding access to safe and effective treatments, and increasing the availability of care and support services for people living with lupus. In 2016, the Foundation co-founded the World Lupus Federation, uniting more than 200 lupus organizations worldwide around a shared vision of a life free from lupus. 

    Today, LFA continues to be the premier lupus patient advocacy organization both nationally and globally, leading efforts to increase public and private investment in lupus research, advance safer and more targeted therapies, and improve awareness and understanding of lupus among patients, healthcare professionals, and the public. Working with tens of thousands of lupus advocates across the nation, we’ve generated more than $795 million in federal research funding for lupus in the last five years alone. Our research portfolio spans basic, translational and clinical research, and has contributed to nearly every significant lupus research advancement. Every day, we provide caring support and answers through hundreds of trustworthy resources and programs that cover every aspect of lupus.

    2. May is Lupus Awareness Month. How is LFA raising awareness, and what are some common misconceptions about the barriers patients with Lupus face?

    As the national convener of Lupus Awareness Month, LFA brings communities together each May to raise public understanding of lupus. The nationwide and global effort raises awareness of the signs and symptoms of lupus, amplifies the real-life stories of the day-to-day impact of living with lupus, and raises funds to support lupus research, education programs and support services. 

    This year’s Make Lupus Visible campaign is reaching hundreds of thousands of people, and also features the newly updated KNOW Lupus Quiz. This interactive tool to educate the public engages thousands every week, helping them learn more about lupus in a fun and memorable way. 

    3. Lupus disproportionately impacts women. What do we know about the underlying biology and what is driving these gender differences?

    Lupus is a complex and debilitating chronic autoimmune disease where the immune system is unable to tell the difference between healthy tissue and foreign invaders. This can result in inflammation, debilitating pain, and damage to any organ system in the body. The symptoms of lupus often disrupt daily life and may lead to serious—and sometimes life-threatening—complications, including lupus nephritis (lupus-related kidney disease), which affects up to 60% of people with lupus and can result in kidney failure.

    An estimated 1.5 million Americans are living with lupus. While lupus can affect anyone, nine out of 10 people living with lupus are women, most often diagnosed during their childbearing years. The disease also disproportionately affects women from certain racial and ethnic backgrounds, including Black/African American, Hispanic/Latino, Asian American, and Pacific Islander communities, who are 2–3 times more likely to develop the disease than Caucasian women. 

    Researchers are still seeking answers to this question and aren’t quite sure why lupus is more common in women. However, they think that differences in our genes and hormones—chemical messengers that deliver messages from the brain to the body—may play a role. Some studies show that estrogen contributes to the development of lupus and makes it more severe. 

    4. LFA is playing a key role in accelerating scientific breakthroughs. What are some of your key areas of focus and what is the next frontier in Lupus treatments?

    Since its inception, LFA has funded hundreds of researchers at medical institutions across the United States and around the world. LFA supports innovative, high-impact research aimed at accelerating progress and improving quality of life for people affected by lupus. By challenging outdated approaches and investing in promising science, the organization works to uncover the causes of lupus, better understand disease progression, and advance the search for safer, more effective treatments and ultimately a cure. 

    Recognizing that many existing therapies still fail to meet patient needs, LFA created RAY (Research Accelerated by You)®, a patient registry designed to place the experiences of people with lupus and their caregivers at the center of research to help deepen the understanding of this complex disease and accelerate the development of treatments for people living with lupus. With thousands of participants, RAY has become the world’s largest lupus patient data repository. Participants contribute through surveys, focus groups, and advisory panels that help shape drug development and patient-centered therapies. The platform also promotes diverse representation in research, reflecting the broad range of experiences across race, ethnicity, disease type, and disease severity. In addition, RAY has supported education and awareness efforts for nearly 20 clinical trials focused on CAR-T therapy, systemic lupus erythematosus (SLE), lupus nephritis, discoid lupus erythematosus, and related conditions. 

    Improving pregnancy outcomes for women with lupus has also been a major research priority. With donor support, LFA funded the IMPACT Study (IMprove Pregnancy in APS with Certolizumab Therapy), which evaluated whether adding certolizumab to standard treatment could reduce pregnancy risks for women with lupus and/or antiphospholipid syndrome (APS), a disorder associated with abnormal blood clotting. Recently, the study completed its Phase II research and produced encouraging results: 93% of participants with healthy pregnancies delivered healthy babies, compared with a 38% survival rate in participants’ prior pregnancies. These findings offer renewed hope for women with lupus and their families. In February 2025, the MiSLE Phase II clinical trial, which studies mesenchymal stromal cells as a potential lupus treatment, reached a major milestone by completing enrollment. Co-funded by LFA and NIH, this study is advancing a promising new approach to lupus treatment. 

    LFA also continues to prioritize strengthening the future lupus research workforce, through our Gina M. Finzi Memorial Student Summer Fellowship and the Gary S. Gilkeson Career Development Award, which we present each year. 

    Today, LFA’s investments in research are helping reshape the standard of care for lupus. Recently, LFA funded 12 new studies focused on understanding symptoms such as fatigue and cognitive impairment, advancing treatments for immune and kidney complications, and improving self-management and medication adherence. LFA also continues to lead efforts in pediatric lupus research and in studies focused on predicting and preventing the disease. 

    Advocacy for lupus research remains a central focus of LFA’s work. Each year, LFA plays the leading role advocating for federal funding for lupus research and for implementation of federal lupus research and awareness programs. Together with advocates and supporters nationwide, LFA helps secure millions in federal funding for lupus research and education programs while advancing policies aimed at expanding access to care and reducing treatment costs for people living with lupus. LFA collaborates with federal agencies and coalitions to advance lupus priorities at the National Institutes of Health, Centers for Disease Control and Prevention, Department of Defense, Office of Minority Health, Centers for Medicare & Medicaid Services, and the Food and Drug Administration. 

    Due to the efforts of the LFA and our decades of work, public awareness of lupus has grown significantly over the years, helping foster greater understanding of the disease and its impact. Through national campaigns, global partnerships, and advocacy initiatives, the Lupus Foundation of America continues to drive awareness because earlier diagnosis, compassionate care, and better treatment outcomes depend on greater public understanding.

    To learn more about Lupus and the work that the Lupus Foundation of America is doing, visit HERE.

    The post Lupus Awareness Month meets Women’s Health Month: Advancing care, research, and advocacy appeared first on Bio.News.

    ]]>     Technique Yields Uniform, High-Quality EVs at Scale https://edusehat.com/en/technique-yields-uniform-high-quality-evs-at-scale-10631 https://edusehat.com/en/technique-yields-uniform-high-quality-evs-at-scale-10631 Thu, 14 May 2026 05:10:21 +0700 Edusehat Technique, Yields, Uniform, High-Quality, EVs, Scale Mesenchymal stem cell-derived extracellular vesicles (MSC-EVs) play an outsized role in intracellular communications, influencing such functions as inflammation and tissue repair. With the possible applications of these small, membrane-bound particles growing, an efficient, cost-effective production method has been on drug manufacturers’ wish lists for some time.

    A novel, streamlined chromatographic production and isolation method developed by scientists at Satorius BIA Separations in Slovenia may fulfill that wish, yielding uniform, high-quality EVs at scale. The method concentrates MSC-EVs directly from conditioned media. It also removes 97% of protein impurities and 95% of double-stranded DNA-related impurities, increasing their potential as therapeutics or drug delivery vessels.

    Microcarrier + suspension

    The method relies upon preferential exclusion chromatography, Katja Vrabec, head of product application area (EVs) at Sartorius, notes in a recent paper. In it, Vrabec and colleagues explain the method “uses monolithic hydroxyl columns to purify and concentrate the MSC-EVs,” and biochromatography analytics to track EV-specific surface antigens.

    First, the team expanded the MSCs in growth media, and then produced the EVs in a lean media formulation to limit production of protein and particle contaminants. That part is standard.

    Here’s what’s different: The scientists used a microcarrier-based system rather than flask-based 2D cultivation to scale the MSC cultures and increase the ratio of EVs to contaminants in conditioned media. They also used a suspension culture to enhance cell growth surface-to-volume ratios, and thereby increase EV yield. Then, they used a monolithic hydroxyl column to capture and purify the EVs directly from harvest.

    Increasing cell density and the cell-to-impurity ratio lowers buffer consumption downstream and lays the groundwork for biomanufacturers to transition to a scalable bioreactor system.

    Because the main impurities in EV harvests don’t interact with the chromatographic column in high-salt-binding conditions, the team recommends choosing a low-salt buffer for elution to reduce the need for buffer exchange before the polishing step. The optimal binding condition, they report, is “sodium citrate of 0.75M at pH 7.0.”

    This research highlights the need to consider upstream and downstream processing as a cohesive system, to design a simple, scalable, holistic process, and to apply reliable analytics. This all is particularly challenging, the team admits, given “the heterogeneous nature of EVs and the presence of similarly-sized components in biological samples.”

    The post Technique Yields Uniform, High-Quality EVs at Scale appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Yeast We Can: Cutting Costs by Optimizing Cell-Free Expression Systems https://edusehat.com/en/yeast-we-can-cutting-costs-by-optimizing-cell-free-expression-systems https://edusehat.com/en/yeast-we-can-cutting-costs-by-optimizing-cell-free-expression-systems Thu, 14 May 2026 05:10:16 +0700 Edusehat Yeast, Can:, Cutting, Costs, Optimizing, Cell-Free, Expression, Systems Choosing the right additives could help “cell-free” expression systems finally fulfill their potential and provide biopharma with a low-cost way of making protein drugs, according to a recent research report.

    The new study looked at how cell-free systems, in which biochemical reactions occur independently of cells, could be fine-tuned to provide drug makers with alternatives for large-scale protein production.

    And the potential of the approach is significant, says Karen Polizzi, PhD, a professor from the department of chemical engineering at Imperial College London, who adds, “Cell-free protein synthesis (CFPS) is a flexible manufacturing technology. It can be used for on-demand synthesis in low-resource environments or to make difficult-to-express products, especially medicines that are toxic to the cell. Cell-free reactions scale well across microliter to liter scale without needing adjustments.”

    The Imperial team’s research focused on expression systems based on the yeast species Pichia pastoris, which, as Polizzi explains, “has machinery capable of post-translational modifications of proteins that can be necessary for function.”

    As an expression host, P. pastoris combines elements of both prokaryotic and eukaryotic systems, such as a rapid growth rate and the ability to perform post-translational modifications (PTMs).

    The problem is that current commercially available Pichia systems are only able to produce low amounts of protein. According to Polizzi and her co-authors, the productivity of P. pastoris-based cell-free systems usually ranges from 6 to 100 µg/mL, which is only approximately five percent of that achieved by comparable E. coli systems. In addition, the additives required by Pichia-based systems are more expensive than those required by equivalent platforms.

    Additives to improve yields

    To address this, Polizzi and co-authors systematically evaluated a variety of chemical additive combinations to identify the most effective stabilizers and crowding agents to be incorporated in the reaction.

    The researchers also used a machine learning model to predict translation initiation rates and optimized the Kozak sequence—the protein translation initiation site in most eukaryotic mRNA transcripts—to enhance expression.

    In addition, the Imperial team evaluated lower-cost glycolytic intermediates as substrates for ATP regeneration to reduce the cost of goods.

    Polizzi says, “We focused on how to improve the yields and reduce the cost of production. We identified some additional additives that boost the yield without substantially increasing the cost. We also identified a different energy source that can be used.”

    She adds, “This work underscores the importance of protein-stabilizing additives and the role of rationally designed DNA sequences with minimized mRNA structural complexity to enhance yield in CFPS. Our demonstration of glycolytic intermediates as a potential secondary energy system additionally provides the foundation for the development of a cost-effective P. pastoris CFPS.”

    The post Yeast We Can: Cutting Costs by Optimizing Cell-Free Expression Systems appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Why AI Alone Isn’t Enough for Oligonucleotide Discovery https://edusehat.com/en/why-ai-alone-isnt-enough-for-oligonucleotide-discovery https://edusehat.com/en/why-ai-alone-isnt-enough-for-oligonucleotide-discovery Thu, 14 May 2026 05:10:14 +0700 Edusehat Why, Alone, Isn’t, Enough, for, Oligonucleotide, Discovery AI is reshaping drug discovery, and nucleic acid–based medicines, including mRNAs, gene therapy, and oligonucleotide therapeutics, are no exception. By optimizing sequences and chemical modifications for experimental testing, AI accelerates discovery timelines, which  is particularly critical for oligo therapeutics, a modality central to the n=1 rare diseases, which afflict mostly young patients for whom there is additional urgency.

    However, a familiar caveat remains: AI is only as powerful as the data from which it learns. How can we provide enough high-quality input data to fuel this engine and design next-generation precision medicine?

    A typical workflow for developing an AI-powered oligo predictive model begins with collecting experimental outcomes of oligo sequences, with each sequence annotated using a defined set of features. This data is then used to train AI models that identify patterns associated with improved activity and safety.

    However, as is often the case with pioneering technologies such as oligonucleotides, the scarcity of data is a major problem. To overcome this limitation, scientists trawl public resources such as publications and patents to extract this data. ASOptimizer, OligoAI, and eSkip-Finder are examples of newer oligo-predicting AI models that are trained using publicly available data.

    While these models are advancing in the right direction, relying primarily on this data comes with several disadvantages, such as:

    • inconsistent experimental conditions between the datasets,
    • limited diversity in sequences and chemistries,
    • lack of negative data, and
    • insufficient coverage of critical information such as toxicity and off-target effects.

    Furthermore, since data sourcing and annotating often require the use of automated, AI-powered tools, there is a risk of mislabeling and misinterpretation. As such, correlation statistics between predicted and experimental values for these models are not too high, generally hovering between 0.4 and 0.7.1,2, 3

    Building the data foundation for AI drug discovery

    The most valuable training data is:

    • designed to span broad chemical space and probe critical safety features,
    • produced under controlled conditions,
    • consistently processed and annotated, and
    • generated in a controlled environment, ideally internally.

    Large-scale screening campaigns are essential in that context as they provide the dense, reliable, datasets required to train AI models and extract meaningful insights for sequence and chemistry prediction.

    Ming Wang, PhD
    Ming Wang, PhD

    Brett Monia, CEO of Ionis Pharmaceuticals, describes this reality as “hard, brutal screening–screening a lot of oligonucleotides with different decorations, different amounts of chemistries, different sequences. We have plenty of (design) rules, but we still don’t have enough.”4

    One way to address this challenge is through intentional screening design: deliberately varying sequence motifs and positional chemistries within screening libraries to systematically explore chemical landscapes and expand the empirical foundation on which both rules and AI models are built.

    With the advent of faster and more affordable transcriptomic technologies, high-throughput RNA-seq can now be incorporated into oligonucleotide screening workflows. This method enables the systematic detection of off-target effects, including those that arise through mechanisms beyond straightforward.5,6

    While these approaches generate large and complex datasets, they represent a critical investment—one that lays the foundation for a faster, more efficient, and ultimately more cost‑effective future of oligo discovery.

    Digital infrastructure, engineering AI-ready data at scale

    While generating large datasets may be hard and brutal, managing, curating, and analyzing them doesn’t need to be. For data to be truly reliable and trustworthy, quality must be engineered from the start. Important aspects to consider include:

    • A single source of truth–a centralized FAIR data repository, where all data is systematically stored and governed for controlled access and use;
    • Comprehensive metadata capture, including protocols, batch numbers, and reagent references to ensure results can be interpreted correctly and are not driven by experimental artifacts;
    • Automated quality control and data analysis of large screens for large‑scale screens, ensuring consistent, efficient, and reproducible data processing; and
    • Consistent ontology and nomenclature for oligo sequences and their chemistries, as exemplified by Roche’s open-source tool (HelmShaker) for translating molecules into HELM notation.

    In practice, these principles are implemented through integrated digital infrastructures that combine molecular registration systems with automated analytics across diverse experimental modalities such as high‑throughput screening, next‑generation sequencing, mass spectrometry, and chromatography.

    Such approaches are increasingly used across the pharmaceutical and biotechnology sectors to manage oligonucleotide ADME, process development, and screening data, thus helping teams maintain data integrity and continuity throughout the oligo discovery and development lifecycle.

    AI promises to redefine what is possible in oligo discovery, and the field is already beginning to see its impact. But AI alone is not the breakthrough—data is. Only large, high‑quality experimental datasets, generated intentionally and prospectively, can unlock AI’s full predictive power.

    Organizations that invest early in both systematic data generation and robust data infrastructure will be best positioned to lead the next wave of oligonucleotide discovery. This shift is especially urgent for n = 1 rare diseases, where speed, precision, and learning from every experiment can make the difference between possibility and progress.

    Ming Wang, PhD, is scientific business manager at Genedata.

    References:

    1Hwang, G., Kwon, M., Seo, D., Kim, DH., Lee, D., Lee, K., Kim, E., Kang, M., Ryu, JH., ASOptimizer: Optimizing antisense oligonucleotides through deep learning for IDO1 gene regulation. Mol Ther Nucleic Acids. 2024 Apr 6;35(2):102186. doi: 10.1016/j.omtn.2024.102186

    2Chiba, S., Lim, KRQ., Sheri, N., Anwar, S., Erkut, E., Shah, MNA., Aslesh, T., Woo, S., Sheikh, O., Maruyama, R., Takano, H., Kunitake, K., Duddy, W., Okuno, Y., Aoki, Y., Yokota, T. eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping. Nucleic Acids Res. 2021 Jul 2;49(W1):W193-W198. doi: 10.1093/nar/gkab442

    3Hill, B., Jaques, M.R., Nair, RR., Whiffin, N., Wood, MJA., Sanders, SJ., Oliver, PL., Hill, AC., Rinaldi, C. Accurately modelling RNase H-mediated antisense oligonucleotide efficacy. bioRxiv. 2025 Oct 30. https://doi.org/10.1101/2025.10.29.685292

    4Accelerating Oligonucleotide Therapeutics. Evotec eBook.

    5Pekker, D., Kuntz, S., McArthur, M., Nicholson-Shaw, T., Yanke, S., Mukhopadhyay, S. A Dose-Response Model for Accurate Detection and Quantification of Transcriptome-Wide Gene Knockdown for Oligonucleotide-Based Medicines. bioRxiv. 2024 May 29. https://www.biorxiv.org/content/10.1101/2024.05.28.596270v1.full.pdf

    6In-silico siRNA Off-Target Predictions: What Should We Be Looking For? OTS Oligonucleotide Therapeutics Society, Webinar, 2024 May 2

     

     

    The post Why AI Alone Isn’t Enough for Oligonucleotide Discovery appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ApexGO: AI-Driven Approach to Faster Antibiotic Discovery https://edusehat.com/en/apexgo-ai-driven-approach-to-faster-antibiotic-discovery https://edusehat.com/en/apexgo-ai-driven-approach-to-faster-antibiotic-discovery Thu, 14 May 2026 05:10:14 +0700 Edusehat ApexGO:, AI-Driven, Approach, Faster, Antibiotic, Discovery

    Antibiotic resistance is on the rise around the world, creating an urgent need for faster and more dependable approaches to design antimicrobial candidates. While AI-driven methods have accelerated antimicrobial discovery, most have focused on screening fixed libraries or generating broad candidate sets.

    Now, researchers at the University of Pennsylvania have developed ApexGO—a novel, AI-powered method that starts with a small number of candidates and improves them, using a predictive algorithm to evaluate each modification and guide the next.

    “Antibiotic discovery is fundamentally a search problem across an enormous molecular space. ApexGO gives us a way to navigate that space with far more direction,” says César de la Fuente, PhD, presidential associate professor in the School of Engineering and Applied Science at UPenn.

    This work is published Nature Machine Intelligence in the paper, “A generative artificial intelligence approach for peptide antibiotic optimization.”

    “What is striking is that ApexGO’s predictions held up in the real world,” says Jacob R. Gardner, PhD, assistant professor in computer and information science (CIS) at UPenn. “ApexGO was optimizing against another computer model, so one concern was that it might find molecules that looked good to the model but failed in the lab. Instead, the majority of the molecules it designed actually worked.”

    indeed, 85% of the AI-generated molecules halted bacterial growth, while 72% outperformed the peptides from which they were derived. In mice, two antimicrobial peptides created by ApexGO reduced bacterial counts at levels comparable to the antibiotic polymyxin B.

    “This result points toward a future in which we can optimize molecules for a desired function in a fraction of the time,” adds de la Fuente, “using machines to guide discovery through chemical spaces too vast for humans to explore by trial and error.”

    For years, the de la Fuente lab has looked for antibiotic candidates in unlikely places, from frog secretions to ancient microbes. Two years ago, the group released APEX, an AI model that predicts whether or not a given peptide is likely to have antimicrobial properties.

    “APEX helped us find promising antibiotic candidates in enormous biological datasets,” says Marcelo Torres, PhD, research assistant professor of psychiatry in the Perelman School of Medicine. “ApexGO takes the next step: once we have a promising molecule, it helps us ask how to make it better.”

    One part of ApexGO (short for APEX Generative Optimization) suggests molecular tweaks, while the previously published APEX model predicts whether those changes are likely to increase antimicrobial activity. ApexGO then uses those predictions to guide the next round of proposed edits.

    While some of the molecules proposed by ApexGO showed promising antibiotic activity, the researchers emphasize that even the best-performing peptides are still early-stage candidates. Before any could be used to treat infections in humans, they would need to be further optimized for safety, stability, and how long they remain active in the body.

    Still, the study suggests that AI can help researchers decide which molecules are worth making and testing in the first place. For de la Fuente, the approach could eventually extend beyond antibiotics. “In this case, we wanted to optimize peptides for antimicrobial activity,” he says. “But you could imagine applying the same idea to peptides with other biological functions, like modulating the immune system or targeting tumors.”

    “ApexGO shows that AI can do more than predict which molecules might work: it can help us improve them,” adds de la Fuente. “At a time when antibiotic resistance is rising worldwide, we need technologies that help us move faster from an idea to a real therapeutic candidate. ApexGO is an important step toward that future.”

    The post ApexGO: AI-Driven Approach to Faster Antibiotic Discovery appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Synthesizing a Natural Sunscreen and Antioxidant in E. coli https://edusehat.com/en/synthesizing-a-natural-sunscreen-and-antioxidant-in-e-coli https://edusehat.com/en/synthesizing-a-natural-sunscreen-and-antioxidant-in-e-coli Thu, 14 May 2026 05:10:12 +0700 Edusehat Synthesizing, Natural, Sunscreen, and, Antioxidant, coli Researchers at Jiangnan University have engineered microbial “cell factories” to sustainably produce the UV-protective compound gadusol, a compound that could eventually serve as a sunscreen ingredient and an antioxidant additive.

    Found in the eggs of various fish and other marine organisms, gadusol helps protect against ultraviolet damage. However, “it is scarce in nature, and extracting it is inefficient and can carry environmental costs,” said research lead Ping Zhang, PhD. “We want to find a scalable and greener way to produce gadusol.” Previous efforts to acquire gadusol have relied mainly on extraction from fish eggs or tissues at specific developmental timepoints. For their reported study, rather than harvest the molecule from nature, the team turned microbes into mini chemical factories, increasing yield more than 90 times.

    The researchers reported on their developments in Trends in Biotechnology, in a paper titled “Multidimensionally engineered Escherichia coli for efficient gadusol biosynthesis with high-throughput quantitative analysis, in which concluded “This work provides valuable insights into gadusol biosynthesis and establishes a solid basis for future investigations into the feasibility of its industrial applications.”

    How do fish survive sunlight in the open waters without getting burned? They make their own natural sunscreen. Gadusol is “a naturally occurring cyclohexenone compound that has attracted significant attention due to its potent UV-absorbing properties and broad biological functionalities,” the authors wrote. “Gadusol, as a cyclohexenone compound with potent antioxidant and biological properties, has shown significant potential for applications in pharmaceuticals, cosmetics, and nutraceuticals.”

    The compound has been identified in the eggs of various fish species, including zebrafish, salmon, and sturgeon, as well as in coral reef ecosystems, where it shields marine embryos and coral from UV radiation. Moreover, the researchers explained, “Beyond its photoprotective role, gadusol also demonstrates pronounced antioxidant activity, with extracts from fish eggs exhibiting free radical scavenging capacity comparable to that of ascorbic acid under physiological pH and even superior chain-breaking activity against peroxyl radicals.”

    However, the team pointed out, extracting gadusol from fish eggs is labor intensive and costly, and results in low yields.  For their newly reported study the team rebuilt a zebrafish’s pathway for making gadusol inside the bacterium Escherichia coli, then tweaked the microbes’ genetics and growing conditions. “In this study, we constructed the gadusol biosynthetic pathway in E. coli and established an integrated engineering strategy to enable its efficient production,” they noted.

    These modifications increased gadusol yield by nearly 93 times, from 45.2 milligrams per liter to 4.2 grams per liter. The compound’s antioxidant property inspired a useful shortcut, in the form of a color-based screening test. In the test, a purple chemical signal turns yellow when gadusol neutralizes free radicals. This color shift allows researchers to quickly identify bacterial strains that produce more of the compound. “Compared with traditional chemical analysis, this approach is more convenient, efficient, and economical,” said senior author Ruirui Xu,” PhD.  “In addition, an efficient downstream process for gadusol extraction and purification was established, strengthening the translational potential of this platform,” the authors noted.

    Producing gadusol through a microbial cell factory for sun protection. [Science Center for Future Foods, Jiangnan University]
    Producing gadusol through a microbial cell factory for sun protection. [Science Center for Future Foods, Jiangnan University]
    The lab-made compound showed promise in preliminary UV-protection tests. “Achieving this level of production in the lab is very promising,” says Zhang. “It suggests that we may be able to meet future demand for natural sunscreen ingredients through microbial production.” The compound may offer more than just sun protection. In experiments, gadusol showed antioxidant activity comparable to that of vitamin C, suggesting it may help neutralize cell-damaging free radicals from UV exposure.

    In their paper the authors suggested that “Beyond laboratory-scale optimization, this work demonstrates a practical and scalable route for manufacturing gadusol, a natural, nontoxic UV-protective, and antioxidant cyclohexenone.” The research comes amid growing interest in alternatives to some conventional sunscreen ingredients, which can irritate sensitive skin, harm marine organisms, or rely on petrochemicals. Gadusol’s combination of UV protection and antioxidant activity could make it an attractive ingredient for sunscreens and skin-care products. The team in addition pointed out, “By enabling sustainable microbial production independent of marine extraction, this strategy provides a renewable alternative to synthetic UV filters and antioxidants that may raise environmental or health concerns.”

    But gadusol won’t make it into our beach bags just yet. The study did not compare gadusol head-to-head with commercial sunscreens, nor assess long-term safety or large-scale manufacturing. Regulatory approval would also be needed. Still, Xu believes that the research provides a starting point for moving gadusol toward practical applications. Based on current technology, he expects to start seeing some products appear on the market within two years.

    “For small molecules with application potential, we hope people look beyond traditional extraction methods,” Zhang commented. “Microbial cell factories are emerging as a greener and more sustainable way to bring laboratory discoveries into real-world use.”

    The post Synthesizing a Natural Sunscreen and Antioxidant in E. coli appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ASGCT President Terry Flotte Touts Rare Disease Initiatives as His Term Ends https://edusehat.com/en/asgct-president-terry-flotte-touts-rare-disease-initiatives-as-his-term-ends https://edusehat.com/en/asgct-president-terry-flotte-touts-rare-disease-initiatives-as-his-term-ends Thu, 14 May 2026 05:10:10 +0700 Edusehat ASGCT, President, Terry, Flotte, Touts, Rare, Disease, Initiatives, His, Term, Ends President of the American Society of Gene and Cell Therapy (ASGCT), Terry Flotte, MD, is excited to host this year’s conference in his own backyard. It will be a short drive east on the Mass Turnpike from his office at UMass Chan Medical School in Worcester to the Menino Convention and Exhibition Center in Boston’s Seaport district. Flotte is hopeful that the 2026 conference will draw the largest attendance in the meeting’s history. His tenure as president ends this week on the last day of the conference, May 15.

    In the run-up to this year’s conference, GEN spoke with Flotte, who is also Editor in Chief of GEN’s sister journal Human Gene Therapy, about the central themes and most anticipated sessions at this year’s conference. “I have a full dance card, let me tell you,” Flotte joked. The conference will highlight several themes of Flotte’s productive tenure.

     

    (This interview has been edited for length and clarity.)

     

    GEN: Terry, what’s the theme of this year’s ASGCT conference?

    Terry Flotte: We’re working very hard on access for rare and ultra-rare conditions and have been for some time. You’ll see that in the presidential symposium. This is in the context of our mission to improve access to rare disease cell and gene therapy (CGT). This is the guiding principle of our strategic plan: we want to work for universal access to CGT. There are two orthogonal axes to this: I’m focusing on rare and ultra-rare diseases. ASGCT is going to continue to work in parallel on universal access in a more global context.

    We have created a first-of-its-kind exchange for shelved CGTs. An increasing number of CGTs for rare and ultra-rare diseases are being discontinued or deprioritized after they reach the clinical stage—not because they lack clinical efficacy but because they lack market viability. We have partnered with Orphan Therapeutics Accelerator to create a new entity called CGTxchange. This collaborative venture is meeting the need of these promising clinical-stage CGTs that are not progressing. This entity will be an AI-enabled digital platform that will list the available clinical-stage CGT programs and generate AI-enhanced profiles, digest the data, score them for their level of advancement and the robustness of their responses, and essentially shorten the due diligence that investors normally have to do, enabling the connections to work faster.

    I estimate there’s at least 50-100 of these programs. We had our own personal experience with Sio Gene Therapies [formerly Axovant] on both GM1 and GM2 gangliosidosis. This is part of a broader set of initiatives. Over the past few years, we created a taskforce in response to this increasing rate of discontinuation of these therapies. The two main outgrowths that the ASGCT board has endorsed are to create a consortium of CGT developers that might be able to offer non-profits less expensive manufacturing in a limited way but also work toward a drug master file sharing data for those who benefit from the less expensive vectors—in addition to the clearinghouse I just mentioned.

     

     

    GEN: What else is new this year?

    Flotte: A new thing for ASGCT is we’re having a patient advocate presenting. Terry Pirovolakis pioneered the CGT therapy for spastic paraplegia type 50 (SPG50) by developing his own company, Elpida Therapeutics, which has taken SPG50 to the clinic and now is doing that for other rare and ultra-rare diseases.

    The second example is from Claire Booth, MBBS, PhD, (Great Ormond Street Children’s Hospital, London). Her team has received market authorization to be the

    pseudo-commercial manufacturer of a fully licensed therapeutic for different forms of SCID.

    Those are two direct examples of alternatives to get things to the clinic, other than getting a new commercial sponsor. [Hopefully] we can end up getting more of those picked up, whether through the CGTxchange or direct outreach. We’re also going to honor Timothy Yu, MD, PhD, with the Jerry Mendell Translational Research Award. He will be talking about the N=1 Collaborative with the parallel effort with oligonucleotide therapeutics. There is a purposeful theme to this meeting, aiming to make a big change in how things can get to the clinic and stay in the clinic.

     

    GEN: Last year in New Orleans, the conference was dominated by the Baby KJ story. Will anything stand out in the same way this year?

    Flotte: We are honoring the three primary authors of the Baby KJ story—Kiran Musunuru, MD, PhD, Rebecca Ahrens-Niklas, MD, PhD, and Fyodor Urnov, PhD.

    I have also selected the work of Lindsey George, MD (Children’s Hospital of Philadelphia) as a presidential abstract. She is going to present the first case of an AAV-induced tumor—or at least an aggressive and autonomously growing malignancy…. This occurred in an MPS1 patient who received a high dose of AAV into the ventricles. It is not exactly a meningioma, but it’s arising from the neuroepithelial cells lining the ventricles. The tumor has AAV integrated with a strong promoter immediately upstream of a known oncogene. I put that into the presidential lecture, even though it’s not good news—but I’m not a [gene therapy] campaign manager here! I think this is a significant finding that we’ll have to pay attention to.

    Lindsey is not saying that nobody should ever do this again. She’s going to point out aspects of this that were very manageable and how this patient overall has a dramatically better outcome than they would have without the therapy. In a way, [this is] somewhat like when those leukemia cases developed in Europe in the early SCID [gene therapy] trials. It is in a way parallel to that.

     

    GEN: This will be your last conference as president of ASGCT!

    Flotte: Yes, it ends on May 15th! We only get to be president for one year. I started the Rare Disease Task Force as vice president. This was my cause over the past three years [as an officer]. I’m very pleased we were able to stand this up.

    We have an actual corporation, a joint venture, 50% owned by ASGCT. We set up this manufacturing consortium. Somewhat related, we set up our own charitable foundation, the ASGCT Foundation. We will have our first event—a gala at the conference. It will have a lot of time to grow. The foundation has just been incorporated as a subsidiary not-for-profit.

     

    GEN: How do you view things at FDA currently?

    Flotte: We will have a fireside chat with the new director of CBER, Katherine Szarama, PhD. We are very encouraged—she’s a very highly trained professional. We love that FDA is paying a lot of attention to rare diseases, but we need some scientific and evidence-based guidelines on how to do this consistently. We’re looking to someone who has regulatory experience.

     

    GEN: What else has got you and your colleagues in the gene therapy space excited of late?

    Flotte: I’m hoping we’re going to better understand high-dose AAV toxicity… I think what we’ve got is several different syndromes, but many of them may have a common link… We’ve been seeing with high-dose AAV a very broad distribution, but the doses are incredibly high and there have been deaths—the DMD patient deaths that occurred in the first two weeks are the best-known examples, but there have been other ones.

    In my lab, we’re trying to figure out the primary pathogenesis. We have found a number of situations with unexpected vector expression in the endothelial cells and then seeing vascular leakage into some of these tissues causing tissue injury. So, in the post-mortem analysis we helped on, we saw high expression in the lungs and alveolar capillaries. They had diffuse leakage into the capillaries leading to a syndrome known as acute respiratory distress syndrome (ARDS). But in some of the others where they’re seeing some complement activation, we think that small vessel injury could be a convergent pathway. Now, where does this come into play in the broader sense?

    One of the holy grails of recent AAV gene therapy is to design an AAV capsule that efficiently crosses the blood-brain barrier. Many diseases that are appropriate for AAV are diseases of the central nervous system (CNS). You can think of, for instance, the easiest cells to access in the CNS are the spinal motor neurons, hence the SMA1 treatment, Zolgensma. So, if you treat an SMA newborn, that is essentially solved or at least adequately solved. But in none of the diseases that affect the brain have we seen an IV gene therapy that is robustly efficacious—just giving an AAV at a high enough dose to get across the blood-brain barrier. Many different companies are trying to develop AAV capsids that will penetrate the blood-brain barrier, the first one that got to clinic was a vector designed by Capsida Biotherapeutics. But the first patient treated on the Capsida trial developed cerebral edema and died.

    One of the important challenges for the field is to understand if we can separate a blood-brain barrier penetration from endothelial cell toxicity, because you could think perhaps a vector designed to get through the blood-brain barrier could cause injury as it crosses to the endothelial cells in the brain. I think there may be ways around this, but to me this is a central issue because the CNS is affected in so many single-gene disorders. The parents see a child who has a disability or degenerating, as in Tay-Sachs, and they want to be able to do an IV therapy. They don’t want to have to have a direct brain injection or some other invasive intervention. So that’s what I’m looking for at ASGCT 2026.

     

     

    The post ASGCT President Terry Flotte Touts Rare Disease Initiatives as His Term Ends appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BMS, Hengrui Pharma Partner on 13 Programs in Up-to-$15.2B Collaboration https://edusehat.com/en/bms-hengrui-pharma-partner-on-13-programs-in-up-to-152b-collaboration https://edusehat.com/en/bms-hengrui-pharma-partner-on-13-programs-in-up-to-152b-collaboration Thu, 14 May 2026 05:10:09 +0700 Edusehat BMS, Hengrui, Pharma, Partner, Programs, Up-to-15.2B, Collaboration Bristol Myers Squibb (BMS) will partner with Hengrui Pharma to co-develop 13 early-stage programs in oncology, hematology, and immunology, the companies said today, through a collaboration that could generate more than $15.2 billion for the Chinese drug developer.

    BMS and Hengrui have inked global strategic collaboration and license agreements covering the 13 candidates—consisting of four oncology/hematology assets from Hengrui, four immunology assets from BMS, and five “innovative” assets to be jointly discovered and developed by both companies.

    The companies said their collaboration is intended to combine BMS’ research and discovery strengths, global clinical development capabilities, regulatory expertise, and commercial scale with Hengrui’s discovery engine, platform technologies, and efficient early-stage development expertise.

    To that end, Hengrui has agreed to fully oversee early clinical development in order to accelerate clinical proof of concept for these programs. Hengrui has the option to co-develop select assets and the potential to conduct certain commercialization activities globally with BMS.

    “By leveraging Hengrui’s growing R&D capabilities and proven efficiency in discovering and advancing innovative therapies, we are poised to advance the best of both pipelines,” Frank Jiang, MD, PhD, Hengrui’s executive vice president and chief strategy officer, said in a statement. “It also reflects Hengrui’s continued commitment to strengthen our global presence.

    BMS will obtain exclusive worldwide rights to the Hengrui‑originated candidates outside China, Hong Kong Special Administrative Region (SAR), and Macau SAR—Hengrui’s territory of operation—while Hengrui will gain exclusive rights to the BMS‑originated assets within those areas, with BMS retaining rights for the rest of the world.

    $950M over two years

    BMS has agreed to pay Hengrui up to $950 million over two years, to consist of a $600 million upfront payment, a $175 million first anniversary payment, and a second contingent anniversary payment of $175 million in 2028.

    The approximately $15.2 billion value of the collaboration includes exercising available options for the joint discovery programs and achieving development, regulatory, and commercial milestones for all programs. Hengrui also is eligible to receive tiered royalties on net sales of products commercialized outside its territory.

    The collaboration deal is expected to close in the third quarter, subject to review under the Hart‑Scott‑Rodino Antitrust Improvements Act and other customary closing conditions.

    “This strategic collaboration reflects our commitment to advancing innovative science while maintaining a disciplined approach to portfolio management,” stated Robert Plenge, MD, PhD, BMS executive vice president and chief research officer. “By leveraging complementary capabilities across geographies, we aim to accelerate early clinical learning and make informed decisions that support driving top tier growth in the next decade and, ultimately, our mission to deliver medicines that help patients prevail over serious diseases.”

    Recouping ‘patent cliff’ losses

    Behind that focus on top-tier growth for BMS, as with other pharma giants, is a quest to recoup the billions of dollars in sales it stands to lose as aging blockbuster drugs head for the proverbial “patent cliff” by losing exclusivity in the U.S. and other key markets.

    Of the Top 20 Drugs Heading for the Patent Cliff through 2029—the subject of a GEN A-List last November—BMS had three marketed treatments: The cancer drug Revlimid® (lenalidomide), indicated for forms of multiple myeloma, myelodysplastic syndromes, and three forms of lymphoma, which lost U.S. exclusivity in January; and two drugs set to lose exclusivity in 2028: the cancer immunotherapy Opdivo® (nivolumab), and the factor Xa-inhibiting blood thinner Eliquis® (apixaban).

    Eliquis generated $14.443 billion in product revenue last year plus another $4.137 billion in the first quarter. Opdivo made $10.049 billion in 2025 plus $2.146 billion in Q1, while Revlimid racked up $2.951 billion and $349 million.

    BMS has laid groundwork for rebuilding its pipeline over the past year through a series of collaborations and acquisitions with companies that include:

    • Janux Therapeutics: An up-to-$850 million partnership announced in January to co-develop a tumor-activated therapeutic targeting an undisclosed “validated solid tumor antigen expressed across several human cancer types.” ($50 million upfront).
    • Harbour BioMed: An up-to $1.125 billion partnership with the Chinese biopharma—owned to discover and develop next-generation multi-specific antibodies ($90 million upfront), announced in December 2025.
    • Orbital Therapeutics: A $1.5 billion cash acquisition of the developer of RNA therapies designed to treat disease by reprogramming cells in vivo, announced in October 2025.
    • 2seventy bio: An approximately $286 million buyout of its partner in developing the blockbuster multiple myeloma drug Abecma® (idecabtagene vicleucel), announced in March 2025. Abecma made $427 million last year. The drug’s sales are no longer reported individually but within BMS’ “Growth portfolio” that garnered $581 million in Q1 2026.

    Five castoffs

    BMS also outlicensed five pipeline assets to Beeline Medicines, an autoimmune and inflammatory drug developer formed in April with a $300 million Series A financing from Bain Capital. Beeline’s pipeline of BMS castoffs includes afimetoran, being developed for both cutaneous lupus erythematosus (CLE) and systemic lupus erythematosus (SLE), BMS-986326 (atopic dermatitis, CLE, and SLE); lomedeucitinib (formerly BMS-986322, plaque psoriasis), and two IND-stage next-generation biologics for unspecified diseases that target the IL-18 and IL-10 pathways.

    Hengrui last September outlicensed its cardiac myosin inhibitor RS-1893 to Braveheart Bio ($65 million upfront, up to $1.013 billion in milestones); and two months earlier inked an up to $12.5 billion ($500 million upfront) partnership with GlaxoSmithKline (GSK) to develop to develop chronic obstructive pulmonary disease (COPD) candidate HRS-9821 and 11 additional programs across respiratory, immunology and inflammation, as well as oncology indications.

    The post BMS, Hengrui Pharma Partner on 13 Programs in Up-to-$15.2B Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     “It Was Not a Cure”: Musunuru Cautions ASGCT on Baby KJ Promise https://edusehat.com/en/it-was-not-a-cure-musunuru-cautions-asgct-on-baby-kj-promise https://edusehat.com/en/it-was-not-a-cure-musunuru-cautions-asgct-on-baby-kj-promise Thu, 14 May 2026 01:35:15 +0700 Edusehat “It, Was, Not, Cure”:, Musunuru, Cautions, ASGCT, Baby, Promise BOSTON – When Kiran Musunuru, MD, PhD, walked to the microphone to deliver remarks on behalf of the team that won the American Society of Gene and Cell Therapy (ASGCT) 2026 Catalyst Award, most of the thousands of attendees surely expected a feel-good speech.

    After all, it was 12 months ago that Musunuru, addressing the same convention in New Orleans, shared the exciting news regarding the delivery of a bespoke base editor to an infant, Baby KJ, with a rare urea cycle disorder. Musunuru and his colleague, Rebecca Ahrens-Niklas, MD, PhD, were recently named to the TIME 100 Most Influential People of 2026. “A decade from now,” stated Nobel laureate Jennifer Doudna, PhD, “their names will be in medical textbooks, not only for Baby KJ, but for opening the door to personalized genetic medicine for thousands of children after him.”

    Musunuru and Ahrens-Niklas, from the University of Pennsylvania and Children’s Hospital of Philadelphia (CHOP), respectively, were honored alongside Doudna’s colleague Fyodor Urnov, PhD (Innovative Genomics Institute) and Danaher Corporation, for building the remarkable academia-industry consortium that designed and delivered the gene editing therapy, resulting in Baby KJ’s discharge from CHOP and a wave of national television appearances.

    Indeed, Musunuru opened his ASGCT remarks in upbeat mood. “The potential is there to [deliver personalized therapies] over and over again for hundreds of diseases centered in the liver.” But halfway through his speech, Musunuru’s tone changed. While most grateful for the recognition from ASGCT, he said it was important to always “be your own worst critic.”

    “I’ll be brutally honest,” Musunuru said. Despite the unquestionable “enthusiasm and excitement” surrounding the Baby KJ story, “there are some profound limitations. It was not really science at all!” Musunuru continued. “It was not a clinical trial. It was not clinical research. It was not a cure.”

    “The best we can say is we hope we’ve turned a devastating disease into a milder, manageable condition. But it’s too early to say that… This was a personalized N-of-1 therapy—we can’t say what this means for anyone.”

    Drawing applause from the audience, Musunuru pushed on: “We mustn’t be snake oil salesmen or give false hope… We have a profound ethical responsibility not to mislead families over what is possible.”

    “We don’t actually know anything,” Musunuru said. “We need to do clinical trials—scientifically and ethically.”

    The path forward

    Musunuru set the Baby KJ story in the broader context of his group’s work on phenylketonuria (PKU), one of the classic inborn errors of metabolism. A few years ago, Musunuru and Ahrens-Niklas set about designing gene editing therapies targeting the first and sixth most common PKU mutations using adenine base editors. (There are more than 1,000 known mutations that cause PKU.)

    After testing in humanized mouse models, the researchers were delighted to see the phenylalanine levels rapidly drop to normal, sustained for the lifetime of the mice. Flush with funding from the Somatic Cell Genome Editing program at NIH, Musunuru and Ahrens-Niklas began talks with the U.S. Food and Drug Administration in February 2024 to settle the question: Do we need separate Investigational New Drug applications (INDs) for each PKU variant?

    “It is basically the same drug, the same gene, the same disease, the same clinical endpoints. Can’t we cover both variants in a single IND and a single ‘umbrella’ clinical trial?” summarized Musunuru. The answer was “maybe”—the agency needed to consider the full implications of the proposal.

    The Philadelphia team began to develop workflows for four more PKU mutations, leading them to propose an umbrella trial for a revised total of six variants. Following another meeting with FDA officials in early 2025, the response was extremely positive: a single IND application would be appropriate, with a single toxicology study conducted in a single species. The FDA also agreed to consider additional variants.

    In parallel, Ahrens-Niklas and Musunuru were studying sick patients with urea cycle disorders. Although these are liver disorders, “the real harm happens in the brain,” Musunuru said, resulting from toxic levels of ammonia. Enter Baby KJ’s diagnosis with CPS1 deficiency, and the notion that there was chance to design a personalized therapy.

    In the Fall of 2024, Musunuru and Ahrens-Niklas held a pre-IND meeting with FDA officials. The idea was to streamline applications for a group of urea cycle disorders caused by mutations in seven different genes.

    The FDA judged that all seven therapies could be evaluated in a single Phase I/II trial, but separate INDs would be required for each gene. “We’d have to do it piece by piece,” Musunuru said. First, file a master protocol for urea cycle disorders; after that IND clears, then file additional gene-specific INDs and amend the original IND.

    “This is how we can make the trial accessible to all UCD patients across the country,” he said.

    Back to the future

    Coming back to the present, Musunuru stated that although the primary IND had been filed, “this does not mean the trial is open or we can enroll patients.” Musunuru listed three major issues:

    • The team has not yet manufactured any gene therapy product.
    • As seven INDs are needed to fully open the clinical trial, it will be well into 2027 until all INDs are submitted.
    • In February 2026, the FDA issued a draft Plausible Mechanism Framework. Musunuru’s team held another pre-IND meeting with the FDA to advocate for the use of prime editing for urea cycle disorders. After all, Musunuru reasoned, why should therapies be restricted to base editing approaches (G-to-A substitutions) but not patients who harbor a G-to-C mutation? The FDA indicated that a separate IND/BLA would be needed for each gene, and that process validation should be finalized before any dosing of Phase II subjects.

    The path forward, Musunuru said, was to adopt an adaptive, real-time clinical trial design. That involves testing therapies, then advancing therapies from proof-of-concept to the validation phase. At that point, if all goes well, they can submit a BLA. Ahrens-Niklas and Musunuru laid out more details of their approach and dealings to date with the FDA in a commentary published late last year entitled: “How to create personalized gene editing platforms.”

    With that, Musunuru hastily closed and exited stage left to give a keynote address at another conference across the road.

     

    The post “It Was Not a Cure”: Musunuru Cautions ASGCT on Baby KJ Promise appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Technique Yields Uniform, High-Quality, EVs at Scale https://edusehat.com/en/technique-yields-uniform-high-quality-evs-at-scale https://edusehat.com/en/technique-yields-uniform-high-quality-evs-at-scale Thu, 14 May 2026 01:35:14 +0700 Edusehat Technique, Yields, Uniform, High-Quality, EVs, Scale Mesenchymal stem cell-derived extracellular vesicles (MSC-EVs) play an outsized role in intracellular communications, influencing such functions as inflammation and tissue repair. With the possible applications of these small, membrane-bound particles growing, an efficient, cost-effective production method has been on drug manufacturers’ wish lists for some time.

    A novel, streamlined chromatographic production and isolation method developed by scientists at Satorius BIA Separations in Slovenia may fulfill that wish, yielding uniform, high-quality EVs at scale. The method concentrates MSC-EVs directly from conditioned media. It also removes 97% of protein impurities and 95% of double-stranded DNA-related impurities, increasing their potential as therapeutics or drug delivery vessels.

    Microcarrier + suspension

    The method relies upon preferential exclusion chromatography, Katja Vrabec, head of product application area (EVs) at Sartorius, notes in a recent paper. In it, Vrabec and colleagues explain the method “uses monolithic hydroxyl columns to purify and concentrate the MSC-EVs,” and biochromatography analytics to track EV-specific surface antigens.

    First, the team expanded the MSCs in growth media, and then produced the EVs in a lean media formulation to limit production of protein and particle contaminants. That part is standard.

    Here’s what’s different: The scientists used a microcarrier-based system rather than flask-based 2D cultivation to scale the MSC cultures and increase the ratio of EVs to contaminants in conditioned media. They also used a suspension culture to enhance cell growth surface-to-volume ratios, and thereby increase EV yield. Then, they used a monolithic hydroxyl column to capture and purify the EVs directly from harvest.

    Increasing cell density and the cell-to-impurity ratio lowers buffer consumption downstream and lays the groundwork for biomanufacturers to transition to a scalable bioreactor system.

    Because the main impurities in EV harvests don’t interact with the chromatographic column in high-salt-binding conditions, the team recommends choosing a low-salt buffer for elution to reduce the need for buffer exchange before the polishing step. The optimal binding condition, they report, is “sodium citrate of 0.75M at pH 7.0.”

    This research highlights the need to consider upstream and downstream processing as a cohesive system, to design a simple, scalable, holistic process, and to apply reliable analytics. This all is particularly challenging, the team admits, given “the heterogeneous nature of EVs and the presence of similarly-sized components in biological samples.”

    The post Technique Yields Uniform, High-Quality, EVs at Scale appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Operator Protection as Core Design Principle for ADC Bioprocessing https://edusehat.com/en/operator-protection-as-core-design-principle-for-adc-bioprocessing https://edusehat.com/en/operator-protection-as-core-design-principle-for-adc-bioprocessing Thu, 14 May 2026 01:35:13 +0700 Edusehat Operator, Protection, Core, Design, Principle, for, ADC, Bioprocessing Antibody-drug conjugates (ADCs) continue to gain momentum as one of biopharma’s most promising therapeutic classes, particularly in oncology. But while the science behind ADCs advances rapidly, manufacturing these highly potent therapies forces two requirements to coexist: strict aseptic processing and high-containment handling of highly potent active pharmaceutical ingredients (HPAPIs).

    For Ashley Harp, a fellow in containment and bioconjugates at the consultancy CRB, operator protection is not simply an environmental health and safety issue—it is a core design principle for successful ADC bioprocessing.

    “One of the primary concerns in ADC bioprocessing is protecting operators from exposure to highly potent compounds, which can exist in both solid and liquid form,” Harp says. “Those risks extend far beyond the core manufacturing team to include quality control, maintenance, and calibration staff—anyone who may interact with the process or equipment over its lifecycle.” That broader view is increasingly important as commercial bioprocessors scale ADC production.

    Potential exposure points abound. “Across all stages of ADC bioprocessing, additional risks are associated with handling solid and liquid waste, collecting samples, changing or maintaining HVAC filters, and performing maintenance or calibration activities,” Harp says. These tasks often involve residual potent compounds that remain on equipment surfaces or within process systems, creating exposure risks long after active manufacturing ends.

    Tackling those risks starts long before production begins. “Addressing operator protection risks starts with rigorous risk assessments and the implementation of recommendations based on those assessments,” Harp says.

    She emphasizes the importance of involving experts in containment, industrial hygiene, and collaborative facility and equipment design early in project planning. Identifying hazards upfront makes it easier—and less costly—to build effective safeguards into the process rather than retrofitting them later.

    Where higher-risk activities cannot be avoided, Harp recommends multiple layers of protection rather than relying on a single solution. Closed processing systems, equipment designed to contain materials at the source, and technologies that support safe cleaning and transfer all play a role. Examples include rigid and flexible containment approaches, containment valves, split valves, specialized piping systems, continuous liners, containment enclosures, spray balls, wash wands, and manual wiping protocols.

    At the same time, smarter process design can reduce risk even further. “In parallel, thoughtful process development can reduce or even eliminate the need for direct personnel interaction with the manufacturing process,” Harp says. Technologies such as flow chemistry, process intensification, and robotics can significantly reduce manual handling and intervention, limiting the chances of exposure while also improving consistency.

    For commercial bioprocessors, implementing these solutions requires organizational alignment. “Successfully implementing these solutions requires early and ongoing collaboration across disciplines,” Harp says. Environmental health and safety, industrial hygiene, maintenance, calibration, operations, engineering, and quality teams all need to be involved from the beginning to form truly cross-functional design teams.

    Facilities must also remain flexible. ADC pipelines evolve quickly, and containment strategies need to evolve with them. Specialized expertise in integrating process equipment within high-containment environments is crucial, as are strong R&D capabilities—or partnerships that can provide them.

    Although “high containment requirements inherently increase the operational cost and complexity of manufacturing ADCs compared to non-potent therapies,” Harp argues that strategic improvements can offset some of those costs over time. Process intensification, reduced manual handling, and stronger containment can improve raw-material efficiency and reduce waste generation. Implementing new systems might initially extend development timelines or delay time to market, but the long-term result can be safer, more sustainable operations for both people and products.

    As ADC pipelines continue to expand, operator protection is shifting from a compliance checkpoint to a competitive necessity.

    The post Operator Protection as Core Design Principle for ADC Bioprocessing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Yeast We Can Cut Costs By Optimizing Cell-Free Expression Systems https://edusehat.com/en/yeast-we-can-cut-costs-by-optimizing-cell-free-expression-systems https://edusehat.com/en/yeast-we-can-cut-costs-by-optimizing-cell-free-expression-systems Thu, 14 May 2026 01:35:12 +0700 Edusehat Yeast, Can, Cut, Costs, Optimizing, Cell-Free, Expression, Systems Choosing the right additives could help “cell-free” expression systems finally fulfill their potential and provide biopharma with a low-cost way of making protein drugs, according to a recent research report.

    The new study looked at how cell-free systems, in which biochemical reactions occur independently of cells, could be fine-tuned to provide drug makers with alternatives for large-scale protein production.

    And the potential of the approach is significant, says Karen Polizzi, PhD, a professor from the department of chemical engineering at Imperial College London, who adds, “Cell-free protein synthesis (CFPS) is a flexible manufacturing technology. It can be used for on-demand synthesis in low-resource environments or to make difficult-to-express products, especially medicines that are toxic to the cell. Cell-free reactions scale well across microliter to liter scale without needing adjustments.”

    The Imperial team’s research focused on expression systems based on the yeast species Pichia pastoris, which, as Polizzi explains, “has machinery capable of post-translational modifications of proteins that can be necessary for function.”

    As an expression host, P. pastoris combines elements of both prokaryotic and eukaryotic systems, such as a rapid growth rate and the ability to perform post-translational modifications (PTMs).

    The problem is that current commercially available Pichia systems are only able to produce low amounts of protein. According to Polizzi and her co-authors, the productivity of P. pastoris-based cell-free systems usually ranges from 6 to 100 µg/mL, which is only approximately five percent of that achieved by comparable E. coli systems. In addition, the additives required by Pichia-based systems are more expensive than those required by equivalent platforms.

    Additives to improve yields

    To address this, Polizzi and co-authors systematically evaluated a variety of chemical additive combinations to identify the most effective stabilizers and crowding agents to be incorporated in the reaction.

    The researchers also used a machine learning model to predict translation initiation rates and optimized the Kozak sequence—the protein translation initiation site in most eukaryotic mRNA transcripts—to enhance expression.

    In addition, the Imperial team evaluated lower-cost glycolytic intermediates as substrates for ATP regeneration to reduce the cost of goods.

    Polizzi says, “We focused on how to improve the yields and reduce the cost of production. We identified some additional additives that boost the yield without substantially increasing the cost. We also identified a different energy source that can be used.”

    She adds, “This work underscores the importance of protein-stabilizing additives and the role of rationally designed DNA sequences with minimized mRNA structural complexity to enhance yield in CFPS. Our demonstration of glycolytic intermediates as a potential secondary energy system additionally provides the foundation for the development of a cost-effective P. pastoris CFPS.”

    The post Yeast We Can Cut Costs By Optimizing Cell-Free Expression Systems appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Adopting Creative Chemistry to Optimize Bioprocessing Workflow https://edusehat.com/en/adopting-creative-chemistry-to-optimize-bioprocessing-workflow https://edusehat.com/en/adopting-creative-chemistry-to-optimize-bioprocessing-workflow Thu, 14 May 2026 01:35:11 +0700 Edusehat Adopting, Creative, Chemistry, Optimize, Bioprocessing, Workflow Taking a creative approach to chemistry can help developers of antibody-drug conjugates (ADCs) improve the stability and purity of their products. That’s the view of Sunny Zhou, PhD, professor of chemistry and chemical biology at Northeastern University. Zhou will be speaking at the Bioprocessing Summit in Boston in August.

    According to Zhou, the structure of ADCs can make them vulnerable to bioprocessing issues that don’t affect traditional antibodies. As one example, he says, the payloads of antibody drug conjugates often significantly absorb above 280 nm, making them markedly more sensitive to light.

    “There’ll be photochemistry induced by the payload that can damage both the antibodies and payloads, such as crosslinking that likely leads to aggregation,” he says. “We’ve already published some work showing light-induced protein modifications, crosslinking, and aggregation.”

    According to Zhou, some initiatives are already underway to address this issue. For example, by engaging in antibody production and downstream processing in dim or safe light (e.g., yellow or red light) instead of the more commonly used bright white light.

    Another issue, he says, is that the linker connecting the antibody and drug payload is designed to be cleaved by enzymes in human patients.  On the other hand, it also means that similar enzymes in host cell proteins (HCPs) may prematurely cleave the linker during production and storage, thereby decomposing the drug and contaminating the final product.

    “Many host cell proteins contain such enzymes, but they don’t cleave antibodies. With these ADC linkers, however, enzymes that didn’t create problems before might do so now,” he says.

    Zhou explains that premature cleavage of ADC linkers has been observed in an industrial setting. Fortunately, he says, his research team, in collaboration with companies like Takeda, is already creating universal platforms and workflows to identify and effectively remove these potential HCP contaminants, as well as working to better understand the stability of the linkers.

    “These drugs circulate in the body for maybe two to three weeks, and stability issues can be amplified during circulation,” he says. “So, making the linker more stable [during manufacturing] may also help improve stability during circulation, further down the line.”

    Zhou’s team is now hoping to look at other creative chemistries in bioprocessing. Among these is, for example, removing reagents, by-products, and impurities by filtration, which may be faster than relying on chromatography, he says.

    The post Adopting Creative Chemistry to Optimize Bioprocessing Workflow appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cellares and ProTgen Automate Manufacturing of Progenitor T-Cell Therapy for Blood Cancer https://edusehat.com/en/cellares-and-protgen-automate-manufacturing-of-progenitor-t-cell-therapy-for-blood-cancer https://edusehat.com/en/cellares-and-protgen-automate-manufacturing-of-progenitor-t-cell-therapy-for-blood-cancer Thu, 14 May 2026 01:35:10 +0700 Edusehat Cellares, and, ProTgen, Automate, Manufacturing, Progenitor, T-Cell, Therapy, for, Blood, Cancer Cellares, an Integrated Development and Manufacturing Organization (IDMO) that combines automated manufacturing platforms with global Smart Factory infrastructure, and ProTgen, a therapeutic company pioneering targeted Notch activators to reactivate the thymus and reconstitute the adaptive immune system, have announced a partnership to automate manufacturing and quality control of ProT-096, ProTgen’s personalized progenitor T-cell therapy for patients with refractory leukemia and other hematologic malignancies. In this collaboration, Cellares will apply the company’s Cell Shuttle and Cell Q platforms to ProT-096 while providing regulatory support toward IND submission.  

    Fabian Gerlinghaus, co-founder and CEO of Cellares, says hematologic malignancies have waited too long for cell therapy to deliver on its promise, with manufacturing complexity being one of the main bottlenecks. ProT-096 represents “exactly the kind of innovative program” for which Cellares was founded. 

    “Early-stage developers should not have to choose between advancing their science and securing the manufacturing foundation they need to scale,” said Gerlinghaus. “By automating the manufacturing process and providing regulatory expertise toward IND submission, we can help ProTgen move faster and with greater confidence toward the clinic.” 

    Patients with refractory hematologic malignancies often face a compromised immune system following intensive treatment. While ProT-096 mandates precision manufacturing at scale, achieving the reproducibility, process consistency, and cost efficiency needed to support clinical development requires advanced manufacturing approaches. 

    Cell Shuttle’s automated, end-to-end manufacturing workflow reduces manual touchpoints, minimizes variability, and enables standardized execution across runs, equipment, and facilities. Combined with Cell Q, the workflow is designed to meet the demands of clinical- and commercial-scale production while maintaining quality standards for GMP manufacturing. The partnership also adds personalized progenitor T cells to Cellares’ platform capabilities across CAR T-cell therapies and HSC programs. 

    ProTgen’s proprietary targeted Notch activator platform programs cell fate both in vivo and ex vivo. The company’s initial focus is to reactivate the thymus and rebuild a diverse, functional immune repertoire for patients with compromised or aging immune systems. 

    Carter Cliff, CEO of ProTgen says ProT-096 represents a new approach to immune reconstitution, with the potential to address a significant unmet need for patients whose immune systems have been severely compromised by hematologic malignancy and prior treatment. 

    “This partnership allows us to pair our targeted Notch activator platform with an automated, scalable manufacturing foundation designed to support the path toward IND submission and, ultimately, clinical development,” he said. 

    The post Cellares and ProTgen Automate Manufacturing of Progenitor T-Cell Therapy for Blood Cancer appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     A plan to make drugs in orbit is going commercial https://edusehat.com/en/a-plan-to-make-drugs-in-orbit-is-going-commercial https://edusehat.com/en/a-plan-to-make-drugs-in-orbit-is-going-commercial Wed, 13 May 2026 22:05:03 +0700 Edusehat plan, make, drugs, orbit, going, commercial
  • A big deal: Varda Space Industries says it has signed a pharmaceutical company as a commercial customer, marking what could be a landmark moment for in-orbit manufacturing.

  • Space as a lab: The bet is that microgravity causes drug molecules to crystallize into atomic arrangements impossible on Earth, potentially unlocking new versions of existing medicines.

  • Economics favor drugs: At $7,000 per kilogram to reach orbit, space manufacturing is impractical for most industries — but blockbuster drugs can be worth over $100 million per kilogram, making them a rare exception to the brutal math of rocket launches.

  • Still more experiment than factory: Despite the excitement, no product has ever been manufactured in space, brought back, and sold on Earth.
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    Varda Space Industries, a startup that’s been pitching its ability to perform drug experiments in space, says it has signed up the pharmaceutical company United Therapeutics in what may be remembered as a notable step toward in-orbit manufacturing.

    The idea of building things in outer space for use on Earth has so far been explored mostly on board the International Space Station, and only in small-scale experiments backed by governments.

    But Varda, based in El Segundo, California, is now telling drug companies it has a practical, and repeatable, way to produce novel molecules in microgravity. 

    “This is the first commercial path to products made in space,” says Michael Reilly, Varda’s chief strategy officer.

    The scientific idea is that chemical mixtures have different properties under weightless conditions. For instance, water will hang together in a wiggly sphere, since without gravity, surface tension is the strongest force present.

    The plan is to launch versions of United Therapeutics’ drugs into orbit, where they can be allowed to form solid crystals. The hope is that in microgravity, they’ll take on atomic arrangements not seen on Earth, possibly leading to new versions with improved stability or other valuable properties.

    United is led by CEO Martine Rothblatt, who worked on early  telecommunications satellites. Since then, she’s built a multibillion-dollar health franchise with a succession of drugs to treat a lung disease called pulmonary arterial hypertension, which her daughter suffers from, and a subsidiary developing genetically modified pigs as a source of organs for transplantation.

    Rothblatt says space could be the next step if orbital conditions permit United to identify “even more amazing” versions of its drugs.

    Space to reformulate

    Pharmaceutical companies often try to keep their blockbuster franchises alive by creating improved versions of drugs or reformulating them—for example, making the switch from a pill to an inhaled version, as United has done with some of its products. Doing so can keep imitators at bay and create extra decades of patent protection.

    Assisting drugmakers are specialist companies, such as Halozyme and MannKind, that earn profits by helping to reformulate other companies’ drugs, often taking a royalty on future sales.

    That’s the business Varda has been trying to break into—by using excursions into space instead of nebulizers, patches, or nanoparticles. The company was formed in 2021 by Delian Asparouhov, a partner at Peter Thiel’s Founders Fund, along with Will Bruey, a former avionics engineer with Elon Musk’s SpaceX who is now the company’s CEO.

    The pair’s bet is that space manufacturing will become viable once rocket launches become frequent enough—and cheap enough—to support a business model in which raw materials are sent into orbit, processed, and then returned to Earth in a new form.

    And that’s starting to happen. To get into space, Varda has been purchasing rides from SpaceX—which now launches a rocket every two or three days, usually a reusable Falcon 9. 

    Those rockets have a nose cone, or payload fairing, about the size of a moving truck that gets filled with satellites or instruments, which are then released into orbit.

    Starting in 2023, Varda began sending up small satellites that have a boulder-size capsule attached. The capsule contains equipment to carry out experiments, and it can detach and fall back to Earth, entering the atmosphere at a speed of around Mach 25 before slowing via air resistance and eventually drifting to land with a parachute. (Varda lands its craft in the Australian outback.)

    That speedy reentry has also drawn interest from the US military, including the Air Force, which has paid Varda to fly instruments and take measurements relevant to hypersonic missile technology. Of the six craft Varda has paid to put into orbit so far, half have been dedicated to military research and half carried drug-related demonstrations. 

    At Varda, such “dual use” of technology is accepted as part of being in the space business, which remains reliant on government support. The company’s founders say Varda may be the only company that employs hypersonic engineers and pharmaceutical chemists under the same roof.

    At Varda’s headquarters, drug samples are loaded into a spinning arm that creates extra-high G-forces. While the opposite of microgravity, increased weight can provide clues into whether a drug will act differently under new conditions.
    COURTESY VARDA

    Launching industries

    Actual space manufacturing still remains mostly an aspirational project. In 2021, Jeff Bezos, after his first trip aloft in a rocket, suggested that polluting industries should be moved beyond the atmosphere. “We need to take all heavy industry, all polluting industry, and move it into space. And keep Earth as this beautiful gem of a planet that it is,” he told MSNBC.

    Weight is the big obstacle to such dreams. It still costs around $7,000 to launch a single kilogram of payload into orbit, which makes it impractical to, say, send cotton into space to be dyed there, or even to launch the acids and solvents needed to make a semiconductor chip.

    But drugs may be among the few exceptions to this economic rule, since pound for pound, they can be as valuable as rare radioactive isotopes and fine-cut diamonds.

    For instance, just one kilogram of the weight-loss drug Ozempic is worth more than $100 million at retail. (The reason your Ozempic bill is only $1,000 a month is that minute quantities of the active ingredient are present in the shots.)

    That’s why Varda thinks it may eventually be able to manufacture drugs in orbit. However, its effort with United is more of a flying experiment to learn whether the company’s lung medicines will crystallize differently in microgravity.  

    The terms of the deal between Varda and United aren’t public, and the companies haven’t said which specific drugs the collaboration will study. But Rothblatt did confirm that United is paying Varda to help it identify new crystal forms of its drugs (also called polymorphs), which it hopes could have improved properties.

    “One has to do the experiment to find out if that is so. The first part of the experiment is to see what polymorphs of these molecules can be made without the influence of gravity,” she says. “Then, once we have those polymorphs, we will test them.” 

    There is good evidence that crystals form differently in space. For instance, in 2017 the pharmaceutical giant Merck sent samples of its cancer immunotherapy drug Keytruda to the International Space Station, where it was found to form crystals of  a single size. On Earth, the drug tended to form two different sizes at once.

    That experiment offered clues for how to formulate the drug as a shot instead of administering it intravenously. Still, when Merck introduced a Keytruda injection last year, it ended up using a different approach. That means there’s still no straight-line connection between orbital discoveries and any drug here on Earth.  Actual space factories are another step further from reality. 

    “We’ve been learning from space for years, but I can’t name anything manufactured in space, brought down to Earth, and sold,” says Reilly. “So that is a first—or it will be a first.”

    Reilly says that Varda anticipates launching United Therapeutics’ drugs into orbit sometime early next year. 

    ]]>     ASGCT 2026: Victoria Gray Roadshow Returns to Boston https://edusehat.com/en/asgct-2026-victoria-gray-roadshow-returns-to-boston https://edusehat.com/en/asgct-2026-victoria-gray-roadshow-returns-to-boston Wed, 13 May 2026 22:00:11 +0700 Edusehat ASGCT, 2026:, Victoria, Gray, Roadshow, Returns, Boston BOSTON – The annual American Society of Cell and Gene Therapy (ASGCT) conference got underway in Boston this week with a guest appearance by one of gene therapy’s greatest ambassadors and patient advocates.

    Victoria Gray, the sickle cell warrior who was successfully treated in the exa-cel clinical trial sponsored by Vertex Pharmaceuticals/CRISPR Therapeutics seven years ago, spoke in an evening workshop organized by the Emily Whitehead Foundation and ScaleReady.

    Boston is becoming a regular stomping ground for Victoria. Last November, she spoke at the Genetic Agency Technology Conference, hosted by Dyno Therapeutics. Last month, she finally received an invitation to visit the headquarters of Vertex and speak in a town hall meeting.

    In an extemporaneous 20-minute speech, Victoria talked about her lifelong journey with sickle cell disease (SCD). She recalled her first major pain crisis, when she was a young girl—a lightning-type pain that began in one arm before traveling across her chest and down the other arm. “In minutes, my entire body was engulfed in pain,” she said. “The pain felt like getting struck by lightning and hit by a truck. It took me to the floor.” Her grandmother provided hot towels and Tylenol, but nothing worked—not even prayer. After a week in hospital, Victoria returned home but still felt fatigued.

    Stricken by regular pain crises, a hallmark of SCD, Victoria encountered numerous disappointments growing up. Her hematologist said she could not join the cheer team. In eighth grade, she was told she could not join the basketball team, because the exertion would provoke a pain crisis. “As a kid, I was like a Timex: I could take a licking and keep on ticking,” she joked.

    In high school, she signed up to join the United States Navy. “I wanted to serve my country,” Victoria recalled. As she was preparing for basic training, she learned that her disease prevented her from enrolling. “So that was another dream lost.” Next, she turned her attention to nursing. Victoria graduated high school in 2003, but it took another seven years before she could qualify for a nursing program. “Professors didn’t understand because I looked whole and complete. They didn’t think I was sick.”

    In 2010, just before Halloween, Victoria had the worst pain crisis of her life, stripping her ability to walk or use her arms to feed herself. “I couldn’t do anything, facing some of the worst pain of my life. I was getting strong pain medicines like Dilaudid, ketamine, but still couldn’t move. Pain had taken over my thoughts.” Unable to sleep or even take a nap, Victoria was desperate to go home to her family.

    Later, she asked the doctors if they had heard about a haplo-bone marrow transplant (BMT). “I can’t continue living like this,” she said. The doctors looked at each other and said no. After weeks of prayer, Victoria received a call from her hematologist. “Victoria, I have good news, but I only want to tell you in person.” For the first time in her adult life, Victoria was excited about a doctor’s appointment.

    She traveled to Nashville with her brother, who would be her BMT donor, and her husband. She met Haydar Frangoul, MD, whom Victoria calls, “the nicest doctor that I’ve met in my adult life.” Frangoul told her: “Victoria, I wish I had met you ten years ago!’

    Although Victoria’s brother was a suitable BMT match, Victoria was scared of the possibility of graft vs. host disease (GVHD). “My purple pill basket was filled to the brim with medicine every day. If I would acquire [GVHD], that basket would have to triple in size.”

     

    “I’m a human!”

    On her next visit to Nashville, she had to extend her stay because of another pain crisis. But that stay changed her life. Frangoul sat next to her bedside. “Victoria, have you ever heard of CRISPR?” he asked. Victoria shook her head.

    Frangoul used a typo-in-a-textbook analogy and reassured Victoria that there was no chance of GVHD, because she would be receiving her own modified stem cells. “You’ll be the first person to do this, Victoria,” he said. “First human?” she asked. “Yes,” Frangoul said, “but it’s been tested in primates.”

    “But I’m a human!” she said.

    After being reassured that she could still try a bone marrow transplant if the procedure did not work, Victoria agreed to move forward. The chemotherapy, was “hell on Earth,” she recalled. “I lost my hair, which I was prepared for, but the mucositis, the sores in my mouth, the inability to eat for two weeks, was gruesome.”

    Victoria swallowed her tears and decided to fight. This was the first time she had been in the hospital by her choice, to live for her children. About eight months after receiving her CRISPR-edited stem cells in July 2019, she woke up one morning, not feeling anything. “Oh my God, I’m dead,” she thought. She called her kids into the room and hugged them, slowly realizing that “this is what normal feels like.” For the first time in more than 25 years, Victoria did not have any pain in her lower back and hips. She was able to breathe deeply without wincing.

    A few years after her therapy, Victoria was finally able to take her first ever flight, to Washington D.C. to visit her husband, who was on deployment. “It was the first time that I was ever able to show up for the man who has shown up for me,” she said. She has since watched her daughter dance in a Christmas parade and supported her son playing high school football. “The little things have brought me great joy,” she said.

    Her second flight was a business class trip to London with her husband in March 2023, where she spoke at the third International Summit on Human Genome Editing. “I got to keep my covenant that I made with God, that God, if you do this for me, I would tell the world about what you did.”

    Victoria welcomed her first granddaughter on Christmas Eve, 2024. Next week, another milestone: she will be in the audience as her twins graduate high school. And next month, she will publish a children’s book called Hema’s Journey, the tale of her inspiring journey with CRISPR gene therapy. She’s currently training for a group effort to climb Mt. Kilimanjaro.

    Perhaps at next year’s ASGCT conference in Philadelphia, she will be invited to present in a plenary session on the main stage. It would be hard to think of a more fitting speaker.

    The post ASGCT 2026: Victoria Gray Roadshow Returns to Boston appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Patient advocacy drives innovation: James Roe’s asthma story comes to BIO 2026 https://edusehat.com/en/patient-advocacy-drives-innovation-james-roes-asthma-story-comes-to-bio-2026 https://edusehat.com/en/patient-advocacy-drives-innovation-james-roes-asthma-story-comes-to-bio-2026 Wed, 13 May 2026 18:30:04 +0700 Edusehat Patient, advocacy, drives, innovation:, James, Roe’s, asthma, story, comes, BIO, 2026 When BIO President & CEO John F. Crowley met INDY NXT racer James Roe at a Notre Dame game, something clicked. It wasn’t a shared love of football, though both are avid fans of the Fighting Irish. It was a mutual understanding of the patient and caregiver experience, and why it is key to informing and driving innovation in the lab, in the clinic, and even on the track. 

    Roe is a patient. Crowley is a caregiver and industry leader. Both share a commitment to patient advocacy and biotech, which made Roe keen to promote the Biotechnology Innovation Organization (BIO) and speak at the 2026 BIO International Convention in San Diego.

    Roe will step onto the BIO Storytelling Stage on June 24 to share his journey of managing acute asthma while competing at the highest levels of motorsport. Additionally, Roe’s No. 8 Topcon car, carrying the BIO logo across six INDY NXT races from March 28 through June 7, will be on display on the exhibition floor throughout the convention.

    Patient-centered innovation in biotech starts here

    “I was diagnosed with asthma at five years of age and spent weeks and weeks and weeks in hospital as a kid. I also had an uncle pass away from asthma when he was younger,” explained Roe in an interview with Bio.News.

    “I wanted to get some advice on what I could do with my own racing career to advance patient advocacy, because I’m fortunate enough to have made a professional racing driver career here in North America,” he said.

    From this question, an exciting partnership took off.

    The theme of the 2026 BIO International Convention is Driven By Purpose—a reflection of the industry’s unwavering conviction to solve the impossible, to push boundaries, and to achieve better outcomes for patients, public health, and the world. Roe’s story as both a patient and a racer was a perfect fit.

    “James’s story embodies what it means to be ‘Driven by Purpose,’” according to Crowley. “His resilience and commitment to overcoming personal health challenges reflect the same determination we see every day across the biotech community.”

    How Roe turned asthma into advocacy

    “I was diagnosed with type 2 asthma. So for me, the risks to my health were quite specific,” explained Roe. “Early on in my youth, there was always the fear of a sudden asthma attack. And the chances of an attack go up with increased exertion, so as I began my racing career in Ireland, it was something we had to keep an eye on.”

    Asthma, for a long time, was considered a single, albeit complex, disease, but thanks to breakthroughs in research and treatment, it is now understood as a spectrum. Unlike the wheezing symptoms triggered by environmental allergens in less severe forms of asthma, severe type 2 asthma is often genetic and categorized by type 2 inflammation, a systemic allergic response that involves the activation of immune cells, including eosinophils, mast cells, and T-cells, per the Allergy and Asthma Network.

    “Growing up, everything that I did came with the caveat of, Hey, you gotta be careful of this. You gotta be careful of that. You gotta make sure you have your inhaler with you, blah, blah, blah, blah, blah. It was always just a thing,” Roe recalled. Every soccer, rugby, or any other game, he notes, came with extra risk, extra concern, extra caveats. 

    “I just had to make sure that I didn’t let all of those caveats hold me back,” Roe continued.

    Over time, Roe learned to manage his asthma through fitness and medication. As he progressed into motorsports, he adapted that management to high-intensity environments—ones filled with tire and fuel fumes and sustained physical strain, where his heart rate can average more than 130 beats per minute.

    “It’s all a juggling act,” he says.

    As Roe drives forward his career, his history as a patient informs his perspective. With thousands of race fans watching Roe compete, he sees an opportunity to raise awareness—particularly at the BIO International Convention, which brings together more than 20,000 industry leaders each year.

    “Whilst I have a platform and such a fan base in North American motorsport, I want to make sure I am telling the story,” he said. “I want to work with an industry that listens to patients, especially since it reflects my sport, which listens to drivers. It’s about telling my story that asthma didn’t hold me back, and it doesn’t have to hold you back. And if it changes the opinion, or the life, or the perspective, or the outlook on asthma management for one child, that’s enough of a reason to do it.”

    What racing reveals about biotech R&D

    “At the end of the day,” Roe explains, “we’re all in a results-based business.” 

    In racing, that means constant iteration—refining engineering, improving pit crew performance, and analyzing data to gain even fractions of a second. It means “never being satisfied,” per Roe.

    “I think that’s very comparable to the biotech industry because it’s constantly evolving,” Roe continued. “New products are coming out. Companies are spending hours and hours and hours, and engineers are spending hours and hours and hours on R&D. Years of work are spent on certain products and research, all with the purpose of being better and more efficient and addressing scenarios and problem-solving.”

    He has seen that progress firsthand.

    “Thanks to modern-day innovation, technology, and biotech development, no matter what you’re managing in life, it doesn’t have to hold you back,” he says. “There is always a solution. There’s always an answer.”

    Roe is excited to be attending BIO 2026 in San Diego.

    “I’m honored and, quite frankly, humbled that John thought of me as a speaker at the BIO Convention, which is the largest in the world,” he said. And, by Roe’s account, this is only the beginning. 

    “I’m looking to expand my patient advocacy beyond the BIO Convention,” he adds. “This is not a one-and-done thing. I want this to be something that is part of my journey and my legacy, and I’m always looking to grow that piece.”

    The post Patient advocacy drives innovation: James Roe’s asthma story comes to BIO 2026 appeared first on Bio.News.

    ]]>     Gene Therapy ETX101 Improves Seizures and Neurodevelopment in Dravet Syndrome in Phase I/II https://edusehat.com/en/gene-therapy-etx101-improves-seizures-and-neurodevelopment-in-dravet-syndrome-in-phase-iii https://edusehat.com/en/gene-therapy-etx101-improves-seizures-and-neurodevelopment-in-dravet-syndrome-in-phase-iii Wed, 13 May 2026 18:25:09 +0700 Edusehat Gene, Therapy, ETX101, Improves, Seizures, and, Neurodevelopment, Dravet, Syndrome, Phase, III Dravet syndrome has long represented one of the most challenging pediatric epilepsies encountered in neurology and genetic medicine. Caused primarily by loss‑of‑function variants in SCN1A, the disorder emerges in infancy with prolonged febrile seizures and evolves into a lifelong condition marked by treatment‑resistant epilepsy, developmental delay, and significant morbidity. As the gene and cell therapy community gathers for the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, the field’s attention is turning toward approaches capable not only of reducing seizures but also of altering the developmental trajectory that defines the disorder. This year’s Presidential Symposium features new data on ETX101, an investigational gene regulation therapy from Encoded Therapeutics, that appears to move the needle on both fronts.

    Encoded Therapeutics, a clinical‑stage biotechnology company developing precision genetic medicines for severe neurological disorders, has engineered ETX101 as a one‑time AAV9‑based therapy designed to increase expression of SCN1A. Rather than replacing or editing the gene, ETX101 aims to restore physiologic sodium channel function in inhibitory interneurons. The company’s Phase I/II POLARIS program is evaluating the therapy across multiple international sites in children ranging from six months to seven years of age.

    The dataset presented at the ASGCT Presidential Symposium expands the emerging clinical profile of ETX101, incorporating additional patients, early readouts from the highest dose level, and longer‑term follow‑up. Across the cohort, treatment with a single intracerebroventricular dose produced a robust and dose‑dependent antiseizure effect that persisted through 52 weeks of observation. At dose level three, children experienced a median seizure reduction of approximately 76%, a notable finding given that this developmental window is typically associated with escalating seizure burden despite standard therapies. Early data from the top dose level suggest even stronger responses in participants who did not receive sirolimus, consistent with preclinical evidence that the drug can dampen protein expression.

    Beyond seizure control, the therapy appears to influence developmental domains that are rarely improved in Dravet syndrome. Children who reached one year of follow‑up demonstrated measurable gains across communication, motor function, and other adaptive behaviors, as assessed by caregiver‑reported Vineland Adaptive Behavior Scales. Particularly striking were the trajectories of children treated before age two. In this group, cognitive assessments showed early and sustained divergence from the stagnation observed in the ENVISION natural history study, with trajectories more consistent with neurotypical development over the first year after treatment.

    Families and clinicians have taken note of the dual signal emerging from the POLARIS dataset. “Parents of children with Dravet syndrome live with the fear of every seizure and the heartbreak of watching development stall,” said Mary Anne Meskis, CEO of the Dravet Syndrome Foundation. “To see the early and robust seizure reductions paired with meaningful developmental gains is profoundly encouraging. Families have been waiting for therapies that don’t just manage symptoms but give their children a chance to keep learning and growing.”

    Encoded’s chief medical officer, Sal Rico, MD, PhD, underscored the significance of the findings. “Watching these young children not only achieve durable seizure reduction but also show early evidence of neurodevelopmental rescue is truly remarkable,” he said. “These data reinforce our belief that ETX101 has the potential to change the course of the disease and future outlook for the Dravet community.”

    ETX101 has been well tolerated across all four dose levels, with no treatment‑related serious adverse events. Transaminase elevations, a known AAV class effect, were the most common treatment‑related finding; they were asymptomatic and resolved with standard management.

    As the ASGCT community continues to explore the boundaries of genetic medicine, ETX101’s early results highlight the promise of targeted gene regulation as a therapeutic modality. For a disorder like Dravet syndrome, the possibility of addressing both seizures and developmental delay marks an important moment for the field.

    The post Gene Therapy ETX101 Improves Seizures and Neurodevelopment in Dravet Syndrome in Phase I/II appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     10x Genomics, Harvard Target Element’s Multiomics Platform in Patent Lawsuit https://edusehat.com/en/10x-genomics-harvard-target-elements-multiomics-platform-in-patent-lawsuit https://edusehat.com/en/10x-genomics-harvard-target-elements-multiomics-platform-in-patent-lawsuit Wed, 13 May 2026 11:15:09 +0700 Edusehat 10x, Genomics, Harvard, Target, Element’s, Multiomics, Platform, Patent, Lawsuit 10x Genomics and Harvard University have sued Element Biosciences, alleging that Element’s AVITI24™ multiomics platform, Teton chemistry, and related services infringe on four university-owned patents exclusively licensed by the genomics and sequencing tools giant—while Element has denied wrongdoing and promises to fight the lawsuit.

    At issue are four patents—three of which cover technologies related to methods of analyzing the nucleic acids of a cell, awarded between 2021 and 2023, and all titled, “Compositions and methods for analyte detection”: U.S. Patent Nos. 11,021,737 (‘737), 11,566,276 (‘276), 11,566,277 (‘277).

    The fourth patent in question, U.S. Patent No. 12,264,358 (‘358), covers methods of selectively sequencing amplicons in a biological sample, and was awarded in April 2025 under the title, “Method of selectively sequencing amplicons in a biological sample.”

    “Element’s employees, customers, collaborators, and partners have practiced and continue to practice (with Element’s knowledge) one or more claims of the Harvard patents by using the AVITI24 platform and Teton workflow,” 10x and Harvard alleged in a court complaint filed May 7 in U.S. District Court for the District of Delaware. “Through the development and subsequent making, using, selling, offering for sale, and/or importing of the Accused Instrumentalities, Element has and continues to infringe the Harvard Patents.”

    At the deadline, the case, 1:26-cv-00538-UNA, had not been assigned to a U.S. District Judge.

    10x and Harvard have asked the court to find that Element infringed the four patents in question, declare that Element’s infringement “has been willful and deliberate;” declare each Harvard-owned patent to be “valid and enforceable;” issue a permanent injunction barring Element from using technologies based on the patents—otherwise pay 10x and Harvard “an award of post-judgment royalty to compensate for future infringement;” as well as require Element to pay 10x’s and Harvard’s attorneys’ fees and “such other and further relief as the Court may deem just, reasonable, and proper.”

    Element responds

    Element responded to the complaint a day later with a statement denying wrongdoing and promising to fight the lawsuit.

    “Element strongly disagrees with the allegations made by 10x Genomics and believes the claims are without merit,” the firm stated. “Element will vigorously defend its technology and remains focused on delivering differentiated solutions that expand scientific discovery and customer choice. Element was founded to expand innovation and competition in the life sciences industry. We remain confident in our technology, our intellectual property, and the differentiated value our products provide to customers,” the company added. “We will continue to focus on supporting our customers and advancing the next generation of scientific innovation.”

    AVITI24, launched in 2024 as a top-tier successor to its AVITI™ benchtop DNA sequencer and introduced two years earlier as a next-gen sequencing (NGS) platform, aims to enable simultaneous analysis of multiple biological signals—DNA, RNA, proteins, phosphoproteins, and cell structure within single cells—through a 5D multiomics system designed to deliver both spatial, single cell multiomics as well as high-quality, affordable sequencing.

    Earlier this year, Element announced plans to begin offering Direct In Sample Sequencing to tissue—both FFPE and fresh frozen—during the second half of this year through AVITI24. “By contrast, 10x’s Xenium product included fresh frozen and FFPE tissue capabilities at the time it was introduced in 2022,” 10x countered in the complaint.

    Element has applied AVITI24 to offer customers a combined in situ multiomics and next generation sequencing service through its AVITI24 Technology Access Program.

    “Element practices the Harvard patents by using the AVITI24 platform on behalf of its own scientists and researchers and for its AVITI24 TAP customers,” 10x and Harvard alleged in their complaint, adding: “Through the AVITI24 TAP, customers submit samples to Element. Element practices the Harvard Patents by analyzing the samples using the Teton workflow on the AVITI24 platform.”

    Stifling or protecting?

    In its statement, Element also repeated an argument made by several other companies sued by 10x in recent years: “We believe this lawsuit yet again reflects a broader pattern of using the same patent portfolio to stifle innovations across the industry.”

    Countered Michael Schnall-Levin, 10x’s chief technology officer, chief strategy officer, and founding scientist: “We strongly disagree with that characterization. 10x welcomes competition and innovation. “Over the past decade, we’ve invested more than $2 billion in R&D that has transformed how biology is studied. Protecting that work is part of our responsibility as innovators and is what enables us to continue investing in the next generation of technologies.”

    Element is the latest of 10x’s rivals to find itself the target of a patent infringement lawsuit filed by 10x. Last year, 10x settled separate lawsuits with Vizgen and Bruker—the latter acquiring NanoString in 2024 from U.S. Bankruptcy Court after the company sought protection from creditors under Chapter 11 of the U.S. Bankruptcy Code, blaming a $31 million jury award assessed against it in 2023 in a patent infringement case filed by 10x.

    In October, 10x joined with two licensor partners, Prognosys Biosciences and Roche Sequencing Solutions, to file a pair of federal lawsuits accusing sequencing and multiomics giant Illumina of infringing on nine patents related to spatial biology and single-cell sequencing. Earlier last year, Illumina sued Element, accusing its smaller rival of infringing on five of its patents covering flow cell and imaging technologies used in automated genetic sequencing.

    Schnall-Levin said 10x’s latest wave of litigation, starting last year, was not a response to competitive pressures the company has discussed publicly, such as academic customers slowing down spending due to federal budget cuts: “Our litigation is driven by the merits of each case based on clear evidence of others infringing our patents.”

    “We consistently act when others infringe our single cell and spatial patents,” Schnall-Levin added.

    Speaking with GEN last year, Serge Saxonov, PhD, co-founder and CEO of 10x Genomics, defended the company’s approach to protecting its intellectual property: “If others end up infringing and copying our inventions, then we have to protect them. It is incumbent on us, both as a means of serving our mission and as a means of being fair to all our stakeholders.”

    Patents detailed

    In their lawsuit, 10x and Harvard allege that the ‘358 amplicon sequencing patent’s claims cover “concrete, unconventional” methods for sequencing RNA molecules in cell or tissue samples by generating circularized RNA molecules by crosslinking or copolymerizing circular DNA, amplifying the circular DNA to produce amplicons, then using selective sequencing primers to sequence different subsets of the amplicons at a time: “These additional unconventional combinations include sequencing by synthesis (Claim 15) and using at least sixteen different selective sequencing primers (Claim 18).”

    The other three patents, which cover analyte detection, include claims directed to “concrete, unconventional methods that cover:

    • ‘276: multiplex detection of analytes in biological samples by contacting the biological sample with ‘detection reagents.’
    • ‘277: multiplex detection of analytes in cell and tissue samples by binding ‘detection reagents’ to the cell or tissue sample.
    • ‘737: Multiplex detection of analytes in cell and tissue samples by contacting the cell or tissue sample with ‘detection reagents.’”

    10x says the methods of all three patents are practiced by its two spatial biology platforms—the Xenium In Situ single-cell spatial imaging platform, launched in December 2022, and the Atera spatial platform, unveiled last month during the American Association for Cancer Research (AACR) conference.

    Atera, which is designed to enable whole-transcriptome spatial biology at scale, offers more plex, throughput, and sensitivity than Xenium, thus enabling whole-transcriptome at scale. Atera includes an all-in-one instrument for sample imaging, liquid handling, hardware stabilization, and onboard analysis.

    According to 10x, Atera enables 800 whole transcriptome samples per year, a 500 mm2 imageable area per slide, and four slides per instrument run. Users can choose from Atera WTA, which allows for analysis of 18,000 genes, or Atera Select 1,000 custom gene panels with optional stacking of up to two 1,000-gene panels. Compared to Xenium, Atera has four times the throughput, six times higher plex capacity for targeted assays, 3.6x higher plex, and 2–3x sensitivity for whole transcriptome assays.

    10x has said it plans to make Atera available in the second half of this year, though it has begun taking orders for the Atera, which is list-priced at $495,000 and measures roughly 53” x 36” x 64” or (4.42 ft × 3 ft × 5.33 ft).

    Acquired for $350M

    Xenium In Situ was developed by 10x using technology from ReadCoor, a company that 10x acquired in 2020 for $350 million. In 2016, Harvard licensed patents covering the technology exclusively to ReadCoor, a company founded by George M. Church, PhD, of Harvard Medical School.

    Church and Je Hyuk Lee, MD, PhD, founder of Terrain Life Science and an investigator who developed Fluorescent In Situ Sequencing while a research fellow in Church’s lab more than a decade ago, are listed as first and second investors on all four patents.

    The analyte detection patents (‘737, ‘276, and ‘277) also list two other Church lab alumni: Daniel Levner, PhD, co-founder and CTO of Emulate; and Michael Super, PhD, senior director, translational R&D with the Hansjörg Wyss Institute for Biologically Inspired Engineering at Harvard University, as co-inventors.

    The amplicon sequencing patent (‘358) lists as co-inventors two other Church lab alumni: Richard C. Terry, who became ReadCoor’s CEO before his current post as CEO, CTO, and founder of cell therapy manufacturer Harton, and Evan R. Daugharthy, PhD, Harton’s president, CSO, and founder.

    The post 10x Genomics, Harvard Target Element’s Multiomics Platform in Patent Lawsuit appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     State of play: BIO Coffee Chat covers how state policies impact access https://edusehat.com/en/state-of-play-bio-coffee-chat-covers-how-state-policies-impact-access https://edusehat.com/en/state-of-play-bio-coffee-chat-covers-how-state-policies-impact-access Wed, 13 May 2026 04:10:03 +0700 Edusehat State, play:, BIO, Coffee, Chat, covers, how, state, policies, impact, access “We’re finding that the most disruptive pieces of legislation are much more likely to advance in state legislatures first, before eventually gaining traction at the federal government level,” said Karin Hoelzer, Senior Director of Patient Advocacy at BIO as she opened the discussion during the Biotechnology Innovation Organization’s (BIO) April Coffee Chat.

    And indeed, whether it is 340B transparency, price controls, utilization management, vaccine access, or any of the other hot-button issues, many first play out on the state level before they gain steam and trickle up to the federal level.

    And this is why collaboration—across states and between patient advocates and other key stakeholders in the biotech industry—is so important when it comes to navigating state legislatures and their impact on patient access to needed therapies.

    “States ARE the laboratories of democracy—they really are,” explained Melanie Lendnal, Esq., Senior Vice President of Public Policy & Advocacy at The ALS Association.

    So what trends are we seeing across state legislatures, and what can patient advocates do to bring down access barriers and make sure patients get the care they need?

    Advocates are getting creative to protect coverage of life-changing therapies

    Many patient advocates have seen a particularly frustrating pattern in several states: insurers are increasingly coming up with new barriers to not cover life-changing drugs. One particularly egregious example concerns therapies approved under the Food and Drug Administration’s (FDA) Accelerated Approval Pathway.  In the amyotrophic lateral sclerosis (ALS) community, this is proving to have significant real-world consequences according to Lendnal.

    “There is an ALS drug that was approved for a small population of people who have a specific type of genetic ALS. The drug was approved through the Accelerated Approval pathway by FDA,” Lendnal explained. “Although the FDA approved the therapy, some insurance companies have restricted access, calling it ‘experimental’ – which is clearly wrong.”

    What that meant in practical terms, particularly when it came to Medicare Advantage plans, is their policies classified this particular treatment as “experimental” or “investigational” and therefore would not cover it, even though it is FDA approved and Medicare covers it.

    This is particularly infuriating, Lendnal explained, because this treatment doesn’t just slow the progress of patients’ ALS, it is stopping, and in some cases even reversing it—something previously all but unheard of in the neurodegenerative space.

    “In about 60% of cases, people are actually regaining function, which is a massive scientific breakthrough,” said Lendnal. “The fact that people who have health insurance and have this disease, with this genetic mutation, are not able to access it because their policies say it’s ‘experimental’ because it was approved through accelerated approval, is incredibly offensive.”

    So, what can patient advocates like Lendnal do? “We worked to open up this barrier using a combination of tactics,” she said.

    By combining public affairs, direct engagement with payers, media engagement, and working directly with the Centers for Medicare and Medicaid Services (CMS), the ALS Association saw crucial progress. By the end of 2024, advocates were able to convince CMS to issue a directive to all Medicare Advantage plans throughout the country.

    “It told them that, in no uncertain terms, it is unacceptable to state in your policies that you will not cover this drug because you deem it experimental or investigational,” Lendnal said—the first time the CMS ever issued such a directive.

    Advocates have a lot more work to do but now advocates have a helpful case study to guide their action. The ALS Association also published an insurance navigator tool that can be used by anyone trying to deal with appeals.

    Drug pricing legislation threatens patient access in many states

    Despite concerns raised by patient advocates, as well as repeated examples of failures, drug price control models such as Prescription Drug Affordability Boards (PDABs), remain a persistent issue on the state level. This is exacerbated, in part, by federal drug pricing efforts including Most Favored Nation (MFN) type approaches and price setting under programs such as the 2022 Inflation Reduction Act (IRA).

    “We think that PDABs are bad for all patients because they don’t help patients with any cost relief at the pharmacy counter, yet they do create a lot of potential interruptions to access and to supply chains,” explained Brian Warren, VP of State Government Relations at BIO.

    Despite repeated efforts by advocates to mitigate the problems with the Colorado PDAB, the governor and his administration have remained dogged. And this is notable because Colorado’s PDAB is arguably the highest profile in the U.S.

    PDAB legislation in other states remains a complex topic. For example, in Virginia, the legislature has passed a PDAB bill despite the governor raising concerns, and stakeholders are now trying to ask the governor to veto the bill.

    Some of the concern raised by stakeholders are around the effects on Medicaid pricing and reimbursement.

    “There is (proposed) legislation in Louisiana that is designed a little bit differently than what we see in other states in terms of PDABs, but it ultimately maintains the same problems—especially paired with a bill called SB369, which would mandate Medicare pricing across all state sponsored plans,” explained Jen Laws, President and CEO, Community Access National Network (CANN). “Regardless of what anybody says, that includes Medicaid, and that would be detrimental to sustainability and funding of Medicaid.”

    In Illinois, comparatively, they are considering PDAB legislation that includes a carve out that mandates that Medicaid can’t adopt any upper payment limit (UPL) unless Medicaid has done a financial assessment to say that it’s not going to cost them money.

    “That originates from a study done in Oregon,” explained Laws. The study demonstrated that implementing a UPL requires careful consideration of many drug pricing and supply chain complexities and requires additional state resources.  “In essence, when you carve out that UPL, what you’re doing is taking the Medicaid Drug rebate value away from the program, which is the reinvestment opportunity for the Medicaid program.”

    “So it ends up costing you money when you impose a UPL on Medicaid,” he said.

    304B continues to be a challenge, but transparency is slowly improving

    340B transparency is ever top of mind for advocates, Warren notes, and so far 340B legislation has only passed in one state this year, Washington, with the passage of SB5981.

    “It was a really interesting situation,” added Jen Laws, President and CEO, Community Access National Network (CANN). “The governor’s budget did explicitly carve out Medicaid, because what happens when a provider opts for the 340B reimbursement versus the Medicaid reimbursement is that Medicaid loses their own rebate value from it, which increases state budgetary cost and strain on Medicaid, while the provider runs away with the dollars.”

    However, Laws noted, “the legislature there rejected that carve out in pushing for their mandate in terms of expansion. Immediately, the day after, they were sued. So we’ll be seeing how that goes, especially with the turn of the federal government deciding to support manufacturers in limiting 340B’s expansion.”

    The core of CANN’s concern, Law explained, is around how 340B is being leveraged by large hospital systems to deplete value from Medicaid, as well as hinder and increase access barriers for patients, which translates into a lack of charity care or rebates for patients—essentially not passing the intended savings onto the patients who need them most.

    However, 340B transparency has gained traction in many states. In this year’s report to the Legislature, the state of Minnesota found that, “Based on the second year of reported data … Minnesota Covered Entities earned a collective net 340B revenue of at least $1.34 billion for the 2024 calendar year. This net revenue figure is over twice the reported net 340B revenue for the 2023 calendar year—which was $630 million.”

    The report, Warren noted, has been well received by legislators, who recognize that the amount going to hospitals is much higher than many policymakers realize. “It’s not going to the clinics that need this the most,” Warren observed. “It’s going to large hospital systems.”

    In fact, accountability and even basic knowledge of where this revenue is going is practically nonexistent.

    State legislators “don’t want to see health facilities losing revenues and don’t want to see health facilities closing, and certainly neither do we,” said Warren. “But then there’s also a lot of hospitals and other facilities that are basically just using 340B as a blank check.”

    And this blank check mentality has real-world consequences, as Laws explained.

    “There was a massive scandal in Iowa with the AIDS Drug Assistance Program,” he recalled. “For the first time in more than a decade, the state’s going to have to introduce a waiting list for people who need assistance with their AIDS drug access because the contract pharmacy serving the ADAP ran away with $22 million in rebates and then went belly up.”

    Biomarkers are changing the game – but access may be in peril

    Biomarker legislation is going more mainstream this year with a number of states expanding access to biomarker testing, and if the current trend keeps up, this could be very good for patients.

    “Biomarker testing is increasingly important for connecting patients with targeted treatments that are driving huge improvements in cancer survival, but the testing is the key that unlocks access to these new therapies,” said Hilary Gee Goeckner, Director, State & Local Campaigns – Access to Care, American Cancer Society Cancer Action Network. “Unfortunately, there are big disparities in who is benefiting from biomarker testing and biomarker informed care.”

    What Goeckner worries is that, if the system is not updated to fit the new science, then healthcare disparities that we are seeing today will only exacerbate. And this is an important thing to consider in the cancer space because biomarker testing can oftentimes be analogous to giving a patient and their doctor a flashlight in a darkened room—treatment wise.

    “Without access to biomarkers, it means patients may be going through more trial and error,” Goeckner said. “They might be taking treatments that their doctor could have known weren’t going to be effective or not taking a treatment that is most likely to work for their cancer.”

    But to ensure that biomarker testing is available and accessed, a couple things need to happen: insurers need to cover the tests, and patient privacy needs to be maintained to ensure the data cannot later be used “against” a patient, for instance by increasing life insurance premiums.

    “We’ve been leading campaigns at the state level for the last about four years to expand insurance coverage of biomarker testing and tying that to the latest evidence so that the coverage can keep pace with the science,” said Goeckner. To date, 24 states have enacted laws expanding insurance coverage of biomarker testing.

    “One example of the regulatory work we have been doing is genetic privacy when it comes to collecting biomarkers,” said Lendnal. “We don’t want that being a factor when it comes to, for example, life insurance policies. More and more we know where precision medicine is going. We have things like gene therapies and CRISPR, and we want people not to be afraid to get genetic testing because of the impact that might have on life insurance, long term care insurance, disability insurance, etc. So we’ve been working at the state level on this and are cautiously optimistic.” Patient Advocacy organizations can sign on to ACS-CAN’s State Policy Principles to Support Access to Biomarker Testing here.

    But even as biomarker legislation gains traction across the country, advocates are increasingly concerned about the financial and regulatory pressures that could complicate progress—in particular due to an issue called “defrayal.”

    “Defrayal is not a particularly fun topic,” said Goeckner.

    Under the Affordable Care Act, all health insurance plans offered through the ACA marketplace exchange must, at a minimum, cover federally defined essential health benefits (EHBs)—a set of 10 core categories of services typically included in employer-sponsored insurance plans, such as coverage for emergency services, hospitalizations, or maternity and newborn care. States can mandate that insurers cover additional benefits beyond the EHBs in all plans offered on the ACA exchange—but the states may be required to “defray” the added costs.

    According to Goeckner, since the ACA went into effect, states have increased coverage for critical therapies and services, including biomarker testing, without defraying the costs for those benefits. However, a proposed change to federal insurance regulations could change that dynamic.

    “New proposed regulations for insurers came out earlier this year, as part of what is called the notice of benefit and payment parameters (NBPP). The rule proposes many, many concerning changes to private health insurance, including changing what would constitute a mandated benefit beyond EHBs and warrant defrayal. This change is seen as signaling that this administration wants to crack down on states mandating additional benefits and perhaps not making these payments,” Goeckner said.

    For patient advocates, the concern is that this change could discourage states from adopting or maintaining coverage requirements that could be construed as mandates for services like biomarker testing.

    “That is very troubling for patient advocates like us, and we are worried that if that rule is finalized as drafted, it could lead to some states undoing important progress they’ve made at the state level, whether it’s for biomarker testing or other access issues, and certainly be a big deterrent to states making additional progress going forward,” Goeckner said.

    Advocates say that reality makes patient storytelling increasingly important in legislative debates, particularly as states grapple with tightening healthcare budgets and rising Medicaid costs.

    “We really want to keep our work patient-led and highlight stories of how these issues impact real people in order to get lawmakers to care about the issue and really buy in and be willing to allocate money for an issue like defrayal or additional Medicaid costs or additional costs for state employees, because they see that it really matters,” Goeckner added. “We also need to acknowledge that there are challenges and headwinds with state budgets generally. Budgets for Medicaid are a big challenge right now, especially. We need to acknowledge that, but also keep pushing for policies that will make a difference for patients and caregivers.”

    Making fast-paced state advocacy feasible

    It is astounding to see just how much work has already been done on the state level this year  by patient advocates.

    As Warren explained, “It’s about ensuring that innovative therapies continue to make their way to patients, and that patients are able to actually access them and afford them.”

    And to do that, cooperation and focus is key.

    “I would strongly encourage folks to find and join coalitions, especially on issues that are already happening, and get tied into what’s already happening in a particular state or a particular issue that you care about so that you’re not trying to create this out of whole cloth,” said Goeckner.

    “Prioritize what you’re doing,” said Lendnal. “One of the things that we learned early on when it comes to state work is you can stretch yourself so thin by trying to do all of the things, but really be intentional about zeroing in on the things that you want to make sure you do really well.”

    “Ultimately, what we’re doing is state-based community building across issues, education for folks who have time, and trying to build resources for folks who don’t have a lot of time, but maybe still want to make sure that their voice is heard,” concluded Laws. “Those investments into the future are really the way to go. Those investments into being able to touch a legislator out of session is astronomically powerful.”

    The post State of play: BIO Coffee Chat covers how state policies impact access appeared first on Bio.News.

    ]]>     Acute Myeloid Leukemia Therapy Improved by CRISPR Stem Cell Transplant https://edusehat.com/en/acute-myeloid-leukemia-therapy-improved-by-crispr-stem-cell-transplant https://edusehat.com/en/acute-myeloid-leukemia-therapy-improved-by-crispr-stem-cell-transplant Wed, 13 May 2026 04:05:10 +0700 Edusehat Acute, Myeloid, Leukemia, Therapy, Improved, CRISPR, Stem, Cell, Transplant For highly aggressive types of blood cancer, stem cell transplantation is often the only potentially curative therapy. Yet, these cancers can often return even after a transplant. Notably, CAR T cell therapy has not been effective against all blood cancers, including acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

    A recent Phase I/II multicenter clinical trial, led by researchers at Washington University School of Medicine in St. Louis, shows that a stem cell transplant, that removes CD33 from donor cells using CRISPR, can help prevent cancer recurrence.

    The work was published in Nature Medicine and titled, “CRISPR−Cas9 CD33-deleted allogeneic hematopoietic cell transplantation with gemtuzumab ozogamicin maintenance in AML: a Phase I/II trial.” The study was conducted at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine, and 14 other sites in the U.S. and Canada. 30 adult patients with AML or MDS at high risk of relapse received the stem cell transplant.

    Myeloid cancers, such as AML and MDS, are difficult to treat with CAR T cells because the same proteins targets are present on both cancer cells and healthy myeloid cells, leading to toxicity risks.

    “We are encouraged by the results of this study showing that a CD33-deleted stem cell transplant looks very similar to the outcomes of standard stem cell transplantation,” said John DiPersio, MD, PhD, professor of medicine at WashU Medicine and corresponding author of the study. “In the future, we are hopeful we will be able to combine this with CD33-targeted immunotherapies, such as CAR T cells, and improve treatment options for patients with these very aggressive blood cancers.”

    As proof of concept, patients also received a maintenance therapy that targets CD33, after completion of the stem cell transplant. While not a CD33-targeted CAR T cell, the maintenance therapy, called gemtuzumab ozogamicin, is an engineered antibody that targets CD33 and carries an anti-cancer drug. Gemtuzumab ozogamicin is approved by the Food and Drug Administration (FDA) to treat CD33-positive AML and is in clinical trials for CD33-positive MDS. While it helps prevent relapse, the drug’s use is limited because it can cause liver toxicity and damage to blood cells, including dangerously low counts of white blood cells, red blood cells, and platelets.

    All patients from the trial achieved engraftment of their transplanted stem cells by day 28. Some patients met this goal sooner with platelet production returning by day 16 on average. These timeframes are comparable to those of standard transplanted stem cells.

    Average survival was just over 14 months. Nineteen patients received at least one cycle of the antibody maintenance therapy as part of a dose-escalation protocol. The authors found that patients maintained blood cell counts across all doses, suggesting that the gene-edited stem cell transplant protected patients from low blood cell counts typically seen following a standard stem cell transplant.

    DiPersio and colleagues published a single case study detailing a patient with high-risk AML who received a CD33-deleted stem cell transplant. Upon relapse after the transplant, the patient received a CD33-targeted CAR T cell therapy, which used T cells from the same donor who provided the stem cell transplant.

    The treatment resulted in complete remission and the patient remains cancer free over one year after receiving the CAR T cell therapy. Normal blood cell production returned with all blood cells lacking CD33, providing evidence that the genetically engineered donor cells had established themselves in the bone marrow.

    DiPersio said the results of the study lay the groundwork for developing paired CD33-deleted stem cell transplant and CD33-targeted immunotherapy interventions that avoid destruction of healthy donor cells in the course of cancer treatment.

    The post Acute Myeloid Leukemia Therapy Improved by CRISPR Stem Cell Transplant appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Brain Histamine Map Links Genetic Factors to Mental Health and Psychiatric Disorders https://edusehat.com/en/brain-histamine-map-links-genetic-factors-to-mental-health-and-psychiatric-disorders https://edusehat.com/en/brain-histamine-map-links-genetic-factors-to-mental-health-and-psychiatric-disorders Wed, 13 May 2026 04:05:09 +0700 Edusehat Brain, Histamine, Map, Links, Genetic, Factors, Mental, Health, and, Psychiatric, Disorders A study headed by researchers at King’s College London and the University of Porto has mapped the histamine system in the brain. Histamine, a molecule more commonly associated with allergies, plays a separate but poorly understood role in brain function. The new study addresses this gap, building the first multiscale map of the histamine system which spans from genetics to behavior and related mental health conditions.

    The findings provide a new framework for understanding how this often-overlooked chemical system contributes to brain function and could point towards new treatment strategies for histamine-related conditions such as depression, ADHD, and schizophrenia. The study was funded by the National institute for Health and Care Research (NIHR) Maudsley Biomedical Research Centre.

    Daniel Martins, MD, PhD, visiting senior research fellow at the Institute of Psychiatry, Psychology & Neuroscience (IoPPN) King’s College London, said, “This work provides a crucial foundation for future research. By integrating molecular biology, brain imaging, and computational analysis, it offers a new perspective on how neurotransmitter systems are organized across the human brain. As neuroscience moves toward more integrated and personalized models of mental health, understanding systems like histamine may prove essential for unlocking new approaches to diagnosis and treatment.”

    Martins is first and corresponding author of the team’s published paper in Nature Mental Health, which is titled “Mapping histamine pathway networks in the human brain across cognition and psychiatric disorders.” In their paper the team concluded, “This study provides an integrated characterization of the histaminergic system in the human brain, leveraging transcriptomic, neuroimaging, and functional datasets to delineate its molecular organization and relevance to brain function underlying cognition and psychiatric disorders.”

    Histamine is a neurotransmitter, a molecule crucial for neurons to communicate with one another, the authors explained. “Neuronal histamine plays a crucial role in the regulation of brain function, serving as a neuromodulator with widespread influence across multiple neurotransmitter systems.” However, neuroscience research has classically focused on understanding other neurotransmitter systems such as dopamine and serotonin.

    As the investigators noted, the organization of histamine in the human brain remains incompletely characterized. However, they explained, dysregulation of the histaminergic system has been implicated in a number of neuropsychiatric conditions, including anxiety, depression, schizophrenia, and autism spectrum disorder (ASD), as well as neurodegenerative diseases including Alzheimer’s, Parkinson’s, and Huntington’s diseases. “Therefore, targeting the brain histamine system has garnered significant attention as a potential new therapeutic strategy for treating these disorders, with pharmacological interventions aimed at modulating histamine receptor activity showing promise in preclinical models.”

    Histamine acts through four known histamine receptors, which are responsible for how the signal will influence receiver neurons. Each of these histamine receptors, (histamine receptor H1 (encoded by HRH1), H2 (HRH2), H3  (HRH3) and H4 (HRH4)), mediates distinct functions. For their newly reported study, Martins and colleagues carried out what they described as multimodal analysis, integrating transcriptomic, neuroimaging, developmental and functional datasets to map the architecture of the histaminergic system.

    To build a comprehensive map of how histamine acts in the brain, researchers first combined genetic and molecular data with physical maps of the brain.

    This revealed which brain regions receive more input from the brain’s histamine system, and which parts show greater capacity to respond to histamine. These molecular data were then linked with positron emission tomography imaging of histamine receptors in living individuals, as well as functional neuroimaging databases that map brain regions to specific cognitive processes and mental health conditions. This type of scan shows how different parts of the brain are working by tracking a tiny amount of radioactive tracer in real time.

    Their results found that different histamine receptors were found on brain cells that either turn activity up (excitation) or turn it down (inhibition). “The findings reveal that histaminergic genes exhibit distinct cellular and regional expression profiles, closely aligning with known histaminergic neuroanatomy and function,” they wrote. “At the single-cell level, histamine receptor H1 and histamine receptor H2 were enriched in excitatory neurons, whereas histamine receptor H3 showed preferential expression in inhibitory populations.” This suggests histamine may be important in maintaining the balance between excitation and inhibition, a fundamental property of healthy brain function.

    Brain regions with higher histamine-related gene expression were consistently associated with processes such as emotional regulation, stress and fear responses, decision-making, impulsivity, reward, sleep, and memory.

    The parts of the brain where histamine-related genes were most active also overlapped significantly with brain regions known to be affected in several psychiatric conditions, including attention-deficit/hyperactivity disorder, major depressive disorder, schizophrenia, and anorexia nervosa. This is in keeping with previous hypotheses linking histamine to these disorders. “By linking histaminergic gene expression to brain-cell types, neurotransmitter systems, cognitive domains and psychiatric disorders, these correlational findings generate several hypotheses concerning histamine’s critical role in brain organization, neurodevelopment and mental health, which further experimental mechanistic work should prioritize and build onto investigate causal relationships,” the investigators concluded.

    Martins said, “Current psychiatric treatments largely target neurotransmitters such as serotonin and dopamine, yet histamine interacts closely with these systems and influences their activity. By providing a detailed map of histamine-related pathways, this work suggests new opportunities for developing treatments that target this system more directly, particularly for symptoms such as cognitive dysfunction, fatigue, and impaired motivation.

    While these findings do not establish a direct causal role, they suggest that histamine signalling may contribute to regional vulnerability in these disorders. This aligns with a growing view in psychiatry that mental health conditions arise from disruptions across interacting brain systems rather than a single chemical imbalance.”

    This new map paints a neural picture of a previously lesser-studied molecule. It opens up future avenues of research into exactly what histamine is doing in various cell types and parts of the brain.

    “We want to emphasise that these findings are hypothesis-generating and based on large-scale datasets that capture patterns rather than direct mechanisms,” commented senior author Steve Williams, PhD, professor of neuroimaging at IoPPN King’s College London. Future studies will focus on testing how histamine signaling changes in living individuals, for example through pharmacological interventions or longitudinal imaging approaches.

    Co-author Daniel Van Wamelen, PhD, clinical senior lecturer in neuroscience at IoPPN, King’s College London and one of the authors on the paper said: “This kind of work is already taking place at King’s College London, for example in the iMarkHD project. In this project we use Positron Emission Tomography scans to study a specific histamine receptor (called H3) in people with Huntington’s disease, an inherited condition that affects the brain. The goal is to see how histamine activity changes in different parts of the brain over time, and how these changes relate to symptoms such as apathy, depression, and anxiety.”

    The post Brain Histamine Map Links Genetic Factors to Mental Health and Psychiatric Disorders appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AAVs in Focus: Practical Approaches to Capsid Analytics and Plasmid DNA Control https://edusehat.com/en/aavs-in-focus-practical-approaches-to-capsid-analytics-and-plasmid-dna-control https://edusehat.com/en/aavs-in-focus-practical-approaches-to-capsid-analytics-and-plasmid-dna-control Wed, 13 May 2026 04:05:08 +0700 Edusehat AAVs, Focus:, Practical, Approaches, Capsid, Analytics, and, Plasmid, DNA, Control

    REGISTER NOW

    Adeno-associated viruses (AAVs) continue to be a foundational platform for gene therapies, with rapid advances in vector design, analytics, and manufacturing practices. This three-part USP webinar series on AAV guides participants through a progressive learning journey starting with the current AAV landscape, then advancing to focused, practical discussions on capsid characterization, and ending with specialized session focused plasmid DNA starting materials.

    Throughout this series, experts from USP and industry will share first-hand experiences, case studies, and practical insights on how AAV analytics and material controls are evolving. This series will identify common AAV challenges and explore effective solutions across different stages of development.

    Why Attend the AAV Webinar Series?

    • Broaden attendees’ understanding of the current AAV landscape by learning from experts about key scientific, analytical, and manufacturing considerations

    • Learn how developers approach capsid characterization, full/empty analysis, and data comparability, and how production and analytical teams work together to optimize AAV manufacturing

    • Discover more about the critical role of plasmid DNA as a starting material, and how expectations change from early development to later stages

    • Engage directly with subject matter experts during live Q&A sessions

    May 19, 2026

    June 16, 2026

    July 8, 2026

    AAV Manufacturing: Best Practices in Quality Control and the Role of USP Standards

    Practical Strategies for AAV Capsid Characterization

    Characterizing Plasmid DNA to Improve AAV Manufacturing 

    A live Q&A session will follow the presentation, offering you a chance to pose questions to our expert panelists. 

    Produced with support from:

    usp logo

    The post AAVs in Focus: Practical Approaches to Capsid Analytics and Plasmid DNA Control appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cholesterol Drug May Weaken Ovarian Cancer’s Metastatic Defense https://edusehat.com/en/cholesterol-drug-may-weaken-ovarian-cancers-metastatic-defense https://edusehat.com/en/cholesterol-drug-may-weaken-ovarian-cancers-metastatic-defense Tue, 12 May 2026 10:10:11 +0700 Edusehat Cholesterol, Drug, May, Weaken, Ovarian, Cancer’s, Metastatic, Defense The accumulation of fluid in the belly, known as ascites, is something that women with advanced ovarian cancer may know all too well. The results of research by a team at Duke University School of Medicine now suggest that, more than causing discomfort, this fluid may protect cancer cells from a form of cell death known as ferroptosis, helping cancer cells survive and spread. The studies also indicated that a decades-old cholesterol-lowering drug, bezafibrate, may be able to disrupt that protection.

    The findings, derived through lab experiments and an analysis of patient samples, do not show that bezafibrate treats ovarian cancer. But they do suggest that changing the environment that cancer depends on could make it more vulnerable to existing cancer treatment.

    “Doctors have mostly viewed ascites as a symptom rather than an active driver of disease,” said Jen-Tsan Chi, PhD, a professor in the department of molecular genetics and microbiology and co-leader of the Cancer Biology Program at the Duke Cancer Institute. “We’ve learned it gives cancer a survival advantage, which fills a major gap in understanding how ovarian cancer spreads.”

    Chi is senior and corresponding author of the team’s published paper in Nature Communications, titled “Ascites protects against ferroptosis and enables the peritoneal growth of ovarian cancer.” In their paper, the authors concluded, “Our findings identify ascites as a key determinant of ferroptosis resistance in metastatic OVCA and highlight its role in promoting tumor survival and dissemination within the peritoneal cavity.”

    The peritoneum is a frequent site of metastasis in ovarian cancer (OVCA), the authors explained, and this is often accompanied by the accumulation of ascites in the peritoneal cavity. And while ascites is observed in other diseases such as liver cirrhosis, it’s most often associated with metastatic OVCA. The fluid occurs in 90% of those with advanced ovarian cancer. Doctors will drain ascites to ease pain, improve mobility, and make breathing easier, which offers patients relief even if it doesn’t stop the disease.

    “Due to the enrichment of cellular and acellular factors, the ascitic fluid is reported to harbor a growth-promoting and immune-evading environment for cancer cells and is thought to serve as a medium for cancer cell dissemination and tumor progression and metastasis,” the team continued. However, they noted, “Despite its prevalence, ascites and its role in the peritoneal growth of OVCA remain poorly understood.”

    According to the newly reported study findings, ascites also acts as a shield, helping cancer cells evade a specific form of cell death called ferroptosis. Ferroptosis is a kind of cellular rusting. It happens when iron inside a cell reacts with certain fats, causing the cell membrane to break apart. Many metastatic cancer cells—those that float freely through the abdomen looking for new places to grow—are naturally vulnerable to this kind of damage. “… we and other groups have reported that detached and metastasizing OVCA cells are especially vulnerable to ferroptosis, a form of cell death characterized by iron dependency and an irreversible accumulation of lipid hydroperoxides,” the authors wrote.

    The study in Nature Communications shows how they survive anyway. For their research, the scientists bathed cancer cell lines and patient-derived tumor cells in ascites collected from patients, and observed how they responded to ferroptosis triggers. “Nothing is currently known about how ascites may influence OVCA cells’ ferroptosis,” they noted. “Given the common occurrence of ascites with peritoneal metastasis, ascites may be crucial for the peritoneal spread of OVCA.”

    From left, Duke University School of Medicine researchers Susan K. Murphy; Andrew Berchuck, MD; Yasaman Setayeshpour, PhD, and Jen-Tsan Ashley Chi, PhD, are studying how a common class of cholesterol drugs, called fibrates, can strip away a key defense used by ovarian cancer cells, making them more vulnerable to treatment. [Duke University School of Medicine/Mark Dolejs]
    (From Left) Susan Murphy, Andrew Berchuck, Yasaman Setayeshpour, and Jen-Tsan Chi pose in the lab. Research accepted to Nature Communications led by Chi, professor in MGM, integrative immunology, and medicine, found that a cholesterol drug may make it harder for ovarian cancer cells to survive. In lab studies, fibrates weakened the protective effect of abdominal fluid that helps tumors resist a type of cell death, known as ferroptosis. Chi is also a professor of biomed engineering, cell biology and pharmacology and cancer biology and a member of DCI. [Mark Dolejs for Duke University School of Medicine]
    The team found that the fluid protected cancer cells by changing how the cells store fats and control iron levels, effectively blocking cell death. The protection required only trace amounts. As little as 2% immersion shielded cancer cells from destruction, even though in patients these cells are entirely enveloped by the fluid.

    “What surprised us was how selective this effect was,” said first author Yasaman Setayeshpour, a graduate student in molecular genetics and microbiology at Duke School of Medicine. “Ascites didn’t protect the cancer cells from other well-known types of cell death, like apoptosis or necrosis—it only blocked ferroptosis.

    “To figure out why, we broke ascites down into major parts, like lipids, proteins, and small molecules, and tested what happened when each was removed. When we took the lipids out, the protective effect disappeared. That told us lipids are the key reason ascites helps these cancer cells survive,” Setayeshpour said.

    The researchers also found an unexpected helper in the form of bezafibrate, an older type of cholesterol-lowering drug that is used to lower triglycerides by altering how the body processes fats. “The idea behind testing lipid-lowering drugs was to mimic what happens when lipids are removed from ascites,” Setayeshpour explained.

    The studies showed that bezafibrate restored sensitivity to ferroptosis, but only when ascites was present. On its own, the drug did not trigger cell death, nor did it slow tumor growth in mice. The researchers found that the drug’s impact hinged on the cancer’s surroundings, in this case, the fat-rich fluid bathing the tumor. The studies showed that targeting this environment, using repurposed drugs like bezafibrate, could leave cancer cells more exposed to existing cancer treatments. “Given the intrinsic vulnerability of metastatic cancer cells to ferroptosis, these data suggest that ascites-mediated protection represents a critical mechanism supporting peritoneal survival,” the team noted. “Importantly, re-sensitization to ferroptosis by bezafibrate raises the possibility that therapeutic targeting of this pathway may limit peritoneal dissemination.”

    Chi said the finding could have implications beyond ovarian cancer. Other cancers, including colorectal and pancreatic cancers, can also spread within the abdominal cavity. “These findings may also extend beyond ovarian cancer to other metastatic settings, including peritoneal colorectal cancer and pleural, brain, or spinal metastases,” the authors stated.

    “This work shows how much the environment around a tumor matters,” Chi said. “Biological fluids like ascites don’t just give cancer cells a place to move. They actively help drive how cancer spreads.”

    The post Cholesterol Drug May Weaken Ovarian Cancer’s Metastatic Defense appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Stable Producer Cell Line Generation Platform Adds to VIVEbiotech’s Lentiviral Vector Manufacturing Capabilities https://edusehat.com/en/stable-producer-cell-line-generation-platform-adds-to-vivebiotechs-lentiviral-vector-manufacturing-capabilities https://edusehat.com/en/stable-producer-cell-line-generation-platform-adds-to-vivebiotechs-lentiviral-vector-manufacturing-capabilities Tue, 12 May 2026 10:10:10 +0700 Edusehat Stable, Producer, Cell, Line, Generation, Platform, Adds, VIVEbiotech’s, Lentiviral, Vector, Manufacturing, Capabilities VIVEbiotech, a Spanish lentiviral vector (LVV) CDMO, has added EvoLVcell to its catalog of LVV-manufacturing products. EvoLVcell is a stable producer cell line (SCL) generation platform designed to reduce batch‑to‑batch variability, improve vector quality, and provide a more predictable long‑term production strategy for LVVs. The fully integrated platform requires only the addition of a developer’s transgenic element to the fully characterized monoclonal PCL, avoiding transfection, explained a VIVEbiotech spokesperson, who added that this simplifies the manufacturing workflow, improving long-term predictability and reducing reliance on plasmid and transfection reagent supply chains.

    The characterized, inducible, monoclonal lentiviral packaging cell line (PCL) derisks SCL generation by incorporating the therapeutic transgene that typically would be transfected together with the helper plasmids, noted Andrés Lamsfus-Calle, PhD, product development manager at VIVEbiotech. This SCL platform generation enables VIVEbiotech to get a prototype cell line in three months, pointed out Lamsfus-Calle.

    EvoLVcell reduces variability and produces fewer impurities, of particular importance to gene therapeutics, according to a company statement. “At the same time, the platform’s extensive characterization enables rapid yield assessment through a rapid proof-of-concept, allowing developers to reach a clear go/no-go inflection point in just three months,” continued the statement.

    “Achieving cost efficiency and high reproducibility is essential for the scalable production of lentiviral vectors and for expanding patient access. Stable producer cell lines provide a reliable and scalable manufacturing platform to support this goal,” said Lamsfus-Calle.

    The post Stable Producer Cell Line Generation Platform Adds to VIVEbiotech’s Lentiviral Vector Manufacturing Capabilities appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Genewiz Launches Gene Synthesis 2.0 https://edusehat.com/en/genewiz-launches-gene-synthesis-20 https://edusehat.com/en/genewiz-launches-gene-synthesis-20 Tue, 12 May 2026 06:35:11 +0700 Edusehat Genewiz, Launches, Gene, Synthesis, 2.0 Genewiz, Azenta Life Sciences’ genomics services provider, launched Gene Synthesis 2.0, an updated platform designed to simplify how researchers design and order genes.

    Built around three packages, Sprint, Flex, and Flex+, Gene Synthesis 2.0 introduces a streamlined, intuitive ordering experience, according to Trey Martin, president of Genewiz, who adds that researchers can design cloning strategies, select vectors, assess sequence complexity, and place orders in minutes.

    The product also introduces an upgraded codon optimization capability that combines established heuristic approaches with machine‑learning models trained on a high‑expression dataset, continues Martin. This approach can improve expression reliability across a wide range of organisms and sequence architectures while maintaining customer control over design choices, he points out.

    “Gene Synthesis 2.0 reflects how researchers work today—where speed, clarity, and reliability matter at every step,” says Martin, “By simplifying the path from sequence design to execution, this offering helps scientists move faster in rapidly evolving fields such as antibody discovery, AI-enabled biology, and gene and cell therapy.”

    Gene Synthesis 2.0 is being highlighted at ASGCT this week at booth 915.

     

    The post Genewiz Launches Gene Synthesis 2.0 appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     How HIV-1 Develops Resistance to Broadly Neutralizing Antibodies https://edusehat.com/en/how-hiv-1-develops-resistance-to-broadly-neutralizing-antibodies https://edusehat.com/en/how-hiv-1-develops-resistance-to-broadly-neutralizing-antibodies Tue, 12 May 2026 06:35:10 +0700 Edusehat How, HIV-1, Develops, Resistance, Broadly, Neutralizing, Antibodies

    One of the most challenging aspects of combatting HIV-1 infection is that the virus continually evades neutralizing antibodies. However, one consequence of this is that a small percentage of people with HIV-1 (1-5%) develop rare, broadly neutralizing antibodies (bNAbs) that can neutralize a large fraction of global HIV-1 isolates. These broadly neutralizing antibodies are among the most promising new long-acting HIV treatments, offering the potential to forego traditional daily dose of antiretroviral drugs. Indeed, a recent trial found that participants who received a single dose of two bNAbs maintained a nearly undetectable viral load for up to 20 weeks, and a third did so for about a year.

    Despite the known promise of bNAbs, the pathways through which the virus escapes these antibodies remain incompletely understood across diverse HIV-1 strains.

    “Knowing how different strains of the virus respond to leading bNAb therapies will greatly improve our ability to anticipate whether a particular therapy will be effective for individual patients,” says Paul Bieniasz, PhD, professor at The Rockefeller University and an HHMI Investigator. “And if we can identify broadly neutralizing antibodies that the majority of strains have great difficulty escaping from, we can create more robust treatments.”

    Now scientists have established the most comprehensive view to date of how HIV-1 can escape bNAbs. Using thousands of parallel viral selection experiments combined with bioinformatic analysis and experimental validation, the team discovered viral mutations that make HIV-1 strains resistant to two bNAbs: 3BNC117 and 10-1074.

    This work is published in Nature Microbiology in the paper, “Diverse paths to broadly neutralizing antibody escape among HIV-1 strains.“

    The researchers sought to investigate the relationship between different HIV-1 strains and bNAbs collected from HIV infected persons. Only a handful of resistance mutations have been identified in a limited number of viral strains. The researchers wanted to expand that number to represent global viral diversity.

    “No one has attempted to do this at such a scale before,” said Theodora Hatziioannou, PhD, research professor at The Rockefeller University.

    The team developed an approach that would allow them to study the mutational pathways to escape among 15 strains of HIV-1 sourced from around the globe. The goal was to pinpoint the mutations that were contributing to each strain’s propensity to develop resistance.

    “We found that most viral strains can escape bNAb neutralization, but there’s substantial variation in the likelihood that they will and the mechanisms that enable it,” says Alex Stabell, MD, PhD, an infectious disease physician and clinical scholar at The Rockefeller.

    Stabell devised a pipeline that began by growing large amounts of virus in cell culture. The bulk populations were used to seed thousands of parallel selection experiments with varying concentrations of bNAbs. Viruses that were able to spread in the presence of the bNAbs were isolated and sequenced. Custom bioinformatic processing gave a list of putative resistance mutations, which were subsequently experimentally validated for each viral strain.

    Using this method, called RISC (resistance identification via selection and cloning), the team found more than 100 bNAb escape mutations across the 15 viral strains tested, dramatically expanding the known number. Surprisingly, they found that in most cases, a single amino acid change may be enough to confer resistance. That turned out to be true for 12 of the 15 viruses tested against the 3BNC117 antibody and for all nine tested against 10-1074.

    “It was striking that it’s actually quite easy for most HIV strains to escape these special antibodies,” Bienasz says. “But it’s not true for all strains—a handful Alex worked with needed multiple amino acid substitutions or unusual ways to replicate in order to escape.”

    “The genetic barrier to resistance was higher for these viruses,” Stabell adds. “One of the goals of therapy these days is not simply to have therapies that are transiently effective, but to have this high genetic barrier.”

    They also identified a surprising number of mutations occurring outside the epitope on the viral envelope recognized by bNabs that target the CD4 binding site, such as 3BNC117. (10-1074 aims for a more mutable envelope target, which may help explain why it’s easier to escape.) “These were quite prominent and unexpected,” says Hatziioannou. “No one would have predicted these would affect bNAb sensitivity.”

    In the future, the team will use Stabell’s method to identify to discover resistance mutations to other bNAbs as well as to combinations of them.

    “HIV-1 mutates so fast and the diversity in the population is already quite enormous, so we’ve long known that a multidrug approach is the best course of treatment,” Hatziioannou says. “We hope to identify combinations that potentially raise the genetic barrier to resistance and are therefore more effective.”

    The post How HIV-1 Develops Resistance to Broadly Neutralizing Antibodies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     RegVelo AI Model Predicts Cell Fate, Tackles Developmental Disorders and Cancer https://edusehat.com/en/regvelo-ai-model-predicts-cell-fate-tackles-developmental-disorders-and-cancer https://edusehat.com/en/regvelo-ai-model-predicts-cell-fate-tackles-developmental-disorders-and-cancer Tue, 12 May 2026 03:00:14 +0700 Edusehat RegVelo, Model, Predicts, Cell, Fate, Tackles, Developmental, Disorders, and, Cancer In a new study published in Cell titled, “RegVelo: gene-regulatory-informed dynamics of single cells,” researchers from Stowers Institute of Medical Research have developed a new AI model that connects two areas of single-cell biology that have often remained separate: estimating how cells change over time and inferring the gene regulatory networks controlling those changes.  

    “You can imagine if you had a very early set of cells, having a particular set of instructions could allow you to reproduce, in vitro, some of these cell types in a very natural way. These cells could then be used in cell therapies in regenerative medicine,” said Tatjana Sauka-Spengler, PhD, Stowers Institute Investigator and co-senior author of the study.  

    While development is often described as a series of static snapshots of cell states, RegVelo models how these fate decisions are encoded in gene regulatory networks over time and space, and what drives cell state transitions. In zebrafish neural crest development, RegVelo identified an early driver of pigment cell formation (tfec) and revealed a previously unknown regulator of pigment cell fate (elf1). The neural crest is a developmental system that gives rise to many different cell types, including pigment cells, craniofacial tissues, and parts of the peripheral nervous system. 

    CRISPR/Cas9-mediated knockout and single-cell Perturb-seq supported predictions, showing that the model could do more than describe developmental changes and generate biologically meaningful hypotheses that held up in living systems. 

    Alejandro Sánchez Alvarado, PhD, Stowers President and chief scientific officer says RegVelo’s value “extends well beyond” neural crest cells and is applicable to any system in which cells change over time, from basic developmental biology to modeling tumor trajectories and the cellular outcomes that may inform treatment. 

    “Sauka-Spengler and her collaborators have developed a meaningfully different way to process this kind of data,” said Sánchez Alvarado. “It allows us to infer the most likely path of each component through space and time, and to use deep learning to predict those dynamics and test them experimentally.” 

    Single-cell biology research has made it possible to build increasingly detailed maps of development. RNA velocity methods can help researchers estimate how cells move through developmental landscapes, while gene regulatory network approaches can identify relationships among genes. However, these methods have typically been used in parallel rather than together.  

    “For a long time, cellular dynamics and gene regulation have largely been modeled separately,” said Fabian Theis, PhD, the study’s co-senior author and director of the institute of computational biology at Helmholtz Munich. “RegVelo brings those pieces together, allowing us to ask not only how cells are changing, but which regulatory interactions are helping drive those changes.”  

    The framework jointly models splicing kinetics and gene regulatory relationships, allowing researchers to map the hidden timeline of cell development, predict how cells shift from one state to another, and test what might happen when specific regulators are perturbed. 

    The framework can incorporate additional regulatory layers, including chromatin, protein activity, and other multimodal measurements. While the study’s limitations include simplifying assumptions around latent time, regulatory interactions, and computational cost, the results demonstrate a compelling proof of principle.

    “When dynamic cell-state modeling is linked directly to gene regulation, it becomes possible to move closer to mechanism and then discovery,” Sauka-Spengler said. 

    The post RegVelo AI Model Predicts Cell Fate, Tackles Developmental Disorders and Cancer appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Experts explain how to drive business development with BIO 2026 https://edusehat.com/en/experts-explain-how-to-drive-business-development-with-bio-2026 https://edusehat.com/en/experts-explain-how-to-drive-business-development-with-bio-2026 Mon, 11 May 2026 19:45:05 +0700 Edusehat Experts, explain, how, drive, business, development, with, BIO, 2026 The BIO International Convention, the world’s largest annual gathering of biotechnology stakeholders, provides an exceptional opportunity to make the connections that enable development of biotech innovations.

    In discussing plans for BIO 2026, running June 22-25 in San Diego, prospective licensees have said they hope to find companies with good data, while companies spoke of seeking investors who share their vision. Both groups said any partnership requires a strategic fit.

    Veterans of the BIO International Convention explained how they use the event to drive business development in a May 6 online webinar, part of a series of webinars on business development presented by the Biotechnology Innovation Organization (BIO), which organizes the convention.

    “BIO 2026 does not start in June. It starts long before, because it takes time to prepare, assess, build your presentations, etc.” said Carlos Velez, managing partner at Lacerta Bio and the panel’s moderator.

    Panelists said a big part of preparation involves engaging with the BIO Partnering™ platform, a bespoke system allowing partners with matching needs to find one another and schedule meetings before BIO 2026 begins. Panelists praised the platform’s real-time communication and scheduling functions.

    Mackensie Vernetti, SVP of Partnering at BIO, informed the webinar audience of a newly added feature allowing participants to create a direct link to their company’s platform space, so they can share via social media or email. BIO Partnering™ platform participants who click on the link go straight to a form to request a meeting. (See more on this and other resources here.)

    Why go to BIO 2026?

    For companies, business development at BIO 2026 obviously involves finding investors, but it’s also about finding partners with similar research interests, said Anjan “AJ” Aralihalli, CEO of Raya Therapeutic, Inc., which develops drugs for ALS and other neurodegenerative diseases.

    “My goals at BIO 2026 are going to be primarily to look for and talk to investors—as well as strategic partners who are interested in the neurodegenerative neuromuscular disease space,” he said.

    Christian Rohlff, CEO and Founder of Oxford BioTherapeutics, said the BIO International Convention is an opportunity to touch base with current partners and find new ones.

    The situation is similar for those seeking to invest in small biotechs, according to Jennifer Halbleib, U.S. Partnering Transactions Head, Roche and Genentech Corporate Business Development. She said her BIO 2026 goals include “identifying new opportunities for folks we haven’t previously engaged with, as well as hearing updates from companies that we’ve been chatting with for a while.”

    Jennifer O’Cain, Catalyst Pharmaceuticals’ VP of Business Development – Search, Evaluation & Scientific Competitive Intelligence, said her firm focuses on rare diseases and uses “a buy-and-build” model, so she’ll be seeking investments.

    “My activity at BIO 2026 is going to be back-to-back meetings focused on somewhat mature opportunities, something like post-proof-of-concept with a regulatory pathway,” she said.

    Finding partners with a ‘strategic fit’

    Roche and Genentech’s Business Development team receives about 1,000 meeting requests for a typical BIO International Convention, according to Halbleib. She said the deciding factor when choosing which meetings to accept is whether companies have a “strategic fit,” a concept underscored by the other panelists.

    Juan Cueva, Senior Director of Search & Evaluation at Johnson & Johnson said his firm won’t seek invitations. “We’re the ones reaching out to companies to say, we’d like to meet with you,” he explained. “And we do that based on already assessing whether or not this is a strategic fit.”

    Cueva and others mentioned using AI to screen potential partner matches, and for this reason he urged conference attendees to give thorough entries to the BIO Partnering™ platform. “My advice to the companies that are going to BIO 2026: Please fill out your profile, provide a non-confidential deck, because that’s what we’re using to identify who we want to reach out to,” he explained.

    When Catalyst meets potential partners, they have already “done our homework going into the meeting,” O’Cain said. “We’re very interested in the status and timelines of clinical development, also the regulatory path, just to really understand, you know, how this fits within our strategy.”

    What makes a successful meeting at BIO 2026

    In discussing what makes them want to continue a conversation with a potential partner, a common theme among the investors was hard data.

    “So what we really want to see are the proof points, the data, depending on the stage, especially if it’s an early stage asset, we want to see that in vivo or in vitro discovery data, validating the asset,” said Cueva of Johnson & Johnson.

    Making your story clear and concise can save a lot of time, O’Cain added. She suggested companies treat their initial contact like a 30-second elevator pitch, summarizing their advantages.

