insitro Announces a Strategic Collaboration with AstraZeneca for AI-enabled Discovery of Novel Therapeutics for MASH and Cardiomyopathies

Collaboration applies insitro's Virtual Human causal AI discovery platform to two diseases defined by patient heterogeneity and high unmet medical need

insitro to receive $40 million upfront and up to approximately $1.46 billion in potential discovery, development, and commercial milestones, and tiered royalties

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--insitro, an AI company unlocking causal human biology to systematically generate high-value therapeutics to treat grievous illness, today announced a worldwide exclusive research collaboration agreement with AstraZeneca to discover novel targets and therapeutics in metabolic dysfunction-associated steatohepatitis (MASH) and cardiomyopathies. Under the terms of the agreement, insitro will receive $40 million upfront and is eligible for up to approximately $1.46 billion in potential discovery, development, and commercial milestones. In addition, single-digit royalties will be based on potential global commercial sales.

Under the agreement, insitro will apply its Virtual Human™ platform to identify causal disease biology in both indications and advance AstraZeneca-nominated targets from discovery through to development candidate. AstraZeneca will lead all clinical development, regulatory work, and commercialization activities.

"MASH and cardiomyopathies are prevalent, underdiagnosed diseases where high-content data holds immense potential for uncovering disease drivers. By applying AI to these complex datasets, we can unlock greater discovery power and reveal novel genetic insights," said Daphne Koller, Ph.D., founder and CEO of insitro. "Our platform simultaneously deconvolutes disease biology and identifies causal drivers of disease pathologies. Partnering with AstraZeneca allows us to pair our deep biological precision with their pharmaceutical expertise and scale with potential for accelerated advancement of novel programs through development and to patients."

Both diseases are defined by the problem the collaboration is built to address. In each case, a single diagnostic label spans patients whose underlying biology differs. The heterogeneity across these diseases has made them difficult to treat, and the condition under which insitro’s existing machine learning capabilities on human data is most useful.

Sharon Barr, Executive Vice President and Head of BioPharmaceuticals R&D, AstraZeneca, said, “We know that disease biology is incredibly complex and one of the most critical decisions in R&D is selecting the right target. This strategic collaboration underscores our commitment to maximizing the potential of cutting-edge AI to tackle the root cause of disease. Partnering with insitro, we are using AI to identify the biological drivers of disease to support the rapid discovery of high-quality novel medicines, advancing our ambition to deliver the next generation of medicines to patients faster.”

About insitro

insitro is the physical AI company unlocking causal human biology to systematically advance high-value therapeutics to treat grievous illness, founded and led by AI pioneer Daphne Koller. The thesis: biology remains illegible to human and machine intelligence for lack of the scaled human and cellular data needed to unlock true domain intelligence to discover better medicines. Simply, you cannot connect the dots that have not been measured. Since 2018, insitro has built the world’s largest integrated multi-modal corpus of human and cellular data; the Virtual Human™, a genetically anchored AI engine that reveals how disease begins, progresses, and can be resolved; and the TherML™ platform, which designs medicines against the causal drivers it identifies. Every interrogated disease makes the model smarter, compounding our lead as the corpus grows. The long-term goal is to make health predictable by turning biology into a true AI problem.

insitro is advancing a first-in-class pipeline of nine programs, including novel genetic master regulators of liver fat and fibrosis entering the clinic in early 2027, a proprietary oligonucleotide therapy with disease-modifying potential in 97% of ALS patients, and the first reported genetic regulator of brown fat for metabolic health and longevity. The company is backed by $750M+ in capital from world-class investors including a16z, ARCH, BlackRock, CPP, Deviation, Foresite, GV, T. Rowe Price, and Temasek, alongside collaborations with BMS, Lilly, and Gilead.

Learn more at insitro.com and deepphenotype.substack.com.