Inclisiran is the first siRNA approved by both US FDA and EMA for the treatment of hypercholesterolemia; the subcutaneous injection of a lipid nanoparticle formulation of the drug, which specifically binds hepatocyte asialoglycoprotein receptors, displays high potency in lowering liver PCSK9 and plasma LDL-C levels [161,162]. This evidence concerns the gene PCSK9 and familial hypercholesterolemia.