Bayer AG's AskBio Receives IND for Gene Therapy AB-1009 Targeting Pompe Disease

Bayer AG Advances Gene Therapy for Pompe Disease with IND Acceptance

Bayer AG's wholly owned subsidiary, AskBio Inc., celebrates a pivotal achievement with the acceptance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for AB-1009, a gene therapy aimed at treating late-onset Pompe disease. This acceptance marks a significant step forward, allowing the AB-1009 program to progress into Phase 1/2 clinical trials. The recruitment of the first patient is anticipated to commence in early 2026, a timeline that holds promise for those affected by this rare genetic disorder. The approval represents not just a regulatory milestone but also a testament to Bayer's commitment to advancing innovative medical solutions in the genetic therapy landscape.

Pompe disease, a lysosomal storage disorder characterized by the deficiency of the enzyme acid alpha-glucosidase (GAA), leads to severe health complications including muscle weakness and respiratory failure. The condition manifests in two forms: the rapidly fatal infantile variant that affects cardiac function and the more slowly progressing late-onset variant that primarily impacts skeletal muscles. With an estimated global patient population of 5,000 to 10,000 individuals, the need for effective treatments is critical. The IND acceptance of AB-1009 is a significant step in addressing this unmet medical need and showcases Bayer's dedication to pioneering treatments that can transform the lives of patients suffering from such debilitating conditions.

The strategic collaboration between AskBio and Belief BioMed (BBM), initiated in September 2024, further enhances the development of AB-1009. BBM has granted AskBio a sublicense to utilize its proprietary AAV capsid technology, as well as vector production services, which are integral to the development of the gene therapy. Dr. Xiao Xiao, BBM’s Co-founder and Chief Science Officer, expresses excitement over the collaboration, emphasizing how it leverages both companies' technological strengths to push forward the gene therapy agenda. This partnership not only accelerates the progress of the AB-1009 program but also underlines the synergy between the two firms in the quest to deliver innovative therapies to patients in need.

In summary, Bayer AG's commitment to advancing gene therapies is exemplified by AskBio's recent IND acceptance for AB-1009. As the company prepares to enter clinical trials, the collaboration with BBM highlights the importance of strategic partnerships in driving innovation within the industry. This development is a positive indicator for the future of gene therapy in addressing rare diseases like Pompe disease, paving the way for potential breakthroughs in treatment.