Regeneron Pharmaceuticals announced promising results from its Phase 1/2 CHORD trial for DB-OTO, an investigational gene therapy aimed at treating profound genetic hearing loss caused by otoferlin (OTOF) gene variants. In the trial, 10 of 11 children who received at least one post-treatment assessment showed significant hearing improvements. The first child dosed demonstrated near-normal hearing levels at key speech frequencies and notable speech development over 72 weeks.

The therapy, which uses an intracochlear injection to deliver a functional OTOF gene, was well tolerated, with only transient post-surgical vestibular effects reported. DB-OTO has received multiple designations from the U.S. FDA, including Orphan Drug and Fast Track, but remains under clinical investigation.

The CHORD trial continues to enroll participants in the U.S., U.K., and Spain.

source: Regeneron Pharmaceuticals, February 24, 2025.