    Aralihalli said it’s important for companies to just be clear about their situation with investors.

    “They’re not going to expect every single box to necessarily be checked. But I do believe, being honest and being very transparent about what you do have and what you don’t have is going to be very important,” he said.

    Rohlff agreed, “it’s super important to be realistic about expectations.” Once the data and possibilities are clear, the decision about whether to partner depends on “positive energy, momentum, and following your gut feeling a bit,” he said.

    Find out more about the BIO International Convention.

    Find out more about the BIO Partnering™ platform.

    The post Experts explain how to drive business development with BIO 2026 appeared first on Bio.News.

    ]]>     StockWatch: enGene Shares Crater on Declines in Complete Response Rates to Bladder Cancer Therapy https://edusehat.com/en/stockwatch-engene-shares-crater-on-declines-in-complete-response-rates-to-bladder-cancer-therapy https://edusehat.com/en/stockwatch-engene-shares-crater-on-declines-in-complete-response-rates-to-bladder-cancer-therapy Mon, 11 May 2026 08:55:09 +0700 Edusehat StockWatch:, enGene, Shares, Crater, Declines, Complete, Response, Rates, Bladder, Cancer, Therapy enGene (NASDAQ: ENGN) stock suffered an 83% plunge this past week that reflects just how competitive the field is among drug candidates for nonmuscle invasive bladder cancer (NMIBC)—and how questions over clinical data are enough to send investors scurrying to sell their shares.

    enGene shares cratered 81% Thursday from $8.85 to $1.72, then fell another 13% Friday, reaching a 52-week low as it finished the week at $1.50.

    The sharp falloff followed enGene reporting updated data from its Phase II LEGEND trial (NCT04752722) assessing its nonviral gene therapy candidate detalimogene voraplasmid in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC patients with carcinoma in situ (CIS) with or without concomitant papillary disease (CIS±papillary). The data showed a drop in response rates from a November 2025 readout, falling short of company and Wall Street expectations.

    As of the April 21 data cutoff, 67 of 124 evaluable patients treated with detalimogene (formerly EG-70) achieved a 54% complete response (CR) at any time, a rate that fell to 43% (52 of 121 evaluable patients) CR rate at six months. That compares with the 63% CR at any time and 62% CR rate at six months shown for the first 62 patients assessed, as announced by enGene in November 2025.

    Also, detalimogene showed a nine-month CR rate of 32.7%, which fell to 13.3% at 12 months after treatment. Engene said it will present its data at the American Urological Association Annual Meeting (AUA 2026), set for May 15–18 in Washington, DC.

    “While durability outcomes to date are not what we hoped, these data are preliminary,” enGene president and CEO Ron Cooper stressed in a statement. “We are focused on evaluating the totality of the data as it evolves and plan to continue to engage with the FDA and the medical community.”

    Speaking to analysts, Cooper added: “The data are not yet fully mature, and the durability picture is incomplete. We plan to await longer-term durability data for all of Cohort 1 in the second half of the year and continue our ongoing discussion with the FDA regarding both our statistical analysis plans and plans for potential BLA filing.”

    “Below key benchmarks”

    At least two analysts warned that the updated data will make it harder for enGene to compete with other developers of NMIBC treatments now in the clinic.

    “Durability now screens below key benchmarks in the setting, which makes it hard for us to underwrite meaningful commercial upside for detalimogene in what will be a crowded mkt, where ~40% 12‑mo CR is the bar to clear,” cautioned Maury Raycroft, PhD, equity analyst with Jefferies, in a research note.

    Raycroft slashed enGene’s 12-month price target 82% from $28 to $5. More ominously, Raycroft chopped Jefferies’ peak sales forecasts for detalimogene by 79%—from the $1.7 billion forecasted for the gene therapy across CIS±papillary, papillary‑only, and BCG‑naïve NMIBC, to $350 million for CIS±papillary alone.

    The NMIBC treatment space has expanded in recent years as established drugs like the cancer immunotherapy blockbuster Keytruda® (pembrolizumab), marketed by Merck & Co. (NYSE: MRK), have been joined on the market by newer therapies.

    One is Johnson & Johnson (NYSE: JNJ)’s Inlexzo™ (gemcitabine), an intravesical drug releasing system (iDRS) designed to provide sustained local delivery of a cancer treatment into the bladder. Two other newer therapies are Adstiladrin® (nadofaragene firadenovec-vncg), a nonreplicating adenoviral vector-based gene therapy indicated for high-risk BCG-unresponsive NMIBC with CIS±papillary and marketed by privately held Ferring Pharmaceuticals (which licensed the drug from another private company, FKD Therapies, in 2018); and ImmunityBio (NASDAQ: IBRX)’s interleukin-15 (IL-15) receptor agonist Anktiva® (nogapendekin alfa inbakicept-pmln), indicated with BCG. ImmunityBio is part of the privately held NantWorks portfolio of companies.

    Those marketed drugs are expected to be joined over the next couple of years by candidates being developed by:

    • CG Oncology (NASDAQ: CGON), which is expected to report topline data from the Phase III PIVOT-006 trial (NCT06111235) of cretostimogene grenadenorepvec, also called CG0070, as an adjuvant therapy in intermediate-risk NMIBC. First results on the combo of cretostimogene with gemcitabine from the Phase II CORE-008 trial (NCT06567743) are to be presented at AUA 2026.
    • Relmada Therapeutics (NASDAQ: RLMD), which will present two abstracts focused on its NDV-01, a sustained-release intravesical formulation of gemcitabine and docetaxel being developed to treat NMIBC at AUA 2026. Relmada plans to present nine-month complete response data from its open-label Phase II trial (NCT06663137) assessing NDV-01 in high-risk NMIBC, as well as discuss its open-label Phase III BOOST trial (NCT07313891), which is evaluating NDV-01 vs. surveillance following transurethral resection of bladder tumor (TURBT).

    Mani Foroohar, MD, senior managing director, genetic medicines, and a senior research analyst with Leerink Partners, noted that enGene’s 54% any time and 43% six-month updated CR rates lagged behind those of three competitors: Anktiva (71% and 56%), cretostimogene (75.5% and 64%), and Inlexzo (82% and 59%). However, he added that Adstiladrin was more comparable to detalimogene, which showed rates of 51% and 41%.

    Competitive position concern

    “Weaker data in recent patient cohorts and lower-than-expected durability trends undermine our confidence in [detalimogene]’s competitive position and essentially foreclose the possibility of closing the efficacy gap with CGON, JNJ, etc., in a later LEGEND update,” Foroohar wrote in a research note.

    “With shares trading at a level pricing in program failure, investor focus will center on cohort consistency, data evolution with longer follow-up, and FDA interpretation of the totality of the dataset (questions that will take time to address and will not be resolved prior to full LEGEND data/subsequent regulatory engagement,” Foroohar added.

    Like Raycroft, Foroohar also now projects $350 million in unadjusted peak year sales for detalimogene, shredding his firm’s projection by 65% from $1 billion.

    “From here, investor discussion will center on whether longer follow-up and additional cohort maturation can stabilize efficacy trends ahead of further FDA engagement in 2H26.”

    enGene and Cooper insist that detalimogene is more than up to the challenge of competing with other NMIBC treatments. Of the 52 patients who responded at six months, 37 of 44 patients who had a nine-month assessment were in CR (an additional eight patients are pending evaluation), while 13 of 22 patients who had a 12-month assessment were in CR (an additional 11 patients are pending evaluation).

    Among patients who showed the 43% six-month CR rate, 14% (6 of 43) successfully converted from non-CR to CR post re-induction.

    The company cited other data from the LEGEND trial showing that the progression rate to muscle-invasive or advanced disease was 3.2%, a figure enGene calls low. Detalimogene was generally well tolerated, the company added, with 55% of patients having experienced a treatment-related adverse event (TRAE), mostly mild (Grade 1 and 2), though six patients (4.8%) reported a Grade 3 TRAE.

    “These updated data continue to reinforce the favorable safety and tolerability profile of detalimogene and its clinical activity in a heavily pretreated, high-risk NMIBC patient population with limited therapeutic options,” enGene’s Cooper stated. “Importantly, the low rate of progression to muscle-invasive disease leaves patients eligible for other bladder-sparing therapies.”

    Detalimogene’s favorable safety profile was also acknowledged by Jefferies’ Raycroft: “We continue to view this administrability/safety profile as a core differentiator vs other intravesical gene/immune constructs (and a key adoption lever), even as durability to date is tracking below expectations.”

    IPO roundup: Odyssey raises $304M, drops 9%

    Odyssey Therapeutics (NASDAQ: ODTX) raised $304 million in gross proceeds through an upsized initial public offering (IPO) by selling 15.5 million shares priced Thursday at $18 a share, the top of its $16–$18 range, and well above the 13.2 million shares it disclosed in its prospectus just before the offering. Concurrently, an affiliate of TPG Life Sciences Innovations purchased 1.39 million shares at the IPO price, raising another $25 million for Odyssey.

    But on their first full day of trading on Friday, Odyssey’s shares fell 9%, closing at $16.42, as investors deemed the upsizing to be aggressive.

    Odyssey, a developer of targeted therapies for autoimmune and inflammatory diseases, said it would use approximately $135 million in IPO proceeds to advance its lead candidate, the oral small molecule RIPK2 scaffolding inhibitor OD-001, through 12-week induction readouts from its planned Phase IIa combination trial and Phase IIb monotherapy trial in ulcerative colitis.

    Plans also call for using approximately $50.0 million of proceeds to advance Odyssey’s oral small molecule SLC15A4 inhibitor OD-002 through IND-enabling activities and a planned Phase I/IIa trial; and the rest for additional discovery, preclinical, and clinical activities for disclosed or future programs, enabling capabilities, as well as general corporate purposes, working capital, and other capital expenditures.

    An option to purchase up to an additional 2.325 million shares at the IPO price is held by Odyssey’s IPO underwriters: J.P. Morgan, TD Cowen, and Cantor are joint book-running managers, while Wedbush PacGrow and Oppenheimer are co-lead managers.

    The Odyssey IPO is one of four biotech IPOs emerging in recent weeks:

    • Hemab Therapeutics Holdings (NASDAQ: COAG), based in Cambridge, MA, and Copenhagen, closed its IPO on May 4, having raised $346.7 million by selling 19,262,500 shares at $18 a share—the original offering of 16.75 million shares, plus all 2,512,500 shares for which underwriters held purchase options. Goldman Sachs, Jefferies, and Evercore ISI were joint book-running managers; Wedbush PacGrow was the lead manager. Shares have since jumped 40%, closing Friday at $25.12.
    • Seaport Therapeutics (NASDAQ: SPTX) of Boston garnered $254.88 million on April 30 by selling 14.16 million shares at $18 a share. Seaport’s underwriters have a 30-day option to buy an additional 2.124 million shares at the IPO price less underwriting discounts and commissions. Goldman Sachs, J.P. Morgan, Leerink Partners, Citigroup, and Stifel are joint book-running managers. Shares have since slipped 11%, closing Friday at $16.05.
    • A day earlier, Avalyn Pharma (NASDAQ: AVLN), also of Boston, launched an IPO that garnered $300 million by selling 16,666,667 shares at $18 a share—and revived the market of biotech initial offerings after a lull during March and early April. Avalyn ultimately racked up $345 million in gross proceeds after its underwriters exercised in full their option to buy 2.5 million more shares at the IPO price. Morgan Stanley, Jefferies, Evercore ISI, and Guggenheim Securities were joint book-running managers. Shares have since vaulted 52%, closing Friday at $27.33.

    Leaders and laggards

    • Atara Biotherapeutics (NASDAQ: ATRA) shares nearly doubled, leaping 92% from $5.15 to $9.93 Thursday after saying its partner Pierre Fabre Pharmaceuticals (PFP) had a productive Type A meeting with FDA officials to discuss an approval path for tabelecleucel (tab-cel). PFP’s Biologics License Application (BLA) has been rejected twice by the agency through Complete Response Letters, the most recent one in January. The FDA, Atara said, agreed that a single-arm study with an “appropriate” historical control, conducted in a pre-specified manner, “could serve as an adequate and well-controlled study and provide safety and efficacy data in support of a future marketing application.” Atara said PFP intends to submit updated data with longer follow-up from the pivotal Phase III ALLELE trial (NCT03394365) assessing tabelecleucel in adults and children ages 2+ with relapsed/refractory Epstein-Barr virus plus post-transplant lymphoproliferative disease (PTLD) following solid organ transplant or hematopoietic cell transplant.
    • Entrada Therapeutics (NASDAQ: TRDA) shares nosedived 57% Thursday from $16.03 to $6.85 after the genetic medicine developer reported topline data from Cohort 1 of the multiple ascending dose (MAD) portion of the Phase I/II ELEVATE-44-201 trial (NCT07037862) that the company called positive, but which disappointed investors. Entrada said its Duchenne muscular dystrophy (DMD) candidate ENTR-601-44 met the trial’s primary objective by showing favorable safety and tolerability, no discontinuations, and no serious adverse events. But data showed an increase of 2.36% in dystrophin from a baseline of 4.00%—compared to the 10% increase sought by analysts such as Myles R. Minter, PhD, of William Blair. “Management is attributing the miss on biomarker data to lower-than-expected drug exposure,” Minter wrote, “likely due to the transition from dosing adults to juvenile patients.” Entrada cited other positive data, such as lower plasma exposure in Cohort 1 participants ages 6–17 vs. healthy adult volunteers; and markers of kidney function via eGFR, Cystatin C, and magnesium all falling within normal ranges and comparable to placebo.
    • Moderna (NASDAQ: MRNA) shares rose 12% Friday from $48.54 to $54.35 after a news report that the messenger RNA (mRNA)-based vaccine developer was researching vaccines designed to protect against hantaviruses. In a statement, Moderna disclosed that it has carried out early-stage vaccine research on hantaviruses with the U.S. Army Medical Research Institute of Infectious Diseases and is also partnering with the Vaccine Innovation Center at Korea University College of Medicine on a potential jab. “These efforts are early-stage and ongoing and reflect Moderna’s broader responsibility to develop countermeasures against emerging infectious diseases,” Moderna told Bloomberg News. As of Friday, the World Health Organization (WHO) has reported eight cases of hantavirus, including three deaths, among passengers aboard the MV Hondius cruise ship, traveling between Argentina and the Canary Islands in the Atlantic Ocean.
    • Viridian Therapeutics (NASDAQ: VRDN) shares jumped 33% from $14.06 to $18.75 Tuesday after the autoimmune and rare disease drug developer announced positive topline data from the Phase III REVEAL‑2 trial (NCT06625398) assessing elegrobart in chronic thyroid eye disease (TED). Doses of elegrobart given every 4 and 8 weeks achieved what Viridian called “highly statistically significant” 50% and 54% proptosis responder rates (PRR) at week 24, respectively, vs. 15% for placebo. Elegrobart is a subcutaneously delivered, half‑life‑extended monoclonal antibody targeting the insulin‑like growth factor‑1 receptor (IGF‑1R). Viridian said it remains on track to submit a Biologics License Application (BLA) to the FDA for elegrobart in Q1 2027. Viridian cashed in on the news by launching underwritten public offerings of $150 million in convertible senior notes due 2032 and $100 million in Series B nonvoting convertible preferred stock.

    The post StockWatch: enGene Shares Crater on Declines in Complete Response Rates to Bladder Cancer Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Diabetes Drug Metformin’s Blood Glucose-Lowering Effects Tied to Action on Gut Cells https://edusehat.com/en/diabetes-drug-metformins-blood-glucose-lowering-effects-tied-to-action-on-gut-cells https://edusehat.com/en/diabetes-drug-metformins-blood-glucose-lowering-effects-tied-to-action-on-gut-cells Sun, 10 May 2026 22:15:05 +0700 Edusehat Diabetes, Drug, Metformin’s, Blood, Glucose-Lowering, Effects, Tied, Action, Gut, Cells For decades, physicians and scientists have thought that metformin, a biguanide drug that is prescribed for millions of people worldwide for type 2 diabetes (T2D), mainly targets the liver to suppress glucose production. A Northwestern University-led study in mice has now found that this “wonder drug” instead acts primarily on the gut, and prevents glucose levels from rising in the blood by driving glucose utilization inside cells lining the intestine.

    The research found that metformin slows mitochondrial energy production in gut cells by inhibiting mitochondrial complex I in the intestinal epithelium. This then “co-opts” the intestines to function as a glucose sink, forcing the intestine to metabolize extra sugar. The study also found that another biguanide drug, phenformin, and the structurally unrelated supplement berberine, which is known as “nature’s Ozempic,” appear to engage the same pathway in the gut as does metformin.

    The preclinical findings could help to explain several gut-related clinical effects in people who take metformin and suggest that modulating mitochondrial metabolism in the gut may represent an effective strategy for controlling blood sugar. “Metformin essentially helps the intestine suck the glucose out of the bloodstream, which further highlights that the gut plays a major role in regulating blood sugar levels,” said corresponding author Navdeep Chandel, PhD, professor of biochemistry and molecular genetics at Northwestern University Feinberg School of Medicine.

    Chandel is senior and co-corresponding author of the researchers’ published paper in Nature Metabolism, titled “Metformin inhibits mitochondrial complex I in intestinal epithelium to promote glycaemic control.” Chandel is also the David W. Cugell, MD, Professor of Medicine (Pulmonology and Critical Care), Biochemistry and Molecular Genetics, and an investigator with the Chan Zuckerberg Initiative. The study’s first author is Zach Sebo, PhD, a postdoctoral fellow in the Chandel lab who will soon start his own research group at the University of Kansas School of Medicine.

    Metformin is the most widely prescribed medication for type 2 diabetes and the biguanide class drug approved by the FDA, the authors wrote. However, they noted, “Despite its extensive use, the mechanisms underlying its clinical effects, including attenuated postprandial glucose excursions and elevated intestinal glucose uptake, remain unclear.”

    The body relies on glucose as a fast and versatile fuel, but too much glucose can lead to insulin resistance and ultimately damage blood vessels and organs. The newly reported study builds on findings from previous work in Chandel’s lab, which found that metformin lowers blood sugar by blocking a specific part of the cell’s energy-making machinery, mitochondrial complex I, a key enzyme in cellular respiration. The research reported in Nature Metabolism extends that work by pinpointing the specific tissue targeted by metformin.

    The study used a mouse model genetically engineered to express a yeast enzyme (NDI1) that mimics mitochondrial complex I but is resistant to inhibition by metformin. By expressing NDI1 specifically in intestinal cells, those gut cells resisted metformin’s effects. In these mice, the drug’s ability to lower blood glucose was significantly reduced, demonstrating that inhibition of mitochondrial complex I in the gut is a key driver of its therapeutic action. “In this study, we show how metformin exerts multiple clinical effects through selective inhibition of mitochondrial complex I in the intestinal epithelium,” they wrote.

    Corresponding author Navdeep Chandel in his lab in Chicago. [Kristin Samuelson, Northwestern University]
    Corresponding author Navdeep Chandel, PhD, in his lab in Chicago. [Kristin Samuelson, Northwestern University]
    Metformin is currently the only FDA-approved biguanide drug, but the team found that another biguanide, phenformin, which had previously been used to control blood glucose but was then withdrawn, also lowered blood glucose through the same mechanism. The findings suggest that directing drugs or supplements to the gut could be an effective strategy for controlling blood sugar, Chandel said. Sebo added, “Our study suggests that revisiting assumptions about metformin’s mechanism may offer a more detailed understanding of how it works.”

    The study revealed unexpected parallels with berberine, a popular plant-derived OTC supplement that is often used to control blood sugar. Berberine has recently gained attention on social media as “nature’s Ozempic,” though experts caution that evidence is still limited, and it should not be used as a substitute for approved medications. The study by Chanel and colleagues has now found that berberine appears to engage the same pathway as metformin in the intestine. “Thus, we identify mitochondrial complex I in intestinal epithelium as a shared and essential therapeutic target for metformin, phenformin, and berberine,” the authors stated.

    “Metformin has decades of clinical evidence behind it, whereas supplements like berberine are far less rigorously tested,” Chandel said. “If you’re going to use berberine, you may as well use the real deal.”

    The study results may help to explain clinical observations among people who take metformin. According to Chandel, individuals who take metformin tend to have lower blood sugar after meals, and the study suggests that metformin turns the gut into a “sponge” that soaks up extra sugar. Individuals taking metformin also tend to have lower levels of circulating citrulline, which is made only by mitochondria in small intestine cells. If metformin inhibits mitochondria, citrulline production drops. Taking metformin also increases levels of GDF15, a hormone linked to reduced appetite and weight loss. The gut senses energy stress and sends out GDF15, which tells the brain to eat less and adjust metabolism.

    “In addition to enhanced intestinal glucose utilization and blood glucose clearance, this mechanism accounts for metformin-induced citrulline depletion, improved postprandial glycaemia, and elevated lactoyl-phenylalanine (Lac-Phe) and growth differentiation factor 15 (GDF15) levels—all of which are definitive clinical outcomes caused by metformin treatment,” the authors wrote in summary.

    “People have always wondered how one drug can do 10 things,” Chandel said. “Well, it can do that if the drug is hitting a big node in a cell, and hitting mitochondria in a cell is a big node. So, if you can get into those cells and inhibit mitochondria, it’s going to have huge effects.”

    The post Diabetes Drug Metformin’s Blood Glucose-Lowering Effects Tied to Action on Gut Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     How Digital Orchestration Is Redefining Regulatory Infrastructure for Cell and Gene Therapy https://edusehat.com/en/how-digital-orchestration-is-redefining-regulatory-infrastructure-for-cell-and-gene-therapy https://edusehat.com/en/how-digital-orchestration-is-redefining-regulatory-infrastructure-for-cell-and-gene-therapy Sat, 09 May 2026 06:35:11 +0700 Edusehat How, Digital, Orchestration, Redefining, Regulatory, Infrastructure, for, Cell, and, Gene, Therapy The rapid growth of cell and gene therapies is exposing structural limitations in how traditional biopharmaceutical systems were designed. Unlike batch-based manufacturing models, these therapies are patient-specific, requiring tightly coordinated execution across clinical, manufacturing, and quality domains.

    In this environment, maintaining chain-of-identity and chain-of-custody is not simply a regulatory requirement, but a foundational design constraint. As programs move from early clinical development into global commercialization, gaps in system-level orchestration can translate directly into compliance and operational risk.

    These pressures are driving renewed attention toward how digital infrastructure is architected in regulated life sciences, particularly around validation, traceability, and cross-system integration.

    Monika Birdi, global product strategy leader for SAP’s Cell and Gene Therapy Orchestration (CGTO) platform, has spent more than two decades working on regulated enterprise systems across industries, with a recent focus on advanced therapy manufacturing and supply chains. In this interview with GEN, Birdi discusses SAP CGTO’s architecture, regulatory design in SAP Batch Release Hub (BRH) and Intelligent Clinical Supply Management (ICSM), and digital frameworks for inspection readiness, jurisdictional control, and chain-of-identity enforcement. She also shares insights on balancing innovation with GxP validation and building digital infrastructure for global commercialization of patient-specific therapies.

    GEN: What foundational lessons about compliance architecture, data integrity, and large-scale system design most influenced your transition into life sciences and cell and gene therapy?

    MB: The biggest shift I experienced after moving to life sciences was in perspective and significance. During the many years I worked building large-scale regulated systems in diverse industries, a failure of data integrity meant financial loss or reputational damage. However, when I moved into the pharma and biotech life science sectors, I realized that in this domain, there was zero margin for error. If the chain-identity breaks anywhere in that journey, there is no fallback.

    Monika Birdi
    Monika Birdi

    This reality puts compliance at the heart of architecture. Controls need to be built from the start and cannot be retrofitted later. We must stop treating compliance and scalability as competing priorities, because when you architect them together, one enables the other.

     

     

    GEN: When you began working in advanced therapies, what operational or digital fragilities did you observe in early-stage programs that signaled a structural gap in how regulated supply chains were being architected?

    MB: The first thing we need to understand is that advanced therapies are completely different from traditional small molecule manufacturing. Hence, for organizations that have been in the pharma business, shifting to manufacture advanced therapies is different from both scientific and architectural perspectives. The same tools that worked for small molecules will not serve the end-to-end business for advanced therapies.

    The common structural gaps I observed were around chain of identity and chain of custody tracking. Since these are typically not part of the native system design, every single process runs via piles of papers that are difficult to organize and trace. In these cases, there was no orchestration layer and all the systems—such as clinical, manufacturing, quality—were running in silos.

    GEN: You have led the design and commercialization of SAP CGTO. What operational failures or regulatory risks did you observe in early-stage CGT programs that convinced you digital orchestration had to be architected differently from traditional pharma systems?

    MB: Traditional pharma systems are built around batch manufacturing, where thousands of units are manufactured in one batch. Even if one batch fails the compliance, the next batch can be used by discarding the non-compliant batch. Chain-of-identity is being maintained through a combination of spreadsheets. This is what convinced me to rethink orchestration. You can’t take a traditional batch management system and configure your way into CGT compliance.

    The right therapy needs to reach the right patient. So, with CGTO, the design question was never “how do we adapt existing functionality” to make a compliant solution. Our approach was to ask, “if we are building this from scratch for one patient and one batch, what should the process actually look like?”

    GEN: Autologous cell and gene therapy manufacturing is patient-specific and tightly synchronized. How did you architect SAP CGTO to enforce chain-of-identity and chain-of-custody controls at every transition point rather than relying on retrospective reconciliation?

    MB: Personalized advanced therapies operate under a unique manufacturing cycle, which makes conventional post-production reconciliation processes ineffective. We needed an architecture that shifts the control point from “detect and correct” to “prevent and confirm” for every transaction.

    With SAP CGTO, every transition point, from receipt at the plant, disposition, manufacturing start, and allocation to final shipment, is within an order and includes validations to make sure the right patient gets the right therapy. The system won’t let you proceed with a mismatched identity. These validations ensure that the process is stopped immediately, instead of alerting the user at a later point in time.

    GEN: In your work on SAP BRH, you focused on jurisdiction control and regulatory components. How do digital release architectures need to evolve to support multi-country regulatory environments while preserving data integrity and inspection readiness?

    MB: Most organizations use local Standard Operating Procedures (SOPs) and spreadsheets, utilized by people who have been around long enough to manage the regulatory requirements. This system will likely fail if a critical employee leaves, or if you are entering a new market under pressure, or when an inspector asks you to reconstruct a release decision from two years ago.

    Jurisdictional controls should be part of the solution. Once validations are embedded directly into workflows, compliance becomes part of the business process. The audit trails are thus automatically created as a natural result of doing the job.

    GEN: You believe designing infrastructure before scale exposes operational gaps. What are the most common digital fragilities you see when sponsors defer enterprise architecture decisions until Phase III or Phase IV?

    MB: In cell and gene therapy, many sponsors treat digital infrastructure like office furniture: something to worry about later. The largest problems tend to be in two areas: traceability and coordination.

    gene therapy
    Credit: Metamorworks/Getty Images

    In terms of traceability, it’s common for programs to track materials in whatever way is convenient at that time, such as spreadsheets, half-set-up software, or systems that don’t talk to one another. But for a hundred patients across many sites, it becomes nearly impossible to track it all correctly, especially when the FDA starts asking questions.

    But as trials get bigger, it becomes difficult to manage all of it through those means—and in fact, it can become dangerous. Then, as the trial advances, scale your investment in alignment with emerging trial results.

    GEN: Across CGTO, BRH, and ICSM, you have helped define regulatory-ready digital architectures for emerging therapy models. What measurable operational or compliance outcomes have resulted from these implementations, and how do they demonstrate advancement in the field’s digital maturity?

    MB: The actual benefit of digitalization in advanced therapies shows up in audits and inspections. By ensuring that digital systems are properly implemented from the beginning and integrated with quality and batch record systems, decisions regarding the release of batches are much faster and more accurate.

    In the area of clinical trials, integrated systems enable teams to predict and prevent problems rather than simply reacting to them. This results in fewer delays, and the benefits are easy to demonstrate and prove to the authorities.

    GEN:  What principles guide your approach to building compliant cloud-native platforms that remain modular, secure, and extensible?

    MB: I believe modularity itself is a compliance strategy. Highly integrated platforms introduce hidden risk, because every regulatory change or market requirement can trigger system‑wide retesting.

    I design security and extensibility together, with clear separation between what is validated, configurable, and subject to formal change control. That clarity allows teams to move faster without compromising compliance.

    Commercialization teaches you that adoption depends on validation of reality. A platform can be technically strong, but if it is difficult to validate and operate in a regulated environment, it will not be scaled. The most successful products do not transfer the customer’s validation burden.

    GEN: As advanced therapy pipelines expand and manufacturing networks become more geographically distributed, which architectural capabilities will determine whether organizations can sustain compliant commercialization without repeated remediation cycles?

    MB: We need to start building compliance as the core of architecture instead of treating it as per each unique market need. Once the foundation has been set, market regulations can be adjusted accordingly. A few capabilities in which I would invest early include real-time chain-of-identity enforcement, jurisdictional logic embedded in workflows rather than documented beside them, and audit structures that generate inspection-ready data as a natural output, as opposed to a reconstruction exercise. What really matters most is the flexibility of architecture. Regulations evolve, new markets pop up, and manufacturing networks constantly adapt and relocate. This requires organizations to build adaptable and modular architecture that can evolve with growth.

     

    The post How Digital Orchestration Is Redefining Regulatory Infrastructure for Cell and Gene Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Here’s what you need to know about the cruise ship hantavirus outbreak https://edusehat.com/en/heres-what-you-need-to-know-about-the-cruise-ship-hantavirus-outbreak https://edusehat.com/en/heres-what-you-need-to-know-about-the-cruise-ship-hantavirus-outbreak Sat, 09 May 2026 03:05:02 +0700 Edusehat Here’s, what, you, need, know, about, the, cruise, ship, hantavirus, outbreak Eight passengers aboard a Dutch-flagged cruise ship have contracted a type of hantavirus, a rare virus transmitted by rats. Three of them have died. As the ship prepares to dock in the Canary Islands, plans are being finalized to let the remaining passengers and crew disembark safely.

    The virus in question appears to have a high fatality rate. Read on for answers to the big questions surrounding the outbreak—and to hear why health experts don’t expect a rerun of the covid-19 pandemic.

    What is hantavirus?

    Hantaviruses are a group of viruses that typically infect rodents but can be transmitted to humans through exposure to the animals or their droppings, urine, or saliva. The viruses don’t seem to cause illness in rodents, but they can make people very unwell. The symptoms can depend on the type of hantavirus a person has been exposed to. Varieties found in the Americas can cause hantavirus cardiopulmonary syndrome, which affects the lungs and heart and has a fatality rate of up to 50%.

    That condition made headlines last year when it caused the death of pianist Betsy Arakawa, the wife of actor Gene Hackman. 

    How many cases have there been so far?

    On April 6, a man aboard the MV Hondius developed respiratory symptoms. He became very unwell and died just five days later. His wife, who left the ship at the island of Saint Helena, also developed symptoms. Her health deteriorated during a flight to Johannesburg, South Africa, and she died the following day, on April 26. South Africa’s National Institute of Communicable Diseases tested samples taken from the woman and confirmed that she had hantavirus.

    A third person aboard the ship, who developed symptoms on April 28, died on May 2. Four other passengers who became ill were evacuated—one to South Africa and three to the Netherlands.

    An eighth person had disembarked in Saint Helena and reported similar symptoms once he was in Zurich, Switzerland. A team at Geneva University Hospitals confirmed that he had become ill from the Andes virus—a form of hantavirus that can be spread between people.

    Could this be the start of the next pandemic?

    Health experts don’t believe so. They stress that the situation is nothing like the one the coronavirus that causes covid-19 presented in 2020. For a start, the Andes virus is not a mysterious new virus—scientists already have an understanding of it, and Argentina is sharing diagnostic kits it has already developed.

    The virus also doesn’t spread in the same way. Officials at the World Health Organization emphasized that the spread of hantavirus requires close contact—the kind a person might have with a partner, household member, or medical caregiver.

    The cruise ship outbreak represents “a specific confined setting where people are interacting in a prolonged close contact,” Abdirahman Mahamud, the alert and response director for the WHO’s health emergency program, said at a press event on Thursday. “With the experience our member states have, and the actions they have taken, we believe that this will not lead to a subsequent chain of transmission.”

    What about the rest of the people onboard the ship?

    All the remaining passengers have been asked to stay in their cabins, which the WHO says are being disinfected. Doctors and health professionals from the WHO and the European Center for Disease Prevention and Control have boarded the ship and are assessing everyone on board.

    So far, no one else on board has developed symptoms, Maria Van Kerkhove, WHO acting director for epidemic and pandemic management, said at the press event. That’s “a good sign,” she said, but she added that the Andes virus has a long incubation period (around six weeks). Passengers are being advised to wear a medical mask when they leave their rooms.

    At the same event, WHO director general Tedros Adhanom Ghebreyesus said he was in regular contact with the ship’s captain, who was reporting that “morale had increased significantly” since the ship started its journey to the Canary Islands.

    What do we know about the Andes virus?

    The Andes virus is the only hantavirus that is known to be transmitted between people. That transmission seems to rely on prolonged, intimate contact.

    There was an Andes virus outbreak in Argentina around eight years ago. Between November 2018 and February 2019, there were 34 confirmed cases of infection, and 11 deaths. That outbreak was triggered when a person with symptoms attended a social gathering, said Tedros. “We are in a similar situation right now,” he said. “A cluster in a confined space with close contact.”

    The fact that the 2018 outbreak was limited to 34 cases should be somewhat reassuring, he implied. “We believe this will be a limited outbreak if the public health measures are implemented and solidarity is shown across all countries,” he said.

    How is hantavirus treated?

    Unfortunately, we don’t have any specific antiviral treatments or vaccines for hantavirus. The WHO recommends early intensive care for people who develop symptoms. “This can save lives,” Anaïs Legand, WHO technical lead on viral hemorrhagic fevers, said on Thursday.

    How did people get infected in the first place?

    We don’t yet have an answer to that. But we do know that the couple who died had traveled through Argentina, Chile, and Uruguay on a birdwatching trip before they boarded the ship. That trip included visits to areas where species of rats that carry the Andes virus are known to live. The WHO is working with authorities in Argentina to try to retrace the couple’s movements on that trip.

    Has the virus spread beyond the ship?

    We don’t yet know for sure. The WHO is receiving reports of “potential suspect cases,” Van Kerkhove said at the Thursday briefing. Some of them have links to the ship or its passengers. Each “alert” will be followed up by health authorities in the relevant country, she said.

    Has the US withdrawal from WHO affected anything?

    Five US states have said they are monitoring US nationals who have disembarked from the ship. WHO officials are stressing that they are still sharing technical information with the US Centers for Disease Control and Prevention. “Things are … as they used to be,” Tedros said. “WHO’s mission is to help the world to be safe … and we want the American people to be safe as well.”

    But it’s worth noting that cuts made by the Trump administration aren’t exactly putting the US in a good position for events like these. Last year, all full-time employees in the CDC’s Vessel Sanitation Program—which helps prevent and control illness outbreaks on cruise ships—were laid off. Further cuts to the CDC have left public health experts worried about how ill prepared the US is to deal with future disease outbreaks.

    What will happen next?

    Any suspected cases will be monitored by health authorities. Passengers are due to disembark in Tenerife in the Canary Islands on Sunday, May 10, and the WHO has said it will work with the Spanish government to ensure that the risk to residents remains low and that the passengers are treated with dignity and respect.

    In the meantime, scientists are working to fully sequence the genome of the virus from patient samples. They want to find out if it is different from the viruses involved in the previous cases. “So far, we haven’t seen anything unusual,” said Van Kerkhove.

    ]]>     G-Link CAR T Delivery Platform Showcased at ASGCT https://edusehat.com/en/g-link-car-t-delivery-platform-showcased-at-asgct-10512 https://edusehat.com/en/g-link-car-t-delivery-platform-showcased-at-asgct-10512 Sat, 09 May 2026 03:00:10 +0700 Edusehat G-Link, CAR, Delivery, Platform, Showcased, ASGCT Vyriad reports that it will showcase its latest CAR T delivery technology platform, G-Link, through a schedule of presentations, technical sessions, and exhibition activities at ASGCT. The modular plug-and-play protein adapter developed in collaboration with Menachem Rubenstein, PhD, of the Weizmann Institute allows drug developers to cap and retarget existing lentiviral vectors for in vivo delivery, according to the company.

    By leveraging G-Link, wild-type lentiviral vectors can be reprogrammed for in vivo applications without the need for intensive vector re-engineering, effectively shortening development timelines for next-generation CAR T and other cell therapies, notes a company spokesperson, who adds that G-Link can also be used to simplify ex vivo CAR T manufacturing and significantly improve T cell transduction efficiency without redesigning vectors.

    “I believe that G-Link can address some of the most persistent challenges in in vivo delivery and we are excited to unveil it at ASGCT this year,” says Stephen Russell, PhD, CEO of Vyriad. “Our participation this year underscores our clear mission: to replace complex, weeks-long manufacturing cycles with precise, off-the-shelf immunotherapies. With G-Link, we aim to foster collaborations that will define the next generation of in vivo cell therapies.”

    Vyriad’s VV169 in vivo CAR T program will progress into clinical development later this year, while the G-Link platform will advance towards clinical translation later in the future, continues Russell.

     

    The post G-Link CAR T Delivery Platform Showcased at ASGCT appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Remembering J. Craig Venter, PhD https://edusehat.com/en/remembering-j-craig-venter-phd https://edusehat.com/en/remembering-j-craig-venter-phd Sat, 09 May 2026 03:00:08 +0700 Edusehat Remembering, Craig, Venter, PhD J. Craig Venter, PhD recently passed away at the age of 79 from complications following a cancer diagnosis. He was well known in both science and industry and was an integral part of sequencing the human genome in the late 90s, competing with the government organized Human Genome Project. Throughout his career, he made many other important contributions in microbiology, with the “minimal cell,” in synthetic biology, and in personalized medicine. GEN editors share anecdotes of their experiences with him, reflect on the impact that his work has had on various fields in biology, in biotech, and in how the world has responded to the disruptions caused by Venter.

    Listed below are links to the GEN stories referenced in this episode of Touching Base:

    Genomics Pioneer and Life Sciences Entrepreneur J. Craig Venter Dies at 79
    GEN, April 30, 2026

    J. Craig Venter Describes a Human Genomics Revolution Still In Progress
    By J. Craig Venter, PhD, GEN, June 13, 2025

    Lessons from the Minimal Cell
    By Hana El-Samad, PhD, GEN, August 21, 2023

    From Sequencing to Sailing: Three Decades of Adventure with Craig Venter
    By Fay Lin, PhD, GEN, March 8, 2023

    “Cracking the Genome”
    By Kevin Davies, PhD

    Touching Base Podcast
    Hosted by Corinna Singleman, PhD

    Behind the Breakthroughs
    Hosted by Jonathan D. Grinstein, PhD

    The post Remembering J. Craig Venter, PhD appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ASGCT Honors Mohamed Abou‑el‑Enein as Outstanding New Investigator https://edusehat.com/en/asgct-honors-mohamed-abouelenein-as-outstanding-new-investigator https://edusehat.com/en/asgct-honors-mohamed-abouelenein-as-outstanding-new-investigator Fri, 08 May 2026 23:25:11 +0700 Edusehat ASGCT, Honors, Mohamed, Abou‑el‑Enein, Outstanding, New, Investigator Some scientists build tools. Others build bridges. Mohamed Abou‑el‑Enein, MD, PhD, does both—engineering a high‑dimensional platform that temporally maps CAR T cells, while constructing the translational infrastructure needed to move them from concept to clinic. That dual lens has now earned him two of the American Society of Gene and Cell Therapy’s (ASGCT) top honors: the 2026 Outstanding New Investigator Award and, to his lab, the Best of Molecular Therapy Award, which features the contributions of leading early-career authors to the Molecular Therapy family of journals.

    This year’s Molecular Therapy recognition highlights a study from Abou‑el‑Enein’s lab, published in May 2025, that uses spectral flow cytometry to map how CAR T cells remodel during manufacturing, revealing a five‑day window when the cells are most potent. The work reflects a core principle of his group: you cannot rationally engineer what you cannot precisely measure. Their high‑dimensional analytical tools are designed to simultaneously capture the full profile of each engineered cell, information that directly shapes how next‑generation therapies are built.

    “Spending my career bridging scientific discovery and patient care, both by supporting others and through our own research, makes this dual recognition especially meaningful,” said Abou‑el‑Enein. “The contribution of translational scientists has long been underestimated and under‑acknowledged, and having a committee of peers recognize its value means a great deal to me.”

    Mohamed Abou-el-Enein and Amaia Cadinanos-Garai [Anson Cheung]
    As a physician‑scientist, Abou‑el‑Enein brings comprehensive training to the problems of cell and gene therapy translation, a trajectory shaped across leading institutions globally. His path spans clinical medicine, regulatory science, biomanufacturing, and data science—training that now converges at the University of Southern California (USC), where he leads the USC/Children’s Hospital Los Angeles Cell Therapy Program and directs the institution’s cGMP manufacturing facility. His team has built a platform capable of producing a wide range of advanced therapies, from viral vectors to stem‑cell–based products. As USC’s Chuck Murry, MD, PhD, director of the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research noted, he brings “an eclectic mix of cell biology, biomanufacturing, and patient‑centered humanity” to the role.

    The Abou‑el‑Enein lab extends that mission by developing computational platforms that unify analytical data with predictive modeling. Their newest effort—UNICORN (UNIfying Cell Therapy Outcome prediction and Regulatory Navigation)—integrates high‑dimensional analytics with machine learning to forecast therapeutic performance and streamline regulatory decision‑making for pediatric cancers and rare diseases. It’s a natural evolution of the group’s earlier work, which established a powerful analytical framework for tracking CAR T cell states over time.

    Their broader analytical ecosystem includes tools for single‑cell characterization, computational modeling, and non‑viral precision genome engineering, all designed to support translation from the earliest stages of design.

    As he prepares to deliver his Outstanding New Investigator lecture in Boston, Abou‑el‑Enein said the meeting is a chance to reconnect with the community that shaped his translational philosophy.

    “These awards are catalysts that give me motivation to keep going,” he said. “I always remind my team… we do science because we believe that what we do will make a difference. It’s really about helping patients and making sure they have a real chance.”

    The post ASGCT Honors Mohamed Abou‑el‑Enein as Outstanding New Investigator appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Gene Therapy Briefs: Regeneron Wins FDA Approval for First Neurosensory Gene Therapy https://edusehat.com/en/gene-therapy-briefs-regeneron-wins-fda-approval-for-first-neurosensory-gene-therapy https://edusehat.com/en/gene-therapy-briefs-regeneron-wins-fda-approval-for-first-neurosensory-gene-therapy Fri, 08 May 2026 23:25:10 +0700 Edusehat Gene, Therapy, Briefs:, Regeneron, Wins, FDA, Approval, for, First, Neurosensory, Gene, Therapy The FDA has granted accelerated approval to Regeneron Pharmaceuticals’ Otarmeni™ (lunsotogene parvec-cwha) as the first gene therapy designed to restore a neurosensory function to normal levels.

    Otarmeni is an adeno-associated virus vector-based gene therapy indicated for treating children and adults with severe-to-profound and profound sensorineural hearing loss, defined as any frequency >90 decibel hearing level [dB HL], associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear.

    Otarmeni (formerly DB-OTO) is the first and only in vivo gene therapy indicated for OTOF-related hearing loss. Regeneron said it will make Otarmeni available for free in the U.S.

    The FDA based its accelerated approval decision on the improvement of hearing sensitivity as measured by average pure tone audiometry (PTA) at week 24 during the Phase I/II CHORD trial (NCT05788536). Twenty participants ages 10 months to 16 years received a single dose of Otarmeni via intracochlear infusion—10 patients in one ear, the other 10 in both ears. Data from CHORD showed:

    • 80% of participants (16 of 20) reported hearing improvements per pure tone audiometry assessments at ≤70 dB HL at 24 weeks, achieving the trial’s primary endpoint, while one additional participant achieved the threshold by week 48.
    • 70% (14 of 20) showed an auditory brainstem response (ABR) at ≤90 decibels at 24 weeks, achieving the trial’s key secondary endpoint.
    • Among participants followed to 48 weeks, all prior responders maintained a response to therapy, and 42% of all participants (five of 12) achieved normal hearing that included whispers (≤25 dB HL).

    “This unprecedented breakthrough in gene therapy has already proven to be life-changing for many of the children in our clinical trial and their families,” said George D. Yancopoulos, MD, PhD, board co-chair, president and chief scientific officer of Regeneron. 1

    The FDA said its accelerated approval may hinge upon verification and description of clinical benefit in the confirmatory portion of the CHOIRD trial, a first-in-human, multicenter, open-label trial designed to assess the safety, tolerability and preliminary efficacy of DB-OTO in infants, children, and adolescents with otoferlin variants.

    Otarmeni is the first gene therapy, and second new molecular entity, to win FDA approval under the agency’s Commissioner’s National Priority Voucher (CNPV) pilot program.

    Launched in October by FDA Commissioner Martin A. Makary, MD, CNPV awards vouchers to drug developers whose work is deemed to address a health crisis in the U.S., deliver more innovative cures, address unmet public health needs, and increase domestic drug manufacturing as a national security issue. The vouchers entitle companies to reviews of their final applications within a target timeframe of 1–2 months rather than the standard 10–12 months.

    Intellia’s Lonvo-Z begins rolling BLA following positive Phase III data

    Intellia Therapeutics has launched a rolling Biologics License Application (BLA) submission to the FDA seeking regulatory approval of lonvoguran ziclumeran (lonvo-z), after announcing positive topline results from the global Phase III HAELO trial (NCT06634420) in hereditary angioedema (HAE)—the first Phase III data reported for an in vivo gene editing therapy.

    HAELO is a randomized, double-blind, placebo-controlled Phase III trial designed to evaluate the efficacy and safety of a one-time 50 mg dose of lonvo-z in adults and adolescents aged 16 years and older with Type I or Type II HAE. The trial’s key endpoints focused on the number of HAE attacks experienced by patients, quality of life, safety and tolerability. Eighty patients were enrolled with 52 receiving lonvo-z and 28, placebo.

    HAELO met its primary endpoint. For the six-month efficacy evaluation period (weeks 5 to 28), a one-time infusion of lonvo-z reduced attacks by 87% vs. placebo, with a mean monthly attack rate of 0.26 in the lonvo-z arm vs. 2.10 in the placebo arm.

    Other key findings from HAELO:

    • The trial met all key secondary endpoints with statistical significance (p<0.0001). These included a 62% rate of patients who were entirely attack free and therapy free in the lonvo-z arm for the six-month efficacy evaluation period, vs. 11% of patients in the placebo arm.
    • Lonvo-z showed favorable safety and tolerability data. The most common treatment emergent adverse events (TEAEs) during the primary observation period (infusion through week 28) were infusion-related reactions, headache and fatigue. All TEAEs reported as of the data cutoff (February 10, 2026) were mild or moderate, with no serious adverse events observed in the lonvo-z arm.
    • As of the data cutoff, all patients who received lonvo-z at baseline or in crossover after week 28 remained long-term prophylaxis (LTP) free.

    “Today’s HAELO results represent a profound milestone for Intellia, the broader CRISPR and precision medicine fields and, most importantly, the HAE community,” said John Leonard, MD, Intellia’s president and CEO. “These data affirm lonvo-z’s potential, with one dose, to offer prolonged freedom from both attacks and the need for ongoing therapy.” 2

    Intellia said researchers plan to present additional clinical data from HAELO at the 2026 European Academy of Allergy and Clinical Immunology Congress (EAACI), set for June 12-15 in Istanbul, Turkey (Abstract #100217).

    Lonvo-z is designed to inactivate the kallikrein B1 (KLKB1) gene in order to permanently lower kallikrein and bradykinin levels. Lonvo-z is designed as a one-time treatment that is administered in an outpatient setting.

    Intellia said it is preparing for a potential U.S. launch of lonvo-z in the first half of 2027.

    J. Craig Venter Dies: Pioneer in gene discovery, genomics, and synthetic biology

    Craig Venter, PhD, a pioneer in gene discovery, human genomics, and synthetic biology, died April 29 in San Diego after a brief hospitalization for unexpected side effects that arose from treatment of recently diagnosed cancer. He was 79.

    Venter was founder, board chair, and CEO of the institute that bears his name in La Jolla, CA. Earlier at the National Institutes of Health (NIH), he helped pioneer gene discovery using expressed sequence tags (ESTs), enabling rapid identification of large numbers of human genes and accelerating genome mapping efforts.

    He went on to lead efforts that produced the first draft sequences of the human genome. He and colleagues later published the first high-quality diploid human genome, a scientific milestone that demonstrated the importance of capturing genetic variation inherited from both parents.

    Venter’s work helped define and advance modern genomics, as well as launched the field of synthetic biology, where he and his teams constructed the first self-replicating bacterial cell controlled by a chemically synthesized genome—proof that genomes could be designed digitally, built from chemical components, and like a computer, booted up to run a living cell.

    Through the Sorcerer II Global Ocean Sampling Expedition, Venter and his teams used metagenomics to reveal microbial diversity, reporting the discovery of millions of new genes and expanding the known universe of protein families—work that added to knowledge and insight of the ocean microbiome and its role in planetary systems.

    “Venter was controversial and often challenged the scientific orthodoxy, with critics accusing him of hype and going overboard on privatization,” said John Sterling, Editor in Chief of Genetic Engineering & Biotechnology News, who has known and worked editorially with Venter over the past 35 years. “To many, he was a visionary focusing on technological acceleration and blending academic science with the zeal of an entrepreneur. Supporters saw him as a pioneer who sped up genomics by years.” 3

    In addition to founding the J. Craig Venter Institute (JCVI), Venter was a serial entrepreneur who co-founded Synthetic Genomics, Human Longevity, and most recently Diploid Genomics, advancing efforts designed to translate genomics and synthetic biology into tools for health and society.

    He was also a fierce advocate for robust federal science funding, as well as for partnerships that accelerate progress across government, academia, and industry.

    “Craig believed that science moves forward when people are willing to think differently, move decisively, and build what doesn’t yet exist,” said Anders Dale, president of JCVI. “His leadership and vision reshaped genomics and helped ignite synthetic biology. We will honor his legacy by continuing the mission he built—advancing genomic science, championing the public investments that make discovery possible, and partnering broadly to turn knowledge into impact.” 4

    Rocket Pharma selling Priority Review Voucher for Kresladi™ for $180M

    Rocket Pharmaceuticals has agreed to sell for $180 million the Rare Pediatric Disease Priority Review Voucher (PRV) it was awarded by the FDA after the agency granted accelerated approval of Kresladi™ (marnetegragene autotemcel).

    Kresladi is an autologous hematopoietic stem cell-based gene therapy indicated to treat children with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.

    The indication was approved in March under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression. The accelerated approval of Kresladi is subject to confirmation of its clinical benefit, to be based on an evaluation of longer-term follow-up data of treated patients in the ongoing Phase I/II trial (NCT03812263) and through a post-marketing registry. The study generated positive topline data showing 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all nine LAD-I patients with 18 to 42 months of available follow-up.

    The accelerated approval followed a resubmission of Rocket’s Biologics License Application for Kresladi. The original submission was rejected by the FDA in 2024 through a Complete Response Letter that requested additional Chemistry, Manufacturing, and Controls (CMC) information, but did not raise safety or efficacy issues about the gene therapy.

    Rocket said it plans to use proceeds from the PRV sale toward advancing its prioritized cardiovascular gene therapy pipeline, including clinical-stage programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM).

    “The monetization of our PRV, following the FDA approval of Kresladi, provides meaningful non-dilutive capital and extends our cash runway into the second quarter of 2028,” said Gaurav Shah, MD, Rocket Pharmaceuticals CEO. “This strengthens our ability to advance key clinical milestones across our cardiovascular gene therapy pipeline, with all programs on track.” 5

    Passage Bio cuts staff 75%, launches strategic review

    Passage Bio said it will eliminate 75% of its staff in a cost-cutting restructuring that is part of the company’s effort to review strategic alternatives.

    “The Company expects that the aggregate severance and exit costs for the Restructuring Plan will be approximately $3.3 million, which will be recorded primarily in the second quarter of 2026,” Passage Bio said in a regulatory filing. 6

    Passage Bio has said it plans to review strategic alternatives that may include merger or acquisition transactions, a reverse merger, a sale of assets of the company, strategic partnerships, licensing opportunities, or other potential paths.

    The restructuring followed Passage Bio receiving feedback during a Type C meeting with officials at the FDA that indicated that the company will be required to complete a randomized controlled registrational trial evaluating its lead pipeline candidate PBFT02 as a treatment for frontotemporal dementia (FTD) with granulin (GRN) mutations.

    PBFT02 is a gene replacement therapy that uses an adeno-associated virus serotype 1 (AAV1) viral vector to deliver, through intra cisterna magna (ICM) administration, a functional GRN gene that encodes the progranulin protein (PGRN).

    The 75% workforce cut amounts to approximately 18 people, based on the 24 full-time employees it reported as of December 31, 2025, according to its annual report.

    LEO Pharma acquires Replay for $50M upfront, milestones

    LEO Pharma has agreed to acquire Replay, a developer of gene therapies for rare genetic dermatological conditions, in a deal that the buyer said will add deep expertise and a next-generation gene therapy platform to its pipeline, namely Replay’s high‑payload herpes simplex virus (HSV) delivery vector.

    LEO Pharma plans to acquire Replay for $50 million upfront, plus milestone payments and tiered single-digit royalties.

    Replay’s gene therapy platform is designed to leverage HSV’s capacity to deliver large genes, which according to LEO makes it well suited for addressing rare, genetically driven dermatological conditions. The genetically modified HSV therapy is formulated as a topical gel that targets the deficient gene when applied directly to the skin.

    “Replay’s HSV gene therapy platform holds significant promise for patients with rare genetic skin diseases, and realizing its full potential requires focused expertise in medical dermatology—an area where LEO Pharma brings decades of leadership, scale and proven execution,” LEO Pharma CEO Christophe Bourdon said. “The acquisition aligns with our strategy of investing in the most impactful opportunities in dermatology and positions LEO Pharma at the forefront of next‑generation gene therapy.” 7

    LEO Pharma agreed to acquire Replay after identifying Replay as a high‑potential opportunity using its artificial intelligence (AI) scouting platform, Innoviewer™. Replay’s lead pipeline drug program is a preclinical phase candidate designed to treat dystrophic epidermolysis bullosa (DEB).

    MeiraGTx buys rights to XLRP treatment from J&J for $25M upfront

    MeiraGTx Holdings has agreed to acquire from Johnson & Johnson (J&J) all interests in botaretigene sparoparvovec (bota-vec), a gene therapy being developed to treat X-linked retinitis pigmentosa (XLRP).

    Under the companies’ asset purchase agreement, MeiraGTx agreed to pay J&J $25 million cash upfront, a one-time regulatory and commercial milestone payment tied to U.S. approval and U.S. sales performance of bota-vec for the treatment of XLRP, plus what MeiraGTx called a high double-digit royalty on global net sales starting in mid-2029.

    The sale comes nearly a year after bota-vec failed the 95-patient, Phase III LUMEOS trial (NCT04671433)  by missing the study’s primary endpoint of demonstrating statistically significant vision-guided mobility in patients with XLRP, as measured by a Visual Mobility Assessment (VMA) or maze.

    However, MeiraGTx has emphasized results showing that subjects treated with bota-vec were 2.4x more likely to respond than untreated subjects. A Low Luminance Questionnaire – Patient-Reported Outcome (LLQ PRO) showed significant benefit in mobility and dim light function, qualities tested by the VMA—thus indication, according to MeiraGTx, that the maze was not sensitive enough to capture these benefits.

    The company characterized data from the LUMEOS trial’s secondary endpoints as very strong, with clinically meaningful and statistically significant improvements shown in each of three domains of vision.

    MeiraGTx is the commercial manufacturer of bota-vec and had collaborated in its development with J&J from Phase I development onward. The FDA has granted Fast Track and Orphan Drug Designations to bota-vec, while the European Medicines Agency has granted Priority Medicines (PRIME), Advanced Therapy Medicinal Product (ATMP), and Orphan Drug designations to bota-vec.

    “This is a unique opportunity to gain an asset at this stage in development with data supporting a meaningful benefit in patients with no alternative treatment, many of whom are waiting for this life changing therapy and hoping for expeditious approval,” said Alexandria Forbes, PhD, MeiraGTX’s president and CEO. 8

    She added that MeiraGTx intends to start filing a Biologics License Agreement (BLA) with the FDA and applications for regulatory approval in the European Union and Japan as soon as possible.

     

    References

    1. Regeneron Pharmaceuticals. Otarmeni™ (lunsotogene parvec-cwha) Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss; Regeneron to Provide Otarmeni for Free in the U.S. April 23, 2026. (Last accessed May 1, 2026)

    2. Intellia Therapeutics. Intellia Therapeutics Reports Positive Phase 3 Results in Hereditary Angioedema, Marking a Global First for In Vivo Gene Editing. April 27, 2026. (Last accessed May 1, 2026).

    3. Genetic Engineering & Biotechnology News. Genomics Pioneer and Life Sciences Entrepreneur J. Craig Venter Dies at 79. April 30, 2026. (Last accessed May 1, 2026)

    4. Craig Venter Institute. J. Craig Venter, genomics pioneer and founder of JCVI and Diploid Genomics, Inc., dies at 79. April 29, 2026. (Last accessed May 1, 2026)

    5. Rocket Pharmaceuticals. Rocket Pharmaceuticals Announces $180 Million Sale of Priority Review Voucher. April 28, 2026. (Last accessed May 3, 2026)

    6. Passage Bio. Form 8-K, filed April 28, 2026. (Last accessed May 5,2026)

    7. LEO Pharma. LEO Pharma bolsters rare skin disease focus through acquisition of Replay gene therapy platform. April 30, 2026. (Last accessed May 3, 2026)

    8. MeiraGTx Holdings. MeiraGTx Announces the Acquisition of Botaretigene Sparoparvovec (bota-vec) for the Treatment of X-linked Retinitis Pigmentosa (XLRP). April 16, 2026. (Last accessed May 3, 2026)

    The post Gene Therapy Briefs: Regeneron Wins FDA Approval for First Neurosensory Gene Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ParcelBio Unveils Programmable mRNA Platform Backed by $13M Financing https://edusehat.com/en/parcelbio-unveils-programmable-mrna-platform-backed-by-13m-financing https://edusehat.com/en/parcelbio-unveils-programmable-mrna-platform-backed-by-13m-financing Fri, 08 May 2026 23:25:09 +0700 Edusehat ParcelBio, Unveils, Programmable, mRNA, Platform, Backed, 13M, Financing Biotechnology company ParcelBio said this week that it has raised $13 million in a seed financing round led by Breyer Capital with participation from General Catalyst, Y Combinator, Metaplanet, SurgePoint Capital, ZAKA VC, and other investors. The financing will support the development of the company’s proprietary Amplified and Prolonged EXpression mRNA (APEXm™) platform and advance its pipeline including an in vivo CAR T program for autoimmune disease, as well as additional programs in oncology and encoded protein therapeutics.

    The company, which is developing what it describes as a new class of durable mRNA medicines, will debut APEXm and share some preclinical data at the American Society of Gene and Cell Therapy annual meeting. This year’s meeting is being held in Boston, Massachusetts and will run from May 11-15. The company claims that its data will demonstrate that ParcelBio’s APEXm RNA drives significantly higher and more durable protein expression compared to another clinical mRNA design, and yields more complete target cell depletion in in vivo CAR T models. 

    “mRNA has transformed medicine, but today’s technologies are fundamentally limited in how much protein they can produce and for how long,” said David Weinberg, PhD, chief executive officer and co-founder of ParcelBio. His company’s proprietary technology addresses this problem by engineering RNA molecules to recruit the cell’s native RNA-stabilizing machinery, which enables higher and more durable protein expression. The company claims that its approach will result in medicines that reach thresholds that have historically been challenging for mRNA-based therapeutics. “We engineered RNA to work with the cell’s machinery rather than against it, enabling meaningful improvements in both expression and durability that we believe are essential for true disease modification,” Weinberg said. 

    Furthermore, ParcelBio’s platform maintains a simple, linear RNA architecture unlike circular RNA and other approaches, whose structure introduces manufacturing complexity or reduces output. Its broad applicability across proteins and cell types makes it suited for various therapeutic applications including immune programming and protein replacement. 

    “Most RNA platforms force a tradeoff between potency, durability, and manufacturability,” said Chris Carlson, PhD, chief scientific officer and co-founder of ParcelBio. “Our approach eliminates that tradeoff, enabling both higher peak expression and longer duration within a manufacturable system, and opening the door to entirely new classes of medicines.”

    ParcelBio’s lead program focuses on in vivo CAR T therapies that target pathogenic B cells across autoimmune diseases, with the goal of achieving deep B-cell depletion for durable, drug-free remission. By enabling sustained CAR expression without viral delivery or ex vivo manufacturing, the company aims to develop scalable, off-the-shelf therapies. Additional programs leveraging the technology are currently in development in oncology and encoded protein therapeutics.

    The post ParcelBio Unveils Programmable mRNA Platform Backed by $13M Financing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ASGCT CEO David Barrett Previews the Upcoming Conference in Boston https://edusehat.com/en/asgct-ceo-david-barrett-previews-the-upcoming-conference-in-boston https://edusehat.com/en/asgct-ceo-david-barrett-previews-the-upcoming-conference-in-boston Fri, 08 May 2026 23:25:08 +0700 Edusehat ASGCT, CEO, David, Barrett, Previews, the, Upcoming, Conference, Boston

    The 29th American Society of Gene & Cell Therapy (ASGCT) meeting kicks off in Boston next week. The annual event will be a whirlwind of sessions, keynotes, fireside chats, posters, and exhibitors.

    For the second year in a row, GEN spoke with David Barrett, JD, who has been the CEO of ASGCT since 2016. In this interview, we discuss his perspective on the event, if there is anything new that attendees should be looking out for, and what he, personally, is most looking forward to.

    This interview has been edited for length and clarity.

    LeMieux: The ASGCT meeting is an annual event. What are some of the things that will make this year’s meeting special?

    Barrett: There is a lot that is special this year. First and foremost, it feels like a bit of a homecoming which is really exciting. The last time we were in Boston was in 2008. And Boston is a city and community where gene therapy, biotech, and research are all located. You can feel it when you’re in Cambridge and I think you are absolutely going to feel that when you’re inside the convention center.

    The fact that the meeting is in Boston this year is also special for me because one of the very first things I did when I joined ASGCT in 2016, was to source the location for the 2020 annual meeting at the Hynes Convention Center in Boston. I was very excited and it was the first time we were going to take up an entire convention center. But that meeting, of course, did not happen; it had to be canceled because of COVID. So that makes this meeting in Boston particularly special. We finally get to have the meeting in Boston that I’ve been hoping for since 2016!

    And we are growing. We are at the bigger of the two convention centers in Boston. We are going to surpass the total number of people that we had last year and I have every expectation that we’ll see significant growth year over year.

    As far as other things that are that are new and interesting this year… I said this last year, but it’s worth adding it again—the science is always different. It is very consistent that we will have great science every year, and it is a wonderfully fun question mark of what exactly that science is going to look like. It’s always exciting because the science is always different year after year. So, by its very nature, it will be an exciting new conference this year.

    Also, we’ll have a puppy park in the exhibit hall, so that’s really fun!

    LeMieux: What are some things that will be highlighted at the meeting that ASGCT has been working on over the past year?

    Barrett: ASGCT has done a lot this year. There is a lot that we have been very vocal about so far, and there is a lot that we’ll be sharing during the annual meeting.

    Number one is that we partnered with Orphan Therapeutics Accelerator (OTXL) to found CGTxchange—the first and only clearing house and marketplace of its kind for cell and gene therapy assets. It is being built as we speak and we’ll have some exciting announcements during the annual meeting about assets that will hopefully be in the CGTxchange by that point. It is the culmination of a lot of work on what to do about commercially pre-viable (not non-viable) cell and gene therapies and the work that we’re doing to make those more commercially possible.

    Also, ASGCT is hosting its Momentum Gala—the first formal gala at our annual meeting. That event has resonated really well with sponsors and donors. In fact, it is sold out! That event is going to be used to celebrate the launch of ASGCT Foundation, which is a separately incorporated 501C3 charitable foundation to support ASGCT’s mission to advance early career researchers and enable the development of cell and gene therapies. Also at the gala, we’ll be announcing some new initiatives to support patient access and reduce barriers to diagnosis, clinical trial participation, and treatment with cell and gene therapies.

    Another major thing that’s going on is a considerable expansion of our educational activities. We recently launched a new e-learning tool and platform—the ASGCT Learning Center—a really fun project that we’ve been working on to expand how we we are getting new content to our new and expanding audiences.

    We recognize that we have a really broad audience at ASGCT that is made up of cell and gene therapy basic science researchers, translational researchers, physician scientists and others in the ecosystem of drug development and administration for cell and gene therapies. And we’re looking at new ways to provide content that can help satisfy the learning needs of that really broad audience. The learning center is a big tool in our quiver to be able to do that.

    LeMieux: What do you hope people take away from the meeting?

    Barrett: I hope they take away a couple of things… number one, I hope they take knowledge, education, and awareness of what’s going on in the space and what has been happening over the course of the last 12 months. I hope that they take that back to their individual place of work. And I hope that, generally speaking, we fulfill our mission by expanding that knowledge base among all of the stakeholders in cell and gene therapy. Another thing that I hope people take away from this is that, after a lot of ups and downs and undulations in this field over the course of the past two to three years, that there is an extraordinary sense of excitement about the next phases in the development of cell and gene therapy drugs.

    We have some really exciting new regulatory pathways. We have a lot of development of personalized gene editing technologies and techniques that can bring gene therapies much more quickly and effectively to patients who need them. We have seen significant advancements in more traditional or classic AAV gene therapies that are allowing these to be safer and more efficacious. And we’re seeing an expansion of cell-based gene therapies through an ever-expanding portfolio of indications that are reached by CAR Ts, primarily in cancer, but in an expanding outlook for the use of CAR Ts outside of cancer as well. So, I am hopeful that attendees come away with a renewed energy and vigor for the development of satellite gene therapies.

    LeMieux: Is there anything specific planned at the meeting to touch on the concerns of the challenges that the scientific community is facing right now—with funding or other barriers?

    Barrett: We are very excited to have Katherine Szarama, PhD—who was recently named acting director of FDA’s Center for Biologics Evaluation and Research (CBER)—participating in a fireside chat, addressing regulatory uncertainties. [Szarama replaced Vinay Prasad, MD, MPH, on May 1st.]

    We have two other fireside chats focused on regulation, as well. The three fireside chats will offer attendees an opportunity to learn a little more, ask some questions, and hear from some of the individuals in those sessions specifically.

    But I think that people will also see, more broadly, the ongoing work that ASGCT is doing to continue to create a partnership and a positive working relationship with the FDA to support those regulatory concerns.

    LeMieux: What are you most looking forward to?

    Barrett: I think I said this last year, but it really is one of my favorite components of the annual meeting. Every year, I look forward to taking some time to watch the exhibit hall being built. When the rope drops and people enter the exhibit hall for that very first reception, the hall is in pristine condition. And one of my favorite parts is watching it get to that pristine condition because it is just so exciting to see everything being built and come to a head, to have the whole field enter all at one space, and to be able to see an industry live and in person. Because so much of what we do is at our computer screens—and what we read about, hear about, or listen to people talk about. But when you actually see the field of gene and cell therapy on display, it is really exciting and satisfying.

    Lastly, I will add that I’m looking forward to eating too much clam chowder while in Boston (chuckling).

    The post ASGCT CEO David Barrett Previews the Upcoming Conference in Boston appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     What is PDUFA—and why does it matter for biotech innovators, FDA &amp; patients? https://edusehat.com/en/what-is-pdufaand-why-does-it-matter-for-biotech-innovators-fda-patients https://edusehat.com/en/what-is-pdufaand-why-does-it-matter-for-biotech-innovators-fda-patients Fri, 08 May 2026 19:55:04 +0700 Edusehat What, PDUFA—and, why, does, matter, for, biotech, innovators, FDA, patients Key takeaway: PDUFA enables the FDA to collect user fees from the biopharmaceutical industry, allowing for efficient and more predictable reviews. This funding model provides essential stability for biotech companies of all sizes and ensures the FDA maintains its status as the global leader in bringing innovative new treatments to patients.

    Next year, Congress will reauthorize the Prescription Drug User Fee Act (PDUFA)—a little-known but essential law that allows the U.S. Food and Drug Administration (FDA) to collect fees from companies to support its review of new medicines. This law has provided critical stability for the agency and for the innovative companies navigating the review process. And it has enabled rigorous and efficient review of new treatments, bringing breakthroughs to patients sooner and bolstering U.S. biotech leadership.

    Below is a primer on what PDUFA is, how it works, and why its user fee model is so important — for patients, biotech innovators, and the U.S. FDA.

    What is PDUFA?

    The Prescription Drug User Fee Act empowers the FDA to collect user fees from companies when they submit new drug applications and program fees (as outlined below).

    PDUFA was enacted in 1992 to address the fact that drug reviews in the U.S. were slow, unpredictable, and under-resourced. Since then, it has transformed the U.S. drug approval process into a world-class system that brings new medicines to Americans before most other countries.

    How does PDUFA work?

    Under PDUFA, companies pay two types of user fees to the FDA: an application fee for each New Drug Application or Biologics License Application they submit, and a program fee for each approved application they already hold. Roughly 20% of user fee revenue comes from application fees and 80% from program fees, meaning most funding stems from successful, marketed drugs.

    In return, the FDA commits to specific review timelines, meeting schedules, and process improvements. This creates a stable, predictable structure for how drugs are reviewed, which is vitally important for the biotech industry that develops the majority of new medicines. Regulatory predictability and transparency help companies secure the investment needed to fund development, plan more effectively, and avoid costly delays that can be devastating for companies with limited resources—and for the patients awaiting treatments.

    Congress reauthorizes PDUFA every five years, giving the FDA an opportunity to work with biotech innovators, patients, and other stakeholders to optimize the review process and identify the necessary resources.

    What do PDUFA user fees fund?

    Once collected, PDUFA fees fund the FDA’s human drug review program.

    User fees support a wide range of FDA activities essential to bringing innovative treatments to patients, such as reviewing investigational new drugs and applications, inspecting manufacturing facilities and product standards, and conducting studies of drugs already on the market. It is important to note that user fees are independent of review outcomes. The fee paid to submit an application has no bearing on the regulatory outcome of the review.

    PDUFA does not support the review of generic or over-the-counter drugs not associated with a New Drug Application, research unrelated to human drug review, or advertising review of marketed products.

    How does PDUFA benefit patients?

    PDUFA has dramatically improved the speed and consistency of the drug review process, ensuring patients get access to innovative medicines sooner.

    Before PDUFA became law, new drug approvals often took more than two years. Today, the FDA aims to complete standard reviews in about 10 months, and priority reviews in roughly 6 months.

    The law has also made the United States a leader in approving new medicines. Before it was enacted, patients in other countries often gained access to new medicines before Americans. Today, over half of new drugs are approved first in the United States.

    Finally, the law has enabled the FDA to maintain high standards while bringing safe and effective treatments to patients. Stable user fee revenue allows the agency to staff expert reviewers to keep pace with innovation, implement cutting-edge data systems, and conduct ongoing safety monitoring.

    What’s the bottom line?

    PDUFA is the backbone of the modern drug review system.

    It helps create the conditions for new drugs to be developed by providing the stability biotech companies need to turn scientific breakthroughs into real treatments. It also ensures the FDA has the resources to keep pace with innovation and review new drugs efficiently while maintaining rigorous, independent standards.

    Put simply, PDUFA provides patients with access to innovative treatments. Without it, the FDA would be less predictable, slower, and have fewer resources—resulting in fewer new therapies reaching patients.

    The post What is PDUFA—and why does it matter for biotech innovators, FDA & patients? appeared first on Bio.News.

    ]]>     Here’s how technology transformed babymaking https://edusehat.com/en/heres-how-technology-transformed-babymaking https://edusehat.com/en/heres-how-technology-transformed-babymaking Fri, 08 May 2026 19:55:02 +0700 Edusehat Here’s, how, technology, transformed, babymaking Technology is changing the way we make babies. The pioneering work of the scientists who invented IVF led to the birth of the first “test tube baby” in 1978. We’ve come a long, long way since then.

    This week, I’ve been working on a piece about the cutting edge of IVF technologies and what’s coming next. Think AI and robots and, potentially, gene-edited embryos.

    My reporting has also made me think about just how much progress has been made in the last five decades. Clinicians have improved hormonal treatments. Embryologists have devised ways to culture embryos in the lab for longer. IVF clinics today offer multiple genetic tests for embryos.

    In recent years, we’ve had reports of babies born with DNA from three people, babies born following “IVF on wheels,” babies born from decades-old embryos, and even babies “conceived” with the aid of a sperm-injecting robot.

    The technology has also had a huge social impact. It has allowed for changes in the structure of families and provided more reproductive choices for would-be parents. So this week, let’s consider the technologies that have transformed babymaking.

    Alan Penzias, a reproductive endocrinologist at Boston IVF, has been working in IVF since the early 1990s. In those days, his lab at Yale would collect a person’s eggs, fertilize them, and culture any resulting embryos for two days, until the embryos had two or four cells.

    The embryos couldn’t survive any longer outside a body, so they’d be transferred to the uterus at that point. All of them. Even if there were, say, five embryos in total. Typical healthy patients could expect a live birth rate of 12% to 15%, he says.

    Then Penzias heard that other teams were managing to culture embryos for three days. “We thought, No, that’s not possible,” he recalls. He learned that scientists had achieved this by tinkering with the culture medium—the nutrient-rich fluid the embryos are grown in.

    Those three-day embryos, which had around six to 10 cells, seemed to have a better chance of resulting in a live birth. The teams culturing embryos for longer saw their success rates climb to 25% among similar patient groups, says Penzias. Again, he couldn’t believe it. “We thought they were making it up,” he says.

    In the years since, teams have made more improvements to culture medium. Today, most IVF embryos are cultured for five or six days—a point at which they have 80 to 100 cells. The culturing process can act a little like a stress test—the embryos that make it to day six are generally more likely to go all the way and develop into a healthy baby.

    Over the same period, advances in other technologies have opened up the options for what we can do with those embryos. Scientists learned they were able to freeze embryos and use them at a later date. A little over a decade ago, clinics shifted to a “vitrification” approach that rapidly cools the embryos to a glassy state. Vitrified embryos are more likely to survive freezing and thawing, so this approach quickly caught on.

    As a result, doctors no longer needed to transfer multiple embryos at once. This made it less likely that patients would have twins or triplets, which can increase the risk of pregnancy complications.

    Vitrification has also made IVF safer in other ways, including by affording patients a bit of time between fertility treatments. The hormonal treatments used in the first phase of IVF are designed to increase the production of mature eggs that can be collected. These treatments carry a small risk of a condition called ovarian hyperstimulation syndrome (OHSS), which in rare cases can be life-threatening. The ability to freeze all your embryos and use them at a later date is thought to give the body a chance to recover from hormonal treatment and reduces the risk of OHSS.

    And because clinics are now able to culture embryos for up to a week, they can take a few of the 100 or so cells and send them for genetic testing before freezing the embryos. People undergoing IVF can get genetic readouts of all the embryos before deciding which to implant. (It is worth noting, however, that these testing technologies are not perfect.)

    “Those are really radical changes, and we take them for granted,” says Penzias.

    These technologies have also changed the function of IVF. What was once a treatment for infertility is now used to preserve fertility. People who want to delay parenthood can opt to freeze their eggs or embryos and use them later. They might opt to transfer one embryo in a year’s time and a second several years later. “We’ve been able to empower women to be able to have much more reproductive choice and get more reproductive mileage from a single IVF cycle,” says Penzias.

    People who are about to undergo cancer treatments that might damage the testes or ovaries can opt to store their eggs or sperm ahead of time, too. Scientists have even been able to preserve pieces of ovarian and testicular tissue and reimplant them later, enabling recipients to have healthy babies.

    Today, more people than ever have access to safe IVF options that offer multiple paths to parenthood. Those options look set to expand. But if you want to find out more about the AI and IVF robots, you’ll have to read this week’s story, here!

    This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.

    ]]>     G-Link CAR-T Delivery Platform Showcased at ASGCT https://edusehat.com/en/g-link-car-t-delivery-platform-showcased-at-asgct https://edusehat.com/en/g-link-car-t-delivery-platform-showcased-at-asgct Fri, 08 May 2026 08:50:12 +0700 Edusehat G-Link, CAR-T, Delivery, Platform, Showcased, ASGCT Vyriad reports that it will showcase its latest CAR T delivery technology platform, G-Link, through a schedule of presentations, technical sessions, and exhibition activities at ASGCT. The modular plug-and-play protein adapter developed in collaboration with Menachem Rubenstein, PhD, of the Weizmann Institute allows drug developers to cap and retarget existing lentiviral vectors for in vivo delivery, according to the company.

    By leveraging G-Link, wild-type lentiviral vectors can be reprogrammed for in vivo applications without the need for intensive vector re-engineering, effectively shortening development timelines for next-generation CAR T and other cell therapies, notes a company spokesperson, who adds that G-Link can also be used to simplify ex vivo CAR T manufacturing and significantly improve T cell transduction efficiency without redesigning vectors.

    “I believe that G-Link can address some of the most persistent challenges in in vivo delivery and we are excited to unveil it at ASGCT this year,” says Stephen Russell, PhD, CEO of Vyriad. “Our participation this year underscores our clear mission: to replace complex, weeks-long manufacturing cycles with precise, off-the-shelf immunotherapies. With G-Link, we aim to foster collaborations that will define the next generation of in vivo cell therapies.”

    Vyriad’s VV169 in vivo CAR T program will progress into clinical development later this year, while the G-Link platform will advance towards clinical translation later in the future, continues Russell.

     

    The post G-Link CAR-T Delivery Platform Showcased at ASGCT appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     From Sequence to Patient in Under 12 Months: A Case Study in Advancing Complex Cancer Immunotherapies https://edusehat.com/en/from-sequence-to-patient-in-under-12-months-a-case-study-in-advancing-complex-cancer-immunotherapies https://edusehat.com/en/from-sequence-to-patient-in-under-12-months-a-case-study-in-advancing-complex-cancer-immunotherapies Fri, 08 May 2026 08:50:11 +0700 Edusehat From, Sequence, Patient, Under, Months:, Case, Study, Advancing, Complex, Cancer, Immunotherapies

    Image of Joseph Shultz

    Joseph Shultz

    Vice President of Technical Development and Manufacturing
    Ottimo Pharma

    Panelist

    Image of Joseph Shultz

    Joseph Shultz

    Joseph Shultz is the vice president of technical development and manufacturing at Ottimo Pharma. His more than 30 years in the industry span development, manufacturing, quality, and technology development. He has held influential positions at Amgen, Novartis Pharma, the Battelle Memorial Institute, Evelo Biosciences, and Resilience. He initiated the technologies and led the strategies that resulted in next-generation biomanufacturing plants at both Amgen and Novartis.
    Image of Imroz Ghangas

    Imroz Ghangas

    Vice President of Commercial Sales
    Asimov

    Panelist

    Image of Imroz Ghangas

    Imroz Ghangas

    Imroz Ghangas and his team drive partnerships to advance Asimov’s genetic design platform and AI capabilities. With over 25 years in biotech, Imroz has evolved from process development scientist to commercial leader, bridging technical innovation with scalable solutions. His expertise spans bioprocess development and platform integration, with deep knowledge of biomanufacturing workflows from gene to drug product. He leverages his technical foundation to accelerate the adoption of next-generation bioprocessing technologies.

    Broadcast Date: 
    • Time: 

    Complex biologics such as bifunctional antibodies are opening new therapeutic possibilities in oncology, but these molecules present significant challenges for manufacturing teams. Non-standard architectures can often translate to low expression and difficult developability, making cell line development a critical bottleneck between a promising sequence and a viable clinical candidate.


    In this GEN webinar, Joseph Shultz (vice president of technical development and manufacturing, Ottimo Pharma) and Imroz Ghangas (vice president of commercial sales, Asimov) discuss strategies for achieving high-performing clonal titers and advancing a dual-paratopic cancer immunotherapy from sequence to dosed patient in under a year. Attendees will learn about the unique attributes of Ottimo’s molecule and how a specialist partnership with Asimov accelerated the program. The presenters will also introduce the CHO Edge System, which combines Asimov’s proprietary GS knock-out CHO host, hyperactive transposase, library of characterized genetic elements, and AI-driven genetic design tools to routinely deliver clonal titers of 8-12 g/L.

    A live Q&A session will follow the presentation offering you a chance to pose questions to our expert panelists.

    Produced with support from:

    asimov logo

    The post From Sequence to Patient in Under 12 Months: A Case Study in Advancing Complex Cancer Immunotherapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Macrophages Use Cell Volume Changes to Sense Danger and Amplify Inflammation https://edusehat.com/en/macrophages-use-cell-volume-changes-to-sense-danger-and-amplify-inflammation https://edusehat.com/en/macrophages-use-cell-volume-changes-to-sense-danger-and-amplify-inflammation Fri, 08 May 2026 08:50:08 +0700 Edusehat Macrophages, Use, Cell, Volume, Changes, Sense, Danger, and, Amplify, Inflammation Macrophages are often described as the immune system’s first responders, but new work suggests they are also remarkably attuned to the physical state of their environment. A study published in the Journal of Cell Biology titled “Disruption of macrophage cell volume drives inflammatory responses and type I interferon signaling” reveals that shifts in cell volume act as a previously underappreciated danger signal—one that can rewire macrophage gene expression, heighten antiviral defenses, and intensify inflammatory responses.

    The research, led by Jack Green, PhD, and colleagues at the University of Manchester, centers on the Volume‑Regulated Anion Channel (VRAC), a protein complex that helps cells maintain osmotic balance. When VRAC is missing, macrophages lose the ability to correct swelling under hypo‑osmotic stress. “Cell volume disruption induced type I interferon signaling through a DNA- and TBK1-dependent mechanism, but independent of cGAS and 2′3′-cGAMP transport,” the authors wrote. That loss of control, the team found, is far more consequential than a simple biophysical hiccup. It fundamentally alters how macrophages interpret threats.

    Green noted that although earlier studies hinted at a connection between cell volume and inflammatory signaling, the underlying biology remained murky. “Despite the reported indications that cell volume and VRAC are involved in inflammatory signaling, the basic biological mechanisms of how the regulation of cell volume shapes inflammation were unknown,” he said. To probe that gap, the team examined VRAC‑deficient macrophages exposed to mild osmotic stress.

    The swelling triggered broad reprogramming of gene expression, including the induction of antiviral and proinflammatory pathways. Many of the most strongly upregulated genes belonged to type I interferon signaling cascades or nucleic acid–sensing systems. First author James Cook frames the finding succinctly: “Together, these findings suggest that cell volume acts as an additional layer of danger sensing in macrophages that shapes and tunes the nature of immune responses to pathogens.”

    That prediction held up in functional assays. When challenged with Influenza A virus, VRAC‑deficient macrophages mounted a more potent antiviral response than their wild‑type counterparts. The heightened sensitivity extended beyond viral infection. In mouse models of systemic hyperinflammation, animals lacking VRAC showed elevated levels of a key inflammatory mediator, indicating that dysregulated cell volume can exacerbate cytokine‑driven pathology in vivo.

    Rather than responding solely to biochemical cues, these cells appear to fold physical perturbations—such as osmotic imbalance—into their danger‑sensing logic. Green argued that this perspective may help explain why inflammatory diseases can escalate unpredictably when tissue conditions shift. “Understanding disruptions in the tissue microenvironment leading to alterations in cell volume is therefore an important consideration in our understanding of inflammation and disease pathogenesis,” he concluded, adding that “future studies will reveal the potential for regulating VRAC‑dependent cell volume changes in macrophages in disease.”

    The post Macrophages Use Cell Volume Changes to Sense Danger and Amplify Inflammation appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cellular Origins Collaborates with Immatics on Automation for Cell Therapy Manufacturing https://edusehat.com/en/cellular-origins-collaborates-with-immatics-on-automation-for-cell-therapy-manufacturing https://edusehat.com/en/cellular-origins-collaborates-with-immatics-on-automation-for-cell-therapy-manufacturing Fri, 08 May 2026 05:15:13 +0700 Edusehat Cellular, Origins, Collaborates, with, Immatics, Automation, for, Cell, Therapy, Manufacturing Cellular Origins agreed to collaborate with Immatics in utilizing Cellular Origins’ automated mobile robotic platform, Constellation®, within certain parts of the company’s manufacturing processes. The collaboration will explore how automation technologies can further contribute to more efficient and scalable manufacturing processes for next-generation cell therapies.

    “Following the first cell therapy approvals in 2017, there has been widespread success in treating blood cancers, while progress in solid tumors has been more limited. Immatics is now working to advance clinically validated approaches that could expand treatment options for these,” said Edwin Stone, PhD, CEO, Cellular Origins. “Current manual manufacturing methods can limit the number of patients who are able to access approved therapies,” he added.

    “Effective cell therapies for solid-tumor patients is one of the most exciting developments in our field but will need the manufacturing challenges to be addressed to deliver on its potential. Our partnership with Immatics aims to support the scalable and cost-effective manufacturing of their therapies so that more patients could potentially benefit.”

    “Immatics has generated extensive data demonstrating the potential of precision targeting PRAME, a target expressed in more than 50 cancers,” noted Ali Mohamed, PhD, senior vice president of CMC, Immatics. “As we continue to advance our programs, it is important that we also develop manufacturing capabilities that can support future scale. We are pleased to collaborate with Cellular Origins to explore how the Constellation platform and our integrated manufacturing processes could support the scalable production of our therapies as we move toward potential approvals.”

     

     

    The post Cellular Origins Collaborates with Immatics on Automation for Cell Therapy Manufacturing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Digital PCR Playbook: Applications and Challenges Across Research and Clinical Labs https://edusehat.com/en/digital-pcr-playbook-applications-and-challenges-across-research-and-clinical-labs https://edusehat.com/en/digital-pcr-playbook-applications-and-challenges-across-research-and-clinical-labs Fri, 08 May 2026 05:15:12 +0700 Edusehat Digital, PCR, Playbook:, Applications, and, Challenges, Across, Research, and, Clinical, Labs

    Image of Alex Zevin, PhD

    Alex Zevin, PhD

    Director, Genomics Shared Resource
    Fred Hutchinson Cancer Center

    Panelist

    Image of Alex Zevin, PhD

    Alex Zevin, PhD

    Alex Zevin, PhD, began serving as the director of the Genomics Shared Resource at Fred Hutch in December 2022. Before that, he was a research scientist at ArcherDX where he developed NGS in vitro diagnostic devices including several clinical trial assays and an approved companion diagnostic. He also previously worked at InBios International and developed a rapid test for detection of anthrax.

    Zevin has a bachelor’s degree in biochemistry from Colorado State University and a PhD in molecular biology from Arizona State University where he developed methods to characterize bacterial communities in engineered systems and conducted postdoctoral research at the University of Washington studying host-microbe interactions in non-human primate models.

    Broadcast Date: 
    • Time: 

    Digital PCR has emerged as a powerful approach for precise nucleic acid quantification, but it is constrained by limited dynamic range and the difficulty of multiplexing. Newer platforms, like Countable Labs’ single-molecule counting PCR, address both by offering precise quantification across a broad range of target abundances while simplifying multiplexing through single-molecule isolation and fluorescent imaging across millions of spatially fixed compartments.

    In this GEN webinar, Alex Zevin, PhD, director of Fred Hutchinson Cancer Center’s Genomics Shared Resource, draws on hands-on experience with managing a suite of nucleic acid quantification technologies, including standard qPCR, digital droplet PCR, and Countable PCR, to share practical guidance for labs considering or expanding their PCR quantification capabilities. Key insights from the webinar include:

    • How single-molecule counting differs from conventional digital PCR—and the sensitivity, precision, and multiplexing advantages it enables

    • Real-world applications suited to single-molecule counting PCR, including validating NGS results, replacing or supplementing existing assays, and generating clinically actionable data

    • Common challenges for converting qPCR and dPCR assays to single-molecule counting PCR, and how to overcome them

    A live Q&A session will follow the presentations offering you a chance to pose questions to our expert panelist.

    Produced with support from:

    Countable Labs logo

    The post Digital PCR Playbook: Applications and Challenges Across Research and Clinical Labs appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Oral Small-Molecule GLP-1s Linked to Deep Brain Activity and Reduced Cravings in Mice https://edusehat.com/en/oral-small-molecule-glp-1s-linked-to-deep-brain-activity-and-reduced-cravings-in-mice https://edusehat.com/en/oral-small-molecule-glp-1s-linked-to-deep-brain-activity-and-reduced-cravings-in-mice Fri, 08 May 2026 05:15:11 +0700 Edusehat Oral, Small-Molecule, GLP-1s, Linked, Deep, Brain, Activity, and, Reduced, Cravings, Mice Interest in glucagon-like peptide 1 receptor agonists (GLP-1s) continues to surge due to their effectiveness in reducing body weight and improving metabolic outcomes. This includes interest in small molecule oral GLP-1s which are more bioavailable and more easily manufactured than their injectable counterparts.

    Now data from a new study in mice performed by scientists at the University of Virginia shows that this emerging class of weight-loss drugs suppress hedonic eating by modulating a reward circuit deep in the brain that is separate from previously described mechanisms that broadly affect appetite. The scientists believe that this pathway could be an avenue by which GLP-1s treat other dysfunctions in reward processing such as substance use disorders.

    Details of the National Institutes of Health-funded study were published this week in a Nature paper titled “A brain reward circuit inhibited by next-generation weight-loss drugs in mice.” In it, the team reported that they investigated the small-molecule GLP-1s including Eli Lilly’s recently approved drug orforglipron, also known by the brand name Foundayo, as well as danuglipron, an oral GLP-1 that was being developed by Pfizer until the company decided to discontinue its development in 2025. 

    Previous studies that explored the effects of larger peptide GLP-1s such as semaglutide in the brain have found that they suppress hunger-driven eating by engaging networks in the hypothalamus and hindbrain. What has been less clear is the mechanism by which small-molecule GLP-1s work. “As the accessibility of these medications continues to rise and patient uptake increases, it’s crucial that we understand the neural mechanisms underlying the effects we’re seeing,” said Lorenzo Leggio, MD, PhD, clinical director of NIH’s National Institute on Drug Abuse.

    The current study gets scientists one step closer to that goal. According to the paper, the scientists first used gene editing to modify the GLP-1 receptors of mice to make them more humanlike. They then administered orforglipron or danuglipron to the mice, and identified brain regions where the drugs induced activity. The results showed that in addition to inducing activity in familiar pathways, the drugs also triggered the central amygdala, a region associated with desire that is deeper in the brain than scientists previously thought GLP-1s could directly reach. Further testing showed that once activated, the central amygdala reduced the release of dopamine into key hubs of the brain’s reward circuitry during hedonic feeding. 

    “We’ve known that GLP-1 drugs suppress feeding behavior driven by energy demand,” said co-corresponding author Ali Guler, PhD, a professor of biology at the University of Virginia. “Now it seems oral small-molecule GLP-1s also dial back eating for pleasure by engaging a brain reward circuit.”

    Given the effect of these drugs on eating for pleasure, future studies could explore whether small-molecule GLP-1s can also suppress cravings for other addictive substances. It is a question that the team hopes to explore in follow up studies focused specifically on substance use disorder. 

    The post Oral Small-Molecule GLP-1s Linked to Deep Brain Activity and Reduced Cravings in Mice appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Human Antibodies Identified That Have Potential To Prevent and Treat Measles Virus https://edusehat.com/en/human-antibodies-identified-that-have-potential-to-prevent-and-treat-measles-virus https://edusehat.com/en/human-antibodies-identified-that-have-potential-to-prevent-and-treat-measles-virus Fri, 08 May 2026 05:15:10 +0700 Edusehat Human, Antibodies, Identified, That, Have, Potential, Prevent, and, Treat, Measles, Virus Scientists at La Jolla Institute for Immunology (LJI) say they are the first in the world to characterize human monoclonal antibodies (mAbs) capable of neutralizing measles virus (MeV). The antibodies, derived from the memory B immune cells of an individual who had previously received the MMR vaccine years previously, bind to key hemaggglutinin (H) and fusion (F) surface virus proteins, preventing viral entry into host cells.

    The researchers, headed by Erica Ollmann Saphire, PhD, LJI professor, president, and CEO, say the new panel of human antibodies may form the basis for future medical therapies against measles infection. In their newly reported study the team showed that an infusion of the antibodies resulted in 500-fold lower viral load in a rodent model of measles infection.

    “These antibodies work as prophylaxis—to protect from initial infection—and they work after viral exposure as a treatment to fight measles infection, said Saphire. “It may be possible to give someone an infusion of these antibodies and deliver the immune response they wish they had.”

    In their study (“Human neutralizing antibodies targeting the measles virus hemagglutinin and fusion surface proteins”) reported in Cell Host & Microbe, the team concluded “Characterization of these fully human mAbs provides avenues for prophylactic or therapeutic intervention against re-emerging MeV.”

    Measles virus is “… a highly transmissible paramyxovirus, can cause severe complications and death, particularly in infants and young children,” the authors wrote. “A live-attenuated vaccine derived from a genotype A MeV strain provides vaccinees with lifelong immunity and protective antibodies against all 24 MeV genotypes in circulation.”

    However, in recent years, decreased vaccination rates have led to deadly measles outbreaks across the U.S. and around the world. This sharp rise in measles cases is especially dangerous for the millions of people who cannot receive a measles vaccine. While the measles vaccine is incredibly safe and effective, it does contain a live, weakened virus. This means that people who have compromised immune systems, such as those who are pregnant or receiving chemotherapy, including children, cannot receive the vaccine. The very young are also at risk. Infants must wait until they are 12 months old to be vaccinated, and most children in the U.S. aren’t fully vaccinated against measles until they are six years of age.

    “There are a growing number of people that can’t be vaccinated or haven’t been fully vaccinated,” said Saphire. “The very same people who can’t be vaccinated or can’t be vaccinated yet, are the same people for whom a measles virus infection would be the most severe—or be lethal.”

    Until recently, enough people were vaccinated against measles virus that the risk of exposure for this unvaccinated group was very low. Unfortunately, that community protection—herd immunity, is no longer. LJI scientists are on a mission to find treatment options for the most vulnerable.

    There are currently no measles-specific therapies to help patients. The new study shows that monoclonal antibody therapies may may be a feasible option. Monoclonal antibody treatments contain many copies of a neutralizing antibody, and are widely used for a variety of infectious diseases. Even infants receive monoclonal antibody therapies each year to prevent respiratory syncytial virus (RSV).

    To design a monoclonal antibody treatment for measles, researchers need a clear picture of how human antibodies fight the virus. However, as they noted, “Despite the global presence of MeV and widespread use of the vaccine, few studies have mapped the human antibody response. We do not yet know how human antibodies, from either measles vaccination or natural infection, recognize and protect against the virus.”

    Saphire and her colleagues began by harnessing an imaging technique, cryo-electron microscopy (cryo-EM), to capture the first-ever glimpses of how antibodies bind to the measles virus. They started by examining mouse antibodies, and published that work in a recent paper. That initial study showed where measles virus is vulnerable to antibody attack. The mouse antibodies, the researchers showed, latched onto the virus fusion protein, to block viral entry into a cell.

    To find out whether human antibodies could do the same thing, the researchers analyzed blood from a clinical research volunteer. “We evaluated 15 MMR-vaccinated donors for their polyclonal MeV responses to identify individuals with vaccine-induced, protective, circulating antibodies,” they explained. The 56-year-old female volunteer they selected had been vaccinated against measles many years before, and already had antibodies ready to fight measles virus. This individual “… demonstrated the highest polyclonal response and the most H- and F-reactive memory B cells.”

    From the one blood sample, the LJI scientists isolated antibodies that bind to the measles virus fusion protein, along with other antibodies that bind to the virus hemagglutinin protein. They then captured 3D images of these antibodies bound together with the measles virus. “We found that these antibodies are exceptionally potent,” said study first author, LJI Instructor Dawid Zyla, PhD. “Two orders of magnitude better than comparable molecules reported at conferences.”

    Measles virus is a shape-shifting virus. When it meets a human cell, it unfolds to reveal viral machinery that fuses with the host cell membrane. The new study shows that antibodies targeting the fusion protein work by locking the protein in place, leaving the virus unable to shape shift and infect a host cell. The next step was to test these antibodies in a preclinical animal model. Study collaborators at The Ohio State University carried out key experiments in cotton rats. They found that all four lead antibodies reduced viral load when given either before measles exposure or within 24 to 48 hours after measles virus infection. One antibody, designated 3A12, which binds to a site on the F protein, rendered the circulating virus actually undetectable.

    While more work needs to be done, the researchers see these antibodies as promising tools in the fight against measles. Their new images of the antibody structures provide the materials needed to make the world’s first before- or after exposure treatment for measles virus. “Now we know what we’re aiming for, and we have the antibodies we need,” said Saphire.

    In their paper the authors stated, “The protective mAbs identified here target four distinct, non-competing epitopes, and may be combined as cocktail therapies to enhance treatment potency, maintain durable protection, and reduce the risk of viral escape.… these human mAbs themselves, which recognize conserved sites and inhibit measles by complementary mechanisms, represent a basis to develop a treatment that is urgently needed as measles virus infections surge globally.”

    The post Human Antibodies Identified That Have Potential To Prevent and Treat Measles Virus appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     What’s next for IVF https://edusehat.com/en/whats-next-for-ivf https://edusehat.com/en/whats-next-for-ivf Thu, 07 May 2026 22:10:02 +0700 Edusehat What’s, next, for, IVF
  • Helping embryos stick: Even healthy-looking embryos only implant 40–60% of the time. Researchers in Spain are trialing a device that physically injects embryos directly into the uterine lining at the press of a button.

  • AI and robots are taking over the lab: Automated systems can now select sperm, fertilize eggs, and culture embryos without human hands. At least 19 children have already been born through fully automated IVF.

  • Genetic testing is getting complicated: Standard embryo screening helps reduce miscarriage, but newer tests claiming to predict IQ or height are gaining ground in the US—and making many fertility doctors deeply uncomfortable.

  • Gene editing is quietly creeping back: Years after He Jiankui went to prison for editing human embryos, startups are revisiting CRISPR as a way to prevent serious inherited disease—raising hopes, and familiar fears about a slippery slope.
    • " data-chronoton-post-id="1136946" data-chronoton-expand-collapse="1" data-chronoton-analytics-enabled="1">

    MIT Technology Review’s What’s Next series looks across industries, trends, and technologies to give you a first look at the future. You can read the rest of them here.

    Forty-eight years ago this July, Louise Joy Brown became the world’s first person born with the help of in vitro fertilization. Millions more IVF babies have entered the world since then. And that’s partly thanks to advances in technology that have made IVF safer and more effective.

    But it’s still not perfect. The process can be slow, painful, and expensive—and that’s for the lucky people who are able to access it in the first place. And by at least one measure, IVF success rates have been declining in recent years.

    Reproduction is complex, and there’s a lot that embryologists and gynecologists still don’t know and can’t control. They don’t know why many healthy-looking embryos don’t “stick” in the uterus, for example. They don’t always have an explanation for why their patients can’t get pregnant. And they can’t always account for vast differences in IVF success rates between individuals and between fertility clinics.

    Scientists are working on all those questions and more. They’re wrestling with complex ethical questions about how new genetic tools will be used to analyze or even alter embryos. Meanwhile, technologies designed to standardize treatment, eliminate human error, boost success rates, and make IVF more accessible are already beginning to usher in a new era for assisted reproduction—one aided by AI and robots.

    1. Helping embryos stick

    Some of those technologies are being developed at the Carlos Simon Foundation in Valencia, Spain. When I visited in March, researchers gave me a tour of the labs and showed me a device that had been used to keep a human uterus alive outside the body for the first time.

    While some members of the team dream of building artificial uteruses that might one day be able to carry a fetus to term, they first want to use such devices to learn more about implantation—the moment at which a fertilized egg makes contact with the lining of the uterus, burrows inside, and essentially “hatches,” triggering the start of a pregnancy.

    Despite decades of advances in IVF, that process is still poorly understood. Even healthy-looking embryos stick no more than 40% to 60% of the time.

    In IVF techniques used today, clinics can create early-stage embryos and wait until the uterus is deemed most receptive, but once they insert the embryo into the uterus, it’s on its own. Xavier Santamaria, senior clinical scientist at the Carlos Simon Foundation, and his colleagues are trialing a different approach. They’ve developed a device that, at the press of a button, injects the embryo into the uterine lining.

    Scientists in Valencia showcase Transfer Direct.
    JESS HAMZELOU / MITTR

    In a demonstration I watched with a prototype, Santamaria picked up his speculum and turned to face the vaginal opening of his “patient,” which in this case was just a model of the real thing—a plastic bottom with labia, a vagina, a uterus, and ovaries, two short stumps representing what would normally be a pair of legs held in stirrups.

    He hunched over and peered inside. “Embryo,” he called. His colleague Maria Pardo, an embryologist, passed him a thin needle containing a mouse embryo she had recently collected from a petri dish.

    Santamaria’s device allows for the embryo-containing needle to be connected to a delivery tube. This tube also has a camera, a light, and a sensor that lets the doctor know when the needle reaches the uterine lining. Once it has been fed into the uterus, the gynecologist can see the inside of the organ and direct the tube to the lining.

    Scientists in Valencia showcase Transfer Direct.
    JESS HAMZELOU / MITTR

    “When everything is ready, you just press the button,” Santamaria said as he activated it using a foot pedal, allowing the embryo to be injected. “There it goes.”

    The team has just started a trial of the device; so far, fewer than 10 women have undergone the procedure, and none of those have become pregnant. But foundation director Carlos Simon is hopeful, noting that the inventors of IVF had to perform over 160 cycles before Louise Brown was born (between 1969 and 1978, that team performed 457 cycles in 250 people, resulting in only two live births). “The trial is ongoing,” he says.

    2. Picking the “best” eggs, sperm, and embryos

    One long-running challenge of IVF has been selection. Say you manage to collect 10 eggs from one partner and a decent-looking semen sample from the other. How do you choose which cells to use? The same question comes up once the resulting embryos have been cultured in a dish for a few days: Which should you transfer to the uterus?

    Traditionally, these judgments have been made by eye. Embryologists literally pick the ones that look the best in terms of their shape or, in the case of sperm, how they move. But scientists have been working on alternatives. And over the last decade or so, many have turned to genetic testing to hint at which embryos have the best chances of creating a healthy baby.

    The most commonly used test is called PGT-A, which stands for preimplantation genetic testing for aneuploidy. Aneuploidy essentially means having an “incorrect” number of chromosomes, and it is thought that embryos with such characteristics are more likely to be lost through miscarriage or potentially develop into babies with genetic conditions.

    Once embryologists have created embryos in the lab, they can pinch off a few cells and test them for aneuploidies. The tests are especially beneficial for women over the age of 38, says Alan Penzias, a reproductive endocrinologist at Boston IVF. “You start to see an improvement: more babies and fewer miscarriages,” he says. The tests can shorten the time to pregnancy.

    This type of genetic testing is possible thanks to multiple advances in technology—not just in genomics, but also in the ability to keep embryos alive in a dish for five to six days and the technique of freezing embryos while the cells undergo testing and thawing them once the results are in. And it has become hugely popular—some clinics do PGT-A tests on all their embryos.

    But PGT-A won’t give you a perfect readout of a future baby’s genetics, says Sonia Gayete-Lafuente, a reproductive endocrinologist at the Center for Human Reproduction in New York City. And some of the abnormalities might be able to self-correct with time. Gayete-Lafuente and her colleagues have transferred some of those “abnormal” embryos into patients’ uteruses and seen them develop into perfectly healthy children, she says.

    Other forms of PGT are even more controversial. PGT-P tests are designed to predict an embryo’s chances of developing complex traits that rely on multiple genes, including medical disorders but also physical characteristics like height or cognitive factors like IQ. These tests are new, and they are illegal in some countries, including the UK. But they are gaining ground in the US. Nucleus Genomics—a company that invites customers to “have [their] best baby”—promises to predict traits running the gamut from eye color and intelligence to left-handedness and risk of Alzheimer’s.

    When I asked IVF practitioners how they might respond if a patient asked for this service, most dodged the question and told me there’s not enough evidence that any of these tests actually work. They also cautioned that selecting for one trait might inadvertently introduce new risks. None seemed especially keen on the idea of using genetic testing for anything other than preventing serious disease.

    3. Speeding things up with AI

    Some seemed more excited about the potential for AI. After all, AI tools are generally good at recognizing patterns. Many researchers have attempted to train tools to spot healthy sperm, eggs, and embryos.

    And they’ve had some success. A team at Columbia University Medical Center in New York has developed a device that uses AI to examine semen samples from men who have only tiny numbers of healthy sperm. An embryologist might struggle to find a single healthy sperm in such a sample. But the Sperm Tracking and Recovery (STAR) system can analyze over a million microscope images in an hour. It has already been used to create healthy embryos. The team behind the work announced the first pregnancy resulting from the treatment in November last year.

    Other teams are using AI tools to advance IVF in more dramatic ways. Around a decade ago, a reproductive endocrinologist named Alejandro Chavez-Badiola began developing an AI tool trained to rank embryos, another to rank eggs, and another to select sperm. He recalls being struck by a realization that these tools were “the brains that have the potential to drive robots in the future,” he says.

    4. Using robots to standardize IVF

    In the early 2020s, Chavez-Badiola and his colleagues decided to combine technologies and develop an automated system for IVF. In theory, a robotic system loaded up with AI tools could undertake most of the steps required in the IVF process: selecting the eggs and sperm, fertilizing eggs to create embryos, culturing those embryos in a dish, and selecting the “best” one for transfer. Such a system could “do everything in a standard way” without ever getting tired, he says.

    Chavez-Badiola, who is now founder and chief medical officer at Conceivable, started building prototypes by motorizing regular IVF equipment and connecting it to computers. He and his colleagues started testing their system with animal cells before eventually moving on to human ones. “We were able to prove that integrating robots to automate different steps in IVF is doable,” he says.

    The device is now being used to prepare sperm and eggs and create embryos. At least 19 children have been born following the automated IVF. It is early days, but Chavez-Badiola is hoping that future iterations of the machine could each process thousands of IVF cycles in a year, potentially making the procedure more affordable and accessible.

    Many in the field are excited about the potential for automated devices like Conceivable’s. “This is all time saved for the embryologists,” says Laura Rienzi, a clinical embryologist and scientific director of the IVIRMA network of fertility centers in Italy. She also hopes it will help standardize IVF treatments. “Automation [will allow for] every patient to be treated in the same way in every single lab in the world,” she says.

    5. Controversial edits are on the table

    There’s a catch, however: All these technologies rely on the availability of at least some healthy sperm, eggs, and embryos at the outset. Embryologists and IVF patients have to work with what they’ve got. And sometimes, what they’ve got won’t result in a healthy baby. 

    That’s why some scientists are proposing a controversial idea: using gene-editing technologies like CRISPR to tinker with the genome of an IVF embryo before it is implanted. The biophysicist He Jiankui infamously took this approach to create embryos that resulted in the births of three children in the late 2010s. He was widely condemned by the scientific community and ultimately spent three years in a Chinese prison. 

    His former romantic partner Cathy Tie, who now leads startup Origin Genomics, is pursuing the technology as a potential way to prevent serious disease in children. At a recent event held at the Hastings Center for Bioethics, Tie made the case for using embryo editing to prevent diseases like cystic fibrosis, Huntington’s, and sickle-cell.

    It won’t be straightforward from a technical, legal, or ethical perspective. Diseases that are known to be caused by single-gene mutations are good first candidates, but as the Center for Human Reproduction’s Gayete-Lafuente points out, most diseases are much more complicated than that. “I wish we could understand the genetic basis of every disease to be able to prevent it,” she says. So far, we can’t. Besides, most diseases can be influenced by our diets, behaviors, and environments as well as our genes.

    As things stand, no one knows if editing a human embryo to eliminate the risk of one disease might increase a future child’s risk of some other disorder. And some scientists worry that such edits might be a slippery slope to genetic enhancement or eugenics.

    Rienzi hopes that the technology might be developed in a safe way with regulatory oversight, and only for a specific list of diseases. “It has to be within a legal context,” she says. “But to me, it’s a dream.”

    In the meantime, the field looks set to keep transforming with the development of new technologies that are already creating healthy babies. Watch this space. 

    ]]>     Bayer to Acquire Perfuse for Up to $2.45B, Seeing Ophthalmology Opportunity https://edusehat.com/en/bayer-to-acquire-perfuse-for-up-to-245b-seeing-ophthalmology-opportunity-10461 https://edusehat.com/en/bayer-to-acquire-perfuse-for-up-to-245b-seeing-ophthalmology-opportunity-10461 Thu, 07 May 2026 22:05:13 +0700 Edusehat Bayer, Acquire, Perfuse, for, 2.45B, Seeing, Ophthalmology, Opportunity Bayer has agreed to acquire Perfuse Therapeutics for up to $2.45 billion, the companies said, in a deal designed to broaden the buyer’s ophthalmology pipeline with Perfuse’s sole pipeline drug and two clinical phase programs for eye disorders.

    Perfuse’s PER-001 is a small molecule endothelin receptor antagonist being developed for the treatment of ophthalmic diseases. Two of PER-001’s four programs are in Phase II development: One designed to treat open-angle glaucoma by improving the visual field for patients, and the other designed to treat diabetic retinopathy (DR) by improving contrast sensitivity and reducing ischemia in patients with the disorder.

    Last year, Perfuse announced positive results from two Phase II clinical trials evaluating PER-001.

    One was a Phase IIa trial (NCT05822245) assessing PER-001 in glaucoma, which showed that six months after a single intravitreal administration of PER-00, added to existing standard-of-care intraocular pressure (IOP)-reducing therapies, 22.2% of low-dose and 37.5% of high-dose patients experienced ≥7 decibel (dB) improvement in a pre-defined retina region of minimal five test points compared to 0% in control in six months.

    The improvement was 8–14x better than the natural history of disease (2.7%) with currently available treatments, Perfuse said at the time.

    In the other Phase IIa trial (NCT06003751), which focused on DR, patients showed a mean of +0.9 dB improvement in low luminance contrast sensitivity in the high-dose group and +0.65 dB in the low-dose group across multiple frequencies measured at week 20. In contrast, a mean of -2.1 dB worsening occurred in the control group over the same period.

    The low luminance, low contrast visual acuity was better by a mean difference of 5.5 and 5.1 letters from baseline in low- and high-dose groups compared to control measured at week 20, Perfuse said at the time.

    PER-001 is also in preclinical development for dry age-related macular degeneration (AMD)/geographic atrophy, as well as for retinal vein occlusion.

    “We are excited by the work of the team at Perfuse Therapeutics and encouraged by the potential of PER-001,” Juergen Eckhardt, MD, head of business development and licensing at Bayer Pharmaceuticals, said in a statement. “With this acquisition, we are complementing our expertise in ophthalmology and our pipeline, reinforcing our commitment to developing urgently needed therapies for patients.”

    Looking beyond Eylea®

    Bayer’s ophthalmology pipeline has long been dominated by the blockbuster drug Eylea® (aflibercept), co-marketed with Regeneron Pharmaceuticals and initially approved in 2011. However, Eylea is close to losing exclusivity for key U.S. patents: According to Regeneron’s Form 10-K annual report for 2024, patents for Eylea expire between 2027 and 2039, starting with four formulation patents expiring on June 14, 2027. Patents for the higher-dose version, Eylea HD®, expire between 2027 and 2032, starting with two formulation patents expiring on June 14, 2027.

    Last year, Eylea and Eylea HD saw their sales slip in the mid-teens, generating a total combined $8.04 billion in revenue, consisting of $4.385 billion in U.S. net sales for Regeneron and €3.11 billion in ex-U.S. sales for Bayer (about $3.655 billion today, up from the $3.506 billion reported in January).

    During the first quarter of this year, Regeneron reported $941 million in U.S. sales, down 10% from a year ago; Bayer plans to report Q1 sales on May 12.

    PER-001 is an intravitreal bio-erodible implant administered into the vitreous cavity of the eye using a single-use, 25-gauge applicator and designed to provide a sustained release of the drug, allowing for a convenient dosing regimen, according to Perfuse and Bayer.

    Bayer has agreed to pay $300 million upfront for Perfuse, which is headquartered in San Francisco with R&D facilities in Durham, NC. The remaining up to $2.15 billion in deal value hinges on Bayer achieving development, regulatory, and commercial milestones.

    The acquisition deal is subject to approval by Perfuse shareholders and antitrust clearances.

    “I’m incredibly proud of what the Perfuse team has accomplished and deeply thankful to all our investors and collaborators,” stated Sevgi Gurkan, MD, Perfuse’s founder and CEO. “Bayer’s vision aligns closely with ours, and they have the scale and global resources to unlock the full potential of PER-001 to change the trajectory of human blindness. We are very excited to see our mission continue with even greater momentum.”

    The post Bayer to Acquire Perfuse for Up to $2.45B, Seeing Ophthalmology Opportunity appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Next Gen Leadership Awards Presented at the AGBT Agricultural Meeting https://edusehat.com/en/next-gen-leadership-awards-presented-at-the-agbt-agricultural-meeting https://edusehat.com/en/next-gen-leadership-awards-presented-at-the-agbt-agricultural-meeting Thu, 07 May 2026 22:05:10 +0700 Edusehat Next, Gen, Leadership, Awards, Presented, the, AGBT, Agricultural, Meeting Last month, the AGBT Agricultural Meeting was held in Phoenix, Arizona. The conference is focused on agricultural genomics—plant and animal genetics. During the meeting, the recipients of the 2026 Next Gen Leadership Awards were announced.

    These awards recognize outstanding early-career scientists and graduate students whose work and potential are shaping the future of agricultural genomics, including advances in plant and animal genomics. Award recipients receive financial support to attend and present their research at the AGBT Agricultural Meeting, with opportunities to engage with leaders in the field and build connections across the genomics community.

    “These awardees reflect the strength and diversity of emerging talent in agricultural genomics,” said Sarah Hearne, PhD, chief science and innovation officer at CIMMYT and co-chair of the AGBT Agriculture Scientific Organizing Committee. “AGBT Agriculture plays an important role in bringing these scientists into conversation with leaders across the field, helping accelerate the translation of genomics into practice.”

    The awardees represent rising leaders in agricultural genomics, advancing research across genomic variability, genetic analysis, molecular diagnostics, pathogen surveillance, and quantitative trait genomics to improve crop performance, strengthen food safety, and advance sustainable agriculture.

    “This award represents a transformative opportunity to grow as a scientist and contribute more effectively to innovation in animal breeding,” said Larissa Bordin Temp, a 2026 Next Gen Leadership Award recipient.

    The 2026 AGBT Agricultural Meeting Next Gen Leadership awardees were:

    • Boris ME Alladassi, PhD: postdoctoral research associate at the University of Illinois Urbana-Champaign
      • Research focus: Connecting the evolutionary and statistical views of epistasis in quantitative trait genomics
    • Mythri Bikkasani: graduate student at Punjab Agricultural University, India
      • Research focus: Connecting the dots: from high-throughput feed phenotyping to genomic dissection of heterosis in maize
    • Larissa Bordin Temp: graduate student at São Paulo State University, Faculty of Agricultural and Veterinary Sciences
      • Research focus: Genomic evaluation of rump fat–adjusted residual feed intake in zebu cattle: implications for selection strategies
    • Lauren Johnson: graduate student at Gluck Equine Research Center, University of Kentucky
      • Research focus: Functional introgression within the horse mhc genes
    • Mehak Kapoor: graduate assistant at Iowa State University
      • Research focus: Cell-type resolved gene expression signatures to identify and predict persistent PRRSV infection
    • Pedro Nuñez Romano, PhD: postdoctoral researcher at Universitat Politècnica de València
      • Research focus: Integrating technology to refine the estimation of social genetic effects in pigs
    • Viona Osei: graduate student at Tuskegee University
      • Research focus: Exploiting genomic variability in Listeria for the development of molecular diagnostic markers
    • Kyungyong Seong, PhD: postdoctoral fellow at the University of California, Davis
      • Research focus: Resurrection of the plant immune receptor Sr50 to overcome pathogen immune evasion
    • Jade van Wijk: graduate student at Earlham Institute
      • Research focus: Using airborne DNA sequencing to monitor sporulation, infection and relative abundance of cereal rust fungi

    The post Next Gen Leadership Awards Presented at the AGBT Agricultural Meeting appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Bayer to Acquire Perfuse for up to $2.45B, Seeing Ophthalmology Opportunity https://edusehat.com/en/bayer-to-acquire-perfuse-for-up-to-245b-seeing-ophthalmology-opportunity https://edusehat.com/en/bayer-to-acquire-perfuse-for-up-to-245b-seeing-ophthalmology-opportunity Thu, 07 May 2026 11:10:10 +0700 Edusehat Bayer, Acquire, Perfuse, for, 2.45B, Seeing, Ophthalmology, Opportunity Bayer has agreed to acquire Perfuse Therapeutics for up to $2.45 billion, the companies said, in a deal designed to broaden the buyer’s ophthalmology pipeline with Perfuse’s sole pipeline drug and two clinical phase programs for eye disorders.

    Perfuse’s PER-001 is a small molecule endothelin receptor antagonist being developed for the treatment of ophthalmic diseases. Two of PER-001’s four programs are in Phase II development: One designed to treat open-angle glaucoma by improving the visual field for patients, and the other designed to treat diabetic retinopathy (DR) by improving contrast sensitivity and reducing ischemia in patients with the disorder.

    Last year, Perfuse announced positive results from two Phase II clinical trials evaluating PER-001.

    One was a Phase IIa trial (NCT05822245) assessing PER-001 in glaucoma, which showed that six months after a single intravitreal administration of PER-00, added to existing standard-of-care intraocular pressure (IOP)-reducing therapies, 22.2% of low-dose and 37.5% of high-dose patients experienced ≥7 decibel (dB) improvement in a pre-defined retina region of minimal five test points compared to 0% in control in six months.

    The improvement was 8–14x better than the natural history of disease (2.7%) with currently available treatments, Perfuse said at the time.

    In the other Phase IIa trial (NCT06003751), which focused on DR, patients showed a mean of +0.9 dB improvement in low luminance contrast sensitivity in the high-dose group and +0.65 dB in the low-dose group across multiple frequencies measured at week 20. In contrast, a mean of -2.1 dB worsening occurred in the control group over the same period.

    The low luminance, low contrast visual acuity was better by a mean difference of 5.5 and 5.1 letters from baseline in low- and high-dose groups compared to control measured at week 20, Perfuse said at the time.

    PER-001 is also in preclinical development for dry age-related macular degeneration (AMD)/geographic atrophy, as well as for retinal vein occlusion.

    “We are excited by the work of the team at Perfuse Therapeutics and encouraged by the potential of PER-001,” Juergen Eckhardt, MD, head of business development and licensing at Bayer Pharmaceuticals, said in a statement. “With this acquisition, we are complementing our expertise in ophthalmology and our pipeline, reinforcing our commitment to developing urgently needed therapies for patients.”

    Looking beyond Eylea®

    Bayer’s ophthalmology pipeline has long been dominated by the blockbuster drug Eylea® (aflibercept), co-marketed with Regeneron Pharmaceuticals and initially approved in 2011. However, Eylea is close to losing exclusivity for key U.S. patents: According to Regeneron’s Form 10-K annual report for 2024, patents for Eylea expire between 2027 and 2039, starting with four formulation patents expiring on June 14, 2027. Patents for the higher-dose version, Eylea HD®, expire between 2027 and 2032, starting with two formulation patents expiring on June 14, 2027.

    Last year, Eylea and Eylea HD saw their sales slip in the mid-teens, generating a total combined $8.04 billion in revenue, consisting of $4.385 billion in U.S. net sales for Regeneron and €3.11 billion in ex-U.S. sales for Bayer (about $3.655 billion today, up from the $3.506 billion reported in January).

    During the first quarter of this year, Regeneron reported $941 million in U.S. sales, down 10% from a year ago; Bayer plans to report Q1 sales on May 12.

    PER-001 is an intravitreal bio-erodible implant administered into the vitreous cavity of the eye using a single-use, 25-gauge applicator and designed to provide a sustained release of the drug, allowing for a convenient dosing regimen, according to Perfuse and Bayer.

    Bayer has agreed to pay $300 million upfront for Perfuse, which is headquartered in San Francisco with R&D facilities in Durham, NC. The remaining up to $2.15 billion in deal value hinges on Bayer achieving development, regulatory, and commercial milestones.

    The acquisition deal is subject to approval by Perfuse shareholders and antitrust clearances.

    “I’m incredibly proud of what the Perfuse team has accomplished and deeply thankful to all our investors and collaborators,” stated Sevgi Gurkan, MD, Perfuse’s founder and CEO. “Bayer’s vision aligns closely with ours, and they have the scale and global resources to unlock the full potential of PER-001 to change the trajectory of human blindness. We are very excited to see our mission continue with even greater momentum.”

    The post Bayer to Acquire Perfuse for up to $2.45B, Seeing Ophthalmology Opportunity appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Microproteins and Peptideins Expand Boundaries of the Human Proteome https://edusehat.com/en/microproteins-and-peptideins-expand-boundaries-of-the-human-proteome https://edusehat.com/en/microproteins-and-peptideins-expand-boundaries-of-the-human-proteome Thu, 07 May 2026 07:35:12 +0700 Edusehat Microproteins, and, Peptideins, Expand, Boundaries, the, Human, Proteome A research team led by scientists at the Princess Máxima Center for Pediatric Oncology, the University of Michigan Medical School, EMBL European Bioinformatics Institute, and the Institute for Systems Biology, has uncovered more than 1,700 new proteins that could have implications for human diseases, including cancer.

    Mostly very small, these proteins have been discovered in what’s known as the “dark proteome,” which covers gene products from previously overlooked sections of DNA. These proteins have unusual properties, motivating scientists to coin a new concept, peptideins, to help understand their potentially unique biology. Research co-lead Sebastiaan van Heesch, PhD, a group leader at the Princess Máxima Center, commented, “We know that the current overview of recognized proteins doesn’t capture the full picture. With this study, we show that thousands of overlooked genetic sequences contribute to the dark proteome by producing a new class of protein-like molecules, microproteins, that had been missed before now. But for most of them, we don’t yet know what they do.”

    Research co-lead and co-corresponding author Robert Moritz, PhD, professor and head of proteomics at the Institute for Systems Biology, further noted, “Biology has long relied on a relatively small cast of well-characterized proteins to explain the regulatory logic of the cell, but peptideins suggest that beneath that familiar layer lies an entire untapped layer of molecular actors whose functional roles in gene regulation, signaling, and cytopersistence, many we are only beginning to imagine. Given their smaller size and the diversity of cellular contexts in which they appear, I believe peptideins may prove to be among the most versatile and consequential regulatory molecules we have yet encountered in human biology. This is not the end of a search—it is the opening of a vast and fertile new territory for the entire scientific community to explore and exploit, and I look forward to seeing what the broader scientific community uncovers as these molecules, and many more that are yet to be confirmed, are brought into the light.”

    Research co-lead John Prensner, MD, pediatric neurooncologist at the University of Michigan Medical School, together with Van Heesch and Moritz, are co-senior and co-corresponding authors of the researchers’ published paper in Nature titled “Expanding the human proteome with microproteins and peptideins.” The team is sharing its discoveries with scientists worldwide in an open-source format to stimulate further research.

    Van Heesch added, “With growing interest in industry and academia, peptideins are at the center of multiple drug development initiatives. Similarly, we see them increasingly turning up as important players in diseases, including childhood cancers. We hope to inspire a new wave of research into peptideins and to unlock new insights and drug targets across human biology, particularly for the development of cellular immunotherapies and cancer vaccines.”

    The study is the work of the TransCODE Consortium, an international collaboration of more than 60 researchers at over 30 institutions worldwide, co-led by the Princess Máxima Center for Pediatric Oncology in the Netherlands, the University of Michigan Medical School, the EMBL European Bioinformatics Institute in Hinxton, and the Institute for Systems Biology in Seattle.

    Genes in DNA provide the recipe for cells to produce peptides. Historically, peptides have been called proteins if they are long enough and have existing evidence for a biological role, such as the appearance of the same protein across species in evolution. “Protein-coding genes are the bedrock of biomedical investigations, including the overwhelming majority of drug development programs,” the authors wrote. A large, curated international database of proteins contains some 19,500 entities.

    But increasingly, scientists believe the traditional definition of a protein needs to be broadened. “Whether the human genome encodes substantially more than the approximately 19,500 canonical protein-coding genes has sparked a spirited debate in recent years,” the scientist continued. “Therefore, any wholesale addition of protein-coding genes creates ripple effects across human bioscience.”

    Through their newly reported study the team looked at more than 7,200 previously understudied sections of the DNA called non-canonical open reading frames (ncORFs). They found that some 25% of these sections—more than 1,700—generated detectable protein-like molecules. These proteins, smaller than traditional proteins, are referred to as “microproteins.”

    Generating their results involved looking at 3.7 billion individual bits of raw data that may support known and previously unknown proteins—drawing upon 95,520 experiments. “We show that about 25% of a set of 7,264 ncORFs gives rise to detectable peptides in a large-scale analysis of 95,520 proteomics experiments,” they wrote. The process took around 20,000 hours for computers to complete, working non-stop. They found 1,785 microproteins, a number that at first glance would increase the protein databases by nearly 10%.

    Predicted binding between a non-canonical open reading frame (blue) and traditional protein (yellow). [Leron Kok/Princess Máxima Center for pediatric oncology]
    Predicted binding between a non-canonical open reading frame (blue) and traditional protein (yellow). [Leron Kok/Princess Máxima Center for Pediatric Oncology]
    Moritz further explained, “By deploying our battle-hardened Trans Proteomic Pipeline across nearly 100,000 mass spectrometry experiments encompassing 3.7 billion spectra—derived from the world’s collective publicly available mass spectrometry data, with the results housed within PeptideAtlas at ISB for the scientific community to view and share—we were able to confirm, with high confidence, the existence of more than 1,700 of these newly identified peptideins that would otherwise have largely remained invisible to science.”

    But most of these 1,785 microproteins didn’t resemble the other 19,500 traditional proteins. For example, they were very small: 65% were fewer than 50 amino acids in length, compared to less than 1% of the 19,500 previously catalogued. Looking more closely at the microproteins the investigators saw that only a few—perhaps a dozen—resembled the traditional proteins. The team then spent more than a year trying to make sense out of the remaining bulk.

    Working with protein experts from across the globe in the TransCODE consortium, the scientists coined a new biological concept, which they coined peptidein. For decades, the research community has had a binary view of the relationship between human DNA and human proteins.  A given piece of DNA either does or does not produce a protein. In their new study, the scientists propose a third choice, which is that DNA could make a protein, a peptidein, or neither.

    The team defined a peptidein as existing in cells as a protein-like molecule, meaning that it is made of amino acids, as are proteins. But the role of a peptidein is ambiguous. Perhaps it has a function in normal human biology, or perhaps not; this is the key distinction with traditional proteins, where all are believed to have a function in normal human biology even if the details of that function are not fully known yet. “To advance these ncORFs in biological inquiry, we invoke the emerging umbrella term of peptidein, which we define as an ORF with experimentally confirmed RNA translation and protein synthesis, but for which the data are currently insufficient to claim conventional protein-coding gene status,” the investigators stated in their report.

    Importantly, this definition of peptidein leaves the door open for it to become a ‘protein’ in the future—that is, if scientists gather more evidence on it.  To start exploring this idea, the team searched for peptideins without which cells cannot survive. These so-called pan-essential peptideins can be important candidate drug targets in cancer and other diseases.

    Using large-scale CRISPR gene editing, the scientists found six peptideins that looked promising. For example, one of these was a peptidein produced from OLMALINC, a genetic sequence previously thought not to produce proteins. When the researchers switched this gene off, 85% of more than 485 cancer cell lines showed impaired survival. The researchers confirmed that this effect comes from the peptidein itself, not the RNA molecule it sits on, and found that it plays a role in cell division and DNA damage response. “Our work here highlights c10riboseqorf92 (in the OLMALINC transcript),” they commented. “… while we do not yet have sufficient evidence that this ncORF encodes a bona fide protein, its CRISPR-based phenotypes in the context of cancer cells are intriguing.”

    Many of the newly detected peptideins are presented on cell surfaces for recognition by the immune system, making them potential targets for cancer immunotherapy. A number of such molecules presented to the immune system are already under development as drug targets, and there is growing interest from both academia and industry in exploiting this new class of cancer antigens. Peptideins could also shed light on genetic diseases that conventional gene analysis has been unable to explain, simply because genetic diagnostics were unaware that these molecules were encoded by the human genome.

    Members of the consortium had previously uncovered an essential role for a microprotein, ASNSD1-uORF, in children with a high-risk form of the brain cancer, medulloblastoma. Scientists at the Princess Máxima Center are now carrying out further research to determine its role in additional pediatric cancers with the activated MYC oncogene, such as neuroblastoma.

    van Heesch commented, “It felt really special to discuss and decide what to do with this new class of molecules, as we had gathered enough early evidence to suspect that they might be widespread across cell types and tissues. By classifying these molecules of unknown functionality as peptideins, we’ve given them a formal place in reference databases so the wider community can study them.”

    In their paper the researchers concluded, “The extent of the undiscovered proteome is one of the central questions in human biomedicine. This work reflects the multi-consortium collaboration between the TransCODE Consortium, the HUPO-HPP/PeptideAtlas project, the HIPP immunopeptidomics project and the GENCODE gene annotation group to coalesce a generalizable approach towards understanding which ncORFs can be understood as encoding proteins … Through our efforts, we bring microproteins and alternative protein molecules into reference gene annotation by defining them as either a protein-coding gene or a peptidein, a new concept referring to confirmed protein molecules of indeterminate consequence.”

    Prensner added, “We’re just beginning to see what this ‘dark proteome’ has to offer.  It’s like the trailer to a movie. We see the outline of a game-changing view of human biology.  We’re incredibly excited that the coming years will open new doors to help solve and treat human diseases such as cancer.”

    Moritz further stated, “Our collaborative work represents a culmination of decades of investment from federal funding agencies in building the computational and data infrastructure needed to interrogate the proteome at truly unprecedented scale at the Institute for Systems Biology … What excites me most is not simply that these molecules exist, but what their existence implies.”

    The researchers are making we make all ncORFs, peptides and spectra publicly available through PeptideAtlas.

    The post Microproteins and Peptideins Expand Boundaries of the Human Proteome appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     From Discovery to GMP: Building Scalable Cell Therapy Manufacturing https://edusehat.com/en/from-discovery-to-gmp-building-scalable-cell-therapy-manufacturing https://edusehat.com/en/from-discovery-to-gmp-building-scalable-cell-therapy-manufacturing Thu, 07 May 2026 07:35:11 +0700 Edusehat From, Discovery, GMP:, Building, Scalable, Cell, Therapy, Manufacturing

    From Discovery to GMP: Building Scalable Cell Therapy Manufacturing eBook

    Over the past decade, our industry has witnessed the promise of cell and gene therapies. Patients with rare diseases or hard-to-treat diagnoses now have treatment options harnessing human cells and genes to alter disease. The accessibility of these therapies remains constrained not by what’s biologically possible, but how they are designed and manufactured.

    The field has reached an inflection point. We’ve demonstrated the scientific foundation and its curative potential. To make advanced therapies sustainable as a pillar of medicine, we must make them more accessible. The companies that will define cell and gene therapy’s future will be those who can eliminate the distance between top science and efficient manufacturing.

    This eBook brings together perspectives from Genetic Engineering News and ElevateBio to examine both the technical and operational realities shaping cell therapy today. From emerging innovations to persistent manufacturing challenges, the goal is to connect scientific progress with the systems required to scale it.

    Traditional drug development has relied on siloed pathways, where therapeutics are designed and developed by one team and then manufactured by another. This approach is especially challenging in cell therapy, often leading to delays, setbacks, or outright failures. ElevateBio was built differently. Therapeutic design, development, and manufacturing operate as an integrated ecosystem, enabling tighter coordination and faster iteration.

    The future of cell therapy depends on therapies designed with manufacturability in mind from the start. Process development, analytical strategy, and quality considerations must be embedded early, allowing manufacturing insights to inform development decisions in real time. This includes optimizing constructs, delivery systems, and processes to ensure scalability, reproducibility, and readiness for GMP production.

    Looking beyond the science, a sustainable cell therapy ecosystem requires more than better therapeutics. It requires expanded treatment infrastructure, new commercial models, and systems capable of supporting broader patient access. But that ecosystem cannot scale on unreliable manufacturing.

    As cell therapies expand into larger patient populations and new indications, the need for manufacturing designed for reliability and scale from day one becomes more urgent. The therapies being developed today have the potential to transform millions of lives—but only if the systems supporting them are built to deliver at scale.

    Michael Paglia, Chief Technology Officer, ElevateBio

    The post From Discovery to GMP: Building Scalable Cell Therapy Manufacturing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     UCB to Acquire Candid for Up to $2.2B, Expanding Presence in TCE Antibodies for Immunology https://edusehat.com/en/ucb-to-acquire-candid-for-up-to-22b-expanding-presence-in-tce-antibodies-for-immunology-10418 https://edusehat.com/en/ucb-to-acquire-candid-for-up-to-22b-expanding-presence-in-tce-antibodies-for-immunology-10418 Thu, 07 May 2026 00:20:17 +0700 Edusehat UCB, Acquire, Candid, for, 2.2B, Expanding, Presence, TCE, Antibodies, for, Immunology UCB has agreed to acquire Candid Therapeutics for up to $2.2 billion, the companies said, in a deal to expand the buyer’s presence in T-cell engager (TCE) antibodies designed for immunology indications by adding Candid’s pipeline of bispecific and trispecific antibody candidates.

    The deal upends plans announced in March by Candid to enter a reverse merger with Rallybio, in which Rallybio would have acquired Candid but retained Candid’s name and created new shares to be traded on NASDAQ. The new company was to have developed Candid’s pipeline using $505 million in concurrent financing from a syndicate of healthcare institutional investors and mutual funds.

    Based in San Diego, privately held Candid’s pipeline of autoimmune and inflammatory disease candidates includes treatments licensed from Chinese biotechs.

    Candid’s lead asset, cizutamig, is a bispecific antibody for autoantibody-driven autoimmune diseases. Licensed from Shanghai-based EpimAb Biotherapeutics, cizutamig is directed to B-cell maturation antigen (BCMA) on plasma cells and CD3 on T cells, with the aim of enabling T-cell–mediated cytotoxicity against both kinds of cells while limiting cytokine release. Cizutamig is currently in multiple Phase I clinical studies in over 10 autoimmune indications, with clinical evaluation in more than 100 patients with multiple myeloma (completed with 40 patients) and autoimmune diseases (68 patients across multiple indications in China and Europe).

    Also in Phase I development within Candid’s pipeline is CND261, a CD20 x CD3 bispecific antibody TCE that the company licenses from Shanghai-based Genor Biopharma. CND261 is being developed to treat autoimmune diseases by targeting a variety of B-cell subtypes, from pro-B-cells to plasmablasts/plasma cells. Candid has completed a 93-patient Phase I dose escalation study of CND261 in non-Hodgkin’s lymphoma (NHL).

    The rest of Candid’s pipeline consists of two preclinical candidates in IND-enabling studies:

    • CND319, a CD19xCD20xCD3 trispecific antibody designed to target the CD19 and CD20 antigens on a broad range of B-cell subtypes, and licensed from WuXi Biologics last year for up to $925 million in upfront and milestone payments, plus royalties.
    • CND460, a BCMAxCD19xCD3 trispecific antibody designed to target the BCMA and CD19 antigens on a broad range of B-cell subtypes.

    CND261, CND319, and CND460 represent a pipeline of multi-specific TCE antibodies designed to enable deep, targeted depletion of pathogenic B cell populations in immune-mediated diseases to achieve immune reset—what UCB termed a modular, multi-antigen targeting strategy to address complementary B-cell subsets.

    “We started Candid with the goal to redefine the standard of care for immune-mediated diseases. We purposefully built a broad portfolio of TCE assets against a number of clinical indications,” stated Ken Song, MD, Candid’s chairman, CEO, and president. Previously, as president, CEO, and board director of radiopharmaceutical developer RayzeBio, Song negotiated the approximately $4.1 billion sale of the company to Bristol Myers Squibb, completed in 2024.

    “Our focus has been to efficiently generate clinical data so as to identify where our TCEs could provide maximal clinical benefit for the broadest number of patients,” Song explained.

    Investors appeared less enthusiastic about the deal. UCB stock is traded primarily on Euronext Brussels, where the company’s shares barely budged Monday, dipping 0.65% to €228.30 ($267.28) as of 10:37 am ET.

    Platform-based strategy

    UCB’s plan to acquire Candid comes roughly two months after the Belgian biotech giant agreed to license exclusive global rights to further develop, manufacture, and commercialize Hong Kong-based Antengene’s ATG-201, a CD19 and CD3 bispecific TCE antibody designed to target B cell-related autoimmune diseases. UCB agreed to pay Antengene $80 million in upfront and near-term milestone payments, plus up to approximately $1.1 billion in payments tied to achieving development and commercial milestones under the agreement, which also granted UCB access to Antengene’s associated manufacturing technology in relation to ATG-201.

    UCB said its planned acquisition of Candid, plus the Antengene deal, reflects a platform-based strategy of complementary investments intended to expand its reach across multiple B-cell targets and disease mechanisms, thus strengthening its ability to address antibody-mediated autoimmune diseases through multiple approaches rather than relying on a single asset or modality.

    “This acquisition demonstrates our inorganic innovation strategy in action and marks a pivotal moment for UCB, as we secure a significant technological advancement in the field with the addition of cizutamig to our pipeline,” UCB CEO Jean-Christophe Tellier said in a statement. “This exemplifies the next wave of therapies to treat immune-mediated diseases and reflects our commitment to setting new standards to achieve immune reset.

    Tellier added that UCB considers cizutamig “a potential transformative asset, that complements our existing programs, and is poised to redefine treatment expectations for severe, underserved immune-mediated diseases, offering the potential to deliver meaningful improvements in patient outcomes and quality of life.”

    UCB has agreed to pay Candid $2 billion upfront and up to $200 million in potential future milestone payments. The transaction is subject to closing conditions that include antitrust clearance and other customary conditions and is expected to close by the end of the second quarter or early Q3 2026.

    The deal is good news for Two River and Vida Ventures, funds that played central roles in the creation and early development of Candid, which was launched in 2024 with more than $370 million in capital. Two River, together with Third Rock Ventures, helped found Candid, which was created through the merger of Two River-founded TRC 2004 and Vignette Bio.

    “This outcome reflects the power of bringing together bold science, disciplined company building, and the right strategic partners,” said Arie Belldegrun, MD, founder and senior managing director of Vida Ventures, chairman of Two River, and co-founder of Bellco Health.

    UCB added that it expects the anticipated financial impact of the Candid acquisition to be “manageable.” The company has not changed its most recent 2026 guidance, which calls for revenue growth in the high single‑digit to low double‑digit range at constant exchange rates, while underlying profitability, measured by “adjusted” earnings before interest, taxes, depreciation, and amortization (EBITDA), which excludes one-time expenses, is expected to increase in the high single‑digit to mid‑teens range.

    “UCB’s successful track record in immunology, including development, launch, and commercialization, will enable the continuation of our clinical programs and help deliver on the potential for our pipeline,” Song added.

    The post UCB to Acquire Candid for Up to $2.2B, Expanding Presence in TCE Antibodies for Immunology appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Light-Controlled Gene Expression Platform Reportedly Doubles Standard Fed-Batch Manufacturing Performance https://edusehat.com/en/light-controlled-gene-expression-platform-reportedly-doubles-standard-fed-batch-manufacturing-performance https://edusehat.com/en/light-controlled-gene-expression-platform-reportedly-doubles-standard-fed-batch-manufacturing-performance Thu, 07 May 2026 00:20:15 +0700 Edusehat Light-Controlled, Gene, Expression, Platform, Reportedly, Doubles, Standard, Fed-Batch, Manufacturing, Performance Prolific Machines, an Emeryville, CA-based biotech company focusing on light-controllable biomanufacturing powered by optogenetics, reported a productivity milestone of 21 g/L in a 15-day intensified fed-batch CHO run for a monoclonal antibody (mAb). This is more than double the industry standard of below 10 g/L, and the company is on track to reach its goal of 25 g/L in fed-batch by the end of 2026, according to company officials.

    Prolific’s light-controlled biomanufacturing platform uses light and optogenetics to directly and dynamically control gene expression inside living cells. Unlike conventional biomanufacturing approaches, this technology gives developers precise, real-time control over when and how much protein a cell produces, says Deniz Kent, PhD, co-founder and CEO.

