A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).

NCT Number	NCT07282847
Lead Sponsor	AskBio Inc Collaborators: Bayer
Conditions	Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Enrollment	12 participants
Start Date	2026-04-15
Primary Completion	2028-09 (estimated)
Study Completion	2032-09 (estimated)
Updated on ClinicalTrials.gov	2026-07-20