DB-OTO, a dual AAV1 gene therapy encoding human otoferlin, met prespecified week-24 hearing endpoints in 9 of 12 pediatric patients with biallelic OTOF-related profound deafness. Treated ears showed improved pure-tone thresholds and auditory brain-stem responses; some patients demonstrated speech-perception gains with follow-up extending to 72 weeks. Delivery was a single intracochlear infusion; adverse events were mostly related to surgical delivery, with two resolved serious events and no persistent vestibular findings. The study was small, open-label, and single-group, with limited primary follow-up and some endpoints not originally prespecified.

Source: The New England Journal of Medicine