AskBio has dosed the first participant in the Phase 1/2 PROGRESS-GT LOPD trial of AB-1009 for late-onset Pompe disease. The clinical trial evaluates an AAV-based gene therapy approach in adults. It takes place within the current treatment landscape, where Enzyme Replacement Therapy (ERT) remains the established standard. For global Pompe research and advocacy, this milestone matters because safety, tolerability, early efficacy data and Patient-Reported Outcomes need careful evaluation before expectations rise. AB-1009 is investigational, not approved; its benefits and risks are not yet established. PROGRESS-GT LOPD (NCT07282847) is estimated to enroll 12 participants across the United States.
Source: Press release