Expression Therapeutics is focused on the treatment of hemophilia A. Hemophilia A is a recessive, genetically inherited bleeding disorder that is caused by the deficiency of blood clotting factor VIII. Hemophilia A occurs in approximately 1 in 10,000 males worldwide. Existing treatments for hemophilia A involve intravenous infusion of factor VIII to temporarily raise the level of factor VIII in the blood, thereby permitting a more effective clotting process. However, replacement factor VIII products can be expensive: prophylactic treatment schedule for a severe case of hemophilia in the United States can be in excess of $300,000 per year. Consequently, some 70 percent of persons with hemophilia worldwide do not have access to any form of factor VIII therapy. Those who do not receive pharmaceutical intervention typically have mortality rates approaching 100 percent by young adulthood. Expression Therapeutics has developed a novel, patented gene therapy technology that has the potential to cure hemophilia A for the lifetime of the patient. Expression Therapeutics has also developed a high expression factor VIII technology that substantially decreases the cost of manufacturing factor VIII, thereby putting factor VIII within the reach of more people around the world.
