TABLE 1.

Examples of FDA approved and clinical trial of small molecules that bind to RNAs with applications in oncology and neurology (Childs-Disney et al., 2022).

Therapeutic area	Associated disease	Compound/institution	RNA target	Mode of action	Stage of development	Ref
Oncology	Triple-negative breast cancer and chronic myelogenous leukaemia	Zotatifin (eFT226)/eFFECTOR Therapeutics & Inception Therapeutics	Polypurine sequences in the 5′UTR of a subset of oncogenic mRNAs	Inhibits translation initiation by clamping eIF4A to polypurine RNA sequence in the 5′UTR	Phase I–II clinical trial: NCT04092673	Ernst et al. (2020)
Neurology	SMA	Risdiplam/Roche and PTC Therapeutics	SMN2 pre-mRNA exon 7–intron junction	Promotes exon inclusion by stabilizing the binding of the splicing machinery	FDA approved	Ratni et al. (2018)
Neurology	SMA	Branaplam	SMN2 pre-mRNA exon 7–intron junction	Promotes exon inclusion by stabilizing the binding of the splicing machinery	Phase I/II clinical trial (for SMA: NCT02268552; for HD: NCT05111249)	Palacino et al. (2015)