Tag: Biopharma

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Standardizing Personalized CRISPR Gene-Editing Therapies

The revolutionary success of Baby KJ, the first patient to be treated with a personalized CRISPR gene-editing therapy, is spurring the industry to develop platforms for standardizing the manufacturing of future individualized therapies.

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

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

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

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

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

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

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

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

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

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Cytiva Completes Doubling of Utah Site’s Liquid Media Production Capacity

Cytiva has completed an expansion of its Logan, UT, facility that effectively doubles its liquid media production capacity, a project designed to support supply chain continuity for customers relying on the company for their cell culture needs.

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

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

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

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

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

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

AI’s “two major impacts”

Pierre-Alain Ruffieux, Cytiva group executive, bioprocess

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

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

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

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

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

“Faster and better”

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

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

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

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

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

AMT designation

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

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

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

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

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

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

Bringing “the entire workflow”

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

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

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

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

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

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

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

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Claude Science is Anthropic’s newest flagship product

At an event for pharmaceutical executives, biotech founders, and researchers on Tuesday, Anthropic announced Claude Science, a major new product intended to support scientific research in the same way that Claude Code supports software engineering. Like Claude Code, Claude Science can autonomously carry out meaningful work when given concise, high-level instructions, and it has access to tools that make it particularly useful for research in computational biology and drug development. Along with launching and previewing Claude Science, which is now available to all paid Claude subscribers, Anthropic also announced that it will be using the product to pursue some of its own research into drugs for rare, neglected diseases.

This is not Anthropic’s first foray into AI for science. In October, the company released plug-ins that help Claude make use of scientific software and databases under the heading “Claude for Life Sciences.” But unlike this earlier release, Claude Science is a full-featured, standalone product. Anthropic’s decision to elevate Claude Science to the same rank as Claude Code and Claude Cowork indicates that the company is taking AI’s scientific applications very seriously—or at least wants to give the impression that it is.

“It represents how important this is to our mission that this is right up there with Claude Code and Claude Cowork as the next really significant product that we’re releasing,” says Eric Kauderer-Abrams, Anthropic’s head of life sciences. “Our mission is to develop AI that serves humanity’s long-term well-being, and we believe that by far the greatest opportunity to do that is in the life sciences.”

For the past decade, one company—Google DeepMind—has been at the vanguard of AI for science. CEO Demis Hassabis and researcher John Jumper won the Nobel Prize in chemistry for their work on the company’s AlphaFold model, and DeepMind has also made major contributions to meteorology, materials science, and a variety of other disciplines. But in the past several months, the fast-advancing frontier of AI progress seems to have left DeepMind in the dust. When it comes to coding, which has become the most lucrative use case for LLMs, DeepMind is stuck playing catch-up.

Anthropic is well positioned to take up DeepMind’s scientific mantle. Like Hassabis, Anthropic CEO Dario Amodei is a PhD scientist—unlike OpenAI CEO Sam Altman, who’s a businessman through and through. Many scientists are already avid users of tools such as Claude Code. These days, a lot of scientific research involves some amount of coding, but not all scientists are expert software engineers, and so tools like Claude Code can make a huge difference for their productivity. And the company has recently earned a major scientific vote of confidence: Earlier this month, Jumper announced that he is leaving DeepMind for Anthropic.

Since agents powered by LLMs, including Anthropic’s Opus model series, became capable of useful, independent work in late 2025, scientists have been seeing just how much they can do. In a blog post published on Anthropic’s website, the Harvard physicist Matthew Schwartz estimated, on the basis of his work with Claude Code and other Anthropic tools, that the company’s Opus 4.5 model is about as capable of executing scientific projects as a second-year graduate student.

According to Kauderer-Abrams, Claude Science isn’t intended to displace Claude Code and Claude Cowork in scientists’ workflows. Instead, it’s designed to build on what scientists already find useful about Anthropic’s products. For instance, it not only writes code but also helps scientists run their code on powerful computer clusters, which many many scientists need for their work but can be difficult to manage. And it prioritizes reproducibility, so that scientists can trace back the source of any figure or result and check it for accuracy and validity.

