Tag: Biotech

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STAT+: Stealth biotech Stipple bets on secretive ADCs

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A four-month FDA delay prompted a cash-strapped biotech company to shutter. The White House’s proposed NIH cuts are drawing bipartisan backlash. Peptide hype is outpacing evidence. And more!

The need-to-know this morning

  • Neurocrine Biosciences said this morning that it would buy Soleno Therapeutics and its treatment for Prader-Willi syndrome for $2.9 billion. Neurocrine is paying $53 a share for Soleno, a 34% premium to its closing price on Thursday.

A four-month FDA delay derailed a small biotech. Is it a sign of the times?

FDA delays can happen. For large drugmakers, they can be frustrating; for small drugmakers, they can be existential.

Continue to STAT+ to read the full story…

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Nanotube Injector Boosts Mitochondrial Performance Through Cytoplasmic Transfer

Extracting cytoplasmic material such as proteins, RNA, and mitochondria often relies on cell lysis using detergents or enzymes, which destroy the cells. Ultrasound and other sophisticated physical disruption methods need to be carefully tuned to avoid damaging biomolecules, potentially rendering them too time-consuming.

Delivering material into cells presents further challenges. Lipid-based carriers are limited to small molecules, viral vectors are costly, and microinjection techniques are difficult to scale. To date, no approach allows for controlled and efficient cytoplasmic transfer without compromising cell viability, according to researchers from Waseda University in Japan.

The team published a study “A Nanotube Injector for Cytoplasmic Transfer and Enhanced Mitochondrial Function” in Small Science that reports the development of a nanotube membrane-based injector—a platform that combines nanomaterials and fluid physics to directly transfer cytoplasmic contents between cell populations. The system consists of a thin gold membrane with vertically aligned nanotubes mounted on a glass tube. When this membrane is carefully pressed against cultured cells, the nanotubes penetrate the phospholipid bilayer of the living cells without causing significant damage. By adjusting the internal air pressure of the glass tube, the researchers can “suck up” cytoplasmic material from the source cells, hold it as the tube is repositioned over the target cell culture, and gently flush it into this new population using microliters of a buffer solution.

Infographic from Waseda University in Japan
Credit: Waseda University

Through several experiments using fluorescent dyes and protein assays, the researchers say they confirmed that cytoplasmic contents could be extracted in a pressure-dependent manner. They also found that careful selection of nanotube diameter, nanotube density, and applied pressure was key to minimizing cellular damage. Notably, under optimized conditions, cell viability hovered around 95%, with a cytoplasmic transfer efficiency of well over 90%, note the scientists.

To further test the capabilities of their platform, the team investigated whether it could transfer intact mitochondria. To this end, they labeled mitochondria in donor cells with a fluorescent tag and observed them in the recipient cells via confocal microscopy. They found that dozens of mitochondria could be reliably delivered per cell.

Most importantly, according to Takeo Miyake, PhD, team leader, these mitochondria remained functional, as evidenced by markedly higher levels of adenosine triphosphate (ATP) produced in recipient cells compared to controls.

“This technology establishes a new paradigm for cell manipulation—transforming cells not by genetic modification but by reconstructing intracellular composition itself,” explains Miyake, adding that such controlled cytoplasmic engineering, enabled by the proposed nanotube injector, could support the development of next-generation cell therapies, improved disease models, and more precise drug screening platforms.

“Directly transferring healthy mitochondria or cytoplasmic components into target cells is particularly relevant for regenerative medicine, where therapeutic cells often suffer from reduced metabolic activity or functional heterogeneity after isolation and expansion,” highlights Miyake, “By restoring or augmenting mitochondrial function without genetic modification, the technology offers a new strategy to improve cell quality prior to transplantation.”

Overall, this innovative system paves the way for a new level of control in cell biology research, as well as bioengineering and biomedical applications, points out the research team.

The post Nanotube Injector Boosts Mitochondrial Performance Through Cytoplasmic Transfer appeared first on GEN – Genetic Engineering and Biotechnology News.

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STAT+: How a four-month FDA delay forced a small biotech company to close its doors

In February, a small biotech company called Kezar Life Sciences reached a breakthrough with the Food and Drug Administration, agreeing to a plan for a clinical trial it hoped could lead to the approval of its treatment for a rare, debilitating liver disease called autoimmune hepatitis. The problem: The agreement came four months too late.

The meeting to discuss trial design, a critical step in the drug development process, had been scheduled for last October. But the FDA abruptly canceled it without explanation. The company could no longer proceed as planned and, without clarity from regulators, its path forward was unclear. Kezar’s investors wanted out, and the biotech was forced to start the process of winding down.

It laid off most of its staff of about 60 people. Then, it auctioned off its lab equipment and sold much of its office furniture, except for the table and chairs in one conference room it kept in case the company got its meeting with FDA staff.

Last week — after the meeting and the breakthrough happened — the company said it would be sold. Kezar hopes the buyer, Aurinia Pharmaceuticals, will take the drug forward, though how quickly that can happen, if at all, is not guaranteed.

It’s also not clear why Kezar’s initial meeting was canceled. But to CEO Chris Kirk, the chain of events fits a pattern over the past year in which volatility at the FDA — including staff departures and decision-making seen as inconsistent — has ricocheted across the industry, impacting drugmakers. Those impacts can fall disproportionately on small companies, which, unlike major drugmakers, often operate on one financing to the next. 

“In my career, I’ve often not agreed with what the FDA has said, but I’ve at least relied on their consistency,” said Kirk, who’s worked in biotech for more than two decades. “That doesn’t appear to be what’s happening now. It feels more stochastic and maybe even capricious, what’s going on at the FDA. And this isn’t good for patients. It’s definitely not good for the biotech ecosystem as a whole.”

