Skip to main content
NACFC 2025 Main banner
Icon Legend
This session is not in your schedule.
This session is in your schedule. Click again to remove it.
Presentation Icons
Livestreamed Session
Livestreamed Session

APPTT

PTAC/CFTR
Session Type: Workshop

W07--Agnostic Avenues: Alternatives to CF Gene Therapy

Thursday, October 23, 2025
10:15 AM - 12:15 PM PDT
Location:435

This workshop will contain presentations regarding alternative approaches to CFTR gene correction or replacement. Topics will include cellular mechanisms regulating ENaC activity and readthrough mechanisms, molecular prosthetics and synthetic anion transporters, and new approaches to deliver circular RNA. This workshop will discuss new basic science advances in these areas and examine how they may be therapeutically applied to alleviate CF pathophysiology.

Learning Objectives:

  • Identify alternative approaches to alleviate defective CFTR function that do not require CFTR gene correction or replacement.
  • Examine the ability to augment endogenous ion channel function and to restore anion movement through alternative ion channels.
  • Discuss technological approaches to manipulate endogenous cellular processes regulating protein function and to identify approaches for delivery of circular RNA.

Presentations:

  • 10:15 AM - 12:15 PM PDT
    W07.1- Elucidating the mechanism of ENaC activity reduction by CC-90009

    Speaker: Emily Bulik-Sullivan – UNC-Chapel Hill

  • 10:15 AM - 12:15 PM PDT
    W07.2- Novel readthrough compounds for the treatment of cystic fibrosis patients with nonsense mutations

    Speaker: Isabelle Sermet-Gaudelus – INSERM

  • 10:15 AM - 12:15 PM PDT
    W07.3- Pulmonary delivery of circular RNA for cystic fibrosis therapy

    Speaker: Zitong Wang, n/a – University of Michigan

  • 10:15 AM - 12:15 PM PDT
    W07.4- Molecular prosthetics restore anion movement in cystic fibrosis intestinal models

    Speaker: Nohemy Celis, n/a – University of Illinoit at Urbana-Champaign

  • 10:15 AM - 12:15 PM PDT
    W07.5- Restoration of anion transport to W1282X-CFTR expressing human bronchial epithelial cells by synthetic anion transporters

    Speaker: Yanira Manzanares Fornies – University of Bristol and Macquarie University