BEDFORD, Mass., and SHANGHAI, China, July 12, 2023 – An application for an investigational study in human patients with FT-004, an innovative gene therapy drug independently developed by Frontera Biotechnology (Suzhou) Co., Ltd. (hereinafter referred to as “Frontera Biotechnology”), was approved by the Center for Drug Evaluation (CDE) of China National Medical Products Administration for the indication of hemophilia B (endogenous FIX activity ≤ 2%). This is the third innovative AAV gene therapy product approved for investigational clinical studies by Frontera Biotech.
FT-004 Injection is a novel recombinant adeno-associated virus vector gene therapy drug. Nonclinical data showed that after FT-004 injection into model animals, hepatocytes could be efficiently transfected to continuously and stably express and secrete active hFIX (human coagulation factor 9) protein, improve the coagulation function of model animals, with a good safety profile. For hemophiliacs, patients are expected to receive one injection for long-term efficacy.
About hemophilia B
Hemophilia B (HB) is an X chromosome-linked recessive hemorrhagic disease. It is mainly caused by mutations in the factor 9 (F IX) gene leading to F IX deficiency or dysfunction, affecting the production of normal prothrombin activator, leading to coagulation dysfunction and bleeding. Hemorrhage is mainly manifested in joints, muscles and deep tissues, but also in the gastrointestinal tract, central nervous system and other visceral organs. Repeated bleeding, if untreated, can lead to joint deformity and/or pseudotumor formation, seriously impacting the quality of life of patients, and can be life-threatening. In the male population, the incidence of hemophilia B is 1 in 25,000.