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

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.