Molecular Medicine Israel

Interfering with bad cholesterol

Over the past few decades, the number of deaths from cardiovascular disease has declined substantially in developed countries. Statins, drugs that lower serum levels of low-density lipoprotein cholesterol (LDL-C), have been instrumental in this trend. Not everyone responds to statins, however, and new types of cholesterol-lowering drugs are attracting great interest. Fitzgerald et al. tested inclisiran, a drug based on small interfering RNA technology, in a small phase 1 trial. Inclisiran induces degradation of the mRNA encoding PCSK9, a liver-derived protease whose activity increases serum LDL-C levels. Subcutaneous injection of inclisiran durably reduced PCSK9 levels by as much as 83% and LDL-C levels by as much as 59% without serious toxicities.

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