NEJM: Regeneron's DB-OTO gave clinically meaningful hearing to 11 of 12 children born deaf from OTOF mutations

Regeneron Pharmaceuticals/Otarmeni (DB-OTO)

Interim results of the CHORD trial, presented at the AAO-HNSF meeting and published in NEJM, showed that a one-time dual-AAV gene therapy restored meaningful hearing in 11 of 12 children with OTOF deafness. Three reached normal hearing levels. Regeneron said it planned to file for US approval before the end of 2025.

• 11 of 12 patients (14 of 15 treated ears) improved; 9 met the primary goal of hearing at 70 dB HL or better at 24 weeks.
• 6 could hear soft speech without a device and 3 could detect whispers.
• In 8 patients followed 36 weeks or more, hearing improved or held steady for up to 72 weeks.
• No adverse events were judged related to DB-OTO itself; short-lived dizziness and nausea after surgery resolved.