DB-OTO Gene Therapy - Breakthrough in hearing restoration

New data from Regeneron's Phase 1/2 CHORD trial shows clinically meaningful improvements in hearing for nearly all children with profound genetic hearing loss caused by OTOF gene variants. As presented at ARO earlier this year, 10 out of 11 children showed significant hearing gains after treatment with the investigational gene therapy DB-OTO, with speech and developmental progress already observed in the first child treated. Findings were recently published in an article in the New England Journal of Medicine co-authored by UW Oto-HNS professor Jay Rubinstein, MD, PhD.

“Sound is a significant part of the human experience that connects us to each other and our environment,” said Dr. Rubinstein, a CHORD clinical trial investigator. “A year after treatment in one ear with DB-OTO, a child born profoundly deaf was able to enjoy music, engage in imaginative play and participate in bedtime reading when the cochlear implant on their other ear was removed. These seemingly small interactions are life-changing for these children as well as their families, and these results continue to underscore the revolutionary promise of DB-OTO as a potential treatment for otoferlin-related hearing loss.” See the full article in the New England Journal of medicine. 

The pre-clinical research was conducted at the Primate Center by UW Oto-HNS professor Jim Phillips, PhD, and Jayme Richards, while the clinical trial surgeries and subject testing were carried out at Seattle Children's Hospital by Meg Meredith, AuD, CCC-A, and Wendy James.