Bersama Kita Sehat - : Berita

Astrocytes Preserve Memory Persistence Through Ankyrin-2 Protein in Mice

Edusehat — Wed, 08 Jul 2026 22:10:09 +0700

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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Patient portal messages, EHR chat: How much time do docs spend?

Edusehat — Wed, 08 Jul 2026 22:00:08 +0700

Two new studies look at how patient portal messages and secure chat in the EHR are eating away at physicians’ time, with one finding that female hospitalists are spending more time on digital communication than their male counterparts.

A JAMA research letter found that the number of messages sent through patient portals jumped from 0.99 per patient per year in 2020 to 2.50 in 2025. During that time, patients who used portals to communicate with clinicians went from sending 2.2 messages to 5.4 messages per year.

The authors of the report claim it’s the first scientific look at how many messages patients are sending health care providers through portals.

The research letter looked at data from more than 2,000 hospitals and nearly 50,000 clinics encompassing 1.34 billion messages from patients. Researchers also examined patterns in 1.59 billion phone calls and 146 million telehealth encounters between patients and clinicians.

A MedPage Today analysis on the study reported that telephone encounters during the same period fell slightly, from 2.33 per patient per year to 2.20. Researchers also found a 17% increase in patient visits, from 2.37 visits per patient per year to 2.77.

A separate study of digital communication in the inpatient setting didn’t look at the growth of secure chat, but it did look at differences in the amount of time physicians spend on secure messaging based on gender.

The study, published in the Journal of Hospital Medicine, found that while female hospitalists in an academic hospital worked similar hours as men, women spent more time in the hospital’s EHR: 248 minutes per day vs. 222 for male hospitalists. That’s a difference of about 12%.

Researchers found that female hospitalists spent more time on secure chat than their male colleagues (38 vs. 34 minutes per day). Women hospitalists also exchanged more messages, sending 62 vs. 53 messages per day and receiving 56 vs. 48 messages per day.

The study looked at activity from July 2023 to June 2024 of just over 200 hospitalists. The group included a mix of adult and pediatric hospitalists and attendings and residents.

Trends in digital communication were similar in residents and attendings. And even when a new scheduling model that had physicians work less hours was implemented, the researchers found that gender differences in digital communication remained.

The authors warned that while digital communication technology was supposed to increase efficiency, “these tools may contribute to unequal digital workloads.” They said their data “underscore the need for equity-informed strategies” to reduce the burden of digital communications in the inpatient setting.

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Germline‑Targeting HIV Vaccine Generates Broadly Neutralizing Antibodies in Primates

Edusehat — Wed, 08 Jul 2026 11:25:10 +0700

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

Edusehat — Wed, 08 Jul 2026 11:25:09 +0700

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

Edusehat — Wed, 08 Jul 2026 07:50:07 +0700

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

Edusehat — Wed, 08 Jul 2026 04:10:12 +0700

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

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

Edusehat — Wed, 08 Jul 2026 04:10:11 +0700

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

Edusehat — Wed, 08 Jul 2026 04:10:10 +0700

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 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.

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

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
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
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
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
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
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
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
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

Edusehat — Wed, 08 Jul 2026 04:10:09 +0700

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 19^thcentury, they weren’t included on diagnostic criteria until the 21^st 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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Igniting Discovery: Cardiac research with a personal touch

Edusehat — Wed, 08 Jul 2026 04:05:13 +0700

HN Summary

• Sunnybrook Health Sciences Centre researchers are advancing personalized cardiac care through international clinical trials evaluating new minimally invasive procedures, medications and lifestyle interventions for heart disease.

• Recent studies found that a device-based procedure to prevent stroke in patients with atrial fibrillation can provide an effective alternative to long-term blood thinners, while another trial challenged the belief that coffee worsens irregular heart rhythms.

• Researchers say expanding treatment options and tailoring care to individual patient needs could improve outcomes, particularly for older adults and those with complex health conditions.

Heart disease is still the leading cause of death in North America. Healthy habits like eating well, staying active, and managing risk factors are important, and new tests and treatments are also helping people live longer and healthier lives.

In recent years, researchers at Sunnybrook’s Schulich Heart Program have been driving a shift toward more personalized and precise cardiac care, leading some of the largest randomized international clinical trials, and pioneering innovative approaches to treating heart disease and damaged blood vessels.

“There is no one-size-fits-all solution for treating heart conditions,” says Dr. Christopher Cheung, a cardiologist and director of the Schulich Heart Program’s clinical trials unit at Sunnybrook Research Institute (SRI).

“We need to personalize treatment options according to a patient’s health condition and also expand access to our treatments to those that may benefit. By expanding options for patients – including those traditionally considered higher risk due to age or other health conditions – we can better tailor care to each individual, helping them not only to live longer, but live better.”

Clinical trials testing interventional procedures

New waves of less-invasive and device-based cardiac procedures – being tested in clinical trials – are showing increasing promise as alternatives to open-heart surgery or long-term drug therapy, particularly for those with complex or multiple medical conditions, or elderly and frail patients that may not tolerate a traditional procedure or surgery.

Current and recent studies have spanned a number of cardiac procedures, addressing valve defects with minimally invasive techniques, to ablation of abnormal heart rhythms, to new treatments for heart attacks. One recent example, published in The New England Journal of Medicine in March 2026, adds to a growing body of innovation in cardiology research; with more advances on the horizon.

Led locally by Sunnybrook investigator Dr. Sheldon Singh, the CHAMPION-AF clinical trial enrolled patients across North America, Europe and beyond to evaluate whether a device-based procedure to prevent stroke in patients with irregular heart rhythm could perform as well as standard long-term blood-thinning therapy, while reducing bleeding risk.

The findings showed that left atrial appendage closure (LAAC) can offer comparable protection against serious cardiovascular events, while significantly lowering the risk of bleeding. This approach may provide an important alternative for patients who cannot tolerate blood-thinning medications, as well as for those with bleeding complications relating to blood thinners. The study authors emphasize treatment decisions should always consider individual patient risk factors and preferences.

CHAMPION-AF is one of many international clinical trials facilitated by the Schulich Heart Program clinical trials unit, many of which are focused on catheter-based interventions compared to standard care. These procedures involve guiding thin flexible tubes and wires (“catheters”) through blood vessels in the groin, arm or neck to the heart using real-time X-ray imaging.

Clinical trials studying the role and impact of lifestyle factors and modifications

In addition to a wide range of interventional therapies, the Schulich Heart Program’s Clinical Trials Unit also studies the role and impact of lifestyle factors and modifications one can make to improve their cardiac health.

The aptly-named DECAF study, for example, led by Dr. Cheung, sought out to test a long-standing belief that many doctors and patients have that coffee – especially caffeinated coffee – could trigger or worsen arrythmia (an abnormal heart rhythm), or atrial fibrillation (a specific, common type of irregular heart rhythm or arrythmia).

Presented at the American Heart Association Scientific Sessions in November 2025 and published in the Journal of the American Medical Association, the DECAF trial found that coffee drinkers who continued drinking coffee had less arrhythmias compared to those who stopped drinking coffee.

While the researchers outline the limitations of the study, the bottom line is that for most people with atrial fibrillation, the routine advice to completely avoid coffee may no longer make sense for many patients, say the authors. They do, however, stress there are always exceptions to the rule and individual reactions still matter, as some people may still notice caffeine triggers their atrial fibrillation and need to maintain close communication with their physician.

“Whether it’s lifestyle and medication management, minimally invasive procedures, techniques to correct heart rhythms, or open-heart surgery—there has never been more choice in cardiology,” says Dr. Cheung, co-lead author of the DECAF study, and a cardiac electrophysiologist (a specialist in heart rhythms). “It represents a meaningful shift toward more tailored care and precision medicine, which has been the goal of our research.”

“These newer options may be especially beneficial for select groups of patients—for example, older individuals in their seventies and beyond, or those with complex health conditions where surgery may be too risky or long-term therapies may pose challenges. In many cases, it ultimately comes down to patient preference—whether that’s a procedure, surgery, or drug therapy.”

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A New Development Playbook for PROTACs

Edusehat — Wed, 08 Jul 2026 00:35:16 +0700

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

Edusehat — Wed, 08 Jul 2026 00:35:15 +0700

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.”

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AI Tackles Tuberculosis, Identifies Drugs that Penetrate Bacteria Membrane

Edusehat — Wed, 08 Jul 2026 00:35:12 +0700

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

Edusehat — Wed, 08 Jul 2026 00:35:11 +0700

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.

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.

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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2025 ABIM Pass Rates: How Difficult are the ABIM Exams?

Edusehat — Wed, 08 Jul 2026 00:05:08 +0700

The American Board of Internal Medicine (ABIM) offers initial certification exams across 19 medical specialties. Newly released pass rate data for 2025 sheds light on trends and challenges faced by healthcare professionals pursuing board certification. These statistics provide some extra insight on just how difficult the ABIM board exams are, and the importance of adequate exam prep. We look at the 2025 ABIM pass rates by program:

Internal Medicine

In 2025, the pass rate for first-time test takers remained level, with 86% of takers passing the exam.

Cardiovascular Disease

In 2025, the pass rate for first-time test takers remained level, with 86% of takers passing the exam.

Critical Care Medicine

The Critical Care Medicine board exam saw a slight drop in pass rates from 2024 to 2025, with 86% passing the exam.

Gastroenterology

The Gastroenterology board exam saw a slight decrease in pass rates, going from 97% in 2024 to 94% in 2025.

Geriatric Medicine

The Geriatric Medicine board exam saw a rise in pass rates from 2024 to 2025, going from 89% to 94%.

Hematology

The Hematology board exam pass rate was steady from 2024 to 2025, with 93% passing in 2025.

Infectious Disease

The Infectious Disease board exam pass rate dropped slightly in 2025, with 93% of takers passing the exam.

Medical Oncology

In 2025, the pass rate for first-time test takers remained the same, with 90% of takers passing the exam.

Nephrology

The Nephrology board exam saw a slight increase in pass rates from 2024 to 2025, going from 82% to 83%.

Pulmonary Disease

The Pulmonary Disease board exam saw steady pass rates of 93% from 2024 to 2025.

Rheumatology

The Rheumatology board exam saw a slight increase in pass rates, going from 90% in 2024 to 92% in 2025.

Sleep Medicine

The Sleep Medicine board exam saw the largest drop in pass rates of all ABIM exams, going from 90% in 2024 to 83% in 2025.

Studying for one of the ABIM exams? BoardVitals offers top prep products targeted to 2026 ABIM blueprints.

What Do the Pass Rates Mean?

While a high pass rate on the ABIM board exams may be encouraging, it’s crucial for aspiring candidates to recognize that higher pass rates do not negate the need for thorough preparation. A high overall pass rate reflects the overall average performance of candidates, but individual experiences may vary. Success in the exams is contingent on a comprehensive understanding of the material, critical thinking skills, and the ability to apply knowledge in diverse clinical scenarios.

Don’t become complacent. Every candidate should approach the exams with a mindset of continuous improvement and a commitment to refining their understanding of internal medicine. Don’t put off studying – you don’t want to regret not being as prepared as possible.

How Can I Prepare for the ABIM Board Exams?

Effective preparation is key to passing ABIM board exams. Utilize reputable study resources and review courses that align with the exam’s requirements. The ABIM makes blueprints for all exams easily accessible. Familiarize yourself with these content specifications so you know exactly what to study for your exam.

One of the most popular and effective study methods is incorporating practice questions into your study regimen. Practice questions not only assess your knowledge but also enhance your critical thinking and problem-solving skills, mirroring the challenges you may encounter in the actual exam.

BoardVitals offers question banks for a number of ABIM exams. Each question bank is tailored to the most updated ABIM blueprint. All questions include explanations for correct and incorrect answers, letting you uncover problem areas before test day.

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Should You Prep for the AOBIM or ABIM Exam?

Edusehat — Wed, 08 Jul 2026 00:05:08 +0700

In the field of Internal Medicine, there are two recognized board certification exams: The American Board of Internal Medicine (ABIM) exam and the American Osteopathic Board of Internal Medicine (AOBIM) exam. How do these exams compare, and which should medical professionals take based on their practice and career goals?

How are the AOBIM Exam and ABIM Exam Similar?

The AOBIM and ABIM exams share many similarities, including:

Both the AOBIM and ABIM internal medicine exams are designed to test the candidates’ ability to diagnose and treat various medical conditions, including acute and chronic illnesses.
Both exams are computer-based tests that consist of multiple-choice questions and case studies.
Both exams require candidates to have completed a residency program in Internal Medicine.
Both exams require candidates to complete continuing medical education (CME) credits to maintain their certification.

How are the AOBIM Exam and ABIM Exam Different?

Although the AOBIM and ABIM exams share many similarities, there are some notable differences that physicians should consider before choosing an exam:

Osteopathic Approach: One of the main differences between the AOBIM and ABIM exams is the Osteopathic approach. The AOBIM exam emphasizes the Osteopathic principles and practices, which focus on treating the whole person and promoting wellness. In contrast, the ABIM exam is more focused on allopathic medicine, which is a traditional approach to medicine that emphasizes the use of drugs and surgery to treat illnesses.

Accreditation: The AOBIM exam is accredited by the American Osteopathic Association (AOA), while the ABIM exam is accredited by the Accreditation Council for Graduate Medical Education (ACGME).

Exam Length: The AOBIM exam has a slightly different format than the ABIM exam. The AOBIM exam is a single day exam consisting of 320 multiple choice questions, while the ABIM exam lasts approximately 10 hours and consists of a maximum of 240 multiple choice questions.

Is AOBIM easier than ABIM?

As both exams have their own unique requirements and content areas, it is difficult to say whether the AOBIM or ABIM is the more difficult exam. In 2025 the first-time pass rate for the ABIM exam was 86%. There is no publicly available pass rate data for the AOBIM.

Should You take the AOBIM Exam, the ABIM Exam, or Both Internal Medicine Board Exams?

The AOBIM and ABIM exams both offer unique benefits, but which test is more beneficial? In short, the decision depends on the physician’s personal preference and career objectives. The choice between the two exams will depend on the physician’s practice and career goals. Those who practice Osteopathic Medicine may find the AOBIM exam more aligned with their philosophy and approach, while those who practice allopathic medicine may prefer the ABIM exam.

How do I review for the AOBIM or ABIM?

No matter which exam you choose to take, having top board prep tools is essential to your success. The BoardVitals Internal Medicine board review question bank has been newly updated for 2025-2026. It now provides more than 1,650 Internal Medicine board questions targeted to the latest ABIM and AOBIM Content Blueprints.

Along with expertly written sample questions, the BoardVitals Internal Medicine Question Bank contains innovative technology to help you make the most of studying. Create quizzes with adaptive testing technology, which presents questions tailored to your current competency level. The Internal Medicine Question Bank also includes AI-powered risk assessment performance data, providing insight into your most at-risk subjects to optimize study efficiency.

Both the AOBIM and ABIM exams are rigorous and comprehensive tests that evaluate the medical knowledge and clinical competence of physicians. Whether you sit for one or both exams, you will be prepared to practice in the field of Internal Medicine.

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Biomanufacturing in Space to Be Key Topic at ISSCR 2026

Edusehat — Tue, 07 Jul 2026 21:00:10 +0700

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

Edusehat — Tue, 07 Jul 2026 10:15:09 +0700

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.

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

Edusehat — Tue, 07 Jul 2026 10:15:08 +0700

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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Pressure increases on health employers to invest in stabilizing BC’s health-care system

Edusehat — Tue, 07 Jul 2026 06:35:09 +0700

Beginning on Tuesday, July 7 at 5:30 a.m. PT, BC nurses will escalate job action with picket line at Vancouver General Hospital (VGH), while maintaining essential service levels to protect patient safety. The announcement comes one day after a province-wide non-nursing ban and restriction on overtime for Nurses’ Bargaining Association (NBA) members.

“Nurses do not want to be on picket lines,” says BCNU President Adriane Gear. “We are in this profession to care for patients. But the provincial government and health employers can no longer ignore the demands of our health-care system, nor can they disregard the incredible pressure nurses face day in and day out. Even now, health employers are attempting to undermine nurses’ rights to take lawful job action by threatening discipline against those who are holding the line.”

In addition to the VGH picket line planned for July 7, NBA members across the province will continue to refuse non-nursing duties and restrict overtime. Nurses are set to be back at the bargaining table with health employers on Monday afternoon.

“The provincial government must come back to the table with an offer that respects nurses and recognizes their value in our health-care system,” says BCNU CEO Jim Gould. “This government is not going to solve the nursing shortage without making serious improvements to nurses’ working conditions. The union will also not tolerate any attempts by the employer to threaten or intimidate our members who are participating in lawful job action.”

The current job action follows an historic strike vote in which 50,850 nurses participated, with 98.2 per cent voting in favour of job action. Members later rejected a tentative agreement by 67 percent, signalling that the government’s bargaining mandate failed to address nurses’ core concerns, including a meaningful general wage increase and solutions that improve nurse retention and workplace safety

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Pharma Races to Scale AI as Billions Flow into Drug Discovery

Edusehat — Tue, 07 Jul 2026 03:05:09 +0700

“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

Edusehat — Tue, 07 Jul 2026 03:05:08 +0700

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.

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ALS Drug Extends Survival in Mice, Targets TDP-43 Low-Complexity Domain

Edusehat — Mon, 06 Jul 2026 23:30:11 +0700

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.”

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StockWatch: New UC Data Sparks Smoother Sailing for Abivax

Edusehat — Mon, 06 Jul 2026 05:35:08 +0700

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.

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A device that revives eyeballs from dead donors could make eye transplants possible

Edusehat — Sat, 04 Jul 2026 07:05:02 +0700

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.

Just Getting Started with the USMLE Step 1/COMLEX Level 1? This Beginner’s Guide Has You Covered

Edusehat — Fri, 03 Jul 2026 20:35:04 +0700

Introduction: The First Big Hurdle

Every medical student knows that Step 1 or COMLEX Level 1 is a defining moment. Even though Step 1 has shifted to pass/fail, both exams still carry weight, and both require months of preparation. The good news is that the students who succeed aren’t necessarily the ones who know every detail, they’re the ones who start smart.

Step 1: Begin With the End in Mind

The smartest way to start is to pick a test date. It’s tempting to wait until you “feel ready,” but that usually leads to endless review without real progress. Once you have your date, schedule the exam so it feels real! Work backward to decide how many weeks you have for dedicated prep. Most students take 6-10 weeks of full-time (8-10 hours per day) studying, though part-time prep alongside rotations can work too if you’re strategic.

When you’re mapping out your schedule, set weekly goals that are specific and measurable. Instead of “study microbiology,” aim for “complete and review 200 UWorld questions this week.” Goals like that keep you accountable and help you see progress.

Step 2: Limit Your Resources

There are tons of Step 1/COMLEX resources. The trap is thinking you need them all. You don’t. You need two or three high-yield resources that cover the essentials, and you need to use them consistently in-depth.

The foundation for most students is UWorld, it’s not just practice, it’s a learning tool. Pair it with First Aid for a consolidated review, and then choose one supplemental resource like Boards & Beyond, Pathoma, or Sketchy for reviewing material at the level of detail you’re expected to know for the exam. Spaced repetition should absolutely be part of your preparation and Anki (especially the AnKing deck) is the best tool I’ve found to help cement the details. That’s it. Stick to your core set and resist the temptation to keep adding more.

Step 3: Don’t Delay Practice Questions

A common trap students fall into is thinking they need to finish “all” of content review before they can start practice questions. This is especially the case if students’ pre-clinical curriculum extends into the initial months they want to start preparing for their exam. It feels safer to highlight, re-read, and make notes because it feels like you have’t learned all the material you need to know yet. But here’s the problem: Step 1 and COMLEX are not pure memorization exams. They test whether you can integrate knowledge and reason your way through unfamiliar clinical vignettes. Further, the NBME-style questions are usually quite different from the questions you see on your medical school subject exams so you need to get used to this type of format. And the only way to get good at this is to practice.

That’s why UWorld (or another high-quality question bank like AMBOSS) should be part of your plan from the very beginning. Even if you feel unprepared, start with small sets, 10 or 20 questions at a time. Every explanation you read is a mini-lesson. Over time, those lessons add up to a far deeper understanding than passive review alone could ever give you.

The real power of practice isn’t in the number of questions you answer, but in how you review them. Don’t just note that you got something wrong and move on. Ask yourself:

Was this a content gap? If yes, add the concept to Anki so you can continue to review it.
Was this a test-taking mistake? Maybe you misread the question stem or ignored a key clue in the labs.
Was this a reasoning issue? Did you struggle to connect multiple steps or eliminate distractors?

By categorizing your errors, you’ll start to see patterns. Maybe you’re consistently shaky on renal physiology, or maybe you rush through long stems and miss critical details. Once you recognize those trends, you can fix them deliberately.

Another strategy that works well is to slow down and treat each UWorld block as a learning opportunity, not just a score check. Read the full explanations for every answer, right and wrong. Ask yourself how you could have gotten to the correct answer faster. Could you have eliminated two distractors quickly? Was there a lab value or keyword that should’ve stood out? This is how you sharpen the clinical reasoning skills that Step 1 and COMLEX are built to test.

As you progress, gradually increase the size of your question blocks. Early on, untimed, shorter blocks of certain subject questions can help you focus on understanding. Later, full-length mixed question blocks will build the pacing and endurance you’ll need for the real exam. And don’t underestimate that endurance piece, both Step 1 and COMLEX are long exams. You need to train your brain (and your focus) to handle hours of questions under pressure.

Practice exams also fit into this picture. NBMEs for Step 1 and COMSAEs for COMLEX are predictive, but they’re more than just diagnostic snapshots. They show you how ready you are, but they also reveal exactly where your weaknesses lie. After each practice test, spend time breaking down not just the score, but the story behind the score: which subjects dragged you down, which question types slowed you, and where you made careless mistakes.

Finally, create a “review ritual” that works for you. Some students keep a detailed error log spreadsheet; others create Anki cards out of every missed question. What matters is that you’re turning your mistakes into active learning. Over weeks, you’ll start to notice those once-frustrating topics becoming strengths.

The takeaway: practice is not something you tack on at the end of your prep. It is your prep. UWorld and practice exams are where the real growth happens, and reviewing them thoughtfully is what turns effort into results.

Step 4: Build Daily Structure

A good study day balances practice and review. Many students like to start with a timed UWorld block in the morning, spend the midday reviewing explanations and adding to Anki, and finish the afternoon or evening with targeted content review. This is just an example, figure out what works best for you. Study schedules will differ for everyone and that’s ok. There is not a “one size fits all” approach. The most important thing is that you stick to a routine that promotes consistency and that you’re seeing growth and progression in your practice scores.

This routine helps you build both breadth and depth while keeping your days predictable. And just like with any other big exam, burnout is a real risk during dedicated prep, so build in breaks, exercise, and sleep to keep yourself sharp. If you don’t take care of yourself and manage your stress, you probably won’t see the progress that you’re capable of.

Step 5: Extra Notes for COMLEX Takers

If you’re taking COMLEX specifically, don’t neglect OMM. It can be easy to focus only on “Step-style” questions, but osteopathic manipulative medicine is heavily tested on COMLEX. A concise, high-yield resource like Savarese’s OMT Review will give you what you need. Pair it with UWorld for general prep, and add a COMLEX-style question bank like COMBANK or TrueLearn.

Step 6: Use Practice Exams as Your Compass

NBMEs (for Step 1) and COMSAEs (for COMLEX) are the best predictors of how you’ll do on test day. Take them every 1-2 weeks to check your progress and make adjustments. They’re not just score reports, they’re diagnostic tools to help you fine-tune your plan. If you’re not seeing improvement in your scores, your study plan is not working for you and you need to figure out how to make an effective change.

Takeaway

Step 1 and COMLEX Level 1 feel huge when you’re at the starting line. But success doesn’t come from memorizing every fact, it comes from starting smart. Schedule your test date, back into a realistic schedule, commit to a small set of resources, and begin practicing early. Take care of yourself along the way, and you’ll not only pass, you’ll build the foundation you need for the rest of your medical career.

The post Just Getting Started with the USMLE Step 1/COMLEX Level 1? This Beginner’s Guide Has You Covered appeared first on Elite Medical Prep.

The UK’s generational tobacco ban might not work. I’m supporting it anyway.

Edusehat — Fri, 03 Jul 2026 20:20:02 +0700

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

Edusehat — Fri, 03 Jul 2026 06:00:03 +0700

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

Edusehat — Fri, 03 Jul 2026 02:20:11 +0700

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

Edusehat — Fri, 03 Jul 2026 02:20:10 +0700

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

Edusehat — Fri, 03 Jul 2026 02:20:09 +0700

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.

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Peptistar Began Operation of the Asahi Kasei FO-MD System at Manufacturing Scale

Edusehat — Thu, 02 Jul 2026 22:45:10 +0700

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 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]

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.

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Derm Deep Dive: Sun, Saltwater & Chlorine Tips for This Summer

Edusehat — Thu, 02 Jul 2026 22:45:06 +0700

Protecting Your Hair and Scalp This Summer at the Pool, Beach, and Under the Sun

We’re back with another Derm Deep Dive, where we provide expert advice from our board-certified dermatologists about common skin conditions or treatments.

Using sunscreen daily, especially in the summer is widespread advice for protecting the skin from UV rays. But your skin isn’t the only thing that needs protection in the summer.

The sun, chlorine, and saltwater can do a number on your hair and scalp, but we often don’t consider proper protection before or after spending time at the pool or on the beach.

To help you prepare, and to alleviate any unexpected challenges this summer, we have 4 tips for taking care of your hair and scalp that will leave you sun and fun ready.

1. The Sun & Your Scalp: Use an SPF on Your Part Line

One of the easiest places to get a sunburn is your scalp. It’s also one of the easiest places to protect by applying an SPF directly to your part line and hairline, or wearing a wide-brimmed or UPF-rated hat.

Even individuals with thick hair are susceptible to UV rays, which can carry multiple risks:

Skin cancer from repeated UV exposure

Sun damage and severe sunburn

Drying out the scalp and causing flakiness or irritation

Not a fan of putting sunscreen on your scalp? Consider trying a broad-spectrum powder sunscreen, scalp spray, or lightweight mist, all of which are easy to use and won’t leave the hair feeling or looking greasy. Choose an SPF 30 or higher and remember to reapply every two hours if you’re swimming or sweating heavily.

2. The Sun & Your Hair: Use a Heat Protectant Before Heading Outside

While heat protectants are helpful as styling products for blow drying, straightening, or curling hair, they can also help minimize damage from prolonged UV exposure. Some leave-in conditioners and heat protectants contain ingredients that help coat the hair shaft, lock in moisture, and prevent color fade from the sun’s rays.

Armor your hair with a lightweight leave-in conditioner or UV-protectant spray. These products can also reduce dryness, breakage, or frizziness caused by the sun.

3. Saltwater & Your Hair: Rinse With Clean Water Before Jumping In

Wet hair absorbs less saltwater than dry hair, so if you can, rinse with clean water before dunking in the ocean. Even better – rinse again as soon as possible after swimming and use a hydrating conditioner or hair mask to restore moisture.

If you swim in saltwater a lot and want a summer-long solution for the effects of saltwater on your hair and scalp, try these tips:

Use sulfate-free shampoos to cleanse hair without stripping its natural moisture

Apply leave-in conditioner before swimming, which creates a hydrophobic barrier around the hair shaft

Limit excessive heat styling right after beach days to prevent additional damage

Use moisturizing scalp treatments if irritation develops

You may experience softer hair or “beachy waves” after swimming in the ocean, but saltwater can also suck moisture out of your hair, leaving strands feeling brittle or more susceptible to damage. It can also lighten color-treated hair and increase photosensitivity, which can cause breakage.

4. Chlorine & Your Hair: Wear a Swim Cap When Possible

Chlorine is a chemical designed to kill bacteria and keep pools safe for swimming, but when it’s absorbed by hair, it can strip away natural oils, leaving hair feeling dry, brittle, and more prone to breakage.

Frequent chlorine exposure can also affect hair color, and blonde hair is particularly vulnerable because chlorine can react with metals like copper in pool water, sometimes causing lighter hair to develop a greenish tint. Color-treated hair may also fade faster with repeated exposure.

The best option for protecting your hair from chlorine is to wear a swim cap, but you can also follow these tips to reduce chlorine absorption:

Wet your hair with fresh water before entering the pool

Apply a leave-in conditioner or protective hair oil before entering the pool

Shampoo and condition with hydrating products as soon as possible after swimming

Clarifying shampoos are popular for removing chlorine buildup but should be used only when necessary; overuse can further dry out the hair.

Signs of Sun Damage and the Risks of Skin Cancer

The scalp is one of the most overlooked areas for sun protection, especially for people with thinning hair, wider parts, or hairstyles that expose the scalp. Repeated UV exposure increases the risk of developing skin cancer or sun damage.

It’s never too late to start protecting your scalp from UV rays, but it’s also important to be aware of the signs of sun damage, as chronic sun damage can contribute to precancerous lesions and skin cancers over time.

Watch for:

Scalp tenderness or redness

Peeling along the part line

Increased scalp dryness or flaking

Brittle or rough hair texture that doesn’t return to normal

Faded hair color

Increased breakage or split ends

If you notice persistent rough patches, sores that don’t heal, or scalp irritation, schedule an appointment with your board-certified dermatologist for a full evaluation.

Healthy Hair and Scalp, Happy Summer!

You don’t have to avoid the outdoors or going for a refreshing swim in the ocean or pool. With the right protection, you can enjoy the summer while maintaining your healthy hair and scalp – and staying safe from sun damage.

Treat your scalp with the same care you give the rest of your skin. If you experience any hair or scalp challenges during the summer, especially flaky skin or increased hair damage, get an expert opinion from your dermatologist.

Schedule an appointment with our team if you’d like some extra help preparing for or managing your summer hair and skin health.

FAQs About Protecting Your Hair and Scalp from Sun, Saltwater, and Chlorine

Should I put sunscreen on my scalp?

Yes. The scalp is highly susceptible to sunburn and UV damage, especially along the hair part, hairline, or thinning areas. Dermatologists recommend using broad-spectrum SPF 30 or higher on exposed scalp areas.

Does chlorine permanently damage hair?

Chlorine does not usually cause permanent damage after occasional exposure, but repeated exposure can dry out the hair shaft, weaken hair, strip natural oils, and contribute to breakage or color fading over time.

Why does blonde hair turn green in pools?

The green tint often comes from copper and other metals in pool water reacting with chlorine and binding to the hair shaft. Blonde and lighter-colored hair tends to show the discoloration more visibly.

Is saltwater bad for your scalp?

Saltwater can dry out the scalp and worsen irritation or flaking in some individuals. Rinsing with fresh water after swimming and using moisturizing scalp products can help restore hydration.

What is the best way to protect hair from sun damage?

The best approach combines multiple strategies: wearing hats, applying SPF to exposed scalp areas, using UV-protective or heat-protectant hair products, limiting prolonged sun exposure, and restoring moisture after swimming or sun exposure

The post Derm Deep Dive: Sun, Saltwater & Chlorine Tips for This Summer appeared first on Optima Dermatology.

First 3D Structure of Malaria’s “Moving Junction” Solves Infection Mystery

Edusehat — Thu, 02 Jul 2026 08:25:08 +0700

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

Edusehat — Thu, 02 Jul 2026 04:50:09 +0700

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.”

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In Huntington’s Mouse, Optogenetic Activation of VIP Neurons Restores Brain Function

Edusehat — Thu, 02 Jul 2026 04:50:08 +0700

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.”

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.”

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Trait Combining Key to More Effective Vector Production Hosts

Edusehat — Thu, 02 Jul 2026 01:15:12 +0700

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

Edusehat — Thu, 02 Jul 2026 01:15:11 +0700

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.

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Standardizing Personalized CRISPR Gene-Editing Therapies

Edusehat — Thu, 02 Jul 2026 01:15:09 +0700

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

Edusehat — Thu, 02 Jul 2026 01:15:08 +0700

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.

Can You Practice Medicine in the U.S. Without Residency? What the New 2026 State Laws Actually Mean

Edusehat — Wed, 01 Jul 2026 22:00:04 +0700

Over the past few years, one topic has spread rapidly across international medical graduate (IMG) forums, Reddit threads, WhatsApp groups, and medical school advising meetings: “Can international medical graduates now practice in the U.S. without residency?”

For many IMGs, the idea sounds almost too good to be true. After years of hearing that repeating residency in the United States was essentially unavoidable, several states have now introduced alternative licensing pathways that appear to allow some internationally trained physicians to practice medicine without completing a traditional U.S. residency program.

Naturally, this has created excitement, confusion, misinformation, and a lot of unrealistic expectations.

The short answer is yes, some states are now creating pathways that may allow certain experienced international physicians to practice without repeating U.S. residency training. But the longer answer is much more complicated, and that nuance is where many applicants misunderstand what these laws actually mean. (Niskanen Center)

What Has Recently Changed?

In response to worsening physician shortages, especially in rural and underserved areas, more than a third of U.S. states have either enacted or explored alternative licensure pathways for internationally trained physicians (ITPs) and IMGs. (Niskanen Center)

Traditionally, the pathway for IMGs in the United States has been extremely rigid. Regardless of prior experience abroad, physicians generally needed to complete the following steps:

ECFMG certification
USMLE exams
Matching into a U.S. residency
Completing residency training in the U.S. or Canada
Obtaining board certification and full licensure afterward

The residency bottleneck has long been one of the biggest barriers. In 2025 alone, over 47,000 applicants competed for approximately 37,000 first-year residency positions. Match rates for IMGs remained significantly lower than those for U.S. MD and DO seniors. (Niskanen Center)

As a result, states facing physician shortages began exploring whether experienced doctors trained abroad could safely practice under supervised or provisional pathways instead of repeating years of residency training they may have already completed overseas.

What These New Laws Actually Allow

This is where the biggest misconception happens.

These laws generally do not mean that anyone with a foreign medical degree can suddenly start independently practicing medicine in the United States next month.

Most of the newer pathways are designed for physicians who already completed residency training abroad, practiced independently in another country for several years, passed licensing exams, and can demonstrate recent clinical experience. (Niskanen Center)

In many states, applicants still need:

ECFMG certification
USMLE Step completion (often all three step exams)
Proof of postgraduate training abroad
Active or recent clinical practice
Sponsorship from a healthcare employer
Supervised or provisional practice periods

In other words, these laws are usually not bypassing physician competency standards. They are mainly bypassing the requirement to repeat an entire U.S. residency program from the beginning. (Niskanen Center)

That distinction matters enormously. The Biggest Misunderstanding: These Are Often Provisional Pathways One of the biggest pitfalls IMGs fall into is assuming these are immediate full-license opportunities.

In reality, many states use a provisional or supervised licensing model first. Physicians may initially work under supervision at approved healthcare systems or sponsoring employers before becoming eligible for unrestricted licensure later. (Niskanen Center)

Some states also limit practice settings to underserved communities, rural hospitals, academic systems, or specific facilities. This means these pathways often function more like a monitored transition period rather than an instant independent attending role. For some physicians, that may still be an excellent opportunity. But it is important to understand that the details vary significantly by state.

State Requirements Are Not Uniform

Another major source of confusion is that there is no single national IMG alternative pathway. Every state has different eligibility rules, timelines, supervision requirements, and definitions of acceptable foreign training. (Niskanen Center)

Some states require several years of independent practice abroad. Others focus more heavily on residency equivalency. Some allow eventual conversion to unrestricted licensure, while others only offer temporary or limited licenses.

For example, some states emphasize recent clinical practice, while others are stricter about how similar foreign residency programs must be compared to U.S. training standards. (Niskanen Center). This means that a physician who qualifies in one state may not qualify in another.

