FDA Clears IND for AB-1009: Advancing AAV Gene Therapy to Phase 1/2 in LOPD

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.