8:30 am Morning Coffee & Light Breakfast
8:50 am Chair’s Opening Remarks
Improving RNA Splicing Modulation through Assay & Screening Strategies for Identifying more Effective, Selective & Potent Small Molecules
9:00 am Development of Biophysical Assays to Explore MOA of HTT Splicing Modulation by SM
Synopsis
- Development of MST and SPR assays to measure SM binding to HTT RNA
- SM-RNA Chair’s epitope mapping by NMR
- Development of biophysical assays to measure SM-mediated U1snRNP association
New company & New Data Badges
9:30 am Selectivity Profiling of RNA Splicing Modulators to Understand Efficacy & Off-Target Effects
Synopsis
- Progress toward a comprehensive understanding of selectivity as applied to small molecule splicing modulators
- Transcriptomic analysis of differential gene expression and splicing events
- Delineating relationships between activity, selectivity, and structure
New Data Badge
10:00 am Morning Break & Networking
Leveraging RNA Assays to Realize the True Mechanism of Action of RNA-Targeting Small Molecules to Validate Therapeutic Effect
11:00 am Panel Discussion – Revolutionizing RNA Assays to Characterize the Mechanism of Action for Advancing RNA Targeted Drug Development
Synopsis
- Comparing different assay and screening approaches which the field is currently using to better understand the mechanism of action
- How to optimize current assays to better understand the mechanism of action?
- How to utilize phenotypic methods to delve into the underlying mechanism?
12:00 pm Lunch & Networking
Uncovering Innovative Assays & Screens for Understanding the Potential of RNA Binding Proteins to Expand the Landscape of Treatable Targets
1:00 pm Chemical Proteomic Methods for Quantifying Protein-RNA Interactions in Cells
Synopsis
- Chemical proteomics can screen RNA-binding protein (RBP) composition and function in cells using a small molecule probe for rapid gel- and high-content-based detection
- Chemical proteomics can detect RNA binding activity on known and non-canonical RNA-binding domains with amino acid resolution
- RBP chemical proteomics offers a screening approach to determine protein off-target activity for RNA binding small molecules
New company & new data badge
1:30 pm Assay Technologies to Characterize RNA Binding Proteins & Their Interaction with Target RNA
Synopsis
Details to be revealed
2:00 pm Afternoon Break & Networking
3:00 pm Drug Discovery Efforts on RNA Modifying Proteins
Synopsis
- Modulation of RNA biology can be driven by inhibition of the proteins that use RNA as a substrate for enzymatic reactions, known as RNA modifying proteins (RMPs)
- RMPs represent a large class of novel targets for oncology and other disease indications that can be targeted with small molecule inhibitors
- Accent has developed potent and selective small molecule inhibitors for RMPs across protein target classes, including helicases, deaminases, nucleases and methyltransferases
New company & new data badge
3:30 pm Next-Generation Transcriptomic Strategy to Exploit Chemical Biology in RNA Regulator Targeting
Synopsis
- Utilizing transcriptome-wide methodologies, such as STAMP/eCLIP, commonly employed for identifying RNA-binding protein (RBP) binding sites, to profile RBP-RNA interactions within cells upon perturbation by small molecules
- Profiling small molecule libraries rapidly against RBPs enables the swift generation of comprehensive cellular datasets highlighting the selectivity against RNA targets and other RBPs
- Leveraging these binding profiles offers an avenue to gain deeper insights into the functional role of targeted RBPs in disease contexts
New company & new data badge