Oak Hill Bio announced the publication of rugonersen Phase 1 TANGELO study results in Nature Medicine.
The study provides evidence of improvement in brain activity and developmental ability in children with Angelman syndrome compared to natural history.
“These are promising results that mark a major milestone in the development of disease-modifying therapies for Angelman syndrome,” said Dr. Mark Shen, one of the principal investigators and authors of the paper.
Full press release.
Community Summary of the Results.
Nature Medicine Publication.
April 2025 Press Release announcing Oak Hill Bio’s exclusive rights to rugonersen and plans to initiate a Phase 3 study in patients with Angelman syndrome in early 2026.