OTOF Gene Therapy: From Breakthroughs to Roadmaps

Wang Q, Moser T, Petit C, MedComm March 2026.

In a recent study published in Nature Medicine, adeno-associated virus (AAV)-mediated OTOF gene replacement in autosomal recessive nonsyndromic deafness form 9 (DFNB9) patients restored near-physiological hearing and markedly improved speech perception [1]. These results move otoferlin therapy from preclinical promise to a realistic, early-life precision treatment, positioning OTOF as a pathfinder for broader genetic cures for hereditary deafness and motivating scalable trial, manufacturing, and regulatory roadmaps.