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Formulation and Delivery
Keynote

Keynote: Lipid Nanoparticles-Based mRNA Delivery for the Treatment of Genetic Diseases

Tuesday, November 11, 2025
1:30 PM - 2:30 PM CT
Location:Stars at Night B2/B3

Formulation and Delivery

In this presentation, Dr. Sahay will discuss cutting-edge strategies to navigate the endosomal space, enabling efficient release of RNA cargo into the cytoplasm. He will highlight recent advances in targeting therapeutics to the retina and lungs, showcasing how these approaches can address blinding retinal disorders and severe pulmonary diseases.

Learning Objectives:

  • Explain the biological barriers that limit efficient delivery of nucleic acids and proteins to intracellular targets, with a focus on endosomal escape. Describe current and emerging methods for quantifying endosomal escape in vivo and their relative advantages and limitations.
  • Discuss strategies for targeted delivery to the retina and lungs, including receptor-mediated uptake and tissue-specific lipid nanoparticle (LNP) design.
  • Evaluate experimental data demonstrating improved delivery efficiency through optimized lipid chemistry
  • Apply principles from ocular and pulmonary targeting to conceptualize delivery strategies for other hard-to-reach tissues.