Regeneron disclosed NEJM‑published data showing that nine of 12 children treated with its gene therapy for a congenital form of deafness achieved hearing levels where cochlear implants were no longer required, and the company said it will pursue FDA approval. The study reported durable functional gains in a small pediatric cohort and supports a filing strategy built on robust, clinically meaningful endpoints. The readout provides rare high‑impact positive human data for a pediatric gene therapy and will prompt regulators to weigh benefit versus known gene‑therapy risks in young children. Regeneron indicated preparations to move toward regulatory submission and briefing materials based on the NEJM dataset. Clinicians and payers will closely monitor durability and safety in extended follow‑up; if approved, the therapy could shift treatment paradigms for the targeted form of congenital hearing loss.