Continue to STAT+ to read the full story…

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Injectable Microgel Developed to Reduce Bleeding in Infants Undergoing Surgery

Biomedical researchers headed by a team at the Lampe Joint Department of Biomedical Engineering, University of North Carolina at Chapel Hill/North Carolina State University, have developed an injectable microgel to help reduce bleeding in infants who require surgical care. Tests in an animal model showed that the hemostatic microgels, known as B-knob-triggered microgels (BK-TriGs), reduced bleeding by at least 50%.

Research lead Ashley Brown, PhD, who is the Lampe Distinguished Professor of Biomedical Engineering, is co-corresponding author of the team’s published paper in Science Advances, titled “Hemostatic B-knob-triggered microgels (BK-TriGs) to address bleeding in neonates.” In their paper the team concluded “This study highlights the potential of BK-TriGs, designed for neonatal-specific clotting mechanisms, to address the heightened bleeding and thrombosis risks in neonates, who face 4.4 times higher postsurgery mortality … Our findings support BK-TriGs as a promising approach for improving hemostasis in neonates, offering a tailored, effective solution for this vulnerable patient population.”

When adults cut themselves, a multi-step process called hemostasis stops the bleeding from the injured blood vessel. But hemostasis in infants is different from hemostasis in adults. This difference can be problematic if infants require surgery to address significant medical problems. In surgeries, neonatal patients normally receive blood from adult donors to compensate for blood lost during the operation. “Current treatments rely on transfusing adult blood products, which may cause complications resulting from structural and functional differences between neonatal and adult fibrinogen,” the authors wrote. “… these transfusions pose serious safety concerns by increasing morbidity, prolonging intensive care unit stays, and elevating posttransfusion thrombosis risks in neonates.”

Brown noted, “… if you give adult blood to an infant, the difference in adult hemostasis versus infant hemostasis can lead to too much clotting. That can increase the likelihood of thrombosis, where blood clots form in the lungs or elsewhere and put the baby at risk … “My research team has done a lot of work on surgery-related bleeding in newborns, and we wanted to develop a therapeutic intervention that would reduce bleeding and—by extension—reduce the need for infants to receive adult blood transfusions during surgery.”

The scientists have now reported on their development of a material called B-knob triggered microgels (BK-TriGs). “Fibrin is the main clotting protein in human blood,” Brown explains. “There is a short amino acid sequence called a ‘B peptide’ that links together fibrin molecules to create blood clots where they are needed—and these B peptides play a particularly important role in hemostasis for infants. The BK-TriGs are engineered particles that are studded with those B peptides.”

The particles can absorb water and become squishy hydrogels, which mimic the mechanical properties of natural platelets in a way that maximizes the ability of the B peptides to create fibrin networks and stanch bleeding. “Functionalized with a fibrin hole b–specific peptide, BK-TriGs enhance clot density and resistance to degradation,” the team noted.

The researchers first tested the BK-TriGs by using microfluidic devices that allowed them to conduct in vitro testing to see how the microgels affected clotting in blood plasma from human adults and infants. “In vitro studies using neonatal platelet-poor plasma (PPP) showed that at an optimal concentration, BK-TriGs increased clot density by more than 100% and improved stability by reducing fibrinolysis,” they wrote in summary. “Under flow conditions BK-TriGs promoted robust clot formation compared to plasma-only controls.” Brown noted, “We found that BK-TriGs worked better at improving blood clotting in infant plasma than in adult plasma, which was what we expected to see.”

To further test the efficacy of the BK-TriGs, the researchers worked with lab mice that were genetically engineered to not make fibrinogen, the precursor to fibrin. This allowed the researchers to first introduce infant fibrinogen into the lab mice so that the mice exhibit a form of hemostasis similar to infants. “This innovative model enabled the evaluation of BK-TriGs in a setting that replicates key aspects of neonatal fibrinogen polymerization and fibrinolytic sensitivity, providing preliminary insights into their potential clinical utility.”

Brown added, “We found that the BK-TriGs outperformed any of the other options we tested at reducing blood loss. Specifically, the BK-TriGs reduced blood loss by 50-60% compared to the control group.”

The authors further stated, “The findings highlight the potential of BK-TriGs as a promising synthetic platelet-mimetic approach for enhancing clot density and stability, particularly in neonatal plasma where traditional blood products may pose risks … A fibrin-targeted approach like BK-TriGs, which enhances clot formation without introducing systemic thrombotic risk, may offer a safer alternative to adult fibrinogen transfusions.”

Next steps for the work are to see how BK-TriGs compare to other hemostatic therapeutics that are on the market, either on their own or when used in conjunction with BK-TriGs. “The results we’re reporting here are exciting, but we are still far removed from clinical use,” Brown, acknowledged. “We need to make sure there are no unforeseen risks associated with blood clotting.” The team also commented, “Expanding this research to include different clinical bleeding scenarios will be essential to advancing these materials toward therapeutic applications.”

“This is particularly relevant in neonates, where the most severe bleeding complications often arise in critical sites such as the gastrointestinal tract and the brain,” Brown continued, “But if we do find BK-TriGs are safe and effective, we’re optimistic this could be a cost-effective way to make surgery safer for infants. Manufacturing the BK-TriG particles would be relatively inexpensive—certainly in comparison to blood products.”

The post Injectable Microgel Developed to Reduce Bleeding in Infants Undergoing Surgery appeared first on GEN – Genetic Engineering and Biotechnology News.

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Four things we’d need to put data centers in space

MIT Technology Review Explains: Let our writers untangle the complex, messy world of technology to help you understand what’s coming next. You can read more from the series here.

In January, Elon Musk’s SpaceX filed an application with the US Federal Communications Commission to launch up to one million data centers into Earth’s orbit. The goal? To fully unleash the potential of AI without triggering an environmental crisis on Earth. But could it work?

