DAY ONE
8:00 am Check-in Opens & Light Breakfast
8:50 am Chair’s Opening Remarks
Supercharging RNA Structure Understanding & Target Discovery Using Primary, Secondary & Tertiary Structure Data for Improved Target Prediction
9:00 am Keynote Address: Unlocking RNA Dynamics for Targeted Drug Discovery
Synopsis
Details to be revealed
9:30 am Large-scale Discovery of RNA Tertiary Structures & Disease Relevant RNA Targets
Synopsis
- RNA motifs with complex 3D structures are promising targets for small molecule therapeutics but are still very hard to find
- A new technology that identifies RNA tertiary structures, transcriptome-wide, typically with direct functional roles, will be presented
- Thousands of targetable sites likely exist across the human transcriptome
10:00 am Leveraging Conformational Dynamics of lncRNAs to Identify Disease Relevant Targets & Selective Small Molecules
Synopsis
- Investigating conformational dynamics of lncRNAs to understand their structure further for enable better targeting
- Linking lncRNA target structure to disease to correctly identify disease relevant targets to ensure functional outcomes
- Identifying small molecules which will sufficiently restrict the RNA target structure to cause a therapeutic effect
10:30 am Morning Break & Speed Networking
Synopsis
This session is your opportunity to get face-to-face with many of the brightest minds working in the RNA Targeting Small Molecule field, and establish meaningful business relationships to pursue for the rest of the conference.
11:30 am NMR as a Tool for Structure Characterization of RNA Targets in Drug Discovery
Synopsis
- Using methods to predict and confirm RNA secondary structure
- Using NMR to better understand the structure of RNA in structure guided drug design
- Using NMR in combination with other methods to characterize drug binding on RNA targets
Exploiting Strategies for Accelerating Disease Relevant RNA Target Identification to Ensure Functional Outcomes
12:00 pm New Fluorogenic Toolkits for Diverse RNA-Targeted Drug Discovery Applications
Synopsis
- Real-time imaging tools to track RNA movement, measure RNA half-life, and assess RNA target engagement in cells
- HTS platforms for RNA degradation, splicing, aggregation, and structure-focused hit identification
12:10 pm Panel Discussion: Evaluating High-Value Direct RNA & Protein/RNA Complex Target Traits for Ensuring Efficient Binding & Therapeutic Effect for Accelerating Relevant Drug Discovery
Synopsis
- What are the key characteristics which validated RNA and RNA/protein targets share?
- How to conduct high resolution characterization of RNA targets including the different proteins and binding compounds present in the body
- How to determine RNA sub-targets which can be both bound by small molecule targets and cause therapeutic effects
1:00 pm Lunch & Networking
Discovering Drug-Like Small Molecules with Improved Functional Effects to Fast-Track More Potent Leads Candidates Towards Patients
2:00 pm Genetic Medicines: Discovering Small Molecules that Interdict RNA Function
Synopsis
- RNA targets are now broadly validated clinically and commercially, but many attractive targets remain out of reach to current modalities
- Arrakis has built a novel and highly differentiated platform for discovering drug-like small molecule ligands for RNA targets
- Arrakis platform has thus far produced at least two small molecule programs that clearly mimic the biological activity of oligonucleotide therapeutics
2:30 pm A Complete Workflow for the Identification & Characterization of Small Molecule Modulators of RNA
Synopsis
- A comprehensive workflow starting with approaches to identify small molecules that selectively bind an RNA of interest, determining the binding affinity of binders to target and off-target sequences , and characterization of the functional activity of those binders in cell-based proteomics assays will be presented
- Additional workflows to quantify and characterize covalent/irreversible adducts of RNA will be also be discussed
3:00 pm Afternoon Networking Break & Scientific Poster Session
Synopsis
Contribute to the conversation and share your cutting-edge research with your fellow RNA[1]targeting community to showcase your breakthrough discoveries to a vast audience of experts. Register your place to submit an abstract for review to showcase your poster*
*Please visit the website for the T&Cs for presenting a poster
Uncovering Novel Targets on Proteins, Enzymes & RNA/Protein Complexes to Improve Targeting of Disease-Causing RNA for More Efficacious Treatments
4:00 pm Leveraging Strategies to Drug RNA-Protein Complexes with Small Molecule
Synopsis
- Identifying disease relevant RNA-protein targets for previously intractable diseases
- Developing screening assay strategies for the discovery of novel small molecule splicing modulators
- Optimizing toward more potent and efficacious small molecule therapeutics: moving from early discovery to preclinical development for more streamlined translation towards the clinic
4:30 pm RNA-Modifying Enzyme Inhibitors as Synthetic Lethal Cancer Therapeutics
Synopsis
- Expanding the potential impact of helicase inhibitors beyond MSI-H cancers with DHX9-targeted therapeutics
- Validation and drug discovery enablement of RNA modifying nuclease target XRN1 in tumors with elevated interferon signaling