TY - JOUR
T1 - RNA therapeutics in cardiovascular medicine
AU - Damase, Tulsi Ram
AU - Cooke, John P.
N1 - Publisher Copyright:
Copyright © 2025 Wolters Kluwer Health, Inc. All rights reserved.
PY - 2025/5/1
Y1 - 2025/5/1
N2 - Purpose of review RNA therapeutics came to global attention when mRNA-based vaccines provided an answer to the SARSCoV-2 pandemic. The immense significance of this development notwithstanding, it is important to note that almost a decade prior to the pandemic, RNA drugs had made important inroads toward the amelioration of disease. The first class of RNA therapies to be introduced into clinical use were the antisense oligomers and siRNA drugs which generally induce a therapeutic effect by acting to brake or to modulate mRNA expression. RNA therapeutics is quickly becoming the fourth pillar of pharmacotherapy, and will have broad applications, including for the treatment of cardiovascular disease. Recent findings The United States (US) Food and Drug Administration (FDA) has approved several antisense oligomers (ASOs) and siRNA-based drugs to treat disorders associated with cardiovascular disease. In addition, multiple RNA-based drugs are in clinical trials to assess their safety and efficacy in patients with cardiovascular disorders, such as Zodasiran, a siRNA therapy that targets angiopoietin-like protein 3 (ANGPTL3) to reduce LDL cholesterol. Summary Because of limitless sequence choice; speed of design; and relative ease of synthesis, RNA drugs will be rapidly developed, will have broad applications, and will be generated at lower cost than other drug types. This review aims to highlight RNA therapies for cardiovascular diseases that are approved, and those that are under clinical evaluation.
AB - Purpose of review RNA therapeutics came to global attention when mRNA-based vaccines provided an answer to the SARSCoV-2 pandemic. The immense significance of this development notwithstanding, it is important to note that almost a decade prior to the pandemic, RNA drugs had made important inroads toward the amelioration of disease. The first class of RNA therapies to be introduced into clinical use were the antisense oligomers and siRNA drugs which generally induce a therapeutic effect by acting to brake or to modulate mRNA expression. RNA therapeutics is quickly becoming the fourth pillar of pharmacotherapy, and will have broad applications, including for the treatment of cardiovascular disease. Recent findings The United States (US) Food and Drug Administration (FDA) has approved several antisense oligomers (ASOs) and siRNA-based drugs to treat disorders associated with cardiovascular disease. In addition, multiple RNA-based drugs are in clinical trials to assess their safety and efficacy in patients with cardiovascular disorders, such as Zodasiran, a siRNA therapy that targets angiopoietin-like protein 3 (ANGPTL3) to reduce LDL cholesterol. Summary Because of limitless sequence choice; speed of design; and relative ease of synthesis, RNA drugs will be rapidly developed, will have broad applications, and will be generated at lower cost than other drug types. This review aims to highlight RNA therapies for cardiovascular diseases that are approved, and those that are under clinical evaluation.
KW - RNA therapeutics
KW - antisense oligonucleotide
KW - cardiovascular medicine
KW - messenger RNA
KW - microRNA
KW - small interfering RNA
UR - http://www.scopus.com/inward/record.url?scp=105002429835&partnerID=8YFLogxK
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U2 - 10.1097/HCO.0000000000001210
DO - 10.1097/HCO.0000000000001210
M3 - Review article
C2 - 39998478
AN - SCOPUS:105002429835
SN - 0268-4705
VL - 40
SP - 139
EP - 149
JO - Current Opinion in Cardiology
JF - Current Opinion in Cardiology
IS - 3
ER -