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ultragenyx

Hemophilia A is a genetic disorder where blood does not clot normally due to low levels of clotting factor VIII (FVIII). More than half of hemophilia A patients have a severe form of the disease, characterized by heavy bleeding after minor injury and spontaneous internal bleeding that affects muscles, joints, and organs such as the kidneys, GI tract, and brain. Current treatment manages the disease with recombinant factor VIII infusions multiple times a week for life. GTP is supporting Dimension’s development of an AAV-mediated FVIII gene therapy (DTX201) to achieve sustained FVIII expression with the goal of reducing or discontinuing replacement therapy infusions for an extended period of time.