One-Time gene shot aims to ease wilson disease burden
Disease control
Recruiting now
This early-phase trial tests a single-dose gene therapy (LY-M003) in 18 adults and children with Wilson disease, a rare genetic condition causing copper buildup. The treatment delivers a working gene to liver cells to help remove excess copper. The main goals are checking safety …
Phase: EARLY_PHASE1 • Sponsor: Chaohui Yu • Aim: Disease control
Last updated May 17, 2026 05:19 UTC