ATSN-101

ATSN-101 is an investigational gene therapy designed to treat Leber congenital amaurosis type 1 (LCA1), a rare inherited retinal disease caused by mutations in the GUCY2D gene. The therapy uses an engineered adeno-associated virus serotype 5 (AAV5) vector to deliver functional copies of the human GUCY2D gene directly to photoreceptor cells in the retina via subretinal injection. By introducing healthy GUCY2D, ATSN-101 aims to restore guanylate cyclase activity and improve visual function in patients with LCA1. Clinical trials have shown that high-dose ATSN-101 leads to durable and clinically meaningful improvements in vision with a favorable safety profile[1][3][7]. The drug was originally developed at the University of Florida and has received Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT), and Rare Pediatric Disease designations from regulatory agencies for its potential as a first-in-class treatment for this condition[5][7].

Brand names

ATSN-101ATSN101ATSN 101

Other names

AAV5-GUCY2DAAV-5-GUCY2DAAV 5-GUCY2D