DAY TWO
Thursday, February 27, 2025
8:00 am Check-In & Morning Coffee
8:50 am Chair’s Opening Remarks
Bolstering the Industries-Leading Delivery Vehicle: Optimizing LNP Formulation & Characterization to Enhance Delivery Efficacy & mRNA Payload
9:00 am Building an Analytical Platform for mRNA & LNP to Streamline Separation & Characterization of mRNA-LNP Formulations
Synopsis
- Reviewing the advantages of analytical platform using multi-detector asymmetric flow filed-flow fractionation in separation and characterization of mRNA-LNP
- Discussing the development of separation strategy for mRNA and LNP using asymmetric flow field-flow fractionation
- Exploring potentials of MD-AF4 analytical platform in LNP formulation optimization by leveraging online sizing, morphology and encapsulation efficiency characterization
9:30 am Unlocking Platform Technology Designation to Fast-Track Analytical Development of mRNA Therapeutics & Vaccines into the Clinic & Beyond
Synopsis
- Understanding the regulatory basis for definition of a platform technology
- Outlining the advantages of having a analytical platform for mRNA technologies
- Leveraging learnings and prior knowledge from an established platform for a new submission
10:00 am mRNA Analytics: Last chance for Platform Methods?
Synopsis
- Exploring the current state of art for mRNA Analytics
- Considerations on advantages and limitations of platform approach
- Reviewing a platform method case study
10:30 am Morning Break
Pioneering Characterization Methods to Profile the Identity of mRNA Medicines for Faster mRNA Drug Approvals
11:30 am Leveraging Next-Generation Sequencing for mRNA Vaccine Characterization & Quality Control
Synopsis
- Understanding how NGS is being used, and for which quality aspects, for mRNA vaccine development and manufacturing
- Addressing current challenges using NGS for mRNA vaccine characterization and quality control
- Progressing analytical validation of NGS to ensure vaccine identity and fidelity
12:00 pm mRNA Analytical Challenges & NGS Based Solutions
Synopsis
- mRNA CQA challenges and solutions in Analytical Development
- Current NGS approaches for mRNA Analytical Development
- Emerging technologies and applications to address mRNA medicine complexity
12:30 pm Elevating QC Standards in mRNA Manufacturing: A Rapid, Sensitive, & Simple Assay for dsRNA Detection
Synopsis
- Detecting dsRNA in mRNA manufacturing: a persistent QC challenge
- Leveraging engineered luciferase for rapid and sensitive dsRNA detection
- Delivering a simple, robust assay for efficient QC workflows
12:40 pm Roundtable Discussion: Leveraging Next-Generation Sequencing to Characterize the Identity & Fidelity of mRNA Products
Synopsis
- Which sequencing techniques to deploy to qualitatively and quantitatively study the transcriptome and ensure sample purity and identity?
- Qualifying sequencing workflows and instruments to comply with GMP and ensure the appropriate scale-up
- What is the phase-appropriate manufacturing cadence for performing sequencing?
1:40 pm Lunch & Networking Break
Examining Alternative & Emerging Delivery Vehicles to Fuel the Clinical Progression of Novel mRNA Loaded Nanomedicines with Improved Safety & Efficacy
2:40 pm Panel Discussion: Showcasing Strides in Cell-Targeted mRNA Delivery to Improve Safety & Elicit Stronger & Durable Immune Responses
Synopsis
- What is the need for targeted delivery and what are the current approaches being deployed?
- Assessing the specificity of targeted delivery systems, such as targeting ligands or antibodies conjugated to carriers, and measuring ligand-receptor interactions
- What additional analytical methods need to be developed and qualified to permit their safe progression?
3:25 pm Investigating the Development & Optimization of Novel Nanomedicines for mRNA Delivery
Synopsis
- What are the most effective strategies for developing and optimizing carriers to advance mRNA therapeutics?
- Characterizing novel mRNA delivery systems to evaluate their performance under varying physiological conditions
- Discussing how they differ from LNPs
*New Data*