8:00 am Check-in & Morning Coffee
Workshop A
9:00 am Visualizing RNA Structure & Linking It to Function to More Reliably Discover Functional Binders
Synopsis
Understanding RNA structure is essential for decoding its functional roles in disease and for identifying high-affinity, selective small molecule binders. Yet, RNA’s inherent flexibility, context-dependent conformations, and limited structural data make this a formidable challenge. This workshop explores cutting-edge experimental and computational techniques—from NMR and Cryo-EM to in silicon modeling—that are advancing our ability to visualize RNA in both isolated and cellular contexts.
Attendees will cover:
• Utilizing NMR spectroscopy to map RNA-ligand interactions, monitor conformational dynamics and screen binders, offering atomic-resolution insights for RNA-targeted drug discovery
• Advancing RNA-targeted screening by integrating AI-driven small molecule discovery, high-throughput screening, and structural data to enhance accuracy, reproducibility and structure-based docking
• Understanding structural flexibility and cellular context to distinguish biologically meaningful RNA conformations and improve binder discovery strategies
12:00 pm Lunch Break & Networking
Workshop B
1:00 pm Emerging Modalities Beyond Splice Modulation: Harnessing Covalent Binders, Proximity-Inducing Compounds & lncRNA Targets to Discover Functional RNA Binders
Synopsis
While splice modulation has paved the way for RNA-targeted therapeutics, the next frontier lies in unlocking the therapeutic potential of directly targeting RNA. This workshop explores innovative strategies exploring covalently binding RNA, bivalent compounds inducing proximity between RNA and effector molecules, and the untapped opportunity of targeting long non-coding RNAs.
Attendees will cover:
• Exploring covalent binders and proximity-inducing molecules to introduce functional outcomes into RNA targeted strategies
• Developing and validating a novel LC/MS-based chromatography method to quantify covalent reactivity with nucleotides/nucleobases, enabling comparison of RNA- vs protein-targeted electrophiles
• Developing lncRNA targeted small molecules to inhibit aggressive cancer phenotypes similarly to knock-out models