Cambridge Healthtech Institute’s 6th Annual
RNA-Modulating Small Molecule Drugs
Novel Approaches to Target RNA Structure, Binding, Interactions, and Function
April 14, 2025 ALL TIMES PDT
With an increased understanding of RNA structure, function, and interactions, there is greater interest in finding small molecules to target RNA for therapeutic intervention, as they offer 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. Challenges also exist in terms of optimizing the specificity, selectivity, and safety of these small molecules in vivo. Cambridge Healthtech Institute’s symposium on RNA-Modulating Small Molecule Drugs will highlight some of the innovative approaches and technologies being used.