From small molecules to oligonucleotides: artificial intelligence for RNA-targeted drug discovery

Artificial intelligence (AI) has advanced rapidly in recent years, offering transformative opportunities across chemistry and the biological sciences. Yet effectively integrating these tools into everyday research remains a key challenge. This mini review summarizes recent advances in applying AI to RNA-targeted drug discovery, encompassing both small-molecule approaches and oligonucleotide therapeutics. It examines how AI-driven RNA structure prediction, target-site discovery, and interaction prediction underpin both modalities. Current limitations and future opportunities are also discussed, with particular emphasis on improving the reliability, automation, and translational utility of AI-enabled therapeutic design.