Hemophilia is a rare and serious X-chromosome linked congenital bleeding disorder that affects the blood's ability to clot.
When a person bleeds, 13 different proteins—called clotting factors—work together to form a blood clot. In people living with hemophilia, one of the clotting factors is missing or reduced, which prevents this process from working. They have trouble forming blood clots—so it’s harder to stop bleeds when they happen.
Hemophilia is a lifelong condition that has a significant impact on the lives of individuals and their families. Recent innovations are changing the treatment landscape for hemophilia and other rare bleeding disorders.
People living with hemophilia are typically born with it. In most of these cases, it is passed to a child from the mother's side of the family. Sometimes, a person may develop hemophilia without any family history. In fact, nearly one-third of hemophilia cases are not inherited and have no family history of the disease. Since the genes for coagulation factors VIII and IX are on the X chromosome, hemophilia most commonly affects males who inherit an affected maternal X chromosome.
Due to a lack of bleeding at birth or because the family doesn't have a history of hemophilia, some people who have hemophilia are not diagnosed as newborns.
Since hemophilia and rare bleeding disorders are associated with several types of clotting factors, there are different treatments. Current therapies are administered by infusion therapy injected into a vein or administered subcutaneously.
Clotting factor therapy can be given to combat a bleeding episode that is in progress or it can also be administered at home on a regular schedule to help prevent bleeding episodes. For some people living with hemophilia, they must receive continuous preventative replacement therapy.
By combining our experience in protein design with collaboration in the global scientific community, our scientists are exploring innovative, long acting and subcutaneous treatment solutions for hemophilia and rare blood disorders. These solutions aim to improve clinical outcomes and complement research into oral treatments and gene therapy.