Though Claude Science could in principle assist with any area of scientific research, it seems designed and marketed as a tool for molecular and cellular biology, and for drug development in particular. It can interface with various tools used in genetics, chemistry, and protein biology, all of which could come in handy for researchers on the hunt for new drugs. During the Tuesday event, Alexander Tarashansky, who led the development of Claude Science, demonstrated how the system could autonomously identify new drug candidates for phenylketonuria, a rare genetic disease.

And Anthropic isn’t leaving all that work to the pharma companies and university labs that were represented at the event. Armed with Claude Science, it will be pursuing its own research into drug candidates for neglected diseases—both to help move science forward and to gain a clearer sense of how Claude Science works in the real world.

There are obvious humanitarian reasons to prioritize drug development when creating a general-purpose scientific research tool, and AI industry leaders often cite curing disease as a major potential upside of the technology. But it’s also notable that pharmaceutical companies have far deeper pockets than academic researchers. Anthropic says it’s set to see its first profitable quarter, and if major new contracts with pharmaceutical companies are forthcoming, they could help ensure it stays profitable as the tokenmaxxing craze dies down—something that’s ever more important as an IPO approaches later this year.

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<![CDATA[Why psychiatry’s growing “tribalism” harms care—and how clinicians, academia, and pharma can unite to tackle untreated mental illness.]]>
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Shilpa Commissions Integrated ADC Drug Substance GMP Manufacturing Facility

India-based Shilpa Biologicals commissioned an antibody–drug conjugate (ADC) GMP manufacturing facility, purpose-built and designed to meet global regulatory approval standards including U.S. FDA, EMA, and other major health authority requirements. The facility is fully operational, with GMP qualification protocols now underway.

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

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

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Opinion: STAT+: The U.S.-China biotech crackdown may hurt the scientists America needs the most

I spent nearly two decades covering the biotech and pharma sector, first in Washington, D.C., and then in Beijing. My first story, published in 2007, covered the U.S. Food and Drug Administration’s decision to open its inaugural overseas office in China, a fast-emerging market I later covered for 12 years.

In that time, I saw a lot of discussion about what the growth of China’s biotech sector would mean for the U.S. Never have I been more concerned about that discourse than I am now.

In recent years, China has become a biotech innovator, accounting for 34% of roughly 14,088 new drugs in global clinical development, leading the United States’ 27%. China is now contributing to 40% of new drugs entering clinical stage each year, according to data provider Pharmcube. The company also noted that China’s out-licensing deals rose nearly tenfold from 2021 to 2025, to an record $137.7 billion. Amid all of this are concerns about stolen intellectual property and the China government’s state-run companies.

Continue to STAT+ to read the full story…

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STAT+: Moderna co-founder Kenneth Chien on the future of mRNA — and Moderna 

Among all the awards and mementos Moderna co-founder Kenneth Chien has accumulated over a 50-year career in science, one stands out for its sheer luxe: an Hermes belt.

It was a 2013 gift from Moderna CEO Stéphane Bancel, one of biotech’s more dapper CEOs, after Chien helped secure a large partnership between the then-tiny mRNA startup and the Swedish-British pharmaceutical giant AstraZeneca. 

The deal accelerated Moderna’s ascent, helping bring billions in funding and of course the life-saving mRNA Covid vaccines. But the actual medicines AstraZeneca planned to build through those deals — up to 40 mRNA-based drugs for cancer and cardiovascular disease — never materialized. 

Continue to STAT+ to read the full story…

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

Insilico Medicine will partner with SK Biopharmaceuticals to discover new artificial intelligence (AI)-based drug candidates for disorders affecting the neuroimmune area of the central nervous system (CNS), through a collaboration that could generate up to $2.5 billion for the AI-based drug developer.

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

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

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

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

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

Beyond epilepsy

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

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

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

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

“Very difficult space”

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

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

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

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

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

Longevity focus

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

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

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

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

As in later this year?

