2023-10-27 07:02:48 ET
Regeneron ( NASDAQ: REGN ) on Friday announced positive results from a trial investigating its otoferlin gene therapy (DB-OTO) in children with profound genetic hearing loss due to mutations of the otoferlin gene.
DB-OTO is an investigational cell-selective, adeno-associated virus gene therapy designed to provide durable, physiological hearing to individuals with congenital hearing loss caused by otoferlin gene mutations.
Preliminary data from the first patient (<2 years of age) dosed in the Phase 1/2 CHORD trial showed positive safety and efficacy results. The study marks Regeneron's ( REGN ) first auditory program and is currently enrolling patients.
"These preliminary DB-OTO results provide early and encouraging proof-of-concept for the treatment of otoferlin-related hearing loss, as well as our pipeline of gene therapies to address more common forms of genetic hearing loss and other therapeutic areas," said Christos Kyratsous, Ph.D., Senior Vice President of Research and co-head of Genetic Medicines at Regeneron.
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Regeneron's hearing loss gene therapy shows positive outcomes in trial