- Series A financing co-led by Balyasny Asset Management, venBio, and Janus Henderson Investors
- Rugonersen, a potentially best-in-class antisense oligonucleotide (ASO) therapy for Angelman syndrome, is expected to enter a Phase 3 clinical study in mid-2026
- Doug Fambrough, Rich Gaster, and Sandeep Kulkarni join board of directors
CAMBRIDGE, Mass., June 01, 2026 (GLOBE NEWSWIRE) — Oak Hill Bio, a clinical-stage rare disease therapeutics company, today announced the closing of a $32.5M Series A financing. The round was co-led by Balyasny Asset Management, venBio, and Janus Henderson Investors with participation from KCap Biotechnology Fund.
Proceeds from the financing will be used to advance rugonersen, an investigational ASO therapy for the treatment of Angelman syndrome, into a pivotal Phase 3 clinical study. Angelman syndrome is a devastating neurodevelopmental disorder affecting approximately 30,000 diagnosed patients in the U.S. and EU5 with no approved treatments. Rugonersen was originally developed by Roche as a highly potent and specific therapy to restore UBE3A production in neurons. Several former members of the rugonersen program have joined Oak Hill Bio to lead development.
“We are thrilled to welcome these exceptional investors and company-builders to our team. We really appreciate that they’ve recognized the strong science and data behind rugonersen and the tremendous need for a disease-modifying therapy in the Angelman syndrome community,” said Josh Distler, Chief Executive Officer of Oak Hill Bio. “With the financing and additions to our board, we are well-positioned to advance rugonersen into a Phase 3 study in the middle of 2026.”