Bayer AG's AskBio Advances Gene Therapy for Pompe Disease with FDA IND Approval

Bayer AG Advances Gene Therapy for Pompe Disease with IND Acceptance

Bayer AG's subsidiary, AskBio Inc., marks a significant achievement in the realm of gene therapy with the recent acceptance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for AB-1009. This groundbreaking adeno-associated virus (AAV) gene therapy is specifically designed to target late-onset Pompe disease, a debilitating lysosomal storage disorder. The FDA's acceptance propels the AB-1009 program into Phase 1/2 clinical trials, with plans to recruit the first patient by early 2026. This milestone not only highlights AskBio's commitment to advancing genetic medicine but also reflects Bayer's strategic focus on addressing unmet medical needs in rare diseases.

Pompe disease is caused by a deficiency in the enzyme acid alpha-glucosidase (GAA), leading to harmful glycogen accumulation within cells. This condition manifests in two forms: the infantile variant, which is rapidly fatal and can severely impact heart function, and the late-onset variant that progressively affects skeletal muscle strength and respiratory health. With an estimated global prevalence of 5,000 to 10,000 cases, the need for effective therapies is urgent. AskBio's progress in developing AB-1009 is a promising step toward providing patients with a viable treatment option that could significantly improve their quality of life.

The strategic collaboration with Belief BioMed (BBM), established in September 2024, plays a crucial role in the development of AB-1009. BBM has granted AskBio a sublicense for a proprietary AAV capsid, along with essential AAV vector production services. Dr. Xiao Xiao, Co-founder and Chief Science Officer at BBM, emphasizes the collaborative effort's success in propelling gene therapy advancements, showcasing a strong synergy between the two companies. This partnership illustrates Bayer's dedication to leveraging innovative technologies to combat rare diseases like Pompe, further solidifying its position in the gene therapy landscape.

In parallel to this development, AskBio continues to enhance its research capabilities, aiming to explore additional applications for gene therapies beyond Pompe disease. The company’s focus on innovative treatments reinforces Bayer’s commitment to leading advancements in the biopharmaceutical sector. As clinical trials for AB-1009 commence, stakeholders remain hopeful that this gene therapy will pave the way for breakthroughs in treating genetic disorders.