One-Time gene shot could free wilson patients from daily pills
Disease control
Recruiting now
This early-phase trial tests a single dose of LY-M003, a gene therapy that delivers a working copy of the ATP7B gene to liver cells. The goal is to see if it is safe and can reduce the need for standard Wilson disease medications. The study enrolls 18 adults and children with con…
Phase: EARLY_PHASE1 • Sponsor: Chaohui Yu • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC