Experimental gene therapy aims to restore sight in rare childhood blindness
Disease control
Recruiting now
This study tests a new gene therapy called ATSN-201 for people with X-linked retinoschisis, a rare genetic eye disease that causes vision loss. The trial includes children and adults aged 6 and older, and will check if the treatment is safe and can improve vision. The therapy wor…
Phase: PHASE3 • Sponsor: Atsena Therapeutics Inc. • Aim: Disease control
Last updated May 02, 2026 06:34 UTC