    “To our knowledge, this result represents the highest reported titer for an antibody produced in a fed-batch format,” continues Kent. “The real opportunity lies in next-generation complex biologics such as multispecifics, cytokines, and fusion proteins. These molecules represent more than 50% of today’s early pipelines, yet developability challenges and chronically low yields continue to compromise their commercial viability. We’ve shown up to three-fold yield increases in that space, and are now working with multiple partners to enable high-yield manufacturing of promising molecules.”

    “After more than 20 years in the industry, I’m proud to have been one of the first scientists to have witnessed fed-batch titers exceed 20 grams per liter,” adds Julien Meissonnier, independent director for Prolific Machines, and former CSO of Catalent at the time it launched its GPEx® Lightning CHO cell line development platform. “To emphasize the strength of Deniz’s team’s results: in my experience in the industry, I’ve never seen data of this quality at this stage. This is clearly different and has a great potential to enable more complex biologics to reach patients and become the standard for the future of biomanufacturing.”

     The “record-setting run demonstrated exceptional process robustness, maintaining approximately 87% viability at harvest, reaching a peak viable cell density of approximately 33 million cells/mL, and sustaining a specific productivity of around 60 pg/cell/day,” notes a Prolific spokesperson, who cites these metrics in support of the strength of the company’s intensified process design and the underlying stability of its production platform.

     

    The post Light-Controlled Gene Expression Platform Reportedly Doubles Standard Fed-Batch Manufacturing Performance appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     CoCoGraph AI Model Generates Molecules that Comply with Rules of Chemistry https://edusehat.com/en/cocograph-ai-model-generates-molecules-that-comply-with-rules-of-chemistry https://edusehat.com/en/cocograph-ai-model-generates-molecules-that-comply-with-rules-of-chemistry Thu, 07 May 2026 00:20:14 +0700 Edusehat CoCoGraph, Model, Generates, Molecules, that, Comply, with, Rules, Chemistry Developing new molecular compounds is crucial to address pressing challenges, from drug discovery to sustainable materials. However, discovering viable new molecules is challenging due to the vastness of the search space. 

    In a new paper published in Nature Machine Intelligence titled, “A collaborative constrained graph diffusion model for the generation of realistic synthetic molecules,” researchers from Universitat Rovira i Virgili (URV) have developed an AI tool capable of generating molecules that are guaranteed to comply with the rules of chemistry. The model, named CoCoGraph, operates similarly to generative AI tools for text or images, such as ChatGPT or Dall-E.  

    “These models create new content that looks very much like the real thing. Our algorithm does the same, but with molecules,” said Roger Guimerà, PhD, ICREA research professor in the department of chemical engineering at URV and co-corresponding author of the study. He explains that the number of possible chemical molecules could be up to 10⁶⁰ variants, which is more than the number of water molecules in the ocean.  

    CoCoGraph uses a diffusion model, a technique common in image generation, which progressively “disorders” a real molecule and trains the system to learn how to reconstruct it. 

    Marta Sales-Pardo, PhD, professor in the department of chemical engineering at URV and co-corresponding author of the study, explains that the model begins with a real molecule, breaks the bonds, and then creates new bonds at random. The model then learns to reverse this process to reconstruct coherent structures. 

    Notably, CoCoGraph directly incorporates the basic rules of chemistry, such as maintaining the correct number of bonds, to guarantee that generated molecules are chemically valid. The system is also more efficient, uses fewer parameters, and requires less computing power to generate molecules more quickly. 

    The research team has compared the performance of CoCoGraph with other state-of-the-art models and analyzed 36 physicochemical properties, such as solubility and structural complexity. For two-thirds of these properties, the CoCoGraph generated molecules are chemically more realistic than those from other models. 

    Although the model cannot yet design molecules with a specific function, researchers have identified molecules with properties similar to the drug, paracetamol. They have also explored techniques to partially modify an existing molecule to create new variants with similar characteristics, which are useful for optimizing drugs or developing new materials. 

    The next step is to design molecules with specific properties, such as solubility and low toxicity. If successful, the technology could accelerate the discovery of new solutions across pharmacology and materials science in a chemical universe that is still practically unexplored. 

    The post CoCoGraph AI Model Generates Molecules that Comply with Rules of Chemistry appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Regulators Should Rely on Peers’ GMP Audits to Cut Inspection Burden https://edusehat.com/en/regulators-should-rely-on-peers-gmp-audits-to-cut-inspection-burden https://edusehat.com/en/regulators-should-rely-on-peers-gmp-audits-to-cut-inspection-burden Thu, 07 May 2026 00:20:13 +0700 Edusehat Regulators, Should, Rely, Peers’, GMP, Audits, Cut, Inspection, Burden Biopharma is a global industry with drug firms routinely supplying medicines to multiple markets from the same manufacturing plant. But while globalization has helped expand revenues, it has also increased the number of GMP inspections developers undergo.

    The average biopharmaceutical production facility has 2.68 good manufacturing practices (GMP) inspections a year, with auditors spending up to nine days on site per visit, according to recent analysis.

    Preparing for an inspection typically involves GAP analysis to determine how current practices measure up to regulations, followed by corrective actions.

    Companies also need to ensure they have the correct documentation for all operations. How long these preparatory steps take varies for each company. However, according to the U.S. Center for Professional Innovation and Education, getting set up for an audit can take anywhere from six months to a year.

    Down with duplication

    But drug companies should not have to undergo multiple GMP visits, according to the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), which says regulators can cut the number they carry out through collaboration.

    Sérgio Cavalheiro Filho, IFPMA’s regulatory affairs manager, tells GEN, “The most pressing compliance challenge relating to good manufacturing practice today is the inefficiency created by duplicative inspections.

    “In an increasingly complex and globalized manufacturing landscape, it is critical that we look to reduce unnecessary duplication through greater inspection reliance amongst those national regulatory agencies that belong to the Pharmaceutical Inspection Co-operation Scheme.”

    For the uninitiated, the Pharmaceutical Inspection Co-operation Scheme is an informal arrangement between regulators focused on GMP. Its key aims are to harmonize inspections and promote information sharing between regulators.

    It also aims to foster trust between regulatory agencies, with the idea being to encourage them to rely on GMP inspections carried out by fellow regulators rather than re-auditing sites themselves each time certification is sought.

    “Greater inspection reliance would allow both regulators and companies to focus resources where they matter most: patient safety and product development,” Filho says.

    IFPMA made the case for greater inspection reliance in a position paper, arguing that while pilot mutual recognition efforts have shown promise, regulators have yet to fully embrace the approach.

    Filho tells GEN, “Regulators have made meaningful progress on GMP harmonization through frameworks such as PIC/S and ICH, but more consistent use of inspection reliance is needed to translate alignment on paper into real efficiency.”

    Part of the problem is that advanced modalities, such as mAbs and cell and gene therapies, are often perceived as being higher risk, which means, despite the various mutual recognition agreements, regulators still tend to carry out their own inspections.

    However, in such cases, trusting others’ audits is a more efficient option, according to Filho, who says, “Relying on trusted regulatory partners where appropriate is a well‑tested and effective strategy that enables regulators to focus on higher‑risk activities. And, any steps to reduce the incidence of the GMP audits they face would be welcomed by biopharma, Filho adds.

    “Industry supports moving from pilots to routine reliance, underpinned by sound legal and data‑sharing frameworks. GMP challenges are also increasingly addressed through collaboration between manufacturers and technology suppliers, and through digitalization, automation, and AI‑enabled tools that strengthen monitoring and quality oversight within robust quality systems,” he says.

    The post Regulators Should Rely on Peers’ GMP Audits to Cut Inspection Burden appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Moss Powering the Next Drug Frontier https://edusehat.com/en/moss-powering-the-next-drug-frontier https://edusehat.com/en/moss-powering-the-next-drug-frontier Thu, 07 May 2026 00:20:12 +0700 Edusehat Moss, Powering, the, Next, Drug, Frontier For decades, Chinese hamster ovary (CHO) cells have been the gold standard for producing biologic drugs, from monoclonal antibodies to enzyme therapies. But for some of the most complex and fragile proteins, even CHO systems can fall short. Now, an unlikely contender—moss—is offering a new path forward.

    At Germany-based Eleva, researchers are using Physcomitrium patens, a simple moss species, as a suspension cell culture system for producing recombinant human proteins that are difficult and sometimes impossible to manufacture in conventional platforms. According to Andreas Schaaf, PhD, Eleva’s CSO, these include “glycoproteins with complex or sensitive glycosylation requirements,” as well as cytokines, immune-cytokines, complement regulators, enzymes for rare metabolic disorders, and advanced antibody formats such as antibody-toxin conjugates.

    The technology has deep academic roots. Plant biotechnologist Ralf Reski, PhD, at the University of Freiburg, helped develop P. patens into a model species for synthetic and systems biology and co-invented the moss bioreactor. His research led to the founding of Greenovation, now known as Eleva, which has since advanced the platform toward clinical-stage drug development.

    The company’s first moss-produced drug candidate to enter clinical studies was a recombinant alpha-galactosidase enzyme replacement therapy (ERT) for Fabry disease, a rare lysosomal storage disorder. Current ERT options for Fabry patients are limited by short circulating half-life, inefficient uptake into key affected cell types, and immunogenicity. Eleva believes the more uniform glycosylation achieved through moss production could help overcome these limitations.

    A particularly significant demonstration of the platform is Eleva’s recombinant complement Factor H candidate, currently in Phase Ib. Factor H is a large complement-regulatory glycoprotein used to target complement-related renal diseases such as C3 glomerulopathy (C3G), lupus nephritis (LN), and potentially dry age-related macular degeneration (AMD).

    Schaaf notes that Factor H “had long resisted reliable expression in conventional systems such as yeast or CHO cells.” For patients with C3G—many of whom are young and face a 50% rate of kidney failure within ten years—the ability to restore natural Factor H activity could represent a major therapeutic shift. Current treatments often rely on broader complement suppression and carry boxed warnings for serious infections.

    So why moss?

    Unlike mammalian cells, which often generate heterogeneous glycan mixtures, moss produces more uniform glycosylation profiles due to its simplified glycan-processing pathway. This matters because glycosylation can directly affect a drug’s stability, efficacy, and immunogenicity.

    Moss cells are also largely unaffected by toxic cytokine feedback, which in mammalian systems can inhibit growth or trigger apoptosis, limiting secretion efficiency and yields. Plant-specific chaperones and folding assistants, including protein disulfide isomerases, also help prevent protein aggregation and support the correct assembly of complex multimeric proteins.

    “Moss offers clear advantages over other expression systems for certain protein classes that are difficult or impossible to manufacture otherwise,” Schaaf says, adding that such therapies might otherwise be “deprioritized or abandoned.”

    There are practical manufacturing advantages, too. Moss requires no animal-derived media supplements, eliminating mammalian virus risk and removing the need for costly viral filtration in downstream processing. It is also less sensitive to fluctuations in temperature and pH, giving manufacturers greater process flexibility and potentially lowering production costs.

    Still, Eleva is careful not to position moss as a replacement for CHO. Björn Cochlovius, PhD, CEO of Eleva, says standard proteins will continue to be best served by established systems. “The goal is not to replace CHO or other systems with moss when those other systems deliver well,” he explains.

    Instead, the aim is to ensure that the range of therapeutic candidates in development is not defined by the limits of existing manufacturing platforms. Yields for large-scale GMP production and improving predictability remain ongoing challenges, but commercially viable titers are already being achieved through continuous optimization.

    Cochlovius believes regulatory precedent and growing CDMO partnerships will further strengthen adoption. “A moss-based platform capable of reliably producing this category of proteins at scale would open a pipeline of programs that are currently inaccessible,” he says.

    For biotech developers—and for patients with limited treatment options—that could make all the difference. Sometimes, the future of medicine grows in the smallest places.

    The post Moss Powering the Next Drug Frontier appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Researchers’ Spinout Focuses on Simplifying Viral Vector Purification https://edusehat.com/en/researchers-spinout-focuses-on-simplifying-viral-vector-purification https://edusehat.com/en/researchers-spinout-focuses-on-simplifying-viral-vector-purification Thu, 07 May 2026 00:20:11 +0700 Edusehat Researchers’, Spinout, Focuses, Simplifying, Viral, Vector, Purification U.S.-based researchers have developed a portfolio of peptide ligands for purifying viral vectors for gene therapies and have launched a company that develops affinity technologies for biopharmaceutical manufacturers.

    ChromaGenix, a spinout from North Carolina State University (NC State), commercializes peptide ligands as an alternative to the traditional protein ligands used in affinity chromatography.

    According to Stefano Menegatti, PhD, CSO at ChromaGenix and a professor in the Department of Chemical and Biomolecular Engineering at NC State, the ligands are cheaper and less likely to trigger an immune response in patients than the protein ligands traditionally used.

    “Protein ligands have been a fantastic enabler of advanced biological therapies over the past two decades, but they do have shortcomings,” he explains. “Proteins can denature or degrade, potentially releasing immunogenic fragments, which poses a certain level of risk.” As such, Menegatti says, protein ligands have a short lifetime and must be replaced frequently, adding further to production costs. They can also bind too strongly to the product, making it harder to recover it from the chromatographic step.

    By contrast, peptide ligands, which are very small proteins, overcome these issues, he says. They can be produced synthetically, making them cheaper than proteins. As they don’t have a complex structure, they can be cleaned under harsh conditions without becoming inactive. They have a longer lifespan and a much lower immunogenicity risk, Menegatti says. Also, as they’re small, he explains,they can be cleared during final product filtration.

    This “represents a new frontier of gene therapy manufacturing, says Menegatti, “as it boosts the efficiency of the viral vector manufacturing pipeline.”

    Having developed peptide ligands for a wide variety of viral vectors, ChromaGenix is already selling to many companies, Menegatti says. The researchers and the company are now hoping to move beyond gene therapies.

    “Our next chapter is going to be developing ligands for the purification of therapeutic cells, starting with CAR [chimeric antigen receptor] T cells,” he says.

    The post Researchers’ Spinout Focuses on Simplifying Viral Vector Purification appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Heavy-Chain BsAbs More Manufacturable than Light-Chains https://edusehat.com/en/heavy-chain-bsabs-more-manufacturable-than-light-chains https://edusehat.com/en/heavy-chain-bsabs-more-manufacturable-than-light-chains Thu, 07 May 2026 00:20:09 +0700 Edusehat Heavy-Chain, BsAbs, More, Manufacturable, than, Light-Chains Bispecific antibodies (BsAb) were first approved in 2014. Since then, a total of 19 have been approved globally, and approximately 250 BsAbs are being developed by some 180 companies, according to a report from Research and Markets. As this therapeutic class moves out of the lab and into clinical and commercial sectors, manufacturing may be its biggest challenge.

    These dual-targeting compounds are considered difficult to produce, especially at scale. With low yields, potential chain mispairing, stability and aggregation issues, and analytical characterization challenges, biomanufacturers are eager to find expedient solutions. “There is still significant work to be done in bioprocess engineering to substantially improve the efficiency of bispecific antibody design and manufacturing,” Laura A. Palomares, PhD, senior researcher, Universidad Nacional Autónoma de Mexico (UNAM), tells GEN.

    To that point, rather than relying upon transient expression systems or evaluating various antibody sequences, Palomares and her team are identifying manufacturable BsAbs by correlating their architectures to growth, productivity, downstream recovery, production of product-related variants, and in vitro binding to Zika virus and transferrin receptor (TfR). They found BsAb architecture is crucial in terms of those manufacturing criteria.

    Structure governs performance

    Specifically, as much as a 70% difference in productivity was found between symmetric heavy-chain scFv fusion BsAbs—which performed like the parental antibody—and BsAbs that were designed as light-chain scFv fusion, dual-variable domain immunoglobulin (DVD), or asymmetric antibodies. That’s according to a recent study by Palomares, doctoral student Juan Carlos Rivera-Castro, and senior researcher Octavio T. Ramirez, PhD.

    Asymmetric BsAbs had the worst culture performance and productivity of the BsAb architectures tested. Asymmetry created imbalanced chain expression and formed homodimeric and half-antibody by-products, they reported, which dropped purity to approximately 68% after protein A purification. They found that binding to the LC-scFV diminished the binding to Zika virus, and that DVD increases it. Also, binding to TfR varied according to BsAb valency and configuration.

    In contrast, “Bivalent heavy-chain scFV formats show[ed] stronger apparent binding than monovalent formats,” they report.

    For the best manufacturability, the team says, “Avoid the modification of the light chain and preserve symmetric assembly.” This strategy resulted in higher cell viability, productivity, and final purity.

    “The construction and head-to-head comparison of various formats, including the effect of the formats on antigen binding, can guide those planning the design and production of BsAbs,” Palomares says, by understanding the relative tradeoffs of various architectures as they design and clone BsAbs for specific functionalities.

    “Format selection should prioritize manufacturability, with complex designs reserved for cases with particular functional requirements,” the scientists conclude.

    Next steps, Palomares says, are to “determine the in vivo functionality of the constructed formats to neutralize Zika virus after traversing the blood-brain barrier. The results of those experiments will also be useful to scientists interested in BsAb design.”

    The post Heavy-Chain BsAbs More Manufacturable than Light-Chains appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Novelty Nobility Expands AGC Biologics Deal to Take Product Candidate Through GMP Manufacturing https://edusehat.com/en/novelty-nobility-expands-agc-biologics-deal-to-take-product-candidate-through-gmp-manufacturing https://edusehat.com/en/novelty-nobility-expands-agc-biologics-deal-to-take-product-candidate-through-gmp-manufacturing Wed, 06 May 2026 20:40:09 +0700 Edusehat Novelty, Nobility, Expands, AGC, Biologics, Deal, Take, Product, Candidate, Through, GMP, Manufacturing Korea-based Novelty Nobility expanded its agreement with CDMO AGC Biologics to advance their bispecific antibody drug candidate through process development and GMP manufacturing at AGC Biologics’ facility in Chiba, Japan. The project leverages AGC Biologics’ global network, having completed cell line development in Copenhagen, Denmark, to be followed by tech transfer to the Chiba site for the next manufacturing stages.

    “We believe this bispecific antibody has the potential to offer a truly differentiated treatment option for patients with neovascular retinal diseases,” said Sang Gyu Park, CEO of Novelty Nobility.

    The product candidate, NN4101, a first-in-class, connects a fully human anti-c-Kit monoclonal antibody with a vascular endothelial growth factor (VEGF) trap.

    “Our mammalian expression teams are adept at handling complex proteins, and we are a world leader in applying flexible, single-use bioreactor technology,” commented said Tadashi Murano, president of the AGC Life Science Company. “We are proud to support Novelty Nobility as they advance this innovative candidate toward the clinic.”

    To further support growing demand in the region and globally, AGC is also expanding its facility in Yokohama, Japan, which is designed to utilize single-use bioreactor technology to offer large-scale GMP manufacturing.

     

    The post Novelty Nobility Expands AGC Biologics Deal to Take Product Candidate Through GMP Manufacturing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Chromosome Engineering Reveals New Locus for Fusarium Resistance in Wheat https://edusehat.com/en/chromosome-engineering-reveals-new-locus-for-fusarium-resistance-in-wheat https://edusehat.com/en/chromosome-engineering-reveals-new-locus-for-fusarium-resistance-in-wheat Wed, 06 May 2026 09:35:12 +0700 Edusehat Chromosome, Engineering, Reveals, New, Locus, for, Fusarium, Resistance, Wheat Fusarium head blight (FHB) remains one of the most destructive diseases in global wheat production, and its impact is only intensifying. Warmer climates and crop rotations that favor pathogen survival have expanded the prevalence of FHB outbreaks, leading to major yield losses and contamination of grain with mycotoxins such as deoxynivalenol (DON), nivalenol (NIV), and zearalenone (ZEN).

    While fungicides offer partial control, reduced sensitivity and rising costs have made genetic resistance in wheat the most sustainable long‑term strategy. Yet despite decades of breeding, only a handful of major FHB resistance loci—Fhb1 through Fhb9—have been formally designated, and just two have been cloned. The scarcity of strong, deployable resistance genes has become a bottleneck for wheat improvement.

    A new study published in the Journal of Experimental Botany, “Identification of a novel Fusarium head blight resistance locus Fhb.Er‑1StL from Elymus repens introgressed into wheat,” expands that genetic toolkit. Researchers at Sichuan Agricultural University report the discovery of a previously unknown FHB resistance locus, Fhb.Er‑1StL, derived from the wild grass Elymus repens—a species better known as an agricultural weed than a genomic resource.

    “Both research and breeding practice have shown that developing and deploying resistant wheat cultivars is the fundamental solution to FHB,” said first author Fei Wang. “However, current efforts are limited by a scarcity of major resistance sources, narrow genetic backgrounds, and inefficient use of resistance genes.”

    The team began by characterizing the genome of a wheat E. repens partial amphidiploid, P1142‑1‑2, which carries the full wheat genome plus seven pairs of alien chromosomes or chromosome fragments. Using sequential GISH and FISH cytogenetics, they mapped the alien chromatin and identified a pair of chromosomes containing the long arm of the E. repens 1St chromosome. From crosses with the susceptible wheat cultivar Chuannong16, they isolated two derivative lines carrying either a 1StL isochromosome or a 1StL telosome, both of which conferred strong resistance to FHB.

    To pinpoint the resistance locus, the researchers applied a targeted sequencing approach using the Wheat–St 45K liquid microarray GBTS platform. This allowed the researchers to precisely identify the alien 1StL segment and develop markers to track it in breeding lines. Plants carrying this segment showed markedly improved resistance, and molecular assays confirmed that the region represents a previously unknown FHB resistance locus, now designated Fhb.Er‑1StL.

    “We believe this work is of practical importance for accelerating the breeding of resistant, high‑yielding wheat varieties and breaking the bottleneck in FHB resistance breeding,” said senior author Yinghui Li, PhD.

    Next steps include fine‑mapping the locus and generating smaller translocation lines to reduce linkage drag—an essential step before the trait can be widely deployed in commercial breeding.

    “With the aid of modern genomic technologies and precise breeding strategies, Fhb.Er-1StL holds promise as a cornerstone for developing next-generation wheat cultivars with durable resistance to FHB,” concluded the authors.

    The post Chromosome Engineering Reveals New Locus for Fusarium Resistance in Wheat appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     UCB to Acquire Candid for up to $2.2B, Expanding Presence in TCE Antibodies for Immunology https://edusehat.com/en/ucb-to-acquire-candid-for-up-to-22b-expanding-presence-in-tce-antibodies-for-immunology https://edusehat.com/en/ucb-to-acquire-candid-for-up-to-22b-expanding-presence-in-tce-antibodies-for-immunology Wed, 06 May 2026 09:35:11 +0700 Edusehat UCB, Acquire, Candid, for, 2.2B, Expanding, Presence, TCE, Antibodies, for, Immunology UCB has agreed to acquire Candid Therapeutics for up to $2.2 billion, the companies said, in a deal to expand the buyer’s presence in T-cell engager (TCE) antibodies designed for immunology indications by adding Candid’s pipeline of bispecific and trispecific antibody candidates.

    The deal upends plans announced in March by Candid to enter a reverse merger with Rallybio, in which Rallybio would have acquired Candid but retained Candid’s name and created new shares to be traded on NASDAQ. The new company was to have developed Candid’s pipeline using $505 million in concurrent financing from a syndicate of healthcare institutional investors and mutual funds.

    Based in San Diego, privately held Candid’s pipeline of autoimmune and inflammatory disease candidates includes treatments licensed from Chinese biotechs.

    Candid’s lead asset, cizutamig, is a bispecific antibody for autoantibody-driven autoimmune diseases. Licensed from Shanghai-based EpimAb Biotherapeutics, cizutamig is directed to B-cell maturation antigen (BCMA) on plasma cells and CD3 on T cells, with the aim of enabling T-cell–mediated cytotoxicity against both kinds of cells while limiting cytokine release. Cizutamig is currently in multiple Phase I clinical studies in over 10 autoimmune indications, with clinical evaluation in more than 100 patients with multiple myeloma (completed with 40 patients) and autoimmune diseases (68 patients across multiple indications in China and Europe).

    Also in Phase I development within Candid’s pipeline is CND261, a CD20 x CD3 bispecific antibody TCE that the company licenses from Shanghai-based Genor Biopharma. CND261 is being developed to treat autoimmune diseases by targeting a variety of B-cell subtypes, from pro-B-cells to plasmablasts/plasma cells. Candid has completed a 93-patient Phase I dose escalation study of CND261 in non-Hodgkin’s lymphoma (NHL).

    The rest of Candid’s pipeline consists of two preclinical candidates in IND-enabling studies:

    • CND319, a CD19xCD20xCD3 trispecific antibody designed to target the CD19 and CD20 antigens on a broad range of B-cell subtypes, and licensed from WuXi Biologics last year for up to $925 million in upfront and milestone payments, plus royalties.
    • CND460, a BCMAxCD19xCD3 trispecific antibody designed to target the BCMA and CD19 antigens on a broad range of B-cell subtypes.

    CND261, CND319, and CND460 represent a pipeline of multi-specific TCE antibodies designed to enable deep, targeted depletion of pathogenic B cell populations in immune-mediated diseases to achieve immune reset—what UCB termed a modular, multi-antigen targeting strategy to address complementary B-cell subsets.

    “We started Candid with the goal to redefine the standard of care for immune-mediated diseases. We purposefully built a broad portfolio of TCE assets against a number of clinical indications,” stated Ken Song, MD, Candid’s chairman, CEO, and president. Previously, as president, CEO, and board director of radiopharmaceutical developer RayzeBio, Song negotiated the approximately $4.1 billion sale of the company to Bristol Myers Squibb, completed in 2024.

    “Our focus has been to efficiently generate clinical data so as to identify where our TCEs could provide maximal clinical benefit for the broadest number of patients,” Song explained.

    Investors appeared less enthusiastic about the deal. UCB stock is traded primarily on Euronext Brussels, where the company’s shares barely budged Monday, dipping 0.65% to €228.30 ($267.28) as of 10:37 am ET.

    Platform-based strategy

    UCB’s plan to acquire Candid comes roughly two months after the Belgian biotech giant agreed to license exclusive global rights to further develop, manufacture, and commercialize Hong Kong-based Antengene’s ATG-201, a CD19 and CD3 bispecific TCE antibody designed to target B cell-related autoimmune diseases. UCB agreed to pay Antengene $80 million in upfront and near-term milestone payments, plus up to approximately $1.1 billion in payments tied to achieving development and commercial milestones under the agreement, which also granted UCB access to Antengene’s associated manufacturing technology in relation to ATG-201.

    UCB said its planned acquisition of Candid, plus the Antengene deal, reflects a platform-based strategy of complementary investments intended to expand its reach across multiple B-cell targets and disease mechanisms, thus strengthening its ability to address antibody-mediated autoimmune diseases through multiple approaches rather than relying on a single asset or modality.

    “This acquisition demonstrates our inorganic innovation strategy in action and marks a pivotal moment for UCB, as we secure a significant technological advancement in the field with the addition of cizutamig to our pipeline,” UCB CEO Jean-Christophe Tellier said in a statement. “This exemplifies the next wave of therapies to treat immune-mediated diseases and reflects our commitment to setting new standards to achieve immune reset.

    Tellier added that UCB considers cizutamig “a potential transformative asset, that complements our existing programs, and is poised to redefine treatment expectations for severe, underserved immune-mediated diseases, offering the potential to deliver meaningful improvements in patient outcomes and quality of life.”

    UCB has agreed to pay Candid $2 billion upfront and up to $200 million in potential future milestone payments. The transaction is subject to closing conditions that include antitrust clearance and other customary conditions and is expected to close by the end of the second quarter or early Q3 2026.

    The deal is good news for Two River and Vida Ventures, funds that played central roles in the creation and early development of Candid, which was launched in 2024 with more than $370 million in capital. Two River, together with Third Rock Ventures, helped found Candid, which was created through the merger of Two River-founded TRC 2004 and Vignette Bio.

    “This outcome reflects the power of bringing together bold science, disciplined company building, and the right strategic partners,” said Arie Belldegrun, MD, founder and senior managing director of Vida Ventures, chairman of Two River, and co-founder of Bellco Health.

    UCB added that it expects the anticipated financial impact of the Candid acquisition to be “manageable.” The company has not changed its most recent 2026 guidance, which calls for revenue growth in the high single‑digit to low double‑digit range at constant exchange rates, while underlying profitability, measured by “adjusted” earnings before interest, taxes, depreciation, and amortization (EBITDA), which excludes one-time expenses, is expected to increase in the high single‑digit to mid‑teens range.

    “UCB’s successful track record in immunology, including development, launch, and commercialization, will enable the continuation of our clinical programs and help deliver on the potential for our pipeline,” Song added.

    The post UCB to Acquire Candid for up to $2.2B, Expanding Presence in TCE Antibodies for Immunology appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Psilocybin-Induced Brain Changes May Explain Therapeutic Effects https://edusehat.com/en/psilocybin-induced-brain-changes-may-explain-therapeutic-effects https://edusehat.com/en/psilocybin-induced-brain-changes-may-explain-therapeutic-effects Wed, 06 May 2026 06:00:09 +0700 Edusehat Psilocybin-Induced, Brain, Changes, May, Explain, Therapeutic, Effects Researchers at University of California, San Francisco and Imperial College London have shown that a single dose of psilocybin, the psychedelic compound found in magic mushrooms, causes likely anatomical brain changes that last for up to a month after the experience.

    The study, involving healthy volunteers who had never taken a psychedelic, links temporary shifts in brain “entropy”—which is the diversity of neural activity occurring in the brain—to insight. This suggests the psychedelic trip itself is important to the drug’s longer term therapeutic effects.

    The researchers found that a high dose of psilocybin led to increased entropy in the minutes and hours after taking the drug. The degree of entropy predicted how much insight, or emotional self-awareness, the participants felt the next day; and this, in turn, forecasted improvements in their sense of wellbeing a month later.

    The findings may help to explain psilocybin’s therapeutic effects on conditions such as depression, anxiety, and addiction. “Psychedelic means ‘psyche-revealing,’ or making the psyche visible,” said senior author Robin Carhart-Harris, PhD, the Ralph Metzner distinguished professor of neurology at UCSF. “Our data shows that such experiences of psychological insight relate to an entropic quality of brain activity and how both are involved in causing subsequent improvements in mental health. It suggests that the trip—and its correlates in the brain—is a key component of how psychedelic therapy works.”  Carhart-Harris is senior and corresponding author of the team’s published paper in Nature Communications, titled “Human brain changes after first psilocybin use.”

    “Psychedelics have robust effects on acute brain function and long-term behavior but whether they also cause enduring functional and anatomical brain changes is largely unknown,” the authors wrote. Psilocybin is the precursor of the compound psilocin, a serotonin receptor agonist. “Converging evidence supports a role for serotonin 2A receptor  (5-HT2AR) agonism in eliciting the characteristic brain and subjective effects of this and related psychedelics in humans,” the team continued.

    For their newly reported study, Carhart-Harris and colleagues carried out an exploratory, placebo-controlled, within-patient study in 28 psychedelic-naïve participants who each received a single, high-dose (25 mg) of psilocybin. The researchers used an assortment of brain imaging and brain measurement techniques, some of which were carried out during the peak of the psychedelic experience, as well as before and one-month after drug administration. “This was an exploratory, hypothesis-generating mechanistic study in healthy volunteers,” the authors noted. None of the 28 people in the study had a diagnosed mental health condition, which gave the scientists greater freedom to do more testing.

    In the first part of the experiment the subjects were given a 1 mg dose of psilocybin, which the researchers regarded as a placebo, and were then monitored with EEG, which records brain activity from electrodes on the scalp.  Over the next few weeks, the researchers measured their subjects’ psychological insight, wellbeing, and cognitive ability. They examined brain activity with functional MRI (fMRI) and brain connectivity with diffusion tensor imaging (DTI).

    One month after the placebo, the subjects were given 25 mg of psilocybin, a dose capable of eliciting a strong psychedelic trip. During the experience, researchers again measured the subjects’ brain activity with EEG, and in the following weeks they repeated the same tests they had given after the 1 mg dose.

    This enabled the scientists to compare the effects of the psychedelic trip on the brain and mind to the effects of the placebo. “The multimodal neuroimaging design allowed us to observe changes in brain function and (potential) anatomy from 1-h (EEG) to 1-month (DTI) after high-dose psilocybin,” they explained.

    The investigators found that within 60 minutes of taking the 25 mg dose of psilocybin, EEG revealed higher entropy, suggesting that the brain was processing a richer body of information under the psychedelic. A month later, the researchers looked at their subjects’ brains using DTI, which measures the diffusion of water along neural tracts in the brain, and found that they were denser and had more integrity. This is the opposite of what happens in aging, which makes these tracts more diffuse.

    The researchers cautioned that more work needs to be done to better understand the meaning of this finding, but the result is a never-before-seen sign of how psychedelics can change the brain. ”The inclusion of DTI enabled us to test for long-term changes in the integrity of white matter tracts post psilocybin,” the authors stated. “Results revealed decreased axial diffusivity in prefrontal-subcortical tracts 1-month post 25mg psilocybin.”

    The day after the 25 mg dose, all but one of the 28 subjects rated the trip as the “single most” unusual state of consciousness they had ever experienced. The remaining person rated it as among their top five. The study participants said they had experienced more psychological insight after taking the 25 mg of psilocybin than they had after the 1 mg placebo.  The subjects also reported increased wellbeing two and four weeks after the study. This was measured from responses to statements such as, “I’ve been feeling optimistic about the future,” and “I’ve been dealing with problems well.”

    As the scientists noted in their paper, “A predictive relationship was also found between brain entropy and longer-term mental-health changes—namely, improved wellbeing. Improved wellbeing could be predicted directly from acute increases in brain entropy as early as 1-h post dosing.”

    A month after the study the study individuals also scored better on a test of cognitive flexibility.  “Psilocybin seems to loosen up stereotyped patterns of brain activity and give people the ability to revise entrenched patterns of thought,” said first author Taylor Lyons, PhD, a research associate at Imperial College London. “The fact that these changes track with insight and improved well‑being is especially exciting.”

    The scientists found that the subjects who had experienced the largest increases in brain entropy in the minutes to hours after taking psilocybin were the most likely to have increased insight the next day and increased wellbeing a month later. The researchers concluded that improved wellbeing was driven by the experience of insight.

    The authors suggest that the study findings could improve treatment for people with mental illness using psilocybin, for example, by ensuring that the right dosage is used to produce the right amount of brain entropy to promote insight. “We already knew psilocybin could be helpful for treating mental illness,” Carhart-Harris said. “But now we have a much better understanding of how.”

    In their paper the team concluded, “The present multi-modal neuroimaging study in healthy participants sheds light on the brain effects of first-time high-dose psychedelic use and the therapeutic action of psilocybin-therapy, suggesting that therapeutically relevant changes—i.e., improved wellbeing—can be forecast via an acute human brain action, i.e., an entropic brain effect, that is well-known to relate to the psychedelic experience … Results support a role for psychological insight in mediating the causal association between the entropic brain effect and potentially enduring improvements in wellbeing.”

    The post Psilocybin-Induced Brain Changes May Explain Therapeutic Effects appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     New Human-Computer Interaction Software Designed to Support Diverse Online Teamwork Styles https://edusehat.com/en/new-human-computer-interaction-software-designed-to-support-diverse-online-teamwork-styles https://edusehat.com/en/new-human-computer-interaction-software-designed-to-support-diverse-online-teamwork-styles Tue, 05 May 2026 22:45:16 +0700 Edusehat New, Human-Computer, Interaction, Software, Designed, Support, Diverse, Online, Teamwork, Styles Remote collaboration software tools, such as Zoom or Google Docs, have become essential for teamwork. But they often overlook the fact that people do not all approach collaboration in the same way, according to researchers at North Carolina State University (NCSU).

    Scientists report that they have now developed a new human-computer interaction (HCI) method called RemoteCollabEval (RCE) to identify barriers to collaboration and inclusivity, allowing designers and developers to build software features that better support diverse teamwork styles.

    The work is part of the broader HCI field, which examines how people use digital systems and how interfaces can be optimized for clarity and ease of use.

    “At present, most remote collaboration platforms are evaluated by designers and developers using established HCI inspection methods,” says Sandeep Kuttal, PhD, the principal investigator behind the work and an associate professor of computer science at NCSU. “One of the most widely used inspection methods is a ‘groupware walkthrough,’ where designers essentially play out how a collaborative effort might unfold between two or three hypothetical users. However, these methods typically assume all users behave in similar ways.”

    As senior author of a paper, “Equity by Design: A New HCI Method for Surfacing Inclusivity Issues in Remote Collaboration Software,” that will be presented at the ACM Designing Interactive Systems Conference (DIS 2026) in Singapore, from June 13-17, Kuttal notes that “It’s well-established that people from various backgrounds often have different collaboration and communication styles. “Existing HCI inspection methods don’t account for these differences, which limits how inclusive and effective these tools can be. That’s what we set out to address.”

    Six key personality facets

    As a first step, the researchers drew on established social science and software engineering research to identify six key personality facets that influence collaborative behavior:

    • Leadership style: Does the individual take a democratic or authoritative approach?
    • Interruption style: Does the individual interrupt others or wait for cues?
    • Non-verbal cues: Is the individual expressive or reserved in digital spaces?
    • Relationship-seeking: Does the individual focus on building rapport or primarily on achieving goals?
    • Social awareness: Is the individual attentive to or unaware of what their teammates are doing?
    • Collaborative self-efficacy: How confident is the individual in the group’s ability to perform?

    The researchers then created hypothetical users called “personas,” which are detailed representations of different types of users that incorporate descriptions of each of the six facets. These personas allow designers to simulate interpersonal friction and uncover “inclusivity bugs” that might otherwise go unnoticed during standard testing.

    “Because we have descriptions of all six facets for each persona, we can incorporate those key characteristics into our assessment of how well a given platform allows for effective collaboration between people of different backgrounds,” explains Kuttal.

    The team then modified existing groupware walkthrough methods, requiring designers and developers to explicitly consider these six facets as part of the process and created a specialized walkthrough. This combination of personas that account for personality facets and the specialized walkthrough forms the RCE method.

    As a proof-of-concept study, the scientists recruited 29 undergraduate and graduate students and split them into 10 teams. Five teams inspected an existing remote collaboration platform using the conventional Groupware Walkthrough method; the other five teams inspected the same platform using RCE.

    “The teams who used the RCE method identified six times more inclusivity issues than the conventional method,” continues Kuttal. “Essentially, RCE did a better job of identifying when conflicting styles would make collaboration between personas difficult. This is important, because identifying these challenges gives designers and developers an opportunity to modify features and user interfaces to improve these remote collaborative platforms. And, ultimately, to improve collaboration itself.

    “Because RCE is a standardized, systematic method, it can be used by designers and developers anywhere. It doesn’t require a huge budget, or an expensive research effort. It’s a method that can easily be used to make these platforms better.”

    The post New Human-Computer Interaction Software Designed to Support Diverse Online Teamwork Styles appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Blood Stem Cells Evade Immune Attack in Aplastic Anemia Through Gene Mutations https://edusehat.com/en/blood-stem-cells-evade-immune-attack-in-aplastic-anemia-through-gene-mutations https://edusehat.com/en/blood-stem-cells-evade-immune-attack-in-aplastic-anemia-through-gene-mutations Tue, 05 May 2026 08:25:10 +0700 Edusehat Blood, Stem, Cells, Evade, Immune, Attack, Aplastic, Anemia, Through, Gene, Mutations Scientists headed by a team at St. Jude Children’s Research Hospital have found that in individuals with the life-threatening blood disorder aplastic anemia (AA), different blood stem cells within the same person independently acquire gene mutations that allow cells to escape the immune attack. Through their study, the team, together with collaborating institutions, used state-of-the-art genomic techniques to profile 619 children and adults with AA. The study showed that for some patients, these “rescuing” stem cell clones were enough to restore blood production and provide long-term remission.

    “We found that each patient with aplastic anemia that escapes autoimmunity has multiple, independent genetic events in different blood stem cells that allow those cells to escape autoimmunity,” said Marcin Wlodarski, MD, PhD, St. Jude Department of Hematology. “Stem cells silence the risk HLA allele through several mechanisms, and our data show that these events are protective, benign events that don’t cause progression to MDS or leukemia, even when the rescued clones grow and dominate the bone marrow.”

    Corresponding author Wlodarski and colleagues reported on the study, which they say includes the largest pediatric cohort of its kind reported to date, in Nature Genetics. In their paper titled “High-resolution single-cell mapping of clonal hematopoiesis and structural variation in aplastic anemia,” the team wrote, “These findings reveal parallel evolutionary pathways used by hematopoietic cells to evade immune attack.”

    Aplastic anemia is a rare, life-threatening bone marrow failure (BMF) syndrome where patients are unable to make enough blood cells due to the immune system’s attack on hematopoietic stem and progenitor cells (HSPCs). The condition can progress to myelodysplastic syndrome (MDS) and leukemia.

    In AA, autoreactive T cells target and destroy blood stem cells that display peptides on a specific protein on their surface. These are encoded by the human leukocyte antigen (HLA) gene. Each person inherits one copy of this gene from each parent, which can have different variations. People with aplastic anemia often carry a particular “risk” HLA allele (gene variant) that is thought to trigger the disease. As the authors noted, “While the precise mechanism underlying HSPC recognition by autoimmune T cells remains elusive, specific human leukocyte antigen (HLA) alleles are overrepresented in patients with AA compared with healthy controls, suggesting a role in aberrant immune recognition.”

    Some blood stem cells evade the immune attack by acquiring changes that silence the risk HLA allele. This can happen via loss-of-function HLA mutations or through uniparental isodisomy 6p (UPD6p), where the risk allele is replaced with a non-risk allele. “HLA loss, manifesting as uniparental disomy of chromosome 6p (UPD6p) or loss-of-function (LOF) mutations in HLA, is postulated to inactivate HLA risk alleles (presumed to mediate autoantigen presentation), effectively shielding HSPCs from autoimmune attack,” the investigators noted. Two other types of escape in blood stem cells are known: paroxysmal nocturnal hemoglobinuria (PNH) or mutations in clonal hematopoiesis (CHIP) genes. However, it was unclear if all these changes arise in a single stem cell or arise independently to help the blood stem cells hide from the immune system. It was also unclear how this process of immune evasion impacted clinical outcomes and cancer risk.

    “The clinical implications of clonal alterations in AA vary,” the investigators stated. “HLA loss is generally considered a nonmalignant adaptive lesion, large PNH clones require complement inhibitor therapy, and CHIP-mutant clones may be associated with MDS, thereby necessitating hematopoietic stem cell transplantation (HSCT).”

    (L to R) Corresponding author Marcin Wlodarski, MD, PhD, and lab member Diantha Van De Vlekkert, MSc, both of the St. Jude Department of Hematology, and second author Sushree Sahoo, PhD, formerly of the St. Jude Department of Hematology. [St. Jude Children's Research Hospital]
    (L to R) Corresponding author Marcin Wlodarski, MD, PhD, and lab member Diantha Van De Vlekkert, both of the St. Jude Department of Hematology, and second author Sushree Sahoo, PhD, formerly of the St. Jude Department of Hematology. [St. Jude Children’s Research Hospital]
    Blood stem cells give rise to all other blood cells, meaning their progeny are genetically identical, including any mutations gained over time. The relative abundance of a specific stem cell’s genetic “clones” measures the genetic diversity of these blood-making cells. Using single-cell analyses, the researchers showed that protective mutations happen independently in different blood stem cells and not sequentially within a single cell. These independent clones then repopulate the marrow without being found and killed by the immune system. “We saw that patients with blood stem cell clones that escape autoimmunity can improve their blood counts,” Wlodarski said. “We also learned that these clones do not indicate an increased risk for leukemia. On the contrary, they often indicate the possibility of long-lasting remission.”

    To assess these clones, the scientists analyzed bone marrow and blood samples from 619 (256 children and 363 adults) patients with AA. “We present a high-resolution genomic landscape in AA patients using single-cell targeted DNA/protein sequencing, PacBio long-read whole-genome sequencing (WGS), and single-cell WGS,” they explained. They found that overall, 69% of patients carried at least one acquired change: HLA mutations or UPD6p clones were found in 16%, PNH clones in 44%, and CHIP mutations in 21%.

    First author Masanori Yoshida, MD, PhD, St. Jude Department of Hematology, then established and applied a single-cell DNA sequencing assay to simultaneously profile mutations and cell-surface proteins of 304,902 single cells from 48 samples. The study was complemented by long-read whole-genome sequencing and single-cell whole-genome sequencing.

    The experiments showed that acquired mutations are just as common in children as in adults, but in pediatric patients, 65% of the CHIP mutations occurred in just three genes (BCOR, BCORL1, and ASXL1), compared with 27% in adults. Because age-related CHIP mutations are not expected to preexist in children, these mutations seem to be immune-escape events acquired in response to the autoimmune attack. “In children, where preexisting CHIP is not expected, mutations in these three genes may represent bona fide immune escape mechanisms arising in direct response to T-cell-mediated attack,” the authors stated.

    To understand how these protective events arise and to count them precisely, the authors performed whole-genome sequencing on many single blood stem cells. They expected to see one to three events per individual; instead, they found a median of three per patient, and in one patient, 15 independent clones, all resulting in the loss of the risk HLA allele, showing convergent evolution to escape a strong immune attack. “Strikingly, HLA loss clones emerge independently through mutational events that converge on inactivating a single specific HLA risk allele, with up to 15 clones per patient identified using the scWGS platform … Our analyses reveal that somatic alterations in AA arise as independent clones rather than through sequential acquisition, and most patients carry multiple independent clones,” the investigators noted.

    That extreme diversity pointed to an unusual, convergent evolutionary process, so the scientists reconstructed a phylogenetic “family tree” of individual blood stem cells by reading all mutations acquired throughout life in single whole genomes. This method enabled them to pinpoint each clone’s origin. “We had expected that these mutations occur right before disease onset,” Wlodarski said. “But we found some of these HLA-loss clones arose many years before clinical diagnosis.”

    The team also showed that long-lived, rescued clones had higher expression of CD34, a surface marker for blood stem and progenitor cells. This suggests that CD34 enrichment could serve as a biomarker of long-lasting recovery. In addition, clones with loss of HLA risk alleles and CHIP mutations almost never co-occurred in the same cells, indicating that HLA loss provides enough of a proliferative advantage on its own that additional CHIP mutations, which can predispose to MDS, are not selected. So, they appear to act as protective events against their MDS and leukemia evolution.

    “Clones with higher CD34+ expression levels measured in our scDNAseq/protein analysis, particularly those with HLA-loss alterations, demonstrated long-term fitness, multilineage contribution, and were often associated with stable blood counts and prolonged treatment-free intervals,” the team pointed out. These results challenge prior assumptions about when and how protective clones arise in aplastic anemia, and their presence can be a factor in restoring blood formation.

    “Aplastic anemia shows us convergent evolution in miniature: Multiple independent mutational events arise in different cells, all leading to the same escape from autoimmunity,” Wlodarski said. “It shows the amazing nature of human hematopoiesis to cure itself from bad actors, like the autoimmune T cells, and reconstitute the bone marrow.” In their paper, the team concluded, “These findings enhance our understanding of clonal dynamics in AA and provide a foundation for future precision medicine approaches to address BMF in this life-threatening syndrome.”

    The post Blood Stem Cells Evade Immune Attack in Aplastic Anemia Through Gene Mutations appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Gene Syntax Determines DNA Supercoiling and Modulates Gene Expression https://edusehat.com/en/gene-syntax-determines-dna-supercoiling-and-modulates-gene-expression https://edusehat.com/en/gene-syntax-determines-dna-supercoiling-and-modulates-gene-expression Tue, 05 May 2026 08:25:09 +0700 Edusehat Gene, Syntax, Determines, DNA, Supercoiling, and, Modulates, Gene, Expression When synthetic biologists sketch gene circuits, they usually think in terms of promoters, repressors, and transcription factors—biochemical parts that toggle genes on or off. But DNA is not a flat schematic. It’s a physical polymer that twists, coils, and buckles as genes are transcribed. A pair of new papers from MIT and collaborators shows that this physicality could suggest approaches to controlling the output of gene circuits.

    In a recent Science study titled “Gene syntax defines supercoiling-mediated transcriptional feedback,” researchers demonstrate that the order and orientation of neighboring genes—what they call gene syntax—can reshape local DNA supercoiling and, in turn, amplify or suppress the expression of adjacent genes.

    “Syntax will be really useful for dynamic circuits. Now we have the ability to select not only the biochemistry of circuits, but also the physical design to support dynamics,” said Katie Galloway, PhD, an assistant professor of chemical engineering at MIT.

    The team engineered human cell lines and hiPSCs with synthetic two‑gene reporter circuits arranged in tandem, divergent, or convergent configurations. Their earlier modeling predicted that divergent syntax should boost the expression of both genes, while tandem syntax should suppress the downstream gene. “The thing that we were trying to solve in this paper was: When you put two genes on the same piece of DNA, how does their physical interaction become coupled?” said Galloway. The experimental results matched those predictions: divergent circuits amplified both genes, while tandem circuits showed strong upstream‑to‑downstream repression, with effects reaching up to 25‑fold.

    To understand why, the researchers used Region Capture Micro‑C, a high‑resolution genome‑folding mapping technique, to visualize how transcription reshapes DNA. When a gene was activated, the DNA downstream tightened into plectonemes—twisted structures that hinder RNA polymerase binding—while upstream DNA loosened. “Supercoiling impacts transcription of adjacent genes by altering RNA polymerase binding, forming a feedback loop,” the authors of the first paper wrote.

    The second paper, published in Nature Biomedical Engineering and titled “STRAIGHT-IN Dual: a platform for dual single-copy integrations of DNA payloads and gene circuits into human induced pluripotent stem cells,” introduced STRAIGHT‑IN Dual, a platform that enables simultaneous, allele‑specific, single‑copy integration of two DNA constructs into hiPSCs. This system allowed the team to “investigate how promoter choice and gene syntax influence transgene silencing and how these design features affect reporter expression and forward programming of hiPSCs into neurons, motor neurons, and endothelial cells,” according to the authors of the second paper.

    Using STRAIGHT‑IN Dual, the researchers also demonstrated a practical application: a divergent circuit expressing two components of a yellow fever antibody produced higher output than other configurations.

    “This is really exciting because we can coordinate gene expression in ways that just weren’t possible before,” Galloway said. “Now that we understand the syntax, I think this will pave the way for us to program dynamic behaviors.

    “If you want coordinated expression, a divergent circuit is great. If you want something that’s either/or, you can imagine using a convergent or tandem circuit, so when one turns on, the other turns off, and you can alternate pulses,” Galloway added.

    The post Gene Syntax Determines DNA Supercoiling and Modulates Gene Expression appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Optical Pooled CRISPR Screen Reveals Regulators of NF-κB Dynamics in Human Cells https://edusehat.com/en/optical-pooled-crispr-screen-reveals-regulators-of-nf-kb-dynamics-in-human-cells https://edusehat.com/en/optical-pooled-crispr-screen-reveals-regulators-of-nf-kb-dynamics-in-human-cells Tue, 05 May 2026 04:50:11 +0700 Edusehat Optical, Pooled, CRISPR, Screen, Reveals, Regulators, NF-κB, Dynamics, Human, Cells

    Image of Tilmann Buerckstuemmer, PhD

    Tilmann Buerckstuemmer, PhD

    CSO
    Myllia Biotechnology

    Panelist

    Image of Tilmann Buerckstuemmer, PhD

    Tilmann Buerckstuemmer, PhD

    Tilmann Buerckstuemmer, PhD, is a CRISPR enthusiast since the early days of CRISPR. Originally trained as a biochemist, he joined Haplogen as principal scientist and later became their CSO. Following the acquisition by Horizon Discovery, Tilmann served as director of research and development and later as head of innovation, where he oversaw the company’s technology platform and innovation agenda. In 2018, he co-founded Myllia Biotechnology which focuses on single-cell CRISPR screens. He is also the CEO of bit.bio discovery, a joined venture between Vienna-based Myllia Biotechnology and Cambridge-based bit.bio. Tilmann is passionate about science and enjoys working with multi-disciplinary and multi-national teams.
    Image of Jens Durruthy Durruthy, PhD

    Jens Durruthy Durruthy, PhD

    Director of Product Management
    Element Biosciences

    Panelist

    Image of Jens Durruthy Durruthy, PhD

    Jens Durruthy Durruthy, PhD

    Jens Durruthy Durruthy, PhD, is the director of product management at Element Biosciences. Prior experience includes a decade at 10x Genomics, where he developed and oversaw the product portfolio for Chromium products. Jens held the position of LSA Bio/Genomics Fellow at Life Science Angels, conducting extensive research on investment opportunities in biotech and genomics startups, and has worked in various consulting roles, focusing on product development and market analysis. Educational credentials include a PhD in biomedical engineering from Stanford University and a diploma in medical biotechnology from Technische Universität Berlin.

    Broadcast Date: 
    • Time: 

    Integrated pooled CRISPR screening linked to imaging readouts accelerate target identification and functional characterization of signaling pathways. A good example of this can be found in studies of NF-κB signaling, which is central to inflammatory responses and driven by rapid nuclear translocation of the p50/p65 complex to activate transcriptional programs following cytokine stimulation.

    In this GEN webinar, Tilmann Buerckstuemmer, PhD, CSO at Myllia Biotechnology will show how high-throughput pooled CRISPR screening combined with cell painting readouts characterized important signaling pathways using NF-κB nuclear translocation as a case study. During the webinar, you will learn how the AVITI24™ platform from Element Biosciences profiled ~440,000 cells in a pooled CRISPR screen targeting 195 genes. Linking genetic perturbations to p65 subcellular localization and cell painting features in a single workflow enabled identification of known pathway components, uncovered regulatory roles for chromatin-modifying complexes, and improved interpretation of phenotypic outcomes using morphological features.

    Key takeaways include:

    • Strategies for linking CRISPR perturbations to protein localization and morphological features at single-cell resolution

    • Identification of hitherto poorly characterized chromatin modifying complexes in regulating NF-κB signaling

    • The value of multimodal readouts, including morphology, in adding depth and confidence to recovered biology

    • How this approach supports mechanism-of-action studies and enables identification of both positive and negative regulators of signaling pathways

    A live Q&A session will follow the presentation offering you a chance to pose questions to our expert panelists.

    Produced with support from:

    Element Bio logo

    The post Optical Pooled CRISPR Screen Reveals Regulators of NF-κB Dynamics in Human Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     TRACS Enables Strain-Level Tracking of Microbial Transmission https://edusehat.com/en/tracs-enables-strain-level-tracking-of-microbial-transmission https://edusehat.com/en/tracs-enables-strain-level-tracking-of-microbial-transmission Tue, 05 May 2026 04:50:10 +0700 Edusehat TRACS, Enables, Strain-Level, Tracking, Microbial, Transmission

    Tracking microbes is challenging, particularly when there are coexisting strains of the same species within metagenomic data. However, overcoming that challenge is important for inferring transmission of both pathogenic and commensal microbes.

    A new tool, called TRAnsmision Clustering of Strains (TRACS), distinguishes between closely related bacterial strains. The “highly accurate algorithm” can be used for “estimating genetic distances between strains at the level of individual single nucleotide polymorphisms, which is robust to intra-species diversity within the host.”

    Researchers used the TRACS tool to map the transmission of SARS-CoV-2, Streptococcus pneumoniae, and Plasmodium falciparum (the causative agent of malaria) across different populations. The tool may play an important role in infection prevention, outbreak response, and the development of treatments designed to help the human microbiome fight infection. They note that this tool can be used across microbial kingdoms to uncover strain dynamics.

    “Traditionally, this has been very difficult for us to achieve, yet it is incredibly important to know, as people can carry several slightly different versions or strains of the same species at once, which makes it challenging to understand how microbes move between individuals,” notes Gerry Tonkin-Hill, PhD, group leader at the the Peter MacCallum Cancer Centre and the Peter Doherty Institute at the University of Melbourne, Australia. “Using this new technology, we can now overcome this challenge and gain a clearer picture of how microbes are shared between people. This will give us a better understanding of how microbes spread to help us prevent infection in vulnerable populations, like our cancer patients.”

    This work is published in Nature Microbiology in the paper, “Strain-level transmission inference across multi-kingdom metagenomic data using TRACS.”

    Being able to track the spread of pathogens using genomics has become a major tool in public health and can help inform new ways to prevent transmission. Additionally, it can help understand more about how lifestyle and environmental factors are involved in the transmission of these pathogens, and their role in the microbiome.

    Currently, genomic tools used to track multiple bacterial species do not have the speed and flexibility required for routine public health monitoring and can struggle to distinguish between samples transmitted recently and those transmitted years ago. Furthermore, it can be difficult to continuously add in new samples, making real-time surveillance difficult.

    The TRACS algorithm identifies and analyzes Single Nucleotide Polymorphisms (SNPs) to estimate how closely related the pathogens are, and if they are likely to have recently been transmitted. This approach allows for the continuous integration of new samples, making it an ideal tool for accurately identifying transmission networks and ruling out transmission events in ongoing public health applications.

    In this new study, the team used TRACS to map pathogen transmission networks across three different populations, all of which had different genomic data. They applied it to SARS-CoV-2 data from U.K. hospitals, deep population sequencing data of Streptococcus pneumoniae and single-cell genome sequencing data from malaria patients infected with Plasmodium falciparum. They found that the tool was able to identify different pathogens in one sample and infer where these were each transmitted.

    They also used TRACS to study how microbes are passed from mothers to infants and found that one beneficial bacterium, Bifidobacterium breve, persisted in infants longer than previously recognized, something that previous methods have missed.

    More superficially, the authors note that “applying TRACS to gut metagenomic samples from a mother–infant cohort revealed species-specific transmission rates and identified increased the persistence of Bifidobacterium breve in infants, a finding previously missed owing to the presence of multiple strains.”

    “This research could support the development of new treatments that use beneficial microbes to improve health,” notes Trevor Lawley, PhD, group leader at the Wellcome Sanger Institute. “By understanding exactly how microbes move between people and which of them are more likely to thrive in their microbiome, we could design better ways to increase helpful gut microbes and investigate whether there are ways to use these to help prevent infections, opening the door to safer healthcare environments and new microbiome-based therapies.”

    The post TRACS Enables Strain-Level Tracking of Microbial Transmission appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Protein Biomarkers in Practice: Strategies to Reduce Drug Development Risk https://edusehat.com/en/protein-biomarkers-in-practice-strategies-to-reduce-drug-development-risk https://edusehat.com/en/protein-biomarkers-in-practice-strategies-to-reduce-drug-development-risk Tue, 05 May 2026 04:50:09 +0700 Edusehat Protein, Biomarkers, Practice:, Strategies, Reduce, Drug, Development, Risk

    Drug development demands scientific rigor, sustained investment, and confident decision-making under uncertainty. As programs move from early discovery into clinical development, teams must balance biological complexity, timelines, and capital allocation— often without sufficient translational insight. Selecting the wrong target or patient population can result in costly delays and increased clinical risk.

    Protein biomarkers are becoming central to how pharmaceutical leaders reduce that risk and guide strategy. Unlike static genomic associations, proteins provide dynamic, functional insights into disease biology, reflecting pathway activity, target engagement, and treatment response in real-time. Advances in high-throughput proteomic technologies have transformed protein biomarkers from exploratory tools into strategic assets applied across the drug development lifecycle.

    When integrated early, biomarker-driven approaches can strengthen target validation, support proof-of-mechanism studies, enable more precise patient segmentation, and provide measurable indicators of efficacy and safety. The result is more informed decision-making, improved trial design, and greater confidence as programs advance.

    This eBook is designed to deliver both strategic insight and practical guidance. It opens with a White Paper informed by expert perspectives from senior translational leaders at leading pharmaceutical organizations. These experts explore how protein biomarkers mitigate risk across the drug development continuum, from early target validation to clinical trial design, by strengthening biological confidence and enhancing decision quality.

    Building on these strategic insights, the eBook presents seven real-world application examples that illustrate how these approaches are implemented in practice. Together, these perspectives provide readers with actionable frameworks and concrete use cases to help reduce uncertainty, optimize patient selection, improve trial efficiency, and make more confident, data-driven decisions earlier in development.

    The post Protein Biomarkers in Practice: Strategies to Reduce Drug Development Risk appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Tailoring AI solutions for health care needs https://edusehat.com/en/tailoring-ai-solutions-for-health-care-needs https://edusehat.com/en/tailoring-ai-solutions-for-health-care-needs Tue, 05 May 2026 01:15:03 +0700 Edusehat Tailoring, solutions, for, health, care, needs The AI market is full of big promises of grand transformation. Health care is a prime target for those promises, beset as it is by financial pressures, labor shortages, and the growing burden of caring for an aging population. AI developers are targeting functions that vary widely, from curing cancer and performing surgery to streamlining routine administrative tasks.

    The opportunity is genuine, but execution can be difficult. Numerous software vendors have tried to “fix” health care challenges but failed because they misunderstood the environment. “Health care is very complex,” says Steve Bethke, vice president of the solution developer market for Mayo Clinic Platform, which supports the buildout and deployment of digital solutions for health care companies through data-based insights and expert validation. “Solution developers must have a deep focus on clinical and technical capabilities, and then align their solutions to the relevant business impacts. If they miss any dimension, the solution will not be adopted or drive value.”

    DOWNLOAD THE REPORT

    AI applications for health care are proliferating rapidly. The U.S. Food and Drug Administration has approved more than 1,300 AI-enabled medical devices, mostly for interpreting diagnostic images. More than half of these were approved in the past three years, with the earliest dating as far back as 1995. Non-radiological applications carry out tasks as diverse as tracking sleep apnea, analyzing heart rhythms, and planning orthopedic surgeries.

    AI applications that do not count as medical devices— for example, those that handle scheduling and administrative tasks—are more difficult to track but are also rapidly increasing. AI can help coordinate complex tasks and workflows that are often conventionally managed by whiteboards and sticky notes. Such functions may well outstrip clinical uses in their impact on health systems. A recent survey of technology leaders found that 72% said their top priority for AI was reducing caregiver burden and improving caregiver satisfaction, while over half (53%) cited workflow efficiency and productivity.

    Any health care-related application can potentially impact patient care, whether directly or indirectly, and AI apps that are poorly designed or inadequately trained and validated can put patients at risk. Providers recognize that risk: In the same survey, 77% said immature AI tools are a significant barrier to adoption. Regulators and lawmakers are also keeping an eye on the risks as development and adoption burgeon, though the U.S. regulatory picture is still in flux, as a 2024 report to Congress on AI in health care observes.

    To tackle some of the technical challenges, many health care providers are partnering with application developers to build AI solutions. In a recent study, McKinsey found that 61% of health care organizations intend to pursue partnerships with third-party vendors to develop customized generative AI solutions as a primary strategy as opposed to building them in-house or buying off-the-shelf products.

    But health care-specific AI applications must also be tailored to the nuanced clinical needs of medical providers as well as the complex business and regulatory considerations of the wider sector. This is where developers can benefit from working with a partner with a deep understanding of the health care environment to tailor applications to what providers want and need most. Doing so helps to position AI products for maximum impact and value, avoiding the pitfalls unique to the health care environment.

    Download the report.

    This content was produced by Insights, the custom content arm of MIT Technology Review. It was not written by MIT Technology Review’s editorial staff. It was researched, designed, and written by human writers, editors, analysts, and illustrators. This includes the writing of surveys and collection of data for surveys. AI tools that may have been used were limited to secondary production processes that passed thorough human review.

    ]]>     Big Tech Targets Drug Discovery with Wave of Life Science Platforms https://edusehat.com/en/big-tech-targets-drug-discovery-with-wave-of-life-science-platforms https://edusehat.com/en/big-tech-targets-drug-discovery-with-wave-of-life-science-platforms Tue, 05 May 2026 01:10:09 +0700 Edusehat Big, Tech, Targets, Drug, Discovery, with, Wave, Life, Science, Platforms Nvidia CEO, Jensen Huang, asserts that accelerated computing has a missing word: application acceleration. The “vertically integrated” and “horizontally open” chip maker is set on building the infrastructure that delivers AI into real world use. 

    “Accelerated computing is not a chip problem,” said Huang when he took the stage for his annual NVIDIA GTC keynote in San Jose in March. “The only way for us to accelerate applications and bring tremendous speed up and cost reduction is through domain specific acceleration.”  

    That mission has hit drug discovery, where approval timelines exceed a decade and clinical trial failure rates approach 90%.  

    A new wave of platforms from Amazon Web Services (AWS), OpenAI, and Anthropic have customized general-purpose assistants into AI-powered workflows for science research. The trend points to the growing role of cloud infrastructure and agentic AI in unifying fragmented tools, streamlining data management, and making domain expertise more accessible. 

    Lab-in-the-loop 

    In April, AWS introduced Amazon Bio Discovery, a “lab-in-the-loop” workflow that combines access to more than 40 open-source and proprietary biological foundation models with AI agents that guide experimental design. The platform also integrates CRO partners, including Twist Bioscience, Ginkgo Bioworks, and A-Alpha Bio, for lab validation. The launch was announced at the AWS Life Sciences Symposium at the Javits Center in New York. 

    In collaboration with Memorial Sloan Kettering Cancer Center, Amazon Bio Discovery designed nanobodies with nanomolar affinities by generating nearly 300,000 candidates that were narrowed to the top 100,000 for wet lab testing in weeks, a noticeable reduction from the up to one year timeline typical of traditional methods. 

    Dan Sheeran, vice president and general manager, healthcare and life science at AWS, explains that while biological AI models have driven breakthroughs in areas, like protein design, their reliance on coding expertise and complex compute infrastructure remains a significant barrier to broader accessibility. 

    “Choosing the right model for a given task is itself a significant challenge. Computational biologists, the specialists who bridge AI and biology, are in short supply,” Sheeran told GEN Edge. “The result is a collaboration bottleneck, not because the science isn’t available, but because the tooling doesn’t support how these teams need to work together.” 

    David Younger, PhD, co-founder and CEO of A-Alpha Bio, adds that the partnership with AWS highlights a “fundamental gap” in AI-powered drug discovery, the lack of high-quality, experimental data at scale to evaluate protein design models. In silico candidates designed using Amazon Bio Discovery can be rapidly validated in the lab with A-Alpha’s AlphaSeq platform, which quantitatively measures protein-protein interactions by the hundreds to millions. 

    “The convergence of technology and life sciences isn’t just about faster compute or better algorithms,” Younger told GEN Edge. “It’s about connecting those advances to real-world, experimental observations.” 

    Amazon Bio Discovery is built on the same AWS infrastructure that is currently adopted by 19 of the top 20 global pharmaceutical companies. Each organization’s data is isolated within its application environment, and all proprietary data, models, and designs remain customer-owned. 

    Rosalind reasons 

    Two days after Amazon Bio Discovery’s launch, OpenAI announced GPT-Rosalind, a specialized reasoning model that supports evidence synthesis, hypothesis generation, and experimental planning for research across biology, drug discovery, and translational medicine. The platform includes a freely accessible life sciences research plugin for Codex that connects to over 50 public multiomics databases, literature repositories, and computational biology tools.  

    The model is available through a trusted-access program for qualified enterprise customers in the U.S. Amgen, Moderna, the Allen Institute, and Thermo Fisher Scientific are among GPT‑Rosalind’s customers. 

    “Research organizations are actively looking for systems that are built for scientific workflows, not adapted from general-purpose models, and life sciences remains one of the most important areas where better tools could meaningfully accelerate progress,” wrote OpenAI in an email to GEN Edge when describing the motivation for building GPT-Rosalind. 

    Named after Rosalind Franklin, PhD, whose work was critical in the discovery of the DNA double helix, the model scored 0.751 on BixBench, a benchmark that evaluates large language model (LLM) performance in bioinformatics and computational biology tasks. The score was a modest lead ahead of GPT-5.4, xAI’s Grok 4.2, and Google’s Gemini 3.1 Pro. 

    On LABBench2, a benchmark spanning literature retrieval, database access, sequence manipulation, and protocol design, GPT-Rosalind outperformed GPT-5.4 on six out of 11 tasks. The largest improvement was shown on CloningQA, which requires end-to-end design of DNA constructs and enzyme reagents for molecular cloning workflows. 

    GPT-Rosalind is one step in OpenAI’s growing momentum across pharma and healthcare. In recent weeks, the company introduced ChatGPT for Clinicians to support clinical workflows, such as documentation and medical research, alongside partnerships with Novo Nordisk to enhance workforce AI readiness and improve manufacturing and supply chain efficiency, and Massive Bio to expand access to clinical trials. 

    Inference inflection 

    Anthropic is forging its own path into life sciences, having recently drawn attention for acquiring Coefficient Bio, a roughly 10-person AI drug discovery start-up founded by former Genentech scientists, for $400 million.  

    The OpenAI competitor has also been building Claude for Life Sciences, the AI assistant specialized for researchers, clinical coordinators, and regulatory affairs managers, since last fall. 

    In an October blog post, Anthropic reported that the customized platform powered by Claude Sonnet 4.5 scored 0.83 in Protocol QA, a benchmark that tests the model’s understanding of laboratory protocols. The score outperformed the human baseline of 0.79 and Sonnet 4’s performance of 0.74. Claude for Life Sciences also incorporates several connectors to scientific platforms, including Benchling’s digital notebooks, PubMed literature, and 10x Genomics tools for single cell and spatial analysis.  

    “We want to give scientists the same experience as software engineers of having a brainstorming partner to work with and to delegate tasks,” said Eric Kauderer-Abrams, PhD, head of biology and life sciences at Anthropic, in a video accompanying the product launch. 

    In January, Anthropic expanded the platform to Claude for Healthcare, a complementary set of tools that allow healthcare providers, payers, and health tech companies and startups to use Claude for medical purposes through HIPAA-ready products.

    When reflecting on these life science releases, Enke Bashllari, PhD, founder and managing director at Arkitekt Ventures, says the three are “playing different games.” OpenAI is selling the “sharpest reasoning engine” with limited access, while AWS is building infrastructure and lab integration. Anthropic is betting on breadth of workflow and making acquisitions to close the specialization gap.  

    “For startups, the question isn’t which platform wins. It’s which layer you build on,” wrote Bashllari on LinkedIn. 

    Chris Leiter, founder and general partner at Atria Ventures, believes the shift to bioconsumerism will be the “most significant period of disruption for life sciences in the modern era.” 

    “Medicine is the use case that justifies the entire buildout,” wrote Leiter on LinkedIn. “The public skepticism starts to erode when the output is a drug that reaches a patient five years early, or a diagnostic that catches a cancer no doctor would have seen.” 

    As models increasingly move beyond isolated predictions into complex reasoning across biological systems, the question is no longer whether to adopt, but how quickly the industry can adapt to a new scientific discovery paradigm. 

    Huang says it best, “we are now in the beginning of a new platform shift. The inference inflection has arrived.” 

    The post Big Tech Targets Drug Discovery with Wave of Life Science Platforms appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     StockWatch: Patient Death, Rival’s Patent Challenge Sink Erasca Shares https://edusehat.com/en/stockwatch-patient-death-rivals-patent-challenge-sink-erasca-shares https://edusehat.com/en/stockwatch-patient-death-rivals-patent-challenge-sink-erasca-shares Mon, 04 May 2026 03:35:09 +0700 Edusehat StockWatch:, Patient, Death, Rival’s, Patent, Challenge, Sink, Erasca, Shares Sometimes a patient’s death is enough to send a stock tumbling. Other times, the fear of litigation that often arises whenever a new therapeutic approach emerges can send shares sinking. This past week, Erasca (NASDAQ: ERAS)—whose name is a portmanteau for “erase cancer”—ran into both, causing its share price to nosedive 53%.

    The precision oncology company, whose drugs and combination therapies focus on fighting cancer by shutting down the RAS/MAPK pathway, shared preliminary positive albeit early clinical dose escalation data for ERAS-0015, pooled from two Phase I trials. The oral pan-RAS molecular glue is being developed to treat solid tumors that include non-small cell lung cancer (NSCLC) and pancreatic cancer (formally pancreatic ductal adenocarcinoma or PDAC).

    ERAS-0015 achieved unconfirmed overall response rates (uORR) of 62% to 75% in KRAS G12X NSCLC patients dosed at 16–32 mg once daily, with the low percentage in second line or greater KRAS G12X NSCLC (37 patients), and the high percentage in post-ICI/platinum (second and third line) KRAS G12X NSCLC (37 patients). In pancreatic cancer, which has an overall five-year survival rate of just 13%, ERAS-0015 achieved unconfirmed overall response rates ranging from 40% to 50% in second-line positive KRAS G12X PDAC.

    However, tucked in footnotes on pages 25 and 43 of its investor presentation detailing the positive data was the disclosure that a 66-year-old male patient died after his grade 3 treatment-related adverse event (TRAE) of pneumonitis progressed to grade 5 after supportive care was withdrawn at the patient’s direction. The patient, who had “heavily pretreated” metastatic pancreatic cancer, received 24 mg of ERAS-0015, Erasca said.

    “The patient had pulmonary metastases, a history of right lung cryoablation, and no history of lung radiation. The patient presented to the ER approximately a month after starting ERAS-0015 with grade 3 pneumonitis that was treated aggressively with immediate discontinuation of ERAS-0015, high-dose steroids, and infliximab,” Erasca explained. “The patient requested withdrawal of supportive care and ultimately died of the event.”

    “Different outcome”

    Jonathan E. Lim, MD, Erasca’s chairman, CEO, and co-founder

    During an April 27 conference call, Jonathan E. Lim, MD, Erasca’s chairman, CEO, and co-founder, told analysts the death was “a very rare event” as pneumonitis is a rare drug-related toxicity seen in many oncology drugs.

    “The withdrawal of supportive care is really why this progressed from grade 3 to grade 5,” Lim said. “The investigator told us directly that he thought that if the patient had continued supportive care, then it might have been a different outcome. So yes, it’s very unfortunate for the patient, but that was the feedback.”

    Lim added that Erasca hasn’t seen any other grade 4 or grade 5 TRAEs. He told analysts that both FDA-approved KRAS G12C inhibitors have warnings and precautions for pneumonitis in their labels—Lumakras® (sotorasib), marketed by Amgen (NASDAQ: AMGN); and Krazati® (adagrasib), marketed by Bristol Myers Squibb (NYSE: BMY). Lim also cited Revolution Medicine (NASDAQ: RVMD)’s daraxonrasib (formerly RMC-6236), which Erasca is citing as a comparator to ERAS-0015, had also reported pneumonitis at a level of 1 out of 50 patients for monotherapy.

    The same day as the data release and patient death disclosure, Erasca also revealed in a regulatory filing that it had received a letter from legal counsel for Revolution, Erasca’s arch-rival developer of RAS inhibitors against cancer. Revolution told Erasca that ERAS-0015 was “substantially equivalent” to compositions claimed by Revolution in its U.S. Patent No. 12,409,225, titled “RAS Inhibitors”, and as a result, Erasca “infringes” the patent.

    “The disclosure features macrocyclic compounds, and pharmaceutical compositions and protein complexes thereof, capable of inhibiting Ras proteins, and their uses in the treatment of cancers,” according to the text of the patent, which lists Revolution as its assignee.

    Revolution also contended that Erasca was liable as licensee for ERAS-0015, and that Erasca “improperly” compared preclinical data of ERAS-0015 and daraxonrasib publicly.

    No wrongdoing, Erasca insists

    Erasca insists it did nothing wrong, stating in the filing that it “intends to contest the allegations vigorously.”

    Lim echoed the company’s response in the filing during an interview with GEN, where he stated that the company believes Revolution’s assertions to be without merit.

    “We have no reason to believe that ERAS 15 infringes any patent—including RevMed’s accusation, which is based on the doctrine of equivalence, rather than an accusation of direct infringement,” Lim said. “RevMed did not provide details to support its claim that others engaged in trade secret misappropriation, and we have no reason to believe any such thing occurred.”

    “In addition, we believe that all of our preclinical data comparisons have been appropriate. And all of our data presented on the R&D Day were not based on head-to-head studies but on cross-trial comparisons. We made that very clear during the presentation,” Lim added.

    Through a spokesperson, Revolution told GEN the company was acting to defend its inventions and the IP behind them.

    “We are committed to protecting the strong foundation of innovation we have built over more than a decade through transformative science and significant investment,” Revolution stated. “While we do not comment on the specifics of ongoing legal matters, we remain confident in the strength of our intellectual property. Our focus remains on advancing our science to deliver innovative medicines that make a meaningful impact for patients.

    Slumping stock

    Erasca investors reacted coolly to the patient death and prospect of a legal wrangle with Revolution. Erasca shares slumped 11% from $21.49 to $19.15 the day of the data announcement and regulatory filing. The share price plunged 48% to $9.90 Tuesday and fell another 8% Wednesday to $9.11 before bouncing back 17% to $10.65 Thursday as investors bought the dip, then finished Friday sliding 6%, closing at $10.03.

    Despite the selloff, Erasca shares have more than quadrupled over the past six months, rocketing 314% from $2.42 on October 31 and catapulting 573% over the past year (from $1.49 on May 1, 2025).

    Revolution shares rose 3% this week. After sliding nearly 3% from $135.30 to $131.67 on April 27, the stock benefited from Erasca’s slump by jumping 10% to $144.83 before fluctuating, closing Friday at $139.48.

    “We view the (-)ve [negative] post‑data reaction as overdone, as ERAS‑0015 looks like a formidable competitor in the pan‑RAS landscape,” Maury Raycroft, PhD, equity analyst with Jefferies, wrote in a research note.

    Raycroft cited Erasca’s comparing its 62%/75% uORR in NSCLC to the confirmed 38% ORR cited for daraxonrasib in a 2025 study by researchers from Revolution and several clinical partners. Among second and third-line treatment patients post‑ICI/platinum, efficacy appeared broadly consistent across geographies, Raycroft noted, w/ uORR of 71% in the United States (12 patients), where ERAS-0015 was studied in the Phase I AURORAS-1 trial (NCT06983743), and 73% in China (11 patients), where partner Joyo Pharmatech sponsored a trial known as STAR or JYP0015M101 (NCT06895031).

    “Early durability is also encouraging, w/ progression observed in 1/27 pts in the China study and 2/12 in the U.S. study; though, follow‑up remains limited (median likely ~3–4 mos), so durability conclusions are premature,” Raycroft cautioned.

    In pancreatic cancer, pooled U.S. and Chinese patient data ranged from 40% uORR at 16–32 mg (20 patients), to 42% uORR at 24–32 mg (12 patients), and 50% uORR at 32 mg once daily (two patients).

    Expansion doses

    “Right now, we think the lower doses at 8 and 16 milligrams, for instance, seem to be relatively underpowered against pancreatic cancer compared to 24 to 32 milligrams. So, based on the totality of efficacy, safety, tolerability, and PK data, we have determined 24 and 32 milligrams to be the recommended doses for expansion,” Lim told GEN. “So we have already begun to expand out those doses to treat patients at both 24 and 32 milligrams.”

    All but one of 24 responding NSCLC patients, and 20 out of 23 responding PDAC patients, remain on treatment, including all responders treated at the 24 and 32 mg once daily recommended doses for expansion (RDEs).

    Erasca said ERAS-0015 showed clinical potential for combinability with standard-of-care doses of the anti-EGFR monoclonal antibody panitumumab in fighting colorectal cancer (CRC), where EGFR is a key mechanism for acquired resistance. Through the data cutoff date of March 31, Erasca showed no dose-limiting toxicities among three patients, of which one showed an unconfirmed partial response in an efficacy-evaluable patient with metastatic CRC: “That’s really exciting to have,” Lim said.

    Compelling opportunities

    “For monotherapy, we think lung and pancreatic are compelling opportunities, and then for colorectal cancer, combination therapy will likely be required, so we’re focused on the combination with EGFR antibody for colorectal cancer,” Lim said.

    “The fact that we have been able to successfully combine ERAS15 with panitumumab in three patients, with two of them passing the DLT window, is exciting. And we did that with a 16 milligram dose of ERAS-15, which is within the pharmacologically active dose range of 16 to 32 milligrams. It’s really exciting to have safety and tolerability that is promising with that combination and activity within the PAD.”

    The Erasca-Revolution dispute appears to explain a decline early this past week in the American depositary shares of another cancer drug developer whose pipeline includes RAS-targeting therapies: Adlai Nortye (NASDAQ: ANL) shares skidded 10.5% from $14.80 to $13.25 the day of Erasca’s announcement and filing, then dropped another 8% to $12.17 Tuesday before rebounding 27% the rest of the week, closing Friday at $15.50.

    Adlai Nortye is developing AN9025, an oral, small-molecule pan-RAS(ON) inhibitor designed to treat a variety of advanced solid tumors with RAS mutations. AN9025 is under study in a Phase I trial (NCT07252479) whose first patient was dosed in the United States in February. The trial is being conducted with Jiangsu Aosaikang Pharmaceutical, which holds rights to AN9025 in China, Hong Kong, and Macao.

    Andrew Berens, MD, senior managing director, targeted oncology, and a senior research analyst with Leerink Partners, wrote in a research note that a conversation with Adlai Nortye management offered reasons for confidence that the company will avoid legal trouble in connection with AN9025.

    “ANL’s discovery of AN9025 was conducted in-house and independently, suggesting that trade secret misappropriations are unlikely,” Berens reported. “Further, while both drugs feature a macrocyclic scaffold, according to ANL, ERAS-0015 has significantly less branch change modifications and features a bridge, while 9025 underwent numerous branch modifications and the addition of a ring to the scaffold.”

    Leaders and laggards

    • Esperion Therapeutics (NASDAQ: ESPR) shares leaped 55.5% from $2 to $3.11 Friday after the developer of cardiometabolic and rare/orphan disease therapies said it agreed to be acquired by funds managed by the healthcare-focused investment firm Archimed in a deal valued at up to approximately $1.1 billion if Esperion achieves specified commercial-based milestones. Esperion shareholders will receive $3.16 per share in cash at closing, plus the right to participate in contingent milestone payments of up to $100 million tied to future U.S. net sales for products containing bempedoic acid and products containing bumetanide. The upfront cash consideration represents a premium of 58% to Esperion’s closing share price on Thursday. Founded in 2008, Esperion specializes in developing drugs designed to fight the risk of cardiovascular disease. Esperion’s board has unanimously approved the acquisition deal, which is expected to close in the third quarter of 2026, subject to customary closing conditions that include approval by Esperion’s shareholders and required regulatory approvals.
    • Novocure (NASDAQ: NVCR) shares jumped 27% from $11.93 to $15.21 Thursday following several positive announcements by the oncology drug/device developer. Novocure raised its 2026 guidance to investors, increasing its net revenue forecast range to $690 million–$710 million (from $675 million–$705 million), and adjusted earnings before interest, taxes, depreciation, and amortization (EBITDA) from a $15 million operating loss to zero (from a $20 million operating loss to zero). The company said its launch of Optune Pax®, a wearable device designed to deliver its Tumor Treating Fields (TTFields) therapy for adults with locally advanced pancreatic cancer concomitant with gemcitabine and nab-paclitaxel, was successful, with 800+ prescribers certified and 160+ prescriptions received through March 31. TTFields are alternating electric fields designed to cause cell death by disrupting cancer cell replication. Novocure finished Q1 with net revenue of $174.055 million, up 12% year-over-year from $154.994 million.
    • Sangamo Therapeutics (NASDAQ: SGMO) shares plummeted 35% from 20 cents to 13 cents on Wednesday after the genomic medicine developer said it will begin trading its shares on the OTCQB Venture Market on May 5, under its existing ticker symbol SGMO. The switch comes after Nasdaq notified Sangamo that its common stock will be delisted from the Nasdaq Capital Market due to non-compliance with Nasdaq’s minimum $1 per share bid price requirement. While Sangamo said it intends to request a hearing from Nasdaq to appeal the delisting determination, that hearing will not stay the suspension of trading of Sangamo’s common stock. Sangamo said the shift is not expected to result in material impacts to its business or operations: “Sangamo remains focused on pursuing opportunities to raise additional capital, including an assessment of all strategic options to maximize the value of its assets.” To that end, the company said, it is negotiating “multiple potential business development transactions.”
    • uniQure (NASDAQ: QURE) shares climbed 19% from $16.73 to $19.95 Thursday after the gene therapy developer offered regulatory updates that included having been granted a granted a Type B meeting with the FDA to occur in the second quarter: “The company expects to discuss key elements of a potential Phase III trial design and to receive feedback on the proposed statistical analysis plan for the four-year analysis expected in the third quarter.” uniQure added that it held a pre-submission meeting with the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) and plans to submit a Marketing Authorization Application (MAA) for AMT-130 for the treatment of Huntington’s disease in the third quarter. The company said it expects to submit an MAA based on a three-year analysis from its ongoing U.S. and European Phase I/II clinical trials.

    The post StockWatch: Patient Death, Rival’s Patent Challenge Sink Erasca Shares appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Figurate SCADA System Launched to Overcome Digital Bottlenecks During Biopharma Manufacturing https://edusehat.com/en/figurate-scada-system-launched-to-overcome-digital-bottlenecks-during-biopharma-manufacturing https://edusehat.com/en/figurate-scada-system-launched-to-overcome-digital-bottlenecks-during-biopharma-manufacturing Sat, 02 May 2026 04:40:11 +0700 Edusehat Figurate, SCADA, System, Launched, Overcome, Digital, Bottlenecks, During, Biopharma, Manufacturing Cytiva and Rockwell Automation launched the figurate supervisory control and data acquisition (SCADA) system designed to remove digital bottlenecks during biopharmaceutical manufacturing. Working across multiple instrument vendors and modalities, Figurate SCADA provides the connectivity needed to enable digital integration to advance modern bioprocessing, according to Matt Weaver, vice president of global industry life sciences at Rockwell.

    “Biopharma teams are under pressure to move more quickly, but their systems are often not built to keep up,” says Weaver. “This collaboration with Cytiva marks a pivotal step in our mission to democratize digital manufacturing, enabling biopharma innovators to deploy SCADA faster, smarter and more affordably.”

    Many biopharma teams have long juggled proprietary systems that cannot communicate with one another, creating operational silos, manual workarounds, and data integrity risks. The new system directly addresses this roadblock by having an open architecture, allowing for third-party instrument integration, and real-time oversight of integration capable unit operations from a single interface, notes a Cytiva spokesperson, who explains that the platform features include:

    • Native interoperability: The platform is natively integrated with Cytiva bioprocessing equipment and Rockwell Automation’s FactoryTalk software suite, enabling seamless interoperability across systems.
    • Scalable growth: A single platform expands from process development to commercial manufacturing without system redesign.
    • Cost-effective compliance: A streamlined digital manufacturing system reduces capital and operational costs and enables cGMP compliance.
    • Rapid implementation: Pre-engineered templates and modular design shorten deployment and validation timelines.
    • Enhanced operational insight: Centralized alarms, real-time monitoring, process intensification and batch reporting tailored to bioprocess workflows.

    “This collaboration is designed to empower the next generation of biomanufacturers,” says Nicolas Pivet, manufacturing and digital solutions at Cytiva.

    Industry data shows increasing demand for next generation process control systems as organizations transition toward data driven process intensification and continuous manufacturing. Equipment fragmentation remains one of the top pain points cited by biomanufacturers, particularly those advancing programs from R&D to clinical scale. By giving teams a unified digital control layer, the Figurate SCADA reduces the risk of human error, accelerates tech transfer, and supports reliable scaleup as workloads grow in complexity, points out the Cytiva spokesperson.

     

    The post Figurate SCADA System Launched to Overcome Digital Bottlenecks During Biopharma Manufacturing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Restoring Protein Recycling Reverses T-Cell Exhaustion in Mice https://edusehat.com/en/restoring-protein-recycling-reverses-t-cell-exhaustion-in-mice https://edusehat.com/en/restoring-protein-recycling-reverses-t-cell-exhaustion-in-mice Sat, 02 May 2026 04:40:10 +0700 Edusehat Restoring, Protein, Recycling, Reverses, T-Cell, Exhaustion, Mice New research published by scientists at the University of California, San Diego (UCSD), describes an unexpected factor underlying T-cell exhaustion. The details of their work in mice are published in a new Cell paper titled “Proteostasis sustains T-cell differentiation potential and tumor-infiltrating lymphocyte function.”

    T cells are critical members of the immune system but there are limits to their defensive capabilities. When fighting cancer cells, T cells often burn out and become dysfunctional. A major focus of current cancer immunotherapy efforts is rescuing T cells from this state and getting them back into cancer-fighting shape. The new Cell study led by scientists in the lab of Ananda Goldrath, PhD, a professor of molecular biology at UCSD, and their collaborators elsewhere, suggests that a potential solution to T-cell exhaustion might have to do with protein recycling.

    Specifically, their finding has to do with proteostasis, the network of cellular processes that orchestrates the proper construction, movement, and destruction of proteins in cells. A component of this network features a type of recycling function where healthy cells continuously dismantle old and damaged proteins to preserve energy and reuse building blocks to make new proteins. According to the paper, the scientists uncovered an impaired protein recycling function as the surprise culprit in T-cell exhaustion. 

    “We found that exhausted T cells’ recycling programs are falling apart, leading to damaged and misfolded proteins that pile up with nowhere to go,” said Nicole Scharping, PhD, a post-doctoral fellow in the Goldrath lab and lead author on the paper. Additionally, the scientists also uncovered a way to reverse the accumulation of misfolded proteins by fixing the broken recycling function and restoring normal proteostasis. As Scharping explained, the issue can be resolved with a “tag and sort” fix. This is accomplished using E3 ligase enzymes which act as labelers at a recycling facility, tagging worn-out proteins so the cell knows to break them down.

    “In exhausted T cells, many of these enzymes get switched off, and recycling grinds to a halt,” said Scharping. After examining thousands of proteins, the scientists honed in on NEURL3, RNF149 and WSB1 as the E3 ligases responsible for rescuing T cell recycling functions. “When we restored specific E3 ligases, the buildup cleared, and the T cells regained their function and worked better at clearing tumors.” While the new study was conducted in mice, the researchers indicate that similar strategies could be employed for immunotherapy treatments in human cancer.

    Importantly, the findings may have implications in other diseases as impaired protein processing is not unique to exhausted T cells. “We think this loss of proteostasis resembles what occurs in neurons in other protein aggregate diseases such as Parkinson’s and Alzheimer’s,” said Goldrath. “Rescuing these cells from exhaustion could improve the ability of T cells to respond to both chronic infection as well as tumors.”

    The post Restoring Protein Recycling Reverses T-Cell Exhaustion in Mice appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     DNA-Containing Extracellular Vesicles Boost Antitumor Responses in Mice https://edusehat.com/en/dna-containing-extracellular-vesicles-boost-antitumor-responses-in-mice https://edusehat.com/en/dna-containing-extracellular-vesicles-boost-antitumor-responses-in-mice Sat, 02 May 2026 04:40:09 +0700 Edusehat DNA-Containing, Extracellular, Vesicles, Boost, Antitumor, Responses, Mice A study led by investigators at Weill Cornell Medicine has found that activated T cells secrete extracellular vesicles (EVs) containing DNA, which can enter other immune and tumor cells to stimulate the body’s defense systems. Preclinical experiments showed that this vesicle-associated DNA could be useful therapeutically, boosting T cell attacks against tumors that otherwise evoke little or no immune response.

    Studies in live mice showed that these activated T cell-derived-EVs (AT-EVs) enhanced antigen processing and presentation (APP) in tumor cells and dendritic cells (DCs) across different immunologically cold tumors. The ATEVs also synergized with immune checkpoint inhibitors (ICIs) to trigger antitumor immunity and hold back tumor growth.

    The discovery extends the scientific understanding of the immune system, identifies a new strategy for boosting immunity against cancers, and potentially offers a new tool for delivering genetic payloads to other cells. “These findings reveal a natural mechanism for treating immunologically silent tumors and other diseases that stem from insufficient immune surveillance,” said David Lyden, MD, PhD, the Stavros S. Niarchos professor in pediatric cardiology and a member of the Gale and Ira Drukier Institute for Children’s Health and the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine.

    Lyden is co-senior author of the researchers’ published paper in Cancer Cell, titled “Activated T cell extracellular vesicle DNA transfer enhances antigen presentation and anti-tumor immunity,” in which they stated, “We uncover a mechanism whereby activated T cell-derived extracellular vesicles (ATEVs) drive a positive feedback loop that enhances antigen presentation and immune responses in normal physiology and cancer … Notably, ATEVs hold promise as an acellular immunotherapy, restoring APP and synergizing with checkpoint blockade in immunotherapy-refractory tumors.”

    Most animal cells secrete extracellular vesicles which can contain cargo including proteins, snippets of DNA, and other molecules. “Extracellular vesicles (EVs) are nanoparticles naturally released by all living cells, containing proteins, lipids, and genetic material, that facilitate intercellular communication,” the investigators wrote.

    The Lyden lab in recent years has made seminal discoveries about extracellular vesicles and their functions, finding for example that vesicles secreted by tumor cells can influence the immune system’s anti-tumor response. Their findings, they noted, “… raised the possibility that EVDNA from immune cells, such as T cells, may also have immune-related functions.” For their new study the team examined the roles of vesicles secreted by immune cells, and specifically T cells, which are the immune system’s principal tumor-fighters.

    In their initial experiments, the scientists found that under physiological conditions, T cell-secreted vesicles tend to home to lymph nodes, spleen and other centers of immune activity. There the vesicles are preferentially taken up by antigen-presenting immune cells, including dendritic cells, which assist in T cell activation, a critical process in the immune response. The researchers found that the overall effect of these vesicles released by activated T cells is to boost the antigen-presenting process, thus promoting T cell priming and broader immune activation. The key payloads in these immune-boosting vesicles turned out to be snippets of T cell DNA.

    “These surprisingly abundant DNA fragments are mostly on the surfaces of the vesicles, and are not just random—they are enriched for immune-related genes, including genes that help cells display antigens to the immune system,” said co-senior author Haiying Zhang, PhD, an assistant professor of cell and developmental biology in pediatrics and member of the Lyden lab. “We also found that these vesicles have, attached to their surfaces, a special enzyme that acts as a molecular drill, enabling the transfer of vesicle-carried DNA into the nucleus of the recipient cell where they can be expressed transiently,” added study co-first author Diao Liu, PhD, a postdoctoral research associate in the Lyden Lab.

    Infusing DNA-carrying vesicles from activated T cells into mice with tumors, the researchers found that the vesicles were taken up not only by antigen-presenting cells but also by tumor cells themselves. The treated tumors grew more slowly and were better infiltrated by T cells and other immune cells, indicating that the vesicles induced a stronger anti-tumor response. “Our work reveals an EV-mediated mechanism through which activated T cells enhance APP across diverse recipient cells—from DCs in physiological conditions to cancer cells across tumor types,” the authors noted. Although cancers—and viruses—frequently suppress the antigen-presenting process to make malignant or infected cells “invisible” to the immune system, the main effect of the extracellular vesicular DNA was to reverse this process, restoring tumor cells’ visibility.

    The team demonstrated the effectiveness of this approach, alone and in combination with existing immunotherapy, in preclinical models of three different immunologically silent cancers: glioblastoma, pancreatic and triple-negative breast cancer. “By boosting APP machinery, ATEVs enhance tumor immunogenicity and elicit robust anti-tumor responses, particularly when combined with ICIs in otherwise resistant tumors, including pancreatic, breast, and brain cancers,” they stated. “These findings reveal the translational potential of activated T cell-derived extracellular vesicles (ATEVs) by exploiting a naturally occurring immune-boosting process to overcome immune evasion, particularly in immunologically silent cancers.”

    Co-senior author Irina Matei, PhD, an assistant professor of immunology research in pediatrics and member of the Lyden lab, stated, “There seems to be a positive-feedback loop, in which the DNA-carrying vesicles from activated T cells amplify the immune response by acting on both antigen-presenting cells, which increase expression of the machinery processing tumor antigens, and tumor cells, promoting their recognition by the immune system as well as their own production of DNA-laden vesicles.”

    The researchers are now working to translate their findings into a new, vesicle-based cancer treatment, which could be used on its own or in conjunction with standard immunotherapies or other cancer treatments. “The surprising ability of these vesicles to transfer DNA from donor T cells into the nuclei of recipient cells suggests their potential as a natural, non-viral platform for transient gene delivery,” said co-first author Mengying Hu, PhD, a postdoctoral research associate in the Lyden Lab who led the research and is now an assistant professor of pharmaceutical sciences at the Ohio State University. “The results point to a broadly applicable gene-transfer strategy that may offer improved safety and efficiency compared with current gene therapy approaches.”

    In their paper the authors concluded, “Overall, ATEVs emerge as an acellular immunotherapy and delivery modality that can prime antitumor immunity, synergize with existing therapies, and serve as a vaccine adjuvant,” they concluded. “Our findings provide a foundation for the therapeutic application of ATEVs through a deeper understanding of the biological role of AT-EVDNA.”

    The post DNA-Containing Extracellular Vesicles Boost Antitumor Responses in Mice appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO on the American Road tours gene therapy hub in Ohio https://edusehat.com/en/bio-on-the-american-road-tours-gene-therapy-hub-in-ohio https://edusehat.com/en/bio-on-the-american-road-tours-gene-therapy-hub-in-ohio Fri, 01 May 2026 21:25:03 +0700 Edusehat BIO, the, American, Road, tours, gene, therapy, hub, Ohio Ohio’s diverse biotech sector provides the state with employment for 65,000 and provides the world with medical breakthroughs.

    Of the first eight gene therapies approved by the Food and Drug Administration, two—for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy Type 1 (SMA1)—were developed by researchers at Nationwide Children’s Hospital in Columbus, a city that’s driving gene editing research and production.

    A group from the Biotechnology Innovation Organization (BIO) learned more about that work during a BIO on the American Road tour, led by BIO President & CEO John F. Crowley on April 28.

    “Every state wants to be a leader in biotech. It takes people, capital, great universities, facilities—and a vision,” Crowley said, during the tour. In roundtables and presentations at various locations, BIO and local leaders in the sector discussed strategies for building on Ohio’s advantages and increasing the state’s leadership.

    BIO’s visit to Columbus focused on the capital city’s biotech ecosystem, where collaboration between major institutions has created a nationally recognized hub for gene therapy.

    Nationwide Children’s influence

    BIO Ohio
    Visiting Nationwide Children’s Hospital.

    “We talk about the so-called ‘Columbus way’—this deep sense of partnership between the public sector, the private sector, and the academic community,” said Dennis Durbin, President of the Abigail Wexner Research Institute at Nationwide Children’s Hospital—the first stop on BIO’s tour. “Columbus has become a leading destination for manufacturing of gene therapies, because of our early success at Nationwide Children’s.”

    He emphasized that, beyond hosting research, Nationwide Children’s technology transfer work ensured the resulting innovations became commercial products.

    “That process by which we go from laboratory discovery to a globalized product is something that we developed for our gene therapy program and are now replicating beyond the gene therapy space,” Durbin said.

    Speeding clinical trials

    The journey from lab to product includes clinical trials, an area where lawmakers and biotech companies urge improvement. As a BIO Board Member testified in Congress, China is taking American leadership in clinical trials, because bottlenecks delay trials in the U.S.

    “Nationwide Children’s prides itself on expediting first-in-human clinical trials,” Durbin said. “We have developed a series of resources to facilitate the translation from an early-stage laboratory discovery to a clinical trial.”

    As a research hospital, Nationwide Children’s attracts scientists interested in accessing facilities for care and clinical trials, and patients interested in accessing leading-edge therapies, he said.

    Ohio State University (OSU), who also hosted BIO’s team, is doing its own work to expedite clinical trials. OSU is involved in organizing a Big Ten Neurosurgery Consortium BTNC, which would see a network of universities collaborate to speed clinical trial starts by centralizing the work of Institutional Review Boards (IRBs) and contracting. OSU hosted a conference convening BTNC stakeholders in January.

    OSU’s Gene Therapy Institute

    BIO Ohio
    At OSU, the BIO team participates in a roundtable discussion with more than 30 biotech leaders committed to advancing Ohio’s life sciences, biomanufacturing, and innovation economy.

    OSU’s groundbreaking work in neurosurgery also involves gene therapy. A researcher at OSU’s Wexner Medical Center developed a gene therapy that is delivered by surgery to treat a rare genetic condition known as AADC deficiency. Researchers say the treatment has potential to treat additional conditions, such as Alzheimer’s and Parkinson’s. Other OSU research in this area includes a center for non viral-gene delivery.

    During the visit to OSU’s Gene Therapy Institute, Office of Research, Wexner Medical Center and James Comprehensive Cancer Center, BIO engaged in a round table discussion with members of the biotech ecosystem.

    “We were pleased to welcome John Crowley and the Biotechnology Innovation Organization (BIO) team to Ohio State,” said John M. Horack, Vice President for Research at OSU. “His visit underscored the importance of collaboration between academia and industry and reinforced our shared commitment to advancing biomedical research that translates discovery into real‑world impact.”

    Sarepta expands its footprint

    Crowley participated in a fireside chat with Louise Rodino-Klapac, President, R&D and Technical Operations at Sarepta Therapeutics, during a visit to Sarepta’s Genetic Therapies Center of Excellence (GTCOE).

    “The Ohio biotech ecosystem is what I dreamt of when I came here 26 years ago. What Sarepta has built, what Nationwide and Ohio State have built—it’s a great community, and there’s no limit to what we can do in Ohio,” Rodino-Klapac said during the panel.

    Sarepta’s gene therapy work in Columbus led the Massachusetts-based firm to invest in the 140,000-square-foot GTCOE, which opened in 2021 and houses both gene therapy research and manufacturing.

    “Our growing presence in Ohio will help us strengthen our close working relationships with long-standing local partners such as Nationwide Children’s Hospital, while we work with the greatest urgency to advance our pipeline, further the science of genetic medicine, and create an environment where future generations of scientific talent will thrive,” Rodino-Klapac said during the launch of the GTCOE .

    The full value chain

    “Ohio is unique in that we truly represent the entire life science value chain right here within our borders,” said Eddie Pauline, President and CEO of Ohio Life Sciences (OLS), a BIO affiliate with a mission to grow Ohio’s life sciences market and ensure patients can access the latest innovations. “If you’re coming in as an investor or entrepreneur, you know Ohio’s opening up a lot of different entry points to the life science ecosystem.”

    Pauline noted Ohio’s long-time leadership in life sciences distribution, with headquarters of DHL Life Sciences and Cardinal Health, and McKesson’s Jeffersonville, OH distribution center. Manufacturing includes Amgen’s New Albany plant, where a $900 million expansion is planned.

    Institutions like Cleveland Clinic, Case Western Reserve University, and Cincinnati Children’s Hospital drive research specialties in their city’s ecosystems, Pauline said. Cleveland, Cincinnati, and Columbus are the “innovation districts” that make up the Ohio Discovery Corridor, an initiative to connect innovative life sciences firms with the state’s innovation infrastructure and talent.

    Attracting talent is key to growing Ohio’s biotech, according to Pauline. The public-private initiative Jobs Ohio has invested $300 million in the life sciences innovation districts. Meanwhile, OLS is looking to develop wet lab space and funding to support biotech, he said.

    “We want to make sure that the great IP that is being developed in our institutions stays in Ohio,” Pauline explained. “So as something spins out, they can find the right people, capital and wet lab space to grow.”

    At top is a photo taken at Sarepta Therapeutics, where Louise Rodino-Klapac, President, R&D and Technical Operations at Sarepta, hosted a fireside chat with BIO President & CEO John F. Crowley.

    The post BIO on the American Road tours gene therapy hub in Ohio appeared first on Bio.News.

    ]]>     Collaboration vs. Coordination https://edusehat.com/en/collaboration-vs-coordination https://edusehat.com/en/collaboration-vs-coordination Fri, 01 May 2026 21:20:21 +0700 Edusehat Collaboration, vs., Coordination
    Edmund Champness
    Edmund Champness
    CSO, Company Director
    Optibrium

    In early-phase drug discovery, we do a good job of tracking the obvious productivity metrics, like compound attrition rates, hit-to-lead timelines, and computational resource spend. Other costs are harder to capture on dashboards, but contribute to overall efficiency just the same.

    There’s time lost waiting for every email response, compounded when teams are split across multiple sites or time zones. There is effort spent by team members extracting and preparing the data they need for their specific role. There is work required to consolidate the ever-evolving picture of compounds and their data into a single, coherent story, often just in time for a project meeting. Institutional knowledge walks out the door when a colleague moves on, leaving the next person to reconstruct not just what was tried, but why. And often, the lessons learned on one project never make it to the next, because there’s no obvious place to capture or share them.

    Rae Lawrence
    Rae Lawrence, PhD
    Product Manager
    Optibrium

    Some of these issues may be symptoms of poor coordination. Others we perhaps consider as the cost of running a complex business. But should we simply accept this? It’s tempting to point the finger at team members; if only they talked more, shared more, or documented better. However, the real focus should be on the infrastructure and how to get it working for you, rather than against you.

    The illusion of collaboration

    R&D efficiency and productivity are among the biggest challenges in drug discovery. The industry has responded in part by increasing its collaboration efforts through specialist partnerships, open innovation networks, and an ever-growing suite of digital tools. Yet pharmaceutical R&D costs continue to rise (up to £3.7 billion in 2023), while the number of new drugs approved annually remains relatively stagnant1. This tells us that current models aren’t yet delivering the results we need.

    There are several collaboration tools that we use regularly. Platforms like Slack, Jira, and shared documents have proven genuinely useful, changing how teams communicate and manage shared projects. But the frictions of duplicated efforts, version confusion, and lost knowledge persist. That’s because they’ve been designed for generic use cases and not drug discovery.

    Optibrium Collaboration image
    Effective coordination in computational drug discovery requires a shared data environment that gives each team member the freedom to explore and analyze in their own way. [Optibrium]
    Computational drug design generates a lot of data in distinct and complex formats, including molecular structures, 3D conformations, assay results, and multidimensional scoring profiles. The tools built for documents and spreadsheets aren’t designed to handle this. The result is fragmented workflows and lost insights; not because people won’t collaborate, but because the infrastructure can’t coordinate.

    What coordination actually requires

    What does genuine coordination in drug discovery look like in practice? It comes down to three things:

    1. Individual freedom within a shared reality

    Having a shared reality is the obvious part. In drug discovery, that means that everyone on the team needs access to the same underlying compounds and data. However, individual freedom is less obvious. In a team, every person doesn’t need to interact with the data in the same way. For example, while the project lead may want to explore the complete project data set while investigating new opportunities, individual members of their team may be focused on understanding and expanding different chemical series.

    Most collaborative platforms assume that a shared view equals a shared understanding, but in drug discovery, forcing everyone into a shared view of the data creates bottlenecks and leads to users with different goals tripping over each other. What’s needed is the freedom for exploration that allows individuals to view and analyze data in their own way, then share findings when they’re ready, without broadcasting every intermediate step to the whole team.

    2. A single source of truth with real-time synchronization

    A shared reality is only valuable if it stays that way over time. Drug discovery is a dynamic process; new compounds are designed, experimental assays and predictive models yield new data, and priorities shift as teams learn. A snapshot shared in last week’s meeting is already history a week later.

    What’s needed is an environment where new compounds, data, and analyses are shared automatically across the team, without manual uploads or chasing for updates. By minimizing the gap between data generation and visibility, we can start to work in the quick, iterative feedback cycles that drive efficient discovery.

    This also eliminates a scenario you might find familiar: two chemists advocating for different compound series because they’re working from different versions of the same data. That’s not a scientific debate; that’s an infrastructure failure.

    3. Persistent knowledge capture

    While real-time synchronization keeps us aligned on where we are with a project, it’s also important to understand how we got there. That means not just capturing the data, but also the rationale behind the decisions. Why was this series deprioritized? What concerned us about this scaffold? What information was missing when we made that call?

    Without answers to these questions, new team members start from scratch, and experienced ones repeat avoidable mistakes. Too often, this reasoning lives in emails, slide decks, or even just in people’s memories, and it vanishes when they move on. Instead, we need an automatic, comprehensive record of both the “what” and the “why,” so that teams get the full value of their accumulated knowledge.

    What makes a tool fit for purpose

    Beyond these three principles, there are other practical considerations that determine how well a platform can meet the demands of computational drug discovery.

    • Native handling of chemical data: Structures need to be searchable objects, not static images. A platform needs to work fluently with SMILES strings, descriptors, and 3D conformations.
    • Performance at scale: We have access to larger compound libraries than ever before, and any software needs to be able to keep up in real-time.
    • Vendor-neutral integration: Discovery teams rely on a suite of in-house and third-party systems, including databases, modeling tools, and compound registration platforms. Any system that promises coordination should be able to connect to these equally, and not just those from a single provider.
    • Ease of use: Any approach that requires specialist training to operate just introduces friction from the start. It needs to be intuitive enough that it stays out of the way of the science.
    • Granular access control: Discovery often involves working with CROs, academic partners, or other external collaborators. You need to be able to provide sufficient access to relevant data without exposing proprietary information.

    Is effective collaboration valuable

    In the last year, how much time did your team lose to the friction of getting information to the right people, whether that was waiting on responses, preparing data for meetings, or consolidating multiple versions of the same data set? And how much of what your team learned last year will be easily accessible to the team working on a related project two years from now?

    We’re collaborating more than ever, but productivity isn’t keeping pace. The missing piece is a coordination layer built for the realities of computational drug discovery; one that gives individuals the freedom to explore, keeps everyone working from the same current data, and preserves the knowledge that makes each design cycle smarter than the last.

    These are the costs that don’t show up on dashboards, but they’re precisely where the opportunity lies.

     

    Edmund Champness is the chief scientific officer and company director at Optibrium and Rae Lawrence, PhD, is product manager at Optibrium.

     

    References

    1. Fernald KDS, Förster PC, Claassen E, Linda. The pharmaceutical productivity gap – incremental decline in R&D efficiency despite transient improvements. Drug Discovery Today. 2024;29(11):104160-104160. doi.org/10.1016/j.drudis.2024.104160

    The post Collaboration vs. Coordination appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     What’s Next in the Evolution of Standards for Biologics Development https://edusehat.com/en/whats-next-in-the-evolution-of-standards-for-biologics-development https://edusehat.com/en/whats-next-in-the-evolution-of-standards-for-biologics-development Fri, 01 May 2026 21:20:20 +0700 Edusehat What’s, Next, the, Evolution, Standards, for, Biologics, Development
    Fouad Atouf
    Fouad Atouf, PhD
    Chief Science Officer
    United States Pharmacopeia (USP)

    Documentary standards such as monographs, general chapters, and reference standards are essential for efficiently meeting regulatory requirements and ensuring the intended efficacy and safety of drugs. At the most fundamental level, compendial standards for medicines, set by pharmacopeias such as the United States Pharmacopeia (USP), address common requirements such as sterility, endotoxin limits, particulate matter, and microbial contamination.

    At the product level, standards can address the measurement of specific quality attributes for different therapeutic modalities. For small-molecule drugs, the use of standards plays an important role in promoting competition and reducing medicine costs1.

    For biologics, standards are needed more than ever as novel modalities become a cornerstone of healthcare.2 Monoclonal antibodies (mAbs) and their biosimilars (follow-on versions of approved biologics) are among the most widely used biologics today. Since the first mAb was approved by the U.S. Food and Drug Administration (FDA) in 1986, more than 100 therapeutic mAb drugs are now on the market. In recent years, novel antibody formats such as antibody-drug conjugates (ADCs), bispecific antibodies, fusion proteins, and other biologics have shown significant growth.

    antibody
    Examples of critical quality attributes related to mAbs and biosimilars.
    [Adapted from Atouf and Venema.3 usp]
    As the pace of innovation accelerates, drug manufacturers strive to rapidly scale production to meet global patient demand, the need to ensure product consistency, quality, and safety grows more critical.

    Why biological standards matter

    Biological standards play a critical role in enabling innovation, ensuring product quality, and protecting patient safety across the product lifecycle. By providing established methods and reference materials, standards eliminate the costly and time-consuming duplication of effort required for manufacturers to develop and validate their own approaches to assessing product quality.

    For biosimilar developers, access to globally harmonized standards can enable access to methods vetted through a public standard-setting process and subsequently support a competitive marketplace. Standards also provide a stable foundation as analytical technologies evolve, allowing legacy methods to be bridged with newer techniques while maintaining confidence in data continuity and comparability over time.

    For therapeutic proteins, including mAbs, standards help define and control critical quality attributes. These attributes influence bioavailability, clearance, and functional activity and must be managed through robust control strategies that minimize manufacturing variability. By establishing common benchmarks and measurement tools, standards enable consistency.

    Harmonized standards help ensure manufacturing and lot-to-lot consistency and support reliable comparison of products over time, giving patients confidence that efficacy and safety are maintained when switching between an innovator product and a biosimilar or among biosimilars.

    From a regulatory perspective, standards create a common foundation for streamlined evaluation. When products’ dossiers are submitted using compendial methods, regulatory agencies such as the FDA and European Medicines Agency (EMA) traditionally accept these methods to demonstrate compliance with certain quality attributes, enabling more predictable regulatory review while preserving regulatory authority over final determinations of equivalence.

    The evolution of biologics standards

    The evolution of standards for biologics reflects the growing complexity and maturity of the field, highlighting the need for both general and product-specific guidance. Early efforts by pharmacopeias mirrored the approach used for small molecules, creating individual monographs for each biologic drug. This approach worked for simpler biologics like insulin or growth hormone, which could be reliably deployed using a defined set of standardized tests.

    However, as biologics became more complex, this model became increasingly challenging. Variability inherent to these molecules, often due to differences in manufacturing processes, made it difficult to define a single set of acceptance criteria that could consistently apply across products.

    In response, in 2010, our focus shifted toward developing standards that apply across product classes to support the analysis of product quality attributes (PQAs). These included broad, platform-based methods for measuring characteristics such as glycosylation, aggregation, and residual DNA, attributes shared across product classes or expression systems.

    These general chapters provided a strong foundational framework, which can be used throughout the product lifecycle and across diverse biologic products. There is now growing recognition of the complementary need for product-specific standards, especially for therapeutic proteins, which make up a significant portion of the biologics market.

    While general standards address common analytical needs, individual biologics like trastuzumab or erythropoietin have unique structural and functional characteristics that benefit from more tailored methods. Rather than shifting from one type of standard to another, the most effective approach blends both, supporting innovation while ensuring consistency, comparability, and regulatory confidence throughout the product lifecycle.

    To support ensuring the quality of complex biological products, these approaches are used:

    • Standards for individual biologic drugs, which historically focused on legacy biologics such as insulin drugs
    • Standards for cross-cutting attributes (e.g., measurement of glycosylation across biologics)
    • Platform-based methods and standards for testing products sharing production systems (e.g., mAbs made in CHO cells)

    USP is now engaging stakeholders to explore proposals for flexible documentary standards to support the quality of drug substances used in a wide range of biological products. These standards support biologics, including biosimilars, in three ways:

    • Ensuring consistency when making process changes or introducing new manufacturing processes
    • Enabling comparative analytical assessment when products move from single to multi-manufacturer settings
    • Detecting substandard products, which can otherwise go unnoticed without an appropriate benchmark

    Evolving biologics and standards

    As biologic products have evolved, they have become increasingly complex, expanding beyond mAbs to include ADCs, cell and gene therapies (CGTs), genome-editing platforms, and mRNA-based products. Standards remain critical to ensuring product quality, consistency, and patient safety, but those for emerging modalities cannot always take the same form as those applied to small molecules or conventional biologics.

    Next-generation therapies introduce new quality challenges that extend beyond the final drug product to individual components and upstream processes. For example, CRISPR-based therapies combine guide RNA, enzymes, and DNA templates, making it essential to control and characterize the quality of each building block. This represents a shift from a traditional end-product focus to a more integrated approach in which quality must be established throughout development and manufacturing.

    At the same time, not all advanced therapies are well-suited to traditional product-specific monographs. Some CGTs are highly individualized, developed for a single patient or small populations, making classical monographs impractical or unnecessary. In these cases, standards that address shared critical quality attributes across a product class or manufacturing platform are more appropriate.

    As scientific understanding and manufacturing practices mature, opportunities may emerge to develop more targeted standards that address challenges associated with scaling and consistency of manufacturing processes.

    mRNA-based therapies further illustrate this evolving landscape. While mRNA has a defined nucleotide sequence that could eventually enable biosimilar development, standards must address not only physicochemical attributes such as sequence integrity and purity, but also functional performance that varies depending on intended use, whether vaccination or therapeutic protein expression. Cross-cutting standards that support common analytical needs therefore represent a critical starting point, with more tailored standards following as consensus develops.

    Recognizing these realities, we are proposing a hybrid model designed to balance scientific rigor with flexibility. While monographs remain the gold standard for defining identity, strength, purity, and quality of small molecules and some well-characterized biologics, advanced modalities often require alternative approaches. The framework includes:

    • Informational general chapters that provide best practices and shared scientific understanding across product classes
    • Emerging standards that introduce early developmental concepts and invite community engagement
    • Procedural general chapters with accompanying reference standards to assess quality attributes common across a product class
    • Analytical Reference Materials (ARMs) that provide early benchmarks for assay development. These may evolve into reference standards to establish system suitability and to monitor assay performance for physicochemical and functional CQAs.

    Over time, elements of this hybrid approach may mature into compendial general chapters or full monographs, for example, when an emerging method for mRNA purity gains sufficient regulatory and industry support. This model maintains public trust and scientific rigor while remaining responsive to innovation and enabling global harmonization.

    Importantly, as advanced therapies mature and move into broader clinical and commercial use, the need for product-specific standards becomes increasingly important. Platform and cross-cutting standards establish a critical foundation, but they cannot fully capture the unique attributes and risks associated with individual products. Product-specific standards provide the precision needed to guide development, support comparability, and establish robust control strategies, accelerating progress while reducing duplication, cost, and uncertainty.

    Meaningful standards are only valuable if they are developed using scientific and public processes, adopted, and implemented. We must equip scientists and manufacturers with the knowledge to apply those tools effectively and cultivate awareness across the broader community about the value of standards.

    Continued collaboration among regulators, industry leaders, and standards-setting organizations is critical. Together, we can shape a standards ecosystem that keeps pace with scientific advances while providing the clarity and consistency needed for global development and regulatory approval, patient access, and patient health.

     

    References

    1. Murimi-Worstell B, Ballreich M, Seamans G, Alexander, C. Association between US Pharmacopeia (USP) monograph standards, generic entry and prescription drug costs. Published: November 12, 2019.

    2. Gupta, RK. The vital role of biological standardization in ensuring efficacy and safety of biological products–Historical perspectives. Journal of Pharmaceutical Sciences, 2025; 114(2): 690–700.

    3. Atouf F and Venema J. Do Standards Matter? What is Their Value? Journal of Pharmaceutical Sciences, 2020; 109(8): 2387-2392.

     

    Fouad Atouf, PhD, is the chief science officer at United States Pharmacopeia.

    The post What’s Next in the Evolution of Standards for Biologics Development appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Conserved Protein May Transform Anti-Inflammatory Strategies https://edusehat.com/en/conserved-protein-may-transform-anti-inflammatory-strategies https://edusehat.com/en/conserved-protein-may-transform-anti-inflammatory-strategies Fri, 01 May 2026 21:20:19 +0700 Edusehat Conserved, Protein, May, Transform, Anti-Inflammatory, Strategies

    Inflammation is at the core of many diseases. Yet, although many therapeutics have been developed to treat inflammatory diseases, they typically are designed to suppress the immune system, which uses inflammation as a tool to fight infection. Often, this inflammatory process gets out of control. That is the case with acute respiratory distress syndrome (ARDS), a complex condition that currently lacks any effective, approved treatments.

    BioAegis Therapeutics is flipping that paradigm by supplementing the body’s natural supplies of plasma gelsolin (pGSN) and enabling precision modulation of the inflammatory process. Unlike existing therapies for inflammatory diseases, pGSN works without suppressing the immune system. This highly conserved protein is largely overlooked by researchers but is at the core of immune functions, helping regulate innate immunity and localize inflammation, as well as helping clear pathogen and cellular debris. “It’s [routinely] circulating in our blood and can go to the points where it’s needed,” Susan Levinson, PhD, CEO, says.

    modulating levels of plasma gelsolin illustration
    By modulating levels of plasma gelsolin, an important regulator of inflammation, scientists expect to precisely modulate inflammation without suppressing the immune system. [BioAegis]

    “But there’s a catch,” she adds, pGSN is quickly depleted during the inflammatory process. “If the levels fall too low, the human or animal is at risk of severe morbidity or mortality.” Therefore, BioAegis restores pGSN levels with the identical recombinant protein to bring inflammation under control.

    BioAegis is the only company pursuing pGSN supplementation as a treatment for inflammation, although some others are investigating a target within the overall mechanism that pGSN interacts with.

    Human studies dosed healthy individuals with recombinant human pGSN (rhu-pGSN) levels “that were 10-fold higher than baseline with no concerning safety findings,” Levinson notes. It works by interrupting the activity of the NLRP3 inflammasome, acting directly on neutrophils and macrophages, which are responsible for countering threats like bacterial infection or injury.

    “I think of pGSN as a rheostat that can use the tools of the immune system to regulate inflammation up or down,” Levinson says.

    What most intrigues her, she says, is that while “rhu-pGSN is not an antibiotic, it has some antibiotic properties.” In animal trials involving severe bacterial infections such as gram-negative, gram-positive, and antimicrobial-resistant bacteria, flu, and pneumonia, it also helped cells kill the bacteria. In contrast, “The other anti-inflammatories suppress the immune system.”

    ARDS and decompression sickness

    BioAegis has an FDA Fast Track designation for clinical trials for two indications: ARDS and decompression syndrome. “These Fast Track designations acknowledge that we’re pursuing diseases of great importance and that have no alternative options currently available,” Levinson says. Fast Track designation increases the company’s access to the FDA “and is the first step in seeking a Breakthrough Designation, once we have clinical proof of concept,” she elaborates, which may then speed potential approval.

    “ARDS is the moonshot for us,” and is the focus of most of the company’s efforts, Levinson says. This life-threatening lung condition can lead to organ damage, organ failure and even death.

    Its fifth clinical study using pGSN, a global, 600-patient Phase II trial for ARDS, pGSN’s primary indication. “pGSN gives us precise modulation of the inflammatory process, and that’s what’s needed for ARDS,” Levinson says. Many cases of ARDS “are caused by infection. One of the drawbacks of all the anti-inflammatories is that they lower inflammation by suppressing the immune system, which is the last thing you want when you have an infection!” In contrast, pGSN does not suppress the immune system.

    Trial participants are severely ill. “Most of these patients are in the ICU, and most are on ventilators, which is similar to what happened during the COVID pandemic,” Levinson says. This trial is focused on the lung condition rather than any particular threat or infection. It compares outcomes of severely ill patients who are receiving standard of care with those who are additionally receiving pGSN.

    “We expect to have the first hints about its progress by the end of the year,” Levinson says.

    Another Phase II trial is underway to treat decompression syndrome. The work is based on research from a principal investigator at the University of Maryland that shows a connection to the inflammatory process. Specifically, levels of endogenous pGSN drop after dives, similar to what is seen in inflammatory diseases.

    By blocking activation of the NLRP3 inflammasome in neutrophils, rhu-pGSN “leads to decreased expression of those inflammatory nitrogen-containing particles…and the bubbles!” Levinson explains. Importantly, preclinical research suggests pGSN can prevent decompression syndrome when it is administered before a dive, and also can reverse it when administered after a dive. 

    “We’ve completed one study that we did in a what I call a controlled environment, basically, we recreated some of the conditions of diving in a hyperbaric chamber,” Levinson reports, to establish relevant biomarkers. A field study is being designed, supported by the U.S. Office of Naval Research, involving divers who encounter enhanced risks (such as commercial or military divers).

    BioAegis also is working with the Divers Alert Network to support sport divers—particularly cave divers in Florida. “It’s still early days. We’re still figuring out exactly how to do this,” she cautions.

    Early days

    BioAegis evolved from research conducted by Thomas Stossel, MD, professor of medicine (now deceased), at Harvard Medical School. “He discovered gelsolin and built a technology estate around it.”

    “When I heard about his technology, I was fascinated with the science and, in particular, the concept of this normal human protein that, when reduced, causes morbidity and mortality. It seemed like something that really needed to be brought to patients,” Levinson recalls.

    After working with Stossel to further refine the concept, Levinson and her co-founders created the company in 2011 and licensed the technology. Operations moved from Massachusetts to New Jersey—home to two of the four co-founders—to access some of that state’s business incentives.

    As the company matured, the challenges evolved from establishing the company to focusing on the most promising opportunities. “The science has provided many wins, but the opportunities far outweigh our ability to fund them, Levinson says. While the company maintains its tight focus on ARDS and, to a lesser extent, decompression syndrome, “We are continually looking for funders with the vision to reimagine the treatment of inflammatory disease!”

    Part of its strategy is to maximize effectiveness by outsourcing key portions of the work. BioAegis is a small company, she points out. As such, “We feel the best [approach] is to work with someone with great expertise in whatever is needed rather than scaling up…” personnel and infrastructure to support them.

    That approach led the team to a major biologics manufacturer to produce and lyophilize the protein, Levinson says. As a powder, pGSN is stable and easier to handle in a hospital environment, she adds.

    Once BioAegis gets preliminary data from its Phase II ARDS trial, it may expand into chronic diseases where, she says, “there are many opportunities…in autoimmune and neuroinflammatory diseases.” BioAegis also looks to expand beyond intravenous administration to make administration easier for patients with chronic conditions. 

    “We see rhu-pGSN as an opportunity to completely change the way inflammatory disease is managed in the future,” Levinson says.

    BioAegis Therapeutics

    Location: New Jersey Bioscience Center, 685 US-1, North Brunswick Township, NJ 08902

    Contact: slevinson@bioaegistx.com and www.bioaegistherapeutics.com/contact

    Website: www.bioaegistherapeutics.com

    Principal: Susan Levinson, PhD, CEO

    Number of Employees: 10

    Focus: BioAegis is developing recombinant human plasma gelsolin as a potential therapeutic for acute respiratory distress syndrome (ARDS) and other indications. A large, multinational Phase II trial is underway for that indication.

    The post Conserved Protein May Transform Anti-Inflammatory Strategies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Big Improvements for CGT Manufacturing as Development Scales Up https://edusehat.com/en/big-improvements-for-cgt-manufacturing-as-development-scales-up https://edusehat.com/en/big-improvements-for-cgt-manufacturing-as-development-scales-up Fri, 01 May 2026 21:20:17 +0700 Edusehat Big, Improvements, for, CGT, Manufacturing, Development, Scales As cell and gene therapies (CGTs) move from the research stage to commercial applications, biomanufacturers are seeing that many of the production techniques they relied upon at lab scale are no longer their best options.

    Life sciences solutions providers are well aware of the challenge and are responding with innovations of their own. New solutions include customized reagents that scale from bench to clinical trials, industrialized lentiviral vector manufacturing techniques, and innovations to boost lentiviral transduction.

    Scalable reagents

    Stephen Gunstream
    Stephen Gunstream, PhD
    CEO, Teknova

    Scalability is one hurdle. “The infrastructure for reagents is set up for blockbuster drugs that are made in 10,000 L batches. But, when you produce CAR T therapeutics and other personalized therapies for rare diseases, you may need only one liter of (a given) reagent…that may have a minimum order of 350 L,” Stephen Gunstream, CEO of Teknova, says. “Generally, these aren’t stock reagents that last a few years. They are made to order [for specific products].”

    Teknova small batch manufacturing
    Small batch manufacturing has the flexibility to make “the things you didn’t know you’d be making” when the facility was designed. [Teknova]

    Manufacturers’ dilemma, therefore, is that the custom, research-grade buffers they used for development lack the Good Manufacturing Practice (GMP)-level safeguards needed for human trials and may not be animal-free, but the animal-free, GMP products developers need aren’t available in small batches.

     

    Continuing to use research-use only (RUO) reagents through scale-up creates other challenges. Aside from the obvious sterility control and consistency concerns, “Changes in mixing and manufacturing during scale-up change the product. Making one liter of product is different from making 1,000 L.” The ability to use the same raw materials and manufacturing processes, but at the appropriate grade, would help manufacturers tremendously.

    Flexible small-batch facility

    To that point, three years ago, Teknova completed a new facility specifically for small batch, modular manufacturing. It produces custom reagents quickly and at scale and—importantly—at various grades. Using this new facility, “We’ve done custom batches as small as one liter,” Gunstream says.

    Step one involved designing a flexible, modular, regulatory-compliant manufacturing facility for RUO, RUO+, and GMP-grade reagents. This enables manufacturers to use the same reagents start to finish, selecting the appropriate grade for each step. That ability minimizes reagent costs, reduces risks, increases speed to market, and enhances product quality and consistency.

    The facility, Gunstream points out, has been validated under multiple production scenarios, and features a “robust microbial contamination strategy that scales across manufacturing grades.”

    Things you didn’t know you’d make

    Teknova employs a bracketed validation strategy to create “a quality system designed to work with things you didn’t know you’d be making,” Gunstream says. That entailed analyzing raw materials used for custom reagents, knowing which have been used successfully in approved products, and validating these materials themselves at certain ranges.

    To smooth development and scale-up, Gunstream advises selecting a supplier that:

    • Can scale from beginning to clinical trials
    • Is flexible and fast
    • Has experience making small, custom batches
    • Shares their insights

    Manufacturing LVVs at scale

    With roughly one dozen CAR T therapies in or nearing clinical trials, no single delivery technology dominates.

    “From a manufacturing standpoint, this [plethora of options] places strong demands on vector production, purification, analytics, and scalability. As the field evolves, robust, flexible manufacturing platforms are essential,” Brian Tomkowicz, PhD, vice president and head of R&D and virology fellow, SK pharmteco, said in a recent webinar.

    SK pharmteco is optimizing production of lentiviral vectors (LVVs), one of the go-to delivery CGT options, by industrializing ex vivo LVV production even as CAR T therapeutic delivery shifts toward in vivo modalities. With that shift, LVVs are defined as a drug product rather than an intermediate. Consequently, “The demands placed on antiviral vector manufacturing are shifting dramatically,” Tomkowicz said.

    “An under-appreciated reality in LVV manufacturing is that downstream yield is overwhelmingly constrained by ion exchange chromatography (IEX) recovery,” he pointed out. “Although upstream harvest and clarification stems preserve the majority of vector material, a substantial loss occurs during the ion exchange step…even in processes that are optimized.” Consequently, “Total [functional particle] recovery often plateaus around 15% to 40%, with 20% being typical.”

    In contrast, SK pharmateco’s high-throughput convective IEX delivers a functional particle recovery rate that ranges from 60% to 90%. Charts show a 98% host cell protein clearance and a transducing unit recovery rate exceeding 85% across multiple runs. To obtain such results, SK pharmateco replaced IEX’s traditional packed-bed resin with a membrane adsorber.

    IEX, using a membrane absorber, cut total processing time to one hour or less (down from two to four hours using resin). That’s thanks in part to load capacities up to 250 mL/MV and residence times of less than six seconds, which help make throughput more than five times faster than packed-bed resin-based IEX. “This enables same-day processing,” Tomkowicz said.

    Importantly, this high-throughput, membrane absorber-based IEX performance is maintained as process stress increases. Multiple tests involving a variety of input concentrations and higher viral particles measured physical recovery and functional titers. The highest levels of recovery peaked at 60–85%.

    “Membrane [IEX] enables smaller footprints, disposable formats, and substantially lower material and buffer costs across both the 50L and 500 L production scales. This results in a six- to 20-fold cost reduction,” Tomkowicz explained.

    The total cost of producing a 50 L batch using membrane adsorber IEX is about $3,000, he says, down from the $60,000 to $70,000 cost of a resin-based approach.

    The most important optimization lesson, he said, is that “Recovery is governed by mass transfer physics rather than brand-specific attributes.”

    As the industry moves from ex vivo to in vivo modalities, “The key difference is not just how we manufacture an LVV, but what we package inside it. Therefore, architecture and rational payload design become essential to allow manufacturers to exploit variants,” Tomkowicz emphasized. “While our focus is on scalable manufacturing, payload design and process requests must develop together.”

    Tomkowicz called this a simple, scalable, fit-for-purpose platform for clinical and commercial manufacturing, predicting it will be “a manufacturing inflection point.” Before this method is widely adopted, however, Tomkowicz said he would like to see the work expanded to more vectors and more patients.

    LVVs for adoptive cell therapy

    Chris Lowe
    Chris Lowe, PhD
    LentiBOOST business leader
    Revvity

    “Perhaps the greatest challenge in the field of CGT today is the high cost of developing and manufacturing lentiviral vectors,” Chris Lowe, PhD, LentiBOOST business leader at Revvity, says. “One of the key drivers of this cost is the difficulty in achieving reproducible lentiviral transduction efficiency across a range of clinically relevant cell types (including T cell, hematopoietic stem cells, and progenitor cells) without compromising viability, phenotype, or function,” he tells GEN.

    The issue manufacturers face is that even optimized vectors may require a high copy number if transduction is inefficient. “Transduction efficiency, therefore, can be the difference between a program that reaches the clinic and one that gets stuck in the lab because vector supply, manufacturing costs, or product quality are not sustainable at scale,” Lowe points out.

    To address this challenge, industry scientists are exploring approaches that include optimizing vector design and production, improving physical and process-based activities, and developing chemical and biological enhancers.

    For example, transduction can be improved by refining envelope pseudotypes, promoter selection, and genome architecture to improve tropism, especially when those technologies are accompanied by advances in vector manufacturing that increase functional titer and consistency. “While beneficial,” Lowe says, “these strategies enhance transduction indirectly and can increase manufacturing complexity.”

    LentiBOOST illustration
    LentiBOOST™ increases cell permeability, facilitating the entry of lentivirus particles into the cell. [Revvity]

    Likewise, spinoculation, controlled temperature shifts, and other physical and process-based methods to increase virus-cell contact (often combined with media and serum optimization) may improve cell fitness during transduction but are not without their own challenges. “Specifically,” Lowe cautions, “care must be taken to avoid issues with cell health, and these techniques can be challenging to scale up. These methods are often best paired with complementary chemical and biological enhancers.”

    Such enhancers typically increase lentiviral transduction by promoting virus-cell interaction of membrane fusion, Lowe elaborates. “Traditional approaches such as polycations and extracellular matrix components are well established but can be associated with cytotoxicity, activation of cellular stress pathways, variable performance across primary cell types, and limited availability in consistent GMP-grade formats.

    “Polymer-based enhancers offer an alternative approach that addresses some of these limitations,” Lowe continues. “Non-ionic poloxamer formulations, including technologies such as [Revvity’s] LentiBOOST™ enhancer, have been shown to improve transduction efficiency and increase vector copy number while maintaining cell viability.”

    By providing a receptor-independent way to modulate the interface between viral and cellular membranes, the poloxamer in Revvity’s LentiBOOST technology promotes fusion. This increases the odds that each particle will deliver its cargo successfully across lentiviral constructs and multiple cell types—including primary T cells and human stem cells.

    Multiple options

    Today the industry is exploring “a variety of different approaches to enhance lentiviral transduction, including nanoparticle-assisted delivery, transient pathway modulation, and next-generation surface or matrix technologies that improve virus—cell contact or intracellular trafficking,” Lowe says.

    That said, there are strong reasons to optimize the cellular environment. “Lentiviral transduction is most efficient when cells are metabolically active, not stressed, and can support membrane fusion and reverse transcription,” he says. Revvity, through BioLegend, which it acquired in 2021, also offers cell culture reagents in its Cell Vive™ family.

    As CGT developers look to increasingly challenging cell types and ever-more-complex engineering, “there is a corresponding need to improve transduction efficiency…to increase therapeutic response rates, and to lower manufacturing costs,” Lowe says.

    In the future, he adds, “Lentiviral transduction will increasingly coexist with non-viral and hybrid delivery systems. In that context, effective, GMP-grade enhancers will help ensure that lentiviral vectors remain a cornerstone technology where stable, durable gene delivery is required.”

    With the plethora of advances being developed today, CGT manufacturers are on the cusp of improvements that will reduce process times, cut costs, enhance flexibility, and support scalability.

     

    Fouad Atouf, PhD, is the chief science officer at United States Pharmacopeia.

    References

    1. Murimi-Worstell B, Ballreich M, Seamans G, Alexander, C. Association between US Pharmacopeia (USP) monograph standards, generic entry and prescription drug costs. Published: November 12, 2019.

    2. Gupta, RK. The vital role of biological standardization in ensuring efficacy and safety of biological products–Historical perspectives. Journal of Pharmaceutical Sciences, 2025; 114(2): 690–700.

    3. Atouf F and Venema J. Do Standards Matter? What is Their Value? Journal of Pharmaceutical Sciences, 2020; 109(8): 2387-2392.

    The post Big Improvements for CGT Manufacturing as Development Scales Up appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Smarter AAVs Drive Gene Therapy’s Next Chapter https://edusehat.com/en/smarter-aavs-drive-gene-therapys-next-chapter https://edusehat.com/en/smarter-aavs-drive-gene-therapys-next-chapter Fri, 01 May 2026 21:20:15 +0700 Edusehat Smarter, AAVs, Drive, Gene, Therapy’s, Next, Chapter Gene therapy has entered a complicated phase. Over the past several years, the field has delivered remarkable scientific breakthroughs alongside sobering challenges—clinical safety concerns, manufacturing bottlenecks, and treatments priced in the millions of dollars. Some biotechnology companies have scaled back programs, while investors have grown cautious about the pace of commercial progress.

    Despite these growing pains, gene therapy remains one of the most promising strategies for treating genetic diseases. Beneath the headlines, researchers and technology developers are quietly transforming the infrastructure that makes these therapies possible.

    At the center of that transformation is the adeno-associated virus (AAV), a tiny viral vector that has become the dominant platform for directly delivering therapeutic genes into human tissues. AAV-based therapies have already shown encouraging clinical results in diseases ranging from inherited retinal disorders to neurological syndromes.

    But the rapid expansion of AAV-based therapies has also exposed important limitations. Manufacturing these complex biological products at scale is difficult, ensuring consistent product quality requires advanced analytical tools, and naturally occurring AAV variants often lack the tissue-specificity needed to efficiently deliver genes without high doses.

    Across the cell and gene therapy ecosystem, scientists and companies are now addressing these challenges through innovations in bioprocessing, vector engineering, and computational design. These advances could determine whether gene therapy evolves into a sustainable therapeutic platform capable of reaching larger numbers of patients.

    Building scalable workflows

    Manufacturing viral vectors remains one of the most technically demanding aspects of gene therapy development. Producing AAVs requires complex biological systems, typically involving cultured mammalian cells that generate the virus after being supplied with the necessary genetic components.

    As gene therapy programs move from laboratory research into clinical trials, production requirements expand rapidly. Processes that work well at a small scale often struggle to maintain efficiency and consistency when scaled to large bioreactors.

    One crucial challenge involves balancing upstream production—where the virus is generated—with downstream processing—where the vector is purified and formulated for clinical use.

    Pouria Motevalian, PhD, director, CMC development, pharma services, at Thermo Fisher Scientific, says that successful scale-up depends on developing a detailed scientific understanding of the process early in development.

    “Effective downstream scale-up begins with the establishment of reliable scale-down models,” Motevalian explains. “These models are then used to define the design space—the scientifically established operating region in which critical process parameters and material attributes can vary without compromising product quality.”

    This concept of design space comes from quality by design, a development philosophy that emphasizes understanding how manufacturing variables influence product attributes. By mapping these relationships early, developers can design processes that remain robust as they scale.

    “Parameters are defined not only by target setpoints, but also by proven operating ranges,” Motevalian says. “As a result, facility, equipment, and material constraints can be accommodated during scale-up without compromising process performance or product quality.”

    Such flexibility becomes increasingly important as upstream technologies improve. New cell-culture strategies, optimized media formulations, and refined production platforms are allowing developers to generate higher quantities of AAV particles. But these gains can introduce downstream bottlenecks if purification systems are not prepared to handle the additional load.

    “As upstream yields increase, downstream bottlenecks related to loading capacity, filtration throughput, buffer demand, and facility fit can be proactively addressed while maintaining recovery and product quality,” Motevalian notes.

    Automation and high-throughput development platforms are also helping accelerate process optimization. These technologies enable rapid testing of multiple process conditions, allowing researchers to evaluate purification strategies and more efficiently optimize workflows.

    Transforming viral-vector analytics

    Manufacturing improvements alone cannot support large-scale gene therapy production. Developers must also demonstrate that each batch of viral vectors meets strict quality standards, which places heavy demands on analytical technologies. “The latest innovation in viral-vector analytics is centered on faster turnaround, lower sample consumption, and more multiplexed methods,” Motevalian says.

    Traditional techniques, such as analytical ultracentrifugation (AUC), remain essential for evaluating vector composition, but emerging complementary technologies are providing additional insights. “Increasing emphasis is being placed on rapid orthogonal tools, such as mass photometry, as complements to gold-standard methods, such as AUC,” Motevalian explains. “At-line approaches, such as size-exclusion chromatography with multi-angle light scattering, also enable quicker assessment of vector-genome and capsid titers.” These methods allow developers to monitor vector quality more quickly during development, reducing delays and enabling faster decision-making.

    Meanwhile, molecular techniques are becoming more efficient. Multiplex droplet digital PCR (ddPCR) assays, for example, allow researchers to simultaneously analyze several genomic regions. “Multiplex ddPCR strategies are expanding analytical efficiency by enabling simultaneous interrogation of multiple genome regions and selected impurities,” Motevalian says.

    Another emerging trend is the adoption of multi-attribute analytical methods based on liquid chromatography–mass spectrometry (LC-MS). These approaches allow several critical quality attributes to be monitored within a single analytical workflow.

    The ability to track multiple parameters simultaneously strengthens process understanding and supports regulatory comparability assessments.

    Engineering better vectors

    Although manufacturing and analytics are improving rapidly, gene-therapy developers must still contend with the biological limitations of naturally occurring AAV vectors. Many widely used AAV serotypes were discovered decades ago and did not originally evolve for precision gene delivery in humans. As a result, they often lack the targeting specificity needed for efficient therapy.

    “There are three main roadblocks, and they are all interdependent: delivery, safety, and the cost of scaling up manufacturing,” says Amos Gutnick, PhD, associate director of product development at PackGene. “Natural AAVs, like AAV9 or AAV2, still struggle with precision, so patients currently require massive doses in order to benefit from a therapeutic effect.”

    Those high doses can “introduce serious safety risks, like liver toxicity and adverse immune reactions,” Gutnick notes. “They also drive production costs through the roof, often making the program commercially untenable.”

    To overcome these obstacles, developers are working simultaneously on improving vector design and optimizing manufacturing efficiency. “At PackGene, our company motto is literally: ‘Make Gene Therapy Affordable,’” Gutnick says. Part of that effort involves improving bioprocessing platforms to generate higher yields and better quality vectors.

    “On the manufacturing end, we’re constantly innovating our bioprocessing platforms to boost overall yields and purity,” Gutnick explains. “These innovations empower us to manufacture GMP-grade plasmids at record-breaking low costs and maximize the percentage of full, functional AAV capsids at every scale.”

    AAV-capsid screening workflow
    This infographic shows the AAV-capsid screening workflow using PackGene kits, where pooled barcoded variants are delivered to cells or models and analyzed by NGS for entry and gene expression. [PackGene]
    At the same time, advances in capsid engineering are enabling more precise gene delivery. “On the design side, we work to engineer novel AAV capsids that are highly specific to the target tissue with the goal of increasing safety and drastically lowering the therapeutic dose,” Gutnick says. “When you combine better manufacturing yields with much lower clinical doses, the economics completely change.” And, he adds, “that can mean the difference between an interesting paper and an actual, commercially viable gene therapy program.”

    AI accelerates vector design

    Artificial intelligence (AI) is increasingly shaping how new viral vectors are developed. Machine-learning algorithms can analyze large datasets describing how capsid sequences behave in biological systems and use those insights to design improved variants.

    As Gutnick says, “AI is a cornerstone of our π-Icosa Capsid Engineering Platform.” These models are trained to optimize both biological performance and manufacturability. “Biologically, the AI helps us design novel AAV capsids with incredible tissue targeting while actively detargeting the liver,” Gutnick explains.

    Avoiding liver accumulation is particularly important because the liver often receives a large share of systemically delivered vectors and can become a source of toxicity. Equally important is ensuring that engineered capsids can be produced efficiently. “But just as importantly, we use AI to design for high manufacturability from day one,” Gutnick says.

    Historically, some promising vectors discovered in the laboratory proved difficult to manufacture at scale. Designing with production in mind helps reduce that risk.

    Faster effective-vector discovery

    Identifying the right AAV capsid for a particular therapeutic application can take years. To accelerate this process, researchers are developing tools that allow large numbers of vector variants to be tested simultaneously.

    As Gutnick says, “One of the biggest bottlenecks for translational researchers is simply the time and money it takes to find the right vector.”

    To help address that challenge, PackGene collaborated with the Children’s Medical Research Institute to develop capsid-discovery kits that enable multiplex screening experiments. “This partner used this off-the-shelf kit to run high-throughput, multiplexed in vivo screens on dozens of capsids all at once,” Gutnick explains.

    The screening system uses next-generation sequencing (NGS) to track both where vectors travel in the body and whether they successfully express their genetic payload. “By using dual NGS readouts to track both where the AAV went and if it actually worked, they were able to quickly pinpoint a lead capsid that hit their target tissue perfectly while avoiding off-target areas,” Gutnick says. “By giving researchers access to these kits, we’re helping them cut months—even years—off their discovery timelines.”

    Platforms streamline production

    Although improvements in vector design and analytics are essential, the infrastructure needed to manufacture gene therapies at scale is also evolving.

    For example, Catalent developed an AAV production platform designed to accelerate the path from gene discovery to clinical manufacturing. The approach relies on standardized suspension HEK293 cell culture systems and integrated supply chains that combine plasmid production, process development, and manufacturing services.

    Charles River Laboratories introduced its nAAVigation vector platform to streamline viral-vector development. Built around a high-productivity HEK293 suspension cell line and optimized upstream and downstream processes, the platform is intended to reduce development timelines while enabling scalable production.

    Synthetic biology company Asimov is exploring another strategy through its AAV Edge Stable Producer System. Rather than relying on transient transfection for each production run, the system uses engineered producer cell lines in which viral genes are integrated directly into the genome. These stable cell lines can produce AAV vectors more consistently and might reduce manufacturing costs by eliminating the need for multiple plasmids.

    Meanwhile, Andelyn Biosciences is applying its AAV Curator manufacturing platform to support gene-therapy programs targeting rare diseases. The company is collaborating with the Drake Rayden Foundation and researchers at the University of Texas Southwestern to manufacture clinical-grade vectors for a potential therapy aimed at nonketotic hyperglycinemia, a severe inherited metabolic disorder.

    Toward a sustainable ecosystem

    The rapid evolution of AAV technology reflects a broader shift in gene therapy from experimental science toward industrial-scale medicine.

    Manufacturing innovations are enabling more efficient production of viral vectors. Advanced analytics are providing deeper insight into vector quality and performance. And computational approaches are unlocking new possibilities for designing safer, more precise delivery systems.

    Together, these advances suggest that the next chapter of gene therapy will be defined not only by scientific discovery but also by the ability to manufacture and deliver treatments reliably and at scale. If those efforts succeed, AAV vectors could help transform the treatment of genetic disease—turning once-experimental therapies into widely accessible medicines capable of changing patients’ lives.

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    ]]>     Gene Editing at Scale, Clinic Seeks Generalizable Therapies https://edusehat.com/en/gene-editing-at-scale-clinic-seeks-generalizable-therapies https://edusehat.com/en/gene-editing-at-scale-clinic-seeks-generalizable-therapies Fri, 01 May 2026 21:20:14 +0700 Edusehat Gene, Editing, Scale, Clinic, Seeks, Generalizable, Therapies Ajay Gannerkote, president of Integrated DNA Technologies (IDT), says what’s most exciting about CRISPR is its potential to shift medicine from managing disease to directly correcting its root cause. “For patients with severe genetic conditions, especially those with no existing treatment options, that represents a fundamental change in what’s possible,” he said.

    IDT played a pivotal role in manufacturing the personalized gene editing therapy given to baby KJ Muldoon to treat his rare metabolic disorder. Today, KJ is free from the toxic ammonia buildup that drives a 50% mortality rate for his condition in infancy. While his story highlights the life-changing potential of gene editing, the field now wrestles with the next challenge: expanding these therapies to benefit broader patient populations.

    In contrast to KJ’s urea cycle disorder, which stemmed from a single disease-causing mutation that could be precisely targeted, many genetic disorders arise from numerous mutations scattered across a gene where individualized corrections are too resource-intensive to scale.

    Gannerkote says turning powerful gene editing tools into broadly accessible clinical therapies requires progress across multiple fronts. Many CRISPR therapies are still bespoke, with manufacturing processes that are not yet standardized or easily repeatable, leading to long timelines and high costs. In regulation, therapy developers and government regulators face a learning curve when evaluating new modalities, particularly when speed is critical for patients with life-threatening conditions.

    Today’s gene editing companies reflect on what’s required to scale personalized CRISPR therapies for maximized impact in the clinic.

    End-to-end

    Sadik Kassim, PhD, CTO of Genomic Medicines at Danaher, explains that personalized therapies do not naturally lend themselves to traditional drug-development models. Gene editing companies are now seeking “platformization,” where common manufacturing processes are standardized, and limited elements, such as guide RNAs, are customized for each patient to reduce costs and speed timelines.

    “Baby KJ’s treatment succeeded because multiple elements aligned simultaneously,” explained Kassim. The foundational science, which achieved successful gene corrections in animal models of phenylketonuria (PKU), an inherited metabolic disorder caused by mutations in the PAH gene that impair the enzyme responsible for breaking down phenylalanine, had already been developed in the academic labs led by Children’s Hospital of Philadelphia (CHOP) physician scientists, Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD. Teams were then able to move quickly when the clinical need became clear.

    Regulatory engagement was also critical. Danaher teams worked directly with the FDA to streamline the treatment approval process without compromising patient safety. That collaboration compressed a timeline that would normally take 18–24 months down to roughly six months.

    “Replicating this for future patients will require moving away from one‑off efforts and toward repeatable platforms with established processes, validated assays, and clearer regulatory precedents, so that speed becomes the norm rather than the exception,” Kassim said.

    Amy Pooler, PhD, CSO of ElevateBio, agrees that the transition steps between therapy design and manufacturing are often where the greatest delays occur. ElevateBio seeks to address this bottleneck by building an end-to-end genetic medicine platform.

    “A critical driver for the company is making sure we have a clear line of sight into manufacturing from the very beginning,” Pooler said. “One reason Baby KJ’s case was successful is that Danaher managed the handoffs smoothly.”

    Pooler also describes developing genetic medicines as “building the plane while you’re flying it.” The field still lacks enough data to reliably predict patient outcomes. Every clinical trial readout provides a valuable lesson for the field.

    “I’m excited about the clinical evidence that’s starting to accumulate, showing gene editing can be transformative for patients, which we didn’t have five to ten years ago,” she said.

    Large gene, generalizable therapy

    ElevateBio’s expanding CRISPR toolbox includes base, prime, and epigenetic editing. Notably, the Durham-based company’s AI platform generates novel recombinases for targeted gene insertion, an approach that holds promise as a generalizable medicine that could treat patients regardless of their underlying disease-causing mutation.

    Using AI-guided design, ElevateBio explores entirely new regions of protein space to discover potent and highly specific recombinases that expand the range of diseases amenable to gene editing. These engineered enzymes, which possess 50% or less homology to known proteins, can access novel genomic regions that remain difficult to target with existing CRISPR technologies.

    Ben Kleinstiver, PhD, associate investigator at Massachusetts General Hospital (MGH) and co-author of the NEJM study describing KJ’s case, says the FDA’s Plausible Mechanisms Pathway has helped address some of the regulatory challenges to streamline the path to the clinic. Yet, there remains a major motivation for pan-mutation approaches that are more widely applied across patients.

    Kleinstiver’s research group, in collaboration with Full Circles Therapeutics, recently developed a circular single-stranded DNA donor (ssDNA) that enables safer kilobase-scale integration for human cells.1 The technology provides an alternative to double-stranded DNA (dsDNA) donors that evoke harmful immune responses yet are required for recognition by the diverse suite of genome editing enzymes. Notably, the new circular donor maintains recombinase compatibility by attaching a short region of dsDNA that can go undetected by the cytosolic DNA sensor and immune system activator, cGAS.

    Patients now

    While the gene editing field often concentrates on large indications driven by a single common mutation, Edward Kaye, MD, CEO and director of Aurora Therapeutics, aims to extend these technologies beyond the “lucky few” who share the same mutation.

    Aurora’s leadership team
    Aurora’s leadership team, from left: Morgan Maeder, PhD, Edward Kaye, MD, and David Litvak, MBA [Aurora]

    Co-founded by Jennifer Doudna, PhD, CRISPR Nobel Laureate, and Fyodor Urnov, PhD, scientific director of the Innovative Genomics Institute, Aurora launched in January to build a sustainable pipeline to scale rare disease treatments. Traditionally, developing therapies for these ultra-rare or N-of-1 conditions can require several million dollars for a single patient.

    Aurora is pursuing an “umbrella IND” strategy that allows multiple guide RNAs to be evaluated within a single clinical trial. The company’s initial focus is on PKU.

    PKU offers several advantages for early clinical development. Patients are routinely identified through newborn screening programs shortly after birth, which facilitates trial participant identification and enrollment. The condition also benefits from a clear regulatory precedent: reductions in phenylalanine levels are an established clinical endpoint used to move therapies toward approval.

    “What we learn from PKU will be used for many other diseases because we have the systems in place,” said Kaye. “It expands gene editing into many more patients, by going after one disease first.”

    Kaye also stresses the importance of engaging patient communities, whose input can ensure studies and regulatory processes are not overly burdensome for patients and families.

    Maher Masoud, CEO of MaxCyte, emphasizes putting patients at the forefront. He adds that most gene-editing therapies in the clinic require significant patient conditioning, which can lead to lengthy treatment cycles and clinical trial timelines. Yet he sees these barriers to scale being eroded over the near term. As an example, modalities, such as allogeneic cell therapies, require far less patient conditioning and easier dosing regimens to support cheaper therapies.

    In 2013, MaxCyte partnered with CRISPR Therapeutics on early work that led to the first FDA-approved therapy based on CRISPR-Cas9, Casgevy, with MaxCyte’s ExPERT electroporation platform enabling the efficient delivery of gene editing machinery into cells.

    More than a decade later, the company has developed more than 1,000 applications and protocols. The broad engineering platform can repeatedly engineer batches of at least 20 billion cells using CRISPR-Cas9 in addition to base and prime editing.

    Masoud says low-significant gene editing commercial success has been a bottleneck to scaling personalized therapies. Yet, he reiterates that CRISPR and other gene editing technologies were discovered a short 12 years ago.

    “With CRISPR, we are finally seeing cures, Casgevy, LYFGENIA, and baby KJ are proof of that,” he says. “This is just the beginning.”

     

    References

    1. Tou, C.J., Xie, K., Ferreira da Silva, J., et al. Invasive DNA donors and recombinases license kilobase-scale writing. Nature. 2026. DOI: 10.1038/s41586-026-10241-z.

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    ]]>     Breaking Through the Barrier https://edusehat.com/en/breaking-through-the-barrier https://edusehat.com/en/breaking-through-the-barrier Fri, 01 May 2026 21:20:13 +0700 Edusehat Breaking, Through, the, Barrier According to the American Brain Foundation, over one in three people around the world are affected by neurological conditions, the leading cause of illness and disability worldwide. This silent epidemic is not country-specific. Neurological conditions such as lysosomal storage disorders, rare enzyme deficiencies, and Alzheimer’s and Parkinson’s disease take their victims, regardless of age, race, or location.

    For decades, scientists have struggled to deliver therapeutics to the brain, only to be thwarted by the highly protective blood-brain barrier (BBB). First-generation approaches demonstrated proof of principle but still require advancements to improve the ability to reach specific areas of the brain, or specific cell types, safely, and with sufficient dosage to enable meaningful therapeutic effects.

    Although much remains unknown generally about brain biology and its defensive mechanisms, novel therapies for devastating neurological diseases are progressing into clinical trials. There is no magic bullet—no promises, no cures—but a gleaming light can be seen in this particular long and dark tunnel.

    Dedicated scientists continue to work on gene therapies for the indications that most benefit from a once-and-done approach, in addition to neurological shuttles to address those disorders that require therapeutic tempering and dosage control.

    Expanding platform technologies

    In 2021, JCR Pharmaceuticals received regulatory approval for the first biotherapeutic, IZCARGO™ (pabinafusp alfa), designed to cross the BBB to deliver a therapeutic enzyme for the treatment of a lysosomal storage disorder called mucopolysaccharidosis type II (MPS II) or Hunter syndrome.

    The platform technology has been expanded to exploit receptor-mediated transcytosis (RMT) to address other lysosomal storage and neurodegenerative diseases. Still, delivery to specific cells or parts of the brain remains challenging, along with efficient delivery of antisense oligonucleotides or siRNA.

    “The issue is not delivery across the BBB, but the endosomal escape to efficiently suppress the target RNA,” said Hiroyuki Sonoda, PhD, representative director, president, and CSO, at JCR Pharmaceuticals. “Small molecule CNS delivery is related to physicochemical properties. The structural design needs to make them lipophilic, yet also able to evade typical transporter clearing mechanisms.”

    blood-brain barrier penetration technology diagram
    The first approved blood-brain barrier penetration technology was developed into the J-Brain Cargo platform that can help drugs cross the blood-brain barrier. [JCR Pharmaceuticals]

    J‑Brain Cargo® uses RMT, mainly focusing on the transferrin receptor (TfR). Other promising candidates target different receptors. “We have successfully transported enzymes, antibodies, peptides, decoy receptors, antisense oligos, and siRNA into the CNS,” commented Sonoda. J‑Brain Cargo is particularly suited for enzyme replacement therapies in lysosomal storage disorders and conditions where dose control, reversibility, and titration are important.

    For gene therapies, JCR developed the JUST-AAV platform technology. Novel changes in the capsid almost completely eliminate liver tropism. The modified capsids express miniaturized antibodies on the capsid surface against receptors on selected tissues, organs, or the BBB, enhancing targeted delivery. JUST‑AAV is for diseases where continuous transgene expression is desired to achieve the optimal effect.

    Several candidates are in global clinical trials, including JR-141 (pabinafusp alfa) for individuals with MPS II (also known as Hunter syndrome), JR-171 to treat MPS I (also known as Hurler, Hurler Scheie, or Scheie syndromes), and JR-441 for individuals with MPS IIIA (also known as Sanfilippo syndrome A).

    Programs in collaboration with MEDIPAL HOLDINGS CORPORATION are in different stages of clinical and pre-clinical development for individuals with MPS IIIB (also known as Sanfilippo syndrome B), Fucosidosis, and GM2 gangliosidosis (including Tay-Sachs and Sandhoff disease).

    Collaborating with leading pharmaceutical companies is core to JCR’s strategy to bring these platform technologies to broader application. “We enable our partner by turning their biologics into CNS-penetrating versions of their original molecule,” said Sonoda.

    JCR manufactures most of its drug products in-house. Last year, they were selected for the Ministry of Economy, Trade and Industry’s “Regenerative CDMO Subsidy” to expand biomanufacturing capacity for regenerative, cell, and gene therapies.

    Optimizing BBB transport

    “Protein engineering architecture differentiates our delivery technology along with its optimization for efficacy, safety, and tolerability,” said Ryan Watts, PhD, co-founder and CEO of Denali Therapeutics.

    The TransportVehicle™ (TV) technology has the RMT binding site integrated directly into the constant domain (Fc) of an antibody for optimal properties and modularity. This allows the same TV sequences to transport a range of large molecule biotherapeutics such as enzymes, oligonucleotides, and antibodies for systemic administration. The engineered Fc domains bind to specific natural transport receptors expressed at the BBB, such as TfR.

    Transport Vehicle technology illustration
    The integration of the receptor-mediated transcytosis binding site into the TransportVehicle (TV) technology allows the same TV sequences to transport a range of large molecule biotherapeutics, such as enzymes, oligonucleotides, and antibodies, for systemic administration. [Denali Therapeutics]

    “Our research recently demonstrated that a TV platform-enabled anti-Ab antibody improved distribution in the brain and significantly reduced risk of Amyloid-Related Imaging Abnormalities (ARIA) in a mouse model of Alzheimer’s disease, when compared with a conventional anti-Ab antibody.1 The study provides the first mechanistic insight for mitigating the risk of ARIA,” detailed Watts.

    The Enzyme TransportVehicle (ETV) contains a fusion of a therapeutic enzyme. The Fc portion of the fusion molecule binds the apical surface of the TfR to avoid interference with normal iron transport.

    In March 2026, Denali’s lead ETV program, Avlayah™ (tividenofusp alfa-eknm), received FDA accelerated approval for the pediatric treatment of the lysosomal storage disorder MPS II. Avlayah is the foundation for their broader ETV franchise, addressing other lysosomal storage disorders such as MPS IIIA. Results from the open-label Phase I/II clinical trial are available.2

    Their Oligonucleotide TransportVehicle (OTV) platform is an engineered TV conjugated to an oligonucleotide for the systemic delivery of genetic medicines to the brain. Extensive characterization and research demonstrate the ability of OTV to elicit broad biodistribution of oligonucleotide therapies throughout the CNS following systemic exposure.

    “For example, our investigational therapy DNL628 for the treatment of Alzheimer’s disease is designed to cross the BBB and reduce the tau protein by targeting the MAPT gene that encodes for tau,” explained Watts.

    Lastly, the Antibody TransportVehicle (ATV) platform is designed to enable brain delivery of antibodies capable of selective immune activation and a targeted therapeutic approach after intravenous administration. The investigational anti-Ab antibody therapy DNL921, for example, is designed to reduce amyloid plaques and avoid ARIA.

    The TV-enabled clinical development portfolio also includes candidates for frontotemporal dementia-granulin and Pompe disease.

    Advancing clinical options

    “It is exciting to begin to see that delivery through the BBB is possible using gene therapy or shuttle approaches,” said Todd Carter, PhD, CSO at Voyager Therapeutics. Although first-generation therapeutics are demonstrating meaningful levels of delivery, optimization, and improvement of the functionality, exposure duration, and therapeutic effects are still needed.

    “For some diseases, gene therapy is the preferred treatment modality, as both the capsid and the payload can be modified to perform a specific job,” said Carter. But viral vector delivery for gene therapy has had problems with liver-based toxicity.

    For the best human translation opportunities, Voyager developed a model in non-human primates (NHPs) requiring cross-species activity across multiple NHP species. This strategy resulted in the company’s TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) technology, used to screen tens of millions of vector variants using barcoded libraries in which capsids were modified with slight insertions of seven to nine amino acids.

    TRACER, Voyager’s unbiased capsid and receptor discovery engine
    TRACER, Voyager’s unbiased capsid and receptor discovery engine, identified ALPL as a broadly enabling brain delivery receptor. [Voyager Therapeutics]

    Successful expression in neurons demonstrated that the capsids crossing the BBB worked. Directed evolution improved them. “Next, we needed to determine the mechanism—the receptors they were targeting,” said Carter. This led to the identification of the receptor, alkaline phosphatase (ALPL), tissue nonspecific.

    Now, Voyager has multiple families of capsids that mediate delivery into the brain, are detargeted from the liver, and, for the most advanced, have improved the capsid’s ability to target the brain using ALPL. “Using the ALPL receptor elevates delivery to the brain and allows us to substantially reduce dosage,” said Carter.

    “I would not have picked ALPL just on face value,” added Mihalis Kariolis, PhD, vice president of non-viral therapeutics at Voyager Therapeutics. “It highlights the power of the unbiased TRACER approach. Expanding the number of brain delivery receptors provides highly differentiated options to reduce side effects and expand the diversity of treatment modalities.”

    Both gene therapy and shuttle approaches have opportunities in different indications. Once-and-done gene therapy is not tweakable, whereas shuttle-based dosing is. “In our APOE gene therapy program, we want to reduce existing APOE4 and replace it with APOE2 permanently,” said Carter. “The shuttle has advantages in situations where permanent ongoing delivery is not required.”

    Voyager’s most advanced program (VY7523) is a tau monoclonal antibody that is exquisitely specific for pathological tau. Data will be available in the second half of the year. A gene therapy (VY1706) moving into the clinic this year is designed to knock down tau mRNA and protein intracellularly. A collaboration with Neurocrine Biosciences focuses on Friedreich’s ataxia (FA) and is also expected to enter the clinic this year.

    Combining transport receptors

    The protective BBB is crucial for maintaining homeostasis and ensuring proper neurological function. Comprised of both cellular and acellular components, this sophisticated structure tightly regulates information flow between the periphery and the brain. According to Tanya Wallace, PhD, vice president of neuroscience discovery research at AbbVie, despite the BBB’s importance, many seemingly basic biological questions remain unanswered, fueling additional global research.

    The complexity of the BBB also represents a significant bottleneck for advancing therapeutics targeting brain-related disorders. Historically, achieving therapeutically relevant levels of drugs in the brain has been a major challenge in treating serious diseases such as Alzheimer’s and Parkinson’s diseases. “A notable success story is the development of L-DOPA, a prodrug that leverages existing transport mechanisms to cross the BBB,” said Wallace. Once in the brain, L-DOPA is metabolized into dopamine, offering a key symptomatic treatment for Parkinson’s disease.

    Breakthroughs in delivery now allow scientists to leverage more technologies that can bring not only small molecules but also complex biologics into the brain. The Modular Delivery (MODELTM) platform exemplifies this progress. The platform enables engineering of bispecific antibodies, capable of targeting naturally expressed BBB receptors such as TfR and CD98. TfR and CD98 are well-characterized at the BBB, and, together, they offer distinct advantages for increasing brain exposure to therapeutics.

    “By engaging these transport pathways, the platform can enhance the uptake of a variety of therapeutics, including antibodies and oligonucleotides,” highlighted Wallace. “This multi-receptor strategy provides flexibility to optimize the balance of uptake, release, and distribution in the brain, paving the way for potentially more effective treatments across neurological disease areas.”

    This platform technology facilitated the development of ABBV-1758, which is progressing in clinical development. ABBV-1758 utilizes TfR to transport a 3pE-Ab antibody across the BBB to enable the removal of amyloid beta plaques, a pathological hallmark of Alzheimer’s disease.

    As scientists aspire to further refine delivery strategies, ongoing research is exploring additional receptors and innovative approaches, including insulin-like growth factor 1 receptor (IGF-1R) and brain cell-type-specific targeting. The field is rapidly evolving to advance more precise, personalized interventions for challenging neuroscience conditions.

    “Successful brain delivery requires more than just advances in transport technology; it demands interdisciplinary collaboration, novel preclinical models, and thoughtful clinical translation,” Wallace pointed out. Continued biological research and investment into innovative discovery platforms will be crucial for bringing transformative therapies to patients with the greatest unmet needs.

     

    References

    1. Pizzo ME, Plowey ED, Khoury N et al. Transferrin receptor-targeted anti-amyloid antibody enhances brain delivery and mitigates ARIA. Science. 2025 Aug 7;389(6760):eads3204. doi: 10.1126/science.ads3204.
    2. Muenzer J, Burton BK, Harmatz P et al. An intravenous brain-penetrant enzyme therapy for mucopolysaccharidosis II. N Engl J Med. 2026 Jan 1;394(1):39-50. doi: 10.1056/NEJMoa2508681.

    The post Breaking Through the Barrier appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Going Non-Viral: Gene Delivery Enters Its Translation Era https://edusehat.com/en/going-non-viral-gene-delivery-enters-its-translation-era https://edusehat.com/en/going-non-viral-gene-delivery-enters-its-translation-era Fri, 01 May 2026 21:20:11 +0700 Edusehat Going, Non-Viral:, Gene, Delivery, Enters, Its, Translation, Era Kunwoo Ryan Lee
    Kunwoo Ryan Lee, PhD
    CEO, BreezeBio

    Kunwoo Ryan Lee, PhD, knew as early as 2012 that solving the delivery problem would be crucial in fulfilling the promise of the newly discovered CRISPR-Cas9 gene editing technology. He felt strongly that gene editing had potential to transform medicine by curing genetic disorders, but the viral and non-viral vectors available at the time had significant drawbacks. With the support of CRISPR pioneers Jennifer Doudna and Stanley Qi, Lee completed his doctoral thesis on a gold nanoparticle delivery system for Cas9 ribonucleoprotein. He went on to co-found BreezeBio, formerly GenEdit, in 2016 with the aim of creating the next generation of gene editing-based therapeutics. To realize that goal, Lee and his team looked beyond traditional viral gene delivery systems and instead invented a new technology from the ground up.

    Most clinical gene therapy trials use viral vectors, including retroviruses, lentiviruses, adenoviruses, and adeno-associated viruses. However, viral vectors are limited in the size of the gene they can deliver. They also tend to trigger strong immune reactions and usually can’t be dosed more than once due to acquired immunity.

    Non-viral vectors are an alternative technology that offer greater gene loading capacity, more straightforward preparation, and less likelihood of triggering problematic immune reactions. BreezeBio and other biotechnology companies are reimagining gene delivery through non-viral approaches like targeted LNPs, transposons, and novel chemistry.

    BreezeBio’s laboratory
    Flow cytometry analysis of immune cells in BreezeBio’s laboratory. [BreezeBio]
    BreezeBio’s hydrophilic nanoparticle (HNP) platform, NanoGalaxy, hearkens back to Lee’s doctoral work. Lee said he and his cofounders realized that a hydrophobic molecule was needed to deliver a gene payload into cells, because the cell membrane is a lipid bilayer. Lee also noted that the best molecule for targeting different cell types is an antibody, a hydrophilic molecule. Pairing these two elements introduced a complex manufacturing challenge that the company solved by using a polyamide as a backbone structure and conjugating a hydrophobic small molecule to that backbone for targeting, resulting in the hydrophilic HNP. The company then used artificial intelligence to optimize HNPs for different tissue types.

    “Using the platform, we have demonstrated that we can deliver to the spleen, immune system, heart, and lung,” Lee said. The firm also developed a set of nanoparticles targeted to the central nervous system.

    Based on those targeted delivery profiles, the Brisbane, California-based BreezeBio has worked with multiple partners to provide delivery solutions for their products, including a multiyear collaboration with Genentech, a member of the Roche Group, signed in 2024. Meanwhile, the company is also advancing its own pipeline of therapeutics built on the NanoGalaxy platform, including a lead candidate for type 1 diabetes, as well as investigational therapies for autoimmune disease and cancer.

    Lee said a key advantage of the NanoGalaxy platform for their pipeline, which heavily leans toward autoimmune disease, is that, unlike a viral vector, the company’s studies have shown it does not activate the innate immune system. “That enabled us to use our technology for autoimmune applications and in more targeted oncology applications, as well,” Lee said.

    Snug as a bug in a rug

    The red flour beetle, a notorious scourge of grain and cereal stores, is the surprising source of Bio-Techne’s transposon-based, non-viral gene delivery system. The system, dubbed TcBuster for the beetle’s scientific name, Tribolium castaneum, was invented by B-MoGen, a spin-out of the University of Minnesota, which was acquired in 2019 by Minneapolis-based Bio-Techne. Researchers at B-MoGen and Bio-Techne developed a hyperactive version of the natural TcBuster transposon by creating a library of three million unique genetic variants and screening each in mammalian cells. In a proof-of-concept study, CAR NK cells engineered using TcBuster demonstrated in vitro functionality and improved survival in a preclinical model of Burkitt lymphoma with a single dose.1

    “The reason you want a hyperactive version is that wild-type transposon systems are fairly low activity,” said Miles Smith, PhD, a product manager for cell and gene therapy at Bio-Techne. “For generating a cell therapy, you want something that’s going to be comparable to the state of the field, and that’s lentivirus.”

    TcBuster system illustration
    The TcBuster system is electroporated into cells where its components are translated, and TcBuster transposes and excises genes of interest and inserts DNA cargo into the host cell genome. [Bio-Techne]
    Smith said the TcBuster system, which comprises an mRNA encoding transposase and a DNA transposon, can be produced faster than a lentiviral vector. The system is also more scalable, more cost-effective, and has increased gene cargo capacity, according to Smith. TcBuster can deliver multiple genes in a single vector, and it can be multiplexed with other gene therapy tools. “If you wanted to use base editors or CRISPR-based knockout gene editing in conjunction with TcBuster, you could do that in one step, compared to multiple steps with a viral system,” Smith said.

    Unlike other commercial transposon systems for gene delivery, like Sleeping Beauty and PiggyBac, Smith said TcBuster is not restricted by exclusive licensing. “The turnaround time for GMP material is just a couple of months,” Smith said. “Versus something that might take a lot longer if you have to go through licensing or create a viral batch.”

    Gene therapy SORTed

    ReCode Therapeutics is developing a pipeline of genetic medicines based on its selective organ targeting (SORT) LNP platform, which adds an additional lipid to the standard LNP formulation, allowing it to zero in on specific organs. Conventional LNPs comprise four lipids—cholesterol, a helper phospholipid, a PEGylated lipid, and an ionizable lipid—that encapsulate a therapeutic gene cassette. These traditional LNPs are primarily taken up by the liver after intravenous administration, limiting their usefulness for other organs and systems. ReCode engineered its SORT LNPs with a biochemically distinct fifth lipid that enables the body to direct the particle to the targeted organ, such as the lung or spleen, bypassing the liver, if necessary.

    “Because mRNA in a cell has a relatively short half-life, maybe a day or so, in order to have constant protein production, you need to administer it relatively frequently,” Vladimir Kharitonov, PhD, senior vice president of CMC and pharmaceutical sciences at ReCode, said. “With viral delivery, you can’t really administer it repeatedly.”

    ReCode’s laboratory in Menlo Park
    Scientists conducting qualification of the semi-automated filling and stoppering machines at ReCode’s laboratory in Menlo Park. [ReCode]
    The Menlo Park, California-based firm’s two clinical-stage therapies are given by inhalation using a nebulizer. SORT lipids enable targeting specific cell types in the lung epithelium.

    In 2024, ReCode presented preclinical data from its cystic fibrosis program showing its mRNA-based therapeutic RCT2100 significantly restored CFTR function in human bronchial epithelial cells derived from patients with cystic fibrosis. In vivo studies using a ferret model demonstrated improvement in mucociliary clearance. ReCode launched the first clinical trial of RCT2100 later that year. The LNP for RCT2100 contains SORT lipids to fine-tune its delivery to the airway epithelial cell types that have a defective or mutated CFTR protein, causing cystic fibrosis. The company is also developing a second mRNA therapy delivered via SORT LNP, RCT1100, for primary ciliary dyskinesia, which targets different cell types in the lung epithelium.

    From idea to therapy faster

    Gene delivery is just one of many services offered by GenScript to support research from discovery through clinical testing, including gene synthesis, CRISPR reagents, antibodies, and more.

    Jianpeng Wang
    Jianpeng Wang, PhD
    Senior Director, GMP Manufacturing
    GenScript

    “Gene editing is entering a new era, and the focus has shifted from discovery to translation,” said Jianpeng Wang, PhD, senior director of nucleic acid and peptide R&D at GenScript. “Our goal at GenScript is to help scientists move from idea to therapy faster.”

    When it comes to non-viral vectors, the firm offers off-the-shelf and bespoke solutions to fit the customer’s need for delivery of DNA, RNA, siRNA, peptides, and other molecules. Through its targeted LNP service, GenScript offers LNPs designed to enhance precision in directing genetic material to cells. GenScript’s ReadyEdit LNP solutions include Cas9 knock-in and knock-out, Cas12 knock-out, and base or prime editing tailored for the customer’s needs.

    “In our ecosystem, we include all of the materials needed,” Wang said. “This integration can help scientists evaluate gene editing efficiency early, both ex vivo and in vivo.”

    Wang said the choice of a vector is heavily dependent on the specific therapeutic program. “There isn’t a universally effective or better way to deliver a therapy, either viral or non-viral,” Wang said. He noted, for example, that viral vectors remain a good choice when long-term gene expression is desired. And for viral vectors, the manufacturing process might be more mature, easing transfer to a contract development and manufacturing organization.

    However, Wang cautioned that viral vectors still present certain safety concerns. “In recent years, an increasing number of scientists and the FDA have recognized these risks,” he said, “leading to a surge in interest for non-viral delivery methods—particularly for in vivo CAR T therapy and gene editing.”

    GenScript has provided LNP services to several customers globally. The most advanced of those is using GenScript’s GMP CRISPR materials (gRNA, HDR templates, and nuclease) alongside a customized LNP encapsulation recipe and is preparing an investigational new drug application.

    Foundational LNP science

    Vancouver-based Genevant traces its scientific lineage through a string of predecessor companies dating back to the early 2000s and controls foundational intellectual property for the field. Based on its scientists’ work at Protiva Biotherapeutics, the intellectual property comes to Genevant via Arbutus Biopharma, which acquired Protiva in 2015 and partnered with Roivant in 2018 to establish Genevant.

    Unlike many companies developing nucleic acid delivery platforms that focus on a single payload modality, Genevant has applied its LNP to many payloads, including mRNA, siRNA, and gene editors in fields spanning antiviral, oncology, and metabolic disorders. The firm’s LNP platform is part of the first RNA-LNP product to achieve regulatory approval, Alnylam Pharmaceuticals’ Onpattro (patisiran), a treatment for polyneuropathy in people with hereditary transthyretin-mediated amyloidosis. Genevant’s LNP technology is also behind Moderna’s COVID-19 vaccines, which were confirmed earlier this month with the resolution of a longstanding patent dispute. An infringement case against Pfizer and BioNTech is pending. Genevant collaborated with Chula Vaccine Research Center and the University of Pennsylvania to develop a COVID vaccine for low- and middle-income countries in Southeast Asia during the pandemic. The program had success, demonstrating non-inferiority to Pfizer and BioNTech’s Comirnaty in clinical trials.

    Some key differentiators for Genevant’s LNPs include strategies for optimized delivery in non-human primates instead of mice,2 which has resulted in improved gene editing in the liver, and novel chemistries for biodegradable LNPs that prevent accumulation in tissue.3 The company has recently disclosed data showing targeted delivery to T cells for in vivo CAR T therapy, as well as hematopoietic stem and progenitor cells (HSPC) and hepatic stellate cells.

     

    References

    1. Skeate JG, Pomeroy EJ, Slipek NJ, et al. Evolution of the clinical-stage hyperactive TcBuster transposase as a platform for robust non-viral production of adoptive cellular therapies. Mol Ther. 2024;32(6):1817-1834. doi:10.1016/j.ymthe.2024.04.024
    2. Lam K, Schreiner P, Leung A, et al. Optimizing lipid nanoparticles for delivery in primates. Adv Mater. Published online March 27, 2023. doi: 10.1002/adma.202211420
    3. Holland, R, Lam K, Jeng S, et al. 2024. Silicon ether ionizable lipids enable potent MRNA lipid nanoparticles with rapid tissue clearance.” ACS Nano. 2024;18 (15): 10374–87. doi:10.1021/acsnano.3c09028.

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    ]]>     Top 10 Best Selling Gene Therapies https://edusehat.com/en/top-10-best-selling-gene-therapies https://edusehat.com/en/top-10-best-selling-gene-therapies Fri, 01 May 2026 21:20:10 +0700 Edusehat Top, Best, Selling, Gene, Therapies The groundbreaking partnership that successfully treated a rare metabolic disorder in KJ Muldoon, or “Baby KJ,” with personalized CRISPR therapy last year has led therapy developers, researchers, and regulators, including the FDA, to craft a pathway for expanding the universe of gene therapies to advance the development of N-of-1 gene-editing therapies.

    In February, the FDA unveiled its Plausible Mechanism Pathway draft guidance, a series of initiatives designed to increase regulatory flexibility and spur the development of bespoke gene-editing therapies for rare and ultra-rare disorders, which collectively total about 30 million individuals in the United States.

    “The Agency anticipates that substantial evidence of effectiveness for individualized therapies could be established based on a single adequate and well-controlled clinical investigation with confirmatory evidence,” the draft guidance stated.

    Last June, at a historic roundtable of cell and gene therapy researchers and clinicians hosted by the FDA, base editing pioneer David Liu, PhD, of Harvard University and the Broad Institute of MIT and Harvard, stated: “With sufficient organization and federal support and partnership with the FDA, I believe it will be possible by 2030 to treat at least 1,000 patients with personalized genetic treatments.”

    Meanwhile, conventional gene therapy development continued in 2025. Last year saw four U.S. gene therapy approvals, bringing the number of FDA-approved gene and cell therapies up to 26, according to the American Society of Gene and Cell Therapies (ASGCT)—more than half of the 40 tallied by the organization as being approved worldwide.

    Of those 26, 18 were gene therapies, of which 10 had disclosed sales high enough to be included on this A-List, which ranks top-selling gene therapies based on sales and net product revenue figures furnished by the companies in regulatory filings, annual reports, and/or press releases. Each gene therapy is listed with its sponsor(s), type, indication, and initial FDA approval date.

    Not included are gene therapies with sales below the top 10, a category that includes two gene therapies approved in 2025: Precigen’s Papzimeos™ (zopapogene imadenovec-drba), which generated $3.4 million in net product revenue last year after becoming the first-and-only FDA-approved treatment for adults with recurrent respiratory papillomatosis (RRP) in August; and Abeona Therapeutics’ Zevaskyn® (prademagene zamikeracel), an autologous cell sheet-based gene therapy approved to treat wounds in adults and children with recessive dystrophic epidermolysis bullosa (RDEB).

    Three gene therapies did not have disclosed sales in 2025, including:

    • Encelto™ (revakinagene taroretcel-lwey), an allogeneic encapsulated cell-based gene therapy marketed by Neurotech Pharmaceuticals and indicated for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel).
    • Imlygic® (talimogene laherparepvec), a genetically modified oncolytic viral therapy marketed by BioVex (Amgen) and indicated for local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery.
    • Waskyra™ (etuvetidigene autotemcel), a cell-based gene therapy and the first FDA-approved treatment for Wiskott-Aldrich syndrome (WAS). Developer Fondazione Telethon is the first non-profit organization to have successfully led full development of an ex vivo gene therapy from lab research (at Milan’s San Raffaele Telethon Institute for Gene Therapy or SR-Tiget) to regulatory approval.

    Also not included this year are sales of three gene therapies that had been marketed by Bluebird Bio: Beta thalassemia treatment Zynteglo™ (betibeglogene autotemcel), sickle cell disease treatment Lyfgenia® (lovotibeglogene autotemcel), and cerebral adrenoleukodystrophy (CALD) treatment Skysona® (elivaldogene autotemcel).

    Last year, Bluebird Bio went private after being acquired by funds managed by Carlyle and SK Capital Partners, then rebranded in September as Genetix Biotherapeutics. Genetix does not disclose sales but did announce on March 2 that more than 100 patients received infusions of the three gene therapies during 2025.

    Also last year, Pfizer halted development and commercialization of Beqvez™ (fidanacogene elaparvovec-dzkt), which had been co-marketed with Roche-owned Spark Therapeutics, after it generated no sales in 2024. Last August, Pfizer terminated its license agreement with Spark for Beqvez, an adeno-associated virus (AAV) vector-based gene therapy indicated for forms of moderate to severe hemophilia B in adults.

     

     

     

    Top 10 Best Selling Gene Therapies
     

    1. Zolgensma®  (onasemnogene abeparvovec-xioi)

    2025 Sales: $1.232 billion 1

    Sponsor(s): Novartis2

    Type: AAV vector-based gene therapy

    Indication(s): Treatment of pediatric patients less than two years of age with spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene.

    Initial FDA Approval Date: May 24, 2019
     

    2. Elevidys®  (delandistrogene moxeparvovec-rokl)

    2025 Sales: $898.7 million

    Sponsor(s): Sarepta Therapeutics

    Type: AAV vector-based gene therapy

    Indication(s): Treatment of ambulatory pediatric patients aged four through five years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.3

    Initial FDA Approval Date: June 22, 2023 (Accelerated Approval)
     

    3. Vyjuvek®  (beremagene geperpavec-svdt)

    2025 Sales: $389.13 million

    Sponsor(s): Krystal Biotech

    Type: Herpes-simplex virus type 1 (HSV-1) vector-based gene therapy

    Indication(s): Treatment of wounds in patients six months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

    Initial FDA Approval Date: May 19, 2023
     

    4. Adstiladrin®  (nadofaragene firadenovec-vncg)

    2025 Sales: €172.673 million ($199.329 million)

    Sponsor(s): Ferring Pharmaceuticals

    Type: Non-replicating adenoviral vector-based gene therapy

    Indication(s): Treatment of adults with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

    Initial FDA Approval Date: December 16, 2022
     

    5. Casgevy®  (exagamglogene autotemcel; “exa-cel”)

    2025 Sales: $115.8 million

    Sponsor(s): Vertex Pharmaceuticals and CRISPR Therapeutics

    Type: Autologous genome-edited hematopoietic stem cell-based gene therapy

    Indication(s): Treatment of patients aged 12 years and older with sickle cell disease with recurrent vaso-occlusive crises (VOCs), or transfusion-dependent β-thalassemia (TDT).

    Initial FDA Approval Date: December 8, 2023
     

    6. Hemgenix®  (etranacogene dezaparvovec-drlb)

    2025 Sales: A$92 million ($64.9 million)4

    Sponsor(s): CSL Behring

    Type: AAV vector-based gene therapy

    Indication(s): Treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

    Initial FDA Approval Date: November 22, 2022
     

    7. Kebilidi™ / Upstaza®  (eladocagene exuparvovec-tneq)5

    2025 Sales: $56.626 million

    Sponsor(s): PTC Therapeutics

    Type: AAV vector-based gene therapy

    Indication(s): Treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.

    Initial FDA Approval Date: November 13, 2024
     

    8. Luxturna®  (voretigene neparvovec-rzyl)

    2025 Sales: CHF 41 million ($51.8 million)

    Sponsor(s): Spark Therapeutics (Roche)

    Type: Adeno-associated virus vector-based gene therapy

    Indication(s): Treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).

    Initial FDA Approval Date: December 18, 2017
     

    9. Lenmeldy™ / Libmeldy™  (atidarsagene autotemcel)6

    2025 Sales: ¥6.4 billion ($40.2 million)

    Sponsor(s): Orchard Therapeutics (a wholly owned subsidiary of Kyowa Kirin)

    Type: Autologous hematopoietic stem cell-based gene therapy

    Indication(s): Treatment for children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).

    Initial FDA Approval Date: March 18, 2024
     

    10. Roctavian®  (valoctocogene roxaparvovec-rvox; “val-rox”)

    2025 Sales: $36 million

    Sponsor(s): BioMarin Pharmaceutical

    Type: AAV vector-based gene therapy

    Indication(s): Treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to AAV serotype 5 detected by an FDA-approved test.

    Initial FDA Approval Date: June 30, 2023
    References
    1. Includes sales of ITVISMA® (onasemnogene abeparvovec-brve), approved by the FDA in November 2025 to treat SMA in adult and pediatric patients two years of age and older with a confirmed mutation in the SMN1 gene. ITVISMA has the same active ingredient as Zolgensma but is administered via a single intrathecal injection, while Zolgensma is administered intravenously.
    2. Novartis is the successor to AveXis, which successfully completed the development of Zolgensma in 2019 by receiving FDA approval for the therapy. In 2014, AveXis licensed from REGENXBIO the AAV9 vector used in the Phase I SMA clinical trial at Nationwide Children’s Hospital. REGENXBIO licensed exclusive rights to key intellectual property covering novel recombinant AAV vectors discovered at the University of Pennsylvania in the lab of James M. Wilson, MD, PhD.
    3. Following the deaths of two DMD patients receiving Elevidys last year, Sarepta halted shipments of Elevidys for non-ambulatory patients and paused the Phase III ENVISION trial (NCT05881408). The study remained paused at deadline. Following a third death, that of an eight-year-old Brazilian boy, the FDA demanded Sarepta pause shipments of Elevidys to ambulant patients. Sarepta initially refused before agreeing in July 2025. A few days later, after an FDA reversal, Sarepta resumed Elevidys shipments to ambulant patients, after Brazilian authorities ruled out treatment with the gene therapy as a factor in the boy’s death.
    4. Sales figure is for the fiscal year ending June 30, 2025. CSL Behring has since disclosed sales of $57 million ($40.2 million) for July–December 2025 but has only furnished a comparison to the year-ago period in terms of constant currency without disclosing a specific sales figure.
    5. Eladocagene exuparvovec-tneq is marketed as Kebilidi in the U.S. and as Upstaza outside the U.S.
    6. Atidarsagene autotemcel is marketed as Lenmeldy in the U.S. and as Libmeldy within the European Union.

     

     

     

    The post Top 10 Best Selling Gene Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Approaches to Reducing Toxicity and Side Effects in Cell and Gene Therapy https://edusehat.com/en/approaches-to-reducing-toxicity-and-side-effects-in-cell-and-gene-therapy https://edusehat.com/en/approaches-to-reducing-toxicity-and-side-effects-in-cell-and-gene-therapy Fri, 01 May 2026 21:20:09 +0700 Edusehat Approaches, Reducing, Toxicity, and, Side, Effects, Cell, and, Gene, Therapy Cell and gene therapy encompasses a broad range of therapeutic interventions for diseases that have proved refractory to treatment with conventional pharmaceutical approaches. Perhaps the most familiar FDA-approved modality in the cell and gene therapy field is chimeric antigen receptor (CAR) T-cell therapy, which involves genetic modification of a patient’s own T cells to identify and eliminate malignant cell lineages in acute lymphoblastic leukemia, non-Hodgkin lymphoma, and multiple myeloma.

    Although only 20 or so cell or gene therapies have been FDA-approved, the area holds considerable promise for investment. The global market was valued at nearly $9 billion in 2025, and growth has been projected at over 15% per year from 2026 to 2035. As with any pharmaceutical product, however, the potential of cell and gene therapy relies in large part upon minimizing risks to patient health from adverse effects. Numerous companies, from both prominent names in the field to smaller startups, are developing solutions to mitigate the deleterious consequences of cell and gene therapy.

    Reducing cytokine release syndrome

    Cytokines are a broad family of small proteins and peptides that cell lineages of the innate and adaptive immune systems employ to communicate with each other and coordinate timely and appropriately scaled responses to foreign antigen-containing cells. Cytokine release syndrome (CRS) occurs when hyperactivation of one or more immune lineages results in the release of excessive quantities of cytokines into the circulation.

    “As a scientific community, we’ve been researching CAR T-cell therapy for over 30 years and have grown together in our understanding of the body’s immune response to treatment, from both a safety and efficacy perspective,” says Rosanna Ricafort, MD, vice president and global program lead of hematology and cell therapy at Bristol Myers Squibb. “We have evolved our ability to characterize, stage, and manage potential side effects, allowing for timely and thoughtful interventions of the most commonly associated side effects like CRS.”

    Ricafort cited clinical data presented at the 2025 American Society for Clinical Oncology (ASCO) meeting in Chicago demonstrating that over 95% of instances of CRS and other adverse events arising from BMS’s CD19-directed CAR T-cell therapy (BreyanziR) occurred in the first two weeks after onset of therapy. “These and other studies have helped establish the largely predictable safety profile of CAR T-cell therapy to date,” Ricafort pointed out.

    Minimizing side effects

    The NF-κB and prostaglandin E2 pathways are prominent regulators of the activation and differentiation of pro-inflammatory T cell lineages. Excessive signaling through these pathways results in cytokine amplification, which contributes to CRS and immune effector cell-associated neurotoxicity syndrome (ICANS), a complication of some types of CAR T-cell therapy.

    CytoAgents, a clinical-stage biotech company, is developing CTO1681, an orally administered prostaglandin signaling inhibitor that has been shown to offset CRS and ICANS toxicities associated with CAR T-cell therapy of lymphoma patients. At the 2025 European Society for Medical Oncology (ESMO) Immuno-Oncology Congress in London, CytoAgents presented non-clinical data showing that CTO1681 treatment reduced secretion of TNF-α, IL6, and other key CRS-associated cytokines with no impairment of CAR T-cell mediated cytotoxicity on lymphoma cells.

    “These data suggest CTO1681 could enable safer CAR T-cell therapy administration, support outpatient treatment paradigms, and broaden patient access without compromising anti-tumor efficacy,” said Teresa Whalen, CEO at CytoAgents. CTO1681 is currently in Phase Ib/IIa trials for cancer patients undergoing CAR T-cell therapy, with potential expansion into additional therapeutic spaces including asthma and chronic obstructive pulmonary disease.

    Adding immunosuppressants

    A potential side effect of adeno-associated virus (AAV)-based gene transfer approaches is acute liver injury resulting in part from CRS in patients receiving AAV therapy. Duchenne muscular dystrophy (DMD) is a progressive, degenerative muscular disorder caused by mutations or changes in the DMD gene, resulting in reduced levels of the protein dystrophin.

    Adeno-associated virus
    Credit: Kateryna Con / Getty Images / Science Photo Library

    Elevidys, developed by Sarepta Therapeutics, is an AAV-based therapy approved for the treatment of DMD that stimulates targeted production of a truncated form of dystrophin in skeletal muscle. “Individuals with non-ambulatory Duchenne face profound unmet need and fewer treatment options,” says Louise Rodino-Klapac, PhD, president of R&D and development and technical operations at Sarepta. Topline data released earlier this year showed that Elevidys treatment resulted in significant improvement in key clinical ambulatory metrics in patients.

    As part of its ENDEAVOR clinical trial, Sarepta Therapeutics is evaluating the potential of supplementing Elevidys with sirolimus to reduce potential acute liver injury (ALI) complications. Sirolimus is a mammalian target of rapamycin (mTOR) kinase inhibitor that suppresses responses of T and B cells to interleukin 2, which functions to stimulate proliferation of helper, cytotoxic, and regulatory T cells.

    Developing non-integrating therapies

    As an alternative approach to supplementing cell and gene therapy modalities with existing immunosuppressants, other companies are modifying CAR T-cell therapy to reduce the risk of CRS and other side effects. Myasthenia gravis, a chronic fatigue-inducing autoimmune disorder in which signals between nerves and muscles are compromised, results in part from the secretion of autoantibodies from B-cell maturation antigen (BCMA)-expressing B plasma cells.

    Conventional BCMA-directed CAR T-cell approaches rely on the integration of lentiviral or gamma-retroviral vectors to encode the CAR and typically involve lymphodepletion chemotherapy that can be accompanied by acute and delayed toxicity. In contrast, non-integrating (i.e., mRNA-based) BCMA-directed CAR T-cell therapies may circumvent this toxicity due to the lack of requirement for chemotherapy.

    Cartesian Therapeutics is developing an mRNA-based BCMA-targeted CAR T-cell therapy for myasthenia gravis, Descartes-08. At the 2025 American Academy of Neurology (AAN) Annual Meeting in San Diego, results were reported of a Phase IIb clinical trial of Descartes-08 in myasthenia gravis. In the trial, adverse event rates were similar between groups receiving Descartes-08 and the placebo group, and were predominantly mild to moderate in nature, with no cases of CRS or ICANS reported.

    “The impressive strength and duration of response shown in the data reinforce our confidence in the potential of Descartes-08 to transform the current treatment landscape in MG, offering patients a safe, flexible, and durable treatment option,” said Carsten Brunn, PhD, president and CEO of Cartesian.

    Engineering chimeric receptors

    Modifications of CAR T-cell therapy to improve clinical efficacy and reduce side effects can also encompass modification of the molecular structure of the chimeric receptor itself. D domains are highly selective targeting domains incorporated into newer generations of CARs that enhance targeting of pathological cell types and reduce immunogenic responses in patients that give rise to unwanted side effects.

    One example of such next-generation CAR T-cell therapies, anito-cell, has been co-developed by Arcellx, Kite Pharma, and Gilead. Anito-cel is an autologous anti-BCMA CAR T-cell therapy for the treatment of relapsed/refractory multiple myeloma patients.

    Phase II trial results in multiple myeloma presented at the 2025 American Society of Hematology (ASH) meeting in Orlando showed an overall response rate of 97% and a complete response rate of 68%. Importantly, in the context of side effects, there were no delayed neurological symptoms, and for most patients, only low-grade CRS was observed, which was resolved within a few days.

    “The anito-cel D-domain BCMA binder could be important to our work in in vivo cell therapy, further strengthening our potential in oncology and inflammation,” said Daniel O’Day, chairman and CEO of Gilead. “Anito-cel could become a foundational treatment for multiple myeloma over time, including earlier lines of therapy.”

    The post Approaches to Reducing Toxicity and Side Effects in Cell and Gene Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Brain Glucose Levels Act as a Metabolic Switch for Myelin Formation https://edusehat.com/en/brain-glucose-levels-act-as-a-metabolic-switch-for-myelin-formation https://edusehat.com/en/brain-glucose-levels-act-as-a-metabolic-switch-for-myelin-formation Fri, 01 May 2026 10:35:08 +0700 Edusehat Brain, Glucose, Levels, Act, Metabolic, Switch, for, Myelin, Formation Scientists have long known that myelin doesn’t appear everywhere in the brain at once. Some regions myelinate early, others much later, and the timing shapes everything from motor development to cognitive maturation. What has remained elusive is why these regional differences emerge in the first place. A new study in Nature Neuroscience, titled “Glucose-dependent spatial and temporal modulation of oligodendrocyte progenitor cell proliferation via ACLY-regulated histone acetylation,” points to an unexpected driver: shifting glucose levels that act as a metabolic switch, telling progenitor cells when to divide and when to mature into myelin‑forming oligodendrocytes.

    The work, led by researchers at the Advanced Science Research Center at the CUNY Graduate Center (CUNY ASRC), maps glucose distribution across the developing mouse brain and reveals that these spatial and temporal fluctuations are not just metabolic background noise. They are instructive signals. “Regions with high glucose levels exhibited greater OPC proliferation and histone acetylation than regions with low glucose,” the authors wrote in the paper’s abstract, suggesting glucose as a key regulator of oligodendrocyte progenitor cell (OPC) population dynamics.

    Using MALDI imaging at the CUNY ASRC MALDI Imaging Core Facility, the team visualized glucose concentrations across brain regions during early development in mice. Areas rich in glucose contained actively dividing OPCs, while regions with lower glucose levels harbored cells beginning to differentiate into oligodendrocytes. This pattern suggested that glucose availability helps determine whether OPCs expand their numbers or transition toward myelin production.

    “Our findings show that glucose is not just fuel for the brain, it’s also a signal for the cells to divide,” said lead author Sami Sauma, PhD, a postdoctoral researcher with the CUNY ASRC Neuroscience Initiative. “When glucose levels are high in a particular brain region, progenitors use it to drive proliferation. As glucose levels shift, the same cells switch gears and begin maturing.”

    An enzyme, ATP‑citrate lyase (ACLY), which converts glucose‑derived citrate into acetyl‑CoA in the nucleus, is central to this process. This acetyl‑CoA fuels histone acetylation, activating genes required for cell proliferation. When the researchers deleted Acly in OPCs, the cells could no longer proliferate efficiently, leading to a temporary reduction in myelin due to decreased OPC numbers. Yet differentiation still occurred, thanks to a compensatory pathway: mature oligodendrocytes can generate acetyl‑CoA outside the nucleus from alternative fuels such as ketone bodies.

    This metabolic flexibility proved more than a biochemical curiosity. When mice lacking ACLY in OPCs were placed on a ketogenic diet, their myelin deficits improved. “The same cell lineage interprets different metabolic signals at distinct stages of development,” said senior author Patrizia Casaccia, MD, PhD, founding director of the CUNY ASRC Neuroscience Initiative. “By understanding how glucose and alternative energy sources regulate proliferation and myelin formation, we are uncovering new metabolic strategies that could be harnessed to protect myelin in the developing brain.”

    The developmental window examined in mice corresponds to roughly 32 to 40 weeks of human gestation—a period when premature infants are particularly vulnerable to white‑matter injury. The findings raise the possibility that metabolic support during this stage could help preserve the progenitor cells responsible for building myelin. They may also inform future approaches to repairing myelin in disorders such as multiple sclerosis.

    The post Brain Glucose Levels Act as a Metabolic Switch for Myelin Formation appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Genomics Pioneer and Life Sciences Entrepreneur J. Craig Venter Dies at 79 https://edusehat.com/en/genomics-pioneer-and-life-sciences-entrepreneur-j-craig-venter-dies-at-79 https://edusehat.com/en/genomics-pioneer-and-life-sciences-entrepreneur-j-craig-venter-dies-at-79 Fri, 01 May 2026 07:00:06 +0700 Edusehat Genomics, Pioneer, and, Life, Sciences, Entrepreneur, Craig, Venter, Dies
    j. Craig Venter, PhD [Heather Kowalski]
    j. Craig Venter, PhD [Heather Kowalski]
    J. Craig Venter, PhD, the founder, board chair, and CEO of the J. Craig Venter Institute (JCVI) has died in San Diego following a brief hospitalization for unexpected side effects that arose from the treatment of a recently diagnosed cancer, reported the JCVI in a press statement.

    Venter helped define modern genomics and launch the field of synthetic biology. He was skillful in building interdisciplinary teams, pushing for new ideas and faster methods, and insisting that discovery should translate into real-world impact. He was also a major advocate for strong federal science funding and for partnerships that accelerate progress across government, academia, and industry.

    “Craig believed that science moves forward when people are willing to think differently, move decisively, and build what doesn’t yet exist,” said Anders Dale, PhD, president of JCVI. “His leadership and vision reshaped genomics and helped ignite synthetic biology. We will honor his legacy by continuing the mission he built—advancing genomic science, championing the public investments that make discovery possible, and partnering broadly to turn knowledge into impact.”

    “Venter has been recognized as an essential force in the impetus to evolve genomics from a slow, academic discipline into a fast-moving, data-driven, and commercially relevant enterprise, leaving a lasting imprint on biotechnology, medicine, and synthetic biology,” says John Sterling, GEN’s Editor in Chief, who has known and worked editorially with Venter over the past 35 years.

    “Venter was controversial and often challenged the scientific orthodoxy, with critics accusing him of hype and going overboard on privatization. To many, he was a visionary focusing on technological acceleration and blending academic science with the zeal of an entrepreneur. Supporters saw him as a pioneer who sped up genomics by years.”

    At the NIH, he played a key role in driving gene discovery using expressed sequence tags (ESTs), enabling rapid identification of large numbers of human genes and accelerating genome mapping efforts. He went on to lead efforts that, along with the NIH, produced the first draft sequences of the human genome, a milestone that helped usher biology into the digital age. He and colleagues later published the first high-quality diploid human genome, demonstrating the importance of capturing genetic variation inherited from both parents.

    In synthetic biology, the Venter group constructed the first self-replicating bacterial cell controlled by a chemically synthesized genome—proof that genomes could be designed digitally, built from chemical components, and “booted up” to run a living cell. He also pursued scientific discovery at global scale.

    Through the Sorcerer II Global Ocean Sampling Expedition, metagenomics was used to reveal amazing microbial diversity, reporting the discovery of millions of new genes and expanding the known universe of protein families—work that deepened understanding of the ocean microbiome and its impact on planetary systems.

    Beyond his scientific achievements, and in addition to founding the JCVI, he also co-founded Synthetic Genomics, Human Longevity, and most recently Diploid Genomics, advancing efforts to translate genomics and synthetic biology into tools for the benefits of human health and environmental sustainability.

     

    The post Genomics Pioneer and Life Sciences Entrepreneur J. Craig Venter Dies at 79 appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO panel at HERS explores women’s health biotech ecosystem https://edusehat.com/en/bio-panel-at-hers-explores-womens-health-biotech-ecosystem https://edusehat.com/en/bio-panel-at-hers-explores-womens-health-biotech-ecosystem Fri, 01 May 2026 03:25:04 +0700 Edusehat BIO, panel, HERS, explores, women’s, health, biotech, ecosystem Advancing innovation in the women’s health space has long been an uphill battle. But a number of leaders in the biotech community have taken up the vanguard and are changing the game.

    Their efforts were highlighted at the inaugural Health Executive and Research Summit in San Diego, where the Biotechnology Innovation Organization (BIO) hosted the panel, Capital to Commercial: The Women’s Health BIOtech Ecosystem.

    “The mission of BIO’s Women’s Health Task Force is to ensure a robust pipeline of biopharmaceutical innovations for women’s health. As part of this mission, we are committed to elevating women’s health and that is what brings us together today,” said Michele Oshman, BIO’s Chief Patient Advocate, who also serves as the executive sponsor of BIO’s board-level task force.

    Leaders in the women’s health innovation space understand that the risky nature of biotech investment, coupled with historic stigma around the topic of women’s health, require a collaborative and creative approach. The panel reflected that approach, bringing together leaders in women’s health from across the healthcare investment spectrum.

    Going farther together

    “We often talk about the cost that it takes to bring a product to market,” said Sabrina Martucci Johnson, President and CEO at Daré Bioscience, a BIO member company specializing in women’s health. “We’re talking tens of millions of dollars.”

    Only six percent of private health care investment goes toward women’s health—though women make up half the population, says a January 2026 report from the Boston Consulting Group.

    “Of these limited funds, 90% flow into just three areas: women’s cancers, reproductive health, and maternal health, leaving other high-burden, high-prevalence conditions that impact women uniquely, differently, or disproportionally, undercapitalized,” according to the report.

    Community building is essential to mitigate this imbalance, LaToya Wilson, Managing Director, Global Co-Head of Morgan Stanley Inclusive & Sustainable Ventures told the panel.

    “Our accelerator portfolio spans a wide array of companies, including those in enterprise software and healthcare, bringing together founders with different experiences—especially around challenges like fundraising,” Wilson explained. “That’s how you advance things—by sharing knowledge, bridging gaps, and helping to move forward in a collective effort.”

    Johnson agreed. “We’ve really had to deploy a lot of creative public-private partnerships to advance our most interesting products and most first-in category—and therefore high-risk products—in the portfolio,” she said.

    These partnerships include grants and collaboration with federal agencies or major private funders, but the search for creative solutions doesn’t stop there.

    “We’ve also deployed strategic partnerships with pharma as well,” Johnson said. “For our first FDA-approved product, we partnered with Organon to bring that product to market and to commercialize it.”

    It was this partnership with Organon that allowed her firm Daré to obtain advances based on future royalties that they could then reinject back into their pipeline to fund further innovation.

    Organon, a diverse healthcare company with a focus on women, derives benefit from partnerships that drive innovative developments, said Vanessa Belozeroff, Head of R&D Portfolio and Project Management at Organon.

    “That’s one of the reasons why, last year at Organon, we piloted our accelerator program,” she said. “We don’t offer the capital component of it, but we offer the brainchild component of it.”

    Organizations applying for Organon’s accelerator are selected based upon capability and capacity matches within the organization.

    “Organizations we work with might not necessarily have built out all of their capabilities, but they might want to access them,” said Belozeroff.

    Organon is working to be a role model for how the industry connects and builds networks, “so that we can help each other out. So that we’re supporting the innovation to advance,” Belozeroff said.

    Another exciting initiative that Daré shared during the panel was their efforts to democratize investment opportunities by enabling small investors to participate and invest in Daré’s portfolio, thereby allowing women to be owners of their own healthcare solutions.

    “Typically, these are only available to super high net worth people or institutions,” she noted.

    Having the conversation

    As many in the field (and many women themselves) know, women’s health innovation can be a frustratingly taboo subject.

    Johnson recalled being banned from a podcast for saying the word vagina too many times. Yet men’s reproductive health does not face the same squeamishness.

    “If you’re developing something for the prostate, for example, you have to be able to describe it,” said Johnson. And everyone sitting at the table has to get over the giggles, as Oshman reminded, when using scientific terms to refer to women’s health.

    “We’ve gotten very comfortable with breast cancer, saying breasts, but there was a time when we didn’t say breasts when we were talking about breast cancer,” Johnson remembered.

    “Sometimes you need a therapeutic to remove the stigma,” she added. “We haven’t had a product for female sexual arousal disorder, for example. We haven’t had many vaginal drug delivery products, which is a fantastic way to deliver drugs to women. But we haven’t had the conversations and we all need to be having them.”

    In many ways, these conversations could even be baked into the normal education for new investors in the biotech space.

    When it comes to biotech investment—especially in areas where there is a greater need—investors are required to have a bit more patience due to unique hurdles to get to commercialization.

    “There may not be revenue generation for a certain amount of time,” said Wilson. “So we have to educate the investment community on the opportunities in and around this type of investing.”

    Unstoppable force

    The panelists stressed a need for diversification of approaches, ideas, and initiatives to turn the tide on women’s health investment.

    Johnson said a key challenge is showing it can be done.

    “What is the definition of insanity,” she posited. “It’s doing the same thing over and over again, expecting a different outcome, right? We have to demonstrate the returns. We have to demonstrate that we can get products across the finish line, not just to an FDA approval, but to a commercial success.”

    The post BIO panel at HERS explores women’s health biotech ecosystem appeared first on Bio.News.

    ]]>     Advanced Neural Probes Reveal Predictable Patterns in Epileptic Brain Activity https://edusehat.com/en/advanced-neural-probes-reveal-predictable-patterns-in-epileptic-brain-activity https://edusehat.com/en/advanced-neural-probes-reveal-predictable-patterns-in-epileptic-brain-activity Fri, 01 May 2026 03:20:08 +0700 Edusehat Advanced, Neural, Probes, Reveal, Predictable, Patterns, Epileptic, Brain, Activity In addition to suffering seizures, many people with epilepsy also experience bursts of abnormal brain activity called interictal epileptiform discharges (IEDs). These can happen thousands of times a day and interfere with attention, memory, language, and sleep. New data from a study led by scientists at University of California, San Francisco (UCSF) shows that these brain blips are not random events as once thought. The data shows that they unfold in a predictable pattern that can be detected before they occur, suggesting it may be possible to prevent them. 

    Details of their work are published in Nature Neuroscience in a paper titled “Laminar organization of cellular microcircuits modulating human interictal epileptiform discharges.” In it, the scientists explain that they used a high-resolution technology recently adapted for humans that records individual neuron activity to track more than 1000 neurons in four patients undergoing surgery for epilepsy. The so-called Neuropixel probes provide “a view into new ways we might address a debilitating aspect of epilepsy that we haven’t been able to tackle,” said Jon Kleen, MD, PhD, an associate professor of neurology at UCSF and co-senior author of the study. 

    Preventing brain blips would be a boon for patients’ quality of life because over time, the effects of these mental disruptions can be significant and may account for some of the cognitive impairment experienced by about half of people with epilepsy. 

    Neuropixels probes, which are thin devices lined with hundreds of sensors, are designed to record activity throughout the human cortex. This means that unlike current sensors which are limited to brain signals on the surface of the brain, Neuropixels can provide a three-dimensional view of brain activity. For the study, the scientists implanted the probes seven millimeters deep into the part of the brain where patients’ seizures originate—this is the tissue that surgeons typically remove to reduce epilepsy symptoms. 

    Inserting the probes here made it possible to observe what happened in the neurons before, during, and after each IED. While seizures appear as a burst of neurons firing in synchrony, when IEDs occur, they unfold sequentially. Specifically, one set of neurons was active about a second before the IED started followed by another set that generated the sharp electrical spike at its peak, and then a third set became active as the IED faded. “We could see individual neurons that were just microns apart from each other playing different roles in the process,” said Alex Silva, the study’s first author and a medical student and doctoral candidate in the UCSF-UC Berkeley Joint PhD program in bioengineering. “It was really striking.”

    Previous studies have demonstrated that most neurons involved in IEDs are used in normal cognitive processing. According to this study, nearly 80% of the neurons involved in IEDs were also involved in language and perception. Current implantable devices for epilepsy may be able to help. They include closed loop neurostimulators that can detect abnormal brain activity and deliver electrical pulses that interrupt it. So in the case of IEDs, devices that monitor single neurons could use the activity of the first set of neurons announcing the arrival of the abnormal pattern as a warning signal. “That would be a major step forward, changing treatment from reactively responding to abnormal brain bursts to proactively preventing them in the first place,” Kleen said.

    The post Advanced Neural Probes Reveal Predictable Patterns in Epileptic Brain Activity appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Long-Term SHIV Suppression Using AAV Gene Therapy https://edusehat.com/en/long-term-shiv-suppression-using-aav-gene-therapy https://edusehat.com/en/long-term-shiv-suppression-using-aav-gene-therapy Thu, 30 Apr 2026 23:45:12 +0700 Edusehat Long-Term, SHIV, Suppression, Using, AAV, Gene, Therapy While the overwhelming scope of tragic outcomes from HIV infection at the origin of the AIDS epidemic are in the past, those living with HIV still require daily treatments. One option includes combination antiretroviral therapy (cART) which can suppress viral replication to undetectable levels. While this therapy is effective, HIV-infected CD4+ T cells still remain in the body and inconsistent adherence to therapy schedules can result in increased viral replication to detectable levels, possibly also causing symptoms.

    Treatment with C-C chemokine receptor 5 (CCR5)-specific antibodies are one of a few alternative therapies for HIV infection, however dosing strategies and maintenance is challenging for both patients and manufacturers.

    Researchers at Oregon Health & Science University Oregon National Primate Research Center aimed to address the need for long-term expression of CCR5-specific antibodies to establish protection from HIV using adeno-associated virus (AAV) vectors.

    Their work was published in Science Translational Medicine under the title, “Adeno-associated virus gene therapy-mediated CCR5 blockade suppresses virus replication long-term in SHIV-infected macaques.”

    “We explored the ability of AAV vectors expressing the CCR5-blocking antibody leronlimab to mediate a functional cure in simian-human immunodeficiency virus (SHIV)–infected rhesus macaques by interrupting viral access to the viral entry co-receptor CCR5,” wrote the authors.

    Leronlimab is an antiviral HIV drug that targets and blocks the CCR5 receptor, thus blocking HIV’s ability to invade immune cells. Nineteen SHIV-infected macaques were treated with leronlimab expressing AAVs. All but one treated macaque produced detectable levels of leronlimab following AAV administration. The single animal that didn’t produce leronlimab had preexisting leronlimab-specific antidrug antibodies (ADA).

    About half of the animals developed an immune response to the therapy, producing ADA clearing of leronlimab, however, over a year of observation, researchers found latent increase in stable expression of the leronlimab. Macaques that did not exhibit an immune response maintained leronlimab expression throughout the same year of observation.

    Most macaques that produced sufficient number of antibodies showed long-term partial or full suppression of SHIV. “Of the nine macaques producing sufficient leronlimab to achieve full CCR5 receptor occupancy on blood CD4+ T cells, AAV-leronlimab drove stringent or partial control of SHIV viremia in six macaques long term,” wrote the authors. The three remaining macaques, when given an additional dose of leronlimab, showed either complete viral suppression or 100-fold reduction in viral load.

    The authors explain that these results indicate that there is a “threshold of leronlimab expression [that] is necessary to effectively halt SHIV replication.” They also point out that while they tested multiple capsids and promotors, they were limited in assessing vector design or dose, but surmise that the AAV-leronlimab could be combined with other AAV-delivered antivirals for a multitargeted approach.

    “These results demonstrate the potential of gene therapy–mediated long-term antibody-based CCR5 blockade for HIV functional cure but highlight challenges in achieving sufficient antibody expression when targeting an abundant self-antigen,” concluded the authors.

    The post Long-Term SHIV Suppression Using AAV Gene Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Pathogenic Bacterium Rewires Gut Environment to Colonize and Cause Disease https://edusehat.com/en/pathogenic-bacterium-rewires-gut-environment-to-colonize-and-cause-disease https://edusehat.com/en/pathogenic-bacterium-rewires-gut-environment-to-colonize-and-cause-disease Thu, 30 Apr 2026 23:45:10 +0700 Edusehat Pathogenic, Bacterium, Rewires, Gut, Environment, Colonize, and, Cause, Disease An international research team headed by scientists at Vanderbilt University Medical Center has shown how an intestinal pathogen reshapes the gut environment to fuel its own colonization and cause disease. The team’s studies found that enterotoxigenic Bacteroides fragilis (ETBF) uses a toxin it produces, Bacteroides fragilis toxin (BTF), to reprogram intestinal cell metabolism and generate conditions that support its growth. ETBF is a classically anaerobic bacterium that causes diarrhea and has been implicated in inflammatory diseases, including colitis and colorectal cancer. The study findings point to potential new therapeutic strategies for disrupting the growth of pathogens such as ETBF.

    “Our findings suggest that disease-associated microbes don’t just respond to inflammation—they can actively drive it by reshaping host metabolism,” stated Wenhan Zhu, PhD, assistant professor of pathology, microbiology and immunology. “This opens up new possibilities for intervention, such as by targeting metabolic interactions between host and microbes to prevent or disrupt diseases like infectious diarrhea and colorectal cancer.

    Zhu is lead corresponding author of the team’s published paper in Cell, titled “An anaerobic pathogen rewires host metabolism to fuel oxidative growth in the inflamed gut.” In their paper the team wrote, “Here, we demonstrate that ETBF leverages its virulence factor, BFT, to reprogram epithelial cell metabolism, thereby reshaping the gut nutritional landscape. This reprogramming leads to increased levels of lactate and oxygen, which fuel ETBF’s unique oxidative metabolism.”

    Independent studies have implicated ETBF in both inflammatory diarrheal diseases and in colorectal cancer, the authors noted. “These pathogenic effects are primarily driven by the virulence factor Bacteroides fragilis toxin (BFT), which elicits a range of physiological alterations in host cells.” However, the team noted, “… the specific mechanisms by which BFT facilitates ETBF niche establishment and promotes persistent colonization in the gut remain largely undefined.”

    Zhu has long been interested in how pathogens succeed in the competitive intestinal environment. “The gut is one of the most densely populated microbial environments in the body, with heavy competition for nutrients, yet certain microbes can still take hold and drive disease,” he said. “These microbes are ultimately competing for nutrients, and processes like inflammation and cancer may be ways they alter the environment to gain access to those resources.”

    Though the percentage of people who carry ETBF varies from study to study, it can be a common member of the gut microbiota and is considered a classical anaerobe, a type of bacteria that requires low-oxygen conditions (such as those in the large intestine) to survive. It produces a toxin, BFT, that interacts with intestinal host cells, causing inflammation and increasing oxygen and oxidative stress—conditions that are usually harmful to anaerobes such as ETBF.

    Zhu and colleagues are exploring how ETBF navigates and exploits these conditions, to gain insight into microbial physiology and host-microbe interactions, he said. Through their newly reported study the investigators found that ETBF uses its toxin, BFT, to reprogram intestinal epithelial cell metabolism.

    The researchers discovered that ETBF reshapes the intestinal landscape in unexpected ways, for example by driving epithelial cell proliferation and manipulating immune signaling pathways and bile acid biology. “BFT manipulates colonic epithelial signaling and the bile acid recycling pathway, inducing a metabolic shift in the epithelium from oxidative phosphorylation to glycolysis,” they wrote.

    This metabolic shift reduces oxygen consumption by host cells, increasing oxygen availability in the gut. The resulting environment supports the growth of ETBF, despite it being traditionally considered an anaerobe. “This shift increases local concentrations of lactate and oxygen, nutrients that support oxidative metabolism in ETBF,” they continued. These changes also create conditions that promote disease-associated microbial communities linked to colorectal cancer.

    “One of our most surprising findings was that a classically anaerobic bacterium can benefit from, and even help create, an oxygen-rich environment,” Zhu said. “This challenges the traditional view that anaerobic microbes simply cannot tolerate oxygen.”

    The team is continuing to explore how ETBF modifies its environment to successfully colonize and cause disease; how broadly the mechanisms apply across other microbes and disease settings; and whether these interactions can be therapeutically targeted. In their report the investigators stated, “… by sculpting an oxidative niche, ETBF both fuels its own growth and suppresses its microbial competitors. Importantly, this distinct metabolic program could potentially be leveraged to selectively target and remove ETBF.” Zhu added, “Ultimately, we hope to identify strategies to disrupt these disease-promoting niches before they lead to long-term pathology.”

    The post Pathogenic Bacterium Rewires Gut Environment to Colonize and Cause Disease appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Bioprocessing Applications Laboratory Opened in Korea by Ecolab Life Sciences https://edusehat.com/en/bioprocessing-applications-laboratory-opened-in-korea-by-ecolab-life-sciences https://edusehat.com/en/bioprocessing-applications-laboratory-opened-in-korea-by-ecolab-life-sciences Thu, 30 Apr 2026 20:10:09 +0700 Edusehat Bioprocessing, Applications, Laboratory, Opened, Korea, Ecolab, Life, Sciences Officials at Ecolab Life Sciences report that the company is expanding its bioprocessing business with the launch of a new bioprocessing applications lab (BPAL) in Dongtan, Korea. They say the goal is to provide biopharmaceutical manufacturers across Asia with better local access to downstream process development support.

    The site is Ecolab’s first bioprocessing facility in Asia and joins an established applications network in the U.S. and U.K.

    BPAL Korea supports process development from early-stage resin screening through studies designed to replicate commercial manufacturing conditions, according to Jenny Tan, vice president and general manager, Ecolab Life Sciences APAC and India. On-site scientists work alongside customers across Asia to help optimize chromatography steps, improve yield and productivity, and accelerate regulatory pathways, with the aim of reducing the need to ship resins and reference materials overseas for development work, she continues.

    Asia has become one of the world’s most active biopharmaceutical manufacturing regions, with Korea, China, Japan, India, and Singapore all home to growing pipelines in biosimilars and monoclonal antibody processes that scalable downstream purification. With local technical support now in place, manufacturers across the region can shorten development cycles and maintain consistency with global operations while working to tight regulatory and cost targets, continues Tan.

    “Biopharmaceutical manufacturers across Asia are under increasing pressure to scale with speed while meeting demanding regulatory and performance expectations,” she explains. “BPAL Korea strengthens our ability to work side by side with customers, bringing local expertise together with Ecolab’s global, integrated bioprocessing network.”

    By combining local scientific support with Ecolab’s innovative Purolite™ resin portfolio, Ecolab’s new BPAL was created to help enable manufacturers to address process challenges earlier, reduce development risk, and advance programs with greater confidence as they prepare for scaleup, says Tan.

    The post Bioprocessing Applications Laboratory Opened in Korea by Ecolab Life Sciences appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Restoring Vision with Stem Cell–Derived Retinal Cells by Overcoming ILM Barrier https://edusehat.com/en/restoring-vision-with-stem-cellderived-retinal-cells-by-overcoming-ilm-barrier https://edusehat.com/en/restoring-vision-with-stem-cellderived-retinal-cells-by-overcoming-ilm-barrier Thu, 30 Apr 2026 09:20:10 +0700 Edusehat Restoring, Vision, with, Stem, Cell–Derived, Retinal, Cells, Overcoming, ILM, Barrier

    Degeneration of retinal ganglion cells can cause irreversible vision loss. Pluripotent stem cells (PSCs) could, in theory, be used to replace lost ganglion cells. However, past attempts at injection of these cells have failed because the cells are not able to reach the retina.

    Now, researchers have successfully demonstrated that disrupting an eye structure long suspected of blocking the growth and survival of transplanted nerve cells—the internal limiting basement membrane (ILM)—may help restore vision in people with optic nerve damage.

    The work suggests that altering or removing the thin layer of tissue, which separates the light-sensing retinal tissue at the back of the eye from the gel-like vitreous fluid that fills the eye, was needed for the survival and migration of donor human PSC-derived retinal ganglion cells into the retina of mice, rats, and nonhuman primates. This technique could help transplanted retinal ganglion cells survive and grow in people with blinding optic nerve damage.

    This work is published in Science Translational Medicine in the paper, “The internal limiting basement membrane inhibits functional engraftment of transplanted human retinal ganglion cells in vivo.”

    Damage, or optic neuropathy, occurs when retinal ganglion cells die of disease, inflammation, or injury and stop carrying electrical signals to the brain. Common causes of damage include glaucoma, optic nerve inflammation (optic neuritis), and ischemic optic neuropathy (sudden loss of blood flow to the optic nerve).

    Healthy, functional human retinal ganglion cells can be grown in a lab, but most die when transplanted, said Thomas Vincent Johnson III, MD, PhD, a professor of ophthalmology at the Johns Hopkins Wilmer Eye Institute. “Even when the retinal ganglion cells survive, they remain on the retina’s surface and do not migrate into the tissue or form the connections with other nerve cells necessary to detect light,” he noted.

    Researchers have speculated that the internal limiting membrane, present in many vertebrates, including humans, may be causing transplant failures.

    Starting with immunosuppressed rodents, the researchers injected lab-grown human retinal ganglion cells (hRGCs) into the vitreous humors of mice with an inborn gene mutation that caused an incomplete, patchy internal limiting membrane to form. They then injected the human retinal ganglion cells into a second group of mice treated with an enzyme solution known to partially digest the membrane without damaging the eye. Lastly, they injected a third, control group of mice treated with an inactive sterile solution. After two weeks, the team observed transplantation survival in 95% of eyes (45/50) with the inborn structural defect, 80% of enzymatically disrupted eyes (32/40), and 75% of control group eyes (12/16).

    The researchers then traced where the surviving human retinal ganglion cells settled and grew in the mice, noting that a much greater percentage reached the retinal ganglion cell layer in mice born with a patchy internal limiting membrane and in those treated with the enzyme.

    Capturing 3D images of the migrated cells, the researchers say they observed that 2% (plus or minus 0.6%) and 7.1% (plus or minus 1.6%) surviving cells in enzyme-treated and mutant eyes, respectively, matured to form dendrites. In contrast, migration and maturation only occurred in 0.01% plus or minus 0.01% of surviving control human retinal ganglion cells.

    Conducting similar experiments in larger eyes and donated eye tissue replicated the group’s findings, establishing evidence that the inner limiting membrane is indeed a structural obstacle to neuron replacement, the researchers noted. They also established a surgical procedure for retinal ganglion cell transplantation that could be used in clinical trials, thus advancing potential methods for restoring vision in humans with optic neuropathy.

    While the study’s results are promising, Johnson cautions that further work is still needed before their experimental findings can be applied to people. “We know our methods are effective, but we don’t know if completely removing the internal limiting membrane helps or harms the retinal ganglion cells in the long run,” he said. “It will likely take several years before our findings could become available as an experimental therapy, but the methods we developed will guide the field moving forward.”

    The post Restoring Vision with Stem Cell–Derived Retinal Cells by Overcoming ILM Barrier appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     “Click Clotting” Technique Rapidly Creates Stronger Blood Clots https://edusehat.com/en/click-clotting-technique-rapidly-creates-stronger-blood-clots https://edusehat.com/en/click-clotting-technique-rapidly-creates-stronger-blood-clots Thu, 30 Apr 2026 05:45:09 +0700 Edusehat “Click, Clotting”, Technique, Rapidly, Creates, Stronger, Blood, Clots Researchers at McGill University have developed a rapid way to engineer blood clots that stop severe bleeding and support tissue healing more effectively. Their technique, called “click clotting,” links red blood cell surface proteins through a chemical reaction, resulting in a biocompatible clot that is 13 times more resistant to fracturing and four times more adhesive than natural blood clots. The team said the method could be used to develop life-saving biomaterials to help control severe bleeding, as well as benefit people with clotting disorders.

    “Natural blood clots can be slow to form and mechanically fragile, which limits their ability to stop severe bleeding and can compromise healing,” said Jianyu Li, PhD, senior author and professor of mechanical engineering and Canada research chair in tissue repair and regeneration. “Our work shows that, when engineered appropriately, red blood cells can play a central structural role, enabling the design of stronger and more functional biomaterials.”

    Senior and corresponding author Li, together with first author Shuaibing Jiang, PhD, reported on the development in Nature, in a paper titled “Engineering tough blood clots for rapid hemostasis and enhanced regeneration.” In their paper the team concluded, “Our strategy enables instantaneous clotting and markedly enhanced fracture resistance despite low structural polymer content, while preserving the intrinsic bioactivity of blood clots to enhance hemostasis and regeneration.”

    Jiang, now a postdoctoral associate at Harvard Medical School, led the research during his PhD studies at McGill. Researchers at the University of British Columbia, the Medical College of Wisconsin, the University of Colorado Boulder, the University of Toronto and the research institute Versiti also contributed.

    “Blood clots are pivotal for hemostasis and regeneration, but they are mechanically weak and form slowly, posing risks for life-threatening hemorrhage and limiting broader applications,” the authors wrote. “These limitations are attributed to complex coagulation cascades, abundant mechanically ineffective cells, and little structural polymers.”

    Previous efforts to crosslink red blood cells (RBCs) have used chitosan, a polymer derived from crustacean shells, but these led to brittle clots, ruptured cells, and inconsistent clotting. In “click clotting,” the clot structure is fundamentally strengthened through a fast, bio-safe chemical reaction that connects proteins on the red blood cell surface, forming a solid gel in just five seconds. Because the “click” reaction doesn’t interfere with normal blood chemistry, it can work alongside the body’s natural clotting process. As a result, the artificial cell‑based gel, called a “cytogel,” can be added to whole blood, where it becomes embedded within the body’s own fibrin clot.

    Shuaibing Jiang (left) and Jianyu Li [Jianyu Li]
    Shuaibing Jiang (left) and Jianyu Li [Jianyu Li]
    “Here we report a strategy that rapidly crosslinks red blood cells into tough cytogels and integrates them within blood clots,” the team further explained. “The resulting engineered blood clots (EBCs) form within seconds and exhibit a 13-fold increase in fracture toughness, and a 4-fold improvement in adhesion energy compared with native clots … Our strategy is advantageous over previously reported methods using chitosan to crosslink RBCs, which lead to brittle clots, hemolysis or inconsistent clotting.”

    Li added, “The technology enables both autologous clots (using the patient’s own blood) and allogeneic clots (using type-matched donor blood). Autologous clots can be prepared in approximately 20 minutes, while allogeneic clots can be prepared within about 10 minutes. Given typical clinical time constraints, this approach has strong potential for in-patient emergency care, wound management and related settings.”

    The team confirmed their results through in vitro testing, as well as through tests in rodents. “In vivo studies demonstrate that EBCs can rapidly halt hemorrhage, promote tissue regeneration, mitigate inflammation and foreign body reactions, and prevent postoperative adhesion,” the authors stated. Of particular note was effective healing and regeneration observed in the injured liver, with performance exceeding that of a clinically used product tested also tested as part of the study. “Compared with previously reported biomaterials for liver regeneration, EBC demonstrated milder inflammation and more efficient tissue regeneration,” the authors noted. Analyses showed minimal evidence of immune reactivity and no toxicity in major organs.

    The researchers say that while further study is required before the cytogel can be used in clinical settings, the research establishes a foundation for its design and application.  “Overall, EBC, as a native scaffolding material, can promote tissue regeneration with minimal inflammation and foreign body responses, and prevent postoperative adhesions, outperforming the clinically used products,” the scientists concluded. “This work may motivate the development and translation of highly cellularized materials for bleeding control, wound management, tissue repair and regenerative medicine.”

    “Engineered blood clots have strong potential for broad clinical use and could improve outcomes across many medical situations,” Li said.

    The post “Click Clotting” Technique Rapidly Creates Stronger Blood Clots appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Circio Partners with TraffikGene Project to Advance Non-Viral circVec Delivery https://edusehat.com/en/circio-partners-with-traffikgene-project-to-advance-non-viral-circvec-delivery https://edusehat.com/en/circio-partners-with-traffikgene-project-to-advance-non-viral-circvec-delivery Thu, 30 Apr 2026 02:10:20 +0700 Edusehat Circio, Partners, with, TraffikGene, Project, Advance, Non-Viral, circVec, Delivery Oslo-based Circio, which is developing circular RNA expression technology for gene and cell therapy, agreed to collaborate with Universidad de Santiago de Compostela (USC) in Spain through USC’s TraffikGene project, to explore non-viral delivery of circVec circular RNA expression vectors for next-generation gene and cell therapies.

    The collaboration brings together the circVec platform and TraffikGene’s peptide amphiphile carrier system. The combination of these complementary technologies is designed to enable high-throughput screening of circVec delivery with enhanced tissue targeting. The aim is to identify optimized formulations capable of prolonged, efficient, and targeted delivery of non-viral circVec vectors into specific cell and tissue types.

    “Combining TraffikGene’s carrier discovery capabilities with Circio’s innovative circular RNA scaffolds opens a compelling new avenue for the development of next-generation nucleic acid medicines,” said Javier Montenegro, PhD, principal investigator of the TraffikGene project at USC.

    The collaboration will involve three stages: initial screening of peptide carriers combined with non-viral circVec vectors in vitro, physicochemical optimization of lead formulations, and in vivo evaluation in mouse models to assess expression kinetics, biodistribution, and delivery efficacy.

    “Cutting edge delivery technologies are essential to reach new tissues in an efficient and safe manner,” added Thomas B Hansen, PhD, CTO of Circio. “This collaboration is an excellent opportunity to evaluate whether TraffikGene’s non-viral carriers can unlock the full potential of Circio’s circVec platform. In addition, it will allow us to evaluate circular RNA expression dynamics and tissue-specific performance in more detail, which are key research areas to identify new therapeutic applications for the circVec platform.”

     

     

     

    The post Circio Partners with TraffikGene Project to Advance Non-Viral circVec Delivery appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Loss of Smell Therapies Informed by Olfactory Receptor Spatial Mapping https://edusehat.com/en/loss-of-smell-therapies-informed-by-olfactory-receptor-spatial-mapping https://edusehat.com/en/loss-of-smell-therapies-informed-by-olfactory-receptor-spatial-mapping Thu, 30 Apr 2026 02:10:18 +0700 Edusehat Loss, Smell, Therapies, Informed, Olfactory, Receptor, Spatial, Mapping A new study published in Cell titled, “A spatial code governs olfactory receptor choice and aligns sensory maps in the nose and brain,” led by researchers from Harvard Medical School (HMS) has created the first detailed map of the spatial distribution of over 1,000 olfactory receptors in the epithelium. The study informs the development of therapies for loss of smell, where treatment options are limited.

    The researchers examined approximately 5.5 million neurons in more than 300 individual mice using single-cell sequencing and spatial transcriptomics. Results showed that neurons are organized into tight, overlapping, horizontal stripes from the top to the bottom of the nose based on the type of smell receptor expressed. This highly organized receptor map was consistent across mouse models and mirrored the organization of smell maps in the brain. Similar maps have been observed in vision, hearing, and touch.

    Notably, the olfactory map was informed by a gradient of retinoic acid in the nose, which allowed each neuron to express the correct type of smell receptor based on its spatial location.  

    “Our results bring order to a system that was previously thought to lack order, which changes conceptually how we think this works,” said Sandeep (Robert) Datta, PhD, professor of neurobiology at HMS and senior author and corresponding author of the study. “We show that development can achieve this feat of organizing a thousand different smell receptors into an incredibly precise map that’s consistent across animals.” 

    The authors also found that the receptor map in the nose matches up with smell maps in the olfactory bulb of the brain, shedding insight into how information moves from the nose to the brain. 

    While sensory maps that describe how receptors in the eye, ear, and skin are organized to capture and interpret auditory, visual, and touch information, mapping olfactory receptors has been a longstanding challenge due to high receptor diversity. As an example, mice have approximately 20 million olfactory neurons that express more than a thousand types of smell receptors, compared with only three main types of visual receptors for color vision. Each type of smell receptor detects a unique subset of odor molecules. 

    The team is also studying smell receptors in human tissue to understand to what degree the smell map is consistent across species to inform treatments, such as stem cell therapies and loss of smell and its consequences, such as an increased risk of depression. 

    “Smell has a really profound and pervasive effect on human health, so restoring it is not just for pleasure and safety but also for psychological well-being,” Datta said. “Without understanding this map, we’re doomed to fail in developing new treatments.” 

    The post Loss of Smell Therapies Informed by Olfactory Receptor Spatial Mapping appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Adaptive, Agent-Oriented Control for Biomanufacturing Systems https://edusehat.com/en/adaptive-agent-oriented-control-for-biomanufacturing-systems https://edusehat.com/en/adaptive-agent-oriented-control-for-biomanufacturing-systems Thu, 30 Apr 2026 02:10:16 +0700 Edusehat Adaptive, Agent-Oriented, Control, for, Biomanufacturing, Systems Agentic AI goes beyond predictive and generative AI and, in biomanufacturing, has the potential to enhance efficiency by integrating with existing manufacturing infrastructure such as IoT sensors, process information management systems, management execution systems, and even enterprise resource planning software. The challenge, however, is that industrial biomanufacturing processes are complex, demand resilience, and are tightly regulated.

    The Adaptive Agent-Oriented System Control (AAOSC) framework developed by a team from the Technical University of Denmark (DTU) and SiC Systems addresses that challenge through a decentralized control layer. In it, “specialized autonomous agent ‘hives’ [are] coordinating digital twin enabled manufacturing infrastructure and real-time communications protocols.” The latter lets biomanufacturers integrate models, make learning-based inferences, and control process systems.

    Four AAOSC case studies were discussed in a recent paper by Seyed Soheil Mansouri, PhD, professor at DTU and co-founder and CSO of SiC Systems and Christopher J. Savoie, PhD, co-founder and CEO of SiC Systems, and inventor of the agentic AI technology behind Siri. Those case studies “demonstrate AAOSO’s prowess [in] reducing deviating durations, averting shutdowns in severe fault scenarios, and boosting efficiency through virtual quantum and classical sensing and decentralized reasoning, all while aligning with regulatory imperatives…”

    Despite its capabilities in monitoring process, identifying discrepancies, and recommending solutions, agentic AI “is not yet fully ready for complete, independent control in biopharmaceutical manufacturing,” Mansouri tells GEN. “Any AI that directly affects medicine quality still needs strong human oversight and full approval. We are getting closer, but full integration requires official [regulatory] clearance.”

    The AAOSC framework that Mansouri and colleagues built may be unique in the industry. It isn’t all-knowing and “God-like,” he points out. Instead, “our methods are grounded in physics, chemistry, and biology within an agent ‘hive’—an orchestration of rule-based, mathematically informed agents. So, AAOSC is, foundationally, a different philosophy of building AI [in which] humans are in control.”

    First, run in shadow mode

    To introduce agentic AI, Mansouri advises starting gradually. “Run the AI alongside your current control systems in shadow mode—it watches everything and gives recommendations, but doesn’t make any actual changes without human oversight. This lets the teams learn how it works without any risks to production. Once confident, you can slowly expand its role while always keeping humans in final control.”

    Both the FDA and EMA require systems that are fixed rather than continuously learning, he points out, and that can complicate adoption. To minimize the potential for regulatory issues that may arise by integrating AI into manufacturing processes, “work closely with your quality and regulatory teams from the beginning.

    “Always maintain clear human responsibility, so no one is left wondering who is accountable if something goes wrong. Strong cybersecurity is essential,” Mansouri adds, “because these AI agents connect and talk to each other.” Therefore, “Start small, test thoroughly, and talk to regulators early.”

    The post Adaptive, Agent-Oriented Control for Biomanufacturing Systems appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Hopes Raised for More Sustainable Oligonucleotide Manufacturing https://edusehat.com/en/hopes-raised-for-more-sustainable-oligonucleotide-manufacturing https://edusehat.com/en/hopes-raised-for-more-sustainable-oligonucleotide-manufacturing Thu, 30 Apr 2026 02:10:14 +0700 Edusehat Hopes, Raised, for, More, Sustainable, Oligonucleotide, Manufacturing Large-scale manufacturing of oligonucleotides could become more environmentally friendly if the biotech industry can overcome the challenges of a promising technique for synthesizing them. That’s according to QurAlis, a clinical-stage biotech company targeting neurodegenerative disease.

    Hagen Cramer, PhD, QurAlis’s CTO, thinks synthesizing oligonucleotides using enzymes could be more sustainable than traditional solid-phase synthesis methods, but challenges remain for the industry.

    “Solid-phase synthesis is convenient—you can have everything automated, it’s fast, and can be used for [many] types of therapeutics,” he says. “However, because it’s a solid-phase synthesis, you have to wash away the external reagents with lots of solvents, and that’s why the mass intensity is high.”

    By contrast, manufacturing RNA and DNA using a process that happens in nature and is aqueous-based uses fewer materials in the production of any given mass of product, notes Cramer. However, creating a wide selection of enzymes to manufacture multiple products remains a challenge for the industry.

    “Enzymatic synthesis]was explored a long time ago, but it went away because people couldn’t figure out the challenges,” he points out. “But there’s now a lot more money in the industry as we have approved drugs and, hence, it’s now being reinvestigated.”

    Other challenges include using enzymatic techniques for manufacturing above the 100-g scale and also speeding up these techniques to be comparable with solid-phase synthesis.

    “With solid-phase synthesis, if you have a 20-mer oligonucleotide, you might have to take 80 chemical steps, and you can be efficient and complete all of that in a day, but—with an enzymatic approach—it’s going to take much longer and the development time is also large,” explains Cramer, adding that clinical-stage companies making smaller volumes may want to stick with solid-phase synthesis. But, he continues, commercial-stage companies producing large volumes of product may want to investigate enzymatic approaches as they become available.

    “At a certain stage, if you’re working at commercial stage already, you can plan ahead and I think the industry will move toward these new approaches starting post-market,” he says.

    The post Hopes Raised for More Sustainable Oligonucleotide Manufacturing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Milk Exosomes Transform Therapeutic Bioprocessing https://edusehat.com/en/milk-exosomes-transform-therapeutic-bioprocessing https://edusehat.com/en/milk-exosomes-transform-therapeutic-bioprocessing Thu, 30 Apr 2026 02:10:13 +0700 Edusehat Milk, Exosomes, Transform, Therapeutic, Bioprocessing Breast milk has long been understood as more than infant nutrition. It is a biologically active system packed with molecular signals that help shape immune development, metabolism, and even brain function. Among its most intriguing components are milk-derived extracellular vesicles—tiny lipid-bound particles often called milk exosomes—that are rapidly becoming one of bioprocessing’s most promising therapeutic tools.

    These nanoscale carriers are naturally designed for transport. They can survive digestion, move into circulation, and distribute cargo throughout the body, with studies suggesting they may even reach the brain during early development. Researchers have shown that these vesicles can influence central nervous system communication, particularly through interactions with microglia, which are crucial to the brain’s immune cells. The ability of milk exosomes to carry microRNAs and regulate epigenetic pathways, including DNA methyltransferase 1 (DNMT1), points to a sophisticated biological delivery system that the industry is now learning to harness.

    That potential is especially compelling in drug manufacturing, where delivery often determines whether a therapy succeeds or fails. Traditional nanoparticles can trigger toxicity, instability, or poor absorption. Milk exosomes offer a more elegant alternative: they are biocompatible, naturally abundant, and scalable for pharmaceutical development.

    Huiming Tu, MD, a researcher and clinician in the department of gastroenterology at the Affiliated Hospital of Jiangnan University in Wuxi, China, and his colleagues recently demonstrated this with ulcerative colitis. Their team developed an oral delivery platform called mEXOs@TOF, which loads the pan-JAK inhibitor tofacitinib into milk-derived exosomes. The resulting formulation showed strong pharmaceutical performance, including consistent particle size, high drug-loading efficiency, and strong stability during delivery.

    More importantly, the therapy improved anti-inflammatory outcomes through multiple mechanisms. It lowered inflammatory mediators such as IL-6, IFN-γ, and nitric oxide, while increasing anti-inflammatory IL-10. It also reduced oxidative stress and suppressed activation of the JAK-STAT3 signaling pathway. In both laboratory and animal studies, the system delivered strong therapeutic benefits without detectable toxicity—an ideal benchmark for translational bioprocessing.

    Cancer therapy is seeing similar innovation. Min Suk Shim, PhD, professor of nano-bioengineering at Incheon National University in the Republic of Korea, and colleagues focused on sonodynamic therapy, in which ultrasound activates a sensitizing drug to destroy tumors. Their challenge was improving intracellular delivery of chlorin e6 (Ce6), a common sonosensitizer.

    The team engineered glutathione-responsive milk exosomes by incorporating a diselenide bond-bearing fatty amine derivative. This allowed the vesicles to remain stable during circulation but release Ce6 inside breast cancer cells, where glutathione concentrations are higher. Once ultrasound was applied, reactive oxygen species production increased dramatically, leading to significant cancer cell death in MCF-7 breast cancer models. The work shows how responsive bioprocess design can turn natural vesicles into precision-triggered therapeutics.

    Meanwhile, scientists from Hong Kong and China have reviewed the broader landscape of milk exosomes in breast cancer treatment. Beyond acting as delivery vehicles for drugs like doxorubicin, paclitaxel, and 5-fluorouracil, milk exosomes may also have direct anti-tumor effects. They can promote apoptosis, interrupt the cell cycle, and regulate pathways such as NF-κB and STAT3. Combined with plant-derived compounds like curcumin and resveratrol, they form hybrid nanoparticles with enhanced therapeutic power.

    For bioprocessing, the message is clear: milk exosomes are no longer a niche curiosity. They represent a scalable, safe, and highly adaptable platform for next-generation therapeutics—one that begins with biology’s oldest delivery system and may define medicine’s next one.

    The post Milk Exosomes Transform Therapeutic Bioprocessing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Supply Chain Digital Twins: An Evolution, Not a Breakthrough https://edusehat.com/en/supply-chain-digital-twins-an-evolution-not-a-breakthrough https://edusehat.com/en/supply-chain-digital-twins-an-evolution-not-a-breakthrough Thu, 30 Apr 2026 02:10:12 +0700 Edusehat Supply, Chain, Digital, Twins:, Evolution, Not, Breakthrough Digital twins help optimize drug production processes by modeling the thousands of interactions that cells, raw materials, and reagents undergo in culture. And new analysis suggests they could do the same thing for supply chains.

    Researchers at the U.S. National Institute of Standards and Technology (NIST) and EMD Millipore put forward the idea, arguing that twins could make drug distribution, which is also characterized by thousands of interactions, more resilient and efficient.

    Lead author Perawit Charoenwut, a logistics researcher at NIST’s systems integration division, tells GEN, “A digital twin could be extremely helpful in all phases of the biopharmaceutical supply chain. Starting from demand planning triggered by global events such as pandemics, regional disease outbreaks, aging demographics, etc., through to being able to provide visibility on capacity requirements and limitations.”

    In silico models could also provide solutions to disruption by identifying alternative supply options, such as distribution centers or regional inventories, in less time, Charoenwut says.

    “Digital twins could also be helpful in evaluating different suppliers by running simulations on their potential performance, based on different demand scenarios versus their individual capacities and capabilities,” he continues.

    Standards

    In theory, digital twins are a good option for supply chain modeling and management. In practice, however, firms interested in the approach will need to overcome some technical challenges.

    For example, one major hurdle is the lack of data standardization, according to study co-author Boonserm Kulvatunyou, PhD, a computer engineer at NIST. “Supply chain digital twins require data from across organizations and third-party sources,” he tells GEN. “The lack of industry standards creates challenges in obtaining all the necessary data.”

    With this in mind, the NIST’s Industrial Ontology Foundry (IOF) is working with the National Innovation Institute for Manufacturing Biopharmaceuticals (NIIMBL) to develop open-source ontology and schema standards for connecting data.

    Kulvatunyou says, “The aim is to provide a semantic foundation for connecting data and knowledge across the manufacturing and supply chain operations.

    “Further work is being conducted to cover broader materials, processes, and quality data,” he says. “We would like to invite industry and academia to join this effort and benefit from these new standards.”

    Industry interest

    Biopharma firms interested in digital supply chains will also need to establish a solid data infrastructure, according to Charoenwut, who says companies should start small and pace themselves.

    “We think that biopharma companies do believe that digital twins could make a significant difference in their supply chain efficiency and resiliency. Many of them are probably building prototypes and proofs-of-concept to demonstrate the value and potential benefits, but then soon realize the digital data foundation gaps that need to be addressed in parallel in order to fully adopt this technology.

    “As digital twins can vary in detail and complexity, companies should strategize digital twin adoption by starting with lower-complexity cases based on available digital data and progressively moving up the scale to gain greater precision and new capabilities. In other words, the implementation of digital twins should be viewed as an evolution rather than a breakthrough,” he says.

    The post Supply Chain Digital Twins: An Evolution, Not a Breakthrough appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Hepatocyte Detargeting Improves mRNA Vaccine Immunity in Lymphoma Model https://edusehat.com/en/hepatocyte-detargeting-improves-mrna-vaccine-immunity-in-lymphoma-model https://edusehat.com/en/hepatocyte-detargeting-improves-mrna-vaccine-immunity-in-lymphoma-model Wed, 29 Apr 2026 18:55:45 +0700 Edusehat Hepatocyte, Detargeting, Improves, mRNA, Vaccine, Immunity, Lymphoma, Model mRNA vaccines work by delivering genetic instructions into cells, but a new study shows that which cells express the mRNA can alter the resulting immune response. A new study in Nature Biotechnology shows that detargeting mRNA expression away from hepatocytes strengthens T‑cell immunity in preclinical lymphoma models, revealing a new design principle for next‑generation mRNA vaccines and therapeutics.

    The work comes from researchers at the Icahn School of Medicine at Mount Sinai, who report that non‑immune cells—including muscle fibers and hepatocytes—play a decisive role in determining mRNA vaccine potency. Their paper, “mRNA vaccine immunity is enhanced by hepatocyte detargeting and not dependent on dendritic cell expression,” was published today. The findings overturn a long‑held assumption that mRNA vaccines must deliver their payload to dendritic cells to prime strong T‑cell responses.

    “This study fundamentally changes how we think mRNA vaccines work,” said senior author Brian D. Brown, PhD, director of the Icahn Genomics Institute. “For years, the field has assumed that getting the mRNA into dendritic cells, the immune cells that activate T cells, was essential. We show that’s not the case. These cells are still important, but mRNA delivery to them is not required.”

    To dissect how different cell types influence immunity, the team used a microRNA‑based technology developed in Brown’s lab that allows researchers to “turn off” mRNA expression in specific cell populations. By incorporating short microRNA target sequences into the mRNA, they selectively silenced expression in dendritic cells, hepatocytes, or muscle cells while leaving other tissues unaffected.

    The results were striking. Silencing mRNA expression in dendritic cells did not impair T‑cell priming, including for SARS‑CoV‑2 antigens, suggesting that cross‑presentation by other cell types is sufficient to initiate immunity. “This was unexpected,” said Brown. “It tells us that other cells are producing the vaccine antigen and handing it off to the immune system.” In contrast, turning off expression in muscle fibers weakened the immune response, while turning off expression in hepatocytes tripled it.

    “We found that hepatocytes actively dampen the immune response to mRNA vaccines,” said Sophia Siu, an MD/PhD student and co‑lead author. “This is notable because hepatocytes can take up a lot of mRNA, particularly when it’s injected intravenously. For vaccines, we discovered that we don’t want expression in hepatocytes. However, for mRNA therapeutics, hepatocyte expression can be beneficial because it may help prevent immunity to the mRNA-encoded protein.”

    “In mice bearing tumor-associated antigen (TAA)-expressing lymphoma cells, miRT-mediated hepatocyte-silenced TAA mRNA vaccine enhanced immune response and reduced tumor burden,” wrote the authors. The approach also reduced hepatocyte death when mRNA was used to boost pre‑existing T cells, an important consideration for gene‑editing and CAR T–related applications.

    “These results show that we can make mRNA cancer vaccines more effective simply by controlling where the mRNA‑encoded antigen is expressed,” said Joshua D. Brody, MD, director of the Lymphoma Immunotherapy Program at the Mount Sinai Tisch Cancer Center. “It’s a new lever for improving immunotherapy.”

    Beyond oncology, the findings could influence the design of mRNA‑based vaccines for infectious diseases and therapeutics for autoimmune and genetic disorders. By tuning expression in specific cell types, researchers can amplify or dampen immune responses as needed.

    “mRNA technology is transformative for medicine,” Brown said. “Our work provides a new set of design rules for mRNA vaccines and therapeutics. As this technology continues to evolve, understanding and controlling where mRNA is expressed will be critical.”

    The post Hepatocyte Detargeting Improves mRNA Vaccine Immunity in Lymphoma Model appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     ‘Type’ Casting: Flagship-Founded Serif Modifying DNA into New Therapy Class https://edusehat.com/en/type-casting-flagship-founded-serif-modifying-dna-into-new-therapy-class https://edusehat.com/en/type-casting-flagship-founded-serif-modifying-dna-into-new-therapy-class Wed, 29 Apr 2026 04:35:12 +0700 Edusehat ‘Type’, Casting:, Flagship-Founded, Serif, Modifying, DNA, into, New, Therapy, Class What happens when the scalability and redosability of messenger RNA (mRNA) is combined with the durability and programmability of gene therapy?

    According to Serif Biomedicines, a five-year-old startup that emerged from stealth mode this month, the result is “modified DNA,” a new class of therapeutics designed to be programmable, durable, scalable, and redosable—while minimizing the drawbacks of both mRNA and gene therapy.

    Modified DNA builds upon generative protein and mRNA platforms created by Flagship Pioneering, the venture capital giant which founded Serif in 2021. On April 21, Flagship formally launched Serif with an initial commitment of $50 million in financing—capital that Serif intends to use toward developing its scalable platform for optimizing and manufacturing Modified DNA treatments, aided by artificial intelligence (AI), and advancing its first drug discovery programs.

    “The reason we’re bringing the company out of stealth mode now is we think we have made progress. We’ve made real progress that we’re excited to share with the world, that we’re excited to get feedback from the broader scientific community on, and we want to tell that story more broadly,” Jacob (Jake) Rubens, PhD, Serif’s co-founder and CEO, and an Orig­i­na­tion Part­ner at Flag­ship Pio­neer­ing, told GEN.

    “It’s been on our minds for a long time: What might be possible when DNA becomes an engineerable biotechnology for the first time?”

    It’s a question pursued by numerous researchers and companies over the years as they sought to capitalize on DNA’s qualities of being a durably expressing molecule capable of coding for any gene, producing proteins or RNAs in a cell-specific way, as well as being scalable to manufacture and capable of re-dosing for patients.

    “Those are, I think, the key differentiating attributes of theoretical DNA medicines. So the question for us became not, would this be valuable if we could do it, but why hasn’t anyone done it yet?” Rubens explained. “We’ve known about the centrality of DNA in biology, the central information molecule in DNA. We’ve known this for 75 years since Watson and Crick’s seminal discoveries around how the structure of DNA enabled it to function as an information molecule.”

    Two key problems

    Jacob (Jake) Rubens, PhD, Serif Biomedicines co-founder and CEO

    “And when we looked at this space,” he continued, “we saw that there were two key problems: The first is that DNA is a highly inflammatory molecule. The second is that DNA needs to be delivered not just into a cell, but into the nucleus, the center of the cell.”

    To create Mod­i­fied DNA, Serif alters the struc­tur­al and chem­i­cal form of DNA in order to min­i­mize innate immuno­genic­i­ty as lipid nanoparticles drop off the DNA not in the nucleus, but in the cytoplasm of the cell.

    Once inside the cell nucleus, Mod­i­fied DNA reverts to unmod­i­fied DNA, enabling tran­scrip­tion into ther­a­peu­tic RNA and proteins. The resulting treatments are designed to last longer, be giv­en more than once, and be pro­grammed for cell-spe­cif­ic expres­sion. To enhance durability, Serif delivers with its Mod­i­fied DNA proteins which help the DNA access the nucleus. The proteins, called mRNA co-fac­tors, are designed to tran­sient­ly express pro­teins that enhance entry into the nucleus and gene expression.

    Pending an announcement it expects to make later this year, Serif isn’t revealing specifics of its initial drug discovery programs, except to say that they focus on rare diseases and immune programming.

    “This is not meant to be a limited list of where we could go but the areas that we think we’re going to go first, which are likely in addressing protein deficiencies in genetic diseases,” Rubens said.

    Modified DNA has shown itself to be disease agnostic, he added, reflecting DNA’s qualities as a general, programmable information molecule: “One of the reasons we’re so excited about, the future of modified DNA as a new biotechnology akin to RNA, akin to protein, is its centrality in biology. It is the fundamental information molecule inside of all of us, inside of every living thing on this planet. So that is really the existence proof that it is generalizable.”

    Tolerability and sustained expression

    Also later this year, Serif plans to present data at an as-yet-unspecified scientific conference that will show modified DNA’s tolerability in non-human primates, as well as sustained gene expression with therapeutic effects in preclinical models following intravenous (IV) administration.

    Serif aims to transform Modified DNA into treatments as effectively and commercially successfully as Amgen, Genentech (now a member of the Roche Group), and later Regeneron did with engineered proteins, as Alnylam Pharmaceuticals did with small interfering RNA (siRNA), and as Moderna more recently accomplished with mRNA—most notably in developing its SpikeVax® COVID-19 vaccine, which the FDA authorized for emergency use in 2020 and fully approved in 2022.

    Flagship launched Moderna in 2010; the company went public in 2018 by raising $604 million, the largest-ever U.S. biotech initial public offering (IPO) until Kailera Therapeutics raised $625 million earlier this month.

    At Flagship, Rubens is a sci­en­tist entre­pre­neur who leads the firm’s Pio­neer­ing Busi­ness Unit, which establishes and grows com­pa­nies based on new biotechnology. In addition to Serif, Rubens co-founded Quo­tient Ther­a­peu­tics, which develops therapies based on its somatic genomics platform; Tessera Ther­a­peu­tics, which writes therapeutic messages into the genome through a genome engineering approach called GeneWriting™; and Sana Biotech­nol­o­gy, a developer of treatments based on engineered cells. He also launched Kalei­do Bio­sciences, a microbiome therapeutics company that ceased operations in 2022.

    Before join­ing Flagship, Jake received his PhD in micro­bi­ol­o­gy from MIT, work­ing with Tim Lu, MD, PhD, a core member of the Synthetic Biology Center, through the sup­port of a Nation­al Sci­ence Foun­da­tion Grad­u­ate Research Fel­low­ship. At MIT, Jake helped enable ​“intel­li­gent” cell therapies by invent­ing gene cir­cuits that allow engi­neered cells to do nov­el ana­log, dig­i­tal, and hybrid com­pu­ta­tions.

    Based in Cambridge, MA, Serif employs about 50 people and as of Wednesday was disclosing five open positions on its website in its three areas of focus: Chemistry (associate scientist and senior scientist, both specializing in LNP formulations), Molecular Biology (research associate and senior scientist), and Research/Discovery (scientist specializing in bioanalytical assays).

    “I’m not at this point going to provide any guidance on how much more we will or won’t grow,” Rubens said. “We’re quite agile and responsive to the company’s needs.”

    The post ‘Type’ Casting: Flagship-Founded Serif Modifying DNA into New Therapy Class appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     WHO Designates Network of Regional Biomanufacturing Training Centers https://edusehat.com/en/who-designates-network-of-regional-biomanufacturing-training-centers https://edusehat.com/en/who-designates-network-of-regional-biomanufacturing-training-centers Wed, 29 Apr 2026 04:35:11 +0700 Edusehat WHO, Designates, Network, Regional, Biomanufacturing, Training, Centers The World Health Organization (WHO) has designated a network of regional training centers for biomanufacturing across all six WHO regions, which is intended to mark a major step forward in strengthening the global workforce needed to produce vaccines, biotherapeutics and other biological products.

    As global efforts continue to expand manufacturing infrastructure and advance technology transfer, WHO is placing equal emphasis on the people and systems required to make these investments sustainable and impactful.

    The designation follows a global selection process conducted through two calls for expressions of interest and forms part of the WHO Biomanufacturing Workforce Training Initiative established in 2023. This flagship effort addresses critical skills gaps across the biomanufacturing value chain, enabling countries to translate technological advances into sustainable local production.

    Yukiko Nakatani, MD, PhD [WHO]
    Yukiko Nakatani, MD, PhD [WHO]
    “Building a skilled biomanufacturing workforce is fundamental to advancing equitable access to health products and strengthening global health security. By designating regional training centers across all WHO regions, we are investing in people and systems that enable countries not only to produce quality-assured essential health technologies, but to sustain and scale them,” said Yukiko Nakatani, MD, PhD, WHO assistant director-general for health systems, access, and data. “This network reflects a strategic shift towards more resilient, geographically diversified manufacturing capacity, grounded in science and collaboration.”

    The newly designated regional training centers will operate as part of a coordinated global network, delivering context-specific training aligned with regional priorities, regulatory environments and languages, according to Nakatani. By partnering with academia and industry, they plan to expand access to training, strengthen regional expertise and foster collaboration across countries, supporting the development of a skilled and sustainable workforce. While operating independently, they will work in close collaboration with WHO under agreed frameworks to ensure quality, alignment, and accountability, note WHO officials.

    The selected institutions are:

    • African Region: Institut Pasteur de Dakar, Senegal; Council for Scientific and Industrial Research, South Africa
    • Region of the Americas: Oswaldo Cruz Foundation (Fiocruz), Brazil
    • South-East Asia Region: Translational Health Science and Technology Institute, India
    • European Region: National Institute for Bioprocessing Research and Training, Ireland
    • Eastern Mediterranean Region: Center for Continuing Professional Development, Egyptian Drug Authority, Egypt Western Pacific Region: Peking University, China

    These centers will complement the Global Training Hub for Biomanufacturing (GTH-B), established in 2022 in collaboration with the Ministry of Health and Welfare of the Republic of Korea.The Global Hub delivers standardized training programs that combine hands-on experience and classroom-based learning, while also supporting the WHO initiative through training-of-trainers programs.

    The WHO Biomanufacturing Workforce Training Initiative was designed to directly support the implementation of World Health Assembly resolution WHA74.6 on strengthening local production of medicines and other health technologies. By investing in workforce development, WHO states that it is helping to address longstanding inequities in access to health products and to ensure that all countries are better equipped to respond rapidly and effectively to future health emergencies.

    As global health systems move from crisis response to long-term resilience, building a skilled and geographically distributed biomanufacturing workforce is emerging as a cornerstone of pandemic preparedness and health security, points out a WHO spokesperson.

     

     

    The post WHO Designates Network of Regional Biomanufacturing Training Centers appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Method Identifies Cellular Makeup of Microenvironments Favoring Tumor Metastasis https://edusehat.com/en/method-identifies-cellular-makeup-of-microenvironments-favoring-tumor-metastasis https://edusehat.com/en/method-identifies-cellular-makeup-of-microenvironments-favoring-tumor-metastasis Wed, 29 Apr 2026 04:35:10 +0700 Edusehat Method, Identifies, Cellular, Makeup, Microenvironments, Favoring, Tumor, Metastasis Researchers at Baylor College of Medicine and collaborating institutions have developed a method that reveals the cellular makeup of tissues that support metastatic cancer growth, which is the primary cause of death for most patients with solid tumors. The technique, sortase A-based microenvironment niche tagging (SAMENT), is designed to selectively label cells encountered by cancer cells during metastasis. The team’s tests using SAMENT not only revealed cellular features shared by metastatic niches of multiple cancer models but also uncovered an unexpected driver of immune suppression in bone metastasis.

    “As tumors progress, cancer cells leave the original site and spread or metastasize to other organs where they seed new tumors,” said Xiang Zhang, PhD, William T. Butler, MD, Endowed Chair for Distinguished Faculty, professor of molecular and cellular biology, and director of the Lester and Sue Smith Breast Center at Baylor. “Our lab is interested in better understanding what cellular and molecular features support metastasis as these could guide the development of therapies to prevent, slow down, or eliminate them. In the current study, we first developed a new method to identify the makeup of metastatic niches.”

    Zhang, also a member of Baylor’s Dan L Duncan Comprehensive Cancer Center, is senior and corresponding author of the team’s published paper in Cell, titled “Unbiased niche labeling maps immune-excluded niche in bone metastasis.”

    During metastasis, cancer cells interact constantly with other normal cells in the body, and these interactions affect cell behavior, fate, and even response to therapies. “Numerous previous studies have elucidated the roles of specific microenvironment niches (i.e., cells that are immediately adjacent to cancer cells) in the progression of metastasis,” the authors wrote.

    For their newly reported study the team developed the SAMENT technology. “Our method allowed us to identify specific cells encountered by cancer cells during metastasis,” said co-first author Fengshuo Liu, graduate student in the Cancer and Cell Biology Program working in the Zhang lab. “The method, called Sortase A–Based Microenvironment Niche Tagging (SAMENT), selectively labels normal cells that come into direct contact with cancer cells.”

    The authors further explained, “By combining SrtA and synthetic ligand-receptor binding, we aim to label any cells that are physically encountered by cancer cells.”

    The investigators’ tests using SAMENT revealed that pro-metastatic microenvironments of multiple cancer models in all the organs studied, including bone, lung, liver, and brain, shared common features, including an abundance of macrophage immune cells and shortage or absence of immune T cells, which typically help fight tumors. “Among all cell types, macrophages occur most frequently surrounding disseminated cancer cells and appear to be phenotypically re-programmed upon interaction with metastases,” they wrote.

    Liu added, “However, bone metastases stood out. We were surprised to find that macrophages surrounding cancer cells in bone metastases activated a protein called estrogen receptor alpha (ERα). This protein is best known for its role in hormone-responsive breast cancer but is much less studied in macrophages or other immune cells.” The team added, “It also plays an important role in many other cell types, including macrophages, T cells, osteoblasts, and osteoclasts.”

    The study showed that macrophages with active ERα signaling were not detected in normal bone or in primary tumors in other tissues. ERα-active macrophages were also present in human bone metastasis samples from patients with breast, lung and kidney cancers—including male patients. This showed that this finding is not limited to one cancer type or to women.

    The researchers also investigated how cancer cells turned macrophages, which would typically fight cancer, into their allies. Cancer cells deliver small molecules called fatty acids (FAs) to macrophages, likely through tiny particles known as extracellular vesicles (EVs). These fatty acids activate a metabolic pathway in macrophages that turns on ERα signaling. “Taken together, our data indicate that ERα expression in macrophages is driven by cancer cell-derived FAs through paracrine interaction mediated by EVs,” they wrote.

    Once ERα is active, macrophages become immunosuppressive—instead of helping the immune system attack cancer, they form a barrier that physically and chemically blocks T cells from reaching tumor cells. ERα-active macrophages act as bodyguards for metastatic cancer in bone.

    “To test whether ERα in macrophages can drive bone metastasis, we genetically removed the ERα gene specifically from macrophages in mice,” Liu continued. “As a result, cancer cells were far less able to colonize bone in multiple cancer models. Tumors grew more slowly, and metastases in other organs that often arise from bone tumors were also reduced. Importantly, removing ERα from macrophages did not disrupt normal bone health—bone structure and remodeling remained intact.” In their paper the scientists stated, “Taken together, our results strongly support the hypothesis that ERα in macrophages plays an important role in bone colonization.”

    “When macrophage ERα was genetically removed or when mice were treated with fulvestrant, an FDA-approved cancer drug that degrades estrogen receptors, T cells were able to enter metastatic lesions in bone and kill tumor cells,” Zhang said. “Our findings support conducting future human clinical trials to assess the value of estrogen-blocking therapies combined with other therapies to treat bone metastases across multiple cancer types, in both women and men.”

    The authors added, “Furthermore, as shown in the final set of experiments, inhibition of ERα in macrophages may not be effective by itself but could synergize with immunotherapies because it facilitates T cell infiltration into static lesions.” The team acknowledged that they didn’t see any synergy between Erαknockout in macrophages and anti-PD1 treatment. However, they noted, “… it is still worth exploring the combinatory effects with other immunotherapies. Therefore, our findings may warrant future clinical trials on combined endocrine and immunotherapies on patients with bone metastases, and this combination may be extended to other cancer types and to patients of both genders.”

    The post Method Identifies Cellular Makeup of Microenvironments Favoring Tumor Metastasis appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Force-Sensing Mobile Microgrippers for Gentle Bioassembly of Spheroids https://edusehat.com/en/force-sensing-mobile-microgrippers-for-gentle-bioassembly-of-spheroids https://edusehat.com/en/force-sensing-mobile-microgrippers-for-gentle-bioassembly-of-spheroids Wed, 29 Apr 2026 01:05:04 +0700 Edusehat Force-Sensing, Mobile, Microgrippers, for, Gentle, Bioassembly, Spheroids

    Spheroids can be useful to model complex human tissues because they can re-create specific cell-to-cell and cell-to-matrix interactions. But spheroids are fragile, and common techniques for moving them manually—via suction—can easily damage them. In tissue engineering, the tiniest bit of improper force can harm a living culture. Now, a force-sensing miniature robot—a mobile microgripper (MMG)—has been developed that can handle spheroids with care.

    “Other techniques for cell spheroid bioassembly can affect the tissue construct and/or apply limited manipulation forces,” said David Cappelleri, PhD, professor of mechanical engineering and assistant vice president for Research Innovation School of Mechanical Engineering at Purdue University. “The force-sensing MMG presented here addresses these current issues by allowing the safe bioassembly of different spheroids into a single construct.”

    This work is published in APL Bioengineering, in a paper entitled, “Force-sensing mobile microrobotic grippers for gentle and precise bioassembly of cell spheroids.”

    Integrating different types of spheroids into one culture is key for tissue engineering. But individual spheroids have to be grown in place and then moved around, introducing the chance of damage to the spheroid.

    The MMG is a microscopic robot made of two arms connected by a hinge for a controlled—and gentle—gripping. Also, it is controlled by magnets, which are biocompatible with spheroids, decreasing the risk of collateral damage.

    “This was a big part of the design—figuring out a way to use magnetic fields for both locomotion and for controlling the opening and closing of the gripper jaws,” Cappelleri said.

    The gripping force is monitored and adjusted in real time, allowing researchers to adapt to the delicate nature of the cells. After simulating the efficacy of the MMG, in vitro testing showed that the device was able to successfully move and organize spheroids into neat patterns.

    The researchers also verified that the range of gripping forces exerted by the MMG was compatible with the movement and subsequent survival of the spheroids.

    Currently, the robot can successfully assemble the spheroids in a cellular “sheet,” but in the future, the researchers want to use their tiny robots to create full engineered tissues. In addition, the researchers want to take their microgrippers a step further, transitioning from manual control to automated spheroid assembly.

    The post Force-Sensing Mobile Microgrippers for Gentle Bioassembly of Spheroids appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Prostate Cancer Therapy Targets Disordered Region of Androgen Receptor https://edusehat.com/en/prostate-cancer-therapy-targets-disordered-region-of-androgen-receptor https://edusehat.com/en/prostate-cancer-therapy-targets-disordered-region-of-androgen-receptor Wed, 29 Apr 2026 01:05:02 +0700 Edusehat Prostate, Cancer, Therapy, Targets, Disordered, Region, Androgen, Receptor In a new study published in Nature Signal Transduction and Targeted Therapy titled, “Drugging the intrinsically disordered transactivation domain of androgen receptor,” researchers from the University of British Columbia and BC Cancer present a new approach for designing drugs that bind more strongly to intrinsically disordered proteins. These proteins play a central role in a wide range of diseases, including cancer, neurodegenerative disorders, heart disease and autoimmune conditions, and are extremely difficult to target due to their flexible nature.

    Transactivation domains (TADs) of transcription factors are enriched in intrinsically disordered regions (IDRs) that lack a stable three-dimensional structure. The plasticity of an IDR permits dynamic conformations that regulate cellular and biological functions. 

    The new study developed inhibitors that bound to the TADs of the androgen receptor, a therapeutic target for prostate cancer. While therapeutic interventions often target its folded ligand-binding domain (LBD), resistance ultimately develops due to reactivation of androgen receptor signaling. 

    Inhibitors stabilized the protein in the inactive state to prevent the activation of genes that drive cancer growth. In animal studies, several compounds slowed prostate cancer growth more effectively than a commonly used prostate cancer treatment. Notably, several antigen receptor TAD inhibitors displayed strong binding affinities higher than, or were comparable to the LBD-inhibitor enzalutamide, with dissociation constants in the picomolar to low-nanomolar range 

    “Most drug discovery is like designing a key for a very specific lock,” said Marianne Sadar, PhD, professor of pathology and laboratory medicine at the UBC faculty of medicine, distinguished scientist at BC Cancer, and co-corresponding author of the study. “But disordered proteins don’t behave like locks at all, they’re more like moving strands of spaghetti.”   

    “This study shows that proteins previously thought to be undruggable can be drugged with remarkable efficacy,” she continued. “The findings could have profound implications for the treatment of cancer and other diseases, providing a roadmap for the development of new treatments.”   

    “What surprised us was how effectively these molecules could attach to a protein that doesn’t have a fixed structure,” said Raymond Andersen, PhD, professor in UBC’s department of chemistry and co-corresponding author of the study. “We were able to shut down the androgen receptor even in situations where current prostate cancer drugs stop working.”   

    The researchers now aim to advance the most promising candidates toward clinical trials, with the goal of developing prostate cancer drugs for early intervention and with fewer side-effects.  

    “If the approach continues to prove successful, it could dramatically expand the number of proteins that scientists can target with medicines—turning what was once considered a dead end into a promising new frontier for drug discovery,” said Sadar. 

    The post Prostate Cancer Therapy Targets Disordered Region of Androgen Receptor appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Retron-Powered Approach Enables Genome Editing Across Diverse Bacterial Species https://edusehat.com/en/retron-powered-approach-enables-genome-editing-across-diverse-bacterial-species https://edusehat.com/en/retron-powered-approach-enables-genome-editing-across-diverse-bacterial-species Tue, 28 Apr 2026 09:00:10 +0700 Edusehat Retron-Powered, Approach, Enables, Genome, Editing, Across, Diverse, Bacterial, Species For decades, the ability to precisely rewrite bacterial genomes has been largely confined to a single workhorse organism: Escherichia coli. That limitation has slowed efforts to study pathogens, engineer sustainable biomanufacturing strains, and probe how microbes influence human health. While genome editing tools have transformed eukaryotic biology, most high‑efficiency bacterial editors simply haven’t worked outside E. coli.

    A new study from the Gladstone Institutes aims to change that. In a large, nine‑lab collaboration, researchers have translated a retron‑based DNA editing system from E. coli into 14 additional bacterial species spanning three major phyla. The work, published in Nature Biotechnology and titled “Genome editing of phylogenetically distinct bacteria using cross-species retron-mediated recombineering,” demonstrates that retrons, bacterial immune elements that continuously produce short DNA strands, can be engineered into portable genome editing modules the authors call recombitrons. “Recombitrons—a genome editing tool created by pairing modified, donor-producing bacterial retrons with single-stranded binding and annealing proteins—have increased the efficiency of recombineering to install flexible, precise edits in the prokaryotic chromosome,” the authors wrote.

    Retrons normally function as part of a viral defense system, generating DNA fragments that help bacteria detect and respond to infection. Seth Shipman, PhD, a Gladstone Investigator and senior author of the study, has spent years repurposing this machinery. “We’ve been easily editing E. coli genomes using retrons for years now, which has substantially increased the pace of our fundamental biology and our molecular technology development,” he said. “But we kept hearing from the broader field, asking when there would be a version of this technology that could be put to work in other bacterial species that matter for the environment, industrial processes, or human health.”

    Shipman’s lab previously showed that retrons can act as cellular DNA-making factories, generating the donor strands needed for genome editing. In bacteria, the resulting editing tool built by pairing modified retrons with single‑stranded DNA–binding and annealing proteins is known as a recombitron. Until now, however, functional recombitrons existed only in E. coli.

    To test whether the architecture could travel, the team designed a panel of 10 retron-based editing systems and partnered with other labs specializing in diverse bacterial species. “We designed all the molecular parts at Gladstone, then sent them to the collaborators, where they ran the experiment in their labs,” said first author Alejandro González‑Delgado, PhD. Samples were then returned to Gladstone for centralized analysis.

    The results show broad functionality. The recombitrons worked in all 15 species tested, including clinically relevant pathogens such as Klebsiella pneumoniae and Pseudomonas aeruginosa, as well as fast‑growing biotechnology strains like Vibrio natriegens and Pseudomonas putida. Editing efficiencies varied widely—from fractions of a percent to more than 90%—but the team demonstrated that modifying retron structure or other system components could boost performance in lower‑efficiency hosts.

    “Each retron worked differently in different bacteria,” González‑Delgado noted. “This reinforces why it’s important to have lots of different retrons, so scientists can choose the ones best suited to their favorite bacterial species.”

    The study provides a roadmap for expanding genome editing into species that have historically been difficult to engineer. Researchers studying microbial pathogenesis, gut ecology, or industrial bioproduction can now match retron systems to their organism of interest.

    “My lab builds molecular technology, and we want these technologies to be used as broadly as possible to uncover new biology and intervene in disease,” Shipman said. “We hope it will continue to spread from here.”

    The post Retron-Powered Approach Enables Genome Editing Across Diverse Bacterial Species appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Pencil Beam Laser Could Help Researchers Design Brain-Targeted Therapies https://edusehat.com/en/pencil-beam-laser-could-help-researchers-design-brain-targeted-therapies https://edusehat.com/en/pencil-beam-laser-could-help-researchers-design-brain-targeted-therapies Tue, 28 Apr 2026 05:25:10 +0700 Edusehat Pencil, Beam, Laser, Could, Help, Researchers, Design, Brain-Targeted, Therapies Scientists at MIT say they made a finding in optical physics that could enable a new bioimaging method that’s faster and higher-resolution than existing technology. They discovered that, under the right conditions, laser light clutter can spontaneously self-organize into a highly focused “pencil beam.”

    Using this self-organized pencil beam, the team captured 3D images of the human blood-brain barrier 25 times faster than the gold-standard method, while maintaining comparable resolution, according to the scientists.

    By showing individual cells absorbing drugs in real-time, this technology could help scientists test whether new drugs for neurodegenerative disease like Alzheimer’s or ALS reach their targets in the brain, with greater speed and resolution, they add.

    “The common belief in the field is that if you crank up the power in this type of laser, the light will inevitably become chaotic. But we proved that this is not the case. We followed the evidence, embraced the uncertainty, and found a way to let the light organize itself into a novel solution for bioimaging,” says Sixian You, PhD, assistant professor in the MIT department of electrical engineering and computer science (EECS), a member of the research laboratory for electronics.

    You is senior author of a paper “Self-localized ultrafast pencil beam for volumetric multiphoton imaging” on this imaging technique in Nature Medicine.

    A better beam

    When the researchers performed characterization experiments of this pencil beam, it was more stable and high-resolution than many similar beams. Other beams often suffer from “sidelobes,”  blurry halos of light that can distort images.

    Their beam was more pristine and tightly focused, according to You. Building on those experiments, the researchers demonstrated the use of this pencil-beam in biomedical imaging of the human blood-brain barrier.

    Scientists and clinicians often want to see how drugs flow inside the vasculature of the blood-brain barrier and whether they reach their targets within the brain. But with standard optical settings, the best one can do is capture one 2D section of the vasculature at a time, and then repeat the process multiple times to generate a fuller image, You explains.

    Using this new technique, the researchers created an ultrafast, high-precision pencil beam that enabled them to dynamically track how cells absorb proteins in real-time.

    “The pharmaceutical industry is especially interested in using human-based models to screen for drugs that effectively cross the barrier, as animal models often fail to predict what happens in humans. That this new method doesn’t require the cells to have a fluorescent tag is a game-changer,” notes Roger Kamm, PhD, the Cecil and Ida Green Distinguished Professor of Biological Science and Mechanical Engineering.

    “For the first time, we can now visualize the time-dependent entry of drugs into the brain and even identify the rate at which specific cell types internalize the drug.”

    “Importantly, however, this approach is not limited to the blood-brain barrier but enables time-resolved tracking of diverse compounds and molecular targets across engineered tissue models, providing a powerful tool for biological engineering,” points out postdoctoral fellow Sarah Spitz, PhD.

    The team reports that it captured cellular-level 3D images that were higher quality than with other methods, and generated these images about 25 times faster.

    “Usually, you have a tradeoff between image resolution and depth of focus—you can only probe so far at a time. But with our method, we can overcome this tradeoff by creating a pencil-beam with both high resolution and a large depth of focus,” You says.

    In the future, the researchers want to better understand the fundamental physics of the pencil-beam and the mechanisms behind its self-organization. They also plan to apply the technique to other scenarios, such as imaging neurons in the brain, and work toward commercializing the technology.

    The post Pencil Beam Laser Could Help Researchers Design Brain-Targeted Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Sun Pharma Aims for Top 3 in Women’s Health with $11.75B Organon Purchase https://edusehat.com/en/sun-pharma-aims-for-top-3-in-womens-health-with-1175b-organon-purchase https://edusehat.com/en/sun-pharma-aims-for-top-3-in-womens-health-with-1175b-organon-purchase Tue, 28 Apr 2026 05:25:09 +0700 Edusehat Sun, Pharma, Aims, for, Top, Women’s, Health, with, 11.75B, Organon, Purchase Sun Pharmaceutical Industries has agreed to acquire Organon, the women’s health drug developer spun out of Merck & Co., for $11.75 billion in a deal intended to catapult the buyer into a top 25 global biopharma—top three in women’s health—by growing its innovative medicines business and expanding its product offerings into biosimilar drugs, the companies said today.

    Headquartered in Jersey City, NJ, Organon was spun out of Merck in 2021 and has since then grown its portfolio to more than 70 women’s health and general medicines products, including biosimilars, that have been commercialized in the U.S. and some 140 countries worldwide. In addition to the U.S., Organon’s largest markets include Brazil, Canada, China, and the countries of the European Union. Organon said it has six manufacturing facilities across the EU and emerging markets.

    Sun Pharma said the combined company created by the deal will have annual revenue of $12.4 billion, a figure the company said would propel it into a top 25 global pharma—though the company was ranked No. 14 in GEN’s most recent A-List of Top 25 Biotech Companies Heading Into 2026, compiled last December, based on its market capitalization (share price times the number of outstanding shares) of INR 4.31 trillion ($50.8 billion).

    Sun Pharma said Organon’s portfolio was similar to its own, and that the acquisition of Organon was aligned with its strategies of growing its Innovative Medicines business (to a 27% revenue share) and expanding into biosimilars as a Top 10 global company.

    The combined company, Sun Pharma and Organon said, would be top three in global women’s health, creating a commercial platform for future growth; the seventh largest global biosimilar player; and a presence in 150 countries worldwide, with 18 large markets that would each generate more than $100 million in revenues.

