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  • Plenary Session I: Wednesday, October 21, 2020

    Laying the Foundation for the Path to a Cure

    Speakers: Brian R. Davis, Ph.D. and Maria P. Limberis, Ph.D.

    To lay the foundation for the Path to a Cure, an ambitious initiative to accelerate treatments for the underlying cause of the disease and a cure for cystic fibrosis, we need to understand the cells to target and the challenges of delivering genetic-based therapies (such as gene editing and gene therapy).

    In this plenary, Brian Davis, Ph.D. from the University of Texas Health Science Center at Houston, will focus on the importance of understanding the underlying cellular architecture and biology of the epithelial tissues (e.g., the lungs) that must be treated. Maria Limberis, Ph.D. from the University of Pennsylvania, will explore the complex physical properties of the lungs that make the surface of the airway an effective barrier against the viral and bacterial pathogens, but that also make the successful delivery of genetic-based therapies more difficult.

    Their presentations will provide insight into the current status of promising state-of-the-are genetic-based therapies as well as put in context a common vocabulary and conceptual framework as we move forward with developing a cure for all.

    Topics: Gene Editing, CFTR, Plenary
  • Plenary Session II: Thursday, October 22, 2020

    Defining the New CF in the Era of Highly Effective Modulators

    Speaker: Jennifer L. Taylor-Cousar, M.D., MSCS, ATS

    In this session, the latest research into highly effective CFTR modulators will be highlighted, providing and in-depth overview of the CFTR modulator research landscape one-year post-approval of Trikafta. The speaker will describe the ongoing and upcoming studies looking at the holistic impact of highly effective CFTR modulation on the lives of people with CF. Finally, there will be a review of on-going and upcoming studies for the development of new modulators as well as the potential impact of modulators on complications.

    Topics: Health Outcomes, Treatment, CFTR, Plenary, Clinical Research
  • Plenary III: Friday, October 23, 2020

    Advancing GI Frontier for People with CF

    Speaker: Steven D. Freedman, M.D., Ph.D.

    In this session, Dr. Freedman walks us through the gastrointestinal aspects of CF, focusing on intestinal, liver, and pancreatic disease and the exciting new directions in GI as part of specialized CF care. Research initiatives and advances in developing a GI workforce through the CF Foundation's DIGEST program will be highlighted.

    Topics: Treatment, CF Complications, Plenary, Clinical Research
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