Gene therapy may free gaucher patients from lifelong infusions
Disease control
Recruiting now
This Phase 3 trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. The goal is to see if a single dose can keep blood counts stable so patients can stop their regular enzyme replacement or substrate reduction therapy. The study will enroll 45 people who…
Phase: PHASE3 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC