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

Stereo Chemistry · Jan 3, 2019 · 1m

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…