SpaceX is the latest in a string of high-tech companies extolling the potential of orbital computing infrastructure. Last year, Amazon founder Jeff Bezos said that the tech industry will move toward large-scale computing in space. Google has plans to loft data-crunching satellites, aiming to launch a test constellation of 80 as early as next year. And last November Starcloud, a startup based in Washington State, launched a satellite fitted with a high-performance Nvidia H100 GPU, marking the first orbital test of an advanced AI chip. The company envisions orbiting data centers as large as those on Earth by 2030.

Proponents believe that putting data centers in space makes sense. The current AI boom is straining energy grids and adding to the demand for water, which is needed to cool the computers. Communities in the vicinity of large-scale data centers worry about increasing prices for those resources as a result of the growing demand, among other issues.

In space, advocates say, the water and energy problems would be solved. In constantly illuminated sun-synchronous orbits, space-borne data centers would have uninterrupted access to solar power. At the same time, the excess heat they produce would be easily expelled into the cold vacuum of space. And with the cost of space launches decreasing, and mega-rockets such as SpaceX’s Starship promising to push prices even lower, there could be a point at which moving the world’s data centers into space makes sound business sense. Detractors, on the other hand, tell a different story and point to a variety of technological hurdles, though some say it’s possible they may be surmountable in the not-so-distant future. Here are four of the must-haves we’d need to make space-based data centers a reality. 

A way to carry away heat 

AI data centers produce a lot of heat. Space might seem like a great place to dispel that heat without using up massive amounts of water. But it’s not so simple. To get the power needed to run 24-7, a space-based data center would have to be in a constantly illuminated orbit, circling the planet from pole to pole, and never hide in Earth’s shadow. And in that orbit, the temperature of the equipment would never drop below 80 °C, which is way too hot for electronics to operate safely in the long term. 

Getting the heat out of such a system is surprisingly challenging. “Thermal management and cooling in space is generally a huge problem,” says Lilly Eichinger, CEO of the Austrian space tech startup Satellives.

On Earth, heat dissipates mostly through the natural process of convection, which relies on the movement of gases and liquids like air and water. In the vacuum of space, heat has to be removed through the far less efficient process of radiation. Safely removing the heat produced by the computers, as well as what’s absorbed from the sun, requires large radiative surfaces. The bulkier the satellite, the harder it is to send all the heat inside it out into space.

But Yves Durand, former director of technology at the European aerospace giant Thales Alenia Space, says that technology already exists to tackle the problem.

The company previously developed a system for large telecommunications satellites that can pipe refrigerant fluid through a network of tubing using a mechanical pump, ultimately transferring heat from within a spacecraft to radiators on the exterior. Durand led a 2024 feasibility study on space-based data centers, which found that although challenges exist, it should be possible for Europe to put gigawatt-scale data centers (on par with the largest Earthbound facilities) into orbit before 2050. These would be considerably larger than those envisioned by SpaceX, featuring solar arrays hundreds of meters in size—larger than the International Space Station.

Computer chips that can withstand a radiation onslaught

The space around Earth is constantly battered by cosmic particles and lashed by solar radiation. On Earth’s surface, humans and their electronic devices are protected from this corrosive soup of charged particles by the planet’s atmosphere and magnetosphere. But the farther away from Earth you venture, the weaker that protection becomes. Studies show that aircraft crews have a higher risk of developing cancer because of their frequent exposure to high radiation at cruising altitude, where the atmosphere is thin and less protective.

Electronics in space are at risk of three types of problems caused by high radiation levels, says Ken Mai, a principal systems scientist in electrical and computer engineering at Carnegie Mellon University. Phenomena known as single-event upsets can cause bit flips and corrupt stored data when charged particles hit chips and memory devices. Over time, electronics in space accumulate damage from ionizing radiation that degrades their performance. And sometimes a charged particle can strike the component in a way that physically displaces atoms on the chip, creating permanent damage, Mai explains.

Traditionally, computers launched to space had to undergo years of testing and were specifically designed to withstand the intense radiation present in Earth’s orbit. These space-hardened electronics are much more expensive, though, and their performance is also years behind the state-of-the-art devices for Earth-based computing. Launching conventional chips is a gamble. But Durand says cutting-edge computer chips use technologies that are by default more resistant to radiation than past systems. And in mid-March, Nvidia touted hardware, including a new GPU, that is “bringing AI compute to orbital data centers.” 

Nvidia’s head of edge AI marketing, Chen Su, told MIT Technology Review, that “Nvidia systems are inherently commercial off the shelf, with radiation resilience achieved at the system level rather than through radiation‑hardened silicon alone.” He added that satellite makers increase the chips’ resiliency with the help of shielding, advanced software for error detection, and architectures that combine the consumer-grade devices with bespoke, hardened technologies.

Still, Mai says that the data-crunching chips are only one issue. The data centers would also need memory and storage devices, both of which are vulnerable to damage by excessive radiation. And operators would need the ability to swap things out or adapt when issues arise. The feasibility and affordability of using robots or astronaut missions for maintenance is a major question mark hanging over the idea of large-scale orbiting data centers.

“You not only need to throw up a data center to space that meets your current needs; you need redundancy, extra parts, and reconfigurability, so when stuff breaks, you can just change your configuration and continue working,” says Mai. “It’s a very challenging problem because on one hand you have free energy and power in space, but there are a lot of disadvantages. It’s quite possible that those problems will outweigh the advantages that you get from putting a data center into space.”

In addition to the need for regular maintenance, there’s also the potential for catastrophic loss. During periods of intense space weather, satellites can be flooded with enough radiation to kill all their electronics. The sun has just passed the most active phase of its 11-year cycle with relatively little impact on satellites. Still, experts warn that since the space age began, the planet has not experienced the worst the sun is capable of. Many doubt whether the low-cost new space systems that dominate Earth’s orbits today are prepared for that.