One of the biggest mistakes IMGs make is reading headlines or social media summaries without reviewing the actual medical board requirements for the state they are interested in. It’s important to do the research and find out the important details.

These Laws Mainly Target Experienced Physicians

Another important reality is that these pathways were largely designed for experienced international physicians, not necessarily newly graduated IMGs who have never practiced independently.

Many laws specifically focus on doctors who:

Already completed residency abroad
Have active licensure overseas
Have recent years of clinical practice
Can demonstrate ongoing competency

This is a critical distinction because many students or recent graduates interpret these laws as alternatives to residency matching altogether.

For newer graduates without significant independent clinical experience, traditional residency training may still remain the most realistic and stable path into U.S. medicine. And it’s important not to forget that this is an option.

Physician Shortages Are Driving These Changes

The driving force behind these reforms is the growing physician shortage in the United States. Policymakers, hospitals, and advocacy groups increasingly argue that experienced international physicians are being underutilized while many communities struggle to access care. (Niskanen Center)

Some internationally trained physicians living in the U.S. currently work in research, administration, medical assistance, or non-clinical jobs despite years of prior physician experience abroad. Reform advocates argue that forcing all experienced foreign-trained doctors to repeat residency from scratch may unnecessarily waste medical talent. (Niskanen Center)

At the same time, critics raise concerns about standardization, patient safety, physician compensation, supervision quality, and long-term implementation challenges. (Reddit)

That debate is still evolving.

What IMGs Should Be Careful About

One of the biggest mistakes IMGs make right now is assuming these pathways are already fully functional and widely available.

In reality, implementation has been slower than many people expected. Some state medical boards are still developing regulations, reviewing credentialing systems, and determining how supervision and sponsorship structures will work. (Niskanen Center)

Just because a law was passed does not necessarily mean hospitals are already hiring large numbers of physicians through these pathways.

Another major issue is sponsorship. Many pathways require healthcare systems or employers willing to supervise and sponsor internationally trained physicians. Even if the law exists, finding an employer prepared to navigate the process may still be difficult. (Niskanen Center)

Visa issues also remain complicated. These laws do not automatically solve immigration barriers, work authorization issues, or sponsorship logistics. Some IMGs online have pointed out that visa sponsorship may still remain one of the biggest practical hurdles. (Reddit)

Does This Mean Residency Is Becoming “Optional”?

For most IMGs, the answer is still no. Traditional residency training remains the standard and most universally accepted pathway into U.S. medical practice. Completing residency in the United States still provides:

Board eligibility
Stronger employment flexibility
Easier credentialing
Fellowship access
Greater geographic mobility
More predictable long-term career options

Many of the newer pathways are still evolving, and long-term outcomes are not fully established yet. Some employers, insurers, and credentialing bodies may also treat these alternative pathways differently from traditional residency-trained physicians.

That does not mean these laws are unimportant. They absolutely represent one of the biggest changes in IMG policy discussions in years. But they are not replacing residency overnight.

Why So Much Misinformation Exists Online

Part of the confusion comes from how these laws are discussed online. Headlines often simplify the story into phrases like “practice medicine without residency,” which leaves out the important details about supervision, licensing restrictions, exams, sponsorship, and eligibility.

On forums and social media, some physicians are optimistic and see these laws as long-overdue reform. Others remain skeptical about whether hospitals, boards, insurers, and employers will fully embrace the pathways. (Reddit)

The truth likely falls somewhere in the middle. These laws are real, and they are expanding. But they are also new, variable, and still being implemented differently across states.

What IMGs Should Focus on Right Now

For most IMGs, the smartest approach right now is to stay informed without abandoning traditional planning.

Students and physicians should still prioritize:

Passing USMLE exams
Obtaining ECFMG certification
Building strong clinical experience
Understanding state-specific licensing rules
Monitoring medical board updates directly
Keeping residency applications as an option whenever possible

One of the biggest mistakes applicants make is assuming these pathways eliminate the need for preparation or competitiveness. In reality, many states still require strong credentials and documented clinical competence.

At the same time, experienced physicians who previously felt permanently locked out of U.S. medicine may now have additional opportunities worth exploring carefully.

The Bottom Line

The new 2025–2026 state laws do not mean residency suddenly disappeared in the United States. What they do mean is that some states are beginning to recognize that experienced international physicians may not always need to completely restart their training from the beginning.

Fill out the form below to receive your free Residency Application Timeline via email!

These pathways are best understood as carefully supervised alternative licensure models, not shortcuts into unrestricted medical practice for inexperienced physicians.

For some internationally trained physicians, especially those with extensive prior experience abroad, these laws could become meaningful opportunities over the next several years. For others, traditional residency will likely remain the ideal and most stable route.

Either way, one thing is clear: the conversation around IMG licensing in the United States is changing rapidly, and these laws represent one of the most significant shifts in physician workforce policy in recent times.

The post Can You Practice Medicine in the U.S. Without Residency? What the New 2026 State Laws Actually Mean appeared first on Elite Medical Prep.

Roundtables: Longevity’s Next Frontier: “Reprogramming” Your Body

Edusehat — Wed, 01 Jul 2026 07:25:02 +0700

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

Edusehat — Wed, 01 Jul 2026 07:20:08 +0700

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

Edusehat — Wed, 01 Jul 2026 03:45:09 +0700

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

Edusehat — Wed, 01 Jul 2026 03:45:08 +0700

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]

“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

Edusehat — Wed, 01 Jul 2026 03:45:07 +0700

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.

Where AI is making the biggest impacts in healthcare

Edusehat — Wed, 01 Jul 2026 03:40:11 +0700

Highlights from the e‑Health Conference 2026

HN Summary

• AI is improving healthcare efficiency by reducing physician burnout, streamlining documentation and enhancing patient care through tools such as medical scribes and AI-assisted treatment planning.

• Healthcare organizations across Canada are using AI to improve system performance, including reducing long-term care wait times, enhancing mental health monitoring and streamlining patient referrals.

• Speakers emphasized that AI investments must deliver measurable clinical and financial value to help healthcare systems maximize limited resources.

s healthcare leaders from across Canada and abroad gathered in Halifax, Nova Scotia, for the 2026 e-Health Conference, one topic dominated the conversation: the rapidly expanding role of artificial intelligence (AI) in healthcare.

Throughout the conference, speakers shared compelling examples of how AI is improving efficiency across the healthcare system while emphasizing that successful implementation requires careful planning, evaluation and strategic investment.

During the opening plenary session, three healthcare leaders discussed what they described as the “low-hanging fruit” of AI—solutions that are relatively easy to implement while delivering significant benefits. Among the most promising are AI medical scribes and documentation tools that listen to or analyze patient consultations and automatically generate structured clinical notes, referral letters and treatment plans.

“The evidence is no longer refutable that this is a technology that should be in every clinician’s hands,” said Tej Shah, Managing Director at Accenture.

Shah shared data showing that, at some organizations, AI scribes have reduced physician burnout by 20 per cent while decreasing time spent on documentation by approximately 10 per cent. That additional time, he said, allows clinicians to focus on what matters most—caring for patients.

AI is also transforming patient care in other ways. Dr. Andy Smith, President and CEO of Sunnybrook Health Sciences Centre, highlighted how AI integrated with radiation therapy systems is reducing the number of treatments many cancer patients require.

“If instead of coming in [for treatment] 30 times, you only have to come five times, that is a profound leap forward in terms of the positive patient experience,” said Smith.

Despite its potential, AI adoption presents challenges. With countless new technologies entering the market, choosing the right solutions can be overwhelming. At the same time, healthcare organizations continue to operate under significant financial pressures and must carefully prioritize investments.

Karen Oldfield, President and CEO of Nova Scotia Health, stressed that return on investment must remain a key consideration.

“The bottom line for any healthcare system is that the benefits of AI also need to deliver monetary value,” she told attendees, noting that every dollar saved through greater efficiency can be reinvested into patient care.

Throughout the remainder of the conference, presenters explored real-world examples demonstrating how AI is improving efficiency while generating measurable savings.

Increasing long-term care capacity in Nova Scotia

During one session, Glenda Keenan, Senior Director of Continuing Care at Nova Scotia Health, described the province’s partnership with Strata Health to implement real-time scheduling software for long-term care beds.

The platform tracks every long-term care bed across Nova Scotia, identifies the types of patients each bed can accommodate and continuously analyzes waitlists to prioritize placements.

Peter Smith, President of Strata Health VitalHub Corp., said the software has significantly improved system capacity.

“You’re literally creating thousands of days of capacity in the system,” said Smith.

Keenan explained that Continuing Care Nova Scotia coordinated more than 3,400 long-term care beds last year. Before implementing the platform, staff managed placements manually using phone calls, fax machines and email.

“We had no line of sight on what was happening at the provincial, local or zone level, so it was imperative that we move forward with a better solution,” she said.

Since introducing the system in 2022, the median time required to fill a vacant bed has dropped from 10.7 days to six days—a 43 per cent improvement. Keenan estimates the economic value of the initiative at approximately $845,000.

She added that the system has improved not only coordination, but also strategic planning by providing better access to data.

“It has supported our ability, as well as the government’s, to make informed decisions because we now have much better insight,” she said.

Real-time mental health monitoring at CAMH

Another presentation highlighted how the Centre for Addiction and Mental Health (CAMH) has implemented Oracle Fusion Cloud Applications to create a centralized operations management system.

David Rotenberg, Chief Analytics Officer and Operations Director at the Krembil Centre for Neuroinformatics at CAMH, said the platform enables clinicians to monitor patient progress with unprecedented precision.

“We have dashboards embedded directly into patient charts that show whether people are getting better or not,” he said.

The dashboards support approximately 50 different care pathways, including psychosis, dementia and depression.

“You name it, we have a pathway for it.”

Rotenberg said the real-time insights help clinicians identify when patients require additional support and allow for earlier, more timely interventions.

Reducing wait times and improving patient experience in British Columbia

Natasha Kumari, Growth and Marketing Specialist at Thrive Health, described how her company has helped streamline referral and intake processes for healthcare organizations across British Columbia.

Through interviews and workflow assessments, Thrive identified several common challenges, including incomplete referrals, missing patient information and time-consuming follow-up calls.

“Some of the things we found were that there are a lot of incomplete referrals and information coming into the clinic, which leads to follow-up calls, chasing information and spending valuable appointment time collecting medical history that could have been provided beforehand,” Kumari said.

To address these challenges, Thrive implemented its Thrive Clinical platform at an ENT clinic in British Columbia.

The platform automates much of the information traditionally entered manually. Patients complete their medical history before arriving for their appointment, and the system generates additional questions based on individual responses while automatically sending reminders for incomplete forms.

“Because patients present with different diagnoses and conditions, the system can trigger additional follow-up questions depending on the patient’s responses,” Kumari explained.

Previously, clinicians spent an hour with every new patient simply reviewing medical history. According to Kumari, those sessions have now been virtually eliminated.

The platform also supports patients while they wait for specialist appointments by providing referral updates, personalized recommendations for managing their condition and opportunities to report changes in their health status.

Rather than relying on a static waitlist, Thrive Clinical creates a dynamic triage system that keeps information current for both patients and care providers.

“The idea is to automate the small, manual tasks that take up so much time, provide the right information to the right person at the right time, and help patients move through care faster,” Kumari said.

The post Where AI is making the biggest impacts in healthcare appeared first on Hospital News.

How Competitive Is Pediatric Residency? Match Rates & Acceptance Factors

Edusehat — Wed, 01 Jul 2026 00:30:07 +0700

This blog was originally published on September 23rd, 2019, and updated in August of 2024.

How to Match into a Pediatrics Residency Program

Residency application season is here and today we are looking at what it takes to match into a Pediatrics Residency Program! The NRMP conducts annual surveys to determine what factors Program Directors consider when deciding which candidates to interview and rank for their pediatric residency program. The results were compiled into a “at a glance” tool, which is extremely helpful in gauging where your application packet stands compared to the applicant pool, and whether adding additional components, like a letter of recommendation (LOR) in the specialty, could give you a leg up as programs review your application. Many students who are researching, “how competitive is pediatric residency” use NRMP data to evaluate pediatric residency competitiveness and improve their pediatric residency match chances.

The most recent program director’s survey was conducted following the 2024 residency match cycle. We recommend taking a look at the specialty you’ll be applying to before submitting your ERAS application packet. The information below can also help you better understand the application process and make informed decisions throughout medical school.

Selection Criteria for an Interview for Pediatrics Residency

A passing Step 1 score and your MSPE/Dean’s Letter rank highly among the most important residency matching factors pediatric program directors cite when granting interview invitations. Step 1 pass was cited as most important, and notably any USMLE/COMLEX failure was ranked as the #5 most important factor.

Step 2 Scores

Interestingly, 17% of program directors said that they do not consider Step 2 scores at all, and an additional 36% said they prefer, but do not require a score. An additional 32% said they require a pass, and only 15% said they require a target score.

The Step 2 score does seem to be more important for IMG applicants, but still only 26% of directors require a target score.

It should be noted that while Step 2 scores don’t seem to be a major concern for directors when selecting whom to interview, 59% of directors still cite them as an important factor for ranking. This still falls behind other aspects such as interactions during the interview and interpersonal skills, but should be kept in mind.

Students interested in competitive pediatrics residency programs often ask about the average Step 2 score for pediatrics residency, though most programs still evaluate applicants holistically. For students aiming for a 90th percentile score, strong shelf exam performance can help strengthen an application.

Step 2CK – Timing of Taking the Exam

Only 47% of programs required applicants to have passed Step 2 CK at the time of application. This means that for many programs, though certainly not all, you may wait to take Step 2 CK until after you have submitted ERAS.

Failing Step 1 or 2 will hurt you, but won’t sink your chances:

Failing any step of the USMLE is a BIG deal in ANY specialty, and pediatrics is no exception. So, if you’ve failed an exam, you can still apply, but you should consider applying to MANY (40+) programs. 79% of programs reported that a Step 1 failure was seldom an important factor when considering whom to interview.

Types of Applicants Considered:

International Grads (U.S. and non-U.S.) have more difficulty matching in pediatrics than domestic applicants, but it’s not impossible: Approximately one-third (31%) of programs consider interviewing U.S. IMGs on a routine basis, and approximately half (53%) seldom do; only 16% never do. For non-U.S. IMGs, chances are worse, but still possible: 21% routinely interview and rank non-U.S. IMGs, 49% seldom interview and rank them, while 30% never do.

We recommend doing your homework to determine which programs may be more likely to consider IMGs to maximize your chances of success. IMGs who have completed their training and residencies abroad may consider applying to work in Tennessee under the new HB1312 law to bypass the need for redundant residencies. Applicants should also research IMG friendly pediatrics residency programs to identify institutions with a history of interviewing international graduates.

Osteopathic graduates have a good chance of matching: 71% of programs will routinely interview DO applicants, while only 7% never do. This still pales in comparison with U.S. Seniors (91%), so it is still important to do your research to identify these programs so that you can best increase your chances of matching. This is encouraging for applicants pursuing pediatrics residency for DO students.

Pediatrics Residency Interviews

Understanding what program directors look for in pediatrics applicants can help you prepare more effectively for interview season.

Interviewees Get Ranked!: If you receive an interview invitation, you are likely to be placed on a program’s rank list if you complete the interview. In 2024, the likelihood of matching for applicants who ranked just one program was 80%. Additionally, out of 206 applicants interviewed, 194 were ranked in the 2024 cycle. That likelihood approached 100% for applicants who ranked four contiguous programs. We recommend ranking at least 8-10 programs to be safe, so once you receive 10 residency interview invitations, you can feel okay with starting to prioritize and cancel as more come in given you are likely to get ranked.
Expect to receive interview invitations in September and October: If you have not heard from programs within several weeks of submitting your application, it may indicate that your application is less competitive for some pediatrics programs.
Expect to interview in October-December: Few interviews will occur in January or later, which means you should plan to have free time in October-December to maximize your chances of being off during peak interview season. For further discussion on how to prep for your interview, see our blog post on how to crush your residency interview: https://elitemedicalprep.com/preparing-for-your-residency-interview/

Certain factors less important for Pediatrics than for other programs:

Research isn’t particularly important for Pediatrics — again, it can strengthen an application, but was not in the top 10 most important factors listed by all PDs.
AOA status isn’t as important in Pediatrics as it is in other specialties. Again, it can certainly strengthen an application, and is likely important at more competitive programs, but is not expected or required to match.
Having an LOR from a pediatrician is definitely important, but is not quite as important as some other fields (e.g., neurosurgery, dermatology). This likely means that a strong application could easily feature LORs from 1-2 pediatricians with additional letters of recommendation for pediatrics residency from practitioners outside of the field.
Achieving awards or honoring clinical rotations in the desired specialty is not as important.

Summary for Pediatrics Residency Applications:

A solid pediatrics application would include a passing score on Step 1, a strong MSPE or Dean’s letter, at least one LOR from a pediatrician, and demonstrating strong interpersonal skills during your interview. A strong pediatric residency personal statement can further support your application to a pediatric residency training program.

Pediatrics is somewhat more IMG friendly, than other fields, but matching as an IMG is far from certain. Be sure to do your research to identify programs with active IMG residents and establish proper connections. Students frequently ask, “how hard is pediatric residency to match into,” but the overall pediatric residency match rate remains favorable compared to many specialties.

If you’ve failed an exam, you can still apply in Pediatrics, but you should apply very broadly and consider applying for a back-up specialty.

Expect Interviews in October-December. Reach out to programs you have high interest in if you haven’t heard from them by mid-November. Once you have about 10 interview invitations, you can consider being a bit more selective about which ones you actually take (but not before).

Good Luck! ☺

Be sure to review individual pediatrics residency requirements as you build your application strategy and evaluate the competitiveness of your application. If you need further or specialized assistance understanding which residency specialties you are best suited to apply to, you can contact us or visit our residency advising page to learn more about our pediatrics shelf exam tutoring services and other Elite Medical Prep residency advising services.

The post How Competitive Is Pediatric Residency? Match Rates & Acceptance Factors appeared first on Elite Medical Prep.

What to Bring to Step 1: Your USMLE Exam Day Checklist

Edusehat — Wed, 01 Jul 2026 00:30:06 +0700

For many medical students, the USMLE Step 1 is one of the most stressful and overwhelming experiences of medical school. However, with proper planning and organization, you can make test day feel more manageable. In this post we will not only provide you with a USMLE Step 1 checklist of what to wear and what to bring to Step 1, but also some tips and tricks on how to prepare in the 72 hours before your exam.

Looking for a more in-depth Step 1 testing day guide? See our USMLE Step 1 6-week and 10-week study calendars if you’re just getting started!

What Should I Do to Prepare in the Days Leading Up to My Step 1 Exam?

Preparation for Step 1 begins long before test day and following these Step 1 exam day tips and tricks can greatly help mitigate any anxiety about approaching test day.

Drive to the USMLE Step 1 Test Center 3 Days Before Your Test

Visit the Prometric test center three days prior to your scheduled test. Familiarize yourself with the transportation mode you plan to use on the actual exam day. If someone is providing transportation for you, kindly request their assistance. If you’re using a family member’s car, ensure you are comfortable with driving it. In the case of public transportation, utilize the same mode for your practice visit. This USMLE exam day preparation will help ensure a smoother experience on the day of your test. This will allow you to 1) prepare for any uncertainties – is there construction? Is the car low on gas? 2) have a clear idea of how long it will take you to get to the center on test day, and plan accordingly.

The Day Before Your Step 1 Exam

Stop studying by noon and take the afternoon to relax. Doing a short workout (nothing too intense!) can help tire your body out a little bit and make it easy to fall asleep. Also consider doing something relaxing or fun the evening before your test day to help unwind.

Charge Your Electronics

Even though you can use your phone between blocks during the break, I recommend keeping your phone off the whole time you’re in the center. However still bring it with you so that you can make calls in case of emergencies or coordinate your pick up after Step 1 exam.

Make a Packing List

Using the Step 1 exam day packing guide below, make a list of Step 1 exam day essentials to pack before your test a few days before and pack them the evening before!

What Should You Wear on Test Day?

One of the most common questions is what should I wear on my Step 1 test day? Comfort, comfort, comfort! The most important thing on test day is dress in a way that will allow you to focus on your test. Whatever outfit you are most comfortable in is the best outfit. We recommend that in the week leading up to your test day that you take a practice test in your “test day outfit” to make sure that it’s comfortable during the test.

For what you should wear to your Step 1 exam:

Shirt and Pants

we recommend a shirt without a collar and pants without pockets. Remember that every time you enter and leave the room you have to turn your pockets inside and out and collars have to be flipped up and down. Wearing clothes without collars or pockets will save you time on your breaks.

A Sweater

We also recommend wearing layers. Prometric test centers are often kept cool so having a light jacket will allow you to adjust between blocks if you find yourself getting cold or overheating. Remember however that this layer cannot be taken off while in your exam room, but it might be handy to have in your locker.

Socks/ Shoes

For shoes, wear something comfortable. If you might want to slip your shoes during the test you may want to consider wearing slippers you can slip off during testing.

Accessories

Skip them! Don’t wear any jewelry, watches, etc. on test day.

What Should I Bring to Prometric for Step 1?

It’s really essential to have a list of what to bring with you on your Step 1 test day and just as important to know what not to bring. Here is a list of USMLE test day requirements you should pack with you:

Your Scheduling Permit

You must have your scheduling permit from registration with you on test day. If you forget your scheduling permit you won’t be allowed to test

Government Issued Form of ID

Bring two forms of ID, such as a passport and driver’s license. Remember that the required ID for Step 1 must be government issued and have your photo and signature. Although you only need one, having a backup never hurts if you have one that you can bring.

Bring Snacks and Drinks to Your Step 1 Exam

You are allowed to bring snacks and drinks to Prometric. Bring your snacks in a clear plastic bag and your drink in a clear bottle. They will be stored in a locker during testing. For your snacks for the Step 1 exam, make sure to pack plenty of high nutrition snacks with you! Some of my favorites included: yogurt, granola bars, and hummus! Make sure to pack sweet and salty snacks – you’ll never know what you’ll be craving between blocks.

Medications

If you’re on any prescription medications make sure to take them with you and take them as prescribed. In addition, if you are prone to upset stomach, headaches, allergies and know you may need to take a medication during the test make sure to pack those too.

Other Things to Consider

Depending on Covid 19 guidelines and testing center policies, you may need to show your vaccine card, a negative test, or wear a mask during your USMLE Step 1 exam. Make sure you call your Prometric center or look online to see what the current guidelines are as well as any other Prometric Step 1 rules.

Although you are allowed to bring notes, I strongly recommend against bringing any notes to test day. This often causes more stress for students. You can never predict what question will be next and it is often tempting to look up answers to questions you were uncertain on. However, this may just shake your confidence and is not worth it!

What to Expect at Prometric?

They will give you headphones if you want them:Yes, Prometric provides headphones for test takers. The headphones are noise-canceling and cover your entire ear. They are provided to block out external noise and distractions.

Check in can take time: An important part of our Step 1 arrival instructions is to get to your prometric testing center at least 30 minutes early to ensure you start your test on time

Watch the prometric preparation video: On Prometric’s website you can find a video that walks you through some of the basics of Prometric testing centers. Click this link (link: https://www.prometric.com/test-takers/what-expect ) to learn more about what to expect on test day and the items allowed at Prometric. This will also let you know about any prohibited items that you can’t bring to test day.

Preparing for the USMLE Step 1 can be a daunting experience. But with proper planning and organization, you can make the test day experience more manageable. With these tips on how to prepare and what to bring to USMLE Step 1 test day – you can cross one more thing off your list! Remember – make sure you dress comfortably, bring only necessary items, and review the Prometric testing center rules beforehand.

Good luck on your USMLE Step 1 test! If you’re looking for help preparing for your USMLE exams, consider enlisting the help of an Elite Medical Prep USMLE Step 1 tutor! Schedule a complimentary consultation today to learn more about how we can help you succeed!

The post What to Bring to Step 1: Your USMLE Exam Day Checklist appeared first on Elite Medical Prep.

The Ultimate 4-Week COMLEX Level 2 Study Plan

Edusehat — Wed, 01 Jul 2026 00:30:05 +0700

With only 4 weeks left until your upcoming COMLEX Level 2 exam, it’s time to get organized and crack down on studying. We’ve got you covered with our ultimate COMLEX Level 2 Study Plan! We provide a recommended week-by-week plan to review the content that will be tested on the COMLEX Level 2 exam.

This study plan can serve as a perfect template and jumping-off point for you to customize based on your specific needs while studying for COMLEX Level 2. It can be used as a COMLEX Level 2 study schedule alone, or to combine studying for the COMLEX Level 2 and the USMLE Step 2 at the same time.

We recommend taking osteopathic principles and practice before the COMLEX exam, though some students may prefer a different testing order. For more information on which exam you should take first, read our post on the topic.

Edit the organ system you review on different days based on the weak areas discovered during your self-assessment or customize with your favorite resources. For instance, if you performed well in OB/GYN and pediatrics on your second self-assessment but are still having trouble with cardiology, consider doing just one block of pediatrics and OB/GYN on the following day in favor of another cardiology block.

Make sure you keep track of your COMLEX Level 2 practice exams and are seeing a steady trend of improvement throughout your dedicated study period. Suppose after your third practice exam on week 3 you still are not close to passing and have been plateauing or seeing irregular practice exam scores. In that case, it may be time to consider delaying your COMLEX Level 2 exam until you are better prepared.

Tips for Success Using the COMLEX Level 2 Study Planner

Embarking on the path to ace the COMLEX Level 2 exam requires dedication, strategic planning, and a little insight into what works best. Here are some essential tips for making the most out of the COMLEX Level 2 Study Planner:

Customize Based on Your Needs: While the Ultimate COMLEX Level 2 Study Plan provides an excellent foundation, ensure you adapt it to highlight your weak points and strengths.
Diversify Your Resources: The COMLEX Level 2 exam is comprehensive. Lean into a variety of study materials, QBank questions, and practice tests. Not only does this provide a more rounded understanding, but it also exposes you to different question formats and presentations. Read our blog post dedicated to this topic to help you select a good variety of resources, without getting too overwhelmed.
Schedule Regular Self-Assessments: Recognizing areas that need additional attention is crucial. Periodic self-assessments not only highlight these areas, but also give you a sense of progress and a boost in confidence.
Stay Consistent: The key to retaining information and staying on track is consistency. Set aside dedicated study hours each day and stick to your plan, adjusting as necessary based on your self-assessments.
Seek External Guidance: Engage with COMLEX Level 2 resources like Elite Medical Prep tutoring for personalized study strategies, effective time management techniques, and expert advice. Our tailored approach can be invaluable in ensuring that you cover all necessary content effectively.
Review Foundational Concepts: COMLEX Level 2 doesn’t only test advanced knowledge. It’s crucial to bridge any gaps from Level 1 and reinforce foundational concepts to ensure you have a holistic understanding.
Practice, Practice, Practice: The more questions you tackle, the more comfortable you’ll be on exam day. Use the planner to allocate time for mock tests and practice questions, honing your skills and understanding your mistakes.
Stay Updated and Informed: Keep abreast of any changes in the exam scoring or syllabus. Elite Medical Prep and other resources often provide insights and updates that can be beneficial.
Mind Your Well-being: Remember that it’s not just about hitting the books. Ensure you’re getting enough sleep, maintaining a balanced diet, and incorporating stress-relieving activities into your routine.
Stay Positive and Trust the Process: Lastly, believe in your preparation and the study plan. While challenges are part of the journey, staying motivated and positive can significantly impact your performance.

By integrating these tips into your study routine and making the most of the COMLEX Level 2 Study Planner, you’ll be well-positioned for success on exam day.

How to Balance COMLEX Level 2 and Step 2 CK Preparation

Many osteopathic students choose to take both COMLEX Level 2 CE and Step 2 CK during the same study period. Fortunately, there is significant overlap between the two exams, so much of your clinical knowledge review will benefit both tests.

We generally recommend taking Step 2 CK first and then using the remaining days in your COMLEX Level 2 study timeline to focus on osteopathic principles and OMM-specific content. While the core clinical material is similar, COMLEX includes additional emphasis on osteopathic diagnosis, treatment concepts, and patient scenarios that require a dedicated osteopathic principles review.

If you’re wondering how to study for Level 2 CE while also preparing for Step 2 CK, it’s best to keep your resources focused. A solid question bank, targeted OMM review materials, and a few high-yield COMLEX Level 2 prep resources are often enough. Many students find that too many resources create confusion rather than improvement.

One effective strategy for dual board exam preparation is to dedicate separate study blocks to OMM topics such as viscerosomatics, Chapman points, autonomics, and counterstrain while continuing your broader high yield clinical review.

If you need additional guidance with your study plan or osteopathic board exam prep, consider reaching out to work with one of our COMLEX Level 2 tutoring experts.

How to Analyze Practice Exam Performance During COMLEX Level 2 Preparation

Practice exams are one of the most valuable tools available during clinical rotation exam prep, but it’s important to look past the score report. Take the time to review missed questions and identify why you missed them. Were there content gaps? Did you misread the question? Were you rushing? These patterns often reveal the common mistakes during board prep that can hold students back.

As you continue studying, use practice exam results to adjust your schedule. If you’re struggling with a specific subject area, dedicate more time to it during your daily review sessions. Students frequently ask how to study multiple subjects in one day, and practice exam data can help determine where their time is best spent.

Practice tests also help answer questions such as how long to study for osteopathic board exams by showing whether your scores are improving consistently. If scores fluctuate dramatically between assessments, it may point to issues with stamina, retention, or test-taking strategy rather than knowledge alone.

Finally, remember that learning how to avoid burnout during dedicated study is just as important as improving your score. Be sure to leave enough time between your final practice exam and test day to review weak areas, build confidence, and avoid last-minute cramming.

Crafting Your Ideal COMLEX Level 2 Study Schedule with Elite Medical Prep

Making the most of your preparation time for the COMLEX Level 2-CE can feel overwhelming. With Elite Medical Prep, you’re not alone in this journey. Our team is equipped to help you strategize and optimize your study plan for maximum success.

Why Choose Elite Medical Prep for Your COMLEX Level 2 Preparation?

Personalized Approach: Every medical student has their strengths and weaknesses. We analyze where you stand and tailor a COMLEX or dedicated board study schedule specifically to your needs.
Time Management Guidance: Confused about how long to study for COMLEX Level 2? Our expert team will help you allocate your time effectively, ensuring every topic gets the right attention.
Resource Recommendations: We don’t just help you with a Level 2 COMLEX study plan; we also recommend the best resources to complement your preparation.
Practice Questions Review: Hone your skills with focused practice questions. Understand your mistakes and refine your approach for the real deal.
Holistic Preparation: It’s not just about rote learning. Learn how to approach challenging material, assess yourself periodically, and stay on track with our guidance.

A Few Tips from Our Experts for COMLEX Level 2-CE:

Self-Assessment: Always start your COMLEX Level 2 preparation by understanding where you stand. Recognize your strong and weak areas to allocate study time efficiently.
Diverse Resources: Don’t rely solely on one resource. Diversify your materials and practice questions for more rounded preparation.
Consistent Practice: Consistency is key. Regularly solve practice questions and periodically take mock tests to measure your progress.
Mind the Gaps: While preparing for COMLEX Level 2, ensure you bridge the knowledge gaps from Level 1, reinforcing foundational concepts.

Navigating Medical Milestones with Elite Medical Prep

Embarking on a medical journey is filled with both challenges and rewarding milestones. Elite Medical Prep stands ready to illuminate your path, ensuring you excel at every significant turn, whether in medical school or transitioning into specialized domains. Here’s how we make a difference:

USMLE Step 1 Support: Establish a robust foundation in your medical school curriculum and step into the 3rd year with unmatched expertise, courtesy of our USMLE Step 1 preparation.
USMLE Step 2 CK (Step 2) Excellence: Transitioning from foundational knowledge to clinical applications is seamless with our focused strategies for USMLE Step 2CK.
USMLE Step 3 Strategy: We aid in refining your clinical decision-making skills, ensuring your USMLE Step 3 performance is top-notch.
COMLEX Level Suite:
1. Level 1: Build your foundation with the first of the COMLEX level examinations.
2. Level 2-CE: Dive deeper into clinical expertise, especially in areas like Level 2 CE, ensuring you’re ready for challenges in both COMLEX and USMLE spheres.
3. Level 3: Perfect your clinical application skills, setting the stage for your future medical endeavors.
NBME Shelf Exams Insight: As you progress in your medical school journey, shelf exams become pivotal. With our strategies, approach each shelf exam with confidence and precision.

Navigating the intricate maze of COMLEX and USMLE becomes significantly easier with Elite Medical Prep by your side. Catering specifically to the unique needs of students in colleges of osteopathic medicine, we’re dedicated to ensuring that every year, especially the challenging 3rd year, becomes a foundation for your future success.

Learn More with Elite Medical Prep

Having a COMLEX Level 2 study plan is important to keeping yourself organized and on track while studying for your exam. For more help customizing your study plan, reviewing your incorrect questions on practice tests or QBank questions, or talking through challenging material, consider enlisting the help of a 1-on-1 COMLEX Level 2 tutor!

Your success in COMLEX Level 2-CE is our goal. To learn more about how we can help you in preparing for COMLEX Level 2 and get deeper insights tailored to you, contact Elite Medical Prep today!

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The Physicians Foundation Survey Finds Lack of Access to Nutritious Food is Undermining Chronic Disease Care and Prevention

Edusehat — Tue, 30 Jun 2026 20:10:04 +0700

New survey examines how gaps in access to healthy foods are impacting chronic disease outcomes and what it will take to close them.

BOSTON, June 30, 2026 — The Physicians Foundation today announced findings from its 2026 Survey of America’s Physicians: Impact of Healthy Food on Chronic Disease Care and Prevention, revealing a critical disconnect in U.S. healthcare: while physicians recognize nutrition as foundational to preventing and managing chronic disease, the healthcare system is not fully equipped to enable its use – leaving many patients unable to follow clinical guidance.

Chronic disease may be diagnosed in the exam room, but too often it is shaped by whether patients can access nutritious food and make healthy lifestyle choices. At a moment when national attention is increasingly focused on nutrition as a core tool for improving health, a survey of more than 1,000 U.S. physicians, conducted in collaboration with Medscape, finds that gaps in access to key drivers of health – particularly healthy food – are contributing to worsening control of chronic conditions and limiting physicians’ ability to deliver effective care.

Key findings point to widespread challenges affecting both patients and physicians – and potential solutions to address these issues:

More than three quarters of physicians (78%) encounter patients at least monthly whose chronic disease care is compromised by a lack of access to healthy food.
Nearly half of physicians (45%) report that patients with chronic diseases frequently or almost always experience worsening control of symptoms primarily due to drivers of health, rather than clinical factors alone.
68% of physicians support insurance coverage for healthy food benefits to make it easier for patients with chronic conditions to make healthier lifestyle or diet choices.

“Physicians understand that nutrition is a critical clinical intervention to prevent and manage chronic disease,” said Dr. Gary Price, President of The Physicians Foundation. “When patients lack access to the healthy foods they need and therefore cannot follow clinical recommendations, it undermines care and leads to worse outcomes. As our survey makes clear, insurance coverage for nutritious food benefits is a crucial tool in enabling patients to follow their doctors’ guidance to eat real food.”

The Physicians Foundation’s findings reinforce the growing recognition across the country – including by leadership at the federal and state level – that improving access to nutritious food is essential to addressing diet-related and other chronic diseases and reducing healthcare costs. Aligning healthcare coverage, policy frameworks, and community resources to support nutrition-based care will be critical to improving outcomes and supporting both patients and physicians.

