For decades, scientists believed in the seemingly endless possibilities of custom-made messenger RNA, or mRNA, and the launch of the mRNA vaccines by Pfizer and Moderna changed the therapeutics world overnight.
However, transforming scientific promises into medical reality is more complicated. It is challenging to modify synthetic mRNA precisely. With no template to scale sequences quickly, the industry relies on experts who deeply understand LNP analytics and bioassays, which is critical to accelerating commercialization.
The rapid development and approval of messenger-RNA (mRNA) vaccines during the COVID-19 pandemic has motivated and encouraged the pharmaceutical industry to explore and expand therapeutics breakthroughs for a wide range of diseases, including many cancers, and transform medicine.
The mRNA technology is new and still in the early stages, with no standardized manufacturing protocols. One of the biggest challenges is the formulation of the mRNA delivery systems like lipid nanoparticles (LNPs). The currently approved mRNA vaccines are formulated with LNP delivery vehicles as LNPs are the gold standard technology showing a good track record of protecting the mRNA cargo from degradation and mediating a targeted, safe, and efficient intracellular delivery due to their: modularity and capacity. The lipid excipients can be mixed, matched, and modified to deliver a variety of nucleic acid APIs, and LNPs also allow the delivery of larger mRNAs along with the potential to co-deliver multiple RNAs, enabling new therapeutics.
Learn about evading impurities in lipids, the significance of the 'biological readout,' and the future of mRNA-based genomic medicines.