Gene therapy hope for pompe disease patients in early safety trial
Disease control
Completed
This early-stage study tested the safety of a new gene therapy called ACTUS-101 in 7 adults with late-onset Pompe disease. The treatment aims to help the body produce an enzyme it lacks, potentially reducing the need for regular infusions. The study focused on side effects and la…
Phase: PHASE1 • Sponsor: AskBio Inc • Aim: Disease control
Last updated May 15, 2026 11:56 UTC