9:00 am Morning Coffee

9:25 am Chair’s Opening Remarks

Spotlighting Novel Approaches of Direct RNA Binders to Treat Disease with Unmet Need

9:30 am RNA Degradation – Novel Approaches to Targeting RNA with Small Molecules

  • Matt Disney Institute Professor & Chair of Chemistry, The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology


  • Is RNA degradation approach the future of RNA-targeted drug discovery?
  • Identifying small molecules that bind to RNA and understanding their functionality as part of their drug design
  • What are the current tools being used to study knock-down effects?

10:00 am Allele-Selective Targeting of HTT RNA with Small Molecules


  • Selection of the RNA target by leveraging structural tools
  • Development of methods for screening and characterizing RNA-binding small molecules
  • Identification of small molecules that interact with HTT RNA

10:30 am Morning Break & Scientific Poster Session

11:30 am AI in mRNA Biology: Small Molecule Drug & Target Discovery

  • Gal Gur Vice President - Business Development, Anima Biotech


  • Visualizing the complete mRNA life cycle from multiple disease models using highcontent mRNA imaging technologies
  • Decoding the mRNA biology underlying disease phenotypes with AI technology using mRNA image neural networks, knowledge graph, large language model, and its copilot
  • Examples and case studies in oncology

Turbocharge Screening Tools & AI to Improve Translatability from In Vitro to In Vivo

12:00 pm Magnetic Force Spectroscopy – a Novel Approach to Improve the Hit-to-Lead & Lead Optimization Phases of RNA-Targeted Small-Molecule Drug Discovery


  • Overview of MAGNA technology which employs magnetic force spectroscopy to analyze biomolecular interactions with single molecule resolution
  • Acquisition of kinetic and structural information about small molecule binding to RNA targets

12:30 pm Lunch & Networking Break

Successfully Develop Highly Specific Small Molecule Inhibitors in Oncology for Advanced Translation

1:30 pm Discussing Novel Best-in-Class Inhibitors of RNA Modifying Enzymes in Oncology for Preclinical Progression


  • Targeting different RNA modifying enzymes using in-house established discovery platform to identify and develop novel highly specific small molecule inhibitors for advanced translation and progression into clinic
  • Exploring case studies of preclinical programs of the best, first in class molecules in oncology with the goal of providing novel therapeutic approaches for patients with unmet medical need

2:00 pm Roundtable: Discussing the Different Strategies & Approaches of Translating Binding & Functionality Effects to the Clinic


  • Understanding tonality and the evolution of novel small molecule inhibitors for improved efficacy in in vivo models
  • What are the current strategies and approaches to successfully translate functionality into the clinic and how can these be improved?

3:00 pm DHX9 Inhibitors as Novel Therapeutic Modalities in MSI-High Cancers


  • DHX9 is an RNA helicase with important roles in replication, transcription, translation, RNA splicing and RNA processing
  • Microsatellite instable high (MSI-high) cancer cell lines are selectively dependent on DHX9 activity
  • Potent inhibitors of DHX9 have been identified that show antiproliferative activity in MSIhigh cancer models in vitro and in vivo

3:30 pm Chair’s Closing Remarks

4:15 pm End of the 6th RNA-Targeted Drug Discovery & Development Summit