Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness.
A gene therapy restored hearing in deaf mice within weeks and is now being tested in people with a common genetic form of deafness.
SENS-501, a dual-AAV8 otoferlin gene therapy, restored hearing in congenitally deaf mice within 3 weeks with effects sustained to 10 months, and demonstrated acceptable safety in NHP GLP toxicology studies; a phase 1/2 clinical trial in DFNB9 patients is underway. DFNB9 (caused by OTOF mutations) is one of the most prevalent forms of genetic non-syndromic sensorineural hearing loss.
What the study was
- Study design
- Preclinical study with GLP toxicology in NHP
- Population
- Otof-/- mouse model; non-human primate toxicology
- Category
- Drug Development
- Maturity
- Exploratory
- Journal
- Molecular Therapy Advances
Why it surfaced
Promising gene therapy with NHP safety data and active phase 1/2 trial for rare congenital deafness. Preclinical, so capped at 6.
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