AskBio Advances AAV Gene Therapy AB-1009 Into Phase 1/2 Clinical Trial for Late-Onset Pompe Disease

DURHAM, N.C. — January 8, 2026— AskBio Inc., a gene therapy company wholly owned by Bayer AG, announced that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for AB-1009, an adeno-associated virus (AAV) gene therapy for the treatment of late-onset Pompe disease (LOPD), enabling advancement into a Phase 1/Phase 2 clinical trial in the United States.

AB-1009 is an AAV-based gene therapy designed to address the underlying genetic cause of Pompe disease by enabling sustained production of acid alpha-glucosidase (GAA), the enzyme deficient in affected patients. Following IND clearance, AskBio has initiated the PROGRESS-GT LOPD clinical trial and expects to enroll the first patient in early 2026.

The AAV gene therapy has also received FDA Fast Track and Orphan Drug designations, underscoring the unmet medical need in LOPD and supporting expedited development and regulatory review. These designations provide development incentives for AAV gene therapies targeting rare, serious genetic disorders, including enhanced FDA interaction and potential eligibility for accelerated approval pathways.

Pompe disease is a rare, progressive metabolic disorder that can lead to severe muscle weakness, respiratory failure, and premature death. While enzyme replacement therapies exist, they require lifelong administration and may lose efficacy over time, highlighting the need for durable AAV gene therapy alternatives capable of long-term enzyme expression.

The advancement of AB-1009 AAV gene therapy was achieved through collaboration with Belief BioMed, Genethon, and Duke University. AB-1009 remains investigational, and its safety and efficacy have not yet been established.