May 11, 2026 —
AskBio, a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that the first participant has been dosed in PROGRESS-GT LOPD, a Phase 1/2 clinical trial evaluating AB-1009, an investigational AAV-based gene therapy for late-onset Pompe disease.
Pompe disease is a rare inherited lysosomal storage disorder caused by deficiency of acid alpha-glucosidase (GAA), an enzyme required to break down glycogen. Reduced or absent GAA activity leads to glycogen accumulation in cells, contributing to progressive muscle weakness, respiratory impairment, and other disease manifestations. Late-onset Pompe disease primarily affects skeletal muscle and can cause worsening mobility and breathing difficulties over time.
Although multiple enzyme replacement therapies using recombinant human GAA are approved for Pompe disease, significant unmet need remains. Some patients may experience reduced clinical response over time, and lifelong ERT can create a substantial treatment burden. AB-1009 is designed to address the underlying genetic defect by enabling sustained production of GAA, potentially offering a different therapeutic approach for patients with LOPD.
The PROGRESS-GT LOPD trial is a non-randomized, single-arm, open-label, dose-escalation study evaluating the safety, tolerability, and efficacy of AB-1009 in adult participants with LOPD. AskBio initiated recruitment in the United States earlier in 2026, and the study is expected to enroll approximately 12 participants across U.S. sites.
AB-1009 has received FDA Fast Track and Orphan Drug designations, and the FDA accepted the investigational new drug application for the program earlier this year. These regulatory milestones may support more frequent FDA interactions and potential expedited development pathways if future clinical data support continued advancement.
The first patient dosing marks an important step for AskBio’s Pompe disease program and reflects continued momentum in applying AAV gene therapy to lysosomal storage disorders. While AB-1009 remains investigational and its safety and efficacy have not yet been established, the program represents a potentially important effort to move beyond chronic enzyme replacement toward durable genetic approaches for rare neuromuscular diseases.