    “This transaction represents a significant opportunity for Sun Pharma to build on its vision of Reaching People and Touching Lives,” Sun Pharma executive chairman Dilip Shanghvi said in a statement. “Organon’s portfolio, capabilities, and global reach are highly complementary to our own, and we believe that bringing the two organizations together can create a stronger and more diversified platform. We have deep respect for Organon’s mission and look forward to building on its legacy while driving sustainable long‑term growth.”

    Deal speculation

    The deal ends two weeks of speculation that began with an April 10 report in the Indian news outlet The Economic Times stating that Sun Pharma had submitted a $12 billion all-cash offer for Organon. On Friday, the news outlet followed up with a report stating that Sun Pharma had submitted a revised $13 billion offer.

    Investors appeared to support the deal, as Sun Pharma shares on India’s National Stock Exchange rose about 7% to INR 1,733.50 ($18.41) at the close of trading today.

    Sun Pharma has agreed to acquire 100% of Organon’s issued and outstanding shares for cash. Sun said it planned to fund the acquisition through a combination of available cash resources and committed financing from banks.

    “Together, we will become a partner of choice for acquiring and launching new products,” stated Kirti Ganorkar, managing director of Sun Pharma. “Our immediate priorities will be business continuity, disciplined integration, and responsible value creation. We see strong potential in leveraging Organon’s talent pool. In addition, there is a scope for synergies including significant revenue upside opportunities to be realized over the coming years.”

    Those synergies were later quantified by Sun Pharma as approximately $350 million within two to four years of the deal’s completion.

    Sun Pharma did say, however, that the acquisition of Organon will strengthen its generation of cash, with its earnings before interest, taxes, depreciation, and amortization (EBITDA) and cash flow set to nearly double, supporting future efforts to reduce the net debt/EBITDA of 2.3x resulting from the deal.

    Sun Pharma finished the first nine months of its fiscal year ending March 31, 2026, with a net profit of INR 87.654 billion ($931.5 million) and EBITDA of INR 137.772 billion ($1.464 billion; up 19.2% from the year-ago period), on sales of INR 436.604 billion ($4.64 billion), up 11.3% year over year.

    During its fiscal year ending March 31, 2025, Sun Pharma reported adjusted net profit (excluding one-time items) of INR 119.844 billion ($1.274 billion), up 19% from a year earlier, on sales of INR 520.412 billion (about $5.53 billion). Reported net profit for FY 2025 was INR 109.290 billion ($1.161 billion), vs. Rs. 95.764 billion ($1.017 billion) during FY 2024.

    Organon finished last year with adjusted EBITDA of $1.9 billion on revenue of $6.2 billion. The company reported debt of $8.64 billion—down from the $9.5 billion in debt it reported when it separated from Merck—and a cash balance of $574 million.

    Planned sale

    In November, Organon announced plans to sell its JADA® System, designed to control and treat abnormal postpartum uterine bleeding or hemorrhage, to Laborie Medical Technologies for up to $465 million—$440 million to be paid at closing, subject to adjustments, and up to $25 million tied to achieving 2026 revenue targets. Net proceeds from the divestiture will contribute to Organon’s cash balance as of March 31, 2026.

    Organon will merge with a subsidiary of Sun Pharma, with Organon surviving the merger. The transaction is expected to close in early 2027 subject to customary conditions, including regulatory approvals and Organon stockholder approval.

    The boards of both Sun Pharma and Organon have approved the deal.

    “Following a comprehensive review of strategic alternatives, our Board determined that this all‑cash transaction offers compelling and immediate value to Organon stockholders,” stated Carrie Cox, executive chair of Organon. “We believe Sun Pharma is well positioned to support Organon’s businesses, employees, and patients globally, and to further advance our commitment to delivering impactful medicines and solutions.”

    The post Sun Pharma Aims for Top 3 in Women’s Health with $11.75B Organon Purchase appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Genetic Medicine Delivery Enhanced by Producer Cell Modifications https://edusehat.com/en/genetic-medicine-delivery-enhanced-by-producer-cell-modifications https://edusehat.com/en/genetic-medicine-delivery-enhanced-by-producer-cell-modifications Tue, 28 Apr 2026 01:55:06 +0700 Edusehat Genetic, Medicine, Delivery, Enhanced, Producer, Cell, Modifications Gene editing has emerged as a powerful approach for targeting the genetic causes of disease, yet delivering the editing machinery into the correct cells efficiently, safely, and at the scale needed for therapies remains one of the biggest bottlenecks.  

    Among the leading delivery vehicles are engineered virus-like particles (eVLPs), which can enter human cells similar to viruses but carry no viral genes. Instead, these delivery vehicles carry gene editing tools for therapeutic applications.  

    In a new study published in Nature Communications titled, “Genome-wide screening reveals producer-cell modifications that improve virus-like particle production and delivery potency,” researchers from Whitehead Institute have developed a platform that systemically identifies which genes drive or block particle assembly to engineer cells that yield more potent delivery vehicles. 

    “We can engineer the particles as much as we want, but if we don’t understand how the producer cells are actually making the particles, we’re limited in how much we can improve production,” said Aditya Raguram, PhD,  Valhalla Fellow at Whitehead Institute and corresponding author of the study.

    As virus-like particles are assembled inside cultured human cells, the authors ran a genome-wide search to identify which genes are crucial in the production process by generating a large pool of producer cells in which nearly every gene in the human genome was switched off in the population. This approach generates eVLPs loaded with guide RNAs that identify the genetic perturbation in the cell that produced a particular particle. The team could then identify which gene shutdowns enabled and disabled particle production. 

    “One thing that surprised me was how clearly the search was able to highlight specific pathways that play a major role in the production of these particles,” said Diana Ly, research technician at Whitehead Institute and first author of the study. 

    The single gene whose removal most boosted production normally reduces the cell’s output of guide RNAs. Disabling this gene enabled cells to generate more guide RNA and particles to carry more functional cargo. 

    The improvement also extended across different gene editing tools and particle designs. The team tested the modified producer cells with diverse gene editors and four other delivery-vehicle systems from external labs, and produced improved particles. 

    “Because guide RNA loading is basically universal across different cargo types and particle types, this improvement could be quite broadly useful beyond the particles we’ve developed,” Raguram says. 

    Looking ahead, the authors are extending the screening platform to expand beyond switching off one gene at a time to examine how other cellular changes influence particle production. The team is sharing its engineered cell lines with the research community to improve the delivery of gene editing tools into immune cells, neurons, and other cell types important for treating disease. 

    For Raguram, the work speaks to a broader task facing the gene editing field. 

    “This delivery challenge is one of the last remaining bottlenecks that really limits the widespread application of gene editing technologies,” he says. “Solving the challenges associated with production could move virus-like particles closer to being ready for use in patients.” 

    The post Genetic Medicine Delivery Enhanced by Producer Cell Modifications appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Monoclonal Antibodies: Unlocking Cost and Efficiency Gains in Downstream Processing https://edusehat.com/en/monoclonal-antibodies-unlocking-cost-and-efficiency-gains-in-downstream-processing https://edusehat.com/en/monoclonal-antibodies-unlocking-cost-and-efficiency-gains-in-downstream-processing Tue, 28 Apr 2026 01:55:05 +0700 Edusehat Monoclonal, Antibodies:, Unlocking, Cost, and, Efficiency, Gains, Downstream, Processing

    Image of Kristina Pleitt

    Kristina Pleitt

    Senior Staff Scientist
    Thermo Fisher Scientific

    Panelist

    Image of Kristina Pleitt

    Kristina Pleitt

    Kristina Pleitt is a senior staff scientist at Thermo Fisher Scientific with 15 years of experience in biopharmaceutical process development and manufacturing. Her expertise includes downstream process development, process scale-up, integrated continuous processing, and clinical and commercial production, with a particular focus on advancing intensified downstream strategies that improve process efficiency, performance, and manufacturability.

    Broadcast Date: 
    • Time: 

    Analytical developers and downstream scientists are under constant pressure to improve process efficiency while maintaining product quality for monoclonal antibodies (mAbs). Yet, many platform purification processes still leave untapped opportunities for optimization, cost reduction, and simplification.

    In this webinar, our speaker will explore a series of practical, chromatography-focused strategies designed to enhance mAb purification performance while reducing cost of goods (COGs). Using real-world examples and process scenarios, attendees will gain insight into:

    • Identifying opportunities to simplify traditional mAb purification workflows

    • Improving Protein A performance and lifetime to drive cost savings

    • Enabling efficient two-step downstream processes through better impurity management

    • Addressing difficult HCP and aggregate clearance challenges with advanced polishing strategies

    A live Q&A session will follow the presentation, offering you a chance to engage directly with our expert and discuss how these approaches can be applied to your own processes.

    Produced with support from:

    Thermo Fisher logo

    The post Monoclonal Antibodies: Unlocking Cost and Efficiency Gains in Downstream Processing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Anticancer Strategy Targets Defense Mechanism in Senescent Cells https://edusehat.com/en/anticancer-strategy-targets-defense-mechanism-in-senescent-cells https://edusehat.com/en/anticancer-strategy-targets-defense-mechanism-in-senescent-cells Tue, 28 Apr 2026 01:55:04 +0700 Edusehat Anticancer, Strategy, Targets, Defense, Mechanism, Senescent, Cells Scientists headed by a team at MRC Laboratory of Medical Sciences (LMS) and Imperial College London have found that a new set of drugs can exploit a recently-revealed weakness in senescent—or ‘zombie-like’—cells, a finding that could lead to new treatments for cancer and age-associated diseases.

    Senescent cells walk a tightrope, risking cell death with high levels of iron and other damaging agents, but compensating for this by overproducing a protective protein, GPX4, which staves off death. The team showed that targeting this defense mechanism removes the shield and could be used to treat diseases that are associated with senescence, including cancer. Tests showed that combining anticancer therapies with GPX4 inhibitors eliminated senescent tumor cells in models of melanoma, prostate and ovarian cancer. This approach, they say, could complement existing treatments to bring much-needed improvements for cancer patients.

    Mariantonietta D’Ambrosio, PhD, a postdoctoral researcher at the LMS, is first author of the international research team’s published paper in nature cell biology, titled “Electrophilic compound screening identifies GPX4-dependent ferroptosis as a senescence vulnerability.”

    Cancers grow as a result of unconstrained cell division. But within most tumors, there is a portion that does not divide at all: senescent cells. Chemotherapy often increases the proportion of senescent cells in a tumor as it aims to stem the rapid proliferation, the team explained. However, while these senescent cells don’t directly increase the size of a tumor, they can wreak havoc in their own way.

    Senescent cells, which are also a defining feature of aging conditions such as fibrosis, influence neighboring cells by secreting molecules that increase proliferation, the spread of the cancer, and unwanted immune system activity. “Senescent cells drive aging and age-related pathologies, including cancer,” the team wrote. There is therefore an increasing interest in developing drugs that directly target and kill senescent cells, in cancer and beyond. “Consequently, senolytics, drugs that selectively kill senescent cells, have broad therapeutic appeal,” they continued. “Compounds that selectively kill senescent cells (senolytics) can treat different age-related pathologies.”

    The study by D’Ambrosio and colleagues has identified a new approach to killing senescent cells in cancer.  “Senescence was considered for a long time to be positive, because senescent cells don’t proliferate, which is the core feature of cancer,” D’Ambrosio explained. “Normal chemotherapy induces senescence blocking the proliferation of cancer cells, so the tumor doesn’t get bigger. But with time you also see the negative side of the senescent cells, because they secrete a lot of factors that influence neighboring cells and induce even more proliferation, metastasis, and recruitment of bad parts of the immune system that will provoke even more aggressiveness in the tumor.  For this reason, we tried to find some drugs that were able to kill the senescent cells.”

    The researchers cast a broad net in their search for new drugs that might kill senescent cells. Together with collaborators at the Department of Medicinal Chemistry at Imperial, they decided to examine covalent compounds, a class of inhibitors that can form a covalent bond with their target, which can result in the inhibition of proteins previously considered undruggable. The investigators introduced 10,000 different covalent compounds to both senescent cells and normal cells, looking for the ones that preferentially killed senescent cells and classing the drug as “senolytic,” or senescent-killing.

    They narrowed their results down to just four promising compounds and found that three of them affected a particular protein, GPX4, which has a protective role in cells, helping stave off ferroptosis, a type of cell death associated with high levels of iron and destructive reactive oxygen species. To protect themselves against the high levels of iron and other ferroptosis-causing agents, senescent cells have high levels of GPX4. It is like proactively taking a painkiller so a person can keep running on an ankle. The damage and danger remains, but the immediate risks are bypassed. Removing the painkiller makes the pain unbearable.

    “Senescent cells are primed for ferroptosis and upregulate GPX4 as a protective mechanism,” the team noted. Ferroptosis had only recently been revealed as a potential weakness of senescent cells. D’Ambrosio commented, “recent papers have shown this predisposition of senescent cells to ferroptosis, but it’s a new senescence vulnerability. That creates an opportunity for us to exploit. So now there is research to find senolytic drugs to kill cells through ferroptosis.”

    The researchers found that blocking the activity of GPX4 removes the shield, making fatal ferroptosis unavoidable. The authors further commented, “We concentrated our studies on four chloroacetamides displaying senolytic activity in different models of senescence … GPX4 was a target of three of the four senolytic chloroacetamides. GPX4 is a glutathione peroxidase that prevents ferroptosis by reducing lipid peroxidation.”

    The team tested their drugs with three different mouse models of cancer and saw improved outcomes as a result of senescent cell death in each case. Translating this to patients could be a huge asset to cancer treatments. “In mouse models we saw that these drugs reduced tumor size, and improved survival,” noted professor Jesus Gil, PhD, senior author and head of the senescence group at the LMS. “Now we need to see the effect on the immune system. Is the improvement also awakening the ‘good side’ of the immune system (T cells, natural killer cells) that helps to kill the tumor? … Once we know more, the next step is to understand which cancer cell types or specific patients might better respond to this treatment. For example, if a patient undergoing chemotherapy overexpressed GPX4 then you could use this approach in combination with existing drugs to improve efficacy.”

    This approach offers a much-needed new perspective on cancer therapy, pinpointing senescent cells as an underexploited target. D’Ambrosio says it has potential to transform treatment. “Targeting senescence is a huge opportunity for cancer treatments, and ultimately it can play a supporting role in addition to chemotherapy and immunotherapy.”

    The post Anticancer Strategy Targets Defense Mechanism in Senescent Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Abdominal Contractions May Drive Brain Fluid Flow, Aiding in Neural Waste Clearance https://edusehat.com/en/abdominal-contractions-may-drive-brain-fluid-flow-aiding-in-neural-waste-clearance https://edusehat.com/en/abdominal-contractions-may-drive-brain-fluid-flow-aiding-in-neural-waste-clearance Mon, 27 Apr 2026 18:40:10 +0700 Edusehat Abdominal, Contractions, May, Drive, Brain, Fluid, Flow, Aiding, Neural, Waste, Clearance Data from a new study in Nature Neuroscience shows that the brain may be more mechanically connected to the body than previously appreciated. Using mice and computational simulations of fluid motion, the team identified a possible biological mechanism that helps explain why exercise benefits brain health. Specifically, they found that abdominal contractions compress blood vessels that are connected to the spinal cord and brain, which helps the organ move gently within the skull. This movement facilitates the flow of cerebrospinal fluid over the brain, potentially washing away neural waste and preventing the development of neurodegenerative disorders. 

    The work, which is described in a paper titled “Brain motion is driven by mechanical coupling with the abdomen,” builds on past studies exploring how sleep and neuron loss influence how and when cerebrospinal fluid flushes the brain, according to Patrick Drew, PhD, a professor of engineering science and mechanics, neurosurgery, biology, and biomedical engineering at Penn State University. Drew is the corresponding author on the study. 

    “Our research explains how just moving around might serve as an important physiological mechanism promoting brain health,” said Drew. The contraction of abdominal muscles to push blood from the abdomen into the spinal cord acts “just like in a hydraulic system” that puts pressure on the vertebral venous plexus, a network of veins that connect the abdominal cavity to the spinal cavity which causes the brain to move. Computational simulations show “that this gentle brain movement will drive fluid flow in and around the brain” removing harmful waste. 

    To view this mechanism in moving mice, the scientists used two-photon microscopy, which allows for high-definition imaging of living tissue, and microcomputed tomography, which supports high-resolution three-dimensional examination of whole organs. They observed the brains shifting in the moments before the mouse moved and right after their abdominal muscles tightened, anticipating further movement. 

    To ensure that the abdominal contractions were the reason for the observed shift rather than other movements, the scientists applied gentle and controlled pressure to the abdomens of anesthetized mice. They observed that the mice’s brains moved in response. “Importantly, the brain began moving back to its baseline position immediately upon relief of the abdominal pressure,” Drew said, suggesting “that abdominal pressure can rapidly and significantly alter the position of the brain within the skull.” 

    The next step was digging deeper into the fluid’s movement in the brain as well as assessing if the brain’s movement could induce fluid flow. For this task, members of the team developed various techniques to capture this information including conducting imaging experiments of living mice and generating computational simulations of fluid motion. 

    “Modeling fluid flow in and around the brain offers unique challenges because there are simultaneous, independent movements, as well as time-dependent, coupled movements,” explained Francesco Costanzo, PhD, a professor of engineering science and mechanics, biomedical engineering, mechanical engineering, and mathematics, who led the computational modeling aspects of the project. “Accounting for all of them requires accounting for the special physics that happens every time a fluid particle crosses one of the many membranes in the brain. So, we simplified it” using the analogy of a sponge for the brain. By simplifying it in this way, Costanzo explained, the team could model how fluid flows through a structure with varied spaces.  

    Sticking with the analogy, “we also thought of it as a dirty sponge—how do you clean a dirty sponge?” Costanzo continued. “You run it under a tap and squeeze it out. In our simulations, we were able to get a sense of how the brain moving from an abdominal contraction can help induce fluid flow over the brain to help clear waste products.”  

    Further studies are necessary to understand how this mechanism works in human bodies particularly how it cycle cerebrospinal fluid around the brain, and helps to protect against neurodegenerative disease. “This kind of motion is so small. It’s what’s generated when you walk or just contract your abdominal muscles, which you do when you engage in any physical behavior. It could make such a difference for your brain health,” Drew said.  Overall, “our research shows that a little bit of motion is good, and it could be another reason why exercise is good for our brain health.”  

    The post Abdominal Contractions May Drive Brain Fluid Flow, Aiding in Neural Waste Clearance appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     StockWatch: Trump Order Lifts Psychedelic Drug Shares https://edusehat.com/en/stockwatch-trump-order-lifts-psychedelic-drug-shares https://edusehat.com/en/stockwatch-trump-order-lifts-psychedelic-drug-shares Mon, 27 Apr 2026 04:15:09 +0700 Edusehat StockWatch:, Trump, Order, Lifts, Psychedelic, Drug, Shares Stocks of most publicly traded psychedelic drug developers jumped when President Donald Trump signed Executive Order 14401, directing the FDA and other federal agencies to accelerate research and improve access to psychedelic drugs, citing their potential as promising treatments for serious mental illnesses.

    Among its provisions, the order directs the FDA to provide Commissioner’s National Priority Vouchers (CNPVs) to “appropriate” psychedelic drugs that were granted the agency’s Breakthrough Therapy designation and met the voucher program’s criteria. The FDA’s parent agency, the Department of Health and Human Services (HHS), is required to spend at least $50 million through the Advanced Research Projects Agency for Health (ARPA-H) “to support and partner with” state governments that have enacted or are developing programs to advance psychedelic drugs for serious mental illnesses.

    “This is an unmet public health need and potentially promising treatments. That’s why there’s a sense of urgency around this, and why we’re doing it now,” FDA Commissioner Martin A. Makary, MD, said at the ceremony where Trump signed the order. “Applications are about to come in, and this is the perfect timing for this announcement.”

    At least one analyst agreed that the timing was right for Washington to spur the development of psychedelic drugs.

    “Investor mindshare should rise meaningfully ahead of pot’l approvals in 2027–30,” Andrew Tsai, equity analyst with Jefferies, observed in a research note. “As we approach the first pot’l FDA approval of a psychedelic in 2027, President Trump is providing an official stamp of validation to the class in the form of an executive order, reassuring us that the FDA/HHS/White House’s support of psychedelics is real/actionable (not rhetorical).”

    Proving correct

    Makary said the FDA planned to issue CNPVs to three serotonin 2a agonists, a class that includes LSD and other psychedelic drugs. While he did not reveal specific companies and drugs by name, market watchers immediately speculated that one of the drugs was COMP360 synthetic psilocybin, the lead clinical candidate of Compass Pathways (NASDAQ: CMPS)—speculation that proved correct when COMP360 won a CNPV on Friday.

    COMP360 is expected, according to Tsai, to be the first psychedelic drug to win FDA approval in 2027. In February, Compass announced what it called statistically significant and clinically meaningful data from two Phase III trials assessing COMP360 in treatment-resistant depression (TRD), COMP005 (NCT05624268) and COMP006 (NCT05711940). The data showed positive effects for COMP360 within one day, lasting at least through six months after just one or two doses among those who have a clinically meaningful response.

    COMP360 is also in Phase II trials for both PTSD and anorexia nervosa.

    Compass fueled speculation about an FDA voucher approval by issuing a statement supporting the executive order: “Today’s announcement aligns regulatory urgency with patient need, and we applaud the Administration for taking this important step forward in accelerating access, without compromising rigorous science.”

    Investors celebrated with Compass, whose shares soared 42% from $6.66 to $9.46 on April 20, the first trading day after the order signing. Shares yo-yoed the rest of the week, sliding 7.5% to $8.75 Wednesday before rebounding nearly 5% to $9.15 Thursday and rising another roughly 5% to $9.58 Friday on news of the voucher approval. Year-over-year, Compass shares have more than doubled, soaring about 140% from $5.22 on April 24, 2025.

    FDA names additional voucher grantees

    The FDA indeed issued three CNPVs on Friday—one to Compass as previously mentioned, one to Usona Institute, a nonprofit medical research organization, for psilocybin for major depressive disorder (MDD), and one to Otsuka Pharmaceutical (Tokyo Stock Exchange: 4578) for methylone (TSND-201) for post-traumatic stress disorder (PTSD). Otsuka is acquiring the methylone program as part of its up-to-$1.225 billion ($700 million upfront) purchase of privately held Transcend Therapeutics, announced last month.

    Launched in October by Makary, CNPVs are awarded to drug developers whose work is deemed to address a health crisis in the United States, deliver more innovative cures, address unmet public health needs, and increase domestic drug manufacturing as a national security issue. The vouchers entitle companies to reviews of their final applications within a target timeframe of 1–2 months, rather than the current 10–12 months.

    “Ultimately, we do not see the FDA’s issuance of the first set of CNPVs as precluding other psychedelic players from also obtaining CNPVs in the future—so we think the FDA’s action today bodes well for the space broadly,” Tsai wrote after the FDA announced the voucher recipients. “Net-net, the macro backdrop for psychedelics is improving.”

    That improvement, Tsai added, reflects Trump’s endorsement of psychedelic drugs, a collaborative FDA, and growing interest in the space by big pharma giants such as Johnson & Johnson (NYSE: JNJ), which generated $1.696 billion in 2025 sales and $468 million in first quarter sales from Spravato (esketamine), an NMDA receptor antagonist indicated for treatment-resistent depression (TRD) and depressive symptoms in adults with MDD with acute suicidal ideation or behavior in conjunction with an oral antidepressant.

    The voucher decision hardly budged Otsuka shares, which dipped nearly 1% Friday from ¥10,870 ($68.18) to ¥10,810 ($67.80).

    However, Compass was one of several psychedelic drug companies to see their shares surge on news of the executive order.

    AtaiBeckley (NASDAQ: ATAI), formed last November by the merger of atai Life Sciences and Beckley Psytech, jumped 22% from $4.03 to $4.90 on April 20, then plateaued the rest of the week, finishing Friday at $4.63 and a 15% one-week gain. AtaiBeckley shares year-over-year have more than tripled, rocketing 204% from $1.53 a year ago Friday.

    Definium Therapeutics (NASDAQ: DFTX) shares rose 5% over two days, from $22.68 the Friday before Trump signed the order to $23.84 on Tuesday, but gave back all the week’s gain, finishing Friday at $22.48. Long-range investors have fared better, as Definium shares have more than tripled, zooming 249% from $6.43 on April 24, 2025.

    GH Research (NASDAQ: GHRS) shares climbed 17% from $18.34 to $21.50 the first day after the executive order, only to drop 6% the rest of the week, closing Friday at $20.25 and settling for a 10% one-week gain. GH’s shares doubled year-over-year, growing 101% from $9.50 a year ago Friday.

    Showing volatility

    The executive order wasn’t enough to boost shares of Cybin, which operates under the name Helus Pharma (NASDAQ: HELP). Helus showed the most volatility in the days following the order signing, tumbling 12% from $5.61 to $4.93 on April 20. The drop followed Helus’ announcement that its CEO, Michael Cola, stepped down immediately at the request of its board, succeeded by interim CEO Eric So, while the board carries out a search for a permanent chief executive.

    “It’s like they can’t give investors a break,” fumed “SamZaki320” on a Reddit chat board. “The only positive is that the executive order sentiment is pushing back against a total disaster. Not that it’s a good thing, other companies are up double digits.”

    Helus shares bounced back 17% to $5.77 Wednesday and dipping 0.35% to $5.75 Thursday despite the company announcing two powerhouse additions to its scientific advisory board—Robert Langer, ScD, the David H. Koch Institute professor at MIT and a co-founder of Moderna (NASDAQ: MRNA); and Stephen Brannan, MD, a neuroscience drug development expert with over 20 years of experience designing and implementing clinical programs for psychiatric and neurological disorders. Shares fell 2% Friday, closing at $5.61.

    In a statement, interim CEO So lauded Trump’s order: “The Executive Order reflects growing recognition of the urgent need for new treatment options in serious mental health conditions and the importance of advancing innovative therapies through rigorous, research-based development.”

    Looking beyond Washington

    Yet So acknowledged that Washington alone can’t advance psych drug development beyond what its science can accomplish: “Policy momentum is meaningful, but the future of this field will ultimately be determined by the strength of the clinical evidence and the ability to deliver safe, reliable treatments at scale.”

    As did Helus and Compass, Definium also praised the executive order: “We applaud the Administration’s recognition that psychedelic medicines may represent meaningful new treatment options for patients,” Definium CEO Rob Barrow stated. He cited his company’s clinical development program for DT120 (lysergide tartrate) for conditions that include generalized anxiety disorder (GAD) and MDD.

    At the ceremony where he signed the executive order, Trump acknowledged being asked to address psych drug development by podcaster Joe Rogan and others, which the president said led to talks with Makary as well as HHS Secretary Robert F. Kennedy Jr., NIH Director Jay Bhattacharya, MD, PhD, and Mehmet Oz, MD, administrator for the Centers for Medicare & Medicaid Services.

    “Research has been going on for quite some time. But usually with things like this, nothing ever happens, no matter how the research ends up. We’re changing that,” Trump said. “Why would we wait three or four years to get it done? Or 10 years? Frankly, let’s get it done immediately—and that’s what happened.”

    Leaders and laggards

    • Daiichi Sankyo (Tokyo Stock Exchange: 4568) shares slipped 10% from ¥2,790 ($17.50) to ¥2,499 ($15.67) on Friday after the drug developer announced it was delaying the release of its annual earnings results for the fiscal year that ended March 31, from April 27 to May 11,  “as additional time is required to finalize the financial figures.” May 11 is the day when Daiichi Sankyo plans to release its five-year business plan. “The company is currently reviewing the supply plans for its oncology products portfolio and development pipeline in light of rapidly changing business conditions. As a result, additional deliberation is required to reasonably estimate the amount of loss provisions to be recorded in connection with contracts with contract manufacturers,” Daiichi Sankyo added in a statement.
    • Inhibrx Biosciences (NASDAQ: INBX) shares leaped 37% from $84.08 to $115.09 Wednesday after Reuters reported, citing unnamed sources, that Merck & Co. (NYSE: MRK), Merck KGaA (XETRA: MRK), and Ono Pharmaceutical (Tokyo Stock Exchange: 4528) were in talks with Inhibrx for a joint spinoff of two precision-engineered cancer candidates, INBRX-106 and ozekibart (INBRX-109). The treatments could have a combined value of more than $9 billion if their clinical trials prove successful, the report stated. Inhibrx declined to comment, while the other companies cited did not respond to Reuters queries. INBRX-106 is a hexavalent sdAb-based, OX40-targeting candidate being studied as monotherapy and in combination with Merck & Co.’s cancer immunotherapy blockbuster Keytruda® (pembrolizumab). Ozekibart is a tetravalent death receptor 5 (DR5) agonist antibody designed to exploit the tumor-biased cell death induced by DR5 activation. On Tuesday, Inhibrx announced ozekibart showed positive data in a Phase I/II trial (NCT03715933) assessing the drug plus Folfiri in patients with locally advanced or metastatic, unresectable colorectal cancer.
    • Organon (NYSE: OGN) shares surged 31% from $8.60 to $11.26 Friday after the Indian news outlet The Economic Times reported that Sun Pharmaceutical Industries (NSE: SUNPHARMA and BSE: 524715) had submitted a $13 billion offer for the women’s health drug developer spun out of Merck & Co. (NYSE: MRK) in 2021. The deal would be Sun’s largest ever merger-and-acquisition (M&A) deal—if Sun can prevail over at least two other would-be suitors for Organon, German-based private family-owned drug developer Grünenthal, and EQT (Nasdaq Stockholm: EQT), a Swedish-based global investment organization. The latest surge comes two weeks after Organo shares zoomed 28% on an April 10 Economic Times report stating that Sun Pharma had submitted a $12 billion all-cash offer for Organon.
    • Spruce Biosciences (NASDAQ: SPRB) shares tumbled 26% from $69.89 to $51.69 Tuesday after the neurological disorder drug developer priced a $69 million offering of common stock and pre-funded warrants that generated $64.4 million in net proceeds. The offering consisted of 1.15 million shares of common stock priced at $50 per share and pre-funded warrants to purchase 50,000 shares at $49.99 per share. Al shares and pre-funded warrants were sold, and underwriters of the offering exercised in full their option to purchase up to an additional 180,000 shares at the public offering price on Tuesday. “We intend to use the net proceeds from this offering to advance the company’s pre-commercial and launch activities, for planned clinical trials, and for working capital, capital expenditures, and other general corporate purposes,” Spruce stated in its prospectus supplement filed Tuesday. Leerink Partners, Guggenheim Securities, and Oppenheimer & Co. acted as joint book-running managers, while Jones and Craig-Hallum acted as co-managers for the offering.

    The post StockWatch: Trump Order Lifts Psychedelic Drug Shares appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     One Biosciences Chooses Albany, NY, as Its U.S. Location https://edusehat.com/en/one-biosciences-chooses-albany-ny-as-its-us-location https://edusehat.com/en/one-biosciences-chooses-albany-ny-as-its-us-location Sat, 25 Apr 2026 05:40:10 +0700 Edusehat One, Biosciences, Chooses, Albany, NY, Its, U.S., Location Paris-based One Biosciences, an Institut Curie-backed startup, plans to set up, staff, and equip a high-complexity lab and computational analytics operation in Albany, NY, as its first U.S. location.

    Empire State Development is supporting this expansion with up to $525,000 in performance-based Excelsior Jobs Program tax credits in exchange for the company’s job commitments, which anticipate 42 life science jobs and $18 million in investments over the next five years.

    Officials at One Biosciences say the company will bring its proprietary technology to the first-of-its-kind hub in Albany to address the unmet clinical and scientific needs to characterize the tumor ecosystem by means of a single-cell profiling approach.

    “We are excited to accelerate support of our pharma, biotech, and academic collaborators through our AI-driven single-cell technologies, which will ultimately benefit physicians and their patients,” added Vincent Miller, MD, executive chairman, One Biosciences. “The local Albany life sciences ecosystem gives us access to a community of like-minded researchers and physicians committed to leveraging technology to improve health and is an ideal location from where to serve the U.S. globally.”

    “Life science research and development is vital to creating the treatments that help people heal, survive and live longer,” said New York governor Kathy Hochul. “Through our targeted efforts, we are working to ensure that cutting edge companies like One Biosciences not only grow here, but that the next generation of medical breakthroughs happen in New York State.”

    The post One Biosciences Chooses Albany, NY, as Its U.S. Location appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Single-Cell Atlas of the Prenatal Brain Reveals How Down Syndrome Reshapes Development https://edusehat.com/en/single-cell-atlas-of-the-prenatal-brain-reveals-how-down-syndrome-reshapes-development https://edusehat.com/en/single-cell-atlas-of-the-prenatal-brain-reveals-how-down-syndrome-reshapes-development Sat, 25 Apr 2026 05:40:09 +0700 Edusehat Single-Cell, Atlas, the, Prenatal, Brain, Reveals, How, Down, Syndrome, Reshapes, Development

    A cellular-resolution molecular map details how Down syndrome alters human brain development before birth. The study analyzed more than 100,000 nuclei from human prenatal neocortex samples collected across 26 pre-genotyped donors during gestational weeks 13 to 23—the only window during which all the cortical neurons a person will carry for their entire life are generated. The findings suggest that Down syndrome disrupts the developmental sequence of that process, creating shifts that may help explain later differences in cognition, learning, and sensory processing.

    This work is published in Science in the paper, “A single-cell multiomic analysis identifies molecular and gene-regulatory mechanisms dysregulated in developing Down syndrome neocortex.“

    “There’s a new level of detail here that had never existed before,” said Luis de la Torre-Ubieta, PhD, an assistant professor of psychiatry and biobehavioral sciences at UCLA and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research. “For the first time, we can really try to understand systematically what’s going on in the developing brain of individuals with Down syndrome.”

    “No one had looked at the developing human brain in Down syndrome directly using single-cell genomics,” he continued.

    The Down syndrome research field has historically focused on two areas: the adult brain and the disorder’s connection to neurodegeneration. What remained largely unexamined, despite clear indicators that Down syndrome is a developmental condition, was how the condition shapes the developing brain itself.

    The development of the prenatal neocortex typically follows a tightly orchestrated sequence. Progenitor cells must first divide repeatedly to expand their own pool, building up a sufficient base for all future neurons. Only then do they begin differentiating into neurons, starting with deep-layer cell types and progressing toward upper-layer cells in a carefully timed order.

    The study found that progenitor cells appear to rush prematurely into neuron production, depleting their own pool and skewing the balance of neuron types generated. Specifically, the researchers observed a relative increase in upper-layer intratelencephalic neurons and a reduction in deep-layer corticothalamic neurons.

    Those two cell populations play fundamentally different roles: CT neurons project outward from the cortex—connecting to brain structures and to the spinal cord to govern sensation and movement; IT neurons wire within the cortex, connecting the two hemispheres and contributing to information processing. This finding offers a new hypothesis for how early developmental changes might contribute to the cognitive profile of the condition.

    The finding also offers a new answer to a longstanding question in the field: Why do people with Down syndrome tend to have smaller brains? Earlier theories centered on elevated rates of cell death. The current study found less evidence of widespread neuronal death and instead points to the depletion of the progenitor pool.

    The study employed paired single-nucleus multiomics to reconstruct not just a snapshot of which cells are present, but the regulatory programs that guide cell fate—and how those programs are disrupted in Down syndrome. Systems-level approaches also led them to uncover alterations in cell metabolism and changes in how the vasculature interacts with the developing nervous system, both of which could speed up neuron production.

    The study’s significance extends beyond Down syndrome. The researchers specifically tested for overlap between the molecular disruptions they identified and the genetic risk signatures associated with other neurodevelopmental and neuropsychiatric conditions, including autism, epilepsy, and developmental delay. They found substantial convergence, particularly in the gene-regulatory networks governing the specification of IT versus CT neurons.

    “Down syndrome could be a model to understand intellectual disability and neuropsychiatric disorders more broadly,” de la Torre-Ubieta said. “Also to uncover the shared biology underlying these conditions—because the mechanisms are often still unknown.”

    The publication coincides with a companion paper from researchers at the University of Wisconsin-Madison, appearing in the same issue of Science. While the UCLA study focuses on the prenatal period, the Wisconsin team examined the postnatal brain, studying Down syndrome between approximately one and five years of age.

    Together, the two papers provide a continuous molecular view of Down syndrome brain development from mid-gestation through infancy—a resource that did not previously exist and that the researchers expect will serve as a reference for their field for years to come.

    While the researchers are careful to emphasize that the findings do not point to a near-term clinical application, the study provides the clearest picture yet of the cellular and molecular events that distinguish the Down syndrome brain during development, and a framework for identifying future therapeutic targets.

    The post Single-Cell Atlas of the Prenatal Brain Reveals How Down Syndrome Reshapes Development appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AI Learns to Predict Breast Cancer Risk from How Single Cells Respond to Pressure https://edusehat.com/en/ai-learns-to-predict-breast-cancer-risk-from-how-single-cells-respond-to-pressure https://edusehat.com/en/ai-learns-to-predict-breast-cancer-risk-from-how-single-cells-respond-to-pressure Sat, 25 Apr 2026 05:40:08 +0700 Edusehat Learns, Predict, Breast, Cancer, Risk, from, How, Single, Cells, Respond, Pressure A study headed by researchers at City of Hope and the University of California, Berkeley has found that physical and mechanical properties of normal human mammary epithelial cells can offer a “functional readout” of biological age and breast cancer susceptibility.

    The team created a novel, high-throughput microfluidic platform that can assess women’s breast cancer risk at the cellular level. The mechano-node-pore sensing (mechano-NPS) platform, which the researchers claim is the first of its kind, squeezes individual breast epithelial cells, creating a taxing environment to measure how they deform, recover, and behave under stress.

    Using the platform the researchers uncovered an unexpected insight, which is that breast cells appear to have a “mechanical age” separate from a person’s chronological age, demonstrated by how the cells physically respond to stress. For their study the team developed a machine learning classifier, MechanoAge, to estimate chronological age based on the mechanical phenotypes, and a biological age-based risk index, Mechano-RISQ.

    “We learned that the older the mechanical age, as determined by how cells respond to being squeezed through our microfluidic device, the higher the risk for breast cancer,” explained Lydia Sohn, PhD, the Almy C. Maynard and Agnes Offield Maynard Chair in Mechanical Engineering at UC Berkeley. The researchers suggest that, as more than 90% of women lack a known genetic predisposition to or a family history of breast cancer, their innovative approach could fill a critical gap in risk assessment and save countless lives.

    Sohn is co-senior author of the team’s published paper in eBioMedicine, titled “MechanoAge, a machine learning platform to identify individuals susceptible to breast cancer based on mechanical properties of single cells,” in which they concluded, “Age-related biomechanical changes may represent a fundamental hallmark of cellular function, with distinct mechanical phenotypes underlying critical processes in aging, cancer, and potentially other diseases. Recognizing and utilizing these biomechanical markers could greatly enhance early detection, refine risk stratification, and improve targeted intervention strategies.”

    Breast cancer is one of the most frequently diagnosed cancers worldwide and a leading cause of cancer-related mortality among women, the authors noted, and “… has long been the subject of efforts to improve risk stratification and early-detection strategies.”

    About 6% of women who develop breast cancer carry known genetic mutations. But for women outside this group, risk is estimated indirectly based on population models or measurements like breast density. These approaches can both overestimate and underestimate women’s individual breast cancer risk, leading to over-screening, under-screening, unnecessary worry or missed warning signs. And despite significant progress in screening technologies and therapeutic interventions, accurately determining which individuals—particularly among those considered average risk—are most likely to develop breast cancer remains what the team calls “one of the most persistent challenges in oncology and public health.”

    For these “ostensibly average-risk individuals,” the team added, “it remains difficult to identify those with latent risk that stems from cellular, molecular, and biophysical alterations that current models are not designed to capture.”

    Researchers Mark LaBarge of City of Hope (right) and Lydia Sohn (left) UC Berkeley [City of Hope and UC Berkeley]
    Researchers Mark LaBarge of City of Hope (right) and Lydia Sohn (left) UC Berkeley [City of Hope and UC Berkeley]
    Currently, there is no non-genetic test available that can identify women at higher risk for breast cancer. A downside to screening mammograms is that they can catch cancer only once it has begun to grow. Co-senior author, Mark LaBarge, PhD, a professor in the Department of Population Sciences at City of Hope, said “For women with a known genetic risk factor for breast cancer, there are things you can do like follow a higher-risk screening protocol. For everybody else, you’re left wondering, ‘Am I at high risk?’”

    Emerging evidence links cellular aging and biophysical alterations with cancer susceptibility. For their reported study the researchers used the mechano-NPS platform to profile primary human mammary epithelial cells (HMECs) from women of different ages and risk backgrounds. They also developed a machine learning algorithm that identifies and measures cells that show signs of accelerated aging, quantifying an individual breast cancer risk score.

    In this type of mechano-node-pore sensing, an electrical current is measured across a liquid-filled channel, much like how current is measured across a wire. As cells pass through, they disrupt the current, generating measurements about the cells’ size and shape. By making parts of the channel very narrow, researchers squeeze cells, then measure how long it takes each cell to recover its normal shape.

    Machine-learning algorithms developed by the researchers were then used to detect differences in cells from older and younger women. The researchers found that the physical properties of breast cells changed with age; cells from older women were stiffer and took longer to bounce back after being squeezed.

    Then came a surprising finding: a subset of younger women had cells that behaved like they came from older women. These cells came from women with genetic mutations that put them at high risk of breast cancer. Researchers then refined the algorithm to assign a risk score based on all the mechanical and physical properties measured in the cells. This algorithm successfully identified women with known genetic risks. Next the team used it to compare cells from healthy women, women who had family history of breast cancer and cells taken from the healthy breast of women with breast cancer in the other breast. “Normal epithelial cells from women with germline mutations, strong family history of cancer, or contralateral breast cancer exhibit mechanically aged phenotypes despite normal histology,” the investigators stated. “Together with prior molecular and epigenetic studies, these findings support a model in which accelerated biological aging of mammary epithelia may underpin breast cancer susceptibility across genetic and non-genetic risk groups.”

    Using the MechanoAge platform, researchers shifted the scientific lens to the cellular level, calculating risk by looking for physical changes in individual cells. “Mechanical phenotyping captures an integrative cellular state that reflects underlying molecular networks rather than single biomarkers,” the team noted. “Mechano-RISQ offers a proof of principle approach for identifying individuals at elevated risk of breast cancer, especially among average-risk populations, and may complement existing risk models by incorporating biophysical measures of mammary epithelial cell aging.”

    “With accuracy, we were able to figure out which women were at high risk of breast cancer and which women didn’t seem to be,” LaBarge said. “By translating physical changes in cells into quantifiable data, this tool gives women something tangible to discuss with their doctors—not just risk estimates, but evidence drawn directly from their own cells.” In their paper the scientists further stated, “This approach could enable earlier, individualized risk stratification, particularly for women who lack identifiable high-risk mutations yet harbor susceptible tissue states.”

    Importantly, the AI platform uses simple electronics that would be easy and affordable to replicate on a large scale. “Our team isn’t the first to measure the mechanical properties of cells; however, other approaches require advanced imaging technology that’s expensive, cumbersome and has limited availability,” said Sohn. “In contrast, MechanoAge uses computer chips that are simpler than an Apple Watch and ‘Radio Shack parts’ that are cheap and easy to assemble, potentially making the device highly scalable.”

    While engineers study the aging of materials such as metals, concrete and polymers, this is the first time that mechanical age has been quantified in biological cells. The finding that cells have a “mechanical age” separate from the individual’s chronological age would not have been possible without MechanoAge.

    This work grew out of more than 12 years of collaboration between the two labs, combining engineering innovation with cancer and aging biology. The long-term partnership enabled discoveries that neither group could have reached alone.  “It’s a true collaboration. We’ve learned a lot from each other,” Sohn said. “In my view, this is what happens when you have a real collaboration that develops over a long time,” LaBarge added. “This result is not what we imagined at the beginning.”

    The post AI Learns to Predict Breast Cancer Risk from How Single Cells Respond to Pressure appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AI in Oncology Takes Off, Tackling HIV and Liver Disease, Pharma’s Recent Gains https://edusehat.com/en/ai-in-oncology-takes-off-tackling-hiv-and-liver-disease-pharmas-recent-gains https://edusehat.com/en/ai-in-oncology-takes-off-tackling-hiv-and-liver-disease-pharmas-recent-gains Sat, 25 Apr 2026 02:00:12 +0700 Edusehat Oncology, Takes, Off, Tackling, HIV, and, Liver, Disease, Pharma’s, Recent, Gains Some GEN editors were in sunny San Diego covering the hottest research, trends, and products from the American Association for Cancer Research meeting. We kick things off with news from America’s Finest City, particularly around the growing role of AI in oncology. Then we dive into two new research studies. In the first, scientists used CRISPR to identify genes in primary CD4+ T cells that promote or restrict HIV infection. The second study described engineered implantable liver constructs that could eventually serve as a stopgap for patients waiting for donor transplants. Finally, the acquisitions keep coming as Eli Lilly scoops up CAR T cell therapy developer Kelonia for $7B. Also, Revolution Medicines has shared some impressive data from a Phase III trial of its pancreatic cancer drug.

    Listed below are links to the GEN stories referenced in this episode of Touching Base:

    AACR 2026: A Video Update from San Diego
    By Julianna LeMieux, PhD, and Damian Doherty, GEN, April 21, 2026

    AACR 2026 Video Update: Cancer Research Edges Toward an AI-Driven Era
    By Fay Lin, PhD, and Jonathan Grinstein, PhD, GEN, April 22, 2026

    Using AI in Healthcare Ethically by Considering Humanity
    By Corinna Singleman, PhD, IPM, November 18, 2025

    10x Genomics Unveils Atera Spatial Platform at AACR Meeting
    By Julianna LeMieux, PhD, GEN, April 19, 2026

    CRISPR Screens Map Human T‑Cell Genes That Promote or Block HIV Infection
    GEN, April 20, 2026

    Synthetic Biology and Tissue Engineering Grow Liver Tissue In‑Body
    GEN, April 20, 2026

    StockWatch: Revolution’s Phase III Pancreatic Cancer Data Dazzles Investors, Analysts
    By Alex Philippidis, GEN Edge, April 19, 2026

    Lilly to Acquire Kelonia for Up to $7B, Expanding Cancer Cell Therapy Pipeline
    By Alex Philippidis, GEN Edge, April 20, 2026

    Touching Base Podcast
    Hosted by Corinna Singleman, PhD

    Behind the Breakthroughs
    Hosted by Jonathan D. Grinstein, PhD

    The post AI in Oncology Takes Off, Tackling HIV and Liver Disease, Pharma’s Recent Gains appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Health-care AI is here. We don’t know if it actually helps patients. https://edusehat.com/en/health-care-ai-is-here-we-dont-know-if-it-actually-helps-patients https://edusehat.com/en/health-care-ai-is-here-we-dont-know-if-it-actually-helps-patients Fri, 24 Apr 2026 22:10:03 +0700 Edusehat Health-care, here., don’t, know, actually, helps, patients. I don’t need to tell you that AI is everywhere.

    Or that it is being used, increasingly, in hospitals. Doctors are using AI to help them with notetaking. AI-based tools are trawling through patient records, flagging people who may require certain support or treatments. They are also used to interpret medical exam results and X-rays.

    A growing number of studies suggest that many of these tools can deliver accurate results. But there’s a bigger question here: Does using them actually translate into better health outcomes for patients?

    We don’t yet have a good answer.

    That’s what Jenna Wiens, a computer scientist at the University of Michigan, and Anna Goldenberg of the University of Toronto, argue in a paper published in the journal Nature Medicine this week.

    Wiens tells me she has spent years investigating how AI might benefit health care. For the first decade of her career she tried to pitch the technology to clinicians. Over the last few years, she says, it’s as though “a switch flipped.” Health-care providers not only appear much more interested in the promise of these technologies, they have also begun rapidly deploying them.

    The problem is that many providers aren’t rigorously assessing how well they actually work.

    Take “ambient AI” tools, for example. Also known as AI scribes, they “listen” to conversations between doctors and patients, then transcribe and summarize them. Multiple tools are available, and they are already being widely adopted by health-care providers.

    A few months ago, a staffer at a major New York medical center who develops AI tools for doctors told me that, anecdotally, medics are “overjoyed” by the technology—it allows them to focus all their attention on their patients during appointments, and it saves them from a lot of time-consuming paperwork. Early studies support these anecdotes and suggest that the tools can reduce clinician burnout.

    That’s all well and good. But what about patient health outcomes? “[Researchers] have evaluated provider or clinician and patient satisfaction, but not really how these tools are affecting clinical decision-making,” says Wiens. “We just don’t know.”

    The same holds true for other AI-based technologies used in health-care settings. Some are used to predict patients’ health trajectories, others to recommend treatments. They are designed to make health care more effective and efficient.

    But even a tool that is “accurate” won’t necessarily improve health outcomes. AI might speed up the interpretation of a chest X-ray, for example. But how much will a doctor rely on its analysis? How will that tool affect the way a doctor interacts with patients or recommends treatment? And ultimately: What will this mean for those patients?

    The answers to those questions might vary between hospitals or departments and could depend on clinical workflows, says Wiens. They might also differ between doctors at various stages of their careers.

    Take the AI scribes, as another example. Some research on AI use in education suggests that such tools can impact the way people cognitively process information. Could they affect the way a doctor processes a patient’s information? Will the tools affect the way medical students think about patient data in a way that impacts care? These questions need to be explored, says Wiens. “We like things that save us time, but we have to think about the unintended consequences of this,” she says.

    In a study published in January 2025, Paige Nong at the University of Minnesota and her colleagues found that around 65% of US hospitals used AI-assisted predictive tools. Only two-thirds of those hospitals evaluated their accuracy. Even fewer assessed them for bias.

    The number of hospitals using these tools has probably increased since then, says Wiens. Those hospitals, or entities other than the companies developing the tools, need to evaluate how much they help in specific settings. There’s a possibility that they could leave patients worse off, although it’s more likely that AI tools just aren’t as beneficial as health-care providers might assume they are, says Wiens.

    “I do believe in the potential of AI to really improve clinical care,” says Wiens, who stresses that she doesn’t want to stop the adoption of AI tools in health care. She just wants more information about how they are affecting people. “I have to believe that in the future it’s not all AI or no AI,” she says. “It’s somewhere in between.”

    This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.
     

    ]]>     Japanese Pharma Companies Turning to CDMOs Earlier in Product Life Cycle https://edusehat.com/en/japanese-pharma-companies-turning-to-cdmos-earlier-in-product-life-cycle https://edusehat.com/en/japanese-pharma-companies-turning-to-cdmos-earlier-in-product-life-cycle Fri, 24 Apr 2026 22:05:09 +0700 Edusehat Japanese, Pharma, Companies, Turning, CDMOs, Earlier, Product, Life, Cycle Japanese pharmaceutical companies are engaging CDMOs earlier in the development cycle, as increasing complexity in peptide programs places greater strain on in-house capabilities, according to officials at Neuland Laboratories, which is attending CPHI Japan this week. The company says it has seen a notable shift in demand over the past 12–24 months, with more early-stage programs seeking external support.

    This trend is being driven in part by growing activity from venture-backed biotech companies and spinouts emerging from large pharmaceutical R&D organizations, reports a Neuland spokesperson, who adds that as these programs advance into clinical development, demand for specialized CDMO capabilities is increasing.

    Neuland has observed a rise in peptide-related engagements from Japanese companies, particularly at the preclinical and early clinical stages, where technical requirements are more demanding, notes Sharadsrikar Kotturi, PhD, CSO at Hyderabad, India-based Neuland Labs.

    Peptide development presents several challenges compared with traditional small molecules, explains Kotturi. Analytical complexity remains a key issue, with structural characteristics making characterization, impurity detection, and purity assessment more difficult, he continues. Scaleup is also constrained by the availability and quality of protected amino acids, which can affect manufacturing timelines, cost, and overall success rates.

    Regulatory expectations further add to the burden, points out Kotturi. Demonstrating purity, consistency, and process control to authorities such as Japan’s Pharmaceuticals and Medical Devices Agency requires extensive data, while shifting requirements introduce additional hurdles during development and approval. Simultaneously, pricing and regulatory pressures in Japan are increasing the operational load on drug developers, he states. Frequent drug price revisions are pushing companies to improve cost efficiency, reinforcing the case for outsourcing.

    “The bottleneck isn’t discovery anymore. It’s execution,” says Kotturi. “In peptides, programs are running into challenges around analytical complexity, scaleup, and the availability of key raw materials such as protected amino acids.”

     

     

     

    The post Japanese Pharma Companies Turning to CDMOs Earlier in Product Life Cycle appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO is expanding its work to defend IP https://edusehat.com/en/bio-is-expanding-its-work-to-defend-ip https://edusehat.com/en/bio-is-expanding-its-work-to-defend-ip Fri, 24 Apr 2026 18:30:05 +0700 Edusehat BIO, expanding, its, work, defend When a biotech firm sells investors on the promise of a new drug, what they are really selling is the intellectual property behind their innovation. That’s why biotech firms need a strong patent system to safeguard their IP.

    That’s also why the Biotechnology Innovation Organization (BIO) has always worked vigilantly to promote America’s patent system. BIO continues to build its work in this area, according to Joe Franklin, BIO’s Chief Legal and Policy Officer, who laid out the new initiatives and the issues involved ahead of International IP Day on April 26.

    “We are working on Capitol Hill, in the courts, and in international fora to advocate and educate stakeholders about how IP supports a strong biotech ecosystem—and how best to protect it,” said Franklin.

    As David Lachmann, BIO Senior Director for Federal Government Relations, explained, policies and court decisions that weaken U.S. IP protections risk America’s biotech leadership. “China has a history of IP theft, which continues, but they are innovating successfully and strengthening their IP system to protect those innovations,” Lachmann said. 

    “That’s going to be the challenge to the U.S. going forward.  Are we still going to be the gold standard for IP protection? Are people going to want to invest here? Do we want this essential industry in our country?” Lachmann asked. “At this point, these are national security questions. If we fall behind in IP, we fall behind in biotech, leaving Americans with reduced access to medicines and surrendering a strategic advantage.”

    BIO’s new work in IP

    BIO’s longstanding work to promote IP protections has included regularly convening the BIO IP Counsels Committee, a group of legal experts working for BIO members who collaborate on IP issues.

    “The BIO IP Counsels Committee is an awesome set of subject matter experts with legal expertise on patent and IP issues, like Hatch-Waxman and biosimilars,” said John Delacourt, BIO’s Deputy General Counsel. “Now we are looking to expand that network to bring together different disciplines—lawyers, Hill experts, patient advocates, and others with a policy or communications lens.”

    BIO is currently organizing an IP Task Force, where experts from member companies will come together with BIO staff to drive BIO’s IP strategy and specific policy activities, according to Franklin. Meanwhile, BIO’s Board has created the Economic Growth, Innovation, and Intellectual Property Committee, which will set BIO’s IP strategy and oversee the IP-related work performed by the IP Task Force and elsewhere at BIO.

    These initiatives will enable a focus on the most important policy priorities and advocacy efforts in the area of IP, to ensure BIO has the highest possible impact on issues that matter, according to Franklin. Along with advocacy, the work will include support for thought leadership in the form of reports and other publications, to help lawmakers and the public better understand the issues, he added.

    Specific areas for action

    According to Lachmann, one issue the public could understand better is multiple patents. Several patents can be necessary to protect different aspects of a drug, or improvements, such as inventing means for taking medicine as a pill instead of an injection, he explained.

    “Judging by the public debate, most people think patents are basically good, but they are suspicious of multiple patents because they don’t understand the difference between a single invention and a product that may include multiple inventions,” according to Lachmann.

    That’s why BIO is working to explain to lawmakers and the public about the risks of the ETHIC Act, proposed legislation that would render all but one patent associated with a pharmaceutical unenforceable, he said.

    BIO is engaged in congressional advocacy for IP protections on several fronts. For example, BIO entered a statement for the record in an April 16 hearing before the Senate Health, Education, Labor & Pensions Committee.

    The statement explained the importance of exclusivity periods for newly invented biologics and discussed a proposal for the Food and Drug Administration (FDA) to improve transparency of this exclusivity.

    “Put simply, life science innovations are hard to discover and develop, and yet they are often very easy to copy, which illustrates why patent protections are so vital,” said BIO’s statement.

    In addition to work on the Hill, BIO is also working through the courts. BIO recently partnered with PhRMA to discuss the implications of a pending Supreme Court patent case on drug innovation in an amicus brief submitted to the Court on March 27.

    The brief outlined the importance of enabling generic drugs while protecting innovation, a balance enshrined in the 1984 Hatch Waxman Act. It also explained the importance of research into new indications.

    “Medical progress depends on costly investment in research and development, including investments to study existing medicines for potential new uses,” according to the amicus brief. “That post-approval research has delivered important new treatments for hard-to-treat and rare diseases, and the benefits of some of these new uses have dwarfed the benefits of the original indication several times over.”

    Franklin said the amicus brief delves into complex topics that deserve to be better understood.

    “The brief emphasizes the nuances of the biopharma IP landscape and stresses the importance of the Hatch-Waxman framework, which balances innovation with market entry of generics,” he explained. “To be relevant in the IP space, we have to address complex legal and policy issues with nuance.”

    BIO’s international work includes submitting comments to the U.S. Trade Representative’s office for the annual Special 301 report. That report informs trade policy by identifying practices that weaken IP protections, including when drug makers are pressured to give up their licensing rights and localize their technology in exchange for accessing a country’s market.

    The submission explains why it’s important to address such unfair practices by China and other international competitors. “A robust biotech ecosystem in the U.S. is a national security priority,” Franklin said. “IP is obviously central to that.”

    This centrality of IP is a message that Franklin wants to emphasize.

    “IP intersects with so many areas that are really important to BIO, like national security, trade, and regulatory modernization,” Franklin said. “By showing how these areas are connected with IP, we can help to promote IP policies that enable a strong biotech ecosystem.”

    The post BIO is expanding its work to defend IP appeared first on Bio.News.

    ]]>     New ADC Yields Encouraging Clinical Benefit in Platinum-Resistant Ovarian Cancer https://edusehat.com/en/new-adc-yields-encouraging-clinical-benefit-in-platinum-resistant-ovarian-cancer https://edusehat.com/en/new-adc-yields-encouraging-clinical-benefit-in-platinum-resistant-ovarian-cancer Fri, 24 Apr 2026 03:55:13 +0700 Edusehat New, ADC, Yields, Encouraging, Clinical, Benefit, Platinum-Resistant, Ovarian, Cancer

    Patients with advanced platinum-resistant ovarian cancer whose disease had progressed on standard therapy experienced clinical benefit when treated with the investigational antibody-drug conjugate (ADC) QLS5132.

    This finding is according to results from a Phase I clinical trial presented at the American Association for Cancer Research (AACR) Annual Meeting 2026, held in San Diego.

    Patients diagnosed with platinum-resistant ovarian cancer face both a poor prognosis and limited treatment options, explained Tao Zhu, MD, chief physician and vice president of Zhejiang Cancer Hospital in China, who presented the study.

    Zhu and collaborators tested an investigational ADC, QLS5132, which targets the protein CLDN6. QLS5132 combines a CLDN6-targeting monoclonal antibody with a cytotoxic payload, topoisomerase-1 inhibitor, at a drug-to-antibody ratio of 8:1. CLDN6, Zhu said, makes an ideal target as a protein with very high expression on the surface of ovarian cancer cells and minimal cell-surface expression in healthy tissues.

    “The primary purpose of this first-in-human study was to evaluate the safety, tolerability, and pharmacokinetic profile of QLS5132 in patients with platinum-resistant ovarian cancer and determine the recommended Phase II dose for future clinical development,” Zhu said. “Additionally, we aimed to assess preliminary antitumor activity to establish an early signal of clinical benefit in this heavily pretreated population with limited options.”

    The Phase I, single-arm, dose-escalation trial enrolled 28 patients with a median age of 57.5 who had been diagnosed with advanced platinum-resistant ovarian cancer and who had experienced progression while on standard therapy. The research team administered QLS5132 as an intravenous infusion every three weeks at dose levels of 1.6 mg/kg, 3.2 mg/kg, 4.8 mg/kg, 5.6 mg/kg, and 6.4 mg/kg.

    Treatment-related adverse events (TRAEs) occurred in 26 (92.9%) patients, with nausea, anorexia, anemia, and weakness occurring most frequently. Nine (32.1%) patients experienced TRAEs of grade 3 or higher, and of those grade ≥3 TRAEs, seven were instances of hematological toxicity. No TRAEs led to treatment discontinuation or death, and no patients experienced interstitial lung disease, ocular toxicity, or febrile neutropenia, Zhu said.

    After a median follow-up of 2.2 months, nine patients had a partial response at various dose levels. Two of these partial responses occurred in patients who had no detectable CLDN6 expression.

    Across all dose levels, 18 evaluable patients experienced an objective response rate of 50% and a disease control rate of 94.4%. When calculated for the 17 evaluable patients who had received dose levels ≥3.2 mg/kg, the objective response rate and disease control rate rose to 52.9% and 100%, respectively. These responses to QLS5132 occurred irrespective of patients’ CLDN6 expression levels at baseline.

    “The most encouraging finding from our study was that QLS5132 demonstrated compelling antitumor activity in patients with platinum-resistant ovarian cancer, with an objective response rate exceeding 50%,” said Zhu. “Equally important, at the potential recommended Phase II dose, we observed a favorable safety profile with no reported cases of interstitial lung disease, ocular toxicity, oral mucositis, or febrile neutropenia.”

    Zhu also noted that, though more research would be needed to confirm, preliminary data indicated antitumor activity from QLS5132 regardless of CLDN6 expression levels—which, he said, could expand its potential as a treatment option to a broad cohort of patients with platinum-resistant ovarian cancer.

    Zhu acknowledged that further research would be needed to fully understand why QLS5132 can have anticancer effects in patients with undetectable CLDN6 tumoral expression. But he suggested the phenomenon may have a few explanations, including tumor heterogeneity, as well as a potent bystander effect resulting in antitumor efficacy even in cells with low or no CLDN6 expression.

    “These findings support the advancement of QLS5132 into Phase III studies, with the goal of providing a much-needed new treatment option for these patients,” said Zhu.

    Some limitations of this study include a small sample size and an exploratory single-arm design.

    This study was funded by Qilu Pharmaceutical. Zhu discloses no conflicts of interest.

    The post New ADC Yields Encouraging Clinical Benefit in Platinum-Resistant Ovarian Cancer appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AACR 2026 Video Update: Cancer Research Edges Toward an AI-Driven Era https://edusehat.com/en/aacr-2026-video-update-cancer-research-edges-toward-an-ai-driven-era https://edusehat.com/en/aacr-2026-video-update-cancer-research-edges-toward-an-ai-driven-era Fri, 24 Apr 2026 03:55:12 +0700 Edusehat AACR, 2026, Video, Update:, Cancer, Research, Edges, Toward, AI-Driven, Era SAN DIEGO – At the American Association for Cancer Research (AACR) Annual Meeting 2026, the conversation around AI-driven cancer research has moved decisively past theory. Now the focus is on what’s being deployed and how to gain researchers’ and clinicians’ trust.

    Fay Lin, PhD, senior editor, technology at GEN, and Jonathan D. Grinstein, PhD, North American editor at Inside Precision Medicine, discuss how AI is increasingly embedded across cancer research areas, from organoid models to pathology. Yet challenges such as data integration, longitudinal patient tracking, and clinician confidence continue to hinder its impact on patient outcomes.

    Watch the full discussion below for a clearer view of the trends, tensions, and inflection points in AI shaping the future of cancer research:

     

    The post AACR 2026 Video Update: Cancer Research Edges Toward an AI-Driven Era appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Regenerative Medicine: Promise, Hype, and What Actually Works https://edusehat.com/en/regenerative-medicine-promise-hype-and-what-actually-works https://edusehat.com/en/regenerative-medicine-promise-hype-and-what-actually-works Fri, 24 Apr 2026 03:55:11 +0700 Edusehat Regenerative, Medicine:, Promise, Hype, and, What, Actually, Works From stem cells to platelet-rich plasma, regenerative medicine is often positioned as the future of healthcare. But not all approaches deliver on that promise. As interest grows, so do questions regarding what actually works. GEN’s Editor in Chief John Sterling spoke with Thomas Buchheit, MD, founder and medical director of the Triangle Regen Medicine and Biologics Center in Chapel Hill, NC, in relation to the science, the hype, and the realities shaping the field today.

     

    GEN: How do you define regenerative medicine?

    Buchheit: Many people think of regenerative medicine as growing new organs, but I define it more broadly as any therapy that improves tissue health or function. With that definition, we can include platelet-rich plasma (PRP), stem cells, and autologous conditioned serum (ACS). These approaches aim to enhance tissue health and improve function.

    GEN: The field is promising, but also sometimes criticized as overhyped. Which areas deserve that criticism, and which have gained credibility through clinical validation?

    Buchheit: Some criticism is valid, especially around stem cells. We’ve all seen claims over “miracle” stem cells that regrow cartilage. In reality, while these cells can be therapeutic, they typically don’t survive long after injection. Instead, they work by activating the body’s immune-based healing mechanisms. They can improve tissue health, but they’re not the miracle cures they were once portrayed to be.

    On the other hand, therapies like PRP and ACS have gained credibility when properly applied and studied, particularly in musculoskeletal conditions.

    GEN: How do you incorporate regenerative medicine into your practice?

    Buchheit: I focus on patient function—what people can do now and what they want to achieve. Then tailor therapies accordingly. I prioritize treatments with strong evidence. One example is ACS, also known as the Regenokine* program. It’s highly standardized and supported by over 20 years of research in osteoarthritis, sciatica, and radiculopathy.

    Thomas Buchheit, MD
    Thomas Buchheit, MD

    I also use PRP, which can be effective, but only when properly dosed. That’s been a major challenge since there are many ways to prepare PRP. We now know that dose matters. For example, treating knee osteoarthritis typically requires close to 10 billion platelets. At our clinic, we measure platelet counts before and after preparation to ensure accuracy, something often not done enough or at all.

    GEN: Where did these approaches originate, and how widely are they used?

    Buchheit: ACS originated in Germany in the 1990s with Dr. Peter Wehling. It was initially developed as an alternative to steroids for treating sciatica. The process involves incubating whole blood under controlled conditions, which stimulates the release of anti-inflammatory proteins, growth factors, and exosomes.

    It became popular as patients, including athletes, traveled to Germany for treatment. Today, it’s available in the United States, though still more common in Europe. We now better understand how it works. Our research shows that exosomes play a key role in long-term benefits. If you remove them, effectiveness drops significantly.

    GEN: Your new book Healing Joints and Nerves—who is it for?

    Buchheit: It’s written for patients and a broad audience. I focused on authoring a book on regenerative medicine based on scientific accuracy and depth. I wanted to create a resource that explains these therapies clearly and truthfully—what they can and cannot do. It took over six years to complete. The book covers the history of stem cells and concludes with ACS, including both research and my personal experience with it as an avid runner and bicycle rider.

    GEN: You often mention “good” vs. “bad” inflammation. What’s the difference?

    Buchheit: Chronic inflammation is harmful. It damages tissue, drives pain, and contributes to diseases like osteoarthritis. But acute, controlled inflammation is essential for healing. It triggers the body’s repair processes. Exercise is a good example. It creates cycles of inflammation and recovery that make us stronger. Regenerative therapies aim to harness this same mechanism.

    Interestingly, suppressing inflammation too aggressively can backfire. Studies show that patients who take anti-inflammatories after acute injuries may have a higher risk of chronic pain. Repeated steroid injections can also worsen joint damage over time.

    GEN: Does all PRP work for osteoarthritis?

    Buchheit: No. PRP must contain a sufficient platelet dose to be effective. Research shows that below approximately three billion platelets, it’s unlikely to work. Above four billion, effectiveness improves, and near 10 billion provides optimal results.

    A practical tip: patients should ask how much blood is drawn. If only 10 mL is used to produce PRP, it’s mathematically impossible to achieve a high dose. Proper preparation typically requires 60–120 mL. Patients should also ask whether platelet counts are measured.

    GEN: Please talk a bit more about Regenokine.

    Buchheit: The program is based on ACS, enhanced through a controlled incubation process. This stimulates cells to release anti-inflammatory proteins, growth factors, and exosomes. Treatment typically takes roughly a week. Patients often come to the clinic for that duration. We’ve seen strong results in osteoarthritis and spine conditions, especially in patients who haven’t responded to other treatments, including stem cells.

    GEN: What about safety, efficacy, and durability of results?

    Buchheit: Outcomes vary by patient, but the primary goal is restoring function—whether that’s walking a dog or running a marathon. My approach is to stay as evidence-based as possible. That’s critical in a field where there is some overpromise or poorly validated treatments.

    There are real concerns regarding product quality, sourcing, and transparency in some parts of the market. We need to know exactly what we’re using, how it works, and what evidence supports it. That’s how regenerative medicine will continue to advance responsibly.

    Thomas Buchheit, MD, founded the Triangle Regen Medicine and Biologics Center in Chapel Hill, NC, to bring a range of regenerative therapies to patients. He now serves as an adjunct associate professor at Duke and continues to work with scientists at the Center for Translational Pain Medicine.

    Buchheit began studying nerve injury pain and served as chief of pain medicine at Duke University Medical Center. He investigated the immune basis of pain relief following injury and the mechanisms behind regenerative therapies, including platelet-rich plasma, stem cells, and autologous conditioned serum. He has led several studies funded by the NIH and the Department of Defense.

    *Regenokine was developed by Peter Wehling, MD, in Germany, originally in the 1990s. It utilizes a patient’s own blood to create a serum rich in anti-inflammatory proteins, particularly the interleukin 1 receptor antagonist (IL-1Ra), which helps reduce inflammation and promote healing in joints and tendons. The treatment is used for conditions like osteoarthritis and has gained popularity among athletes seeking pain relief. While it has shown promise in small studies, it is not yet FDA-approved and is not covered by insurance in the United States.

     

     

    The post Regenerative Medicine: Promise, Hype, and What Actually Works appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Heart’s Constant Beating Suppresses Tumor Growth in Cardiac Tissues https://edusehat.com/en/hearts-constant-beating-suppresses-tumor-growth-in-cardiac-tissues https://edusehat.com/en/hearts-constant-beating-suppresses-tumor-growth-in-cardiac-tissues Fri, 24 Apr 2026 03:55:10 +0700 Edusehat Heart’s, Constant, Beating, Suppresses, Tumor, Growth, Cardiac, Tissues The results of a study by researchers at the International Centre for Genetic Engineering and Biotechnology (ICGEB) suggest that the heart’s constant beating may actively suppress tumor growth in cardiac tissues. The collective findings from the team’s research in mouse models and in engineered heart tissues (EHT) suggests that this is because cellular pathways in these tissues alter gene regulation in cancer cells to keep them from proliferating.

    Headed by Giulio Ciucci, PhD, and Serena Zacchigna, MD, PhD, at the ICGEB Cardiovascular Biology Laboratory, the scientists say the findings shed light on the role of mechanical forces in protecting the heart from cancer and may pave the way to new cancer therapies based on mechanical stimulation. First author Ciucci, together with senior author Zacchigna and colleagues reported on their findings in Science, in a paper titled “Mechanical load inhibits cancer growth in mouse and human hearts.” In their report the authors concluded “Collectively, the data presented in this work provide evidence that mechanical load in the heart inhibits cancer cell proliferation, likely explaining the low incidence of cardiac tumors.”

    Heart cancer is very rare in mammals, but as the authors noted, “The mechanisms that protect the heart remain elusive.” The adult human heart in addition has a limited capacity for self-renewal, with cardiomyocytes regenerating at roughly 1% per year. “This suggests that the same mechanisms that halt the proliferation of cardiac cells could also inhibit the growth of cancer cells in the adult heart,” the authors continued. One proposed explanation for this loss of cardiomyocyte proliferative capacity lies in the intense mechanical demands placed on heart tissues, which must continuously pump blood against significant resistance. “We hypothesized that it could similarly hamper the proliferation of cancer cells in the heart,” the investigators reported.

    Using a genetically engineered mouse model, Ciucci et al. first showed that the heart is remarkably resistant to cancer-causing mutations, even when potent oncogenic changes were introduced. To understand why, the authors developed a transplantation model in which the heart’s mechanical workload could be reduced. By grafting a donor heart into the neck of a compatible mouse, they created a “mechanically unloaded” organ, one that remained perfused with blood but did not bear physiological strain. “To assess the contribution of mechanical load to the low incidence and growth of cancer in the heart, we used a model of in vivo cardiac unloading by heterotopically transplanting a donor heart into the neck of a recipient syngeneic mouse,” they explained.

    Image of lung cancer cells (in green) growing in a heart, in which cardiomyocytes are stained in red. Nuclei are stained in blue. [Ciucci et al., Science 2026]
    Image of lung cancer cells (in green) growing in a heart, in which cardiomyocytes are stained in red. Nuclei are stained in blue. [Ciucci et al., Science 2026]
    After injecting human cancer cells directly into the heart muscle, they compared tumor behavior in the unloaded transplanted heart versus the animal’s native, mechanically active heart. Across their experiments, Ciucci et al. found that mechanical load consistently suppressed the growth of various cancer types, while unloading the heart promoted tumor cell proliferation within cardiac tissue.

    According to the study findings, mechanical forces within the tissue reshape the cancer cell genome’s regulatory landscape, influencing whether cells can proliferate. Central to this process is Nesprin-2, a protein that transmits mechanical signals from the cell surface to the nucleus. “Nesprin-2, a protein known to mediate mechanotransduction from the cytoplasm to the nucleus, emerged as a key molecule sensing mechanical forces operating in beating hearts and translating them into reduced cell proliferation,” the scientists reported.

    Nesprin-2, a component of the LINC complex, senses the mechanical microenvironment of the heart and functionally alters chromatin structure and histone methylation, reducing gene activity linked to tumor cell proliferation. When Nesprin-2 was silenced in cancer cells, those cells regained the ability to grow in the mechanically active environment of the heart, forming tumors. “Silencing of Nesprin-2 in lung cancer cells prior to their implantation in the heart in vivo restored the capacity of the cells to proliferate in the presence of physiological mechanical load, resulting in the formation of large tumors,” the authors stated.

    The team noted that their collective results shed light on the role of mechanical forces in protecting the heart from cancer and may pave the way to new approaches to cancer therapy. “This offers fundamental insights into the biology of cell proliferation within the myocardium, and additionally, the mechanical stimuli that operate in a beating heart could be exploited for the development of a mechanical therapy for cancer.”

    The post Heart’s Constant Beating Suppresses Tumor Growth in Cardiac Tissues appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     CRISPR Base Editing Repairs Hard-to-Treat Cystic Fibrosis Mutation in Cell Models https://edusehat.com/en/crispr-base-editing-repairs-hard-to-treat-cystic-fibrosis-mutation-in-cell-models https://edusehat.com/en/crispr-base-editing-repairs-hard-to-treat-cystic-fibrosis-mutation-in-cell-models Fri, 24 Apr 2026 03:55:09 +0700 Edusehat CRISPR, Base, Editing, Repairs, Hard-to-Treat, Cystic, Fibrosis, Mutation, Cell, Models Affecting an estimated 100,000 people globally, cystic fibrosis (CF) cases stem from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In the past several decades, scientists have successfully engineered various small-molecule therapies that lessen the severity of the disease. However there are still treatment challenges. Now, data from a new study in a cell model demonstrates that a gene therapy can successfully repair an “untreatable” mutation associated with a particularly severe form of the disease. Details of the potential therapy are published in a new Science Translational Medicine paper titled “Functional correction of the untreatable CFTR 1717-IG>A mutation through mRNA- and sgRNA-optimized base editing.” 

    Many current therapies benefit patients with the most common disease-associated mutation, F508del. However they often have little effect on patients who harbor other types of mutations. For example, some patients have a mutation named 1717-1G>A, which is relatively common but doesn’t have any approved therapies due to being a splicing mutation that results in little to no protein production. In fact, “about 10% of people with CF do not qualify for any of the available CFTR modulator therapies, particularly those people with severe splicing mutations that result in frameshifts and the formation of premature termination codons.”

    The 1717-1G>A mutation is the target of the therapy described in the paper, which was written by scientists from the University of Trento and their collaborators elsewhere. Specifically, the team developed an “adenine base editing strategy to efficiently correct the 1717-1G>A mutation,” they wrote in Science Translational Medicine. “By harnessing the SpRY-­ ABE9 system, which we delivered as optimized RNAs for both the base editor and single guide RNA (sgRNA), we achieved functional correction in patient-derived models.” 

    Furthermore, the scientists note that they opted to use base editing rather than strategies like base editing because it has the advantage of “typically higher nucleotide modification efficiencies and a streamlined system requiring only the editor and an sgRNA” and because it has been used in other CF studies. 

    Using their ABE9 base editor and modified CRISPR-Cas9 tool, the scientists report successfully editing up to 30% of target DNA in human embryonic kidney cell lines and patient-derived airway epithelial cells with minimal off-target effects. It also corrected the mutation in intestinal organoids derived from CF patients as evidenced by restored CFTR activity. 

    Additional studies are needed, especially in animals, to fully assess the effectiveness of potential therapy but early results are promising. Overall, the approach achieved an editing efficiency of 13%. Prior studies showed that 10% efficiency may be enough for functional recovery. The results suggest that the therapy could benefit the subset of patients whose disease is caused by 1717-1G>A.

    The post CRISPR Base Editing Repairs Hard-to-Treat Cystic Fibrosis Mutation in Cell Models appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Enabling In Vivo Lentiviral Therapies: Manufacturing Strategies to Improve Purity, Scalability, and Clinical Readiness https://edusehat.com/en/enabling-in-vivo-lentiviral-therapies-manufacturing-strategies-to-improve-purity-scalability-and-clinical-readiness https://edusehat.com/en/enabling-in-vivo-lentiviral-therapies-manufacturing-strategies-to-improve-purity-scalability-and-clinical-readiness Fri, 24 Apr 2026 03:55:08 +0700 Edusehat Enabling, Vivo, Lentiviral, Therapies:, Manufacturing, Strategies, Improve, Purity, Scalability, and, Clinical, Readiness

    Lentiviral vectors are gaining momentum not just as ex vivo tools but as potential in vivo therapeutic platforms. But with that shift comes a number of manufacturing challenges, including higher doses, tighter control of impurities, greater batch consistency, and scalable processes to meet both clinical and commercial needs.

    In this GEN Podcast, two experts from SK pharmteco, a global CMO, address these challenges and lay out some best practices that guide the manufacture of lentiviral vectors with the requisite purity, robustness, and economic feasibility required for widespread clinical adoption..

     

    Podcast Guests:

    Tatiana Nanda

    Tatiana Nanda, PhD
    CTO, Cell and Gene Therapy
    SK pharmteco

    Mardhani Aparajithan

    Mardhani Aparajithan
    Director of Manufacturing,
    Science and Technology
    SK pharmteco


    Produced with support from:

    skpharmteco logo

    The post Enabling In Vivo Lentiviral Therapies: Manufacturing Strategies to Improve Purity, Scalability, and Clinical Readiness appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Novel KIR‑CAR T Approach Shows Early Activity Against Solid Tumors https://edusehat.com/en/novel-kircar-t-approach-shows-early-activity-against-solid-tumors https://edusehat.com/en/novel-kircar-t-approach-shows-early-activity-against-solid-tumors Fri, 24 Apr 2026 00:20:10 +0700 Edusehat Novel, KIR‑CAR, Approach, Shows, Early, Activity, Against, Solid, Tumors

    CAR T cell therapies have revolutionized outcomes for certain blood cancers, yet their impact on solid tumors has lagged. The field has long wrestled with T cell exhaustion—a state in which engineered cells lose their potency and fail to sustain an anti‑tumor response.

    At this year’s AACR annual meeting in San Diego, researchers from the Perelman School of Medicine at the University of Pennsylvania presented first‑in‑human Phase I data pointing to a possible solution. Their novel “KIR‑CAR” T cell therapy demonstrated a favorable safety profile and early signals of activity across multiple solid tumor types.

    The investigational therapy, SynKIR-110, represents a departure from traditional CAR T designs. Rather than using a single-chain receptor, the therapy is modeled after natural killer (NK) cell receptors and uses a “multi-chain” architecture.

    This design separates tumor recognition from activation, effectively creating an intrinsic “on-off” mechanism. The T cell remains in a resting state until it encounters its target, at which point the receptor components assemble to trigger an immune attack.

    “The KIR-CAR design provides a natural ‘on-off’ mechanism, which helps avoid the problem of T cell exhaustion,” said Janos L. Tanyi, MD, PhD, principal investigator of the study. “The CAR turns on when it finds its target, kills it, and then rests, rather than constantly burning energy.”

    This contrasts with conventional CAR T cells, which remain continuously active and can become depleted over time, limiting their effectiveness—particularly in the more complex microenvironment of solid tumors.

    The Phase I dose-escalation trial enrolled nine patients with advanced, mesothelin-expressing cancers, including ovarian cancer, mesothelioma, and cholangiocarcinoma. These patients had limited treatment options, having received an average of four prior lines of therapy.

    Although the primary goal of the study was to assess safety, early signs of efficacy were observed. Disease stabilization was reported in four patients, and one patient in the highest dose cohort achieved an ongoing partial response.

    “These are cancer types that have never had an approved cell therapy,” Tanyi said. “We’re seeing good efficacy signals, even at low doses, and limited toxicity.”

    The results suggest that the therapy may be able to generate meaningful anti-tumor responses even in heavily pretreated populations.

    Safety has been another major barrier for CAR T therapies, particularly in solid tumors. However, the KIR-CAR approach appears to mitigate some of these concerns.

    No dose-limiting toxicities were observed in the initial cohorts. Cytokine release syndrome (CRS), a common side effect of CAR T therapy, occurred in 33% of patients but was limited to low-grade events. Notably, there were no cases of immune effector cell-associated neurotoxicity syndrome (ICANS), a more severe complication sometimes seen with CAR T therapies.

    The ability to limit toxicity while maintaining activity is a key step toward broader application of cell therapies in solid tumors.

    SynKIR-110 targets mesothelin, a protein expressed on the surface of several solid tumors but largely absent from normal tissues. This makes it an attractive target for immunotherapy, particularly in cancers such as ovarian cancer and mesothelioma, where treatment options are limited.

    The trial results indicate that the therapy’s activity is not confined to a single tumor type, raising the possibility of broader applicability across mesothelin-expressing cancers.

    The findings come amid growing efforts to adapt CAR T technology for solid tumors. While the approach has revolutionized hematologic malignancies, solid tumors present additional challenges, including immunosuppressive microenvironments, physical barriers to T cell infiltration, and antigen heterogeneity.

    Researchers are exploring multiple strategies to address these barriers, including improved targeting, combination therapies, and next-generation receptor designs such as KIR-CAR.

    As noted by CAR T pioneer Carl June, MD, advancing cellular therapies into solid tumors remains a central goal for the field.

    The Phase I study continues to enroll patients, aiming for a 42‑person cohort to define the maximum tolerated dose ahead of Phase II. Early readouts show that CAR T expansion rises with dose, a pattern that may translate into stronger anti‑tumor activity at higher levels.

    While still preliminary, the findings highlight the potential of multi‑chain CAR designs to sustain activity without added toxicity. If confirmed, KIR‑CAR therapies could usher in a new generation of engineered immune cells that more closely mirror natural immune regulation.

    For now, the data offer a promising sign that CAR T innovation may finally be gaining ground in solid tumors.

    The post Novel KIR‑CAR T Approach Shows Early Activity Against Solid Tumors appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Andelyn Partners with S. Korea-Based ENCell to Accelerate Global Delivery of Gene Therapies https://edusehat.com/en/andelyn-partners-with-s-korea-based-encell-to-accelerate-global-delivery-of-gene-therapies https://edusehat.com/en/andelyn-partners-with-s-korea-based-encell-to-accelerate-global-delivery-of-gene-therapies Thu, 23 Apr 2026 06:20:11 +0700 Edusehat Andelyn, Partners, with, Korea-Based, ENCell, Accelerate, Global, Delivery, Gene, Therapies Andelyn Biosciences and ENCell, both CDMOs, signed a collaboration agreement to create a strategic manufacturing bridge between the United States and Asia-Pacific (APAC) regions to accelerate the global delivery of gene therapies.

    The partnership leverages both companies’ GMP manufacturing facilities, technical expertise, and regional networks to fast-track the development, manufacturing, and global expansion of client programs, according to officials at both organizations.

    This partnership is designed to enable a streamlined “dual hemisphere” workflow. By providing a direct route between U.S. and APAC manufacturing hubs, the collaboration could help remove a number of the regulatory and logistical complexities of international expansion.

    Most importantly, facilitating in-country manufacturing for in-country clinical trials ensures regional supply chains can meet the specific needs of local patient populations, greatly reducing lead times and accelerating the path to commercialization, pointed out Wade Macedone, CEO at Andelyn.

    “Our partnership with ENCell is a powerful step forward in Andelyn’s mission to help bring life-saving therapies to patients worldwide,” he said. By joining forces with such a respected leader in South Korea, we are not just expanding our global footprint; we are leveraging our unique strengths to deliver a truly seamless international manufacturing network.”

    “This partnership with Andelyn represents a significant step in expanding the global CGT ecosystem,” added Jong Wook Chang, PhD, CEO of ENCell. “By combining Andelyn’s expertise in viral vector development and cGMP manufacturing with ENCell’s clinical and manufacturing capabilities across APAC, we are establishing a seamless manufacturing platform connecting the United States and Asia-Pacific.

    “Together, we will enable more efficient development and scalable production of gene therapies, supporting our clients from early-stage development through global clinical trials and commercialization.”

    The post Andelyn Partners with S. Korea-Based ENCell to Accelerate Global Delivery of Gene Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cellular Mechanisms Behind Diabetes-Derived Vascular Disease Unveiled https://edusehat.com/en/cellular-mechanisms-behind-diabetes-derived-vascular-disease-unveiled https://edusehat.com/en/cellular-mechanisms-behind-diabetes-derived-vascular-disease-unveiled Thu, 23 Apr 2026 06:20:10 +0700 Edusehat Cellular, Mechanisms, Behind, Diabetes-Derived, Vascular, Disease, Unveiled Diabetes affects over half a billion people globally. Along with direct consequences to those with the disease, it also contributes to and predisposes affected individuals to a host of other conditions. Specifically, it is a known contributing factor in the development of vascular disease, including peripheral artery disease. While therapies exist, they are not very effective, and peripheral artery disease can lead to restricted blood flow in peripheral limbs, which sometimes leads to amputation. Understanding the mechanism driving the connection at the tissue and cellular level has the potential to improve therapy options and the development of new treatments.

    Normal function of the peripheral vasculature requires communication and cooperation between the vascular endothelium and macrophages. “Monocytes patrol the vascular endothelium and remove damaged cells, and intimal-resident macrophages maintain a nonthrombogenic endothelial state,” wrote the authors of a study led by Zhen Chen, PhD, at City of Hope. They explained that under stress, macrophages can modulate vascular remodeling and in certain conditions, like cancer, they “can secrete inflammatory mediators to disrupt endothelial cell tight junctions and increase endothelial cell permeability.”

    The team decided to explore the cellular cross-talk between macrophages and endothelial cells, as well as the resulting vascular function, to better understand the mechanisms behind peripheral artery disease induced by diabetes.

    They published their work in a paper titled “Diabetes-induced TREM2–endothelial cell signaling impairs ischemic vascular repair” in Science Translational Medicine.

    Using samples collected post-mortem from either donors with type 2 diabetes or donors without diabetes, the researchers aimed to systematically map the interactions between macrophages and endothelial cells in the arterial wall. They “leveraged single-cell RNA sequencing and spatial transcriptomics to profile human mesenteric arteries…generating a transcriptome and interactome atlas of diabetic vasculature.”

    Their analysis identified increased expression in paired genes between macrophages and endothelial cells. Triggering receptor expressed on myeloid cells 2 (TREM2) is a gene previously identified as connected with metabolic disease, atherosclerosis, cancer, and neurodegeneration. In macrophages, TREM2 had increased expression in tissues from donors with type 2 diabetes, with concurrent expression of the TREM2 ligands in endothelial cells. Macrophages with increased TREM2 presented with a foamy cell structure, indicative of a pro-inflammation phenotype. Additionally, these cells had a proinflammatory gene profile.

    Inhibiting TREM2 in vitro resulted in proinflammatory responses in macrophages and endothelial cells, along with increased migration of endothelial cells. To replicate TREM2 inhibition in vivo, the researchers used a mouse model for diabetes with hindlimb ischemia and treated them with a neutralizing antibody. This resulted in symptom improvement and improved blood vessel flow. Alternatively, activation of TREM2 with an agonist resulted in reduced blood flow and vascular damage.

    Further analysis of human donor samples “confirmed elevated endothelial cell TREM2 signaling in human peripheral arterial disease, particularly in the setting of diabetes mellitus, highlighting its translational relevance.”

    Together, these data show how TREM2 is involved with macrophage-endothelial cell communication within the peripheral vasculature. The authors pointed out that while TREM2 is a therapeutic target in treating Alzheimer’s disease and cancer, it might also have utility in treating peripheral artery disease.

    “Plasma sTREM2 may be useful as a circulating marker of endothelial/vascular dysfunction in peripheral artery disease for risk stratification and outcome prediction,” they wrote. “In addition, our findings suggest caution when considering TREM2-enhancing therapeutics, particularly in individuals with existing diabetes mellitus and ischemic disease.”

    This work underscores the need for more research into the details of disease mechanistic function to both better understand the cause of disease and to identify potential therapeutic targets.

    “Future studies will need to dive deeper into how insulin deficiency or resistance and hyperglycemia activate macrophages to augment TREM2 expression and induce vascular dysfunction,” wrote Michael Chang, Michael T. Patterson, PhD, and Jesse Williams, PhD, in a related focus.

    “Overall, the work of Malhi et al. advances our mechanistic understanding of type 2 diabetes-driven peripheral artery disease and has laid the foundation for developing targeted therapies for a disease with few viable treatment options,” they concluded.

    The post Cellular Mechanisms Behind Diabetes-Derived Vascular Disease Unveiled appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Brain Astrocytes Form Far-Reaching Connections in Mice https://edusehat.com/en/brain-astrocytes-form-far-reaching-connections-in-mice https://edusehat.com/en/brain-astrocytes-form-far-reaching-connections-in-mice Thu, 23 Apr 2026 06:20:09 +0700 Edusehat Brain, Astrocytes, Form, Far-Reaching, Connections, Mice A study in mice headed by NYU Langone Health researchers has found that cells long thought to play a secondary role in brain function build their own far-reaching connections. These pathways appear to connect distant regions in ways that had not been mapped before.

    Experts usually describe the brain as a network of nerve cells (neurons) that send each other signals to pass along information. These neurons are maintained by another kind of brain cell, the star-shaped astrocyte, which ferries in nutrients and carries away waste.

    The newly reported study, headed by Melissa Cooper, PhD, a postdoctoral fellow in the department of neuroscience at NYU Grossman School of Medicine, revealed that, like neurons, astrocytes form organized webs, which enable them to communicate with other specific astrocytes across the brain rather than only sending local, generalized signals. In some cases, the pathways were found to link areas that were not already joined together by neurons.

    “For more than a century, neuroscientists have thought of neurons as the main actors in the brain,” said Cooper. “Yet our findings suggest that astrocytes, which are usually viewed as merely support cells, are also running their own widespread signaling pathway, adding another layer to how brain regions stay connected.” The team suggests that while their study was carried out in mice, not humans, the findings form the basis for future studies investigating how astrocyte networks might link with injury, disease, or aging and to learning and memory.”

    Cooper is first and co-corresponding author of the team’s published work in Nature, titled “Astrocytes connect specific brain regions through plastic networks,” in which the researchers stated, “Astrocyte networks can directly link brain regions that are not connected by neurons, suggesting that previously unassociated brain regions communicate with one another through gap junction-coupled astrocytes.”

    “Neuronal axons have traditionally been considered to be the primary mediators of functional connectivity among brain regions,” the authors wrote, and the role of communication mediated by astrocytes has been largely underappreciated. “This communication occurs through gap junctions—membrane channels that connect the cytoplasm of neighboring cells, enabling them to redistribute resources and share biochemical signals,” the team continued. “Studies using mice lacking astrocyte gap junctions have shown that these gap junctions are necessary for memory formation, synaptic plasticity, coordination of neuronal signaling, and closing the visual and motor critical periods.”

    In earlier work, Cooper reported that in a mouse model of the visual neurodegenerative disease glaucoma, astrocytes can redistribute resources from astrocytes around healthy neurons to damaged neurons. Yet the team had no way to see whether this kind of support-cell network extended across the entire brain.

    Cooper said the newly reported study is the first to map active, brain-wide communication networks built by astrocytes and to show that these pathways are highly specific. The research relied on a custom-built tracing tool that let the team follow the cells’ connections in far greater detail than had been possible using past methods. “Despite the importance of astrocyte gap junctional networks, studying them has been challenging,” the investigators noted. “Current methods such as slice electrophysiology disrupt network connectivity and introduce artefacts due to tissue damage.”

    For their study, the researchers used a harmless virus to deliver “network tracers” into astrocytes in selected brain regions of lab mice. These tracers tagged small molecules as the molecules passed through the gap junctions linking one astrocyte to another, allowing the team to see which cells were part of the same signaling pathway.

    The scientists then made the mice’s brains transparent and used a specialized microscope to capture three-dimensional images of every tagged astrocyte. By doing this across hundreds of mice, they could map astrocyte webs across brain areas. “These networks selectively connect specific regions, rather than diffusing indiscriminately, and vary in size and organization,” they reported. “We observe local networks that are confined to single brain regions and long-range networks that robustly interconnect multiple regions across hemispheres, often exhibiting patterns distinct from known neuronal networks.”

    A 3D network of interconnected astrocytes imaged inside a whole, transparent mouse brain. Each astrocyte's color shows its distance from the viewer; closer astrocytes are blue, while more distant astrocytes are red. [Cooper et al. Astrocytes connect specific brain regions through plastic networks. Nature. 2026. doi:10.1038/s41586-026-10426-6.]
    A 3D network of interconnected astrocytes imaged inside a whole, transparent mouse brain. Each astrocyte’s color shows its distance from the viewer; closer astrocytes are blue, while more distant astrocytes are red. [Cooper et al. Astrocytes connect specific brain regions through plastic networks. Nature. 2026. doi:10.1038/s41586-026-10426-6.]
    The tracing tool and brain-clearing method were designed to be relatively low-cost and easy to reproduce so that other labs could use them to study the networks in many brain diseases.

    In another part of the study, the team assessed mice that were genetically engineered with astrocytes that lacked gap junctions. The communication networks largely disappeared, suggesting that the pathways are active and depend on these physical bridges.

    “By challenging our understanding of how the brain communicates over long distances, our results may offer fresh insight into how it develops, ages, and behaves in conditions such as Alzheimer’s and Parkinson’s diseases,” said study co-senior author Shane A. Liddelow, PhD, an associate professor in the neuroscience and ophthalmology departments at NYU Grossman School of Medicine.

    Another key finding was that astrocyte networks are dynamic. When the team trimmed whiskers on one side of the mice’s faces—“this manipulation is known to induce robust structural remodeling in neurons,” the team noted—a pathway from the region that processes whisker touch got smaller and reconnected to different astrocyte partners.

    “The fact that astrocyte networks shrink and reroute after a loss of sensory signals suggests they may be shaped by experience,” said study co-senior author Moses V. Chao, PhD, a professor in the cell biology, neuroscience, and psychiatry departments at NYU Grossman School of Medicine. “It also raises the possibility that each of us has a somewhat unique pattern of connections molded by what our brains have learned and lived through.”

    The authors plan to investigate which molecules move through the networks and to apply their tracing tool to models of brain disorders. They also hope to examine how these webs change during development and aging, said Chao.

    Liddelow emphasized that while gap junctions and astrocytes exist in humans, it remains unknown whether the networks link the same regions in the same way as in mice. Nevertheless, in their paper, the team concluded that their findings “… establish foundation for future exploration of how astrocyte network structure and function are shaped by injury, disease, development, aging and experience-dependent processes such as learning and memory.”

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    ]]>     Overcoming the VLP Purification Bottleneck https://edusehat.com/en/overcoming-the-vlp-purification-bottleneck https://edusehat.com/en/overcoming-the-vlp-purification-bottleneck Thu, 23 Apr 2026 02:45:13 +0700 Edusehat Overcoming, the, VLP, Purification, Bottleneck Virus-like particles (VLPs) are a popular platform for biomanufacturers because of their good biosafety profile, immunogenicity, and ease of engineering, although downstream purification remains bottlenecked.

    “Successful purification of VLPs cannot rely on any single unit operation, but instead requires integrated, product-specific process design guided by the critical quality attributes of the target particle,” Jingchao Zhang, PhD, Chengdu University of Technology, and Chen Chen, Tianjin University, point out in a recent review. The complex processes needed to generate VLPs result in multiple routes to success, and they are each vulnerable to environmental and process-induced stress, they note.

    One option to mitigate such stress is buffer optimization. When developing buffers, they advise evaluating pH, ionic strength, ion species, excipients “such as nonionic surfactants,” and stabilizers that “improve thermal and freeze-thaw robustness.” Start by identifying conditions that most often cause the target molecule to fail, they advise. “This stress-informed characterization is particularly valuable because the stability of VLPs cannot usually be inferred from a single condition alone and may depend on both particle type and solution context,” they write.

    Another option is “gentle chromatography.” By that, Zhang and Chen mean macroporous (100 nm or greater pore sizes) chromatography media that support process scaleup by improving binding capacity, increasing mass transfer rates and recovery, and are gentler on VLPs than the narrow (less than 30 nm) agarose media that often are used. Emerging options include non-woven structures and medium-to-large pore hydrogel microspheres, both of which have achieved success, respectively, with adeno-associated viruses and exosomes. “Overall, the chromatographic strategy for VLP purification should be framed as a balance between separation performance and particle preservation,” they conclude.

    Process analytical technology is also increasingly valuable as technologies emerge to enable real-time monitoring, analysis, and control, they add.

    Managing product heterogeneity is another challenge, as VLP downstream purification must address process- and product-related impurities. Typically, this is a multi-step endeavor “including clarification, ultrafiltration/diafiltration, chromatography, and, where appropriate, disassembly/reassembly-based purification,” Zhang and Chen report.

    So far, there haven’t been many viral clearance studies for VLPs, they say. Part of the challenge is the many different expression systems used, such as E. coli, Chinese hamster ovary cells, and insect baculovirus expression vector systems.

    In comparing the major downstream viral clearance strategies mentioned in the literature, Zhang and Chen report:

    • Solvent/Detergent (TritonX-100) treatment has some environmental concerns and mainly inactivates enveloped viruses.
    • Anion-exchange chromatography shows robust viral clearance only if the isoelectric points of the virus and VLPs differ.
    • Ion-exchange chromatography is constrained in high-salt or complex sample matrices.
    • Cation-exchange chromatography is highly effective in specific conditions.
    • Virus filtration is gentle and clears enveloped and non-enveloped viruses, but large VLPs may be larger than the filter pore size.

    Looking forward, Zhang and Chen predict near-term VLP purification advances will include: responsive materials and media that enable precise control; AI and machine learning that predicts structure-performance relationships to accelerate materials screening; greater process intelligence; continuous processing; increased use of quality-by-design principles; parallel development in regulatory science; and clearer regulatory standards.

    The post Overcoming the VLP Purification Bottleneck appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Monitoring Mammalian and Microbial Bioprocesses in Real Time https://edusehat.com/en/monitoring-mammalian-and-microbial-bioprocesses-in-real-time https://edusehat.com/en/monitoring-mammalian-and-microbial-bioprocesses-in-real-time Thu, 23 Apr 2026 02:45:12 +0700 Edusehat Monitoring, Mammalian, and, Microbial, Bioprocesses, Real, Time At the 2026 BiOS conference in San Francisco, researchers presented a biosensing platform aimed at improving how living cells and tissues are monitored during drug bioprocessing. Known as TissueSense, the system provides continuous, real-time insight into cellular behavior without disrupting the biological environment.

    In biopharmaceutical manufacturing, maintaining consistent cell health and productivity is essential. Yet many monitoring approaches still rely on intermittent sampling or endpoint measurements, offering only partial visibility into dynamic biological processes. TissueSense addresses this limitation by enabling continuous, in situ observation—capturing changes as they unfold.

    The platform combines resonator-based photonic sensing with phase contrast microscopy, allowing simultaneous detection of biochemical activity and structural changes in cells. This dual approach provides a more complete picture of how cells respond to process conditions, such as nutrient shifts or environmental stress, which directly impact production outcomes.

    A defining feature of the system is its label-free operation. Conventional biosensing methods often require fluorescent markers or reagents that might alter cell behavior or limit long-term monitoring. By removing these constraints, TissueSense supports extended observation of living systems in conditions closer to their natural state, an advantage for prolonged bioprocesses.

    Data from the platform are analyzed using machine learning to simultaneously quantify up to 18 biomarkers, linking molecular outputs—such as secreted proteins—to tissue structure and function. This multiplexed capability is particularly relevant in drug manufacturing, where small variations in cellular activity can influence yield, quality, and reproducibility.

    While TissueSense focuses on mammalian tissue models, parallel advances in microbial systems highlight a broader shift toward continuous, high-resolution monitoring across bioprocessing platforms. In yeast-based systems, for example, researchers have developed microbead-based cultivation methods that enable high-throughput, label-free screening of millions of individual mutants in extremely small volumes. These approaches can enrich desirable traits, such as resistance to metabolic inhibitors, by thousands-fold, supporting strain optimization for industrial bioproduction.

    Similarly, in bacterial bioreactors, automated flow cytometry techniques now allow real-time tracking of population dynamics and physiological states. By combining DNA staining with indicators of active replication, these systems provide continuous insight into growth rates and cell cycle behavior, helping optimize feed strategies and overall process performance.

    Together, these developments point toward a more integrated future for bioprocess monitoring—one that spans mammalian, yeast, and bacterial systems. Continuous, non-destructive sensing technologies are enabling researchers and manufacturers to move beyond static measurements toward dynamic control of biological production.

    The post Monitoring Mammalian and Microbial Bioprocesses in Real Time appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AI Wizard Adapts Processes in a Self-Driving Lab https://edusehat.com/en/ai-wizard-adapts-processes-in-a-self-driving-lab https://edusehat.com/en/ai-wizard-adapts-processes-in-a-self-driving-lab Thu, 23 Apr 2026 02:45:10 +0700 Edusehat Wizard, Adapts, Processes, Self-Driving, Lab German researchers who run a self-driving laboratory have created an agentic AI wizard to help their students rapidly design and implement new processes.

    The wizard, which uses N8N software, can guide a student through establishing experiments without the need for coding, allowing them to quickly set up a new process.

    According to Matthias Franzreb, PhD, a professor and departmental leader in bioengineering and biosystems at the Karlsruhe Institute of Technology, developing wizards could help any autonomous laboratory where the experimental setup needs to change fast.

    “Each of our bachelor’s and master’s students has their own type of experiment and, in the beginning, going into Python scripting, it used to take two months to have the whole thing programmed,” he says.

    By contrast, he says, the AI agent can help the student develop a new process within one or two days. It has so far been used to develop around six processes, he says, for a slightly larger number of students, as the same template can be used more than once.

    Bioprocessing, like many other areas of human endeavor, is experiencing disruptive change with the growing use of digital tools at both the laboratory and commercial scale, Franzreb explained in a talk at Bioprocessing Summit Europe.

    Among these changes is the difference between classical labs, which have automated equipment, such as liquid handling stations, but where scientists must design and set up their own experiments, and self-driving labs. In the latter, he explains, machine learning uses a first set of experiments to autonomously decide what experiments should be next.

    In his talk, Franzreb also showed how a wizard could be used for designing a chromatography experiment. An experiment was set up to determine batch parameters at a small-scale in 96-well plates. From this, the software used a chromatography simulation to find the optimal conditions for the experiment and then ran it in a real chromatography system to validate the results.

    According to Franzreb, the next step for the self-driving laboratory will be working with the German Research Center for Artificial Intelligence (DFKI) and other research partners to develop ontological capabilities for the wizards so they can extract context for the experiments from Standard Operating Procedures (SOPs) or the academic literature.

    “I think this is simple in principle,” he explains. “But at the moment we don’t have it, and it will be a challenge to roll out.”

    The post AI Wizard Adapts Processes in a Self-Driving Lab appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Viral Contamination Still a Challenge for CGT Industry https://edusehat.com/en/viral-contamination-still-a-challenge-for-cgt-industry https://edusehat.com/en/viral-contamination-still-a-challenge-for-cgt-industry Thu, 23 Apr 2026 02:45:09 +0700 Edusehat Viral, Contamination, Still, Challenge, for, CGT, Industry Raw material testing will remain the foundation of cell and gene therapy (CGT) sector quality control strategies for the foreseeable future, according to new analysis, which shows the industry still lacks suitable virus detection and inactivation methods.

    Biopharmaceutical raw materials—the culture media ingredients, the reagents, and even the production cell lines themselves—are the biggest source of viral contamination in drug manufacturing.

    To mitigate the risks, the protein drug industry has developed downstream virus detection, inactivation, and removal strategies to make sure products do not pose an infection risk.

    For CGT firms, ensuring products are virus safe is more of a challenge, says Yoshiaki Maruyama, PhD, from the office of cellular and tissue-based products at Japan’s Pharmaceuticals and Medical Devices Agency (PMDA).

    “Viral contamination of CGT products may arise from virus-contaminated raw materials or ancillary materials of human or animal origin or from the inadvertent introduction of viruses during the manufacturing process.

    “Appropriate raw material controls and robust quality control parameters must be established and maintained throughout the manufacturing process to effectively manage the risk of viral contamination,” he tells GEN.

    Inactivation and removal challenges

    The big problem is that cell and gene therapies are too sensitive to survive current viral inactivation methods, most of which were developed with protein therapeutics in mind.

    Maruyama says, “Most conventional virus inactivation or removal processes inevitably result in cell damage or loss in cell therapy and tissue-engineered products or adversely affect viral vectors in gene therapy products.”

    As a result, CGT sector quality control efforts have focused on screening raw materials and finished products, according to Maruyama, who looked at current regulations and common approaches in a recent study.

    “In the CGT sector, viral safety is achieved by implementing a comprehensive viral testing program. The use of inactivation and removal processes is challenging for CGT products and raw materials, so quality control strategies relying on screening are generally used,” he says.

    Technological solutions?

    In future, technologies may play a greater role, according to Maruyama, who says, “

    “NGS technologies are expected to be applicable to the detection of adventitious viruses in human or animal cells. NGS offers a powerful, unbiased approach for detecting known and unknown viral contaminants,” they write.

    However, as the authors point out, further development will be required as NGS systems detect nucleic acids rather than viable, infectious virus particles.

    “Currently, there are no globally accepted NGS-based procedures or validated analytical methods that have reached a consensus on their use as substitutes for conventional viral tests. Therefore, the use of NGS as an alternative to conventional viral tests, including reducing the use of experimental animals, requires further evaluation depending on the specific test to be replaced,” they write.

    And in the future, artificial intelligence (AI) systems may also play a role.

    “This is largely speculative, and there are currently no concrete examples, but AI-based tools have been applied to manufacturing control for deviation prediction and similar approaches might also be useful for controlling viral contamination risks in CGT products and raw materials,” he says.

    The post Viral Contamination Still a Challenge for CGT Industry appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Antigen‑Stabilized Nanobody Probes Enable On‑Demand, Multicolor Protein Imaging https://edusehat.com/en/antigenstabilized-nanobody-probes-enable-ondemand-multicolor-protein-imaging https://edusehat.com/en/antigenstabilized-nanobody-probes-enable-ondemand-multicolor-protein-imaging Wed, 22 Apr 2026 19:35:10 +0700 Edusehat Antigen‑Stabilized, Nanobody, Probes, Enable, On‑Demand, Multicolor, Protein, Imaging Fluorescent probes have reshaped how biologists study living systems, making it possible to watch viruses invade cells, follow the cell’s internal waste‑disposal machinery, and track the signaling events that fuel tumor growth. Yet even with decades of innovation, a fundamental limitation has persisted: most fluorescent nanobody probes glow whether or not they are bound to their targets. That constant background haze can blur the very molecular details researchers are trying to resolve.

    A new imaging platform developed by scientists at Albert Einstein College of Medicine and the Salk Institute for Biological Studies aims to eliminate that problem entirely. The technology, described in Nature Methods in a paper titled “Synthetic multicolor antigen-stabilizable nanobody platform for intersectional labelling and functional imaging,” uses engineered fluorescent nanobodies that become brightly fluorescent only when they bind their intended protein targets. These “on‑demand” probes, known as VIS‑Fbs (visible-spectrum target-stabilizable fluorescent nanobodies), illuminate proteins inside living cells and animals with far greater clarity than conventional tools.

    “The key advantage of our approach is that the signal appears only where the target protein is present,” said Vladislav Verkhusha, PhD, co‑corresponding author and professor of genetics at Einstein. “That eliminates the background glow that has long limited the precision of intracellular imaging.” His collaborator, Axel Nimmerjahn, PhD, professor and the Françoise Gilot‑Salk Chair at Salk, added, “This work establishes a versatile platform for imaging proteins with high specificity and minimal background. It opens new opportunities to study how molecular and cellular processes unfold in real time across diverse biological systems.”

    Nanobodies have become increasingly valuable for live‑cell imaging because they can be engineered to bind specific proteins with high affinity. But their ongoing fluorescence has remained a stubborn obstacle. The VIS‑Fb design solves this by making the probes unstable when unbound; they rapidly degrade unless they encounter their target. Binding stabilizes the nanobody and triggers bright fluorescence, reducing background noise by as much as 100‑fold. The team also created VIS‑Fbs that span nearly the entire visible spectrum, from blue to far red, enabling simultaneous tracking of multiple proteins or cellular processes within the same cell.

    The researchers developed a modular engineering platform, instead of a single probe, capable of generating VIS‑Fbs for a wide range of targets and experimental needs. They integrated more than 20 fluorescent proteins and biosensors into multiple nanobody scaffolds, creating a flexible system that supports multicolor imaging, light‑switchable variants for precise temporal control, and functional readouts of ions and metabolites. This allows the probes not only to show where proteins are but also to show what those proteins are doing in real time. According to first author Natalia Barykina, PhD, “The VIS‑Fb approach allows us to identify and track specific cell populations in living organisms based on the proteins they express, rather than just their location.”

    In mice, VIS‑Fbs allowed for high‑contrast imaging of neuronal and astrocyte activity during behavior. In zebrafish embryos, the probes captured rapid developmental changes and responses to drugs that modulate signaling pathways. “Our results show that this imaging platform offers a much clearer and more precise view of how proteins behave inside living systems,” Verkhusha said. “It opens the door to studying complex biological processes, such as cell signaling, development, and disease progression, in new ways.”

    The post Antigen‑Stabilized Nanobody Probes Enable On‑Demand, Multicolor Protein Imaging appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cosmo Pharma Eyes 2027 NDA for Baldness Candidate After Positive Phase III 12-Month Data https://edusehat.com/en/cosmo-pharma-eyes-2027-nda-for-baldness-candidate-after-positive-phase-iii-12-month-data https://edusehat.com/en/cosmo-pharma-eyes-2027-nda-for-baldness-candidate-after-positive-phase-iii-12-month-data Wed, 22 Apr 2026 05:15:11 +0700 Edusehat Cosmo, Pharma, Eyes, 2027, NDA, for, Baldness, Candidate, After, Positive, Phase, III, 12-Month, Data Cosmo Pharmaceuticals says it plans to file for FDA approval of its androgenetic alopecia (AGA) candidate clascoterone 5% topical solution early next year, after the androgen receptor inhibitor generated 12-month Phase III data showing statistically significant continued hair growth as well as positive safety for chronic use.

    Data from Cosmo’s Phase III program for clascoterone confirmed the drug’s long-term safety profile was comparable to vehicle, supporting suitability for chronic use in a lifelong condition. Clascoterone also showed a novel mechanism designed to target the underlying biology of hair loss and continued efficacy with ongoing use, Cosmo said.

    A total of 1,465 patients were enrolled in the Phase III program, the largest for any topical treatment candidate for male AGA, according to Cosmo. The program consists of the SCALP 1 (NCT05910450) and SCALP 2 (NCT05914805) trials, which evaluated patients across 51 study centers in the United States and Europe.

    Patients who remained on continuous clascoterone treatment for the full 12 months achieved a statistically significant 239% improvement in Target Area Hair Count (TAHC) compared with patients who received clascoterone for six months and were then switched to vehicle from month 7 to month 12, according to Cosmo.

    That’s down slightly from the 252% improvement in TAHC shown for clascoterone versus “vehicle” or placebo in Cosmo’s six-month results, released in December. Cosmo Pharma CEO Giovanni Di Napoli told GEN that the 6- and 12-month results were not directly comparable due to differences in Part 1 and Part 2 of the Phase III placebo-controlled program and the corresponding patient groups being compared.

    Part 1 is a double-blind study assessing if clascoterone was effective and safe compared to placebo when applied twice daily for up to six months. Part 2 is a single blind study that measured clascoterone’s long-term safety and efficacy versus placebo for an additional six months in patients who had responded to the study drug in Part 1. During Part 2, participants were re-randomized to receive either clascoterone 5% solution or vehicle solution.

    SCALP 1 enrolled 702 patients in the United States, while SCALP 2 enrolled 763 patients in the Unites States as well as Germany and Poland.

    Primary outcome measures

    Change in vellus TAHC (hair of up to 30 micrometers in diameter) from baseline was the trials’ primary outcome measure, paired with a patient-reported outcome assessing participants’ perception of hair growth improvement.

    Additional assessments of the trials included investigator-reviewed global scalp photography and secondary endpoints that included changes in non-vellus TAHC (thicker, pigmented hair >30-40 micrometers in diameter), and changes in subject’s assessment of satisfaction score.

    Patients treated with clascoterone for 12 months reported a statistically significant +24.5% relative improvement in treatment satisfaction versus vehicle groups, according to Cosmo. The clascoterone users also reported positive ease of use and product acceptability at month 12—results that according to the company support positive real-world usability and long-term adherence potential for the drug.

    Cosmo said it plans to submit its full Phase III dataset for publication in a leading peer-reviewed medical journal and present its findings at future major dermatology congresses.

    ‘Defining moment’

    “These 12-month Phase III results mark a defining moment for clascoterone and for the treatment of male hair loss,” Di Napoli stated. “We are now seeing the combination that matters most: positive long-term safety, statistically significant continued hair growth through one year, and clear evidence that ongoing treatment drives sustained benefit.”

    Investors responded to the positive 12-month data by sending Cosmo shares traded on the SIX Swiss Exchange rising 6% on the day of the announcement, from CHF95.50 ($122.69) to CHF101.40 ($130.27) on April 15. Since then shares have fluctuated in the high CHF 90 range, closing Monday at CHF 98.50 ($126.55).

    Di Napoli said clascoterone has the potential to emerge as a major new therapeutic option and a highly valuable growth platform for Cosmo by tackling the most common cause of hair loss in men. Androgenetic alopecia, also called male pattern hair loss, affects approximately 40% of men worldwide—including 39% of males in the United States (65 million men).

    “We are moving with urgency toward regulatory submissions and commercialization discussions,” Di Napoli added.

    Cosmo’s results “likely now enable more advanced partnership discussions, in our view, with detailed presentation of results the next step to fully de-risk the asset,” Benjamin Jackson, equity analyst with Jefferies, wrote April 15 in a research note.

    Jackson predicted clascoterone could generate $4 billion in peak-year worldwide sales.

    “Our $4 billion WW potential peak sales require just 4% penetration of treated and 6% penetration of untreated men at peak, assuming a capable commercial partner is successfully found,” Jackson added.

    Cosmo said it is preparing to submit not only an NDA for clascoterone in the U.S., but a marketing authorization application to the European Medicines Agency.

    Clascoterone’s 1% formulation is already FDA-approved and marketed as Winlevi® for topical treatment of acne vulgaris in patients ages 12 and older.

    The post Cosmo Pharma Eyes 2027 NDA for Baldness Candidate After Positive Phase III 12-Month Data appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AACR 2026: Lung Cancer Immunotherapy Response Predicted by Pathomics AI Model https://edusehat.com/en/aacr-2026-lung-cancer-immunotherapy-response-predicted-by-pathomics-ai-model https://edusehat.com/en/aacr-2026-lung-cancer-immunotherapy-response-predicted-by-pathomics-ai-model Wed, 22 Apr 2026 05:15:09 +0700 Edusehat AACR, 2026:, Lung, Cancer, Immunotherapy, Response, Predicted, Pathomics, Model SAN DIEGO – A new AI model applied to routine pathology slides accurately predicts outcomes and response to immunotherapy in patients with metastatic non-small cell lung cancer (NSCLC). The study was study presented at the American Association for Cancer Research (AACR) Annual Meeting. 

    “Immunotherapy has transformed cancer treatment, but only a subset of patients benefit from it, and predicting who will respond remains challenging,” said Rukhmini Bandyopadhyay, PhD, a postdoctoral fellow at The University of Texas (UT) MD Anderson Cancer Center.  

    “This study represents, to our knowledge, the first deep learning-based pathomics biomarker rigorously validated across international real-world cohorts and a Phase III randomized clinical trial, directly addressing one of the most urgent unmet needs in precision oncology: reliable patient selection and stratification for immunotherapy,” he continued. 

    Pathomics applies computational and machine learning methods for high-throughput analysis of digital pathology images to extract large-scale data related to cell and tissue architecture linked to disease outcomes. 

    Bandyopadhyay and colleagues developed a deep learning survival prediction model called Pathology-driven Immunotherapy Optimization (Path-IO), which can study patterns across tissue to identify patients most likely to benefit from immunotherapy. The model then combines imaging and clinical data to estimate whether a patient may have a higher or lower risk of poor outcomes from immunotherapy. 

    The researchers tested the platform in a study that included 797 immune checkpoint inhibitor-treated NSCLC patients from UT MD Anderson, with external validation in 280 additional patients from Mayo Clinic, Gustave Roussy, and the Phase III Lung-MAP S1400I trial in which immunotherapy-naïve patients with lung squamous cell carcinoma, a subtype of NSCLC, were treated with immune checkpoint inhibitors. 

    The model reliably stratified patients into higher and lower risk groups. In the UT MD Anderson cohort, patients in the high‑risk group had more than double the risk of death or disease progression compared with patients in the low‑risk group. 

    Model performance was evaluated using the concordance index (C-index), which measures how well each biomarker distinguishes between patients with different outcomes. Notably, Path-IO consistently outperformed PD-L1, the U.S. Food and Drug Administration-validated standard-of-care biomarker for guiding immunotherapy use in NSCLC patients, across both discovery and test cohorts.  

    PD-L1 alone showed limited prognostic performance, with C-indices of 0.58 for overall survival (OS) and 0.57 for progression-free survival (PFS) in the discovery cohort, declining to 0.50 and 0.51, respectively, in the test cohort. In contrast, Path-IO demonstrated stronger discriminative ability, achieving C-indices of 0.69 for OS and 0.65 for PFS in the discovery cohort and 0.63 for OS and 0.58 for PFS in the test cohort.  

    Combining pathology-based predictions with radiomics and clinical data further improved the model’s performance, with the C-index increasing from 0.58 to 0.70 for PFS and from 0.63 to 0.75 for OS.  

    Given that the approach was designed to be applied to routine pathology slides, the platform can be incorporated into existing clinical workflows without significant expense compared to other emerging data-based technologies. 

    As the study is retrospective, further investigation is needed to go beyond the identification of patients who would benefit from immunotherapy and help predict what type of immunotherapy they can benefit from. Future directions include prospective validation and the integration of paired, more comprehensive molecular profiling to enhance predictive performance.

    The post AACR 2026: Lung Cancer Immunotherapy Response Predicted by Pathomics AI Model appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Alzheimer’s Linked to Cancer Mutations in Brain Immune Cells https://edusehat.com/en/alzheimers-linked-to-cancer-mutations-in-brain-immune-cells https://edusehat.com/en/alzheimers-linked-to-cancer-mutations-in-brain-immune-cells Wed, 22 Apr 2026 05:15:08 +0700 Edusehat Alzheimer’s, Linked, Cancer, Mutations, Brain, Immune, Cells As the body ages, cells naturally accumulate dozens of genetic mutations each year. New research reported by researchers at Boston Children’s Hospital suggests that the brain’s resident immune cells, microglia, amass mutations in specific cancer-driving genes, yet they don’t manifest as cancer. Instead, these mutations may help drive Alzheimer’s disease.

    The research team, led by Christopher Walsh, MD, PhD, chief of the Division of Genetics and Genomics at Boston Children’s and an investigator of the Howard Hughes Medical Institute, and collaborators Alice Eunjung Lee, PhD, and August Yue Huang, PhD, also in the Division of Genetics and Genomics—who are all professors at Harvard Medical School and associate members of the Broad Institute of MIT and Harvard—say their study findings may provide insights into new Alzheimer’s disease diagnostics and treatments.

    “We find that to some extent, Alzheimer’s disease is a little like cancer—driven by the same mutations that drive blood cancers like lymphoma and leukemia,” said Walsh. “This is helpful because we have a lot of drugs to fight cancer and some of them might be useful therapeutically for Alzheimer’s disease.”

    The researchers reported on their work in Cell, in a paper titled “Somatic cancer variants enriched in Alzheimer’s disease microglia-like cells drive inflammatory and proliferative states.”

    Microglia function as the brain’s resident immune cells, acting as garbage collectors, eating debris and infected or dying cells. “The importance of microglia in Alzheimer’s disease (AD) pathogenesis has been demonstrated by large-scale genetic association studies, which have identified AD risk variants in a growing list of microglia-related genes,” the authors wrote. “Once abnormally reactive in AD, microglia can promote synaptic and neuronal loss while exacerbating tau proteinopathy.”

    Unlike the rest of the immune system cells that circulate in the blood throughout the body, microglia don’t cross the blood brain barrier—or so experts thought. For their newly reported study the research team sequenced 149 cancer-driving genes from tissue samples in 190 brains donated from people with Alzheimer’s disease compared to 121 healthy brains. The Alzheimer’s samples had more single DNA letter changes than the healthy tissue with the most changes found repeatedly in the same five cancer driver genes, meaning the microglia were amassing mutations in specific genes. “Deep (>1,000×) panel sequencing of 311 brain samples revealed enrichment of somatic single-nucleotide variants (sSNVs) in cancer driver genes in AD brains, especially in genes associated with clonal hematopoiesis (CH),” the team stated.

    The cancer gene mutations the researchers discovered in the microglia are commonly found in blood cancers. Because of this, the team tested blood samples from people with Alzheimer’s disease for these same mutations. The team didn’t expect the blood to have these mutations. However, Walsh’s team found the blood cells of the same Alzheimer’s patients carried the same cancer mutations too.

    Microglia-like immune cells with cancer mutations (purple) emerge in the brain. Separately, clumps of proteins, like Tau or amyloid, accumulate in the brain, making the environment hostile. Those microglia cells with mutations get selected for survival and proliferation, creating an inflammatory environment that makes innocent bystander neurons die, contributing to Alzheimer’s disease. [Christopher Walsh and colleagues at Boston Children's Hospital]
    Microglia-like immune cells with cancer mutations (purple) emerge in the brain. Separately, clumps of proteins, like tau or amyloid, accumulate in the brain, making the environment hostile. Those microglia cells with mutations get selected for survival and proliferation, creating an inflammatory environment that makes innocent bystander neurons die, contributing to Alzheimer’s disease. [Christopher Walsh and colleagues at Boston Children’s Hospital]
    “These sSNVs were associated with clonal expansion and carried by both microglia-like brain macrophages (MLBMs) in multiple brain regions as well as paired blood, suggesting a likely hematopoietic origin,” the investigators stated. “It was actually a really unexpected finding that suggests a totally new mechanism for Alzheimer’s disease pathogenesis,” said Huang. “The findings mean that the blood’s immune cells with cancer mutations are likely getting into the brain and contributing to disease.”

    The researchers theorize that the blood-brain barrier weakens, either by age or injury, allowing the blood’s immune cells to cross into the brain. These new arrivals then convert into microglia-like cells. Separately, clumps of proteins accumulate in the brain, triggering microglia to proliferate and respond. The cells most likely to dominate are those with a selective advantage, such as the microglia-like cells with the cancer mutations. However, these mutant microglia also make the environment more inflammatory and hostile than that of the healthy microglia, causing innocent bystander neurons to die off, which leads to Alzheimer’s disease. “These findings suggest that clonal somatic driver variants in MLBMs are enriched in AD, potentially promoting neuroinflammation and neurodegeneration,” the researchers noted. “Potential roles of somatic cancer driver variants in AD pathogenesis open up a whole new range of therapeutic avenues in AD, complementary to approaches emphasizing amyloid and tau.”

    Lee added, “Because it’s hard to access brain tissue in a living patient, genetic screens using blood samples could be developed to test whether a person carries these mutations, and has an increased risk of developing Alzheimer’s disease.” Lee and Huang performed a follow-up study, now posted as a preprint on bioRxiv. Here, they demonstrated that cancer driver mutations observed in patient blood samples increased risk of Alzheimer’s disease independently of a well-established genetic risk factor, APOE4.

    The post Alzheimer’s Linked to Cancer Mutations in Brain Immune Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Navigating with Excellence: The Multi-Faceted Service Lines of Precision Logistics https://edusehat.com/en/navigating-with-excellence-the-multi-faceted-service-lines-of-precision-logistics https://edusehat.com/en/navigating-with-excellence-the-multi-faceted-service-lines-of-precision-logistics Wed, 22 Apr 2026 01:40:10 +0700 Edusehat Navigating, with, Excellence:, The, Multi-Faceted, Service, Lines, Precision, Logistics Navigating with Excellence The Multi-Faceted Service Lines of Precision Logistics eBook coverThe global pharmaceutical industry operates within one of the most demanding and high-stakes environments of our modern world. Unlike traditional retail supply chains, pharmaceutical logistics is a discipline defined by extreme sensitivity, rigorous regulatory oversight, and an unwavering commitment to patient and product safety. It is the pinnacle of expertise on what happens insideand outside a shipment during its journey.

    Marken UPS Healthcare Precision Logistics doesn’t simply move packages. We manage a complexecosystem of specialized service lines designed to maintain the integrity of priority shipping and lifesaving medications from the point of manufacture to the patient’s bedside. Understanding these services is essential for an appreciation of how modern medicine reaches the global population with its efficacy intact.

    Specialty logistics distinguishes itself from general freight through a relentless commitment to technical precision and customized infrastructure. While standard shipping relies on high-volume throughout and routine packaging, specialty logistics demands a bespoke approach to every mile of the journey. This often involves the integration of advanced cold chain innovation to maintain biological integrity or the deployment of specialized technology for visibility of critical materials and equipment in transit.

    Beyond the physical hardware, the sector is defined by a rigorous regulatory landscape where practitioners must navigate a complex web of international compliance, hazardous material protocols, and detailed chain-of-custody requirements. In this environment, good enough is never an option. Every variable, from the precise humidity levels of a storage facility to vibration dampening on a pallet shipper, is carefully monitored.

    This level of granularity ensures that whether a shipment contains a life-saving pharmaceutical batch or a one-of-a-kind special piece of equipment, it arrives not just on time, but in its exact intended state. Consequently, precision logistics serve as the invisible backbone for industries where the cost of failure far exceeds the cost of transport, necessitating a fusion of engineering, legal expertise, and sophisticated data analytics to manage the inherent risks of moving the world’s most challenging cargo.

    Transitioning from the theoretical complexities of the industry to the practical execution of a global supply chain requires an adaptable, expert-led approach. While the challenges of specialized logistics are diverse, ranging from strict thermal requirements to extreme environmental demands, Marken’s operational framework is built on seven distinct pillars of excellence.

    Each of these service lines has been engineered to address a specific facet of the logistical puzzle, providing the specialized equipment, certified personnel, and rigorous oversight necessary to mitigate risk. By categorizing our capabilities into these dedicated sectors, we ensure every project receives a novel strategy rather than a one-sizefits- all solution.

    These service lines represent more than just transportation or clinical trial categories. They are specialized disciplines derived from decades of work in logistics that allow us to maintain a high rate of success for the world’s most sensitive and high-value cargo. In this eBook, Marken experts share how these precision-driven services ensure the performance, reliability, and success of global supply chains.

    The post Navigating with Excellence: The Multi-Faceted Service Lines of Precision Logistics appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AACR 2026: A Video Update from San Diego https://edusehat.com/en/aacr-2026-a-video-update-from-san-diego https://edusehat.com/en/aacr-2026-a-video-update-from-san-diego Tue, 21 Apr 2026 22:05:09 +0700 Edusehat AACR, 2026:, Video, Update, from, San, Diego The American Association for Cancer Research (AACR) meeting is off and running in San Diego. Julianna LeMieux, PhD, Deputy Editor in Chief at GEN, and Damian Doherty, Editor in Chief at Inside Precision Medicine, are on the ground—in the talks, expo hall, and press room, covering as much of the news as they can. Here, they take a moment to chat about the first few days at the meeting.

    The post AACR 2026: A Video Update from San Diego appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     CRISPR Screens Map Human T‑Cell Genes That Promote or Block HIV Infection https://edusehat.com/en/crispr-screens-map-human-tcell-genes-that-promote-or-block-hiv-infection https://edusehat.com/en/crispr-screens-map-human-tcell-genes-that-promote-or-block-hiv-infection Tue, 21 Apr 2026 11:20:11 +0700 Edusehat CRISPR, Screens, Map, Human, T‑Cell, Genes, That, Promote, Block, HIV, Infection How does HIV, armed with only nine genes, manage to hijack the immune system so effectively? For decades, researchers have known that the virus depends on human proteins to enter, replicate, and persist—yet the full roster of those host factors has remained elusive. One major reason: most HIV studies have relied on immortalized cell lines rather than the primary CD4+ T cells the virus actually infects in the body. As a result, scientists have lacked a comprehensive picture of how real human T cells respond when HIV attacks.

    A new study from Gladstone Institutes and the University of California, San Francisco (UCSF), changes that. In the study, titled “Systematic Discovery of Pro- and Anti-HIV Host Factors in Primary Human CD4+ T Cells” and published in Cell, researchers report the first genome‑wide map of human genes that either promote or restrict HIV infection in primary human CD4+ T cells, offering a long‑sought blueprint of the host–virus interface.

    “HIV has been a global crisis for over 40 years,” said Alex Marson, MD, PhD, director of the Gladstone‑UCSF Institute of Genomic Immunology and senior author of the study. “By studying human T cells, which are the primary target of the virus, we’ve finally mapped the genes—many of which were previously unknown—that influence whether or not they can be infected by HIV.”

    Gladstone Institutes
    Scientists in the Marson Lab at Gladstone Institutes have opened a new door to understanding HIV by creating the first genetic roadmap of how the virus interacts with real human cells. [Gladstone Institutes]
    Achieving this required overcoming a fundamental technical barrier. “One challenge of using real human T cells for research is they’re very difficult to infect with HIV; out of a whole dish of cells, typically only one or two percent would get infected,” said first author Ujjwal Rathore, PhD. After years of optimization, the team pushed infection rates to roughly 70%, enabling genome‑scale CRISPR perturbations in primary cells for the first time.

    With that platform in hand, the researchers performed orthogonal genome‑wide CRISPR activation (CRISPRa) and CRISPR knockout (CRISPRn) screens in CD4+ T cells, systematically testing nearly every human gene. Disrupting genes revealed those HIV depends on, while overactivating genes exposed natural antiviral defenses that HIV normally suppresses. “Over‑activating the genes gave us a wealth of information,” said co–first author Eli Dugan, a PhD candidate in Marson’s lab. “We discovered natural antiviral proteins that were previously invisible because the virus could effectively silence them.”

    Across both screens, the team identified hundreds of host factors that shape HIV infection. Among the most striking were two previously unrecognized antiviral proteins: PI16 and PPID (Cyp40). “PI16 interacts with host factors involved in HIV fusion and inhibits viral entry, whereas PPID, a paralog of the proviral cyclophilin CypA, binds capsid and reduces nuclear import of the HIV core,” wrote the authors. Targeted mutagenesis, along with structural modeling and evolutionary analyses, pinpointed residues essential for PPID’s restriction activity, and engineered variants were up to tenfold more potent, according to Dugan.

    To test whether these defenses could counter real‑world viral strains, the team collaborated with HIV pioneer Jay Levy, MD, who provided isolates from the early AIDS epidemic. Elevated levels of PI16 or PPID restricted even these aggressive HIV strains.

    “This was the first genome‑wide effort to show how human genes affect HIV infection in cells taken directly from human blood samples,” said Nevan Krogan, PhD, director of the HIV Accessory and Regulatory Complexes (HARC) Center. “Our findings could eventually lead to new treatments that help the body’s immune system resist the virus.”

    Beyond identifying antiviral factors, the study offers the potential for a powerful new platform for probing HIV latency—the persistent reservoir that evades antiretroviral therapy. “Now, we have the platform to ask the biggest questions in the field,” Rathore said, “and hopefully learn how to eliminate hidden HIV that current drugs can’t reach.”

    The post CRISPR Screens Map Human T‑Cell Genes That Promote or Block HIV Infection appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     CleanAssure Launched as an ISO Class 5 Controlled Cleanroom for Sterile Single-Use Assemblies https://edusehat.com/en/cleanassure-launched-as-an-iso-class-5-controlled-cleanroom-for-sterile-single-use-assemblies https://edusehat.com/en/cleanassure-launched-as-an-iso-class-5-controlled-cleanroom-for-sterile-single-use-assemblies Tue, 21 Apr 2026 11:20:10 +0700 Edusehat CleanAssure, Launched, ISO, Class, Controlled, Cleanroom, for, Sterile, Single-Use, Assemblies Freudenberg Medical, a Kaiserslautern, Germany-based contract design manufacturing partner, launched CleanAssure, a new ISO Class 5 controlled cleanroom designed to deliver clean and sterile single-use assemblies for biopharmaceutical customers, according to the company.

    Freudenberg Medical manufactures silicone and thermoplastic elastomer (TPE) tubing for bioprocessing and critical fluid transfer. The company specializes in seamless, overmolded single-use assemblies used in vaccine production, cell cultivation, fluid transfer, and fill-finish operations.

    The controlled cleanroom enables customers to receive ready-to-use washed, dried, and gamma sterilized single-use assemblies with the highest product quality, sterility, and process consistency, noted Rudi Gall, vp, global pharma, Freudenberg Medical.

    “CleanAssure allows us to support our customers beyond component manufacturing,” he said. “By integrating controlled cleaning and sterilization into our single-use assembly services, we help reduce contamination risk, streamline validation activities, and support a reliable supply for our customers. We can now support customers with their entire component value chain and allow them to focus on their core manufacturing capability.”

    Freudenberg’s cleaning process uses ultrapure water and air, operating within ISO 5 conditions. The water is produced using a multi-stage filtration process, resulting in high-purity water specifically suitable for pharmaceutical applications.

    Key biopharma industry challenges

    Biopharmaceutical manufacturers increasingly rely on single-use systems but face ongoing challenges related to cleaning validation, contamination risk, and production downtime. Customer-managed cleaning processes are often time-intensive, costly, and require additional resources while directly impacting supply reliability, according to Gall.

    The company explained that its controlled cleaning environment addresses these challenges by reducing cross-contamination risk through tightly controlled ISO Class 5 processing; alleviating customer cleaning validation burden by delivering assemblies washed and sterilized under cGMP, validated conditions; minimizing production downtime by removing cleaning as a process step; and supporting a consistent, reliable supply of high-quality single-use assemblies.

    Freudenberg will be attending INTERPHEX New York, April 21–23, Booth 1673, to exhibit its new products and services.

    The post CleanAssure Launched as an ISO Class 5 Controlled Cleanroom for Sterile Single-Use Assemblies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Lilly to Acquire Kelonia for Up to $7B, Expanding Cancer Cell Therapy Pipeline https://edusehat.com/en/lilly-to-acquire-kelonia-for-up-to-7b-expanding-cancer-cell-therapy-pipeline https://edusehat.com/en/lilly-to-acquire-kelonia-for-up-to-7b-expanding-cancer-cell-therapy-pipeline Tue, 21 Apr 2026 04:10:10 +0700 Edusehat Lilly, Acquire, Kelonia, for, 7B, Expanding, Cancer, Cell, Therapy, Pipeline Eli Lilly has agreed to acquire Kelonia Therapeutics for up to $7 billion, the companies said today, in a deal that would bolster the buyer’s oncology pipeline with an early clinical phase lentiviral in vivo chimeric antigen receptor T-cell (CAR T) therapy under study in relapsed/refractory multiple myeloma.

    Kelonia’s lead program KLN-1010 is a one-time intravenous gene therapy designed to generate anti-B-cell maturation antigen (BCMA) CAR T cells, targeting the BCMA protein expressed on the surface of multiple myeloma cells.

    In December at the American Society of Hematology (ASH) 2025 Annual Meeting, Kelonia presented positive early clinical data for KLN-1010 from the Phase I inMMyCARTM trial (NCT07075185). The data showed the CAR T therapy to have 100% minimal residual disease (MRD)-negative response rate across four patients, all of whom remained in response through the longest follow up of five months.

    Those and other results, according to the company, provided initial clinical validation of KLN-1010 and demonstrated promising tolerability. In January, Kelonia won FDA clearance for an investigational new drug (IND) application for KLN-1010, enabling the trial to expand from Australia into multiple clinical sites across the United States.

    “The early clinical data for KLN-1010 are highly encouraging, both as a potential step forward for patients with multiple myeloma and as proof of concept for Kelonia’s platform,” Jacob Van Naarden, executive vice president and president of Lilly Oncology and head of corporate business development, said in a statement.

    Investors appeared more sanguine about the Kelonia acquisition as Lilly shares were all but flat in early Monday trading as of 11 a.m. ET, to $927.16 from Friday’s close of $927.03. Kelonia is privately held.

    KLN-1010 applies the company’s in vivo gene placement system (iGPS®), which uses engineered lentiviral-based particles designed to efficiently and selectively enter T-cells inside the body, enabling a patient’s own body to generate CAR T therapies designed to treat underlying disease.

    Lilly and Kelonia reason that KLN-1010 could transform treatment of multiple myeloma by eliminating challenges associated with both ex vivo patient-specific cell therapy manufacturing, and pre-administration chemotherapy.

    “Autologous CAR T therapies have meaningfully improved outcomes for patients with various cancers, but significant manufacturing, safety, and access barriers mean that only a fraction of eligible patients actually receive them,” Van Naarden added. “Kelonia’s in vivo platform has the potential to change that by delivering rapid, durable responses in a far simpler, off-the-shelf format.”

    Kelonia marks Eli Lilly’s fourth announced acquisition of a smaller biotech this year:

    Behind the deals

    Behind all the deals is the pharma giant’s desire to capitalize on the billions of dollars it is generating from sales of its obesity and diabetes drugs based on glucagon-like peptide 1 (GLP-1) receptor analysts alone or in tandem with a glucose-dependent insulinotropic polypeptide (GIP). Lilly markets tirzepatide, a GLP-1/GIP dual agonist, in obesity as Zepbound® ($13.542 billion in 2025 sales) and in diabetes as Mounjaro® ($22.965 billion).

    Lilly stands to generate even more in obesity-related sales in coming years once it brings to market its oral obesity drug Foundayo™ (orforglipron), a small molecule GLP-1 receptor agonist—though analysts predict the drug’s 2026 sales will likely be lower than once expected because of the price war Foundayo faces competing head to head with Lilly’s arch-rival in obesity drugs, Novo Nordisk. In December, Novo Nordisk got a jump on Lilly when the Danish biotech giant won FDA approval for oral Wegovy® (semaglutide), a once-daily 25 mg GLP-1 receptor agonist tablet indicated for chronic weight management.

    A Lilly buyout of Kelonia could compel Johnson & Johnson to take a closer look at acquiring Legend Biotech, Kostas Biliouris, PhD, a managing director on the biotechnology research team of Oppenheimer, wrote Sunday in a research note. He cited the fact J&J’s Janssen Biotech successfully partnered with Legend to develop Carvykti® (ciltacabtagene autoleucel), a B-cell maturation antigen (BCMA)-directed CAR T-cell therapy indicated for adults with relapsed or refractory multiple myeloma who have received at least one prior line of therapy. Carvykti generated $1.877 billion in sales last year, up nearly double (96%) from $963 million in 2024.

    Also, Biliouris cited the presence in Legend’s pipeline of LUCAR-G39D, a clinical in vivo CAR T program designed to treat B-cell non-Hodgkin’s lymphoma by targeting CD19xCD20. LUCAR-G39D showed positive first-in-human safety and efficacy data from a Phase I trial (NCT06395870) at ASH last December.

    “We believe in vivo CAR T technology has strong potential, as treatment process is fast and circumvents the need for lymphodepletion, but think it will likely take ~6-8years before safety/durability questions are addressed, and regulatory approval is granted,” Biliouris predicted.

