8:00 am Morning Networking Coffee

8:50 am Chair’s Opening Remarks

Unraveling RNA Degradation to Expand the Druggable Genome

9:00 am Sequence-Based Design of Small Molecule RNA Degraders

  • Matthew Disney Professor & Chair; Department of Chemistry, The Scripps Research Institute


  • How to find small molecules that bind to RNA
  • How to assess target engagement in cells
  • How to convert to a degrader

9:30 am Targeting RNA Through Induced Proximity

  • Ryan Potts Executive Director & Head - Induced Proximity Platform, Amgen


  • Discuss the concept of induced proximity
  • How can induced proximity be utilized for targeting RNA
  • Evaluating RNA effectors for RNATACs

10:00 am Current Landscape & Future Look at RNA Targeted Small Molecules

  • Ioana Panait Lead Analyst – RNA, Beacon Targeted Therapies


  • An overview of the current RNA landscape
  • A landscape analysis of RNA-targeted small molecules
  • A future look into RNA-targeted small molecules landscape

10:30 am Discovery of RNA Targeting Small Molecules Using Novel Screening Technology


  • SHAPE-MaP is a powerful method for fast RNA structure and binding site verification
  • Multiple screening technologies including DEL and FBDD are feasible to screen RNA target
  • Various orthogonal assays can be used to measure RNA small molecule interaction

10:45 am Morning Networking Break

Evolving Platform Technologies to Optimize Biologically Relevant RNA-related Events

11:15 am Next Generation Splicing Modulators

  • Scott Barraza Discovery Chemistry Group Leader, PTC Therapeutics


  • The approval of Risdiplam for the treatment of SMA validated the approach of selectively modulating splicing with small molecules
  • PTC has developed a comprehensive discovery platform to identify novel splicing modulators for the treatment of diseases with unmet needs
  • Novel splicing modulators are being optimized for recently identified disease-relevant splicing events

11:45 am mRNA Lightning Platform – Discovery of Small Molecule mRNA Drugs & Their Mechanisms of Action


  • Target space opportunity: an overview of Anima’s mRNA biology modulators
  • mRNA Lightning platform combines high scale phenotypic screening with MOAi technology using AI to elucidate the mechanism of action of active molecules
  • Anima’s Lightning software oversees the whole discovery process, from hit generation to AI-driven MOA elucidation

12:15 pm Networking Lunch Break

Afternoon Chair

Emerging Non-Coding RNA-Targeted Small Molecules to Overcome Therapeutics Hurdles

1:15 pm Developing Small Molecule Therapeutics Targeting lncRNAs


  • LncRNAs play an important role in human disease
  • LncRNAs exert their functions through interaction with proteins
  • NextRNA is developing small molecules inhibiting RNA-protein interaction

1:45 pm Unraveling Novel Small Molecules for Neuroprotective microRNA Activation in Alzheimer’s Disease

  • Anna Krichevsky Associate Professor, Brigham and Women’s Hospital & Harvard Medical School


  • Diverse non-coding RNAs regulate all biological functions in a cell and expand the landscape of therapeutic targets in the CNS pathologies
  • miR-132 is the top neuroprotective miRNA associated with pathology in Alzheimer’s disease and other tauopathies
  • The first screen on human neurons identified small molecule activators of miR-132-mediated neuroprotection

2:15 pm Delineating Non-Coding RNA Therapeutics Avenues


  • Bioavailability and target interactions
  • Targetable sequence heterogeneity
  • Reverse translational consideration

2:45 pm Exploring the Undiscovered Country of RNA Drug Targets: Finding Bioactive Ligands Against Xist RNA with Affinity-Selection MS Screening


  • Affinity-selection MS screening can find binders to a wide variety of RNA targets
  • RNA-binding ligands can then be profiled to determine structural features that bias them to bind to RNA targets
  • Biophysical and cell-based techniques have been applied to characterize activities of novel binders to be bacterial and mammalian non-coding RNA targets

3:15 pm Afternoon Networking Break

Optimizing Selectivity & Specificity to Enable Potent Small Molecules Targeting RNA

3:45 pm Panel Discussion – Modelling Selectivity – Can we Develop a Model to Map Selectivity Binding?


  • How to define selectivity and specificity?
  • How to improve efficient and potent cell, tissue, and disease selectivity?
  • Can we construct a suitable model for selectivity?
  • How to guarantee selective and specific binding of your RNA targeting small molecules?

4:15 pm Discovery of Novel Oncoribosome Selective Protein Translational Inhibitors via Synthetic Modified Macrolides


  • The TURBO-ZM platform represents an innovative leap in synthetic chemistry to generate Ribosomal Modulating Agents (RMAs) selective for the oncoribosome
  • RMAs spare normal cells while targeting a subset of cancers addicted to oncoribosome-driven protein translation
  • Proteomic analysis shows extreme susceptibility to protein translation inhibition of highly positively charged proteins that participate in translation initiation and ribogenesis

4:45 pm Chair’s Closing Remarks