A plan to dodge space debris

Both large-scale orbiting data centers such as those envisioned by Thales Alenia Space and the mega-constellations of smaller satellites as proposed by SpaceX give a headache to space sustainability experts. The space around Earth is already quite crowded with satellites. Starlink satellites alone perform hundreds of thousands of collision avoidance maneuvers every year to dodge debris and other spacecraft. The more stuff in space, the higher the likelihood of a devastating collision that would clutter the orbit with thousands of dangerous fragments.

Large structures with hundreds of square meters of solar arrays would quickly suffer damage from small pieces of space debris and meteorites, which would over time degrade the performance of their solar panels and create more debris in orbit. Operating one million satellites in low Earth orbit, the region of space at the altitude of up to 2,000 kilometers, might be impossible to do safely unless all satellites in that area are part of the same network so they can communicate effectively to maneuver around each other, Greg Vialle, the founder of the orbital recycling startup Lunexus Space, told MIT Technology Review.

“You can fit roughly four to five thousand satellites in one orbital shell,” Vialle says. “If you count all the shells in low Earth orbit, you get to a number of around 240,000 satellites maximum.”

And spacecraft must be able to pass each other at a safe distance to avoid collisions, he says. 

“You also need to be able to get stuff up to higher orbits and back down to de-orbit,” he adds. “So you need to have gaps of at least 10 kilometers between the satellites to do that safely. Mega-constellations like Starlink can be packed more tightly because the satellites communicate with each other. But you can’t have one million satellites around Earth unless it’s a monopoly.”

On top of that, Starlink would likely want to regularly upgrade its orbiting data centers with more modern technology. Replacing a million satellites perhaps every five years would mean even more orbital traffic—and it could increase the rate of debris reentry into Earth’s atmosphere from around three or four pieces of junk a day to about one every three minutes, according to a group of astronomers who filed objections against SpaceX’s FCC application. Some scientists are concerned that reentering debris could damage the ozone layer and alter Earth’s thermal balance. 

Economical launch and assembly

The longer hardware survives in orbit, the better the return on investment. But for orbital data centers to make economic sense, companies will have to find a relatively cheap way to get that hardware in orbit. SpaceX is betting on its upcoming Starship mega-rocket, which will be able to carry up to six times as much payload as the current workhorse, Falcon 9. The Thales Alenia Space study concluded that if Europe were to build its own orbital data centers, it would have to develop a similarly potent launcher. 

But launch is only part of the equation. A large-scale orbital data center won’t fit in a rocket—even a mega-rocket. It will need to be assembled in orbit. And that will likely require advanced robotic systems that do not exist yet. Various companies have conducted Earth-based tests with precursors of such systems, but they are still far from real-world use.

Durand says that in the short term, smaller-scale data centers are likely to establish themselves as an integral part of the orbital infrastructure, by processing images from Earth-observing satellites directly in space without having to send them to Earth. That would be a huge help for companies selling insights from space, as many of these data sets are extremely large, and competition for opportunities to downlink them to Earth for processing via ground stations is growing.

“The good thing with orbital data centers is that you can start with small servers and gradually increase and build up larger data centers,” says Durand. “You can use modularity. You can learn little by little and gradually develop industrial capacity in space. We have all the technology, and the demand for space-based data processing infrastructure is huge, so it makes sense to think about it.”

Smaller facilities probably won’t do much to offset the strain that terrestrial data centers are placing on the planet’s water and electricity, though. That vision of the future might take decades to come to fruition, some critics think—if it even gets off the ground at all. 

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STAT+: Biotech investors’ plea to Trump, and a busy M&A week

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The Trump administration is using newly announced 100% tariffs as leverage to push both large and small drugmakers into confidential pricing and manufacturing agreements.

Also, the burgeoning peptide craze is highlighting a trust gap in medicine, in which patients increasingly favor unproven treatments over well-established drugs.

Continue to STAT+ to read the full story…

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Hydrogel-Based Axon Model Improves Early Testing for MS Remyelination Therapies

Axons—the long, cable‑like projections that relay electrical signals across the nervous system—depend on tightly wrapped layers of myelin to keep those messages fast and reliable. When this insulation is damaged, as in multiple sclerosis (MS) and other neurodegenerative diseases, signal transmission slows and neurons eventually degenerate. Although oligodendrocytes can repair myelin early on in the process, this capacity declines with age and repeated inflammatory attacks, leaving researchers searching for therapies that can restore myelin more effectively.

A team at University College London (UCL) has now developed a more physiologically realistic way to study how myelin forms—and how potential drugs might influence that process. Their new hydrogel‑based axon model, described in Nature Methods in a paper titled “Tunable hydrogel‑based micropillar arrays for myelination studies,” recreates both the geometry and softness of real axons. The platform is designed to address a longstanding problem in the field: many drug candidates that appear promising in rigid, plastic‑based lab models ultimately fail in human trials.

“To stop MS, we need therapies that repair myelin,” said senior author Emad Moeendarbary, PhD, professor of cell mechanics and mechanobiology at UCL and CEO of BioRecode. “Promising drug candidates in the past have failed when tested in human patients. One factor might be that laboratory models do not replicate the basic physical properties of the human brain.”

The UCL team engineered vertical micropillars—each tens of times thinner than a human hair—using a microfabrication process called photolithography that allowed them to precisely tune diameter, spacing, and stiffness. Unlike earlier artificial axons made from hard polymers, these pillars are composed of polyacrylamide hydrogel, a material whose elasticity can be adjusted to match the ~5 kPa softness of native axons. As the authors noted in the paper, the system “mimics the three‑dimensional architecture and softness of axons,” enabling oligodendrocytes to form “multilayered compact myelin” around the pillars.

