SHANGHAI, Jan. 15, 2026 /PRNewswire/ — AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, recently announced that the United States Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for AB-1009, an investigational adeno-associated virus (AAV) gene therapy being developed for the treatment of late-onset Pompe disease. With this announcement, the AB-1009 program advances to Phase 1/2, and AskBio initiates a clinical trial in the United States to explore the safety of AB-1009. The company anticipates recruiting its first patient in early 2026 [1].
In September 2024, Belief BioMed (BBM) and AskBio entered astrategic collaboration to explore the potential for new gene therapies [2]. BBM granted AskBio a sublicense to use the proprietary AAV capsid in the AB-1009 program and provided AAV vector production services. Within the collaborative development framework, BBM worked closely with the AskBio team, and its contributions were instrumental to the IND acceptance.
Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, "We are delighted to learn that our partner AskBio has received IND acceptance for its gene therapy. This marks a significant milestone in our strategic collaboration and represents a practical achievement in globalized innovation within the gene therapy field. On behalf of Belief BioMed, I extend our sincere congratulations to the AskBio team! This IND acceptance reflects AskBio’s outstanding R&D capabilities and efficient clinical advancement, while also demonstrating the strong synergy between our technological resources and platforms. Looking ahead, Belief BioMed hopes to deepen our collaboration with AskBio to accelerate the global development of innovative therapies."
About Pompe Disease
Pompe disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time and is accompanied by diaphragmatic involvement, leading to respiratory insufficiency early in the course of the disease. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.
References
1. https://www.askbio.com/
2. https://www.beliefbiomed.com/newsd-749.html
About Belief BioMed
Belief BioMed Inc. (BBM) is a global biotech company that integrates the research and development, manufacturing and clinical application of gene therapy products. The company is committed to providing innovative and more effective gene therapies for severe genetic and chronic diseases through safe and efficient viral vector technology. BBM has developed hundreds of key vector technologies, including HEK293 cell suspension serum-free culture process and full-scale chromatography purification process, and has established a commercial production platform for gene therapy drugs. The company has been building up its capabilities in a variety of fields including novel AAV capsids targeting different tissues, efficient transgene expression cassette design, and advanced clinically applicable vector manufacturing process. It has also established an extensive R&D pipeline covering a wide range of unmet clinical needs in different therapeutic areas such as hemophilia, DMD, Parkinson’s disease, osteoarthritis, etc. Several product pipelines have entered clinical studies or submitted IND filings. The Biologics License Application (BLA) of a gene therapy for the treatment of adult patients with hemophilia B, has been approved by the NMPA of China. For more information, visit www.beliefbiomed.com.
Statement
This information is only for the purpose of introducing the company’s event and information on that date, and is not intended to promote any company’s products and/or services, nor should it be construed as providing any advice or recommendation on the selection of any drugs, medical devices and treatment options.
For information about any company products, diseases and/or treatments, please consult a healthcare professional.
AB-1009 described herein has not been approved for marketing.
