ATSENA THERAPEUTICS is evaluating ATSN-301, 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. Approximately 20,000 patients in the U.S. and EU have USH1B. These children are born deaf, have vestibular dysfunction and begin to progressively lose vision in their first decade of life. 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. The therapeutic gene to treat USH1B (MYO7A) is too large to fit inside a single AAV vector. To address this barrier, Atsena 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.