One-Time gene therapy could change gaucher treatment forever
Disease control
Recruiting now
This study tests a new gene therapy called LY-M001 for adults with Gaucher disease type 1, a genetic disorder that causes organ damage and other health problems. The therapy uses a harmless virus to deliver a working copy of the GBA1 gene to liver cells, aiming to restore the mis…
Phase: PHASE1, PHASE2 • Sponsor: Lingyi Biotech Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC