DB-OTO Delivers Dramatic Hearing Improvement: 11 of 12 Children Respond to Therapy
In a remarkable advancement for children born with profound genetic hearing loss, Regeneron Pharmaceuticals has unveiled new data from its CHORD trial of DB-OTO—a gene therapy for otoferlin (OTOF) gene-related deafness—showing that 11 out of 12 participants experienced clinically meaningful hearing gains. Even more impressively, three of those children achieved normal hearing levels, with improvements sustained or progressing further among those with extended follow-up.
Three Children Achieve Normal Hearing; Improvements Persist Beyond 36 Weeks
Among the 12 trial participants (ranging from 10 months to 16 years old), nine received therapy in one ear and three in both ears. Nearly all (14 out of 15 treated ears) showed improved hearing within weeks of the procedure, which uses a method similar to cochlear implantation but enables earlier intervention—even in infants.
The stability and durability of these outcomes are clear: Of the eight participants followed for at least 36 weeks (up to 72 weeks), all maintained or improved upon their gains. In a rare development, three children’s hearing improved to normal, enabling one to understand whispered speech—a first for a group previously facing permanent silence.
| Key DB-OTO CHORD Trial Outcomes | Number of Participants |
|---|---|
| Clinically meaningful hearing improvement | 11/12 |
| Achieved normal hearing levels | 3/12 |
| Stable/continued improvement at =36 weeks | 8/8 |
| Significant speech improvement (at 48+ weeks) | 3/3 |
| Adverse findings related to DB-OTO | 0 |
Speech Perception Boosts Quality of Life
The improvements were not limited to audiology charts. Of three children who completed detailed speech assessments at 48+ weeks, all made marked gains, including one child now able to understand spoken words without needing to lip-read or rely on assistive devices—even in noisy environments. Families have described the transformation as “unimaginable” compared to their situation just one year prior.
No Therapy-Related Adverse Findings Observed
Importantly, neither DB-OTO nor the procedure to administer it resulted in any reported adverse events directly attributable to the gene therapy. The most significant side effects, such as post-surgical dizziness and nausea, were temporary and resolved completely. Two serious adverse events were deemed unrelated to DB-OTO itself.
U.S. Regulatory Submission Expected: What’s Next for DB-OTO?
With DB-OTO already granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations, Regeneron aims to file for U.S. approval later this year, pending final FDA discussions. While DB-OTO’s ultimate regulatory fate remains ahead, these robust results underscore its promise for addressing an unmet medical need in the 20–50 U.S. newborns annually affected by OTOF gene mutations.
Key Takeaway: Transformative Potential in Ultra-Rare Genetic Hearing Loss
Regeneron’s gene therapy data mark a new era in pediatric hearing loss. For investors, clinicians, and families, the stability and scope of hearing and speech improvement documented in the CHORD trial hint at not only the program’s clinical viability but also its significant market potential, pending successful regulatory review. This case also demonstrates the emerging impact of gene therapies in rare genetic disorders—a space that may increasingly define Regeneron’s innovative pipeline in years to come.
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