Regeneron reported pivotal clinical data showing a gene therapy produced significant hearing improvements in nearly a dozen children with a rare genetic form of deafness, and said it will seek regulatory approval. The study was published in The New England Journal of Medicine and the company described the results as "game-changing." The lead sentence: Regeneron plans to file for approval after NEJM-published data demonstrating measurable audiologic gains in pediatric patients with a specific genetic deafness. The trial reported clinically meaningful improvements on standard hearing measures and functional assessments, with investigators detailing the patient cohort, dosing approach and follow-up time in the NEJM paper. Regeneron’s filing strategy will hinge on durability and safety data; regulators typically require clear risk–benefit evidence for pediatric gene therapies. The NEJM publication and company statements provide the primary sources for the data and next steps. One technical note for readers: the therapy delivers a corrective payload to inner-ear cells via a targeted vector—an intracochlear delivery approach designed to restore protein function in auditory transduction. Industry teams should watch for forthcoming regulatory filings, manufacturing scale plans and any advisory committee signals that will shape label scope and commercialization timelines.