One-Shot gene therapy tested for rare copper disease
Disease control
Recruiting now
This is an early-stage study testing a single-dose gene therapy injection called LY-M003 for Wilson disease. The goal is to see if it is safe and if it can help the body properly process copper, potentially reducing the need for lifelong medications. The study will include a smal…
Phase: EARLY_PHASE1 • Sponsor: Chaohui Yu • Aim: Disease control
Last updated Mar 23, 2026 15:17 UTC