AAV Gene Therapy Shows Promise for LCA1 Caused by GUCY2D Mutations

Sep 10, 2024, 07:31 AM

Recent advancements in AAV gene therapy have shown promising results for patients with Leber congenital amaurosis 1 (LCA1), a rare inherited eye disorder caused by mutations in the GUCY2D gene. AAV gene therapy has demonstrated dramatic improvements in vision for affected patients, with benefits sustained for over a year. The safety and efficacy of this therapy, specifically ATSN-101, were evaluated in a phase 1/2, multicenter, open-label, unilateral dose escalation study. Atsena Therapeutics is at the forefront of this research, highlighting the potential of gene therapy in treating inherited blindness.