Gene therapy hope for pompe patients in early safety trial
Disease control
Completed
This early-phase study tested a new gene therapy called ACTUS-101 in 7 adults with late-onset Pompe disease. The main goal was to check safety and side effects. Participants received a single intravenous dose, and researchers monitored their health over time.
Phase: PHASE1 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC