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Cincinnati Children’s Hospital Medical Center has shared the stories of a brother and sister, both born with profound hearing loss, who regained hearing after receiving a gene therapy for inherited deafness. The therapy, known as DB-OTO and marketed as Otarmeni, targets a form of congenital deafness caused by variants in the OTOF gene. The children were treated under ongoing clinical trials, and the hospital reported that a 3-year-old boy went from detecting virtually no sound to hearing in the normal range within weeks, and has begun developing speech. His younger sister also experienced notable improvement.
The OTOF gene produces otoferlin, a protein needed to transmit sound signals from the inner ear to the hearing nerve. The therapy delivers a working copy of the gene directly into the cochlea through a one-time surgical infusion. In the trial’s first group of 12 children, published in The New England Journal of Medicine, nine gained meaningful hearing improvement, four reached normal to near-normal hearing, and one did not respond, for reasons that are not yet understood.
Several caveats apply. This therapy addresses one specific genetic cause of deafness and does not apply to the many other causes of hearing loss. It remains investigational outside of its approved use, long-term durability is still being studied, and outcomes vary between individuals.
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