The 2003 discovery of proprotein convertase subtilisin–kexin type-9 (PCSK9) – a circulating protein targeting the low density lipoprotein (LDL) receptor for its degradation1,2 – paved the way for the development of several therapeutic approaches which inhibit the protein itself (by monoclonal antibodies) or its RNA (by RNA interference “RNAi”).3 These drugs are a very promising and attractive therapy for high-risk cardiovascular (CV) patients, patients with statin intolerance, and those with familial hypercholesterolemia. The gene discussed is PCSK9; the disease is familial hypercholesterolemia.