Emerging and future therapies for hemophilia

Marcus E Carr,1,2 Bartholomew J Tortella3,4 1Robert Wood Johnson Medical School, Rutgers University, New Brunswick, NJ, 2Worldwide Research and Development, Pfizer, Inc., Cambridge, MA, 3Drexel University College of Medicine, Philadelphia, PA, 4Global Innovative Pharma, Pfizer, Inc., Collegeville, PA, USA Abstract: The evolution of care in hemophilia is a remarkable story. Over the last 60 years, advances in protein purification, protein chemistry, donor screening, viral inactivation, gene sequencing, gene cloning, and recombinant protein production have dramatically enhanced the treatment and lives of patients with hemophilia. Recent efforts have produced enhanced half-life (EHL) clotting factors to better support prophylaxis and decrease the frequency of infusions. Medical needs remain in the areas of alternate modes of administration to decrease the need for venous access, better treatment, and prophylaxis for patients who form antibodies to clotting factors, and ultimately a cure of the underlying genetic defect. In this brief review, the authors summarize data on EHL clotting factors, introduce agents whose mode of action is not clotting factor replacement, and list current gene therapy efforts. Keywords: extended half-life clotting factors, gene therapy, anti-tissue factor pathway inhibitor antibodies, clotting factor analogs, nonfactor replacement therap