    Lilly has agreed to acquire Kelonia for $3.25 billion upfront plus up to $3.75 billion in future payments tied to achieving specified clinical, regulatory, and commercial milestones. The acquisition deal is subject to regulatory approvals and other customary closing conditions, and is expected to close in the second half of 2026.

    Upon closing, Lilly said, it will determine how to account for the transaction in accordance with Generally Accepted Accounting Principles (GAAP), then reflect the deal in future financial results and financial guidance.

    “Kelonia’s leadership in advancing the immense promise of in vivo cell therapy is unmatched, extending its reach and impact beyond the traditional boundaries of personalized medicine,” Kelonia CEO Kevin Friedman, PhD, stated. “We have demonstrated the ability to achieve deep multiple myeloma remissions with significantly reduced complexity and cost relative to ex vivo CAR T-cell approaches.”

    “In combination with Lilly’s strengths, our in vivo iGPS platform is positioned to broaden the reach of cell therapy beyond the current CAR T landscape in hematologic malignancies and to transform treatment across a far wider range of cancers and other serious diseases,” Friedman added.

    The post Lilly to Acquire Kelonia for Up to $7B, Expanding Cancer Cell Therapy Pipeline appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Synthetic Biology and Tissue Engineering Grow Liver Tissue In‑Body https://edusehat.com/en/synthetic-biology-and-tissue-engineering-grow-liver-tissue-inbody https://edusehat.com/en/synthetic-biology-and-tissue-engineering-grow-liver-tissue-inbody Tue, 21 Apr 2026 04:10:08 +0700 Edusehat Synthetic, Biology, and, Tissue, Engineering, Grow, Liver, Tissue, In‑Body Damage to the liver in patients developing end-stage liver disease has become too severe for the organ’s normally extraordinary regenerative capacity to repair or compensate for that damage. Once this point of no return has been reached the only option is an organ transplant. However, donor livers are in high demand and very limited supply.

    Ambitious efforts are on the way that eventually could enable the engineering of entire implantable liver organs. However, the maximum size of laboratory-engineered liver constructs remains limited and cannot yet provide therapeutic benefits for patients. A research team at the Wyss Institute at Harvard University, Boston University, and MIT has now approached this important problem from a different angle.

    “We asked if it would be possible to first implant a small-scale liver construct and then drive it to expand in the body following its engraftment,” said Christopher Chen, MD, PhD, a Wyss Institute core faculty member and the William Fairfield Warren Distinguished professor of biomedical engineering and director of the Biological Design Center at Boston University. “A sufficiently grown, functional ‘satellite liver’ could immediately relieve the metabolic burden in a damaged liver and help bridge the time until a transplant becomes available.”

    Chen co-led the research together with associate faculty member Sangeeta Bhatia, MD, PhD, who is the John J. and Dorothy Wilson Professor of Health Sciences and Technology and of Electrical Engineering and Computer Science at the Koch Institute for Integrative Cancer Research at MIT, and a Howard Hughes Medical Institute investigator. Chen is also a leader of the Wyss Institute’s 3D Organ Engineering Initiative, and team lead of the recently awarded ARPA-H PRINT-supported ImPLANT project, which focuses on whole organ liver engineering at the Wyss and collaborating institutions.

    The project, spearheaded by Amy Stoddard, PhD, (MIT ’25), who developed the approach in her doctoral research and then as a postdoctoral fellow, integrates tissue engineering and synthetic biology tools in a genetic strategy the team has named “bioengineered on-demand outgrowth via synthetic biology triggering,” or BOOST. By specifically rewiring the gene expression of primary liver hepatocytes and supportive fibroblast cells, the scientists were able to effectively switch on a tissue growth program in small, engineered liver constructs after their implantation into mice.

    “Using engineered liver tissue as a proof-of-concept application, we integrated synthetic biology and tissue engineering tools to build liver tissues that can be expanded on-demand after implantation in vivo,” the team reported in their published paper in Science Advances, which is titled “Synthetic control of implanted engineered liver tissue growth.” In the paper they concluded “In this study, we define the first steps toward an unconventional approach to cell therapy scale-up: engineering a small construct and then inducing it to grow in situ … “This strategy, which we have named BOOST, could provide several key advantages, including circumventing the need for large quantities of cellular raw materials and bypassing the formidable challenge of generating a rapidly perfusable construct that can survive the engraftment period.”

    The authors wrote, “Organ transplant is currently the only curative treatment for patients with end-stage organ failure, yet this therapy is inaccessible to many due to the paucity of organs available for transplant.” And while significant progress has been made in the field of engineering tissue-based cell therapies that could represent alternatives, or bridges to transplant, they acknowledge, “… scaling of these constructs to sizes of therapeutic relevance remains a barrier to clinical translation.”

    In order to address current challenges associated with fabrication, Chen and colleagues looked at the problem from different angle, asking whether it would be possible to first implant a small-scale construct and then trigger it to expand in situ, after its engraftment into the host.

    To be able to induce growth of an implanted small liver constructs in situ within a recipient’s body the researchers first needed to identify the relevant cues that would allow them to do so. “A key first step toward this method of in situ scale-up would be the successful control of cellular growth within the engineered construct after engraftment,” they wrote. Since liver growth is known to be regulated by soluble growth factors (GFs), Stoddard screened a collection of candidate factors to identify those that, when added to cultured human primary hepatocyte cells (HEPs), had the strongest growth-inducing effects.

    The genetic “BOOST” strategy integrates tissue engineering and synthetic biology tools to enable on-demand liver growth inside the body. By specifically rewiring the gene expression of primary liver hepatocytes and supportive fibroblast cells, a tissue growth program is switched on in a small, engineered liver construct after its implantation into recipients and upon addition of an inducing agent (shown as a pill). As a result, the hepatocytes in the construct start and continue to proliferate until a desired construct size has been reached and the inducing signal is not provided anymore. In mice, BOOST resulted in robust and healthy liver growth. [Wyss Institute at Harvard University]
    The genetic “BOOST” strategy integrates tissue engineering and synthetic biology tools to enable on-demand liver growth inside the body. By specifically rewiring the gene expression of primary liver hepatocytes and supportive fibroblast cells, a tissue growth program is switched on in a small, engineered liver construct after its implantation into recipients and upon addition of an inducing agent (shown as a pill). As a result, the hepatocytes in the construct start and continue to proliferate until a desired construct size has been reached and the inducing signal is not provided anymore. In mice, BOOST resulted in robust and healthy liver growth. [Wyss Institute at Harvard University]
    “We ended up with a set of four growth factors, HGF, TGFa, WNT2 and RSPO3, that potently induced sparsely scattered HEPs to grow in the culture dish,” said Stoddard. “But when we tested whether they could do the same in 3D liver tissues consisting of densely packed HEPs and fibroblasts, they turned out to be ineffective. This led us to hypothesize that there must be an additional mechanism at work in human HEPs that inhibits cell proliferation in high-density conditions.”

    The team homed in on a protein, YAP, that senses mechanical signals, and which was known to move from cells’ cytosol to their nucleus in low-density conditions to help express genes involved in cell proliferation. However, in high-density conditions when cells are compressed, YAP is degraded in the cytosol, which prevents the activation of those target genes and restricts proliferation.

    “Importantly, when we overexpressed a non-degradable version of YAP in HEPs, which also reaches the nucleus in high-density conditions to partake in gene regulation, we successfully overrode this density checkpoint in HEPs,” Stoddard said. “Interestingly, we found that HEPs needed to be stimulated with both YAP and GFs in order to grow in densely packed 3D liver tissues.”

    Toward the goal of safely inducing and controlling HEP proliferation in a living organism, and eventually human patients, the researchers deployed synthetic biology tools to locally install control of these signaling pathways in HEPs and fibroblast cells within the engineered 3D liver tissues themselves. “We set out to engineer a synthetic biology toolkit capable of locally modulating growth factor and YAP signaling within engineered liver tissue, enabling on-demand control of proliferation even after implantation,” they noted.

    The team engineered fibroblast cell lines that each secreted one of the four GFs, and HEPs that expressed the non-degradable YAP protein. And they made the expression of all proteins doxycycline (DOX)-inducible. They determined in time course experiments that a continuous seven-day treatment with DOX led 3D liver tissue composed of engineered cells to robustly expand in size and cell numbers in the culture dish. On DOX removal the HEPs reverted back to a non-proliferating state.

    However, Stoddard noted, “… when we compared the gene expression of single cells in BOOST-engineered, DOX-induced 3D liver tissue to that of cells in non-engineered or BOOST-engineered, non-induced 3D liver tissue, we noticed that the expansion came with a trade-off: high proliferation rates went hand in hand with a less functional HEP state. While we believe this is a natural trade-off seen in a wide variety of biological settings, we hope to be able to address this in the future, recognizing that the liver also has native re-functionalization signals to harness.”

    The litmus test for BOOST-engineered growth in 3D liver tissues was to see whether they would similarly expand following their implantation into living mice that were systemically treated with DOX for the same seven-day duration. Experiments showed that the implanted tissue exhibited a striking 500% increase in proliferation with a doubling of the engineered HEPs alone, and was vascularized to accommodate the metabolic demands of the expanded tissue. The tissue implants were also well tolerated by the mice, with no signs of fibrosis due to invading immune cells and fibroblast inflammation, or of tumor growth.

    “These results were particularly exciting to us,” said Stoddard. “Prior to our work, injury to the host liver has always been required to trigger hepatocyte engraftment and proliferation. Here we were able to relieve this dependence, and trigger on-demand growth of implanted liver tissue in a completely healthy host.”

    In the future, the team will explore the capacity of BOOSTed liver tissue to rescue the host in the setting of liver injury. “Our BOOST strategy lays the foundation for a future when solid organ cell therapies can be controlled non-surgically according to the needs of patients and their physicians,” Bhatia noted. “Beyond treating liver disease, the premise of BOOST could be applied to other engineered tissue therapeutics that are similarly constrained by challenges associated with tissue scale-up, such as engineered heart or pancreatic tissue to address serious diseases.”

    In their paper the authors concluded, “… this work serves as an exciting proof-of-concept demonstration that scale-up of tissues via growth could be possible … Together, this work helps lay the foundations for a future of ‘smart’ tissue therapeutics that can be scaled to a patient’s needs and thereby offer treatment for numerous, previously incurable, diseases.”

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    ]]>     AACR 2026: Cancers of Unknown Primary Identified by DNA Methylation AI Model https://edusehat.com/en/aacr-2026-cancers-of-unknown-primary-identified-by-dna-methylation-ai-model https://edusehat.com/en/aacr-2026-cancers-of-unknown-primary-identified-by-dna-methylation-ai-model Tue, 21 Apr 2026 00:40:04 +0700 Edusehat AACR, 2026:, Cancers, Unknown, Primary, Identified, DNA, Methylation, Model SAN DIEGO – Researchers from Kindai University in Japan have developed a machine learning model that accurately predicts the origin of diverse cancer types in patients with cancers of unknown primary (CUP) by analyzing CpG-based DNA methylation. Results showed that the model correctly identified the cancer type in about 95% of cases in the test cohort, and achieved 87% accuracy when applied to an independent validation cohort from 31 cases representing 17 different cancer types. The work was presented at the American Association for Cancer Research (AACR) Annual Meeting.  

    “Our findings suggest that DNA-based approaches can help identify where a cancer may have started, even when the original tumor is not visible,” said Marco A. De Velasco, PhD, a faculty member in the department of genome biology at Kindai University in Japan.  

    CUP are metastatic malignancies in which the primary cancer site could not be identified. These cancers are often associated with poorer outcomes, as patients are typically treated with broad, nonspecific chemotherapy regimens rather than therapies targeted to a specific cancer type. 

    Approximately only 15-20% of patients with CUP show features that allow site-specific therapies. Patients receiving site-directed therapy can survive up to 24 months, compared with six to nine months for those receiving standard treatment. 

    Patterns in tumor biology, such as gene activity or chemical modifications to DNA, can differ between cancer types and persist even after the cancer has spread and guide development of these therapies. While some methods have shown promise, they have yet to demonstrate clear survival benefits in clinical trials. 

    The model was developed using methylation data from nearly 7,500 patients with 21 different cancer types obtained from The Cancer Genome Atlas Program and other public datasets. Using machine learning, the researchers identified CpG methylation and built methylation profiles that were associated with different tumor types. 

    Del Velasco emphasized that the study achieved high accuracy in predicting the origin of diverse cancer types using a small subset of DNA markers, about 1,000 CpG regions selected from hundreds of thousands across the genome. “This is important because it shows that we can simplify complex molecular data while still maintaining strong predictive performance,” he said. 

    As a limitation, the model was developed using cancers with known origins, rather than true CUP. Testing in CUP patients is important to understand how well the model performs in clinical settings. Additionally, not all tumors are easily accessible for genetic testing, particularly tumors in advanced stage. Looking ahead, the authors aim to adapt and evaluate the model using blood-based biopsy to analyze circulating tumor DNA instead of relying on DNA from tissue samples. 

    The post AACR 2026: Cancers of Unknown Primary Identified by DNA Methylation AI Model appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Colossal Biosciences said it cloned red wolves. Is it for real? https://edusehat.com/en/colossal-biosciences-said-it-cloned-red-wolves-is-it-for-real https://edusehat.com/en/colossal-biosciences-said-it-cloned-red-wolves-is-it-for-real Mon, 20 Apr 2026 21:05:03 +0700 Edusehat Colossal, Biosciences, said, cloned, red, wolves., for, real If you want to capture something wolflike, it’s best to embark before dawn.

    So on a morning this January, with the eastern horizon still pink-hued, I drove with two young scientists into a blanket of fog. Forty miles to the west, the industrial sprawl of Houston spawned a golden glow. Tanner Broussard’s old Toyota Tacoma bumped over the levee-top roads as killdeer, flushed from their rest, flew across the beams of his headlights. 

    Broussard peered into the darkness, looking for traps. “I have one over here,” he said, slowing slightly. A master’s student at McNeese State University, he was quiet and contemplative, his bearded face half-hidden under a black ball cap. “Nothing on it,” he said, blandly. The truck rolled on.

    Wolves and their relations—dogs, jackals, coyotes, and so on—are classed in the family Canidae, and the canid that dominated this landscape in eastern Texas was once the red wolf. But as soon as white settlers arrived on the continent, Canis rufus found itself under siege. The war on wolves “lasted 200 years,” federal researchers once put it, in a surprisingly evocative report. “The wolf lost.” By 1980, the red wolf was declared extinct in the wild, its population reduced to a small captive breeding population.

    Still, for decades afterward, people noted that strange wolflike creatures persisted along the Gulf Coast. Finally, in 2018, scientists confirmed that some local coyotes were more than coyotes: They were taller, long-legged, their coats shaded with hints of cinnamon. These animals contained relict red wolf genes. They became known as the ghost wolves.

    Broussard grew up in southwest Louisiana, watching coyotes trot across his parents’ ranch. The thrilling fact that these might have been not just coyotes but something more? That reset a rambling academic career. In 2023, Broussard had recently returned to college after a seven-year pause, and his budding obsession with wolves narrowed his focus. Before he finished his bachelor’s degree, he began to supply field data to a prominent conservation nonprofit.

    a wolf pup chews on a terrycloth toy
    The American red wolf, Canis rufus, is the most endangered wolf species in the world. This pup is one of four animals said to be clones of this native North American species.
    COURTESY OF COLOSSAL BIOSCIENCES

    Then, last year, just before he began his master’s studies, he woke to disconcerting news. A startup called Colossal Biosciences claimed to have resuscitated the dire wolf, a large canid that went extinct more than 10,000 years ago. Pundits debated the utility of the project and whether the clones—technically, gray wolves with some genetic tweaks—could really be called dire wolves. But what mattered to Broussard was Colossal’s simultaneous announcement that it had cloned four red wolves.  

    “That surprised pretty much everybody in the wolf community,” Broussard said as we toured the wildlife refuge where he’d set his traps. The Association of Zoos and Aquariums runs a program that sustains red wolves through captive breeding; its leadership had no idea a cloning project was underway. Nor did ecologist Joey Hinton, one of Broussard’s advisors, who had trapped the canids Colossal used to source the DNA for its clones. Some of Hinton’s former partners were collaborating with the company, but he didn’t know that clones were on the table.

    There was already disagreement among scientists about the entire idea of de-extinction. Now Colossal had made these mystery clones, whose location was kept secret. Even the purpose of the clones was murky to some scientists; just how they might restore red wolf populations was unclear. 

    Red wolves had always been a contentious species, hard for scientists to pin down. The red wolf research community was already marked by the inevitable interpersonal tensions of a small and passionate group. Now Colossal’s clones became one more lightning rod. Perhaps the most curious question, though, was whether the company had cloned red wolves at all. 

    You can think of the red wolf as the wolf of the East—an apex predator that once roamed the forests and grasslands and marshes everywhere from Texas to Illinois to New York. Smaller than a gray wolf (though a good bit larger than a coyote), this was a sleek beast, with, according to one old field guide, a “cunning fox-like appearance”: long body, long legs; clearly built to run across long distances. Its coat was smooth and flat and came in many colors: a reddish tone that comes out in the right light, yes, but also, despite the name, white and gray and, in certain regions and populations, an ominous all black.

    We know these details thanks to a few notes from early naturalists. As writer Andrew Moore writes in his new book, The Beasts of the East, by the time a mammalogist decided to class these eastern wolves as a standalone species in the 1930s, the red wolf had been extirpated from the East Coast and was rapidly dwindling across its range. Working with remnant skulls and other specimens, the mammalogist chose the name red wolf—which was later enshrined with the Latinate Canis rufus—because that’s what these wolves were called in the last place they survived. 

    The looming extinction of the red wolf turned out to be a good thing for coyotes. Canis latrans is a distant relative of wolves that split away from a common ancestor thousands of years ago and might be considered, as one canid biologist put it to me, the “wolf of the Anthropocene.” Their smaller size means they need less food and can survive in smaller and more fragmented territory, the kind that modern humans tend to build. 

    The last red wolves, which lived in Louisiana and Texas, decided a strange and smaller mate was preferable to no mate at all.

    Red wolves had kept coyotes out of eastern America, outcompeting them for prey. Now, as the wolves declined, the coyotes began to slip in. The last red wolves, which lived in Louisiana and Texas, decided a strange and smaller mate was preferable to no mate at all. Soon the territory became a genetic jumble, home to both wolves and coyotes and hybrids that, after several generations of intermixing, came in every shade between. Scientists call such a population a “hybrid swarm,” and it poses a genetic threat to the declining species: As more coyotes poured east, and as all the canids kept interbreeding, there would be nothing that was “purely” wolf. 

    ""
    Ron Wooten surveys a location on the edge of Galveston Island State Park in Texas. In 2016, Wooten’s photographs of oversized local coyotes got the attention of Joey Hinton, then a postdoctoral researcher at the University of Georgia.
    TRISTAN SPINSKI

    For years, no one seemed to notice. Perhaps trappers in the region mistook the new hybrids for wolves—or were happy to take the higher bounty that a wolf pelt earned. Finally, though, by the 1960s, as the concept of endangered species first emerged, biologists began to worry for the disappearing wolf. 

    The best solution they could come up with was a program of mass extermination. Over several years, trappers rounded up hundreds of canids in Texas and Louisiana. Those deemed true red wolves (on the basis of their howls and skull shape) were whisked away to breed in captivity. Most of the rest were euthanized. In 1980, the red wolf was declared extinct in the wild. To put it plainly: The red wolf was wiped out intentionally, in a roundabout effort to keep it alive.

    Just 14 individuals survived this gauntlet; today’s wolves descend from 12 of those. They became the ark, the source material for the few hundred red wolves that live today. There are about 280 in the “Species Survival Plan” population, living in captivity, and another 30 or so that roam a federal refuge in coastal North Carolina, and that the government deems “nonessential” and “experimental.” According to the US Fish and Wildlife Service, to be classified as a representative of the protected entity known as Canis rufus, an animal must trace at least 87.5% of its lineage to the 12 founders. 

    The scientist who led this trapping-and-breeding program understood that the federal government would be narrowing the red wolf’s gene pool precipitously—so much so that the result could be an entirely new species. None of those notably black wolves persisted in the new population, for example. But what other choice existed? A new kind of wolf, free of the taint of the invading coyote, seemed better than no wolf at all.

    After I learned about Colossal’s clones, I decided to travel to eastern Texas. The clones were hidden away on an unnamed refuge, but on this coastline, I might be able to at least see the animals that provided their genetic material. I arrived in the small town of Winnie on a balmy afternoon in January and met up with Broussard and another graduate student, Patrick Cunningham, at a Tex-Mex joint to discuss the challenges of studying red wolves.

    “We don’t have a good reference genome,” Cunningham said. We can collect DNA from the descendants of the 12 founders, but not from the countless wolves that had been killed. It’s difficult to extract usable DNA from old samples. So our picture of what the species used to look like is limited. 

    Studies of the genes we do have, meanwhile, have proved controversial. When a Princeton geneticist named Bridgett vonHoldt dug into the genome of the Species Survival Plan population, she found little about their DNA that could set them apart from other wolflike American canids. In 2016, in a paper in Science Advances, vonHoldt and her coauthors wondered if there ever really was a separate southern wolf species. Perhaps the 12 founders were just coyotes injected with some smaller portion of wolf.

    It’s long been clear that North America’s soup of Canis genes is something less like a family tree and more like a river—one that’s broken by islands and sandbars into many braided channels that split and merge and re-split.

    Her paper called for complex new interpretations of the Endangered Species Act. We should, she wrote, focus less on species and more on the function a group of animals performs. The red wolves deserved protection, then, as creatures that filled the same role as truly endangered wolves and carried some of their genetics. Nonetheless, for Canis rufus, the timing of the paper was bad news.

    The red wolves roaming that federal reserve in North Carolina are supposed to be a first step toward the species’ return to the wild. But some locals never liked the idea of living alongside wolves. By 2016, state officials had turned against the recovery program and were requesting its termination. The wild population, which had included as many as 120 a few years earlier, was falling. But the US Fish and Wildlife Service had paused further releases of wolves. Now a group of scientists, led by vonHoldt, was saying that the red wolf showed “a lack of unique ancestry.” Why spend money, some people wondered, on a species that does not exist? 

    Part of the problem was that the concept of a “species” is less sturdy than your high school biology teacher might have led you to believe. The most familiar definition is that a species consists of animals that can produce fertile offspring. But that’s a rule various species of canids violate all the time; it’s long been clear that North America’s soup of Canis genes is something less like a family tree and more like a river—one that’s broken by islands and sandbars into many braided channels that split and merge and re-split.

    VonHoldt suggested that the modern red wolf is a channel in that river, part wolf and part coyote, that appeared surprisingly recently. But a year after her study came out, other researchers claimed that her data, if interpreted differently, could suggest that the red wolf braid had emerged tens of thousands of years ago, meaning this was a species that had long been on its own evolutionary journey. 

    These nuances were confusing for the policymakers who oversaw actual, living animals. “Congress was just like, ‘What is going on?’” Cunningham said. “‘Why is there not just a simple explanation for what this thing is?’”

    Given the policy implications, the National Academies of Science, Engineering, and Medicine tasked a panel of scientists with finding that simple answer. Their report, published in 2019, declared that the red wolf is, by virtue of its appearance and seemingly long-standing isolated population, a species. As their study got underway, though, a new question was arising: What to make of the strange canids on the Gulf Coast, those today called the ghost wolves?

    The path to that name began in 2008, when a photographer from Galveston Island, Texas, grew obsessed with the oversized local coyotes. He began to take photos of the packs, which he distributed to scientists, seeking answers: What were they? By 2016, the photos had reached Joey Hinton, then a postdoctoral researcher at the University of Georgia.

    Hinton had spent more than a decade trapping wolves and coyotes in North Carolina, and his work has always focused on live animals, especially visual ways to distinguish red wolves and coyotes. So he was a good choice for helping the photographer, Ron Wooten, figure out the status of the canids. In his freezer Wooten also had tissue samples he’d collected from road-killed coyotes. These could be used by a geneticist to give a fuller picture of the canids’ ancestry. So vonHoldt was brought in too. The result was a 2018 paper, with Hinton as a coauthor, that identified the Galveston Island canids as at least part red wolf.

    These canids were not, to be clear, actual red wolves; no canid on the Gulf Coast is descended from the government’s 12 canonical founders, so under current policy, none can be officially classified as a wolf. Subsequent studies have found that, on average, the ancestry of the region’s canids is less than half red wolf, and often far less. In scientific terms, the red wolf had introgressed into the Gulf Coast population—its genes had leaked across the species boundary and lodged themselves in a different population.

    Hinton, vonHoldt, and their coauthors also noted the presence of what they called “ghost alleles”—DNA sequences unknown in any other named species. The Occam’s razor assumption was that, in these already wolfy coyotes, these sequences likely represented Canis rufus genetics that had not been captured in the sweep of the marsh that yielded the Species Survival Plan population. Since so much of the red wolf gene pool had been lost, these genes seemed to be a potential resource for the species—a way to expand its diversity. When the New York Times covered this discovery a few years later, the headline popularized the “ghost wolf” moniker that has proved so indelible. 

    As it happened, a separate team, focused on canids in and around federally protected marsh in Louisiana, published a similar paper in 2018, at nearly the same time. The twin discoveries raised new questions—What should we make of these creatures, the latest branch in the canid river? What do they mean for the wolves in North Carolina?—and helped researchers secure new funding.

    In 2020, vonHoldt and Kristin Brzeski, a former postdoc under vonHoldt and now a professor at Michigan Technological University, launched what they called the Gulf Coast Canine Project. Brzeski, who led the field work, hired Hinton to do much of the canid trapping and sample collection. In 2022, vonHoldt, Hinton, and Brzeski were all coauthors of another paper that identified even more red-wolf-descended canids in Louisiana and noted a positive correlation between red wolf ancestry and body mass—the more red wolf genes, the bigger the animal. The paper also suggested that given this newly discovered reservoir of red wolf DNA, “genomic technologies” could prove useful in the long-term survival of the species.

    ""
    Bridgett vonHoldt (left) and Kristin Brzeski (center) visit a location where canids have been spotted with an animal control worker.
    TRISTAN SPINSKI

    VonHoldt and Brzeski eventually conceived of an ambitious project. They hoped that by carefully matching the most wolf-­descended canids and breeding them together, over three generations they’d increase the proportion of red wolf genes—de-introgression. “I’m expecting, based on these pairings of animals, that I can stitch together the puzzle pieces,” vonHoldt told me recently. “We are very likely to get puppies each generation that are higher and higher red wolf content”—enough wolf content, she hopes, to eventually win her permission to breed the resulting animals with the Species Survival Plan population of red wolves. They’d essentially be adding a new founder to the limited lineage.

    Hinton told me he felt he’d been kept in the dark about the de-introgression idea. He was also worried, he says, to learn that Colossal Biosciences hovered in the background. (In a draft proposal for the project, vonHoldt indicated that Colossal would be in charge of “live capture.”) Hinton says he was not comfortable collecting materials for a for-profit company that has to keep its shareholders happy. 

    Hinton says he reached out to state and federal officials and found they knew little about the project. (The US Fish and Wildlife Service declined to make anyone available for an interview for this story, and the Louisiana Department of Wildlife and Fisheries did not reply to requests for comment.) He knew the group’s next phone call would be difficult, and indeed it was. He wound up speaking one-on-one with vonHoldt for at least half an hour.

    “We didn’t reach an agreement,” he says. After the call, he sent her a text: He was exiting the project. He believes that had Colossal not been involved, they’d all still be working as a team. Both vonHoldt and Brzeski declined to comment on what felt to them like a matter of interpersonal relationships rather than a scientific dispute. “There were challenges over time, and the tone and manner of the interactions became increasingly difficult to navigate productively,” Brzeski said in an email. 

    Colossal was cofounded in 2021 by George Church, an eminent Harvard geneticist who, thanks to investors, could finally embark on a long-discussed dream. He wanted to make de-extinction a reality—using CRISPR gene-editing technology to, say, turn a modern elephant into something like the extinct woolly mammoth. The concept has drawn skepticism from the beginning—at best it would only be possible to make something like a woolly mammoth. Was there any point to that? Some scientists note that genes alone do not teach an animal how to exist in the world; indeed, since social structures affect how genes are expressed, an animal without parents may not effectively fill its ecological niche.

    Less reproachable, though, was Colossal’s interest in partnering with scientists who, like vonHoldt and Brzeski, focus on extant species that are endangered. This gave more heft to Colossal’s gee-whiz de-extinction projects: They would, along the way, supply technology that could save our natural world.

    For red wolves, such technologies could offer a quick way to expand the limited gene pool. Through genetic engineering, Colossal could take clones of the Gulf Coast canids and tune up the wolf, tune down the coyote. It would be a high-tech shortcut past vonHoldt and Brzeski’s careful breeding program. “You can do the same thing much more precisely, much more quickly, much more efficiently, in vitro,” says Matt James, Colossal’s chief animal officer and the executive director of the Colossal Foundation, the company’s nonprofit arm. VonHoldt notes that the old-fashioned approach, with breeding, means she has to take a few individual canids out of the wild, into captivity—never ideal but, in her view, a worthwhile price for progress. The advantage of cloning, which Colossal has managed to do with blood samples alone, is that the wild canid populations can be kept intact. 

    VonHoldt has always been an advocate for wolves. Indeed, when she hypothesized that the red wolf had hybrid origins, in 2016, she’d framed it as an argument for protecting the gray wolf, which the federal government was considering removing from the Endangered Species List. (In short: If all wolves were one wolf, then it was undeniable that the species’ range had contracted precipitously.) But she’d grown frustrated with the federal government’s efforts to restore the red wolf, which after half a century had seen few meaningful successes, she says. 

    VonHoldt joined Colossal’s scientific advisory board in 2023. “I love the bold, the shock and awe,” she told me, explaining her decision. She saw the fact that Colossal sparked controversy as an asset, given the problems she sees in conservation: “Get something out there. Start pushing buttons and start forcing these conversations,” she says. The red wolf was akin to a terminal patient who was ready to accept any and all therapies, however experimental. Why not embrace biotech? 

    She also notes that the federal budget for endangered species conservation is incredibly limited. Rely only on that money and “we can kiss our world goodbye,” she said in an e-mail. The $100 million raised by the Colossal Foundation is essential, then, she says. As for the samples the team had collected on the Gulf Coast, she says, limited freezer space is often devoted to animals that are officially categorized as threatened or endangered, which the Gulf Coast canids are not. Colossal could take the samples, and the team passed them along to the company.

    Dr. Joey Hinton
    Ecologist Joey Hinton trapped the canids that Colossal Biosciences used to source the DNA for its clones. He dismisses the clones as a way for the company to earn headlines and attract funding.
    RICH SAAL

    It was Hinton—a source for a former story—who first alerted me to Colossal’s work on red wolves; he described vonHoldt and Brzeski’s de-introgression project, which won federal funding in late 2024, as nefarious-sounding work to “disappear” canids off the Gulf Coast. But he did not have all the details of the project, which had changed after he left the team. He suggested they’d be “just throwing animals together,” whereas vonHoldt described a careful program of observing the canids in the wild so she could determine which acted most wolflike, findings she’d cross-­reference with their genetic data.

     Colossal did not wind up participating in the de-­introgression project. But the company is doing work on the red wolf that ­vonHoldt views as complementary: Its scientists are assembling a “pangenome” of North American canids by studying samples pulled from museums, universities, zoos, and other institutions. This data set is expected to clarify both what genetic sequences are shared across the entire canid family and what snippets differ in certain populations. The hope is that this will provide a clearer picture of the red wolf in its early days, before the coyotes arrived and the gene pool narrowed. That might shift what Colossal’s James calls the government’s arbitrary definition of the red wolf, to encompass more of the species’ full former diversity. 

    The pangenome, then, might allow vonHoldt’s de-­introgressed canids, descended from the Gulf coast canids, to qualify as actual red wolves. Indeed, James suggested to me that more information about historic red wolves might force the government to take a new look at the Gulf Coast canids; some individuals might have high enough red wolf ancestry to be classified as red wolves. (“That has management implications that terrify state and federal government,” he added.)

    hair in Zip-Loc bags on a metal tray
    Blood and tissue samples collected by the Galveston Island Humane Society from canid roadkill will be shipped to Princeton University for DNA analysis.
    TRISTAN SPINSKI

    The purpose of vonHoldt’s de-introgression project is to bring back certain lost red wolf genes—to create a whole new wolf lineage. But she has also pushed against the idea of “genetic purity,” which she thinks limits what we protect with conservation laws; she told me emphasizing it reminds her of the human history of eugenics and “makes every part of my soul hurt.” She cares less about what species are out there, in the landscape, than what ecological function the animals play, and she sees coyotes and red wolves as closely related animals that may have a role to play in one another’s future survival.

    As for Colossal’s clones, even vonHoldt seems to describe them as something less than a conservation breakthrough. They are a “proof of principle that we, collectively, as a scientific community, know how to do it,” she told me. If an urgent need arises to clone red wolves, the groundwork has been laid. 

    Hinton, meanwhile, is one of several scientists I spoke with who were skeptical Colossal was doing good science, given that so much is conducted behind closed doors. He implied that the clones were nothing but an empty showpiece, a way to earn headlines and attract funders. “The work is anything but symbolic,” James responded via e-mail. “It expands the genetic toolkit available for critically endangered species, demonstrates scalable approaches to biodiversity restoration, and contributes directly to preserving imperiled lineages.” He noted that Colossal had intentionally decided to avoid the “snail’s pace” of the peer review process and suggested that the skepticism from scientists may actually be a “panicked response to being outpaced.”

    Until some evidence confirms that the Gulf Coast canids—the source material for the clones—are red wolves, they can’t legally be classified as such for federal conservation purposes. Nonetheless, Colossal’s press release claimed that the company had “birthed two litters of cloned red wolves, the most critically endangered wolf in the world.” On the same day that press release dropped, Colossal’s CEO and cofounder, Ben Lamm, appeared on The Joe Rogan Experience and claimed that he had offered to create hundreds of red wolves for the federal government to use in recovery—for free! He was miffed when the government, under the Biden administration, replied that it wanted to spend several years and many millions of dollars to study the potential for cloning before it would take any action. (The company has gotten more traction with the Trump administration, Lamm said.)

    When I first spoke to James at Colossal, he said that he was “cognizant” of the concerns over the names and labels and that the company’s own materials described the clones as “red ‘ghost’ wolves.” He suggested that if anyone assumed the clones were actual red wolves, that was because journalists had failed to grasp the nuances of the science. But this phrase appears so late in a long document that it was cut off in some versions. Later, over email, James indicated that further analysis had convinced him that what the company had created were red wolves, and that anyone who disagreed either could not grasp the science or is “so ideologically opposed to Colossal’s conservation revolution that they are willing to compromise their scientific integrity.”

    VonHoldt has had her own issues with the company’s communications; she told me it was “stressful” when Lamm described the clones as red wolves—which, she notes, “federally, they’re not.” But she values the company’s work, she says, and “the thing that I value the most is shaking things up.” People are paying attention to red wolves. If it’s hard to decide what to call the animals on the Gulf Coast—where some heavily wolfy animals live alongside others that are more coyote—that’s just proof that our concept of a “species” does not capture the complex realities on the ground. 

    In 2025, the same year as Colossal’s wolf announcement, Hinton launched the Texas-Louisiana Canid Project. He’s working in partnership with Broussard, the master’s student at McNeese, in slightly different territory from vonHoldt and Brzeski—and focusing more on the animals’ appearance and behavior than their genes. The Gulf Coast canids are stable and faring better than the North Carolina red wolves, and his hope is that if we learn why they’ve been successful for so many years, we might be able to help the official red wolf population, which is only just limping along. 

    a wolf crosses a road outside of the city
    Galveston locals hope that the presence of these remarkable creatures—red wolves or not—might rein in the rapid development of the island’s last stands of green.
    TRISTAN SPINSKI

    I had planned to join Hinton in the field, but by the time I was able to visit, he’d had to go home to his family. So I joined Broussard on his last days trapping in Texas that season. Before I’d left for Winnie, I’d told my friends I’d be out chasing the last surviving red wolves. But there, on the Gulf Coast, I came to understand that this was just as much a story about coyotes.

    That’s what Broussard and Cunningham both called the creatures. Hinton does too; he considers the animals to be a specific “ecotype” of coyote, featuring an injection of wolf DNA that has helped them adapt to the local marshes. 

    At vonHoldt’s behest, I drove an hour down the coast to Galveston Island, where she and Brzeski began working with the island’s animal control department; when locals find a coyote, the animal is captured so its blood can be collected and a GPS collar fitted on its neck. A small group of locals who support the project have come to call themselves the “ghost wolf team.” They hoped that the presence of these remarkable creatures might rein in the rapid development of the island’s last stands of green. Still, the people I spoke to in Galveston conceded that the animals were, if special, nonetheless a form of coyote. 

    VonHoldt describes Galveston Island as a potential model for what conservation could look like in the future. Top-down recovery hasn’t been working, but helping more places fall in love with their local animals might. And for that to happen, we need to stop obsessing over whether or not something is a “pure” wolf. What matters, she argues, is that an animal is doing what a larger predator does in an ecosystem. She embraces the “ghost wolf” name because, more than “Gulf Coast canid,” it makes clear that there’s something special on the coast—something worth protecting. 

    Her vision is enticing: Focus on function over purity. Let evolution proceed. Stop protecting the wolf of the past and consider the wolf of the future. Such rapid genetic exchange may be necessary to help predators adapt to a hotter, increasingly shattered world, she says. 

    If we throw out the concept of “endangered species,” will we really protect “endangered functions” instead?

    Then again, we already know what’s adapted to the world we’re building: coyotes. The argument against genetic purity can sound like giving up on wolves entirely, with the possible exception of whatever specimens we produce in cloning facilities. And there is the matter of politics: If we throw out the concept of “endangered species,” will we really protect “endangered functions” instead? Under an administration already rolling back environmental protections, the likeliest outcome may be protecting nothing at all.

    I tried in Galveston, too, to see the coyotes. Ron Wooten, the local resident who helped alert scientists to this population, dropped some pins on a map, pointing me toward several likely spots. That evening, after the sun set, I chose a quiet road that passed through marshes until it reached the island’s eastern beach. It was mating season, Wooten had noted. The animals should be on the move, he said; look to the bushes. As I drove up and down the road, my headlights revealed only empty darkness. No coyote. No wolf. Fitting, perhaps—isn’t absence the essence of a ghost? But whether this was a good omen was less clear. As individuals, these animals do best by avoiding us humans. As a group, their survival—like the survival of the red wolves—depends on our knowing that they are here, and were here, and deciding that is reason enough to care.

    In Winnie the next morning, I went out one last time with Broussard, and we struck out again. With no coyotes in his traps and the new semester looming, he decided to take down his game cameras. Back at the hotel, I caught at least an image of what I’d been chasing: In black and white, the animals were appropriately silver, spectral, dashing across the midnight fields. In one clip, a canid paused and howled. “That’s super cool,” Broussard said quietly, as an echoing, interweaving chorus responded from somewhere deeper in the marsh. 

    Boyce Upholt is a journalist based in New Orleans and founding editor of Southlands, a magazine about Southern nature. 

    ]]>     Decoding Resistance to Targeted Therapy via New Cancer Models https://edusehat.com/en/decoding-resistance-to-targeted-therapy-via-new-cancer-models https://edusehat.com/en/decoding-resistance-to-targeted-therapy-via-new-cancer-models Mon, 20 Apr 2026 21:00:11 +0700 Edusehat Decoding, Resistance, Targeted, Therapy, via, New, Cancer, Models ATCC and the Broad Institute report the development of engineered isogenic cancer models designed to replicate resistance to targeted therapies, beginning with osimertinib, the latest-generation epidermal growth factor receptor (EGFR) inhibitor used to treat non-small cell lung cancer (NSCLC) with EGFR mutations.

    According to the researchers, the work addresses a critical challenge in oncology—treatment resistance that emerges over time. EGFR-mutant lung cancer was among the first subsets of a major epithelial cancer where directly targeting an oncogene was associated with marked clinical benefit. While targeted therapies have significantly improved overall survival, resistance inevitably develops.

    cancer drug resistance
    Understanding resistance mechanisms is essential for identifying combination therapies capable of producing durable responses and potentially disease-free remissions. [Planet Flem/Getty Images]
    Developing resistant models directly from patient tumors can take years due to the scarcity of samples. In contrast, engineering resistance mechanisms in controlled laboratory models allows researchers to systematically study multiple escape pathways much faster.

    To accelerate discovery, scientists from ATCC and the Broad Institute collaborated to engineer a panel of drug-resistant NSCLC models using CRISPR gene editing and gene overexpression techniques. These models systematically model the resistance mechanisms that arise in patients treated with osimertinib, note the researchers.

    “With this powerful new set of tools, drug-sensitive and drug-resistant cancer cells can be studied side by side to understand therapeutic resistance and the underlying drivers,” says Roth Cheng, PhD, CEO of ATCC. “By creating and providing these cancer models along with a rich data-set to the global research community, our hope is to reveal hidden targets and combination strategies that turn today’s treatment failures into tomorrow’s breakthrough. We look forward to extending this approach to additional cancer types.”

    Engineering drug-resistant lung cancer models

    Led by William R. Sellers, MD, director of the cancer program at the Broad Institute, Fang Tian, PhD, director of biological content at ATCC, and Francisca Vazquez, PhD, director of the Cancer Dependency Map (DepMap) at the Broad Institute, the team identified representative classes of resistance mechanisms to osimertinib. They then selected three disease-representative, osimertinib sensitive NSCLC cell lines as the foundation for developing the new isogenic drug-resistant cell models.

    ATCC engineered the selected authenticated cell lines with resistance mechanisms using CRISPR-based methods. The six resistance mechanisms included: PIK3CA E545K mutation, KRAS G12D mutation, BRAF V600E mutation, EGFR C797S mutation, CCDC6-RET fusion, and TPM3–NTRK1 fusion.

    In addition, scientists at the Broad Institute are generating additional resistant cell lines driven by gene amplification mechanisms using overexpression methods.

    These engineered isogenic model systems allow researchers to compare genetically matched cancer cells that differ only by a specific resistance alteration—providing a powerful framework to study how tumors evolve under targeted therapy.

    The models will be integrated into the DepMap, a global effort to identify genetic vulnerabilities across hundreds of cancer cell models. The collaboration also contributes to the development of a Response and Resistance Map (ResMap), an emerging framework designed to systematically characterize how cancers respond to therapy and how resistance evolves.

    cancer researchers
    Engineered isogenic model systems allow researchers to compare genetically matched cancer cells that differ only by a specific resistance alteration—providing a powerful framework to study how tumors evolve under targeted therapy. [Sanjeri/Getty Images]
    “Drug resistance remains one of the most significant barriers to durable cancer treatment,” said Kirsty Wienand, PhD, senior research scientist in DepMap at the Broad. “Systematically engineering resistance mechanisms in well-characterized cell models allows us to study how tumors adapt to targeted therapy. Integrating these models into DepMap will help researchers worldwide identify new vulnerabilities and potential therapeutic combinations.”

    The collaboration ensures that both the biological models and the associated data will be widely accessible to the scientific community, says the research team. Data will be integrated into the DepMap portal, with links to the corresponding ATCC cell line identifiers. In addition, the engineered cell lines will be distributed globally through ATCC following authentication and quality control.

    Systematically engineering clinically relevant resistance mechanisms in lung cancer models, the collaboration establishes a scalable framework for studying how tumors escape targeted therapies, explain the scientists, adding that the resulting models and datasets will help researchers identify new vulnerabilities and therapeutic strategies to overcome drug resistance and improve outcomes for patients with cancer.

    By combining advanced cell engineering, functional genomics, and computational biology, the collaboration should provide an important resource for studying drug resistance, cancer vulnerabilities, and precision oncology strategies.

     

    ATCC and the Broad Institute will present the research findings at the American Association for Cancer Research® (AACR) Annual Meeting 2026, April 17–22 in San Diego:

    Title: Engineering isogenic models harboring resistance mechanisms to the latest-generation EGFR inhibitor in non-small cell lung cancer

    Session Category: Experimental and Molecular Therapeutics; Session Title: Drug Resistance 2: Tyrosine Kinase Inhibitors

    Date: April 22, 2026, 9:00 AM–12:00 PM, Poster Section 11, Poster Board: 8, Poster Number: 7029

    The post Decoding Resistance to Targeted Therapy via New Cancer Models appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO kicks off ‘Fight of Our Lives,’ highlighting urgency of biotech progress at Axios event https://edusehat.com/en/bio-kicks-off-fight-of-our-lives-highlighting-urgency-of-biotech-progress-at-axios-event https://edusehat.com/en/bio-kicks-off-fight-of-our-lives-highlighting-urgency-of-biotech-progress-at-axios-event Mon, 20 Apr 2026 17:30:04 +0700 Edusehat BIO, kicks, off, ‘Fight, Our, Lives, ’, highlighting, urgency, biotech, progress, Axios, event As biotech celebrates its 50th year, we are locked in “the fight of our lives” to ensure patients get more of the medical innovations they need and America remains a leader in biotech, BIO President & CEO John F. Crowley told an Axios event.

    The official launch of the “Fight of Our Lives” campaign by the Biotechnology Innovation Organization (BIO), hosted by Axios in Washington DC on April 16, featured discussions with Crowley, along with a patient advocate, a biotech CEO, and a congresswoman championing biotech.

    The “Fight of Our Lives” campaign uses human stories to hail 50 years of biotechnology breakthroughs—while providing a reminder that this momentum cannot be taken for granted.

    “Today, the U.S. biotechnology industry stands at a crossroads,” according to Crowley. “The ‘Fight of Our Lives’ recognizes what our industry has already delivered, and what is possible over the next 50 years with smart policies, sustained investment, and continued American leadership.”

    A revolution facing headwinds

    “We are in the midst of the most exciting revolution in science and medicine in human history,” Crowley told the Axios event, noting advances in neuroscience, genetic medicine, rare diseases, cancer, and many other areas.

    But there are forces slowing this progress, he warned.

    “We’re running up against headwinds that are almost entirely man-made,” Crowley said. “The good news is, if they’re man-made problems, we can come up with man-made and woman-made solutions.”

    Headwinds include ineffective policy moves, like “the heavy hand of tariffs,” and efforts aimed at affordability that do not achieve their goals. The real solution, Crowley said, is to address inefficiencies within the system.

    “The United States is the only country where more than half of the price of medicines don’t go to the inventors, don’t go to the innovators. It gets lost in the system of middlemen,” he said. “So let’s tackle that.”

    By simplifying the system, and addressing costs and barriers to access caused by pharmacy benefit managers and insurers, we can reduce out-of-pocket costs and increase patient access while encouraging biotech innovation and growth, Crowley said. This is especially important, he added, given China’s efforts to replace the U.S. as the world leader in biotechnology, which would hurt our economy and security.

    “We have the lead. We need to maintain and advance that lead, because at the end of the day, we care about biotechnology for our ability to make newer and better medicines, as an engine of economic growth, and for our national security,” Crowley said. “Biotech dominance is national security.”

    Concerns about China were raised in another discussion at the event, with Rep. Diana DeGette (D-CO), who has sponsored legislation to support biotech research. She said we must focus on supporting biotech rather than engaging in distractions. China is committed to biotech, “while the U.S. is having these really unnecessary debates about science,” Rep. DeGette warned.

    Noubar Afeyan, cofounder and CEO of Flagship Pioneering and a Moderna cofounder and board member, also spoke of distractions from those who would discredit science.

    “In the 39 years I’ve been involved in this industry, and starting companies, the scientific progress and technology is really beyond comparison,” he said, “and yet, the underlying science has been massively questioned by policymakers.” Afeyan worried that “the notion that science advances based on debate, based on facts, has been under unprecedented attack. I’ve not seen anything like it in my whole career.”

    With urgent action, Crowley said, we can still win the fight to ensure America leads the way in biotech innovation.

    “We are 50 years in, celebrating the founding of our very first biotechnology company, Genentech, in April of 1976,” he said. “Now we have to think about what the next 50 years look like.”

    ‘Fight of Our Lives’ campaign

    Before Crowley spoke, there was a showing of a video that encapsulates BIO’s “Fight of Our Lives” campaign. The video “humanizes who we are, what we do, why we do it, and for whom we do it,” Crowley said. He stressed that more videos and messaging would be forthcoming during the campaign.

    The “Fight of Our Lives” campaign puts a focus on the urgency of supporting biotech, through the real stories of the patients, families, caregivers, researchers, and entrepreneurs who fight every day to find answers to our health challenges.

    It elevates the people behind the progress and reinforces what it will take to sustain innovation, strengthen economic leadership, and deliver the next generation of breakthroughs.

    The website includes stories of some determined patients and provides the opportunity for other patients to share their own journeys.

    It also has an interactive timeline of highlights from the first 50 years of biotech, to remind us why the next 50 years matter.

    Visit the Fight of Our Lives website.

    Alex Missen contributed to this report.

    The post BIO kicks off ‘Fight of Our Lives,’ highlighting urgency of biotech progress at Axios event appeared first on Bio.News.

    ]]>     StockWatch: Revolution’s Phase III Pancreatic Cancer Data Dazzles Investors, Analysts https://edusehat.com/en/stockwatch-revolutions-phase-iii-pancreatic-cancer-data-dazzles-investors-analysts https://edusehat.com/en/stockwatch-revolutions-phase-iii-pancreatic-cancer-data-dazzles-investors-analysts Mon, 20 Apr 2026 06:40:09 +0700 Edusehat StockWatch:, Revolution’s, Phase, III, Pancreatic, Cancer, Data, Dazzles, Investors, Analysts Pancreatic cancer is one of the most difficult cancers to treat, with an overall five-year survival rate of 13%, according to the American Cancer Society, stretching from 3% for metastatic (Stage 4) to 44% for localized (Stages 1 and 2).

    Dismal odds such as these explain the enthusiastic response of investors when Revolution Medicine (NASDAQ: RVMD), a developer of RAS-addicted cancer therapies, announced dazzling data from its Phase III RASolute 302 trial (NCT06625320) evaluating its once-daily oral daraxonrasib in patients with metastatic pancreatic ductal adenocarcinoma (PDAC) who had been previously treated.

    In the trial’s overall (intent-to-treat) study population, daraxonrasib showed a median overall survival (OS) of 13.2 months, nearly double the 6.7 months demonstrated for standard-of-care chemotherapy, with a hazard ratio (HR) of 0.40 (p < 0.0001). Daraxonrasib also presented what Revolution called a manageable safety profile and no new safety signals.

    “These results represent a potentially transformative advance for patients and underscore daraxonrasib’s potential to redefine the treatment landscape. We are moving with urgency toward global regulatory submissions and remain committed to rapidly advancing this therapy for patients with a broad range of RAS-addicted cancers, Revolution’s CEO and chairman Mark A. Goldsmith, MD, PhD, said in a statement.

    Investors and analysts largely agreed with Goldsmith. Revolution’s stock reacted to the data release by soaring 54% this past week, starting with a 41% surge that sent the share price soaring from $96.43 on April 10 to $136.30 on April 13. Since then, the stock has jumped another 12%, reaching $152.54 at Wednesday’s closing bell. Profit-taking by investors led to a 2% slide on Thursday (to $149.27) and a 0.43% dip on Friday (to $148.63).

    “Our base case from stats sim [statistics simulation] was 11 vs. 7 mos, and based on our investor discussions, OS >12 months (and/or >6 mos delta vs. chemo) should drive meaningful stock upside,” Faisal Khurshid, an equity analyst with Jefferies, correctly predicted in an April 13 research note.

    A “clear win” scenario, Khurshid explained, would show daraxonrasib with an OS of greater than 11 to 12 months, and/or a daraxonrasib difference vs. chemo of >4–6 months, and/or an HR of <0.5–0.6.

    “Best-case outcome”

    “The disclosed data materially exceeds these expectations,” Khurshid declared. “This is by any measure a best-case outcome for RVMD [emphasis in original]. Darax’s performance was roughly in line with the Ph1 experience, and chemo only slightly outperformed historical benchmarks.”

    Revolution’s positive data sets the bar high for other cancer treatment developers—including Erasca (NASDAQ: ERAS), which is expected by the end of the first half to announce initial monotherapy data from its Phase I trial (NCT06983743) assessing ERAS-0015, a RAS-targeting molecule, in patients with RAS-mutant solid tumors.

    Khurshid’s colleague at Jefferies, Maury Raycroft, PhD, noted Erasca has said it believes a >10% improvement in response rates in PDAC or non-small cell lung cancer compared to daraxonrasib could support ERAS-0015 as being differentiated from Revolution’s candidate, as would improvement in two or more safety/tolerability attributes, such as rash, gastrointestinal diseases, and stomatitis.

    “Given the efficacy seen in ERAS’ 8 mg cohort and escalation to 40 mg, we remain (+)ve on the pot’l for stronger activity at higher doses,” Raycroft wrote in an April 13 research note. “That said, improved safety may be a key differentiator, particularly to enable combinations, especially as the competitive benchmark in PDAC continues to move higher.”

    At Leerink Partners, Jonathan Chang, PhD, senior managing director, emerging oncology, and a senior research analyst, raised the firm’s 12-month share price target 28%, from $115 to $147, “to reflect greater conviction in pipeline opportunities.”

    “Although RAS pathway drug development is highly competitive, we continue to believe encouraging clinical data from the innovative RAS(ON) platform, coupled with the large addressable population of RAS-dependent cancers, support a positive long-term outlook for RVMD,” Chang wrote.

    Leerink colleague Andrew Berens, MD, senior managing director, targeted oncology, and a senior research analyst, observed that daraxonrasib could set a standard for positive data that several RAS-based cancer drug developers are working to improve upon, citing:

    • Adlai Nortye (NASDAQ: ANL): Its panRAS inhibitor AN9025 shares the same method of action as daraxonrasib but with potentially greater potency and durability. The company’s pipeline also includes AN4035, a panRAS antibody-drug conjugate.
    • BridgeBio Oncology Therapeutics (NASDAQ: BBOT): Its BBO-11818, a pan KRAS ON/OFF inhibitor, has shown efficacy signals in early PDAC clinical studies. “The more targeted ON/OFF approach may lead to greater potency and less toxicity.”
    • Immuneering (NASDAQ: IMRX): Its atebimetinib showed 64% OS at 12 months as a first-line pancreatic cancer treatment in updated data announced January 7.

    “Not insurmountable”

    “Dara[xonrasib] sets a high bar that is not insurmountable. The data for dara look encouraging, with a clear benefit over SOC [standard-of-care] chemo, but could leave room for other novel approaches to improve on efficacy and/or tolerability,” Berens wrote. “We think dara could be the first targeted therapy for RAS mutant PDAC patients and potentially become the 2L SOC, establishing RAS inhibitors as key backbone therapies in PDAC.”

    That could lead to more RAS-based combination therapies, which Berens said has favorable implications for Tango Therapeutics (NASDAQ: TNGX)’s vopimetostat, an oral, selective PRMT5 inhibitor being studied in combinations with either daraxonrasib and another Revolution RAS(ON) cancer candidate, zoldonrasib, in a Phase I/II trial (NCT05732831).

    Revolution said it plans to present its data at the American Society of Clinical Oncology’s 2026 ASCO Annual Meeting, set for May 29–June 2 in Chicago. Data will also be presented to regulators as Revolution files a New Drug Application (NDA) with the FDA, which has selected daraxonrasib for its Commissioner’s National Priority Voucher (CNPV).

    Launched in October by FDA Commissioner Martin A. Makary, MD, CNPV is a pilot program that awards vouchers to drug developers whose work is deemed to address a health crisis in the United States, deliver more innovative cures, address unmet public health needs, and increase domestic drug manufacturing as a national security issue. In return, the vouchers entitle companies to reviews of their final applications within a target timeframe of 1–2 months rather than the current 10–12 months.

    The stock surge boosted Revolution’s market capitalization (share price times the number of outstanding shares) to approximately $30 billion. That’s the midpoint of the $28 billion to $32 billion acquisition that Merck & Co. (NYSE: MRK) was pursuing for Revolution in January, according to the Financial Times. That prospective deal reportedly collapsed after the companies failed to agree on the value of daraxonrasib and Revolution’s other cancer-fighting candidates.

    Merck never commented on its pursuit of Revolution, while AbbVie (NYSE: ABBV) flatly denied an earlier report that it sought to acquire the cancer drug developer. All the acquisition talk surrounding Revolution landed the company on GEN’s updated A-List Top 10 Takeover Targets of 2026, published March 9.

    Cashing in

    Revolution quickly cashed in on its positive data and stock surge, first proposing a $1 billion public offering of stock and debt, then doubling the size to $2 billion. The $2 billion offering consisted of concurrent public offerings of 10,563,381 shares of common stock at $142 per share (approximately $1.5 billion in gross proceeds) and $500 million of 0.50% convertible senior notes due 2033. Revolution also granted underwriters of the common stock offering a 30-day option to purchase up to an additional 1,584,506 shares.

    J.P. Morgan, TD Cowen, and Guggenheim Securities are book-running managers for the stock and note offering, with LifeSci Capital acting as lead manager.

    Daraxonrasib (formerly RMC-6236) is an oral RAS(ON) multi-selective, non-covalent inhibitor designed to target cancers driven by a variety of common RAS mutations, including PDAC, non-small cell lung cancer (NSCLC), and colorectal cancer. It is now under study in four global Phase III registrational trials—three in PDAC, the other in NSCLC. Daraxonrasib has been granted the FDA’s Breakthrough Therapy and Orphan Drug designations for the treatment of patients with previously treated metastatic PDAC harboring G12 mutations.

    The RASolute 302 trial is a 501-patient global, randomized, registrational clinical study designed to evaluate the efficacy and safety of daraxonrasib as a monotherapy in patients with previously treated metastatic PDAC. Patients were randomized to receive either an oral dose of 300 mg daraxonrasib once daily or investigator’s choice of standard of care cytotoxic chemotherapy. The trial enrolled patients with metastatic PDAC harboring a wide range of RAS variants, including those with RAS G12 mutations (such as G12D, G12V, and G12R), as well as patients without an identified tumor RAS mutation (wild type).

    Primary endpoints of RASolute 302 are OS and progression-free survival (PFS), as well as OS in patients with tumors harboring RAS G12 mutations. Secondary endpoints include PFS and OS in all enrolled patients (the intent-to-treat population) encompassing patients with and without identified tumor RAS mutations, as well as objective response rate, duration of response, and patient-reported quality of life.

    Kailera makes history with $625M IPO

    The “sign of life” StockWatch reported on last week when Avalyn Pharma filed paperwork for an initial public offering (IPO) is blooming this spring into a full blown comeback for IPOs, paced by what market watchers called the largest-ever public offering for a U.S. biotech—the eye-popping $625 million IPO carried out by Kailera Therapeutics—with at least two other companies submitting paperwork for filings of their own.

    Kailera is a developer of therapies for obesity and weight management based on glucagon-like peptide receptor 1 (GLP-1) agonists, alone or in combination with glucose-dependent insulinotropic polypeptide (GIP) receptor agonists. The company priced an IPO on Thursday that generated $489.7 million in net proceeds through the sale of 39,062,500 shares of common stock at $16 per share—the high end of the pricing range of $14–$16.

    On Kailera’s first full day of trading on Friday, investors showered the company with buys, propelling a 72% leap that sent shares to a high of $27.50 before the stock settled for a 62.5% gain, closing at an even $26.

    The company earlier anticipated $458.7 million in net proceeds based on a $15 per share IPO price—though any $1 increase to the IPO price would increase what Kailera netted from the offering by an additional $31 million, according to an amended Form S-1 registration statement filed April 13 with the U.S. Securities and Exchange Commission (SEC).

    Net proceeds could ultimately be even higher, since Kailera has granted its underwriters a 30-day option to purchase up to an additional 5,859,375 shares at the IPO price minus underwriting discounts and commissions. J.P. Morgan, Jefferies, Leerink Partners, TD Cowen, and Evercore ISI are joint book-running managers for the offering, with William Blair acting as lead manager.

    Pipeline development

    Kailera said the IPO plus its cash, cash equivalents, and marketable securities would give the company resources that it intended to spend on developing its four clinical-phase pipeline candidates, all in-licensed for $100 million upfront from Jiangsu Hengrui Pharmaceuticals (Shanghai Stock Exchange: 600276):

    • Ribupatide, the company’s lead product and a once-weekly injectable GLP-1/GIP receptor dual agonist peptide, including to fund three ongoing global Phase III KaiNETIC clinical trials into the second quarter of 2028 (more than $625 million, the estimate based on the $15 share price)
    • Oral ribupatide, a once-daily oral tablet formulation of ribupatide, including the funding of planned Phase III trials into the second quarter of 2028 (more than $150 million)
    • KAI-7535, a once-daily oral small molecule GLP-1 receptor agonist, including through the completion of a planned Phase II clinical trial (more than $50 million)
    • Other R&D activities, including development of KAI-4729, a once-weekly injectable GLP-1/GIP/glucagon receptor tri-agonist, as well as for working capital and other general corporate purposes (Remaining proceeds, not quantified)

    Kailera gained exclusive global rights outside Greater China to Jiangsu Hengrui’s GLP-1 portfolio in 2024. That year, Kailera was launched with a $400 million Series A financing co-led by Atlas Venture, Bain Capital Life Sciences, and RTW Investments. Last October, Kailera garnered an additional $600 million in Series B financing led by a new investor, Bain Capital Private Equity.

    “Our obesity-first approach seeks to capitalize on and improve upon proven science to advance product candidates which have the potential to maximize weight loss and address other critical needs in the current therapeutic landscape and to provide options, including oral options and alternative mechanisms, for people living with obesity no matter where they are in their treatment journey,” Kailera stated in its amended registration statement.

    Kailera has adjusted the value of its cash and equivalents plus marketable securities from $652.728 million to a pro forma $1.142 billion in assets, reflecting the conversion of all outstanding preferred shares into common stock upon closing of the offering, plus an amended and restated certificate of incorporation.

    Kailera’s IPO has surpassed the previous record-high among U.S. biotechs, the $604 million offering of Moderna (NASDAQ: MRNA) in December 2018, two years before the messenger RNA (mRNA) vaccine developer won FDA emergency authorization for its COVID-19 vaccine.

    In the works

    At least two other biotechs have filed Form S-1 registration statements for future IPOs in recent days, without disclosing how many shares they plan to raise or their offering prices.

    • Seaport Therapeutics is a developer of treatments for depression, anxiety, and other debilitating neuropsychiatric disorders based on its GlyphTM platform, a lymphatic-targeting prodrug technology designed to enhance a drug’s oral bioavailability and reduce side effects by bypassing first-pass metabolism. “Through our differentiated approach, we identify clinically validated mechanisms with established efficacy and safety profiles that have historically been limited by high first-pass metabolism, low bioavailability, and/or side effects,” Seaport stated in its Form S-1
    • Hemab Therapeutics, a developer of subcutaneous treatments for rare blood coagulation disorders, said its lead candidate, sutacimig (HMB-001), is a bispecific antibody in Phase I/II trials for the prophylactic treatment of Glanzmann thrombasthenia and Phase II studies for the prophylactic treatment of Factor VII deficiency. Another therapeutic candidate, HMB-002, is a monovalent antibody in Phase I/II trials for the subcutaneous prophylactic treatment of Von Willebrand disease. “We are building a franchise designed to address select coagulation disorders where we believe advances in biology, drug modality, and care delivery have the potential to meaningfully improve disease management,” Hemab stated in its Form S-1.

    Leaders and laggards

    • MeiraGTx (NASDAQ: MGTX) shares yo-yoed, rising 26% from $8.97 to $11.29 Tuesday, after the company announced plans to present three-year data from its Phase I AQUAx trial (NCT04043104) evaluating AAV-hAQP1 in Grade 2/3 radiation-induced xerostomia. MeiraGTx reported “clinically meaningful” improvements in xerostomia symptoms, such as the average XQ score improving by 17 points (39.5%) at month 12, bilaterally treated participants reporting greater improvement than those treated unilaterally (21 points vs 13 points), and 75% of bilaterally-treated patients reporting transformative (≥10 point) improvement at month 12. After dipping 0.4% to $11.25 Wednesday, shares slumped 16% to $9.48 Thursday as MeiraGTx priced an approximately $100 million offering of 11,111,111 shares at $9 per share. Proceeds plus existing cash and cash equivalents are expected to fund commercial launches of AAV-hAQP1 and botaretigene sparoparvovec (“bota-vec”), a gene therapy for XLRP that MeiraGTx agreed to acquire from Johnson & Johnson (NYSE: JNJ) for $25 million cash upfront, a $50 million one-time payment tied to achieving specified regulatory and commercial milestones, plus a “mid-teens” royalty on global net sales starting on or after July 1, 2029.
    • Travere Therapeutics (NASDAQ: TVTX) shares soared 37% from $30.70 to $42.13 Tuesday after the rare disease drug developer won full FDA approval for Filspari® (sparsentan) in a second rare kidney disease. Filspari has become the first and only treatment for focal segmental glomerulosclerosis (FSGS), specifically to reduce proteinuria in adults and younger patients ages eight years and older with FSGS without nephrotic syndrome. Filspari won FSGS approval based on positive data from the Phase III DUPLEX trial (NCT03493685), where researchers reported a statistically significant 46% reduction in proteinuria from baseline to Week 108 in patients treated with Filspari vs. 30% for those treated with standard of care maximum labeled dose irbesartan, marketed by Sanofi (Euronext Paris: SAN) as Avapro®. Filspari first won FDA approval in 2023 to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression.

    The post StockWatch: Revolution’s Phase III Pancreatic Cancer Data Dazzles Investors, Analysts appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     10x Genomics Unveils Atera Spatial Platform at AACR Meeting https://edusehat.com/en/10x-genomics-unveils-atera-spatial-platform-at-aacr-meeting https://edusehat.com/en/10x-genomics-unveils-atera-spatial-platform-at-aacr-meeting Sun, 19 Apr 2026 12:45:09 +0700 Edusehat 10x, Genomics, Unveils, Atera, Spatial, Platform, AACR, Meeting The genomics community’s long wait for 10x Genomics’ highly anticipated news is finally over. On Saturday night, at the Hard Rock Café Hotel in San Diego—across the street from the American Association for Cancer Research (AACR) conference—the company hosted the “Impossible” party to announce its new spatial instrument—the Atera.

    Serge Saxonov, PhD, CEO of 10x Genomics, walking onto the stage to thunderous applause, noted that there is “a gap between what we need to see and what we have been able to measure.” The Atera, which enables whole-transcriptome spatial biology at scale, “obliterates the typical trade offs” that come with existing spatial tools, he said.

    “This is the biggest launch in our history. I am the most excited I’ve ever been about any product, or any product category, across the board,” Saxonov told GEN. “It has been a long time in development, and it is what we have known the world needs for a long time. I think it will fundamentally change how we measure and understand biology, and it really puts research on a new trajectory. It is really exciting to be at a place now where we can deliver it to the world.”

    Nuts and bolts

    Atera offers more plex, throughput, and sensitivity than 10x Genomics’ Xenium—enabling whole-transcriptome at scale. More specifically, when compared to Xenium, Atera has four times the throughput, six times higher plex capacity for targeted assays, 3.6x higher plex, and 2–3x sensitivity for whole transcriptome assays.

    10x Genomics Atera
    10x Genomics Atera

    The price for Atera is $495,000, and the instrument measures roughly 53” x 36” x 64” or (4.42 ft × 3 ft × 5.33 ft). Orders are currently being taken, and the instrument will be available in the second half of this year.

    The instrument can run up to 800 1 cm2 whole transcriptome samples (FFPE and fresh frozen) per year, with flexible run configurations, and a greater than 5 cm² imageable area per slide (for greater than 2,000 mm² total tissue per run when using all four slides.)

    There are 18,000-genes on the Atera WTA (whole transcriptome) with stackable customization of 1,000-gene Atera Select panels available now, and optional stacking of up to three 1,000-gene panels coming in the future.

    “Spatial genomics with whole-transcriptome profiling capabilities is the ultimate approach to measure single cells in their tissue context,” Holger Heyn, PhD, ICREA professor at the Centro Nacional de Análisis Genómico (CNAG) and member of the Human Cell Atlas, added. “All other lower-plexity approaches have been just a warm-up phase leading to this application.”

    Jasmine Plummer, PhD, associate member of the St. Jude Faculty and director of the Center for Spatial OMICs points out that the whole transcriptome, while exciting, can bring a big “sticker shock” for many researchers because it will require a lot more probes in contrast to a sequencing-based platform, where a library accesses all of the genes.

    The instrument uses standard glass microscopy slides, which is exciting to Plummer. In the past, she said, slides have posed a challenge when coordinating with other researchers, and using regular slides will be more “pathology friendly.”

    An end to tradeoffs? 

    Existing spatial technologies, which are still relatively nascent in genomics, have been constrained by tradeoffs between plex, resolution, and throughput. Researchers have had to make choices and prioritize.

    “In general, with the landscape as it is today, there is a tradeoff,” Nick Banovich, PhD, VP of scientific development at TGen, and professor of bioinnovation and genome sciences division and director of the Center for Spatial Multi-Omics (COSMO), told GEN. “The closer you walk toward whole transcriptome, the lower the per gene sensitivity.”

    “The most exciting thing [about Atara],” he continued, “is that there is still quite good sensitivity with whole transcriptome breadth. That’s the huge advantage of this system; there is no tradeoff anymore.”

    However, this launch comes just over three years after Xenium’s launch. Purchasing a new instrument so soon may pose a challenge. Plummer notes: “In this economy, with the uncertainty of scientific funding, it is concerning to ask customers—many of whom just landed a machine—to spend another several hundred thousand dollars.”

    Why AACR?

    Oncology is one of the most exciting, most promising applications of spatial, especially in the near term, noted Saxonov. This is, in large part, because the work exists across the spectrum—from basic discovery to translation to clinical applications. Spatial is unambiguously important, he asserted.

    Unveiling at AACR “just made a lot of sense.”

    In addition to the party, the company will host a digital launch event on Tuesday, April 21. Within the AACR program, a presentation from the German Cancer Research Center (DKFZ) will include data generated on the platform, highlighting Atera’s ability to uncover cancer biology not accessible with legacy approaches. Researchers distinguished multiple malignant and stem cell states across disease stages, within a single colorectal tumor sample, and mapped how these populations interact with the surrounding immune microenvironment. The data reveal a more complex immune landscape that could inform future therapeutic strategies and drug development. In addition, two posters (#7116, #6216) will include data from Atera.

    The future

    10x Genomics said that Atera will play a role in advancing large data studies. For example, the company noted that Atera will enable the goal of the Human Cell Atlas (HCA) as it continues its mission to map every cell type in the human body.

    “With the Human Cell Atlas entering its next phase of generating spatially resolved atlases, whole-transcriptome approaches will be the workhorse for data generation,” Heyn told GEN.

    “I am excited to see the Atara platform being launched now,” he added. “It is very timely as we ramp up production for the Human Cell Atlas 2.0 phase.”

    Atera’s future

    The company presented a roadmap with future plans at the AACR event, highlighting spatial proteomics, automation, base by base sequencing (a de novo sequencing assay) and software improvements.

    Atera, Saxonov told GEN, is a fundamental platform that the company will continue enabling. It lays the groundwork for the next decade of research and work. This point in time, he said, feels similar to the early days of next generation sequencing (NGS). And although the company will continue to develop its other platforms and product lines, Atera has “massive amounts of headroom to keep building on top of it. It is the convergence of all these different technology stacks and different fields onto one.”

    “What the platform can do right out of the gate is exciting. And all the things that it can do in the future will be really, really exciting,” he asserted.

    The post 10x Genomics Unveils Atera Spatial Platform at AACR Meeting appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     CPC Biotech Launched to Offer a Fluid Management Component Portfolio https://edusehat.com/en/cpc-biotech-launched-to-offer-a-fluid-management-component-portfolio https://edusehat.com/en/cpc-biotech-launched-to-offer-a-fluid-management-component-portfolio Sat, 18 Apr 2026 08:05:09 +0700 Edusehat CPC, Biotech, Launched, Offer, Fluid, Management, Component, Portfolio CPC Biotech was launched as a combined entity that brings together the connector technology of CPC and the flow control expertise of PSG Biotech. The goal is to create a fluid management component portfolio.

    Operating under the CPC Biotech brand as part of PSG, the new company, says a spokesperson, will serve as a single source for sterile connectors, low-shear pumps, flow meters, and sensors.

    “We will drive increased innovation to better serve our customers and help advance the biopharma industry,” said John Boehm, general manager, CPC Biotech.

    CPC Biotech’s solutions support a range of bioprocessing applications, spanning the upstream and downstream biologic drug manufacturing process, as well as cell and gene therapy production, according to Boehm. The company’s products are manufactured across facilities in North America, Europe, and Asia, with customers in more than 50 countries through a network of channel partners.

    CPC Biotech will make its public debut at INTERPHEX 2026, taking place April 21–23 at the Jacob K. Javits Convention Center in New York City, located at Booth #3129.

     

     

    The post CPC Biotech Launched to Offer a Fluid Management Component Portfolio appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Targeted Gene Delivery Calms Lung Inflammation in Respiratory Infection Mouse Models https://edusehat.com/en/targeted-gene-delivery-calms-lung-inflammation-in-respiratory-infection-mouse-models https://edusehat.com/en/targeted-gene-delivery-calms-lung-inflammation-in-respiratory-infection-mouse-models Sat, 18 Apr 2026 04:30:13 +0700 Edusehat Targeted, Gene, Delivery, Calms, Lung, Inflammation, Respiratory, Infection, Mouse, Models A group of scientists have developed a targeted delivery platform that can induce anti-inflammatory cytokine expression in mouse lungs, which helps restrict tissue damage from respiratory infections without triggering systemic side effects. Full details are published in Science Immunology in a paper titled “Gene delivery of immunomodulatory cytokines to the lung preserves respiratory function during inflammatory challenge.”

    The study was led by scientists in the pathology department at the University of Cambridge working alongside collaborators elsewhere. Together, they “developed a gene delivery system to express anti-­inflammatory cytokines in the lung, which reestablishes local immune homeostasis without triggering systemic effects,” according to details provided in the paper. Specifically,  they used an adeno-associated virus cargo system (AAV6.2-CC10) to induce “production of interleukin-­2 (IL-­2), IL-1 receptor antagonist (IL-­1RA), and IL-­10 in situ in the lung microenvironment.” They accomplished this “with no detectable expression or immunological deviation in the peripheral immune system.”

    According to the developers, their work could lead to new therapeutics that control inflammation following several viral infections, which has been linked to higher mortality rates in cases of SARS-CoV-2 and influenza. Prolonged inflammation during a viral infection also increases the chances that patients could contract bacterial and fungal infections. Importantly, the approach provides a way to harness the “therapeutic potential of immunomodulatory cytokines” which to date have had limited success as biologic drugs due in part to the short half-lives of cytokines as well as the risks of multiorgan effects. “This tool has been proven to deliver sustained and localized expression as evidenced by the results from three tested cytokines,” the effects of which were “restricted to the lungs” and resulted in “prolonged production over the course of weeks.” 

    The paper goes into the details of how the scientists characterized their method and demonstrated that it induced expression only in specific lung epithelial cells without off-target accumulation. Also provided are details of how they used the system to assess how lung-specific expression of IL-2, IL-1RA, and IL-10 affected disease severity in mouse models of influenza. They found that IL-2 expression was not especially beneficial during infection, possibly due to the amplification of protective regulatory T cells and proinflammatory CD8 T cells in the lungs. However, IL-1RA and IL-10 reduced tissue damage and improved recovery after infection and inflammation. 

    In addition, data from their experiments showed that delivering either individual cytokines or a cocktail of all three protected mice from influenza-associated aspergillosis. In fact, treated mice showed “reduced neutrophil infiltrates and improved health outcomes,” including reduced weight loss compared to untreated mice, the scientists wrote. 

    Future experiments with human cell culture systems could lay the groundwork for preclinical testing. However, there are still some limitations. For example, “we did not evaluate the kinetics of repeated administration of the same AAV vectors,” the scientists wrote. “Repeated administration can lead to the development of neutralizing antibodies, which can hinder the uptake of AAVs in subsequent treatments.” Another challenge is with the cargo itself. Though it performs well in mouse models, its “utility in a patient-based setting needs to be tested,” the scientists said. 

    The post Targeted Gene Delivery Calms Lung Inflammation in Respiratory Infection Mouse Models appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Tunneling Neurons in Adult Bird Brains Provide New Insights into Neurogenesis https://edusehat.com/en/tunneling-neurons-in-adult-bird-brains-provide-new-insights-into-neurogenesis https://edusehat.com/en/tunneling-neurons-in-adult-bird-brains-provide-new-insights-into-neurogenesis Sat, 18 Apr 2026 04:30:11 +0700 Edusehat Tunneling, Neurons, Adult, Bird, Brains, Provide, New, Insights, into, Neurogenesis Despite its small size—it could sit in the palm of your hand—the zebra finch is a remarkable learner. A songbird native to Australia, it’s renowned for its ability to pick up new songs. That talent has made it a favorite of scientists studying how animal brains imprint new skills, particularly vocal learning, or the capacity to perfect new sounds.

    Researchers at Boston University, working with scientists at the Max Planck Institute for Biological Intelligence and the MRC Laboratory of Molecular Biology, have now discovered another quirk to the zebra finch brain—one that could also have implications for understanding our own. In a study that looked at the bird’s brain in unprecedented detail, the scientists uncovered new insights into neurogenesis—the birth, migration, and maturation of neurons—that may help the brain learn, add new skills, and restore and repair itself.

    Observing the finch brain using a high-powered microscope, the researchers watched as new neurons made their way through the brain en route to bolstering existing circuits and connections. The expectation was that these neurons would step around established brain structures, including more mature brain cells, to better preserve them. Instead, the investigators saw the neurons tunnel right through. According to the BU-led team, the findings could help explain human vulnerability to a range of brain disorders. The researchers also noted that cell tunneling is used by some metastatic cancer cells.

    “We found that in songbirds, new neurons in the adult brain behave like explorers forging a path through a dense jungle,” said Benjamin Scott, PhD, a BU College of Arts & Sciences assistant professor of psychological and brain sciences and the study’s corresponding author. That may help them learn new things or repair damage, but it could come with a cost to existing cells and memories—and that might be why neurogenesis is, in humans, something that doesn’t seem to extend beyond the womb. “This potentially disruptive behavior may help explain why humans and other mammals have limited capacity to regenerate brain tissue in adulthood,” commented Scott, “leaving us more vulnerable to neurodegenerative disorders such as Alzheimer’s disease.”

    Scott is senior and corresponding author of the team’s published paper in Current Biology, titled “Songbird connectome reveals tunneling of migratory neurons in the adult striatum,” in which the researchers commented that their collective findings “… suggest that migrating neurons may physically reshape the mature circuit to reach their targets, revealing an unexpected degree of structural and functional plasticity in the adult brain.”

    At birth our brains have pretty much all the neurons they are ever going to have. Other organs—from your skin to your heart—might get frequent cell updates, but the brain is working on version 1.0. That’s true for most mammals, but not for fish, reptiles, and birds—their brains get a regular refresh.

    “This raises two questions,” said Scott, who’s also affiliated with BU’s centers for neurophotonics, photonics, and systems neuroscience. “Why do other species have high rates of neurogenesis throughout life and why is it so restricted in humans? And is there something we can learn from their biology that we might be able to harness in future?”

    Scott typically studies the neural circuits that control behavior in humans and other mammals, but chose the zebra finch to investigate neurogenesis because it has a reputation as a champion species—it’s really good at generating new neurons. “Songbirds are valuable model organisms for the study of neuron migration in the adult brain,” the authors wrote. “In these species, new neurons integrate into brain regions that control complex learned behaviors, where they establish synapses with mature neurons and respond to sensory stimuli.”

    However, the team pointed out, a key question is how these new neurons interact with mature circuit structures in the brain. “It is not known whether neurons pursue migratory routes that flexibly avoid these structural obstacles or deform surrounding tissue to reach their targets,” they wrote. “While prior studies have examined the molecular mechanisms and functional consequences of adult neurogenesis, few have investigated the physical interactions between migrating neurons and their surrounding microenvironment.”

    For their newly reported study the team used electron microscopy (EM)-based connectomics to examine how migrating neurons interact with mature circuit elements. “We applied a new tool to study this process [neurogenesis] called electron microscopy-based connectomics—basically a really high-powered microscope—to image these cells at a very high resolution,” Scott explained. “Our first hope was just to say, what does this look like at a detail we couldn’t see before?”

    Their resulting data revealed intricate interactions between migratory neurons in the adult striatum and their environment, but also showed up the tunneling neurons. “Our findings support a model in which migrating neurons disperse throughout dense neural tissue in multiple directions, making various contacts with surrounding structures,” the team wrote in summary. “In addition, our data reveal a previously undescribed form of neuron migration in which new neurons cause deformities in nearby neurons and synapses.”

    The authors say that, to their knowledge, tunneling migration by neurons hasn’t previously been reported in the vertebrate nervous system. It’s possible that this is due to the constraints of study methods used, but it’s also possible that tunneling is a specialization of neurogenesis in birds.

    If these new neurons are deforming brain tissue, commented Scott, are they also disrupting memories along the way? And, if neurogenesis comes with a cost, how does that balance against the brain’s capacity for learning new things and repairing after injury? And as the authors pointed out, “Interestingly, tunneling-like behavior has been described in metastatic cancer cells, which navigate confined spaces by actively deforming their microenvironments. Tunneling may therefore reflect a conserved strategy adopted by specialized migratory cell types in dense tissues.”

    Scott has two—as yet untested—hypotheses for what the findings might mean for the human brain. The first is that our brains evolved to limit neurogenesis after birth as a form of protection—a way of making sure determined neurons couldn’t barge through mature connections and damage memory storage. “There is an alternative framing that is more optimistic,” he also noted. “Our discovery of tunneling shows how cells can move without glia scaffolds.”

    These are the structures that operate as highways for migrating neurons. “Most glia scaffolds are lost in humans after birth, and this loss was thought to be an obstacle for neurogenesis in the adult brain,” says Scott. “However, our work shows that new neurons in the bird do not need this glia scaffold. This is exciting because it means that brain repair may not require specialized glia scaffolds.” That opens the door for scientists to explore potential stem-cell therapies that would spark neurogenesis in humans.

    In summary, the authors wrote, “These results reveal the value of applying EM connectomics to adult neurogenesis and suggest that migratory neurons may dramatically perturb the existing functional circuits as they migrate and integrate. Furthermore, they reveal the remarkable structural flexibility of mature neural circuits.”

    In current studies, Scott and the team in his BU Laboratory of Comparative Cognition are digging into the biology driving neurogenesis to uncover which genes are regulating the process. Much of the work merges ideas and tools from biomedical engineering and neuroethology, the study of the mechanisms underpinning animal behavior.

    “Right now, we’re using a technique called single-cell RNA sequencing to identify genes that are expressed by these new neurons as they migrate,” said Scott. “We want to know what other cells they’re talking to as they move and how they are speaking to these different cells.” That’ll help them figure out whether neurons warn other cells they’re traveling through and how they know where to stop and integrate with a current circuit.

    “We share a lot with our animal relatives on this planet,” noted Scott. And, while the term “bird brain” might be an insult, by learning more about the biology of songbird brains, he says, we could learn some remarkable things about our own.

    The post Tunneling Neurons in Adult Bird Brains Provide New Insights into Neurogenesis appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     The State of Multiomics &amp; NGS https://edusehat.com/en/the-state-of-multiomics-ngs https://edusehat.com/en/the-state-of-multiomics-ngs Sat, 18 Apr 2026 00:55:15 +0700 Edusehat The, State, Multiomics, NGS

    REGISTER NOW

    Broadcast Date: 
    • Time: 

    Next-generation sequencing (NGS) has never been cheaper or more accessible. In turn, spatial biology, single-cell, and proteomics are fueling exciting advances in biology. The rapidly declining cost of whole-genome sequencing is empowering researchers to ask questions that were beyond reach just a few years ago. 

    In The State of Multiomics & NGS virtual summit—sponsored exclusively by Illumina—GEN invites you to watch a superb line-up of presenters covering key topics in the world of multiomics, spatial biology, and NGS. This year’s summit features a broad selection of talks and panel discussions from renowned experts that offers our audience rich insights into the latest NGS platforms, spatial biology applications in neurodegenerative diseases and cell biology, and a critical look at data management challenges and solutions.

    Among the highlights are:

    • A keynote presentation from Miranda Orr, PhD, on the use of spatial biology in studying Alzheimer’s and other neurodegenerative diseases

    • A deep dive into the latest NGS platforms and applications with veteran genomics analysts Keith Robison, PhD, and Shawn Baker, PhD

    • Modeling the proteome architecture of human cells with Noorsher Ahmed, PhD (Lundberg lab)

    • Chris Dwan on data management strategies for multiomics

    • And a sponsored breakout conversation featuring Illumina’s chief medical officer, Eric Green, MD, PhD, and cancer biologist Bodour Salhia, PhD (USC)

    We look forward to seeing you on April 29. Registration is absolutely free! !

    Guest Speakers Include

    The State of Multiomics & NGS speakers

    illumina logo

    The post The State of Multiomics & NGS appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     For What’s Next: Preparing Today’s Lab or Tomorrow’s Discoveries https://edusehat.com/en/for-whats-next-preparing-todays-lab-or-tomorrows-discoveries https://edusehat.com/en/for-whats-next-preparing-todays-lab-or-tomorrows-discoveries Sat, 18 Apr 2026 00:55:13 +0700 Edusehat For, What’s, Next:, Preparing, Today’s, Lab, Tomorrow’s, Discoveries For What’s Next: Preparing today’s lab for tomorrow’s discoveries eBook coverModern biology is accelerating at an unprecedented pace, and with it comes increasing complexity. As a result, researchers are shifting toward more patient reflective models, uncovering richer phenotypes, and generating multidimensional datasets that push the limits of traditional workflows. However, become more sophisticated, operational challenges grow. Manual steps introduce variability, workflows don’t scale cleanly across teams or sites, and data pipelines struggle to keep pace with expanding volume and nuance. The result is a familiar bottleneck—ambitious science constrained by throughput, reproducibility, and the significant hands-on time required just to keep experiments moving.

    This eBook brings together a curated collection of resources to help you break through those constraints. Each asset addresses the fundamental challenge of how to build workflows that are reproducible, scalable, and capable of generating confident, decision ready data. From emerging trends in 3D biology to actionable insights for implementing advanced models, the resources in this collection reflect how labs are adapting tools, methods, and data strategies to keep pace with increasingly complex science.

    Discover how automation elevates upstream processes such as colony picking, clone selection, and supports specialized microbiome workflows by reducing bottlenecks and tightening reproducibility. Explore how AI powered automation helps ensure consistent performance across users and sites for both 2D and 3D model generation and expansion while reducing variability, supporting reliable organoid expansion, and returning valuablevtime to scientists by removing repetitive work. Learn how fast, quantitative plate based assays help teams quickly pinpoint meaningful biological responses, focusing deeper profiling where it will have the greatest impact. And see how high content imaging paired with AI enabled analysis reveals subtle phenotypes that traditional readouts often overlook, connecting treatment effects to underlying biology with far greater clarity.

    Together, these insights reflect a unified strategy built FOR WHAT’S NEXT: integrated workflows and intelligent precision-automation that deliver reproducible results, scale seamlessly, reduce manual intervention, and support increasingly complex biology.

    The post For What’s Next: Preparing Today’s Lab or Tomorrow’s Discoveries appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     TRACERx MRD Results Showcase ppmSeq’s Ultra-Sensitive ctDNA Detection at AACR https://edusehat.com/en/tracerx-mrd-results-showcase-ppmseqs-ultra-sensitive-ctdna-detection-at-aacr https://edusehat.com/en/tracerx-mrd-results-showcase-ppmseqs-ultra-sensitive-ctdna-detection-at-aacr Fri, 17 Apr 2026 21:20:10 +0700 Edusehat TRACERx, MRD, Results, Showcase, ppmSeq’s, Ultra-Sensitive, ctDNA, Detection, AACR Minimal residual disease (MRD) continues to be a central focus at the AACR meeting. The small numbers of cancer cells that remain in the body after treatment helps gauge the effectiveness of a treatment and relapse risk. The ability to detect those cells, even in tiny amounts, is an ongoing goal of the cancer community.

    At this year’s AACR, the sequencing company Ultima Genomics is announcing new findings in this area using its ppmSeq technology. The data will be presented across six abstracts, including a plenary session.

    Highlighting the program will be initial TRACERx (TRAcking Cancer Evolution through therapy (Rx)) MRD data showcasing performance of ppmSeq relative to ultrasensitive bespoke panels.

    TRACERx is a long-term study—one of the largest tumor evolution studies—funded by Cancer Research UK. The program analyzes how cancer evolves, spreads to other parts of the body, and develops resistance to treatments. Instead of taking just one biopsy, researchers sample different parts of the same tumor and metastases; the program involves sequencing multi-region and multi-time-point genetic data from over 3,200 tumor samples from over 800 lung cancer patients.

    The data will be presented at a plenary session by Charles Swanton, FRCP, BSc, PhD,  professor at The Francis Crick Institute in the U.K. He will present an early validation pilot of ppmSeq across 50 plasma samples—using tumor-specific variants identified from prior whole genome sequencing—achieved high analytical sensitivity for ctDNA detection at low single-digit parts-per-million.

    “TRACERx has always followed the science of cancer evolution wherever it leads,” said Swanton. “Improving the sensitivity of ctDNA detection is central to the wider ambition for MRD monitoring, and expanding studies across a broader patient population will give us the statistical power and clinical context to determine how whole genome MRD monitoring can be deployed at NHS scale and beyond.”

    Data from collaborators will also be presented at the conference. Labcorp will present data from an independent analytical study of an assay developed in coordination with ppmSeq technology, including the performance across multiple solid tumor types in pre-surgical, treatment-naive plasma samples. This analysis of 120 non-cancerous donor samples showed specificity exceeding 99.9%, underscoring the ability of ppmSeq whole genome sequencing to accurately differentiate between cancerous and non-cancerous samples, minimizing false positives. Additional analysis across three commercially available cancer cell lines spanning 13 concentration levels from 0.5 to 500 parts per million showed a 95% limit of detection below 3 ppm, demonstrating the assay’s capacity to detect ultra-low levels of circulating tumor DNA (ctDNA).

    “For a long time, the question has been whether you can get truly ultra-sensitive MRD detection from a whole genome approach without all the complexity of bespoke assays,” notes Gilad Almogy, PhD, CEO of Ultima Genomics. “What these AACR data show is that the answer is yes. We’re seeing ppmSeq deliver the level of sensitivity needed to make whole genome MRD practical, scalable, and much easier to deploy globally.”

    The post TRACERx MRD Results Showcase ppmSeq’s Ultra-Sensitive ctDNA Detection at AACR appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Q&amp;A with National Health Council: Driving patient-centred policy https://edusehat.com/en/qa-with-national-health-council-driving-patient-centred-policy https://edusehat.com/en/qa-with-national-health-council-driving-patient-centred-policy Fri, 17 Apr 2026 17:50:04 +0700 Edusehat Q&A, with, National, Health, Council:, Driving, patient-centred, policy Since its founding in 1920, the National Health Council (NHC) has brought diverse organizations together to forge consensus and drive patient-centered health policy in the United States. 

    Their work is more important than ever as a myriad of challenging policy initiatives and payment models have threatened patient access and affordability. As such, their 2026 Science of Patient Engagement Summit is focused on turning patient-gathered evidence into impact, empowering participating organizations to turn patient engagement into improved outcomes by strengthening how care is designed and delivered.

    Bio.News sat down with Randy Rutta, Chief Executive Officer at the NHC, to discuss the organization’s work, as well as what participants can expect during this year’s summit.

    Why is patient engagement so critical to NHC and its mission?

    Patient Engagement is at the heart of what the NHC does. We exist to ensure that patients are not only represented but are also at the center of health care. The NHC’s footprint in this field equips all actors in the health sector to meaningfully engage patients from drug and device development to patient care protocols and shared clinical decision-making.

    NHC has played a key role in ensuring the patient perspective is incorporated in key policies such as the drug price ‘negotiations’ under the Inflation Reduction Act (IRA). What do you see as the key to successful patient engagement?

    The NHC has worked tirelessly to ensure that patients have been included in the Initial Price Applicability Year (IPAY) drug negotiations from the start. While not required by statute, the NHC successfully advocated for Centers for Medicare and Medicaid Services (CMS) to incorporate meaningful patient input into the IPAY process, underscoring that doing so is essential to ensuring that any actions taken do not inadvertently undermine patient access to affordable treatments. This approach reflects what effective patient engagement should look like in practice and has already led to meaningful improvements in the process. 

    What resources does NHC provide to member organizations and the broader community to support effective patient engagement?

    The NHC has a wide range of patient engagement resources on our website. You can find information and resources about the current IPAY process, the NHC’s Patient Engagement Compensation and Contracting Toolbox, Rubric to Capture the Patient Voice, and much more. Many of these tools and more will be highlighted in our upcoming Science of Patient Engagement Summit.

    One of NHC’s flagship convenings, the Science of Patient Engagement Summit, is now in its 9th year. How have you seen patient engagement evolve over time?

    Patient Engagement has evolved from a “nice to do” to a business-critical function that enables companies to develop treatments and products that truly meet the needs and expectations of patients. At this year’s Science of Patient Engagement Summit, May 11-12 in Arlington, VA, we are focused on how patient engagement is used to shape research, care delivery, and decision-making throughout the health care sector. The Summit is designed to share concrete examples, lessons learned, and approaches you can apply across your work. For the first time, the Summit is open to a wider, health-sector audience, not just NHC members.

    The post Q&A with National Health Council: Driving patient-centred policy appeared first on Bio.News.

    ]]>     Stem Cell Editing Programs the Immune System to Make Own Therapeutic Proteins https://edusehat.com/en/stem-cell-editing-programs-the-immune-system-to-make-own-therapeutic-proteins https://edusehat.com/en/stem-cell-editing-programs-the-immune-system-to-make-own-therapeutic-proteins Fri, 17 Apr 2026 10:35:11 +0700 Edusehat Stem, Cell, Editing, Programs, the, Immune, System, Make, Own, Therapeutic, Proteins For pathogens like HIV, malaria, and rapidly evolving influenza strains, coaxing the immune system to produce the rare, highly potent antibodies needed for protection has long been a scientific bottleneck. Vaccines can train B cells to evolve such broadly neutralizing antibodies, but only under ideal conditions—and only in a small fraction of people. Even attempts to genetically edit mature B cells produced responses that faded as the cells died out.

    A team at the Rockefeller University has now taken a more upstream approach: programming hematopoietic stem and progenitor cells (HSPCs)—the source of all B lymphocytes—to carry permanent genetic instructions for therapeutic antibodies or other proteins. Because the immune system naturally amplifies rare, useful cells after vaccination, even a tiny number of edited stem cells can seed a durable, boostable immune response.

    “The immune system is inefficient in that it produces a vast quantity of cells to protect itself,” said Harald Hartweger, a research assistant professor in Michel Nussenzweig’s Laboratory of Molecular Immunology. “We wanted to take advantage of the immune system’s ability to amplify useful, rare cells.”

    The study, published in Science and titled “B lymphocyte protein factories produced by hematopoietic stem cell gene editing,” demonstrates that CRISPR‑edited HSPCs can mature into B cells that express engineered antibodies upon vaccination. A standard vaccination then acts as the trigger: antigen exposure drives those edited B cells to expand, differentiate into plasma cells, and secrete high titers of the inserted antibody that last long-term.

    According to the paper, as few as ~7,000 edited HSPCs were enough to generate “high titers of long‑lasting protective or therapeutic antibodies and/or cargo proteins.” In mice engineered to produce a broadly neutralizing influenza antibody, this response was strong enough to protect against an otherwise lethal viral infection.

    The platform proved unexpectedly versatile. Edited B cells could also secrete non‑antibody proteins, pointing to potential applications in genetic diseases. And by mixing HSPCs engineered with different antibody instructions, the researchers created immune systems capable of producing multiple antibodies simultaneously, an approach that could limit viral escape in HIV or other rapidly mutating pathogens. Human HSPCs edited using the same strategy produced functional human B cells in an immunodeficient mouse model, offering an early sign of translational feasibility.

    “Our goal is to permanently impact the genome with a single injection, so that the body can make proteins of interest,” Hartweger said. “That protein could be an antibody that’s universally protective against HIV or influenza, but it could also be any therapeutic protein.”

    The team is now moving toward preclinical testing in non‑human primates to evaluate protection against HIV and exploring whether similar strategies could be applied to T cells. The broader vision is a generalizable, long‑term protein‑production platform, one that could support treatments for infectious disease, protein deficiencies, autoimmunity, metabolic disorders, and cancer, according to Hartweger.

    As Nussenzweig puts it, “The present study proposes a workaround for the antibody problem—a way of getting around the possibility that we may never get to a universal HIV vaccine, while still providing a promising, long‑lasting solution.”

    The post Stem Cell Editing Programs the Immune System to Make Own Therapeutic Proteins appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Advances in Stem Cell‑Derived Insulin‑Producing Cells for Type 1 Diabetes https://edusehat.com/en/advances-in-stem-cellderived-insulinproducing-cells-for-type-1-diabetes https://edusehat.com/en/advances-in-stem-cellderived-insulinproducing-cells-for-type-1-diabetes Fri, 17 Apr 2026 03:25:14 +0700 Edusehat Advances, Stem, Cell‑Derived, Insulin‑Producing, Cells, for, Type, Diabetes Researchers at Karolinska Institutet and KTH Royal Institute of Technology have developed an improved method for creating insulin-producing cells from human stem cells. In a newly published study, the team demonstrated that these cells effectively regulate blood sugar levels in laboratory tests and can reverse diabetes in mice.

    “We have developed a method that reliably produces high-quality insulin-producing cells from multiple human stem cell lines,” said Per-Olof Berggren, PhD, professor at the Department of Molecular Medicine and Surgery, Karolinska Institutet. “This opens up opportunities for future patient-specific cell therapies, which could reduce immune rejection.” Berggren and Siqin Wu, PhD, researcher at Spiber Technologies AB (formerly at Karolinska Institutet), are co-corresponding authors of the researchers’ published paper in Stem Cell Reports, titled “An optimized protocol for efficient derivation of pancreatic islets from multiple human pluripotent stem cell lines.”

    Type 1 diabetes (T1D) occurs when the immune system destroys insulin-producing cells in the pancreas, meaning the body can no longer absorb glucose from the blood and regulate blood sugar levels. “In type 1 diabetes (T1D), autoimmune destruction of β cells results in loss of glycemic control,” the authors wrote.

    One possible treatment strategy is to replace these cells with new ones. However, previous methods of producing such cells from stem cells have often yielded mixed results. Stem cell therapy for type 1 diabetes is already being tested in several clinical trials. However, a challenge with previous methods is that the stem cells often develop into a combination of the desired and undesired cell types, increasing the risk of complications. Another challenge is that the insulin-producing cells created are often not mature enough to respond well to glucose.

    “The success of cell therapy for type 1 diabetes (T1D) depends on reliable differentiation of stem cells into functional pancreatic islets,” the authors noted. They pointed out that previous protocols have exhibited variable efficiency across different human pluripotent stem cell (hPSC) lines. “Differentiation beyond the stage (S) 4 pancreatic progenitor (PP) stage frequently yields heterogeneous cultures containing proliferative non-endocrine cells and immature endocrine cells … increasing the risk of cyst or tumor formation,” the team further commented.

    The newly optimized production process reported by Berggren and colleagues yields more mature and purer insulin-producing cells than previous methods. In a laboratory setting, the cells were able to secrete insulin and responded strongly to glucose. When the researchers transplanted these cells into streptozotocin (STZ)-induced diabetic mice, the animals gradually regained the ability to regulate their blood sugar. “By adjusting the culture steps and allowing the cells to form three-dimensional clusters themselves, many unwanted cell types are eliminated and the cells gain a better ability to respond to glucose, according to the researchers. “Single-cell analyses show that the SC-islets are free of non-endocrine cell populations before and after transplantation,” the team stated.

    The transplantation was performed in the anterior chamber of the eye (ACE) which provides a transparent and accessible site for noninvasive monitoring of engrafted SC-islets through the cornea, the team pointed out. Transplantation into this compartment is also straightforward and minimally invasive.  In their paper, the team noted, “Intraperitoneal glucose tolerance tests (IPGTT) at three, four, and six months post-transplantation showed improved glucose handling over time … SC-islet transplantation reversed hyperglycemia by three months, and by five–six months blood glucose levels fell slightly below pre-STZ baselines.”

    Berggren commented, “This is a technique we use to monitor the development and function of the cells over time in a minimally invasive way. We observed that the cells gradually matured after transplantation, retaining their ability to regulate blood sugar for several months, which demonstrates their potential for future treatments.”

    Fredrik Lanner, PhD, professor at the Department of Clinical Science, Intervention and Technology, Karolinska Institutet, and last author of the paper, added, “This could solve several of the problems that have previously hindered the development of stem cell-based treatments for type 1 diabetes. Building on this, we will work towards clinical translation aiming at treating type 1 diabetes.” In their report the authors concluded, “Our protocol generated glucose-responsive SC-islets from all eight hPSC lines tested … demonstrating potential for autologous applications … Our efficient differentiation protocol represents a key step toward autologous cell therapy, though further work is required to realize this goal.”

    The post Advances in Stem Cell‑Derived Insulin‑Producing Cells for Type 1 Diabetes appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Proteins.1 Launches to Develop Single Molecule Protein Amplification Tech for Diagnostics https://edusehat.com/en/proteins1-launches-to-develop-single-molecule-protein-amplification-tech-for-diagnostics https://edusehat.com/en/proteins1-launches-to-develop-single-molecule-protein-amplification-tech-for-diagnostics Fri, 17 Apr 2026 03:25:13 +0700 Edusehat Proteins.1, Launches, Develop, Single, Molecule, Protein, Amplification, Tech, for, Diagnostics Finnish deep-tech startup, Proteins.1, launched with €4.7 million in pre-seed funding, led by Lifeline Ventures and Cloudberry Ventures, with in-kind support from VTT and Business Finland. Harnessing technology transferred from VTT Technical Research Centre of Finland, Proteins.1 is developing a PCR-like enzyme-free, ultra-sensitive amplification platform for the detection of proteins at the single-molecule level. The firm says it aims to transform early disease diagnostics by enabling detection of disease-related molecular warning signals long before there are clinical signs.

    While polymerase chain reaction (PCR) technology has transformed modern diagnostics by allowing tiny amounts of DNA to be amplified into detectable signals, no equivalent amplification method has existed for proteins, which often signal the earliest onset of cancer, neurodegeneration, cardiovascular disease, and inflammatory conditions, the company notes. Proteins.1 aims to leverage its technology to establish a new category of ultra-sensitive protein diagnostics, combining high multiplexing, scalable chip-based detection, and significantly lower capital costs compared to existing systems.

    The patented, physics-based technology introduces cyclic signal amplification for proteins, potentially enabling up to 1,000 times better sensitivity than current gold-standard platforms, Proteins.1 claims. Unlike conventional immunoassays that rely on enzymatic reactions prone to variability and noise, the Proteins.1 approach is solid-state, enzyme-free, and compatible with semiconductor-based photonic detection.

    The platform replaces enzymatic signal amplification with a physics-based magnetic cycling mechanism that repeatedly reads a single captured protein molecule, accumulating signal clarity without increasing background noise. The company says this supports ultra-high sensitivity combined with high multiplexing, potentially enabling the simultaneous measurement of hundreds of biomarkers from a few drops of blood.

    “For decades, diagnostics has been limited not by biology, but by what our instruments can detect,” commented Proteins.1 co-founder and CEO Prateek Singh, who is inventor of the core technology. “The body produces early warning signals long before disease becomes visible. Our mission is to make those signals measurable and actionable, years earlier than today.”

    Built on research conducted at VTT and further validated through European Union breakthrough innovation funding, the technology has been granted U.S. and Finnish patents, and additional international applications are pending. Initially, the company aims to develop research-use-only applications in oncology, neurology, and immunology, before progressing toward regulated clinical diagnostics. “Early detection dramatically improves survival rates in diseases such as cancer and neurodegenerative disorders,” Singh continued. “If we can detect disease at the molecular stage rather than the symptomatic stage, we entirely change treatment possibilities.”

    Proteins.1 plans to expand its engineering and product development team in Finland during 2026–2027, positioning itself as a European hub for next-generation diagnostic technology. “Proteins.1 represents the kind of deep scientific breakthrough that can redefine an entire industry,” said Jyri Engeström at Lifeline Ventures. “The team combines world-class research with proven experience in building and scaling regulated medtech businesses.” Cloudberry Ventures further highlighted the company’s strong alignment with European strengths in photonics, microfabrication, and precision engineering.

    Added Rene Kromhof, at Cloudberry VC, “What sets Proteins.1 apart is a fundamentally new sensing approach. Rather than using enzymes that give you one chance to detect a protein, they use light and thin-film transistors to amplify the signal from a single protein until it rises above the noise. That dramatically improves sensitivity, and ultimately, how early disease can be caught.”

    CEO Prateek Singh has previously raised venture capital for microfluidics ventures and holds multiple patent families. Co-founder and COO Harri Hallila previously built and exited a regulated medical device company. The broader team includes commercial leadership with experience in leading diagnostics platforms.

    The post Proteins.1 Launches to Develop Single Molecule Protein Amplification Tech for Diagnostics appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Brain Circuits Underlying Placebo Pain Relief Identified in Mice https://edusehat.com/en/brain-circuits-underlying-placebo-pain-relief-identified-in-mice https://edusehat.com/en/brain-circuits-underlying-placebo-pain-relief-identified-in-mice Fri, 17 Apr 2026 03:25:12 +0700 Edusehat Brain, Circuits, Underlying, Placebo, Pain, Relief, Identified, Mice Though the placebo effect is a well documented phenomenon, the neurological mechanisms that underlie the process are still not fully understood. Now scientists from multiple institutions led by a team at the University of California San Diego (UCSD) have pinpointed the brain circuitry in mice that they believe is responsible for placebo pain relief. Details of their findings are published in a new paper in the journal Neuron. In it, they describe brain regions that support placebo effects and highlight sites where endogenous opioid neuropeptides send signals that are important for placebo pain relief. 

    The paper is titled “Top-down control of the descending pain modulatory system drives multimodal placebo analgesia.” According to the team, theirs is the first study to establish placebo mechanisms by adapting a protocol used for humans to work in mice. Working alongside labs at the University of Pennsylvania, University of California Irvine, and elsewhere, the UCSD team detected activity in parts of the mouse brain that correspond to those previously implicated in human studies. Furthermore, by precisely mapping neural pathways and brain activity in the mice, the team identified essential roles for neural circuits that link the cortex to the brainstem and spinal cord during placebo pain relief. 

    They also found that training mice to exhibit a placebo effect with one type of pain results in relief from several different types of pain including pain from injuries. That is particularly notable because it has “direct implications for how placebo training in humans might be used to produce resilience to future pain that results from injury,” explained Matthew Banghart, PhD, an associate professor in UCSD’s neurobiology department and lead author on the study. The findings also open a door to “expectancy-driven” placebo effects as a substitute for addictive painkillers, he noted, meaning that it might be possible to use placebo conditioning to train patients to build preemptive resilience to pain.

    Full details of the findings and methods used are provided in the paper. In it, the teams explain that they used sensor technology and a light-activated drug developed in the Banghart lab to study the role of naturally-occurring opioid peptides in the brain. Specifically, they used the sensors to detect opioid peptide signaling in the ventrolateral periaqueductal gray (vlPAG) region, a known hub for pain signaling, during placebo trials. They then used the light-activated drug called photoactivatable naloxone, or PhNX, to establish that these opioid peptides actually drive pain relief in a manner similar to drugs like morphine. The light allowed the scientists control and timing of the opioid signaling interference. Using PhNX, they confirmed that both morphine-induced pain relief and placebo pain relief use the same opioid signaling pathway in the vlPAG region of the brain. 

    Essentially, “we trained a mouse brain to create its own broad-spectrum painkillers on demand, precisely where they are needed to treat pain, without the off-target effects of opioid-based painkillers,” said Janie Chang-Weinberg, a PhD student in the biological sciences graduate program at UCSD and one of the first authors on the study. 

    Future studies planned by the team will dig more deeply into how placebo learning unfolds in the brain and evaluate different placebo training strategies in mice with an eye towards developing protocols that readily translate to produce placebo pain resilience in people living with chronic pain.

    The post Brain Circuits Underlying Placebo Pain Relief Identified in Mice appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Intercellular Communication via Condensate Corona-Nanoparticle Complexes https://edusehat.com/en/intercellular-communication-via-condensate-corona-nanoparticle-complexes https://edusehat.com/en/intercellular-communication-via-condensate-corona-nanoparticle-complexes Fri, 17 Apr 2026 03:25:10 +0700 Edusehat Intercellular, Communication, via, Condensate, Corona-Nanoparticle, Complexes Cells and tissues have a multitude of methods for intercellular communication. Nanoscale assemblies that transfer proteins and RNAs between cells are known, but the impacts of external additions or synthetic materials is unclear.

    Researchers from the University College of Dublin’s Centre for BioNano Interactions (CBNI) explored detailed changes in nanostructure-biological hybrid complexes as they leave one cell and enter another.

    “We had long believed that there are natural couriers and gateways that allow special, very small particulates to communicate in organisms,” said lead author Kenneth Dawson, DPhil, CBNI director.

    The team published their work in a paper titled, “Condensate corona–nanoparticle complexes transfer functional biomolecules between cells” in Nature Materials.

    In rare instances, a subset of nanoparticles that enter a cell undergo an unexpected transformation, acquiring a coating known as a “condensate corona.” This corona allows for regulated entrance into the cell.

    “By gaining access to these natural gateways, it could be possible to ferry ‘toolkits’ of functional biomolecules, for example, extended corrective messages, directly into previously inaccessible areas within cells, and across biological barriers, greatly improving the effectiveness and, importantly, the safety of RNA-, gene- and protein-based therapies,” said lead author associate professor Yan Yan, PhD, UCD School of Biomolecular and Biomedical Science.

    Using “magnetic-cored, silica-shelled nanoparticles precoated with a grafted or adsorbed biomolecular corona,” the researchers created a scaffold that provided the cell with a recognition cue, allowing for the cells to deposit a secondary corona. With magnetic cores, and silica shells that carry fluorescent labels, the nanoparticles are easily controlled, extracted, and visualized.

    Live-cell imaging showed that these additionally transformed nanoparticles were re-exported and retained both their original corona, along with their new cell-derived layer.

    “By combining magnetic core extraction with an optimized pulse–chase regime and post-isolation washing, we obtained highly reproducible particle-complex isolates with minimal background contamination,” the authors wrote. Analysis showed that the cell-derived corona was “solid-like, structurally stable and biochemically robust.”

    They also identified protein profiles using stable-isotope amino acid labelling (SILAC) in the cells producing the corona, followed by mass spec analysis. These proteins have a high affinity for the ER and mitochondria and about 70% of the proteins have been previously associated with mesoscopic intracellular RNA granules.

    “With the prototype in our hands, we were able to break into these communications and understand how biological information is shared between cells. From there, we began to send our own messages via the same system,” Dawson noted.

    In further tests, the team found that within endosomes of the recipient cell, the corona detaches from the core and the fates of the core and corona diverge, with the proteins and RNA components of the corona escaping the endosome—and escaping degradation—to be distributed within and access targets in the cell. They were able to disrupt this process and keep the corona and the attached materials, in the endosome by grafting short peptides onto the coronal surface.

    Utilizing CRISPR-Cas9 they tested the functionality of corona-bound particles that escape the endosome. They generated particle complexes for bioluminescent markers to monitor functionality. Analysis revealed “intact enzymatic activity can be delivered to recipient cells by condensate-borne cargo.”

    The authors explained that together, their data suggest these condensates function as an encoded biomolecular transfer program that are activated by the recipient cell. They wrote: “It is remarkable that such architectures, built entirely from endogenous biomolecules of producer cells, can embody transfer programs that overcome most of the challenges faced within nanoscale therapeutics.”

    “The findings provide a new blueprint for sending strategic and therapeutically effective biological messages to currently inaccessible locations in the body. That points towards a new concept of medicine that could reverse, rather than manage, currently intractable diseases,” concluded Dawson.

    The post Intercellular Communication via Condensate Corona-Nanoparticle Complexes appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Landmark Pancreatic Cancer Trial Highlights Promise of RAS-Targeting Daraxonrasib https://edusehat.com/en/landmark-pancreatic-cancer-trial-highlights-promise-of-ras-targeting-daraxonrasib https://edusehat.com/en/landmark-pancreatic-cancer-trial-highlights-promise-of-ras-targeting-daraxonrasib Thu, 16 Apr 2026 23:50:18 +0700 Edusehat Landmark, Pancreatic, Cancer, Trial, Highlights, Promise, RAS-Targeting, Daraxonrasib Earlier this week, Revolution Medicines reported positive results from a global Phase III trial of its RAS‑targeting inhibitor daraxonrasib (RMC-6236) in metastatic pancreatic ductal adenocarcinoma (PDAC). In the RASolute 302 trial, patients receiving daraxonrasib achieved longer progression‑free survival (PFS) and overall survival (OS) than those on standard cytotoxic chemotherapy.

    The RASolute 302 trial enrolled patients with pancreatic tumors harboring a wide range of RAS variants, including those with RAS G12 mutations (such as G12D, G12V, and G12R), as well as those without an identified RAS mutation. The primary endpoints of the trial were PFS and OS in patients with tumors harboring RAS G12 mutations. Secondary endpoints assessed PFS and OS in all enrolled patients (the intent-to-treat population), including those with tumors with and without (wild type) an identified RAS mutation.

    Daraxonrasib patients achieved a median OS of 13.2 months versus 6.7 months for chemotherapy. The drug was generally well tolerated, with a manageable safety profile and with no new safety signals.

    “With these unprecedented results, daraxonrasib has the potential to achieve our goal of bending the mortality curve in pancreatic cancer. Unlike chemotherapy, daraxonrasib is a RAS-targeted medicine that targets RAS in its active ‘ON’ state, shutting down a key signaling pathway that drives aggressive tumor growth. This is especially important in pancreatic cancer, which is among the most RAS-driven cancers, with more than 90% of tumors harboring a RAS mutation that is the driver of the cancer,” asserted Mark A. Goldsmith, MD, PhD, CEO and chairman of Revolution Medicines.

    Pancreatic cancer carries one of the highest mortality rates of any solid tumor, a consequence of late-stage diagnosis and resistance to standard chemotherapy. In the United States, recent estimates point to roughly 60,000 new cases and nearly 50,000 deaths each year. With most PDAC tumors driven by RAS alterations, the early success of emerging RAS‑targeted strategies hints at how much more may be possible as this therapeutic space continues to expand.

    RAS is the key oncogenic driver of pancreatic cancer. Nearly all RAS mutations occur at KRAS position G12, but RAS mutations in other isoforms and at KRAS positions G13 and Q61 are also observed. Daraxonrasib works by suppressing RAS signaling through inhibition of the interaction between both wild-type and mutant RAS(ON) proteins and their downstream effectors.

    Pancreatic cancer is the most RAS-addicted of all major cancers, with more than 90% of patients harboring tumors driven by mutations in RAS proteins. These mutations span a range of RAS variants that fuel aggressive tumor behavior. Daraxonrasib, a multi-selective inhibitor of RAS(ON) proteins, is the first investigational agent in a novel class of RAS inhibitors designed to address a diverse and broad spectrum of oncogenic RAS drivers.

    “For patients with metastatic pancreatic cancer, new treatment options are urgently needed to increase survival time and improve quality of life,” said Brian M. Wolpin, MD, MPH, professor of medicine at Harvard Medical School, director of the Hale Family Center for Pancreatic Cancer Research at Dana-Farber Cancer Institute, and principal investigator for the RASolute 302 trial. “The widely anticipated results of this study indicate that daraxonrasib provides a clear and highly meaningful step forward for patients with pancreatic cancer who have experienced progression on prior treatment, typically chemotherapy. I believe that this new approach is a very important advance for the field that I expect will be practice-changing for physicians and improve the care for patients with previously treated metastatic pancreatic cancer.”

    Revolution Medicines now intends to submit the drug for approval by regulatory authorities, including the U.S. Food and Drug Administration as part of a future New Drug Application, and for presentation at the 2026 American Society of Clinical Oncology Annual Meeting. 

    The post Landmark Pancreatic Cancer Trial Highlights Promise of RAS-Targeting Daraxonrasib appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Integrated Sample Preparation System Standardizes and Streamlines Pre-Analytical Workflows https://edusehat.com/en/integrated-sample-preparation-system-standardizes-and-streamlines-pre-analytical-workflows https://edusehat.com/en/integrated-sample-preparation-system-standardizes-and-streamlines-pre-analytical-workflows Thu, 16 Apr 2026 23:50:17 +0700 Edusehat Integrated, Sample, Preparation, System, Standardizes, and, Streamlines, Pre-Analytical, Workflows Invivoscribe launched the PrepQuant™ sample preparation platform that integrates nucleic acid extraction, concentration, and quantification with a single automated instrument. The product is designed to standardize sample preparation and simplify pre-analytical workflows to reduce costs and eliminate a primary source of inconsistency in molecular testing.

    Developed in collaboration with Hitachi High-Tech Corporation, PrepQuant combines Invivoscribe’s experience in developing standardized molecular assays, providing global clinical testing services, along with Hitachi High-Tech’s technological and manufacturing capabilities

    The PrepQuant system is assay agnostic, generating highly concentrated genomic DNA and cell free DNA (cfDNA) yields for next-generation sequencing (NGS), qPCR, and digital PCR (dPCR) assays. By consolidating multiple steps in a single platform, the system can lab operating costs, sample variability, and lab bench space, while optimizing tests results.

    “PrepQuant represents a significant advancement in our commitment to standardize the entire testing process, starting with the pre-analytical workflow,” said Jeff Miller, CEO and CSO of Invivoscribe. “This is particularly important in the era of precision medicine, where reliability of measurable residual disease and liquid biopsy results depend markedly on the quality and consistency of the starting material.”

    “The concept for the [product] was driven directly by insights from LabPMM, our global network of testing laboratories,” added Jordan Thornes, vice president, global clinical lab operations. “Our teams recognized the limitations of currently available automated instruments, particularly the labor-intensive nature and increased risk of errors associated with running three separate protocols across multiple instruments. This all-in-one system was designed to reduce costs, while addressing those challenges and significantly improving operational efficiency.”

    The PrepQuant is designed and validated for use with blood, plasma, and bone marrow specimens, with ongoing development for additional specimen types. Invivoscribe will officially unveil the product at the American Association for Cancer Research (AACR) Annual Meeting in San Diego, at booth #3459 from April 19–22.

     

    The post Integrated Sample Preparation System Standardizes and Streamlines Pre-Analytical Workflows appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AACR 2026 Chairs Identify Themes and Highlights from the Conference https://edusehat.com/en/aacr-2026-chairs-identify-themes-and-highlights-from-the-conference https://edusehat.com/en/aacr-2026-chairs-identify-themes-and-highlights-from-the-conference Thu, 16 Apr 2026 23:50:15 +0700 Edusehat AACR, 2026, Chairs, Identify, Themes, and, Highlights, from, the, Conference The American Association for Cancer Research (AACR) Annual Meeting kicks off this weekend in San Diego. A whirlwind of sessions, keynotes, fireside chats, posters, and exhibitors, the meeting is THE annual event for the cancer community.

    Before the conference, GEN spoke with AACR program chairs Paul S. Mischel, MD, Professor and Vice Chair for Research for the Department of Pathology at Stanford Medicine of Stanford University and Alice T. Shaw, MD, PhD, Chair of the Department of Medical Oncology and the Chief of Strategic Partnerships at Dana-Farber. In this interview, they share their perspectives on the event, what attendees should be looking out for, and what they, personally, are most looking forward to.

    This interview has been edited for length and clarity.

     

    GEN: What did you feel were some of the most important themes to include in the conference program?

    Shaw: First of all, it’s been such an honor for me to work with Paul as well as our president, Lillian Siu, MD. We had an expert program committee and incredible staff at AACR who all helped shape the program.

    This year’s annual meeting feels more meaningful than ever, because of everything going on in the world, including funding challenges, challenging geopolitics, and everything else. It has felt even more important that we have this time to bring together our global community of cancer researchers and investigators.

    When Paul and I met last summer, we felt strongly that this meeting was not just about designing an incredibly strong scientific program to showcase the science and all of the innovation, but we wanted to make a point to demonstrate to the audience, and the world, the tangible benefits of scientific research to patients with cancer, and to highlight how all the research we do is done with an eye toward improving the lives of patients with cancer.

    We intentionally planned a scientific program with patients and patient impact front and center and have tried to incorporate the patient perspective and even patient voices in some sessions—to emphasize that science drives impact for patients.

    Mischel: When we started about a year ago, our conviction—that there probably has never been a more important year for an AACR meeting—grew over the year. This organization is a beacon of light at a time in which there’s been extraordinary progress in cancer, and [there is] the potential to really make a difference in patients’ lives at the face of some very major headwinds. What we’re seeing is a level of enthusiasm and engagement in coming together in the community that’s saying: we won’t be stopped in making a difference for patients with cancer. And there were a number of themes that were central to this meeting.

    For example, precision—that you can use information about patients to identify what’s gone wrong and how to develop therapies based upon deep molecular knowledge. Partnership—The growing recognition of how we work together to make a difference for patients. It’s not a winner-take-all strategy. It’s not a race to the top for individuals. It’s a race to the top for people with cancer. And we do it effectively by joining hands to make a difference for patients. And global work—another major theme that we’re really talking about this year, because together we can make a real difference for people with cancer.

    We work together with people that span an enormous range of disciplines and expertise. A number of themes came front and center during the year. The technologies to interrogate what’s happening in human beings, whether it’s in their tissue, their blood, or their images—it’s nothing short of breathtaking. The ability to either forestall cancer by detecting if it’s going to happen, or catch it early, or monitor our most effective treatments is really changing the game. New modalities for developing treatments. We’ve heard a lot about harnessing the immune system and about the development of small molecules. There’s all kinds of new chemistry, molecular glues and degraders that are leading the way. And they’re tied to deep investigations into the fundamental biology.

    AI is changing the game as well. We are very excited that we’ve brought together perhaps the most interesting AI sessions that you can imagine, that talk about all of the ways that AI can be used—not like an oracle but actually in partnership with helping make a difference for patients, whether it be in the diagnostics or the development of therapeutics. AI is only beginning to be tapped to help us think about how to integrate knowledge across all of these domains. And it goes beyond that because it’s not only about the molecular composition of a person’s tumor, it’s about a human being, what they eat, where they live, what they do, and various other social determinants of health that might increase risks of cancer. A lot of attention is paid in the meeting to that aspect of it as well. So, I think we’re in for an incredibly interesting meeting.

    Shaw: One of the themes that came right out from everyone on the program committee was how important it was to highlight AI; I think everyone believes that AI is going to be transformative and it’s going to impact all aspects of cancer research and clinical care in the coming years. We had a number of AI experts on our program committee. They really helped embed AI topics throughout the scientific and also the educational program.

    In fact, when Paul and I were planning the opening plenary session, we really wanted one of the opening plenary speakers to be able to speak on AI. So, we have Regina Barzilay, PhD, from MIT, who’s going to speak on her work in the AI space, both in terms of drug discovery and all the way out to clinical applications. We also have an AI-focused plenary session all unto itself as well, to drive home the importance of AI tools and technologies. It is incredible how AI is already being used in terms of foundational discovery and in terms of real-world, clinical data mining and implementation. And Paul already mentioned genomics, precision medicine, biomarker discovery, histopathology, radiology, all of which are already being impacted by AI.

    Mischel: One of the things that is perhaps most stunning is this idea that you might be able to prevent cancer. We already see real world examples of that with things like HPV vaccines. The work is advancing so quickly that it might be possible to build vaccines against [something] that might prevent people who are at high risk of developing cancer from getting those cancers. Our colleague and AACR President Lillian Siu, MD, has a presidential symposium focused on that. What I hope that we’re getting across is the real scale and power of the work that’s being presented and the way that it crosses so many disciplines at this meeting.

    Shaw: We are going to have a large focus, as we usually do, on molecularly targeted therapies. We have several sessions on the basic research side, but also in the clinical trial sessions around targeting RAS. RAS, of course, is the most commonly mutated oncogene in human cancer and has been undruggable for decades.

    In the last five to 10 years, we now finally have small molecule therapies that can target different RAS mutations. In fact, you may have just heard the news about a new RAS inhibitor, daraxonrasib from Revolution Medicines, and pivotal Phase III trial data in previously treated pancreatic cancer. This was an incredibly positive study, doubling overall survival. Not even knowing those results, though, we already had planned quite a lot around RAS.

    In that context of precision therapies, I also want to mention that I’m excited about one of our discovery science plenaries on Saturday that is going to focus on minimal residual disease (MRD) in solid tumors. Here the question is, if we have such incredibly effective targeted therapies for oncogene-driven cancers like RAS, EGFR, and ALK, why aren’t we curing more patients who have these types of cancers? We believe that a lot of the reason is because we can’t eliminate every cancer cell. And if we could just understand what allows those residual cells to survive, perhaps we could eradicate those and then be on the road to curing patients who have advanced disease. So, this whole plenary will focus on the science around MRD and how that then leads to clinical applications as well.

    Mischel: Fundamental science is deeply central to this process. We frame a lot of things in terms of what it means for patients’ lives. I think an important part of this meeting is also integrating how those discoveries really flow from the work in fundamental science. For example, in the opening plenary, we’re going to be hearing about how tumors change their stripes effectively to become resistant to treatments, the lineage plasticity, this idea that they adopt new states to become resistant. And then what can you do about it?

    When people used to be diagnosed with terminal cancer, it meant that they were going to die soon, and now people can be diagnosed with terminal cancer and live for years. That’s stunning. And that is happening because of cancer research and the integration of cancer research all the way from the most mechanistic to the most applied. One of the deepest themes of this meeting comes into this concept of partnership, that we highlight the critical nature of each component and the integration of those components, all the way from fundamental discovery to translation to patients.

     

    GEN: What are some of the biggest scientific challenges you’re seeing right now in cancer biology that you think people will be discussing at the meeting?

    Mischel: Cancer is hard because it is evolution on steroids. The mechanism that I study—extrachromosomal DNA, the ability of cancer cells and tumors to change quickly to resist treatment—is all about that. And it comes from us; it’s our cells that have gone bad. We have to find ways to show how they’re different and target those differences. We’re getting better at it through our understanding of science.

    Shaw:  I am someone who sits between basic research and the clinic, so I do a lot of translational research. What the AACR meeting does well is gets at this key challenge around how we translate basic discoveries into the clinic. We all just want better cancer therapies for our patients. There are many aspects that really make the translation of discoveries difficult, and these will come out in various forms at the meeting. We’ve been talking about how hard it is to understand the biology, and to identify and validate new targets for drug discovery, for cancer treatments in the future.

    I have spent some time on the industry side, so I also recognize how challenging it is, even when you have what you think is a perfect target, to drug that target. At the annual meeting, we’re going to talk a lot about different modalities, ways of thinking about going after what we believe are important targets, be it a small molecule or maybe it’s a new degrader or maybe it’s some other very complicated biologic.

    I want to emphasize that to use or to identify the optimal modality requires that we understand the biology and the science behind it. The other challenge that I’ve seen in the translational space is around identifying which patients are going to benefit from a new therapy. A good example of where we’ve seen struggles is immunotherapy and identifying novel immunotherapy combinations and which ones have robust activity. But we can’t tell exactly which patients are deriving that benefit. Oftentimes with no biomarker to guide us, we can’t move forward with what could be a promising combination. At the annual meeting, we try to highlight a lot of these correlative or translational biomarker studies from early phase clinical trials.

    The last thing I’ll mention is around how we use preclinical models most effectively to predict what’s going to be a promising new therapy. Often, these models are just models; they’re simpler than human cancers. They can’t recapitulate the complexity of human biology, and they can lead us the wrong way. For example, to overestimate how effective a therapy may be. Fine-tuning our models and making them as predictive as possible is a key challenge.

    Mischel: Data seems to suggest that very often you might need to combine agents to make differences for patients. And that of course makes enormous sense from a biological standpoint, but it’s much harder to do when you start thinking about how you design the trials to do that. It’s a slow process. And so, there is increasing recognition of the need to figure out how to combine agents and hopefully ways to figure out how practically to begin to test them more effectively and in a more cost-effective fashion.

     

    GEN: What have been some of the biggest advances since the last meeting?

    Mischel: I just keep coming back to RAS because it’s such a big deal. An undruggable target that we’re now seeing a huge change in. It’s a huge deal.

    Shaw: I would agree with that. And it’s not simply the RAS inhibitor itself. Many of us believe that that is just the start of how we most effectively treat RAS-driven cancers. We need the best RAS inhibitor to serve as an anchor and then we will build these combinations around that which will hopefully be even more effective and allow us to maximally cytoreduce or debulk cancers and then allow us to take in other even higher order combinations.

    We have sessions this year all around RAS-mutant cancers. We have a designated session just focused on pancreatic cancer biology, because understanding that biology well is going to be critical to developing these types of combination approaches.

    The other thing that’s exciting—and this space is always evolving—is a plenary session on innovative new therapeutic modalities. This session will focus on a couple key modalities that are already transforming the space. Antibody-drug conjugates (ADC), for example. They are basically entering every therapeutic space that we have in cancer [and] understanding of the biology around how you’re targeting certain tumor-selective antigens.

    Also, the design of the ADC itself can be very, very sophisticated and can be tweaked to further enhance activity. We’ll have some great talks around the next generation of ADCs that are going to be even more effective and even safer than what we currently have.

    And in that same session around innovative modalities, we’ll also have talks around immune cell engagers; also, new data and next generation immune cell engagers that have built upon the early data with the first-generation immune cell engagers. The other very innovative new therapy that we will highlight, even in more detail than last year, is around radioligand therapies—a way to selectively target tumor cells with radiation. Unlike ADCs where the payload is chemotherapy, here the payload is radiation therapy. We’ve already seen really that these radioligand therapies are incredibly important for patients; they are coming out in all different therapeutic spaces. We thought it was important to highlight the latest advances in radioligand therapies, and we also have some education sessions so that physicians and scientists understand the basics around this innovative and exciting modality.

    Mischel: The concepts of glues and degraders open the therapeutic landscape in a very different way. In many ways, the landscape has been limited to enzymes that you can inhibit, and not all good cancer targets are going to be enzymes that you can inhibit, and these glues and degraders change what you can do, whether it’s getting rid of them, moving them, giving them new functions. It’s a very powerful technology that is getting ready to make an enormous difference in clinic.

    Shaw:  In the opening plenary session, George Winter will speak on glues and degraders. I also wanted to highlight the “New Drugs on the Horizon” sessions. We do this every year at the annual meeting. I love these sessions because they are first time disclosures of novel cancer therapies that have just entered the clinic or they’re about to enter the clinic. These talks go deep into the biology of the disease and how the drug was discovered and developed into early clinical plans. Several talks in this session this year are going to feature molecular glue degraders. That will be a nice way to tie together this theme around the degraders and the power of this new modality.

    Mischel: One other thing I want to squeeze in is why on Earth are younger people getting cancer, particularly colorectal cancer? That’s really disturbing. We have sessions that are data rich that go right at that, and the answers are interesting.

     

    GEN: Will there be any programming at the meeting that addresses the current state of funding?

    Shaw:  We’re fortunate that Tony Letai, MD, PhD, the NCI director, is attending and speaking at the meeting in our opening ceremony on Sunday. He’s also participating in a workshop that we’re holding on grant writing and the scientific review process. On Monday, he will give an NCI director’s address and participate in a fireside chat where I’m sure he’s going to get a lot of questions around funding.

    There are also sessions within the science and health policy track at the meeting that are going to focus on federal funding of grants. There is even a researcher town hall that’s really going to talk a lot about this.

     

    GEN: Do you have any advice for young cancer researchers that may be attending AACR for the first time?

    Mischel: I have two bits of advice. One of them is to know that what you’re doing is incredibly important. You are welcome. You’re one of us, you’re important. Do what you need and go forward because the work that you’re doing is going to matter an enormous amount. The second thing is do not be afraid. Do not think that the senior people at the meeting, the “bigwigs,” are too busy for you. Do not think they do not want to meet you, because they do. You’re the future. Go up, introduce yourself, say hello, tell us who you are.

     

    GEN: What are things you are looking forward to outside of the sessions? 

    Shaw:  I love the AACR annual meeting because it’s such an opportunity not just to learn, but to network and reconnect with friends and collaborators who you may not have seen in a while. I also think it’s a great venue for many of us to have formal sit-down meetings with industry partners and talk through the latest data that were just presented and discuss new collaborations. I personally am looking forward to the 5K race that Paul and I are speaking at. I’m going to try to run the race! One of my sons runs marathons and I thought, well, the least I can do is try and run a 5K.

    Mischel: I’m looking forward to having a drink with Alice after the meeting ends and debriefing on putting this meeting together, which has been an absolute pleasure. I wish I were running the 5K race. I’m doing an education session at that time. I’m looking forward to meeting the students. There are these brilliant young people from all around the world and they’re just at the start of their career and they draw inspiration from this meeting, and I really enjoy it when they come up to me and I get to meet them. You see the brilliance and excitement in these people’s faces. And I’m looking forward to that.

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    ]]>     Fujifilm Biotechnologies Opens New QC Lab in Denmark https://edusehat.com/en/fujifilm-biotechnologies-opens-new-qc-lab-in-denmark https://edusehat.com/en/fujifilm-biotechnologies-opens-new-qc-lab-in-denmark Thu, 16 Apr 2026 05:45:10 +0700 Edusehat Fujifilm, Biotechnologies, Opens, New, Lab, Denmark Fujifilm Biotechnologies, a CDMO, celebrated the opening of its new, 2,000‑square‑meter quality control (QC) laboratory at its Hillerød, Denmark, commercial‑scale manufacturing site. The expanded QC footprint will enable bioassay and virology operations and support the site’s planned expansion, according to the company.

    The laboratory features ventilation systems, personnel, and material airlocks, and an open‑plan layout. The space supports approximately 100 quality team members to conduct viral safety testing for drug substance/product release, scale capacity for complex cell‑based potency and ELISA methods, and perform raw material and critical total organic carbon cleanability studies to accelerate future partner campaigns.

    The QC laboratory also incorporates robotics and an ongoing LIMS implementation across the company’s global network of sites to enable digital harmonization and data integrity.

    The company doubled its Hillerød capacity in 2024 from six to 12 x 20,000 L mammalian cell culture bioreactors, increasing the complexity and volume for QC testing. The expanded production scale required expanded QC capabilities and advanced analytical equipment to support current operations and anticipated future demand. The QC lab is housed within a new 7,600-square-meter building that also features employee amenities, office and collaboration space, utility services, and an emergency generator to ensure uninterrupted operations and timely delivery of critical test results.

    Construction of the lab was completed in last month and subsequently received approval from the Danish Medicines Agency (DKMA) following an on‑site inspection. Laboratory operations will begin in May 2026.

    The new QC laboratory is part of Fujifilm Biotechnologies’ kojoX modular, connected network of manufacturing facilities, where harmonized equipment, layouts, methods, and digital systems are used to enable cross‑site workflows and consistent application of quality standards across regions, explains Christian Houborg, senior vice president and Hillerød site lead.

    “Today, we are opening a world-class GMP-approved QC laboratory to elevate our quality control and be ready for the upcoming expansion, thereby continuing to manufacture advanced biological treatments for patients with severe diseases, such as cancer and rare autoimmune diseases. Together, we’re making a measurable impact for patients and partners around the world,” he said.

     

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    ]]>     Pregnancy Sickness Study Identifies New Genetic Links https://edusehat.com/en/pregnancy-sickness-study-identifies-new-genetic-links https://edusehat.com/en/pregnancy-sickness-study-identifies-new-genetic-links Thu, 16 Apr 2026 05:45:09 +0700 Edusehat Pregnancy, Sickness, Study, Identifies, New, Genetic, Links The University of Southern California (USC) research team that identified the hormone-encoding gene GDF15 as a key driver of pregnancy sickness has identified nine additional genes linked to its most severe form, hyperemesis gravidarum (HG). Six of the identified genes had not been previously linked to the condition.

    The Keck School of Medicine of USC team and international collaborators conducted a genome-wide association study (GWAS), scanning the entire genome for differences between women who developed HG during pregnancy and those who did not. They analyzed data from more than 10,000 women with the condition and more than 450,000 controls across European, Asian, African, and Latino ancestries. Their findings offer new clues about the condition and new hope for those affected.

    Marlena Fejzo, PhD, a clinical assistant professor of population and public health sciences in the Center for Genetic Epidemiology at the Keck School of Medicine, led the present study and earlier research linking GDF15 to HG. Fejzo told GEN, “The study is much larger than previous studies and on a more diverse population allowing for identification of new genes associated with HG … The new genes give us new directions to explore for prediction, diagnosis, treatment, and response to therapies.”

    Fejzo is first author of the team’s published report in Nature Genetics (“Multi-ancestry genome-wide association study of severe pregnancy nausea and vomiting”), in which the team stated, “Potential roles for candidate genes in appetite, insulin signaling, and brain plasticity provide pathways to explore etiological mechanisms and therapeutic avenues.”

    HG, which affects about 2% of women, causes nausea and vomiting so severe that eating can become extremely difficult. “Most pregnancies are affected by nausea and vomiting (NVP), but in 0.3–10.8% of pregnancies the symptoms can be severe enough to cause maternal weight loss and adverse maternal and fetal outcomes,” the authors wrote. HG in its most severe form can even be life threatening.

    HG was long misunderstood and often dismissed as psychological, growing evidence shows that it has a strong biological and genetic basis and can lead to severe malnourishment, putting both mother and baby at risk. Current treatments for HG are frequently ineffective in improving patient symptoms, the authors further pointed out, and so increase the risk of pregnancy termination, postpartum depression, and suicidal ideation, along with other maternal and offspring comorbidities. “Therefore, understanding of HG etiology is critical to begin to address the negative impact severe NVP has on maternal and child health.”

    While historical hypotheses have previously centered around human chorionic gonadotropin (hCG), recent large-scale genetic studies have implicated the GDF15 gene encoding growth differentiation factor-15—a hormone associated with nausea and vomiting, the authors further pointed out. Earlier research by Fejzo and an international team had shown that the link between HG and GDF15 lies in women’s sensitivity to the hormone. They found that women exposed to lower levels of the hormone before pregnancy because of a mutation in the gene experience more severe symptoms, while women exposed to higher levels of the hormone before pregnancy have less severe nausea and vomiting symptoms.

    “GDF15 was identified as the greatest genetic risk factor for HG in both a genome-wide and an exome-wide association study, and a rare mutation in GDF15 was associated with a greater than tenfold increased risk for HG,” the scientists noted in their newly reported study. Fejzo explained to GEN, “The mutation in GDF15 is rare. People who carry the mutation have abnormally low levels of GDF15 when they are not pregnant and that increases their risk of being hypersensitive to it during pregnancy when it is produced in massive amounts by the placenta.”

    Commenting on their prior work implicating a role for GDF15 and HG, Fejzo further explained to GEN, “In our first GWAS study we found the association between the GDF15 gene and HG. Next, we published a whole-exome sequencing study that identified a mutation in GDF15 associated with HG. Then we published our study in Nature which provided strong evidence that hypersensitivity to the rise of GDF15 in pregnancy (due to low pre-pregnancy GDF15 in circulation) is the main driver of the condition.”

    For their newly reported study the researchers carried out a multi-ancestry genome-wide association study of 10,974 HG/excessive vomiting in pregnancy cases and 461,461 controls across European, Asian, African, and Latino ancestries from nine contributing studies.

    The results identified 10 genes that were linked to HG, including four that had previously been identified, and six new genes. “Because this is the largest study of HG ever conducted, we’ve been able to tease out important new details that were previously unknown,” said Fejzo. “The fact that we’ve studied women from multiple ancestry groups suggests that these results may be generalizable across a broad population.”

    The four genes previously identified were growth differentiation factor 15 (GDF15), GFRAL, which produces the receptor for the GDF15 hormone of the same name, and IGFBP7 and PGR, both of which are involved in development of the placenta. The strongest link by far was to GDF15, which rises sharply during pregnancy. “We know that GDF15 and it’s receptor GFRAL are the main drivers and are in a signaling pathway that causes aversions, nausea, and vomiting,” Fejzo told GEN. “More work needs to be done to explore the other associations, but since studies suggest manipulating progesterone and/or IGFBP7 may not be safe in pregnancy, current studies are focusing on the GDF15 pathway.”

    The six newly identified genes offer further clues that might help explain the basis of HG or point to new ways of treating it. They include FSHB, TCFL72 SLITRK1, SYN3, IGSF11, and CDH9. “Now that we’ve more than doubled the genes associated with HG, we can dig deeper into the biology behind this condition, as well as new possible pathways for treating it,” Fejzo said. Speaking to GEN, the researchers noted, “Because the new associations are novel, we need to understand the roles they may play in normal pregnancy and then compare that to pregnancies affected by HG.”

