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Belief BioMed's Collaboration with AskBio

In a remarkable development for the field of gene therapy, Belief BioMed Inc. (BBM) proudly congratulates its partner, AskBio Inc., on the recent acceptance of their Investigational New Drug (IND) application by the United States Food and Drug Administration (FDA) for AB-1009, a groundbreaking gene therapy targeting late-onset Pompe disease. This achievement is a significant milestone not just for AskBio, but also for the collaborative efforts of both companies, reflecting the innovative spirit and dedication within the burgeoning field of gene therapy.

What is AB-1009?

AB-1009 is a novel adeno-associated virus (AAV) gene therapy designed to address the genetic disorder known as Pompe disease. This rare and often debilitating condition is caused by a deficiency in the enzyme acid alpha-glucosidase (GAA), which leads to the accumulation of glycogen in cells. Consequently, individuals affected by Pompe disease typically experience severe muscle weakness and other serious health complications, particularly affecting respiratory function.

As individuals with late-onset Pompe disease may experience progressive muscle weakness and debilitating impacts on daily living, the development of effective treatment options such as AB-1009 is crucial. The IND acceptance means that AskBio can now initiate clinical trials aimed at evaluating the safety of the therapy, with the first patient recruitment anticipated to begin early in 2026.

The Collaboration Between Belief BioMed and AskBio

The partnership between Belief BioMed and AskBio dates back to September 2024, when they entered into a strategic collaboration agreement to further develop innovative gene therapies. Under this partnership, Belief BioMed has provided AskBio with a sublicense to use its proprietary AAV capsid technology in the AB-1009 program, along with crucial AAV vector production services.

Dr. Xiao Xiao, the Co-founder, Chairman, and Chief Science Officer of Belief BioMed, expressed his enthusiasm regarding the IND acceptance, stating, "This milestone signifies not only a pivotal development in our strategic partnership but also highlights the remarkable progress being made in the gene therapy sector. We are thrilled for the AskBio team and look forward to continuing to work together in the quest for innovative treatment solutions."

The Importance of Gene Therapy in Healthcare

The acceptance of the IND application represents not merely a step forward for AskBio but reinforces the potential of gene therapy as a transformative approach in medicine. Gene therapies, particularly those aimed at severe genetic and chronic diseases, have the capacity to change treatment paradigms entirely, offering hope to patients where none existed before. This shift is essential for conditions like Pompe disease, which affects approximately 5,000 to 10,000 people globally.

Looking Ahead

As the collaboration continues, Belief BioMed aims to deepen its partnership with AskBio, supporting the acceleration of innovative therapies that may change the lives of patients battling genetic disorders. With a robust pipeline of gene therapy products and a commitment to harnessing advanced vector technologies, Belief BioMed remains at the forefront of biotech innovation, working to address unmet clinical needs in a variety of therapeutic areas, including hemophilia, Duchenne Muscular Dystrophy (DMD), and Parkinson's disease.

In conclusion, the acceptance of the IND application for AB-1009 signifies a vital advance in the fight against late-onset Pompe disease and paves the way for further development of essential gene therapies. Belief BioMed is proud to stand alongside AskBio in this important journey, championing advancements that hold the promise of better health outcomes for those affected by this challenging condition.