The Progress and Promise of RNA Medicine─An Arsenal of Targeted Treatments

J Med Chem. 2022 May 26;65(10):6975-7015. doi: 10.1021/acs.jmedchem.2c00024. Epub 2022 May 9.

Abstract

In the past decade, there has been a shift in research, clinical development, and commercial activity to exploit the many physiological roles of RNA for use in medicine. With the rapid success in the development of lipid-RNA nanoparticles for mRNA vaccines against COVID-19 and with several approved RNA-based drugs, RNA has catapulted to the forefront of drug research. With diverse functions beyond the role of mRNA in producing antigens or therapeutic proteins, many classes of RNA serve regulatory roles in cells and tissues. These RNAs have potential as new therapeutics, with RNA itself serving as either a drug or a target. Here, based on the CAS Content Collection, we provide a landscape view of the current state and outline trends in RNA research in medicine across time, geography, therapeutic pipelines, chemical modifications, and delivery mechanisms.

Publication types

  • Review

MeSH terms

  • COVID-19 Drug Treatment*
  • COVID-19 Vaccines*
  • Humans
  • RNA
  • RNA, Messenger / metabolism
  • SARS-CoV-2

Substances

  • COVID-19 Vaccines
  • RNA, Messenger
  • RNA