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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 Session I: Thursday, October 31, 2019

    "Emerging Technologies for CFTR Restoration in All People With CF"

    Speaker: Marie Egan, M.D.

    Genetic therapies, including RNA therapies, gene therapies, and gene editing, hold the key to a cure for cystic fibrosis and are moving toward the clinical realm at an unexpected pace. Moving from the promise of these technologies to the reality of effective therapies for the underlying cause of the disease for all people with CF and a cure for CF will require time, tenacity, and vision.

    Marie Egan, MD, from the Yale School of Medicine, reviews the power of genetic therapies, outlines the obstacles and challenges that lie ahead, and discusses what success looks like as we move toward therapies that address the underlying defect for all people with CF and a cure for cystic fibrosis. 

    Topics: Gene Editing, Health Outcomes, CFTR, Plenary
  • Plenary Session II: Friday, November 1, 2019

    "Entering the Era of Highly Effective Modulator Therapy"

    Speaker: Jane C. Davies, M.D.

    In this session, Professor Jane Davies from the National Heart & Lung Institute, Imperial College in London, describes the progress and the promise of highly effective CFTR modulator therapy for people with CF. She highlights the long-term benefits such therapies have had on the relatively small proportion of the CF community for whom a highly effective modulator has been available for several years -- those with the gating mutation G551D who are on ivacaftor -- and uses this evidence to describe the impact we might expect from a triple-combination drug in a larger population. The additional opportunities posed by introducing CFTR functional restoration in early life is illustrated with pre-school and infant data.

    Topics: Health Outcomes, CFTR, Plenary
  • Plenary Session I: Thursday, October 18, 2018

    "Improving Outcomes of Infections in the Age of CFTR Modulators"

    Speaker: Lisa Saiman, M.D., M.P.H.

    In this session, Dr. Lisa Saiman will address the challenges of infections in people with CF and discuss why infections remain a major focus of the CF community, even in the era of effective CFTR modulators. She will provide an overview of CF infections, describe ongoing studies to increase our understanding of CF microorganisms and their optimal treatment, and discuss current strategies to develop new anti-infective agents to treat even the most difficult infections.

     

    Topics: Health Outcomes, Treatment, CFTR, Plenary, Clinical Research
  • Plenary Session II: Friday, October 19, 2018

    "Anti-inflammatories & Mucociliary Clearance Therapies in the Age of CFTR Modulators"

    Speaker: Felix Ratjen, M.D., Ph.D.

    This plenary will review the effect of CFTR dysfunction and CFTR restoration on both inflammation and mucociliary clearance in individuals with CF. Current and future therapeutic strategies to address inflammation and mucociliary clearance will be discussed, as well as how these strategies have potential to benefit both those patients using CFTR modulators and those not eligible for modulators.

    Topics: Health Outcomes, Treatment, CFTR, Plenary, Clinical Research
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