Oak Hill Bio Secures $32.5 Million Series A Funding to Advance Angelman Syndrome Therapy

Oak Hill Bio, a clinical-stage biotechnology company based in Cambridge, Massachusetts, has raised $32.5 million in a Series A funding round.

The financing was co-led by Balyasny Asset Management, venBio, and Janus Henderson Investors. KCap Biotechnology Fund also participated in the round.

The company plans to use the proceeds to advance rugonersen, its investigational antisense oligonucleotide (ASO) therapy, into a pivotal Phase 3 clinical trial. The therapy is being developed as a potential treatment for Angelman syndrome, a rare neurodevelopmental disorder that currently lacks approved disease-modifying therapies.

Funding to Support Late-Stage Clinical Development

Oak Hill Bio focuses on acquiring and developing rare disease treatments that have been deprioritized by larger pharmaceutical companies.

Its lead candidate, rugonersen (OHB-724), is entering Phase 3 development. The company believes the therapy could offer a new treatment option for patients living with Angelman syndrome.

The latest funding is expected to support clinical development activities and preparations for the upcoming study.

Company Leadership and Focus

The company is led by Chief Executive Officer Josh Distler.

Oak Hill Bio operates as a clinical-stage biotechnology company with a focus on rare diseases. Its strategy centers on identifying promising therapies and advancing them through later stages of development.

New Board Appointments Announced

Alongside the financing, Oak Hill Bio announced three additions to its Board of Directors.

Doug Fambrough

Doug Fambrough is the founder and portfolio manager of KCap Biotechnology Fund. He brings experience in RNA therapeutics, drug development, company formation, and business development.

Previously, he founded Dicerna Pharmaceuticals and served as its President and Chief Executive Officer. Dicerna developed RNA interference-based medicines before its acquisition by Novo Nordisk in 2021.

Rich Gaster

Rich Gaster serves as Managing Partner at venBio.

His background includes medicine, entrepreneurship, and life sciences investing. Over the years, he has been involved in launching and supporting several biotechnology companies within the venBio portfolio.

Sandeep Kulkarni

Sandeep Kulkarni has more than 20 years of experience across biotechnology, investing, entrepreneurship, and medicine.

He is currently the Co-Founder, Chief Executive Officer, and Director of Zura Bio. Earlier in his career, he co-founded Tourmaline Bio and served as its Chief Executive Officer before the company was acquired by Novartis in 2025.Oak Hill Bio, a clinical-stage biotechnology company based in Cambridge, Massachusetts, has raised $32.5 million in a Series A funding round.

The financing was co-led by Balyasny Asset Management, venBio, and Janus Henderson Investors. KCap Biotechnology Fund also participated in the round.

The company plans to use the proceeds to advance rugonersen, its investigational antisense oligonucleotide (ASO) therapy, into a pivotal Phase 3 clinical trial. The therapy is being developed as a potential treatment for Angelman syndrome, a rare neurodevelopmental disorder that currently lacks approved disease-modifying therapies.

Funding to Support Late-Stage Clinical Development

Oak Hill Bio focuses on acquiring and developing rare disease treatments that have been deprioritized by larger pharmaceutical companies.

Its lead candidate, rugonersen (OHB-724), is entering Phase 3 development. The company believes the therapy could offer a new treatment option for patients living with Angelman syndrome.

The latest funding is expected to support clinical development activities and preparations for the upcoming study.

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