OTOF Gene Therapy Restores Hearing

The FDA approved the first gene therapy to restore hearing for people born deaf from a defective OTOF gene. In a 20‑patient cohort, billions of engineered adeno‑associated viruses carrying a split OTOF transgene were infused into the inner ear, producing measurable hearing within weeks and durable benefit for at least two years. — The decision establishes a regulatory and clinical precedent for in‑ear gene delivery, accelerates prospects for treating other genetic (and eventually common) hearing loss, and raises questions about access, cost, and long‑term safety.