Novel RNAi-Based Therapies for Atherosclerosis

Anna-Kaisa Ruotsalainen1,2, Petri Mäkinen1, Seppo Ylä-Herttuala3,4

  • 1A.I. Virtanen Institute for Molecular Sciences, University of Eastern Finland, P.O. Box 1627, FIN-70211, Kuopio, Finland.

Insights

Novel RNA interference (RNAi) therapies are emerging for atherosclerosis, a condition causing cardiovascular diseases. Inclisiran, an approved RNAi drug, effectively lowers cholesterol by silencing PCSK9, marking a new era in cardiovascular disease treatment.

Area of Science:

  • Cardiovascular Science
  • Molecular Biology
  • Pharmacology

Background:

  • Atherosclerosis, characterized by arterial inflammation and lipid accumulation, underlies major cardiovascular diseases (CVD) like coronary artery disease, peripheral artery disease, and stroke.
  • Effective management of atherosclerosis risk factors is crucial for preventing CVD events.

Purpose of the Study:

  • To review and describe novel RNA interference (RNAi)-based therapies for atherosclerosis.
  • To highlight emerging RNAi treatments in clinical trials and on the market.

Main Methods:

  • Review of current clinical trials and approved RNAi therapeutics.
  • Analysis of the mechanism of action and clinical outcomes of RNAi therapies targeting atherosclerosis risk factors.

Main Results:

  • The first RNAi-based therapies are now available for managing atherosclerosis risk factors, specifically blood cholesterol levels.
  • Inclisiran, an approved RNAi therapy, silences proprotein convertase subtilisin/kexin type 9 (PCSK9), significantly reducing plasma cholesterol and hypercholesterolemia.
  • New RNAi therapies are entering the market, with ongoing evaluation in larger patient populations.

Conclusions:

  • RNAi therapeutics represent a significant advancement in cardiovascular disease pharmacotherapy.
  • The clinical utility and market integration of these novel RNAi drugs for atherosclerosis require further assessment in real-world settings.
Abstract

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