The researchers seeded the hydrogel pillars with human and rodent oligodendrocytes and tested several candidate remyelination drugs. When the pillars were tuned to realistic softness, drug performance dropped—suggesting that overly rigid models may have produced misleading hits in the past. “Our work suggests that commonly used rigid models, hundreds of times stiffer than real axons, can generate misleading drug hits,” Moeendarbary said. “We believe that our more life-like model can be used as a more robust early test of drug candidates and as a platform to discover new drugs.”

The study also marks the first demonstration of compact, multilayered myelin grown from human oligodendrocytes in a fully hydrogel‑based system. The platform’s design allows high‑content imaging, transcriptomic profiling, and systematic variation of mechanical cues—capabilities that could help researchers dissect how myelin forms and why it fails in disease.

Building such a soft, microscale structure was not trivial. “Hydrogel is a close mimic of living cells… but to fabricate a soft hydrogel at such a small scale is not an easy task,” Moeendarbary noted, crediting the five years of work led by PhD student Soufian Lasli and Claire Vinel, PhD.

By more faithfully recreating the physical environment of the brain, the UCL team hopes their model will provide a more reliable proving ground for remyelination therapies before they reach clinical trials.

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AI In Silico Multi-Omics Technique Cuts Therapeutic Development Costs

Bringing a drug from discovery through clinical trials takes too long and is too expensive, with preclinical costs alone estimated at $15 to $100 million. Employing artificial intelligence (AI) early in the process can lower those costs dramatically.

AI itself isn’t a panacea, though, Jayson Uffens, CTO and chairman of GATC Health, tells GEN. Instead, “Smart computing makes smart people smarter. There’s still a lot of expertise from people on the ground who bring a lot of value—maybe the ultimate value—to the mix.”

GATC Health, an AI-driven therapeutic discovery company, uses AI to raise the floor on opportunities to get high-potential compounds into human studies faster and thereby drive success.

Its proprietary approach to hit and lead identification and program derisking can cut preclinical development costs, according to Uffens, who maintains that the earlier AI is used in a program, the more dramatic the results.

The success GATC Health touts is based on deploying Operon™, the company’s proprietary AI platform. Operon deploys in silico models to simulate human biology and takes a multi-omics approach to analysis. That approach has allowed GATC to deliver three to five optimized compounds within six months, claims Uffens, versus the up to 48 months associated with traditional high-throughput screening methods.

Such acceleration occurs by using advanced in silico models to circumvent the “hundreds of thousands of dollars’ worth of experiments performed to get a hit and, ultimately, a lead,” Uffens says.

Rather than relying upon one huge model, he elaborates, “We attack the problem from multiple facets, looking at individual problems with various models and different architectures…and coordinate hundreds of AI models to answer different questions. That’s the starting point. There’s a lot of value in how we curate and parameterize our data in those specific contexts.”

The company also launched the Derisq™ AI Report, an in-depth analysis of drug candidates that highlights safety concerns, efficacy, and non-obvious risks early, while decision-makers can still modulate those risks.

This predictive intelligence layer is, in fact, a key element of GATC’s clinical trial insurance product. Underwritten by Medical and Commercial International (MCI) under the Lloyd’s of London framework, this insurance product leverages GATC’s predictive capabilities to identify risk. It reimburses the full cost of the trial if safety or efficacy endpoints aren’t met.

Typically, MCI’s preclinical trial insurance clients would provide that company with the relevant trial information, which would be run through the Derisq tool as part of their risk analysis.

Buyers for this insurance tend to be biopharma companies that aren’t large enough to self-insure their own trials. “Capital is expensive for them,” Uffens points out. “The insurance product is there to help them lower the cost of capital and open capital doors that may not be open otherwise.”

Multiomics to Discovery

What’s different about GATC’s approach to AI, Uffens says, is that “We come in, generally, as outsiders.” The founding team includes computer scientists as well as those with strong biology and genetics backgrounds, but not necessarily industry experience.

“We built our technology originally as a genetics interpretation platform,” he recalls, “and expanded it to find additional value.” The company was formed officially in 2020.

The turning point came when GATC became involved in a failed, big pharma program for addiction research.

“(The big pharma company) hadn’t found a solution, but had really valuable data and samples. A partner of ours was working with it to identify biomarkers and thought we could validate them. We discovered that not only could we validate the biomarkers, but we could also identify the therapeutic targets. That’s how we moved from multi-omics analysis into discovery,” Uffens recalls.

Moving forward, “We want to empower researchers,” he says. This means not only helping clients advance existing programs but also by identifying potentially more valuable targets.

Working with GATC

GATC’s key partners most likely will be biotech rather than big pharma, Uffens predicts. And, he notes, “We’re fairly agnostic to therapeutic area.”

“Most of our customers have called us because they want to realize the benefits of AI sooner rather than later,” Uffens says. “There is a lot of risk in the space. Folks who are willing to adopt AI at this stage…are looking for additional help before they risk more capital…” to solve particular challenges.

For a company to begin working with GATC, he explains, “The data we’re looking for is very similar to what they would include in an Investigational New Drug (IND) package. The earlier they are in the process, the less data they will have, but, at a minimum, we need some particulars on their therapeutic’s chemistry and the intended mechanism of action.”

Challenges

Drug development is a difficult space with plentiful challenges, he admits. Therefore, “We approach things as a tech company. We iterate through a problem and find where we can succeed or fail as quickly as we can to develop a solution. We’ve gone through multiple generations of architectures, finding ways that work best.”

The next milestone is to accumulate multiple successes with Operon and Derisq in human trials. “‘Wins in humans’ is our [next] frontier,” he says. That includes wins for its insurance underwriting partners as well as for companies working directly with GATC to advance therapeutics to human trials.

As part of that goal, GATC and BioAtla are closing a deal for a Phase III trial of ozuriftamab vedotin for oropharyngeal squamous cell carcinoma and to further develop conditionally active biologic senolytic therapies. Termed a special purpose vehicle transaction—a financial entity designed to hold specific assets that last for the life of the project—the $40 million deal formed Inversagen AI, LLC, to leverage the strengths of the founding companies.

“GATC and BioAtla are equal partners in Inversagen,” Uffens says. “GATC will own a percentage of ozuriftamab vedotin and a larger stake in future joint discoveries,” thus potentially discovering new therapeutic combinations that may be effective as conditionally active biologics.

Currently, the GATC is fine-tuning its own project prioritization. “The AI landscape is both beneficial and challenging,” Uffens acknowledges. “People have certain expectations about what AI can and should do, how it works, and how they might adopt it. Getting them to hear our unique perspective comes back to our focus on wins in humans.”

GATC Health Corp.

Location: 2030 Main Street, Suite 660, Irvine, CA 92614

Phone: (833) 333-4282

Website: gatchealth.com

Principal: John Stroh, CEO

Number of Employees: 60

Focus: GATC Health Corp. is a pioneer in AI-driven drug discovery and disease prediction, with proprietary technology platforms that help therapeutic developers slash both development timelines and costs.

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Top 10 Organoid Companies

The past year marked a proverbial inflection point for organoid models designed to uncover biological insights previously unattainable through traditional cell culture experiments or animal models.

The FDA in October approved the first-ever investigational new drug (IND) submission supported solely through human vascularized organoid-based combination studies, without relying on traditional animal efficacy proof-of-concept (POC) testing. The IND application by SillaJen enabled the South Korea-based developer of oncolytic virus immunotherapeutics to begin clinical trials for a combination therapy consisting of tislelizumab or paclitaxel and BAL0891, a dual inhibitor of threonine tyrosine kinase (TTK) and polo-like kinase 1 (PLK).

SillaJen’s combo therapy incorporating BAL0891 is being evaluated in a Phase I trial (NCT05768932) whose primary completion date is estimated at December 24. SillaJen’s IND included preclinical efficacy data generated through the vascularized tumor immune microenvironment model (vTIME) developed by Qureator.

The vTIME platform and SillaJen’s trial are early examples of the shift away from animal testing toward new approach methodologies (NAMs), which the FDA sought to advance through the FDA Modernization Act 2.0, enacted in 2022. The measure removed the animal testing requirement for new FDA-regulated products that was imposed through the Federal Food, Drug, and Cosmetics Act of 1938.

“By leveraging AI-based computational modeling, human organ model-based lab testing, and real-world human data, we can get safer treatments to patients faster and more reliably, while also reducing R&D costs and drug prices,” FDA Commissioner Martin A. Makary, MD, stated last year. “It is a win-win for public health and ethics.”

The changing regulatory climate is expected to nearly triple the size of the global organoids market over the next five years, from $1.20 billion last year and a projected $1.42 billion this year to $3.29 billion in 2031—a compound annual growth rate (CAGR) of 18.31%, according to a Mordor Intelligence report released in February. The report listed market share leaders in several categories, including:

  • Source: Stem-cell-derived models (58.43%)
  • Organ type: Intestinal cultures (28.65%)
  • Application: Drug discovery and screening (46.54%)
  • End users: Biopharma companies (55.63%)
  • Technology: Scaffold-based 3D culture (32.65%)

Given their growing role in drug discovery and prospects for future growth, GEN has compiled its first-ever A-List of organoid companies.

Public companies are ranked by their combined revenues for 2025—or if not available, by their combined revenues for the first nine months of 2025 and fourth quarter of 2024—as disclosed in regulatory filings, including sales of products or services, as well as revenue from collaborations and R&D activity.

The top five public companies are ranked below. Just outside the top five at #6 was Takara Bio, whose reagents business includes organoids. Reagents generated a combined ¥31.211 billion ($197.19 million) in net sales between January and March 2025, the final quarter of its 2025 fiscal year, and April-December 2025, the first three quarters of its FY 2026. Also outside the top five was Tecan Group, whose Life Sciences Business racked up CHF 377.1 million (about $483 million) in 2025 revenue. Two Chinese companies, ACRObiosystems and Sino Biological, reported smaller revenue figures.

Private companies are ranked by the total capital they have raised, as disclosed by the companies themselves, either in press statements or in responses to GEN queries verifying figures compiled by other sources. Companies that failed to respond at deadline have been ranked according to their most recently published figures for total capital raised.

The top five private organoid companies are ranked below. Private companies placing between #6 and #10 in GEN’s rankings include Pandorum Technologies (a reported $43.7 million in total capital raised), Parallel Bio ($30 million), Mimetas (a reported $29.4 million), 28bio ($24 million), and Curi Bio (a reported $20.1 million).

28bio and publicly traded Corning co-sponsored GEN’s recent Spotlight on Organoids, a virtual summit exploring how, from drug developers to universities to research institutions, investigators are increasingly using organoid models. This inaugural GEN Spotlight is available to watch on demand; registration is free.

Not included among the ranked private companies is Crown Bioscience. While the San Diego provider of translational oncology services—including the organoid panel screening platform OrganoidXplore—has raised a reported $108 million in total capital, Crown announced plans last November to be sold by Sunnyvale, CA-based JSR Life Sciences for $204 million to Hangzhou, China-based Adicon Holdings, a portfolio company of The Carlyle Group.

