Updated: Feb 17, 2020
Messenger RNA-based protein expression technology is validated by numerous preclinical studies and clinical trials to be a safe and efficacious modality for exogenous protein expression in humans. mRNA is the best way to transiently express proteins in cells or the organisms. When considering hit-and-run applications such as vaccination, tissue engineering, or gene editing protein expression (e.g. Cas9) mRNA provides unique advantages because of mRNAs do not cause insertional mutagenesis by integrating into the genome. In a clinical setting, transient nature of mRNA mediated protein expression limits the duration of possible adverse effects. Moreover, because mRNA works inside the cells, unlike traditional mAb or recombinant protein-based therapies, mRNA opens the door to cell specific protein expression.
Compared to DNA, mRNA is easier to deliver into cells, including non-dividing ones, because it does not require nucleofection. Compared to viral and cellular therapies, mRNA is easier to manufacture because it is produced via scalable in vitro transcription reaction, which is a cell-free process and therefore does not require cell culture or complex biological processes. This low cost and high yield manufacturing process also enables advanced medical applications such as personalized medicine and multi-antigen (cocktail) vaccines. Finally, mRNA delivery is enabled via lipid nanoparticle technology, which is also clinically shown to be safe and effective for RNA delivery in humans.