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We are entering an era of innovative small molecule drug discovery by leveraging new modalities. Among them, directly targeting RNA and modulating RNA processes represents an exciting direction. Directly targeting RNA provides unique therapeutic opportunities that are not accessible for other modalities; more importantly, some of the approaches have achieved clinical success. As of now, human RNA as a drug target class has largely remained underexplored and holds significant potential. However, there are considerable challenges and risks, including target selection, mechanism of action, hit finding, validation and optimization, and translation of small molecule-RNA binding to functional phenotype, to be carefully evaluated to avoid pitfalls and increase the chances of success.

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