Oligonucleotide synthesis has made significant progress, driven by the growing popularity of oligonucleotide-based therapeutics in drug discovery, as well as the increasing number of approved oligonucleotide drugs. Unlike traditional small molecule drugs that primarily act on proteins, oligonucleotide therapeutics function by binding to specific mRNAs, enabling the regulation of targets that were considered undruggable by conventional small molecules. This unique mechanism makes oligonucleotide therapeutics theoretically applicable to virtually any therapeutic targets.
Since the approval of the first therapeutic oligonucleotide, Fomivirsen (Vitravene), by the U.S. FDA in 1998, oligonucleotide-based therapies have emerged as powerful therapeutics for the treatment of human diseases. Recent innovations and breakthroughs in oligonucleotide delivery technologies have paved the way to various oligonucleotide modalities for tackling previously considered undruggable targets and unmet medical needs.
