8:00 am Registration & Virtual Coffee

8:50 am Chairs Opening Remarks

Optimizing Medicinal Chemistry to Streamline Proof-of-Concept & Enhance Robust Translation

9:00 am Rational Design of Bioactive Small Molecules Targeting RNA

  • Matthew Disney Professor, Department of Chemistry & Neuroscience, The Scripps Research Institute & Scientific Founder of Expansion Therapeutics


• Exploring the utility of different compounds as both advancers of lead medicines and as chemical probes to advance understanding of previously unknown RNA biology
• Highlighting how the sequence-based small molecule rational design tool, dubbed Inforna, allows for targeted degradation of RNAs in cells and animals
• Understanding how these advances imply a necessary reassessment for
the future for SMIRNAs to deliver precision medicines

9:30 am Examining Methodologies, Chemical Biology & Structural Dynamics to Target mRNA with Small Molecules

  • Jen Petter Founder and Chief Scientific Officer , Arrakis Therapeutics


• Examining the facts for mRNA targeting
• Understanding RNA structure and dynamics
• Outline and discuss the importance of small molecules focus

10:00 am Translation Control Therapeutics: Discovery of Selective mRNA Translation Modulators

  • Yochi Slonim Chief Executive Officer & Co-Founder, Anima Biotech


• Discovery of small molecule drugs that selectively control mRNA translation in a novel target space
• Highlighting importance and utility of tissue selective drugs
• Navigating strong In Vivo data

10:30 am Virtual Speed Networking & Morning Break

Advancing Effective & Accurate Small Molecule Target Identification & Validation

11:30 am The Landscape Beyond SMA: Expanding the Drug Discovery Toolbox for the Treatment of Genetic Disorders

  • Matt Woll Vice President & Head of Chemistry , PTC Therapeutics


• Exploring how the discovery and clinical success of risdiplam in treating SMA
• Reimagining how the use of small molecules to influence pre-mRNA splicing become a fertile ground for drug discovery
• Understanding can non-canonical 5’ splice sites to shift alternative splicing and cause exon skipping or induce inclusion of pseudo-exons
• State of the art platform technologies can aid discovery of novel splicing modifiers for therapeutically relevant splicing events

12:00 pm Revealing Fragment-Based Screening Approaches Using NMR Demonstrates the Druggability of Biologically Active RNA


• Discuss how NMR-based fragment screening demonstrates the druggability of biologically active RNAs
• Outlining how SMN2 splice modulators can enhance pre-mRNA association and rescue SMA mice
• Evaluating the utility of fragment-screening library-NMR-based quality control

12:30 pm Chemical Libraries for Exploring & Developing RNA Targeted Chemical Probes


Obtaining and utilizing chemical libraries is the fundamental starting point for subsequent successful small molecule RNA targeting. Optimal chemical libraries will facilitate and accelerate discovery of RNA targeted therapeutics.
• Evidence suggests a privileged chemical space exists for biologically active RNA-targeted small molecules, but more data is needed
• RNA-biased library curation may lead to higher quality hits
• Assays to validate these libraries must be carefully considered

1:00 pm Session Details To Be Confirmed

1:10 pm Lunch & Virtual Networking

Unlocking the Facts: RNA Biological Mechanisms & How to Utilize for Viable Therapeutic Intervention

2:00 pm Inducing Reversal of Disease Phenotypes Using RNA-Targeting Proteins – Ensure Optimal & Successful Translation into the Clinic

  • Gene Yeo Professor , University of California San Diego


• RNA-Targeted proteins to modify RNA-mediated diseases
• Learning how to harness neurodegenerative models from IPSC cells model RNA defects
• Leveraging transcriptome-wide measurements confirm reversal of disease

2:30 pm RNA-Binding Proteins, RNA Processing & Gene Expression

  • Chris Burge Professor of Biology , Massachusetts Institute of Technology


• A new biophysical model can extract quantitative descriptions of protein-RNA binding from in vitro measurements
• Harnessing such models can enhance predictability of In Vivo binding and function, detect cooperative binding and the RNA accessibility footprint of an RNA-binding protein
• Discuss how pre-mRNA processing can impact transcription as well as other steps in gene expression

3:00 pm Networking & Virtual Poster Session

3:30 pm Panel Discussion: From Theory to Reality: How to Target RNA Biology with Small Molecule Approaches

  • Yochi Slonim Chief Executive Officer & Co-Founder, Anima Biotech
  • Jen Petter Founder and Chief Scientific Officer , Arrakis Therapeutics
  • Tim Luker Senior Director External Innovation, Eli Lilly & Company
  • Peggy Thompson Executive Director of Translational Medicine , eFFECTOR Therapeutics


• Explore successful methods and mechanisms for the targeting of RNA through small molecule compounds
• How can we utilize industry achievements to aid progression into the clinic?
• Identifying, evaluating, and overcoming key challenges to unleash the full therapeutic potential of the field
• Investigating the benefits and disease applications of effective RNA targeting

4:30 pm Session Details To Be Confirmed

  • Peggy Thompson Executive Director of Translational Medicine , eFFECTOR Therapeutics

5:00 pm Chair’s Closing Remarks & End of Day One