PURE FACTOR IX FOR TREATMENT OF HEMOPHILIA B

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Hemophilia B is a severe bleeding disorder characterized by a functional deficiency in the blood coagulation protein factor IX (Christmas factor). Therapy for bleeding episodes in these patients requires the intravenous replacement of factor IX given as fresh frozen plasma or with commercially available concentrates of factor IX in which the protein is only partially purified. Although these commercial materials have proven efficacy in this disease, they contain many impurities, are often contaminated with virus, and their use is associated with a significant morbidity. The goal of this project is the development of a highly purified form of factor IX. The factor IX will be obtained by using a recently described invention which employs conformation-specific antibodies against human factor IX to purify this protein. This factor IX will be homogeneous and thus suitable for intravenous therapy. Published studies of factor IX purified by this methodology indicate that it is free of contaminants and infectious agents responsible for complications associated with the currently commercially available concentrates. In this Phase I study sufficient factor IX will be purified to accomplish studies of acute toxicity in rabbits and the ability to correct the bleeding deficiency in hemophiliac dogs.