Gene therapy trial tests new approach for rare genetic disorder
Disease control
Completed
This first-in-human study tested the safety and early effects of a new gene therapy called FLT201 in adults with Gaucher disease type 1. The therapy aims to boost a missing enzyme to help the body break down harmful substances that build up in cells. Six adults who were already o…
Phase: PHASE1 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Mar 19, 2026 14:56 UTC