Pre-Conference Focus Day

Tuesday 12th December 2023

8:15 am Registration Opens & Welcome Coffee

8:50 am Chair’s Opening Remarks

Optimizing Screening Technologies to Validate RNA Targets for Accelerated Hit to Lead

9:00 am Applying Biophysical Screening to Determine RNA Structure & Validate Targets


  • Understanding the importance of biophysical screening in progressing in a rational way
  • Understanding how compounds interact with RNA
  • Optimizing compounds to validate RNA Targets for small molecules – do the RNA-small molecule interactions have an effect?

9:30 am Comprehensive Interactome Profiling of RNA Library vs Compound Library Identifies Selective RNA Binders

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


  • A highly structured RNA library that includes RNA structures identified through in-cell probing
  • In vitro interactome of ~30,000 drug-like compounds vs ~1,000 RNA structures extracted from naturally occurring RNA
  • Quantitative profiling of selectivity between RNA structures and small molecules
  • Simultaneous analysis of binding modes at the single-nucleotide resolution

10:00 am Roundtable Discussion: Investigating the Dynamic Interactions Between RNA & Small Molecules to Determine Targetable RNA Regions

  • Mark Ledeboer Senior Vice President - Drug Discovery, Expansion Therapeutics


  • Fundamental understanding of the RNA structures being inhibited by small molecules
  • Exploring the current database to understand the different confirmations of RNA to identify targets
  • What are the approaches to narrow down potential candidates for drug targets that bind small molecules and elicit a response?

11:00 am Morning Networking Break

12:15 pm Structural Biology of mRNA Driving Drug Design


  • Identification of relevant mRNA isoforms and druggable regions
  • Selection of accurate and fast screening methods
  • Cellular assays for hit confirmation and optimization

Successfully Develop an Effective Toolbox to Fast-Track RNA Target Discovery

12:45 pm Panel Discussion: What Makes a ‘Good’ RNA Target?

  • Matthew Jones Head Of Platform Technology, NextRNA Therapeutics
  • Shunnichi Kashida Co-Founder, Representative Director, President & Chief Executive Officer, Officer xFOREST Therapeutics
  • Katie Warner Co-founder and VP of RNA Biology, Ribometrix
  • Balajee Somalinga Director - RNA Biology, CHDI Foundation


  • Understanding how to find the best RNA target to drug the undruggable
  • Which RNA structures would make suitable targets?
  • How to achieve successful proof-of-concept with validated targets?
  • Deciphering different validation levels

2:00 pm Lunch & Networking Break

Analyzing Screening Techniques to Determine Biological Effect & Improve Drug Affinity

3:00 pm Roundtable Session: Ensuring On-Target Effects when Using Phenotypic Screening & Fragment-Based Screening to Select Functional RNA Targets


  • Identifying targets that have a biologically functional effect
  • Leveraging the toxic side-effects of using phenotypic screening
  • Developing phenotypic screens to validate RNA targets that successfully bind the small molecule
  • Understanding how to generate fragment maps to get better predictability of targetable RNA
  • Screening large numbers of targets to synthesize RNA libraries

4:15 pm Chair’s Closing Remarks & End of Pre-Conference Focus Day