Small-molecule treatments have attracted interest in not just targeting proteins but also in RNA regulation. For targets in difficult-to-treat cancer especially, this offers fresh opportunities. The promise of RNA targeting is shown by the effectiveness of PCSK9 inhibition with R-IMPP, a small molecule that binds especially to ribosomes. In clinical studies, experimental data on RNA-targeted drugs, including linezolid and ribocil, has shown their efficacy. This implies that RNAs causing diseases can offer particular sites where medications might bind. As our knowledge of structure develops, we could be able to apply several strategies of action, including translational stalling and splicing control.