mRNA-based pharmaceuticals : A literature study based on current knowledge of mRNA

Sammanfattning: This literature study provides a comprehensive overview of the current knowledge of mRNA-based pharmaceuticals, technologies and applications.  mRNA-based pharmaceuticals have newly been bought into focus as a potential brand-new drug class and many believe that it has the potential to transform areas of medicine. The corona pandemic showed that mRNA vaccines could be created fast and safely which highlighted the unique potential of mRNA therapy. This resulted in a major investment in mRNA therapeutics and attracted attention to synthetic mRNA as a new drug modality. The sequence of nucleotides in synthetically produced (IVT) mRNA can easily be altered to produce different proteins in vivo. This leads to a broad range of therapeutical applications such as protein replacement therapy, immunotherapy, genome editing, cellular programming, and vaccines.  The disadvantages of the mRNA technique will be discussed; primarily the low stabilization in vivo and low target delivery, which have partly been solved today. By nucleobase modifications the stabilization can be improved and by delivery systems an increased target delivery can be achieved.  The clinical promise of mRNA therapeutics and vaccines is crystal clear, however, their novelty brings new manufacturing challenges. Technologies and strategies to address these challenges will be concluded in this paper.