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PTAC/CFTR
Session Type: Symposium

S32--Gene Delivery: Targeted Nonviral Delivery to Airway Epithelial Cells | Room: 427-429

Saturday, October 25, 2025
10:15 AM - 12:15 PM PDT
Location:427-429

Recorded Session

The development of effective genetic therapies for CF hinges on successful delivery to airway epithelial cells, the principal sites of lung pathology. This session delves into nonviral systems that aim to overcome the specific challenges associated with targeting therapeutic delivery to these cells. Attendees will gain insights into the latest advancements in the design and optimization of nonviral vectors for CF therapeutics, including the selection of materials (ex., lipids or polymers) and targeting ligands to enhance cellular uptake and transfection. The session will also cover strategies to address physiological barriers and promote sustained therapeutic benefits. Overall, this session will highlight the promise of nonviral approaches, address current limitations, and outline the potential directions for translating these strategies into clinical applications for CF.

Learning Objectives:

  • Understand the design considerations for nonviral delivery systems: learn about the engineering and optimization of nonviral vectors aimed at targeting airway epithelial cells in CF, including the use of novel materials and targeting ligands
  • Evaluate the advancements in nonviral delivery methods that improve targeting and efficacy in airway epithelial cells and their potential for translation into clinical therapies
  • Identify the current limitations of nonviral delivery systems and assess strategies to overcome physiological barriers to effective genetic therapeutic delivery

Presentations:

  • 10:15 AM - 12:15 PM PDT
    S32.1- SORT Lipids

    Speaker: Daniel Siegwart, PhD – University of Texas Southwestern Medical Center

  • 10:15 AM - 12:15 PM PDT
    S32.2- Peptide Targeted LNPs

    Speaker: Gaurav Sahay, PhD – college of pharmacy- Oregon state university

  • 10:15 AM - 12:15 PM PDT
    S32.3- Surface Functionalized Polymeric Nanoparticles for Cell-Specific Delivery of Genetic Therapies

    Speaker: Alexandra Piotrowski-Daspit, PhD – University of Michigan

  • 10:15 AM - 12:15 PM PDT
    S32.4- Peptide Decorated LNPs for Targeted Airway Epithelial Delivery

    Speaker: Stephen L. Hart – Department of Genetics and Genomic Medicine, UCL Great Ormond Street Institute of Child Health,London, UK