Archives

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.

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.

Advancing GI Frontier for People with CF

Speaker: Steven D. Freedman, M.D., Ph.D.

In this session, Dr. Freedman walks us through the gastrointestinal aspects of CF, focusing on intestinal, liver, and pancreatic disease and the exciting new directions in GI as part of specialized CF care. Research initiatives and advances in developing a GI workforce through the CF Foundation's DIGEST program will be highlighted.

"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. 

"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.

"Evolution of CF Care: Innovation & Impact"

Speaker: Peter Mogayzel, M.D., Ph.D.

In this session, Dr. Peter Mogayzel walks 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, from Oregon Health & Science University; Terri Laguna, MD, MSCS, from Ann and Robert H. Lurie Children's Hospital of Chicago; and Cindy Brown, MD, of Indiana University, 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.