Regeneron is advancing a gene therapy for a congenital form of deafness toward U.S. regulatory approval after clinical data showed substantial hearing improvement in children. New results published in The New England Journal of Medicine report that a majority of treated patients reached hearing levels that could obviate cochlear implants. The company notified the FDA it will seek approval based on those findings. The trial enrolled children with a specific genetic form of hearing loss; nine of 12 patients achieved functional hearing thresholds in follow-up, according to Regeneron and the NEJM report. The therapy uses a targeted delivery approach to restore function in sensory cells, and the dataset supplied by the company forms the core of the planned approval package. Regulators will assess durability and safety data as well as manufacturing controls before any authorization decision is made.