The Physicians Foundation is advancing research to better integrate drivers of health into care delivery and improve chronic disease outcomes.

Read the full survey results and methodology here.

About The Physicians Foundation

The Physicians Foundation is a public charity seeking to advance the work of practicing physicians and help them facilitate the delivery of high-quality health care to patients. As the U.S. health care system continues to evolve, The Physicians Foundation is steadfast in strengthening the physician-patient relationship, supporting medical practices’ sustainability and helping physicians navigate the changing health care system. The Physicians Foundation pursues its mission through research, education and innovative grant making that improves physician wellbeing, strengthens physician leadership, addresses drivers of health and lifts physician perspectives. For more information, visit www.physiciansfoundation.org.

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Two Proteins with Opposing Functions Found to Support Healthy Skin Maintenance

Edusehat — Tue, 30 Jun 2026 09:50:09 +0700

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.”

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BIO 2026: Industry PDUFA negotiators describe their agreement with FDA

Edusehat — Tue, 30 Jun 2026 02:45:03 +0700

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.

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CCRM Ireland Would Be Established to Hasten Translation of Advanced Therapies Into Patient Treatments

Edusehat — Tue, 30 Jun 2026 02:40:12 +0700

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.

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Shilpa Commissions Integrated ADC Drug Substance GMP Manufacturing Facility

Edusehat — Tue, 30 Jun 2026 02:40:11 +0700

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.”

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Iron Accumulation Drives Neurodegeneration via Chronic Stress Pathway

Edusehat — Tue, 30 Jun 2026 02:40:10 +0700

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

Edusehat — Tue, 30 Jun 2026 02:40:09 +0700

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

Edusehat — Mon, 29 Jun 2026 23:05:10 +0700

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.

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David Crowe, MD Joins Optima Dermatology’s Macedonia, OH Practice

Edusehat — Mon, 29 Jun 2026 23:05:07 +0700

Patients in Macedonia, OH Will Have Access to Nearly Two Decades of Dermatology Experience for a Wide Range of Skin Conditions

Board-certified dermatologist Dr. David Crowe is joining Optima Dermatology’s Macedonia, OH practice, expanding access for patients to treat skin conditions ranging from skin cancer to psoriasis, with a special clinical interest in autoimmune conditions.

Since 2007, David Crowe, MD, has treated patients 8 years and older for a variety of skin issues, drawing on his lengthy experience in dermatology and medicine.

Dr. Crowe completed his dermatology residency at the University of Cincinnati Medical Center in Ohio after graduating with his Doctor of Medicine from the University of Cincinnati College of Medicine, where he was elected to the Alpha Omega Alpha Medical Honor Society.

Since his residency, Dr. Crowe has been active in healthcare advancement and advocacy, authoring more than 15 scientific publications. He worked at MetroHealth Systems for 15 years as Department Chairman, was heavily involved in resident education, and twice received the Brian R. Davis Award for Excellence in Resident Education.

He currently holds the rank of Associate Professor at the Case Western Reserve School of Medicine, and is a member of the American Academy of Dermatology, the Ohio Dermatological Association, and the Cleveland Dermatological Society.

Industry-Leading Dermatology Care Without Question

Dr. Crowe is excited to deliver personalized and comprehensive dermatology care to patients in the Macedonia area, especially for complex skin issues that require dedicated treatment plans and an understanding ally.

Dr. Crowe is a perfect fit for the Optima Dermatology culture with his commitment to delivering an unparalleled patient experience and the highest quality of care.

We recently had the opportunity to learn more about Dr. Crowe, his approach, and the path that led him to become a board-certified dermatologist.

What made you decide to pursue a career in dermatology? What did the path to get there look like for you?

Part of my interest in dermatology began in my teenage years when I suffered from severe hand

eczema, which gave me perspective into how much skin conditions can affect your life. After college and medical school I was fortunate enough to earn a dermatology residency position in Cincinnati. I moved back home to Cleveland in 2011 and have been practicing here ever since.

What is your favorite aspect of being a board-certified dermatologist?

I truly enjoy the challenge and satisfaction of solving a complex medical problem, and

dermatology is filled with unusual challenges and rare conditions. I also get to follow patients over time with yearly skin checks and get to form great relationships with them.

What is your approach to treating your patients?

I try to make a personal connection with every patient that I meet. Sometimes a first visit to a

doctor can make people nervous, especially when dealing with a skin problem. Building trust with patients will make it more likely that we can arrive at a solution to their problem and follow through on it.

What is your number one tip when it comes to taking care of your skin?

Don’t expect the world from over-the-counter products. They can be quite costly, often have

many unnecessary ingredients, and may have incorrect claims about how well they work.

What is one thing you want your patients to know about you?

My hands have always been a bit chilly. Warm heart, I promise!

What do you like to do when you are not in the office treating patients?

I spend time with my wife and two children. We love watching movies and spicy food!

What to Expect From Dr. Crowe and His Team

Patients visiting with Dr. Crowe and his team at Optima Dermatology will receive the highest quality care backed by decades of advanced training and experience.

Dr. Crowe is currently accepting new patients at our Macedonia, OH location. Request an appointment or call us at (330) 306-9844.

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Emergency department wait times in Canada: Insights from a health system perspective

Edusehat — Mon, 29 Jun 2026 19:25:10 +0700

How gaps in the system show up in emergency department wait times

Emergency department (ED) wait times are rising across Canada. It’s a shared reality felt by both patients seeking care and health care workers trying to deliver it. And yet, it would be a misdiagnosis to frame this issue as an ED problem.

According to a new report from the Canadian Institute for Health Information, Emergency department wait times in Canada: Insights from a health system perspective, the reality is that the ED is often where symptoms of a strained system become visible, not where they begin. Wait times are driven by both increasing patient complexity and a system that is struggling to manage the demand. When Canadians can’t access timely community care, can’t be admitted to hospital beds, and can’t be discharged to home care or long-term care (LTC), those unmet needs show up in ED wait times.

Patient flow through the ED

Unlike a linear queue, patient flow through the ED is dynamic. When a patient presents to the ED, the wait is not one queue, but rather several that overlap and are spread across triage, admission and discharge. Each phase is measured as a separate wait time and can involve its own potential delay. Patients may see a crowded waiting room and assume it’s a busy day, but the data reveals that in most cases, the waits are systemic, not simply the result of a busy day.

When patients present to the ED, care is prioritized by urgency rather than arrival time, meaning the sickest patients are seen first. In 2024–2025, half of ED patients in Canada waited just under 2 hours to be assessed by an ED physician, while 1 in 10 patients waited more than 6 hours.

While the delays in the ED waiting room may feel the most immediately visible to a patient, the most significant bottlenecks affecting flow through the ED occur after the decision to admit the patient is made. For the 1 in 10 ED patients who are admitted to acute care, waiting for an inpatient bed accounts for nearly two-thirds of a patient’s total ED stay. And if a patient can’t be moved elsewhere, the ED becomes the buffer and is forced to absorb the overflow.

From a patient’s perspective, this can feel like being stuck. They know they are sick enough to be admitted, but without a clear sense of where they will go or when they will move, there is little sense of clarity about how their care plan may progress.

From a staffing perspective, this bottleneck creates a domino effect throughout the hospital. Patients who are admitted and waiting for a bed still require monitoring, treatment and nursing care. This means that ED staff are caring for admitted patients while simultaneously trying to attend to new emergencies.

A system under strain

Dr. Paul Parks — an emergency medicine physician in Medicine Hat, Alberta — describes what is happening in EDs across Canada as a national crisis of the health system. “We’ve optimized everything. We’ve taken all the elasticity out of the system, but the tap is still flowing,” he said. “There’s a crisis: insufficient connected community resources, and little continuing care and long-term care.”

Dr. Parks’ metaphor points to an overextended health system that was designed for different pressures than the ones it faces today. The bottlenecks in the ED are not isolated; they are a result of co-existing and compounding pressures driven both upstream and downstream of hospitals themselves.

Within the hospital, on the downstream side, beds are not available because of patients waiting to access other services such as LTC or home care. These patients, designated as alternate level of care, are medically ready to be discharged but can remain in the hospital a median of 44 days while waiting for community supports. This limits the hospital’s ability to admit new patients from the ED.

Outside of the hospital, on the upstream side, patients presenting to the ED are increasingly older and have more medically complex conditions. In Canada, adults age 55 and older are among the most frequent ED patients. In 2024–2025, nearly one-third of ED visits involved patients with multiple comorbidities, meaning they required more complex assessment, testing and care coordination.

While some of this stems from an aging population, it also reflects gaps in access to primary and community care. Without access to these supports, people may turn to the ED as their entry point into the health system.

In some instances, patients present with conditions that could have been managed in the community if timely care had been available; in others, lack of access to primary care means those conditions become more complex and difficult to treat by the time patients arrive in the ED. This is particularly true for people living in the lowest-income neighbourhoods, who account for nearly half of all ED visits and often face greater barriers to accessing community care — often relying on the ED to fill the gap.

Together, these upstream and downstream factors cause unmet needs throughout the system to show up in ED wait times.

The ED as a warning signal

The ED serves as a sign of how the wider health system is performing. It becomes the face where the effects of insufficient community care and delayed discharge pathways are most immediately seen, not because the ED is the source of these pressures, but because it’s where these pressures converge in real time.

Improving ED wait times means looking beyond the ED itself, and instead to the broader health system within which it operates.

Canadian Institute for Health Information. How gaps in the system show up in emergency department wait times.

Emergency department wait times reflect pressures across Canada’s entire healthcare system

Emergency department (ED) wait times have become one of the most visible indicators of the challenges facing Canada’s healthcare system. But according to a new report from the Canadian Institute for Health Information (CIHI), the causes of long waits extend far beyond the walls of the emergency department itself.

Rather than viewing emergency department overcrowding as an isolated issue, the report highlights how patient flow through the ED reflects the performance of the entire healthcare system—from access to primary care and diagnostic imaging to the availability of hospital beds, home care and long-term care.

In 2024–2025, Canadian emergency departments recorded approximately 16.1 million visits, representing about 89 per cent of all ED visits across the country. While most patients were treated and discharged home, nearly 12 per cent required admission to hospital, placing additional pressure on already strained inpatient units.

Perhaps most concerning, more than 1.2 million emergency department visits ended before patients were ever assessed by a physician. Although these cases were not included in the report’s analysis, they serve as an important indicator of overcrowding and prolonged wait times.

Understanding where delays occur

The report examines patient flow through three key stages of an emergency department visit: triage and registration, time under clinical care and disposition, when patients are discharged, transferred or admitted to hospital.

Each stage offers insight into different system pressures.

Patients are first assessed using the Canadian Triage and Acuity Scale (CTAS), which prioritizes care based on the severity and urgency of their condition. Once under care, they may require physician assessment, diagnostic imaging, laboratory testing or consultations with specialists before a treatment decision can be made.

For many patients, the longest delay occurs after the decision has already been made to admit them to hospital. When inpatient beds are unavailable, admitted patients remain in the emergency department, continuing to require nursing care, monitoring and treatment while occupying valuable clinical space. This creates a ripple effect throughout the department, slowing care for newly arriving patients.

The pressures begin long before patients arrive

The report emphasizes that many emergency department visits are driven by limited access to healthcare services elsewhere in the system.

Nearly one in five Canadian adults does not have a regular primary care provider. Even among those who do, timely access remains challenging. Only about one-quarter report being able to see their provider the same or next day, while evening and weekend appointments remain difficult to obtain.

Access to specialist consultations and diagnostic imaging also contributes to emergency department demand. Many patients face months-long waits for MRI or CT scans, while others experience lengthy delays accessing home care or community services. As health conditions worsen, the emergency department often becomes the only available option.

An aging population further compounds these challenges. Older adults are more likely to live with multiple chronic illnesses requiring urgent assessment, symptom management and diagnostic testing that may not be readily available outside hospital settings.

As emergency physician Dr. Simon Berthelot notes in the report, strong primary care can prevent many chronic conditions from deteriorating to the point where emergency care or hospitalization becomes necessary.

Solving emergency department overcrowding requires system-wide solutions

While emergency departments continue to introduce new models of care and improve internal processes, the report suggests meaningful improvements in wait times will depend on broader health system capacity.

Increasing access to family physicians, specialists, diagnostic imaging, home care and long-term care could reduce avoidable emergency visits while helping patients receive care earlier in their illness.

At the same time, improving hospital capacity and patient flow beyond the emergency department would allow admitted patients to move to inpatient units more quickly, freeing emergency resources for incoming patients.

Patient partner Cristyana Aloysious, whose perspective is included in the report, says the current system can be particularly challenging for people living with chronic illnesses who repeatedly return to the emergency department because they lack coordinated, ongoing care.

The report concludes that emergency department wait times should not simply be viewed as an emergency medicine issue. Instead, they provide a window into how effectively the healthcare system functions as a whole. Addressing delays will require coordinated investments across the entire continuum of care—from community-based services and primary care to hospital capacity and post-acute supports.

As Canada’s healthcare system continues to evolve, improving patient flow throughout the system—not just within emergency departments—may prove to be one of the most important steps toward reducing wait times and improving access to care for all Canadians.

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Gene therapy is changing how we treat disease

Edusehat — Mon, 29 Jun 2026 08:40:08 +0700

HN Summary

• Gene therapy is shifting medicine from treating symptoms to targeting disease at its genetic source, offering new hope for conditions such as Huntington’s disease, sickle cell disease and inherited neurological disorders.

• Advances in viral delivery systems and gene-editing technologies like CRISPR are accelerating the development of therapies that could slow or even prevent disease progression.

• Canadian researchers and biotechnology companies are playing a leading role in advancing gene therapy research, manufacturing and clinical innovation, with the potential to transform treatment for both rare and common diseases.

Gene therapy aims to target illness at its genetic source rather than manage symptoms. Here’s how the science works, and why researchers think it could transform medicine.

The neurosurgeon has no visual field. There is only a catheter, fed into the skull through a hole, no bigger than the tip of a crayon, and a set of coordinates that correspond to a target deep within the brain. A trajectory has been plotted from MRI scans taken with a stereotactic frame bolted to the patient’s head, a reference system that turns the brain’s interior into a 3D grid.

The catheter advances along a pre-plotted line toward its destination: the striatum — specifically, the subcortical structures key to movement and thought, and among the first destroyed by Huntington’s disease. Once there, a solution of engineered viruses called AMT-130 is infused in a continuous trickle over roughly 12 hours. On an MRI monitor, the surgical team watches the liquid bloom through the brain in real time.

All that — the catheter through functioning brain tissue, the hours-long neurosurgery, the viral solution — is just the delivery system. The real therapeutic payload is what that viral solution is carrying: a scrap of synthetic genetic material that promises, for the first time, a way to actually slow one of the most confounding neurological diseases. It also represents a proof of concept for gene therapy, an entirely different way of thinking about medicine.

So what is gene therapy, exactly?

Most conventional drugs are small molecules that circulate through the body and interfere with a protein or process that’s causing problems. Gene therapy works further upstream. Instead of dealing with the mess created by a faulty gene, it tries to intervene in the genetic instructions themselves, by delivering a healthy copy of a gene, silencing one that’s causing harm or, in more aggressive approaches, editing the DNA sequence directly, explains Rachel Harding, a molecular biologist and an assistant professor at the University of Toronto in the department of pharmacology and toxicology.

Huntington’s disease is an inherited neurodegenerative disorder caused by a mutation in a single gene. That mutation produces a toxic protein called huntingtin, which accumulates inside neurons and gradually kills them. The result is a crushing progression of symptoms — involuntary movements, trouble speaking and swallowing, cognitive impairment and psychiatric disturbances — that typically emerge in midlife and worsen over time. “There’s never a competition over which disease is the worst,” says Harding, whose work mapping the structure of huntingtin earned her the 2024 Nancy S. Wexler Young Investigator Prize. “But most neurologists agree that Huntington’s is one of the most devastating.”

Until recently, every existing treatment managed what Huntington’s does to a person, but nothing tackled the root cause or the underlying neurodegeneration. Then last fall, uniQure, a Dutch biotech company, released preliminary results from its AMT-130 trial. The therapy uses a viral vector to deliver DNA instructions into neurons, prompting cells to produce something called microRNA — basically, benign interlopers that piggyback on and interfere with the RNA molecules responsible for producing huntingtin.

Three years after treatment, patients showed a 75 percent deceleration in disease progression. Researchers also reported declines in a biomarker associated with neurodegeneration, which suggests the therapy may not just be masking symptoms but actually delaying the death of brain cells.

Wait — what’s a viral vector? Is that a virus?

Basically, yes. The delivery problem is one of the central challenges in gene therapy. We can’t just inject DNA and RNA into the bloodstream and expect them to slip inside the right cells — our immune systems are designed to detect and destroy foreign genetic material. So scientists often repurpose modified viruses as delivery vehicles, exploiting their natural ability to weasel their way into cells and deposit genetic instructions.

The most common are adeno-associated viruses, or AAVs, engineered to carry genetic cargo without causing disease. Different versions target different tissues. The AAV5 vector used in AMT-130, for instance, is particularly good at infecting neurons in the striatum.

Why are we suddenly hearing so much about gene therapy?

The idea for gene therapy has been around for decades: What if, instead of treating symptoms, doctors could fix disease at its genetic source? Early experiments in the 1980s and ’90s proved the concept was possible, including a landmark 1990 trial that treated a four-year-old girl born with a severe immune disorder. But the field was rocked in 1999, when 18-year-old Jesse Gelsinger, who had a rare metabolic liver condition, died after suffering a severe immune reaction during an experimental gene therapy trial. Research ground to a halt for years.

Then came a series of breakthroughs. The completion of the Human Genome Project in 2003 gave scientists, for the first time, a full map of human DNA. Less than a decade later, researchers Jennifer Doudna and Emmanuelle Charpentier revealed the gene-editing capabilities of CRISPR-Cas9, a bacterial defense mechanism they showed could be reprogrammed to locate and cut specific sequences in any genome with unprecedented precision. Faster, cheaper and easier to use than earlier technologies, it transformed the field almost overnight, and won the pair the Nobel Prize in Chemistry in 2020.

What kinds of diseases can be treated with this technology?

According to Janet Rothberg, a senior director with the Toronto not-for-profit Centre for Commercialization of Regenerative Medicine, gene therapy is most powerful for diseases caused by a single identifiable mutation — one broken gene, one clean target. There are roughly 7,000 such diseases that affect more than 400 million people worldwide. These include inherited blood disorders such as sickle cell and hemophilia, as well as a range of neuromuscular, neurological and rare pediatric disorders, from immune deficiencies to various forms of vision and hearing loss.

Is gene therapy the same as cell therapy?

“Not quite,” says Rothberg. “In gene therapy, we’re trying to deliver something that fixes the person’s own cells. In cell therapy, we’re actually giving them new cells.” Those cells, either modified or grown in a lab, are themselves the treatment. They’re meant to perform a job that can no longer be performed by the patient’s body.

CAR-T cancer therapy, for example, involves removing a patient’s immune cells, genetically engineering them to recognize cancer, and infusing them back into the body. That’s where the lines get blurry — many modern cell therapies also rely on genetic engineering. And both rely on genomics, the science of reading and mapping the genome, to identify their targets.

Why does gene therapy cost so much?

These therapies are extraordinarily expensive to produce, as each one is essentially handcrafted from biological material rather than synthesized in bulk. Manufacturing is heavily manual, R&D costs are enormous and patient populations for rare diseases are small, meaning costs can’t be spread across millions of prescriptions the way those for a common drug can. The first CRISPR-based gene therapy, a sickle cell treatment called Casgevy, costs U.S.$2.2 million per patient.

CCRM, which operates OmniaBio, Canada’s largest cell and gene therapy manufacturing facility in Hamilton, Ontario, is actively working to automate the manufacturing process to help drive costs down. “The power of these therapies can’t be limited to certain people,” says Rothberg.

What role is Canada playing in all this?

From research to manufacturing to clinical application, Canada has built a meaningful presence across the cell and gene therapy pipeline. Specific Biologics, a Toronto company spun out of Western University research, is focused on one of the central limitations of first-generation gene editing: many tools can effectively break genes, but are less good at repairing them precisely. Its platform, called Dualase, uses a two-cut editing system designed to remove and replace errant bits of code more accurately. In diseases caused by repeated stretches of faulty DNA, like Huntington’s, those paired cuts can excise the harmful repetitions entirely. “Gene-editing therapies that restore a genetic sequence precisely have been described as the ‘holy grail’ of gene therapies,” says Specific Biologics CEO Brent Stead.

Other Canadian companies are tackling different problems. Montreal-based Jenthera Therapeutics is developing a CRISPR platform that skips viral delivery systems entirely. The approach, which targets cancer, is designed with the aim of mitigating challenges related to both manufacturing complexity and adverse immune system responses in patients. Toronto’s Mediphage Bioceuticals, meanwhile, is working on synthetic DNA molecules that are easier to redose, a major hurdle with virus-based therapies, which typically can’t be readministered once the immune system recognizes the viral vector.

What happens next?

AMT-130 hasn’t been approved yet, and the trial is small. But the implications may extend far beyond Huntington’s. If scientists can safely deliver genetic instructions deep into the brain and silence a disease-causing gene, the same platform could potentially be adapted for a range of neurological disorders, including ALS and Parkinson’s. Many of these conditions have been hard to treat because the underlying proteins can’t be reached by conventional drugs — either they’re too structurally elusive for traditional pharmaceuticals to latch onto, or they’re tucked behind the blood-brain barrier. Gene therapy offers a possible workaround by targeting the genetic instructions upstream instead.

“It opens up the opportunity to drug the undruggable,” says Harding. “We could be in for an explosion of therapies, of new ways we can treat diseases.”

The pharmaceutical industry is watching closely. Gene therapy has largely targeted rare diseases, but the tools that made AMT-130 possible are now being pointed at some of the most common conditions in medicine.

In May, researchers published the preliminary results of a trial led by Verve Therapeutics, a biotech focused on applying gene editing to cardiovascular disease that was acquired last year by Eli Lilly for more than $1 billion. The study, published in The New England Journal of Medicine, showed that a single infusion of a gene-editing treatment reduced levels of LDL cholesterol — the bad kind — by as much as 62 per cent in patients with genetically high cholesterol, with effects that appear to persist over time.

Researchers hope the approach could eventually extend to anyone at risk of heart disease, which kills nearly 20 million people around the world each year. A one-and-done treatment for the leading cause of death globally would shift gene therapy from niche medicine to something much larger. nH

Caitlyn Walsh Miller writes about technology for MaRS. Hospital News has partnered with MaRS to highlight Canadian innovations in health.

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Global clinical trial reveals safest, most effective antibiotics for staphylococcal bloodstream infections

Edusehat — Mon, 29 Jun 2026 08:40:08 +0700

HN Summary

• An international clinical trial led by researchers including the Research Institute of the McGill University Health Centre found that cefazolin and benzylpenicillin are safer and as effective as cloxacillin for treating serious staphylococcal bloodstream infections.

• The findings, published in the The New England Journal of Medicine and The Lancet, showed the alternative antibiotics were associated with lower rates of kidney injury and, in some cases, lower mortality.

• Researchers say the results are expected to change international treatment guidelines and improve outcomes for patients with life-threatening bloodstream infections.

A landmark international clinical trial has identified the optimal antibiotics for staphylococcal bloodstream infections, a breakthrough that is set to reshape treatment for the life-threatening condition. The SNAP Trial found that the standard antibiotic, cloxacillin (called flucloxacillin in many countries), should no longer be the drug of choice to treat the infection, revealing that cefazolin and benzylpenicillin offer safer and equally effective alternatives to patients.

The Staphylococcus aureus Network Adaptive Platform Trial (SNAP Trial), led by researchers at the Peter Doherty Institute for Infection and Immunity (Doherty Institute) and the University of Newcastle in Australia, is the largest international clinical trial ever conducted for Staphylococcus aureus infections involving more than 150 hospitals across more than 14 countries. The multi-centre trial rapidly evaluates different antibiotics and treatment strategies to reduce mortality and improve patient outcomes. In Canada, the Research Institute of the McGill University Health Centre (The Institute) coordinated the trial across six provinces and 32 hospitals.

Staphylococcal infections cause over one million deaths per year. The most serious form of infection is when it enters the bloodstream, with a mortality rate of 15 to 25 per cent. While there are effective antibiotics to treat the bloodstream infections, uncertainty has remained over which treatments lead to the best patient outcomes.

Findings from the SNAP Trial, published simultaneously on June 17 in the New England Journal of Medicine (NEJM) and The Lancet, challenge the long-held assumption that cloxacillin should remain the default treatment and provide important new evidence to guide treatment strategy.

“For decades, we have not known the best way to treat this serious infection which affects hundreds of thousands of patients worldwide. We were taught that cloxacillin was better based on expert opinion and in vitro data. So it is critical that we now have randomized data that teaches us this thinking was not correct,” says Dr. Todd Lee, co-lead investigator on both papers, a scientist in The Institute’s Infectious Diseases and Immunity in Global Health (IDIGH) Program, infectious diseases and internal medicine physician at the McGill University Health Centre and Professor at McGill University. “International trials like SNAP help ensure that the results are reliable, broadly applicable and capable of changing care on a global scale.”

The NEJM study—Comparing cefazolin and cloxacillin

In the study published in the NEJM, researchers compared antibiotics used to treat methicillin-susceptible Staphylococcus aureus (MSSA) infections. They found that cefazolin is at least as effective as cloxacillin, but associated with fewer side effects and a lower risk of kidney injury.

The Royal Melbourne Hospital’s Professor Steven Tong, an infectious diseases physician at the Doherty Institute and global co-lead investigator of the SNAP Trial, says the results provide clear evidence that cefazolin should be considered the first-line option to treat MSSA bloodstream infections.

“In the treatment of MSSA infections, there is an 89 per cent probability that cefazolin is associated with lower mortality,” says Prof. Tong. “Patients treated with cefazolin fare better, with fewer deaths within 90 days (15 per cent compared to 17 per cent for those who received flucloxacillin). Cefazolin was also associated with fewer cases of acute kidney injury, at 14 per cent, compared to 20 per cent with flucloxacillin.”

“The results are sufficiently compelling that I immediately made the switch in my own clinical practice,” he adds.

The Lancet study—Comparing benzylpenicillin and cloxacillin

In the paper published in The Lancet, the study evaluated whether benzylpenicillin could be used to treat penicillin-susceptible Staphylococcus aureus (PSSA) infections where laboratory testing confirmed the susceptibility to penicillin.

Dr. Lee, first author of the NEJM study and senior author of The Lancet study, who led the recruitment of hundreds of patients across Canada, says benzylpenicillin was as effective as cloxacillin and likely safer.

“Patients treated with benzylpenicillin experienced less kidney damage, and mortality was also lower at 14 per cent compared with 22 per cent in the cloxacillin group,” says Dr. Lee.

A shift away from cloxacillin

Researchers say these results mark a turning point in the treatment of MSSA and PSSA bloodstream infections, signalling a shift in clinical practice.

Penicillin was once widely used to treat Staphylococcus aureus, but antibiotic resistance led clinicians to adopt cloxacillin as the standard treatment for MSSA and PSSA infections. In recent years, researchers like Dr. Matthew Cheng at The Institute have described the reemergence of penicillin susceptibility.

The findings support moving away from cloxacillin as the default treatment for MSSA and PSSA infections, given safer and equally effective alternatives are available.

Professor Joshua Davis, an infectious diseases physician at the University of Newcastle and the Hunter Medical Research Institute, and global co-lead investigator of the SNAP Trial, says some strains are once again susceptible to penicillin, renewing interest in carefully reintroducing older antibiotics.

“These findings show clinicians can confidently use penicillin susceptibility results to guide treatment where laboratory testing is available,” says Prof. Davis.

Lyn Whiteway, a sepsis survivor and patient partner on both trials, welcome the findings.

“The SNAP Trial shows what is possible when patients are truly at the centre of research. These findings will save lives and spare people from unnecessary harm,” says Ms. Whiteway.

“We are profoundly grateful to the Canadian patients who participated in SNAP. Clinical trials are only possible because individuals are willing to contribute to research at a vulnerable moment in their lives. Their participation has generated evidence that will improve care for patients with serious Staph aureus infections today and for many years to come,” says Dr. Emily McDonald, a scientist in The Institute’s IDIGH Program and co-principal investigator of the studies.

The next challenge: translating the findings

Researchers say the next challenge will be translating the findings into routine clinical practice.

While cefazolin availability may need to increase in some countries, researchers say implementation will ultimately depend on hospitals, laboratories and guideline groups incorporating the findings into clinical care.

“By bringing together patients, researchers and hospitals from around the world, we can answer important questions more quickly and with greater confidence than any single centre could on its own,” says Dr. Lee. “Trials generate the evidence, but the next step is making sure that evidence changes practice.”

About the SNAP Trial

• The SNAP Trial is a major Australia-led global clinical study investigating the most effective treatments for Staphylococcus aureus bloodstream infections across all age groups.

• This trial is the world’s largest study ever undertaken to improve treatment for Staphylococcus aureus infections.

• So far in the overarching SNAP Trial, participants have been enrolled in Australia, Canada, France, Germany, Israel, Japan, Malaysia, the Netherlands, New Zealand, Singapore, South Africa, Sweden, the United Kingdom and the United States. The trial will continue testing new approaches to improve outcomes for patients facing this serious infection.

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Major international study on respiratory support in critical illness

Edusehat — Mon, 29 Jun 2026 08:40:08 +0700

HN Summary

• A major international study led by Scarborough Health Network found that CPAP, high-flow nasal cannula and bilevel non-invasive ventilation can reduce the need for invasive ventilation in critically ill patients with acute hypoxaemic respiratory failure.

• The analysis of 44 clinical trials involving more than 9,700 patients also found that CPAP and high-flow nasal cannula may improve survival compared with standard oxygen therapy.

• Researchers say the findings provide stronger evidence to guide critical care decisions and support the early use of non-invasive respiratory therapies to improve patient outcomes.

A new international study led by Dr. Christopher Yarnell, an intensive care physician at Scarborough Health Network (SHN), is helping answer an important question in critical care: what is the most effective way to support patients who are struggling to breathe without needing to use a ventilator?

Published in The Lancet Respiratory Medicine, the study is among the most comprehensive analyses to date on non-invasive respiratory support. Drawing on data from 44 clinical trials and more than 9,700 patients worldwide, Dr. Yarnell’s and his co-authors’ research provides valuable clarity for clinicians treating some of the sickest patients in hospital. The study’s first author was Kevin Lee, a medical student at the University of Ottawa. Dr. Peter Reardon and nurse Thecla Kattakkayam from SHN were also co-authors.

Understanding a common and serious condition

The study focuses on acute hypoxaemic respiratory failure (AHRF), a condition in which the lungs cannot provide enough oxygen to the body. This is a common reason patients are admitted to intensive care units (ICUs), accounting for up to 15 per cent of ICU admissions.

Patients with AHRF often need help breathing. In severe cases, this involves invasive ventilation, where a breathing tube is inserted into the airway and connected to a machine to provide oxygen to the lungs when the patient cannot do so adequately on their own. While potentially lifesaving, this approach can come with risks such as infections, physical distress, and longer recovery times.

Whenever possible, clinicians aim to use non-invasive respiratory support, which helps patients breathe using masks or nasal devices. These methods can reduce complications and improve patient comfort, but until now, it has not been entirely clear which non-invasive approach is the most effective for more severe cases.

Dr. Yarnell and his colleagues examined four commonly used non-invasive methods:

• Continuous positive airway pressure (CPAP), which delivers steady air pressure via a tight-fitting face-mask to keep airways open;

• High-flow nasal cannula (HFNC), which provides warm, humid oxygen through the nose at high flow rates;

• Bilevel non-invasive positive pressure ventilation (NIPPV), which alternates between higher and lower pressures via a tight-fitting facemask to assist breathing;

• And standard oxygen therapy, where oxygen is delivered at low flows by a loose-fitting mask or nasal prongs.

The findings showed that CPAP, HFNC, and bilevel NIPPV all reduce the need for invasive ventilation compared with standard oxygen therapy. Another finding was that CPAP and HFNC may improve survival. Results were the same in comparable studies that used both specific criteria and case-by-case judgment for switching to invasive ventilation. This means the results apply to real-world critical care contexts.

“Our results show that these therapies are consistently helpful for patients with major breathing problems, regardless of whether intubation decisions are guided by judgment or specific criteria,” shared Dr. Yarnell. “This is very exciting, because it validates the approach we take at SHN. We use CPAP, HFNC, and bilevel NIPPV to try and avoid invasive ventilation while supporting patients during their illness.”

By strengthening confidence in these approaches, the study helps support safer, more effective treatment decisions for critically ill patients.

“This study reinforces what we see at the bedside every day—that thoughtful use of non-invasive respiratory support can meaningfully change a patient’s care journey,” said Dr. Martin Betts, Chief of Critical Care.

“It can give healthcare professionals greater confidence to act early and avoid invasive ventilation when possible, while also reflecting the kind of practice-led research at SHN that is directly shaping how critical care is delivered.”

Building better care through discovery

Dr. Yarnell is a clinician-scientist in SHN’s Department of Critical Care and a researcher with the SHN Research Institute. He is also affiliated with the University of Toronto’s Interdepartmental Division of Critical Care Medicine. This latest publication highlights SHN’s growing impact in high-quality clinical research with global reach.

“This work demonstrates how our physicians are contributing to international conversations in critical care,” shared Dr. Samir Grover, Executive Vice President of Academics at SHN. “Dr. Yarnell’s work helps ensure that the care we provide is grounded in the best available evidence, ultimately improving outcomes for patients both here in Scarborough and around the world.”

In addition to its key findings, the study points to areas for future research, including the importance of learning more about whether and when to use invasive ventilation in patients with AHRF. Another observational study led by Dr. Yarnell, currently recruiting patients at all 3 SHN hospitals, aims to address that exact problem.

As SHN continues to expand its research programs, studies like this reflect a broader commitment to advancing patient-centred care through rigorous, impactful science.

By helping clinicians better understand how to support breathing in critically ill patients, Dr. Yarnell is contributing to safer, more effective care for some of the most vulnerable people in the health system, and reinforcing SHN’s role as a leader in research and innovation.

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Helping people breathe easier in a changing climate

Edusehat — Mon, 29 Jun 2026 08:40:08 +0700

HN Summary

• In response to the growing challenges from climate change, VCHRI researchers conducted a quality improvement project to develop a Wildfire Smoke and Extreme Heat Action Plan.

• Project lead Dr. Emily Brigham’s goal was to give people the tools and knowledge needed to protect themselves in extreme heat events.

• The action plan was developed collaboratively with patient partners, CREs, KT specialists and PH experts.

• The team also created a provider guide with evidence-based recommendations to support clinicians.

As wildfires and extreme heat become more frequent, a new action plan helps people with chronic respiratory conditions stay safe and better manage their lung health.

With climate change intensifying, British Columbia is experiencing longer wildfire seasons and more frequent heat waves. These weather events pose serious health risks, particularly for people living with respiratory conditions such as asthma and chronic obstructive pulmonary disease (COPD). The 2021 heat dome, which sparked catastrophic wildfires and led to a sharp rise in heat-related hospitalizations and deaths, underscored the urgency of these risks.

To respond to this growing challenge, researchers at the Vancouver Coastal Health Research Institute conducted a quality improvement project developing and testing a written Wildfire Smoke and Extreme Heat Action Plan. This simple, one-page tool is designed to help people prepare for and respond to smoke or heat events with clear, practical steps.

“As health care providers, we see the impacts of climate change in our practice,” says Dr. Emily Brigham, the project’s lead. “Our goal was to give people the knowledge and tools to protect themselves when smoke and extreme heat threaten their lungs.”

