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Gene Delivery and Gene Editing (Focus Group - GDGE)

(209) Advanced Functionalization Strategies for Lipid Nanoparticles: Expanding Delivery Beyond the Liver

Introduction:

Lipid nanoparticles (LNPs) are a leading platform for nucleic acid delivery, but their natural liver accumulation limits extrahepatic applications. Two strategies can address this: (i) modifying lipid composition to alter biodistribution and (ii) ligand-based surface functionalization for active targeting. While lipid modifications can influence organ tropism, they often lack precision and may lead to rapid clearance.In contrast, ligand decoration provides a versatile and modular platform for targeted delivery, independent of lipid composition constraints. 

Learning Objectives:

  • Understand how LNP surface functionalization expands delivery beyond the liver.
  • Explore the benefits of click chemistry for precise LNP functionalization.
  • Evaluate polymer end-group modifications as a scalable tool for targeted LNP delivery.