Overview

• Ten patients aged one to 24 at five hospitals in China received a single AAV-OTOF injection through the cochlear round window in a Karolinska Institutet–led trial
• Most participants began regaining some hearing within one month of treatment
• Pure-tone averages improved from 106 decibels at baseline to 52 decibels at six months, demonstrating substantial functional gains
• No serious adverse events were reported over six- to 12-month follow-up; the most common mild side effect was a transient reduction in neutrophil counts
• Researchers have launched extended monitoring to assess long-term durability and are planning to adapt the AAV approach to other genes causing genetic deafness