Gene therapy brings near-normal hearing to most with inherited deafness in largest trial
In the largest trial to date, a gene therapy delivering functional copies of the OTOF gene to the inner ear improved hearing in about 90% of participants (38 of 42) with congenital deafness caused by OTOF mutations, across eight sites in China. Improvements began within weeks and persisted for up to two years in many cases; some participants achieved normal conversational hearing, while a few showed no benefit. The therapy had no serious side effects, though some mild vertigo or inner-ear inflammation occurred, and younger patients tended to improve more. If further trials confirm long-term safety and broader applicability, the approach could offer a one‑and‑done alternative or complement to cochlear implants, with next steps including longer follow-up and dosing studies.
