OTOF Gene Therapy Restores Hearing in All 10 Patients
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- Karolinska Institutet researchers, collaborating with five hospitals in China, treated 10 patients aged 1–24 with congenital deafness caused by OTOF gene mutations, and every patient showed hearing improvement after a single injection.
- The AAV-based therapy delivers a working copy of the OTOF gene through the round window membrane of the cochlea, with most patients regaining some hearing within one month of treatment.
- Average sound detection improved from 106 decibels before treatment to 52 decibels after six months, according to the study published in Nature Medicine.
- Children aged five to eight showed the most dramatic gains—one seven-year-old girl regained nearly full hearing and could hold everyday conversations with her mother four months after treatment.
- The trial is the first to test the method in teenagers and adults; smaller prior Chinese studies included only young children, according to corresponding author Maoli Duan.
- The treatment was safe and well-tolerated, with the only common side effect being a temporary decrease in neutrophils; no serious adverse reactions occurred over 6–12 months of follow-up.
- Duan said the team is now expanding to other deafness-causing genes including GJB2 and TMC1, which are more common but more difficult to treat.
Why it matters: The 106-to-52 dB shift moves patients from profound deafness into a range where everyday conversation becomes possible—one seven-year-old's experience confirms this. Expanding the trial to teens and adults, previously excluded from prior studies, widens the potential patient pool, and Otovia Therapeutics' commercial backing signals a path toward clinical availability.
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