Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness - PubMed
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- SENS-501 is a dual-AAV8 gene therapy designed to treat DFNB9 congenital deafness caused by OTOF mutations.
- In Otof -/- mice, intracochlear SENS-501 injection restored auditory function within 3 weeks, with effects lasting up to 10 months.
- The therapy showed good tolerance in wild-type mice and non-human primates, with vector biodistribution mostly confined to the injection site.
- Preclinical studies support SENS-501's safety and efficacy, leading to an ongoing phase 1/2 clinical trial in children with severe-to-profound hearing loss.