Roche's rugonersen finds a new home

Cambridge, USA-based biotech Oak Hill Bio, which is focused on acquiring and developing life-changing rare disease therapeutics, has entered into an exclusive license agreement with Roche (ROG: SIX) to obtain global rights for rugonersen (RO7248824).

Rugonersen is an ASO designed as a potential best-in-class treatment for individuals with Angelman syndrome, a rare, genetic neurodevelopmental condition for which there is no approved disease-modifying treatment. Oak Hill plans to initiate a Phase III study in early 2026.

Roche dropped development of rugonersen in 2023, when it reported Phase I trial results that showed a lower level of efficacy than the Basel-based pharma giant had hoped for, rather than any safety concerns. At the time, Roche said it would seek a partner for the drug,