One-Time gene shot aims to tame rare enzyme disorder
Disease control
Recruiting now
This early-phase trial tests a single injection of gene therapy (LY-M001) in 12 adults with Gaucher disease type 1, a genetic condition causing enzyme deficiency and organ damage. The therapy delivers a working gene to the liver to produce the missing enzyme. The main goals are s…
Phase: PHASE1, PHASE2 • Sponsor: Lingyi Biotech Co., Ltd. • Aim: Disease control
Last updated May 14, 2026 12:04 UTC