Sensorion’s OTOF-GT (SENS-501) gene therapy development program aims to restore hearing in people living with Otoferlin deficiency, one of the most common forms of congenital deafness. The Otoferlin protein is large and is delivered to the inner ear using two standard gene therapy vectors (AAV) and assembled in place.
This dual gene therapy approach has been proved in concept in preclinical studies: the two parts of the Otoferlin gene were successfully delivered in mice, the protein reassembled and hearing restored
GENE THERAPY – DUAL AAV APPROACH
Akil et al. 2019 (link)