Can a single injection restore sight in a rare childhood blindness?
Cure
Ongoing
This trial tests a gene therapy called ATSN-101, given as a one-time injection under the retina, for people with Leber congenital amaurosis caused by GUCY2D gene mutations. The goal is to see if the treatment is safe and can improve vision. Participants receive the therapy in one…
Phase 1/2 • Sponsor: Atsena Therapeutics Inc. • Aim: Cure
Last updated Aug 05, 2026 00:00 UTC