Hearing Loss

Regeneron’s DB-OTO gene therapy showed significant hearing improvements in children with OTOF-related hearing loss in the CHORD trial. 11 of 12 participants demonstrated clinically meaningful hearing gains, with 3 achieving normal hearing. The trial met its primary endpoint, and improvements were durable up to 72 weeks. DB-OTO was generally well-tolerated. Regeneron plans to file for U.S. regulatory approval later in 2025, pending FDA discussions. DB-OTO holds Orphan Drug and other FDA designations.

Sensorion reported positive H1 2025 results, highlighting progress in gene therapies. The Audiogene Phase 1/2 trial for SENS-501 (OTOF-GT) showed a positive safety profile and early hearing improvement signs. Enrollment in SENS-401’s Phase 2a Cisplatin-Induced Ototoxicity study is complete, with topline data expected in H2 2025. GJB2-GT is on track for a Q1 2026 CTA filing. The company’s cash position of €57.1m funds operations into Q3 2026. R&D expenses increased slightly, reflecting intensified clinical trial activities. Net loss was -€16.0 million.