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AstraZeneca strikes $40 million AI drug discovery deal with insitro. Posted on October 15, 2026 · Last updated: October 15, 2026 AstraZeneca and insitro launch $40 million ai-driven drug discovery deal. AI collaboration targets liver and heart diseases. By Patrick Wingrove Overview of the partnership. LONDON, Oct 15 (Reuters) - AstraZeneca has teamed up with artificial intelligence biotech company insitro to discover and develop new treatments for difficult to treat liver and heart conditions, the British drugmaker said on Thursday. Financial terms and potential milestones. Under the agreement, AstraZeneca will pay $40 million up front, with California-based insitro eligible for up to about $1.46 billion in potential development and commercial milestones, as well as a single-digit percentage of future sales in royalties. Focus areas and technology. Targeted diseases: MASH and cardiomyopathies. The multi-year collaboration will focus on metabolic dysfunction-associated steatohepatitis (MASH), a severe liver disease, and cardiomyopathies, a group of diseases that affect the heart muscle. Both have historically lacked effective therapies because researchers have struggled to pinpoint their root biological drivers, the drugmaker said. AI platform: Virtual Human. Insitro will use its "Virtual Human" AI platform, which combines lab-grown cell models and machine learning, to identify causal disease biology and advance AstraZeneca-nominated targets from discovery to development-candidate stage. AstraZeneca will then run and fund future clinical trials. Insitro's growing industry presence. Recent partnerships. Bristol Myers Squibb collaboration. The deal builds on insitro's expansion across the pharmaceuticals industry. The biotech company recently expanded its partnership with Bristol Myers Squibb to advance a broader portfolio of targets for amyotrophic lateral sclerosis (ALS). Eli Lilly AI model initiative. It also teamed up with Eli Lilly to build AI models that predict pharmacological properties of small molecules in living organisms. Expert insights. Challenges in drug discovery. "Choosing the new targets that we want to put into our discovery pipeline is one of the hardest things we do across research and development," Sharon Barr, executive vice president of biopharmaceuticals R&D at AstraZeneca, told Reuters. "By the time we read about a new pathway or target (in major scientific journals), it is no longer novel. It's in the public domain and we're already a few steps too late. But when we identify these unique connections early and test them in cellular systems, we're ahead of the game." AI and scientific expertise. Barr added that pairing AI tools with experienced scientists allows the company to test fewer molecules in the lab, reduce guesswork and speed the search for effective medicines. (Reporting by Patrick Wingrove in LondonEditing by David Goodman) Key takeaways. * AstraZeneca invests $40 million upfront in AI drug discovery for liver (MASH) and heart (cardiomyopathy) diseases previously lacking effective therapies. * Insitro's Virtual Human(TM) platform combines lab-grown cell models and machine learning to uncover causal disease biology and accelerate target development. * The deal could yield insitro up to $1.46 billion in milestones and royalties, and builds on its growing AI collaboration portfolio with Bristol Myers Squibb and Eli Lilly. Frequently asked questions. What is the focus of the AstraZeneca and insitro partnership? How much is AstraZeneca investing in the insitro deal? What technology will insitro use in the collaboration? Who will conduct clinical trials for the discovered treatments? What diseases are being targeted in this AI-driven drug discovery project?

insitro has appointed Hideo Makimura as Chief Medical Officer, effective 14 September. Makimura will lead clinical development strategy as the AI therapeutics company advances its metabolic disease and neuroscience pipeline towards first-in-human trials. Makimura brings two decades of experience across drug modalities and clinical stages. He has led over a dozen programmes from early-stage testing through Phase 4 trials, spanning small molecules, antibodies, siRNA, and gene therapy. Most recently, he served as Chief Medical Officer at Affinia Therapeutics, advancing an AAV gene therapy programme to FDA IND acceptance and Fast Track designation. Previously, he spent six years at Johnson & Johnson, rising to Global Vice President and Head of Global Translational and Experimental Medicine. The South San Francisco-based company is advancing nine first-in-class programmes, with its first candidates expected to enter clinical trials in early 2027.

