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Thursday

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.

Learning Objectives:

  • Review emerging genetic therapies and the promise they hold. 
  • Discuss/Describe the unique challenges we face regarding delivering these therapies in CF.
  • Discuss/Describe the incredible advantages and resources the CF community will harness to overcome obstacles.

Friday

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.

Learning Objectives:

  • Summarize the progress to date in CFTR modulator development, including the recent successful Phase 3 trials of triple-combination therapies for people with either a single copy of the F508del mutation (heterozygous) or two copies of F508del (homozygous). 
  • Illustrate the long-term benefits of highly effective modulator therapies, particularly the opportunities of initiating treatment early, by referencing the use of ivacaftor in people with gating mutations.  
  • Identify future challenges in ensuring that highly effective treatments are available to all people with CF.

    Saturday

    We count our success in lives, per Brent James, this is how we measure the quality of care we provide people with CF. In this plenary, Dr. Peter Mogayzel will walk us through past challenges and how our care model has met those challenges and built upon what we've learned to prepare for the future. As we enter a new era of therapeutics, now is the moment to consider what should shape how CF care evolves to meet the changing needs of people with CF and their families.

    Learning Objectives:

    • Recall how CF care has evolved to meet past challenges.
    • Describe foundations of the CF care model that have enabled us to meet past challenges.
    • Identify key factors to help shape how CF care evolves to meet the changing needs of patients and their families.