Conference Day 1
Wednesday 6th December 2023
8:00 am
Registration & Morning Coffee
8:50 am
Chair’s Opening Remarks
Examining Potency Assays to Accurately Interpret Biological Activity for Better Understanding of mRNA Drugs
9:00 am Considerations for mRNA Potency Assay Development for Gene Editing Products
Synopsis
- Overview of matrix approach for potency assay development
- Review of available regulatory guidances and expectations for phased-based approach to potency assay development
- Tools and considerations when measuring biological cascade of mRNA delivery in-vitro
- Challenges associated with potency assay development for gene editing products
9:30 am Roundtable: Trailblazing Potency Assays for a Fine-Tuned Approach to Meeting Specifications & Ensuring the Correct Response is Elicited
Synopsis
- How is the field aligning potency assays which are used to show that mRNA expresses the protein?
- Assessing which assays can be used to show whether the drug product elicits the correct response
- What is the most reliable combination of potency assays to carry out?
- Are robust potency assays needed for both drug substances and drug products?
10:45 am Morning Break & Speed Networking
Synopsis
- As the mRNA assay development community comes together, this valuable session will ensure you can reconnect with your peers in the room to make new and lasting connections. All attendees will have the opportunity to meet and network with their academic and industry colleagues!
Optimizing Bioanalytical Methods to Investigate Mechanism of Action & Determine Quantitation for Robust Translation
11:45 am Bioanalytical Strategies for Advancing mRNA Therapeutics
Synopsis
- Lipid Nanoparticle quantitation
- RT-qPCR vs bDNA for mRNA quantitation
- Approaches for protein and Anti-Protein Antibody quantitation
12:15 pm Illuminating the Mechanism of Action for LNP/mRNA-Based Vaccines by Employing Fluorescent Based Assays
Synopsis
- Fluorescent-based assays to explore the Mechanism of Action for LNP/mRNA-Based Vaccines
- Determining cellular LNP internalization, trafficking, and RNA cargo release by High content confocal microscopy
- Assess the translation of released mRNAs by fluorescent microscopy assays
12:45 pm
Networking Lunch Break
Shining a Spotlight on Advances in mRNA Quality Assessment Tools to Improve Drug Efficacy
1:45 pm Digital PCR for Analysis of RNA Quality Parameters
Synopsis
- Introducing dPCR technology for quality control analytics
- Analysis of identity and ratio of RNA mixtures
- Correlation of dPCR RNA quality data with classic capillary electrophoresis method
2:15 pm Development of Analytical Methods for Endless RNA (eRNA)
Synopsis
- Overview of endless RNA (eRNA)
- Key structural differences between mRNA and eRNA
- Development of a platform-based HPLC purity method
Increasing Assay Sensitivity to Accurately Assess mRNA Expression for Improved Safety & Efficacy
2:45 pm Methods to Assess Functional Protein Expression & Biodistribution for Development of Inhaled mRNA Therapeutics
Synopsis
- What methods can you use to ensure that functional protein is being expressed in vitro and in vivo preclinical models?
- Methods to confirm expression of your protein in the desired target cells/tissues
- Sensitive tools to look for expression of your protein in non-target organs