    Of the newly identified genes, TCF7L2 stands out because it is one of the strongest genetic risk factors for type 2 diabetes and is also associated with gestational diabetes. “This is a brand-new target, and it’s not yet clear what it’s doing in pregnancy,” Fejzo said. In further commentary to GEN, Fejzo added, “The TCF7L2 gene is a type 2 diabetes-associated gene and a transcription factor that may control glucagon-like peptide-1 (GLP-1) expression and has been associated with liraglutide effects resulting in greater weight loss in obesity. So understanding its role in that rapidly evolving therapeutic arena has potential.”

    Several of the other genes identified are involved in appetite and nausea, as well as brain plasticity, or how the brain learns and adapts to new information. Fejzo suggests the brain may learn to associate certain foods with feeling sick, leading to strong, lasting aversions during pregnancy. More research is needed to explore this possibility. “Other genes are associated with learning flexibility so we hypothesize that they may play a role in conditioned taste aversion and may provide new targets to alter or dampen learned aversions,” Fejzo told GEN. Historically, people believed the pregnancy hormone hCG was the cause, but we found no evidence to support that and instead, fascinatingly, we found a link to the follicle stimulating hormone receptor.”

    Of the ten candidate genes six—GDF15, GFRAL, IGFBP7, PGR, TCF7L2 and SYN3—have been linked with cachexia—a wasting condition with similar symptoms to HG, including loss of appetite, weight loss and muscle wasting, the scientists noted. “Manipulation of GDF15, GFRAL, IGFBP7, PGR and TCF7L2 in animal models has shown effectiveness in reducing symptoms of cachexia. Thus, assuming analogous functions for these factors in HG, there is both genetic and biological support for causal and potentially reversible contributions for these genes in NVP.”

    The researchers also found that some genes linked to HG were associated with other pregnancy outcomes. “This study also identified individual associations between risk genes and adverse outcomes including shorter pregnancy duration, pre-eclampsia, and birth weight,” they noted.

    Several medications are available for treating HG, but even the most effective, Zofran, only partly relieves symptoms for about half of patients. The new findings reveal new potential treatment targets and could possibly also help match existing medications to patients based on their genetic profiles. “The ten genetic associations provide intriguing avenues to advance our understanding and pursue therapeutic pathways for a common pregnancy condition that in its most severe form is associated with substantial morbidity and even mortality for mothers and exposed offspring,” the scientists concluded.

    Fejzo and her team just received approval to launch a clinical trial of metformin, a widely used diabetes medicine that increases GDF15 levels. The study will test whether taking metformin before pregnancy can desensitize women to the hormone, potentially reducing nausea and vomiting or preventing HG in women who have had it before. GEN was told, “We will be initiating a clinical trial to increase GDF15 prior to pregnancy in patients with a history of HG and planning to conceive to desensitize them to the hormone’s rapid rise in early pregnancy. We and others have shown preliminary evidence that this approach may work as in our retrospective study pre-pregnancy metformin use was associated with a significant reduction in HG risk.”

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    ]]>     No one’s sure if synthetic mirror life will kill us all https://edusehat.com/en/no-ones-sure-if-synthetic-mirror-life-will-kill-us-all https://edusehat.com/en/no-ones-sure-if-synthetic-mirror-life-will-kill-us-all Thu, 16 Apr 2026 02:15:02 +0700 Edusehat one’s, sure, synthetic, mirror, life, will, kill, all For four days in February 2019, some 30 synthetic biologists and ethicists hunkered down at a conference center in Northern Virginia to brainstorm high-risk, cutting-­edge, irresistibly exciting ideas that the National Science Foundation should fund. By the end of the meeting, they’d landed on a compelling contender: making “mirror” bacteria. Should they come to be, the lab-created microbes would be structured and organized like ordinary bacteria, with one important exception: Key biological molecules like proteins, sugars, and lipids would be the mirror images of those found in nature. DNA, RNA, and many other components of living cells are chiral, which means they have a built-in rotational structure. Their mirrors would twist in the opposite direction. 

    Researchers thrilled at the prospect. “Everybody—everybody—thought this was cool,” says John Glass, a synthetic biologist at the J. Craig Venter Institute in La Jolla, California, who attended the 2019 workshop and is a pioneer in developing synthetic cells. It was “an incredibly difficult project that would tell us potentially new things about how to design and build cells, or about the origin of life on Earth.” The group saw enormous potential for medicine, too. Mirror microbes might be engineered as biological factories, producing mirror molecules that could form the basis for new kinds of drugs. In theory, such therapeutics could perform the same functions as their natural counterparts, but without triggering unwelcome immune responses. 

    After the meeting, the biologists recommended NSF funding for a handful of research groups to develop tools and carry out preliminary experiments, the beginnings of a path through the looking glass. The excitement was global. The National Natural Science Foundation of China funded major projects in mirror biology, as did the German Federal Ministry of Research, Technology, and Space.

    By five years later, in 2024, many researchers involved in that NSF meeting had reversed course. They’d become convinced that in the worst of all possible futures, mirror organisms could trigger a catastrophic event threatening every form of life on Earth; they’d proliferate without predators and evade the immune defenses of people, plants, and animals. 

    “I wish that one sunny afternoon we were having coffee and we realized the world’s about to end, but that’s not what happened.”

    Kate Adamala, synthetic biologist, University of Minnesota

    Over the past two years, they’ve been ringing alarm bells. They published an article in Science in December 2024, accompanied by a 299-page technical report addressing feasibility and risks. They’ve written essays and convened panels and cofounded the Mirror Biology Dialogues Fund (MBDF), a broadly funded nonprofit charged with supporting work on understanding and addressing the risk. The issue has received a blaze of media attention and ignited dialogues among not only chemists and synthetic biologists but also bioethicists and policymakers.  

    What’s received less attention, however, is how we got here and what uncertainties still remain about any potential threat. Creating a mirror-life organism would be tremendously complicated and expensive. And although the scientific community is taking the alarm seriously, some scientists doubt whether it’s even possible to create a mirror organism anytime soon. “The hypothetical creation of mirror-­image organisms lies far beyond the reach of present-day science,” says Ting Zhu, a molecular biologist at Westlake University, in China, whose lab focuses on synthesizing mirror-image peptides and other molecules. He and others have urged colleagues not to let speculation and anxiety guide decision-making and argued that it’s premature to call for a broad moratorium on early-stage research, which they say could have medical benefits. 

    But the researchers who are raising flags describe a pathway, even multiple pathways, to bringing mirror life into existence—and they say we urgently need guardrails to figure out what kinds of mirror-biology research might still be safe. That means they’re facing a question that others have encountered before, multiple times over the last several decades and with mixed results—one that doesn’t have a neat home in the scientific method. What should scientists do when they see the shadow of the end of the world in their own research? 

    Looking-glass life

    The French chemist and microbiologist Louis Pasteur was the first to recognize that biological molecules had built-in handedness. In the late 19th century, he described all living species as “functions of cosmic asymmetry.” What would happen, he mused, if one could replace these chiral components with their mirror opposites? 

    Scientists now recognize that chirality is central to life itself, though no one knows why. In humans, 19 of the 20 so-called “standard” amino acids that make up proteins are chiral, and all in the same way. (The outlier, glycine, is symmetrical.) The functions of proteins are intricately tied to their shapes, and they mostly interact with other molecules through chiral structures. Almost all receptors on the surface of a cell are chiral. During an infection, the immune system’s sentinels use chirality to detect and bind to antigens—substances that trigger an immune response—and to start the process of building antibodies. 

    By the late 20th century, researchers had begun to explore the idea of reversing chirality. In 1992, one team reported having synthesized the first mirror-image protein. That, in turn, set off the first clarion call about the risk: In response to the discovery, chemists at Purdue University pointed out, briefly, that mirror-life organisms, if they escaped from a lab, would be immune to any attack by “normal” life. A 2010 story in Wired highlighting early findings in the area noted that if a such a microbe developed the ability to photosynthesize, it could obliterate life as we know it. 

    The synthetic biology community didn’t seriously weigh those threats then, says David Relman, a specialist who bridges infectious disease and microbiology at Stanford University and a trailblazer in studying the gut and oral microbiomes. The idea of a mirror microbe seemed too far beyond the actual progress on proteins. “This was almost a solely theoretical argument 20 years ago,” he says. 

    Now the research landscape has changed. 

    Scientists are quickly making progress on mirror images of the machinery cells use to make proteins and to self-replicate. Those components include DNA, which encodes the recipes for proteins; DNA polymerases, which help copy genetic material; and RNA, which carries recipes to ribosomes, the cell’s protein factories. If researchers could make self-replicating mirror ribosomes, then they would have an efficient way to produce mirror proteins. That could be used as a biological manufacturing method for therapeutics. But embedded in a self-­replicating, metabolizing synthetic cell, all these pieces could give rise to a mirror microbe. 

    When synthetic biologists convened in Northern Virginia in 2019, they didn’t recognize how quickly the technology was advancing, and if they saw a threat at all, it may have been obscured by the blinding appeal of pushing the science forward. What’s become apparent now, says Glass, is that scientists in different disciplines, all related to mirror life, were largely unaware of what other scientists had been doing. Chemists didn’t know that synthetic biologists had made so much progress on creating mirror cells with natural chirality from scratch. Biologists didn’t appreciate that chemists were building ever-larger mirror macromolecules. “We tend to be siloed,” Glass says. And nobody, he says, had thought to seriously examine the immune system concerns that had already been raised in response to earlier work. “There was not an immunologist or an infectious disease person in the room,” Glass says, reflecting on the 2019 meeting. “I may have come closest, given that I work with pathogenic bacteria and viruses,” he adds, but his work doesn’t address how they cause infections in their hosts.

    on the left, a hand with petri dish and the same image inverted on the right
    GETTY IMAGES

    These scientists also didn’t know that around the same time as their meeting, another conversation about mirror life was happening—a darker dialogue that was as focused on danger as it was on discovery. Starting around 2016, researchers with a nonprofit called Open Philanthropy had begun compiling research files on catastrophic biological risks. The organization, which rebranded as Coefficient Giving in 2025, funds projects across a range of focus areas; it adheres to a divisive philanthropic philosophy called effective altruism, which advocates giving money to projects with the highest potential benefit to the most people. While that might not sound objectionable, critics point out that the metrics devotees use to gauge “effectiveness” can prioritize long-term solutions while neglecting social injustices or systemic problems. 

    Someone in Open Philanthropy’s bio­security group had suggested looking into the risks posed by mirror life. In 2019 the organization began funding research by Kevin Esvelt, who leads the Sculpting Evolution group at the MIT Media Lab, on biosecurity issues, including mirror life. He began reading up to see whether mirror life was something to worry about.

    Esvelt made waves in 2013 for pioneering the use of CRISPR to develop a gene drive, a technology that could spread genetic changes introduced into a living organism through a whole population. Researchers are exploring its use, for example, to make mosquitoes hostile to the parasite that causes malaria—and, as a result, lower their chance of spreading it to humans. But almost immediately after he developed the tool, Esvelt argued against using it for profit, at least until proper safeguards could be set and its use in fighting malaria had been established. “Do you really have the right to run an experiment where if you screw up, it affects the whole world?” he asked, in this magazine, in 2016. At the Media Lab, Esvelt leads efforts to safely develop gene drives that can be deployed locally but prevented from spreading globally. 

    Esvelt says he’s often thinking about the security risks posed by self-sustaining genetically engineered technologies, and research led him to suspect that the threat of mirror organisms hadn’t been seriously interrogated. The more he learned about microbial growth rates, predator-prey and microbe-microbe interactions, and immunology, the more he began to worry that mirror organisms, if impervious to the innate defenses of natural ones, could cause unstoppable infections in the event that they escaped the lab. 

    Even if the first experimental iteration of such a germ were too fragile to survive in the environment or a human body, Esvelt says, it would be a light lift to genetically engineer new, more resilient versions with existing technology. Even worse, he says, the results could be weaponized. The possible path from 2019 to global annihilation seemed almost too direct, he found. 

    But he wasn’t an expert in all the scientific fields involved in research on mirror life, so he started making calls. He first described his concerns to Relman one night in February 2022, at a restaurant outside Washington, DC. Esvelt hoped Relman would tell him he was wrong, that he’d missed something over the years of gathering data. Instead, he was troubled. 

    The concern spreads

    When Relman returned to California, he read more about the technology, the risks, and the role of chirality in the immune system and the environment. And he consulted experts he knew well—ecologists, other microbiologists, immunologists, all of them leaders in their fields—in an attempt to assuage his concerns. “I was hoping that they’d be able to say, I’ve thought about this, and I see a problem with your logic. I see that it’s really not so bad,” he says. “At every turn, that did not happen. Something about it was new to every person.” 

    The concern spread. Relman worked with Jack Szostak, a professor of chemistry at the University of Chicago, and a group of researchers to see if it was possible to make an argument that mirror life wasn’t going to wipe out humanity. Included in that group was Kate Adamala, a synthetic biologist at the University of Minnesota. She was a natural choice: Adamala had shared the initial grant from the NSF, in 2019, to explore mirror-life technologies. 

    She also became convinced the risk was real—and was dumbfounded that she hadn’t seen it earlier. “I wish that one sunny afternoon we were having coffee and we realized the world’s about to end, but that’s not what happened,” she says. “I’m embarrassed to admit that I wasn’t even the one that brought up the risks first.” Through late 2023 and early 2024, the endeavor began to take on the form of a rigorous scientific investigation. Experts were presented with a hypothesis—namely, that if mirror cells were built, they would pose an existential threat—and asked to challenge it. The goal was to falsify the hypothesis. “It would be great if we were wrong,” says Vaughn Cooper, a microbiologist at the University of Pittsburgh and president-elect of the American Society for Microbiology. 

    Relman says that as the chemists and biologists learned more about one another’s work and began to understand what immunologists know about how living things defend themselves, they started to connect the dots and see an emerging picture of an unstoppable synthetic threat.

    Some scientists have pushed back against the doomsday scenario, suggesting that the case against mirror life offers an “inflated view of the danger.”

    Timothy Hand, an immunologist at the University of Pittsburgh who hadn’t participated in the 2019 NSF meeting, wasn’t initially worried when he heard about mirror life, in 2024. “The mammalian immune system has this incredible capability to make antibodies against any shape,” he says. “Who cares if it’s a mirror?” But when he took a closer look at that process, he could see a cascade of potential problems far upstream of antibody production. Start with detection: Macrophages, which are cells the immune system uses to identify and dispatch invaders, use chiral sensing receptors on their surfaces. The proteins they use to grab on to those invaders, too, are chiral. That suggests the possibility that an organism could be infected with a mirror organism but not be able to detect it or defend against it. “The lack of innate immune sensing is an incredibly dangerous circumstance for the host,” Hand says.

    By early 2024, Glass had become concerned as well. Relman and James Wagstaff, a structural biologist from Open Philanthropy, visited him at the Venter Institute to talk about the possibility of using synthetic cell technology—Glass’s specialty—to build mirror life. “At first I thought, This can’t be real,” Glass says. They walked through arguments and counterarguments. “The more this went on, the more I started feeling ill,” he says. “It made me realize that work I had been doing for much of the last 20 years could be setting the world up for this incredible catastrophe.” 

    In the second half of 2024, the growing group of scientists assembled the report and wrote the policy forum for Science. Relman briefed policymakers at the White House, members of the defense community, and the National Security Agency. Researchers met with the National Institutes of Health and the National Science Foundation. “We briefed the United Nations, the UK government, the government of Singapore, scientific funding organizations from Brazil,” says Glass. “We’ve talked to the Chinese government indirectly. We were trying to not blindside anybody.” 

    A year and a half on, the push has had an impact. UNESCO has recommended a precautionary global moratorium on creating mirror-life cells, and major philanthropic organizations that fund science, including the Alfred P. Sloan Foundation, have announced they will not finance research leading to a mirror microorganism. The Bulletin of the Atomic Scientists highlighted considerations about mirror life in its most recent report on the Doomsday Clock. In March, the United Nations Secretary-General’s Scientific Advisory Board issued a brief highlighting the risks—noting, for example, that recent progress on building mirror molecules could reduce the cost of creating a mirror microbe. 

    “I think no one really believes at this stage that we should make mirror life, based on the evidence that’s available,” says James Smith, the scientist who leads the MBDF, the nonprofit focused on assessing the risks of mirror life, which is funded by Coefficient Giving, the Sloan Foundation, and other organizations. The challenge now, Smith says, is for scientists to work with policymakers and bioethicists to figure out how much research on mirror life should be permitted—and who will enforce the rules.

    Drawing the line

    Not everyone is convinced that mirror organisms pose an existential threat. It’s difficult to verify predictions about how mirror microbes would fare in the immune system—or the larger world—without running experiments on them. Some scientists have pushed back against the doomsday scenario, suggesting that the case against mirror life offers an “inflated view of the danger.” Others have noted that carbohydrates called glycans already exist in both left- and right-handed forms—even in pathogens—and the immune system can recognize both of them. Experiments focused on interactions between the immune system and mirror molecules, they say, could help clarify the risks of mirror organisms and reduce uncertainty. 

    Even among those convinced that the worst-case scenario is possible, researchers still disagree over where to draw the line. What inquiries should be allowed and what should be prohibited?

    Andy Ellington, a biotechnologist and synthetic biologist at the University of Texas at Austin, doesn’t think mirror organisms will come to fruition anytime soon. Even if they do, he isn’t sure they will pose a threat. “If there is going to be harm done to the human race, this is about position 382 on my list,” he says. But at the same time, he says it’s a complicated issue worth studying more, and he wants to see the conversations continue: “We’re operating in a space where there’s so much unknown that it’s very difficult for us to do risk assessment.” 

    Even among those convinced that the worst-case scenario is possible, researchers still disagree over where to draw the line. What inquiries should be allowed and what should be prohibited? 

    Adamala, of the University of Minnesota, and others see a natural line at ribosomes, the cellular factories that transform chains of amino acids into proteins. These would be a critical ingredient in creating a self-replicating organism, and Adamala says the path to getting there once mirror ribosomes are in place would be pretty straightforward. But Zhu, at Westlake, and others counter that it’s worth developing mirror ribosomes because they could possibly produce medically useful peptides and proteins more efficiently than traditional chemical methods. He sees a clear distinction, and a foundational gap, between that kind of technology and the creation of a living synthetic organism. “It is crucial to distinguish mirror-image molecular biology from mirror-image life,” he says. That said, he points out that many synthetic molecules and organisms containing unnatural components, including but not limited to the mirror-image subset, might pose health risks. Researchers, he says, should focus on developing holistic guidelines to cover such risks—not just those from mirror molecules. 

    Even if the exact risk remains uncertain, Esvelt remains more convinced than ever that the work should be paused, perhaps indefinitely. No one has taken a meaningful swing at the hypothesis that mirror life could wipe out everything, he says. The primary uncertainties aren’t around whether mirror life is dangerous, he points out; they have more to do with identifying which bacterium—including what genes it encodes, what it eats, how it evades the immune system’s sentinels—could lead to the most serious consequences. “The risk of losing everything, like the entire future of humanity integrated over time, is not worth any small fraction of the economy. You just don’t muck around with existential risk like that,” he says. 

    In some ways, scientists have been here before, working out rules and limits for research. Two years after the start of the covid-19 pandemic, for example, the World Health Organization published guidelines for managing risks in biological research. But the history is much deeper: Horrific episodes of human experimentation led to the establishment of institutional review boards to provide ethical oversight. In the early 1970s, in response to concerns over lab-acquired infections and growing use of biological warfare, the US Centers for Disease Control and Prevention established biohazard safety levels (BSLs), which govern work on potentially dangerous biological experiments.

    And in 1975—at the dawn of recombinant DNA research, which allows researchers to put genetic material from one organism into another—geneticists met at the Asilomar conference center in Pacific Grove, California, to hammer out rules governing the work. There were concerns over what would happen if some virus or bacterium, genetically engineered to have traits that would make it particularly dangerous for people, escaped from a lab. Scientists agreed to self-imposed restrictions, like a moratorium on research until new safety guidelines were in place. As a result of the meeting, in June 1976 the NIH issued rules that, among other things, categorized the risks associated with rDNA experiments and aligned them with the newly adopted BSL system.

    Asilomar is often hailed as a successful model for scientific self-governance. But that perception reflects a tendency to recall the meeting through a nostalgic haze. “In fact, it was incredibly messy and human,” says Luis Campos, a historian of science at Rice University. Equally brilliant Nobelists argued on either side of the question of whether to rein in rDNA research. Technical discussions dominated; talks about who would be affected by the technology were missing. The meeting didn’t start establishing guidelines, says Campos, until the lawyers mentioned liability and lab leaks. 

    For now it’s unclear whether these examples of self-­governance, which arose from the demonstrated risks of existing technologies, hold useful lessons for the mirror-life community. Three competing images of the future are coming into focus: Mirror life might not be possible, it might be possible but not threatening, or it might be possible and capable of obliterating all life on Earth. 

    Scientists may be censoring themselves out of fear and speculation. To some, shutting down the work seems necessary and urgent; to others, it is unnecessarily limiting. What’s clear is that the question of what to do about mirror life has been both illuminating and disorienting, pushing scientists to interrogate not only their current research but where it might lead. This is uncharted territory. 

    Stephen Ornes is a science writer based in Nashville, Tennessee.

    ]]>     Novel Targets for Complex Cancer Revealed by Genetic Regulatory Node Mapping https://edusehat.com/en/novel-targets-for-complex-cancer-revealed-by-genetic-regulatory-node-mapping https://edusehat.com/en/novel-targets-for-complex-cancer-revealed-by-genetic-regulatory-node-mapping Thu, 16 Apr 2026 02:10:16 +0700 Edusehat Novel, Targets, for, Complex, Cancer, Revealed, Genetic, Regulatory, Node, Mapping In a new study published in Nature titled, “Mapping convergent regulators of melanoma drug resistance by PerturbFate,” researchers from The Rockefeller University have developed a platform called PerturbFate that can systematically map how diverse disease-associated genetic variations reshape cells. By tracking gene regulation in single cells over time, the team identified regulatory nodes common to diverse variations. Using melanoma drug resistance as a proof-of-concept, results showed that these shared points of control offer a path toward combination therapies that can target disease across many genetic causes. 

    “Once you know that a disease is associated with hundreds of genes, how do you design one therapy to target it?” posed Junyue Cao, PhD, head of the Laboratory of Single-Cell Genomics and Population Dynamics at Rockefeller. “We wondered whether all these different genes may be mediated by some shared downstream signaling that we can discover and target instead.” 

    Advances in genomic sequencing and genetic screening have allowed researchers to identify hundreds of genetic mutations linked to disease. Yet these genes often span diverse pathways with broad functionalities, from gene regulation to cell signaling, making them difficult to target collectively.  

    Cao proposed that if these mutations converge on shared downstream programs, the key challenge is not to target each mutation individually, but to identify the common control points known as regulatory nodes.  

    PerturbFate allows researchers to observe how different genetic changes reshape a cell in real time by tracking DNA accessibility, and RNA production and processing. By capturing these changes in the same single cell, the system reveals the networks of genes that control cell behavior and how different genetic variations can have the same effect. 

    “This technology lets us perturb hundreds to thousands of genes in parallel and then measure the detailed molecular changes in each individual cell,” says Cao. “That allows us to link many different genetic perturbations to their downstream effects and identify regulatory nodes.” 

    To test the platform, the authors focused on melanoma drug resistance. Using PerturbFate, they selected 143 genes linked to resistance to the common melanoma drug, Vemurafenib. PerturbFate then tracked how deactivating each of these genes reshaped the cell. Cao explains the platform captures gene expression, RNA dynamics and chromatin state, all critical components when identifying upstream regulators that drive these disease states. 

    After analyzing more than 300,000 cells, the researchers found that diverse genetic perturbations pushed melanoma cells into the same drug-resistant state. Drug resistance dropped significantly when these common control points were targeted, pointing to a promising strategy for combination therapies. 

    The platform also revealed an important nuance involving the transcriptional coactivator, Mediator Complex. Disrupting different parts of this same complex could trigger drug resistance through routes that ultimately converged on the same survival signal in melanoma cells, called VEGFC. Resistant cells could no longer proliferate after blocking that signal. 

    The team has made both the experimental and computational tools behind PerturbFate openly available, and plans to extend the approach from cultured cells to living systems. Cao and colleagues are currently applying PerturbFate to conditions, such as aging and Alzheimer’s disease, to uncover shared vulnerabilities that can guide more effective treatments. 

    “This is just a starting point,” says Cao. “Now that we’ve demonstrated the approach in a simple model, we’re working to extend it into living systems to study even more complex diseases.” 

    The post Novel Targets for Complex Cancer Revealed by Genetic Regulatory Node Mapping appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Biopharma Adopting AI Despite Remaining GMP Compliance Questions https://edusehat.com/en/biopharma-adopting-ai-despite-remaining-gmp-compliance-questions https://edusehat.com/en/biopharma-adopting-ai-despite-remaining-gmp-compliance-questions Thu, 16 Apr 2026 02:10:15 +0700 Edusehat Biopharma, Adopting, Despite, Remaining, GMP, Compliance, Questions The biopharma industry is embracing artificial intelligence (AI) in manufacturing, even though questions remain about how best to use the technology in a GMP environment.

    At least, so says Sanjay Konagurthu, PhD, senior director, science and innovation, pharma services, at Thermo Fisher Scientific, who argues that the key to successful AI adoption is a clear use case.

    “As is true in most industries, adoption of AI and machine learning (ML) is real and accelerating across the biopharma industry. However, most use cases center around applications that augment teams without completely redefining a validated process, such as smarter quality and inspection workflows.

    “We’re seeing that the hesitation isn’t so much reluctance to adopt AI as it is the practical constraints of operating in a good manufacturing practices-regulated environment. To adopt AI and ML in regulated environments, you need clear intended use, strong data foundations, traceable governance, and set parameters for disciplined control and monitoring,” he tells GEN.

    Data foundations

    In addition to establishing a strong use case, drug companies need an IT infrastructure that facilitates the flow of process data, according to Konagurthu, who says data silos are a persistent problem in biopharma.

    “As with most scientific endeavors, vast quantities of data are generated across the biopharma industry, among labs, organizations, consortia, and nations, and much of this data is stored in a singular system. So, there’s a connectivity challenge, but that’s not the only reason why processing technologies struggle to exchange information.

    “The data is often captured according to different standards and exists in a variety of formats, which means context is easily lost. Also, the data exists in a wide variety of structured and unstructured formats, which compounds the challenge in effective curation and analysis,” he says.

    Failure to establish an effective infrastructure or standardize data has multiple negative consequences, Konagurthu adds.

    “When data from early development can’t be connected through to commercialization, teams end up re-running experiments and analysis. They may even miss early signals that could impact downstream manufacturing or risk quality.

    “When companies look to scale or implement new technologies like AI and ML, fragmented data can become prohibitive. Ultimately, for biopharma, this could extend the time it takes to bring a promising molecule to market,” he says.

    Formul-AI-tion development

    Beyond process development and control, formulation is another area where more and more biopharmaceutical companies are making a use case for AI.

    Konagurthu says, “Biopharma scientists have historically used trial-and-error approaches to determine the right solubility and bioavailability of OSD [oral solid dose] therapies. With AI and ML models, teams can make earlier, better-informed decisions on formulation pathways.

    “Early-stage acceleration in discovery and formulation echoes all the way into manufacturing and clinical supply, so improving the front end can compress timelines across the entire pipeline,” he adds.

    The post Biopharma Adopting AI Despite Remaining GMP Compliance Questions appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     iPSC-Based Manufacture vs. Autologous Model Production Costs Examined via Financial Analysis https://edusehat.com/en/ipsc-based-manufacture-vs-autologous-model-production-costs-examined-via-financial-analysis https://edusehat.com/en/ipsc-based-manufacture-vs-autologous-model-production-costs-examined-via-financial-analysis Thu, 16 Apr 2026 02:10:14 +0700 Edusehat iPSC-Based, Manufacture, vs., Autologous, Model, Production, Costs, Examined, via, Financial, Analysis Autologous and allogeneic cell therapies are establishing viable clinical pathways but cannot be manufactured cost-effectively at scale. Manufacturing natural killer (NK) cell therapies, and possibly T-cell therapies, using induced pluripotent stem cells (iPSCs) is understood to be significantly more cost-effective. Now those cost advantages have been quantified.

    Specifically, the cost of goods per treatment can be reduced as much as 95% when manufacturing via iPSCs rather than using traditional autologous or allogenic production methods. By decoupling production from the patient, manufacturers can benefit from large-scale batch production, standardized processes, less labor, and a less complex infrastructure than either autologous or allogeneic production. Details are spelled out in a white paper by Cellistic, based on an intense cost-of-goods analysis of NK cell therapy manufacturing performed by Astrid Van Damme, PhD, head of project management at Cellistic, for her MBA thesis.

    In it, Van Damme advocates creating a universal master cell bank that feeds multiple working cell banks. Those working cell banks, in turn, generate intermediate hematopoietic stem cells that are differentiated into the final therapeutic product. “This cascade creates an essentially inexhaustible, standardized source material for the entire commercial lifecycle of the product,” she asserts.

    Cellistic’s internal review compared seven economic drivers for each of the three cell therapy manufacturing options. Notable advantages for an iPSC manufacturing strategy include:

    • Commercial scale production
    • Exponential scale-up or scale-out
    • Industrial-scale reproducibility
    • Use of standard cold-chain logistics
    • Minimal patient interactions
    • Reduced patient attrition
    • Potentially global market reach

    An iPSC manufacturing strategy for cell therapies drops the cost of goods sold to about $5,000 per dose, down from $115,000 per dose for autologous therapeutics and $40,000 per dose for allogeneic therapeutics, Van Damme reports.

    Autologous and allogeneic manufacturing, in contrast, both have severe constraints that increase costs for manufacturers and payers alike. Materials and labor alone account for 50% to 70% of autologous cell therapies—roughly $80,000 to $150,000. That’s a huge driver for U.S. list prices that, for the oncology therapeutics Kymriah® and Carvykti®, are at or above an adoption-limiting $475,000 per dose. Even after factoring in regional pricing differences and payer discounts, the net per-dose costs to payers are still extremely high.

    Compared to iPSC manufacturing at clinical scale (150 vials and 200 M cells per vial) and at commercial scale (450 vials with 400 M cells per vial), Van Damme indicates:

    • Labor costs constituted about 13% of the costs of goods (vs. about 70% for autologous methods)
    • Costs per vial drop approximately 40%
    • Fixed costs were diluted by a factor of three

    “Once a minimum threshold of operational maturity and throughput is achieved, iPSC-based manufacturing economics become comparatively robust to routine operational variability,” the paper concludes.

    The post iPSC-Based Manufacture vs. Autologous Model Production Costs Examined via Financial Analysis appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Ultra- and Diafiltration Clear Leachables Effectively https://edusehat.com/en/ultra-and-diafiltration-clear-leachables-effectively https://edusehat.com/en/ultra-and-diafiltration-clear-leachables-effectively Thu, 16 Apr 2026 02:10:12 +0700 Edusehat Ultra-, and, Diafiltration, Clear, Leachables, Effectively In the push to de-risk biologics manufacturing, downstream purification steps are increasingly under the microscope. Now, new research led by Jonathan Bones, PhD, principal investigator in the characterization and comparability group at the National Institute for Bioprocessing Research in Dublin, and his colleagues provided compelling evidence that ultrafiltration and diafiltration (UF/DF) deliver robust clearance of process-related leachables—while also offering a predictive framework to better understand that performance.

    Although UF/DF has long been assumed to reduce small-molecule contaminants, systematic data have been scarce. To address this gap, the team evaluated 28 representative organic compounds spiked into three distinct protein systems. Using liquid chromatography–high resolution mass spectrometry, they tracked how effectively these compounds were removed during UF/DF operations.

    The results were striking. Twenty-four of the compounds demonstrated greater than 98% clearance across all three protein processes. Notably, variations in protein characteristics and process parameters had minimal impact on removal efficiency. Instead, clearance behavior was remarkably consistent, as reflected in similar sieving coefficients across the systems.

    The intrinsic physicochemical properties of the leachables impacted clearance. Among these, lipophilicity—expressed as the octanol-water partition coefficient (Log P)—emerged as the dominant factor. Compounds with Log P values below four exhibited near-ideal clearance, while even highly hydrophobic molecules (Log P above seven) still achieved removal rates exceeding 93%. Molecular weight, polarizability, and solvent-accessible surface area also contributed to clearance outcomes.

    Beyond empirical findings, the study advances the field with predictive modeling. By applying orthogonal partial least squares (OPLS) regression, the researchers developed tools capable of estimating sieving coefficients based on compound properties. These models could prove invaluable for anticipating leachable behavior without exhaustive experimental testing.

    The implications are significant. As regulatory scrutiny around extractables and leachables intensifies, demonstrating effective clearance becomes central to product safety. This work not only confirms that UF/DF is a powerful mitigation step but also equips developers with quantitative tools to support risk assessments.

    In an industry where unseen contaminants can pose outsized risks, the ability to both measure and predict their removal marks a meaningful step forward.

    The post Ultra- and Diafiltration Clear Leachables Effectively appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Digital Twin Process Could Slash Microbial Protein Costs https://edusehat.com/en/digital-twin-process-could-slash-microbial-protein-costs https://edusehat.com/en/digital-twin-process-could-slash-microbial-protein-costs Thu, 16 Apr 2026 02:10:11 +0700 Edusehat Digital, Twin, Process, Could, Slash, Microbial, Protein, Costs A consortium of companies has developed what they call a digital twin of a microbial process to produce protein A.

    Novasign, based in Vienna, hopes its participation through the ECOnti consortium will help manufacturers slash the costs of microbial proteins by improving experimental design.

    According to the company, the digital twin can reduce the number of experiments needed to understand process behavior by 70% compared to Design of Experiments (DoE).

    “Generally, the biggest problem in the industry right now is it’s not very efficient,” explains Mark Duerkop, PhD, CEO of Novasign.

    “We need methods to learn more efficiently from experiments, design better experiments, and adapt process trajectories if something goes wrong.”

    According to Duerkop, Novasign began developing an end-to-end digital twin of the full processing chain for microbial protein production as part of the ECOnti consortium three years ago.

    Novasign says it develops digital twins spanning an entire process—from upstream to downstream—with the goal of improving both process development and manufacturing efficiency.

    “The digital twin supports process development by systematically recommending the next set of experiments based on model-informed insights,” he says.

    Setup of the Novasign ECOnti Digital Twin Technology

    “During manufacturing, it can detect deviations from the intended process trajectory and support corrective actions.”

    For example, if the digital twin is used to recover a process following disturbances, such as pH shifts or feed pump failure, manufacturers could significantly reduce product losses.

    However, this remains for the future, he says, as the U.S. Food and Drug Administration (FDA) requires extensive validation before approving self-optimizing or autonomous manufacturing processes.

    At the recent Bioprocessing Summit Europe, Duerkop presented a showcase on using the Novasign Studio software for full process control for 30 consecutive days.

    He also showed how the software can use small-scale experimental data to inform scale-up and, in biosimilar development and viral vector manufacturing, can reduce experimental effort by up to 64%.

    The post Digital Twin Process Could Slash Microbial Protein Costs appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO Launches ‘Fight of Our Lives’: The Real Stories, Power, and Promise of American Biotech at a Defining Moment https://edusehat.com/en/bio-launches-fight-of-our-lives-the-real-stories-power-and-promise-of-american-biotech-at-a-defining-moment https://edusehat.com/en/bio-launches-fight-of-our-lives-the-real-stories-power-and-promise-of-american-biotech-at-a-defining-moment Wed, 15 Apr 2026 22:40:04 +0700 Edusehat BIO, Launches, ‘Fight, Our, Lives’:, The, Real, Stories, Power, and, Promise, American, Biotech, Defining, Moment At some point in our lives, many of us will find ourselves in a health fight we didn’t expect. A diagnosis. A test result. A moment that changes everything.

    The Biotechnology Innovation Organization (BIO)’s new “Fight of Our Lives” campaign brings this reality into focus through the real stories of the patients, families, caregivers, researchers, and entrepreneurs who fight every day to find answers. 

    Over the past 50 years, American biotechnology has delivered extraordinary breakthroughs – preventing illness, improving treatment, and advancing therapies once thought impossible. But that momentum cannot be taken for granted. Nearly 200 million people in the United States live with at least one chronic condition, more than 30 million are affected by rare conditions, and cancer prevalence is projected to exceed 22 million by 2035. Millions are counting on the next generation of American innovation – and on our ability to build on this foundation to deliver what comes next.

    Through powerful, human-centered storytelling, Fight of Our Lives brings that urgency into focus – elevating the people behind the progress and reinforcing what it will take to sustain innovation, strengthen economic leadership, and deliver the next generation of breakthroughs.

    Meet the incredible people behind the first featured Fight of Our Lives stories: 

    • Ben, whose parents’ relentless search for answers uncovered a rare genetic condition – transforming him from an inconsolable baby to an unstoppable kid through access to a targeted therapy – and the broader scientific effort that made that breakthrough possible
    • Marci, diagnosed with multiple sclerosis who, in partnership with her physician, became the first patient to receive an investigational CAR-T therapy, an innovative approach that reprograms the immune system and has shown unprecedented results in a disease once defined by progression.
    • Katy, whose life changed in an instant due to an antibiotic-resistant infection, and who now advocates for awareness and innovation, alongside scientists working to harness artificial intelligence to develop a new generation of more effective antibiotics.

    Together, these individuals reflect what American biotechnology makes possible – and why its progress matters to the more than 100 million Americans living with conditions it can help address. But millions more are still waiting for answers.

    At a time when policy challenges and global competition are rapidly evolving, sustaining an environment that enables scientific progress is essential. American biotechnology is rewriting what comes next – bringing forward new options, new treatments, and new answers for the fight ahead.

    This is the Fight of Our Lives.

    The post BIO Launches ‘Fight of Our Lives’: The Real Stories, Power, and Promise of American Biotech at a Defining Moment appeared first on Bio.News.

    ]]>     Engineered Miniature CRISPR Boosts Gene‑Editing Efficiency in Human Cells https://edusehat.com/en/engineered-miniature-crispr-boosts-geneediting-efficiency-in-human-cells https://edusehat.com/en/engineered-miniature-crispr-boosts-geneediting-efficiency-in-human-cells Wed, 15 Apr 2026 07:55:09 +0700 Edusehat Engineered, Miniature, CRISPR, Boosts, Gene‑Editing, Efficiency, Human, Cells One of the biggest obstacles in targeting CRISPR therapy deliveries directly into the body isn’t the editing chemistry, it’s the size of the editors themselves. The field’s workhorse nucleases, including Cas9 and Cas12a, are considerably large (exceeding 1,300 amino acids) to fit inside adeno‑associated virus (AAV) vectors, the most widely used delivery vehicle for in vivo gene therapy. That size mismatch has forced most clinical applications to rely on ex vivo editing of blood or bone‑marrow‑derived cells, leaving many tissues out of reach. A smaller CRISPR system that can be packaged into AAV without sacrificing efficiency has long been a key missing piece.

    A new study published in Nature Structural & Molecular Biology takes a major step toward that goal. Researchers at the University of Texas at Austin and collaborators report the discovery and engineering of a compact Cas12f nuclease that performs robustly in human cells, a notable advance for a class of miniature enzymes that have historically shown lower efficiencies in mammalian cells compared to larger systems. The paper is titled, “Comparative characterization of Cas12f orthologs reveals mechanistic features underlying enhanced genome editing efficiency.”

    The team began by mining metagenomic datasets for naturally small CRISPR enzymes and identified a previously uncharacterized ortholog, Alistipes sp. Cas12f (Al3Cas12f). Despite its compact size—roughly one‑third that of Cas9—the nuclease showed unexpectedly strong activity in human cells. In initial screens, Al3Cas12f produced more than 50% editing at many genomic sites and exceeded 90% at several targets. The authors wrote, “Results from a gRNA screen targeting intron 1 of the ALB gene, exon 3 of the APOA1 gene and the AAVS1 site within PPP1R12C intron 1 showed that 27 target sites displayed >10% editing, 19 sites displayed >50% editing and 10 sites displayed >90% editing across AAVS1 and APOA1.”

    Cryo‑EM structures revealed why this miniature enzyme punches above its weight. Compared with other Cas12f orthologs, Al3Cas12f forms a more extensive and interlocking dimer interface, creating a stable, preassembled complex that supports efficient R‑loop formation. The guide RNA scaffold also appears naturally streamlined: unlike other Cas12f gRNAs, it lacks an extraneous stem‑loop and adopts a compact conformation that docks cleanly into the protein. As the authors noted, Al3Cas12f achieves “efficient R‑loop formation through a stable dimer interface and a naturally optimized gRNA.”

    Using these structural insights, the team engineered an enhanced variant, Al3Cas12f RKK, that dramatically boosts editing efficiency across genomic loci. In human cells, the variant increased editing from below 10% to more than 80% at many targets, with some sites reaching 90%. The researchers tested the system in a leukemia‑derived human cell line, focusing on genes implicated in cancer, atherosclerosis, and ALS.

    The mechanistic comparisons were equally revealing. By solving the structures of two additional Cas12f orthologs—Oscillibacter sp. Cas12f and Ruminiclostridium herbifermentans Cas12f—the team noted “divergent architectures and regulatory features governing protospacer-adjacent motif recognition, gRNA binding, dimerization, and DNA cleavage.” Al3Cas12f’s extended helices and mortise‑and‑tenon‑like interactions appear to be lineage‑specific adaptations that stabilize the nuclease and support high activity.

    The next step is to test whether the enzyme maintains its performance when packaged into AAV vectors. If successful, the system could offer a blueprint for engineering future generations of compact CRISPR tools.

    The post Engineered Miniature CRISPR Boosts Gene‑Editing Efficiency in Human Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     GLP-1 Drug Improves Liver Health Independent of Weight Loss, Mouse Study Finds https://edusehat.com/en/glp-1-drug-improves-liver-health-independent-of-weight-loss-mouse-study-finds https://edusehat.com/en/glp-1-drug-improves-liver-health-independent-of-weight-loss-mouse-study-finds Wed, 15 Apr 2026 04:20:12 +0700 Edusehat GLP-1, Drug, Improves, Liver, Health, Independent, Weight, Loss, Mouse, Study, Finds Studying mice, researchers at Toronto’s Sinai Health have found that semaglutide—the active ingredient in popular weight loss drugs that mimic the gut hormone GLP-1—acts directly on a subset of liver cells to improve organ function, and does so independently of weight loss. The finding challenges long-held assumptions about how GLP-1 medicines work in the liver and could reshape how physicians treat metabolic liver disease.

    Headed by Daniel Drucker, MD, a senior investigator at the Lunenfeld-Tanenbaum Research Institute, the team reported on their findings in Cell Metabolism, in a paper titled “The weight-loss-independent hepatoprotective benefits of semaglutide are orchestrated by intrahepatic sinusoidal endothelial GLP-1 receptors.”

    For years, the liver benefits of semaglutide have puzzled scientists. “Glucagon-like peptide-1 (GLP-1) medicines improve metabolic liver disease through weight-loss-dependent and -independent actions,” the authors wrote. The drug was known to lower blood sugar and promote weight loss, but patients’ livers were improving in ways that those effects alone could not explain. And as the authors further noted, “The therapeutic scope of GLP-1 medicines extends beyond glycemic control and weight loss, with benefits evident in people with atherosclerotic heart disease, heart failure with preserved ejection fraction (HFpEF), peripheral artery disease, diabetic kidney disease, knee osteoarthritis, and obstructive sleep apnea (OSA).” However, as the team further pointed out, “… the mechanisms by which GLP-1 medicines improve organ dysfunction remain incompletely understood.”

    Drucker has been at the forefront of GLP-1 research since the 1980s when his pioneering discoveries helped lay the groundwork for the development of GLP-1 medicines. After transforming treatment of type 2 diabetes and obesity, semaglutide and other GLP-1 medicines have been approved for other conditions including MASH (metabolic dysfunction-associated steatohepatitis). MASH is a severe form of fatty liver disease in which fat build-up, inflammation, and tissue scarring can lead to cirrhosis and liver failure. It affects about 25% Canadian adults and because it is closely linked with obesity and type 2 diabetes, treatment typically includes lifestyle interventions to reduce weight. “The approval of semaglutide for MASH highlights the importance of understanding the hepatoprotective mechanisms of GLP-1 action,” the investigators stated.

    Drucker and colleagues have now found that semaglutide acts directly on the liver to reduce inflammation and scarring and improve organ function in a way that is independent of weight loss. Their finding overturns a prevailing assumption in the field that liver cells do not carry the receptor that semaglutide binds to, meaning the drug had no direct route to the organ.

    Postdoctoral researcher Maria Gonzalez-Rellan, PhD, spearheaded the work that combined sophisticated mouse models of MASH with deep molecular analyses of liver cells. Her work identified two cell types carrying semaglutide receptors: liver sinusoidal endothelial cells (LSECs) and immune T cells. Although LSECs account for only about 3% of liver cell volume, they proved to be the key driver of semaglutide’s liver benefits.

    A pioneer in GLP-1 biology Dr. Daniel Drucker has dedicated his career to understanding how the GLP-1 hormone, and the therapies derived from it, function in the body. His early discovery that GLP-1 stimulates insulin secretion in a glucose-dependent manner paved the way for today's widely popular medications for type 2 diabetes and obesity. Dr. Drucker's ongoing research continues to shine light on the less understood aspects of GLP-1 biology including its effects on the liver and in regulating inflammation. [Colin Dewar, Sinai Health]
    A pioneer in GLP-1 biology, Daniel Drucker, MD, has dedicated his career to understanding how the GLP-1 hormone, and the therapies derived from it, function in the body. His early discovery that GLP-1 stimulates insulin secretion in a glucose-dependent manner paved the way for today’s widely popular medications for type 2 diabetes and obesity. Drucker’s ongoing research continues to shine light on the less understood aspects of GLP-1 biology including its effects on the liver and in regulating inflammation. [Colin Dewar, Sinai Health]
    LSECs line the tiniest blood vessels in the liver and are studded with pores that allow them to act as a molecular sieve, filtering substances passing between the liver and the bloodstream. Gonzalez-Rellan showed that semaglutide reversed MASH in mice that lacked the brain receptors controlling appetite, demonstrating that weight loss is not required for liver benefits. “Unexpectedly. semaglutide improves hepatic inflammation, fibrosis, and immune remodeling through actions on Glp1r+ pericentral liver sinusoidal ECs (LSECs) independent of changes in body weight (BW),” the team reported. “… we leveraged a unique model of GLP-1R deficiency, Glp1rWnt1-/- mice, which are resistant to GLP-1RA-induced weight loss. Remarkably, semaglutide markedly improved hepatic steatosis, fibrosis, and immune remodeling in the absence of weight reduction.”

    In a further test, mice lacking LSEC receptors showed no liver improvement on semaglutide even after losing 20% of their body weight. Detailed molecular analyses of liver cell types showed that semaglutide shifts gene activity in LSCEs, prompting them to release anti-inflammatory molecules that act on the broader liver environment, pushing it toward a state more closely resembling a healthy, disease-free liver. “Together, the data using mouse models of MASH reveal an EC-specific, weight-loss-independent, semaglutide-regulated, GLP-1R-dependent intrahepatic network for improving liver health,” the scientists said.

    “It turns out that the receptor responsible for these benefits is in a very specialized population of liver cells,” commented Drucker, who is also a professor of medicine at the University of Toronto. “And this receptor orchestrates the production of molecules that talk to many different types of liver cells to calm down the inflammatory environment that is the problem in metabolic disease.”

    The findings carry practical implications. GLP-1 medicines have become widely prescribed, yet their mechanism of action in the body, beyond appetite suppression and blood sugar control, have remained incompletely understood. Knowing that semaglutide improves liver health independently of weight loss could influence prescribing decisions. “We’ve seen in clinical trials that patients who lose very little weight see the same reductions in liver inflammation, scarring and enzyme levels as those who lose a great deal of weight. Now we know why,” Drucker pointed out. In their paper the team concluded “Hence, semaglutide produces a broad proteomic remodeling of the liver, enabling restoration of metabolic homeostasis and suppression of fibrogenic and inflammatory programs. The strong concordance between single-cell transcriptional changes, bulk tissue proteomics, and biomarker signatures underscores the breadth of GLP-1R-mediated hepatic reprogramming.”

    Physicians may choose lower doses that avoid the side effects associated with the higher doses needed for significant weight loss, potentially also lowering costs for patients, Drucker suggested adding “We’re not saying weight loss isn’t important because many things improve when patients lose weight. But we now know that weight shouldn’t be the only measure of success, because GLP-1 medicines will improve liver health whether or not the patient loses weight.”

    The post GLP-1 Drug Improves Liver Health Independent of Weight Loss, Mouse Study Finds appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Regeneron, Telix Launch Up-to-$4.3B Cancer-Focused Radiopharma Drug, Diagnostic Collaboration https://edusehat.com/en/regeneron-telix-launch-up-to-43b-cancer-focused-radiopharma-drug-diagnostic-collaboration https://edusehat.com/en/regeneron-telix-launch-up-to-43b-cancer-focused-radiopharma-drug-diagnostic-collaboration Wed, 15 Apr 2026 04:20:11 +0700 Edusehat Regeneron, Telix, Launch, Up-to-4.3B, Cancer-Focused, Radiopharma, Drug, Diagnostic, Collaboration Regeneron Pharmaceuticals plans to expand its pipeline into radiopharmaceutical therapies through an up to $4.3 billion collaboration with Telix Pharmaceuticals to co-develop and co-commercialize precision oncology treatments and companion diagnostics.

    The companies have agreed to partner on next-generation radiopharmaceutical therapies aimed at up to eight solid tumor targets from Regeneron’s portfolio of antibodies, generated from VelocImmune® technology, which uses the company’s own mouse platform engineered with a genetically humanized immune system.

    Regeneron and Telix also said they plan to develop radio-diagnostics designed to support patient selection and treatment response assessment.

    The collaboration is intended to combine the biologics expertise of Tarrytown, NY-based Regeneron, including bispecific antibody discovery, with the radiopharmaceutical development platform, global manufacturing capabilities, and supply chain infrastructure of Telix, which is headquartered in Melbourne, Australia.

    “Regeneron is excited to enter the targeted radiopharmaceuticals space and explore the utility of these agents either as monotherapy or rationally combined with our immunotherapy platform, particularly in areas of high unmet patient need such as lung cancer, where our PD-1 inhibitor is a global standard of care,” Israel Lowy, MD, PhD, Regeneron’s senior vice president and clinical development unit head, oncology, said in a statement.

    Lowy referred to Libtayo® (cemiplimab-rwlc), a programmed death receptor-1 blocking antibody approved for multiple oncology indications including forms of non-small cell lung cancer (NSCLC), as well as cutaneous squamous cell carcinoma, and basal cell carcinoma. Libtayo finished 2025 with $1.453 billion in worldwide net product sales, up 19% from $1.217 billion in 2024. Figures include $425 million in Q4 2025 global net product sales, up 16% from $367 million in the year-ago quarter.

    ‘An ideal partner’

    “In our view, the deal with Regeneron validates Telix’s differentiated capabilities in radiopharmaceutical development and handling of complex supply chain logistics,” Andy T. Hsieh, PhD, a partner and biotechnology analyst with William Blair, wrote Monday in a research note. “Furthermore, given Regeneron’s track record of developing successful commercial therapeutics, we believe it is an ideal partner in bringing forth antibody-based theranostic assets.”

    Telix investors appeared to somewhat agree with that analysis. The company’s ordinary shares traded on the Australian Stock Exchange climbed nearly 8% from A$14.64 ($10.34) to A$15.77 ($11.13). Telix’s American depositary shares traded on NASDAQ rose about 7%, from $10.56 to $11.24.

    Hsieh reiterated William Blair’s “Outperform” rating for Telix shares based on several potential value-creating inflection points, including:

    • Continuing gains in market share gains within the prostate-specific membrane antigen (PSMA) positron emission tomography (PET) diagnostic imaging market, based on rising sales and price stability as payers have offered clarity on reimbursement—factors he said enable Telix to expand its precision medicine franchise “from a position of strength.”
    • Therapeutic franchise potential, as supported by recent positive preliminary data from part 1 of the ongoing Phase III ProstACT GLOBAL trial (NCT06520345) assessing TLX591 in metastatic androgen pathway modulation resistant prostate cancer.
    • Potential approvals of two PET imaging agents—TLX250-CDx (Zircaix®, 89Zr-DFO-girentuximab), designed to non-invasively detect and characterize clear cell renal cell carcinoma (ccRCC); and TLX101-Px (Pixclara®, Floretyrosine F 18 or 18F-FET), designed to image glioma. Both could “meaningfully” contribute to Telix’s profit-and-loss statement next year, the analyst predicted.

    The FDA rejected both Zircaix and Pixclara last year via separate complete response letters. The agency held in April 2025 that Zircaix required additional confirmatory clinical evidence, which the company agreed to provide. On Friday, Telix said the FDA accepted its resubmitted New Drug Application (NDA) for Pixcara, assigning a target decision date of September 12 under the Prescription Drug User Fee Act (PDUFA).

    In August 2025, the FDA rejected Zircaix via complete response letter, alleging deficiencies relating to its chemistry, manufacturing, and controls (CMC) package—deficiencies the company said were “readily addressable.”

    “We look forward to additional updates pertaining to efficacy parameters, such as progression-free survival, an approvable endpoint, likely later this year,” Hsieh added.

    Growth through acquisitions

    Telix has built up its radiopharma infrastructure in recent years through acquisitions, spending $13.6 million to purchase IsoTherapeutics, a contract development and manufacturing organization (CDMO) focused on providing services to Telix and other radiopharmaceutical companies—followed by an up to $82 million buyout of radioisotope production technology firm ARTMS, which stands for alternative radioisotope technologies for medical science.

    In September 2024, Telix expanded its manufacturing footprint by acquiring RLS Radiopharmacies for up to $250 million, part of an investment strategy focused around creating vertically integrated supply chain, manufacturing, and distribution.

    The global radiopharmaceuticals market is predicted to grow at a compound annual growth rate of 10.1%, more than doubling from $14.2 billion this year to $31 billion in 2032, then soaring again to $54.6 billion by 2040, according to a Roots Analysis report issued in January.

    Telix briefly pursued a U.S. initial public offering, which it withdrew in June 2024. The company cited market conditions as biotech IPOs met with chilly receptions on Wall Street and asserted that the offering was not predicated on the need to raise capital.

    Regeneron has agreed to pay Telix $40 million in upfront cash for access to its radiopharmaceutical manufacturing platform for four initial therapeutic programs, with Regeneron holding an option to expand the collaboration to include four additional programs with additional upfront payments.

    Telix and Regeneron have agreed to share equally their global commercialization costs and potential profits, with Telix retaining the option to co-promote certain potential products. However, if Telix were instead to opt out of the co-funding model for any of the original four programs, it would then be eligible to receive up to $535 million in development and commercial milestone payments, plus low double-digit royalties on future net sales, for that program.

    If Telix opts out of co-funding for all four, company could achieve $2.14 billion in payments tied to achieving milestones.

    For the diagnostics to be covered by the collaboration, Telix and Regeneron have agreed to jointly develop diagnostic assets, with Telix leading commercialization and Regeneron receiving a set percentage of profits.

    “The collaboration with Regeneron reflects a highly complementary set of capabilities and a unique opportunity to explore what true ‘next gen’ biologics-based radiopharmaceuticals can potentially do for patients,” added Christian Behrenbruch, DPhil, managing director and group CEO at Telix. “We are well positioned to work toward the shared goal of advancing next-generation precision radiopharmaceuticals for patients with hard-to-treat cancers.”

    The post Regeneron, Telix Launch Up-to-$4.3B Cancer-Focused Radiopharma Drug, Diagnostic Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     CAR T Cell Therapy Biomanufactured by Cellares Infused Into First Two Patients https://edusehat.com/en/car-t-cell-therapy-biomanufactured-by-cellares-infused-into-first-two-patients https://edusehat.com/en/car-t-cell-therapy-biomanufactured-by-cellares-infused-into-first-two-patients Wed, 15 Apr 2026 04:20:10 +0700 Edusehat CAR, Cell, Therapy, Biomanufactured, Cellares, Infused, Into, First, Two, Patients Cellares reported that the first two patients have been dosed with Cabaletta Bio’s investigational CAR T cell therapy rese-cel (resecabtagene autoleucel) manufactured on Cellares’ Cell Shuttle™ instrument. The administration of an autologous cell therapy, which met all release criteria and was manufactured on an automated manufacturing platform, represents an important step on the journey to realizing a future where scalable manufacturing of autologous products to supply thousands of patients per year can be achieved with minimal capital investment and a low cost of goods, according to a Cellares spokesperson.

    While the transformative clinical benefits of autologous CAR T cell therapy are well established in oncology, the high manufacturing costs, lack of scalability, process inconsistency, and operational inflexibility associated with the current highly manual way of manufacturing have created meaningful barriers to patient access, reducing patient accessibility to these therapies.

    “This is an important milestone that reflects three years of focused collaboration between the teams at Cabaletta and Cellares,” said Steven Nichtberger, MD, co-founder, chairman, and CEO of Cabaletta Bio. “The dosing of these first two patients is an important demonstration of Cellares’ GMP manufacturing and supply chain capabilities with their automated manufacturing platform and thus represents a significant achievement toward our goal of securing high-capacity flexible supply with minimal capital investment and a low cost of goods.”

    “This milestone is a transformative moment for the field of autologous cell therapy,” added Fabian Gerlinghaus, co-founder and CEO of Cellares. “For years, the promise of autologous CAR T has been constrained by manufacturing models that were never designed to scale.”

    Rese-cel (formerly referred to as CABA-201) is an investigational, autologous CAR T cell therapy engineered with a fully human CD19 binder and a 4-1BB co-stimulatory domain, designed specifically for the treatment of autoimmune diseases. Administered as a single, weight-based infusion, rese-cel is intended to transiently and deeply deplete CD19-positive cells, with the goal of resetting the immune system and achieving durable clinical responses without the need for chronic therapy.

    Cabaletta is evaluating rese-cel in the RESET™ (REstoring SElf-Tolerance) clinical development program, which includes multiple ongoing company-sponsored trials across a diverse and growing range of autoimmune diseases in rheumatology, neurology, and dermatology.

    The post CAR T Cell Therapy Biomanufactured by Cellares Infused Into First Two Patients appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     From Colossal to Chickens: The Scientists Behind Neion Bio’s Biologics Platform https://edusehat.com/en/from-colossal-to-chickens-the-scientists-behind-neion-bios-biologics-platform https://edusehat.com/en/from-colossal-to-chickens-the-scientists-behind-neion-bios-biologics-platform Wed, 15 Apr 2026 00:45:10 +0700 Edusehat From, Colossal, Chickens:, The, Scientists, Behind, Neion, Bio’s, Biologics, Platform Twenty years ago, Sven Bocklandt, PhD, sought to create a hypoallergenic cat. He had the genetic engineering chops to do it, but the embryology was beyond his capabilities. At a small animal genetic engineering conference, known as TARC (Transgenic Animal Research Conference), held near Lake Tahoe, he met James Kehler, VMD, PhD, whose research at that time was to make transgenic and knockout cats as models of human disease.  

    The two men bonded, agreed the hypoallergenic cat idea was “crazy enough,” and decided to move forward with it. They worked together, completely unfunded, for years—FedEx’ing samples back and forth as Bocklandt was on the west coast and Kehler on the east coast—trying to make their “garage cat” while each one worked different day jobs.  

    Bocklandt, passionate about animal genome engineering, continued to develop different ideas for genome engineering in animals. Around the same time that he started sharing his ideas with scientists like George Church, PhD, a start-up focused on animal genome engineering was taking shape—Colossal Biosciences, co-founded by Church. Introductions were made, and Bocklandt joined in 2022 as species director to work on the dire wolf project. Kehler joined a short time later as VP. And everyone knows the rest of that story (there was no shortage of media coverage).  

    The pair eventually succeeded with the cat project: his name is Archie, and he is, Kehler noted, only partially hypoallergenic. But the generation of Archie and the dire wolves may not be the successes of this story. The real success may be what Bocklandt and Kehler learned along the way—and what they are going to do next.  

    Chickens as the next biologic factory

    Neion Bio, co-founded by Dimi Kellari and Sam Levin, PhD, and located on the Rockefeller University campus on the east side of Manhattan, is aiming to re-engineer eggs to produce drugs in chickens. The team uses genetic engineering to integrate therapeutic proteins into native egg proteins, creating a new manufacturing platform for drugs that runs on grain and water.  

    Bocklandt joined the team at Neion Bio as CSO after leaving Colossal in 2024; Kehler joined more recently, as head of avian sciences. 

    When thinking about producing complex proteins, using the chicken “makes a lot of sense,” Bocklandt told GEN. Breeding and genetic engineering are all established in the chicken. And the vaccine industry has established an existing infrastructure to grow eggs under disease-free conditions. Purifying proteins out of an egg, Bocklandt added, is easier than purifying them out of a Chinese hamster ovary (CHO) culture (the traditional cell choice for drug production) because there are fewer host proteins.  

    Sven Bocklandt, PhD [Marco Figueroa]
    It makes “far more sense” than what we’re doing right now, Bocklandt noted, which is using CHO cells. “Everyone is doing that because everyone has been doing it that way,” he asserted.  

    “The fact that we’re now seriously questioning whether CHO cells should remain the default manufacturing platform for biologics is long overdue,” noted Ola Wlodek, PhD, CEO of Constructive Bio. “Any credible new approach that breaks this decades-old lock-in is ultimately good for patients and for the field.”  

    For Kehler, who did his graduate work in the lab of stem cell pioneer Hans Schöler, PhD, the chicken is a clear choice because it is the only species, besides the mouse, where the primordial germ cells have been used to transmit genetically modified gametes to the next generation.  

    Mike McGrew, PhD, group leader at the Roslin Institute in the U.K., and an advisor to Neion Bio, demonstrated years ago that modifying chicken primordial germ cells is a reliable way of making gene-edited chickens. This background is comforting to Kehler, who noted that, “unlike at Colossal, where everything was bleeding edge, we are able to focus on a single species and capitalize on some pretty tried and true technology.”  

    Drugs in eggs meet biomanufacturing reality

    The lab space on the Rockefeller University campus can support research and even house chickens. But it cannot support the production of a drug. When asked about turning their egg-borne proteins into drugs, the company leans on the existing infrastructure that supports vaccines in specific pathogen free (SPF) eggs. The idea is that the egg whites will be frozen in giant batches and then processed in a CDMO.  

    When asked about potential challenges, Bocklandt noted that, “technically, there’s not much to worry about. I have no concerns about Neion Bio being able to do what we want to do or what we need to do.”  

    But there may be hurdles ahead. Rahul Dhanda, co-founder, president, and CEO of Syntis Bio, told GEN that “at the beginning, everything can look like it has infinite potential—it’s when you actually build and operate the system that the real challenges show up.”  

    More specifically, Dhanda pointed out that biomanufacturing “ultimately comes down to reliable, consistent, and cost-efficient production.” Leveraging animal biology for drug manufacturing is exciting, he noted, “but scalability and cost are still open questions, especially at this early stage. Biological variability between animals and individual outputs, like eggs, introduces additional risk compared to more controlled cell-based systems,” Dhanda added.  

    Wlodek agreed: “because egg-based production is inherently a biological supply chain, it will face avian flu risks, batch-to-batch variability from seasonal and flock effects, animal-welfare/regulatory overhead, and practical limits on how fast you can expand output compared with stainless-steel or single-use fermenters.” 

    Microbial and yeast systems still “win decisively on GMP containment, land/water footprint,” she noted, and “the ability to go from a few liters to tens of thousands of liters in weeks rather than months.” 

    Dhanda agreed that “getting it to work in principle is far different from getting it to work at scale, and that seems far off.” 

    If these challenges can be addressed at scale, safely and humanely, Dhanda noted, the approach could deliver meaningful health benefits—”but there are still significant logistical and technical hurdles to work through.”  

    Engineering the chicken genome

    Creating dire wolves at Colossal started with deriving wolf cells, editing them, and cloning them back into a live animal. But cloning doesn’t exist in birds. To genetically engineer chickens, the Neion Bio team edits the germline, starting the process with a fertilized egg.  

    Neion Bio
    Neion Bio [Marco Figueroa]
    The egg is incubated for 65 hours, at which point germ cells float in the blood because the ovaries and testes don’t exist yet. A microliter of the blood is removed, put into cell culture media, and the germ cells grow out. The transgene that codes for the therapeutic protein is inserted using CRISPR-Cas enzymes, in the coding region of a gene that codes for Ovalbumin—which makes up a bit over 50% of the egg white protein. This protein is made “on a massive scale” by the oviduct, the company noted.  

    The genome is screened for correct integration and potential off-target edits. Once the clone is approved, several thousand cells are injected back into another embryo (also at 65 hours old). After incubation, the egg hatches and becomes a chicken. 

    Kanuma set the precedent—but not the scale

    In 2015, the U.S. Food and Drug Administration approved Kanuma (sebelipase alfa) to treat Lysosomal Acid Lipase (LAL) deficiency, also known as Wolman disease. The drug, an enzyme replacement therapy, was the first treatment for patients with the rare disease and the first drug to be made in chickens. Kanuma is produced by Alexion Pharmaceuticals, which was acquired by AstraZeneca in 2021.  

    This historical precedent may provide a proof of concept for Neion Bio. That said, “the scale required for Kanuma is very different from what would be needed for large biosimilars,” explained Wlodek.  

    An Odyssean journey

    For both Bocklandt and Kehler, the move to Neion Bio feels like their careers are coming full circle. When Bocklandt first left Colossal, he was not sure how he would surpass that level of excitement. But the move came at an interesting time for him; the call to join Neion Bio came just weeks after he learned that his sister had been diagnosed with leukemia.  

    He thought, “Well, maybe this is not such a bad use of my skills.”  

    Earlier in his career, he didn’t think that he had anything special to add to a field like cancer research. But now Bocklandt sees it differently: throughout his career, he has pushed the state-of-the-art of genetic engineering. Now, he said, “I bring something to the field. And the fact that I can do my passion, animal genetic engineering, and apply that to make drugs better, cheaper, and more accessible, is really exciting.”  

    As for Kehler, Neion’s goal was his goal all along. He went to the University of Pennsylvania to make better animal models to test drugs for humans. “It never really dawned on me that we could use animals to make the drugs for humans. But taking everything I know about stem cell biology, germ cell biology, and gene editing, and bringing that to bear to make what should be a disruptive, transformational approach to making drugs—it feels like the culmination of my career.” 

    Neion (pronounced Neon) Bio is named after the birthplace of Odysseus; Mount Neion is a mountain mentioned in Homer’s The Odyssey as a landmark on Ithaca—Odysseus’ island home. As described by the company, the name is a testament to the shared qualities between the Greek hero and the company’s goals: relying on intelligence and resourcefulness over strength. And yes, Odysseus was successful in his return home to reclaim his throne. But it was a bittersweet success given the enormous cost and hardship.

    Neion Bio’s name may mirror the resilience and ingenuity required to undertake the journey, but time will tell how long the similarities in the namesake are shared between the two.

    The post From Colossal to Chickens: The Scientists Behind Neion Bio’s Biologics Platform appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AWS Launches Amazon Bio Discovery Agentic AI to Accelerate Drug Development https://edusehat.com/en/aws-launches-amazon-bio-discovery-agentic-ai-to-accelerate-drug-development https://edusehat.com/en/aws-launches-amazon-bio-discovery-agentic-ai-to-accelerate-drug-development Wed, 15 Apr 2026 00:45:09 +0700 Edusehat AWS, Launches, Amazon, Bio, Discovery, Agentic, Accelerate, Drug, Development AWS has now unveiled Amazon Bio Discovery, an AI platform that grants researchers direct access to a broad library of biological foundation models that can be fine-tuned for specific use cases in drug discovery. Announced at the AWS Life Sciences Symposium at the Javits Center in New York, the platform is supported by an AI agent that can select models for research goals, and evaluate candidates for synthesis and testing to enable a rapid lab-in-the-loop experimentation cycle.  

    While rising AI models show promise, they require coding skills and the ability to manage computing infrastructure. Additionally, diverse models face benchmarking challenges and moving candidates from computational design to physical synthesis remains a multi-step process. Given that data live in disconnected systems, scientists must manage multiple lab partners and manually coordinate timelines and execution.

    Amazon Bio Discovery addresses these challenges with three capabilities: a benchmarked library of AI models and analysis packages, an AI agent that supports experimental lab, and integrated lab partners that test top antibody candidates and route results back to the researchers. This feedback loop improves the next round of design. 

    “AI agents make powerful scientific capabilities accessible to all drug researchers, not just those with computational expertise,” said Rajiv Chopra, PhD, vice president of AWS Healthcare AI and Life Sciences.  

    Currently, 19 of the top 20 global pharmaceutical companies use AWS to power research workloads. Amazon Bio Discovery will bring enterprise-grade scale, privacy, and security to researchers across pharmaceutical, biotech, and academic research organizations. MSK, Bayer, the Broad Institute, and Voyager Therapeutics are among early adopters of Amazon Bio Discovery. 

    Among the Amazon Bio Discovery broad catalog includes open-source and commercial models from Apheris and Boltz. Biohub and Profluent are expected to join the platform. 

    Amazon Bio Discovery enables scientists to fine-tune the model by feeding prior experimental data from their organization’s lab results into the application without complex training pipelines or custom code. In-house models can also easily be deployed and hosted within Amazon Bio Discovery.  

    To support model selection, an antibody benchmark dataset is available to evaluate the likelihood of a drug candidate to have favorable biological properties, such as manufacturability and stability. 

    Candidates selected for experimental validation can be directly sent to Amazon Bio Discovery’s integrated network of laboratory partners, including Twist Bioscience, Ginkgo Bioworks. A-Alpha Bio is also anticipated to join the network. 

    The post AWS Launches Amazon Bio Discovery Agentic AI to Accelerate Drug Development appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Building the Genome Grid https://edusehat.com/en/building-the-genome-grid https://edusehat.com/en/building-the-genome-grid Tue, 14 Apr 2026 06:40:15 +0700 Edusehat Building, the, Genome, Grid Sponsored content brought to you by

    MGI logo

    A decade ago, gene sequencing was still defined by scarcity. Instruments were concentrated in elite labs. Large studies were rare. Budgets dictated ambition. Today, genomics looks less like a boutique research tool and more like public infrastructure—scaled, automated, globally distributed. MGI’s 10-year trajectory offers a lens into how that shift happened—and why it matters.

    “Over the past decade, lower cost and greater scale have shifted genomics from isolated experiments to shared infrastructure,” says Duncan Yu, president of MGI. That phrase—shared infrastructure—captures the transformation more clearly than any performance benchmark.

    In 2018, MGI launched the T7 sequencer, capable at the time of processing up to 60 human genomes per day. The milestone wasn’t just about throughput; it signaled a new operational mindset. Sequencing at scale, Yu argues, “is not simply doing the same thing more times. It introduces challenges in workflow, consistency, maintenance, and data handling.”

    Those challenges are invisible to most outside the lab, but they are what enable population-scale genomics. Over the past decade, national initiatives have expanded from tens of thousands of samples to hundreds of thousands of genomes. That leap required platforms that ran continuously, predictably, and accurately. “This is what allows genomics to support national programs, large population initiatives,” Yu says.

    Cost has been the most public battleground. Launched in 1990, the Human Genome Project required 13 years and nearly $3 billion. By 2007, sequencing had dropped to roughly $1,000 per genome—still too high for most clinical settings. Sustained engineering drove even more economic improvements in sequencing. “By pushing costs down sustainably, we helped shift genomics from small, selective studies to population-scale research, where entirely new questions can be asked,” Yu explains.

    Affordability alone, however, does not democratize science. Deployment does. Over the past decade, MGI expanded globally—not just by shipping systems, but by building local capacity. “Access means global deployment, local training, service support,” Yu says. “We’ve spent years working with partners around the world to ensure sequencing capacity is not concentrated in a few regions, but can be built and sustained locally.”

    This redistribution of capability changes where innovation happens. When sequencing is locally available, researchers can tackle region-specific diseases, agricultural resilience, or environmental risks without outsourcing core analysis abroad. “By lowering structural barriers, sequencing has become part of everyday scientific and public infrastructure,” Yu adds.

    Technology evolution followed that philosophy. In 2022, MGI introduced new platforms, including the self-luminous sequencing–based E25 and the fast mid-throughput G99, emphasizing flexibility alongside performance. In 2023, the launch of T20x2 pushed throughput to a new level—up to 50,000 whole genomes per year—at a record-breaking cost profile of less than $100. By 2025, benchtop and ultra-high-throughput systems such as T1+ and T7+ reflected a design priority: faster turnaround without sacrificing scalability. Moreover, the sub-$100 milestone crossed a psychological threshold: sequencing could move from selective research into routine health infrastructure.

    Yet Yu is careful to shift attention away from individual machines. “When people talk about genomics, they often think about machines or breakthroughs,” he says. “But an open ecosystem and infrastructure are something different. It means sequencing is no longer an exception or a privilege, but a dependable foundation for science, healthcare, and public decision-making.”

    Artificial intelligence (AI) has become essential to that foundation. As datasets ballooned, AI-driven quality control and variant detection moved from experimental to indispensable. Looking forward, Yu sees AI’s role expanding beyond the speed of analysis. “We expect AI to help connect sequencing data with other data types and be well integrated into sustainable, trusted sequencing ecosystems,” he says.

    In February 2026, MGI expanded its genomics footprint with the acquisitions of STOmics and CycloneSeq, adding spatiotemporal multi-omics and nanopore sequencing to its portfolio. The move positions MGI Tech as the only provider uniting short-read, long-read, and stereo-seq spatial transcriptomics in one ecosystem. Researchers have often stitched together platforms from different vendors, risking reagent mismatches, calibration drift, workflow friction, and data silos. Those cracks widen at scale, driving redundant validation and operational drag. By consolidating technologies, MGI offers labs a coherent framework that blends throughput, structural insight, and spatial context, enabling flexible strategy without rebuilding infrastructure at every stage. The goal is not just to read genomes faster, but to integrate layers of biological insight into cohesive systems that can serve healthcare and research at scale.

    The past decade proved that sequencing could be made faster and cheaper. The deeper achievement was structural: turning genomics into durable infrastructure. If the next decade succeeds, sequencing will not be a headline-grabbing breakthrough. It will be something more powerful—ordinary, embedded, and everywhere it needs to be.

    The post Building the Genome Grid appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Advancing Fully Walkaway Automation in Genomics Workflows https://edusehat.com/en/advancing-fully-walkaway-automation-in-genomics-workflows https://edusehat.com/en/advancing-fully-walkaway-automation-in-genomics-workflows Tue, 14 Apr 2026 06:40:14 +0700 Edusehat Advancing, Fully, Walkaway, Automation, Genomics, Workflows SPT Labtech and the European Molecular Biology Laboratory’s Genomics Core Facility (EMBL GeneCore) in Heidelberg, Germany, agreed to collaborate to advance fully walkaway automated genomics workflows. As part of the collaboration, SPT Labtech’s firefly®+ all-in-one liquid handling platform has been installed at EMBL GeneCore.

    Officials at EMBL GeneCore say they will expand the facility’s capacity to develop new protocols and further validate and optimize existing workflows for challenging applications such as low-input and metagenomics samples to support the broader genomics community. The SPT platform is designed to simplify complex genomics workflows, combining pipetting, dispensing, incubating, and shaking technologies into a single instrument.

    The automated protocols use New England Biolabs (NEB) library preparation kits, NEBNext®, to generate libraries from a wide input range. According to a SPT spokesperson, the installation of the company’s firefly+ platform at EMBL GeneCore, combined with NEB kits, creates strong foundation for fully walkaway automation, enabling more streamlined, end-to-end workflows and supporting labs to scale automation more easily.

    “The installation of SPT Labtech’s firefly+ platform as part of our collaboration underscores our commitment to remain at the forefront of scientific innovation. Fully walkaway automation will address key bottlenecks in genomics workflows, helping us develop high-quality, scalable NGS protocols,” says Vladimir Benes, head of EMBL GeneCore.

    “Our latest collaboration with EMBL GeneCore marks a significant step towards advancing fully walkaway automation, providing end-to-end genomics workflows for a much wider range of applications, including environmental and rare species research,” adds Morten Frost, CCO, SPT Labtech.

    “Integration of our library prep kits with SPT Labtech’s firefly+ platform at EMBL GeneCore creates a compelling opportunity for faster, scalable DNA and RNA-Seq workflows,” notes Bjoern Textor, PhD, sales and senior applications manager, New England Biolabs.

    The post Advancing Fully Walkaway Automation in Genomics Workflows appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     AACR 2026: Professional Awards Acknowledge Community’s Contributions to Cancer Research https://edusehat.com/en/aacr-2026-professional-awards-acknowledge-communitys-contributions-to-cancer-research https://edusehat.com/en/aacr-2026-professional-awards-acknowledge-communitys-contributions-to-cancer-research Tue, 14 Apr 2026 06:40:12 +0700 Edusehat AACR, 2026:, Professional, Awards, Acknowledge, Community’s, Contributions, Cancer, Research The American Association for Cancer Research (AACR) has released the names of the recipients of several annual professional awards. These awards recognize outstanding accomplishments and achievements in cancer research, therapy development, education, mentorship, and more. The honorees, listed below, will give lectures during this year’s meeting, which is being held in San Diego, CA. This year’s meeting runs from April 17-22.

     

    AACR Lifetime Achievement in Cancer Research Award 

    James P. Allison, PhD, FAACR

    This award honors individuals who have made fundamental contributions to cancer research through a single scientific discovery or a body of work. Allison is being recognized for his contributions to cancer research and patient care. Most notably, he is being celebrated for his identification of CTLA-4 as a negative regulator of T-cell activation, an insight that has since been translated into a first-in-class therapy that revitalized the field of cancer immunology and led to a revolution in cancer immunotherapies. Allison is a fellow of the AACR Academy, a professor and chair of the department of immunology, vice president for immunobiology, and the founding director of the James P. Allison Institute at the University of Texas MD Anderson Cancer Center.

     

    AACR Outstanding Achievement in Basic Cancer Research Award 

    Housheng Hansen He, PhD

    This award recognizes early-career investigators for meritorious achievements in basic cancer research. He is a professor in the department of medical biophysics at the University of Toronto and a senior scientist in the Princess Margaret Cancer Center. He is recognized for contributions to cancer epigenetics and RNA medicine, particularly in revealing how chromatin accessibility and epigenomic landscapes govern oncogenic transcription. His studies of FOXA1-androgen receptor networks, noncoding RNAs, and RNA modifications have improved scientists’ understanding of tumor progression, plasticity, and therapeutic resistance.  

     

    AACR Outstanding Achievement in Blood Cancer Research Award 

    John F. DiPersio, MD, PhD

    This award recognizes individuals with meritorious achievements and contributions to blood cancer research. DiPersio is this year’s recipient for his work in leukemia and stem cell biology, including essential contributions to the development of the hematopoietic stem cell mobilizing agents plerixafor and motixafortide. DiPersio identified AK1/2 signaling in graft-versus-host disease, which led to the identification and approval of JAK inhibitors, including ruxolitinib (Jakafi). DiPersio is the Virginia E. and Sam J. Golman professor of medicine and a professor of medicine, immunology, and pathology at Washington University School of Medicine in St. Louis. He is also director of the Center for Gene and Cellular Immunotherapy at Siteman Cancer Center at Barnes-Jewish Hospital and WashU Medicine.

     

    AACR Outstanding Achievement in Chemistry in Cancer Research Award 

    Cheryl H. Arrowsmith, PhD

    This award honors individuals who have done novel and significant chemistry research that has led to important contributions in basic and translational cancer research, cancer diagnosis, prevention, and treatment. Arrowsmith is being recognized for foundational studies defining the structure and function of chromatin-associated proteins that regulate gene expression in cancer. Her work enabled the development of chemical probes that target epigenetic regulators. She is a senior scientist at the Princess Margaret Cancer Centre, University Health Network and chief scientist of the Structural Genomics Consortium. She is also a professor in the department of medical biophysics at the University of Toronto.

     

    AACR Daniel D. Von Hoff Award for Outstanding Contributions to Education and Training in Cancer Research

    Charles W.M. Roberts, MD, PhD, FAACR

    This award recognizes significant contributions to education and training for cancer scientists and physicians at any career level. Roberts is a fellow of the AACR Academy and the executive vice president and director of the St. Jude Comprehensive Cancer Center. He is also a member in the department of oncology and the Lillian R. Cannon Comprehensive Cancer Center Director Endowed Chair at the St. Jude Children’s Research Hospital. This award recognizes his leadership and dedication to the education and training of cancer researchers across the spectrum of childhood cancer research, including basic, translational, clinical, and population science. 

     

    AACR James S. Ewing-Thelma B. Dunn Award for Outstanding Achievement in Pathology in Cancer Research 

    David L. Rimm, MD, PhD

    The award celebrates pathologists who have contributed to advancing cancer research, diagnosis, treatment, and prevention. Rimm is recognized this year for innovations in quantitative biomarker science that transformed cancer diagnostics and treatment. His invention of the fluorescence-based Automated Quantitative Analysis platform improved immunohistochemistry by enabling precise, reproducible protein quantification in tissue specimens. Rimm is the Anthony N. Brady professor of pathology, a professor of medicine in oncology, director of quantitative diagnostics in the anatomic pathology lab, director of Yale Pathology Tissues Services, and director of the physician scientist training program in pathology at Yale University School of Medicine. He is also a member of Yale Cancer Center and director of the Yale Cancer Center Tissue Microarray Facility.

     

    AACR Margaret Foti Award for Leadership and Extraordinary Achievements in Cancer Research

    Antoni Ribas, MD, PhD, FAACR

    This award recognizes individuals whose leadership and achievements contributed to the acceleration of progress against cancer, raising national or international awareness of the importance of cancer research, among other achievements. Ribas is being recognized for contributions to melanoma biology and cancer immunotherapy that were instrumental to the clinical development of pembrolizumab (Keytruda) and other transformative therapies. His research helped define mechanisms of immunotherapy response and resistance, which guided the design of innovative combination therapy approaches. Ribas is a fellow of the AACR Academy and AACR Past President. He is also a professor of medicine, surgery, and molecular and medical pharmacology at the University of California Los Angeles (UCLA), as well as director of the tumor immunology program at the UCLA Jonsson Comprehensive Cancer Center. He also serves as the director of the Parker Institute for Cancer Immunotherapy Center at UCLA. 

     

    AACR Team Science Award

    The Cancer Dependency Map (DepMap) team

    This award recognizes interdisciplinary research teams for science that advances or is likely to advance our fundamental knowledge of cancer, or a team that has applied existing knowledge to advance the detection, diagnosis, prevention, or treatment of cancer. The Broad Institute Cancer Dependency Map (DepMap) team is recognized this year for systematically mapping genetic dependencies across cancer cells and creating a comprehensive resource that reveals genes and pathways essential for tumor survival. By combining large-scale CRISPR functional genomic screens, drug response data, and multiomic profiling, the team uncovered lineage- and genotype-specific cancer vulnerabilities, including synthetic lethal dependencies such as WRN in microsatellite instability cancers and PRMT5 dependencies in cancers with MTAP deletions. 

     

    AACR American Cancer Society Award for Research Excellence in Cancer Epidemiology and Prevention

    Elizabeth A. Platz, ScD, MPH 

    This award recognizes research accomplishments in cancer epidemiology, biomarkers, and prevention. Platz is the Martin D. Abeloff, MD Scholar in Cancer Prevention in the epidemiology department at the Johns Hopkins Bloomberg School of Public Health. She is also the associate director of population sciences at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. This award recognizes her contributions to scientists’ understanding of prostate cancer development, progression, and prevention. Her research linked intraprostatic inflammation to prostate cancer risk, identified telomere length patterns as prognostic biomarkers, and demonstrated protective associations between statin use, cholesterol, and disease lethality. 

     

    AACR Cancer Research Institute Lloyd J. Old Award in Cancer Immunology 

    Kenneth M. Murphy, MD, PhD 

    This award recognizes scientists whose research has had a major impact on the cancer field and has the potential to stimulate new directions in cancer immunology.  Murphy is the Eugene Opie First Centennial Professor in pathology and immunology at the Washington University School of Medicine in St. Louis. This award recognizes his work on discoveries related to the development and functional specialization of dendritic cell subsets that regulate adaptive immune responses. His work elucidated the transcriptional programs that control dendritic cell lineage commitment, including the role of transcription factors such as BATF3 in the development of cross-presenting dendritic cells required to prime cytotoxic T-cell responses. 

     

    AACR G.H.A. Clowes Award for Outstanding Basic Cancer Research 

    Andrew P. Feinberg, MD, MPH

    This award, which has the distinction of being AACR’s oldest award, recognizes individuals who have made outstanding recent accomplishments in basic cancer research. Feinberg is recognized this year for discoveries about the fundamental role of epigenetic alterations in cancer, including the identification of early, widespread DNA methylation abnormalities and the role of genomic imprinting in tumor development. His research demonstrated that large-scale epigenomic alterations contribute to tumor initiation, progression, and cellular heterogeneity, leading to the concept of epigenetic plasticity as a driver of cancer evolution. Feinberg is the Bloomberg Distinguished Professor at the Johns Hopkins University Schools of Medicine, Engineering, and Public Health. He also serves as director of the Center for Epigenetics of the Institute for Basic Biomedical Sciences.

     

    AACR Irving Weinstein Foundation Distinguished Lectureship Award

    Dennis Lo, DM, DPhil

    The recipient for this award is selected by the AACR president, and acknowledges individuals whose personal innovation in science and whose position as a thought leader in fields relevant to cancer research have the potential to inspire creative thinking and new directions in cancer research. Lo is the vice-chancellor and president of the Chinese University of Hong Kong, where he also serves as the Li Ka Shing Professor of Medicine and professor of chemical pathology. He is being recognized for his discovery of fetal DNA in maternal plasma. Lo was the first to identify cell-free fetal DNA and fetal epigenetic markers in maternal plasma, enabling safer and earlier prenatal diagnostics. He also demonstrated that DNA released by tumors may be used for cancer screening, an insight that led to the development of circulating DNA-based tools for early cancer detection and screening. 

     

    AACR Joseph H. Burchenal Award for Outstanding Achievement in Clinical Cancer Research 

    Luis A. Diaz Jr., MD, FAACR

    This award recognizes outstanding achievements in clinical cancer research. Diaz, a fellow of the AACR Academy, heads the division of solid tumor oncology and is the Grayer Family Chair at the Memorial Sloan Kettering Cancer Center. This award recognizes his pioneering discoveries such as biomarker-driven immunotherapies and for demonstrating that tumors with mismatch repair deficiencies and microsatellite instability are highly responsive to immune checkpoint blockade. Diaz has also advanced the use of circulating tumor DNA to detect minimal residual disease and led clinical trials of PD-1 blockade in mismatch repair-deficient cancers. 

     

    AACR Minorities in Cancer Research Jane Cooke Wright Lectureship

    Ahmedin M. Jemal, DVM, PhD

    This lectureship recognizes scientists with meritorious contributions to the field of cancer research and who have furthered the advancement of minority investigators in cancer research. This year’s awardee is recognized for research that quantified temporal and geographic trends in cancer burden using large-scale analysis of cancer registries, mortality rates, and risk factor data, and identified population-level determinants of cancer incidence, survival, and stage at diagnosis across demographic groups. Jemal’s work linked changes in risk factor exposure, screening uptake, and treatment advances to declines in cancer mortality and informed strategies for cancer prevention, early detection, and population-level cancer control. He is the senior vice president of the Surveillance, Prevention, & Health Services Research department at the American Cancer Society. He is also an adjunct professor in the department of epidemiology at the Rollins School of Public Health at Emory University. 

     

    AACR Princess Takamatsu Memorial Lectureship 

    David C. Lyden, MD, PhD

    This award recognizes individual scientists whose work has had or may have a far-reaching impact on the detection, diagnosis, treatment, or prevention of cancer. Lyden is the Stavros S. Niarchos Professor in pediatric cardiology and professor of pediatrics at Weill Cornell Medicine. He is also director of the physician-scientist training program in pediatrics, a founding member of the Drukier Institute for Children’s Health  and a member of the Sandra and Edward Meyer Cancer Center. He is being recognized for describing how primary tumors systemically promote metastasis by forming pre-metastatic niches in distant organs. Lyden’s research demonstrated that tumor-derived extracellular vesicles and exomeres, together with bone marrow-derived progenitor cells, remodel distant microenvironments and determine organ-specific metastatic tropism.

     

    AACR St. Baldrick’s Foundation Award for Outstanding Achievement in Pediatric Cancer Research

    Kimberly Stegmaier, MD, FAACR

    This award recognizes individuals who have contributed to pediatric cancer research, resulting in the fundamental improvement of the understanding and/or treatment of pediatric cancer. Stegmaier serves as chair in the department of pediatric oncology at Dana-Farber Cancer Institute and the David G. Nathan professor of pediatrics at Harvard Medical School. She is also the associate chief of the division of hematology/oncology at Boston Children’s Hospital and an institute member at the Broad Institute. This award recognizes her genomic discoveries that defined the molecular landscape of childhood cancers and led to the identification of key drivers of fusion oncoprotein positive malignancies. Her research used systematic functional genomic screening and chemical biology strategies to identify critical dependencies in high-risk acute leukemias and pediatric solid tumors. 

     

    AACR Waun Ki Hong Award for Outstanding Achievement in Translational and Clinical Cancer Research

    Eliezer M. Van Allen, MD

    This award recognizes cancer researchers under the age of 51 who have conducted meritorious translational and clinical cancer research anywhere in the world. Van Allen is the Chandra Nohria Family Chair for AI in Cancer Research and chief of the division of population sciences at Dana-Farber Cancer Institute. He is also a professor of medicine at Harvard Medical School and an institute member at the Broad Institute. Through large-scale tumor sequencing and integrative genomic analyses, Van Allen’s research defined genomic mechanisms underlying resistance to targeted therapies, including BRAF inhibition in melanoma, and identified genomic features associated with response to immune checkpoint blockade. His work has advanced biomarker discovery and the use of genomic data to guide personalized cancer treatments, as well as bridged advances in artificial intelligence with translational cancer research.

     

    AACR Women in Cancer Research Charlotte Friend Lectureship 

    Maryellen L. Giger, PhD

    This award recognizes scientists’ contributions to the cancer research field and those who have furthered the advancement of women in science through leadership or by example. Giger is the A.N. Pritzker Distinguished Service Professor of Radiology at the University of Chicago. Giger’s research has established quantitative imaging and radiomics approaches that extract high-dimensional features from radiologic images to characterize tumor phenotype and predict cancer risk, diagnosis, and treatment response. She has also guided more than 120 trainees and consistently championed the careers of women scientists and clinicians.

     

    Pezcoller Foundation-AACR International Award for Extraordinary Achievement in Cancer Research

    Douglas R. Lowy, MD, FAACR and John T. Schiller, PhD, FAACR

    This award is presented to international scientists who have made a scientific discovery in basic cancer research or who have made significant contributions to translational cancer research. Lowy is principal deputy director of the National Cancer Institute (NCI) and chief of the Laboratory of Cellular Oncology at NCI. Schiller is deputy chief of the Laboratory of Cellular Oncology at NCI and chief of the lab’s neoplastic disease section. Both awardees are also fellows of the AACR Academy and NIH Distinguished Investigators. They are being recognized for pioneering the molecular and immunologic foundations of human papillomavirus vaccines, engineering virus-like particles for safe and effective immunization, and driving their translation into global cancer prevention strategies that have dramatically reduced cervical and other HPV-related cancer incidence.  

    The post AACR 2026: Professional Awards Acknowledge Community’s Contributions to Cancer Research appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Salk to Lead $41.3M ARPA-H Effort to Advance Sonogenetics Therapies https://edusehat.com/en/salk-to-lead-413m-arpa-h-effort-to-advance-sonogenetics-therapies https://edusehat.com/en/salk-to-lead-413m-arpa-h-effort-to-advance-sonogenetics-therapies Tue, 14 Apr 2026 06:40:11 +0700 Edusehat Salk, Lead, 41.3M, ARPA-H, Effort, Advance, Sonogenetics, Therapies The scientists responsible for developing sonogenetics, which refers to the application of low-intensity ultrasound to noninvasively achieve precise control of cellular proteins, have secured an influx of federal funding that will allow them and their collaborators transform the technology into a potential therapy for various conditions starting with peripheral neuropathies.

    Late last week, Salk Institute for Biological Studies announced that Sreekanth Chalasani, PhD, an associate professor in Salk’s molecular neurobiology laboratory, and his partners in collaborating laboratories elsewhere, were awarded $41.3 million from the Advanced Research Projects Agency for Health (ARPA-H), an agency within the United States Department of Health and Human Services. Working on multiple fronts over the next five years, the partners will develop core biological tools and ultrasound delivery systems while generating the preclinical evidence needed to move sonogenetics into human clinical trials. 

    “This award is a major step toward a long-held goal—a drug-free way to deliver therapy exactly where it’s needed and only when it’s needed,” said Chalasani, who serves as the principal investigator for the grant and is also the co-founder of SonoNeu, a startup launched to commercialize therapies based on the technology. Key to accomplishing that goal is “a platform that pairs engineered ultrasound-sensitive proteins with wearable ultrasound technology, which, unlike conventional pharmaceutical treatments, could let us treat conditions with cellular and temporal control.” 

    Reaching cells through sound

    In 2011, armed with support from Salk’s Innovation and Collaboration Grants program, Chalasani and his team pioneered sonogenetics, a technique for sensitizing specific cell types to ultrasound by equipping them with ultrasound-responsive proteins. In 2015, his group first identified a particular protein in the roundworm Caenorhabditis elegans (C. elegans) that makes cells sensitive to low-frequency ultrasound. When they added this protein to C. elegans neurons that did not usually have it, they were able to activate these cells using ultrasound waves. 

    Since that initial discovery, Chalasani’s team and others have shown that they can use sonogenetics to manipulate mammalian cells. Some of their work was published in a 2022 Nature Communications paper which describes efforts to engineer a human channel protein in cultured mammalian cells and living animal models to confer cell-specific sensitivity to ultrasound stimulation.

    Chalasani noted in an interview with GEN that the pace of their progress from an idea to potential clinical translation in the span of about 15 years is remarkable, compared to the typical multi-decade timeline for most new therapies. “In terms of how quickly this has gone from a [research] idea to what patients should we look at [and] how are we going to help them? The pace has been overwhelming,” he said. He attributed much of that progress to the work of the trainees, post-doctoral students, and collaborators through the years who were willing to take on “this crazy idea and work on this project, even though there was no guarantee it would get anywhere.” He also highlighted the early investment from Salk and other entities including the National Institutes of Health’s Brain Initiative as key to project’s success.  

    Though Chalasani and his lab pioneered sonogenetics, the next phase of its development is not a solo effort. It involves a multiple institutions and teams all of whom are contributing essential and specialized expertise. The list of collaborators includes Scripps Research, where a team led by 2021 Nobel Laureate Ardem Patapoutian, PhD, will support the discovery and engineering of ultrasound-sensitive proteins. Then a team at St. Boniface Hospital Research and the University of Manitoba led by Paul Fernyhough, PhD, will help define how ultrasound-triggered signals move through cellular machinery and drive nerve repair pathways.

    Another team, led by Aravind Asokan, PhD, at Duke University will work on targeted vectors for delivering ultrasound-sensitive proteins to specific cell types. Separately, a team led by Xuanhe Zhao, PhD, at Massachusetts Institute of Technology will work on targeted mechanisms for delivering ultrasound to animal and human targets. Then scientists at the University of California, San Diego, led by Nigel Calcutt, PhD, will validate the efficacy of sonogenetics across established paradigms and in mammalian systems. Finally, Ghassan Kassab, PhD, and his team at California Medical Innovations Institute, will support advanced translational validation and clinically relevant assessment in preclinical systems. 

    For their part, Chalasani and his team at Salk will work on finding additional actuators or sensors for ultrasound that will be optimized for targeted delivery. “What we have are proteins that can respond to that small amount of mechanical deflection that ultrasound can cause,” Chalasani explained. “These proteins are channels [that] sit on the membrane of the cell and when the cell experiences ultrasound, the protein gets activated, it opens up, and allows calcium [for example] into the cell. Because these proteins can actuate an effect, they are called ultrasound actuators.” 

    Currently, the group has identified proteins that can move things like calcium and chloride into cells in response to ultrasound but they are hunting for other proteins that can activate various signaling pathways. Besides new actuators, the Salk scientists will work on validation studies in mouse models, and lay the groundwork for experiments in larger animal models, specifically pigs, Chalasani told GEN. 

    There is a plan in place to commercialize therapies developed using sonogenetics technology. Salk spinout SonoNeu will receive a portion of the ARPA-H funding to help  move potential therapies through the regulatory process and commercialization. Chalasani is listed as a co-founder as is Venkat Reddy, chief scientific officer of General Inception, a firm that partners with scientific founders to build their companies. The target, Chalasani said, is to have something ready for the U.S. Food and Drug Administration in the next five years. 

    The initial treatment target condition is peripheral neuropathies and in that context, “there are a lot of interesting places for us to evaluate,” he said. “But I think the real question is going to be what pathway are we targeting? We can do calcium and chloride [but] is that enough to get a therapeutic benefit in a patient or should we have to activate something else other than that? Do we have to activate an enzyme [or] a kinase? And how would we do that? Can we link these proteins to those signals? So there are some unknowns here.”  

    If all goes well, other patient populations could benefit from sonogenetics-based therapies besides peripheral neuropathies including people with diabetes, heart conditions or it could help with bladder control, Chalasani said. There are even possible applications in the context of brain-computer interfaces. 

    The post Salk to Lead $41.3M ARPA-H Effort to Advance Sonogenetics Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Sex-Related Differences in Immune System Aging May Impact Disease Susceptibility https://edusehat.com/en/sex-related-differences-in-immune-system-aging-may-impact-disease-susceptibility https://edusehat.com/en/sex-related-differences-in-immune-system-aging-may-impact-disease-susceptibility Tue, 14 Apr 2026 06:40:10 +0700 Edusehat Sex-Related, Differences, Immune, System, Aging, May, Impact, Disease, Susceptibility Immune system aging, known as immunosenescence, is associated with changes in immune cell composition and function that increase susceptibility to disease. The results of a study by researchers at the Barcelona Supercomputing Center – Centro Nacional de Supercomputación (BSC–CNS) have now shown that immunological aging follows different dynamics between men and women.

    The team analyzed single-cell RNA sequencing data from the peripheral blood mononuclear cells (PBMCs) of 982 female and male donors across adulthood, to identify cells and genes involved in immunosenescence, and potentially provide a molecular explanation for the differences that had previously only been observed globally in the population.

    “Until now, most studies analyzed the immune system based on the average of many cells at once, which makes it difficult to capture the progressive effects of aging,” said Maria Sopena-Rios, PhD. “With cell-by-cell analysis and a much larger sample, we were able to detect these patterns and compare them robustly between biological sexes.” Their strategy identified sex-related differences in immunological aging may help to explain why women have an 80% higher incidence of autoimmune diseases than do men, and why men are more likely than women to develop hematological cancers and chronic infections.

    Sopena-Rios is co-first author of the team’s published paper in Nature Aging, titled “Single-cell analysis of the human immune system reveals sex-specific dynamics of immunosenescence,” in which the investigators concluded “Together, our findings provide a high-resolution map of sex-specific immune aging and lay the groundwork for tailored sex-specific strategies to monitor and improve immune health across the lifespan.”

    Statistics show clear differences in the population’s immune system according to sex: men are more susceptible to infections and cancers, while women have stronger immune responses, which translate, for example, into better responses to vaccines. Even so, with a more reactive immune system, the probability of the body attacking itself also increases, causing 80% of autoimmune disease development to occur in women. In this context, understanding the aging of the immune system is key since, with age, the composition of immune cells changes and their protective functions deteriorate, causing a greater susceptibility to diseases.

    Aging of the immune system, or immunosenescence, refers to “… the gradual decline of the immune system, which predisposes to multiple diseases, including infection, cancer, and autoimmune and vascular diseases,” the authors wrote. “Importantly, the age-related decline of the immune system involves both changes in the composition of immune cell populations and molecular alterations.” However, understanding how sex influences this profound transformation was not possible until now. “…how biological sex shapes immune aging at the cellular level remains poorly understood,” the investigators stated.

    For their reported study, the team analyzed blood samples from nearly 1,000 people of different ages covering the entire adult life, and carried out single-cell RNA sequencing to analyze the activity of 20,000 genes in more than one million blood cells. This approach allowed them to identify how the immune system changes over the years and detect clear differences between sexes. Although evidence existed that the immune system ages differently according to sex, women have been traditionally underrepresented in studies, the authors comment. This is the first time that large quantities of samples were analyzed with a balance between men and women, a fact that was decisive in obtaining these results.

    “Many studies still do not take sex into account in their analyses, or directly only use data from men, so they leave key questions unanswered,” said study director Marta Melé, PhD, leader of the Transcriptomics and Functional Genomics group at BSC. “Our research was born precisely from this need and combines a scientific outlook with a sex perspective, inclusive data, and great computational power.”

    BSC researchers Aida Ripoll-Cladelles (left), Marta Melé (center) and Maria Sopena-Rios (right) in front of MareNostrum 5 supercomputer. [Mario Ejarque / BSC-CNS]
    BSC researchers Aida Ripoll-Cladelles (left), Marta Melé (center) and Maria Sopena-Rios (right) in front of MareNostrum 5 supercomputer. [Mario Ejarque / BSC–CNS]
    The results revealed that women present more pronounced changes in the immune system with age, with an increase in inflammatory immune cells. This finding could help explain why autoimmune diseases are mainly developed by women, especially at advanced ages, as well as the worsening of certain inflammatory pathologies after menopause.

    “Female individuals typically mount stronger immune responses, enhancing resistance to infections and vaccine efficacy, but also contributing to an 80% higher incidence of autoimmune diseases,” the authors noted. “Aging further increases autoimmune risk, and we showed that this is accompanied by the female expansion of cell subpopulations with pivotal roles in autoimmunity.”

    On the other hand, the study found that changes associated with immune system aging observed in men are globally less extensive, but an increase in certain blood cells presenting pre-leukemia alterations was observed, a fact that could explain why some blood cancers are more frequent in older men. “Male immune aging is less transcriptionally pronounced, with fewer sex-specific signatures and subtler shifts in immune cell abundance,” the investigators wrote. “.… a subset of male participants shows an age-associated expansion of a B cell population linked to an asymptomatic precursor state of chronic lymphocytic leukemia … Our findings suggest that male immune remodeling may contribute to increased vulnerability to hematological malignancies and chronic infections.”

    To manage, process, and analyze a volume of data of this magnitude, the scientific team required the use of advanced computational methods that had never been applied to such complex data sets, with the MareNostrum 5 supercomputer as a key piece to make possible a study that would not have been viable without high-performance computing infrastructure.

    With these discoveries, the study establishes the bases for incorporating biological sex as a key variable in precision medicine for aging. The identification of sex-specific aging cells and biomarkers opens the door to the development of preventive, diagnostic, and therapeutic strategies better adapted to women and men, contributing to more individualized and equitable healthcare in an increasingly aging population.

    “The immune system plays a fundamental role throughout the organism; therefore, the differences we observed have a very important generalized impact on the entire body. Better understanding the aging of the immune system can help us understand processes that go beyond the blood and affect multiple tissues,” noted co-first author Aida Ripoll-Cladellas, PhD.

    Treating aging as a homogeneous process in the entire population hides key biological differences, and understanding how it varies between women and men, the authors concluded, will be essential to improve immune health and promote healthy aging within everyone’s reach. “Stratifying analyses according to sex uncovers key sex-specific features of immunosenescence that may otherwise be misinterpreted as shared effects,” they stated. “This underscores the importance of considering sex as a biological variable to ensure biologically accurate conclusions. Ultimately, our findings lay the foundation for sex-tailored strategies to monitor immune aging and mitigate the burden of age-related immune dysfunction.”

    The post Sex-Related Differences in Immune System Aging May Impact Disease Susceptibility appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Neural Mechanism Underlying Sensory Behavior Revealed in C. elegans https://edusehat.com/en/neural-mechanism-underlying-sensory-behavior-revealed-in-c-elegans https://edusehat.com/en/neural-mechanism-underlying-sensory-behavior-revealed-in-c-elegans Mon, 13 Apr 2026 23:30:09 +0700 Edusehat Neural, Mechanism, Underlying, Sensory, Behavior, Revealed, elegans Animal behavior reflects a complex interplay between an animal’s brain and its sensory surroundings. In a new study published in Nature Neuroscience titled, “Neural sequences underlying directed turning in Caenorhabditis elegans,” researchers from Massachusetts Institute of Technology (MIT) have shown how neuron circuits within C. elegans nematode worms respond to odors and generate movement as they pursue favorable versus unfavorable smells. The results inform understanding of the basic principles of the sensory nervous system for therapeutic applications. 

    “Across the animal kingdom, there are just so many remarkable behaviors,” said Steven Flavell, PhD, associate professor at the Picower Institute at MIT, Howard Hughes Medical Institute (HHMI) investigator, and corresponding author of the study. “With modern neuroscience tools, we are finally gaining the ability to map their mechanistic underpinnings.” 

    Whether moving toward a food source or away from a predator, animals must integrate sensory stimuli to navigate to favorable locations. The neural circuits for navigation are tasked with generating directed movement while simultaneously integrating sensory input to update behavior. Understanding how neural circuits select, execute and adapt sensory-guided navigation behaviors uncovers basic principles of how nervous systems are organized to integrate sensory information and control behavior. 

    In C. elegans, the authors identified error-correcting turns during navigation and used whole-brain calcium imaging and cell-specific perturbations to determine their neural underpinnings. Defined neurons activated in a stereotyped order during each turn. Distinct neurons in this sequence respond to the spatial distribution of attractive and aversive olfactory cues, anticipate upcoming turn directions and drive movement, linking key features of this sensorimotor behavior across time. 

    “One thing that really excited us about this study is that we were able to see what a sensorimotor arc looks like at the scale of a whole nervous system: all the bits and pieces, from responses to the sensory cue until the behavioral response is implemented,” Flavell said.  

    The electrical activity of more than 100 neurons was tracked during sensory movement. Notably, C. elegans only have 302 neurons total. Instead of random movements, the worms executed turns with advantageous timing and at well-chosen angles.  

    The activity of SAA neurons was crucial for integrating odor detection with planned movement and predicted the direction of upcoming turns. Several neurons showed different activity patterns depending on the location of odors were and whether the worm was moving forward or in reverse. 

    Additionally, the neuromodulator, tyramine, was essential for turning and shifting gears. When the worms moved in reverse, tyramine from the neuron RIM enabled other neurons in the sequence to change their activity appropriately to execute the turns. In several experiments, the scientists knocked out RIM tyramine, which disrupted the navigation behaviors and the sequence of neural activity. 

    The post Neural Mechanism Underlying Sensory Behavior Revealed in C. elegans appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO Coffee Chat explores venture philanthropy https://edusehat.com/en/bio-coffee-chat-explores-venture-philanthropy https://edusehat.com/en/bio-coffee-chat-explores-venture-philanthropy Mon, 13 Apr 2026 20:00:05 +0700 Edusehat BIO, Coffee, Chat, explores, venture, philanthropy Patient advocacy is a driving force behind some of today’s most promising breakthroughs in biotechnology—where progress is increasingly powered by strong, productive collaboration between biopharma companies and patient advocacy leaders. Together, they are advancing science— and reshaping how innovation happens.

    “Patient advocates play a fundamental role in biotech innovation—elevating the patient voice to help shape better research, smarter trial designs, and more meaningful patient outcomes,” said Karin Hoelzer, Senior Director of Patient Advocacy at BIO. “In many cases, patient advocates are helping the entire innovation ecosystem work most effectively —from drug discovery through market access and beyond.”

    This evolving role of patient advocacy, and the success of new partnerships such as venture philanthropy, was the focus of a recent Biotechnology Innovation Organization (BIO) Patient Advocacy Coffee Chat. The discussion highlighted that such collaboration is not just aspirational: it is happening, and it is strengthening the entire innovation ecosystem.

    Patient Advocates Leveraging Venture Capital strategies

    One exciting development in the biotech space is the rise of venture philanthropy. Patient advocacy organizations are increasingly stepping forward as investors, partners, and strategic guides alongside biopharma companies to help derisk early-stage assets. By deploying patient-driven capital, coupled with deep disease expertise and patient-generated data, these organizations are helping potentially transformative therapies successfully cross the Valley of Death.

    Venture Funds like the National Bleeding Disorders Foundation Venture Fund, Pathway to Cures, demonstrate the power of this approach. Venture philanthropy supplies the risk‑tolerant capital needed to advance early, preclinical science, ensuring potentially transformative innovations are not abandoned before they can be sufficiently derisked with the evidence required to attract more traditional investment.

    Advocacy-led venture funds are identifying promising early-stage science, investing in emerging biotech companies, and working hand-in-hand with start-up companies to ensure development aligns with the greatest patient needs. These partnerships bring together complementary strengths.

    “What we’ve learned is that a lot of biotechs, and even in some cases the Big Pharma, are not looking enough at what we call Lived Experience Experts (LEEs),” explained Tim Brent, MBA, Venture Principal at Pathway to Cures. “LEEs can be patients, but they are also parents and guardians of these affected individuals.”

    As Brent explained it, oftentimes the input of LEEs are not being taken into consideration early enough in the R&D or clinical trial process, sometimes not at all.

    “A company may have a great discovery and want to provide assistance with the disease, which is great, but if they don’t interact with LEEs, their clinical trial process can be very challenging,” he said. “The earlier the biotechs incorporate the patient perspectives, the more effectively the therapies can meet actual needs, and at the early stages, input from these folks can also shape the study design and product development.”

    Venture Philanthropy as a model is proving to be both effective and scalable. Venture philanthropy is helping to de-risk drug development by aligning scientific innovation with lived experience from the outset. Companies benefit from better trial design, more relevant endpoints, and stronger patient engagement, while patients benefit from therapies that are more likely to succeed and deliver meaningful impact.

    Key to this progress is data—often generated through close partnerships with patient advocacy organizations.

    Data – An Investment Opportunity for Good

    Will Greene, Board Member at the Foundation for Prader-Willi Research and lead author of a new World Economic Forum white paper, Making Rare Diseases Count: How Better Data Can Unlock a Multitrillion-Dollar Opportunity, emphasized the key role of data.

    “Our policy recommendations ultimately center on data capture and analysis,” he explained. While the Forum also examined priorities such as increased public R&D funding, improved reimbursement pathways for innovative medicines, and stronger advocacy networks, high-quality data emerged as a common foundation across all of them.

    “We realized that nearly every strategy for investment depends on having robust, reliable data. Without it, you can’t accurately assess needs, set priorities, or measure the impact of interventions,” explained Greene. “The paper calls for greater investment in rare disease broadly, but with data as a foundational pillar. The message to government and industry leaders is straightforward: if you’re deciding where to invest, start with high-quality data infrastructure.”

    The paper showed that, among other findings, an increase in rare disease investments would:

    • Lower out-of-pocket costs for patients and families,
    • Significantly save costs for healthcare systems,
    • Lower employer insurance premiums,
    • Lessen the strain on social system,
    • Open up a variety of new market opportunities, and
    • Reduce R&D risk, among many, many more positive outcomes.

    As the white paper demonstrates, patient-centered approaches are a powerful engine for better health outcomes, stronger economies, and faster scientific progress.

    “The benefits extend far beyond the patients, families and communities most directly affected; they also accrue to healthcare systems, employers, payers, governments, industry and investors,” concluded Greene.

     

    But what is “high quality data” in this context, and where is it coming from? The answer is disarmingly simple: in many cases, it comes from patients.

    The Power of Patient Generate Data

    At the Friedreich’s Ataxia Research Alliance (FARA), patient advocacy leaders worked closely with researchers and industry partners to help advance the first approved therapy for the condition. FARA played a key role in the first approval of a drug for Friedreich’s Ataxia by collecting the natural history study data from patients for years, while also helping to fund early-stage research and nurturing a diverse drug development pipeline.

    “One of the drug approaches in our pipeline did lead to significant findings in preclinical models and labs,” said Myriam Rai, PhD, Director of Global Relations & Initiatives at FARA. “Thanks to a mother on the FARA board, who acted as a citizen scientist, we were connected to a small company in Texas who had similar molecules. It took a little while to convince them to launch the first clinical trial.”

    The trial met the primary endpoint but the company struggles were far from over. Close collaboration between industry and patient advocates —anchored in patient-generated data—again came to the forefront.

    “Unfortunately, the FDA raised concerns about the results at first,” Rai explained. “But the FA community believed the trial results were persuasive.” After extensive discussions, FDA eventually agreed that the natural history data FARA had meticulously collected through the years could be used, leading to the drug’s approval on Rare Disease Day February 28, three years ago.

    Greene, who is the father of a son with Prader-Willi Syndrome, shared a similar experience from his community. “Over roughly a decade, our community built a registry with more than 2,000 participants and 60,000 completed surveys,” Greene explained. “That dataset delivered tremendous value for our community.”

    The registry data, he explained, not only helped parents like him better understand the natural history of the condition; it also informed clinical guidelines and clarified which interventions are most effective.

    “Crucially, it reached a level of rigor that regulators could rely on to evaluate therapies,” he added. “Data was one of the highest-leverage investments we made.”

    Similarly, on March 30th, 2026, FDA’s Rare Disease Innovation Hub (RDIH) hosted its third RISE workshop on ‘Data Sharing across the Rare Disease Ecosystem,’ bringing together multi‑sector stakeholders to identify strategies, infrastructure needs, and collaborative models to make data sharing a consistent and reliable component of rare disease drug development. The themes from this workshop also emphasized the power of patient data, illustrating how patient‑contributed information can transform research, regulatory decision‑making, and therapeutic progress through close partnerships.

    The Power of Data to Inform Good Policy

    Patient-generated data is not only accelerating research—it is also exposing critical gaps in the healthcare system and opening opportunities for data-informed policy change.

    “We just did a survey of 200 plus patients with chronic and rare diseases to better understand their experiences,” Sarah Jones, MPA, MS, Community Engagement at the Eosinophilic & Rare Disease Cooperative explained. “We were concerned about 340B abuses and what was happening with the 340B program at Disproportionate Share Hospitals (DSH). Their hospital systems are charging patients astronomical costs for their medications and in-hospital treatments, all while knowing that they’re getting these at deeply discounted rates without passing any of the savings on to their patients. And similarly, we’ve been absolutely concerned as we looked downstream and see what happened with these foreign price controls, and taking into consideration what happened the last time they were proposed. People with rare disease rely on a large number of medications, not just rare disease treatments, to manage their complex issues.” The data ERDC collected provide unique insights into the impact of these policies on chronic disease patients and how they experience access to the care they need.

    Patrick Wildman, Senior Vice President, Advocacy & Government Relations, Lupus Foundation of America echoed this sentiment, noting “the MAPRx coalition has produced a couple of reports looking at how access barriers are undermining the affordability provisions in the Inflation Reduction Act (IRA) with the out of pocket cap, because we are seeing significant barriers that have been erected as a result of the IRA, whether it’s increasing cost shifting from co-pays to coinsurance, limited plan choice, a lot of things that we’re seeing challenges on the access side.” Having the data to truly understand the impact on patients and to guide policy solutions is key.

    From early discovery to market access and policy, one message is clear: patient advocacy – including advocacy-led funds and data collection efforts – represents a powerful driving force that helps turn the hope of biotech innovation into treatments and cures for patients.

     

    The post BIO Coffee Chat explores venture philanthropy appeared first on Bio.News.

    ]]>     StockWatch: IPO Market Shows Sign of Life with Avalyn Filing https://edusehat.com/en/stockwatch-ipo-market-shows-sign-of-life-with-avalyn-filing https://edusehat.com/en/stockwatch-ipo-market-shows-sign-of-life-with-avalyn-filing Mon, 13 Apr 2026 05:35:08 +0700 Edusehat StockWatch:, IPO, Market, Shows, Sign, Life, with, Avalyn, Filing The initial public offering (IPO) market showed signs of life for the first time in more than a month as Boston-based Avalyn Pharma filed a registration statement on Wednesday seeking to raise capital to develop its pipeline of respiratory disease treatments.

    It’s too early to know how much money Avalyn plans to raise—the registration statement includes a placeholder “$100 million” figure that will inevitably be revised, and doesn’t say how many shares will be sold. It’s also too soon to know how much of the proceeds will go toward each of the three pipeline candidates cited in the filing to the U.S. Securities and Exchange Commission:

    • AP01—An inhaled version of pirfenidone, a small molecule modulator of cytokines and growth factors whose development the IPO would advance through Phase IIb topline data and into Phase III. AP01 is under study in the Phase IIb MIST trial (NCT06329401) as a potential treatment for progressive pulmonary fibrosis.
    • AP02—An inhaled version of nintedanib, a small molecule inhibitor of multiple tyrosine kinases, being developed to treat idiopathic pulmonary fibrosis (IPF). Avalyn plans to advance AP02 into the Phase II AURA-IPF trial (NCT07194382) after completing single-ascending dose (SAD) and multiple-ascending dose (MAD) Phase I trials in healthy adult volunteers and IPF patients.
    • AP03—A preclinical inhaled fixed-dose combination of AP01 and AP02 designed to combine what Avalyn says is their ability to substantially reduce or eliminate the adverse effects of oral pirfenidone and oral nintedanib.

    Pirfenidone is an IPF drug marketed as Esbriet® by Genentech, a member of the Roche Group, with several other companies selling generic versions. Nintedanib is a kinase inhibitor with indications in treating IPF and chronic fibrosing interstitial lung diseases (ILDs) and slowing the rate of decline in pulmonary function in adults, marketed as Ofev® by Boehringer Ingelheim, with generic versions approved this month.

    “The change we aim to make in the treatment paradigm of pulmonary fibrosis and other ILDs parallels the decades-long evolution seen in the treatment of asthma and COPD,” Avalyn stated in its S-1 statement.

    In those diseases, the company explained, treatments advanced from broad, systemic oral therapies to targeted inhaled treatments, and ultimately to combination inhalers.

    Pulmonary fibrosis “opportunity”

    “We see a similar opportunity in pulmonary fibrosis, where the field still relies on oral antifibrotics today. Our programs are designed to drive a similar evolution, first by shifting treatment toward inhaled, lung-targeted formulations of existing antifibrotics that aim to improve safety and efficacy,” Avalyn explained. “We aspire to deliver inhaled therapies that combine complementary mechanisms into a single device for even greater therapeutic impact.”

    In discussing the use of its proceeds, Avalyn said it envisioned advancing AP01 and AP02 through Phase IIb and Phase II topline data, respectively, into Phase III trials. AP03 would be advanced into the clinic and Phase I topline data using capital from the IPO.

    Whatever isn’t spent on the pipeline candidates will be set aside for R&D activities for additional programs, working capital, and general corporate purposes, Avalyn added.

    Avalyn is the first biotech IPO filing since Generate: Biomedicines completed the year’s largest to date, raising $400 million in gross proceeds toward clinical trials, as well as platform and pipeline R&D efforts. To date, seven companies have completed biotech IPOs, raising just over $1.7 billion in combined proceeds, Jefferies analyst Andrew Tsai wrote in a research note.

    “The IPO market has been more of a laggard but showed signs of strength this quarter, with Q1 offerings the largest in the past four years,” Tsai wrote. As a result, he added, the IPO market is on pace to exceed historical levels except for the 2020–2021 IPO boom due to the COVID-19 pandemic.

    Mixed on IPO improvement

    Heading into 2026, analysts were mixed on whether this year would see improvement in the IPO market compared to 2025, when 11 U.S. companies raised a total of $3 billion on Wall Street. “We think it will be slightly better, but we have not seen enough to suggest that it’s truly rebounding,” Subin Baral, EY global life sciences deals leader, told GEN.

    However, Michael Allwin, head of biopharma investment banking, Truist Securities, told GEN that IPOs are typically “the last shoe to drop” after other non-IPO financings show signs of recovery, giving him hope and optimism that 2026 would see a much more active IPO market than 2025: “While we’re not anticipating a resurgence in activity to the tune of what we saw at all-time highs in 2020 and 2021, we are anticipating a more normalized level of activity, maybe on parity with 2019.”

    As for Avalyn, should its planned IPO raise the placeholder $100 million amount, it would nearly double the $138.359 million in cash, cash equivalents, and marketable securities with which Avalyn finished 2025.

    Avalyn ended last year with no revenue and a net loss of $85.204 million, a 71% increase over the $49.744 million net loss the company reported for 2024. As a result, Avalyn’s accumulated deficit rose from $180.2 million at the end of 2024 to $265.4 million on December 31, 2025.

    The IPO comes nine months after Avalyn completed its last financing, an oversubscribed $100 million Series D round completed in July and led by investment firms Suvretta Capital Management and SR One.

    Survetta and SR One are two of 18 firms that have invested in Avalyn. The 18 include Novo Holdings, the asset manager of the foundation that controls Novo Nordisk.

    Novavax rises on shareholders’ opposition

    Novavax (NASDAQ: NVAX) enjoyed a small but noticeable surge in its stock price this past week after its second-largest shareholder ramped up its opposition to the vaccine developer’s leadership on several fronts.

    Shah Capital Opportunity Fund, which holds an approximately 9% stake in Novavax, said it will oppose the company’s nominees for re-election to the board of directors when Novavax holds its annual shareholder meeting, scheduled for June (no date had been announced at deadline).

    In an open letter to Novavax’s board, Raleigh, NC-based Shah Capital also requested that Novavax:

    • Shrink the board from eight to five members and elect new members “with emphasis on pragmatic entrepreneurial experience to turn Novavax into an equity success story.”
    • Buy back 10 to 20 million shares.
    • Retire its outstanding $225 million convertible bond with cash on hand “at the earliest.” Novavax reported $244.213 million in convertible notes payable and $240.634 million in cash and cash equivalents as of December 31, 2025.
    • Persuade a strategic long-term investor to take a 10–20% ownership stake “to reshape Novavax entirely.”

    Shah Capital cited a 27% drop in Novavax’s share price from $11 on January 1, 2023, when John C. Jacobs took over as president and CEO, to $8 on March 31, 2026. The fund also expressed frustration that the COVID-19/influenza combination vaccine Novavax is developing with Sanofi (Euronext Paris: SAN)—a potential $5+ billion category, according to Shah Capital—hasn’t yet begun Phase III trials. Sanofi shared positive Phase I/II data in December and told Novavax it is working with regulators on next steps.

    “Management has failed to implement aggressive cost-cutting measures necessary to achieve consistent profitability,” Himanshu H. Shah, the fund’s managing partner and chief investment officer, advocated in an open letter to Novavax’s board. “The current senior management team should be reduced by 30% to reflect Novavax’s new royalty and partnership business model.”

    “The board size should also be reduced to five from eight, including electing new members with emphasis on pragmatic entrepreneurial experience to turn Novavax into an equity success story,” Shah added.

    At odds for months

    Shah has been at odds with Jacobs and Novavax leadership for months, having called for a sale of the company last October. Shah has held off pursuing a proxy campaign since the board’s majority has favored current management.

    Novavax is based in Gaithersburg, MD, and reported approximately 749 employees as of December 31, 2025, down 21% from 952 a year earlier, according to Form 10-K annual reports.

    Novavax investors responded to the Shah Capital letter with a buying spurt that sent shares climbing 5.5% Wednesday, from $7.98 to $8.42 after rising to $8.60 during intraday trading. The momentum continued somewhat on Thursday as shares rose another 1.4%, to $8.54, though Novavax slumped 5% Friday to finish the week at $8.12.

    Shah Capital’s letter also sparked a statement to GEN and other news outlets from Novavax, which asserted that its board and management team “are committed to progressing our growth strategy, which is designed to leverage partnerships and R&D innovation to maximize the value of our technology.”

    The statement cited recent Novavax efforts that include its up-to-$530 million (plus royalties) partnership with Pfizer (NYSE: PFE), which entered into a non-exclusive license agreement with Pfizer for use of Novavax’s Matrix-M® adjuvant; additional and expanded material transfer agreements with pharmaceuticals; and what the company called “continued progress” on its partnership with Sanofi, from which Novavax generated $225 million in milestone payments last year.

    “In addition, we continue to make targeted investments in R&D with the intention of driving further value from our technology, while continuing to significantly reduce costs in our lean and efficient operating model,” Novavax continued. “We maintain constructive dialogue with our shareholders, and we welcome collaborative input that is in the best interest of Novavax and all of its shareholders.”

    Shah essentially controls 14,845,097 shares of Novavax stock, including 125,359 shares he owns personally, and 14,719,738 shares owned by Shah Capital and its investment adviser.

    Leaders and laggards

    • Invivyd (NASDAQ: IVVD) shares jumped 32% from $1.35 to $1.78 Thursday after the company announced positive progress in its REVOLUTION clinical program for VYD2311, a monoclonal antibody candidate designed to prevent symptomatic COVID-19. As of April 6, when the first 1,500 of 1,818 subjects reached Day 45, clinical events supported statistical powering for the high end of anticipated VYD2311 efficacy levels in the Phase III DECLARATION trial (NCT07298434), with about half of the base study still to be carried out, Invivyd said. DECLARATION will enroll ~500 additional subjects, which, according to the company, will likely, depending on recruitment rates, push back the timing for data release by approximately two months, from mid-year to Q3 2026. Invivyd also announced the discovery and advancement of a “highly potent,” half-life-extended, high-resistance-barrier measles monoclonal antibody candidate, VMS063.
    • Replimune Group (NASDAQ: REPL) shares tumbled 19.5% from $5.91 to $4.76 Friday after the developer of oncolytic immunotherapies disclosed that the FDA for a second time had rejected the company’s biologics license application (BLA) for its lead product candidate RP1 (vusolimogene oderparepvec) in combination with nivolumab to treat advanced melanoma, instead issuing a complete response letter (CRL). Replimune criticized the FDA for an inconsistent review process, saying the agency contradicted earlier guidance to the company and assessed the resubmitted BLA through a different review team that replaced the team that previously interacted with the company. Replimune also defended the combination therapy’s data in the Phase II IGNYTE trial (NCT03767348)—a 34% response rate with a median duration of 24.8 months and a favorable safety profile, the basis of the combo’s breakthrough therapy designation. “We have no choice but to eliminate jobs, including substantially scaling back our U.S.-based manufacturing operations,” stated Replimune CEO Sushil Patel, PhD. Nivolumab is the cancer immunotherapy marketed as Opdivo® by Bristol Myers Squibb (NYSE: BMY).

    The post StockWatch: IPO Market Shows Sign of Life with Avalyn Filing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     What’s in a name? Moderna’s “vaccine” vs. “therapy” dilemma https://edusehat.com/en/whats-in-a-name-modernas-vaccine-vs-therapy-dilemma https://edusehat.com/en/whats-in-a-name-modernas-vaccine-vs-therapy-dilemma Sat, 11 Apr 2026 03:30:03 +0700 Edusehat What’s, name, Moderna’s, “vaccine”, vs., “therapy”, dilemma Is it the Department of Defense or the Department of War? The Gulf of Mexico or the Gulf of America? A vaccine—or an “individualized neoantigen treatment”?

    That’s the Trump-era vocabulary paradox facing Moderna, the covid-19 shot maker whose plans for next-generation mRNA vaccines against flus and emerging pathogens have been dashed by vaccine skeptics in the federal government. Canceled contracts and unfriendly regulators have pushed the Massachusetts-based biotech firm to a breaking point. Last year, Robert F. Kennedy Jr., head of the Department of Health and Human Services, zeroed in on mRNA, unwinding support for dozens of projects—including a $776 million award to Moderna for a bird flu vaccine. By January, the company was warning it might have to stop late-stage programs to develop vaccines against infections altogether.

    That raises the stakes for a second area of Moderna’s research. In a partnership with Merck, it’s been using its mRNA technology to destroy tumors through a very, very promising technique known as a cancer vacc—

    “It’s not a vaccine,” a spokesperson for Merck jumped in before the V-word could leave my mouth. “It’s an individualized neoantigen therapy.”

    Oh, but it is a vaccine. And here’s how it works. Moderna sequences a patient’s cancer cells to find the ugliest, most peculiar molecules on their surface. Then it packages the genetic code for those same molecules, called neoantigens, into a shot. The patient’s immune system has its orders: Kill any cells with those yucky surface markers.

    Mechanistically, it’s similar to the covid-19 vaccines. What’s different, of course, is that the patient is being immunized against a cancer, not a virus.

    And it looks like a possible breakthrough. This year, Moderna and Merck showed that such shots halved the chance that patients with the deadliest form of skin cancer would die from a recurrence after surgery.

    In its formal communications, like regulatory filings, Moderna hasn’t called the shot a cancer vaccine since 2023. That’s when it partnered up with Merck and rebranded the tech as individualized neoantigen therapy, or INT. Moderna’s CEO said at the time that the renaming was to “better describe the goal of the program.” (BioNTech, the European vaccine maker that’s also working in cancer, has shifted its language too, moving from “neoantigen vaccine” in 2021 to “mRNA cancer immunotherapies” in its latest report.)

    The logic of casting it as a therapy is that patients already have cancer—so it’s a treatment as opposed to a preventive measure. But it’s no secret what the other goal is: to distance important innovation from vaccine fearmongering, which has been inflamed by high-ranking US officials. “Vaccines are maybe a dirty word nowadays, but we still believe in the science and harnessing our immune system to not only fight infections, but hopefully to also fight … cancers,” Kyle Holen, head of Moderna’s cancer program, said last summer during BIO 2025, a big biotech event in Boston.

    Not everyone is happy with the word games. Take Ryan Sullivan, a physician at Massachusetts General Hospital who has enrolled patients in Moderna’s trials. He says the change raises questions over whether trial volunteers are being properly informed. “There is some concern that there will be patients who decline to treat their cancer because it is a vaccine,” Sullivan told me. “But I also felt it was important, as many of my colleagues did, that you have to call it what it is.”

    But is it worth going to the mat for a word? Lillian Siu, a medical oncologist at the Princess Margaret Cancer Centre, in Toronto, who has played a role in safety testing for the new shots, watches US politics from a distance. She believes name change is acceptable “if it allows the research to continue.”

    Holen told me the doctors complaining to Moderna were basically motivated by a desire to defend vaccines—which are, of course, among the greatest public health interventions of all time. They wanted the company to stand strong. 

    But that’s not what’s happening. When Moderna’s latest results were published in February, the paper’s main text didn’t use the word “vaccine” at all. It was only in the footnotes that you could see the term—in the titles of old papers and patents.

    All this could be a sign that Kennedy’s strategy is working. His agencies often appear to make mRNA vaccines a focus of people’s worries, impede their reach, devalue them for companies, and sideline their defenders. 

    Still, Moderna’s strategy may be working too. So far, at least, the government hasn’t had much to say about the company’s cancer vacc— I mean, its individualized neoantigen therapy.

    This article first appeared in The Checkup, MIT Technology Review’s weekly biotech newsletter. To receive it in your inbox every Thursday, and read articles like this first, sign up here.

    ]]>     Drugs from a Text Prompt, Wegovy Pill Competition Dampens Lilly’s Surge https://edusehat.com/en/drugs-from-a-text-prompt-wegovy-pill-competition-dampens-lillys-surge https://edusehat.com/en/drugs-from-a-text-prompt-wegovy-pill-competition-dampens-lillys-surge Sat, 11 Apr 2026 03:25:13 +0700 Edusehat Drugs, from, Text, Prompt, Wegovy, Pill, Competition, Dampens, Lilly’s, Surge From designing drugs with a simple text prompt to running experiments guided by extended reality, a new wave of agentic AI is transforming the modern lab. Our editors discuss the latest autonomous systems accelerating biological discovery. In business deals, Gilead Sciences has acquired Tubulis in a transaction worth up to $5 billion, strengthening the buyer’s position in antibody–drug conjugates for cancer. Correspondingly, Eli Lilly and Biogen are each making billion-dollar-plus bets, acquiring Centessa, a sleep disorder drug developer, and Apellis, known for its work in immunology and rare diseases. Our episode rounds out by unpacking the dynamic obesity drug market, where intensifying competition from Novo Nordisk’s Wegovy pill is prompting Lilly to temper the 2026 sales outlook for its oral obesity drug, Foundayo.

     

     

    Listed below are links to the GEN stories referenced in this episode of Touching Base:

    Can AI Agents Automate Scientific Discovery?
    By Fay Lin, PhD, GEN Edge, April 1, 2026

    Gilead to Acquire Tubulis for Up to $5B, Expanding Cancer ADC Capabilities
    By Alex Philippidis, GEN Edge, April 7, 2026

    Lilly Acquires Centessa for Up to $7.8B; Biogen Buys Apellis for Up to $6.1B
    By Alex Philippidis, GEN Edge, March 31, 2026

    StockWatch: Price War Dampens Lilly Surge After Oral GLP-1 Wins FDA Nod
    By Alex Philippidis, GEN Edge, April 5, 2026

    Touching Base Podcast
    Hosted by Corinna Singleman, PhD

    Behind the Breakthroughs

    Hosted by Jonathan D. Grinstein, PhD

    The post Drugs from a Text Prompt, Wegovy Pill Competition Dampens Lilly’s Surge appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cryo-EM Structural Biology Facility Opened in San Diego by FairJourney Bio https://edusehat.com/en/cryo-em-structural-biology-facility-opened-in-san-diego-by-fairjourney-bio https://edusehat.com/en/cryo-em-structural-biology-facility-opened-in-san-diego-by-fairjourney-bio Sat, 11 Apr 2026 03:25:12 +0700 Edusehat Cryo-EM, Structural, Biology, Facility, Opened, San, Diego, FairJourney, Bio FairJourney Bio (FJBio), a CRO, opened its advanced cryo-electron microscopy (cryo-EM) structural biology facility in San Diego. The new site significantly expands the company’s presence in the U.S. market and incorporates atomic-resolution structural biology directly into its antibody discovery platform, according to Christopher Arthur, PhD, CSO, structural biology, FairJourney Bio.

    The facility houses a 300 kV cryo-EM infrastructure, including two ThermoFisher Titan Krios 5 systems, which enables native-state structure determination of antibody-target complexes. The technology is designed to provide detailed insights across the R&D value chain—from epitope mapping and hit generation to structure-guided lead optimization and candidate selection.

    FairJourney Bio’s San Diego facility opening event. [FairJourney Bio]
    FairJourney Bio’s San Diego facility opening event. [FairJourney Bio]
    The cryo-EM services, launched in January 2026, complement FJBio’s antibody discovery and biologic development portfolio. The services enable scientists to visualize protein structures at atomic resolution, including protein-protein and protein-ligand complexes, providing high-quality and interpretable results to inform confident decision-making across programs.

    The facility is strategically positioned within a leading global biotech hub, complementing FJBio’s existing U.S. presence in San Francisco and its operations across Europe.

    “Structural biology has historically been a late-stage tool, used to confirm decisions already made,” notes Arthur. “We are redefining that paradigm. In San Diego, we are building a premier, full-service cryo-EM CRO that brings together decades of deep expertise in sample preparation, data collection, and computational analysis, embedding structural insight at the very start of discovery, where it shapes epitope selection and determines which leads are worth advancing.”

    The post Cryo-EM Structural Biology Facility Opened in San Diego by FairJourney Bio appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     First Detailed Insight into Bornavirus Nucleoprotein–RNA Complex Reveals Unique Assembly https://edusehat.com/en/first-detailed-insight-into-bornavirus-nucleoproteinrna-complex-reveals-unique-assembly https://edusehat.com/en/first-detailed-insight-into-bornavirus-nucleoproteinrna-complex-reveals-unique-assembly Sat, 11 Apr 2026 03:25:10 +0700 Edusehat First, Detailed, Insight, into, Bornavirus, Nucleoprotein–RNA, Complex, Reveals, Unique, Assembly Borna disease virus 1 (BoDV-1) is a neurologic disease of horses and sheep that causes rare human infections. The outcome in those who develop disease almost always results in inflammation in the brain or fatal encephalitis.

    The nucleoprotein–RNA complex in viruses protects the RNA genome and supports viral RNA synthesis. Increasing our understanding of the structure of this complex is essential to targeting viral replication. Structural characterization has been completed for several viruses in the same order as BoDV-1 (Mononegavirus) that more commonly infect humans, but detailed information for the family Bornaviridae has not been sufficiently explored.

    “Bornaviruses are less well known than many other human RNA viruses, yet they represent the last major unresolved case for nucleoprotein–RNA structural analysis among human-infecting mononegaviruses,” says Yukihiko Sugita, PhD, associate professor at Kyoto University. “Closing this long-standing gap and connecting structural biology with virological function were major motivations for our team.”

    Using cryo-electron microscopy, researchers from Kyoto University, Osaka Dental University, and Osaka Metropolitan University obtained high-resolution images of BoDV-1 nucleoprotein–RNA complexes and performed computational classification to separate and reconstruct the distinct assembly states of each complex in the sample. They also used mutational and functional assays to test nucleoprotein–RNA residues and evaluate their roles in viral RNA synthesis and assembly.

    This work is published in Science Advances in the paper, “Structure and assembly of Borna disease virus 1 nucleoprotein-RNA complexes.”

    These findings are the first detailed structural description of the nucleoprotein–RNA complex in the family Bornaviridae and revealed the three-dimensional structure of this nucleoprotein-RNA complex, showing ring-like assemblies and viral RNA binds in the inner groove. The researchers also found that each nucleoprotein subunit accommodates eight RNA nucleotides, suggesting a binding mode distinct from those reported for other related viruses.

    The work also reveals that mutations impairing RNA binding disrupt viral RNA synthesis, but that nucleoprotein assemblies can form even without RNA. Together, these findings suggest an incremental model in which nucleoprotein assembly and RNA engagement are separate but coordinated processes.

    This study provides a molecular framework for a systematic comparison of Bornaviridae nucleoprotein–RNA architecture alongside that of other mononegaviruses, and supports broader questions about the principles governing nucleoprotein–RNA interactions. It also lays the groundwork for future antiviral studies targeting viral replication through nucleoprotein–RNA interactions.

    Next, the team would like to analyze complexes derived from infected cells as well as those with longer RNA segments. They also plan to integrate structural analysis and biochemical approaches in order to observe intermediate complex formation states and compare them with those of related viruses.

    The post First Detailed Insight into Bornavirus Nucleoprotein–RNA Complex Reveals Unique Assembly appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Epigenetics at Birth Links Microbiome to Neurodevelopment, Potentially ASD and ADHD https://edusehat.com/en/epigenetics-at-birth-links-microbiome-to-neurodevelopment-potentially-asd-and-adhd https://edusehat.com/en/epigenetics-at-birth-links-microbiome-to-neurodevelopment-potentially-asd-and-adhd Sat, 11 Apr 2026 03:25:09 +0700 Edusehat Epigenetics, Birth, Links, Microbiome, Neurodevelopment, Potentially, ASD, and, ADHD The results of a study headed by researchers at Li Ka Shing Institute of Health Sciences, The Chinese University of Hong Kong, indicate that the gut microbiome and epigenetics are intertwined, and that both contribute to neurodevelopment.

    The researchers showed that epigenetic changes present at birth can impact how an infant’s gut microbiome develops during their first year. They also identified specific epigenetic changes and gut microbes that were associated with signs of autism spectrum disorder (ASD) and attention-deficit/hyperactivity disorder (ADHD) when the children were three years old.

    “Certain bacteria seem to offer protection, which is exciting because it suggests there could be ways to support a child’s development through diet or probiotics in the future,” said research lead and gastroenterologist Francis Ka Leung Chan, MD. Chan is co-senior author of the team’s published paper in Cell Press Blue, titled “Epigenome-microbiome interplay in early life associates with infants’ neurodevelopmental outcomes,” in which they stated, “We showed that epigenetic alterations at birth were associated with early-life microbiome development and that they determine the risks of neurodevelopmental consequences in children.”

    The first years of life are critical for brain development and immune system maturation. Though previous studies have shown that both early epigenetic changes and gut microbiome development can impact health in later life, little is known about how these two systems interact. “Recent data suggest that epigenetic programming of gene expression profiles is sensitive to the early-life environment and can impact health outcomes in children,” the authors wrote. “One environmental cue known to trigger host epigenetic modifications is the genes of bacteria, fungi, and viruses inside the human body, collectively known as the microbiome.”

    Co-senior author and public health researcher Hein Min Tun, PhD, of The Chinese University of Hong Kong, commented, “We wanted to see how the epigenome and microbiome interact in early life and if their interaction could influence a child’s risk of developing neurodevelopmental conditions like ASD and ADHD.” The authors added, “New understanding of host-microbe-epigenome interactions and mechanisms of epigenetic changes in early life can be leveraged for the prevention, early detection, and novel interventions of common childhood diseases.”

