DB-OTO

DB-OTO is an investigational, adeno-associated virus (AAV)-based gene therapy designed to treat congenital hearing loss caused by biallelic mutations in the otoferlin (OTOF) gene. The therapy delivers a functional copy of the OTOF gene directly into the cochlea via intracochlear injection, using two AAV1 vectors that together encode the full-length otoferlin protein. By restoring otoferlin expression specifically in cochlear inner hair cells through a cell-selective Myo15 promoter, DB-OTO aims to enable these cells to produce the missing protein and restore auditory function. It is being developed primarily for infants and children with severe-to-profound sensorineural hearing loss due to OTOF mutations. Clinical trials have shown promising results, with most treated children experiencing clinically meaningful improvements in hearing and speech perception[1][4][5][6][7][10].

Brand names

Otarmeni

Other names

DB OTODB XX2