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PTAC/CFTR
Workshop

W05--Advances Along the Path to a Cure

Thursday, September 26, 2024
10:15 AM – 12:15 PM ET
Location:210 A-C

The Cystic Fibrosis Foundation's Path to a Cure initiative aims to accelerate the discovery and development of breakthrough therapies to cure cystic fibrosis (CF). This session focuses on innovative strategies that advance this mission. We will explore several gene delivery methods, including DNA nanocarriers, nanoparticle systems, and viral vectors that demonstrate promise in both in vitro and in animal models of CF. Additionally, the session will highlight promising therapeutic approaches such as CFTR super exons and splice-switching antisense oligonucleotides. Overall, the session aims to emphasize progress and identify key challenges along the path to a cure for all individuals with CF.

Learning Objectives:

  • Understand the potential and challenges of various gene delivery methods for CFTR, including DNA nanocarriers, nanoparticles, and viral vectors; describe recent advancements in therapeutic approaches aimed at enhancing CFTR function, such as CFTR super exons and splice-switching antisense oligonucleotides.
  • Evaluate the positive and negative attributes of therapeutic strategies and their potential for translation into clinical therapies.
  • Identify the key physiological and technical barriers in delivering gene therapies for CF and describe future directions to overcome these challenges.

Presentations:

  • 10:15 AM – 12:15 PM ET
    In vitro delivery of CFTR gene cargo using DNA nanocarriers

    Speaker: Isabella Ferranti (she/her/hers) – Carnegie Mellon University

  • 10:15 AM – 12:15 PM ET
    Optimized CFTR E2-27 super exon cargos rescue CFTR function

    Speaker: Kezhi Yan, PhD – Cystic Fibrosis Foundation

  • 10:15 AM – 12:15 PM ET
    Splice-switching antisense oligonucleotides to recover CFTR function disrupted by C-terminal truncating nucleotide variants

    Speaker: Wren Michaels, PhD (she/her/hers) – Rosalind Franklin University of Medicine and Science

  • 10:15 AM – 12:15 PM ET
    W05.4- AAV1 or 6-Δ27-264 CFTR successfully ameliorated clinical symptoms in G551D Ferrets.

    Speaker: Liudmila Cebotaru, MD (she/her/hers) – Johns Hopkins University

  • 10:15 AM – 12:15 PM ET
    W05.5- Poly(amine-co-ester) (PACE) nanoparticle delivery of CFTR mRNA shows restoration of CFTR activity in cystic fibrosis airway models.

    Speaker: Ju Hyun Lee, MM, MPhil, MS, MPhil – Yale University