Gene therapy trial aims to fix enzyme defect in gaucher disease
Disease control
Completed
This early-stage trial tested a new gene therapy called FLT201 in 10 adults with Gaucher disease type 1. The therapy uses a harmless virus to deliver a working copy of the gene that produces a missing enzyme. The main goal was to check safety and see if the treatment can boost en…
Phase: PHASE1 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC