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PCSK9-gene-silencing, cholesterol-lowering drug impresses

Nature Biotechnology volume 37, pages 1385–1387 (2019)Cite this article

The Medicines Company’s partnered drug uses RNA interference to tackle stubbornly high LDL cholesterol.

If it succeeds, inclisiran will join two approved monoclonal antibody (mAb) drugs that target the same molecule, PCSK9 (proprotein convertase subtilisin/kexin type 9), in the liver (Table 1). Inclisiran, however, is an N-acetylgalactosamine-conjugated 2′-O-methyl, 2′-fluoro, 2′-H-phosphorothioate siRNA-based oligonucleotide that targets PCSK9 mRNA translation. All these drugs dramatically decrease serum levels of low-density-lipoprotein cholesterol (LDL-C), a major component of arterial plaques, which narrow or block arteries. But inclisiran has the advantages of cheap manufacture and easy long-term storage over the two already approved mAb-based PCSK9 inhibitors, Repatha (evolocumab; Amgen) and Praluent (alirocumab; Sanofi, Regeneron Pharmaceuticals). For physicians, inclisiran is certainly more convenient, requiring fewer injections.

Table 1 Selected PCSK9 inhibitors
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    Cormac Sheridan

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Sheridan, C. PCSK9-gene-silencing, cholesterol-lowering drug impresses. Nat Biotechnol 37, 1385–1387 (2019). https://doi.org/10.1038/s41587-019-0351-4

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