Conference Day 2
8:50 am
Chair’s Opening Remarks
Leveraging Assays to Better Investigate Protein Expression & Function for Improved Therapeutic Effect
9:00 am Assay Development for Monitoring mRNA Functionality
Synopsis
- Overview of methods used to confirm that the target protein is functional.
- Exploring the relationship between mRNA integrity and functionality.
- Cell-based potency assay development for both DS and DP samples – case studie
9:30 am LC-MS Based Assay Development for Absolute Quantification of Protein Expression from mRNA Vaccine Candidates
Synopsis
- Overview of assays used for measuring protein expression from mRNA vaccines
- A robust and sensitive assay for mRNA expression using targeted proteomics
- Case study of determining mRNA expression from mRNA vaccines
10:00 am
Morning Networking Break
Assessing Bioanalytical Tools to Evaluate the Purity of mRNA Drug Substances for Reduced Immunogenic Side Effects
11:00 am Evaluate mRNA Immunogenicity Using Macrophage/Monocyte Cell Lines with CRISPR-Mediated Gene Knockout
Synopsis
- Immune cells provide a more sensitive readout of immunogenic side products in mRNA preps than non-immune cells
- CRISPR knockout of key innate immune sensors in immune cell lines
- Detect source of contaminants and their immune sensing pathways using such immune cell lines
11:30 am Roundtable: Reviewing Assays to Determine the Presence of dsRNA & Reduce Undesired Immunogenicity
Synopsis
- Which methods are currently used by the field to measure the presence of dsRNA?
- What types of controls and standards are being used in these assays to facilitate accurate and reliable quantitation?
- What approaches are being employed by the field to translate in vitro to in vivo response? Which biomarkers are most useful?
- What novel/next-generation methods are being developed to address existing challenges?
- What characteristics would define an ideal assay to measure immunogenicity?
- How is the field approaching the definition of impurity limits? Does this approach differ based on therapeutic area?
12:30 pm
Networking Lunch Break
Evaluating In Vitro Tools to Accurately Assess mRNA Drug Products for Improved Quality & Activity
1:30 pm Development of HPLC-CAD to Characterize Lipids in mRNA Related Drug Research & Delivery
Synopsis
- How to develop analytical method for characterizing lipids used in LNP mRNA Drug Research
- Lipid analysis utilizing HPLC-CAD
- Bio-analytical methods optimized for analysis of lipid delivery platforms
2:00 pm Microphysiological Systems for the Assessment of Activity of mRNA Therapeutics
Synopsis
- Overview of the current state of microphysiological systems (MPS)
- Opportunities for the use of MPS in drug development
- Challenges with the use of MPS in mRNA therapeutics
Optimize Manufacturing with Robust & Reproducible Assays to Enable Successful Scale-Up
2:30 pm Quantitatively Distinguishing Full (Correct) Length mRNA From Truncated or (Self-)Extended mRNAs
Synopsis
- Degradation, truncation, and self-primed extension occur during manufacturing
- Evaluation of these behaviors is critical to optimizing manufacturing approaches conditions
- New approach allows relatively easy assays
3:00 pm
Chairs Closing Remarks
3:15 pm
End of mRNA Assay Development Summit 2023
