Overview

• Trial participants aged 1 to 24 regained hearing from a 106-decibel threshold to a 52-decibel average within six months after a single injection
• This first-in-patient AAV-OTOF trial marks the first demonstration of gene therapy restoring auditory function in adolescents and adults as well as young children
• No serious adverse events were reported over six to twelve months of follow-up, with the most common side effect being a transient drop in neutrophil counts
• Brainstem response measurements improved by an average of 62% and behavioral hearing assessments rose by 78%, with some participants detecting speech within weeks of treatment
• Researchers plan to apply the same AAV platform to target other genes linked to genetic deafness, including GJB2 and TMC1