Rare Blood Disorders

Hemophilia is a type of rare blood disorder caused by an inherited somatic mutation in the gene for the clotting factor protein needed to form a blood clot. These mutations may prevent the clotting protein from working properly or may be missing altogether. Hemophilia A and hemophilia B are characterized by a deficiency in FVIII and FIX, respectively. They are the most common forms of the disease as they are both linked to the X chromosome and thus only require one defective gene in males for the disease to manifest.

The severe or moderate hemophilia A patient population is estimated to be approximately 10,000 patients in the United States and approximately 95,000 worldwide. Most of these patients are treated with prophylactic recombinant FVIII intravenous infusion several times per week as well as additional infusions for bleeding episodes.

Based on our Shielded Living Therapeutics™ platform, we developed SIG-001 as a potential novel treatment for patients with hemophilia A, with the goal of consistently delivering blood clotting factors to help prevent bleeding episodes.