04 January 2024 | Thursday | News
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BBM-H803 is Belief BioMed's first gene therapy for the treatment of hemophilia A and is the company's second product which has obtained IND approval from the National Medical Products Administration (NMPA). In December 2022, BBM-H803 was granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). As of today, there have not been adeno-associated virus (AAV)-based gene therapies available in China for the treatment of adults with hemophilia A.
This registrational study is a multi-center, single-arm, open-label and single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of a single-time intravenous administration of BBM-H803 in patients ≥18 years of age with severe hemophilia A. With an in-house developed engineered capsid, the product features relatively lower immunogenicity and higher liver delivery efficiency. The proposed clinical dose is also lower than that of similar AAV-based gene therapy products launched abroad, and the safety is expected to be better. Patient enrollment for the previous exploratory study was completed in August 2023, and from which data generated has been used to support subsequent registrational trials.
Dr. Xiao Xiao, Co-founder, Chairman, and Chief Science Officer of Belief BioMed, said, "As we welcome in the New Year with its sense of hope and new beginnings, it gives us great pleasure to share that today is a special day for both Belief BioMed and hemophilia A patients, as the first patient dosing has been successfully completed in the registrational trial of BBM-H803. The achievement of this milestone means that Belief BioMed has made another major breakthrough in the field of gene therapy in hematological therapeutical area, and it once again confirms the company's strong and robust research and development capabilities. We will continue to pursue our original goals, working together - both internally and externally - to comprehensively and efficiently conduct this clinical study, all while striving to bring this innovative gene therapy to the vast number of Chinese patients that need it as soon as possible."