Hemgenix Approved as First Gene Therapy for Hemophilia B

Hemgenix Approved as First Gene Therapy for Hemophilia B

Hemgenix is approved for patients who currently use factor IX prophylaxis therapy or have serious hemorrhage or spontaneous bleeding episodes. The adeno-associated virus vector-based gene therapy delivers a functional copy of Factor IX, a protein involved in blood clotting that is missing or not made in enough amounts in patients who have the disease due to mutations in the gene that codes for it. Hemophilia B primarily affects men.

The prevalence of Hemophilia B in the population is around one in 40,000 and 15 percent of patients with hemophilia have the B type. Many women carriers of the disease have no symptoms while 10 to 25 percent have mild symptoms and in rare cases, women may have moderate or severe symptoms.

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