Regeneron announced preliminary, positive safety and efficacy results from the first patient (<2 years of age) dosed in the Phase 1/2 CHORD trial investigating otoferlin gene therapy (DB-OTO) in children with profound genetic hearing loss due to mutations of the otoferlin gen.
“The children who are being enrolled in CHORD are often born with profound hearing loss due to mutations in a single gene, otoferlin, which essentially turns off their auditory circuits,” said Professor Manohar Bance, M.B., an ear surgeon and principal trial investigator at Cambridge University Hospitals NHS Foundation Trust in the United Kingdom. “Cochlear implants are the current standard of care but are unable to replicate the full complexity and range of sound. With these very preliminary DB-OTO results, we now have encouraging evidence that this gene therapy may be able to help turn these auditory circuits back on. We look forward to following this child and others further to determine if DB-OTO gene therapy can restore clinically meaningful hearing as they are learning to interact with the world.”
Congenital hearing loss (hearing loss present at birth) is a significant unmet medical need with no approved pharmacologic treatment options that affects approximately 1.7 out of every 1,000 children born in the U.S. While hearing loss caused by mutations of the otoferlin gene is ultra-rare, the majority of permanent, congenital hearing loss cases diagnosed in developed countries are sensorineural and result from a single gene defect, making them suitable targets for gene therapy.
“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., SVP of Research and co-head of Genetic Medicines at Regeneron. “The ongoing CHORD trial is our first clinical-stage auditory program, and we are incredibly grateful to the investigators and the family of this child for embarking on this breakthrough trial. We remain committed to advancing this research and hope these results mean that children with genetic hearing loss will eventually be able to benefit from the revolutionary promise of gene therapies like DB-OTO.”
👉 Regeneron Shares Preliminary Results Showing Gene Therapy Improves Auditory Responses in Child with Profound Genetic Hearing Loss | Regeneron Pharmaceuticals Inc.
