Oak Hill Bio focuses on acquiring and developing rare disease therapeutics that were deprioritized by larger pharmaceutical companies. Its lead program, rugonersen (OHB-724), is an antisense oligonucleotide designed to unsilence the paternal UBE3A allele and has demonstrated promising preclinical and Phase 1 data. Rugonersen was originally developed at Roche and is expected to enter a Phase 3 study in mid-2026. Several former members of the rugonersen program have joined Oak Hill Bio to lead its development. The company was formed in 2024 as a subsidiary of Oak Hill Bio Holdings and is based in Cambridge, Mass. Oak Hill Bio Holdings is also developing OHB-607, a replacement therapy currently in a Phase 2b study.

Total raised

$33M

Funding rounds

1

Latest round

Series A

Latest activity

Jun 2026