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CF Foundation | Challenges & Progress in the Pursuit of Genetic Therapies for Cystic Fibrosis

During plenary 1 of our North American Cystic Fibrosis Conference, the speakers explore different approaches to overcoming the biological obstacles and unique clinical trial challenges for developing cystic fibrosis genetic therapies. Daniel Siegwart, PhD, from the University of Texas Southwestern Medical Center, discusses how scientists are leveraging emerging research to address the significant biological hurdles, such as overcoming physical barriers like mucus, targeting the correct types of cells, and strategies for delivery of genetic medicines to the lungs. Joe Pilewski, MD, from the University of Pittsburgh, will highlight how new research advances and approaches in translational science are crucial for developing effective genetic therapies for CF, and how taking innovative approaches to clinical trials and community engagement are essential to success. Livestreamed from NACFC 2024 in Boston, Massachusetts September 26, 2024.