insitro presents new data demonstrating its ai-discovered MASH candidate shows anti-fibrotic signal beyond liver-fat reduction at the American Diabetes Association 86th Scientific Sessions. * Findings from insitro's lead therapeutic CTRO-1013 program demonstrate that liver-targeted silencing of IRS1 lowered circulatory biomarkers associated with fibrosis progression and hepatocyte injury in an industry-standard fibrogenic preclinical mouse model of MASH * Data presented today at ADA provide converging evidence from human genetics and preclinical models supporting IRS1's effect on liver fat and liver fibrosis * The findings extend insitro's AI-driven discovery of IRS1 - the strongest of 480 genetic signals for liver fat - and support the development of CTRO-1013 toward first-in-human trials this year NEW ORLEANS-(BUSINESS WIRE)-insitro, the physical AI company unlocking causal human biology, today presented new preclinical data from its metabolic dysfunction-associated steatohepatitis (MASH) therapeutic development program during a podium presentation at the American Diabetes Association (ADA) 86th Scientific Sessions. The data demonstrate that liver-targeted silencing of IRS1 reduced fibrosis progression and injury-associated circulatory biomarkers in an industry-standard fibrogenic preclinical mouse model, with effects that appear partly independent of reductions in liver fat. The findings support the continued advancement of CTRO-1013, the company's liver-targeted IRS1 candidate, through IND-enabling studies toward first-in-human trials this year. The presentation, "Hepatic IRS1 as a Therapeutic Target in MASLD: AI/ML Driven Genetic Discovery and In Vivo Therapeutic Validation," was delivered by Santhosh Satapati, Ph.D., Senior Director of Metabolic Diseases at insitro. Metabolic dysfunction-associated steatotic liver disease (MASLD) is a common chronic liver disease marked by excess fat accumulation in the liver. MASH is the progressive form of MASLD in which fat accumulation is accompanied by inflammation, liver injury, and fibrosis. Fibrosis - the scarring that can progress to cirrhosis, liver failure, and liver cancer - is one of the strongest predictors of poor outcomes for patients with MASH. Yet finding high-confidence targets has been difficult because the most relevant liver phenotypes, fibrosis among them, are hard to measure at the scale needed for human genetic discovery. Human genetic analyses revealed an IRS1-associated fibrosis signal that persists even after adjusting for liver fat, suggesting a fibrotic mechanism independent of steatosis. To evaluate this genetic hypothesis, insitro tested liver-targeted IRS1 silencing in preclinical models. Data presented today showed significant reductions in TIMP-1 and CK-18 levels, key biomarkers associated with fibrosis progression and hepatocyte injury. IRS1 inhibition also reduced liver fat in AMLN-DIO mice. Together, these results reinforce prior insitro data demonstrating improvements in liver-injury endpoints and fibrosis-related gene expression, establishing a consistent role for IRS1 across both human genetics and preclinical models. "MASH remains an area of significant unmet need, with a dearth of interventions capable of fundamentally shifting the disease trajectory," said Daphne Koller, Ph.D., founder and CEO of insitro. "The primary obstacle has been bridging the gap between early metabolic indicators, like de novo lipogenesis, and the fibrotic processes that dictate clinical outcomes. Utilizing our Virtual Human(TM) platform, we applied AI-derived liver fat and fibrosis phenotypes from multimodal human data to identify causal targets, such as IRS1, where metabolic stress and fibrosis biology appear to converge." To translate this target discovery into clinical development, insitro is advancing CTRO-1013, an investigational liver-targeted siRNA therapeutic designed to selectively silence IRS1. "IRS1 stands out because it bridges what have often been treated as two separate problems: metabolic dysfunction and fibrotic scarring," said David Lloyd, Ph.D., Senior Vice President of Metabolic Disease and Translational Pharmacology at