Clinical Trials Arena May 9, 2024
The two children with rare genetic otoferlin hearing loss enrolled in the Phase I/II trial for Regeneron’s gene therapy observed improvements in hearing.
Regeneron Pharmaceuticals’ gene therapy for otoferlin hearing loss, DB-OTO, has restored hearing to normal in an 11-month-old child within 24 weeks and improved hearing in another four-year-old child at six weeks.
The data is from an open-label Phase I/II CHORD trial (NCT05788536), which was presented at the 2024 American Society of Gene and Cell Therapy (ASGCT) annual conference taking place in Baltimore from 7 to 11 May.
As part of the CHORD study, both children received a single intracochlear injection of DB-OTO in one ear. The trial’s secondary endpoints include hearing improvement, measured using auditory brainstem response...
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