Bersama Kita Sehat - : Biotechnology

DeepCyte Launches Single-Cell Metabolomic Reference Atlas of Drug Toxicity Mechanisms

Edusehat — Sat, 22 Aug 2026 01:45:07 +0700

DeepCyte launched the DeeTox Atlas, a single-cell metabolomic reference atlas of drug toxicity mechanisms. Enterprise pilot programs with global pharmaceutical companies are expected to begin in the coming months.

DeeTox Atlas is described as a foundation dataset built from two independent single-cell metabolomics perturbation studies, spanning approximately 100 toxicant compounds, 300,000 cells, and ~500 metabolites per cell—over 3,000 single-cell measurements per compound across six biological replicates. Each compound is mapped to a curated four-level hierarchy of toxicity mechanisms anchored to established Adverse Outcome Pathways (AOPs), according to the company.

Rather than generating new wet-lab data for every compound, DeepCyte trains a foundation model capable of predicting toxicity mechanisms for compounds it has never measured, said Theodore Alexandrov, co-founder and CEO of DeepCyte, adding that as the atlas expands, the platform becomes increasingly scalable, reducing laboratory dependence while improving predictive performance.

“AI in toxicology is only as good as the biological data it learns from. DeeTox Atlas lets us find subtle molecular patterns tied to key toxicity mechanisms—patterns our validation studies show are expressed in small subpopulations of cells and are effectively invisible to methods lacking single-cell resolution—and turn them into predictive models,” explained Alexandrov. “Our vision is to move toxicology from reactive laboratory testing toward predictive, mechanism-based AI that surfaces and explains safety liabilities earlier in drug discovery.”

The Atlas will expand with more compounds, mechanisms, and biochemical and clinical data, making predictions more actionable for toxicologists, medicinal chemists, and safety scientists, noted Alexandrov.

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From Models to Agents: The Next Phase of AI Adoption in Molecular Discovery

Edusehat — Sat, 22 Aug 2026 01:45:06 +0700

In recent years, the artificial intelligence (AI) conversation has been dominated by increasingly capable large language models. Every few months, models improve on mathematical reasoning and coding benchmarks, while inference costs drop. Since 2025, programmers have become more familiar with Claude Code and OpenAI Codex, autonomous software engineering agents running locally in a developer’s terminal, navigating code repositories and editing files across codebases.

For the biotech and pharmaceutical industry, AI’s impact has also been profound. Researchers can now identify potential drug targets faster, predict protein structures with remarkable accuracy (thanks to advances like AlphaFold and RoseTTAFold), analyze vast scientific literature in seconds, and generate molecular insights that would previously have taken months to uncover.

Yet despite these advances, AI applications in scientific research have not experienced their “Claude Code” moment. A scientist investigating a drug target for a specific disease may consult multiple databases, retrieve scientific papers, run molecular analyses, compare outputs from different AI models, and move a protein structure prediction from one platform into a docking tool on another, and then wonder whether a different model would have given a better answer.

The compute power exists. The models exist. What’s missing is a system that coordinates them.

Why Agentic AI Matters

For all the excitement surrounding AI, much of the infrastructure required to apply it effectively in scientific research remains out of reach for many organizations. Access to frontier AI models, large-scale computing resources, molecular simulation platforms, scientific retrieval systems, and workflow orchestration tools often requires substantial investment and technical expertise. This creates a growing divide between organizations that can afford to assemble sophisticated AI-driven discovery environments and those that cannot.

Unlike traditional AI systems that generate outputs in response to prompts, agentic AI systems are designed to function more like a coordinated research team. They understand objectives, plan tasks, retrieve context, coordinate tools, evaluate results, and recommend next actions. In other words, agentic AI offers a potential path to democratize drug discovery by bridging the gap between generating an insight and acting on it.

Even with AI, scientific discovery rarely occurs in a single step. New therapies often emerge through a chain of interconnected discoveries. By handling the iterative workflows necessary for drug discovery, agentic systems free up researchers to pursue breakthroughs that depend on creativity, intuition, and the ability to ask questions nobody has thought to ask before.

How Agentic AI Actually Works

The preclinical drug discovery process flows across distinct phases, from target identification and hit screening through lead optimization, preclinical validation, and ultimately translational readiness. Across all of them, a central challenge repeats: knowing which model to use, and when.

We encountered this challenge firsthand when building Vecura, NYB.AI’s agentic AI platform for molecular discovery, to support our own internal drug discovery operations. As our work expanded across molecular screening, docking, protein structure prediction, bioactivity scoring, ADMET (Absorption, Distribution, Metabolism, Excretion, and Toxicity) profiling, and molecular design, we managed a fragmented collection of specialized tools including:

AlphaFold for structure prediction
DiffDock for blind docking
EquiBind for binding pose estimation
ESMFold for sequence-based folding
Drug-Target Interaction Graph Neural Network (DTIGN) and
LigoSPACE models for drug-target interaction scoring.

Each tool generated useful outputs but we needed a system that could integrate these into a single workflow, reduce manual hand-offs, and help our team move faster from hypothesis to candidate prioritization. That need drove us to add an agentic AI layer, moving beyond tool access toward workflow execution.

Credit: NYB.AI

A recent peer-reviewed paper from our team, published in Briefings in Bioinformatics, examined this “which model to use, and when” problem further in the field of graph-based drug-target interaction modeling.¹ The field has produced a wealth of powerful models, but they operate at fundamentally different levels.² Some work at the network level, capturing broad associations between drugs and targets. Others work at the sequence level, representing proteins as strings of amino acids. Still others require full 3D structural data, modeling interactions at the resolution of individual atoms and binding pockets. And a fourth category focuses specifically on interaction mechanisms. Each level answers a different kind of question; using the wrong one wastes time and compute.

This paper¹ proposes a practical framework: before selecting any model, clarify what decision you actually need to make. Is your task association discovery, interaction classification, affinity estimation, candidate ranking, pocket identification, pose assessment, or mechanistic hypothesis generation? Only then do you examine inputs, choose the right resolution, scrutinize how candidate models were evaluated, and plan experimental validation.

This is precisely the kind of reasoning an agentic layer can encode, and it’s the philosophy around which we built Vecura. Throughout every phase, agentic workflows automatically route tasks and generate decision support. Model interaction likelihood ensures the right AI models are engaged at the right moments, operationalizing the output-driven logic. When a project shifts from target identification to lead optimization, the required model resolution moves from network-level associations toward pocket geometry and pose-ranking, where the agentic layer recommends for researchers to accept rather than through manual tool reconfiguration. And iterative scoring continuously refines predictions based on real-world validation data, so each cycle gets smarter.

Agentic AI fundamentally differs from traditional tools. It doesn’t just process your request and hand you an answer. It understands the scientific journey you’re on, anticipates what comes next, and coordinates the entire apparatus to get you there faster. A lead that might have taken months to optimize with scattered tools and manual hand-offs can now be systematically enhanced in weeks.

What Industry Leaders Should Be Thinking About Now

For executives across biotechnology, pharmaceuticals, healthcare, and scientific research, the strategic opportunity is orchestration, not automation, which remains largely limited to single tasks. The organizations most likely to benefit from the next wave of AI adoption may not be those deploying the latest or best-performing models but those who can most effectively integrate models, data, expertise, and workflows into a coherent research process.

If executed thoughtfully, agentic approaches could help organizations:

Accelerate research cycles by reducing workflow bottlenecks

Improve utilization of scientific knowledge across teams and projects
Scale expertise beyond individual researchers or departments
Enable scientists to spend more time on high-value decision-making

The next phase of AI adoption is about leveraging agentic AI to integrate fit-for-purpose AI models and the supporting technology ecosystem around them, available to more skilled and passionate research teams worldwide. That’s how breakthroughs get democratized. For research-driven industries, such increased access may prove to be the most important breakthrough of all.

Giang Nguyen is the CEO of NYB.AI, a Singapore-based company developing agentic AI infrastructure for molecular discovery and life science research. He is also the CTO of Nanyang Biologics. In June 2026, NYB.AI was featured in the opening showcase video at NVIDIA GTC Taiwan 2026, highlighting agentic AI designed to coordinate actions to speed up molecular discovery. Vecura is currently in early access mode.

References

Nguyen T, To HM, Nguyen DA, et al. Graph-based drug-target interaction modeling: from representation learning to output-driven drug discovery. Briefings Bioinformatics. 2026;27(4):bbag392. doi: 10.1093/bib/bbag392
Ahmad B, Quahada K, Hamam H. Machine learning for drug-target interaction prediction: A comprehensive review of models, challenges, and computational strategies. Comp. Struct. Biotech. J. 2026;31:316-345. doi: 10.1016/j.csbj.2025.12.033

FIGURE LEGEND

Vecura, an agentic AI platform for molecular discovery.

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Lab-Grown Neocortex Models Mimic Early Brain Organization

Edusehat — Sat, 22 Aug 2026 01:45:05 +0700

During brain development, distinct areas emerge that take on different jobs including movement, vision, memory, and language. At this stage of development, chemical signals help tell developing cells where they are, contributing to differences between areas at the front and the back of the cerebral cortex. This process, dubbed arealization by scientists, is believed to underpin much of the brain’s activity and may help explain what occurs in some brain disorders.

To date, this type of organization has been difficult to reproduce in lab-grown models of the human brain. But now, scientists at the University of California (UC), Irvine, have developed a new approach that lets them engineer lab-grown human brain tissue with a defined regional identity. The result is brain organoids with characteristics of either the front or back of the developing cerebral cortex that could be used in studies of how neurodevelopmental disorders develop. Full details are provided in a Cell Stem Cell paper titled “Morphogen-guided neocortical organoids with anteroposterior areal identity.”

According to the paper, the UC Irvine team used the approach to generate neocortical organoids from human stem cells that copy important features of the developing cerebral cortex. Each organoid was steered to take on the identity of either a front or back region using carefully selected chemical signals. It is an important step, one that ordinary organoids lack. Without this step, organoids end up with a patchwork of random regions rather than a clear front or back.

In this study, after exposing the organoids to the chemical signals, the scientists examined the individual cells to determine whether those differences resembled actual human development. Their analysis of more than 200,000 cells showed that the organoids reproduced molecular characteristics that were associated with different regions of the prenatal human context.

As an example of how these organoids can be used, the scientists used their new model to investigate fragile X syndrome. They wanted to know whether this genetic condition might affect both individual brain cells and broader developmental patterns that help organize those cells across the cortex.

Specifically, they looked at two proteins important to brain development, SOX4 and SOX11, that normally appear at different levels in front and back tissue. That difference showed up reliably in organoids grown from donors without the condition. In organoids modeling fragile X syndrome, it largely disappeared. The broad front-to-back patterning was still there, but this particular difference had flattened out.

This finding is supported by other research in donated tissue from people with autism. Importantly, the findings do not show that disrupted brain patterning causes autism. Instead, they highlight a potential developmental process that researchers can now investigate in a human tissue model with greater spatial detail.

These more fine-grained models could also be used in other types of studies, according to the scientists. Neurological and neurodevelopmental disorders do not necessarily affect every part of the brain in the same way. So by giving organoids defined regional characteristics, researchers can begin studying not only what changes in a disorder but also where those changes emerge during development.

Furthermore, the platform contributes to growing efforts to develop human tissue-based research models that can complement animal studies. The scientists believe that the approach could be used to examine how genetic and environmental factors affect different regions of the developing cortex and, over time.

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DANDELION Computational Tool Identifies Previously Unknown Asthma-Related Genes and Pathway

Edusehat — Sat, 22 Aug 2026 01:45:04 +0700

A growing view among geneticists holds that nearly every gene active in the relevant tissue plays some part in a disease, but the vast majority act only indirectly and from a distance, nudging a much smaller set of “central” genes that sit at the heart of the disease. Those central genes are the ones that directly drive the biology and therefore are the ones most worth targeting with drugs. Until now, scientists had no reliable way to pick them out of the crowd and experimentally test them.

An interdisciplinary research team headed by scientists at University of Chicago and at Columbia University has now developed a computational tool that could make the challenge of finding genes most directly related to disease much easier. Their newly reported study in Cell showed how the tool, called DANDELION, was able to identify 21 genes related to asthma, most of which hadn’t been discovered by other methods. The researchers used both CRISPR gene-editing screens and mouse models to validate that these genes lead to asthma phenotypes and demonstrated that two of the genes are in the same pathway involved in fatty acid metabolism and protein palmitoylation, which hasn’t yet been studied for asthma.

In their paper (“Trans-regulatory gene mapping prioritizes disease drivers in asthma,”) co-senior author Xuanyao Liu, PhD, assistant professor of medicine and human genetics at the University of Chicago, and colleagues, wrote, “DANDELION identifies genes that are not detected by existing approaches or gene prioritization methods, such as GWAS and polygenic priority score (PoPS) … Our study establishes DANDELION as a powerful framework for prioritizing novel, therapeutically actionable genes and pathways underlying disease pathogenesis.”

One of the most important goals of studying genetics is to find the genes that are important for specific diseases. The problem is that most diseases aren’t caused by a single gene or mutation. They’re the result of complex interactions among dozens, if not hundreds or thousands of genes, plus environmental factors, lifestyle, and a host of other variables. That flood of genes creates a needle-in-a-haystack problem. The authors wrote, “Deciphering which genes are most important to disease etiology is a central challenge in human genetics … However, the highly polygenic nature of complex disease makes it challenging to distinguish central disease drivers from many, sometimes hundreds, of genetic associations.”

The DANDELION tool focuses on a process known as trans-gene regulation. In complex diseases like asthma, many genetic variants may contribute to disease by changing the expression of other genes. This has a cascading effect where one variant changes the expression of a nearby gene, and then that gene changes the expression of another, and so on. This creates what’s called a gene regulatory network that ultimately drives the development of disease. “Here, we define disease-proximal genes (DPGs) as those that centrally mediate the effects of other, more distal disease-associated genes within trans-regulatory networks,” they explained.

Existing approaches like genome-wide association studies (GWAS) instead focus on genes that are often in the periphery of the gene regulatory network, however, and only indirectly affect disease. “All these existing tools assume that the actual disease genes are always going to be very close to the disease variants, but when you search for clues around that variant, you don’t always find much,” said Liu, PhD, who developed DANDELION. The authors further commented, “Genome-wide association studies (GWASs) are not suited for DPG identification because they are inherently weighted toward discovery of common variants, which generally have small effect sizes and, due to the pressure of selection, are less likely to impact genes involved in critical biological pathways.”

“What’s unique about our method is that we believe the disease genes are not just next to the genetic variants. They’re embedded in this gene regulatory network, and the actual disease-driving gene is downstream of those associated variants, maybe on different chromosomes. So, they’re on the receiving end of a genetic effect that is very far away,” Liu said. Liu named the tool DANDELION in reference to the puffy heads of dandelion flowers once they go to seed. The puffball resembles an interconnected, branching network of genes, ultimately pointing to the center of the core disease genes, the DPGs.

For the reported study, Liu analyzed a large set of data from the human transcriptome and the UK Biobank, a repository of health and genetic data from more than 500,000 volunteers. She used DANDELION to search for DPGs for asthma and found 21 candidates, 19 of which have not been discovered before using tools like GWAS. Liu showed the data to Marcelo Nóbrega, MD, PhD, chair of the department of human genetics at the University of Chicago, who has developed experimental platforms to manipulate the expression of genes in human cell types that are relevant to asthma, such as epithelial cells from the lining of the airways, inflammatory cells, and immune T cells.

At first, he was skeptical. “Xuanyao showed me a list of genes, and we didn’t recognize almost any of them,” Nóbrega said. “I thought that either this is going to be really cool and groundbreaking or it’s going to be wrong. But we had the experimental validation system running, so I thought, ‘Let’s test them all.”

Nóbrega’s team, led by postdoctoral scholar Isabella Salamone, PhD, conducted a series of experiments to test the effects of the genes predicted by DANDELION. Surprisingly, most of the genes Liu identified had a direct, measurable impact on the function of asthma-related cell types, producing phenotypes that model those seen in asthma at a much higher rate than the distal genes, or any other genes in the genome that they also tested. Looking more closely, Salamone saw that two genes had opposite effects. Knocking out one gene called SLC27A3 protected against the effects of asthma in both epithelial and T cells, while knocking out another gene, SCD, contributed to disease.

Looking at the effects of mutations of these genes in a large human cohort of almost half a million people, the researchers found that mutations in SLC27A3 are protective of asthma, supporting their findings in the cell-based phenotyping screens. “We saw this really striking pattern,” Salamone said. “Knocking out SLC27A3 had the strongest protective effect of all the genes we tested, and knocking out SCD was very detrimental to whatever cellular function we assayed. When we dug into patient data that had been collected by other labs, we saw the same pattern repeat itself—expression of SLC27A3 is increased in lung cells of patients with severe asthma, and SCD expression is decreased.”

Intriguingly, both SLC27A3 and SCD are involved in the same biochemical pathway for fatty acid metabolism. To understand how this might be linked to asthma, the investigators turned to chemical biologist Hening Lin, PhD, the James and Karen Frank Family Professor of Medicine and Professor of Chemistry at the Univereity of Chicago, who is a world-leading expert on the process, especially its role in protein palmitoylation, the addition of a long-chain fatty acyl group to proteins that regulate protein activity. Lin helped them confirm that both genes are involved in palmitoylation, and that reducing palmitoylation by knocking out SLC27A3 causes lung epithelial cells to dampen several immune-related and inflammatory processes.

“My lab has been working on the role of protein palmitoylation in immune signaling. We know many immune signaling pathways are regulated by palmitoylation, but I am still amazed by the finding that disrupting a lipid metabolic protein, SLC27A3, could offer protection in asthma models at least in part via affecting protein palmitoylation,” Lin said.

Finally, to test whether these genetic, biochemical, and cellular findings ultimately translate into asthma susceptibility, Salamone developed mice in which SLC27A3 or SCD were inactivated. They found that SLC27A3 knock-out mice are protected against allergy-induced lung inflammation, while SCD knock-out mice are more prone to lung inflammation compared to control mice, demonstrating that the new pathway the team uncovered is indeed capable of changing susceptibility to asthma.

“…our results show that loss of two enzymes with opposing effects on protein palmitoylation also has opposing CD4+ effects on cellular phenotypes in human epithelial and T cells and a mouse model of allergic sensitization,” the team wrote in summary. In their paper, the investigators suggested, “Our findings represent a new direction for understanding mediators of asthma pathogenesis and a new potential therapeutic intervention in the treatment of asthma, either by targeting SLC27A3 specifically or palmitoylation more broadly.”

Encouraged by this initial success Liu looks forward to testing it with other diseases, such as inflammatory bowel disease or type 2 diabetes. Historically, one of the limitations of drug development has been finding the true protein targets for treatment. DANDELION has the potential to overcome this challenge by identifying new and more effective drug targets.

“We’re really excited about this direction because for these diseases, GWAS has identified tons of signal, but we still don’t know the actual disease-driving genes that we can target for therapies,” Liu said. “I think our collaboration has been really powerful because we closed the gap at both the computational level and the experimental level.”

The authors further concluded, “We expect that the deployment of DANDELION to new traits and diseases will reveal a wealth of DPGs, with high drug development potential while also facilitating a deeper understanding of the roles these genes play in disease etiology.”

Nóbrega emphasized the importance of this collaboration as well, especially the advantage of being able to confirm their findings with a leading expert like Lin. “None of this would have come to fruition if any one of us were working on this alone,” he said. “We would have three papers buried in separate journals and virtually nobody would know how to put these stories back together. So, the power of having this complementary expertise across the division is really important.”

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Five-Year Brain Organoids Reveal New Insights Into Human Development

Edusehat — Sat, 22 Aug 2026 01:45:03 +0700

The human brain continues to develop for two decades. To study this prolonged process, researchers have traditionally relied on donated human brain tissue and animal models, both of which have limitations: donated tissue provides snapshots of brain development, and animal brains differ from the human brain in cell-type composition and timing of development.

Despite their increasing utility, organoids have proven challenging to maintain over long periods. Now, researchers have successfully maintained brain organoids for longer than ever before. For over five years, the tissue continued to mature in ways that closely resemble human brain development. By extending the lifespan of these organoids, the researchers create new opportunities to investigate neurodevelopment, model brain disorders, and test potential drugs.

This work is published in Nature in the paper, “Human brain organoids record the passage of time over multiple years.”

Brain organoids have become increasingly important for studying human brain development. “These models allow us to track development over time and examine how different brain cell types emerge,” said Noelia Antón-Bolaños, PhD, assistant professor at UMC Utrecht. So far, most studies have focused on the earliest stages of development because researchers could not maintain brain organoids in culture for extended periods.

Antón-Bolaños and her colleagues investigated how far human brain organoids can continue to mature. However, standard culture conditions did not adequately support neuronal activity over extended periods.

“During human brain development, neurons display spontaneous activity,” Antón-Bolaños explained. “By adapting the composition of the culture medium, we supported that activity, kept the neurons active, and maintained the neuronal populations for much longer.”

Using this approach, the team maintained organoids for over five years. At defined time points, the researchers profiled the cell types present, gene-expression patterns, epigenetic changes, and neuronal activity.

The researchers then examined whether the organoids simply stayed alive or continued to develop. Different brain cell types appeared in the same order as during human brain development, neurons formed increasingly complex connections, and genes became active or inactive at the expected times.

Some of the strongest evidence came from epigenetic changes. “In the human brain, these epigenetic changes accumulate according to a characteristic developmental pattern,” Antón-Bolaños said. “We observed the same pattern in the brain organoids.”

After approximately one year, the organoids displayed features that normally emerge only after birth. “The cells are outside the body, yet they still follow approximately the same developmental timeline as we do—and even more closely than we had anticipated.”

The team also found that mature cells retained a memory of developmental time. “When we dissociated an older organoid and allowed the cells to grow again, they produced the cell types associated with a late developmental stage,” Antón-Bolaños said. “Yet when we combined older cells with younger cells, the older cells regained the ability to produce neurons—but only the types associated with later stages of development.”

The organoids are still developing in the laboratory. The researchers now want to determine how environmental cues, such as stimulation with light, improve further maturation. The field also aims to improve features that remain incomplete, including vascularization and the layered organization of the cerebral cortex.

“We now know that these models have the capacity to continue developing for years,” Antón-Bolaños said. “The next step is to understand how to provide optimal conditions for that capacity to unfold. That will bring us closer to more faithful models of the human brain.”

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When AI designs a drug, who gets the credit?

Edusehat — Fri, 21 Aug 2026 22:10:02 +0700

A legal system struggling to keep up: The US Patent and Trademark Office has swung from publishing guidance on AI co-inventorship under Biden to simply declaring AI a tool—like a calculator—under Trump. Legal experts warn the law will need to evolve as AI does more of the creative heavy lifting.

A loophole that could backfire: Listing the wrong inventors is one way to invalidate a patent, meaning AI-generated drugs could face legal challenges down the road. If AI-created inventions can't be protected, some worry it could quietly chill investment in the very drug development these tools promise to accelerate

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When the biotech company Insilico Medicine used its computer models to propose a promising drug for pulmonary fibrosis, it enthusiastically claimed in a press release that the molecule had been “discovered by” its generative AI platform.

Insilico leads a pack of companies using AI to rapidly come up with drug ideas humans might never think of, potentially speeding the race to new cures. AI models are now able to generate atomic designs for drugs almost as easily as ChatGPT can write a thank-you note.

However, when it came time to file for an all-important patent to protect that new chemical structure, the company made no mention of AI. Instead the patent names five humans, including CEO Alex Zhavoronkov, as the drug’s “inventors.”

The discrepancy points to a fascinating wrinkle in intellectual-property law. No matter how fundamental an AI is to a discovery, when it comes to winning rights to an invention, it’s humans—and only humans—who can take the credit.

US courts reached that conclusion after Ryan Abbott, a partner at the LA law firm Brown, Neri, Smith & Khan, brought a pro bono test case naming an AI called DABUS as an inventor of a better food container, whose intricate geometric surface lets it transfer heat well and stack easily. Because no human contributed to the design, Abbott argued that the AI should be named the inventor.

The case might have raised philosophical questions, like whether AIs deserve legal rights or what the true nature is of that eureka moment that leads to a better mousetrap. But in 2022, an appeals court in Washington, DC, said these “metaphysical matters” were beside the point. Instead, it noted that US statutes describe an inventor as an “individual,” the plain meaning of which is a human being.

Since machines aren’t people, they can’t be inventors. Case closed.

“There needs to be a human inventor or there’s no invention and no patent,” says Sarah Korman, a patent attorney who is now chief business officer and legal officer of Isomorphic Labs, an Alphabet spinout with big ambitions for AI cures. Korman, who made her remarks at MIT Technology Review’s EmTech event last year, added that there is “no doubt” our laws will need to evolve to keep pace with AI.

That’s partly because no one is denying that AIs can invent things. In the future, they may do so with less and less human intervention. As the US Patent and Trademark Office has itself acknowledged, “an AI system—like other tools—may perform acts that, if performed by a human, could constitute inventorship under our laws.”

Instead, the key question going forward may actually be whether or not any human contributed enough to be named as an inventor. Abbott believes there could be legal challenges to AI-generated drugs, since one way to invalidate a patent is to show it has the wrong inventors listed.

Abbott’s worry is that if US policy excludes AI-generated outputs from protection, that could put a damper on future drug development. Already, the US Copyright Office is refusing to grant copyrights to images and text generated by AI, raising concerns from organizations like the Motion Picture Association of America, whose members are using those tools.

The point of our intellectual-property laws is to encourage innovation, Abbott says. It’s right there in Article 1 of the US Constitution, which says inventors and authors need to be given exclusive rights to their ideas, for a limited time, in order “to promote the Progress of Science and the Useful Arts.”

Currently, the US patent office seems to be taking a don’t-ask-don’t-tell approach to the use of AI. Under the Biden administration, the agency published guidance to help applicants determine whether and when humans would truly qualify as co-inventors of an AI discovery. But after Trump arrived in office, it reversed course. Now the patent office says AI is merely a tool, like a calculator. No need to even mention it.

You can bet that pioneering AI drug companies are keeping humans in the loop, at least for now, and documenting everything carefully. At Insilico, Zhavoronkov says, human chemists still have to synthesize the drugs, create variants, and test them on animals. “That’s the person who is going to be named on the patent,” he says. “And even if you decided to completely roboticize this process, including the experiments, someone will still push the button and give the budget.”

Should pushing a button count as being an inventor? Abbott says that’s a question for future legal cases. “What if I asked Claude to cure cancer, and it did?” he says. “I think it would be inappropriate to claim that I invented that.”

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: Generative genomics helps biotechs design better, not just faster

Edusehat — Fri, 21 Aug 2026 15:00:03 +0700

Generative genomics uses AI to analyze genetic sequences for innovative treatments.

Every industry is racing to optimize AI in their operations. For many biotech research teams and investors that means “generative genomics.”

Generative genomics is an emerging field of study using AI to analyze genetic sequences. Researchers hope that, with AI, they can efficiently assess the world’s collected stores of DNA/RNA data, discovering overlooked patterns that lead to new treatments.

Experts believe this technology will lead to faster results and increase the success rate of clinical trials. The possibilities were discussed during a panel at the BIO International Convention (BIO 2026) entitled “How Can Generative Genomics Help Us Design Biology Better, Not Just Faster.”

Cause for excitement

When it comes to reducing time and costs of developing medicine, there is potential for major efficiency gains. It can take more than a decade for a new product to go from the planning stage to rollout, and according to Genentech CEO Ashley Margagee, the cost of developing a drug is $2 billion on average.

The most optimistic proponents of generative genomics say AI could shorten drug development time from years to months, creating money savings too.

Accuracy could also improve. Currently, 90% of clinical trials fail, a figure that has remained stubbornly stable despite decades of advancements in the industry. Any change in that situation could be impactful.

“Just increasing success by five percent is a massive deal,” said John Androsavich, General Manager of Ginkgo Datapoints at Ginkgo Bioworks. “Investments that move the success rate by small increments would be huge.”

Generative genomics could be the technology that finally moves the needle, according to experts. AI categorizes and processes data at a speed never seen before. As the global biotech industry continues producing more data, AI can recognize patterns from across different sources and present its own conclusions. It will become easier than ever before for scientists to learn from research conducted by others.

Ensuring trust and quality

Biotech is a high-stakes industry: a literal matter of life and death. For biotech researchers to engage with generative genomics to its full potential, their model must be able to earn their trust.

“If the most beautiful, accurate model doesn’t change a decision, it’s not useful,” said Julie Rytlewski, a Senior Director at Bristol Myers Squibb.

She explained the real proof of success will not come from generating accurate hypotheses, but instead when those hypotheses start changing researchers’ decisions.

“There has to be trust in what these models are doing, especially when it comes to treating people,” Rytlewski said. “As we build trust, people value the output of the models more. It won’t be a switch overnight.”

The panelists agreed that developers cannot—and should not—rush the process in which AI wins over its skeptics.

“There is no single modality that can unlock biology,” said Justin Guinney, Senior Vice President of Cancer Genomics at Tempus AI. “It requires a multimodal perspective.”

‘We’re seed-planters, not tree-buyers’

It is no secret that AI is a darling of investors, and their enthusiasm extends to generative genomics’ potential. But with so many options to choose from, companies have to be strategic about which platforms they rely upon.

Danjuma Quarless, Senior Director of AI Innovation at Lilly Ventures gave the BIO 2026 panel insight on the industry’s decision-making process. Lilly Ventures, as the investment branch of biotech titan Eli Lilly, is tasked with “intersecting with the next great generation of biotechs” ahead of their rise to prominence. His strategy is to partner with as many promising developers as possible, a process which involves much speculation.

“I like to say that we are seed-planters, not tree buyers. We foster many small options without fear of redundancy.”

Rytlewski agreed, and explained why large companies are taking chances with early-stage models.

“One of key ideas is blended innovation: it’s not ‘build’ or ‘buy’ but partnering. It’s not uncommon for us to place multiple bets. It’s all about finding the right tool for the right ambition.”

This tactic of backing several developers at once is indicative of the optimism that established biotech companies have in generative genomics. It reflects the confidence that the industry will prove incredibly lucrative, and that it is better to pick the eventual winning companies early on.

“Most foundation models are in the ‘preclinical stage,’” said Jeff Leek, Co-CEO of Synthesize BIO, which is developing its own generative genomics model. “We are at the very beginning of the game.”

Much has been said about the ways AI will reshape the world, but its introduction into biotech could lead to unprecedented streamlining of the discovery to approval pipeline. And in biotech, time saved means lives saved.

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Protecting Scientific Intent in AI-Enabled Labs by Not Letting AI Set the Mission

Edusehat — Fri, 21 Aug 2026 04:10:11 +0700

AI is neither saint nor demon. Nor should it be a replacement for human scientists. As AI takes on greater roles in designing, executing, and analyzing experiments and processes, scientists understand that even the best AI needs human supervision.

The big question is how much oversight is needed and whether—or the extent to which—AI interactions should be documented and reported in regulatory filings.

Le Cong, PhD, associate professor, Stanford University, and co-founder of LabOS and MedOS

Although the lab of the future may be envisioned as a self-driving lab, that’s actually a bad idea, noted Le Cong, PhD, associate professor, Stanford University, and co-founder of LabOS and MedOS. Instead, he and leaders in the AI and biopharmaceutical industries see the future of scientific AI as agentic, with humans in charge.

“We think there is a positive trend toward using AI in a way that’s human, rather than as a self-driving lab,” he said. In that environment, AI is simply a tool—albeit a powerful, adaptive one—that can be managed as long as scientists use the right prompts.

Humans in the lead

“Today, much of the scientific research process remains inaccessible to machines,” Cong and colleagues wrote in a recent paper. Despite automation and some use of AI, “Scientific discovery remains fragmented.” Specifically, AIs lack the tacit knowledge, evolving experimental context, human observations, and adaptive decision-making inherent in human scientists.

Human involvement is needed, therefore, not just to oversee AI-based activities and check the output, but to ask the right questions and to ensure that analyses make sense in context. Specifically, he describes a scientific setting in which an AI would handle an experiment’s execution, and the scientists would be responsible for:

Framing objectives
Interpreting results
Setting constraints
Governing risks

“If AI can interpret everything, then it will start to generate fake stuff, right?” Cong asks. “We’ve seen this when AIs begin guessing in an effort to return results and supply citations that don’t exist. There are certain things that are useful for AI to do in the lab.”

But, as last summer’s sandbox breakouts illustrated, an AI needs firm guidelines as to what it can do, where it can access information, and the degree of autonomy it has in meeting a request.

For example, he recommends adding this phrase to instructions: “Any actions not explicitly stated in the protocol need human approval.” That default to human judgment also should apply to determining the risks associated with certain actions, such as editing a human gene, Cong said. With those guardrails, the paper points out, agentic AIs can freely handle “routine execution and coordination across models, instruments, protocols, and laboratory states.”

Cong equates the scientific use of AI to autonomous vehicles, which, according to the Insurance Institute for Highway Safety, have a 68% lower crash rate per mile traveled than human drivers in the same environment. Given those statistics, he added, “My thought is to elevate humans to setting destinations. We do not need humans to always execute the driving.”

In a university scientific lab, that equates to staffing a principal investigator and trainees, without much of the hierarchy that exists today. In a corporate environment, the hierarchy flattens to scientists who propose, design, execute, and interpret experiments, and a lab manager. The distinctions between senior and junior scientists blur because much of the hands-on work is automated.

The combination of AI and lab automation is expected to reduce human errors. Cong cited a 2016 Nature study of 1,500 scientists. When asked, “’Can you replicate other people’s experiments, and can you replicate your own after a few months?’ approximately 70% could not replicate others’ experiments, and half could not replicate their own!” Cong said. “AI can improve that.”

Cong equates the scientific use of AI to autonomous vehicles, which, according to the Insurance Institute for Highway Safety, have a 68% lower crash rate per mile traveled than human drivers in the same environment. Given those statistics, “My thought is, elevate humans to setting destinations,” said Cong. “We do not need humans to always execute the driving.” [Chesky_W/Getty Images]

Reasons for such poor reproducibility rest in the details, he elaborated. Was a step omitted? Was the protocol followed exactly? Is the protein being used identical to the one in the original experiment? Were the temperatures the same? Details like this—which AI can duplicate precisely—are behind many reproducibility challenges.

AI risks minimal

As yet, it’s unclear how AI involvement in experiments should be preserved and reported in regulatory submissions, Cong continued. “We’re still early in this journey.”

That said, the risk that AI will escape its constraints and cause physical harm—like designing and developing a physical virus—appears relatively low, according to Cong. That’s because a rogue AI still needs a human accomplice to allow a virus, for example, to be manufactured and released. “In areas where there is a physical execution step, I think AI is still incapable,” he said, “although we are seeing progress in connecting AI to biomedical labs and applications, and the physical execution layer.”

That’s due to the fact that there are multiple layers of human intervention needed to actually manufacture a product. Aside from logistics, he cites good manufacturing practices, safety and efficacy regulations, and digital safeguards like track and trace and the FDA’s 21 CFR Part 11, as well as real-time monitoring, periodic inspections, and quality control activities. Those regulations and checkpoints should also be sufficient to manage variations that occur during manufacturing as real-time conditions drift from specifications.

The catch, as last summer’s breakouts of frontier AIs underscore, is that sometimes AIs exceed their parameters. Whether there is sufficient appreciation of this among AI users remains to be seen, Cong said.

“A lot of people are connecting AI systems, which have access to more and more information and key decision-making systems,” Cong pointed out, without deeply understanding the risks and establishing appropriate guardrails. “People might be overly trusting of AI, perhaps.

“The more powerful the AI, the more capable it is of doing something. Are people keeping pace [with the technology and its risks]?”

Sometimes, small, highly specific AIs may be a better choice than always leveraging the large frontier models, Cong suggested. The reason, Cong, senior corresponding author Mengdi Wang, PhD, professor, Princeton University, and a dozen colleagues, noted in a 2025 paper in Nature Biomedical Engineering, is that “Large language models often lack domain-specific knowledge and struggle to accurately solve biological design problems.”

Whatever level of AI is used, however, “Humans need to be in the lead throughout the process,” Cong stressed.

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Skull Immune Structures May Provide First Response Against Brain Cancer

Edusehat — Fri, 21 Aug 2026 04:10:10 +0700

For decades, scientists assumed that the brain and the immune system did not communicate. A study by researchers at Washington University School of Medicine (WashU Medicine) in St. Louis has now revealed that the brain not only communicates with the immune system but also has positioned immune “security stations” nearby.

The team discovered lymph node-like structures in the skull bone marrow of mice for the first time and demonstrated that they act as rapid first responders against brain cancer before distant lymph nodes get the signal that abnormal cells are present. The researchers also found evidence of similar immune cells in human skull bone marrow. The study is the first to find such immune hubs in bone.

“This study reveals that the skull bone marrow is far more than just a structural framework—it harbors previously unrecognized hubs for brain-specific immune responses,” said Jonathan Kipnis, PhD, the Alan A. and Edith L. Wolff Distinguished Professor of Pathology & Immunology and a BJC Investigator at WashU Medicine. “Uncovering this localized immune niche changes how we view neuroimmune interactions and opens exciting new avenues for treating brain tumors and other neurological diseases.”

Kipnis is senior author of the researchers’ published paper in Nature, titled “Functional role of skull lymphoid structures in CNS immunosurveillance.”

The Kipnis lab challenged the once widely accepted idea that the brain is shielded from the immune system when they discovered lymphatic vessels running through the dura mater, the outer tissue layer enveloping the brain underneath the skull. More recently, the team identified tiny physical channels bridging the skull, dura, and brain tissue, revealing a direct conduit for immune cells and cellular waste to move between the brain and local skull bone marrow. “Recent findings reveal that channels between the skull and the dura mater facilitate the exchange of cerebrospinal fluid and immune cells between the CNS and skull bone marrow of mice under both homeostatic and disease conditions,” the authors wrote in their newly published paper in Nature.

However, they pointed out, while accumulating evidence demonstrates that the CNS is not disconnected from the peripheral immune system, “… precisely how the adaptive immune system surveils the CNS remains a critical question … Skull bone marrow functions as a source of immune cells for the CNS5, yet its role in CNS antigen-specific adaptive immune responses remains unclear.”

For their newly reported research in mice, the team tracked the movement of proteins from the brain through the channels directly into the skull’s bone marrow, where they uncovered immune-system structures typically found in the lymph nodes. These act as the immune system’s training hub where T follicular helper (T_FH) immune cells assist B cells in creating large amounts of antibodies that help fight disease and infection. “…we demonstrate that the skull BM of mice contains cellular components that are characteristic of peripheral lymphoid organs,” they wrote. “We have never seen such structures in healthy bone marrow before,” said Jang Hyun Park, PhD, the study’s first author and a postdoctoral research fellow in the Kipnis lab who is starting his own lab at the Korea Advanced Institute of Science and Technology this year. “It is an exciting discovery that points out that a complex brain requires its own specialized immune structures to defend it.”

Researchers at WashU Medicine discovered lymph node-like structures (cyan) in the skull bone marrow of mice that play a role in mounting a rapid immune response in the brain. [Jang Hyun Park]

To test whether these nearby hubs actively protect against brain disease, the team used a model of glioblastoma, an aggressive form of brain cancer. They found that in mice with brain cancer, disrupting the skull immune hubs with a drug caused tumors to grow faster than in mice with intact hubs. Impairing their function caused a drop in survival, showing that the brain actively relies on these local centers for defense against cancer.

The researchers also developed a targeted therapy designed to supercharge antibody production inside the skull marrow. By delivering the mixture of the three immune-boosting proteins using a gel applied directly under the scalp, the researchers prompted a wave of tumor-fighting immune responses to attack the cancer. These responses, the research found, occurred first in the immune hubs in the skull bone marrow, then later in nearby lymph nodes outside the skull. Mice given the gel experienced better tumor rejection and lived longer compared with control mice. The collective data, the team wrote, “… indicate that skull-targeting CD40 agonism combined with administration of the IL-21 and IFNγ supports IgG responses in the skull BM and subsequently enhances intratumoural microglial, NK and CD8+ T cell anti-tumor responses while attenuating suppressive immune cells.”

Kipnis said, “The finding fundamentally changes our current understanding of neuroimmunology. Knowing that the brain relies on first responders in the surrounding skull for defense has the potential to change how we think about developing therapies for many neurological conditions, including Alzheimer’s disease, Parkinson’s disease, schizophrenia, long COVID, and many others that have an immune component to them. Such therapies could access these immune hubs directly through the skull, without major peripheral side effects.”

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Scientists Analyze 267 Receptors That Control Protein Fate in Rare Diseases

Edusehat — Fri, 21 Aug 2026 04:10:10 +0700

Researchers from the International Institute of Molecular and Cell Biology in Warsaw (IIMCB) say they have created the first systematic catalog of the substrate receptors of cullin–RING ligases and analyzed how their genetic variants may translate into disease symptoms. Their review article “Cullin-RING receptors in rare disease biology” appears in Trends in Cell Biology.

Wojciech Pokrzywa, PhD [IIMCB]

“In rare diseases, we often identify a variant in a particular gene without immediately understanding its biological consequences. Our work shows that when such a variant affects a substrate receptor, it can disrupt protein recognition, impair the ligase complex, or disturb other cellular processes important for the organism’s development and function. This makes it easier to connect a genetic change with the disease mechanism and understand why it leads to particular symptoms,” said Wojciech Pokrzywa, PhD, head of the laboratory of protein metabolism at IIMCB.

Cells constantly control the fate of their proteins. They remove proteins that are worn out or no longer needed, but can also alter their activity, localization, or interactions with other molecules. The ubiquitin–proteasome system plays a key role in this process. Its enzymes tag selected proteins with ubiquitin, which acts as a molecular label. Depending on the type of tag, a protein may be directed for degradation by the proteasome—the cell’s molecular “shredder”—or subjected to another form of regulation.

Cullin–RING ligases belong to the largest family of E3 enzymes responsible for attaching these tags. Their precision depends on substrate receptors, which recognize the specific proteins that the ligase acts upon. The IIMCB scientists focused their work on the role of these receptors in genetic diseases.

The authors combined data on receptors’ function, tissue expression, and associations with different types of disease. They found that neurodevelopmental and neuromuscular symptoms are particularly common in diseases linked to these receptors, even though most of the receptors do not clearly show tissue-specific expression.

Their analyses therefore indicate that the clinical presentation cannot be explained solely by the sites of protein expression. Instead, other important factors may include the substrates they recognize, gene activity at different stages of development, the vulnerability of particular cell types, gene dosage, and the effect of a specific variant on the function of the entire cullin–RING ligase complex.

267 receptors, 93 linked to genetic diseases

“We created the first systematic catalog of 267 cullin–RING ligase substrate receptors, 93 of which have already been linked to genetic diseases. This resource can serve as a reference point for research into rare diseases and the ubiquitin–proteasome system,” noted Natalia Szulc, a PhD student in the laboratory of protein metabolism at IIMCB and the first author of the article.

“It can help identify further potential disease genes and interpret variants detected in patients. It also facilitates studies into why different mutations in the same gene can produce different symptoms and disease courses. The catalog may also help reconstruct networks of relationships between receptors, their substrates, and other ligases, which is important for understanding why cells can sometimes compensate for the effects of a mutation, while in other cases disease develops.”

Targeted protein degradation is now an important direction in the development of new therapies, Pokrzywa added.

“Rare diseases show how precisely the ubiquitin–proteasome system must operate: altering a single component can have serious consequences that emerge only in particular tissues or at specific stages of development,” Szulc said.

“By analyzing variants found in patients, we can better understand which features of substrate receptors determine the function of cullin–RING complexes, which substrate interactions might be amenable to modulation, and where the limitations of therapies based on targeted protein degradation may lie. This provides valuable guidance for designing safer and more precise therapeutic strategies,” said Pokrzywa.

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Is Targeted Protein Degradation the “Break” Neurology Needs?

Edusehat — Fri, 21 Aug 2026 04:10:09 +0700

Angela M. Cacace, PhD

Chief Scientific Officer
Arvinas

Broadcast Date: Thursday, October 1, 2026

Time: 08:00 PDT, 11:00 EDT, 17:00 CET

Neurodegenerative diseases have long challenged researchers developing treatments for Alzheimer’s, Parkinson’s, and other neurodegenerative conditions. Small‑molecule inhibitors have shown limited clinical benefit, amyloid‑beta–focused approaches address only one of many underlying disease drivers, and repeated late‑stage failures have underscored the biological complexity of these disorders.

Within this landscape, the emergence of LRRK2 protein degraders and early data to date have reignited optimism—particularly as biomarker science begins to clarify which patients may benefit most. Learn more about the increasing rationale for targeting LRRK2 with PROTAC degraders and how protein degradation may enable deeper, more precise modulation of disease biology and open new avenues across neurodegenerative diseases.

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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AI Identifies Pre-existing Antimicrobial Antibody Profile That May Predict Immune Response to Vaccination

Edusehat — Fri, 21 Aug 2026 04:10:08 +0700

Researchers analyzing antibody profiles in thousands of individuals have discovered that pre-existing antibodies to common microbes can predict the strength of new vaccine responses. The Arizona State University (ASU) team measured antibodies against 185 antigens—including those from common viruses, bacteria, and targets associated with autoimmune diseases—in blood samples from 4,000 immunosuppressed and healthy individuals.

The researchers then used artificial intelligence to analyze antibody patterns in samples collected before and after COVID-19 vaccination, identifying antibody signatures that helped distinguish strong vaccine responders from weak ones. In particular, they found that pre-existing antibodies to common microbes consistently predicted post-vaccination antibody responses in both healthy and immunosuppressed individuals.

These “sentinel antibodies,” the researchers suggest, may represent biomarkers of immune responsiveness to vaccination. “What our study found is that certain biomarkers, when analyzed with AI, can predict who is likely to respond well to a vaccine, even before they receive it,” said study lead Joshua LaBaer, PhD, executive director of the Biodesign Institute at ASU and director of the Virginia G. Piper Center for Personalized Diagnostics. “This suggests that some people may be more immune-ready than others.”

The team’s approach is one of the first to use a broad, pre-vaccine antibody “fingerprint” to assess immune readiness. Unlike some prediction methods that rely on genetic analyses, this strategy uses antibody patterns in blood, which may be easier to adapt for clinical use.

LaBaer and colleagues reported their findings in Cell Press Blue, in a paper titled “Pre-vaccine sentinel antibodies predict blunted vaccine responses,” stating that their results “… identify pre-existing antimicrobial antibody profiles as scalable biomarkers of humoral immune responsiveness and provide a framework for predicting vaccine responses before immunization.”

Vaccines protect most people from serious illness, but the strength of that protection can vary considerably from one person to another. Before a vaccine ever enters the body, the immune system may already hold clues to how strongly it will respond. Age, sex, genetics, prior illnesses, and underlying health conditions have all been linked to how strongly people respond to vaccines. People with immune-compromising conditions are often at higher risk of weaker responses. But even within these groups, outcomes can differ sharply.

Usually, scientists evaluate vaccine response after the shot has been given by measuring whether the immune system has produced antibodies against the target. For their newly reported study, LaBaer and team asked whether antibody patterns already present in the blood might predict an individual’s immune readiness and response to vaccination.

The team looked at antibody responses to 185 antigens, including SARS-CoV-2 antigens, other common viral and bacterial antigens, and targets associated with autoimmune diseases. To do this, the researchers analyzed 8,687 samples from 4,089 participants, including 2,445 healthy volunteers and 1,644 people with conditions or treatments linked to immune suppression, such as HIV, multiple myeloma, solid organ malignancy, autoimmune disease, inflammatory bowel disease, and solid organ transplantation.

They found that several immunosuppressed groups were more likely to have blunted responses to COVID-19 vaccination. But those categories were imperfect predictors. Some immunosuppressed participants mounted strong responses, while about 5% to 6% of healthy participants demonstrated weak responses. The results did find that higher levels of certain preexisting antibodies, including antibodies to common bacteria and viruses, such as Staphylococcus aureus, respiratory syncytial virus, and human respirovirus 3, were associated with stronger COVID-19 vaccine responses.

The researchers describe these as “sentinel” antibodies because they may indicate a person’s baseline immune readiness. They are not necessarily fighting the vaccine target directly. Instead, they may reflect how responsive the antibody-producing arm of the immune system is likely to be. “These broadly prevalent antimicrobial antibodies represent sentinel antibodies that may serve as biomarkers of system-level humoral immune competence,” the team stated.

The researchers then asked whether the full antibody fingerprint, not just a few individual markers, could help identify people likely to have weak vaccine responses. A deep-learning model analyzed patterns across the antibody panel, combining measurements into a broader immune profile. “Using global antimicrobial antibody profiles, we developed a deep-learning predictive model that stratified individuals according to their likelihood of mounting blunted vaccine responses,” they explained.

The study highlights a key strength of AI in health research: its ability to find subtle, predictive patterns in millions of biological data points that might otherwise remain hidden. The approach suggests that vaccine readiness may be better understood by looking at the immune system as a whole, rather than focusing only on a single disease or a single antibody.

The work also highlights the value of newer technologies that can measure large numbers of antibody responses at once. Instead of asking whether someone has antibodies to one pathogen, the method can scan a wider immune landscape, capturing patterns formed by many previous encounters with viruses, bacteria, and other immune targets.

The researchers say the findings could have implications beyond COVID-19 if they are validated in additional studies and with other vaccines. Sentinel antibody profiling could help guide vaccine testing, vaccine development, and clinical care for people at risk of weak immune responses. “Together, these findings identify pre-existing antimicrobial antibody profiles as scalable biomarkers of humoral immune responsiveness and provide a framework for predicting vaccine responses before immunization,” the authors wrote in summary.

The approach might eventually help doctors identify patients who need additional vaccine doses, closer follow-up, or alternative protective measures. It could also help researchers better understand why some people respond well to vaccination while others do not. The work points toward a future in which vaccine decisions could be guided by a person’s own immune readiness.

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Built Without Bacteria: Bringing Cell-Free Synthesis to the Bench

Edusehat — Thu, 20 Aug 2026 21:00:03 +0700

Matthew D. Youngblut, PhD

Co-founder and Chief Scientific Officer
Flock Bio

Divya Vijay Pratheek

Vice President of Product
Ribbon Bio

Broadcast Date: Thursday, September 10, 2026

Time: 08:00 PDT, 11:00 EDT, 17:00 CET

Complex DNA workflows are often slowed by bacterial cloning, challenging genome sequences, and lengthy production cycles. A technology that brings cell-free DNA synthesis directly to the bench would give researchers an accessible, on-demand way to build high-fidelity DNA sequences without bacteria. Such an approach is now offered by Ribbon Bio’s MiroMine^TM: this helps avoid bacterial contaminants such as endotoxins and host-cell DNA, while supporting complex or sensitive sequences that can be difficult to produce through conventional cloning.

In this GEN webinar, speakers from Flock Bio and Ribbon Bio will explore how bench-based, cell-free DNA production can complement expertise in custom DNA design and manufacturing. Drawing on applications including CRISPR screening, enzyme evolution, lineage tracing, and massively parallel reporter assays (MPRAs), they will discuss how MiroMine^TM can reduce dependence on conventional cloning, shorten production cycles, and give researchers greater control and confidentiality. Attendees will learn how accessible cell-free synthesis can accelerate the path from design to experiment and enable cleaner, faster, and more flexible research workflows.

A live Q&A session will follow the presentations, offering you a chance to pose questions to our expert panelists.

Produced with support from:

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Connecting more patients with psoriasis to the care they need

Edusehat — Thu, 20 Aug 2026 06:20:02 +0700

For people living with psoriatic disease, treatment innovation has advanced dramatically. Yet too many patients remain undiagnosed, untreated, or undertreated. The National Psoriasis Foundation (NPF) set out to understand why progress in treatment has not consistently translated into progress in patients’ lives.

Bio.News sat down with Katie Southwick, Vice President of Marketing & Communications at NPF to discuss their research on unreached patient populations and the lived experiences shaping their decisions.

Tell us more about your research on unreached patient populations. What inspired it? Why is there such a great need for this research?

There has been tremendous progress in psoriatic disease treatments. Today, patients are surrounded by messaging about the disease and new treatment options, whether on television, social media, or online. Despite that visibility, a significant portion of people living with psoriatic diseases remain undiagnosed or are still not taking advantage of modern treatment options. That disconnect led us to ask a simple question: Why?

As we dug deeper, we found that out-of-pocket cost and concerns about side effects were certainly a big driver. Importantly, we found that patients’ lived experiences often have a great impact on whether they continue engaging with the healthcare system. Negative encounters with healthcare providers, feeling dismissed, feeling unheard, or becoming discouraged after unsuccessful treatments can all influence future decisions about seeking care.

The need for this research is substantial. There are approximately 8 million people living with psoriatic disease in the United States, and another roughly 600,000 remain undiagnosed. Not managing psoriasis often leads to soaring health care expenses, productivity losses, and expensive long-term medical comorbidities such as cardiovascular disease or diabetes. Yet, many patients have no formal care plan or lack access to specialist care, particularly in rural communities. Understanding why patients disengage is essential if we want advances in treatment to translate into better outcomes for patients.

Your research suggests that the biggest barrier to care is “patient momentum.” Can you explain what this means?

Our research showed that many patients become stuck in what we call a “doom loop.”

Living with a psoriatic disease can be exhausting. Symptoms affect physical health, mental health, confidence, relationships, and daily routines. Over time, patients may experience frustration with the healthcare system, disappointment with not immediately finding the right treatment option that works best for them, or feelings of hopelessness. All of those factors build on one another and make it harder to take the next step toward care.

“Patient momentum” is really about helping people move forward again. Effective engagement is not simply telling someone there is a new treatment available. It is helping them believe that things can improve, that support exists, and that they do not have to continue struggling alone.

The study found that patients often settle into a “good enough” mindset, managing symptoms without seeking better outcomes. What are the risks of this approach?

Psoriasis is not just a skin condition. It is a chronic, systemic immune-mediated disease that can have serious long-term consequences when left inadequately managed.

When patients settle for “good enough,” they may not realize they are still at risk for disease progression and related health complications. For some individuals, that can mean the development or worsening of psoriatic arthritis, which can cause irreversible joint damage if not treated appropriately. Psoriatic disease is also associated with several comorbid conditions that can affect overall health and quality of life.

That is why one of our key messages is that good enough is not actually good enough. We want patients to understand that better outcomes are possible, and that staying engaged in care can help reduce the risk of more serious issues down the road.

What role does trust play in keeping patients engaged in care?

Trust is everything.

Patients are navigating an overwhelming amount of information today, and it is often difficult to determine what is evidence-based and what is not.

Organizations like NPF play an important role because patients need trusted sources of information and guidance. We work alongside leading clinicians, researchers, and patient advocates to help connect people with credible resources and support.

Trust also means recognizing that every patient’s journey is different. The same treatment approach does not work for everyone. Patients want to be seen, heard, and respected in their decision-making. Building that trust is critical to helping people move toward optimal care.

What needs to happen next to ensure that advances in treatment translate into action for the patients who remain disengaged from care?

Patients need to be met where they are. Disengaged patients are not necessarily choosing to ignore their disease. Many have experienced setbacks that have slowed their momentum. By listening to patients, validating their experiences, and demonstrating that better outcomes are possible, we can help more people move beyond the doom loop and toward a healthier future. The encouraging reality is that today’s treatments, combined with trusted support and education, can make a meaningful difference.

August is Psoriasis Action Month. NPF has launched our Is This a Thing campaign, which has the goal of empowering patients through education on psoriasis and validation that: “No matter what this disease does to make you ask: Is this a thing? The answer is typically: Yes, this is psoriasis.

NPF has also recently launched our Milestones to a Cure initiative. As part of this initiative, NPF is investing in innovative research designed to accelerate progress toward a cure for psoriatic disease. This funding opportunity will support researchers pursuing transformative, high-impact projects aimed at advancing our understanding of disease mechanisms, improving treatment approaches, and ultimately bringing us closer to a cure.

If you are interested in learning more about NPF and Psoriasis Action Month, visit https://www.psoriasis.org/psoriasis-action-month. Letters of intent for Milestones to a Cure are due Aug. 31. You can learn more here: https://www.psoriasis.org/milestones-to-a-cure-rfp/

Additionally, if you are a patient advocate, consider registering for the Biotechnology Innovation Organization’s Patient Advocacy Changemakers Event (PACE): an empowering and inspiring event focused on breaking barriers to access and ensuring that innovative medicines reach the patients who need them.

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Rare Immune Cell Type in Supercentenarians May Help Maintain Healthy Aging

Edusehat — Thu, 20 Aug 2026 06:15:07 +0700

Aging is accompanied by an increased risk of certain diseases, alongside weakened immunity to illnesses that younger individuals might easily overcome. This has raised an important question for researchers, which is why do some people live to a very old age while avoiding major diseases?

The results of research by a team at Osaka University studying Japanese supercentenarians aged 110 years and older, now suggest that a rare type of immune cell known as CD4 cytotoxic T lymphocytes, or CD4 CTLs, becomes increasingly abundant with extreme age. These rare cells have the ability to both recognize threats and destroy dangerous cells.

“Immune aging is not simply a process of decline,” said Kosuke Hashimoto, PhD, an associate professor at the University of Osaka in Japan. “The selective expansion of certain T cells suggests that, even in extreme old age, the immune system may continue to adapt to age-related challenges … CD4 CTLs are an atypical and relatively rare T cell population. So their marked increase in supercentenarians may provide important clues as to how the immune system is maintained in extreme old age.”

Hashimoto is first author of the researchers’ published paper in Cell Reports, titled “CD4 CTLs in supercentenarians: Signs of adaptive expansion in healthy aging,” in which they say that their collective findings “… suggest that CD4 CTLs expand and diversify as an adaptation to persistent antigens, potentially contributing to longevity through cancer suppression.”

While maintaining good health as we age is important, it is also difficult, considering that the risk of certain diseases increases with age, alongside weakened immunity to some illnesses. “Aging is an inevitable biological process, characterized by gradual declines in physical and cognitive functions. This process is driven by the accumulation of molecular and cellular alterations, including DNA mutations, mitochondrial dysfunction, and cellular senescence,” the authors wrote.

Among the various changes that occur at the cellular and molecular level, the team continued, “… aging of the immune system compromises its ability to defend against external pathogens and eliminate internal abnormal cells such as precancerous or senescent cells, increasing the risk of various diseases.”

This has raised an important question for researchers: why do some people live to a very old age while avoiding major diseases? Aging itself is not a disease, they commented, nor does it necessarily lead to the failure of essential physiological systems.

“Supercentenarians—individuals who live to or beyond 110 years—provide a model of healthy aging, achieving longevity while avoiding or delaying major age-related diseases such as cardiovascular disorders and cancer,” the investigators commented. “They maintain immune, cardiovascular, and epigenomic profiles that appear younger than expected for their chronological age.”

One class of immune cells, T cells, is traditionally divided into two groups: helper cells that coordinate immune responses and killer cells that destroy infected or cancerous cells. However, supercentenarians accumulate an unusual hybrid known as CD4 cytotoxic T lymphocytes, or CD4 CTLs. “Our previous study identified CD4 cytotoxic T lymphocytes (CD4 CTLs) as a hallmark of supercentenarians,” the team stated. These rare cells have the ability to both recognize threats and destroy dangerous cells. “CD4 CTLs are an atypical and relatively rare T cell population,” Hashimoto said. “So, their marked increase in supercentenarians may provide important clues as to how the immune system is maintained in extreme old age.”

To understand exactly how rare these hybrid cells are, the scientists analyzed blood samples of 28 adults who had been divided into three age groups: 70–99 years, 100–109 years, and 110 years and older. “In this study, we conducted integrated single-cell profiling of T cells, combining transcriptome, surface protein, and TCR sequence data across different age groups, including rare cohorts of centenarians and supercentenarians,” the investigators explained.

They found that the proportion of CD4 CTLs increased with age, with median percentages of 4%, 9.6%, and 17.6%, respectively. The hybrid cells remained uncommon throughout most of life, but were found to expand dramatically around the age of 100. In supercentenarians, these cells made up nearly one-fifth of all T cells in the blood, whereas they only comprised about 4% of total cells in the younger study participants. “We analyzed a rare cell population that is enriched in supercentenarians and found signs of immune remodeling in extreme old age,” said Hashimoto. “Rather than showing signs of exhaustion, they remain highly active and may help the body cope with persistent threats that increase with age.”

While the study findings suggest CD4 CTL expansion begins around 100, this phenomenon was not unique to centenarians and supercentenarians; one participant younger than 100 had the highest proportion of these cells. The team then looked at participants’ T cell receptors and determined that clonal expansion helps drive this increase. CD4 CTLs clone themselves when the immune system is under attack. The newly reported study found that the most prominent clone accounted for an average of 33.3% of CD4 CTLs, indicating that older adults may be responding to persistent immune threats. In one centenarian’s blood sample, a single clone accounted for 53.8% of their CD4 CTLs.

Next, the researchers matched the receptor sequences of each participant’s top CD4 CTL clone to those in a public database. Nearly three dozen matches belonged to people with cancer—namely lung, breast, and liver cancers. Because none of the centenarians or supercentenarians studied had been diagnosed with these cancers, the researchers theorized that their CD4 CTL expansion may reflect early immune responses. “Some CD4 CTLs may recognize cancer-related targets, although their exact targets remain unknown,” Hashimoto said. “The similarity between these receptor sequences and those found in T cells from tumors suggests that these cells may help recognize tumors before they become clinically detectable. It may be that immune changes in extreme old age are better understood as the immune system reorganizing itself rather than just wearing out.”

The study, which focused on T cells circulating in the blood, does not prove that having an abundance of CD4 CTLs prevents cancer or causes a person to live longer. However, the findings provide one of the clearest pictures yet of how the immune system adapts in people who achieve exceptional lifespans, offering insight into healthy aging and potentially contributing to protection against disease in supercentenarians.

“Our results revealed that CD4 CTLs expand with age and exhibit plasticity in cytokine production,” the team wrote in summary. “This suggests that CD4 CTLs may contribute to cancer suppression and longevity through adaptive responses to persistent antigens, emphasizing their role in immune resilience and healthy aging.”

Hashimoto noted that the team’s next step is to research how these cells behave in human tissues. “As we age, abnormal cells, including senescent and cancerous cells, become more common,” Hashimoto stated. “Our findings suggest that immune adaptation to these changes may contribute to exceptional longevity.”

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Human Multi-Organ Chip Offers New Insight Into Cancer Metastasis

Edusehat — Thu, 20 Aug 2026 06:15:06 +0700

Cancer metastasis is responsible for at least two-thirds of cancer deaths. Drugs targeting the metastatic progression have largely failed, in part due to the lack of predictive models that would help identify the underlying mechanisms of metastasis.

Now, new work reports the development of a multi-organ chip that mimics how cancer cells spread from vascular flow to distant organs—the first model of cancer metastasis of its kind. The chip includes compartments with millimeter-sized engineered human bone and lung tissues, and the vascular flow that contains circulating breast cancer cells and allows the dynamic cross-talk of the tissues being colonized.

“The key advantages of this advanced model of metastasis are that it is human and can be patient-specific,” said Gordana Vunjak-Novakovic, PhD, university professor and professor of biomedical engineering and professor of medical sciences at Columbia University. “It faithfully mimics some of the key aspects of human metastasis that are otherwise largely inaccessible for direct study.”

This work is published in Science Translational Medicine in the paper, “Organ-specific colonization and niche remodeling in a human tissue model of metastasis.”

“The pressing need for developing human tissue models of metastasis has been a key motivation for our study,” said Vunjak-Novakovic. “Our objective was to probe the ability of cancer cells to adhere to and traverse across endothelium [inner lining of blood vessels], and to determine their capacity to survive in the tissues they are colonizing through cell reprogramming and niche remodeling.”

The study sheds light on a critical phase of metastasis, known as organ colonization, which is difficult to study using animal models. The process is highly complex, requiring the cancer cells to evade tissue defense and adapt to the specific organs they invade.

The multi-organ chip allows scientists to investigate, in detail, metastatic progression with patient cells and tissues. The platform enables controlled experimentation of cancer cell-tissue interactions within organ-specific microenvironments, toward revealing molecular pathways and therapeutic targets for metastasis.

To demonstrate the chip’s capabilities, the team examined the colonization of circulating human breast cancer cells in bone and lung. Bone and lung, common sites of metastasis for breast cancer, and the vascular endothelium were engineered from induced pluripotent stem cells (iPSCs) using tissue-specific scaffold-bioreactor culture systems. The engineered tissues were maintained in individual compartments of the chip, which were each optimized for tissue maturation and long-term maintenance of functionality, and linked to each other by vascular circulation.

A selectively permeable endothelial barrier separates tissue compartments from the vascular channel, as in the human body. Once the platform was established, the researchers introduced breast cancer cells into the vascular circulation to observe patterns of organ-specific colonization. In line with what happens inside the human body, cancer cells that typically gravitate toward the bone showed stronger bone colonization and induced more pronounced bone degeneration. In contrast, cancer cells that typically gravitate toward the lung caused greater disruption in the lung tissue and only modest colonization of bone. Distinct patterns of tissue colonization and secreted factors demonstrate that this device mimics key features of organ-specific metastasis observed in the human body.

In addition, a post-analysis of the engineered tissue revealed that cancer cells condition the distant organs to be more receptive to colonization. The team saw signs of this process—called pre-metastatic niche formation—across both tissue compartments.

“Cancer is very smart, unfortunately. We learned how the cells cross barriers to get from blood circulation into the tissues,” said Vunjak-Novakovic. “We were also able to reproduce something that happens in patients, where cancer cells condition the target tissues, even before they colonize them, to make them more receptive.”

The work is aligned with the trend of more preclinical research in the future, including engineered human tissues that can complement what is learned from animal models. “As the FDA and NIH place growing emphasis on new approach methodologies, this study is a concrete example of what that shift can look like in practice, applied to one of cancer’s most challenging hallmarks: metastasis,” noted Ilaria Baldassarri, a PhD student at Columbia University.

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Not all patients benefit from progress against psoriasis

Edusehat — Thu, 20 Aug 2026 02:45:02 +0700

Tell us more about your research on unreached patient populations. What inspired it? Why is there such a great need for this research?

Your research suggests that the biggest barrier to care is “patient momentum.” Can you explain what this means?

Our research showed that many patients become stuck in what we call a “doom loop.”

The study found that patients often settle into a “good enough” mindset, managing symptoms without seeking better outcomes. What are the risks of this approach?

Psoriasis is not just a skin condition. It is a chronic, systemic immune-mediated disease that can have serious long-term consequences when left inadequately managed.

What role does trust play in keeping patients engaged in care?

Trust is everything.

Patients are navigating an overwhelming amount of information today, and it is often difficult to determine what is evidence-based and what is not.

What needs to happen next to ensure that advances in treatment translate into action for the patients who remain disengaged from care?

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Gene Switch Uses Electromagnetic Fields to Control Genes Remotely

Edusehat — Thu, 20 Aug 2026 02:40:12 +0700

Researchers say gene switches currently offer limited temporal and spatial precision and can also have adverse effects. Now, a recent study, “Electromagnetic field-inducible in vivo gene switch for remote spatiotemporal control of gene expression” in Cell, describes an electromagnetic field-inducible gene switch that enables fully reversible, safe, and precise control over gene expression.

The scientific team from Dongguk University in South Korea believes this represents a powerful non-invasive tool for understanding gene expression and for gene therapy.

DNA contains regulatory elements that control when, where, and to what extent specific genes are turned on or off. They can be co-opted to create “gene switches” that hold significant potential for understanding gene expression and for therapeutic applications, particularly for the non-invasive treatment or management of genetic disorders.

In recent years, researchers have developed several gene switches that enable the remote control of gene expression in living organisms using stimuli such as drugs, light, heat, ultrasound, and electrical signals. However, current versions are limited in offering precise control over the timing and duration of gene expression, according to the Dongguk University investigators. Additionally, drug-based gene switches can have undesirable adverse effects, while some stimuli, such as light, can make penetrating deeper tissues challenging.

Addressing these limitations, Jongpil Kim, PhD, and doctoral student Yerim Hwang from the Institute for Stem Cells and Regenerative Medicine at Dongguk led a group that developed a novel electromagnetic field (EMF)-responsive gene switch.

Researchers have developed several gene switches that enable the remote control of gene expression in living organisms using stimuli such as drugs, light, heat, ultrasound, and electrical signals. However, current versions are limited in offering precise control over the timing and duration of gene expression. [Nicolas/Getty Images]
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“In previous studies, extremely low frequency EMF fields have been shown to modulate expression of specific genes involved in stress response, epigenetic remodeling, and cellular signaling pathways. Moreover, EMF is non-invasive, fully reversible, and can precisely penetrate target tissues or areas of the body, making it highly attractive for remote control of gene switches,” explained Kim. “In this study, we utilized the promoter of the Lgr4 gene to create a robust EMF-inducible gene switch and demonstrated its applications in Alzheimer’s disease (AD) modeling and reversing aging markers in mice.”

To identify EMF-responsive genes, the researchers performed single-cell RNA sequencing (scRNA-seq) on mouse brain tissue following exposure to an EMF of 2.0 millitesla at 60 hertz. The team found exclusive upregulation of Lgr4 expression. Through a series of validation experiments, the team found that the promoter of Lgr4 was well suited for constructing an EMF-inducible (Ei) gene switch, exhibiting precise activation with no detectable adverse effects during the study.

To evaluate the system in living animals, the researchers linked the Ei element to a reporter that produces green fluorescent protein (GFP), allowing gene activity to be visualized. They then generated transgenic mice carrying this reporter. Following EMF exposure, the mice showed strong GFP expression throughout the body, while targeted EMF exposure produced localized gene expression in specific organs.

When EMF stimulation was discontinued, gene expression returned to baseline within 24 hours, demonstrating that the Ei gene switch is highly tunable, reversible, and capable of precise remote control of gene expression.

Using a genome-wide CRISPR-Cas9 knockout screen, the researchers identified cytochrome b5 type B (Cyb5b), a membrane-associated protein, as the biological sensor for EMF. “This may be the first reported molecular sensor for electromagnetic fields,” noted Kim. Further tests revealed that due to EMF exposure, Cyb5b produces rhythmic calcium influx oscillations in cells, functioning as a specific code for activating the target gene.

The researchers also demonstrated several applications of the Ei gene switch, e.g., they established an AD mouse model that decouples brain aging from amyloid β plaque deposition. In addition, cyclic EMF exposure was used to achieve partial cellular reprogramming in aged and progeroid mice, improving several aging-associated markers without detectable adverse effects. The team also restored serotonin levels and reduced depression-like behaviors in mice by controlling expression of the Tph2 gene.

“This technology could move gene therapy away from a single, irreversible dose and toward simpler, real-time treatments administered by physicians or even wearable devices,” said Hwang.

Although further validation and testing are required, the Ei gene switch represents a promising platform for developing non-invasive, remotely controlled gene therapies, she added.

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Vector Production a Bottleneck for Gene Therapy Sector

Edusehat — Thu, 20 Aug 2026 02:40:10 +0700

Inefficiencies that limit the global supply of viral vectors are negatively impacting the gene therapy industry, according to researchers, who say there is an urgent need for better downstream purification materials.

The call came from Stefano Menegatti, PhD, professor, Department of Chemical and Biomolecular Engineering at North Carolina State University, who says the limited availability of viral vectors has become a major bottleneck.

“Gene therapy is one of the most transformative frontiers in modern medicine. It holds the promise of curing devastating diseases with a single treatment. Adeno-associated viruses (AAVs) are the leading delivery vehicles for these therapies.

“Yet for all the excitement in the field, manufacturing remains a critical bottleneck: producing AAVs at the purity, potency, and scale needed for clinical use is enormously challenging,” he tells GEN.

A key obstacle is that current purification materials—usually resin-based affinity adsorbents—cannot distinguish between full AAV capsids that carry genetic material and empty capsids that have no payload.

Another issue with current purification technologies is the need to use low flow rates, which increases processing costs and, ultimately, gene therapy prices, Menegatti says.

“Purification technologies operate at slow flow rates, require harsh chemical conditions that can damage the product, and wear out quickly, all of which drive up manufacturing time and cost.

“For patients waiting on life-saving treatments, these are not abstract engineering problems. They translate directly into delayed access and higher prices,” he says.

Purification materials research

In May, Menegatti and colleague Michael Daniele, PhD, were awarded a NIIMBL grant to further develop a purification method with novel materials that they claim can differentiate between full and empty capsids.

“At the heart of it are AvXcel affinity adsorbents—developed by ChromaGenix—which substantially accelerate purification. More importantly, they selectively enrich full, gene-loaded AAV capsids directly at the capture step.

“Our preliminary data show that the fraction of full capsids increases from roughly 20−30% in the raw material to 34−48% in the affinity eluate, far outperforming the industry benchmark,” he says.

In addition, the membranes can also withstand harsh cleaning processes—up to 50 cycles with caustic solutions—which is in line with industry needs, Menegatti adds.

“The combination of speed, selectivity, and durability is genuinely unprecedented. We are not making incremental improvements; we are redesigning the purification step from the ground up, with the goal of transforming a months-long process development campaign into one that takes weeks.”

Machine learning

The NIIMBL grant will also support the ongoing development of a machine learning-based analytical software platform, called Beacon, designed to help manufacturers optimize vaccine purification.

Daniele tells GEN, “Even with next-generation purification materials, optimizing the process for each new AAV target remains a major challenge.

“Today, the standard approach is a trial-and-error methodology that requires dozens to hundreds of experiments to map out the right operating conditions for each new product. This must be repeated essentially from scratch for every new AAV serotype and transgene combination, which is both time-consuming and expensive.”

Beacon, or Bayesian-Enhanced AAV Chromatography Optimization Network, is a machine learning platform designed to help process developers avoid such repetition.

“It uses a Gaussian Process algorithm, a type of Bayesian AI that learns from each experiment and predicts the next most informative one to run. Rather than blindly sweeping through conditions, Beacon intelligently navigates the optimization landscape, reducing the experimental burden by 30−50% compared to conventional DOE while simultaneously optimizing multiple performance criteria: yield, full capsid enrichment, impurity clearance and productivity,” Daniele continues.

Another key Beacon feature is the use of North Carolina’s VVIRAL database, which comprises thousands of AAV purification experiments, to “warm-start” new campaigns.

Danielle tells GEN, “In other words, it doesn’t start from zero for each new product; it leverages everything we’ve already learned. And unlike a black-box AI, Beacon provides interpretable, quantitative outputs through SHAP analysis, so scientists understand why a particular protocol works, enabling smarter decisions and better risk management.

“Beacon will be released as an open-access, cloud-hosted platform to the broader biomanufacturing community, democratizing access to cutting-edge AI tools that were previously available only to a handful of computational specialists,” he adds.

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Cell Therapy Company Aims to Pioneer Analytics Automation

Edusehat — Thu, 20 Aug 2026 02:40:09 +0700

BlueRock Therapeutics says it has developed an automated system for high-throughput auditing of this relatively new class of therapeutics. According to BlueRock, the system, which accommodates cell culture, liquid handling, and processing with small volume samples, is designed to fill a gap between the need for solutions and those currently available.

“In modalities like small molecules, platforms are more or less plug-and-play and so are the systems used for automation, but there’s been a gap as people have moved to this new modality,” explains Ronnie Lum, PhD, director of analytical and quality control.

To contribute to the growing cell therapy ecosystem, Lum and his team have adapted readily available tools and systems to the smaller batch volumes produced in cell therapies.

“This field is new,” he says. “We want to be a pioneer and really push the envelope of where we can go with automation. Specifically in the analytical audit space, there aren’t many solutions out there, so we’ve really put the effort in.”

The customized system has been designed and built in collaboration with Hamilton Robotics and involves adapting the company’s existing analytical systems to liquid handling and, they hope, high-throughput automation with the aim of scaling up to commercial volumes in this new field.

The company hopes their new system will help them reach the market faster with their own pipeline of therapies and can be adapted easily afterward to the processes involved with their new products.

In general, they hope these technologies will drive down the cost of analytics across the cell therapy industry by reducing the sample volumes needed for testing and, thus, the cost of reagents used in complex assays.

Going forward, BlueRock Therapeutics urges other companies to join them in developing systems to address technological gaps. “We’re willing to push the boundaries by investing in these technologies and trying to innovate.”

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Cutting Costs in Serum-Free FGF2 Processing With Recycled Media

Edusehat — Thu, 20 Aug 2026 02:40:08 +0700

Producing fibroblast growth factor 2 (FGF2) using fortified spent cell culture media from Lactococcus lactis can cut the costs of serum-free cell culture media while maintaining high production titers, according to recent research from Singapore’s Agency for Science, Technology and Research (A*STAR). This approach helps overcome some of the barriers associated with serum-free media and eventually may even eliminate the need for the vast quantities of fetal bovine serum used today.

“This project establishes a high-value circular manufacturing framework by capturing nutrient-rich spent media side-streams generated during biopharmaceutical cell culture and repurposing them as low-cost feedstock for precision microbial fermentation,” Dave Ow, PhD, a principal scientist and group leader, microbial cell bioprocessing at Bioprocessing Technology Institute (BTI), A*STAR, tells GEN.

The Singaporean team led by Ow and Prashant Mainali, PhD, a bioprocess scientist and researcher at the BTI, A*STAR, chose L. lactis as the production cell because it has a doubling time of 35−60 minutes and can secrete recombinant proteins, thus minimizing downstream purification steps. Because the spent fermentation media still contains nutrients, reusing and fortifying it lowers the cost of formulating fresh media, thereby enhancing productivity and lowering FGF2’s cost per gram.

For further gains, the team adapted this approach for a continuing manufacturing process for both intracellular production and secretion of FGF2, optimizing process conditions and integrating that model with downstream purification. Using it, they achieved 16 mg/L titers for intracellular forms and 396 µg/L titers for secreted forms of FGF2. Optimal conditions, they report, are 10 g/L glucose, 35°C cultivation temperature, and 100 ng/mL nisin.

Optimization focused around glucose concentration, temperature, and nisin concentration. “The interaction between glucose concentration and temperature was statistically significant,” they note. The glucose/nisin interaction was not. Increasing dilution rates increased FGF2 production to a point, after which washout occurred. Therefore, balancing the tradeoffs—for example, minimizing nutrient loss or maximizing FGF2 concentration or total output—is critical.

The scientists used a chemostat process, which enables continuous bioprocessing under steady-state culture conditions, to simulate process outcomes under varying conditions. The results, they report, “captured overall trends…[and] accurately predict the final concentrations of cells, glucose, lactate, and total intracellular FGF2 at the end of the chemostat run.” It underpredicts FGF2 concentrations, however.

Then they added depth filtration and crossflow filtration to the chemostat process, mimicking typical purification steps used in Escherichia coli, showing that such integration and streamlining is feasible. The FGF2 from this process was used without further purification to supplement cell cultures. When applied to a culture of Anguilla japonica (Japanese eel) cells, “Their growth was comparable to cells cultured with commercially available FGF2 and FBS-supplemented media,” Mainali and colleagues note.

“Our findings demonstrate that fortified spent cell culture media can support the growth of L. lactis and the secretion of FGF2,” Ow, Mainali, and colleagues conclude. Secretion simplified downstream purification, and the reuse of spent media is both cost-effective and environmentally sound, “potentially lower[ing] the cost of FGF2”…and “helping to overcome one of the principal economic barriers to serum-free media.”

Already, Ow says, “There is interest from biopharma manufacturers to potentially use this approach to upcycle spent media for their cell culture production processes to reduce media waste toward a more sustainable biomanufacturing future.”

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CAR T Manufacturing Innovation Expands Patient Access

Edusehat — Thu, 20 Aug 2026 02:40:07 +0700

As the next generation of CAR T therapies moves closer to broader commercialization, industry leaders say the future will depend less on choosing between ex vivo and in vivo approaches and more on advancing both in parallel. Improvements in manufacturing, automation, and vector engineering are expected to expand patient access while addressing long-standing challenges around scalability, cost, and consistency.

Speaking to GEN, experts from VIVEbiotech, Terumo Blood and Cell Technologies, and Bracco describe an industry transitioning from demonstrating scientific feasibility to building robust manufacturing systems capable of delivering life-changing therapies to many more patients.

Parallel paths for CAR T innovation

“Ex vivo CAR T therapies will continue to play a critical role, while in vivo approaches are rapidly advancing and have the potential to broaden patient access by simplifying treatment pathways,” Natalia Elizalde, PhD, chief business development officer at VIVEbiotech, says.

Rather than viewing the two technologies as competitors, she expects them to develop side by side, each serving different clinical and operational needs. According to Elizalde, the industry’s priorities are shifting beyond proof-of-concept toward making therapies more widely available and economically sustainable.

She identifies advances in lentiviral vector engineering and targeted delivery technologies as major drivers of future progress. Improvements in manufacturing scalability and process robustness will also be crucial as commercial demand grows.

“The future of CAR T will not be defined by a single technological approach,” Elizalde says. “It will be defined by the ability of different platforms to address unmet patient needs while improving scalability, accessibility, and sustainability.”

Automation tackles manufacturing variability

While scientific innovation continues, manufacturing remains one of the biggest barriers to broader adoption. According to Wenyan Leong, PhD, director, APAC commercial, cell and gene therapies at Terumo Blood and Cell Technologies, the greatest challenge in ex vivo CAR T production is not any individual manufacturing step but the cumulative variability created throughout the workflow.

“Every manual intervention, open manipulation, operator-dependent activity, and process handoff introduces opportunities for variability, deviation, and operational complexity,” Leong says.

That variability extends from cell collection through processing, expansion, quality control, and final-product release. As manufacturers scale production, maintaining consistent product quality becomes increasingly difficult and expensive.

Leong believes automation offers a practical solution, although implementation should be tailored to each developer’s manufacturing strategy rather than applied universally.

“A useful framework is to automate where risk is highest, where variability has the greatest impact on product quality, and where scaling creates the most operational burden,” she explains.

For many manufacturers, cell expansion represents the most logical place to automate because it is labor-intensive, highly dependent on operator expertise, and difficult to reproduce consistently across multiple sites. By reducing manual processes and simplifying operations, manufacturers can lower costs while improving reproducibility and expanding patient access, Leong says.

Integrated manufacturing supports industrialization

Sophie He, PhD, vice president, cell therapy at Bracco, points to additional manufacturing bottlenecks that continue to slow commercialization. For example, she says ex vivo CAR T manufacturing must overcome complex supply chains, logistical challenges, and significant analytical demands that increase both timelines and production costs.

“In autologous manufacturing, patient-to-patient variability and poor starting-cell quality lead to inconsistent transfection and expansion,” He says. “In allogeneic approaches, the priority is obtaining highly pure starting material before stem cell differentiation into T cells.”

He also highlights fragmented cell selection and activation workflows as major contributors to cellular stress, inconsistent yields, and reduced product quality across both therapy modalities.

“Integrating multiple processing steps, such as cell selection and activation, into a single closed, continuous workflow reduces handling, washing, production time, and cost while improving cell viability and consistency,” He says.

According to He, combining workflow integration with automation and flexible manufacturing models, including point-of-care production, will enable reliable, high-throughput, and cost-effective CAR T manufacturing.

Taken together, these experts see the future of CAR T therapies resting on a combination of scientific advances and manufacturing innovation. As vector technologies mature and automated production becomes more sophisticated, both ex vivo and in vivo approaches are expected to complement one another, creating a more scalable and accessible ecosystem capable of bringing advanced cell therapies to far more patients.

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Build patient access by strengthening innovation, BIO tells senators

Edusehat — Wed, 19 Aug 2026 23:10:03 +0700

BIO’s response to a request for information from the Drug Price Working Group explains why price setting won’t work.

Improving patient access to new treatments requires policy that supports America’s innovation ecosystem—not price setting that discourages investment, BIO tells a Senate working group.

Written comments by the Biotechnology Innovation Organization (BIO) advise against importing foreign pricing or increasing government setting of prescription drug prices.

Instead, BIO recommends reducing patient out-of-pocket costs, through measures like pharmacy benefit manager (PBM) reform, as well as policy to encourage investment in innovation and address aggressive biotech competition from China.

BIO’s 46-page policy analysis is a response to a “Request for Information: Commonsense Policy Options to Lower Drug Prices for Patients,” issued by the Drug Price Working Group led by Senate Finance Committee Ranking Member Ron Wyden (D-OR) with Sens. Catherine Cortez Masto (D-NV), Peter Welch (D-VT), Ruben Gallego (D-AZ), Mark Kelley (D-AZ), Tammy Baldwin (D-WI), Maggie Hassan (D-NH), Jeff Merkley (D-OR), Chris Van Hollen (D-MD), Tammy Duckworth (D-IL), and Richard Blumenthal (D-CT).

The goal of the RFI is to provide detail that can shape proposals from the Democratic working group.

“Sustaining America’s world-leading biotechnology pipeline will require policymakers to ensure that policies intended to improve affordability strengthen, rather than weaken, the ecosystem that makes continued innovation possible,” BIO’s Aug. 17 comments say.

BIO addresses various areas for action suggested by the Working Group’s RFI.

Price setting

The current drug-price negotiation program (DPNP), which will set prices on 100 drugs within the next five years, is already a drag on investment, and there is no need to increase its reach, BIO says. In particular, proposals to use international reference pricing ignore the realities of the U.S. market, which provides quicker and more robust access to medicines, the comments note.

To ensure investment in needed innovation, it is recommended that certain protections from DPNP are added or extended. Biologics and small molecule drugs should both have 13 years of protection from the DPNP, and there should be additional exemptions to promote development of orphan drugs for rare diseases, BIO says. Furthermore, the IRA Small Biotech Exception should not be allowed to expire in 2028:

“Protections for small biotech companies from the DPNP should be extended to support small and emerging companies that drive early-stage innovation,” say BIO’s comments.

Across the more than 2,000 biotech companies in the U.S., approximately 300 are publicly listed, and most are small or mid-sized companies, according to a BIO fact sheet. Today, these small and mid-sized companies account for 71% of the industry’s total employment, originate 49% of all new drugs launched globally, and account for 54% of Food and Drug Administration (FDA) filings.

BIO’s comments to the RFI also recommend policies to enable value-based pricing: “A holistic consideration of a treatment’s value to patients, families, and society should be the overarching principle when developing policies to provide sustainable access to novel, innovative, and transformative therapies.”

Increasing patient access

BIO’s recommendations for reducing the amount patients pay for drugs include reforming PBM practices and a range of other steps to ensure patients have access to needed medicines.

For example, automatic enrollment in the Medicare Prescription Payment Plan (MPPP) would make sure all patients have help in “smoothing” out payments over the course of a year.

Regarding “step therapy,” in which patients are expected to “fail first” on a cheaper medicine before receiving coverage for the drug their doctor prescribes, BIO calls for establishing clear exemption standards for patients in all types of coverage and eliminating step therapy for Part B drugs in Medicare Advantage.

BIO also encourages Congress to address other insurer practices that can restrict access, including eliminating copay accumulator programs, “so all forms of copay assistance count toward patient deductibles.” Furthermore, alternative funding programs (AFPs) that delay or prevent access by diverting insured patients to charitable assistance programs should be prohibited, BIO says.

Regarding PBMs, which use their market power to profit by driving up the cost of prescription drugs, BIO recommends several reforms, including:

“Requiring fees to be transparent, delinked from medicine prices and formulary placement, consistent with fair market value, and not contingent on business conducted with PBM affiliates.”
Insulating clinical decision-making from rebate and fee incentives.
“Addressing vertical integration and associated value extraction enabled by self-dealing.”
Expanding PBM transparency requirements.
“Removing contractual barriers that prevent plans, employers, manufacturers, and patients from pursuing lower-cost options.”

Encouraging innovation to maintain U.S. biotech leadership

BIO notes the remarkable advances achieved through biotech innovation.

“Continued progress depends on a research ecosystem that supports scientific discovery, rewards risk-taking, and enables innovators to efficiently translate breakthrough science into therapies that reach patients,” BIO says.

BIO’s comments urge expansion of federal programs supporting translational research, such as the National Institutes of Health (NIH) and ARPA-H, as well as addition of new types of support for acceleration and commercialization.

Along with providing new treatments, innovation in the U.S. allows America to maintain its global biotech leadership in the face of China’s concerted efforts at competition, which can pose supply chain and security threats, BIO says.

“Investments in clinical trial infrastructure, regulatory modernization, workforce development, and manufacturing capacity can make the United States more competitive, while maintaining the scientific standards that distinguish FDA and the broader American system,” according to BIO.

China has become particularly competitive in conducting first-in-human clinical trials by enabling these trials to take place more rapidly there than in the U.S. BIO recommends several clinical trial reforms, including: streamlining regulatory processes, reducing operational burdens, improving patient participation and site activation, and modernizing Investigational New Drug (IND) requirements and review processes.

Overall, BIO recommends leaning into the natural strengths of the U.S. biotech ecosystem. “America’s advantage has always been its ability to innovate,” say BIO’s comments. Policies that drive innovation by enabling investment are therefore encouraged.

Read BIO’s full comments on the RFI.

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Mid-year review: State-level legislative action impacting biotech

Edusehat — Wed, 19 Aug 2026 16:00:03 +0700

2026 was a busy year on the state level. Whether it was the expected healthcare policy priorities or newer ones, there was plenty to keep legislators, patient advocates, and industry leaders busy.

“Given the majority of state legislatures are adjourned and the fact we have a major election in November, which includes 39 gubernatorial elections, I think we’ve pretty much seen what we’re going to see from the states this year, says Patrick Plues, Senior Vice President, State Government Affairs and Affiliate Relations at the Biotechnology Innovation Organization (BIO).

So what were some of the major policy areas this year, and how are they affecting patients?

340B, PDABs, and PBMs

“The issues we saw this year on prescription drug affordabihlity boards (PDABs), Most Favored Nation (MFN) and Maximum Fair Price (MFP) reference pricing, continued scrutiny of vaccines—we’ll anticipate seeing those again next year,” says Plues.

When it comes to PDABs, there was legislation happening in a number of states, including Colorado, Hawaii, Illinois, Louisiana, Minnesota, Virginia, Vermont, and West Virginia. However, these boards are having trouble proving their financial viability and usefulness when established.

“No new PDABs were introduced this year, and I think that is because of a better understanding of the many actors within the supply chain who impact prescription drug prices,” explains Plues. “There is also the question: Do PDABs actually work? These boards are expensive, and to date, we have not seen them successfully bring down drug prices for patients, and legislators see that.”

BIO has also tracked 63 340B-related bills across 20 states and Puerto Rico.

“340B is a perennial issue in the US,” says Plues. “There needs to be more transparency and accountability from the entities that are receiving 340B dollars on where those dollars are going, and there needs to be better tracking of how 340B products are being dispensed.”

In particular, advocates and biotech leaders are calling out the issue of duplicate discounts, a prohibited practice where a 340B hospital might receive a 340B upfront discount and the state would also receive a Medicaid rebate.

If a hospital provides medication to a Medicaid patient, Plues explains, then that product needs to be reimbursed either through the Medicaid program with the State claiming a rebate, or the state forgoes the rebate and reimburses the providers at the 340B discount rate—often because it may be lower. However, what ends up happening is that manufacturers often end up paying both the Medicaid rebate and discount, with hospitals being the ultimate beneficiary because they get 340B revenue that they are not entitled to.

“Those duplicate discounts are prohibited in the federal statute that governs the 340B program,” Plues says. “Hospitals must work to protect against duplicate discounts, but their current practices only obfuscate and make it harder to identify them. There needs to be legislation to address that because it costs the state and patients more money in the long run.”

And that is not the only 340B-related issue either. In Arkansas, the Attorney General has moved to file a complaint in federal court against manufacturers who are not complying with Arkansas Act 1103 enacted in 2021. This is significant because Arkansas Act 1103 was one of the earliest state laws to address certain 340B-related practices between contract pharmacies, manufacturers, pharmacy benefit managers (PBMs), and payers.

“This is the first court case we’re seeing of a state trying to sue manufacturers for non-compliance of a state 340B law,” explains Plues. “That’s pretty significant, and it is something we are watching closely.”

Lastly, there have been a number of bills aimed at addressing PBM transparency and pharmacy vertical integration in the states.

While BIO currently remains neutral on vertical integration bills, it does agree that comprehensive structural reforms addressing PBM vertical integration and practices are best overseen through coordinated federal and state action.

The vaccine battle

“One trend that began popping up in a number of states are bills aimed at holding manufacturers liable for any injury caused by a vaccine,” says Plues. “Really it is just another attempt by the anti-vaccine movement to curb vaccine usage in their states.”

The federal Vaccine Injury Compensation Program (VICP), formed by bipartisan legislation in 1986, is a program, funded by a tax on manufacturers, that has ensured people injured by certain vaccines are provided with a fair and efficient process for compensation. People who have been injured have their cases heard by dedicated judges with experience in vaccine injury, making the process oftentimes faster and more efficient than civil litigation.

BIO maintains the VICP program is the most effective and efficient mechanism to address vaccine-related injuries. BIO counters state-level vaccine-injury bills with a focus on educating state policymakers on why the VICP program is a better alternative for people seeking relief and restitution than developing state liability bills. Learn more about VICP with this BIO resource.

Thankfully, there is also some positive vaccine legislation on the state level.

“We just saw Pennsylvania become the first state to make immunization rates public for all of their school districts, and that’s something we think is a good idea—no other state has done that yet,” says Plues. Learn more with the Pennsylvania School Immunization Rates Interactive Data Tool.

Artificial Intelligence

“There is great interest among state legislators to regulate AI, particularly given that we don’t have a federal law in place yet,” says Plues. “So the states are taking it upon themselves to regulate it.”

In particular, AI’s potential to help drug manufacturers research and develop newer and more effective medicines could change the healthcare landscape as we know it. AI’s use, BIO notes, should be facilitated and protected.

Yet, there is another area of the healthcare world where the use of AI has already caused some concern: insurance coverage. BIO supports legislation that would implement safeguards around insurers’ use of AI in coverage and reimbursement decisions. State and federal guardrails need to be in place to ensure that AI is not being used to systematically deny or restrict access to care.

As we round out 2026 and head into 2027, BIO will continue to work on the ground in the states to ensure that patients of all kinds not only have access to the drugs and treatments they need, but that biotech can continue to innovate and develop breakthroughs.

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Sugar-Sweetened Beverages Daily Can Lead to Higher Stomach Cancer Risk

Edusehat — Wed, 19 Aug 2026 05:10:10 +0700

Mass General Brigham Cancer Institute researchers found that consuming one or more sugar-sweetened beverages every day was associated with an increased risk of developing gastric cancer, while artificially sweetened beverages were not associated with an increased risk. Results from the study were published in Gastro Hep Advances, in an article titled “Association between sugar-sweetened and artificially sweetened beverage intake and gastric cancer incidence.”

Previous research has linked sugar-sweetened beverages with an increased risk of colorectal, breast, and liver cancers, but evidence on gastric cancer has been limited. The study analyzed data from 112,284 participants in the Nurses’ Health Study and Health Professionals Follow-Up Study. Both studies gathered detailed diet, lifestyle, and health information on U.S. adults over many decades. During the follow-up period, which spanned decades, 278 participants developed gastric cancer.

“This is the first study to demonstrate an association between sugar-sweetened beverage intake and gastric cancer in a U.S. population,” said senior author Andrew T. Chang, MD, a gastroenterologist and epidemiologist with the Mass General Brigham Cancer Institute. Gastric cancer is the fifth leading cause of cancer death worldwide, but we’ve known little about how diet might contribute to this cancer.

After accounting for other potential risk factors, participants who consumed at least one serving of a sugar-sweetened beverage per day had a 2.45 times higher risk of gastric cancer compared with those who consumed less than one serving per month. The association was observed in both women and men. Higher total fructose intake (fructose is the main sweetener in these drinks) was also associated with greater gastric cancer incidence.

For instance, among women in the Nurses’ Health Study, consuming more than one such beverage per day was associated with a 3.04-fold higher risk of gastric cancer, compared to those who rarely consumed sugar-sweetened beverages. In contrast, after controlling for other factors, higher consumption of artificially sweetened beverages wasn’t associated with a higher incidence of gastric cancer.

Researchers defined sugar-sweetened beverages as carbonated beverages, punch, lemonade, and sports drinks, while artificially sweetened beverages were defined as low-calorie carbonated beverages.

Because Helicobacter pylori infection increases the risk of gastric cancer, researchers also assessed H. pylori status in a subset of 940 participants. While just over one-third of these participants had evidence of H. pylori infection, there was no association between sugar-sweetened beverage consumption and infection.

Limitations of the study include its observational design. It is possible that higher rates of gastric cancer in people who consumed more sugar-sweetened beverages were related to other factors. The study had limited information on H. pylori status and family history of gastric cancer. In addition, participants were predominantly white, limiting the researchers’ ability to examine differences across racial and ethnic groups.

However, the large study population and detailed health and lifestyle information gathered over many years allowed the researchers to control for many other possible potential risk factors for gastric cancer.

The researchers say further studies are needed to confirm the findings and investigate why these beverages might increase gastric cancer risk.

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Embedding Regulatory Strategy in Cell and Gene Therapy Development

Edusehat — Wed, 19 Aug 2026 05:10:09 +0700

Jeffrey Mocny, PhD

VP Scientific Leadership
Rose BioSolutions

Felicia Irons, BS, CSSGB

Global CDMO Regulatory Affairs & Compliance Lead
Rose BioSolutions

Broadcast Date: Thursday, September 17, 2026

Time: 08:00 PDT, 11:00 EDT, 17:00 CET

For cell and gene therapy programs, early development decisions can have a significant impact on regulatory success, development timelines, and overall program risk. Embedding regulatory strategy from the outset helps developers identify critical challenges early, align development activities with product-specific requirements, and build a stronger foundation for future clinical and commercial milestones.

In this GEN webinar, experts from Rose BioSolutions, a CDMO and Cell Solutions organization formed from Charles River Laboratories’ businesses, will discuss how integrating regulatory strategy at the earliest stages of development can reduce overall risk and improve program outcomes. Attendees will learn how a well-defined Target Product Profile (TPP) can guide decision-making across the drug development lifecycle, and help teams prioritize activities, anticipate regulatory expectations, and develop a fit-for-purpose CGT manufacturing and testing strategy. Presenters will use real-world case studies to highlight common cell and gene therapy product development challenges and practical approaches to addressing issues such as sterility testing, interim product release, and device compatibility at patient infusion.

Key Takeaways

How to implement a regulatory strategy during program scoping to align development activities with product-specific risks and compliance expectations

Why a Target Product Profile (TPP) should be established at the beginning of development and how it guides strategy, critical quality requirements, and risk mitigation plans

How early identification of product-specific challenges can help minimize unnecessary regulatory information requests, avoid manufacturing delays, and support timely patient treatment

Practical strategies for developing a more efficient, inspection-ready path from early development through clinical manufacturing and commercialization

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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Exercise-Boosting Molecule May Help Improve Muscle Health in Type 2 Diabetes

Edusehat — Wed, 19 Aug 2026 01:35:10 +0700

An international research team headed by scientists at the University of Leeds discovered that a key molecule released during exercise strengthens muscles, improves exercise performance, and could be a therapeutic treatment for muscle damage caused by type 2 diabetes. Their collective results of the study, involving experiments in mice, and human plasma analyses, identified the muscle-derived metabolite beta-aminoisobutyric acid (BAIBA) as a central regulator of how muscles remodel, strengthen, and improve their endurance in response to exercise training. The findings further showed that it’s the L-enantiomer, L-BAIBA, that acts as the primary mediator of muscular effects.

Research lead Lee Roberts, PhD, professor of molecular physiology and metabolism at the University of Leeds, said, “Our research identifies L-BAIBA as an exercise-induced signal that helps muscles adapt, improve their metabolic capacity and ability to contract, and ultimately enhances physical performance. This gives us exciting new insight into how exercise benefits the body and highlights a promising target for future therapies aimed at preserving muscle function in chronic conditions such as diabetes.”

Roberts is senior and corresponding author of the team’s published paper in Nature Communications, titled “The metabokine β-aminoisobutyric acid mediates exercise performance and skeletal muscle adaptation through a PGC1α-BAIBA-PPARδ axis.” In their paper, the team noted, “It may be that BAIBA can offer a novel therapy for other diseases with a skeletal muscle atrophy or dysfunction component, such as chronic heart failure, cancer cachexia, or sarcopenia. Conducting intervention studies using BAIBA to target muscle function, exercise capacity, and quality of life in these clinical populations will be important for future translation.”

Exercise training involves multiple bouts of activity that challenge whole-body physiology, driving adaptations across cells, tissues, and organs, the author wrote. “Exercise is also an effective intervention for the prevention or treatment of a range of diseases and pathological risk factors, including obesity, type 2 diabetes (T2D), and cardiovascular disease, and may facilitate improvements in age-related reduction in quality of life.”

But how exercise impacts the body’s ability to respond to challenges is not fully understood. The team continued, “Elucidating the fundamental molecular mechanisms and intra- and inter-organ signals through which exercise mediates systemic adaptations, driving beneficial effects, holds potential for the identification of both therapeutic targets and new strategies to treat a range of diseases, including T2D.”

Given the health benefits of exercise, Roberts and colleagues set out to discover whether this mechanism could hold the potential for treatment for chronic conditions such as type 2 diabetes. L-BAIBA has been studied in the past, and Roberts carried out research in 2014, which showed that it had beneficial effects on metabolism, boosting the fat-burning effects of exercise. It can now be purchased as a nutritional supplement.

The newly reported study has found that it triggers muscles to respond and change, linking exercise to beneficial alterations in muscle structure and metabolism. “Circulating L-BAIBA associated with aerobic fitness in humans,” the team noted in summary. “The current study identified that in mice, BAIBA supplementation in drinking water increases skeletal muscle mitochondrial number and functional capacity, improves muscle contractile function, and drives phenotypic remodelling similar to aerobic exercise training. We find that BAIBA supplementation in mice, when combined with exercise training through wheel running, leads to an increased adaptive response in skeletal muscle.”

L-BAIBA also protected muscle from damage that occurs in type 2 diabetes, which can limit a person’s quality of life, such as loss of muscle and strength, and impaired metabolism. Improving muscle resistance to fatigue could be transformative, as exercise can help to manage diabetes. The discovery that BAIBA not only mimics the adaptive response of muscle to exercise but can also contribute to translating the benefits of exercise to improved muscle and systemic function may have important clinical implications, the investigators continued. “In our study, BAIBA treatment exhibited therapeutic potential by improving the free-wheel running capacity and skeletal muscle molecular, metabolic, and functional phenotype in a mouse model of obesity and T2D-induced skeletal muscle dysfunction.”

Anna Morris, assistant director of research strategy and partnership at Diabetes UK, said, “Exercise plays an important role in managing type 2 diabetes, but for many people, muscle weakness and fatigue can make staying active a real challenge. This research helps us better understand how muscles adapt to exercise and identifies a potential new target for improving muscle health. While these findings are still at an early stage, they could help shape future approaches that support people with type 2 diabetes to remain active and live healthier, longer lives.”

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Albumin-Fused Antibodies May Reduce Fetal Exposure to Therapeutics

Edusehat — Tue, 18 Aug 2026 07:40:07 +0700

Researchers at the University of Oslo and Oslo University Hospital, together with national and international collaborators, have reported on the results of preclinical research that could reshape the future design of biologic medicines that can be delivered during pregnancy with limited fetal exposure.

Led by Jan Terje Andersen, PhD, at the University of Oslo and Oslo University Hospital, the team’s studies in mice and in ex vivo human tissues found that the placenta distinguishes between antibodies and albumin. The scientists showed that although the neonatal Fc receptor binds both immunoglobulin G (IgG) antibodies and albumin, FcRn expressed in the placenta selectively transports IgG to the fetus, while largely excluding albumin. Their experiments demonstrated that fusing IgG antibodies to albumin minimized their transport across the placenta and prevented adverse effects in offspring in mice.

Andersen said, “Rather than asking whether existing biologic medicines are safe to use during pregnancy, our findings show that we can now design them differently. The placenta selectively transfers protective IgG antibodies while preventing albumin from crossing. By understanding this, we now have the opportunity to develop a new generation of biologic medicines that combine long-lasting efficacy with improved safety during pregnancy. This study shows how fundamental discoveries in biology can directly inspire the design of better medicines.”

Andersen is corresponding author of the team’s published paper in Science Immunology, titled “Fusion of IgG antibodies to albumin inhibits transport across the placenta,” in which they concluded, “These findings identify albumin as an attractive fusion partner for biologics intended to minimize fetal exposure during pregnancy.”

Albumin and IgG antibodies are the most prevalent soluble proteins in blood, the team explained. “Whereas IgG is pivotal in the fight against infectious diseases, albumin transports a plethora of insoluble ligands, such as fatty acids, hormones, and waste products, for delivery to tissues and organs.”

IgG-based monoclonal antibodies are among the fastest-growing class of biologics that are being used to treat a range of acute and chronic diseases, the team noted, and are effective therapies for cancer, autoimmune diseases, and migraine. “However, they are actively transported across the placenta by the neonatal Fc receptor (FcRn), limiting their use during pregnancy.” Evidence that supports safe use of such therapeutics during pregnancy remains limited.

For their studies, the team focused on albumin, a transport protein that also has a long half-life and binds FcRn at a different site to IgG. The researchers showed that FcRn transferred maternal IgG, but not albumin, across the placenta and into fetal pups in mice. To uncover the mechanism, the researchers combined studies in conventional and genetically humanized mouse models with an advanced ex vivo human placental perfusion system using placentas donated immediately after childbirth. Across all models, the findings were strikingly consistent: IgG antibodies were transferred efficiently, whereas albumin was not.

The researchers then took advantage of the discovery to make a platform for engineering next-generation biologics. They found that fusion of albumin to therapeutic IgG antibodies produced biologics with both FcRn-mediated long plasma half-life and substantially reduced placental transport. An even greater effect was achieved by fusing antibody fragments to an engineered albumin variant (QMP) with optimized human FcRn binding, demonstrating that both reduced placental transfer and long plasma half-life can be tuned through rational protein design.

The concept was validated in human placental tissue and disease models. In a mouse model of fetal and neonatal alloimmune thrombocytopenia (FNAIT)—a potentially life-threatening pregnancy complication in which maternal antibodies attack fetal platelets—the engineered antibodies resulted in substantially reduced fetal exposure and associated adverse effects in the offspring.

“This is first and foremost a discovery of how the placenta works,” Andersen said. “For decades, we have known that FcRn binds both IgG and albumin, yet only IgG reaches the fetus by an FcRn-dependent mechanism. We show that the placenta has a remarkable ability to distinguish between these two soluble proteins, revealing a level of biological selectivity that was previously unrecognized.”

The findings address a rapidly growing unmet need. While monoclonal IgG antibodies are increasingly used to treat chronic diseases that affect women during their reproductive years, decisions concerning treatment during pregnancy often involve balancing the benefits of disease control against risks to the developing fetus. The authors propose that further research is needed to understand the mechanisms that restrict albumin transport, which could inform more tailored therapeutics with varied fetal exposure profiles.

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Human Cell Atlas at 10: A Decade of Discovery and the Road to HCA 2.0

Edusehat — Tue, 18 Aug 2026 04:05:13 +0700

The Human Cell Atlas (HCA) was co-founded in 2016 by Aviv Regev, PhD, currently the head, executive vice president of research and early development at Genentech and Sarah Teichmann, FMedSci FRS, professor at the University of Cambridge and vice president translational research at GSK.

This year, the group celebrated its 10-year anniversary, at the annual meeting in June, held in Boston. The first day was focused on the successes of the past decade. The second day looked ahead to the future, and the impact the HCA will make on research and drug discovery. GEN spoke with Holger Heyn, PhD, ICREA professor at the Centro Nacional de Análisis Genómico (CNAG) and co-founder of Omniscope about the meeting and what the next 10 years may bring. Heyn has been with the HCA since (almost) the beginning and currently serves as the co-chair of the Standards and Technologies working group, co-chair of the Industry Partnership Program, an organizing committee member, and a member of the task force to design HCA 2.0.

LeMieux: Let’s start with the meeting, which covered a lot of ground. Can you share what the main takeaways were?

Heyn: The first day was about the past—the last 10 years. The second day was about the future. And the third day was reserved for working group meetings.

Holger Heyn, PhD
Co-founder, Omniscope

Talking about the past, the first day celebrated the completion of most organ and tissue atlases of the HCA. The HCA activities are broken down into 18 different biological networks (or bionetworks): lung, liver, heart, etc. All of those will have either published or submitted their atlases in 2026.

After 10 years, we have come a long way and we delivered what we set out to do, which was the generation of the HCA, single-cell resolved, for all main tissue and organ systems. That is exactly the mission of the HCA: completely grassroots—to deliver representative atlases of tissues and organs with openly available data. In total, we have already 450 publications, that have been cited 100,000 times, and a global representation with almost 4,000 members from a hundred countries. Recently, the representation from Africa and South America has grown a lot. The HCA is now a really global effort.

LeMieux: On the point of increasing the globalization of the HCA, how is the technology being democratized to reach more areas of the world?

Heyn: This has been approached in two separate ways. One was when my group published a method to cryopreserve cells, about eight years ago. It allowed researchers to put cells in the freezer and then thaw them to run them anywhere. The idea was to centralize data processing, so each hospital or each center does not need to have a single-cell instrument. More recently, we have been able to use formalin fixation, which allows researchers to fix and sequence later. Both technologies offer flexibility in terms of where the data is generated and to disconnect the sampling from the processing time. Being able to collect and store, then process later, makes single-cell analysis much more accessible.

In addition, there has been a drop of cost per cell. Although the assay is still costly, over the years, people get more cells per dollar, allowing the generation of scalable atlases. Now, most of these atlases are represented by millions of cells. Ten years ago, there was no way to afford that.

LeMieux: Now let’s move into the future. The first day of the meeting was called HCA 1.0. But now let’s talk about day two, or HCA 2.0.

Heyn: Day two was all about the future roadmap and where the project is going. Aviv Regev highlighted this in her talk. Even though the HCA is now split into phase one and phase two, the that was published 10 years ago in eLife laid everything out clearly. There are the two pillars, single-cell and spatial, to build atlases. Single-cell technology was available earlier and only now is spatial becoming scalable. The initial plan was always to do a single-cell and the spatial cell atlas—to profile cells in their tissue context. But technology had to catch up.

For HCA 2.0, we now have a five-year time frame where we scale the atlas in multiple different directions. One is the already mentioned spatially resolved atlases: we will use spatial methods, very advanced capture-based sequencing methods, and full transcriptome imaging-based methods. The focus of HCA 2.0 is to move away from cells in dissociation and toward generating spatial data at scale. With this, we can use the single cell references that we have generated in the past and map cells back into their natural context.

The second focus is the global representation. One area where this goal has been very nicely implemented is in Asia. HCA Asia is one of the largest, most active communities. In HCA 2.0, we aim at better diversity, representing not only blood, which is the best represented sample type right now, but also other major tissue types.

The third, and very important one, is opening from a healthy reference atlas to include a disease focus. We had a discussion with the HCA community at the meeting where to focus first. We asked, what are global disease burdens, what are local efforts we want to support, and what samples are available?

Disease also brings us closer to collaborating with pharma partners. We are in discussion with multiple partners to make our atlas generation efforts useful for diagnostics and drug development. For increasing impact, you eventually need commercialization and innovation on top of that. Pharma partners are already developing drugs using our healthy atlases. Now it will be crucial to align with these collaborators for diseased atlas efforts as well.

Fourth, imagine a Venn diagram with the single-cell atlases, spatial, disease, and genetic diversity. And now picture in the middle a small circle as a union: these are the foundation models. The HCA is generating a unique resource to build foundation models. Most foundation models to date are already built on single cells from our atlases. However, many recent initiatives are also building foundation models based on spatial data, in order to have the tissue context represented. To make these foundation models useful, you have to train them on a specific task. And this is where the disease context becomes important. With the healthy atlases, you have the representation of cellular biology of the human system. Fine tuning models can then be based on predictive tasks, such as drug target identification, prediction of therapy outcome, and patient stratification. This brings out the real value of those models, to make them actionable and to use them for predictive tasks.

LeMieux: It seems like every time I turn around there is a new virtual cell project, whether it’s being done by CZI or Tahoe or another group. So how do all of these come together with HCA?

Heyn: We are a virtual cell community. HCA is in active discussion with all of the stakeholders. We all have the same goal to build a virtual cell that is representative of biology and diseases. We all have very different approaches. The approach that the HCA is offering is that we use spatial technology to put cells back in their natural context and then to use such data to train models on the natural perturbation of interacting cells. But everyone has their own niche. There is no right or wrong for now. We are following strategies that will be complementary and synergistic in the future.

LeMieux: Moving toward disease, where do you think that the HCA will start to make inroads?

Heyn: Today it is still hard to say. We did some analysis, asking what our community chooses as diseases of interest? We also talked to the main stakeholders to make those atlases impactful. We are basically in the landscaping phase to see what disease areas are suitable for pilot flagship projects on specific tissues and diseases. But we already want to connect this initial phase to the end goal. Therefore, we need the different stakeholders on board to make informed decisions together with all parties.

There are two main objectives for a pharma company when using the atlases. One is to find new drug targets. The second is to predict toxicity to have on target and not off target effects. To do that, they need both a healthy reference and a disease cohort. Traditionally, you would have done a differential expression analysis to find something in the disease cohort that is missing in the healthy reference. Now you can actually map that out spatially at cellular resolution.

LeMieux: How much of a spatial focus do you think there will be in a decade from now?

Heyn: Over the next five years, there will be a heavy focus on spatial data generation. There will be large-scale, flagship projects using spatial technologies. Single-cell will not go away though. For immune related references, diversity, and certain diseases, you still need single-cell analysis. Spatial will be the driver though toward more clinical applications. Spatial is the way forward to really look at clinical cohorts and diseased tissue at scale and for digital pathology implementations.

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Rewriting Disease: Oligonucleotides Take Aim at the Untreatable

Edusehat — Tue, 18 Aug 2026 04:05:11 +0700

For decades, drug discovery has revolved around a familiar challenge: finding ways to modulate proteins that drive disease. Small molecules and biologics have transformed medicine, yet vast portions of the human genome remain stubbornly out of reach. Many disease-causing targets are considered undruggable, leaving researchers with few options for intervention, but oligonucleotide therapeutics are changing that equation.

Built from short, synthetic strands of DNA or RNA, oligonucleotides offer a fundamentally different way to influence disease biology. Rather than targeting proteins after they are produced, these molecules act upstream, engaging RNA and gene-regulatory pathways to alter protein expression with remarkable precision and duration. Their growing success has transformed them from a niche modality into one of the most promising frontiers in therapeutic discovery.

Today, advances in artificial intelligence (AI), multiomics analysis, human genetics, and disease modeling are accelerating the identification of new oligonucleotide targets. Across neurodegeneration, fibrosis, metabolic disease, and rare genetic disorders, companies are using these technologies to uncover biological mechanisms that were previously hidden. The result is a rapidly expanding therapeutic landscape where researchers are no longer limited by conventional notions of druggability.

A new class of precision medicines

Oligonucleotides are engineered sequences of nucleic acids, typically ranging from 10 to 50 nucleotides in length. Because they can be designed to recognize specific genetic sequences, they provide highly selective control over biological processes. Chemical modifications give sustained effects with patients receiving maintenance doses every three to 12 months.

As explained by Evotec: “Unlike gene therapy, oligonucleotide drugs targeting RNA, such as small interfering RNA (siRNA) and antisense oligonucleotides (ASOs) have a transient effect in the body, and do not permanently modify the patients’ genome. Sequence-based targeting of RNA uses synthetically produced, matching stretches of oligonucleotides to bind with exquisite precision to a specific RNA target, causing subsequent changes in protein expression, either by altering splicing of the immature RNA or causing rapid turnover of that RNA before a protein can be made.” This temporary and programmable nature has made oligonucleotides an attractive therapeutic platform.

The field has expanded far beyond simple gene silencing. Modern oligonucleotide approaches can redirect RNA splicing, edit RNA transcripts, enhance protein expression, block RNA-binding proteins, and even target proteins directly through aptamers, which are structured nucleic acid molecules capable of binding proteins with antibody-like affinity.

Overall, oligonucleotides are increasingly viewed not merely as another therapeutic modality, but as a platform capable of unlocking entirely new classes of targets.

Guiding drug discovery with microRNA

One area generating significant excitement involves microRNAs, which David Salzman, PhD, CEO of Gatehouse Bio, describes as “master regulators of biological pathways.” Rather than targeting a single gene, a microRNA can control entire biological programs, including cellular stress responses, inflammation, fibrosis, and protein production. Because these pathways are often disrupted across multiple diseases, microRNAs offer an opportunity to intervene at key regulatory nodes, particularly in central nervous system (CNS) disorders where distinct diseases frequently share underlying mechanisms.

Beyond their regulatory functions, microRNAs offer another advantage as druggable targets, Salzman explained. “They have dual roles as both biomarkers and drug targets.”

Researchers can inhibit harmful microRNAs using ASOs or restore beneficial microRNAs with synthetic mimetics, creating a direct bridge between diagnosis and therapeutic intervention.

These images from lung slices grown ex vivo from healthy tissue and patients with idiopathic pulmonary fibrosis show the distribution in lung tissue of the microRNA isomer, GHB1589, which localizes to disease-driving fibroblasts and alveolar cells. [Gatehouse Bio]

AI has become a powerful tool for identifying clinically relevant oligonucleotide targets. While much of the industry focuses on molecule design, Gatehouse Bio uses AI to identify patient subgroups most likely to respond to specific therapies. “Even the best AI-designed molecules will fail if they’re tested in the wrong patient population. We use microRNAs to tell us which patients to treat and also how to treat them.” Salzman says.

The approach reflects growing recognition that many diseases are more heterogeneous than clinical diagnoses suggest. In neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and vascular dementia, patients with similar symptoms might have different molecular drivers. By analyzing thousands of microRNA measurements alongside clinical outcomes, pathology, imaging, and other molecular datasets, AI can uncover patterns that traditional methods often miss.

“Ultimately, our goal is not simply to identify biomarkers,” Salzman said. “We use AI to connect microRNAs to disease biology, patient subgroups, and therapeutic opportunities.” The strategy moves researchers beyond correlation and toward causal, mechanistic understandings, a crucial step in validating therapeutic targets.

Multiomics and biomarkers

Although AI can identify disease-associated signals, determining whether those signals drive disease requires additional biological context. This is where multiomics approaches have become indispensable.

By integrating microRNA sequencing with transcriptomics, proteomics, genomics, pathology information, and clinical-outcome data, researchers can reconstruct disease networks. If a microRNA appears altered in patients with a specific outcome or pathology, investigators can assess whether related genes and proteins change in a coordinated manner. This helps distinguish passengers from drivers when prioritizing therapeutic targets.

“The power of multiomics is that it allows us to connect microRNAs to disease biology, patient subgroups, and therapeutic opportunities with much greater confidence than any single data type alone,” Salzman noted.

Gatehouse Bio’s fibrosis program illustrates how oligonucleotide discovery combines AI, multiomics, patient stratification, and experimental validation. In fibrotic tissues and blood samples from patients with idiopathic pulmonary fibrosis, the company identified reduced levels of a microRNA called miR-92.

Initially, the finding appeared to be only a biomarker. However, integrated analyses revealed something more significant. Reduced miR-92 activity mapped to fibrosis-related pathways involving TGF-beta signaling, WNT signaling, extracellular matrix remodeling, and integrin biology. Patients with lower circulating miR-92 levels experienced worse outcomes, and experiments showed that reducing miR-92 activity worsened fibrosis, whereas restoring it produced anti-fibrotic effects. The result was GHB1589, an miR-92 mimetic.

For Gatehouse Bio, the program represents a blueprint for target discovery: identify disease-associated RNA signatures, connect them to biological mechanisms, stratify patients, validate causality, and translate the findings into therapeutics.

Recreating disease in human neurons

Another major challenge in oligonucleotide discovery involves generating disease models that accurately reflect human pathology. AcuraStem has addressed this problem by using patient-derived induced pluripotent stem cells (iPSCs) that are directly converted into induced motor neurons. Unlike many traditional laboratory models, these neurons preserve aging characteristics and disease-specific pathology from the original donor.

According to AcuraStem’s CEO Sam Alworth these patient-derived models have been instrumental in uncovering novel therapeutic targets. “AcuraStem uses transcription factor-mediated lineage conversion to reprogram patient-derived iPSCs into induced motor neurons that retain the aging and pathological markers of the donor,” Alworth explained. The company’s iNeuroRx platform integrates these disease models with ASO design, screening, and pharmacology tools, creating a comprehensive discovery engine.

In patients with the motor-neuron disease amyotrophic lateral sclerosis (ALS), antisense oligonucleotides might lead to new treatments. [Kateryna Kon/Science Photo Library/Getty Images]

AcuraStem’s work centers on TDP-43, a protein whose dysfunction is implicated in the majority of amyotrophic lateral sclerosis (ALS) cases and several forms of dementia. “TDP-43 pathology is a crucial contributor to neurodegeneration,” said Marcel van der Brug, PhD, AcuraStem’s CSO. Normally, TDP-43 regulates RNA processing. In disease, however, it becomes depleted from the nucleus, leading to widespread RNA dysregulation and altered gene expression.

AcuraStem’s disease models reproduce TDP-43 pathology without requiring artificial stressors or genetic manipulation—a capability the company believes is unique. This platform has enabled the discovery of several therapeutic targets, including the gene SYF2, which appears capable of restoring TDP-43 localization and function.

The work highlights a broader trend within oligonucleotide discovery: targeting upstream molecular mechanisms rather than downstream symptoms. “RNA dysregulation, as a direct and measurable consequence of TDP-43 dysfunction, is a target that can be modulated by technologies such as antisense oligonucleotides,” Alworth said.

An expanding oligonucleotide toolbox

While some companies begin with disease biology, others start with genetics. Aperture Therapeutics, for example, is building its discovery strategy around naturally occurring protective human genetic variants. The company’s platform analyzes large-scale genomic and clinical datasets to identify resilience mechanisms that protect individuals from neurodegenerative disease. These insights are then translated into oligonucleotide strategies, including gene-expression modulation and splice-switching approaches. By focusing on genetically validated targets, Aperture aims to reduce development risk and improve the probability of clinical success. The strategy reflects a growing industry consensus: human genetics provides one of the strongest forms of biological validation available.

The oligonucleotide field is also expanding beyond traditional antisense and RNA interference approaches. As an example, Opprtna Therapeutics is developing aptamer-based therapeutics for CNS diseases. Unlike many aptamers that primarily serve delivery functions, the company is designing molecules that directly influence disease biology. Using structured DNA molecules capable of binding proteins associated with RNA and DNA regulation, Opprtna hopes to target pathways that have traditionally been inaccessible to conventional drug modalities. The approach underscores how oligonucleotide chemistry itself continues to evolve, creating entirely new therapeutic opportunities.

Meanwhile, Riboway Therapeutics is pursuing RNA-targeting technologies capable of increasing, decreasing, activating, or inhibiting protein expression depending on the therapeutic need. The company’s AI-driven platform focuses on decoding RNA regulation to identify previously inaccessible intervention points.

In many ways, oligonucleotides are redefining what drug discovery can be. Rather than asking whether a target is druggable, researchers are increasingly asking how RNA biology can be leveraged to reach it. That shift may ultimately prove to be one of the most important developments in modern therapeutics.

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The Microbiome Field Enters Its Next Chapter

Edusehat — Tue, 18 Aug 2026 04:05:10 +0700

Sebastião van Uden, PhD
CEO, Bac3Gel

When scientists at Bac³Gel hear from potential customers, they typically express a mixture of frustration, curiosity, and skepticism, Sebastião van Uden, PhD, the company’s CEO, tells GEN. Usually, they approach the company after their own culture efforts have failed to produce consistent results, or they are on the hunt for a solution for bacteria “that is hard to grow,” or they are concerned about the costs and duration of current bioreactor experiments.

These are issues that the company believes it can address with its proprietary biomaterial platform, which uses a synthetic, organic gel, made from food-grade ingredients, to reproduce the structural and chemical properties of human mucus. The gel is designed to support the growth of highly complex microbial communities in the laboratory setting and is used for applications in academia, biotech, and pharma research and nutrition. Importantly, the gel can be customized to grow microbial communities that are found in different environments including the gut, lung, skin, and vagina.

The company claims that its technology addresses key industry pain points, including hard-to-culture bacteria as well as the high cost and low throughput of existing bioreactors. According to numbers shared with GEN, the company’s gel can enable growth of up to 92% of a microbiome sample’s species, which is a significant improvement over the 2–3% that is achievable with standard methods.

Bac³Gel is adopting a three-step market strategy that starts with research tools, moving into the food supplement space in partnership with existing corporations, and finally developing live biotherapeutics. It is the most prudent path for the early-stage startup, van Uden says. “Our business strategy lies in [moving] step-by-step, increasing risk while we expand in markets.”

Like most biotech companies, Bac³Gel is exploring ways to deploy artificial intelligence (AI) that make sense for its portfolio. One avenue is to leverage the large datasets that it has generated to create digital twins of the human body that allow it to test how particular strains affect the existing microbiome population or how particular probiotics impact people in different geographies with unique diets. “This is something very interesting, and we started working on it,” van Uden says. “I think AI has a place here in the microbiome.”

Prebiotic substrates for phenotyping

As consumables product manager at Biolog, Juan Sanchez, has a good overview of the company’s microbiome portfolio. In fact, “I actually introduced our first microbiome target product,” he tells GEN. “We had other products that could be used for this type of research, but this one is really for the gut microbiome.” Specifically, these are three different plates with prebiotic substrates that at their core are designed to help scientists who are studying human or animal gut microbiomes simplify their work.

Juan Sanchez
Product Manager, Biolog

The so-called PreBioM line of microplates is designed for phenotyping gut microbiomes by testing their ability to metabolize various prebiotic substrates. Plates come pre-loaded with 90 different prebiotic substrates, removing the need for scientists to handle the plate formulation and testing on their own. And though the product was designed for the gut microbiome initially, it is applicable to other mammalian microbiomes such as oral and skin. “We remove all of the trouble from the researcher having to guess which substrates are relevant, what concentrations should these substrates be at,” Sanchez says. “We did all of that work for them.”

Primary customers for PreBioM plates are scientists in universities, university hospitals, and pharmaceutical companies that are developing live biotherapeutics. “The live biotherapeutic space is really one direction where the microbiome research has been evolving to,” Sanchez says. “Before it was just making probiotic pills, but now they are a lot more specialized.”

And that brings up a new challenge: the need to standardize not just the final product but also what goes into producing the product. This is another area where Biolog stands out. “We’ve actually been making media for growing the bacteria that will be used for the live biotherapeutic in bioprocess bags,” he tells GEN. This product, TruPRAS, is the only commercial media manufactured under true pre-reduced, anaerobically sterilized conditions, according to Biolog.

The evolving live biotherapeutics market is also driving greater interest in customized media offerings, Sanchez notes. In fact, it is “one of the bigger trends for live biotherapeutics that I’ve seen coming up,” he says.

Building immune tolerance

“The big bold vision that we’ve always had at Siolta Therapeutics is that we can actually stop diseases before they start,” Nikole Kimes, PhD, tells GEN. Kimes co-founded the company with Susan Lynch, PhD, a professor in the department of medicine and director of the Colitis and Crohn’s Disease Microbiome Research Core at the University of California, San Francisco (UCSF).

Nikole Kimes, PhD
Co-founder and CEO
Siolta Therapeutics

Kimes, who serves as the company’s CEO, joined Lynch’s lab at UCSF back in 2014 at a time when scientists were learning a lot about how allergic disease develops. “What we kept seeing was that early life risk factors were the most highly associated with disease development downstream,” she says. “We started to look into the gut microbiome early in life” for “signatures that were consistent across kids who went on to develop either atopic dermatitis, food allergy, asthma [or] any of the IgE-mediated atopic diseases.” Their idea was to create a cocktail of beneficial microbes that could be used to treat infants whose signatures suggested they were at greater risk of disease. In theory, by replenishing the gut microbiome, they would be able to support immune tolerance rather than immune inflammation.

Their research led them to examine the microbial communities of infants in different cohort studies globally, looking for signals that indicated they lacked beneficial microbes. “What we’re really interested in is the functionality, what role are they playing?” Kimes explains. This is an important point because two people might have drastically different strains in their microbiomes that have functionally similar roles. They also looked at the functional capacity of the microbes that conferred healthy benefits to their hosts. This way, they identified a subset of potential candidates for the cocktail that would have the greatest efficacy and be easy to manufacture.

At Siolta Therapeutics, scientists are developing a cocktail of beneficial microbes gleaned from the gut microbiome that could improve outcomes for babies at risk of developing IgE-mediated atopic disease. [Siolta Therapeutics]

The team at Siolta has spent the last nine years building out its Precision Symbiotics Platform^TM, which includes all the computational tools, biobanks, and assays needed to develop its therapeutics. “We started the company out of necessity,” Kimes says. At the time, “there was nobody out there developing preventative drugs from a novel modality” because “it was considered too challenging to do. We thought it was too much of an opportunity to not try.”

The company has now completed a Phase II proof-of-concept clinical trial of its live biotherapeutic in 238 newborn infants with a familial history of allergic disease. “We have great data showing that this cocktail of organisms, when given over the first year of life, is reducing atopic dermatitis and food allergy at one year of age.” By age two, “we actually see reductions across atopic dermatitis, food allergy, and even asthma.”

An edge in the obesity market

For years, the ketogenic diet has been effectively used to treat drug-resistant epilepsy and other metabolic conditions by pushing the body to burn fat as an energy source. Bloom Science is turning to the microbiome to develop therapeutics for neurological and metabolic disorders that work in much the same way without dietary changes. “Our core technology and program is actually based on what we like to say is reverse engineering the ketogenic diet,” says Chris Reyes, the company’s CEO and founder. “We looked at the ketogenic diet as our reference point” and asked, “How does it work? What are the key bioactive molecules that have been shown or are being focused on to explain this effect?”

Christopher Reyes, PhD
Founder, CEO, Bloom Science

Underpinning the company’s therapeutic pipeline is a proprietary platform dubbed IrisRx. Leveraging this platform, it has developed live biotherapeutics that treat obesity, Dravet syndrome, Alzheimer’s disease, and amyotrophic lateral sclerosis. Its lead candidate, BL-001, is currently in Phase Ib testing for obesity. Early signals indicate that the biotherapeutic is well tolerated with no serious adverse events, and patients with overweight had statistically significant placebo-adjusted weight loss.

“We took a very traditional drug discovery and development approach,” Reyes explains to GEN. After identifying strains of bacteria that could replicate the diet, “we created a library of strains within these species and other species, and we screened them like we would an antibody library or a small molecule library looking for optimal functional fit.” The company is also working on engineering specific improvements to some strains to further optimize them for different indications.

If Bloom’s first candidate is successful, BL-001 could become an alternative to GLP-1s. In fact, Reyes believes that even with the success of GLP-1s, the population of people with obesity in the United States is still underserved. “Maybe about a third of the obese population is actually on or has been on GLP-1s,” meaning “two-thirds are not.” He attributes this partly to hesitation among this population due to factors like drug cost and tolerability.

Bloom Science is mining the microbiome to develop live biotherapeutics for neurological and metabolic disorders. Its lead candidate for obesity, currently in Phase Ib testing, is targeting one of healthcare’s largest markets. [Bloom Science]

“What we’ve seen is that there’s a huge preference for therapies that have steady weight loss with fewer side effects than rapid weight loss with higher side effects,” he continues. And though it is still in the testing, the Phase I results for its lead candidate are very promising. “We think we’re going to be competitive on the efficacy side with other oral obesity drugs” and “we believe we’ll have a much more tolerable profile.” Also, “we have very preliminary data to suggest that we might be able to identify a responder class based on their starting baseline metabolic state,” he adds, stressing that this research is still in its very early stages.

Macrophages to aid immunotherapies

The constant interaction between the microbiome and the immune system is of particular interest to scientists at Exeliom Biosciences. As Benjamin Hadida, the company’s co-founder and CEO, explains it, “we are trying to modulate the immune system by using some components that come from the microbiome.”

The French biotech company is developing a single asset, dubbed EXL01, that is delivered orally and is currently in Phase II testing in six different clinical studies across three therapeutic areas, Hadida tells GEN. EXL01, the company’s immunomodulator, is a strain of the bacterium Faecalibacterium prausnitzii, which is prevalent in healthy human guts. It is also one of the first to be impacted in systemic inflammation seen with conditions like inflammatory bowel disease or in patients undergoing chemotherapy.

Four of their studies are focused on oncology where the asset is being evaluated in combination with several approved checkpoint inhibitors in gastric cancer, renal cell carcinoma, hepatocellular carcinoma, and non-small cell lung cancer. Essentially, “we’re developing an immunomodulator that acts on the innate part of the immune system,” he explains. “We activate macrophages so that we can make patients more able to respond to immunotherapies.”

The company is positioning EXL01 as an add-on to existing immunotherapies since it targets a different type of immune cell. For patients on these treatments, whether that is in oncology or immune disease, “one of the key issues is the durability of the effect. We work on macrophages so that we keep the response to these treatments sustainable,” he says.

“We took it to the lab and tested it in some models trying to identify the mechanism at play,” he continues. It turns out that this bacterium has a unique component on its membrane that makes it a specific agonist of a key receptor in macrophages. Upon binding, it activates the NOD₂-CARD₉ pathway that reprograms the macrophages to resist the immunosuppressive tumor environment and keep the T cell pool in shape.

Industry skepticism toward microbiome therapeutics in the last several years has created both challenges and opportunities for companies like Exeliom. “In the oncology world, [there have been a] number of Phase III failures that we’ve seen when [pharmas] try to combine things with checkpoint inhibitors to increase the efficacy,” Hadida says. “Some of them were able to increase the response rate, but they all failed in terms of progression-free survival. What’s interesting is when you look at all these strategies, they are all focusing on the adaptive immune system. None of them tried to act on the innate immune system.” It is a common blind spot that Exeliom is targeting. “People are maybe a bit more open [to] new strategies that look somewhere else,” Hadida says.

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Malaria Mosquito Bites Turned Into Immune Boosters in New Chemovaccination Strategy

Edusehat — Tue, 18 Aug 2026 04:05:09 +0700

More than 600,000 people—predominantly pregnant women and children under the age of five—die from malaria every year. According to the World Health Organization, one child in Africa dies from malaria every two minutes.

Plasmodium parasites multiply and mature in the liver before exiting the tissue and infecting red blood cells, triggering the symptoms of malaria. Vaccines that arrest infection during the liver stage of Plasmodium infection can induce potent immunity; however, they have challenges such as complex production and repeated rounds of IV delivery in field settings. With drug resistance continuing to undermine malaria control, there is an urgent need for new strategies to stop infections.

Now, researchers developed a novel immunization strategy, chemovaccination, that paired mosquito-delivered malaria parasites with an investigational class of antimalarial drug compounds. The compounds blocked the parasite’s development at a critical stage of the malaria lifecycle, preventing disease and triggering a robust immune response that provided durable protection against malaria. Subsequent mosquito bites then reinforced this immunity and protection.

In chemovaccination, exposure to live parasites is accompanied by the administration of antimalarial drugs that arrest the parasite life cycle, preventing illness and allowing the immune system to respond to the attenuated parasite.

Now, researchers from WEHI (Melbourne, Australia) have demonstrated that chemovaccination can prime the immune system to fight malaria parasites before they cause disease, with subsequent mosquito bites acting as boosters to strengthen immunity over time—turning mosquito bites into ongoing immune boosters

This approach protected mice against malaria for the study period—a rare outcome that could inform the development of next-generation prevention strategies for one of the world’s deadliest infectious diseases. The study is the first to target malaria parasites at the late liver stage using an antimalarial drug candidate discovered by WEHI and the global biopharmaceutical company MSD (tradename of Merck & Co., Inc., Rahway, NJ).

The research is published in Science in the paper, “Chemovaccination with a late-liver-stage antimalarial induces durable immunity against malaria.”

“Using this new drug compound, we’ve found a way to turn mosquito bites—the very thing that spreads malaria—into vaccination events in mice,” said Justin Boddey, PhD, associate professor at WEHI. “This represents a shift in the way drugs could be employed to prevent malaria.”

Justin Boddey, PhD, in the dissection room of a high-containment insectary at WEHI, used for studying malaria infection. [WEHI]

“This means the parasite was stopped just before it could cause illness, while giving the immune system a fuller preview of the potential threats,” Boddey said. “The immune response generated required only a very small dose of parasites but was broader and longer-lasting than most current vaccine approaches. This is because our approach allowed parasites to amplify and then triggered both antibodies and CD8+ T cells to protect against reinfection. Importantly, this included liver‑resident memory T cells, which have the potential to respond rapidly to future infections and eliminate them before disease develops.”

The antimalarial drug candidates used in the study, WM382 and MK-7602, are both dual inhibitors of plasmepsin IX and X—two “master regulators” that are crucial for parasite survival. The drug candidates are the result of a decade-long research collaboration between WEHI and MSD.

John A. McCauley, senior director, discovery chemistry at MSD, said: “Current approaches often rely on genetically attenuated parasites, which can provide strong protection but require high doses and are difficult to produce, scale, and administer in real‑world settings. By using a drug to arrest parasites at the late liver stage, we’ve enabled the immune system to recognize a broader range of malaria antigens using a smaller parasite dose. This approach may provide a broader response against the diversity of malaria parasites seen in the real-world and go beyond what genetically attenuated laboratory strains can achieve.”

As WM382 and MK-7602 target enzymes that are highly conserved across malaria species, researchers hope this will enable their approach to provide protection against a wide range of malaria “variants” in the future—potentially allowing people in endemic areas to build immunity from natural mosquito bites over time. A long-acting injectable based on the compounds is in preclinical development.

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University of Rhode Island Wins Grant to Study Cerebral Amyloid Angiopathy Prevalent in Alzheimer’s Patients

Edusehat — Tue, 18 Aug 2026 04:05:09 +0700

A professor of pharmaceutical and biomedical sciences at the University of Rhode Island’s (URI) College of Pharmacy received a $1.5 million grant to research cerebral amyloid angiopathy (CAA), a disease prevalent in the elderly population and commonly found in patients with Alzheimer’s disease.

CAA is characterized by the abnormal buildup of amyloid-beta protein in the walls of the brain’s blood vessels. Over time, vascular amyloid accumulation can damage and ultimately weaken the vessels, increasing a person’s risk for brain bleeds, hemorrhagic stroke, and contributing to cognitive decline. The condition is difficult to accurately diagnose and lacks targeted treatment options.

“Despite the prevalence of the disease, there are no reliable early-stage biomarkers, and disease-modifying therapies currently do not exist,” said William Van Nostrand, PhD, co-executive director of the George & Anne Ryan Institute for Neuroscience at URI.

The grant is part of a $9 million five-year Transatlantic Networks of Excellence grant from the Leducq Foundation. The initiative is known as TRAFFIC (Translational Framework For Innovation in Cerebral Amyloid Angiopathy).

The goals of the TRAFFIC study are to find and clarify disease mechanisms, identify early-stage biomarkers, and reveal potential targets for disease intervention.

“Our role at URI will be to perform studies to address why CAA develops in people and how certain risk factors for disease can increase the chance for bleeding in the brain,” explained Van Nostrand. “This will be done using an animal model developed here at URI that mimics CAA found in humans.”

The project will begin in January 2027, and Van Nostrand will collaborate with scientists from Harvard Medical School, Vanderbilt University Medical Center, Vall d’Hebron Research Institute (Spain), and Otto von Guericke University Magdeburg (Germany).

Since 2024, Van Nostrand has been working on another project funded by the Leducq Foundation that focuses on the disruption of brain waste clearance mechanisms that contribute to the biological processes by which CAA develops.

“Support from these highly competitive and prestigious transatlantic networks will reveal new insights into the disease, paving the way for better diagnosis and development of disease-modifying therapies,” said Van Nostrand.

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CancerCare project highlights barriers to patient access

Edusehat — Tue, 18 Aug 2026 00:35:02 +0700

When it comes to fighting cancer, time is of the essence. But far too often, utilization management (UM) practices such as prior authorization and step therapy create precarious delays to care. These delays consume patients’ valuable time and money, create mental anguish, interfere with treatment decisions, and can often lead to worse health outcomes.

To capture the human impact of UM practices and to empower patients to effectively advocate for themselves, CancerCare, a national non-profit organization that provides emotional, practical and financial support for anyone affected by cancer, recently launched the Micro Insights, Macro Impact project.

The resources developed in conjunction with the project include the Focus Group Report, “Behind the Barriers: Patient Voices on Access to Treatment,” an Advocacy Toolkit, and three videos.

‘Fail first’ policies fail cancer patients

“My insurance company asked me to try and fail one treatment before I can get the actual treatment that I needed, and I was devastated by that,” said Maria, a patient living in New Jersey, with stage 3b uterine cancer. In a video testimony for Micro Insights, Macro Impact, Maria details her experience with step therapy, a utilization management practice where health insurance companies require patients to try other, often less effective, treatments first before they provide access to the treatment the patient was actually prescribed by their healthcare provider.

Maria’s case demonstrates the devastating impacts of step therapy on patients: only after experiencing harmful effects from the insurance-required treatment did Maria become eligible for the treatment her doctors prescribed. “I was then informed by doctors, Oh, you had the rash, so now you’re actually eligible for the better chemo. And I was like, What do you mean, better chemo? You have to have a side effect first before we allow you to advance to the better chemo. I was shocked,” explained Maria.

‘Prior authorization’ prioritizes insurers, not patients

Maria also experienced barriers from prior authorization, a process most patients know all too well. Prior authorization is a strategy used by health insurance plans that requires providers to obtain approval from the health insurance plan for a medicine before insurance will cover it. Oftentimes, prior authorization increases administrative burden on patients and providers and delays access to medically necessary treatments.

“It was really hard to get anything filled,” said Maria. “I had to wait until I got an authorization. I had a waiting period of about a month and a half. No treatment, no medication, just waiting for the new coverage.”

Maria’s experience is all too common, and costs patients and caregivers valuable time. As CancerCare’s 2025 Insurance Red Tape report found, among patients directly impacted by prior authorization protocols, 51% lost up to a full business day, 27% lost up to 2–3 business days, and 12% lost a full business week or more dealing with a single authorization incident. The Red Tape report labeled this issue as “time toxicity,” where a patient or caregiver spent substantial time communicating with insurers, gathering documentation, and appealing decisions. And patients are not just losing time out of their day, they are losing valuable treatment days. The report also found 29% of participants reported diagnosis delays, and 40% reported treatment delays due to authorizations. Additionally, 14% experienced abrupt coverage stoppages in the past year and 64% of those experienced treatment interruption.

The Micro Insights, Macro Impact project conducted a series of patient focus groups which captured poignant, real-life examples of the UM challenges and barriers documented in the Red Tape Report. These stories culminated in a telling report titled, “Behind the Barriers: Patient Voices on Access to Treatment.”

Like Maria, Steven, a patient from the suburbs of New York with stage four colorectal cancer, provided additional context to the focus group report through his video testimony about his UM challenges and how they affected his life. “They don’t know who you are. They don’t care who you are. You’re just a number,” said Steven. “When you have cancer, it’s stressful enough. And when you’re dealing with insurance companies, it’s a double whammy, right? From prior authorizations to delays to denials, they throw a bunch of terms at you, but you know no one bothers to translate what it actually means. How do I navigate through this?”

“This isn’t a game. This is my health,” said Steven, “Why is this so difficult? It can be a bit dehumanizing. I think it’s important for the decision makers to really think about it from a more humane perspective. The system’s broken, and I think we could do a better job in making people’s lives easier.”

In a third video, “What It Feels Like When Insurance Stands Between You and Your Care,” three cancer patients succinctly and boldly speak about the unacceptable consequences of UM on their lives and what they would say to the people who denied their coverage.

Improving access and affordability

Individuals living with cancer deserve better. Utilization management practices are causing real harm to patients for whom time is of the essence.

“It is a nightmare waking up every day wondering if some people halfway across the country will approve what my doctors have said is my only chance,” said a patient in the Micro Insights, Macro Impact report.

Ultimately, many of the barriers imposed by utilization management are wholly unnecessary. “Among the 1,201 respondents who experienced prior authorization,” the Red Tape report states, “95% ultimately had their most recent request approved—89% after initial review and 6% after appeals—yet delays still frequently occurred, even when the initial request was approved.” These findings put in question the premise behind utilization management – WHY the initial treatment was denied at all in the first place, which jeopardizes the health of cancer patients who cannot afford to wait.

Empowering patients

Another key takeaway of the Micro Insights, Macro Impact project is the importance of empowering patients and caregivers to engage in policy advocacy by sharing their personal stories with decision makers. To help facilitate this goal, the project’s Advocacy Toolkit helps familiarize patients with UM health policy terms and provides an easy-to-understand framework for people to reach out to policy makers via letter writing, social media posts, and meetings. The Toolkit provides “how-to” examples of the different policy advocacy methods to help demystify the process and boost the confidence of patients and caregivers.

Micro Insights, Macro Impact marks a critical step forward in moving the needle toward meaningful utilization management reform, showcasing the people and stories behind the numbers.

“Utilization management practices are being implemented in ways that harm patients. Micro Insights, Macro Impact spotlights the shared challenges that disrupt patients’ daily lives from diagnosis and beyond. These stories put a human face to the data, point to where policy change is needed most, and empower patients and caregivers to help drive necessary change” said Kim Czubaruk, JD, Vice President of Policy, CancerCare.

If you are interested in learning more, you can visit https://www.cancercare.org/, where you can read Micro Insights, Macro Impact, the 2025 Red Tape Report, and other CancerCare resources.

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Your AI Is Only as Good as Your Inputs

Edusehat — Tue, 18 Aug 2026 00:30:11 +0700

Augustė Užuotaitė
R&D Supervisor, Genetic Sciences
Thermo Fisher Scientific

Lab leaders are under pressure to adopt AI and automation, and most of that pressure is well-founded. When properly implemented, AI and robotic systems, including automated polymerase chain reaction (PCR) workflows, can reduce manual variability, ease workflow bottlenecks, and produce high-quality data. When integration runs ahead of the science underneath it, new tools can introduce errors and slow down the work they were meant to accelerate.

In genetic analysis, credibility is built on reproducibility. New instruments and software continue to enter the market with the promise of faster and higher-quality results. However, the variable that is most often overlooked is also the most consequential: the reagents and consumables that influence every reaction. Real-time PCR (qPCR) assays, master mixes, primers, probes, plates, seals, and tips quietly determine whether a workflow can be trusted, especially as new platforms enter the market with the promise of faster, cleaner results.

When labs chase speed without stabilizing the basics, they risk building sophisticated workflows on unstable foundations. AI and automation cannot reduce variability on their own. They depend on consistent consumables, robust chemistry, and strong traceability. Without that foundation, small issues can scale into batch-level failures. Labs that want to succeed with new technology must first invest in analytically validated reagents that make that technology trustworthy.

Automation amplifies everything

Automation can generate more data faster, but it can also scale small problems into larger ones when inputs from genetic analysis experiments, such as assay design, reagent chemistry, and consumables, are inconsistent. For example, a master mix that performs well in a manual benchtop workflow may behave differently after sitting on an automated deck for an extended period. If evaporation, temperature exposure, or incomplete mixing shifts reaction concentration in only a subset of wells, the result may not appear as a complete run failure. Instead, the lab may see subtle threshold cycle (Ct) shifts, increased well-to-well variation, or edge effects that are difficult to trace after the run is complete. In high-throughput qPCR, reproducibility often depends less on the thermal cycler than on reagent performance and reaction assembly.

Automation without consumable consistency across assays and master mixes does not always save time. Rather, it shifts time from pipetting to troubleshooting. Before scaling, labs should evaluate whether assays, master mixes, and consumables can perform under real automated conditions (e.g., hold times, ambient exposure, and mixing steps); not just under ideal bench conditions.

Consumable quality is critical

Multiplex qPCR leaves almost no margin for uncontrolled variability, which makes consumable consistency across qPCR assay design and chemistries an especially important consideration in genetic analysis labs. When several targets are amplified in a single reaction, noise sources, such as baseline instability, spectral bleed, and inconsistent fluorescence transmission, can compete with the real signal. The result is curves that are difficult to interpret and translate into action.

Consumables that perform consistently lot-to-lot, with low-binding surfaces, robust sealing, and low-background optics, paired with multiplex-stable master mixes, give labs fewer reruns and cleaner target differentiation. The practical step is to qualify every new lot under the conditions where it will actually run, including hold times, mixing steps, and full multiplex panels, before releasing it into production. That requires evaluating plates, seals, and tips for autofluorescence, adsorption, and lot-to-lot optical consistency, not just for sterility or general compatibility.

What your controls aren’t saying

Common assumptions about consumable quality create blind spots that can become dangerous at scale. Three are worth calling out. First, “sterile” does not automatically mean nuclease-free or DNA-free. These certifications cover different things, and a label that satisfies a procurement specification may not satisfy a sensitive amplification reaction. Second, passing controls does not guarantee an entire plate is unaffected. Controls can miss edge effects; low-input loss and mild inhibition can still distort results in target wells. Third, master mixes are not interchangeable. Formulation and lot variation change efficiency, inhibitor tolerance, and multiplex behavior, sometimes in ways that only surface after a method transfer.

A practical step that genetic analysis labs can take is to test consumables under worst-case conditions: low input, edge wells, and extended hold times. That is closer to how an automated workflow actually behaves.

AI is not a guarantee

AI has the potential to add real value in genetic analysis workflows, but only when it sits on top of consistent physical inputs and good metadata. It does not replace the need for widely used consumables and traceability.

AI has the potential to flag issues early, including Ct drift, edge effects, and unusual amplification curves. It tracks trends over time and links problems to reagent lots, instruments, or staging conditions. However, the dependency runs in both directions. AI-driven quality monitoring is only as good as the data it receives. If consumables introduce uncontrolled variability through inconsistent optics, adsorption losses, or leachables, AI may detect symptoms without identifying root causes. Even worse, it may normalize drift if the baseline itself is unstable. The difference between a useful flag and a misleading one usually comes down to whether the lab can trace what changed.

Consider this scenario: A lab uses AI to monitor plate-to-plate performance, and the system flags a subtle upward Ct trend. With appropriate traceability, meaning lot numbers for plates, seals, master mix, and tips linked to each run, the team traces the issue to a new consumable lot with higher autofluorescence. Without that metadata, the trend is visible but unexplainable. The model can flag that something is wrong, but it cannot explain it.

The implication: traceability is a workflow discipline problem, not a software problem. Scientists need to build traceability into every run (e.g., link reagent lots, consumable lots, staging conditions, and instrument IDs to results). Without this metadata for AI to consume, there is no AI-driven quality monitoring.

Get the foundation, then scale

The real innovation ahead is not just smarter software or technology. It is robust assay design, reagent chemistry, and consumables designed for automated workflows: stable at room temperature, tolerant to inhibitors, and consistent in multiplex performance. Better traceability, with reagents and labware tracked by lot and linked to QC data, supports more unattended runs, fewer failures, and faster troubleshooting when something does go wrong.

AI and automation can move genetic analysis forward, but only when the underlying workflow is already controlled. The lab of the future will not be defined by how much human judgment it removes. It will be defined by how well it preserves that judgment with better data, better context, and fewer avoidable sources of variation. That starts with unglamorous work: qualifying consumable lots, documenting reagent and labware changes, stress-testing chemistries on automated decks, and making traceability part of every run. These are not side details. They are the foundation that determines whether AI-enabled genetic analysis produces insight or simply scales uncertainty.

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Roundup: Removing Impurities from Biomanufacturing

Edusehat — Tue, 18 Aug 2026 00:30:10 +0700

Effective impurity control is critical to biomanufacturing, supporting process, cell line, and formulation stability while preserving biotherapeutic potency and shelf life. Residual impurities can drive oxidation, enzymatic degradation, protein unfolding, and aggregation. GEN asked leaders in biomanufacturing: What technologies do you use to carry out effective impurity removal at specific points throughout upstream and downstream processing to ensure product quality and patient safety?

Agilent Technologies

Lizzy Nye, PhD
Global Biopharma Segment Manager

Effective impurity removal in biomanufacturing requires strategically integrated separation and analytics across both upstream and downstream workflows. Early-stage control focuses on removing process-related impurities, such as host cell proteins (HCPs), DNA, and media components, where affinity chromatography remains foundational for bulk clearance and process robustness

As molecules increase in complexity, orthogonal analytical approaches become critical. High-resolution multidimensional separations enable deeper interrogation of complex mixtures and reduce the risk of co-eluting impurities. Agilent Bio-inert LC platforms are developed to minimize metal interactions that can compromise protein integrity or mask low-level impurities, improving sensitivity and reproducibility for biologics and metal-sensitive analytes.

David Wong, PhD
Global Biopharma Segment Manager

Downstream purification strategies are increasingly tailored to modality. LC solutions paired with integrated software workflows, support efficient peptide purification with yield and purity tradeoffs. For oligonucleotides, impurity removal often relies on orthogonal ion-pairing reverse phase (IP-RP) and anion exchange (AEX) chromatography to resolve sequence failures and closely related variants.

Ultimately, combining robust purification technologies with advanced analytical workflows ensures not only effective impurity clearance, but also comprehensive characterization critical to maintaining product stability, regulatory compliance, and patient safety.

Lonza

Driving effective impurity control in biomanufacturing requires orthogonal diversity coupled with capacity redundancy and holistic process stewardship.

Sam Maurice
Associate Director, Global Process and Analytical Sciences
Integrated Biologics

Bioinformatics can inform liabilities before even picking up a pipette. Cell line stability, clone selection and high-throughput upstream optimization reduce host cell protein (HCP) accumulation by preventing upregulation of lipases and proteases that cause oxidation and unfolding but serve to protect cellular function under prolonged stress.

Often overlooked, effective primary harvest depth filtration combined with charged media is the first line of defense, separating impurities based on size and electrostatic, hydrophobic, and hydrogen bonding mechanisms. This focus is central as upstream processes intensify.

Downstream processing should leverage heterogeneous product characteristics: selection of specialized affinity resins, enhanced washes that disrupt “hitchhiking” HCP-target molecule-histone complexes, and reduced proteases that contribute to ligand leachate. Viral inactivation precipitates impurities to aid physical removal when followed by charged depth filtration, which should be optimized for pH and conductivity conditions. Multimodal resins combining IEX, HIC and hydrogen bonding provide powerful synergistic polishing for removing aggregates, fragments, stubborn HCPs, endotoxins, viruses, and residual affinity ligand to provide representatively pure product, essential for successful liquid formulation studies. Changes to regulatory guidance mandate future HCP characterization using LC-MS informed early process development, to ensure patient safety, product stability, and promote the maximum possible shelf life.

MilliporeSigma

Chad Rogers
Strategic Product Manager
Process Materials and Cell Culture

Effective impurity control is enabled through a combination of advanced analytics, targeted purification technologies, and upstream material design. Upstream, MilliporeSigma minimizes impurity introduction through stringent raw material qualification, trace element (TE) control strategies, and high-sensitivity analytical platforms such as ICP-MS for multi-element profiling. These capabilities allow precise characterization and specification of low-level metal impurities that can catalyze oxidation or destabilize proteins. Engineered cell culture media further reduce variability by controlling impurity ingress at the source, supported by supplier qualification and risk-based raw material selection. In-process controls and real-time monitoring ensure consistency throughout bioreactor operations.

A recent supply chain disruption involving ferric ammonium citrate (FAC) put these capabilities to the test. When alternative sourcing introduced material with markedly different TE impurity profiles, MilliporeSigma leveraged its integrated chemical manufacturing expertise and ICP-MS analytical platforms to engineer an FAC solution aligned with historically established TE baselines. Defined impurity targets preserved process comparability and avoided customer reformulation, demonstrating how analytical comparability and change management frameworks keep impurity profiles within defined limits when supply or process adjustments are required.

In summary, MilliporeSigma integrates analytical rigor, engineered impurity control, and robust purification technologies across upstream processes, providing a scalable, supply-resilient foundation that safeguards product quality, process consistency, and patient safety while reinforcing leadership in differentiated bioprocessing solutions.

Repligen

Effective impurity control begins with a well-designed purification strategy. Across downstream processing, the objective is straightforward: remove process- and product-related impurities while preserving the quality, potency, and stability of the therapeutic.

Brian Douglass
Senior Vice President
Chief Product Officer

Repligen has built a comprehensive portfolio of purification and analytical technologies designed to address impurity challenges across a wide range of biologics. Affinity chromatography remains one of the most powerful tools available, routinely removing more than 95% of process-related impurities in a single step while maintaining yields above 90%. Our portfolio includes both catalog affinity resins and industry-leading capabilities for the rapid development and commercialization of custom affinity ligands. These custom solutions can address particularly challenging separations, including product-related impurities that are difficult to resolve using conventional polishing techniques.

Filtration plays an equally important role throughout the purification workflow. Tangential flow filtration, using either flat-sheet or hollow-fiber formats, supports impurity clearance, enhances chromatography performance, and enables efficient formulation of the final drug product. Together, chromatography and filtration create a highly effective platform for delivering consistent product quality and process robustness.

What differentiates Repligen is the integration of purification technologies with advanced process analytical technologies (PAT). We believe effective impurity control requires not only removing contaminants but also measuring and understanding them in real time. Our portfolio spans at-line, on-line, and real-time analytical solutions that provide actionable process insight directly to operators and manufacturing teams.

Because no single analytical technology can address every challenge, we have deliberately built a broad PAT portfolio that enables customers to apply the right tool to the right problem. Repligen provides solutions that provide real-time UV-Vis and Raman analytics for upstream and downstream process monitoring, while platforms enable deeper characterization of media components, proteins, and critical impurities.

Ultimately, successful impurity control requires the seamless integration of purification, filtration, and analytics. By combining these capabilities into a connected bioprocessing ecosystem, Repligen helps customers accelerate development, strengthen process understanding, and consistently manufacture safe, high-quality biologics.

Sartorius Stedim Biotech

Effective impurity control is essential in biomanufacturing, with strategies tailored to the target molecule and process. Downstream processes for monoclonal antibodies (mAbs) have demonstrated a direct impact on product quality and patient safety. Key impurities—host cell proteins (HCPs), residual DNA, endotoxins, aggregates, and process-related chemicals—originate from the expression system or manufacturing steps. If not adequately removed, HCPs and DNA can trigger immune responses or toxicity in patients, aggregates may induce immunogenicity or reduce efficacy, and endotoxins can cause severe inflammation. Process chemicals, like Protein A leachates or buffer residues, further contribute to safety risks.

Anke Boerdgen
Head of Product Management Chromatography Consumables
Separation Technologies

Industry-leading technologies are applied in downstream processing to ensure impurity removal. Protein A affinity chromatography captures mAbs and eliminates bulk HCPs and DNA, mostly followed by a prolonged hold step at low pH to facilitate virus inactivation. Polishing steps with ion exchange as well as hydrophobic interaction chromatography remove charged contaminants, viruses, and aggregates. Sartorius membrane chromatography enables a complete membrane-based workflow for efficient impurity removal and high productivity, while bioburden, sterile, and virus filters ensure reliable removal of particulates, bacteria, and viruses, maintaining the critical quality attributes before final formulation. All process steps are usually complemented by several ultrafiltration and diafiltration steps, which facilitate further impurity removal, buffer exchange, as well as concentration of the molecule of interest.

Regulatory entities rigorously assess these purification steps during drug approval, ensuring impurity removal meets stringent safety standards to protect patient health, as demonstrated in many commercial processes.

Veranova

For antibody-drug conjugates (ADCs), it is critical to tightly control reaction stoichiometry, buffer composition, temperature, and reaction time. Hold times during antibody modification and conjugation should also be assessed, as delays in processing the antibody-linker intermediate may alter reaction stoichiometry or increase product-related impurities that complicate downstream purification.

Thomas Rohrer
Vice President of Bioconjugation

Tangential flow filtration (TFF) is widely used with minimal yield loss to remove small-molecule stabilizers from antibody storage buffers prior to conjugation, as well as for ADC concentration, buffer exchange, and clearance of process-related impurities such as free payload, linker, and organic solvents. Compared to chromatography, TFF is typically more scalable, cost-effective, and higher yielding, largely due to the significant size difference between the ADC (~150 kDa) and payload (<2 kDa).

In some cases, carbon filtration is applied after TFF as an orthogonal step to remove residual linker-payload species. Depending on antibody subtype, conjugation method, and payload, product-related impurities may include aggregates and undesired isoforms. In these situations, size exclusion chromatography or hydrophobic interaction chromatography may be necessary, although they can reduce yield and increase manufacturing cost.

As novel conjugation technology evolves using bispecific mAbs, Fc-fusion proteins, and chemical site-specific conjugation, traditional TFF systems will be sufficient for purification alone or in combination with chromatographic separation techniques to deliver homogeneous ADCs with uniform drug-to-antibody ratios.

WuXi Biologics

Residual impurities are primarily process-related, including DNA, host cell proteins (HCPs), leached Protein A, and upstream additives. Effective control of these impurities requires an integrated strategy across both upstream and downstream processes. While cell line and process development help define and minimize the initial impurity burden, downstream purification takes the main responsibility for their removal.

Sherry Gu, PhD
Executive Vice President, CTO and Chief Client Officer

Among the residual impurities, lipases and metal ions can be particularly detrimental due to their roles in protein degradation and instability. In downstream processing, Protein A affinity chromatography is one of the most effective unit operations for removing such impurities, owing to its high selectivity for antibodies/Fc-fusions. However, certain HCPs and other impurities may interact with the target molecule and copurify.

To address this, it is critical to disrupt these interactions. Therefore, we extensively optimize Protein A wash conditions using additives such as amino acids, chaotropic salts, organic solvents, or detergents to enhance HCP clearance. In one case, we successfully removed cathepsin B, a CHO endogenous protease that chops the target antibody, by adding sodium caprylate into the wash buffer.¹ Similarly, EDTA is used in the wash buffer to remove metal ions. High-pH eluting Protein A resins and Protein A membranes have also demonstrated advantages for HCP clearance, reducing HCP levels to below those achieved with regular Protein A resins.^2,3

Beyond the affinity step, intermediate depth filtration following low-pH inactivation/ neutralization and subsequent polishing steps are further optimized to reduce residual impurities. Among polishing techniques, hydrophobic interaction chromatography (HIC) has shown particular effectiveness in removing lipases, and ongoing evaluations focus on resins with varying hydrophobicity to enhance performance.

References

1. Hu L, Tang J, Zhang X, Li Y. Sodium caprylate wash during Protein A chromatography as an effective means for removing protease(s) responsible for target antibody fragmentation. Protein Expr Purif. 2021, 186: 105907.

2. Li Z, Hu L, Li Y. The advantage of high pH eluting Protein A resins over their regular counterparts in aggregate and host cell protein clearance. Protein Expr Purif. 2026, 239: 106859.

3. Yang Y, Zhang X, Li Y. Deciphering the inconsistent performance of Protein A membrane on host cell protein clearance. Protein Expr Purif. 2026, 240: 106891.

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Malaria-Causing Plasmodium in Uganda Show Rapid Spread of Drug Resistance Mutations

Edusehat — Mon, 17 Aug 2026 17:20:09 +0700

Drug resistance is one of the biggest threats to controlling malaria in endemic areas of the world. This issue was first recognized as early as the 1950s when chloroquine resistance was identified in Plasmodium falciparum. Since then, chloroquine resistance has spread to nearly all areas where malaria is endemic and P. falciparum has acquired resistances to all currently available drugs including sulfadoxine/pyrimethamine, mefloquine, and quinine.

Now, researchers have identified new mutations associated with the parasite’s decreased susceptibility to current treatments. By sequencing the whole genomes of Plasmodium falciparum from the blood of hundreds of infected people in Uganda, the team found that a cluster of genetic variants showed significantly decreased susceptibilities to the drugs most commonly used to treat malaria in Africa and the United States.

The findings were published in Nature Medicine in the paper, “Emergence and spread of Plasmodium falciparum PX1 polymorphisms associated with decreased susceptibility to antimalarials in Uganda.”

“It’s very concerning that these new mutations are spreading so rapidly—it tells us they are important to the parasite’s survival,” said Jeffrey Bailey, MD, PhD, associate professor of translational research and of pathology and laboratory medicine at Brown University. “Malaria still is a major killer, particularly in sub-Saharan Africa. As drug resistance continues to emerge, we worry it will undermine control of its spread and result in even more deaths for a large number of people there and beyond.”

Because of growing drug resistance, surveillance systems are being built to track known mutations in the pathogen as well as drug performance over time and identify biological markers of drug resistance.

“We knew that the parasites were changing so that over time, their susceptibility to malaria treatments was decreasing, and we wanted to know the exact genetic determinants of this shift,” said Karamoko Niaré, PhD, formerly a postdoctoral researcher in Bailey’s lab and now an adjunct assistant professor of pathology and laboratory medicine at Brown. “We decided to sequence the entire genome to get a better sense of what was going on.”

For the last two decades or so, the primary treatment for uncomplicated malaria in Uganda has been artemether-lumefantrine (AL), the most used artemisinin-based combination therapy (ACT) across sub-Saharan Africa. As of 2026, the Centers for Disease Control and Prevention has been recommending a longer course of therapy because standard doses failed to cure several travelers returning home, suggesting that the parasites are becoming less susceptible to treatment.

The researchers identified an area in the genome with 69 genes. More specifically, three specific mutations and two deletions were associated with decreased susceptibilities to the drugs artemisinin and lumefantrine (both components of AL) as well as the malaria drug mefloquine. The mutations most likely to drive this selection were found in a gene that encodes phosphoinositide-binding protein (PX1 protein) which is often found near another gene known to cause moderate resistance to the drug artemisinin.

This is the first time researchers have correlated a gene mutation with reduced susceptibility to multiple drugs used in the combination therapy for malaria.

“We didn’t have any validated molecular marker of lumefantrine resistance—we knew that there was a gene involved in partial resistance to artemisinin but couldn’t explain changes observed for lumefantrine,” Niaré said. “Our work identifies a molecular marker that could be used by surveillance studies to track the emergence and spread of reduced susceptibility to front-line malaria treatments across Africa. That’s a very important tool for public health.”

Since this effect was studied in the lab in parasites that had been collected from malaria patients, Bailey said future research should investigate how these mutant parasites impact clinical outcomes of malaria treatment with ACTs. While the authors found that the mutation was spreading rapidly in Uganda, how far it has spread beyond Uganda’s borders is unknown and needs to be examined.

The finding has major implications for sustaining an effective malaria treatment program, Bailey said. “It underscores the need to develop prediction models for when the drug will stop working altogether and also highlights the urgency to develop new drugs to treat malaria.”

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CDMOs Expand and Build for Future Growth

Edusehat — Sun, 16 Aug 2026 01:50:09 +0700

The top contract development and manufacturing organizations (CDMOs) have been busy in recent months expanding their service offerings, building new facilities, and navigating how to incorporate artificial intelligence (AI) into their operations. Following is a roundup of recent activity by five CDMOs, based on recent interviews with GEN:

Fujifilm Biotechnologies

Fujifilm Biotechnologies has celebrated the selection of its commercial-scale cell culture manufacturing facility in Holly Springs, NC, among seven facilities chosen by the FDA for its PreCheck Pilot Program, designed to strengthen the nation’s pharmaceutical manufacturing capabilities.

Last year, Fujifilm Biotechnologies opened the first phase of its $3.2 billion end-to-end biomanufacturing facility in Holly Springs. The company initially committed $2 billion to a facility consisting of eight 20,000 L mammalian cell culture bioreactors, then in 2024 announced plans to invest an additional $1.2 billion by building eight additional 20,000 L bioreactors at the site by 2028. About 800 people are based at Holly Springs, a figure set to grow to 1,400 when the second phase is completed.

One biopharma customer has been announced for the second phase—the immunology drug developer argenx, which has agreed to manufacture drug substance for efgartigimod alfa-fcab marketed as intravenous Vyvgart^® and combined with hyaluronidase-qvfc as injectable Vyvgart Hytrulo^®, both indicated for adults with generalized myasthenia gravis or chronic inflammatory demyelinating polyneuropathy.

Holly Springs is one of Fujifilm Biotechnologies’ two large-scale biomanufacturing facilities. The other is Hillerød, Denmark, where in April the company cut a ceremonial ribbon to mark the opening of a 2,000‑square‑meter (21,528-square-foot) quality control (QC) laboratory at its commercial‑scale manufacturing site.

Approximately 100 members of the company’s Quality team are based at the lab, where they conduct viral safety testing for drug substance/product release, scale capacity for complex cell‑based potency and ELISA methods, and perform raw material and critical total organic carbon cleanability studies. The QC lab also includes robotics and supports the implementation of an ongoing laboratory information management system (LIMS) across company sites to enable digital harmonization and data integrity.

Operations began in May at the QC lab, which is housed within a new 7,600-square-meter (81,806-square-foot) building with employee amenities, office and collaboration space, utility services, and an emergency generator to ensure uninterrupted operations and timely delivery of test results. The Hillerød site—which parent company Fujifilm Corp. acquired from Biogen in 2019 for $890 million—was expanded in 2024 from 6 to 12 x 20,000 L mammalian cell culture bioreactors, increasing the complexity and volume for QC testing.

“Simply, we needed more QC analytical capacities to support all the increased number of batches and the increased number of testing programs,” Toshihisa Iida, director, corporate vice president, general manager of Life Sciences Strategy Headquarters and the Bio CDMO Division of Fujifilm, told GEN.

“Our current focus is to get all our facilities online, go live, and focus on operational excellence because we already have big contracts with big clients. Our mission is to deliver as promised,” Iida said. “Before we decide on any next-phase investment, we will really focus on investing more in our people, investing more in our quality system, getting first-class operations, then delivering to our clients.”

Fujifilm Biotechnologies is part of the Fujifilm Life Sciences Group along with Fujifilm Biosciences (formerly Fujifilm Irvine Scientific), a provider of products and services from discovery research through large-scale production—and three other companies, one of which is Fujifilm Cellular Dynamics, a developer and manufacturer of human induced pluripotent stem cells (iPSCs) used in drug discovery, toxicity testing, stem cell banking, and cell therapy development.

In May, Fujifilm Cellular Dynamics celebrated the opening of its new 175,000-square-foot headquarters and iPSC development and manufacturing facility in Madison, WI, which features cell culture manufacturing laboratories, process development laboratories, and a center of excellence for gene editing—all designed to support both research-grade iPSC products and development services for partners developing next-generation cell therapies. The nearly 200-employee facility is part of a previously announced $200 million strategic investment by Fujifilm.

Lonza Group

As AI reshapes how drugs are discovered, developed, clinically studied, and commercialized, Lonza Group says its operations are evolving to reflect that change. Lonza is in the process of building a team of 20 to 30 AI-focused professionals that tap into the CDMO’s experience stretching back to the early 1980s of helping customers launch more than 80 drugs.

“There is retained expertise that customers come to us for, and what I’m excited about is that we should use AI to enhance that,” Matthew Moorcroft, PhD, Lonza’s chief intelligence and analytics officer and head of the CEO office, told GEN. “What we’ve learned over the last nearly 45 years is in our DNA, and it’s in our people. Using AI to further enhance that further empowers us to do better product launches, to serve customers quicker, and to interact with them in better ways. That’s our ambition.”

Customers have shown interest, he said, in incorporating AI into molecule discovery and designing molecules, as well as into their clinical trial processes.

In addition to strengthening its AI capabilities, Lonza is enhancing its two major operational facilities in Switzerland. The company announced plans to expand its aseptic drug product capacity in Stein by adding another commercial-scale multi-purpose filling line for antibody-drug conjugates (ADCs), expected to be operational in 2030. The Stein expansion is supported by a long-term collaboration agreement with an undisclosed “major pharmaceutical” company for clinical and commercial ADC supply. Also at Stein, Lonza is expanding its commercial capabilities to high-value small molecule drug products, with those operations expected to start in 2028.

In June, Lonza also revealed plans to expand its Visp site to establish new commercial-scale capabilities for manufacturing highly potent active pharmaceutical ingredients (HPAPI) and ADC payload-linkers, reinforcing Lonza’s position as a fully integrated CDMO partner in ADC development. Lonza says the expanded Visp facility—set to be fully operational in 2028—will be able to offer additional payload-linker production and purification alongside dedicated analytical and process development labs.

Beyond facilities, Lonza Group is transforming into a pure-play CDMO, announcing plans in March to sell its Capsules & Health Ingredients (CHI) business to Lone Star Funds for CHF 2.3 billion (2.836 billion), a sale expected to close before year’s end. The deal—set to close in the second half—will give Lonza CHF 1.7 billion ($2.2 billion) upfront, and a 40% stake in CHI—which finished the first half of 2026 with core earnings before interest, taxes, depreciation, and amortization (EBITDA) of CHF 1.152 billion ($1.42 billion) on revenue of CHF 3.374 billion ($4.16 billion), up 11% year-over-year.

Once the deal closes, Lonza says it plans to invest its upfront proceeds into growth-focused activity reflecting its “One Lonza” strategy, which simplified operations from three divisions and nine business units into three integrated CDMO business platforms (Integrated Biologics, Advanced Synthesis, and Specialized Modalities), as well as fund acquisitions of smaller complementary or “bolt-on” businesses. Through a share buyback program, Lonza plans to return CHF 500 million ($616 million) to shareholders.

“What we’ve agreed and what we’ve decided is to be purely focused on delivering value to our CDMO customers. That’s where Lonza’s experience is; that’s what we do,” Moorcroft explained. “When we did our organizational review two years ago, we asked, ‘What is non-core to that vision, in terms of our vision, purpose, and values?’ And being a contract manufacturer for our customers—not making our own products, not competing with them—was actually core to what we believed in. And we believe that pure play focus is really important to keep winning business and to convince our customers to stay with us. There’s no distraction.”

MilliporeSigma (Merck KGaA, Darmstadt, Germany)

Merck KGaA, Darmstadt, Germany, whose Life Sciences business in the United States and Canada is known as MilliporeSigma, is completing construction of a €300 million ($346.5 million) Bioprocessing Production Center in Daejeon, South Korea, that is set to open by year’s end.

“Since COVID-19, we have implemented a global manufacturing region-for-region strategy where we are building manufacturing capacity in every geography, on every continent. And we continue to do that,” Sebastián Arana, the head of Process Solutions for the Life Science business of Merck KGaA, Darmstadt, Germany, told GEN.

Over the past four to five years, Arana added, Merck KGaA’s Life Sciences business has deployed more than €3 billion ($3.465 billion) in capital expenditures (capex) toward building out capacity to meet customer demand.

In Daejeon, Arana said Merck KGaA aims to be closer to customers in Korea and the rest of the Asia-Pacific region. He cited growth momentum in South Korea, fueled by business activity as well as President Lee Jae Myung committing his government last year to easing regulation of biotech R&D, and catapulting the nation into a top-five biotech industry powerhouse by 2030.

“Korea has intentionally invested in creating a life science ecosystem in the last few years,” Arana said, “with wonderful government support for life science being a critical area to invest in the economy, that’s number one. Korea is an easy country to do business with, so a lot of the Western pharma companies feel very safe working with Korean suppliers. All of the supply chain is very reliable, very, very quick. Plus, they have a history of very good manufacturing capabilities and have now expanded to bioprocessing. They are very flexible and super quick—things that pharma is looking for. They can do technology transfers in months.”

Another portion of capex investment for expansion of manufacturing came in Cork, Ireland, where Merck KGaA last year opened a €150 million ($173.25 million) filtration manufacturing facility in Blarney Business Park. The 3,000-square-meter (32,292-square-foot) cleanroom facility was designed to support global demand for filtration products used in the manufacturing of vaccines and therapies, including monoclonal antibodies, and emerging modalities such as cell and gene therapies.

“More and more, customers are asking for supply chain resiliency, redundancy, to make sure that if something happens with their supply chain, they’re ready to jump from one side to the other. That’s the fundamental strategy we’re executing,” Arana said. “Ideally, they want the closest site for them, but also, we try to have a second or even a third site qualified in case something happens.”

Another factor driving increased business for Merck KGaA is ongoing reshoring efforts by U.S.-based biopharmas that are shifting more production to the United States, whether to meet growing Stateside customer demand, or to avoid tariffs from the administration of President Donald J. Trump, or both.

“We see some good traction in our U.S. business with customers feeling pretty good about the next two years,” Arana said. “It’s not modality-driven. It’s more just global expansion-driven, and you see some of the large molecules continue to grow and be manufactured in the United States. And on top of that, you need to think that the biopharma space is growing. So, there’s a natural investment that should come every year just to keep up the global demand.”

Merck KGaA expects that demand to continue to grow: “We see the market growing in the 8 to 10% range in the coming years,” Arana said, citing what he called an extremely healthy market for bioproduction of pipeline molecules by drug developers. “MilliporeSigma is very well positioned to continue to grow above market or in the upper side of the range.”

Samsung Biologics

Samsung Biologics expanded into the United States earlier this year when it completed its $353 million acquisition of a former GlaxoSmithKline (GSK) manufacturing site in Rockville, MD. The Rockville site adds two cGMP plants totaling 60,000 L of drug substance capacity, capable of supporting both clinical and commercial biologics production across multiple manufacturing scales, to Samsung Biologics’ total global capacity, which now stands at 845,000 L.

Samsung Biologics retained the workforce of more than 500 that had worked for GSK, which took over the site when it completed its $3.6 billion acquisition of Human Genome Sciences in 2012.

“The Rockville facility gives us our first U.S. footprint and allows us to move into a strategic area within the United States East Coast that is closer to clients, allowing for some additional flexibility with supply from U.S. soil,” John Gagliardi, sales director, key account management with Samsung Biologics, told GEN in a recent interview. “We also wanted to be in a major life science hub, which we have within the DMV [District of Columbia-Maryland-Virginia] area.”

The region, which brands itself BioHealth Capital Region, ranks No. 3 in GEN’s A-List of “Top 10 U.S. Biopharma Clusters.”

The remainder of Samsung Biologics’ capacity is the 785,000 liters available at its Bio Campus I and II in Songdo, Incheon, South Korea, where the company is headquartered. Within Bio Campus II, Samsung Biologics will be collaborating with Lilly Gateway Labs (LGL) to establish an open innovation center, a hub designed to support early-stage and emerging biotechnology companies. The center will have capacity for up to 30 companies to be jointly selected by Lilly and Samsung Biologics, and will occupy a five-story, 125,000-square-foot facility set to be completed in July 2027.

Samsung Biologics has also secured land for BioCampus III, laying the groundwork for future capacity expansion to support next-generation therapies and emerging modalities.

“BioCampus III is meant to house additional complex modalities or new modalities that we would get into outside of mAbs [monoclonal antibodies],” Gagliardi explained. “This is meant for cell and gene therapies or peptides, depending on how the market needs shape up. We will bring that capacity to the market to help as the biopharma industry grows.”

Most recently, on July 19, Samsung Biologics signaled its intent to expand further in the United States, as well as across Europe and India, by announcing plans to acquire publicly traded PolyPeptide Group, a CDMO specializing in the production of peptide-based active pharmaceutical ingredients (APIs), for CHF 1.46 billion (about $1.8 billion).

Samsung Biologics said the purchase of PolyPeptide will enable it to expand its capabilities beyond antibodies and antibody-drug conjugates (ADCs) to include peptide therapeutics, whose fast growth has been fueled by a surge in global demand for obesity treatments and continued expansion of peptide-based therapies into new disease areas.

Headquartered in Baar, Switzerland, PolyPeptide has manufactured APIs for some 70 years and has produced more than 1,000 therapeutic peptides to date.

At CHF44.31 ($54.63) a share, Samsung Biologics’ offer represents a 40% premium to PolyPeptide’s closing price of CHF31.65 ($39.02) on the SIX Swiss Exchange as of April 10, before the company’s acquisition became the subject of market speculation.

Samsung Biologics says its buyout of PolyPeptide is expected to be completed “toward the end of 2026,” subject to customary conditions that include owners of at least 66⅔% of PolyPeptide shares accepting the deal, regulatory approvals, publication of the offer prospectus in accordance with Swiss takeover law, and other conditions.

PolyPeptide’s largest shareholder, representing approximately 55.65% of outstanding shares (excluding treasury shares), supports the deal through an irrevocable tender offer, while the company’s independent and non-conflicted board members have unanimously endorsed the planned acquisition.

Thermo Fisher Scientific

Thermo Fisher Scientific says its two-year-old Accelerator Drug Development platform, a suite of expanded contract research organization (CRO) and CDMO services, has attracted customers seeking an end-to-end partner for services ranging from drug substance development and manufacturing, no matter the modality, to packaging, labeling, distribution, and carrying out clinical trials through the company’s Clinical Research group.

“All of these services within the same company help reduce the time and complexity and bring all the things required for a robust CMC package that our sponsors would then submit for approval and get these effective medicines to patients faster,” Anil Kane, PhD, executive director, global head of technical and scientific affairs at Thermo Fisher Scientific, told GEN.

The company enhanced its ability to glean insights from clinical trial data in March, when it completed its acquisition of Clario Holdings, a provider of endpoint data solutions for clinical trials, for $8.875 billion cash, plus potential additional milestone and other payments.

Thermo Fisher launched Accelerator in 2024, three years after acquiring the CRO PPD for $17.4 billion, and seven years after expanding into the CDMO market by buying Patheon for $7.2 billion. At its Investor Day in May, Mike Shafer, Thermo Fisher’s executive vice president and president of biopharma services, offered examples of how two undisclosed customers benefited from Accelerator.

For a large U.S.-based biopharma seeking a fast commercial launch for its respiratory drug candidate for competitive reasons. Thermo Fisher ramped up a Phase III trial by activating over 160 sites in less than eight weeks, reducing enrollment time and the dosage timeline by more than 50%. The other was a South Korean drug developer that saved 12 months of activity by using Accelerator to eliminate bottlenecks that included formulation issues, trial design issues, and regulatory alignment requirements.

“We took something that was delayed to accelerating it by almost a year,” Shafer said.

In April, Thermo Fisher opened its flagship U.S. Bioprocess Design Center (BDC) at the company’s Plainville, MA, site, expanding the facility to support customers in developing and scaling biologics. The new center brings together advanced bioproduction capabilities and hands-on collaboration, with the aim of helping customers accelerate process development and bring therapies to patients faster.

“Our customers have been asking about opportunities to develop on a small scale, within a U.S. site,” Kane said. “Our Plainville site was ideally located for our customers based in North America to perform experimentation in the development center. We also have sterile fill-finish capability at the Plainville site. By combining the capabilities of bioprocessing as well as sterile fill-finish, our customers could develop their molecule and the product, then decide the next steps of a clinical product that they can take to Phase I and beyond.”

Thermo Fisher agreed in January to integrate into its lab instruments the AI technology of Nvidia, with the aim of developing intelligent and increasingly autonomous laboratories. The Nvidia announcement came three months after Thermo Fisher agreed to embed OpenAI application programming interfaces into areas ranging from product development, service delivery, customer engagement, and operational efficiency.

“We have applied AI tools in quality management, quality operations, and manufacturing. The idea here is to bring efficiencies and reduce that turnaround time of quality deviations, CAPA [Corrective and Preventive Action] management, to bring efficiencies in planning, scheduling of operations, and reduce the downtime of equipment,” Kane said.

Thermo Fisher has yet to publicly quantify the savings in time and cost from AI applications, though it says it has seen promising results: “We will continue to monitor those and quantify those as we generate more case studies, as we generate more data, because this is certainly of importance to us and our partners.”

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A Decade of Advances in Single-Cell Functional Immunomics

Edusehat — Sat, 15 Aug 2026 00:30:25 +0700

The field in 2015

When GEN first covered our work in single-cell functional assays in 2015,¹ immunology was entering a new era of molecular resolution. Researchers were gaining deeper insight into immune-cell heterogeneity through rapidly advancing single-cell technologies, reshaping how disease biology could be studied.

Tania Konry, PhD
Co-founder, Feromics

Yet an important challenge remained unresolved: molecular measurements could describe what immune cells appeared to be, but they did not always predict what immune cells would actually do over time.

Immune cells with activated molecular profiles sometimes failed to sustain durable antitumor activity, while cells that expanded efficiently during manufacturing did not necessarily maintain persistence or therapeutic efficacy. The biological processes that determine therapeutic outcome unfold dynamically through behavior, adaptation, persistence, and dysfunction over time, requiring approaches capable of observing immune behavior directly alongside molecular state.

At Northeastern University, our laboratory focused on developing technologies designed to study immune function in a more dynamic and biologically relevant way. Much of this work centered on creating single-cell systems capable of observing immune-cell behavior in real time while preserving the biological context required for meaningful downstream molecular analysis.

Over the following decade, advances in microfluidics, live-cell imaging, computational analysis, and single-cell biology made it possible to connect directly observed immune behavior with downstream molecular and clinical information at scales that were previously unattainable. What began as an effort to observe immune-cell behavior more faithfully evolved into integrated functional-immunomics systems capable of supporting translational analysis, therapeutic characterization, and more sophisticated computational modeling.

These efforts contributed to the emergence of functional immunomics—an approach centered on understanding immune behavior and its relationship to disease and therapeutic outcome.

A shift toward functional immunomics

One of the major transitions in immunology over the past decade has been the recognition that immune-cell behavior represents a critical biological variable.

Traditional molecular approaches continue to provide essential insight into gene expression, signaling state, and cellular composition. Functional approaches extend those modalities, adding behavioral context to molecular information.

Persistence, resistance to dysfunction, therapeutic durability, and effective immune response are fundamentally functional properties. Behaviors such as serial killing, sustained cytotoxicity, and resistance to exhaustion cannot be fully captured through static measurements alone because they emerge over time.

Advances in microfluidics, live-cell imaging, and single-cell analysis made it increasingly possible to observe these processes directly. In our own work, controlled single-cell pairing systems enabled immune cells to be placed into defined microenvironments with tumor targets, allowing functional behavior to be observed before downstream molecular analysis was performed.^2,3

This sequence—observing what a cell does before analyzing what it contains—represented an important conceptual shift. Rather than inferring function from molecular correlation alone, functional approaches make it possible to anchor molecular interpretation to directly observed biological behavior.⁴

In our early single-cell experiments, what struck me most was how often immune cells with strong activation signatures failed to sustain functional killing over time, and how often cells that appeared less remarkable molecularly turned out to be the ones driving effective cytotoxic responses. After seeing that pattern repeatedly across experiments, it became increasingly difficult to think about immune behavior as something a molecular state alone could fully explain. That observation gradually shifted my own perspective from focusing primarily on what immune cells contained to focusing on what they actually did.

A growing number of academic and industry groups are now contributing to this growing emphasis on function-linked immune analysis. Over time, our own work evolved into what we described internally as “Function-to-Omics”—a conceptual framework in which immune behavior serves as a foundational reference point for understanding disease and therapeutic response.

Much of the foundational intellectual property underlying this work originated through my research at Northeastern University and was later advanced translationally through Feromics, a functional immunomics company focused on AI-enabled immune analysis. The broader goal has been to establish function as a key framework for immune analysis, therapeutic development, and predictive modeling.

The AI inflection point

The rise of AI in biology has amplified the importance of this transition.

Machine-learning systems are fundamentally shaped by the biological quality and structure of the data used to train them. Bulk population-averaged datasets introduce biological noise by averaging across heterogeneous cell states, whereas function-linked analyses resolve this heterogeneity and yield cleaner, more therapeutically informative immune signatures.

A mixture of cells in different states—some highly cytotoxic, some exhausted, some transitional—may produce a population-level signal that reflects none of those states precisely. In practice, this means that cells expressing canonical activation markers may still fail to demonstrate sustained cytotoxic activity when observed functionally over time. Functional analysis makes it possible to separate those behaviors rather than averaging them into a single composite measurement.

As functional single-cell datasets mature, they create opportunities for computational systems capable of linking immune behavior more directly to therapeutic outcome.

More importantly, they change the structure of the learning problem itself.

Rather than training models primarily on correlative molecular associations, function-linked datasets allow computational systems to learn from experimentally observed biological outcomes at the level of individual cells.

This represents an important conceptual shift for the field: moving from systems built primarily on correlation toward approaches informed by experimentally observed biology.

At Feromics, this convergence between functional biology and AI is now being advanced through the development of large functional immunomics datasets linking immune behavior with downstream molecular and clinical information.⁵ Supported in part through a contract with the Advanced Research Projects Agency for Health (ARPA-H)⁶, these efforts have shown potential to predict response to immune-based therapies using function-labeled immune datasets linked to clinical outcome.

In these systems, models are trained on datasets in which observed immune-cell behavior—including cytotoxic activity, persistence, and resistance to dysfunction—is connected directly to downstream molecular and clinical data. The goal is to generate biologically meaningful datasets capable of supporting more predictive and clinically relevant computational models.

The long-term implication is the possibility of improved biological analysis and computational systems that better represent patient-specific immune behavior and therapeutic response.

Clinical translation

The clinical implications of functional immunomics are beginning to emerge across multiple areas of immunotherapy and translational medicine.

In cell therapy, conventional metrics alone often do not fully capture therapeutic potential. Functional approaches create opportunities to improve therapy characterization, donor evaluation, patient stratification, and response prediction through a deeper understanding of immune behavior.

This is already becoming visible in donor-derived immune-cell studies, including work in acute myeloid leukemia (AML) and lymphoma, where functional stratification prior to molecular analysis has begun to reveal distinct transcriptomic programs associated with antitumor activity. These types of approaches create opportunities to identify high-performing immune subsets based on directly observed function rather than molecular inference alone.

Therapy-response prediction represents another important area of development. Profiling patient immune cells based on how they behave in the presence of disease-relevant targets creates the possibility of predicting therapeutic response before treatment begins.

Functional approaches may also influence target discovery and biomarker development by connecting biological activity more directly with therapeutic outcome. In this context, molecular information becomes most powerful when interpreted alongside observed behavior rather than independently from it.

Several of these concepts are now transitioning from academic research into translational development. Through Feromics, technologies originating from my research at Northeastern University are being advanced toward applications in immunotherapy development, precision immune profiling, AI-enabled therapeutic prediction, and next-generation engineered immune-cell therapies.

Over time, functional immunomics may support more individualized therapeutic strategies and more predictive models of immune response across oncology, autoimmune disease, and broader immune-mediated disorders.

Looking ahead

The next decade will likely focus less on proving the value of functional biology and more on integrating it into scalable research and clinical frameworks.

Reproducibility, standardization, data integration, and clinical translation remain major challenges for the field. Functional biology is inherently complex and capturing immune-cell behavior in ways that faithfully reflect human disease remains an ongoing scientific challenge.

For much of modern immunology, the central challenge was describing the immune state with increasing molecular precision. The next phase of the field may be defined not only by molecular description, but also by the ability to measure how immune systems behave dynamically over time—functionally, adaptively, and in ways directly connected to therapeutic outcome.

The question that motivated much of our work in 2015—what is the immune system actually doing?—is now becoming possible to answer at single-cell resolution, at a meaningful scale, and in ways increasingly connected to clinical decision-making.

The convergence of functional biology, AI, and translational medicine is beginning to reshape how immune systems are studied, modeled, and therapeutically engineered.

References

Marusina K. Single Cell Is No Longer a Limit. Genetic Engineering & Biotechnology News. March 15, 2015;35(6).
Sharkey C, Akligoh H, Finocchiaro M, et al. High-throughput 3D matrigel-based droplet microfluidics for single-cell function-to-omics analysis of cytotoxic immune cells in solid tumor interactions. Adv Mater Technol. 2026;202501801.
Sullivan MR, White RP, Dashnamoorthy Ravi, et al. Characterizing influence of rCHOP treatment on diffuse large B-cell lymphoma microenvironment through in vitro microfluidic spheroid model. Cell Death Dis. 2024;15(1):18.
Sharkey C, White R, Finocchiaro M, Thomas J, Estevam J, Konry T. Advancing point-of-care applications with droplet microfluidics: from single-cell to multicellular analysis. Annu Rev Biomed Eng. 2024;26(1):119-139.
White R, Sharkey C, Vyas J, et al. Transcriptomic profiling of T cell immune states before and after donor lymphocyte infusion in a patient with acute myeloid leukemia. Blood. 2025;146(Suppl 1):2566.
ARPA-H Contract No. 75N91024C00036. Advanced Research Projects Agency for Health. (Contract awarded to Feromics Inc., $4.1 million; publicly disclosed.)

Tania Konry, PhD, is the cofounder of Feromics and associate professor at Northeastern University.

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Back-to-School at USP

Edusehat — Sat, 15 Aug 2026 00:30:20 +0700

Brian McNally, PhD
Director, Global Biologics Marketing & Strategic Collaborations, USP

The pharmaceutical and biotechnology sectors are experiencing a historic wave of investment. Across the United States and around the world, companies are expanding manufacturing capacity, establishing new facilities, and accelerating development of innovative medicines ranging from monoclonal antibodies and antibody-drug conjugates to mRNA vaccines, gene therapies, and other advanced modalities.

Recent announcements show the scale of this momentum. In Virginia, AstraZeneca committed $4.5 billion near Charlottesville, Merck broke ground on a $3 billion manufacturing center in Elkton, and Eli Lilly announced a $5 billion facility in Goochland County. Lilly also announced a separate $3.5 billion Pennsylvania investment to support advanced therapies and injectable medicines. Together, these projects represent thousands of new jobs and more than $16 billion in capital investment.^1,2,3,4.

Yet facilities, technologies, and equipment are only part of the equation. Realizing the full value of these investments depends on something equally important: a skilled workforce capable of developing, manufacturing, testing, and regulating high-quality medicines.

This need has become a central theme across industry. At the BIO International Convention in June, workforce readiness featured prominently in discussions such as Future-Ready Solutions to the Global Biomanufacturing Talent Challenge. The message was consistent: maintaining growth in life sciences requires parallel investments in education, training, and talent development.

In many ways, the industry is already responding. In Virginia, AstraZeneca, Eli Lilly, and Merck have committed up to $120 million toward development of the Virginia Center for Advanced Pharmaceutical Manufacturing, a workforce initiative expected to support thousands of future workers through credentialing and education pathways.⁵ Similarly, Eli Lilly has partnered with Lehigh Carbon Community College in Pennsylvania to help establish a pharmaceutical workforce development hub aligned with regional manufacturing growth.

Importantly, this trend extends far beyond the United States. In Australia, the State of Victoria partnered with Monash University to establish the Monash Centre for Advanced mRNA Medicines Manufacturing and Workforce Training, supporting the growing ecosystem surrounding Moderna and BioNTech investments.⁶ In South Korea, Yonsei University’s K-NIBRT Education Center, developed in collaboration with Ireland’s National Institute for Bioprocessing Research and Training (NIBRT), was created to address anticipated shortages in biopharmaceutical manufacturing talent and provide hands-on training at scale.⁷

These examples illustrate a broader reality: successful pharmaceutical ecosystems require sustained investment not only in manufacturing infrastructure, but also in people.

Workforce development for quality

For USP, workforce development is closely tied to medicine quality and accessibility. As an independent scientific organization whose standards are used in more than 150 countries, USP has long viewed education as a critical enabler of quality across the pharmaceutical lifecycle.

The importance of training grows as technologies evolve. Analytical methods become more sophisticated, regulatory expectations expand, and novel therapeutic modalities introduce new scientific considerations. The ability to consistently produce quality medicines increasingly depends on access to current knowledge and practical training.

USP’s approach reflects a simple principle: workforce development should not be limited to a single audience or delivery model. Instead, it should serve the broader ecosystem responsible for bringing medicines to patients.

USP pursues this mission through three complementary lenses: broad-based professional education, regulatory capacity building, and practical training solutions.

Global manufacturing workforce support

The first lens focuses on providing education that can support professionals regardless of geography, employer, or therapeutic modality.

USP Education was established to help pharmaceutical scientists, quality professionals, manufacturers, regulators, and laboratory personnel effectively understand and apply public quality standards. The mission is straightforward: ensuring that the education required to interpret and apply USP quality standards remains accessible to those who need it. The program currently reaches more than 25,000 learners annually across thirty-eight countries.

Because a pharmacopeia’s primary role centers on quality standards and analytical science, much of USP’s educational portfolio focuses on analytical methodologies, quality systems, and the practical application of standards. Courses cover topics spanning compendial methods, microbiology, biologics characterization, pharmaceutical manufacturing, and regulatory science.

A distinguishing feature of the program is its connection to the standards development process itself. USP courses are developed by subject matter experts, either Expert Volunteers or USP staff, who are involved in creating USP standards. Course instructors are separately selected and qualified using a formal framework designed to assess both subject matter expertise and teaching capabilities.

Developing effective education, however, requires more than subject matter expertise. USP has invested considerable effort into the pedagogy behind its training programs. In a 2022 LTEN Focus article, USP Education leader Tim Greiner described a structured methodology for evaluating instructor qualifications that emphasizes both technical expertise and teaching effectiveness.⁹ The framework includes standardized instructor assessments, defined content ownership, feedback mechanisms, and continuous qualification processes designed to ensure consistency and instructional quality. This approach reflects a broader philosophy that educational excellence requires the same rigor applied to scientific and operational processes.

As the pharmaceutical and biotechnology industry’s workforce evolves, scalable training approaches such as online learning, virtual instruction, and blended educational models increasingly allow organizations to develop talent independent of location or facility-specific investments.

Strengthening regulatory capacity

Workforce development extends beyond manufacturers: medicine quality also depends on regulators who review applications, evaluate manufacturing processes, interpret analytical data, and oversee product quality throughout the lifecycle. For this reason, USP places significant emphasis on regulatory education and capacity building.

One recent example is USP’s growing collaboration with the African Medicines Agency (AMA). In April 2026, USP and AMA signed a Memorandum of Understanding establishing a framework for cooperation in regulatory science, pharmacopeial standards, capacity building, product quality monitoring, and regulatory harmonization across Africa.⁸ The partnership aims to strengthen regulatory systems and advance access to quality-assured medical products throughout the continent.

The agreement builds upon decades of USP engagement with national and regional regulatory authorities across Africa and complements broader efforts focused on local manufacturing, regulatory convergence, and post-market quality systems.

Strong science-based regulatory systems are increasingly important for advancing local manufacturing, enabling regulatory harmonization, and supporting access to innovative technologies. These activities help ensure that growing pharmaceutical manufacturing capacity is matched by corresponding regulatory readiness.

In the Asia-Pacific region, USP likewise supports regulator education through participation in the Asia-Pacific Economic Cooperation’s APEC regulatory training initiatives and centers of excellence. Training programs have included educational activities focused on advanced therapy products, including gene therapies. For example, USP recently conducted specialized training on qualification of raw and starting materials for gene therapies, helping regulators and industry professionals navigate evolving quality expectations associated with emerging therapeutic modalities.

By investing in regulator education alongside manufacturer training, USP seeks to strengthen the broader quality ecosystem that supports medicine availability and patient confidence.

Training beyond the classroom

As pharmaceutical manufacturing expands, workforce development increasingly requires more than traditional classroom-based instruction. Employers need personnel who can enter manufacturing environments with practical familiarity and direct experience relevant to current production technologies.

One example is the Maryland Tech Council’s BioHub Maryland program, which recently celebrated its first group of graduates. The program was designed in collaboration with regional life science employers and prioritizes curriculum development based on feedback from an industry advisory panel. This model helps to align workforce preparation with the skills most needed by local manufacturers.

Importantly, the curriculum adapts as the industry evolves. With companies such as Kite Pharma expanding cell therapy manufacturing capabilities in the region and AstraZeneca announcing significant investments in advanced therapeutics, BioHub has incorporated training focused on cell therapy production and microbial contamination control. By connecting educators directly with employers, programs such as BioHub help ensure that workforce development efforts remain relevant to emerging manufacturing needs.

These initiatives illustrate an important principle: workforce development is most effective when educational institutions, industry, regulators, and scientific organizations work together to align training with evolving technologies and quality expectations.

While hands-on programs provide valuable experiential learning, organizations may also seek objective ways to measure competency as personnel begin performing critical activities. In high-growth manufacturing environments where employee turnover and rapid hiring may occur, objective competency assessment tools can provide additional confidence that critical techniques are being performed correctly and consistently.

Importantly, these tools do not replace training. Instead, they complement educational programs by helping organizations measure practical performance and reinforce quality-focused behaviors.

A holistic model

The pharmaceutical sector’s expansion presents an extraordinary opportunity. Billions of dollars are flowing into new manufacturing facilities, advanced technologies are progressing across biologics, mRNA therapeutics, cell and gene therapies, and precision medicines, and regional life sciences ecosystems continue to grow around the world.

Realizing the long-term value of these investments will require a workforce prepared to sustain them.

Examples from the United States, Australia, South Korea, and many other regions show that workforce development is increasingly recognized as a strategic necessity, not a supporting activity.

USP’s experience points to a holistic model: accessible education for industry professionals, support for regulators who shape medicine quality ecosystems, and practical tools that reinforce competency and continuous improvement.

As students return to classrooms and professionals continue building new skills, the life sciences industry is entering its own global back-to-school season. Facilities may be new, and technologies may evolve, but the objective remains constant: ensuring that high-quality medicines reach patients everywhere.

And that objective starts with people.

References

Eli Lilly Pennsylvania manufacturing investment announcement (January 2026)
Eli Lilly Virginia manufacturing facility announcement (September 2025)
AstraZeneca Virginia manufacturing investment announcement (October 2025)
Merck Elkton, Virginia manufacturing center announcement (October 2025)
Virginia Center for Advanced Pharmaceutical Manufacturing MOU announcements (October–November 2025)
Monash University and Victoria Government mRNA workforce training initiative
Yonsei University K-NIBRT Education Center and NIBRT partnership
African Medicines Agency–USP Memorandum of Understanding (April 2026)
Tim Greiner, “The Who, How and When of Instructor Qualification,” LTEN Focus Magazine (Fall 2022)
AMA–USP Regulators Forum educational program materials

Brian McNally, PhD, Director of Global Biologics Marketing & Strategic Collaborations at USP

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SFA Spectroscopy Chases Away Cloudy Samples

Edusehat — Sat, 15 Aug 2026 00:30:16 +0700

Brendan Darby, PhD
Co-founder and CEO
Marama Labs

Characterizing the RNA and ligands in nanoparticle-based therapeutics has, historically, been a cumbersome process. The lipid nanoparticles (LNPs) that encase RNA and payloads are cloudy by nature, which means analysis can’t be accomplished using the UV/Vis spectrometers that are ubiquitous throughout the life sciences industry.

Marama Labs has developed a technology that eliminates the light scatter in cloudy samples—even in samples as cloudy as milk—thereby “unlocking a huge capability for complex particle analysis,” Brendan Darby, PhD, co-founder and CEO of Marama Labs, tells GEN.

The technology, CloudSpec, employs scatter-free absorbance to remove the signal interference caused by a solution’s cloudiness, in effect, chasing away the clouds. To do this, Darby and colleagues created a highly reflective spherical chamber called an integrating sphere, which harnesses the properties of physics—specifically, the Beer-Lambert Law regarding light attenuation—and an algorithm to precisely measure analyte concentrations. This enables clean measurements similar to those of a standard spectrometer. Using this device, LNP lysing and fluorescent dyes are unnecessary for payload quantification.

Research from Victoria University of Wellington (New Zealand), University College Cork (Ireland), and Marama Labs indicates this analysis method outperforms fluorescence-based quantification assays in terms of precision and agreement with expected RNA concentrations.

CloudSpec delivers results across the 240 to 850-nm spectral range that are “within 10% of the nominal value expected for RNA concentration,” Darby says. And, he adds, “The technique is very precise.” It’s also fast and simple. Sample dilution is the only preparation needed, and the measurement itself takes only 15 seconds.
Alternative technologies, in contrast, he says, can involve eight to 10 steps and between 30 minutes and two hours to analyze each sample, with significant variability between samples and also between operators.

Marama Labs’ CloudSpec scatter-free absorption spectroscopy technology eliminates the light scatter in cloudy samples, making complex particle analysis possible even for extremely cloudy samples. [Marama Labs]

Physics, wine, and RNA

The idea for this novel quantification technology emerged during Darby’s doctoral work in the RAMAN Lab of Eric Le Ru, PhD, professor at Victoria University of Wellington, now Marama Labs’ CSO.

“We were trying to understand how light interacts with nanoparticles from a very fundamental physics point of view,” Darby recalls. “We were looking at the fundamental optics of scattering and light absorbance with nanoparticles and found that what we were trying to measure was impossible with the existing instruments at our university. We realized that light scattering was causing a major source of error in measurements.”

“Most liquids are cloudy by nature, unless they are purified in a lab or filtered. When we (produced) the same quality and repeatability as UV/Vis, but in unprocessed samples, we immediately understood that this technology could potentially become the gold standard for characterization of complex samples,” Darby says. UV/Vis, he explains, measures extinction—both scatter and absorption. CloudSpec, however, measures extinction and absorption directly and uses those measurements to determine scattering.

At that point, Darby, Le Ru, and Matthias Meyer, PhD, CTO and co-founder, began the process of forming Marama Labs, which was incorporated in 2019.

In his thesis research, Darby measured molecules on the surface of metallic nanoparticles at ultra-low, previously unmeasurable, concentrations accurately. That finding was extended to RNA therapeutics and validated in collaboration with CureVac researchers.

From the beginning, though, the team understood that CloudSpec’s greatest contributions would be in the life sciences, but its first applications were in the New Zealand wine industry.

“Winemakers in New Zealand are very technology-centric and innovative,” he says. At the time, “There was no easy method to measure color and tannin at the very earliest stage of winemaking. They used their palettes and their eyes.” CloudSpec’s advanced analytics provided the data that those winemakers needed to better control their processes and thus craft the style of wine they needed for their market needs, while, he says, “being respectful of the grape.”

To transition to life sciences, the challenge was how to evolve the technology and apply learnings from the wine industry to the pharmaceutical industry. “Our primary focus is in life sciences and RNA therapeutics,” he reiterates.

Formulations to QA

Currently, most of CloudSpec’s applications are in therapeutic formulation development. There, this technology helps companies optimize formulations to provide both effective nanoparticle delivery and effective therapeutic response. Additional applications include manufacturing and quality assurance.

CloudSpec technology is currently single-parameter focused, measuring RNA and targeting-ligand concentrations. While these are quite important, they are only one of several chemical and physical parameters that must be measured. The size of the nanoparticles, their distribution, and surface charge, for instance, are among the critical quality parameters that biomanufacturers must measure.

With that in mind, Darby hints that an additional measurement capability “for one of the most widely-used attributes in the LNP formulation space” may be added to the CloudSpec soon. “It’s been demonstrated and validated internally that CloudSpec can do this measurement, and do it faster and more repeatably than the existing method. We don’t just add on capability for the sake of adding,” he stresses. What that new measurement is couldn’t be released at the time of the interview.

In addition to expanding its technology, Marama Labs is also growing its global presence. Its core research and development team and manufacturing facilities are based in Wellington, NZ. Darby heads the company’s commercial and strategic development from his office in Dublin. The company also recently opened an office in Boston to support its growing customer base there.

That combination provides access to the major European and North American markets while maintaining close ties to its innovation team, effectively chasing away the clouds wherever LNP analysis is performed.

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Newly Discovered Role for Polyamines Could Point to Potential Anticancer Strategies

Edusehat — Sat, 15 Aug 2026 00:30:13 +0700

Our cells need iron to produce energy, carry oxygen throughout the body and power countless chemical reactions that sustain life. But when too much iron is left free inside cells it can trigger destructive reactions that break down DNA, proteins, and even cell membranes.

Researcher headed by a team at the Whitehead Institute have now discovered that cells rely on small molecules called polyamines as an unexpected protector against this threat. The newly reported study by White Institute member Ankur Jain, PhD, together with former postdoc Whitney Henry, PhD, and graduate student Pushkal Sharma, and colleagues, revealed that polyamines act like storage lockers for iron, safely holding the metal in a non-reactive state until cells need it. As part of their research the team developed a genetically encoded fluorescent reporter allowing them to quantitatively measure redox-active iron in living cells.

The collective findings solve a decades-old mystery about why cells maintain such extraordinarily high levels of polyamines and also uncover a previously unknown defense mechanism that protects cells from toxic iron overload. The discovery could potentially help scientists develop better cancer treatments, by allowing iron overload to trigger cancer cell death. The study results could also offer up new clues about disorders such as early-onset Parkinson’s disease, in which mutations affect polyamine levels within neurons.

Jain and Henry are co-senior and co-corresponding authors of the team’s published paper in Cell titled “Polyamines buffer labile iron to suppress ferroptosis,” in which they stated that the findings “… reposition polyamines as key regulators of iron homeostasis, with implications for ferroptosis-linked disease states and cellular redox balance.”

The Jain Lab studies RNA and is particularly interested in how RNA folds, misfolds, and sometimes clumps inside cells. Jain and Sharma first began studying polyamines because these molecules bind to RNA and help to shape its structure. However, they suspected that polyamines must be playing other roles inside cells. Polyamines are among the most abundant small molecules within cells, present at levels comparable to ATP, the molecule that cells use as their energy currency.

“We’ve known that without polyamines, cells stop growing and dividing,” explained Jain, who is also an associate professor of biology at the Massachusetts Institute of Technology (MIT). “But their best-known function only requires a small fraction of the polyamine levels cells actually have.”

To uncover the hidden function of polyamines inside cells the researchers used a large-scale genetic approach that allows them to screen the entire genome at once, rather than testing genes one-by-one, in order to find out which cellular processes are impacted when polyamine levels are changed within cells. “To identify cellular dependencies that emerge during polyamine stress, we performed a genome-wide CRISPR-Cas9 screen in human cells under polyamine-depleted conditions,” they noted.

The screen revealed that when cells have reduced levels of polyamines, a protein called GPX4 becomes essential for survival. GPX4 is known to prevent harmful chemical reactions that damage the fatty molecules that make up cell membranes. “This synthetic lethal screen revealed a surprising link between polyamines and iron homeostasis: polyamine depletion rendered cells highly dependent on glutathione peroxidase 4 (GPX4), an antioxidant enzyme that protects membranes from lethal lipid peroxidation and ferroptotic cell death,” the team added.

The team also found that cells with lower polyamine levels have higher amounts of another protein that acts as an iron sponge and keeps the metal in a mineralized form. Together, these findings led the researchers to hypothesize that polyamines might be helping keep iron in a safe, non-reactive state within cells.

To test this idea, they developed a new fluorescent sensor that would allow them to measure chemically reactive iron inside living cells. The new sensor causes living cells to glow based on the amount of chemically reactive iron they contain, allowing researchers to track any changes under a microscope in real time.

The team paired the new iron sensor with another sensor they had previously developed that measures polyamine levels within cells. By employing them simultaneously, they observed a striking pattern: as polyamine levels dropped within cells, the amount of chemically reactive iron went up, offering new evidence that polyamines play a key role in preventing toxic iron build up inside cells. “Single-cell analysis revealed that labile iron levels increase as polyamines decline,” the team wrote in summary. They suggest that their collective findings “… support a model in which millimolar polyamines help restrain labile iron availability, linking polyamine metabolism to redox balance and ferroptosis sensitivity … Altogether, these data demonstrate a tight, inverse coupling between polyamine and labile iron levels at the single-cell level and establish our genetically encoded reporter as a robust tool for the quantitative dissection of iron biology.”

Beyond answering a fundamental biological question, these findings could have implications for cancer treatment. Cancer cells often rely on high polyamine levels to support their rapid growth and division. However, cancer drugs designed to lower polyamine levels to stop cell division have had limited success.

“We saw that when polyamine levels fall, cells rely on GPX4 to protect themselves from iron toxicity,” said first author Sharma. “This could mean that combining drugs that lower polyamine levels with those that block GPX4 might be more effective for killing cancer cells than targeting either pathway alone.”

The discovery may also have implications beyond cancer. Mutations in genes that help move polyamines around cells are linked to a rare form of early-onset Parkinson’s disease, and scientists have long observed unusually high levels of iron in the brains of Parkinson’s patients.

While it is still unclear whether excess iron directly contributes to neuron death in Parkinson’s, the discovery that polyamines help buffer reactive iron inside cells offers a possible explanation for this link and opens new directions for future investigation.

In addition, the researchers expect the new iron sensor to be a valuable tool for other scientists. By allowing them to track chemically reactive iron inside living cells, it could power new discoveries in aging, cancer, and neurodegeneration. “We anticipate that this reporter will provide a powerful platform to facilitate future discoveries into iron biology, ferroptosis, and disease mechanisms,” they stated.

“There are a lot of promising future directions for this work,” Jain noted. “It’s exciting to think about how these tools and findings could help answer further questions about disease pathways and potentially help design better therapies.”

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BMS’s New Facility, Recursion–Genentech Partnership, Long-COVID and Persistent C. auris

Edusehat — Sat, 15 Aug 2026 00:30:10 +0700

Bristol Myers Squibb has selected Houston as the site for a new $2.3 billion multi-modal manufacturing campus designed to support drug product and finished goods manufacturing from late development through launch. Recursion, Roche, and its Genentech subsidiary have announced plans to co-develop a neuroscience discovery program based on the first validated target discovered through an AI map. Editors reminisce over the changes in science since the COVID-19 pandemic in light of a new large NIH-funded study that shows COVID-19 can reactivate dormant viruses, including Epstein-Barr and cytomegalovirus. In other infectious disease news, drug-resistant Candida auris was found to persist in hair follicles by exposing chitin, triggering interferon-γ that suppresses skin defenses and creates a niche for fungal colonization.

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

BMS Chooses Houston for $2.3B Manufacturing Facility
By Alex Philippidis, GEN Edge, Aug 10, 2026

Next 10 U.S. Biopharma Clusters
By Alex Philippidis, GEN, Aug 12, 2026

Recursion Partners with Genentech to Advance First Validated Neuro Target Discovered Through AI Map
By Alex Philippidis, GEN Edge, Aug 5, 2026

COVID-19 Reactivates Dormant Viruses, Offering New Clues to Long COVID
GEN, Aug 5, 2026

Trump’s Treatments: Regeneron’s Antibodies and Gilead’s Remdesivir Explained
By Alex Philippidis, GEN, Oct 5, 2020

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This scientist is helping build a missing map of childhood

Edusehat — Fri, 14 Aug 2026 21:00:03 +0700

In 2017, Deanne Taylor attended a presentation at the University of Pennsylvania, just a short walk from her office. A researcher was there to unveil the Human Cell Atlas, an ambitious project that aimed to map every cell in the human body. Taylor was floored, and then concerned. As details emerged, she discovered that the project’s researchers had only made plans to study adults. “That’s when my little alarm went off,” she says. “Not again.”

Since joining the Children’s Hospital of Philadelphia (CHOP) as the director of bioinformatics three years earlier, Taylor had been disappointed by the lack of investment in medical research focused on children. The dominant view, she says, was that children are exactly like small adults. They’re not. Children’s cells are different from grownups’ cells in the way they express genes—switching them on and off or turning them up or down. Those variations can cause drastically different and even deadly responses to drugs that adults tolerate well.

The 2017 talk was the moment Taylor didn’t know she’d been waiting for. She quickly channeled her concern into a campaign, joining the Human Cell Atlas’s volunteer team and helping write a section on children for a white paper outlining the group’s goals and plans. She then rallied a cross-hospital coalition of pediatric researchers to contribute to the project and spearheaded a 2019 paper that outlined the case for studying children—a bid to attract more interest and funding to the field. “It put a flag in the ground,” she says. “Why don’t we have healthy models of children’s development?”

So far, the push has paid off. In 2021 the NIH awarded a $38.5 million grant to the Developmental Genotype-Tissue Expression Project (dGTEx), a major initiative aimed at establishing the first comprehensive database of healthy pediatric tissue. The project banks samples collected from otherwise healthy children who have died and whose parents agreed to donate their bodies, and maps how genes across all the major organ systems are expressed. Taylor and her team curate and standardize the information associated with each tissue donation, including family history and details about the samples. A separate group does analysis on the samples themselves, and then all the information is combined to create a database—a baseline of what gene expression looks like in children. It’s the first step to enabling research that could advance our knowledge of normal development, disease, drug effectiveness, and other phenomena.

The dGTEx team will eventually feed its data into the Human Cell Atlas, which, thanks to Taylor and many of the coauthors of the 2019 paper, now includes a pediatric section.

Taylor’s primary responsibility may be collecting and organizing data for dGTEx, but colleagues say she’s also the glue holding diverse research projects together. That’s especially important for the Human Cell Atlas, which depends on contributions from a loose coalition of researchers, all pursuing their own objectives. “Deanne took a big-picture view and said, We don’t just need to understand the pediatric kidney or the pediatric brain or the pediatric immune system. We need a holistic view of pediatric development,” says Sarah Teichmann, a cofounder of the Human Cell Atlas. “She embodies that interdisciplinary spirit.”

A healthy baseline

Taylor describes her career as a “random walk,” driven by a singular intensity she now attributes to undiagnosed autism and ADHD. At five, she began reading her mom’s medical texts. By 12, she was checking out physics books from the library. Physics provided mysteries to solve, and she wanted to understand how things worked.

Taylor got her PhD in biophysics, in 2001, but was inspired by the then-active Human Genome Project to change gears and take on a postdoc at Pfizer, writing code to handle complex data in rare-disease research. Then she moved to reproductive medicine, where she worked on some of the first computer programs to screen embryos for chromosomal abnormalities—many of which are still in use today.

HANNAH YOON

Despite this seemingly winding road, Taylor says her focus has always been on understanding why the same illness hits people differently. How can two people carry the same disease-associated gene variant, but only one get sick?

The Human Cell Atlas—including all the data feeding into it from dGTEx and other projects—could at last help researchers find answers. The effort is a natural extension of the Human Genome Project. That initiative, which wrapped up in 2003, helped researchers link specific genes to specific diseases. But a map of the genome is a bit like a DIY kit with all the parts and no assembly manual. It doesn’t tell you where and how cells use each gene throughout the body.

After all, “we’re just older kids,” Taylor says. “By ignoring the pediatric side of things, I think people are missing a window of intervention in human disease.”

For that, you need to know how the genes are expressed. Gene expression generally involves making a protein that does a specific job in the body, like building tissue or sending signals. Unlike DNA, which largely remains the same throughout our lives, the way the genes in DNA are expressed changes as we develop.

Differences in gene expression can determine whether a therapy will work—or could harm more than it helps. Because of the way cardiac genes are expressed in children, chemotherapy drugs can attack not only tumors but also children’s developing hearts, potentially causing lifelong damage. Other treatments can affect the entire body, sometimes triggering a reversible but potentially fatal immune-system reaction called cytokine release syndrome.

The dGTEx database aims to create a baseline for gene expression in children—a molecular map of how the body’s roughly 20,000 genes do their work in healthy tissue cells. It is only one of the collaborations Taylor manages. She’s a principal investigator for the Kids First Data Resource Center, which sequences diseased tissues collected from children enrolled in other studies nationwide. And she has been collaborating with researchers on HubMAP, an effort that’s building a resource complementary to the Human Cell Atlas, to secure funding to create 3D maps of children’s cells like the ones it’s already made for adults.

Extending such initiatives to children is important, Teichmann argues. Much of human development happens in childhood; key brain cells called astrocytes form in the first five years, for instance, and the immune system matures in puberty. “Those changes are really important to understand from a disease point of view,” she says. A granular view of how individual cells work “will change pediatric medicine, for sure.”

Herding cats

Taylor helps the dGTEx machine run, coordinating researchers across multiple organizations that each contribute different pieces to the puzzle. These include a nonprofit group that secures tissue samples from deceased children soon after death and CHOP pathologists who assess each sample’s quality and type. Tissues are frozen and stored for future researchers to use with the group’s permission, while samples are sent to organizations including the nonprofit Broad Institute, which analyze gene expression. Data streams in at all these steps—information that the Human Cell Atlas effort can eventually draw on.

This coordination is “like herding cats,” says Rebecca Linn, a pediatric pathologist at CHOP. “So many individuals with different goals.” Taylor says an important part of her role is mediating among participants. That means, for example, explaining to researchers who want to use dGTEx’s tissues that it’s impossible to divide a one-month-old’s tiny testes 20 ways.

Colleagues describe Taylor as a well-connected collaborator who unites people across diverse specialties—essential qualities for a multidisciplinary, international effort like the Human Cell Atlas. It also helps that Taylor is full of surprises. She has tattoos of Schrödinger’s and Boltzmann’s equations and dabbles in painting and photography; a nondescript rock from Burning Man, where she volunteered in the kitchen, sits on her desk. “She can make friends and be memorable through her interests and knowledge and questions about all these different subjects. It really draws you in,” says Linn.

Taylor, however, believes the life-changing potential of the work itself is enough to motivate colleagues. Comparing a sick person’s cells with the healthy, age-matched baseline the Human Cell Atlas provides could yield biomarkers of health and disease that could serve as drug targets or diagnostic markers. A pediatric chapter in that atlas could produce similar insights for children—and strengthen our understanding of how our genetics and environments affect health and disease at various stages of development.

Extending the atlas to children may even help reveal how adult diseases trace back to distinct signals in childhood, raising the possibility that we could screen for and treat chronic conditions years or even decades before they surface. That could not only improve outcomes but help people prevent debilitating symptoms before they ever develop. After all, “we’re just older kids,” Taylor says. “By ignoring the pediatric side of things, I think people are missing a window of intervention in human disease.”

Taylor hopes the project will shift how research views pediatrics. It’s a big goal, one that will require big data—and forces like her to help pull everything together.

Colleen de Bellefonds is a science journalist based in Paris.

Cloning could be used to save species—or make human “organ sacks”

Edusehat — Fri, 14 Aug 2026 21:00:03 +0700

This week I spoke to scientists who have found a way to turn male mouse embryos female. They’ve developed a CRISPR-based approach to essentially cut out the Y chromosome. It allowed them to create female clones of male mice.

That’s right: female animals that are genetically identical to males, except for the missing Y chromosome. Takashi Ishiuchi, a reproductive biologist at the University of Yamanashi who co-led the work, told me it felt a bit like sci-fi.

Ishiuchi and his colleague Shogo Matoba of the Riken BioResource Research Center hope their approach could be helpful in conservation efforts, especially in cases where we might have only a few individuals of a species left. But cloning has multiple uses, ranging from the cool to the outright creepy.

We can’t talk about cloning without mentioning Dolly, the celebrity sheep born in 1996 and the first mammal successfully cloned from an adult cell. In that case, scientists took the DNA-containing nucleus of an adult mammary cell from one sheep and transferred it into an egg cell that had had its own nucleus removed. The resulting embryo was transferred to a surrogate sheep, which gave birth to Dolly—an animal genetically identical to the DNA donor.

The scientists behind that work were interested in genetically modifying livestock. Farmers have essentially been doing this for thousands of years through selective breeding, but cloning allows scientists to create genetic replicas of animals with desirable traits.

Cloning is also being used to replicate deceased pets, including, famously, those of Barbra Streisand and Tom Brady, among others. For a price somewhere in the tens of thousands of dollars, a company can take cells from your pet and turn them into a living, breathing clone.

Considering that cloning also requires egg cells from another animal, and a surrogate animal to carry the pregnancy, not everyone is on board with this, especially since there is no medical or environmental need for the procedures. One bioethicist, Jessica Pierce, has described this aspect of dog cloning as “the exploitation of the canine underclass.”

The case for cloning is stronger when it comes to conservation—where some argue there is environmental value.

Scientists have been preserving animal tissues for years. Some of these tissues are cryopreserved at low temperatures in “frozen zoos.” The facility at the San Diego Zoo, for example, currently has cells from over 1,300 species. Some of these samples were taken decades ago.

Preserved tissues like these have enabled scientists to create clones of animals considered close to extinction, including black-footed ferrets and Przewalski’s horse. But they might also help us bring back extinct animals.

In 2009, researchers in Spain described how they’d cloned an extinct wild goat, the Pyrenean ibex, using skin cells that had been cryopreserved a decade earlier. In that research, the team used egg cells from domestic goats to create a total of 439 embryos. Ultimately, only one goat—a female—was born. She died minutes later because of a defect in her lungs.

Poor Pyrenean ibex. It’s the only animal we know of that has gone extinct twice.

The biotech company Colossal Biosciences is hoping to use old—and potentially ancient—genetic material to bring back long-extinct species like the thylacine and woolly mammoth. So far, the company’s efforts have largely involved modifying the genomes of modern-day animals.

Technically, it’s also possible to clone humans. As far as we know, no one has done it. But some have played with the idea. One biotech startup founder has pitched an idea for “brainless clones”—human clones that lack a brain but contain all the organs people might need to replace their own in future. My colleague Antonio Regalado described that pitch in March. (I had to pause eating my lunch while rereading it.)

Scientists have done a hell of a lot with cloning over the last few decades. I’m excited—but also slightly nervous—about what the coming decades will bring.

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.

Studies show value of biotech IP for the economy and society

Edusehat — Fri, 14 Aug 2026 17:25:03 +0700

Biotech innovation provides better-than-average salaries and pumps hundreds of billions of dollars into the U.S. economy annually—and the societal benefits of this innovation can be measured in hundreds of trillions, according to recent research.

Industries based on intellectual property, including biotech, are responsible for $11.4 trillion in U.S. gross domestic product, or 44% of total private sector GDP, according to a report released by the U.S. Patent and Trademark Office (USPTO) on Aug. 3. Out of this total, pharmaceuticals contributed $247 billion to the U.S. GDP in 2024, the report says.

When looking at the broader societal value of biotech, the impact is much greater, according to a recent analysis produced by a University of Chicago economist for the U.S. Chamber of Commerce.

“Medical innovations in four disease areas: HIV, heart disease, breast cancer, and obesity generated $167.5 trillion in societal value over a 30-year horizon across all four disease areas,” the U.S. Chamber report finds.

The importance of patents to biotech

The USPTO report highlights the importance of patents to the overall U.S. economy, with patent-reliant industries providing 65.8 million jobs, or 44% of total private sector jobs. These are also better-paying jobs, “with average weekly earnings 53% higher than those received by workers in other industries,” USPTO says.

Pharmaceuticals rely on several types of IP, according to the report, including utility patents, design patents, and trademarks.

The biopharma sector is one of the most research-intensive industries, which means IP provides its value. When a biotech firm sells investors on the promise of an innovative new drug, what they are really selling is IP developed through research. That’s why biotech firms need a strong patent system to safeguard their IP. Research has shown that venture capital funding for life sciences startups plummeted after legal changes weakened the patent system.

Given the importance of IP to biotech, the Biotechnology Innovation Organization (BIO) advocates policy to strengthen patent protections. BIO continues to expand its efforts in this area, working on Capitol Hill, through courts, and in international fora, with initiatives like BIO’s IP Task Force.

The importance of biotech to society

Beyond providing jobs, biotech provides treatments that patients need. The U.S. Chamber of Commerce report looks at the social benefits of medical innovation and assesses the monetary value of “saving lives, extending lifespans, and driving economic growth through increased productivity and tax revenue.”

The study analyzes the period 30 years after the introduction of breakthrough therapies for HIV (1995–2024), heart disease (1986–2024), and breast cancer (1991–2020), and projects the benefits of obesity drugs (2021–2050).

“HIV patients who would have died within two years in 1995 now live full lifespans—an average of 40 additional years,” says a summary of the report. It notes that heart disease and breast cancer patients are living years longer. It projects that “new obesity treatments are poised to add nearly a year of life while dramatically reducing the chronic disease burden that has plagued millions of Americans.”

Based on these findings, “every dollar invested in medical R&D has returned 27 dollars in societal value—demonstrating that innovation is an investment, not a cost,” according to the U.S. Chamber.

As these studies underline, biotech’s productive output, job creation, and additional years of healthy living provide huge benefits for America’s economy and society.

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Pangenome-Guided Breeding Boosts Yield and High-Altitude Adaptation in Buckwheat

Edusehat — Fri, 14 Aug 2026 10:10:10 +0700

Agricultural genomics is beginning to move beyond the limits of a single “reference” crop genome. In a proof-of-concept study, researchers used pangenome-guided breeding to recover useful DNA variation left behind during domestication and combine two traits that often work against each other in the field: high-altitude adaptation and yield.

The study, “Pangenome-guided breeding restores high-altitude adaptation and improves yield in Tartary buckwheat,” was published in Cell. The international team was led by the Chinese Academy of Agricultural Sciences and included researchers from 22 institutions across 10 countries, including scientists at Murdoch University’s Centre for Crop and Food Innovation (CCFI). Tartary buckwheat (Fagopyrum tataricum), a nutrient-dense grain grown in the Himalayan highlands, was selected as a model because its wild relatives tolerate harsh conditions such as cold and intense UV-B radiation.

Most genomics-assisted breeding compares crop lines with a single reference genome and focuses heavily on single-nucleotide polymorphisms. But that approach can miss larger structural variants, including gene copy-number changes, that influence agriculturally important traits. To capture that hidden variation, the team generated a telomere-to-telomere reference genome and assembled a graph-based pangenome from 16 accessions spanning Himalayan wild populations and globally distributed landraces. They also integrated genomic data from 994 accessions across 15 countries and cataloged 123,131 nonredundant structural variants.

“Most modern breeding compares a crop’s genome against a single reference, which is like judging a language by a single dictionary,” said Rajeev Varshney, FRS, FAA, CCFI director, and co-corresponding author of the study. “A pangenome captures the whole vocabulary, including the words a crop lost along the way. And that’s where a lot of the useful genetics for resilience is hiding.”

CCFI contributing authors Rajeev Varshney, Anu Chitikineni, and associate professor Reyazul Rouf Mir pictured by drone at a field trial for wheat in Northam, Western Australia. [CCFI]

The analysis identified FtRNH, a wild-specific gene present in high-altitude wild plants but missing from cultivated varieties. The gene is associated with the repair of UV-B–induced DNA damage and enhanced high-altitude adaptability. The researchers also identified structural variation at the FtPLATZ locus, including copy-number variation and a 28-bp promoter insertion linked to seed-size variation.

Using marker-assisted selection, the team crossed these superior FtRNH and FtPLATZ alleles into candidate breeding lines, effectively stacking high-altitude adaptation with larger seed size. In high-altitude field trials, the resulting lines showed improved growth, larger seeds, and significantly higher yields compared with the standard variety.

“Resilience and yield are usually a trade-off, meaning that when you increase one, you lose the other,” Varshney said. “What the pangenome lets us do is identify the specific DNA segments underlying each trait and deliberately stack them. That’s a template other breeding programs can follow.”

Whether the approach will translate broadly remains to be tested, but the study provides a framework for looking beyond elite crop genomes. By capturing variation in wild relatives and landraces, pangenomes may give breeders a clearer view of alleles that could be combined to improve adaptation without sacrificing yield.

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Thermo and Michael J. Fox Foundation Collaborate to Advance Proteomics-Enabled Parkinson’s Therapy

Edusehat — Fri, 14 Aug 2026 10:10:09 +0700

Thermo Fisher Scientific completed an Olink Explore HT proteomic analysis of approximately 5,500 research samples from the Michael J. Fox Foundation’s (MJFF) landmark study, the Parkinson’s Precision Medicine Initiative (PPMI). The resulting data are now in PPMI’s data repository, where they are available to the global research community.

The project creates a large-scale proteomics resource designed to help researchers study Parkinson’s disease at the protein level and investigate biological signals associated with disease progression, patient heterogeneity, and potential biomarker development. By applying Olink technology to deeply characterized PPMI samples, Thermo Fisher officials said the company is expanding access to MJFF’s dataset that may help advance precision medicine approaches to Parkinson’s research.

Deep proteomic insights

An estimated 10 million people worldwide are living with Parkinson’s disease, and that number is expected to rise as populations age. Although the disease is still diagnosed and monitored largely through clinical symptoms, research increasingly shows Parkinson’s disease involves multiple biological pathways and distinct patient subtypes. That complexity has made it difficult to develop biomarkers that reliably identify patient groups, track disease progression, and support more targeted therapeutic development.

As the field shifts toward biology-driven precision medicine, large-scale proteomic datasets are helping researchers better understand disease biology and generate hypotheses that can be tested across cohorts and methods.

Olink, part of Thermo Fisher, was designed to enable high-throughput, affinity-based proteomic analysis using Proximity Extension Assay (PEA) technology. Olink’s PEA-based platform is a scalable solution for protein biomarker analysis, supporting applications from discovery through translation and population-scale proteogenomics, according to Thermo.

Proteomics can help researchers identify patient subtypes, characterize disease progression, uncover biological pathways linked to inflammation, lysosomal function, and neuronal stress, and discover candidate biomarkers for future validation. Combining proteomic insights with longitudinal clinical, genetic, imaging, and other molecular data can further enrich understanding of health and disease.

Defining Parkinson’s by its underlying biology

PPMI, sponsored by the Michael J. Fox Foundation, launched in 2010 and was recently renamed the Parkinson’s Precision Medicine Initiative to reflect the field’s shift toward defining Parkinson’s by its underlying biology rather than clinical symptoms alone. PPMI’s open-access data are available to researchers worldwide and have been downloaded more than 50 million times.

“Parkinson’s disease is incredibly complex, and understanding the biological changes that drive its onset and progression requires looking across many layers of biology,” explained Samantha Hutten, PhD, principal biomarker scientist, translational research, MJFF. “This is what the Foundation’s global PPMI study was built to do. By expanding proteomic analyses within the study, including through partners like Olink, we’re creating new opportunities to identify biomarkers and uncover pathways that may lead to earlier diagnosis, better disease monitoring, and more targeted therapeutic approaches for people living with Parkinson’s disease.”

The analysis was completed as proteomics and multi-omics approaches gain broader use in neurodegenerative disease research. These methods can reveal dynamic biological changes that may not be captured through genomics or clinical assessment alone. Biobanks and large population studies are also adopting proteomic analysis to build richer molecular datasets that support biomarker discovery and precision medicine.

“Discovery is only the beginning in Parkinson’s research,” said Yan Zhang, PhD, president of proteomic sciences at Thermo Fisher. “The next challenge is determining which molecular signals are reproducible, clinically meaningful, and useful for advancing Parkinson’s precision medicine. Making these data available to the research community helps put that validation work into motion.”

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The Coming Shift from Biomarkers to Biological Trajectories

Edusehat — Fri, 14 Aug 2026 06:35:12 +0700

Diana Abdueva, PhD
Founder and CEO, Aqtual

For most of modern medicine, we have treated disease as a static condition. A patient has cancer. A patient has rheumatoid arthritis. A patient has inflammatory bowel disease. Diagnosis has largely meant identifying the condition, assigning a label, and selecting a treatment based on the standard of care.

But disease is not static, it is a dynamic biological process that evolves over time. Biology operates through change, and what we call a disease is often just the name we assign to a single point along a continuous biological journey, shaped by interactions among cells, tissues, and the immune system, by environmental exposures, and by the selective pressures of therapy itself. The lines we draw between health and disease, response and progression, are snapshots of a process that never stops moving.

Seen this way, the most consequential questions in healthcare are not about presence but about direction. Where is the patient today? Where are they heading? Which future states remain possible, and how can we change the path? The next era of medicine will be defined not by earlier detection alone, but by the ability to measure biological trajectories and ultimately intervene to change them.

A state-transition problem

Consider the decisions that actually determine patient outcomes. Diagnosis asks what biological state a patient is in. Therapy selection asks which intervention is most likely to move them toward a better one. Treatment monitoring asks whether they are moving in the expected direction. Drug development asks how an intervention alters biological trajectories across an entire population.

These look like distinct challenges, but at their core they are the same problem: understanding how biological systems move from one state to another. Yet most of our measurement tools were never designed to answer that question. They were built to detect the presence of something—a mutation, a protein, a lesion, a biomarker. Those measurements have transformed medicine, but they are static snapshots. They tell us what is present at a given moment far better than they reveal what is changing, why, or where the biology is headed next.

The challenge facing medicine is no longer detection alone. It is understanding dynamics.

A larger transformation

The evolution of liquid biopsy mirrors this broader shift. In 1997, Dennis Lo, DM, DPhil, and colleagues showed that cell-free fetal DNA circulates in maternal plasma, establishing that biological information could be read non-invasively from blood. The first generation of tests asked a simple question: Can disease-associated DNA be detected non-invasively?

The second generation moved from detection to sequence. Circulating tumor DNA assays identified cancer-associated mutations, monitored molecular residual disease, and flagged emerging resistance. The third generation went further still, using methylation and fragmentomic patterns to infer tissue of origin—which organs or cell types had contributed DNA to circulation.

Each advance extracted more information from a single blood draw. But all three generations remained focused on what was present and where it came from. A more fundamental question is now emerging: What was happening inside the cell before that DNA entered circulation? Not which tissue released it, nor which mutations it carried, but the functional state that generated the signal in the first place. Answering that question moves liquid biopsy beyond measuring static signals and toward measuring the biological processes that produced them.

Tissue of origin to biological state

The field has already begun to move past the question of where DNA comes from. The harder question is what those tissues were doing. Disease is rarely the work of isolated cells acting alone. Cancer emerges through interactions among tumor, stromal, and immune compartments. Autoimmune disease arises from cross-talk between infiltrating immune cells and tissue-resident populations. Fibrosis develops through coordinated remodeling across multiple cell types. These processes are not defined by single genes or mutations, but by regulatory programs, cell-to-cell communication, and transitions between biological states. And traces of that functional information appear to persist in circulation: A growing body of work indicates that cell-free DNA can carry signals of immune activation, fibroblast activity, and tissue remodeling, making aspects of gene regulation and cellular state readable directly from plasma.

This shifts liquid biopsy from identifying tissue of origin toward measuring tissue and disease states. Across a range of immune-mediated diseases, circulating signals increasingly reflect the stromal and immune programs seen in tissue and single-cell studies—not isolated molecular changes, but coordinated states of matrix remodeling, immune activation, and tissue stress. Just as important, repeated measurements turn these signals from snapshots into longitudinal trajectories, letting clinicians watch how biology evolves and responds to treatment over time. The critical question is no longer only what state a patient is in today, but where that state is heading next.

The emergence of trajectory medicine

Once biological states become measurable, a different model of care becomes possible. Many diseases are still managed by observation and iteration. In rheumatoid arthritis, for example, patients often cycle through therapies until one happens to work. The problem is not a shortage of options, but limited visibility into the biology driving disease in a given patient. The same constraint runs through oncology, inflammatory bowel disease, fibrosis, neurodegeneration, and transplantation.

This is no longer purely theoretical. In a peer-reviewed study led by investigators at Princess Margaret Cancer Center,¹ a single blood-based assay simultaneously measured immune, stromal, and tumor-associated biology in leiomyosarcoma—a cancer where low tumor mutation burden limits conventional circulating tumor DNA approaches and repeat biopsies are rarely feasible. Plasma-derived promoter activity showed strong concordance with matched tumor RNA sequencing, and longitudinal sampling captured shifts in immune and stromal biology tied to immunotherapy response and resistance.

Similar observations are beginning to appear beyond oncology. In immune-

mediated diseases such as rheumatoid arthritis² plasma-derived signals have recovered distinct, tissue-anchored stromal and immune programs consistent with the biology of the affected tissue. These findings are still early—generated in relatively small cohorts and in need of validation in larger, independent studies—but together they suggest that functional biological state, across both cancer and chronic inflammatory disease, can be measured from blood and followed over time.

When biological state becomes measurable, treatment shifts from reacting to symptoms toward managing trajectories. The questions change: not where a patient is today, but which path they are on, which intervention is most likely to change course, and how early a divergence from the expected response can be caught. Answering them requires a measurement framework built to capture biological change, not just biological presence.

The next platform layer in medicine

Every major advance in medicine has been unlocked by a new layer of measurement. Clinical chemistry enabled laboratory medicine. Imaging-enabled anatomical medicine. Genomics enabled precision medicine. The next layer will come from the ability to measure functional biological states and how they change over time.

The implications reach well beyond diagnostics. Therapy selection becomes a question of which intervention is most likely to alter a patient’s course. Drug development becomes a question of mapping how interventions reshape biology across populations. Disease interception becomes a question of recognizing an unfavorable path before symptoms appear. Healthcare itself becomes organized around how biological systems evolve, respond, adapt, and recover.

The organizations that define this future will not simply identify biomarkers. They will build the technologies and analytical frameworks that make biological change measurable, predictable, and ultimately actionable. The next era of medicine will be defined not by detecting disease earlier, but by understanding where biology is headed and learning how to change its course.

Problems that will define the field

None of this will come easily, and the challenges are worth stating plainly. Reading regulatory signal from cell-free DNA demands sophisticated computational methods and large, well-annotated datasets that account for variation across individuals, disease states, and platforms. Without rigorous analytical and technical standardization, such measurements will be hard to compare, reproduce, and interpret at scale.

The challenges of clinical translation are greater still. Prospective studies must show that measuring biological state from plasma improves clinical decisions and patient outcomes, especially where treatment selection remains largely empirical. Regulatory and reimbursement frameworks, built for mutation- and burden-based tests, may be a further hurdle. Functional biomarkers will need to prove that they can reliably characterize biological state, predict clinically meaningful change, and shift management in ways that improve outcomes.

These are not reasons for doubt. They are the next set of problems that will define the field and the test any serious platform will have to pass.

Where biology is going next

Twenty-five years of liquid biopsy trace a steady expansion of what blood can tell us: from detecting DNA fragments, to identifying their tissue of origin, to understanding the functional states that produced them. The patients who stand to benefit reach far beyond oncology, into the many chronic diseases whose underlying biology is increasingly understood but still hard to measure in routine care.

The shift from detection to function is already underway. How much it ultimately changes medicine will depend on how well we translate biological insight into clinical decisions. The question is no longer whether disease can be detected from blood, but whether we can see where biology is going next and intervene before the outcome is set.

References

Lopes, C.D.H., Wu, HT., Dilger, K. et al. Predicting immunotherapy benefit in leiomyosarcoma through active chromatin cfDNA profiling. npj Precis. Onc. (2026). doi: 10.1038/s41698-026-01451-9.
Taylor P., Antonova J., Geis J. et al. Detection of Synovial Signatures in Peripheral Blood of Patients with Rheumatoid Arthritis via a Novel Blood-Based DNA Capture Assay [abstract]. Arthritis Rheumatol. 2023; 75 (suppl 9).

Diana Abdueva, PhD, is the founder and CEO of Aqtual.

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Producing Recombinant Antibodies for In Vivo Research

Edusehat — Fri, 14 Aug 2026 06:35:11 +0700

Average expression yield (mg/L) for IgG and bispecific antibody formats produced using Bio X Cell’s recombinant transient expression platform, compared with representative published transient expression benchmarks.

Antibody reagent variability is a leading but frequently underappreciated source of irreproducible preclinical data. For in vivo studies, the problem is multidimensional: supply must simultaneously meet specifications for yield, purity, endotoxin control, and lot-to-lot consistency, and standard production approaches commonly treat at least one of these as a flexible trade-off. Higher yield at the expense of purity, or rigorous quality control at volumes too small to support multi-cohort designs, are the familiar compromises. Specifying all four attributes together as non-negotiable engineering targets, rather than aspirational outcomes, changes what study designs can responsibly assume and what data can be trusted.

This article examines what that specification looks like in practice, using recombinant antibody production and analytical data across multiple formats to illustrate how yield, purity, endotoxin performance, and lot-to-lot consistency can each be controlled simultaneously. Bispecific antibodies are the most technically demanding format in this analysis and serve as a useful reference point for understanding the production architecture required across all formats.

Why platform design matters

A standard Immunoglobulin G (IgG) is a symmetric homodimer, and its production, while technically demanding, benefits from that symmetry at every stage from expression through purification. Bispecific formats deliberately break that symmetry by pairing two distinct heavy chains, two distinct light chains, or both simultaneously, and every stage of production is harder as a result.¹

A common challenge in bispecific antibody production is chain mispairing and the correct assembly of complex antibody formats. Compared with conventional IgG molecules, bispecific antibodies require coordinated expression and pairing of multiple antibody components, increasing production complexity and placing greater demands on expression, purification, and analytical workflows.^1–3

As a result, achieving strong yield, high purity, low endotoxin, and lot-to-lot consistency simultaneously is substantially more challenging for bispecific formats than for conventional monoclonal antibodies.

This is why platform design matters. The combination of attributes that preclinical researchers need, sufficient yield to supply multi-cohort studies, purity adequate for in vivo use, endotoxin control across independent lots, and analytical consistency that makes lots interchangeable, is harder to deliver simultaneously for bispecific formats than for any other. A platform that can consistently produce bispecific antibodies at high yield and quality demonstrates the ability to maintain consistent production performance across recombinant antibody formats.

Yield across formats

Transient transfection of mammalian cells, principally HEK293 and CHO systems, is the standard expression approach for research-grade recombinant antibody production. One advantage of transient mammalian expression systems is rapid material generation, enabling recombinant antibody production within weeks rather than the months often required for stable cell line development. These systems are also compatible with the co-transfection strategies used for complex antibody formats such as bispecifics.

When expression workflows are properly optimized across DNA mass ratios, transfection reagent formulations, culture media composition, and harvest timing, yields well above historical benchmarks are achievable across formats. Data from this platform demonstrate conventional murine IgG production averaging 633 mg/L, compared to previously published transient murine expression examples averaging approximately 114 mg/L, a more than five-fold improvement. For murine bispecific formats, the same platform averaged 180.5 mg/L versus previously reported transient murine bispecific examples averaging 35.5 mg/L. The proportional improvement is comparable across both format classes. Gram-scale preclinical production is achievable even for the most structurally demanding formats without requiring stable cell line development.

Purity and endotoxin control

Yield at the expense of purity is not a viable tradeoff for in vivo applications. Recombinant antibodies intended for in vivo use must meet quality standards across multiple dimensions simultaneously: product-related impurities, including aggregates, fragments, and endotoxin, must be controlled within defined specifications at every lot.

Endotoxin is a particular concern for studies measuring immune activation, cytokine biology, or tumor microenvironment dynamics, where uncontrolled endotoxin burden can introduce signals that are indistinguishable from treatment effects. Endotoxin control is therefore not a release formality but a scientific requirement for study interpretability.

Across 300 released custom service lots spanning multiple antibody formats, endotoxin levels were consistently maintained within defined internal release specifications. Ninety-one percent of lots measured ≤0.4 EU/mg, well within the 0.5 EU/mg threshold commonly referenced for in vivo study readiness.⁴ That distribution reflects process consistency rather than occasional good performance: the same controlled purification conditions and quality checkpoints applied across every lot produce a predictable endotoxin outcome, independent of format or production scale.

Lot-to-lot consistency

Purity and endotoxin performance on a single lot are necessary but not sufficient for multi-cohort in vivo research. The question that governs longitudinal and multi-site study design is whether lot three will be analytically interchangeable with lot one. Lot-to-lot antibody variability is among the most common and most underappreciated contributors to irreproducible preclinical data, and it is a confounder that experimental design cannot correct for retrospectively.

Treating lot-to-lot consistency as a built-in specification rather than a hoped-for outcome requires that the same analytical release package be applied to every lot, and that the production process be controlled tightly enough to make the results of that package predictable. High-performance SEC (HP-SEC) provides the most sensitive window into that consistency, resolving monomer from high-molecular-weight aggregate species and capturing subtle process drift through retention time and peak shape metrics.

HP-SEC data from four independently produced lots of two representative antibodies, MAR1-5A3-CP056 and 29F.1A12-CP005 (n=8 total), demonstrate what that consistency looks like in practice. Monomer purity exceeded 98.7% in every lot across both antibodies, with a combined range of 98.57 to 99.35% and within-antibody RSD of ≤0.05%. HMW pre-peak area remained ≤1.43% across all eight lots (range 0.65 to 1.43%), with within-antibody variability of ≤0.78 percentage points. Monomer retention time was stable within each antibody to ≤0.05% RSD, and peak width at half maximum, a sensitive indicator of column performance and process consistency, remained stable within each antibody across all lots. Together, these metrics demonstrate that the production and analytical processes are sufficiently controlled to deliver interchangeable lots across independent production runs.

Translating analytical requirements

Endotoxin Control Across Released Lots. Endotoxin values measured across 300 released lots. Ninety-one percent of lots measured ≤0.4 EU/mg, reflecting consistent endotoxin control within defined internal release specifications across independent production runs.

Treating antibody reagent quality as a study design variable, rather than a procurement decision, changes how specifications should be set before work begins. For endotoxin, the relevant question is not whether a lot passes a generic release threshold but whether that threshold is appropriate for the biology being measured. Studies interrogating immune activation, cytokine signaling, or tumor microenvironment dynamics require more stringent endotoxin limits than studies where immune readouts are not the primary endpoint; the specification should follow the assay, not the other way around.

For lot-to-lot consistency, the practical requirement is that the same analytical release package, minimally, SEC purity profile, monomer retention time, and HMW aggregate content, be obtained for every lot used in a study, and that acceptable ranges be pre-specified rather than assessed retrospectively. When those ranges are defined in advance, an out-of-specification lot can be identified before animals are dosed rather than after results are in hand.

Yield determines whether these specifications are enforceable at the scale a study actually requires. A consistency specification that cannot be met at the multi-gram scale is not a specification; it is an estimate. The data presented here illustrate that strong yield is achievable for both conventional and bispecific formats, while analytical consistency can be maintained across independent recombinant antibody lots. For researchers designing studies where the antibody must behave the same way every time, that combination is the starting point for a defensible experimental design.

HP-SEC lot-to-lot consistency for MAR1-5A3-CP056 and 29F.1A12-CP005 (n=4 lots per antibody, 8 lots total). Panel A: normalized HP-SEC overlays for MAR1-5A3-CP056. Panel B: normalized HP-SEC overlays for 29F.1A12-CP005. Panel C: summary statistics table reporting monomer RT, monomer area, HMW pre-peak area, HMW RT, and FWHM for each antibody (mean ± SD), combined range across all eight lots, and within-antibody variability. Monomer purity ≥98.7% across all lots; monomer RSD ≤0.05%. Method: AdvanceBio SEC 300Å, 2.7μm, 4.6×150mm; 35mM sodium phosphate, 0.25M NaCl, 0.1M arginine, pH 6.8; 10μL injection; 280nm detection.

References

Chen S, et al. Immunoglobulin gamma-like therapeutic bispecific antibody formats for tumor therapy. J Immunol Res. 2019 Feb 11; 2019:4516041. doi:10.1155/2019/4516041.
Wei H, et al. Structural basis of a novel heterodimeric Fc for bispecific antibody production. Oncotarget. 2017 May 2;8(31):51037-51049. doi: 10.18632/oncotarget.17558.

Schaefer W, et al. Immunoglobulin domain crossover as a generic approach for the production of bispecific IgG antibodies. Proc Natl Acad Sci USA. 2011;108(27):11187–11192. doi:10.1073/pnas.1019002108.
Malyala P, Singh M. Endotoxin limits in formulations for preclinical research. J Pharm Sci. 2008;97(6):2041–2044. doi: 10.1002/jps.21152.

Christina Bouwens is a marketing manager at Bio X Cell.

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Long-Term Memory Loss Secrets Revealed with Artificial Hibernation

Edusehat — Fri, 14 Aug 2026 06:35:10 +0700

Memories can survive even after the brain temporarily loses more than half of its synaptic connections, according to the results of a study in mice that challenge the long-held view that long-term memories depend on stable individual synapses. The researchers, headed by a team at Okinawa Institute of Science and Technology (OIST), and including teams at the University of Tsukuba, Exploratory Research Center on Life and Living Systems (ExCELLS), and National Institutes of Physiological Sciences, used a mouse model of artificial hibernation to examine structural mechanisms underlying memory retention.

Their findings indicate that memory may be preserved not through individual synapses, but through resilient patterns of neural architecture, including specific clusters of connected synapses that remain protected during widespread hibernation-associated brain remodeling. These preserved structural motifs could act as a “core memory trace,” allowing the brain to rebuild functional networks after major disruptions.

Research lead Kazumasa Tanaka, PhD, head of OIST’s Memory Research Unit, said, “Previously, synaptic strengthening was thought to be key to memory recall, and that stronger synapses with larger dendritic spines were fundamental to long-term memory retention. Here, we show that not every synapse matters, and demonstrate instead the vital importance of engram architecture. The study indicates that small clusters of engram-engram synapses are preserved to enable accurate recall even after hibernation.”

Tanaka is senior and corresponding author of the team’s published paper in Science, titled “Artificial hibernation reveals synaptic engram architecture associated with memory retention.”

Understanding how memories are stored in the brain is one of the central challenges in neuroscience, the authors wrote. For decades, scientists have believed synaptic potentiation—the adaptive strengthening of our brain’s cellular connections—to be the key to memory retention. The newly reported research by Tanaka and colleagues now demonstrates the importance of higher-order synaptic architecture, suggesting that specific clustered patterns of connections between brain cells may be key to retaining long term memory.

“The structural underpinning of memory has been one of the most fundamental topics in neuroscience for decades,” the authors wrote. Much like computer storage, we humans require a physical memory trace to be stored in the brain. This physical trace, known as an engram, is encoded through a dedicated network of brain cells undergoing changes at their synapses, the junctions where they meet. When connecting brain cells repeatedly fire together, their synapses strengthen, increasing neurotransmitter release and triggering structural changes, such as larger dendritic spines, which expand the contact area between the two cells. Conversely, when a particular connection isn’t very active, the synaptic bonds between the cells weaken, and may eventually disappear altogether.

The synapses with larger, more stable dendritic spines have traditionally been seen as key for memory. While we might expect these connections to stay consistent over the course of a memory, recent studies have found that the structures and numbers of cells involved in a particular engram can change over time, without affecting recall. “… recent studies have revealed that synapses can be highly dynamic, with dendritic spines frequently appearing and disappearing, neuronal representations drifting over time, and memories remaining retrievable even after previously strengthened synaptic connections are disrupted,” the team continued. These observations raise a fundamental question, they noted. “How can memories remain stable despite extensive structural remodeling of neuronal networks?”

To investigate, the researchers turned to an artificial model of hibernation, as an experimental model for studying memory stability. In a hibernation state, decreased metabolism enables creatures to survive harsh, wintery conditions with little food, and can also causes brain activity to dramatically reduce.

In 2020, a team led by coauthor Takeshi Sakurai, PhD, at the International Institute for Integrative Sleep Medicine (WPI-IIIS), Tsukuba Institute for Advanced Research (TIAR), University of Tsukuba, induced artificial hibernation for the first time in mice. Their research uncovered the brain circuitry necessary for inducing hibernation, unlocking a new tool for neuroscience research. They later reached out to Tanaka to collaborate. “Our brains are incredibly complex. If hibernation can reduce and simplify brain activity and structure, it could make studying these convoluted systems a bit easier. That’s why I wanted to use artificial hibernation techniques to study memories,” he said.

By imaging mouse brains before, during and after artificial hibernation, the researchers found that more than half of synapses in the hippocampus region of the brain disappeared in hibernation, and neuronal firing rate, a measure of brain activity, reduced by roughly 70%. Synapse elimination didn’t seem to be influenced by dendritic spine size, with synapses involving both large and small spines equally likely to be removed.

“Artificial hibernation caused a profound reduction in neuronal activity and eliminated more than half of hippocampal synapses,” the investigators noted.

The surprise came in behavioral tests after artificial hibernation, which showed that the animals’ memory recall remained the same, or even improved in some instances. “Despite these large-scale structural changes, mice retained previously acquired memories and preserved neuronal representations of experience,” they stated.

This diagram shows two characteristic patterns found within the study. On the left, we see a multi-synaptic bouton (MSB), where one presynaptic terminal joins to multiple different dendritic spines on different cells. The researchers found that MSBs were more likely to be conserved following hibernation, suggesting their essential role in memory retention. On the right, we see clustered engram patterns. Dendritic spines that are close together but that connect to various axons of neurons in a different section of the brain are active within the same engram. Again, the researchers found that clustered engram synapses were preferentially conserved after artificial hibernation, which suggests their importance in memory retention. [Luo-chu Yang]

First author Yu-Ju Lin, PhD, commented, “It was astonishing. Logically, if all our engram synapses were essential in memory retention as traditionally thought, memory should have massively deteriorated.”

To investigate why memory retention may have stayed intact or improved, the researchers used a technique called CLEM—correlative light and electron microscopy. “CLEM combines fluorescence microscopy with high-resolution electron microscopy, explained co-author and technician Ai Takahashi. “By labelling samples with fluorescent tags, we can see different parts of the cell or organism in different colors. This can, for example, help us to pinpoint important proteins. Then using electron microscopy, we can zoom in on those same areas, to examine substructures in much higher detail.”

The team’s study is the first to use CLEM to observe engrams, a significant achievement given how small and sparse engram synapses are. “Successfully correlating the light microscopy and electron microscopy datasets to image engrams is a very technically challenging feat,” added Tanaka. “We hope our contributions to developing this method may provide new platforms for studying other important neuroscientific questions in future.”

The team fluorescently labelled the synapses thought to be involved in a particular memory trace and examined these before and after artificial hibernation. They found a significant decrease in synapses. However, certain clusters of synapses seemed to be spared. “Dendritic spines were eliminated regardless of their size, but many reappeared at the same dendritic locations after recovery,” they wrote. “Notably, synaptic connections between neurons encoding the memory were organized into spatially clustered groups.” Tanaka said, “This suggests that for long-term memory, only particular clusters of synapses matter—the rest may be dispensable. Interestingly, dendritic spine size, which has been shown to increase in initial memory encoding, doesn’t seem to play a factor in memory retention.”

Lin added, “We’ve observed this interesting correlation between clusters and memory retention, but not yet proven a causal link. As technology develops, it will be interesting to study these clusters in more detail, to answer these remaining questions.”

Looking forward, the researchers hope to continue their studies on memories, with the aim of understanding the mechanisms by which these core clusters are protected. “A deeper understanding of the mechanism by which hibernation protects the core memory trace and maintains the network integrity would reveal more comprehensive principles of memory,” the investigators concluded. “

Tanaka noted, “We’ve unlocked some insights into the architecture needed for memory retention. But there are many more questions to explore. How does the brain maintain this structure over time? How do different memories interplay? We have so much left to learn.”

The researchers also plan to study other aspects of artificial hibernation. “This work focused on mice, but the same neuronal circuitry for hibernation is well-conserved across many mammals, including humans,” Tanaka pointed out. “Therefore, through artificial hibernation studies, we may be able to discover new insights or applications that can translate to human health and neuroscience.”

