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Multiple Choice

Inclisiran is what type of therapy?

Inclisiran is an siRNA therapy that uses RNA interference to silence PCSK9 production in liver cells. The small interfering RNA is designed to bind specifically to PCSK9 mRNA, and once inside hepatocytes it is incorporated into the RNA-induced silencing complex to degrade that mRNA. With less PCSK9 produced, more LDL receptors remain on the surface of liver cells, enhancing clearance of LDL cholesterol from the blood and lowering LDL-C levels. This approach targets the source of PCSK9 rather than the circulating protein, which is why it’s distinguished from monoclonal antibodies that neutralize PCSK9 in the bloodstream. It’s not gene therapy, since it doesn’t edit the DNA, and it’s not a small-molecule inhibitor.

Inclisiran is an siRNA therapy that uses RNA interference to silence PCSK9 production in liver cells. The small interfering RNA is designed to bind specifically to PCSK9 mRNA, and once inside hepatocytes it is incorporated into the RNA-induced silencing complex to degrade that mRNA. With less PCSK9 produced, more LDL receptors remain on the surface of liver cells, enhancing clearance of LDL cholesterol from the blood and lowering LDL-C levels. This approach targets the source of PCSK9 rather than the circulating protein, which is why it’s distinguished from monoclonal antibodies that neutralize PCSK9 in the bloodstream. It’s not gene therapy, since it doesn’t edit the DNA, and it’s not a small-molecule inhibitor.