The European Commission will award the Brussels-based HemAcure Consortium €5.6 million in next-stage funding of a therapy called Cell Pouch —  developed in collaboration with Canada’s Sernova — for hemophilia A patients. The therapy delivers corrected factor VIII with the help of cells that sit in a transplanted pouch under the skin. The cell pouch…

Chugai Pharmaceutical filed a New Drug Application for the use of emicizumab as a potential treatment to prevent or decrease the occurrence of bleeding episodes in patients with hemophilia A with factor VIII inhibitors. The application is based on…

Once-monthly injection of the investigational RNA therapy Fitusiran reduced the levels of the protein antithrombin, a natural inhibitor of several components of the clotting system, and restored the balance of clotting elements production in adult patients with hemophilia A or B. These findings were featured in the article “Targeting…

Shire has acquired worldwide rights to the antibody-based therapy that Novimmune developed as a treatment for hemophilia A. Geneva-based Novimmune creates therapies for inflammatory diseases, autoimmune diseases and cancer. It uses antibodies that target tumor-associated proteins and the immune checkpoint protein CD47. Shire, which is based in Dublin, is a global…

Hemophilia A patients treated with CSL Behring’s Afstyla (recombinant factor 8 single-chain) require less factor to control bleeding compared to those taking octocog alfa, the company recently announced. The lower consumption holds both for patients using clotting factors on-demand and those who require preventive, or prophylactic treatment. CSL Behring…

Shire has asked the U.S. Food and Drug Administration to grant Investigational New Drug status to its hemophilia A treatment SHP654. The gene therapy helps generate a clotting protein known as factor VIII that is missing or defective in hemophilia A. Shire said an FDA approval of its request will…

Results from two Phase 3 trials support the use of emicizumab for the preventive treatment of hemophilia A in children, adolescents, and adults, Genentech announced. These results will be presented at the 26th International Society on Thrombosis and Haemostasis (ISTH) Meeting July 8-13 in Berlin, Germany. Emicizumab is an…

Shire is developing a potential new strategy to improve the effectiveness of genetic delivery of clotting factor VIII (FVIII) for the treatment of patients with hemophilia A, and clotting factor IX (FIX) for patients with hemophilia B. The most recent findings on these new therapeutic strategies and the latest…

The U.S. Food and Drug Administration (FDA) has accepted Bioverativ’s investigational new drug application (IND) for BIVV001, an investigational Factor VIII therapy designed to extend protection from bleeds in hemophilia A patients. The FDA will now review the IND application for safety. The Waltham, Massachusetts-based company plans to initiate a Phase…

The European Medicines Agency (EMA) has granted orphan medicinal product status to SB-525, a clinical stage gene therapy candidate under development for the treatment of hemophilia A, according to Sangamo Therapeutics and Pfizer. The announcement coincides with the opening of patient enrollment for a Phase 1/2…