Dalya Gartzman

Identifying RNA targets that are both biologically meaningful and chemically tractable

remains one of the biggest bottlenecks in RNA-targeted drug discovery. Many RNA

regions are capable of binding small molecules, but only a subset controls diseaserelevant

biology in a way that can support a therapeutic hypothesis.

Join RNA biologists, screening scientists, translational researchers, and discovery

teams to explore practical strategies for:

• Identifying which RNA transcripts, structures and regulatory elements are suitable for small-molecule intervention
• Moving beyond one-at-a-time target discovery with scalable screening and functional assay approaches
• Using chemical probing to distinguish dynamic RNA structures from functionally important conformational switches
• Matching each RNA target to the most appropriate therapeutic hypothesis, from splice modulation to direct binding, degradation or translational inhibition
• Learning from screening approaches that failed to generate actionable targets, functional hits or reliable discovery decisions