On January 8, 2026, AskBio Inc., a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that the United States Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for AB-1009. This investigational adeno-associated virus (AAV) gene therapy is designed for the treatment of late-onset Pompe disease (LOPD), a rare, progressive genetic metabolic disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent GAA levels lead to glycogen accumulation in muscles and tissues, resulting in progressive skeletal muscle weakness, respiratory insufficiency, and significant morbidity; without early intervention, the disease can lead to premature death.
The IND acceptance marks a key advancement, transitioning the AB-1009 program to Phase 1/Phase 2. AskBio has initiated a clinical trial in the United States (PROGRESS-GT LOPD, NCT07282847), a single-arm, open-label, dose-escalation study evaluating the safety, tolerability, and preliminary efficacy of a one-time intravenous infusion of AB-1009. The company anticipates enrolling its first patient in early 2026. This follows recent FDA grants of Fast Track and Orphan Drug designations for AB-1009, which provide accelerated development pathways and potential market exclusivity incentives for therapies addressing unmet needs in rare diseases.
AB-1009 aims to deliver a functional copy of the GAA gene to enable increased production of the deficient enzyme, potentially offering a more durable treatment compared to current enzyme replacement therapy (ERT). The development builds on collaborations with Belief BioMed Inc., Genethon, and Duke University. Notably, AskBio’s earlier LOPD trial with ACTUS-101 (NCT03533673) will continue with existing participants but cease new recruitment as focus shifts to AB-1009.
As an investigational therapy, AB-1009 has not been approved by any regulatory authority, and its efficacy and safety remain under evaluation. This progress underscores AskBio’s commitment to advancing novel AAV-based gene therapies for rare neuromuscular and metabolic conditions, leveraging Bayer’s support to address critical unmet needs in the Pompe disease community.