insitro. "By targeting IRS1 with CTRO-1013, we see improvements in liver fat alongside encouraging shifts in biomarkers linked to fibrosis progression. While still preclinical, these data suggest that IRS1 inhibition could offer a path to meaningfully altering the trajectory toward severe, end-stage liver disease." insitro identified IRS1 through analysis of human phenotypic, biomarker, and genetic data, including UK Biobank imaging and clinical datasets linked to liver fat and adipose biology. Genetic analyses prioritized IRS1 as a therapeutic target associated with liver fat biology and revealed a fibrosis-associated signal that persisted after adjustment for fat. The company then validated the target experimentally. A potent, selective, liver-targeted siRNA against IRS1 suppressed lipid accumulation in human hepatocytes. In diet-induced obese mice, a single dose reduced hepatic IRS1 expression by 85%, de novo lipogenesis by 59%, and hepatic triglycerides by 45% after one month, while significantly improving NAS score versus vehicle. Liver-targeted IRS1 silencing also led to significant reductions in TIMP-1 (37%) and CK-18 (68%) after 16 weeks of treatment (1 mg/kg, Q4W) in a standard fibrogenic preclinical mouse model compared to vehicle-treated controls. In cynomolgus monkeys, liver-targeted IRS1 silencing produced sustained target reduction over 90 days without affecting glucose homeostasis. "The strength of these findings lies in the consistency of the signal - from human cells to preclinical models and non-human primates - across lipogenesis, liver injury, and fibrosis," said Philip Tagari, Chief Scientific Officer of insitro. "This breadth of evidence is critical; it indicates that IRS1 modulation impacts disease pathology through pathways that are distinct from simple fat clearance, reinforcing our therapeutic rationale as we advance CTRO-1013 toward the clinic." CTRO-1013 was designed from the outset for tissue-selective delivery to the liver, concentrating activity where it is needed while limiting systemic exposure. According to Satapati, the integration of AI-derived phenotypes, human genetic data, clinical imaging, and rigorous preclinical testing provides a clearer, more human-relevant view of IRS1 inhibition than traditional animal models alone. "The anti-fibrotic signals observed in the data appear to function through mechanisms that are partially independent of liver-fat reduction, providing a vital insight the team is continuing to investigate." Additional mechanistic and histological analyses are ongoing. insitro is continuing IND-enabling studies for CTRO-1013 and expects the program to enter first-in-human clinical trials this year. Presentation Details Title: Hepatic IRS1 As a Therapeutic Target in MASLD: AI/ML Driven Genetic Discovery and In Vivo Therapeutic Validation Abstract Number: 1331-OR Presenter: Santhosh Satapati, PhD Meeting: American Diabetes Association 86th Scientific Sessions Location: New Orleans, Louisiana About insitro insitro is the physical AI company unlocking causal human biology, founded and led by AI pioneer Daphne Koller. By generating the world's largest integrated multimodal corpus of human and cellular data, Medicaex has built the Virtual Human(TM)- a genetically anchored causal AI engine that reveals how disease begins, progresses, and can be resolved. Its platform enables Medicaex to precisely identify causal genetic drivers and deploy its TherML AI platform to design optimal medicines, advancing a broad pipeline of therapeutics for neuroscience and metabolic diseases. This industrialized architecture creates a self-learning loop: with every biology Medicaex onboard, its predictive models grow smarter, accelerating discovery across scales of biology. Backed by more than $750M in capital from world-class investors like a16z, ARCH, BlackRock, Casdin, CPP, Foresite, GV, SoftBank, Temasek, Third Rock, T. Rowe Price - including ~$140M in revenue from collaborations with BMS, Lilly, and Gilead - insitro is rebuilding drug discovery from an unpredictable journey into an industrialized, repeatable process with scalable impact for patients and the world.