insitro, the AI therapeutics company built on causal biology, today announced the appointment of Hideo Makimura as Chief Medical Officer, effective Sept. 14. Makimura will lead clinical development strategy and execution as insitro advances its AI-derived metabolic disease and neuroscience pipeline toward first-in-human trials — a key step in the company’s evolution toward clinical-stage development.

Makimura joins as insitro transitions its initial therapeutic candidates toward inaugural clinical trials. The company’s pipeline is anchored in human genetic evidence – targets with human genetic support have historically been two to four times more likely to reach approval – and his mandate is to carry that advantage into the clinic through operational excellence, biomarker strategy, and innovative trial design.

“Hideo has spent his career using human genetics and biomarkers to guide and accelerate clinical programs from initial patient studies through late-stage development,” said Daphne Koller, founder and CEO of insitro. “He has taken more than a dozen programs into the clinic across nearly every modality, and spanning all of insitro’s therapeutic areas. As a scientist and physician, he combines scientific rigor with firsthand experience caring for the patients these therapies are designed to help. As insitro heads into the clinic, I could not imagine a better partner to help us translate the insights generated by our platform into meaningful medicines for patients.”

Makimura brings a two-decade record of academic work and clinical execution across modalities and stages. He has personally led programs from early-stage human testing through Phase 4 clinical trials, spanning small molecules, large molecules (antibodies), therapeutic peptides, small interfering RNA (siRNA), and adeno-associated virus (AAV) gene therapy, with more than a dozen successful investigational new drug (IND) and clinical trial application (CTA) filings.

Most recently, as Chief Medical Officer of Affinia Therapeutics, he led clinical development, clinical operations, regulatory, and quality functions. He, along with his team, advanced AFTX-201, an AAV gene therapy for BAG3-associated dilated cardiomyopathy, to the U.S. Food and Drug Administration (FDA) acceptance of its IND, Health Canada authorization of its CTA, and FDA Fast Track designation, and received the FDA’s Orphan Drug designation. His efforts supported Affinia’s successful Series C financing.

Before Affinia, he spent six years at Johnson & Johnson (J&J), rapidly rising to Global Vice President and Head of Global Translational and Experimental Medicine, responsible for early clinical development from late discovery through Phase 2a across cardiometabolic, kidney, retinal, and pulmonary disease. In 2024, he was appointed Global Head of Research & Development for Specialty Ophthalmology within J&J’s neuroscience therapeutic area.

His work has consistently used human genetics to de-risk development. At J&J, he led early clinical development of a GalNAc-siRNA targeting PNPLA3 in metabolic dysfunction-associated steatohepatitis (MASH) through Phase 1, with results published in the New England Journal of Medicine. Earlier, at Merck, he served on the global new drug application (NDA) filing team for ertugliflozin, the SGLT2 inhibitor approved by the FDA in 2017, gaining experience that spanned the full arc of development through approval.

Makimura began his career in academic medicine as an NIH-funded faculty member at Harvard Medical School, where his research and clinical practice focused on metabolic and endocrine disease. He earned his M.D. and a Ph.D. in neuroscience from the Icahn School of Medicine at Mount Sinai and is board certified in endocrinology, diabetes, and metabolism, a foundation at the intersection of the two therapeutic areas insitro is advancing.

“insitro has built a fundamentally distinct platform by grounding its AI models in causal human data and the complex realities of human genetics, principles I have long championed,” said Makimura. “My focus will be on building a world-class clinical organization that leverages biomarkers and precision patient selection to give our therapeutic programs the highest probability of success in the clinic. This is an important moment for insitro, and I’m thrilled to join the team as we work to translate the promise of the platform into medicines for patients.”

About insitro

insitro is the physical AI company unlocking causal human biology to systematically advance high-impact therapeutics to treat grievous illness, founded and led by AI pioneer Daphne Koller. Our thesis: biology remains illegible to frontier AI for lack of the scaled human and cellular data needed to unlock true domain intelligence. Since 2018, we’ve 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 our TherML™ platform, which designs medicines against the causal drivers it identifies. Every disease we interrogate makes the model smarter, compounding our lead as the corpus grows.

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

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