New hope for rare disease: first human test of FLT201 begins
Disease control
Completed
This early-stage study tested a new drug, FLT201, in 6 adults with Gaucher disease type 1. The main goal was to check safety and how well the body tolerates the drug. Researchers also looked at whether FLT201 can boost the missing enzyme and reduce harmful buildup in cells.
Phase: PHASE1 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated May 12, 2026 13:42 UTC