A siRNA-targeting PCSK9, inclisiran, is already approved by both FDA and EMA and is in clinical use in the USA and Europe for lowering LDL-C levels in patients with (i) primary hypercholesterolemia (familial or not) or mixed dyslipidemia in combination with a statin or with a statin and another lipid-lowering agent in patients that are not at their LDL-C target or (ii) alone or in combination with other lipid-lowering drugs (without a statin) in patients with statin intolerance or in those where a statin is contraindicated. The gene discussed is PCSK9; the disease is familial hypercholesterolemia.