 

Top 5 Public Companies
 

1. Thermo Fisher Scientific  (Life Sciences Solutions segment)

Revenue: $10.374 billion in 2025

Thermo Fisher Scientific’s Life Sciences Solutions segment includes sales from products used in developing organoids, such as OncoPro Tumoroid Cell Lines to support tumoroid development, StemFlex Medium for robust expansion of pluripotent stem cells, and Geltrex Flex matrix for the growth of a variety of cells in 3D cell cultures. In December, Thermo Fisher and AIM Biotech announced a partnership to develop standardized, reliable microphysiological systems (MPSs), focused initially on creating vascularized tumoroid models that the companies said could revolutionize cancer research and immunotherapy development. AIM Biotech contributed its organiX MPS for organoids and biopsies, as well as its VasQ Kit, all-in-one vascularization solution, and technical expertise, while Thermo Fisher provided well-characterized patient-derived tumoroid models, fit-for-purpose OncoPro Tumoroid Culture Medium, and supporting reagents.
 

2. Merck KGaA, Darmstadt, Germany (Life Science business)

Revenue: €8.98 billion ($10.348 billion) in 2025

Merck KGaA, Darmstadt, Germany, aims to build a leading presence in organoids through foundational technology, a growing portfolio of patient-derived models, and scalable commercial capabilities. In January 2025, the company announced its acquisition of organoid development pioneer HUB Organoids Holding, based in Utrecht, The Netherlands. By integrating HUB’s patient-derived organoid technology with its existing cell culture expertise, Merck KGaA envisioned enhancing its value to researchers seeking to apply 3D cell culture and next-generation biology to understand drug response earlier in development. In October, Merck KGaA launched a partnership with Promega to develop assays capable of tracking cellular activity in real time using a reporter system within organoids, allowing for testing in models that are physiologically more relevant than traditional two-dimensional models.
 

3. Danaher (Life Sciences segment)

Revenue: $7.334 billion in2025

In a December 3 post on its blog, Danaher tallied eight companies within its family of operating companies as being involved in developing organoids: Abcam, Beckman Coulter (non-diagnostic business), Genedata, IDBS, Leica Microsystems, Molecular Devices, Phenomenex, and SCIEX. The eight offer a comprehensive suite of products and technologies designed to support every stage of organoid development, from sample preparation to advanced data analysis. In December, researchers at Cincinnati Children’s Hospital Medical Center’s Center for Stem Cell and Organoid Medicine (CuSTOM), Molecular Devices, and other partners published a study detailing a new human liver organoid microarray developed by the hospital and Roche—a study co-funded by Danaher, Roche, and the Farmer Family Foundation. CuSTOM and Danaher launched their organoid development partnership in 2024.
 

4. Charles River Laboratories (Discovery and Safety Assessment segment)

Revenue: $2.403 billion in 2025 1

“From models to living systems, next-generation organoids are on the rise,” Charles River Laboratories declared in a December 4 post on its Eureka blog. “As drug discovery and development accelerate the adoption of NAMs, organoids themselves are entering a transformative era,” added Tània Martiáñez Canales, PhD, senior scientist, and Ludovico Buti, PhD, senior research leader. Immune and vascular-competent tumor organoids now capture the full complexity of the tumor microenvironment, while recent liver organoid models now approach the quality of transplant-grade tissues by exhibiting complete metabolic zonation, recapitulating the three liver’s metabolic zones, and even organ-specific vasculature. In November, Charles River committed to “evaluating opportunities to enhance its scientific capabilities” in NAMs while refining its portfolio to maximize financial performance and divest underperforming or non-core assets.
 

5. Corning (Life Sciences segment)

Revenue: $972 million in 2025

Corning offerings for organoid development include a software extension enabling Corning Cell Counter® operators to capture rapid data of 3D cell cultures based on the structure’s morphology, to the company’s Corning® Matrigel® Matrix, a solubilized basement membrane preparation used as a scaffold option to support cell expansion in organoid cultures, and Matrigel Matrix 3D plates. Matrigel and a Corning 96-well round-bottom ultra-low adhesion plate were among supplies from numerous companies used by researchers at Bernhard Nocht Institute for Tropical Medicine in Hamburg, Germany, in creating a West Nile virus encephalitis model using human cerebral organoids generated with male induced pluripotent stem cells—an effort detailed in a paper published March 7 in Nature Communications.
 

1 2025 revenue consists of the 12 months ending December 27, 2025

 

 

Top 5 Private Companies
 

1. Emulate

Total Capital Raised: $250 million

Emulate partnered with FujiFilm Cellular Dynamics in November to launch the Emulate Brain-Chip R1, a first-in-class isogenic model of the neurovascular unit designed to offer researchers a new platform for studying drug transport across the blood-brain barrier, as well as investigating mechanisms of neuroinflammation. Brain-Chip R1 integrates FujiFilm’s iCell® products co-cultured with Emulate’s induced Brain Microvascular Endothelial Cells. In June, Emulate commercially introduced the AVA™ Emulation System, a self-contained instrument designed to culture, incubate, and image up to 96 individual organ-chip samples or “Emulations” in a single run—as well as to deliver in vivo-level insights faster than animal models while cutting consumable costs fourfold and in-lab labor by half compared to current generation technologies.
 

2. Prellis Biologics

Total Capital Raised: “More than” $88 million

Prellis Biologics has combined its EXIS™ organoid and AntiGen AI platforms into a platform called Biological AI that is being applied by Eli Lilly to develop next-generation antibodies, under a collaboration of undisclosed value announced in September. Lilly agreed to pay Prellis an upfront payment, payments tied to achieving development and sales milestones, plus royalties for the licensed antibodies. “With industry-leading speed (about 3-4 weeks), the EXIS™ platform generates diverse, high-affinity antibodies, derived from fully human artificial lymph node organoids against a wide array of targets and target classes, including GPCRs. These hits are then matured by artificial intelligence into drug candidates,” stated Prellis CEO Mike Nohaile, PhD.
 

