Generation 2.5 investigational LICA antisense medicine
ION547 is an investigational ligand-conjugated (LICA) antisense medicine designed to inhibit the expression of factor XII (FXII). FXII is a clotting factor produced in the liver which plays an important role in the coagulation pathway. Ionis’ studies have shown that inhibiting FXII prevents thrombosis in models of venous, arterial and catheter-induced thrombosis2,3 without increasing bleeding. In humans naturally lacking FXII, and in FXII-knockout mice, there is normal hemostasis without abnormal bleeding1.
Thrombosis is the formation of blood clots inside blood vessels. Blood clots can obstruct blood flow to prevent sufficient oxygen flow to tissues and organs. In addition, clot fragments can break off from the blood clot and travel to occlude other parts of the circulation. Thrombosis is responsible for many heart attacks and strokes and is the leading cause of morbidity and mortality worldwide.
Current antithrombotic treatments include anticoagulants such as warfarin, Factor Xa inhibitors and thrombin inhibitors. Although these drugs are effective at lowering the risk of thrombosis, these drugs can place patients at significant risk of bleeding because they target factors required for normal coagulation.
Clinical Trials Posting