Restoring hearing in people with a rare form of deafness

A gene therapy delivering a functional OTOF gene to inner-ear hair cells improved hearing in around 80% of participants within 24 weeks in the CHORD clinical trial, which enrolled 24 participants aged between 10 months and 16 years with OTOF-associated hearing impairment. Each participant received Otarmeni as a one-time infusion, either in one ear or in both ears. In April, Otarmeni became the first gene therapy for hearing loss approved by the US Food and Drug Administration.