Exploring RNA-Focused Chemical Space Beyond Known Chemotypes to Inspire New Hit Discovery Strategies
The chemical space explored for RNA-targeted small molecules remains relatively
narrow and heavily influenced by existing patents, public datasets, and legacy screening
collections. While these efforts have produced important advances, they have also
concentrated discovery around a limited set of chemical frameworks and design
principles. As more organizations pursue splicing modulation, RNA degradation,
translational control, and direct RNA binding, there is growing interest in identifying
chemical matter that extends beyond familiar RNA-targeted chemotypes while
remaining productive, interpretable, and actionable for discovery.
This workshop will bring together medicinal chemists, computational chemists,
screening scientists, chemical biologists, and RNA drug discovery leaders to discuss
how the field can broaden RNA-focused chemical space.
Topics for discussion include:
- Examining where current RNA-targeted chemistry is concentrated across patents, public datasets, commercial libraries, and internal collections
- Exploring how chemically diverse yet information-rich chemical spaces can improve both hit discovery and the extraction of actionable design principles
- Comparing chemical requirements across splicing modulation, RNA degradation, translational inhibition, and direct RNA binding
- Discussing how molecular glue degrader thinking could inspire new approaches to RNA or protein depletion
- Evaluating how public structures, shape-based searching, cheminformatics, and AI can support broader chemical exploration