Cambridge Healthtech Institute’s 6th Annual

RNA-Modulating Small Molecule Drugs

Novel Approaches to Target RNA Structure, Binding, Interactions, and Function

April 14, 2025

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.

Coverage will likely include:

Identifying and targeting the right RNA structures and mechanisms for therapeutic intervention
New functional and screening assays for quantifying small molecule-RNA binding and downstream function
Strategies for characterization and optimization of the RNA modulators, binders, and degraders
Novel approaches for targeting and degrading RNA-modifying proteins and enzymes
Using AI and machine learning to design and predict RNA modulating activity

The deadline for priority consideration is September 27, 2024.

All proposals are subject to review by session chairpersons and/or the Scientific Advisory Committee to ensure the overall quality of the conference program. Additionally, as per Cambridge Healthtech Institute’s policy, a select number of vendors and consultants who provide products and services will be offered opportunities for podium presentation slots based on a variety of Corporate Sponsorships.

Opportunities for Participation: