Pharmaceutical firm uniQure N.V. has developed an optimized large-scale process to manufacture its lead gene, therapydate, to treat hemophilia B. This new platform will allow the Dutch company to boost production of its AMT-060 gene therapy at its Lexington, Massachusetts, facility, in accordance with Good Manufacturing Practices guidelines.
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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…
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CSL Behring’s Afstyla Allows Hemophilia A Patients to Use Less Factor Replacement, Analysis Shows
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…
The latest results of CSL Behring’s recombinant coagulation factor IX (FIX) Idelvion, also known by its generic name CSL654, demonstrated long-lasting therapeutic potential for both pediatric and adult patients with hemophilia B. The findings were detailed in poster presentations at the 26th Biennial…
Bayer has awarded a combined $2 million in hemophilia research and patient-care grants to 16 scientists and doctors from eight countries. Four of the winners are from the United States. Three represent the Children’s Hospital of Philadelphia and one the Uniformed Services University of the Health Sciences in…
Treatment with Eloctate and Alprolix improves joint health, bleeding episodes and quality of life of patients with hemophilia, Bioverativ and Sweden’s Sobi recently announced. The data is being presented at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress,  through July 13 in Berlin, Germany. Hemophilia is a…
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…
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