Messenger RNA as a Novel Therapeutic Approach

The concept of mRNA as a therapeutic platform has historically been ignored owing to challenges in oligonucleotide delivery and, maybe more importantly, the perceived shortcomings of mRNA with regard to stability and immunogenicity. Advances in several areas have recently prompted a reexamination of such dogma. Significant improvements in oligonucleotide delivery have been realized over the past decade and their application to mRNA has enabled a more rapid path toward clinical development of this new modality. Similarly, recent discoveries in mRNA chemistry further enhance the attractiveness of this platform by attenuating innate immune activation and maximizing protein expression. With these advances, mRNA is positioned to become an important new therapeutic modality.