    For their study the researchers characterized DNA methylation patterns from the umbilical cord blood of 571 infants. They paired this information with gut microbiome data collected from 969 infants at two, six, and 12 months of age, and from their parents during the third trimester of pregnancy. When the children reached 36 months of age, the researchers used a behavioral questionnaire to assess their neurodevelopment and investigate links between the microbiome, epigenome, and early signs of ASD and ADHD.

    “This, to our knowledge, represents the first longitudinal study with multiple sample types to depict the intimate interplay between perinatal exposures, epigenetic hallmarks, and gut microbiome development and neurodevelopmental outcomes within the first three years of life,” the authors stated.

    They found that an infant’s epigenome at birth was associated with birth mode, length of gestation, having older siblings, and maternal allergies, but it was not affected by their parents’ gut microbiomes. Microbiome development, on the other hand, was associated with birth mode, antibiotics, having older siblings, and breastfeeding. Infants who were born by Caesarean section (CS) showed different patterns of DNA methylation for several genes involved in immune responses and brain development. “Some of the changes in methylations of immune- and nervous-system-related genes, associated with CS delivery, are linked to neurodevelopmental outcomes,” they noted.

    Their reported findings, the team suggested, “… resonate with studies linking CS to increased risks of immune-mediated and neurodevelopmental disorders, providing mechanistic plausibility through epigenomic and microbial dysbiosis.” The team also showed that an infant’s epigenome at birth impacted how their microbiome developed during their first year. Specifically, infants developed less diverse gut microbiomes at 12 months of age when they showed higher rates of DNA methylation in immune genes involved in recognizing pathogens. “We found that methylation rates in the major histocompatibility complex (MHC) region of infants at birth were linked to differences in the diversity of the infant gut microbiome at 12 months,” they commented.

    The behavioral survey revealed that signs of ASD and ADHD in three-year-olds were associated with specific epigenetic patterns and the presence of certain gut microbes. “Importantly, we reported that epigenetic modifications were associated with an increased susceptibility to neurodevelopmental conditions in children, and these effects were in part mediated by microbial colonization.”

    However, other microbial species seemed to mitigate these effects: infants with epigenetic patterns associated with ASD or ADHD were less likely to show signs of the disorders if they acquired Lachnospira pectinoschiza and Parabacteroides distasonis, respectively, during their first year. “We discovered a kind of conversation happening: a baby’s epigenetic setting at birth can influence their risk for neurodevelopmental disorders, but the presence of certain ‘good’ bacteria in their gut can step in and modify the risk,” Tun reported. “The foundations for brain health are laid very early, even before birth. However, we don’t want people to think this means a child’s developmental path is fixed at birth. These are complex conditions with many causes, and we’ve only uncovered a small piece of a very large puzzle.”

    The researchers are continuing to follow the children who participated in the study to see how these early-life factors relate to their health as they grow. They note that laboratory experiments are needed to confirm the associations between gut microbes and neurodevelopment. In their discussion, the team wrote, “In conclusion, our findings revealed dual alterations to the neonatal epigenome and gut microbiome by perinatal factors and highlight the role of the ‘holo-epigenome’—the integrated host epigenome and microbiome—as a key mediator of neuro-immune outcomes. Interventions targeting microbial restoration or epigenetic modulation during critical developmental windows may mitigate risks of neurodevelopmental disorders.”

    First author and gastroenterologist Siew Chien Ng, MD, PhD, added, “The ultimate goal is to develop safe, non-intrusive early interventions such as specific probiotics or live biotherapeutics, that could help nurture a healthy gut microbiome and potentially reduce the risk of neurodevelopmental challenges.”

    The post Epigenetics at Birth Links Microbiome to Neurodevelopment, Potentially ASD and ADHD appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Cancer Drug Shortfalls Tied to How BET Inhibitors Hit BRD2 and BRD4 Differently https://edusehat.com/en/cancer-drug-shortfalls-tied-to-how-bet-inhibitors-hit-brd2-and-brd4-differently https://edusehat.com/en/cancer-drug-shortfalls-tied-to-how-bet-inhibitors-hit-brd2-and-brd4-differently Fri, 10 Apr 2026 09:30:13 +0700 Edusehat Cancer, Drug, Shortfalls, Tied, How, BET, Inhibitors, Hit, BRD2, and, BRD4, Differently For more than a decade, BET inhibitors have been touted as one of cancer therapy’s most promising drug classes. The logic was straightforward: many tumors rely on oncogenes that depend on BET (bromo- and extra-terminal domain) proteins—chromatin‑binding regulators that help switch genes on. Block the BET family, the thinking went, and cancer cells should lose their transcriptional fuel. In the lab, the strategy often worked. But in clinical trials, the results were far more uneven: modest responses, substantial side effects, and little clarity about which patients might benefit.

    A new study from the Max Planck Institute of Immunobiology and Epigenetics (MPI‑IE) may finally explain why. Published in Nature Genetics, the work uncovers a previously underappreciated division of labor within the BET family—one that helps clarify why drugs that block all BET proteins at once have struggled in the clinic. The paper is titled, “Histone acetylation-dependent clustering of BRD2 instructs transcription dynamics.”

    Most BET inhibitors were designed to block a shared bromodomain that all BET proteins use to bind chromatin. That approach assumed the proteins—BRD2, BRD3, BRD4, and BRDT—perform similar roles. But the new study paints a more nuanced picture. Using rapid protein degradation, chemogenomics, and super‑resolution microscopy in mouse embryonic stem cells, the team dissected the distinct contributions of BRD2 and BRD4 to transcription.

    Super-resolution microscopy images of cell nuclei showing BRD2 (green) and BRD4 (red) inside the nucleus. When transcription elongation is blocked with flavopiridol (right), BRD2 clusters increase markedly while BRD4 distribution shifts—visually demonstrating that the two proteins respond differently to the same perturbation, reflecting their distinct roles in initiating (BRD2) versus driving gene transcription (BRD4). [MPI of Immunobiology & Epigenetics, Asifa Akhtar]
    Their findings reveal that BRD4 drives the well‑known step of releasing paused RNA polymerase II into productive elongation. BRD2, however, acts earlier. It helps recruit and organize the transcription initiation machinery at promoters, particularly under conditions where pause‑release is impaired. As the authors wrote, BRD2’s role becomes “particularly critical under the conditions of impaired pause release,” a mechanistic insight that reframes how BET proteins collaborate during gene activation.

    The MPI‑IE team likens BRD2 to a stage manager. “BRD2 sets up the stage: assembling the props, costumes, and actors to ensure preparations run smoothly. BRD2 then gives BRD4, the actor, the ‘start’ signal to begin with the performance,” said senior author Asifa Akhtar, PhD. Blocking both proteins simultaneously—exactly what current BET inhibitors do—disrupts two different steps of transcription at once, producing unpredictable and context‑dependent effects.

    “Our data shows that the setup work happening before is just as critical for gene activation,” explained Akhtar.

    A key discovery is that BRD2’s recruitment depends on histone H4 acetylation placed by the enzyme MOF. When MOF was rapidly depleted or deleted, BRD2 lost its grip on chromatin, while BRD3 and BRD4 remained largely unaffected. “The findings support a model in which acetylated chromatin creates a platform that allows regulatory proteins like BRD2 to concentrate and prepare the transcription machinery,” noted first author Umut Erdogdu, PhD.

    The team also showed that BRD2 forms dynamic clusters at promoters. Removing only the BRD2 region responsible for clustering stalled transcription almost as completely as deleting the entire protein.

    The study suggests a path forward: instead of blocking all BET proteins indiscriminately, future therapies may need to distinguish between BRD2‑ and BRD4‑specific functions. “Thus, these findings support a model in which histone acetylation-dependent spatiotemporal dynamics of BRD2 coordinate the transcription machinery to regulate transcription initiation,” the authors wrote.

    For a field long puzzled by the uneven performance of BET inhibitors, BRD2’s newly revealed role offers a compelling piece of the puzzle—and a clearer blueprint for next‑generation cancer therapeutics.

    The post Cancer Drug Shortfalls Tied to How BET Inhibitors Hit BRD2 and BRD4 Differently appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Labguru Customer Portal Launched for CRO/CDMO and Client Collaboration https://edusehat.com/en/labguru-customer-portal-launched-for-crocdmo-and-client-collaboration https://edusehat.com/en/labguru-customer-portal-launched-for-crocdmo-and-client-collaboration Fri, 10 Apr 2026 05:55:12 +0700 Edusehat Labguru, Customer, Portal, Launched, for, CROCDMO, and, Client, Collaboration Officials at Cenevo say the company has launched the Labguru Customer Portal to help streamline client communication for CROs, CDMOs, and others.

    CROs and CDMOs operate in high-throughput, service-driven environments where speed, accuracy, and transparency are critical, explains Eran Sandman, product manager, Cenevo. Labguru allows CROs and CDMOs to centralize operational visibility across departments and projects, standardize workflows, and manage multiple client programs at scale, he adds.

    Integrated with Labguru’s ELN and LIMS platforms, the new portal was designed to extend internal workflows to support external collaborators. With this structured approach, labs experience increased efficiency while minimizing version conflicts, human error, and compliance risks, maintains Sandman, noting that clients can submit requests, monitor progress and access results, while labs retain full control and visibility over shared information.

    Lab managers can use the portal to gain actionable insights across every client individually and all clients in aggregate, as they relate to equipment and consumables usage, client activity trends, regulatory compliance, and other critical information, points out Sandman.

    “Our goal is to make our clients’ operations run more smoothly,” he says. “High-performing CROs are moving toward shared digital environments. With the Customer Portal, CROs and their clients are able to see the same information and collaboration becomes faster and more strategic. Lab managers don’t have to spend all their time on back-and-forth communications.”

    The post Labguru Customer Portal Launched for CRO/CDMO and Client Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Seer to Apply Deep Proteomics Tech to Singapore Population Cohort Study https://edusehat.com/en/seer-to-apply-deep-proteomics-tech-to-singapore-population-cohort-study https://edusehat.com/en/seer-to-apply-deep-proteomics-tech-to-singapore-population-cohort-study Fri, 10 Apr 2026 05:55:11 +0700 Edusehat Seer, Apply, Deep, Proteomics, Tech, Singapore, Population, Cohort, Study This week, Seer, a developer of proteomics technology, said that scientists involved in the PRECISE-SG100K initiative will use the company’s Proteograph® product suite to profile the plasma proteome of about 10,000 participants. The data from this workflow will be combined with information from the same participants that was generated using Thermo Fisher Scientific’s Olink® Reveal, a next-generation sequencing-based proteomics solution, and its Orbitrap Astral™ mass spectrometers.

    PRECISE-SG100K is the second phase of a broader initiative in Singapore that aims to support various research studies that advance scientists’ understanding of health and diseases. Touted as a landmark population study of approximately 100,000 Singaporean residents, PRECISE-SG100K is designed to integrate genomic, proteomic, lifestyle, imaging, and other health data from a multi-ancestry Asian population. By combining data from Seer’s Proteograph platform alongside information from other technologies that are being used for the project, the scientists aim to develop what they believe will be one of the most comprehensive multiomic datasets available to date. 

    “PRECISE-SG100K is one of the most ambitious and carefully designed multiomic health initiatives in the world,” said Omid Farokhzad, MD, chair and CEO of Seer. And that fits with Seer’s vision for Proteograph, which was that “deep, unbiased proteomics becomes the mainstay for population-scale multiomic studies.”

    Seer already has an existing relationship with Thermo Fisher dating back several years. In 2024, the companies announced a co-marketing and sales agreement that allowed Thermo to jointly promote Proteograph alongside Orbitrap Astral mass spectrometers to provide customers of the Orbitral Astral with an integrated solution for unbiased proteomic analysis. The combined solutions have since been used in a number of large population studies providing high-throughput, deep proteome coverage. 

    “A key goal of PRECISE-SG100K is to create a deeply characterized, multi-ancestry resource that can reveal how genetics, environment, and lifestyle shape disease risk and treatment response,” said John Chambers, PhD, chief scientific officer of PRECISE and lead principal investigator of the PRECISE-SG100K study. “By adding deep, unbiased plasma proteomics enabled by Seer and Thermo Fisher, we can more directly link genomic variation to protein networks and health outcomes, uncovering insights critical to ensuring precision medicine reflects the diversity of Asian populations.”

    Ultimately, the data generated from population projects like this are expected to support biomarker discovery in key disease areas including cardiometabolic, ophthalmic, and neurologic disorders. They could also inform the development of predictive models for assessing disease risk and response as well as efforts to validate and prioritize biomarkers identified through affinity-based platforms. 

    The post Seer to Apply Deep Proteomics Tech to Singapore Population Cohort Study appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     DNA Uptake in Cholera May Increase Defense Mechanisms https://edusehat.com/en/dna-uptake-in-cholera-may-increase-defense-mechanisms https://edusehat.com/en/dna-uptake-in-cholera-may-increase-defense-mechanisms Fri, 10 Apr 2026 05:55:10 +0700 Edusehat DNA, Uptake, Cholera, May, Increase, Defense, Mechanisms Cholera, caused by the bacterium Vibrio cholerae, remains a major global health threat. Like most bacteria, Vibrio cholerae lives under constant attack from viruses. To survive, bacteria equip themselves with antiviral immune systems. Previous work has shown that V. cholerae carries a large genetic element called a sedentary chromosomal integron (SCI). This structure contains hundreds of small mobile DNA units known as “gene cassettes” arranged in a long array, like a chain of pearls. A new study by researchers at the School of Life Sciences, Ecole Polytechnique Fédérale de Lausanne (EPFL), has now shown that V. cholerae can efficiently acquire new SCI gene cassettes from extracellular DNA released by other V. cholerae strains, and non-cholera vibrio species, potentially adding to their own defense arsenals.

    Research lead Melanie Blokesch, PhD, and colleagues reported on their findings in Science, in a paper titled “Competence-mediated DNA uptake diversifies Vibrio cholerae sedentary chromosomal integrons,” in which they concluded “Given the widespread presence of SCIs and the conservation of natural competence across the genus, we propose that SCIs function as genus-wide reservoirs of exchangeable protective genes.”

    Bacteria often survive viral attack and environmental stress by sharing genes that enhance their defenses, the authors wrote. A “defining genomic feature” of V. cholerae is its sedentary chromosomal integron (SCI), a genetic element containing hundreds of mostly promoterless gene cassettes. While the function of many cassettes remains unknown, some do encode antiviral immune systems. “Although most cassettes encode proteins of unknown function, ~10% encode phage defense systems, suggesting that SCIs as well as mobile integrons function as reservoirs, or “biobanks,” of defense genes,” the authors continued.

    However, most of these genes are located far from the start of the array and remain silent. Prevailing models proposed that cassettes could be internally reshuffled to activate them, yet no such rearrangements have been observed in the pandemic lineage of V. cholerae for decades. “Cassettes are thought to reshuffle under stress to the favorable first array position, yet the SCI in pandemic V. cholerae has remained static for more than 60 years.”

    This raises a key question: if internal reshuffling is rare, how are cassette-encoded immune systems activated, and how do new cassettes enter the array at all? To address this question, a team led by Blokesch at the Laboratory of Molecular Microbiology at EPFL investigated whether the SCI might capture gene cassettes from genetic material entering the cell from the outside. “We asked whether SCI cassettes move horizontally rather than by intracellular reshuffling.”

    A key feature of this process is natural competence, the ability of bacteria to take up free DNA from their surroundings. V. cholerae becomes naturally competent when it grows on chitinous surfaces, a polymer found in the shells of crustaceans that is abundant in aquatic environments.

    In the laboratory, the team mimicked these conditions by growing bacteria on chitin and supplying DNA from different Vibrio cholerae strains or from other Vibrio species. They then tracked whether newly acquired gene cassettes were inserted into the first position of the SCI array.

    Through their studies the team confirmed that that V. cholerae can acquire new SCI gene cassettes from extracellular DNA. Collective experimental results, the authors stated, “We show that SCI cassettes are efficiently acquired by naturally competent V. cholerae and inserted at the first SCI array position in an integrase-dependent manner. This process incorporates cassettes not only from other V. cholerae strains but also from diverse Vibrio species.”

    In aquatic habitats, DNA is released when bacterial cells are killed by viruses, antimicrobial compounds, or bacterial weapons. Nearby competent bacteria can take up this DNA and incorporate selected fragments into their own SCI. “A loose comparison would be the following,” said Blokesch. “Imagine your grandmother passes away and, as a farewell gift, hands over the immunity she built up against the Spanish flu a century ago, immediately protecting you from that same virus. Wouldn’t that be amazing? This is essentially what we show that V. cholerae can do.”

    The team also showed that cassettes inserted in this position are functional. Several defense systems provided protection against viruses that infect Vibrio species, known as vibriophages. They stated, “In this study, we show that SCI diversification efficiently occurs by horizontal transfer linked to the genus’s aquatic lifestyle: DNA released from lysed cells is taken up by naturally competent vibrios and integrated into the first position of the SCI array, the primary site of strong expression, where it confers resistance to phage and potentially other threat,” the wrote in summary. “Together, these results demonstrate that SCI cassettes can cross species boundaries, supporting a model in which SCIs may function as genus-wide reservoirs of exchangeable genes, including defense genes, that confer selective advantages under certain conditions.”

    An important exception emerged. In the pandemic 7PET lineage of V. cholerae, the SCI appears largely static. “The SCI of 7PET V. cholerae is large but remarkably stable,” the authors noted. They propose that this reflects adaptation to a human-associated niche. “We propose that this reflects adaptation to a human-associated niche, where chitin is less abundant and competence induction—requiring growth on chitin to high cell density plus relief from catabolite repression is unlikely to occur,” they suggested. “As a result, SCI-mediated diversification may be largely inactive in pandemic strains.”

    However, if pandemic strains were to encounter environmental conditions that enable SCI cassette acquisition, they could expand their antiviral defenses. Blokesch commented. “This possibility matters because vibriophage-based approaches are currently being explored to prevent cholera in endemic regions, and such evolutionary flexibility could ultimately affect how effective these strategies remain.”

    In their paper the team concluded that since onset of the seventh pandemic, “… acquisition of novel and diverse, large defense-related genomic regions by 7PET strains appears to have been relatively limited. Consequently, reduced SCI-mediated diversification may lower the capacity of this lineage to rapidly evolve new defenses—an important consideration for ongoing efforts to deploy phage-based prophylaxis against cholera in endemic settings such as Bangladesh.”

    The post DNA Uptake in Cholera May Increase Defense Mechanisms appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BBB Access Route via Proteomic Vascular Mapping https://edusehat.com/en/bbb-access-route-via-proteomic-vascular-mapping https://edusehat.com/en/bbb-access-route-via-proteomic-vascular-mapping Fri, 10 Apr 2026 05:55:09 +0700 Edusehat BBB, Access, Route, via, Proteomic, Vascular, Mapping A limiting feature of many neurological therapies is the ability of molecules to cross the blood-brain barrier (BBB) from the circulatory system. Since the BBB prevents simple diffusion of materials across the divide, identifying the proteins responsible for transport is necessary for effective design of BBB-crossing therapies.

    “So basically, everything in the circulating blood, if they want to have an exchange with the organ, they need to pass through this interface,” says senior author Jiefu Li, PhD, Janelia Research Campus Group Leader at the Howard Hughes Medical Institute.

    Identification of the structures within blood vessels involved with the processes of molecular movement across the BBB has been somewhat elusive. However, Li and his team have developed a technique that not only identifies proteins within the luminal surface—the inner lining—of the vasculature, but also works in vivo, allowing them to track how these features change across the aging brain.

    “Understanding how the blood-brain barrier works, particularly figuring out the molecular targets that you can play with to open and close the barrier, will provide new possibilities for drug delivery,” Li says.

    Their work is published in Science in a paper entitled, “Luminal surface proteome of the brain vasculature uncovers blood-brain barrier regulators.”

    Using mice, the team developed a proteomic profiling method that can be used not only in brain vasculature, but throughout the body. “Briefly, a lectin-conjugated peroxidase is perfused and anchored to the luminal surface of blood vessels to catalyze the biotinylation of adjacent proteins, thereby enabling subsequent protein enrichment and mass spectrometry analysis,” wrote the authors.

    They tested the method in the brain, kidney and intestine, in both mice and northern tree shrew, showing functionality and applicability across organs and species.

    “This will allow us to say: we know that the vasculature system is doing different things in different organs and it relies on this luminal surface, but how does that happen? What are the molecular players there?” Li says.

    Using quantitative proteomics of the luminal surface—from early development through adulthood and aging—they found that over time there was a decrease in angiogenic and transport proteins. They also found an increase in proteins that increased stiffness in the vasculature.

    In addition to developing this in vivo technique, the team identified two proteins that are temporally distinct in their expression while both playing a role in modulating BBB permeability. Knockouts of nitric oxide synthase Nos3 and arginine transporter Slc7a1 resulted in BBB leakage in neonates, but not adults, while genetic screens identified hyaluronidase HYAL2 as being required for maintaining BBB integrity throughout the lifespan of mice.

    “What we know now is that we have two new pathways, potentially, to open the blood-brain barrier and to inform some therapeutic developments,” says Li.

    Utilization of this proteomic based method in vivo both opens up new avenues of functional research across the cardiovascular system, and also provides data and a methodology for novel therapeutic targets for crossing the BBB.

    “This method solves an important need but it’s also a very easy-to-use method, so everyone can use it,” Li says.

    The post BBB Access Route via Proteomic Vascular Mapping appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO Coffee Chat: Price controls like MFN harm access, increase costs https://edusehat.com/en/bio-coffee-chat-price-controls-like-mfn-harm-access-increase-costs https://edusehat.com/en/bio-coffee-chat-price-controls-like-mfn-harm-access-increase-costs Thu, 09 Apr 2026 15:40:35 +0700 Edusehat BIO, Coffee, Chat:, Price, controls, like, MFN, harm, access, increase, costs “Biotechnology innovation is advancing at an unprecedented pace, delivering new treatments and cures that offer long-awaited hope to patients and their families,” said Karin Hoelzer, Senior Director of Patient Advocacy at the Biotechnology Innovation Organization (BIO), during BIO’s March Coffee Chat.

    “Yet, affordability and timely access remain a persistent challenge, both in the United States and abroad,” she continued. “Recent efforts by policymakers to implement arbitrary drug price controls are exacerbating these barriers while, paradoxically, increasing out-of-pocket costs for many patients.“

    Hurting patient access through misguided policies

    Nearly four years after the Inflation Reduction Act’s (IRA) “price negotiations” were passed into law, one thing has become increasingly clear: for many patients, the law has done little to reduce out-of-pocket costs, while driving up utilization management by health insurers and raising concerns about the future of biotech innovation.

    While the law’s $2,100 cap on Medicare beneficiaries’ annual out-of-pocket costs and the ability to spread these costs throughout the plan year provides crucial protections for some, many beneficiaries face additional cost-shifting from their health plan. Mounting evidence shows that, on average, patients who do not reach the cap may actually be paying more—in some cases more than double the average expected out-of-pocket cost—due to increases in deductibles and shifts from co-pays to co-insurance.

    Medicare Access for Patients’ (MAPRx) recent report, “Inflation Reduction Act: Access Barriers Undermine Affordability,” certainly supports this. The report finds that, as the program enters 2026, “early evidence suggests that the benefits of the out-of-pocket cap are increasingly being offset by changes in plan behavior. Beneficiaries are facing higher premiums, fewer plan choices, and more aggressive formulary management. These developments risk shifting financial and administrative burden back onto beneficiaries in ways that were not the focus of the IRA’s affordability reforms.”

    Meanwhile, proposals such as Most Favored Nation (MFN) type policies—which would tie U.S. drug prices to those in other countries—could exacerbate these trends and further limit access by importing foreign pricing systems that often use health economics metrics that discriminate against people with chronic conditions and limit the availability of new therapies.

    These tensions were a central focus of the March Biotechnology Innovation Organization (BIO) Patient Advocacy Coffee Chat, Advocacy in Action: The Hope of Biotechnology and the Threat of Misguided Policies such as MFN-Type Drug Pricing Policies. Panelists explored how drug pricing policies, in the U.S. and globally, are narrowing patient access under the guise of improved affordability.

    ‘Affordability’ – but for whom?

    To frame the day’s conversation, Patrick Wildman, Senior Vice President, Advocacy & Government Relations at the Lupus Foundation of America and a key leader in the MAPRx Coalition, reminded the audience “We have to look at affordability and access in tandem.”

    “When we’re looking at the different policy issues out there, we want to see if there’s a balance when it comes to affordability and access,” he said.

    He continued to lay out four key questions patient advocates ask to evaluate policy proposals:

    • “First, we want to understand: Who is actually benefiting from these policies? Is it truly improving affordability for patients, or is it for the system or for other stakeholders?
    • Does affordability come at a cost? Are we creating barriers to care? Is it leading to increased utilization management, increased prior authorization, more step therapy, or limitations on formularies? Is it leading to cost shifting? Are we seeing higher deductibles? Are we seeing higher premiums or other shifts in cost sharing?
    • What do all these policies mean for the availability of care? Will these policies narrow available networks and access to specialists for patients, or ensure their availability?
    • And what does it mean for the future of care? How are these policies impacting the development of new treatments?”

    Evaluating each policy holistically, Wildman and other patient advocates strongly oppose MFN-type policies such as those recently outlined by the Centers for Medicare & Medicaid Services (CMS) that would tie U.S. reimbursement to foreign countries.

    In particular, MAPRx advocates have opposed the Guarding U.S. Medicare Against Rising Drug Costs (GUARD) model, which aims to align U.S. prices with international ones.

    As MAPRx explains in their comments to the CMS and Department of Health and Human Services (HHS), instead of lowering costs, the model is projected to actually increase beneficiary out-of-pocket spending; it creates strong incentives for plans to restrict access through various utilization management tools; and threatens access for orphan and protected class drugs, among other issues.

    These “solutions” fail to take into account the access issues that patients face abroad, and instead risk importing them to the U.S.

    Challenging Access Environments Abroad

    Will Greene, board member at the Foundation for Prader-Willi Research, recounted his experiences as the parent of a rare disease patient, living and working in Singapore.

    “Even though I worked for a global biopharmaceutical company with a multi-billion-dollar rare disease portfolio, congenital diseases were carved out of my employer-provided health insurance plan while I worked in Singapore,” he said. “So, I was essentially on my own financially. After more than $100,000 dollars in out-of-pocket expenses, I said, Okay, enough.”

    Greene and his family moved back to the U.S. and started over in the Bay Area. “Our experiences here in the U.S. have been much, much better than they were in Asia Pacific,” Greene said.

    His experience will not come as a surprise to many patient advocates. While the public discussion of drug affordability and access often assumes that healthcare systems abroad translate into better access, the reality is far more complex—and in many cases, far more restrictive for patients, particularly for those with rare genetic diseases.

    Access can vary dramatically abroad—a point made even clearer when looking at Europe’s complex drug approval and reimbursement systems.

    “In Europe, drug approval is centralized, but access is not,” explained Myriam Rai, PhD, Director of Global Relations & Initiatives at Friedreich’s Ataxia Research Alliance (FARA). “So a therapy may receive European-level approval, but whether a patient can actually receive it really depends entirely on the country where the person lives. This is a fundamental fragmentation that creates a big discrepancy from country to country.”

    Abroad, economic metrics that devalue people with chronic diseases are widely used

    As Rai noted, countries across Europe also differ in how they define and assess the value of a drug. “Some prioritize the clinical benefit, and others will focus more on cost effectiveness,” she said. “In both cases, unfortunately, proving clinical benefit and how meaningful it is for the quality of life is often really challenging. Additionally, most of the time, unfortunately, the voice of the patient is not being considered early enough.”

    For patients, these dynamics create a compounding effect: inconsistent national standards, rigid cost-effectiveness thresholds, and the use of quality-adjusted life year (QALY) metrics. In many countries abroad, cost-effectiveness frameworks rely on QALY metrics which systematically and inherently devalue treatments for people with chronic diseases, disabilities, or older adults.

    And for patients waiting to access transformative new therapies, time is of the essence. “You have the other big piece: the pricing negotiation,” Rai said. “While it will take maybe a year and a half to get reimbursement in some efficient countries in Europe, it will take much longer in other countries—and some may never reach reimbursement at all.”

    And, contrary to the U.S., there is no pathway to pay out-of-pocket for therapies.

    “Comparatively, in our closed European system, if it’s not greenlit to be reimbursed, you can’t even access the drug—even if you had philanthropy or could raise funds that enabled you to cover it,” Rai explained. “You would need to travel to the U.S. to get it, which is a vicious circle, and which is not fair.”

    Improving access in the U.S. by simplifying the system

    Just like in Europe, when patients get caught up in the overly complex U.S. healthcare system, access barriers can mean life or death, as Sarah Jones, MPA, MS, Community Engagement at the Eosinophilic & Rare Disease Cooperative (ERDC) explained.

    Her organization recently conducted a survey of over 200 patients with rare diseases (which will be published later this year), and their findings were stark. The vast majority of patients interviewed had health insurance coverage but still struggled with both affordability and access. ERDC found many people filing for bankruptcy or maxing out their credit cards just to cover the exorbitant out-of-pocket costs for their medications. Others were rationing their medication, delaying treatment and experiencing active disease as a result.

    As Jones explained, many chronic disease patients, herself included, rely on a large number of therapies to manage their conditions and all the associated symptoms. And, she and her organization are concerned that things are not trending in the right direction, with policies such as IRA and MFN actually increasing out-of-pocket costs across the board.

    “You look at things like the foreign price controls that are lurking out there, and you think, that’s only going to complicate things, and currently there is no mechanism in the proposed legislation to pass the savings on to patients” she said. “I keep thinking, we have got to get the word out there. You hear something like these foreign price controls, and the general community thinks that’s a great idea because that’s the talking point.”

    The post BIO Coffee Chat: Price controls like MFN harm access, increase costs appeared first on Bio.News.

    ]]>     Single-Cell Atlas of Maternal–Fetal Interface Sheds Light on Pregnancy Complications https://edusehat.com/en/single-cell-atlas-of-maternalfetal-interface-sheds-light-on-pregnancy-complications https://edusehat.com/en/single-cell-atlas-of-maternalfetal-interface-sheds-light-on-pregnancy-complications Thu, 09 Apr 2026 12:00:09 +0700 Edusehat Single-Cell, Atlas, Maternal–Fetal, Interface, Sheds, Light, Pregnancy, Complications The biological connection between a pregnant woman and her developing baby—the human maternal–fetal interface—is a specialized, transient organ composed of uterine cells from the mother and fetal cells that acts as a barrier, supports fetal growth, and maintains the mother’s health. The cellular complexity of the maternal-fetal interface has limited scientists’ ability to study how healthy pregnancies develop and why complications arise. The underlying cellular, molecular, and spatial programs of the interface—which forms about a week after fertilization and lasts until birth—has remain incompletely defined.

    Now, the human maternal–fetal interface has been mapped in unprecedented detail by scientists at the University of California, San Francisco (UCSF), revealing new cell types and providing insights into conditions such as preeclampsia, preterm birth, and miscarriage.

    “By examining this tissue cell by cell across pregnancy, we can begin to understand both normal development and what may go wrong,” said Susan J. Fisher, PhD, professor of obstetrics, gynecology, and reproductive sciences at UCSF.

    The team generated a comprehensive atlas of the human maternal–fetal interface across normal pregnancies, from early gestation to term. The researchers did this by “integrating large-scale paired single-nucleus transcriptomic and chromatin accessibility profiling with submicrometer-resolution spatial transcriptomics and CODEX multiplex protein imaging.”

    Using these tools, the researchers analyzed about 200,000 individual cells and compared them with nearly one million cells in their original positions within the uterine and placental tissue. This enabled them to identify different cell types, track how they develop, and see how they are linked to pregnancy complications.

    “This work gives us a much clearer picture of this critical region than ever before,” said Jingjing Li, PhD, associate professor in UCSF’s Department of Neurology and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.

    This work is published in Nature in the paper, “Single-Cell Spatiotemporal Dissection of the Human Maternal–Fetal Interface.”

    The atlas revealed a previously unknown maternal cell type located where fetal placental cells first enter the uterus. These cells appear to regulate how deeply placental cells invade uterine tissue, a process that is essential for establishing blood flow to the fetus. The researchers found that these cells carry a cannabinoid receptor, and exposure to cannabinoid molecules caused them to further restrict placental cell invasion.

    “Population studies have linked cannabis use during pregnancy to poorer outcomes,” said Cheng Wang, PhD, a postdoctoral fellow at UCSF. “This cell type may help explain the biological basis of that association.”

    To understand how complications arise, the team integrated genetic data from more than 10,000 patients. They mapped genetic risk signals for conditions including preterm birth, preeclampsia, and miscarriage onto regulatory regions of DNA that control gene activity. This approach allowed the researchers to identify the specific cell types and states most strongly associated with each condition.

    The team then focused on preeclampsia, a potentially life-threatening disorder marked by sudden high blood pressure. They found that the most affected cell types are involved in remodeling the mother’s uterine blood vessels, a process required to supply adequate blood to the placenta. The findings suggest that preeclampsia may result from disrupted communication between maternal and fetal cells that normally coordinate this process.

    Having established a detailed map of healthy pregnancies, the researchers plan to study complicated pregnancies to identify potential targets for treatment.

    The post Single-Cell Atlas of Maternal–Fetal Interface Sheds Light on Pregnancy Complications appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Organ-on-Chip Integrated Into Preclinical Glioblastoma Research https://edusehat.com/en/organ-on-chip-integrated-into-preclinical-glioblastoma-research https://edusehat.com/en/organ-on-chip-integrated-into-preclinical-glioblastoma-research Thu, 09 Apr 2026 08:25:09 +0700 Edusehat Organ-on-Chip, Integrated, Into, Preclinical, Glioblastoma, Research Dynamic42 and EPO (Experimental Pharmacology and Oncology), both based in Germany, report that they are addressing the limited availability of preclinical models in brain cancer research by forming a strategic collaboration that focuses on bringing organ-on-chip technologies “closer to the core of preclinical drug development.”

    The partnership combines Dynamic42’s organ-on-chip platforms with EPO’s expertise in translational oncology and access to well-characterized tumor models and patient-derived material. Together, the teams are developing experimental setups designed to reflect human tumor biology more closely and generate data that translates more reliably into clinical outcomes.

    The first joint projects target glioblastoma and the blood–brain barrier (BBB). Using Dynamic42’s human-based BBB-on-chip model, the partners will explore how differences between human and non-human BBB-biology can influence therapeutic responses, which is a major factor for the limited activity of brain cancer drugs.

    “Too often, critical decisions in drug development rely on data that do not fully reflect human biology,” said Thomas Sommermann, PhD, head of cancer research at Dynamic42. “We want to change that. By bringing human-based models earlier into the process, we can sharpen decision-making and reduce late-stage failure risks.”

    “For us, this collaboration is about strengthening the translational link,” added Jens Hoffmann, CEO at EPO. “Integrating advanced in vitro systems allows us to look at tumor biology from a different angle and to build robust experimental in vivo strategies.”

    The collaboration is designed as a complementary approach that connects established preclinical in vivo expertise with emerging human-based in vitro technologies. It supports more targeted, biology-driven research strategies and the principles of the 3Rs (Replace, Reduce, Refine), contributing to the ongoing shift toward more human-relevant experimental systems.

    Beyond joint research, the partnership includes model development activities, elaboration of commercialization strategies, and close scientific exchange, including collaboration between early-career researchers from both organizations.

    Dynamic42 and EPO will jointly present the first results of their collaboration at the American Association for Cancer Research® Annual Meeting 2026. Both companies plan to expand the collaboration further, exploring additional indications and extending the use of organ-on-chip technologies across different areas of drug development.

    The post Organ-on-Chip Integrated Into Preclinical Glioblastoma Research appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Scalable Embryonic Stem Cell Platform Enables Mitochondrial DNA Research in Mice https://edusehat.com/en/scalable-embryonic-stem-cell-platform-enables-mitochondrial-dna-research-in-mice https://edusehat.com/en/scalable-embryonic-stem-cell-platform-enables-mitochondrial-dna-research-in-mice Thu, 09 Apr 2026 04:50:11 +0700 Edusehat Scalable, Embryonic, Stem, Cell, Platform, Enables, Mitochondrial, DNA, Research, Mice Salk Institute scientists have developed a biological platform for studying mitochondrial DNA in physiology, adaptation, disease mechanisms, and therapeutic development. Headed by Ronald Evans, PhD, professor and director of the Gene Expression Laboratory and holder of the March of Dimes Chair in Molecular and Developmental Biology at Salk, the team has already used the platform to generate a library of 155 mitochondrial DNA mutant cell lines and reveal correlations between mouse development and mitochondrial function. They suggest that the platform, library, and findings will accelerate therapeutic development for mitochondrial disorders, as well as help scientists treat mitochondrial dysfunction in other diseases and conditions like cancer or aging.

    “Mitochondrial DNA accumulates mutations at a high rate, and more than 260 inherited disease-causing mtDNA mutations have been identified in humans,” said Evans. “Until now, a lack of models representing this diversity has limited mechanistic insight and therapeutic development. Our new platform will allow scientists to investigate mitochondrial DNA variation in health, disease, and evolution, which will enable therapeutic innovation for mitochondrial disorders.”

    Evans is co-corresponding author of the team’s published paper in PNAS, titled “A scalable embryonic stem cell–based platform for efficient generation of mitochondrial DNA mutant mice,” in which they concluded that their new platform, “… opens the door to mechanistic dissection of how mtDNA variation influences metabolism, adaptation, and disease, and provides a strategically valuable foundation for accel­erating therapeutic development through genetically precise mito­chondrial disease models.”

    Some of your most important life partners are the mitochondria that power all your cells. You and these little cellular powerhouses are in a 1.5-billion-year-old evolutionary relationship—but mitochondria brought some baggage. Mitochondria brought their own DNA with them when they joined with the bigger, more complex cells so long ago, and today that mitochondrial DNA influences human health. Mitochondrial DNA does the extremely important job of creating the proteins needed for energy production—but it also has an especially high rate of mutation, and those mutations can accumulate thanks to inefficient repair mechanisms. Because mitochondria are essential parts of every cell, their dysfunction can lead to body-wide dysfunction, with especially devastating impact on high-energy organs like the brain and heart. Without enough power in your cells, symptoms like migraines, muscle weakness, and loss of hearing or sight can begin to manifest.

    “Mitochondria are central to energy metabolism and cellular signaling, and mutations in mitochondrial DNA (mtDNA) can disrupt these processes and contribute to human disease,” the authors wrote. “Mitochondrial DNA (mtDNA) accumulates mutations at a high rate, and more than 260 pathogenic germline mtDNA mutations have been identified in humans, producing diverse and often tissue-specific disorders.”

    The chronic and broad impact of mitochondrial dysfunction makes it especially important to study. However, trying to pinpoint the outcome of specific mitochondrial DNA mutations has for many years been a slow, arduous process for many years. “… progress in defining how mtDNA variation influences adaptation, pathophysiology, and disease susceptibility has been limited by the lack of suitable animal models,” the team continued. “Researchers would create mouse models one-by-one with different mitochondrial DNA mutations, with just one model sometimes taking years,” said Salk staff scientist Weiwei Fan, PhD. This was a problem that Fan had noted early in his scientific career and set his mind to as a PhD student.

    The new Salk model is a scalable, embryonic stem-cell (ES)-based platform creating mice with mutations to their mitochondrial DNA. “This new work is all building off an original platform I generated during my PhD,” says Fan, first and co-corresponding author of the study. “That platform was inefficient—it took a long time to generate just one mitochondrial DNA mutant. With some technological improvements and modifications, this new platform is much more efficient and can create dozens of mutants with far greater ease.”

    A playful representation of a mitochondrion moving into a larger cell, bringing with it the "baggage" of mutated mitochondrial DNA. Researchers at the Salk Institute developed a new platform for studying that mutated mitochondrial DNA, helping explain the ways it influences human health. [Salk Institute]
    A playful representation of a mitochondrion moving into a larger cell, bringing with it the “baggage” of mutated mitochondrial DNA. Researchers at the Salk Institute developed a new platform for studying that mutated mitochondrial DNA, helping explain the ways it influences human health. [Salk Institute]
    The authors explained, “… we developed a scalable ES cell–based platform that integrates mtDNA mutagenesis, cybrid technology, high-sensitivity mutation detection, and optimized mouse transgenesis.” The platform starts with a protein, called mitochondrial DNA polymerase, generating randomly mutated mitochondrial DNA. That mutated mitochondrial DNA is then transferred into stem cells, which can be integrated with mouse embryos to create mice for study. Once one of these mice is established, researchers can investigate the specific symptoms of their specific mitochondrial DNA mutation and the mechanisms by which those symptoms arise—insight that can be used to design targeted therapies down the line. “Optimized ES cell–embryo aggregation enables robust contribution of mtDNA mutant ES cells to host embryos, producing chimeric mice with germline transmission,” the investigators noted.

    Using this platform, the Salk team generated a library of 155 mitochondrial DNA mutation cell lines, each with its own distinct impact on mitochondrial performance. “Using this platform, we generate a library of 155 donor fibroblast lines carrying distinct homoplasmic single-nucleotide mtDNA mutations that produce diverse mitochondrial phenotypes, including impaired oxidative phosphorylation, increased reactive oxygen species, and altered mitochondrial membrane potential,” they stated. They then used that library to validate that the cells could be used to generate mice with single mitochondrial DNA mutations. These mice allowed them to find a strong correlation between mitochondrial function and early embryonic development, suggesting a baseline energy level is required for normal development.

    “Our library is a huge milestone and is very diverse, with a scale of diversity similar to the known human disease-causing mutation diversity of around 260,” said Fan. “And with this collection of mutant cells, we can not only look at inherited mutations but also at ones that occur based on other stresses like environmental cues or aging.” The authors added, “Together, the advances outlined in this study establish a powerful and generalizable platform for systematically modeling the functional diversity of human mtDNA mutations and polymorphisms in vivo.”

    The new platform and library are cracking open the world of mitochondrial DNA. With the ability to generate mitochondrial DNA mutants more rapidly, therapeutic development for mitochondrial disease and dysfunction will come more rapidly, too. The mouse models are already a huge step forward for the field, but the researchers are also eager to move into human models in a more human-relevant context.

    “The majority of human diseases come with or cause mitochondrial dysfunction,” said Evans. “Progress in this field has been limited, but this new platform is going to fuel so much important research that points to therapeutic approaches to combat mitochondrial diseases, as well as diseases or conditions associated with mitochondrial dysfunction like cancer or aging.”

    In their paper the team concluded, “The library provides a unique and comprehensive resource for modeling the diversity of human mtDNA variation in vitro and can also be used to generate in vivo models through ES-cybrid technology … By enabling the generation of both pathogenic and physiologically relevant mtDNA variants—including those resembling somatic mutations associated with aging and cancer—this platform substantially expands the toolkit available to mitochondrial researchers.”

    The post Scalable Embryonic Stem Cell Platform Enables Mitochondrial DNA Research in Mice appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Neuroblastoma Tumor Growth in Mice Suppressed by Blocking Enzyme to Inhibit mTOR Signaling https://edusehat.com/en/neuroblastoma-tumor-growth-in-mice-suppressed-by-blocking-enzyme-to-inhibit-mtor-signaling https://edusehat.com/en/neuroblastoma-tumor-growth-in-mice-suppressed-by-blocking-enzyme-to-inhibit-mtor-signaling Thu, 09 Apr 2026 04:50:09 +0700 Edusehat Neuroblastoma, Tumor, Growth, Mice, Suppressed, Blocking, Enzyme, Inhibit, mTOR, Signaling Neuroblastoma is the most common tumor among children under a year of age, and while in its gentlest form neuroblastoma can regress on its own, it can also take an aggressive form, with high-risk neuroblastoma carrying a five-year survival rate of about 40%.

    Researchers at The Hebrew University of Jerusalem have now discovered a mechanistic explanation for how neuroblastoma sustains itself and identified a potential approach to severing that mechanism, by inhibiting nitric oxide (NO) production to suppress mTOR signaling. The collective results from work in human neuroblastoma cells and experiments in a mouse xenograft model showed that inhibiting the enzyme neuronal nitric oxide synthase (nNOS) to inhibit NO production suppressed mTOR signaling and slowed tumor growth.

    Professor Haitham Amal, PhD, head of The Laboratory of Neuromics, Cell Signaling, and Translational Medicine, is senior and co-corresponding author of the team’s published paper in Brain Medicine, titled “Targeting nNOS suppresses AKT–TSC–mTOR signaling and inhibits neuroblastoma growth.” In their paper the team concluded “Inhibition of nNOS suppresses mTOR signaling, reduces cellular malignancy, and attenuates tumor growth in vivo, identifying the nNOS-mTOR axis as a promising therapeutic target in neuroblastoma.”

    Neuroblastoma accounts for roughly 28% of all cancers diagnosed in infants across Europe and the United States. “Neuroblastoma (NB) refers to a spectrum of neuroblastic tumors that originate from the neural crest cells during fetal development,” the authors wrote. “Neuroblastoma is predominantly a pediatric malignancy, with approximately 97% of cases occurring in children.”

    NBs can range from spontaneous regression to maturation to an aggressive, deadly metastatic disease. And as the investigators noted, “Despite major advances in multimodal therapy, high-risk neuroblastoma remains associated with poor prognosis, frequent relapse, and therapy resistance, underscoring the need for a better understanding of the signaling pathways that regulate tumor cell survival, differentiation, and metabolic adaptation.”

    Nitric oxide (NO) is an essential regulator of carcinogenesis in various tumors, including NB, the authors pointed out. “Nitric oxide (NO) is a ubiquitous free radical signaling molecule produced in multiple organs and tissues), such as those of the central and peripheral nervous systems.” But at elevated concentrations NO becomes reactive, generating nitrogen species that chemically modify proteins through a process called S-nitrosylation. That modification has been implicated in every stage of cancer progression.

    The relationship between nitric oxide and tumors is not simple. Very high concentrations can damage DNA and trigger apoptosis. Lower, sustained levels appear to do the opposite, promoting survival and metastasis. Amal and colleagues had previously demonstrated that nitric oxide drives glioblastoma progression. The question that remained was whether the same enzyme, neuronal nitric oxide synthase, was performing a similar service for neuroblastoma, and if so, through which downstream pathway. The answer turned out to be mTOR.

    The team attacked nNOS from two directions. They treated human SH-SY5Y neuroblastoma cells with BA-101, a selective pharmacological inhibitor, at 100 μM for 24 hours. Separately, they silenced the nNOS gene with small interfering RNA. The reasoning was that if a drug and a genetic tool produce the same result, you are looking at biology, not pharmacological noise.

    The experiments produced the same result. BA-101 reduced NADPH-diaphorase activity, the standard readout of NOS function, by 35-40%. Genetic silencing cut it by 45-50%. Nitrite levels, a stable proxy for nitric oxide production, fell 65-70% with BA-101 and 55-60% with siRNA. Colony formation, the most direct measure of proliferative capacity, dropped significantly after both BA-101 treatment (p < 0.001) and nNOS silencing (p < 0.01). The cells were losing their ability to multiply.

    What followed downstream was systematic. Protein tyrosine nitration, measured by 3-nitrotyrosine immunoreactivity, fell sharply after BA-101 treatment (p < 0.01) and nNOS silencing (p < 0.001). The chemical signature of nitrosative stress was fading.

    The results then confirmed that AKT phosphorylation decreased (p < 0.01 with BA-101; p < 0.05 with siRNA), while total AKT remained unchanged. Phosphorylation of mTOR itself declined under both conditions (p < 0.01 each). The downstream mTORC1 substrate ribosomal protein S6 followed (p < 0.05 with BA-101; p < 0.01 with siRNA).

    And here, the most telling detail, that TSC2, a master negative regulator of mTOR signaling, rose significantly under both treatments (p < 0.05). Removing the nitric oxide signal had allowed the cell’s own braking system to re-engage. In summary, the authors noted, “Pharmacological inhibition of nNOS with BA-101 (100 μM, 24 h) or genetic silencing of nNOS with siRNA caused upregulation of the key negative regulator TSC2 and decreased phosphorylation of AKT, mTOR, and RPS6, indicating suppression of mTOR pathway activity.”

    Synaptophysin, a neuroendocrine tumor marker used to gauge the malignant identity of neuroblastoma cells, decreased significantly with BA-101 (p < 0.01) and nNOS knockdown (p < 0.05). The tumor cells were not merely growing more slowly. They were becoming, at a molecular level, less recognizably cancerous. In summary, the investigators noted, “Our results show that inhibition of NO production in the human NB cell line (SH-SY5Y cells), either by pharmacological intervention using the selective nNOS inhibitor BA-101 (41) or by genetic ablation using the specific siRNA, successfully suppressed NB malignancy.”

    Schematic model illustrating the NO-mTOR signaling axis in neuroblastoma. Under basal/pathological conditions (left panel), and nNOS inhibition (right panel). [Haitham Amal]
    Schematic model illustrating the NO-mTOR signaling axis in neuroblastoma. Under basal/pathological conditions (left panel), and nNOS inhibition (right panel). [Haitham Amal]
    But if blocking nitric oxide suppresses mTOR signaling, then flooding the cell with nitric oxide should amplify it. The researchers tested this by exposing SH-SY5Y cells to SNAP, a nitric oxide donor, at 200 μM for 24 hours. This converse experiment produced the converse result. 3-nitrotyrosine rose (p < 0.05), and TSC2 fell (p < 0.01). Phosphorylation of AKT, mTOR, and RPS6 all increased (p < 0.05 for each).

    The team then tested their findings in a xenograft mouse model of neuroblastoma, treated with BA-101. “Importantly, to extend these findings to an in vivo context, we further assessed the impact of pharmacological nNOS inhibition on tumor growth in a xenograft NB model,” they stated. The investigators found that while tumors in control animals grew to approximately 1.5 cm in their largest dimension, the treated tumors did not. Final tumor volume and weight were dramatically reduced in the BA-101 group. ‘Quantitative analysis revealed a dramatic decrease in the final tumor volume and weight in the BA-101-treated group (p < 0.001) compared with controls,” they noted.

    Body weight did not differ significantly between groups, suggesting that the compound was tolerated without gross systemic toxicity. In summary, the authors wrote, “Our finding demonstrate that the pro-tumorigenic effects of nNOS in SH-SY5Y involve activation of themTOR signaling pathway.” Importantly, both genetic inhibition of nNOS using siRNA and pharmacological inhibition with BA-101 effectively suppressed mTOR pathway activation and reduced malignant properties of NB cells, highlighting the therapeutic relevance of targeting nNOS signaling.  “These findings indicate that pharmacological inhibition of nNOS effectively suppresses xenograft tumor progression, highlighting the critical role of nNOS-derived NO in promoting neuroblastoma growth in vivo.”

    “The magnitude of the in vivo suppression caught our attention,” said Amal, the study’s corresponding author, who holds appointments at the Institute for Drug Research, School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, and the Rosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center at Boston Children’s Hospital, Harvard Medical School. “We had demonstrated the role of nitric oxide in glioblastoma previously, but the consistency of the neuroblastoma results across every assay, from protein phosphorylation to colony formation to xenograft growth, points to nNOS as something more than a contributor. It appears to be a central driver of the signaling that sustains this tumor.”

    Added first author Shashank Kumar Ojha, PhD, first author of the study and a researcher at the Institute for Drug Research, The Hebrew University of Jerusalem, added, “What convinced me was the concordance between the pharmacological and genetic approaches. When BA-101 and siRNA independently produce the same pattern of effects across NADPH-diaphorase activity, nitrosative stress markers, mTOR pathway phosphorylation, and clonogenic growth, you can be confident the biology is real. That reproducibility is what gives you a therapeutic hypothesis worth testing further.”

    The authors acknowledged limitations to their study. The in vitro work relied on a single cell line, SH-SY5Y, which cannot capture the full genetic heterogeneity of neuroblastoma or the complexity of the tumor microenvironment. The chemical identity of BA-101 is currently undisclosed pending patent issuance, which means independent replication by other laboratories must wait. Whether nitrosative stress directly underlies its functional impairment, or whether an intermediary mechanism is involved, remains an open question that the authors explicitly flag for future investigation. “Future studies using patient-derived cells, organoids, or genetically engineered mouse models will be important to further validate and extend these observations,” they stated. Nevertheless, the authors suggest, the limitations do not diminish the central discovery of a druggable nNOS–mTOR axis.

    mTOR inhibitors such as rapalogs and catalytic mTOR inhibitors have shown limited efficacy as monotherapies in neuroblastoma, undermined by feedback activation and resistance mechanisms. The present study suggests the potential for a different attack strategy. Rather than targeting mTOR at the lock, intervene upstream at the hand that turns the key. By reducing nitric oxide-dependent mTOR activation, nNOS inhibition may sidestep the compensatory pathways that have frustrated direct mTOR blockade. “Collectively, these results identify the nNOS-mTOR axis as a key driver of neuroblastoma progression and suggest that nNOS inhibition represents a promising strategy for NB treatment,” they concluded.

    The post Neuroblastoma Tumor Growth in Mice Suppressed by Blocking Enzyme to Inhibit mTOR Signaling appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Childhood Dementia Explained by Synaptic Dysfunction, Opens New Therapies https://edusehat.com/en/childhood-dementia-explained-by-synaptic-dysfunction-opens-new-therapies https://edusehat.com/en/childhood-dementia-explained-by-synaptic-dysfunction-opens-new-therapies Thu, 09 Apr 2026 01:15:13 +0700 Edusehat Childhood, Dementia, Explained, Synaptic, Dysfunction, Opens, New, Therapies In a new study published in Nature Communications titled, “Modelling synaptic dysfunction in childhood dementia using human iPSC-derived cortical networks,” researchers from Flinders University in Adelaide have uncovered how hyperactive and dysregulated synaptic circuits emerge in the brain tissue of children impacted by Sanfilippo syndrome, a common form of childhood dementia. 

    In Australia, an estimated 1400 children currently live with childhood dementia, with hundreds of thousands of cases worldwide. Sanfilippo syndrome is a rare genetic condition that causes fatal brain damage. Children typically reach early developmental milestones before rapidly losing cognitive skills, speech, and mobility. Early symptoms often include hyperactivity and sleep disturbance. 

    Alterations in synaptic communication play key roles in neurodegenerative disease progression and cognitive decline. Yet few studies have explored how excitation and inhibition synaptic imbalances contribute to pediatric neurodegenerative disorders. 

    Cedric Bardy, PhD, professor and head of the Laboratory for Human Neurophysiology and Genetics at the South Australian Health, describes the study findings as “significant progress.” Chronic overactivity in the brain appears to be a fundamental mechanism contributing to cognitive deterioration in children with Sanfilippo syndrome. 

    Using human stem cell-derived cortical neurons and electrophysiology, the team demonstrated that excitatory synapses in the neurons of affected children become abnormally active during early brain development. 

    While these neurons initially developed and functioned normally, they became increasingly overactive over time. Brain cell networks showed bursts of intense, highly synchronized electrical activity as they matured, mirroring the hyperactivity and neurological symptoms seen in children with the condition. 

    “This hyperactivity offers a clear biological explanation for early behavioral changes, and it brings us closer to understanding the complex mechanisms contributing to childhood dementia,” said Bardy.

    Results also demonstrated that these neurons are vulnerable to stress. When exposed to mild nutrient deprivation, excitatory synaptic abnormalities increased, suggesting that common illnesses or physiological stressors may accelerate neurological decline. 

    “Our research shows that disrupted synaptic communication is not simply a byproduct of degeneration. It is an early driver of the disease,” Bardy says. 

    Childhood Dementia Initiative CEO and founder, Megan Maack, is a co-author of the study and has been involved in guiding the project since its inception. 

    “This research is significant not just for Sanfilippo syndrome, but for the field of childhood dementia as a whole,” said Maack. “By identifying the precise cellular mechanisms driving the disease, we are moving towards a personalized medicine approach—the kind of targeted treatment strategy that has transformed outcomes for children with cancer.”

    Researchers are now evaluating whether drugs that are already on the market for use in other conditions could be repurposed for childhood dementia. Bardy says the team has already demonstrated that these synaptic imbalances can be corrected with certain medications in the laboratory, indicating that they represent a genuine therapeutic target. 

    The post Childhood Dementia Explained by Synaptic Dysfunction, Opens New Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Plant Molecular Farming Comes of Age https://edusehat.com/en/plant-molecular-farming-comes-of-age https://edusehat.com/en/plant-molecular-farming-comes-of-age Thu, 09 Apr 2026 01:15:12 +0700 Edusehat Plant, Molecular, Farming, Comes, Age Plant molecular farming (PMF) may seem like a bold option for companies accustomed to mammalian or microbial systems, but recent advances have transformed plant-based bioproduction into serious, scalable biomanufacturing platforms able to produce even complex biologics cost-effectively.

    “A major advantage is sustainability,” Marco P.C. Marques, PhD, associate professor, University College London (UCL), tells GEN. This comes at a time when “…regulators and global initiatives are putting real pressure on industry to reduce environmental footprint(s). Because plants grow using low energy inputs rather than stainless steel reactors or energy-intensive systems, they can bring down operating costs, reduce carbon emissions, and provide more flexible manufacturing options.”

    Additional benefits include PMF systems’ ability to support eukaryotic protein-folding and post-translational modification pathways, their lack of human pathogens, minimal biosafety risks, and compatibility with distributed manufacturing.

    PMF reached its current state because sensors, host plant engineering, AI-enabled models, and related technologies have become more mature, reliable, and predictable in the past few years. Consequently, “PMF platforms can deliver consistent, good manufacturing practice (GMP)-compatible performance while needing far less infrastructure, [which] allows much faster setup than conventional approaches,” Marques says.

    Robust, economic, responsible

    In a recent paper, he and first author Teresa Iucci, PhD, a bioprocessing scientist at Sapienza University of Rome and UCL, cite 13 companies that are using or have used plants to produce a variety of proteins, including antibodies, enzymes, and peptides, for vaccines and other biologics. Many are at clinical or commercial scale.

    Those examples show “that controlled cultivation, advanced transient-expression systems, and more refined downstream workflows can overcome many of the technical and regulatory hurdles historically associated with plant-based biomanufacturing.” In particular, they note substantial improvements in host plant engineering. Now, they point out, Nicotina plants can produce mAbs and Fc-fusion proteins that closely match those derived from CHO cells.

    However, “Realizing the full value of these biological innovations will depend on aligning PMF with contemporary digital manufacturing principles,” Iucci and Marques stress.

    “There is a lot of scope for continued innovation…particularly on the molecular biology side, where further gains in expression, stability, and product quality are very achievable,” Marques elaborates. “Downstream processing could also be better tailored to plant-based hosts,” to lower costs further.

    The benefits of PMF are well-recognized, but biomanufacturers also need clear, streamlined regulatory pathways and the internal determination that PMF is worth sustained investment.

    For biomanufacturers, “A good starting point is simply to treat PMF as a genuine production platform rather than an interesting alternative,” he says. To be able to compare PMF products with those derived from traditional mammalian or microbial cultures, he calls for the industry to standardize unit operations and generate regulatory-grade datasets, and then to run comparability studies and pilot-scale campaigns.

    Running such campaigns is becoming increasingly practical with the conjunction of sensors and data-driven processors. In vertical farming facilities, for example, every parameter critical for plant growth is tightly monitored and controlled using digital sensors to enable precise, real-time environmental adjustments.

    Ultimately, this allows producers to select the optimal timing of such events as infiltration and harvest at levels not possible in conventional greenhouses. “The long-term objective is a semi-continuous, digitally regulated PMF production line that links infiltration, extraction, and purification into a coherent, self-correcting workflow,” Iucci and Marques write.

    Transitioning from mammalian or microbial systems to PMF, “is easier said than done…especially when companies already have well-established mammalian or microbial platforms with validated processes and established supply chains,” Marques acknowledges. “In many respects, it would be simpler to design a PMF-based approach from scratch than to retrofit it into an existing operation…but with the right incentives (such as additional revenue streams from side processes), application cases, and evidence, we may well see more companies prepared to make that shift.”

    The post Plant Molecular Farming Comes of Age appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Redefining Bioprocessing Using Reservoirs of Biochemical Diversity https://edusehat.com/en/redefining-bioprocessing-using-reservoirs-of-biochemical-diversity https://edusehat.com/en/redefining-bioprocessing-using-reservoirs-of-biochemical-diversity Thu, 09 Apr 2026 01:15:10 +0700 Edusehat Redefining, Bioprocessing, Using, Reservoirs, Biochemical, Diversity In the global race to improve how medicines are made, scientists are turning to an unlikely source of innovation: the microscopic life thriving in some of the harshest soils on Earth. Beneath wild plants in Saudi Arabia’s arid landscapes, researchers have identified biological tools that could redefine bioprocessing.

    A recent study by Saudi Arabia-based Rewaa S. Jalal, PhD, associate professor of biology at the University of Jeddah, and Fatimah M. Alshehrei, PhD, associate professor of microbiology at Umm al-Qura University, focuses on the rhizosphere—the thin layer of soil surrounding plant roots—where dense microbial communities interact with their host plants. These environments, shaped by extreme heat and limited water, are proving to be reservoirs of biochemical diversity with direct relevance to drug manufacturing.

    The researchers zeroed in on enzymes known as glycosyltransferases, which play a central role in building complex sugar structures on proteins and other molecules. In pharmaceutical bioprocessing, this step—glycosylation—is crucial. It determines how therapeutic proteins behave, influencing everything from stability to effectiveness and immune compatibility.

    What makes these enzymes especially compelling is their environmental pedigree. The microbes that produce them have adapted to survive under intense stress, evolving systems that remain functional in high temperatures and low-moisture conditions. These traits could translate into more robust and flexible bioprocessing workflows, where maintaining strict environmental control is often costly and technically demanding.

    The study also reveals that different plant species cultivate distinct microbial communities, each enriched with unique enzyme families. For example, the rhizosphere of Moringa oleifera shows a different enzymatic profile compared to Abutilon fruticosum, highlighting how plant-microbe partnerships shape biochemical potential. For bioprocessing, this diversity could enable the selection of highly specific enzymes tailored to particular drug production needs.

    Beyond protein modification, the identified enzymes are linked to the synthesis of key biomolecules such as cellulose, chitin, and β-glucans. These materials are already used in areas like drug delivery, wound care, and tissue engineering. Improving how they are produced through advanced bioprocessing could expand their applications and reduce manufacturing constraints.

    Despite the promise, the researchers emphasize that their findings are based on computational analysis of genetic data. The real-world performance of these enzymes in industrial bioprocessing systems remains to be tested.

    Still, the implications are significant. As pharmaceutical companies seek more sustainable and efficient ways to produce complex biologics, enzymes shaped by extreme environments might offer a powerful advantage. Instead of engineering solutions from scratch, scientists are increasingly uncovering them in nature—already optimized through evolution.

    In this emerging vision of bioprocessing, the future of medicine might be shaped not only by cutting-edge technology but also by the resilient microbial ecosystems hidden beneath desert plants.

    The post Redefining Bioprocessing Using Reservoirs of Biochemical Diversity appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Faster Process Development via “Transfer Learning” https://edusehat.com/en/faster-process-development-via-transfer-learning https://edusehat.com/en/faster-process-development-via-transfer-learning Thu, 09 Apr 2026 01:15:09 +0700 Edusehat Faster, Process, Development, via, “Transfer, Learning” An emerging artificial intelligence technique called “transfer learning” could help drug makers use data to speed up the development of biopharmaceutical manufacturing processes, according to new analysis.

    In transfer learning, predictive models that have been trained on historical data are used to improve the performance of a task.

    Unlike machine learning (ML)—where the training process begins from scratch—transfer learning applies existing knowledge to new but related problems, reducing the amount of data and time required to build the model.

    Researchers at the Karlsruhe Institute of Technology in Germany, who looked at the approach, identified several potential biopharma applications, according to lead author Daniel Barón Díaz, citing reactor modeling as an example.

    “Transfer learning models can be used to predict critical outcomes like viable cell density (VCD) and product titre from online sensor data—for example, pH, temperature, gas flow—from historical data from a different, but related process.”

    The approach can also optimize process monitoring. Díaz tells GEN that, “Transfer learning-enhanced soft sensors can be established to monitor protein concentrations in real-time by leveraging existing models from related fermentations.”

    Data limitation

    When compared with other model-building techniques, transfer learning offers potential cost and time savings, according to Díaz, who cites a reduced experimentation burden as an example.

    “Conventional machine learning requires large, structured datasets that are often unavailable in biopharma due to the high cost and labor-intensive nature of experiments. Transfer learning allows companies to leverage historical data and existing models to build reliable predictors for new processes with very limited data.

    “By reusing prior knowledge, transfer learning can significantly decrease the number of experiments required—sometimes needing only one to three batches to achieve robust simulations,” he says.

    However, the ultimate benefit is that transfer learning speeds up process model development, according to Díaz, who adds, “It can make model adaptation faster than retraining from scratch, facilitating quicker process design and digital twin deployment.”

    Challenges

    So, transfer learning has the potential to create predictive models for manufacturing development. However, the key caveat is that the processes involved must be sufficiently similar for it to be effective, Díaz says.

    “For transfer learning to be effective, the source and target domains must be meaningfully related. If the processes are too different, the assumptions and learned representations may not align, leading to negative transfer, where the transferred knowledge actually degrades the model’s performance.

    “Data sets obtained at different scales or under varying conditions are often inconsistent, which can hinder the successful transfer of knowledge. Fine-tuning complex neural network architectures on very small target datasets can lead to overfitting, where the model fails to generalize to new data,” he says.

    To address this, manufacturers will need to establish metrics to determine similarity, Díaz explains.

    “There are currently no standardized metrics for measuring domain similarity in bioprocessing, nor are there comprehensive benchmark datasets to easily compare different transfer learning techniques.”

    Another challenge is the current lack of AI expertise in the industry, Díaz says.

    “There is often a disciplinary knowledge gap between process engineers and data scientists, and ML models without a mechanistic backbone may be perceived as opaque black boxes, hindering trust and industrial adoption,” he tells GEN.

    The post Faster Process Development via “Transfer Learning” appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Forecasting Protein Aggregation with an Improved Algorithm https://edusehat.com/en/forecasting-protein-aggregation-with-an-improved-algorithm https://edusehat.com/en/forecasting-protein-aggregation-with-an-improved-algorithm Thu, 09 Apr 2026 01:15:08 +0700 Edusehat Forecasting, Protein, Aggregation, with, Improved, Algorithm A new, improved algorithm for studying protein aggregation could help biologics manufacturers design better-performing products with less experimental effort. The software, developed by scientists based in Barcelona, offers the ability to analyze the aggregation of proteins drawn from the AlphaFold protein structure database, as well as helping companies identify more soluble alternatives.

    “Protein aggregation is a bottleneck in the production and manufacturing of biologics,” explains Salvador Ventura, PhD, a professor in the department of biochemistry and molecular biology at the Autonomous University of Barcelona (UAB).

    The problem, he explains, is that many proteins used as therapies evolved to be soluble at the concentrations found in the human body. But therapeutics, such as antibodies, are produced in as high a concentration as possible.

    “We want the product to deliver the maximum dose with the minimum amount of injection,” he says. “But proteins aren’t designed to be soluble at these concentrations, and their aggregation causes different effects.”

    These can include the patient’s immune system reacting negatively or the aggregated product ceasing to work.

    To overcome this problem, Ventura says, companies and labs try to forecast protein aggregation, usually experimentally with high-throughput combinational assays. But these approaches are not convenient for startups or small spinoff companies.

    A computational approach, such as his algorithm, now in its fourth generation, can help these companies predict and design around protein aggregation.

    It offers the ability to draw protein structures from AlphaFold to analyze likely protein aggregation using simulations of molecular dynamics. Users, he says, can also choose to mutate selected parts of the protein, identify other proteins in the same family, and even look at the possible impact of pH on solubility.

    “Our lab is both computational and experimental, so most of the designs we’ve made, we’ve already proved by experiment,” Ventura says.

    Limitations include the scarcity of high-quality experimental data available to train the algorithm, he explains.

    Going forward, the team intends to model which solution and formulation conditions best maintain the stability of therapeutic proteins in manufacturing and clinical settings. “We’re working on these next steps already,” he says. “Although, as yet, we don’t have an algorithm for this.”

    Ventura spoke about the latest version of his algorithm at the Bioprocessing Summit Europe in March.

    The post Forecasting Protein Aggregation with an Improved Algorithm appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     New Single‑Cell Platform Tracks RNA and Protein in Immune Signaling https://edusehat.com/en/new-singlecell-platform-tracks-rna-and-protein-in-immune-signaling https://edusehat.com/en/new-singlecell-platform-tracks-rna-and-protein-in-immune-signaling Wed, 08 Apr 2026 18:05:10 +0700 Edusehat New, Single‑Cell, Platform, Tracks, RNA, and, Protein, Immune, Signaling A new single‑cell sequencing method is giving researchers a clearer view of how immune cells actually behave—capturing not just what they plan to do, but what they are doing in real time. The platform, called CIPHER‑seq, measures RNA and proteins simultaneously inside the same immune cell, exposing gaps between genetic intent and functional output that have long complicated studies of cytokine signaling. The work, titled “CIPHER-seq enables intracellular multimodal profiling of cytokine responses in single immune cells,” appears in Scientific Reports.

    Single‑cell RNA sequencing has reshaped immunology by revealing which genes are switched on across thousands of cells at once. But RNA alone can be misleading, especially for cytokines. However, RNA is only a set of instructions; proteins carry out the action. And for cytokines, RNA levels often fail to predict how much protein a cell actually produces. “In immune cells, RNA and protein don’t always rise and fall together,” said co‑senior author Emiliano Cocco, PhD, an assistant professor of biochemistry and molecular biology at the Miller School.

    CIPHER‑seq (Cytokine Intracellular Protein High-throughput Expression with RNA-sequencing) was designed to close that gap. Developed by researchers at the Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine, together with collaborators at UCSF and the Helen Diller Family Comprehensive Cancer Center, the method gently preserves cells and captures multiple molecular layers at once. From a single immune cell, CIPHER‑seq can quantify genome‑wide RNA, surface proteins, intracellular proteins, and cytokines that have not yet been released—creating a more complete snapshot of immune activity than RNA‑only approaches.

    “RNA gives us clues about where a cell is headed,” said co‑senior author Justin Taylor, MD, a Sylvester physician-scientist. “Proteins show us where it actually arrives, and this clearer picture could help scientists design better immunotherapies and help clinicians predict which patients are most likely to benefit from them.”

    The team validated the platform by stimulating peripheral blood mononuclear cells (PMBCs) and tracking their responses. According to the study, CIPHER‑seq captured robust induction of key cytokines—including interferon‑gamma and tumor necrosis factor—while also resolving metabolic remodeling during activation. Importantly, the method revealed the timing of these events: RNA signals rose first, followed by delayed but consistent protein accumulation. First author Avni Bhalgat, PhD, described it as “seeing the plan before the action. Cytokines help determine whether immune cells attack cancer, ignore it, or even help tumors grow.”

    The researchers also compared CIPHER‑seq with standard single‑cell workflows and found a notable difference: cells processed with CIPHER‑seq showed far fewer mitochondrial stress signatures. Some existing protocols inadvertently damage cells during preparation, triggering artificial stress responses. By reducing these artifacts, CIPHER‑seq provides a cleaner readout of immune behavior.

    The authors emphasize that this multimodal view is especially valuable for studying cancer, inflammation, and treatment resistance—contexts where cytokine timing and protein abundance can shape therapeutic outcomes. “The platform helps us move beyond inference and toward understanding how immune responses truly unfold—one cell at a time,” Taylor added. By tracking RNA and protein together, CIPHER‑seq moves researchers beyond inference and toward a step‑by‑step understanding of how immune responses unfold.

    The post New Single‑Cell Platform Tracks RNA and Protein in Immune Signaling appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO expert: Future of biotech depends on competing with China, updating FDA https://edusehat.com/en/bio-expert-future-of-biotech-depends-on-competing-with-china-updating-fda https://edusehat.com/en/bio-expert-future-of-biotech-depends-on-competing-with-china-updating-fda Wed, 08 Apr 2026 03:50:03 +0700 Edusehat BIO, expert:, Future, biotech, depends, competing, with, China, updating, FDA Ensuring a brighter future for U.S. biotech requires modernizing the Food and Drug Administration (FDA) and addressing challenges from China, according to Kelly Seagraves, VP for National Security and International Affairs at the Biotechnology Innovation Organization (BIO).

    Seagraves explained these and other BIO priorities at an April 1 online panel on “the future of U.S. biotech” hosted by Research!America, a non-profit medical and health research advocacy alliance. In laying out the goals of the panel, moderator Jenny LaRay of Research!America explained, “We’re going to explore biotech’s importance to patients, to our economy, current challenges, and policy priorities.”

    While the second panelist, Garrett Dunlap of the Engineering Biology Research Consortium (EBRC), discussed engineering using biotech, Seagraves focused on health biotech and the concerns of BIO’s members. Seagraves was asked to explain the biggest challenges to the industry that BIO is seeking to address.

    “Any policies that bring in uncertainty are big concerns for our members,” she said, “international reference pricing policies, tariffs, and other trade-related policies, the uncertainty and the kind of back and forth there really hinders our members from making business decisions.”

    She also mentioned the need for FDA reform and the need to compete with China, two subjects she expanded upon during the rest of the panel.

    “One of my roles at BIO is leading on national security,” Seagraves explained. “We’re really trying to make sure that we are advocating for advancing American biotech in order to compete with China and make sure that we can keep American biotech leadership here at home.”

    Concerns regarding competition with China

    “China’s multi-decades-long concerted policy to try and really dominate the biotech ecosystem is leading to successes,” Seagraves said. “They’re really becoming a serious and very accelerated competitor in the biotech ecosystem. And from BIO’s perspective, we want to make sure that we are addressing potential risks.”

    She explained that China’s advances in biotech raise two key concerns: continued access to the latest healthcare innovations for American patients and potential national security threats.

    “We’re trying to figure out how to find solutions that will solve those problems and create resiliency,” Seagraves said. “One of the key partners that we use there to try and find some of those ideas is the National Security Commission on Emerging Biotechnology. I think their report really provides us with a good overview of the situation and a good roadmap for solutions.”

    Established with bipartisan support as part of the 2022 defense budget, the NSCEB was tasked by Congress to conduct “a comprehensive review of emerging biotechnology’s impact on national security and provide practical recommendations to preserve American dominance in this field.”

    Seagraves said the report and its conclusions provide necessary guidance for addressing challenges from China. The solutions that are put in place cannot involve completely eliminating that country from the supply chain. “Decoupling from China is not simple,” she said. “The biotech ecosystem is really globally integrated.”

    Nonetheless, biotech can be used to strengthen America’s domestic supply chain. One potential tool for achieving this, according to Seagraves, is the Genesis Mission, which was created by an Executive Order and is led by the Department of Energy (DOE) and the 17 DOE national laboratories. In collaboration with industry, academia, and other stakeholders, the Genesis Mission is harnessing AI for breakthroughs in discovery science, energy, and national security. Seagraves said the NSCEB has recommendations that could be implemented through the Genesis Mission initiative that could help improve biotech supply chains when needed, such as in emergencies.

    “Making biotechnology predictably engineerable and solving these kinds of problems at speed and at scale would be really fantastic to help the United States be able to pivot in these types of events, whether it’s from a war or major weather events or something else,” she said. “We’re really hoping at BIO that the Genesis Mission will continue to form partnerships with other departments and agencies around the US government, such as NIH (National Institutes of Health) that have more human health focus.”

    FDA modernization

    China’s competition with America also extends to clinical trials, where the ability to expedite trials is giving China an advantage, Seagraves said. Improvements at FDA would help America in this competition, and it would provide an essential boost to biotech in general, she said.

    “BIO sees modernization of the FDA as absolutely critical to American biotechnology, human health and biotechnology competitiveness,” according to Seagraves.

    She cited a BIO white paper, “Strategies for enhancing FDA as global gold standard,” which highlights three primary recommendation areas:

    • reducing the time and cost of early drug development, including through streamlining clinical trials;
    • strengthening regulatory review to make it more predictable and efficient, “which really ensures that the U.S. system for clinical development remains globally competitive”;
    • supporting new models for external engagement and transparency.

    Seagraves concluded her remarks by agreeing with her fellow panelist about the importance of explaining the needs of biotech to lawmakers.

    “What Garrett just said about the power of biotechnology as a tool, and trying to encourage bio-literacy around thinking of [biotechnology] as a tool, I think, is really important for advancing biotech innovation and the policies that we want to see happen,” she said.

    Read more from BIO:

    The post BIO expert: Future of biotech depends on competing with China, updating FDA appeared first on Bio.News.

    ]]>     Gilead to Acquire Tubulis for Up to $5B, Expanding Cancer ADC Capabilities https://edusehat.com/en/gilead-to-acquire-tubulis-for-up-to-5b-expanding-cancer-adc-capabilities https://edusehat.com/en/gilead-to-acquire-tubulis-for-up-to-5b-expanding-cancer-adc-capabilities Wed, 08 Apr 2026 03:45:12 +0700 Edusehat Gilead, Acquire, Tubulis, for, 5B, Expanding, Cancer, ADC, Capabilities Gilead Sciences has agreed to acquire German-based Tubulis for up to $5 billion, the companies said today, in a deal designed to expand the buyer’s antibody–drug conjugate (ADC) capabilities with a focus on fighting cancer.

    Headquartered in Munich, privately held Tubulis has developed next-generation ADC candidates based on its own conjugation, linker and payload technologies intended to more selectively deliver diverse payloads to tumors deemed to be of high unmet need. The companies said Tubulis’ programs and platforms have broad potential across multiple tumor types, complementing Gilead’s development and commercialization expertise in oncology.

    “We like the strategic fit and deal terms of the Tubulis (private) acquisition,” Daina M. Graybosch, PhD, senior managing director, immuno-oncology and a senior research analyst at Leerink Partners, wrote this morning in a research note. “This is more than an oncology bolt-on; we see real platform value in application of Tubulis’ ADC technologies to other therapeutic areas, namely virology.”

    Tubulis’ lead pipeline candidate, TUB-040, is a sodium-dependent phosphate transport protein 2B (NaPi2b)-targeting topoisomerase-I inhibitor (TOPO1i) ADC that is now under study in the Phase Ib/II NAPISTAR1-01 trial (NCT06303505) assessing its safety, pharmacokinetics, and preliminary efficacy as a treatment for platinum-resistant ovarian cancer and non-small cell lung cancer (NSCLC).

    In October at the European Society for Medical Oncology (ESMO), Graybosch noted, Tubulis presented data for TUB-040 showing a confirmed 50% overall response rate (ORR) and a 60% unconfirmed ORR across dose levels and irrespective of target antigen—results that were competitive with more mature datasets from leading TOPO1i ADCs.

    “Though the dataset was early, and our primary outgoing question was how durability would mature, we suspect that Gilead saw durability maturing positively in their diligence,” Graybosch added. “If TUB-040 proves active in NSCLC, the program could complement their Trodelvy and IO [immune-oncology] lung programs. We wonder if Gilead saw early clinical NSCLC data in their diligence and if excitement around the emerging signal drove some of Tubulis’ valuation.”

    Another Tubulis pipeline candidate, TUB-030, is a 5T4-targeting ADC that according to the companies has shown promising initial clinical data across various solid tumor types. TUB-030 is currently under study in the Phase I/IIa 5-STAR 1-01 trial (NCT06657222), a first-in-human study which aims to evaluate the safety, tolerability, pharmacokinetics, and efficacy of TUB-030 as a monotherapy in patients with advanced solid tumors. Tubulis has said it is developing TUB-030 for up to 13 undisclosed solid tumor indications.

    Partners since 2024

    The acquisition deal follows a two-year, up-to-$465 million collaboration with Tubulis launched in December 2024. Gilead gained access to Tubulis’ Tubutecan and Alco5 platforms after signing an exclusive option and license agreement to discover and develop an ADC against a solid tumor target.

    At the time, Gilead agreed to pay Tubulis $20 million upfront, received an option that if exercised would have given Tubulis an additional $30 million—plus up to $415 million in payments tied to achieving development and commercialization milestones, as well as mid-single to low double-digit tiered royalties on sales of marketed products resulting from the collaboration.

    “Today’s agreement follows a two-year collaboration with Tubulis, which has given us strong conviction in their programs and research capabilities,” Gilead Chairman and CEO Daniel O’Day said in a statement. “The agreement to acquire Tubulis is a significant milestone in Gilead’s progress in oncology. The company brings a clinical-stage candidate that is a potential new treatment for ovarian cancer, as well as a next-generation ADC platform and a promising early pipeline.”

    “Bringing this potential into Gilead would further expand what is already the strongest and most diverse pipeline in our company’s history,” O’Day declared.

    Investors appeared less enthusiastic about the acquisition, as shares of Gilead dipped 1.7% in early Tuesday trading to $137.80 as of 12:01 p.m. ET.

    Tubulis is Gilead’s third announced acquisition this year. The biotech giant announced plans in March to buy Ouro Medicines for up to $2.18 billion, and in February agreed to acquire Arcellx for up to $7.8 billion—for which it agreed last week to extend its tender offer until 5 p.m. ET on April 24.

    Under the acquisition deal, Gilead agreed to acquire all of the outstanding equity of Tubulis for $3.15 billion in upfront cash payable at closing, and up to $1.85 billion in payments tied to milestones.

    The transaction is expected to close in the second quarter subject to expiration or termination of specified regulatory filings and other customary conditions.

    Upon closing of the deal, Tubulis will operate as a dedicated ADC research organization within Gilead, with the Munich site serving as a hub for ADC innovation, building on its integrated discovery, manufacturing, and clinical capabilities to advance next generation ADCs.

    Gilead said it plans to finance the transaction with a combination of cash on hand and senior unsecured notes. Gilead finished 2025 with $10.605 billion of cash, cash equivalents and marketable debt securities, up from $9.991 billion as of December 31, 2024.

    The post Gilead to Acquire Tubulis for Up to $5B, Expanding Cancer ADC Capabilities appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     High-Throughput Platform for Fast-Acting Covalent Protein Therapies https://edusehat.com/en/high-throughput-platform-for-fast-acting-covalent-protein-therapies https://edusehat.com/en/high-throughput-platform-for-fast-acting-covalent-protein-therapies Wed, 08 Apr 2026 03:45:10 +0700 Edusehat High-Throughput, Platform, for, Fast-Acting, Covalent, Protein, Therapies Researchers at Westlake University in China, lead by Bobo Dang, PhD, and Ting Zhou, PhD, report the development of a high-throughput platform for engineering fast-acting covalent protein therapeutics. The team says their study “A high-throughput selection system for fast-acting covalent protein drugs,” published in Science, opens new avenues for next-generation biologics.

    Covalent small-molecule drugs have shown great success in cancer therapy by forming irreversible bonds with their targets. This has inspired efforts to extend covalent strategies to protein therapeutics, especially engineered miniproteins. However, their development is limited by a kinetic mismatch. Miniproteins are rapidly cleared in vivo, while covalent bond formation is typically slow. In addition, high-throughput platforms for systematically optimizing covalent protein reactivity have been lacking.

    To address this challenge, the researchers proposed that precise spatial positioning of chemical warheads within protein scaffolds could enable molecular preorganization, thereby accelerating covalent bond formation without increasing intrinsic reactivity (see figure).

    The principle for developing fast-acting covalent proteins via comprehensive crosslinker and protein sequence engineering. [Bobo Dang's Lab at Westlake University]
    The principle for developing fast-acting covalent proteins via comprehensive crosslinker and protein sequence engineering. [Bobo Dang’s Lab at Westlake University]

    Based on this concept, the team created a high-throughput platform that combines yeast surface display with chemoselective protein modification to screen diverse crosslinkers and millions of protein variants. The platform enables rapid and irreversible target engagement.

    Using this platform, the researchers developed a covalent antagonist targeting PD-L1, termed IB101. Structural analysis revealed that IB101 forms a defined binding pocket that precisely positions the active moiety in a reactive conformation, greatly accelerating covalent bond formation.

    Functionally, IB101 effectively blocks the PD-1/PD-L1 immune checkpoint pathway and demonstrates strong antitumor activity in mouse models. Notably, despite its short in vivo half-life, IB101 achieves durable target engagement and tumor suppression, outperforming conventional antibody-based therapies under comparable conditions, according to the scientists.

    The platform was further applied to cytokine engineering, leading to the development of a covalent IL-18 variant, IB201. This engineered cytokine rapidly forms a covalent interaction with its receptor, enhancing signaling strength and duration. In vivo studies showed that IB201 induces potent antitumor immune responses without detectable systemic toxicity. These results highlight the potential of covalent engineering to improve the efficacy and safety of cytokine-based therapies.

    Beyond immunotherapy targets, the platform was also applied to develop a covalent inhibitor targeting the receptor-binding domain (RBD) of SARS-CoV-2. This molecule showed durable viral neutralization, demonstrating the versatility of the approach across different therapeutic modalities, note the researchers, adding that the study establishes a general strategy for engineering fast-acting covalent protein therapeutics.

    By enabling covalent bond formation on timescales compatible with rapid in vivo clearance, the platform overcomes a fundamental limitation in the field, say the scientists. These findings, they continue, provide a new framework for designing biologics with both rapid kinetics and sustained target engagement, with broad implications for cancer immunotherapy, antiviral therapy, and beyond.

     

     

     

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    ]]>     Neurocrine Grows in Endocrinology, Rare Disease with $2.9B Soleno Buyout https://edusehat.com/en/neurocrine-grows-in-endocrinology-rare-disease-with-29b-soleno-buyout https://edusehat.com/en/neurocrine-grows-in-endocrinology-rare-disease-with-29b-soleno-buyout Wed, 08 Apr 2026 03:45:09 +0700 Edusehat Neurocrine, Grows, Endocrinology, Rare, Disease, with, 2.9B, Soleno, Buyout Neurocrine Biosciences has agreed to acquire Soleno Therapeutics for $2.9 billion, the companies said, in a deal designed to bolster the buyer’s portfolio of marketed endocrinology and rare disease therapies.

    “This transaction will advance Neurocrine’s mission to deliver life-changing treatments while accelerating our revenue growth and portfolio diversification strategy,” Kyle W. Gano, PhD, Neurocrine’s CEO, said in a statement.

    The acquisition would bolster Neurocrine’s offerings to include three treatments that have already reached the market:

    • Crenessity® (crinecerfont), a treatment of classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency that received FDA approval in December 2024
    • Ingrezza® (valbenazine), a vesicular monoamine transmitter 2 (VMAT2) drug approved in 2017 as a treatment for tardive dyskinesia and the chorea associated with Huntington’s disease
    • Vykat™ XR (diazoxide choline), approved last year as the first and only therapy indicated to treat hyperphagia in patients ages four and older with Prader-Willi syndrome (PWS).

    “Neurocrine is the right strategic partner to expand the reach of Vykat XR in the Prader-Willi syndrome community given their experience in endocrinology and rare disease and their proven ability to execute successful commercial launches,” stated Anish Bhatnagar, MD, Soleno’s chairman and CEO. “We are excited to accelerate Vykat XR’s impact for PWS patients following completion of the transaction by leveraging Neurocrine’s strong commercial capabilities.”

    Soleno finished 2025 with $190.4 million in net revenue from sales of Vykat XR—including $91.7 million generated during the fourth quarter, pushing the company to profitability with positive net income of $20.9 million.

    ‘A little surprising’

    Stifel analysts Paul Matteis and James Condulis called the planned acquisition “a little surprising” since Vykat XR is projected to garner approximately $400 million in annual net revenue, he commented in a note reported by Bloomberg News.

    Vykat XR won FDA approval in March 2025. From then through December 31, 859 active patients were prescribed the drug by 630 unique prescribers (136 of them in Q4), while the company received 1,250 patient start forms (207 in Q4).

    Neurocrine expects Vykat XR’s numbers to improve in coming years, since the drug is positioned as a foundational first-line therapy for PWS and is supported by a patent portfolio that is expected to protect the drug’s exclusivity into the mid-2040s.

    Vykat XR would join Neurocrine’s marketed portfolio which includes Ingrezza and Crenessity. Ingrazza racked up blockbuster net revenue numbers of $2.51 billion up 9% year-over-year (including $657.5 million during Q4, up 7% from the year-ago quarter). Neurocrine has credited double-digit prescription volume growth in total prescriptions and new (first-time) prescriptions, partially offset by a lower net price that the company called new “formulary access investments” designed to support long-term growth.

    Crenessity generated $301.2 million in net product sales last year for Neurocrine, including $135.3 million in the fourth quarter, reflecting 2,048 total new patient enrollment start forms, 431 of them in Q4 2025.

    Neurocrine reasons that the three drugs will deliver sustained revenue growth for the combined company through the end of the decade.

    Also for Neurocrine, a buyout of Soleno presents a “more sensible way into metabolic disease” than by developing its own pipeline candidates, which are in preclinical phases, and risking competitive and regulatory challenges, BMO Capital Markets analyst Evan Seigerman observed in a research note reported by Reuters.

    Neurocrine has disclosed plans to begin Phase I studies this year for two preclinical obesity candidates: NBIP-‘2118, a CRF2 agonist; and ‘NBIP-‘1968, a combination of ‘2118 and the company’s own GIP (glucose-dependent insulinotropic polypeptide)/ GLP-1 (glucagon-like peptide-1) preferring triple agonist, which Neurocrine calls “light” on glucagon activity.

    News of a potential buyout of Soleno by Neurocrine was first reported Sunday by the Financial Times.

    Soleno investors signaled approval of the buyout Monday by sending shares to $52.25, up 32% from Thursday’s close of $39.49 (Markets were closed Friday for Good Friday). However, Neurocrine’s investors weren’t as supportive of the deal as that company’s shares barely budged, closing at $132.48, up 0.67% from $131.60 on Thursday.

    Second thoughts?

    A potential reason: Neurocrine investors may have second thoughts about a deal that would add to its pipeline Vykat XR, whose prescribing label includes warnings and precautions about past reports of hyperglycemia and fluid retention/edema, as Sumant Kulkarni, a senior analyst covering biotechnology with Canaccord Genuity, commented in a research note.

    “We believe NBIX would have to articulate its plans very well for investors to display enthusiasm from the get-go,” Kulkarni wrote.

    Yet two things could work in Neurocrine’s favor, Kulkarni added: The company’s solid track record of commercialization as seen with Ingrezza and Crenessity, and the prospect of adding to the portfolio Vykat XR given its approval for a rare form of obesity.

    San Diego-based Neurocrine reported approximately 2,000 employees as of December 31, 2025, with plans during the first quarter to complete the expansion of sales teams for Ingrezza and Crenessity “to maximize our commercial momentum.” Soleno is based in Redwood City, CA, and reported a workforce of 182 full-time employees as of the end of 2025.

    At $53 per share cash, the purchase price represents a premium of about 34% above Soleno’s closing share price Thursday, and a premium of 51% to Soleno’s 30-day volume-weighted average price (VWAP).

    The boards of both Neurocrine and Soleno have approved the transaction, which is expected to close within 90 days subject to satisfying customary closing conditions that include receipt of regulatory approvals.

    Neurocrine will acquire Soleno by launching a tender offer for that company’s outstanding shares. Following a successful completion of the tender offer, a wholly owned subsidiary of Neurocrine will merge with Soleno, and the outstanding Soleno shares not tendered in the offer will be converted into the right to receive the same $53 per share in cash paid in the tender offer.

    Consummation of the tender offer is subject to the tender of at least a majority of the outstanding shares of Soleno, the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and other customary conditions.

    Neurocine said it will fund its acquisition of Soleno using a “modest amount” of pre-payable debt plus cash on hand. Neurocrine reported $1.48 billion in cash, cash equivalents, and marketable securities as of December 31, 2025—up 37.5% from $1.076 billion a year earlier.

    The post Neurocrine Grows in Endocrinology, Rare Disease with $2.9B Soleno Buyout appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Autoimmune Disease-Related Inflammation Reduced with ENDOtollins Drug https://edusehat.com/en/autoimmune-disease-related-inflammation-reduced-with-endotollins-drug https://edusehat.com/en/autoimmune-disease-related-inflammation-reduced-with-endotollins-drug Wed, 08 Apr 2026 00:10:07 +0700 Edusehat Autoimmune, Disease-Related, Inflammation, Reduced, with, ENDOtollins, Drug A new study published in Nature Chemical Biology titled, “Munc13-4–STX7 inhibitors impair endosomal TLR activation and systemic inflammation,” scientists from Scripps Research have developed a new class of drug compounds, called ENDOtollins, that reduce harmful inflammation while maintaining the body’s ability to fight infections. The results offer new directions to treat autoimmune diseases, such as lupus, and rheumatoid and juvenile arthritis, which together affect more than 15 million Americans. 

    “A key component of our approach is to begin by understanding the biological mechanisms at play,” said Sergio Catz, PhD, professor at Scripps Research and corresponding author of the study. “By accomplishing this first, we can more easily target the pathway driving inflammation without affecting other important processes.” 

    Current autoimmune disease treatments, such as hydroxychloroquine, function by broadly blocking endosomes. While effective, this approach can lead to significant side effects, including gastrointestinal problems and, less commonly, vision damage, that cause patients to stop treatment. 

    The authors focused on two proteins, Munc13-4 and syntaxin 7, that bind together to activate Toll-like receptors (TLRs), immune sensors that activate endosomes. This mechanism plays a key role in detecting foreign DNA and RNA from viruses and bacteria. In autoimmune diseases, TLRs become overactive and trigger chronic, damaging inflammation in the absence of a threat. 

    The team screened roughly 32,000 compounds and identified molecules that specifically block the Munc13-4–syntaxin 7 interaction without disrupting other cellular functions. Given that Munc13-4 is found mainly in immune cells, the compounds offer a targeted approach to reduce inflammation. 

    “Most treatments for autoimmune diseases manage symptoms; they don’t change the underlying course of the disease,” said Hugh Rosen, MD, PhD, professor at Scripps Research and co-author of the study. “What’s exciting about this approach is its potential to be disease-modifying: targeting the specific molecular machinery that drives inflammation, rather than broadly suppressing the immune system.” 

    Notably, the study screened compounds in an intact cellular environment which contrasts from many drug screening approaches, which extract proteins from the cell. 

    “By maintaining the proteins in their natural environment, we increase the likelihood that compounds we find will actually work in living cells,” said Jennifer Johnson, PhD, first author and senior staff scientist at Scripps Research. 

    The most potent compound, ENDO12, reduced inflammation in animal models that were also given a TLR-activating molecule. Blood levels of inflammatory markers, including immune system activators IL-6 and IFN-γ, and the enzyme myeloperoxidase, dropped significantly in animals that were treated. 

    ENDO12 treated animals demonstrated normal antiviral immune response when exposed to a virus. This selectivity addresses the concern that dampening inflammation with immunosuppressive drugs may leave patients vulnerable to infections. 

    Looking ahead, the team will test ENDOtollins in models that more closely mimic human autoimmune diseases and evaluate the compounds’ chemistry for potential clinical use. 

    Beyond autoimmune conditions, the researchers suggest ENDOtollins might help treat cytokine storms, the dangerous immune overreactions seen in patients with severe COVID-19 and as a side effect of CAR T cancer therapy. Both involve excessive IL-6 and runaway inflammation. 

    While translating these findings into treatments for patients remains a long-term goal, Catz emphasizes that the mechanistic insights are valuable in their own right. ENDOtollins can serve as precision tools to probe other cellular processes regulated by endosomes and lysosomes, including pathways implicated in neurodegeneration and immune dysfunction.  

    The post Autoimmune Disease-Related Inflammation Reduced with ENDOtollins Drug appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Microplastics in Human Bile Drive Mitochondrial Dysfunction and Senescence https://edusehat.com/en/microplastics-in-human-bile-drive-mitochondrial-dysfunction-and-senescence https://edusehat.com/en/microplastics-in-human-bile-drive-mitochondrial-dysfunction-and-senescence Tue, 07 Apr 2026 09:45:09 +0700 Edusehat Microplastics, Human, Bile, Drive, Mitochondrial, Dysfunction, and, Senescence Microplastics have become a defining environmental signature of modern life, turning up in oceans, soil, food, drinking water, and even the air. But their biological fate inside the human body remains far less understood. A new study suggests that these particles may be doing more than simply passing through. Instead, they may be accumulating in one of the body’s most overlooked fluids—bile—and leaving behind measurable cellular damage that could shape future thinking about environmentally driven biliary injury and long‑term health effects. As the authors noted in their abstract, “the long-term accumulation patterns and chronic toxic effects of microplastics within the human biliary system are largely unknown,” underscoring the need for deeper investigation into how these particles behave in the enterohepatic circulation.

    Researchers from the Tenth Affiliated Hospital of Southern Medical University (Dongguan People’s Hospital), Sun Yat-sen University, Guilin Medical University, and collaborating institutions reported the findings in Environmental Science and Ecotechnology. Their study, “Microplastics accumulate in human bile and drive cholangiocyte senescence,” provides the first direct evidence that microplastics are not only present in bile but may also contribute to mitochondrial dysfunction and premature aging in cholangiocytes, the epithelial cells that line the bile ducts.

    The team collected bile from 14 surgical patients (five without gallstones and nine with gallstones) and used a multimodal analytical approach—pyrolysis–gas chromatography–mass spectrometry, laser direct infrared spectroscopy, and scanning electron microscopy—to characterize the particles. According to the paper, “we show the universal presence of microplastics in human bile,” identifying six polymer types dominated by polyethylene terephthalate and polyethylene, with most particles measuring 20–50 μm. Patients with gallstones carried substantially higher microplastic burdens, raising questions about whether biliary stasis or altered bile composition may influence microplastic retention.

    bile and microplastics study
    This schematic summarizes the study workflow and main findings. Human exposure to microplastics may occur through multiple routes, including industrial pollution, airborne exposure, food packaging, drinking-related plastics, and consumer products. Bile samples collected from individuals with and without gallstones were analyzed using Py-GC/MS, LDIR, and SEM, which confirmed the presence, polymer composition, particle size, and morphology of microplastics in human bile. Mechanistic experiments further showed that nanoplastic exposure induced cholangiocyte senescence by triggering mitochondrial dysfunction, including increased mitochondrial reactive oxygen species, enhanced Drp1-mediated fission, reduced mitochondrial membrane potential, and decreased ATP production, while melatonin partially alleviated these toxic effects. [Environmental Science and Ecotechnology]
    To probe biological effects, the researchers exposed cultured human cholangiocytes to low-dose polystyrene nanoplastics for seven days, simulating chronic exposure. The cells exhibited mitochondrial dysfunction, elevated reactive oxygen species, reduced ATP, Drp1‑mediated mitochondrial fission, and G1 cell‑cycle arrest—hallmarks of senescence. As the authors wrote, chronic exposure “induces mitochondrial dysfunction-associated senescence in cholangiocytes,” suggesting a mechanistic link between environmental microplastics and biliary aging.

    One of the most intriguing findings is that melatonin, a widely used antioxidant, partially reversed the mitochondrial and inflammatory damage. While far from a therapeutic recommendation, the result hints at a potential intervention point and gives the study translational relevance.

    The work reframes the biliary system as something far more active than a simple transit channel. The data indicate that bile can serve as a reservoir for microplastics and that prolonged exposure may age cholangiocytes by driving mitochondrial dysfunction. The partial rescue with melatonin adds a mechanistic foothold for future intervention, even as the authors caution that broader human studies are essential.

    For biotech, the implications are broad. The work highlights bile as a clinically accessible matrix for exposure assessment, opening the door to new diagnostics for environmental toxicology. The mitochondrial stress signature aligns with pathways already being targeted by companies developing senolytics, mitoprotective agents, and anti‑inflammatory therapeutics. The authors wrote that the research provides “a mechanistic foundation for assessing the health risks of plastic pollution and developing therapeutic interventions for environmentally driven biliary disorders.”

    The post Microplastics in Human Bile Drive Mitochondrial Dysfunction and Senescence appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Low-Cost, Single Sample Blood Test Detects Different Cancers, Liver Disorders, and Other Diseases https://edusehat.com/en/low-cost-single-sample-blood-test-detects-different-cancers-liver-disorders-and-other-diseases https://edusehat.com/en/low-cost-single-sample-blood-test-detects-different-cancers-liver-disorders-and-other-diseases Tue, 07 Apr 2026 06:10:14 +0700 Edusehat Low-Cost, Single, Sample, Blood, Test, Detects, Different, Cancers, Liver, Disorders, and, Other, Diseases UCLA scientists have developed a simple and cost-effective blood test that, in early studies in more than 1,000 people, showed promise in detecting multiple cancers, various liver conditions, and organ abnormalities simultaneously.

    The new method, called MethylScan, works by analyzing cell-free DNA (cfDNA), tiny fragments of genetic material released into the blood when cells die. Because cells from every organ shed DNA into the bloodstream, cfDNA carries molecular signals that reflect what is happening throughout the body.

    The researchers say MethylScan could represent a powerful and more affordable approach to early disease detection and comprehensive health monitoring. “Early detection is crucial,” said research lead Jasmine Zhou, PhD, a professor of pathology and laboratory medicine and investigator at the UCLA Health Jonsson Comprehensive Cancer Center. “Survival rates are far higher when cancers are caught before they spread. If you detect cancer at stage one, outcomes are dramatically better than at stage four.” Zhou is senior author of the team’s published paper in PNAS, titled “Toward the simultaneous detection of multiple diseases with a highly cost-effective cell-free DNA methylome test.”

    “When cells die, they do not simply vanish; they leave behind molecular traces, including cell-free DNA (cfDNA) in the blood stream,” the authors wrote. “cfDNA is a mixture of DNA fragments released from various organs, offering valuable insights into the health of these organs.” Zhou added, “Every day, 50 to 70 billion cells in our body die. They don’t just disappear, their DNA goes into the bloodstream. That means we already have information from all our organs circulating in the blood.”

    The idea of using blood to detect cancer, sometimes called a liquid biopsy, isn’t new. Some tests already look for mutations in tumor DNA to screen for certain cancers. But those tests often focus on a limited number of genetic changes and can be expensive, in part because they require deep sequencing to detect faint tumor signals.

    Instead of searching for mutations, the UCLA team examined DNA methylation, chemical tags attached to DNA that help regulate gene activity. Methylation patterns differ by tissue type and can change when cells become cancerous or diseased. “Unlike an individual’s genome, which remains largely stable across tissues and over time (except for rare somatic mutations), the DNA methylome is tissue-specific and dynamically changes with the tissue’s disease status,” the team continued.

    “DNA methylation reflects the health status of a tissue,” said co-corresponding author Wenyuan Li, PhD, a professor of pathology and laboratory medicine at UCLA and co-corresponding author of the study. “It’s a very informative signal.”

    The challenge is that most cell-free DNA in the bloodstream doesn’t come from tumors or injured organs. About 80% to 90% originates from normal blood cells. That creates background noise, making it difficult and costly to detect the relatively rare fragments that might signal early cancer. “A major challenge in using the cfDNA methylome for disease detection is the high cost of sequencing,” the team stated. “In healthy individuals, about 85% of cfDNA originates from blood cells, creating substantial background noise that can obscure cfDNA from tumors or diseased organs.” And as the authors further pointed out, “Current cfDNA methylation assays primarily focus on single clinical indications by targeting specific genomic loci.”

    For their newly reported research the team built on past work to develop a technique to remove much of the background DNA before sequencing. Using specialized enzymes, they selectively cut away unmethylated DNA fragments that largely come from blood cells. By designing a genome-wide hybridization panel, the captured DNA fragments are enriched for methylated DNA from solid organs, including those that are potentially diseased. “The MethylScan method is a targeted methylation assay that combines Methylation-Sensitive Restriction Enzymes (MSRE) digestion with a custom panel to enrich hypermethylated cfDNA from tissues beyond blood, enabling cost-effective detection of multiple diseases,” they wrote.

    By removing the noise, the researchers say they can dramatically reduce the amount of sequencing needed, lowering costs while maintaining sensitivity. Achieving an effective sequencing depth of 300× per sample requires only 5 Gb of data, which would cost less than $20 if the price per gigabase is under $4.

    To test the accuracy of MethylScan, the researchers analyzed blood samples from 1,061 people, including patients with liver, lung, ovarian and stomach cancers; individuals with liver diseases such as hepatitis B, hepatitis C, alcohol-related liver disease, and metabolic-associated liver disease; people with benign lung nodules; and healthy participants. Machine learning algorithms were then applied to analyze the complex methylation data.

    For multi-cancer detection, the test achieved a high level of overall accuracy. At a specificity of 98%, meaning few false positives, it detected about 63% of cancers across all stages and roughly 55% of early-stage cancers. The test also performed well in liver cancer surveillance among high-risk individuals, including those with liver cirrhosis or HBV, detecting nearly 80% of cases at a specificity of just over 90%, meaning a less than 10% false positive rate.

    Beyond simply detecting cancer, the methylation patterns helped identify where in the body a signal was coming from, known as the tissue of origin. “Being able to trace signals back to their source is important because a positive blood test needs to be followed by imaging or other diagnostic procedures directed at the right organ,” said Li.

    MethylScan can work like a health radar for the body. By reading DNA signals in the blood, it can tell when specific organs, such as the liver or lungs, are under stress or damaged, even without knowing the disease in advance. The researchers also showed that the blood test could distinguish between different types of liver disease, including viral hepatitis and metabolic-associated liver disease. It correctly classified about 85% of patients, suggesting blood-based DNA testing could reduce the need for invasive liver biopsies.

    Although larger prospective trials will be needed to confirm its performance in real-world screening, Zhou said the work represents an important step toward a single, affordable blood assay that can detect a broad spectrum of diseases earlier and more comprehensively than current methods allow.

    “Because cfDNA in blood originates from multiple organs, and ‍MethylScan captures a broad spectrum of robust hypermethylation markers, this assay has the potential to detect a variety of diseases, provided that appropriate training cohorts are available,” the investigators stated. “This versatile approach enables affordable, wide-ranging cfDNA tests that can identify various health conditions simultaneously, with the potential to transform early disease detection and health monitoring across diverse clinical settings.

    Zhou added, “This study demonstrates that blood-based methylation profiling can deliver clinically meaningful information across multiple diseases. It’s an exciting advancement that brings us closer to realizing the dream of a single assay for universal disease detection.”

    The post Low-Cost, Single Sample Blood Test Detects Different Cancers, Liver Disorders, and Other Diseases appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Immunotherapy Enhanced by Restoring Mitochondrial Function in Dendritic Cells https://edusehat.com/en/immunotherapy-enhanced-by-restoring-mitochondrial-function-in-dendritic-cells https://edusehat.com/en/immunotherapy-enhanced-by-restoring-mitochondrial-function-in-dendritic-cells Tue, 07 Apr 2026 06:10:13 +0700 Edusehat Immunotherapy, Enhanced, Restoring, Mitochondrial, Function, Dendritic, Cells In a new study published in Science titled, “Mitochondrial metabolism and signaling direct dendritic cell function in antitumor immunity,” researchers from St. Jude Children’s Research Hospital have uncovered a new metabolic mechanism for how tumors disable immune “gatekeeper” cells that initiate response in the presence of cancer. The results offer a new path to improve immunotherapy.

    Dendritic cells alert and activate tumor-killing immune cells as a critical part of anticancer immune response. The authors found that tumors reduce dendritic cell function by minimizing mitochondrial fitness to prevent anticancer immune response. Correspondingly, boosting mitochondrial function in dendritic cells enhances antitumor immune activity and strengthens the efficacy of existing immunotherapies.  

    Within the nutrient-sparse tumor microenvironment, dendritic cells progressively lose mitochondrial activity, which drives cell dysfunction and weakens immune defenses against cancer. When dendritic cells with high mitochondrial activity were introduced into tumors in preclinical mouse models, results showed that immunogenic activity was restored while improving tumor control.  

    “We found that tumors reprogram mitochondrial metabolism in dendritic cells, reducing their ability to activate the immune system against cancer,” said Hongbo Chi, PhD, St. Jude Department of Immunology chair and corresponding author of the study. “By enhancing mitochondrial function, we could restore dendritic cell activity and rescue antitumor immunity.”  

    Immunotherapies for cancer, such as immune checkpoint blockade, have greatly improved care for many malignancies, but have not been successful in all cancers. To determine whether these findings could improve immunotherapy effectiveness in tumor-bearing mice, the authors evaluated the administered dendritic cells with high mitochondrial activity in combination with immune checkpoint blockade. 

    “We saw the most pronounced therapeutic effect in mice treated with the combination of dendritic cells that had high mitochondrial activity and immune checkpoint blockade,” said co-first author Zhiyuan You, PhD, researcher at St. Jude Department of Immunology. “Those combinations synergistically slowed or stopped tumor growth and extended survival far more than either treatment alone.”  

    To test long-term effects, the researchers exposed combination therapy treated mice to a new tumor after a few months. New tumor growth stopped for these mice, indicating durable, long-term immune memory. 

    To better understand the relationship between mitochondrial function and dendritic cells, the researchers examined metabolic pathways affected by the tumor microenvironment. They identified a signaling axis composed of two proteins, OPA1 and NRF1, that regulate communication between mitochondria and the nucleus. Expression was greatly downregulated in dendritic cells during tumor progression and acted as a metabolic switch to shut down dendritic cell immunogenic activity.  

    “We’re seeing a direct regulation of dendritic cells by the tumor microenvironment,” said co-first author Jiyeon Kim, PhD, researcher at St. Jude Department of Immunology. “We have characterized how that results in mitochondrial reprogramming of dendritic cells to benefit cancer, giving us new opportunities to reverse the process.”  

    The study’s mechanistic insights enable new directions to rewire dendritic cell function and enhance cancer treatments.  

    “These findings reinforce the central role of dendritic cells in cancer immunity,” Chi said. “By exploring their mitochondrial function in the tumor microenvironment, we have provided a proof-of-principle of how we may be able to improve the next generation of immunotherapies.” 

    The post Immunotherapy Enhanced by Restoring Mitochondrial Function in Dendritic Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     CRISPR at 25: The Past, Present, and Future of Genome Editing https://edusehat.com/en/crispr-at-25-the-past-present-and-future-of-genome-editing https://edusehat.com/en/crispr-at-25-the-past-present-and-future-of-genome-editing Tue, 07 Apr 2026 06:10:12 +0700 Edusehat CRISPR, 25:, The, Past, Present, and, Future, Genome, Editing

    Panelists:

    Image of Rodolphe Barrangou, PhD

    Rodolphe Barrangou, PhD

    North Carolina State University

    Panelist

    Image of Rodolphe Barrangou, PhD

    Rodolphe Barrangou, PhD

    Rodolphe Barrangou, PhD, is the T. R. Klaenhammer Distinguished Professor at North Carolina State University, where he leads the CRISPR Lab. Rodolphe spent nine years at Danisco and DuPont, where he made seminal contributions in the functional characterization of CRISPR as a microbial immune system. He has been at NC State since 2013.

    For his CRISPR work, Rodolphe has received several international awards, notably the Canada Gairdner International Award, and has been elected to the National Academy of Sciences, the National Academy of Engineering, and the National Academy of Inventors. Rodolphe is a scientific co-founder of Intellia Therapeutics, Locus Biosciences, TreeCo, Ancilia Biosciences, and CRISPR Biotechnologies, and an advisor to Inari and the IGI. He is also the founding Editor in Chief of The CRISPR Journal (published by Mary Ann Liebert, Inc., a Sage partner), which launched in 2018.

    Rodolphe holds a degree from Paris Descartes University and a PhD in functional genomics from NC State.

    Broadcast Date: 
    • Time: 

    It has been almost 25 years since the acronym “CRISPR” was first coined. Since then, CRISPR has become a household word, a star of books and films, and a Nobel Prize–winning discovery. This powerful and disruptive genome editing technology has transformed countless fields, including gene therapy, xenotransplantation, de-extinction and agbiotech. Researchers continue to build on the CRISPR chassis, devising new platforms for bespoke genome editing. But major questions remain around clinical safety, commercial development, ethical deployment, and regulatory oversight.

    In the first of a new series of GEN Keynote Webinars, Professor Rodolphe Barrangou, PhD (North Carolina State; EIC, The CRISPR Journal) offers a front-row perspective of the CRISPR revolution, the seminal advances, clinical highlights, and rising applications. Almost two decades ago, Barrangou provided the first experimental demonstration of the functional role of CRISPR. With numerous advisory and entrepreneurial activities in the gene editing space, Barrangou is the ideal guide to discuss CRISPR’s progress in the clinic; the state of the CRISPR toolbox; and the regulatory roadblocks and ethical challenges that will shape the application of CRISPR in agbiotech, germline editing, and other arenas.

    Registration for this GEN Keynote Webinar is free. Following this live presentation, Dr. Barrangou will answer audience questions.

    Produced with support from:

    Elevate bio logo

    The post CRISPR at 25: The Past, Present, and Future of Genome Editing appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Machine Learning and Single-Cell Technology Combined to Drive High-Performance Cell Line Development https://edusehat.com/en/machine-learning-and-single-cell-technology-combined-to-drive-high-performance-cell-line-development https://edusehat.com/en/machine-learning-and-single-cell-technology-combined-to-drive-high-performance-cell-line-development Tue, 07 Apr 2026 02:35:10 +0700 Edusehat Machine, Learning, and, Single-Cell, Technology, Combined, Drive, High-Performance, Cell, Line, Development OneCyte, which focuses on high-throughput single-cell analysis and cell line development technologies, and Kemp Proteins, which specializes in protein engineering and expression solutions, signed a strategic partnership agreement to deliver cell line development services for biopharmaceutical companies.

    The collaboration brings together OneCyte’s proprietary single-cell platform for high-throughput and high-speed clone selection with Kemp Proteins’ molecular engineering capabilities, including its machine learning–driven platform, PROTiQ™.

    Biopharma companies continue to face significant challenges in cell line development, including long development cycles, suboptimal yields, and high failure rates, particularly for novel and complex molecules, according to Konstantinos Tsioris, PhD, co-founder and president of OneCyte. These challenges can delay regulatory timelines and slow the progression of therapies into the clinic.

    The OneCyte-Kemp partnership addresses these pain points by integrating predictive in silico design with rapid and high throughput experimental validation, say officials at both companies. As part of the workflow, amino acid sequences are evaluated using Kemp’s PROTiQ platform to assess developability risks, identify sequence liabilities, and generate structural insights.

    The optimized candidates are then paired with OneCyte’s high-performance cell line development platform, which reportedly enables identification of elite clones with higher productivity.

    Unlike traditional, rigid development workflows, this integrated approach is designed to adapt quickly to the evolving needs of new therapeutic modalities, notes Tsioris.

    “By combining our single-cell technology with Kemp’s deep expertise in protein expression, we are confident that we can address the hardest challenges associated with new modalities, delivering faster timelines and industry-leading titers,” he continues.

    “OneCyte’s class-leading single-cell technology, stacked on top of our molecular design and expression capabilities, will provide a powerful and differentiated solution for our global biopharma customers,” says Michael Keefe, CEO of Kemp Proteins.

    The post Machine Learning and Single-Cell Technology Combined to Drive High-Performance Cell Line Development appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     BIO’s comments for USTR report highlight global threats to intellectual property https://edusehat.com/en/bios-comments-for-ustr-report-highlight-global-threats-to-intellectual-property https://edusehat.com/en/bios-comments-for-ustr-report-highlight-global-threats-to-intellectual-property Mon, 06 Apr 2026 23:05:04 +0700 Edusehat BIO’s, comments, for, USTR, report, highlight, global, threats, intellectual, property Strong intellectual property protections sustain biotechnology innovation by enabling the investment needed to develop new medical breakthroughs.

    In comments submitted for the 2026 U.S. Trade Representative (USTR) Special 301 Report, the Biotechnology Innovation Organization (BIO) underscores the need to strengthen and enforce IP protections globally. In markets where protections fall short, biotech companies face significant barriers to entry.

    “This directly harms their ability to innovate, invest, and create jobs in the United States,” according to BIO’s comments. “BIO, therefore, strongly urges USTR to take appropriate and proportionate actions including enforcement of existing trade agreements and U.S. trade laws to efficiently remedy IP issues with key trading partners.”

    The Special 301 report, produced annually by the USTR, provides a review of IP protection and enforcement worldwide. “The United States uses the review and resulting Report to focus our engagement on these issues,” in discussions with trading partners, according to the USTR.

    The report is typically released around the end of April, and BIO submitted its comments to inform the 2026 report earlier this year.

    Why biotech and IP matter

    BIO’s comments explain the importance of biotech to the U.S. economy.

    “According to the TEConomy/CSBA/BIO 2024 Report on the Bioscience Economy, the U.S. bioscience industry directly employs nearly 2.29 million people across nearly 150,000 U.S. business establishments,” BIO says. “Our sector’s economic impact on the U.S. economy totaled $3.2 trillion in 2023, as measured by overall output.”

    Of course, biotech advances have an impact beyond the economy. New treatments help us live longer, healthier lives. But we need market incentives to fund these treatments, BIO explains.

    “Without strong and predictable patent protection, investors will shy away from investing in biotech innovation and will simply put their money into projects or products that are less risky—without regard to the great value that biotechnology offers society,” BIO’s comments say.

    BIO cites a University of California Berkeley survey in which 73% of the biotechnology entrepreneurs reported that potential funders, “indicated patents were one of the most important factors in their investment decisions.”

    Given that many of BIO’s members are small and medium-sized enterprises with no products on the market, they “rely heavily on the strength and scope of their IP to generate investments needed to develop and commercialize their technologies,” BiO says. These businesses need global IP protections to be able to expand beyond American markets.

    Actions that weaken IP globally

    The comments note that some U.S. trade partners employ various practices that interfere with the IP protections biotech needs.

    For instance, drugmakers are often pressured to give up their licensing rights and localize their technology in exchange for access to a country’s market.

    “Efforts by governments and activists, including in Colombia, Russia, and Brazil, to normalize the use of routine, non-emergency compulsory licenses undermine the legal and economic property rights framework that enables the world-leading U.S. investment in biopharmaceutical technology innovation,” BIO says.

    There have also been efforts taken within multilateral institutions, such as the World Trade Organization and the World Health Organization, that treat IP as a barrier to spreading innovation, according to BIO.

    It is essential that USTR oppose these attempts to weaken IP, BIO says.

    “Accordingly, we strongly encourage USTR to play an active role in IP discussions with key trading partners and within multilateral fora to defend U.S. interests by protecting the IP of U.S. companies,” BIO says.

    The need to protect trade secrets

    BIO’s comments put a particular focus on the challenge of protecting trade secrets. In some countries, inadequate legal protections enable commercial competitors to obtain trade secrets from regulators or other sources. In other cases, governments make concerted efforts to gain access to trade secrets, even from third countries.

    “Economic espionage targeting the U.S. biopharmaceutical sector has become a strategic priority for countries such as China and Russia,” the comments explain. “These countries leverage their economic, political, and intelligence relationships with many countries around the world, to gain advantages that include acquisition of U.S. intellectual property.”

    Any firm operating directly within China and Russia faces further threats to their trade secrets.

    “China’s Biosecurity Law and Human Genetics Regulation require foreign firms to partner with Chinese entities and share records, data, and other information as part of doing business in China,” BIO says. Russia has decreed that Russian businesses may freely use Western intellectual property “with the aim of reducing Russian reliance on U.S. companies and further countering the effects of sanctions.”

    To protect trade secrets and other IP, BIO recommends that the USTR:

    • pursue strong trade secret commitments in bilateral and multilateral trade agreements;
    • develop coordinated responses with allies to address nation state-sponsored trade secret theft;
    • enhance information sharing between government and industry regarding specific threats to biopharmaceutical intellectual property.

    “BIO urges USTR to prioritize these issues in its engagement with trading partners, recognizing that the protection of trade secrets is fundamental to maintaining U.S. leadership in biopharmaceutical innovation and ensuring continued investment in developing life-saving medicines for patients worldwide,” BIO concludes.

    Read BIO’s comments for the Special 301 Report here.

    The post BIO’s comments for USTR report highlight global threats to intellectual property appeared first on Bio.News.

    ]]>     Nanotube Injector Boosts Mitochondrial Performance Through Cytoplasmic Transfer https://edusehat.com/en/nanotube-injector-boosts-mitochondrial-performance-through-cytoplasmic-transfer https://edusehat.com/en/nanotube-injector-boosts-mitochondrial-performance-through-cytoplasmic-transfer Mon, 06 Apr 2026 23:00:12 +0700 Edusehat Nanotube, Injector, Boosts, Mitochondrial, Performance, Through, Cytoplasmic, Transfer Extracting cytoplasmic material such as proteins, RNA, and mitochondria often relies on cell lysis using detergents or enzymes, which destroy the cells. Ultrasound and other sophisticated physical disruption methods need to be carefully tuned to avoid damaging biomolecules, potentially rendering them too time-consuming.

    Delivering material into cells presents further challenges. Lipid-based carriers are limited to small molecules, viral vectors are costly, and microinjection techniques are difficult to scale. To date, no approach allows for controlled and efficient cytoplasmic transfer without compromising cell viability, according to researchers from Waseda University in Japan.

    The team published a study “A Nanotube Injector for Cytoplasmic Transfer and Enhanced Mitochondrial Function” in Small Science that reports the development of a nanotube membrane-based injector—a platform that combines nanomaterials and fluid physics to directly transfer cytoplasmic contents between cell populations. The system consists of a thin gold membrane with vertically aligned nanotubes mounted on a glass tube. When this membrane is carefully pressed against cultured cells, the nanotubes penetrate the phospholipid bilayer of the living cells without causing significant damage. By adjusting the internal air pressure of the glass tube, the researchers can “suck up” cytoplasmic material from the source cells, hold it as the tube is repositioned over the target cell culture, and gently flush it into this new population using microliters of a buffer solution.

    Infographic from Waseda University in Japan
    Credit: Waseda University

    Through several experiments using fluorescent dyes and protein assays, the researchers say they confirmed that cytoplasmic contents could be extracted in a pressure-dependent manner. They also found that careful selection of nanotube diameter, nanotube density, and applied pressure was key to minimizing cellular damage. Notably, under optimized conditions, cell viability hovered around 95%, with a cytoplasmic transfer efficiency of well over 90%, note the scientists.

    To further test the capabilities of their platform, the team investigated whether it could transfer intact mitochondria. To this end, they labeled mitochondria in donor cells with a fluorescent tag and observed them in the recipient cells via confocal microscopy. They found that dozens of mitochondria could be reliably delivered per cell.

    Most importantly, according to Takeo Miyake, PhD, team leader, these mitochondria remained functional, as evidenced by markedly higher levels of adenosine triphosphate (ATP) produced in recipient cells compared to controls.

    “This technology establishes a new paradigm for cell manipulation—transforming cells not by genetic modification but by reconstructing intracellular composition itself,” explains Miyake, adding that such controlled cytoplasmic engineering, enabled by the proposed nanotube injector, could support the development of next-generation cell therapies, improved disease models, and more precise drug screening platforms.

    “Directly transferring healthy mitochondria or cytoplasmic components into target cells is particularly relevant for regenerative medicine, where therapeutic cells often suffer from reduced metabolic activity or functional heterogeneity after isolation and expansion,” highlights Miyake, “By restoring or augmenting mitochondrial function without genetic modification, the technology offers a new strategy to improve cell quality prior to transplantation.”

    Overall, this innovative system paves the way for a new level of control in cell biology research, as well as bioengineering and biomedical applications, points out the research team.

    The post Nanotube Injector Boosts Mitochondrial Performance Through Cytoplasmic Transfer appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     StockWatch: Price War Dampens Lilly Surge After Oral GLP-1 Wins FDA Nod https://edusehat.com/en/stockwatch-price-war-dampens-lilly-surge-after-oral-glp-1-wins-fda-nod https://edusehat.com/en/stockwatch-price-war-dampens-lilly-surge-after-oral-glp-1-wins-fda-nod Mon, 06 Apr 2026 12:05:10 +0700 Edusehat StockWatch:, Price, War, Dampens, Lilly, Surge, After, Oral, GLP-1, Wins, FDA, Nod Eli Lilly (NYSE: LLY) won the approval it sought when the FDA authorized the company’s oral obesity drug Foundayo™ (orforglipron), but the pharma giant’s post-approval stock bounce was short-lived.

    Lilly shares rose 4% from $919.77 to $954.52 on the day of the announcement. But the momentum reversed into a 2% loss to $935.58 on Thursday, and for one important reason beyond simply the overall market decline triggered by investors losing confidence in a speedy end to the Iran war.

    Investors (as reflected in analysts’ mixed observations) appeared divided on how quickly Lilly can generate sales this year for Foundayo, a small molecule glucagon-like peptide-1 (GLP-1) receptor agonist. That division results from the competition shaping up on the drug’s price with obesity arch-rival Novo Nordisk (shares traded on Nasdaq Copenhagen as NOVO-B; ADRs traded on NYSE as NVO).

    Novo Nordisk got a jump on Lilly in the oral obesity drug front in December when the Danish biotech giant won FDA approval for oral Wegovy® (semaglutide), a once-daily 25 mg tablet indicated for chronic weight management. Analysts consider oral Wegovy sales to have started strong, with total prescriptions reaching 577,000 and 52,000 filled during the week ending March 20.

    Lilly is expected to make Foundayo available directly to patients through its LillyDirect direct-to-consumer services and support platform starting Monday. There, a starting dose of 0.8 mg is being priced at $149/month, rising to $199/month for 2.5 mg, $299/month for the 5.5 mg and 9 mg doses, and up to $349/month for the highest doses of 14.5 mg and 17.2 mg. However, high-dose patients will automatically receive the $299/month price on their first purchases and keep it if they refill their prescription within 45 days of their previous prescription.

    Patients paying through commercial insurance plans will be eligible for discounts that reduce Foundayo’s out-of-pocket cost to patients for 1-, 2-, or 3-month prescription fills to $5 a month.

    “A positive surprise is that the approval was for a tablet formulation, which is less expensive to manufacture” than the capsule versions studied by Lilly during clinical trials, David Risinger, a senior managing director and senior research analyst covering diversified biopharmaceuticals at Leerink Partners, shared in a research note.

    Lilly told Risinger that it conducted a bioequivalence study comparing capsules to tablets, which, according to the company, can be manufactured more efficiently than capsules and use less active pharmaceutical ingredient. The high dose studied in Lilly’s Phase III trials of orforglipron, 36 mg, corresponds to the highest capsule dose of 17.2 mg.

    Price competition

    Lilly has sought to price its oral obesity drug competitively with Novo Nordisk’s oral Wegovy, which starts at $149/month for the lowest dose of 1.5 mg, then rises to $199/month for the 4 mg dose, with new patients paying $149/month through August 31. Prices rise to $299/month for 9 mg and 25 mg doses.

    However, patients who sign up for a 12-month subscription to oral Wegovy through Novo Nordisk’s telemedicine partner-providers enjoy a $50 discount that brings their monthly cost down to $249. And commercial insurance patients who agree to local pharmacy pickup with a savings offer can pay as little as $25/month, subject to a maximum savings of $100/month.

    The price competition explains why buyers of securities for themselves or clients—the “buy side” in Wall Street jargon—have lowered their 2026 forecasts for Foundayo sales by more than half, from about $4 billion to less than $2 billion, Trung Huynh, an analyst with RBC Capital Markets, wrote in a research note. Huynh cited a consensus of analysts which is projecting approximately $1.6 billion in 2026 sales, though a Reuters spot check of investment brokerages found a range for this year’s projected sales of $1.5 billion to $2.8 billion.

    “Although there have been headwinds on pricing erosion in the GLP-1 space, we believe there is substantial upside with the expected Medicare Part D expansion later this year,” Huynh added. The expansion of sales to the Medicare Part D program would cap patient copays at $50 per month.

    Huynh and RBC Capital have projected Foundayo will reach peak-year sales of $36 billion—14% above the $31.68 billion racked up by last year’s best-selling prescription drug, the multi-indication cancer immunotherapy blockbuster Keytruda® (pembrolizumab) marketed by Merck & Co. (NYSE: MRK).

    The highest peak sales forecast comes from Citi Research, where Geoff Meacham, PhD, the firm’s head of healthcare research and a managing director specializing in U.S. pharma and biotech research, has projected more than $40 billion. At the low end, a consensus of analysts surveyed by Bloomberg News expects Foundayo peak sales to reach $18 billion by 2030.

    $50M upfront

    Both ends are a far, far cry from the $50 million upfront that Lilly paid in 2018 to license orforglipron, then called OWL833, from Chugai Pharmaceutical, which discovered the drug and is owned by Roche Holding (SIX Swiss Exchange: ROP and RO; OTCQX: RHHBY). Lilly also agreed to pay Chugai up to $390 million in potential payments tied to achieving milestones.

    At the time, OWL833 was deemed Phase I ready for clinical studies in type 2 diabetes—the indication for which Lilly markets its GLP-1/GIP (glucose-dependent insulinotropic polypeptide) receptor agonist tirzepatide as Mounjaro®, and Novo Nordisk markets its GLP-1 receptor agonist semaglutide as Ozempic®.

    “Overall, we continue to believe that injectable anti-obesity medications will retain the majority of market share (roughly 80%, based on our estimates) in the United States due to the high potency of injectables coupled with a more elevated U.S. BMI population,” Andy T. Hsieh, PhD, a partner and biotechnology analyst with William Blair, wrote in a research note.

    Beyond competitive pricing and lower manufacturing costs, Lilly has emphasized a convenience advantage over oral Wegovy—starting with its public announcement of the approval, whose headline referred to Foundayo as “the only GLP-1 pill for weight loss that can be taken any time of day without food or water restrictions.”

    While Foundayo can be taken morning, afternoon, or evening, oral Wegovy, by contrast, requires patients to take the pill with up to 4 ounces of water on an empty stomach as soon as they wake up, then fast for 30 minutes before they can eat or drink.

    “Accordingly, we expect Foundayo to blunt the uptake of oral Wegovy upon its availability (starting on April 6), though oral Wegovy retains a pricing advantage,” Hsieh concluded.

    Huynh of RBC Capital agreed, citing a survey by his firm of about 200 patients, payers, and prescribers: “Our recent survey indicated that Foundayo would be a preferred oral option amongst patients since it has no dosing restrictions.”

    Not so, Novo Nordisk CEO Maziar (Mike) Doustdar told CNBC last month: “People are really interested because it’s the most efficacious pill right now in the market.”

    Novo Nordisk cites efficacy

    Novo Nordisk sought to reinforce that message via an announcement trumpeting that its 25 mg dose of oral Wegovy showed “significantly” greater mean weight loss than the 36 mg dose of Foundayo, according to a population-adjusted indirect treatment comparison using data from two clinical trials:

    • OASIS 4 (NCT05564117), a 307-patient randomized study which evaluated once-daily oral Wegovy 25 mg in overweight or obese adults with at least one self-reported unsuccessful dietary effort to lose body weight, to measure their percentage change in body weight and whether it was ≥5% at the end of treatment at week 64.
    • ATTAIN-1 (NCT05869903), a 72-week, randomized, double-blind, placebo-controlled trial comparing the efficacy and safety of orforglipron (6 mg, 12 mg, and 36 mg) as monotherapy vs. placebo in 3,127 adults with obesity, or overweight with at least one of the following comorbidities: hypertension, dyslipidemia, OSA, or cardiovascular disease, who did not have diabetes.

    Novo Nordisk plans to present details of its study, called ORION, at the Obesity Medicine Association’s annual Obesity Medicine 2026 conference, set for April 10-12 in San Diego.

    News of the FDA approval for Foundayo caused Novo Nordisk shares to dip 0.1% Wednesday from DKK 231.15 ($35.65) to DKK 230.90 ($35.61). But the shares finished the trading week climbing nearly 3% Thursday to DKK 236.90 ($36.53).

    U.S. and major European markets were closed on Friday in observance of Good Friday.

    Foundayo is the fifth prescription drug to be authorized under the FDA’s Commissioner’s National Priority Voucher program, through which the agency awards vouchers to drug developers whose work will address a health crisis in the United States, deliver more innovative cures, address unmet public health needs, and increase domestic drug manufacturing as a national security issue.

    The approval came nearly 10 months ahead of Foundayo’s target decision date of January 20, 2027, under the PDUFA (Prescription Drug User Fee Act) program, and just 50 days after Lilly filed a new drug application (NDA) for the oral obesity drug with the FDA.

    Leaders and laggards

    • Inovio Pharmaceuticals (NASDAQ: INO) shares nosedived 35% from $1.74 to $1.13 Thursday after the developer of DNA therapies to treat and prevent HPV-related diseases, cancer, and infectious diseases announced that it priced at $1.40 a share an underwritten public offering of 12.5 million shares of common stock, plus accompanying Series A and Series B warrants, each series enabling potential purchase of up to 12.5 million shares (or pre-funded warrants in lieu thereof). The dilutive offering is projected to raise $17.5 million in gross proceeds—$16 million in net proceeds, rising to $18.4 million if underwriter Piper Sandler exercises in full its 30-day option to purchase up to 1.875 million additional shares and all Series A and Series B warrants (1.875 million shares each series). Net proceeds are intended to fund a potential commercial launch of INO-3107, a recurrent respiratory papillomatosis candidate under FDA review with a target decision date of October 30, as well as a confirmatory trial for INO-3107 if required, pipeline development, and general corporate purposes.
    • Oric Pharmaceuticals (NASDAQ: ORIC) shares tumbled 19% from $12.67 to $7.47 Wednesday after the cancer drug developer said it will advance rinzimetostat (ORIC-944) into a 600-patient Phase III trial (Himalaya-1 ) after generating positive data from its Phase Ib trial (NCT05413421) assessing rinzimetostat plus the Bayer (XETRA: BAYN)-marketed Nubeqa® (darolutamide) as a once-daily treatment for metastatic castration-resistant prostate cancer (mCRPC) in patients previously treated with abiraterone acetate (abiraterone). At a median follow-up of 4.9 months, the combination showed radiographic progression-free survival (rPFS) rates of 93% at 3 months, 84% at 4 months, and 84% at 5 months—rates consistent, Oric said, with the competitor PRC2 inhibitor currently in Phase III in post-abiraterone mCRPC patients and superior to available standard-of-care therapies showing rPFS rates of approximately 60% to 75%. Also, 47% of patients (7/15) achieved a PSA50 response, with 33% (5/15) confirmed, while 71% of patients (10/14) achieving >50% circulating tumor (ctDNA) reduction. Jefferies analyst Maury Raycroft, PhD, reported that some investors viewed rinzimetstat as “a better fit strategically” with Johnson & Johnson (NYSE: JNJ)-marketed Erleada® (apalutamide).
    • Sangamo Therapeutics (NASDAQ: SGMO) shares climbed 20% from 25 cents to 30 cents between Tuesday and Thursday after the genetic medicine developer said it expects to complete this summer its rolling Biologics License Application (BLA) submission seeking FDA approval for isaralgagene civaparvovec (ST-920), a gene therapy candidate being developed to treat Fabry disease, subject to securing adequate additional funding, while continuing business development discussions for a potential Fabry commercialization agreement. Sangamo said it is advancing the chemistry, manufacturing, and controls (CMC) module ahead of completion of the rolling BLA submission. Since launching its rolling BLA submission in December under an Accelerated Approval pathway, Sangamo has submitted preclinical and clinical modules, as well as submitted its antibody assay companion diagnostic, designed to screen patients for eligibility with isaralgagene civaparvovec, to the FDA’s Center for Devices and Radiological Health (CDRH), seeking Premarket Approval (PMA).

    The post StockWatch: Price War Dampens Lilly Surge After Oral GLP-1 Wins FDA Nod appeared first on GEN - Genetic Engineering and Biotechnology News.

    ]]>     Injectable Microgel Developed to Reduce Bleeding in Infants Undergoing Surgery https://edusehat.com/en/injectable-microgel-developed-to-reduce-bleeding-in-infants-undergoing-surgery https://edusehat.com/en/injectable-microgel-developed-to-reduce-bleeding-in-infants-undergoing-surgery Sat, 04 Apr 2026 06:20:10 +0700 Edusehat Injectable, Microgel, Developed, Reduce, Bleeding, Infants, Undergoing, Surgery Biomedical researchers headed by a team at the Lampe Joint Department of Biomedical Engineering, University of North Carolina at Chapel Hill/North Carolina State University, have developed an injectable microgel to help reduce bleeding in infants who require surgical care. Tests in an animal model showed that the hemostatic microgels, known as B-knob-triggered microgels (BK-TriGs), reduced bleeding by at least 50%.

    Research lead Ashley Brown, PhD, who is the Lampe Distinguished Professor of Biomedical Engineering, is co-corresponding author of the team’s published paper in Science Advances, titled “Hemostatic B-knob-triggered microgels (BK-TriGs) to address bleeding in neonates.” In their paper the team concluded “This study highlights the potential of BK-TriGs, designed for neonatal-specific clotting mechanisms, to address the heightened bleeding and thrombosis risks in neonates, who face 4.4 times higher postsurgery mortality … Our findings support BK-TriGs as a promising approach for improving hemostasis in neonates, offering a tailored, effective solution for this vulnerable patient population.”

    When adults cut themselves, a multi-step process called hemostasis stops the bleeding from the injured blood vessel. But hemostasis in infants is different from hemostasis in adults. This difference can be problematic if infants require surgery to address significant medical problems. In surgeries, neonatal patients normally receive blood from adult donors to compensate for blood lost during the operation. “Current treatments rely on transfusing adult blood products, which may cause complications resulting from structural and functional differences between neonatal and adult fibrinogen,” the authors wrote. “… these transfusions pose serious safety concerns by increasing morbidity, prolonging intensive care unit stays, and elevating posttransfusion thrombosis risks in neonates.”

    Brown noted, “… if you give adult blood to an infant, the difference in adult hemostasis versus infant hemostasis can lead to too much clotting. That can increase the likelihood of thrombosis, where blood clots form in the lungs or elsewhere and put the baby at risk … “My research team has done a lot of work on surgery-related bleeding in newborns, and we wanted to develop a therapeutic intervention that would reduce bleeding and—by extension—reduce the need for infants to receive adult blood transfusions during surgery.”

    The scientists have now reported on their development of a material called B-knob triggered microgels (BK-TriGs). “Fibrin is the main clotting protein in human blood,” Brown explains. “There is a short amino acid sequence called a ‘B peptide’ that links together fibrin molecules to create blood clots where they are needed—and these B peptides play a particularly important role in hemostasis for infants. The BK-TriGs are engineered particles that are studded with those B peptides.”

    The particles can absorb water and become squishy hydrogels, which mimic the mechanical properties of natural platelets in a way that maximizes the ability of the B peptides to create fibrin networks and stanch bleeding. “Functionalized with a fibrin hole b–specific peptide, BK-TriGs enhance clot density and resistance to degradation,” the team noted.

    The researchers first tested the BK-TriGs by using microfluidic devices that allowed them to conduct in vitro testing to see how the microgels affected clotting in blood plasma from human adults and infants. “In vitro studies using neonatal platelet-poor plasma (PPP) showed that at an optimal concentration, BK-TriGs increased clot density by more than 100% and improved stability by reducing fibrinolysis,” they wrote in summary. “Under flow conditions BK-TriGs promoted robust clot formation compared to plasma-only controls.” Brown noted, “We found that BK-TriGs worked better at improving blood clotting in infant plasma than in adult plasma, which was what we expected to see.”

    To further test the efficacy of the BK-TriGs, the researchers worked with lab mice that were genetically engineered to not make fibrinogen, the precursor to fibrin. This allowed the researchers to first introduce infant fibrinogen into the lab mice so that the mice exhibit a form of hemostasis similar to infants. “This innovative model enabled the evaluation of BK-TriGs in a setting that replicates key aspects of neonatal fibrinogen polymerization and fibrinolytic sensitivity, providing preliminary insights into their potential clinical utility.”

    Brown added, “We found that the BK-TriGs outperformed any of the other options we tested at reducing blood loss. Specifically, the BK-TriGs reduced blood loss by 50-60% compared to the control group.”

    The authors further stated, “The findings highlight the potential of BK-TriGs as a promising synthetic platelet-mimetic approach for enhancing clot density and stability, particularly in neonatal plasma where traditional blood products may pose risks … A fibrin-targeted approach like BK-TriGs, which enhances clot formation without introducing systemic thrombotic risk, may offer a safer alternative to adult fibrinogen transfusions.”

    Next steps for the work are to see how BK-TriGs compare to other hemostatic therapeutics that are on the market, either on their own or when used in conjunction with BK-TriGs. “The results we’re reporting here are exciting, but we are still far removed from clinical use,” Brown, acknowledged. “We need to make sure there are no unforeseen risks associated with blood clotting.” The team also commented, “Expanding this research to include different clinical bleeding scenarios will be essential to advancing these materials toward therapeutic applications.”

    “This is particularly relevant in neonates, where the most severe bleeding complications often arise in critical sites such as the gastrointestinal tract and the brain,” Brown continued, “But if we do find BK-TriGs are safe and effective, we’re optimistic this could be a cost-effective way to make surgery safer for infants. Manufacturing the BK-TriG particles would be relatively inexpensive—certainly in comparison to blood products.”

    The post Injectable Microgel Developed to Reduce Bleeding in Infants Undergoing Surgery appeared first on GEN - Genetic Engineering and Biotechnology News.

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