First human trial launches for Gene-Based MPS i treatment
Disease control
Recruiting now
This early-stage study tests whether ISP-001 is safe and tolerable in 11 people with a rare genetic disease called MPS I (Hurler-Scheie or Scheie types). Participants receive the experimental treatment and are monitored for side effects. The goal is to gather safety data for futu…
Phase: PHASE1 • Sponsor: Immusoft of CA, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:02 UTC