NACFC Features Gene and CFTR Therapies, and Potential New Directions for CF Care

The first plenary session at this year's North American Cystic Fibrosis Conference (NACFC) will focus on new research to develop cutting-edge therapies, including novel approaches for people with nonsense and ultra-rare mutations, as well as the challenges and opportunities that lie ahead as we work to develop novel genetic therapies. Plenary II will focus on what we can expect in the new era of highly effective modulator therapies and the challenges remaining to develop treatments for people with rare mutations. Plenary III will cover how CF care will change to meet the changing needs of the CF community.

Plenary I

Thursday, Oct. 31, 4:30-6 p.m. CT 

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, will review the power of genetic therapies, outline the obstacles and challenges that lie ahead, and discuss 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. 

Plenary II

In this session, Professor Jane Davies from the National Heart & Lung Institute, Imperial College in London, 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 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 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. 

Plenary III

Evolution of CF Care: Innovation & Impact
Saturday, Nov. 2, 9-10 a.m. CT

Peter Mogayzel, MD, PhD, from the Johns Hopkins School of Medicine, 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. Following his presentation, Sue Sullivan, RN, Oregon Health & Science University OHSU, Terri Laguna, MD, MSCS, from Ann and Robert H. Lurie Children's Hospital of Chicago, and Cindy Brown, MD, of Indiana University, will join Dr. Mogayzel for a panel discussion about the factors that should influence how CF clinical care evolves to support the needs of everyone with CF.