Oak Hill Bio

Oak Hill Bio is a clinical-stage biotechnology company that acquires and develops deprioritized late-stage therapeutics for rare diseases. The company launched in February 2022, founded by Josh Distler and Tauhid Ali, with a business model centered on advancing promising drug candidates that larger pharmaceutical companies have discontinued, often due to strategic shifts. This strategy was initiated with the acquisition and licensing of a six-program pipeline from Takeda Pharmaceutical, which in return received an upfront payment, an ownership stake, and potential future milestones and royalties.

Co-founder Josh Distler, who now serves as CEO, has a background in building and investing in biotechnology companies, including roles at Athanor Capital and the D. E. Shaw Group. The company is headquartered in Cambridge, Massachusetts, with operations in the United Kingdom. In June 2026, Oak Hill Bio secured a $32.5 million Series A financing round to advance its lead programs.

The company's pipeline is focused on neonatology and rare autoimmune diseases. Key programs include: • **Rugonersen:** An antisense oligonucleotide (ASO) therapy for Angelman syndrome, a severe neurodevelopmental disorder. Acquired from Roche, it is designed to restore the function of the UBE3A gene. Oak Hill plans to advance Rugonersen into a Phase 3 trial in mid-2026. • **OHB-607:** A recombinant human insulin-like growth factor 1 (IGF-1) replacement therapy aimed at preventing complications in extremely premature infants, such as bronchopulmonary dysplasia (BPD). Infants born before 28 weeks have low IGF-1 levels, which is crucial for organ development. OHB-607 is currently in a Phase 2b trial in collaboration with Chiesi Group. • **OHB-101:** A first-in-class soluble recombinant version of the FcγR2B receptor designed to treat a range of immune complex-driven autoimmune diseases by preventing the inflammatory cascade. It has been studied for conditions like lupus and immune thrombocytopenia. • **OHB-401:** An oral plasma kallikrein (pKAL) inhibitor in preclinical development for diabetic macular edema.

Keywords: clinical-stage biotechnology, rare disease therapeutics, deprioritized drugs, neonatology, autoimmune diseases, Angelman syndrome, rugonersen, antisense oligonucleotide, ASO, premature birth complications, OHB-607, recombinant IGF-1, bronchopulmonary dysplasia, OHB-101, FcγR2B receptor, diabetic macular edema, OHB-401, Takeda, Chiesi Group, orphan drugs, asset acquisition, in-licensing, clinical development, pediatric rare diseases, neurodevelopmental disorders, immunology, drug pipeline, biopharmaceutical