OTOF Clinical Trials 2025 Dual-AAV Validation
Five clinical trials running for OTOF/DFNB9 using dual-AAV. This is the most advanced gene therapy work in hearing loss right now.
Regeneron DB-OTO (CHORD trial): Published in NEJM, October 2025. 11 out of 12 children showed improved hearing. 3 achieved normal hearing. FDA submission planned. This is the closest to market.
Eli Lilly AK-OTOF: An 11-year-old, deaf for more than 10 years, had hearing restored across all frequencies in 30 days. That’s a remarkable result for someone with over a decade of auditory deprivation.
Sensorion AUDIOGENE: 5 patients dosed. Early positive signals but less mature data than the other two.
The takeaway for STRC: dual-AAV platform is proven in humans. It works. Our transduction model shows a 2.8x advantage for single-vector approaches, but “lower efficiency” doesn’t mean “doesn’t work.” It clearly does.
Important caveat: OTOF targets inner hair cells (synaptic release protein). STRC targets outer hair cells (structural protein). Different cell type, different biology, different challenges. OHCs are harder to transduce with current AAV capsids. But the delivery platform itself is validated.
Connections
- STRC Gene Therapy — dual-AAV platform validation for hearing
- Dual Vector AAV Strategy — clinical proof that dual-AAV works in cochlea
- STRC Dual-Vector vs Single-Vector Transduction — our model predicts 2.8x single-vector advantage
- DFNB16 Hearing Loss — STRC target vs OTOF target comparison
- STRC Gamma-Poisson Transduction Model — efficiency modeling context