ARO-C3

ARO-C3 is an investigational small interfering RNA (siRNA) therapeutic developed by Arrowhead Pharmaceuticals. It is designed to reduce the production of complement component 3 (C3) in hepatocytes via RNA interference, thereby inhibiting the activation of the complement cascade. This mechanism targets early steps in complement-mediated diseases, aiming to prevent tissue injury and disease progression associated with dysregulated complement activity. The drug is being evaluated primarily for various complement-mediated renal diseases, including IgA nephropathy, membranoproliferative glomerulonephritis, and paroxysmal nocturnal hemoglobinuria. Clinical studies have shown that ARO-C3 achieves deep and sustained reductions in C3 levels and alternative pathway activity markers with infrequent subcutaneous dosing[1][2][5][7].