8:00 am Check-In Opens & Light Breakfast

8:50 am Chair’s Opening Remarks

Uncovering Tools to Accurately Determine & Validate RNA Target Structure for Better Knowledge of Druggable Pockets to Improve RNA Binding

9:00 am NMR as a Tool for Structure Characterization of RNA Targets in Drug Discovery

  • Peter Connolly Vice President, Structural Biology & Biophysics, Expansion Therapeutics


  • Using methods to predict and confirm RNA secondary structure
  • Using NMR to better understand the structure of RNA in structure guided drug design
  • Using NMR in combination with other methods to characterize drug binding on RNA targets

9:30 am From HTT RNA Target Validation to Identifying Compounds that Show Selective Binding


  • Validation of the RNA target using structural tools
  • Development of assays to screen and characterize RNA binders
  • Identification of compounds that show selective binding

10:00 am Morning Break & Speed Networking


This session is your opportunity to get face-to-face with many of the brightest minds working in the

RNA targeted drug discovery field, and establish meaningful business relationships to pursue for the

rest of the conference.

Optimizing Methods Used to Validate RNA-Small Molecule Interactions for Discovery of Efficacious RNA Targeting Therapeutics

11:00 am Fireside Chat – Discussing How to Develop Assays for Accurate Validation of RNA Binding to Fast-Track Candidates Towards Patients

  • Ku-Lung (Ken) Hsu Associate Professor - Chemistry, University of Texas at Austin
  • Shunnichi Kashida Co-Founder, Representative Director, President & Chief Executive Officer, xFOREST Therapeutics Co. Ltd.


  • How is the field currently approaching validating RNA targets and what learnings can be taken from validating protein targets?
  • Given there is no one size fits all model, how can you approach translating validation assays between different targets to reduce the time between finding a hit and then developing into a lead?
  • How much customization is required for a validation assay for a novel target?

12:00 pm Lunch & Networking

Turbocharging Small Molecule Selectivity by Optimizing Current Screening & Assay Methods to Accelerate Targeted Drug Discovery

1:00 pm An Integrative Structure-Based Approach to Discovering RNA-targeted Small Molecules


  • RNA structure identification, screening, and hit characterization
  • Integrative approach to inform on RNA/ligand interaction
  • High-resolution structure determination of RNA/small molecule complexes to accelerate ligand optimization

1:30 pm Discovering Small Molecule RNA Interactions by High Sensitivity Equilibrium Screening


  • Leveraging insights from riboswitches for understanding small molecule interactions with eukaryotic RNAs
  • Deploying functional assays early in hit validation
  • Enhancing screening hit confidence through integration of diverse binding data sources

2:00 pm Afternoon Break & Scientific Poster Session


Contribute to the conversation and share your cutting-edge research with your fellow RNA-targeting community to showcase your breakthrough discoveries to a vast audience of experts. Register your place to submit an abstract for review to showcase your poster*

*Please visit the website for the T&Cs for presenting a poster

3:00 pm Discovery of Selective RNA Binders Through a Quantitative High Throughput Primary Biochemical Screen

  • Chuhern Hwang Co-Founder & Head of Drug Discovery, Wayfinder Biosciences


  • Utilizing biophysical design methods to develop RNA switch-based sensors to identify compounds that modulate target RNA structure
  • Rapidly generating highly quantitative datasets on binding and selectivity of compound libraries against RNA targets
  • Leveraging machine learning models trained on highly quantitative HTS data to generate novel binders for RNAs through virtual screening of millions of molecules

3:30 pm Comprehensive Interactome Profiling for Screening & Optimizing RNA Binders with High Affinity & Selectivity

  • Shunnichi Kashida Co-Founder, Representative Director, President & Chief Executive Officer, xFOREST Therapeutics Co. Ltd.


  • Highly structured RNA exhibit potential for high affinity with small molecules
  • vitro interactome profiling of 30,000~ drug-like compounds vs. 2,000~ RNA structures extracted from disease related RNA
  • SAR analyses of target binding modes at single-nucleotide resolution and binding selectivity among compound derivatives

4:00 pm Chair’s Closing Remarks & End of Conference Day One

4:30 pm End of Conference Day One