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Financial Value of AI Agents in Drug Development

Edusehat — Fri, 14 Aug 2026 06:35:09 +0700

A new Tufts Center for the Study of Drug Development (CSDD) analysis shows that an AI clinical monitoring agent can deliver net financial gains as high as $21 million per drug development program and 82 times the return on investment.

The findings are based on benchmarked oncology program and clinical trial data from Tufts CSDD and contract value data from Medable, which provides a cloud-based software platform designed to modernize and accelerate clinical trials for the pharma and life sciences industries.

Tufts CSDD officials believe that the study marks the first time that eNPV modeling based on actual use and benchmark data has been applied to quantify the net financial impact of an agentic AI solution deployed to support a drug development program. [Prime Images/Getty Images]

The Tufts CSDD analysis specifically assessed the impact of Medable’s Clinical Monitoring Agent across three top-line metrics:

Expected net present value: The agent showed expected net present value (eNPV) gains of approximately $7.5 million (Phase II trial), $11.3 million (combined Phase II and Phase III) development, and $21 million (Phase III trial).

Overall return on investment: The study found an estimated ROI of 64x for Phase II and 82x for Phase III clinical trials.
Direct operating cost savings: Tufts CSDD estimated direct operating cost reductions in on-site monitoring per clinical trial of approximately $4.4 million per Phase II and $5.6 million per Phase III study.

“To our knowledge, this is the first time that eNPV modeling based on actual use and benchmark data has been applied to quantify the net financial impact of an agentic AI solution deployed to support a drug development program,” said Ken Getz, Tufts CSDD executive director. “The financial value created by the investment and deployment of the monitoring agent was driven by operational efficiencies such as the reduction in the number of on-site visits and reduced travel costs as well as accelerated enrollment and database lock timelines.”

Additional analysis identified and valued administrative off-site monitoring task efficiencies of approximately $600,000 (Phase II) and $1.7 million (Phase III). These savings reflect clinical research associate time that could be reallocated to other studies and were not included in the eNPV calculations.

The analysis also found that agentic AI can accelerate clinical development by approximately 10 weeks. By shortening activities on the critical path of development, agents help sponsors complete studies sooner, advancing regulatory submission and potential commercialization while increasing the expected financial value, according to the study.

Key contributors include faster patient enrollment, reducing enrollment timelines by approximately 109–119 days, earlier database lock, shortening closeout activities by about two weeks, and earlier realization of future revenue and lower development costs.

“The potential impact is magnified when applied across a large oncology portfolio,” said Pamela Tenaerts, MD, chief medical officer at Medable. “For a sponsor with 20 active indications, deploying a clinical monitoring agent across Phase II and III studies could generate as much as $226 million in incremental portfolio eNPV.

“For a sponsor with 50 active indications, that figure could jump to as much as $565 million. Bottom line? We now have evidence demonstrating sizable value creation of agents in clinical research, helping break longstanding barriers.”

In addition to these high-level findings, Tufts and Medable will publish a more detailed peer-reviewed paper later this year. Sign-up to be the first to receive the final published paper here.

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Advancing Microbial-Derived Biologics from Scale-Up to Commercial Production

Edusehat — Fri, 14 Aug 2026 06:35:08 +0700

Sam Zhang, PhD

Vice President, Head of Microbial and Viral Platforms (MVP)
WuXi Biologics

Broadcast Date: Tuesday, September 29, 2026

Time: 08:00 PDT, 11:00 EDT, 17:00 CET

In biomanufacturing, there are complex demands around fermentation scale-up, process consistency, technology transfer, and manufacturing capacity that must be addressed for microbial-derived biologics to complete commercialization. Meeting those demands depends on specialized infrastructure and an integrated strategy for process validation, process performance qualification (PPQ), and long-term operational readiness.

In this GEN webinar, our expert speaker, Sam Zhang, PhD, will discuss emerging trends and capacity needs across the global late-stage microbial pipeline and CDMO landscape. He will introduce WuXi Biologics’ integrated late-stage microbial capabilities including the newly constructed commercial facility in Chengdu, China, designed for microbial-derived biologics. He will demonstrate features of the comprehensive tech transfer and scale-up platform, as well as PPQ and commercial readiness strategies. He will also share a case study featuring a peptide program that will elucidate WuXi Biologics’ tailored solutions for microbial-derived biologics. Key takeaways will include:

Critical success factors for technology transfer, process scale-up, PPQ readiness and commercial manufacturing

Large-scale microbial manufacturing capabilities at the Chengdu site, with fermentation scales of up to 15,000 L

Practical learnings from a complex peptide program, with broader relevance to recombinant proteins and other microbial-derived modalities

A live Q&A session will follow the presentations, offering you a chance to pose questions to our expert panelists.

Produced with support from:

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Scientists just created female clones of male mice

Edusehat — Thu, 13 Aug 2026 09:05:02 +0700

A lifeline for endangered species: The technique could help rescue species where only males survive, by generating females for reproduction. Frozen cell banks already exist for hundreds of species that could benefit.

Still imperfect, but promising: Y-CUT still needs egg cells from a female of the same or similar species, and XO chromosomes cause infertility in most mammals beyond mice — but complementary technologies are emerging fast.

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Scientists have deliberately turned male mouse embryos into females for the first time. A team based in Japan used a CRISPR-based approach to remove the Y chromosome from male cells and create female clones of male mice.

“No one has done this before,” says Monika Ward, a reproductive biologist at the University of Hawaii, who was not involved in the research.

The feat could change the way scientists think about reproduction, says Takashi Ishiuchi, a reproductive biologist at the University of Yamanashi, who co-led the work. The findings were published in a preprint paper shared on bioRxiv earlier this month, which has not yet been through the peer-review process. “There’s a fixed concept in our scientific field that we need both females and males for reproduction,” says Ishiuchi. “I think we could change this concept.”

He and his colleague Shogo Matoba of the Riken BioResource Research Center in Ibaraki also hope their technique could help rescue endangered species, particularly in cases where only a few individuals remain.

“It’s exciting to see,” says Ben Novak, lead scientist at the wildlife conservation organization Revive & Restore, who was not involved in the work. “I am confident there will be plenty of applications, particularly for conservation purposes.”

Sex change

Ishiuchi says he and his colleagues were inspired by the Okinawa rubble goby, a fish that can change its sex in certain situations. If no males are present, a female can do this in order to reproduce with the other females. Males can also change sex to female.

This ability to change sex might be useful when it comes to rescuing endangered species, including mammals. There’s some precedent in the lab—albeit not intentional. In 2009, researchers reported the accidental birth of a single female pup in a batch of 27 clones created from male mouse cells.

Sometimes the surviving population of a species falls so low that scientists will try to clone those animals. Cloning isn’t perfect—it can be tricky and inefficient, and it creates genetically identical individuals whose offspring might be more vulnerable to disease. But it has helped scientists with efforts to bring some species back from the brink of extinction, including black-footed ferrets and Przewalski’s horse.

Cloning an individual can only replicate its genes, so cloning a male animal will create all male offspring, for example. That won’t help much in the hypothetical situation where only male individuals of a species are left.

Ishiuchi has been working on a way to overcome this challenge by altering the chromosomes in cells. Mammals’ DNA is organized in pairs of chromosomes, including one pair of sex chromosomes. These sex chromosomes are typically XX in females and XY in males.

It’s the Y chromosome that makes mammals male. Ishiuchi and his colleagues have developed a CRISPR-based tool to get rid of it. Their approach targets a section of the Y chromosome that plays an important role in ensuring that, each time a cell divides, the “daughter” cells inherit the Y chromosome.

Cutting the Y

When the researchers tested their technique—which they call Y-CUT—in early-stage mouse embryos, they found they were able to eliminate the Y chromosome. Treated XY embryos were transferred to surrogate mice, which gave birth to female pups. The effect can be described as a “sex reversal,” say the researchers.

The female pups had XO chromosomes, which means they had one X chromosome rather than the usual two. But this didn’t seem to affect the animals, which grew up healthy and fertile, say Ishiuchi and Matoba.

In a second experiment, the researchers found they could also use Y-CUT to create female clones from male mice.

A standard approach to cloning involves taking the DNA-containing nucleus of a cell from an adult animal and inserting it into an egg cell that has had its own DNA removed. Under the right conditions, the resulting cell can develop into an animal that is genetically identical to the original donor.

Matoba and his colleagues used a similar method. Once they had a glut of these cloned cells, they treated some with Y-CUT before transferring them to surrogate mice to carry the pregnancies.

This allowed them to create female clones of male mice. The females are genetically identical to the original male, apart from the missing Y chromosome, says Matoba. “It’s like sci-fi,” says Ishiuchi.

Courtesy of Takashi Ichiushi and Shogo Matoba, as published in their bioRxiv preprint.

In other experiments, the scientists were able to create female clones from male cells that had been cryopreserved—and the cloned males and females could mate to produce healthy pups. This suggests the Y-CUT approach might allow scientists to create female clones from male samples in “frozen zoos” that store cryopreserved cells and tissues from a range of animal species.

It could have uses beyond conservation efforts, too. “This could be used potentially for producing genetically engineered animals,” says Ward. Creating an animal with multiple genetic edits can be time-consuming and expensive; creating male and female clones of that animal could help scientists time and money. Ward also hopes the technique could be a useful tool to study the biology of sex chromosomes.

Complementary techniques

The Y-CUT approach isn’t perfect. For now, it still requires hollowed-out egg cells, which need to come from females of the same species or at least a closely related one. And it won’t be useful for endangered species in which only females survive.

The technique works well in mice, partly because XO female mice are fertile. But while the approach might help some of the 355 endangered and vulnerable species of rodents, other mammals with XO chromosomes tend to experience infertility.

But other new technologies could complement Y-CUT. In 2023, Katsuhiko Hayashi of Osaka University and his colleagues showed they could turn cells taken from male mice into egg cells. This enabled them to create mice with two dads—but the same approach could also provide the hollowed-out egg cells needed for the Y-CUT technique. “It’s a complementary story,” says Matoba.

Ishiuchi is also working on a technique that involves inserting a second X chromosome into cells, which might restore the fertility of the resulting female animals.

In addition, there might be a work-around for situations where scientists have females but need males. A couple of months ago, Sayaka Wakayama of the University of Yamanashi in Japan and colleagues showed they could insert rat chromosomes into mice. That could potentially be used to create XY male embryos, says Novak.

That would be useful in cases like that of the black-footed ferret, he adds. A conservation team recently cloned a female ferret using cells taken from another animal in the 1980s. That female is considered incredibly valuable, says Novak. But females can produce only a few litters in their lifetime. A male clone, which might be able to contribute to dozens of litters in a lifetime, would be “desirable.”

“It’s really exciting to see more diverse tools being developed,” says Novak. “There are so many different scenarios in which they could be used for rare and endangered species.”

Biopharma’s Drawdown Is a Chance to Build Biotech Back Better

Edusehat — Thu, 13 Aug 2026 05:25:12 +0700

The biopharma industry is emerging from one of the sharpest boom-and-bust cycles in its history. Following a sharp decline in biotech venture funding (2025 deal counts down by two-thirds since 2021, according to Crunchbase) and rising lab vacancies (over 50% in Seattle, according to HughesMarino), the industry is now rebounding.

American biotech hubs like Boston, Seattle, and San Diego are starting to wake up again. But will they be able to learn from the lessons of the recent past? For many biopharma industry stakeholders, the post-COVID downturn provides a truly constructive opportunity: a forced reset of how biotech companies are built, and an opportunity to build them with an eye towards the future.

How we got here

Simply put, over the past 25 years, biopharma’s business model grew unsustainable.

The headline numbers tell the story. In Deloitte’s annual analysis of biopharma R&D productivity, the industry’s internal rate of return (IRR, a common measure of investment profitability) from drug discovery fell to just 1.2% in 2022 as prices soared and sales diminished. R&D productivity recovered somewhat in 2024 (5.9%), but this is still far below the industry’s average cost of capital (>11%), especially given rising interest rates.

The dismal data echoes the infamous “Eroom’s Law” (Moore’s Law backward)—the observation that, unlike the famously improving productivity of microchips, the number of drug approvals per billion dollars invested has been declining for at least 30 years.

When incorporating the cost of drug development failures and capital costs, a two-decade analysis in the Journal of the American Medical Association showed that the total cost of drug development rose from $172 million (in 2018 dollars) to $879 million. The need to effectively control costs was listed as paramount amidst further barriers to getting drugs to market.

Academics debate the reasons for Eroom’s law, but the Baumol effect is likely a key part of the story. Named after economist William J. Baumol, emerged in the 1960s to explain why prices rise in some areas of the economy (especially services) and fall in others (especially manufacturing). Briefly summarized, costs generally to rise faster than inflation in labor-intensive sectors—ones that experience lower productivity growth.

As the Baumol effect predicts, between 1998 and 2018, services became more expensive while many manufactured goods became cheaper. Note the modest increase in average wages in the middle. [Creative Commons license CC BY-SA 4.0]

One classic example is a live performance of a string quartet: it takes just as many labor hours to perform today as 300 years ago. By contrast, manufactured goods like television sets grow cheaper every year. As the Baumol effect predicts, between 1998 and 2018, services became more expensive while many manufactured goods became cheaper. And few activities in the economy are more labor intensive than clinical-stage drug development.

The COVID-19 pandemic dramatically amplified those pressures. Predictably, when a wall of public research money (Operation Warp Speed and other programs) rammed into the hard realities of biomedical research, scarcity ensued. In many cases supply was simply unavailable at any price. Even mundane items like pipette tips—became scarce.

To be sure, 2021 was experienced by most in biotech as a boom time—low interest rates ensured plenty of capital was available to meet biotech payrolls. But this capital also meant a lack of incentives to control spending, resulting in spiraling costs and short-term decision-making. Boom times breed bloat and complacency, and we’re living through the aftermath.

Masking the malaise

For a time, however, these warning signs were easy to ignore due to the existence of two counter-trends: declining interest rates and increasing drug prices.

As interest rates decline, the value of any capital asset increases—it works the same for single-family homes and drug development programs. Moreover, biopharma is unusually sensitive to interest rates, given their long development timelines and heavy upfront costs. Steadily declining interest rates therefore created an appearance that ever‑rising expenditures were sustainable.

A second counter-trend unfolded on the revenue side, as big pharma explored the upper limits of what American taxpayers and insurance policyholders would tolerate. When Gilead priced Harvoni at $84,000 in 2014, it sparked an outrage, despite solid health economics justifications; ten years later, six-figure pricing was commonplace. The high water mark of this phenomenon was surely the 2017 conviction of “pharma bro” Martin Shkreli, who had become a household name for buying and price-jacking the ultra-rare disease drug Daraprim from $15 to $750 overnight.

In short, it was interest rates and pricing power, not innovation, that arguably carried industry financials forward through the 2010s—masking the structural decline in efficiency and encouraging capital allocation decisions that could not withstand tighter pricing or financing conditions.

The tide goes out in a perfect storm

These counter trends that had been masking declining productivity came to an abrupt halt in 2022. As Warren Buffett famously quipped about financial bubbles, “Only when the tide goes out do you discover who’s been swimming naked.”

First, the Federal Reserve began the fastest series of interest rate hikes in four decades in response to surging post-pandemic inflation.

The inflation dinosaur that devoured a thousand biotechs. U.S. federal funds target rate, 2014-2024. Data is the midpoint of the federal funds target range. [U.S. Federal Reserve Bank. www.federalreserve.gov/aboutthefed/fedexplained/accessible-version.htm]

Meanwhile, U.S. drug pricing tolerance hit its political limits, culminating in the passage of the Inflation Reduction Act in August 2022, which introduced government price controls on biopharmaceuticals for the first time in the U.S. These pressures have continued, with President Trump continuing to pressure drug companies to lower prices.

As if that weren’t enough, drug development costs were also spiking. During the boom years, more and more late-stage clinical development migrated to the United States, colliding with a relatively inelastic supply of trial sites, specialized labor, GMP manufacturing capacity, and experienced managers. The result was predictable: costs exploded. And the resulting downturn affected everything from venture funding to lab vacancies.

This downturn has understandably been painful for many due to steady industry layoffs. Layoffs in early 2026 have so far exceeded the comparable prior year period, despite hopes of a turnaround. And despite recent gains in public biotech stock indices, venture capital fundraising—the lifeblood of the biotech industry—hit an eight-year low in 2025.

But history suggests that periods like this often precede fundamental reinvention. The reinvention of the technology sector, driven by cloud computing and mobile broadband led to lower costs, and durable, scalable innovation that reshaped the global economy. Biotech now stands at a comparable inflection point. The old model was viable only under extraordinary macroeconomic conditions for a limited window of time. Instead of waiting for them to return, the industry has an opportunity to evolve.

But glimpses of a new, even more value-accretive future are already apparent, and changes are apparent on both sides of the supply and demand equation.

Start with supply: signs of regeneration are already visible, even in the absence of significant interest rate cuts. The collapse in demand for lab space and equipment has sharply reduced barriers to entry.

More fundamentally, emerging technologies like lab automation and generative AI are promising to revolutionize workflows.

Regulatory innovation in Australia, the U.K. and—above all—China, is eroding clinical development costs so rapidly that even the FDA is talking about serious reforms to accelerate innovation.

The success of Ozempic and its GLP-1 competitors shows the powerful appeal of mass-market drugs priced accessibly to the global middle class and for preventing disease—investment themes that defy the conventional wisdom of the pre-COVID era, with its strong bias for specialty-pharma and orphan-disease drugs with niche markets and ultra-high prices.

With newly opened minds—and newly affordable drug-development infrastructure—a new world stands waiting, at least for those with the ambition and cunning to scale and price their products commensurate with global demand.

In sum, the winning strategies for the next wave of biotech—Biotech 2.0—are probably staring us in the face. Just like the rise of cloud computing and mobile were obvious to certain tech entrepreneurs amid the carnage of the dot-com bubble burst, the seeds of the next wave of innovation are quietly germinating right now in the U.S. Some of the most important opportunities, especially for products that can deliver better health outcomes at lower cost, are simply waiting for an opportunity in the marketplace.

As biotech markets finish working through the messy process of creative destruction, we must remain alive to the amazing possibilities it creates for innovators—newcomers, no longer crowded out by old ways of thinking.

Brian Finrow is co-founder and CEO of Lumen Bioscience, a clinical-stage biotechnology company in Seattle. Kevin Klowden works as a global economist and strategist, and a fellow at the Milken Institute, an economic think tank.

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Human Biological Datacenter to Launch to Train World Model of Human Biology

Edusehat — Thu, 13 Aug 2026 05:25:11 +0700

Vivodyne reports that it has launched “the world’s largest human biological datacenter,” with 12 robotic HIVE laboratories and the annual capacity to perform controlled trials on 3.1 million large human tissues per year—estimated at twice the scale of every clinical trial in the U.S. combined.

A row of automated human-tissue testing machines in Vivodyne’s Human Datacenter. [Vivodyne]

The company also introduced its Series 2 TissueDisk, a wafer-scale biological chip that simultaneously grows hundreds of large, functional, living human tissues and is manufactured end-to-end on Vivodyne’s own robotic production line.

Eight of the world’s largest pharmaceutical companies have paid for early access to the platform, according to Vivodyne, to discover and test new medicines “in humans” before ever testing in people, notes Andrei Georgescu, PhD, CEO and co-founder of Vivodyne.

Together, the TissueDisk and Vivodyne’s robotic HIVE laboratories create something that neither pharmaceutical research nor artificial intelligence has possessed before: a large-scale experimental environment in which the same reinforcement learning technique that has driven the explosion in AI language models can finally be harnessed to learn the workings of our physiology, explains Georgescu.

For pharmaceutical companies, that means learning how actual human tissue responds to a drug while decisions about targets, chemistry, dosing, and safety can still be made, he continues adding that Vivodyne’s approach allows millions of therapeutic interventions to be introduced into living human tissues, and their causal biological consequences measured directly with the most advanced, paired-data modalities available today: 3D scanning, transcriptomic sequencing, and deep proteomic analysis.

Vivodyne’s platform provides the foundation of the first world model of human biology, claims Georgescu. Previously, the controlled experiments required to reveal complex, physiological cause-and-effect could not be performed safely in patients or at nearly the needed scale, and Vivodyne makes those experiments possible in living human tissue outside the body, he says.

“Superintelligence in biology is needed more than ever, because we’re running out of diseases that can be cured with the simple, single-target medicines of today,” states Georgescu. “You cannot fix a car by turning a single screw, and the idea that the complex malfunctions in cancer, fibrosis, autoimmune disorders, or neurological disease can be fixed with a conventional single-target drug is wishful denial. To create AI that understands our intricate human biology, we need to continuously generate and train on huge amounts of human data, and we can’t get that by risking people. So, we grow these functional human tissues by the millions instead; large living tissues that grow their own blood vessels and immune cells and all the structures of native tissues. They mature, get diseases, bleed, scar, and, at huge scale, we learn how to make them heal.

“Every human response gives our AI something it cannot learn from a paper or a simulation: a living substrate to poke so that it can learn, from richer data than has ever been gathered, how it pokes back. At Vivodyne’s scale of automated human-tissue trials, all those learned consequences together become the training landscape for a world model of the human body, and the physical evidence that a pharmaceutical company needs before a drug is brought to patients.”

22 human organ systems grown

Vivodyne grows over 20 types of different human organ tissues, both healthy and with patient-linked diseases, including liver, lungs, gut, bone marrow, pancreas, kidney, eyes, lymph nodes, and more, with disease-specific versions spanning fibrosis, site-specific solid tumors, inflammation, metabolic disorders, vascular disease, and countless others. The company trains causal, multimodal AI models on the experiments conducted within each organ type, alone and combined.

Connecting those models across organ systems builds a world model of the human body that can answer what happens when a pair of receptors is drugged, a biological pathway is interrupted, a therapy causes an unexpected side effect, how cells respond and communicate, and whether disease is aggravated, stopped, or reversed, points out a company official. These predictions can then be real-world tested at scale to confirm what actually happens in human tissue, and then refined and advanced.

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Cortical Organoids Reveal Radial Glial Progenitor Lineage Dynamics

Edusehat — Thu, 13 Aug 2026 05:25:10 +0700

The cerebral cortex of the brain, responsible for higher-level cognitive processes, movement control, and sensory input processing, is composed of a wide variety of neurons and glial cells. Radial glial progenitors (RGPs) produce all excitatory neurons in the developing cerebral cortex. Now, for the first time, a quantitative framework of RGP lineage progression has been revealed. The team of researchers from the Institute of Science and Technology Austria (ISTA) established Mosaic analysis with double markers (MADM)-based lineage tracing in vivo—in mouse embryonic stem cells in a self-organizing cortical organoid system. The findings present critical time windows in development when compared to the real mouse brain.

This work is published in Nature in the paper, “Temporal uncoupling of radial glia lineage progression in cortical organoids.”

“In our lab we study how the brain develops from stem cells,” Simon Hippenmeyer, PhD, professor at the ISTA explains. “How a brain reaches the right size, how stem cells know when and into which neurons they should develop, but also what happens when something goes wrong during development or disease—for example, in microcephaly or macrocephaly, where the brain is unusually small or large.”

Two mouse cortical organoids at 13 days in culture. Cortical rosettes, the actual portions of the organoids that resemble aspects of the developing brain, are seen as lumps along the outer edge of the organoid. [Stouffer et al. / Nature]

The researchers compared specific developmental stages of the mouse brain with those of the organoids. Using single-cell sequencing, they examined which cell types show up in both systems, their relative abundance, and at what point in time they emerge or disappear again.

“In the developmental stages we examined, we see very similar cell populations of the mouse brain with those of the organoids,” Hippenmeyer explains. “The molecular programs are similar.”

“Now that we had this rigorous organoid system, we were able to examine even more closely what happens to stem cells during cortical structure development—and compare these processes directly with our in vivo model, the mouse,” he continues.

Using MADM technology—a unique genetic method that makes it possible to track stem cell division during organogenesis—the group produced a clear roadmap of development in the mouse brain at the single progenitor cell level.

The team found that “RGPs exhibit a high level of plasticity in proliferative potential in organoids rather than strict temporally stereotyped lineage progression as observed in vivo.” In addition, RGPs in organoids showed “increased lineage restriction, diminishing cell-type diversity in clones of cortical projection neurons, despite uniform single-cell transcriptional signatures of RGPs and a unitary lineage trajectory.”

The researchers suspect that organoids lack certain external signals. The microstructures in Petri dishes form through self-organization, and due to being cultured in the lab, they lack many of the external influences that are present in the living organism.

Researchers from the Hippenmeyer group at ISTA who carried out the study. From left to right: Simon Hippenmeyer, Osvaldo Miranda Romero, Fabrizia Pipicelli, and Carmen Streicher. [ISTA]

“In our organoids, this does not seem to work perfectly,” says Hippenmeyer. “The physical force of self-organization alone is apparently not enough. Factors present in in vivo systems are missing—the so-called stem-cell niche.”

The stem-cell niche is the specific microenvironment in which stem cells live and are regulated. It includes, for example, neighboring cells, blood vessels, signaling molecules, and growth factors, as well as mechanical signals.

They write, “critical non-cell-autonomous cues that are absent in self-organizing systems and/or the genuine stem cell niche are essential for faithful temporal control of RGP lineage progression and the generation of clonal cortical cell-type diversity.

This work presents a robust protocol for producing cortical organoids from mouse cells. It also highlights the processes in development which are sensitive to changes to—or a lack of—the stem cell niche.

These findings are important for organoid research. They show how similarly certain developmental processes unfold in organoids and in the mouse brain—and up to what point, based on current knowledge, specific aspects of brain development can be reliably studied in an organoid.

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In Vivo Protein Generation Using Nanoparticle Delivery: Emerging Platforms and CMC Strategies

Edusehat — Thu, 13 Aug 2026 05:25:09 +0700

Tao (Tony) Li, PhD

Senior Director, Head of mRNA & Nanoparticle Platform
WuXi Biologics

Broadcast Date: Wednesday, September 9, 2026

Time: 08:00 PDT, 11:00 EDT, 17:00 CET

In vivo protein generation using nanoparticle delivery has evolved from vaccine applications into protein replacement, immunotherapy, and gene editing. Lipid nanoparticles (LNPs) and other nanocarriers remain the platform of choice for protecting cargo, enhancing cellular uptake, and facilitating endosomal escape. Scaling across oncology, infectious diseases, and genetic disorders demands progress on mRNA/DNA design, nanoparticle delivery, manufacturability, CMC strategy, cost, timeline, and speed. In this GEN webinar, our expert speaker, Tao (Tony) Li, PhD, will discuss nanoparticle delivery opportunities beyond oncology and infectious disease, compare emerging nanocarrier platforms, and examine important CMC considerations critical for progressing from research through IND. He will also present a case study on decorated mRNA/LNP for in vivo CAR T, from R&D gap evaluation and process development to full GMP manufacturing. Key takeaways from the webinar include:

A deeper understanding of the landscape beyond vaccine and oncology applications

How emerging platforms compare including their advantages, limitations, and development considerations

Key CMC challenges from research through IND including manufacturing, analytical characterization, and quality attributes

Strategic insights for future directions derived from fundamental questions

A live Q&A will follow the presentation offering you a chance to pose questions to our expert panelist.

Produced with support from:

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Cancer-Fighting Abilities of NK Immune Cells May Be Disrupted by Some Omega Fatty Acids

Edusehat — Thu, 13 Aug 2026 05:25:09 +0700

Polyunsaturated fatty acids (PUFAs), and especially some omega PUFAs, are recognized for their myriad health benefits, but their role in the biology of cancer cells and tumor immunity has been less clear. The results of a preclinical study by researchers at Yale University School of Medicine now indicate that some omega fatty acids can restrain the cancer-killing capabilities of natural killer (NK) cells by acting through a receptor, LRP5.

In their paper in Science Signaling, titled “LRP5-dependent transport of polyunsaturated fatty acids serves as an immune checkpoint for natural killer cells,” first author Yi Luan, PhD, and colleagues stated, “Given the widespread promotion of polyunsaturated fatty acids (PUFAs) as beneficial dietary supplements, greater public awareness of their potential unintended effects is warranted.” The authors call for further work to explore the mechanisms that shape the expression of LRP5 in different contexts, which they say could unveil potential therapeutic targets.

PUFAs are essential lipid molecules characterized by multiple double bonds in their hydrocarbon chains, the authors wrote. They are broadly classified into two groups, omega-3 (n-3) and omega-6 (n-6) PUFAs, which are found mostly in fish, nuts, and vegetable oils. They play many essential roles in the body, including by maintaining the fluidity of cell membranes and supporting signaling within cells. “Their uptake and intracellular distribution rely on lipid transporters, which facilitate their availability for metabolic and signaling functions,” the team explained.

PUFAs, especially the omega-3s, have become widely touted for their various health benefits and anti-inflammatory properties. However, PUFAs have a more complex role in tumor biology than generally assumed, as some research indicates they can influence the biology of both tumor cells and anticancer immune cells such as natural killer cells. Research has highlighted the role of n-3 PUFAs in preventing tumor progression, the team noted. “Beyond their direct influence on tumor cells, they also modulate immune cell function within the tumor microenvironment, further emphasizing the complex role of PUFAs in tumor development,” they commented. “However, their specific impact on NK cell biology remains poorly understood.”

For their newly reported study, Luan and colleagues probed how PUFAs shape the biology of natural killer cells, which play a central role in immune surveillance. They knocked out the LRP5 receptor, which acts as the conduit for PUFA transport into natural killer cells, and found that this reduced the intake of PUFAs. However, the loss of LRP5 enhanced the cells’ ability to eliminate colon cancer cells and slowed the growth of tumors in mice.

Feeding wild-type mice a diet free of PUFAs similarly suppressed tumor growth. Additional work showed that PUFAs constrained natural killer cells by suppressing the mTORC1 signaling pathway and glycolysis metabolism. “Mechanistically, LRP5-mediated PUFA transport suppressed mTORC1 signaling and glycolysis in NK cells, a metabolic pathway essential for NK cell cytotoxicity,” they stated. “Thus, our study identified LRP5 as an immune checkpoint that restrains NK cell activity through PUFA transport–dependent suppression of mTORC1 signaling.” They suggest that future studies should further explore the regulatory mechanisms governing LRP5 expression in different contexts, “… potentially revealing therapeutic opportunities.”

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Next 10 U.S. Biopharma Clusters

Edusehat — Thu, 13 Aug 2026 01:45:14 +0700

The days of biotech businesses and institutions limiting their research and commercial activity to top clusters like Boston/Cambridge, MA, and the San Francisco Bay Area are passing, as the life sciences expand their horizons to regions and states not usually thought of as hotspots for the industry.

“Life science entrepreneurship and new product development is flourishing outside traditional coastal epicenters, creating a dynamic and distributed map of regional hubs across the United States,” wrote John Flavin, founder and CEO of venture capital firm Portal Innovations, and Pat Flavin, John’s brother and Portal’s president, in Biotech’s Future Will Be More Distributed, a guest editorial published December 15, 2025, by Timmerman Report.

Portal itself reflects that trend, having U.S. locations not only in Chicago (No. 9 on GEN’s most recent A-List of Top 10 U.S. Biopharma Clusters, published June 1), but in Providence, RI; the New York suburb of New Brunswick, NJ; and Houston (Portal also has a fifth office in Dublin, Ireland).

The Flavin brothers cited several factors in the industry becoming more ‘‘distributed” or scattered beyond the largest clusters:

Academic research shifting—Over the past decade, research has shifted to a translational focus, to address the need for more practical application in life sciences.
Federal funding flattening—Cutbacks in or leveling amounts of NIH funding (depending on the region) have forced universities to adopt new avenues for attracting and retaining research talent—such as pursuing closer relationships with industry.
COVID-19—The pandemic that wreaked havoc on the world “acted as a global shock to the life sciences innovation system, accelerating pre-existing trends and forcing a rapid re-evaluation of how and where innovation happens,” John and Pat Flavin wrote.

The growth of biotech hubs outside of the top 10 regions and states ranked since 2014 in GEN’s nationally- and regionally-cited top 10 clusters A-List has increasingly sparked a question among readers: What are the next up-and-coming hubs for life sciences activity?

GEN attempts to answer this question through this first-ever A-List of Next 10 U.S. Biopharma Clusters. This list is actually three lists in one: A top five ranking of emerging regions below the top 10 long highlighted by this publication; a top five ranking of states that have shown initiative and accomplishment in building up their life-sci ecosystems but whose individual regional clusters alone do not rise to the top; and an unranked list of up-and-coming regions and states to watch, based on recent announcements.

GEN bases its regional 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 July 6, plus all of fiscal year 2025 (October 1, 2024, through September 30, 2025).
Venture capital funding: Figures for all of 2025 and, where available, 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.

The ranking of states proved tougher than anticipated because while the sources of lab space info (commercial real estate firms and industry groups) do not compile statistics for entire states, or have not done so recently, sources for venture capital and workforce numbers either collect only regional info or have not collected it within the past year.

Next 5 Regions

1. Houston, TX

Eli Lilly is planning a $6.5 billion manufacturing site to produce active pharmaceutical ingredients for oral drugs—including Foundayo^® (orforglipron), the glucagon-like peptide-1 (GLP-1) receptor agonist obesity drug. [Eli Lilly]

Like many of the largest biopharma clusters, Greater Houston is reaping the fruits of Eli Lilly’s investment surge: The pharma giant is planning a $6.5 billion manufacturing site to produce active pharmaceutical ingredients for oral drugs—including Foundayo^® (orforglipron), the glucagon-like peptide-1 (GLP-1) receptor agonist obesity drug. Lilly grew its regional footprint in April by acquiring Houston startup CrossBridge Bio, a developer of next-generation dual-payload antibody-drug conjugates (ADCs), for up to $300 million. Another Houston startup, Duracyte, was formally launched to commercialize its Hybrid Advanced Molecular Manufacturing Regulator (HAMMR) technology, designed to produce therapeutic proteins continuously inside the human body. Duracyte’s co-founders include Robert Langer, ScD, institute professor at MIT: “He sees a lot of potential for the Houston ecosystem,” Omid Veiseh, PhD, another Duracyte co-founder and managing partner of RBL LLC, an incubator evolved from Rice University Biotech Launch Pad, told GEN. “There’s a lot of great talent. But the unique advantage that we have is we are able to benefit from a lot of unique clinical infrastructure and clinician insights. There are a lot of clinicians here who are eager to partner on investigator-initiated trials.”

Houston’s Generation Park, a 4,300-acre master planned mixed-use campus, has been selected by Bristol Myers Squibb for a $1 billion, 600,000-square-foot manufacturing site set to create about 500 jobs by 2031. The region’s largest life-sci campus is the world’s largest medical complex, the 1,345-acre, 54 million-square-foot Texas Medical Center (TMC), home to The University of Texas MD Anderson Cancer Center and Baylor College of Medicine’s primary campus. TMC and the Korea Health Industry Development Institute (KHIDI) on June 29 expanded their TMC Korea BioBridge, a partnership designed to assist South Korean biotech, digital health and medtech companies seeking to enter and expand Stateside.

Among emerging regions, Greater Houston leads in NIH funding (2,262 awards totaling $1.251 billion), is second highest among emerging regions in jobs (28,000+, according to BioHouston), and places second in lab space with about eight million square feet. “We have a very good supply of lab space in this area,” BioHouston’s chairman Jeff Wade told GEN. He said the region scooped up about a half-billion dollars in VC funding between 2025-2026 to date, which would propel Houston to second, significantly more than the approximately $300 million tallied by PitchBook. In patents, Houston finishes sixth with 2,760 families.

2. Minneapolis-St. Paul, MN

The Minneapolis headquarters of Bio-Techne. The tools, analytical technologies, and consumables giant has agreed to be acquired for $11.3 billion by Merck KGaA, Darmstadt, Germany. [Bio-techne]

Minneapolis-St. Paul’s life sciences sector was jolted June 25 when Minneapolis-based tools, analytical technologies, and consumables giant Bio-Techne agreed to be acquired for $11.3 billion by Merck KGaA, Darmstadt, Germany. The news raised fears that Bio-Techne would shed at least some local jobs within its 3,100-person global workforce (2,300 in the U.S.), since Merck KGaA said it will carry out cost-cutting “synergies” of approximately €140 million (about $160 million) that are expected to be fully realized by the third year after closing. The region’s life-sci industry has long been heavy on medical device and medtech: “We are seeing some demand from pharmaceutical companies, too, but we are nowhere near the power of Raleigh or the Boston-Cambridge area,” Chris Lyles, director of life science and technology with Minneapolis-based Knutson Construction, told REjournals in April.

In St. Paul, nonprofit University Enterprise Laboratories (UEL), which is unaffiliated with the University of Minnesota, is working to raise the $3 million it says is needed to convert a 4,000-square-foot storage area into a shared lab space with 45 lab benches for companies at the earliest stages, plus an adjacent “learning lab” for student training and engagement. “The market is telling us there’s demand,” UEL board chair Barbara Nelsen told The Minnesota Star Tribune in April. The current space has been filled for five years, with UEL executive director Sam Shuster telling the news outlet it receives a constant two to three requests for lab space weekly.

“MSP,” as locals call the region, indeed lacks lab space, with just 4.4 million square feet (Revista, 2024), placing fifth, but leads emerging regions in jobs (32,748 biopharma, agritech, and distribution jobs in 2022, though the state touts 334,500 by including healthcare and medical device positions) and places a strong second in patents (3,924 families). The “Twin Cities” also finishes sixth in life-sci VC funding with $157 million last year (Colliers) but just eighth in NIH funding with 1,206 awards totaling $649.987 million.

3. Denver-Boulder, CO

CordenPharma, a peptide drug substance contract development and manufacturing organization (CDMO), leased 64,000 square feet in Boulder at BioMed Realty’s Flatiron Park (5505 Central Ave.) in March [BioMed Realty]

Area biotech executives joined 430+ executives and investors across tech specialties to form Ensuring Colorado’s Innovation Future. The group has voiced to Gov. Jared Polis (D) its concern that the Denver-Boulder region and the rest of Colorado were losing their luster with tech—a perception fueled when Palantir Technologies in February announced the relocation of its HQ from Denver to Miami. Polis responded by seeking to reassure the group of his commitment to improving the state’s business climate: “We always want to double down on our successes, and we want to change whatever isn’t working,” the governor told The Denver Post.

Denver-Boulder has had its share of biopharma successes in recent months: CordenPharma, a peptide drug substance contract development and manufacturing organization (CDMO), leased 64,000 square feet in Boulder, CO, at 5505 Central Ave. within Flatiron Park in March. Also, that month at Flatiron Park, vaccine platform tech developer VitriVax signed a lease for 31,450 square feet at 5500 Central Ave., relocating within Boulder from 3415 Colorado Ave. near the University of Colorado. The deal came five months after VitriVax closed a $17.25 million Series B financing round whose proceeds were intended to fund development and commercialization of the company’s Atomic Layering Thermostable Antigen and Adjuvant (ALTA^®) technology.

However, Boulder, CO-based Enliven Therapeutics last month said it was moving to the San Francisco suburb of Burlingame, CA. CEO Rick Fair, a former Genentech executive, told the San Francisco Business Times the move will enable Enliven to tap into the Bay Area’s deeper talent pool and life sciences cluster, which ranked second in GEN’s A-List of Top 10 U.S. Biopharma Clusters. Yet Intero Biosystems, the developer of an induced pluripotent stem cell (iPSC)-derived human miniature intestine for predictive preclinical testing, moved to Denver from Michigan after receiving a $250,000 Advanced Industries grant from the Colorado Office of Economic Development & International Trade (OEDIT). Intero’s co-founders told The Denver Post that Denver offered the potential for a better quality of life than possible within the top-tier biopharma clusters on the East and West coasts.

Among emerging regions, Denver-Boulder scored best in VC funding, leading with $565 million (Colliers), and placed fourth in patents (3,062 families) and lab space (6.2 million square feet, according to Revista). The region ranked fifth in NIH funding (1,833 awards totaling $862.286 million), but ninth in workforce (14,856 jobs, according to Colliers).

4. St. Louis, MO

The Donald Danforth Plant Science Center in the St. Louis suburb of Creve Coeur, MO, has played a key role in positioning the region as a global leader in plant science. [Donald Danforth Plant Science Center]

This year marks 25 years of BioSTL, a regional life-sci industry group whose co-founders include William Danforth, MD, the civic leader who established the Donald Danforth Plant Science Center (named for his father) in the St. Louis suburb of Creve Coeur, MO, serving as its chairman through 2013. “It was never plant science or medical science. It was always both,” Donn Rubin, BioSTL’s founding president and CEO, told St. Louis Magazine in May: “Our region has remarkable strengths across that entire spectrum.”

Those strengths include top-tier districts like the 35+ company, agtech-focused 39 North AgTech Innovation District, anchored by Bayer Crop Science and the Danforth Center; and the Cortex Innovation District, which finished 2025 as home to 406 companies employing 5,400 people and generating $163.8 million in taxes between 2014–2025. Cortex also achieved 100% occupancy last year when pathogen detection tools developer Varro Life Sciences moved in, investing $42.5 million and creating 33 new jobs. Within Cortex, C2N Diagnostics—a developer of tests for Alzheimer’s disease and related forms of neurodegeneration—is set to move into Catalyst: Powered by WashU, where it will anchor the $100 million redevelopment of the former Goodwill building (4140 Forest Park Ave.) spearheaded by Washington University in St. Louis and a developer affiliated with the school, BOBB. The project consists of renovating the existing seven stories and adding a four-story addition. In June, C2N agreed to bring its tests to Latin America and the Caribbean through a collaboration with Miami-based diagnostics developer SouthGenetics. As for the Danforth Center, it named a new COO in June: Derek Rapp, previously a managing director with life-sci focused VC firm RiverVest Venture Partners, which has an office in St. Louis as well as in Cleveland and San Diego.

Reflecting the strength of its institutions, St. Louis dominates emerging regions in patents, where it leads with 11,049 families, and is a strong second in NIH funding with 1,855 awards totaling $1.186 billion. The region places sixth in VC funding with $134 million (BioSTL), seventh in workforce with 21,376 life-sci jobs (BioSTL), and 10^th in lab space (an estimated 3.185 million square feet based on Cortex’s 1.9 million, 39 North’s 1 million, and 285,000 square feet of incubator space (BioSTL + GEN research).

5. Dallas-Fort Worth, TX

In suburban Plano, TX, Dallas-based investment firm NexPoint is spearheading development of the $4 billion Texas Research Quarter, which is envisioned to transform the former Electronic Data Systems HQ into 4 million square feet of R&D space on 200 acres, in four phases. [Texas Research Quarter]

The city of Dallas calls itself “Biotech’s Next Frontier” while Fort Worth boasted during the recent BIO International Convention about its “Location, Location, Location.” The region’s life-sci attractions include Pegasus Park, the 23-acre former ExxonMobil HQ off Stemmons Freeway, where four buildings totaling 750,000 square feet have been activated. The park’s Biotech+ hub has attracted three anchors in recent years: BioLabs Pegasus Park, a 37,000-square-foot facility offering flexible lab, training, and office space; the Advanced Research Projects Agency for Health (ARPA-H) Customer Experience Hub; and Bridge Labs at Pegasus Park, which offers 135,000 square feet of R&D space. At Bridge Labs, the University of Texas at Arlington and Texas A&M Engineering Experiment Station opened a new biomanufacturing training and research hub in December.

Dallas is also home to de-extinction-focused Colossal Biosciences, whose nonprofit Colossal Foundation pledged in June to collect, sequence, and preserve the genetic material of more than 2,300 threatened and endangered plant and animal species, and release the resulting data at no cost through a partnership with the U.S. Fish and Wildlife Service. In suburban Plano, TX, Dallas-based investment firm NexPoint is spearheading development of another campus for life-sci users, the $4 billion Texas Research Quarter, which is envisioned to transform the former Electronic Data Systems HQ into four million square feet of R&D space on 200 acres, in four phases. Another Dallas suburb, Denton, TX, is where Novartis plans to build a 46,000-square-foot radioligand therapy (RLT) manufacturing site, announced by the pharma giant in February. The site—Novartis’ first in Texas—is expected to become fully operational in 2028.

Dallas-Fort Worth only has 3.3 million square feet of lab space, says Cushman & Wakefield (good for eighth), but that number is expected to multiply in coming years since no less than 61.2 million square feet are under construction. “DFW” fares best in workforce size and VC funding, scoring third with 26,000+ jobs (Dallas’ Office of Economic Development) and $218 million (Dealroom.com data cited by the 2025 Austin Bio & Health Report, published in February). The region also finishes sixth among emerging regions in NIH funding (1,566 awards totaling $858.342 million) and eighth in patents (1,497 families).

Next 5 States

1. Ohio (including Cincinnati, Cleveland, and Columbus)

In the Columbus suburb of New Albany, OH, Amgen is completing a $900 million expansion of its final product advanced assembly and packaging plant, which when completed in 2027 will expand the company’s investment to $1.4 billion and its workforce from 400 to 750 [Amgen]

Columbus, Cleveland, Cincinnati, and their suburbs have enough life sciences activity for the Buckeye State to emerge first among states with emerging clusters. In June, the Ohio legislature approved $3.7 billion for 2027–28 that included $1.5 million for the Ohio Life Science Training Center (OLSTC), designed to train adults for entry-level biomanufacturing and cGMP jobs. An effort between JobsOhio, a private, nonprofit promoting economic development statewide (which last year committed $30 million to the effort) and the Ohio Life Sciences Association, OLSTC will rise in the Columbus suburb of New Albany, OH—where Amgen is completing a $900 million expansion of its final product advanced assembly and packaging plant, which when completed in 2027 will expand the company’s investment to $1.4 billion and its workforce from 400 to 750. In June, Resilience announced plans to move its corporate headquarters from San Diego to the Cincinnati suburb of Blue Ash, OH, and expand sterile injectable manufacturing at its plant in another Cincy suburb, West Chester, OH.

Some 25 miles east of downtown Cincinnati in Williamsburg Township, OH, Lonza Group is considering construction of a $1 billion plant on two parcels totaling 161 acres at Half Acre Road and State Route 32 (James A. Rhodes Appalachian Highway) on land owned by Clermont County. On June 29, the Ohio Tax Credit Authority approved a 1.860%, 20-year Job Creation Tax Credit for the project, which would create 650 full-time-equivalent positions.

In June, Hikma Pharmaceuticals announced a $267 million, two-site expansion that will grow its injectable pharmaceutical manufacturing capabilities, adding 300 jobs in the Cleveland suburb of Bedford, OH, while adding 50 jobs by expanding its oral solid dose and nasal inhalation manufacturing operations in Columbus. Also in June, Cleveland Clinic launched a partnership with 10x Genomics to advance research in novel diagnostics for bladder cancer. Cleveland Clinic patients with advanced bladder cancer who are undergoing emerging therapeutic regimens will have their tumor samples examined using 10x’s spatial and single-cell biology tools—part of 10x’s expansion into clinical applications.

Among emerging states, Ohio leads in NIH funding (2,763 awards totaling $1.538 billion), patents (54,536 families), and jobs (54,536 biopharma, research/testing, agritech, and distribution jobs according to Ohio Life Sciences Association).

2. Indiana (including Indianapolis)

Eli Lilly has announced plans to spend an additional $4.5 billion across its two manufacturing sites in Lebanon, IN, where on May 6 it opened its first dedicated genetic medicine facility in Lebanon’s LEAP Innovation District. [Eli Lilly]

Indianapolis-based Eli Lilly intends to be an even larger presence in the Hoosier State, announcing plans in May to spend an additional $4.5 billion across its two manufacturing sites in Lebanon, IN, where it opened a new genetic medicine facility in Lebanon’s LEAP Innovation District. The capital will cover new process designs and technologies at Lilly Lebanon API, a future active pharmaceutical ingredient site set to open in 2027, and Lilly Lebanon Advanced Therapies, which opened May 6 as the pharma giant’s first dedicated genetic medicine manufacturing facility. Since 2020, Lilly says, it has spent $21 billion+ on capital expansion projects statewide.

Lilly is hardly Indiana’s only biopharma headliner: In March, Gov. Mike Braun (R) announced that the Indiana Economic Development Corp. is committing $1 billion in tax credits over 10 years to add 100,000 jobs to the Hoosier State’s life-sci and agritech workforce. That would nearly double a statewide life-sci workforce of 52,526 biopharma, life-sci distribution, and agritech jobs (not including 17,473 medical device jobs), according to the online dashboard of state life-sci industry group BioCrossroads. “This investment will make Indiana the re-shoring and expansion epicenter and premier destination for human therapeutics, animal health, agritech, biotechnology, and environmental innovation,” Braun vowed.

Also expanding is Roche Diagnostics, which last year announced plans to grow its North American headquarters campus in Indianapolis by spending up to $550 million through 2030 to establish the 170-acre site as a central manufacturing hub for its next-generation continuous glucose monitoring systems. The expansion is expected to add 650 manufacturing and distribution jobs to the site’s existing 3,200+ employees.

Indiana lags in NIH funding (1,161 awards totaling about $611.594 million) but finished a strong second in both patents (52,526 families) and jobs (49,211 biopharma, research/testing, agritech, and distribution jobs, according to BioCrossroads).

3. Florida (including Jacksonville and Miami-Fort Lauderdale)

Andrew Majdoch, vice president of technical operations at GBI Biomanufacturing in Plantation, FL. Earlier this year GBI, a biologics CDMO, doubled its stem-cell manufacturing capacity by expanding its Plantation site to all of the 40,000-square-foot 1850 N.W. 69th Ave. from 28,000 square feet. GBI has long manufactured therapeutic proteins and antibodies there. [GBI Biomanufacturing]

Johnson & Johnson (J&J) Vision Care in June announced a $1 billion expansion of its Jacksonville, FL, operations for its Acuvue^® contact lenses. Plans call for a new Northwest distribution facility (50 jobs), set to be fully operational in 2028, and new equipment at its existing Southside manufacturing and corporate site within the Deerwood Park campus. Earlier this year GBI Biomanufacturing, a biologics CDMO, doubled its stem-cell manufacturing capacity by expanding at its Plantation, FL, site to all of the 40,000-square-foot 1850 N.W. 69th Ave. from 28,000 square feet. GBI Bio has long manufactured therapeutic proteins and antibodies there. And in January, Novartis chose the Orlando suburb of Winter Park, FL, for its fourth of five planned radioligand therapy (RLT) manufacturing facilities across the United States. The 35,000-square-foot site is set to open in 2029.

In Pensacola, FL, biotech manufacturing tools and services giant Cytiva last year completed the construction of new filtration manufacturing lines designed to increase the production capacity for filter membranes for North America by 20%. ILiAD Biotechnologies, based in the Fort Lauderdale suburb of Weston, FL, closed in February on an oversubscribed $115 million Series B VC financing whose proceeds are intended to fund advancement of a next-generation pertussis vaccine candidate, BPZE1. And in the Miami suburb of Coral Gables, FL, Catalyst Pharmaceuticals found a buyer in May when Italy’s Angelini Pharma agreed to acquire the rare neuromuscular and neurological disease drug developer for approximately $4.1 billion.

South Florida’s emergence as a biotech hub is reflected in the region hosting the first conferences held in the region by three industry groups, the Biotechnology Innovation Organization (BIO), LSX, and the Miami Biotech Collective. Last December, the Collective partnered with J.P. Morgan to launch an annual summit after being created earlier in 2025 by about 500 industry executives and investors.

Florida is a strong second in NIH funding (2,232 awards totaling $1.38 billion) but third in jobs (40,000 according to BioFlorida, which includes medical device jobs, a category tallied by South Florida Business Journal at 24,000 alone) and fourth in patents (4,682 families).

4. Georgia (including Atlanta and Augusta)

UCB announced plans in March for a $2 billion, 460,000-square-foot biologics manufacturing facility near Atlanta in suburban Gwinnett County. The Belgian biopharma bought 79 acres from the Rowen Foundation, which is developing a 1,900-acre campus on Highway 316, launched in 2020 as a “knowledge” community for biopharma and other innovation-based fields. Gwinnett County has committed $174 million in incentives and infrastructure investments to support UCB’s project, which is expected to create more than 330 permanent jobs once operational.

UCB’s project is the brightest recent success of Georgia life sciences leaders, who are working through industry group Georgia Life Sciences, laid out a 10-year “Roadmap” last year with the aim of “positioning Georgia as the scale-up manufacturing hub of the Southeast.” Other smaller-scale successes include Micron Biomedical, which in May celebrated the grand opening of a 26,000-square-foot manufacturing site for its dissolvable vaccine and therapeutic technology in the Atlanta suburb of Alpharetta, GA. And two hours east of Atlanta in Augusta, GA, Manus Bio broke ground in May on an expansion that will enable its biomanufacturing facility to domestically produce artemisinin, a key ingredient in anti-malaria treatments. The project, first announced in 2024 with the Administration for Strategic Preparedness and Response (ASPR), marks the first project of a partnership that has grown to $47.4 million—including $15 million awarded in February toward domestic production of shikimic acid, a key component of the active ingredient in Tamiflu^® (oseltamivir phosphate).

Atlanta is known for longtime life-sci anchors Emory University and the U.S. Centers for Disease Control and Prevention, the state’s largest life-sci employer at 6,500 people, according to Georgia Power’s Select Georgia economic development initiative. More recently, Portal Innovations’ Portal Atlanta at Science Square has grown in less than a year to 30+ startups in biotech and other industries, plus established companies like vaccine developer GeoVax, which relocated its lab and R&D staffers there late last year while moving its HQ within suburban Smyrna, GA.

Georgia showed third in NIH funding (2,153 awards totaling $1.184 billion, fourth in jobs (36,000 according to Lightcast data cited by SelectGeorgia), and fifth in patents (3,447 families).

5. Wisconsin (including Madison and Kenosha)

Wisconsin Gov. Tony Evers (D) (center) joins Fujifilm Biotechnologies executives in May to officially open Fujifilm Cellular Dynamics Inc. (FDCI)’s new headquarters and human-induced pluripotent stem cell (iPSC) development and manufacturing facility in Madison, WI. [Fujifilm Biotechnologies]

Wisconsin’s manufacturing heritage has helped the Badger State build a life sciences industry that, while heavy on diagnostics and medical device development, also includes growing biopharma activity. South of Kenosha in Pleasant Prairie, WI, Eli Lilly has committed $4 billion toward expanding its production of injectable diabetes and obesity therapies by repurposing the 84,000-square-foot facility it acquired from Nexus Pharmaceuticals in 2024 for $925 million and adding two facilities, a 54,166-square-foot office-manufacturing building and a 13,940-square-foot, four-dock warehouse. The project is expected to create 750 jobs.

In May, Fujifilm Biotechnologies executives joined state officials in officially opening Fujifilm Cellular Dynamics Inc. (FCDI)’s new headquarters and human-iPSC development and manufacturing facility in Madison, WI. The facility employs nearly 200 people and will quadruple FCDI’s capacity for its iPSC-based research products and services manufacturing footprint, with an eye to meeting future demand for contract manufacturing of cell therapy products.

Thermo Fisher Scientific in February signed two 20-year lease extensions for 8500-8551 Research Way, a pair of lab buildings totaling 233,694 square feet in the Madison suburb of Middleton, WI, where the life-sci tools giant carries out pharmaceutical product development and clinical research. Catalent last year completed a $45 million production facility expansion in Madison, WI, creating about 200 jobs. The Madison facility provides development, manufacturing, and analytical services for new biologics; houses Catalent’s GPEx^® Lightning cell line technology, designed to create high-yielding mammalian cell lines genetically modified to create large quantities of a desired protein; and features development and manufacturing labs for flexible preclinical, clinical, and commercial production of biologics from 50- to 4,000-liter scale.

The manufacturing heritage gives the state a competitive advantage, according to the public-private Wisconsin Economic Development Corp. (WEDC), which says suppliers from across the state provide $7.8 billion in products to “biohealth” companies, including pharma and life-sci R&D businesses, as well as medical device developers.

Among emerging states, Wisconsin is fourth in both NIH funding (1,557 awards totaling $913.388 million) and patents (17,944 families), and sixth in jobs (30,749 biopharma, research/testing, agritech, and distribution jobs according to WEDC).

Regional and State Clusters to Watch

Phoenix, AZ

Staffers at Madrid-based ROIS, the contract development and manufacturing organization (CDMO) arm of ROVI, celebrate plans to add U.S.-based sterile production capacity after acquiring a 370,000-square-foot Phoenix manufacturing site from Bristol Myers Squibb. ROIS plans to install an Optima pre-filled syringe (PFS) isolator line in a segregated non-potent area in 2027, which is projected to add ~65–70 million PFS capacity annually once operational. [ROIS]

Madrid-based ROIS, the CDMO arm of ROVI, said April 1 it plans to add U.S.-based sterile production capacity by acquiring a 370,000-square-foot Phoenix manufacturing site from Bristol Myers Squibb. ROIS plans to install an Optima pre-filled syringe (PFS) isolator line in a segregated non-potent area in 2027, which is projected to add ~65–70 million PFS capacity annually once operational. The U.S. facility will be equipped for commercial-scale sterile fill-finish and packaging across vials, PFS and cartridges. Also, cancer drug developer Breakthru Medicine emerged from stealth mode in January, closing on $60 million in Series A VC financing. Breakthru is developing a molecular glue platform, as well as a pipeline of small molecule and ADC candidates.

Regional co-anchors include the downtown 30-acre Phoenix Bioscience Core, home to Arizona’s three public research universities—the University of Arizona, Northern Arizona University, and Arizona State University (ASU). In April, ASU broke ground on a 200,000-square-foot “ASU Health” building in Phoenix that will house the John Shufeldt School of Medicine and Medical Engineering, set to matriculate its inaugural class in August. Another anchor district is the 120-acre Discovery Oasis site, home to Mayo Clinic and MedTech Accelerator, a flagship program of Mayo and Arizona State University Alliance for Health Care. Mayo Clinic is carrying out a $1.9 billion, 1.2 million-square-foot expansion that will increase clinical space at the Phoenix campus by nearly 60% with a new procedural building, a four-floor expansion of the Mayo Clinic Specialty Building, 11 new operating rooms, and two new patient units with 48 additional beds.

Phoenix-area bioscience grew by 8+ million square feet between 2019-25, Mayor Kate Gallego said last year in her State of the City address. The region’s life-sci workforce expanded to 26,000+ by 2024 (Greater Phoenix Economic Council), up 66% since 2015. Greater Phoenix institutions have won 1,470 NIH awards totaling $538.4 million, while inventors have been granted patents totaling 1,470 families.

Pittsburgh, PA

ElevateBio and the University of Pittsburgh (Pitt) next year are set to open the 185,000-square-foot BioForge Biomanufacturing Center within Hazelwood Green, a former steel mill site along the Monongahela River. ElevateBio, a Waltham, MA-based CDMO for advanced therapies, will occupy 70% of the BioForge site as anchor tenant and a scientific partner for Pitt by operating BaseCamp Pittsburgh, a 125,000-square-foot commercial gene and cell therapy biomanufacturing hub. [University of Pittsburgh]

The “Steel City” and vicinity have pivoted to life sciences and healthcare since the region’s steel industry began shriveling in the 1970s, with some 95,000 manufacturing jobs lost between 1980-83 alone. A key milestone in Pittsburgh’s life-sci revival should come next year, when ElevateBio and the University of Pittsburgh (Pitt) are set to open the 185,000-square-foot BioForge Biomanufacturing Center within Hazelwood Green, a former steel mill site along the Monongahela River. ElevateBio, a Waltham, MA-based CDMO for advanced therapies, will occupy 70% of the BioForge site as anchor tenant and a scientific partner for Pitt by operating BaseCamp Pittsburgh, a 125,000-square-foot commercial gene and cell therapy biomanufacturing hub.

Among home-grown companies, Peptilogics in March launched its pivotal Phase II/II RETAIN registration trial (NCT07214311) assessing its lead program of its first-in-class anti-biofilm drug candidate PLG0206 as an irrigation solution to treat prosthetic joint infections. Peptilogics last year completed an oversubscribed $78 million Series B2 financing whose proceeds are funding the trial. Pittsburgh is one of the Top 20 metropolitan statistical areas (MSAs) identified last November by Nasdaq Entrepreneurial Center as “America’s Entrepreneurial Growth Engines,” a report that included three other regions listed here: Atlanta, Minneapolis-St. Paul, and Richmond, VA.

Pittsburgh’s life-sci efforts won national attention in February, when members of the National Security Commission on Emerging Biotechnology visited BioForge and the Carnegie Mellon University (CMU) Biological and Chemical Innovation Cloud Lab at the AI Science Foundry, which enables AI-guided autonomous research within and across biology, chemistry, and metals and alloys. According to Pittsburgh Life Sciences Alliance, the region has a life-sci workforce of 22,268, a lab/life-sci space inventory of 1.7 million square feet, and finished last year with $247 million in VC raised over 15 years.

Greater Richmond, VA

A rendering of the $5 billion manufacturing facility that Eli LIlly plans to build in Virginia’s Goochland County near Richmond. The planned manufacturing plant will be the company’s first-ever dedicated, fully integrated active pharmaceutical ingredient (API) and drug product facility for the pharma giant’s bioconjugate platform and monoclonal antibody portfolio. [Eli Lilly]

Greater Richmond last year attracted the largest of Virginia’s three multi-billion-dollar biomanufacturing projects announced by pharma giants: Eli Lilly last September announced a $5 billion manufacturing plant just west of Richmond in suburban Goochland County that will be the first-ever dedicated, fully integrated active pharmaceutical ingredient (API) and drug product facility for the pharma giant’s bioconjugate platform and monoclonal antibody portfolio. The plant, set to employ 650 people, will manufacture APIs for therapies designed to treat cancer, autoimmune diseases, and other disorders—including ADCs.

The Richmond region’s life-sci anchors include the Alliance for Building Better Medicine, a stakeholder group in Richmond and nearby Petersburg, VA, that promotes advanced pharmaceutical manufacturing, workforce development, and supply chain development; and the 34-acre VA Bio+Tech Park, home to nearly 70 companies, research institutions, and state and federal labs, plus the VA Bio+Tech Center incubator. Also in Richmond is Phlow, a CDMO emphasizing advanced development and manufacturing within the U.S. Last October, the company won inclusion in the FDA’s first Commissioner’s National Priority Voucher (CNPV) Pilot Program when the agency selected Phlow-produced ketamine among nine products for accelerated agency reviews.

Haleon last year committed $54.2 million toward upgrading and expanding its global R&D Center of Excellence, one of three it operates worldwide. The project will also enable the over-the-counter drugmaker to launch a five-year paid internship program with Richmond-based Virginia Commonwealth University and other state schools. Haleon is the former consumer healthcare business of GlaxoSmithKline (GSK), spun out in 2022.

Greater Richmond’s numbers, smaller than many emerging regions, are trending positively: The region has won 1,203 NIH awards totaling $707.5 million and generated patents totaling 904 families. According to the Greater Richmond Partnership, the region has a life-sci workforce of 7,650 jobs while its startups raised $80.135 million in VC funding last year.

South Carolina

In Columbia, SC, Ritedose, a contract development and manufacturing organization (CDMO) specializing in blow-fill-seal (BFS) technology and generic drug manufacturing, has a $17 million, two-phase expansion of its 16,000-square-foot cGMP laboratory coming on line later this year. [Ritedose]

Swedish-based Octapharma, the world’s largest privately owned and independent fractionator of plasma used toward creating hematology, immunotherapy, and critical care treatments, announced plans June 29 to expand within South Carolina by moving its North American headquarters and its 300 jobs from Charlotte, NC, to its suburb of Rock Hill, SC. There, Octapharma plans to build a $1.5 billion combined HQ/advanced manufacturing facility with 1,200 new jobs. In June, GNQ Insilico—which helps precision medicine developers through a platform integrating AI, quantum computing, and multiomics insights at the systems biology level—opened an initial lab at the Clemson University Biomedical Engineering Innovation Campus (CUBEInC), with plans to relocate its headquarters to Greenville from Pleasanton, CA, and base 20-30 people there by year’s end.

Greenville is one of the Palmetto State’s hubs for life-sci activity; others include Charleston, SC, and Sumter, SC. In Columbia, SC, Ritedose, a CDMO specializing in blow-fill-seal (BFS) technology and generic drug manufacturing, has a $17 million, two-phase expansion of its 16,000-square-foot cGMP laboratory coming on line later this year.

According to life-sci industry group SCbio, South Carolina has a life-sci workforce of 63,725 people and saw $63.1 million in bioscience VC raised in 2023 (latest available figure), with Medical University of South Carolina launching two funds totaling $30 million last year to help grow life-sci and healthcare companies. James Chappell, PhD, SCbio’s president and CEO, acknowledges the state needs more lab space for startups. SCbio hopes to meet that need by partnering with a developer—but that challenge could be met if any of several life-sci employers proceed with new or expanded facility projects now in the works but not yet disclosed.

Utah

Cytiva staffers outside the company’s newly expanded Logan, UT, facility at a ceremony celebrating completion of the company’s animal-derived component-free (ADCF) liquid media expansion facility (A1X) project. The expansion effectively doubles the site’s liquid media production capacity, a project designed to support supply chain continuity for customers relying on the company for their cell culture needs. [Utah Governor’s Office of Economic Development, Gov. Spencer Cox (R)]

Salt Lake City is home to AI-based drug developer Recursion, which is making news beyond its pipeline. In May, Recursion-supported accelerator Altitude Lab—SLC is an average 4,327 feet above sea level—announced that its portfolio companies had raised more than $205 million in early-stage funding since its launch in 2020.

Two of those companies raised Series A rounds last year: Peel Therapeutics garnered $20 million toward development of PEEL-224, an optimized TOP1 inhibitor, while Rebel Medicine collected $7.5 million to help develop its non-opioid pain candidate Alevatrix, a bupivacaine reformulation. As for Recurison, the company in May reported encouraging initial safety and PK data in its Phase I/II DAHLIA trial (NCT06678659) of RBM39 degrader REC-1245, a candidate vs. solid tumors and lymphoma, and dosed its first patient in a Phase I ENLYGHT trial (NCT07517198) assessing another cancer-fighting candidate, REC-4539, one designed to target epigenetic drivers.

In March, VC firm Portal Innovations announced plans to expand into Salt Lake City, where it will establish “Woodbine Labs, Powered by Portal,” a 30,000-square-foot incubator set to open in the second quarter of 2027, and create a fund to support early-stage life sciences and biotech startups. Statewide industry group BioUtah is supporting Portal, whose new facility would nurture startups spinning out of the University of Utah and Brigham Young University.

Outside the Salt Lake City region, biotech manufacturing tools and services company Cytiva has completed an expansion of its Logan, UT, facility—namely a new animal-derived component-free (ADCF) liquid media expansion facility (A1X)—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.

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Five Steps to Enable Self-Optimizing Process Purification

Edusehat — Thu, 13 Aug 2026 01:45:12 +0700

Self-optimization is the next evolutionary step for bioprocessing purification systems and, although that step is near, it currently is hindered by missing integrations among its many parts and by loose usage of precise concepts.

At an operational level, self-optimized purification is supported by process analytical technology, hard and soft sensors, digital twins and digital shadows, physics-informed modeling, and real-time optimization capabilities. In that milieu, biopharmaceutical processing is the most advanced. What it lacks, however, is operational comparability linking measurement, state estimation, model updating, decision support, and closed-loop actions for purification platforms and components such as membranes, adsorption and cyclic gas separations, chromatography, and integrated purification trains.

“The limiting factor across domains…is the incomplete integration of measurement design, hidden-state estimation, updating, uncertainty, control authority, governance, and economic justification under realistic drift and scale change,” Vasileios M. Pappas, PhD, post-doctoral senior researcher, University of Thessaly in Greece, explains in a recent review.

To remedy this, Pappas recommends:

Prioritizing sensors and sampling strategies that identify the critical hidden states of each purification platform
Reporting model-updating, recalibration and invalidation rules explicitly, “rather than treating them as implementation details”
Including “drift, delay, sensor failure, feed disturbance, cleaning/regeneration history, and scale transfer [data] as part of the validation process, in addition to nominal operating data
“Distinguish[ing] advisory, supervisory, and autonomous authority as well as fallback logic when model confidence is insufficient”
Reporting economic, regulatory, cybersecurity, and data-governance constraints alongside predictive accuracy

For biomanufacturers, these steps enable more thorough integration throughout the purification process. They enable “trustworthy self-optimizing purification…by demonstrating that process state, model confidence, and operating authority remain linked under disturbances that matter industrially,” Pappas elaborates. “The central test is whether the system can infer the hidden state early enough, update itself responsibly, and support or execute an operating action that protects purity, recovery, productivity, safety, and robustness.”

The path to self-optimization

Before implementing those suggestions, however, Pappas stresses the importance of “conceptual precision” as a starting point, noting loose usage of the defining terms. “A regressor trained on historical campaigns is not a twin simply because it runs online,” he notes, by way of illustration.

“Conceptional precision matters because the operational value of a digital layer depends on what it can infer, how it stays calibrated, how uncertainty is treated, and whether its output can support a qualified operating decision,” he points out. Using the correct specifications for the digital layers minimizes misunderstandings, thereby increasing the chances that the initial concept and the final design agree.

Otherwise, Pappas notes, the system may revert to monitoring and offline optimization or becomes a digital shadow—a model updated from processing data—rather than the operationally-significant bidirectional model known as a digital twin.

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Allogeneic CAR T Company Prepares to Widen Access to Patients

Edusehat — Thu, 13 Aug 2026 01:45:11 +0700

A CAR T-cell therapy company using donor cells is planning to move to a pivotal Phase III clinical trial thanks to their genome editing strategies and optimization of their product, including choice of donors under 30.

Caribou Biosciences says its allogeneic CAR T-cell therapies have been shown to be as effective and long lasting as approved autologous CAR T therapies in certain blood cancers.

“We’ve figured out how to make allogeneic CAR T cells work as well as autologous CAR T cells through a combination of gene editing strategies as well as optimization of the product, such as selecting donors under age thirty,” explains Justin Skoble, PhD, vice president of technical operations at Caribou Biosciences.

According to Skoble, the benefits of allogeneic therapy include having off-the-shelf products available, which will improve patient access.

“What’s novel is our ability to begin addressing the access issues that have been a struggle,” he says. “Depending on indication and who you’re talking with, 75 to 90% of patients eligible for CAR T don’t receive it.”

Skoble says this is “Either because their disease is progressing too rapidly to go through the referral process, cell collection, and manufacture of an autologous CAR T or they face challenges, such as socioeconomic barriers or insurance issues, that prevent them from getting to a center where it’s available.

“We believe an off-the-shelf approach […] reduces the logistical burden because the patient can be dosed with our allogeneic CAR T cells without the need to wait for bespoke manufacturing. We have a donor match strategy where we pick the best match we have in inventory and, because we can scale to hundreds of doses per manufacturing batch, the cost of goods is low.”

The company’s approach involves what Skoble describes as a high-fidelity genome-editing technology chRDNA (pronounced chardonnay), which Caribou Biosciences uses to reduce off-target effects and armor the CAR T cells for functional persistence.

They’ve also found they had better durability of response if they matched the patient’s human leukocyte antigens (HLAs) to donor HLA in inventory, and also if they use donors aged under 30, he says.

The company hopes their approach will transform, and inspire, the CAR T therapy industry. Over the next few months, they hope to start their randomized-controlled Phase III study with the goal of launching their first product, vispa-cel, as a second-line treatment for patients with large B cell lymphoma.

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Bioprocessing’s Green Shift Gains Momentum

Edusehat — Thu, 13 Aug 2026 01:45:10 +0700

As bioprocessing companies race to meet ambitious sustainability goals, the industry’s biggest environmental challenges are becoming increasingly clear, and increasingly solvable. To find out more about this, GEN reached out to CRB—a provider of sustainable engineering, architecture, construction and consulting solutions—and interviewed two of its experts by email: Zach Page-Belknap and Maya DeHart, both energy and sustainability specialists.

According to the CRB experts, reducing carbon emissions and minimizing waste remain formidable obstacles. However, advances in facility design, data management, and energy optimization are giving manufacturers practical ways to make meaningful progress without compromising production.

“Sustainability in bioprocessing presents many challenges, but two of the most difficult to solve at scale are process-heating decarbonization and single-use waste diversion,” they said.

Historically, high-temperature processes have relied on natural-gas boilers, making them among the largest contributors to operational carbon emissions. Electrifying these systems can dramatically reduce fossil-fuel use, but it also places greater demand on a facility’s electrical infrastructure and backup power systems. As Page-Belknap and DeHart pointed out, “emerging technologies such as high-temperature heat pumps offer promising alternatives, although adoption has been slowed by high upfront costs, safety considerations, and the relatively immature market for the technology.”

Meanwhile, facilities that depend heavily on disposable plastic equipment—particularly those involved in autologous manufacturing—face a different challenge. Although methods exist to divert decontaminated single-use plastics from landfills, economics and limited recycling infrastructure continue to restrict widespread implementation.

The CRB team noted that waste diversion can be financially competitive when facilities are located near processors capable of handling specialized materials. However, organizations without local treatment options often face disposal costs more than twice those of conventional waste management.

Data makes the difference

Despite those challenges, one of the industry’s biggest sustainability breakthroughs has come from something far less visible: better data. Rather than relying on isolated information from individual systems, many companies are investing in centralized platforms that track energy, water, and waste across entire facilities. That broader view allows operators to identify inefficiencies, prioritize upgrades, and increasingly leverage artificial intelligence to optimize performance.

“It’s fair to say that meters and monitors themselves do not improve sustainability in bioprocessing directly,” continued Page-Belknap and DeHart. “They are only a means to improve visibility into the actual operational performance of a facility.”

Installing that monitoring infrastructure is often far less expensive than major equipment replacements. Individual meters typically cost around $5,000 to install, although larger projects involving dozens of monitoring points and upgraded management systems can range into the hundreds of thousands of dollars.

For commercial bioprocessors looking to improve sustainability, resisting the temptation to chase the newest technology first was recommended. Instead, facilities should begin by aligning site-level priorities with broader corporate sustainability goals, benchmarking current performance, and understanding where the greatest opportunities exist.

For newer facilities, existing operational data might already reveal low-cost improvements, such as optimizing cleanroom air change rates or making better use of building management systems. Older facilities should take a phased approach by establishing utility baselines, identifying major energy and water consumers, evaluating equipment lifecycles, and incorporating sustainability improvements into planned capital replacement schedules.

As the CRB experts concluded: “Ultimately, sustainability improvement is less about any single technology and more about building the data infrastructure and decision framework needed to invest in the right measures, at the right time, for each specific site.”

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Software Can Solve CGT’s Sectors Traceability Challenges

Edusehat — Thu, 13 Aug 2026 01:45:09 +0700

The most important role of industrial software for the cell and gene therapy sector is the ability to precisely trace materials from collection site through the plant and back to the patient. So says Jonathan Wofford, CCO at Phoenix, Arizona software developer, Title21 Health Solutions, who told GEN, “CGT manufacturing typically involves fragmented, bespoke workflows.

“The complexity is compounded by several variables, including diverse collection methods and protocols, highly variable starting materials, operator techniques, and challenging logistics. Therapies are time-sensitive and orchestration between collection centers, manufacturing facilities, and treatment centers is critical.

“As a result, CGT manufacturing requires coordination across a complex ecosystem that includes clinical operations, manufacturing, supply chain, quality, and data management, unlike mature and standardized mAb production processes.”

And this is where software makes its biggest, if somewhat underacknowledged, impact, according to Wofford.

“The primary value of software systems is not replacing or automating manufacturing processes, but rather bringing control, standardization, and traceability of manual data workflows,” he pointed out. “CGT manufacturing is variable and typically involves multiple changes in custody…each handoff creates opportunities for human error, documentation discrepancies, and compliance risk.”

Traceability in the plant

Software can also enhance the traceability of data within the manufacturing facility.

“Software platforms can help bring quality control and standardization to data entry and workflow management manufacturing through multiple features,” he continued, using electronic batch records as an example. “Industrial software system can also facilitate real-time chain-of-identity and chain-of-custody tracking, with live dashboards and analytics capabilities to drive enhanced insights into production runs, as well as real-time automated warnings for in-process issues or data values.”

The ability to trace materials and data in the plant is always going to be important, even if technology advances allow CGT process developers reduce the requirement for manual handling.

“Adoption of closed-system manufacturing may increase over time. However, today, software systems remain a more immediate solution for improving data accuracy, reducing manual data entry and processes, strengthening compliance controls, and driving analytics,” said Wofford, noting that this is where the emerging cell and gene therapy industry should focus its efforts rather than on promising yet untested technologies like artificial intelligence (AI) based process control systems.

“CGT organizations should be prioritizing technologies that can deliver meaningful and measurable operational and compliance benefits now,” he emphasized. “In the not-too-distant future, AI will likely contribute to predictive manufacturing and autonomous process control. But the greatest value today lies in targeted applications that improve efficiency, strengthen compliance, accelerate batch reviews, and generate actionable insights from complex data.”

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Steroid Curbs Chronic Lung Inflammation Without Weakening Immune Defenses

Edusehat — Wed, 12 Aug 2026 11:25:08 +0700

Nontuberculous mycobacterial infections have become an increasing concern for people with chronic lung disease, in part because the infections can linger despite months of antibiotic treatment. Among the most common culprits is Mycobacterium avium, which can trigger persistent inflammation in the lungs and leave patients with ongoing symptoms even when antimicrobial therapy is underway.

Now, researchers from Trinity College Dublin and St. James’s Hospital report that dexamethasone, a widely used anti-inflammatory steroid, reduced inflammatory signaling in human macrophages infected with M. avium without compromising the cells’ ability to control bacterial growth. The study, “Dexamethasone Reduces Glycolysis and Inflammation in Human Macrophages Infected With Mycobacterium avium Without Compromising Bacterial Control,” was published in The Journal of Infectious Diseases.

The findings point to a possible host-directed strategy for nontuberculous mycobacterial disease—one aimed not at killing the bacteria directly, but at limiting the damaging inflammation that can accompany chronic infection. “Our study suggests that it may be possible to fine-tune this response by reducing damaging inflammation while still preserving the immune defenses that help control infection,” said Donal Cox, PhD, senior author of the research at Trinity College Dublin.

To test that idea, the team studied human monocyte-derived macrophages—immune cells that serve as a first line of defense against infection. The cells were treated with dexamethasone before being infected with M. avium subsp. hominissuis 104. The researchers then used real-time metabolic flux analysis to measure glycolysis and oxygen consumption, RT-qPCR to assess metabolic and antimicrobial gene expression, colony-forming unit assays to measure bacterial burden, and ELISA assays to quantify inflammatory cytokines.

The study showed that M. avium infection drove a glycolytic response in macrophages, but dexamethasone dampened that metabolic shift. The steroid reduced glycolytic proton efflux and lowered expression of glycolysis-associated enzymes, including PFKFB3, GAPDH, and PKM2, while leaving oxygen consumption unchanged. Importantly, dexamethasone did not increase the recoverable bacterial burden over 120 hours, according to the authors.

The anti-inflammatory effects were broad. Dexamethasone significantly reduced production of TNF, IL-6, IL-8, and IL-1β in infected macrophages, and at the higher dose abrogated induction of IL-10, the authors report. It also did not alter the expression of several NADPH oxidase complex genes, supporting the authors’ conclusion that key antimicrobial functions may be preserved. The paper stated that the findings support “further investigation of dexamethasone as a potential host-directed strategy to limit inflammation while preserving host defense in nontuberculous mycobacterial disease.”

“Current treatment strategies for NTM disease focus primarily on killing the bacteria,” added Cox. “However, inflammation itself can contribute significantly to symptoms and tissue damage in patients, so finding ways to control inflammation without impairing antimicrobial innate immunity offers a potential gateway to much more effective therapies.”

The authors cautioned that the work was performed in macrophages from healthy donors and used a single M. avium strain, so the results will need to be validated in models that better reflect patients with NTM disease, including alveolar macrophages, macrophages from susceptible patients, and clinical M. avium isolates. Future studies will also need to test steroid treatment after infection and in combination with antimycobacterial therapy before dexamethasone can be considered as an adjunctive approach for chronic NTM lung disease.

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Parasite-Specific Protein Helps Toxoplasma Adapt to Crowded Conditions

Edusehat — Wed, 12 Aug 2026 07:50:07 +0700

Toxoplasma gondii is a parasite that infects hundreds of millions of people around the world. Although cases are often mild, it can cause severe symptoms in people with weakened immune systems and in developing fetuses. Toxoplasma can also persist for years by forming long-lived cysts in tissues, allowing infection to become chronic.

During chronic infection, hundreds of Toxoplasma parasites can pack into a tissue cyst inside a brain or muscle cell. That crowded life carries a cost: nutrients become harder to obtain, waste accumulates, and energy-producing reactions can become damaging. How Toxoplasma reshapes its metabolism to keep growing under such strained conditions has been unclear.

Now, a study by researchers in the lab of Whitehead Institute Member Sebastian Lourido, PhD, who is also an associate professor of biology at the Massachusetts Institute of Technology (MIT), has identified a parasite-specific protein that helps coordinate this response in Toxoplasma.

The preclinical study carried out in cells and including tests in infected mice revealed a previously unknown way that parasites regulate metabolism and identified a dedicated regulator of metabolic gene expression in apicomplexans, the group of parasites that includes Toxoplasma and the Plasmodium parasites that cause malaria.

The protein, TgPRO, allows Toxoplasma to manage oxidative stress—the buildup of reactive oxygen molecules that can damage cells—by controlling genes involved in energy production and iron use. Led by Christopher Giuliano, PhD, a former graduate student, and by graduate student Chinmay Kalluraya in the Lourido lab, the study in addition points to a possible future therapeutic strategy, indicating that inhibiting pathways controlled by TgPRO could make Toxoplasma more vulnerable to antiparasitic drugs that induce oxidative stress.

Giuliano and Kalluraya are co-lead authors of the researchers’ published paper in Cell, titled “Convergent evolution of metabolic regulation governs redox adaptation in Toxoplasma,” commenting in their report, “TgPRO is likely critical for the efficient transmission of T. gondii by enabling metabolic adaptation during chronic stages.”

Different organisms adjust metabolic gene expression during crowding, when they encounter nutrient scarcity, oxidative stress, and waste accumulation, the authors noted. “Organisms adapt to these stresses through either broad repression of biomass production or focused modulation of specific pathways.” Apicomplexans also encounter crowded environments as part of their infection cycles, the team continued. “However, while apicomplexan parasites experience these stresses during intracellular growth within host cells, they lack known regulators of metabolic adaptation.”

To discover the genes that support Toxoplasma’s ability to live in crowded cells, the researchers used a genome-wide CRISPR screen to compare Toxoplasma growing at low and high densities. The screen tests the effects of turning off genes one by one at both population densities in order to determine which genes are essential, specifically in crowded conditions. The results highlighted pathways that make or recycle NAD and NADP, molecules important for energy production and defending against oxidative damage. It also pointed to TgPRO, a previously unstudied protein that was especially important when parasites became crowded. “Nicotinamide adenine dinucleotide (NAD)(P)+ biosynthesis was required at high parasite density, along with several parasite-specific factors, including an RNA-binding protein we named ‘‘T. gondii parasite response to oxidation,’’ (TgPRO),” they stated.

“A genome-wide screen was a powerful way to ask how crowding affects parasite fitness,” Kalluraya said. “TgPRO emerged as very important at high density. Because almost nothing was known about it, we wanted to understand what it was doing.”

The researchers’ study showed that parasites lacking functional TgPRO accumulated more reactive oxygen molecules and struggled to compete at high density. Experiments showed that the loss of TgPRO disrupted the mitochondria and changed how parasites processed glucose and other nutrients. Providing additional iron or restoring an important chemical balance inside the mitochondrion improved parasite growth, connecting TgPRO’s effects to iron-dependent energy metabolism. “Collectively, TgPRO enables parasites to maintain redox balance under the metabolic strain that accompanies crowded environments,” they noted.

The team then traced the response to a molecular mechanism. TgPRO is an RNA-binding protein, attaching to the molecular messages (RNAs) that cells use to make proteins. The researchers found that it binds and stabilizes a select set of messages involved in nutrient use, mitochondrial activity, and the assembly of iron-sulfur clusters, small structures that many enzymes need to function. The experiments connected the original observation—that some parasites faltered only when crowded—to a precise interaction between a regulatory protein and its RNA targets.

“One of the really nice elements of the story is our ability to connect it all the way through—from the original observation and genome-wide screen to the metabolic consequences and the direct interaction between TgPRO and its target RNAs,” Lourido said. The researchers found that lowering oxygen levels also reduced oxidative stress and partially restored the growth of parasites without TgPRO. Toxoplasma is commonly grown in laboratories at atmospheric oxygen levels, which are considerably higher than those found in most animal tissues. The result suggests that oxygen conditions can strongly shape parasite metabolism, and the researchers caution others studying Toxoplasma to take this into consideration.

After testing the role of TgPRO in artificially crowded settings, the team also tested whether TgPRO matters during chronic infection, when Toxoplasma forms cysts in the brain. Mice infected with parasites lacking functional TgPRO developed smaller brain cysts, suggesting TgPRO supports parasite growth in the naturally dense environment of a chronic-stage cyst.

“The chronic stage is still somewhat elusive,” Giuliano said. “Showing that TgPRO affects cyst growth suggests that these same metabolic changes are needed in the brain and gives us clues about how the parasites persist there for months or years.”

TgPRO bears little resemblance to the proteins that regulate similar metabolic programs in mammals, yeast, and bacteria, yet it controls many of the same kinds of genes that these organisms adjust when cells face oxidative stress or changing nutrient conditions. This is an example of convergent evolution: distantly related organisms evolved different molecular machinery to solve a similar biological problem. That convergence suggests that coordinating these metabolic pathways may be a fundamental requirement for cells adapting to stress.

The study establishes a new paradigm for how apicomplexan parasites regulate their metabolism and advances the foundation for investigating how Toxoplasma persists inside its hosts. “Through posttranscriptional control of a coherent set of metabolic pathways, TgPRO allows T. gondii to adapt to oxidative stress, which particularly impacts chronic-stage cation,” the authors concluded. “Within apicomplexan parasites, this pathway represents a unique example of an environmentally responsive regulator driving metabolic adaptation.”

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Bispecific Antibody Purification’s Complexity Problem and How to Fix It

Edusehat — Wed, 12 Aug 2026 04:15:08 +0700

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Much has been written about how bispecific antibodies have expanded the biologics landscape by making it possible to engage multiple targets simultaneously and unlock new mechanisms of action. But their structural complexity introduces significant workflow challenges for downstream purification that are often more pronounced than those associated with traditional monoclonal antibodies.

Figure 1: Bispecific antibodies come in a multitude of different formats. Examples are shown above. [Thermo Fisher Scientific]

Part of the challenge is that unlike monoclonal antibodies, bispecifics frequently generate a different array of product-related variants, including mispaired light and heavy chains, half antibodies, homodimers, and aggregates that can be difficult to resolve due to their physicochemical differences. Additionally, some bispecific formats exhibit reduced compatibility with conventional purification approaches such as protein A affinity chromatography.

To move bispecific therapies into the clinic faster, developers need robust purification processes that can achieve high purity and yield. This is where approaches that leverage analytical insights with flexible process design become increasingly important. In a recent webinar, Joshua Orchard, a field applications staff scientist at Thermo Fisher Scientific, used a series of case studies to outline the key purification challenges of bispecific antibodies and how analytical insights can guide the selection and optimization of affinity and non-affinity chromatography steps.

A central theme of the webinar is that the purification should be guided by the analytics. The diversity in format, size, domain architecture, and binding configurations of bispecifics create something of a moving target, where no single platform can be universally applied. Being successful requires “listening to what the analytics are telling us” and “what directions we need to go” in terms of what technologies to use, Orchard said. “We’ve got all of these bispecific antibodies that have so many different challenges [and] so many unique situations” and so “we have to make smart decisions.”

Figure 2: Structure of bsAb target molecules and related impurities. [Thermo Fisher Scientific]

Case study one: light chain–driven separation

The first case study involved bispecifics that contained both kappa and lambda light chains. Initial experiments focused on screening several resins to identify those with a high dynamic binding capacity for different bispecifics. Within the kappa-targeting resins, the CaptureSelect^TM KappaXP Affinity Resin and a protein L affinity resin demonstrated the strongest performance of those tested with binding capacities exceeding 40 mg/mL.

Subsequent pH-gradient elution experiments (from pH 6 to pH 2.5) revealed a clear separation between the target bispecifics and homodimer impurities, with the highest resolution coming from the CaptureSelect KappaXP resin.

Figure 3: Comparison of the dynamic binding capacities of commercially available affinity resins targeting antibody light chains. [Thermo Fisher Scientific]

Further optimization required the use of elution modifiers. Experiments were conducted at 10 g/L loading over a 20-column volume gradient, evaluating various modifiers including sodium citrate and magnesium chloride. Of the modifiers tested, magnesium chloride had the most pronounced increase in separation. Elution modifiers also increased the retention of bispecific product on the CaptureSelect^TM LambdaXP Affinity Resin and successfully removed mispaired species. Purity increased from approximately 87% in the load to as high as 98% post-separation, with substantial reductions in aggregates, half antibodies, and free light chains.

Figure 4: Credit: Thermo Fisher Scientific

Despite these gains, there are trade-offs. Specifically, better separation often came at the cost of reduced yield, and the higher elution modifier concentrations required to achieve separation may be challenging to scale-up.

Case study two: aggregate reduction at capture

Improving process efficiency is at the heart of this next case study. Specifically, reducing the burden on downstream polishing by addressing high aggregate levels earlier in the workflow. The starting point was a bispecific antibody with elevated aggregate content (~17–23%) that was not effectively removed during traditional protein A capture. For the study, the scientists evaluated another Fc-targeting resin that binds a different epitope than protein A, CaptureSelect^TM FcXP Affinity Resin.

According to the results, the MabSelect SuRe^TM LX protein A resin had a typical elution profile and the resulting pool contained ~91.3% monomer and ~8.4% aggregate. In contrast, the CaptureSelect FcXP resin produced a distinct elution profile with about 99.7% monomer and only about 0.2% aggregate.

The scientists then extended the approach to a more challenging bispecific with 23% aggregate. Using the alternative Fc-binding resin, scientists obtained a clearer separation with reduced aggregate content of less than 5% in the elution pool, monomer purity of up to 99% in optimal fractions, and overall recovery of above 80%.

Case study three: leveraging CH1 selectivity for complex mixtures

Figure 5: Variant forms of BsAb CD3-TAA–expressed products. [BioProcess International]

This next case study demonstrated how the molecular architecture of bispecifics can directly inform resin selection. In this case, the target bispecific presented a key challenge. Specifically, protein A co-eluted both active and inactive species which limited its effectiveness for purification. Additionally, the impurity profile included homodimers and half antibodies along with excess free light chain.

These characteristics made CH1 affinity chromatography a logical alternative. That is because CH1 resins selectively bind antibodies containing the CH1 domain but do not bind free light chains making it possible to exclude the three types of impurity during capture, with only minor residual impurities.

Figure 6: Analytical SEC HPLC results post affinity purification. [Thermo Fisher Scientific]

According to the results, using the CaptureSelect^TM CH1-XL Affinity Resin produced a markedly cleaner elution profile than protein A, particularly for homodimer species. In fact, monomer purity improved from 72% with protein A to 98% with CH1-XL. Also, the mild elution conditions used in this experiment reduced the aggregation levels significantly.

Case study four: charge-based separation of complex variants

A particularly challenging case study focused on a bispecific with an Fc domain fused to single-chain variable fragments, leading to the formation of multiple variants including disulfide-linked diabodies. This example shifts the focus away from affinity chromatography toward the application of ion exchange resins for the removal of product-related impurities.

According to the data, initial weak cation exchange HPLC analysis revealed separation between monomer charge variants and the diabody species suggesting that charge-based methods might be an effective preparative tool. As a first step, scientists evaluated separation across increasing pH conditions. The clearest separation was achieved at pH 8.1 which resulted in about 98% monomer purity and effective variant removal. Then the team evaluated the performance of the POROS^TM 50 HS Strong Cation Exchange Resin against a smaller particle size resin. Though the initial chromatogram showed a slightly lower resolution of the species, the analytical data revealed a better performance. Starting from a load with about 89% purity and 12.5% diabody content, the resin reduced the diabody to below detectable levels in the elution and achieved ~74% yield, compared to 68% from thealternative resin in the study.

Figure 7: Yield and purity results of two resins and flowrates. {Thermo Fisher Scientific]

Furthermore, the data showed that increasing the flow rate improved process efficiency with some tradeoffs including reduced yield and partial reappearance of the diabody species. It suggests that further gradient optimization could improve the resin performance.

Case study five: exploiting pI differences

Figure 8. Target bsAb and homodimer impurities. [Thermo Fisher Scientific]

This case study demonstrated how charge differences can enable high-resolution separation. In this example, the system consisted of the target bispecific and a homodimer impurity. Here the target bispecific with a pI of ~7 was successfully separated from a homodimer impurity with a pI of ~6 using anion exchange chromatography.

Initial screening across multiple resins identified the POROS^TM 50 HQ Anion Exchange Resin as offering superior separation potential of the resins screened in this study. By optimizing the pH, conductivity, and loading conditions, this approach achieved greater that 90% heterodimer yield and about 99% removal of the homodimer impurity. An additional benefit of the approach was a three-fold reduction in aggregate levels.

Case study six: reversing the strategy based on charge profile

This case study builds on the previous example with a variation in impurity behavior. It involves an asymmetric IgG captured using protein A followed by a polishing step to reduce product-related impurities that accounted for about 30% of the product pool. Importantly, these impurities had a higher isoelectric point (pI) than the intact bispecific unlike the previous case where the target molecule had a higher pI.

Figure 9. Purity results with POROS 50 HQ AEX resin at different column loadings. [Thermo Fisher Scientific]

This flip in pI necessitates a different purification strategy. For this product, scientists evaluated a bind-and-elute anion exchange approach using the POROS 50 HQ Anion Exchange Resin. The goal was to selectively retain and remove higher pI impurities during washing while recovering the target bispecific. After testing a range of conditions, the scientists found that at pH 8 and 5 mS/cm conductivity, the intermediate wash demonstrated effectiveness with higher loading column which correlates to improved removal of byproducts.

There was a trade-off as step yield decreased when loading increased, with overall recovery ranging from about 53% to 63% pointing to some possible opportunities for optimization particularly balancing impurity clearance with yield.

Case study seven: resolving light chain mispairing using charge-based separation

Charge-based separation can also address light chain mispairing as this next case study demonstrated. Correctly paired bispecifics display distinct, positively charged surface patches, while mispaired species disrupt this charge distribution. The differences in surface changes point to the possibility of using cation exchange chromatography.

Initial experiments identified a range of pH 5.5–6.5 and 200–350 mM sodium acetate as the optimal condition for achieving selective separation. Under these conditions, the wash step effectively removed mispaired species while the elution step selectively recovered the bispecific. The process increased product purity from ~60% to ~95%.

Resolving aggregate challenges across modalities

Aggregates remain one of the most persistent challenges for bispecifics developers. While they can be difficult to manage, there are a range of chromatography options that can be helpful for addressing them. “Traditional cation exchange chromatography works very well” and “our POROS XS Strong Cation Exchange Resin has a high dynamic binding capacity, which should help alleviate some of [the] more moderate aggregate issues.” For slightly more severe cases, hydrophobic interaction chromatography can be effective. Resins such as POROS^TM Benzyl Ultra Hydrophobic Interaction Chromatography Resin operate in flowthrough mode and can be easily coupled to upstream anion exchange.

Figure 10. POROS-based chromatography resins that can be used for aggregate removal. [Thermo Fisher Scientific]

For exceptionally high aggregate content, a mixed-mode chromatography solution can provide an added layer of control. By combining ionic and hydrophobic interactions, this approach, using POROS Caprylate Mixed-Mode Cation Exchange Resin, demonstrates aggregate reductions of up to 20%. However, it typically requires more extensive process development.

Also, cation exchange chromatography can contribute to aggregate reduction. Internal studies comparing multiple resins showed that aggregate levels could be reduced from ~7% to below 2% under optimized conditions for a model IgG (pI ~8) following protein A capture.

Conclusion

Bispecific antibodies present a wide range of structural and physicochemical challenges, that makes applying standardized platform approaches significantly more challenging. For the most optimal results, manufacturers need customized purification strategies that account for the structural diversity of these therapeutics. Affinity-based approaches, including CaptureSelect affinity resins, provide valuable options when protein A binding is altered or absent.

Figure 11. Affinity resin toolbox from Thermo Fisher Scientific, including a traditional Protein A resin as well as CaptureSelect resins targeting other antibody subdomains. [Thermo Fisher Scientific]

At the same time, ion exchange and mixed-mode resins offer flexible solutions for separating product-related variants by exploiting differences in charge and hydrophobicity.

Bispecific antibodies are gaining momentum, and the demands on downstream purification will continue to grow. The case studies presented here illustrate how analytical insight forms the foundation of effective process development. Ultimately, effective purification depends on selecting appropriate tools based on analytical insights and systematically exploring process conditions to build workflows that balance purity, yield, and scalability.

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Human iPSC-Derived Heart Assembloids Reproduce Valve Development and Disease

Edusehat — Wed, 12 Aug 2026 04:15:07 +0700

A multi-disciplinary, multi-institutional group of researchers said they relied on their expertise in genetics, mechanics, chemistry, and biology to create a chip the size of a postage stamp to model a particular class of heart conditions.

The team, led by Guang Li, PhD, an associate professor in the University of Pittsburgh School of Medicine’s department of cell biology, has grown heart valves on organoids. The study “Human iPSC-derived heart valve-like assembloids model valve development and disease pathology” appears in Cell Stem Cell and is an important step toward better understanding and treating a number of serious heart disorders, according to the scientists.

This kind of research often depends on animal models, where researchers can study the development of heart valves that grow much quicker than those of humans (which take nearly 10 weeks to fully develop), and don’t raise the same ethical dilemmas as it would in humans. But, Li said, “human valves are very different from animal valves.” Imagine the physiological and genetic differences between a person and, for instance, a zebrafish. “To study human valve diseases, we need human valve models.”

The organoids were grown from pluripotent, adult human stem cells, which can be generated from skin, blood, or other cells, then coaxed into developing into cells from a body part of interest; in this case, a human heart. Different types of organoids can be combined into “assembloids” to better model complex organs that natively originate from combinations of different tissues.

However, a functioning heart is more than a cluster of certain types of cells. Its development and continued operation are dependent, among other things, on a complex interaction of different forces. To build analogs of those forces into the model, Li sought the engineering expertise of colleagues, including Lance Davidson, PhD, the William Kepler Whiteford Professor of bioengineering in the Swanson School of Engineering and Si-Yang Zhen, PhD, a professor of biomedical engineering at Carnegie Mellon University.

“This kind of project is really a hallmark of the community of researchers in Pittsburgh,” Davidson said.

Valve grown on heart assembloid surface

To create a model, Li grew a valve on the surface of a heart assembloid. Then the team stimulated growth by designing ways to mimic the forces that would act on an embodied heart, a flowing medium to simulate blood, an endothelial culture which simulates cells that line heart valves, and even a set of magnetized beads that moved according to the placement of a magnetic belt to simulate muscle contraction.

With the organoid working to simulate a heart with valves, the team now had a model they could use to study four types of valve disorders, including mitral valve prolapse (MVP), a genetic disorder affecting seven to eight million individuals in the US at any given time.

When Li introduced a mutation associated with the disease, the developing valves showed signs of MVP. In other cases, damage was simulated or introduced to mirror the damage that can occur to a person’s valves throughout life in conditions such as valve calcification; cryo-injury; and complications from hypoglycemia and diabetes.

Li was able to begin studying the organoids, identifying some pathways responsible for the development problems associated with MVP and ways they can be corrected. He was also able to develop models for the acquired deficiencies and will go on to look for ways to treat them.

Next, however, Li plans to add complexity to his assembloids, growing them with two chambers and growing the valves inside them, instead of on the surface, to better model a real human heart.

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The State of Biotech 2026

Edusehat — Wed, 12 Aug 2026 04:15:06 +0700

John Maraganore, PhD

Co-Founder and Executive Chair
City Therapeutics

Jeremy Levin, DPhil, MB BChir

Co-Founder and Executive Chairman
Ovid Therapeutics

Shweta Maniar

Director, Healthcare & Life Sciences
Google Cloud

Daphne Zohar

Founder and CEO
Seaport Therapeutics

Broadcast Date: Wednesday, September 23, 2026

Time: 08:00 PDT, 11:00 EDT, 17:00 CET

The world of biotech is showing strong signs of growth and clinical impact while also experiencing new challenges. The explosion of artificial intelligence is reshaping areas from drug discovery and protein folding to gene editing and clinical trials. Recent successes in developing GLP-1s and inhibiting once undruggable cancer targets show the immense power of biotechnology. But upheaval at the FDA, the looming patent cliffs facing several big pharma companies, and growing competition from China, are among the many challenges confronting the industry.

In GEN’s flagship virtual event, The State of Biotech—with exclusive sponsorship by Cytiva—we hear from a distinguished group of business executives, award-winning scientists, and journalists. Among the highlights:

A keynote conversation with John Maraganore, PhD, the founding CEO of Alnylam.

In honor of Sickle Cell Awareness month, 2026 Breakthrough Prize winner Stuart Orkin, MD, presents the research behind the discovery of the fetal-to-adult hemoglobin switch that powered the approval of Casgevy.

A timely conversation about the challenges faced by life sciences and healthcare users in applying artificial intelligence effectively for maximum ROI with Shweta Maniar, global director of Life Sciences Strategy & Solutions for Google Cloud.

Jeremy Levin, MB BChir, DPhil, chairman of Ovid Therapeutics and author of a new book, Biotech in the Balance, argues that we need a globally competitive biotechnology industry built to serve patients and earn lasting public trust.

Our annual catch-up with the hosts of the popular Biotech Hangout podcast—Daphne Zohar, Brad Loncar, and Chris Garabedian.

And in closing, an interview with GEN’s veteran editor in chief, John Sterling, as he prepares to step down after more than four decades with the publication.r) discussing the intersection of AI and CRISPR.

Breakout sessions from the summit sponsors.

Registration is free. We look forward to welcoming you to The State of Biotech!

Produced with support from:

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GLOBE and GUARD approaches ignore the realities of out-licensing at small- and mid-size biotech companies, threaten innovation

Edusehat — Tue, 11 Aug 2026 21:15:03 +0700

There are a lot of reasons to be skeptical of the HHS effort to bring international reference prices into Medicare via the proposed GLOBE and GUARD models. Such efforts are counterproductive, hampering innovation without improving patient affordability.

But the GLOBE and GUARD models are also problematic because they pose a unique danger to small- and mid-size biotechnology companies that develop over half of all new drugs that patients need. These companies are the backbone of an American innovation ecosystem that is the envy of the world, driving the successive waves of innovation that have transformed the treatment of disease after disease.

When smaller companies invent new medicines, the funding that allows them to pursue research and development goals often comes from selling the rights to market their medicines in different countries. That means that the company that developed a given treatment may have no say in how its licensee sells that medicine in another country.

In some cases, a smaller biotech company may sell the rights to market the medicine to multiple different companies, creating an even more complicated arrangement where the company that holds the patent is different from the company that sells the product in the United States, with a third company marketing the medicine overseas.

This is a standard part of the biotech life cycle, a way for companies to raise the funds needed to finance R&D and keep a focus on moving science forward rather than making investments in commercial infrastructure in dozens of countries. But out-licensing also injects complexity into cross-border policymaking. The company that created a drug may have no control over the commercial decisions made by its licensees selling the product in Europe or Asia.

That’s a problem, because GLOBE and GUARD both demand that the prices paid in Europe and countries in other regions be used to determine prices in Medicare. As BIO detailed in its comment letters (see BIO’s comment letter on GLOBE here, and the BIO letter on GUARD here), the realities of biotech out-licensing make those programs unworkable. Once a medicine is licensed, the originator company often has no right to determine commercial decisions, such as price. Additionally, country-specific confidentiality rules create a further hurdle; in some cases, the company selling a medicine in Europe is prohibited by law from providing the licensing company details about net prices.

As a result, the company that controls the pricing of a medicine in the United States may have no legal authority or practical leverage to change international prices. That creates the risk that price controls will be imposed in the United States—harming innovation, particularly the work of smaller companies—without any mechanism to deter foreign freeloading.

BIO’s View

International reference pricing is inherently fraught, imposing values around access and innovation that have devastated drug development in Europe and deprived patients in Europe and other countries of many new treatments. The damage that would be inflicted by international reference pricing is exacerbated by creating new barriers to the kind of collaborative arrangements that have served as a critical tool that sustains smaller biopharma companies at the early stage of research.

These smaller companies already face long odds, and out-licensing provides the fuel to accelerate efforts to bring new medicines to patients at exactly the moment those resources are needed.

Laws that create obstacles to this kind of licensing will inevitably harm the companies that need capital the most, raising the risk that tomorrow’s cures won’t make it over the finish line.

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Greater Richmond’s Pharma Cluster Keeps Compounding

Edusehat — Tue, 11 Aug 2026 21:10:13 +0700

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A year after Eli Lilly and Co. put Greater Richmond on the national map with a $5 billion manufacturing announcement, the region’s advanced pharmaceutical cluster is still adding milestones, most of them driven by companies and institutions already rooted in the City of Richmond and surrounding counties of Henrico, Hanover, and Chesterfield.

Lilly’s September 2025 announcement remains the anchor. The facility, expected to create 650 high-wage jobs and roughly 1,800 construction jobs, will be the company’s first dedicated, fully integrated site for active pharmaceutical ingredients and drug products treating cancer, autoimmune conditions, and other advanced therapies. Lilly CEO Dave Ricks said the company fielded more than 400 proposals from 46 states before choosing the region, citing local workforce potential, incentives, utility access and favorable zoning. The plant is the first of four domestic facilities that Lilly plans to open as part of a $50 billion reshoring commitment.

Within weeks, Lilly, AstraZeneca, and Merck built on that momentum with a $120 million commitment to the Virginia Center for Advanced Pharmaceutical Manufacturing, a workforce training program developed with the Virginia Innovation Partnership Corporation and multiple Virginia colleges and universities. The center aims to graduate 2,000 to 2,500 Virginians annually with a stackable credential or degree, from technician certifications through advanced degrees.

That kind of talent pipeline matters because the region’s pharma cluster didn’t start with Lilly. It traces to 2020, when private companies, elected officials, economic development organizations, and university researchers began meeting to discuss what it would take to reshore pharmaceutical manufacturing. That effort became the Alliance for Building Better Medicine, anchored by Frank Gupton’s, PhD, Medicines for All Institute at Virginia Commonwealth University’s (VCU) College of Engineering, which drew more than $60 million from the Gates Foundation to develop low-cost AIDS drugs and inspired the continuous-manufacturing approach behind Richmond-based Phlow Corp.

The Alliance’s newest milestone landed in July, when it secured $15.9 million from the U.S. Economic Development Administration, the first direct federal funding for Virginia’s Advanced Pharmaceutical Manufacturing Tech Hub since its 2023 designation. The money will be used to launch an “end-to-end” commercialization project in which Phlow and fellow Richmond company Occam Systems will manufacture chemical building blocks and active ingredients for ketamine, midazolam, norepinephrine, and succinylcholine, drugs used in emergency, anesthesia and critical care settings. Civica Rx, a nonprofit drugmaker, will then produce the finished medicines at its nearby plant.

Alliance chairman Robby Demeria called initiative the long-sought step toward a fully domestic supply chain for essential medicines: “These are low-margin medications, but wildly important to sustaining health and conquering disease here in the U.S.”

Greater Richmond’s research infrastructure grew alongside it. In July, Activation Capital, which operates the Virginia Bio+Tech Park in downtown Richmond, announced a roughly 10,500-square-foot addition built through a lease with VCU. The addition will bring 13 new wet labs, including a clean room, to the 10 already operating there, more than doubling the center’s lab capacity. The added space will support Activation Capital’s Basecamp, Pathfinder, Frontier BioHealth, and Pioneer Connect programs, which help life sciences entrepreneurs move from early concept and company formation to investor readiness and commercial production.

Existing employers have expanded alongside the newcomers. Consumer health company Haleon invested $54 million to upgrade its Richmond facility and launched a paid internship pipeline with VCU. Anton Paar, the Austrian precision-instrument maker, continues building out its U.S. headquarters in Hanover County. In Chesterfield, Civica broke ground on a 50,000-square-foot R&D and quality-testing lab expected to grow to 350 local employees, supporting the company’s broader regional manufacturing footprint.

Together, the announcements reflect a strategy that regional leaders have pursued since 2020: build the manufacturing capacity, the research space, and the trained workforce at the same time, rather than waiting for one to follow the other. The region’s Tech Hub designation from the U.S. Department of Commerce in 2023 gave that strategy federal recognition. The steady run of investment since, from Lilly’s $5 billion plant to a 10,500-square-foot lab expansion in downtown Richmond, suggests the region intends to keep building on it.

To learn more, visit www.grpva.com.

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The Future of Aging Research Is Longitudinal, Multiomic, and Single-Cell

Edusehat — Tue, 11 Aug 2026 21:10:12 +0700

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Aging is a complex, lifelong biological process that progressively increases the risk of developing chronic disease. As the global population ages, the prevalence of chronic disease is rising, placing increasing pressure on healthcare systems. Research into the biological processes underpinning aging is therefore essential for improving prevention strategies and developing treatments that support healthier aging. The complex longitudinal nature of aging means that research approaches must capture multiomic insights across long time scales.

Aging cannot be understood in a snapshot

Single-timepoint genomic analysis can inform disease risk and prevention strategies, but it cannot track the dynamic molecular changes associated with aging. By contrast, longitudinal studies that collect samples over years and decades can reveal the sequence of molecular and cellular changes that occur before age-associated phenotypes, such as frailty and cognitive decline, emerge. Studies published in Nature Medicine and Nature Aging suggest that aging processes are highly personalized and progress nonlinearly, highlighting the need to track individual biological trajectories with multiple analyses over long time periods.

Recognizing the value of longitudinal research is an important first step. However, translating that understanding into practice requires robust infrastructure that supports consistent sample collection, processing, and storage, while maintaining resilience to challenges that emerge over the course of a long-term study.

Aging complexity requires multiomic insight

Aging is shaped by a complex network of interacting factors, including environmental exposures, such as diet, treatment history, pollution, and stress, alongside genetic predisposition. As a result, aging manifests in diverse, personalized ways, with different individuals aging faster in different organ systems or biological pathways and ultimately developing different diseases.

The molecular and cellular effects of aging are similarly varied and are not confined to a single omics layer. While genomics remains central to understanding disease risk, epigenetics is particularly important in aging research because it reflects the interplay between environmental influences, molecular changes, and gene expression. Analysis of other omics layers, such as the proteome, can identify clinically accessible biomarkers that capture meaningful biological changes and help translate research into clinical diagnostics.

Single-cell methods expose hidden variation

Aging produces different effects across cell populations within a given tissue, and certain cell types contribute disproportionately to the overall aging phenotype. As a result, bulk omics analyses can miss both the impact of aging on specific cells and the distinct contributions individual cells make to the aging process. Age-associated immune cell dysregulation contributes to increased susceptibility to infections, autoimmune disorders, and other diseases. Senescent cells accumulate with age and play a direct role in driving chronic inflammation, impairing tissue regeneration, and contributing to age-related diseases. Other cell types with important roles in aging include stem cells and organ-specific cell populations that influence cardiovascular and brain health, two major areas of age-associated morbidity. Single-cell omics can identify distinct cell types and define their regulatory and activation states, providing deeper insight into age-related disease processes and rare cell populations.

Integrated services for aging research

Aging research requires scalable infrastructure that preserves samples over long periods and supports consistent processing and analysis. This consistency is essential for generating reliable conclusions from samples collected at different time points and across diverse cohorts. Complementary multiomic platforms are also needed to untangle the complexity of aging and generate actionable insights.

Sampled is a fully integrated analytical laboratory and biorepository, combining scalable, ISBER-compliant, and CAP-accredited biobanking with a comprehensive multiomics platform in a CLIA-certified lab spanning genomics, transcriptomics, epigenomics, proteomics, single-cell, and spatial omics.

References

1. Ahadi S, Zhou W, Schüssler-Fiorenza Rose SM et al. Personal aging markers and ageotypes revealed by deep longitudinal profiling. Nat Med 26, 83–90 (2020).

2. Shen X, Wang C, Zhou X et al. Nonlinear dynamics of multi-omics profiles during human aging. Nat Aging 4, 1619–1634 (2024).

Contact a Sampled expert today to lay strong foundations for your longitudinal aging research program.

sampled.com/gen

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How South Carolina Became the Southeast’s Fastest Growing Biotech Hub

Edusehat — Tue, 11 Aug 2026 21:10:11 +0700

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South Carolina just landed a $1.5 billion investment and more than 1,200 jobs, as Octapharma Plasma relocates its U.S. headquarters from Charlotte to Rock Hill and builds its first U.S. manufacturing facility there. Leaving an established base for somewhere newer is a bet, and Octapharma isn’t alone. It’s the latest data point in a five-year run that has landed South Carolina on GEN’s list of Next10 Emerging Biotech Hubs, ecosystems built for established manufacturers and growth-stage companies alike, not a copy of hubs elsewhere.

Manufacturers are choosing South Carolina to expand, drawn by talent, a business-friendly climate, and quality of life. In Charleston, SHL Medical’s $220 million, 300-job drug-delivery facility, producing autoinjectors and pen injectors for pharmaceutical partners worldwide, is one recent example. Suppliers are also increasing their investments. Charles River Laboratories has expanded its Charleston County operations with advanced cleanroom and automation capabilities, helping ensure biologics and vaccines reach patients faster and more safely.

In Columbia, the Ritedose Corporation, a leading Blow-Fill-Seal CDMO for generic pharmaceuticals, is on track to produce over two billion doses per year with its new 225,000-square-foot distribution center, part of an $81 million investment that has created 100 additional jobs.

The pipeline behind that scale starts at South Carolina’s research universities. Clemson University is advancing precision medicine and bioengineering through its Institute for Human Genetics, which excels at disease modeling, genomics, and drug discovery; and its Biomedical Engineering Innovation Campus, which brings together world-renowned bioengineers, Prisma Health clinicians, and innovative startups in a 31,000-square-foot facility.

As the state’s only NCI-Designated Cancer Center, the Medical University of South Carolina attracts nationally-recognized researchers and federal funding. With plans to double its research faculty and build a new facility for its cellular therapy program, the university ranks in the top 15% most innovative institutions nationwide on the CURE Innovation Index. And the University of South Carolina opened a 65,000-square-foot Brain Health Center, part of a $350 million investment bringing clinical care, imaging, and research together for patients with Alzheimer’s disease and related dementias.

That talent pipeline is also pulling in companies that didn’t start here. GNQ Insilico, a techbio company using artificial intelligence, quantum computing, and digital twin technology to predict how patients respond to treatment, moved its U.S. headquarters and lab from Silicon Valley to Greenville. “We want Greenville to know that we are building something enduring here,” said GNQ founder and CEO Rehan Huda. This is not a temporary outpost, but a long-term bet on the state’s talent, collaboration, and business climate.

People often ask why MassBio, based in the country’s most established life sciences hub, chose to partner with South Carolina at all. The answer traces back to its visionary leadership, who saw this ecosystem taking shape years before most people noticed and wanted their resources to give good science a real chance beyond Boston. That’s the thinking behind Drive, the accelerator powered by SCbio and Massbio. Drive has connected emerging life sciences companies with mentors from partners such as Eli Lilly and Company and Labcorp. Since launching in 2022, it has supported 70 early-stage companies that have raised $137 million and created nearly 100 jobs.

And it’s a pattern that holds: companies that locate here tend to expand here, and it’s exactly why South Carolina made this list. PAI Pharma, a Greenville-based leader in oral liquid medications, has invested millions in expansions since arriving decades ago; AmbioPharm has grown its Aiken County peptide operations too.

A hub doesn’t stay “emerging” forever. The window to get in early is now. Join us in South Carolina, where we’re building the life and the science that propel the future.

Why South Carolina?

www.scbio.org

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Indiana Emerges as a Leading U.S. Biopharma Hub

Edusehat — Tue, 11 Aug 2026 21:10:09 +0700

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The next generation of leading life science regions will not be defined by scientific discovery alone. Their advantage and impact will come from the ability to move promising ideas through development, manufacturing, commercialization, and delivery.

Indiana has spent decades building those capabilities, becoming a national leader in life sciences and pharmaceutical exports. Its strengths extend across universities, research institutes, manufacturers, suppliers, healthcare systems, and communities throughout the state. Together, they form a strategically connected ecosystem with the breadth, depth, and scale to support innovation at every stage and move products from the lab to patients worldwide. That proximity matters. When intellectual property timelines are ticking, speed to market is critical.

Indiana’s rising national position is driven not by one company, institution, or scientific specialty, but by the concentration and connectivity of capabilities across the state. Those strengths span human and health, animal health, and plant science, which share common functions across the value chain. In human health, Indiana has deep capabilities in large- and small-molecule discovery and manufacturing, medical devices and diagnostics, and clinical research and testing. That breadth creates partnering opportunities as ideas advance.

A discovery may begin at a university, move through clinical development and regulatory review, draw on specialized manufacturing expertise, and ultimately reach patients through global supply chains. Each capability is important on its own. Together, they create an integrated ecosystem for turning science into impact and expanding opportunity for the more than 70,000 people employed in life sciences statewide.

That collaborative model is becoming increasingly important. Scientific advances are moving faster, development pathways are becoming more complex, and companies need access to specialized talent, infrastructure, capital, and partners at every stage. Indiana is applying its established strengths to areas shaping the next generation of life sciences, including artificial intelligence and computational medicine, radiopharmaceuticals, diagnostics, precision medicine, and advanced pharmaceutical manufacturing.

Indiana also has significant momentum. In 2025, the state’s life sciences sector surpassed $100 billion in economic activity. Indiana is home to Eli Lilly and Company, the world’s highest-valued life sciences company, accounted for one in five life sciences M&A transactions during the first half of 2026.

In 2023, Indiana was designated a federal Tech Hub for biomanufacturing and received a $51 million Economic Development Administration grant, including support for a new biomanufacturing workforce training facility at 16 Tech Innovation District. Lilly Endowment awarded $70 million to establish a fund to attract leading researchers to Indiana University, Purdue University, the University of Notre Dame, and Rose-Hulman Institute of Technology. A coalition led by Indiana University also secured a National Science Foundation Engines award to accelerate innovation in musculoskeletal health and build on Indiana’s position as home to the Orthopedic Capital of the World.

The governor has announced a $1 billion, 10-year investment to grow the biosciences sector in Central Indiana. Indiana is also building on assets in radiopharmaceuticals, where global companies, specialized suppliers, and Purdue University’s leading nuclear medicine program support the development and manufacturing of innovative cancer therapies.

Despite these strengths, Indiana has not consistently told its story. BioHeartland Indiana was launched to elevate awareness of the state’s capabilities and clearly express its ambition to expand its leadership in biosciences.

What has been built here is more than a strong life sciences sector. It is an environment where ideas can find partners, companies can find the resources to grow, and products can move from discovery to patients and markets.

For investors seeking compelling inventions, researchers looking for clinical partners, and companies deciding where to expand, Indiana offers a rare combination: the capacity to invent, the infrastructure to make, and the networks to move life sciences products at scale.

To learn more, visit www.inbioheartland.com.

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BMS Chooses Houston for $2.3B Manufacturing Facility

Edusehat — Tue, 11 Aug 2026 10:25:09 +0700

Bristol Myers Squibb (BMS) has selected Houston as the site for a new $2.3 billion multi-modal manufacturing campus designed to support drug product and finished goods manufacturing from late development through launch by producing multiple modalities of treatments—including small molecules, biologics, and antibody-drug conjugates (ADCs)—across disease areas.

The campus will rise within Generation Park, a 4,300-acre master planned mixed-use campus. BMS’ planned campus is part of the pharma giant’s previously announced commitment to invest $40 billion over five years in U.S.-based R&D, technology, and manufacturing.

“This investment reflects our confidence in America’s continued leadership in biopharmaceutical innovation,” Christopher Boerner, PhD, BMS’ board chair and CEO, said in a statement. “We’re building the domestic manufacturing capabilities needed to deliver the next generation of medicines and support future scientific breakthroughs. Houston and the state of Texas offer the talent, infrastructure, and partnership needed to help bring that vision to life.”

BMS isn’t the only pharma giant to plan a multi-billion-dollar campus at Generation Park. Eli Lilly is developing a $6.5 billion manufacturing site designed to produce active pharmaceutical ingredients for oral drugs—including Foundayo® (orforglipron), the glucagon-like peptide-1 (GLP-1) receptor agonist obesity drug.

Lilly grew its regional footprint in April by acquiring Houston startup CrossBridge Bio, a developer of next-generation dual-payload antibody-drug conjugates (ADCs), for up to $300 million.

Reshoring plans

Lilly and BMS are among U.S.-based biopharma giants that have announced plans over the past year and a half to reshore significant portions of their manufacturing operations Stateside. Companies have cited a need to meet growing demand for new treatments—and an apparent desire to avoid the 100% tariff on treatments produced outside the United States, a tariff that took effect on July 31 based on Section 232 of the Trade Expansion Act of 1962, which allows investigations into whether drug imports by multinational biopharmas threaten national security.

Section 232 was amended earlier this year to allow a president to impose restrictions on goods imports or enter into negotiations with trading partners if the U.S. Secretary of Commerce determines, following an investigation, that the quantity or other circumstance of those imports “threaten[s] to impair” U.S. national security.

The planned Houston campus, BMS said, will be modular in design, enabling the company to add and reconfigure manufacturing capacity as needed in order to develop its current and future pipeline. By being modular as well as multi-modal, BMS reasons, the Houston campus will be flexible enough to evolve with the needs of the company and particularly patients into the future.

BMS plans to create nearly 500 skilled jobs at the campus, which the company says will be designed to grow in scale, capability, and workforce for decades to come. The company also projected creating about 2,000 construction and other indirect jobs between 2027–2030 as the facility is built and brought online.

Decision factors

BMS, which is headquartered in Lawrenceville, NJ, said it selected Texas and Generation Park after conducting what it termed an extensive, competitive evaluation of multiple markets in the Central and Eastern United States.

Among key factors in the decision, according to the company, were the emerging strength of Greater Houston’s life sciences workforce, proximity to utilities and transportation infrastructure, an overall business climate conducive to long-term growth—and available incentives.

“Our decision to build this state-of-the-art manufacturing campus in Houston, TX, reflects our confidence in the region’s ability to support a world-class, digitally advanced supply operation,” stated Karin Shanahan, a BMS EVP and the company’s chief supply chain and operations officer.

The state of Texas has extended a $4.89 million grant from its Texas Enterprise Fund to BMS and has designated the BMS Houston facility as a “qualified project” and thus eligible for incentives to be awarded through the Texas Jobs, Energy, Technology, and Innovation (JETI) program.

“With lower operating costs and easy access to markets across the U.S. and the world, Texas drives affordability for consumers,” added Gov. Greg Abbott (R), who is seeking re-election to a fourth term.

The region’s largest life-sci campus is the world’s largest medical complex, the 1,345-acre, 54 million-square-foot Texas Medical Center (TMC), home to The University of Texas MD Anderson Cancer Center and Baylor College of Medicine’s primary campus. The region is also home to six medical schools, three Tier One research universities, and specialized training programs through San Jacinto College’s NIBRT-licensed Center for Biotechnology, Lone Star College, and Texas A&M’s National Center for Therapeutics Manufacturing.

Greater Houston is home to some 28,000 life sciences jobs, according to regional industry group BioHouston, and has about eight million square feet of lab space. BioHouston’s chairman Jeff Wade told GEN that the region had scooped up about a half-billion dollars in VC funding between 2025 and the first half of 2026.

“Bristol Myers Squibb’s announcement is a tremendous win for Texas and the Houston region, further reinforcing our position as a premier destination for life sciences and advanced manufacturing,” stated Steve Kean, president and CEO of the Greater Houston Partnership, a regional economic development group.

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Spatial Transcriptomics Uncovers Heterogeneity in Heart Transplant Rejection

Edusehat — Tue, 11 Aug 2026 06:50:09 +0700

A team of researchers at Vanderbilt Health and the Translational Genomics Research Institute (TGen) has used image-based spatial transcriptomics to profile cellular programs involved in heart transplant rejection, an analysis the group says could help sharpen diagnosis, predict treatment response, and stratify long-term risk after transplantation.

The study, “Dynamic cellular programs of human cardiac allograft rejection revealed by spatial transcriptomics,” was published recently in Nature Cardiovascular Research. In it, the investigators applied image-based spatial transcriptomics to longitudinal human endomyocardial biopsy samples from 62 adult and pediatric heart transplant recipients during and after histologically diagnosed rejection.

Allograft rejection remains a major challenge after solid organ transplantation, with up to approximately 40% of recipients experiencing rejection within one year after transplant, and can contribute to long-term graft failure and death. In heart transplantation, clinicians routinely monitor for rejection using endomyocardial biopsies, with current diagnostic approaches relying heavily on histology. However, histologic findings do not always reflect a patient’s clinical course: “clinical presentation varies dramatically within the same grade of histologic rejection” and “response to antirejection therapy is heterogeneous, including lack of response in some patients,” the authors wrote.

“We see substantial variability in histologic rejection grades, which impacts the precision of our immunosuppressive therapies, with a potential for over- or under-immunosuppression with downstream clinical consequences,” said co-senior author Ravic Shah, MD, the Gottlieb C. Friesinger II professor of cardiovascular medicine and professor of medicine at Vanderbilt Health. “We directly examined molecular phenotypes in tissue during rejection and antirejection therapy after heart transplantation to start to understand this heterogeneity.”

The researchers analyzed longitudinal biopsy samples collected from the same patients during acute rejection and after various immunomodulatory therapies. Using spatial transcriptomics, they mapped gene expression across tissue architecture at subcellular resolution and identified 28 cell types, including immune and parenchymal cells, that differed across rejection classes. The team found broad overlap in transcriptional states across rejection severity, as well as substantial molecular heterogeneity within the same rejection grades—variation that was not apparent by histology alone.

Baseline rejection biopsies also differed between patients who responded to augmented immunomodulatory therapy and those who did not. In the paper, the authors reported that nonresponders showed “baseline T cell hyperactivation and tissue remodeling genes,” suggesting that molecular profiling could eventually help distinguish patients likely to benefit from standard approaches from those who may need more intensive or alternative therapies.

The analysis also linked cell-specific gene expression patterns to cardiac allograft vasculopathy (CAV), a chronic form of rejection that limits long-term survival after heart transplantation. “The molecular heterogeneity may help explain the spectrum of clinical presentations—from complete lack of symptoms to cardiogenic shock—for the same histologic grade of rejection,” said co-corresponding senior author Nicholas Banovich, PhD, vice president of scientific development and professor at TGen. “We expect that data generated through approaches like ours will inform early biomarker and drug discovery to meaningfully prolong transplanted organ survival.”

Together, the findings suggest that spatial transcriptomics could add clinically relevant molecular context to conventional biopsy interpretation, helping researchers better subtype rejection, predict therapeutic response, and identify patients at risk for long-term complications such as CAV.

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Bacterial Trait-Finding and Gene Mapping Platform Speeds Microbial Engineering for Biotech Applications

Edusehat — Tue, 11 Aug 2026 06:50:08 +0700

Scientists at the Department of Energy’s Oak Ridge National Laboratory (ORNL) have created a platform that can pinpoint genetic triggers that turn microbes into efficient factories for new chemicals and materials. The platform identifies specific genetic triggers for useful complex traits, supporting the design and reprogramming of microbes that exhibit targeted capabilities. Potential applications of such bacterial factories might include the breakdown and conversion of plant lignin into valuable products, or the uptake of critical minerals.

The team’s approach, which combines synthetic biology expertise, artificial intelligence, and statistical mapping techniques, enables rapid, precise reprogramming of bacteria as biotechnology tools, and builds on previous work by ORNL scientists, who adapted a technique called protoplast fusion to create the diverse microbial offspring needed for genetic mapping. “Unlike past approaches that study the effect of gaining or losing whole genes, the new approach lets us determine how small differences in the nucleotide sequence affect bacterial function,” said Josh Michener, PhD, project co-lead and Biological Systems Design group leader at ORNL. “Variations in strains at the nucleotide level have a huge impact on the resulting phenotype, especially when you’re engineering microbes with specific mutations. The method also lets us study natural mutations in parental strains that make them ideal biotechnology tools.”

Michener is co-senior and co-corresponding author of the team’s report in Nature Communications (“Genome shuffling enables quantitative trait locus mapping in Bacillus subtilis”) in which they reported on the development of their platform and its validation using gene editing in bacteria.

To determine which genes control certain characteristics in organisms, scientists have used a method called quantitative trait locus (QTL) mapping. QTL mapping involves analyzing the traits of lots of varied offspring from two distinct parents, and is a common approach in mapping the genes of other organisms such as plants. The method examines many genetic differences at once and precisely identifies candidate genes in a single workflow.

However, linking DNA sequences to observable physical traits in bacteria is challenging, the authors noted. “Even in the best-studied model bacteria, many genes have unknown functions, and little is known about the genetic networks underlying complex phenotypes or the functional effects of natural sequence variation in bacterial genes and regulatory elements.”

