Novel RNAi-Based Therapies for Atherosclerosis

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.