WORKSHOP DAY
8:00 am Check-in Opens & Light Breakfast
WORKSHOP A – 9:00am
Leveraging Innovative Computational & High Throughput Screening Approaches to Accurately Identify Selective RNA Binders for Improved Therapeutic Effects
Synopsis
To carefully select the most promising small molecules and RNA targets, many new and innovative high throughput screening and computational based approaches have been developed. However, the field is currently plagued by limited sensitive and robust methods, hence, in this session the community will uncover latest strategies for identifying selective and specific small molecules.
This session will focus on:
- Deep diving into novel computational models and screening strategies to identify disease-relevant RNA targets and selective RNA binders
- Improving synergy between different computational and screening strategies to accelerate RNA target discovery
- Uncovering new methodologies which have been developed which are more robust and reproducible
11:00 am Morning Break & Networking
WORKSHOP B – 12:00pm
Achieving Good Small Molecule Drugs by Delving into Optimal Chemical Features to Fast-Track Hit Discovery of Relevant Small Molecules
Synopsis
There’s currently only a small variety of small molecules which have bound to RNA and shown functional effects. Hence, there’s only a small number of relevant small molecule drugs to compare your drug discovery too. To ensure you are advancing the right small molecules, it is vital to understand what pharmacological features to look out for to ensure they can be developed into drugs later in the drug development pipeline.
This session will focus on:
- What is the pharmacology of a good small molecule drug targeting RNA?
- Identifying chemical features which make small molecules good drugs, such as understanding the ideal permeability and stickiness.
- Understanding the key pharmacological properties which small molecule drugs need to enable optimization of algorithms and models.
- How does a good small molecule interact with the RNA target, both in a cell and in a disease setting?
2:00 pm Lunch & Networking
WORKSHOP C – 03:00pm
Uncovering RNA Targets Beyond Splicing Modifiers to Expand the Potential of RNA Targeting for Treating More Diseases
Synopsis
There are many novel RNA targets in addition to splice sites, such as long non-coding RNAs which have highly structured motifs, meaning they are good targets. Hence, understanding the new and innovative areas to expand this field is exciting and can increase the potential of treating more diseases.
This session will focus on:
- Uncovering the involvement of long non-coding RNAs in disease and understanding if they are suitable to target by small molecules
- Identifying new disease relevant RNA sequences, beyond splice sites
- Highlighting RNA targets for ASOs which may be relevant for targeting by small molecule drugs for better oral bioavailability
- How can targeting repeat RNAs lead to functional consequences?