The genome-wide scale association analysis that scanned the entire genome without bias provided an unprecedented opportunity for drug repurposing by linking disease indications with druggable genes, i.e., “genetics-driven genomic drug discovery” [22, 38, 39], which is exemplified by the identification of PCSK9 for the treatment of hypercholesterolemia [40]. The gene discussed is PCSK9; the disease is familial hypercholesterolemia.