SPC5001

SPC5001 is a locked nucleic acid (LNA)-modified antisense oligonucleotide (ASO) designed to target and inhibit the mRNA of proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein that regulates low-density lipoprotein cholesterol (LDL-C) levels in the blood. By inhibiting PCSK9, SPC5001 increases LDL receptor expression on hepatocytes, thereby enhancing clearance of LDL-C from the bloodstream. The drug was developed for the treatment of hypercholesterolemia and familial hypercholesterolemia. In clinical studies, SPC5001 demonstrated dose-dependent reductions in PCSK9 protein levels and LDL-C concentrations but was associated with mild to moderate injection site reactions and renal tubular toxicity at higher doses. Due to these safety concerns, clinical development of SPC5001 was terminated[3][5][6].

Other names

SPC5001 sodiumSPC-5001 sodiumSPC 5001 sodium