Explore the Agenda
8:30 am Morning Coffee & Registration
8:55 am Chairs Opening Remarks
Delivery & LNP Functionality in mRNA Therapeutics to Strengthen Characterization, Precision & Standardization
9:00 am Leveraging Single Particle Characterization to Guide LNP Pre-Formulation for Emerging mRNA Modalities
- Applying single particle analysis to understand heterogeneity in LNP size, charge, and encapsulation efficiency, and why this matters for reproducibility
- Using particle-level insights to de-risk pre-formulation decisions in novel modalities (e.g., self-amplifying RNA, circular RNA, gene editing payloads)
- Linking particle attributes to critical quality attributes (CQAs) such as potency, immunogenicity, and stability to support early stage development
9:30 am Optimizing LNP Characterization & Storage to Enable Long-Term Stability in mRNA Therapeutics
- Assessing how lyophilization impacts LNP heterogeneity and functionality, and approaches to reduce variability
- Understanding how formulation variables and lipid interactions drive aggregation and stability loss during storage
- Comparing cold-chain and room-temperature strategies to evaluate trade-offs in potency, durability, and feasibility
- Establishing standardized characterization protocols to improve reproducibility, regulatory confidence, and global distribution readiness
10:00 am Standardizing Potency & LNP Characterization for In-Vivo Gene Editing mRNA
- Define phase-appropriate potency strategies for editor mRNA payloads to generate data regulators will trust across IND through late stage
- Align LNP single-particle and bulk characterization readouts to improve cross-site comparability and decision-making
- Prioritize release and stability assays that balance scientific depth with QC practicality to accelerate clinical readiness
10:30 am Advancing mRNA Stability Studies to Improve Assay Comparability & Regulatory Confidence
- Investigating how mRNA performs at different temperatures to strengthen reliability of stability studies
- Developing analytical strategies to detect degradation pathways and aggregation events to improve assay comparability
- Embedding robust stability assays into development workflows to reduce variability and protect submissions from regulatory challenge
11:00 am Morning Break
Analytical Development & Quality Control for Novel RNA Modalities to Overcome ModalitySpecific Characterization & Functionality Challenges
11:30 am Case Study: Driving Analytical Robustness & CMC Readiness for Efficient Development of Novel RNA Modalities
- Navigating unique analytical and regulatory challenges when developing novel RNA-based vaccines
- Designing phase-appropriate QC and analytical strategies that can adapt as novel modalities progress toward GMP readiness
- How learnings from novel platforms can inform broader industry best practices in analytical robustness and CMC submissions
12:00 pm Measuring Replication Competence, Integrity & dsRNA in saRNA to Strengthen Potency, Reduce Immunogenicity & Build Regulatory Confidence
- Characterizing saRNA replication intermediates and byproducts to understand their impact on potency and safety
- Developing sensitive assays to monitor integrity and control dsRNA levels to reduce unwanted immunogenicity
- Translating early saRNA assays into QC-ready methods to build regulator confidence in this emerging modality
12:30 pm Integrating Circular RNA & Virus-Like Particle Platforms: Building Analytical & CMC Frameworks for Next-Generation RNA Therapeutics
-  Establishing quality and analytical control strategies to support circular RNA–VLP platform development from early R&D through scalable GMP manufacturing
- Characterizing encapsulation efficiency and RNA integrity across distinct delivery modalities to ensure comparability, consistency, and safety
- Designing robust CMC pathways that enable regulatory readiness and streamline technology transfer for novel RNA-based vaccine and therapeutic platforms
1:00 pm Lunch Break
2:00 pm Adapting Analytical & QC Strategies for Emerging RNA Modalities to Ensure Robust Characterization & Release
-  How evolving RNA modalities like circular and self-amplifying RNA are reshaping analytical design and QC readiness
- Translating flexible R&D assays into GMP-compliant QC methods without compromising analytical sensitivity
- Balancing innovation and standardization to build adaptable yet regulator-ready analytical frameworks for next-generation RNA therapeutics
2:30 pm Building Characterization Assays to Support Early-Stage saRNA Development and QC Alignment
- Establishing characterization assays that define saRNA product quality and inform key CMC decisions in early development.
- Ensuring analytical consistency and data integrity when partnering with CROs and CDMOs for assay execution and method transfer.