OTOF – Medxy AI

AAV‑OTOF Gene Therapy Restores Substantial Hearing in DFNB9 Across Ages — Rapid, Age‑dependent Benefits in a First-in‑Human Trial

By MedXY 11/04/2025

A single‑arm trial of AAV‑OTOF (Anc80L65) in 10 patients (1.5–23.9 years) with autosomal recessive deafness 9 showed good tolerability and rapid, clinically meaningful hearing gains, with optimal outcomes in children aged 5–8 years; longer follow‑up is required.

news Otorhinolaryngology Pediatrics

OTOF Gene Therapy Rivaled—and in Some Domains Outperformed—Cochlear Implants in Restoring Hearing and Speech in Congenital Deafness

By MedXY 11/04/2025

A 2022–2024 nonblinded cohort study found OTOF gene therapy produced rapid, durable gains in auditory thresholds, speech perception, mismatch-negativity responses, and music perception versus cochlear implantation in children with genetically driven congenital deafness.

news Otorhinolaryngology Pediatrics

DB-OTO Restores and Sustains Hearing in Otoferlin-Deficient Mice: A Hair-Cell–Targeted Dual-AAV Strategy Advances to Clinical Testing

By MedXY 11/04/2025

DB-OTO, a hair-cell-specific dual-AAV gene therapy encoding full-length human otoferlin, achieved dose-dependent and sustained auditory restoration in OTOF-deficient mice, supporting an ongoing Phase I/II pediatric trial.