A platform for RNA

An information explosion on the number of functional RNA molecules expressed in cells and the mechanisms by which they control gene expression^1,2 is driving the formation and funding of biotechs developing oligonucleotide-based therapeutics. Pharmas and big biotechs are carving up the space via partnerships that add multiple nucleic acid-based mechanisms and technologies to their drug discovery toolboxes.

The newest additions to the nucleic acid platform include noncoding RNA targets such as microRNAs and long noncoding RNAs (lncRNAs). Both types of molecule create the opportunity to selectively turn on expression of a given gene, potentially achieving biological and therapeutic outcomes that no other drug platform can...