RNA-Targeted Small Molecule Drug Discovery
Novel Strategies Using Small Molecules to Target RNA Structure, Binding, Interactions, and Function
April 19, 2027 ALL TIMES PDT
RNA molecules have complex, dynamic structures which make them challenging drug targets. However, advances in elucidating RNA structure, function, and interactions have led to finding small molecules to target RNA for therapeutic intervention. AI models and machine learning algorithms have also helped predict RNA-ligand interactions and optimize compounds with enhanced stability, oral bioavailability, and better drug-like properties. However, identifying the right disease-causing RNA and evaluating the downstream physiological responses after small molecule binding can be quite challenging. Cambridge Healthtech Institute’s symposium on RNA-Targeted Small Molecule Drug Discovery will highlight some of the early discovery and preclinical work being done to target mRNA, non-coding RNA, microRNA, and other modalities for cardiometabolic, infectious, and neurologic disorders, oncology, and other genetic indications.