Hemophilia

AMA005 is a gene replacement therapy product for the treatment of patients with hemophilia B (HEMB). Hemophilia A (HEMA) and HEMB are rare X-linked genetic disorders where mutations in the genes encoding blood clotting factor VIII (FVIII) and IX (FIX), respectively, lead to impaired blood clotting. In hemophilia patients even a minor injury can result in severe and life-threatening blood loss. Currently, there is no cure for hemophilia and patients need regular infusions of the missing clotting factor. Gene replacement therapy using AAV vectors in which a gene encoding the missing clotting factor is introduced into the liver of patients cells has been shown to be a highly promising alternative to protein replacement therapy. In AMA005 we intend to restore the blood clotting process in HEMB patients using an SVec vector encoding human FIX.