PCSK9 inhibitors have marked a major advance in the treatment of dyslipidemia and cardiovascular prevention, representing a paradigmatic example of translational research “from gene to drug”. From the discovery of PCSK9 and its role in LDL receptor degradation to the development of monoclonal antibodies and siRNA-based therapies such as inclisiran, progress has been linear and biologically driven. Beyond LDL lowering and cardiovascular risk reduction, ancillary effects on platelet function and glucose metabolism have been investigated. Although PCSK9 loss-of-function variants are associated with a modest increase in diabetes risk, experimental and clinical evidence shows that inhibition of circulating or hepatic PCSK9 does not impair pancreatic β-cell function, supporting its metabolic safety.