Sangamo is a genomic medicine company that is starting to develop gene therapies for a number of conditions. Sandy Macrae, PhD, Chief Executive Officer of Sangamo talks about the work they are currently doing to develop a gene therapy for hemophilia A.

While great advances in gene therapy for hemophilia has occurred in the past few years, including those by Biomarin and Spark Therapeutics, Dr. Macrae said, “We are close on their heels,” adding that his company has shown some very encouraging results.

Dr. Macrae added that the goal of their therapy is to make sure they are providing a treatment that the patients want. According to Dr. Macrae, patients basically want three things from a therapy – predictability, sustainability, and safety. “All of those things are part of the contract that we have with the patients we serve.”

Hemophilia A is a rare bleeding disorder due to a mutation in the Factor VIII gene. The severity of the condition is largely dependent on the amount of Clotting FVIII the person can produce. Those that can produce 6-49% of normal levels of Clotting Factor VIII will likely have mild symptoms whereas those with 0 – 5% of normal will have more severe forms of the disease.

Common symptoms include excessive bleeding and bruising. The excessive bleeding can be external (cuts that take a long time to clot, nosebleeds, etc) and internal. The latter type of bleeding can be life threatening (e.g., due to bleeding in brain) and/or extremely painful (e.g., due to bleeding in the joints).

Current treatment options for hemophilia A include replacement therapy (on demand and/or prophylactic), desmopressin, and aminocaproic acid.

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