Cholesterol, a type of lipid molecule, can be produced by the body itself and obtained from food. Cholesterol has many biological functions. It is a major component of cell membrane, and the body uses it to make hormones, vitamin D, and bile acids. However, too much cholesterol in the blood can cause serious health consequences. The most common form of the body's cholesterol complexes is LDL cholesterol, which is also called "bad" cholesterol because having high levels in your blood leads to atherosclerosis, a major cause of heart attacks, stroke, and peripheral vascular disease. There are a handful of drugs that lower LDL cholesterol levels, such as statins, niacin, and bile acid resin. But these drugs may cause side effects, and not everyone responds well to them. Previous studies have discovered that a protein called PCSK9 promotes the degradation of the LDL receptor, which typically mediates the endocytosis of cholesterol-rich LDL and thus maintains the plasma level of LDL. So targeting PCSK9 provides a therapeutic strategy in people with high LDL levels. Now a study in New England Journal of Medicine shows that a single dose of inclisiran, a new drug designed to target PCSK9 messenger RNA, lowers PCSK9 and LDL cholesterol levels in patients at high risk for cardiovascular disease who have elevated LDL cholesterol levels. The study is Imperial College London researchers. The researchers used a method known as RNA interference (RNAi), in which small interfering RNAs (siRNAs) are used to selectively silence the translation of their target messenger RNAs, preventing the expression of the harmful proteins. In previous work, the researchers tested inclisiran -- a siRNA molecule that targets the PCSK9 messenger RNA -- in healthy volunteers and found that it produced sustained reductions in LDL cholesterol levels. For the current work, they conducted a phase 2 clinical trial of the drug in people at high risk for cardiovascular disease who had increased LDL cholesterol levels. A total of 501 people were randomly assigned to receive placebo or inclisiran. People receiving inclisiran had significant reductions in PCSK9 and LDL cholesterol levels. Moreover, one dose of inclisiran was enough to produce durable effects. Serious adverse events occurred in 11% of the inclisiran group and in 8% of the placebo group. Overall, the trial reveals that inclisiran has the potential to control LDL cholesterol levels for a long period of time among people at high cardiovascular risk with elevated LDL cholesterol but more investigation is needed. CusAb offers PCSK9 and Recombinant DSC2.