Conference Day One: Tuesday, April 15
7:15 Check-In & Coffee with Light Breakfast
8:15 am Chair’s Opening Remarks
Deep Diving into the Greatest Bottlenecks in the LNP Field to Improve Dosing, Scaling & Delivery for Advancing More LNPs Towards Approval
8:30 am Industry Leaders’ Panel Discussion: Overcoming the Challenges of Toxicity, Extrahepatic Delivery & Scaling-Up in LNP Development to Accelerate a Wider Range of Therapeutics towards the Clinic
Synopsis
• Uncovering the strategies for achieving specific, extrahepatic delivery with LNPs
• Delving into approaches for improving therapeutic index, reducing toxicity, and achieving better translatability for safer LNPs
• Highlighting methods for improving LNP and payload stability for better therapeutic development
• Achieving scalability of LNPs which can be used for extrahepatic purposes and unlocking the strategies for maintaining key product attributes
9:00 am Unlocking the Future of LNPs for Delivery of a Wide Range of Human Medicines
Synopsis
- Delving into the LNP landscape to unleash the latest and greatest LNPs in the field
- Addressing key bottlenecks which the LNP field is facing, including advancing LNPs for successful gene therapy delivery
- Uncovering the future potential for LNPs to deliver more human medicines
9:30 am “The PEG Dilemma” – A Solution
Synopsis
- Anaphylaxis to the approved mRNA vaccines is not IgE-mediated classic mast cell degranulation
- Anti-PEG antibodies – both IgG & IgM – are boosted by the approved mRNA vaccines
- Anti-PEG antibodies can result in severe infusion reactions, including fatal reactions
- The Serina LNP Laboratory has characterized a PEOZ-lipid that can replace the PEG-lipids and is immune silent in LNP formulations
10.00 Morning Break & Speed Networking
Synopsis
This session is your opportunity to get face-to-face with many of the brightest minds working in the LNP field and establish meaningful business relationships to pursue for the rest of the conference. Also, don’t forget to enjoy some refreshments before we split off into the 3 different tracks: Analytical Development & Characterization, Formulation & Delivery & Process Development & Manufacturing.
CHAIR - Anni Chang, Senior Scientist II, Excipients GSSD, US Pharmacopeia
Enhancing Ionizable Lipid & LNP Properties by Assessing Biodegradability, Surface Change & Size for More Heterogeneous LNP Development
11:00am: Optimizing Ionizable Lipid Development through Analysis of Biodegradability & Adjuvant Properties
- Optimizing the pKa of ionizable lipids for intramuscular mRNA vaccines
- Assessing the biodegradability of ionizable lipids
- Uncovering the adjuvant properties of ionizable lipids
Julien C. Senécal, Co-Founder, Azane Therapeutics
11:30am: Analytical Ultracentrifugation for Multi-Attribute LNP Characterization
- Analytical ultracentrifugation (AUC) is a classical, first-principle hydrodynamic and thermodynamic technique
- AUC provides a multi-attribute evaluation of LNPs ranging in size, composition, and heterogeneity
- LNP processing workflows can be guided by information obtained using AUC, and final products can be evaluated under cGMP
Alexander Yarawsky, Scientist, BioAnalysis LLC
12:00pm: Heightened Characterization Strategies of mRNA/ LNPs & the Power of NMR
- Heightened Characterization Roadmap for mRNA/LNP
Modalities - How NMR can support LNP Characterization
- A case study demonstrating how NMR can detect surface
LNP changes
Erika Jensen, Principal Scientist, Pfizer
12.30 Lunch Break & Networking
Improving Analytical Techniques for Assessing Quality, Stability & Lipid Properties Leading to Reduced Toxicity Challenges
1:30pm: Analytical Strategies to Understand LNP Quality & Stability
- Implementing MALs and HPLC into the analytical strategy for improved monitoring of LNPs
- Showcasing novel analytical methods have been developed to understand stability and predict shelf-life for targeted LNPs
- Evaluating assays that can be used to understand the degradation mechanisms of LNPs
Felipe Gazos Lopes, Associate Director, Analytical Development, Tessera Therapeutics
2:00pm: Platinum® Pro Protein Barcoding: Advancing Therapeutic mRNA-LNP Drug Development through Multiplexed Screening with Direct Protein-Level Detection
- Developing an effective mRNA therapeutic requires screening for candidates with the greatest protein output per delivered mRNA molecule
- Unfortunately, current approaches for assessing protein production rely on large arrayed screens or multiplexed pooled screens with an indirect (e.g. NGS-based) readout
- Quantum-Si has developed the Platinum® Pro protein sequencer and a novel protein barcoding application, the combination of which enables pooled multiplexed screening of mRNA candidates and delivery vehicles with a direct, protein-level readout in vitro and in vivo. This technology has the potential to streamline and enhance mRNA-LNP drug development
Joel McDade, Director, Technical Sales, Quantum-Si
2:30pm: Roundtable Discussion: Delving into the Physicochemical Properties of Lipids to Improve Product Performance & Shelf-Life
- What are the key strategies and methods for achieving deep characterization of lipids?
• How are we developing new lipids which stabilize products for long-term storage?
• Where are our gaps in knowledge when it comes to the lipid component of LNPs?
Magali Hickey, Independent Consultant
3:00pm: Afternoon Networking Break & Scientific Poster Session
Contribute to the conversation and share your cutting-edge research with your fellow LNP community to showcase your breakthrough discoveries to a vast audience of experts. Register your place to submit an abstract for review to showcase your poster*
Evaluating Excipient Quality & Analytical Techniques for Advancing Pure & High-Quality LNP Formulations to Deliver Editing Therapeutics & Beyond
4:00pm: Showcasing USP’s Perspective on New Frontiers in Excipient Quality & Development, including for Developing Safe & Compliant LNPs
- Reviewing USP’s perspective on excipient quality and development
- Discussing opportunities for incorporating lactide (or lactic acid) and glycolide (or glycolic acid) (LG) polymers in nanoparticles for improved delivery of therapeutics and vaccines
- Highlighting USP’s work on phospholipids and providing new updates
Peng Zhang, Principal Scientist, US Pharmacopeia
4:30pm: Cryo-TEM - A powerful multi-attribute characterization tool for LNP formulations
- Cryo Transmission Electron Microscopy (cryo-TEM) is a powerful, high-resolution tool that enables direct visualization of individual nanoparticles in their near-native state. It provides critical insights into homogeneity, size distribution, morphology, lamellarity, structural integrity, purity, payload distribution, and aggregation
- Learn how cryo-TEM, combined with advanced computational image analysis and deep learning algorithms, enables precise, quantitative characterization of LNP size and morphology
- Discover how direct visualization and image analysis tools enhance every stage of the LNP development pipeline - from formulation and process optimization to quality control and regulatory support
Mandy Janssen, Scientific Group Leader, Characterization, NIS
5:00pm: Advancing Analytical Method Development for LNP-based Prime Editing Therapeutics
- Analytical challenges in characterizing LNPs with multiple payloads of significantly different sizes and multiple distinct lipid compositions
- Key analytical methods for LNP characterization, with a focus on quantification, purity, and interpretation of data from orthogonal methods
- A case study of mRNA-lipid adduct analysis, including strategies to control lipid-mRNA adduct formation in LNPs
Yi Pu, Associate Director, Analytical Development, Prime Medicine
5:30pm: Chairs Closing Remarks
CHAIR - Anni Chang, Senior Scientist II, Excipients GSSD, US Pharmacopeia