Therapeutic oligonucleotides: the promises are up for delivery

corresponding

JESPER WENGEL
Department of Chemistry, Farmacy and Physics, University of Southern Denmark

Dear reader,
This editorial is dedicated to therapeutic oligonucleotides, their existing challenges and how to effectively overcome them. In the OLIGONUCLEOTIDE AND PEPTIDES section of this issue you will in fact find two insights on RNA-based therapies, with historical perspectives and the pivotal role they are expected to have in the future. I would like to start with a bit of history and definitions. After about 40 years of development, RNA medicines now constitute a novel and emerging class of drugs. Most prominent are the two mRNA-based vaccines, BNT162b2 and mRNA-1273 developed by BioNTech/Pfizer and Moderna, respectively, which proved effective in establishing control of the coronavirus disease 2019 (COVID-19). As landmarks of successful science and innovation within the biotech and pharma industries, they have resulted in profound thrust in RNA-based drugs not only within these industries, but also among decision makers and the public. Scientific discoveries made in the preceding years including RNA delivery within lipid nanoparticles, in vivo translation of exogenous mRNA-like molecules, and modifications of RNA nucleotides, have enabled the realization of these ...