Pre-Conference Workshop Day

Tuesday, December 8, 2020

9:00 - Workshop A : RNA Modelling: Secondary & Tertiary Structure of RNA – How Can We Leverage Structural Insights?

Uncertainty and lack of clarity for structure and function of RNA remain a caveat for progression of RNA-targeted therapies into the clinic. With a better understanding of RNA folding and three-dimensional structures, the pool for disease targets will increase exponentially.

Join this workshop to:
• Explore computational predictions of RNA structure
• Discover how to effectively utilize chemical probing data to predict
RNA folding
• Evaluate the advantages of using three-dimensional structures to image
tangible pockets for ligand binding

Workshop Leaders:

RNA Targeted Drug Discovery Speakers

11:00 - Morning Refreshments & Networking

12:00 - Workshop B: Harnessing Advanced Screening Approaches for RNA Targeting Small Molecule Lead Discovery

Despite the success in the recent discovery of several RNA-targeting small molecules, our knowledge of the RNA-targeting chemistry space is still very limited. To fully unlock the therapeutic potentials of targeting RNA with small molecules, novel screening approaches for RNA-targeting lead discovery and optimization would need to be developed and explored. Advanced techniques such as NMR, reporter-based assays, and TR-FRET binding assays are just some of the examples of approaches that could be readily adapted and applied for RNA-targeting small molecule lead discovery and optimization. In this workshop, we will review emergent screening approaches in the field and discuss the challenges and opportunities to maximize the success of RNA-targeting drug discovery.

• Discussions on small molecule lead generation
• How can we utilize this approach to streamline RNA target discovery?
• Exploring promising data from recent NMR data

Workshop Leaders:

RNA Targeted Drug Discovery Speakers

2:00 - Afternoon Refreshments & Networking

3:00 - Workshop C: Discovering Cross Learnings Between RNA-Targeted Small Molecule & mRNA Delivery Vehicle Development

Identifying small molecules that interact with specific endogenous RNA targets is the common goal of companies working in the RNA-targeted drug development space. Similarly, identifying small molecules capable of packaging exogenous RNAs for efficient in vivo delivery is a common goal of companies working in the mRNA therapeutics and gene editing space. A deep dive into the general principles of RNA-targeted small molecule drug design and RNA delivery vehicle design can reveal valuable cross-learnings to aid optimization of both therapeutic types.

• Outline and consider the general principles for small molecules to effectively interact with RNA
• Discuss how to engineer small molecules for selective and non-selective binding to mRNA
• Consider RNA-targeted library design to optimize drug discovery

Workshop Leaders:

RNA Targeted Drug Discovery Speakers