The progress and promise of RNA medicine—an arsenal of targeted treatments

In the last decade, there has been a shift in research, clinical development, and commercial activity to exploit the many roles of RNA in physiology 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 a regulatory role in cells and tissues. These RNAs have potential as new therapeutics, with the RNA itself serving as either a drug or a target. These new types of RNA drugs require a plethora of modification chemistries to improve their therapeutic benefit. We describe the current state of the art for RNA medicine. Using the CAS Content Collection data, we examine the publication trends covering the roles of RNA in the cell, the application of RNA in medicine, and the use of chemical modifications and nanotechnology to deliver effective RNA pharmaceuticals to their cellular targets. This review reveals the sustained global effort that propelled this field to the cusp of realization for novel medical applications of RNA in many diseases. It serves as an easy-to-understand overview so that scientists from many different disciplines can appreciate the current state of the field of RNA medicine and join in solving the remaining challenges for fulfilling its potential.