https://www.youtube.com/watch?v=pTIVORH7anc This video from Wochit News is all about a new treatment for severe hemophilia A. The Children’s Hospital of Los Angeles, California, has developed a breakthrough treatment that can reduce the bleeding episodes in people living with hemophilia A by up to 90 percent. MORE: Emicizumab shows…

Three patients who received SPK-8011 — a gene therapy developed by Spark Therapeutics — for hemophilia A show increased production of clotting factor VIII without any safety issues reported, says the company. The patients are the first to receive the treatment as part of a Phase 1/2 clinical trial, with…

BioMarin Pharmaceutical  will add an additional Phase 3 study to the clinical development of its investigational gene therapy BMN 270 for hemophilia A. BioMarin will conduct two separate trials testing the effectiveness and safety of two different doses of BMN 270. Both trials are expected to initiate in the…

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…