The action plan was developed collaboratively, with input from patient partners, certified respiratory educators (CREs), knowledge translation specialists and public health experts. Patient partners shared lived experience to ensure the tools reflect real-world needs, while CREs — respiratory therapists who support people with chronic lung disease — provided clinical expertise. Together, they helped shape the resource that is available online and has been shared with providers nationally and internationally.

Putting preparedness into practice

Action plans are widely used in the care of patients with asthma or COPD to help individuals living with these diseases recognize symptom changes and respond appropriately, including adjusting medications or seeking care. This new action plan builds on that model by addressing environmental risks.

Patient partners and CREs emphasized the importance of addressing extreme heat alongside wildfire smoke in the plan, noting these events often occur together. They also highlighted the need for accessible formats, leading to the development of both a printable plan and a forthcoming digital version.

“It was important for us to engage patient partners and end users early on,” explains Brigham. “Their perspectives helped make this tool more practical and relevant for everyday use.”

Early feedback has been encouraging. Participants who responded to the team’s survey reported a better understanding of climate-related health risks and increased confidence in taking preventive action. CREs involved in focus groups noted the provider guide was particularly helpful in completing the tool and provided insightful feedback on ways to further optimize the tool and its dissemination for greatest effect on preparedness.

Building climate-ready health care systems

The project highlights the importance of embedding climate preparedness into routine care. By consolidating established evidence into a one-stop resource, the action plan is designed to support people in protecting their health. It also acknowledges that not all recommendations can be implemented by everyone, emphasizing that even small actions can make a meaningful difference.

“Readiness looks different for everyone,” notes Brigham. “We want patients to leave their appointments feeling more prepared than when they arrived.”

The team continues to grow through collaboration, and is continuing to refine the tool and is engaging with organizations including the BC Lung Foundation, Canadian Thoracic Society, Asthma Canada and COPD Canada. Efforts are also underway to adapt the plan to a digital platform, and to consider unique needs and preferences of rural and Indigenous communities.

The action plan is part of a broader effort to integrate climate preparedness into health care delivery. Since its initial pilot in B.C., the team has connected with new partners in British Columbia, and across Canada, the U.S. and Australia, contributing to a growing global effort to address climate-related health risks.

“Climate change is a shared challenge we are all facing, and no one can address it in isolation,” says Brigham. “Our team’s goal is to contribute to solutions that make a positive impact for patients.”

This work was made possible through the contributions of past and present Action Plan project team members, including participants, trainees, patient partners and co investigators. The research team also acknowledges the valuable support of partners involved in the Digital and Indigenous Action Plan projects: Drs. Samir Gupta, Stacey Butler, Femke Hoekstra, Sameh Mortazhejri, Pat Camp and Briony Gray; clinical research educators Jacqueline Turvey and Jerome Beaupre; and Carrier Sekani Family Services.

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One chord at a time: Ukulele program builds connection for people living with dementia

Edusehat — Mon, 29 Jun 2026 08:40:08 +0700

HN Summary

• Researchers at the Vancouver Coastal Health Research Institute found that a 12-week community ukulele program helped people living with dementia build confidence, social connections and a greater sense of belonging.

• Unlike traditional music therapy, the Uke Connect program focused on actively learning a new skill, encouraging participants to play the ukulele, build friendships and work toward a shared public performance.

• Researchers say community-based music programs can help reduce social isolation, challenge stigma and support a better quality of life for people living with dementia.

Over 700,000 people currently live with a neurocognitive disorder or dementia in Canada. With 61% of them living in a community setting, there is a growing need for equitable, person-centred engagement and accessible community programs that challenge stigma and foster inclusion.

A new Vancouver Coastal Health Research Institute (VCHRI) study led by VCHRI researcher Dr. Lillian Hung and University of British Columbia (UBC) biomedical engineering student Jason Fu found that learning music together may help build confidence, social connection and improve quality of life of people living with dementia in the community.

The study reviewed the development and delivery of the 12-week Uke Connect, a community music program launched in partnership with UBC and Burnaby Neighbourhood House. The program provided a supportive learning environment that brought together 15 people living with dementia and three care partners for weekly Uke Connect gatherings. Participants learned how to play the ukulele, practicing simple chords, learning songs and building confidence and relationships with others.

“Music connects people. It is universal,” says Hung. “Music is not and should not be a luxury — it needs to be accessible.”

A program shaped by lived experience

The Uke Connect program welcomed participants from diverse cultural backgrounds and varying cognitive abilities, creating an environment where everyone could participate regardless of musical experience.

Unlike many music programs designed for dementia care in long-term care homes — which often focus on listening or sing-along activities — Uke Connect emphasized active learning. Participants picked up the instrument themselves and learned in real time how to play, showing how learning a new skill can stimulate creativity, coordination and cognitive engagement while strengthening social connection.

The study was based on a co-leadership model, where researchers and people with lived experience work together to test ideas and continually refine programs based on participant feedback. Hung and Fu partnered with Mario Gregorio, a long-time patient partner who lives with dementia, to guide program priorities and decisions related to accessibility, participation and inclusion.

Gregorio challenged traditional assumptions about how research programs engage people living with dementia.

“Mario taught us that participants were not only there to join the program. They should be actively engaged partners in the project,” says Hung.

Many participants arrived unsure whether they could learn an instrument, Hung shares.

“People living with dementia are often hesitant to go out on their own,” she says. “This can lead to social isolation, which is a significant barrier to achieving quality of life for this population.”

But, after attending the first Uke Connect class, many participants kept returning.

Music as a pathway to belonging

Through study feedback, participants reported a shift in how they saw themselves: as active members of a community. And this extended outside of the classroom. Participants encouraged one another to attend Uke Connect each week, calling other participants, who had now become friends, with reminders and invitations.

“Having a sense of agency is powerful,” says Hung. “And not just individual agency, relational agency grown from mutual support.”

The group also worked toward a shared goal of performing together in public. At the end of the program, participants performed in a public space at Metrotown Skytrain Station, sharing their music with passersby in a celebration of connection and visibility.

For Hung, the experience highlights the importance of expanding how dementia research defines success.

“In traditional research, we often measure success by clinical outcomes,” she says. “But participants in the program emphasized different outcomes, which can be equally as important, especially for a group where loneliness has a significant impact on quality of life.”

“Connection, joy and belonging are clear markers that made this project so impactful.”

Hung hopes programs like Uke Connect will further inspire communities to rethink how they support people living with dementia.

“These kinds of programs can build age-friendly and dementia-friendly communities and strengthen intergenerational engagement,” she says. “People living with dementia can still live a very good life. They can learn. They’re important members of society. They belong.”

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Canada should stop the expansion of MAID for mental illness – once and for all

Edusehat — Mon, 29 Jun 2026 08:40:07 +0700

What began as a narrowly framed end-of-life exception evolved into a system that places disability at the centre of eligibility debates.

In an open letter to the Prime Minister and federal Ministers of Justice and Health, more than 90 disability and mental health organizations called on Parliament to permanently halt the planned expansion of medical assistance in dying (MAID) for people whose sole underlying condition is a mental illness.

Now, the Special Joint Committee on Medical Assistance in Dying has aligned with that position. The task before MPs and Senators is clear: follow through on the committee’s advice and end the planned expansion permanently.

Over the past decade, Canada’s MAID framework has undergone continual expansion beyond the narrow circumstances originally contemplated by the Supreme Court of Canada in Carter v. Canada. What was presented to Canadians in 2015 as a tightly safeguarded end-of-life exception has steadily broadened through legislative and judicial decisions into a framework where disability and mental illness have become increasingly central to eligibility debates.

In Carter, the Supreme Court struck down the blanket prohibition on assisted dying for competent adults experiencing grievous and irremediable suffering. Parliament’s response through Bill C-14 limited MAID to situations where natural death was “reasonably foreseeable,” reflecting an attempt to balance autonomy with safeguards for vulnerable persons.

That balance shifted following the 2019 Truchon decision, when the reasonably foreseeable death requirement was struck down and the federal government chose not to appeal. Bill C-7 followed in 2021, creating Track 2 MAID for people who are not nearing the end of life. A Senate amendment then added the future expansion of MAID to individuals whose sole underlying condition is a mental illness.

Bill C-7 marked a profound shift in Canada’s MAID framework. What began as a narrowly framed end-of-life exception evolved into a system that increasingly places disability at the centre of eligibility debates.

Critics, including these disability organizations, argue that Track 2 MAID created a separate

legal pathway tied to disability status by allowing eligibility based largely on suffering associated with disability, even where death is not reasonably foreseeable. In practice, the framework creates differential treatment on the basis of disability and singles out disability as a basis for eligibility in ways that continue to raise serious equality-rights concerns under the Charter.

These concerns are intensified by the reality that many disabled Canadians still face major barriers to housing, mental-health care, income security, employment and community supports. Canada should not continue expanding pathways to assisted death while core commitments to disability inclusion remain inadequately implemented.

This concern is particularly significant for many Autistic people and families.

Autism is not classified as a mental illness under the DSM. It is a neurodevelopmental condition. However, many Autistic people experience co-occurring mental-health conditions, often shaped by chronic exclusion, poverty, bullying, isolation and inadequate supports.

Autistic people experience disproportionately high rates of depression and suicidality, while continuing to face barriers to timely mental health care, disability supports, housing and employment. The concern is that broad psychiatric eligibility categories could indirectly place some Autistic people at increased risk within a system that still fails to provide adequate supports for living.

Importantly, Canada’s existing MAID framework already permits access for individuals who may experience mental illness alongside other qualifying medical conditions. The current debate is about something fundamentally different: whether mental illness alone should become sufficient grounds for eligibility.

A country that cannot consistently provide accessible and adequate support cannot conclude that suffering is irremediable.

The government should demonstrate meaningful implementation of the commitments they have already made to disabled Canadians. That includes fully implementing Canada’s Autism Strategy, advancing the Disability Inclusion Action Plan, strengthening community mental-health services and addressing the broader social determinants of health and well-being.

The Special Joint Committee has now done its work. Its recommendation reflects years of testimony from disability organizations, mental health advocates, clinicians, researchers and people with lived experience. MAID for mental illness is not a temporary implementation problem waiting to be solved. It raises fundamental questions about equality, disability rights and how Canada responds to suffering.

The immediate task for Parliamentarians is clear: stop this expansion from becoming law.

The answer to unmet needs, isolation and system failure cannot be assisted death. It must be the supports, care and inclusion that allow people to live with dignity. nH

Jonathan Lai, PhD, is the Executive Director of Autism Alliance of Canada.

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Supporting teens and young adults living with cancer

Edusehat — Mon, 29 Jun 2026 08:40:07 +0700

Adolescents and young adults (AYA) living with and beyond cancer have unique needs and perspectives that clinicians should be aware of when providing health care. A practice article in CMAJ (Canadian Medical Association Journal) provides easy-reference guidance on how to support this demographic of people aged 15 to 39 years.

1. Disrupted milestones — As cancer disrupts milestones in life for adolescents and young adults, clinicians should try to address their unique needs and the effect of disruptions in various stages of development. This should include addressing topics such as fertility, sexual health, and advance care planning.

2. Mental health impacts — Cancer has a mental health impact on all patients. As adolescents and young adults are especially at risk of anxiety and depression, clinicians should regularly screen for mental health challenges and support patients with counselling, referrals, and appropriate medications if relevant.

3. Patient-centred language — Avoid language that might minimize the effect of being diagnosed with cancer and use terms preferred by the patient.

4. Post-treatment support — As most AYAs will live 50 to 60 years after initial treatment, ongoing guideline-aligned follow-up in collaboration with specialists is necessary. Patients may have long-term physical impacts, including potential cancer recurrence.

5. Intersectional identities — Adolescents and young adults have varied identities beyond age, and clinicians should ask about preferred names, pronouns, and other aspects of identity.

“Adolescents and young adults diagnosed with cancer are navigating far more than a diagnosis; they are often managing facing significant life transitions at the same time,” says coauthor Dr. Perri Tutelman, a clinician psychologist and assistant professor, University of Calgary, Calgary, Alberta. “Clinicians can make a meaningful difference by delivering care that is responsive to each patient’s unique needs, priorities, and identities, and tailored to their stage of development. In this article we explore practical ways that clinicians can do this based on our personal and professional lived experiences.”

CMAJ recently published 2026 cancer trends in Canada and research on the risk of later cancers in people treated for cancer as adolescents and young adults.

“Adolescents and young adults living with and beyond cancer” is published June 22, 2026.

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Female patients with traumatic brain injury less likely to be admitted to trauma centres

Edusehat — Mon, 29 Jun 2026 08:40:07 +0700

Female patients with traumatic brain injury (TBI) are 26% less likely to be admitted to a specialized trauma centre than males, according to a study on data from Ontario published in CMAJ (Canadian Medical Association Journal). This difference persisted even after the researchers accounted for factors such as age, severity of injury, other health conditions, and socioeconomic circumstances.

Traumatic brain injuries, often from falls, are the leading cause of trauma-related death and disability globally.

In this study based on ICES data of 55 606 patients admitted to hospital for TBI in Ontario between April 2009 and March 2020, 39% (21 719) were female. From this total, 18 650 patients were admitted to a specialized trauma centre, with 26% (5666) of females and 38% (12 984) of males admitted. Female patients were much older (median age 78 years) than males (median age 67 years) and were more likely to have dementia and hypertension. By contrast, male patients had higher rates of severe head trauma (33%) than females (25%).

Several factors may contribute to these variations in admission rates.

“First, injuries in female patients are more often associated with lower-energy mechanisms, such as ground-level falls, that may attract less attention and may lead to lower prehospital priority,” writes Dr. Natalia Angeloni, a critical care physician at Sunnybrook Health Sciences Centre and PhD student at the University of Toronto, Toronto, Ontario, with coauthors. “Second, unconscious (implicit) sex-related bias may contribute to differential recognition of severity of injury.”

As well, smaller numbers of female patients with TBI in research studies may contribute to a narrow understanding of the way trauma presents in females.

The authors suggest more research is needed to understand sex-based discrepancies in trauma care.

“In Ontario, triage performance is suboptimal, with high rates of both overtriage and undertriage, suggesting variability in decision-making, even when standardized guidelines are in place,” say the authors. “Understanding how this variability interacts with sex and gender is critical. The role, if any, of conscious and unconscious bias in clinical decision-making in care of patients with TBI should be explored, as has been done for other clinical conditions; results should guide targeted interventions to reduce the disparities we have identified.”

“Differences in admission to trauma centres by sex among adults with traumatic brain injury: a population-based cohort study” is published June 15, 2026.

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Hantavirus and Ebola virus disease: 10 things to know

Edusehat — Mon, 29 Jun 2026 08:40:07 +0700

Two deadly infectious diseases, Ebola and hantavirus, have made headlines in recent weeks as they pose serious threats to public health. They both require rigorous infection and prevention control (IPAC) practices and often present with similar early symptoms.

Two succinct articles in CMAJ (Canadian Medical Association Journal) provide information about each disease for clinicians.

Hantavirus:

1. A nationally notifiable disease in Canada — In Canada, 4 to 5 cases are confirmed every year and must be reported. These are usually acquired from rodents in agricultural settings in Manitoba, Saskatchewan, Alberta, and British Columbia. The Andes strain is unique as it can be transmitted from person to person.

2. Causes 2 clinical symptoms — Strains in the Americas, which include the Andes virus featured recently in the news, cause hantavirus cardiopulmonary syndrome. The European and Asian strains cause hemorrhagic fever and kidney dysfunction. Both forms take about 2 to 4 weeks to incubate, and symptoms include fever, headache, muscle aches, and abdominal pain.

3. Serology and polymerase chain reaction (PCR) tests are diagnostic — The National Microbiology Laboratory in Winnipeg performs these tests.

4. Supportive treatment — As there is no specific antiviral treatment or vaccine for hantavirus, treatment is supportive to help alleviate symptoms.

5. IPAC protocols are essential — Patients with suspected Andes strain infection must be isolated with airborne, droplet, and contact precautions, with infectious diseases experts involved and public health notified.

Ebola virus disease:

1. Sporadic outbreaks have occurred in Central and West Africa since 1976 — There are 3 main viruses that can infect humans, and evidence suggests they come from fruit bats. Ebola virus is spread via person-to-person contact through bodily fluids like vomit, sperm, diarrhea, and blood, as well as by touching infected surfaces or objects. The current outbreak in the Democratic Republic of Congo is Bundibugyo ebolavirus, with a fatality rate of 30% to 50%.

2. Fewer than 50% of patients have hemorrhagic symptoms — Symptoms include fever of 38°C or higher, fatigue, muscle pain, and gastrointestinal distress. Incubation is 2 to 21 days, and diagnosis is made with PCR testing.

3. People with potential symptoms and exposure risk should be tested — People who have travelled to countries with Ebola virus disease or who have been in close contact with infected people or bats, primates, or game from the affected areas should be tested.

4. Stringent IPAC must be used for suspected cases — Health Canada has a detailed process for screening, assessment, and IPAC precautions, which must include a fit-tested N95 respirator, face shield, gloves, and fluid-impermeable gear for full protection.

5. Important advances in prevention and management of the disease have been made — Vaccines to prevent Zaire ebolavirus are very effective, and 2 antivirals can reduce mortality from 50% to 35%. However, there are no current vaccines or medications to prevent or treat Bundibugyo ebolavirus, for which supportive care is the main approach.

“Hantavirus” and “Ebola virus disease”were published June 22, 2026.

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To curb overprescribing for seniors, researchers urge annual prescription checkups

Edusehat — Mon, 29 Jun 2026 08:40:07 +0700

To address the growing problem of overprescribing for seniors, a new Canadian guideline is calling for routine medication reviews.

In Canada, roughly two in three adults age 65 or older take five or more medications. While often necessary, complex drug regimens can increase the risk of side effects and affect quality of life.

The study underpinning the guideline – co-led by researchers at McGill University and the University of Toronto and developed by an interdisciplinary panel of experts – aligns with federal legislation calling for a strategy on appropriate medication use.

“We have a significant issue with overprescribing in Canada and globally. Patients taking 10, 15 or even 20 medications are not uncommon” said co-lead author Dr. Emily McDonald, Associate Professor in McGill’s Department of Medicine.

Complex drug combinations are rarely studied together and can interact in unpredictable ways, contributing to falls, confusion, bleeding complications and other effects that are often mistaken for normal aging.

As there has been little guidance for clinicians, medication reviews often happen only after a complication arises, she added.

Drawing on evidence from more than 100 clinical trials, the guideline recommends annual medication reviews for adults 65 or older, particularly those taking five or more medications. The goal is to catch unnecessary or potentially harmful prescriptions and, where appropriate, safely reduce or stop them.

The authors also highlight the need for government support to help make these checkups more widely available through physicians, pharmacists and nurse practitioners.

Patients don’t need to wait for their health care provider to initiate a prescription checkup, said McDonald. Seniors taking multiple medications are encouraged to ask for one, using five key questions to guide the discussion.

“Patients should understand their medications and feel empowered to ask whether non-drug options are possible,” said McDonald, who also directs the Canadian Medication Appropriateness and Deprescribing Network.

The network’s Medication Review Service Finder can help users find publicly funded medication review services in their area.

“Screening for potentially inappropriate prescribing in primary care” by Emily McDonald, Nav Persaud and Aine Workentin et al., was published in Canadian Family Physician. nH

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

Edusehat — Mon, 29 Jun 2026 05:10:07 +0700

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.”

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Backed by $165M, Bionyra Pharma Launches to Advance Inflammatory Disease Biologics

Edusehat — Sat, 27 Jun 2026 06:35:07 +0700

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.

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Intravesical CAR T-Cell Therapy Reduces Bladder Cancer Growth in Preclinical Model

Edusehat — Sat, 27 Jun 2026 03:00:10 +0700

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.”

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Genome Editing at the Turning Point—Bringing CRISPR to Clinical Reality

Edusehat — Sat, 27 Jun 2026 03:00:09 +0700

Laura Sepp-Lorenzino, PhD

Former CSO
Intellia Therapeutics

Shengdar Tsai, PhD

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

Broadcast Date: Wednesday, July 15, 2026

Time: 09:00 PDT, 12:00 EDT, 18:00 CET

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:

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Circio’s circVec and Tcelltech’s nanoSMAR Technologies Combined to Generate Nextgen In vivo CAR-T and TCR-T Cells

Edusehat — Fri, 26 Jun 2026 23:25:10 +0700

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.”

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

Edusehat — Fri, 26 Jun 2026 23:25:09 +0700

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.”

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Heat waves mess with your brain. Scientists are trying to figure out why.

Edusehat — Fri, 26 Jun 2026 19:40:03 +0700

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’

Edusehat — Fri, 26 Jun 2026 08:55:05 +0700

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 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.

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.”

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BIO 2026: Biotech threatened by pressures on patent protection

Edusehat — Fri, 26 Jun 2026 08:55:05 +0700

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.

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AI Framework Surfaces New CAR T Target with Multi‑Cancer Potential

Edusehat — Fri, 26 Jun 2026 08:55:03 +0700

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.”

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BIO 2026: 340B faces growing calls for transparency, patient-focused reform

Edusehat — Fri, 26 Jun 2026 05:15:02 +0700

“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.”

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BIO 2026: FDA Leadership Confront Workforce Losses, China Competition in Drug Development

Edusehat — Fri, 26 Jun 2026 05:10:10 +0700

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.”

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

Edusehat — Fri, 26 Jun 2026 05:10:09 +0700

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.

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Antibiotics Trigger Protein Sharing Among Bacteria, Aiding Persister Cells

Edusehat — Fri, 26 Jun 2026 05:10:08 +0700

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.”

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InduPro Licenses Lonza’s Linker Payload Technologies and Bioconjugation Platforms

Edusehat — Thu, 25 Jun 2026 22:00:11 +0700

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.”

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What Is a Good USMLE Step 1 and USMLE Step 2 Score?

Edusehat — Thu, 25 Jun 2026 18:45:03 +0700

FAQ Section

1. Is Step 1 still scored?

No, Pass/Fail since Jan 2022.

Passing is essential, but more importantly, Step 1 builds the foundation for Step 2.

2. So which score matters now?

Step 2 CK. Scored 1-300; Pass = 218; Average = 245-247

As part of their holistic review, programs now look closely at Step 2 CK when comparing applicants.

3. What’s a “good” Step 2 CK score?

Depends on specialty:

– Primary care: 220–235

– Mid-competitive (IM, OB, EM, gen surg): 235-245

– Competitive (derm, ortho, plastics, ENT, neurosurg): 250+

Score expectations rise with specialty competitiveness. Knowing your target helps guide prep.

4. Can I retake Step 2 CK to improve my score?

No! If you achieve any passing score, you cannot retake the exam.

A passing score locks in forever. Programs see every attempt, so make the first one your best.

5. Do scores matter as much as they used to?

Probably not but it’s hard to say.

Scores are just one piece of the puzzle. Letters, rotations, and interviews carry huge weight too.

Step Scores Have Changed, But Strategy Still Matters

For years, Step 1 was the gatekeeper of residency competitiveness. But now that it’s pass/fail, students are asking:

“What score matters now?”
“How do I stand out without a Step 1 number?”
“What Step 2 score do I need for my specialty?”

Let’s break it down with clarity: what counts as a good score on Step 1 (pass), what Step 2 CK targets you should aim for, and why your first attempt matters.

Step 1: Pass/Fail, But Still Crucial

As of January 2022, Step 1 is reported only as pass/fail. This means that residency programs no longer use it as a numerical filter. But don’t think that this means doing well on Step 1 isn’t important. Step 1 still covers the basic foundational sciences so mastering Step 1 content makes Step 2 CK easier. Even further, a lot of Step 2 questions sneak in some Step 1 material that you thought you were going to leave behind forever.

Bottom line: Step 1 matters for passing, doing well on clerkship rotations, and building your Step 2 CK knowledge.

Step 2 CK: The Score That Counts Now

Step 2 CK is scored numerically from 1-300 with a passing score of 218 (recently raised from 214) and an average score among U.S. MD examinees typically being 245-247.

Now it’s time to address the elephant in the room. Does your Step 2 CK score matter as much as Step 1 scores used to before Step 1 went pass/fail? The answer is it’s hard to say. Step 1 went pass/fail largely in an effort to alleviate the pressure that students felt with this exam being the “end all be all” for their future in medicine. Now that Step 2 is the scored exam, the pressure has somewhat shifted. However, it is important to note that residency programs are performing holistic reviews of applications, with clerkship evaluations and recommendation letters being weighed very heavily. Your Step 2 score is far from the deciding factor or the only thing that matters on your application, but it’s certainly still a component that is seriously considered.

It’s also important to note that the type of program may inform how heavy an emphasis they put on Step 2 scores. If your dream program is a highly academic or research oriented program, they will probably care more about high Step 2 scores. On the other hand, community-based or rural programs may tend to de-emphasize Step 2 scores and place more weight on your experience with their patient populations, recommendation letters, and interview skills.

What’s a “Good” Step 2 Score by Specialty?

What makes a “good” or “competitive” Step 2 score really depends on the specialty you’re applying into. Let’s look at general ranges:

Primary care (family med, pediatrics, psych):
Competitive scores are 220–235.
Moderately competitive (IM, OB/GYN, EM, general surgery):
Aim for 235–245 to stand out.
Highly competitive (derm, plastics, ortho, ENT, neurosurgery):
You’ll want 250+, with many successful applicants scoring 255–260+.

These aren’t hard cutoffs, but they reflect trends in match data and program director surveys. Remember that residency programs are doing more holistic reviews of applications now but Step 2 scores do still matter quite a bit and a great score can help you stand out.

For IMG and DO students applying to competitive specialties or programs that historically have prioritized MD candidates, it is especially important to show that you can score within or even better, above the average score range for that specialty. This will reassure programs that you’re able to excel academically and pass challenging board exams, which are qualities that are highly valued by many programs across the United States.

Why Your First Attempt Is Critical

Unlike the MCAT, if you pass Step 2 CK, you cannot retake it for a better score. Programs see all attempts, and a failure is a red flag. So, your first attempt is your only shot if you pass, so it needs to be your best shot.

So it probably goes without saying that it is extremely important to start preparing early (even during your core rotations). Use UWorld and NBME practice exams. Simulate test conditions and pace yourself so that you can get realistic practice for the real deal.

Tutor Tips for Step Success

Treat Step 1 seriously even though it’s pass/fail. It builds your Step 2 foundation.
Start Step 2 prep during rotations. Learn actively as you go.
Use UWorld questions daily. Don’t wait until the end.
Take NBME practice tests seriously. They’re your best predictors.
Know your specialty’s score expectations. Dermatology ≠ Family Medicine.

Takeaway

Step 1: Pass/fail, but use it to learn deeply for Step 2.
Step 2 CK: Aim for at least 245, but target 250+ if you’re going for competitive specialties.

Unlike the MCAT, there are no do-overs on Step 2 if you pass, so make that first attempt count.

Residency programs still care about your scores, but they also care about your letters, your story, and your fit. Play the long game, prep smart, and you’ll put yourself in a strong position to match into the specialty you love.

If you’re looking for expert advice on preparing for USMLE exams or putting together the optimal residency application, feel free to reach out to EMP to be strategically paired with an expert tutor to help you reach your test score and residency matching goals!

The post What Is a Good USMLE Step 1 and USMLE Step 2 Score? appeared first on Elite Medical Prep.

BIO 2026: American biotech leadership protects patients and national security

Edusehat — Thu, 25 Jun 2026 11:20:03 +0700

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

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?

Edusehat — Thu, 25 Jun 2026 07:45:03 +0700

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

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

Edusehat — Thu, 25 Jun 2026 07:40:08 +0700

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

Edusehat — Thu, 25 Jun 2026 04:10:03 +0700

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.

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Stripe, Anthropic, and OpenAI are backing an effort to stop respiratory infections

Edusehat — Thu, 25 Jun 2026 04:10:03 +0700

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

Edusehat — Thu, 25 Jun 2026 04:05:08 +0700

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.

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First-in-Human Stem Cell Therapy Trial for Huntington’s Disease Begins at UCI Health

Edusehat — Thu, 25 Jun 2026 04:05:07 +0700

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.

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Stripe, Anthropic and OpenAI are backing an effort to stop respiratory infections

Edusehat — Thu, 25 Jun 2026 00:35:03 +0700

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.

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.

“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.

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.

Spotlight on RNA Therapeutics

Edusehat — Thu, 25 Jun 2026 00:30:45 +0700

Drew Weissman, MD, PhD

Professor in Vaccine Research
Penn Medicine

Zachary Ives, PhD

Professor of Computer and Information Science
University of Pennsylvania

Silvi Rouskin, PhD

Asst. Professor of Microbiologyy
Harvard Medical School

Broadcast Date: Wednesday, July 29, 2026

Time: 09:00 PDT, 12:00 EDT, 18:00 CET

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:

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Medra Launches Reasoning Layer for Drug Discovery Robotics

Edusehat — Thu, 25 Jun 2026 00:30:34 +0700

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.

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WHO Selects NIBRT as Training Hub to Help LMICs Build Biopharma Capacity

Edusehat — Thu, 25 Jun 2026 00:30:32 +0700

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.”

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Scaling Stem-Cell Manufacturing for Therapies

Edusehat — Thu, 25 Jun 2026 00:30:31 +0700

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.

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Recoded E. coli Promises More Scalable Weight Loss Drug Production

Edusehat — Thu, 25 Jun 2026 00:30:20 +0700

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.

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Novel Feeder Cell Line Dramatically Expands NK Cell Production

Edusehat — Thu, 25 Jun 2026 00:30:09 +0700

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.”

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Biotech at 50: Can the innovation ecosystem deliver the next generation of breakthroughs?

Edusehat — Wed, 24 Jun 2026 21:00:04 +0700

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?”

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California Still Golden Despite Job Losses: Industry Group

Edusehat — Wed, 24 Jun 2026 20:55:08 +0700

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.”

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Hospitalist-led procedure service: A model for community hospitals

Edusehat — Wed, 24 Jun 2026 20:45:06 +0700

A hospitalist-led procedure service in a community-hospital setting delivered procedures to inpatients faster than specialist-led teams, reducing length of stay without hurting safety—or breaking the bank.

A study in the Journal of Hospital Medicine looked at a hospitalist-led procedure service in two community hospitals in Grand Rapids, Mich. Researchers examined results among patients who received ultrasound-guided paracentesis and thoracentesis between April 2021 and May 2023, comparing procedures performed by an interventional radiology-led team and a hospitalist-led team.

The study found not only that the hospitalist-led team cut delays in the procedures, but they uncovered data about how delays affected length of stay. Here’s an overview of the findings and the effectiveness of the hospitalist-led procedure service in a community hospital setting.

How the service works

Before launching the hospitalist-led procedure service, the two hospitals in the study trained 13 hospitalists over 16 weeks to perform ultrasound-guided paracentesis and thoracentesis. Each hospital is now staffed by a single hospitalist credentialed in procedures who carries roughly one-half of a standard inpatient census while performing procedures. Those hospitalists perform most of the two procedures.

Nearly one-third of the hospitalists on the procedure service reported they had no ultrasound experience. About 40% had ultrasound training or procedural experience during residency, but no experience as attendings.

Length-of-stay reductions

The JHM study found that procedures performed by the hospitalist-led team were associated with a LOS reduction of nearly one day, or 14.8%. The study also found that the delay between when services were ordered and completed dropped from about 34 hours for the specialist-led procedure service to about 13 hours for the hospitalist-led team.

After looking at the data, researchers concluded that the length-of-stay reduction had more to do with the timing of procedures than who was performing the procedures. For every hour between when a procedure was ordered and performed, for example, the data showed a 0.36% increase in LOS.

That meant a 24-hour delay in performing a procedure was associated with an 8.6% increase in LOS. Because the hospitalist-led procedure service was able to reduce turnaround times by about 21 hours, it was able to shave off about one day of LOS in those patients.

The JHM study identified a key factor that likely increased the delay of procedures performed by the interventional radiology-led team: The specialists used a stricter policy for anticoagulation washout for patients on anticoagulant therapies. Researchers said that “clinical holds” created by those strict policies likely created a bottleneck in procedures among the specialist-led team.

The hospitalist-led service, by comparison, used anticoagulation washout guidelines from the Society of Interventional Radiology to determine timing of procedures.

Patient safety and complications

Researchers found that procedures performed by the hospitalist-led team were just as safe as those performed by interventional radiologists. Complication rates were low in both cohorts, the study found, and “consistent with published norms.”

Researchers looked at procedural complications including pneumothorax, major bleeding (defined as the need for red blood cell transfusion within 24 hours of the procedure), transfusion of fresh frozen plasma or platelets, ICU transfer within 24 hours after the procedure, and mortality both at 48 hours and during hospitalization.

Financial impact of a hospitalist-led procedure service

Most previous studies have looked at hospitalist-led procedure services in academic centers staffed by hospitalists with previous experience in procedures. The JHM study, by comparison, looked at a new service with freshly-trained hospitalists working at two community hospitals.

The study concluded that the hospitalist-led unit may be financially feasible for community hospitals because the hospitalists performing procedures retain a half-time patient load. That model could be ideal for community hospitals that don’t have enough demand to justify a full-time proceduralist but have too much demand for a subspecialist team.

Researchers concluded that for community hospitals that can’t afford dedicated proceduralists, “training existing hospitalists to perform procedures and round on hospitalized patients may be the most scalable and cost-effective solution.”

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Gene Editing Pioneer Sangamo Files for Chapter 11 Bankruptcy; Agrees to Sell Assets

Edusehat — Wed, 24 Jun 2026 13:45:08 +0700

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.

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BIO 2026: As capital returns, focus on quality over quantity

Edusehat — Wed, 24 Jun 2026 10:15:04 +0700

“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.”

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BIO 2026: Gary Sinise, Marcus Freeman on leadership, service, and resilience

Edusehat — Wed, 24 Jun 2026 10:15:03 +0700

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

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.

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BIO 2026: Biotech dealmakers navigate uncertainty, pursue opportunities

Edusehat — Wed, 24 Jun 2026 03:05:03 +0700

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.”

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Nextgen Platform Combines VectorBuilder and Maxcyte Technologies to Boost Clinical-Grade Cell Engineering

Edusehat — Wed, 24 Jun 2026 03:00:11 +0700

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.”

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Nvidia Unveils Science Reasoning AI Suite with BioNeMo Agent Toolkit

Edusehat — Wed, 24 Jun 2026 03:00:10 +0700

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.

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Three Subtypes of Severe Pneumonia Might Inform Personalized Therapies

Edusehat — Wed, 24 Jun 2026 03:00:09 +0700

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.”

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Cytomos’ AuraCyt Digital Predictive Cell Analytics Platform Showcased at BIO Conference

Edusehat — Tue, 23 Jun 2026 23:25:09 +0700

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 22-25^th of June, 2026.

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BIO 2026: Where AI is delivering – and where biotech must go next

Edusehat — Tue, 23 Jun 2026 09:10:03 +0700

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.

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BIO 2026: The business of biotech runs on partnerships

Edusehat — Tue, 23 Jun 2026 09:10:03 +0700

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.

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Long‑Range Gene Networks Uncover 641 New Schizophrenia‑Associated Genes

Edusehat — Tue, 23 Jun 2026 09:05:08 +0700

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.

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Pet visits bring “a small moment of home” to long-term care, study finds

Edusehat — Tue, 23 Jun 2026 09:00:10 +0700

Nursing researcher identifies mental health benefits for both residents and staff.

Residents in an Edmonton long-term care home are getting regular visits from therapy dogs this summer, thanks to a pilot project designed by University of Alberta nursing researcher Brittany DeGraves.

The project builds on DeGraves’ recently-published research on the feasibility of using animal-assisted programs to benefit both residents and staff.

“Bringing even a small moment of home for the individuals in care homes is addressing not only mental health but also social isolation,” DeGraves says. “No matter what age or what conditions we have, we all deserve to live a life with joy. And I think that’s something that is often missing in long-term care.”

DeGraves cites research that shows 69 per cent of residents in Canadian long-term care homes have dementia and 87 per cent have some form of cognitive impairment.

For her feasibility study, she interviewed 14 long-term care staff, two long-term care residents and two community members, one an animal therapy volunteer and one a person living with dementia.

They agreed that bringing pets into long-term care settings can improve the social isolation, mental health, and well-being of the older adults living there.

“More rigorous research into this potentially important non-pharmacological intervention is urgently needed,” concludes DeGraves, who is a PhD candidate under the supervision of Dr. Carole Estabrooks, principal investigator for the Translating Research in Elder Care Team.

This summer’s pilot project will take a rigorous clinical trial approach, starting in one care home and continuing in another in the fall. Volunteers and their dogs from the St. John’s Ambulance Therapy Dog Program will visit some residents on a regular basis over six weeks. They will be compared with a group that receives human-only visitation and a usual care group.

“I’ll be looking at the trends in resident outcomes including perceived benefits and measures such as residents’ quality of life from pre- to post-intervention,” she says. “Then hopefully building this into a much larger project with standardized guidance for how these programs should be delivered.”

This is where these older adults are going to live, most of them for the rest of their days. So, let’s make those days enjoyable. Brittany DeGraves

DeGraves, a lifelong “dog person”, first became interested in pet therapy when her grandfather had a stroke. He could no longer recognize his spouse, children or grandchildren. But he lit up when a therapy dog visited his hospital room.

“This was something that reminded him of himself and let us see the man he used to be,” she remembers. “It was a reminder that he was still that person and he deserved to have that little source of happiness, however short it may have been.”

DeGraves saw the impact of pets again when she did a placement in long-term care as a student nurse.

“There were some residents who didn’t really open up to the staff or students, but when the animals came around, their faces just lit up and they finally talked,” she says. “It was so interesting to see that something so small could mean so much.”

She did a term at Maastricht University in the Netherlands, studying Green Care Farms, innovative dementia care facilities where residents participate in daily activities such as cooking, cleaning and animal care as much as they can.

“They had sheep, cows, rabbits and dogs and it was just an eye-opening experience to see not only how happy the residents were, but how independent they felt,” DeGraves says.

DeGraves’ research identifies some barriers to pet therapy programs in long-term care, including some residents’ fear of animals, allergies, and concern about potential injury or infection.

In order to overcome these barriers, she suggests good communication with the residents, their families and staff in advance of visits, making sure the visits are regularly scheduled and carried out on a one-to-one basis, and ensuring that pets and volunteers are well-screened.

DeGraves’ ultimate goal is to see pet therapy as part of standard care for residents of long-term care homes in Canada.

“Let’s recognize that these individuals had a life before this, so what can we bring that reminds them of their life, that brings them into the present, and that gives them consistent moments of joy?” she asks.

“This is where these older adults are going to live, most of them for the rest of their days. So, let’s make those days enjoyable.”

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Southlake Health opens new outdoor patio for mental health patients

Edusehat — Tue, 23 Jun 2026 09:00:10 +0700

Patients will be able to safely access fresh air, natural light and nature as part of their care journey.

Southlake Health has officially opened a new outdoor patio designed specifically for patients receiving inpatient mental health care, creating a safe and therapeutic environment that promotes comfort, connection and access to nature.

“Access to outdoor space plays an important role in the healing experience,” said Mahdi Memarpour, MD, Chief of Psychiatry at Southlake Health. “Southlake’s new patio gives patients the opportunity to step outside in a safe and therapeutic setting that supports their care journey and overall mental wellness.”

Located on the third floor of Southlake’s West Building, the 2,900-square-foot seasonal patio provides patients with safe, direct access to fresh air, natural light, and the outdoors. The thoughtfully designed patio features a variety of seating areas for quiet reflection and social connection, along with planters filled with seasonal flowers and greenery that bring nature into this new healing environment. With a primarily southern exposure and open views through glass safety partitions, the space offers patients a welcoming outdoor setting, while maintaining necessary safety measures.

“More than 1,500 patients who receive inpatient mental health care at Southlake each year will benefit from the new therapeutic patio,” said Katrina Scott, Director, Mental Health Program at Southlake Health. “This important enhancement reflects Southlake’s commitment to continually improving the patient experience and advancing mental health care.”

The patio was made possible through the generosity of donors who supported Southlake Health Foundation’s Better Begins Today Mental Health Campaign. Community support helped fund more than $7.5 million in purpose-built, healing spaces for Southlake’s Mental Health Program, providing privacy, dignity and support when patients need it most. Southlake Health Foundation extends special gratitude to Michael and Sharon Croxon for their long-standing philanthropic leadership and meaningful support of the Better Begins Today Campaign to help empower Southlake to make these transformative upgrades.

“Since the initial launch of the Better Begins Today Campaign, through to this final piece of vital expansion, our generous and thoughtful donor community has helped to transform the patient experience in our Mental Health Program,” said Jennifer Ritter, President and CEO of Southlake Health Foundation. “Every patient who walks through our doors seeking hope and healing in their most vulnerable moments feels the impact of donor generosity in the spaces they have helped create. From the NewRoads Automotive Group Emergent Mental Health Assessment Unit to our expanded Adult Inpatient Mental Health Unit, to the creation of this new outdoor patio space, I hope every person in our communities who generously supported this Campaign feels great pride in the impact they have made for our loved ones, friends, and neighbours.”

Over the next decade, Southlake will transform into a Distributed Health Network (DHN) that includes a redeveloped Davis Drive campus in Newmarket. Updates like the new patio help Southlake continue to deliver leading edge care, close to home, while planning for the DHN is ongoing.

QUICK FACTS

Southlake’s experienced team of psychiatrists, nurses, child and youth counselors and other allied health providers care for more than 2,000 patients each year, supporting their recovery and mental wellness.
In 2023, Southlake opened a new 12-bed adult inpatient mental health unit, increasing mental health capacity to support more patients. With special furnishing, the units are tailored to meet the unique needs of mental health patients.

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AI Discovers Potential Antimicrobial “Prionin” Peptides

Edusehat — Tue, 23 Jun 2026 05:30:10 +0700

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.”

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AbbVie to Acquire Apogee Therapeutics for $10.9B

Edusehat — Tue, 23 Jun 2026 01:55:08 +0700

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

Edusehat — Mon, 22 Jun 2026 22:25:04 +0700

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.

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Breast Milk Fatty Acid Shapes Immune Development in Mice

Edusehat — Mon, 22 Jun 2026 22:20:10 +0700

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

Edusehat — Mon, 22 Jun 2026 22:20:09 +0700

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.”

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NRMP Rank List Timeline Explained: Deadlines, Strategy & Match Process

Edusehat — Mon, 22 Jun 2026 19:05:05 +0700

After months of applications, interview invitations, travel, thank-you emails, and endless conversations with mentors, residency applicants finally reach one of the most important stages of the Match process: creating their rank order list.

For many students, this period feels surprisingly stressful. Interviews are over, but now comes the question that can keep applicants awake at night: How should I rank my programs?

The good news is that the Match process is designed to favor applicants, and understanding the timeline can help reduce a lot of unnecessary anxiety. Here’s a step-by-step guide to the major rank list deadlines and what you should be doing at each stage.

First, Understand the Different Match Systems

One common source of confusion is that not every specialty participates in the same Match.

Most residency specialties use the National Resident Matching Program (NRMP), but ophthalmology and urology have separate matching systems with their own timelines.

As a result, applicants pursuing these specialties will finalize their rankings and learn their results earlier than applicants participating in the Main Residency Match.

For the 2027 cycle, the major specialty-specific milestones are:

Early January 2027 – Urology Rank Lists Due
Late January 2027 – Ophthalmology Rank Lists Due
Early February 2027 – Urology & Ophthalmology Match Day

For everyone participating in the NRMP Main Residency Match, the timeline begins shortly afterward.

January 29, 2027: Standard NRMP Registration Deadline

This is an easy date to overlook, but it’s important.

January 29, 2027 is the standard registration deadline for both the Match and SOAP (Supplemental Offer and Acceptance Program). Applicants who register after this date can still participate, but additional fees apply.

If you haven’t already registered with the NRMP, don’t wait until the last minute. Technical issues, forgotten passwords, and payment problems always seem to appear at the worst possible time. Plan ahead so you’re not scrambling at the last minute!

Consider this your final “administrative housekeeping” deadline before the rank list season officially begins.

Fill out the form below to receive your free Residency Timeline via email!

February 1, 2027: Rank List Entry Opens

On February 1, 2027, applicants can begin entering programs into their NRMP Rank Order List through the R3 system.

This is the point when many students feel pressure to have their list completely finalized. In reality, that’s rarely necessary.

Most applicants spend the first week or two organizing their thoughts, reviewing interview notes, speaking with mentors, and comparing programs. The system allows you to edit your list as often as you’d like before the certification deadline. Even after certifying your list, you can continue making changes as long as you recertify before the deadline. This is important, you must recertify each list that you make changes to or your updates won’t be saved.

Think of February as your decision-making month.

How Should You Build Your Rank List?

This is when applicants feel a lot of stress and pressure. The NRMP matching algorithm is applicant-favorable, meaning your best strategy is remarkably simple: Rank programs in your true order of preference.

Not where you think you’ll match. Not where someone told you you’re “most competitive.” Not where you think the program ranked you.

Simply rank programs based on where you would most like to train. The Match algorithm works best when applicants honestly express their preferences. The NRMP specifically recommends constructing your rank list according to genuine preference rather than attempting to game the system. This is because the algorithm tries to put applicants at their top choice programs first instead of trying to give programs their top choice applicants.

A common mistake is moving a dream program lower because you assume the program won’t rank you highly. In reality, doing so will only hurt your chances of matching there.

If Program A is your favorite, rank it first. If Program B is your second favorite, rank it second. The algorithm will handle the rest.

February: Questions to Ask Yourself

As you organize your list, it can be helpful to revisit the factors that matter most to you.

Consider questions such as:

Which program felt most supportive during interviews?
Where could I realistically see myself living for the next 3 to 7 years?
Which program offers the strongest training in my career interests?
How important are location, family, and lifestyle?
Which residents seemed happiest?
Where would I be most excited about when I open Match Day results?

There is no universally “correct” rank list. A perfect program for one applicant may be a poor fit for another. The goal is not to create the rank list that impresses other people. The goal is to create the rank list that reflects your own priorities.

March 3, 2027: Rank Order List Certification Deadline

This is the big one. Applicants must certify and submit their rank order list by March 3, 2027 at 9:00 PM Eastern Time. Programs must also certify their lists by this deadline.

A few important reminders:

First, simply entering programs is not enough. Your list must be officially certified in the system.

Second, do not wait until the final hour. Every year, applicants experience internet issues, login problems, or last-minute panic. Submitting early provides peace of mind while still allowing you to make changes later if needed. Again, you can change your list as long as it’s prior to this deadline and you make sure that you always recertify it!

Finally, verify that every program on your list is the correct track and program code. Many institutions have multiple tracks, preliminary positions, research pathways, or affiliated sites. Triple-check everything before certifying.

What Happens After You Submit?

Once the certification deadline passes, applicants enter what many students consider the hardest part of the process: waiting.

The NRMP uses its matching algorithm to compare applicant preferences with program preferences and determine final placements. Both applicant and program rank lists are considered in the process.

At this point, there is nothing left to optimize. No additional emails. No secret strategy. No last-minute maneuvering.

The Match outcome is determined entirely by the algorithm using the certified rank lists that were submitted before the deadline.

The Most Important Strategy Advice

If there is one takeaway I can share from this entire process, it’s this: Trust your instincts.

Every year, applicants spend countless hours trying to predict how programs will rank them. In reality, applicants rarely have enough information to make accurate predictions.

There is no way to outsmart the algorithm, focus on identifying where you genuinely want to train. The Match was built to reward honest preferences.

Create a rank list that reflects where you would be happiest, certify it before the deadline, and then give yourself permission to step away from residency planning for a few days. You’ve already done the hard part. Now it’s time to let the Match work as intended.

Quick 2027 Rank List Timeline

Early January 2027
Urology Rank Lists Due

Late January 2027
Ophthalmology Rank Lists Due

January 29, 2027
Standard NRMP Registration Deadline for Match & SOAP

Early February 2027
Urology & Ophthalmology Match Day

February 1, 2027
First Day to Submit and Edit NRMP Rank Order Lists

March 3, 2027
Deadline to Certify and Submit NRMP Rank Order Lists (9:00 PM ET)

The post NRMP Rank List Timeline Explained: Deadlines, Strategy & Match Process appeared first on Elite Medical Prep.

Match Week & SOAP Guide: What Happens During Residency Match Week?

Edusehat — Mon, 22 Jun 2026 19:05:03 +0700

After months of applications, interviews, second-guessing your rank list, and counting down the days until Match results, residency applicants finally arrive at the moment they’ve been waiting for: Match Week.

For many medical students, Match Week is equal parts excitement, anticipation, and anxiety. It’s the culmination of years of hard work, and in just a few short days, you’ll learn where you’ll spend the next several years of your training, which will undoubtedly shape the trajectory of your career.

At the same time, Match Week can feel confusing. What happens on Monday? What if you don’t match? How does SOAP work? When do you find out your actual residency program? This guide walks through the entire week so you’ll know exactly what to expect.

Fill out the form below to receive your free Residency Timeline via email!

What Is Match Week?

Match Week is the final stage of the residency application process. After all the applicants and programs submit their rank lists, the National Resident Matching Program (NRMP) then uses its matching algorithm to pair applicants and programs based on those rankings.

Match Week is the week that those results are shared. Importantly, applicants do not learn where they matched initially. Instead, they first learn whether they matched at all.

The specific program is revealed later in the week on Match Day.

March 15, 2027: Match Status Day

The first major event of Match Week occurs on Monday, March 15, 2027. At approximately 10:00 AM Eastern Time, applicants receive an email informing them whether they matched.

The email is intentionally brief. It does not tell you the program where you matched. Instead, it simply answers one question:

Did you match?

For applicants who receive a “Congratulations, you matched” message, the biggest uncertainty of the application season is over. While there is still curiosity about where they matched, they can spend the rest of the week celebrating and preparing for Friday’s reveal.

For applicants who learn that they did not match, or matched only partially in specialties that include preliminary and advanced positions, the week takes a different path.

That’s where SOAP begins.

March 15, 2027: SOAP Applications Open

SOAP stands for the Supplemental Offer and Acceptance Program. SOAP exists to help eligible unmatched applicants obtain positions in residency programs that still have unfilled spots after the Match algorithm is completed.

Although many students fear SOAP throughout the application cycle, it’s important to remember that every year thousands of residency positions are successfully filled through the process. Programs want to fill all of their available positions with qualified applicants just as much as applicants want to match!

SOAP begins immediately after unmatched applicants receive their Match status notifications. During this phase, applicants gain access to a list of programs with available positions and can begin submitting applications through ERAS.

The process moves much faster than the regular residency application cycle. Decisions that previously took weeks or months may now happen over the course of hours. As a result, preparation and flexibility become extremely important.

If You Learn You Didn’t Match

Finding out that you did not match can feel devastating. Most students spend years imagining Match Week ending one specific way, so receiving an unmatched notification can be emotionally overwhelming.

If this happens, the most important thing to remember is that your next steps matter more than your initial reaction. Many successful physicians have gone through SOAP and ultimately built outstanding careers.

Rather than focusing on what didn’t happen, your energy should shift toward understanding the available opportunities and creating the strongest possible SOAP strategy.

Reach out to your advisors, student affairs office, mentors, and specialty leadership immediately. Most medical schools have dedicated teams ready to help students navigate SOAP.

You do not need to figure everything out alone. Lean on your support network.

March 16–18, 2027: SOAP Interviews and Selection Process

The middle portion of Match Week is where SOAP becomes especially fast-paced. From March 16 through March 18, programs review applications and contact candidates for interviews.

Unlike traditional residency interviews, SOAP interviews are often shorter and more focused. Some may be conducted virtually, while others may be brief phone conversations. Programs are trying to identify candidates who are genuinely interested, flexible, and likely to succeed within their training environment.

Applicants should be prepared to discuss:

Why they are interested in the program
Their career goals
Their application strengths
Any concerns or obstacles in their application
Why they are a good fit for the specialty

One of the most challenging aspects of SOAP is the uncertainty. Communication can happen quickly. Offers may be extended in multiple rounds. Applicants often spend much of the week waiting for updates while simultaneously preparing for potential interviews.

The process can feel exhausting, but every round creates new opportunities.

What Should You Do If You Already Matched?

Ironically, students who successfully matched on Monday often spend the rest of the week wondering how to stay busy. After the stress of interview season and rank list decisions, there is finally very little left to do.

Many students use the week to:

Celebrate with classmates and family
Relax and breathe a sigh of relief
Attend Match Week events
Reflect on how far they’ve come

If you’ve matched, give yourself permission to enjoy the moment. Medical school often trains students to immediately move on to the next goal. Match Week is one of the rare opportunities to pause and appreciate a major accomplishment. Live in the moment and commend yourself on how far you’ve come and what you’ve accomplished.

March 19, 2027: Match Day

Finally, the day arrives. On Friday, March 19, 2027, applicants learn where they matched.

For most students, Match Day is one of the most memorable days of medical school. Schools celebrate in different ways. Some host large ceremonies, while others hold smaller gatherings. Family members, friends, faculty, and classmates often attend to share the experience.

The moment itself is surprisingly simple. You open an envelope, or receive your result electronically, and discover the residency program where you will train.

After years of exams, clinical rotations, applications, interviews, and uncertainty, you finally have your answer. For many students, it feels surreal. One moment you’re a fourth-year medical student. The next, you’re preparing to become a resident physician.

What If Match Day Doesn’t Go as Planned?

It’s easy to think of Match Day as a single success-or-failure event, but reality is often more complicated. Some students match at their first-choice program. Others match lower on their rank list. Some find themselves training in cities they never expected to call home. And some begin residency through SOAP placements rather than the traditional Match process.

The important thing to remember is that where you match is only the beginning of your career story. Years from now, your growth as a physician will be shaped far more by your work ethic, relationships, mentorship, and dedication to patient care than by the exact position of a program on your rank list. Somehow, no matter how you feel on Match Day, you’ll find that you ended up where you were meant to be.

Match Day is a major milestone, but it is not the final destination. It’s the starting line.

Final Thoughts

Match Week is one of the most emotional experiences in medical education. For some students, it brings immediate relief. For others, it presents unexpected challenges and difficult decisions. Regardless of the path, every applicant enters the week carrying years of hard work, sacrifice, and determination.

If you’re approaching Match Week, remember that uncertainty is normal. Trust the effort you’ve already invested. Lean on your support system. Celebrate your accomplishments. And know that whether your path includes a straightforward Match result or a SOAP journey, there are many ways to build a successful and fulfilling medical career. The finish line of medical school is finally in sight.

Quick Match Week Timeline (2027)

March 15, 2027
NRMP Applicant Match Status Announced

March 15, 2027
SOAP Applications Begin

March 16–18, 2027
SOAP Interviews & Selection Process

March 19, 2027
Match Day – Applicants Learn Their Residency Program

The post Match Week & SOAP Guide: What Happens During Residency Match Week? appeared first on Elite Medical Prep.

StockWatch: Positive Phase III Data Sells Investors on Intellia

Edusehat — Mon, 22 Jun 2026 11:35:08 +0700

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

Edusehat — Fri, 19 Jun 2026 22:35:03 +0700

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.

2026-2027 Match Cycle Residency Application Timeline

Edusehat — Fri, 19 Jun 2026 19:15:04 +0700

This article was updated on June 19, 2026, by Rebecca Lapides to reflect the most current information and recommendations.

The final year of medical school marks the start of your residency application journey. To succeed, you need a clear understanding of the application process and its key milestones. For this purpose, we’ve created a detailed timeline to help you navigate the application process! This valuable resource includes:

Key residency application deadlines and milestones
Expert advice to stay on track
A to-do list with all of your application tasks
A clear roadmap to guide you through the application process

Watch our video below for a “Basic Overview of the Residency Application Process” from our Residency Roundtable webinar to familiarize you with the residency program start dates and residency application process. Register for this year’s webinar in July related to ERAS Readiness Check: How to Strengthen Your Application and ask all your questions in real time!

Our downloadable residency application timeline is designed to simplify the application process, providing a clear roadmap to help you stay organized and focused. Enter your email above to get instant access to this valuable resource.

If you’d like personalized guidance and support throughout the application process, consider connecting with a residency advisor from Elite Medical Prep. They can offer tailored insights and expertise to help you succeed. Schedule a complimentary consultation to learn more. Let’s dive straight into the important dates, and tasks you should be completing over each of the next few months!

April to May:

Building a Strong Residency Application

By now, you’ve likely chosen your desired specialty and are preparing your residency application. It’s essential to focus on the core components of your application:

A compelling personal statement
Strong letters of recommendation
Solid exam scores (USMLE, COMLEX, etc.)
Impressive clerkship grades
A comprehensive ERAS application, showcasing your research, volunteer work, and other activities

Writing a Compelling Personal Statement and Securing Strong Letters of Recommendation

As you begin drafting your personal statement, consider who can write an excellent letter of recommendation on your behalf. Typically, you’ll need a standard letter from your medical school, plus two additional letters from mentors in your specialty or those you’ve worked closely with. For expert tips on obtaining great letters of recommendation, check out our past Residency Roundtable session.

Summer Planning: Away Rotations and Sub-Internships

If you’re completing away rotations, submit your applications around early spring. Consider programs that you would like to learn more about and think could be a good fit. Do not wait until the last minute to apply, as some applications have requirements that can be time consuming to complete. Also consider that if you do away rotations by July, you can consider asking an attending at the program for a letter of recommendation, which will significantly strengthen your application.

June to July:

Begin ERAS and Create a List of Potential Residency Programs

Have your printable residency application timeline ready! We’re getting down to business. In June, you will receive an ERAS (electronic residency application system) token from your school.

It’s now time to register an account and begin filling out your application with your basic information. The ERAS application will open on June 4th 2026. It is important to start filling out your application early. The goal is to submit your ERAS application the day that submission opens (given many interviews are provided on a rolling basis). Your ERAS application will include:

Your personal statement
Letters of recommendation,
Grades
Your CV

Since you have to manually enter the components of your CV into the designated spots in ERAS, this process is time-consuming and will require several rounds of edits to ensure no errors are missed.

Additionally, it is important to compile a list of residency programs that you want to apply to. Work with an EMP residency advisor to take into account how many programs to apply to, where to apply, and what backup plans to consider if necessary. It is also helpful at this point to send your personal statement to some individuals in your field for feedback prior to its submission.

Lastly, by the end of this time you will need to formally request letters of recommendation from faculty to allow them enough time (ideally more than 4 weeks) to complete the letter.

August to September:

Finalizing and Submitting Your Residency Application

As you enter the final stretch of the residency application timeline, focus on uploading your letters of recommendation and personal statement, and finalizing the details of your application. Don’t forget to release your USMLE scores to ERAS, allowing programs to view them. Also, be sure to include a professional photo with your application.

Medical Student Performance Evaluation (MSPE) Letter

Your medical school will create an MSPE letter, a standardized letter of recommendation that includes a compilation of your clerkship evaluations and a narrative statement of your educational journey. You’ll have the opportunity to review and make minor edits as needed. Be sure that you work with your school to ensure that this letter highlights all the qualities and experiences that you want it to include.

Submit Your Application and Register with the NRMP

Submit your application on the earliest possible date, for the 2026-2027 residency application cycle that’s in early September (September 2, 2026). Additionally, register with the National Resident Matching Program (NRMP) on September 15th, 2026 to gain access to the program where you’ll eventually submit your match list. Residency programs can begin reviewing applications by late September, so ensure all materials are uploaded to ERAS by this date to maximize your chances of success with interviews.

October to December:

Schedule Your Interviews and Prepare Accordingly

Once you have submitted your ERAS application, programs will begin to review applications on September 23rd, 2026. You will want to ensure you get notifications of your ERAS interview requests. Desirable interview slots are often taken on a first come first serve basis leaving those who review the email later at a disadvantage. For this reason, many students will have their emails forwarded to their phones and have friends or loved ones cover their inboxes during times they may be away for a longer period of time.

During this downtime, you will want to prepare for your residency interviews. When interviews for residency start, you want to be ready. Consider some responses to commonly asked questions, and ensure you have an elevator pitch for yourself when given the opportunity. Consider what questions you will ask programs ahead of time. Often students will take physical notes or record voice memos following these experiences. This will provide additional information with which to make a match list come February.

Additionally, if you do not receive interview invitations from programs you are highly interested in, consider reaching out to the programs who welcome applicant communication to demonstrate interest and increase your odds of receiving an interview.

To help you stay on track, download our Residency Interview Toolkit! It includes a preparation guide, checklist, sample questions, thank-you email templates, and more to streamline your interview prep. Enter your email below to get this free resource sent straight to your inbox.

Fill out the form below to receive your free Residency Interview Toolkit via email!

It’s important to note here that if you are part of the military match, this takes place in mid-December, 2026.

January:

End of Interview Season for Residency and Begin Ranking Programs

This is the end of the residency interview season, and during this time you will be completing your last residency interviews. Given that you will ultimately decide between programs months after you interview there, you will want to make note of interview impressions the day of and distinguish features of a program that may not be visible on their website or in other supporting documentation.

Many students will also send thank you notes to the interviewers who spent time speaking with them. Note that some programs explicitly request that you do not send thank you letters or any post-interview communication. Be sure to abide by these requests, if present. You will also want to check in with an advisor around this time to ensure you have secured enough interviews to have a high probability of success in the match.

By the end of this period, you will want to have a preliminary rank order list of programs. Once you have decided on your top choice program where you have interviewed, you should send a letter of intent. This serves to notify them that you will be ranking them first and why. Again, only send this if it’s a program that welcomes post-interview communication.

Note that the deadline to register with the NRMP is January 29th, 2027. After this date, you will pay a late fee. You must register with the NRMP by March 4th, 2026 to be eligible for the Supplemental Offer and Acceptance Program (SOAP).

February to March:

Submit Your Rank Order List and Then Review Your Residency Application Results!

The rank order list opens by the beginning of February (February 1, 2027). The deadline to certify your rank order list is March 3rd, 2026. You will find out if you have matched on March 15th, 2027. Individuals who did not match can enter the Supplemental Offer and Acceptance Program (SOAP) the same day.

Finally, Match Day is on March 19th, 2026, where you find out your new home for your next stage of training!

For help navigating this year’s residency application timeline including CV and personal statement review and editing, mock interviews, letters of intent for residency programs and more, consider enlisting the help of an Elite Medical Prep residency advisor! Schedule your complimentary consultation today to learn more!

The post 2026-2027 Match Cycle Residency Application Timeline appeared first on Elite Medical Prep.

New report details how U.S. can maintain biotech competitiveness

Edusehat — Fri, 19 Jun 2026 04:35:03 +0700

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

Edusehat — Fri, 19 Jun 2026 04:30:10 +0700

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

Edusehat — Fri, 19 Jun 2026 04:30:09 +0700

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.

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China Sets Framework for Advanced Therapeutic Development

Edusehat — Fri, 19 Jun 2026 00:55:11 +0700

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.

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.

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.”

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IDBS and Alchemi Agree to Accelerate AI-Driven Biopharma Regulatory Filings

Edusehat — Fri, 19 Jun 2026 00:55:09 +0700

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.

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Ricoh Canada referral management technology to be implemented in Ontario

Edusehat — Fri, 19 Jun 2026 00:50:09 +0700

Ricoh Canada Inc. has entered into an agreement to support Ontario Health’s Central Intake implementation across multiple regions of the province.

Central intake models, sometimes referred to as single point of access models, are designed to receive, triage and route referrals centrally to the appropriate clinical setting. In Ontario Health’s, Ricoh’s referral management technology will support this model through centralized intake hubs.

Ricoh’s referral management technology supports digital workflows for referral intake, information capture, data validation and workflow visibility.

“Ricoh is pleased to support Ontario Health’s Central Intake implementation across multiple regions,” said Kartik Rajan, VP, Digital Services, Ricoh Canada Inc.
Modernizing referral management across Ontario

Referral management has often relied on manual processes and multiple hand-offs between referring providers, hospitals, and specialty clinics.
Central Intake is intended to support more consistent receipt, processing, and routing of referrals.
Ricoh’s referral management technology supports intake and routing workflows by capturing and directing referral documentation to the appropriate systems and teams.
Ricoh’s role in Central Intake is part of its broader healthcare services portfolio, which includes workflow automation, enterprise content management, IT services and managed services.

“Central Intake is one example of the use of digital workflow tools in healthcare operations,” added Kartik.

For 90 years since our founding, Ricoh has upheld its mission and vision of empowering individuals to find Fulfillment through Work—and that commitment continues today. By understanding and transforming how people work, we unleash their potential and creativity to realize a sustainable future.

For further information, please visit www.ricoh.com

Ricoh is a global integrator in workplace transformation, operating in approximately 200 countries and regions and headquartered in Tokyo. Supporting customers’ value creation, Ricoh offers workplace services and solutions that empower organizations to work smarter through advanced technologies—including AI— together with long-standing expertise rooted in printing. Ricoh also operates commercial and industrial printing businesses and delivers new solutions leveraging inkjet technology. In the financial year ended March 2026, Ricoh Group had worldwide sales of 2,608 billion yen (approx. 16.4 billion USD).

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Nirrin Technologies and S.T. Japan Partner on Next-Gen Protein Quantitation Tech

Edusehat — Thu, 18 Jun 2026 21:20:10 +0700

Nirrin Technologies appointed S.T. Japan as its exclusive distribution partner for the TALOS^TMprotein 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.”

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Perseverance, Persistence Key to CAR T Success, Say Ross Prize Winners Carl June and Michel Sadelain

Edusehat — Thu, 18 Jun 2026 21:20:09 +0700

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 13^th 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]

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].

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 14^th Ross Prize will be selected in January 2027.

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Largest gift in University of Alberta’s history continues historic partnership transforming women’s and children’s health

Edusehat — Thu, 18 Jun 2026 06:55:09 +0700

A $93.5-million commitment and 30-year collaboration is advancing research shaped by patients and improving care for families.

WCHRI member Samina Ali, professor of pediatrics and emergency medicine at the U of A, is working with a parents’ advisory board to inform pain management protocols for child patients. (Photo: Laughing Dog Photography)

Kayla Sinnamon knew her newborn daughter was in pain — she just didn’t know how to help her.

Sinnamon’s daughter, Natalie, was born with hydrocephalus, a condition that causes cerebrospinal fluid to build up inside the brain. Natalie underwent surgery at eight days old to have a shunt installed, but the procedure cascaded into a complication-ridden seven-week stay at the Stollery Children’s Hospital in Edmonton. Every day, Sinnamon watched her daughter’s tiny body writhe, and eventually seize, with the discomfort of each new needle.

Sinnamon knew the needles were necessary, but she wondered: “How can we do it in a way that’s better for her?”

She didn’t know it at the time, but researchers at the Women and Children’s Health Research Institute (WCHRI) and the hospital’s medical team were already finding answers to her question.

Kayla Sinnamon (second right) and her family have seen the positive difference health research through WCHRI can make since Kayla’s daughter Natalie (centre) underwent complicated surgery as a baby. (Photo: Hello Public)

Based at the University of Alberta, WCHRI is Canada’s only research institute dedicated to investigating women’s, children’s and perinatal health. It operates through a partnership with the Stollery Children’s Hospital Foundation, Alberta Women’s Health Foundation and Alberta Health Services.

Today, that partnership is making history. The Stollery Children’s Hospital Foundation and the Alberta Women’s Health Foundation announced a landmark gift of $93.5 million to WCHRI over the next 10 years — the largest gift in the U of A’s history. This commitment marks the third consecutive decade of the collaboration, adding to the foundations’ combined support of more than $112 million since 2006.

WCHRI executive director Sandra Davidge says the renewed partnership reflects the organizations’ goals of bringing research-driven change to patients.

“It reinforces that children and women’s health is not a niche interest but a top-tier academic and clinical priority,” Davidge says.

Sandra Davidge, executive director of WCHRI (left), with trainee Angie Stokes. Davidge says the landmark commitment from the Stollery Children’s Hospital Foundation and the Alberta Women’s Health Foundation will support work for women and children traditionally overlooked in health research. (Photo: Katrina Grey)

U of A president and vice-chancellor Bill Flanagan notes that the university has the research expertise, infrastructure and talent necessary to translate scientific discovery into improved care for patients and families. Such transformational progress, he says, does not happen in isolation.

“It is built through strong, sustained partnerships,” Flanagan says. “Together, we are advancing a future where health is more than the absence of illness. It is the opportunity for every woman and child to thrive. Supported by this historic gift, we are shaping the wellness of generations to come.”

The partnership has supported the research of clinician-scientists such as Samina Ali, a pediatric emergency physician at the Stollery Children’s Hospital whose research focuses on reducing children’s pain and distress in the hospital. In 2025, under Ali’s leadership, the Stollery became certified by ChildKind International — a distinction shared by only 20 others worldwide that recognizes the hospital’s use of evidence-informed practices to alleviate pain in its young patients. Following Natalie’s hospital stay, Ali recruited Sinnamon to a parents’ advisory board that helps inform the hospital’s new pain management protocols.

Natalie is now a thriving six-year-old, and Sinnamon, now a peer-support volunteer at the hospital, sees the tangible difference this research has made. Children can now inhale nitrous oxide (laughing gas) during painful procedures, for instance, and mothers can choose to breastfeed while their babies have blood drawn.

Seeing the real-life impact of such research breakthroughs motivated the Stollery Children’s Hospital Foundation’s most recent $70-million commitment to WCHRI.

“Through WCHRI, we’re able to support clinicians and researchers who are pushing the boundaries of children’s health and turning bold ideas into real breakthroughs,” says Karen Faulkner, president and CEO of the Stollery Children’s Hospital Foundation.

Sharlene Rutherford, president and CEO of the Royal Alexandra Hospital Foundation and Alberta Women’s Health Foundation, has witnessed the partnership generate powerful results far beyond the province, and is proud to support a $23.5-million commitment over the next 10 years.

“The gap in women’s health is a national concern, but we can be the generation that closes it,” Rutherford says, sharing a striking statistic: Only seven per cent of health research in Canada is specific to women’s conditions and diseases. As a result, women are often left with more questions than answers about their health.

Sarah Chambers knows this feeling too well. After the Edmonton resident was diagnosed with polycystic ovary syndrome (PCOS), her family doctor didn’t have much medical advice to share beyond what would be useful if she were trying to have a baby.

Sarah Chambers says taking part in a clinical trial helped her manage her PCOS and finally feel she had control over her body. She went on to help WCHRI researcher Donna Vine develop two information resources now being used by doctors and patients in more than 30 countries. (Photo: Hello Public)

WCHRI researcher Donna Vine, however, has upended the misconception that PCOS is mainly a reproductive condition. After finding that women with PCOS are three times more likely to develop diabetes and cardiovascular disease, Vine is now operating clinical trials with patients from the Lois Hole Hospital for Women to establish methods of detecting and treating these diseases.