The problem with applying QTL mapping to bacteria is that these microorganisms reproduce asexually with limited genetic variation. “Quantitative trait locus (QTL) mapping generally relies on sexual recombination to break linkages between genes, yet bacteria rarely undergo sufficient homologous recombination to generate suitable mapping populations,” the team continued.

ORNL researchers overcame the hurdles associated with applying QTL to bacteria using protoplast fusion, a tool first developed in the 1970s. Using the fusion technique, researchers were able to cross Bacillus strains, producing a large population of genetically varied offspring, called recombinants. “We have previously shown that genome shuffling by protoplast fusion between genetically diverse Bacillus strains generates frequent, unbiased, genome-wide recombination that mimics the effects of sexual recombination,” they noted. Bacillus are model bacteria that serve as workhorses for fermentation, enzyme production, and plant growth and health. Referring to their newly published paper, the team added, “In this study, we leveraged protoplast fusion to establish a bacterial QTL mapping platform.”

Researchers measured properties of the bacteria and identified DNA variants that could explain the differences in those traits. The team tested the method across several other bacterial groups, demonstrating alternative genome shuffling methods that expand the tool’s usability on different types of microbes used as biotechnology tools. These included Clostridium thermocellum, a bacterium that tolerates industrial processes and is good at breaking down and fermenting plant cellulose. Also in Novosphingobium aromaticivorans, a bacterium that excels at breaking down aromatic compounds from plant lignin and converting the molecules into high-value chemicals. Also in Stutzerimonas stutzeri, a versatile bacterium used in applications such as bioremediation and to fix nutrients in soil, supporting plant growth and suppressing plant pathogens.

They validated their findings by using CRISPR gene editing tools to swap gene sections and confirm the effects in bacteria. “We have now, for the first time ever, put all these pieces together for a platform that gets results on complex genes-to-traits linkages much faster,” Michener said. “We built the genetically diverse bacteria population, identified DNA variants, and confirmed the work with gene editing.”

The authors added, “In contrast to traditional loss-of-function and gain-of-function genetic methods, our approach enables rapid detection of the effects of natural genetic variation in both coding and noncoding regions on bacterial phenotypes, beyond gene presence or absence.”

By creating such broad diversity in the bacterial offspring, scientists faced a challenge in the research: phenotyping all the progeny. They tackled it with automation and AI, setting up a robotic system to quickly and repeatedly place plates with precision so that high-resolution digital imaging could be accomplished at the same angle and lighting for comparable data between the recombinants. The phenotyping was accomplished 10 times faster with automation, the scientists noted.

Getting consistent data was crucial to the application of mathematical algorithms and the use of a computer vision model that processed the images and extracted traits, explained co-lead Dan Jacobson, ORNL computational systems biologist. “We built this project with a very multidisciplinary lineup,” Jacobson said. “The team did everything from building the robotics, conducting imaging and image processing, performing the statistical work, the mapping and assemblies, the genome shuffling work, growing these different isolates from the population and extracting DNA to send for sequencing, then growing them again for the phenotype assays. It’s an example of the kind of good collaboration that’s possible at a national lab, and how that research can enable whole new areas of inquiry across the nation’s science ecosystem.”

Scientists continue to deploy the method to study and engineer microbes for better manufacturing processes as part of the DOE Center for Bioenergy Innovation (CBI) at ORNL. The platform is also being used to study plant-associated microbes as part of the DOE Secure Ecosystem Engineering and Design Science Focus Area (SEED SFA), as well as by a program at Colorado State University studying airborne microbes.

The microbial QTL mapping platform is available for licensing at ORNL.

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Candida auris Persists in Hair Follicles, Hijacks Immune Signaling

Edusehat — Tue, 11 Aug 2026 03:15:08 +0700

Since its discovery in 2009, Candida auris—a multidrug-resistant pathogenic yeast—has caused deadly outbreaks around the world and is responsible for roughly 3,000 deaths in patients in hospitals and long-term care facilities per year in the U.S. The fungus is known to colonize human skin, however, the mechanisms that it uses to persist on skin remain unclear.

To understand the mechanisms C. auris uses to colonize the skin, a team of researchers compared it with Candida albicans—a common skin fungus that the immune system normally clears quickly. The team used mouse models, fungal and mouse genetics, immunology, single-cell RNA-seq, and volumetric quantitative confocal microscopy. In mice, C. albicans disappeared within days, but C. auris persisted, taking refuge in hair follicles.

This work is published in Science in the paper, “The fungal pathogen Candida auris exposes chitin to trigger IFNg and persist in hair follicles.”

“Candida auris colonizes skin way better than most other fungi, setting it up to invade once the immune system is weakened,” said Dean Merrill, MD, a dermatologist and professor at UCSF. “The big clinical problem is that we have no effective way to remove it from the skin.”

The researchers discovered not only that C. auris colonized mouse skin with higher titers and greater persistence than C. albicans—exhibiting direct hair-binding activity and a strong tropism to hair follicles—but also that the two produced very different immune responses.

More specifically, they write, “Whereas C. albicans elicited a host-protective type 3/17 skin immune response driven by interleukin-17A (IL-17A), as previously described, C. auris triggered a type 1–skewed immune response, characterized by hair follicle–associated expansion of type 1 conventional dendritic cells (cDC1), type 1 cytotoxic T cells (Tc1), and T helper 1 immune cells (Th1), as well as increased interferon-γ (IFNγ) production. IFNγ signaled directly to hair follicle keratinocytes, reducing the expression of genes and downstream programs driven by IL-17A and associated with skin barrier function and antimicrobial defense.”

The team also used mice with defects in cytokine signaling to establish that IFNγ “promoted the persistence of C. auris in the epidermal niche while maintaining its classic host-protective role during deeper skin or blood infections.”

The C. auris remodels its exterior cell wall to expose more chitin, which spurs immune cells to release interferon gamma around the hair follicle. The interferon gamma blocked the skin’s antifungal defenses, including IL-17. It also slowed the natural replacement of hair follicle cells, leading to a buildup of older, damaged cells—a niche where C. auris could flourish.

“Chitin is widespread in nature, so it’s not like the human skin never encounters it, but we were surprised to see that C. auris actively uses its chitin to turn the skin into a perfect nest,” said Suzanne Noble, MD, PhD, professor of microbiology at UCSF.

The findings reveal potential targets for preventing C. auris from persisting on the skin. One approach could involve drugs that tilt the immune system away from interferon gamma signals and toward IL-17, which drives the skin’s normal antifungal clearing process. Or perhaps drugs that block chitin could prevent the fungus from amplifying the interferon gamma signals. More broadly, the researchers say the work offers a new way to think about how microbes can quietly coexist with us before becoming pathogenic.

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BaseMap ABE Launched for Off-Target Characterization in Base Editing Therapeutic Development

Edusehat — Mon, 10 Aug 2026 23:40:09 +0700

Cambridge, U.K.-based Broken String Biosciences launched BaseMap ABE, built on the company’s INDUCE-seq^® technology, to support adenine base editing (ABE) applications.

Base editing is gaining momentum as a powerful therapeutic tool that enables precise single-base genomic changes. However, as therapeutic programs increasingly adopt ABE, researchers require reproducible and standardized methods to comprehensively characterize unintended off-target editing events, according to Terry Pizzie, CEO, Broken String Biosciences.

Terry Pizzie, CEO, Broken String Biosciences

Unlike prediction-based approaches, BaseMap ABE generates genome-wide data directly from biologically relevant cells, providing researchers with clear understanding of editor specificity under physiologically relevant conditions, he says, adding that the platform enables rapid, unbiased identification of off-target events, while generating standardized and robust data to support guide optimization, editor selection and preclinical decision making.

BaseMap ABE represents the first phase of Broken String Biosciences’ expansion into base editing applications. Through the Early Access Program, the company states that it will collaborate closely with researchers developing genome editing therapies, providing experience for early adopters and generating feedback to inform future product development.

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Early-Life Stress Leaves Epigenetic “Scars” That Prime Stress Sensitivity

Edusehat — Mon, 10 Aug 2026 20:05:09 +0700

Experiencing severe stress during childhood can make a person more vulnerable to anxiety, depression, and other mood disorders when faced with hardships as an adult. Researchers at Washington University School of Medicine (WashU Medicine) in St. Louis and Princeton University have now uncovered how trauma early in life can leave a lasting effect on the brain. Scientists already knew that stress early on in life changes the activity of genes in the brain. The team’s newly reported study in mice indicated that this is due to alterations in how brain cells package DNA, which leaves the brain’s genetic stress response vulnerable to being turned on easily and reducing tolerance to stress.

“We have uncovered a new biological process linking experience of early-life adversity to this long-term vulnerability to mental illness,” said Meaghan Creed, PhD, associate professor of anesthesiology at WashU Medicine. “This finding reveals a physical scar left by trauma experienced during development inside brain cells, providing scientists with a concrete biological target to develop new treatments and interventions.”

Creed is co-corresponding author of the researchers’ published paper in Neuron, titled “Early-life stress alters H3K4me1 in VTA to prime stress sensitivity,” in which they say that their findings “… link early-life stress experience to long-term stress hypersensitivity within the brain’s dopaminergic circuitry, providing a mechanism by which early-life stress increases risk for mood and anxiety disorders later in life.”

More than half of the world’s children are exposed to early-life stress (ELS) from abuse, household dysfunction such as violence or drug use, or other traumatic experiences. Accumulation of four or more such experiences can trigger much higher risks for long-term mental and physical health challenges in adulthood. “Early-life stress (ELS) is a risk factor for mental health and substance use disorders due to increasing sensitivity to subsequent stressors,” the authors noted.

The researchers set out to understand how trauma during early development physically changes the brain to make it more sensitive to stress later in life. They focused on a region of the brain called the ventral tegmental area (VTA), where brain cells that produce the chemical messenger dopamine are responsible for processing important things in the environment, including rewards and adversity. When these brain cells are activated abnormally, which can happen in response to stress, they disrupt how the brain processes rewards, leaving individuals vulnerable to anxiety and depression.

“The ventral tegmental area (VTA) is a key dopaminergic brain region and has been extensively implicated in the pathophysiology of mood, anxiety, and substance-use disorders,” the team explained. “Stress-induced adaptations in gene expression and cellular activity in the VTA have been causally linked to changes in motivation, reward learning, and stress response.”

Focusing on these dopamine-producing neurons, the team zoomed in on epigenetic molecular tags that direct the cell’s machinery to turn genes on and off, which affects cells’ activity. “We used a combination of bottom-up mass spectrometry, viral-mediated epigenome editing, RNA sequencing, patch-clamp electrophysiology of dopamine neurons, and behavioral quantification in a mouse model of early-life stress, focusing on the ventral tegmental area (VTA), a key dopaminergic brain region,” they stated.

Inside cells, DNA is coiled like a slinky, explained senior and co-corresponding author Catherine Jensen Peña, PhD, an assistant professor at the Princeton Neuroscience Institute. The DNA coils are wrapped around histone proteins that help determine how tightly or loosely the coil is wound. When this genetic slinky is compressed, its genes are turned off, but as the DNA coil stretches and opens, the genes are more easily accessible to be turned on.

The researchers found that an enzyme called SETD7 was more abundant in the dopamine neurons of young mice that had experienced stress, compared with its abundance in mice reared in a typical environment. SETD7 helps place a chemical tag, H3K4me1, on the genetic slinky, marking the structure for uncoiling, which in turn makes the cell more reactive to everything going on in the environment, explained Peña.

The researchers then artificially boosted levels of SETD7 in young, stress-free mice. Even without early-life stress, these mice grew up with a stretched-open DNA structure in their dopamine-producing brain cells, making it easier to turn on the genes that respond to stress. The animals had a lower tolerance for stress in adulthood. The researchers found that, as adults, the mice that had boosted SETD7 levels when they were young had more reactive dopamine neurons and exhibited more anxious behavior compared to mice with normal levels of SETD7 throughout their lives.

Conversely, when the researchers blocked the SETD7 enzyme from adding too much of the H3K4me1 tag after early-life stress, the slinky remained closed, shielding mice from becoming hypersensitive to stress later in life. Despite experiencing both early-life and adult stress, mice with their SETD7 levels dampened were able to remain as social and exploratory as unstressed mice, and their dopamine neurons were active at normal levels.

In their paper, the team wrote in summary, “Mimicking early-life stress through postnatal overexpression of Setd7 and enrichment of H3K4me1 in the VTA sensitizes transcriptional, physiological, and behavioral responses to adult stress, while Setd7 knockdown ameliorates the impact of early-life stress.”

“There are currently no treatments for what early-life stress does to the brain, partially because we have not had a clear picture of what molecular mechanisms to target,” Peña said. “This work is exciting because it reveals a clear mechanism, and also helps explain why the impact of stress is both latent and broad. Additionally, if we can step in with supportive care, therapy, or social resources to buffer children during those sensitive windows of development, we may be able to protect the epigenome—preventing the genetic slinky from locking into an open position and perhaps giving the developing brain a chance to build natural resilience.”

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StockWatch: Lilly Rises on Revenue Leap; Analysts High on Krystal Despite Revenue Miss

Edusehat — Mon, 10 Aug 2026 16:30:08 +0700

Like Ol’ Man River of song, Eli Lilly (NYSE: LLY) keeps rollin’ along thanks to its ongoing windfall of cash from tirzepatide, which is marketed for obesity as Zepbound® and for adult type 2 diabetes as Mounjaro^®.

That windfall for tirzepatide, a dual agonist of glucagon-like peptide-1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP), continued when the pharma giant reported second-quarter revenue and other results that beat analyst forecasts.

Lilly shares jumped 6.9% this past week, from $1,115.68 on August 4 to $1,192.28 on Thursday, before profit-taking trimmed the share price to $1,185.71 at Friday’s closing bell and a 6.3% one-week gain. The mini-surge followed Lilly releasing powerhouse Q2 earnings results in which it raised its investor guidance for 2026 GAAP revenue.

Lilly now expects to rack up between $85 billion and $87 billion in revenue this year, up 2.4% to 3.7% from its previous range of between $82 billion and $85 billion. That change raised the midpoint of Lilly’s revenue forecast from $85.5 billion to $86 billion.

However, Lilly also lowered its high-end EPS guidance by 1.4% from $37 to $36.50 per share, though the company kept the low end of its forecast flat at $35.50. Lilly blamed the guidance dip on the $2.8 billion ($3.03 per share) in acquired in-process research and development (IPR&D) charges it incurred from business development activity—primarily related to Lilly’s $2.4 billion acquisition of Orna Therapeutics, a circular RNA therapy developer, and up-to-$2.3 billion buyout of JAK2 inhibitor developer Ajax Therapeutics, both completed during Q2.

Lilly finished the second quarter with $7.095 billion in net income, up 25% from $5.661 billion in Q2 2025, on revenue that leaped 48%, to $22.974 from $15.558. EPS grew 26% year-over-year to $7.94 from $6.29. Lilly’s EPS was 25% above, and its revenue 11% above, consensus forecasts cited by Leerink Partners.

Exceeding expectations

“LLY significantly exceeded 2Q consensus expectations, and the income statement shows the tremendous operating leverage as sales upside drives much higher margins and profits,” David Risinger, a senior managing director and senior research analyst covering diversified biopharmaceuticals at Leerink Partners, commented in a research note.

Risinger raised Leerink’s 12-month price target on Lilly shares 6%, from $1,232 to $1,309. Both numbers are 25 times Lilly’s estimated adjusted earnings per share for 2027, which Leerink now predicts will rise from $49.28 to $52.34. Similarly, Lilly raised its EPS forecast for 2026 by 7%, from $33.87 to $36.20.

Lilly’s above-forecast revenue, according to Risinger, was largely driven by sales of adult type 2 diabetes drug Mounjaro outside the United States ($5.162 billion), which grew about 14% above consensus forecasts, as well as U.S. sales of Mounjaro ($4.791 billion), which rose about 10% above consensus. Overall, Mounjaro quarterly revenues nearly doubled, catapulting 91% to $9.943 billion from $5.199 billion in Q2 2025.

Another GLP-1/GIP dual inhibitor proved to be a growth driver for Lilly during Q2—Zepbound, the obesity drug whose revenues vaulted 46% to $4.928 billion from $3.381 billion in April–June of last year.

Again in Q2, Lilly outperformed the GLP-1 inhibitor blockbuster drugs of Novo Nordisk (shares traded on Nasdaq Copenhagen as NOVO-B; American depositary receipts or ADRs traded on NYSE as NVO), which reported quarterly results on Tuesday.

Novo Nordisk’s adult type 2 diabetes drug Ozempic® garnered DKK 31.375 billion ($4.851 billion), up 5% year over year, while its injectable Wegovy® obesity/weight control drug generated sales of DKK 19.484 billion ($3.013 billion), up just 1% from Q2 2025. The company also made DKK 3.218 billion (about $498 million) in sales from oral Wegovy, which reached the U.S. market on January 5.

Lilly’s competing oral obesity drug Foundayo® (orforglipron), a small molecule GLP-1 receptor agonist, generated $98 million in its first quarter on the market, having won FDA approval on April 1. That’s about $3 million below consensus forecasts cited by Jefferies.

“LLY continues to be one of our top picks in LC pharma,” Jefferies equity analyst Akash Tewari wrote in a research note, referring to “large cap” pharmas with $10 billion or more of market capitalization (share price times the number of outstanding shares). “While Foundayo’s initial U.S. launch has been muted, we think investor focus will shift toward whether LLY’s GLP-1/obesity franchise as a whole (including Foundayo) can beat expectations in ’26.”

Tewari offered two other arguments for finding Lilly a top pick among the largest biopharmas: The huge potential for obesity drug sales outside the U.S. and through the Medicare GLP-1 Bridge Program, a temporary program ending December 31, 2027, that includes Zepbound (KwikPen® pre-filled injection version), Foundayo, and Novo Nordisk’s Wegovy. Medicare GLP-1 Bridge Program participants pay a fixed $50 monthly copayment.

How much higher can Lilly climb? The sky’s the limit, chairman and CEO David A. Ricks suggested in a statement. He cited Lilly’s Phase III next-generation obesity blockbuster retatrutide, a triple hormone receptor agonist targeting glucagon receptors as well as GLP-1 and GIP; as well as Lilly’s ongoing U.S. manufacturing expansion totaling $55 billion in projects since 2020.

Driving those projects is the company’s need for more manufacturing—and a desire, as with most biopharma giants, to avoid tariffs levied by the administration of Donald J. Trump on imports of drugs produced outside the United States.

“Lilly is building for the future,” Ricks declared. “With our next-generation weight-loss medicine retatrutide and its complete clinical data package in hand, new manufacturing capacity coming online, and exciting new assets entering our pipeline through business development, Lilly’s future, after 150 years, has never been brighter.”

Krystal shines for analysts

Krystal Biotech (Nasdaq: KRYS) shares endured an 11% one-week slump after the genetic medicine developer reported second quarter results that included less revenue than market watchers expected for Vyjuvek^® (beremagene geperpavec-svdt or B-VEC), the company’s marketed gene therapy for the rare skin disorder dystrophic epidermolysis bullosa or DEB.

Vyjuvek finished Q2 with $119.222 million in net product revenue, 0.7% to 1.4% below consensus analyst forecasts that projected between $120.1 million and $120.9 million in quarterly revenue—though 24% above the $96.042 million reported for the year-ago quarter.

Despite the slight revenue miss vs. forecasts, Krystal enjoyed positive feedback from analysts that stemmed the stock slide enough for Krystal shares to plateau the rest of the week.

They began by attributing the lower-than-expected revenue to the fact that Q2 was the first full quarter of price accruals for Vyjuvek in Germany—the recording of expenses that Krystal has incurred but not yet been paid for, since the gene therapy is the subject of pricing and reimbursement talks with German officials. That helped flatten European sales to $19.3 million, compared with $91.6 million in the United States (the remaining $8.3 million in sales was generated in Japan).

However, analysts noted, patient vial growth in Europe and Japan was at double-digit levels with more than 180 patients treated outside the United States, Krystal said.

In the United States, Krystal secured over 730 Vyjuvek-related reimbursement approvals as of Q2, up 35 from the first quarter. As a result, penetration of the gene therapy had risen to more than 60% of patients diagnosed with DEB.

Over the past year, Vyjuvek has added 35–40 U.S. patients quarter over quarter, Jefferies equity analyst Roger Song, MD, wrote in a research note.

“Demand remains robust”

“Despite a modest rev[enue] miss driven primarily by German pricing accrual dynamics, underlying Vyjuvek demand remains robust across U.S. and ex-U.S.,” Song wrote. “Ex-U.S. launches are tracking favorably despite expected reimbursement-related volatility, [while] expansion of global footprint carries on.”

Sami Corwin, PhD, a biotechnology-focused healthcare analyst with William Blair, observed in a research note that in the United States, Vyjuvek has attracted more than 640 unique prescribers since its launch in 2023, with 70 new prescribers in the second quarter alone—progress that she said highlighted the impact of Krystal’s expanded sales force.

As for Germany, Corwin reported Krystal’s expectation that pricing talks will conclude by year’s end, to be followed in the second half of 2027 by the conclusion of pricing talks with officials in France. Similar negotiations are in progress in Spain and Italy, with commercial launches also expected there by the end of 2026.

“We continue to believe that the ex-U.S. launches will be key near-term drivers of Vyjuvek revenue growth in 2026, and the continued positive patient experience will contribute to a strong revenue tail,” Corwin wrote.

Krystal’s stock drop began with profit-taking by investors that sent Krystal’s shares sliding 7% from $366.85 to $341.12 on July 31. After dropping another 8% to $312.57 on August 3, Krystal shares bounced back 3% to $322.69 Thursday and finished the week rising another 1%, closing at $326.45 on Friday.

Leaders and laggards

Emergent BioSolutions (NYSE: EBS) shares tumbled nearly 30% Thursday from $7.54 to $5.31 after the developer of medical countermeasures (MCMs) announced a restructuring that included eliminating approximately 93 positions—about 10% of Emergent’s approximately 900-person workforce as of December 31, 2025—as well as eliminating about 21 vacant positions and shutting down its wet labs in Gaithersburg, MD, where the company is based. The restructuring is projected to achieve annualized savings of approximately $40 million. Emergent finished the second quarter with a net loss of $180.2 million, 1,402% worse than the year-ago quarter, largely due to a $191.3 million non-cash impairment charge related to Narcan® Nasal Spray assets. Narcan is among Naloxone products whose revenues fell $15.1 million, or 22%, compared with Q2 2025, as over-the-counter Narcan saw decreases in U.S. sales volumes. Revenue zoomed 66% from Q2 2025 to $234.3 million, primarily due to a 150% year-over-year jump in smallpox MCM revenue, from $40.6 million to $101.6 million.
Iovance Biotherapeutics (Nasdaq: IOVA) shares surged 43% from $4.34 to $6.21 Thursday after the developer of polyclonal tumor-infiltrating lymphocyte (TIL) cancer therapies said it will review its revenue guidance to investors after reporting second quarter results that included a record for quarterly revenue of $99.313 million. Nearly all of that revenue consisted of the ~$91 million generated by Iovance’s tumor-derived autologous T cell immunotherapy Amtagvi® (lifileucel), the company’s sole marketed product. Amtagvi revenue jumped 40% year-over-year while total revenue leaped ~60% from $59.952 million in Q2 2025. Amtagvi is indicated for adults with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. Iovance previously guided investors to 2026 revenue of between $350 million and $370 million.

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Novel Molecular Glue Discovery Platform Unlocks Undruggable Cancer Targets

Edusehat — Sat, 08 Aug 2026 03:00:09 +0700

Targeted protein degradation is a therapeutic strategy that leverages the ubiquitin–proteasome system to eliminate disease-associated proteins—including those that have traditionally been considered undruggable.

Protein degraders remove unwanted proteins from inside a cell by disposing of them using the cell’s built-in recycling system. Specifically, a molecular glue degrader binds an E3 ligase and redirects it to tag a disease-related protein for disposal.

Now, investigators at Dana-Farber Cancer Institute have developed a platform for systematically discovering molecular glues that could become protein degradation drug candidates. The platform could help drug developers expand the range of disease-related proteins that can be therapeutically targeted for elimination via protein degradation. The platform also enabled their discovery of the first molecular glue degrader that is metabolically activated, suggesting that molecular glues could be more context dependent and potentially tunable than previously thought.

The study was published in Nature in the paper, “DCAF11-dependent molecular glue degrader activated by glutathionylation.”

“This novel platform is an exciting scalable approach to the discovery of molecular glues that could help drive the significant expansion of molecular glue applications for the treatment of cancer and other diseases,” says Eric Fischer, PhD, professor of Biological Chemistry and Molecular Pharmacology at Harvard Medical School.

In 2014, Benjamin Ebert, MD, PhD, president and CEO of Dana-Farber, found the mechanism of action behind the multiple myeloma drug lenalidomide to be a molecular glue degrader of a transcription factor. Because transcription factors tend to have few pockets for inhibitor drugs to bind to, they were thought to be “undruggable.” Degrading transcription factors opened a new way of thinking about the treatment of cancer.

Today, several protein degraders have entered clinical testing. However, these degraders only leverage a small handful of the 600 E3 ligases in the human genome.

“There is an incredible range of opportunity for discovering new molecular glue degraders,” says Ebert. “This systematic approach could help accelerate the discovery of novel degraders that could change the way we think about the treatment of cancer.”

The screen in the new research fixes a subset of E3 ligases to magnetic beads in a well and bathes them in cellular lysate and a library of drug compounds. A hit occurs when a drug binds to one of the E3 ligases and increases its affinity for a given protein. The team used mass spectrometry to determine which cellular proteins have affinity for the drug-bound E3 ligase and would be likely to be tagged for disposal inside a cell.

They tested the system by screening seven E3 ligases and found the DDX18 protein was drawn to the E3 ligase DCAF11 and that the compound M12 enabled the connection. Using cryo-EM, the team found that M12 had been altered by glutathionylation; it would only act as a molecular glue inside cells with elevated levels of metabolites related to oxidative stress in the cell—something common in cancer cells.

“This was a huge surprise, and it is the first observation of a molecular glue that has been activated metabolically by glutathionylation,” says Franziska Wachter, MD, pediatric oncologist and instructor in pediatrics at Dana-Farber Cancer Institute.

Further exploration of activated M12 revealed that it functions as a prodrug that is activated through glutathione S-transferase-mediated glutathionylation and reprograms the E3 ligase DCAF11 to degrade DDX18. More specifically, the authors write that it “the glutathione moiety binds to an evolutionary conserved glutathione-binding site on DCAF11, and the exposed M12 moiety facilitates neo-substrate recruitment.”

By binding additional proteins to the complex, the team was able to tune the system to degrade multiple other proteins, including cancer-related protein targets such as SMARCA2, WEE1 and CDK7. “This systematic approach to discovering novel molecular glue degraders opens up the possibility for expanding the number of proteins that can be targeted for degradation as a treatment for cancer,” says Ebert.

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Senators seek solutions for AMR, maintaining US biotech leadership

Edusehat — Fri, 07 Aug 2026 23:20:02 +0700

There was bipartisan support for the PASTEUR Act’s approach to addressing AMR and for NSCEB recommendations, both BIO priorities.

A bipartisan Senate hearing sought strategic approaches to two related challenges facing U.S. security—maintaining American biotech leadership and tackling antimicrobial resistance (AMR).

Lawmakers from both parties in the Aug. 4 Senate Finance Health Subcommittee hearing agreed on the need to support American innovation in developing not only antimicrobials, but all types of medicine.

“China’s rise in biotechnology and the growing risks of antimicrobial resistance present new challenges to the United States,” said Subcommittee Chair Todd Young (R-IN).

Chair Young, who noted 70,000 Indianans are employed in biotech, has worked on solutions to both challenges. In June, he, Sen. Michael Bennet (D-CO), and others reintroduced the Senate version of the PASTEUR Act, which would encourage development of new antimicrobials. In an effort to preserve American biotech leadership, Chair Young oversaw the National Security Commission on Emerging Biotechnology (NSCEB) report.

Additional approaches to these challenges were also discussed in the hearing.

Support for AMR research and innovation

Senators analyzed the threat of AMR, the market challenges to addressing AMR, and some policy solutions.

Witness Kevin Outterson, whose organization CARB-X leads a public-private partnership to fund new antimicrobials, explained the importance of antibiotics. He said soldiers who are wounded, mothers having C-sections, and older adults getting a knee replacement “depend on these amazing drugs as a safety net to prevent the infection.” But persistent use of antimicrobials lets pathogens develop resistance, creating a public health concern.

“AMR is linked to 170,000 deaths annually, and infections contribute to half of cancer deaths and are the second leading cause of maternal mortality,” in the U.S., explained witness Vera Luther, MD, an infectious disease expert at Dartmouth Hitchcock Medical Center. “Healthcare costs from the six biggest AMR threats are more than $4.6 billion annually.”

The need to use antimicrobials judiciously makes it nearly impossible for the innovative biotech developing these drugs to recoup their investment. Of the last 11 small biotechs gaining Food and Drug Administration (FDA) approval for novel antimicrobials, two approvals are new and the other nine “have either gone into bankruptcy or the economic equivalent. Their R&D investors are completely wiped out,” Outterson said.

The proposed PASTEUR Act was supported as a solution by Outterson and Luther. Lawmakers expressed interest in its concepts, and several asked pointed questions at the hearing, including Sens. Catherine Cortez Masto (D-NV), Bill Cassidy, MD (R-LA), Roger Marshall (R-KS), and Subcommittee Ranking Member Maggie Hassan (D-NH). Sen. Bennet provided a statement for the record expressing support for the PASTEUR Act at the hearing.

Instead of paying by volume, PASTEUR would use a contracting model that provides predictable support for qualifying new antimicrobials developed, ensuring we have effective treatments against the most threatening infections when they arise.

Government incentives for new AMR treatments would save lives, and money, Outterson said. “This would be a remarkably great value for the U.S. taxpayers,” he maintained. “A Center for Global Development study found a 28-to-1 return.”

The Biotechnology Innovation Organization (BIO) also supports the PASTEUR Act. “Updated PASTEUR legislation would help to repair the broken marketplace for antimicrobials by enabling a business model centered around the appropriate use of new and novel treatments,” said BIO President & CEO John F. Crowley, when the bill was reintroduced in Congress earlier this year.

BIO has called attention to patient challenges with AMR, including by amplifying the story of patient advocate Katy Grainger in “The Fight of Our Lives.”

Supporting small biotechs

Suggestions for supporting small biotechs came from several lawmakers, including Ranking Member Hassan, who mentioned developing legislation to give tax incentives for biotechs working on new antibiotics.

Sen. Cassidy urged supporting small innovative biotechs in general, and said the drug price controls established by the Inflation Reduction Act (IRA) is a drag on these companies.

“For small biotechs right now, their biggest challenge is raising critical capital,” he explained. “The IRA, which is going to begin to include small biotechs in 2028, is a real hindrance to raising capital now, they tell me, because the drugs for which they’re currently raising money may be subject to the IRA.”

Cassidy has proposed the Small Biotech Innovation Act to allow small biotechs that are investing heavily in research to claim some exemptions from IRA price controls.

The threat from China

BIO supports many recommendations of the NSCEB report on maintaining U.S. biotech leadership. As Chair Young told the hearing, the report outlines the threat posed by China’s concerted strategy to replace America by developing and manufacturing most of the world’s medicine.

One of the witnesses in the hearing, Michelle Rozo, Vice Chair of the NSCEB, described why that matters.

“America’s growing dependency on Chinese biopharmaceutical supply chains is a strategic vulnerability,” she testified. “If China has already restricted access to critical minerals, why would we assume it would continue supplying life-saving medicines during a conflict?”

The NSCEB report Rozo and Chair Young oversaw includes 49 recommendations to maintain biotech superiority, such as creation of a federal office to coordinate the work of diverse agencies and further spending by Congress.

While some improvements are under consideration by Congress and the administration, Rozo said we need to implement more—soon. Responding to a question from Sen. Cortez Masto, Rozo confirmed China has apparently begun implementing some NSCEB recommendations in their own country to compete with the U.S.

Subcommittee Ranking Member Hassan was one of several senators who appeared to appreciate the urgency.

“If the United States does not keep up with the science and innovation coming from countries like China, we risk falling behind and becoming reliant on critical new medications that are only available abroad,” she told the hearing.

Several Senators, including Sens. Roger Marshall, MD (R-KS), James Lankford (R-OK), and Mark Warner (D-VA), asked about challenges of onshoring, particularly as China dominates manufacture of the key starting materials underpinning much of drug production. Outterson, Luther, and Rozo said government incentives can drive innovation to potentially enable safe, low-cost production in the U.S.

Sen. Ron Wyden (D-OR) was one of several lawmakers praising the bipartisan agreement at the hearing. He said he hoped that next year “we’re working on these issues and that we have the same kind of coalition that you’re seeing here.”

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Algae-Based Food Product Development Earns Gates Foundation Award

Edusehat — Fri, 07 Aug 2026 12:20:10 +0700

The Gates Foundation awarded a $500,000 Grand Challenges grant to a University of Connecticut (UConn) researcher specializing in the development and commercialization of sustainable food products using algae and other natural materials.

A photo of the novel strain of the microalgae being developed by Mingyu Qiao, PhD, at 400x magnification under a microscope. [UConn]

The title of the project proposed by Mingyu Qiao, PhD, assistant professor of innovation and entrepreneurship in the College of Agriculture, Health and Natural Resources (CAHNR), Department of Nutritional Sciences, was “Ultra Low-Cost DHA Powder from Algae Grown on Digested Grain Spent.” Qiao’s lab specializes in using eco-friendly biomanufacturing and bioprocessing technologies to create scalable, cost-effective ways to produce nutritious, safe, and affordable food and nutrients.

Many view Qiao as an innovator in the eco-manufacturing of nutrients using microalgae. At UConn, he led a multidisciplinary team that won a $500,000 National Science Foundation Future Manufacturing Grant to develop a novel biomanufacturing technology using microalgae to produce essential amino acids for poultry feed.

The latest project is a continuation of that research, though the end product is different. This one focuses on docosahexaenoic acid (DHA), an essential omega-3 fatty acid critical to fetal and infant brain and eye development, which will be produced from a novel strain of microalgae recently isolated in Qiao’s lab.

Brewer’s Yeast (Saccharomyces cerevisiae), under a microscope. [Artur Plawgo/Getty Images]

Using spent brewer waste to cultivate the microalgae offers a means of producing supplemental DHA for humans at a significantly lower cost than existing production methods. In addition, this method can easily be deployed in low- and middle-income countries (LMICs) in Africa and elsewhere to produce the nutrients locally and create jobs.

Qiao’s proposal details the potential impact of the project. While global health authorities recommend at least 200 mg of DHA for pregnant and lactating women, DHA deficiency remains widespread in LMICs around the world. Current pharmaceutical-grade algal DHA powders cost more than $52 per kilogram, due largely to the expense of producing DHA-rich microalgae using refined sugars, nutrients, and capital-intensive fermentation and downstream processing technologies.

That requirement alone accounts for up to 85% of total cost of production and limits the feasibility of manufacturing DHA in less wealthy countries without an established biomanufacturing infrastructure and workforce. Spent brewery waste is an abundant, underutilized resource in LMICs. Global production of the nutrient-rich byproduct of the brewing process exceeds 40 million tons annually, making it widely accessible.

Qiao’s co-PIs on the project include Jeffrey McCutcheon, PhD, a UConn professor of chemical and biomolecular engineering; Qing Jin, PhD, an assistant professor of food science at the University of Maine; and Bo Wang, PhD, an assistant professor of bioproducts and system engineering at the University of Minnesota Twin Cities.

The award covers the initial 18-month development phase of the project, Qiao said. If successful, the project could be eligible for additional funding to establish manufacturing sites in Africa and beyond.

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FMT Shows Promise for Peanut Allergy Tolerance in Phase I Trial

Edusehat — Fri, 07 Aug 2026 12:20:09 +0700

For people with severe food allergies, avoiding a trigger food like peanuts or tree nuts can mean constant vigilance. Approximately 8% of children and 10.8% of adults in the United States experience some type of food allergy. Even trace exposures can provoke reactions, and while oral immunotherapy can raise the threshold for some patients, the protection often depends on continued treatment and is associated with disease relapse in many patients. A new study suggests that another route to food tolerance may run through the gut microbiome.

In a small Phase I open-label trial (NCT02960074), researchers at Boston Children’s Hospital tested whether oral encapsulated fecal microbiome transplantation (FMT) could safely increase peanut tolerance in adults with peanut allergy. The work, published in Science Translational Medicine, also used mouse models to probe how donor gut microbes might help restore oral tolerance. The study is titled, “Fecal microbiome transplant in food allergy in humans and mice identifies a role for bile acid metabolites in oral tolerance.”

The trial enrolled 15 adults who reacted to 100 mg or less of peanut protein at baseline, less than half a peanut. Ten participants received a one-time dose of 36 frozen FMT capsules without antibiotic pretreatment; three of these participants showed an increased peanut reactivity threshold after treatment. A second cohort of five participants received antibiotics before FMT, and three of those participants also showed increased tolerance without safety issues, the authors wrote. Across the full study, six of 15 participants met the secondary efficacy endpoint, with responses persisting through the four-month clinical endpoint. No FMT-related allergic reactions or grade 3 or higher adverse events were reported.

“This landmark study was the first to demonstrate that a microbiome-based therapy may improve food allergy in people while also revealing how gut bacteria, their metabolites, and the immune system work together to influence treatment response,” said Rima Rachid, MD, director of the Food Allergy Program and the Allergen Immunotherapy Program at Boston Children’s Hospital.

The mechanistic findings pointed to a possible explanation. In participants who responded to FMT, the researchers observed an increase in tolerogenic RORγt-positive regulatory T cells and a decrease in type 2 helper T cells, immune shifts consistent with restored oral tolerance. When the team transferred post-FMT microbiomes from responders into allergy-prone mice, those mice were protected from the food allergy, whereas mice receiving microbiomes from nonresponders were not.

The responder-associated protection was linked to increased colonization with members of the gut Bacteroides genus and higher levels of bile acid metabolites in both humans and mice. The researchers further showed that deleting a bile salt hydrolase gene from a candidate protective Bacteroides strain weakened food allergy suppression in mice, suggesting that bacterial bile acid metabolism contributes to the tolerogenic effect.

“Food allergy reflects a failure of oral tolerance, the process by which the gut immune system learns to accept food, and what this study shows is that the right bacteria can help restore that process, working through bile acid metabolites to promote the immune cells that enforce tolerance,” said Talal Chatila, MD, director of translational immunology at Boston Children’s Hospital. “Knowing how the bacteria restore tolerance to food in allergic individuals allows us to optimize the therapy for more effective outcomes.”

The authors cautioned that the trial was small, open-label, and limited to adults, and that “of the six responders, five were males as opposed to two of the nine nonresponders, suggesting a potential male sex bias in response to therapy.”

“Larger studies of fecal and microbiota transplantation are now essential to confirm these findings, identify the patients most likely to benefit, and discover beneficial bacteria that could be developed into targeted probiotic therapies for food allergy,” added Rachid. Rachid is now leading follow-up studies testing a purified, concentrated microbial formulation in teenagers and in combination with peanut oral immunotherapy.

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Tau and Alzheimer’s Disease

Edusehat — Fri, 07 Aug 2026 08:40:11 +0700

Alzheimer’s

Disease eBook cover” class=”wp-image-336117″/>

Toward this end, at Nautilus we’re proud that researchers are using the Nautilus Voyager Platform and its underlying Iterative Mapping methodology to explore AD and related dementias at an unprecedented level of depth. They’re deploying the Tau Proteoforms Assay to quantify tau variants or proteoforms (the versions of proteins found in biological systems) with singlemolecule resolution and discovering how changes in the previously hidden proteoform landscape may be implicated in disease. In this edition of the Nautilus Biotechnology “Proteomics and neuroscience” eBook, we’re excited to:

Dive into tau biology

Share what researchers are learning about tau and its role in AD using the Nautilus Voyager Platform

Invite you to use the Nautilus Voyager Platform in your own work through the Iterative Mapping Early Access Program – the first offering available through this program is the Tau Proteoforms Assay, and we’ll be adding new offerings including broadscale proteomics and additional proteoforms soon.

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Engineered Human Interneuron Transplants Repair Respiratory Circuits in Injured Rats

Edusehat — Fri, 07 Aug 2026 08:40:10 +0700

About 15 to 20 million people globally are impacted by spinal cord injuries, which can impair movement, limit their independence, and disrupt important bodily functions. For example, damage to the spinal cord that occurs at the neck disrupts signals that control the diaphragm, the main muscle used in breathing. The body does not naturally rebuild lost neural connections and there are no approved therapies that can regenerate the neurons and connections affected by a spinal cord injury. But that could change thanks to new research from scientists at Gladstone Institutes.

Full details of the work, which was done in rats, are published in Science Translational Medicine in a new paper “Human spinal interneurons repair the injured rat spinal cord through synaptic integration.” It shows that human stem cell-derived spinal interconnected neurons or interneurons—critical cells for breathing and movement—can survive following transplantation in injured rats, form connections with the receiving animals’ neural circuits, and improve breathing-related motor function. As Lana Zholudeva, PhD, a Gladstone investigator and the paper’s first author, puts it, “this study demonstrates that a specific type of human spinal interneuron can be engineered from stem cells and transplanted into an injured spinal cord” in such a way that “the cells not only survive, but form new pathways to repair damaged networks.”

For the study, the scientists focused on a subtype of the interneurons called V2a interneurons. These are relay cells that play a role in controlling movement. Previous research by Zholudeva’s team and others have shown that these cells are implicated in recovery after traumatic spinal cord injury, including in the neural circuits involved in breathing and walking.

Using human induced pluripotent stem cells, Zholudeva and her team generated transplantable human V2a interneurons that were optimized for repairing injured spinal circuits. Specifically, “we engineered human V2a-enriched SpINs from an optogenetic channelrhodopsin-2 (ChR2) expressing the human induced pluripotent stem cell line,” they wrote in the paper. Getting the process right took some doing, according to Deepak Srivastava, MD, Gladstone president and senior author of the study “it took about a year and a half of trial and error to get the recipe right to make this particular neuron out of stem cells, but it really paid off.” They also ensured that cells could be frozen in vials and later thawed for use, making it possible to use them in human clinical trials down the road.

Next, the scientists transplanted the interneurons into adult rats one week after they sustained injuries to their cervical spinal cords. Two months post transplantation, the scientists found that the new cells not only survived the hostile environment of the injury site but also formed connections with nearby cells. Furthermore, when the scientists activated the transplant site, they observed increased activity in the diaphragm. They also activated the rats’ own brainstem neurons and found that the transplanted cells switched on in response.

The scientists also tested the rats’ breathing under different conditions. Under normal conditions, the difference in the animals’ breathing was less noticeable. But in a low oxygen or high carbon dioxide environment, most of the injured, untreated controls showed signs of respiratory failure. In contrast, most of the rats that received the new V2a interneurons passed the challenges without difficulty. “The transplanted cells seem to be providing that additional capacity,” Zholudeva said.

One component of the study involved looking at why some transplants worked better than others. The scientists identified a specific subset of transplanted V2a interneurons that seemed especially likely to connect with the host animal’s breathing circuit. They plan to follow up on the finding as part of their next steps. Further down the road, they plan to test the potential therapy in larger animals. And they will evaluate whether it is as effective in the injured spinal cord months or years after injury, not just in the immediate aftermath.

The team also hopes to test the treatment in other neural circuits. Specifically, they are considering circuits that control arm and hand function, something that people with cervical spinal cord injuries often identify as their highest priority for recovery. “We’ve shown a proof of principle that this can work, that you can engineer a defined cell type, transplant it, and have it actually repair a specific circuit,” Zholudeva said. “Now we have to make it work more consistently, in more circuits, and eventually in people.”

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Genetic Findings Provide Insights Into Leading Cause of Back Pain

Edusehat — Fri, 07 Aug 2026 08:40:09 +0700

Neck and back pain could be caused by changes in gene activity that trigger the breakdown of the spine’s natural shock absorbers, according to researchers in the U.K. Findings from a study in zebrafish, titled “Targeted modulation of phosphate and lipid metabolism reduces ligament mineralization in col9a1b deficient zebrafish,” and published in Communications Biology, suggest that changes in gene activity can lead to a build-up of minerals in the spine—similar to unwanted bone forming in the wrong place—causing it to harden.

Experts say the findings point to potential future drug targets to treat back pain and suggest zebrafish could be a valuable tool for testing them.

Back pain affects most people at some point in their lives. One of the main underlying causes is the gradual breakdown of spinal discs (which cushion the bones of the spine), known as intervertebral disc degeneration (IVDD).

Despite how common and costly IVDD is, there are currently no drugs that can stop or reverse the condition. Surgery remains the only long-term option.

Genetics are known to play a role in the development of IVDD. A gene connected to a protein called collagen IX, which helps hold the disc’s structural fibers together, has been repeatedly linked to early-onset disc problems.

The findings from a recent zebrafish study published in Communications Biology point to potential future drug targets to treat back pain and suggest the fish could be a valuable tool for testing them. [Connect Images/Matt Lincoln/Getty Images]

Scientists from the Universities of Edinburgh and Bristol studied zebrafish that were bred to lack a working copy of the gene to better understand how genetic faults could lead to disc disease. As the fish aged, their spines developed problems strikingly similar to human disc disease. The bones of the spine fused together, and the tissue between vertebrae became abnormally hardened with mineral deposits.

The team found that this hardening was preceded by a breakdown in a supportive scaffold layer in the developing spine, well before any mineral began to build up.

Researchers looked at which genes were switched on or off in the fish. They uncovered disruptions to how the body handles fat and to a growth-control pathway called mTOR, alongside changes in phosphate handling and vitamin A signaling, all processes linked to mineral buildup.

Erika Kague, PhD, University of Edinburgh Institute of Genetics and Cancer

The team was also able to demonstrate ways to reduce the damage. The bone-protecting drug bisphosphonate, which is already used for osteoporosis, blocked the mineral buildup. Simply restricting the fish’s food intake, or using drugs that dampen fat metabolism, also reduced spinal fusions.

The findings point to phosphate handling and fat metabolism as promising targets for future drugs, according to the research team.

“For decades, surgery has been the only real answer for disc disease. By understanding the biology that drives the spine to harden, our zebrafish studies point to several ways of slowing it down, including a drug already used safely in patients,” said Erika Kague, PhD, study lead from the University of Edinburgh’s Institute of Genetics and Cancer. “There’s more work to do, but for a condition that’s affected people for generations without a treatment in sight, this is super exciting.”

“For the 9.5 million people across the U.K. living with back pain, this research brings fresh hope that potential new therapeutic approaches are on the horizon,” added Caroline Aylott, PhD, head of research delivery at Arthritis UK. “We are proud to fund research that is unlocking the science behind the processes leading to spinal disc degeneration. Back pain is one of the U.K.’s most common conditions that has blighted millions over generations.

“Dr. Erika Kague and her team at the University of Edinburgh have uncovered important genetic evidence that could pave the way for new treatments, bringing us one step closer to a future where fewer people have to live with the daily pain and challenges that back pain can bring.”

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Renewed Importance of CEX in Monoclonal-Antibody Purification

Edusehat — Fri, 07 Aug 2026 04:55:08 +0700

In today’s biopharmaceutical industry, success is no longer defined by how much therapeutic antibody you can produce, but by how well you can purify it. As upstream systems generate increasingly higher titers, downstream purification must evolve to keep pace. At the center of this evolution is cation exchange (CEX) chromatography, a polishing technique chosen not by convention, but by its trusted ability to separate what looks nearly identical.

Monoclonal antibodies (mAbs) have become an important class of therapeutics in modern medicine, used to treat cancers, autoimmune diseases, and infectious threats ranging from Ebola to COVID-19.¹ But producing these powerful biologics is only half the battle. The real challenge—and increasingly the defining step—lies in purification.

Over the past decade, upstream bioprocessing has advanced dramatically. Modern expression systems routinely generate high titers of antibody from mammalian cell cultures, pushing productivity to levels that were once unattainable. However, this progress has shifted the burden downstream. The resulting product streams are denser, more complex, and filled with impurities that must be removed to meet strict regulatory and safety standards.

As Alejandro Becerra, PhD, principal applications scientist and global purification technical lead at Thermo Fisher Scientific, notes, “Cation exchange chromatography is one of the key polishing steps because antibodies have relatively high isoelectric points, or pIs, and many impurities have lower pIs.” His point underscores a broader reality: Although anion-exchange chromatography (AEX) is somewhat standardized, the increased impurity burden of more complex biologic feed streams needs to be addressed by intermediate polishing, usually CEX.

Fig 1. A representation of the monoclonal antibody production process and the broad categories of impurities. [Image generated using ChatGPT by OpenAI, 2026]

The impurities fall into two broad categories. Process-related impurities include host cell proteins (HCPs), residual DNA, and viral contaminants introduced during production. Product-related impurities, meanwhile, arise from the molecule itself and include aggregates, fragments, and charge variants. As antibody modalities evolve, incorporating bispecific formats, antibody–drug conjugates, and engineered scaffolds, this heterogeneity becomes even more pronounced.

Traditional purification strategies, anchored by affinity capture followed by polishing steps, are increasingly being pushed to their limits. While Protein A chromatography can deliver high purity for conventional antibodies, more complex molecules such as Fc fusion proteins, Fab fragments, bispecific antibodies, and antibody-drug conjugates, often emerge from capture with significantly lower purity—sometimes below 80%. If optimization of the capture step is deemed too much of a challenge, the burden of achieving final product quality therefore shifts to downstream polishing, where subtle differences between molecules must be resolved with precision.

Hydrophobic interaction chromatography (HIC) can be used for aggregates and/or HCPs, but CEX is the more common and powerful tool used to remove charge variants or other impurities with similar pIs. The mechanism is also well understood in the context of the separation, and a well-developed CEX unit operation can advance a candidate molecule toward clinical use.

Why CEX matters

Fig 2. A representation of Cation Exchange Chromatography (CEX) resins [Thermo Fisher Scientific]

CEX chromatography relies on charge-based interactions to separate molecules. Under mildly acidic conditions, mAbs typically carry a net positive charge and bind to negatively charged chromatography media. Impurities interact differently depending on their own charge distribution, the specific chemistry of the resin, and the composition of the mobile phase. By correctly choosing a suitable CEX resin and systematically developing the appropriate operating conditions, challenging impurities can be separated and removed.

What sets CEX apart is its ability to remove product-related impurities that closely resemble the target molecule. Among these, high molecular weight aggregates are particularly crucial. These multimers can form during cell culture, downstream processing, or even within the chromatography columns themselves. Because they are structurally similar to the desired antibody, they are difficult to remove using traditional purification steps.

Purity is “one of the main product-quality measures that we look for,” says David Brown, PhD, associate director, process development at KBI Biopharma. “Aggregates can form during production, and they are a key measure of product quality.” These aggregates are closely monitored because they can trigger immune responses or compromise therapeutic efficacy.

Fig 3. A representative process chromatogram for cation-exchange chromatography (CEX) [BioProcess International]

There are no specific regulatory requirements for aggregates. Each drug sponsor determines the acceptable value based on safety and efficacy as well as stability of the final drug product. Some programs require levels below two percent, others below one percent and, in some cases are acceptable with levels as high as four or five percent Achieving these targets consistently requires both precise control of process conditions and the use of high-performance chromatography materials capable of resolving barely discernable differences in molecular charge and structure.

CEX chromatography also plays a role in removing other types of challenging impurities, including charge variants and residual contaminants that persist after affinity capture. Its versatility and precision make it an indispensable component in the modern antibody-purification workflows.

CEX provides a degree of flexibility that is particularly valuable in development environments. Because separation can be tuned through relatively simple adjustments in buffer composition and the correct CEX resin selection, scientists can rapidly explore different operating conditions to optimize performance. This adaptability is especially important when working with novel or poorly characterized molecules, where prior knowledge might be limited and iterative experimentation is required. It also allows teams to respond quickly when upstream changes introduce new impurity profiles that must be addressed downstream.

The resin decision

The effectiveness of CEX chromatography depends heavily on the resin used. Resin selection is not merely a technical choice; it is a strategic decision that influences process efficiency, scalability, and cost. Developers must consider multiple factors simultaneously, including binding capacity, resolution, robustness, and pressure-flow behavior.

Becerra explains that the process begins with defining what the purification operation should accomplish. As he adds, this end goal must be attained “without losing sight that these processes will be eventually scaled up.” This dual focus ensures that conditions optimized during development can be translated into manufacturing environments.

Brown highlights the practical considerations: “We’re looking at aggregate removal, step yield, binding capacity, and pressure/flow dynamics.” Each of these factors plays a crucial role. High binding capacity reduces the amount of resin required, lowering cost. Strong resolution ensures effective separation of impurities. Favorable pressure/flow characteristics enable high flow rates without excessive backpressure, supporting efficient large-scale operation.

To navigate these trade-offs, many organizations adopt systematic approaches to resin selection. KBI Biopharma, for example, uses a “resin toolbox strategy, screening multiple resins against a library of molecules to build a robust dataset,” Brown says. This allows rapid decision-making when new programs arise, reducing development time while maintaining confidence in performance.

An example of screening a CEX resin toolbox is the comparative study of several commercially available CEX resins, “Streamlining cation exchange chromatography process development for therapeutic monoclonal antibody purification” published by Lau et al in the peer-reviewed Journal of Chromatography A. From their study of 3 mAb feeds and 5 CEX resins, the authors concluded that for industrial applications POROS XS had outperformed the other resins tested, “demonstrating outstanding column performance and impurity clearance. Application of Poros XS contributes to a highly efficient and robust manufacturing process, enhancing productivity while maintaining high product quality.”²

Fig 4. A graph showing how the capacity of different CEX resins compares to Thermo Fisher’s POROS XS Strong CEX resin [Thermo Fisher Scientific]

Thermo Fisher’s POROS XS Strong CEX resin has emerged as a trusted option. Its design addresses several of the key challenges in modern purification, especially resolution. This resin’s relatively small particle size and large through-pore structure allow more accessible surface area, which leads to high dynamic binding capacity. POROS XS resins can achieve capacities exceeding 100 g/L for mAbs which can improve throughput and reduce resin volume requirements comparted to lower capacity CEX resins.³

Moreover, the resin’s rigid poly(styrene-divinylbenzene) backbone provides mechanical strength and supports high flow rates with minimal pressure increase. This enables stable operation and scalability across different process scales.

Fig 5. Three main attributes differentiate POROS from other chromatography resins: 1) polystyrene-divinylbenzene beads there are stable, linear, and have scalable pressure-flow performance; 2) a large pore structure, that reduces mass transfer; and 3) an average particle size of 50um that improves separation and achieves effective purity removal [Thermo Fisher Scientific]

Real-world experience demonstrates this resin’s utility. Brown notes: “We’ve used the POROS XS resin extensively, and it has shown a good balance of aggregate clearance, step yield, capacity and pressure/flow dynamics.” This combination of attributes makes it particularly well suited for both development and manufacturing environments.

Importantly, the robustness of POROS XS resins extends beyond performance metrics. Its chemical stability across a wide pH range and tolerance to harsh cleaning conditions allow for extended resin lifetime and reuse. This contributes to lower overall cost of goods and improved process sustainability—factors that are increasingly important as biologics manufacturing scales globally. In large-scale facilities where chromatography columns are cycled repeatedly, durability translates directly into fewer resin replacements, reduced downtime, and more predictable manufacturing schedules.

Driving efficiency

Although resin performance is crucial, process design can further enhance efficiency. In “Cation exchange chromatography performed in overloaded mode is effective in removing viruses during the manufacturing of monoclonal antibodies,” which was published in Biotechnology Progress, by Masuda et al, scientists investigated alternative operating modes for CEX chromatography.⁴

Traditionally, CEX polishing is performed in bind-and-elute mode, where the antibody binds to the resin under low-salt conditions and is later eluted by increasing salt concentration or pH. Although effective, this approach requires significant resin volumes, increasing costs at scale.

To address this, Masuda and her colleagues evaluated an overloaded mode of operation. In this approach, the column was intentionally loaded with POROS XS resin beyond its nominal binding capacity. Instead of relying solely on binding, separation was driven by differences in binding affinity between the antibody and impurities.

The results were striking. Even at extremely high loading levels (up to 2,000 grams of antibody per liter of resin), viral clearance remained effective. Viruses such as murine leukemia virus were found to bind more strongly to the resin than the antibody, remaining on the column while the purified product was eluted.

This behavior enabled the simultaneous removal of multiple impurities, including aggregates, HCPs, and viruses—all in a single step. Importantly, viral clearance performance was not significantly affected by resin type nor by antibody variant, suggesting that the approach is broadly applicable.

The implications are substantial. Overloaded operation reduces resin requirements, lowering costs and improving process efficiency. It also simplifies workflows by combining multiple purification functions into a single step. At the same time, it highlights the importance of understanding molecular interactions, as these interactions ultimately govern separation performance.

Beyond cost savings, the study also underscores a shift in thinking about purification design. Rather than treating each step as a fixed unit operation, researchers are increasingly exploring flexible modes that adapt to process needs. Overloaded CEX represents one such innovation, demonstrating how established techniques can be reimagined to meet modern manufacturing demands.

Precision separation

Advanced purification will also be required for next-generation therapeutics. Bispecific antibodies present unique purification challenges because they require the correct pairing of multiple heavy and light chains. Mispaired variants so closely resemble the desired product that they can be difficult to remove.

Fig 6. Bispecific antibodies present unique purification challenges since mispaired variants can often be like the desired product [Getty Images/Love Employee].

In “Structural study of a light chain mispaired bispecific predicts mechanism of downstream separation,” published in the Journal of Chromatography A, by Cha et al, researchers addressed this challenge using CEX chromatography with POROS XS resin, combined with detailed structural analysis.⁵ In one case study, a mispaired variant disrupted a positively charged region on the antibody surface. This disruption weakened its interaction with the resin, allowing it to be selectively removed during washing.

Through high-throughput screening and careful optimization of pH and salt conditions, the team identified a process that enabled clear separation between the desired product and mispaired variants. The result was a significant improvement in purity, with the final product reaching 94.78%.⁵

What makes this work particularly notable is the integration of computational modeling with experimental chromatography. By analyzing electrostatic surface properties, researchers predicted how different variants would interact with the resin. This predictive capability enabled more targeted optimization and reduced reliance on trial-and-error experimentation.

The study demonstrates how subtle differences in molecular structure, such as changes in surface-charge distribution, can have a profound impact on purification outcomes. It also highlights the potential of combining structural biology and chromatography to address increasingly complex purification challenges. As antibody formats continue to diversify, such integrated approaches are likely to become standard practice, particularly for molecules where traditional purification heuristics fall short.

Toward smarter, integrated processes

The development of advanced CEX processes is undergoing a transformation driven by data, automation, and modeling. High-throughput screening platforms allow researchers to test multiple conditions simultaneously, exploring a wide range of pH, conductivity, and loading parameters. Statistical design methods help define optimal operating windows, while mechanistic models provide insight into the underlying processes.

One of the key insights from these approaches is the trade-off between yield and purity. Conditions that maximize binding strength might not produce the cleanest separations, while conditions that improve purity may reduce recovery. The optimal process lies in balancing these competing factors within a defined operating space.

Mechanistic modeling is playing an increasingly important role in achieving this balance. By simulating how molecules move, bind, and separate within a chromatography column, these models can predict process performance under different conditions. Once calibrated with experimental data, they provide a powerful tool for reducing development time and improving process understanding.

At the same time, advances in resin chemistry are expanding the capabilities of chromatography. New materials are being developed to address specific challenges associated with next-generation therapeutics, including higher levels of aggregation and increased structural complexity.

In addition, integrated approaches could transform purification from a reactive process into a proactive, design-driven discipline. Increasingly, developers are viewing purification as an interconnected system rather than a sequence of isolated steps, enabling more holistic optimization across the entire workflow.

From bottleneck to advantage

Purification is no longer a downstream bottleneck struggling to keep pace with upstream production. It is becoming a strategic advantage—one that determines not only product quality, but also development speed and manufacturing efficiency.

Fig 7. To effectively address current challenges and industry demands, process development scientists must balance multiple factors when developing new processes. As a result, capacity, resolution, and speed must be simultaneously optimized [Thermo Fisher Scientific]

By combining high-performance resins like POROS XS Strong CEX Resin with data-driven optimization and collaborative development approaches, the industry is transforming how mAbs are refined. Companies are increasingly working in partnership with technology providers to design processes that are robust, scalable, and adaptable.

This shift reflects a broader change in mindset. Purification is no longer viewed as a necessary but secondary step. Instead, it is recognized as a crucial component of therapeutic development, one that requires the same level of innovation and attention as upstream production.

As biologics become more complex and production scales continue to rise, the importance of precise, efficient purification will only grow. In this evolving landscape, the ability to separate what is nearly indistinguishable and helping to remove important impurities will define success.

CEX chromatography now stands at the forefront of that effort. By enabling the removal of crucial impurities and supporting scalable, cost-effective manufacturing, it helps modern therapeutics to be purified with the precision required for improved therapies.

Learn more at: thermofisher.com/porosXS

REFERENCES

Lu, R-M., Chiang, H-L., Yuan, J. P-Y., et al. Technological advancements in antibody-based therapeutics for treatment of diseases. J. Biomed. Sci. 32:98 (2025).
Lau, W.Y., Mi, X., Dumont, A., Yang, L. Streamlining cation exchange chromatography process development for therapeutic monoclonal antibody purification. J. Chromatogr. A. 1762, 466391 (2025).
Thermo Fisher Scientific. POROS XS Strong Cation Exchange Resin. https://documents.thermofisher.com/TFS-Assets/BPD/Flyers/poros-xs-resin-flyer.pdf
Masuda, Y., Tsuda, M., Hashikawa-Muto, C., et al. Cation exchange chromatography performed in overloaded mode is effective in removing viruses during the manufacturing of monoclonal antibodies. Biotechnol. Prog. 35(5), e2858 (2019).
Cha, M., Xu, A., Williams, A.J. Structural study of a light chain mispaired bispecific predicts mechanism of downstream separation. J. Chromatogr. A. 1730, 465117 (2024).

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Tau Protein Linked to Mitochondrial Reverse Electron Transport in Preclinical Models

Edusehat — Fri, 07 Aug 2026 01:15:10 +0700

Studies by researchers at Stanford University School of Medicine and at the University of California, San Francisco, have found that the protein tau, which is implicated in neurodegenerative disorders including Alzheimer’s disease, may be linked to these such disorders in a way that differs greatly from the pathological pathway usually ascribed to it.

Hyperphosphorylation and aggregation of tau are hallmarks of primary and secondary tauopathies including frontotemporal dementia (FTD) and AD, and potentially also Huntington’s disease and Parkinson’s disease. Such disorders also share another common pathology, which is deteriorating performance of the cell’s mitochondria. The powerhouses may number in the dozens or in the tens of thousands within a single cell, depending on the cell type’s energy needs. Nerve cells have especially high mitochondria demand.

To date the connection between tau and mitochondrial pathologies has been unclear. Working in cells and in preclinical animal models, the Stanford and UCSF scientists have now shown that phosphorylated tau can enter mitochondria and interfere with the electron transport chain, initiating a vicious cycle of pathological events and triggering reverse electron transport (RET) and the detriments that ensue.

“This is the first demonstration of exactly what tau does inside mitochondria,” said Bingwei Lu, PhD, Stanford professor of pathology, “Our discovery of a whole new mechanism driving tauopathies renders these disorders amenable to new therapeutic interventions.” Lu is senior author of the researchers’ published paper in Neuron, titled “Tau-induced mitochondrial reverse electron transport drives neurodegeneration,” in which they concluded “Our results suggest that RET may serve as a common pathogenic mechanism linking tau abnormalities to mitochondrial dysfunction across diseases.”

Tau is a soluble protein enriched in neuronal axons but is also found in neuronal dendrites, cell bodies, and non-neuronal cells, the authors explained in their report. Tau is also increasingly viewed as one of the strongest instigators of Alzheimer’s disease. The appearance of telltale forms of the protein in cerebrospinal fluid or in the bloodstream strongly predicts impending Alzheimer’s symptoms. Neuroimaging studies and postmortem inspections indicate the presence of neurofibrillary tangles—long filaments largely composed of tau—inside Alzheimer’s patients’ nerve cells.

Neurofibrillary tangles and other aspects of tau’s misbehavior—notably, a tendency to rack up chemical modifications that shift that protein’s disposition—have been reported in Parkinson’s disease and Huntington’s diseases and in other tauopathies such as frontotemporal dementia and progressive supranuclear palsy. “Tau is phosphorylated at many sites under normal conditions but becomes hyperphosphorylated in disease,” the team also commented.

Tauopathies share another common pathology, which is deteriorating mitochondrial performance. “Mitochondrial dysfunction is also a common feature of tauopathies,” the investigators stated. “The mechanistic link between tau abnormalities and mitochondrial dysfunction and its relationship to the physiological function of tau, however, is unclear.”

In its healthier manifestation, tau is believed to play a role in stabilizing microtubules, skeletal structures in nerve cells that are critical to these cells’ proper operation. Tau molecules do indeed spend some of their time sitting on microtubules, straddling those structures’ identical subunits. So, not unreasonably, the consensus is that tau’s perch on microtubules helps keep them from falling apart.

The newly discovered pathological pathway is entirely independent of both neurofibrillary tangle formation and microtubule instability. Instead, it involves a switch in the directionality of mitochondria’s energy-production line, with a resulting disruption of mitochondria’s primary function, that being the conversion of calories from glucose or fat to energy by the electron-transport chain. This multiple-component complex passes electrons from one to the next of its components, the last of which converts a precursor molecule into ATP, the cell’s universal energy currency.

The new study shows that when the hyperphosphorylated tau molecule interacts with a key mitochondrial component it jams up the electron transport conveyor belt, causing electrons to flow backward. Aptly named reverse electron transport (RET), this snarl produces large amounts of reactive oxygen species (ROS), with accompanying inflammation and damage to proteins.

Reverse electron transport is an area of intense recent interest in biology. Although it was first discovered in the 1960s, there’s still no clear evidence that it serves any constructive physiological role. “In healthy cells, very little reverse electron transport is happening,” Lu said.

The new study shows that reverse electron transport is activated under stress. It may initially serve some beneficial function—for example, providing short-term adaption to that stress—but nothing like that has been proven. “Here, we demonstrate that tau regulates mitochondrial reverse electron transport (RET), which produces excess reactive oxygen species (ROS), reduces the NAD+/NADH ratio, and is activated by aging or stress,” the investigators noted.

The team carried out an extensive series of experiments in fruit flies, mice, human brain tissue and cultured human nerve cells that in some cases contained mutated genes for tau identical to those found in tauopathy patients. They also employed lab-generated nerve cells carrying a well-studied gene duplication that promotes accelerated acquisition of Alzheimer’s disease.

Through their studies they demonstrated that reverse electron transport was occurring in animal models of tauopathy as well as in tauopathy-afflicted human brain tissues. Healthy nerve cells, largely spared of hyperphosphorylated tau’s malevolent presence, showed no sign of reverse electron transport or its downside effects.

Next, the investigators showed how reverse electron transport is activated, finding that tau molecules enter mitochondria, but only when they’re phosphorylated. There, they can bind to a component of the electron-transport chain called NDUSF3, warping that protein’s shape. When this happens, electrons drop off the conveyor belt and start flowing backward. “Mechanistically, tau enters mitochondria and directly interacts with the complex I subunit NDUFS3 to promote RET in a phosphorylation-dependent manner,” they continued. “Elevated RET further drives tau hyperphosphorylation, establishing a self-perpetuating pathological loop.”

Studies showed that genetically or pharmacologically depleting tau halted this defection. An experimental drug called CPT prevented hyperphosphorylated tau from binding to NDUSF3, blocking reverse electron transport without impairing normal electron flow. Experimental animals that were genetically altered to produce no or little tau, suffered none of the cognitive or other behavioral deficits or brain pathophysiology exhibited by tau-producing, but otherwise genetically identical animals under stress conditions. “In flies, mice, and human induced pluripotent stem cell (hiPSC)-derived neurons, tau depletion eliminates stress-induced RET and confers resilience,” the authors stated.

Deleting the gene for tau, for instance, protected fruit flies from the severe, life-shortening nervous-system damage that normally results from prolonged exposure to elevated temperatures. CPT treatment of the tau-producing normal flies not only protected them against heat stress but extended their lifespan.

The study showed equivalent findings in mic engineered to not produce tau. Cognition in these animals was protected by CPT treatment from the detrimental effect of heat stress. CPT also protected tau-producing normal mice subjected to heat stress.

Tau hyperphosphorylation proved critical for promoting reverse electron transport. Only tau molecules that had undergone particular phosphorylation events could get inside mitochondria, bind to NDUFS3, and induce reverse electron transport.

In tauopathy mice with severe cognitive deficiencies, an extended CPT regimen inhibited reverse electron transport in the brain mitochondria. This significantly improved the animals’ performance on a wide range of behavioral tests and prevented nerve-cell inflammation as well as several characteristic markers of neurodegeneration, such as diminished cortical thickness and total brain volumes.

“Crucially, therapeutic inhibition of RET mitigates tau-induced neurotoxicity in multiple models, without observable detrimental effects on normal animals,” the authors noted. “The RET inhibitor CPT effectively disrupts the pathological loop between RET and tau phosphorylation, ameliorating neurotoxicity across species. In fly and mouse models of tauopathy, CPT treatment rescues behavioral deficits, reduces neuroinflammation, and mitigates neurodegeneration.”

Reverse electron transport is a textbook example of a vicious circle, Lu said. The massive release of highly reactive chemicals dramatically boosts the odds that individual tau molecules will get hyperphosphorylated, leading to additional activation of reverse electron transport. “Once this gets started, it can become self-perpetuating,” he commented. Reverse-electron-transport inhibition holds promise as a therapeutical strategy for tauopathies and, potentially, other maladies characterized by aberrant tau phosphorylation and mitochondrial dysfunction, such as brain tumors, stroke and traumatic brain injuries, Lu suggested.

“RET regulation thus represents a previously unrecognized normal function of tau that becomes pathological in disease, providing a therapeutic target for various conditions characterized by tau abnormalities and mitochondrial dysfunction,” the authors stated in their paper. “These findings suggest that RET inhibition holds promise as a therapeutic strategy for not only tauopathies but potentially other brain diseases characterized by aberrant tau phosphorylation and mitochondrial dysfunction.”

Lu added, “The main results we observed in our animal models were also seen in patient brain tissues and in the laboratory generated nerve-cell models we derived from tauopathy-patients’ cells. This suggests that what we learned from this study is applicable to the human nervous system. In fly and mouse models of tauopathy, CPT treatment rescues behavioral deficits, reduces neuroinflammation and mitigates neurodegeneration. In hiPSC-derived neurons carrying pathogenic tau mutations, CPT protects against stress-induced cellular abnormalities.”

These are early days for this compound’s clinical development, he stated. “Much more work remains to be done before it can undergo clinical trials.” Lu is co-founder and sits on the advisory board of Cerapeut, a company that is developing CPT as a therapeutic drug for the treatment of neurodegenerative diseases.

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DNA Script Wins ARPA-H Award to Advance DNA Manufacturing

Edusehat — Thu, 06 Aug 2026 21:40:09 +0700

DNA Script, in collaboration with GE HealthCare, has been awarded up to $26 million in funding for a four-year initiative from the Advanced Research Projects Agency for Health (ARPA-H). The award will support the advancement of the Flexible Automation for Scalable Health (FLASH) program, focused on the development of scalable cell-free DNA bioproduction capabilities and powered in part by DNA Script’s enzymatic DNA synthesis (EDS) technology and SYNTAX^® platform.

Led by GE HealthCare, the FLASH program aims to develop a modular, automated platform for the rapid, distributed manufacturing of high-fidelity DNA to support research and potential future applications in personalized medicines, vaccines, and other genetic health technologies. DNA Script’s EDS technology supports this goal by enabling faster and more flexible DNA manufacturing workflows.

DNA Script, which is contributing its EDS technology and acting as the technical solution integrator for the project, says the company has adapted its solution to incorporate GE HealthCare’s proprietary DNA scaling technology, complementing DNA Script’s EDS technology, with the goal of eventually deploying the integrated platform across U.S. medical research centers, universities, and federal agencies.

“ARPA-H solutions are designed to address hard problems with bold, practical approaches,” says John Schiel, program manager, ARPA-H. “Projects like FLASH exemplify how scalable, platform-based technologies can transform the way health solutions are developed, produced, and delivered.”

“Our enzymatic DNA synthesis platform is ideally suited for the on-demand manufacturing model FLASH is pioneering,” adds Marc Montserrat, CEO, DNA Script. “Collaborating with GE HealthCare on an ARPA-H program of this scale shortens the path from research bench to patient bedside for next-generation therapies, personalized vaccines, and the broader genetic-medicine pipeline.”

“As personalized and time-sensitive genetic medicines continue to advance, there is a growing need for manufacturing approaches that are faster, more flexible, and more accessible,” notes John Nelson, senior bioscience principal and FLASH program lead, GE HealthCare’s Technology and Innovation Center. “Our vision for the FLASH program is to develop new DNA-based medicines on demand quickly, safely, and at scale.”

The FLASH program has been designed to bring together expertise in automation, DNA synthesis and scaling, purification, and genomic validation.

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COVID-19 Reactivates Dormant Viruses, Offering New Clues to Long COVID

Edusehat — Thu, 06 Aug 2026 07:20:08 +0700

Chronically infecting viruses—such as Epstein Barr, cytomegalovirus, and herpes virus—are ubiquitous in humans. Although their presence is often innocuous and asymptomatic, the viruses can reactivate during stress, and emerging evidence suggests that their reactivation may contribute to autoimmune disease and other chronic conditions. SARS-CoV-2 infection is known to reactivate some chronic viruses, yet the full extent of the effects is not well understood.

Now, a study including 15 biomedical research institutions across the United States, Boston Children’s Hospital researchers and their collaborators have discovered that COVID-19 reactivates certain dormant viruses in hospitalized patients. These findings expand understanding of chronically infecting viruses and could inform development of strategies to combat their reactivation.

This work is published in a new study in Nature, entitled, “Virus reactivation in acute and long COVID-19.”

The study leveraged multiomic longitudinal data of 1,154 patients with COVID-19 from the Immunophenotyping Assessment in a COVID-19 Cohort (IMPACC) study across 20 U.S. biomedical research hospitals. It was designed to define biomarkers of COVID-19 severity and outcomes.

“This is the largest and most comprehensive biomarker study of COVID-19, in which we followed more than one thousand patients, collected more than 200,000 samples, and generated more than one billion data points over the course of a year for this public resource,” says Joann Diray Arce, PhD, who leads the PVP-Data Management and Analysis Core and is the lead of the study’s Clinical and Data Coordinating Center.

The research team detected 11 reactivated viruses in patients within the first 40 days from admission, with the most detected ones being Epstein-Barr, herpes simplex 1, cytomegalovirus, and Anelloviridae viruses. Notably, reactivation of Anelloviridae, a poorly understood family of viruses typically latent in about 90 percent of the population, was associated prominently with long-term physical disability and long COVID.

“This association with long COVID is an interesting finding as millions around the world suffer from this chronic condition,” says Ofer Levy, MD, PhD, director of the Precision Vaccines Program (PVP) at Boston Children’s. “Having new insight as to the molecular and viral associations with long COVID could point the way to better understanding and ultimately better diagnostics and treatments.”

In an analysis of the blood samples from the patients, Epstein-Barr and cytomegalovirus seemed to activate in response to inflammation rather than immune system suppression. The researchers say this is a surprising new mechanism, challenging the prevailing view that chronic viral reactivation is primarily a consequence of immunosuppression. This finding demonstrates that reactivations occur frequently in apparently immunocompetent individuals during severe illness and in association with increased systemic inflammation.

In addition, the authors write, the findings “challenge the prevailing view that chronic viral reactivation is primarily a consequence of immunosuppression, demonstrating that reactivations occur frequently in immunocompetent individuals during severe illness and in association with increased systemic inflammation.” They also demonstrate persistence of viral reactivation in convalescence and report an association of Anelloviridae with long COVID.

“Although many no longer think of COVID being a problem, up to 50,000 Americans died of COVID in 2025-2026 respiratory season and some estimates suggest over 10 million U.S. adults suffer from long COVID,” says Levy. “We need to help these patients recover with the best outcomes.” He adds “Moreover, sooner or later, there may be another coronavirus pandemic, which means we need to learn all the lessons we can from COVID-19 to be better prepared.”

Next steps for this work will be to uncover how the immune system responds to these viruses over the course COVID-19, with the aim of identifying effective therapeutics and establishing the optimal timing of any interventions.

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Enzene Targets Local Biomanufacturing with New Solutions

Edusehat — Thu, 06 Aug 2026 00:10:16 +0700

CDMO Enzene launched NeX, which it labels “an end-to-end partnership model that enables governments, institutions, and biopharma organizations to establish world-class biomanufacturing capabilities wherever they need them.”

NeX, built on the EnzeneX^® continuous manufacturing (FCCM) platform, is designed to deliver a turnkey and scalable way to make biologics manufacturing transportable, close to patients and supply chains, while avoiding the capital burden of conventional fed‑batch facilities.

By enabling local FCCM‑based manufacturing, governments can significantly expand patient affordability and access to life‑saving biologics, according to Himanshu Gadgil, PhD, CEO of Enzene.

“A conventional biologics plant often requires between $300 million and $400 million to build,” he says. “With our fully-connected continuous manufacturing platform, the same output can be achieved for $60–$80 million. The efficiency comes from a compact, integrated system architecture rather than the scale-intensive layouts of fed-batch facilities, allowing for long-term cost advantage and high product yields for locally manufactured biomedicines.”

Enzene’s NeX program is intended to help regions accelerate the establishment of local biopharma capabilities and risk-mitigated supply chains, develop domestic scientific talent, and reduce long-term manufacturing costs for local patients, continues Gadgil, adding that the company estimates that facilities built under the program can achieve validation and commercial readiness in under three years.

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Electrical Stimulation’s Effects on Neurons, Gene Expression Mapped in Living Human Brain Tissue

Edusehat — Thu, 06 Aug 2026 00:10:15 +0700

Neurons from living human brain tissue have helped researchers trace how electrical stimulation reshapes brain cell communication and gene activity—work that could guide more precise neuromodulation strategies for cognitive decline and other neurological conditions in the future.

In a study published in Nature, researchers from UCLA Health and the University of Texas Southwestern Medical Center developed an ex vivo platform using human temporal cortex tissue donated by neurosurgery patients and maintained alive in the laboratory for several days. The approach allowed the team to apply electrical stimulation resembling deep brain stimulation, record neuronal activity, and map gene expression changes across individual brain cell types.

The paper, titled “Stimulation modulates gene-linked cell assemblies in the human brain,” addresses a key gap in understanding how stimulation-based therapies affect human brain tissue at the cellular and molecular levels. Although deep brain stimulation is already used for disorders such as Parkinson’s disease and obsessive-compulsive disorder, its effects on different human brain cell types and the genes they activate have not been well defined.

To investigate those mechanisms, the researchers integrated microelectrode array stimulation with simultaneous recording and single-nucleus genomics from resected temporal cortex obtained from neurosurgery patients. In the abstract, the authors wrote that they developed the platform “to directly investigate the mechanisms of neuromodulation elicited by human brain stimulation.” They reported that stimulation strengthened coordinated groups of neurons, or cell assemblies, and then connected those physiological changes to cell-type-specific gene regulatory networks.

After stimulation, brain cells became more synchronized in how they communicated. “These assemblies exhibited stimulation-dependent increases in activation strength and membership flexibility, with analogous properties to compositional drift observed in memory-related assemblies in vivo,” the authors write. The team also found that neurons and non-neuronal support cells, including astrocytes, activated distinct genetic programs in response to stimulation.

“Not only was it a privilege and challenge to work with donated living human brain tissue, but to see it reveal the genes and cell types underlying human brain plasticity as new targets for future therapies makes the work feel even more meaningful,” said senior author Genevieve Konopka, PhD, chair of the department of neurobiology at UCLA Health.

The donated samples came from the temporal cortex, a region on the sides of the brain’s outer layer that is important for memory and related cognitive functions. The authors noted that stimulation of cortical circuits is being explored as a therapeutic strategy for restoring cognitive function, but the biological mechanisms underlying its effects in humans have remained largely unexplored.

The study also points to several open questions. Additional work is needed to determine the molecular effects of long-term stimulation, how stimulated cells influence neighboring cells, and whether similar mechanisms are active in deeper brain regions, which are harder to obtain from living donors. Still, the authors concluded that the work establishes “a foundation for identifying targetable genetic signatures linked with physiology” that could potentially be harnessed through neuromodulation strategies.

“By understanding exactly which genes turn on in which cells during stimulation, we can start to design more precise approaches to deep brain stimulation and potentially augment this clinical strategy with pharmacological therapies to help slow cognitive decline,” added Konopka.

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Seamless Integration in Gene Therapy Process Development

Edusehat — Thu, 06 Aug 2026 00:10:14 +0700

The manufacturing question over adeno-associated viruses (AAV) for gene therapies has moved beyond whether a product can be made to whether it can be made consistently. That’s according to Forge Biologics, a contract manufacturer specializing in AAV production.

According to Sumit Dutta, associate director of upstream process development at Forge Biologics, AAVs maturing as a technology means the industry should become more focused on how best to bring products to market.

“There’s a very heavy focus and investment on late-stage programs, and we need to bring the focus to [process robustness] and thinking about approaches carefully so therapies can get to market, and become available to patients, sooner,” he says.

Forge Biologics works on multiple different AAV programs, allowing them to spot patterns between programs and learn from that, explains Dutta, who argues that they focus on reducing risk and seamless integration between process development for early- and late-stage clinical programs.

“We have early engagement with our key stakeholders so we’re walking in lockstep about what we’re developing, who we’re developing it for and our final target,” he explains. “We ensure the process and technical solutions the development team are coming up with is what is required for the manufacturing process to succeed.”

That can involve identifying critical process parameters and critical quality attributes early and confirming how to study them with high throughput by developing representative scale-down models, he says.

Forge Biologics have also integrated considerations of U.S. Food and Drug Administration (FDA) and other regulatory guidelines throughout their process development, Dutta says.

“Integrating those approaches and ensuring quality and commercialization needs are [also] met along the way is a [key part of] this approach,” he says.

Dutta will be speaking about Forge Biologics’ late-stage development approach at the Bioprocessing Summit in Boston this month.

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New Bioreactor Design Could Boost mAb Yields

Edusehat — Thu, 06 Aug 2026 00:10:13 +0700

Current bioreactors are struggling to meet growing demand for mAb therapeutics, according to researchers, who suggest an innovative rotating-drum design could help boost output and reduce production costs.

Monoclonal antibodies (mAbs) are employed in a broad range of therapeutic applications—from the treatment of cancer and autoimmune diseases to the management of viral infections and the prevention of tissue rejection.

They are a major focus of biopharmaceutical industry R&D efforts—13 of the 16 biologic products approved by the FDA in 2024 were mAb-based drugs.

And—based on a recent forecast by McKinsey—demand for mAb therapeutics is set to go on increasing over the next decade.

Various technologies are used to make mAbs—from bubble column to fluidized bed bioreactors. However, the most widely used systems are stirred-tank bioreactors, consisting of a tank, an impeller for homogenizing the culture medium, and a sparger for supplying oxygen to the cells.

Stirred-tank bioreactors are effective for mAb production, but the yields they achieve are still relatively low—typically, less than a tenth of a gram per liter.

Output is largely dependent on a reactor’s ability to make sure cells have the nutrients they need to growth, say researchers at the Italian National Agency for New Technologies, Energy and Sustainable Economic Development (ENEA).

“The main bottleneck limiting the maximal efficiency of these traditional bioreactors is the availability of dissolved oxygen throughout the entire fermentation cycle, due to its low solubility in water.

“As culture density increases, oxygen demand rises, often making oxygen transfer a rate-limiting factor in bioreactor systems. Inadequate oxygen supply can lead to hypoxic stress, resulting in reduced cell growth, decreased protein expression, and shifts toward undesirable metabolic pathways,” they write.

Innovation

To address this, manufacturers typically increase agitation to help cells access oxygen more effectively. However, this can increase shear stress, which is detrimental to CHO cell growth because it can disrupt membranes and induce apoptosis.

A more promising potential alternative, according to the ENEA researchers, is a new rotating drum bioreactor originally designed for bacterial growth for applications in wastewater treatment.

The prototype consists of a horizontal chamber, coupled to a slow-rotating perforated basket. The basket contains two perpendicular paddles, designed to ensure the efficient homogenization of the culture. The chamber is equipped with several inlet and outlet ports and probes for monitoring temperature, pH, foam formation and O₂ level.

According to the authors, “The main innovative principle underlying this prototype involves increasing the liquid surface area exposed to the headspace, thereby promoting gas exchange at low rotational speeds.”

Head-to-head test

Trials of the bioreactor suggest the decision to focus on boosting oxygen availability was the correct approach. In head-to-head comparisons, the bioreactor achieved a titer of 1.3 ± 0.09 g/ L at day 10. In contrast, a titer of 0.71 ± 0.006 g/ L was obtained in a traditional bioreactor.

According to the authors, rotating drum bioreactors are a promising alternative to conventional systems, supporting mammalian cell growth while maintaining high viability and enhancing mAb production.

“Monoclonal antibody production reached 1.3 ± 0.09 g/ L, almost doubling the yield obtained in the stirred-tank reactor. The improved outcomes observed in the innovative bioreactor could be associated with the distinct operating and hydrodynamic conditions established by the system configuration,” they conclude.

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AI “Council of Models” Improves Workflows and Outcomes

Edusehat — Thu, 06 Aug 2026 00:10:12 +0700

To maximize the benefits of AI, biopharmaceutical manufacturers need to take an end-to-end systems engineering approach to their data, preparing it for AI while orchestrating the right models for each stage of a workflow. Relying solely on a single foundation model is often insufficient, particularly as complexity increases and therapeutics advance from pilot stages into production.

No single model excels at every task. Running the same engineering process through different AI models often produces significantly different results. Even repeated runs on the same model can yield inconsistent outputs, Farshid Sabet, CEO and co-founder of Corvic AI, tells GEN.

Variability may be acceptable for low-risk activities, but it becomes problematic when engineering diagrams, flow directions, operational relationships, and other complex data are involved. “Small inaccuracies can compound quickly,” he cautions, leading to unreliable results in production environments.

Corvic AI recently benchmarked leading frontier AI models against Corvic V5’s workflow orchestration platform, assessing their ability to extract piping and instrumentation diagrams (P&IDs) into XML files.

“For general text generation, today’s frontier AI models perform remarkably well and the differences between them are relatively small,” Sabet says. “But engineering workflows introduce an entirely different level of complexity.”

The benchmark found that relying on foundation models alone often resulted in inconsistencies, hallucinations, and poor repeatability. Corvic addresses these challenges by combining semantic data preparation with workflow orchestration that coordinates multiple AI models, validation steps, retrieval, and enterprise context to improve reliability.

Rather than replacing frontier models, Corvic’s platform integrates and orchestrates them, selecting the best model for each stage of a workflow based on the task, performance requirements, and cost.

Like wild horses

As Sabet says, “AI models are like wild horses. They’re incredibly powerful, but they need guidance, structure, and context before they can consistently solve complex enterprise problems.”

Typically, AI developers focus on improving the models themselves through training, fine-tuning, or prompt engineering while assuming enterprise data is already AI-ready. Corvic, instead, focuses on organizing enterprise knowledge through a semantic layer that enables AI systems to understand relationships across engineering documents, databases, diagrams, and operational systems.

“We work with the data independently of whether it’s manufacturing, chemistry, or biology,” Sabet says. “The data has to be organized in a way that allows AI models to recognize context and relationships. That’s the semantic layer.”

Once enterprise knowledge is structured appropriately, organizations can intelligently orchestrate multiple AI models throughout a workflow rather than relying on a single model for every task. Sabet refers to this approach as a “council of models,” where each model contributes its strengths to improve overall accuracy, repeatability, and efficiency.

A former Intel executive, Sabet founded Corvic AI to help organizations operationalize AI across complex enterprise environments. Today, the company works with manufacturers, life sciences organizations, and other enterprises to transform fragmented operational knowledge into reliable AI workflows that improve productivity and decision-making.

For organizations evaluating AI platforms, Sabet recommends looking beyond benchmark scores and considering three factors: how enterprise data is secured and governed, whether the platform intelligently matches AI models to different stages of a workflow, and how success will be measured through meaningful productivity outcomes.

To maximize the benefits of AI, Sabet reiterates, biopharmaceutical manufacturers “need to look at their data from a systems engineering perspective.

“The future of enterprise AI isn’t about finding one perfect model,” he says. “It’s about intelligently orchestrating enterprise data, semantic understanding, and specialized AI models into repeatable workflows that organizations can trust.”

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Building a Bioprocessing Workforce Through Partnerships

Edusehat — Thu, 06 Aug 2026 00:10:10 +0700

As president of the Children’s GMP facility at St. Jude Children’s Research Hospital, Frank Fazio oversees a complex biomanufacturing operation that depends on a highly skilled and diverse workforce. This team is responsible for the manufacture of investigational therapeutics that conform to the FDA’s good manufacturing (GMP). In this interview, he discusses the challenges and opportunities in building and sustaining a bioprocessing workforce.

GEN: How would you characterize the workforce requirements for bioprocessing?

Frank Fazio: We’re essentially a fully integrated little biomanufacturing company. We need skill sets and education levels that range from jobs requiring a GED or high school diploma to PhDs with postdoctoral experience—and everything in between. We need technicians, new PhDs, and experienced scientists. So it’s not a single pipeline of employees; it’s multiple pipelines.

Geography also plays into it. In markets like Boston, Research Triangle Park, or San Francisco, the candidate pool is deep because there are many bioprocessing companies. In other markets, finding people with GMP experience becomes much more difficult.

Organizations like NIIMBL [National Institute for Innovation in Manufacturing Biopharmaceuticals] are also helping enhance the workforce by supporting workforce development through grants, training, and educational content.

GEN: What are the biggest challenges in attracting and retaining a skilled workforce?

Fazio: They’re really two separate challenges: attracting people and keeping them.

The mission of an organization is often what attracts talent. Whether it’s a startup developing a breakthrough technology or addressing an unmet medical need, people want to be part of something meaningful.

Retention depends on organizational culture. You have to reward people for what they do, provide intellectual challenges, and create opportunities for scientific and career growth.

In highly competitive markets, compensation also matters. A startup may suddenly offer salaries that are 15% or 20% above market rates, changing the competitive landscape. Organizations need strategies to either keep pace with those shifts or successfully ride them out.

GEN: Does the St. Jude mission provide an advantage in recruiting?

Fazio: I think that’s true. The mission is what brought me to St. Jude, and the opportunity to be part of that mission is certainly a benefit. But people can also get excited about the technology itself. Gene therapies, messenger RNA technologies, and other advances have generated tremendous enthusiasm, and that excitement helps attract talented people.

GEN: What strategies are most effective for supporting workforce development?

Fazio: I’m fortunate because Children’s GMP is a 70-person organization within a 6,000-person institute. That creates opportunities for employees to grow within St. Jude.

We want people to gain valuable skills in Children’s GMP and then continue advancing elsewhere in the institute if that’s the right next step. That keeps talented employees within St. Jude while giving them meaningful career opportunities. Many of our employees want to stay in the Memphis area, so providing those pathways helps them grow professionally without feeling limited.

GEN: Do you have additional initiatives that are strengthening workforce development?

Fazio: We’ve been working on this aggressively for the past four years through partnerships. We partnered with Southwest Tennessee Community College to create a biomanufacturing-technician internship. Students complete internships during their final semester, gain experience at St. Jude, and have opportunities to apply for full-time positions. We’ve also reached into local high schools to introduce students to careers in biomanufacturing and make them aware of biotechnology programs. At the university level, we partner with the University of Memphis, Rhodes College, and the University of Mississippi through internships, mentoring, presentations, and recruitment efforts. These experiences help students decide whether to pursue graduate school, enter manufacturing, or build careers within the regional biotechnology ecosystem.

GEN: Why are these partnerships so important?

Fazio: I’m an old manufacturing person, so if something isn’t available, you’ve got to make it. That’s how I feel about the workforce pipeline. If the pipeline doesn’t exist, then you have to build it.

We understand the range of skills and educational backgrounds we need, and these partnerships allow us to develop that pipeline. We’re proud of the work we’ve done around workforce development. Creating access to advanced therapeutics is important, but so is education. Being part of an organization like St. Jude means education is in our culture, and our employees take great pride in contributing to that effort.

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Immune Pathway Identified That Prevents C. albicans Infection from Becoming Deadly

Edusehat — Thu, 06 Aug 2026 00:10:09 +0700

King’s College London researchers have identified an immune pathway that prevents what is normally a harmless fungus, Candida albicans, from developing into a fatal infection.

The team’s study, including experiments in mice, identified a central role for the IL-1 family in mediating rapid and protective immunity against C. albicans mucosal infection. If the results of the preclinical study are confirmed in humans, they could help better understand who is at risk of developing fatal fungal infections and also point to a potential therapeutic target.

The results provide the first potential clues as to why only some patients with weakened immune systems—including those undergoing chemotherapy or living with HIV—are at risk of life-threatening Candida albicans infection. James S. Griffiths, PhD, research fellow, King’s College London, said, “Most people carry Candida albicans harmlessly as part of the body’s natural microbiome, but in immunocompromised patients it can spread throughout the body and become life-threatening. A major challenge has been understanding why a fungus that is normally harmless can suddenly spread beyond its natural niche and cause invasive disease. Our study identified the IL-1 family as a critical early immune defense system that helps prevent this fungus from escaping the mouth and gut and spreading to multiple organs. We hope these findings will help identify patients at greatest risk of invasive fungal disease and provide a foundation for developing new ways to strengthen protective antifungal immunity.”

Griffiths is corresponding author of the team’s published paper in Nature Microbiology, titled “IL-1 family signaling drives mucosal defense against systemic Candida albicans infection.”

C. albicans is a fungus that normally lives harmlessly in the mouth and gut but can sometimes spread through the body and cause fatal disease. Fungal infections kill more than 2.5 million people each year, and C. albicans alone kills almost a million. “While mucosal infection is common and contributes to morbidity, it is invasive systemic disease that drives mortality,” the authors explained.

However, scientists haven’t fully understood why fungi can escape their natural locations in the mouth and gut and cause life-threatening disease in around 10% of patients who have a weakened immune system. “With increasing resistance to antifungals, poor diagnostic tools and limited therapeutics, understanding how C. albicans mucosal infections develop and, critically, how they disseminate, is vital to managing C. albicans disease,” the investigators continued.

For their reported study they focused on a signal, IL-1, produced by the immune system to trigger symptoms to fight off infection. IL-1 family members are potent regulators of immunity, the investigators noted, and both insufficient IL-1 activity, and excessive activity, may be implicated in disease. “Here, we investigated how the combinatorial IL-1 family shapes the host immune response to mucosal C. albicans infection and explored the role of the IL-1 family in mucosal–systemic dissemination,” they noted.

The scientists’ study showed that mice genetically modified not to produce IL-1 experienced severe disease when exposed to Candida albicans. The study results suggested that the IL-1 immune pathway is critical in preventing Candida albicans from spreading around the body and causing life-threatening disease.

The team investigated this further by injecting IL-1-deficient mice with a drug that removes neutrophils, a type of white blood cell that is among the first to respond to infections and help fight threats such as bacteria and fungi. This approach allowed the researchers to mimic the weakened immune system seen in some immunocompromised patients. By then introducing Candida albicans to the mouths of those mice, the scientists for the first time observed the fungus spread throughout the body and cause fatal disease, confirming that IL-1 is critical in preventing disease spreading. “Critically, absence of IL-1 family signaling coupled with neutropenia permits C. albicans dissemination from the mucosa, first to the liver and then into multiple organs, mimicking disease experienced by severely immunocompromised patients,” they reported.

While the study focused specifically on Candida albicans, the researchers say the IL-1 immune pathway may be a broader defense mechanism that helps keep fungi normally found in healthy microbiomes from spreading and causing fatal disease, and further research is needed to confirm whether this applies to other fungal species. Understanding what causes fungi that are naturally present in our microbiomes, such as Candida albicans, to cause life-threatening disease could help spot at-risk patients earlier.

The researchers suggest that, if confirmed in humans, the findings could lead to a test that identifies which immunocompromised patients have low levels of IL-1 and so are at heightened risk of Candida albicans escaping their microbiomes and causing disease.

While drugs such as antibiotics are currently used to treat life-threatening fungal diseases, more targeted therapies are needed that tackle the root cause of infection. The researchers suggest future clinical studies in humans could test whether drugs targeting IL-1 could work as a personalized therapy for preventing life-threatening Candida albicans infection.

Co-author Lea Lortal, PhD, a postdoctoral researcher in mycology at the University of California, San Francisco (UCSF), said, “Fungal infections are severely overlooked: they affect more than one billion people worldwide. Yet, there are still no clinically approved vaccines against any fungal pathogen, and our understanding of the immune mechanisms that protect us from fungal disease remains incomplete. What normally keeps fungi, such as Candida albicans, in check has remained a major unanswered question. In this study, we identified the IL-1 family as a key early coordinator of the immune response that helps contain Candida before it can become invasive. Understanding how these protective responses are initiated is an important step toward developing better ways to prevent and treat invasive fungal infections.”

In summary, the authors wrote, “Our findings suggest that combinatorial IL-1 family function plays a crucial role in dissemination risk, offering potential for a personalized therapeutic approach. Consequently, therapeutically enhancing IL-1 family function to augment mucosal immunity and reduce dissemination could have substantial clinical implications.”

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Recursion Partners with Genentech to Advance First Validated Neuro Target Discovered Through AI Map

Edusehat — Wed, 05 Aug 2026 20:35:10 +0700

The 4.5 year, up-to-$12 billion artificial intelligence (AI) drug discovery collaboration of Recursion, Roche, and its Genentech subsidiary marked a milestone Wednesday when plans were announced for Recursion and Genentech to co-develop a neuroscience small molecule early discovery program based on the first validated target discovered through an AI map they created to find new targets in a long-challenging therapeutic area.

Genentech has exercised the collaboration’s first validated target option after accepting from Recursion a validation package for the target. That acceptance will trigger a $3 million milestone payment from the biopharma giants to Recursion. That latest payment raises to $216 million the total cash paid out by the biopharma giants to Recursion since the AI-based drug developer joined with the pharma giant and its subsidiary to launch their partnership in 2021.

For each program developed, Recursion can receive up to $300 million in payments tied to achieving development, commercialization, and net sales milestones, as well as tiered royalties up to high single digits per small molecule program. Roche and Genentech have committed to using Recursion’s platform to advance therapies in 40 programs that include “key areas” of neuroscience and an undisclosed gastrointestinal (GI)-oncology indication.

The companies are not disclosing what the target is, or what neuroscience disorders it has the potential to treat. Recursion does say, however, that it took the companies 15 months to go from initiation of target validation to a validation package.

The target’s significance, according to Recursion and its partners, rests in its potential to expand the universe of neuroscience treatments beyond the handful of familiar targets in the space.

While more than three billion people are affected by neurological conditions worldwide, only one in 40 neuroscience drugs to ever reach the clinic have been approved. Just 8.4% of neurology drug candidates that enter Phase I studies reach all the way to approval, according to a Clinical Development Success Rates 2006-2015, a study published by the Biotechnology Innovation Organization (BIO), BioMedTracker, and Amplion (acquired in 2022 by Science and Medicine Group or SMG).

“Finding new targets in neuroscience has historically been challenging, and this milestone highlights our ability to uncover novel biology in areas where conventional approaches have struggled,” Recursion CEO Najat Khan, PhD, said in a statement.

New tools

New hope for discovering and developing new neuroscience drugs, the companies say, is a result of new tools developed to study the mostly unexplored genome in living neurons.

Those new tools start with Recursion’s Data Factory, an end-to-end platform developed and perfected for more than a decade to create a repeatable, scalable system for generating biological data designed for AI models.

“We really took an approach to be unbiased in our full genome-wide scan of these opportunities to uncover new biology,” Christopher Winrow, PhD, Recursion’s vice president of neuroscience, told GEN.

Partnering with Roche and Genentech, Recursion built the first whole-genome CRISPR knockout map generated from a subset of over one trillion internally manufactured neuronal cells derived from induced pluripotent stem cells (iPSCs)—about 12 brains’ worth of neurons—after outside cell manufacturers told the partners that the scale of production they were seeking was too difficult and too expensive to carry out.

“The cell context is important. We’re starting with human iPSCs and driving these into a very clearly homogenous population of neurons that we can test—and we do this at scale,” Winrow said. “You need a certain differentiation period for the iPSCs to form into the neurons that you want to study. There’s also a certain QC [quality control] that you need to do to make sure that those neurons are what we expect them to be, and that that’s robust and reproducible.”

Recursion subjected the potential targets they identified to what the company said was a rigorous validation process developed jointly with Genentech. Candidate targets advanced through successive stages of pathway validation, functional validation, and disease validation to determine whether modulating the target altered neurological disease phenotype. Only targets that consistently showed compelling evidence across

each stage advanced into a validation package.

Researchers from Recursion and Genentech applied a large-scale perturbation set using whole-genome CRISPR-Cas9 knockouts spanning more than 17,000 genes and thousands of small molecules. “You look for what we would call gene-compound interactions, to uncover some of those insights, at the same time as looking at the gene-gene interactions,” Winrow said.

“We’re looking at whole genome-wide knockout, not just a handful of areas or pathways of interest. That’s really a big game changer, in that we have this broad view,” he added. “As we perturb the cells, we start to see the known actors. But more importantly, we’re starting to see these unexplored areas really come to light. So that’s what’s changed, taking that broad view at a genome-wide level with the scale and throughput that we have at Recursion.”

46M+ images

With the cells produced and perturbations added in, the next step entails imaging designed to capture more than 46 million cellular images. Each image is analyzed across hundreds of features such as mitochondrial shape, nuclear morphology, using AI foundational models.

“What even is more mind-blowing beyond that is there are multiple features within each of those images,” Winrow said. “Traditionally as a scientist, I’d go in and I’m really interested in mitochondria. So, I look at the image and I say ‘Wow, am I seeing mitochondrial fragmentation?’ Well, that’s great. Yes, I am, right?’ But if you’re looking at hundreds and hundreds of different features, that could be a mitochondrial shape, it could be nuclear capacity, it could be a whole bunch of different things that the AI models are trained upon, that then enables a real richness to come out of that dataset.”

“So, it’s not just the scale of those images, it’s actually what’s within those images that we require the AI approaches to really uncover,” Winrow added.

The resulting data is analyzed to identify patterns resembling disease biology using Recursion’s BioHive-2 supercomputer, completed in 2024 based on Nvidia technology and proprietary AI models. Those insights are then turned into hypotheses that govern further research and ultimately, validated targets.

“Each target is individual. They are related to a core disease process,” Winrow said. “We have these core areas of biology that are looked at by a lot of different groups. We want to understand the unexplored biology around those areas, and that can be anything. It’s an unbiased approach, so all of those things that are associated have not necessarily been connected to this target in the past, so you come up with all sorts of different targets.

“Validation, then, is central to that core biology in exploring a whole bunch of different new intersecting targets,” he added.

Next steps

Next steps for the partners include advancing the discovery program from a target into a drug, using Recursion’s chemistry platform to design a potential first-in-class molecule—as well as identifying and validating additional targets for potential new programs.

Recursion said it will continue to combine its phenomics dataset with Genentech’s proprietary transcriptomics data to build additional multimodal maps designed to explore potential novel targets and pathways by systematically linking gene perturbations to cellular phenotypes.

The neuronal map is one of two whole genome neuroscience phenomaps produced by the Recursion-Roche-Genentech partnership. The other is a map of specialized microglial immune cells unveiled last year, which the companies also plan to use toward revealing significant new targets in neurodegenerative diseases.

“This provides us really two distinct but CNS-focused maps that are rooted in human biology. I think the opportunities are there to really mine both of those. The neuro map is a little bit ahead, and so we’ve been able to take some of the learnings that we gained there and apply that to our work on the microglia side as well.”

The two neuro-focused maps, in turn, are among six total whole genome phenomaps produced by the companies; the other four are designed to discover targets for a single undisclosed indication in GI oncology. The maps are designed to allow Recursion, Roche, and Genentech to explore potential undiscovered targets and pathways, systematically linking gene perturbations to cellular phenotypes.

“Because we can reuse these maps again and again, I might find something in the microglia map that’s really intriguing that I can now follow up in in the neuron map, and vice versa,” Winrow commented. “I think that’s a real power of this approach as well.”

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Fur Real: Biotech Startup Eliminates Major Dog Allergen in Beagle Pups Using CRISPR

Edusehat — Wed, 05 Aug 2026 20:35:09 +0700

Researchers at Kindred Companion Sciences, a New York-based start-up company, have successfully used CRISPR-Cas9 gene editing to breed dogs lacking the primary protein responsible for human dog allergies.

The study, published online in The CRISPR Journal (a sister journal to GEN), details the engineering and birth of a pair of healthy beagle puppies, Alfie and Bailey. The puppies represent a key first step towards addressing canine allergenicity and mark potentially a significant advance in veterinary biotechnology. The CRISPR Journal article is entitled: “Targeted Genetic Knockout of Can f 1, the Major Allergen in Dogs.”

About one in seven people (15 percent of the world population) experience allergies to dogs, resulting in allergic rhinitis and asthma. Among them is Canadian geneticist Matt Walker, PhD, CEO of Kindred Companion Sciences and lead author of the new study.

Matt Walker, PhD, top dog at Kindred Companion Sciences

“I’ve been allergic to dogs my whole life,” Walker told GEN in an interview. Proximity to dogs results in skin rashes, sneezing, and itchy eyes. The symptoms were so bad that Walker’s family could not own a dog until he left for university, when it finally got a goldendoodle. Although the breed is generally considered hypoallergenic, Walker still suffered allergic reactions when he returned home.

Years later, Walker was working on gene editing in the Columbia University lab of the late Nobel laureate, Martin Chalfie, PhD, when the family dog died. “That made me question this imprecise and ineffective way that we breed dogs for certain traits,” Walker recalled. “I wondered if we could more directly address the problem of allergies by targeting the allergen at its biological source.”

Targeting Can f 1

The chief allergen in dogs is a small lipocalin protein called Can f 1. The protein is secreted from the tongue tissue into saliva as well as some glands in the skin, where it’s deposited into dander. Researchers had previously shown that knocking out the homologous gene in mice had no detrimental effect on the health and viability of the animals. That gave Walker and colleagues confidence that the gene could be safely knocked out in dogs.

Working with canine primary fibroblasts, Walker used CRISPR to introduce a single-base insertion in exon 1 of the Can f 1 gene. The resulting frameshift mutation disrupted production of the corresponding protein. (The team performed whole-genome sequencing to establish that no off-target mutations or large-scale chromosomal rearrangements occurred.)

“We didn’t introduce any foreign DNA,” Walker said. “This type of genetic change occurs naturally in dogs. All we did was direct it at this specific site.”

Those gene-edited cells were next used as nuclear donors for somatic cell nuclear transfer. The resulting embryos were transferred to a surrogate beagle, leading to the healthy births of Alfie and Bailey, genetically identical twins, in September 2024.

Western blot analysis of saliva and dander extracts from the two pups failed to detect any residual Can f 1 protein, while high levels were present in poodle and goldendoodle controls. Walker’s group also performed skin prick testing: while the sensitized subject reacted strongly to extracts from wild-type beagles and poodles, there was no response to extracts from Alfie or Bailey.

“When I had no reaction to Bailey after she came home with us, my immediate reaction was skepticism,” Walker recalled. “What if my allergies have just gone away? What if I’m not allergic to puppies? So, I called some friends in Brooklyn and spent some time with their dog and confirmed that my allergies were still roaring.”

In the paper, the authors write: “These findings demonstrate that targeted genetic knockout of the major dog allergen is compatible with canine development and can abolish the IgE-mediated allergic response, supporting the feasibility of a gene-based approach to reducing canine allergenicity.”

A key concern in animal bioengineering is whether the removal of a physiologically relevant protein will impair the animal’s health. However, Alfie and Bailey, now almost two years old, have shown normal physical development, steady growth, and no apparent health or behavioral abnormalities. The study supports the notion that Can f 1 is biologically non-essential for the host dog, despite its high immunological impact on humans.

Interestingly, the Can f 1 allergen in dogs is unrelated to the major allergen found in cats. Following a 2022 report in The CRISPR Journal authored by Nicole Brackett and colleagues that demonstrated the feasibility of using CRISPR to edit the gene encoding the Fel d 1 allergen, researchers in South Korea engineered the first gene-edited hypoallergenic cats in 2024.

Kindred spirit

Kindred Companion Sciences was originally named Can9 Bioengineering, but Walker told GEN that the name—a play on both ‘canine’ and Cas9—proved too hard for people to pronounce. Walker says that starting a company is not unlike finishing a PhD, which he did at Columbia last year. “Both of these things come down to solving problems in imaginative ways and I definitely learned how to do that during grad school!”

While Bailey, adopted by Walker, enjoys life on the Upper West Side of Manhattan, Alfie lives with Kindred Companion’s other co-founder, Nick Gavin, in Florida. Walker and Gavin originally met when they were students at Harvard; Walker is the sole employee of the company. He makes use of “an awesome co-working wet lab facility in Harlem” near Columbia, where numerous young startups share wet lab space. “We each have our own small bench. It’s pretty scrappy, but it’s a wonderful community.”

Walker and his colleagues note in the paper that their research represents a “conceptual departure from conventional allergy therapeutics that primarily act by modulating the patient’s immune response.” Walker hopes to establish a genetic platform for future efforts that could target a number of secondary dog allergens, potentially opening the door for a new generation of allergy-safe companion and service animals.

“We’re going to be tackling other breeds and hopefully applying our work to service animals. We’re going through the regulatory process,” he said. Genome-edited organisms are regulated by the U.S. Food and Drug Administration, as Walker explains it, “to make sure the health and safety of the animals are not affected.” Eventually, Walker hopes to breed hypoallergenic animals for several dog breeds that are not traditionally hypoallergenic.

I asked Walker via Zoom how it feels to finally have a dog curled up sleeping at his feet. “It’s amazing! She’s totally changed my life!”

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Onshoring Is Here. The Hiring Wave Is Still a Decade Out

Edusehat — Wed, 05 Aug 2026 13:25:09 +0700

Guest Commentary

Since 2025, pharmaceutical companies have committed more than $370 billion to build manufacturing in the United States—a response to the threat of steep tariffs on imported drugs and to hard lessons about supply chains. If you have been watching for a hiring surge to match the headlines, you have not seen one.

That is not a sign the boom stalled. It is a sign of where we are in the cycle.

The cranes are up, but the scientists, quality specialists, and process engineers who will run these plants are still a year or two from being hired. The full workforce impact will not spike overnight, but be built steadily over the next decade, and the companies that plan for it now will be the ones ready when it arrives.

Where we are now: The construction phase

Across major life sciences hubs—Indiana, Virginia, North Carolina, Texas and others—large-scale pharmaceutical plants are under construction. Eli Lilly alone has committed to four new U.S. sites, one of them in Virginia, where Merck & Co. has broken ground on a $3 billion facility. That means demand right now is concentrated in skilled trades, project engineers, and project managers. The scientific process engineers, quality specialists, and regulatory professionals who will ultimately staff these plants come later.

This is a phased approach. Building a highly technical, highly regulated pharmaceutical facility takes years, and we are in the part of the cycle where the work is physical, not scientific. Companies and their talent partners need to know exactly where they sit in that timeline and plan against it.

While permanent facilities are years from operation, companies cannot afford to pause. Supply chain security was the original driver of onshoring. The COVID-19 pandemic exposed how dependent U.S. drug production had become on overseas raw materials and capacity, and the lesson stuck.

Contract development and manufacturing organizations (CDMOs) are filling the gap today. With U.S.-based infrastructure, biosafety labs and scalable capacity already in place, they let companies meet supply chain requirements now, without waiting for their own plants to come online. For the interim period, CDMO partnerships are a strategic bridge, not a stopgap.

The longer tail: How onshoring reshapes hiring

When the new plants do open, the workforce they need will look different from what many expect. The era of large, labor-intensive manufacturing floors is giving way to automation, AI integration and what the industry calls “lights-out manufacturing.” The demand will land in technical roles: automation engineers, process engineers and regulatory specialists. Where traditional production lines remain, the goal is to elevate workers into more sophisticated roles, not to hire by the thousands.

Regulation deserves particular weight. Life sciences is a uniquely regulated environment. A drug must meet the same safety and efficacy standards whether it is made in New Jersey or abroad. As companies reshore, they face a dual challenge: deploying advanced automation while maintaining the compliance infrastructure that protects patients. Unlike chip plants or other sectors going through similar automation shifts, a failure in pharmaceutical manufacturing can directly harm the people who depend on the product.

The competition for this talent will not happen in a vacuum. Defense contractors, chip makers and biotech firms all want the same automation engineers. Life sciences employers will have to look beyond their usual talent pools, recruit from other regulated industries and invest in workforce development that moves current employees into more technical roles.

What could slow this down

Several factors could delay or derail these projects. Cost is the most immediate. U.S. labor is structurally more expensive than the markets companies are leaving, and while automation offsets that over time, the upfront capital is enormous—and it is being spent while existing operations still run.

Policy is the variable to watch. In April 2026, the administration imposed Section 232 tariffs of up to 100% on patented pharmaceutical imports, phasing in over the second half of the year. Crucially, companies with manufacturing plans approved by the Commerce Department pay a far lower rate while they build—a direct financial reward for onshoring, but one that depends on regulatory guidance still taking shape. The rules around AI in regulated manufacturing are evolving too.

Talent itself could become the bottleneck. If the competition for automation engineers outruns the training pipelines meant to supply them, companies may find they cannot staff the plants they have built. And timing is the subtlest risk of all: companies that run too lean during construction—cutting costs while waiting for demand—risk losing the institutional knowledge they will need when the ramp-up comes.

The case for aligning now

The companies best positioned when onshored manufacturing comes online are the ones building strategic partnerships today.

That takes patience and honesty about timelines. The money is committed and the buildings are going up. What has not arrived is the workforce, and it will not arrive all at once.

The opportunity ahead—in oncology, in GLP-1 therapies, in personalized medicine—is real. Getting there means building not just the facilities, but the people power to run them.

Luke Moran is the executive director of life sciences at Actalent, a global leader in engineering and sciences services and talent solutions.

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Beyond the Technology: AI Readiness in Regulated Laboratories

Edusehat — Wed, 05 Aug 2026 02:30:11 +0700

Artificial intelligence readiness involves much more than upgrading technology. For laboratories operating in regulated environments, it requires connected workflows, trusted data, effective human oversight, and compliance built into systems from the outset. Fragmented processes and disconnected platforms can undermine AI performance while creating challenges around governance, auditability, and regulatory compliance.

In this GEN podcast, an expert from LabVantage explores how regulated laboratories can build this foundation, break down data silos, and prepare for AI adoption in clinical and commercial settings. The discussion examines the technological, operational, and governance practices needed to move AI from experimentation into regulated laboratory workflows.

Podcast Guest:

Matthew Grulke
Director of Manufacturing,
Chief Technology Officer
LabVantage Solutions

Produced with support from:

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AI Uses MRIs to Generate Brain Aging Maps for Neurodegenerative Disease Research

Edusehat — Wed, 05 Aug 2026 02:30:09 +0700

Researchers at the University of Southern California have developed an approach that uses artificial intelligence to generate detailed maps that highlight differences in how distinct parts of the brain age. The researchers, led by associate professor Andrei Irimia, PhD, at the USC Leonard Davis School of Gerontology, used magnetic resonance imaging (MRI) from nearly 15,000 cognitively healthy individuals to train a deep learning AI model. The data provided a baseline against which the model could measure local brain age (LBA), or how old specific regions of the brain appear.

While most studies of brain age measure this phenomenon using a single number, the new model provides a much richer picture of typical aging and neurodegeneration. Rather than assigning a single “brain age” (BA) to an individual, the approach generates a detailed map showing how old different parts of the brain appear relative to what is typical for someone of the same chronological age.

When the AI model was then used to analyze MRI images from people with mild cognitive impairment and Alzheimer’s disease (AD), it revealed distinct patterns of accelerated aging in brain regions known to be affected early in neurodegeneration.

“Not all brain regions age at the same rate,” Irimia said. “Some areas appear to be more resilient, while others are more vulnerable to aging and disease. By measuring local brain aging, we can identify where the brain is aging faster than expected and how those changes relate to cognitive function.”

In their in paper in PNAS, titled “Deep learning maps local brain aging in relation to cognition across human adulthood,” senior author Irimia and colleagues stated, “By providing spatially resolved measures of brain aging, this work enables more precise investigation of how neuroanatomic alterations and cognitive impairment affect brain anatomy, above and beyond global brain age measures.”

Aging is a prominent risk factor for the onset of brain diseases, including Alzheimer’s disease and related dementias, the authors wrote. “One of the most prominent biological features of brain aging is atrophy, i.e., brain volume decrease that often involves loss of brain cells and neural connectivity.”

The newly reported research builds on previous efforts to estimate BA, an emerging neuroimaging biomarker that compares a person’s brain structure to patterns seen in healthy people across the lifespan. But while human brain aging is not uniform across cortical regions, traditional methods typically reduce the brain to a single age estimate, which can obscure important regional differences. The new approach instead measures local brain age at the voxel level—the three-dimensional units that make up an MRI scan—producing a much more detailed picture of structural aging throughout the brain.

“This more nuanced understanding of how the brain ages could pave the way for earlier identification of dementia, a better understanding of what factors affect risk and new ideas for treatment approaches,” Irimia said.

To develop the model, the researchers trained a deep-learning neural network using MRI scans from 14,748 cognitively normal adults ages 19 years to 100 years, drawn from six large public datasets, including the UK Biobank, the Human Connectome Project and the Alzheimer’s Disease Neuroimaging Initiative. The team then tested the model using MRI scans from more than 1,900 additional participants in the Alzheimer’s Disease Neuroimaging Initiative, including cognitively normal adults, people with mild cognitive impairment and people with Alzheimer’s disease.

Across healthy adults, the model consistently found that the frontal and temporal lobes—regions involved in decision-making, memory and other higher cognitive functions—appeared biologically older than the parietal and occipital regions, which are involved in spatial awareness and sensory processing functions. “Our approach consistently reveals spatial patterns of aging, including relatively advanced aging in frontal and temporal regions, across both typical aging and Alzheimer’s disease,” the investigators noted. The researchers also found that the brain’s right hemisphere tended to show slightly more advanced aging than the left, a pattern that persisted regardless of whether participants were right- or left-handed.

As cognitive impairment progressed, the differences became even more pronounced. Compared with cognitively normal adults, participants with mild cognitive impairment (MCI) or Alzheimer’s disease showed significantly older local brain ages in structures that are among the first affected by Alzheimer’s pathology, including the hippocampus, amygdala and several deep brain regions involved in memory and cognitive processing.

The researchers also found that older local brain age was associated with poorer performance on cognitive assessments, strengthening the link between structural brain changes and real-world function. “Deviations from normative regional aging are significantly associated with cognitive performance supported by neural processes linked to those regions … thereby relating anatomic aging to functional outcomes,” they stated. The strongest relationships appeared in people with Alzheimer’s disease, suggesting that regional brain aging may become increasingly informative as neurodegeneration advances.

Because the model produces anatomically detailed maps, it could eventually help scientists better understand why some people experience faster decline in specific cognitive abilities than others. The approach may also prove useful for tracking disease progression or evaluating whether experimental therapies are slowing degeneration in targeted brain regions. “By providing spatially resolved measures of brain aging, this work enables more precise investigation of how neuroanatomic alterations and cognitive impairment affect brain anatomy, above and beyond global brain age measures,” they commented.

Although the findings are promising, Irimia emphasized that the method remains a research tool. The model was trained primarily on research-quality MRI data and will require additional validation using more diverse clinical datasets before it can be adopted in routine patient care. The study also relied largely on cross-sectional data, meaning that future longitudinal studies will be needed to determine whether local brain aging can reliably predict who will progress from healthy aging to mild cognitive impairment or Alzheimer’s disease.

Still, the researchers believe that moving beyond a single measure of brain age represents an important advance for neuroscience. “By quantifying the anatomy of brain aging and aligning it with cognition and disease stage, this work establishes a foundation for mechanistic inquiry and personalized intervention in neurodegeneration,” the authors stated. “This scalable framework paves the way for monitoring a broad spectrum of neurodegenerative and aging related disorders,” Irimia added, “Brain aging isn’t uniform. “By understanding how individual regions age, as well as how those patterns differ from person to person, we’re moving toward a much more precise understanding of healthy aging and neurodegenerative disease. Ultimately, that could help us identify people at risk earlier and develop more personalized approaches to preserving brain health.”

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NSF-Funded Test Bed Lets Researchers Program Automated Biomanufacturing Workflows

Edusehat — Tue, 04 Aug 2026 22:55:09 +0700

The U.S. National Science Foundation (NSF) awarded the University of Maryland in College Park $17.3 million to launch a test bed for users from across the U.S. to program automated workflows for experiments in biomanufacturing.

UMD’s Collaborative for the Realization of Autonomous Biomanufacturing (CRAB) Lab is one of 20 Programmable Cloud Laboratory Node sites supported through a $400 million NSF investment. The network’s goal is to create AI-enabled laboratories that test, scale, and demonstrate novel methods and tools for automated science, engineering discoveries, and translation.

“Being selected for this award reflects UMD’s commitment to solving real biomanufacturing challenges,” said William Bentley, PhD, Robert E. Fischell Distinguished Professor, director of the Robert E. Fischell Institute for Biomedical Devices and CRAB Lab principal investigator. “What excites me most about the CRAB Lab is that it’s not just conducting research. It’s contributing to a national resource where researchers across academia, industry, and government will be able to access experiment data and automation capabilities that would otherwise be out of reach.”

Provides datasets for training AI models

Biomanufacturing faces a critical need for real-time data processing. Traditional molecular measurement processes are too slow to train AI models, limiting the potential for experiment optimization and scaling. The CRAB Lab will incorporate an electronic measurement tool, developed at the Fischell Institute in partnership with the National Institute of Standards and Technology (NIST) and the FDA.

The tool allows for high-velocity, high-volume measurements to complement molecular data, providing comprehensive datasets for training AI models. With this information, models will be able to identify new biological vital signs—key combinations of measurements that correlate to product quality, titer and other critical process attributes—to optimize biomanufacturing processes for scale, according to Bentley, who is also appointed in UMD’s Fischell Department of Bioengineering and Institute for Bioscience and Biotechnology Research (IBBR).

The CRAB Lab builds on a long-standing partnership between UMD and NIST through IBBR, which will serve as the physical home for instrumentation, facilities and scientific expertise. The lab plans to integrate UMD’s AI and biosensor technology proficiency with industrial-scale biomanufacturing expertise from Ginkgo Bioworks.

The CRAB Lab is also aligned with the recently initiated Center for Biomeasurement and Biomanufacturing Innovation, a $33 million collaboration between UMD, UMD-Baltimore, and NIST. It is designed to accelerate the translation of new technologies to industry, to “increase the pace and lessen the costs of developing the next generation of biotherapeutics,” noted IBBR director Jonathan Dinman, PhD, a professor of cell biology and molecular genetics at UMD.

The CRAB Lab will initially serve 30 biopharmaceutical companies addressing industry-wide challenges through the Advanced Mammalian Biomanufacturing Innovation Center, an NSF Industry-University Cooperative Research Center.

“AI paired with autonomous labs is how discoveries get made and scaled at the same time,” pointe out Jason Kelly, PhD, co-founder and CEO of Ginkgo Bioworks. “The CRAB Lab gives researchers and manufacturers a shared, real-time feedback loop that could move biomanufacturing innovation from years to months.”

In addition to accelerating research, the CRAB Lab will establish on-site and remote educational programs through a dedicated training hub at The Universities at Shady Grove. These initiatives are intended to develop the workforce required for high-demand jobs in biomanufacturing.

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Genome Mapping Reveals Autoimmune Disease Risk Genes in Innate Lymphoid Cells

Edusehat — Tue, 04 Aug 2026 19:20:08 +0700

A new study published in Nature Genetics suggests that looking beyond the nearest gene may be essential for understanding how immune disease risk variants act in rare immune cells.

The paper, “High-resolution promoter interaction analysis implicates genes involved in activation of type 3 innate lymphoid cells in immune disease risk,” was co-led by researchers at Cincinnati Children’s Hospital, the MRC Laboratory of Medical Sciences, Imperial College London, along with collaborators. The team mapped long-distance DNA interactions in type 3 innate lymphoid cells, or ILC3s, a rare population of tissue-resident immune cells enriched in the gut, airways, and mucosal lymphoid tissues.

ILC3s help regulate inflammation and maintain barrier integrity, but their rarity has made them difficult to study with conventional genome-organization methods. Many approaches for mapping chromosomal contacts require millions of cells, limiting their use in cell types that may be particularly relevant to disease.

“This work opens the door to studying long-distance DNA interactions in rare immune cells,” says Stephen Waggoner, PhD, scientist in the Center of Autoimmune Genomics and Etiology at Cincinnati Children’s. “Until now, most methods required millions of cells, which limited what we could learn from the cell types most relevant to disease.”

To address that limitation, the investigators used a low-input, high-resolution Promoter Capture Hi-C (PCHi-C) approach to map promoter-anchored chromosomal contacts in primary human ILC3s, alongside CD4+ T cells. They then combined those maps with genome-wide association study data using a Bayesian framework, multiCOGS, to connect Crohn’s disease risk variants with the genes they are most likely to regulate.

Researchers mapped long-range DNA interactions in rare tonsil-derived ILC3 immune cells to identify regulatory mechanisms linked to autoimmune disease risk. [Cincinnati Children’s]

The analysis linked Crohn’s disease risk variants to more than 100 candidate genes in ILC3s, including both known inflammatory bowel disease genes and less expected candidates. Among the latter was CLN3, a gene best known for its role in Batten disease, a rare neurodegenerative disorder.

“While some disease risk variants act on the genes nearest to them, others do not, so if we only look at the nearest gene, we may get the underlying mechanisms wrong,” says Mikhail Spivakov, PhD, head of the Functional Gene Control Research Group at MRC Laboratory of Medical Sciences. “What is more, the patterns of genome folding differ across cell types, so it is important to study the 3D connections between variants and the genes they control in the cells that are relevant for the disease.”

Follow-up experiments in a mouse ILC3-like cell line supported a possible role for CLN3 in regulating inflammatory activity. According to the paper, CLN3 was downregulated after cytokine stimulation, while increasing CLN3 expression altered stimulation-induced transcriptional programs and cytokine secretion. The findings do not establish CLN3 as a causal gene in Crohn’s disease, but they point to a potential immune-related function for a gene more commonly discussed in the context of neurodevelopmental disease.

The researchers also extended the approach to five additional autoimmune conditions, generating a catalog of ILC3-linked risk genes. These genes were enriched for regulators of the ILC3 inflammatory response identified in a CRISPR interference screen.

The next steps appear to include clarifying how CLN3 influences immune-cell function, testing whether the pathways identified in ILC3s can help explain disease mechanisms, and applying the low-input mapping strategy to other rare cell types that have been difficult to study. “Studying genetic regulation in rare cell types allows us to move closer to mechanism, not just association, and that’s essential for making genetic findings meaningful across medicine,” says Waggoner.

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Combined Phage Therapy and FMT Reduces Recurrent UTIs and Antibiotic Use in First Human Case Series

Edusehat — Tue, 04 Aug 2026 04:45:15 +0700

Recurrent urinary tract infections (rUTIs) are among the most common bacterial infections worldwide. The difficult-to-treat condition, which affects primarily women, is defined as two urinary tract infections (UTIs) within six months or three UTIs within the past year. They are a leading cause of outpatient antibiotic use, accounting for more than 15% of all prescriptions.

While antibiotics remain the standard treatment, frequent recurrences and rising antibiotic resistance highlight the need for alternative therapeutic approaches. Now, researchers have, for the first time, administered a combined phage therapy (PT) with fecal microbiota transplantation (FMT), to decolonize urinary and intestinal reservoirs of rUTI patients.

The novel treatment approach, combining PT and elective FMT, was administered to three female patients, between May and July 2023, with rUTI that did not respond to antibiotics and commonly used non-antibiotic strategies. All had microbiologically confirmed E. coli in multiple infections.

All three women received PT orally and intravesically (locally applied to the bladder via catheter) for eight days outside of acute episodes. Two of these three patients were elected to receive subsequent FMT. Since the bacteria causing the recurring infections often reside in the gut as well as the urinary tract, the FMT treatment targeted the intestinal reservoirs of E. coli that survive antibiotic treatment of acute infections and can become increasingly resistant.

“While phage therapy acts to remove the pathogen, FMT aims to restore a healthy microbiome, combining both immediate and long-term effects,” Lena Biehl, MD, PhD, group leader at Fraunhofer ITMP, deputy lead of Cologne Microbiota Bank, University Hospital Cologne.

This work is published in Nature Microbiology in the paper, “Combined Phage therapy and fecal microbiota transplantation to treat recurrent urinary tract infection: a case series.”

