Gene therapy could free gaucher patients from lifelong infusions
Disease control
Recruiting now
This study tests a one-time gene therapy called FLT201 for adults with Gaucher disease type 1. Participants currently on regular enzyme replacement therapy will stop that treatment after receiving FLT201. The goal is to see if the gene therapy can keep blood counts stable and con…
Phase: PHASE3 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated May 07, 2026 18:40 UTC