Chambers finally came to understand the steps she can take to manage her PCOS after enrolling in one of Vine’s clinical trials. Later, Vine consulted Chambers as she developed two resources: one guiding doctors in diagnosing and treating the condition, the other explaining the process to PCOS patients. Today, these resources are being used in more than 30 countries. Chambers says the experience helped her finally feel she had control over her body.

As WCHRI’s Davidge says, Sinnamon’s and Chambers’ experiences are just two examples of how this landmark gift will enable the U of A and its partners to continue empowering women and children traditionally overlooked by health research.

“With our strategic funding partners, researchers and health-care teams, we aren’t just closing these gaps — we’re creating limitless potential for the future of children’s and women’s health around the world.”

Learn more about how WCHRI is transforming lives to help women and children thrive

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Personalized Cartilage Graft Developed for Life-Threatening Infant Airway Narrowing

Edusehat — Thu, 18 Jun 2026 03:25:10 +0700

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.”

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World-first burn treatment with “absolutely remarkable” results performed at leading Canadian hospital

Edusehat — Thu, 18 Jun 2026 03:20:11 +0700

Hamilton Health Sciences (HHS) burn experts are the first in the world to use a novel biological treatment to save the face of a young woman who was severely burned in a London, Ontario house fire last December.

The treatment used exosomes – tiny particles released by cells that carry signals from one cell to another to help coordinate rapid healing and tissue repair and reduce inflammation. The particles are collected, typically from lab-grown cells, and injected into the injured areas to accelerate healing – much more so than the current standard of skin grafting, which can leave scarring and a patch-like appearance.

Exosomes have been studied for years as part of burn research, but not yet in humans. Clinical trials involving humans have used exosomes for other types of wound healing, with promising results.

Exosome Burn Procedure – Dr. Marc Jeschke & Kaitlin Jeffrey – HGH

Western University student Kaitlin Jeffrey, 18, of Toronto, received the innovative treatment and healed faster, and with better results, than another young student whose burns from that same fire were serious, but not as severe. The other student wasn’t a candidate for exosome treatment because their injuries did not require skin grafting.

“My vision for Kaitlin was to avoid skin graft surgery to her face and neck at any cost,” says Dr. Marc Jeschke, vice president of research and innovation at HHS, burn surgeon and researcher, and a global authority on burns, who performed Jeffrey’s surgery. Dr. Jeschke is also a professor of surgery at McMaster University, HHS’s key academic partner.

Dr. Jeschke is also the medical director of the regional burn program at HHS’ Hamilton General Hospital – one of Ontario’s two regional burn centres where the most seriously injured patients in the province are treated. Co-located on the Hamilton General Hospital campus is HHS’ Centre for Burn Research, where teams are leading medical advancements and research into burn treatment, including exosome therapy.

After getting the green light from Jeffrey and her parents, Jeschke sent an urgent application to Health Canada to try exosome therapy on compassionate grounds. Receiving no objection, Jeschke and his HHS team became the first in the world to perform this treatment on a burn patient. Jeffrey’s two treatments, which took place several days apart, used one trillion exosomes sourced from the United States.

“You can do the best graft on the planet, but you won’t return the skin to normal. And, for a young person, a skin graft to the face and neck can be absolutely devastating,” he says.

“It’s honestly a miracle,” Jeffrey says of results with respect to her face. “Being injured in the fire has also had deep impact on my mental health, and it’s something I’m continuing to deal with. But having such good results, particularly to my face, is helping me move forward.”

Jeffrey and her family are extremely grateful to Dr. Jeschke and the entire burn centre team. With further research, Jeschke hopes the world-first treatment will become the new standard of care for burn patients in Canada and beyond.

“Like Dr. Jeschke, my family and I would love to see exosome therapy become the standard of care for patients like myself in Canada, so that when horrific things happen, it doesn’t change people’s lives forever,” says Jeffrey.

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BIO 2026: What to know about the AI Summit

Edusehat — Wed, 17 Jun 2026 23:55:03 +0700

“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:

AI Summit Kickoff: Data on AI Adoption in Biotech, 1:00–1:30PM
Beyond the Hype: How AI is Actually Transforming Biopharma in 2026, 1:45–2:45PM
We Are All Winners! AI-Bolstered Digital Precision Health Benefits Patients, Payers, and Providers in Asia Pacific Region, 1:45–2:45PM
How Can Generative Genomics Help Us Design Biology Better, Not Just Faster? 3:00–4:00PM
Data Hunger, Enterprise Risks: Biomedical Data and Cybersecurity in the AI Era, 3:00–4:00PM
Quantum Computing in Drug Discovery, 4:15–5:15PM
Agentic AI in Clinical Trials: Accelerating Recruitment and Streamlining Operations for Large-Population Diseases, 4:15–5:15PM

Additional AI sessions include:

In AI We Trust? The Shift from Calculator to Collaborator, Tuesday June 23, 11:00AM–12:00PM
AI Transformation – Building Organizations that Turn Potential into Breakthroughs, Tuesday June 23, 1:45–2:45PM
Reality Check: Finding the Right AI Partners to Fuel R&D, Tuesday June 23, 3:00–4:00PM
From Code to Clinic: Investing in AI and Life Sciences, Tuesday June 23, 3:00–4:00PM
Breaking Down Silos: Building Multidisciplinary Teams for End-to-End AI Drug Development, Tuesday June 23, 4:15–5:15PM
Accelerating Discovery While Protecting Patient Data: Federated Learning at Scale, Wednesday June 24, 11:00AM–12:00PM
Turning AI Hype into Real-World Breakthroughs and Measurable Value for Pharma, Wednesday June 24, 11:00AM–12:00PM
Regulating the Future: Global Perspectives on AI Governance, Wednesday June 24, 1:45–2:45PM
AI x Nature: Unlocking Biology’s Hidden Blueprint for the Next Generation of Medicines, Wednesday June 24, 1:45–2:45PM
Smart Science, Smarter Deals: How AI Platforms are Transforming Drug Discovery Deals, Wednesday June 24, 3:00–4:00PM
Overcoming R&D Constraints with Generative Artificial Intelligence Supported by High-Throughput Data Generation, Wednesday June 24, 3:00–4:00PM
Putting AI in Its Proper Place – Where and When to Empower Expertise by Embedding AI, Wednesday June 24, 4:15–5:15PM
Breaking Through the Noise: Building Marketable AI Platforms in Biotech, Wednesday June 24, 4:15–5:15PM

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Age-Related Inflammation Linked to R-Loop Nucleic Acids, Opens Therapies

Edusehat — Wed, 17 Jun 2026 23:50:14 +0700

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.

Solvent Recovery Gains Ground in Bioprocessing

Edusehat — Wed, 17 Jun 2026 23:50:12 +0700

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.

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Bacterial Expression Tech Prompts NorthX and enGenes Collaboration

Edusehat — Wed, 17 Jun 2026 23:50:11 +0700

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.

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Continuous Production Platform Offers New Gene Therapy Options

Edusehat — Wed, 17 Jun 2026 23:50:10 +0700

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).

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Analytics Map Purification Optimization Tradeoffs

Edusehat — Wed, 17 Jun 2026 23:50:09 +0700

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.”

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Is nursing job satisfaction falling (again)?

Edusehat — Wed, 17 Jun 2026 20:05:07 +0700

Key Takeaways

A 2026 survey found that job satisfaction among nurses dropped for the first time since the pandemic.
Contributing to a drop in satisfaction, more than half of nurses experienced verbal threats or aggressive language in the past year.
Nearly half of nurses—41%—said “financial necessity” kept them on the job and in the specialty.

After nursing job satisfaction reached a low during the pandemic before rebounding, nurses are once again raising alarm bells in a survey on their attitudes toward the profession.

The data come from the 2026 State of Nursing Survey, which is conducted by Nurse.org. This year’s survey found that after three consecutive years of gains on most metrics, growth in satisfaction levels among nurses has stalled or reversed.

The number of nurses who said they’re likely to leave the bedside has grown, for example, and the number of nurses who say they are likely to leave the profession entirely jumped by nearly 50% in the last year.

Here’s a look at the main findings from the 2026 State of Nursing Survey.

Nursing job satisfaction

In 2025, the survey found that following the pandemic, job satisfaction nearly doubled from 28% in 2022 to 55% in 2025. This year, that number dropped to 47%.

The highest levels of nurse job satisfaction were seen among nurse educators (68%) and NICU nurses (66%). At the low end, 23% of progressive care nurses and 31% of geriatric nurses said they were satisfied with their job. Low satisfaction ratings also came from emergency nurses (35%) and telemetry nurses (34%).

The survey also noted that 2026 marked the first time since 2022 that nursing job satisfaction hadn’t improved on the survey.

Will you leave the bedside in the next year?

Nearly half (43%) of nurses in the survey said they are likely to leave the bedside in the next year. That’s an increase from 39% in last year’s survey.

Will you leave nursing in the next year?

Nearly one-quarter (23%) of nurses in the survey said they’re likely to leave the profession within the next year. That’s up from 15% in last year’s survey, or a jump of nearly 50%.

Why are nurses staying on the job?

Nearly half of nurses—41%—said “financial necessity” kept them on the job and in the specialty. Schedule convenience was identified by 32% of nurses as a motivating factor, while 28% said commitment to patient care kept them going. Only 8% of nurses said they stayed on the job because of support from management/leadership.

Are you happy with your decision to become a nurse?

The percentage of respondents who said they were happy with their decision to become a nurse—68%—remained the same as in last year’s survey.

Would you recommend a career in nursing?

The number of nurses who said they would recommend a nursing career to friends or family remained steady at 47%.

Has staffing on your unit improved?

When it came to staffing issues, 42% of nurses said staffing had worsened in their unit over the past year. By comparison, 8% said it had improved,

Working conditions for nurses

Just under half of nurses in the survey (42%) said their working conditions had worsened, while 8% reported conditions had improved.

Threats in the workplace

More than half of nurses (52%) said they experienced verbal threats or aggressive language in the past year.
More than one in four nurses (27%) reported being physically assaulted.
10% of nurses said they experienced sexual harassment or unwanted sexual contact.
34% of nurses said they do not feel safe from violence in their workplace.

Financial outlook for nurses

One-quarter of nurses (25%) said their paycheck barely covers or doesn’t cover their “essential” monthly expenses.
Just under half (49%) of nurses said their paycheck barely covers essential expenses, but only with careful budgeting.
Only 20% of nurses said they are financially comfortable, and 37% said they couldn’t cover an unexpected expense of $1,000 without going into debt.
37% of nurses said financial pressure forced them to work extra shifts or overtime in the previous 12 months.
About 25% of nurses said they had a secondary source of income, typically from additional nursing jobs.

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Pancreatic Cancer Cell Death Triggered by Caspase‑8 Blockade in Preclinical Models

Edusehat — Wed, 17 Jun 2026 09:30:10 +0700

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.”

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Merck, Protillion Launch AI Drug Discovery Collaboration with Up-to-$510M in Milestone Payments

Edusehat — Wed, 17 Jun 2026 09:30:09 +0700

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.

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Single-Cell RNA Sequencing Reveals Gene Activity Changes in Crohn’s Disease

Edusehat — Wed, 17 Jun 2026 05:55:11 +0700

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.”

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Six Takeaways from the Danaher Bioprocessing Summit

Edusehat — Wed, 17 Jun 2026 05:55:10 +0700

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]

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.

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From discovery to care: Advancing precision health for children and youth

Edusehat — Wed, 17 Jun 2026 02:15:08 +0700

HN Summary

• Holland Bloorview is

advancing precision health for children and youth, moving beyond diagnosis to personalized care based on biology, environment, and lived experience.

• Researchers are leveraging genomics, AI, and large datasets to better match interventions, therapies, and supports to each child’s unique needs.

• National collaborations and clinical trials are helping translate research into real-world solutions, improving outcomes for children with neurodevelopmental differences and disabilities.

Children and youth with neurodevelopmental differences, brain injuries and other childhood-onset disabilities can have different needs and experience very different outcomes, even when they share the same diagnosis.

Increasingly, researchers and clinicians are recognizing that more personalized approaches to care may better reflect the biological, clinical and lived-experience factors that shape health and well-being. This is called precision health.

At the Bloorview Research Institute (BRI), Holland Bloorview Kids Rehabilitation Hospital (Holland Bloorview), researchers are advancing precision health approaches through genomics, data science, rehabilitation research and individualized models of care.

Precision health at BRI

According to BRI’s 2023-2030 strategic priorities, precision health is “a proactive and personalized approach to care where interventions, services and accommodations are informed by each individual’s biology and environment, including their genetics, neurobiology, clinical presentation and sociodemographic context.”

Rather than focusing on diagnosis alone, researchers at BRI are looking beyond labels to better understand the variability within and across individuals to support more personalized interventions and supports.

“It is not always the diagnostic labels that will help us understand what interventions, therapies and services one may mostly benefit from … but their individual characteristics,” says Dr. Evdokia Anagnostou, vice president of research at Holland Bloorview and director of BRI.

Using genomics and AI to support personalized approaches

In 2025, Holland Bloorview joined the Canadian Precision Health Initiative (CPHI), a national genomics effort supported by Genome Canada that aims to sequence 100,000 genomes representing the diversity of Canada’s population.

Through this initiative, Holland Bloorview and collaborators are working to create one of Canada’s largest genomic datasets focused on childhood-onset disabilities including neurodevelopmental differences, mental conditions, brain injury.

Researchers at BRI are also exploring how AI and advanced analytics may support more personalized approaches to care and intervention.

Looking beyond genetics alone

At BRI, precision health research extends beyond genomics alone. Researchers are examining how biology intersects with behaviour, lived experience and socio-environmental factors to better understand which interventions and supports may work best for different children and youth.

For example, researchers at BRI are also exploring how AI and advanced analytics may support more personalized approaches to care and intervention.

Leveraging the Province of Ontario Neurodevelopmental Disorders (POND) Network, researchers are using research and clinical data to develop personalized medical interventions for neurodiverse children and youth. Researchers including Dr. Azadeh Kushki, senior scientist and associate chief of data science and Canada Research Chair (Tier II) in Neurodiversity and Personalized Health, and Dr. Danielle Baribeau, clinician scientist and psychiatrist, are exploring how AI-informed approaches may support more personalized mental health care for neurodivergent children and youth. This includes exploring how data-informed approaches may support medication decision-making.

“Combining our large datasets with advanced analytics, we are working on moving from one-size-fits-all approaches to care that is personalized to the uniqueness of each child.” says Dr. Kushki.

This work is also influencing rehabilitation research and clinical trial design.

Holland Bloorview is a lead site within the Kids and Beyond Neurodevelopmental Trials Network (KINDtrials), a national clinical trials network bringing together researchers, families, advocacy organizations and industry partners to advance therapies and supports for individuals with neurodevelopmental conditions and brain injury.

“KINDtrials was created to speed up the path from discovery to solutions that can make a meaningful difference in people’s lives,” says Dr. Anagnostou.

Researchers at BRI are also developing personalized rehabilitation technologies and interventions. For example, Dr. Elaine Biddiss, senior scientist at BRI, and her team are developing personalized, video game-based motor therapies for children with movement challenges that can be used in homes and clinical settings.

Building more personalized models of care

As health systems increasingly adopt data-driven and personalized approaches to care, researchers say it is important that children and youth with disabilities and developmental differences are represented in precision health research.

Through initiatives spanning genomics, brain imaging, physical and mental health insights, rehabilitation research and clinical trials, Holland Bloorview is helping lead and convene researchers, clinicians, health-system partners, families and communities around more personalized approaches to care. Together, these efforts aim to improve health and well-being outcomes for children and youth with disabilities and developmental differences.

By Priyanka Shah

By Priyanka Shah, Manager, Research Communications, Bloorview Research Institute, Holland Bloorview Kids Rehabilitation Hospital.

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As policy ecosystem threatens biotech, new treatments could drop by over 50% in 20 years

Edusehat — Tue, 16 Jun 2026 22:50:03 +0700

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.

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BioTrinity 2026 Showcases Delivery Technologies and Promising Therapeutic Candidates

Edusehat — Tue, 16 Jun 2026 22:45:09 +0700

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.”

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.

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.

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

Edusehat — Tue, 16 Jun 2026 22:45:08 +0700

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.

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Therapy-Resistant Residual Cancer Cell Dependencies Mapped

Edusehat — Tue, 16 Jun 2026 12:00:10 +0700

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 EGFR^mutcell lines (PC9 and MGH134) and two with KRAS inhibitor sotorasib (KRASi)–treated KRAS^G12Ccell 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.

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Silica Nanoparticles Induce Ferroptosis, Reprogram Immunity in Prostate Cancer Models

Edusehat — Tue, 16 Jun 2026 08:25:10 +0700

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.

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.

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This man with ALS is “the first power user” of a brain implant that lets him speak

Edusehat — Tue, 16 Jun 2026 04:55:02 +0700

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

Edusehat — Tue, 16 Jun 2026 04:50:12 +0700

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.

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mRNA Flu Vaccine Shows Stronger, Longer-Lasting Immune Response

Edusehat — Tue, 16 Jun 2026 04:50:10 +0700

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.”

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When Process Design Fails: 5 Common Planning Gaps That Create Downstream Purification Bottlenecks

Edusehat — Tue, 16 Jun 2026 04:50:09 +0700

Panelists

Victoria Hepworth

Product Manager
Greenfield Global

Cole Cordes

Head of Manufacturing,
Supply Chain and MSAT
Bionova Scientific

Broadcast Date: Thursday, July 16, 2026

Time: 08:00 PDT, 11:00 EDT, 17:00 CET

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:

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

Edusehat — Tue, 16 Jun 2026 01:15:08 +0700

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.”

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Board-Certified Physician Assistant Kieran Lombard, PA-C, Joins Optima’s Scarborough, ME Practice

Edusehat — Tue, 16 Jun 2026 01:15:06 +0700

Lombard is Eager to Provide Expert Medical Dermatology Services to Patients of All Ages

Optima Dermatology is excited to welcome board-certified Physician Assistant Kieran Lombard, PA-C, to our Scarborough, ME practice. Kieran has been providing dermatology services to patients in New England since 2020 and treats a variety of skin conditions, including acne, eczema, psoriasis, and skin cancer.

Kieran received his Bachelor of Health Sciences from Western New England University, graduating cum laude, and later received his Master of Physician Assistant Studies from Massachusetts College of Pharmacy & Health Sciences.

After numerous years providing dermatology services to patients in New England, Kieran is excited to bring his expertise and passion to Optima’s Scarborough practice in Maine.

Delivering the Care Patients Expect and Deserve

Kieran is committed to helping every patient with an individualized treatment plan. His personal experiences with acne allow him to understand what patients need and expect, and meet them with a personalized and compassionate approach that mirrors Optima’s mission of treating patients the way we would treat our own family and friends.

We recently had the opportunity to learn more about Kieran, his approach, and the path that led him to become a board-certified physician assistant.

What made you decide to pursue a career in dermatology? What did the path to get there look like for you?

My path to dermatology started as a patient. I struggled with severe acne growing up and spent a lot of time seeing a dermatologist. One of the physician assistants I saw made a big impression on me and offered to let me shadow him in clinic, which was my first real exposure to the field. I found that I really enjoyed the mix of medicine and patient care involved in dermatology. I had already decided to pursue a career as a physician assistant, but this helped guide the direction of my journey, and I went on to graduate school to earn my PA master’s degree. While in school, I completed a rotation in dermatology, solidifying the love I had for the specialty; the medicine, the procedures, and especially the relationships you build with patients over time.

What is your favorite aspect of being a board-certified physician assistant?

My favorite aspect of being a board-certified physician assistant is the combination of a broad medical foundation and the autonomy I have in dermatology. PA training gave me experience across all major areas of medicine, which allows me to approach patient care with a well-rounded perspective. In dermatology, I can spend time with patients and practice in a way that aligns with my values, while still having the support of a collaborative team and supervising physicians. I truly enjoy that balance of independence and teamwork.

What is your approach to treating your patients?

My approach to treating patients is centered on education and shared decision-making. I aim to provide patients with clear information about their condition and the available treatment options so they can make informed choices about their care. By discussing the benefits and considerations of each option, patients can decide what matters most to them and what best fits their goals. My role is to guide them through that process and support the decision that works best for them.

What is your number one tip when it comes to taking care of your skin?

I think my number one tip is ‘less is more’. It’s easy to get lost in the sea of products claiming to make your skin better, but unless there is a true underlying disorder, the best thing you can do for your skin is wear a good sunscreen, use bland soaps, moisturize, and get your yearly skin check.

What is one thing you want your patients to know about you?

The one thing I want my patients to know about me is that I understand what it’s like to be on the other side of the exam room. Having struggled with severe acne myself, I know how frustrating and personal skin issues can feel. That experience helps me approach patients with empathy and understanding. I strive to maintain professionalism while creating a comfortable, approachable environment where patients feel heard, supported, and confident in the care they’re receiving.

What do you like to do when you are not in the office treating patients?

Outside the office, I try to stay active and enjoy a variety of outdoor pursuits. I play ice hockey and mountain bike whenever I can, and I love spending time outdoors or doing any kind of athletics. I also help manage a small family farm and pursue photography as a side project, which lets me combine creativity with being outside.

What to Expect From Dermatological Care Delivered by Kieran and His Team

Patients visiting Optima Dermatology with Kieran and his team will receive the highest-quality care, backed by decades of advanced training and experience.

Kieran is currently accepting new patients at our Scarborough, ME location. Request an appointment online or call us at (855) 277 – 9689.

The post Board-Certified Physician Assistant Kieran Lombard, PA-C, Joins Optima’s Scarborough, ME Practice appeared first on Optima Dermatology.

Patient groups praise Virginia PDAB veto

Edusehat — Mon, 15 Jun 2026 21:40:03 +0700

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

Edusehat — Mon, 15 Jun 2026 07:05:09 +0700

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 (AR^ON), 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.

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Collagen Resides Inside Cells in Liquid Condensate-Like Form

Edusehat — Sun, 14 Jun 2026 23:55:06 +0700

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 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.

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SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines

Edusehat — Sat, 13 Jun 2026 04:45:09 +0700

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.

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Hantavirus One-Shot mRNA Vaccine Fully Protects in Syrian Hamster Model

Edusehat — Sat, 13 Jun 2026 04:45:07 +0700

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.

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Why “reprogramming” is the buzziest approach to reversing aging right now

Edusehat — Fri, 12 Jun 2026 21:40:03 +0700

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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

Edusehat — Fri, 12 Jun 2026 21:40:02 +0700

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

Edusehat — Fri, 12 Jun 2026 10:45:11 +0700

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.”

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.

Top Mistakes IMGs Make During Residency Applications: An Experience-Based Guide

Edusehat — Fri, 12 Jun 2026 03:55:05 +0700

Applying to residency as an international medical graduate (IMG) can feel like navigating foreign territory. Between understanding the nuances of ERAS, optimizing your application, securing interviews, and preparing for the Match, there are countless moving pieces, and unfortunately, plenty of opportunities to go wrong.

Over the years, I’ve seen incredibly capable IMG applicants fall short not because they lacked intelligence or work ethic, but because they made avoidable strategic mistakes. This guide is meant to walk you through the most common pitfalls in a practical, experience-based way, so you can approach the process with clarity and confidence.

Understanding the Process

One of the most common mistakes IMGs make is underestimating how early the residency application process actually begins. Many applicants think the journey starts when ERAS opens, but by then, most of your application is already “baked.” Your USMLE scores, clinical experiences, letters of recommendation, and research output are ideally finalized well before submission season.

Waiting until the year of application to start building your portfolio can leave you scrambling to fill gaps that programs will notice immediately. Strong IMG applicants often begin preparing at least one to two years in advance, strategically planning US clinical experiences to build relationships at programs that will later translate into meaningful letters.

USMLE Exams

Another major pitfall is treating USMLE exams as just a hurdle rather than a cornerstone of the application. For IMGs, scores often carry even more weight because they serve as a standardized way for programs to evaluate candidates across different educational systems. A common mistake is taking exams before being fully prepared, thinking that “passing is enough.” In reality, many specialties require strong scores to even clear screening filters. Retaking exams or explaining low scores can be difficult and sometimes limiting. The better approach is to delay taking an exam until you are consistently performing at your goal range on practice tests.

Tell Your Story

Programs are not just evaluating your metrics, they’re trying to understand who you are, why you chose your specialty, and what your journey has looked like. Some IMG applicants submit applications that feel fragmented, with experiences that don’t clearly connect to their career goals or their “why medicine” story. For example, someone applying to OB/GYN might have scattered experiences without demonstrating a clear commitment to the field. A strong application tells a story. Your personal statement, experiences, and letters should all reinforce a consistent theme that helps programs understand your path and what drives you. Most importantly, convey to the programs what your story is and why you want to train in the US.

Clinical Exposure

Another frequent issue is not prioritizing US clinical experience appropriately. Many IMGs have excellent clinical backgrounds in their home countries, but residency programs in the United States place a high value on familiarity with the US healthcare system. Observerships alone are often not enough, especially in competitive specialties. Applicants sometimes rely heavily on passive experiences rather than seeking out hands-on roles when possible. While opportunities vary depending on visa status and institutional policies, aiming for experiences where you can actively participate, demonstrate your clinical skillset, and build relationships with physicians can make a significant difference.

Letters of Recommendation

Letters of recommendation are another area where mistakes are common. Some applicants focus on obtaining letters from well-known physicians without considering how well those physicians actually know them. A generic letter from a big name is far less impactful than a detailed, enthusiastic letter from someone who has worked closely with you and knows you well. Another mistake is failing to ensure that letters are specialty-specific. If you’re applying to pediatrics, your letters should ideally come from pediatricians who can speak to your abilities in that field. Strong letters often include specific examples (often stories of your interactions with patients) of your clinical reasoning, teamwork, and professionalism, which can only come from meaningful interactions from clinicians that observed them.

Application Submission

Timing also plays a crucial role, and many IMGs underestimate how competitive the early application window is. Submitting your ERAS application even a few days late can significantly reduce your chances of receiving interviews. Programs often review applications on a rolling basis, and interview spots fill quickly. Some applicants delay submission because they are waiting for one more letter or trying to perfect their personal statement, but in many cases, it’s better to submit a complete and polished application on time rather than a “perfect” one that arrives late.

Another common mistake is applying too narrowly or unrealistically. It’s understandable to have dream programs or preferred locations, but applying to a limited number of highly competitive programs can be risky, especially for IMGs. On the other hand, some applicants take the opposite approach and apply extremely broadly without strategy, which can be financially and emotionally draining. The key is to build a balanced list that includes a mix of reach, target, and safety programs, while also considering factors like visa sponsorship, IMG-friendliness, and overall fit.

Making Your Application Stand Out Beyond the Basics

Another mistake is underutilizing the personal statement. Some applicants treat it as a formality rather than an opportunity to stand out. A generic or overly formal personal statement can make it difficult for programs to connect with you. On the other hand, a well-written statement that tells a story, reflects your personality, experiences, and motivations can leave a lasting impression. It’s important to strike a balance between professionalism and authenticity, allowing your voice to come through while maintaining a clear focus on your specialty.

Applicants also sometimes neglect the importance of their ERAS experiences section. Listing activities without context or reflection can make them feel less meaningful. Programs want to understand not just what you did, but what you learned and how those experiences shaped you. Providing concise but thoughtful descriptions can help bring your application to life.

One subtle but important mistake is failing to tailor your application to your chosen specialty. Some IMGs apply to multiple specialties without adjusting their personal statement, experiences, or letters accordingly. This can create the impression that you are not fully committed to a particular field. If you do choose to apply broadly, it’s important to ensure that each application feels intentional and aligned with the specialty.

Acing the Interview

Interview preparation is another area where applicants often fall short. Some candidates assume that securing an interview means they are already “good enough,” and they don’t prepare adequately. In reality, interviews are where programs assess your communication skills, professionalism, and overall fit. Common mistakes include giving overly rehearsed answers, failing to articulate your personal story, or not being able to discuss your experiences in depth while still remaining concise. Strong candidates prepare by practicing answers to common questions without memorizing scripts, reflecting on their journey, and developing thoughtful questions for programs.

Communication etiquette is also more important than many applicants realize. Small missteps, such as delayed responses to interview invitations or unprofessional email tone, can create a negative impression. Similarly, failing to send thank-you notes or follow-up communication when appropriate can be a missed opportunity to reinforce your interest. While these details may seem minor, they contribute to the overall impression you leave on programs.

Crafting Your Rank List

Another area where applicants struggle is ranking programs. Some IMGs base their rank list heavily on perceived prestige rather than considering factors like training environment, location, support systems, and long-term goals. While reputation can be important, your day-to-day experience during residency will have a significant impact on your well-being and success. Taking the time to reflect on what matters most to you can lead to a more thoughtful and satisfying rank list.

Ironing Out the Details

A particularly challenging area for IMGs is navigating visa considerations, and mistakes here can be costly. Some applicants apply to programs without confirming whether they sponsor their required visa type, leading to wasted applications. Others fail to clearly communicate their visa status in their application, which can create confusion or lead to automatic filtering. Being transparent and strategic about visa-related factors is essential.

Another overlooked mistake is not seeking mentorship or guidance during the process. Many IMGs try to navigate residency applications independently, which can lead to missed opportunities or preventable errors. Connecting with mentors, whether through alumni networks, clinical experiences, or online communities, can provide valuable insights into program selection, application strategy, and interview preparation. Learning from others who have successfully matched can help you avoid common pitfalls.

The Big Picture

Resilience is another factor that often gets underestimated. The application process can be long and emotionally taxing, especially for IMGs who may face additional barriers. Some applicants become discouraged after receiving rejections or a limited number of interviews and lose momentum. Others may not have a backup plan in place, which can create significant stress if things don’t go as expected. Approaching the process with flexibility and a contingency plan can help you stay grounded.

One of the most important mistakes to avoid is losing sight of the bigger picture. The residency application process can feel all-consuming, but it’s just one step in your medical career. It’s easy to become fixated on specific outcomes or compare yourself to others, but each journey is unique. Staying focused on your goals, maintaining perspective, and continuing to grow throughout the process can make a meaningful difference.

At the end of the day, being an IMG is not a disadvantage, it’s a unique strength when approached thoughtfully. Your diverse experiences, adaptability, and determination are qualities that residency programs value. By avoiding these common mistakes and approaching the process strategically, you can position yourself for success.

The residency application journey is challenging, but it is also an opportunity to reflect on your path, refine your goals, and take the next step in your career. With preparation, insight, and resilience, you can navigate it effectively and find a program that aligns with your aspirations.

The post Top Mistakes IMGs Make During Residency Applications: An Experience-Based Guide appeared first on Elite Medical Prep.

USMLE Test Accommodations Process Changes in 2026: What Medical Students and IMGs Need to Know

Edusehat — Fri, 12 Jun 2026 03:55:04 +0700

The United States Medical Licensing Examination (USMLE) has announced an important update to its test accommodations request process that will affect all Step exam candidates, including international medical graduates (IMGs), beginning June 1, 2026.

If you plan to request testing accommodations for USMLE Step 1, Step 2 CK, or Step 3, understanding these changes can help you avoid delays and ensure your application is submitted correctly.

What Is Changing?

Starting June 1, 2026, all USMLE test accommodation requests will be submitted and managed through the MyUSMLE Portal. This represents a significant shift from the previous process, which relied on PDF forms and email-based submissions.

According to the USMLE program, the new system is designed to improve transparency by allowing examinees to:

Track accommodation requests in real time
View submission history
Monitor request status updates
Access previous accommodations records directly through the portal

The goal is to streamline a process that handles thousands of accommodation requests each year while providing students with greater visibility throughout the review process.

Important Deadline: May 15, 2026

As part of the transition, the USMLE program temporarily paused new accommodation submissions. The accommodation request system was unavailable from May 15 through May 31, 2026, while data and records were migrated into the new platform.

Students who submitted accommodation requests before May 15, 2026, at 5:00 p.m. ET will still have their requests processed. Those requests will automatically transfer into the MyUSMLE Portal once the migration is complete. Beginning June 1, 2026, all new requests must be submitted through the MyUSMLE Portal.

What Happens to Existing Accommodation Requests?

The transition includes historical records dating back to 2015.

If you previously submitted a USMLE accommodations request, your information will automatically be transferred to the new system. Once you log into the MyUSMLE Portal, you will be able to access:

Previous accommodation requests
Current requests in progress
Historical accommodation decisions
Future accommodation submissions

Students with active requests should receive instructions directly from the USMLE program regarding portal access.

Does This Affect International Medical Graduates (IMGs)?

Yes. The new accommodation process applies to all USMLE candidates, including students and graduates of international medical schools.

IMGs who need testing accommodations will be required to create a MyUSMLE account if they do not already have one. Once registered, they will be able to manage accommodation requests entirely through the portal. For IMGs navigating the broader USMLE process, it is also important to stay current on exam requirements, timelines, and application deadlines.

Are the Accommodation Requirements Changing?

No. The USMLE has confirmed that only the submission process is changing. The eligibility guidelines, documentation requirements, and review standards remain the same. Candidates should continue to provide all required supporting documentation, including the Certification of Prior Test Accommodations (CPTA) form when applicable.

The USMLE program still recommends submitting accommodation requests as early as possible because decisions may take approximately 60 business days to process.

Which Exams Are Affected?

The new process applies to all USMLE Step exams, including:

After June 1, 2026, PDF accommodation request forms will no longer be accepted for any Step examination. Students preparing for upcoming exams should build accommodation planning into their study timeline.

Why Is USMLE Making This Change?

According to the USMLE program, the transition aims to improve both transparency and efficiency. Previously, examinees had limited visibility into the status of their accommodation requests after submission. The new portal-based system allows candidates to confirm receipt of documents, monitor progress, and access their accommodation history in one centralized location.

The change is also expected to help the USMLE program process requests more efficiently as accommodation applications continue to increase each year.

Key Takeaways

If you plan to request accommodations for a future USMLE exam, here are the most important points to remember:

All accommodation requests now use the MyUSMLE Portal.
PDF request forms are no longer accepted after May 15, 2026.
The change applies to Step 1, Step 2 CK, and Step 3.
The change applies to both U.S. medical students and IMGs.
Documentation requirements and eligibility guidelines remain unchanged.
Candidates should continue submitting requests as early as possible and allow approximately 60 business days for review.

As the USMLE continues to modernize its systems, staying informed about administrative updates is just as important as maintaining an effective study plan. Whether you’re preparing for Step 1, Step 2 CK, or Step 3, having the right guidance can make the process more manageable. Explore Elite Medical Prep’s personalized USMLE 1:1 tutoring services to get expert support tailored to your goals and exam timeline.

The post USMLE Test Accommodations Process Changes in 2026: What Medical Students and IMGs Need to Know appeared first on Elite Medical Prep.

New mRNA Delivery Platform Restores Muscle Function in DMD Models

Edusehat — Fri, 12 Jun 2026 03:35:11 +0700

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

Edusehat — Fri, 12 Jun 2026 03:35:10 +0700

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

Edusehat — Fri, 12 Jun 2026 00:00:10 +0700

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.

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]

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]

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

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
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
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
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
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

Edusehat — Thu, 11 Jun 2026 05:35:10 +0700

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

Edusehat — Thu, 11 Jun 2026 05:35:09 +0700

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.

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GSK to Acquire Nuvalent for $10.6B, Boosting Cancer Pipeline with Precision NSCLC Treatments

Edusehat — Thu, 11 Jun 2026 05:35:08 +0700

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.

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BIO 2026: Partnering meetings fill up for industry’s biggest gathering

Edusehat — Thu, 11 Jun 2026 02:00:04 +0700

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.

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Innovative Tech Testing in Response to GMP Revisions

Edusehat — Thu, 11 Jun 2026 01:55:20 +0700

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.