The treatments were well tolerated and did not result in noticeable side effects. The two patients who received the combination therapy have experienced a long-term reduction of UTIs over the course of two years, while the one patient with PT only has experienced further episodes but with reduced symptoms. For all patients, although E. coli was detected in follow-up samples, quality of life improved significantly, while the need for antibiotic treatment was substantially reduced.

“Three patients are not a sufficiently large sample to establish this new therapy, and control groups were lacking. However, this experience has helped to lay the foundation for a clinical trial in order to test the clinical utility of this treatment approach and make it more widely available and sustainable for patients,” comments Shawna McCallin, MD, at the Balgrist University Hospital in Zurich.

The clinical trial is scheduled to begin in June 2027 at the participating institutions as part of the REPhRAME project, which is funded by the European Commission through the Horizon Europe funding programme.

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Evolutionarily Diverse Organisms Switch Genes on Simply and Switch Them off Dynamically

Edusehat — Tue, 04 Aug 2026 01:10:09 +0700

The signals that cells use to switch genes on have remained almost unchanged across two billion years of evolution, but the ones used to switch genes off vary dramatically from one branch of life to another, according to a new study by researchers at the Centre for Genomic Regulation (CRG), Barcelona Institute of Science and Technology (BIST).

The findings result from the broadest comparative exercise to date of how different life forms regulate their genomes. The researchers carried out the first detailed analysis of chromatin, the protein scaffold that controls how DNA is read, in several major branches of life that have been largely absent from studies to date, including lineages such as discobans, rhizarians, ichtyosporeans, and cryptomonads.

The work helps understand how genomes evolved on Earth and could have implications for medical research into diseases involving faulty gene regulation. It also delivers a new method, developed at the CRG, which can help support international efforts to characterize life on Earth at the molecular level.

“The cell’s instructions for activating genes are essentially the same in a human, a sea anemone and a soil amoeba,” said Arnau Sebé-Pedrós, PhD, ICREA Research Professor and senior author of the team’s published paper in Nature Genetics. “But the instructions for silencing genes and other genomic elements like transposons have been continuously evolving since our last common eukaryotic ancestor. Different branches of life have developed different molecular toolkits to do the same thing.” The team’s report is titled “Diversity and evolution of chromatin regulatory states across eukaryotes.”

DNA is wrapped, inside every cell, around proteins called histones. Small chemical tags attached to these proteins tell the cell which stretches of DNA to read and which to ignore. The chemical tags are ancient, dating back roughly two billion years to a single-celled organism known as the last eukaryotic common ancestor, or LECA, the founder of all complex cellular life, from which every plant, animal, fungus and protist on Earth descends. The system, known as chromatin regulation, is what allows the same genome to produce a liver cell or a neuron, and a leaf or a root. Faults in the regulation of chromatin underpin many human diseases, including cancers.

“Histone post-translational modifications (hPTMs) are central to defining functional chromatin states,” the authors explained further. “These hPTMs are conserved across diverse eukaryotes, with dozens tracing back to the last eukaryotic common ancestor, which we confirmed by histone mass spectrometry.”

The enzymes that add and remove the tags are also broadly shared across plants, animals, fungi and microbial eukaryotes. Until now, however, almost all detailed knowledge of how these tags work has come from a handful of laboratory species such as humans, mice, fruit flies, yeast and the model plant Arabidopsis. The vast majority of life’s diversity has remained unexplored at this level.

The authors’ project began in 2017, when Sebé-Pedrós and David Lara-Astiaso, PhD, were using a technique called iChIP to study chromatin in comb jellies and placozoans, animals not traditionally studied in the lab. The researchers wondered whether the approach could be scaled up for use in other species in the eukaryotic tree of life.

“We wanted to map epigenetic states in scarce cell types in mice and humans,” recalls Lara-Astiaso, now at the Arc Institute in California. “Eventually, we managed to transform that precursor into a general method for mapping genome regulation across the tree of life—more streamlined, more sensitive, and finally able to handle the particularities of very different species.”

The new method, iChIP2 can label chromatin from many species with unique molecular barcodes and read them all in a single experiment. Using the technology helped profile twelve chemical tags, or histone modifications, across twelve phylogenetically diverse species, spanning amoebae, fungi, plants, algae, single-celled predators and animals.

Some organisms had never had their chromatin mapped before. “We initially hoped to build a completely universal protocol, but species differ too much for that,” noted co-first author Cristina Navarrete, PhD. Plants and algae have cell walls that require specialized preparation, for example. Once a lab has extracted chromatin from their favorite species, iChIP2 takes over robustly, and from very small amounts of material.”

The researchers found that the signature of an active gene, marked by the pattern of histone modifications clustered around its start and along its body, was nearly identical in every species the team examined. The signature of a silenced gene was not. The results indicated that different lineages used different combinations of modifications, in different patterns, to keep stretches of DNA silent. “Our analyses revealed highly conserved euchromatin states at active gene promoters and gene bodies,” the investigators stated. “In contrast, we observed diverse configurations of repressive heterochromatin states associated with silenced genes and transposable elements …”

In some species, one modification silenced transposable elements while a different tag silenced unused genes. In others, the same modifications piled up together on the same regions. In the soil amoeba Acanthamoeba, a chemical mark that signals gene activation in animals had been repurposed to switch genes off.

“We’ve established so many new rules from looking at such few species,” said study co-author Sean Montgomery, PhD, “It’s the power of looking at non-model organisms to see how evolution has brought about many differing solutions to the same problems.”

The researchers suggest the diversity reflects an ancient and ongoing conflict between genomes and the parasitic DNA within them, like transposable elements, also known as “jumping genes,” and endogenized viruses. Every genome carries within it stretches of jumping genes, sequences that copy and paste themselves into new locations, sometimes harmlessly, sometimes destructively. In a human genome, they account for roughly half of all DNA. In their paper the team wrote, “The diversity of repressive states across eukaryotes, compared with the highly conserved active states, reflects the history of genomic invasions by parasitic elements in different lineages and could also define the permissiveness of these genomes to future invasions.”

Keeping jumping genes silenced is a matter of survival, but they evolve. Their parasitic nature means they acquire new sequences and sometimes even fragments of the chromatin machinery itself to evade detection.

“If a species loses its repressive mechanisms completely, it can’t tolerate parasitic elements like transposable elements or endogenized viruses. The result is that it’s no longer there. It’s dead,” says Sebé-Pedrós.

Over hundreds of millions of years, the result is host and parasite adapting and a tree of life on which each branch has developed its own bespoke strategy to silence genes. Some of those strategies, the team suggests, were later borrowed for other purposes.

The work lands at an important moment for comparative genomics. International efforts such as the Earth BioGenome Project and the Wellcome Sanger Institute’s Tree of Life programme, with which Sebé-Pedrós is affiliated, are sequencing the genomes of life on Earth at unprecedented speed.

The data generated by the initiatives offer potential new insights into how life has evolved on Earth, but a genome sequence alone says little about how the genome is used. Methods like iChIP2 make it possible to ask how life forms regulate their genomes. “… our results exemplify the potential of biodiversity epigenomic profiling,” the team suggested. “As genome sequencing is rapidly advancing across the tree of life, this approach offers a valuable opportunity to similarly expand our understanding of eukaryotic genome function and regulation.”

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Following court decision, Arkansas will not enforce Act 630 on drug distribution

Edusehat — Mon, 03 Aug 2026 21:35:03 +0700

On Tuesday July 28, the Arkansas State Board of Pharmacy suspended enforcement of a 2025 Arkansas law, Act 630, two months after a federal court deemed the measure likely unconstitutional.

Act 630 purportedly aimed to “prevent pharmaceutical manufacturers from restricting prescription medication distribution to a limited network of pharmacies, particularly out-of-state pharmacies,” requiring them to include some Arkansas pharmacies into such networks approved by the state if such networks were maintained more than three months after launch. If companies did not comply, the law would impose a $10,000 per day noncompliance penalty and potentially could result in loss of Arkansas Medicaid coverage for all of the manufacturer’s drug products.

The Board of Pharmacy emphasized that its decision was based on the opinion from a federal court that Act 630 likely violates the U.S. Constitution’s prohibition on state laws that unduly restrict interstate commerce, as well as on advice from the Arkansas Attorney General’s Office that the law is unconstitutional and could not be successfully defended in future litigation.

But the issues with the law are not restricted to its unconstitutionality or to other potential conflicts with federal law that the court noted in its opinion. Act 630 also poses significant risks to patient safety as many of the drugs and treatments distributed in a limited distribution network are the subject of an FDA-required Risk Evaluation and Mitigation Strategy (REMS) or other special handling or safety protocols.

“Therapies are getting more complex because they’re treating more complex diseases,” explains Patrick Plues, Senior Vice President, State Government Affairs & Affiliate Relations at the Biotechnology Innovation Organization (BIO). “Many drug manufacturers became very concerned about the impact of this legislation on the ability of healthcare providers to effectively distribute and deliver these therapies to patients in a safe way.”

So what are industry concerns with Act 630 when it comes to patient safety?

Impacts on small, ultra-rare, or high-risk patient populations

“The main question that legislators had when considering the bill last year was basically: If we’re licensed pharmacists, why can’t we be included in the limited distribution networks?” recalls Russell Palk, Director of Government Affairs in the Southeast and Mid-Atlantic, for BIO. “The problem is that these networks often incorporate complex patient services and monitoring—meaning that these patient populations require healthcare providers with highly concentrated expertise to safely deliver and manage these medications.”

As BIO explained in its letter to the Arkansas State Board of Pharmacy last year, “many therapies placed in limited distribution require close monitoring of patients, including frequent dosage adjustments, patient education on administration (e.g. for injectable or infused therapies), monitoring for adverse events, and adherence support.”

Not maintaining these high standards of delivery and care could critically threaten a treatment’s efficacy and achieving the treatment’s intended clinical outcomes. In particular, BIO emphasized the adverse impact of Act 630 on “patient populations requiring complex or rare disease treatments that need specialized handling.”

Furthermore, the limited distribution networks are used for the distribution of drugs that require additional safeguards to prevent misuse or improper administration. To comply with an FDA-required REMS, some drugs may only be dispensed through certified pharmacies with REMS compliance procedures, properly trained staff, and systems to collect and report REMS-required data.

And that is not the only capacity issue that traditional pharmacies would face within the limited distribution system.

“Many medications require strict temperature control, specialized packaging, or other handling protocols to maintain their stability and effectiveness,” BIO writes. “Distributing these drugs through a select network of pharmacies and distributors helps ensure that storage conditions meet necessary safety standards, preventing potential loss of efficacy or potential harm to patients.”

Protecting access

“Though it might feel a bit counterintuitive,” Palk continues, “limited distribution networks have an incredibly important role in protecting access to these complex treatments.”

When it comes to therapies for complex patient populations, it simply is not a scalable practice for traditional pharmacies to maintain access—in fact, it would be a major drive towards inventory uncertainty.

“Open access would strain inventory levels, raise risks of drug shortages, negatively impact inventory stability, and undermine business continuity and emergency response plans,” BIO writes. “Moreover, pharmacies with one or a small number of patients are unlikely to need full case quantities, yet their case orders will divert inventory from other patients in need and lead to excess carrying costs and significant product waste, as the drugs have limited shelf lives and will expire without being used.”

In reality, Act 630’s “same day access” requirement would add significant cost and inefficiency to the entire healthcare system in Arkansas.

Issues with patient access don’t just start behind the pharmacy counter, there are issues when it comes to distribution logistics as well.

For drugs and treatments that require specialized storage, delivery, and management, limited networks of delivery do more to guarantee access at a given location rather than risk it.

“Selected pharmacies have stock on hand and therefore don’t need to order upon receipt of a patient’s script,” writes BIO. “For patients who may live far from specialty medical centers or have difficulty traveling, limited distribution networks often include direct-to-patient shipping options.”

Finally, the limited distribution system also helps to prevent counterfeit or substandard products from entering the supply chain—a priority that is especially important to maintain when working with high-risk rare disease patients as expanding distribution indiscriminately increases the chances of supply chain disruptions, contamination, or improper handling.

“Ultimately, we have to make sure Arkansas considers legislation that works with patient needs in mind and complies with federal law,” says Palk. “A little extra time and consideration goes a long way as we work to increase access and lower barriers to medications for patients everywhere.”

Read: BIO’s letter to the Arkansas State Board of Pharmacy.

The post Following court decision, Arkansas will not enforce Act 630 on drug distribution appeared first on Bio.News.

BIO 2026: How biotechs break the $100M barrier in funding rounds

Edusehat — Mon, 03 Aug 2026 21:35:03 +0700

The tap is opening up for venture capital in the biotech industry.

Venture investment in biotech jumped 23% year-over-year in 2025 to $23 billion, according to BIO’s State of Emerging Biotech Report. This amounted to a thaw in a four-year period of stagnation deemed the “biotech winter” by Cognito Therapeutics CFO Steve Worthy, in a June 25 panel at the BIO International Convention. As Endpoints Correspondent Kyle LaHucik told the panel, 40 biotech companies had financing rounds above the $100 million “mega-round” threshold in the first half of 2026, up from 33 in half one last year.

That freer flow of cash has spurred excitement among emerging biotech companies, eager to close an ever-elusive mega-round. But many find it hard to stand out from their competition and make a convincing case for venture funding, said experts in the panel on Breaking the $100M Barrier: How Biotechs Closed Mega-Rounds in a Tough Market.

The panel featured three biotech company representatives, each of whom secured at least one mega-round in venture funding, who shared details on how they broke through to investors. Additionally two investment representatives came to give their side of the story, detailing how they choose which companies are worth backing.

Prioritize the right product

Kenneth Greenberg, co-founder and CEO of SonoThera, admitted that he had to make hard choices in determining which of his firm’s projects are attractive to investors. SonoThera is developing a best-in-class gene therapy for hemophilia A, which Greenberg had originally put forth as his company’s flagship project.

“But the sentiment that we got across the board from venture investors was ‘don’t do it.’ Despite the data looking quite good, the commercial risk of a hemophilia gene therapy product was too insurmountable for investors to build that conviction.”

SonoThera ultimately found success by prioritizing its bubble-based gene delivery system and closing with $125 million in series B funding.

Gather robust clinical data

The market is notoriously fickle, said Cognito CFO Worthy. He offered advice to firms struggling to navigate its peaks and troughs.

Worthy shared how the appetite of Cognito’s prospective investors oscillated wildly between enthusiasm and skepticism over the course of 2025, particularly after April’s Liberation Day tariffs. As such, the milestone the company sought to fulfill in February 2025 took until February 2026 to fulfill.

Cognito’s position was complicated by the company’s specialty in treating Alzheimer’s; “A disease rife with failure,” remarked Worthy, “with billions of dollars burned chasing hypotheses proven to be untrue.”

Worthy recognized that investors in volatile markets in a risky field want proof of viability above anything else. As such, Cognito went all-in on its data gathering, evolving its 74-patient phase 2 case study into a 673-patient phase 3. The increase in study size demonstrated not only that the company was confident in its product, but that it also had the financial competence to responsibly manage its funding. In return, they were rewarded for their meticulousness with $105 million series C funding.

Demonstrate ‘China-like efficiency’

Meanwhile, Enveda raised $550 million of capital due to its commitment to what CEO Viswa Colluru calls “China-like efficiency.” This means streamlining the research to production pipeline as much as possible: Enveda has a team of 200 scientists based in India working in conjunction with its Colorado headquarters to turn its molecules into medicine.

This commitment to efficiency is part of a larger China strategy employed by emergent biotech firms, experts said. It addresses reluctant investors’ fear of heavily backing an American biotech firm’s project, only for one of China’s companies to come out with a product that makes it obsolete.

The investor’s perspective

“Personally, I think these are kind of the golden years for public trading,” said Josh Schimmer, a Biotech Equity Research Analyst at Cantor Fitzgerald. “We’re starting to see the IPO window open back up after four years.”

He said his optimism is emblematic of the mood that seems to be infusing the greater investor community, founded on the strides in efficiency the biotech industry is making.

“This is one of the most exciting times for biotech innovation,” he continued. “We’re seeing a dramatic acceleration, and it is really breathtaking and overwhelmingly super exciting.”

Jakob Dupont, Executive Partner at Sofinnova Investments, speculated that biotech investors are looking for companies that gather all their resources on just a few promising products and backing them with robust clinical data.

Attracting investment is about easing the concerns of investors. Those who successfully close mega-rounds do this more than anyone else.

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Receptor.AI and Sethera Plan to Create Closed-Loop Discovery and Optimization Workflow for Difficult Therapeutic Targets

Edusehat — Mon, 03 Aug 2026 21:30:08 +0700

Receptor.AI and Sethera partner to create a closed-loop drug discovery workflow. Sethera will generate and experimentally screen architecture-diverse polymacrocyclic peptide libraries. Receptor.AI will apply physics-based modeling, artificial intelligence, and multiparameter optimization to interpret sequence, architecture, enrichment, and activity data; develop binding hypotheses; prioritize candidate series; and guide focused optimization cycles.

Subsequently, the companies will design, synthesize, and experimentally test new candidates, using the resulting data to inform each subsequent cycle.

Sethera’s platform installs one to six stable cross-links to generate polymacrocyclic, nested, in-line, and interpeptide structures across large, encoded libraries. Unlike constrained-peptide approaches centered on a predetermined structural motif, Sethera’s platform explores multiple experimentally accessible topologies, allowing target biology and screening data to identify the most productive molecular architecture, notes a Sethera spokesperson.

By combining this chemical space with Receptor.AI’s computational capabilities, the companies intend to advance screening-derived hits toward validated lead series with improved potency, selectivity, stability, permeability, and other developability characteristics.

The initial program will focus on a mutually selected hard-to-drug target. The companies will prospectively assess whether the integrated workflow improves hit confirmation, target selectivity, and lead optimization compared with conventional enrichment- and assay-led prioritization. Following validation of the initial workflow, Receptor.AI and Sethera intend to pursue additional internal programs and jointly structured discovery collaborations with pharmaceutical and biotechnology partners across selected target classes and therapeutic areas.

Alan Nafiiev, PhD

“Sethera’s platform creates experimentally accessible peptide architectures that conventional design approaches do not readily reach,” said Alan Nafiiev, PhD, founder and CEO of Receptor.AI. “Our objective is to use physics and AI not as a substitute for experimentation, but to learn from each experimental cycle and direct the next one. That closed feedback loop is where we believe the collaboration can create distinctive value.”

“Sethera can search enormous physical libraries and identify target-binding sequence and architecture families that cannot be generated through conventional peptide design alone,” adds Karsten Eastman, PhD, CEO and co-founder of Sethera Therapeutics. “Receptor.AI adds a powerful layer for understanding why those hits work and how they can be improved. Together, we intend to create a coordinated design-make-test-learn process that moves more efficiently from experimental discovery to validated lead series.”

Karsten Eastman, PhD

The collaboration is specifically focused on applying Receptor.AI’s computational platform to Sethera’s proprietary polymacrocyclic peptide chemistry, encoded screening data, and resulting therapeutic candidates. Each company will retain its background platform technologies, and joint programs will be conducted under coordinated research plans with defined experimental, computational, data, and program-management responsibilities.

Through the alliance, Receptor.AI and Sethera officials say they aim to establish a repeatable discovery system that continuously learns from sequence, architecture, counterselection, binding, functional, and developability data generated across experimental campaigns. The resulting workflow is intended to reduce the number of design cycles required to progress from initial screening hits to differentiated peptide lead series.

The post Receptor.AI and Sethera Plan to Create Closed-Loop Discovery and Optimization Workflow for Difficult Therapeutic Targets appeared first on GEN - Genetic Engineering and Biotechnology News.

StockWatch: Capricor Plunges as FDA Panel, Staff Question Effectiveness of Lead Candidate Deramiocel

Edusehat — Mon, 03 Aug 2026 10:40:10 +0700

After seeing its lead candidate rejected by the FDA last year, Capricor Therapeutics (Nasdaq: CAPR) is hoping for a better outcome for its resubmitted biologics license application (BLA) for its lead pipeline candidate Deramiocel, a cell therapy indicated as a treatment for cardiomyopathy in Duchenne muscular dystrophy (DMD).

That hope appeared less likely than ever as the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee on Wednesday recommended against agency approval of Deramiocel, concluding in a 9-3 vote with no abstentions that the available evidence from the Phase III HOPE-3 trial (NCT05126758) did not “provide substantial evidence of effectiveness” for Deramiocel as a treatment for cardiomyopathy in Duchenne muscular dystrophy (DMD).

The advisory committee vote is likely to influence how the FDA acts on the resubmitted BLA for Deramiocel, with the agency having set an August 22 target decision date under the Prescription Drug User Fee Act (PDUFA). The FDA typically (but not always) heeds the advice of its advisory committees or “adcomms,” which in turn typically (but not always) heed the evaluations of agency staff.

Deramiocel is an allogeneic cardiosphere-derived cell (CDC) therapy candidate. CDCs are designed to act by secreting exosomes that target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype.

According to Capricor, preclinical and clinical studies have shown Deramiocel to preserve cardiac and skeletal muscle function in muscular dystrophies such as DMD by exerting strong immunomodulatory and anti-fibrotic activity.

Negative FDA evaluation

FDA reviewers paved the road to Deramiocel’s poor reception from the adcomm on July 27 with a negative evaluation of the resubmitted BLA. Their assessment concluded that data submitted to the FDA from HOPE-3 and the earlier Phase II HOPE-2 trial (NCT03406780) “does not provide substantial evidence of effectiveness for Deramiocel in DMD”—though Capricor’s indication for Deramiocel is specifically cardiomyopathy in DMD.

The unnamed FDA staffers took issue with:

Whether Deramiocel achieved HOPE-3’s primary and secondary endpoints.
The hypersensitivity shown by 42% of Deramiocel patients vs. 15% of placebo patients;

Capricor’s failing to submit to the agency an updated statistical analysis plan (SAP) for review before it resubmitted its BLA for Deramiocel in February.

Capricor declared HOPE-3 a successful trial in December, citing as a statistically significant benefit the reported 54% slowing of skeletal muscle disease progression on the primary endpoint, Performance of the Upper Limb version 2.0 (PUL 2.0) percentage change from baseline in the 105-patient intent-to-treat (ITT) population with evaluable PUL v2.0 assessments at 12 months. Capricor also reported a 91% slowing of progression measured by left ventricular ejection fraction (LVEF) in the 83-patient ITT population with centrally reviewed and evaluable cardiac MRI LVEF assessments at 12 months.

The FDA, however, says HOPE-3 can only be deemed a success after the company made changes to its SAP that included modifications to the primary and key secondary endpoint definitions, its analytical methods; and the data imputation strategy for intercurrent events.

“Although the applicant provides justifications for these changes, FDA does not agree that the scientific rationale for those changes was supported and considers the changes unwarranted based on the study’s design, powering, and original statistical assumptions,” the FDA staffers contended.

The reviewers also alleged that the distinctive adverse event profiles seen between Deramiocel and placebo patients “raises the possibility that treatment assignment could be inferred even under formal blinding conditions.”

“This risk of functional unblinding,” they added, “was further extended by the open-label period of HOPE-3, during which additional treatment-related data accumulated and may have made treatment assignment more apparent.”

Capricor answers back

Capricor answered back the same day. CEO Linda Marbán, PhD, told Reuters she was “completely shocked at how they decided to review and analyze this data,” while the company issued a statement faulting the FDA for relying on an “obsolete” analysis: “Our results are governed by the final analysis plan, SAP version 3.0, which was finalized prior to unblinding.”

“It is critical to understand that the post-hoc analyses in the FDA’s briefing materials rely on SAP version 1.1, an unsigned incomplete internal draft which became obsolete with the addition of cohort B and did not include content specifically requested by FDA,” Capricor explained. “We believe Deramiocel offers a meaningful treatment option for boys and young men living with Duchenne, who continue to face a significant unmet medical need.”

The company sought to back up that contention on Wednesday, when it released updated data from HOPE-3 that were published in The Lancet. The updated data showed Deramiocel to have improved cardiac and skeletal muscle function in Phase I–II studies of DMD, and also found that deramiocel could slow muscle weakening in boys and young men with advanced DMD, and may also slow heart damage in those who already have heart muscle disease.

But at 12 months of follow-up, Deramiocel’s performance on the study’s key secondary endpoint of LVEF “did not reach statistical significance, although the difference [favored] Deramiocel,” researchers reported, as the Deramiocel group vs. placebo showed a least-squares mean ranked change in LVEF of 57·47 ranks compared with 45·82 for placebo.

“These findings reinforce deramiocel as a safe, effective, and promising therapy for individuals living with DMD. Longer follow-up is needed to establish durability, long-term safety, and effects on clinically important cardiac outcomes,” the research team from Capricor and its clinical partners wrote in the study.

They added: “A 54% reduction in mean skeletal-muscle disease progression over 12 months, if sustained, would be equivalent to delaying approximately 1 year of untreated progression over 2 years.”

Investors unpersuaded

Capricor’s responses during the week failed to persuade investors. They responded to the negative FDA staff briefing on Deramiocel with a sharp sell-off that sent the company’s shares nosediving 64.5% to $7.00, from $19.70 at the close of trading July 24. The decline reached 85% when Capricor shares hit a 52-week low of $2.97 early Thursday.

After the downgrades and second stock plunge of the week, however, Capricor investors began to “buy the dip” and sent the company’s shares partially rebounding to $4.19 on Thursday (a 36% one-day slide) and $3.85 at Friday’s closing bell, down 8%. Overall for the week, Capricor’s stock suffered an 80% one-week decline.

News of the FDA adcomm vote led to downgrades of Capricor stock and severe 12-month price target downgrades by at least six investment firms:

Piper Sandler (Edward Tenthoff)—From “Overweight” to “Neutral,” all but wiping out its price target 97%, from $58 to $2.
Cantor Fitzgerald (Kristen Kluska)—From “Overweight” to “Neutral,” eviscerating its price target 94%, from $62 to $3.50.
Ladenburg Thalmann (Aydin Huseynov, MD)—From “Buy” to “Neutral,” no price target announced.
Maxim Group (Jason McCarthy, PhD)—From “Buy” to “Hold,” no price target announced.
C. Wainwright (Joseph Pantginis, PhD)—From “Buy” to “Neutral,” removing its $60 price target reiterated in May.
Oppenheimer (Leland Gershell, MD, PhD)—From “Outperform” to “Perform,” removing its $54 price target reiterated in March.

“The briefing documents raise many more concerns versus what we originally were anticipating, putting Capricor in a tough situation” for the adcomm meeting, Kluska said Monday in remarks reported by Reuters.

The six firms joined three others that lowered their ratings on Capricor shares earlier in the week:

Alliance Global Partners (Matthew Venezia)—From “Buy” to “Neutral,” chopping its price target 86%, from $51 to $7 on Tuesday.
Riley Financial (Madison El-Saadi, PhD)—From “Buy” to “Neutral,” slashing its price target 84% from $63 to $10 on Monday.
Roth Capital Partners (Boobalan Pachaiyappan, PhD)—From “Buy” to “Neutral,” slicing its price target 82% from $38 to $7 on Monday.

MapLight data divides investors, analysts

Investors and the Wall Street analysts who cover their favorite companies sometimes don’t see eye to eye. That was apparent this past week when MapLight Therapeutics (Nasdaq: MPLT) shares went on something of a roller-coaster ride, as mixed clinical results for its lead drug in a mid-stage trial in schizophrenia sent the stock nosediving on investor fears—until reassurances from analysts reversed the slide and sent those shares back in the positive direction.

The up-and-down week ended with MapLight shares sliding 64%.

MapLight’s wayward week started on July 27 when the company released data from its 307-patient Phase II ZEPHYR trial (NCT07038876) assessing its lead pipeline candidate ML-007C-MA in adults with an acute exacerbation of schizophrenia. ML-007C-MA is an oral, extended-release, fixed-dose combination of the M₁/M₄ muscarinic agonist candidate ML-007, co-formulated with a peripherally acting anticholinergic.

MapLight trumpeted what it termed positive results from ZEPHYR, though the data appeared to be more mixed: On the positive side, the 210/3 mg twice-daily (BID) dose of ML-007C-MA showed statistically significant and clinically meaningful reduction in its Positive and Negative Syndrome Scale (PANSS) total score compared to placebo at Week 5 in a In the modified intent-to-treat (mITT) population, with an effect size of 0.37 and a least squares mean 4.5-point improvement vs. placebo (p=0.015).

However, the 330/6 mg once-daily (QD) dose of ML-007C-MA did not achieve statistical significance on the primary endpoint, even as it showed an effect size of 0.23 and a 2.8-point improvement over placebo (p=0.110)—as well as separation on CGI-S (p=0.036), PANSS positive Marder factor (p=0.045), and Readiness for Discharge Questionnaire (p=0.027), and numerical separation on other endpoints.

That result investors scurrying to sell off their MapLight shares, since it raised questions about whether ML-007C-MA could effectively with Cobenfy^® (xanomeline and trospium chloride), the schizophrenia drug marketed by Bristol Myers Squibb (BMS; NYSE: BMY). Cobenfy, which won FDA approval in 2024, showed larger PANSS reductions of 8.4 and 9.6 points in a pair of Phase III trials compared with placebo.

Cobenfy generated $119 million in product revenues in the first half of this year, nearly double (up 92%) from January–June 2025), in addition to $155 million during all of last year.

The BID dose also showed robust and clinically meaningful improvement in cognitive performance, based on the pre-specified secondary endpoint assessed via the Cogstate battery in participants with baseline cognitive impairment (effect size=0.51; 0.44 points vs. placebo; p=0.041). But the cognitive benefit did not show correlation with the change in PANSS score, something that MapLight said suggested that “the effect was independent of, and not secondary to, improvement in psychotic symptoms.”

“We are very encouraged by these results, which show that ML-007C-MA delivered clinically meaningful antipsychotic efficacy alongside a favorable tolerability profile designed to translate into real-world use,” Chris Kroeger, MD, MapLight’s co-founder and CEO, said in a statement.

Encouraged enough, Kroeger added, that MapLight plans to discuss a path forward for ML-007C-MA in schizophrenia, including the design of a Phase III trial, at an End-of-Phase II (EOP2) meeting with FDA officials. Data from that trial, combined with results from ZEPHYR, are intended to support an initial New Drug Application (NDA) submission for the drug.

Investors sharply disagreed with MapLight’s optimism, sending the company’s shares plummeting 73% on July 27, from $36.56 to $9.90. But several analysts questioned the wisdom of investors selling off shares on a single PANSS number.

“The PANSS score is but one component of what might drive success from a commercial point of view,” cautioned Sumant Kulkarni, a senior analyst covering biotechnology with Canaccord Genuity, wrote in a research note. “At the same time, we need to see more data from additional trials on safety and efficacy.”

That data could come, he continued, from the Phase II VISTA trial (NCT06887192) assessing ML-007C-MA as a treatment for hallucinations and delusions associated with Alzheimer’s disease psychosis, a potentially larger market for the drug.

However, Kulkarni cut Canaccord Genuity’s peak-year 2037 sales forecast for ML-007C-MA by more than half in schizophrenia, from approximately $1 billion to approximately $400 million. He also shrunk by one-third his firm’s peak sales forecast for ML-007C-MA in ADP, from $3 billion to $2 billion, and lowered ***HOW its forecast of MapLight’s operating expenses.

As a result of these changes, Kulkarni cut Canaccord Genuity’s 12-month price target on MapLight shares 44%, from $43 to $24.

“Although [ML-007C-MA] did not meet the Street’s upside expectations, there are still several positives to consider,” Jefferies equity analyst Andrew Tsai wrote Friday. He said ZEPHYR was still successful enough as a pivotal Phase II trial to count as one of two positive Phase II or III trials needed for FDA approval. And twice daily ML-007C-MA showed competitive adverse event percentages among patients compared to Cobenfy, he added, citing:

Constipation—9% for ML-007C-MA vs. 13–21% for Cobenfy.
Nausea—29% vs. 19%.
Vomiting—13% vs. 9–16% for Cobenfy.

By mid-week, investors appeared to take the analyst commentary to heart. MapLight shares rebounded, climbing 24% to $12.31 on Tuesday, then jumped another 22% to $15.02 Wednesday. The rest of the week didn’t look as good for MapLight, however, as its shares fell about 7% to $14.03 Thursday and dropped another 7% Friday, finishing the week at $13.03.

Leaders & laggards

Novo Nordisk (Nasdaq Copenhagen: NOVO-B) shares slumped 8% from DKK 330.90 ($51.03) to DKK 306.50 ($47.27) Friday, while its American Depositary Shares (Nasdaq: NVO) skidded 9% from $51.61 to $47.08, after the cardiometabolic drug giant acknowledged that its once-monthly 15 mg dose of ziltivekimab failed the Phase III ZEUS trial (NCT05021835) assessing the IL-6 inhibitor vs. placebo in reducing the risk of major adverse cardiovascular events (MACE), defined as cardiovascular death, non-fatal heart attack, or non-fatal stroke. Ziltivekimab failed to translate reductions in cardiovascular inflammation into fewer major cardiovascular events, Novo Nordisk said. Overall rates of adverse events (AEs) and serious AEs in ziltivekimab patients were similar to those seen with placebo. A higher proportion of people treated with ziltivekimab had serious infections compared to placebo—a finding consistent with targeting IL-6 inhibition, according to the company—while no difference in all-cause mortality was seen.
Replimune Group (Nasdaq: REPL) shares more than doubled, jumping 107% from $5.41 to $11.20 Friday, the day after the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee sided with the company by voting 10-3 that the results from the Phase I/II IGNYTE trial (NCT03767348) were evaluable and clinically meaningful. Repligen is seeking FDA approval of its third biologics license application (BLA) for RP1 (vusolimogene oderparepvec, a genetically engineered oncolytic viral immunotherapy, in combination with nivolumab, the programmed death-1 (PD-1) immune checkpoint inhibitor marketed by Bristol Myers Squibb (NYSE: BMY) as Opdivo^®, as a treatment for advanced melanoma in patients who have progressed on prior anti-PD-1 therapy. “We are encouraged by today’s outcome and would like to thank the committee for its thoughtful discussion of the IGNYTE data,” Repligen CEO Sushil Patel, PhD, said in a statement. Cantor Fitzgerald analyst Li Watsek upgraded Replimune shares from “Neutral” to “Overweight,” with no price target on the stock.

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Molecular GPS Guides Neutrophils to Sites of Infection

Edusehat — Sat, 01 Aug 2026 04:35:08 +0700

Data from a scientific collaboration between scientists at the University of Bath and UMass Chan Medical School helps explain how neutrophils, part of the immune system’s defense system, move through the body. Their findings shed light on the mechanisms that control neutrophil movement to specific infection sites without damaging healthy tissues along the way. According to the scientists, their findings point to a potential new target for anti-inflammatory drugs that can treat different conditions including chronic inflammatory diseases of the gut and lung.

Details of their work are published in Science Advances in a paper titled “Transient Receptor Potential Vanilloid 2 Functions as a Directional Driver for Hepoxilin A3-Mediated Neutrophil Migration.” In the paper, the scientists identify a multi-step process whereby neutrophils that emerge from blood vessels near an infection are guided to specific tissue sites. It involves the release of a molecule called hepoxilin A3 by infected cells.

“Neutrophils are cells that can act like bombs, releasing a deadly cocktail of chemicals to kill off disease-causing microbes once they reach a site of infection,” said Randy Mrsny, PhD, a professor in the department of pharmacy and pharmacology at the University of Bath and one of the study’s co-leads. “Unfortunately, in patients with chronic inflammation, their neutrophils can get incorrect signals, making them act as though there is an infection to be neutralized, setting off these bomb-like events and leading to unnecessary tissue damage.”

The current research builds on previous studies from Mrsny and his collaborators that showed how type 2 cannabinoid receptor, CN2R, activation by endocannabinoids could suppress hepoxilin A3-mediated neutrophil migration in the absence of infection. “After nearly 15 years working on this area, we’ve identified exactly how neutrophils ‘know’ how to move, stop, and even change direction to specifically target the infection site and unleash their anti-infection weapons at just the right moment to limit damage to healthy tissues.”

Here’s how that process works. Infected cells release hepoxilin A3 that is detected by TRPV2, a sensor protein on the surface of neutrophils. Once the molecule is detected, TRPV2 combines with CB2R, to form a signaling complex that directs migration of neutrophils selectively towards the infection site. Crucially, the neutrophils do not release any caustic agents during the migratory process, which is how they avoid damaging tissues along the way, the scientists explained.

“One of the greatest challenges in treating chronic inflammatory disease is preserving the immune system’s ability to fight infection while preventing unnecessary tissue damage,” said Beth McCormick, PhD, professor and chair in the microbiology department and founding director of the UMass Chan program in microbiome dynamics. McCormick is also a co-lead on the study. “By uncovering this molecular navigation system that precisely directs neutrophils to sites of infection, we’ve identified a promising therapeutic strategy that could restore precision to inflammation rather than simply suppressing it.” She added that the findings represent “an important step toward a new generation of targeted anti-inflammatory therapies.”

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Ketamine Triggers Sex-Specific Brain Recovery Responses

Edusehat — Sat, 01 Aug 2026 04:35:07 +0700

During ketamine anesthesia the brain’s nerve cells fall silent, and as consciousness returns, they begin to reconnect. A study in mice by researchers at the Institute of Science and Technology Austria (ISTA) and colleagues at Allen Institute for Brain Science in Seattle, has now for the first time shown that immune cells in the brain play a critical role in this process, and indicates that there are differences between female and male brains.

Senior and corresponding author Sandra Siegert, PhD, at ISTA, reported on their findings in Science Advances, in a paper titled “Corticosterone-linked microglial activity underpins sexually dimorphic neuroplasticity after ketamine anesthesia,” in which they concluded “Our study uncovers significant sex-specific differences in neuronal adaptation during recovery from ketamine anesthesia, driven by microglia.”

Recovery from anesthesia is critical for resuming normal physiological and neuronal functions, but the mechanisms involved remain elusive, the authors wrote. “Ketamine distinguishes itself from other anesthetics by its unique pharmacological properties as an N-methyl D-aspartate (NMDA) receptor antagonist, which preferentially targets GABAergic inhibitory interneurons.”

And unlike many other anesthetics, ketamine does more than induce unconsciousness. It alters how we perceive pain and form memories. It dampens communication between neurons—the very network that must later resume normal function as the patient awakens. Exactly how this recovery process unfolds—and whether male and female brains differ in this regard—has been unclear. “… ketamine anesthesia induces mild anxiety behavior phenotypes, interestingly, only in females,” the team continued, “suggesting inherent sex differences in anesthesia recovery with neuronal consequences that extend beyond the immediate sex-dependent metabolic processing described for low-dose ketamine.”

Working with mice, Siegert, together with Alessandro Venturino, PhD, and their colleagues at ISTA, and researchers at the Allen Institute, now offer the first answers to some of these questions. Microglia are specialized immune cells that constantly scan the brain and, when needed, trigger anti‑inflammatory responses. They also monitor neurons and their connections, thus helping to maintain optimal brain function. “Ketamine, across different dosages, affects microglia, which are embedded within the neuronal network,” the team explained. “Locally, microglia influence the synaptic machinery and neuronal firing properties by responding to environmental changes.”

As early as 2017, Siegert’s group at ISTA noticed that male and female mice respond differently to ketamine anesthesia, and more precisely, their microglia do.

For the newly reported study, using a cranial window—a surgically implanted opening that allows microscopic access to the living brain—Venturino analyzed how microglia and neurons behave while mice recover from ketamine anesthesia. Both cell types were labeled with fluorescent markers to glow under the microscope.

The researchers observed microglia processes in their dynamic action towards neurons. Surprisingly, as female mice recovered from anesthesia, microglia began forming prolonged contacts with neurons, coinciding with the onset of synaptic remodeling and plasticity. Notably, this phenomenon was not observable in male mice.

Furthermore, in mice lacking microglia, no such synaptic remodeling occurred, indicating that microglia are critical mediators of this recovery-associated plasticity. “What was fascinating,” Venturino explains, “was that we observed this plasticity—the brain’s ability to change, adapt, and in this case recover—only in females.”

Despite many other projects—or perhaps because of them—the researchers kept returning to their initial observation. “I’ve always believed that women have greater brain plasticity,” Siegert said with a smile. “Alessandro and I just couldn’t let it go—we wanted to know why.”

Further experiments revealed that this plasticity depends on corticosterone, one of the major stress hormones. “During recovery from anesthesia, corticosterone levels rise,” Venturino explained. “In female mice, this specifically activates the stress‑response gene Fkbp5 in microglia. The gene encodes the protein FKBP51, which helps the cell manage stress signals—and apparently prompts microglia to interact with neurons.”

The authors further noted, “Mechanistically, we found that female microglia selectively up-regulated the cochaperone Fkbp5/FK506-binding protein 51 (FKBP51), which is a key intermediary in the corticosteroid-induced stress response … Our findings underscore that microglia serve as a relevant interface between the endocrine stress response and the brain -immune cell system.”

To confirm this link, the team removed the adrenal glands—the endocrine organs that produce corticosterone. Without them, the close contact between microglia and neurons during recovery disappeared. “These results clearly show that corticosterone triggers this reaction in female mice,” said Venturino.

Siegert added, “They also remind us that stress is not always harmful—stress hormones are essential for certain processes in the brain.” In their paper the investigators commented, “The selective hypothalamic activation and elevated blood plasma corticosterone levels during the recovery phase in females shape the microglia-neuron interactions, highlighting a link between the endocrine and the brain-immune axes.”

Why this process differs between female and male mice remains uncertain; it is still unclear whether the male brain uses a similar mechanism, just delayed, or has another strategy. “Microglia enable rapid adaptation, and these cells in females are likely more sensitive to specific stress signals,” Siegert noted.

From an evolutionary viewpoint, she speculates, females may have faced greater demands for social, emotional, and multitasking adaptability—for example, in childcare, food gathering, or coordinating group activities. The female brain, therefore, had to adapt and respond more swiftly. “That’s a good thing,” Siegert added “But if this plasticity becomes too frequent or too intense, it can increase the risk of depression. We also know that psychiatric disorders are more prevalent in women than in men.”

Siegert further pointed out that during the literature review, her team found very few studies in which ketamine had been tested in females. “There were only a handful of anecdotal studies showing that women experience nausea and sickness more often after ketamine anesthesia,” she stated. Given that ketamine is also used as an antidepressant, understanding how its mechanisms differ between the sexes is all the more important. “It’s astonishing how readily people assume that men and women respond to drugs in the same way—when clearly they do not,” Siegert stressed.

Research like this is a step in the right direction: it highlights that medications can act differently in women and men and serves as a call to consider sex‑specific differences in future studies. “Our findings contribute to a growing body of evidence recognizing sex-specific differences in brain function and immune responses, the latter of which is already well known for increased susceptibility to infection and autoimmune diseases,” the team pointed out. “Our results identified a link between microglia-specific Fkbp5 expression and ketamine action, warranting a reevaluation of assumptions that ketamine is a general anesthetic and fully reversible across sexes.”

Insights into the microglia response to ketamine have since inspired Siegert and Venturino to co‑found Syntropic Medical, a start‑up in ISTA’s XISTA ecosystem exploring how 60 Hz flickering light can soften such neural networks in the brain.

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Mitochondrial–Epigenetic Pathway Linked to Age-Related Inflammation

Edusehat — Sat, 01 Aug 2026 01:00:15 +0700

Scientists have identified a mechanism that helps aging cells drive the chronic inflammation that is linked to many age-related diseases. The findings help explain how dysfunctional mitochondria work with the cell’s epigenetic machinery to turn on inflammatory genes. Full details are available in a Nature paper titled “Mitochondrial metabolism and epigenetic crosstalk drive SASP” that is available now.

Existing research shows that senescent cells accumulate with age and remain metabolically active even though they no longer divide. As they accumulate, they release a cocktail of inflammatory molecules known as the senescence-associated secretory phenotype, or SASP. This inflammation is thought to contribute to frailty, cardiovascular disease, cancer, neurodegeneration, and other disorders of aging.

In the current study, the scientists focused on switching off this inflammation rather than on getting rid of senescent cells as some other approaches have tried.

The study was done by teams at Mayo Clinic and Sanford Burnham Prebys Medical Discovery Institute and their collaborators elsewhere. It builds on previous work done by João Passos, PhD, a Mayo Clinic researcher and senior author of the study. Passos’ lab previously demonstrated that damaged mitochondria leak mitochondrial DNA and RNA into the cell and this activates immune pathways that trigger inflammation. The current Nature study identifies a second, independent pathway that is equally essential.

“We found that inflammatory signaling alone isn’t enough,” according to Helene Martini, PharmD, PhD, a Mayo Clinic researcher and first author of the study. “The cells also need a metabolic signal from mitochondria that changes how inflammatory genes are turned on.”

Specifically, the scientists discovered that senescent cells increase production of acetyl-CoA. This molecule can be generated from a range of sources in the body, but the data indicate “that in senescent cells, mitochondria constitute a dominant source of acetyl-CoA for chromatin modification.” Once released, acetyl-CoA enables epigenetic modifications that make inflammatory genes more accessible, allowing them to be expressed. Essentially, “we found that dysfunctional mitochondria can promote inflammation by controlling epigenetic switches that turn inflammatory genes on,” Martini said.

In addition, the team identified a promising therapeutic target. They found that blocking a mitochondrial citrate transporter known as SLC25A1 reduced the supply of acetyl-CoA, which “reduces histone acetylation at SASP loci.” In other words, it limited inflammatory gene activation even though the initial immune signals remained present. “These findings position SLC25A1 inhibition as a novel therapeutic target that modulates the inflammatory output of senescent cells through metabolic–epigenetic coupling,” the scientists wrote. “More broadly, they suggest that targeting metabolic inputs into chromatin regulation may represent a tractable strategy to mitigate age-associated inflammation and functional decline.”

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A Computational Framework for Designing Disordered Proteins at Large Scale

Edusehat — Sat, 01 Aug 2026 01:00:14 +0700

The vast majority of proteins in our bodies contain regions that are in a constant state of wiggling, shape-shifting every few nanoseconds to completely change how they look. Information on how these proteins work is critical to understanding health and disease and to developing drugs for cancer, neurodegeneration, and myriad other conditions. However, it has been challenging for scientists to pin down precisely how these regions function, and how they go awry in disease.

Researchers at Washington University School of Medicine in St. Louis and at Syracuse University have now built a tool that can design such “disordered” proteins (intrinsically disordered protein regions; IDRs) and untangle their functionality. The team suggests the innovation has the potential to accelerate scientific exploration of a vast and underexplored area of biology.

Alex Holehouse, PhD, an associate professor in the WashU Medicine Department of Biochemistry and Molecular Physics, is research co-lead and co-corresponding author of the team’s published paper in Nature, titled “Rational design of disordered proteins for sequence–function investigation,” in which they explained, “Our work uses rational sequence design as a powerful method for exploring function in IDRs and provides a versatile tool for designing functional disordered proteins.”

An important way scientists study proteins is to design synthetic equivalents of the molecules that they can then test in various ways. Until now, advances in such protein design have applied almost entirely to “folded” proteins—or their folded parts—that have a defined three-dimensional shape.

Yet 70% of human proteins also contain what’s known as intrinsically disordered protein regions (IDRs) that don’t have a stable 3D structure. “IDRs exist as a dynamic collection of rapidly interconverting and structurally distinct conformations,” the team explained. These regions can play critical roles in a variety of different cellular processes and human diseases. Researchers’ ability to predict how they will behave, or to design synthetic versions to study their function, has been limited. “Despite their importance, systematically testing the relationship between IDR sequence and molecular function remains challenging,” the authors further wrote. “While rational design of folded proteins has seen substantial recent progress, our ability to design IDRs remains more limited.”

Holehouse said, “The way people would typically try to study and design stable, folded proteins doesn’t really work very well for disordered proteins.” He and colleague Ryan Emenecker, PhD, a faculty instructor in the same department and lead developer and co-corresponding author on the study, have been working on an alternative way to tackle this challenge for almost five years.

Holehouse, Emenecker and their collaborators, including co-corresponding author Shahar Sukenik, PhD, a faculty member in the Department of Chemistry at Syracuse University, have now reported on development of the protein-design system, which they called GOOSE (an acronym derived in an appropriately disordered way from Generate disOrdered prOtiens Specifying propErties).

Loaded with a large library of the sequences for protein building blocks that are associated with specific cell functions, GOOSE produces blueprints for custom-built disordered proteins that are then created in genetically engineered cells. Scientists can remove or add building blocks as desired and test what effect they have on the activities of a cell. “GOOSE enables rapid design of de novo synthetic IDRs and variants of provided sequences, facilitating broad exploration of sequence space,” they commented. “GOOSE can design IDRs by sequence properties (such as amino acid composition, charge, hydrophobicity and charge patterning), conformational properties, chemically specific intermolecular interactions or any arbitrary design constraint (such as specific three-dimensional conformational ensembles).”

Emenecker stated, “The ability to design disordered proteins at a large scale with our platform now allows us to learn how their component sequences affect the cell, and it gives us a lens through which we can learn how naturally occurring changes in these proteins might drive diseases like cancer.”

The technique has potential for driving medical advancements. Holehouse, who is a research member of Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine, is seeking to optimize therapeutics that rely upon disordered proteins. Holehouse and Emenecker have received a grant to improve CAR T cells, an anti-cancer therapy in which immune cells are genetically modified to attack tumor cells.

A key protein on the surface of CAR T cells contains a disordered region that guides the cell’s attack response. Until now, scientific efforts to improve its performance in destroying cancer cells have been conducted largely by trial and error.

“With our technique, we can design better versions of these disordered regions to do the signaling in different ways,” said Holehouse. “The hope is we won’t be limited by the types of constraints that are currently hurting the efficacy of CAR T therapies. That’s a very concrete place where these tools can move medicine forward.”

Among various applications, one of GOOSE’s first tests was to generate synthetic proteins that could help cells respond to changes in external stressors—in this case, drought. “We were able to very quickly design 2,300 different proteins that would respond to drought conditions in yeast,” said Emenecker, who commented that many of these synthetic proteins proved GOOSE’s utility by working as intended, helping the cells’ recovery after drying out. Even more promising, many of them performed much better than the yeast’s natural proteins.

This work directly contributes to Holehouse, Emenecker and Sukenik’s ongoing work as part of a larger National Science Foundation initiative to engineer more environmentally resilient crops.

“More broadly, this opens the possibility of being able to make new sensors that are sensitive to things outside what natural sequences would be attuned to, like toxins or cell damage,” Emenecker said. “It has the potential to be very valuable.” In their paper the team concluded, “Taken together, our work highlights how GOOSE can be used to gain insights into IDR sequence–function relationships.”

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Spatiotemporal Multiomics Charts Cellular Dynamics of Liver Metastasis

Edusehat — Sat, 01 Aug 2026 01:00:13 +0700

Metastasis remains one of cancer’s most difficult biological transitions to capture: tumor cells must leave a primary tumor, survive circulation, enter a distant organ, and then either disappear, persist, or eventually grow into clinically detectable lesions. A spatiotemporal study in mice and human samples identifies transient tumor-cell and immune-niche states that may offer windows for intercepting metastatic colonization.

A new study published in Science provides a high-resolution look at that process in liver cancer, suggesting that metastatic colonization unfolds through ordered changes in both disseminated tumor cells and the immune microenvironments that surround them.

In the study, “Spatiotemporal multiomics uncover tumor ecosystem dynamics during metastatic colonization,” researchers led by Yunfan Sun, MD, PhD, at Zhongshan Hospital, Fudan University, applied spatiotemporal multiomics to experimental hepatocellular carcinoma mouse models and human metastatic samples. Their goal was to reconstruct how disseminated tumor cells, or DTCs, survive the earliest stages of lung colonization and later transition into metastatic outgrowth.

The team integrated high-resolution spatial transcriptomics, single-cell RNA sequencing, and chromatin-accessibility profiling across nine sequential stages of lung colonization in mouse models. The resulting atlas followed liver cancer cells from their first arrival in the lungs through later metastatic progression, while also mapping changes in nearby immune cells.

The analysis indicated that early metastatic seeding is not simply a random survival event. “After a massive innate immune clearance, primarily by neutrophils and natural killer (NK) cells, a rare subpopulation of DTCs survived by entering a transient, quiescent Phgdh^high state,” the authors write. These cells were associated with an immune-scarce niche, allowing them to avoid elimination during a vulnerable early window.

Mechanistically, the authors linked this state to metabolic and epigenetic remodeling. Alveolar type 2 cells enriched near surviving DTCs appeared to promote the Phgdh^high phenotype. Elevated PHGDH activity fueled one-carbon metabolism and increased levels of S-adenosylmethionine (SAM). That shift was tied to H3K27me3-mediated silencing of proinflammatory chemokine genes, including Ccl2 and Cxcl10, which would otherwise help recruit immune cells to the niche.

Perturbing this axis genetically or pharmacologically restored chemokine expression, increased immune surveillance, and reduced metastatic outgrowth in the models, according to the study. Lineage-tracing experiments further suggested that many macrometastases derived from ancestors that had passed through the transient Phgdh^high state.

The researchers also identified a second niche-remodeling step before rapid metastatic expansion. At this stage, Cx3cr1^high interstitial macrophages accumulated in the DTC niche. These “macrophages recruited immunosuppressive cells (T regulatory cells, neutrophils, and alveolar macrophages) and provided growth signals through the IGF1-IGF1R axis that trigger the transition of DTCs from quiescence to rapid proliferation,” the authors report in the study. Depleting these macrophages reduced metastatic burden in mouse experiments.

Together, the findings point to metastatic colonization as a temporally organized process shaped by reciprocal interactions between tumor cells and their local microenvironment. First, a rare tumor-cell state helps establish early immune evasion. Later, macrophage-driven remodeling appears to convert a quiescent niche into one that supports metastatic outgrowth.

Although the work is largely preclinical, the authors suggest that these transient states may represent vulnerabilities for micrometastasis-targeting approaches. By defining when and how early DTCs evade immune attack, the study offers a framework for developing interventions aimed not only at established metastases, but also at the earliest stages of metastatic colonization.

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Axiom’s Hong Kong Bet, Latigo’s Positive Phase II, and Base Editors for Huntington’s

Edusehat — Sat, 01 Aug 2026 01:00:12 +0700

In the business news section of this week’s episode, Axiom Biosciences recently announced its plans to go public and made headlines for its plans to do so on the Hong Kong Exchange rather than a U.S. market. We dig into the company’s reasoning. Then we turn our attention to Latigo’s report of positive Phase IIb data for its non-opioid acute pain candidate, and its plans to go through a planned IPO soon. Switching to some science news, we discuss findings from a study that identified genetic risk factors for fibromyalgia syndrome as well as links to other neurodegenerative, autoimmune, and metabolic diseases. And lastly, a potential base editing therapy for Huntington’s disease that has shown promise in mice.

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

StockWatch: Axiom CEO Explains Plans for Hong Kong IPO
By Alex Philippidis, GEN Edge, July 27, 2026

Latigo Reports Positive Phase IIb Data for Non-Opioid Acute Pain Candidate
By Alex Philippidis, GEN Edge, July 30, 2026

Genetic Study of Fibromyalgia Points to Neurological Basis
GEN, July 29, 2026

Base Editing Strategy Alleviates Huntington’s Disease in Mice
GEN, July 29, 2026

Touching Base Podcast
Hosted by Corinna Singleman, PhD

Behind the Breakthroughs
Hosted by Jonathan D. Grinstein, PhD

The State of Biologics Testing 2026
Charles River Laboratories and GEN, June 10, 2026

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Microbiota-Derived Metabolite Enhances HIV Therapy in Monkeys

Edusehat — Sat, 01 Aug 2026 01:00:11 +0700

HIV infection remains a major global public health issue. In 2025, an estimated 40.9 million people were living with HIV, and approximately 1.2 million people acquired new HIV infections. In 2025, around 570,000 people died from AIDS-related illnesses worldwide.

Gut-associated lymphoid tissue is an early target of HIV. The virus severely damages the immune and epithelial cells in the gut’s lining, leading to an inflamed, leaky gut, a weakened defense system, and decreased nutrient absorption. The virus also disrupts mitochondrial function.

Even with strict adherence to antiretroviral therapy (ART), many people living with HIV have persistent gut inflammation caused by the virus. Previous research has suggested the bacterium Lactiplantibacillus plantarum—a common lactic acid bacterium found in the human gut, over-the-counter probiotics, and fermented foods—could quickly heal the chronically inflamed leaky gut associated with HIV.

Now, researchers have identified the metabolite that repairs gut damage caused by HIV infection and significantly improves the effectiveness of ART in the nonhuman primate model of HIV/AIDS.

The findings were published in Nature Microbiology in the paper, “Microbiota-derived 10-hydroxystearic acid activates PPARα to restore gut epithelial barrier integrity and enhance anti-retroviral therapy.”

“Current HIV therapies are remarkably effective at controlling viral replication, but they do not fully repair the profound damage HIV causes in the gut,” said Satya Dandekar, PhD, professor in the Department of Medical Microbiology and Immunology at UC Davis Health. “Our findings suggest that restoring the gut’s structural and immune health can enhance antiretroviral treatment and opens an entirely new avenue for achieving more durable control of HIV.”

For this study, the researchers identified metabolites produced by L. plantarum in the virally inflamed gut environment in the non-human primate model of HIV/AIDS. Among the hundreds of molecules created by L. plantarum, one metabolite, 10-hydroxystearic acid (10-HSA), emerged as the strongest candidate for repairing the gut barrier and reducing inflammation.

Using X-ray crystallography showed that 10-HSA directly binds to PPAR-alpha, a nuclear receptor that regulates key biological processes. This binding promoted inducing lipid metabolism, mitochondrial regeneration and subsequent epigenetic histone crotonylation, thereby promoting gut epithelial renewal.

The team also conducted two independent studies at the UC Davis National Biomedical Research Institute in non-human primates infected with simian immunodeficiency virus (SIV). The first study evaluated 10-HSA without ART, which led to intestinal repair, improved key markers of gut function, improved mitochondrial health, reduced inflammatory signaling and partially restored the gut microbiota.

When 10-HSA was given in combination with ART, the combined treatment showed faster clearance of viral burden than ART alone. The combination also promoted faster recovery of gut immune cells, reduced immune activation, restored epithelial barrier integrity and restored beneficial gut microbiota and microbial diversity.

“The findings suggest repairing the tissue damage caused by HIV may be as important as suppressing the virus itself with antiretroviral therapy,” said Dylan Kramer, PhD, a recent graduate from the Dandekar Lab. “By rebuilding the gut barrier, restoring mitochondrial function and reducing inflammation, 10-HSA helps create conditions that support stronger immune recovery and more effective antiviral therapy for HIV.”

The authors caution that the results are from preclinical models. The studies showed no adverse effects. The research supports testing the safety and effectiveness of 10-HSA in humans.

“Our research shows that host health, microbial health and viral control are deeply interconnected,” Dandekar said. She noted that treating the damaged gut ecosystem facilitates recovery of the immune system, leading to regained functions that have been lost during HIV infection. Additionally, treating the damaged gut during HIV infection can improve outcomes beyond what antiviral drugs can achieve alone.

“The restoration of gut barrier integrity and microbial balance through 10-HSA supplementation may represent a promising therapeutic strategy, with implications that extend beyond HIV to other chronic inflammatory diseases of the gastrointestinal tract,” Dandekar said.

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Montana’s new “right to try” law can’t come soon enough for some

Edusehat — Fri, 31 Jul 2026 21:15:07 +0700

Kris DeVault is desperate.

His son, Brody, was born in March 2023. It wasn’t long before he started to show signs of developmental delay, says DeVault. As time went on, Brody started missing key milestones in speech, movement, and coordination, he says.

When Brody was around two and a half years old, a genetic test revealed creatine transporter deficiency—a rare condition in which the brain and muscles lack the energy they need to develop.

There are no cures for Brody’s condition. But DeVault has learned of a company developing a drug that might help. That drug is still in the early stages of development and has only been tested in animals and a small number of healthy adults. Doctors can’t prescribe it.

DeVault knows the drug might not work. But he’s doing all he can to access it regardless. And a new law in Montana could make it easier for people in his position to get access to treatments—at least in theory.

Today, Brody is three years old. His dad describes him as a happy, curious, and loving little boy who wants to learn. But Brody struggles to communicate. “He’s got no words, really,” says DeVault. “He wants to communicate more than he’s able to … which then turns into frustration.”

It’s difficult for Brody to tell his parents whether he’s hot, cold, hungry, thirsty, uncomfortable, or even in pain, says DeVault. He recently found Brody standing on an anthill in the backyard, being bitten by red ants. “These fire ants were just going to town on his feet … and he was just looking,” he says.

Brody has muscle weakness too. “He can’t move very fast, he doesn’t have a ton of strength … and it takes a lot of energy for him to walk balanced,” says DeVault. “His arms are skinnier than [those of] his nine-month-old sister.”

It’s concerning, but DeVault is most worried about Brody’s neurological development. Toddlers’ brains are exceptionally “plastic”—the first years of a child’s life are thought to be crucial for long-term brain development.

A biotechnology company in France is working on a drug to help people like Brody. Creatine usually provides brain cells with energy. People with creatine transporter deficiency (CTD) can’t get creatine into the brain.

The team at Ceres Brain Therapeutics is developing a treatment designed to bypass this issue and effectively deliver creatine directly to the brain. So far, the team has seen promising results in mice, says Ceres CEO Thomas Joudinaud.

The company also recently completed a phase I clinical trial that involved testing various doses of the drug, which is delivered as a nasal spray, in 48 healthy adult volunteers. That trial has not yet been published, says Joudinaud. The drug has not been tested in people with CTD, or in children.

“I look at this, and I’m like, that is my one shot for Brody,” says DeVault.

Kris DeVault, his son Brody, and his wife and young daughter.

COURTESY OF THE DEVAULT FAMILY

Joudinaud is planning a phase II trial in people with CTD, as well as others with amyotrophic lateral sclerosis. But that trial will take place in France, and it’s unlikely that Brody will be able to take part, says DeVault.

Ceres can’t make the drug available to Brody under an expanded access scheme run by the US Food and Drug Administration either, because the drug has not been registered with the FDA, and because it is currently manufactured in a way that does not comply with FDA regulations, says Joudinaud.

Even if that phase II trial is successful, and if the drug is ultimately approved, it is unlikely to reach the US market for at least a few years. DeVault is worried that will be too late for Brody—he’ll be “past his plasticity window” by then, he says.

Now, with the adoption of a new law in Montana, he theoretically has another option. Montana has had a “right to try” law—which allows terminally ill people to apply for access to unapproved drugs—in place since 2015. In 2023, a new law technically expanded this option to people who were not terminally ill, providing the drugs have been through preliminary phase I clinical trials. A second law aimed to clarify how clinics could sell and administer those treatments to patients. And last weekend, the state’s department of Health and Human Services finalized a set of rules for those clinics.

An experimental treatment review board (ETRB) has been established to review applications for access to experimental, unproven, and unapproved drugs. And it is set to review its first two applications in the coming weeks.

Ceres could also apply to Montana’s ETRB to sell its experimental treatment to Brody’s parents via a clinic in the state. But Joudinaud is reluctant, at least for the time being. While he thinks that Montana’s setup is “very interesting and very pragmatic” and “suitable for our drug,” he’s worried about getting on the wrong side of the FDA.

DeVault has been pleading with FDA staffers for a written statement essentially promising that biotech companies participating in Montana’s program won’t be penalized later on, especially when they eventually try to get their drugs approved in the US. But he hasn’t made any progress.

Now he’s looking beyond Montana. He’s considering accessing treatment in Próspera, a private city and “special economic zone” in Roatán, Honduras, where a clinic sells unproven stem-cell and gene therapies, among others.

Many scientists have cautioned against the use of such “offshore” clinics. Even when it comes to Montana, scientists, bioethicists, and health law experts will caution that phase I clinical trials don’t prove a drug is safe. And they certainly don’t prove a drug’s efficacy, either.

When I spoke to Aaron Kesselheim, a professor of medicine at Harvard Medical School with expertise in health policy and drug regulation, about the Montana law earlier this week, he made his concerns clear. “Patients who want these kinds of treatments deserve them to be rigorously assessed so that [they] can better understand what they’re getting themselves into, and what they’re paying their hard-earned money for.”

But DeVault pushes back on these arguments. “I’m a full-grown human being,” he says. “I’m capable of going to Vegas right now … blowing it all on the poker table, [or] I can go to the gun shop and buy a silenced [semi-automatic rifle] … how come I can’t make a decision to purchase a potential treatment that might change the entire trajectory of my son’s life?”

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.

Montana’s plan to become an experimental medical hub just pushed forward

Edusehat — Fri, 31 Jul 2026 06:30:02 +0700

As of this week in Montana, any biotech company with an experimental drug has a clear path to selling it to consumers. Companies whose drugs have been through preliminary testing—sometimes in as few as 10 healthy people—can pay $12,500 to apply to a newly established review board for approval. Once its treatment is rubber-stamped, the company can set the price of the drug and sell it via experimental treatment clinics, the first of which is likely to be up and running around the end of this year.

Montana’s latest right-to-try legislation is unique. While other jurisdictions with similar laws limit access to drugs to people with terminal illness, in Montana access is theoretically available to anyone who gives informed consent and can pay. That includes people desperate for treatments for rare diseases. It also includes those who are interested in longevity and want to try out drugs pitched as preventive therapies.

The state’s Department of Health and Human Services recently finalized rules to implement the law. The rules stipulate that patient consumers provide fully informed consent and that each application be reviewed by a board that includes a Montana-certified doctor, expert scientists, and an ethicist. Supporters of the law stress that they want the process to be responsible. “It will be done in a very rigorous way, with qualified medical professionals and appropriate oversight,” says Matt Kaeberlein, a scientist on the first board, which was formed independently of the state health department.

But other experts are worried about the potential for harm in selling unproven treatments to people without oversight from the US Food and Drug Administration. “I would be concerned,” says Aaron Kesselheim, a professor of medicine at Harvard Medical School with expertise in health policy and drug regulation.

There has been a growing movement to make unapproved drugs more accessible in the US. But the story of Montana’s law is unique. It’s been driven and drafted by longevity enthusiasts instead of the usual libertarian and patient groups.

An unusual origin story

Montana first passed a right-to-try law in 2015. In 2023, with the support of state senator Ken Bogner, the state expanded the law to include all patients, not those just with terminal disease. Last year, Bogner told MIT Technology Review that his vision was to focus “more on preventative medicine” rather than “just treating diseases once they show up.”

Bogner says he had “started working on a bill” that would become the 2023 law when the Alliance for Longevity Initiatives (A4LI), a nonprofit “dedicated to advancing legislation and policies aimed at increasing healthy human lifespan,” got in touch. A4LI connected Bogner with others who helped draft the bill and testified in support of it.

Once that law was in place, the tech entrepreneur and longevity enthusiast Niklas Anzinger got involved. Anzinger has been working to establish a jurisdiction to fast-track the search for drugs that might deliver radical life extension. He is based in Próspera—a private city and “special economic zone” in Roatán, Honduras, which is already home to a separate clinic that sells experimental stem-cell and gene therapies. Anzinger founded a community there called Infinita City; he has also founded an investment company and a “service providing” company, both of which include the name Infinita.

Over the last couple of years, Anzinger has switched his focus to the US. “Now we think that Montana is a better model, because it’s building on … existing regulatory precedents,” he says. Once Montana’s 2023 law was passed, he adds, he worked with a handful of unnamed biotech companies to draft a second bill—one that laid out the specific terms under which clinics can offer unapproved drugs. That law was passed in April 2025 and adopted the following month.

Since then, Anzinger, Bogner, and others have been waiting for the state’s Department of Health and Human Services to finalize specific rules for treatment centers—a set of operational guidelines and requirements that any clinic offering treatments unapproved by the FDA must meet under Montana’s law. “The rules have been taking a very long time,” says Anzinger. “Then on Friday, we heard they were effective … from Saturday [July 25].” The rules have since been published online.

Following the rules

With the new rules in hand, Anzinger and his colleague Stephen Martin, Infinita’s US lead, got to work. The first step was to establish an independent experimental treatment review board—a panel of five experts to evaluate applications for access. Anzinger and Martin started recruiting candidates months ago.

The state’s first board, named the Montana ETRB, was officially announced by Infinita earlier this week. For the time being, it is the state’s only review board, although Anzinger says that other groups are free to establish their own. After Bogner raised concerns that the board’s website wrongly implied that it was an official state body, the site was updated to note that “It is a private service run by Montana Governance Services Inc.” That company is “a local Montana registered entity, but it is under the Infinita umbrella,” says Anzinger.

Infinita will pay board members a flat fee, funded by the $12,500 companies will have to pay to have their applications reviewed. Anzinger stresses that the board members, and their decisions, will be independent of Infinita.

In accordance with the rules, the board includes a Montana-licensed doctor: James Burke, an oncologist. It also includes a bioethicist: Jessica Flanigan, a libertarian who is known for her strong views in support of self-medication and her book Pharmaceutical Freedom.

The other three members are familiar faces in the longevity community—all of whom are well respected in the field. “When we looked at our own network, these were some of the best guys,” says Martin. They include Felipe Sierra, who formerly held a senior role at the National Institutes of Health’s arm focused on aging. More recently, Sierra served as chief scientific officer at Hevolution Foundation, a nonprofit that funds research into extending healthy lifespan with the support of the government of Saudi Arabia.

Matt Kaeberlein, who formerly led the Dog Aging Project and has studied the potential for rapamycin as a longevity therapeutic, also features. So does Jamie Justice, a gerontologist who is also executive director of the X Prize Healthspan competition, which has $101 million in prize money up for grabs for researchers who find ways to treat the signs of aging.

“I saw an opportunity to help build a safe, transparent, and scientifically rigorous process for implementing Montana’s newly expanded right-to-try legislation, particularly as it applies to longevity medicines and aging-related interventions,” says Justice. “Science is moving quickly, and I wanted to help ensure that as it develops, it does so with real rigor and accountability.”

Kaeberlein, who has a prominent media presence, has long raised his own concerns about access to other unproven treatments, including peptides and stem-cell therapies. He sees Montana’s setup as offering a more regulated environment—one that offers scientific oversight, ensures informed consent, and allows for data collection.

Applications incoming

While many of the bill’s original supporters were interested in longevity, the initial interest in making drugs more accessible in Montana is coming from companies and individuals looking to treat specific diseases.

“We were actually surprised that much of the interest … is actually more from oncology [and] neurodegenerative disease,” says Anzinger. This focus, he says, is “very compatible” with Infinita’s mission. “We’re not trying to convince everyone … to support radical life extension,” he says. Anything that extends health and human life, including treating cancer and neurodegenerative disease, is part of what longevity means to him, he says.

Martin says that two applications have already been submitted to the newly formed review board. They’ve come from biotech companies that are developing drugs for neuropathy and hearing loss, he says. “I expect we’re going to get started on them this week,” he says.

One of the applications was submitted by Stanley Kim, CEO of WinSanTor. His company is developing a treatment for peripheral neuropathy, a painful nerve condition that can be a consequence of cancer treatment or diabetes. The drug is currently in phase II trials, but Kim says he regularly receives messages from people who are desperate to access it, to the point of being suicidal. He hopes that not only will he be able to make the drug accessible to those people, but he’ll also be able to collect data from them—data that might help accelerate the drug’s approval process.

“We have a newsletter [that is sent to] around 15,000 patients,” says Kim. “Not all of them will be able to go to Montana, but many of them, I think, will.” His company still plans to continue with regular clinical trials as well.

But not all biotech companies with early-stage drugs feel comfortable submitting an application—at least not yet. Thomas Joudinaud, CEO of a French biotechnology company called Ceres Brain Therapeutics, has fielded a request from a person keen to access the company’s experimental drug in Montana. He says that while Montana’s system is “very interesting and very pragmatic” and “suitable for our drug,” he won’t be submitting an application for the time being. He is concerned that if anything goes wrong in Montana, it may jeopardize the company’s standing with the FDA, which wields the power to approve or reject the sale of its treatments to broader populations.

Martin and others have asked the FDA for some kind of assurance that biotech companies participating in Montana’s program won’t be penalized later on. But the agency hasn’t provided them with more than a restatement of the federal Right to Try Act.

“As a matter of policy, the FDA does not comment on state legislation,” an FDA spokesperson wrote in response to a request for clarification from MIT Technology Review.

Even if the FDA were to provide some kind of assurance, it wouldn’t necessarily protect biotech companies in the long term, cautions Chris Robertson, a specialist in health law at Boston University. The FDA’s position could change with a new presidential administration, he says: “I wouldn’t bet on anything that the FDA is saying today being applicable when the rubber hits the road later.”

Companies that want to stay on good terms with the FDA would be safest taking the expanded-access route, says Robertson. That’s the pathway the FDA already uses for people who are seriously or terminally ill, have run out of options, and want to try experimental drugs that have not yet been through clinical trials. The FDA approves over 99% of these applications, says Harvard’s Kesselheim.

“The FDA isn’t a bottleneck but in fact exists to help ensure that expanded-access programs are aboveboard and that patients who receive [the drugs] are able to contribute knowledge about [them],” says Kesselheim. He says he doesn’t think that any “legitimate manufacturer” should fear having to go through the FDA’s expanded-access process, which the agency says takes “less than 45 minutes” to fill out.

The cost of experimenting

There are some key differences between expanded access, which allows seriously ill people to apply for access to experimental drugs that might not have been through any human trials, and Montana’s approach. In theory, a person doesn’t need to be seriously ill to access experimental drugs in Montana.

“In Montana, patients may be eligible for preventive or earlier-stage interventions if they provide informed consent and meet the program’s requirements, so the breadth of potential therapies and situations is much broader,” says Kaeberlein, the Montana ETRB member, who is an affiliate professor at the University of Washington in Seattle.

Kaeberlein also highlights another key difference, which is cost. Companies that make their treatments available through expanded access are only able to charge for the costs of making, transporting, and monitoring the drug, and they must justify the eventual price to the FDA. In Montana, they can charge whatever price they want. Stanley of WinSanTor says he plans to sell his drugs “at cost.” But Ceres’s Joudinaud says that he’d be more interested in selling his at a market price. When asked what that might be, he hinted that the prices of new drugs for rare diseases can be high. In recent years, the median price of such drugs was $218,872.

“Instead of simply creating a legal pathway for patients, it also creates a business model that companies may actually be willing to use,” says Kaeberlein.

Beyond the financial cost, there will be risks associated with any experimental drug. Phase I trials don’t conclusively reveal whether a drug is safe. Around 17% of drugs are found to be inadequately safe during phase III trials. “The idea that a drug has been proven safe because it’s been subject to a phase I study is very, very wrong,” says Kesselheim. Bioethicists have raised concerns about the ethics of promoting and selling unproven treatments and the risk of harm should something go wrong.

But the moment when people start spending money on these treatments is already fast approaching. While Montana’s first ETRB prepares to review its first applications, clinics that hope to be part of the program are busy addressing the requirements laid out in the state’s new rules. Treatment rooms are being outfitted. Medical directors are being hired. And experimental treatments should be reaching patients in the coming months.

Latigo Reports Positive Phase IIb Data for Non-Opioid Acute Pain Candidate

Edusehat — Fri, 31 Jul 2026 02:40:10 +0700

Researchers from Latigo Biotherapeutics and its clinical partners have reported positive Phase IIb data for its lead pipeline candidate, the non-opioid acute pain treatment LTG-001, which showed significantly greater pain reduction scores than placebo over 48 hours in patients with moderate or severe pain after abdominoplasty.

LTG-001 achieved the primary endpoint of the Phase IIb LTG-001-010 trial (NCT07102459) with high statistical significance by achieving better-than-placebo scores in the time-weighted sum of the pain-intensity difference (SPID) over the 48-hour treatment period (SPID48), based on the 0-10 range of scores of the Numeric Pain Rating Scale, with higher SPID48 values indicating greater pain reduction.

“The publication of these findings adds to the growing scientific understanding of non-opioid approaches to pain management and comes at a time when there is broad recognition of the need for additional treatment options in the context of the ongoing opioid crisis,” Neil Singla, MD, Latigo’s chief medical officer, said in a statement.

LTG-001 is an oral, non-opioid, selective Na_v 1.8 inhibitor designed to deliver opioid-level analgesia. According to Latigo, LTG-001 has led to high levels of Na_v 1.8 target inhibition due to both its potency and degree of penetration into the peripheral nerve.

Among the 85 patients dosed with the low dose of LTG-001, the least-squares mean (LSM) was 161.05 (95% CI), while the 86 high-dose patients showed a LSM of 185.30, compared with 164.08 among 86 patients treated with hydrocodone bitartrate-acetaminophen, and just 123.22 among the study’s 86 placebo patients.

“High-dose LTG-001, but not low-dose LTG-001, was associated with significantly lower opioid consumption than placebo, as well as a significantly higher percentage of patients who did not receive opioid rescue medication, with more than half the patients in the high-dose group not receiving opioid rescue medication,” the researchers reported in “Phase 2b Trial of a Na_v 1.8 Inhibitor for Acute Pain,” a Latigo-funded study published Thursday in The New England Journal of Medicine.

Secondary endpoints for the study included the amount of opioid rescue medication consumed in morphine milligram equivalents (MME) and patients receiving no opioid rescue medication.

LTG-001 also showed positive results on the secondary measures: High-dose LTG-001 patients showed lower consumption of rescue opioids compared with placebo (—7.35 difference in MME; 95% CI, p=0.01), and a higher percentage of patients not receiving opioid rescue treatment (30 percentage point difference, 95% CI, P<0.001).

Median time to meaningful pain relief, defined as a reduction of 2 or more points in the Numeric Pain Rating Scale (NPRS) score from baseline, was 60.0 minutes with low-dose LTG-001 and 51.7 minutes with high-dose LTG-001—both better than the 82.8 minutes shown by hydrocodone bitartrate-acetaminophen and the 87.5 minutes shown by placebo.

“Further investigation is warranted to confirm these results, characterize the efficacy and safety profile of LTG-001 in additional models of acute pain, and compare the relative efficacy of LTG-001 with that of other Na_v 1.8 inhibitors,” the researchers added.

Going public

Latigo, which is based in Thousand Oaks, CA, declined comment on the study, since it is in a quiet period before going public through a planned initial public offering (IPO). The company has applied to list its common stock on The Nasdaq Global Select Market under the symbol “LTGO”.

On July 17, Latigo filed a Form S-1 registration statement with the U.S. Securities and Exchange Commission (SEC) disclosing plans to raise an undetermined amount of capital; the number of shares to be sold, and their IPO price, have yet to be set.

However, Latigo did say that an undetermined portion of proceeds from the IPO toward advancing the development of LTG-001 through Phase III bunionectomy and open-label safety topline results, toward the submission of a New Drug Application (NDA) with the FDA, as well as toward commercial readiness.

“We plan to initiate a placebo-controlled Phase III trial in participants undergoing bunionectomy and an open-label Phase III safety trial exploring LTG-001 within a broader population of patients with moderate to severe acute pain across a variety of post-surgical and non-surgical settings in the second half of 2026, with topline results expected in the second half of 2027,” Latigo disclosed.

Also in Latigo’s pipeline is LTG-321, a next-generation Na_v 1.8 inhibitor initially being developed as a treatment for chronic musculoskeletal pain, starting with osteoarthritis (OA). Latigo has launched a Phase II proof of concept trial for LTG-321 in patients with OA of the knee. The trial is designed as a randomized, double-blind, placebo controlled, within subject crossover study in approximately 120 patients with Western Ontario and McMaster Universities.

The schools’ Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) pain is the primary endpoint to establish clinical proof-of-concept for LTG-321 in chronic musculoskeletal pain and inform subsequent pivotal trial design. Latigo said it expects to report topline results in the second half of 2027.

A Phase I trial of LTG-321 has produced data that showed, as of May 15, 2026, that the candidate achieved robust pharmacodynamic activity as measured by an increased pain tolerance threshold, with continued activity at 24 hours after a single dose in the cold pressor test (CPT). Latigo says it has refined its CPT methodology into a quantifiable and repeatable clinical endpoint that has translated into clinical trial outcomes for its lead product candidate LTG-001.

In its registration statement, Latigo acknowledged the first-in-class non-opioid, non-addictive selective pain signal Nav 1.8 inhibitor that reached the market last year—Journavx^® (suzetrigine), a sodium channel blocker marketed by Vertex Pharmaceuticals and consisting of a 100mg loading dose and 50mg maintenance dose. Journavx is indicated for the treatment of moderate to severe acute pain, including postoperative pain, in adults.

During the first quarter of 2026, more than 350,000 prescriptions of Journavx were filled, generating $29 million in revenue—more than 22 times the $1.3 million recorded in Q1 2025, soon after the drug’s launch in early March of last year. For all of 2025, more than 550,000 prescriptions for Journavx were written, generating $59.6 million in revenue. Vertex is set to release second quarter results on August 3, after the close of financial markets.

Since the launch of Journavx, more than one million prescriptions have been filled for the drug across hospital and retail settings for a broad range of acute pain conditions, according to Vertex.

“Despite the availability of multiple therapies for pain management, a substantial proportion of patients continue to experience inadequate pain control,” Latigo stated in its IPO registration filing, adding that Journavx “represents a safer non-opioid alternative, but is limited by efficacy, slow onset and contraindications.”

Journavx’s label includes one contraindication: Concomitant use with strong CYP3A inhibitors.

‘Critical unmet need’

“These constraints prevent adequate pain relief and force clinicians to balance incomplete analgesia against dose-limiting AEs [adverse events], addiction and contraindications. Consequently, current pain management strategies are frequently multi-modal, requiring patients to receive multiple classes of medications to achieve acceptable pain management,” Latigo added.

Even with such approaches, Latigo asserted, outcomes remain suboptimal. The company cited the findings of a 2015 study showing that up to 54% of patients with OA reported inadequate pain relief (IPR) despite taking prescription pain medications.

“This highlights the critical unmet need for safer, more effective, non-addictive pain alternatives,” Latigo stated in its IPO filing.

A total 343 patients were randomized 1:1:1:1 to low dose LTG-001 (300 mg loading dose, then a maintenance dose of 150 mg every 12 hours); high dose LTG-001 (450 mg loading dose, 300 mg maintenance dose every 12 hours), and an opioid comparator, oral hydrocodone bitartrate-acetaminophen (HB/APAP, commonly known as Vicodin; 5 mg hydrocodone bitartrate and 325 mg acetaminophen), or oral placebo (every six hours).

The researchers acknowledged limitations that included:

Evaluation of LTG-001 as monotherapy rather than the multimodal manner in which acute pain is managed in clinical practice: “The magnitude of effect within a combined pain-management approach is unknown.”
Patients with chronic pain conditions and previous use of opioids were excluded, though the vast majority of abdominoplasties are carried out in women, a reality reflected in the trial population.

“Further research is needed to confirm a potential effect of sex on the size of the treatment effect with LTG-001,” the researchers wrote.

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Elix and University of Vienna Aim to Advance Drug Discovery Using AI

Edusehat — Thu, 30 Jul 2026 08:40:09 +0700

Tokyo-based Elix and the University of Vienna signed a joint research agreement aimed at advancing drug discovery using AI technologies.

One of Elix’s business models is the provision of its integrated AI drug discovery platform, Elix Discovery, developed under the concept of “medicinal chemists can truly use it.” The product comes with an intuitive graphical user interface (GUI) that automatically constructs predictive models for optimal compound profiling. Elix says it also features diverse structure generation capabilities, with a strength in “proposing structures that humans would not conceive.”

By integrating curated structure generation models, including proprietary ones, with predictive models and parameters built into the intuitive GUI, researchers can rapidly and intuitively refine molecular design, notes a company spokesperson, adding that platform supports both ligand-based drug design (LBDD), including pharmacophore modeling and structure-based drug design (SBDD), the latter utilizing docking simulations and other methods, thus enabling exploration across a broader range of approaches.

The second business model focuses on collaborative drug discovery research with pharmaceutical companies, biotech ventures, and academia.

Led by Julien Orts, MSc, PhD, associate professor at the University of Vienna, this research group specializes in NMR spectroscopy techniques to decode the atomic-resolution structures, dynamics, and interactions of biomacromolecules. The lab’s primary focus involves studying protein conformational switches and allostery in signaling, utilizing groundbreaking methodologies they developed such as INPHARMA for validating small-molecule binding modes and NMR for the automated determination of protein-ligand structures.

INPHARMA is a European research and training network focused on improving drug formulation processes, enhancing patient safety, and reducing animal testing in pharmaceutical development.

By applying exact nuclear Overhauser enhancement (eNOE) distance measurements with 0.1 Å accuracy to resolve protein ensembles, scientists in the Orts Lab note that they provide the thermodynamic insights necessary to tackle undruggable targets and advance modern structure-based drug design.

In this joint research project, Elix will collaborate with the Orts research. The parties see this arrangement as merging Elix’s proprietary expertise in chemoinformatics-based approaches and AI-driven molecular generation with the Orts group’s capabilities in structural dynamics and NMR-validated molecular interactions.

The collaboration aims to design and develop novel compounds against traditionally undruggable targets, with a specific focus on intrinsically disordered proteins (IDPs) and proteins implicated in epigenetic signaling and cancer. By integrating AI’s predictive power with experimental atomic-resolution data, the project seeks to bypass the limitations of traditional drug discovery, delivering new therapeutic insights and next-generation candidates for complex diseases, according to the researchers.

We are thrilled to partner with Elix to bridge the gap between advanced structural biology and artificial intelligence. My laboratory has always been driven by the desire to push the boundaries of what NMR can achieve in drug discovery,” says Orts. “By combining our ability to resolve protein dynamics at atomic precision with Elix’s sophisticated AI-driven generation, we can move beyond static structures and begin to target the complex, transient behaviors of proteins that were once considered out of reach. This synergy is exactly what is needed to accelerate the discovery of transformative medicines for the next generation.”

“At Elix, our mission is to rethink drug discovery by bridging cutting-edge AI with experimental innovation,” points out Shinya Yuki, PhD, CEO at Elix. “Collaborating with the [Orts] group allows us to pursue this mission on a global scale, uniting expertise in AI drug discovery with structural biology. We believe this partnership will open new possibilities for targeting diseases that have long remained beyond the reach of traditional drug discovery.”

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Celonic and Leukocare Collaborate to Support Complex Biologics and Advanced Drug Development

Edusehat — Thu, 30 Jul 2026 05:00:12 +0700

Celonic, a Swiss-based biologics CDMO, and Leukocare, which provides drug product development services, entered into a collaboration to support biopharmaceutical companies developing increasingly complex biologics.

The deal brings together Celonic’s expertise in cell line development, drug substance process development, intensified bioprocessing, and GMP manufacturing with Leukocare’s data science-driven approach to drug product development. By combining complementary scientific capabilities, the companies plan to support selected client programs involving complex biologics, including bispecific antibodies, multispecific antibodies, fusion proteins, and high-concentration formulations.

Celonic offers cell line and process development, process optimization, and clinical and commercial GMP manufacturing for advanced biologics. Company officials say its CHOvolution^® GS CHO‑K1 platform, supported by advanced transposase‑enabled integration, provides strong expression performance and is suited for both standard mAbs and complex molecules such as bispecifics and multispecific constructs.

Celonic is also a specialist in intensified and perfusion bioprocessing, having implemented next‑generation continuous and semi‑continuous upstream strategies that reportedly enable higher productivity, improved product quality, and more robust manufacturing outcomes, according to a Celonic spokesperson, adding that these intensified processes are particularly advantageous for bispecifics, multi‑domain constructs, and high‑concentration biologics.

Leukocare specializes in data science-driven drug product development for biologics and advanced therapeutic modalities, combining molecular modeling, predictive analytics, advanced biostatistics, Design of Experiments (DoE), and formulation expertise.

Celonic and Leukocare report that they will work together on client programs where their complementary expertise could create additional value. By considering formulation strategy, molecular characteristics, manufacturability, and process development in parallel, the companies aim to help clients establish strong development strategies at every stage of development from preclinical through to commercialization.

This integrated scientific approach enables a comprehensive understanding of each molecule and supports the development of drug products that are optimized not only for stability and manufacturability but also for scalable production and patient-friendly administration, explains Samanta Cimitan, CEO of Celonic.

“Our collaboration highlights the decisive role of formulation in enabling complex biologics to reach the clinic,” continues Cimitan. “By combining Celonic’s CHO‑based development, intensified and perfusion bioprocessing, and GMP manufacturing capabilities with Leukocare’s development expertise, we create measurable value for clients developing complex molecules, bispecifics, and high‑concentration drug products.”

“As biologics become increasingly sophisticated, formulation development must become more predictive, data driven, and closely connected with process development,” adds Michael Scholl, CEO of Leukocare. “Our collaboration with Celonic brings together complementary expertise that enables clients to make well-informed development decisions earlier, mitigate technical risk, and accelerate the path toward robust and manufacturable drug products.”

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Landmark Phase III Trial Finds Cell Therapy Slows Muscle Decline in Advanced DMD

Edusehat — Thu, 30 Jul 2026 05:00:11 +0700

Duchenne muscular dystrophy (DMD) is an X-linked genetic disease that causes the muscles—including the heart—to gradually weaken and waste away. Affecting almost exclusively boys and young men, most patients lose the ability to walk as the disease progresses and come to depend on their arms and hands for everyday tasks and independence. There is no cure.

Deramiocel, a heart-derived cellular therapy consisting of human allogeneic cardiosphere-derived cells, improved cardiac and skeletal muscle function in Phase I–II studies of DMD. Now, a new study reporting on Phase III trial data shows that deramiocel could slow muscle weakening in boys and young men with advanced DMD, and may also slow heart damage in those who already have heart muscle disease.

These Phase III clinical trial data are published in The Lancet in the paper, “Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial.”

This report is the first Phase III trial of a cell therapy made from donor cells and administered through the bloodstream to treat a genetic disease, and the first such trial in boys and young men whose DMD is already advanced.

The HOPE-3 trial involves 106 boys and young men aged 10 to 22 with advanced DMD, who were treated at 20 trial sites across the U.S. They were randomly assigned to receive either deramiocel (54 people) or a placebo (52 people), given as a drip into the bloodstream every three months for a year at an outpatient clinic.

After one year, participants given deramiocel were losing the use of their arms more slowly than those given the placebo. Their overall arm movement declined about 54% more slowly than in the placebo group, and their elbow movement about 65% more slowly. Across all participants, the therapy made no clear difference to how well the heart pumped blood. Among the 64 participants who already had heart muscle disease and had suitable heart scans, heart function was better preserved with deramiocel than with placebo. In a smaller group of 22 participants whose scans could be compared before and after treatment, deramiocel was also linked to less spread of heart scarring, but further research is needed to confirm this finding.

The therapy was generally safe, and no deaths were reported during the trial. Allergic-type reactions were more common with deramiocel (42%) than with placebo (15%). Almost all side effects were mild or moderate and cleared up within a day or two. The most common were headache, cough, fever, nausea, and a fast heartbeat.

The authors say deramiocel has the potential to help people with any type of DMD mutation, because it targets the swelling and scarring the disease causes in the muscles rather than the gene mutation itself. Heart problems are a major cause of death in DMD, and the authors call for further studies to find out whether the heart benefits seen in this trial help people live longer.

The U.S. Food and Drug Administration (FDA) is scheduled to discuss deramiocel at an advisory committee meeting on July 29, 2026, with a decision on whether to approve the therapy expected by August 22, 2026.

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Base Editing Strategy Alleviates Huntington’s Disease in Mice

Edusehat — Thu, 30 Jul 2026 05:00:10 +0700

According to at least one estimate, approximately 41,000 people in the United States have symptoms of Huntington’s disease, and more than 200,000 are at-risk of inheriting the disease. Symptoms of the neurodegenerative disorder, which is caused by a CAG expansion within exon 1 of the huntingtin gene, include personality changes, unsteady gait and involuntary movements, slurred speech, and more. Currently, there are multiple efforts underway to develop effective treatments, some of which are in clinical trials, that could slow or stop disease progression.

One of those is an approach that uses in vivo base editing to precisely edit a portion of the gene that causes Huntington’s disease. When tested in mice, the CRISPR tool, which was designed by scientists at the University of Illinois Urbana-Champaign (UIUC), reduced toxic protein fragments and symptoms associated with the disease. Details of the method are published in a new Nature Biomedical Engineering paper aptly titled “In vivo CRISPR base editing for treatment of Huntington’s disease.”

The work was led by Pablo Perez-Pinera, MD, PhD, and Thomas Gaj, PhD, both associate professors in UIUC’s department of bioengineering. Rather than using CRISPR to turn the Huntington gene off, the team designed base-editors that alter a specific point of the gene—the mutation that makes the Huntington protein prone to being cleaved into the toxic fragments that gradually kill brain cells. Specifically, the base editors that they used “generate proteolysis-resistant HTT isoforms by disrupting the splice acceptor of HTT exon 13, an exon that encodes critical proteolytic cleavage sites implicated in N-terminal fragment production,” they wrote in the paper. This change enables the cell’s machinery to skip that small section.

Pablo Perez-Pinera, MD, PhD, (left) and Thomas Gaj, PhD, (right) both associate professors in the bioengineering department at UIUC, are lead authors on the study. [University of Illinois Urbana-Champaign]

“Our base editors were developed to target the region of HTT that, when cleaved, can initiate the chain of events that leads to the toxic fragments,” Gaj said. “The result is that instead of turning the protein off completely, we alter how the gene is read so that the most damaging protein fragments are not produced.” It’s a different way of thinking about using gene editing to treat Huntington’s disease, Perez-Pinera added. “Instead of inactivating the protein completely or targeting collateral pathways, we introduce a very small edit in the gene that changes how the protein is processed by the cells.”

For the study, the scientists designed and screened more than 140 base editors to identify options that best targeted the exon of interest with the fewest unintended effects. They then injected them into the brains of mice with mutant HTT genes using AAVs as the delivery vehicle. Their analysis of the mice showed that those that received the treatment accumulated fewer toxic protein fragments, had fewer symptoms, and had less degeneration within the brain than untreated mice.

As part of their next steps, the scientists plan to evaluate the lead HTT exon 13-skipping editors in humanized mice models to assess the tolerability of the treatment and determine whether editing reduces the wild-type HTT below a tolerated threshold. They also plan to evaluate “target engagement and tolerability in large animals across a range of doses to define the therapeutic window and guide future dose selection,” according to the paper.

Other plans are to refine delivery of the base editors to the brain, to make it less invasive and less reliant on viruses for transport, said Kyrollos Shenouda, a graduate student at UIUC and one of the authors on the paper. “We’re also interested in adapting this approach to target other regions of the HTT gene to decrease other toxic aspects of the protein,” Shenouda said.

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Genetic Study of Fibromyalgia Points to Neurological Basis

Edusehat — Thu, 30 Jul 2026 05:00:09 +0700

An international team of researchers has identified multiple new genetic risk factors associated with fibromyalgia, a syndrome characterized by widespread pain and tenderness, fatigue, and problems with sleep, memory and mood.

The team analyzed genetic data from more than 2.5 million adults, of which 55,000 were fibromyalgia patients. They identified DNA sequence variants in 26 regions of the genome that affect the risk of developing fibromyalgia. Many of the genes implicated in these regions are involved in brain and nerve function.

The results provide the strongest evidence yet that fibromyalgia is primarily a nervous system disorder rather than an autoimmune disease, as has long been debated. “This work changes how we think about fibromyalgia at a fundamental level,” said Michael Wainberg, PhD, an investigator at the Lunenfeld-Tanenbaum Research Institute, part of Sinai Health, and the University of Toronto. “For decades, patients have been dismissed or told their pain is simply psychological. Our findings confirm the condition has a clear biological basis.”

Weinberg is co-senior author of the researchers’ published paper in Nature Medicine, titled “The genetic architecture of fibromyalgia across 2.5 million individuals,” in which they concluded, “This study provides robust genetic evidence defining fibromyalgia as a central nervous system disorder, thereby establishing a biological framework for its complex pathophysiology and extensive clinical comorbidities.”

Fibromyalgia is a multifaceted syndrome that can encompass chronic widespread musculoskeletal pain, fatigue, sleep issues, cognitive impairment, and somatic symptoms, the authors stated. Fibromyalgia also commonly co-occurs with other pain conditions, including irritable bowel syndrome, chronic fatigue syndrome, autoimmune and neuropsychiatric disorders, and metabolic syndrome. Despite affecting about two percent of the global population, its existence has been debated, largely because its biological causes have remained unclear. “Whether fibromyalgia has an autoimmune component is a matter of long-standing debate,” the authors added.

Bringing together data from 11 health research studies from the U.S., U.K., Finland, Estonia, Denmark, and Iceland and 53 researchers across seven countries, the newly reported study was jointly led by Weinberg and collaborators at Fred Hutch Cancer Center and University of Washington in Seattle, and at the University of Helsinki in Finland and Massachusetts General Hospital in Boston.

The team conducted a multi-ancestry genome-wide association study meta-analysis across 2,563,755 individuals (54,629 cases and 2,509,126 controls) from 11 cohorts. They scanned millions of genetic differences of individuals with and without fibromyalgia to find changes that were more common in those with the condition. Their results identified DNA sequence variants in 26 regions of the genome that affect the risk of developing fibromyalgia. Many of the genes implicated in these regions are involved in brain and nerve function. The variant most strongly linked to fibromyalgia risk was a coding variant within the HTT gene. Other mutations in this gene cause Huntington’s disease (HD), a severe, progressive and fatal neurodegenerative disorder.

Another variant pointed to a receptor called GPR52 that regulates HTT levels. This receptor is already being investigated as a possible drug target in Huntington’s disease. “Our strongest association (~9% increased risk of fibromyalgia) was with a common coding variant in HTT, the causal gene for HD, although this variant is distinct from the rare repeat expansion that causes HD,” the investigators wrote. “The variant results in the deletion of a single glutamic acid residue in the HTT protein. We also observe an association near GPR52, a regulator of HTT.”

By integrating their findings with a massive dataset of 20 million cells from various tissues, the researchers found further evidence for a neurological origin of fibromyalgia. Genes near fibromyalgia genetic risk factors were more active in nervous system cells than in other types of cells, which sets fibromyalgia apart from classical autoimmune conditions. “Overall, our results suggest that fibromyalgia is not primarily an autoimmune disorder, although it may nonetheless have a peripheral immune and/or neuroimmune component,” they stated. “Power to detect this may have been limited by the predominantly European composition of our sample and by healthy participant bias in biobank cohorts.”

The study also revealed substantial genetic overlap between fibromyalgia and a range of other conditions, including low back pain, irritable bowel syndrome, and post-traumatic stress disorder. “Fibromyalgia showed strong, positive genetic correlation with a wide range of chronic pain, psychiatric and somatic disorders, including genetic correlations above 0.7 with low back pain, post-traumatic stress disorder and irritable bowel syndrome,” the scientists stated.

They think that shared biological mechanisms within the nervous system may make people susceptible to several of these conditions, explaining why they often appear together. “We know that chronic pain syndromes cluster together in individuals and families and are genetically similar,” said co-author Frances Williams, PhD, a rheumatologist at TwinsUK, King’s College London. “Targeting the shared mechanisms underlying them could potentially benefit a whole cluster of disorders.”

Even so, the study found that genetics is not the main determinant of whether someone develops fibromyalgia. The authors suspect that even people carrying many fibromyalgia genetic variants likely require another risk factor, such as a painful arthritic condition, to trigger fibromyalgia syndrome. “Understanding how genes, environmental exposures, and life events jointly contribute to risk of fibromyalgia syndrome is critical,” said co-senior author Nasa Sinnott-Armstrong, PhD, assistant professor at Fred Hutch Cancer Center. “Further research into triggers of fibromyalgia and corresponding changes to neural tissues will help understand what drives fibromyalgia and how to treat it.”

Despite fibromyalgia being diagnosed roughly three times more often in women than in men, the researchers did not find any genetic differences in risk between the sexes. This suggests that the higher prevalence in women could be driven by non-genetic factors, such as hormonal or environmental, or differences in pain sensitivity and diagnostic patterns.

The findings do not mean that fibromyalgia can now be diagnosed with a genetic test, nor do they immediately lead to a new treatment. However, they provide important new starting points for understanding the biology of fibromyalgia that will help guide future research into better diagnosis and treatment.

Williams added, “This study provides important new insights into why some people develop fibromyalgia syndrome and identifies biological pathways that could lead to new treatment approaches. One of these pathways is already the focus of drug trials for Huntington’s disease, raising the possibility that existing pharmaceutical research could eventually benefit people with fibromyalgia. The findings also help us better understand why fibromyalgia so often occurs alongside conditions such as anxiety and depression, bringing us closer to understanding the condition as a whole.”

In summary, the team stated, “Our study maps the genetic architecture of fibromyalgia, identifying 26 risk loci and providing robust genetic validation of the notion that fibromyalgia is primarily a central nervous system disorder. Identifying specific risk loci provides the field with concrete molecular starting points, enabling hypothesis-driven studies of pathophysiology and shared etiology with comorbid conditions.”

The study’s researchers have founded the Chronic Pain Genomics Consortium (https://paingenomics.org) to investigate other chronic pain syndromes, starting with pelvic pain. The consortium sees fibromyalgia as only the beginning of a broader exploration of the landscape of chronic pain conditions.

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Sage Inks Licensing Deal with Causaly’s AI Platform Focusing on Drug Discovery Research

Edusehat — Thu, 30 Jul 2026 01:25:15 +0700

In a new publishing deal, Causaly and Sage have announced a partnership to bring full-text scientific peer-reviewed literature into the world of AI-powered drug discovery research.

According to Sage vice president Katie Metzler, who leads the publisher’s global licensing team, this deal follows a similar partnership that Sage struck in January 2026 with Consensus, an AI workspace for scientific research based in San Francisco. The deal also marks Causaly’s first partnership with a major STM publisher.

“It’s an example of a broader shift happening with AI agents reading the full text of journals and providing human researchers with the sections they need, showing them how ideas are connected via their knowledge graph, saving time over traditional search and discovery methods and driving new insights that they may never have found via a traditional lit review methods.”

Headquartered in London, Causaly’s mission is “to accelerate discovery in life sciences through transformative AI technologies,” offering new ways “to find, visualize and interpret biomedical knowledge and automate critical research workflows.”

The newly announced partnership grants Causaly AI agents access to digest the full text of peer-reviewed papers from a group of some 400 Sage journals. Relevant insights from those articles are served directly within the Causaly platform, helping mutual customers “unlock deeper evidence” from their existing institutional licenses.

“Sage has always been committed to connecting researchers with knowledge in the most useful and meaningful way,” says Bob Howard, executive vice president, global journals at Sage. “With Sage content deeply integrated inside Causaly’s agentic AI platform, we can extend our reach and impact where researchers do their important work. Access and intelligence go hand in hand, and this partnership puts them in one place.”

“Partnering with Sage gives our customers something they have been asking for: a single platform that brings together evidence and governed scientific reasoning,” said Marco Costa, COO at Causaly, in a press release. “When our agents read the full paper first and surface the most relevant evidence and insights right in scientists’ workflows, our customers can research with more confidence and get answers faster.”

Causaly selected some 400 journals from Sage’s portfolio of more than 1,500 research journals. While Sage’s traditional strength is in social sciences, it buttressed its biomedical research strength with the 2025 acquisition of Mary Ann Liebert—the founding publisher of GEN. Liebert published more than 100 peer-reviewed titles, many of which are included in the new partnership.

“Causaly’s powerful AI platform, combined with trusted scientific content from Sage and Mary Ann Liebert journals, enables researchers to uncover insights faster and make more informed decisions,” Howard told GEN. “The future of drug discovery will be shaped by partnerships that combine authoritative scientific knowledge with domain-specific, agentic AI. Together, we are helping to make that future a reality, delivering greater value to our shared pharmaceutical customers and accelerating the pace of scientific discovery.”

Drawing upon the full text of Sage journal articles, including the methods, results, tables, and supplemental data, Causaly’s AI agents surface a comprehensive picture of the study.

The Sage journal full-text integration is now available to all Causaly customers as a separate add-on to their existing platform subscription. Sage subscribers can link straight from Causaly’s extracted evidence view to the full article on the Sage website. Causaly customers without a Sage subscription can view a snapshot of the full-text article, along with an in-platform pathway to purchase the article on Sage’s platform. By running its full-text relevance analysis before any link-out, Causaly customers can assess the value of a given paper before reading or purchasing it.

RAGs, not training

Metzler manages Sage’s global licensing team that was involved in negotiating this and other retrieval augmented generation (RAG) licensing deals. She gave GEN some background on the partnership and its broader significance.

It is important to distinguish between AI licensing for training and RAG deals, Metzler says. “There are two kinds of AI licensing. There’s licensing for training, where the content is used to train the underlying model. Then there’s licensing for RAG, which does not allow training of the underlying foundation model but instead allows the licenser to create a vector database of the content—embeddings of our content—then the agents retrieve snippets of the content to display to users.”

RAG deals with other companies are in negotiation, Metzler says. “From the Sage perspective, this is a story about how discovery is changing, how the behavior of researchers as a result of AI is changing. Increasingly, the starting place for a researcher’s journey is not on a journal platform or a Google search page, but instead on some kind of AI-powered natural language tool. Our strategy around discovery needs to evolve, so licensing is just one part of that.”

Like most other publishers, Metzler says that Sage is assessing how AI is changing different parts of the discovery process—how researchers find content, how they access it, ensuring that that access is rights compliant and that publishers are protecting the rights to published content. “There is also the trust piece,” she said. “How does AI change in a discovery context, how do you know what to trust when you find it through an AI-mediated platform?”

Publishers also have to consider the measurement of that usage. “How does that look different than it did in a world where we were thinking about organic web search and library discovery services? Those channels still exist, but there’s now a number of mediators that are changing that picture.”

Spirit of learning

Like Sage, Metzler said Causaly is entering this partnership in the spirit of learning. “We are both learning together about what the impact is on the value that they’re able to offer their customers and also the referrals or usage that drives to our content. It feels like this is a partnership, not just a licensing arrangement. I think there is a lot to learn on both sides.”

“We’re still as an industry figuring out what good looks like, in terms of these answer engine referral relationships, because there is this fear that you end up in a zero-click-world where nobody ends up clicking through to the full text and everyone just gets delivered answers in their AI tool. So you start to see usage really degrade. But there is also the possibility that this generates a new value that could drive traffic from places that we’re not currently realizing that value…We need to learn about what this new discovery path looks like.”

Some authors, Metzler acknowledges, may have concerns about their content being used in this way. She notes that the industry could do a better job of educating authors in this regard. “People hear AI and think of big tech companies gobbling everything up without permission or payment, using it to train their [large language models] and then capturing all that value for themselves and not giving any of that back. To be clear, I also think that’s terrible. I don’t think that’s good for authors. We don’t want that to be the expectation or the future.”

The Causaly deal is not AI training, but there is a misperception that everything involving AI is about training. “The first reason for authors to want us to participate in licensing on their behalf is because it is a way for us to push back against the granting of broad copyright exceptions for training, which we don’t think is the right thing for the creative industries and for academic publishing,” Metzler says.

A second argument, more specific to RAG, is that this is a part of discovery and the future of how content will be discovered. Metzler says: “What I would say to authors is, ‘we’ve all worked so hard, spent our lives producing all of this incredible science, and the way people are consuming that science is changing. AI is now going to be a part of that. If we want our content to be used and to be useful, and if we want to realize the benefits that are being promised from this ‘AI future’ that you may or may not have asked for, then we do need to participate.”

Metzler says her team is talking to start-ups that are competing with some of the big tech players. “I don’t want to see a future where there’s literally only three tools out there. I think that researchers should have choice between a range of different tools.” Domain-specific companies like Causaly are thinking about how to serve as a trust layer.

“It’s about discovery, access and trust. There are going to be more and more examples of those layers of trust being built around both trusted content, but also the technology layer that adds that additional layer of trust. People want to use AI, but they don’t want to trust their clinical decision making to Claude or ChatGPT,” Metzler says. “They want to be using tools that they think are more likely to be trusted.”

Causaly closed a $60-million Series B round in July 2023 and currently has more than 120 employees, including scientists with experience deploying AI in pharma R&D. Causaly’s core customer base includes leading biotech and pharma companies. Causaly says a dozen of the top 20 global pharmaceutical companies use its platform to accelerate drug discovery, including Novo Nordisk, Novartis, Takeda, Ipsen, and J&J.

Additional deals are likely in the wake of the Sage announcement. Earlier this year, Wiley struck a similar deal with OpenEvidence to bring Wiley’s medical content into the OpenEvidence platform.

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Genome Mining Points to Less Toxic, More Potent Polyene Antifungals in Mice

Edusehat — Thu, 30 Jul 2026 01:25:14 +0700

Fungal infections are becoming an increasingly difficult clinical and global threat, driven in part by antimicrobial resistance and challenges in developing new antifungal treatments. Polyenes, a powerful class of antifungal drugs that includes amphotericin B and nystatin A1, remain among the most effective options for some life-threatening infections. But their usefulness is limited by significant toxicity and poor solubility, because fungal and human cells share features that make it difficult to kill pathogens without harming healthy tissue.

Now, researchers at Imperial College London and The University of Manchester say they have used genome mining and an enzyme-based approach to reshape polyene bioactivity, generating derivatives that showed improved antifungal activity and reduced toxicity in preclinical testing.

The work, described in “Enzymatic glycosylation and amidation reshapes polyene bioactivity,” began with a search for pathways capable of producing previously undescribed polyenes. The researchers then characterized enzymes involved in making and modifying the compounds, including glycosyltransferases that add sugar groups and an amidotransferase that can alter a carboxylate substituent associated with unfavorable properties to reduce toxicity while increasing potency.

“The most effective antifungal agent currently available is a polyene molecule called amphotericin produced by soil bacteria,” says first author Saadia Nasr Mirza, PhD, a postdoctoral research associate at The University of Manchester. “Although amphotericin is very potent, it is highly toxic, so we set out to discover if bacteria can produce different types of polyenes that are safer than amphotericin. We developed a bioinformatics pipeline, which surprisingly showed that many bacterial species have the capability to produce novel polyenes.”

The team used nuclear magnetic resonance (NMR) to determine the structures of recently discovered polyenes and built a library of derivatives for testing. “The addition of a second sugar, combined with carboxylate modification, leads to new polyene derivatives with increased antifungal activity, lower toxicity and higher solubility than the parent polyenes used at present in the clinic,” write the authors.

The researchers identified Nys34 as a leading candidate after it demonstrated three-to-eightfold lower toxicity than amphotericin B in multiple human cell lines and reduced fungal burden in a mouse model of invasive aspergillosis. While mice tolerated three repeat doses of the compound, toxicity was observed after a fourth dose, underscoring the need for further optimization and testing.

Senior author Jason Micklefield, PhD, professor in the department of chemistry at Imperial College London, says the team was encouraged by the activity of the derivatives. “We were pleased to find that several of the new polyene derivatives were more potent and less toxic than amphotericin and nystatin, which is another important polyene that is also used in the clinic,” he said. “The key advance was the use of these methods to find biosynthetic gene clusters that produce polyenes with multiple sugars, which can have a profound effect on bioactivity. Most of the existing polyenes have just one sugar moiety.”

The researchers also reported that Nys34 appeared to act differently from amphotericin, a finding that could be important if confirmed in further studies. “We were particularly surprised that one of polyene compounds we developed, Nys34, has a different mechanism of action to the existing polyene drugs,” Micklefield told GEN. “Polyenes are thought to target components of the cell membrane rather than a specific enzyme like most antimicrobial drugs. This means that a simple mutation in one enzyme cannot confer resistance, but rather the makeup of the cell membrane must be altered (or the drug degraded by an enzyme). The fact that Nys34 acts via a different mechanism to amphotericin is a promising sign that it can evade resistance.”

Beyond the individual compounds, the study highlights an alternative approach to improving polyene drugs. Because polyenes are structurally complex, modifying them through conventional chemistry can require “many steps, extensive use of protecting groups, and deleterious reagents,” write the authors. The authors point to a recently reported amphotericin B derivative with reduced toxicity that required 12 chemical steps with a 0.7% overall product yield, illustrating the challenges of optimizing these molecules through traditional synthesis.

“In future work, we aim to explore Nys34’s mechanism of action. Also, the methods we have developed here are broadly applicable across a wide variety of polyene scaffolds and substrates (e.g. different sugars). We are currently exploring more combinations of these modifications to identify additional promising drug candidates,” added Micklefield.

By contrast, this new approach could allow researchers to generate modified polyenes through fermentation or enzymatic routes. The team suggests that such an approach may be more scalable and cost-effective than conventional multistep synthesis, though additional work will be needed to determine whether the platform can support development and manufacturing beyond the laboratory.

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Capsid Experts Hope to Slash the Cost of Gene Therapies

Edusehat — Thu, 30 Jul 2026 01:25:13 +0700

A company with expertise in capsid discovery say they have boosted adeno-associated virus (AAV) yields ten-fold compared to the industry standard. Affinia Therapeutics believes that, by adopting their methodical approach to AAV manufacturing, the industry could improve patient access to gene therapies by dramatically cutting costs.

“Often in industry articles, you hear how manufacturing costs remain a barrier to making [AAV] a more utilizable platform,” explains Rob May, chief technical operations officer at Affinia.

“Our message is that this really isn’t the case. The quality and manufacturability to reduce the cost of goods are there if you use all the [techniques] available today.”

According to May and colleague Matt Edwards, head of process science at Affinia, the company carefully screened for what they felt was the best commercially available cell line for AAV manufacturing.

Subsequently, they picked the best from a large selection of transfection reagents and, by redesigning the plasmids used for transient transfection, were able to dramatically increase the yields of AAV.

“We looked at a lot of different levers we could pull,” Edwards explains. “But much of the most impactful work we’ve done is on the plasmids and how they’re designed.”

The company says their boost in yields means they can now manufacture AAVs in a 50-liter rather than a 500-liter bioreactor. A smaller bioreactor, Edwards explains, is cheaper to run.

Affinia is now in negotiations with half a dozen companies to license their technology, May says. “We’ve presented this technology multiple times and had people come to us and say ‘Hey, we want to learn more’.”

He adds, “And then, what we want to try to do is for people to have the technology in their own hands, as they have their own manufacturing platforms, cell lines, and ways of doing things.”

Going forward, Affinia hopes today’s technology will help revolutionize the industry, allowing AAVs to be produced in the low single-digit thousands of dollars, rather than upwards of hundreds of thousands of dollars, as can happen currently.

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Biopharma Hungry for GLP-1 Receptor Agonist Manufacturing Skills

Edusehat — Thu, 30 Jul 2026 01:25:11 +0700

Demand for engineers with GLP-1 receptor agonist (RA) manufacturing skills is increasing as the market for the popular anti-obesity drugs continues to grow, according to Ireland’s NIBRT, which has set up a training course to help meet the need.

The new course will cover biosynthetic and chemical production routes for peptide-based therapies as well as quality, regulatory, and supply chain considerations.

John Milne, PhD, NIBRT’s director of bioprocess training, tells GEN that development of the course was prompted by predictions about the substantial growth worldwide for GLP-1 receptor agonists over the next decade.

“These medicines have evolved quickly from niche drugs to treat diabetes when first launched to, now, blockbuster status for addressing type 2 diabetes, obesity, and cardiovascular health.

“Ireland can continue to play its part with respect to the ongoing and future manufacturing of these therapeutics, building on the track record of manufacturing sites, such as Lilly’s campus in Kinsale, which has contributed greatly to the global supply of its own GLP-1 RA portfolio.”

Ireland’s critical role in GLP-1 RA production was underlined in March when Novo Nordisk said it would make an oral version of Wegovy at its campus in Athlone, County Westmeath.

Such investments also helped convince NIBRT Ireland was an ideal location for a dedicated training course, Milne says.

“Building on the undoubted interest that exists in these therapeutics and helping trainees to contextualize these treatments from both a medical and a manufacturing perspective were important in our decision.”

Chemistry and biotech

Making GLP-1 RAs is a complex undertaking involving synthetic chemistry and protein expression, both of which will be covered by the new course, Milne says.

“Currently, the largest class of approved treatments globally are peptide-based GLP-1 RAs where the active target peptide is chemically synthesized, coupled to a long chain fatty acid with the resulting material being purified using chromatographic and filtration operations, prior to formulation and final filling.

“In other products, the active peptide can also be manufactured using recombinant technologies by expressing the active peptide in a cell-based system such as yeast cells or mammalian cells, followed by subsequent purification and formulation,” he says.

More recently, developers have advanced oral peptide- and non-peptide-based GLP-1 RAs—notably Eli Lilly’s Foundayo—which has further expanded the skill set production engineers require, according to Milne.

“In general terms, there is some overlap in the skill sets required to manufacture GLP-1 RAs, but there are also some obvious nuances specific to GLP-1 RAs. As an example, most GLP-1 analogues approved to date are administered by injection and hence are manufactured using similar fill and finish platforms and processes that are seen for other traditional biologic medicines.

“With the upsurge in manufacturing demand globally, there will undoubtedly be more facilities constructed by innovator companies and indeed more contract manufacturing organizations partnering with industry to address supply constraints. New personnel will be needed, and sites will need to be repurposed to manufacture these products, in what will be a very competitive market space,” he adds.

Efficiency

Predictably, in such an environment, manufacturers are looking at ways of making production more efficient, Milne says, citing automation and AI as examples.

“Both automation and AI are becoming increasingly important with respect to improving capacity, quality, and reducing costs. As with most biomanufacturing operations, automation is fundamental to commercial GLP-1 production.

“Most commercial peptide synthesis systems are now fully automated and would include real-time process monitoring and control systems. This helps maintain GMP compliance, improve reproducibility, and robustness.”

He continues, “AI adoption is less mature but is growing across biomanufacturing, and a future state where AI will be applied in process optimization, continuous manufacturing, creating digital twins, automated quality monitoring, and supporting predictive maintenance is likely to emerge.”

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Downstream Purification Platform Breaks Complex Vector Bottleneck

Edusehat — Thu, 30 Jul 2026 01:25:10 +0700

Downstream processing is becoming a bottleneck as biopharmaceutical manufacturing shifts to more complex bispecific antibodies, FC-fusion proteins, and adeno-associated viral (AAV) vectors. A purification platform developed by a team of 24 researchers at North Carolina State University removes that bottleneck by providing both high flow-through and superior impurity capture.

For biomanufacturers, the combination of process intensification and more complex vectors has meant higher upstream titers that increased both product- and process-related impurities, thus increasing the downstream processing burden. The ramification is that some high-risk impurities, such as proteases, lipases, redox-active enzymes, and chromatin-associated species, have slipped through conventional capture and polishing steps and have been implicated in batch failures, clinical holds, and product recalls.

A recent paper outlines the strategies Wenning Chu, PhD, research scholar and first author, and colleagues devised.

Industry-relevant options

For proteins derived from Chinese hamster ovaries (CHO), the platform consisted of a resin-based pre-capture step that combines peptide ligand technology and a size-exclusion matrix, followed by a Protein A capture step, and a single-use size-exclusion mixed-mode resin for polishing. It enabled “product yields exceeding 70%,” final product pool concentration of 19–23 g/L/hour—notably more than the industry standard or approximately 15 mg/mL, “and monomeric purity of approximately 99%.” Host cell protein clearance ranged from 4 to 11 ppm.

“By shifting the impurity clearance from a bind-and-elute polishing step to a modular flow-through operation, this approach decouples impurity removal from product capture and reduces the process sensitivity typically associated with modality-specific polishing development,” Chu and colleagues wrote. “The productivity gains…were substantial.”

For AAV purification, the team used a mixed-bed absorbent followed by a capture step using either a platform AAV resin or a single-use, high-capacity chromatography membrane. By enforcing a 1:3 charcoal-to-resin ratio and optimizing the load volume, this method achieved an approximate 50% AAV recovery and a host cell protein level of 350 ng/mL—less than 100 ng per dose.

This fit-for-purpose platform purifies a wide range of products and impurities with modular flexibility and, the researchers note, “enables targeting impurity classes that often persist through conventional platforms and are removed only at the expense of yield, while still sustaining high productivity.”

These flow-through and pseudo-affinity technologies expand downstream processing options, “enabling more efficient and reliable manufacturing of complex biologics….” The team considers them “industrially relevant” particularly in light of feed variability.

Further research may focus on operations at low residence times and productivity enhancements, as well as scale-up and good manufacturing practice considerations.

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Large Bioprinted Tissues Get a Precision Boost

Edusehat — Thu, 30 Jul 2026 01:25:09 +0700

Improving the cultivation of large bioprinted tissues could help make laboratory-grown tissue models more reliable for drug development and regenerative medicine. At the International Society for Cell & Gene Therapy (ISCT) 2026 annual meeting in Dublin, Elliot Cowles, a doctoral student in bioprocess engineering at Université Claude Bernard Lyon, and colleagues presented a perfusion-based culture system, which was described in Cytotherapy, that addresses this challenge. Although biofabricated tissues are increasingly used for in vitro research, many remain relatively small and are typically grown under static conditions that limit scalability and consistency.

The team’s approach combines custom-designed, 3D-printed components with an Ambr250 bioreactor to maintain tightly regulated culture conditions. The researchers validated the custom parts as autoclavable and leak-proof before using them to support an 8-cm² macroporous bioprinted tissue. Culture medium was continuously pumped through the tissue at 1.5 mL/min while the bioreactor regulated oxygen levels, temperature, and pH.

To better understand what was happening inside the tissue without damaging it, the researchers used high-resolution magnetic resonance imaging. The scans reconstructed the tissue’s three-dimensional structure and tracked how fluid moved through it. Rather than flowing uniformly, the liquid followed uneven pathways that differed from computer-aided design predictions, revealing a more complex internal environment than expected.

Over a 21-day culture period with this platform, temperature, dissolved oxygen, and pH remained stable across a broad range of oxygen settings. The team did detect a consistent difference between oxygen levels measured in the regulation vessel and those reaching the tissue chamber, but traced the discrepancy to oxygen entering the culture medium through flexible tubing. Still, the researchers successfully perfused three large mesenchymal stem cell–based tissues using minimal culture medium.

Cowles and his colleagues concluded that the new platform provides a controlled environment for cultivating larger bioprinted tissues while offering detailed, non-destructive monitoring of internal flow patterns. Looking ahead, they plan to improve quantitative flow measurements and develop computational fluid dynamics models capable of mapping local tissue microenvironments, with the goal of advancing reproducible large-scale tissue maturation.

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Sage Inks Publishing Deal with Causaly’s AI Platform Focusing on Drug Discovery Research

Edusehat — Wed, 29 Jul 2026 21:50:08 +0700

In a new publishing deal, Causaly and Sage have announced a partnership to bring full-text scientific peer-reviewed literature into the world of AI-powered drug discovery research.

Drawing upon the full text of Sage journal articles, including the methods, results, tables, and supplemental data, Causaly’s AI agents surface a comprehensive picture of the study.

RAGs, not training

Spirit of learning

Additional deals are likely in the wake of the Sage announcement. Earlier this year, Wiley struck a similar deal with OpenEvidence to bring Wiley’s medical content into the OpenEvidence platform.

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Nova Biomedical Acquires NanoCellect Assets to Expand Cell Line Development Portfolio

Edusehat — Wed, 29 Jul 2026 03:55:10 +0700

Nova Biomedical acquired certain assets of NanoCellect, which develops microfluidic cell sorting technology, to expand its capabilities in cell line development and strengthen support for biopharmaceutical workflows from early research through commercial manufacturing.

The acquisition adds NanoCellect’s WOLF G2^® cell sorting platform to Nova’s biopharma portfolio. The microfluidic-based technology enables gentle cell selection and isolation and complements Nova’s Solentim platform for clone generation, verification, and growth analysis, according to Nova Biomedical. Together, the technologies provide a more integrated workflow spanning cell selection, single-cell isolation, and clone verification.

By combining these capabilities, Nova aims to accelerate cell line development, improve clone selection, and create a stronger foundation for downstream process development, scale-up, and manufacturing.

The expanded portfolio also integrates with Nova’s existing analytical technologies for cell culture monitoring, including the BioProfile^® FLEX2 metabolite analyzer, OsmoTECH^® osmolality measurement systems, and the BioProfile^® FAST CDV cell counting and viability analyzer. These instruments provide critical process data throughout bioprocess development and manufacturing.

According to the company, the addition of NanoCellect’s technology advances its strategy of offering a connected portfolio of tools that support continuity, data-driven decision-making, and process optimization across the entire bioprocessing lifecycle.

“NanoCellect adds a critical upstream capability to our portfolio and strengthens our ability to connect key steps in cell line development,” said John Luck, president of Nova Biomedical’s Biopharma Business. “By integrating cell sorting with clone isolation and high-confidence verification, alongside analytical control points such as cell culture monitoring and osmolality, we are enabling customers to reach a successful clone more quickly and generate insights that help optimize clone selection and drive better performance during process development, scaleup, and manufacturing.

“This is an important step in building a more connected and differentiated platform for cell line development and bioprocessing.”

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Argenx Expands Immunology Portfolio with $2.2B Buyout of Forte Biosciences

Edusehat — Wed, 29 Jul 2026 03:55:09 +0700

Argenx has agreed to acquire Forte Biosciences for $2.2 billion, the companies said, in a deal intended to expand the buyer’s immunology portfolio with an early clinical stage candidate that has shown promising data.

Earlier this month Forte announced positive results for its lead candidate FB102, from a Phase Ib trial assessing the anti-CD122 monoclonal antibody in vitiligo. FB102 achieved a 29.6% mean Facial Vitiligo Area Scoring Index (FVASI) improvement from baseline at week 24 (p-value = 0.020), with statistically significant improvements seen as early as Day 64 after dosing (p=0.023), continuing through week 24 after completion of the 12-week treatment period.

FB102 also achieved 43.2% mean FVASI improvement from baseline at week 24 (p-value = 0.006) in subjects with greater disease involvement having baseline FVASI ≥0.75 (approximately one-quarter of face depigmented). That percentage increased at 58.8% at FVASI50 but fell to 23.5% at FVASI75.

Forte announced positive Phase Ib data for FB102 last year in celiac disease, with Phase II data expected to be released in the second half of this year.

The celiac disease and vitiligo studies were key drivers, Argenx said, of its decision to go from being a strategic investor in Forte to acquiring the company, since the clinical data covers indications with significant unmet need and limited treatment options. Argenx was among investors in Forte’s $150 million public offering (5,709,936 shares priced at $26.27), which closed in April.

Pipeline-in-a-product

Argenx CEO Karen Massey

As with Forte, Argenx reasons that FB102 is a pipeline-in-a-product because of its potential to treat alopecia areata and additional autoimmune diseases. FB102 adds to Forte’s pipeline a mechanism focused on pathogenic T-cell and NK-cell activity, which according to the company broadens its ability to pursue diseases driven by different dimensions of the immune system.

“The addition of FB102 to our portfolio aligns perfectly with the Argenx playbook: compelling biology, strong clinical validation and broad potential to address patient need,” Argenx CEO Karen Massey said in a statement.

Argenx says FB102 complements its antibody-based portfolio, which is led by marketed drugs Vyvgart^® (efgartigimod alfa-fcab) injection, indicated to treat adults with generalized myasthenia gravis (gMG); and Vyvgart Hytrulo^® (efgartigimod alfa and hyaluronidase-qvfc), a subcutaneous treatment combination of the neonatal Fc receptor blocker and endoglycosidase that is indicated for gMG and chronic inflammatory demyelinating polyneuropathy (CIDP).

Vyvgart and Vyvgart Hytrulo comprise a blockbuster franchise, having generated $2.813 billion in the first half of this year, up 62% from $1.739 billion in January-June 2025. The franchise racked up $4.151 billion in all of 2025, up 90% from $2.186 billion in 2024.

“The company is setting itself up for continued growth beyond the Vyvgart peak that is anticipated in the first half of the 2030s,” analysts at Van Lanschot Kempen wrote in a research note, as reported by Bloomberg News. “All in all, the right deal at the right time.”

40% surge

Forte’s investors appeared to share that upbeat sentiment, as the company’s shares traded on Nasdaq surged about 40% Monday on news of the deal, to $76.50 from $54.78 at Friday’s close. Argenx’s ordinary shares traded on Euronext Brussels slid 1.8%, to €789.80 ($895.67) from €804.40 ($913.24), while American Depositary Shares (ADSs) traded on Nasdaq fell 3%, to $888.82 from $918.22.

Under its generic name, efgartigimod is also being developed for ocular myasthenia gravis (oMG), primary immune thrombocytopenia (ITP), Graves’ disease, myositis and related diseases (Immune-mediated necrotizing myopathy or IMNM; Antisynthetase syndrome or ASyS; and diabetic myositis); Sjogren’s disease (partnered with IQVIA); systemic sclerosis; and antibody mediated rejection or AMR.

Also in Argenx’s pipeline is:

Empasiprubart (formerly ARGX-117), designed to be a humanized sweeping antibody that binds specifically to C2 in a pH- and Ca²⁺-dependent manner. Empasiprubart is in registrational studies for CIDP and multifocal motor neuropathy (MMN), and in proof-of-concept studies for delayed graft function (DGF).
Adimanebart, a MuSK-targeting candidate in proof-of-concept studies for congenital myasthenic syndromes (CMS) and spinal muscular atrophy (SMA).
Four Phase I candidates, all with undisclosed indications: ARGX-109 targeting IL-6, ARGX-121 targeting IgA, ARGX-124 targeting FcRn and ARGX-213, which also targets FcRn.

