One-Shot gene therapy trial offers new hope for gaucher disease
Disease control
Recruiting now
This early-stage study is testing a new, one-time gene therapy called LY-M001 for adults with Gaucher Disease Type 1. The goal is to see if a single injection is safe and can help the liver produce a crucial missing enzyme for a long time, which could reduce harmful substance bui…
Phase: PHASE1, PHASE2 • Sponsor: Lingyi Biotech Co., Ltd. • Aim: Disease control
Last updated Mar 18, 2026 14:40 UTC