3. InSphero

Total Capital Raised: $63.5 million 1

Swiss-based InSphero, in February, joined PharmaNest to launch a translational fibrosis partnership of undisclosed value, through which the companies will apply machine learning tools in combination with human preclinical models to decipher complex pathological phenotypes toward the identification of effective therapies. The collaboration combines InSphero’s advanced 3D spheroid models with PharmaNest’s high-resolution, single-fiber digital pathology, with the aim of enabling AI-assisted, precise phenotyping of fibrosis severity and remodeling for liver fibrosis in metabolic dysfunction-associated steatohepatitis (MASH) and other fibrotic 3D in-vitro models. Also in February, InSphero completed its acquisition for an undisclosed price of Doppl and its Sun Bioscience Gri3D® organoid culture platform. “For our customers, this acquisition means access to an even broader, more integrated portfolio of scalable 3D cell culture plates and organoid technologies designed to work seamlessly together,” InSphero CEO and co-founder Jan Lichtenberg, PhD, stated on LinkedIn.
 

4. CN Bio

Total Capital Raised: $60 million

CN Bio isn’t an organoid company per se, but it told GEN its organ-on-a-chip (OOC) technology is positioned to improve the human accuracy and predictivity of organoid workflows. CN Bio recommends supplementing organoids with OOC cultures designed to represent 3D tissues with more human-relevant spatial organization: “Supplementing organoid use with OOC provides the means to further advance workflows by unlocking the ability to detect deeper mechanistic insights, more complex and latent effects that may otherwise be missed,” Emily Richardson, PhD, a lead scientist on CN Bio’s R&D team, wrote on the company’s blog. In October, CN Bio launched PhysioMimix® Core, an all-in-one OOC microphysiological system (MPS) designed to be the first OOC solution to deliver validated performance across single-organ, multi-organ, and higher-throughput configurations.
 

5. Inventia Life Science

Total Capital Raised: AU$65 million ($46.5 million)

Inventia Life Science’s RASTRUM™ platform is designed to help researchers generate reproducible organoids in minutes by enabling the automated, high-throughput 3D bioprinting of cell-laden hydrogels. Last year, Sydney-based Inventia launched its next-generation version of the platform, RASTRUM™ Allegro, whose specs include producing 3D cell models in six minutes for a 96-well plate and nine minutes for a 384-well plate, with a throughput of 35+ plates a day. Optimized for patient-derived samples and translational research, RASTRUM Allegro is intended to enable the creation of more models from limited cell numbers, up to 3.5x more cell models compared to previous generations—a milestone, says the company, toward democratizing 3D cell culture for all researchers.
 

1 Figure published by PitchBook. At deadline, InSphero had not responded to GEN queries seeking to confirm the total capital raised figure.

 

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Growing Conductive Polymers Directly in the Brain

Interfacing biological tissues in the brain with electronic systems seems like science fiction, but developing effective strategies can aid in the treatment of neurodegenerative disorders, open opportunities for neurologically controlled prosthetics, or aid in modulating cardiovascular disease management, among other applications.

Creating devices with the ability to interface with biological systems is a unique challenge. Utilizing conductive polymers can improve biocompatibility over alternatives including metals and inorganic semiconductors. Pre-formed polymers implanted into organisms are not always well tolerated, so alternative techniques for polymer assembly in situ may offer a more effective and robust alternative.

Researchers at Purdue University, led by Jianguo Mei, PhD, are exploring how to form these conducting polymers from monomers applied directly to tissues. Their goal is to develop a system that is efficient and specifically integrated into the biological system, while limiting adverse effects, like inflammation or behavioral changes.

The team focused on a system to assemble n-doped poly(benzodifurandione) (n-PBDF) in vivo from injected monomers, using an organism’s native catalysts, specifically, the hemoproteins, which are abundant in the blood, to build the polymers.

Their research is published in a paper entitled, “Blood-catalyzed n-doped polymers for reversible optical neural control,” in Science.

“The development of n-type conducting polymers that assemble directly in vivo offers transformative, substrate-free strategy for stable electrical interfaces,” wrote the authors.

Using zebrafish and mice, the researchers tested both the safety and efficacy of injecting monomers that would polymerize into functional molecules. Zebrafish embryos injected in the yolk showed formation of the polymer, which was assessed through a color change in the yolk followed by molecular confirmation by spectroscopy analysis. The researchers found no behavioral changes or other developmental ill-effects and the embryos had an 80% survival rate one week after injection.

Mice injected with the monomers directly into the brain also showed polymerization of n-PBDF, with similar lack of negative response in physiology and behavior. They further showed that the polymer was functional within the tissues.

“The material formed stable deposits without signs of inflammation, neural cell loss, or changes in animal behavior,” the authors wrote. “Imaging and blood vessel assays supported its safety, whereas electrophysiological recordings revealed its effects: n-PBDF altered the activity of sodium and potassium channels, mechanisms critical for controlling neuronal firing.”

The researchers were also able to easily reverse the effect using two-photon near-infrared light stimulation. This allows for both localized application and controlled modification of neuronal behavior on a millisecond scale.

In a related Perspective, Maria Rosa Antognazza, PhD, and Guglielmo Lanzani, PhD, concur that this method holds promise for clinical applications. “Combining the approach with other mechanisms of neurostimulation—for example, by using magnetically responsive materials—may further broaden the clinical applicability and reduce the invasiveness.” However, they caution that more work must be done to explore other polymer structures, and test the technique in larger organisms, including humans.

This work shows the functional ability to polymerize n-PBDF in living organisms reversibly with long-term functionality, offering a promising path for alternative methods for connecting biosynthetics that are functional and robust, while reducing side effects. The authors concluded that, “This versatile, ultrasoft electrode, synthesized and actuated in situ, offers a new paradigm for minimally invasive bioelectronic interfaces.”

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