Three preclinical candidates: ARGX-118, a Galectin-10 targeting candidate for airway inflammation; ARGX-125, undisclosed target and indication; and TSP-101, also undisclosed target and indication.

Acquiring Forte will build upon Argenx’s Vision 2030 strategy, which sets goals of treating 50,000 patients globally with its treatments, securing 10 labeled indications across its approved medicines, and progressing five pipeline candidates into Phase III development, all by 2030.

“Our Vision 2030 strategy is well-defined and on track, and our discovery, development and commercialization engines are delivering real value for patients,” Massey added. “The acquisition of Forte Biosciences builds on the strength of that foundation and advances our ambition to be the leading immunology innovator of the future.”

The boards of Argenx and Forte have approved the transaction, which is expected to close in the third quarter.

Through a wholly owned subsidiary, Argenx plans to launch a cash tender offer to acquire all outstanding shares of Forte’s common stock at $77 per share cash. The deal price represents an approximately 86% premium to Forte Biosciences’ volume-weighted average price (VWAP) since reporting positive Phase Ib data in vitiligo on July 9.

The tender offer is subject to the tender of at least a majority of the outstanding shares of Forte Biosciences, the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and other customary closing conditions.

Upon successful completion of the tender offer, a wholly owned subsidiary of Argenx will merge with Forte Biosciences, and the outstanding Forte Biosciences shares not tendered in the tender offer will be converted into the right to receive the same $77 per share in cash paid in the tender offer.

“We are incredibly proud of what we have achieved in advancing FB102 through clinical development and firmly believe that Argenx is the ideal strategic partner to unlock the full potential of this novel anti-CD122 antibody across a broad range of autoimmune diseases,” stated Paul A. Wagner, PhD, Forte’s CEO and chairperson.

“By combining FB102’s promising clinical profile with Argenx’s proven development expertise, global reach and commercial capabilities, we have a unique opportunity to accelerate its development and maximize its impact for patients living with vitiligo, celiac disease, alopecia areata and other autoimmune conditions,” Wagner added. “We are excited about the future of FB102 and the potential to bring this innovative therapy to many more patients worldwide.”

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Comprehensive Human Vagus Nerve Map Unveiled

Edusehat — Tue, 28 Jul 2026 20:30:09 +0700

Scientists at Northwell Health’s Feinstein Institutes for Medical Research said they have released the world’s first comprehensive human vagus nerve anatomical map. The achievement could change our understanding of the autonomic nervous system and accelerate the development of bioelectronic medicine and neuromodulation therapies, according to the researchers.

The first dataset release, collected over three years from 30 human donors encompassing 60 vagus nerves, is now available to the global scientific community via SPARC Science.

The vagus nerve is the longest cranial nerve and a critical “information superhighway,” consisting of two main bundles (one on the left side of the neck and the other on the right side of the neck) containing more than 200,000 individual nerve fibers stretching from the brainstem to all major organs.

Stavros Zanos, MD, PhD [Feinstein Institutes]

The nerve manages automatic functions such as heart rate, breathing and digestion, and serves as the body’s “on/off switch” for immune response and inflammation. To better understand the function of each vagal fiber, this new dataset resource offers a 3D view into the intricate anatomy of the human vagus nerve, utilizing techniques such as microCT imaging, immunohistochemistry, and ultrasound.

By mapping the organization of fascicles and fibers, investigators expect to gain critical insights into how the vagus nerve communicates with various organs and influences human health and disease.

“This dataset represents a major step forward in bioelectronic medicine, offering the most detailed anatomical reconstruction of the human vagus nerve to date,” said Stavros Zanos, MD, PhD, associate professor in the Institute of Bioelectronic Medicine at the Feinstein Institutes and co-leader of the project. “For the first time, we can visualize the vagus nerve’s complex architecture that will allow us to design more precise, effective and safe neuromodulation therapies and devices.”

The accomplishment marks a milestone that began with a $6.7 million National Institutes of Health (NIH) grant awarded to the Feinstein Institutes in October 2022 for its Reconstructing Vagal Anatomy (REVA) project, part of the NIH Common Fund’s SPARC program. The successful delivery of the map was supported by Peter J. Pappas, Jr., whose donation provided philanthropic support towards the goals of this project.

Kevin J. Tracey, MD [Feinstein Institutes]

“Decoding the vagus nerve’s intricate language is an important advance for science and medicine,” said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes, Karches Family Distinguished Chair in Medical Research and author of the book The Great Nerve: The New Science of the Vagus Nerve and How to Harness Its Healing Reflexes. “This knowledge will further empower researchers to re-engineer human biology and unlock novel therapies for future patients.”

The Feinstein Institutes for Medical Research is a global scientific leader in bioelectronic medicine and vagus nerve stimulation, where medical researchers use modern technology to develop new device-based therapies to treat disease and injury, according to a Feinstein spokesperson, who points out that the field of bioelectronic medicine integrates insights from neuroscience, molecular medicine and biomedical engineering, and researchers at the Feinstein Institutes leverage the connection between the brain and the immune system to develop bioelectronic medicine interventions.

The vagus nerve helps regulate blood pressure, heart rate, sleep, mood, breathing, bladder function, digestion, and the immune system. [Feinstein Institutes]

The discovery that initiated the field of bioelectronic medicine—called the “inflammatory reflex”—was made more than 30 years ago by Tracey, continues the Feinstein official. This discovery emerged from studies on vagus nerve signaling and showed that the brain and body communicate to regulate inflammation and, if uncontrolled, inflammation could lead to disease, said Tracey.

It was the first FDA-approved vagus nerve stimulation device in July 2025 to treat rheumatoid arthritis. Northwell Health was the first in the nation to implant the newly approved treatment in patients in August 2025.

Today, engineers, computer scientists, immunologists, neuroscientists and clinicians develop cutting-edge medicine, including neuroimmune modulation, miniature implants for stimulating and recording the vagus nerve, noninvasive ultrasound neuromodulation to suppress inflammation, and novel brain-computer interfaces to overcome injuries of the nervous system, according to a Feinstein Institutes statement. These collaborative efforts are focused on converging to create personalized, precise treatments that hold promise in treating acute and chronic diseases, often with fewer side effects compared to current therapies.

Scientists believe these treatments have the potential to enhance or replace existing treatments across a range of conditions such as arthritis, heart disease, inflammatory bowel diseases, diabetes, cancer, and autoimmune disorders. By producing bioelectronic medicine knowledge, disease and injury could one day be treated by our own nerves without costly and potentially harmful pharmaceuticals, predict a number of researchers.

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Parkinson’s Disease Medication Monitored with Fingertip Sweat Patch

Edusehat — Tue, 28 Jul 2026 09:35:10 +0700

Engineers and neuroscientists at the University of California (UC) San Diego have developed a soft, wearable fingertip patch that continuously tracks a Parkinson’s disease (PD) patient’s levodopa medication levels by measuring chemicals in their sweat, with no batteries required. Tests in healthy volunteers and in Parkinson’s disease patients showed that measurements generated using the device were comparable to those obtained by standard laboratory blood tests.

The wearable device offers a way to continuously track real-time concentrations of levodopa in the body and could enable doctors to precisely customize daily medication schedules for patients at home.

The research was led by Tamoghna Saha, PhD, a postdoctoral researcher in the lab of Joseph Wang, DSc, professor in the Aiiso Yufeng Li Family Department of Chemical and Nano Engineering at the UC San Diego Jacobs School of Engineering. Saha is co-first author of the team’s published paper in PNAS titled “A wearable patch for continuous levodopa monitoring in sweat: Towards exertion and power-free pharmacodynamic assessment in Parkinson’s disease.” In their paper the authors wrote in summary, “Overall, our easy-to-use, energy-efficient wearable supports real-time, stimulation-free monitoring, potentially enabling at-home dosage adjustments and paving the way for future autonomous closed-loop L-dopa therapeutic system development.”

Parkinson’s disease is the second most common and fastest-growing neurodegenerative disorder worldwide, the author wrote. “While no cure for PD exists, levodopa (L-dopa) is the most effective symptomatic treatment, which is typically administered via oral tablets or capsules, and in advanced cases, through inhaled powder or continuous intrajejunal or subcutaneous infusions.” Prescribing the right dose is challenging: reducing levodopa leaves patients unable to move, while too much triggers severe, uncontrollable jerking movements. Initially, the drug’s effects can last several hours.

But as the disease progresses, the therapeutic window narrows down to two hours. Currently, clinicians must rely on subjective patient diaries to adjust treatment. Unfortunately, these methods fail to catch dangerous dosing gaps. “Precision management of Parkinson’s disease (PD) requires frequent levodopa (L-dopa) dose adjustments, yet current monitoring relies on subjective symptom reporting and infrequent blood testing,” the team continued.

Levodopa monitoring patch showing the assembly of the hydrogel and levodopa sensor with the paper fluidic channel on the fingertip. [Tamoghna Saha.]

Saha and the engineering team developed the new finger patch technology in joint collaboration with the lab of Irene Litvan, MD, MPhil, professor in the department of neurosciences at UC San Diego School of Medicine. The project is part of a longstanding collaboration between the Wang and Litvan teams to develop wearable levodopa monitors that can improve personalized care for people living with PD.

Worn on the fingertip, which is packed with a high density of sweat glands, the patch is equipped with a specially engineered absorbent gel that acts like a sweat sponge. The gel contains a highly-concentrated mixture of salts and benign solvents—and that draws sweat out of the pores, since water naturally flows toward areas with higher salt concentrations. Collected sweat is drawn into a serpentine fluidic channel with a self-powered levodopa biosensor connected to a wireless transmitter.

When levodopa in the patient’s sweat comes into contact with enzymes embedded in the patch it triggers a chemical reaction, which in turn generates a small, measurable voltage. This chemical reaction is what powers the patch. The amount of voltage generated also serves as an indicator of the patient’s levodopa level, such that lower voltage signals low levels, while higher voltage signals high levels.

Unassembled integrated levodopa monitoring patch. [David Baillot (University of California, San Diego, San Diego, CA).]

Experimental results from three to five healthy participants and four individuals with PD indicated that levodopa concentrations in sweat measured by the patch are strongly correlated with blood concentrations measured by high-performance liquid chromatography. The patches captured pharmacodynamic responses and patient-specific levodopa clearance trends that could be used to calibrate dosage estimates for individuals.

The data revealed that individuals with Parkinson’s clear levodopa from their systems significantly faster than healthy individuals. This result explains why a patient’s Parkinson’s symptoms can deteriorate so suddenly, the researchers noted.

This technology could lay the groundwork for a closed-loop system, where a levodopa monitoring patch could communicate with a pump to automatically deliver the precise doses of the drug right when the body needs it, the authors suggested. “This approach establishes a foundation for real-time, at-home therapeutic optimization and advances the development of future closed-loop treatment systems for PD.”

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ALS Could Be Predicted Years Before Symptoms, Proteomics Study Finds

Edusehat — Tue, 28 Jul 2026 06:00:09 +0700

Neurodegenerative diseases are typically defined by the presence of characteristic clinical phenotypes. However, it is increasingly recognized that early intervention could give people the best chance for meaningful positive effects. As a result, early detection and prevention are becoming major priorities.

Months to several years before amyotrophic lateral sclerosis (ALS) symptoms arise, levels of certain blood proteins may dramatically shift. By anticipating the arrival of symptoms, investigators could intervene with preventative therapies before the irreversible motor neuron damage that is characteristic of ALS sets in.

In this study, researchers analyzed data from the long-running, National Institutes of Health (NIH)-funded Pre-symptomatic Familial ALS (Pre-fALS) study, to identify a lineup of key proteins that may predict the emergence of clinically manifest ALS.

This work is published in Nature Medicine in the paper, “Longitudinal plasma proteomics predict phenoconversion to clinically manifest ALS.”

“If someone carrying an ALS-associated genetic variant had asked me in the past when they would become symptomatic, I would have struggled to provide a reasonable estimate,” said Michael Benatar, MD, PhD, professor of neurology and public health sciences at the University of Miami. “These biomarkers give us a far better idea of the timing, allowing us to estimate the time to symptom onset with an average error of about 18 months. That’s something we can work with.”

For nearly 20 years, the Pre-fALS study has collected data and biological samples from people who are at significantly elevated genetic risk for ALS but have not yet progressed, or phenoconverted, to the disease. While this cohort is unique, permitting the examination of presymptomatic ALS, recent studies suggest that findings from Pre-fALS are likely relevant to the broader population.

In 2017, an analysis of ten Pre-fALS participants who had developed symptoms showed that neurofilament light chain (NfL), a structural protein in neurons, spiked in their blood in the months preceding ALS phenoconversion. As more study participants have begun showing symptoms or signs of disease, new opportunities to search for other pre-symptomatic ALS biomarkers have emerged.

Now, using Olink Explore, investigators report a high-throughput, proteomic study on 516 serially collected plasma samples from 137 study participants; 33 phenoconverters, 35 patients with ALS, 10 pre-symptomatic pathogenic variant carriers and 59 controls.

The team identified 92 whose levels differed in people before they eventually showed symptoms. Using machine-learning techniques, the authors tested how various combinations of proteins could predict future risk of phenoconversion. Characterizing the longitudinal trajectory of these proteins, they identified a core panel of 19 proteins (including NfL) which, collectively, the authors note, predicted phenoconversion over the 0.5-year to 5-year time horizons and yielded estimates of time to phenoconversion with a mean absolute error of 1.6 years.

They also produced similar results using data from the UK Biobank, which, despite some limitations, is more representative of the general population than the genetically predisposed cohort of Pre-fALS.

“With preventative gene-targeting treatments now becoming available, there is a particularly urgent need for reliable biofluid-based signatures that indicate near-term onset in individuals that carry ALS risk genes,” said Amy Bany Adams, PhD, acting director of NIH’s National Institute of Neurological Disorders and Stroke (NINDS).

Tofersen, a drug approved for symptomatic ALS, is currently being evaluated as a preventative therapeutic in pre-symptomatic ALS through ATLAS, a clinical trial designed by Benatar in partnership with the company Biogen. ATLAS will test whether starting treatment shortly before symptoms appear could avert or delay the onset of ALS.

“This is all possible because of the members of the carrier community who believe in our mission of preventing ALS and have supported and participated in our research. It has been one of my life’s greatest privileges to give something back,” Benatar said.

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Solving the Mystery of Why Blocking and Stimulating a Brain Receptor Helps Weight Loss

Edusehat — Tue, 28 Jul 2026 06:00:08 +0700

Researchers headed by a team at the Institute of Metabolic Science, University of Cambridge, have solved the mystery of why both stimulating and blocking a particular receptor, or switch, in the brain can help people lose weight. Their study in mice indicated that the answer lies in where the receptor, called GIPR, is located. The results showed that stimulating this switch in the brainstem suppresses appetite, while the same effect can be achieved by blocking it in the hypothalamus. The researchers say their findings could help in the development of more effectiveness therapeutic strategies.

Jo Lewis, PhD, at the Institute of Metabolic Science at the University of Cambridge, said, “Understanding which brain circuits respond to these medications—and how they do so—could help us design better drugs that produce more weight loss with fewer side effects, and which might work in combination with other obesity medicines to even greater effect.” Lewis is first author of the team’s published paper in Nature Metabolism, titled “Distinct brain regions mediate regulation of food intake in response to GIPR agonism or antagonism.”

More than a billion people worldwide are living with obesity, which increases the risk of diseases such as type 2 diabetes (T2D), cardiovascular disease (CVD) and cancer. Weight loss can help mitigate these complications, but losing weight through diet and exercise alone can prove challenging.

In the past few years, a new generation of weight loss drugs has emerged that target particular receptors in the brain, reducing appetite and leading to weight loss, as well as helping to control blood sugar levels. Several of these drugs, such as Wegovy and Ozempic, work by stimulating the glucagon-like peptide 1 receptor (GLP-1R).

Other weight loss drugs act on both this receptor and on GIPR. “The development of dual agonists for the glucagon-like peptide-1 receptor (GLP-1R) and glucose-dependent insulinotropic polypeptide receptor (GIPR) has been a landmark moment in the treatment of type 2 diabetes and obesity,” the authors wrote.

However, some drugs, such as Mounjaro and Zepbound, stimulate GIPR, while others, such as the Phase III-stage MariTide, block it. Why these opposite actions have the same result has puzzled scientists. “… for reasons that are incompletely understood, in preclinical and clinical studies, adding either a GIPR agonist or GIPR antagonist to GLP-1R agonism causes additional weight loss,” the team continued. “There is emerging evidence that GIPR agonism and antagonism exert their paradoxically similar effects on weight loss via distinct neuronal populations.”

The investigators’ newly reported preclinical research has now shown that the two different types of GIPR drugs act on distinct regions of the brain, but also that they can boost weight loss when combined with certain GLP-1-based weight-loss drugs. For their reported study the team turned to genetically engineered mice and selectively removed GIPR from different parts of the brain to see which regions were responsible for the effects of the obesity drugs.

One group of mice lacked GIPR in the brainstem—the area at the base of the brain, just above the spinal cord, involved in appetite and nausea. A second group lacked GIPR in the hypothalamus, a major center controlling hunger and body weight. A third, control group included normal, unmodified mice. The researchers treated the mice with various combinations of a GIPR agonist (which activates the receptor), a GIPR antagonist (which blocks the receptor) and a GLP-1 drug, and measured food intake, body weight, fat mass, glucose control and brain activity.

“We knock out Gipr in either the area postrema (AP) or hypothalamus of mice (Gipr^AP-KOand Gipr^hypo-KO, respectively) and compare body weight and food intake responses to GIPR agonists and antagonists, alone and in combination with the GLP-1R agonist liraglutide,” they wrote in summary.

By comparing the responses of normal mice with mice lacking GIPR in different brain areas the investigators showed that GIPR agonists act on the brainstem to suppress appetite and reduce weight. They then showed that GIPR antagonists help weight loss by acting on this receptor in the hypothalamus, where they release a “brake” that otherwise limits the brainstem’s ability to respond to signals telling us we are full. Blocking GIPR also appeared to boost the effect of emerging new drugs targeting the amylin receptor—such as cagrilintide (Cagri)—suggesting that GIPR antagonists could potentially be used to strengthen several types of anti-obesity medicines.

“Overall, our results suggest that the AP is responsible for the appetite-suppressing effects of GIPR agonism but that GIP receptors in the hypothalamus underlie the ability of GIPR antagonism to enhance the weight loss effects of GLP-1R and amylin receptor agonists,” they stated. “GIPR antagonism and Gipr^hypo-KOalso sensitize to cagrilintide-induced weight loss.

The findings explain why drugs such as MariTide, which combines GIPR antagonism with GLP-1 receptor agonism, are effective, and suggests how to design even better combination therapies. And as the authors noted, “Future work is still, however, required to identify the neuronal networks underlying GIPR interactions in the AP and hypothalamus and their crosstalk with other appetite-regulating circuitry.”

Lewis said the work strengthens the idea that the brain is central to obesity treatment, commenting, “Obesity drugs are not acting simply on the gut or pancreas. Instead, they have important effects on specific, identifiable brain circuits that regulate appetite and food intake.”

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StockWatch: Axiom CEO Explains Plans for Hong Kong IPO

Edusehat — Mon, 27 Jul 2026 15:30:10 +0700

Axiom Biosciences, a privately held developer of regenerative therapies and targeted biologics that rebranded last month from Cytonus Therapeutics, made headlines around the world when it announced plans to go public through an initial public offering (IPO), since companies that go public typically do so quietly, by filing registration statements with regulators and exchanges.

What made Axiom’s IPO plans even more newsworthy: the San Diego-area company (based in suburban Carlsbad, CA) said it intends to trade its first public shares on the Hong Kong Exchange (HKEX) rather than a U.S. market like Nasdaq, the leading market for biotech IPOs, or the New York Stock Exchange.

Why Hong Kong? Axiom says the special administrative region of China possesses deep biotechnology expertise, a strong appetite for clinical-stage innovation, and direct proximity to the Asian partners and capital advancing the company’s science.

“For us, Hong Kong is a very good fit for the stage of company that we’re in, and the timing is right,” Remo Moomiaie-Qajar, MD, Axiom’s founder, CEO, and chairman, told GEN. “Hong Kong has a very well-established investor base that really understands the time requirements and the capital needs for biopharma, but in particular, cell therapies. And ultimately, given the fact that we have been working in Asia with partnerships for several years, this landing spot for us in Hong Kong seemed to be the right choice in a broader strategy.”

Does that mean the United States is finished for biotech innovation?

“I would not state that at all,” he replied. “I think the United States is very much still a leader. It is also very central to our strategy, and the FDA is really core to all of our decisions moving forward into clinics. I just see that this is, and I speak only for us, part of a bigger global strategy which includes both Asia and the United States.”

“Ecosystem to thrive”

Remo Moomiaie-Qajar, MD, Axiom Biosciences founder, CEO, and chairman

However, Axiom has publicly offered other reasons for its move that convey a warmer biopharma climate in Hong Kong compared with the United States. In its announcement, the company said its Hong Kong IPO plans were “reflecting a broader shift in where the world’s most ambitious science finds the ecosystem to thrive.”

And speaking on CNBC, Moomiaie-Qajar raised a concern with the U.S. biopharma climate when it comes to financing: “Some of the most important science in the world is being built in the United States, but the way it gets funded hasn’t kept pace.”

He elaborated on that remark, telling GEN: “That speaks to a broad range of problems that we have in financing within this industry. In particular, I was referencing the private side.”

“The reality is, as you progress with your pipelines and you cross over the threshold of being a clinical stage company, and then you have clinical success, it requires a significant amount—more capital to get your assets to move forward, and hopefully, to a BLA [Biologics License Application],” Moomiaie-Qajar explained. “But the number of check writers diminishes at the same time. So, there is seemingly no shortage of capital within biopharma, but I do believe there’s a financing issue and an access issue.”

Hence Axiom’s exploration of whether this was the right time to go public—a question Axiom is answering in the affirmative: “Hong Kong, given our strategic fit, and relationships and proximity to our partners in Asia, was the right first decision in establishing our public identity as a company.”

Comeback mode

The IPO market has been in comeback mode most of this year, with 14 companies selling their first public shares on U.S. markets since January, and another five doing so overseas, in Asian markets that include the Tokyo Stock Exchange, South Korea’s tech-focused KOSDAQ, and the Hong Kong Exchange.

The biggest American biotech IPO—this year, and of all time—was an upsized offering that took place last month, when Parabilis Medicines (Nasdaq: PBLS) raised an eye-popping $770.5 million in gross proceeds by selling some 38.5 million shares at $20 per share. Parabilis’ shares have risen 56% since then, to $31.28 at Friday’s closing bell.

The latest biotech IPO, also upsized, came on Thursday when Scribe Therapeutics (Nasdaq: SCTX), a developer of in vivo CRISPR gene-edited therapies, raised $128.7 million gross by selling 8.58 million shares at the high end of its price range at $15 per share. The shares jumped 44% on Friday, finishing the day at $21.65. Scribe also raised another $7.5 million gross by selling 500,000 shares at the IPO price to Sanofi (Euronext Paris: SAN) in a concurrent private placement.

But the best-performing U.S. biotech IPO is Veradermics (NYSE: MANE), a developer of treatments for dermatology and aesthetic conditions whose shares have catapulted more than six-fold, rocketing 545% since pricing its IPO at $17 per share on February 3, closing Friday at $109.66 per share. Earlier this month, Veradermics announced positive topline results from its open-label Phase II Study 207 trial (NCT06527365) assessing VDPHL01, an extended-release oral minoxidil formulation, in women with mild-to-moderate pattern hair loss.

Hong Kong’s largest biotech IPO so far this year is Suzhou Ribo Life Science (6938.HK), a developer of oligonucleotide treatments based on RNA interference and other technologies. Ribo raised more than HKD 1.8 billion ($229.5 million) by selling 31,610,400 shares at HKD 57.97 ($7.39) on January 9. Since then, however, Ribo’s stock price has dipped 7.5%, closing Thursday at HKD 53.60 ($6.83).

Also going public via HKEX this year were medtech companies such as Hangzhou Diagens Biotechnology (2526.HK), a developer of artificial intelligence (AI)-based medical imaging tools whose customers include specialized genomics research labs and cytogenetics labs, as well as hospital pathology departments. Diagens went public March 30, raising about $101 million by selling 7,999,200 shares at HKD 99.00 ($12.62)—a price that has since more than doubled, leaping 172% after closing Friday at HKD 269.00 ($34.30).

HKEX lists 84 biotech companies, compared with more than 600 for Nasdaq, according to their respective websites.

Staying in America

Over time, Axiom plans to pursue a secondary stock listing in the United States. Moomiaie-Qajar says Axiom will remain an American company: “We are a U.S.-based, U.S.-headquartered company that is going to be something that does not change.”

Axiom says it intends to be the first U.S. biotech company planning to go public in Hong Kong. That’s the path that was successfully trod by AI-based drug developer Insilico Medicine (3696.HK) when it went public in December, raising HKD 2.277 billion (about $292.3 million at the time; now worth $290.3 million) on the Hong Kong Exchange by selling 94,690,500 shares at HKD 24.05 ($3.08, now worth $3.06) each.

Insilico’s stock has nearly doubled, soaring 96% since then, closing Friday at HKD 47.06 ($6.00) thanks to several collaborations with biopharma giants and an upbeat revenue and profit forecast for the first half of 2026.

“From my perspective, it certainly seeded a little bit of certainty in my mind that this was a good decision, because they’ve done really well post-IPO,” Moomiaie-Qajar commented.

Insilico’s parent InSilico Medicine Cayman TopCo lists a registered office in the Cayman Islands, while Insilico’s website lists additional offices in Cambridge, MA (announced in 2024 as the company’s headquarters), New York, Montreal, Abu Dhabi, Hong Kong, Shanghai, and Taipei.

“Very high listing standards”

“One of the reasons you would list in Hong Kong is to openly compete with the companies in the ‘China Gym’ and also take advantage of the increased visibility and transparency with the very high listing standards,” Alex Zhavoronkov, PhD, Insilico’s chairman, executive director, CEO, and CBO, told GEN.

“Many companies want to list there. But the barriers for listing are very high even for the biotech track.”

A company planning to go public, he explained, needs not only an asset in Phase II studies, but a clear funding history from credible investors, several years’ worth of cash to operate, and a level of corporate stability that the exchange will assess.

“For very early biotech companies it may be much easier to list in the United States,” Zhavoronkov said. “In general, it is a positive trend because biotech must become more international and collaborate and compete internationally. Competing for capital is the advanced form of competition because finance usually runs biotech—you cannot discover and develop drugs without it. Companies and ideas in biotech should become more fluid internationally. Public listings make companies and ideas more competitive and transparent.”

Together with Seoul-based, privately held Medinno, Axiom has co-developed its lead regenerative therapy based on umbilical cord-derived, conditioned mesenchymal stem cells (MSCs) sourced from Wharton’s Jelly. The therapy is under study in two pipeline programs that aim to treat newborns with severe brain injury: AX-007 for intraventricular hemorrhage (IVH); and AX-008 for hypoxic-ischemic encephalopathy (HIE).

Positive Phase I results

Earlier this month, Axiom announced positive results from a Phase I dose-escalation study assessing the safety, tolerability, and preliminary efficacy of the regenerative therapy across a range of doses in nine newborns—five diagnosed with severe IVH, four with HIE—following direct administration into the central nervous system.

Across all doses studied, the MSC therapy achieved a 0% mortality rate at 12 months compared to the historical natural 46% mortality rate within the first year of life for infants with severe IVH. The therapy also showed a favorable safety profile, with no treatment-related serious adverse events seen.

“We’re now in discussions with the FDA to move those programs into the next stages, which would be a Phase IIb study,” Moomiaie-Qajar said. The FDA has granted AX-007 and AX-008 its Rare Pediatric Disease and Orphan Disease designations.

Axiom is also evaluating an expansion of its therapy development into adult ischemic stroke, which affects approximately 700,000 adults annually in the United States, and additional neurological indications.

“We feel very confident that given our clinical trial success in Phase I, the expansion of our valuable asset into three, four indications is going to be a good basis for us to launch our IPO, but then after the IPO really go further and deeper into our pipeline that we’ve been developing for eight years,” Moomiaie-Qajar said. “We have a lot now that we need to translate into clinics.”

Leaders and laggards

Immix Biopharma (Nasdaq: IMMX) shares tumbled 14% from $10.25 to $8.80 on July 20 following the arrest of Ronald L. Fischer, 70, who was one of Rhode Island’s Most Wanted fugitives—and who, under the alias of Richard Graydon, MD, PhD, served as the company’s CMO. Fischer was arrested by federal and Rhode Island authorities off the coast of New Jersey on a U.S. Marshals Service Unlawful Flight to Avoid Prosecution warrant, having been a fugitive since fleeing Rhode Island during a criminal trial in 2005, the U.S. Justice Department stated. Fischer was convicted in absentia of First-Degree Sexual Assault after failing to appear for trial and remained wanted for Failure to Appear, First Degree Sexual Assault, and Flight to Avoid Prosecution. Authorities also said Fischer was living on a 56-foot sailing vessel called The Silver Lining, which was registered under the Graydon name. As “Graydon,” Fischer was appointed Immix’s CMO in March, the company announced March 30 in a press release no longer posted on its website. “As of July 17, 2026, Richard Graydon has been terminated and is no longer with the company for reasons unrelated to his activities at the company,” Immix disclosed in a July 20 regulatory filing, adding: “Given his short tenure, management believes there is no material effect on the business.”
Novocure (NVCR) shares jumped 28% from $15.57 to $19.99 Thursday after the Swiss-based oncology drug/device developer developer of the Tumor Treating Fields (TTFields) cancer therapy reported second quarter adjusted earnings before interest, taxes, depreciation, and amortization (EBITDA) of $10.757 million, vs. an adjusted loss of $9.934 million a year earlier, on net revenue that rose nearly 16% year-over-year, to $183.584 million from $158.805 million. Novocure still finished Q2 in the red with a net loss of $15.658 million, improved from the $40.139 million net loss of the second quarter of 2025. Novocure credited its 18% global active patient growth across indications for the positive numbers; as of June 30, more than 280 active patients were on Optune Pax®, a wearable device designed to deliver its TTFields therapy for adults with locally advanced pancreatic cancer concomitant with gemcitabine and nab-paclitaxel. “The main takeaway is that the early U.S. adoption of Optune Pax is off to an encouraging start,” J.P. Morgan analyst Jessica Fye wrote in a research note. Novocure shares reached a 52-week high of $21.35 at the start of the trading day before sliding 12% to $17.65 on apparent profit-taking.

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BIO 2026: To restore trust in vaccines, listen to everyone’s concerns

Edusehat — Sun, 26 Jul 2026 21:35:03 +0700

Listening to people’s concerns about vaccines is key to helping them regain trust, according to experts promoting the need for life-saving immunizations.

“We’ve all gone through a global trauma together,” said Phyllis Arthur, EVP & Chief of Global Health at the Biotechnology Innovation Organization (BIO), noting that mistrust of vaccination grew rapidly during the COVID pandemic. “We need to find commonality with those who are concerned about vaccines, and accept their questions.”

Trust in vaccines has yet to recover to pre-pandemic levels, creating a new public health threat from the potential resurgence of vaccine-preventable diseases. Arthur was among many experts discussing ways to build this trust during several panels at the BIO International Convention in San Diego (BIO 2026).

Challenges to rebuilding trust

“Communications can’t be a one-way street—the goal isn’t to repeat our message louder, but to understand people’s questions and doubts so we can meet them with information that’s relevant, understandable and useful,” according to Silvia Taylor, MBA, Chief Corporate Affairs Officer and Head of Novavax Sweden. Taylor wrote an article describing the panel she joined at BIO 2026 on rebuilding confidence in science and vaccines. (Read Taylor’s full article here.)

“We can do a better job as an industry of acknowledging where the science is not precise,” Taylor said during the panel.

The medical community can no longer simply assert its authority in hopes of returning to the pre-pandemic consensus, said fellow panelist Rebecca Alvania, CEO of the National Foundation for Infectious Diseases. Instead, they should “acknowledge the shifting sands around that trust relationship between the patient and provider,” she said. “We can’t be fighting against forces of change. We have to be navigating them.”

One action that might create misunderstanding around vaccines was the announcement earlier this year that the Department of Health and Human Services was reducing the number of standard recommendations for childhood vaccines, explained Arthur.

“It was not done in a way that was necessarily transparent or led to discussion with the public, industry, and stakeholders, and so we suddenly had a brand new vaccination schedule, and it caused a good bit of confusion,” she said.

The state perspective

While changes at the federal level have a big impact, much of vaccine policy is made at the state level.

In a panel on state vaccination policy, Kate Mevis, Executive Director of Federal and State Vaccine Policy at Merck, recalled meeting vaccine skeptics in 2008 when she worked as an aide in the Senate. Since then, anti-vaccine skepticism has grown.

“Anti-vaccine advocacy has become more organized, and there is that ability for them to have a voice at the state level, talking to legislators,” said Erin Abramsohn, Executive Director of the Infectious Disease Prevention Network.

Abramsohn said her group helped defeat more than 50 “anti-vaccine” bills in 10 states in the 2026 legislative season alone. Overall, she said, polls on the state and national level show most Americans from both parties favor routine vaccination and requirements for school entry, yet in some states anti-vaccination voices are louder.

Those states can pay a price, as we saw with the measles outbreak in Texas.

“We really are living in two Americas where your health care and your health care outcomes are dictated by your zip code,” said Julia Spiegel, CEO of GovAct.

“South Carolina, Utah, Texas, Idaho—we are seeing more outbreaks of vaccine-preventable diseases, and those are really part and parcel of seeing lower vaccination,” said Arthur. “Dips in certain communities have led to outbreaks, and those may have been prevented.”

States have taken a new approach in a time of confusion in vaccine policy. California, Hawaii, Oregon, and Washington formed the West Coast Health Alliance to encourage uptake of vaccines and “to create our own public health consensus that we’re going to follow the science,” regardless of attitudes in Washington, D.C., explained Andy Chason, Vice President of Federal Policy and Advocacy for Blue Shield California.

Messengers matter

A key to addressing anti-vaccine sentiment is to take a holistic approach that allows people to ask questions and also respects the fears motivating their concerns, experts at BIO 2026 agreed.

This approach was favored by speakers on another panel, “Innovating Against Influenza: New Breakthroughs to Save Lives,” who lamented that flu vaccine uptake among children has dropped below 50%. They said more effective countermeasures include allowing entire communities to be vaccinated at once through initiatives such as community vaccination drives.

Alvania emphasized the importance of a group effort.

“Prevention was always a team sport,” she said. “There are many players on the field that have strengths, and we have to be collaborating.”

Several experts spoke of the need to find trusted local voices, including church leaders and community activists, rather than politicians.

One voice that many people listen to is AI, already the primary source for medical advice for millions, experts said. Panelists argued the medical community should understand this and turn AI into an ally against misinformation.

“I think we’re still in this adolescence of understanding that the LLM is now a very important stakeholder,” said Deborah Glasser, Head, Specialty Care, North America, and U.S. Country Lead at Sanofi and a member of the BIO Trust in Science Working Group. But she warned that large-language models (LLMs) need high-quality evidence, noting that, at one point, 70% of the data LLMs gathered came from Reddit, which was erroneously treated as a scientific source.

As with human experts, AI needs to listen carefully—to the right sources. Fortunately, data from AI searches is constantly improving, according to Glasser. “I’m super optimistic about the day that my 82-year-old mother can have an LLM that tells her what’s going on with her biology,” she added.

Meanwhile, experts said, the vaccine community’s willingness to listen to those who may be more hesitant is also helping rebuild trust.

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The quest to keep organs alive outside the body

Edusehat — Sat, 25 Jul 2026 05:35:02 +0700

This week, I covered a fascinating effort to preserve organs outside the body. There’s a huge shortage of donor organs, and one of the main reasons is time—they survive only a matter of hours outside the body, even when they’re kept on ice.

Doctors dream of organ banks—stores of human organs that can be preserved for days, weeks, months, or even longer. That would allow them to run tests on organs, find the best matches for them, and transport the organs to those recipients.

In new research, one team has been able to supercool the kidneys of pigs—animals whose organs are of a similar size to human ones—and preserve them for days. The kidneys survived being stored at −4 °C (25 °F) and eventually reimplanted back into pigs. And that’s just the latest development in a field that is positively buzzing.

It has proved super difficult to freeze organs. Once ice forms in them, they’re done. The ice crystals create all kinds of damage and render the organs unusable. That hasn’t stopped many researchers from trying.

Some have focused on cryopreservation—rapid extreme cooling that essentially leaves cells in a glasslike state. This process is now routine for eggs, sperm, and embryos, which are cooled to −196 °C in less than two seconds and can be used even after decades in storage.

No one has managed to cryopreserve and thaw human organs for transplantation. But plenty of human bodies and brains have been stored at ultra-low temperatures in the hope that they might one day be rewarmed and brought back to life. (You can read more about why some people opt for cryonics here.)

In March, I wrote about Stephen L. Coles, a gerontologist who had opted to cryopreserve his own brain. After the scientist died in 2014, his body was taken to Alcor, a cryonics facility in Arizona. A team at the facility removed Coles’s head, perfused his brain with cryoprotective chemicals (which work like antifreeze), removed the brain from the skull, and cooled it to −146 °C.

When Coles’s friend Greg Fahy, a cryobiologist, studied pieces of his brain years later, he found that the brain cells, which had shrunk, “bounced back” once they were rewarmed. But that doesn’t mean the cells are alive, or that it might one day be possible to reanimate the brain. As Matthew Powell Palm of Texas A&M told me at the time: “There are so many ways those neurons could be toast.”

Powell Palm is working on other ways to preserve organs. It was he, along with his colleagues, who managed to store supercooled pig kidneys and successfully transplant them, in a study described as “a landmark achievement.” Those organs did better than kidneys stored on ice, he says.

His approach didn’t require cryoprotectants. But other teams are exploring potential chemical cocktails that might allow them to store organs at lower temperatures, potentially for longer periods of time. (More on this in The Checkup soon!)

Another way to prolong the lifespan of an organ is to use a machine that perfuses it with nutrients, mimicking what happens inside the body. Machine perfusion devices have become more commonly used over the last decade or so and are typically used to maintain livers and kidneys for up to about 24 hours.

Researchers are now adapting this protocol for a growing list of organs, even eyeballs—a recent feat that might enable whole-eye transplants. In March, I went to visit scientists in Valencia who had developed a perfusion system for uteruses. They had used their device—which they nicknamed “Mother”—to keep a human uterus alive for a day.

It’s an exciting time for organ preservation. Keep an eye out for more coverage from MIT Technology Review in the coming weeks.

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.

Colorectal Cancer Targeted with Mass-Produced iPSC-Derived Allogeneic T Cells

Edusehat — Sat, 25 Jul 2026 05:30:09 +0700

Researchers at Kobe University have generated off-the-shelf, mass-producible induced pluripotent stem cell (iPSC)-derived gamma delta T cells (γδT cells) that in a small preclinical study suppressed tumor growth in mouse colorectal cancer (CRC) xenograft models. The team says their development could point to the potential for developing faster, cheaper cancer immunotherapy.

Research lead Takashi Aoi, PhD, and colleagues reported on the work in Stem Cell Reports, in a paper titled “Allogeneic iPSC-derived γδT cells demonstrate antitumor efficacy against patient-derived tissues,” commenting “Our current findings provide robust preclinical evidence supporting the efficacy of T cell therapy for CRC.”

“Various immunotherapies have been developed to treat malignant tumors, and autologous CAR T-cell therapy is clinically used for certain malignancies,” the authors wrote. However, CAR T-cell therapies demonstrate limited efficacy against solid tumors, and current techniques for modifying T cells extracted from the patient are expensive and time consuming. “… obstacles such as the time and cost required to initiate autologous treatment impede their widespread adoption.”

In the journal Stem Cell Reports, Kobe University stem cell researcher Aoi Takashi and his team report that they created iPS cells from a subclass of T cells that can be used across patients and could reproducibly turn them back into T cells with an overall 80,000-fold multiplication and without using animal cells or extracts, and that the resulting T cells attack and shrink human patient-derived colorectal cancer tumors that were implanted into mice. [Aoi Takashi]

Consequently, the authors noted, there is growing interest in allogeneic, or off-the-shelf, cell therapy. Researchers have considered turning to a subclass of T cells called gamma-delta (γδ) T cells that don’t need to be tailored toward each individual patient but can be harvested from a donor and used in other people. “… the development of novel therapies for CRC, a highly heterogeneous cancer, remains a paramount challenge in global healthcare, and γδT cells are considered a promising candidate modality,” the authors stated. “γδT cells represent approximately 3–5% of peripheral blood lymphocytes and are capable of targeting various types of tumors in an MHC-unrestricted manner with a single type of γδT cell receptor.”

However, these cells are much fewer, making the harvesting approach infeasible, and they also cannot be directly multiplied well in the lab. Aoi stated, “Based on our experience with induced pluripotent stem cells, also called iPS cells, we thought that we could approach this issue by creating such easily storable and growable cells from these specific T cells, and then only turning them back into T cells when actually needed.” In their paper the authors added “We focused on γδT cells as a potential therapeutic modality for colorectal cancer (CRC).”

Through their reported study the investigators showed that they could create iPS cells from the subclass of T cells that can be used across patients and reproducibly turn them back into those T cells with an overall 80,000-fold multiplication. Importantly, they achieved this without relying on animal cells or extracts, which is a requirement for clinical applications. “To the best of our knowledge, this is the first study to report the successful induction of differentiation of γδT cells from iPS cells under feeder-free, serum-free conditions.”

Their study was also the first to show, on a small preclinical scale, that the resulting iPSC-derived γδT cells (iγδT cells) attack and shrink human patient-derived colorectal cancer tumors that were implanted into mice, with tumor weights in treated animals reduced by up to 88%, when compared with control mice.

“We demonstrated that these iγδTs exhibit cytotoxic activity against CRC and leukemia cell lines, as well as against patient-derived CRC organoids in vitro, while also exerting antitumor effects in vivo in xenograft models,” they noted. “Cancers from cell culture lines don’t have the same drug insensitivities as actual cancers and also don’t emulate the physical barriers that actual tumors have,” explained first author Ryoko Futai, PhD. “That’s why patient-derived organoids are highly significant for evaluating new cancer treatment approaches,” explained first author.

When they designed the study, the Kobe University team imagined that their approach would be used fighting metastasizing cancers. They also checked whether their T cells would find their targets not only when administered close to the tumor but when administered intravenously a week after the tumor was implanted. And indeed, even in this setting tumor weights decreased 43%, 82% and 92% in the three treated mice. Futai noted, “This suggests potential for future systemic therapy. We believe this achievement represents an important step toward the development of a new immunotherapy for solid tumors.”

The study was conducted at a small scale, with only three or four mice in each experiment and tumor models derived from only two different patients. This is especially important because colorectal cancer tumors are known for their high variability. “This study is a preclinical investigation demonstrating the potential using iPS cell-derived T cells and is not yet at a stage where it can be used on patients,” cautions Futai.

But by conducting further studies using these easily multipliable and very standardized cells, the Kobe University development may also be used to elucidate where the variability comes from and what steps to take to counter it. Aoi commented, “Furthermore, by combining this approach with cell modification techniques such as CAR therapy, we hope that this research will eventually lead to the development of new therapeutic possibilities for patients with solid tumors.” And in their paper the authors concluded, “Our findings will pave the way for the realization of off-the-shelf allogeneic γδT cell therapy.”

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Oral Antiviral Blocks Measles-Like Virus Transmission in Ferrets

Edusehat — Sat, 25 Jul 2026 01:50:10 +0700

This year, the United States has recorded the highest number of measles cases since 2000, the year that the disease was declared eliminated from the country. As of mid-July, 2,260 measles cases were reported—just 29 cases less than the entire year of 2025. And outbreaks are widespread, with 34 new outbreaks reported in 2026.

The reason for this surge in cases is waning vaccination rates across the country. Given that, the production of new antivirals is an urgent matter.

Map of measles cases in the U.S. [CDC]

Now, the new oral antiviral drug candidate GHP-88310 has shown promising results in a ferret model of infection. When administered before or after direct contact or airborne exposure to canine distemper virus (which causes measles-like disease in ferrets) GHP-88310 blocked transmission of the virus and reduced clinical symptoms in ferrets.

“We were very excited to see that GHP-88310 given by mouth completely prevented airborne transmission in our ferret model of measles,” said Carolin Lieber, PhD, a postdoctoral fellow in the Plemper lab at Georgia State University. “This finding is unprecedented for a viral polymerase inhibitor and demonstrates the extraordinary antiviral potency of this drug.”

This research is published in Nature Microbiology in the paper, “Antiviral GHP-88310 blocks contact-mediated and airborne transmission in a ferret model of measles-like disease.”

“Silencing measles outbreaks quickly is essential to reestablish control over the virus,” said Richard Plemper, PhD, professor and director of the Center for Translational Antiviral Research (CTAR) at Georgia State University. “This study follows our recent development of the drug candidate GHP-88310. It demonstrates that the drug is suitable to augment traditional ring vaccination against measles.”

GHP-88310 (described earlier this year in Science Advances) is known to be an orally efficacious broad-spectrum orthoparamyxovirus polymerase inhibitor. But its effect on viral transmission has remained unclear. This study explored whether prophylactic administration of GHP-88310 prevents virus transmission through close contact or through the air. The results demonstrate that GHP-88310 efficiently blocks both forms of viral spread. In addition, the study showed that treatment of infected animals shortened the time period in which infected animals could transmit the virus.

To explore relevant conditions of viral transmission, the researchers established both direct-contact and airborne canine distemper virus transmission models to examine pharmacological suppression of virus spread. The transmission systems allowed them to pair infected and uninfected animals in direct physical contact or shared airspace, each under controlled environmental parameters.

“We designed the study to recapitulate viral spread between people with direct contact, for instance in a household, and between more distant social contacts, for example in classrooms or other indoor settings that bring people into proximity without direct interaction,” said Plemper.

The results showed that pre- and post-exposure prophylactic GHP-88310, given twice daily to air contacts, prevented transmission. The authors note that once-daily prophylactic administration mediated complete survival with all air contacts undergoing seroconversion. In addition, they note that therapeutic treatment of air contacts mitigated clinical signs, and animals survived, whereas all vehicle-treated air contacts succumbed. In addition, therapeutic treatment of infected source animals shortened the contagious phase by five days.

“In addition to this prophylactic benefit, GHP-88310 used therapeutically shortened the duration of disease in our model,” noted Plemper. “If equally applicable to human hosts, it may shorten the severe social and economic burden of prolonged quarantine of patients and further aid outbreak management.” The investigators are now readying GHP-88310 for formal clinical testing.

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BIO 2026: Realizing the promise of CAR-T cell therapies

Edusehat — Fri, 24 Jul 2026 18:45:03 +0700

“CAR-T cell therapies are remarkable drugs and are the gold standard for patients,” said Rachel Haurwitz, PhD, President & CEO of Caribou Biosciences.

These personalized immunotherapies have proven powerful enough to reprogram a patient’s immune system T cells to target and destroy things like cancer cells, and are useful for a variety of other autoimmune diseases, too. And we are understanding more about them every day.

And yet, only a fraction of eligible patients are getting access to CAR-Ts. For example, only 20-25% of eligible lymphoma patients have access to CAR-Ts, explained Haurwitz—shocking, given their life-saving potential.

For an industry Driven by Purpose, success doesn’t stop with scientific breakthroughs, it also has to ensure every eligible patient has the opportunity to benefit from them. So what is being done today to make sure that CAR-T can go mainstream and help as many patients as possible?

The challenges of CAR-T delivery

Experts on the final day of the 2026 BIO International Convention discussed barriers to access and adoption, and the need for empowered education.

“There is no time like the present to expand into the community setting,” said Ryan Metheny, Head of U.S. Market Access at Kite Pharma.

“The journey from community to the academic circle is not amazing,” said Lynelle Hoch, President of Cell Therapy Organization at Bristol Myers Squibb.

“When we think about solving this, yes, you have to solve the general education problem and get it referred in, but the other problem to solve is that we’ve got to be able to bring this into the community,” she continued. “You can certainly educate more. You can certainly create more of a white-glove journey into the academic centers. But at the end of the day, if modality doesn’t make it into a community, you’re going to have a very small percentage of patients that are eligible to receive them.”

These two issues are compounded by the physical capacity of healthcare settings.

“We’re seeing limitations in capacity,” Metheny continued. “We’re hearing frequently from some of the larger centers that they’re starting to run low on capacity, and as they project out and look at the growth that CAR-T is expected to see in the coming five to seven years, they’re coming to us and saying, ‘We need greater capacity. We need the community to get involved.’ ”

“Patients want to be treated closer to home,” said Hoch. “They want to be surrounded by their family. They have an attachment to their community physicians, and they want to be treated closer to home.”

Another issue is coverage, Metheny noted. With new therapies come new questions, and sometimes even resistance to coverage.

“Predictability and sustainability in economics is a huge issue,” said Metheny.

As Metheny noted, the traditional reimbursement model used for most products for a U.S. oncology physician is fairly straightforward, both in Medicare and commercially: delivery, approval, and reimbursement.

“Right now, in CAR-T, that’s not there,” Metheny continued. “As we think about the single case agreements and the negotiations that these sites have to go through, they’re not equipped like a traditional hospital with transplant experience to be able to work through those case negotiations in the way that a Stanford or an Emory or a large hospital would. So, I think getting to a point where we can have some economic reimbursement models that are more predictable, that are more similar to what they’ve experienced in that space would be a great start.”

How do we bring CAR-T to the masses?

“We’re all here because amazing scientists figured out that if you put this power construct into a T cell, you have the chance to provide curative intent therapy to a huge fraction of patients,” said Haurwitz. “That’s extraordinary.”

And, as was alluded to at the beginning of the conversation, education is an important first step. Patients need to be educated, clinicians need to be educated, and academia needs to be educated to effectively expand the use of CAR-T.

But often, the panelists noted, patients are not educated in the right way.

“We spend too much time educating patients about the complexity of CAR-T and not about the complexity of their life without CAR-T,” said Hoch. “I haven’t met a CAR-T patient yet who hasn’t said, ‘I would do it all over again‘—not even one.”

Reimbursement models also need to be addressed.

“I think there are too many panels like this, quite frankly, where we talk about things, and then not enough action happens,” asserted Metheny. “And I think one of the big reasons is because the payers are not frequently enough at the table with us.”

How CAR-T can take off

There have been a number of steps that the U.S. Food and Drug Administration (FDA) has taken to maintain the momentum of CAR-Ts going mainstream.

“The FDA has taken a lot of heat lately, appropriately so, but I will say I have to pay them a huge compliment,” said Hoch. “There is significant momentum they created when they removed the REMS (Risk Evaluation and Mitigation Strategies) from auto CAR-Ts and reduced the patient burdens … Patients no longer have to be followed for four weeks at the academic centers, don’t have to not drive for eight weeks. Now, 96% of the talks about, and management of, CAR-Ts happen in the first two weeks, and patients typically are able to go back to their normal lives. After all, these patients do feel pretty quickly back to themselves and want to go back to their normal lives.”

But there are still steps that need to be taken.

Hoch noted that it was worth the FDA revisiting the current 15-year follow-up requirement for patients. “We have now reams of data for almost two decades worth of CAR-T,” said Hoch, “that the risk of insertional immunogenesis is no different than the actual impact of the disease. And so, from our standpoint, we can’t keep these hurdles on a modality that are not data-driven anymore.”

Regardless of the challenges, the momentum of CAR-Ts is building. Now, the healthcare and regulatory community need to work together to pave the way for CAR-T to take off.

Every eligible patient should be able to have the conversation about whether CAR-T is right for them, said Metheny. “I’m hopeful that in five years, we’re able to have that conversation with many more patients.”

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Single-Cell Atlas Simultaneously Maps 3D Genome Architecture and DNA Methylation

Edusehat — Fri, 24 Jul 2026 11:30:10 +0700

Scientists at the Salk Institute and the Arc Institute, along with their collaborators, unveiled the first body-wide single-cell atlas of two major epigenetic systems: three-dimensional genome folding and DNA methylation, measured simultaneously in the same cells.

The atlas spans 86,689 cells from 16 human tissues, revealing 35 major cell types and 206 subtypes, and is freely available online. The work is part of the National Institutes of Health’s 4D Nucleome (NIH 4DN) program, which aims to understand how the genome is organized in space and time to regulate gene expression in health and disease.

Because the two epigenetic layers were measured together, the researchers could compare what each layer says about a cell’s identity. And while often the two pictures agree, they found that sometimes they do not.

The Salk paper “Human body single-cell atlas of 3D genome organization and DNA methylation” was published alongside five other NIH 4DN papers in Science, and three others in Science Advances.

The Human Genome Project, completed in 2003, produced a linear read of the three billion DNA letters in the human body. But the letters alone don’t explain how a single genome produces hundreds of different cell types. That information lives in the epigenome in the form of chemical modifications and structural folds layered on top of the DNA sequence, where they can switch genes “on” and “off” in patterns specific to each cell type.

Caption: Salk scientists Jingtian Zhou (left), Jesse Dixon (center), and Joseph Ecker (right) profiled 86,689 cells across 16 human tissues, linking cell-type-specific epigenetic features to disease risk and revealing that a cell’s 3D genome and DNA methylation don’t always tell the same story. [Salk Institute]

Two of the most consequential epigenetic features are 1) DNA methylation, where small chemical groups called methyl groups are attached to specific DNA bases, and 2) 3D genome organization, where intricate loops, folds, and compartments bring distant stretches of DNA into contact. Both influence gene expression, but they had never been measured together in single cells across the human body.

“There has been an appreciation for trying to understand, at the individual cell level, how the genome is organized, so that we can get a better idea of how genetic variants impact disease,” said co-corresponding author Joseph Ecker, PhD, a professor and Salk International Council Chair in Genetics at Salk and a Howard Hughes Medical Institute investigator. “Some cell types may be more vulnerable than others to genetic variants, because the genome is organized differently in different cell types—and whether a variant matters can depend on that organization.”

Why is noncoding DNA relevant in disease?

Most disease-associated genetic variants fall in the noncoding regions of the genome. That has made it difficult to figure out how a variant contributes to disease, which cell type it acts in, and what gene it ultimately affects.

The new atlas identifies more than 1.36 million differentially methylated regions and 283,606 differential chromatin loops across the human body’s cell types, using tissues from the heart, brain, lungs, stomach, skin, and more. When the researchers overlaid genetic variants known to raise disease risk, specific pairings emerged like variants for blood-glucose regulation concentrated in endocrine cells, atrial fibrillation variants in heart muscle cells, balding variants in skin fibroblasts, and bipolar disorder and schizophrenia variants in excitatory and inhibitory neurons.

“A lot of the genetic variation that predisposes someone to disease is in noncoding parts of the genome,” said co-corresponding author Jesse Dixon, MD, PhD, associate professor and Helen McLoraine Developmental Chair at Salk. “By adding in the 3D genome aspect, we can potentially bridge that gap—connecting noncoding variations with the genes they affect in specific cells and tissues.”

Microglia, illustration. Researchers from the New York Genome Center and Columbia University used the atlas’ cross-tissue methylation data to show that a substantial fraction of the brain’s resident immune cells (microglia) are replaced by cells resembling blood monocytes between roughly ages 50 and 75. The finding challenges the long-held view that microglia persist from embryonic development throughout the life span. [Artur Plawgo/Getty Images]

What happens when two epigenetic lenses disagree?

One of the study’s most surprising findings is that DNA methylation and 3D genome structure don’t always tell the same story about a cell. In skeletal muscle, the team found fibers that look like mature, differentiated muscle cells by their 3D genome folding, but still carry the methylation signature of muscle stem cells. The reverse almost never happens. The most plausible explanation, they explained, is that these cells are caught mid-differentiation, with 3D architecture updating first and methylation catching up.

Similar mismatches appeared in Schwann cells of the peripheral nervous system and in placental trophoblasts. The pattern suggests that different epigenetic features update on different time scales during cell state transitions—a finding that could reshape how researchers define “cell type” in adult tissues and how they track cells moving between states in disease.

The atlas also revises a long-standing assumption about “non-CG methylation,” an unusual form of methylation previously thought to be largely confined to brain cells and stem cells. The study shows that it carries cell-identity information across many human tissues, including muscle, pancreas, and immune cell types, at lower but biologically meaningful levels.

“The inconsistency between modalities may be further used to determine what cell populations are switching between each other in adult tissues and diseases, which could, for example, expand our understanding of cancer cell dynamics,” said co-first and co-corresponding author Jingtian Zhou, PhD, a former graduate researcher in Ecker’s lab who now leads his own lab at the Arc Institute.

A public resource for scientists and artificial intelligence

To make the atlas broadly usable, the team built an interactive web browser that lets researchers visualize DNA methylation and 3D chromatin contacts across every tissue, cell type, and subtype in the study. The underlying data, including 195 billion methylation measurements and 18 billion chromatin contacts, are freely available.

The resource arrives as artificial intelligence tools are increasingly used to predict the functional impact of genetic variants. Atlases like this one can provide the labeled, cell-type-resolved training data that models need to make accurate predictions—a bottleneck that has historically limited the field.

For example, in a companion paper in the same issue of Science, a study led by Bing Ren, PhD, from the New York Genome Center and Columbia University used the atlas’ cross-tissue methylation data to show that a substantial fraction of the brain’s resident immune cells, called microglia, are replaced by cells resembling blood monocytes between roughly ages 50 and 75. The finding challenges the long-held view that microglia persist from embryonic development throughout the life span.

“DNA methylation patterns are specific to each cell type and analogous to a cellular barcode,” said Ren, who also co-authored the Salk-led study. “The comprehensive cross-tissue DNA methylation atlases show that the aging microglia in the human hippocampus more closely match the monocytes from peripheral blood than microglia from young adults, providing a crucial clue for the biological identity of these cells.”

The NIH 4D Nucleome consortium, of which this study is a part, aims to extend this kind of mapping into the fourth dimension: time. A 4D understanding of the genome—how its structure and chemistry change as cells develop, age, and respond to disease—remains a major goal, and the cross-tissue atlas provides reference scaffolding that future time-course studies will build on.

Along with scientists from the Salk Institute and Arc Institute, investigators from the University of California, San Diego, Columbia University, New York Genome Center, University of California, Los Angeles, Harvard, Henan University in China, Vanderbilt University, Stanford University, Broad Institute, University of Sheffield in the U.K., Yale, University of Florida, University of Freiburg in Germany, University of Graz in Austria, and Nanchang University in China; and Chongyuan Luo also contributed to the Science paper.

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Single-Cell Maps Reveal Genome Reorganization in Alzheimer’s Brain Cells

Edusehat — Fri, 24 Jul 2026 07:55:08 +0700

While Alzheimer’s disease is the most common cause of dementia, many of the molecular mechanisms that drive its progression remain poorly understood. While researchers have cataloged changes in gene activity across different brain cell types, a key unanswered question has been how the genome’s 3D organization influences those changes. Now, researchers have linked alterations in genome folding to disrupted gene regulation in Alzheimer’s disease, providing a new layer of insight into the biology of neurodegeneration.

The findings, published in Science in the paper “Single-cell multiomics connects 3D genome and transcriptome alterations in Alzheimer’s disease,” were reported by researchers from Carnegie Mellon University’s School of Computer Science, the University of Pittsburgh School of Medicine, the University of Washington, and collaborating institutions. Using single-cell multiomics, spatial transcriptomics, and artificial intelligence (AI), the team generated a multiscale view connecting genome structure, gene expression, and tissue organization in Alzheimer’s disease.

To investigate the role of genome architecture in Alzheimer’s disease, the researchers analyzed postmortem prefrontal cortex tissue from individuals with and without the disease. They used GAGE-seq (genome architecture and gene expression by sequencing), a technique that measures both gene expression and physical genome contacts in the same single cell. The team combined those data with chromatin accessibility data, spatial transcriptomic maps, and a transformer-based AI model called Hicformer, which integrates DNA sequence and 3D genome features to predict cell-type-specific gene activity.

The study revealed widespread changes in chromatin organization across major brain cell types. According to the paper, Alzheimer’s disease was associated with “reduced short-range interactions and increased longer-range interactions” within the genome. Active and inactive genomic regions also exhibited increased mixing, consistent with weaker compartment segregation. The researchers linked these structural changes to cell type–specific alterations in gene expression programs involved in disease-relevant pathways.

Researchers also observed weakening of promoter-proximal interactions and changes in regulatory elements, alongside evidence of senescence-related activation in microglia and sex-dependent dysregulation of X-linked genes in females. Integrating the molecular data with spatial transcriptomics revealed altered cellular neighborhoods and disrupted coordination of gene programs within diseased brain tissue. The authors wrote that the results connect “genome structure, gene regulation, and tissue organization through a unified multimodal analysis.”

Their predictive model Hicformer also demonstrated that “3D genome features provide information beyond DNA sequence alone for explaining AD-relevant gene expression, enabling prioritization of distal regulatory elements whose effects are mediated through chromatin contacts,” the authors wrote.

“Measuring gene activity and genome folding in the same cell allows us to directly connect chromosome structure with disease-related gene programs,” said Yang Zhang, PhD, a project scientist in Carnegie Mellon’s Computational Biology Department and co-lead author. “Across several kinds of brain cells, this paired view revealed a consistent signature of 3D genome reorganization in Alzheimer’s disease and helped us prioritize regulatory regions for future mechanistic and therapeutic investigation.”

The researchers concluded that genome folding represents a previously underappreciated regulatory layer associated with Alzheimer’s pathology. By creating a detailed map linking 3D genome remodeling to gene expression and tissue organization, the study provides a framework for future experiments aimed at determining which structural changes contribute directly to disease progression. This may also provide clues to future therapeutic focuses.

“Alzheimer’s disease cannot be understood one layer at a time,” said senior author Jian Ma, PhD, the Ray and Stephanie Lane Professor of Computational Biology at Carnegie Mellon University. “The genome’s 3D structure is a fundamental regulatory layer that helps to connect DNA sequence to gene activity. By integrating genome folding, cell state, and tissue context, we can move beyond cataloging disease-associated changes toward understanding how they fit together and which mechanisms to test next,” said Ma. “Alzheimer’s disease cannot be understood one layer at a time.”

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Midlife Brain Aging Linked to Immune Cell Remodeling, Blood-Brain Barrier Decline

Edusehat — Fri, 24 Jul 2026 04:20:11 +0700

New data from a National Institutes of Health-funded study shows that midlife, the immune cell landscape of the hippocampus, undergoes substantial remodeling. It points to a potential mechanism by which aging may contribute to the chronic neuroinflammation commonly seen in neurodegenerative disease. Details are published in a new Science paper titled “Epigenetic and 3D genome reprogramming during the aging of human hippocampus.”

The work was done by a collaborative team of scientists from the University of California, San Diego, the New York Genome Center, and the University of California, Irvine. According to the paper, the scientists analyzed postmortem hippocampal tissue from 40 neurologically healthy adults aged 20 to 95 years old.

Digging into the details, the scientists used traditional measures of gene expression alongside more advanced techniques to analyze the genome’s 3D architecture and epigenome. “Gene expression tells us what a cell is doing today, but epigenetic signatures preserve information about where a cell came from,” said Nathan Zemke, PhD, director of single-cell genomics at the UC San Diego Center for Epigenomics and first author on the study. “By combining these approaches, we uncovered a major shift in the identity and lineage of immune cells in the aging human brain’s immune cells that gene expression data alone would not have revealed.”

They found that the brain’s primary immune cells progressively decline from age 50 to 75 years of age, and are replaced by cells with elevated inflammatory signatures and other features that resemble the characteristics of peripheral blood-derived immune cells. It raises questions as to whether microglia, which emerge during embryonic development, may not renew throughout the human lifespan as previously thought. The data also showed that cells that typically maintain the protective blood-brain barrier deteriorated with age. And across many brain cell types, aging accompanied a widespread and coordinated disruption of genome architecture.

“The progressive structural disruptions were closely linked to shifts in gene regulation and cell identity, potentially revealing a fundamental feature of aging in the human brain,” said Bing Ren, PhD, scientific director and CEO of the New York Genome Center, and professor of genetics and development at Columbia University. Ren is also a corresponding author on the study,

Future studies will investigate the mechanisms driving the loss of resident microglia and determine whether the newly identified immune-cell transition contributes directly to Alzheimer’s disease and other age-related neurological disorders. Insights from the current study as well as others could provide new opportunities to develop therapies that help to preserve brain function and reduce vulnerability to neurodegenerative disease.

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Turning Solar Power Into Protein

Edusehat — Fri, 24 Jul 2026 04:20:09 +0700

According to projections from the United Nations, global food demand could increase by around 60 percent by 2050, while only about two percent additional agricultural land is expected to become available. Researchers at the Technical University of Munich (TUM) report that they are exploring new approaches to safeguard food security. A team at the TUM Campus Straubing has developed a process for producing crucial amino acids from carbon dioxide, hydrogen, and renewable energy.

Viktoria Lehmann, a doctoral candidate at TUM, describes one potential application for biotechnologically produced amino acids.

“A dairy cow needs far more than the grass growing in its pasture. High milk yields require supplemental protein, which is typically supplied through animal feed. These feeds are enriched with amino acids, the chemical building blocks of proteins,” she explains. “Across livestock production systems worldwide, millions of tons of amino acids are used as feed additives. However, their production consumes large amounts of land, water, and other resources. We wanted to find a more resource-efficient way to meet this protein demand.”

In a recently published study “Plug and Play – Enzymatic Amino Acid Production from Methanol and Carbon Dioxide” in Nature Communications, the team demonstrated its approach. The concept behind it: solar energy is converted into electricity using photovoltaic systems. This electricity is used to generate hydrogen, which, together with carbon dioxide, is converted into methanol—an alcohol widely used in industry as a chemical precursor. Specialized enzymes then convert the methanol step by step into amino acids. Which amino acid is produced depends on the specific enzymes used.

“Plants use sunlight to build biomass, but they are relatively inefficient at doing so. We are investigating an alternative pathway in which renewable energy is first converted into chemical energy carriers and then into valuable protein building blocks,” notes Volker Sieber, PhD, professor of chemistry of Biogenic Resources and Rector of the TUM Campus Straubing. “In the long term, this approach could help make more productive use of available land and enable a more sustainable production of amino acids.”

A modular platform technology

In 2023, the researchers demonstrated the production of the amino acid L-alanine from green methanol. Their latest work expands the approach to a total of seven amino acids. “Our modular plug-and-play concept can be compared to a construction kit,” says Vivian Willers, PhD, whose doctoral research laid the foundation for the study. “What started with a single amino acid is increasingly evolving into a platform technology for producing protein building blocks from renewable energy.”

The team successfully produced the amino acids glycine, serine, L-aspartic acid, L-valine, L-glutamic acid, and L-proline. In the future, this technology could help reduce dependence on protein-rich feed ingredients such as soy, which are not always produced sustainably. These amino acids are also key components of nutrient media used in cultured meat production. As a result, the researchers see applications extending well beyond conventional agriculture.

While the team was able to demonstrate the entire process chain—from carbon dioxide via methanol ultimately to amino acids—the current production volumes are still too low for commercial use. The researchers are working to further improve the performance of the enzymes involved.

“Our work is primarily a proof of technological feasibility,” points out Sieber. “We have shown that a broad range of biologically relevant amino acids can be produced from CO₂-based methanol. This opens up new possibilities for the sustainable production of protein building blocks.”

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Autism-Like Traits in Mice Improved After Single Rapamycin Dose

Edusehat — Fri, 24 Jul 2026 04:20:08 +0700

The results of a preclinical study led by UCLA Health researchers suggest that inflammation during pregnancy in mice can trigger autism-like brain and behavior changes in offspring, and that the effects may be rapidly but temporarily reversible in adulthood with a short-term dose of the immunosuppressive drug rapamycin.

The study showed that a single dose of rapamycin rapidly improved changes including brain overactivity, seizure risk, sensory sensitivity, repetitive behaviors, and abnormal brain functional network organization. Rapamycin itself is not considered a viable candidate for human therapy, as the effects of the drug were found to be temporary, with repeated dosing losing efficacy, and repeated use also having the potential for toxicity. However, the researchers said the study findings indicate that some autism-related brain changes may still be treatable in adulthood, and point to possible therapeutic approaches that target the underlying pathway rather than only symptoms.

“These results reframe how autism-associated symptoms might be treated,” said Janel Le Belle, PhD, an associate professor in the UCLA Department of Neurosurgery. “If the adult brain remains capable of functional normalization, then some features of autism may be successfully addressed without needing to correct underlying structural differences.” Le Belle is first author of the researchers’ published paper in Nature Communications, titled “Acute rapamycin treatment reveals distinct mechanisms of dysfunction in a maternal inflammation mouse model.”

Neurodevelopmental disorders result from the disruption of brain development in utero or in early life, with genetic, environmental, epigenetic, and immunological factors all potential contributors to complex pathogenesis, the authors wrote. Previous studies have shown that offspring of mothers who experience inflammation while pregnant have a higher likelihood of developing autism-associated traits such as repetitive behaviors and difficulty with social interaction, as well as brain overgrowth and disrupted sensory processing that continue into adulthood. “Maternal inflammatory response (MIR) during early mouse gestation induces a cascade of physiological and behavioral changes associated with autism spectrum disorder (ASD),” they stated.

Rapamycin has been shown in previous mouse autism studies to improve symptoms by suppressing an overactive mTOR pathway that signals cell growth and proliferation. What has been less clear is whether these brain changes could still be modifiable in adulthood, and whether rapamycin’s benefits came from long-term structural repair or faster functional changes. “We wanted to understand the mechanisms that underlie the effects of adult mTOR inhibition, where treatment isn’t aimed at preventing or reversing structural brain abnormalities,” the team stated.

For their newly reported study the scientists exposed pregnant mice to a mild inflammatory trigger early in gestation at a dose that was too low to make the mothers significantly ill. The resulting offspring went on to develop chronic brain and body-wide inflammation, mild brain overgrowth, overactive cell-signaling in the mTOR pathway, disorganized brain functional network connectivity and behaviors associated with autism.

When researchers gave adult offspring a single dose of rapamycin they found rapid improvement across nearly every measure. Neurons that had been firing abnormally calmed down, susceptibility to seizures dropped, brain regions that had been miscommunicating reorganized into more typical patterns and repetitive behaviors and sensory over-responsivity eased. These changes occurred within roughly two hours of drug administration, which was too rapid to be explained by the kind of physical rewiring of brain synapses that typically takes longer.

“The level of functional normalization achieved over this short time suggests new mechanisms by which possible treatments may act,” said the study’s senior author Harley Kornblum, MD, PhD, director of the UCLA Intellectual and Developmental Disabilities Research Center in the Semel Institute for Neuroscience and Human Behavior. “It suggests the adult brain may be more adaptable than we assumed, even when the underlying structural changes from early development are still there. This points us toward the brain’s functional circuitry, not just its physical structure, as a target for future treatment approaches.”

To understand the mechanisms of rapid rapamycin effects, researchers examined gene activity in brain cells before and after treatment. They found that rapamycin reversed abnormal expression of genes tied to autism, epilepsy and ion channel function, particularly in excitatory neurons, suggesting the drug works by quickly rebalancing brain cell excitability rather than by repairing structural brain differences.

The findings suggest that mTOR pathway activity, brain network organization and neuronal excitation levels as potential targets for future therapies aimed at specific autism symptoms such as sensory over-responsivity, a common but difficult-to-treat symptom of autism. “Our findings demonstrate that mTOR dysregulation drives dysfunctional brain development in MIR offspring but the adult brain remains amenable to rapid functional normalization, rescuing core and comorbid ASD associated brain and behavior phenotypes,” the authors stated.

Co-senior author and professor in the UCLA Department of Neurosurgery, Neil Harris, PhD, cautioned that the results showed the treatment effects to be temporary and that daily dosing produced tolerance over several weeks. This, along with rapamycin’s high potential for toxicity and the fact that these studies were performed in mice, makes it unsuitable for broad use in humans. “This points toward new therapeutic targets like sensory circuit neuromodulation or balancing neuronal inhibition and excitation, rather than toward rapamycin itself as a treatment,” Harris said. As the authors further commented in their paper, “Restoring excitatory/inhibitory imbalance and sensory functional network modularity may be important targets for therapeutically addressing multiple ASD phenotypes.”

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FDA Advisory Committee to weigh whether to expand compounding of unapproved peptides

Edusehat — Fri, 24 Jul 2026 00:50:03 +0700

The FDA’s Pharmacy Compounding Advisory Committee (PCAC) will consider this week whether seven unapproved peptides should be added to the Section 503A Bulk Drug Substances List, a decision that could dramatically expand compounding of these unapproved substances.

Why It Matters

Advisory committees provide non-binding recommendations to the FDA for consideration. The FDA then makes an independent decision. A positive decision from the FDA would set a dangerous precedent, allowing compounded drugs that have not met the FDA’s gold standard for safety and effectiveness to flood America’s pharmaceutical supply chain, and have far-reaching implications for patient safety, biomedical innovation, and public trust.

Patient Safety

The FDA’s mission is to protect the health and safety of Americans. None of the seven peptides have been approved by the FDA, and none have established clinical evidence demonstrating they are safe or effective. Several have unresolved safety concerns, and experts at the FDA have recommended against expanding access to these seven peptides.

Further elevating safety concerns is the reality that compounding pharmacies are not subject to the same manufacturing standards or adverse reporting requirements as brand and generic manufacturers. Adding these substances to the Bulk Drug Substances List would do an end-run around long-standing evidence standards meant to protect the quality, safety and effectiveness of America’s medicine cabinet.

Innovation

Developing new treatments depends on a predictable, science-based regulatory pathway. The FDA approval process is essential to innovators who invest in the vital research, clinical development, and manufacturing controls necessary to ensure medicines are brought safely and effectively to the patients in need. Allowing unapproved active ingredients to enter the U.S. market through compounding would usurp the necessary oversight to protect patients.

Over time, an unregulated pathway would weaken incentives that have sustained American leadership in biomedical innovation and could easily be exploited as a perceived signal of regulatory legitimacy. This issue is not limited to peptides. The outcome has implications for every innovative research company developing new medicines and for patients who depend on them.

Public Trust

The decision will also shape confidence in the FDA’s evidence-based regulatory process. Adding these peptides to the list would blur the critical distinction between FDA-approved medicines and compounded products made with unapproved active ingredients. Blurring those lines will sow confusion and distrust about which medicines have met the FDA’s gold standard for safety, effectiveness and quality—and which products haven’t.

Also eroding public trust are apparent conflicts of interest surrounding members of the advisory committee. Six of the 19 PCAC members evaluating the peptide proposal reportedly have professional or financial ties to businesses that market or promote peptides under consideration. This is highly unusual and raises questions about potential self-dealing and the importance of transparency and public confidence in the advisory process.

Advocates Weigh In

Stakeholders across the pharmaceutical supply chain—including BIO—are urging the FDA’s compounding advisory committee to recommend against adding these unapproved substances to the 503A Bulks List, warning it would “risk patient safety, weaken incentives for FDA approval, create uncertainty throughout the legitimate pharmaceutical supply chain, and foster the expansion of an unregulated market …”

BIO’s full comments can be found here.

What’s Next

PCAC will meet July 23-24 to review the nominated peptides and make recommendations to the FDA, which ultimately has the final say on whether they are added to the 503A Bulks List. Advocates who want to protect patient safety, public trust and future innovation will be watching closely.

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Supercooled kidneys have been transplanted into pigs in a “landmark achievement”

Edusehat — Fri, 24 Jul 2026 00:50:02 +0700

When it comes to organ donation, time is everything. As soon as an organ has been carefully removed from a donor’s body, it starts to deteriorate. Surgeons have a matter of hours to get it into a recipient. Leave it too long and the organ will become unusable.

In most cases, organs will be kept on ice during that time, at around 4 °C (39 °F). They cannot be frozen—in previous attempts, ice has formed, causing all kinds of damage.

Matthew Powell Palm at Texas A&M University and his colleagues have an alternative solution—a device that allows organs to be cooled to -4 °C (25 °F) without forming any ice.

Now, in new research with pig organs, his team has shown that kidneys, at least, can be supercooled and preserved in the device for days. Once rewarmed, the organs have been successfully transplanted into animals, and they seem to do better than organs kept on ice.

The work represents “a landmark achievement,” says Kevin Myer, president and CEO of LifeGift, an organ procurement organization based in Texas, who was not involved in the research.

Cooling organs

Powell Palm hopes this approach could ultimately help ease the organ shortage crisis. Today, there are more than 104,000 people waiting for a kidney transplant in the US alone. It is estimated that 17 people die every day in the US while waiting for a transplant. That’s partly due to a lack of donated kidneys, but it’s also because many of those that are available never make it to a recipient. In some years, around one in three donated kidneys end up being discarded, often because they end up too degraded to use by the time they reach a recipient. Kidneys can be stored on ice for around 24 hours or placed in devices that aim to mimic the conditions of the body, also for up to around 24 hours. That’s not always long enough to find a suitable recipient and transport the organ, says Myer.

Scientists around the world have been working on ways to store organs for longer by cooling them to even chillier temperatures. Cooling an organ slows its metabolism—the colder you go, the greater the effect, and the longer you can store it.

We’ve long been able to successfully cryopreserve eggs, sperm, and embryos, but it’s much harder to freeze large organs. Teams have been exploring various temperatures and cryoprotectants (chemicals that essentially work like antifreeze), but so far no one has been able to freeze human organs for transplantation.

As a thermodynamicist, Powell Palm explored another approach. By keeping an organ submerged at a constant pressure, it should be possible to prevent the formation of ice at temperatures a little below 0 °C, without the need for cryoprotectants (which might have side effects and would need to be approved before being used in human transplants).

To test this theory, Powell Palm and his colleagues have created a device that does just that. The device itself is essentially a hermetically sealed chamber with a transparent lid. At its base is a device that monitors the organ’s temperature and checks for the formation of ice. Organs are submerged in a solution that is already commonly used to preserve them for transplant. “I always describe this as low-tech high science,” says Powell Palm. “A lot of work has gone into understanding the … kinetics at play in this system, but ultimately … it’s quite simple.”

Supercooled kidneys

To test their device, Powell Palm and his colleagues first removed single kidneys from pigs. The organs were flushed with the same commonly used solution to remove the blood, just as transplant organs are. The team then kept some kidneys on ice for either two hours or 24 hours, to mimic standard conditions used in human transplantation. They also put some of the removed kidneys in their device for 24, 48, or 72 hours.

The stored kidneys were then each transplanted back into the original donor pigs. Each pig’s second kidney was removed in the same procedure, leaving each animal with only the kidney that had been stored, and reimplanted.

Once the 24-hour supercooled kidneys were transplanted, they immediately began producing urine—a key indication that they were working. The team members also measured other markers of kidney function and found that the organs appeared to be working normally within about 10 days of being transplanted.

A kidney that was supercooled for 72 hours recovers once it is transplanted back into a pig.

COURTESY RONALD SELLERS, POWELL-PALM LAB, TEXAS A&M UNIVERSITY

That’s slower than kidneys stored on ice for two hours but much quicker than kidneys kept on ice for 24 hours, says Powell Palm.

The organs that were kept supercooled for 48 and 72 hours performed similarly, he says. “Even at three days—triple the clinical standard—we’re getting recovery that is faster than … [what has been] the gold standard for the last three decades,” he says. “So we’re really, really pumped about this.”

“It is impressive,” says Heidi Yeh, a transplant surgeon at Mass General Brigham for Children, who also researches organ preservation technologies. “Often kidneys that have been stored for 48 hours [in other studies] take a week or two before they start working again.”

Organs that grow

The supercooled organs seem to work well in the long term, too. Over a 30-day period, the pigs grew by around 30%—and the kidneys grew with them, almost doubling in size to compensate for both the pigs’ growth and the lack of a second kidney. The team monitored one of the pigs for 200 days before removing and analyzing its kidney. Even at that point the organ looked healthy, says Powell Palm. He and his colleagues presented the findings at the American Transplant Congress in Boston last month.

Earlier this year, researchers in Canada showed they could also cool pig kidneys to below-zero temperatures and transplant them into pigs. The team’s protocol included the use of a cryoprotectant, and organs were stored for up to 48 hours before being transplanted into pigs. Those organs survived for a week.

In supercooling organs for 72 hours and showing that they do well for 30 days or more, Powell Palm and his colleagues have broken new ground. “It’s the first time this has ever been reported in history,” he says.

Those extra hours could make all the difference, says Myer of LifeGift. The advance could give doctors more time to evaluate the kidneys, match them to the most suitable donors, and physically get the organs to their intended recipients in time. It could enable international donations and open up cheaper transport options, he adds. “Right now, with kidney transplantation the assumed limit is 18 to 24 hours,” he says. “If we can get up to 72 hours … that would change everything.”

Powell Palm and his colleagues think they may even be able to go beyond 72 hours. In preliminary studies, organs that had been stored for up to 120 hours appeared healthy, although those organs have not yet been transplanted.

And because the process doesn’t require any cryoprotective chemicals, the team members are hoping for an accelerated approval from the US Food and Drug Administration, which would allow them to test the device in human transplantations.

The storage device is simple and compact, so Powell Palm thinks it will be easy to transport. It hasn’t been tested for air travel yet, but it has been used to take supercooled kidneys across the US in the back of a Kia Sorento, he says: “From a stability perspective, we view this as an even higher bar.”

Powell Palm and his colleague Sebastian Giwa plan to launch a company dedicated to developing the technology, along with other protocols that “stop biological time,” in the coming months, he says.

Samsung Proposes Offer to Acquire Swiss CDMO Specializing in Peptides

Edusehat — Thu, 23 Jul 2026 21:05:09 +0700

Samsung Biologics made an all-cash public tender offer of approximately $1.8 billion to acquire Switzerland-based PolyPeptide Group, a CDMO specializing in peptide-based active pharmaceutical ingredients (APIs).

Samsung views the deal as expanding its capabilities beyond antibodies and ADCs to include peptide therapeutics, particularly in obesity and diabetes, including GLP-1 therapies, while advancing innovation across high-growth areas such as oncology and other emerging indications. The transaction brings together Samsung Biologics’ global manufacturing scale with PolyPeptide’s specialized peptide expertise to create a differentiated, end-to-end multi-modality CDMO platform, notes a Samsung spokesperson.

PolyPeptide operates an integrated development-to-commercial model with growth focused on a modular, automation approach which, the company points out, gives it the flexibility to adapt quickly to changing market demand.

The planned acquisition extends beyond adding capacity in that it also lays the foundation for Samsung Biologics’ next phase of growth, supported by a strong pipeline of active peptide projects that includes a deep late-stage portfolio, notes a Samsung official. PolyPeptide operates global sites across Sweden, Belgium, France, the U.S., India, together with a corporate office in Switzerland and a separate Innovation Center in Strasbourg, France, with capabilities in R&D, development, and commercial manufacturing.

Upon completion of the transaction, Samsung will bring together PolyPeptide’s experienced team and specialized peptide expertise with Samsung’s scientific and manufacturing strengths and global operations, says John Rim, chairman of the board of directors and CEO of Samsung Biologics.

“This acquisition reinforces our long-term growth strategy by not only broadening our service portfolio with modality expansion into peptides including GLP-1, but by also boosting our geographic reach and proximity further within the U.S., Europe, and India,” continues Rim.

“After a comprehensive review of strategic options, the Board is convinced that Samsung Biologics’ offer is compelling for our shareholders, delivering an attractive cash price and immediate, certain value today,” adds Peter Wilden, chairman of the board of directors of PolyPeptide. “At the same time, it represents a transformational opportunity to accelerate our strategic ambitions at a scale we could not reach alone.”

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Hybrid Single-Use and Stainless-Steel Plants the Greener Option

Edusehat — Thu, 23 Jul 2026 02:45:15 +0700

Hybrid biopharmaceutical manufacturing facilities that combine stainless steel (SSTs) and single-use (SUTs) technologies are the “sweet spot” from an environmental sustainability perspective, according to new research. The research compared the environmental impact—specifically, the carbon footprint— of hybrid facilities with those where SUTs are used, arguing that ongoing efforts to decarbonize energy generation may have shifted the metrics.

And the key findings are that the CO₂ footprint of SUTs is significantly higher than previously assumed and that key SST process steps can now have a lower carbon footprint than their single-use counterparts.

Lead author Jan Reiners, rer. nat. , from Roche, tells GEN, “For a long time, SUT was considered more sustainable because it avoids the energy needed to clean and steam-sterilize stainless steel. However, this paradigm has now inverted due to the rapid decarbonization worldwide due to the expansion of renewable energy.

“A single-use run at the 2,000L scale generates up to 6.5 tons of CO2e per batch only in plastic and packaging waste. We didn’t do this assessment for a full stainless-steel system, but the hybrid facility used less than half of plastics,” he adds.

Reiners and colleagues used “market-based emissions accounting” data for the analysis, noting it is becoming common for drug companies to decouple emissions from local energy supplies through tools like power purchase agreements and on-site photovoltaic (PV) installations.

“For instance, our own organization, Roche, has recently achieved its global goal of sourcing 100% sustainable electricity across all worldwide operations. And we are not alone: AstraZeneca is reducing energy emissions by 98% by 2026 and transitioning to 100% renewable energy for heat and power. Novartis claims to have become carbon neutral for energy in 2025, as do many other companies,” he says.

The researchers also found that some long-standing assumptions about SUT systems— for example, that they have lower HVAC costs—no longer hold.

“In modern biomanufacturing, cleanroom ISO classifications remain identical for both SUT and SST due to functionally closed processing, while the massive logistics and warehousing required to store SUT consumables actually expand physical space requirements,” continues Reiners. “And finally, the mass of fossil-fuel-based plastics was underestimated and its CO₂ impact actually increased by ~30% in updated global life cycle databases like Ecoinvent v3.12.”

Hybrid alternatives

Considering the findings, Reiners and co-authors suggest that, to minimize emissions and costs, biopharmaceutical manufacturers need to combine stainless-steel systems with SUTs.

“Although SUT is less sustainable, it has other advantages like faster product changeover, more flexibility, less investment, but higher running costs. A hybrid facility is the ‘sweet spot’ of combining SUT and stainless steel to reduce the CO₂ impact,” he maintains. “Depending on the facility layout, various measures can reduce the footprint, but per se it is about retaining stainless steel for simple, high-volume, resource-heavy operations—like buffer and media preparation—while keeping single-use for the complex, flexible core process like bioreactors.

“In our study, comparing a full-SUT facility to a hybrid facility reduced the material-related carbon footprint by 62%, dropping emissions from 6.5 tons of CO2e to just 2.8 tons of CO2e per batch.”

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Scaling Cell and Gene Therapy Manufacturing

Edusehat — Thu, 23 Jul 2026 02:45:14 +0700

As cell and gene therapies (CGTs) move closer to mainstream clinical use, manufacturers are under growing pressure to bridge the gap between scientific breakthroughs and commercial reality. To explore this transition, GEN talked with three industry leaders, and here’s what we learned.

GEN: In today’s bioprocessing for cell and gene therapies, what is the most crucial business challenge and why?

Jonathan Wofford, COO, Title21 Health Solutions: Currently, the most crucial business challenge to solve in cell and gene therapy bioprocessing is scalable, cost-effective manufacturing. While scientific advances and clinical outcomes have accelerated the demand for therapy development, producing consistent, high-quality products at commercial scale remains difficult due to the increasing complexity of processes, limited automation, underdeveloped data-management infrastructures, supply-chain constraints, and patient-specific workflows for autologous therapies.

Justin Irizarry, CEO, OrganaBio: The disconnect between starting material and everything downstream. Despite heavy investment in manufacturing and analytics, the quality and consistency of raw biological material still determines whether a process succeeds. Variable, fragmented sourcing becomes failed runs, slipped milestones, and regulatory risk, and you can’t engineer that variability back out of a living product.

Carol Houts, CEO, Germfree: The most pressing challenge in CGT bioprocessing today is the gap between clinical promise and manufacturing readiness. Developers are advancing therapies faster than the infrastructure, workforce, and supply chain can scale to support them, and that mismatch is costing time and patients.

GEN: What is the most effective way to address these challenges?

Wofford: In theory, the approach should focus on end-to-end process standardization and automation, supported by closed-system manufacturing, digital process infrastructure, and scalable platform technologies. This reduces variability, decreases labor costs, improves regulatory compliance, increases throughput, and enables consistent product quality from clinical development through commercial production. I say in theory because adoption may be limited by the significant upfront investment needed to implement advanced manufacturing technologies.

Irizarry: Vertical integration and co-location: control the chain from donor through processing and testing under one quality system, with a recallable donor pool, identical SOPs across RUO and GMP, and processing near collection sites.

Houts: The most effective response is committing to manufacturing strategy early, not as an afterthought to clinical development. That means purpose-built environments, modular infrastructure that can grow with the program, and partners who understand GMP from day one. The groundwork determines whether a therapy ever reaches the people who need it.

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Cell-Therapy Manufacturers Get Creative on Checking Particulates

Edusehat — Thu, 23 Jul 2026 02:45:13 +0700

Manufacturers of cell-containing products are adopting new techniques to work around unclear regulatory guidance on particulates. That’s the experience of Diana Colleluori, PhD, principal chemistry, manufacturing and controls (CMC) consultant at Biologics Consulting.

According to Colleluori, cell-containing products can be harder to visually inspect for particulates, as they’re not clear and are often stored in opaque bags.

“Processing and testing cell-containing products already has challenges because they can’t be terminally sterilized,” she explains. “It’s also harder to make a visual assessment to meet regulatory requirements because they already contain cells and the final product is usually in a cell-freezing bag.”

The particulates are mostly (90%) plastic that enter the product from contact with the inside of single-use equipment used during manufacturing, she says, adding that other particulates can enter from product manipulation and, thus, it’s best to try to minimize this.

Companies that Colleluori has worked with have tackled this problem by running their process with the formulation buffer, but minus cells, she continues. This allows them to assess particulates in a clear solution, outside of a bag, which they can then extrapolate to running the same process with cells.

“By testing visible and sub-visible particles in those samples, you can prove your product contact materials are expected to meet the limits for your cell-containing products,” according to Colleluori.

The company can also run quality control tests on a small sample of the final product. Among the tests carried out are those based on the United States Pharmacopeia (USP) chapters 790 for visible particulates and injections,1790 for visual inspections of injections, and 788 for the sub-visible particles.

In addition, EU guidance on visual inspection of particles can also be relied upon. Which tests are run should depend on the method of administration, adds Colleluori.

“If you’re doing an intramuscular injection, it’s probably a little less rigorous than an intrathecal injection of products that have stricter regulations on the number of particles and sub-particles that can be present,” she says.

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AI Protein Engineering Model Designed for Biomanufacturing

Edusehat — Thu, 23 Jul 2026 02:45:11 +0700

A cell engineering model designed specifically for the biomanufacturing environment could go a long way toward optimizing host cell performance, reducing development timelines, and streamlining biomanufacturing platform design.

“There’s a real challenge in determining how to engineer a host cell’s genome to maximize its performance in a biomanufacturing application.” That’s the difficulty laid out by Shawn Manchester, PhD, CEO, Triplebar Bio, and a view that’s shared by Yun Song, PhD, professor, University of California, Berkeley, and the industry innovation organization BioMADE. The three, with support from the National Science Foundation, are developing such an AI-informed cell engineering model to solve that challenge.

What’s most notable is that this AI-informed predictive and optimization model focuses on the biomanufacturing environment. The large-scale, application-specific training database is being designed to handle bench- to commercial-scale biomanufacturing.

Biomanufacturing-relevant environment

As Manchester says, “There aren’t a lot of organisms that have naturally evolved to make proteins they’ve never seen before, in an environment they’re not well-evolved for. Our ability to generate data in a setting that is relevant to biomanufacturing and to do so at the scale of hundreds of thousands of cells and data points is innovative…and it’s not something that many others are looking at. Most people are working on AI for protein engineering of therapeutics and other molecules as opposed to cell engineering for use in biomanufacturing.”

This project focuses on data capture from Pichia pastoris as the host cell producing five different proteins that are relevant for bioprocessing, food production, and defense. The learnings will be incorporated into Triplebar’s other programs, too, including optimization for its Chinese hamster ovary (CHO) cells, Manchester says.

“Improving the scalability of these proteins directly benefits [not just bioindustrial or biopharmaceutical applications, but a broad range of] biomanufacturing,” Brandon Simmons-Rawls, program manager, BioMADE, emphasizes.

This AI project combines two core technologies deployed at Triplebar:

Droplet microfluidics, in which a cell is encapsulated in a water and oil emulsion alongside a fluorescent-based sensor to quantify protein production
Multimodal transformer-based AI model

The genomes and transcriptomes of cells that make either more or less protein than baseline are sequenced and fed into the AI’s very large, labeled training sets to identify correlations between genotypes and phenotypes. These correlations are important patterns about how the genome works, Manchester explains, and can be used to generate new genomic designs for optimization of the host cell to the biomanufacturing process.

“We first pre-train models on all relevant sequencing data from thousands of genomes, which gives us an underlying structure to the genome. Then we fine-tune those models with the genotype-phenotype data we generate in the biomanufacturing-relevant environment. This allows us to understand which of those patterns in the genome relate to biomanufacturing-specific performance,” Manchester says.

Once this AI-enabled model rolls out, Manchester says he envisions it being used by bioindustrial and biopharma companies that “need to improve the productivity or efficiency of the organism they use in manufacturing. Results should be faster and more efficient than those derived using guess-and-check methods based on our current understanding of how these organisms work.

“The goal is for people to log onto this AI tool, identify what they’re making and what they’ve done, and ask the AI what else they can do to improve performance,” he continues. “The model will serve them specific genetic designs that they can deploy in their organism.”

Manchester predicts that more than 10% of the designs will produce meaningful improvements—significantly more than traditional, early-stage design-build-test cycles. This streamlines the cell engineering cycle by focusing on modifications likely to yield performance improvements that are most meaningful for biomanufacturing.

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Transcripta Bio Raises $24M for AI-Driven Neurological Disease Therapies

Edusehat — Thu, 23 Jul 2026 02:45:10 +0700

Chris Moxham, PhD, has been a “drug hunter” for three decades, building data-driven platforms as vice president of quantitative biology at Eli Lilly, and CSO of Folcrum Therapeutics, in recent years.

“Technology is now advancing to allow us to interrogate the transcriptome, which is a phenomenal blueprint for cell state and fate,” he told GEN Edge.

Moxham currently leads Transcripta Bio as founder and CEO. The AI-driven drug discovery start-up is developing small molecule therapeutics to modulate gene expression disease signatures.

Transcripta has now announced a $24 million funding raise to advance IND-enabling studies and clinical preparation of its neurological disease portfolio, including autism spectrum disorder (ASD) and facioscapulohumeral muscular dystrophy (FSHD). Mayo Clinic and Omnimed will join JAZZ Venture Partners, BlueYard Capital, and a group of life sciences family offices, as investors.

Phase II clinical trials are “where the rubber meets the road,” says Moxham. He emphasizes that de-risking therapies early is key to improving drug discovery success rates, which often fall below 10%.

Founded in 2023, the Palo Alto-based company currently houses fifteen employees. “This isn’t a company you could have built five years ago,” highlighted Moxham. He cites the intersection of scalable sequencing technology, compute power, and lab automation among the factors enabling the rise of AI-driven biology.

Three-pronged approach

While much of the field has defined transcriptome AI models as the “virtual cell,” Moxham emphasizes Transcripta’s translational focus.

The company’s proprietary platform takes a three-pronged approach. First, disease signatures are identified using patient-derived single cell RNA-seq (scRNA-seq) data. An in-house generated “drug atlas” then measures the effects of small molecule perturbations across 80% of the transcriptome, capturing full dose-response profiles in diverse cellular contexts, including glutamatergic and motor neurons, fibroblasts, and keratinocytes. These data power AI models that identify promising compounds that can therapeutically modulate gene expression.

Transcripta’s neurological disease pipeline is structured as a tiered portfolio of novel molecules and repurposed clinical-stage assets, with the latter benefiting from existing human safety data that can shorten development timelines and lower costs.

In 19q12 syndrome, a form of ASD, the team demonstrated that entrectinib, an FDA-approved oncology drug, could reverse disease when given at low concentrations. One patient case demonstrated clinical benefit within nine months after taking the drug.

In Huntington’s disease, Transcripta’s platform identified novel molecules that could downregulate DNA mismatch repair protein and validated therapeutic target, MSH3. The company plans to file an IND next year. Transcripta is also pursuing pre-IND research in FSHD and myonic dystrophy.

Moxham emphasizes the generalizability of the platform. “We are now looking at hundreds of diseases with this type of approach,” he says.

Transcripta plans to introduce another cohort of therapeutic programs by Q2 of 2027.

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Transmissible Cancer Discovered in Freshwater Fish

Edusehat — Thu, 23 Jul 2026 02:45:09 +0700

Researchers headed by a team at the University of Vermont have discovered that mysterious black skin lesions afflicting catfish in Lake Memphremagog and other New England and Canadian lakes are caused by a transmissible form of cancer, the first ever identified in a freshwater fish species. The scientists’ studies showed that the cancer cells behave more like parasites than conventional tumors, moving from fish to fish.

The discovery marks only the fourth type of transmissible cancer identified in the animal kingdom, and the first in any fish or freshwater species. Previous ones include the Tasmanian devil’s facial tumor disease, a transmissible venereal tumor in dogs, and transmissible leukemia-like diseases in clams, mussels, and other bivalve mollusks. The researchers do emphasize that the disease poses no known risk to humans. The cancer cells cannot infect or survive in another species, and the fish are safe to handle and study.

The new discovery sheds light on how cancer can spread in the wild, and raises important questions about where this cancer originated, how it will affect fish health and populations, and also how cancer works in all animals including humans.

Julie Dragon, PhD, a researcher at the UVM Cancer Center in the Larner College of Medicine, suggests that understanding this newly discovered transmissible cancer could offer new insights into cancer biology more broadly. “By studying how cancers survive and spread outside their original host, we can learn a great deal about what keeps cancers contained—and what happens when those boundaries break down,” she said. “These fish provide an opportunity to look at evolution of cancer.”

Dragon is co-lead author of the researchers’ published report in Nature, titled “Brown bullhead catfish melanoma represents a novel transmissible cancer,” in which they stated, “We found extensive genomic evidence to suggest these melanistic lesions represent a transmissible cancer, to our knowledge, the first known occurrence in a fish species and the first found in a freshwater aquatic setting.”

Since 2012, anglers and biologists have reported a striking increase in brown bullhead catfish (Ameiurus nebulosus) with raised black skin patches in the cross-border lake shared by Vermont and Quebec. By 2014, nearly one in three fish showed the dark lesions. Because brown bullhead are considered indicators of environmental quality, the researchers suspected a contaminant or pathogen linked to pollution. “At first, we thought this disease might be a virus, but that wasn’t panning out,” said Dragon.

Instead, scientists found that these lesions turned out to be melanoma, a skin cancer. “This was surprising,” Dragon said, “and we wanted to know how a bottom-dwelling fish was getting a cancer we associate with exposure to too much sunlight.” For their reported study Dragon and colleagues took what she describes as a “deep dive,” into the genetics of the cancer cells.

UVM professor and cancer researcher Julie Dragon co-leads dissection and tissue collection of catfish with melanoma skin cancer at the Gateway Center on the shore of Lake Memphremagog, Newport, Vermont. Additional participants: Elizabeth Murchison, professor at the University of Cambridge, U.K.; her post-doctoral associate Zoe Clarke; USGS post-doctoral research fish health biologist Cheyenne Smith; her undergraduate student intern Sam Williams (not shown in these photos); Peter Emerson, fish biologist with the Vermont Department of Fish & Wildlife. The team sampled tissue from 17 fish. [University of Vermont]

Using whole-genome sequencing, the team compared DNA from tumors and healthy tissues in affected fish. They expected to find a genetic mutation that makes the fish susceptible to having their own cells become cancerous. Instead, they found that the cancer cells were much more closely related to each other than to their host fish—the signature of a clonally transmissible cancer, in which tumor cells themselves act as infectious agents.

“Hundreds of thousands of genetic variants are shared among tumor samples but absent from host fish, vastly exceeding levels seen in conventional cancers,” the authors reported in their paper.

The research team is now working to understand how the cancer cells spread between animals. “It seems to only happen in larger fish that are of spawning age,” said study co-lead Mark Henderson, PhD, fish biologist at UVM’s Rubenstein School of Environment and Natural Resources. “Maybe some part of spawning behavior leads to the spreading of the cancer between animals.”

The study notes that pollutants or hormonal changes could weaken immune systems, making the lake’s brown bullhead fish more vulnerable to infection. Naturally occurring arsenic may also play a role. The team did find that the fish cancer cells have elevated levels of arsenic and the geographic distribution of the cancer appears to have correlation with levels of arsenic in the lakes’ surrounding soil. “Arsenic is found naturally throughout New England, and there’s really high concentrations of it in the Vermont’s Northeast Kingdom as well as up through Maine, which is where we’ve also seen evidence of this cancer,” said Henderson.

The cancer’s ecological impact remains uncertain. Some transmissible cancers are devastating—the Tasmanian devil tumor has wiped out 90% of some populations. In contrast, the dog tumor has coexisted with its hosts and has done for thousands of years. In Lake Memphremagog, heavily diseased bullhead can survive for years, but long-term population effects are still unknown.

The researchers are beginning to explore how long this cancer may have been around, and how common transmissible cancers may be. They are exploring ponds and rivers in Massachusetts to learn more about the distribution of the cancer and to see if it may have originated there. “Our contention is that maybe it’s not as rare as everyone thinks. We’re just not seeing it,” said Dragon. “In the case of transmissible cancers, it may be that we don’t see many simply because we aren’t looking for them.”

In their paper the team concluded, “Although we cannot rule out a role for conventional pathogens in the original emergence or ecological transmission of this disease, the genomic evidence presented here overwhelmingly supports a transmissible cancer in which the tumor cells themselves act as the infectious entity.”

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Engitix Licenses Lonza’s Technology to Advance ADC Therapies

Edusehat — Wed, 22 Jul 2026 23:10:09 +0700

Lonza and Engitix signed a licensing agreement to advance the development of antibody-drug conjugates (ADCs) as therapies for patients with chronic diseases.

Engitix will access Lonza’s ADC technology platform through a single-target license, including the use of SYNtecan E linker-payload and complementary GlycoConnect^® and HydraSpace^®. These technologies are intended to support Engitix’s goal to develop and commercialize therapies.

Lonza, through one of its affiliated companies, 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 Engitix will work on the R&D, manufacturing, and commercialization of the ADCs.

Engitix has developed a proprietary human extracellular matrix (ECM) platform that allows the study of disease biology directly in human tissue, according to a company official, who adds that this approach focuses on targets within the ECM itself, offering a differentiated pathway compared to traditional methods that focus on cell surface targets.

The collaboration enables Engitix to leverage Lonza’s established ADC technology platform to develop differentiated therapeutic candidates designed to selectively deliver potent payloads to tumor-selective targets identified through Engitix’s proprietary human ECM discovery platform, explains Giuseppe Mazza, MD, PhD, CEO and co-founder of Engitix.

‘We are committed to translating our unique understanding of the disease microenvironment into transformative therapies for patients with high unmet medical need,” he continues. “Licensing Lonza’s clinically validated conjugation and linker-payload technologies provides us with a powerful toolkit to develop next-generation ECM-targeted therapeutics with the potential for enhanced efficacy, reduced toxicity and improved therapeutic index.”

“We are pleased to collaborate with Engitix on this innovative program,” says Jan Vertommen, vice president of commercial development, advanced synthesis, Lonza. “By combining our ADC development and manufacturing expertise with Engitix’s unique ECM-based discovery platform, we aim to advance next-generation ADCs and unlock new possibilities in targeted therapies for patients.”

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UCSD, IGI Partnership Expands CRISPR Innovation Across Health, Agriculture, and the Environment

Edusehat — Wed, 22 Jul 2026 12:25:08 +0700

California may be known as the Golden State, but it may well be referred to as the Genome Editing state after this week’s announcement of an inter-state partnership. The University of California (UC), San Diego, and the Innovative Genomics Institute (IGI), founded by Nobel laureate and CRISPR gene editing co-discoverer Jennifer Doudna, announced a new partnership to jointly develop cutting-edge genomic tools for addressing large-scale challenges in health and the environment.

Doudna founded the IGI in 2015 with the aim of translating the success of gene editing in the lab into practical solutions for large societal problems. Initially formed as a partnership between UC Berkeley and UC San Francisco with a focus on using CRISPR in human health, the IGI expanded to include sustainable agriculture and climate change applications of genome editing, adding UC Davis as a partner. UC San Diego is joining as the IGI’s fourth UC partner campus, combining research strengths in environmental and marine science, engineering and computer science, and biomedical and life sciences to develop the next generation of genome-editing tools and applications.

“I’m thrilled that the IGI is now partnering with UC San Diego,” said Doudna. “The mission of the IGI is to develop solutions that can not only scale to meet the biggest challenges in health and climate, but to make solutions that are accessible to those who need them most. UC San Diego helps us expand that real-world impact.”

An artistic depiction of a jumbo phage infecting a bacterium.
[Margot Riggi]

The relationship between UC San Diego and IGI research labs has been building over the past decade. For several years, the IGI has been collaborating with UC San Diego researchers Joe Pogliano, PhD, professor in the Department of Molecular Biology, and Kit Pogliano, PhD, dean and professor in the School of Biological Sciences, on applied microbiology projects. They have worked with UC Berkeley-based labs on developing new therapies focusing on “jumbo phage” for antibiotic-resistant bacterial infections as well as a novel defense strategy against viral infections.

In 2023, UC San Diego researchers were awarded $10 million by the Howard Hughes Medical Institute to explore the biomedical promise of jumbo phage as therapeutic agents. Also in 2023, the IGI received a $70 million gift through the Audacious Project to develop microbiome-editing tools and apply them to problems caused by microbes and microbiome imbalances, including livestock methane emissions and inflammatory diseases like asthma. Other areas of the effort focus on developing more efficient tools for genome editing across a wide breadth of microorganisms to expand the impact of this technology.

In the environmental arena, UC San Diego researchers have worked closely with UC Berkeley labs since 2018 to develop tools for combatting crop pests, managing disease-carrying mosquito populations, and developing safe protocols for environmental applications of genomic technologies.

“UC San Diego brings exceptional strengths across biological sciences, medicine, engineering, computation, environmental research, and ocean science that align powerfully with the IGI’s mission,” said Pogliano.

In addition to the current collaborations, IGI’s executive director Brad Ringeisen foresees multiple areas of synergy: “UC San Diego’s strengths in engineering, medical devices, environmental research, and the microbiome complement IGI’s current research areas, and allow us to expand our societal impact in new ways,” he said.

New joint projects of particular interest include interdisciplinary programs designed to bolster the climate resilience of our oceans and soils through innovative scalable solutions, and combining UC Berkeley’s biotechnology discovery engine with UC San Diego’s biomedical research and health system to improve the drug discovery pipeline for rapid translation across areas like antimicrobial resistance and neurodegenerative disease.

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Blood Cancer Therapies Informed by Spatial Remodeling of Bone Marrow

Edusehat — Wed, 22 Jul 2026 01:35:10 +0700

In a new study published in Leukemia titled, “Spatial remodeling of bone marrow architecture defines tissue-state signatures of disease activity and therapeutic response in myelodysplastic neoplasm,“ researchers from Weill Cornell Medicine have developed a new AI-based method to assess patients with a form of blood cancer called myelodysplastic neoplasms (MDS). The approach compares the size and shape characteristics of hematopoietic cells in the patient’s bone marrow sample with those in healthy bone marrow to generate a score that reflects disease severity.

MDS is a form of blood cancer that usually affects older adults. About one-third of patients progress to a more aggressive cancer called acute myeloid leukemia. MDS patients must undergo frequent biopsies to identify signs of remission or worsening disease.

“It’s fundamentally a chronic and progressive disease,” said Sanjay Patel, MD, clinical chief of hematopathology, and an associate professor of pathology and laboratory medicine at Weill Cornell Medicine. “With the current methods pathologists use to assess MDS samples, there are some clear-cut cases and a lot of gray area.”

The researchers developed a new method using fully deidentified patient samples that may provide more clarity for patients with MDS and their physicians.

“Using AI to assist us in looking at the spatial architecture of the bone marrow using widely available laboratory assays allows us to improve our capability to assess patients’ prognosis and perhaps triage them for precision therapies,” said David Redmond, PhD, assistant professor of computational biology research in medicine at Weill Cornell Medicine.

The MDS-Microarchitectural Perturbation Score (MDS-MAPS) score ranks patient samples based on 82 features associated with normal tissue and different genetic subtypes of MDS. The tool uses routinely collected samples, tissue staining protocols, and imaging technologies that could be implemented in most hospital pathology departments and laboratories.

“We can generate an MDS-MAPS value for a patient at diagnosis and track how it changes over time,” said Patel.

The next step will be to validate the method in larger cohorts of patient samples. The researchers will collaborate with Pinkal Desai, MD, associate professor of medicine at Weill Cornell Medicine and a hematologist/oncologist at NewYork-Presbyterian/Weill Cornell Medical Center, to study how the tool performs on other forms of MDS, and increasingly recognized precursor conditions.

“Patients with MDS and related precursor conditions have many mutations as part of the disease biology and each patient’s molecular signature is different,” said Desai. “We have always wondered if these mutations signal a different spatial pattern and whether these patterns have an impact in predicting how patients progress and respond to treatments. Together we are poised to harness technology to answer real-world clinical questions.”

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10x Clinical Expansion Advances with CLIA Lab Plans, Cleveland Clinic Partnership

Edusehat — Wed, 22 Jul 2026 01:35:09 +0700

10x Genomics says it is on track to build out a CLIA-certified laboratory set to open next year, part of the spatial and single-cell tools developer’s expansion into clinical diagnostics launched earlier this year.

The lab will open within 10x’s headquarters campus in Pleasanton, CA, 10x co-founder and CEO Serge Saxonov, PhD, told GEN.

Serge Saxonov, PhD, 10x Genomics co-founder and CEO

“This is one great benefit that we have from the fact that we’ve got all the infrastructure here, and that’s why we feel like we can really accelerate some of these kinds of applications: Because we have the space, we have the expertise with the technology, we have the people who really know all the ins and outs of it, and we can very quickly, validate, new assays, test them, refine them, optimize them,” Saxonov said. “We have been seeing that already, in the time that we have been standing up some of these pieces, how enabling it is to have it all under one roof at this stage.

“Building a CLIA lab is definitely not a trivial undertaking, but we’ve been making really great progress,” he added. “The team has been standing up these capabilities, and definitely on track for early next year. So, very much looking forward to that.”

The CLIA lab is a key component of 10x’s move into clinical diagnostics, announced in January. Traditionally focused on research tools for academic, government, and industry customers, 10x has moved this year to launch clinical collaborations with top-tier institutions—the most recent of which was announced last month with Cleveland Clinic.

The nonprofit multispecialty academic medical center is partnering with 10x in a multi-year collaboration aimed at advancing research in novel diagnostics for bladder cancer. Cleveland Clinic has agreed to contribute patient samples with appropriate phenotypes for analysis on 10x’s Flex Apex single cell sequencing and Xenium spatial biology platforms.

“They have great access to patients and the right kinds of clinical trials and therapies that are going through their system,” Saxonov said. “We are working together to run single-cell and spatial analyses on them, collaborating on those and correlating the biology that we learn from single cell and spatial with therapeutic outcomes.”

Bladder cancer biomarkers

10x and Cleveland Clinic aim to identify biomarkers that predict how bladder cancer patients will respond to emerging therapies, such as immunotherapies and antibody-drug conjugates (ADCs).

“Those biomarkers are definitionally known already, but the actual context of their expression isn’t really that well known in terms of being able to predict response,” Saxonov explained. “The question is, if you see their expression in the context of the cancer cells, or the tumor microenvironment, or the immune compartment, it will then also inform response to therapy. And there’s plenty of evidence from scientific literature that there’s a lot of signal there, a really, really powerful signal there. What hasn’t been done is run rigorous, well-powered, clinically, really carefully well-defined studies to measure and evaluate those kinds of biomarkers.”

Oncology is one of two therapeutic areas viewed as priorities for pursuing translational applications with an eye toward potential clinical diagnostics that address therapy selection and monitoring. The other area is autoimmune disease.

Saxonov asserted that 10x’s clinical push was unrelated to its established research business, which shrank last year as its traditional base of academic and government (A&G) customers reeled from cuts in research funding. The cut prompted 10x to announce plans to eliminate about 100 jobs—8% of its workforce—though the workforce appears to have only shrunk by 18 jobs or about 4% last year, from 491 full-time employees as of December 31, 2024, to 473 at the end of last year, according to the company’s form 10-K annual filings.

“We feel our research business gives us an awesome foundation to now invest in this future of clinical applications. It is a very, very much an enabling thing,” Saxonov said. “It gives us a great foundation from which to go forward. It was always our plan, always our mission, always the strategy of the company that over time, as we develop our technologies, we want most naturally to make them actually have a direct clinical impact.”

Several recent trends have combined to support clinical expansion, Saxonov said:

An increasing number of therapies whose effectiveness in patients, and in what combination, remains unknown to many doctors.
A growing body of single-cell spatial signals, such as gene and protein expression, mapped to the exact physical coordinates of individual cells within a tissue.
Increased maturing of single cell, spatial, and multiomics technologies, resulting in more data and higher quality insights that enable their use in the clinic.

‘A really nice position’

“Investments around workflow, investments around logistics, being able to work with distributed collected samples, and also being able to drive the costs down and scale up these technologies—all of that progress now puts us in a really nice position to lean into, first, generating clinical evidence for all these different, therapeutic areas, then taking the resulting information and deploying that in the context of diagnostic tests in the future,” Saxonov said.

“Independent of whatever might be happening in terms of the research market, which will fluctuate over time, is that several large-scale trends have been converging.”

Is 2026 shaping up as an up year or a down year for A&G? Saxonov said he’ll offer insights when 10x releases its second quarter earnings in August.

10x announced its clinical ambitions in January during the J.P. Morgan 44^th Healthcare Conference in San Francisco. The company unveiled clinical collaborations with two Boston-based institutions, Brigham and Women’s Hospital and Dana-Farber Cancer Institute, as well as the New York-based Cancer Research Institute.

The Cancer Research Institute collaboration focuses on generating “very large, AI-ready” data sets for immunotherapy, Saxonov said, while the Dana-Farber and Brigham and Women’s partnerships center more, like the Cleveland Clinic alliance, on generating clinical evidence for future diagnostics applications. Patient flows have been established, and analysis is underway in the collaborations with both Boston institutions.

“At the appropriate time, we’ll be updating the world about what we’re learning,” Saxonov added.

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Revvity Creates Program to Equip Young Biotechs with Scalable Informatics Capabilities Sooner than Later

Edusehat — Tue, 21 Jul 2026 22:00:09 +0700

Revvity reports that its Signals Software business is launching Signals for Startups, a new program designed to help emerging biotech companies adopt scalable informatics capabilities earlier in their growth journey.

Built for early-stage biotechs, the program combines access to Signals software with guided onboarding and best-practice configurations for smaller biotechs to help accelerate innovation, improve scientific productivity and shorten time-to-value, according to a company spokesperson.

Startup biotechs are often under pressure to move quickly with limited IT, informatics, and operational resources while managing increasingly complex discovery data. Signals for Startups addresses this challenge with a purpose-built, scalable Signals environment that helps teams focus on science while establishing a strong digital foundation from day one, explains Kevin Willoe, president of Revvity Signals Software, adding that out-of-the-box configurations for large and small molecules enable companies to accelerate adoption, standardize data, and enhance collaboration on a proven, scalable informatics infrastructure.

“Signals for Startups addresses a critical need for emerging biotech companies that want to move fast without creating data and workflow challenges that limit their ability to scale,” he continues. “By combining startup-friendly access with guided onboarding and scalable Signals workflows, we are helping early-stage teams build the digital foundation they need to advance discovery, support investor readiness and grow with confidence.”

Signals for Startups is expected to be available in the U.S., Europe, the Middle East, and Africa later this month.

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Early Genomic Indicators of Praziquantel Resistance in Schistosoma mansoni

Edusehat — Tue, 21 Jul 2026 11:10:07 +0700

The warning signs are subtle—genetic variants scattered across a vast parasite genome—but together they sketch a picture that disease‑control and elimination programs can’t afford to ignore. A new study published in Science Advances, “Extensive parasite transmission and variation in a functional receptor associated with drug resistance in endemic Schistosoma mansoni,” reveals genomic changes in the parasite that cause schistosomiasis, which may reduce its sensitivity to praziquantel, the only drug currently available to treat the disease.

The international team, led by researchers at the Wellcome Sanger Institute, the Royal Veterinary College, and the Medical College of Wisconsin, analyzed whole‑genome sequence data from 570 Schistosoma mansoni parasites collected across Africa and the Caribbean. As the paper noted, “Mass drug administration (MDA) with praziquantel is the cornerstone of schistosomiasis control and elimination efforts.” Yet after two decades of large‑scale treatment campaigns, the parasite’s genome is beginning to show signs of drug resistance.

The study uncovered extensive long‑distance transmission of S. mansoni and a striking degree of genetic diversity across endemic regions. But the most consequential finding lies in Sm.TRPM_pzQ, a transient receptor potential (TRP) melastatin ion channel recently identified as praziquantel’s molecular target. Researchers found four naturally occurring variants in this receptor with reduced praziquantel sensitivity.

In some cases, parasites persisted even after treatment. As the paper reports, “Analyses of parasite infrapopulations collected from people pre‑ and post‑praziquantel treatment further identified instances of treatment failure, supporting the potential for praziquantel resistance.”

For global health programs that rely entirely on praziquantel, these findings represent an early but important signal.

Stephen Doyle, PhD, co‑senior author and group leader and UKRI Future Leaders Fellow at the Wellcome Sanger Institute, emphasized the shift this genomic insight enables: “Whole‑genome sequencing gives us an unprecedented window into how schistosome populations are structured and evolving across Africa, and by characterizing variation in the drug’s molecular target at scale, we can move from reactive surveillance to proactive monitoring.”

Professor Joanne Webster, DPhil, of the Royal Veterinary College and director of the Global Centre for Neglected Tropical Disease Research, underscored the stakes: “While praziquantel remains largely highly effective, our findings provide a sobering warning about the reliance on a single drug for schistosomiasis control and highlight the need for comprehensive surveillance to monitor the potential emergence of drug resistance.”

Schistosomiasis affects more than 250 million people worldwide, with 90% of infections occurring in sub‑Saharan Africa. With elimination targets set for 2030, the emergence of resistance could jeopardize decades of progress.

The authors argue that genomic surveillance should become a routine part of schistosomiasis control. Their dataset, the largest genomic analysis of S. mansoni from human infections to date, provides a baseline for tracking resistance‑linked variants as MDA programs continue.

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Flu Virus Interaction with Host Cell Machinery Mapped Inside Infected Cells

Edusehat — Tue, 21 Jul 2026 07:35:09 +0700

Researchers at EMBL Hamburg and collaborators at the Leibniz Research Institute for Molecular Pharmacology (FMP) have mapped in unprecedented detail how the influenza A virus (AIV) rewires infected human cells. The researchers developed a customized experimental workflow that used in-cell cross-linking mass spectrometry (XL-MS), combined with AlphaFold-based structural modeling and functional assays, to directly map protein-protein interactions (PPIs) in IAV-infected human cells.

They claim that the study marks the first time that scientists have mapped direct virus-host protein contacts at scale inside intact influenza-infected cells, with enough structural detail to model how the proteins fit together. “Our work provides a new way to study flu-host interactions in their native context and with structural insight,” said Jan Kosinski, PhD, group leader at EMBL Hamburg and the Centre for Structural Systems Biology (CSSB). “The current results are a snapshot of a moment during infection, and it opens the door to studying flu-host interactions across the entire infection cycle.”

Kosinski is co-senior and co-corresponding author of the team’s published paper in Nature Microbiology, titled “Mapping in-cell protein contact sites reveals hijacking of paraspeckles during influenza A virus infection,” stating that their findings “… uncover mechanisms by which IAV exploits and remodels host compartments during infection.”

Every year, seasonal influenza kills up to 650,000 people globally and causes serious illness for 3–5 million individuals. When IAV infects cells, it releases RNA that contains the blueprints for a handful of proteins that spread throughout the host cell and repurpose its molecular machinery to make more viruses. “Its replication relies on protein–protein interactions (PPIs) between up to 14 viral proteins and host factors, often confined to cellular compartments and organelles,” the team stated.

Scientists want to understand this process in detail, as it would help in designing better drug therapies and vaccines against the flu virus. “Understanding these host–IAV PPIs in context is essential for elucidating viral strategies and therapeutic targets,” they added.

Studying protein-protein interactions in action during infection is challenging. Most previous studies relied on biochemical methods that required the cell to be broken open before the interactions could be measured. Once the cell’s compartments were gone, proteins that were never in contact inside the cell could meet in the test tube, and fragile or location-specific contacts could be lost. It was then hard to know which interactions actually happened inside an infected cell.

“This is when we learned that our collaborators—Boris Bogdanow and Fan Liu—at FMP Berlin had developed a specialized version of cross-linking mass spectrometry (XL-MS), a long-established technique for mapping protein contacts, tailored specifically to virus-infected cells,” said Kosinski. This was the critical breakthrough. It allowed researchers to do what previous methods couldn’t, including capturing short-lived and location-specific interactions.

“XL-MS allows us to capture protein-protein interactions directly in infected intact cells, while also providing structural information about how these interactions are happening,” explained Bogdanow, who is now a junior research group leader at the Institute of Virology, Charité—Universitätsmedizin Berlin. “This gives us insight into the interface between the virus and the human cell and may, through structural modelling, help identify actionable targets for future pharmaceutical interventions.”

By combining the results obtained through XL-MS with computational structural modeling, the researchers could identify which viral and human proteins interact and also predict how they physically fit together. For this, they used a modified version of the protein structure prediction algorithm AlphaFold.

“The key advantage of the modified AlphaFold approach is that it allowed us to feed our experimental cross-linking data directly into the structural modeling,” explained Kosinski. “This tells the model which parts of the viral and host proteins are close to each other inside infected cells. This was especially useful for virus-host complexes, which are often difficult to predict reliably.”

The study findings revealed two important ways in which the virus hijacks the cell. One involves hemagglutinin, a protein on the virus’s surface that it uses to bind and enter host cells. Tracing how hemagglutinin moves through the cell’s internal transport and processing system revealed how host proteins, some with previously unknown functions, helped the virus correctly fold and modify hemagglutinin during infection.

The other involves paraspeckles, small droplet-like compartments in the nucleus. The researchers found that infection by the influenza A virus causes these organelles to dissolve, releasing the RNA-binding proteins bound within them, which the virus can then use to replicate. “We identified host factors linked to the maturation of distinct glycoforms of the viral surface glycoprotein haemagglutinin through the membrane-bound endoplasmic reticulum–Golgi system,” the scientists wrote in summary. “In the nucleus, we observed the progressive disassembly of paraspeckles (phase-separated membraneless compartments) across multiple cell lines.”

First author Iuliia Kotova, PhD, former predoctoral fellow at the Kosinski group at EMBL Hamburg, and currently at ETH, said, “What surprised us most was the paraspeckles. Watching these tiny organelles in the nucleus dissolve, consistently across every cell line and every flu strain we tested, told us this isn’t a side effect of infection—it might be a strategy.”

Kosinski added, “There may also be a second benefit for the virus: some evidence suggests paraspeckles contribute to cellular stress responses and antiviral gene regulation, so disrupting them could also weaken parts of the cell’s defense response.”

The researchers believe that their “mapping in context” approach can be used to understand the mechanism of action of other viruses that act similarly. “While the exact host factors and mechanisms often differ from virus to virus, we think our overall approach—combining in-cell cross-linking, structural modeling, and targeted cell-biology follow-up to map native virus-host interactions at specific stages of infection—remains broadly applicable,” Kosinski said.

Bogdanow further commented, “Although this study has focused on a lab-adapted strain, this study lays the groundwork to apply the methodology to viruses of potential pandemic relevance, such as H5N1, and for uncovering the interaction networks that support their multiplication in human cells.”

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Memory Shaped by Brain Remodeling During Adolescence in Mice

Edusehat — Tue, 21 Jul 2026 04:00:11 +0700

The human brain continues developing beyond the teenage years, with crucial changes involving decision-making and emotional regulation extending into the mid-to-late 20s. Researchers at Albert Einstein College of Medicine have identified a biological process in mice that offers new insight into how memory circuits mature during this period of brain development.

The study published in PLOS Biology titled, “Retrosplenial cortical reorganization during late adolescence introduces instability of contextual memory circuits,” found that a key memory region of the mouse brain undergoes a period of remodeling during late adolescence, causing memories formed earlier in life to become temporarily more difficult to retrieve before resurfacing with less precise detail. The findings identify a biological mechanism that may explain how access to memories changes during development.

The study focused on the retrosplenial cortex (RSP) and discovered that protective mesh-like structures, called perineuronal nets, stabilize memory circuits and unexpectedly diminish during late adolescence before rebuilding in adulthood. The changes were confined to the RSP and were not observed in the nearby hippocampus, another brain region essential for memory.

“We’ve known for years that the brain continues developing through adolescence and young adulthood,” said senior author Jelena Radulovic, MD, PhD, professor of neuroscience, psychiatry, and behavioral sciences at Einstein. “Our findings begin to explain what that developmental process looks like in one of the brain’s memory circuits and how it can influence the way earlier experiences are recalled.

Previous studies suggested that the memory circuits reached maturity during early adolescence. Instead, results showed that an important stabilizing system temporarily weakened during late adolescence before recovering in adulthood.

The timing is notable because it corresponds to a period now recognized as one of continued brain maturation in humans. According to the National Institutes of Health, the brain continues developing and maturing into the mid-to-late 20s.

“The behavior matched the biology,” said lead author Hui Zhang, PhD, a research fellow at Einstein. “The retrosplenial cortex is responsible for older, more established memories. As its stabilizing structures declined, access to memories formed earlier in life became less reliable.”

To determine how these brain changes affected behavior, the researchers trained mice to associate a specific environment with a mild foot shock. The mice remembered the experience and froze when returned to the same chamber. Weeks later, many of the mice trained during early adolescence no longer showed that fear response, while mice trained during adulthood retained stable memories over the same period.

When the adolescent mice later experienced another test in a different environment, they once again responded to the original setting, demonstrating that the memories had become temporarily inaccessible rather than erased.

The researchers traced these changes to a decline in key structural proteins that help build and maintain perineuronal nets, along with reduced activity of growth factor, TGFβ2. When TGFβ2 activity was restored, the mice regained their ability to retrieve memories formed earlier in life.

By mid-adulthood, many of those memories resurfaced spontaneously, although they had become less precise. Rather than responding only to the original environment, the mice generalized their fear to unfamiliar settings. The researchers note that this pattern resembles the “reminiscence bump,” a well-known phenomenon in which adults disproportionately recall memories from adolescence and early adulthood while often remembering the emotional significance of an experience more readily than its specific details.

The findings may also have implications beyond memory. Schizophrenia and major depression often emerge in humans during late adolescence. The authors suggest that changes in this developmental process could contribute to vulnerability to psychiatric disorders in genetically susceptible individuals. Additional research is needed to evaluate whether similar mechanisms occur in humans.

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Patient-Derived Tumor Organoids Show Promise for Personalizing Cancer Treatment

Edusehat — Tue, 21 Jul 2026 04:00:10 +0700

The development of patient-derived tumor organoids for preclinical research will undoubtedly advance precision medicine research. Now, researchers have developed a pan-cancer patient-derived organoid (PDO) platform comprising 220 PDOs from 191 patients across 15 cancer types. This advance points to an increase in the use of tumor organoids as models for evaluating and optimizing cancer treatments.

This work is published in Science Advances in the paper, “Patient-derived organoids across cancers reveal conserved tumor heterogeneity and actionable therapeutic vulnerabilities.”

The team characterized the organoids extensively, showing that they retained key characteristics of the parent tumors over extended periods and showed promise in screens to identify unrecognized treatment candidates. The organoids had similarity to their parent tumors in terms of microscopic appearance, driver DNA mutations, gene expression patterns and other features.

More specifically, the comprehensive characterization demonstrated “high fidelity to parent tumors, with 93% histopathology concordance, 80% median genomic concordance for driver mutations, and a 0.85 median gene expression correlation.” Expression profiles remained largely stable over 10 passages, ensuring reproducibility for long-term screening. And clonality analysis, the authors note, showed that 85% of dominant tumor clones were preserved, with genomic concordance directly reflecting clonal similarity.

“Essentially, these organoids appear to be very good preclinical models of the parent tumor, and are practical models because they can be used long-term,” said Andrea Sboner, PhD, associate professor of pathology and laboratory medicine, director of informatics and computational biology in the Englander Institute for Precision Medicine and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell.

