Day One

8:30 am | Registration Opens & Morning Coffee Networking

8:50 am Chair’s Opening Remarks

Exploring RNA Structure, Dynamics & Target Recognition

9:00 am Keynote Address: Details to be Announced

9:25 am Determining High-Resolution Dynamic Ensembles of RNA Structures for Small Molecule Drug Discovery


  • Importance of resolving the motional modes of RNA at high-resolution
  • Methods for RNA ensemble determination and current industry limitations
  • Leveraging high-resolution RNA ensembles for structure-based small molecule discovery and optimization

9:50 am Structure-Based Drug Design for Targeting RNA: Preclinical Validation

  • Valerie Cullen Senior Vice president, Research, Expansion Therapeutics


  • Expansion repeat disorders: unmet medical need
  • Drugging RNA using structure-informed design
  • Preclinical validation of this approach

10:15 am Drugging RNA – A Structure-Based Approach

  • Jennifer Petter Founder and Chief Innovation Officer , Arrakis Therapeutics


  • Discussing the breadth of opportunity offered by drugging RNA
  • Outlining target selection and screening
  • Deeper dive into target structure

10:40 am Live Q&A

11:00 am Panel Discussion: Debating Between Strategies to Obtain Critical & Valuable Structural RNA Insights


For years, frontiers of the field have harnessed various innovative strategies to unlock structural
information to streamline the successful discovery and development of RNA targeted small molecules.
This panel will unite the leaders of the field to compare, contrast and debate between approaches and
successes in RNA structure-based drug design:

11:30 am | Morning Break & Structured Networking

Evaluating Chemical Based Strategies for Mapping Structure in RNA & Target Interactions

12:00 pm Comprehensive Mapping of RNA Ensembles in Living Cells


  • Discussing DANCE-MaP, a chemical probing strategy for directly mapping secondary and tertiary structure in dynamic RNA ensembles in cells
  • Discussing a large-scale structural switch in the human 7SK RNA that regulates transcription
  • Validating an ASO strategy for targeting 7SK to modulate transcription in cells

12:25 pm CryoEM of RNA Structures

  • Wah Chiu Wallenberg -
    Bienenstock Professor & Professor of Bioengineering,
    Microbiology & Immunology, Stanford University


  • Reviewing the advances in single particle cryoEM
  • Discussing what can be learnt from RNA structures at near atomic and subnanometer resolutions
  • Evaluating the prospect of imaging RNA in situ

12:50 pm Structure-Based Modelling & Simulation Tools for Identifying RNA-Targeting Small Molecules

  • Aaron Frank Assistant Professor of Biophysics, University of Michigan


  • Methods to mine RNA structures for ligandable cavities
  • Exploring RNA-specific regions of chemical space
  • QM-driven exploration of RNA-ligand interactions

1:15 pm Live Panel Q&A – Ask the Speakers your Burning Questions

  • Razvan Nutiu Head Life Science Technologies, Bayer
  • Aaron Frank Assistant Professor of Biophysics, University of Michigan
  • Wah Chiu Wallenberg -
    Bienenstock Professor & Professor of Bioengineering,
    Microbiology & Immunology, Stanford University
  • Anthony Mustoe Assistant Professor, Baylor College of Medicine

1:30 pm | Lunch Break & Networking

Splicing Modifiers: Route to Successful Discovery, Novel Chemical Space & Emerging Druggable Targets

2:30 pm Discovery of Risdiplam, Successes, Challenges & Future Directions

  • Ratni Hasane Distinguished Scientist, F. Hoffmann-La Roche


  • Reviewing the route to the successful discovery of Risdiplam, a first-in class splicing modulator for treatment of SMA
  • Discussing the successes and challenges with the discovery and development of Risdiplam
  • Exploring future directions including potential other targets and new chemical space for splicing modulators

2:45 pm Discovery & Development of PTC518, a Selective Huntingtin Lowering Splicing Modifier for the Treatment of Huntington’s Disease


  • Initiated Huntingtin lowering drug discovery program – Identified splicing modifiers and deciphered the mechanism of HTT lowering
  • Lead optimization efforts led to the discovery of PTC518
  • PTC518 data to be shared, demonstrating HTT lowering in cells and animals; POC demonstrated in humans

3:10 pm Drugging Alternative Splicing in Degenerative Disease & Cancer


  • Alternative pre-mRNA splicing is commonly associated with human disease
  • CLK/DYRK family kinases selectively modulate pre-mRNA splice junction selection by the spliceosome in both normal and diseased tissue contexts
  • Emerging highly specific small molecule CLK/DYRK inhibitors indicate these kinases are a druggable intervention point for alternative splicing associated diseases

3:35 pm Live Q&A & Panel Discussion: The Value of Splicing Modulators for Therapeutics & Future Applications


Targeting RNA splicing for disease application intervention has proven a valuable therapeutic intervention. It is now essential to review the clinical landscape of successes which exists with targeting the RNA spliceosome, consider various approaches and motifs being harnessed to target diseases beyond SMA and review future applicability of this modality.

4:15 pm Chair’s Closing Remarks & End of Conference Day One