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 Suitability of Physicochemical Methods for Release Testing of mRNA-LNP Drug Products
Synopsis
• Surveying analytical procedures used to measure physiochemical quality attributes of mRNA Vaccines and therapeutics
• Regulatory expectations for physiochemical methods used for identity, purity/integrity, and concentration/content of mRNA-LNP products
• Identifying and evaluating critical validation parameters to validate physiochemical methods for mRNA-LNP products
9:30 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
10:00 am Advancing Analytical Strategies for Characterizing mRNA Encapsulation Efficiency in LNP Formulations
Synopsis
- Using Ribogreen as a readout to measure encapsulation and mRNA concentration
- Optimizing a Ribogreen method to facilitate QC transfers
- Controlling method variables to determine robustness of the assay
*New Data*
10:30 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
11:00 am Morning Break & Poster Session
Synopsis
As the method development, validation and transfer of mRNA medicines continues to progress, it is more important than ever to collaborate and learn with your peers, as we continue to advance these therapies to patients in need. Join your colleagues to share your latest data and have the first look into what your peers are working on!
Pioneering Characterization Methods to Profile the Identity of mRNA Medicines for Faster mRNA Drug Approvals
12:00 pm 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
12:30 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
1:00 pm Session Reserved for Eclipsebio
1:10 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 are the current approaches being deployed for targeted delivery, and how have these improved safety profiles?
- 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?
3:40 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*