Gene therapy trial aims to save sight in boys and men with rare retinal disease
Disease control
Recruiting now
This study tests a gene therapy called ATSN-201 in males aged 6 and older with X-linked retinoschisis, a genetic eye condition that leads to vision loss. The therapy delivers a working copy of the RS1 gene to the retina. The trial has three parts: first, finding the right dose; s…
Phase 3 • Sponsor: Atsena Therapeutics Inc. • Aim: Disease control
Last updated Sep 10, 2026 03:00 UTC