Explore the Agenda

7:30 am Registration Opens & Morning Coffee

Workshop A

8:00 am Fast-Tracking Ligand & Lipid Selection with Robust Screening Strategies for Precise & Efficient Delivery

Senior Scientist, Formulation Development Group, Regeneron
Principal Scientist, Nucleic Acid Delivery, Biotech Startup

Building workflows to identify high performing targeting ligands and lipid components is essential for accelerating LNP development, however, ensuring speed is balanced with accuracy is vital. In this workshop, examine lipid type and ligand screening strategies and align these with complete targeted LNP formulations to enhance translation. Topics include:

  • Exploring how to efficiently screen vast libraries of ligands for specificity, transfection, and immunogenicity
  • Highlighting reliable strategies to identify lipids which will enable endosomal escape and extrahepatic delivery
  • Improving targeted LNP screening to balance throughput with quality metrics like selectivity and safety

10:00 am Morning Networking Break

Workshop B

10:30 am Achieving Targeted LNP Uniformity by Incorporating Advanced Analytics for Ligand Quantification & Stability

Scientist II, Nucleic Acid Delivery, Novo Nordisk
Vice President, Process Development, Editas Medicine
Senior Director, Advanced Drug Delivery, AstraZeneca

Targeted LNPs are continuing to advance and evolve to enable extrahepatic targeting. However, they are hindered by limited robust analytical strategies. During this workshop deep dive into reliable analytical methods for assessing ligand density, stability and aggregation for more consistent product quality. Topics include:

  • Leveraging comprehensive analytics to quantify ligand density to determine uniformity and minimize heterogeneity
  • Unlocking advanced analytical strategies to understand ligand stability, linker degradation and free ligands for safer targeted LNPs
  • Implementing imaging techniques for assessing aggregation, structural integrity and surface functionalization

12:30 pm Lunch & Networking

Workshop C

1:30 pm Engineering Specific & Selective LNPs for Brain & Muscle Targeting to Treat a Wider Array of Diseases

Director, Alnylam
Scientist, Early Formulation & Drug Profiling, Servier

As the brain and muscle remain key targets for LNP drug products, this workshop will explore the hurdles of the blood-brain barrier and broad muscle biodistribution. Gain insights and have proactive discussions into how to optimize LNP formulations to achieve efficient, specific and selective delivery to muscle and CNS targets. Topics include:

  • Examining how to fine-tune LNP characteristics to enable efficient delivery across extensive muscle areas without compromising immunogenicity
  • Uncovering ligand approaches for achieving selective LNP penetration across the blood–brain barrier
  • Evaluating how dual-targeting strategies may navigate endothelial barriers to deliver payloads into muscle and brain

3:30 pm Afternoon Networking Break

Workshop D

4:00 pm Solving the Immunogenicity Problem by Leveraging Innovative Analytics & Formulations to Enable Repeat Dosing

Associate Director, RNA Delivery Sciences, GlaxoSmithKline
Principal Scientist, Drug Product Development & Delivery, Johnson & Johnson

With immune responses, from anti-PEG and ligand antibodies to component-linked immunogenicity, impacting LNP dosing regimens and limiting clinical success. This workshop will delve into advanced analytics for understanding safety and evaluate innovative solutions to achieve effective repeat dosing.

Topics include:

  • Highlighting analytical strategies to pinpoint the cause of limited repeat dosing, are PEG, targeting ligands, ionizable lipids, or linkers are to blame?
  • Uncovering PEG alternatives that offer the most promise for preserving circulation time and transfection efficiency while reducing immunogenicity
  • Unleashing novel ionizable lipids, targeting ligands, and linker chemistries which degrade quickly, enhance endosomal escape and improve safety

6:00 pm End of Pre-Conference Workshop Day