Advancing Rare Disease Therapies Across the Finish Line
Drug development is a marathon, often taking years to complete. This is especially true in rare diseases, where the development path is rarely linear.
Unfortunately, this process sometimes requires a relay, as corporate strategy shifts result in promising programs getting deprioritized mid-way through development.
Oak Hill Bio was founded to take the baton where others left off—acquiring and developing outstanding deprioritized late-stage rare disease drugs and bringing them across the finish line.
In February 2025, we obtained global rights for rugonersen, an investigational antisense oligonucleotide for the treatment of individuals with Angelman syndrome.
Advancing
Breakthroughs
Oak Hill Bio is a clinical-stage biotechnology company focused on acquiring and developing outstanding rare disease drugs that have been deprioritized by big pharma. Rugonersen (OHB-724) is an investigational Phase-3 ready oligonucleotide that has best-in-class potential as a treatment for Angelman syndrome, a devastating neurodevelopmental disease with a large unmet net.
In The Press
If you're looking for information about Oak Hill Bio Holdings, OHB Neonatology, or OHB-607 please visit www.oakhillbioholdings.com.
