SENS-501

SENS-501 is an investigational gene therapy designed to treat severe to profound hearing loss caused by mutations in the otoferlin (OTOF) gene. This rare genetic disorder leads to a deficiency of the otoferlin protein, which is essential for transmitting sound signals from the inner ear to the brain. SENS-501 uses a dual adeno-associated virus serotype 8 (AAV8) vector system to deliver and assemble a functional copy of the human OTOF gene directly into cochlear hair cells via intracochlear injection. The goal is to restore physiological hearing by enabling production of full-length otoferlin protein in affected children. The therapy is being developed primarily for pediatric patients aged 6–31 months with pre-lingual hearing loss due to biallelic OTOF mutations. Clinical trials are ongoing, with early data showing promising safety and initial signs of efficacy[1][2][3][5][6][7][9][10].