RNA-targeted therapeutics, including antisense oligonucleotide technologies as well as small interfering RNAs, represent a new class of medications that may overcome several of the disadvantages of small molecule drugs or monoclonal antibodies. Specifically, upstream targeting at the mRNA level renders any disease-related protein a potential target, even those pathways previously deemed 'undruggable'. Additional advantages include the comparably simple and cost-effective way of manufacturing and the long dosing intervals. A few agents are already approved and a wide array of cardiovascular drugs are in development, aimed at hypercholesterolemia, hypertension, myocardial storage diseases and the coagulation system. Here, we provide an update on the current status of RNA-targeted therapeutics in the cardiovascular arena.
Keywords: Antisense oligonucleotides; Cardiovascular disease; RNA; SiRNA.
© The Author(s) 2022. Published by Oxford University Press on behalf of the European Society of Cardiology.