PTAC/CFTR
Deepika Polineni, MD, MPH (she/her/hers)
Washington University School of Medicine in St. Louis
Paul McCray, Jr., MD (he/him/his)
University of Iowa
US CFF Patient Registry data support that at least 6% of the US CF population is unable to benefit from CFTR modulators on the basis of genetic ineligibility, and yet higher numbers are estimated when also factoring CFTR modulator intolerance or global genetic diversity of CF. Therapies aimed at addressing restoration of CFTR ion transport are needed for all people with CF. This session will review past clinical studies and recent advances in gene transfer and molecular approaches for restoration of CFTR, critically assessing successful milestones and challenges. Presenters will outline four approaches to addressing CFTR ion transport, including review of new considerations for outcomes and endpoints in testing nucleotide-based interventions for people with CF. This session is aimed at providing a review of important “lessons learned” regarding best practices and future considerations for clinical trials in CF genetic therapies.
Speaker: Joseph Zabner, MD (he/him/his) – University of Iowa Carver College of Medicine
Speaker: Jennifer L. Taylor-Cousar, MD, MSCS – National Jewish Health
Speaker: Batsheva Kerem, PhD (she/her/hers) – Hebrew University
Speaker: Martin D. Burke, PhD, MD (he/him/his) – University of Illinois