ATSN-301

**ATSN-301** is a dual adeno-associated virus (AAV) vector-based gene therapy under development by Atsena Therapeutics for the treatment of Usher syndrome type 1B (USH1B), a rare inherited condition characterized by congenital deafness, balance problems, and progressive vision loss leading to blindness. ATSN-301 is designed to deliver a functional copy of the MYO7A gene to retinal cells, addressing the underlying cause of USH1B and aiming to prevent or reverse vision loss. The therapy uses a gene augmentation approach, leveraging the dual AAV vector platform to accommodate the large size of the MYO7A gene[1][2][3][5][7].

Brand names

ATSN-301ATSN301ATSN 301

Other names

ATSN-301ATSN301ATSN 301