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Hyperlipidemia, a medical condition often referred to as high cholesterol, is characterized by abnormally elevated levels of lipids in the bloodstream. When present in excess, these lipids, specifically cholesterol and triglycerides, can lead to serious health complications, often involving cardiovascular diseases. Illnesses like atherosclerosis, heart attacks, and pancreatitis have all been linked to untreated hyperlipidemia. This means controlling and regulating cholesterol and triglyceride...
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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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Updated: Aug 5, 2025

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Small Interfering Ribonucleic Acid as Lipid-Lowering Therapy: Inclisiran in Focus.

Jelena Rakocevic1, Milan Dobric2,3, Rada Vucic4,5

  • 1Institute of Histology and Embryology "Aleksandar Đ. Kostić", Faculty of Medicine, University of Belgrade, 11000 Belgrade, Serbia.

International Journal of Molecular Sciences
|March 29, 2023
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Summary

Inclisiran, a novel siRNA therapy, offers sustained LDL cholesterol reduction with twice-yearly dosing for cardiovascular patients. This approach improves adherence compared to monthly injections for managing high cholesterol.

Keywords:
LDL cholesterolPCSK9inclisiransiRNAsmall interfering RNA

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Area of Science:

  • Cardiovascular Medicine
  • Pharmacology
  • Molecular Biology

Background:

  • Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) regulates low-density lipoprotein (LDL) cholesterol metabolism.
  • PCSK9 inhibition lowers LDL cholesterol and cardiovascular risk, but current antibody therapies require frequent injections.
  • Therapeutic adherence can be a challenge for cardiovascular patients on multiple medications.

Purpose of the Study:

  • To review current data on inclisiran, a small interfering ribonucleic acid (siRNA) therapy.
  • To critically evaluate major clinical trials assessing inclisiran's safety and efficacy.
  • To explore inclisiran's role in managing elevated LDL cholesterol levels.

Main Methods:

  • Review of available data and clinical trial results for inclisiran.
  • Assessment of inclisiran's efficacy in lowering LDL cholesterol.
  • Evaluation of inclisiran's safety and tolerability profile.

Main Results:

  • Inclisiran effectively and durably lowers LDL cholesterol levels.
  • The siRNA therapy demonstrates a favorable tolerability profile.
  • Twice-yearly administration of inclisiran offers a convenient dosing regimen.

Conclusions:

  • Inclisiran represents a promising therapeutic option for patients with elevated LDL cholesterol.
  • Its sustained efficacy and infrequent dosing may improve treatment adherence.
  • Further evaluation in diverse patient populations is ongoing.