Oligonucleotide and mRNA Therapeutics

Oligonucleotide therapeutics are nucleic acid polymers generally comprised of a few to several dozen bases (including modified bases) linked together. They are produced by chemical synthesis and act directly on organisms without being translated into proteins. Oligonucleotide therapeutics are characterized by the ability to target specific diseases. Another advantage is that it takes less time than conventional methods to find new therapeutic candidates because oligonucleotides are easy to design and synthesize.
However, it is a practical problem that oligonucleotide therapeutics are degraded and excreted rapidly after administration by exonucleases and endonucleases that are abundant in blood and cells. This problem is being addressed by the introduction of modified oligonucleotides to improve chemical stability in vivo and by the development of lesion-targeted DDS (drug delivery systems) technologies.

mRNA therapeutics and vaccines have been under investigation since before the development and approval of COVID-19 vaccines. More recently, they have received regulatory approval for the prevention of infectious diseases beyond COVID-19, with research efforts expanding into cancer vaccines and other therapeutics. Because the mRNA molecules used in these products have a molecular weight far greater than that of conventional nucleic acid drugs—spanning approximately 1,000 to 10,000 nucleotides in length—they possess quality attributes distinct from those of oligonucleotide therapeutics. Consequently, there is a growing need for analytical methods capable of appropriately characterizing these attributes.

Oligonucleotide therapeutics are comprised of a few to several dozen nucleic acids (including modified ones) and chemically synthesized. Oligonucleotide therapeutics can effectively act on specific diseases and are easy to design and synthesize. These characteristics can faster to find new therapeutic candidates.

This infographic presents the different types of therapeutic oligonucleotides and highlights some innovative solutions for their analysis.