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PTAC/CFTR
Symposium

S15--Challenges to Efficient, Effective, & Durable CFTR Rescue

Friday, September 27, 2024
2:30 PM – 4:30 PM ET
Location:157 A-C

Recorded Session

This symposium aims to increase the awareness and understanding of the substantial challenges to delivering effective genetic therapies to persons with CF. From a review of the anatomy of the lung and airways, including extracellular barriers that need to be navigated by aerosol or intravenous delivery technologies, the presentations will become increasingly more granular. Airway cell types will be discussed that need to be targeted by transient therapies, like CFTR mRNA or ASOs, or permanent curative therapies. The last two presentations will describe the two key intracellular challenges, endosomal escape and delivery to the nucleus, and approaches to overcoming these barriers will be proposed.

Learning Objectives:

  • Describe the anatomical, extracellular and intracellular barriers in the human lung which impact the delivery efficiency of a genetic therapy agents delivered either as aerosol or intravenously.
  • Summarize and functionally evaluate the relevant target cells in the cystic fibrosis lung for targeted delivery of gene therapies, be it gene replacement or gene editing strategies.
  • Explain delivery strategies and approaches to overcome the extra- and intracellular barriers to nucleic acid-based therapies will be reviewed and discussed.

Presentations:

  • 2:30 PM – 4:30 PM ET
    S15.1- Respiratory Tract Anatomy & Challenges of Targeted Gene Editing

    Speaker: David K. Meyerholz, DVM, PhD, Dipl. ACVP & ACVM (he/him/his) – The University of Iowa

  • 2:30 PM – 4:30 PM ET
    S15.2- Identifying & Culturing Novel Stem/Progenitor Cells of the Airway for Gene Delivery Targeting & Disease Modeling

    Speaker: Brigitte Gomperts, MD (she/her/hers) – UCLA

  • 2:30 PM – 4:30 PM ET
    S15.3- Strategies to Overcome the Endosomal Escape Bottleneck for Genetic Medicine Delivery

    Speaker: Anders Wittrup, MD, PhD – Lund University

  • 2:30 PM – 4:30 PM ET
    S15.4- Peptide-enabled Strategies for Intracellular Delivery of Gene Therapies

    Speaker: Millie Sullivan, PhD (she/her/hers) – University of Delaware