ATSENA is making rapid advancements in evaluating a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher Syndrome (USH1B), an inherited disease that affects the retina and the inner ear. Four to seventeen children per 100,000 are born with Usher Syndrome. These children are born with varying degrees of hearing and vision impairment depending on the clinical subtype. There is currently no cure for USH1B-related vision loss, while cochlear implants can address deafness.
USH1B is caused by mutations in the MYO7A gene. USH1B patients are born profoundly deaf and begin to progressively lose vision in their first decade. The therapeutic gene to treat USH1B (MYO7A) is too large to fit inside a single AAV vector. To address this barrier, Atsena Therapeutics developed dual AAV vectors capable of delivering this large genetic payload, marking a potentially major advance in preventing blindness in patients affected by this devastating multisensory disorder.