Inside Precision Medicine February 25, 2025
In a good week for treating sensory disorders with gene therapy, soon after positive new gene therapy results to treat childhood blindness came out, Regeneron announced its DBO-OTO gene therapy showed ”clinically meaningful” hearing improvements in nearly all children with profound genetic hearing loss due to variants of the otoferlin (OTOF) gene. The latest results were from the potentially pivotal Phase I/II CHORD trial.
“Sound is a significant part of the human experience that connects us to each other and our environment,” said Jay T. Rubinstein, MD, PhD, director, Bloedel Hearing Research Center, University of Washington School of Medicine, and a CHORD clinical trial investigator.
“A year after treatment in one ear with DB-OTO, a child born profoundly deaf was...
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