DB-OTO for treating patients with hearing loss due to biallelic variants of the otoferlin gene

DB-OTO is in clinical development for the treatment of children with hearing loss due to a mutation in the otoferlin (OTOF) gene. Hearing loss affects a few in every thousand babies born in the UK, but OTOF-related hearing loss is ultra-rare. These mutations lead to a lack of a working otoferlin protein, which is essential for communication between sensors in the ear and the auditory nerves in one’s brain. As a result, all individuals with OTOF-related hearing loss lack the capacity to hear anything (deafness), which may lead to a reduced quality of life due to/such as social isolation or a greater risk of health conditions such as dementia or diabetes. The main treatment for OTOF-related hearing loss is surgical introduction of an electronic implant called a cochlear implant which improves, but does not fully restore, hearing.