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Updated Amplification Tool Rapidly Detects Mycoplasma

Edusehat — Thu, 11 Jun 2026 01:55:19 +0700

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.

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Gorilla Adenovirus Brings Natural Edge to Cancer Therapy

Edusehat — Thu, 11 Jun 2026 01:55:18 +0700

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.

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In Silico Devices May Improve Drug Manufacturability

Edusehat — Thu, 11 Jun 2026 01:55:17 +0700

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.”

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Symeres Expands Spray Drying Capabilities at Its U.S. New Jersey Site

Edusehat — Thu, 11 Jun 2026 01:55:15 +0700

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]

“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.

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The “steroid olympics” were a circus—and a window into our culture

Edusehat — Wed, 10 Jun 2026 22:15:03 +0700

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.”

SAEED RAHBARAN

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.”

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.

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.

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.

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.

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.

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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.

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.

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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.

Icelandic strongman Thor Björnsson actually had to reduce the number of substances he was taking to meet Enhanced’s FDA requirements.

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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.)

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.

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.

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.

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.”

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?

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.

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

Edusehat — Wed, 10 Jun 2026 22:10:12 +0700

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.

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10x Genomics Acquires Proteintech Genomics, Expanding Proteomics Capablities

Edusehat — Wed, 10 Jun 2026 22:10:11 +0700

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 cm²whole 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 44^th 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.”

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mRNA Tails Play Key Role in Folding Regulatory Proteins

Edusehat — Wed, 10 Jun 2026 22:10:10 +0700

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.

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Matching life saving transplants inside UHN’s Transplant Immunology Lab

Edusehat — Wed, 10 Jun 2026 22:05:09 +0700

HN Summary

• UHN’s Transplant Immunology Lab plays a critical role in matching donor organs and stem cells, ensuring safe and successful transplants.

• Using advanced HLA testing and antibody screening, the team helps prevent organ rejection and improve long-term transplant outcomes.

• Their work extends beyond surgery, providing ongoing monitoring and support for transplant patients for life.

In a lab most people will never see, a team of specialists is quietly matching donor organs and stem cells to patients in need of transplants.

The work is fast — often happening overnight — meticulous and often out of sight to the patients whose lives depend on it.

As part of UHN’s Laboratory Medicine Program, the Transplant Immunology Lab — formally known as the Histocompatibility and Immunogenetics Laboratory (HLA Lab) — combines science, precision and care to ensure every organ and stem cell transplant at UHN is safe before a surgery moves forward.

Without the lab’s immunological testing and Human Leukocyte Antigens matching, a transplanted organ or stem cell risks rejection by the recipient’s immune system, causing severe and even life-threatening complications.

“We find the best donor for our patient population so that they’ll have the longest, most successful transplant possible,” says Dr. Jeffrey Kiernan, Director of the Transplant Immunology Lab.

“Each year, our team supports over 900 solid organ transplants and over 200 stem cell transplants, from kidneys and lungs to cutting-edge procedures like stem cell transplants.”

The work underpins the life-saving procedures at UHN’s Ajmera Transplant Centre, Canada’s largest and most comprehensive transplant program recognized for its world-class care.

A look inside the lab where matches are made

Together, the Lab’s team of directors, lab technologists, medical lab assistants and administrative staff use highly specialized laboratory processes and tools to determine whether a donor organ can be safely accepted by a patient.

At the core of the lab’s work is the analysis of Human Leukocyte Antigens — tiny protein “ID tags” on the surface of cells that help your immune system know the difference between what belongs in your body and what does not.

The lab process begins by testing DNA samples to determine the HLA tags of both the donor and patient.

“In transplant medicine, these immune markers are everything. The better the HLA match, the higher the chance of a successful transplant,” says Dr. Kiernan.

The HLA matchmaking process for solid organ transplants works a slightly different way. Through a combination of molecular testing and HLA typing, antibody screening and cross-matching, the team not only determines whether a donor and recipient are compatible, but also whether a recipient has antibodies that would attack a specific donor organ.

“With solid organ transplants, we’re not looking for a perfect match in the sense that they’re identical,” says Michael Naglich, Supervisor, and former Senior Laboratory Technologist in the lab.

Iris Grilo, a medical laboratory technologist at UHN’s Transplant Immunology Lab performs Human Leukocyte Antigens testing, a critical step in determining whether a donor and patient are safely matched. (UHN)

“We’re looking for recipients that do not have opposing antibodies. We’re also checking HLA markers to look for a donor that doesn’t trigger a harmful immune response when matched with the patient’s HLA markers. That’s what makes a transplant safe.”

Once the testing is complete, the results are shared with Trillium Gift of Life Network — the provincial agency that coordinates organ and tissue donation and matching — before being shared with transplant surgeons and teams at UHN.

Beyond UHN’s walls, the lab provides testing and supports transplant teams at St. Michael’s Hospital and The Hospital for Sick Children. From there, co-ordination between the teams at each hospital moves quickly to prepare for transplant surgery.

“We have very thorough quality controls and rigorous standards,” says Naglich. “What I value most is the close collaboration we have with the teams we deal with. Unlike many labs where you rarely interact directly with the clinical side outside of the lab, here we have that direct interaction with transplant physicians and coordinators across the city.”

The team also supports other Transplant Immunology Labs across the province by providing expertise and access to specialized testing tools and guidance that smaller health centres may not have.

Continuing care beyond the transplant

A patient’s immunology testing doesn’t end after the surgery. The lab continues to test and monitor patients for years after their transplant to continue tracking immune responses and watching for signs of rejection.

“Even after a transplant is completed, there’s still a risk that the patient may develop antibodies and begin to reject the organ over time. Our job is to stay ahead of that so we continue regularly monitoring the patient after transplant for life,” says Dr. Kiernan.

“We’re here for as long as they need us.”

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Physician shortage: What will the hospitalist shortage look like in 10 years?

Edusehat — Wed, 10 Jun 2026 22:00:08 +0700

New data show that the country’s physician shortage will continue to rise, leaving a gap of nearly 140,000 physicians by 2038. But some specialties—and some regions of the country—will actually have a surplus of physicians.

Data from the National Center for Health Workforce Analysis show that the physician workforce will go from a projected shortage of 113,380 physicians in 2028 to 141,160 in 2038. That gap between the number of physicians the country needs and actually has will grow from 11% in 2028 to 12% in 2038.

While the U.S. will face a shortage of more than 141,000 physicians in 2038, the problem will be most acute in several southern states. Florida, for example, will face a shortage of more than 32,000 physicians by 2038, and Texas will see a shortage of 24,450.

Those data reflect the trend that nonmetropolitan parts of the country will continue to see the greatest physician shortages. Those areas will have only 42% of the physician workforce they need (a shortage of 58%) compared to metro areas, which will face a shortage of 5% by 2038.

Physician shortage by specialty

Thirty of the 35 physician specialties profiled in the report are projected to have shortages in 2038. Five specialties are projected to have a surplus of physicians. Here’s a look at the top five in each category.

Five specialties with biggest shortages in 2038

Vascular surgery: 66%.

Ophthalmology: 72%.

Thoracic surgery: 73%.

Plastic surgery: 74%.

Family medicine: 76%.

Five specialties with a surplus of physicians in 2038

Emergency medicine: 116%.

Critical care/pulmonology: 112%.

Endocrinology: 109%.

Neonatology: 106%.

Neurology: 104%.

Hospitalist shortage

In 2038, the data say there will be a projected 39,590 hospitalists. That will leave a shortage of 11,380, meaning that the country will have 78% of the hospitalists it needs, or a shortage of 22%.

For 2023, by comparison, the National Center for Health Workforce Analysis says there was a shortage of 2,860 hospitalists, or enough to meet 93% of the nation’s need. By 2026, the shortage of hospitalists had already grown to 5,350, meeting 88% of the nation’s need. While the shortage continues to grow in raw numbers through 2038, the gap as a percentage hovers around the high 80 percent mark.

In 2038, the biggest shortage of hospitalists will be in Florida; the state will have 2,500 out of 4,030 hospitalists it needs, or 62%. Texas will have the second biggest shortage, with 2,860 out of 4,180 hospitalists it needs, or 68%.

Percentage-wise, states with the biggest shortage in 2038 will include Mississippi (with 48% of needed hospitalists), Oklahoma (49%) Kentucky (52%) and Alabama (51%).

Vermont, on the other hand, will have 130 hospitalists when it needs 90, giving the state the biggest surplus (144%). Connecticut comes in a close second, with physician workforce projections saying the state will have 133% of needed hospitalists (640 out of 480).

Other states with projected surpluses of hospitalists in 2038 include Massachusetts (128%), Hawaii (110%), New York (107%) and North Dakota (100%).

Physician supply by specialty in 2038 (percent of projected need)

All physicians: 88%.

Vascular surgery: 66%.

Ophthalmology: 72%.

Thoracic surgery: 73%.

Plastic surgery: 74%.

Family medicine: 76%.

Hospital medicine: 78%.

Allergy and immunology: 83%.

Anesthesiology: 83%.

General internal medicine: 83%.

Geriatrics: 84%.

Pathology: 84%.

Cardiology: 85%.

Nephrology: 85%.

Physical medicine/rehabilitation: 85%.

Ob-gyn: 86%.

Pediatrics: 86%.

Otolaryngology: 87%.

Radiation oncology: 87%.

Neurological surgery: 89%.

Orthopedic surgery: 88%.

Radiology: 90%.

General surgery: 91%.

Infectious diseases: 91%.

Rheumatology: 91%.

Dermatology: 95%.

Hematology/oncology: 96%.

Colorectal surgery: 98%.

Gastroenterology: 98%.

Neurology: 104%.

Neonatology: 106%.

Endocrinology: 109%.

Critical care/pulmonology medicine: 112%.

Emergency medicine: 116%.

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T-Cell Synapse Formation Is Restrained by PTPN22–PSTPIP1 Signaling

Edusehat — Wed, 10 Jun 2026 11:15:09 +0700

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.

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Fifteenth In Vivo Lentiviral Vector-Based Therapeutic Technology Added to VIVEbiotech’s CGT Platform

Edusehat — Wed, 10 Jun 2026 04:05:13 +0700

Spain-based CDMO VIVEbiotech added its 15^th 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.

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Incyte to Acquire Vega Therapeutics for Up-to-$2B, Growing Hematology Pipeline with Phase III VWD Candidate

Edusehat — Wed, 10 Jun 2026 04:05:12 +0700

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 67^th 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

Edusehat — Wed, 10 Jun 2026 04:05:11 +0700

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.

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Complete Connectome of Fruit Fly Central Nervous System Now Open-Source

Edusehat — Wed, 10 Jun 2026 00:25:09 +0700

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.

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Using real-time brain signals to predict and prevent attention lapses in kids

Edusehat — Wed, 10 Jun 2026 00:20:13 +0700

Key Findings

• In children with epilepsy, researchers used machine learning and intracranial recordings to discover a signal that predicted attention lapses.

• Stimulating the brain at precise moments helped children keep their focus despite difficult tasks.

• A non-invasive pulse to that same target spot also significantly improved reaction time and accuracy.

These first in-human findings offer a precision new path to help children maintain attention.

Inside a world-leading deep brain stimulation program at The Hospital for Sick Children (SickKids), researchers have discovered a brain signal that predicts when a child is about to lose attention — and that a brief, targeted intervention in response to that signal can restore their focus.

Their study, published in Nature Neuroscience, represents the first time this approach for attention control has been studied in people.

“Few aspects of the human experience are as integral as attention,” says lead author Dr. George Ibrahim, neurosurgeon and Senior Scientist in the Neurosciences & Mental Health program. “It shapes our perceptions, memories and interactions, but what happens when it is compromised?”

While fluctuations in attention are natural, disruptions in “attentional flexibility” that occur in youth with attention deficit/hyperactivity disorder (ADHD) can have negative behavioural, psychosocial and academic consequences. Medications used today are limited in their impact because, up to now, we didn’t know exactly how attention lapses happen nor which neural networks in the brain are involved.

Unique study offers new insights in the brain

The study began first with an attentional set-shifting task — which measures how attention shifts between stimuli — in a group of 30 children with epilepsy, a condition that significantly raises one’s risk for ADHD.

The team performed intracranial recordings, in which electrodes record directly from the depth of the brain, enabling them to monitor neural activity in milliseconds. At the same time, they used machine learning models to predict fluctuations in attention — which is a world-first. Through the models, they pinpointed a specific pattern of brain activity that anticipated slow or fast attention shifts in all children over several days.

“We were amazed to detect a signature in the brain that arose just before each child’s attention shifting performance was delayed,” says Dr. Nebras Warsi, first author and paediatric neurosurgeon-scientist studying in the Ibrahim Lab. “With precision electrical stimulation right at these moments in time, every child stayed engaged despite the difficult tasks and performed them faster and more accurately.”

During 20- to 30-minute tasks, they monitored the children’s performance through eye-tracking, reaction time and accuracy to spot lapses in attention. The brief electrical stimulation saved their attention only when delivered right at exact moments; when delivered at other times, the participant’s performance declined.

In short: timing was everything.

Study participant Skylar answering questions with brainwave recording at right.

Expanding scope to other children

Could the same results be expected in other children without epilepsy? To find out, the researchers used non-invasive magnetoencephalography imaging in 37 typically developing kids and 25 with ADHD.

By targeting that same signal in their brains, researchers could once again predict attention delays. Next, they used noninvasive transcranial magnetic stimulation-electroencephalography (TMS-EEG) and found that one pulse, delivered to that target area, led to sig1nificantly improved reaction time and accuracy. This time with a simple EEG cap and TMS coil, with no need for intracranial brain electrodes.

This suggests long-term potential for non-invasive tools that could support attention right when it’s needed most.

While these technologies are still in their early days, Ibrahim says this opens a promising new direction for understanding attention challenges and being able to advance Precision Child Health at SickKids, and around the world, by supporting each child in an individual way.

“Many people are studying neurological and neuropsychiatric disorders in adults, but very few people are expanding into paediatrics,” says Ibrahim, who is also the Abe Bresver Chair in Functional Neurosurgery. “We need to ethically advance possible therapies for children and youth and, to do that, we need to understand neural circuitry. The potential to change the lives of so many children is profoundly important.”

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David Sinclair plans to test whole-body rejuvenation drugs in the XPrize competition

Edusehat — Tue, 09 Jun 2026 20:55:02 +0700

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

Edusehat — Tue, 09 Jun 2026 17:20:04 +0700

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

Edusehat — Tue, 09 Jun 2026 10:05:10 +0700

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.”

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

Edusehat — Tue, 09 Jun 2026 10:05:09 +0700

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

Edusehat — Tue, 09 Jun 2026 06:30:09 +0700

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.

The Pros and Cons of a Medical School Application Essay Editor

Edusehat — Tue, 09 Jun 2026 03:15:04 +0700

The medical school application process is one of the most competitive and challenging journeys you will undertake. Many people will often say, “the hardest part of medical school is getting into medical school!”. Almost every year, the number of people applying to medical school increases and it is becoming evermore competitive to gain acceptance to the top programs in the country.

Among the many components of the application, the personal statement and secondary essays play a crucial role in distinguishing applicants from their peers. It is one of the only areas on the application where you can share your voice and personality. Given the high stakes of this application process, many students seek professional editing services to refine their essays.

However, students often wonder if using a medical school application essay editor is the right choice? Below, we explore the pros and cons of using an editor, the different options available, and important considerations prior to applying to medical school.

Understanding the Role of a Medical School Essay Editor

A medical school application essay editor is a professional who reviews, revises, and enhances personal statements and secondary essays to improve their quality and effectiveness. These professionals often consist of medical students, residents, attendings, former admissions officers, or writers with no formal medical training.

The role of an editor can vary widely, from providing minor grammar corrections to deeply restructuring essays for better clarity and impact. Some editors work independently, while others are part of established for hire groups or are even hired by colleges and universities to assist their students. The decision to hire an editor should be based on the applicant’s needs, writing skills, and budget constraints.

It is always recommended that you have multiple different people review your essay. We highly recommend that you have people in and outside of medicine read it so you can gain different perspectives on your writing. Often, people will reach out to their friends, siblings, parents, or mentors for advice. Regardless if you work with a formal medical school application essay editor or not, we recommend you reach out to these other resources if possible. However, there are some unique pros (and cons!) that come with using a formal editor.

Pros of Using a Medical School Application Essay Editor

1. Enhanced Clarity and Structure

Admissions committees read thousands of essays, and a well-structured, clear, and compelling narrative can make a significant impact. If your writing sounds “clunky” or is difficult to follow, it can be extremely challenging to determine what the main takeaways are from your essay. Editors help you refine your writing, ensuring coherence and a logical flow. They will help you eliminate redundancy in your writing, organize your ideas more effectively, and ensure that each paragraph transitions smoothly into the next. Remember, the admissions officers read thousands of essays each year, do not make their job more difficult!

2. Polished and Professional Writing

Even the strongest applicants may struggle with grammar, sentence structure, or wordiness – this is totally OK! Similar to the point made above, editors will fine-tune these aspects to present a polished and professional essay. Again, when your essay is refined and easier to read, there is a higher chance there will be a lasting impression on the admissions officer.

3. Objective Feedback

Friends and family may offer feedback, but given their personal relationship to you, they may not always be fully honest (or conversely, they may be too critical!). Professional editors provide an unbiased, expert perspective. They have read and written hundreds if not thousands of personal statements and, therefore, have the knowledge and experience to tell you what makes a good essay and what makes a bad essay. They understand what medical schools are looking for and can guide you crafting a personal statement that emphasizes your strengths. Many students often receive conflicting advice from different personal sources, and an experienced editor can help filter out unhelpful suggestions and provide clarity in feedback you receive from others.

4. Stronger Storytelling

A compelling personal statement tells a story about who you are rather than simply listing your achievements (this is a common pitfall for many applications). Editors can help you craft a narrative that resonates with admissions committees by showcasing your unique journey, and mentioning your accomplishments along the way. By highlighting your personal experiences, challenges, and aspirations, a strong essay will provide deep insight into your motivations, character, achievements, and failures.

5. Increased Competitiveness

Given the intense competition for medical school admission, a well-edited essay will improve your chance by making their application more memorable. Overall, much of the medical school application is quantitative (MCAT, grades, etc.). There are only a few areas where you get to “speak”. Therefore, the personal statement can serve as a critical deciding factor between who is offered admission and who is denied. Many applicants have similar grades and test scores, so a strong essay can serve as that distinguishing factor.

Cons of Using a Medical School Application Essay Editor

1. Risk of Losing Authenticity

Admissions committees value authenticity (as should you!). Over-editing with a professional can lead to an essay that sounds generic or overly polished, potentially raising concerns about the applicant’s true voice. The best essays are those that maintain the applicant’s natural tone and personality in a manner that has the highest level of clarity and impact. When an essay becomes too refined, it may lose the personal quirks and unique storytelling that makes you, you! To avoid this, you should ensure that their final draft still reflects your personal journey and writing style, rather than sounding like a professionally scripted piece. A good professional editor will know this, and will work with you to balance maintaining your voice and story with clarity and structure.

2. Cost Considerations

Professional editing services can be expensive, with some charging hundreds of dollars per hour. For students on a tight budget, this can be challenging. Students and their families will often view any form of educational support (tutor, teacher, essay editor, etc.) as a significant investment in the future as a physician.

While some students may see this as a worthwhile expense, others may struggle to justify the cost, especially if they are applying to multiple schools and need several essays reviewed (fortunately, you can use the same essays at many schools, but not all of them). Rather than one-on-one editing, more affordable alternatives exist such as peer review groups and university writing centers, that can provide helpful feedback without the price tag.

We recommend you carefully evaluate whether the benefits outweigh the cost before committing to any service. Additionally, not all editors are created equal! Make sure you look for a person/company that has a track record of success. Additionally, if you do not feel comfortable with how they are advising you, that should be a red flag! This is meant to be a team effort, not a one way street.

3. Ethical and Policy Concerns

Some applicants (and admissions officers) worry about whether medical school essay editing should be allowed. While revising an essay with others is obviously acceptable, and expected, having someone else write the essay is unethical and can lead to serious consequences if discovered. If any professional ever offers to write your essay, this is a serious red flag and you should not work with that person/company. Many, if not all, medical schools have strict policies against plagiarism or ghostwriting. They expect your essay to be genuine, and if admissions officers suspect that an essay has been excessively edited or rewritten by another party, it could raise red flags and often lead to immediate rejection. Make sure to use editing services that focus on guidance and revision rather than writing or rewriting.

4. Over-Reliance on External Help

You should remember that no professional editor can replace reflection and genuine effort in writing an impactful essay. Additionally, writing is a learned skill that will follow you throughout your career (even if you do not end up practicing medicine!). Relying too much on an editor can hinder your personal growth and development in communication skills. Medical professionals require strong writing abilities for research, patient notes, and presentations. Developing these skills early is beneficial. If you lean too heavily on external help, you may struggle later when faced with writing tasks that require independent thought and communication. Remember, it is important to view the essay-writing process as an opportunity to develop your communication skills rather than just rely on someone to overcome another hurdle in the journey to becoming a physician.

The Emerging (and controversial) Role of AI in Medical School Essay Editing

AI has become an increasingly prominent tool in essay editing, offering both significant advantages and severe drawbacks for medical school applicants. AI-driven tools such as ChatGPT, Grammarly, and Gemini can provide instant feedback and help you refine your narratives with minimal cost and effort. However, their effectiveness, accuracy, and, importantly, ethical implication, must be carefully considered.

Pros of AI in Medical School Essay Editing

Instant and Cost-Effective Assistance
- AI-powered tools provide real-time suggestions for grammar, clarity, and structure at little to no cost, making them accessible to all applicants. We believe that AI will greatly alleviate the socioeconomic barriers and disparities that some students face during this application process.
Consistency and Efficiency
- AI editors can process multiple drafts quickly, ensuring that students receive consistent feedback throughout the writing process.
Improvement in Readability and Style
- AI tools can, but not always, help refine sentence structures, eliminate redundancies, and enhance vocabulary, making essays more polished and professional.
Brainstorming
- AI can serve as a sounding board to help you develop your thoughts and ideas. It can be like speaking with a friend or colleague to put together a rough outline of what you hope to accomplish with your personal statement.

Cons of AI in Medical School Essay Editing

Lack of Personalized Feedback
- AI lacks the nuanced understanding of human emotions, storytelling, and individual experiences that professional editors provide. Additionally, AI is only as good as the data it is trained on. The medical school application process is a very subjective and personalized experience – it is very unlikely that AI can replicate what a student creates and how an admissions officer will respond.
Potential for Generic Output
- AI tools often rely on patterns and may generate suggestions that result in generic or overly structured writing, reducing the applicant’s unique voice. As you know, it is extremely easy to tell when something is written by AI – it can sound too structured, too generic, and too formal. Your voice and writing style is one of the strongest attributes to a good personal statement, and AI is not trained on your writing alone to replicate that!
Ethical Considerations
- This is one, if not the most important, con of using AI. Some medical schools view heavy AI-assisted editing as compromising authenticity and blatant plagiarism. Over-reliance on AI tools will raise concerns about the originality of your work. You should assume that every single medical school you apply to will screen your application with AI-detection software. Therefore, relying too heavily on AI could compromise your entire application and dreams of becoming a doctor
Inability to Provide Strategic Admissions Insights
- AI can refine your language and structure, but it cannot offer strategic advice on how to frame personal experiences/accomplishments to align with an admissions committee’s expectations.

Balancing AI and Human Editing – our recommendations

To maximize effectiveness, applicants should use AI as a supplement tool rather than a replacement for your personal effort or the feedback from others. A recommended approach includes:

Brainstorming: Work with AI to help flush out ideas, you can share your personal strengths, stories, accomplishments, failures, etc. and use AI to help organize your thoughts into a cohesive outline.
First Draft with AI: Using AI to check grammar and readability – AI can serve as a great “first pass” of your essay. Again, it may structure your writing in a way that literally sounds written by AI, so be careful and weary of the suggestions that it makes.
Peer Review: Having trusted peers, friends, family, or mentors provide personal insights – your final review should not be completed with AI!
Final Review with a Professional Editor or Advisor: They can help ensure that the essay is compelling, strategic, and authentic in a way that aligns with what the admissions committees are looking for.

Options for Medical School Essay Editing

1. Free Online Editing Tools

Platforms like Grammarly, Hemingway Editor, or ChatGPR (all of which use AI) can help with grammar and readability but lack personalized feedback. While these tools can catch minor errors, they do not provide trusted insights into storytelling, structure, or content relevance.

2. Peer Review

Having trusted family, friends, peers, mentors, or professors review your essays can provide valuable insights at little to no cost. Current medical school students, pre-health advisors, and professors with experience in medical education can offer useful feedback from an admissions perspective. Remember, they may not give you the most unbiased feedback given their personal relationship with you – but try and ask them to be as honest as possible!

3. Professional Editing Services

Services like Elite Medical Prep’s Essay Editing offer expert guidance tailored to medical school applications. These services often include multiple rounds of editing, detailed feedback, and consultations with experienced admissions consultants. However, refer back to the potential cons of professional editing services and make sure to find one that aligns with your goals, ethics, and finances.

4. Pre-Health Advisors

Many universities have advisors who can provide essay feedback as part of their pre-medical advising programs. In our personal experience, these advisors can be very hit or miss so you will need to explore their reliability on your own. However, these advisors often have experience reading successful essays and can provide guidance on content, structure, and tone.

Conclusion

Using a medical school application essay editor has clear advantages like improving clarity and increasing competitiveness. The advent of AI has added a new dimension to the editing process, offering efficiency and potential cost savings, but also raising significant concerns about authenticity, originality, and honesty. To craft a compelling and well-structured essay, you should consider a balanced approach that integrates peer feedback and professional guidance.

For expert essay editing tailored to medical school applications, visit Elite Medical Prep’s Essay Editing. Best of luck!

The post The Pros and Cons of a Medical School Application Essay Editor appeared first on Elite Medical Prep.

CiteSentinel Launched to Detect and Prevent AI Hallucinations in Legal Citations

Edusehat — Tue, 09 Jun 2026 02:55:12 +0700

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.”

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.

Christopher Mancuso, DO, Joins Optima Dermatology’s Nashua, NH Practice

Edusehat — Tue, 09 Jun 2026 02:55:09 +0700

Dr. Mancuso Brings More Than a Decade of Expert Dermatology Experience to the Nashua Area

Optima Dermatology is excited to welcome board-certified dermatologist Christopher Mancuso, DO, to its Nashua, NH practice. Dr. Mancuso has been providing dermatology services to patients for more than 10 years, specializing in medical and surgical dermatology care for a range of skin conditions, including acne, psoriasis, eczema, skin cancer, and more.

Dr. Mancuso earned his Doctor of Osteopathic Medicine at Lake Erie College of Osteopathic Medicine and completed his residency and internship training in dermatology at St. Barnabas Hospital in New York.

He is passionate about clinical research and helping every patient find relief for their unique situation with individualized, cutting-edge treatments.

Passionate Care Delivered by Passionate Experts

Dr. Mancuso is excited to deliver personalized and comprehensive care to patients in the Nashua area, especially for often complex skin issues that require dedicated treatment plans and an understanding ally.

His approach to care is aligned with Optima’s mission of treating patients the way we would treat our own family and friends.

We recently had the opportunity to learn more about Dr. Mancuso, his approach, and the path that led him to become a board-certified dermatologist.

What made you decide to pursue a career in dermatology? What did the path to get there look like for you?

In high school, I took a class in anatomy and physiology and realized how interesting the human body is. I happened to also be seeing a dermatologist to treat my severe acne. I was so appreciative for the confidence they were able to restore in me, and I knew that I wanted to do the same for others like me.

What is your favorite aspect of being a board-certified dermatologist?

I love that we treat so many varied medical conditions, from acne to skin cancer to rare cutaneous manifestations of internal diseases like Crohn’s disease. Being able to perform surgeries like skin cancer excisions and using devices like blue light allow me to have a more hands-on approach to care.

What is your approach to treating your patients?

I try to communicate clearly and thoroughly explain the diagnosis, treatment, and possible side effects. The best way for me to instill confidence in my patients is to explain my exact thought process.

What is your top tip for taking care of your skin?

The biggest thing is sun protection, including avoiding direct sunlight, wearing sunscreen, and covering up with clothing.

What is one thing you want your patients to know about you?

I treat all of my patients with respect, and I hope they see me as a friendly advisor and partner in their healthcare.

What do you like to do when you are not in the office treating patients?

I enjoy spending time with my wife and 3 young sons. I like outdoor activities like hiking and running, but I am a bit of a sci-fi and Lord of the Rings nerd as well.

What to Expect From Dermatological Care Delivered by Dr. Mancuso and His Team

Patients visiting with Dr. Mancuso and his team at Optima Dermatology will receive the highest quality care backed by decades of advanced training and experience.

Dr. Mancuso is currently accepting new patients at our Nashua, NH location. Request an appointment online or call us at (603) 441-3223.

The post Christopher Mancuso, DO, Joins Optima Dermatology’s Nashua, NH Practice appeared first on Optima Dermatology.

CRISPR Shreds Undruggable Cancer Cells with Precision

Edusehat — Mon, 08 Jun 2026 23:20:08 +0700

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

Edusehat — Mon, 08 Jun 2026 19:45:03 +0700

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

Edusehat — Mon, 08 Jun 2026 08:55:09 +0700

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.”

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.

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New State Programs Allowing Physicians to Practice Without Residency: What Pre-Meds and Medical Students Need to Know

Edusehat — Sun, 07 Jun 2026 04:00:05 +0700

If you’ve spent any time on medical school or residency forums lately, you’ve probably seen headlines about new state laws allowing physicians, especially international medical graduates (IMGs), to practice without completing a U.S. residency. It can sound surprising, even a little confusing, especially if you’ve always thought that residency is a non-negotiable step in becoming a practicing physician in the United States.

So what’s actually changing? And more importantly, what does it mean for you? Let’s walk through this evolving landscape in a clear, grounded way, because while these policies are real, they’re also nuanced, limited, and still developing.

Why These New Pathways Are Emerging

For decades, the traditional path to practicing medicine in the U.S. has been straightforward: graduate medical school, complete a U.S. residency, pass licensing exams, and then practice independently.

But the system has been under strain. The U.S. is facing a well-documented physician shortage, particularly in rural and underserved areas. At the same time, thousands of highly trained international physicians have been unable to practice due to the bottleneck of residency positions.

In response, many states have begun creating alternative licensing pathways. As of 2025–2026, at least 17–18 states have enacted laws allowing internationally trained physicians to pursue licensure without repeating a U.S. residency under certain conditions. (American Medical Association). These policies are designed less as a shortcut, and more as a workforce solution.

What These Programs Actually Allow (And What They Don’t)

It’s important to clarify one key point: these laws do not mean that anyone can skip residency and immediately practice independently. Instead, most states offer provisional or supervised licenses. These allow internationally trained physicians to work in structured environments, often under supervision and frequently in underserved areas.

For example, some states require physicians to work for several years under supervision before transitioning to full licensure. Others mandate employment at specific healthcare facilities or limit practice to rural communities. (MedSmarter Prep).

In many cases, these pathways still require:

Passing some or all USMLE exams
ECFMG certification
Several years of prior clinical experience abroad

So while residency may be waived, the bar for entry remains high.

States Leading the Change

The movement is growing quickly, with multiple states adopting or proposing these pathways. States like Florida, Texas, Tennessee, Virginia, and Illinois have already implemented programs allowing IMGs to practice without repeating U.S. residency under specific conditions. (American Medical Association).

For example:

Florida allows experienced international physicians to practice with a provisional license tied to employment and supervision. (MedSmarter Prep)
Tennessee offers one of the more flexible pathways for physicians with significant prior experience. (Medtigo)
Texas recently expanded efforts to recruit foreign-trained physicians as part of addressing workforce shortages. (The Texas Tribune)

At the same time, additional states, including New York, New Jersey, and Arizona, are actively considering or introducing similar legislation. (The Match Guy – Best Residency Guidance). This is not a single policy change, it’s a nationwide trend.

Who Is Actually Eligible?

These pathways are not designed for U.S. medical students or graduates who want to bypass residency. They are primarily intended for internationally trained physicians who have already completed medical education and often residency-equivalent training abroad.

Common eligibility criteria include:

A valid medical degree and license from another country
Several years (often 3-5+) of independent clinical practice
Passing U.S. licensing exams (partially or fully)
Securing a job offer in an approved healthcare setting

Many programs also require physicians to practice in shortage areas for a set number of years before qualifying for full licensure. (MedSmarter Prep). In other words, these are not entry-level positions, they’re designed for experienced physicians.

Potential Benefits of These Programs

From a systems perspective, these policies aim to address gaps in healthcare access. Allowing qualified international physicians to enter the workforce more quickly can expand access to care in underserved areas, reduce physician shortages, and utilize an already-trained global workforce.

For IMGs, these pathways offer a long-awaited alternative to the highly competitive U.S. residency match. And for patients, especially in rural communities, this could mean shorter wait times and increased access to care.

Risks and Controversies

That said, these policies are not without debate. One of the biggest concerns is variability in training. Residency in the U.S. is highly standardized, whereas international training can vary widely depending on the country and institution.

Critics worry about:

Differences in clinical training standards
Patient safety and supervision consistency
Fragmentation of licensure requirements across states

Even within these new programs, many states have built in safeguards, such as supervision requirements and gradual transitions to full licensure, to address these concerns. Still, the long-term outcomes of these policies are not yet fully understood.

Career Implications for U.S. Medical Students

If you’re a pre-med or current medical student, this might raise an obvious question: Does this change anything about my path?

Short answer: not really. For U.S. graduates, residency remains the standard, and overwhelmingly preferred, pathway to practice. These new laws are not designed to replace residency, and they do not provide an alternative route for U.S.-trained students.

Residency is still essential for:

Board certification
Competitive job opportunities
Fellowship training
Long-term career flexibility

In fact, most healthcare systems and employers will continue to prioritize residency-trained physicians.

What This Means for the Future of Medicine

What’s happening now is part of a broader shift in how the U.S. healthcare system thinks about workforce shortages and global talent. These programs are likely to continue expanding, but they will also continue evolving. Expect ongoing adjustments to eligibility criteria, supervision requirements, and long-term licensing structures.

For now, think of these pathways as targeted solutions in very specific scenarios, not a replacement for traditional medical training.

Final Thoughts

The idea of practicing medicine in the U.S. without residency would have seemed almost impossible a decade ago. Today, it’s becoming a reality in certain states, but only under specific, structured conditions. If you’re on the traditional path to medical school and residency, nothing about your roadmap has fundamentally changed. Residency is still the cornerstone of physician training in the U.S.

But these new policies are worth understanding, not because they replace your path, but because they reflect a healthcare system that is actively adapting to new challenges. And as medicine continues to evolve, being informed, not just about exams and applications, but about policy and systems, will make you a stronger, more thoughtful future physician.

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Bio-Techne, Refeyn Partner on Workflow for Bispecific Antibody, Biosimilar Characterization

Edusehat — Sat, 06 Jun 2026 05:40:13 +0700

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.”

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Twin Prime Editing Enables Rapid Trait Stacking in Crops

Edusehat — Sat, 06 Jun 2026 05:40:12 +0700

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

Edusehat — Sat, 06 Jun 2026 05:40:11 +0700

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.

Derm Deep Dive: Do Pimple Patches Work?

Edusehat — Sat, 06 Jun 2026 05:40:07 +0700

A Dermatologist’s Guide to At-Home Acne Treatments

We’re back with another Derm Deep Dive, a series that provides expert advice from our board-certified dermatologists about common skin conditions or treatments. June is Acne Awareness Month, a perfect opportunity to educate about this common skin issue and how to treat it.

