Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

You may be eligible if

• Criteria for Inclusion:
• 1. Participants may be of any age, based on Cohort Criteria
• 2. At least two mutations in the otoferlin gene
• 3. Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR
• 4. Preserved distortion product otoacoustic emissions (DPOAEs)
• 5. Able and willing to comply with all study requirements, including willingness to participate in a separate long term follow-up study after completion of this trial
• Criteria for Exclusion:
• 1. Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy
• 2. Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF
• 3. Prior participation in a clinical trial with an investigational drug, within six months prior to administration, or any prior participation in a gene therapy clinical trial