Alnylam Pharmaceuticals has three products in their pipeline, which target transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ALN-TTR02), vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP) for hepatocellular carcinoma (ALN-VSP), and proprotein convertase subtilisin/kexin type 9 (PCSK9) for hypercholesterolemia (ALN-PCS). The gene discussed is PCSK9; the disease is familial hypercholesterolemia.