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

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

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

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

“Most promising”

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

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

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

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

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

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

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

Longevity-linked targets

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

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

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

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

Second multi-billion-dollar collaboration

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

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

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

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

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

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

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

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

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

Though he is trained as a gastroenterologist and scientist, Frédéric Marrache, MD, PhD, has always had something of an entrepreneurial itch. Following his post-doctoral program and a stint in management consulting, he made his way to Sanofi where he would work on early- to mid-stage drug development programs focused on immune-mediated diseases.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Merck KGaA, Darmstadt, Germany, has agreed to acquire Bio-Techne for approximately $11.3 billion, the companies said today, in a deal designed to position the buyer as more of a leader across the life science value chain by expanding its presence in high-growth, next-generation life-sci markets with Bio-Techne’s tools, analytical technologies, and consumables.

The deal would add Bio-Techne’s multiomics offerings, analytical technologies, and integrated workflow solutions to German Merck’s platforms and services in research, bioprocessing and advanced therapeutics, with the aim of creating a combined company capable of helping customers from discovery and translational research through development, testing and commercial manufacturing.

Merck KGaA added that acquiring Bio-Techne would directly deliver on its mid- to long-term strategic agenda, which focuses on adding to its high-growth value drivers, integrated workflows, platformed capabilities—as well as scaling and sourcing innovation through merger-and-acquisition (M&A) deals like the Bio-Techne transaction.

That transaction is the latest in a series of acquisitions for Merck KGaA totaling more than $35 billion, including in the U.S. with acquisitions such as Millipore (for about $7 billion in 2010), as well as Sigma-Aldrich (for $17 billion in a deal announced in 2014 and completed the following year), Versum Materials (for €5.8 billion [about $6.6 billion] in 2019), and last year, SpringWorks Therapeutics (for $3.95 billion).

Merck KGaA said it would also benefit from Bio-Techne’s position as a leading provider of materials, analytics, and process technologies to cell therapy developers. Bio-Techne expects to acquire the ownership in Wilson Wolf it does not own immediately following the end of calendar year 2027 under the terms of a two-part forward contract between the company and Wilson Wolf, a manufacturer of cell culture devices, including the G-Rex product line. Bio-Techne holds 19.9% of Wilson Wolf that it acquired in the fiscal year that ended June 30, 2023.

Merck KGaA employs more than 14,000 people in the U.S. across over 70 company and customer sites.

The $11.3 billion Bio-Techne acquisition is the new third largest biopharma merger-and-acquisition (M&A) deal announced so far this year, behind the €10.7 billion ($12.268 billion) cash buyout offer for Italian-based Recordati being pursued by CVC Capital Partners and Groupe Bruxelles Lambert, which aim to take the company private; and Sun Pharmaceutical Industries’ planned $11.75 billion purchase of Organon, the women’s health drug developer spun out of Merck & Co., in a deal expected to close in early 2027.

The previous third-largest M&A deal this year, now fourth-largest, is the $10.9 billion AbbVie purchase of Apogee Therapeutics, announced on Monday. The fifth largest deal is GlaxoSmithKline (GSK)’s planned $10.6 billion buyout of Nuvalent,  announced June 9 and expected to close in the third quarter.

“Outstanding fit”

“Bio-Techne is an outstanding fit that directly supports our strategic direction focused on delivering cutting-edge products and solutions across the entire industry value chain—from lab customers to those manufacturing in the biotech and pharmaceutical industries,” Kai Beckmann, chairman of the executive board and group CEO of Merck KGaA, Darmstadt, Germany, said in a statement.

“By combining Bio-Techne’s scientific depth, innovation engine and differentiated portfolio with the global scale, manufacturing excellence and customer reach of Merck KGaA, Darmstadt, Germany, we are in a strong position to address some of the most important opportunities in life sciences and support our customers in accelerating the next generation of scientific discovery and therapeutic innovation. This positions us to deliver compelling strategic and financial benefits for shareholders, customers and employees,” Beckmann added.

