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 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
11:00 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.
Exploiting Strategies for Accelerating Disease Relevant RNA Target Identification to Ensure Functional Outcomes
12:00 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 Session Reserved for Momentum Biotechnologies
3:00 pm RNA Binding Small Molecules with Functional Effect
Synopsis
Details to be revealed
3:30 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