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Until now Hemophilia A and B patients received infusions of the respective clotting factor their bodies didn’t produce. Over one in three patient’s immune systems will recognize the clotting factor as foreign and develop an immune response, resulting in inhibitor formation and complications from the disease. Current gene therapies in development are only for patients with no history of inhibitors and work for either Hemophilia A or Hemophilia B, but not both. This leaves patients with inhibitors no gene therapy treatment option and presents a risk of future inhibitor formation for those patients with no prior history.
Everyone makes activated clotting Factor V (FVa), which is part of a common pathway in the clotting cascade. Our approach utilizes cells in the body to increase FVa production, bypassing missing upstream factors and inhibitors.
With a single infusion, we can teach the body how to make healthy proteins required to impact the disease.
Our first gene therapy program is for hemophilia A and B, with or without inhibitors. Our second gene therapy program is for hemophilia arthropathy.