Those benefits, according to German Merck, include immediate accretion to the company’s earnings before interest, taxes, depreciation, and amortization (EBITDA) pre margin for both the Group as a while and its Life Science business segment upon closing of the acquisition deal.

The Life Sciences segment finished last year with €8.98 billion ($10.36 billion) in revenue.  Merck KGaA does not break down its businesses further than its three segments, which also include healthcare (drug development, focused on oncology, neurology and immunology, and “global health” treatments such as for malaria) and electronics (high-tech materials).

The deal is expected to close by late 2026 or early 2027, subject to satisfying customary closing conditions that include obtaining regulatory approvals and approval by Bio-Techne shareholders.

Bio-Techne’s board of directors and the corporate bodies overseeing Merck KGaA, Darmstadt, Germany, have already approved the transaction, which will also add to earnings per share (EPS) by year three after closing, German Merck said.

€140M in “synergies”

Merck KGaA said it will carry out cost-cutting “synergies” of approximately €140 million (about $159.3 million) that are expected to be fully realized by the third year after closing.

The planned acquisition will be funded through a combination of existing cash on hand and proceeds from new debt, Merck KGaA said, adding that it will preserve its “strong” investment-grade credit rating.

For Minneapolis-based Bio-Techne, the acquisition is expected to increase its geographic and omnichannel access for its customers through integration of its offerings with those of Merck KGaA through a synergistic platform.

Bio-Techne has more than 3,000 employees, with approximately 2,300 employees based in the U.S. The company operates 34 global locations and 15 manufacturing facilities across the U.S., Canada, the U.K., Switzerland and China, and generated net sales of more than $1.2 billion in the fiscal year that ended June 30, 2025.

A leader in recombinant proteins with a half-century of heritage in next-generation R&D and new modalities, Bio-Techne said it would bring to German Merck a globally recognized portfolio of cytokines, growth factors, antibodies, and immunoassay kits. Bio-Techne is expected to strengthen the analytical and bioprocess solutions of Merck KGaA by adding to its offerings ProteinSimple, a leader in automated protein detection and analysis instruments. Bio-Techne added that its RNAscope and related in situ hybridization technologies would strengthen the capabilities of Merck KGaA, in spatial biology and diagnostics.

“For 50 years, Bio-Techne has enabled scientific breakthroughs across proteomics, spatial biology, and novel therapeutics,” stated Kim Kelderman, president and CEO of Bio-Techne. “This transaction is a testament to the remarkable company our team has built and to the enduring value we create for our customers and stakeholders.”

Muted enthusiasm

Bio-Techne investors appeared to share only muted enthusiasm for the deal, as the company’s shares traded on Nasdaq rose just 19.8% to $70.53 as of 12:48 pm ET, from Wednesday’s close of $58.88 per share. Merck KGaA shares traded on XETRA rose 4.93% to €147.00 ($167.25).

Puneet Souda, senior managing director, life science tools and diagnostics, and a senior research analyst with Leerink Partners, offered a possible explanation in a research note today: “The acquisition appears to be only a 24% premium to yesterday’s close and 26x the Street’s forecast for FY27 [enterprise value]/EBITDA compared to 16x for its LST [life science technologies] peer group.”

“We see the acquisition multiple undervaluing what is a highly accretive asset in our view,” Souda wrote. “Historically, TECH [Bio-Techne’s stock ticker] traded at much higher multiples given their highly accretive consumables profile (80%+ consumables) of consistent 70%+ gross margins and operating margin potential.”

One rival company in particular may benefit from the deal, Souda said: “The announcement is likely to be viewed positive for peer LST companies today, especially RVTY [Revvity] in our view.”

At $73 per share cash, the deal price represents a 36% premium to Bio-Techne’s one-month volume weighted average trading price.

“As part of Merck KGaA, Darmstadt, Germany, we will have greater scale and expanded capabilities to accelerate innovation and deepen our impact. Together, we will empower our customers to tackle the most important challenges in science and healthcare, helping to improve outcomes worldwide,” Kelderman added.

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