Company

Scientific Leadership of Expression Therapeutics

Our Mission

Our mission at Expression Therapeutics is to develop novel therapies that will dramatically improve the lives of people with hemophilia A around the world.

Overview

Expression Therapeutics is a privately owned biotechnology company founded in 2005 and based in Atlanta, Georgia. The focus of our work is 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. About half of all persons with hemophilia are considered severe and have less than 1 percent of the normal factor VIII activity in their blood. People with hemophilia A can have life threatening bleeding episodes. Existing treatments for hemophilia A involve intravenous infusion of factor VIII --- either “on demand” to stop emergent bleeding or on a prophylactic basis prior to injury --- 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. Even those persons with hemophilia that have access to replacement factor VIII products experience decreased quality of life due to the need to take frequent intravenous infusions (typically, every 2 to 4 days) and the development of immune responses against the factor VIII product itself. Currently, there is no cure for hemophilia A.

Our work promises to dramatically change the treatment for hemophilia A. 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.