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Regeneron Sees Path To Market For Deafness Gene Therapy

 
• By Alaric DeArment

The drug maker presented data from a Phase I/II trial of DB-OTO that it plans to use to seek regulatory approval.

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• Source: Shutterstock

Regeneron Pharmaceuticals intends to pursue the Phase I/II CHORD trial of DB-OTO in a genetically linked form of deafness among children, having presented positive early results from the trial at the Association for Research in Otolaryngology (ARO)’s annual mid-winter meeting.

The Tarrytown, NY-based company presented data on 24 February from 12 children enrolled in CHORD who had profound genetic hearing loss due to variants in the otoferlin (OTOF) gene, including 72-week results showing speech and development progress in the first child, who was dosed at 10 months of age, along

Key Takeaways
  • Regeneron presented positive data for the gene therapy DB-OTO and plans to use the Phase I/II study for pivotal development.
  • The results showed marked improvements in children receiving the therapy, including one who was 72 weeks out and showed progressive improvement

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