US EditionRNA-Targeting Splice Modulators Target Difficult to Treat CNS Disorders
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RNA-Targeting Splice Modulators Target Difficult to Treat CNS Disorders
Once thought undruggable, RNA is now fair game for small molecules. Since the approval of Risdiplam, the first FDA-approved RNA-targeting small molecule, there has been a wave of small molecule splice modulators in preclinical and clinical development. These small molecules act upstream of translation, modulating gene expression via RNA, with the potential to treat repeat expansion and splicing-driven diseases. This review focuses on diseases of…
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