Prospects of Small Molecule Drug Discovery Targeting RNA

Abstract

Progress in RNA research following the decoding of human genomes has revealed that RNA plays an important role in intracellular signal transduction and gene regulation and is involved in many diseases. RNA is expected to provide new opportunities for small molecule drug discovery that have targeted druggable proteins. In August 2020, SMN2-directed RNA splicing modifier, risdiplam was approved by the FDA as a small molecule drug for spinal muscular atrophy. This spurs active discussion on the possibility of small molecule drug discovery targeting RNA. In this review, I will summarize the history of RNA-targeting small molecules and the discovery of risdiplam. I will also describe the prospects of small molecule drug discovery targeting RNA.