Patients often ask us about at-home acne treatments and whether pimple patches and other topical treatments really work. Let’s break down the science behind popular acne solutions so you can set realistic expectations and goals for clearer skin.

Can I Treat My Acne At Home?

Acne affects people of all ages and skin types, and while genetics and hormones do play a role, your diet, stress levels, and even the type of makeup you wear can cause acne flare-ups.

When breakouts happen, there are a number of approaches you can take at your home to treat and manage your acne.

Addressing external factors like diet, stress, and makeup is a great start. Sometimes, simple lifestyle changes can have notable impacts on reducing acne flare-ups.

However, acne almost always requires more targeted treatments, and while doing so at home can provide some relief, results depend on what you use and how you use it.

If you’re unable to see your dermatologist right away, it’s important to know how to treat your acne at home for the best results.

Let’s dive into at-home treatments and what works best for acne versus other types of pimples.

Do Pimple Patches Really Work?

If you’ve ever had a pimple, you’ve probably heard of pimple patches; small, round stickers that attach to pimples and not only protect them from external irritations, but absorb excess oil and dirt from the pimple to reduce redness and inflammation.

These at-home treatments are an understandably popular option because they’re easy to use and can offer quick results.

However, if you have acne, we won’t bury the lead – pimple patches won’t work for you.

While pimple patches can help decrease inflammation, they only target the surface of the skin and are unlikely to provide results for acne, which is rooted more deeply in the skin.

Pimple patches work best for:

Whiteheads
Pimples that have come to a head
Covering pimples to protect them from picking or other external irritation, which can cause scarring or infection

Pimple Patches Deliver Pimple-Fighting Power

Pimple patches are typically made with hydrocolloid, a gel-forming material used to heal wounds, and contain added ingredients that can include:

Salicylic Acid: Helps unclog pores and remove excess oils.
Tea Tree Oil: Effective at drying out the pimple, killing bacteria, and decreasing inflammation and redness.
Hyaluronic Acid: Hydrates the skin, counteracting drying from salicylic acid or tea tree oil.
Niacinamide: Helps stabilize the skin barrier and improve skin tone and texture.
Benzoyl Peroxide: Acts as an antiseptic, killing bacteria in the pimple.

When used as targeted treatments outside of a pimple patch, these ingredients can be effective at fighting acne and are often recommended by dermatologists as part of an acne treatment plan.

We also recommend retinoids, which regulate skin cell turnover to help prevent clogged pores for longer-term acne control.

Beyond the products, at-home acne treatment requires additional diligence to ensure you’re not overdoing it.

Quick Tips for Treating Acne at Home

When we advise patients about at-home acne care, we focus on three key factors:

Ingredients: Use products made with natural ingredients like tea tree oil, which helps reduce inflammation and kill bacteria. Avoid pore-clogging oils, irritants like alcohol, and heavy comedogenic ingredients.
Routine: How often are you washing your face and applying products – and which products are you using? Managing acne flare-ups at home goes hand-in-hand with a gentle skincare routine that supports the skin barrier.
Treatments: If you’re using acne-specific treatments at home, we recommend first consulting with your dermatologist to make sure you’re selecting the best options for your specific needs.

Conversely, there are three things we advise patients not to do when treating their acne at home:

Don’t constantly switch up your products. If a product is causing skin irritation or a bad reaction, absolutely swap it out right away. Otherwise, use your product for at least 4-6 weeks (the length of a single skin cycle) to know if it’s working for you. And if it’s doing the trick, there’s no need to try something new.
Don’t overload your skin with actives. Actives are important components in acne care as they treat at the cellular level to target the root of your issue. However, using too many actives can irritate the skin barrier and make your acne worse. We recommend 1-2 actives per routine, ideally in the morning and at night.
Don’t expect immediate results. We know acne is a difficult skin condition to live with, and can take multiple treatments to get it under control. You might feel like nothing is working, but we’re here to remind you that it does take time, and you always have an expert dermatologist to consult if the going gets tough.

Avoid These Common Acne Treatment Mistakes

Pimples are a nearly universally-shared experience, and pop culture and social media has put some unfortunate ideas out there about pimple treatment that as dermatologists we simply can’t abide.

Steer clear of these common acne treatment mistakes:

Picking and popping
- Increases inflammation
- Introduces bacteria into the skin
- Raises the risk of scarring
Harsh scrubbing
- Can damage or irritate the skin barrier
- May worsen inflammation and redness
Over-exfoliation
- Strips the skin of natural oils necessary to prevent breakouts
- Can lead to excess dryness and pimple development
Overnight fixes
- Acne cannot be treated overnight with a patch or topical product
- Quick fixes rarely work and can worsen acne symptoms

When to Shift From At-Home to In-Office

At-home treatments are tempting – they don’t require any office visits. But if your at-home treatment isn’t working, don’t wait too long to see a dermatologist.

In addition to being difficult to live with, untreated acne can lead to greater consequences for your skin:

The development of painful cystic acne
Scars or dark spots
Breakouts that never seem to improve or go away, even with treatment
Frequent or severe flare-ups

Acne can impact your life, but your dermatologist can help you get it back under control.

Take Back Control of Your Acne

At Optima Dermatology, we create personalized acne treatment plans that can include prescription medications, in-office procedures, or combination therapies that go beyond what’s available over the counter.

Schedule an appointment with our team for an acne evaluation and to discuss the best path forward.

FAQs About Pimple Patches and At-Home Acne Treatments

Do pimple patches work on all types of acne?

No. Pimple patches work best on surface-level whiteheads. They are not effective for cystic acne or deep breakouts beneath the skin.

How long should I leave a pimple patch on?

Most patches should be worn for 6–12 hours or until they turn opaque, indicating they’ve absorbed fluid.

Can I use salicylic acid, benzoyl peroxide, and adapalene together?

Yes, but carefully. Using multiple active ingredients at once can irritate the skin. Start slowly and consider alternating products or consulting a dermatologist.

Why isn’t my acne improving with over-the-counter treatments?

It may take 6–8 weeks to see results. If there’s no improvement after that, your acne may require prescription-strength treatment.

Is it ever safe to pop a pimple?

Dermatologists strongly advise against it. Popping pimples can increase inflammation, delay healing, and lead to permanent scarring.

The post Derm Deep Dive: Do Pimple Patches Work? appeared first on Optima Dermatology.

Baby Botox®: Is It Effective for Early Prevention and More Natural Results?

Edusehat — Sat, 06 Jun 2026 05:40:06 +0700

The Pros and Cons of Starting Small Doses of Botox in Your 20s and 30s

The name for the newest trend in Botox^® – Baby Botox – may make some people do a double-take, but no, it’s not Botox for babies. Rather, it’s the practice of getting smaller, more targeted Botox injections to achieve subtle, more natural-looking results.

It has become a popular choice among individuals in their 20s and 30s because it can deliver a refreshed look without impeding natural facial expressions.

What Makes Baby Botox Different from Traditional Botox?

Like most trends today, Baby Botox gained traction on social media as part of the ever-growing dialog about preventive skincare. Coupled with greater access to aesthetic treatments and younger generations becoming invested in proactive solutions, the rise of Baby Botox took hold among individuals in their 20s and 30s.

Traditional Botox treatments, more common among individuals aged 40-60, reduce deep lines and wrinkles by relaxing the muscles, and are often associated with causing stiff or unnatural facial expressions.

Younger individuals favor Baby Botox, also known as MicroTox, as a preventive approach that delivers the benefits of Botox without that “frozen” look. Baby Botox can soften movement just enough to reduce the appearance of fine lines, achieving a more subtle look.

There is some debate over whether it’s a skin-smart move to get Baby Botox at an earlier age as a preventive strategy, so let’s take a look at how it really works and if it can lead to long-term smooth skin.

Does Starting Botox Early Make Sense?

There has been a shift in attitudes about fine lines and wrinkles; now, the focus is on prevention rather than treatment, and small, targeted Baby Botox injections are thought to delay the development of deeper, static lines over time.

The idea behind the efficacy of Baby Botox is simple: fine lines and wrinkles are caused by repeated facial movements like squinting, smiling, laughing, and frowning. Using small Botox injections starting at younger ages to slightly limit these movements can prevent deep lines from forming in the future.

But does it make sense to start Botox earlier in the hopes of longer-term wrinkle prevention?

Starting Botox in your 20s and 30s has some benefits:

Slows the formation of wrinkles. By reducing repetitive facial movements, early treatment may delay the formation of deeper lines later in life.
Reduces future treatments. Smaller, more consistent Botox injections may help maintain results rather than requiring increased doses later.
Produces natural-looking results. When done properly, Baby Botox can maintain facial movement while subtly smoothing skin. Some note that younger patients may experience smoother skin for longer, particularly in areas of higher movement like the forehead or around the eyes.

However, there are some arguments against starting Botox too early:

Your skin may not need it. If you don’t have any visible lines, Botox, even in small doses, likely won’t provide any meaningful benefit.
Injections aren’t a one-time fix. Botox requires ongoing maintenance every 3-4 months, so the earlier you start injections, the longer you’re committing to treatment – and the cost of those treatments, which adds up quickly.
It’s possible to overuse Botox. Poor technique or unnecessary treatment can lead to unnatural results, even with Baby Botox.

Botox is a treatment that should be tailored to your individual needs, not a blanket treatment for anyone looking to prevent wrinkles down the road.

Should I get Baby Botox?

Botox is an injection and carries risks like any treatment. Be sure it’s right for you before you start receiving injections.

You may be a good candidate for Baby Botox if:

You’re noticing faint lines or wrinkles associated with facial movements
You’re looking for subtle, natural results over time instead of dramatic changes all at once

You may want to wait on injections if:

Your skin is smooth, and you’re not seeing lines or wrinkles at rest or with movement
You’ve been drawn to Baby Botox because of the trend rather than a true need
You’re not ready to take on ongoing maintenance treatments

If you’re unsure whether Baby Botox is a skin-smart move for you, consult your certified aesthetic provider for an honest opinion. Preventive Botox can be a smart strategy for the right person, but it is not a universal recommendation or need for anyone in their 20s and 30s.

Your provider will evaluate:

Your facial anatomy and muscle movement
Your skin’s condition and any early signs of aging
Your long-term, individual skin health goals

You may learn that Baby Botox isn’t the right path for you. There are other strategies, such as daily suncare, retinoids, peptides, and hyaluronic acid, that can treat wrinkles and fine lines topically.

Find the Right Balance for Preventive Treatment

At Optima Dermatology, we create personalized treatment plans for all skin types based on your individual needs. If Baby Botox is the right solution for you, we have expert cosmetic dermatologists who can administer injections and help you reach your skincare goals.

Schedule a consultation with one of our highly trained and specialized medical aesthetic providers to learn how you can best prevent wrinkles and fine lines.

FAQs About Baby Botox

At what age should you start Botox for prevention?

There’s no “perfect” age. Some patients begin in their late 20s or early 30s if they notice early dynamic lines, but many people don’t need it until later.

Does Baby Botox last as long as regular Botox?
Not always. Typically, the duration of Botox is dependent on the dose administered. Because smaller doses are used, results may wear off slightly faster, often within 2–3 months.

Will starting Botox early prevent all wrinkles?
No. While it can help reduce muscle-related wrinkles, it doesn’t stop aging or address issues like sun damage or skin texture.

Is Baby Botox safer than traditional Botox?
It’s not necessarily “safer,” but when performed correctly, the lower doses may reduce the risk of an overdone or frozen appearance.

What are alternatives to preventive Botox?
While nothing will relax muscles the same way Botox can, daily sunscreen, retinoids, antioxidants, and professional skincare treatments can all play a major role in preventing early signs of aging.

The post Baby Botox®: Is It Effective for Early Prevention and More Natural Results? appeared first on Optima Dermatology.

A Billion-Dollar Deal, Trial Trouble, Biohub Updates, and Vaccine Research News

Edusehat — Sat, 06 Jun 2026 01:55:09 +0700

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:

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?

Edusehat — Fri, 05 Jun 2026 22:15:03 +0700

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

Edusehat — Fri, 05 Jun 2026 22:10:09 +0700

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.

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.”

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Novel Intracellular Pathway Identified That Protects Against Viral and Bacterial Infection

Edusehat — Fri, 05 Jun 2026 11:25:09 +0700

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&D‑seq Uses Base Editing to Map DNA–Protein Interactions in Single Cells

Edusehat — Fri, 05 Jun 2026 11:25:08 +0700

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

Edusehat — Fri, 05 Jun 2026 07:50:11 +0700

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.”

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Experimental Adjuvant Could Strengthen Mucosal Immunity with Injectable Polio Vaccines

Edusehat — Fri, 05 Jun 2026 07:50:10 +0700

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.

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Open Letter: In Support of Mandatory Nucleic Acid Synthesis Screening and Recordkeeping

Edusehat — Fri, 05 Jun 2026 07:50:09 +0700

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.

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Immune Response Activated by RNA Splicing Opens Targeted Therapies

Edusehat — Thu, 04 Jun 2026 05:55:16 +0700

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.

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Immune Cell Phenotyping: Cell Surface Architecture Informs Disease Biology

Edusehat — Thu, 04 Jun 2026 05:55:15 +0700

Erdinc Sezgin, PhD

Senior Lecturer
Karolinska Institutet

Hanna van Ooijen, PhD

Scientific Affairs Manager
Pixelgen Technologies

Broadcast Date: Tuesday, June 23, 2026

Time: 08:00 PDT, 11:00 EDT, 17:00 CET

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:

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NIIMBL to Support Vector Production and AI-Ready Training Projects

Edusehat — Thu, 04 Jun 2026 05:55:14 +0700

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.”

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Web App Helps Flag Antibodies Where Manufacturability Might Be an Issue

Edusehat — Thu, 04 Jun 2026 05:55:13 +0700

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.

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Bioproduction Pivots from Centralized to Regional Support

Edusehat — Thu, 04 Jun 2026 05:55:12 +0700

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.”

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Gentler Cell Separation Methods Gain Momentum

Edusehat — Thu, 04 Jun 2026 05:55:12 +0700

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.

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New Antibiotic, Manikomycin, Acts on Novel Ribosomal Target

Edusehat — Thu, 04 Jun 2026 05:55:10 +0700

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.”

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Cellares and TScan Agree to Evaluate Automated Manufacturing TSC-101 for Patients with Hematologic Malignancies

Edusehat — Thu, 04 Jun 2026 05:55:09 +0700

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.

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Small Molecules to Big Partnership: Incyte, Genesis Expand AI Collaboration to $1B+

Edusehat — Thu, 04 Jun 2026 05:55:08 +0700

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.

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Human Hookworm Engineered to Produce, Secrete Anti-Tetrodotoxin Antibody Into Preclinical Host Bloodstream

Edusehat — Thu, 04 Jun 2026 05:55:07 +0700

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.

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Why Passive Studying Is the #1 Reason Medical Students Don’t Improve

Edusehat — Wed, 03 Jun 2026 19:30:03 +0700

If you’re a medical student who feels like you’re studying all the time but your scores aren’t budging, you’re not failing, you’re just using a strategy that doesn’t work as well as you’ve been told.

You reread First Aid for the third time.
You watch every video in your resource stack.
You highlight until the page is neon.

And yet, when you sit down to do practice questions, your brain feels empty. Suddenly, nothing feels familiar anymore. This disconnect is one of the most frustrating experiences in medical school, and it’s incredibly common. The reason isn’t a lack of discipline, intelligence, or motivation. The reason is that most students rely heavily on passive studying, and passive studying creates the illusion of learning without producing real improvement. This is a problem I work with students on frequently in tutoring sessions because in college sometimes passive studying is enough to do well but medical school is a whole different ball game!

Once you understand why passive studying fails, and how active studying works, you can completely change how efficiently you learn, without adding more hours to your already packed schedule.

Why Passive Studying Feels Like It Should Work

Passive studying includes activities where you expose yourself to information with minimal effort required from you. Reading notes, rereading textbooks, watching lectures, highlighting, and even listening to educational podcasts all fall into this category.

These methods feel productive for a reason. They’re familiar, they’re relatively low stress, and they give you a sense of exposure to the material. When you reread a page and recognize what’s written, your brain gives you a comforting signal: I’ve seen this before. That sense of familiarity feels like learning. The problem is that familiarity is not mastery.

Medical exams don’t ask whether something looks familiar. They ask whether you can retrieve information without prompts, apply it in a new clinical context, and distinguish it from closely related alternatives. Passive studying never trains those skills, even though it can feel like you’re “doing the work.”

The Illusion of Learning That Traps So Many Students

One of the most dangerous aspects of passive studying is that it creates an illusion of competence. When you reread or rewatch something, your brain confuses recognition with understanding. The time spent is also a falsely reassuring component. If you spent 5 hours “studying,” surely you must’ve learned something right? Think again! You think you know the material because it looks familiar, not because you can actually produce it on demand.

This is why so many students walk out of exams thinking, “I knew that when I studied it.” What they really mean is, “I recognized it when I saw it.” Recognition is easy. Retrieval is hard. Exams require you to be able to retrieve and apply information at a high level.

Medical school intensifies this problem because the volume of information is enormous. Passive methods make it easy to feel like you’re covering a lot of ground quickly, but that speed comes at the cost of retention and application. When test day arrives, your brain can’t find what it needs because it never practiced pulling that information out on its own.

Why Passive Studying Breaks Down in Medical School

Passive studying might work temporarily for straightforward memorization, especially in earlier stages of education. Medical school is different. You’re not just learning facts, you’re learning systems, mechanisms, and clinical reasoning.

Exam questions rarely ask for isolated details. Instead, they present long vignettes that require you to integrate pathophysiology, clinical presentation, diagnostics, and management. They expect you to reason through unfamiliar scenarios using foundational knowledge.

Rereading and highlighting don’t train your brain to do this. They don’t teach you how to move from mechanism to symptom, or how to rule out tempting distractors. They don’t help you organize information in a way that’s accessible under time pressure.

That’s why students often feel blindsided by exams despite hours of studying. The issue isn’t effort, it’s mismatch. Passive studying prepares you to recognize information, but exams demand that you generate and apply it.

What Active Studying Actually Means

Active studying forces your brain to participate. Instead of absorbing information, you’re required to retrieve it, manipulate it, explain it, or apply it in a meaningful way.

When you study actively, you’re constantly asking yourself questions: Can I explain this without looking? Can I reconstruct this mechanism from memory? Can I apply this concept to a new clinical scenario?

This kind of studying is slower and more uncomfortable, but it’s also far more effective. It strengthens neural pathways by forcing your brain to do the exact kind of work it will need to do on exam day. Active studying isn’t about being perfect. It’s about exposing what you don’t know so your brain can fix it.

Why Engaging More Senses Makes Learning Stick

One of the biggest advantages of active studying is that it naturally engages more senses. Learning becomes more durable when information is encoded through multiple pathways.

Reading alone uses primarily visual input. When you add speaking, writing, and drawing, you create multiple access points to the same information. Explaining a concept out loud forces you to organize your thoughts and exposes where you run into roadblocks. Writing mechanisms from memory forces precision. Drawing pathways forces you to understand relationships instead of memorizing lists.

Each additional sensory input strengthens the memory trace. When exam day comes, your brain has more ways to retrieve the information because it wasn’t stored in just one format. This is why teaching is so powerful. If you can explain something clearly without notes, even to an imaginary audience, you almost certainly understand it well enough to apply it under pressure.

The Discomfort That Signals Real Learning

One reason students avoid active studying is that it feels bad. It exposes gaps in knowledge, slows you down, and makes you confront what you don’t know. Passive studying, by contrast, feels smooth and reassuring.

That discomfort is not a sign of failure. It’s a sign that your brain is actually learning. If studying feels effortless, chances are you’re not pushing your brain hard enough to grow.

How to Shift From Passive to Active Without Studying More Hours

You don’t need to overhaul your entire study system overnight. Small shifts in how you interact with material can dramatically change outcomes.

Instead of rereading a topic multiple times, try reading once and then closing the book. Write down everything you remember, even if it feels incomplete. Then check your gaps and repeat. This single change transforms reading into retrieval practice.

Videos can also become active tools. Pause frequently and predict what will come next. After finishing, summarize the content out loud without notes. If you can’t explain it, rewatch with intention rather than passively letting it play.

Flashcards only work when they force recall. The real learning happens before you flip the card, not after. Saying answers out loud and explaining why they’re correct makes a massive difference. And perhaps most importantly, practice questions should be used as a learning tool, not a judgment of your intelligence. Getting questions wrong is one of the fastest ways to learn if you take the time to understand why each answer choice is right or wrong.

Why Active Studying Actually Saves Time

At first, active studying feels inefficient. You cover less material per hour, use more effort, and progress feels slower. But passive studying requires far more effort and time in the longrun because information never sticks the first time.

Active studying improves first-pass retention. You may move more slowly through content, but you’ll spend far less time relearning it later. Over weeks and months, the foundation you build helps you learn new material even faster. Hence, it gets more efficient with time and leads to better scores, less cramming, and significantly less stress. A win, win, win!

The Shift That Changes Everything

If you’re feeling stuck, the most important question to ask yourself is simple: Am I recognizing information, or am I retrieving it? Medical school rewards retrieval, application, and reasoning. Passive studying doesn’t train those skills, no matter how many hours you put in. The moment you shift from consuming information to actively engaging with it, using your eyes, hands, voice, and reasoning together, is the moment studying finally starts working the way you hoped it would.

You don’t need to be smarter.
You don’t need more resources.
You don’t need longer days.

You just need to stop letting information passively wash over you and start making your brain do the work it was designed to do. That’s when improvement stops feeling out of reach, and starts feeling inevitable.

The post Why Passive Studying Is the #1 Reason Medical Students Don’t Improve appeared first on Elite Medical Prep.

Spatial Single‑Cell Platform Reveals Barriers to Antibody Delivery in Solid Tumors

Edusehat — Wed, 03 Jun 2026 19:10:10 +0700

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

Edusehat — Wed, 03 Jun 2026 19:10:09 +0700

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.

Physician career satisfaction: How do hospitalists compare?

Edusehat — Wed, 03 Jun 2026 19:00:07 +0700

When it comes to career satisfaction for physicians, how do hospitalists compare to other specialties on factors like burnout, feeling valued and plans to leave their job?

A new report from the AMA found that hospitalists posted slightly better scores in some categories and slightly worse in others. And perhaps not surprisingly, hospitalists posted the worst numbers in the category of hours worked per week.

For medicine overall, physicians reported improved scores on most measures since 2024, with job stress dropping 2.2% and burnout decreasing 1.3%.The number of physicians who felt valued by their organization jumped 1.7% since 2024, and their intent to leave their current practice dropped 0.8%.

Here’s a look at the data from the AMA’s latest Organization Biopsy report.

Job satisfaction: “Are you satisfied with your current job?”

All physicians: 77%.

Hospitalists: 76.3%.

Satisfaction rates were lowest for emergency physicians (71.3%) and highest for ob/gyns (80.8%) and pediatricians (80.0%).
The numbers were slightly higher for male physicians (78.6%) than female physicians (77.1%).
Scores were higher for part-time physicians (78.1%) than full-timers (76.0%).
Among all physicians, satisfaction rates were lowest for physicians 11-15 years out of training (73.9%) and highest for physicians with 20-plus years of experience (79.6%).
By race/ethnicity, the numbers were lowest for white physicians (78.0%) and Middle East/North African physicians (77.9%). Satisfaction was highest for Asian doctors (82.0%), Latin/Hispanic physicians (81.6%) and Black doctors (80.3%).

Job-related stress: “I feel a great deal of stress because of my job”

All physicians: 42.9%.

Hospitalists: 40.5%.

Hospitalists reported the lowest level of stress.
Emergency medicine reported the highest levels of job-related stress (52.5%); pediatricians reported the lowest (40.9%).
Female physicians reported higher levels of stress (46.9%) than male physicians (38.6%).
Stress was lower for part-time physicians (39.2%) than full-timers (44.4%).
Stress levels were higher for physicians six to 10 years out training (47.9%) and lowest for physicians with 20-plus years of experience (38.3%).

Burnout: “Experiencing at least one symptom of burnout.”

All physicians: 41.9%

Hospitalists: 38.6%

Hospitalists reported the lowest level of burnout. The next lowest specialty was pediatrics (39.7%) and internal medicine/primary care (40.9%).
The highest rate of burnout was reported by emergency medicine (49.8%) and ob/gyn (45.7%).
Burnout was higher among female physicians (46.0%) than male physicians (37.0%).
Among all physicians five years out of training, 40.8% reported burnout. That number climbed into the high 40% range until physicians had 20-plus years of experience, when burnout levels dropped to 35.8%.

Time: “Average hours on work per week.”

All physicians: 58.4 hours.

Hospitalists: 65.7 hours.

Hospitalists reported the highest number of hours worked per week.
The number was lowest for emergency medicine physicians (51.0 hours).
The reported number of work hours was similar for male physicians (58.1 hours) and female physicians (57.7 hours).

Intent to leave: “Likely to leave current practice within next two years.”

All physicians: 31.1%

Hospitalists: 29.3%

Intent to leave their current practice was highest among internists/PCPs (31.9%) and emergency physicians (31.4%). It was lowest for pediatricians (26.0%).
White physicians (30.8%) were more likely to report intending to leave their practice. The number was lowest among black physicians (24.4%).

Feeling valued: “I feel valued by my organization.”

All physicians: 56.2%.

Hospitalists: 53.2%.

Feelings of valued were lowest among internists/PCPs (52.1%) and emergency physicians (52.7%). The number was highest for family medicine and ob/gyn (both 56.9%).
Physicians six to 10 years out of training were most likely to report feeling least valued (51.2%). Among physicians with 20-plus years of experience, the number was 59.4%.
Feelings of value were higher for male physicians (59.6%) than females (53.3%).

The post Physician career satisfaction: How do hospitalists compare? appeared first on Today's Hospitalist.

Disease Detection Gets Boost from Keck’s New Brain Reference Map

Edusehat — Wed, 03 Jun 2026 04:40:49 +0700

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]

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

“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

Edusehat — Wed, 03 Jun 2026 04:40:48 +0700

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.”

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Cross-Reactive T Cells Could Point to Broad Vaccines or Treatments for Measles, Nipah Virus

Edusehat — Wed, 03 Jun 2026 04:40:46 +0700

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, 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, 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.”

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Kinase Droplets Activate Growth Signals, Path for Cancer Therapy

Edusehat — Wed, 03 Jun 2026 00:50:09 +0700

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.

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Bio-IT World Keynote Highlights Collaborative Intelligence in AI-Driven Drug Discovery

Edusehat — Tue, 02 Jun 2026 21:10:12 +0700

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]

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.

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]

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]

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]

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]

“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.

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Circio and GenAssist Collaborate on Gene Therapy for Muscle Disease and In Vivo Cell Therapy

Edusehat — Tue, 02 Jun 2026 21:10:11 +0700

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.”

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Low-Cost, Portable Biotech Tools Improve Access to Bioresearch and Diagnostics

Edusehat — Tue, 02 Jun 2026 21:10:10 +0700

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.”

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DeepHealth achieves multiple regulatory milestones for Neuro, Prostate and LumbarMR

Edusehat — Tue, 02 Jun 2026 17:30:05 +0700

DeepHealth achieves multiple regulatory milestones for Neuro, Prostate and LumbarMR

DeepHealth, 02 June 26/ in E-News

DeepHealth has received CE Marking for the Brain Health and Brain Age solutions within its Neuro Suite, CE Marking for its LumbarMR solution, and FDA 510(k) clearance and CE Marking for the Prostate AI solution within its Prostate Suite.

“At DeepHealth, we believe we are delivering the world’s most comprehensive portfolio of native clinical AI solutions and services in radiology, with the aim of creating a new standard of AI-powered care. These regulatory clearances reflect the evolution and expansion of the DeepHealth portfolio bringing to market new clinical AI solutions that combine and advance capabilities through organic and inorganic innovation. The solutions are now commercially available, enabling expanded access to our leading-edge technologies designed to stage shift disease and enhance diagnostic consistency while driving meaningful gains in operational efficiency,” said Niccolo Stefani, MD, Business and Product Leader, Population Health & Clinical AI, DeepHealth.

CE Mark for Brain Health and Brain Age

Part of Neuro Suite,^[1] Brain Health and Brain Age are AI-powered solutions that automate and standardize neuroimaging analysis, enabling proactive care through normative data comparison and longitudinal tracking.

Brain Health introduces a new, fully automatic algorithm for White Matter Hyperintensity (WMH) detection and segmentation on FLAIR and removing the need for manual intervention.

Brain Age is a next-generation native clinical AI solution expanding the range of segmented brain structures, including hippocampus, lobes, and subcortical regions, delivering improved measurement performance for memory and dementia workups. The solution is showing 92% sensitivity in identifying hippocampal atrophy specific to Alzheimer’s disease, even in preclinical stages.^[2]

Already FDA 510(k) cleared, both solutions are being deployed and scaling across outpatient imaging sites of RadNet – a leading national provider of freestanding, fixed-site diagnostic imaging services in the United States.

“We are already seeing significant value in implementing DeepHealth’s Neuro Suite in our daily workflow. The combination of AI-assisted detection and classification with structured reporting is helping us improve efficiency, consistency and diagnostic confidence. I look forward to expanding its use across our practices to further enhance workflow efficiency and high-quality patient care,”said Kevin L. Berger, MD, Diagnostic Radiologist.

CE Mark for LumbarMR^[3]

The LumbarMR solution is one of the newest additions to the DeepHealth clinical AI portfolio, resulting from our acquisition of Gleamer SAS. LumbarMR is an AI solution for lumbar spine MRI that supports radiologists in assessing low back pain by automatically detecting, characterizing and reporting on key lumbar pathology findings. The technology achieves up to 94% sensitivity and delivers an average reduction in reporting time of 17% per exam when compared to a resident working without AI assistance.^[4]

FDA 510(k) Clearance and CE Mark for Prostate AI

The Prostate AI solution is part of DeepHealth’s Prostate Suite,^[5] which brings a fully integrated approach to AI-supported prostate cancer detection and interpretation. The solution delivers an end-to-end prostate MRI workflow on a new technology platform, from automated lesion detection and risk classification, intelligent gland segmentation with PSA density calculation and PI-RADS-compliant reporting into a single platform compatible with 11 fusion biopsy systems, all of which support radiologists’ clinical evaluations.

Real-world deployment of Prostate AI detected 27% more lesions,^[6] reduced inter-radiologist segmentation variability by 65%^[6] and reduced workflow time by 37% for biopsy-recommended cases.^[7]

The CE-Marked Prostate Suite solution including Prostate AI is currently part of the TRANSFORM program, the U.K.’s largest prostate screening trial in a generation, which is expected to analyze more than 100,000 scans over the course of the study.

“One of AI’s most significant contributions in prostate cancer detection is expanding access to prostate screening. Solutions like DeepHealth Prostate Suite support radiologists throughout the reporting workflow, minimizing delays and helping ensure more men receive timely, accurate care,” said Pr. Francesco Giganti, Associate Professor of Radiology, University College London.

References

[1] Neuro Suite comprises multiple applications, including Brain Health, Brain Age and DeepHealth Viewer. DeepHealth Viewer is manufactured by eRAD, Inc. and distributed by DeepHealth. Any claims made about Neuro Suite may reference claims associated with its individual components. Not all products and functions are available in all markets.

[2] Preliminary data from the observational study conducted at ASL Roma 2 on 226 geriatric patients starting from February 2023 to date, to investigate early diagnosis of dementia with the use of neuropsychological tests and AI-powered (DeepHealth ND) MRI automated evaluation of brain volumetry.

[3] LumbarMR is manufactured by Gleamer SAS.

[4] Data on file. Metrics are based on the results from an internal study conducted by Gleamer SAS.

[5] DeepHealth Prostate Suite comprises multiple applications, including Quantib Prostate, DeepHealth Prostate AI and DeepHealth Viewer. Quantib Prostate is manufactured by Quantib BV and distributed by DeepHealth, Inc, in the U.S. DeepHealth Viewer is manufactured by eRAD, Inc. and distributed by DeepHealth, Inc. Any claims made about Prostate Suite may reference claims associated with its individual components.

[6] DeepHealth pivotal study for FDA 510(k) clearance of Prostate AI, 12-reader by 250-case MRMC study and standalone testing on the same 250 exams. Data on File.

[7] Guenzel et al. “Diagnostic Utility of Artificial Intelligence–assisted Transperineal Biopsy Planning in Prostate Cancer Suspected Men: A Prospective Cohort Study.” Euro Urology Focus. Sep 2024.

Attacking Gout: Crystalys Sees Room for Its Dotinurad and Other Allopurinol Alternatives

Edusehat — Tue, 02 Jun 2026 06:40:11 +0700

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.

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Organ Chips Move Towards Mainstream Drug Development, with Hurdles Ahead

Edusehat — Tue, 02 Jun 2026 03:05:24 +0700

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 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, 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, 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 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, 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.”

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Targeted Protein Degradation Broadens Its Scope

Edusehat — Tue, 02 Jun 2026 03:05:22 +0700

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).

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, 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 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 Western^TM 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
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

Edusehat — Tue, 02 Jun 2026 03:05:20 +0700

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

Edusehat — Tue, 02 Jun 2026 03:05:18 +0700

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.

1. Boston/Cambridge, MA

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

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 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

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 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 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 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 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 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 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, 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).

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Turning the Patent Cliff into a Bioplant Opportunity

Edusehat — Tue, 02 Jun 2026 03:05:17 +0700

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
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.”

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Neuropixels Opto Integrates Electrophysiology and Optogenetics to Probe Neuronal Function

Edusehat — Tue, 02 Jun 2026 03:05:16 +0700

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.

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China has approved the world’s first invasive brain-computer chip—here’s what’s next

Edusehat — Mon, 01 Jun 2026 23:35:02 +0700

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

Edusehat — Mon, 01 Jun 2026 23:30:20 +0700

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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

Edusehat — Mon, 01 Jun 2026 23:30:19 +0700

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.

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Macrocyclic Peptide Drugs Unlocked, Membrane Permeability Screened at Scale

Edusehat — Mon, 01 Jun 2026 23:30:18 +0700

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

Edusehat — Mon, 01 Jun 2026 23:30:15 +0700

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 (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 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.

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Novel Therapeutic Modalities Target the Undruggable

Edusehat — Mon, 01 Jun 2026 23:30:14 +0700

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.

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 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.

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The Confidence Gap: Why Drug Discovery’s Data Explosion Hasn’t Solved Its Billion-Dollar Decision Problem

Edusehat — Mon, 01 Jun 2026 23:30:13 +0700

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.

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Next Generation Biopharma Innovation

Edusehat — Mon, 01 Jun 2026 23:30:12 +0700

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).

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.

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 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 (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

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.
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.

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State of the Diagnostic Industry: Recombinants on the Rise

Edusehat — Mon, 01 Jun 2026 23:30:11 +0700

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
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.

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.

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House hearing to examine bill balancing data privacy and R&D needs

Edusehat — Mon, 01 Jun 2026 20:00:03 +0700

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.

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Measuring Direct, Bystander, and Off-Target ADC Killing with the HiBiT TCK Platform

Edusehat — Mon, 01 Jun 2026 19:55:22 +0700

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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. 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.

Learn more www.promega.com.

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ProPure™ Endotoxin-Free Proteins for Reliable Cancer Research

Edusehat — Mon, 01 Jun 2026 19:55:21 +0700

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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 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.

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Spatial Atlasing: Why Sensitivity Is the Real Frontier

Edusehat — Mon, 01 Jun 2026 19:55:20 +0700

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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.

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.

Learn more vizgen.com.

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