Otoferlin gene-mediated hearing loss research hub

A Phase 1/2 Trial of AAVAnc80-hOTOF Gene Therapy in Individuals with Sensorineural Hearing Loss due to Biallelic Otoferlin Gene (OTOF) Mutations

This study is testing a new gene therapy, AAVAnc80-hOTOF, for people with hearing loss caused by a specific genetic problem called otoferlin gene mutations. It's an early-stage study to see if the treatment is safe and to check for side effects.