Pre-Conference Assay Development & Screening Focus Day

Tuesday, 13 December | 2022

8:00 am Registration & Morning Networking Coffee

8:50 am Chair’s Opening Remarks

  • Matt Smola Associate Director - RNA Biology, Ribometrix

Exploring the Use of Biophysical & Biochemical Methods to Enable RNA Structure Determination

9:00 am Rational Design of Small Molecule Modulators of RNA: Opportunities & Challenges

  • Peter Connolly Senior Director - Structural Biology, Expansion Therapeutics


  • Targeting RNA with small molecules provides an opportunity to modulate “undruggable” protein targets indirectly
  • The integration of structural, biophysical, and computational methods has proven to be successful in the rational design of small molecules directed to protein targets
  • The relative immaturity of these methods applied to RNA creates a challenge for the rational design of small molecules directed to RNA targets

9:30 am Leveraging Big Data & Machine Learning to Discover Targetable RNA Structures

  • Dave Mauger Director & Head of Data Science, Arrakis Therapeutics


  • Global transcriptomic data are critical to understanding mRNA isoform diversity across cells and tissues
  • Integrating global biochemical assays with biophysical data enables the identification of relevant RNA structures
  • Transcriptomic target engagement data measures selectivity and enables target discovery

10:00 am Studying the effects of RNA-small molecule interactions at scale


  • Learnings from building a map of the druggable transcriptome
  • High throughput methods to determine transcriptome wide – small molecule – RNA interactions

10:30 am Morning Networking Break

Developing Novel Tools & Systems to Optimize RNA-Targeting Small Molecule Drug Discovery

11:00 am Discovery of Small molecules Targeting RNA – Digital Presentation


  • Multiple roles of NMR in early drug discovery
  • From hit to lead through intelligent compound selection as an alternative to HTS
  • Various examples of our target portfolio

11:30 am Tools & Tactics for Targeting RNA with Drug-Like Small Molecules


  • Small Molecule Microarrays as a powerful platform for RNA ligand discovery
  • Development of a predictive algorithm for druglike small molecules that target RNA from unbiased libraries
  • Discussion of diverse technologies to study and identify RNA-ligand interactions

12:00 pm Panel Discussion – Combining Biochemical, Biophysical & Cell-Based Assay Systems for the Future of RNA Drug Discovery


  • Leveraging the learnings to determine the best assays for RNA-targeted drug discovery
  • How to effectively use conventional biophysical methods?
  • How to successfully measure biochemical activity?
  • How to develop novel approaches for cell-based systems?

12:30 pm Networking Lunch Break

Enhancing Cell-Based Assay Capabilities to Leverage Functional RNA-Based Target Discovery

1:30 pm Targeting Functional RNA Structures in Coronaviruses – Digital Presentation


  • The genomes of human-transmissible coronaviruses contain highly conserved RNA structures essential for viral replication
  • NMR-based fragment screening identified druggable RNA elements
  • In-cell assays show inhibition of RNA function after fragment growth

2:00 pm Multi-Targeting mRNA: Antibiotic Against Resistant Bacteria, Thwarts Emerging Resistant Strains & is Active Against Biofilms

  • Paul Agris Professor Duke, University School of Medicine


  • mRNA targeting strategy against antibiotic resistant bacteria
  • Screening with high throughput structure-based virtual screening (SBVS)
  • Assessment of efficacy, thwarting emerging resistance, attacking biofilms

2:30 pm Advancing Drug Discovery for RNA-Protein Interactions


  • Highlight the role of RNA-binding proteins in RNA biology and their druggability
  • Introduce new technologies for assaying RNA-protein interactions in live cells
  • Discuss lessons learned from screening against RNA-protein interactions

3:00 pm Unlocking Functional RNA Features for Drug Discovery


  • Overview of cell-based systems for RNA-based target discovery
  • Functional genomics aided RNA target discovery
  • Assays for validation and confirmation

3:30 pm Chair’s Closing Remarks

  • Matt Smola Associate Director - RNA Biology, Ribometrix