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.
Learning Objectives:
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.
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