This workshop will focus on CFTR biology, from molecular structure/function to impact in disease. The session will introduce the earliest known CFTR by describing its structure and function. It will then be presented how proteostasis network profiling can help predict response to correctors. Additionally, there will be description of how the rate of translation of NBD1 can cause structural changes and affect function of mature CFTR protein. We will also learn about how CF airway epithelial cells exhibit altered localization of epithelial markers leading to a less differentiated and more proliferative state. Finally, a human pancreas-on-a-chip model will be presented as a way to study CF-related diabetes (CFRD).
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Note: Speaker Jae Seok Yoon's presentation was not recorded at his request.
This session highlights the current work in CF pulmonary disease. Discussions will include an exploration of oral versus intravenous antibiotic therapy for pulmonary exacerbations, barriers and outcomes of lung transplantation for advanced lung disease, and a look at a single center's experience in extracorporeal membrane oxygenation.
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This symposium will feature presentations on the clinical outcomes of existing CFTR modulators, focusing on pulmonary and non-pulmonary outcomes.
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Currently available modulator therapies target the defective CFTR protein to restore function. Patients with genotypes that are refractory to treatment with extant, and on the horizon, modulators are a challenge for these small molecule approaches - as nonsense and other changes can prevent production of the CFTR protein, which these modulators target. This symposium will focus on emerging science and technologies that point a way forward for this population. The theme will be to: 1) discuss the cells that require CFTR restoration, 2) new ways to deliver non-small molecule payloads to those cells, 3) methods to evaluate success, and 4) an example of such a non-traditional approach in development.
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Note: Speaker Daniel Seigwart's presentation was not recorded at his request.
Genetic-based therapies are discussed in the news nearly every day. These innovative technologies including RNA therapies, gene therapies and gene editing technologies are moving towards the clinical realm at an unexpected pace. They hold the key for 'a cure for all with CF.' However, moving from the promise of these technologies to the reality of effective therapy and cure for all will require time, tenacity and vision. This plenary will take us down that path to review the power of genetic therapies, outline the obstacles/challenges ahead as these therapies move from the pre-clinical space to clinical trials, and discuss what success looks like as we move to a one-time cure.
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In this session, Professor Jane Davies, will describe the progress and the promise of highly effective CFTR modulator therapies for people with CF. She will highlight the long-term benefits such therapy has 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 use 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 will be illustrated with pre-school and infant data. Whilst celebrating the success of highly effective modulator therapy, she will also discuss some of the challenges that remain, including developing treatments for people with rarer mutations and accessing CFTR modulators in certain regions of the world.
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Infants and young children with cystic fibrosis have abnormal lungs at birth, with congenital defects of the airways, mucus glands, and airway surface liquid already apparent. Infection and inflammation are independent, yet interrelated causes of early childhood pulmonary disease, even in the absence of symptoms. Mucolytics, anti-infectives, and anti-inflammatory therapies are all challenging to implement in treatment of infants and young children for a variety of reasons. In this session, attendees will learn about the latest updates in the use of anti-infectives, mucolytics, and anti-inflammatory strategies.
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Performing daily treatments is complex, time consuming and poses a significant daily challenge for many persons with CF. This session will explore methods allowing patients and health care teams to better achieve goals for care through assessment, collaboration, and technological advancement. These solutions will be all the more important as we encounter more specific therapeutic options in an expanding healthcare system.
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This workshop examines current challenges in the management of pulmonary exacerbations. Consideration for identification of exacerbations using the FEV-1 indicated exacerbation signal (FIES), treatment options related to treatment setting, gender, targeting anerobes, and the influences on recovery of baseline lung function will be presented as well as the role of patients and families in decision making related to exacerbation treatment.
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During this session, we will explore data supporting the development of therapies to address the basic defect in CF including gene editing, mRNA read-through, and CFTR protein modulation. The session will cover CFTR therapeutic development along the entire pathway from pre-clinical technical advancements and target identification to results of Phase I, II and III clinical trials.
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Note: Speaker Matthew Goddeeris' presentation was not recorded at his request.