Kang, C., Nguyen, H. T., Heppner, D. E., Yu, B., & Xu, W. (2026). Small-Molecule Drug Discovery Targeting RNAs: Hope or Hype? Journal of Medicinal Chemistry, 69(3), 1786–1789. https://doi.org/10.1021/acs.jmedchem.6c00070
Abstract:
Over the past decade, RNA has emerged as an attractive and evolving landscape for small molecule drug discovery. RNA functions as an intermediate macromolecule during gene expression and plays a diverse role in regulating cellular processes 1. The promise in targeting RNA as therapeutic interventions arises from recent breakthrough in structural biology, chemical biology, and computational modeling that collectively facilitate understanding of RNA biology. Multiple classes of RNA have been identified contributing to their functional diversity, including messenger RNA (mRNA), transfer RNA (tRNA), ribosomal RNA (rRNA), long non-coding RNA (lncRNA), microRNA (miRNA), and other non-coding RNAs (ncRNAs). While only a tiny fraction of 1.5% of human genome is translated into proteins, approximately 75% is transcribed into RNA2-4, making RNA a vastly larger reservoir of potential drug targets than the traditional protein targets. Moreover, many disease-associated pathways including viral replication, cancer progression, neurodegeneration, and immune regulation are regulated by RNA or through its interactions with different proteins, making RNA an increasingly relevant and strategic focus for next-generation drug discovery.
License type:
Publisher Copyright
Funding Info:
This research is supported by core funding from: Experimental Drug Development Centre (EDDC)
Grant Reference no. : Core