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PTAC/CFTR
Symposium

S07--CFTR 2024: Better Understanding of the Underlying CF Disease Mechanism

Thursday, September 26, 2024
2:30 PM – 4:30 PM ET
Location:104 C

Recorded Session

An in-depth, molecular definition of the CFTR folding pathway proved critical for the development of drugs that improve F508del-CFTR biogenesis and function. In this session, recent data will be presented highlighting an even deeper understanding of CFTR synthesis; drugs that alter the folding trajectory and/or target unique regions in CFTR; impact of recently unearthed mutations on CFTR maturation and activity; and new methods that alter translation machinery to ‘correct’ premature stop codons in the gene encoding CFTR.

Learning Objectives:

  • Summarize advancements in understanding of CFTR protein biosynthesis, channel gating properties, and pharmacologic interactions.
  • Describe novel strategies for addressing divergent theratypes of rare CFTR variants.
  • Assess efficacy of new therapies designed to rescue CFTR nonsense alleles.

Presentations:

  • 2:30 PM – 4:30 PM ET
    S07.1- The CFTR Folding Pathway Explains Why Modulators Act on Multitude of Variants

    Speaker: Ineke Braakman, PhD (she/her/hers) – Faculty of Science, Utrecht University

  • 2:30 PM – 4:30 PM ET
    S07.2- Unifying Principles for the Variant-Specific Effects of Correctors

    Speaker: Jonathan Schlebach, PhD (he/him/his) – Purdue University

  • 2:30 PM – 4:30 PM ET
    S07.3- Understanding the Functional Defects of CFTR Variants & Their Pharmacological Rescue in a Native Cell Model

    Speaker: Nicoletta Pedemonte, PhD – IRCCS Istituto Giannina Gaslini

  • 2:30 PM – 4:30 PM ET
    S07.4- Stop Codon Readthrough as a Therapeutic Approach to CF Attributable to Nonsense Mutations

    Speaker: Allan Jacobson, PhD – UMass Chan Medical School