Hemophilia is a genetic disorder that affects the body's ability to form blood clots. It is caused by a deficiency or dysfunction of one of the clotting factors, which are proteins in the blood that help to stop bleeding. There are two main types of hemophilia: Hemophilia A, caused by a deficiency of clotting factor VIII, and Hemophilia B, caused by a deficiency of clotting factor IX.
Hemophilia Treatment is a one-time gene therapy product administered via IV infusion. Hemgenix is made up of a viral vector that contains a gene for clotting factor IX. The gene is expressed in the liver in order to produce Factor IX protein, which raises blood levels of Factor IX and thus limits bleeding episodes.
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