Ep. 14: On the face of it, RNA is a terrible drug target

RNA should be a terrible drug target. It's long, noodle-like structure lacks the nooks and crannies that small molecule drugs use to grab onto proteins and thereby control them. But a decades-old disregard for RNA is starting to change. In August 2018, the US Food and Drug Administration approved the first-ever RNA interference (RNAi) drug, which uses a double-stranded RNA molecule to prevent the production of disease-related proteins. In the past two years, several startups have launched to show that some RNAs can, just like proteins, be drugged with small molecules. And a third group of companies recently emerged with plans to drug proteins that make modifications to RNA, part of the budding field of epitranscriptomics. In this episode, C&EN visits Alnylam Pharmaceuticals, Novartis, and Accent Therapeutics to discuss these three strategies, and to understand how RNA-modulating therapies will compete in the wider world of drug discovery. Visit our website for a script of this podcast. Music credits: "And...(Insert Problem Here)" by GR∑Y is licensed under CC BY-NC 4.0.  "Raccoon Family Robinson", "Robot Park", and "The Confrontation" by Podington Bear are licensed under CC BY-NC 3.0.  "Wireless" by Lee Rosevere is licensed under CC BY-NC 4.0.  Image credit: J. Am. Chem. Soc.