The team selected a subset representing patient tumors that, based on standard clinical criteria, had been deemed ineligible for treatment with a new class of drugs—PARP inhibitors. They then tested a PARP inhibitor, talazoparib, on the organoids, and found that more than half—58%—showed substantial sensitivity, implying that the current clinical criteria are excluding patients who could benefit from such drugs. The team characterized the mutational and other features that made these organoids susceptible—offering clues to how the clinical criteria might be expanded—and identified drugs that synergistically enhance talazoparib’s effects.

“You can use these organoids as patient ‘avatars’ during clinical trials of experimental therapies, for example, to get an early picture of treatment effects and side effects,” notes Juan Miguel Mosquera, MD, a professor of pathology and laboratory medicine and director of research pathology at the Englander Institute.

The scientists also envision the future use of tumor organoid technology in selecting treatments for individual patients—growing an organoid from a sample of the patient’s tumor and then testing it rapidly with different treatment regimens.

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Protein Protects Against Tau Tangles, Synaptic Loss in Mouse Model of Tauopathy

Edusehat — Tue, 21 Jul 2026 04:00:09 +0700

Alzheimer’s disease (AD) and many other forms of neurodegeneration share a common culprit. In these diseases, tau proteins that normally stabilize neuronal microtubule filaments within nervous system networks instead form noxious knots and gradually disrupt the circuits they would otherwise preserve.

Scientists at Sanford Burnham Prebys have now shown that a different protein known as SORLA offers protection against the effects of these lethal loops. The results of the researcher’s’ study in mice suggests that future research may yield new treatments capable of boosting this protein’s ability to defend the brain.

Timothy Huang, PhD, assistant professor in the Center for Neurologic Diseases at Sanford Burnham Prebys, is senior and corresponding author of the team’s published paper in Science Advances, titled “SORLA up-regulation suppresses pathological effects in aged tauopathy mouse brain,” in which they concluded “These findings reveal a protective role for SORLA in multiple aspects of tauopathy pathogenesis and highlight its potential as a therapeutic target.”

Normally, tau proteins are found throughout the brain and nervous system, helping to maintain the shape and structure of our neuronal wiring. But in certain diseases, including Alzheimer’s disease, tau proteins clump together inside nerve cells, forming what are known as tau tangles. These toxic tangles are linked to cognitive impairment and nerve cell death in diseases known as tauopathies. “In AD, amyloid-β (Aβ) plaques and neurofibrillary tangles (NFTs) comprising hyperphosphorylated tau accumulate in brain,” the authors explained.

The new study focused on the safeguarding capabilities of protein known as SORLA. “A role for the trafficking receptor SORLA (Sortilin-related receptor containing LDLR class A repeats) in reducing Aβ levels has been well established,” the investigators continued. “… however, relatively little is known with respect to whether and how SORLA can potentially affect tau pathology in vivo.”

Timothy Huang added, “In the last 15 or 20 years, considerable data has come out from our lab and other groups showing that SORLA can suppress one of the hallmarks of Alzheimer’s disease—amyloid-beta generation and accumulation. Very little was known, however, about whether SORLA affected the tau tangles reflected on the other side of the coin in Alzheimer’s disease.”

SORLA is expressed in both neurons and glia in mouse and human brain, the authors noted. For their newly reported study the team began by crossbreeding mice that produce extra human SORLA protein, with PS19 (P301S) mice that develop tau tangles, brain atrophy and cognitive deficits. This new mouse model enabled experiments to determine SORLA’s effects on tau protein buildup and its resulting harms.

Their studies showed that an overabundance of SORLA protein protected against a number of biological processes linked to the formation of tau tangles and progression of neurodegeneration. These include reducing the addition of too many phosphate groups to tau—known as hyperphosphorylation—and the ability of misshapen tau to serve as “seeds” that attract more tau and form clumps. This protection also extended to preservation of the synapses at the junction between neurons and the brain’s ability to adjust these connection points—which is called synaptic plasticity. “Using complementary approaches, we show that SORLA overexpression attenuates ventricular enlargement, tau phosphorylation and seeding, synaptic loss, impaired synaptic plasticity, and glial hyperactivation in the PS19 mouse brains,” the team wrote in summary.

An overabundance of SORLA protein protects against a number of biological processes linked to the formation of tau tangles and progression of neurodegeneration. These include reducing the addition of too many phosphate groups to tau, known as hyperphosphorylation. In these biopsy images, less phosphorylated tau—stained to appear green—has accumulated in the bottom sample overexpressing SORLA. [Tim Huang, Huijie Huang, Sanford Burnham Prebys]

“When you upregulate SORLA, you can suppress the negative effects found in tauopathies,” said first author Huijie Huang, PhD, a staff scientist in the Huang lab at Sanford Burnham Prebys. “We found there was less brain atrophy and less tau accumulation, which was very exciting to see.”

Because some people have mutations that disable the gene carrying the code for SORLA, Sorl1, the scientists wanted to compare the outcome of having extra SORLA to having none of it at all. Tests of mice genetically modified to lack Sorl1 told a very different story. “The opposite turned out to be true when we deleted the ability to produce SORLA proteins,” said Timothy Huang. “A lack of SORLA exacerbated the harmful effects observed in tauopathies.”

To address how extra SORLA or a lack of SORLA were either ameliorating or aggravating diseases featuring tau tangles, the research team used a combination of sequencing techniques capturing the levels of all proteins and gene expression in each cell, as well as mapping the spatial relationship of RNA and proteins within brain tissue. The scientists found that upregulated SORLA prevented problematic protein production changes in the synapses between neurons while also suppressing other drivers of tauopathy disease progression. They also observed that extra SORLA tamped down on disease-related gene expression patterns in brain cells known as glial cells that support and protect neurons in many ways. “One particularly notable finding that we can build on is the upregulation of a member of the plexin-B family of receptors in the absence of SORLA,” said Huijie Huang.

“There are unique drugs that can target this class of receptors that we may be able to apply to tau-related dementia disorders,” suggested Tim Huang. “One potential future direction is to repurpose these drugs to target overactivation of glial cells and perhaps reverse some of the phenotypes in tauopathies.”

The scientists also want to better understand what happens in each individual cell type when they upregulate or downregulate SORLA. “While it is not possible to specifically determine how cell-specific modulation of SORLA can affect tau using the global transgenic overexpression/deletion models used here, we are interested in further characterizing specific effects of SORLA on tau in neurons, and the extent of SORLA modulation on glia in influencing overall tau pathology,” they stated. The team plans to graft human neurons or glial cells into the mouse brain to study the effects of different SORLA mutations.

“Mouse cells and human cells are different,” said Tim Huang. “Because we’re looking at human disease, it’s more informative if we can observe the modulation and dysfunction of SORLA in the context of a human cell inside of a diseased brain environment.”

This continued research will reveal more knowledge about the ability of SORLA to safeguard against the toxic effects of tau tangles, and how to develop new treatments or repurpose existing therapies to benefit patients suffering from Alzheimer’s disease and other tau-related dementia disorders.

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New €25 Million BioReliance Testing Facility Opens at Merck KGaA Global Headquarters in Germany

Edusehat — Mon, 20 Jul 2026 20:50:10 +0700

MilliporeSigma opened a new €25 million BioReliance^® testing facility at the company’s global headquarters in Darmstadt, Germany. The facility expands access to commercial drug substance and drug product release testing as well as stability testing for biopharmaceutical companies developing and commercializing therapies in Europe, according to the company.

“As demand for biologics and novel therapies continues to grow, our customers need reliable, compliant testing capabilities closer to where their products are developed and commercialized,” said Paolo Carli, head of advanced solutions for the life science business of Merck KGaA. “Our new testing facility combines best-in-class analytical characterization services with more than 75 years of BioReliance expertise to help our European customers move critical therapies toward patients with greater speed and confidence.”

The 2,000-square-meter facility is designed to help customers meet European requirements for in-region drug substance and drug product release testing and to expand the company’s ability to support customers from drug development through commercialization. The site will also offer GMP-compliant stability studies for monoclonal antibodies and cell therapies, addressing the growing demand for biologics testing across Europe.

Located close to major clinical trial sites in Germany, France, Spain, the Netherlands, Belgium and Italy, the Darmstadt facility is strategically positioned to support biopharmaceutical companies seeking to release drug products into European markets, pointed out Carli. By adding these capabilities in the heart of Europe, the company is strengthening its support for customers managing increasingly complex development, quality and regulatory requirements, he added.

A MilliporeSigma spokesperson noted that the BioReliance sites form a global testing network that allows customers to scale across geographies and work with the company across continents. Among the company’s leading technologies is the Blazar^® platform, which moves the biosafety testing paradigm from traditional methods to rapid molecular approaches to significantly reducing testing timelines for virus detection.

The Aptegra^® CHO genetic stability testing streamlines a previously complex and time-intensive process into a single assay, continued the spokesperson.

MilliporeSigma lists the opening of the Darmstadt facility as one of several significant investments the company has made to grow its global contract testing footprint. In 2024, the company opened a €290 million biosafety testing facility in Rockville, MD, and expanded biosafety testing capacity by 40% across its Glasgow and Stirling sites through a €22 million investment. The company also cites the new BioReliance facility as reflecting the firm’s continued commitment to its global headquarters in Darmstadt, where €2.5 billion has been invested since 2015.

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StockWatch: Lilly’s Up-to-$3.8B Deal for AtaiBeckley a Good Trip for Psychedelic Drugs, Analysts and Investors Agree

Edusehat — Mon, 20 Jul 2026 02:55:08 +0700

It wasn’t too long ago that biopharma giants stayed away from developing psychedelic drugs—but positive clinical data plus a friendlier regulatory climate in Washington have prompted the largest drug developers to embrace the field.

The latest and most telling example of pharma embracing psych drugs came when Eli Lilly (NYSE: LLY) announced that it agreed to acquire AtaiBeckley (Nasdaq: ATAI) for up to $3.8 billion—of which Lilly will pay $2.8 billion upfront. The deal, set to close in the third quarter, expands Lilly’s neuroscience portfolio by adding the pipeline of AtaiBeckley led by BPL-003 (mebufotenin benzoate), a Phase III candidate for treatment-resistant depression (TRD) that is a synthetic form of 5-MeO-DMT administered intranasally. BPL-003 has been granted the FDA’s Breakthrough Therapy designation.

BPL-003 wowed analysts and others back in April after AtaiBeckley published positive data from a Phase IIa trial (NCT05660642) showing that a single intranasal dose of BPL-003 led to rapid and sustained reductions in Montgomery-Åsberg Depression Rating Scale (MADRS) scores from baseline in 12 TRD patients who remained on stable SSRI therapy throughout the study. Both the six patients dosed at 10 mg and six at 12 mg showed a 66.7% antidepressant response rate (defined as ≥50% reduction from baseline MADRS score) at Day 2, with five of six participants in the 10 mg cohort (83%) and four of six in the 12 mg cohort (66.7%) maintaining their response at Week 12.

“Especially with progress on BPL-003, we see the company as positioning itself well to becoming a significant player in the mental health therapeutics space,” Sumant Kulkarni, a senior analyst covering biotechnology with Canaccord Genuity, wrote on news of the positive data, adding: “We also still see this space as large enough to accommodate multiple approaches/competitors.”

$3.7B in projected peak sales

Kulkarni also raised Canaccord Genuity’s peak unadjusted U.S. sales forecast for BPL-003 to $3.7 billion by 2036 from $2 billion, after revising the firm’s model by raising the list price from $20,000 to $30,000 per annual treatment course (not accounting for insurance coverage), about the same price as Spravato® (esketamine), also a nasal spray marketed by Johnson & Johnson (NYSE: JNJ) for TRD plus some depressive symptoms in adults with major depressive disorder (MDD).

Spravato, a noncompetitive N-methyl D-aspartate (NMDA) receptor antagonist, crossed the $1 billion sales threshold during the second quarter, as it generated $584 million, up 25% quarter-over-quarter from $464 million in Q1—and up 43% from $734 million in the first half of 2025.

“Sales are tracking to reach annual sales guidance of $3-3.5B+ by 2027–28,” Jefferies equity analyst Andrew Tsai wrote in a research note focused on J&J’s second-quarter results. “Spravato’s trajectory supports the notion psychedelics can be commercially viable in hard-to-treat mental health disorders, by leveraging JNJ’s infrastructure.”

Given the data for BPL-003, Lilly got a bargain, Tsai wrote in a separate note on the Lilly-AtaiBeckley acquisition.

“We think the deal heavily favors LLY, as ATAI’s lead asset BPL-003 (intranasal 5-MeO-DMT) should have multibillion dollar peak sales potential,” Tsai wrote, rather than the $1 billion-plus that he thinks was implied by the deal price.

Tsai and Jefferies had previously forecast peak sales of between $1 billion and $2 billion—a range he said was “arguably conservative” since BPL-003 could, if it aces its Phase III trial, show superiority to Spravato, which is on track to reach up to $5 billion-plus in peak sales.

Positive implications

“At the same time, we appreciate LLY has significantly more resources to maximize the long-term value of ATAI’s psychedelic assets. In any case, the deal has (+) [positive] implications for the entire psychedelic space,” Tsai added.

Among pharma giants joining J&J in embracing psychedelic drug development in recent years:

AbbVie (NYSE: ABBV), which last year acquired the lead pipeline program of privately held Gilgamesh Pharmaceuticals, the moderate-to-severe MDD candidate bretisilocin (GM-2505), for up to $1.2 billion.
Otsuka Holdings (Tokyo Stock Exchange: 4578), which in 2023 acquired Mindset Pharma, a Canadian psych drug developer focused on psychiatric and neurological disorders, for C$80 million ($56 million).

With its deal for AtaiBeckley, Lilly becomes the latest pharma giant to perceive the positive implications Tsai cited.

“Treatment-resistant depression persists even after multiple treatments have failed. Millions of people are still searching for relief and desperately need a therapy that works,” Carole Ho, executive vice president and president, Lilly Neuroscience, said in a statement. “Advancing AtaiBeckley’s investigational therapies gives us a real chance to change that.”

Investors agreed with Lilly, giving the pharma a 1% increase Thursday, the day the acquisition was announced, from $1,156.63 to $1,169.17—no small feat since buyers typically stay flat or see their shares slide after announcing an acquisition. And not surprisingly, AtaiBeckley investors were enthusiastic about the deal, as its stock leaped 33% from $5.36 to $7.15. On Friday, Lilly inched up 0.8% to $1,178.58 while AtaiBeckley rose 1% to $7.22.

The AtaiBeckley buyout is Lilly’s eighth announced acquisition of a smaller biopharma this year.

Lilly is acquiring three infectious diseases vaccine developers—Vaccine Company for up to $1.55 billion, Curevo for up to $1.5 billion, and LimmaTech Biologics for up to $780 million—as well as in vivo chimeric antigen receptor T-cell (CAR T) developer Kelonia Therapeutics for up to $7 billion); JAK inhibitor developer Ajax Therapeutics for up to $2.3 billion; next-generation dual-payload antibody-drug conjugate (ADC) developer CrossBridge Bio for up to $300 million; and nonviral DNA delivery-focused drug developer Engage Biologics for up to $202 million cash.

The deal spree reflects Lilly’s desire to capitalize on the billions of dollars it is generating from sales of its obesity and diabetes drugs based on glucagon-like peptide 1 (GLP-1) receptor agonists alone or in tandem with a glucose-dependent insulinotropic polypeptide (GIP).

“If we see great ideas that we think we can use to help people that need them, of course we’ll do deals,” Daniel M. Skovronsky, MD, PhD, Lilly’s chief scientific and product officer and president of Lilly Research Laboratories, said on CNBC.

“Positive development”

David Risinger, a senior managing director and senior research analyst covering diversified biopharmaceuticals at Leerink Partners, said his firm viewed Lilly’s buyout of AtaiBeckley “as a positive development because it enhances LLY’s pipeline of potential neuroscience blockbuster candidates.”

That pipeline is led by five Phase III programs involving four drugs, none of them a psychedelic. Two of the programs belong to brenipatide, a dual agonist of both the GIP and GLP-1 receptors. Brenipatide is being developed for both MDD and alcohol use disorder.

Also in Lilly’s late-stage neuroscience pipeline are:

Donanemab, which binds to deposited amyloid plaque in the brain and is being studied for the treatment of cognitively unimpaired Alzheimer’s disease.
Ixoberogene Soroparvovec (Ixo-Vec), an intravitreal gene therapy being studied as a single one-time treatment for vision loss associated with neovascular (wet) age-related macular degeneration (AMD).
Remternetug (LY3372993), which also binds to deposited amyloid plaque in the brain and is under study as a treatment of cognitively unimpaired/mild cognitive impairment due to Alzheimer’s disease, with potential for subcutaneous delivery.

In addition, AtaiBeckley “would provide differentiated exposure in psychiatry and reinforce [Lilly’s] broader effort to diversify beyond its cornerstone cardiometabolic franchise,” observed Evan David Seigerman, a managing director and head of healthcare research at BMO Capital Markets, as reported by Reuters.

AtaiBeckley was formed last November by the merger of atai Life Sciences and Beckley Psytech. The company’s stock has nearly doubled, soaring 98% over the past six months from $3.64 on January 16.

“Going mainstream”

“Psychedelic Medicine is going mainstream,” declared Steve Jurvetson, co-founder of Future Ventures, in a post on X. Jurvetson and Future were among early investors, along with Peter Thiel in atai Life Sciences.

AtaiBeckley is one of numerous psychedelic drug developers to show significant six-month gains since January: As of Friday’s closing bell, Compass Pathways (Nasdaq: CMPS) shares jumped 68% to $12.35, GH Research ballooned 69% to $28.71, while Definium Therapeutics (Nasdaq: DFTX) nearly tripled, zooming 194% to $44.29.

Interestingly, those three companies did not get a solid bounce from AtaiBeckley’s acquisition by Lilly. Since the deal was announced Thursday, Compass fell 7% from $13.31 pre-announcement, Definium dipped 3% from $45.66. GH rose 8% Thursday from $26.92 to $29.13, before sliding 1.4% the following day.

Bucking the trend was Cybin, d/b/a Helus Pharma (Nasdaq: HELP), which has climbed 11% since the Lilly-AtaiBeckley announcement, from $6.51 to $7.25. Its shares have slumped 6% since January—but soared 58% over the past month on positive news, such as the 88%+ enrollment rate of patients in Helus’ Phase III APPROACH pivotal trial (NCT06564818) of HLP003 in MDD, on track for topline data readout in Q4 2026.

“We see the potential for 150–200% upside [jump in stock price] if Phase III data in 4Q26 are positive,” Kulkarni wrote, making it the largest potential jump among psychedelic drug developers.

In addition to favorable data, the stock surges also reflect actions by President Donald J. Trump’s administration to encourage psychedelic drug development. In April, President Trump signed Executive Order 14401, directing the FDA and other federal agencies to accelerate research and improve access to psychedelic drugs, citing their potential as promising treatments for serious mental illnesses.

And on July 13, the FDA published “Psychedelic Drugs: Considerations for Clinical Investigations,” a final guidance designed to provide general considerations for developers of psych drugs, with recommendations for how to conduct clinical trials for the treatments.

“Rather than providing specific recommendations on study design, this guidance will present foundational constructs that all sponsors studying the therapeutic potential of psychedelic drugs, including sponsors without commercial drug development as primary interest (e.g., academic researchers), should consider,” the FDA wrote in the final guidance. “Sponsors are encouraged to request meetings with FDA for advice on a specific drug development program.”

Leaders and laggards

Q32 Bio (Nasdaq: QTTB) shares nearly doubled, leaping 91% from $11.21 to $21.38 July 13 after the autoimmune and inflammatory disease drug developer announced positive 36-week topline results from Part B of the Phase IIa SIGNAL-AA trial (NCT06018428) assessing bempikibart in patients with severe or very severe alopecia areata. Q32 said it saw clinically meaningful efficacy data on the primary endpoint of mean percent change from baseline in SALT score, with a reduction from baseline of 35.3% in the prespecified modified intent to treat (mITT) analysis. The company also reported that 40.0% of patients (10/25) achieved SALT-20 response at Week 36 in the mITT analysis, while 30.3% of patients (10/33) achieved SALT-20 response at Week 36 in the ITT analysis of all enrolled patients.
Veradermics (NYSE: MANE) shares yo-yoed this past week, climbing 12% from $110.17 to $123.70 Wednesday after the pattern hair loss drug developer announced positive topline results from its open-label Phase II Study 207 trial (NCT06527365) assessing VDPHL01, an extended-release oral minoxidil formulation, in women with mild-to-moderate pattern hair loss. Veradermics said most study participants reported improved hair coverage at Month 2, with approximately 88.9% of patients dosed once daily and 90.0% dosed twice daily reporting “improved” or “much improved” outcomes at Month 6. Participants dosed once daily showed a mean increase in non-vellus target area hair count (TAHC) of 22.7 hairs/cm² at Month 6, an average that rose to 23.3 hairs/cm² in twice daily dosed patients. The mini surge was short-lived, however, as investors more than gave back the gain, selling off shares to send them tumbling 14% to $105.83 Thursday amid possible investor questions about whether the good clinical news was already reflected in the stock price.

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Organ Aging Linked to Breakdown in Immune Cell Interaction and Senescent Neutrophil Clearance

Edusehat — Sun, 19 Jul 2026 01:45:07 +0700

We may age at different rates, but none of us escapes aging. A study in mice and in human cells by Stanford Medicine researchers has linked organ aging to the increased inability—with advancing age—of tissue resident macrophage (TRM) immune cells to clear aged neutrophils, another type of immune cell.

The study found that these TRMs appear to be central coordinators of age-related organ decline. Blocking a single receptor, EP2, on these cells preserved the youthfulness of multiple organs in mice, including the brain, heart, skeletal and heart muscle, liver, spleen, bone marrow, kidney, and colon. The receptor binds specifically to a hormone, prostaglandin E2, which is known to cause inflammation and pain in humans as well as in mice.

The researchers found that in mice, selectively disabling this receptor exclusively on tissue-resident macrophages genetically, or using an experimental selective EP2 antagonist drug, prevented chronic-inflammation-driven disorders of age—including frailty, excessive fat accumulation, and heart trouble—and also substantially slowed cognitive decline.

Research lead Katrin Andreasson, MD, the Edward F. and Irene Thiel Pimley Professor in Neurology and Neurological Sciences, said, “We’ve shown that when tissue-resident macrophages don’t have EP2 on their surfaces anymore or when that receptor is plugged up by a drug, this decline doesn’t happen … We’ve been trying to figure out why we age. Now we know at least one big reason for it.”

The discoveries help to clarify systemic inflammation’s significant contribution to aging and the debilities that accompany it. The findings also point to a pharmaceutical approach that could restrain our organs’ unavoidable march toward senescence and so extend overall health span.

Senior author Andreasson, together with first author Jessy Tan, PhD, an instructor in neurology, and colleagues reported on their findings in Science, in a paper titled “Restored clearance of senescent neutrophils by tissue-resident macrophages limits organ aging.” In their research article summary, the team stated, “This work identifies EP2 signaling in TRMs as a central regulator of organ-wide aging through its control of senescent neutrophil clearance, reframing aging as a failure of active cellular clearance rather than passive degeneration.”

Aging is accompanied by parallel functional decline across organs, but the cellular drivers remain unclear, the authors wrote. “Although molecular hallmarks of aging have been identified, the cellular events that initiate and propel tissue decline remain poorly defined.”

The most abundant white blood cells in our immune system are neutrophils, which act as the body’s main first responders. Produced in bone marrow, new neutrophils are transferred to the bloodstream, where they circulate and attack bacterial, viral, or fungal pathogens that they encounter. Neutrophils are also extremely short-lived, surviving just 12–24 hours.

Some 90% of circulating neutrophils end up in the liver, spleen, and bone marrow, awaiting execution clearance by another type of immune cell. “Neutrophils are among the shortest-lived immune cells, aging within hours in the circulation and requiring continuous clearance,” the team noted.

This neutrophil clearance is critical. In aged animals, the vast bulk of neutrophils that never see combat undergo a fast transition to senescence, a zombie-like state in which they may injure, age, and inflame neighboring cells. And as we age, the neutrophil count rises, with senescent neutrophils constituting an ever higher percentage. “Senescent neutrophils are killing our tissues,” Andreasson said. “Clearance of these cells is essential for preventing chronic inflammation.”

That’s a job for macrophages. These cells comb the tissues for pathogens, signal other cells to lend a hand in the fight, and pump out growth factors that help repair damaged tissue. But first and foremost, Andreasson said, “They’re the body’s garbage collection crew. A lot of that garbage is defunct cells.” And a lot of those cells are neutrophils—to the tune of 100 billion a day.

Macrophages come in several subtypes. Tissue-resident macrophages are long-lived and ubiquitous. They take up residence in each of the body’s organs during fetal development and remain for their lifetimes in whatever organ they’ve inhabited, adapting their roles to fit that organ.

One of tissue-resident macrophages’ prime responsibilities is to swallow senescent cells. “A core TRM function is efferocytosis, the clearance of apoptotic, senescent, and damaged cells that is essential for preventing chronic inflammation,” they explained. Especially important targets for this operation, the study showed, are the potentially 100 billion neutrophils produced daily, which start showing signs of senescence within 8 to 12 hours after entering the bloodstream. (Neutrophils that haven’t arrived at senescence yet but have lived long enough and seen enough to put out “kill me now” flags of surrender on their cell surfaces are fair game.)

“Among primary TRM targets are neutrophils, the most abundantly produced immune cell, with more than 10 billion and 100 billion generated daily in mice and humans, respectively,” the investigator noted. “Uncleared aged neutrophils release proteases and extracellular traps that damage tissues, propagate inflammation, and promote aging, and are normally removed efficiently by TRMs in the liver, spleen, and bone marrow.”

But tissue-resident macrophages also grow old. As Andreasson and associates showed in a prior study, over the advancing years these long-lived cells become ever more prone to succumb to aging-associated inflammation and to propagate it. In their newly reported paper, they noted, “TRMs comprise 60–90% of macrophages in the brain, liver, lungs, heart, and kidneys, and their long lifespan makes them particularly vulnerable to aging, as they accumulate metabolic, oxidative, and inflammatory injury over years to decades.”

Immune cells produce hormones called prostaglandins. One of the five varieties of prostaglandin, called PGE2, can exert diverse effects on a cell, depending on which type of surface receptor is expressed on that cell’s surface. Of the various subtypes of receptors for PGE2, the EP2 receptor is highly pro-inflammatory. Tissue-resident macrophages are loaded with EP2.

Infection, injury, and toxic chemicals, including those produced by our aging bodies, increase PGE2 output. As the team’s prior work showed, that output grows substantially as we grow older. So does the concentration of EP2 on tissue-resident macrophages. “TRMs express the prostaglandin E2 (PGE2) receptor EP2, which suppresses macrophage metabolism and phagocytosis in aging,” the investigators noted.

This effectively creates a one-two punch. PGE2’s pro-inflammatory influence increases with age. The resulting unrelenting inflammatory PGE2 stimulation on tissue-resident macrophages, the new study showed, downshifts these cells’ ability to clear neutrophils. Senescent neutrophils then accumulate in tissues and blood.

Andreasson and her colleagues had previously shown that with aging, tissue-resident macrophages undergo a slow decay in their energy metabolism. “Once that starts, there’s a steady decline in a macrophage’s performance,” she said.

For their newly reported study, Andreasson’s lab bioengineered a mouse in which, at a time of the scientists’ choosing, the EP2 gene gets deleted—but only in tissue-resident macrophages. The results of their experiments showed that disappearance of EP2 from these cells reinvigorated the neutrophil-clearance process that PGE2 undermines.

For their experiments, the Stanford Medicine researchers studied younger normal mice, aged 6–8 months, which corresponds to late adolescence or early adulthood in humans, and they also studied older normal mice, at 23 to 25 months of age, whose human counterparts would be in their 60s or 70s. They also looked at older mice whose EP2-encoding gene had been deleted at 4 to 6 months of age (equivalent to their “teenage” years).

The team’s analyses identified 71 proteins, found in blood, whose levels were significantly altered in older normal mice. Of those proteins, 59 stayed at youthful levels in older mice whose tissue-resident macrophages lacked EP2. Many of these proteins originated in the liver. “The liver is one of the body’s most tissue-resident-macrophage-enriched organs and a major contributor to aging-related changes in blood chemistry,” Andreasson said. “It’s the central organ determining the body’s metabolic rate.”

The study showed that in normal old mice, smoldering senescent neutrophils accumulated in the liver, spleen, and bone marrow and, to a lesser extent, in many other organs the researchers looked at.

But the organs of older mice lacking EP2 on their tissue-resident macrophages retained the lower neutrophil numbers of youth. These mice looked younger, leaner, and more physically fit compared with control littermates. They evidenced less visceral fat and greater muscle mass. Their performance on tests of multiple organs’ function equaled that of young mice.

EP2 deletion in addition reduced inflammation in the blood, liver, colon, heart, kidney, and hippocampus (a brain region tightly tied to memory and navigation ability) in the older mice. Their speed, balance, and forelimb grip strength resembled that of young animals.

Reducing EP2 activity in older mice also preserved their memory capabilities. These animals could thread their way through a maze or recall previously encountered objects almost as well as younger mice—and far better than similarly old mice with tissue-resident macrophages expressing functional EP2. “Reducing TRM EP2 signaling in aged mice preserved youthful mitochondrial fitness and prevented cognitive decline, frailty, sarcopenia, adiposity, cardiac impairment, and systemic inflammation,” they wrote in summary.

There are, today, no approved drugs that selectively shut down EP2 activity, although there are several that target PGE2. Non-steroidal anti-inflammatory painkillers work by blocking PGE2 production, Andreasson said. That’s how aspirin and similar drugs reduce pain, fever, swelling, and redness. But to greater or lesser degrees these drugs all block other vital prostaglandins. Even PGE2 has beneficial properties when it binds to receptors other than EP2, rather than the detrimental inflammatory one examined in this study.

As part of their study, the investigators treated otherwise normal 22-month-old mice for two months with an EP2-inhibiting experimental drug. The results showed that the treatment reduced total and senescent neutrophil counts in old mice toward youthful levels. In culture dishes, old age diminished—but the EP2-blocking drug likewise significantly restored—the mice’s tissue-resident macrophages’ ability to engulf and digest burnt-out neutrophils. “Together, these results demonstrate that pharmacologic EP2 inhibition partially reverses age-associated TRM dysfunction and senescent neutrophil accumulation, with strongest rescue in the liver,” they stated.

Finally, the team turned to a large human database characterizing different cell types in young, old, and diseased human livers. This database revealed the same age-related neutrophil buildup, increased neutrophil senescence, tissue-resident-macrophage decline, and heightened EP2 activity in older—and even more so, diseased—livers that the Stanford Medicine researchers had seen in mice. This was a first-time observation in human cells, according to Andreasson. “These human findings, while correlative, position the TRM EP2-efferocytosis axis as a candidate mechanism in human aging that warrants further functional testing,” they noted. “Specifically, future studies should assess whether the impaired clearance of senescent neutrophils also occurs in human TRMs and whether pharmacological EP2 blockade can restore this defect.”

Andreasson suggested that targeting neutrophil clearance may yield big therapeutic benefits. “We need to develop a safe drug that incapacitates EP2 without disrupting upstream events such as PGE2 production.”

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Genetic Study Links Excessive Sweating to Neurological Dysfunction

Edusehat — Sat, 18 Jul 2026 04:15:09 +0700

Data from a new study suggest that a form of hyperhidrosis, or excessive sweating, may be due to genetic mutations that result in the overstimulation of the nerves that control the sweat glands. These findings, which are reported in Science Advances, could open a door to targeted treatments for the condition using existing medicines. Full details of the findings are provided in the paper titled “A neurocutaneous NaV1.8 channelopathy underlies a genetic subtype of primary idiopathic hyperhidrosis.” The international study is led by scientists at Vrije Universiteit Brussel.

Excessive sweating, which affects roughly two to five percent of the population, causes more than just discomfort. The impact of the condition on the daily lives of people living with it can be very severe. Patients often sweat so profusely that they have to change clothes several times a day. Many avoid social contact, experience shame, and develop depression. Yet the condition is often seen as a superficial skin problem and patients often do not receive appropriate care.

That could change thanks to the findings from this study which is the culmination of 10 years of research done by scientists in the lab of Frank Bosmanbs, PhD, at Vrije Universiteit Brussel and their collaborators at Johns Hopkins University. To pinpoint a genetic basis for hyperhidrosis, the scientists analyzed the DNA of more than 180 patients. They discovered defects in the Nav1.8 ion channel, which normally functions as a biological gate that regulates electrical signals in the nervous system.

Specifically, in patients with hyperhidrosis, the gate is left too wide open due to a genetic predisposition. As a result of this, the nerves are constantly overstimulated and in a state of activity, which results in excessive sweating often triggered by emotional or stress-related stimuli. To dig deeper into their theory, the scientists developed an experimental mouse model. Because mice only sweat from their paws, the team developed a microscopic measurement method to count sweat droplets using an iodine-starch mixture.

They found that mice that had the same genetic defect as hyperhidrosis patients also sweated excessively. Furthermore, once the scientists administered a substance that blocked the overactive nerve signals, their symptoms decreased significantly and reversibly. However the genetic picture is more complex. Bosmanbs and his team found a patient who had inherited an inhibitory nerve mutation but still sweated excessively due to a separate mutation in a local water channel within the sweat gland. It suggests that there are different biological pathways that can lead to the same overstimulation that results in hyperhidrosis.

Though the genetic picture is a complex one, the scientists believe that their findings offer the prospect of better treatments for this condition. Currently, some severe forms of hyperhidrosis are treated by severing the sympathetic nerve pathways in the chest. While effective, this treatment is both invasive and can have unwanted side effects. With a deeper understanding of the genetic basis of the condition, scientists may be able to better predict which patients are likely to get the most benefit from localized treatment of the sweat glands, systemic medication or nerve-targeted therapies. Another potential treatment avenue is drug repurposing, which is supported by the evidence from the mouse studies. However, further testing via controlled clinical trials is required.

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KRAS-Targeted Vaccine Crosses First Clinical Milestone in Pancreatic Cancer Prevention

Edusehat — Sat, 18 Jul 2026 00:40:10 +0700

A vaccine to prevent pancreatic cancer, targeting common KRAS mutations, was found to be safe and stimulated KRAS-specific T-cell responses in 90% of study participants who were at high risk of developing pancreatic ductal adenocarcinoma (PDAC), according to Phase I clinical trial results. After a median follow-up of 16.5 months, none of the participants developed pancreatic cancer, and some of the precancerous lesions shrank or stopped growing. The study represents the first proof of concept for the use of vaccines for interception of pancreatic cancer in human patients.

The findings are published in Cancer Discovery, in the paper, “First-in-human Testing of a Mutant KRAS Vaccine for Pancreatic Cancer Interception in High-risk Cohorts.”

PDAC is an aggressive cancer that is often diagnosed at advanced stages and carries a low (five-year) survival rate. Approximately 10% of cases are associated with hereditary predisposition caused by pathogenic mutations in specific cancer susceptibility genes that are passed down from parent to child. The disease evolves over time from precursor lesions such as pancreatic intraepithelial neoplasia and intrapapillary mucinous neoplasms.

“Individuals at high risk due to hereditary predisposition or to the presence of a concerning pancreatic lesion detected on imaging usually undergo surveillance to monitor for changes over time,” said Neeha Zaidi, MD, associate professor of oncology at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University. “If there is a high enough concern for transformation to cancer or if early cancer is detected, the current standard of care is surgical resection. However, the chances of recurrence are up to 80%, and many precursor lesions to pancreatic cancer are microscopic and thus undetectable by imaging.”

The researchers wanted to test if intercepting cancer development through noninvasive approaches could provide an effective strategy to prevent PDAC and improve survival in high-risk individuals. “Prevention and interception save lives and reduce the morbidity associated with cancer development and progression. This is especially important for cancers whose early-onset frequency is increasing and for which we do not have effective methods for early detection,” said Elizabeth Jaffee, MD, FAACR, deputy director of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

Mutations in the KRAS gene are the main oncogenic drivers in more than 90% of PDACs. The researchers previously developed mKRAS-VAX, an off-the-shelf synthetic long peptide vaccine targeting the six most common KRAS mutations found in PDAC and in most pancreatic precancer lesions.

In the new study, the team conducted a Phase I clinical trial to assess vaccination with mKRAS-VAX in 20 individuals at high risk of PDAC due to hereditary predisposition and radiographic evidence of a pancreatic lesion, typically in the form of a small cyst.

“The goal of this study was to test the safety of the vaccine and induction of durable immune responses,” said Jaffee. She added that the trial was based on preclinical data demonstrating the ability of a KRAS-targeted vaccine to prevent progression of early precancers in a genetically engineered mouse model of KRAS-driven pancreatic cancer.

Study participants received mKRAS-VAX via subcutaneous injections according to a prime-boost vaccination strategy, with priming doses on weeks one, three, and five and a boost dose on week 13. Blood was collected at different time points, and optional annual follow-up visits were offered for long-term immune monitoring.

Results showed that the vaccine stimulated mutant-KRAS-specific effector and central memory T-cell responses in 90% of participants. These responses remained detectable in the blood for up to two years after vaccination. “This long-lasting response is particularly noteworthy when assessing for possible interception of cancer, which requires long-lasting immunity,” said Zaidi. “In addition, the vaccine was safe and well tolerated, supporting its use in larger cancer interception studies.”

After a median follow-up of 16.5 months, none of the vaccinated individuals developed cancer. The researchers evaluated changes in cyst size as an exploratory clinical endpoint and found a higher rate of cyst reduction or resolution among the vaccinated individuals (37.5%) relative to an unvaccinated cohort with similar characteristics (6.8%).

“This research underscores the need for further funding to support the development of strategies that can intercept and prevent cancer development in high-risk individuals. More studies are needed to find the best vaccine approaches, the best targets, and the ideal timing for vaccination,” concluded Jaffee.

According to the authors, the study’s limitations include the small size and the fact that the trial was not designed to assess the clinical efficacy of the vaccine. “We observed evidence of stability or regression of the pancreatic cysts in association with the induction and durability of KRAS-specific T-cell responses,” said Michael G. Goggins, MD, professor of pathology, medicine, and oncology and the Sol Goldman Professor of Pancreatic Cancer Research at Johns Hopkins University School of Medicine. “However, larger studies are needed to demonstrate that this effect was in fact due to the vaccine.”

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Parkinson’s in the Clinic, Insilico enters Phase III, Vertex Acquires Crinetics

Edusehat — Sat, 18 Jul 2026 00:40:09 +0700

Promise hits Parkinson’s disease therapy, as a Phase I/II clinical study has demonstrated the feasibility of transplanting stem-cell-derived dopamine progenitor cells into the brain. In preclinical research, a new platform uses the brain’s fluid transport pathways to effectively deliver AAVs to therapeutic targets in mice. We’ll also cover the accelerating infrastructure moment for AI-driven drug discovery, with billion-dollar investments flowing into end-to-end platforms driven by models and compute, rather than single drug assets. In business, Insilico’s revenue leaps as the company’s AI-developed lead candidate moves to Phase III, while Vertex acquires Crinetics for $10 billion.

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

Engineered AAVs Harness Glymphatic System to Reach Brain Targets in Mice
GEN, July 9, 2026

Stem Cell Therapy Shows Promise in First Human Parkinson’s Disease Trial
GEN, July 9, 2026

Pharma Races to Scale AI as Billions Flow into Drug Discovery
By Fay Lin, PhD, GEN Edge, July 6, 2026

StockWatch: Insilico Projects Profit, Revenue Leaps as AI-Developed Lead Candidate Moves to Phase III
By Alex Philippidis, GEN Edge, July 12, 2026

Vertex Eyes Expansion Beyond Cystic Fibrosis with Planned $10B Crinetics Buyout
By Alex Philippidis, GEN Edge, July 7, 2026

Top 20 Drugs Heading for the Patent Cliff, 2026-2029
By Alex Philippidis, GEN Magazine, Nov 1, 2025

Touching Base Podcast
Hosted by Corinna Singleman, PhD

Behind the Breakthroughs
Hosted by Jonathan D. Grinstein, PhD

The State of Biologics Testing 2026
Charles River Laboratories and GEN, June 10, 2026

Produced with support from:

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Gene Therapy Partners Aim to Enhance Protein Expression

Edusehat — Fri, 17 Jul 2026 21:05:09 +0700

Circio Holding and Avenue Biosciences agreed to a research collaboration designed to combine their synergistic technologies to improve long-term expression of secreted proteins, relevant for treatment of a broad range of diseases.

Secreting proteins from cells into surrounding tissue, or into circulation, opens new treatment opportunities beyond classical gene therapy for monogenic disease, say scientists from both companies. Circio maintains that its circVec platform drives higher and more durable protein expression, and a spokesperson from Avenue Biosciences explains that its protein engineering technology improves protein secretion from cells by screening thousands of signal peptide-protein combinations.

Circio and Avenue will jointly explore whether the two technologies in combination can act synergistically to improve the production and secretion of proteins, including antibodies, for the treatment of genetic and chronic diseases.

“Many gene therapies are limited by insufficient protein expression, driving high doses, manufacturing complexity, and cost. The secretory pathway—the cellular machinery that produces and exports proteins—is a largely underutilized engineering opportunity. By combining Circio’s durable circular RNA expression with our technology, we aim to increase protein output per dose and ultimately help more patients benefit from life-changing genetic medicines,” says Avenue Biosciences CEO Tero-Pekka Alastalo, MD, PhD.

In the collaboration, Avenue Biosciences will deploy its protein engineering platform to identify signal peptides that enable improved secretion of therapeutic proteins expressed by circVec. The initial screening will be performed by Avenue Biosciences, followed by further in vitro and in vivo testing by Circio.

“A significant proportion of therapeutically relevant payloads for circVec are secreted proteins,” adds Victor Levitsky, PhD, CSO of Circio. “With the Avenue platform, we will test how signal peptide optimization can enhance secretion of proteins and thereby open novel opportunities for the circVec platform in genetic and chronic disease. This collaboration is an important addition to our pre-clinical development strategy of testing circVec in multiple settings through R&D partnerships to broadly explore the range of therapeutic options available for our unique circular RNA expression technology.”

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There’s a lot of hype around perimenopause. Don’t buy it.

Edusehat — Fri, 17 Jul 2026 17:35:02 +0700

Perimenopause has entered the chat. Perimenopause—and its better-known relative, menopause—used to be considered taboo. Not anymore, thanks at least in part to TV doctors and social media influencers. Perhaps it’s my age, but these days, both my algorithm and my conversations with friends increasingly swing toward perimenopause.

Menopause is defined as the life stage that occurs a year after a person has had their last period. Perimenopause is the sometimes years-long period before that point, which can also feature all the symptoms we’d typically associate with menopause.

Today, information about perimenopause is more prevalent and accessible than ever. If you’re a woman in your 40s and you’re not feeling 100%, chances are there’ll be someone online ready to tell you you’re in perimenopause. And that you might want to start spending your money on blood tests, apps, and supplements or demanding hormone replacement therapy. But as regular readers might have guessed by this point, it’s not that simple.

Perimenopause tends to start around the age of 46 or 47. It’s during this time that many women start to experience some symptoms like hot flashes, irregular or unusually heavy periods, or anxiety, for example. And it can be heavy going. “Often symptoms are at their worst in the perimenopause,” says Mary Ann Lumsden, former president of the International Menopause Society.

That’s because hormones can fluctuate wildly. Levels of estrogen, progesterone, luteinizing hormone, and follicle-stimulating hormone can roller-coaster before leveling off after menopause. And that’s why, despite what some marketers will claim, there is no test for perimenopause.

“You can’t interpret hormone [measures] because they change so much,” says Lumsden. “And that is quite normal.”

That doesn’t mean women should have to put up with symptoms. But exactly how those symptoms are treated is another topic that has been clouded by misinformation.

Last week, I told a friend about some unusually bad pelvic pain I’d experienced. Her immediate advice was to find out if I was perimenopausal and, if I was, to request hormone replacement therapy (HRT) as soon as possible. If my doctor wouldn’t prescribe it, she continued, I should simply find another doctor who would.

This line of thinking has been heavily promoted on social media platforms, says Paula Briggs, a former chair of the British Menopause Society who currently leads the menopause service at Liverpool Women’s Hospital. But it’s not helpful.

HRT is essentially designed to top up or replace hormones like estrogen and progesterone, which naturally decline around menopause. There are lots of different drugs that can be taken in lots of different ways and at various doses.

While it does come with some risks and won’t suit everyone, HRT can be immensely helpful for many menopausal women. Not only can it help with many of the common symptoms of menopause, but it can also help prevent osteoporosis and maintain muscle strength.

But these drugs were trialed in, and approved for, menopausal women, says Lumsden. They won’t have the same effects in perimenopausal women. “If you give standard HRT, it may well get swamped by [the woman’s] own hormone production,” she says.

HRT can also cause abnormal bleeding in perimenopausal women, says Briggs.

She’s concerned about the messaging on perimenopause that is being promoted on social media. Particularly worrisome, she says, is the way younger women are being encouraged to assume they are perimenopausal and seek out HRT treatment.

“It’s almost cult-like, this idea that everybody must have HRT,” she says.

And then there are the supplements. There’s been an explosion in marketing for vitamins and supplements specifically targeted to middle-aged and menopausal women. But the evidence for these, too, is either limited or nonexistent. “I can’t see a mechanism for a lot of them,” says Lumsden.

Women who take these supplements don’t always know what they’re getting. Some of Lumsden’s patients have told her they take testosterone supplements to manage their symptoms. But blood tests revealed no increase in testosterone levels. “Whatever they’re getting, it’s not testosterone,” she says.

At any rate, not all the symptoms women experience in midlife can be blamed on hormones. The lengthy lists of perimenopause symptoms shared on social media include fatigue, brain fog, aches and pains, digestive issues, and more. “These do not link closely to the obvious menstrual cycle changes and hormone changes … across menopause,” says Nanette Santoro, a professor of obstetrics and gynecology at the University of Colorado Anschutz who studies menopause.

If you’re experiencing any symptoms, it’s worth getting them checked out to make sure they’re not being caused by something else. My own pelvic pain, for example, is almost definitely the result of endometriosis—a condition that can be made worse by HRT, Lumsden tells me.

At any rate, by the time women reach their 40s, many are already juggling care for children and aging parents, often while holding down a job (and dealing with pressures from societies that don’t appear to value older women). It’s an exhausting time—and not all of that exhaustion can be blamed on hormones.

As Santoro puts it: “Attributing everything unpleasant that happens to a woman over 35 to perimenopause is not based on any scientific evidence.”

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.

Modular Antibody–ADC Click Chemistry Overcomes Tumor Drug Resistance in Mice

Edusehat — Fri, 17 Jul 2026 13:55:16 +0700

Cancer drugs that can find and kill tumor cells with molecular precision have reshaped oncology—but their power fades when tumors evolve, diversify, or simply stop presenting the right molecular targets. Now, researchers at Washington University (WashU) School of Medicine in St. Louis have unveiled a modular “click‑to‑assemble” strategy that supercharges these therapies inside the body, allowing them to hit multiple tumor targets at once and reverse drug resistance in mice.

The study, published in Nature, is titled “Modular in vivo antibody–ADC click to reverse drug resistance in tumors,” and introduces a bioorthogonal ligation strategy that forms a dual‑targeting antibody–drug conjugate (ADC) with improved tumor uptake and therapeutic efficacy. In pancreatic, gastric, and breast cancer models, the approach slowed or halted tumor progression—even in tumors with low, ultralow, or heterogeneous HER2 expression, where conventional HER2‑directed ADCs typically fail.

“We’ve shown that when two cancer‑targeting antibodies bind together inside the body, they accumulate at the tumor more effectively and improve treatment response,” said senior author Patrícia M. Ribeiro Pereira, PhD, assistant professor of radiology at WashU Medicine’s Mallinckrodt Institute of Radiology. “There is a lot of excitement here because we have shown that it isn’t necessary to create a whole new drug platform for each therapeutic target. We can repurpose antibodies that already exist to improve treatments.”

The team’s strategy—called antibody–ADC click—relies on the rapid ligation between trans‑cyclooctene (TCO) and tetrazine moieties. In the study, one antibody (such as panitumumab, targeting EGFR) was modified with TCO, while a second antibody or ADC (such as trastuzumab‑deruxtecan, T‑DXd) carried tetrazine. When administered sequentially, the two components “clicked” together, forming a higher‑order complex that internalized more efficiently and delivered more cytotoxic payload.

The paper described the problem bluntly: “ADC efficacy remains constrained by its dependence on a single target antigen, which limits tumor targeting and promotes resistance in heterogeneous tumors with variable and low antigen expression.” The click strategy, the authors wrote, “provides a modular and translatable approach for enhanced targeted drug delivery in heterogeneous tumors.”

In mice bearing tumors with mismatched HER2 and EGFR expression, the clicked ADCs accumulated at levels up to 3.2‑fold higher than standard ADCs.

The modularity is the point. “The approach is flexible enough to be adapted to new cancer targets as we learn more about what drives treatment resistance,” Ribeiro Pereira added.

Next steps include further development and optimization of the click-enabled ADC platform prior to future clinical translation, along with expanding the platform to notoriously hard‑to‑treat cancers. “We’re trying to optimize this tool to help antibodies reach tumors that are normally very difficult to treat, such as brain tumors,” Ribeiro Pereira said.

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AI-Designed Synthetic CRISPR-Like Nucleases Show Activity in Cells

Edusehat — Fri, 17 Jul 2026 06:45:08 +0700

A new paper published in Science describes using artificial intelligence (AI) to design functional synthetic RNA-guided nucleases whose activity matches or exceeds that of natural enzymes. In the paper, titled “Structure and evolution-guided design of minimal RNA-guided nucleases,” the scientists wrote that the results “establish a strategy for creating non-natural RNA-guided nucleases and conformationally active nucleic acid binders, enlarging the designable protein space.”

The team includes scientists from Innovative Genomics Institute and the California Institute for Quantitative Bioscience, both at the University of California, Berkeley, and collaborators at other institutions.The findings highlight AI’s ability to expand the CRISPR toolbox to include RNA-guided nucleases with novel properties beyond those found in nature. It is a task that has been challenging for protein design methods because of the complexity of multi-domain proteins, “whose activity depends on coordinated RNA and DNA recognition, activation, and cleavage by distinct conformational states,” the scientists wrote. As such, seemingly small changes can disrupt enzyme activity.

Sequence-based biological language models have been used to successfully design new nucleases by inferring sequence-function relationships, but they often produce versions of these proteins that closely resemble the reference sequences used to train them. Meanwhile, structure-guided rational design approaches, which “offer a robust strategy to sample highly divergent protein sequences” as well as “structures not found in nature” have been used to generate things like dynamic switches and DNA binders. However, designing complex proteins like RNA-guided nucleases with multiple functional domains and conformations has remained challenging for these methods.

In the Science paper, the scientists present an alternate strategy for generating novel functional proteins that combines the ESM Inverse Folding (ESM-IF1) model with evolution-informed residue constraints. As a test case, they used it to generate new variants for TnpB, a family of CRISPR-cas12-like nucleases that mediate RNA-guided DNA cleavage and regulate transcription among other tasks. Members of this enzyme family are “an attractive target for protein design because they couple programmable DNA targeting and a variety of natural functions to a minimal architecture,” the scientists explained in the paper.

The results showed that compared to sequence-based biological language models which generated proteins with binding domains with over 99% identity to natural homology, their approach created “DNA- and RNA-interacting lobes with AI-generated contacts that had 83% and 72% identity to their closest counterparts in nature.”

As part of the study, the scientists screened the activity of the designed proteins, dubbed SynTnpBs, first in the bacterial cells and then selected the most active ones for further testing in plant and human cells. They also used cryo-electron microscopy to determine the structures of the most divergent variants. Their analysis showed that many AI-designed nucleases either retained or surpassed the activity of natural TnpB in multiple cell types. Microscopy studies further revealed that the engineered proteins formed new electrostatic and hydrogen-bonding networks that stabilize interactions at the RNA-DNA interface across different conformations.

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BIO 2026: FDA leaders discuss restoring workforce, institutional knowledge

Edusehat — Thu, 16 Jul 2026 23:40:03 +0700

While the U.S. Food and Drug Administration (FDA) has faced significant change in recent years, industry leaders and agency officials alike say there are reasons for optimism.

This was a key topic of conversation when John F. Crowley, President and CEO of the Biotechnology Innovation Organization (BIO), moderated a June 23 town hall with the agency’s newly appointed leaders at the 2026 BIO International Convention.

One major reason for this optimism: the recent effort to rebuild the FDA’s workforce and reestablish the agency’s institutional knowledge.

Maintaining core principles, while evolving for a new era

“Priority number one is to ensure we are there to support the companies that are innovating, and that we can deliver what they will deliver, and can also ensure they advance,” said Karim Mikhail, B.Pharm, MSc, Acting Director of the Center for Biologics Evaluation and Research (CBER).

“The second priority is that we have a number of open files, some of them the second or third resubmissions, and we want to make sure that we have operational continuity, and that we address some of these challenges together with them,” he continued.

“Number three is to think about the future,” he said. “Are we equipped to handle not only the challenges of June or July 2026, but are we equipped to think about the future? Because if we don’t do this now, very quickly, we are behind.”

Predictability is another priority, added Lowell Zeta, J.D., Acting Chief of Staff at the Office of the Commissioner. This is important so that the FDA can maintain what has worked, while updating execution to meet the needs of an ever more rapidly paced world.

“Our core pillars remain the same,” he said, listing “more cures,” “healthier foods,” “chronic disease, global competition and resiliency, and national security.”

“Our execution has changed a bit,” he continued. “Where we’re working really closely now in this phase with center staff to make sure that we have not just made the right changes, but they’re sustained.”

Why FDA workforce development and retention are key

FDA leaders understand that workforce development and retention are key to meeting these goals.

“My top priority for this initial period in this role is really fortifying the center, and specifically the workforce,” said Michael Davis, M.D., Ph.D, Acting Director of the Center for Drug Evaluation and Research (CDER). Boosting morale is also top of mind as he helps develop the organization’s workforce.

“One thing we are laser-focused on is retaining the amazing staff,” he continued. “You may read about the loss of institutional knowledge and people retiring, and so forth, and that’s true, but what I’ve really seen and appreciated is that the wonderful office leadership at CDER has been very, very stable and a very stabilizing force in the work.”

To maintain attrition, Davis said, FDA leaders are engaging with the staff who weathered the Department of Government Efficiency (DOGE) cuts.

“Slowing or stopping the attrition is important,” said Mikhail. “There is dedicated leadership support to ensure everybody who’s thinking of leaving is getting a leader meeting—not to say, This is an exit interview, but rather, Can you come for a coffee to discuss how we can keep you because what you bring to the table is very important.”

“We are working to engage with staff and really lean on their ideas of how we can really set things into motion in ways that will really stick and take hold and change the way we do things going forward,” said Davis. “I’ve been tracking the attrition rates at CDER. The attrition is actually down to its historical rate.”

How FDA is rebuilding the workforce

The FDA has been approved by the White House Office of Management and Budget (OMB) to hire 2,200 people after shedding more than 3,000 positions last year.

“We are making progress, good progress, and it’s steadier and swifter,” said Zeta. “We’re at about 600 that are in onboarding clearance at various levels, with about 200 already through the process.”

But FDA leaders admit they were unsure how eager job seekers would be, given the recent tumult.

“I thought with everything that had happened, that we’d put a job out and then have very few candidates,” said Mikhail. “But instead we’re seeing very significant numbers of people applying – and very good quality people.”

In addition, the FDA leaders discussed efforts to bring back scientists and leadership who had previously been let go, a notable reversal of early policy.

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See, Blind Mice: Consortium’s Drugs Restore Sight

Edusehat — Thu, 16 Jul 2026 23:35:09 +0700

A consortium led by scientists at the Institute for Bioengineering of Catalonia (IBEC) has developed a series of light-activated small molecule drugs that in preclinicial tests restored sight in blind mice. The team’s approach is based on photopharmacology, a technique for reversibly control drug activity using light.

The newly developed compounds, called prosthe6, mimic the function of light sensing photoreceptor cells, which degenerate in blinding diseases such as age-related macular degeneration (AMD) and retinitis pigmentosa (RP).

The prosthe6 compounds target ON-bipolar neurons and in tests were found to successfully restore saccadic eye movements (optokinetic reflex) in blinded zebrafish larvae, a widely used model for studying visual acuity. Even more strikingly, the researchers demonstrated recovery of innate light-avoidance behavior in mouse models of age-related macular degeneration and retinitis pigmentosa.

Test results suggest that the prosthe6 compounds may be administered by injecting them in the eye, or administered as eye drops. In animal studies the photoswitchable molecules also showed promising preliminary safety profiles, pointing to the development of potential drug candidates for restoring vision in patients with degenerative retinal diseases, without the need for genetic manipulation or implanted devices. Importantly, these compounds are designed to work under normal lighting conditions and do not require light-enhancing devices as optogenetics. They are small, water-soluble molecules that respond to ordinary visible or white light, such as indoor lighting or daylight, without requiring intense or specialized light sources.

“These molecules do not cure blindness, because they do not address the cause of photoreceptor degeneration,” said study co-lead Pau Gorostiza, PhD, ICREA Research Professor at IBEC, leader of the Nanoprobes and Nanoswitches group, member of CIBER-BBN. “But they are remarkably effective at restoring sight, and they do so using a very simple and potentially patient-friendly approach.”

Rosalba Sortino, former PhD student at the University de Barcelona, and currently post-doctoral researcher at Gorostiza’s group at IBEC, added, “Our goal was to restore vision using a molecular mechanism that is as close as possible to how the healthy retina works … Instead of bypassing retinal processing, we aimed to reactivate it right at the same level of the retinal circuit than the lost photoreceptor cells.”

Sortino is co-first author of the team’s published paper in Journal of the American Chemical Society, titled “Restoration of saccadic eye movements and visually guided behavior in ambient white light with photoswitchable small molecules.”

Diseases such as age-related macular degeneration and retinitis pigmentosa affect 200 million people worldwide and are the leading causes of visual impairment and blindness. Beyond the personal impact on quality of life and independence, vision loss places a global economic burden estimated at over US$400 billion per year in healthcare costs and lost productivity.

Researchers Rosalba Sortino (left) and Joaquin Martinez Tambella (right) working in the laboratories of the Institute for Bioengineering of Catalonia (IBEC). Sortino is a post-doctoral researcher at the Nanoprobes and Nanoswitches group at IBEC and co-first author of the study. Martinez is a PhD student at the Nanoprobes and Nanoswitches group at IBEC and co-first author of the study. [Institute for Bioengineering of Catalonia (IBEC)]

In many of these conditions, photoreceptor (PhR) cells—the retina’s light detectors—progressively degenerate and die. Although the downstream retinal neuronal circuitry remains largely intact and functionally viable, it no longer receives the light signals needed to drive visual processing towards the brain. This opportunity has fueled intense research efforts to develop treatments capable of restoring light sensitivity to the eye. Current strategies include gene therapy—effective only for a very small subset of patients with specific mutations—and electronic retinal prostheses, which are invasive, expensive, and require extensive training for effective use.

More recently, optogenetics and light-responsive drugs have entered clinical testing, the latter with encouraging safety results. “Photopharmacology can develop photoswitchable small molecules to restore vision impairment by conferring light sensitivity to ion channels that are widely expressed in the remaining inner retinal neurons, and a first-in-human clinical trial is ongoing,” the team noted. However, achieving high-quality vision at ambient illumination levels remains a major challenge.

The (IBEC)-led consortium has now developed a new class of photoswitchable small-molecule drugs that are capable of restoring key visual functions in animal models of blindness. The team’s photopharmacology-based technique involves modifying a drug’s chemical structure by adding a light-activated molecular switch, enabling control of the pharmacological action using light. “Unlike (opto)genetic manipulation and surgically implanted retinal electronic prostheses, pharmacotherapy is noninvasive, readily reversible, and can be upgraded when new drugs are approved,” the authors noted. “Medicines are preferred by patients, clinicians, and public healthcare systems, they can be developed and manufactured at lower costs than other approaches and assessed by conventional regulatory procedures and clinical assays.”

The reported work builds on more than a decade of research and was carried out in collaboration with the team co-led by Pedro de la Villa at the University of Alcalá (UAH), as well as researchers from the Institut de Química Avançada de Catalunya (IQAC-CSIC), the University of Barcelona (UB), the Institute Ramón y Cajal of Health Research (IRYCIS), the Autonomous University of Barcelona (UAB), and the Fundació Eduard Soler.

Researcher Joaquin Martinez Tambella working in the laboratories of the Institute for Bioengineering of Catalonia (IBEC). Martinez is a PhD student at the Nanoprobes and Nanoswitches group at IBEC and co-first author of the study. [Institute for Bioengineering of Catalonia (IBEC)]

The prosthe6 compounds work by acting on a specific type of retinal cells called ON bipolar cells, which normally receive signals from the photoreceptors. “In healthy vision, ON bipolar cells play a key role in passing on information about the presence of light to the rest of the visual circuit,” explained study co-lead de la Villa. “In degenerative eye diseases, although the photoreceptors are lost, much of this underlying circuitry remains intact but inactive. This creates a major therapeutic opportunity.”

By targeting a protein (mGlu6) in this preserved part of the retina, prosthe6 compounds can take over the role of the missing photoreceptors. “… we have targeted metabotropic glutamate 6 (mGlu6) receptors, which are exclusively expressed in ON bipolar cells (OBCs) and localized postsynaptic to PhR cells, thereby leveraging a privileged position to drive physiological visual circuit,” the investigators explained. When light enters the eye, the molecules respond by changing their shape, triggering signals inside the retina in a way that closely resembles natural vision. In this way, the drugs effectively act as “molecular prostheses,” helping the eye process light again without the need for implants or genetic modifications.

Healthy mice naturally prefer to remain in dark environments and instinctively avoid brightly lit areas, a behavior that relies entirely on a functional visual system. Blind mice, by contrast, lose this preference and move indistinctly between light and dark spaces, as they are unable to perceive light. The team showed that after treatment with prosthe6, blind mice once again showed a clear and spontaneous preference for dark areas, indicating that they could perceive light and use this information to guide their behavior.

This recovery occurred without any training and under light levels comparable to those found indoors or on an overcast day, demonstrating that the treatment restores functional light perception capable of driving natural, visually guided behavior.

Two lead compounds, prosthe6-12 and prosthe6-15, showed particularly promising results. The restored behaviors were observed not only after intraocular injection, but also after topical administration as eye drops. “… at least two compounds (prosthe6-12 and -15) appear to be devoid of adverse effects and restore sight by topical administration, which is linked to higher overall clinical success rate than systemic routes for neurological drugs, and to stronger patient adherence,” the investigators pointed out.

The prosthe6 technology is protected by patent and the researchers are now evaluating its safety and formulation to extend the duration of visual rehabilitation. The team is working with Eyelumina, a spin-off company in formation to secure investments that support translational development and future clinical trials.

“Turning this into a therapy is a long and laborious process,” says Gorostiza. “But the results show that there is a realistic possibility of restoring high-quality vision with drugs, non-invasively, reversibly and with a mechanism that is independent of the specific retinal disorder or genetic mutation to reach a majority of patients.”

If successful in humans, the drug-based approach would offer a widely accessible and affordable alternative to existing vision restoration technologies, especially relevant for patients with advanced retinal degeneration for whom no effective treatments currently exist.

In their paper the team further stated, “From a fundamental perspective, prosthe6 constitute new tools for ophthalmology to study the physiopathology of mGlu6 receptors and retinal circuits in vitro and in vivo and contribute to the medicinal chemistry of allosteric modulators. They also achieve the prediction that upstream targeted photopharmacology can deliver nearly native output signals, taking full advantage of the retinal circuit for high-quality vision restoration.”

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Autolomous and Cellular Origins Expand Cell Therapy Manufacturing Process

Edusehat — Thu, 16 Jul 2026 20:00:10 +0700

Officials at Autolomous and Cellular Origins say the companies have carried out the end-to-end integration of their respective platforms to bring full automation and digitization to the entire cell therapy manufacturing process. This new scalable approach creates a connected manufacturing environment that gives developers greater standardization, traceability, and control across the entire manufacturing process, according to the companies’ spokespersons.

Cellular Origins’ Constellation^® automated platform was created to enable cell therapy manufacturing, integrating mobile robotics, existing bioprocessing technologies, and sterile fluid transfer into a coordinated operation. Its flexible, modular architecture allows developers and manufacturers to scale manufacturing capacity as demand grows while maintaining standardized processes, avoiding therapy redevelopment and reducing scale-up risk, notes Edwin Stone, CEO of Cellular Origins.

Autolomous’ digital autoloMATE^® platform was designed to allow real-time data exchange and integration across existing software and AI systems, as well as devices and robotic platforms across the entire manufacturing process and supply chain, while safeguarding intellectual property. The modular architecture allows each deployment to be configured to customer needs.

The combined solution overcomes fragmentation, limited visibility, and the absence of standardized, interoperable data flows without replacing existing processes, enabling manufacturers to scale to commercially viable levels, maintains Alexander Seyf, CEO of Autolomous. First integrations have already been achieved at the Cell and Gene Therapy Catapult Digital and Automation Testbeds, as part of an Innovate UK-funded project.

“Scientific ambition has never been the bottleneck in bringing innovative cell therapy to patients, but the delivery infrastructure has brought many challenges,” continues Seyf. “Together with Cellular Origins, we enable fast and efficient scaling from research through to patient administration, ensuring standardization, automation and digitization across the entire process.”

“Scaling cell therapy manufacturing is not just a question of employing automation. It requires a manufacturing system that can evolve with demand,” adds Stone. “Our collaboration with Autolomous, and joint work with the Cell and Gene Therapy Catapult, clearly demonstrates how robotic platforms and digital infrastructure can operate as one.”

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BIO expresses concerns over proposed OMB rule to regulate federal grants

Edusehat — Thu, 16 Jul 2026 12:55:03 +0700

A new proposal to regulate federal grants risks destabilizing the system of government support essential to developing innovative therapies American patients need, the Biotechnology Innovation Organization (BIO) warns.

The Office of Management and Budget (OMB) says its proposed Regulation for Federal Financial Assistance “would improve transparency, accountability, and oversight for Federal awards.” The new rule would make it easier to suspend or cancel federal grants, emphasize priority of “agency discretion” over peer review of proposals, and enhance “alignment of Federal awards with administration priorities,” OMB explains.

BIO’s comments focus on the anticipated impact of the proposed regulation on the biotechnology sector, saying it will interfere with the system that generates biomedical innovation.

“Federal funding plays a critical role in enabling innovation across the biomedical research ecosystem, supporting the foundational scientific work that underpins private-sector drug development,” BIO’s comments say. “Disruptions to this funding model—including increased uncertainty, administrative complexity, or constraints on participation—would not only affect academic researchers but also have direct implications for private-sector investment, partnership formation, and the advancement of new therapies.”

Specific concerns

BIO comments cover six general areas of concern:

“The proposed rule will destabilize the biomedical research ecosystem and delay patient access to novel medicines.” The rule would include addition of “at will” termination authority for grant-making agencies and provide new powers to suspend grants. The resulting uncertainty of funding for personnel or infrastructure would make institutions hesitant to embark on new research projects, impacting later private investment and ultimate development of new therapies, BIO says.
“The proposed rule will weaken merit-based funding decisions and redirect resources away from high-impact biomedical research.” By treating independent peer review as merely a recommendation, and stating that program goals must align with Administration priorities, “the proposed rule would undermine review based on scientific merit in favor of more subjective considerations,” BIO says.
“The proposed rule will restrict vital international research collaboration and weaken U.S. competitiveness.” New prohibitions against using funds with foreign companies or entities would prevent involvement of international experts, something that is normal in the globalized biotech ecosystem. “The provision may have significant implications for biomedical research, which often relies on multinational collaborations,” per BIO’s comments.
“The proposed rule will undermine emergency preparedness and U.S. health security.” Being ready for public health emergencies requires long-term preparation work in combination with rapid responses when an emergency arises. Flexible funding mechanisms that enable emergency responses could be hampered by new procedural requirements introduced by the proposed rule, BIO notes.
“The proposed rule will impede scientific communication and slow the translation of research into medical innovation.” The rule would restrict use of federal grants for communication efforts, ranging from publication to conference attendance to advertising. This would make it difficult for researchers to share their innovations, so they can find investors and research partners, and make it harder for the public to become aware of innovations that may impact them, BIO says.
“The proposed rule raises several legal concerns about agency authority, particularly as to proposals that conflict with the statutory purposes of NIH research grants and other key discretionary programs.” “The proposed rule raises a number of legal concerns under the Administrative Procedure Act (APA), including the potential conflicts between the proposed rule and program-specific statutes; the challenges of commenting on so broad a rule that would affect so many different programs,” and several others, according to BIO’s comments.

BIO concludes that the proposed rule is not workable and recommends that other means for introducing transparency and accountability be found.

“As drafted, the proposed rule would introduce significant uncertainty and impose constraints that are fundamentally misaligned with the needs of the national biomedical research ecosystem,” BIO’s comments say. “If finalized, this rule would risk disrupting critical research, weakening U.S. competitiveness, and slowing the pace of innovation—ultimately harming patients.”

Read BIO’s comments in full.

CSBA comments

The Council of State Bioscience Associations (CSBA), a confederation of state-based, non-profit trade organizations recognized by BIO as affiliate organizations, also submitted comments recommending against the current proposed regulation.

“Support from a variety of federal government agencies plays a key role in the ability to take breakthrough innovations from basic scientific research through multi-year development processes before finally reaching an approved therapy American patients can benefit from,” says CSBA’s comments.

“The provisions contained in the proposed rule would destabilize critical research and weaken U.S. leadership in biotechnology innovation,” CSBA’s comments continue. “Therefore, the CSBA urges OMB to either withdraw the proposed rule or revise it, in collaboration with stakeholders.”

Read the CSBA comments in full.

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BIO opposes proposed OMB rule to regulate federal grants

Edusehat — Thu, 16 Jul 2026 05:45:03 +0700

BIO’s comments focus on the anticipated impact of the proposed regulation on the biotechnology sector. They recommend against the proposed regulation, saying it will interfere with the system that generates biomedical innovation.

Specific concerns

BIO comments cover six general areas of concern:

“The proposed rule will destabilize the biomedical research ecosystem and delay patient access to novel medicines.” The rule would include addition of “at will” termination authority for grant-making agencies and provide new powers to suspend grants. The resulting uncertainty of funding for personnel or infrastructure would make institutions hesitant to embark on new research projects, impacting later private investment and ultimate development of new therapies, BIO says.
“The proposed rule will weaken merit-based funding decisions and redirect resources away from high-impact biomedical research.” By treating independent peer review as merely a recommendation, and stating that program goals must align with Administration priorities, “the proposed rule would undermine review based on scientific merit in favor of more subjective considerations,” BIO says.
“The proposed rule will restrict vital international research collaboration and weaken U.S. competitiveness.” New prohibitions against using funds with foreign companies or entities would prevent involvement of international experts, something that is normal in the globalized biotech ecosystem. “The provision may have significant implications for biomedical research, which often relies on multinational collaborations,” per BIO’s comments.
“The proposed rule will undermine emergency preparedness and U.S. health security.” Being ready for public health emergencies requires long-term preparation work in combination with rapid responses when an emergency arises. Flexible funding mechanisms that enable emergency responses could be hampered by new procedural requirements introduced by the proposed rule, BIO notes.
“The proposed rule will impede scientific communication and slow the translation of research into medical innovation.” The rule would restrict use of federal grants for communication efforts, ranging from publication to conference attendance to advertising. This would make it difficult for researchers to share their innovations, so they can find investors and research partners, and make it harder for the public to become aware of innovations that may impact them, BIO says.
“The proposed rule raises several legal concerns about agency authority, particularly as to proposals that conflict with the statutory purposes of NIH research grants and other key discretionary programs.” “The proposed rule raises a number of legal concerns under the Administrative Procedure Act (APA), including the potential conflicts between the proposed rule and program-specific statutes; the challenges of commenting on so broad a rule that would affect so many different programs,” and several others, according to BIO’s comments.

BIO concludes that the proposed rule is not workable and recommends that other means for introducing transparency and accountability be found.

Read BIO’s comments in full.

CSBA comments

Read the CSBA comments in full.

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Ketogenic Diet Shows Opposite Effects on Cancer Risk in Mouse Small Intestine and Colon

Edusehat — Thu, 16 Jul 2026 05:40:10 +0700

Ketogenic diets, originally developed in the 1920s to treat epilepsy, have been adapted in the past few decades as a strategy to lose weight or increase lifespan. This type of diet (a high percentage of fat, low percentage of carbohydrates, and normal or reduced amounts of protein) forces the body to burn fatty acids for energy in place of carbohydrates such as glucose. Burning these lipids produces ketone bodies—primarily β-hydroxybutyrate (BHB) and acetoacetate—as byproducts of fatty acid metabolism. The impact of ketogenic diets on the gastrointestinal tract remains poorly understood.

In recent years, scientists investigated whether this type of diet might affect the development of cancer. While some research has shown that the diet may protect against the development of colon cancer, a new study suggests that in the small intestine, a ketogenic diet may increase the risk of cancer—with a mechanism through fatty acid oxidation rather than ketone metabolism.

This work appears in Nature in the paper, “Ketogenic diet mediates intestinal tumorigenesis through lipids not ketones.”

“Ketogenic diets have distinct effects on different tissues even within the gastrointestinal tract. I think the message here is that we need to be very careful in generalizing the effects that these diets can have, because what might be beneficial for one tissue may be detrimental for another tissue,” says Omer Yilmaz, PhD, director of the MIT Stem Cell Initiative, an associate professor of biology at MIT, and a member of MIT’s Koch Institute for Integrative Cancer Research.

A 2022 study in Nature suggested that ketogenic diets have a protective effect against colon cancer and that BHB—the most abundant ketone body—is responsible for this effect. In the new study, the MIT team wanted to explore whether ketogenic diets might have a similar protective effect in the small intestine.

The researchers fed mice who were genetically predisposed to developing intestinal cancer either a ketogenic diet, a control diet, or a high fat/high calorie diet. They found that mice on a ketogenic diet were more likely to develop tumors of the small intestine than those on a control diet. While they did not become obese, mice on the ketogenic diet developed tumors at rates similar to or even higher than those of mice on an obesogenic high fat/high calorie diet.

Additional studies revealed that ketone bodies did not play a role in tumor development. Instead, tumor growth was driven by fatty acid oxidation. This pathway activates the PPAR family of proteins, which signal stem cells to multiply more rapidly, increasing the chance that some become cancerous.

Surprisingly, the same ketogenic diet that promoted tumors in the small intestine had the opposite effect in the colon. The researchers found, similar to the earlier study back in 2022, that a ketogenic diet suppressed the development of colon tumors. However, the new findings suggest that ketone bodies are not responsible for this protective effect.

“Given how much attention has been paid to ketone bodies like BHB, both as a commercial health trend and in recent high-profile studies suggesting BHB suppresses colon cancer, we fully expected them to be the direct drivers. Instead, our experiments in genetically engineered mice revealed that these molecules are essentially metabolic bystanders. The real surprise is that tumor acceleration is driven entirely by how stem cells process and burn the heavy influx of dietary fat itself,” Yilmaz says.

The researchers now hope to further study why ketogenic diets have such different effects in the colon and the small intestine. As ketogenic diets continue to gain popularity, understanding these tissue-specific effects will be critical for guiding their use, the researchers say.

The findings carry practical implications. Because the diet’s effects—both the tumor acceleration in the small intestine and the protection in the colon—are driven entirely by fat metabolism rather than the ketones themselves, commercial ketone supplements or drinks would not be expected to mimic either the risks or the benefits discovered in this study. This may be especially relevant given that small intestinal tumors have been rising in incidence in recent decades, with the greatest impact on patients with inherited conditions that predispose them to intestinal cancer, such as familial adenomatous polyposis.

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Increased Levels of Micro- and Nanoplastics Found in the Blood of Heart Attack Patients

Edusehat — Thu, 16 Jul 2026 05:40:08 +0700

The results of a newly reported study have shown that people who suffered a serious heart attack had higher levels of micro- and nanoplastics (MNPs) in their blood, compared with MNP levels in patients diagnosed with chronic ischemic heart disease and those who have normal blood vessels supplying the heart. The study findings also revealed that people who smoke and people exposed to higher levels of air pollution had higher levels of micro- and nanoplastics in their blood.

Headed by teams at Sapienza University of Rome, at the University of Verona, and at the Research Centre on Environmental Pollution and Cardiovascular Diseases at the University of Campania “Luigi Vanvitelli,” the study included 61 patients at Sant’Andrea University Hospital or Azienda Ospedaliera Universitaria Integrata of Verona, diagnosed with either a heart attack, chronic ischemic heart disease, or normal coronary arteries.

The researchers say their study adds to growing evidence that environmental pollution may affect cardiovascular health. Research lead Emanuele Barbato, MD, PhD, at Sapienza University of Rome, said, “These findings do not prove that microplastics cause heart attacks, but they reveal a strong association between environmental exposures, microplastics in the blood and cardiovascular disease. In our study, smoking history was strongly linked to microplastics in the blood. Our findings suggest that smoking might make it easier for micro and nanoplastics to enter the blood stream via the lungs. Air pollution may act in a similar way.”

Barbato is director of the Cardiology Unit of Sant’Andrea University Hospital, Rome, Italy, and senior author of the team’s published paper in European Heart Journal, titled “Micro- and nano-plastics in the coronary circulation and air pollution exposure in ischemic heart disease presentation.”

Cardiovascular diseases are increasingly related to lifelong environmental exposures, the authors noted. Among such exposures, MNPs are ubiquitous environmental pollutants, and evidence is increasing that they accumulate in human tissues following exposure and are emerging as a risk factor for health. Pasquale Paolisso, MD, PhD, at Sant’Andrea Hospital Sapienza University of Rome, said, “Micro and nanoplastics are tiny plastic particles that are found virtually everywhere in the environment, including the air we breathe, the water we drink, and many foods we consume. In recent years, scientists have begun to detect these particles in human tissues and organs, raising concerns about their potential health effects.”

Research findings have raised concerns about the potential role that MNPs may play in cardiovascular diseases. “Emerging evidence indicates that MNPs, once considered inert contaminants, are biologically active pollutants contributing to the pathophysiology of cardiovascular diseases, particularly by promoting the development and progression of atherosclerotic plaques and potentially triggering adverse cardiovascular events,” the team stated.

However, as Paolisso further noted, “… very little was known about whether these particles are present in the coronary circulation—the blood flowing through the arteries that supply the heart—or whether environmental exposures such as smoking and air pollution might influence their presence.” As the authors explained, “… current knowledge is predominantly based on in vitro experiments and preliminary ex vivo findings, highlighting the need for in vivo and clinical investigations.”

For their newly reported study the team measured MNPs in coronary and peripheral blood, in 61 patients at Sant’Andrea University Hospital or Azienda Ospedaliera Universitaria Integrata of Verona, who were undergoing coronary angiography for suspected coronary artery disease (CAD). Patients were stratified as those with ST-segment elevation myocardial infarction (STEMI), chronic coronary syndromes (CCS) and controls with normal coronary arteries.

As well as taking blood samples from the vessels supplying the heart and from elsewhere in the body, the team collected data on whether the patients were smokers and their exposure to pollution, both on the day of testing and over the preceding two years. Coronary micro and nanoplastics were analyzed at the Research Centre for Environmental Pollution and Cardiovascular Diseases, University of Campania ‘Luigi Vanvitelli,’ a center dedicated to understanding how environmental pollutants influence cardiovascular health.

The results showed that micro and nanoplastics were detected in 84% of patients diagnosed with heart attack, compared with 40% of patients with chronic ischemic heart disease and 32% of patients with normal coronary arteries. “The observation that IL-6 and TNF-α concentrations were highest in STEMI patients, particularly within the coronary circulation, and were more elevated in the presence of detectable MNPs supports an exploratory association between MNP burden and a localized pro-inflammatory milieu in patients with obstructive CAD,” the investigators suggested.

Heart attack patients also had a greater variety of plastic types in their blood. The most common type of plastic was polyethylene (PE), which is commonly used in packaging and consumer products. “Across all study cohorts, PE was the most frequently identified polymer, being present in 97% of the patients with detectable MNPs,” the investigators added.

Patients exposed to higher long-term levels of air pollution (PM2.5; particles measuring 2.5 μm or less in diameter) were more likely to have microplastics in their blood, and smokers were six times more likely to have microplastics in their blood. All patients who were smokers and were exposed to higher air pollution levels had plastics in their blood, compared with only 12.5% of patients who did not smoke and were not exposed to higher levels of air pollution. “MNPs, PM2.5, and smoking constitute potentially modifiable environmental risk factors for cardiovascular diseases, with significant implications for public health and cardiovascular disease prevention,” the scientists stated. “Future research should aim to quantify individual MNP exposure, assess combined pollutant burden, and validate interventions that target this expanded network of environmental cardiovascular hazards.”

Barbato added, “The results highlight the need to consider microplastic pollution as part of the broader environmental determinants of health. Policies that reduce air pollution, tobacco exposure, and environmental plastic contamination could have benefits that extend beyond environmental protection and potentially improve cardiovascular health.”

In an accompanying editorial Andreas Daiber, PhD, at University Medical Centre of the Johannes Gutenberg University, Mainz, and colleagues pointed to the observation by Paolisso et al. of an association between NMP levels and exposure to air pollution and tobacco smoking. “While the underlying mechanisms remain unclear, this finding underscores a key principle: environmental exposures rarely occur in isolation,” Daiber and colleagues stated. “Individuals are exposed to multiple environmental stressors simultaneously, including air pollution, noise, chemical contaminants, plastics, and climate-related stressors, especially in the urban setting. These exposures may interact through shared biological pathways, leading to additive or synergistic effects on cardiovascular risk.”

And while substantial uncertainties remain, “the convergence of epidemiological, clinical, and mechanistic evidence suggests that plastic pollution may represent a previously underestimated cardiovascular risk factor,” Daiber et al. continued. “Addressing this challenge will require coordinated efforts across disciplines and policy domains. In the era of the Anthropocene, protecting cardiovascular health will increasingly depend on reducing not only traditional risk factors but also the growing burden of environmental pollutants (the detrimental part of the exposome), among which plastics may soon play a central role.”

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Chronic Pancreatitis Therapies Informed by Patient-Derived Organoids

Edusehat — Thu, 16 Jul 2026 02:00:13 +0700

Approximately three million people worldwide struggle with chronic pancreatitis, for which there is no cure. In a study published in Cell Stem Cell titled “Patient-derived organoids reveal ductal dysfunction and CFTR-modulator responses in chronic pancreatitis,” researchers from Salk Institute have developed an organoid platform to uncover the mechanism of chronic pancreatitis development and identify possible therapeutic strategies.

The authors generated 37 organoids from patients who developed chronic pancreatitis spontaneously. The organoids revealed consistent dysfunction in the protein cystic fibrosis transmembrane conductance regulator (CFTR), which was identified as a therapeutic target.

“Though patients can have the same clinical diagnosis of chronic pancreatitis, they can have very different underlying molecular drivers of that disease, which makes treatment especially difficult,” said Dannielle Engle, PhD, assistant professor at Salk and corresponding author of the study. “Our work breaks down a major barrier in the field by establishing an experimental model that preserves patient-specific disease biology and can be used to develop tailored therapies.”

Over the last decade, organoids have become a prevalent tool to bridge the gap between cell and human studies. Each organoid typically begins with stem or progenitor cells from patients. In Engle’s lab, donor pancreas tissues were used to create miniature replicas of the pancreas. Findings based on a patient’s personalized organoid model could improve therapeutic effectiveness.

“By growing organoids directly from patients, we preserve key features of ductal cells and ask which disease mechanisms are active in each individual patient,” said Victoria Osorio-Vasquez, PhD, a postdoctoral researcher in Engle’s lab and first author of the study.

The researchers surveyed the molecular signatures in each organoid and found three subtypes of chronic pancreatitis. This biology-based patient stratification can inform optimal treatment. Results showed that approximately half of the organoids demonstrated dysfunctional CFTR.

“And CFTR dysfunction was not limited to patients with inherited CFTR mutations, suggesting that functional testing may identify therapeutic opportunities that would be missed by genetic testing alone,” Osorio-Vasquez says.

Existing CFTR modulator therapies treat patients with cystic fibrosis. The findings suggest that these same therapies may offer pancreatic benefits. The researchers tested clinically available CFTR modulators and found that these therapies could stabilize or restore CFTR function and reduce inflammatory signaling in responsive pancreas organoids.

The platform also revealed rare alterations to genes, KRAS and TP53, in some chronic pancreatitis organoids, supporting future use of the system to study disease evolution, pancreatic cancer risk, and biomarker discovery at the interface of chronic inflammation and pancreatic cancer.

“These organoids gave us a way to study chronic pancreatitis pathogenesis in human cells for the first time,” says Engle. “Our platform enables a more personalized way of studying and eventually treating chronic pancreatitis, while also blazing the trail for other organoid-based platforms in other inflammatory disease contexts.”

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In vivo CAR T Industry Leaps Forward with Challenges Ahead

Edusehat — Thu, 16 Jul 2026 02:00:11 +0700

In vivo CAR T sounds like the existing class of Chimeric Antigen Receptor (CAR) T-cell therapies used to provide often individualized treatment for cancer. But they’re a new and emerging class of therapeutics with their own challenges and opportunities for manufacturers.

That’s according to Mo Heidaran, PhD, chief scientist at Cellx, who is due to give a talk at the upcoming Bioprocessing Summit in Boston.

“Whether something is a cell or gene therapy, from a regulatory perspective, depends on [the nature of] the product that’s administered to the patient,” Heidaran explains.

“And, in the United States, in vivo CAR Ts are gene therapy products and not cell therapies as some people talk about them.”

The better-known CAR T products are ex vivo, delivered via modification of patient cells, he explains. Whereas this emerging class of therapies involves delivery of a genetically engineered virus or lipid nanoparticle (LNP) that, in some cases, is stably integrated into the patient genome.

According to Heidaran, the risk of integration is higher when viruses are used.

“My colleagues at the FDA want to make sure people understand it’s very important these products must be [designed] to be very specific to the cell type, perhaps based on data about [some of these] therapies having off-target effects,” he says.

Most in vivo CAR T-cell therapies are in very early stages, with none currently approved for patients, although Heidaran says they are increasingly under investigation by larger companies since they are scalable for a wider range of patients. Also, they are believed to be more cost-effective and have similar logistics, as they don’t require lymphodepletion, he adds.

“Essentially the value driver is that you’re pharmaceuticalizing cell and gene therapy since it’s just a vial of the virus or LNP that you can use to treat many patients—almost like a drug or pill,” he says.

Among the challenges for this emerging class is that several ex vivo CAR T-cell therapies are already approved for patients. In vivo CAR T therapies treat some of the same indications, i.e., certain cancers and autoimmune diseases, he says.

“At some point there has to be a decision made by the FDA about how these [new] therapies compare, such as [running] a study or external control as to whether they’re superior or non-inferior to the same or similar approved ex vivo CAR T,” he says.

Other challenges facing this new industry are about batch sizes for manufacturing, as the equipment and processes for treating ten patients are different from needing to treat thousands. Also, he says, in vivo CAR T therapies need to be monitored to look for off-target effects, durability of response, or an immune response by the patient.

“Overall, to develop a safety profile, we need to define what the effective dose is that people are working to, as these therapies may require repeat administration, which is not done with ex vivo-generated CAR T,” he says.

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AI Could Give CGT Sector Deeper Manufacturing Insights and Greater Control

Edusehat — Thu, 16 Jul 2026 02:00:10 +0700

AI could help cell and gene therapy manufacturers gain a deeper understanding of the complex production processes used to make their products and predict problems before they occur.

A team led by researchers at Northeastern University College of Science in Boston made the case for AI use in a recent paper, arguing that the variability inherent in cell and gene therapy production can be difficult to manage using conventional tech.

Lead author, Jared Auclair, PhD, dean of the College of Professional Studies at Northeastern, tells GEN, “Unlike monoclonal antibodies or recombinant proteins, cell and gene therapies are living or highly complex biological products, making them inherently more variable and difficult to manufacture consistently.

“Every step, from sourcing starting material to manufacturing, analytical testing, storage, and delivery, can influence the final product,” he adds.

Understanding complex, multi-parameter interactions is exactly the sort of challenge at which AI excels, Auclair says, citing the ability to identify critical process attributes as an example.

“AI has the potential to transform cell and gene therapy manufacturing by moving from reactive to predictive manufacturing. Machine learning can optimize process parameters, predict batch failures before they occur, enable digital twins to simulate manufacturing changes, and strengthen quality control through real-time monitoring and anomaly detection,” he adds.

“At Northeastern, our research at the intersection of the Bioanalytical Training Laboratory (BATL), the Center for Bioinnovation and Regulatory Sciences, and AI is exploring how AI can accelerate the development, manufacturing, and regulation of advanced therapies,” Auclair says.

Not plug-and-play

AI’s potential to spot patterns in data is attractive.

However, biopharmaceutical companies looking to adopt the technology are likely to encounter challenges, according to Auclair, who cautions that setting up an AI-driven manufacturing operation is about more than simply buying the right software.

“The technology is advancing rapidly, but successful implementation depends on having high-quality, well-curated data, digital manufacturing infrastructure, and multidisciplinary expertise spanning biology, engineering, data science, and regulatory science.

“AI is not a plug-and-play solution; organizations must build integrated data ecosystems and governance frameworks that regulators can trust,” Auclair says.

AI adoption is a multidisciplinary challenge and should involve people with expertise in all parts of drug development and production, according to study co-author Rominder Singh, PhD, professor of practice, regulatory sciences, & AI at Northeastern.

“Research conducted through the BATL and the Center for Bioinnovation, led by Professor Auclair, has focused on addressing many of these scientific and manufacturing challenges that are unique to advanced therapies.

“This is precisely why Northeastern’s pioneering work in RegSciAI is so important: it brings together regulatory science and AI to ensure these technologies are both innovative and deployable in real-world biomanufacturing,” Singh says.

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Free-Floating Bioelectronic Sensors for Fermentation Monitoring

Edusehat — Thu, 16 Jul 2026 02:00:09 +0700

The static, mounted monitoring systems currently used inside fermentation vats are poised to be replaced in the near future with a network of free-floating bioelectronic sensors, if the vision of researchers from Boston University and Capra Biosciences reaches fruition.

Designed for both vat and continuous bioprocessing systems, this bioelectronic sensor network could, ideally “provide spatial information about where they are in a heterogeneous bioreactor platform…as well as multiple measurements of things such as temperature, pH, dissolved oxygen, and dissolved carbon dioxide,” Rabia Yazicigil, PhD, associate professor, Boston University (BU), and lead principal investigator for this project, tells GEN.

Consequently, the network will report data that enable biomanufacturers to determine whether the solution is mixing properly and to identify transit times throughout the process, in addition to specific processing parameters.

“The key innovation…is that these systems integrate living cells into the electronics,” Miguel Jimenez, PhD, assistant professor, BU, emphasizes. The inclusion of microbes—bacteria or yeast cells, for example—“supercharges the sensors,” enabling them to monitor more parameters that are directly relevant to biomanufacturing.

Roughly the size of a chickpea, these sensors never leave the bioreactor. “That allows us to get measurements throughout the reactor… which helps us build a really rich data set that we can then feed into models to help us monitor and predict performance,” notes Mark Poole, PhD, senior director of manufacturing and applied AI, Capra Biosciences.

Paradigm-shifting potential

“Having lots of high-quality measurements at different points in the reactor is game-changing for any biomanufacturing company,” Poole says.

Jon Valdez, program manager at BioMADE, which funded the project as part of a $21.4 million investment in 14 projects to advance the bioindustrial manufacturing industry, agrees, calling it potentially paradigm-shifting. Potential applications extend to clinical monitoring—where a prior collaboration focused on human gut monitoring. The technology is solvent-agnostic but may be most effective in a water-based environment, enabling applications that may include soil and water quality monitoring. Benefits, he says, include lower costs per sensor (estimated at $10−$100) and decreased risk of contamination.

The project is two-tiered. The first tier, the electronics-only sensor, is the nearest to commercialization. Industrial-scale testing will be conducted soon at Capra facilities. “That [alone] would signify a big advance,” Jimenez says, citing the ability to field networked sensors capable of measuring multiple conditions throughout a bioreactor or continuous production process.

The second tier adds the bio component to those sensors. This feature is in academic development. Primary challenges are how to design biohybrid sensors that can be autoclaved or cleaned-in-place, and strategies to stabilize and encapsulate the microbes to be compatible with industrial requirements. The researchers are considering possible approaches now.

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Modular Modeling Drives Smarter mRNA Manufacturing

Edusehat — Thu, 16 Jul 2026 02:00:08 +0700

Researchers are increasingly turning to modular mechanistic models to unlock greater efficiency and robustness in mRNA manufacturing, offering a more flexible way to optimize in vitro transcription (IVT) while reducing costly experimental work. According to Wei Xie, PhD, associate professor of mechanical and industrial engineering at Northeastern University, and her colleagues, modular approaches can increase productivity and product quality.

“A modular modeling approach simplifies the complex IVT reaction network by dividing it into discrete, reusable, mechanistically defined steps,” Xie said. “This structure improves mechanistic understanding by clarifying how each step impacts key quality attributes, including yield, capping efficiency, and transcript integrity.”

Rather than relying on a single monolithic model, the framework separates IVT into individual components, such as initiation, elongation, and termination, as well as parallel processes including mRNA degradation and precipitation. Each module can be independently calibrated, validated, and refined as new experimental data become available, allowing researchers to continuously improve predictive performance without rebuilding the entire model.

The modular architecture also lends itself to the evolving nature of mRNA therapeutics. Because the framework mirrors the modular structure of nucleic-acid sequences, it can be rapidly adapted for new constructs, accelerating process development for emerging vaccines and therapeutic candidates while minimizing redevelopment effort. Beyond improving process understanding, the model provides a powerful diagnostic platform for identifying production bottlenecks that constrain yield, productivity, or product quality.

The framework combines Shapley value-based sensitivity analysis, residual analysis, and simulated reaction trajectories to pinpoint limiting process variables. Sensitivity analysis identifies parameters with the greatest influence on performance, while comparisons between predicted and experimental results reveal missing mechanisms or model deficiencies. Simulated reaction profiles can also highlight issues such as nucleotide depletion or suboptimal magnesium-to-nucleotide ratios before they become significant manufacturing challenges.

“Together, these tools provide a data-driven, mechanistic approach to quickly diagnose constraints and guide targeted process optimization,” Xie explains.

The approach also offers significant advantages during scale-up, one of the most challenging phases of bioprocess development. Because the model is grounded in fundamental molecular reactions and biochemical mechanisms rather than empirical correlations, it maintains predictive capability across different manufacturing scales and can be readily applied to new mRNA sequences, all without extensive redevelopment.

Xie says the framework supports predictive design of scale-dependent control strategies, including dynamic pH regulation and fed-batch nucleotide feeding schemes, helping manufacturers reduce development timelines while improving process robustness during technology transfer.

“A key advantage of the modular architecture is its flexibility and interoperability” Xie says. “New enzymes, reagents, or process steps can be incorporated by simply updating or adding the relevant module, without recalibrating the entire model. The framework’s ability to accommodate heterogeneous datasets generated under varying process conditions further supports rapid evaluation of manufacturing innovations while maintaining model consistency.

Perhaps the greatest impact of Xie’s approach lies in advancing quality-by-design (QbD). Acting as an in silico development platform, the modular model enables researchers to evaluate process variables before entering the laboratory. Coupled with digital twin-based Bayesian optimization, the platform narrows the experimental search space, reducing trial-and-error studies while conserving expensive reagents, such as T7 RNA polymerase.

As mRNA pipelines continue to expand beyond vaccines into broader therapeutic applications, modular mechanistic modeling is emerging as a valuable digital bioprocessing tool, enabling manufacturers to accelerate development, strengthen process understanding, and deliver more consistent product quality with fewer experimental resources.

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Cellares and Sonoma Biotherapeutics Agree to Automate Engineered Treg Cell Therapy Manufacturing

Edusehat — Wed, 15 Jul 2026 15:10:18 +0700

Officials at Cellares and Sonoma Biotherapeutics say their companies will automate the manufacturing of SBT-77-7101, an engineered regulatory T cell (Treg) therapy for autoimmune and inflammatory diseases. The product is in Phase I clinical development for poly-refractory rheumatoid arthritis (RA) in patients who have exhausted all available treatment options.

Cellares plans to translate SonomaBio’s SBT-77-7101 manufacturing process onto its Cell Shuttle and automate in-process and release testing through the Cell Q quality control system. The company operates its first commercial-scale Smart Factory in Bridgewater, NJ, with additional facilities under construction in Europe and Japan.

“Tregs are uniquely sensitive to the manufacturing process. Cellares brings the Cell Shuttle platform, and global infrastructure to help us deliver on our clinical ambitions at scale for the hardest-to-treat RA patients.” said Stephen Dilly, PhD, president, CEO, and board chair of Sonoma Biotherapeutics.

“Every new cell therapy modality we bring to the Cell Shuttle and Cell Q expands what is possible for the field and for patients in need,” added Fabian Gerlinghaus, co-founder and CEO of Cellares. “Tregs are among the most technically demanding cell types to manufacture reliably. We are honored to partner with SonomaBio and demonstrate that our platform can directly translate to Tregs.

“SonomaBio has developed one of the most advanced Treg programs in the clinic, and we look forward to contributing to their clinical success as they bring this groundbreaking therapy to patients.”

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FDA Issues Proposal to Streamline Registration for Advanced Distributed Manufacturers

Edusehat — Wed, 15 Jul 2026 15:10:16 +0700

The FDA states that its newly proposed rule, if finalized, would create a streamlined registration pathway for distributed manufacturing establishments that operate as a single establishment using a “hub-and-spoke” model. The proposed rule would also clarify registration requirements for certain foreign establishments that manufacture drugs, including active pharmaceutical ingredients (APIs), that indirectly enter the U.S. drug supply.

This action is another step forward in the FDA’s coordinated effort to help ensure Americans have reliable access to safe, quality medicines by strengthening domestic pharmaceutical manufacturing, and ensuring that regulatory frameworks keep pace with innovation, according to agency officials.

Distributed manufacturing establishments operate using a hub-and-spoke model, with a central quality oversight hub and multiple equivalent manufacturing units at different locations. Currently, regulations require each manufacturing unit in such a network to register separately, creating unnecessary administrative burdens.

Under the proposed rule, distributed manufacturing establishments could register as a single establishment. Units could be added, relocated, or removed through a streamlined update process, and companies would be required to notify the FDA in advance of any unit relocation, closing a gap in the agency’s real-time oversight.

The proposed rule would also clarify registration and drug listing requirements for certain foreign drug manufacturing establishments. [Reptile8488/Getty Images]

“The FDA is proposing changes to our establishment registration regulations that would reflect how distributed manufacturing actually works—as one single establishment,” said Michael Davis, MD, PhD, acting director of FDA’s Center for Drug Evaluation and Research (CDER). “The proposed changes would make it easier for innovative manufacturers to operate efficiently, and give the FDA a clearer, more accurate picture of how and where drugs are being made.”

The proposed rule would also clarify registration and drug listing requirements for certain foreign drug manufacturing establishments. Currently, some foreign establishments that manufacture drugs (including components of drugs, such as APIs) only for distribution to other foreign establishments may not be registered with the FDA, limiting the agency’s visibility into upstream supply chains.

By aligning the agency’s regulations to the statutory requirements, this proposed rule would make it clearer that these establishments must register with the FDA and report on the drugs they produce, giving the FDA greater ability to detect and respond to potential safety concerns.

“When an active ingredient in a medicine reaches an American patient, the FDA should be able to trace exactly where it came from,” added Davis. “Closing this registration gap for foreign establishments is a concrete step toward increasing the supply chain transparency that patients deserve.”

If finalized, the proposed rule is expected to reduce registration costs for distributed manufacturing companies and generate long-term efficiencies for both industry and the agency. It builds on a series of administration actions aimed at revitalizing American pharmaceutical manufacturing, improving supply chain transparency, and reducing vulnerabilities in the drug supply chain, noted a spokesperson for the FDA.

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Novel Epigenetic Therapy Targets Treatment-Resistant and TP53-Mutant AML

Edusehat — Wed, 15 Jul 2026 15:10:15 +0700

The results of a preclinical study by researchers at the University of Texas MD Anderson Cancer Center have found that an investigational epigenetic therapy called NTX-301 remained effective in treatment-resistant acute myeloid leukemia (AML) by activating the Hippo pathway, a tumor-suppressing pathway linked to cancer growth and drug resistance.

In preclinical models, the hypomethylating agent (HMA) NTX-301 was more effective than standard hypomethylating agent therapy and retained anti-leukemia activity in treatment-resistant and TP53-mutant AML. The team also found that the therapy activated the Hippo pathway through targeted epigenetic changes, revealing a previously unrecognized mechanism that may contribute to its anti-leukemia effects.

“Leukemia cells are remarkably adaptable and often find new pathways to survive after treatment,” said Michael Andreeff, MD, PhD, professor of medicine at the University of Texas MD Anderson Cancer Center and research co-lead. “These findings suggest NTX-301 may disrupt several of those survival mechanisms simultaneously while reactivating pathways that normally restrain cell growth. That dual effect could help explain why NTX-301 remained active in some of the most therapy-resistant forms of AML.”

The findings suggest a potential new strategy for patients whose disease relapses after frontline therapy, including those with TP53 mutations, one of the highest-risk forms of AML. Andreeff, together with leukemia professor and study co-lead Bing Z. Carter, PhD, and colleagues, reported on their studies in Clinical Cancer Research, in a paper titled “The novel hypomethylating agent NTX-301 reprograms epigenetic and Hippo signaling pathways and exhibits preclinical activity in venetoclax-resistant and TP53-mutant AML.”

First-generation hypomethylating agents, including 5-azacytidine (5-AZA) and decitabine (DAC), are used as standard clinical care for patients with AML and myeloid dysplastic syndromes (MDS), the authors wrote. Combining HMAs with the BCL-2 inhibitor venetoclax has further improved outcomes for patients.

“Hypomethylating agent (HMA) and the BCL-2 inhibitor venetoclax (VEN) combinations have evolved into frontline therapies for patients with acute myeloid leukemia (AML), yielding high response rates,” they stated. However, while such combination therapy works well initially, resistance and relapse remain common.

The challenge is particularly significant in AML with mutations in the TP53 gene, which normally helps cells respond to damage and prevent uncontrolled growth. When that gene is mutated, leukemia cells can become resistant to therapy and more difficult to eliminate. “… most patients ultimately relapse, particularly those with TP53 mutations,” the researchers continued.

Efforts have been made to develop improved and more effective HMAs, they noted, and NTX-301 is such a next-generation HMA. But as they pointed out, “… previous reports of NTX-301 preclinical studies in leukemia were conducted primarily in cell lines and xenograft models … its activities in therapy-resistant settings have not been investigated.” And while a Phase I study (NCT04167917) of the oral agent NTX-301in patients with AML and MDS has been completed, the team noted in their paper that the study has not yet been reported.

For their newly reported preclinical study, the researchers evaluated NTX-301 across multiple preclinical models of treatment-resistant AML, including patient-derived xenograft (PDX) models of AML with acquired resistance. Their results showed that NTX-301 therapy consistently reduced leukemia cell survival more effectively than azacitidine (AZA), a commonly used hypomethylating agent.

Importantly, NTX-301 remained active in leukemia cells that had already developed resistance to both hypomethylating therapy and venetoclax, and demonstrated anti-leukemia activity in TP53-mutant AML models. When combined with venetoclax in resistant leukemia samples, NTX-301 produced stronger anti-leukemia effects than either treatment alone. The combination was effective not only against leukemia blasts but also against leukemia stem and progenitor cells, which are believed to contribute to disease persistence and relapse.

In summary, they wrote, “Therapeutically, NTX-301 is more potent than 5-AZA in AML cells with various genetic backgrounds, is active in AML cells with acquired resistance to HMA or VEN, overexpressing VEN-resistant factors MCL-1 or BCL-2A1, and in isogenic AML cells with TP53 deletions/mutations in vitro and in vivo in xenograft models, exhibits activities against AML blasts and stem/progenitor cells from patients resistant to/relapsed from VEN-based therapies and with TP53 mutations in vitro and in vivo VEN/DAC-resistant PDX models, and enhances VEN activity.”

To understand why NTX-301 appeared more effective than existing drugs, researchers analyzed changes in DNA methylation, a process that can switch genes on or off without altering the underlying genetic code. Unlike current hypomethylating therapies, which broadly affect DNA methylation, NTX-301 focused on a more selective set of genes and pathways, including the Hippo pathway, which functions as a natural cell growth regulator.

NTX-301 increased activity of key Hippo pathway genes while reducing activity of YAP, a protein frequently linked to cancer cell survival, treatment resistance, and stemness. These findings suggest Hippo pathway reactivation may be an important reason the therapy remained effective in resistant leukemia models and could represent a new strategy for overcoming treatment resistance in AML. “Collectively, our data suggest that NTX-301 exhibits more potent anti-leukemia activities compared to current HMAs and synergizes with VEN in VEN-resistant and TP53-mutant AML and AML stem/progenitor cells,” the team concluded.

Additional studies are needed to determine whether these results translate to patients and to identify which populations may benefit most. The findings suggest that patients with relapsed AML, venetoclax-resistant disease, and TP53 mutations may be important groups for future clinical evaluation. “Taken together, the numerous NTX-301 targets identified here, its novel mechanism of action, and its superior activity against VEN-resistant and TP53-mutant AML compared to 5-AZA, warrant the future clinical development,” the investigators noted. “Given the strong preclinical data in TP53-mutant AML and the unmet clinical need, this should be a primary target group in the next clinical trial.”

Carter said, “An encouraging aspect of this study is that it identified both a potential therapeutic opportunity and a biological explanation for why it may be effective. The results provide a rationale for continued clinical development and suggest that targeting Hippo signaling may help address treatment resistance in AML.”

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Honoring the Innovators Driving AI’s Next Era in Life Sciences and Healthcare

Edusehat — Wed, 15 Jul 2026 15:10:14 +0700

Nebius, an AI cloud company, sponsored its second “AI Discovery Awards” event and dinner earlier this month in London, where the winning companies were announced. The event highlighted leading startups in biopharma, genomics, medical devices, and digital health that are using AI to deliver advances in healthcare and life sciences.

During the evening awards ceremony, artificial intelligence demonstrated that it is rapidly reshaping biomedical research. However, practitioners agree that AI’s success depends on more than advanced algorithms. [Nebius]

At a Nebius-hosted morning discussion before the awards dinner, several researchers highlighted the importance of powerful computing infrastructure, high-quality biological data, and laboratory validation.

Examples included AI models that predict osteoarthritis years before symptoms and an Alzheimer’s platform, which achieved 97% diagnostic accuracy when paired with protein biomarkers. A Stanford Medicine scientist described CRISPR-GPT, an AI assistant that helps design and troubleshoot gene editing.

The investigators also spotlighted AI-powered lab automation, multimodal datasets, and AlphaFold’s dramatic acceleration of protein structure prediction. Across every application, participants emphasized that collaboration among academia, healthcare, industry, and governments will be essential to advance preventive, personalized medicine and to translate AI discoveries into clinical practice.

Ilya Burkov, PhD, who has a background in clinical medicine, is now global head of healthcare and life science at Nebius. Burkov began his research career focusing on osteoarthritis, osteoporosis, hip and knee replacements, and trying to figure out how such diseases develop and progress.

“My goal was to work backward from the end stage of disease and determine whether we could predict who was at risk years before serious joint damage occurred,” he explained. “If we could identify those patients early enough, perhaps we could delay disease progression.”

Machine learning

Speaking with colleagues in a hospital, he was asked: “Have you looked at it from any machine learning perspective?” Burkov had no formal background in artificial intelligence, but he was intrigued by the idea of using emerging AI models and advanced algorithms to analyze long-term medical imaging data.

Ilya Burkov, PhD [Nebius]

The concept was simple but powerful: if AI could identify patterns shared by patients who later developed osteoarthritis or osteoporosis, it might be able to detect subtle biomarkers long before the disease became clinically apparent.

“That idea became the focus of my PhD research. AI models were not a thing ten years ago when I was in the hospital. There were transformer models and algorithmic-based approaches.

“I developed techniques capable of predicting the early onset of osteoarthritis and osteoporosis with an accuracy of roughly 80% to 90%,” he pointed out. “The models identified imaging features that consistently appeared years before patients required joint replacement surgery.

“This made it possible to examine scans from otherwise healthy individuals and estimate their future risk. In some cases, we could tell patients that, without changes to certain lifestyle factors, they had a high probability of requiring a hip replacement within the next 10 to 15 years.”

For Burkov, that was transformative. AI made it possible to move beyond treating individual patients and instead create tools that could benefit entire healthcare systems. Rather than applying clinical expertise one patient at a time, scalable technologies could be created capable of helping clinicians identify high-risk patients earlier and intervening before irreversible damage occurred.

That realization ultimately convinced him to transition from clinical medicine into industry, where he saw the opportunity to build technologies that could have a much broader impact. Whether it’s a small academic lab with only a handful of researchers or a global pharmaceutical company operating at massive scale, every organization faces different computational challenges.

“At Nebius, our role is to provide the computing infrastructure and technology that enables researchers to train increasingly sophisticated AI models and accelerate scientific discovery to advance biomedical research and improve patient care,” he said.

Alzheimer’s disease

Artificial intelligence is rapidly reshaping drug discovery, but many researchers believe the greatest challenge is not designing drugs—it’s knowing what biological targets to pursue.

Prima Mente, a previous AI Discovery Award winner, is tackling that problem by building foundation AI models designed to uncover the molecular mechanisms behind Alzheimer’s disease and other neurodegenerative disorders. By combining blood-based biomarkers, multimodal biological data, and transformer-based AI, the London startup hopes to identify the molecular drivers of neurodegenerative disease—and ultimately accelerate the development of new therapies.

“If we can diagnose disease earlier, better stratify patients, and understand what’s actually driving Alzheimer’s, we can help create better treatments,” said co-founder Hannah Madan, PhD.

Based in London’s King’s Cross innovation district, Prima Mente has grown to approximately 35 employees across London, San Francisco, and the United Arab Emirates. Madan, whose academic background includes a master’s degree in pharmacology and a PhD investigating the relationship between bowel cancer and diabetes, has spent most of her career building biotechnology startups. Prima Mente is the fifth company she has helped launch.

The company’s mission addresses one of healthcare’s most pressing challenges. Dementia is the leading cause of death in the U.K. and the sixth highest in the U.S. Alzheimer’s disease remains the most common form of dementia worldwide.

Looking beyond traditional biomarkers

Prima Mente’s scientific strategy draws inspiration from advances in cancer diagnostics, particularly liquid biopsy technologies that detect circulating tumor DNA in blood samples. The company wondered whether a similar approach could work for neurodegenerative disease.

“When we started three years ago, many people thought we were a little crazy,” noted Madan. “The prevailing view was that very little DNA from dying brain cells entered the bloodstream.”

Hannah Madan, PhD, co-founder, Prima Mente [Nebius]

The team has since demonstrated that cell-free DNA originating from neurons, microglia, and astrocytes can be detected in blood. More importantly, those DNA fragments retain epigenetic information that may reveal the biological state of brain cells before they died.

Rather than focusing solely on DNA sequences, Prima Mente analyzes methylation patterns carried on cell-free DNA. Because methylation reflects how genes are regulated within specific cell types, these signals can provide insight into disease progression and cellular dysfunction.

“When cells die, they release fragmented DNA into the bloodstream,” Madan explained. “Those fragments preserve methylation signatures that tell us what state those brain cells were in.”

The biological strategy is paired with an equally ambitious computational one. Prima Mente believes that transformer architectures—the same AI technology underlying large language models such as ChatGPT—can learn the language of biology.

“If ChatGPT can understand human language, our hypothesis is that similar models can understand biological languages,” noted Madan.

Instead of converting sequencing data into simplified numerical counts, the company trains models directly on raw biological sequences, including DNA, methylation signals, RNA transcripts, and proteomic data. By preserving more of the underlying biological information, Prima Mente believes its models could uncover relationships that conventional bioinformatics pipelines often overlook.

The company’s first foundation model, known as Pleiades 1, demonstrated the potential of that approach. Initially trained to identify Alzheimer’s disease from blood-derived molecular data, the model successfully diagnosed a subset of patients. After protein biomarkers were incorporated, diagnostic accuracy increased to approximately 97% within the study dataset—exceeding the performance of current clinical standards, according to Madan.

AI tokens are the fundamental units of data processed by AI models during training and inference. They represent smaller components of text, audio, images, or other modalities, enabling models to understand, predict, and generate outputs effectively. Pleiades 1 was trained on 1.9 trillion tokens. Its successor, Pleiades 2, is being trained on 80 trillion tokens spanning five biological data modalities, with the long-term goal of building a 100-billion-parameter foundation model.

Prima Mente partnered with AI infrastructure provider Nebius, which supplied a dedicated 32-node computing cluster powered by NVIDIA GPUs. The additional computing capacity enabled the company to scale from a 1-billion-parameter model to a 10-billion-parameter model within weeks while increasing training throughput from roughly 8,000 tokens per second per device to more than 1.1 million tokens per second across a 16-node cluster.

Beyond model development, Prima Mente is collaborating with the U.K.’s National Health Service (NHS) through the Sandbox Study, which collects blood samples from patients with suspected neurological disease. The real-world data help researchers develop AI models aimed at detecting Alzheimer’s earlier, potentially enabling treatment before irreversible brain damage occurs.

Dementia is the leading cause of death in the U.K. and the sixth highest in the U.S. Alzheimer’s disease remains the most common form of dementia worldwide. [Cecille Arcurs/Getty Images]

Unlike AI companies that rely primarily on public datasets, Madan said Prima Mente is generating much of its own training data. The company collaborates with 20 NHS memory clinics throughout the U.K., collecting blood samples, speech recordings, clinical notes, and imaging data from patients at the earliest stages of cognitive decline. It also participates in the U.K.’s Sovereign AI initiative.

Lab validation is integrated into the company’s development process. Candidate discoveries generated by AI models are tested using stem cell systems, brain tissue, and additional blood-based experiments, creating a continuous feedback loop between computational prediction and experimental validation.

That combination of proprietary data generation, lab experimentation, and AI model development represents what the company views as a significant competitive advantage.

While AI has attracted enormous attention for accelerating drug discovery, Madan argues that identifying the right biological target remains the industry’s greatest bottleneck. She compares today’s AI revolution to the impact AlphaFold had on protein structure prediction. As computational tools become increasingly capable, designing drug candidates may become faster, cheaper, and more routine.

“But if you don’t know what biology actually matters,” she said, “there’s little value in having better tools to build drugs.”

For Prima Mente, Madan says the opportunity lies upstream of drug development—discovering the cellular pathways, biomarkers, and molecular mechanisms that should become tomorrow’s therapeutic targets.

That strategy recently received external validation when the company won the AI Insights Prize for Alzheimer’s from the Gates Foundation, receiving $1 million to expand research into microglial biology. The funding will support AI models designed to identify gene perturbations in specific brain cell types that could serve as the basis for future Alzheimer’s therapies.

As foundation models continue to expand beyond language into biology, Madan is betting that the next major AI breakthrough in medicine will not simply generate better drugs—it will reveal entirely new biology that makes those drugs possible.

CRISPR-GPT

CRISPR-GPT is a large language model developed by Stanford Medicine to automate key steps in CRISPR gene-editing research. Acting as an AI agent, it interprets scientific literature, designs guide RNAs, suggests experimental parameters, and integrates with lab automation systems to execute and refine experiments. By reducing manual planning and accelerating iterative testing, the system enables researchers to complete complex gene-editing workflows more efficiently and consistently, noted Stanford researchers.

CRISPR-GPT is also credited with lowering the barrier for scientists with limited CRISPR expertise, improving accessibility. The platform represents an emerging class of AI tools that can “reason” through complex scientific tasks, recommend next steps, and accelerate discovery. Potential applications include developing gene therapies, improving cancer research, engineering cell therapies, and expanding access to genome-editing technologies.

The goal, according to Le Cong, PhD, assistant professor of pathology and genetics is to help scientists produce life-saving drugs faster. “The hope is that CRISPR-GPT will help us develop new drugs in months instead of years,” he said.

Le Cong, PhD [Stanford Medicine]

Cong and team developed CRISPR-GPT using Nebius AI Cloud as its core infrastructure. The group leveraged Nebius’ GPU clusters to train their specialized CRISPR-Llama3 model, rapidly iterate on architectures, and scale from prototyping to full model training.

CHAT-GPT could also expand the pool of scientists who can effectively use gene editing technology—no experience required, pointed out Cong. “Trial and error is often the central theme of training in science, but what if it could just be trial and done?” he added. Cong is the senior author of a study “CRISPR-GPT for agentic automation of gene-editing experiments,” published July 2025, in Nature Biomedical Engineering.

AI Discovery Awards

At the AI Discovery Awards dinner in the evening, the sponsors announced that the 2026 program added medical devices and medical imaging to the existing biopharma, genomics, and digital health tracks to reflect the growing role of AI in connected medical equipment and diagnostic imaging.

“Our winners—and indeed all of the 647 submissions we reviewed—reflect how rapidly AI is changing the pace of healthcare research,” said Ilya Burkov during a short presentation. “Across all categories, startups are compressing timelines that once took years into months or even weeks, and bringing capabilities to clinical and laboratory settings that simply did not exist before.

Margaret Hua, founding chief of staff at Phylo, accepts $100,000 in GPU cloud credits for first prize in the biopharma category. The company is building AI research assistants that can independently help biomedical scientists think through problems, design experiments, analyze data, and suggest what to do next, with the aim of speeding up scientific and biomedical discovery. [Nebius]

“The AI Discovery Awards exist to accelerate that momentum, and to connect the most promising teams with the compute resources, investor networks, and mentorship they need to move from promising research to bringing products to market.”

Alongside the awards program, Nebius previewed its Nebius Scientific AI and Healthcare Platform, which is an AI infrastructure built to meet the specialist needs of healthcare and life sciences organizations, explained a Nebius official.

The 2026 AI Discovery Awards were open to companies from pre-seed through to Series D that put AI and machine learning at the core of their product. Category winners were selected from 647 applications from around the world by an independent panel of 28 judges representing leading pharmaceutical companies, academic institutions, and venture capital firms. Submissions were evaluated based on the use of AI within the product, use of compute, technical innovation, functionality and advantages, performance and efficiency, global impact, market potential, and business sustainability.

A full list of shortlisted companies, as well as qualification criteria and a jury list, can be found on Nebius’s website.

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Protein Design’s AI Revolution: Inside David Baker’s “Communal Brain”

Edusehat — Wed, 15 Jul 2026 15:10:13 +0700

“I have this idea of a communal brain.” David Baker, PhD, told me as I sat in his office at the University of Washington (UW) surrounded by colorful and complex figurines of protein structures. It was the one-year anniversary of his Nobel Prize in Chemistry win.

Just outside his doors, a lab of more than 100 researchers was united by the shared ambition to design proteins from scratch (or de novo) for powerful applications across pharmaceuticals, vaccines, biosensors, and more. This “communal brain” housed at the UW Institute for Protein Design (IPD), where Baker led as director, was hard at work developing deep learning methods that could achieve atomic precision.

A small protein composed of 100 amino acids had an astronomical 20¹⁰⁰ possible sequences. Yet, only a vanishingly tiny fraction could fold into stable, functional structures. Misplacing a residue by an angstrom could mean the difference between a drug binding tightly to its target or complete failure.

For the antibody drug market worth hundreds of billions of dollars, Nathaniel Bennett, PhD, former postdoctoral researcher in the Baker lab, says AI-guided antibody design that bypasses the need for time-consuming experimental screens has long been a “holy grail” for a breadth of indications, including cancer and autoimmune disease.

Last November, Bennett and colleagues published a Nature paper demonstrating that full length de novo antibodies could bind user-specified epitopes. AI models could now construct antibody loops, the key region involved in binding that has been historically challenging to design due to its flexible nature.

Despite this technological leap, AI-designed proteins that were manufacturable, remained stable in the body, and avoided unwanted side effects, were still a step away. The gap fueled an industry debate over whether generating de novo medicines was even possible.

When I asked Baker to separate the hype from reality, he didn’t hesitate.

“The reality is that we can now design proteins on a computer,” Baker explained in our video interview. “The hype is that for therapeutics, there’s a lot more than the basic activity of a protein binding or catalyzing a reaction. Whether de novo proteins will revolutionize medicine will require improving our understanding of the biology.”

Nobel guests

Bennett is continuing molecular design research as a co-founder at Xaira Therapeutics. The AI-focused biotech launched in 2024 with over $1 billion in total funding and a star-studded leadership team, including Baker, as a scientific advisor, and Marc Tessier-Lavigne, PhD, former president of Stanford and CSO of Genentech, as CEO. Carolyn Bertozzi, PhD, Nobel laureate in chemistry, Scott Gottlieb, MD, former FDA head, and Alex Gorsky, former CEO of Johnson & Johnson, are among the board of directors.

Xaira is among a staggering list of biotech companies that Baker has co-founded over the past three decades.

“Science all becomes obsolete quickly because the field’s moving!” Baker told me. “The people that you mentor are more important than any science you do. They all go on and do great things.”

2024 Nobel Week was a testament to Baker’s scientific reach. Nearly 200 current and former members of his lab gathered in the Grand Hôtel in Stockholm to celebrate the newly named laureate, who was among a cohort of renowned AI experts who swept the awards ceremony.

Baker shared the Nobel Prize in Chemistry with Google DeepMind duo, CEO Demis Hassabis, PhD, and then-senior research scientist, John Jumper, PhD, whose AI model, AlphaFold, solved the protein structure prediction problem and has become one of the most widely adopted computational tools for drug discovery.

Meanwhile, the Nobel Prize in Physics was jointly awarded to Geoffrey Hinton, PhD, professor emeritus at University of Toronto, and John Hopfield, PhD, professor emeritus at Princeton University, for foundational discoveries that enabled machine learning with neural networks.

Together, the prizes represented a pivotal moment. AI was no longer confined to computer science but had become a transformative force across disciplines, earning recognition as a breakthrough deemed to confer the “greatest benefit to humankind.”

Back at the IPD, Baker’s research group spanned multiple floors. Yet, he knew everyone’s name, where they sat, and moved easily between conversations, bringing together researchers whose expertise might unlock a new direction. In the weeks after receiving the historic Nobel call, Baker chose to remain fully present for his team, implementing a strict “no travel rule,” despite the avalanche of invitations and media attention that accompanied the prize.

“David’s really good at forcing you to break the ice with people,” said Seth Woodbury, a graduate student who is designing metallohydrolases, enzymes that cleave some of the strongest bonds in biology for sustainability applications, including degrading pollutants. “Once you talk to your colleagues at happy hour, it’s not so scary to go ask them a question.”

Woody Ahern, graduate student and co-author of the metallohydrolase Nature paper, adds that Baker has a “very reasonable disdain for hierarchy.”

“Anyone can speak up in meetings. Anyone can question the work. It breeds this culture of staying focused on what matters in an interdisciplinary way,” said Ahern.

When Ria Sonigra was applying to graduate schools in the U.S., every option felt equally far from her home in India. She recalled sending Baker a cold email with questions about the lab. He quickly replied and offered to connect her with another international student who could help her navigate the application process. Today, Sonigra is an IPD graduate student, designing programmable nanopores for molecular sensing and sequencing.

“People outside the lab may think that David can’t pay attention to everyone, which is not true,” Sonigra said. “He knows your project and what he expects of you before the next meeting, even if he has a hundred trainees.”

At one point, Baker waved me over with a smile. “You’re missing chocolate hour!” he said, inviting me to one of many small weekly rituals that embodied the collaborative culture he had built.

Lowest energy search

At GEN’s inaugural virtual event, The State of AI in Drug Discovery, I asked Baker for his initial reactions to winning the Nobel.

“My group was not the first to do protein design,” he said humbly.

The field’s early innings trace back to 1988, when William DeGrado, PhD, demonstrated that sequences not found in nature could achieve stable 3D folds. The work challenged the long-held belief that functional proteins could only arise through evolution.

Steps toward computational design came a decade later, when for the first time, an in silico predicted protein was experimentally validated to adopt a target structure. The work was published in a Science study led by Steve Mayo, PhD.

Baker, alongside then-postdoctoral researcher, Brian Kuhlman, PhD, went a step further in 2003, expanding the design scope to include flexible backbones that represented entirely new folds, making it possible to not only modify natural proteins, but to create new ones from scratch.

“The prize was given because protein design has so much promise now, and that reflects the work of the whole community,” Baker continued.

Today, Degrado, Mayo, and Kuhlman are continuing to advance structural biology as prominent faculty members across University of California, San Francisco (UCSF), California Institute for Technology, and University of North Carolina (UNC) Chapel Hill, respectively.

Top7 was the first protein created on a computer with a custom amino acid sequence that folds into a never-before-seen structure. When viewed at an angle, the transparent partition allows the two forms to become superimposed, illustrating the beauty of uniting sequence and structure. [UW Institute for Protein Design]

Decades before OpenAI co-founder, Andrej Karpathy, coined the term “vibe coding,” Baker’s team was writing a program in FORTRAN. Named Rosetta, the molecular modeling suite simulated proteins atom-by-atom based on biophysical properties, from hydrogen bonds to backbone torsion angles. By calculating free energy, Rosetta could estimate which sequences were most likely to achieve a desired structure: the lower the energy, the more stable the predicted fold.

Yet, a protein’s energy landscape is rugged, with countless local minima among an astronomical number of conformations. Success was rare. Researchers were searching for a single grain of sand across the desert.

Still, “Rosetta was impressive,” said Sierin Lim, PhD, an associate professor at Nanyang Technological University, who is among a group of researchers engineering self-assembling nanoscale containers, known as protein cages, for applications across drug discovery, imaging, and materials science. She recalled watching molecules move on her screen in Singapore in the early 2000s. At the time, Rosetta was the only program that could model proteins.

Over the next twenty years, Baker adamantly pushed Rosetta to be openly available, inviting collaborators to not only use the software, but to improve it.

PyRosetta, a user-friendly Python-based implementation developed by Johns Hopkins University researchers led by Jeffrey Gray, PhD, broadened Rosetta’s access for structural biologists without a strong computational background. Meanwhile, progress in generating high affinity and selective ligand binders and epitope scaffolds for vaccine development were bringing computational proteins closer to real-world medicines.

What started as a single lab project grew into the Rosetta Commons, an international collaboration spanning more than 100 laboratories.

“It was a great move making Rosetta open, seeing what it can do now,” Lim said.

CASP14

Then came a seminal 2017 report titled simply, “Attention Is All You Need.”

Researchers from Google introduced the transformer, a neural network architecture that enabled machines to analyze entire sequences at once. By using a “self-attention” mechanism, AI models could now uncover patterns across massive datasets at unprecedented scale. Soon, large language models (LLMs) trained on internet-scale text could not only understand, but converse in eloquent dialogue with humans.

The generative AI era had begun.

While the rest of the world was captivated by chatbots, structural biologists were sitting on a treasure trove of biological data pristine for machine learning.

For over fifty years, researchers had painstakingly deposited hundreds of thousands of experimentally determined structures in the Protein Data Bank (PDB) for public use. This molecular atlas now offered AI a window into the rules of biology.

In 2020, Baker received a phone call from one of the organizers of the Critical Assessment of protein Structure Prediction (CASP) competition, the biannual experiment that assesses the field’s latest state-of-the-art models.

“The first thing he said was, ’David somebody has done amazingly well this year, and it isn’t you!’” Baker recalled during his Nobel banquet speech. “That was how I first learned about the work of Demis and John.”

Instead of relying on human-defined biophysical rules, AlphaFold quickly learned decades of biochemistry from the PDB, uncovering the hidden instructions governing an amino acid sequence to fold into its 3D shape. At CASP14, the model remarkably predicted structures that were indistinguishable from real-world proteins. Months of laboratory work turned into a computational task completed in minutes.

Hassabis was quick to translate the breakthrough into medicine, taking the helm of DeepMind’s drug discovery spinout, Isomorphic Labs, as CEO a year later.

Today, the company’s IsoDD (Isomorphic Labs Drug Design Engine) platform, expands the druggable landscape by probing previously inaccessible biology, including predicting induced-fit interactions, where proteins change shape upon ligand binding, and identifying hidden binding pockets for drug targeting.

Isomorphic was betting, not on single therapeutic assets, but on a general discovery engine applicable across any disease area. That vision has since secured major pharma partnerships with Novartis, Eli Lilly, and Johnson & Johnson.

“I’ve always believed the No.1 application of AI should be to improve human health,” wrote Hassabis on LinkedIn when announcing Isomorphic’s whopping $2.1 billion funding raise in May.

Diffusion evolution

Concurrently, Baker’s team began applying deep learning to de novo design, drawing inspiration from AI’s emerging ability to generate realistic images. These diffusion models could operate on atomic coordinates and create entirely new protein backbones. Designs were conditioned for desired structural and functional constraints, opening the door to programmable biology.

When Baker’s team presented de novo design model, RFdiffusion (RoseTTAFold diffusion), in Nature in 2023, Mohammed AlQuraishi, PhD, assistant professor of systems biology at Columbia University, described the advance as “a really big deal.”

