Conference Day Two
Thursday 20th November 2025
8:30 am Check-In & Light Breakfast
8:50 am Chair’s Opening Remarks
Advancing Multivalent LNPs Through Improved Separation & Purity Testing to Accelerate Clinical Translation & Approval
9:00 am Uncovering QC-Friendly Methods for Accurately Separating Multivalent LNPs to Advance Next Generation Drug Products Towards Approval
Synopsis
- Identifying robust methods to disrupt lipid nanoparticles (LNPs) to access and analyze encapsulated nucleic acid payloads. Applying platform analytical technologies to accelerate the transition from development to clinical application
- Highlighting challenges encountered during the testing of multivalent LNP formulations, particularly those containing varying ratios of multiple payloads
- Leveraging QC-friendly, HPLC-based methods to differentiate and quantify closely sized nucleic acid payloads within multivalent LNPs, ensuring suitability for regulated environments
NEW DATA
9:30 am Leveraging the Advanced Analytical Tools for the Characterization of Multi-Components Prime Editing LNP Drug Product: Case Studies
Synopsis
- LC-CAD-MS for multiple attributes lipid analysis (ID, content, ratio, purity, impurity ID)
- IPRP-HPLC for multiple attributes RNA analysis (RNA ratio, purity, adduct formation)
- Orthogonal methods and case studies
NEW DATA
10:00 am Morning Break & Networking
Deep Diving into Potency & Biodistribution to Enhance In Vivo Translation & Accelerate LNP Drug Products into the Clinic
11:00 am Advanced Analytical Tools for Understanding Gaps Between Physicochemical Property & In Vivo Potency
Synopsis
- Physicochemical properties are not always aligned with in vivo potency
- Advanced analytical tools are crucial for understanding key physicochemical properties of LNPs
- Elucidating the Mechanism of Action (MoA) is essential for comprehending in vivo potency
NEW DATA
11:30 am Novel Bioanalytical Methods to Determine LNP Biodistribution & Stability
Synopsis
- Determination of lipid nanoparticle (LNP) pharmacokinetics is important for understanding targeted and off-target distribution, but has been limited by the lack of generally applicable, bioanalytical methods
- We have recently developed a novel bioanalytical method to quantify DSPC, a common lipid component lipidic formulations, including LNPs
- This presentation will showcase DSPC pharmacokinetic data for multiple lipidic formulations, which, combined with traditional methods address formulation biodistribution and stability
NEW DATA, NEW COMPANY
12:00 pm Lunch & Networking
Enhancing In Vitro Assessment & Novel Excipient Development by Delving into Advanced Assays & Emerging Standards for Safer LNP Therapeutics
1:00 pm Predictive In Vitro Profiling of LNP-Induced Innate Immune Response Using an iPSC-Derived Model
Synopsis
- An unlimited, standardized source that shrinks the bench-to-bedside gap: Enabling high-throughput in-vitro screens of LNP inflammatory responses, speeding translational development
- Deliver stronger, more reproducible cytokine readouts than primary cells: Clearer signals reveal formulation–response relationships that traditional assays miss
- Mirror in-vivo cytokine signatures: allowing precise rank-ordering of LNPs innate immune response
NEW DATA
1:30 pm Assays for Elucidating Anti-PEG Antibodies & Formulations to Overcome Anti-PEG Immunity
Synopsis
- Substantial variations in assay methods and sensitivities can limit conclusions drawn about anti-PEG immunity
- There are pre-existing anti-PEG antibodies, which makes well-characterized and standardizable assays an important part of rigorous evaluation of PEG immunogenicity
- PEG-immunogenicity can be altered by formulation
NEW DATA, NEW COMPANY
2:00 pm USP Emerging Standards & Iterative Approaches to Novel Excipient Development
Synopsis
- Analyze the regulatory and safety assessment challenges that limit the use of novel excipients, focusing on implications for early-phase drug development
- Explore USP’s initiatives to advance novel excipient development, including the creation of Novel Excipients Expert Panel and revisions to General Chapter <1074> Excipient Biological Safety Evaluation Guidelines
- Gain insights into USP’s iterative approaches and emerging standards for novel excipient standard development and stakeholder engagement, highlighting their role in addressing evolving quality needs in pharmaceutical development
NEW COMPANY
2:30 pm Afternoon Break & Networking
Optimizing LNP Development by Correlating Analytical Insights With Process Performance for Higher Quality, Scalable LNP Products
3:30 pm Fireside Chat: Bridging the Gap from R&D to CMC by Advancing Robust Analytical Strategies for Scalable LNP Development
Synopsis
- How do analytical and process requirements evolve when transitioning from R&D to CMC-scale development?
- What are best practices for ensuring assay reproducibility and robustness during tech transfer?
- How can analytical strategies developed during early research be made practical and scalable for CMC teams?
- How can data from early-stage studies be leveraged to inform and de-risk CMC method development?
4:00 pm Aligning Analytical Insights with Process Performance for Improving Process Optimization for Higher Quality & More Stable LNPs
Synopsis
- Mapping CQAs to specific process parameters to understand how manufacturing conditions impact product quality
- Importance of analytical methods for both in process and terminal characterization
- Correlating heightened characterization methods to process performance to improve process optimization
NEW COMPANY