First patients receive experimental gene therapy injection for rare inherited blindness
Disease control
Ongoing
This is the first study in people to test the safety of an experimental drug called PYC-001 for a rare, inherited eye disease that causes progressive vision loss. Up to 18 adults with a confirmed OPA1 gene mutation will receive a single injection of the drug into one eye. The mai…
Phase: PHASE1 • Sponsor: PYC Therapeutics • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC