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PTAC/CFTR
Workshop

W29--Accelerating Progress Along the Path to a Cure

Friday, November 3, 2023
9:30 AM – 11:30 AM MST
Location:212 A-C (West)

Recorded Session

This session features pre-clinical research focused on developing therapies for CF. Topics include CFTR super-exon approaches, anti-CD20 treatment to attenuate immune responses associated with gene transfer, autologous stem cell gene therapy, CFTR mRNA delivery via nanoparticles, and picovectors delivering nonsense suppressor tRNA. These studies highlight important work done to advance the Path to a Cure mission to restore CFTR activity for all people with CF.

Learning Objectives:

  • Present new in vitro and in vivo models to test CFTR complementation.
  • Learn about immunosuppression for repeat delivery.
  • Discover RNA approaches for to restore CFTR.
  • Discuss gene delivery or repair options for the final 10% of people with CF who do not benefit from modulator treatment.

Presentations:

  • 9:30 AM – 11:30 AM MST
    W29.1- A CFTR super-exon strategy in W1282X intestinal organoids and mice

    Speaker: Craig A. Hodges, PhD (he/him/his) – CWRU

  • 9:30 AM – 11:30 AM MST
    W29.2- Anti-CD20 treatment prior to inhaled vector administration attenuates humoral and cellular immune responses and permits secondary gene transfer

    Speaker: Robert D E Clark (he/him/his) – Tulane University

  • 9:30 AM – 11:30 AM MST
    W29.3- Pre-clinical development of an autologous stem cell gene therapy platform for treatment of cystic fibrosis sinus/upper airway disease

    Speaker: Dawn T. Bravo, PhD – Stanford University School of Medicine

  • 9:30 AM – 11:30 AM MST
    W29.4- Delivery of CFTR mRNA using polymeric nanoparticles in a cystic fibrosis mouse model

    Speaker: Alannah Garrison – Yale University

  • 9:30 AM – 11:30 AM MST
    W29.5- Nonsense suppressor tRNA picovectors represent a new therapeutic cargo for the treatment of PTC-associated Cystic Fibrosis

    Speaker: Joseph J. Porter, PhD (he/him/his) – University of Rochester Medical Center