‘‘Prior to the ‘diffusion evolution’, the success rates were probably on the order of 1 to 10,000, if you’re lucky,’’ AlQuraishi told me shortly after RFdiffusion’s publication. ‘‘With diffusion models, the success rates are closer to the single percentages when you get into the laboratory. It’s a huge magnitude improvement of what it used to be.”

Donald Hilvert, PhD, professor emeritus at ETH Zurich, met Baker twenty years ago while working on enzyme design with Defense Advanced Research Projects Agency (DARPA). Traditional Rosetta methods would carve out binding pockets in existing proteins and install a new catalytic apparatus.

“But the activities were not very good,” Hilvert recalled. Designing catalysis, where success depended on precisely positioning chemical groups to stabilize fleeting transition states, proved far more difficult than engineering a stable protein fold. Rosetta struggled to achieve that level of accuracy, prompting much of the field, including Baker, to turn attention elsewhere.

“Two years ago, David called me and said, ‘Why don’t you come and visit? All these new AI-driven techniques are really changing the game!’” Hilvert told me.

Hilvert has spent the past two summers at the IPD, collaborating with Woodbury, Ahern, and IPD postdoctoral researcher, Donghyo Kim, PhD, to design metallohydrolases using RFdiffusion. He “hardly knew how to turn on a computer,” yet was reading Python scripts and generating his first computational designs within weeks. To his amazement, experiments quickly yielded five or six promising hits.

“There is this common purpose of people helping one another,” Hilvert said. “David sets the tone from the top.”

Application generalist

As I walked through the halls of the IPD, I saw the extraordinary reach of protein design applications firsthand. Desks were intermingled across fields. The proximity was deliberate for ideas to travel as far as possible.

Florence Hardy, PhD, is a postdoctoral researcher tackling a new enzyme design project for global health applications, including streamlining the manufacturing process for therapeutics.

“I always say that I can only think in a ten angstrom sphere at a time,” she chuckled. “That’s just as big as the active site.”

“Most medicines focus on inhibitors,” Xinru Wang, PhD, explained when describing her postdoctoral research developing insulin agonists, or binders that lead to activation, to address metabolic disease. In contrast to blocking activity, “turning on” a signaling complex required precise structural tuning that was a natural fit for the IPD’s expertise.

Last November, Wang and colleagues published a study in Molecular Cell, demonstrating that de novo designed insulin receptor (IR) agonists could extend glucose-lowering effects. The findings offered a therapeutic alternative to escalating insulin doses, which is a known contributor to resistance. Notably, these engineered agonists avoided triggering cancer proliferation that is often associated with excessive insulin activation. Wang is currently an assistant professor at Northeastern University.

Tabitha Tcheau designs DNA binding proteins inducible with small molecules that can recognize novel pathogens and trigger the plant immune system. The highlight of her project, she says, is the ability to span interdisciplinary subgroups, from conformational dynamics, small molecules, and nucleic acids.

“One thing that blew me away here is that people are extremely supportive,” Tcheau told me. “Everyone you ask is super eager to help.”

Enisha Sehgal is among a team of researchers designing sequence specific DNA binding proteins that can power programmable transcription factors, targeted gene regulation, and new genome engineering tools.

“Being in this lab allows you to be a specialist in protein design, but a generalist in all the applications,” Sehgal said. “You get answers faster. You can iterate faster. Science moves faster.”

Visiting researcher and machine learning scientist, Kieran Didi, reiterates how the IPD’s interdisciplinary team enables rapid experimental validation of models. “I’m not going to spend two months in this fantasy world of computational benchmarks,” he said. “In the next week, I know if the model is actually working. Someone will quickly put it to the reality test.”

Postdoctoral researcher and chemist, Declan Evans, PhD, concurs and sees himself as the Alpha tester.

“I can go straight to the developer and say, ‘this is not how computational chemists would use this software,’” Evans said. “You can see changes being made in real time.”

Back in Baker’s office, he told me about his regular weekend escape to the mountains, one of the benefits of living in Seattle. Skiing and hiking were activities he valued highly. When asked to contribute an item to the Nobel Prize Museum, Baker chose a broken ski pole as a symbol that progress often comes through overcoming setbacks.

“But I don’t think people get ideas on top of mountains,” Baker tempered. “If you’re going to be a [principal investigator], you have to really like mentoring. For me, it’s super fun!”

Baker’s most enduring creation may not be any single protein, but rather the network he built—the diverse, inviting, and interconnected communal brain.

The post Protein Design’s AI Revolution: Inside David Baker’s “Communal Brain” appeared first on GEN - Genetic Engineering and Biotechnology News.

Tick-Borne Nairoviruses Use OTU Proteases to Evade Human Antiviral Signals

Edusehat — Wed, 15 Jul 2026 15:10:12 +0700

Summer is peak tick season, and with it comes familiar threats like Lyme disease and Rocky Mountain spotted fever. But scientists say another group of tick‑borne pathogens is quietly gaining ground: nairoviruses, a diverse family of negative‑sense RNA viruses carried by ticks across Asia, Europe, Africa—and now the western United States. Several nairoviruses can infect humans, causing high fevers, severe headaches, and, in some cases, organ dysfunction. One member of the family, Crimean‑Congo hemorrhagic fever virus (CCHFV), is often fatal and considered a global‑level threat.

Nairoviruses are found in ticks that feed on wildlife, livestock, and people. Recent human infections have been documented in China and Japan, including Songling virus (SGLV), Tacheng tick virus 1 (TTV1), and Yezo virus (YEZV). A related virus, Beiji virus (BJNV), caused an outbreak involving more than 100 patients in northeastern China. And on the U.S. West Coast, researchers recently identified Pacific Coast Tick nairovirus (PCTNV) in Dermacentor occidentalis, a tick already known to transmit Rocky Mountain spotted fever. As the paper noted, “Pacific Coast Tick nairovirus… was recently identified in Mendocino, California, from tick species known to harbor human pathogens and having a large presence across the state.”

The new study, titled “Insights into the Structure and Function of the OTU Protease Virulence Factors from Emerging Human Nairoviruses,” was published in ACS Infectious Diseases and reveals how these emerging viruses may slip past human immune defenses. All orthonairoviruses encode a specialized enzyme called ovarian tumor protease (OTU), which can remove small protein tags—ubiquitin and ISG15—from human proteins. Those tags normally act as alarm signals that activate immune responses, and removing them effectively evades the immune system. As the paper explained, “OTUs exhibit varying levels of deubiquitinating (DUB) and deISGylating activities that facilitate viral immune evasion, establishing them as key virulence factors.”

In the work, researchers isolated OTU proteases from four emerging nairoviruses—SGLV, TTV1, YEZV, and PCTNV—and compared their ability to strip immune‑signaling proteins. The standout was PCTNV, whose enzyme showed the strongest ability to remove both ubiquitin and ISG15. That suggests PCTNV may be unusually adept at evading human immunity, raising concerns because the virus is carried by a human‑biting tick common along the Pacific Coast.

The team also resolved high‑resolution crystal structures of several OTU proteases. These structural insights allowed the researchers to train computational models that begin to predict which nairoviruses may pose the greatest threat. “The biochemical and structural insights provide a path forward for predicting OTU activity among current and emerging nairoviruses,” the authors wrote.

Such predictive tools could help public‑health agencies monitor new tick‑borne viruses before they spread widely. As corresponding author Scott D. Pegan, PhD, of the University of California, Riverside, noted, “This study reinforces the need to be vigilant about not just tick bites but the type of ticks that an individual has been bitten by, as they may carry diseases beyond what we have been used to looking for.”

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AstraZeneca Licenses Global Rights to Dizal Lung Cancer Drug for Up-to-$1.5B

Edusehat — Wed, 15 Jul 2026 15:10:11 +0700

AstraZeneca has acquired exclusive global rights to develop and commercialize Dizal Pharmaceutical’s marketed lung cancer drug Zegfrovy® (sunvozertinib), through an agreement that could generate up to $1.5 billion for the spinout of the pharma giant’s onetime Chinese R&D operation.

Wuxi City, China-based Dizal has inked an exclusive license agreement with AstraZeneca to expand the development of Zegfrovy into new indications beyond the one for which it has approvals in the United States and China—namely the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations, whose disease has progressed on or after platinum-based chemotherapy.

Dizal has been pursuing approvals from the FDA and China’s Center for Drug Evaluation (CDE) for a new indication for Zegfrovy, as a first-line treatment for NSCLC with exon 20 insertion EGFR mutations. In May, Dizal presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting and simultaneously published in The New England Journal of Medicine (NEJM), positive results in the indication from its Phase III WU-KONG28 trial (NCT05668988).

In the study, Zegfrovy showed a median progression-free survival (PFS) of 10.3 months compared with 7.5 months PFS for platinum-doublet chemotherapy in untreated NSCLC patients with EGFR exon 20 insertion mutations (exon20ins).

“The efficacy of sunvozertinib was superior to that of chemotherapy as first-line treatment for advanced NSCLC with EGFR exon 20 insertions,” the researchers concluded in their study, “First-Line Sunvozertinib in NSCLC with EGFR Exon 20 Insertion Mutations,” which was published May 29 in NEJM.

Dizal also showed Zegfrovy delivering a BICR-assessed best objective response rate (BoR) of 68.1% vs. 35.4% with chemotherapy, and a median duration of response (DoR) of 11.2 months vs. 7.1 months for chemo.

Based on those results, Dizal has filed supplemental New Drug Applications (NDAs) for Zegfrovy in the first line to the FDA and China’s Center for Drug Evaluation (CDE). Both regulators have granted their Breakthrough Therapy designations to Zegfrovy in that setting.

“AstraZeneca is a leader in treating EGFR-mutated lung cancer, and we are eager to add Zegfrovy to our world-class portfolio of innovative medicines for patients whose tumors carry exon 20 insertion mutations,” Dave Fredrickson, executive vice president of AstraZeneca’s Oncology Hematology Business Unit, said in a statement. “With this agreement, we will bring a differentiated, oral targeted treatment to these patients with limited options across the globe.”

20% jump

Dizal shareholders reacted to the agreement with AstraZeneca warmly enough to send shares traded on the Shanghai Stock Exchange jumping 20%, from RMB 46.94 ($6.93) to RMB 56.33 ($8.31). But the news did not appear to wow AstraZeneca investors, as shares of the pharma giant traded on the London Stock Exchange dipped 1.95% today, from 12,610 pence to 12,364 pence. Shares traded on the New York Stock Exchange also fell 1.95% as of 2:17 pm ET, from $169.47 to $166.16.

Dizal was established in 2017 as a joint venture between AstraZeneca and China’s State Development & Investment Corp. (SDIC), with AstraZeneca spinning out the R&D operations of its China Commercial Innovation Center to Dizal as well as three preclinical candidates, one each in cardiometabolic disease, respiratory disease, and oncology, the drug that was eventually developed into Zegfrovy. Xiaolin Zhang, PhD, who headed the innovation center, was appointed Dizal’s CEO, a position he still holds.

AstraZeneca has agreed to pay Dizal $600 million upfront; up to $900 million tied to achieving development, regulatory, and sales-related milestones; plus tiered double-digit royalties on global sales of Zegfrovy. The milestone payments consist of up to $400 million in clinical development-related payments and up to $500 million in sales-related payments, Dizal disclosed in a regulatory filing to the Shanghai Stock Exchange.

In March, Dizal reported that Zegfrovy generated about RMB 576 million (about $85.057 million) in revenue last year, up 85% from 2024. Zegfrovy accounted for nearly three-fourths (72%) of Dizal’s total 2025 sales of RMB 801 million ($118.282 million).

Zegfrovy is a once-daily oral irreversible epidermal growth factor receptor (EGFR) inhibitor approved by the FDA in July 2025 based on evidence from the Phase I/II WU-KONG1B trial (NCT03974022) in patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations whose disease has progressed on platinum-based chemotherapy and received Zegfrovy 200 mg once daily with food.

WU-KONG1B enrolled 202 patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations who had received previous platinum-based chemotherapy. The trial was conducted at 89 sites in the United States, Argentina, Australia, Canada, China, Chile, France, Italy, Malaysia, South Korea, Spain, and Taiwan.

AstraZeneca’s licensing deal with Dizal is expected to close in the second half of this year, subject to customary closing conditions and regulatory clearances. AstraZeneca said the transaction does not impact its 2026 financial guidance to investors, which it reaffirmed on April 29 as calling for a mid-to-high single-digit percentage increase in total revenue, and a low double-digit increase in “core” earnings per share from primary ongoing business activities.

“As a leading global company with a strong lung cancer franchise, AstraZeneca will help ensure patients around the world can benefit from this innovation discovered by Dizal scientists in China,” stated Zhang.

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Genome-Scale CRISPRi Atlas Maps Gene Function Across Human iPSCs

Edusehat — Tue, 14 Jul 2026 07:55:09 +0700

A new genome-scale atlas is offering an unprecedented look at how individual genes shape the transcriptional landscape of human induced pluripotent stem cells (iPSCs). Published in Nature Biotechnology, the resource catalogs the effects of perturbing 11,692 expressed genes across more than 2.5 million single cells, creating a reference framework for understanding how pluripotent identity is maintained and regulated.

The study is titled, “A genome-scale CRISPRi perturbation atlas of human induced pluripotent stem cells.”

Human iPSCs can differentiate into virtually any cell type, yet the functions of most genes within this state remain poorly understood. Prashant Mali, PhD, senior author and professor of bioengineering at UC San Diego, said the team set out to fill that gap by systematically switching off genes one by one using CRISPR interference (CRISPRi) and measuring the resulting transcriptome-wide changes. “The result is a kind of reference atlas; it’s a way to look up what perturbing almost any gene does to a stem cell’s behavior, measured here as the impact on its whole transcriptome,” Mali said.

The dataset captures how gene perturbations cluster into shared molecular signatures, revealing functional relationships among protein complexes, metabolic pathways, and self-renewal genes. By correlating transcriptional phenotypes across thousands of perturbations, the researchers reconstructed a map of the pluripotent state that recapitulates known regulatory modules while surfacing previously unrecognized ones.

Exploring the atlas led the team to identify new regulators of stem cell biology. They uncovered ZBTB41 as a metabolic factor and RNF7 as a contributor to pluripotency regulation, validating both through metabolic tracing, immunofluorescence, and protein–protein interaction assays. The resource also enabled a genome-scale screen of A‑to‑I RNA editing modulators, revealing DBR1 as a potent regulator of adenosine-to-inosine conversion.

Co-first author Yesh Doctor, a bioengineering PhD student in Mali’s lab, described the atlas as a “hypothesis engine” for stem cell researchers. Instead of running thousands of perturbation experiments, scientists can now query the open-access map to identify candidate genes involved in differentiation, metabolism, or disease-relevant pathways. “Scientists can use it to look up the functions of genes and build hypotheses on them instead of having to run the experiments themselves,” Doctor said.

Beyond basic biology, the team sees the atlas as a foundation for computational modeling. The scale and consistency of the dataset make it well suited for training AI systems aimed at predicting genotype–phenotype relationships. “These comprehensive, genome-scale screens enable generation of reference maps that are not just invaluable for basic science discovery, but also an important resource for powering future computational and AI tools for genotype-phenotype prediction,” Mali said.

The open-access atlas is available here, providing a new reference point for understanding how genes shape human stem cell identity and offering a tool for virtual disease modeling and target discovery.

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Analytical Challenges for Antibody-Drug Conjugate (ADC) Manufacturing

Edusehat — Tue, 14 Jul 2026 07:55:09 +0700

Antibody-drug conjugates (ADCs) offer a compelling promise: delivering a cytotoxic payload directly to a tumor cell while sparing the rest of the body from harm. Composed of a tumor-targeting antibody, a cytotoxic agent, and a chemical linker, ADCs combine the selectivity of monoclonal antibodies that bind to tumor-specific antigens with the cytotoxic potency of small-molecule drugs. These therapeutics have the potential to target tumors while reducing systemic toxicity, opening new treatment pathways for many types of cancer. Recent clinical data, including encouraging Phase I findings in platinum-resistant ovarian cancer, validate that potential and fuel the field’s remarkable growth.

Despite the promise of ADCs, their unique structure complicates manufacturing, characterization, and regulatory assessment. Developers must carefully consider the interplay between the antibody, linker, and payload to optimize therapeutic efficacy and safety. The drug-to-antibody ratio (DAR), conjugation site specificity, impurity profile, and linker stability all influence an ADC’s pharmacokinetics, pharmacodynamics, and ultimately its clinical safety profile. Robust analytical methods are therefore critical throughout the development process to ensure both safety and efficacy.

Given the hybrid nature of ADCs, regulatory expectations are still evolving. To navigate this uncertainty, analytical risk management is essential. Moreover, cross-functional collaboration among analytical scientists, process development teams, regulatory experts, and quality assurance professionals is key to ensure that early-stage methods are sufficiently robust and scalable for commercial manufacturing.

Success in this environment requires deep analytical expertise, robust quality-by-design frameworks, and development partners who understand the full arc from early-stage linker-payload synthesis through GMP-compliant manufacture. It requires the capability to handle highly potent compounds safely, to purify structurally complex intermediates at scale, and to translate rigorous quality control into processes that are commercially viable.

This collection of articles and expert perspectives explores the critical challenges shaping ADC development today, from analytical strategy and impurity control to linker technology innovations and evolving regulatory standards. It also examines the collaborative expertise needed to bring these transformative therapies to patients.

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Mung Bean Pan-Genome Study Maps Key Genes for Yield, Nutrition, and Pest Resistance

Edusehat — Tue, 14 Jul 2026 04:20:18 +0700

Because Mung beans (Vigna radiata) are nutritious, inexpensive, have nitrogen-fixing capacity, and are relatively easy to grow with a short growing cycle, they are an important crop for food security in many parts of Asia, Africa, and other regions. Now, researchers have made a significant contribution to a landmark international study that has uncovered tens of thousands of previously hidden structural variations influencing yield, nutritional quality, insect resistance, and other relevant mung bean traits.

The study entitled, “Graph-based pan-genome reveals structural variations associated with agronomic traits in mung bean,” published in Nature Genetics, presents the world’s first graph-based pan-genome for the pulse crop (a legume grown specifically for its dry edible seeds) offering a comprehensive resource for understanding the genetic basis of key agronomic traits and accelerating crop improvement.

The international research team, co-led by researchers at the Chinese Academy of Agricultural Sciences and Murdoch University’s Centre for Crop and Food Innovation (CCFI), assembled chromosome-scale genomes from genetically diverse mung bean accessions and analyzed genomic variation across 580 global accessions. The resulting graph-based pan-genome captures more than 75,000 gene families and identifies over 66,000 structural variants, offering important insights that will help breeders target key agronomic traits and accelerate crop improvement.

More specifically, the authors note that, “integrating these structural variants and single nucleotide polymorphisms, genome-wide association studies across five environments identified candidate genes for 20 agronomic traits, underscoring the pivotal roles of these variants in driving mung bean domestication and improvement.”

Mechanistically, they add, the work demonstrates that “a 68-bp promoter insertion in VrTIFY6B and a 136-bp promoter deletion in VrPGIP1 regulate flavonoid content and confer bruchid resistance, respectively.”

In Australia, mung bean generates over $100 million annually in export revenue. At roughly three times the price of wheat, it represents a highly profitable break crop opportunity for Australian growers; however, seasonal rainfall variability continues to drive significant year-to-year swings in the size and value of the crop.

By cataloging tens of thousands of previously invisible structural variations and linking them to agronomic traits through genome-wide association analysis, this new genomic resource gives breeders a far more complete map of the genetic variation they can work with.

Globally, where mung bean underpins the diets and incomes of millions of smallholder farmers across Asia and Africa, the study’s findings on genes governing seed nutritional compounds and resistance to bruchids, a major storage pest, have direct implications for global food security.

Rajeev Varshney FRS FAA, CCFI director, said the research represents a major advance in crop genomics and demonstrates how next-generation genomic technologies are transforming plant breeding. “Traditional reference genomes capture only part of the genetic diversity within a crop species. By constructing a graph-based pan-genome, we can now identify structural variations that were previously invisible but often have profound effects on important agricultural traits.

“These discoveries provide breeders with powerful new genomic tools to accelerate the development of higher-yielding, more nutritious and climate-resilient mung bean varieties,” Varshney continues. “The genomic resources generated through this work will support marker-assisted breeding, genomic selection and genome editing, enabling breeders to deliver improved varieties to farmers much faster.”

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New Lung Model Reveals RSV Immune Response, Points to Better Treatments

Edusehat — Tue, 14 Jul 2026 04:20:16 +0700

Researchers at University College London (UCL) Great Ormond Street Hospital for Children (GOSH) have built a new lab model of infant lungs to show why respiratory syncytial virus (RSV) makes infants so much sicker than adults, and to allow them to test new treatments. The miniature model of a baby’s airways was created using pediatric airway cells, blood vessel cells, and neutrophils (a type of white blood cell that acts as the immune system’s primary response to infection).

Studies using the new model suggest that future therapies for RSV should target both the virus and its immune response to ensure babies get the best possible outcomes. Research lead Claire Smith, PhD, at UCL Great Ormond Street Institute of Child Health, said, “This model allows us to watch early immune responses unfold and study them in a human setting that reflects the infant airway. That’s something animal models often struggle to capture, especially when it comes to age-specific effects.”

Senior and corresponding author Smith and colleagues reported on their findings in Nature Communications in a paper titled “Neutrophil myeloperoxidase as a functional biomarker for RSV severity: implications for in vitro therapeutic screening.” In their report they concluded, “These findings identify neutrophil–epithelial interactions as a useful target for intervention and support the use of physiologically relevant human models to accelerate the development of therapies that limit immunopathology while preserving antiviral defense.”

RSV is the biggest cause of severe respiratory tract infections in infants and young children, resulting in over three million hospital admissions worldwide every year. RSV infection causes wheezing and breathing difficulties, and in the worst cases infants end up in intensive care. Despite this, treatment options for severe RSV disease remain extremely limited. “The lack of accessible, effective therapies highlights the need for continued advancements in RSV treatment and prevention,” the authors wrote.

During RSV infection neutrophils are rapidly mobilized to the lungs and play a key role in virus-targeting host defenses, the investigators continued. However, excessive neutrophil infiltration and activation can also contribute to airway inflammation, epithelial damage, and disease severity. “Understanding neutrophil behavior and activation during RSV infection, including during their migration across the airway epithelial barrier, is crucial for developing therapeutic strategies to mitigate pathological inflammation without compromising the antiviral response,” they suggested.

For their reported study the team aimed to create an in vitro model that recapitulates key clinical outcomes of infants with RSV bronchiolitis. To do this they established an air-liquid interface (ALI) system that incorporated pediatric airway epithelial cells, endothelial cells, and neutrophils from adults, to mirror the blood-airway barrier. “Differentiated airway epithelial cells (AECs) cultured at the air–liquid interface (ALI) provide a physiologically relevant platform to study neutrophil migration and the effect of antiviral treatments on this process,” they note. To compare with an adult response to RSV, the research team also made a model of an adult’s airways.

When the models were infected with RSV, the team found that the pediatric airway cells attracted far more white blood cells than did the adult airway cells. This influx can block an infant’s small airways and make it harder for them to breathe.

Neutrophils normally circulate in the blood but enter lung tissue in response to infection. In the baby airway model, researchers found that the neutrophils that entered the lung tissue were more activated and triggered a stronger inflammatory reaction than in the adult model. This effect was dependent on the immune cells physically moving through the infected tissue, not just responding to chemical signals released by it, making this type of model essential for study.

The results suggest that it is the infant airway itself, not just the virus, that ramps up the immune response and causes damage to the lungs. First author Machaela Palor, PhD, at UCL Great Ormond Street Institute of Child Health, said: “These findings help explain why RSV is often much more severe in infants than in adults. The pediatric airway actively shapes how immune cells behave during the infection.”

The researchers tested two antiviral drugs (remdesivir and RSV604). they found that both stopped the virus from multiplying, but only RSV604 also calmed the overactive immune response, reducing levels of a key inflammatory protein myeloperoxidase (MPO) released by white blood cells—high levels of which are linked to more severe RSV disease in babies. Remdesivir had no effect on this, suggesting that not all antivirals are equal when it comes to protecting the infant airway from immune-driven damage. “While both drugs reduced viral load, only RSV604 attenuated MPO expression,” the team stated.

The findings suggest that treating severe RSV in babies may require more than just stopping the virus—it may also be important to calm an overactive immune response. “This model suggests that MPO could be useful as a readout of therapeutic efficacy,” the team stated. “Targeting neutrophil-driven inflammatory pathways may be critical for reducing pathology in infant RSV infection.”

The researchers hope their findings and the new approach to research on RSV will accelerate the development of treatments better tailored to infants. “Our model gives us a way to assess both sides of the problem at once,” Smith said. “We can not only ask whether the drug stops the virus but also whether it helps control immune response in the infant airway. “This work reinforces the idea that age matters in respiratory infection. Understanding how infant airways shape immune responses will be key to designing safer and more effective RSV treatments.”

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Genetic Map Opens Door to Development of New Therapies to Reverse Bone Loss

Edusehat — Mon, 13 Jul 2026 21:05:14 +0700

An international team of scientists report that they have successfully mapped the cells and genes that regulate bone formation and loss and discovered the critical role that blood vessel cells play in bone health. By combining genomic sequencing with data from half a million individuals, the research team identified hundreds of previously unknown genes that govern bone health and revealed cells surrounding blood vessels as one of the drivers of bone repair.

The study “Multiscale analysis and functional validation of the cellular and genetic determinants of skeletal disease” is published in Nature Genetics. The team says its findings fundamentally enhance our understanding of skeletal disease. It is hoped the discovery will enable the development of new therapies to rebuild lost bone, offering hope to almost half of all individuals over 50 living with rare and common skeletal conditions such as osteoporosis, osteoarthritis and osteogenesis imperfecta, as well as those with rare bone disorders and cancers that spread to bone.

“Most people don’t realize that bones are constantly changing; the human body replaces its skeleton every 10 years or so,” said Peter Croucher, PhD, professor at the Garvan Institute of Medical Research in Australia. “This is a hugely important process, but until now we’ve had a limited understanding of the cells and mechanisms that control this turnover of bone. “Most of the drugs now available focus only on halting bone disease, rather than rebuilding lost bone, which is really important for reversing damage.”

Detailed map of cells and genes that regulate bone health

The team used single-cell RNA sequencing to measure which genes are switched on within individual cells found in bone, focusing on the interface between the hard bone and bone marrow which is the key site for the formation and breakdown of bone.

The Institute’s Ryan Chai, PhD, pointed out that the team’s analysis found 34 different groups of cells and defined the genes that are active in each of these cell types. “To our surprise, more than half of the genes identified have never before been shown to play a role in maintaining bone health, which is a significant finding,” he added.

Ryan Chai, PhD, and Peter Croucher, PhD, from the Garvan Institute of Medical Research [Garvan Institute]

The team used its map to identify cells involved in rare and common skeletal diseases, including osteogenesis imperfecta and osteoporosis. For the latter, the researchers analyzed the UK Biobank, one of the world’s biggest and most comprehensive collections of biological samples.

By analyzing genetic and bone density data from half a million people participating in the UK Biobank, the team was able to pinpoint exactly which cells drive skeletal disease, according to John Kemp, PhD, associate professor from Mater Research.

“These include cells known to regulate bone formation and bone loss, as well as blood vessel cells that, until now, have had underappreciated roles in bone health,” he said.

Croucher explained that the research uncovered new therapeutic opportunities against not only bone disease, but also cancer. “Bone is the main hiding place for dormant cancer cells and a common site of relapse, so identifying the cells and genes that drive bone turnover also opens new opportunities to prevent cancer metastasis,” he said.

The team is now further investigating the roles of newly discovered bone-regulating cells and genes in the hope of developing new medicines against these targets. Its data has been made accessible to medical researchers worldwide through an open access platform.

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Immune Evasion Uncovered by Virome-Wide Ubiquitin Ligase Discovery

Edusehat — Mon, 13 Jul 2026 21:05:13 +0700

In a new study published in Science titled, “Virome-wide ubiquitin ligase discovery reveals diverse mechanisms of immune evasion,” researchers from Harvard Medical School (HMS) have uncovered how viruses hijack cells’ garbage-disposal systems to evade immune attack.

The study applies ORFeome, a tool that broadens the scale by which researchers study viral proteins. The advance proposes to speed basic discoveries in virology, inform the development of new vaccines and treatments to protect against emerging pathogens.

“This library reveals how viruses manipulate human cells on a scale that simply wasn’t possible before,” said Stephen Elledge, PhD, professor of genetics and medicine at Harvard Medical School and senior author of the study. “We believe it changes virology from studying one virus at a time to discovering the common strategies and surprising innovations that viruses have evolved, providing a powerful new foundation for understanding emerging viral threats.”

ORFeome and other ORF libraries are named after open reading frames, DNA sequences that encode proteins. Previous viral ORF libraries from other groups focused on individual viruses or virus families that contained 100 or 200 sequences each. The new ORFeome contains about 13,000 physical DNA sequences, or constructs, that code for about 9,000 proteins from 513 different viruses, including Andes hantavirus, Ebola virus, and Zika virus.

“Most viruses have never been studied in detail, yet evolution has already performed countless experiments for us. This library gives us a way to read the results of those experiments across the viral world,” said Elledge, who is also a Howard Hughes Medical Institute (HHMI) Investigator.

The team attached a genetic barcode to each ORF, allowing researchers to conduct studies of all 13,000 ORFs at once.

“We can insert the sequences into a population of cells, ask questions like which ones cause the cells to grow better or less, and then identify those by their barcodes when the experiment is finished,” said Colin O’Leary, PhD, HMS research fellow and co-author on the study. “It hasn’t been possible before to do genetic screens like this with viral proteins.”

The team will make the ORFeome freely available for the research community. Elledge and colleagues implemented a flexible design to enable application to other model systems and experiments.

The team conducted genetic screens in three cell types, searching for viral proteins that affect cell proliferation, stop cells from presenting antigens to trigger the immune system to attack, or block the effects of interferon. Results uncovered more than 700 viral proteins that contribute to at least one of those actions.

The study opens opportunities to design drugs that hinder viral activity while sparing normal function.

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StockWatch: Insilico Projects Profit, Revenue Leaps as AI-Developed Lead Candidate Moves to Phase III

Edusehat — Mon, 13 Jul 2026 06:40:09 +0700

Insilico Medicine (Hong Kong Exchange: 3696), an AI-based drug developer whose profile within biopharma has risen with its recent collaborations with industry giants, has offered investors an upbeat revenue and profit forecast for the first half of 2026, driven by its series of partnerships and a wide-ranging pipeline whose first program has reached late-stage development this past week.

Insilico said it expects to finish the first half of 2026 in the black, with “net profit” or net income ranging from approximately $33.5 million to $39.5 million, compared with its $19.2 million net loss in January–June 2025. Insilico also released adjusted non-International Financial Reporting Standards (IFRS) net profit forecasts in the range of approximately $45.5 million to $51.5 million for H1 2026. Non-IFRS metrics exclude one-time costs, such as restructuring charges and asset sales.

The company is additionally forecasting record first-half revenue ranging from approximately $102.5 million to $106.5 million, up approximately 272.7% to 287.3% from a year ago.

By contrast, Insilico finished all of 2025 with $56.239 million in revenue, down 34% from $85.834 million a year earlier, as a 69% slide in pipeline development revenue (to $23.885 million) outpaced the company’s nearly eight-fold increase in drug discovery revenue, to $24.952 million.

Notably, Insilico last year incurred a $352.5 million net loss, more than 20 times the company’s $17.1 million net loss in 2024, a jump the company attributed to the revenue drop as well as a $296.7 million loss from changes in the fair value of financial liabilities at fair value through profit or loss. That loss stemmed from Insilico converting the preferred shares issued in previous financings into ordinary shares when the company went public in December, raising HKD 2.277 billion (about $292.3 million at the time; now worth $283.9 million) on the Hong Kong Exchange.

Yet just as notably, Insilico’s cash and cash equivalents more than tripled last year, to $393.338 million.

“We look forward to achieving sustained profitability,” Alex Zhavoronkov, PhD, Insilico’s founder and CEO, said in a statement.

The forecasts continued the small but noticeable rise in Insilico’s stock price since Wednesday when the company announced that its lead candidate rentosertib, a drug designed to treat idiopathic pulmonary fibrosis (IPF), has advanced to a Phase III trial, the first drug within the company’s expansive 40+ program pipeline to reach that clinical milestone.

“Full arc of our mission”

“Rentosertib is a very important program for Insilico because it represents the full arc of our mission: using AI not only to move faster, but to originate new biology, new chemistry, and new therapeutic opportunities in aging and disease,” Zhavoronkov stated.

That news sparked a mini surge that sent Insilico’s shares climbing 19% over three days. Shares rose 2.7% from HKD$36.02 ($4.49) Tuesday to HKD$37 ($4.61) Wednesday, followed by a 7.7% gain Thursday as the stock rose to HKD$39.84 ($4.97)—then a 7.5% jump Friday, with Insilico closing the week at HKD42.82 ($5.34).

At least one investment firm started coverage of Insilico’s Hong Kong-traded stock with positive commentary: Cui Cui, an equity analyst with Jefferies, initiated the firm’s coverage with a “Buy” rating and 12-month price target of HK$100 ($12.47).

Jefferies’ endorsement capped a year in which Insilico escalated its partnership activity with pharma giants and smaller biotechs, adding roughly up to $7 billion to a potential haul that could exceed $10 billion.

The largest of these collaborations is its up-to-$2.75 billion collaboration ($115 million upfront) with Eli Lilly (NYSE: LLY), under which Insilico granted Lilly an exclusive global license to develop, manufacture, and commercialize “potentially best-in-class, novel oral therapeutics in preclinical development for certain indications,” according to an announcement that didn’t specify the therapeutic areas where the companies plan to partner. The alliance expanded from an “over $100 million” R&D partnership inked last November, which in turn grew from a 2023 licensing agreement allowing Lilly to access Insilico’s Pharma.AI software suite.

“Combining first-mover advantage, wet-lab validation, deep medical science, and in-house clinical expertise to train and refine AI, plus LLY’s endorsement, Insilico looks well positioned for scalable BD [business development] and LT [long-term] monetization,” Cui wrote in a research note.

“As AI-driven productivity cont[inues] to scale PCC [preclinical candidate] output, Insilico is positioned to expand its pool of proprietary assets, enhancing the likelihood of future out-licensing opp[ortunity] and strengthening LT monetization potential,” Cui added.

Zhavoronkov highlighted the research note on his LinkedIn feed, adding: “I think that in many ways the analysts know the industry and the company even better than some of the insiders. Definitely worth a read.”

Phase III plans

Cui’s comments followed Insilico announcing its Phase III plans for rentosertib (formerly ISM001-055), which is designed to treat 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.

Insilico said its planned Phase III trial (NCT07687459) will be a randomized, double-blind, placebo-controlled, parallel-group study that is expected to enroll 320 participants across 47 centers in China. The trial’s primary endpoint will be the annual rate of decline in forced vital capacity (FVC) over 52 weeks, with a key secondary endpoint of time to first occurrence of any disease progression event.

The Phase III trial aims to assess whether rentosertib can provide clinically meaningful benefit in a larger patient population and over a longer treatment period than its two 12-week Phase IIa studies.

Rentosertib has completed a Phase IIa trial (NCT05938920) in China, published in Nature Medicine last year, and is in a separate Phase II trial (NCT05975983) in the United States. In the Chinese trial, rentosertib met its primary endpoint of safety and tolerability across all dose levels, as well as positive secondary endpoint data, namely dose-dependent FVC improvement with a mean FVC change of +98.4 mL at 12 weeks in patients dosed at 60 mg once daily, vs. -20.3 mL for placebo.

During the BIO International Convention in San Diego, Zhavoronkov hinted at the Phase III, highlighting a planned “next step” for the program “in the second half, but maybe closer to the earlier second half,” he told GEN.

At the convention, Zhavoronkov led Insilico in celebrating its latest big-money collaboration, an up-to-$2.5 billion partnership with SK Biopharmaceuticals to discover new AI-based drug candidates for disorders affecting the neuroimmune area of the central nervous system (CNS). 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 is part of a privately held, family-owned chaebol or conglomerate whose parent holding company is public, SK Inc. (Korea Exchange: 034730). Another SK-owned company—SK Hynix (Nasdaq: SKHY), a supplier of high-bandwidth memory chips that power the AI processors of Nvidia and AMD—went public Friday, raising a staggering $26.5 billion by pricing its U.S. American depositary shares (ADS) at $149 each.

Potentially lucrative partnerships

In addition to SK and Lilly, Insilico also has potentially lucrative partnerships with Sanofi (Euronext Paris: SAN), with which Insilico plans to advance up to six targets (up to $1.2 billion); privately held Menarini Group, to which it has outlicensed Phase I cancer treatments targeting KAT6 and KIF18A (up to $1.05 billion in collaborations launched 2024 and 2025); privately held, French-based Servier, also cancer focused (up to $888 million); and Takeda Pharmaceutical (Tokyo Stock Exchange: 4502), drug discovery across its therapeutic areas (up to $600 million).

Also among Insilico’s collaboration partners: Exelixis (Nasdaq: EXEL), to which Insilico outlicensed in 2023 a Phase I BRCA-mutated cancer drug targeting USP1 (“close to” $1 billion plus royalties), Fosun Pharma (Shanghai Stock Exchange: 600196; Hong Kong Exchange: 02196), which is joining Insilico on R&D for four biological targets plus co-development of Insilico’s QPCTL program (up to $82 million, including $13 million upfront and a $15 million equity investment); Fosun-backed but privately held Hygtia Therapeutics, which is co-developing with Insilico ISM8969, a Phase I oral brain penetrant NLRP3 inhibitor, in CNS disorders (up to $66 million, including $10 million upfront and milestones); and Taipei-based TaiGen Biotechnology (Taipei Exchange: TWD), which holds Greater China rights to an oral PHD1/2 inhibitor in anemia of chronic kidney disease (milestones and royalties totaling “two-digit million dollars”).

Rounding out the list of Insilico’s disclosed collaboration partners are Chinese-based Qilu Pharmaceutical Group, which is partnering to jointly develop small molecule inhibitors for specific targets in cardiometabolic disease management (up to “near” $120 million, including milestones and single-digit royalties); China Medical System Holdings (CMS; Hong Kong Exchange: 867 and Singapore Exchange: 8A8), which is teaming up with Insilico on discovering drugs for central nervous system and autoimmune diseases (up to “tens of millions in Hong Kong dollars per project in R&D support”); and Tenacia Biotechnology, a Bain Capital-backed, privately held Sanghai-based drug developer which in March joined Insilico to expand a year-old R&D collaboration aimed at developing therapies for “underserved” neurological disorders (up to $94.75 million in near-term and milestone payments).

Insilico has out-licensed to undisclosed partners rights to a GLP-1R-targeting program designed to treat obesity and metabolic diseases; and Greater China rights to a Nav1.8-targeting program designed to treat pain.

Vaxart takes a double dose of good news

Settlement ends threat of proxy war; COVID-19 pill aces Phase IIb trial

This week’s annual shareholder meeting had threatened to be anything but routine for Vaxart (Nasdaq: VXRT) after its current executive team and three of its six nominees for board seats had been challenged by an activist shareholder through a proxy campaign.

Since last fall, shareholder Daniel P. Houle and allies have offered persistent criticism of Vaxart’s management—led by CEO Steven Lo and Sean Tucker, PhD, senior vice president and CSO—and the company’s board, whose operations and independent oversight are led by a lead independent director, W. Mark Watson, rather than a traditional chair.

But earlier this month, the threat of a proxy war over Vaxart’s direction ended when Houle and five allies signed a cooperation agreement with the company. Vaxart agreed to begin a search for an additional independent director to be conducted within 90 days of the conclusion of the 2026 annual meeting. Vaxart also agreed to work with Houle and allies to identify a “mutually agreeable” candidate for appointment to the board.

In return, the stockholder group consisting of Houle and his allies—Mark Silverberg, MD; Matthew M. Wallace, MD; Patrice Raffy; Marc Eustace Pereira; and Q3 Nominees Pty Ltd.—agreed to withdraw their board nominations for Houle, Silverberg, and Wallace.

The cooperation agreement also calls for:

Creation of a Stockholder Engagement Committee and a Clinical and Regulatory Affairs Committee
A revamp or “refreshment” of board committee chairs, including the selection of new chairs for the Nominating and Governance and Compensation Committees
Adoption of director stock ownership and resignation policies
Customary standstill, voting, engagement, and other provisions

“Vaxart is approaching a series of important value-inflection milestones, and these actions enable the company to move forward with a unified focus on executing its strategy,” Watson said in a statement. “We appreciate the constructive dialogue with the stockholder group toward our shared goal of creating value and are pleased to resolve our proxy contest so we can dedicate our full resources and attention to advancing our pipeline with stockholder interests in mind.”

Houle and allies insisted they believe “deeply” in the promise of Vaxart’s oral vaccine platform and resulting commercial opportunities—but took issue with the company’s declining stock price, capital raises that they said diluted the value of existing shareholders’ stock, and with what they termed insufficient oversight by the board.

In February, Houle launched his campaign to persuade shareholders to elect himself, Silverberg, and Wallace to Vaxart’s board. Silverberg is founder and CIO of Heatjac, a manufacturer of heated medical garments. Wallace is a double board-certified dermatologist and Mohs micrographic surgeon, and managing partner of a medical specialty practice focused on dermatology, dermatologic surgery, and oncology.

“We believe Vaxart possesses a unique technology platform with the potential to reshape vaccine delivery and transform global public health. Yet despite this promise, stockholder value has remained significantly compromised,” Houle and allies, calling themselves the Concerned Vaxart Shareholders, wrote in a June 9 letter to shareholders. “Despite these strengths, stockholders have endured years of disappointing performance, declining market value, and insufficient engagement from those entrusted to represent our interests.”

They also took issue with Vaxart’s two workforce reductions last year. The first was a 10% cut after Advanced Technology International, a nonprofit R&D collaboration manager acting on behalf of the U.S. Biomedical Advanced Research and Development Authority (BARDA), issued the first of two stop-work orders on the company’s Phase IIb trial assessing its government-funded COVID-19 oral pill vaccine. The second was a 21% cut in May–June 2025 intended to lower operating costs and better align Vaxart’s resources with higher-priority clinical programs.

“This election is not about creating conflict. It is about restoring confidence,” the Concerned Vaxart Shareholders added. “It is about restoring accountability, increasing transparency, and ensuring that stockholder interests are once again placed at the center of the company’s decision-making process.

Concerned Shareholders owned 1,515,343 shares of Vaxart stock—including 15,622 owned by Houle himself—as of a May 6 regulatory filing.

In an interview at the recent Biotechnology Innovation Organization (BIO) International Convention in San Diego, Lo and Tucker defended the company-endorsed board nominees as possessing greater biotech-related experience.

Lo defended the workforce cuts: “You want to be at the right size. You want to extend your runway. And we’re very careful with shareholder money. We don’t want to exhaust our funds. The reduction in the workforce was not only to extend our cash runway, but also make sure that this company had the right people to fulfill its mission.”

“Our case is, we have a very experienced management team. We have to stay the course,” Lo added. “We are in a great situation where we have good relationships with the U.S. government, as evidenced by being one of the only companies that has survived stop-work orders. We also have good relationships with pharma, as evidenced by our deal with Dynavax.”

Following a second stop-work order issued in August 2025, Vaxart and BARDA agreed to reduce funding for the Phase IIb trial to about $345 million from up to $453 million, but maintain the study at the estimated 5,485 patients recruited by Vaxart. In November 2025, Vaxart signed an up-to-$700 million global exclusive license for the oral COVID-19 vaccine with Dynavax Technologies, with Vaxart allowed to run the trial. Dynavax was acquired by Sanofi (Euronext Paris: SAN) for $2.2 billion, in a deal completed in February.

The cooperation agreement was one of two positive announcements Vaxart shared on July 6. The other was good clinical news: positive topline data from the approximately 400-participant sentinel safety cohort of its Phase IIb trial (NCT06672055) assessing the company’s oral pill COVID-19 vaccine candidate against an undisclosed approved mRNA vaccine comparator. Among key findings:

No vaccine-related serious adverse events (SAEs) or sustained Grade 3 or higher AEs were reported in either the oral pill vaccine or mRNA arms of the trial.
The most common AEs for oral vaccine patients were malaise/fatigue (20.9%), headache (18.9%), and anorexia (10.0%). Fewer than 10% of participants experienced any other AE.
By contrast, the most common AEs in participants receiving the mRNA vaccine were injection site pain (60.3%), injection site tenderness (40.2%), malaise/fatigue (35.2%), myalgia/muscle pain (33.2%), and headache (28.6%). Arthralgia, chills, anorexia, nausea, diarrhea, and induration/swelling at the injection site were experienced by between 10–15% of participants. Fewer than 10% of participants experienced any other AE.
Thirty-three participants in Vaxart’s oral pill vaccine arm and 30 in the mRNA vaccine arm had symptomatic COVID-19. Asymptomatic COVID-19 cases were reported in 12 participants in each of the trial arms.

“These topline safety data are encouraging and are consistent with the safety profile observed to date in other studies of our oral pill vaccine constructs,” stated James Cummings, MD, Vaxart’s chief medical officer.

Vaxart shares, which trade under $1, rose 16% from 55 cents on June 25 to 64 cents on July 2, the day of the filing disclosing the cooperation agreement. Since then, shares have given back the entire gain, sliding back to 55 cents at Friday’s close.

Leaders and laggards

Chemomab Therapeutics (Nasdaq: CMMB) shares tumbled 29% from $2.77 to $1.97 Wednesday after the developer of therapeutics for immune-fibrotic diseases with high unmet need said it agreed to merge with precision medicine developer Scipher Medicine through an all-stock merger. The combined company plans to operate under the Scipher Medicine name and trade on Nasdaq under the ticker symbol SCIP. Upon completion of the merger, the combined company plans to focus initially on advancing nebokitug, a first-in-class clinical-stage anti-CCL24 antibody, into a Phase II trial for the treatment of rheumatoid arthritis, Chemomab said. The combined company is valued at $150 million before a concurrent $30 million private placement from a syndicate of current Scipher investors led by Northpond Ventures, with participation from Khosla Ventures, Blue Owl Healthcare Opportunities, funds managed by Neuberger, and other leading investors, and is expected to have cash runway into the second half of 2028.

Forte Biosciences (Nasdaq: FBRX) shares rocketed 78% from $20.38 to $36.70 Thursday after the developer of treatments for autoimmune and autoimmune-related diseases announced positive results from the FB102 double-blind placebo-controlled Phase Ib study in vitiligo. FB102 achieved a 29.6% mean Facial Vitiligo Area Scoring Index (FVASI) improvement from baseline at week 24 (p-value = 0.020). Response to FB102 was seen early, Forte said, with statistically significant improvements observed by the day 64 visit (p=0.023), continuing through week 24, after completion of the 12-week treatment period. FB102 achieved 43.2% mean FVASI improvement from baseline at week 24 (p-value = 0.006) in subjects with greater disease involvement having baseline FVASI ≥0.75 (approximately one-quarter of face depigmented), including FVASI50 (58.8%) and FVASI75 (23.5%). Forte shares continued climbing Friday, rising another 20% to $43.92.

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Macrophage Membrane-Derived Nanoparticles Shows Potential Against Candida Infections

Edusehat — Sat, 11 Jul 2026 04:00:09 +0700

By using tiny particles made from the membranes of human immune cells, scientists from the University of California, San Diego and the University of Missouri have created antifungal nanoparticles that target Candida albicans, a fungus responsible for oral and vaginal yeast infections as well as bloodstream infections. Tests in mice with severe Candida infections show that the macrophage-derived nanoparticles reduced the amount of fungus in major organs, including heart, kidneys, lungs, and spleen. The mice also had improved survival rates.

Full details are published in Cell Biomaterials in a paper titled “Cell membrane-derived nanotherapeutic for combating Candida albicans infections.” In it, the scientists write that “this bioinspired nanodisc not only disrupts fungal membranes directly but also enhances host immune clearance, achieving potent antifungal activity.”

Current treatment options for fungal infections are limited and there are growing concerns about drug resistance. Existing medications typically target specific parts of a fungal cell and can lose effectiveness as fungi evolve resistance. The nanoparticles described in the current paper have a more potent strategy. Besides damaging fungal membranes, they also boost the body’s natural immune defenses to better fight infections.

According to the scientists, each nanodisc measures about 10-20 nanometers, about 1,000 times smaller than a normal macrophage. Their tiny size is an advantage as it allows them to fuse directly with fungal cell membranes and destabilize them, which is harder for full-sized macrophages.

To create the nanodiscs, the scientists isolated the outer membranes of the macrophages and broke up them up into tiny pieces. They then fused them onto disc-shaped nanoparticles made from a biodegradable polymer. Since the nanodiscs are built from macrophage cell membranes, they retain the same receptor proteins that the immune cells use to recognize and attack Candida. This means that the nanodiscs can identify and attach to fungal cells more effectively than those made from other cell types such as red blood cells.

Once attached, the nanodiscs weaken the fungal cell’s protective outer membrane until tiny openings form. As the membrane breaks down, the cell’s contents leak out while external substances seep in ultimately killing the fungus. Because this treatment strategy physically damages the fungal cell rather than targeting a specific molecule, the developers believe that it may be harder for the fungus to evolve resistance.

The nanodiscs also provide other countermeasures. They reverse the suppression of antifungal chemicals produced by macrophages during infection, and prevent Candida from forming biofilms that help to shield fungal cells from drugs and the immune system. Testing also revealed that the treatment was effective when administered both before and after infection suggesting that it could also be used as a preventative.

For their next steps, the scientists will further evaluate the antifungal potency of the nanodiscs against a broader range of pathogenic fungal species.

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Sperm donors need limits, says a European fertility group

Edusehat — Fri, 10 Jul 2026 17:20:03 +0700

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Ties van der Meer doesn’t know how many siblings he has.

The 47-year-old was conceived at a private fertility clinic in the Netherlands using sperm provided by an anonymous donor. After the Netherlands banned anonymous donation in 2004, the doctor who ran the clinic destroyed records that might have identified those donors, he says.

He describes the situation as “problematic.” Children have a right to know their biological parents, he says. While he did ultimately track down one sibling, who helped him identify his father along with other genetic relatives, he may have others he’ll never find.

Other donor-conceived people who have been able to track down siblings have found they have tens or even hundreds of them. One donor-conceived woman who found 25 half-siblings over the course of seven years told the Guardian, “It does make you feel a bit mass-produced.”

We need international limits on the number of children a single donor can contribute to, a European fertility organization argued yesterday. At a conference in London, members laid out plans to start with a Europe-wide limit.

Today many countries, including the UK, have banned anonymous egg and sperm donation. But anonymity can’t be guaranteed even in places where it is technically allowed. Genetic tests offered by companies like Ancestry and 23andMe, along with genetic registries, have made it much easier for donor-conceived people to find parents and siblings who share their genes.

And because sperm can be frozen and stored for years before it is eventually used, the current set-up can result in situations where donor-conceived people discover the identity of a genetic parent only after the person’s death. They might also find that they have siblings of very different ages, all around the world.

Some people are finding hundreds of siblings. Sperm from Jonathan Meijer, a Dutch man who began donating in 2007, was used to conceive between 550 and 600 children. (Stichting Donorkind, a foundation and advocacy group for donor-conceived people that’s chaired by van der Meer, took him to court, and he was ordered to stop donating in 2023.)

Stories like these can be distressing for donor-conceived people. And there are other reasons why limits are considered important. The offspring of a prolific donor might be at risk of unknowingly forming romantic or sexual relationships, for instance. And some people are concerned that a donor with a harmful genetic mutation might pass that down to many children.

This is unlikely, given the level of screening that most donors undergo. But it has happened. A man who donated his sperm to a sperm bank in Denmark was found to have a genetic mutation that significantly increased the risk of multiple cancers. But his sperm had already been used to conceive at least 197 children across Europe. Some of those children developed cancer. Some died.

Many countries already have legal limits for donors. In Malta and Cyprus, for example, both egg and sperm donors are allowed to contribute to the birth of just a single child, according to data presented at the European Society of Human Reproduction and Embryology (ESHRE) meeting in London on July 8.

Other countries set limits based on the number of families a single donor can contribute to, allowing recipients to have children who share a genetic link. In the UK, that limit is set at 10 families per donor.

But these limits are difficult to enforce, partly because donated gametes don’t necessarily stay in their original country. In Denmark, the national limit is set at 12 families. But the country is a major exporter of sperm. In the UK, for example, more than half of sperm donations in 2020 were imported—with most of those coming from either Denmark or the US.

“The only thing that really makes sense is a transnational limit,” Jackson Kirkman-Brown, a professor of reproductive biology at the University of Birmingham, said at the meeting.

Kirkman-Brown and his colleagues have spent months putting together a document that represents ESHRE’s position on these limits. After consulting with fertility specialists, clinics, sperm and egg banks, donors, and donor-conceived people, the team has developed a plan to start with a Europe-wide limit on sperm and egg donations.

ESHRE is calling on sperm and egg banks, as well as fertility clinics, to respect an initial limit of 50 families per donor. That’s still very high, according to a handful of people I spoke to at the meeting. But at least it’s a start.

Europe should move toward setting limits at 15 families per donor, Kirkman-Brown said. “We may find that 15 is also too high,” says Vasanti Jadva, who studies the psychological well-being of people conceived using donated eggs, sperm, and embryos at City St George’s in London. “We still don’t know what the right number is.”

It will be difficult to enforce these limits, too. And if they end up limiting the supply of donor sperm, there’s a chance that some people will turn to unregulated sperm donations from people who do not undergo health screening. Unregulated donations can lead to other problems for prospective parents, including the possibility that donors will seek parental rights over the children conceived using their sperm.

And it will be even harder to establish international limits. When I asked the American Society of Reproductive Medicine for its thoughts on ESHRE’s proposed limits, a representative directed me to a guidance document saying “it has been suggested” that for a population of 800,000, single donors should be limited to “no more than 25 births” in order to avoid the risk that relatives will have children together. (Considering the US has a population of over 340 million, the total figure could be pretty high, but many sperm banks opt to limit the number of families contributed to by a single donor at around 25.)

van der Meer thinks that even a limit of five families from a single donor would be high. International donation makes it even harder for donor-conceived people to connect with genetic relatives, so the limit for international contributions should be set at two families, he says.

Still, he thinks ESHRE’s suggested limit is a “positive first step.” Van der Meer has managed to track down a sibling, his father, and nephews, aunts, and uncles. He hopes that future policies respect the rights of donor-conceived children to know, and be in contact with, their genetic relatives.

“But,” he says, “you have to start somewhere.”

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.

Epigenomic Analysis Uncovers New AML Subgroups and Drug Sensitivities

Edusehat — Fri, 10 Jul 2026 10:05:09 +0700

As one of the most aggressive blood cancers, the way acute myeloid leukemia (AML) is classified continues to shape every major clinical decision—from risk stratification to the choice of targeted therapies. For decades, that classification has rested almost entirely on the gene mutations found in leukemic cells. But mutations alone have never fully explained why AML behaves so differently from patient to patient. A new study published in Nature now provides the missing layer: the epigenome.

In the largest chromatin‑profiling effort ever conducted for any cancer, a research team led by Seishi Ogawa, MD, PhD, and Yotaro Ochi, MD, PhD, of Kyoto University, together with Sören Lehmann, MD, PhD, of the Karolinska Institute, mapped the chromatin accessibility landscape of 1,563 AML patient samples. Their analysis—built on ATAC‑seq, RNA‑seq, DNA methylation, ChIP‑seq, whole‑genome sequencing, and single‑cell multiomics—reveals that AML can be classified into 16 distinct epigenomic subgroups, each defined by a characteristic chromatin state and its own regulatory wiring.

As the authors wrote, “ATAC-seq…show[s] that AML can be classified into 16 subgroups on the basis of chromatin accessibility profiles.” This chromatin‑based structure was remarkably stable: single‑cell ATAC‑seq across more than 280,000 cells confirmed that each patient’s leukemic population shares a conserved accessibility fingerprint.

Each subgroup carries a unique combination of driver mutations, differentiation states, transcription‑factor networks, DNA methylation patterns, and super‑enhancer architecture. Many do not align cleanly with existing genomic classifications such as WHO or ICC, according to the researchers. In fact, the team found that even exhaustive decision‑tree analyses of known driver mutations could not explain most subgroup identities. As the paper noted, “Evidence suggests that genetic alterations do not fully explain AML pathophysiology and heterogeneity.”

Clinically, chromatin information sharpened prognostic assessment in both Swedish and Japanese cohorts. Several subgroups also showed unexpected drug sensitivities. Three subgroups responded to MEK inhibitors despite lacking RAS‑pathway mutations. Another subgroup, enriched for RUNX1 mutations and marked by a chromatin profile resembling early B‑cell precursors, proved highly sensitive to ABL inhibitors.

The study, “Chromatin landscape and epigenetic heterogeneity of acute myeloid leukemia,” positions chromatin architecture as a foundational dimension of AML biology. It also provides a practical path toward clinical adoption: the team distilled a 30‑gene expression signature capable of identifying high‑risk chromatin subgroups using standard sequencing workflows.

Looking ahead, the group aims to develop lower‑cost diagnostic approaches and refine treatment strategies tailored to each epigenomic subgroup. The newly generated eCHROMA AML atlas is expected to serve as a resource for cancer epigenomics broadly, enabling discovery of new therapeutic targets and mechanistic insights.

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Engineered AAVs Harness Glymphatic System to Reach Brain Targets in Mice

Edusehat — Fri, 10 Jul 2026 06:30:11 +0700

Crossing the blood-brain barrier and avoiding off-target effects are two important challenges for gene therapies designed to target diseases of the brain. Now, developers of a gene therapy delivery platform that pairs specially engineered adeno-associated viruses (AAVs) with a delivery strategy that harnesses the brain’s fluid transport pathways claim that their approach addresses both issues and could help pave the way to new treatments for neurological disorders like multiple sclerosis, Huntington’s disease, and rare pediatric white matter disorders.

Details of the work were published recently in a Nature Biotechnology paper titled “Efficient targeting of human glial progenitor cells in vivo with engineered AAV vectors and glymphatic delivery.” The research was done by scientists at University of Rochester Medicine and the University of Copenhagen.

While the platform can deliver therapeutic genes broadly throughout the brain, it preferentially targets human glial cells. Steve Goldman, MD, PhD, lead author of the study and co-director of the Center for Translational Neuromedicine at URochester, has spent his career studying glial cells and elucidating their role in disease progression and recovery. Previously, his lab developed human glial progenitor cell models and investigated the link between glial dysfunction and neurological disease. For example, in Huntington’s disease, his lab has shown that healthy human glial progenitor cells could outcompete and replace diseased cells in the brain.

“Over the last decade, we’ve learned that many neurological disorders involve glial dysfunction as a major driver of disease,” Goldman said. “That realization has created an urgent need for tools that can safely and efficiently deliver therapies to these cells throughout the brain.”

The current study gets scientists one step closer to that goal. Digging into the details, Goldman and his colleagues engineered a library of modified AAV5 viral vectors by making small changes to the vectors’ capsids. They then screened the vectors in mice whose brains were transplanted with human glial progenitor cells and tracked their movements to identify which ones most effectively infected the human glial progenitor cells and their descendants including astrocytes and oligodendrocytes.

“Human cells display different molecular signatures than mouse cells, and cells behave differently in the brain than they do in a dish,” Goldman explained. “By selecting vectors under biologically relevant conditions, we were able to identify candidates with a strong preference for human glia.”

Next, the team turned their efforts to studying how best to distribute the AAVs throughout the brain. For that, they turned to the glymphatic system, the brain’s network of fluid-filled pathways used to clear metabolic waste by circulating cerebrospinal fluid through the brain. They delivered the engineered AAVs into the cisterna magna, a fluid-filled compartment at the base of the brain, while using hypertonic treatment to enhance fluid uptake into the network. This approach spread the vectors broadly throughout the brain tissue while largely avoiding the blood-brain barrier, and reducing exposure to peripheral organs like the liver.

“The glymphatic system is changing the way we think about brain drug delivery,” Goldman said. “Rather than trying to force therapies across the blood-brain barrier from the bloodstream, we can use the brain’s own transport pathways to distribute them more effectively where they are needed.”

Immediate targets for this approach are pediatric lysosomal storage diseases and other inherited disorders in which glial cells lack critical enzymes. Essentially, diseases of the brain’s white matter with well-defined biological targets. Further down the road, the approach could support novel therapies for multiple sclerosis, age-related white matter loss, and Huntington’s, among other neurodegenerative disorders where glial dysfunction is involved.

“We envision a future in which vectors can be designed for specific diseases and specific cell populations,” Goldman said. His lab is already exploring whether they can use artificial intelligence to design viral capsids that have specific targeting characteristics. “This study shows that by combining targeted vector engineering with glymphatic delivery, we can begin to build that future.”

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Crohn’s Disease Phage Therapy Neutralizes Inflammatory E. coli in Mouse Model

Edusehat — Fri, 10 Jul 2026 06:30:09 +0700

By taking advantage of the microbe-targeting capabilities of bacteriophages (phages), researchers at McMaster university have devised a supportive therapy for Crohn’s disease that disarms a type of E. coli bacteria that drives inflammation in the gut without disrupting the broader gut microbiome. The team showed that the phage therapy also improved responses to low doses of a conventional corticosteroid in a mouse model of Crohn’s disease, suggesting treatment could both improve outcomes and lower the risk of side effects from standard therapies.

Senior and co-corresponding author Elena F. Verdu, PhD, professor in the Department of Medicine and director of the Farncombe Family Digestive Health Research Institute, and colleagues reported on their study in Science Translational Medicine, in a paper titled “Phage intervention improves colitis and response to corticosteroids by attenuating virulence of Crohn’s disease–associated bacteria,” in which they noted “By reducing bacterial virulence mechanisms without substantially disrupting microbial balance, phage-based treatments align with personalized microbial therapeutics in IBD.”

Inflammatory bowel diseases (IBDs) such as Crohn’s disease and ulcerative colitis (UC) are chronic inflammatory conditions of the gastrointestinal (GI) tract that have strong microbial components, with patients often displaying disruptions in the delicate microbiome of the gut. “Recent studies have demonstrated that changes in the composition and function of the gut microbiota precede CD onset by up to five years,” the author wrote. However, they continued, “Current medications focus on suppressing symptoms and inflammation and can fail, in part, because they do not address underlying microbial drivers.”

Patients with Crohn’s disease tend to harbor strains of adherent-invasive E. coli (AIEC) that adhere to and invade epithelial cells in the intestines. “AIEC is distinct from other IBD-associated taxa because of its ability to adhere, invade, and survive in intestinal epithelial cells,” the team continued. Clinicians will sometimes prescribe antibiotics during symptom flares to try to target these bacteria, but antibiotics aren’t a long-term solution. They are non-specific and can perturb the microbiome even further, and with frequent use lead to side effects.

AIEC bacteria can be difficult to identify and selectively target, making them an important test case for more precise microbiome-based therapies. “One challenge is that AIEC are defined by what they do, not simply by how they appear in a microbiome analysis,” said Verdu. “To identify them, we need to test their behavior, such as their ability to adhere to and invade intestinal cells and persist in immune cells.”

Verdu, together with first author Kyle Jackson, PhD, at Farncombe Family Digestive Health Research Institute, and colleagues theorized they could navigate these roadblocks and neutralize virulent E. coli using bacteriophages. “Phages work like a lock-and-key system—each phage targets only certain bacteria. That precision gives us a way to intervene without wiping out the entire microbiome,” explained co-author Zeinab Hosseinidoust, PhD, associate professor in the Department of Chemical Engineering and the School of Biomedical Engineering.

The concept of using phages to target inflammatory bacteria is already being trialed, the authors noted in their paper. “Commercial phage ‘cocktails’ are presently being evaluated in clinical trials (NCT04737876 and NCT03808103) against proinflammatory bacterial taxa associated with IBD. These trials seek to target adherent-invasive Escherichia coli (AIEC), a group of bacteria enriched in patients with active CD compared with those in remission or healthy individuals.”

For their newly reported study the team developed a Crohn’s disease mouse model with a defined microbiome, and then screened a collection of phages, looking for those that could infect one strain of E.coli, NRG857c, isolated from patients with Crohn’s disease.

The results showed that their approach significantly reduced gut inflammation. The phages did not eliminate the bacteria entirely. Instead, they altered their behaviour by supressing a molecular “grappling hook” that helps AIEC attach to the gut lining and trigger immune responses. When that virulence mechanism was turned off, inflammation subsided.

One phage, designated HER259, countered the bacteria’s virulence by infecting it and switching off a genetic region named fimS. This region promotes the expression of FimH, which supports the bacteria’s ability to adhere to cells and trigger inflammation.

“HER259 ameliorated colitis in gnotobiotic models and attenuated the virulence of AIEC strain NRG857c, including suppression of the FimH adhesin through inversion of the fimS promoter to its ‘off’ orientation,” the authors wrote. “Withdrawal of HER259 treatment led to reversion of the fimS promoter and reactivated colitis.”

Hosseinidoust noted, “The bacteria were still there but they lost the traits that drive inflammation. We like to think of it as knocking out a few teeth. The bacteria can’t do as much damage anymore.” Withdrawing HER259 treatment led to reversion of the fimS promotor, and reactivated colitis, the authors noted.

The researchers also found that phage therapy enhanced the effectiveness of a commonly used steroid treatment for IBD, budesonide. When combined with the phage, a lower-than-standard dose of the drug produced benefits comparable to higher doses of the drug alone.

The HER259 phage in addition enhanced the therapeutic effect of subtherapeutic budesonide independent of microbial drug metabolism, the team wrote. While phages have previously been shown to increase the effectiveness of antibiotics, this is the first time a positive collaboration between phage and a non-antibiotic drug has been reported.

“In summary, using phage HER259 and NRG857c as a model phage–proinflammatory bacterium pair, we reveal a previously unknown mechanism by which targeted phage therapy disrupted key virulence markers in the CD-associated E. coli strain, reducing acute and chronic colitis, preventing reactivation, and enhancing therapeutic responses to budesonide.”

The findings point to a precision‑medicine approach for IBD. The bacterial function targeted by the phage can be measured in stool samples and was found to be higher in a subset of patients with Crohn’s disease, suggesting a potential way to identify those who could benefit most from this therapy.

“If we can identify which patients carry the harmful bacterial function, we could, in the future, intervene with a targeted therapy designed specifically to turn down that activity,” says Verdu. “This is what personalized medicine should look like: matching the right biological tool to the right patient,” added Hosseinidoust.

Next steps for the team include evaluating broader collections of bacterial strains from IBD patients and developing combinations of phages—work that brings the approach closer to human trials.

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Stem Cell Therapy Shows Promise in First Human Parkinson’s Disease Trial

Edusehat — Fri, 10 Jul 2026 06:30:08 +0700

A landmark Phase I/II clinical study led by researchers at Skåne University Hospital and Lund University has shown that transplanting stem-cell-derived dopamine progenitor cells into the brain is feasible. Eight patients with Parkinson’s disease (PD) received transplants of STEM-PD, a cryopreserved, off-the-shelf dopaminergic progenitor product derived from human pluripotent stem cells. The three-year Phase I/II, open-label, multicenter, single-arm, dose-escalation study identified no serious side effects linked to the transplanted cells during the first year of follow‑up.

“The possibility of replacing dopamine neurons that are lost in Parkinson’s disease has been a long-standing goal in the field,” said Malin Parmar, professor of cellular neuroscience at Lund University, and lead of the STEM-PD program. “The findings represent an important milestone for regenerative medicine approaches in Parkinson’s disease and support continued clinical development of stem cell-based therapies.”

Results from the study were reported by Parmar and colleagues in Nature Medicine. In their paper, titled “Human embryonic stem cell-derived dopaminergic cells for Parkinson’s disease: a Phase I/II open-label trial,” the team wrote, “In conclusion, particularly in the context of other recently published trials using human PS cell-derived dopaminergic cell therapies for PD, these findings further support the continued development of this therapeutic approach, including evaluation of the STEM-PD product in larger patient cohorts.”

Parkinson’s disease is the second most common neurodegenerative disorder after Alzheimer’s disease, the authors wrote. In Parkinson’s disease, patients lose nerve cells in the brain that produce dopamine, which leads to symptoms such as slowness of movement, stiffness, gait disturbance, and tremor. “The hallmark pathology of PD involves progressive degeneration of dopaminergic neurons in the substantia nigra pars compacta (SNpc) and the subsequent loss of their projections to the striatum,” the team explained. Current treatments are medications that replace the lost dopamine, but over time these medications often become less effective and cause side effects.

“Intracerebral transplantation of stem cell-derived dopaminergic progenitors to replace lost endogenous dopaminergic neurons offers a new potentially restorative therapeutic approach for PD,” the investigators continued. Scientists have been working to develop standardized and scalable dopamine cell therapy products derived from pluripotent stem (PS) cells, including human embryonic stem (ES) cells and induced PS (iPS) cells, they noted. “Several such products are now in clinical development … with early safety and feasibility data emerging.”

The transplanted stem cell-based dopamine nerve cell product tested in the newly reported trial is designed to replace the cells that produce dopamine, and the goal is that after being transplanted, the transplanted cells will mature into new dopamine-producing nerve cells in the brain. The STEM-PD trial aimed to evaluate the safety, tolerability, and feasibility of intraputaminal transplantation of STEM-PD in patients with moderately advanced PD.

Eight individuals with Parkinson’s disease received the transplanted cell product at two different doses, followed by 12 months of immunosuppression to prevent graft rejection. All patients were treated at Skåne University Hospital. Seven participants completed 12-month follow-up, and one participant died from a pulmonary infection that was not directly related to the cell product.

The surgical procedure was generally well tolerated, and no graft-induced involuntary movements were observed in the transplanted participants. Clinically, patients remained stable. Imaging using dopamine PET scans provided early indications of graft survival at both 6 and 12 months post-transplantation. Six of the seven participants substantially reduced their dopaminergic medication, a result that will be evaluated over time. In their paper, the team wrote in summary, “This Phase I/II clinical trial involving the bilateral intraputaminal transplantation of the STEM-PD dopaminergic progenitor cell product demonstrates its feasibility with no unexpected safety concerns from the cell product.”

Roger Barker, MD, professor of clinical neuroscience at the University of Cambridge, clinical lead of STEM-PD and clinical PI at the U.K. site, said: “This represents an exciting new departure on repairing the brain of individuals with Parkinson’s using dopamine cells- an approach pioneered in Lund some 40 years ago using fetal dopamine cells. The STEM-PD trial harnessing the expertise of scientists and clinicians from Lund and Cambridge has enabled us to undertake and deliver on one of the first ever stem cell-derived dopamine cell therapies for patients with Parkinson’s, and we hope this will be the beginning of an exciting new programme that may ultimately benefit the wider Parkinson’s community.”

Gesine Paul-Visse, MD, professor in neuropsychiatric research and lead PI at Skåne University Hospital, said, “Reaching this primary endpoint and being able to show that the cell product is safe is a great achievement for this trial, our team, the participating patients, but also for all patients suffering from Parkinson’s disease. We are hopeful that the early signs of cell survival and clinical improvement we observe will continue to increase over time and are excited to continue the development of this cell therapy.”

The STEM-PD research team will now continue the long-term follow-up of the participants to further evaluate safety, graft function, and clinical benefit. “Secondary and exploratory outcomes will evaluate the course and efficacy of clinical features, the survival of grafted dopaminergic cells at 36 months as well as additional safety signals occurring between 12 and 36 months and any dose–response effects,” the investigators stated. “Further evaluation of the grafts up to 36 months will determine whether the implanted cells continue to grow, mature and reinnervate the putamen after 12 months.”

STEM-PD builds on decades of research in dopamine cell replacement therapy for PD at Lund University and pioneering work in translation of pluripotent stem cell technology from experimental studies into clinical evaluation. The STEM-PD trial is the first pluripotent stem cell trial approved in Sweden and the first for PD in Europe. “The initiation and execution of this clinical trial have only been possible through close collaboration between scientists, clinicians, GMP manufacturing teams, regulatory experts and, most importantly, the participating patients,” concluded Parmar. In their report, the authors stated, “The STEM-PD trial adds further important confirmatory and complementary evidence to the recently reported feasibility and short-term safety of PS cell-derived dopaminergic progenitor transplantation in PD.”

The academic Phase I/IIa trial was conducted in collaboration with Novo Nordisk. Cellular Intelligence, a Boston-based company, recently acquired the STEM-PD program and will lead its next phase of clinical development, including a planned Phase II trial. The STEM-PD cells and their continued development hold IND clearance with FDA Fast Track Designation, and Cellular Intelligence aims to advance the program through Phase III to market approval.

STEM-PD is an academic European clinical translation initiative, focused on developing stem cell-based therapies for Parkinson’s disease. The program is led from Lund University with partners from Skåne University Hospital, Cambridge University Hospital, and University College London and combines expertise in stem cell biology, GMP manufacturing, neurosurgery, clinical neurology, and regenerative medicine to advance pluripotent stem cell-derived dopamine neuron therapies toward clinical application.

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Texas Biomed Enters Into a CRADA with U.S. Dept. of War to Help Protect Against Biological Threats

Edusehat — Thu, 09 Jul 2026 23:20:13 +0700

Texas Biomedical Research Institute signed a cooperative research and development agreement (CRADA) with the U.S. Department of War to help protect service members and civilians from emerging and high-consequence biological threats.

The agreement with the Capability Program Executive for Chemical, Biological, Radiological, and Nuclear Defense (CPE CBRND), which manages the nation’s investments in chemical, biological, radiological, and nuclear (CBRN) defense diagnostics and medical countermeasures (MCMs), establishes a collaborative framework to accelerate the development, testing, and validation of medical countermeasures while strengthening national preparedness, according to Cory Hallam, PhD, professor and executive vice president of Applied Science and Innovation at Texas Biomed.

“We are proud to offer our unique scientific capabilities to support the nation’s biodefense mission,” said Hallam. “Texas Biomed excels in reducing the time between scientific insight and operational impact, which is critical for proactive preparedness and responding quickly when threats emerge.”

Collaborative framework

The CRADA establishes a framework for Texas Biomed and CPE CBRND to collaborate on MCM projects that protect global populations, including U.S. soldiers serving at home and abroad. The agreement recognizes Texas Biomed’s operational impact capabilities, including high containment research and adherence to Good Laboratory Practice standards that support FDA regulatory requirements.

The Institute is also home to one of the nation’s seven National Primate Research Centers, offering expertise in translational studies and model development.

The CRADA framework is intended to ensure that critical resources for biological threats, whether from naturally emerging diseases or engineered risks, are continuously ready and available on demand, rather than episodically, continued Hallam.

“This is a long-term investment in resilience,” added Larry Schlesinger, MD, president and CEO of Texas Biomed. “As biological threats continue to evolve, partnerships like this are increasingly essential. Under this agreement, we have the potential to build new joint capabilities that will serve the nation not just today, but for decades to come.”

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Advanced Cell Sorting Balances Precision, Scale, and Simplicity

Edusehat — Thu, 09 Jul 2026 23:20:12 +0700

Within complex biological samples, the most important cells are often the hardest to find. Identifying, characterizing, and isolating rare or functionally-distinct populations has become central to modern cell biology, translational research, and cell therapy manufacturing. Among the most powerful tools for this task is fluorescence-activated cell sorting (FACS), a specialized form of flow cytometry that physically separates heterogeneous cell mixtures into defined subpopulations based on fluorescent labeling. Compared with methods such as magnetic-activated cell sorting (MACS), FACS offers higher-resolution analysis and greater flexibility for characterizing diverse, multi-parameter populations.

Flow cytometry itself measures the physical and chemical properties of individual cells as they pass single-file through a laser interrogation point. Although the technology emerged in the mid-1960s, the term “flow cytometry” replaced the earlier “pulse cytophotometry” in the late 1970s as fluorescence-based analysis and physical cell sorting became increasingly intertwined. Using fluorescently-labeled antibodies or probes, investigators can identify and isolate specific populations, including T cells, B cells, cancer cells, stem cells, and genetically-engineered cells.

As cell sorting applications continue to expand, instrument selection increasingly depends on experimental and manufacturing demands. Considerations include fluorochrome capacity, sterility requirements, aerosol containment, throughput, scalability, and budget. GEN spoke with industry leaders about how next-generation sorting platforms are addressing these evolving challenges.

Ease of use, compact size, and increased cell viability rank highly for cell sorting. Bio-techne’s Pala benchtop cell sorter combines microfluidics with gentle dispensing technologies. [Bio-Techne]

Single-cell isolation has traditionally relied on complex FACS systems or labor-intensive manual methods. These approaches can result in low cell viability, cross-contamination, and loss of cell integrity. Brendan Yee, director of cellular analysis at Bio-Techne, summarizes, “Traditional FACS sorters are expensive, difficult to use, and can take a significant amount of time to set up. Manual limiting dilution is based on Poisson distribution, where 60% of wells will be empty, about 30% will have one cell, and 10% will have multiple cells under the best circumstances.”

To address these challenges, Yee says the company developed the Pala platform, which integrates key components of traditional flow cytometry while focusing on being a faster and easier platform for single cell dispensing. “Within minutes, users can initialize the system, identify sorting parameters, and dispense single cells into a microtiter plate. The intuitive and simple software was designed so that all lab members could quickly learn to use the system.”

During operation, cells are loaded into a sterile, disposable microfluidic cartridge pressurized to less than two pounds per square inch (PSI;13.8 kPa, kilopascals). Yee reports, “Traditional flow cytometers are pressurized up to 35 PSI, which has been demonstrated to reduce viability, especially with sensitive cell lines such as induced pluripotent stem cells.”

Approximately the size of a desktop printer, the Pala cell sorter can be operated inside a tissue culture hood. The system is offered with a dual laser configuration, up to six photomultiplier tubes (PMTs) for fluorescence detection, and two photodiodes for light-scatter detection. Yee notes, “The use of a two-laser design allows for up to 11 different fluorophores to be used and therefore flexibility in the design of the experiment.”

Speed also matters for cell viability. After a brief sorting parameter set-up, a high-speed valve pushes target cells into a channel where a one-microliter droplet is dispensed into a microtiter well. For a 96-well microtiter plate, the process takes about two minutes and approximately six minutes for a 384-well plate.

The system simplifies single-cell applications, including cell line development, single-cell genomics, CRISPR editing, antibody discovery, and rare-cell isolation. Yee reports, “We are starting to see an expanded interest in the Pala platform for use with mass spectrometry and single-cell proteomics.”

Simplifying advanced sorting

As cell sorting moves beyond specialized core facilities into broader laboratory settings, a persistent challenge has been balancing performance with usability. This is particularly true when isolating rare or dim cell populations without further complicating operations. Instruments that deliver high sensitivity often demand extensive expertise, limiting adoption across multidisciplinary teams.

Cell sorters have gained popularity in more individual labs due to automated setup workflows and sizes that allow for installation on a benchtop. The CytoFLEX SRT from Beckman Coulter Life Sciences supports these needs while maintaining high sensitivity and sorting purity. [Beckman Coulter]

“The CytoFLEX SRT is designed to bring advanced cell sorting into a more routine, accessible, and automation-ready workflow,” says James McCracken, PhD, portfolio product manager, Beckman Coulter Life Sciences. Built on the CytoFLEX platform (i.e., the same underlying optical detection system, fluidics, and software architecture), the SRT system offers users the CytoFLEX analyzer’s high fluorescence and scatter sensitivity alongside a familiar software interface, enabling resolution of complex or low-abundance populations while supporting broader adoption and easier training.

McCracken also highlights, “A key advantage is that automation programming is built into the instrument, helping laboratories integrate the CytoFLEX SRT into automated workflows with less upfront complexity. The compact benchtop design supports integration into connected lab environments, with flexible sorting into tubes, slides, and multi-well plates. This helps researchers connect sorted cells to downstream applications such as culture, liquid handling, single-cell genomics, transcriptomics, proteomics, or functional testing.” Additional capabilities include up to 15 fluorescence parameters, four-way sorting, and the capability to support sort logic across multiple streams.

James McCracken, PhD
Portfolio Product Manager
Beckman Coulter Life Sciences

McCracken summarizes, “With automated setup and quality control features, the system helps reduce workflow complexity and support reliable, repeatable sorting. For scientists, that means less time spent managing the instrument and more time spent moving from complex cell biology to downstream insights.”

Managing biosafety risks

The expansion of cell sorting into translational and higher-risk biological applications has elevated the issue of biosafety alongside performance. Researchers must not only balance purity, yield, and viability, but also mitigate risks associated with aerosol generation and operator exposure, especially when working with unfixed or human-derived samples. These considerations can further complicate sorting procedures already requiring technical precision and specialized expertise.

Eric Diebold, PhD
VP and General Manager of Instruments and Informatics
Waters Biosciences

Eric Diebold, PhD, vice president and general manager of instruments and informatics at Waters Biosciences (formerly BD Biosciences), a division of Waters Corp, says the BD FACSAria Fusion cell sorter incorporates biosafety directly into the instrument. He explains, “With its fully integrated Class II biosafety cabinet and aerosol management design, FACSAria Fusion enables high-speed, high-purity sorting of unfixed or higher-risk samples while meeting stringent operator and sample protection requirements.”

According to Diebold, “The system is typically deployed in high‑complexity research environments such as academic core facilities, pharmaceutical R&D, and translational research labs, where maximum flexibility, high‑speed sorting, deep multicolor resolution, and integrated biosafety are required to confidently interrogate complex or rare populations.”

Combining high-parameter spectral flow cytometry with real-time imaging allows researchers to derive greater insights and enhance translational studies. Shown is the BD FACSDiscover S8 Cell Sorter [Waters Biosciences]

Other instruments include BD FACSAria III and FACSMelody systems. Diebold reports, “All are built on BD’s distinctive fixed-alignment, gel-coupled cuvette flow cell-based sorter that is a historical differentiator that delivers stream stability, high sensitivity, and day-to-day reproducibility beyond the stream-in-air sorters.”

Another innovative instrument is the BD FACSDiscover S8 cell sorter, which employs spectral flow cytometry (i.e., full-spectrum flow cytometry) to capture the entire emission spectrum of fluorochromes rather than specific wavelength bands. Diebold explains, “The S8 sorter represents a significant step forward by integrating spectral flow cytometry with real‑time imaging, enabling simultaneous measurement of phenotype, morphology, and spatial features at the point of sort. This added imaging context allows researchers to visually confirm cell populations, resolve heterogeneous or ambiguous subsets, improve doublet discrimination, and sort based on characteristics that extend beyond fluorescence alone—capabilities that are increasingly important for complex translational studies and early process development.”

Clinical manufacturing

Successful cell therapies begin with high-quality, functional cells. Thus, GMP-compliant cell sorting focuses on producing consistent, sterile cell populations suitable for clinical applications under tightly controlled regulatory conditions. Therapeutic applications include isolation of stem cells, CRISPR-edited cells, and T cells that will be engineered with chimeric antigen receptors.

Sudheer Gambheer, PhD
Global Product Manager
Miltenyi Biotec

“Clinical cell manufacturing demands the highest standards of safety, precision, and reliability,” notes Sudheer Gambheer, PhD, global product manager for flow cytometry cell sorting portfolio, Miltenyi Biotec. He continues, “The key point is that many of the traditional droplet-based FACS limitations become amplified and intertwined under GMP constraints.”

To address these challenges, the company has developed MACS^® GMP Tyto^® Consumables to enable GMP-compliant multiparameter cell sorting using the MACSQuant^® Tyto Instruments. Employing gentle, microchip-based sorting within a closed-cartridge system, the MACSQuant Tyto Family of cell sorters is designed to preserve cell viability and functionality. Gambheer reports, “In the closed cartridge, cells are kept sterile at all times while never coming into contact with the instrument.”

The system employs GMP-compliant consumables, including unique single-use cartridges in a closed environment. A microfluidic chip housing an ultra-fast mechanical valve (30,000 actuations/sec) lies at the heart of the technology. During sorting, cells flow through the microchip under low air pressure. Lasers detect target cells based on fluorescence and scatter as they flow through the microchannel. Non-target cells pass into the negative collection chamber. When a target cell is detected, a magnetic pulse activates a solenoid to open the valve, redirecting the cell into a positive-sorting chamber. The valve then resets, ready to isolate the next target cell.

From a regulatory perspective, Gambheer notes that the MACS GMP Tyto Consumables come with extensive supporting documentation for regulatory submissions. “Miltenyi Biotec is one of the only vendors providing an end-to-end workflow for easy integration into the GMP environment. This includes everything from GMP antibodies, buffers, and cartridges to the 21 CFR Part 11 software module, simplifying sorting compliance with secure electronic records and signatures.”

Future directions

As cell sorting continues moving from specialized core facilities into translational research and therapeutic manufacturing, future platforms will likely emphasize automation, richer cellular characterization, biosafety, and standardized workflows alongside sensitivity and throughput. Increasingly, the challenge is no longer simply identifying rare cells, but isolating them reproducibly, gently, and at a clinically relevant scale.

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Virtual Cells Go Multiscale to Predict Complex Biology

Edusehat — Thu, 09 Jul 2026 23:20:11 +0700

Virtual cell models that enable the prediction of cell behavior across scales and biological contexts are rapidly emerging at the forefront of drug discovery.

Tom Sercu, PhD, vice president of AI and engineering at Biohub, points to a clinician-scientist studying a rare autoimmune disease as an example of how AI models could reshape translational medicine. Starting from a patient’s genome, researchers could use virtual cells to predict how major immune cell types behave in disease versus healthy states. The result offers an invaluable tool across target and mechanism-of-action discovery, patient stratification, toxicity prediction, and therapeutic development.

Yet, building a virtual cell is not an easy feat.

“Transformative AI in biology does not come from algorithms alone, but when models are trained on large-scale, high-quality, openly accessible datasets,” says Sercu. To capture complex biology, such data must span model systems and organisms, interventional and observational methods, and diverse cellular states.

To support this mission, Biohub announced a $500 million commitment to the Virtual Biology Initiative in April. The five-year campaign will accelerate the generation of technologies and multi-modal datasets needed to power virtual cell models.

Similar to how more than 253,000 experimentally determined molecular structures in the Protein Data Bank (PDB), assembled over five decades, became foundational training data for modern AI protein-structure prediction, Sercu sees an analogous moment for cellular biology.

“We do not yet have the equivalent of the PDB for cells,” he emphasized. “The Virtual Biology Initiative seeks to change that.”

Today’s virtual cell developers reflect on what’s needed for these models to predict complex biology and overhaul drug discovery.

Single or bulk

Much of the industry has defined the virtual cell as transcriptome models that predict how perturbations alter gene expression across cellular contexts.

Among the increasingly crowded ecosystem, Arc Institute’s first-generation virtual cell model, STATE, predicts how stem cells, cancer cells, and immune cells respond to drugs, cytokines, or genetic perturbations. In March, billion-dollar-backed, Xaira Therapeutics unveiled X-Cell, the first scaling law demonstrator in the virtual cell domain, sizing up to a whopping 4.9 billion parameters. These models aim to generalize to unseen biological contexts by training on causal single-cell RNA sequencing (scRNA-seq) data.

To train X-Cell, Xaira has spent its initial years building what the company describes as “the largest genome-wide CRISPRi Perturb-seq dataset ever reported.” Named X-Atlas/Pisces, the dataset is composed of 25.6 million cells across seven screens and 16 biological contexts.

Ginkgo Datapoints, the AI platform division of Ginkgo Bioworks, looks toward bulk transcriptomics rather than a single-cell approach.

“Just like how models benefit from diversity in training data, we as an industry benefit from having diversity of approaches,” said John Androsavich, PhD, general manager at Ginkgo Datapoints. The Datapoints team applies high-throughput automation to create diverse biological datasets, including cell perturbations, antibody developability, and ADME small molecule developability data, to support AI model training for life science partners.

In March, Ginkgo Datapoints delivered the first data release of the Virtual Cell Pharmacology Initiative (VCPI). Approximately 2,280 small molecules were profiled in full dose response using DRUG-seq, a scalable arrayed transcriptomics assay measuring chemical perturbations.

In contrast to scRNA-seq, which covers approximately 1,500 genes per cell, DRUG-seq captures nearly 10,000 genes per condition with higher signal-to-noise to optimize insights for pharmacology. Notably, VCPI has exclusively focused on THP-1, a human monocytic cell line widely used across immunology, oncology, and inflammatory disease research, to understand drug action.

Across space

While the crowding around scRNA-seq has largely been driven by the pursuit of scale, “a cell is not only its RNA,” tempers Hani Goodarzi, PhD, core investigator at Arc Institute. He emphasizes that cells are complex systems shaped by multiple layers of biology beyond gene expression alone, including protein abundance, chromatin state, spatial organization, metabolism, and post-translational regulation.

A useful analogy comes from large language models (LLMs), which became powerful as text provided an exceptionally scalable substrate for training trillions of tokens. Yet, text alone is an incomplete representation of human communication.

“The lesson is not that one modality is sufficient forever,” says Goodarzi, “but that a single high-quality, scalable modality can support general representations when the training corpus is large.”

Emma Lundberg, PhD, co-founder and CSO at GenBio AI, is worried about the “streetlight effect.”

“We’re scaling what we can and not necessarily what we should,” she says.

GenBio AI seeks to develop world models that cross multiscale biology. Instead of concentrating on one data modality, the company’s so-called “AI-Driven Digital Organism” grows expertise in embedding, tokenizing, and training models across scales, from the molecular layer to regulatory networks. Rather than undergo internal data generation, GenBio AI focuses on public data and partnerships to power the company’s models.

GenBio Virtual Cell is a world model that enables biologists to explore cellular and molecular signatures, simulate how perturbations can reshape cell states across modalities and scales, and design small and large molecules for more precise targeting. [GenBio AI]

Lundberg, who is also associate professor of bioengineering and pathology at Stanford University, argues that models can guide the field to which data modalities to pursue. As an example, models that incorporate biological priors, such as protein-protein interactions, can achieve noticeable improvements in predictive performance.

Spatial and temporal data also capture critical dimensions of biological function that sequence data alone cannot resolve. According to the Human Protein Atlas, roughly 60% of human genes encode proteins that localize to multiple cellular compartments, often carrying out distinct functions depending on context.

In a May preprint posted on bioRxiv, Lundberg and colleagues introduced ProtiCelli, a deep generative model that visualizes the spatial organization of nearly the entire proteome within individual cells. By training on 1.23 million images from the Human Protein Atlas, the model simulates microscopy images for 12,800 human proteins while also generalizing to unseen cell types and drug perturbations absent from training.

Through time

Cellular Intelligence is developing a universal virtual cell signaling model designed to simulate cell-state transitions over time, with the goal of expanding the possibilities of regenerative medicine. By learning the underlying “grammar” through which sequences of signaling cues drive cell differentiation, these models aspire to enable the on-demand generation of any cell type.

Less than one percent of known human cell types can be reliably produced for downstream applications in cell therapy. As only 20 fundamental molecular signaling pathways give rise to thousands of cell states, researchers face an unfathomably large search space when engineering a particular cell type.

Cellular Intelligence CEO, Micha Breakstone, seeks to expand regenerative medicine by building a universal virtual cell signaling model to predict cell state. [Cellular Intelligence]

“Every cell that we discover or optimize opens a slew of potential applications,” said Micha Breakstone, CEO and co-founder of Cellular Intelligence. “One could spend a decade and tens of millions of dollars on painstaking trial-and-error to differentiate a new cell type, or solve this problem in one fell swoop, much like AlphaFold for the protein folding challenge.”

The company’s platform leverages a semi-permeable capsule technology, which selectively retains cells and large analytes while being freely accessible to media, enzymes, and reagents. The method enables high-throughput assays combining live-cell culture with genome-wide readouts. Millions of time-varying signal combinations are tested on human stem cell differentiation in parallel, providing 1,000 times higher efficiency than traditional methods.

In May, Cellular Intelligence advanced as a Phase II-ready clinical company after entering an agreement with Novo Nordisk to acquire STEM-PD, an allogeneic cell therapy program for Parkinson’s disease with Fast Track Designation. The deal comes six months after Novo announced its strategic exit from the cell therapy space. The start-up’s AI cell signaling models will address protocol development, one of the biggest obstacles preventing cell therapies from clinical impact.

“Novo selected Cellular Intelligence as the right partner because the next major challenge for complex cell therapy programs is not only the biology,” says Breakstone. “It is manufacturing scale-up, comparability, clinical logistics, and commercial readiness.”

As the diversity of virtual cell models targets new dimensions of complex biology, every approach takes another step closer toward clinical impact.

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Top 10 Best-Selling Drugs 2026

Edusehat — Thu, 09 Jul 2026 23:20:10 +0700

Thanks to booming sales of blockbuster obesity and diabetes treatments, the amount that Americans spent on prescription drugs this year is on track to surpass $1 trillion, according to a report released in April by the American Society of Health-System Pharmacists (ASHP).

That would be a sharp, above-inflation 9.3% increase from the $915 billion in U.S. drug sales recorded last year, one of the fastest one-year percentage jumps ever recorded by the group. That 12.7% jump is higher than the one-year increases achieved by healthcare costs, and growth in the overall economy.

Even more eye-opening: Nearly one-third of prescription drug spending growth came from the $132 billion spent on a single category, namely glucagon-like peptide 1 (GLP-1) receptor agonists indicated for obesity/wight management and for type 2 diabetes.

“GLP-1s have fundamentally reshaped the drug-spending landscape,” Eric Tichy, PharmD, MBA, lead author of the report and division chair of supply chain management at Mayo Clinic. “And we are still on the steep part of the curve.”

According to ASHP, the 2025 and projected 2026 leaps in drug spending reflect more medications being used by more patients, with spending growth being predicted to range from 10–12% overall.

That would appear to explain the robust increases in sales by every one of the top 10 best-selling prescription drugs (based on 2025 sales) featured in this GEN A-List. Top-selling drugs are ranked based on sales or revenue reported for 2025 by biopharma companies in press announcements, annual reports, investor materials, and/or conference calls. Each drug is listed by name, sponsor(s), 2025 sales, 2024 sales, and the percentage change between those years.

The total 2025 aggregate value of the top 10 best-selling drugs was $188.136 billion, up 21.5% from $154.888 billion in 2024—and more than double (up 127.5% over 10 years from the $82.694 billion generated by 2016’s top 10 sellers, as reported by GEN.

Just missing the top 10 at #11 was Novo Nordisk’s Wegovy^®, the glucagon-like peptide 1 (GLP-1) receptor agonist indicated for weight loss in obese or overweight adults. Treatments ranked number 11 through number 15 in 2025 generated between approximately $8.4 billion and $12.2 billion in revenues. In addition to Wegovy, best sellers Nos. 12-15 include:

Opdivo^® and its subcutaneous injection version Opdivo Qvantig (nivolumab / nivolumab and hyaluronidase-nvhy), marketed by Bristol Myers Squibb (BMS) worldwide except Japan, South Korea, and Taiwan, where Ono Pharmaceutical markets the drug.
Trikafta^®(elexacaftor/tezacaftor/ivacaftor and ivacaftor) from Vertex Pharmaceuticals, which markets the drug outside the U.S. as Kaftrio^®.
Ocrevus^® (ocrelizumab) from Roche and its U.S. subsidiary Genentech.
Farxiga^® (dapagliflozin), from AstraZeneca, which markets the drug outside the U.S. as Forxiga^®

Three drugs that ranked between #11 and #15 on last year’s A-List based on 2024 sales placed lower this year: Eylea/Eylea HD (aflibercept) from Regeneron Pharmaceuticals and Bayer, which ranked No. 17 in 2025 sales; Gardasil/Gardasil 9 (Human Papillomavirus Quadrivalent (Types 6, 11, 16, and 18) Vaccine, Recombinant/Human Papillomavirus 9-valent Vaccine, Recombinant) from Merck & Co., now No. 27; and Humira^® (adalimumab) from AbbVie, which had long been the top-selling drug for years until being surpassed by Keytruda^® but is now No. 32.

Eylea and Humira now face competition from biosimilars, while Merck halted Gardasil shipments to China last year, citing declining sales.

1. Keytruda^® / Keytruda Qlex ¹

(pembrolizumab / pembrolizumab and berahyaluronidase alfa-pmph) Merck & Co.

2025 Sales: $31.680 billion ¹

2024 Sales: $29.482 billion

% Change: +7.5%

2. Mounjaro^®

(tirzepatide) Eli Lilly

2025 Sales: $22.965 billion

2024 Sales: $11.540 billion

% Change: +99.0%

3. Eliquis^®

(apixaban) Bristol Myers Squibb and Pfizer

2025 Sales: $22.404 billion ($14.443 billion BMS + $7.961 billion Pfizer)

2024 Sales: $20.699 billion ($13.333 billion BMS + $7.366 billion Pfizer)

% Change: +8.2%

4. Ozempic^®

(semaglutide) Novo Nordisk

2025 Sales: $19.611 billion (DKK 127.089 billion)

2024 Sales: $18.570 billion ² (DKK 120.342 billion)

% Change: +5.6%

5. Dupixent^®

(dupilumab)³ Sanofi and Regeneron Pharmaceuticals

2025 Sales: $18.124 billion (€15.714 billion)

2024 Sales: $15.077 billion ⁴ (€13.072 billion)

% Change: +20.2%

6. Skyrizi^®

(risankizumab-rzaa) AbbVie

2025 Sales: $17.562 billion

2024 Sales: $11.718 billion

% Change: +49.9%

7. Darzalex^® / Darzalex Faspro^®

(daratumumab / daratumumab and hyaluronidase-fihj) Johnson & Johnson and Genmab ⁵

2025 Sales: $14.351 billion ⁵

2024 Sales: $11.670 billion ⁵

% Change: +23.0%

8. Biktarvy^®

(bictegravir, emtricitabine, and tenofovir alafenamide) Gilead Sciences

2025 Sales: $14.334 billion

2024 Sales: $13.423 billion

% Change: +6.8%

9. Jardiance family

(empagliflozin, monotherapy and in combinations with linagliptin and metformin) ⁶ Boehringer Ingelheim and Eli Lilly

2025 Sales: $13.563 billion ($10.132 billion [€8.785 billion] Boehringer Ingelheim + $3.431 billion Eli Lilly] ⁶

2024 Sales: $12.979 billion ($9.638 billion [€8.357 billion] Boehringer Ingelheim + $3.341 billion Eli Lilly) ⁶

% Change: +4.5%

10. Zepbound^®

(tirzepatide) Eli Lilly

2025 Sales: $13.542 billion

2024 Sales: $4.926 billion

% Change: +174.9%

References

Starting in 2025, Merck combined into a single figure the sales of Keytruda (pembrolizumab) and Keytruda Qlex, a subcutaneous injectable immunotherapy consisting of pembrolizumab and berahyaluronidase alfa, and which like Keytruda is indicated to treat multiple types of cancer.
Figure differs from the $18.655 billion reported by GEN in last year’s A-List of Top 10 Best-Selling Drugs due to currency fluctuations.
Sanofi records global net product sales of Dupixent, with each company recording its half-share of profits on global sales of the drug.
Figure differs from the $15.125 billion reported by GEN in last year’s A-List of Top 10 Best-Selling Drugs due to currency fluctuations.
All sales figures are recorded by Johnson & Johnson, with Genmab receiving royalties on worldwide sales from J&J. Genmab does not disclose specific royalty revenues for Darzalex and Darzalex Faspro but has furnished a 2025 royalty figure for the treatments of $2.443 billion, up 12.5% from DKK 13.922 billion ($2.172 billion) in 2024. Genmab changed its reporting and functional currency to U.S. dollars from Danish kroner as of 2025.
Lilly includes revenues from Glyxambi^® (empagliflozin/linagliptin), Synjardy^® (empagliflozin/metformin hydrochloride), and Trijardy® XR (empagliflozin, linagliptin, and metformin hydrochloride) in its revenue figures for the Jardiance family—which includes net product revenue as well as collaboration and other revenue.
Figure differs from the $12.832 billion in 2024 sales reported by GEN in last year’s A-List of Top 10 Best-Selling Drugs due to currency fluctuations.

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Ag Genomics Begins to Bear Fruit

Edusehat — Thu, 09 Jul 2026 23:20:09 +0700

Agrigenomics is, to use an analogy, spring looking toward summer. Wielding modern genomics tools fine-tuned to agriculture, along with AI-driven insights from field technologies and satellites in low Earth orbit, traditional agriculture is transitioning to precision farming and the bounty it brings. Insights are compressing 30 years of field trials into a single growing season and enabling growers to express specific traits without introducing foreign genes and without hit-and-miss Mendelian hybridization.

This intensive application of science starts with comprehensive soil analyses and extends, not only to the major row crops, but also to such lesser-studied areas as fruits and forestry.

It’s all about the soil

Nate Kelly
CEO, Miraterra

Healthy, productive crops all need a good growing environment. “Soil is the most complex, beautiful system on the planet,” Nate Kelly, CEO, Miraterra, points out. Yet, “The soil measurement system in place today is quite archaic,” with manual systems and complex chemistries.

Miraterra’s lead technology is the Digitizer. With shifted-excitement Raman difference spectroscopy as its core, it also employs lasers and advanced signal processing to remove the noise that previously made Raman spectroscopy untenable for soil samples. Robust modeling transforms those data into a molecular fingerprint of soil to 30 ppm, with significantly enhanced resolution anticipated. The Digitizer shortens analysis time from hours to minutes, and makes toxic chemical analysis unnecessary. “Today, we measure texture, organic matter, and carbon and—soon—nutrients,” Kelly says.

After acquiring Trace Genomics and its large soil microbiology data set last year, the company can identify approximately 400,000 different biological components of soil, “things like nitrogen fixers, phosphorus solubilizers, pathogens, and nematodes,” Kelly says.

Miraterra Fill Tray. Healthy crops begin with deep knowledge of soil’s chemistry, biology, and structure. [Miraterra]

As its capabilities increase, Miraterra plans to measure soil chemistry, texture, and biological content. It already accesses public data, satellite imaging, and LiDAR to develop reports, such as nutrient flow, on plots as small as 10 square meters. By adding AI to identify patterns predictive of pest infestations and disease prevalence, he says the company expects to correlate soil health with plant conditions and pathogens.

“Soil is too complex [chemically and biologically] to stick a simple probe in the ground and try to get a reading,” Kelly says. “My aspiration is to cut the cost [of soil analysis] by about 90%, to democratize measurement,” Kelly says. “That means turning a $40 analysis into one that costs less than $5.”

CRISPR: Beyond corn and soy

CRISPR technology is being applied to agrigenomics with the same revolutionary results it created for medicine. As the basis of Pairwise’s Fulcrum Platform, it is expanding agrigenomics well beyond the major crops of corn and soybeans.

Ryan Bartlett, PhD
CTO, Pairwise

Fulcrum technology produced the world’s first seedless blackberry—now in field trials—and a more compact blackberry plant that is commercialized in the Americas. With this compact Fontana blackberry, “Growers can plant more blackberry plants per acre, which helps them be more efficient and produce more yield,” Ryan Bartlett, PhD, CTO, says. A thornless blackberry plant is on the horizon.

The goal is to “make growers more sustainable and efficient, and to give consumers the opportunity to consume more healthy things, like blackberries,” Bartlett says. Fast, precision editing will also help plant breeders quickly adjust to climate change.

Fulcrum features plant-specific CRISPR editing that fine-tunes gene expression rather than simply turning genes on or off. The platform includes additional gene editing tools, enzymes, and trait libraries to help breeders design and test changes and quickly advance specific phenotypes into field trials.

Bartlett calls this “traditional breeding on an accelerated basis.” With Fulcrum, these new blackberries were produced in about three years. Using traditional Mendelian breeding would have taken 10 to 40 years.

Genomics tools coupled with advanced analytics enable growers to plant more per acre. [Pairwise]

The speed comes from genome editing that generates the desired phenotype in one cycle, eliminating the need for generations of crosses to develop the features growers want.

Pairwise is also working with “additional permanent crops like cherries,” Bartlett says. “There haven’t been a lot of advancements in the past 50 years.” That may soon change, as Pairwise is working with Sun World to develop a pitless cherry.

New opportunities

“Accelerating timelines and lowering the cost of crop improvement [will] unlock expansive opportunities in agriculture, new species, new geographics, and new traits,” Brad Zamft, PhD, CEO and co-founder of Heritable Agriculture, says.

Heritable focuses on breeding indoor vegetables and forestry, although it also develops fruits and row crops with strategic partners. “The majority of the unmet need comes from placing existing plants in the right places and determining what crosses would make them better,” Zamft says.

Brad Zamft, PhD
CEO, Heritable Agriculture

Consider strawberries. “We think we’re going to bring a new strawberry variety to the market—start to finish—within four years. The status quo is around a decade.” Heritable can identify desired traits, determine the genes involved, and validate them in growing plants within 18 months, versus 3 to 12 years using traditional methods. Currently, Heritable is adapting genetics from Consorzio Italiano Vivaisti’s (CIV’s) strawberries to grow indoors in Canada.

To speed the process, Heritable uses digital field trials and an AI engine developed in-house specifically for agrigenomics. Potential applications are global. “We have scalable integration of weather and soil to 10-meter resolution anywhere in the world,” Zamft says.

Increasingly accurate correlations of genes to phenotypes shorten the development timeline, Zamft adds. For example, three of the top eight genes Heritable’s team discovered for flowering time in corn proved causative, and three of three in a flavor project for leafy greens. In contrast, he cited a literature analysis that reported that out of 1,671 unique genes that were field-tested across multiple traits, only 22 were identified as validated leads.

To produce and commercialize these innovations, Heritable relies on an alliance of partners in which each partner brings vital strengths to the endeavor, from genomics through production, distribution, and sales. The company also engages in trait development with seed developers and growers, and recently launched a software-as-a-service platform to enable users to predict plant performance under multiple variables.

The company is also active in commercial forestry, using AI to help breeders make placement and breeding decisions that could shorten the breeding cycle by more than half.

Gene editing = no GMOs

Verinomics built its foundation on plant genomics, computational biology, and trait mapping to enable breakthroughs in multiple crops. Most recently, it developed Nonpareil+, a self-pollinating Nonpareil almond variety that may increase per-acre productivity by 30%.

That anticipated productivity gain is directly tied to self-pollination. Because the Nonpareil variety—which is the dominant commercial almond variety in California—is self-incompatible, it requires cross-pollination. That, in turn, requires planting alternating rows of other almond varieties as pollinators and then renting beehives (at about $400 per acre) to pollinate the orchard.

Stephen Dellaporta, PhD
Founder, Verinomics

“There’s no way to create a self-compatible Nonpareil using conventional breeding,” Stephen Dellaporta, PhD, founder of Verinomics and professor at Yale University, points out. Therefore, using Verinomics’ Genesis gene editing platform, “we identified the self-incompatibility gene and edited it to a self-compatible gene.” The resultant Nonpareil+ is genetically identical to the Nonpareil except that it is self-pollinating.

Growers are beginning to plant it this year, with the expectation of reducing or eliminating the need for multi-variety almond orchards and bee hives. If it works as expected, growers can harvest all the trees at once, rather than needing separate harvests for multiple varieties.

Almonds aren’t the only crop on Dellaporta’s mind. Back in the lab, “we just completed 60 whole genome sequences, assemblies, and annotations,” he says. The company works with partners to address concerns in multiple crops, making adaptations without introducing foreign DNA.

The bacterial disease known as citrus greening is an example. It’s killed millions of acres of citrus trees, according to the U.S. Department of Agriculture. “There’s no known resistance within the domesticated citrus germplasm,” Dellaporta says. Crossing a wild-type, resistant citrus with a domesticated citrus would take decades to produce a resistant, domesticated citrus, he points out. “Gene editing may create a modification to allow the plant to be resistant without losing varietal identity.”

Precision farming can bring incredible benefits to agrigenomics, just as precision genomics is doing for medicine. As agrigenomics begins to bear fruit, developers can look forward to a genomics-fueled bounty very, very soon.

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Fujifilm Selected with Six Others in FDA PreCheck Pilot Manufacturing Program

Edusehat — Thu, 09 Jul 2026 05:20:10 +0700

Fujifilm Biotechnologies reports that its commercial-scale cell culture manufacturing facility in Holly Springs, NC, has been selected as one of only seven participants in the FDA’s PreCheck Pilot Program. The program, which also includes Amneal Pharmaceutical, Cellares, Eli Lilly, Kriya Therapeutics, Kyowa Kirin, and Regeneron, is a strategic initiative designed to strengthen America’s pharmaceutical manufacturing capabilities and help accelerate patient access to critical medicines.

“We are honored that our Holly Springs site has been selected to participate in the FDA’s PreCheck Pilot Program,” said Lars Petersen, president and CEO of the company. “We’re committed to helping our customers bring life-changing therapies to patients faster, and participation in this initiative will help support greater access to critical medicines in the U.S.”

The pilot program is designed to enhance FDA-industry engagement by facilitating earlier interactions to minimize uncertainty associated with manufacturing readiness, with the goal of creating a more efficient regulatory review process, and strengthening the resilience of the U.S. pharmaceutical supply chain. The FDA PreCheck Pilot Program will focus on manufacturing readiness, regulatory predictability, and expedited facility inspections.

Fujifilm’s customers at the Holly Springs site includes argenx, Johnson & Johnson, Regeneron, and a number of other pharma firms. On behalf of its customers, Fujifilm’s Holly Springs site manufactures monoclonal antibodies (mAbs), including treatments for complex diseases.

The pilot program is designed to enhance FDA-industry engagement by facilitating earlier interactions to minimize uncertainty associated with manufacturing readiness. [Westend61/Getty Images]

“As our customers continue advancing innovative biologic therapies, manufacturing readiness and regulatory predictability are increasingly important,” said Laurie Braxton, senior vice president and site head, Holly Springs at Fujifilm. “Our participation in the program reinforces our commitment to providing customers with high-quality manufacturing capabilities.”

Fujifilm’s Holly Springs facility is one of North America’s largest end-to-end cell culture biopharmaceutical manufacturing facilities and represents a key node in the company’s growing global manufacturing network, according to Lars Petersen. The $3.2 billion manufacturing site opened with a capacity of 8 x 20,000 L mammalian cell culture bioreactors, and will add a drug product line in early 2027, followed by Finished Goods. An expansion is underway to double drug substance capacity with an additional 8 x 20,000 L bioreactors.

With an increase in demand for U.S. manufacturing capacity, Fujifilm officials say they will accelerate the opening of its expansion by six months, targeting late FY2027. The Holly Springs site recently surpassed 800 employees, with the overall goal of hiring a total of 1,400 local employees by 2031.

Designed with standardized platforms and advanced digital capabilities, the site is part of the company’s interconnected kojoX^TM operating system, which harmonizes systems, equipment, and processes across global sites to enable faster technology transfer, greater manufacturing flexibility, and consistent quality for customers worldwide

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Epitope Editing Strategy Could Enable Less Toxic Stem Cell Transplants

Edusehat — Thu, 09 Jul 2026 05:20:08 +0700

Stem cell transplantation and gene therapy are among the most powerful curative approaches for blood diseases such as sickle cell disease, β-thalassemia, immune deficiencies, and some blood cancers. Replacing or correcting the blood-forming stem cells can offer the possibility of long-lasting benefit or a cure. However, before patients can receive these therapies, they usually need intensive and potentially toxic chemotherapy or radiation to clear space in the bone marrow for the new stem cells.

Researchers headed by teams at Boston Children’s Hospital and Dana-Faber Cancer Institute have developed a new strategy to make stem cell transplants safer by replacing chemotherapy-based treatment with a more targeted approach. Instead of using toxic agents that damage DNA throughout the body, the team developed an donor stem cell epitope-editing strategy and antibodies that recognize surface markers only on the blood-forming stem cells that need to be depleted. Reporting on their developments in Nature (“Non-genotoxic transplantation and in vivo selection through epitope editing,”) the team demonstrated that these antibodies can help clear the patient’s existing stem cells from the bone marrow in a more selective, less toxic way.

Hematopoietic stem/progenitor cell (HSPC) transplantation (HSCT) is a cornerstone therapy for a wide range of malignant and non-malignant conditions, “… leveraging the unique regenerative capacity of HSPCs to replenish the hematopoietic system,” the authors wrote. However, they pointed out, the short-term and long-term effects of pre-transplant genotoxic conditioning represent real barriers to broader use of HSPC transplantation and gene therapies.

Although monoclonal antibodies have been proposed as alternatives to chemotherapy or radiotherapy, they are also associated with efficacy and safety challenges, the team noted. An antibody normally cannot distinguish between the patient’s original stem cells and the infused therapeutic stem cells from the treatment. If the antibody remains in the body, it may also attack these transplanted cells, preventing them from integrating. “… immune-based agents pose efficacy and safety challenges due to nonselective targeting of transplanted HSPCs and prolonged half-life, leading to on-target depletion,” the investigators stated.

Researcher Pietro Genovese, PhD, of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and his team solved this problem by giving the therapeutic stem cells a form of molecular protection. Using precise genome-editing tools, they made changes to a tiny recognition site—epitope—on the surface of the donor stem cells. This small change prevented the antibody from binding to the therapeutic cells, while preserving the normal function of the protein. “… we identified amino acid changes in the extracellular domain of KIT that disrupt the binding of two therapeutic monoclonal antibodies, which impair stem cell factor (SCF)-mediated signaling without affecting KIT expression or functionality,” they explained.

The edited stem cells were in effect given a molecular camouflage. They could hide from the antibody, while the unedited cells remained vulnerable. In previous work the team had used the same general principle of epitope editing to protect healthy blood stem cells from cancer immunotherapies, such as CAR T cells or therapeutic antibodies, while allowing those therapies to attack leukemia cells.

The newly reported approach included therapeutic editing of blood stem cells to increase fetal hemoglobin (HbF), a protective form of hemoglobin that can compensate for the defective adult hemoglobin found in sickle cell disease and β-thalassemia. “In our experiments, KIT and BCL11A were efficiently co-edited in primary HSPCs, endowing their progeny with both HbF induction and mAb resistance,” the investigators commented. The results showed that the protected, epitope-edited stem cells can survive antibody treatment, integrate in the bone marrow, and enrich gradually over time. The findings point to a new way to make room for transplanted cells, and also selectively favor the therapeutic cells after transplantation.

“By avoiding chemotherapy, we can open up stem cell transplants for diseases that are less severe or for fragile patients normally too sick or too high risk for transplantation,” said first author Gabriele Casirati, MD, an instructor in Genovese’s lab. “Typically, bone marrow transplants are reserved for patients with life-threatening diseases but are simultaneously limited to those patients who can tolerate the chemotherapy.”

This work could have implications for the future of both stem cell and gene therapy. First, it may help enable chemotherapy-free or chemotherapy-sparing transplantation approaches, reducing the burden of treatment for patients who currently face the risks of DNA damage. Second, because the antibody can continue to select for protected cells after transplantation, the strategy could help therapeutic stem cells reach the levels needed for clinical benefit. “In conclusion, our findings support the paradigm-shifting potential of epitope editing to design next-generation HSCT,” the team stated.

The broader significance extends beyond inherited blood disorders. Together, these studies suggest that epitope editing could become a flexible platform: one application could make stem cell transplantation and gene therapy safer, while another could expand the use of cancer immunotherapy by protecting normal blood formation from unintended damage. “… by overcoming the limitations of monoclonal antibody pharmacokinetics, epitope editing enables novel hematopoietic replacement regimens that are not limited by on-target graft elimination, allowing prolonged immune-based conditioning that maximizes hematopoietic niche clearance without chemo-radiotherapy or monoclonal antibody wash-out,” they noted.

“Although this work is still preclinical, it points toward a future in which patients may receive curative stem cell therapies with less toxicity, less reliance on chemotherapy, and greater precision,” said Genovese. “By combining targeted biological conditioning with molecularly protected therapeutic stem cells, this strategy offers a new framework for safer and more accessible treatments for a wide range of blood diseases.”

The technology used in this study is jointly owned by Boston Children’s and Dana-Farber Cancer Institute. In their paper the team commented, “We envision a future where patients receive life-saving stem cell therapies without risks of prolonged aplasia, infertility or secondary malignancies, and with minimal or no hospitalization.”

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Oligonucleotide Therapeutic Industry Readies Itself to Move to the Next Phase

Edusehat — Thu, 09 Jul 2026 01:45:15 +0700

Synthetic oligonucleotide therapeutics are now validated for treating a wider range of diseases and, as companies move to larger-scale development, they should become familiar with the Chemistry, Manufacturing, and Control (CMC) aspects of their workflow.

That’s the view of Bao Zhang Cai, PhD, vice president of oligonucleotide CMC at GondolaBio. Cai will be giving a talk about CMC strategies for oligonucleotide therapies at the upcoming Bioprocessing Summit in Boston.

“The trend is very clear. Oligonucleotide therapies are becoming increasingly popular, as they tackle the root causes of diseases, not just the symptoms, and I can confidently say they’re now a validated therapeutic modality,” he says.

“But to achieve their potential, you need the right development strategy at the right time—you don’t want to apply late commercial staging to early development.”

According to Cai, synthetic oligonucleotide therapeutics have seen significant development over the last decade. They started as therapies for rare or ultra-rare diseases, but their applications have now widened.

With more patients needing treatment, a growing number of drugs are reaching the later phases of clinical trials and, as a result, CMC is increasingly important.

Cai suggests that product purification is important to consider, as it is often [genetic] sequence-dependent.

“You need to think about it upfront or as early as possible,” he says. “This is why, in general, it helps to identify and take advantage of an existing platform.”

An oligo-specific challenge, he explains, is that these products consist of a hydrophobic element conjugated to a water-soluble section.

“These two extremes put together generate unique challenges during purification, and you need to find the best route—do you want to perform the conjugation in solution or on a solid support?” he explains.

Finally, he says, as synthetic oligonucleotides are a relatively new product type, the critical quality attributes and product specifications can typically start quite broad. A focus on CMC can help manufacturers collect the data to narrow these specifications over time.

Technology maturity and development stage [Bao Zhang Cai, PhD, GondolaBio]

“Better process understanding can mean that, in the early phase, you have a poorly resolved method, and you get, say, 90% purity. But, later, you have a better method, and the purity will drop, and that’s a tough case to explain to the regulator,” he says.

“But, if you have process knowledge and understanding, you can demonstrate the product quality remains the same, and the purity level only looks lower because your methods have improved.”

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Perfusion Technologies Gain Momentum in Biomanufacturing

Edusehat — Thu, 09 Jul 2026 01:45:14 +0700

Perfusion-based manufacturing is gaining traction across the biopharmaceutical industry as companies seek to boost biologics output, improve product quality, and increase manufacturing flexibility without expanding facility footprints. Long viewed as a promising but operationally complex alternative to fed-batch production, perfusion is benefiting from a wave of technological advances that are making the approach more practical and economically attractive for commercial-scale manufacturing.

“Perfusion helps maintain cells at very high viable cell densities,” said Charles Solanke, senior scientist of upstream process development at Cytovance Biologics. “This produces significantly more product per unit bioreactor volume compared to traditional fed-batch processes.”

One of perfusion’s key advantages is its ability to continuously harvest product while maintaining healthy cell cultures. “Continuous removal of toxic metabolites creates a more stable environment,” Solanke notes. “This supports prolonged culture duration and high cell viability.”

Product-quality improvements are emerging as another major driver of adoption. Because biologics are harvested continuously, manufacturers can reduce variability in critical quality attributes, such as glycosylation patterns and aggregation profiles.

Technological developments are further expanding the performance ceiling of perfusion operations. High-intensity perfusion media can now support cell densities reaching 100–200 million cells per milliliter, enabling substantially greater productivity than previous generations of processes. At the same time, advances in alternating tangential flow (ATF) and tangential flow filtration (TFF) technologies improve cell-retention performance and reduce filter fouling, which is one of the most persistent challenges in long-duration perfusion runs.

Automation is also reshaping the field. Real-time monitoring technologies—including Raman spectroscopy, capacitance probes, soft sensors, and advanced process-analytical technology—are increasingly being integrated into commercial processes to provide continuous insight into culture conditions. “Real-time monitoring and process control help maintain stable culture conditions,” Solanke said. “The advances have also helped improve process robustness and manufacturing reliability.”

Despite the progress, challenges remain. Perfusion systems typically require higher media consumption, more sophisticated process controls, and additional equipment investments than conventional fed-batch operations. Extended run durations can also increase contamination risks if robust aseptic controls are not maintained.

Looking ahead, industry efforts are increasingly focused on developing cell lines specifically optimized for ultra-high-density perfusion cultures, reducing media consumption through concentrated formulations, and deploying fully autonomous process control systems. “The integration of fully automated perfusion control systems represents a promising opportunity,” Solanke said. “Together, these advancements have the potential to make perfusion processes more productive, cost-effective, and easier to operate.”

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Plant Expression Platforms May Be Better Option for Biopharma in the Global South

Edusehat — Thu, 09 Jul 2026 01:45:13 +0700

Mammalian cell-based manufacturing systems cannot meet the needs of the Global South, say researchers, who suggest that local production with plant-based expression platforms is a potential low-cost alternative. The researchers made their case in a recent paper, arguing that although mammalian systems are a good option for countries where centralized production facilities can be established, they are less suited to resource-limited regions.

“Mammalian cell lines, particularly Chinese hamster ovary cells, are the industry standard for producing complex biologics that require human-like PTMs [post-translational modifications], offering high-quality mAbs and vaccines with established regulatory approvals for pandemic applications.

“However, their high operational costs, slow doubling times, susceptibility to viral contamination, and dependence on expensive media significantly limit global equity and accessibility, particularly in resource-constrained settings such as the Global South,” the authors write.

Plant-based expression

Overcoming these constraints and increasing access to medicines in the Global South, will require the establishment of local production capacity that is both economically and environmentally sustainable, the authors say, citing plant-based systems as a potential option.

“Plants are increasingly used as platforms for producing vital biological molecules, such as pharmaceuticals and industrial biomaterials, through advanced strategies, including genetic engineering, process automation, and precision agriculture.”

The authors point to things like the Gaucher’s disease drug, Elelyso, the Ebola treatment, ZMapp, and the COVID-19 vaccine, Covifenz, as examples of current plant-made biopharmaceutical products.

And the potential advantages are significant. For one thing, plant-based systems are generally faster to produce protein and more easily scalable than mammalian platforms, according to the authors.

“Plant-based expression systems, particularly seed-based platforms such as rice, wheat, tobacco, sorghum, etc., offer scalable, field-level production regarded as safe status, low-cost PTMs, and exceptional environmental advantages.

“These systems enable decentralized, long-term stable storage of biologics and the production of animal-free, glycosylated therapeutics, significantly enhancing health security in resource-limited settings,” they write.

And the utility of plant-based expression systems is being further enhanced by new genetic modification techniques. The authors cite AI-driven genomic optimization and glyco-engineering as examples of how such systems are being improved.

“Drought-tolerant plant platforms can significantly enhance local production capacity in developing economies, addressing critical barriers such as limited investment, infrastructure constraints, and regulatory hurdles.”

Seeds not cells

Plant-based systems can also help biopharma address one of the major challenges of working in the global south—the need for extensive cold chain logistics infrastructure.

Mammalian cells are sensitive to environmental conditions and, as a result, manufacturers use temperature-controlled environments to prevent damage. These concerns are less of an issue for plant-based systems produced from seeds.

“Seed-based platforms offer a strategic advantage by enabling ambient-temperature storage of recombinant proteins for extended periods without loss of bioactivity. This capability significantly reduces cold-chain dependency and enhances logistical resilience in resource-limited settings.

“Consequently,” the authors continue, “plant-based expression systems represent a premier, cost-effective pathway for the large-scale production of biologics.”

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Local PBMC Isolation Enables Four-Hour Processing in San Diego

Edusehat — Thu, 09 Jul 2026 01:45:10 +0700

Fast, local cryopreservation is a consistent bottleneck for autologous cell therapy developers, even in major cell and gene therapy hubs. For the peripheral blood mononuclear cells (PBMCs) that form the foundation of immunotherapies, vaccine development and autologous therapies, every hour between leukapheresis collection and cryopreservation can degrade cell viability and functional integrity.

OrganaBio’s new PBMC isolation facility in San Diego removes that bottleneck for one of the three major cell and gene therapy hubs in the world. The opening of the new PBMC processing and cryopreservation laboratory at Excellos Labs (which OrganaBio acquired in May) makes it possible to isolate and cryopreserve PBMCs within three to four hours of collection, rather than losing time and quality by shipping live material across the country.

“The quality you lock in at cryopreservation is the quality the developer gets back months later,” Justin Irizarry, CEO, OrganaBio, tells GEN. “Get the first few hours right and you have protected everything downstream. Having that processing happen locally is what makes the few-hour window realistic rather than aspirational.”

The San Diego lab reports an average PBMC viability of “around 99.1% and recovery above 2.9 million cells per milliliter, with consistency from batch to batch,” Irizarry says. He credits those statistics to rapid time to cryopreservation, validated standard operating procedures “including sponsor-specific protocols,” personnel trained in each of those procedures, and a single quality system for each of OrganaBio’s sites. That combination ensures consistency across batches and sites, so “a sample processed in San Diego is indistinguishable from one processed in Miami,” he emphasizes.

In contrast, one German study comparing cells cryopreserved within six hours or 20 hours of collection notes a higher percentage of apoptotic natural killer (NK) cells associated with the longer hold time. Specifically, after 20 hours, 41% of the cells were apoptotic versus 24% for those preserved within six hours of collection. Robustness also declined with the longer hold times.

Robust local sourcing for PBMCs is a strategic advantage for San Diego’s clinical-stage biotherapeutic developers. In addition to gaining higher-quality starting materials, they can expect to benefit from tighter manufacturing timelines and less supply chain risk. Local processing reduces the inherent risk associated with cold-chain and transportation variables, while enhancing redundancy provided by OrganaBio’s West Coast sites in San Diego, Irvine, and San Francisco Bay, and its East Coast headquarters in Miami.

OrganaBio expects continued growth, “including clinical trial services, manufacturing services, and product manufacturing,” Irizarry says. The company is onboarding new customers and programs and deepening relationships with existing customers. “We also will evaluate opportunities to expand into additional markets for clinical trial services, particularly where there is strong population growth, good ethnic and racial diversity, and a lack of professionalized PBMC isolation services in the market.”

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Interface Mismatches Remain Key Barrier to Continuous Bioprocessing

Edusehat — Thu, 09 Jul 2026 01:45:09 +0700

Achieving end-to-end continuous bioprocessing without introducing surges and pooling remains a goal for biomanufacturers. As they transition from vat to hybrid or continuous processing, biomanufacturers optimize individual steps but often overlook their interfaces, creating an imbalance in the system.

“The primary barrier to end-to-end continuous biomanufacturing is often not the lack of continuous unit operations themselves. The larger challenge is integrating technologies such as perfusion culture, multicolumn chromatography, continuous viral inactivation, and continuous filtration into a coordinated manufacturing train,” Moo Sun Hong, PhD, assistant professor, Seoul National University, tells GEN.

“Our review suggests that unresolved interface mismatches between unit operations, particularly between steady upstream harvest and cyclic downstream purification, remain the dominant obstacle to achieving true end-to-end continuity,” Hong says. Interface engineering is vital to manage throughput, residence times, process robustness, and product quality across interconnected units.

One part of the study compared batch and continuous biopharmaceutical manufacturing across 10 metrics, while another considered the various degrees of continuous processing. They concluded that the advantages of continuous processing result mainly from process intensification, which, in itself, introduces new vulnerabilities such as measurement latency and uncertainty around residence time distribution.

The challenge for manufacturers is that limited coordination between upstream and downstream processing results in flow-rate mismatches. “Unit operations operate with fundamentally different dynamics,” Hong acknowledges. “Upstream perfusion generates a relatively steady harvest stream, whereas many downstream operations operate cyclically. This creates flow-rate and residence-time mismatches that often require surge tanks or hold steps. Eliminating these interruptions requires careful interface engineering, synchronization of cycle times, real-time monitoring, and coordinated control across the entire process train.”

Take a systems approach

Hong and colleagues recommend evaluating batch-to-continuous processing transitions based upon “integrated techno-economic, sustainability, and operational performance metrics rather than isolated unit-operation productivity alone.” Therefore, process engineers can design the interfaces and control strategies in a way that enables a fully-connected, automated, continuous manufacturing platform that functions in a near steady-state without the need for holding tanks between units.

In a continuous processing environment, control strategies based on process analytical technology (PAT) and digital twin technology are vital. Hong calls them “the backbone of integrated continuous manufacturing.”

Specifically, PAT provides visibility and real-time monitoring for rapid responses to process variances, while digital twins provide enhanced predictive models, as well as fault detection, optimization, and analysis. Additional automation and control architectures also should be included “…that connect sensors and programmable logic controller/supervisory control and data acquisition (PLC/SCADA) systems, as well as process data repositories, control loops, and supervisory models across the integrated process train,” the scientists advise.

“In our view,” Hong concludes, “the challenge is not simply making individual operations continuous, but making the entire manufacturing platform function as an integrated system.”

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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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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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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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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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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.

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.”

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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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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.

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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’.”

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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.

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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.”

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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.”

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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.

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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.”

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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.

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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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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.”

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

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