Oak Hill Bio Secures $32.5 Million to Propel Rugonersen Therapy for Angelman Syndrome into Phase 3 Trials
Oak Hill Bio Raises $32.5 Million Series A Financing to Advance Antisense Oligonucleotide Therapy Rugonersen to Phase 3 Study in Angelman Syndrome
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Oak Hill Bio has successfully raised $32.5 million in Series A financing to advance its investigational antisense oligonucleotide therapy, rugonersen, into a Phase 3 study targeting Angelman syndrome, a severe neurodevelopmental disorder. The funding was co-led by Balyasny Asset Management, venBio, and Janus Henderson Investors.
- 01The financing round was co-led by Balyasny Asset Management, venBio, and Janus Henderson Investors.
- 02Rugonersen aims to enter Phase 3 clinical trials by mid-2026, targeting approximately 30,000 patients in the U.S. and EU5.
- 03Angelman syndrome, caused by mutations in the UBE3A gene, currently has no approved treatments.
- 04Doug Fambrough, Rich Gaster, and Sandeep Kulkarni have joined Oak Hill Bio's Board of Directors.
- 05Rugonersen is designed to unsilence the paternal UBE3A allele, potentially restoring normal neuronal function.
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Oak Hill Bio, a biotechnology company focused on rare diseases, has announced the successful closure of a $32.5 million Series A financing round, co-led by Balyasny Asset Management, venBio, and Janus Henderson Investors. This funding will support the advancement of rugonersen, an innovative antisense oligonucleotide (ASO) therapy, into a pivotal Phase 3 clinical study aimed at treating Angelman syndrome, a severe neurodevelopmental disorder affecting around 30,000 patients in the U.S. and EU5 with no existing approved therapies. Rugonersen works by targeting the UBE3A-ATS transcript to restore expression of the paternal UBE3A gene in neurons, potentially addressing the underlying causes of Angelman syndrome. The company has also strengthened its leadership team with the addition of three experienced members to its Board of Directors. CEO Josh Distler expressed confidence in the therapy's potential, highlighting the urgent need for effective treatments in the Angelman syndrome community. The Phase 3 study is anticipated to commence in mid-2026, marking a significant step forward in the development of disease-modifying therapies for this condition.
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The advancement of rugonersen into Phase 3 trials could provide a new treatment option for patients suffering from Angelman syndrome, addressing a significant unmet medical need.
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